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Jun 30, 2020
Mirum Pharmaceuticals Q2 2020 Earnings Report
Mirum Pharmaceuticals reported financial results for Q2 2020 and provided clinical program updates.
Key Takeaways
Mirum Pharmaceuticals reported a net loss of $23.25 million for the second quarter ended June 30, 2020. The company's cash, cash equivalents, and investments totaled $149.3 million as of June 30, 2020. Significant progress was made toward making maralixibat available as an alternative to liver transplantation.
Initiation of rolling NDA submission for maralixibat in ALGS is on track for Q3 2020.
Submission of European marketing authorization application for maralixibat in PFIC2 planned for Q4 2020.
Expanded Access Program for maralixibat in ALGS planned to open for registration in September 2020.
Orphan Drug Designation received by European Medicines Agency for maralixibat in biliary atresia.
Mirum Pharmaceuticals
Mirum Pharmaceuticals
Forward Guidance
Mirum Pharmaceuticals is focused on advancing its pipeline of novel therapies for debilitating liver diseases, with key milestones expected in the near term.
Positive Outlook
- Rolling NDA submission for maralixibat in ALGS to be initiated in third quarter 2020.
- Planning for potential launch in ALGS in the second half of 2021.
- Submission of MAA to European regulators for maralixibat in the treatment of patients with PFIC2 on track for fourth quarter of 2020.
- Enrollment ongoing in Phase 3 MARCH clinical trial in PFIC.
- Phase 2 study initiation planned for maralixibat in biliary atresia.
Challenges Ahead
- The company is still operating at a loss.
- The company is dependent on the success of its drug candidates.
- Regulatory approvals are not guaranteed.
- Clinical trials may not be successful.
- The company may need to raise additional capital in the future.