Here are 1-3 brief analogies for Fulcrum Therapeutics (FULC):

• A Vertex Pharmaceuticals, but focused on a pipeline of other rare genetic diseases like FSHD.

• A Sarepta Therapeutics, but focused on FSHD and other genetic muscle diseases.

• Lofexidine (formerly FTX-6058): A small molecule designed to upregulate fetal hemoglobin (HbF) expression for the potential treatment of sickle cell disease and beta-thalassemia.
• FTX-6058: A small molecule designed to upregulate fetal hemoglobin (HbF) expression for the potential treatment of sickle cell disease and beta-thalassemia.
• FTX-001: An investigational small molecule targeting the FSHD disease pathway for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Fulcrum Therapeutics (symbol: FULC) is a clinical-stage biopharmaceutical company focused on discovering and developing new medicines for serious genetic diseases.

As a clinical-stage company, Fulcrum Therapeutics does not currently have commercialized products on the market generating significant revenue from direct sales. Therefore, it does not have "major customers" in the traditional sense of companies or individuals purchasing its products.

The company's primary activities involve research, development, and clinical trials of drug candidates. Its current revenue streams are typically derived from financing activities (such as equity offerings), grants, and potential future collaboration or licensing agreements with larger pharmaceutical companies if its drug candidates advance through development and regulatory approval.

If and when Fulcrum Therapeutics successfully brings a product to market, its direct customers would likely be pharmaceutical wholesalers and distributors, or potentially a commercial partner pharmaceutical company that would handle sales and distribution to pharmacies, hospitals, and other healthcare providers.

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Alex C. Sapir, President, CEO & Board Member

Alex C. Sapir was appointed CEO and President of Fulcrum Therapeutics effective July 1, 2023. He also joined the company's board of directors. Sapir has over two decades of experience in the biopharmaceutical industry. Prior to Fulcrum, he served as CEO of ReViral Ltd, which was acquired by Pfizer in 2022. He was also previously president and CEO at Dova Pharmaceuticals, where he led its transformation from a private, pre-commercial company to a publicly traded biotech company, which was later acquired by Sobi, Inc.

Alan Musso, Chief Financial Officer

Alan Musso joined Fulcrum as Chief Financial Officer in August 2023. Before joining Fulcrum, he served as CFO of ReViral, which was acquired by Pfizer in 2022. Previously, he was CFO and Treasurer at Peloton Therapeutics Inc., where he secured $150 million in mezzanine financing and prepared the company for a NASDAQ IPO before its acquisition by Merck & Co. Musso also held CFO and Treasurer positions at other biopharmaceutical companies, including Bellicum Pharmaceuticals, Targacept, and Duramed Pharmaceuticals.

Mel Hayes, Chief Operating Officer

Mel Hayes is the Chief Operating Officer at Fulcrum Therapeutics, a position he has held since July 2022. He initially joined Fulcrum Therapeutics in September 2021 as Chief Commercial Officer. Hayes has over 25 years of experience in the biotechnology and pharmaceutical industries, with a proven track record in corporate strategy, commercial operations, and product launches across multiple therapeutic areas, including rare and specialty diseases. Prior to Fulcrum, he served as Global Head Commercial, Vice President, Rare Blood Disorders at Sanofi-Genzyme, and held leadership roles at Bioverativ (acquired by Sanofi-Genzyme), Shire, Baxalta (acquired by Shire), Bayer, and Bristol Myers Squibb.

Curtis Oltmans, Chief Legal Officer

Curtis Oltmans joined Fulcrum as General Counsel in November 2020 and currently serves as Chief Legal Officer and Corporate Secretary. He has over 25 years of experience in corporate law within leading pharmaceutical and biotechnology companies. Before Fulcrum, he was Vice President, Head of Litigation at DaVita Kidney Care, Inc. Prior to DaVita, Oltmans served as Executive Vice President, General Counsel, and Corporate Secretary at Array BioPharma Inc., which is now part of Pfizer Inc. He also held roles as Corporate Vice President and General Counsel for Novo Nordisk, Inc., North America, and spent 13 years at Eli Lilly and Company in various legal capacities.

