RARE Daily

Avenue Enters License Agreement with AnnJi to Develop and Treatment for Spinal and Bulbar Muscular Atrophy

March 3, 2023

Rare Daily Staff

Avenue Therapeutics a specialty pharmaceutical company focused on therapies for the treatment of rare and neurologic diseases, has entered into an exclusive license agreement with AnnJi Pharmaceutical, a Taiwanese clinical-stage drug company, for AJ201, a first-in-class clinical asset currently in a phase 1b/2a study in the United States for the treatment of spinal and bulbar muscular atrophy.

Spinal and bulbar muscular atrophy (SBMA), also known as Kennedy’s Disease, is a rare, inherited, X-linked genetic neuromuscular disease primarily affecting men. The condition is caused by a polyglutamine expansion in the androgen receptor (AR), which leads to production of an abnormal AR protein that forms aggregates responsible for muscle atrophy in the spinal-bulbar region of the body. The weakening of the bulbar muscles affects chewing, speech, and swallowing, with patients prone to choking or inhaling foods or liquids, resulting in airway infection. SBMA also affects muscles in the limbs, leading to difficulty walking and injury caused by falling. Although there is a range of cited prevalence rates in the literature, a recent study used genetic analysis to estimate disease prevalence of 1:6,887 males. Currently, there is no effective treatment for SBMA.

“The license for AJ201 brings a cutting-edge asset into Avenue’s pipeline that is the lead molecule in the clinic to treat Kennedy’s Disease, a debilitating rare neuromuscular disorder,” said Alexandra MacLean, CEO of Avenue. “With AJ201 leading the way, we are confident in the potential of our diversified portfolio of three assets to deliver value for investors in the near term and patients in the longer term.”

AJ201 was designed to modify SBMA through multiple mechanisms including degradation of the abnormal AR protein and by stimulating Nrf1 and Nrf2, which are involved in protecting cells from oxidative stress which can lead to cell death. AJ201 completed a phase 1 clinical trial in 2021, which demonstrated the safety of the molecule. It is currently being studied in a phase 1b/2a multicenter, randomized, double-blind clinical trial in six clinical sites across the United States, and screening of patients with SBMA has begun. This study aims to evaluate the safety and clinical response of AJ201 in patients suffering from SBMA. AJ201 has been granted Orphan Drug designation by the U.S. Food and Drug Administration for the indications of SBMA, Huntington’s disease and spinocerebellar ataxia.

Under the terms of the license agreement, AnnJi will receive upfront payments of $3 million and is entitled to receive future development, regulatory and commercialization milestone payments, as well as royalties on net sales of the licensed product. Avenue will also issue 831,618 shares of its common stock to AnnJi in connection with the initial closing of the license transaction and up to an additional 276,652 shares upon achievement of a clinical milestone, aggregating in total to not more than 19.99 percent of the company’s current total number of outstanding shares of common stock. The agreement includes the United States, Canada, European Union, Great Britain, and Israel as exclusively licensed territories.

Photo: Alexandra MacLean, CEO of Avenue

Stay Connected

Sign up for updates straight to your inbox.