AnnJi Pharmaceutical Co. (AnnJi) has entered into an exclusive license agreement with Avenue Therapeutics Inc. (Avenue, NASDAQ: ATXI) for the development and commercialization of AJ201 in the U.S., Canada, European Union, Great Britain, and Israel for spinal and bulbar muscular atrophy (SBMA), also known as Kennedy’s Disease. 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 amounting up to 250 million USD, as well as up to 2-digit percentage royalty of the net sales. In addition, AnnJi will retain the manufacturing right to provide clinical and commercial supply of AJ201 to Avenue under agreed sale price and will be compensated for any Avenue’s sublicense activity. Avenue will also issue 831,618 shares of its common stock to AnnJi in connection with the initial closing of the license transaction as an equity upfront and additional share upon achievement of a clinical milestone, aggregating in total to not more than 19.99% of Avenue’s current total number of outstanding shares of common stock. This agreement is one of the largest license deals for a small molecule drug in early Phase 1b/2a clinical development in the past twenty-four months. Greenberg Traurig served as AnnJi’s legal advisor in this transaction.

AJ201 is a novel small molecule new drug and a first-in-class treatment, which has the potential to treat Kennedy’s Disease through multiple mechanisms including degradation of the abnormal androgen receptor (AR) protein, which is believed to be the cause of the disease, as well as suppression of  proinflammatory cytokines and protection of cells from oxidative stress. Phase 1 clinical trial in healthy subjects has demonstrated the safety of AJ201 in humans. AJ201 is currently being investigated in a multicenter, randomized, double-blind clinical trial in six clinical sites across the U.S. The study aims to evaluate the safety and clinical response of AJ201 in Kennedy’s Disease patients.

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. AJ201 also received ODD from European Medicines Agency for the indications of SBMA.

About Spinal and Bulbar Muscular Atrophy (SBMA, or Kennedy’s Disease)

The disease is a rare, inherited, X-linked genetic neuromuscular disease, and typically an adult-onset disease, where symptoms occur mainly between the ages of 20 and 50, primarily affecting men. The genetic defect underlying the disease is the expansion of a polyQencoding CAG repeat in the first exon of the AR gene. The accumulation of pathological mutant AR aggregates is believed to be responsible for the cytotoxicity, excessive oxidative stress and chronic neuroinflammation, leading to the muscle atrophy and death of neurons.

The disease is characterized by symptoms such as muscle weakness leading to difficulty in walking and injury caused by falling. Patients have difficulty in speaking, chewing and swallowing, which often result in airway infection. Kennedy disease affects approximately 1/35,000 males worldwide. Currently, there is no effective treatment for the disease, nor an US FDA approved therapy.

About AnnJi

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 for indications with highly unmet needs in the therapeutic areas of neurology, dermatology, and inflammatory disorders, including rare diseases such as idiopathic pulmonary fibrosis and Spinal and Kennedy’s disease. Other development projects including a selective HDAC6 inhibitor for the treatment of chemotherapy-induced peripheral neuropathy, which is expected to enter the first clinical trials in Q4 2023 and a topical AR degradation enhancer for the treatment of alopecia, which is  expected to enter the clinical trials in Q3 2024.

AnnJi is a privately funded new drug development company supported by major biotech venture capitals in Taiwan, including Taiwania Capital, TaiAn Technologies Corporation, Mega Financial Holding, TCB Bank, Fubon Financial, PIDC, SinoPac, Orient EuroPharma, and Saga Unitek Ventures, among others. For more information, visit https://www.ajpharm.com.

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