Fulcrum Therapeutics (NasdaqGM:FULC) FY Conference September 09, 2025 02:00 PM ET Company ParticipantsAlex Sapir - CEOConference Call ParticipantsDr. Jade Montgomery - Associate Biotech Research AnalystDr. Jade MontgomeryGood afternoon, everyone, and thank you for joining the 2025 H.C. Wainwright 27th Annual Global Investment Conference. I'm Dr. Jade Montgomery, an Associate Biotech Research Analyst at the firm, and I'd like you to please join me in welcoming from Fulcrum Therapeutics, Alex Sapir, President ...

Fulcrum Therapeutics (FULC) Conference Call Summary Company Overview - Fulcrum Therapeutics is a clinical stage biotech company focused on benign hematological conditions, developing oral small molecules to modify gene expression for rare diseases [4][5] - The lead asset in the pipeline is Posteridare for sickle cell disease, with additional programs targeting bone marrow failure syndromes [5] Industry Context - The sickle cell disease treatment landscape has seen a mix of hope and disappointment, with four therapies approved between 2019 and 2023, including two cell and gene therapies [7][8] - Recent withdrawals and failures of therapies have led to a state of despair among patients and physicians, but there is renewed hope with ongoing developments [9] Clinical Insights - Vaso-occlusive crises (VOCs) are a significant concern for sickle cell patients, with frequency being the most commonly captured endpoint in clinical trials [10][12] - Historically, the FDA has accepted a 25% to 50% reduction in VOCs as clinically meaningful [12][13] - Induction of fetal hemoglobin (HbF) is a promising mechanism for treating sickle cell disease, with evidence showing that higher HbF levels correlate with reduced VOCs [14][15][18] Recent Data and Findings - The recent phase 1B study involved a more severe patient population, with 100% of patients showing an increase in HbF levels, and nearly half exceeding 20% HbF [26][27] - The study observed a 60% reduction in expected VOCs, with eight patients experiencing zero VOCs during the study [29][30] - A dose-response relationship was noted, with lower baseline HbF levels in the more severe patient cohort compared to previous studies [32] Future Outlook - Fulcrum Therapeutics plans to present data from the 20 mg cohort by year-end and engage with the FDA in early 2026 to discuss the risk-benefit profile of Posteridare [41][42] - The company targets a market opportunity of approximately $1 billion for every 5% market share in the sickle cell treatment space, given the prevalent population of 100,000 patients in the U.S. [44] Financial Position - As of Q2, Fulcrum Therapeutics reported a balance sheet of $214 million, with an expected cash runway into 2028, supporting ongoing and future clinical programs [46] Key Takeaways - The next five years are anticipated to be transformative for sickle cell therapies, with Fulcrum Therapeutics positioned to deliver impactful treatments [47]

CAMBRIDGE, Mass., Aug. 26, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (the “Company”) (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 attend and present at the following investor conferences: Cantor Fitzgerald 2025 Annual Global Healthcare Conference Location: New York, NY, U.S.Date: September 4, 2025Time: 1:35-2:05 PM ETType: Fires ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38978 FULCRUM THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-4839948 (State or other juri ...

Exhibit 99.1 Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Second Quarter 2025 ― Announced results from the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); pociredir was generally well-tolerated with no treatment-related serious adverse events (SAEs) ― ― Observed robust and rapid pan-cellular increases in fetal hemoglobin (HbF); meaningful improvements in key markers of hemolysis and anemia; encouraging trends in vaso-oc ...

Core Insights - Fulcrum Therapeutics reported promising results from the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD), showing a robust mean increase of 8.6% in fetal hemoglobin (HbF) and improvements in key markers of hemolysis and anemia [1][5] - The company ended Q2 2025 with $214.1 million in cash, providing a cash runway into 2028 [1][6] - Collaboration revenue was zero for Q2 2025, a decrease from $80 million in Q2 2024, primarily due to the recognition of an upfront license payment from Sanofi in the previous year [5][11] Recent Business Highlights - The 12 mg dose cohort of the PIONEER trial demonstrated no treatment-related serious adverse events, indicating that pociredir was generally well-tolerated [1][5] - The ongoing 20 mg dose cohort is expected to provide clinical data by the end of 2025 [1][5] - Fulcrum is advancing its program for inherited aplastic anemias and plans to submit an investigational new drug application for Diamond-Blackfan anemia in Q4 2025 [5] Financial Results - Research and development expenses for Q2 2025 were $13 million, down from $17.3 million in Q2 2024, attributed to reduced employee compensation costs and the discontinuation of the losmapimod program [5][11] - General and administrative expenses decreased to $6.8 million in Q2 2025 from $10.2 million in Q2 2024, reflecting lower professional services and employee compensation costs [11] - The net loss for Q2 2025 was $17.3 million, compared to a net income of $55.4 million in Q2 2024 [11][16]

