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1. Company Snapshot

1.a. Company Description

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States.Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy; and FTX-6058, an investigational oral fetal hemoglobin inducer for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia.The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases.


Fulcrum Therapeutics, Inc.has research and discovery collaboration agreement with Acceleron Pharma Inc.to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and has a strategic collaboration and license agreement with MyoKardia, Inc.


to discover, develop, and commercialize novel targeted therapies for the treatment of genetic cardiomyopathies.Fulcrum Therapeutics, Inc.was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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1.b. Last Insights on FULC

Fulcrum Therapeutics' recent performance was negatively impacted by the lack of significant clinical data updates. The company's announcement that it is 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, while positive, did not materialize during the recent quarter. Additionally, the company's recent inducement grants to new employees, while a positive sign of growth, may not have had a significant impact on the company's short-term performance.

1.c. Company Highlights

2. Fulcrum Therapeutics' Q3 2025 Earnings: Progress on Pociredir for Sickle Cell Disease

The company's financial performance for the third quarter of 2025 reflected a net loss of $19.6 million, compared to a net loss of $21.7 million in the same period last year. Research and development expenses were $14.3 million, a decrease of $0.3 million compared to the third quarter of 2024. The actual EPS came out at -$0.31, slightly worse than estimates of -$0.29. The company's cash, cash equivalents, and marketable securities stood at $200.6 million, a decrease of $40.4 million compared to December 31, 2024.

Publication Date: Nov -19

📋 Highlights
  • Pociredir Phase Ib Progress:: 12mg dose showed clinically meaningful fetal hemoglobin increase, hemolysis biomarker improvements, and reduced VOCs; 20mg cohort enrolled 12 patients (60% U.S.).
  • Financial Performance:: Q3 R&D expenses $14.3M (-$0.3M YoY), net loss $19.6M (-$2.1M YoY); cash reserves $200.6M, projected runway to 2028.
  • Market Opportunity:: 20% of 100,000 U.S. sickle cell patients meet PIONEER criteria; 7.7M global patients addressed post-Oxbryta withdrawal.
  • Regulatory Strategy:: FDA End-of-Phase I meeting planned for Q1 2026; 20mg data to inform Phase III design, potentially a registrational trial with VOC reduction as primary endpoint.
  • Open-Label Extension (OLE) Study:: Initiated to address patient/investigator demand for continued treatment post-12-week PIONEER dosing; full 20mg data expected Q1 2026.

Cash Runway and Funding

Fulcrum Therapeutics expects its existing cash to be sufficient to fund its operating requirements into 2028. This cash runway guidance assumes a full success for the organic program, including the Phase III program for pociredir. The company's cash position is expected to support its plans to move forward with pociredir to the next trial and continue progression of work in the preclinical phase.

Pociredir Progress and Future Plans

The company's lead program, pociredir, demonstrated significant progress in the treatment of sickle cell disease. The 12-milligram dose cohort of the Phase Ib PIONEER trial showed a dose-dependent and clinically meaningful increase in fetal hemoglobin, improvement in key biomarkers of hemolysis, and a reduction in vaso-occlusive crises. The company has completed enrollment in the 20-milligram dose cohort and expects to present data from this cohort at the American Society of Hematology conference in early December. As Alexander Sapir, CEO and President, mentioned, "Our overarching goal is to get this drug to the market as quickly as possible to help as many patients as we can, particularly with the withdrawal of Oxbryta and the high costs and risks associated with cell and gene therapy."

Valuation Metrics

Looking at the valuation metrics, the company's P/E Ratio stands at -8.56, and the P/B Ratio is 3.07. The EV/EBITDA ratio is -6.46, indicating that the market is pricing in significant future growth. The ROE (%) is -32.17, reflecting the company's current loss-making position. These metrics suggest that the market is expecting a turnaround in the company's fortunes, likely driven by the progress on pociredir.

