Galmed Pharmaceuticals Ltd 6-K, filed Aug 6, 2020
Galmed Pharmaceuticals Ltd. (EOCN)
About this 6-K
Galmed Pharmaceuticals Ltd filed this 6-K on August 6, 2020.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
Report of Foreign Private Issuer Pursuant to Rule 13a-16 or 15d-16
Under the Securities Exchange Act of 1934
For the Month of August 2020
001-36345
(Commission File Number)
GALMED PHARMACEUTICALS LTD.
(Exact name of Registrant as specified in its charter)
16 Tiomkin St.
Tel Aviv 6578317, Israel
(Address of principal executive offices)
Indicate by check mark whether the registrant files or will file annual reports under cover Form 20-F or Form 40-F.
Form 20-F x Form 40-F ¨
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ¨
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ¨
This Form 6-K contains the quarterly report of Galmed Pharmaceuticals Ltd. (the “Company”), which includes the Company’s unaudited consolidated financial statements for the three and six months ended June 30, 2020, together with related information and certain other information. The Company is not subject to the requirements to file quarterly or certain other reports under Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended. The Company does not undertake to file or cause to be filed any such reports in the future, except to the extent required by law.
On August 6, 2020, the Company issued a press release announcing the filing of its financial results for the three and six months ended June 30, 2020 with the Securities and Exchange Commission. A copy of the press release is attached hereto as Exhibit 99.1 and is incorporated herein by reference.
In addition, on August 6, 2020, the Company issued a press release announcing updated information on the Company’s pipeline program, Amilo-5Mer. A copy of the press release is attached hereto as Exhibit 99.2 and is incorporated herein by reference. The Company has made available a corporate presentation with respect to Amilo-5Mer on its website. A copy of the corporate presentation is furnished as Exhibit 99.3 and incorporated by reference herein.
This Form 6-K, the text under the heading “Recent Clinical & Scientific Developments” and “Financial Summary - Second Quarter 2020 vs. Second Quarter 2019” in Exhibit 99.1, and paragraphs one through ten and paragraph thirteen of the press release in Exhibit 99.2 are incorporated by reference into the Company’s Registration Statement on Form S-8 (Registration No. 333-206292 and 333-227441) and the Company’s Registration Statement on Form F-3 (Registration No. 333-223923).
FINANCIAL INFORMATION
Financial Statements
| GALMED PHARMACEUTICALS LTD. |
| Consolidated Balance Sheets (Unaudited) |
| U.S. Dollars in thousands, except share data and per share data |
As of June 30, 2020 | As of December 31, 2019 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 5,553 | $ | 15,931 | ||||
| Restricted Cash | 113 | 112 | ||||||
| Short-term deposits | 27,406 | 27,938 | ||||||
| Marketable debt securities | 30,470 | 31,622 | ||||||
| Other receivable | 669 | 827 | ||||||
| Total current assets | 64,211 | 76,430 | ||||||
| Right of use assets | 481 | 538 | ||||||
| Property and equipment, net | 157 | 171 | ||||||
| Total non-current assets | 638 | 709 | ||||||
| Total assets | $ | 64,849 | $ | 77,139 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities | ||||||||
| Trade payables | $ | 3,876 | $ | 5,999 | ||||
| Other payables | 838 | 935 | ||||||
| Total current liabilities | 4,714 | 6,934 | ||||||
| Non-current liabilities | ||||||||
| Lease obligation | $ | 251 | $ | 352 | ||||
| Total non-current liabilities | 251 | 352 | ||||||
| Ordinary shares par value NIS 0.01 per share; Authorized 50,000,000; Issued and outstanding: 21,153,166 shares as of June 30, 2020; 21,139,385 shares as of December 31, 2019 | 58 | 58 | ||||||
| Additional paid-in capital | 177,853 | 176,696 | ||||||
| Accumulated other comprehensive gain | 498 | 35 | ||||||
| Accumulated deficit | (118,525 | ) | (106,936 | ) | ||||
| Total stockholders' equity | 59,884 | 69,853 | ||||||
| Total liabilities and stockholders' equity | $ | 64,849 | $ | 77,139 | ||||
The accompanying notes are an integral part of the interim consolidated financial statements.
| GALMED PHARMACEUTICALS LTD. |
| Consolidated Statements of Operations (Unaudited) |
| U.S. Dollars in thousands, except share data and per share data |
| Three months ended June 30, | Six months ended June 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Research and development expenses | 4,971 | 3,494 | 10,521 | 6,763 | ||||||||||||
| General and administrative expenses | 845 | 1,207 | 1,757 | 1,978 | ||||||||||||
| Total operating expenses | 5,816 | 4,701 | 12,278 | 8,741 | ||||||||||||
| Financial income, net | 290 | 532 | 689 | 1,080 | ||||||||||||
| Net loss | $ | 5,526 | $ | 4,169 | $ | 11,589 | $ | 7,661 | ||||||||
| Basic and diluted net loss per share | $ | 0.26 | $ | 0.20 | $ | 0.55 | $ | 0.36 | ||||||||
| Weighted-average number of shares outstanding used in computing basic and diluted net loss per share | 21,153,166 | 21,120,085 | 21,152,003 | 21,102,306 | ||||||||||||
The accompanying notes are an integral part of the interim consolidated financial statements.
| GALMED PHARMACEUTICALS LTD. |
| Consolidated Statements of Comprehensive Loss (Unaudited) |
| U.S. Dollars in thousands |
| Three months ended June 30, | Six months ended June 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Net loss | $ | 5,526 | $ | 4,169 | $ | 11,589 | $ | 7,661 | ||||||||
| Other comprehensive loss: | ||||||||||||||||
| Net unrealized gain on available for sale securities | (725 | ) | (16 | ) | (463 | ) | (52 | ) | ||||||||
| Comprehensive loss | $ | 4,801 | $ | 4,153 | $ | 11,126 | $ | 7,609 | ||||||||
The accompanying notes are an integral part of the interim consolidated financial statements.
| GALMED PHARMACEUTICALS LTD. |
| Consolidated Statements of Changes in Stockholders’ Equity (Unaudited) |
| U.S. Dollars in thousands, except share data and per share data |
| Ordinary shares | Additional paid-in | Accumulated other Comprehensive | Accumulated | |||||||||||||||||||||
| Shares | Amount | capital | loss | Deficit | Total | |||||||||||||||||||
| Balance - December 31, 2018 | 21,018,919 | $ | 58 | $ | 174,322 | $ | (11 | ) | $ | (86,475 | ) | $ | 87,894 | |||||||||||
| Stock-based compensation | - | - | 416 | - | - | 416 | ||||||||||||||||||
| Exercise of options and restricted stock units | 94,147 | (* | ) | 74 | - | - | 74 | |||||||||||||||||
| Unrealized gain from marketable debt securities | - | - | - | 36 | - | 36 | ||||||||||||||||||
| Net loss | - | - | - | - | (3,492 | ) | (3,492 | ) | ||||||||||||||||
| Balance - March 31, 2019 | 21,113,066 | $ | 58 | $ | 174,812 | $ | 25 | $ | (89,967 | ) | $ | 84,928 | ||||||||||||
| Stock-based compensation | - | - | 591 | - | - | 591 | ||||||||||||||||||
| Exercise of options and restricted stock units | 8,271 | (* | ) | 21 | - | - | 21 | |||||||||||||||||
| Unrealized gain from marketable debt securities | - | - | - | 16 | - | 16 | ||||||||||||||||||
| Net loss | - | - | - | - | (4,169 | ) | (4,169 | ) | ||||||||||||||||
| Balance - June 30, 2019 | 21,121,337 | $ | 58 | $ | 175,424 | $ | 41 | $ | (94,136 | ) | $ | 81,387 | ||||||||||||
| Ordinary shares | Additional paid-in | Accumulated other Comprehensive | Accumulated | |||||||||||||||||||||
| Shares | Amount | capital | loss | Deficit | Total | |||||||||||||||||||
| Balance - December 31, 2019 | 21,139,385 | $ | 58 | $ | 176,696 | $ | 35 | $ | (106,936 | ) | $ | 69,853 | ||||||||||||
| Stock based compensation | - | - | 515 | - | - | 515 | ||||||||||||||||||
| Exercise of options and restricted stock units | 13,781 | - | 61 | - | - | 61 | ||||||||||||||||||
| Unrealized loss from marketable debt securities | - | - | - | (262 | ) | - | (262 | ) | ||||||||||||||||
| Net loss | - | - | - | - | (6,063 | ) | (6,063 | ) | ||||||||||||||||
| Balance - March 31, 2020 | 21,153,166 | $ | 58 | $ | 177,272 | $ | (227 | ) | $ | (112,999 | ) | $ | 64,104 | |||||||||||
| Stock-based compensation | - | - | 581 | - | - | 581 | ||||||||||||||||||
| Unrealized gain from marketable debt securities | - | - | - | 725 | - | 725 | ||||||||||||||||||
| Net loss | - | - | - | - | (5,526 | ) | (5,526 | ) | ||||||||||||||||
| Balance - June 30, 2020 | 21,153,166 | $ | 58 | $ | 177,853 | $ | 498 | $ | 118,525 | $ | 59,884 | |||||||||||||
The accompanying notes are an integral part of the interim consolidated financial statements.
| GALMED PHARMACEUTICALS LTD. |
| Consolidated Statements of Cash Flows (Unaudited) |
| U.S. Dollars in thousands |
| Six months ended June 31, |
||||||||
| 2020 | 2019 | |||||||
| Cash flow from operating activities | ||||||||
| Net loss | $ | (11,589 | ) | $ | (7,661 | ) | ||
| Adjustments required to reconcile net loss to net cash used in operating activities | ||||||||
| Depreciation and amortization | 19 | 18 | ||||||
| Stock-based compensation expense | 1,096 | 1,007 | ||||||
| Amortization of premium (discount) on marketable debt securities | 16 | (215 | ) | |||||
| Interest income from short-term deposits | (268 | ) | (71 | ) | ||||
| Gain from realization of marketable debt securities | (10 | ) | (9 | ) | ||||
| Changes in operating assets and liabilities: | ||||||||
| Decrease (increase) in other accounts receivable | 158 | (487 | ) | |||||
| Decrease in trade payables | (2,123 | ) | 764 | |||||
| Decrease in other accounts payable | (141 | ) | (349 | ) | ||||
| Net cash used in operating activities | (12,842 | ) | (7,003 | ) | ||||
| Cash flow from investing activities | ||||||||
| Purchase of property and equipment | (5 | ) | (4 | ) | ||||
| Investment in available for sale securities | (26,979 | ) | (68,717 | ) | ||||
| Investment in short term deposits | (4,000 | ) | (9,000 | ) | ||||
| Maturity of short term deposits | 4,800 | - | ||||||
| Consideration from sale of available for sale securities | 28,588 | 86,248 | ||||||
| Net cash provided by investing activities | 2,404 | 8,527 | ||||||
| Cash flow from financing activities | ||||||||
| Proceeds from exercise of options | 61 | 95 | ||||||
| Net cash provided in financing activities | 61 | 95 | ||||||
| Increase (decrease) in cash and cash equivalents and restricted cash | (10,377 | ) | 1,619 | |||||
| Cash and cash equivalents and restricted cash at the beginning of the period | 16,043 | 24,159 | ||||||
| Cash and cash equivalents and restricted cash at the end of the period | $ | 5,666 | $ | 25,778 | ||||
| Supplemental disclosure of cash flow information: | ||||||||
| Cash received from interest | $ | 317 | $ | 1,057 | ||||
| Non-cash transactions: | ||||||||
| Recognition of right-of-use asset and lease liability from adoption of ASU 2016-02 | $ | 35 | 679 | |||||
The accompanying notes are an integral part of the interim consolidated financial statements.
