GRAND RAPIDS — Drug developer Tetra Discovery Partners Inc. plans to launch a clinical trial later this spring on a potential treatment for an autism spectrum disorder.
The company received U.S. Food and Drug Administration clearance to conduct the Phase 2 trial in humans to test the safety and tolerability of a compound in 30 adult male patients with Fragile X Syndrome, the most common genetic form of autism that causes intellectual disability.
Tetra Discovery, which is based in Grand Rapids, will conduct the clinical trial over 12 to 18 months at Rush University Medical Center in Chicago with Dr. Elizabeth Berry-Kravis, the director of a research program and clinic that provides care for more than 600 patients with Fragile X.
The trial also will check other “exploratory objectives,” such as measuring behaviors, biomarkers and the cognitive abilities of patients “to try and discern whether or not there is any benefit to the drug,” said Tetra Discovery CEO Mark Gurney.
If “all goes well” with the Phase 2 clinical trial, Tetra Discovery will conduct a larger Phase 3 human clinical trial in 2019 that could lead to FDA approval of the drug compound, known as BPN14770, Gurney said.
“It’s a real milestone for our company that our drug has now progressed into mid-stage human trials for patients,” he said. “2018 is breakout year for Tetra. We have a very high-quality drug asset and we’re entering mid-stage human trials. Very quickly, we’ll be seeing whether or not it works, and that will drive us to the next step up in valuation.”
Tetra’s compound could treat other forms of autism as well, Gurney said. The company originally developed the compound to treat Alzheimer’s disease.
Data from a pre-clinical test in mice that Tetra Discovery conducted with the Fraxa Research Foundation in Newburyport, Mass. suggested the compound had the potential to treat memory and cognitive problems in Fragile X patients as well.
Fragile X affects about one in 4,000 to 5,000 males and one in 6,000 to 8,000 females, according to the federal Centers for Disease Control and Prevention (CDC). People with Fragile X are born with the condition.
The CDC classifies Fragile X as an orphan disease because it affects fewer than 200,000 people nationwide.
In reporting last fall on the pre-clinical trial, Fraxa Medical Director and Chief Scientific Officer Michael Tranfaglia called the results “very promising.”
Founded in 2011, Tetra Discovery Partners operates out of the business incubators at GR Current in Grand Rapids and at the Southwest Michigan Innovation Center in Kalamazoo. The company’s R&D has been funded by more than $22 million in grants and contracts with the National Institutes of Health, plus $7 million in private capital from investors that include Grand Angels, Muskegon Angels, Invest Michigan 2.0, the Biosciences Research & Commercialization Center in Kalamazoo, and the Johnson & Johnson Development Corp.
The company last month was named one of the “Michigan 50 Companies to Watch” for 2018.
Tetra Discovery is close to finalizing bridge financing of $1.5 million from existing shareholders to support the Phase 2 clinical trial for Fragile X, Gurney said.
The company must now decide how to proceed with financing a Phase 3 clinical trial for its compound and ultimately how to commercialize the drug, he said.
About 30 clinics in the U.S. see a majority of Fragile X patients, which would provide an easier path for Tetra Discovery to go to market with a drug on its own, Gurney said. However, the company also could pursue a strategic alliance with a partner or sell the intellectual property, he said.
“Those are decisions in the next year or two, and they affect the amount of money we want to raise and paths that we might pursue,” Gurney said.
Tetra Discovery also has completed a Phase 1 clinical trial for the compound that involved 147 Alzheimer’s patients and proved its safety and tolerability and showed cognitive benefits in healthy elderly people. The company wants to enroll 255 patients who are in the early stages of the disease for a Phase 2 trial at 35 sites in the U.S. The Phase 2 trial could cost an estimated $30 million, according to Gurney.