ProQR Therapeutics N.V 6-K, filed Aug 8, 2024
ProQR Therapeutics N.V. (PRQR)
About this 6-K
ProQR Therapeutics N.V filed this 6-K on August 8, 2024.
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 of
the Securities Exchange Act of 1934
August 8, 2024
Commission File Number: 001-36622
PROQR THERAPEUTICS N.V.
Zernikedreef 9
2333 CK Leiden
The Netherlands
Tel: +31 88 166 7000
(Address, Including ZIP Code, and Telephone Number,
Including Area Code, of Registrant’s Principal Executive Offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F ⌧ 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): ◻
Furnished as Exhibit 99.1 to this Report on Form 6-K are the unaudited financial statements of ProQR Therapeutics N.V. (the “Company”) for the three and six-month periods ended June 30, 2024, and furnished as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated August 8, 2024, announcing the Company’s results for the three and six-month periods ended June 30, 2024.
On August 8, 2024, the Company issued a press release titled, “ProQR Announces Second Quarter 2024 Operating and Financial Results,” announcing the Company’s results for the three and six-month periods ended June 30, 2024 and providing a business update. A copy of this press release is attached hereto as Exhibit 99.2 and is incorporated herein by reference.
INDEX TO EXHIBITS
Number |
| Description |
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99.1 | | |
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99.2 | |
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.
| PROQR THERAPEUTICS N.V. | |
| | |
| | |
Date: August 8, 2024 | By: | /s/ Jurriaan Dekkers |
| | Jurriaan Dekkers |
| | Chief Financial Officer |
PROQR THERAPEUTICS N.V.
Index to Unaudited Condensed Consolidated Financial Statements
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| PAGE |
1 | |
2 | |
3 | |
4 | |
Notes to Unaudited Condensed Consolidated Financial Statements | 5 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
| | June 30, | | December 31, |
| | 2024 | | 2023 |
| | €1,000 | | €1,000 |
Assets | |
| |
|
Current assets | |
| |
|
Cash and cash equivalents | 5 | | ||
Financial asset - current | 6 | | — | |
Prepayments and other receivables | 7 | | ||
Other taxes | | | ||
| | | | |
Total current assets | | | ||
| | | | |
Property, plant and equipment | 8 | | ||
Investments in financial assets | 18 | — | | — |
| | | | |
Total assets | | | ||
| | | | |
Equity and liabilities | |
| |
|
Equity | | | |
|
Equity attributable to owners of the Company | | | ||
Total equity | 13 | | ||
| | | | |
Current liabilities | |
| |
|
Borrowings | 10 | | — | |
Lease liabilities | 11 | | ||
Derivative financial instruments | 10 | | ||
Trade payables | | | ||
Social securities and other taxes | | | ||
Deferred income | 12 | | ||
Other current liabilities | 9 | | ||
| | | | |
Total current liabilities | | | ||
| | | | |
Borrowings | 10 | — | | |
Lease liabilities | 11 | | ||
Deferred income | 12 | | ||
| | | | |
Total liabilities | | | ||
| | | | |
Total equity and liabilities | | |
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI
(€ in thousands, except share and per share data)
| | Three month period | | Six month period | ||||
| | ended June 30, |
| ended June 30, | ||||
|
| 2024 | | 2023 |
| 2024 | | 2023 |
| | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Revenue | 14 | | | | ||||
| | | | | | | | |
Other income | 15 | | | | ||||
| | | | | | | | |
Research and development costs | 16 | ( | | ( | | ( | | ( |
General and administrative costs | 17 | ( | | ( | | ( | | ( |
Total operating costs | | ( | | ( | | ( | | ( |
| |
| |
| |
| |
|
Operating result | | ( | | ( | | ( | | ( |
Finance income and expense | | | | | ( | |||
Results related to associates and financial assets | 18 | — | | — | | — | | — |
Results related to financial liabilities measured at fair value through profit or loss | 10 | | | | ||||
Results on derecognition of financial liabilities | 19 | — | | | — | | ||
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Result before corporate income taxes | | ( | | ( | | ( | | ( |
Income taxes | 20 | | | | ||||
| |
| |
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Result for the period | | ( | | ( | | ( | | ( |
Other comprehensive income (foreign exchange differences on foreign operation) | | | | | ( | |||
| |
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Total comprehensive income | | ( | | ( | | ( | | ( |
| | | | | | | | |
Result attributable to | |
| |
| |
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|
Owners of the Company | | ( | | ( | | ( | | ( |
Non-controlling interests | | — | | | — | | ||
| | ( | | ( | | ( | | ( |
Total comprehensive income attributable to | | | | | | | | |
Owners of the Company | | ( | | ( | | ( | | ( |
Non-controlling interests | | — | | | — | | ||
| | ( | | ( | | ( | | ( |
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Share information | |
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Weighted average number of shares outstanding1 | | | | | ||||
| | | | | | | | |
Earnings per share attributable to owners of the Company (Euro per share) | | | | | | | | |
Basic loss per share1 | | ( | | ( | | ( | | ( |
Diluted loss per share1 | | ( | | ( | | ( | | ( |
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
| 1. |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
| Attributable to owners of the Company | | | | | |||||||||||||
|
| Number |
| Share |
| Share |
| Equity settled |
