Fulcrum Therapeutics announced results from the 12 mg dose cohort of the PIONEER trial for sickle cell disease, showing promising outcomes with pociredir. The drug led to robust increases in fetal hemoglobin, improvements in anemia, and no severe adverse events. Data from the 20 mg cohort is expected by the end of 2025, and the company ended Q2 2025 with $214.1 million in cash, ensuring financial stability until 2028.

In the second quarter of 2025, Fulcrum reported a cash position of $214.1 million, collaboration revenue was zero, R&D expenses decreased to $13.0 million, and G&A expenses lowered to $6.8 million. Net loss for the quarter was $17.3 million. Fulcrum now expects its cash to last into 2028 to support ongoing operations and development of treatments for rare diseases.

Fulcrum Therapeutics focuses on developing small molecules for rare genetic diseases. Their lead program, pociredir, showed promising results in the PIONEER trial, increasing HbF levels in SCD patients. The drug is generally well-tolerated and could transform SCD treatment. Fulcrum aims to advance pociredir and other programs, including potential treatments for inherited aplastic anemias.

Sickle Cell Disease (SCD) is a genetic disorder affecting red blood cells due to an HBB gene mutation. This causes less efficient oxygen transport, leading to sickle-shaped cells that can block vessels and cause severe health issues. Fulcrum’s pociredir aims to increase fetal hemoglobin levels to alleviate SCD symptoms, offering hope for improved patient outcomes.

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