Tetra Discovery and Shionogi Team Up to Further Study BPN14770 for Potential in Fragile X, Other Disorders

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by Mary Chapman |

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FMRP, fragile X, autism

A new collaboration between Tetra Discovery Partners and Shionogi & Co. is aimed at accelerating the development of an innovative treatment called BPN14770 for fragile X syndrome and other disorders in strategic Asian markets.

Valued at up to $160 million, the deal is expected to enable clinical-stage biotechnology firm Tetra to complete its ongoing Phase 2 clinical trial (NCT03569631) of BPN14770 in fragile X. It also provides funding for the start of a Phase 2 trial of the therapy in Alzheimer’s patients by early this year. Phase 1 was completed in April 2016.

BPN14770 is an investigational small molecule treatment that inhibits the phosphodiesterase type-4D (PDE4D) enzyme linked to memory formation and learning processes, as well as to neuroinflammation and traumatic brain injury.

Designed to enhance early and advanced stages of memory formation, the agent targets disorders characterized by cognitive and memory issues, including fragile X, Alzheimer’s, and related diseases, developmental disabilities, significant depression, and schizophrenia.

Preclinical animal models demonstrated that BPN14770 could foster the maturation of links between neurons — impaired in fragile X patients — and protect connections between neurons otherwise lost in Alzheimer’s patients.

Tetra’s Phase 2 trial, which is currently recruiting up to 30 men with fragile X, is designed to assess the safety and tolerability of BPN14770 and to evaluate its prospective effects on behavioral, cognitive, and biomarker measures. The therapy’s safety, tolerability, and pharmacokinetics — essentially how the body affects a medicine — were tested in two Phase 1 trials (NCT02840279 and NCT02648672).

The cross-over trial will be conducted in two 12-week treatment periods. Patients will be randomized in the first period to receive either 25 mg of BPN14770 or a placebo twice daily, and then will receive the opposite treatment during the second period. Behavioral and cognitive evaluations will be performed at weeks six and 12 of each treatment period.

Last year, the U.S. Food and Drug Administration accepted the investigational new drug application for BPN14770, and also granted it orphan drug status, a designation that advances and encourages drug development for rare diseases.

Tetra is also currently testing the effects of BPN14770 (10 mg and 50 mg) in reversing cognitive impairment induced by a therapy used to treat Alzheimer’s disease —  scopolamine — in healthy volunteers (NCT03030105).

“We are very pleased to join forces with Shionogi, a company that shares our interests in developing innovative medicines for patients with debilitating central nervous system (CNS) conditions,” Mark Gurney, Tetra chairman and CEO, said in a press release.

“Shionogi scientists contributed greatly to fundamental discoveries concerning the biochemical pathway modulated by BPN14770. The company’s in-depth knowledge of this brain pathway, and focus on CNS drug development, makes Shionogi a very compelling partner for Tetra.”

The collaboration calls for Tetra to grant pharmaceutical company Shionogi development rights to BPN14770  in Japan, Taiwan, and Korea.

“This collaboration, if successful, will enable us to move one step closer in realizing a more vigorous society in which patients can be relieved from debilitating central nervous system conditions,” said Isao Teshirogi, Shionogi’s president and CEO.