| Translation |
| Accumulated |
| Total |
| Non- |
| Total |
|
|
| | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Balance at January 1, 2023 |
| | | | | | ( | | | ( | | |||||||
Result for the period |
| — | | — | | — | | — | | — | | ( | | ( | | | ( | |
Other comprehensive income |
| — | | — | | — | | — | | ( | | — | | ( | | — | | ( |
Recognition of share-based payments |
| — | | — | | — | | | — | | — | | | — | | |||
Treasury shares transferred | | ( | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | ( | | — | | | — | | — | | — | |
Share options exercised | | | — | | | ( | | — | | | | — | | |||||
| | | | | | | | | | | | | | | | | | |
Balance at June 30, 2023 |
| | | | | | ( | | | ( | | |||||||
| | | | | | | | | | | | | | | | | | |
Balance at January 1, 2024 |
| | | | | | ( | | | — | | |||||||
Result for the period |
| — | | — | | — | | — | | — | | ( | | ( | | — | | ( |
Other comprehensive income |
| — | | — | | — | | — | | | | — | | | — | | ||
Recognition of share-based payments |
| — | | — | | — | | | — | | — | | | — | | |||
Treasury shares transferred | | ( | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | ( | | — | | | — | | — | | — | |
Share options exercised / RSUs vested | | | — | | | ( | | — | | | | — | | |||||
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Balance at June 30, 2024 |
| | | | | | ( | | | — | | |||||||
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
| | | | | | | | |
| | Three month period | | Six month period | ||||
| | ended June 30, |
| ended June 30, | ||||
|
| 2024 | | 2023 | | 2024 | | 2023 |
| | €1.000 | | €1.000 | | €1.000 | | €1.000 |
Cash flows from operating activities | |
| |
| |
| |
|
Net result | | ( | | ( | | ( | | ( |
Adjustments for: | | | | | | | | |
— Depreciation | | | | | ||||
— Share-based compensation | 13 | | | | ||||
— Financial income and expenses | | ( | | ( | | ( | | |
— Results related to associates and financial assets | 18 | — | | — | | — | | — |
— Results related to financial liabilities measured at fair value through profit or loss | 10 | ( | | ( | | ( | | ( |
— Results on derecognition of financial liabilities | 19 | — | | ( | | — | | ( |
— Income tax expenses | 20 | ( | | — | | ( | | — |
| | | | | | | | |
Changes in working capital | | ( | | ( | | ( | | |
Cash (used in)/generated from operations | | ( | | ( | | ( | | |
| |
| |
| |
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|
Corporate income tax received/(paid) | | | — | | | — | ||
Interest received | | | | | ||||
Interest paid | | ( | | — | | ( | | — |
| |
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|
Net cash (used in)/generated from operating activities | | ( | | ( | | ( | | |
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Cash flow from investing activities | | | | | | | | |
Increase in financial asset - current | 6 | — | | — | | ( | | — |
Purchases of property, plant and equipment | | ( | | ( | | ( | | ( |
Sales of property, plant and equipment | | — | | — | | — | | |
| |
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Net cash used in investing activities | | ( | | ( | | ( | | ( |
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Cash flow from financing activities | |
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Proceeds from exercise of share options | 13 | | | | ||||
Repayment of lease liability | 11 | ( | | ( | | ( | | ( |
| |
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|
Net cash used in financing activities | | ( | | ( | | ( | | ( |
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Net (decrease)/increase in cash and cash equivalents | | ( | | ( | | ( | | |
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Currency effect cash and cash equivalents | | | | | ||||
Cash and cash equivalents, at beginning of the period | | | | | ||||
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Cash and cash equivalents at the end of the period | | | | | ||||
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
PROQR THERAPEUTICS N.V.
Notes to Unaudited Condensed Consolidated Financial Statements
1. General Information
ProQR Therapeutics N.V., or “ProQR” or the “Company”, is a biotechnology company domiciled in the Netherlands that primarily focuses on the discovery and development of novel therapeutic medicines.
Since September 18, 2014, the Company’s ordinary shares have been listed on Nasdaq. They are currently trading at Nasdaq Capital Market under ticker symbol PRQR.
The Company was incorporated in the Netherlands, on February 21, 2012 (Chamber of Commerce no. 54600790) and was reorganized from a private company with limited liability to a public company with limited liability on September 23, 2014. The Company has its statutory seat in Leiden, the Netherlands. The address of its headquarters and registered office is Zernikedreef 9, 2333 CK Leiden, the Netherlands.
ProQR Therapeutics N.V. is the ultimate parent company of the following entities:
| ● | ProQR Therapeutics Holding B.V. ( |
| ● | ProQR Therapeutics I B.V. ( |
| ● | ProQR Therapeutics II B.V. ( |
| ● | ProQR Therapeutics III B.V. ( |
| ● | ProQR Therapeutics IV B.V. ( |
| ● | ProQR Therapeutics V B.V. ( |
| ● | ProQR Therapeutics VI B.V. ( |
| ● | ProQR Therapeutics VII B.V. ( |
| ● | ProQR Therapeutics VIII B.V. ( |
| ● | ProQR Therapeutics IX B.V. ( |
| ● | ProQR Therapeutics I Inc. ( |
ProQR Therapeutics N.V. is also statutory director of Stichting Bewaarneming Aandelen ProQR (“ESOP Foundation”) and has full control over this entity.