Core Insights - Fulcrum Therapeutics reported positive results from the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD) [2][3] Efficacy Data - The mean absolute fetal hemoglobin (HbF) increased by 8.6% from a baseline of 7.6% to 16.2% after 12 weeks of treatment, with 7 out of 16 patients achieving HbF levels greater than 20% [4][10] - The proportion of F-cells increased from 34% at baseline to 67% at 12 weeks, indicating pan-cellular induction of HbF [4][10] - Key markers of hemolysis showed meaningful improvements, including a mean increase in total hemoglobin of 0.9 g/dL, from 7.8 g/dL to 8.7 g/dL [10] - A trend of reduced vaso-occlusive crisis (VOC) rates was observed, with 50% of patients reporting no VOCs during the treatment period [10] Safety Profile - Pociredir was generally well-tolerated, with no treatment-related serious adverse events (SAEs) reported, and all treatment-related adverse events (AEs) were Grade 1 [3][10] - The safety profile observed in the 12 mg dose cohort was consistent with previously reported data [10] Company Overview - Fulcrum Therapeutics is focused on developing small molecules for genetically defined rare diseases, with pociredir as its lead clinical program aimed at increasing HbF expression for SCD treatment [8][9]

Company Overview - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases, particularly in areas with high unmet medical need [3] - The company's lead clinical program is pociredir, aimed at increasing fetal hemoglobin (HbF) levels for the treatment of sickle cell disease (SCD) [3][4] Upcoming Event - Fulcrum will host a conference call and webcast on July 29, 2025, at 8:00 a.m. ET to present topline results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in SCD [1] Pociredir Details - Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), discovered using Fulcrum's proprietary technology [4] - The drug works by downregulating fetal globin repressors, leading to increased HbF levels, which may benefit patients with SCD [4] - Pociredir has received FDA Fast Track designation and Orphan Drug Designation for SCD treatment [4] Sickle Cell Disease (SCD) Background - SCD is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [5] - Patients with SCD face serious health issues, including anemia, pain, infections, and reduced life expectancy [5]

Fulcrum Therapeutics Inc (FULC) FY Conference June 11, 2025 08:00 AM ET Speaker0 Good morning, everyone. Thanks for joining us here on day three of the Goldman Sachs Global Healthcare Conference. I'm thrilled to be joined this morning with the team from Fulcrum Therapeutics. Maybe I'll let you guys introduce yourself and then provide a brief overview of the company. Speaker1 Sure. Alex, thank you all for joining. I'm Alex Zapier. I'm the President and CEO of Fulcrum. I've been there for about two years and ...

Company Overview - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases with high unmet medical needs [2][3] - The company's lead clinical program is pociredir, designed to increase fetal hemoglobin expression for treating sickle cell disease (SCD) [2][3] Upcoming Presentations - Fulcrum Therapeutics will present at the 5th Global Congress on Sickle Cell Disease in Abuja, Nigeria, from June 3-6, 2025, and at the 2025 European Hematology Association Congress in Milan, Italy, from June 12-15, 2025 [2] - Specific presentations include preclinical and early clinical results of pociredir, a Phase 1b study evaluating safety and pharmacokinetics in SCD patients, and pharmacokinetics and safety in healthy adults [5] Pociredir Details - Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), leading to downregulation of fetal globin repressors and increased fetal hemoglobin levels [3] - Initial data showed proof-of-concept for pociredir in SCD, with well-tolerated results in clinical trials prior to the FDA lifting the clinical hold in August 2023 [3] Sickle Cell Disease Overview - 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 [4] - Patients with SCD face serious health issues, including anemia, pain, infections, and reduced life expectancy [4]