Arvinas and Pfizer are the first to cross the FDA finish line with PROteoosis TArgeting Chimera (PROTAC) therapy, opening the door to a new option for certain breast cancer patients. However, the companies will no longer be involved as they seek a new partner to commercialize the drug following lackluster clinical data.
The drug, called Veppanu (vepdegestrant), has received approval to treat adult patients with ER-positive, HER2-negative advanced or metastatic breast cancer with estrogen receptor 1 mutations after receiving at least one prior endocrine therapy. The agreement arrived one month before the FDA’s scheduled June 5 decision date.
CEO Randy Thiel, Ph.D., said in a company press release that Veppanu is a PROTAC, a type of proteolytic agent that Albinus has been developing since 2013.
“This milestone shows that targeted protein degradation can lead to meaningful clinical effects,” Thiel explained. “It also strengthens our confidence in the breadth and versatility of our exciting clinical pipeline across oncology, neurodegenerative and neuromuscular diseases.”
The drug is Albinus’ first approved product, but the company and its big drug partners are taking their hands off the wheel when it comes to commercialization. In September, the pair outlined plans to license out the commercialization rights to the drug to a third party, believing this was “the best path to maximizing the value of bepdegestrant.”
Albinas said in a statement Friday that the two are “on track” to announce their selections.
The FDA’s validation of Veppanu comes after uncertainty about the drug’s development path, which caused partner companies to narrow their focus.
The companies’ Veritac-2 Phase 3 trials were controversial, pitting Veppanu against AstraZeneca’s standard of care Faslodex. In patients with ESR1 mutations, the drug was shown to reduce the risk of disease progression or death by 43% compared with Faslodex, with a median progression-free survival of 5 months compared to 2.1 months for Faslodex.
However, Veppanu failed to improve progression-free survival overall in study subjects not selected for ESR1. At the time, Albinas’ stock price halved in response to this news. And the pair halted two Phase 3 trials that tested Veppanu in combination therapy.
Consultations with health regulators have confirmed that the treatment is likely to be limited to patients with certain mutations in second-line therapy, Alvinas’ former chief executive Dr. John Huston previously said.
Still, this approval is an important milestone for the patient community, Noah Berkowitz, M.D., Albinus’ chief medical officer, commented in the company’s release on Friday.
Up to 40% to 50% of patients with ER-positive, HER2-negative breast cancer treated with endocrine therapy and CDK4/6 inhibitors have ESR1 mutations, leading to endocrine resistance and faster disease progression, Arvinas said.
The approval sent Alvinas’ stock price up about 6% as of the announcement Friday afternoon.
