After the FDA’s tough stance on the modality following the recent withdrawal of Ipsen’s Tazverik from the U.S. market, Fulcrum Therapeutics has dropped its only clinical-stage program.
In March, Ipsen pulled Tazverik from all indications and markets, citing a data monitoring committee finding of “adverse events of secondary hematologic malignancies” during a phase 1b/3 trial.
The drug comes from France’s Ipsen, which purchased Epizyme in 2022, when it acquired the rights to Tazverik, which is an inhibitor of PRC2. Tazverik had been available in the U.S. for two years by then as treatment for both epithelioid sarcoma and follicular lymphoma.
Fulcrum’s lead asset, pociredir, is also a PRC2 inhibitor, which the company had been looking at in a phase 1b trial in people with sickle cell disease (SCD). The biotech said it was in talks with the FDA to “provide regulatory perspective that there are ‘mechanistic differences’ between the target of the regulatory protein EED and the EZH2 enzyme being targeted by Tazverik.
These targets “perform different biological roles,” which is “relevant to the benefit-risk assessment,” Fulcrum argued in a June 1 post-market release. Sadly, the FDA was not satisfied with Fulcrum’s claims.
Fulcrum explained that the FDA thought about this but found “all pharmacological agents that target the PRC2 complex present a similar degree of malignancy risk to each other, regardless of which subunit is targeted. Its stance is “based on the agency’s previous preclinical observations of malignancy, and with no pathway forward to proceed with clinical development of pociredir,” said the biotech.
The announcement comes as a blow to Fulcrum, which only recently, in late April, reaffirmed its “conviction in pociredir’s potential to address the underlying biology” of SCD, and noted a “strong balance sheet” of $333 million to cover the drug’s development through 2029.
Rather, Fulcrum has had no other way to go but to write off its plans to launch pociredir in SCD and “take strategic steps.” The alternate options may be a sale or a company merger of the asset or company. In the preclinical pipeline, Fulcrum has HbF inducers for SCD and prostate cancer.
Meanwhile, the biotech has already started “serious measures to substantially lower its operating costs and conserve capital.” Fierce has contacted the company to inquire if this includes layoffs.
“After careful consideration of regulatory feedback, the entirety of available data, and implications for a viable regulatory path, we’ve made the tough decision to end development of pociredir,” CEO Alex Sapir said in the release.
“Although there are no new safety signals as yet with pociredir, the FDA was concerned about the potential malignancy risk of the PRC2 complex inhibitor activity of pociredir due to the experience with the recently withdrawn Tazverik,” Sapir added. We made this determination after talking with the FDA, and although there are strong increases in fetal hemoglobin with pociredir and a potential clinical benefit, we don’t see us going forward with pociredir.”
Fulcrum has announced the discontinuation of its sickle cell disease (SCD) program following a firm regulatory stance from the U.S. Food and Drug Administration regarding potential safety hazards. The decision marks a significant shift for Fulcrum, which is now exploring alternative strategic opportunities and reassessing its development priorities.
The move highlights the challenges biotechnology companies face when balancing innovation with stringent regulatory requirements designed to protect patient safety.
FDA Safety Concerns Influence Fulcrum’s Decision
The decision by Fulcrum follows discussions with the FDA regarding safety-related issues associated with the SCD program. Regulatory agencies play a critical role in evaluating both the efficacy and safety of investigational therapies, and the concerns raised ultimately influenced the direction taken by Fulcrum.
By discontinuing the program, Fulcrum aims to focus resources on initiatives that may offer a clearer regulatory pathway and stronger long-term potential.
The decision by Fulcrum to discontinue its sickle cell disease program reflects the impact of FDA safety concerns on drug development strategies. While the move closes one chapter for Fulcrum, it also opens the door to new opportunities as the company evaluates alternative paths for growth and innovation. Stakeholders will be watching closely to see how Fulcrum reshapes its future direction in the evolving biotechnology landscape.
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