An $80M Stamp of Approval for a Promising Yale-Based Drug Platform

Discovering the many potential therapeutic uses for glutamate-modulating agents such as riluzole, says John Krystal, Chair of the Department of Psychiatry at Yale and Chief of Psychiatry at Yale-New Haven Hospital, was a bit like pulling a thread. “You keep pulling it, and the thread keeps coming,” he says. Krystal and colleagues including Gerard Sanacora, professor of psychiatry and director of the Yale Depression Research Program, and Vladimir Coric, former director of the Yale OCD Research Clinic, had long researched the benefits of drugs that modulate glutamate—a neurotransmitter in the brain—to treat depression and anxiety. It was a new approach that showed particular promise with patients who did not respond to traditional treatments. Now Biohaven Pharmaceuticals—the New Haven-based startup where Krystal serves as Scientific Advisor— has just secured an $80M funding round to expand the therapeutic possibilities of glutamate modulation across therapeutic indications.

The company is exploring how their patented glutamate modulators might treat rare, debilitating diseases such as spinocerebellar ataxia (SCA)—where patients progressively lose control of their movements and hand-eye coordination while their minds remain intact. Other diseases in Biohaven’s sights: amyotrophic lateral sclerosis (ALS), migraines and cancer.  

“This intellectual property estate started with our neuroscience research at Yale’s labs and has now evolved into much more,” says CEO Coric, an Associate Clinical Professor of Psychiatry at the Yale School of Medicine who discovered the efficacy of riluzole for treating neuropsychiatric disorders with Krystal over a decade ago and whose industry experience includes developing drugs like Abilify and Opdivo. “As a company, our strategy has been to follow the science of glutamate across diseases and evaluate the effectiveness of our glutamate modulators.”

The size of the round, led by Venrock and other investors like RA Capital, Osage University Partners and Connecticut Innovations, is a testament not only to the promise of Biohaven’s glutamate platform but to the highly skilled team that Coric has pulled together. That management team includes Chief Medical Officer Robert Berman, an adjunct professor at Yale who led multiple programs at Pfizer and was Group Director at Bristol-Myer Squibb (BMS), and Chief Financial Officer Jim Engelhart who served as Executive Director in Finance of the North America and Latin America Regions at Alexion for over 17 years. Behind them is a clinical team with experience at Pfizer and BMS. “We’re excited to create value for the patients, the investors and the New Haven community,” Coric says. “We want to help build a biotech corridor here in New Haven.”

The investment—$30 million more than they were initially seeking—will allow Biohaven to speed up three clinical trials—leading with SCA, which currently has no treatment. The company’s novel glutamate modulator has meant improvements compared to older drugs in this class—better safety and tolerability—and focusing on an orphan illness means they can get to market quicker.  

“With SCA there’s a huge unmet need,” says Coric, “and the study is reasonably sized—around 120 patients. One positive pivotal trial would typically be sufficient to get approval in orphan indications.” Outside the orphan disease space, drug approval requires two clinical trials which, in the case of depression, for instance, can cost $30-$40M each. With an approved drug on the market, they can use it as a platform to explore other indications, developing a pipeline of treatments.

“Innovative mechanisms of action and improvements on older drugs has been for Yale a great entry point for addressing unmet medical needs,” says David Lewin, Senior Associate Director of Licensing at the Office of Cooperative Research which helped to guide the commercialization process for the initial discovery. “Applying clinical experience from known drugs into new indications allows you to build around an innovative concept for a foundational product with well-understood characteristics and clinical history; then you grow from that kernel.”

Krystal adds, “This is a great example of how you translate pure academic research into a company that makes an impact for patients.”

CONTACT: Brita Belli, Communications Officer, Yale Innovation & Entrepreneurship, (203)804-1911, brita.belli@yale.edu.