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 ...

CAMBRIDGE, Mass., May 29, 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 upcoming presentations at the 5th Global Congress on Sickle Cell Disease and the 2025 European Hematology Association Congress. The Company’s presentations are listed below. Following congress publication, copies of the presentatio ...

CAMBRIDGE, Mass., May 29, 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 management will participate in a fireside chat at the 46th Annual Goldman Sachs Global Healthcare Conference in Miami, FL on Wednesday, June 11, 2025 at 8:00 am ET. The webcast of the fireside chat will be accessible HERE or b ...

Fulcrum Therapeutics Inc (FULC) 2025 Conference Summary Company Overview - Fulcrum Therapeutics is a SMIDGAP biotech company focused on rare benign hematological conditions, with its lead program, Posiridir, targeting sickle cell disease currently in Phase 1b studies [4][2] Market Opportunity in Sickle Cell Disease - Sickle cell disease affects approximately 100,000 people in the U.S. and 4.4 million globally, with a significantly decreased life expectancy of 20 to 25 years [6][6] - Recent drug approvals have not met expectations; Voxelotor was pulled from the market, and Crizanlizumab failed to show significant efficacy in reducing vaso-occlusive crises [7][8] - The unmet need in the sickle cell market remains extremely high, creating an opportunity for Posiridir [8][8] Mechanism of Action - Posiridir induces fetal hemoglobin (HBF) expression, which can mitigate the effects of sickle hemoglobin by diluting it and preventing sickling under hypoxic conditions [10][12] - The drug works by inhibiting the PRC2 enzyme complex, leading to an altered gene expression profile that upregulates the HBG gene responsible for fetal hemoglobin production [11][12] Clinical Development and Regulatory Updates - A clinical hold was placed by the FDA in early 2023 due to preclinical findings of hematological malignancies, but the hold was lifted after redefining the patient population to include more severely affected patients [14][16][18] - The new inclusion criteria restrict concomitant use of hydroxyurea, which is labeled as a carcinogen, to ensure patient safety during early development [19][21] Patient Population and Enrollment - Approximately 10% of the 100,000 U.S. sickle cell patients meet the new inclusion criteria, equating to about 10,000 potential participants for the study [22][22] - The company anticipates that demonstrating the drug's efficacy in increasing fetal hemoglobin levels could lead to a relaxation of these criteria in future studies [24][24] Efficacy and Clinical Meaningfulness - There is a strong correlation between increased fetal hemoglobin levels and reduced vaso-occlusive crises, with a target of achieving a clinically meaningful reduction of at least 25% in these crises [29][30] - Initial cohorts showed dose-dependent increases in fetal hemoglobin, with higher doses leading to more significant increases [26][27] Competitive Landscape - Renewed interest in fetal hemoglobin as a treatment mechanism has emerged, with several competitors entering the field, including BMS, Novartis, and GSK, although they are at earlier stages of development compared to Fulcrum [33][34] Financial Position and Future Aspirations - Fulcrum has a cash position of approximately $226 million, with an annual burn rate of $55 to $65 million, providing a runway until at least 2027 [40][41] - The company aims to expand beyond sickle cell disease and become a leader in benign hematological rare conditions [41][42]

Core Viewpoint - Fulcrum Therapeutics, Inc. is actively participating in upcoming conferences to engage with investors and stakeholders, highlighting its focus on developing treatments for genetically defined rare diseases [1][3]. Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing small molecules aimed at improving the lives of patients with genetically defined rare diseases, particularly in areas with high unmet medical needs [2]. - The company's lead clinical program is pociredir, a small molecule designed to enhance fetal hemoglobin expression for treating sickle cell disease (SCD) [2]. - Fulcrum employs proprietary technology to identify drug targets that can modulate gene expression, addressing the root causes of gene mis-expression [2]. Upcoming Events - The company will participate in the H.C. Wainwright BioConnect Investor Conference on May 20, 2025, in New York, New York [3]. - Fulcrum will also take part in the RBC Capital Markets Global Healthcare Conference on May 21, 2025, with a fireside chat scheduled for 3:35 pm ET [3].

Fulcrum Therapeutics Inc (FULC) Q1 2025 Earnings Call May 01, 2025 08:00 AM ET Company Participants Alex Sapir - CEO, President, & Board MemberAlan Musso - Chief Financial OfficerJoseph Schwartz - Senior Managing DirectorIain Fraser - Senior Vice President of Early DevelopmentMatthew Biegler - Managing DirectorAnish Nikhanj - Senior Associate - Biotechnology Equity Research Conference Call Participants Edward Tenthoff - MD & Senior Research AnalystKristen Kluska - Equity Research Analyst Operator Good morni ...

Fulcrum Therapeutics Inc (FULC) Q1 2025 Earnings Call May 01, 2025 08:00 AM ET Company Participants Alex Sapir - CEO, President, & Board MemberAlan Musso - Chief Financial OfficerJoseph Schwartz - Senior Managing DirectorIain Fraser - Senior Vice President of Early DevelopmentMatthew Biegler - Managing DirectorAnish Nikhanj - Senior Associate - Biotechnology Equity Research Conference Call Participants Edward Tenthoff - MD & Senior Research AnalystKristen Kluska - Equity Research Analyst Operator Good morni ...

Fulcrum Therapeutics Inc (FULC) Q1 2025 Earnings Call May 01, 2025 08:00 AM ET Speaker0 Good morning, and welcome to Falcon Therapeutics First Quarter twenty twenty five Financial Results and Business Update Conference Call. Currently, all participants are in a listen only mode. This Speaker1 call Speaker0 is being webcast live and can be accessed on the Investors section of Falcum's website at www.falcumtx.com and is being recorded. Please be reminded that remarks during this call may contain forward looki ...

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 March 31, 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 jur ...

Clinical Trials - Enrollment in the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD) is complete with 16 patients, and the 20 mg dose cohort has been initiated[2]. - Two abstracts accepted for presentation at the 2025 European Hematology Association Congress will highlight preclinical and clinical data related to pociredir[5]. - Pociredir has received FDA Fast Track designation and Orphan Drug Designation for the treatment of SCD[10]. Financial Performance - Cash, cash equivalents, and marketable securities as of March 31, 2025, were $226.6 million, down from $241.0 million as of December 31, 2024, reflecting a decrease of $14.4 million[12]. - Research and development expenses for Q1 2025 were $13.4 million, a decrease of 32.4% compared to $19.8 million in Q1 2024[12]. - General and administrative expenses for Q1 2025 were $7.0 million, down 30.9% from $10.1 million in Q1 2024[12]. - Net loss for Q1 2025 was $17.7 million, compared to a net loss of $26.9 million in Q1 2024, representing a 34.2% improvement[12]. - The total stockholders' equity as of March 31, 2025, was $228.5 million, down from $243.0 million as of December 31, 2024[15]. Future Plans - The company expects its current cash position to fund operations into at least 2027[7]. - The company plans to submit an investigational new drug application (IND) for Diamond-Blackfan anemia during Q4 2025[5].