Pfizer's Sickle Cell Drug Fails to Meet Main Goal, Safety Profile Remains a Potential Asset

Pfizer's experimental sickle cell disease drug, inclacumab, has failed to meet its main goal in a late-stage clinical trial. Although the drug was well-tolerated, it did not reduce vaso-occlusive crises better than a placebo. Pfizer has faced challenges in developing effective treatments for sickle cell disease, and the drug's failure adds to these challenges. Analysts have a Moderate Buy consensus rating on PFE stock with an average price target of $28.59, implying 13.5% upside potential.

Pfizer Inc. (NYSE: PFE) has reported that its experimental sickle cell disease drug, inclacumab, did not meet its primary endpoints in a Phase 3 clinical trial. The drug, an investigational P-selectin inhibitor, aimed to reduce vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD). However, the study, known as THRIVE-131, did not demonstrate a significant reduction in the rate of VOCs compared to a placebo.

The trial, which enrolled 241 participants aged 16 and older with SCD, was designed to evaluate the efficacy and safety of inclacumab over a 48-week period. Participants were randomized to receive either inclacumab or placebo every 12 weeks. Despite the drug being well-tolerated, with common adverse events including anemia, arthralgia, and upper respiratory tract infections, it failed to achieve the desired outcome.

Michael Vincent, M.D., Ph.D., Chief Inflammation & Immunology Officer at Pfizer, acknowledged the disappointment within the community and emphasized Pfizer's commitment to advancing research and exploring other therapeutic options for SCD. The company plans to share detailed analyses of the data with the scientific and patient community.

This setback follows the withdrawal of OXBRYTA® (voxelotor) from Pfizer's rare disease portfolio in 2024 due to concerns about an imbalance in VOCs and fatal events. Osivelotor, another investigational drug in Pfizer's SCD portfolio, is currently undergoing a Phase 3 clinical trial, with enrollment paused due to a partial clinical hold issued by the FDA.

Analysts have a Moderate Buy consensus rating on PFE stock with an average price target of $28.59, implying 13.5% upside potential. The stock's performance may be influenced by investor confidence in Pfizer's ability to navigate the challenges in developing effective treatments for SCD and other rare diseases.

References:
[1] https://www.pfizer.com/news/announcements/pfizer-provides-update-phase-3-inclacumab-study-treatment-people-sickle-cell
[2] https://www.geneonline.com/pfizers-inclacumab-fails-to-meet-primary-endpoints-in-phase-iii-sickle-cell-disease-trial/
[3] https://www.ainvest.com/news/pfizer-sickle-cell-drug-failure-implications-biopharma-risks-2508/