Release Date: July 31, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Fulcrum Therapeutics Inc (FULC, Financial) is on track to report top-line data for the Phase 3 REACH trial of losmapimod by the end of October, ahead of previous guidance.
- The collaboration with Sanofi for the development and commercialization of losmapimod outside the US is progressing well, with an $80 million upfront payment received.
- A high percentage (98%) of patients from the REACH trial opted to enroll in the open-label extension, indicating strong patient interest and potential efficacy.
- Fulcrum's cash position is strong, with $273.8 million in cash, cash equivalents, and marketable securities, expected to fund operations into 2027.
- The company is preparing for the potential NDA filing and US commercial launch of losmapimod, indicating readiness for market entry.
Negative Points
- The Phase 1b PIONEER trial for pociredir is experiencing delays due to long site activation lead times and narrow inclusion-exclusion criteria.
- Fulcrum expects to remain in a loss position for the foreseeable future, excluding potential milestone payments from the Sanofi collaboration.
- There are no approved therapies for FSHD, and the success of losmapimod is crucial for addressing this unmet need.
- The company faces challenges in establishing the value proposition for losmapimod with payers, particularly regarding genetic testing requirements.
- The clinical trial for pociredir is taking longer than expected, with data not anticipated until 2025, delaying potential advancements in sickle cell disease treatment.
Q & A Highlights
Q: Can you provide details on the validation of the reachable workspace metric for the FDA and the cash runway guidance into 2027?
A: The FDA has not specified numerical criteria for the reachable workspace metric, as it hasn't been used for approvals before. We are evaluating it to understand meaningful score differences. Regarding the cash runway, it anticipates full development, filing, and commercialization of losmapimod, completion of the pociredir trial, and ongoing preclinical work, funding operations into 2027. - Alex Sapir, CEO; Iain Fraser, SVP of Development; Alan Musso, CFO
Q: Does the recent data from another company using the reachable workspace endpoint help de-risk this endpoint for FSHD?
A: It's encouraging to see others using this endpoint, reinforcing its validity. The FDA hasn't suggested an alternative primary endpoint for FSHD, which is promising. The recent AVIDITY data validates the DUX4 pathway and supports reachable workspace as a clinical endpoint. - Iain Fraser, SVP of Development; Alex Sapir, CEO
Q: Why is it taking longer to proceed with Cohort 3 of the pociredir trial, and what initiatives are in place to accelerate it?
A: The delay is due to the need to activate new sites with patients matching the revised inclusion criteria post-clinical hold. We're expediting contract reviews and prioritizing site activations, aiming for 20 sites to enroll the next cohorts. - Alex Sapir, CEO
Q: Can you discuss the output from defining the minimally clinically relevant difference on the reachable workspace score for losmapimod?
A: The meaningful score difference is for within-patient change, not a mean score for the population. It will likely reflect proportions of patients exceeding this difference, and we'll report it with the top-line data. - Iain Fraser, SVP of Development
Q: What are the potential challenges and tailwinds in establishing the value proposition for losmapimod with payers?
A: Payers may find it difficult to restrict access due to the lack of available treatments for FSHD. Genetic testing will likely be required, and we're ensuring it won't impede access. Payers expect pricing similar to other rare disease drugs. - Alex Sapir, CEO
Q: Have discussions with the FDA included requests for more data on losmapimod's mechanism of action?
A: The FDA has not requested additional biomarker data. The focus has been on functional endpoints, and our preclinical data robustly shows losmapimod's mechanism of action on DUX4 reduction. - Iain Fraser, SVP of Development
Q: Is the guidance for REACH trial results by the end of October a revision from the previous year-end guidance?
A: It's a fine-tuning of the timeline as we approach the final patient visits, and we are confident in reporting top-line results by the end of October. - Alex Sapir, CEO
Q: How are you coordinating the urgency of building a commercial organization for losmapimod?
A: We are building out our commercial team and expect to announce a Chief Commercial Officer soon. The launch will be properly resourced, and our cash runway guidance includes a well-funded launch in 2026. - Alex Sapir, CEO
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
