Fulcrum Therapeutics (NasdaqGM:FULC) Q4 2025 Earnings call February 24, 2026 08:00 AM ET Company ParticipantsAlex C. Sapir - President and CEOCorinne Johnson - VPGregory Renza - Managing DirectorIain Fraser - SVP of Early DevelopmentJames Condulis - VP of Biotechnology Equity ResearchMartin Steinberg - Professor of Medicine, Pediatrics, Pathology, and Laboratory MedicineMatthew Biegler - Managing DirectorConference Call ParticipantsAnupam Rama - Managing Director and Senior Equity AnalystJoseph Schwartz - M ...

Fulcrum Therapeutics (NasdaqGM:FULC) Q4 2025 Earnings call February 24, 2026 08:00 AM ET Company ParticipantsAlex C. Sapir - President and CEOCorinne Johnson - VPGregory Renza - Managing DirectorIain Fraser - SVP of Early DevelopmentJames Condulis - VP of Biotechnology Equity ResearchMartin Steinberg - Professor of Medicine, Pediatrics, Pathology, and Laboratory MedicineMatthew Biegler - Managing DirectorConference Call ParticipantsAnupam Rama - Managing Director and Senior Equity AnalystJoseph Schwartz - M ...

Fulcrum Therapeutics (NasdaqGM:FULC) Q4 2025 Earnings call February 24, 2026 08:00 AM ET Speaker10Good morning, and welcome to Fulcrum Therapeutics' conference call to discuss 12-week data from the 20-milligram cohort of the phase 1b PIONEER trial of pociredir and sickle cell disease. Currently, all participants are in a listen-only mode. This call is being webcast live and can be accessed on the Investors section of Fulcrum's website at www.fulcrumtx.com, where a replay will be available. I'll now turn the ...

― Announced positive 12-week results from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD) ― ― 20 mg cohort demonstrated rapid and robust fetal hemoglobin (HbF) induction, with mean absolute HbF increasing by 12.2% from a baseline of 7.1% to 19.3% at Week 12, improvements in markers of hemolysis and anemia, and encouraging trends in vaso-occlusive crisis (VOC) reduction ― ― Fulcrum plans to initiate a potential registration-enabling trial in the second half of 20 ...

CAMBRIDGE, Mass., Feb. 17, 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, will host a conference call and webcast on Tuesday, February 24, 2026 beginning at 8:00 a.m. ET to present 12-week results from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease. Members of Fulcrum management will ...

Core Viewpoint - Fulcrum Therapeutics is focused on developing oral small molecules aimed at modifying gene expression for rare diseases, with a current emphasis on sickle cell disease and their lead asset, pociredir, which is a fetal hemoglobin inducer [4]. Group 1: Company Overview - Fulcrum Therapeutics is led by CEO Alex Sapir, who expressed gratitude for the support from JPMorgan and highlighted the quality of their recent report [2]. - The company’s strategic focus is on addressing high unmet needs in rare diseases, particularly in benign hematology [4]. Group 2: Product Focus - The lead product, pociredir, is designed to induce fetal hemoglobin, which is expected to be a best-in-class treatment for sickle cell disease [4].

Fulcrum Therapeutics Conference Call Summary Company Overview - **Company**: Fulcrum Therapeutics (NasdaqGM: FULC) - **Focus**: Development of oral small molecules to modify gene expression for rare diseases, specifically targeting sickle cell disease [1][2] Key Product Information - **Lead Asset**: Pociredir, a fetal hemoglobin inducer for sickle cell disease - **Regulatory Status**: Fast Track and Orphan designation, with patents extending to 2040 [2] - **Current Studies**: Wrapping up a Phase Ib study with plans for an end-of-phase meeting with regulatory agencies in the first half of 2026 [2][21] Financial Position - **Cash Runway**: $352 million at the end of the previous year, sufficient to fund operations through at least 2029 [3][23] Sickle Cell Disease Insights - **Prevalence**: Approximately 7.7 million people globally, with 100,000 in the U.S. and 55,000 in Europe [4] - **Impact**: Patients experience chronic pain and acute vaso-occlusive crises (VOCs), leading to significant hospitalizations and a reduction in life expectancy by over 20 years [5][6] Market Dynamics - **Recent Challenges**: Several treatments, including Adakveo and Oxbrita, have been withdrawn due to ineffectiveness, highlighting the high unmet need in sickle cell treatment [6][7] - **Current Treatment Landscape**: Includes anti-polymerization inhibitors and gene therapies, but these have limitations in patient access and effectiveness [8][9] Clinical Data Highlights - **Efficacy of Pociredir**: - Achieved a fetal hemoglobin (HbF) increase from 7% to 16.9% in the 20 mg cohort after six weeks [14][16] - 50% of patients reached HbF levels above 20%, which is associated with a significant reduction in VOCs [16][17] - Observed a reduction in VOCs from an expected 16 to only 5 during the study period [19][20] Safety Profile - **Adverse Events**: Treatment-related adverse events were generally mild, with no serious safety concerns reported [20][21] Future Milestones - **Upcoming Studies**: Plans to initiate a global registration study in the second half of 2026, with an open-label extension study for current participants [22][23] - **Regulatory Engagement**: Preparing for an end-of-phase meeting with the FDA to discuss trial design and endpoints [41][42] Conclusion - Fulcrum Therapeutics is positioned to address a significant unmet need in the treatment of sickle cell disease with its lead asset, pociredir. The company has a strong financial position and promising clinical data that support its potential for regulatory approval and market entry.

Core Viewpoint - Fulcrum Therapeutics, Inc. is set to present at the 44th Annual J.P. Morgan Healthcare Conference, highlighting its focus on developing small molecules for genetically defined rare diseases [1] Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company dedicated to improving the lives of patients with genetically defined rare diseases, particularly in areas with high unmet medical needs [3] - The company's lead clinical program is pociredir, a small molecule aimed at increasing fetal hemoglobin (HbF) expression for treating sickle cell disease (SCD) [3] - Fulcrum employs proprietary technology to identify drug targets that can modulate gene expression, addressing the root causes of gene mis-expression [3] Event Details - The presentation at the J.P. Morgan Healthcare Conference will take place on January 14, 2026, at 10:30 a.m. ET/7:30 a.m. PT [1] - A live webcast of the presentation will be available on Fulcrum Therapeutics' website, with a replay accessible for at least 30 days post-event [2]

Core Insights - Fulcrum Therapeutics, Inc. (NASDAQ:FULC) is gaining positive analyst sentiment due to encouraging updates from the Phase 1b PIONEER study for pociredir in sickle cell disease, with 80% of analysts being bullish and a median price target of $18.00, indicating a 90.40% upside potential [2] Group 1: Analyst Ratings and Price Targets - Truist raised its price target for Fulcrum Therapeutics from $14 to $18, maintaining a "Buy" rating, citing positive reception of partial Cohort 4 data as a significant factor [3] - Bank of America increased its price target from $6 to $7 but maintained an "Underperform" rating, projecting a potential 2029 launch for pociredir with expected peak sales of $1.5 billion by 2038 [4] Group 2: Clinical Study Updates - Fulcrum Therapeutics reported positive initial results from the 20 mg dose cohort of the PIONEER study, highlighting significant increases in fetal hemoglobin, a clear dose-response relationship, and a favorable safety profile with no serious side effects [5] Group 3: Financial Developments - The company priced a public offering on December 10, 2025, expected to raise $175 million in gross proceeds, enhancing its balance sheet flexibility [6]

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].