Tetra Inks Deal with Shionogi to Support Fragile X, Alzheimer’s R&D

The lead drug of Tetra Discovery Partners is already in mid-stage clinical studies as a treatment for the rare Fragile X Syndrome. But the Grand Rapids, MI, biotech says its drug could also treat a disorder affecting far more people: Alzheimer’s disease. The startup has reached across the Pacific Ocean to land the cash for an Alzheimer’s clinical trial.

Just before the holiday break, Tetra  announced a partnership with Japanese pharmaceutical company Shionogi for the clinical development and commercialization rights for of its drug BPN14770.

In exchange for rights to the drug in Japan, Taiwan, and Korea, Shionogi paid Tetra $40 million in combined upfront funding, which includes an equity investment and licensing payment. Tetra will be eligible to receive up to an additional $120 million if the drug hits development and commercialization milestones in Asia, as well as royalties on sales if BPN14770 is approved and reaches the market. Additional financial details of the agreement were not disclosed.

The Tetra drug selectively blocks phosphodiesterase‐4D (PDE4D), an enzyme involved in molecular mechanisms of memory formation.  Though Tetra’s own clinical research is furthest along in Fragile X Syndrome, an inherited disorder that affects learning and behavior, the company says its drug could potentially treat Alzheimer’s and other brain disorders characterized by cognitive and memory deficits. Designed to treat memory loss in earlier-stage patients, Tetra’s drug differs from amyloid drugs, which break up the buildup of amyloid plaque in the brain, by helping neurons make new connections.

“BPN14770 works on connections between neurons and the mechanism that allows synapses to mature and stabilize,” says Tetra CEO Mark Gurney. “Shionogi’s scientists have worked on this pathway, so they understood it. We met at BIO 2017.”

Gurney says Shionogi seemed like an especially fitting partner given its experience developing drugs, such as the Eli Lilly (NYSE: LLY) antidepressant duloxetine (Cymbalta), for central nervous system disorders. Tetra started out investigating BPN14770 to target early-stage Alzheimer’s but is now developing it for a range of cognitive disorders.

Tetra’s drug is in the midst of a Phase 2 study in 30 males with Fragile X Syndrome. The company is also preparing for a Phase 2 trial of the drug in patients with early Alzheimer’s, which it expects to start by the end of 2020. By taking a different approach to Alzheimer’s, Tetra hopes its drug can stand apart from the many that have fallen short in clinical testing.

“In the Alzheimer’s arena, we’ve seen large, visible clinical trial failures—the field has been focused on the amyloid pathway, but the results have been largely negative,” Gurney points out. “The mechanism for our drug is non-amyloid and relevant to cognitive resilience, which may delay the onset of diseases.”

Gurney mentions a study of Carmelite nuns that sought to find out why some of them showed resistance to the onset of Alzheimer’s even though their brains had the disease’s pathology. The nun study kicked off other epidemiological studies, he says, that found accompanying circumstances such as loneliness, lower education, and poor heart function were factors in the onset of Alzheimer’s, meaning it wasn’t simply a matter of plaque in the brain. Gurney says the data seemed to show that a healthy lifestyle, robust social network, and better heart function increased resistance to Alzheimer’s.

The capital influx from the partnership with Shionogi will allow Tetra’s 11 employees to support clinical development in both Fragile X and Alzheimer’s.  “This is a milestone for us,” Gurney says. “We’re one of the first therapeutic companies in West Michigan to have a [commercial] partner.”

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