Avenue acquires rights to AJ201 for SBMA in certain countries

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by Andrea Lobo |

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Avenue Therapeutics has won the exclusive rights to develop and commercialize AJ201, AnnJi Pharmaceutical’s experimental treatment for spinal and bulbar muscular atrophy (SBMA), in certain regions of the globe under the terms of a new licensing agreement.

Specifically, the agreement lists the U.S., Canada, the European Union, Great Britain, and Israel as newly licensed territories for Avenue.

Under the terms of the deal, AnnJi will receive an initial payment of $3 million, and is entitled to additional earnings when certain developmental, regulatory, and commercialization milestones are reached. The company also will receive royalties on net sales if the product reaches the market.

“The license for AJ201 brings a cutting-edge asset into Avenue’s pipeline that is the lead molecule in the clinic to treat [SBMA], a debilitating rare neuromuscular disorder,” Alexandra MacLean, MD, CEO at Avenue, said in a company press release.

“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,” MacLean added.

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Avenue, AnnJi agree to deal to advance AJ201

Also known as Kennedy’s disease, SBMA is a rare type of SMA marked by the degeneration of nerve cells involved in voluntary movements, leading to progressive muscle weakness and atrophy. It affects mainly men, and symptoms usually start during adulthood.

The disease is caused by mutations in the AR gene, located in the X chromosome, which provides the instructions to make the androgen receptor (AR) protein.

The specific genetic defect that underlies SBMA involves an excessive repetition of three DNA letters in a certain region of the AR gene. This results in an abnormal AR protein with a polyglutamine (polyQ) expansion, which tends to clump up (aggregate) and accumulate inside nerve and muscle cells, disrupting their function.

SBMA weakens the muscles in the mouth and throat, affecting chewing, swallowing, and speech. It also impacts limb muscles, leading to difficulty walking, and falls.

As of yet, there is no effective treatment for the disease.

AJ201 is a new chemical entity designed to degrade the abnormal AR protein, facilitate correct protein folding, and activate anti-oxidative stress responses. It does that by activating three proteins: Nrf1, Hsf1, and Nrf2.

According to AnnJi, AJ201 lowered the accumulation of mutant AR in muscles and reduced muscle atrophy and defects in motor function in an animal model.

The molecule was investigated in humans in a Phase 1 clinical trial (NCT04392830) that demonstrated its safety and tolerability in healthy volunteers. That trial, completed in 2021, involved 72 healthy people, ages 18-55.

We are excited to progress the clinical development of AJ201 to treat SBMA and further expand Avenue as a leading neurological company.

A Phase 1b/2a clinical trial (NCT05517603) will now test the therapy in an estimated 24 SBMA patients, 18 and older. Its aim is to evaluate AJ201’s safety, tolerability, and pharmacokinetics, or the medicine’s movement into, through, and out of the body.

Patient screening has started for the trial, which will be conducted at six clinical sites across the U.S.

“We are excited to progress the clinical development of AJ201 to treat SBMA and further expand Avenue as a leading neurological company,” said Lindsay A. Rosenwald, MD, chairman of the board at Avenue.

AJ201 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for SBMA, Huntington’s disease, and spinocerebellar ataxia.

This designation is given to therapies for diseases affecting fewer than 200,000 people in the U.S., and provides a range of benefits, including exemption from FDA fees and seven years of market exclusivity upon approval.

For the initial closing of the new licensing agreement, Avenue will issue 831,618 shares of its common stock to AnnJi, and up to an additional 276,652 shares once a clinical milestone is reached, up to a maximum of 19.99% of Avenue’s current number of outstanding shares of common stock.

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