| GALMED PHARMACEUTICALS LTD. |
| Notes to Consolidated Financial Statements |
Note 1 - Basis of presentation
Galmed Pharmaceuticals Ltd. (the “Company”) is a clinical-stage biopharmaceutical company primarily focused on the development of therapeutics for the treatment of liver diseases. The Company was incorporated in Israel on July 31, 2013 and commenced operations on February 2, 2014. The Company holds a wholly-owned subsidiary, Galmed International Ltd., which was incorporated in Malta. Galmed International Ltd. previously held a wholly-owned subsidiary, Galmed Medical Research Ltd., which was liquidated during the first quarter of 2019. The Company also holds two additional wholly-owned subsidiaries, Galmed Research and Development Ltd and Galtopa Therapeutics Ltd., both of which are incorporated in Israel.
These unaudited interim consolidated financial statements have been prepared as of June 30, 2020 and for the three and six months period then ended. Accordingly, certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. These unaudited interim consolidated financial statements should be read in conjunction with the audited financial statements and the accompanying notes of the Company for the year ended December 31, 2019 that are included in the Company's Annual Report on Form 20-F, filed with the Securities and Exchange Commission on March 12, 2020 (the "Annual Report"). The results of operations presented are not necessarily indicative of the results to be expected for the year ending December 31, 2020.
Note 2 - Summary of significant accounting policies
The significant accounting policies that have been applied in the preparation of the unaudited consolidated interim financial statements are identical to those that were applied in preparation of the Company’s interim most recent annual financial statements in connection with its Annual Report on Form 20-F.
In June 2016, FASB issued ASU No. 2016-13, “Financial Instruments – Credit Losses – Measurement of Credit Losses on Financial Instruments”, which introduces a model based on expected losses to estimate credit losses for most financial assets and certain other instruments. In addition, for available-for-sale debt securities with unrealized losses, the losses will be recognized as allowances rather than reductions in the amortized cost of the securities. The ASU is effective for the Company in the first quarter of 2020, with early adoption permitted. The adoption of the standard did not have a material effect on the Company’s interim consolidated financial statements.
In August 2018, the FASB issued ASU 2018-13, “Changes to Disclosure Requirements for Fair Value Measurements”, which will improve the effectiveness of disclosure requirements for recurring and nonrecurring fair value measurements. The standard removes, modifies, and adds certain disclosure requirements, and is effective for the Company beginning on January 1, 2020. The adoption of the standard did not have a material effect on the Company’s consolidated financial statements.
Note 3 - Stockholders' Equity
| 1. | During the six months ended June 30, 2020, former employees exercised options into 11,000 ordinary shares of the Company, NIS 0.01 par value per share, for total consideration of $61 thousand. |
| 2. | During the six months ended June 30, 2020, restricted stock units held by certain officers, employees and former employees vested resulting in the issuance of 2,781 ordinary shares of the Company, NIS 0.01 par value per share. |
| 3. | In March 2020, the Company granted options to purchase 67,500 ordinary shares of the Company to several employees. The options are exercisable at $4.21 per share, have a 10-year term and vest over a period of four years. The aggregate grant date fair value of such options was approximately $0.2 million. |
| 4. | On May 15, 2020, the Company amended and restated the Sales Agreement dated December 22, 2017 between the Company and Stifel, Nicolaus & Company, Incorporated to include Cantor Fitzgerald & Co. as an additional sales agent for the Company’s “at the market offering” program (the “A&R Sales Agreement”). Pursuant to a prospectus supplement filed with the SEC on May 15, 2020, the Company may offer and sell $31.9 million of its ordinary shares. During July 2020, the Company sold 136,300 ordinary shares under the A&R Sales Agreement for total net proceeds of approximately $0.8 million. |
Management’s Discussion and Analysis of Financial Condition and Results of Operations
All references to “we,” “us,” “our,” “the Company” and “our Company”, in this Form 6-K are to Galmed Pharmaceuticals Ltd. and its subsidiaries, unless the context otherwise requires. All references to “shares” or “ordinary shares” are to our ordinary shares, NIS 0.01 nominal par value per share. All references to “Israel” are to the State of Israel. “U.S. GAAP” means the generally accepted accounting principles of the United States. Unless otherwise stated, all of our financial information presented in this Form 6-K has been prepared in accordance with U.S. GAAP. Any discrepancies in any table between totals and sums of the amounts and percentages listed are due to rounding. Unless otherwise indicated, or the context otherwise requires, references in this Form 6-K to financial and operational data for a particular year refer to the fiscal year of our company ended December 31 of that year.
Our reporting currency and financial currency is the U.S. dollar. In this Form 6-K, “NIS” means New Israeli Shekel, and “$,” “US$” and “U.S. dollars” mean United States dollars.
Cautionary Note Regarding Forward-Looking Statements
This Form 6-K contains forward-looking statements about our expectations, beliefs or intentions regarding, among other things, our product development efforts, business, financial condition, results of operations, strategies or prospects. In addition, from time to time, we or our representatives have made or may make forward-looking statements, orally or in writing. Forward-looking statements can be identified by the use of forward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should,” “anticipate,” “could,” “might,” “seek,” “target,” “will,” “project,” “forecast,” “continue” or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. These forward-looking statements may be included in, among other things, various filings made by us with the SEC, press releases or oral statements made by or with the approval of one of our authorized executive officers. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward-looking statements. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including, but not limited to, the factors summarized below:
| · | the timing and cost of our pivotal Phase 3 ARMOR trial, or the ARMOR Study, for our product candidate, Aramchol, or for any other pre-clinical or clinical trials; | |
| · | completion and receiving favorable results of the ARMOR Study for Aramchol or any other pre-clinical or clinical trial; | |
| · | the impact of the coronavirus outbreak on our operations; |
| · | regulatory action with respect to Aramchol or any other product candidate by the U.S. Food and Drug Administration, or the FDA, or the European Medicines Authority, or EMA, including but not limited to acceptance of an application for marketing authorization, review and approval of such application, and, if approved, the scope of the approved indication and labeling; |
| · | the commercial launch and future sales of Aramchol and any future product candidates; |
| · | our ability to comply with all applicable post-market regulatory requirements for Aramchol or any other product candidate in the countries in which we seek to market the product; |
| · | our ability to achieve favorable pricing for Aramchol or any other product candidate; |
| · | our expectations regarding the commercial market for non-alcoholic steato-hepatitis, or NASH, in patients or any other targeted indication; | |
| · | third-party payor reimbursement for Aramchol or any other product candidate; |
| · | our estimates regarding anticipated capital requirements and our needs for additional financing; |
| · | market adoption of Aramchol or any other product candidate by physicians and patients; |
| · | the timing, cost or other aspects of the commercial launch of Aramchol or any other product candidate; |
| · | our ability to obtain and maintain adequate protection of our intellectual property; |
| · | the possibility that we may face third-party claims of intellectual property infringement; |
| · | our ability to manufacture our product candidates in commercial quantities, at an adequate quality or at an acceptable cost; |
| · | our ability to establish adequate sales, marketing and distribution channels; |
| · | intense competition in our industry, with competitors having substantially greater financial, technological, research and development, regulatory and clinical, manufacturing, marketing and sales, distribution and personnel resources than we do; |
| · | the development and approval of the use of Aramchol or any other product candidate for additional indications or in combination therapy; and |
| · | our expectations regarding licensing, acquisitions and strategic operations. |
We believe these forward-looking statements are reasonable; however, these statements are only current predictions and are subject to known and unknown risks, uncertainties and other factors that may cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking statements. We discuss many of these risks in our Annual Report on Form 20-F for the year ended December 31, 2019 filed with the SEC on March 12, 2020 and in our Report on Form 6-K filed with the SEC on May 14, 2020 in greater detail under the heading “Risk Factors” and elsewhere in the Annual Report and this Form 6-K. Given these uncertainties, you should not rely upon forward-looking statements as predictions of future events.
All forward-looking statements attributable to us or persons acting on our behalf speak only as of the date hereof and are expressly qualified in their entirety by the cautionary statements included in this report. We undertake no obligations to update or revise forward-looking statements to reflect events or circumstances that arise after the date made or to reflect the occurrence of unanticipated events. In evaluating forward-looking statements, you should consider these risks and uncertainties.
Overview
We are a clinical-stage biopharmaceutical company focused on the development of Aramchol, a liver targeted stearoyl-coenzyme A desaturase-1, or SCD1, modulator, first in class, novel, oral therapy for the treatment of NASH for variable populations. In September 2019, we initiated our Phase 3 pivotal ARMOR Study to evaluate the efficacy and safety of Aramchol in subjects with NASH and fibrosis. We are also collaborating with the Hebrew University in the development of Amilo-5MER, a 5 amino acid synthetic peptide and plan to initiate a first in human study by the fourth quarter of 2020.
Impact of COVID-19 on our Operations
In late 2019, a novel strain of COVID-19, also known as coronavirus, was reported in Wuhan, China. Initially the outbreak was largely concentrated in China, but it rapidly spread to countries across the globe, including in Israel and the United States. Many countries around the world, including in Israel and the United States, implemented significant governmental measures to control the spread of the virus, including temporary closure of businesses, severe restrictions on travel and the movement of people, and other material limitations on the conduct of business. In response, we implemented remote working and workplace protocols for our employees in accordance Israeli Ministry of Health requirements to ensure employee safety. Many of our trial sites in our ARMOR Study are based in areas currently affected by coronavirus and there is a general unease opening of conducting scheduled or elective procedures in medical centers. Given the significant strains on the healthcare system across the globe, we temporarily halted the screening of new patients for the ARMOR Study and temporarily suspended the opening of new trial sites. We continue to monitor the local situation closely in the U.S. and other countries around the world and have begun to resume activity on a country by country, state by state and site by site basis. Accordingly, we have lifted some of the constraints in the U.S. in states identified as “green states” allowing individual investigators to determine whether it is safe to resume screening activities and recruitment and we have opened sites in eight additional countries, including Korea, Turkey, Belgium, France, Spain, Canada, Mexico and Chile. We have also been using the time to advance the opening of new sites so that they are ready for activation when screening and randomization will be possible. To help mitigate cost overrun, we previously took several cost reduction measures including minimizing clinical related expenses, making certain adjustments to clinical staff and pay according to the current and predicted level of activity, and reducing directors’ cash fees by 50% for the first half of 2020.