Kim Hazen, Chief People Officer

Kim Hazen joined Fulcrum in September 2017 with over 25 years of human resources experience. Most recently, she was Vice President of Human Resources at Warp Drive Bio, where she focused on building a drug discovery culture. Before joining Fulcrum and Warp Drive Bio, Hazen served as the Executive Director of Human Resources at Aegerion Pharmaceuticals and as Head of Human Resources at the Broad Institute of MIT and Harvard. Her earlier career involved human resources roles on the investment side at companies such as Pioneer Investments, Fidelity Investments, Liberty Mutual, and Bank of America.

The key risks to Fulcrum Therapeutics (FULC) business are primarily centered around the inherent challenges of drug development in the biotechnology sector.

1. Uncertainty of Clinical Trial Outcomes and Regulatory Approvals: As a clinical-stage biopharmaceutical company, Fulcrum's success is heavily reliant on the successful development and regulatory approval of its product candidates. The company's lead clinical program, pociredir for sickle cell disease (SCD), is currently in a Phase 1b study, and there is no guarantee that future data will be positive or that it will receive U.S. marketing authorization from the FDA. Fulcrum previously experienced a significant setback when its losmapimod program for facioscapulohumeral muscular dystrophy (FSHD) failed to meet its primary endpoint in a Phase 3 trial, leading to its suspension despite promising earlier-stage results. Furthermore, FTX-6058 (now pociredir) for SCD was placed on a clinical hold by the FDA in the past due to preclinical data and concerns related to other polycomb repressive complex 2 (PRC2) inhibitors, highlighting ongoing regulatory risks, even though the hold was later lifted.
2. Need for Substantial Additional Capital and Ongoing Profitability Challenges: Despite demonstrating strong financial strength in terms of liquidity and low debt, Fulcrum Therapeutics faces significant profitability challenges, including negative earnings per share and non-existent operating, net, and gross margins. The company has a negative free cash flow yield, reflecting ongoing cash flow challenges. While Fulcrum recently expanded a public offering to raise $175 million for strategic capital, and projects its current cash, cash equivalents, and marketable securities to fund operations into at least 2027, it will likely require substantial additional capital to achieve its long-term business objectives due to the high costs associated with advancing its product candidates through clinical trials.
3. Intense Competition: Fulcrum operates in highly competitive therapeutic areas. Specifically, in the sickle cell disease market, pociredir faces competition from already approved therapies such as Lyfgenia from bluebird bio and Casgevy from Vertex Pharmaceuticals, as well as other therapies in clinical development like BEAM-101 from Beam Therapeutics. This competitive landscape means there is no assurance that pociredir, even if approved, will achieve significant market share or sales compared to existing and emerging treatments.

• The emergence and advancement of competing RNA-based therapeutic approaches for Facioscapulohumeral Muscular Dystrophy (FSHD) from companies such as Avidity Biosciences (AOC 1004) and DYNE Therapeutics (DYNE-301). These therapies are progressing through clinical trials and represent a potentially more targeted or effective modality compared to Fulcrum's small molecule candidate, losmapimod.
• The approval and increasing adoption of gene therapies for Sickle Cell Disease and beta-thalassemia, notably Casgevy (Vertex Pharmaceuticals/CRISPR Therapeutics) and Zynteglo (bluebird bio). These therapies offer potentially curative treatments, establishing a high bar for efficacy and significantly altering the competitive landscape for any new therapeutic in these indications, including Fulcrum's early-stage small molecule candidate FULC-0401.

For Fulcrum Therapeutics (symbol: FULC), the addressable markets for their main products and services are as follows:

