Release Date: August 07, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Mirum Pharmaceuticals Inc (MIRM, Financial) reported a significant increase in total net product sales, reaching $77.8 million, a 139% increase from the same quarter last year.
- The company achieved important regulatory milestones, including the submission of an NDA for Chenodal and CTX, which could lead to orphan exclusivity.
- Livmarli received approval for Cholestasis pruritus and PFIC in the US and Europe, with a US label update to include patients 12 months and older.
- Positive interim results were reported for the VISTAS PSC and VANTAGE PBC studies, indicating potential for volixibat as a treatment for Cholestatic pruritus.
- Mirum Pharmaceuticals Inc (MIRM) is on track to meet its full-year revenue guidance of $310 million to $320 million, driven by strong commercial performance across its product portfolio.
Negative Points
- Despite the revenue growth, Mirum Pharmaceuticals Inc (MIRM) reported a net loss of $24.6 million for the quarter.
- The company experienced a reduction in cash, cash equivalents, and investments, down by $7.4 million from the previous quarter.
- There is a potential risk of price reference impact on international sales due to recent price negotiations in Germany.
- The EXPAND study, targeting a heterogeneous patient population, may face challenges in enrollment and execution, with completion expected only by 2026.
- Concerns about propylene glycol toxicity were raised in the context of the PFIC label expansion, although no toxicity was observed in clinical studies.
Q & A Highlights
Q: Can you comment on the approval label across Alagille and PFIC in the US and how you plan to broaden Livmarli's reach within the PFIC segment?
A: Christopher Peetz, CEO: The label expansion for Livmarli has played out well, positioning us strongly in Europe and on equal footing in the US for both indications. The initial reception has been strong, and the data for Livmarli in both indications is compelling for prescribers.
Q: Is there any initiative to accelerate enrollment into the VISTAs and VANTAGE trials?
A: Joanne Quan, Chief Medical Officer: Enrollment is progressing well, and we are excited by the interim analysis results. We are working with existing sites and expanding as needed, sharing the excitement with investigators about the potential impact of the medicine.
Q: How do you view the novel expansion population for Cholestatic pruritus, and what is the expected duration of the study?
A: Joanne Quan, Chief Medical Officer: The EXPAND study is launching now, with completion expected in 2026. It targets a heterogeneous group of patients, and we are confident in the role of IBAT inhibitors in treating Cholestatic pruritus, extending its potential use to a wide variety of patients.
Q: Can you provide insights into the commercial opportunity for Chenodal in CTX as an on-label indication?
A: Peter Radovich, President and COO: The CTX opportunity is significant, with an estimated 1,000 to 2,000 prevalent patients in the US, but only 10% diagnosed. We are investing in disease state awareness to increase diagnosis rates and speed up the time to diagnosis, with strong interest from prescribers.
Q: Regarding the PFIC label expansion, is there any concern about propylene glycol toxicity?
A: Joanne Quan, Chief Medical Officer: We have not observed PG toxicity in our clinical studies. The FDA's concern arose with younger patients in mind, but we are confident that prescribing physicians are well-equipped to monitor and manage this risk in routine patient care.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
