Biohaven Plans Phase 3 Trial for Therapy After Buying Global Rights

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by Patricia Inacio, PhD |

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Biohaven Pharmaceuticals has entered into a worldwide licensing agreement to develop and commercialize Bristol Myers Squibb’s taldefgrobep alfa, a Phase 3-ready candidate therapy for spinal muscular atrophy (SMA).

The company is planning to launch a Phase 3 trial to test the therapy (also known as BMS-986089) in SMA patients this year.

“We are extremely excited about the potential for taldefgrobep to improve the lives of patients and families affected by neuromuscular diseases,” Vlad Coric, MD, CEO and chairman of the board of Biohaven, said in a press release.

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“We look forward to partnering with the entire SMA community in bringing this potential treatment — and further hope — to people living with SMA,” he added.

Adnectins are a proprietary protein therapeutic class based on human fibronectin, a protein found in abundance in human blood that functions in a similar way to antibodies. Specifically, adnectins bind their target with higher affinity and specificity than standard antibodies. Also, their stability and favorable half-life — the time required to reduce the amount of a compound by half and an indicator of how long its benefits last — make them attractive for therapeutic development.

Taldefgrobep alfa is a modified adnectin designed to specifically target and bind myostatin, a protein found naturally in the muscles that works to prevent overgrowth of skeletal muscles, those used for voluntary movement. However, in those with SMA, active myostatin can limit muscle growth necessary to achieve developmental and functional milestones.

In preclinical and human clinical studies, inhibition of myostatin led to a significant increase in muscle mass, supporting its therapeutic potential for diseases that involve muscle wasting, such as SMA.

“We believe the development of innovative anti-myostatin therapies designed to enhance muscle mass and strength may represent the next frontier of neuromuscular treatments and will build on the tremendous progress made by existing motor neuron-targeted therapies,” said Coric.

Biohaven’s licensing of taldefgrobep alfa, its third from Bristol Myers Squibb, expands its portfolio of late-stage candidates for treatment of neurologic, neuroinflammatory, and neuropsychiatric disorders.

“The addition of taldefgrobep to Biohaven’s portfolio of clinical-stage rare disease therapies aligns with our mission to make a meaningful difference in the lives of patients and families affected by devastating conditions with high unmet medical need,” said John Tilton, chief commercial officer at Biohaven.

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Under the terms of the agreement, Biohaven will hold the exclusive worldwide rights to taldefgrobep’s development. In turn, Bristol Myers Squibb will be eligible to receive milestone payments if the therapy is approved, as well as tiered, sales-based royalties.

“We are happy to see such great progress with the approvals and the impact of potential genetic treatments for SMA,” said Kenneth Hobby, president of Cure SMA, “but our work is not done yet and we need to now find innovative and efficacious therapies that will work in combination to help restore muscle strength and function, especially for older and symptomatic individuals affected by SMA.”