CAMBRIDGE, Mass., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced that company management will present at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Wednesday, January 14, 2026 at 10:30 a.m. ET/7:30 a.m. PT. A live webcast of the presentation will be accessible HERE or b ...

Fulcrum Therapeutics, Inc. (NASDAQ:FULC) is one of the 12 best multibagger stocks to buy heading into 2026. Bullish Analyst Sentiment on Fulcrum Therapeutics (FULC) Following Positive Updates from Phase 1b PIONEER Study Following encouraging updates from the ongoing Phase 1b PIONEER study evaluating pociredir in sickle cell disease, analyst sentiment around Fulcrum Therapeutics, Inc. (NASDAQ:FULC) appears to be growing. As of December 18, 2025, 80% of analysts are bullish on Fulcrum Therapeutics, Inc. ( ...

Core Viewpoint - Fulcrum Therapeutics, Inc. has announced a public offering of 11,851,853 shares of common stock priced at $13.50 per share, aiming to raise approximately $175 million in gross proceeds before expenses [1][2]. Group 1: Offering Details - The offering includes pre-funded warrants for certain investors to purchase up to 1,111,193 shares at a price of $13.499 per warrant, reflecting a slight discount from the common stock price [1]. - Fulcrum has granted underwriters a 30-day option to purchase an additional 1,944,456 shares under the same terms [1]. - The offering is expected to close around December 11, 2025, subject to customary closing conditions [3]. Group 2: Use of Proceeds - The net proceeds from the offering will primarily be used for general corporate purposes, including working capital, capital expenditures, and research and development expenses [2]. - Specific allocations may include funding clinical trials, regulatory submissions, commercialization efforts, and potential acquisitions or investments in complementary technologies or assets [2]. Group 3: Company Background - Fulcrum Therapeutics is focused on developing small molecules for patients with genetically defined rare diseases, with its lead program targeting sickle cell disease through the modulation of gene expression [7].

Core Viewpoint - Fulcrum Therapeutics, Inc. has initiated an underwritten public offering of $150 million in common stock, with an option for underwriters to purchase an additional $22.5 million [1][2]. Group 1: Offering Details - The offering is being managed by J.P. Morgan, Leerink Partners, Cantor, Oppenheimer & Co., and Truist Securities [2]. - The shares are being offered under an effective shelf registration statement filed with the SEC on February 27, 2024, and declared effective on April 25, 2024 [3]. - A preliminary prospectus supplement and accompanying prospectus will be filed with the SEC and made available on their website [3][5]. Group 2: Use of Proceeds - The net proceeds from the offering are intended for general corporate purposes, including working capital, capital expenditures, research and development expenses, and potential acquisitions or investments [4]. Group 3: Company Overview - Fulcrum Therapeutics is focused on developing small molecules for patients with genetically defined rare diseases, with its lead program being pociredir for sickle cell disease [7]. - The company utilizes proprietary technology to identify drug targets that can modulate gene expression [7].

Core Viewpoint - Fulcrum Therapeutics Inc. is experiencing a significant stock surge following the release of promising initial results from the 20 mg dose cohort of the Phase 1b PIONEER trial for pociredir in treating sickle cell disease [1][2]. Group 1: Trial Results - The mean absolute fetal hemoglobin (HbF) increased by 9.9% at six weeks of treatment with pociredir, rising from a baseline of 7.1% to 16.9% [3]. - At the data cutoff on November 11, 2025, 58% of patients (7 out of 12) achieved absolute HbF levels of ≥20% at week six, with all patients showing a robust increase in HbF [3][4]. - A clear dose-response was observed, with a >3.75-fold mean induction of HbF at week 12 among patients in the 20 mg cohort, compared to a 2.4-fold mean induction in the 12 mg cohort [5]. Group 2: Safety and Efficacy - The proportion of F-cells (HbF-containing red blood cells) increased from a mean of 31% at baseline to 58% at week six, indicating early progression toward pan-cellular HbF induction [6]. - The safety profile in the 20 mg dose cohort remained consistent with previously reported safety data from the 12 mg cohort [6]. Group 3: Analyst Ratings and Stock Performance - HC Wainwright maintains a Buy rating and raises the price forecast from $18 to $25, while Piper Sandler reiterates an Overweight rating and raises the forecast from $16 to $23 [7]. - Cantor Fitzgerald also maintains an Overweight rating, increasing the price forecast from $15 to $24, and RBC Capital raises its forecast from $7 to $10 while maintaining a Sector Perform rating [7]. - Fulcrum Therapeutics stock is up 9.66% at $14.21 at the time of publication [7].

