RARE Daily

Biohaven Raises $225 Million in Public Offering

October 3, 2023

Rare Daily Staff

Biohaven tapped the public markets for $225 million to advance its pipeline of therapies to treat a broad range of rare and common diseases.

The company priced 10.2 million of its common shares at $22.00 per share. In addition, Biohaven has granted the underwriters a 30-day option to purchase up to an additional 1.5 million common shares at the public offering price, less underwriting discounts and commissions. The gross proceeds from the offering are expected to be approximately $225 million before deducting underwriting discounts and commissions and offering expenses payable by Biohaven.

In mid-September, Biohaven completed enrollment in RESILIENT, a phase 3 pivotal study of its lead investigational therapeutic taldefgrobep alfa in spinal muscular atrophy (SMA), which is designed to test the efficacy and safety of taldefgrobep alfa as adjunctive therapy to increase muscle in SMA patients treated with standard of care nusinersen, risdiplam, or the gene therapy Zolgensma.

SMA is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness that is often fatal and typically diagnosed in young children. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. Globally, SMA affects approximately 1 in 11,000 births, and about 1 in every 50 individuals is a genetic carrier.

Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living with SMA when used in combination with other approved treatments. Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.


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