As used in these condensed consolidated financial statements, unless the context indicates otherwise, all references to “ProQR” or the “Company” refer to ProQR Therapeutics N.V. including its subsidiaries and the ESOP Foundation.
2. Significant Accounting Policies
These interim condensed consolidated financial statements for the three and six month periods ended June 30, 2024 have been prepared in accordance with IAS 34 Interim Financial Statements. They should be read in conjunction with the Company’s annual financial statements for the year ended December 31, 2023. These interim condensed consolidated financial statements do not include all information required for a complete set of financial statements prepared in accordance with IFRS Standards. However, selected explanatory notes are included to explain events and transactions that are significant to an understanding of the changes in the Company’s financial position and performance since the last annual financial statements. In the opinion of management, all events and transactions that are significant to an understanding of the changes in financial position and performance of the Company since the end of the last annual reporting period are disclosed in these interim condensed consolidated financial statements. The accounting policies adopted in the preparation of the interim condensed consolidated financial statements are consistent with those applied in the preparation of the Company’s annual financial statements for the year ended December 31, 2023.
During the six month period ended June 30, 2024, the Company invested in financial assets in the form of deposits with an original maturity of longer than three months but shorter than twelve months as described in Note 6. Financial assets are measured at amortised cost as they give rise on specified dates to cash flows that are solely payments of principal and interest on the principal amount outstanding.
The Company’s financial results have varied substantially, and are expected to continue to vary, from period to period. The Company believes that its ordinary activities are not linked to any particular seasonal factors.
The management of ProQR has, upon preparing and finalizing these interim condensed consolidated financial statements, assessed the Company’s ability to fund its operations for a period of at least one year after the date of signing these interim condensed consolidated financial statements. Management expects the Company to continue as a going concern based on its existing funding, taking into account the Company’s current cash position and the projected cash flows based on the activities under execution on the basis of ProQR’s business plan and budget. Based on our current operating plan, we believe that the existing cash and cash equivalents will be sufficient to fund our anticipated level of operations at least into mid-2026. Thus, we continue to adopt the going concern basis of accounting in preparing the interim condensed consolidated financial statements.
The carrying amount of all financial assets and financial liabilities is a reasonable approximation of the fair value and therefore, information about the fair values of each class has not been disclosed.
The Company operates in
3. Adoption of New and Revised International Financial Reporting Standards
New Standards and Interpretations, which became effective as of January 1, 2024, did not have a material impact on our condensed consolidated financial statements.
4. Critical Accounting Estimates and Judgments
In the application of the Company’s accounting policies, management is required to make judgments, estimates and assumptions about the carrying amounts of assets and liabilities that are not readily apparent from other sources. The estimates and associated assumptions are based on historical experience and other factors that are considered to be relevant. Actual results may differ from these estimates.
The estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized in the period in which the estimate is revised if the revision affects only that period or in the period of the revision and future periods if the revision affects both current and future periods.
The significant judgements made by management in applying the Company’s accounting policies, except for the significant judgements related to revenue recognition from milestone payments as further described below and in Note 14, and the key sources of estimation uncertainty were the same as those described in the Company’s annual financial statements for the year ended December 31, 2023.
Revenue recognition for the Eli Lilly collaboration and license agreement
a. Identification of the performance obligation
Note 14 describes the Company’s original research and collaboration agreement with Eli Lilly and Company, and the amended and restated research and collaboration agreement (collectively, the “Collaboration agreement”). Under the Collaboration agreement, ProQR provides Eli Lilly with a license (with a right to sub-license) to exploit compounds resulting from the collaboration. A significant amount of judgement is required to determine whether the license is distinct from the other promises in the contract. The license was concluded not to be distinct from the other promises in the contract based on the following considerations:
| ● | the license has no stand-alone value to Eli Lilly without the Company being involved in the research and development collaboration, and; |
| ● | there are significant interdependencies between the license and the research and development services to be provided by the Company. |
ProQR’s services are evaluated as predominant at inception of the contract and contractually only ProQR can perform these services as they are specific to the skills of the Company. The resulting compounds from the collaboration do not represent a series of distinct promises because they were not predetermined at the inception of the contract and can be terminated or replaced at the discretion of Lilly subject to the terms and conditions of the Collaboration agreement.
b. Determining the timing of satisfaction of performance obligations
Under the Collaboration agreement, the Company recognizes revenue over time, using an input method that estimates the satisfaction of the performance obligation as the percentage of labor hours incurred compared to the total estimated labor hours required to complete the promised services. As our estimate of the total labor hours required is dependent on the evolution of the research and development activities, it may be subject to change. If the progression and/or outcome of certain research and development activities would be different from the assumptions that were made during the preparation of these financial statements, this could lead to material adjustments to the total estimated labor hours, which might result in a reallocation of revenue between current and future periods. Our total deferred revenue balance amounts to €
c. Determining the transaction price
The Company applied judgement to determine whether the equity investments made by Eli Lilly in ProQR are part of the transaction price for the Collaboration agreement. The Company concluded that the differences between the prices that Eli Lilly paid for the shares and the ProQR stock closing prices on the days of entering into the equity investment agreements arose because of the Company’s existing obligations to deliver research and development services to Eli Lilly under the terms of the Collaboration agreement. Therefore, the above differences between the closing share prices on the agreement effective dates and the equity investment prices paid by Eli Lilly are considered to be part of the transaction price of the contract and are initially allocated to deferred revenue.
