Sickle Cell Disease NewsFDA Lifts Its Clinical Hold on Oral Therapy FTX-6058 for SCD

Phase 1b study enrollment is anticipated to restart soon, with minor adjustments.

The FDA has cleared the way for Fulcrum Therapeutics to continue its Phase 1b clinical trial of FTX-6058, an oral treatment for sickle cell disease (SCD). The trial will now proceed with stricter eligibility criteria.

Key Points:

• The Phase 1b clinical trial (NCT05169580) will resume with modified, stricter eligibility criteria.
• FTX-6058 aims to increase fetal hemoglobin (HbF) levels, which are more effective at oxygen transport than adult hemoglobin.
• Early data showed dose-dependent increases in HbF levels.

Additional Points:

• The clinical hold was initially due to concerns about the potential risk of blood cancers.
• Eligible participants must have experienced at least four SCD pain crises in the past year or two crises in the past six months.
• The trial is limited to patients who have not responded to, were intolerant of, or ineligible for other approved SCD therapies like hydroxyurea, Oxbryta, and Adakveo.

Conclusion:

• The lifting of the FDA’s clinical hold allows for the resumption of a promising trial that could offer a new oral treatment option for SCD patients, albeit with stricter eligibility criteria.

“Based on the initial data from the Phase 1b trial, which showed increasing levels of HbF with each dose escalation, we believe in the potential of FTX-6058 to not only shift the current standard of care but importantly, offer these patients a differentiated oral option.”
– Alex C. Sapir, President and CEO of Fulcrum Therapeutics

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