It is still too early to assess the full impact of the coronavirus outbreak on the ARMOR Study, but our current assessment is that by the fourth quarter of 2020, we should be able to resume recruitment in most of our sites however, as previously disclosed, we expect that we will not complete enrollment of the ARMOR Study in our original timeframe. Accordingly, we expect to complete enrollment for the first part of the study in the fourth quarter of 2021 and report top-line results for the first part of the study by the second half of 2023. The rapid development and fluidity of the COVID-19 pandemic precludes any firm estimates as to the ultimate effect this disease will have on our clinical trials, our operations and our business. As a result, any current assessment of the effects of the COVID-19 pandemic, including the impact of this disease on the ARMOR Study as discussed above and our Amilo-5Mer program, is difficult to predict and subject to change and the extent to which the coronavirus impacts our operations will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the duration and severity of the outbreak, the impact of a second and any further wave of COVID-19 and the actions that may be required to contain the coronavirus or treat its impact.
Financial Overview
To date, we have funded our operations primarily through proceeds from private placements and public offerings. At June 30, 2020, we had current assets of $64.2 million, which includes cash and cash equivalents of $5.6 million, short-term deposits of $27.4 million, marketable debt securities of $30.5 million and restricted cash of $0.1 million. This compares with current assets of $76.4 million at December 31, 2019, which includes cash and cash equivalents of $15.9 million, short-term deposits of $27.9 million, marketable debt securities of $31.6 million and restricted cash of $0.1 million. Although we provide no assurance, we believe that such existing funds will be sufficient to continue our business and operations as currently conducted for more than 12 months from the date of issuance of this Form 6-K. However, we will continue to incur operating losses, which may be substantial over the next several years, and we expect that we will need to obtain additional funds to further develop our research and development programs.
Costs and Operating Expenses
Our current costs and operating expenses consist of two components: (i) research and development expenses; and (ii) general and administrative expenses.
Research and Development Expenses
Our research and development expenses consist primarily of outsourced development expenses, salaries and related personnel expenses and fees paid to external service providers, patent-related legal fees, costs of pre-clinical studies and clinical trials and drug and laboratory supplies. We account for all research and development expenses as they are incurred. We expect our research and development expense to remain our primary expense in the near future as we continue to develop Aramchol. Increases or decreases in research and development expenditures are primarily attributable to the number and/or duration of the pre-clinical and clinical studies that we conduct.
We expect that a substantial amount of our research and development expense in the future will be incurred in support of our current and anticipated pre-clinical and clinical development projects. Due to the inherently unpredictable nature of pre-clinical and clinical development studies and unpredictability of the coronavirus outbreak, we are unable to estimate with any certainty the costs we will incur in the continued development of Aramchol for NASH and other indications in our pipeline for potential partnering and/or commercialization. Clinical development timelines, the probability of success and development costs can differ materially from expectations. We currently expect to continue testing Aramchol in pre-clinical studies for toxicology, safety and efficacy, and to conduct additional clinical trials for Aramchol.
While we are currently focused on advancing Aramchol's development, our future research and development expenses will depend on the duration of the ARMOR study, the number of enrolled patients, the clinical success of Aramchol, as well as ongoing assessments of the Aramchol’s commercial potential. As we obtain results from clinical trials, we may elect to discontinue or delay clinical trials for our product candidate in certain indications in order to focus our resources on more promising indications for such product candidate. Completion of clinical trials may take several years or more, but the length of time generally varies according to the type, complexity, novelty and intended use of a product candidate.
We expect our research and development expenses to increase in the future from current levels upon resumption of randomization of patients in the ARMOR Study and continue to advance of our clinical product development and, potentially, the in-licensing of additional product candidates.
The lengthy process of completing clinical trials and seeking regulatory approval for Aramchol requires the expenditure of substantial resources. Any failure or delay in completing clinical trials, or in obtaining regulatory approvals, could cause a delay in generating product revenue and cause our research and development expenses to increase and, in turn, have a material adverse effect on our operations. Because of the factors set forth above, we are not able to estimate with any certainty when we would recognize any net cash inflows from our projects.
General and Administrative Expenses
General and administrative expenses consist primarily of compensation for employees in executive and operational roles, including finance/accounting, legal and other operating positions in connection with our activities. Our other significant general and administrative expenses include non-cash stock-based compensation costs and facilities costs (including the rental expense for our offices in Tel Aviv, Israel), professional fees for outside accounting and legal services, travel costs, investors relations, insurance premiums and depreciation. At this time, we do not anticipate that the effects of the COVID-19 pandemic will materially affect our general and administrative expense.
Financial Income, Net
Our financial income consists mainly of interest income from marketable debt securities and short-term deposits, as well as gains from realization of marketable debt securities and foreign currency gains. Our financial expense consists of fees associated with banking activities and losses from realization of marketable debt securities.
Results of Operations
The table below provides our results of operations for the three and six months ended June 30, 2020 as compared to the three and six months ended June 30, 2019.
| Three months ended June 30, | Six months ended June 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
| (In thousands, except per share data) | ||||||||||||||||
| Research and development expenses | 4,971 | 3,494 | 10,521 | 6,763 | ||||||||||||
| General and administrative expenses | 845 | 1,207 | 1,757 | 1,978 | ||||||||||||
| Total operating expenses | 5,816 | 4,701 | 12,278 | 8,741 | ||||||||||||
| Financial income, net | (290 | ) | (532 | ) | (689 | ) | (1,080 | ) | ||||||||
| Net loss | 5,526 | 4,169 | 11,589 | 7,661 | ||||||||||||
| Other comprehensive income: | (725 | ) | (16 | ) | (463 | ) | (52 | ) | ||||||||
| Comprehensive loss | 4,801 | 4,153 | 11,126 | 7,609 | ||||||||||||
| Basic and diluted net loss per share | $ | 0.26 | $ | 0.20 | $ | 0.55 | $ | 0.36 | ||||||||
Research and Development Expenses
Our research and development expenses amounted to approximately $5.0 million and approximately $10.5 million during the three and six months ended June 30, 2020, respectively, representing an increase of approximately $1.5 million, or 43%, and approximately $3.7 million, or 54%, respectively, compared to approximately $3.5 million and approximately $6.8 million for the comparable period in 2019.
The increase during the three months ended June 30, 2020 primarily resulted from an increase in CMC and formulation expenses in the amount of approximately $1.6 million in connection with the manufacturing of Aramchol API to support the ARMOR Study and the development of Aramchol Meglumine.
The increase during the six months ended June 30, 2020 primarily resulted from an increase in clinical study expenses and drug development expenses in an amount of approximately $1.8 million and $1.3 million, respectively. The increase in clinical trial expenses is in connection with our ongoing ARMOR Study while the increase in drug development expenses relates to CMC and formulation expenses in connection with the manufacturing of Aramchol API to support the ARMOR Study and the development of Aramchol Meglumine.
General and Administrative Expenses
Our general and administrative expenses amounted to approximately $0.8 million and approximately $1.8 million during the three and six months ended June 30, 2020, respectively, representing a decrease of approximately $0.4 million, or 33%, and approximately $0.2 million, or 10%, to approximately $1.2 million and approximately $2.0 million for the comparable period in 2019.
The decrease during the three and six months ended June 30, 2020 primarily resulted from a decrease in professional services and as well a decrease in business development and investor relations related expenses, as compared to such expenses for the comparable period in 2019.
Operating Loss
As a result of the foregoing, for the three and six months ended June 30, 2020, our operating loss was approximately $5.8 million and approximately $12.3 million, respectively, representing an increase of $1.1 million, or 23%, and an increase of $3.6 million, or 41%, respectively, as compared to approximately $4.7 million and approximately $8.7 million for the comparable period in 2019.
Financial Income, Net
Our financial income amounted to approximately $0.3 million and approximately $0.7 million during the three and six months ended June 30, 2020, respectively, representing a decrease of approximately $0.2 million, or 40%, and approximately $0.4 million, or 36%, respectively, compared to $0.5 million and $1.1 million for the comparable period in 2019.
The decrease during the three and six months ended June 30, 2020 primarily resulted from a decrease in interest income from marketable debt securities and short-term deposits, as compared to such income for the comparable period in 2019.
During the three and six months ended June 30, 2020, we recorded unrealized gains of approximately $0.7 million, and approximately $0.5 million, respectively, as a result of change in the market value of its marketable debt securities, as compared to approximately $0.02 million, and approximately 0.05 million, respectively for the comparable period in 2019.
Net Loss
As a result of the foregoing, for the three and six months ended June 30, 2020, our net loss was approximately $5.5 million and approximately $11.6 million, respectively, representing an increase of $1.3 million, or 31%, and an increase of $3.9 million, or 51%, respectively, as compared to approximately $4.2 million and approximately $7.7 million for the comparable period in 2019.
Liquidity and Capital Resources
To date, we have funded our operations primarily through proceeds from private placements and public offerings and we have incurred substantial losses since our inception. As of June 30, 2020, we had an accumulated deficit of approximately $118.5 million and positive working capital (current assets less current liabilities) of approximately $59.5 million. We expect that operating losses will continue for the foreseeable future.
As of June 30, 2020, we had cash and cash equivalents of approximately $5.6 million, restricted cash of approximately $0.1 million, short-term deposits of approximately 27.4 million, and marketable debt securities of approximately $30.5 million invested in accordance with our investment policy, totaling approximately $63.5 million, as compared to approximately $15.9 million, $0.1 million, $27.9 million and $31.6 million as of December 31, 2019, respectively, totaling approximately $75.6 million. The decrease is mainly attributable to our $12.8 million negative cash flow from operating expenses during the six months ended June 30, 2020.
We had negative cash flow from operating activities of approximately $12.8 million for the six months ended June 30, 2020, as compared to negative cash flow from operating activities of approximately $7.0 million for the six months ended June 30, 2019. The negative cash flow from operating activities for the six months ended June 30, 2020 is mainly attributable to our net loss of approximately $11.6 million.
We had positive cash flow from investing activities of approximately $2.4 million for the six months ended June 30, 2020, as compared to a positive cash flow from investing activities of approximately $8.5 million for the six months ended June 30, 2019. The positive cash flow from investing activities for the six months ended June 30, 2020 was primarily due to the net sale of marketable debt securities.