The contract also includes variable consideration, but no variable consideration was included in the initial transaction price at the inception, as it is not highly probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. The Company includes such variable consideration in the transaction price when the uncertainty associated with the variable consideration is resolved.
As further described in Note 14, during 2024, the Company reached milestones under the agreement, which were added to the transaction price and recognized partially as revenue during 2024 with the remainder recorded in future periods based on progress towards satisfaction of performance obligation.
The Collaboration agreement includes sales-based royalties, including commercial milestone payments based on the level of sales. Related revenue is recognized as the subsequent underlying sales occur at a point in time.
Research and development expenditures
Research expenditures are reflected in the income statement. Development expenses are currently also reflected in the income statement because the criteria for capitalization are not met. At each balance sheet date, the Company estimates the level of service performed by the vendors and the associated costs incurred for the services performed.
Although we do not expect the estimates to be materially different from amounts actually incurred, the understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in reporting amounts that are too high or too low in any particular period.
5. Cash and Cash Equivalents
At June 30, 2024, the Company’s cash and cash equivalents were €
6. Financial Asset – Current
This current financial asset relates to a term deposit with an initial maturity longer than 3 months but less than 12 months and does not qualify as a cash equivalent. The deposit is held at a bank with an investment grade credit rating. Short-term credit ratings must be rated A-1/P-1/F1 at a minimum by at least one of the NRSROs specifically Moody’s, Standard & Poor’s or Fitch.
| | June 30, | | December 31, |
| | 2024 | | 2023 |
| | €1,000 | | €1,000 |
Term deposit and interest accrued | | | | — |
| | | — |
7. Prepayments and Other Receivables
| | June 30, | | December 31, |
| | 2024 | | 2023 |
| | €1,000 | | €1,000 |
Prepayments | | | | |
Other Receivables | | | ||
| | |
At June 30, 2024 and December 31, 2023 prepayments consisted principally of payments made by the Company for services not yet provided by vendors. At June 30, 2024 and December 31, 2023 other receivables consisted principally of amounts receivable from research collaboration partners and deposits.
8. Property, Plant and Equipment
At June 30, 2024 and December 31, 2023, property plant and equipment consisted of buildings and leasehold improvements, laboratory equipment and other assets. Buildings and leasehold improvements include a right-of-use asset relating to the lease of our Leiden office and laboratory space, with a carrying amount of €
9. Other Current Liabilities
At June 30, 2024, other current liabilities amount to €
10. Borrowings
| | June 30, | | December 31, |
|
| 2024 | | 2023 |
| | €1.000 | | €1.000 |
Innovation credit | | | ||
Accrued interest on innovation credit | | | ||
| | | | |
Total borrowings | | | ||
Current portion | | ( | | — |
| | — | |
On December 10, 2018 ProQR was awarded an Innovation credit for the sepofarsen program. Amounts were drawn under this facility from 2018 through 2022. The credit of €
In December 2023, ProQR received a conditional waiver of the balance of the Innovation credit including accrued interest. Consequently, the repayment of the total loan of €
In September 2022, ProQR extinguished its debt with Pontifax and Kreos by repaying all outstanding principal amounts. Pontifax’ and Kreos’ warrants remain in place until their
11. Lease Liabilities
At June 30, 2024 and December 31, 2023, lease liabilities primarily consisted of the Company’s lease of office and laboratory facilities at Zernikedreef in Leiden, the Netherlands.
The Company leases office and laboratory facilities of
The initial lease agreement for the Leiden office and laboratory facilities was accounted for as of commencement date July 1, 2020. This period was extended by
The carrying amount of the right-of-use asset is disclosed in Note 8.
12. Deferred Income
The following table summarizes details of deferred income at June 30, 2024 and December 31, 2023. The nature of the deferred income is described in Note 14 and 15.
| | June 30, | | December 31, |
| | 2024 | | 2023 |
| | €1,000 | | €1,000 |
Payments from Eli Lilly | | | ||
Payments from Rett Syndrome Research Trust | | | — | |
Current deferred income | | | ||
| | | | |
Payments from Eli Lilly | | | ||
Non-current deferred income | | | ||
| | | | |
Total deferred income | | |
13. Shareholders’ Equity
The authorized share capital of the Company amounting to €
Translation reserve
The translation reserve comprises all foreign currency differences arising from the translation of the financial statements of foreign operations.
Share options
The Company operates an equity-settled share-based compensation plan, which was introduced in 2013. Options and Restricted Stock Units (“RSUs”) may be granted to employees, members of the Supervisory Board, members of the Management Board and consultants. The compensation expenses included in operating costs for this plan in the six month period ended June 30, 2024 were €
14. Revenue
Eli Lilly
In September 2021, the Company entered into a global licensing and research collaboration with Eli Lilly and Company (‘Lilly’) focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. ProQR and Lilly will use ProQR’s proprietary Axiomer® RNA editing platform to progress new drug targets toward clinical development and commercialization.
