Verastem Oncology has concluded the rolling submission of a new drug application (NDA) to the US Food and Drug Administration (FDA) for a combination of avutometinib and defactinib for low-grade serous ovarian cancer (LGSOC).
Avutometinib is an oral rapidly accelerated fibrosarcoma/mitogen-activated protein kinase kinase clamp while defactinib is an oral selective focal adhesion kinase inhibitor.
The NDA was submitted under the FDA’s accelerated approval pathway, with Verastem requesting a priority review.
If the FDA grants priority review, a decision on the NDA could come within six months after the 60-day filing period.
Verastem initiated the rolling NDA submission in May 2024, based on the preliminary results from the Phase II RAMP 201 study.
The two-part, multicentre, adaptive, parallel cohort, randomised, open-label trial assessed the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent LGSOC.
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The study reported an overall response rate of 44%, median progression-free survival of 22 months, and a six-month disease control rate of 70% in patients with Kirsten rat sarcoma viral oncogene homolog (KRAS) mutant LGSOC.
The treatment was also generally well-tolerated, with a discontinuation rate due to adverse events of 10% across all patients.
In addition to the RAMP 201 study findings, the NDA includes supportive data from the FRAME Phase I trial.
The combination was previously awarded breakthrough therapy designation by the US regulator to treat patients with recurrent LGSOC after one or more previous lines of therapy, along with platinum-based chemotherapy.
Avutometinib, alone and in combination with defactinib, received orphan drug designation status for treating LGSOC.
Verastem is actively enroling patients in the international Phase III RAMP 301 trial, which the company said will serve as a confirmatory study for the initial indication.
This trial is also anticipated to support an expanded indication, irrespective of KRAS mutation status.