Pociredir 20mg Cohort Data Highlights - The 20 mg cohort data raises the bar on Pociredir's best-in-class potential for treating sickle cell disease[14, 17] - At Week 6, the 20 mg cohort (n=12) showed a 99% mean absolute increase in HbF[17] - 58% of patients (7/12) in the 20 mg cohort achieved ≥20% HbF at their latest study visit[17] - In the 20 mg cohort, the six patients who completed the 12-week treatment period achieved >375-fold induction of HbF[17] Clinical Improvements - The study observed a 28% reduction in Lactate Dehydrogenase (LDH) and a 37% reduction in indirect bilirubin, indicating consistent reductions in markers of hemolysis with the 20 mg dose[71, 72] - There was a 31% reduction in Mean Absolute Reticulocyte Count (ARC) and a 22% reduction in Mean Red Cell Distribution Width (RDW-CV) with the 20 mg dose, indicating consistent improvements in red blood cell morphology and erythropoiesis[75] - Mean hemoglobin increased from 73 g/dL to 81 g/dL in the 20 mg cohort[81] - 67% of patients (8/12) reported no vaso-occlusive crises (VOCs) during the 12-week treatment period[86] Safety and Tolerability - Pociredir at 20 mg was generally well-tolerated, with 23% of patients (3/13) reporting treatment-related adverse events[90] - There were no serious treatment-related adverse events in the 20 mg cohort[90]

Core Insights - Fulcrum Therapeutics reported positive initial results from the 20 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease, showing a clear dose-response and clinically meaningful increases in fetal hemoglobin (HbF) levels [1][2][4] Trial Design and Data Overview - The PIONEER trial is a Phase 1b open-label dose-escalation study evaluating pociredir's safety and efficacy in adult patients with severe sickle cell disease, with the 20 mg cohort consisting of 12 adults [3][13] - As of the November 11, 2025 data cutoff, all 12 patients completed the Week 6 visit, and 6 patients reached the Week 12 visit [3] Efficacy Data - At Week 6, the mean absolute HbF increased by 9.9% in the 20 mg cohort, with 58% of patients achieving HbF levels ≥20% [4][10] - A >3.75-fold mean induction of HbF was observed at Week 12 in the 20 mg cohort compared to a 2.4-fold induction in the 12 mg cohort [4][5] - The proportion of F-cells increased from 31% at baseline to 58% at Week 6, indicating early progression toward pan-cellular HbF induction [10] Safety Profile - Pociredir was generally well-tolerated, with no treatment-related serious adverse events reported as of the November 11, 2025 data cutoff [1][11] - The safety profile observed in the 20 mg cohort remained consistent with previously reported safety data from the 12 mg cohort [10][11] Future Plans - Fulcrum plans to host an investor event on December 7, 2025, to discuss the results from the PIONEER trial [1][7] - Updated results from the trial are expected to be reported in Q1 2026 [3]

Core Viewpoint - Fulcrum Therapeutics is set to present new clinical data from the Phase 1b PIONEER trial of pociredir for sickle cell disease (SCD) at an investor event on December 7, 2025, which will also be shared at the 67th American Society of Hematology Annual Meeting [1][3]. Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules aimed at improving the lives of patients with genetically defined rare diseases, particularly in areas with high unmet medical needs [6]. - The company's lead clinical program, pociredir, is designed to increase fetal hemoglobin expression for the treatment of sickle cell disease [6][8]. Clinical Trial Information - The upcoming event will provide initial clinical data from the 20 mg dose cohort and full data from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in SCD [3]. - Pociredir has shown proof-of-concept in initial data from the PIONEER trial, achieving absolute levels of fetal hemoglobin increases that may benefit patients [8]. - The drug has been generally well-tolerated in patients with SCD during the trial, with no treatment-related serious adverse events reported over three months of exposure [8]. Expert Involvement - The event will feature insights from Dr. Sheinei Alan, Director of the Inova Adult Sickle Cell Program, and Dr. Martin Steinberg, a prominent hematologist specializing in sickle cell disease [2][4][5]. - Dr. Alan leads multiple clinical trials and has contributed to the development of national guidelines for sickle cell care, while Dr. Steinberg has extensive research experience and has published over 450 articles on sickle cell disease [4][5]. Disease Context - Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and the formation of sickle-shaped red blood cells, which can cause various serious health complications [9].

