RARE Daily

Advanz Pharma Licenses Applied’s Experimental Therapy for Rare Neurological Disorders

January 5, 2023

London-based Advanz Pharma said it has entered into an agreement to license Applied Therapeutics’ experimental therapy AT-007, which is in development for rare, neurological disorders.

AT-007 is a central nervous system penetrant aldose reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including galactosemia, SORD deficiency, and PMM2-CDG.

In clinical trials, AT-007 significantly reduced plasma galactitol levels vs. placebo in adults and children with galactosemia. AT-007 is currently being studied in a phase 3 clinical outcomes trial (ACTION-Galactosemia Kids) in children ages 2-17 with galactosemia, as well as a long-term open-label study in adults with galactosemia. In a pilot study in adults with SORD feficiency, AT-007 significantly reduced blood sorbitol levels.

AT-007 is currently being studied in a phase 3 trial (INSPIRE) investigating biomarker efficacy and clinical outcomes in adults and children 16 years and older with SORD feficiency. The drug has been generally safe and well tolerated in all clinical studies to date.

Under the terms of the agreement, Applied Therapeutics will be responsible for the clinical development and supply of AT-007, while ADVANZ will manage the registration and commercialization in the European Economic Area, the United Kingdom, and Switzerland.

The companies did not disclose financial terms of the agreement.

“We believe Advanz Pharma is an ideal partner for commercialization of AT-007 in Europe,” said Shoshana Shendelman, founder and CEO of Applied Therapeutics. “This partnership brings us one step closer to making AT-007 available to patients with galactosemia and SORD feficiency – both debilitating rare diseases with no other therapies currently available.”

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.