3. NewsRoom

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Fulcrum Therapeutics to Host Investor Event to Review New Clinical Data from the PIONEER trial of Pociredir in Sickle Cell Disease on December 7, 2025

Dec -02

Source: globenewswire.com

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Acadian Asset Management LLC Grows Stake in Fulcrum Therapeutics, Inc. $FULC

Nov -14

Source: defenseworld.net

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Fulcrum Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Nov -07

Source: globenewswire.com

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Fulcrum Therapeutics to Present New Clinical Data from the PIONEER trial of Pociredir in Sickle Cell Disease at the 67th American Society of Hematology Annual Meeting

Nov -03

Source: globenewswire.com

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Fulcrum Therapeutics, Inc. (FULC) Q3 2025 Earnings Call Transcript

Oct -29

Source: seekingalpha.com

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Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Third Quarter 2025

Oct -29

Source: globenewswire.com

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Fulcrum Therapeutics to Host Third Quarter 2025 Financial Results Conference Call and Webcast on Wednesday, October 29, 2025, at 8:00 a.m. ET

Oct -22

Source: globenewswire.com

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Fulcrum Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Oct -10

Source: globenewswire.com

4. Business Breakdown

4.a. Revenues by Country

4.b. Revenues by Segment

5. Expected revenues mid-term growth (10.43%)

6. Segments

Biopharmaceutical

Expected Growth: 10.43%

Fulcrum Therapeutics' 10.43% growth in biopharmaceuticals is driven by increasing demand for rare genetic disorder treatments, strategic partnerships, and a strong pipeline of novel therapies. The company's focus on gene regulation and epigenetics is also contributing to its growth, as well as its ability to leverage AI and machine learning in drug discovery.

7. Detailed Products

Fulcrum's FTX-6058

FTX-6058 is a small molecule designed to increase the expression of FUS, a protein that is deficient in individuals with FSHD.

Fulcrum's Losmapimod

Losmapimod is a p38α/β inhibitor that has been shown to reduce inflammation and improve muscle function in patients with FSHD.

Fulcrum's FTX-020

FTX-020 is a small molecule designed to increase the expression of a key protein involved in the regulation of muscle function.

Fulcrum's Gene Therapy Programs

Fulcrum's gene therapy programs are designed to treat genetic disorders by delivering a healthy copy of a gene to cells.

8. Fulcrum Therapeutics, Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

Fulcrum Therapeutics, Inc. operates in a niche market with limited substitutes, but the threat of substitutes is still present due to the ongoing research and development in the biotechnology industry.

Bargaining Power Of Customers

Fulcrum Therapeutics, Inc. has a diverse customer base, which reduces the bargaining power of individual customers. Additionally, the company's products are specialized, making it difficult for customers to switch to alternative suppliers.

Bargaining Power Of Suppliers

Fulcrum Therapeutics, Inc. relies on a few key suppliers for raw materials and services. While the company has some bargaining power due to its size, the suppliers also have some leverage due to the specialized nature of their products.

Threat Of New Entrants

The biotechnology industry has high barriers to entry, including significant capital requirements, complex regulatory requirements, and the need for specialized expertise. These barriers make it difficult for new entrants to enter the market.

Intensity Of Rivalry

The biotechnology industry is highly competitive, with many established players and new entrants vying for market share. Fulcrum Therapeutics, Inc. faces intense competition from companies with similar products and technologies.

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

Value
Debt Weight 4.40%
Debt Cost 3.95%
Equity Weight 95.60%
Equity Cost 15.68%
WACC 15.16%
Leverage 4.60%

11. Quality Control: Fulcrum Therapeutics, Inc. passed 3 out of 9 key points

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

Stock-Card
Rigel Pharma

A-Score: 5.7/10

Value: 6.1

Growth: 8.0

Quality: 9.1

Yield: 0.0

Momentum: 9.5

Volatility: 1.7

1-Year Total Return ->

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Stock-Card
Fulcrum Therapeutics

A-Score: 5.4/10

Value: 6.8

Growth: 8.0

Quality: 6.4

Yield: 0.0

Momentum: 9.5

Volatility: 1.7

1-Year Total Return ->

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Assembly Biosciences

A-Score: 4.3/10

Value: 6.2

Growth: 3.8

Quality: 4.6

Yield: 0.0

Momentum: 9.0

Volatility: 2.0

1-Year Total Return ->

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EyePoint Pharmaceuticals

A-Score: 3.7/10

Value: 6.6

Growth: 2.3

Quality: 4.9

Yield: 0.0

Momentum: 7.5

Volatility: 1.0

1-Year Total Return ->

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Lipocine

A-Score: 3.6/10

Value: 8.2

Growth: 6.9

Quality: 3.0

Yield: 0.0

Momentum: 1.0

Volatility: 2.7

1-Year Total Return ->

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Clearside Biomedical

A-Score: 3.3/10

Value: 8.4

Growth: 3.6

Quality: 6.1

Yield: 0.0

Momentum: 0.5

Volatility: 1.0

1-Year Total Return ->

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Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

8.9$

Current Price

8.9$

Potential

-0.00%

Expected Cash-Flows