Biohaven to take a chance on Bristol Myers' once-failed DMD med

Biohaven is taking over Bristol Myer Squibb’s once-failed treatment for Duchenne muscular dystrophy through a worldwide licensing agreement, with plans to develop taldefgrobep alfa in spinal muscular atrophy. 

The two companies have signed an agreement on the licensing rights in exchange for an undisclosed amount of money to be paid to the Big Pharma, contingent on regulatory and sales-based milestones. 

In 2017, BMS licensed taldefgrobep alfa to Roche, which tested it on ambulatory boys with Duchenne muscular dystrophy. But ultimately, the trial was discontinued after a pre-clinical assessment found it was unlikely to have any clinical benefit. 

The drug is meant to limit myostatin, a natural protein that limits skeletal growth. Myostatin tends to be a good thing during muscular development but for patients with neuromuscular diseases, it can critically limit function. The question now is how, if at all, the drug can be used. 

Biohaven believes the answer is in patients with spinal muscular atrophy. The company plans to launch a phase 3 trial later this year to test the theory in patients with the progressive motor neuron disease that weakens muscle function and spurs muscular atrophy over time. 

RELATED: With a phase 3 fail, Biohaven learned the hard way why AstraZeneca dropped a CNS drug in 2018

While financial details about this deal are sparse, Biohaven said sales-based royalties for BMS would begin in the “high teens.” It’s the third time Biohaven has plucked one of BMS’ assets to expand its portfolio of possible therapies for neurologic and neuroinflammatory diseases. 

The agreement to pick up a failed drug from a Big Pharma is a familiar formula for Biohaven, which previously licensed a central nervous system drug from AstraZeneca to test in multiple system atrophy. The drug failed a phase 3 trial on the primary and secondary endpoints in the fall. 

The BMS tie-up wasn’t the Connecticut-based biotech's only move Friday. The company also acquired Knopp Biosciences subsidiary Channel Biosciences and its Kv7 channel targeting platform. The platform’s lead asset, KB-3061, is in preclinical studies for KCNQ2 encephalopathy. The condition arises when potassium channels in brain cells don’t function properly, which can cause the cells to over-generate electrical signals, resulting in seizures. Biohaven intends to bring the drug to clinic this year. 

In exchange for the acquisition, Knopp is receiving $100 million in upfront cash and stock options and could rake in more than $1 billion if the lead asset and larger platform hit regulatory and sales checkpoints.