Core Insights - Agios Pharmaceuticals' shares dropped approximately 51% following mixed results from the phase III RISE UP study of Pyrukynd in sickle cell disease patients [1][6] - The study met its primary endpoint of improving hemoglobin levels but failed to reduce the annualized rate of sickle cell pain crises [1][7] Efficacy Results - Nearly 41% of patients treated with Pyrukynd achieved a hemoglobin response compared to about 3% in the placebo group [2] - Pyrukynd-treated patients showed a numerical reduction in sickle cell pain crises (2.62 vs. 3.05 for placebo) but did not achieve statistical significance [2][3] - Statistically significant improvements were observed in average hemoglobin concentration and indirect bilirubin levels, but no improvement in patient-reported fatigue was noted [3] Regulatory Plans - Despite mixed results, Agios plans to proceed with a regulatory filing for Pyrukynd, focusing on a subgroup of hemoglobin responders who showed clearer clinical benefits [4] - The company aims to submit this filing after discussions with the FDA in the first quarter of 2026 [4] Current Drug Status - Pyrukynd is already approved in the U.S. and Europe for treating hemolytic anemia in adults with pyruvate kinase deficiency [5] - A regulatory filing for label expansion to include non-transfusion-dependent and transfusion-dependent thalassemia patients is under FDA review, with a decision expected by December 7, 2025 [5] Stock Performance - Following the study results, Agios shares reached a 52-week low of $22.24, reflecting investor disappointment [6] - Year-to-date, Agios shares have declined by 32%, contrasting with a 17% growth in the industry [6] Competitive Landscape - Shares of Fulcrum Therapeutics rose over 18% after Agios' announcement, as Fulcrum is developing a different investigational drug for sickle cell disease [9]

Core Insights - Fulcrum Therapeutics is set to present new clinical data from the Phase 1b PIONEER trial of pociredir for sickle cell disease at the upcoming ASH Annual Meeting [1][2] - The company will also showcase preclinical data on its calmodulin pathway modulator program aimed at treating bone marrow failure syndromes [1] Presentation Details - The presentation titled "Pociredir, a novel oral once-daily fetal hemoglobin inducer: Results from the Phase 1b PIONEER study in adult participants with severe sickle cell disease and hydroxyurea intolerance or unresponsiveness" will occur on December 6, 2025, from 5:30 – 7:30 PM ET [2] - Another presentation titled "First-in-class small molecule calmodulin pathway modulators attenuate excess p53 activity and correct erythropoietic defects in models of diamond-blackfan anemia" will also take place on the same date and time [2] - Pociredir's poster has been selected for inclusion in the Poster Walk on Novel and Emerging Therapeutics in Erythrocyte and Iron Disorders on December 7, 2025 [2] Investor Event - Fulcrum will host a live and webcast investor event on December 7, 2025, at 7:00 AM ET in Orlando, featuring company leadership and medical experts [4] - The event will be accessible via the Investor Relations section of Fulcrum's website, with a recording available afterward [4] Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases [5] - The lead clinical program, pociredir, aims to increase fetal hemoglobin expression for treating sickle cell disease [5][7] - Pociredir has received FDA Fast Track designation and Orphan Drug Designation for sickle cell disease treatment [7] About Pociredir - Pociredir is an investigational oral small-molecule inhibitor of EED, leading to downregulation of fetal globin repressors and increased fetal hemoglobin levels [6][7] - Initial data from the PIONEER Phase 1b trial indicated proof-of-concept with well-tolerated results and no treatment-related serious adverse events reported [7] About Sickle Cell Disease - Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [8] - Patients typically experience severe clinical consequences, including anemia, pain, infections, and reduced life expectancy [8]