Under the terms of the agreement, ProQR received an upfront payment and equity consideration, and is eligible to receive milestone payments and royalties on the net sales of any resulting products. In September 2021, the Company issued
With regard to its original collaboration with Lilly, the Company concluded as follows:
| ● | There is |
| ● | The transaction price of this agreement includes fixed components, consisting of an up-front fee and an equity component. The agreement also contains variable parts, which are included in the transaction price to the extent that it is highly probably that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Milestone payments will only be included to the extent that it is highly probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the milestones is subsequently resolved. Sales-based milestones and sales-based royalties will be included as the underlying sales occur. |
| ● | The Company recognizes revenue over time, using an input method that estimates the satisfaction of the performance obligation as the percentage of labor hours incurred compared to the total estimated labor hours required to complete the promised services. |
In December 2022, the Company and Lilly amended their research and collaboration agreement described above, which expanded the collaboration. Under the amended and restated research and collaboration agreement, Lilly will gain access to additional targets in the central nervous system and peripheral nervous system with ProQR’s Axiomer platform.
Pursuant to the amended and restated agreement, the Company issued
With regard to the amended and restated research and collaboration agreement with Lilly, the Company concluded as follows:
| ● | There is |
| the Collaboration agreement. |
| ● | The transaction price of this agreement includes fixed components, consisting of an up-front fee and an equity component (discount), and variable components. The variable components are not yet fully included in the transaction price. Milestone payments will only be included to the extent that it is highly probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the milestones is subsequently resolved. As further described in the note, the Company reached certain milestones. Sales-based milestones and sales-based royalties will be included as the underlying sales occur. |
| ● | The Company recognizes revenue over time, using an input method that estimates the satisfaction of the performance obligation as the percentage of labor hours incurred compared to the total estimated labor hours required to complete the promised services. |
During the six month period ended June 30, 2024, the Company reached milestones amounting to $
15. Other Income
| | Six month period | ||
| | ended June 30, | ||
|
| 2024 | | 2023 |
| | €1.000 | | €1.000 |
| | | | |
Grant income | | | ||
Other income | | — | | |
| | | ||
In January, 2024, the Company entered into an agreement with the Rett Syndrome Research Trust (“RSRT”) that focuses on the design and development of editing oligonucleotides (“EONs”) using the Company’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (“MECP2”) and correcting mutations of interest. Under the agreement, RSRT awarded the Company up to €
Grants are recognized in other income in the same period in which the related R&D costs are recognized.
16. Research and Development Costs
Research and development costs amount to €
17. General and Administrative Costs
General and administrative costs amount to €
18. Investments in Financial Assets
Investment in financial assets consist of the Company’s investment in Phoenicis Therapeutics Inc. (“Phoenicis”) and Yarrow Biotechnology Inc (“Yarrow”).
ProQR holds a
ProQR holds a
19. Results Related to Derecognition of Financial Liabilities
| | Six month period | ||
| | ended June 30, | ||
|
| 2024 | | 2023 |
| | €1.000 | | €1.000 |
| | | | |
Gain on waiver of Amylon convertible loans | | — | | |
| | — | | |
Convertible loans
Convertible loans were issued to Amylon Therapeutics B.V. (“Amylon”), an
In 2023 and 2022, Amylon entered into waiver agreements with its lenders. Such lenders’ loan agreements with Amylon are severed and any claims to repayment of any outstanding debt and accumulated interest are renounced. The amount of convertible loans and accumulated interest that was waived under these agreements in the six month period ended June 30, 2024 is €
In the third quarter of 2023, Amylon was legally dissolved.
20. Income Taxes
The current income tax liability amounts to €
Tax losses may be carried forward indefinitely. However, the offset of losses will be limited in a given year against the first € 1 million of taxable profit. For taxable profit in excess of this amount, losses may only be offset up to 50% of this excess.
21. Events After Balance Sheet Date
None.
Exhibit 99.2
ProQR Announces Second Quarter 2024 Operating and Financial Results
| ● | AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024 |
| ● | $4.5 million in milestones achieved in H1 related to progress in the Lilly partnership |
| ● | €96.2 million cash position as of end of Q2 providing runway into mid-2026, plus additional potential milestones from Lilly partnership |
LEIDEN, Netherlands & CAMBRIDGE, Mass., August 8, 2024 – ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the second quarter ended June 30, 2024, and provided a business update.
“In the second quarter, we took important steps toward advancing our first editing oligonucleotide programs to clinical stage, with AX-0810 targeting NTCP for cholestatic diseases and AX-1412 targeting B4GALT1 for cardiovascular diseases on track to enter the clinic in late 2024/early 2025,” said Daniel A. de Boer, Founder and CEO of ProQR. “We were excited to share data at ASGCT in May, demonstrating in vivo proof of target engagement with meaningful changes in disease-relevant biomarkers in non-human primates for our NTCP program. As we progress our CTA/IND-enabling studies, we plan to announce translational data and more about our clinical plans for AX-0810 in the second half of the year, as well as preclinical proof of concept and translational data for AX-1412.”
de Boer continued, “Beyond our own pipeline programs, we are pleased with the progress we are making in our partnership with Lilly and highlight the achievement of multiple milestones in the first half of the year. With the benefits of a productive partnership, a differentiated pipeline strategy rooted in human genetics, our leading IP portfolio, and a strong cash balance with runway into mid-2026, we look forward to continued execution as we advance our science and pipeline programs.”
