CAMBRIDGE, Mass., March 04, 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 Leerink Partners Global Healthcare Conference in Miami, FL on March 11, 2025 at 8:40 am ET. The webcast of the fireside chat will be accessible HERE and by visiting the " ...

Fulcrum Therapeutics, Inc. (NASDAQ:FULC) Q4 2024 Earnings Conference Call February 25, 2025 8:00 AM ET Company Participants Alex Sapir - President & CEO Alan Musso - CFO Iain Fraser - SVP, Early Development Conference Call Participants Rick Miller - Cantor Fitzgerald Joseph Schwartz - Leerink Partners Matthew Biegler - Oppenheimer Edward Tenthoff - Piper Sandler Anish Nikhanj - RBC Capital Operator Good morning, and welcome to Fulcrum Therapeutics Fourth Quarter and Full Year 2024 Financial Results and Busi ...

Financial Performance - The company reported net losses of $9.7 million and $97.3 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $519.4 million as of December 31, 2024[447]. - The company has not generated any revenue from product sales and does not expect to do so for several years, if at all[451]. - Collaboration revenue increased by $77.2 million from $2.8 million in 2023 to $80.0 million in 2024, primarily due to an upfront license payment from the Sanofi collaboration agreement[472]. - The company expects substantial additional funding will be needed to support ongoing operations and growth strategy[448]. - The company expects to incur substantial operating losses and negative operating cash flows for the foreseeable future due to ongoing research and development activities[484]. Research and Development - Research and development expenses totaled $63.4 million for the year ended December 31, 2024, a decrease from $71.8 million in 2023[463]. - The company plans to submit an IND for DBA during the fourth quarter of 2025, focusing on inherited aplastic anemias[445]. - The company has reinitiated the Phase 1b clinical trial for pociredir at the 12 mg dose level, with 10 patients enrolled and expected clinical data by mid-2025[442]. - The Phase 3 REACH trial of losmapimod did not achieve its primary endpoint, leading to the suspension of its future development[443]. - A strategic plan was announced to reprioritize R&D activities, resulting in a workforce reduction from 80 to 51 employees, expected to save approximately $10.0 million annually starting Q1 2025[444]. - Research and development expenses decreased by $8.4 million from $71.8 million in 2023 to $63.4 million in 2024, mainly due to reduced external research costs and laboratory expenses[473][474]. Cash and Funding - The company had $241.0 million in cash, cash equivalents, and marketable securities as of December 31, 2024, expected to fund operations into at least 2027[450]. - Net cash used in operating activities was $2.2 million in 2024, a decrease of $88.8 million compared to $91.0 million in 2023, largely due to the $80.0 million upfront license payment received[481]. - Net cash provided by investing activities was $32.2 million in 2024, an increase of $68.9 million compared to net cash used of $36.7 million in 2023, driven by net maturities of marketable securities[482]. - The company entered into a controlled equity offering agreement in February 2024 for an at-the-market offering program with an aggregate offering price of up to $100.0 million[478]. - The company expects to finance cash needs through equity offerings, debt financings, and collaboration arrangements, with no current credit facility or committed sources of capital[489]. Expenses and Cost Management - General and administrative expenses decreased by $5.2 million from $41.7 million in 2023 to $36.4 million in 2024, attributed to lower employee compensation and facility costs[475]. - A restructuring plan announced in September 2024 reduced the workforce from 80 to 51 full-time employees to focus on key research and development activities[469]. - Stock-based compensation expense is expected to increase in future periods due to existing unrecognized expenses and additional awards granted[505]. Accounting and Financial Reporting - The company recognizes revenue under ASC 606 when control of promised goods or services is obtained, reflecting expected consideration[494]. - The company assesses collaboration arrangements to determine if they involve joint operating activities and applies appropriate recognition methods[492]. - The company accounts for uncertain tax positions using a more-likely-than-not threshold, with no uncertain tax positions reported as of each balance sheet date[508]. - The company evaluates its estimates and assumptions regarding accrued expenses on an ongoing basis, with no material differences reported to date[502]. Market and Economic Conditions - The company is exposed to interest rate sensitivity, but a 10% change in interest rates would not materially affect the fair market value of its investment portfolio[510]. - The company does not currently hedge foreign currency exchange rate risk, with minimal or no liabilities denominated in foreign currencies as of December 31, 2024[511]. - Inflation has not had a material effect on the company's business or financial condition during the years ended December 31, 2024, and 2023[512].

