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Mar 31, 2020
Mirum Pharmaceuticals Q1 2020 Earnings Report
Reported financial results and provided business updates.
Key Takeaways
Mirum Pharmaceuticals reported its Q1 2020 financial results, highlighting a cash, cash equivalents, and investments balance of $166.1 million. The company is on track to begin the rolling submission of its NDA for maralixibat in Alagille Syndrome (ALGS) in the third quarter of 2020.
On track to begin rolling submission of NDA for maralixibat in Alagille Syndrome (ALGS) in third quarter 2020.
Cash, cash equivalents and investments balance of $166.1 million.
Bolstered balance sheet with $48.0 million gross proceeds from a follow-on public offering in January 2020.
Completed Phase 1 dose-ranging and drug-drug interaction study for volixibat to inform dose regimens for planned registrational program.
Mirum Pharmaceuticals
Mirum Pharmaceuticals
Forward Guidance
Mirum is focused on initiating the rolling submission of the NDA for maralixibat in ALGS in Q3 2020 and continuing discussions with regulators regarding the suitability of maralixibat INDIGO study data in PFIC2 patients.
Positive Outlook
- Initiate rolling submission of NDA for maralixibat in ALGS in the third quarter of 2020.
- Presentations highlighting data from maralixibat and volixibat studies planned for upcoming medical congresses including European Association for the Study of the Liver and the American Association for the Study of Liver Diseases.
- Initiate ALGS expanded access program in the United States and Canada in the fourth quarter of 2020.
- Discuss with regulators the suitability of maralixibat INDIGO study 5-year outcomes data in PFIC2 patients compared to natural history for potential regulatory submission.
- US launch for maralixibat in ALGS remains on track for 2021.
Challenges Ahead
- COVID-19 pandemic has presented challenges to maintaining business continuity.
- There have been, and may continue to be delays in the advancement of some of the company’s programs, including the completion of enrollment for the Phase 3 MARCH study in PFIC2 patients.
- Mirum is unable to predict with confidence the duration of such delays.
- Mirum is withdrawing previous guidance regarding the timing of the initiation and enrollment of the ongoing and planned maralixibat and volixibat studies.
- Delays or difficulties in enrolling patients in its ongoing and planned clinical trials due to COVID-19 pandemic.