Regeneron Rare Disease Drug Shows Zero New Bone Growth In Extended Trial
Regeneron Pharmaceuticals, Inc. (REGN) reported long-term clinical trial data for Pasatru, its treatment for fibrodysplasia ossificans progressiva (FOP). In an extended Phase 3 trial involving 63 adult patients, those receiving Pasatru developed zero new abnormal bone lesions between weeks 56 and 84, compared with nine new lesions in the placebo group. FOP is an extremely rare genetic disorder in which muscles, tendons, and soft tissues gradually turn into bone.
The latest findings support the therapy's overall profile following its approval by the U.S. Food and Drug Administration in August. Pasatru works by blocking Activin A, a protein involved in abnormal bone growth. In addition to rare disease treatments, Regeneron maintains a broad drug pipeline across cancer, eye diseases, and inflammatory conditions, supported by about $17.8 billion in cash and marketable securities reported as of June 30.
European regulators are currently reviewing Pasatru for regulatory approval. Regeneron also plans to start another Phase 3 trial, Optima 2, to evaluate the drug in children and adolescents later this year.
Key points
- Regeneron Pharmaceuticals reported zero new abnormal bone lesions between weeks 56 and 84 in patients taking Pasatru during a Phase 3 extension trial.
- Patients in the placebo group developed nine new abnormal bone lesions during the same trial period among 63 adult participants.
- Pasatru received approval from the U.S. FDA in August and remains under regulatory review in Europe.
- Regeneron plans to initiate the Phase 3 Optima 2 trial involving children and adolescents later this year.
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What were the trial results for Regeneron's drug Pasatru?
In an extended Phase 3 trial, adult patients taking Pasatru developed zero new abnormal bone lesions between weeks 56 and 84, compared with nine new lesions in the placebo group.
What condition does Pasatru treat?
Pasatru treats fibrodysplasia ossificans progressiva (FOP), an extremely rare disorder where muscles, tendons, and other soft tissues gradually turn into bone.
What are Regeneron's upcoming plans for Pasatru?
Regeneron plans to start a new Phase 3 study, Optima 2, for children and adolescents later this year while European regulators review the drug for potential approval.