Financial Position - Fulcrum Therapeutics expects to report approximately $240 million in cash, cash equivalents, and marketable securities as of December 31, 2024[4] - The financial results for the fourth quarter and fiscal year ended December 31, 2024, are still being finalized and are subject to change[5] - The audit of Fulcrum's consolidated financial statements for the year ended December 31, 2024, is ongoing[5] - The information provided in the corporate presentation is unaudited and preliminary, lacking comprehensive details on Fulcrum's financial condition[5] - The report does not include all necessary information for a complete understanding of Fulcrum's financial condition[5] Corporate Information - The company is classified as an emerging growth company under the Securities Act[3] - Fulcrum's common stock is traded on the Nasdaq Global Market under the symbol FULC[2] - The company has not elected to use the extended transition period for complying with new financial accounting standards[3] - The information in the report is not deemed "filed" for purposes of the Securities Exchange Act[6] Corporate Presentation - An updated corporate presentation was published on January 13, 2025, which includes preliminary financial information[7]

― On track to provide clinical data from the 12 mg dose cohort from the Phase 1b PIONEER trial of pociredir in SCD in mid-2025 and the 20 mg dose cohort by the end of 2025 ― ― Ended 2024 with $241.0 million in cash, cash equivalents, and marketable securities; cash runway into at least 2027 ― ― Conference call and webcast scheduled for 8:00 a.m. ET today ― CAMBRIDGE, Mass., Feb. 25, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focu ...

Core Viewpoint - Fulcrum Therapeutics, Inc. is set to release its fourth quarter and full year 2024 financial results on February 25, 2025, and will host a conference call to discuss these results and recent corporate developments [1]. Group 1: Financial Results Announcement - The financial results will be released before the U.S. financial markets open on February 25, 2025 [1]. - A conference call and webcast will take place at 8:00 a.m. ET on the same day to discuss the results [1]. Group 2: Company Overview - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases [3]. - The company's lead program is pociredir, aimed at increasing fetal hemoglobin expression for treating sickle cell disease [3]. - Fulcrum employs proprietary technology to identify drug targets that can modulate gene expression to address the root causes of gene mis-expression [3].

Core Insights - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases [3] - The company will participate in a fireside chat at the Oppenheimer 35th Annual Healthcare Life Sciences Conference on February 12, 2025 [1] - Fulcrum's lead program is pociredir, aimed at increasing fetal hemoglobin expression for treating sickle cell disease [3] Company Overview - Fulcrum Therapeutics specializes in addressing high unmet medical needs through proprietary technology that targets gene expression [3] - The company is actively developing treatments for rare diseases, particularly sickle cell disease, which is a significant area of focus [3] Event Information - The fireside chat will be accessible via a webcast, with a replay available for at least 30 days post-presentation [2]

Core Viewpoint - Fulcrum Therapeutics, Inc. (FULC) has received an upgrade to Zacks Rank 2 (Buy), indicating a positive trend in earnings estimates which is a significant factor influencing stock prices [1][3]. Earnings Estimates and Stock Price Impact - The Zacks rating system emphasizes the importance of earnings estimate revisions, which are strongly correlated with near-term stock price movements [4][6]. - An increase in earnings estimates typically leads to higher fair value calculations by institutional investors, resulting in stock price movements [4]. Recent Performance and Projections - For the fiscal year ending December 2024, Fulcrum Therapeutics is projected to earn -$0.16 per share, reflecting an 89.9% change from the previous year [8]. - Over the past three months, the Zacks Consensus Estimate for Fulcrum Therapeutics has increased by 66.8%, indicating a positive outlook [8]. Zacks Rating System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with only the top 20% receiving a 'Strong Buy' or 'Buy' rating [9][10]. - The upgrade of Fulcrum Therapeutics to Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, suggesting potential for market-beating returns in the near term [11].

