Fulcrum Reports Positive 20 mg Dose Trial Results with 9.9% HbF Increase
Written by Emily J. Thompson, Senior Investment Analyst
Source: Globenewswire
Updated: 59 minutes ago0mins
- Significant Dose Response: In the 20 mg cohort, fetal hemoglobin (HbF) levels increased by an average of 9.9% at six weeks, compared to 5.6% in the 12 mg cohort, indicating a stronger therapeutic potential that could provide more effective treatment options for severe sickle cell disease patients.
- Positive Patient Response: 58% of patients in the 20 mg cohort achieved HbF levels ≥20%, which is associated with a reduction in annual vaso-occlusive crises (VOCs), highlighting the drug's significant impact on improving patient quality of life.
- Good Safety Profile: As of the November 11, 2025 data cutoff, pociredir has been administered to 148 adults with no reported treatment-related serious adverse events, demonstrating favorable tolerability and enhancing its credibility as a potential treatment option.
- Positive Future Outlook: Fulcrum plans to update trial results in Q1 2026, further validating the efficacy and safety of pociredir, which may drive its application in the sickle cell disease market.
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Analyst Views on FULC
Wall Street analysts forecast FULC stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for FULC is 14.50 USD with a low forecast of 6.00 USD and a high forecast of 20.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
9 Analyst Rating
7 Buy
1 Hold
1 Sell
Moderate Buy
Current: 8.760
Low
6.00
Averages
14.50
High
20.00
Current: 8.760
Low
6.00
Averages
14.50
High
20.00
Truist
initiated
$14
2025-11-24
Reason
Truist
Price Target
$14
2025-11-24
initiated
Reason
Truist initiated coverage of Fulcrum Therapeutics with a Buy rating and $14 price target. Fulcrum Therapeutics has shown early signs that pociredir could be competitive in the SCD therapeutic landscape, the analyst tells investors in a research note. Encouraging data from the phase Ib PIONEER study for pociredir shows notable induction of HbF, most recently in patients dosed with 12 mg of drug, which in July showed robust and clinically meaningful absolute mean fetal hemoglobin HbF induction of 8.6% from baseline at 12 weeks, and safety continues to remain in-line with previous dosing cohorts and healthy volunteer data, the firm says.
Oppenheimer
Matthew Biegler
Outperform
maintain
2025-11-19
Reason
Oppenheimer
Matthew Biegler
Price Target
2025-11-19
maintain
Outperform
Reason
Oppenheimer analyst Matthew Biegler notes that Fulcrum Therapeutics' (FULC) shares are rallying this morning in the wake of Agios' (AGIO) failed RISE UP trial. Investors are likely viewing the outcome as removing a potential competitor to Fulcrum in sickle cell disease. Oppenheimer is not entirely surprised by the result. RISE UP ended up resembling HOPE: Marginal improvements in hemoglobin that trended but didn't reach statistical significance on vaso-occlusive crises. The firm continues to favor Fulcrum's approach, which induces fetal hemoglobin rather than indirectly modifying hemoglobin. There's simply more evidence translating HbF levels to clinical benefit in SCD. Fulcrum will present additional data from PIONEER at ASH, and Oppenheimer thinks that could cement its position as the go-to oral option of the future. The firm reiterates an Outperform rating on Fulcrum.
Stifel
analyst
initiated
$20
2025-11-06
Reason
Stifel
analyst
Price Target
$20
2025-11-06
initiated
Reason
Stifel resumed coverage of Fulcrum Therapeutics with a Buy rating and $20 price target. Recent 12mg data already support a "compelling profile" for pociredir and the "bar" for the full 20mg cohort is low, argues the analyst, who adds that there's "a blockbuster opportunity that supports meaningful upside" even as Fulcrum's trial population and criteria exists today.
Piper Sandler
Overweight
maintain
$16
2025-10-30
Reason
Piper Sandler
Price Target
$16
2025-10-30
maintain
Overweight
Reason
Piper Sandler raised the firm's price target on Fulcrum Therapeutics to $16 from $12.50 and keeps an Overweight rating on the shares. The firm notes Fulcrum will report 20mg pociredir data at ASH in December. Fulcrum disclosed that baseline HbF in the 12 patients enrolled was 7.1% mean. Piper is confident that 20mg pociredir will outperform the 12mg cohort across performance measures.
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About FULC
Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. The Company's lead clinical program is pociredir, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease (SCD). It uses technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. In addition to its product candidates, the Company has developed a discovery approach that it employs to systematically identify and validate cellular drug targets that can potentially modulate gene expression to treat known root causes of genetically defined rare diseases. The Company continues to advance its program for the potential treatment of inherited aplastic anemias, such as Diamond-Blackfan anemia (DBA), Shwachman-Diamond syndrome, and Fanconi anemia.
About the author
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Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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