Fulcrum to present topline results from sickle cell disease trial

CAMBRIDGE, Mass. - Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biotech company with a market capitalization of $419 million that has seen its stock surge 92% over the past six months according to InvestingPro, will host a conference call and webcast on Tuesday to present topline results from the 12 mg dose cohort of its Phase 1b PIONEER trial of pociredir in sickle cell disease.

The presentation, scheduled for 8:00 a.m. ET on July 29, will feature Fulcrum management alongside Dr. Sheinei Alan from the Inova Fairfax Adult Sickle Cell Program and Dr. Wally Smith from the VCU Adult Sickle Cell Program. This announcement comes as the company maintains a strong financial position, with InvestingPro data showing more cash than debt on its balance sheet and a healthy current ratio of 28.71.

Pociredir is an investigational oral small-molecule inhibitor designed to increase fetal hemoglobin expression for the treatment of sickle cell disease. The drug works by inhibiting Embryonic Ectoderm Development (EED), which downregulates key fetal globin repressors.

Earlier clinical data demonstrated proof-of-concept with increases in fetal hemoglobin that could potentially benefit patients. The FDA previously placed the trial on clinical hold, which was lifted in August 2023. According to the company’s press release statement, pociredir was "generally well-tolerated" in people with sickle cell disease with up to three months of exposure.

The FDA has granted pociredir both Fast Track designation and Orphan Drug Designation for the treatment of sickle cell disease.

Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, resulting in abnormally shaped red blood cells that can block blood vessels. Patients typically suffer from complications including anemia, pain, infections, and organ damage.

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases. The company’s next earnings report is scheduled for August 6, 2025. For deeper insights into Fulcrum’s financial health and growth prospects, investors can access comprehensive analysis through InvestingPro’s detailed research reports, which are available for over 1,400 US stocks.

In other recent news, Fulcrum Therapeutics reported its Q1 2025 earnings, revealing a net loss of $17.7 million. This marks a significant improvement compared to the $26.9 million loss in Q1 2024. Despite a slight miss on earnings per share forecasts, the company highlighted strategic advancements in its lead program for sickle cell disease and reduced operating expenses. In addition, Leerink Partners upgraded Fulcrum Therapeutics from Market Perform to Outperform, setting a new price target of $12.00. This decision was influenced by promising results from the company’s 12mg sickle cell disease data and the potential for further improvement. Cantor Fitzgerald also maintained its Overweight rating with a $10.00 price target, citing potential upside for the sickle cell treatment. Fulcrum has garnered significant investor interest, particularly concerning its upcoming clinical data readout for pociredir. The company’s progress in resuming its phase 1b PIONEER study under CEO Alex Sapir’s leadership was also noted.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.