We had positive cash flow from financing activities of approximately $0.1 million for the six months ended June 30, 2020, as compared to a positive cash flow from financing activities of approximately $0.1 million for the six months ended June 30, 2019. The positive cash flow from financing activities for the six months ended June 30, 2020 was due to proceeds from exercise of options.
On May 15, 2020, we amended and restated the Sales Agreement dated December 22, 2017 between us and Stifel, Nicolaus & Company, Incorporated to include Cantor Fitzgerald & Co. as an additional sales agent for our “at the market offering” program, or the A&R Sales Agreement. Pursuant to a prospectus supplement filed with the SEC on May 15, 2020, we may offer and sell $31.9 million of our ordinary shares. During July 2020, we sold 136,300 ordinary shares under the A&R Sales Agreement for total net proceeds of approximately $0.8 million. As a result, we had approximately $31.1 million remaining available for future sales under the A&R Sales Agreement.
Although we provide no assurance, we believe that our existing funds will be sufficient to continue our business and operations as currently conducted for more than 12 months from the date of issuance of this Form 6-K.
The timing and degree of our future capital requirements will depend on many other factors, including:
| · | the progress and costs of our pre-clinical studies, clinical trials and other research and development activities; |
| · | the impact of coronavirus on our operations; |
| · | the scope, prioritization and number of our clinical trials and other research and development programs; |
| · | the amount of revenues and contributions we receive under future licensing, development and commercialization arrangements with respect to Aramchol or any other product candidtate; |
| · | the costs of the development and expansion of our operational infrastructure; |
| · | the costs and timing of obtaining regulatory approval for Aramchol or any other product candidate; |
| · | the ability of us, or our collaborators, to achieve development milestones, marketing approval and other events or developments under our potential future licensing agreements; |
| · | the costs of filing, prosecuting, enforcing and defending patent claims and other intellectual property rights; |
| · | the costs and timing of securing manufacturing arrangements for clinical or commercial production; |
| · | the costs of contracting with third parties to provide sales and marketing capabilities for us; |
| · | the costs of acquiring or undertaking development and commercialization efforts for any product candidate or any future products, product candidates or platforms; |
| · | the magnitude of our general and administrative expenses; |
| · | any cost that we may incur under future in- and out-licensing arrangements relating to Aramchol or any other product candidate; and |
| · | market conditions. |
Until we can generate significant recurring revenues, we expect to satisfy our future cash needs through the net proceeds from private or public debt or equity financings or by out-licensing applications of Aramchol. We cannot be certain that additional funding will be available to us on acceptable terms, if at all. Specifically, the COVID-19 pandemic has significantly disrupted global financial markets, and may limit our ability to access capital, which could in the future negatively affect our liquidity. If funds are not available, we may be required to delay, reduce the scope of or eliminate research or development plans for, or commercialization efforts with respect to, one or more applications of Aramchol or any other product candidate. This may raise substantial doubts about the Company’s ability to continue as a going concern. Furthermore, even if we believe we have sufficient funds for our current or future operating plans, we may seek additional capital due to favorable market conditions or strategic considerations.
Trend Information
We are a development stage company, and it is not possible for us to predict with any degree of accuracy the outcome of our research, development or commercialization efforts. As such, it is not possible for us to predict with any degree of accuracy any significant trends, uncertainties, demands, commitments or events that are reasonably likely to have a material effect on our net loss, liquidity or capital resources, or that would cause financial information to not necessarily be indicative of future operating results or financial condition. However, to the extent possible, certain trends, uncertainties, demands, commitments and events are in this “Management’s Discussion and Analysis of Financial Condition and Results of Operations”.
Controls and Procedures
As a “foreign private issuer”, we are only required to conduct the evaluations required by Rules 13a-15(b) and 13a-15(d) of the Exchange Act as of the end of each fiscal year and therefore have elected not to provide disclosure regarding such evaluations at this time.
Risks Factors
Our business is subject to various risks, including those described in Item 3D of our Annual Report on Form 20-F for the year ended December 31, 2019. There have been no material changes from the risk factors disclosed in Item 3D of our Annual Report on Form 20-F and our Report on Form 6-K filed with the SEC on May 14, 2020, except for the additional risk factors set forth below.
Our business is subject to risks arising from epidemic diseases, such as the recent COVID-19 pandemic, which has impacted and could continue to impact our business.
In late 2019, a novel strain of COVID-19, also known as coronavirus, was reported in Wuhan, China. Initially the outbreak was largely concentrated in China, but it rapidly spread to countries across the globe, including in Israel and the United States. Many countries around the world, including in Israel and the United States, implemented significant governmental measures to control the spread of the virus, including temporary closure of businesses, severe restrictions on travel and the movement of people, and other material limitations on the conduct of business. In response, we have implemented remote working and workplace protocols for our employees in accordance Israeli Ministry of Health requirements to ensure employee safety. Many of our trial sites in our ARMOR Study are based in areas currently affected by coronavirus and there is a general unease of conducting scheduled or elective procedures in medical centers. Given the significant strains on the healthcare system across the globe, we temporarily halted the screening of new patients, for the ARMOR Study and temporarily suspend the opening of new trial sites. We continue to monitor the local situation closely in the U.S. and other countries around the world and have begun to resume activity on a country by country, state by state and site by site basis. Accordingly, we have lifted some of the constraints in the U.S. in states identified as “green states” allowing individual investigators to determine whether it is safe to resume screening activities and recruitment and have opened sites in eight additional countries, including Korea, Turkey, Belgium, France, Spain, Canada, Mexico and Chile. We have also been using the time to advance the opening of new sites so that they are ready for activation when screening and randomization will be possible. To help mitigate cost overrun, we previously took several cost reduction measures including minimizing clinical related expenses, making certain adjustments to clinical staff and pay according to the current and predicted level of activity, and reducing directors’ cash fees by 50% for the first half of 2020.
It is still too early to assess the full impact of the coronavirus outbreak on the ARMOR Study, but our current assessment is that by the fourth quarter of 2020 we should be able to resume recruitment in most of our sites however, as previously disclosed, we expect that we will not complete enrollment of the ARMOR Study in our original timeframe. Accordingly, we expect to complete enrollment for the first part of the study in the fourth quarter of 2021 and report top-line results for the first part of the study by the second half of 2023. The rapid development and fluidity of the COVID-19 pandemic precludes any firm estimates as to the ultimate effect this disease will have on our clinical trials, our operations and our business and it is not possible to predict the impact of a second and any further wave of COVID-19. As a result, any current assessment of the effects of the COVID-19 pandemic, including the impact of this disease on the ARMOR Study as discussed above and our Amilo-5Mer program, is difficult to predict and subject to change and we may experience further disruptions that could severely impact our business, clinical trials, and supply chains, including:
| · | delays or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff for the ARMOR Study or any other clinical trial; |
| · | delays or difficulties in enrolling patients for the ARMOR Study or any other clinical trial especially if sites do not reopen to screen and enroll patients; |
| · | diversion of healthcare resources away from the conduct of clinical trials, including the diversion of hospitals and other medical centers serving as our clinical trial sites and hospital and other staff supporting the conduct of our clinical trials; |
| · | interruption of key clinical trial activities, such as clinical trial site monitoring, due to limitations on travel imposed or recommended by federal or state governments, employers and others or interruption of clinical trial subject visits and study procedures, which may impact the integrity of subject data and clinical study endpoints; |
| · | interruption of, or delays in receiving, supplies of Aramchol from our contract manufacturing organizations due to staffing shortages, production slowdowns or stoppages and disruptions in delivery systems; |
| · | delays in clinical sites receiving the supplies and materials needed to conduct the ARMOR Study or any other clinical trial and interruption in global shipping that may affect the transport of clinical trial materials; |
| · | limitations on employee resources that would otherwise be focused on the conduct of the ARMOR Study or any other clinical trial, including because of sickness of employees or their families or the desire of employees to avoid contact with large groups of people; |
| · | interruptions or delays in the operations of the FDA, EMA or other regulatory authorities, including in receiving feedback or approvals from the FDA, EMA or other regulatory authorities with respect to regulatory submissions; |
| · | changes in local regulations as part of a response to COVID-19 which may require us to change the ways in which the ARMOR Study or any other clinical trial is being conducted, which may result in unexpected costs, or to discontinue the clinical trials altogether; |
| · | delays in necessary interactions with local regulators, ethics committees and other important agencies and contractors due to limitations in employee resources or forced furlough of government employees; |
| · | refusal of the FDA, EMA or other regulatory authorities to accept data from clinical trials in affected geographies; and |
| · | impacts from prolonged remote work arrangements, such as increased cybersecurity risks and strains on our business continuity plans. |
In addition, the spread of COVID-19 has had and may continue to severely impact the trading price of shares of our ordinary shares and could impact our ability to raise additional capital on a timely basis or at all. The COVID-19 pandemic continues to rapidly evolve. The extent to which the COVID-19 may impact our operations will depend on future developments, which are highly uncertain and cannot be predicted with confidence, such as the geographic spread of the disease, the duration of the pandemic, travel restrictions, quarantines, shelter-in-place orders and social distancing, business closures or business disruptions and the effectiveness of actions taken to contain and treat the disease. The impact of the coronavirus outbreak may also have the effect of heightening many of the other risks described in this Form 6-K, in the “Risk Factors” section of our Annual Report on Form 20-F for the year ended December 31, 2019 and our Form 6-K filed with the SEC on May 14, 2020.
Our Amilo-5Mer program is being conducted under a research and option agreement with Yissum, the tech transfer company of the Hebrew University. If we decide to further develop Amilo-5Mer beyond our planned first-in-human Phase I study, we plan to exercise our option to negotiate and enter into a definitive license agreement with Yissum. If we are unable to enter into a definitive license agreement, we would not have the ability to continue the development and potential commercialization of Amilo-5Mer.
We are party to a research and option agreement with Yissum with respect to our Amilo-5Mer program. Under this agreement, we are able to research and initially develop Amilo-5Mer, are required to fund the initial research and have been granted an exclusive option to negotiate and enter into a definitive license agreement with Yissum for Amilo-5Mer upon certain pre-agreed upon terms and such other terms to be agreed upon. If we elect to continue development of Amilo-5Mer beyond the currently contemplated first-in-human Phase I study, we plan to exercise our option to negotiate and enter into a definitive license agreement. If we exercise our option to enter into any definitive license agreement with Yissum, there can be no assurance that we will agree upon terms with Yissum or that it will be on terms favorable to us. If we do not enter into a definitive license agreement, then then we will not have the ability to continue the development and potential commercialization of Amilo-5Mer.
