Drug Gets Breakthrough Therapy Designation for HER2-Mutant Lung Cancer


The FDA granted a breakthrough therapy designation to BAY 2927088 after it showed promise in treating patients with HER2 mutations.

Image of a lung scan with tumors highlighted in red.

BAY 2927088, a novel treatment, has been granted a breakthrough therapy designation by the FDA for certain patients with lung cancer.

The Food and Drug Administration (FDA) granted a breakthrough therapy designation to BAY 2927088 for the treatment of adults with pretreated unresectable (unable to be removed via surgery) or metastatic (spread to other parts of the body) non-small cell lung cancer (NSCLC) with activating HER2 (ERBB2) mutations, according to a press release from Bayer, the pharmaceutical company behind BAY 2927088.

According to the FDA’s website, breakthrough therapy designations are granted to treatments for serious illnesses when preliminary clinical evidence shows that the treatment at hand may lead to better outcomes than the current standard of care. Once a drug gets the breakthrough therapy designation status, it is given a fast-track designation (expedited review from the FDA); guidance on drug development programs; and organizational commitment from senior managers at the agency.

Approximately 2% to 4% of advanced cases of NSCLC have HER2-activating mutations, according to the release. In 2022, the FDA approved Enhertu (trastuzumab deruxtecan) for patients with HER2-mutant NSCLC, marking the first targeted option for that patient population.

READ MORE: FDA’s Enhertu Approval is ‘Even More Important’ for a Group of Patients With Difficult to Treat Lung Cancer, Says Expert

Now, this breakthrough therapy designation was based on findings from a first-in-human phase 1 trial that is studying the safety pharmacokinetics (drug-body interactions) and preliminary cancer-fighting ability of BAY 2927088 in approximately 460 patients with HER2- or EFGR-positive NSCLC.

According to the trial’s listing on ClinicalTrials.gov, the main goals of the study focus on safety, side effects and maximum tolerated dose, as well as overall response rate (percentage of patients whose cancer shrinks from treatment), while secondary endpoints include disease control rate, duration of response, 12-month progression-free survival (percentage of patients who are still alive without disease progression one year after treatment) and 12-month overall survival (percentage of patients who are still alive at 12 months).

“Early clinical evidence suggests that BAY 2927088, our investigational novel oral tyrosine kinase inhibitor, has the potential to benefit patients with NSCLC harboring a HER2 mutation that have progressed on a prior systemic therapy and currently have no other approved treatment available,” said Dr. Dominik Ruettinger, Head of Research and Early Development for Oncology at Bayer’s Pharmaceuticals Division, in the press release.

BAY 2927088 — which is administered orally — is a type of drug called a tyrosine kinase inhibitor (also known as a “TKI”) that works by blocking certain tyrosine kinases, which are enzymes that send signals to cancer cells telling them to proliferate. By interrupting those cell messages, the tumors may stop growing and dividing.

In this instance, BAY 2927088 blocks HER2, including HER2 exon 20 insertions and EGFR, which both contribute to lung cancer proliferation.

“This breakthrough therapy designation is a significant milestone in our relentless efforts to develop innovative therapies for the treatment of lung cancer characterized by specific genomic markers. We will continue working closely with the FDA to advance BAY 2927088 through the clinic and look forward to providing these patients with lung cancer and their physicians with a targeted, effective treatment option,” Ruettinger, said.

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