• Pociredir (for Sickle Cell Disease - SCD):
  • The global sickle cell disease treatment market size was valued at approximately USD 2.25 billion in 2022 and is projected to grow to USD 9.84 billion by 2030, exhibiting a Compound Annual Growth Rate (CAGR) of 20.1% during this period.
  • Another estimate indicates the global market size was USD 4.03 billion in 2024 and is expected to reach USD 13.06 billion by 2033, with a CAGR of 13.26% from 2025 to 2033.
  • The U.S. market for sickle cell disease treatment was estimated at around USD 700 million in 2022.
  • More recently, the U.S. sickle cell disease treatment market size accounted for USD 861.84 million in 2024 and is projected to reach approximately USD 3,789.17 million by 2034, growing at a CAGR of 15.96% from 2025 to 2034.
  • North America held the largest revenue share of 38.7% in the global sickle cell disease treatment market in 2024, with the United States accounting for about 89.00% of that North American share.
• Discovery Programs (including Diamond-Blackfan Anemia and Inherited Aplastic Anemias):
  • Diamond-Blackfan Anemia (DBA):
    • The global Diamond-Blackfan Anemia (DBA) syndrome therapeutics market was valued at approximately USD 4.85 billion in 2023 and is expected to reach USD 6.84 billion by 2032, with a CAGR of 4.4%.
    • Another report indicates the global DBA market was valued at USD 5,200 million in 2025 and is projected to reach USD 7,860 million by 2035, reflecting a CAGR of 4.2% over the forecast period.
    • North America dominates the global Diamond-Blackfan Anemia market. In 2023, the total market size for Diamond-Blackfan Anemia was USD 1.56 million across the seven major markets (7MM), with the United States representing about 70% of these cases.
  • Inherited Aplastic Anemia (part of the broader Aplastic Anemia market):
    • The global Aplastic Anemia market size is projected to be USD 7.12 billion in 2025 and is expected to reach USD 10.53 billion by 2033, growing at a CAGR of 5.00% from 2025 to 2033.
    • Another estimate places the global aplastic anemia market at USD 6.93 billion in 2024, anticipated to reach USD 12.75 billion by 2035, with a CAGR of approximately 5.7% between 2025 and 2035.
    • North America held the major market share, exceeding 37% of the global revenue for aplastic anemia in 2025, with a market size of USD 2,636.07 million.
    • Inherited aplastic anemia is expected to gain the highest market share by 2037 within the aplastic anemia treatment market.

1. Commercialization and Market Penetration of Pociredir for Sickle Cell Disease (SCD): The primary driver of future revenue growth is anticipated to be the successful clinical development, regulatory approval, and subsequent commercial launch of pociredir as a novel, once-daily oral therapy for sickle cell disease. Fulcrum is focused on demonstrating pociredir's potential as a "best-in-class" treatment by inducing fetal hemoglobin. Positive clinical data from the Phase 1b PIONEER trial, particularly from the 12 mg and 20 mg dose cohorts, is expected to be presented at the 67th American Society of Hematology (ASH) Annual Meeting in December 2025. Successful progression through these stages and eventual market adoption in key geographies, including the U.S. and potentially Nigeria where trials are ongoing, would be a significant revenue generator.
2. Advancement of the Bone Marrow Failure Syndromes Program: Fulcrum is expanding its pipeline with a program targeting various bone marrow failure syndromes, such as Diamond-Blackfan anemia, 5q deletion syndrome, Shwachman-Diamond syndrome, and Fanconi anemia. The company plans to submit an Investigational New Drug (IND) application for its calmodulin pathway modulator program in this area during the fourth quarter of 2025. Successful initiation of clinical trials and positive early-stage data for this program would open new market opportunities and represent a distinct future revenue stream, diversifying its product portfolio.
3. Positive Clinical Data and Pipeline De-risking for Pociredir: The continued generation of encouraging clinical data for pociredir is crucial. The upcoming data presentation at ASH in December 2025 for the PIONEER trial's 12 mg and 20 mg dose cohorts will be a key event. Positive and compelling results would further de-risk the asset, potentially accelerating its development pathway, enhancing its competitive profile, and increasing the company's valuation. This, in turn, could facilitate future funding, attract potential partnerships, and strengthen its position for subsequent larger clinical trials and eventual commercial success, indirectly driving future revenue.

Share Issuance

• Fulcrum Therapeutics had 54,118,438 shares outstanding as of October 22, 2025.
• The number of shares outstanding for FULC increased by 0.19% in one year.
• In November 2025, Fulcrum granted an aggregate of 270,000 non-statutory stock options and a 50,000 performance-based option to new hires, each with an exercise price of $9.02 per share.

Inbound Investments

• Fulcrum Therapeutics recognized $80.0 million in collaboration revenue in the first nine months of 2024 from a now-terminated agreement with Sanofi.

Outbound Investments

• Fulcrum Therapeutics reported net cash flow from investment acquisitions and disposals of approximately -$55.80 million in 2020, -$127.96 million in 2021, $14.38 million in 2022, -$36.18 million in 2023, and $32.51 million in 2024, indicating periods of both outbound investments (cash used) and divestitures (cash received).

Capital Expenditures

• Capital expenditures for Fulcrum Therapeutics were approximately $0.28 million in 2020, $0.51 million in 2021, $1.96 million in 2022, $1.71 million in 2023, and $1.34 million in 2024.
• In the trailing 12 months, capital expenditures were approximately -$386,000.