Verastem Targets Accelerated Approval for Pancreatic Cancer Drug, Expands Trial Plans

Key Points

• Verastem is targeting a potential accelerated-approval filing for VS-7375 in pancreatic cancer, aiming to complete enrollment in the main TARGET-D 201 cohort by year-end and potentially submit in the second quarter of the following year. Timing depends on response durability and FDA discussions; the company’s reported 41% response rate came from a small 22-patient dataset.
• The company is testing VS-7375 alone and with cetuximab in second-line pancreatic cancer, with data expected as the study matures around midyear. Verastem says the phase 2 trial could potentially support accelerated approval for both regimens, while a frontline phase 3 study is also planned.
• Verastem plans to expand VS-7375 studies into colorectal and lung cancers, including a planned frontline colorectal study in the first half of the following year. The company also said its approved avutometinib-defactinib combination’s sales cover its commercial sales force, and incoming CEO Michael Bailey is expected to start Jan. 1.

Verastem NASDAQ: VSTM is advancing its experimental KRAS G12D inhibitor VS-7375 toward potential accelerated approval in pancreatic cancer, with plans to expand studies in colorectal and lung cancers, company executives said at an investor event.

Vice President of Development Ting Wu said Verastem expects to complete enrollment in the main cohort of its TARGET-D 201 pancreatic cancer study before the end of the year. The company is aiming for a potential second-quarter submission the following year, although Wu said the timing depends on the durability of response data and further communication with the U.S. Food and Drug Administration.

Chief Scientific Officer Jonathan Pachter said approval could come as early as the end of the following year. The timeline is a company projection, not a regulatory commitment.

Pancreatic cancer data and dosing

During the discussion, Guggenheim Senior Biotech Analyst Michael Schmidt cited a 41% response rate for VS-7375 in previously treated pancreatic cancer patients. Wu said the dataset included all patients dosed at 900 milligrams in the phase 1 study and reported confirmed responses. Pachter described the dataset as involving 22 patients, including two complete responses.

Wu also cited a six-month progression-free survival rate of 67%, while noting that the company had not reported a median progression-free survival figure. She said the sample size was not large but described the results as consistent across treatment settings.

Verastem is pursuing a 900-milligram daily dose in its U.S. studies, while partner GenFleet selected 600 milligrams for studies in China. Wu said GenFleet had dosed only three patients at the higher level, while Verastem more fully characterized it. She said exposure increased proportionally between the two doses and that their tolerability profiles were identical.

Wu described the main adverse events as gastrointestinal disorders, predominantly low grade. She said about 10% to 15% were grade 2.

Combination and frontline studies

Verastem is also evaluating VS-7375 with cetuximab. Pachter said preclinical work showed tumor regressions with the combination in pancreatic cancer models. Wu said early clinical observations included responses at what she described as sub-therapeutic doses and deeper, faster responses at higher doses.

Wu said the company could disclose early phase 1 combination observations in the first quarter of the following year. TARGET-D 201 is also randomizing second-line pancreatic cancer patients to VS-7375 alone or with cetuximab, with data expected around midyear as it matures. Pachter said the phase 2 study is moving toward 80 patients in each group and could potentially support accelerated approval for both regimens.

For the frontline pancreatic cancer phase 3 study, TARGET-D 301, Wu said Verastem chose combinations with cetuximab or gemcitabine and nab-paclitaxel rather than combining VS-7375 with FOLFIRINOX. She cited tolerability and said single-agent activity could become important after patients reduce or stop chemotherapy. The study’s control arm will allow either chemotherapy choice, the executives said.

Colorectal and lung cancer plans

Wu said Verastem’s strategy across the three cancers includes single-arm studies in previously treated patients intended to support accelerated approval, alongside frontline confirmatory studies. For colorectal cancer, she said a frontline study is planned for the first half of the following year.

Wu estimated that a 30% response rate could be a benchmark for accelerated approval in third-line or later colorectal cancer and said GenFleet had reported a 44% response rate for VS-7375 plus cetuximab in that population. More than 50 patients had been dosed in Verastem’s phase 2 study, she said.

In previously treated non-small cell lung cancer, the TARGET-D 202 study permits patients with prior taxane exposure, Wu said. Pachter added that the phase 2 trial includes a cohort of 25 patients with asymptomatic brain metastases to assess whether the treatment reduces those lesions.

Commercial product and leadership

Pachter said Verastem’s approved avutometinib and defactinib combination received its first novel approval in May 2025 and that sales now cover the commercial sales force. He also said the company’s recent fundraising supports clinical development.

On leadership, Pachter said Dan Paterson, CEO since 2011, is transitioning out and that incoming CEO Michael Bailey is set to start Jan. 1. He said Bailey is positioned to lead commercialization as the company develops VS-7375.

About Verastem (NASDAQ:VSTM)

Verastem, operating as Verastem Oncology, is a biopharmaceutical company focused on developing and commercializing targeted therapies for cancer. The company's research is centered on tumors driven by abnormalities in the RAS/MAPK signaling pathway, which can promote cancer-cell growth and resistance to treatment.

Verastem's leading product candidates include avutometinib, an oral RAF/MEK clamp designed to inhibit signaling within the RAS/MAPK pathway, and defactinib, an oral focal adhesion kinase inhibitor.

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