Fulcrum Therapeutics (FULC) Q3 Earnings call transcript Nov 13, 2024

In the latest earnings call, Fulcrum Therapeutics provided a comprehensive update on their ongoing efforts to advance their drug development pipeline, particularly focusing on the challenges and opportunities surrounding their sickle cell disease program. The call, led by CEO Alex Sapir, showcased a company in transition, with a renewed focus on its oral HBF inducer, pociredir, and strategic changes aimed at addressing the needs of patients with genetically defined diseases in high unmet need.

A Mixed Quarter: Losmapimod and the Future of Fulcrum

The call began with a discussion of the recent Phase III REACH trial results for losmapimod, which did not meet the primary and key secondary endpoints. This led to the suspension of development for this program and a significant workforce reduction. Despite this setback, Sapir expressed the company's commitment to sharing the full results of the trial and to continuing its mission of improving the lives of patients with genetically defined diseases. This strategic pivot is a clear indication of Fulcrum's resilience and focus on innovation, even in the face of challenges.

Sickle Cell Disease: A Promising New Focus

With the setback of losmapimod, Fulcrum has shifted its focus to its oral HBF inducer, pociredir, for the potential treatment of sickle cell disease. The company emphasized the significant unmet need in this area, with approximately 100,000 people in the U.S. and 4.4 million people worldwide suffering from this lifelong inherited blood disorder. The recent withdrawal of Oxbryta further underscores the need for safe and accessible oral therapeutic options that are broadly protective of sickle cell symptomatology.

Fulcrum's optimism for pociredir is based on its potential to increase fetal hemoglobin levels, which is linked to a decrease in disease severity and even asymptomatic presentation. The company is currently conducting a Phase Ib trial, the PIONEER study, which is expected to provide data in 2025. This focus on sickle cell disease reflects Fulcrum's commitment to addressing significant unmet needs and its confidence in the potential of pociredir to make a meaningful impact on patients' lives.

Financial Stability and Strategic Moves

The call also addressed Fulcrum's financial standing, with a cash position of $257.2 million as of September 30, 2024. This financial stability, coupled with the acquisition of a rich preclinical pipeline, positions Fulcrum for continued growth and innovation. The recent appointment of Rachel King to the Board of Directors, along with the addition of Thomas Winkler as Vice President of Hematology, underscores the company's strategic focus and commitment to advancing its pipeline.

Looking Ahead: Challenges and Opportunities

As Fulcrum Therapeutics navigates this period of transition, it faces both challenges and opportunities. The setback with losmapimod underscores the inherent risks of drug development, but the company's renewed focus on sickle cell disease and the potential of pociredir offers a promising path forward. With a robust financial position and a strategic focus on innovation, Fulcrum is well-positioned to continue making strides in the field of genetic medicine and to address the unmet needs of patients with genetically defined diseases.

In conclusion, Fulcrum Therapeutics' earnings call presented a mixed picture, with both challenges and opportunities. The company's strategic pivot to sickle cell disease and the potential of pociredir, along with its financial stability and strategic appointments, position Fulcrum for continued growth and innovation. As the company moves forward, it will be interesting to watch how it navigates the complexities of drug development and continues to address the unmet needs of patients with genetically defined diseases.