News Release Details
Mirum Pharmaceuticals Provides Corporate Update
- Completion of maralixibat rolling NDA targeted for Q1 2021
- Expecting first-patient-in for volixibat studies in ICP and PSC, and maralixibat study in biliary atresia in Q1 2021
- Launching volixibat primary biliary cholangitis program in H2 2021
- European commercial leader hired in anticipation of potential approval of maralixibat
“I am very excited about what 2021 holds for Mirum. In the first quarter alone, we expect to complete our rolling NDA submission for maralixibat, taking us one step closer to making this medicine available to patients with Alagille syndrome, and expect to initiate three randomized studies of maralixibat and volixibat in new indications,” said
2021 Pipeline Highlights
- Expects completion of rolling new drug application (NDA) submission to the
- Ongoing review of the marketing authorization application to the
- Anticipates randomizing patients into three clinical studies in the first quarter of 2021:
- Phase 2a/2b study (OHANA) of volixibat for the treatment of intrahepatic cholestasis of pregnancy.
- Phase 2b study (VISTAS) of volixibat for the treatment of pruritus associated with primary sclerosing cholangitis.
- Phase 2 study (EMBARK) of maralixibat for the treatment of biliary atresia.
- Planning to initiate a Phase 2b study of volixibat for the treatment of pruritus associated with primary biliary cholangitis in the second half of 2021.
Corporate Highlights - Strategically Financed for Growth
- Cash, cash equivalents and investments of
-
Gross proceeds of
$75 million from follow-on public offering of common stock in the fourth quarter. -
Gross proceeds of
$60 million , including a$10 million equity investment, from the funding arrangement withOberland Capital in the fourth quarter.
- Access to up to an additional
- Maralixibat has rare pediatric disease designation for both the treatment of pruritus associated with Alagille syndrome and the treatment of PFIC2. The designation makes maralixibat eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.
Global Launch Readiness
Today, the company announced the appointment of Alexey Kutahov, MD as its general manager, EMEA, to lead its launch readiness activities for maralixibat in
In connection with his employment, the Compensation Committee of Mirum’s Board of Directors granted Dr. Kutahov an inducement award consisting of a non-qualified stock option to purchase 55,000 shares of common stock under Mirum’s 2020 Inducement Plan. The Compensation Committee of Mirum’s Board of Directors approved the award as an inducement material to Dr. Kutahov’s employment in accordance with Nasdaq Listing Rule 5635(c)(4).
The stock option has an exercise price per share equal to
About
Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. For more information, visit MirumPharma.com.
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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 results, conduct and progress of Mirum’s ongoing and planned studies for its product candidates and the regulatory approval path for its product candidates, the strength of Mirum’s balance sheet and the adequacy of cash and cash equivalents on hand, and commercial readiness activities. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plans,” “will,” ”may,” “expects,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum’s filings with the
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