After an FDA rejection of its spinocerebellar ataxia (SCA) therapy, Biohaven was forced to rethink its strategy—cutting R&D spend by 60% and channeling the spirit of a scrappy startup again as it zeros in on obesity.
The regulatory setback, delivered in November, has left the fate of investigational SCA treatment troriluzole tied up in an FDA appeal process. With no “near-term revenue opportunity” from the ataxia program, the company has pivoted strategy, said CEO Vlad Coric, M.D.
“You have to focus on your closest near-term value inflection points,” Coric told Fierce last week.
For Biohaven, that means prioritizing assets stemming from three late-stage platforms: myostatin-activin inhibitors, extracellular protein degraders and Kv7 ion channel activators.
“You focus your resources on three near-term inflection points,” the CEO explained. “If you hit any one of those three, value will go up and then you [can] follow the science in some of the other areas that we’re excited about and have assets in.”
The company’s cost-cutting measures have largely targeted late-stage programs, Coric explained. The CEO cited Biohaven’s decision to pare down a phase 2/3 generalized epilepsy study of Kv7 drug BHV-7000 but keep money flowing to an adult focal onset epilepsy study of the same asset as an example. Pivotal results for the latter are expected to read out this year.
“We made the decision to decrease phase 3 studies this year,” Coric explained, adding that this included “putting off starting new studies or ending studies that wouldn’t get us to the near-term value inflection and approvals.”
When asked whether a workforce reduction was paired with the cost cutting, Coric said “very little” of Biohaven’s staff was impacted, reiterating that large clinical trials represent the company’s biggest expense.
Addressing what prompted Biohaven to move beyond its longstanding focus on neuroscience, Coric—who helped form the company more than a decade ago—said his duty as a drug developer is to follow the science.
“We’re supposed to solve problems for patients and follow to whatever therapeutic area is a good opportunity to do that in the near return,” he added.
Selling off Nurtec to Pfizer for $11.6 billion in 2022 made that possible for Biohaven, giving Coric’s team the credibility to go beyond neuroscience.
Since the sale, Coric said, the company has returned to a startup mindset. “You have to keep your feet held to the fire,” he said. “You have to find success.”
In pursuit of such success, Biohaven has entered the hotly competitive obesity scene with lead program taldefgrobep alfa.
Biohaven picked up the candidate (also known as BMS-986089 and BHV-2000) from Bristol Myers Squibb in 2022. The Big Pharma had studied the candidate in Duchenne muscular dystrophy but ultimately discontinued the trial after an assessment found the drug unlikely to have any clinical benefit.
Now, Biohaven is testing out the myostatin-targeting recombinant protein in a phase 2 obesity trial, with dosing initiated at the end of last year and top-line results expected in mid-2026, Coric said.
“[With] what we’re learning about myostatin-activated obesity, I think we can solve a problem that the current GLPs aren’t addressing, and that is the muscle loss that occurs with GLPs and the bone density loss,” he said.
The Biohaven CEO believes the company'scandidate, which is designed to block myostatin activity, fits the bill for both fat loss and increased muscle and bone density. Myostatin is a naturally occurring protein that limits skeletal muscle growth.
While obesity is the focus, Biohaven’s neurology roots haven’t been left completely by the wayside. The company is also assessing taldefgrobep alfa in spinal muscular atrophy, a rare neurodegenerative disease.
Though a late-stage trial didn’t outperform a combination of placebo and standard-of-care treatment, Biohaven has highlighted clinically meaningful improvements in motor function and is in talks with the FDA about a potential path forward. The candidate has secured both fast track and orphan drug tags in the indication.
Meanwhile, Biohaven is looking for that near-term value inflection in two pivotal trial launches related to its degrader platform, one in the kidney disease IgA nephropathy and another in an autoimmune disorder known as Graves' disease.
“No one else has an extracellular degrader that’s in the clinic right now—small, little Biohaven does,” Coric said.
The platform, dubbed MoDE, is sourced from science stemming from the lab of Yale University’s David Spiegel, M.D., Ph.D. Biohaven has used the platform to create a class of small molecule degraders that bind to disease-causing proteins.
Coric believes the company is “on the verge of a huge paradigm shift” with the science.
“So, we have to pursue that, even though it’s not neuroscience, right?” he asked rhetorically.
And then there’s the epilepsy asset BHV-7000, designed to selectively activate Kv7.2/7.3 potassium channels.
Earlier this year, the therapy failed to alleviate symptoms of major depressive disorder when compared to placebo.
“We don’t see a signal in depression with this asset, so we’re not continuing it,” he explained. “We know what successful programs look like. We also know when there’s not a drug signal and when to stop a program.”
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