Avenue Therapeutics Enters into a Transformational License Agreement with AnnJi Pharmaceutical to Develop and Commercialize AJ201, a First-in-Class Clinical Asset for the Treatment of Spinal and Bulbar Muscular Atrophy

AJ201 is being evaluated in a Phase 1b/2a clinical trial in the U.S. for the rare X-linked genetic neurodegenerative disease also known as Kennedy’s Disease which currently has no FDA approved therapy

MIAMI, March 02, 2023 (GLOBE NEWSWIRE) — Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of rare and neurologic diseases, today announced that it has entered into an exclusive license agreement with AnnJi Pharmaceutical Co., Ltd. (“AnnJi”), a Taiwanese clinical-stage drug company, for AJ201, a first-in-class clinical asset currently in a Phase 1b/2a study in the U.S. for the treatment of spinal and bulbar muscular atrophy (“SBMA”), also known as Kennedy’s Disease.

“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. 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,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue.

SBMA 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 focused 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 malesⁱ. Currently, there is no effective treatment for SBMA.

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 U.S., 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 (“ODD”) 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% of the Company’s current total number of outstanding shares of common stock. The agreement includes the U.S., Canada, European Union, Great Britain and Israel as exclusively licensed territories.

Lindsay A. Rosenwald, M.D., Chairman of the Board of Avenue, stated, “We are excited to progress the clinical development of AJ201 to treat SBMA and further expand Avenue as a leading neurological company.”

About Avenue Therapeutics
Avenue Therapeutics, Inc. (Nasdaq: ATXI) is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic and rare diseases. It is currently developing three assets including AJ201, a first-in-class asset for spinal and bulbar muscular atrophy, BAER-101, an oral small molecule selective GABA-A α2/3 receptor positive allosteric modulator for CNS diseases, and IV Tramadol, which is in Phase 3 clinical development for the management of moderate-to-moderately-severe pain in adults in a medically supervised healthcare setting. Avenue is headquartered in Miami, FL and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.avenuetx.com.

About AnnJi Pharmaceutical
Founded in 2014, AnnJi Pharmaceutical Co., Ltd. (“AnnJi”) is an R&D based, clinical-stage new drug company dedicated to the development of first-in-class small molecules focusing on indications with highly unmet needs in the therapeutic areas of neurology, dermatology, and inflammatory disorders, including rare diseases such as idiopathic pulmonary fibrosis and Kennedy’s disease, or SBMA (Spinal and bulbar muscular atrophy).

AnnJi’s mission is to develop innovative therapeutics to improve the quality of life of patients with neglected diseases. AnnJi’s goals are to translate and develop unique and highly differentiated drug therapies and to engage global collaborators and business partners in late-stage product development and commercialization.

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Contact:
Jaclyn Jaffe
Avenue Therapeutics, Inc.
(781) 652-4500
ir@avenuetx.com

ⁱ M. Zanovello et al., Unexpected frequency of the pathogenic ARCAG repeat 2 expansion in the general population. Brain, in press (2023).