Even if we enter into a definitive license agreement, we will be subject to various additional obligations, including obligations with respect to funding, development and commercialization activities, and payment obligations upon entering into the definitive license agreement and achievement of certain milestones and royalties on product sales. Furthermore, if the definitive license agreement is terminated or breached, we may:
| ▪ | lose our rights to research, develop or commercialize Amilo-5Mer; | |
| ▪ | not be able to secure patent or trade secret protection for Amilo-5Mer; | |
| ▪ | experience significant delays in the development or commercialization of Amilo-5Mer or may have to cease development entirely; | |
| ▪ | incur liability for damages. |
Additionally, even if not terminated or breached, our intellectual property licenses may be subject to disagreements over contract interpretation which could narrow the scope of our rights to the relevant intellectual property or technology or increase our financial or other obligations. If we experience any of the foregoing, it could have a materially adverse effect on our business.
Furthermore, due to its early stage of development, Amilo-5Mer will require significant additional research, development, manufacturing, preclinical and clinical testing, marketing authorization, and commitment of significant additional resources prior to any commercialization. These activities will require significant cash for which we will need to raise additional capital. In addition, Amilo-5Mer is prone to the risks of failure inherent in pharmaceutical product development, including the possibility that Amilo-5Mer will not be shown to be sufficiently safe and effective for approval by regulatory authorities.
EXHIBIT INDEX
| Exhibit No. | Description | |
| 99.1 | Press Release, dated August 6, 2020 | |
99.2 |
Press Release, dated August 6, 2020 | |
| 99.3 | Amilo-5Mer Corporate Presentation | |
| 101.INS | XBRL Instance Document | |
| 101.SCH | XBRL Taxonomy Extension Schema Document | |
| 101.CAL | XBRL Taxonomy Extension Calculation Linkbase Document | |
| 101.DEF | XBRL Taxonomy Extension Definition Linkbase Document | |
| 101.LAB | XBRL Taxonomy Extension Label Linkbase Document | |
| 101.PRE | XBRL Taxonomy Extension Presentation Document |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| Galmed Pharmaceuticals Ltd. | ||
| Date: August 6, 2020 | By: | /s/ Allen Baharaff |
| Allen Baharaff | ||
| President and Chief Executive Officer | ||
Exhibit 99.1
Galmed Pharmaceuticals Provides Business Update and Reports Second Quarter 2020 Financial Results
- Conference Call and Webcast Today at 8:30 a.m. ET / 5:30 a.m. PT -
TEL AVIV, Israel, August 6, 2020 /PRNewswire/ --Galmed Pharmaceuticals Ltd. (Nasdaq: GLMD) ("Galmed" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of the liver targeted SCD1 modulator Aramchol™, an oral therapy for the treatment of nonalcoholic steatohepatitis, or NASH and fibrosis, provides today updated information on the Company's scientific and clinical development programs and reports financial results for the three and six months ended June 30, 2020. The Company will host a conference call and webcast at 08:30 ET today.
Recent Clinical & Scientific Developments
| · | The Data Monitoring Committee (DMC) of the ARMOR study held a scheduled meeting and recommended that the ARMOR Phase 3 trial for NASH and fibrosis can continue with no changes to the protocol. |
| · | Galmed resumed recruitment in some of ARMOR study sites in the USA, Canada, France, Mexico, Chile, Spain, Belgium, Turkey and South Korea (one quarter earlier than previously anticipated). Galmed maintains its guidance for completion of recruitment of patients for the first part of the study by the fourth quarter of 2021 and reporting of top-line results for the first part of the study in the second half of 2023. |
| · | Galmed continues to monitor the impact of the COVID-19 pandemic on its operations and is committed to ensuring the health, safety and well-being of its clinical study participants, staff at its study sites and employees. The rapid development and fluidity of the COVID-19 pandemic precludes any firm estimates as to the ultimate effect this disease will have on Galmed’s clinical trials and is subject to change. |
| · | Galmed intends to approach the regulatory agencies during the first quarter of 2021 to discuss its Aramchol Meglumine program. A preliminary bioequivalence (BE) study is planned for the third quarter of 2021 this year for identifying the equivalent dose to 300mg BID of aramchol acid, currently being used in the ARMOR study. This study is intended to serve as calibration for the regulatory BE study that Galmed plans on performing during the second quarter of 2021 with the identified Aramchol meglumine dose. |
| · | Galmed is announcing phase 1 readiness of its pipeline program, Amilo-5MER. Amilo-5MER is being developed through a research collaboration between Galmed and the Lautenberg Center for General and Tumor Immunology, the Hebrew University — Hadassah Medical School Jerusalem. A Phase 1 study in healthy volunteers is planned for initiation in fourth quarter of 2020 to support its development for IBD and potentially for severe COVID-19 acute respiratory distress syndrome (ARDS). |
Financial Summary – Second Quarter 2020 vs. Second Quarter 2019:
| · | Cash and cash equivalents, restricted cash, short-term deposits and marketable debt securities totaled $63.5 million as of June 30, 2020, compared to $75.6 million at December 31, 2019. |
| · | Net loss amounted to $5.5 million, or $0.26 per share, for the three months ended June 30, 2020, compared to a net loss of $4.2 million, or $0.20 per share, for the three months ended June 30, 2020. |
| · | Research and development expenses amounted to approximately $5.0 million for the three months ended June 30, 2020, compared to approximately $3.5 million for the three months ended June 30, 2019. The increase resulted primarily from an increase in expenses related to CMC and formulation studies in connection with the manufacturing of Aramchol API to support the ARMOR study and the development of Aramchol Meglumine. |
| · |
General and administrative expenses amounted to approximately $0.8 million for the three months ended June 30, 2020, compared to approximately $1.2 million for the three months ended June 30, 2019. The decrease in general and administrative expenses for the three months ended June 30, 2020 resulted primarily from a decrease in professional services expenses and investor relations related expenses.
| |
| · | Financial income, net amounted to $0.3 million for the three months ended June 30, 2020, compared to financial income, net of $0.5 million for the three months ended June 30, 2019. The decrease primarily relates to a decrease in interest income from financial assets. During the three months ended June 30, 2020, the Company recorded unrealized gains of $0.7 million as a result of change in the market value of its marketable debt securities. |
Conference Call & Webcast:
Thursday August 6, 2020, 8:30 AM Eastern Time.
Toll Free: 1-800-954-0643
Toll/International: 1-212-231-2904
Israel Toll Free: 1-809-457-756
Conference ID: 21966797
Webcast: http://public.viavid.com/index.php?id=140960
Replay Dial-In Numbers
Toll Free: 1-844-512-2921
Toll/International: 1-412-317-6671
Replay Pin Number: 21966797
Replay Start: Thursday August 6, 2020, 11:30 AM ET
Replay Expiry: Thursday August 20, 2020, 11:59 PM ET
About Aramchol and Non-alcoholic Steatohepatitis (NASH)
Aramchol (arachidyl amido cholanoic acid) is a novel fatty acid bile acid conjugate, inducing beneficial modulation of intra-hepatic lipid metabolism. Aramchol's ability to modulate hepatic lipid metabolism was discovered and validated in animal models, demonstrating downregulation of the three key pathologies of NASH: steatosis, inflammation and fibrosis. The effect of Aramchol on fibrosis is mediated by downregulation of steatosis and directly on human collagen producing cells. Aramchol has been granted Fast Track designation status by the FDA for the treatment of NASH.
NASH is an emerging world crisis impacting an estimated 3% to 5% of the U.S. population and an estimated 2% to 4% globally. It is the fastest growing cause of liver cancer and liver transplant in the U.S. due to the rise in obesity. NASH is the progressive form of non-alcoholic fatty liver disease that can lead to cardiovascular disease, cirrhosis and liver-related mortality.
About Galmed Pharmaceuticals Ltd.
Galmed Pharmaceuticals Ltd. is a clinical stage drug development biopharmaceutical company for liver, metabolic and inflammatory diseases. Our lead compound, Aramchol™, a backbone drug candidate for the treatment of NASH and fibrosis is currently in a Phase 3 registrational study. We are also collaborating with the Hebrew University in the development of Amilo-5MER, a 5 amino acid synthetic peptide and plan to initiate a first in human study by the fourth quarter of 2020.
Forward-Looking Statements:
This press release may include forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to Galmed's objectives, plans and strategies, as well as statements, other than historical facts, that address activities, events or developments that Galmed intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as "believes," "hopes," "may," "anticipates," "should," "intends," "plans," "will," "expects," "estimates," "projects," "positioned," "strategy" and similar expressions and are based on assumptions and assessments made in light of management's experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Many factors could cause Galmed's actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including, but not limited to, the following: the timing and cost of Galmed's pivotal Phase 3 ARMOR trial, or the ARMOR Study or any other pre-clinical or clinical trials; completion and receiving favorable results of the ARMOR Study for Aramchol or any other pre-clinical or clinical trial; the impact of the coronavirus outbreak; regulatory action with respect to Aramchol or any other product candidate by the FDA or the EMA; the commercial launch and future sales of Aramchol or any other future products or product candidates; Galmed's ability to comply with all applicable post-market regulatory requirements for Aramchol or any other product candidate in the countries in which it seeks to market the product; Galmed's ability to achieve favorable pricing for Aramchol or any other product candidate; Galmed's expectations regarding the commercial market for NASH patients or any other indication; third-party payor reimbursement for Aramchol or any other product candidate; Galmed's estimates regarding anticipated capital requirements and Galmed's needs for additional financing; market adoption of Aramchol or any other product candidate by physicians and patients; the timing, cost or other aspects of the commercial launch of Aramchol or any other product candidate; the development and approval of the use of Aramchol or any other product candidate for additional indications or in combination therapy; and Galmed's expectations regarding licensing, acquisitions and strategic operations. More detailed information about the risks and uncertainties affecting Galmed is contained under the heading "Risk Factors" included in Galmed's most recent Annual Report on Form 20-F filed with the SEC on March 12, 2020, and in other filings that Galmed has made and may make with the SEC in the future. The forward-looking statements contained in this press release are made as of the date of this press release and reflect Galmed's current views with respect to future events, and Galmed does not undertake and specifically disclaims any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
GALMED PHARMACEUTICALS LTD.
Consolidated Balance Sheets
U.S. Dollars in thousands, except share data and per share data
|
As of June 30, 2020 |
As of December 31, 2019 |
|||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 5,553 | $ | 15,931 | ||||
| Restricted Cash | 113 | 112 | ||||||
| Short-term deposits | 27,406 | 27,938 | ||||||
| Marketable debt securities | 30,470 | 31,622 | ||||||
| Other receivables | 669 | 827 | ||||||
| Total current assets | 64,211 | 76,430 | ||||||
| Right of use assets | 481 | 538 | ||||||
| Property and equipment, net | 157 | 171 | ||||||
| Total non-current assets | 638 | 709 | ||||||
| Total assets | $ | 64,849 | $ | 77,139 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities | ||||||||
| Trade payables | $ | 3,876 | $ | 5,999 | ||||
| Other payables | 838 | 935 | ||||||
| Total current liabilities | 4,714 | 6,934 | ||||||
| Non-current liabilities | ||||||||
| Lease obligation | $ | 251 | $ | 352 | ||||
| Total non-current liabilities | 251 | 352 | ||||||
| Ordinary shares par value NIS 0.01 per share; Authorized 50,000,000; Issued and outstanding: 21,153,166 shares as of June 30, 2020; 21,139,385 shares as of December 31, 2019 |
58 | 58 | ||||||
| Additional paid-in capital | 177,853 | 176,696 | ||||||
| Accumulated other comprehensive gain | 498 | 35 | ||||||
| Accumulated deficit | (118,525 | ) | (106,936 | ) | ||||
| Total stockholders' equity | 59,884 | 69,853 | ||||||
| Total liabilities and stockholders' equity | $ | 64,849 | $ | 77,139 | ||||
GALMED PHARMACEUTICALS LTD.