Core Insights - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases [2] - The company will participate in multiple healthcare conferences in December 2024, including the Piper Sandler 36th Annual Healthcare Conference, Citi 2024 Global Healthcare Conference, and Evercore ISI 7th Annual HealthCONx Conference [1] Company Overview - Fulcrum Therapeutics specializes in small molecules aimed at improving the lives of patients with rare diseases that have high unmet medical needs [2] - The lead program in clinical development is pociredir, which is designed to increase fetal hemoglobin expression for treating sickle cell disease [2] - The company employs proprietary technology to identify drug targets that can modulate gene expression to address the root causes of gene misexpression [2]

Financial Data and Key Metrics Changes - As of September 30, 2024, cash, cash equivalents, and marketable securities were $257.2 million, an increase from $236.2 million as of December 31, 2023, primarily due to an $80 million upfront payment from Sanofi [20] - Collaboration revenue was $0 in Q3 2024, down from $0.8 million in Q3 2023, due to the completion of research services under a collaboration agreement with MyoKardia [21] - Research and development expenses decreased to $14.6 million in Q3 2024 from $18.2 million in Q3 2023, mainly due to cost-sharing reimbursements from Sanofi [22] - General and administrative expenses were $8.4 million in Q3 2024, down from $10 million in Q3 2023, attributed to reduced employee compensation costs following workforce reductions [23] - The net loss for Q3 2024 was $21.7 million, compared to a net loss of $24 million in Q3 2023 [23] - The company expects to end 2024 with approximately $240 million in cash and anticipates a cash burn of $55 million to $65 million in 2025, with sufficient funds to support operations into at least 2027 [24] Business Line Data and Key Metrics Changes - The development of losmapimod was suspended after failing to meet primary and key secondary endpoints in the Phase 3 REACH trial, leading to a workforce reduction of approximately 40% [7][8] - The focus has shifted to advancing pociredir, an oral HbF inducer for sickle cell disease, with ongoing Phase 1b trials and plans for additional studies in healthy volunteers [9][11][13] Market Data and Key Metrics Changes - Sickle cell disease affects approximately 100,000 people in the U.S. and 4.4 million people worldwide, highlighting a significant unmet need for effective treatments [9][10] - The recent withdrawal of OXBRYTA underscores the demand for safe and accessible oral therapeutic options for sickle cell disease [10] Company Strategy and Development Direction - The company is committed to improving the lives of patients with genetically defined diseases and is focusing on advancing pociredir and early-stage development programs in inherited aplastic anemia [8][15] - The management team is exploring potential licensing opportunities for markets outside the U.S. while prioritizing internal development of promising preclinical programs [29] Management's Comments on Operating Environment and Future Outlook - Management expressed disappointment over the losmapimod trial results but remains committed to sharing full trial results and advancing pociredir [8] - The company is optimistic about the potential of pociredir to address unmet needs in sickle cell disease and plans to provide more detailed guidance on data sharing in early 2025 [11][41] Other Important Information - Rachel King joined the Board of Directors, bringing extensive experience in biotech, while Pat Horn, the Chief Medical Officer, will retire at the end of the year [16][18] - The company has initiated Phase 1 clinical trials of pociredir in healthy volunteers to support its comprehensive development program [13] Q&A Session Summary Question: High-level question on platform technology and pipeline - The company has a rich preclinical pipeline, focusing on inherited aplastic anemias and plans to announce updates on development candidates soon [27] Question: Feedback from FDA regarding healthy volunteer studies - The FDA did not require additional clinical data to resume studies in healthy volunteers, and these studies are part of the routine development of the compound [32][34] Question: Guidance on data rollout from the PIONEER trial - The company intends to share data from the 12 mg and 20 mg cohorts at different times in 2025, with more specific guidance to be provided early in the new year [40][41] Question: Balancing early discovery programs and external assets - The company has a robust balance sheet and is focused on advancing internal programs while remaining open to selective external opportunities [57] Question: Activation of additional sites for the PIONEER trial - The company has activated 12 sites and is working to onboard additional sites, with a goal of reaching 20 by the end of the year [62][64] Question: Funding for broader patient scope in the pociredir program - The company plans to approach the FDA about expanding the patient population after completing the current study with more severe patients [70][72]