Sales of Biohaven Pharmaceuticals’ lead migraine medication continue to grow, and the drug developer is now putting some of that cash to work in a pair of deals that expand its scope in neuroscience. The first is the acquisition of Channel Bio, a company whose lead asset is a potential treatment for epilepsy. Separately, Biohaven licensed a Bristol Myers Squibb drug that’s ready for a pivotal test in spinal muscular atrophy.
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New Haven, Connecticut-based Biohaven announced Friday it is acquiring Channel Bio for $100 million in cash and stock. Specific financial terms of the BMS deal were not disclosed.
Channel Bio develops drugs that target the Kv7 ion channel, which is associated with epilepsy and pain. This channel has already been validated by other drugs. Speaking on a conference call, Channel Bio CEO Michael Bozik said that his company’s small molecule offers the potential to best in this class of medications by reducing the sleepiness, fatigue, and vision problems associated with the seizure medications that target Kv7.
The lead target is focal epilepsy, the most common form of the disorder in which the misfiring electrical activity affects a small area of the brain. The biotech’s lead therapeutic candidate, now named BHV-7000, is a small molecule designed to activate parts of the Kv7 pathway involved in neuronal signaling and the regulation of the hyperexcitability characteristic of epilepsy.
Bozik said that his company’s drug is designed to be structurally distinct from two other drugs in its class, ezogabine, a medicine that was marketed by GlaxoSmithKline and Valeant Pharmaceuticals (now Bausch Health Companies) until they discontinued the product in 2017, and XEN1101 a drug that Xenon Pharmaceuticals has advanced to mid-stage testing for focal epilepsy. Channel Bio’s platform has yielded a library of small molecules that could extend the targeting of Kv7 to other indications, Bozik said. A clinical trial testing the lead asset in focal epilepsy is expected to begin later this year.
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Channel Bio is a subsidiary of Pittsburgh-based Knopp Biosciences. The upfront payment to Knopp breaks down to $65 million in Biohaven shares and $35 million in cash. Knopp could earn up to $325 million more tied to the development and regulatory progress of BHV-7000 through approvals in the U.S., Europe, and Japan. Up to $250 million is tied to the development and approvals of Kv7-targeting drugs in other indications. Sales based milestones could bring another $562.5 million, plus royalties.
By licensing BMS drug taldefgrobep alfa, Biohaven is making a bet it can resurrect a program that fell short in a rare muscle disorder. The drug is a fusion protein that targets and blocks myostatin, a protein that inhibits muscle growth. By blocking this protein, scientists hoped the drug would improve muscle mass.
In 2019, Roche discontinued work on the drug in Duchenne muscular dystrophy after an interim analysis of clinical data indicated the therapy was unlikely to help patients. The Swiss pharma giant returned rights to the drug to BMS.
Biohaven now plans to see if taldefgrobep’s ability to block myostatin can help patients with spinal muscular atrophy (SMA), a different inherited disorder that also leads to progressive muscle weakening. Biohaven CEO Vlad Coric said that even though the drug fell short in Duchenne, the earlier clinical research provides plenty of safety data for the drug. He added that clinical data from biotech company Scholar Rock indicate that blocking myostatin can treat SMA.
“That’s really the clinical proof of concept that is advancing our interest in this area,” Coric said on the call. “We also note there’s other competitors. Roche, who have a myostatin targeting agent as well. So, Roche and Scholar Rock are both heading into SMA given the recent clinical [proof of concept] there. We believe this has the potential to be a best-in-class myostatin targeting agent.”
Biohaven licensed global rights to taldefgrobep. BMS is eligible for milestone payments tied to regulatory approval, plus royalties from sales. A Phase 3 clinical trial is expected to begin later this year. That study will test the drug alongside standard of care SMA drugs.
The Biohaven acquisitions were announced concurrent with the Friday release of fourth quarter and full year 2021 financial results. Biohaven reported that its migraine drug, Nurtec, accounted for $462.5 million in revenue for the year, compared to $63.6 million in 2020 revenue. The drug, which was licensed from BMS, is poised for global growth as well. Last November, Pfizer paid $500 million in a cash and stock deal for rights to the Biohaven migraine drug outside of the U.S.
Image: Jolygon, via Getty Images