Consolidated Statements of Operations (Unaudited)
U.S. Dollars in thousands, except share data and per share data
| Three months ended June 30, |
Six months ended June 30, |
|||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Research and development expenses | 4,971 | 3,494 | 10,521 | 6,763 | ||||||||||||
| General and administrative expenses | 845 | 1,207 | 1,757 | 1,978 | ||||||||||||
| Total operating expenses | 5,816 | 4,701 | 12,278 | 8,741 | ||||||||||||
| Financial income, net | 290 | 532 | 689 | 1,080 | ||||||||||||
| Net loss | $ | 5,526 | $ | 4,169 | $ | 11,589 | $ | 7,661 | ||||||||
| Basic and diluted net loss per share | $ | 0.26 | $ | 0.20 | $ | 0.55 | $ | 0.36 | ||||||||
| Weighted-average number of shares outstanding used in computing basic and diluted net loss per share | 21,153,166 | 21,120,085 | 21,152,003 | 21,102,306 | ||||||||||||
GALMED PHARMACEUTICALS LTD.
Consolidated Statements of Cash Flows (Unaudited)
U.S. Dollars in thousands
| Six months ended June 31, |
||||||||
| 2020 | 2019 | |||||||
| Cash flow from operating activities | ||||||||
| Net loss | $ | (11,589 | ) | $ | (7,661 | ) | ||
| Adjustments required to reconcile net loss to net cash used in operating activities | ||||||||
| Depreciation and amortization | 19 | 18 | ||||||
| Stock-based compensation expense | 1,096 | 1,007 | ||||||
| Amortization of premium (discount) on marketable debt securities | 16 | (215 | ) | |||||
| Interest income from short-term deposits | (268 | ) | (71 | ) | ||||
| Gain from realization of marketable debt securities | (10 | ) | (9 | ) | ||||
| Changes in operating assets and liabilities: | ||||||||
| Decrease (increase) in other accounts receivable | 158 | (487 | ) | |||||
| Increase (decrease) in trade payables | (2,123 | ) | 764 | |||||
| Decrease in other accounts payable | (141 | ) | (349 | ) | ||||
| Net cash used in operating activities | (12,842 | ) | (7,003 | ) | ||||
| Cash flow from investing activities | ||||||||
| Purchase of property and equipment | (5 | ) | (4 | ) | ||||
| Investment in available for sale securities | (26,979 | ) | (68,717 | ) | ||||
| Investment in short term deposits | (4,000 | ) | (9,000 | ) | ||||
| Maturity of short term deposits | 4,800 | - | ||||||
| Consideration from sale of available for sale securities | 28,588 | 86,248 | ||||||
| Net cash provided by investing activities | 2,404 | 8,527 | ||||||
| Cash flow from financing activities | ||||||||
| Proceeds from exercise of options | 61 | 95 | ||||||
| Net cash provided in financing activities | 61 | 95 | ||||||
| Increase (decrease) in cash and cash equivalents and restricted cash | (10,377 | ) | 1,619 | |||||
| Cash and cash equivalents and restricted cash at the beginning of the period | 16,043 | 24,159 | ||||||
| Cash and cash equivalents and restricted cash at the end of the period | $ | 5,666 | $ | 25,778 | ||||
| Supplemental disclosure of cash flow information: | ||||||||
| Cash received from interest | $ | 708 | $ | 1,057 | ||||
| Non-cash transactions: | ||||||||
| Recognition of right-of-use asset and lease liability from adoption of ASU 2016-02 | $ | 35 | $ | 679 | ||||
Exhibit 99.2
Galmed Pharmaceuticals Announces Acceleration of its New Clinical Amilo-5MER program
| · | Phase 1a Clinical Trial to Begin in Q4 2020 |
TEL AVIV, Israel, August 6, 2020 /PRNewswire/ --Galmed Pharmaceuticals Ltd. (Nasdaq: GLMD) ("Galmed" or the "Company"), a clinical-stage biopharmaceutical company for liver, metabolic and inflammatory diseases provides today updated information on the Company's pipeline program.
Galmed is happy to announce significant progress in the development of Amilo-5MER, a 5 amino acid synthetic peptide MTADV (Methionine, Threonine, Alanine, Aspartic acid, Valine). The 5 amino acids sequence of Amilo-5MER is homologue to a specific MTADV sequence in the human CD44 variant found in synovial fluid cells from joints of rheumatoid arthritis (RA) patients.
Amilo-5MER is being developed through a research collaboration between Galmed and the Hebrew University of Jerusalem. The molecule originated in the laboratory of Prof. David Naor, from the Lautenberg Center for Immunology and Cancer Research, Faculty of Medicine, The Hebrew University. Prof. Naor and his team were the first to publish this specific sequence in the prestigious scientific communication Journal of Clinical Investigation 1.
Amilo-5MER binds to three pro-inflammatory amyloid proteins, Serum Amyloid A (SAA), Transthyretin and Apolipoprotein B with high affinity. The first two are known to be active only in their aggregated forms. By binding to SAA, Amilo-5MER interferes with SAA aggregation and therefor inhibits the destructive autocrine, self-amplifying cytokine loop that causes additional inflammatory reaction.
We are presenting today mechanistic and pre-clinical data which supports an IND submission and the initiation of first in human studies expected to begin later this year.
SAA constitutes acute phase reactants, whose concentration in serum rise rapidly in response to acute stimuli such as infection and trauma. An elevated concentration of SAA was identified in sera of patients with multiple autoimmune diseases and more recently, an outstanding increase of SAA was also detected in COVID-19 infected patients2-3. SAA in its aggregated form, is a potent and rapid inducer of cytokine secretion (particularly Interleukin 6 (IL-6). IL-6 plays an important role in chronic inflammation and is implicated in the pathogenesis of many autoimmune diseases, such as Multiple Sclerosis (MS), Rheumatoid Arthritis (RA), Inflammatory Bowel Disease (IBD) and acute COVID 19. Interference with SAA polymerization and aggregation is a valid target to prevent chronic inflammatory conditions.
Amilo-5MER has been shown to significantly reduce chronic inflammation in animal models of RA, IBD and MS (research work supported by a grant to Prof. Naor from the National Multiple Sclerosis Society (NMSS) of the USA). Amilo-5MER provides a unique mechanism of action to interfere with this vicious cycle, enabling a specific treatment for chronic inflammatory diseases. Data generated from multiple in-vitro, in-vivo and human ex-vivo models have shown that Amilo-5MER significantly improves clinical symptoms. Histological improvements and reduction of pro-inflammatory cytokine secretion were also observed.
An ex-vivo study to investigate the effect of Amilo-5MER on peripheral blood mononuclear cells (PBMCs) from healthy subjects stimulated by SAA, demonstrated significant reduction of IL-6 secretion. This data suggests that Amilo-5MER may also have a role in the treatment of patients with severe COVID-19 acute respiratory distress syndrome (ARDS), characterized by significantly high levels of SAA and IL-6 secretion which is the main cause for the cytokine storm in these patients. An ex–vivo study on PBMCs of patients infected with Covid-19 is ongoing. An alternative mechanism to reduce IL-6 levels (via humanized monoclonal antibody IL-6 receptor antagonist) is currently being evaluated in a Phase 3 Study sponsored by Hoffman La Roche (COVACTA) in Patients with Severe COVID-19 Pneumonia (ClinicalTrials.gov Identifier: NCT04320615).
Amilo-5MER is considered a New Chemical Entity. As such, it is eligible for NCE patent protection until July 2034. Patents have been granted and maintained in the US (US 1061181937), Europe (EP 3169343) and Australia (AU 2015291151) and have been allowed in Japan (JP 6671363).
“Specifically targeting SAA, the key player in this destructive, autocrine, self-amplifying vicious cycle of inflammation, without suppressing the patient’s ability to mount sufficient immune response, has been an unresolved aim in clinical research. Amilo-5MER provides a unique mechanism of action to interfere with this vicious cycle, enabling a specific treatment of chronic inflammatory diseases and hopefully also acute COVID 19. Amilo-5MER has an excellent safety profile and provides a promising selective immune modulation without affecting the patient’s immune surveillance," said Dr. Liat Hayardeny, Chief Scientific Officer of Galmed.
Allen Baharaff, Galmed’s CEO noted: “Amilo-5MER is an exciting and complimentary addition to Galmed's pipeline which focus on liver, metabolic and immune diseases. The accelerated development of Amilo-5MER from proof of concept to a Phase 1-ready compound demonstrates our core competence in identifying and efficiently advancing scientific innovation molecules from Israeli academia to clinical studies. Galmed’s senior research and development team has years of CMC, toxicology, regulatory and clinical experience in successfully developing peptides for chronic inflammatory diseases from bench to bedside. We are looking forward to the rapid clinical development of Amilo-5MER for the benefit of the many patients in need”.
Recent research and development of Amilo-5MER is being conducted under a research and option agreement between Galmed and Yissum, the tech transfer company of the Hebrew University. Galmed has completed all IND-enabling studies for Amilo-5MER, including API manufacturing, toxicology, and other supporting data. Galmed is planning to submit an IND in Q4 2020 and initiate a first in human Phase 1 study in the UK in Q4 2020. If the Phase 1 study is successful, Galmed plans to exercise its option to receive an exclusive license to the Amilo-5MER technology and enter into a definitive license agreement with Yissum. Initiation of a Phase 1b/2a study for biomarkers (SAA in serum) for IBD is planned in H2 21 as well as potentially for the treatment of COVID-19.
An accompanying powerpoint presentation has been made available under “Events & Presentations” of the investor relations section of Galmed's website at http://galmedpharma.investorroom.com/download/AmiloMERFINAL.pdf
| 1. | Shlomo Nedvetzki et al., A mutation in a CD44 variant of inflammatory cells enhances the mitogenic interaction of FGF with its receptor, J. Clin. Invest. 111:1211–1220 (2003). |
| 2. | Huan Li et al., SAA is a biomarker to distinguish severity and Prognosis of Coronavirus Disease 19 (COVID 19) Journal of Infection. 80, 646-655, 2020 |
| 3. | Xiao-Neng Mo et al., Serum Amyloid A is a predictor for prognosis of COVID-19. Respirology 25, 764-765, 2020 |
About Galmed Pharmaceuticals Ltd.