Recent Progress and Anticipated Upcoming Events
| ● | In June, ProQR presented at the RNA Editing Summit in Boston, highlighting its Axiomer technology platform and AX-0810, the Company’s program targeting NTCP for cholestatic diseases. |
| ● | In May at the American Society of Cell and Gene Therapy (ASGCT) Annual Meeting, ProQR presented preclinical proof of concept in vitro and in vivo data for AX-0810 for Cholestatic |
| Diseases targeting NTCP (poster P-705). ProQR scientists showed for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) with meaningful changes in biomarkers in NHPs using Axiomer RNA Editing Oligonucleotides. At ASGCT, ProQR and partner Eli Lilly presented preclinical data related to Axiomer in poster P-726 titled “Complex Metabolism and Prolonged PK/PD of a GalNAc-Conjugated Editing Oligonucleotide (EON) in Mice”. |
| ● | Anticipated Upcoming Events: |
| ● | AX-0810 targeting NTCP for cholestatic diseases: clinical development candidate translational data to be reported, and design for the clinical trial to be shared in H2 2024 with program to advance to the clinic in late 2024/early 2025. |
| ● | AX-1412 targeting B4GALT1 for cardiovascular diseases: preclinical proof of concept data and translational data to be reported in H2 2024 with program to advance to the clinic in late 2024/early 2025. |
| ● | Potential additional new pipeline target announcements in 2024. |
| ● | Continue to execute on partnership with Eli Lilly and Company (Lilly), with potential additional data updates to come in 2024, along with potential additional milestone income from existing partnership, and potential option to exercise for expansion of deal to 15 targets, which would result in a $50 million opt-in payment to ProQR. |
| ● | ProQR may selectively form new partnerships, which could include multi-target discovery alliances, or product alliances on specific programs. |
Financial Highlights
At June 30, 2024, ProQR held cash and cash equivalents and short term financial assets of €96.2 million, compared to €118.9 million cash and cash equivalents at December 31, 2023. Net cash used in operating activities during the three-month period ended June 30, 2024 was €6.3 million, compared to €10.3 million used for the same period last year. During the first half of 2024, the Company achieved certain milestones in the collaboration agreement with Eli Lilly amounting to $4.5 million (€4.2 million). Milestone income achieved in Q2 was received in July.
Research and development (R&D) costs were €7.1 million for the quarter ended June 30, 2024 compared to €5.9 million for the same period last year.
General and administrative costs were €3.0 million for the quarter ended June 30, 2024 compared to €4.1 million for the same period last year.
Net loss for the three-month period ended June 30, 2024 was €2.7 million, or €0.03 per diluted share, compared to €8.0 million, or €0.10 per diluted share, for the same period last year. For further financial information for the period ended June 30, 2024, please refer to the Q2 financial report filing.
About Axiomer™
ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
About Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC)
Cholestatic disorders refer to a group of diseases presenting excessive and toxic buildup of bile acids in the liver due to bile ducts dysfunction. This leads to liver damage and a range of debilitating symptoms. Without treatment, liver damage can progress through various stages, ultimately leading to liver failure and elevated risk of liver malignancy, affecting life expectancy. Cholestatic diseases remain leading causes of liver transplantation. There are no approved therapies for primary sclerosing cholangitis (PSC) for adults and biliary atresia (BA) for pediatrics It is estimated that 80,000 and 20,000 individuals have PSC and BA, respectively, in North America and in Europe.
About AX-0810 targeting NTCP
The majority of the bile acids present in the liver cells originate from the enterohepatic reuptake cycle. The key transporter responsible for hepatic uptake of bile acids from portal circulation is the sodium (Na+)-taurocholate cotransporting polypeptide (NTCP, SLC10A1 gene) expressed in the liver. AX-0810 is designed to introduce a loss of function variant in SLC10A1 RNA that has been found in human genetics to prevent re-uptake of bile acids in liver via NTCP. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for PSC and BA primarily among other cholestatic diseases.
About Cardiovascular Diseases
Cardiovascular diseases (CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems like heart attacks, heart failure, and stroke. CVDs represent the leading cause of disability and death in the world. Approximately 18 million people die every year from CVDs representing one third of all the global deaths. Despite available lipid lowering therapies and hypertension medications, the risk of CVDs is still projected to increase rapidly over the coming years.
About AX-1412 targeting B4GALT1
Gene–based analysis of rare beta-1,4-galactosyltransferase 1 (B4GALT1) missense variant (p.Asn352Ser) is known to lead to B4GALT1 protein loss of function and showed an association with decreased coronary artery disease. These beneficial effects are mediated by hypo-galactosylation of the apolipoprotein B100 and fibrinogen, known – independent – drivers of increased risk of CVDs. AX-1412 introduces a protective variant into B4GALT1 RNA to address the remaining residual risk of developing cardiovascular diseases. ProQR intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
Learn more about ProQR at www.proqr.com.
