UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
(Exact name of Registrant as Specified in Its Charter)
| (State or Other Jurisdiction of Incorporation) |
(Commission File Number) |
(IRS Employer Identification No.) |
| |
||
| (Address of Principal Executive Offices) | (Zip Code) |
Registrant’s Telephone Number, Including Area Code:
N/A
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
| Item 2.02 | Results of Operations and Financial Condition. |
On April 12, 2023, Mirum Pharmaceuticals, Inc. (the “Company”) issued a press release announcing, among other things, the Company’s preliminary unaudited financial results for the quarter ended March 31, 2023. The full text of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in this Current Report on Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
| Item 8.01 | Other Events. |
On April 12, 2023, the Company provided the following updated overview of its business:
The Company is a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases. The Company focuses on diseases for which the unmet medical need is high and the biology for treatment is clear.
The Company’s product, LIVMARLI® (maralixibat) oral solution (“Livmarli”), a novel, orally administered, minimally-absorbed ileal bile acid transporter (“IBAT”) inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) one year of age and older in the United States and for the treatment of cholestatic pruritus in patients with ALGS two months and older in Europe. The Company believes the prevalent patient population in the United States is approximately 2,000 to 2,500 pediatric ALGS patients, which, based on the Company’s current expectations and beliefs, represents a greater than $500.0 million market opportunity. ALGS is estimated to impact one out of every 30,000 births globally. The Company markets and commercialize Livmarli in the United States and certain countries in Europe through its specialized and focused commercial team. The Company has also entered into license and distribution agreements with several rare disease companies for the commercialization of Livmarli in additional countries. The Company is also developing Livmarli for progressive familial intrahepatic cholestasis (“PFIC”) and biliary atresia. Livmarli has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (“FDA”) for ALGS and PFIC type 2. The Company has submitted positive data from its Phase 3 study of Livmarli in patients with PFIC in a supplemental New Drug Application to the FDA seeking approval for the treatment of cholestatic pruritus in patients with PFIC. The Company also expects to report topline data from its EMBARK Phase 2b clinical trial in biliary atresia in the second half of 2023.
The Company is advancing its second product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. The Company is developing volixibat for the treatment of primary sclerosing cholangitis (“PSC”) and primary biliary cholangitis (“PBC”). Volixibat has been studied in over 400 adults for up to 48 weeks. Clinical trials of volixibat have shown significant activity on IBAT and bile acid markers such as 7αC4, fecal bile acids and cholesterol, demonstrating potent biological activity. The Company expects to conduct an interim analysis of the VISTAS Phase 2b clinical trial in PSC in the second half of 2023, and report interim data from the VANTAGE Phase 2b clinical trial in PBC in the second half of 2023.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits.
| Exhibit No. |
Description | |
| 99.1 | Press Release dated April 12, 2023 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL 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.
| Mirum Pharmaceuticals, Inc. | ||||||
| Date: April 12, 2023 | By: | /s/ Christopher Peetz | ||||
| Christopher Peetz | ||||||
| President and Chief Executive Officer | ||||||
Exhibit 99.1
Mirum Pharmaceuticals Announces Preliminary Unaudited First Quarter 2023 Financial Results
FOSTER CITY, Calif – April 12, 2023 - Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced estimated revenue, including net product sales, and cash balance for the first quarter 2023.
The company’s preliminary unaudited financial results for the first quarter of 2023 include:
| • | Approximately $31.5 million of total revenue, consisting of approximately $29.0 million of product sales, net, and approximately $2.5 million of license revenue, for the three months ended March 31, 2023; |
| • | Approximately $232.1 million of cash, cash equivalents, restricted cash equivalents and short-term investments as of March 31, 2023; |
| • | Consistent to moderately increased operating expenses for the quarter ended March 31, 2023 in comparison to the quarter ended December 31, 2022. |
Additionally, the company updated its guidance for the VISTAS Phase 2b clinical trial evaluating volixibat in patients with primary sclerosing cholangitis. An update from the blinded interim analysis is expected in the second half of 2023. Interim analysis from the volixibat VANTAGE Phase 2b clinical trial in primary biliary cholangitis remains as planned in the second half of 2023, as does the topline data from the maralixibat EMBARK Phase 2b clinical trial in biliary atresia.
The company has not completed quarter-end financial close processes for the quarter ended March 31, 2023. These estimates are preliminary, have not been audited, and are subject to change upon completion of company’s financial statement closing procedures. Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of March 31, 2023.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.
Mirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe (in PFIC for patients two months of age and older). LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause side effects, including:
Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.
A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.
Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.
US Prescribing Information
EU SmPC
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved medication is LIVMARLI® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and in Europe for the same indication in patients two months of age and older. Mirum has submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC for patients three months and older) and in Europe (in PFIC for patients two months and older).
Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.
Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in two potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis. Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the company’s estimated revenue, product sales, net, cash balance, and expenses for the quarter ended March 31, 2023, as well as the anticipated timing and receipt of data and interim analysis in ongoing clinical trials. Words such as “expect” and similar expressions are intended to identify forward-looking statements. Forward-looking statements represent Mirum’s current expectations regarding future events and are subject to known and unknown risks and uncertainties that could cause actual results to differ materially from those implied by the forward-looking statements. Among those risks and uncertainties are risks and uncertainties associated with Mirum’s business in general, the impact of macroeconomic and geopolitical events, including the COVID-19 pandemic and ongoing conflict between Ukraine and Russia, and the other risks described in Mirum’s filings with the Securities and Exchange Commission. The forward-looking statements included in this press release speak only as of the date of this press release, and Mirum does not undertake to update the statements included in this press release for subsequent developments, except as may be required by law.
Contact Information
Media Contact:
Erin Murphy
Investor Contacts:
Andrew McKibben
Sam Martin
Argot Partners