Galmed Pharmaceuticals Ltd. is a clinical stage drug development biopharmaceutical company for liver, metabolic and inflammatory diseases. Our lead compound, Aramchol™, a backbone drug candidate for the treatment of NASH and fibrosis is currently in a Phase 3 registrational study. We are also collaborating with the Hebrew University in the development of Amilo-5MER, a 5 amino acid synthetic peptide and plan to initiate a first in human study by the fourth quarter of 2020.
Forward-Looking Statements:
This press release may include forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to Galmed's objectives, plans and strategies, as well as statements, other than historical facts, that address activities, events or developments that Galmed intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as "believes," "hopes," "may," "anticipates," "should," "intends," "plans," "will," "expects," "estimates," "projects," "positioned," "strategy" and similar expressions and are based on assumptions and assessments made in light of management's experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Many factors could cause Galmed's actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including, but not limited to, the following: the timing and cost of Galmed's pivotal Phase 3 ARMOR trial, or the ARMOR Study; completion and receiving favorable results of the ARMOR Study for Aramchol or any other pre-clinical or clinical trial; the impact of the coronavirus outbreak; regulatory action with respect to Aramchol by the FDA or the EMA; the commercial launch and future sales of Aramchol or any other future products or product candidates; Galmed's ability to comply with all applicable post-market regulatory requirements for Aramchol in the countries in which it seeks to market the product; Galmed's ability to achieve favorable pricing for Aramchol; Galmed's expectations regarding the commercial market for NASH patients; third-party payor reimbursement for Aramchol; Galmed's estimates regarding anticipated capital requirements and Galmed's needs for additional financing; market adoption of Aramchol by physicians and patients; the timing, cost or other aspects of the commercial launch of Aramchol; the development and approval of the use of Aramchol for additional indications or in combination therapy; and Galmed's expectations regarding licensing, acquisitions and strategic operations. More detailed information about the risks and uncertainties affecting Galmed is contained under the heading "Risk Factors" included in Galmed's most recent Annual Report on Form 20-F filed with the SEC on March 12, 2020, and in other filings that Galmed has made and may make with the SEC in the future. The forward-looking statements contained in this press release are made as of the date of this press release and reflect Galmed's current views with respect to future events, and Galmed does not undertake and specifically disclaims any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
Exhibit 99.3
Amilo - 5 MER, Specific, Targeted Immune - Modulator for the Treatment of Chronic Inflammatory Diseases
2 Forward Looking Statements and Disclaimer Statements This presentation contains forward - looking statements about our expectations, beliefs or intentions regarding, among other thing s, our product development efforts, business, financial condition, results of operations, strategies or prospects. In addition, from time to time, we or our representatives have made or may make forward - loo king statements, orally or in writing. Forward - looking statements can be identified by the use of forward - looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. These forward looking statements m ay be included in, but are not limited to, this presentation, various filings made by us with the SEC, press releases or oral statements made by or with the approval of one of our authorized executive officers. For war d - looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward - looking statements relate to matters that have not yet occurred, these statements are inherently subject to risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward - looking statements. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in fo rward - looking statements, including, but not limited to, the factors summarized below. These factors include, but are not limited to, the following: the timing and cost of Galmed's Phase 3 ARMOR trial for Aramchol™ or any other pre - clinical or clinical trial, completion and receiving favorable results of the ARMOR trial for Aramchol™ or any other pre - clincial or clinical trial; the impact of coronavirus on our operation s, regulatory action with respect to Aramchol™ or any other pre - clinical or clinical trial by the FDA or the EMA; the commercial launch and future sales of Aramchol™ or any future product candidates; Galmed's a bil ity to comply with all applicable post - market regulatory requirements for Aramchol™ or any other product candidate in the countries in which it seeks to market the product; Galmed's ability to achiev e f avorable pricing for Aramchol™ or any other product candidate; Galmed's expectations regarding the commercial market for NASH or any other targeted indication; third - party payor reimbursement for Aram chol™ or any other product candidate; Galmed's estimates regarding anticipated capital requirements and Galmed's needs for additional financing; market adoption of Aramchol™ or any other produ ct candidate by physicians and patients; the timing, cost or other aspects of the commercial launch of Aramchol™ or any other product candidate; the development and approval of the use of Aramchol™ or any ot her product candidtate for additional indications or in combination therapy; and Galmed's expectations regarding licensing, acquisitions and strategic operations. More detailed information about the ris ks and uncertainties affecting Galmed is contained under the heading "Risk Factors" included in Galmed's most recent Annual Report on Form 20 - F filed with the SEC on March 12 , 2020 , and in other filings that Galmed has made and may make with the SEC in the future. These statements are only current predictions and are subject to known and unknown risks, uncertainties and other factors tha t m ay cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward - looking statements. Given these uncertainties, you should not rely upon forward - looking statements as predictions of future events. All forward - looking statements attributable to us or persons acting on our behalf included in, but not limited to, this presentation speak only as of the date hereof and are expressly qualified in their entirety by the foregoing. We undertake no obligations to update or revise forward - looking statements to reflect events or circu mstances that arise after the date made or to reflect the occurrence of unanticipated events. In evaluating forward - looking statements, you should consider these risks and uncertainties. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale o f t hese securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. Important Disclaimer Research of Amilo - 5 Mer is currently being conducted under a research and option agreement with Yissum, the tech transfer company of the Hebrew U niv ersity. Under the agreement, Galmed has been granted an exclusive option to negotiate and enter into a definitive license agreement with Yissum for Amilo - 5 Mer upon certain pre - agreed upon terms and such other terms to be agreed upon. Galmed plans to exercise its option if the planned Phase 1 a first - in - human study is successful, however there can be no assurance that Galmed will enter into a definitive license agreeme nt or that it will be on terms favorable to Galmed. If Galmed does not enter into a definitive license agreement, then Galmed will not have the ability to continue the d eve lopment and potential commercialization of Amilo - 5 Mer.
3 Amilo - 5 MER Highlights Amilo - 5 MER is a Differentiated, Specific and Selective Immune - Modulator • Amilo - 5 MER is a penta - peptide that prevents Serum Amyloid A (SAA) polymerization and aggregation • Prevention of SAA polymerization and aggregation results in shut down of chronic inflammation SAA is a validated target for the treatment of chronic inflammation • SAA concentration in serum rise rapidly in response to acute stimuli such as infection and trauma • Elevation of SAA is a common bio - marker as well as main cause of inflammation • SAA is effective in enhancing chronic inflammation only in it’s aggregated form Pursuing multiple large indications • Inflammatory Bowel Disease (IBD) • Rheumatoid Arthritis (RA) • Potential for COVID - 19 - Acute Respiratory Distress Syndrome (ARDS) Main anticipated short - term milestones • First - in - human Phase 1 a topline data expected in Q 1 2021 • Phase 1 b/ 2 a Study in IBD patients – Inc. biomarkers (SAA in serum) expected in H 2 21 Established manufacturing process and IP protection beyond 2034
4 How Was Amilo - 5 MER Discovered? Isolation of joint inflammatory cells Identification of the MTADV (Methionine, Threonine, Alanine, Aspartic acid, & Valine) sequence in the human CD 44 variant Searching in the protein data bank reveals two proteins that contains the complete sequence MTADV Production of synthetic MTADV peptide Amilo - 5 MER Identification of pathological proteins targeted by Amilo - 5 MER
5 Amilo - 5 MER is a Pentapeptide Specifically Sequenced to Interfere with SAA Aggregation to Prevent Inflammation SAA - a validated biomarker and target for acute and chronic inflammatory disease • SAA has pro - inflammatory properties only in it’s aggregated forms • Aggregated SAA is a key player in the destructive autocrine, self - amplifying cytokine loop leading to chronic inflammation and t issue destruction • SAA is elevated by over 1000 fold in multiple autoimmune diseases Amilo - 5 MER – potential to be specific and selective immune - modulator 1 • Amilo - 5 MER is a specific amino acid sequence, homologue to the human CD 44 variant which displays an efficient anti - inflammatory effects • Amilo - 5 MER interferes significantly with SAA aggregation, a key player in the vicious cycle of inflammation • Significant reduction in chronic inflammation in animal models of RA, IBD and MS Amilo - 5 MER – Highly Potent Drug Candidate • Prominent improvement of clinical symptoms, histological features and reduction of pro - inflammatory cytokine secretion in animal models • Addresses downregulation of chronic inflammation with complete preservation of immune surveillance • Strong preclinical package: very good PD effects and excellent safety profile Amilo - 5 MER – key upcoming milestones • Phase 1 a - single and multiple dose in healthy volunteers – topline data expected in Q 1 2021 • Phase 1 b/ 2 a Study for IBD Inc. biomarkers (SAA in serum) planned for H 2 2021 1 David Naor, et al., A HUMAN - DERIVED 5 - MER PEPTIDE (MTADV), WHICH RESTRICTIVELY ALLEVIATES THE PRO - INFLAMMATORY ACTIVITY OF SERUM AMYLOID A (SAA), SUBSTANTIALLY AMELI ORATES IBD PATHOLOGY: NEW POTENTIAL DRUG (MTADV) AND THERAPEUTIC TARGET CANDIDATE (SAA) FOR IBD, Inflammatory Bowel Diseases , Volume 26 , Issue Supplement_ 1 , January 2020 , Pages S 3 – S 4 ,