Forward Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended and potential benefits thereof, including the receipt of milestone and royalty payments from commercial product sales, if any, from the products covered by the collaboration, our ability to selectively form new partnerships and enter into future collaborations, and our financial position and cash-runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings
made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
ProQR Therapeutics N.V.
Investor contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Peter Kelleher
LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com
Media contact:
Robert Stanislaro
FTI Consulting
T: +1 212 850 5657
robert.stanislaro@fticonsulting.com
Financial Tables
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
| | June 30, | | December 31, |
| | 2024 | | 2023 |
| | €1,000 | | €1,000 |
Assets | |
| |
|
Current assets | |
| |
|
Cash and cash equivalents | | 78,970 | | 118,925 |
Financial asset - current | | 17,183 | | — |
Prepayments and other receivables | | 5,116 | | 1,538 |
Other taxes | | 497 | | 523 |
| | | | |
Total current assets | | 101,766 | | 120,986 |
| | | | |
Property, plant and equipment | | 16,285 | | 16,897 |
Investments in financial assets | | — | | — |
| | | | |
Total assets | | 118,051 | | 137,883 |
| | | | |
Equity and liabilities | |
| |
|
Equity | | | |
|
Equity attributable to owners of the Company | | 32,854 | | 41,390 |
Total equity | | 32,854 | | 41,390 |
| | | | |
Current liabilities | |
| |
|
Borrowings | | 4,437 | | — |
Lease liabilities | | 1,675 | | 1,614 |
Derivative financial instruments | | 184 | | 311 |
Trade payables | | 33 | | 1,541 |
Social securities and other taxes | | 1,448 | | 1,659 |
Deferred income | | 23,827 | | 20,569 |
Other current liabilities | | 5,675 | | 8,509 |
| | | | |
Total current liabilities | | 37,279 | | 34,203 |
| | | | |
Borrowings | | — | | 4,292 |
Lease liabilities | | 13,078 | | 13,828 |
Deferred income | | 34,840 | | 44,170 |
| | | | |
Total liabilities | | 85,197 | | 96,493 |
| | | | |
Total equity and liabilities | | 118,051 | | 137,883 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI
(€ in thousands, except share and per share data)
| | Three month period | | Six month period | ||||
| | ended June 30, |
| ended June 30, | ||||
|
| 2024 | | 2023 |
| 2024 | | 2023 |
| | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Revenue | | 6,305 | | 1,205 | | 10,755 | | 1,860 |
| | | | | | | | |
Other income | | 156 | | 38 | | 366 | | 80 |
| | | | | | | | |
Research and development costs | | (7,048) | | (5,909) | | (16,331) | | (11,969) |
General and administrative costs | | (3,013) | | (4,145) | | (6,465) | | (8,171) |
Total operating costs | | (10,061) | | (10,054) | | (22,796) | | (20,140) |
| |
| |
| |
| |
|
Operating result | | (3,600) | | (8,811) | | (11,675) | | (18,200) |
Finance income and expense | | 513 | | 470 | | 1,001 | | (74) |
Results related to associates and financial assets | | — | | — | | — | | — |
Results related to financial liabilities measured at fair value through profit or loss | | 195 | | 221 | | 127 | | 891 |
Results on derecognition of financial liabilities | | — | | 101 | | — | | 509 |
| |
| |
| |
| |
|
Result before corporate income taxes | | (2,892) | | (8,019) | | (10,547) | | (16,874) |
Income taxes | | 200 | | 42 | | 197 | | 42 |
| |
| |
| |
| |
|
Result for the period | | (2,692) | | (7,977) | | (10,350) | | (16,832) |
Other comprehensive income (foreign exchange differences on foreign operation) | | 85 | | 7 | | 276 | | (212) |
| |
| |
| |
| |
|
Total comprehensive income | | (2,607) | | (7,970) | | (10,074) | | (17,044) |
| | | | | | | | |
Result attributable to | |
| |
| |
| |
|
Owners of the Company | | (2,692) | | (7,993) | | (10,350) | | (16,926) |
Non-controlling interests | | — | | 16 | | — | | 94 |
| | (2,692) | | (7,977) | | (10,350) | | (16,832) |
Total comprehensive income attributable to | | | | | | | | |
Owners of the Company | | (2,607) | | (7,986) | | (10,074) | | (17,138) |
Non-controlling interests | | — | | 16 | | — | | 94 |
| | (2,607) | | (7,970) | | (10,074) | | (17,044) |
| |
| |
| |
| |
|
Share information | |
| |
| |
| |
|
Weighted average number of shares outstanding1 | | 81,665,565 | | 80,939,392 | | 81,618,038 | | 80,913,751 |
| | | | | | | | |
Earnings per share attributable to owners of the Company (Euro per share) | | | | | | | | |
Basic loss per share1 | | (0.03) | | (0.10) | | (0.13) | | (0.21) |
Diluted loss per share1 | | (0.03) | | (0.10) | | (0.13) | | (0.21) |
