SNY - Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for First Quarter 2024 | Benzinga
? Entered into a collaboration and ex-U.S. license agreement with Sanofi for the development and commercialization of losmapimod ?
? On track to report topline data for Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) in the fourth quarter of 2024 ?
? Industry veteran, Patrick Horn M.D., Ph.D., appointed as chief medical officer ?
? Conference call and webcast scheduled for 8:00 a.m. ET today ?
CAMBRIDGE, Mass., May 13, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today reported financial results for the first quarter of 2024 as well as an update to the business.
"It's a very exciting time for Fulcrum as we remain on track to report topline data for the Phase 3 REACH trial in the fourth quarter of 2024. As we prepare for the potential NDA filing and commercial launch of losmapimod in the U.S., we are extremely pleased to announce a collaboration with our new partner, Sanofi, to leverage their global reach and rare disease expertise for the benefit of patients outside the U.S." said Alex C. Sapir, Fulcrum's president and chief executive officer. "In our Phase 1b PIONEER trial of pociredir, a highly differentiated oral treatment option for sickle cell disease, we have activated additional clinical trial sites and are building key relationships with leading physicians in the SCD community. We also recently strengthened our leadership team with the addition of Pat Horn as our chief medical officer. Pat brings extensive late-stage rare disease development experience and strategic insight to help us bring potentially transformative therapies to patients."
Recent Business Highlights
- Fulcrum entered into a collaboration and license agreement with Sanofi (NASDAQ:SNY) for the development and commercialization of losmapimod. Under the collaboration and license agreement, Sanofi will obtain exclusive commercialization rights for losmapimod outside of the U.S.
- Fulcrum will receive an upfront payment of $80.0 million and is eligible to receive up to an additional $975.0 million in specified regulatory and sales-based milestones, along with tiered royalties starting in the low-teens of annual net sales of losmapimod outside the U.S. In addition, Fulcrum and Sanofi will equally share global development costs.
- REACH, the Phase 3 clinical trial evaluating losmapimod in patients with FSHD, continues to progress, and Fulcrum expects to report topline data in the fourth quarter of 2024. The trial enrolled 260 patients across sites in the United States, Canada, and Europe. There are currently no approved treatments for FSHD.
- Our Phase 2 study for losmapimod, ReDUX4, which was recently published in The Lancet Neurology, demonstrated improvements in functional outcomes (reachable workspace), structural outcomes (muscle fat infiltration), and patient-reported outcomes for patients treated with losmapimod as compared to placebo. Read the publication here.
- Activated additional clinical trial sites in the Phase 1b trial evaluating pociredir in patients with sickle cell disease (SCD). Cohort 3 of the Phase 1b trial will evaluate pociredir at the 12 mg once daily dose, followed by Cohort 4 at the 20 mg once daily dose. Each cohort is expected to enroll approximately 10 patients.
Corporate Updates
- Appointed Patrick Horn, M.D., Ph.D., as chief medical officer, a seasoned executive with over 20 years of end-to-end drug development experience spanning multiple therapeutic areas, with an emphasis on rare diseases, across both large pharmaceutical and biotech companies. Iain Fraser, MBChB, DPhil, who served as our interim chief medical officer, remains on Fulcrum's executive leadership team as SVP of early development. Together, Drs. Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.
First Quarter 2024 Financial Results
- Cash Position: As of March 31, 2024, cash, cash equivalents, and marketable securities were $213.3 million, as compared to $236.2 million as of December 31, 2023. The decrease in our cash position is due to net cash used in operating activities in 2024. Pro forma cash, cash equivalents, and marketable securities were approximately $293.3 million as of March 31, 2024, inclusive of the $80.0 million milestone due under the collaboration and license agreement with Sanofi.
- Collaboration Revenue: Collaboration revenue was zero for the three months ended March 31, 2024, as compared to $0.3 million for the three months ended March 31, 2023. The decrease of $0.3 million was attributable to the completion of our research services under our collaboration agreement with MyoKardia during the fourth quarter of 2023.
- R&D Expenses: Research and development expenses were $19.8 million for the three months ended March 31, 2024 as compared to $16.7 million for the three months ended March 31, 2023. The increase of $3.1 million was primarily due to increased costs related to the advancement of REACH.
- G&A Expenses: General and administrative expenses were $10.1 million for the three months ended March 31, 2024 as compared to $11.5 million for three months ended March 31, 2023. The decrease of $1.4 million was primarily due to decreased employee compensation costs.
- Net Loss: Net loss was $26.9 million for the three months ended March 31, 2024 as compared to $24.8 million for the three months ended March 31, 2023.
Update to Cash Runway Guidance
Based on its current operating plans, Fulcrum now expects that its cash, cash equivalents, and marketable securities as of March 31, 2024, together with the $80.0 million upfront payment to be received from Sanofi under the collaboration and license agreement, will be sufficient to fund its operating requirements into 2027.
Conference Call and Webcast
Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to review the first quarter 2024 financial results and recent business highlights. Individuals may register for the conference call by clicking the link here. Once registered, participants will receive dial-in details and unique PIN which will allow them to access the call. An audio webcast will be accessible through the Investor Relations section of the company's website at www.fulcrumtx.com or by clicking here. Following the live webcast, an archived replay will also be available.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's two lead programs in clinical development are losmapimod, a small molecule in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD), and pociredir (formerly known as FTX-6058), a small molecule designed to increase expression of fetal hemoglobin and in development for the treatment of sickle cell disease (SCD). Fulcrum uses proprietary technology ...