6 SAA – an Inducer and Biomarker of Chronic Inflammation Increased expression IL - 1 / IL - 6 / TNF - a Infection/Inflammation C - terminal Cleavage b sheet Configuration Fibril Deposition Fibroblast Monocyte IL - 8 INF - a IL - 1 b IL - 18 INF - g TNF - a IL - 6 Monocyte - Derived Supernatant T - cell Th - 17 Th 1 SAA SAA or Hexamer Formation Aggregation
7 Amilo - 5 MER Interferes with SAA Aggregation to Inhibit Chronic Inflammation Pro Inflammatory Cytokines – Inflammation & Tissue Destruction Infection / Stimuli Macrophage SAA Aggregates SAA Aggregates Amilo - 5 MER – Interferes with inflammatory vicious cycle by prevention of SAA aggregation
8 Amilo - 5 MER Prevents SAA Aggregation WITHOUT Amilo - 5 MER WITH Amilo - 5 MER * Research work performed in collaboration with Prof. Mary Cowman from New York University Video recording of Nanoparticle tracking analysis of SAA* Number of SAA aggregates above a certain size counted in defined frame as function of time
Amilo - 5 MER, an Opportunity in Acute and Chronic Inflammatory Conditions: IBD, RA and COVID - 19
10 Inflammatory Bowel Disease (IBD) • IBD (ulcerative colitis and Crohn’s disease) is a chronic lifelong disease • IBD results from the interaction between genetic, microbial and environmental factors • The treatment goal in IBD is to induce and maintain remission Colon with Crohn’s Disease Normal Colon
11 Current IBD Therapies are Modestly Successful due to Undesired Side Effects Unmet need in the treatment of patients with mild to moderate IBD • Treatment of IBD aims to control symptoms sustain remission and reduce complications • Low risk patient – Step Up approach - less potent drug with good safety profile • High risk patient - moderate - severe disease - biologic or immunomodulator therapy Top Down approach The choice of therapy in patients with IBD is dependent upon: • The anatomic location of disease/ disease distribution • The severity of disease • Clinician / patient preferences • Treatment goal (induce or maintain remission) • Insurance coverage/cost. Medical therapies that are used for IBD include • Oral 5 - aminosalicylates (e.g., sulfasalazine, mesalamine) – 5 ’ASA • Glucocorticoids (e.g., prednisone, budesonide) • Immunomodulators (e.g., azathioprine, 6 - mercaptopurine, methotrexate) • Biologic therapies (e.g., TNF α blockers) Severity of Disease 5 ’ASA Corticosteroids TNF α blockers Surgery 6 MP Mercaptopurine
12 IBD, Current and Future Therapy Directions • Current IBD market consists of low - cost generics as well as expensive biologics • Anti - TNFs are essentially effective drugs however, several issues limit their long - term use limitation 1 : • Systemic Immune - Suppression with associated side effects such as an increased risk of infections and lymphoma (rare) • Product label – boxed warning due to increased risk of TB and opportunistic infections; • High price. • Other recently approved biologics include Entyvio (integrin α 4 β 7 Ab for CD and UC) and Stelara (IL 12 /IL 23 Ab for CD); Tofacitinib is an oral, small molecule JAK 3 inhibitor that was recently approved for UC, RA and Psoriasis 2 . • Amilo - 5 MER has the potential to be a backbone treatment of IBD 1 Overview of the medical management of mild (low risk) Crohn disease in adults Authors: Miguel Regueiro, MD, AGAF, FACG, FACP Jan a Al Hashash, MD, MSc Section Editor: Paul Rutgeerts, MD, PhD, FRCP Deputy Editor: Kristen M Robson, MD, MBA, FACG This topic last updated: Nov 25 , 2019 ) . 2 Use of biologics and unmet medical need Gordon et al. European Journal of Gastroenterology & Hepatology 2015 , Volume 27 , Number 7 Biologicals Anti - TNF’s Infliximab Adalimumab Certolizumab Golimumab Anti - Integrins Natalizumab Vedolizumab Etrolizumab PF - 00547659 Anti - IL - 12 / 23 Ustekinumab Briakinumab Brazikumab Guselkumab Risankizumab Mirikizumab Small Molecules Sterods Prednisone Solu Medrol Budesonide 5 ’ ASA’s Sulfasalazine Mesalamine Balsalazide IMM’s Azathioprine 6 - MP Methtrexate Jakinibs Tofacitinib Filgotinib Upadacitinib S 1 P 1 Agonists Ozanimod Etrasimod
13 Amilo - 5 MER Significantly Affects Clinical Symptoms in IBD animal model ( TNBS) STUDY Description: • Evaluation of Amilo - 5 MER’s anti-inflammatory properties was demonstrated in the IBD’s Gold standard model of colitis induced by TNBS in C 57 bl 6 mice. • TNBS model is well-characterized, reliable, reproducible and admitted by regulatory authorities in IBD • 80 C 57 bl 6 mice TNBS induced were randomized in 4 groups • Amilo - 5 MER was administered once a day by subcutaneous injection at 3 and 15 mg/kg in a preventive treatment starting 5 days before colitis induction and until euthanasia at day + 2 . Inflammatory effects were evaluated at the macroscopic level using the validated score of Wallace ( 0 no inflammation, 5 = 2 or more ulcerative and inflammatory sites with an extent > 1 cm, 6 = Ulcerative or inflammatory site > 2 cm) and at the histological level (Ameho’s score 4 = Large inflammatory infiltrate with ulceration area through all the colonic wall, > 50 % of the section). • Study performed by: Intestinal Biotech Development, Lille , France STUDY Results: Amilo - 5 MER demonstrated strong, dose-dependent, anti-inflammatory properties at the macroscopic and histological levels. Moreover, A mil o - 5 MER exerts stronger anti-inflammatory effects at the histological level compared to the positive control, the anti-TNF antibody c ons idered as a benchmark in the treatment of colitis.
14 Amilo - 5 MER Significantly Affects Clinical Symptoms in IBD animal model ( TNBS) Amilo - 5 MER ( 15 mg/kg) exerts stronger anti-inflammatory effects at the macroscopic and histological levels compared to anti-TNF antibody considered benchmark in the treatment of colitis. * : p< 0.05 ** : p< 0.001 * : P< 0.01 ** : P< 0.0001
15 Amilo - 5 MER Reduces Pro - Inflammatory Cytokines in Animal Model for IBD ( TNBS) Amilo - 5 MER ( 15 mg/kg) Significantly Decreases IL - 1 b and IL - 6 gene expression Evaluation of the Colonic mRNA levels in TNBS - induced Colitis in Mice * : P< 0.05 * : P< 0.05
16 1 IBD Market Potential 4 M people afflicted 1.4 M in US 700,000 Crohn’s 70 % require surgery 700 K 100 K Global sales forecast ( by 2026 ) Crohn’s Disease Ulcerative colitis $ 13.8 B $ 6.8 B Physicians visits Hospitalizations 1 Global Drug Forecast and Market Analysis to 2026
17 CIA Sick C 57 BL/ 6 Mouse 9 Days after Amilo - 5 MER treatment Immunization with collagen ( 200 μ g/mouse) and CFA Boost: Collagen ( 200 μ g/mouse) and CFA 3 weeks Onset of disease 1 - 3 days 1 2 3 4 5 6 7 8 9 10 Peptide Injections: Days after disease onset Protocol: Assay under blind manner Collagen - induced arthritis (CIA) is the animal model of Rheumatoid Arthritis. CIA Sick C 57 BL/ 6 mice were treated daily ( 8 injections) for 9 days by Amilo - 5 MER ( 3.5 mg/Kg 70 μ g/ml). Results demonstrate shrinking of the footpad swelling (measured by Micro - caliper Electronic Archimedes device) indicating that the joint inflammation was suppressed. Amilo - 5 MER Significantly Affects Clinical Symptoms in RA ( collagen induced arthritis animal model)
18 Amilo - 5 MER Restores the Normal Anatomy of the Inflamed Joint Following Amilo - 5 MER treatment Control A representative stained histopathological joint section from a mouse with CIA following 9 days treatment with Amilo - 5 MER). Pathology (blinded) assessment indicate a joint of a normal mouse with no damage to the bone and cartilage. Joint inflammation score was < 1.5 on a scale range 0 to 4 . A representative stained histopathological joint section from a mouse with CIA with no treatment showing severe inflammation in the joint with sever damage to bone and cartilage. Joint inflammation score was 4 on a scale range 0 to 4 . P< 0.0001 0 0.5 1 1.5 2 2.5 3 3.5 4 4.5 control RA-peptide Average Inflammation Score
19 Amilo - 5 MER Reduces Pro - Inflammatory Cytokines ex - vivo in Human PBMCs Amilo - 5 MER inhibits SAA ability to stimulate secretion of pro - inflammatory cytokines from white blood cells (designated peripheral blood mononuclear cells, PBMCs) *** : P< 0.001 **** : P< 0.0001
20 Preclinical Safety Studies in Support of First - in - Human Study Completed Maximum Tolerated Dose (MTD) studies in mice and dogs • 7 day MTD in mice at doses up to 1000 mg/kg, in dogs up to 200 mg/kg: No adverse effects were noted • 28 - days in mice at doses up to 175 mg/kg, an in dogs at doses up to 24 mg/kg: No adverse effects were noted Cardiovascular Safety • No inhibition of the potassium hERG channel were noted in an in - vitro system • No adverse effects on the cardiovascular system (ECG, blood pressure and heart rate) in dogs Respiratory Safety • No adverse effects on respiratory rate, tidal volume, and minute volume were noted in mice CNS Safety • No adverse effects on behavior, body posture, body temperature, motor functions and response to stimuli were noted in the Functional Operational Battery of tests in mice Genotoxicity • No potential for genotoxicity was seen in the reverse mutation assay in bacteria and no chromosomal damage was seen in the chromosomal aberration assay in human peripheral blood mononuclear cells Secondary Pharmacology - Off Target Binding • Amilo - 5 MER at 10 μ M showed no significant interaction with a large list of known pharmacological molecular targets
21 Amilo - 5 MER: Phase 1 b/ 2 a IBD Study Design • Planned Study Design: Randomized, double blind, placebo - controlled • Population: 88 subjects aged 18 - 64 with Inflammatory Bowel Disease (IBD) • Dosing: QD (once daily) doses; 4 cohorts; placebo controlled • Treatment duration: 12 weeks • Primary end point: Powered to show statistical difference in mucosal healing. Colonoscopy & SAA as bio marker • Planned trial initiation: Q 2 2021
22 Significant Near Term Anticipated Milestones 2020 2021 Q 3 Q 4 1 H 2 H Initiation of Phase 1 a in the UK Topline data from Phase 1 a Initiation of Phase 1 b/ 2 a IND submission Topline data from Phase 1 b
23 Summary • Chronic and inflammatory diseases are characterized by significant elevation of SAA which is a bio marker and inducer of this process • SAA induces inflammation only in its aggregated form • SAA aggregation and polymerization is a specific target to reduce inflammation • Prevention of SAA aggregation and polymerization interferes with the pro inflammatory chronic inflammation and reduces tissue damage • Amilo - 5 MER is a pentapeptide sequenced specifically to interfere with SAA aggregation to prevent inflammation and tissue damage • Amilo - 5 MER has an excellent safety profile with potential for use for long term / chronic conditions • Phase 1 a topline data expected Q 1 2021 . Phase 1 b/ 2 a Study in IBD patients – Inc. biomarkers (SAA in serum) expected 2 H 21
24 Targeting Pathological SAA aggregation to prevent: chronic inflammation, lymphocyte infiltration, pro - inflammatory cytokine secretion and tissue destruction Immunological Imbalance of the Intestinal Mucosa Auto Immune and Chronic Inflammatory Diseases Amyloid Plaques activation of the osteoclasts, limb paralysis Hyperproliferation of keratinocytes Crohn, Ulcerative colitis IBD Rheumatoid Arthritis RA Psoriasis PS Multiple sclerosis MS Oligodendrocyte Apoptosis Demyelination ARDS COVID 19