| 1. | For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal. |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
| | Attributable to owners of the Company | | | | | ||||||||||||
|
| Number |
| Share |
| Share |
| Equity settled |
| Translation |
| Accumulated |
| Total |
| Non- |
| Total |
|
|
| | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Balance at January 1, 2023 |
| 84,246,967 | | 3,370 | | 412,540 | | 29,052 | | 1,212 | | (379,110) | | 67,064 | | (384) | | 66,680 |
Result for the period |
| — | | — | | — | | — | | — | | (16,926) | | (16,926) | | 94 | | (16,832) |
Other comprehensive income |
| — | | — | | — | | — | | (212) | | — | | (212) | | — | | (212) |
Recognition of share-based payments |
| — | | — | | — | | 1,860 | | — | | — | | 1,860 | | — | | 1,860 |
Treasury shares transferred | | (122,584) | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | (3,873) | | — | | 3,873 | | — | | — | | — |
Share options exercised | | 122,584 | | — | | 4 | | (231) | | — | | 231 | | 4 | | — | | 4 |
| | | | | | | | | | | | | | | | | | |
Balance at June 30, 2023 |
| 84,246,967 | | 3,370 | | 412,544 | | 26,808 | | 1,000 | | (391,932) | | 51,790 | | (290) | | 51,500 |
| | | | | | | | | | | | | | | | | | |
Balance at January 1, 2024 |
| 84,248,384 | | 3,370 | | 412,894 | | 25,159 | | 817 | | (400,850) | | 41,390 | | — | | 41,390 |
Result for the period |
| — | | — | | — | | — | | — | | (10,350) | | (10,350) | | — | | (10,350) |
Other comprehensive income |
| — | | — | | — | | — | | 276 | | — | | 276 | | — | | 276 |
Recognition of share-based payments |
| — | | — | | — | | 1,364 | | — | | — | | 1,364 | | — | | 1,364 |
Treasury shares transferred | | (326,455) | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | (359) | | — | | 359 | | — | | — | | — |
Share options exercised / RSUs vested | | 326,455 | | — | | 174 | | (288) | | — | | 288 | | 174 | | — | | 174 |
|
|
| |
| |
| |
| |
| |
| |
| |
| |
|
Balance at June 30, 2024 |
| 84,248,384 | | 3,370 | | 413,068 | | 25,876 | | 1,093 | | (410,553) | | 32,854 | | — | | 32,854 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
| | Three month period | | Six month period | ||||
| | ended June 30, |
| ended June 30, | ||||
|
| 2024 | | 2023 | | 2024 | | 2023 |
| | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Cash flows from operating activities | |
| |
| |
| |
|
Net result | | (2,692) | | (7,977) | | (10,350) | | (16,832) |
Adjustments for: | | | | | | | | |
— Depreciation | | 711 | | 594 | | 1,402 | | 1,143 |
— Share-based compensation | | 628 | | 765 | | 1,364 | | 1,860 |
— Financial income and expenses | | (513) | | (470) | | (1,001) | | 74 |
— Results related to associates and financial assets | | — | | — | | — | | — |
— Results related to financial liabilities measured at fair value through profit or loss | | (195) | | (221) | | (127) | | (891) |
— Results on derecognition of financial liabilities | | — | | (101) | | — | | (509) |
— Income tax expenses | | (200) | | — | | (197) | | — |
| | | | | | | | |
Changes in working capital | | (4,614) | | (3,622) | | (13,838) | | 48,668 |
Cash (used in)/generated from operations | | (6,875) | | (11,032) | | (22,747) | | 33,513 |
| |
| |
| |
| |
|
Corporate income tax received/(paid) | | 199 | | — | | 196 | | — |
Interest received | | 610 | | 685 | | 1,542 | | 865 |
Interest paid | | (190) | | — | | (379) | | — |
| |
| |
| |
| |
|
Net cash (used in)/generated from operating activities | | (6,256) | | (10,347) | | (21,388) | | 34,378 |
| |
| |
| |
| |
|
Cash flow from investing activities | | | | | | | | |
Increase in financial asset - current | | — | | — | | (17,000) | | — |
Purchases of property, plant and equipment | | (267) | | (294) | | (999) | | (430) |
Sales of property, plant and equipment | | — | | — | | — | | 47 |
| |
| |
| |
| |
|
Net cash used in investing activities | | (267) | | (294) | | (17,999) | | (383) |
| |
| |
| |
| |
|
Cash flow from financing activities | |
| |
| |
| |
|
Proceeds from exercise of share options | | 12 | | 4 | | 174 | | 4 |
Repayment of lease liability | | (294) | | (647) | | (875) | | (906) |
| |
| |
| |
| |
|
Net cash used in financing activities | | (282) | | (643) | | (701) | | (902) |
| |
| |
| |
| |
|
Net (decrease)/increase in cash and cash equivalents | | (6,805) | | (11,284) | | (40,088) | | 33,093 |
| |
| |
| |
| |
|
Currency effect cash and cash equivalents | | 62 | | 860 | | 133 | | 694 |
Cash and cash equivalents, at beginning of the period | | 85,713 | | 138,986 | | 118,925 | | 94,775 |
| |
| |
| |
| |
|
Cash and cash equivalents at the end of the period | | 78,970 | | 128,562 | | 78,970 | | 128,562 |