Fulcrum Therapeutics’ Pociredir Shows Promising Results in Phase 1b Study, Analyst Reiterates Buy Rating

Fulcrum Therapeutics' pociredir has shown promising results in a Phase 1b study, with the 12mg dose demonstrating significant efficacy and 44% of patients achieving clinically significant HbF levels. Analyst Joseph Schwartz of Leerink Partners reiterated a Buy rating and $12.00 price target, citing the potential of pociredir to benefit patients with unmet medical needs. Oppenheimer also reiterated a Buy rating with a $15.00 price target.

Fulcrum Therapeutics, Inc. (FULC) has reported encouraging results from the 12 mg dose cohort of its Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD). The study showed robust and clinically meaningful absolute mean fetal hemoglobin (HbF) induction of 8.6% from baseline at 12 weeks of treatment, with 7 of 16 patients achieving absolute HbF levels greater than 20% [1].

The study also demonstrated evidence of pan-cellular induction of HbF, with a mean increase in F-cells (red blood cells containing HbF) from 34% at baseline to 67% at 12 weeks of treatment. This indicates a significant improvement in red blood cell health, as F-cells are resistant to red blood cell sickling and hemolysis due to HbF-mediated inhibition of sickle hemoglobin (HbS) polymerization [1].

Key markers of hemolysis and anemia showed meaningful improvements with pociredir treatment. Indirect bilirubin, lactate dehydrogenase (LDH), and red cell distribution width decreased by 37%, 28%, and 27%, respectively. Reticulocyte counts, indicating healthier bone marrow function, decreased by 30%. Additionally, the mean hemoglobin concentration increased by 0.9 g/dL at 12 weeks of treatment [1].

A trend of reduced vaso-occlusive crisis (VOC) rates was observed during the study period, with 8 of 16 patients reporting no VOCs during the treatment period. Pociredir was generally well-tolerated, with no treatment-related serious adverse events (SAEs) reported and all treatment-related adverse events (AEs) limited to Grade 1 [1].

Analyst Joseph Schwartz of Leerink Partners reiterated a Buy rating and $12.00 price target, citing the potential of pociredir to benefit patients with unmet medical needs. Oppenheimer also reiterated a Buy rating with a $15.00 price target [2].

Fulcrum Therapeutics will host a conference call and webcast today at 8:00 a.m. ET to discuss the results to date from the PIONEER Phase 1b trial. The company is focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need [1].

References:

[1] https://www.globenewswire.com/news-release/2025/07/29/3123076/0/en/Fulcrum-Therapeutics-Announces-Results-from-the-12-mg-Dose-Cohort-of-the-Phase-1b-PIONEER-Trial-of-Pociredir-in-Sickle-Cell-Disease.html

[2] Leerink Partners, Oppenheimer & Co.