Mature Woman Discussing Problems With Counselor

Taking Stock of a ‘Breakthrough’

Over the past year, those of us working in pharmaceutical oncology R&D have watched with awe as industry colleagues worked together to speed vaccinations for COVID-19 from the lab to patients’ arms in record time.

Breakthroughs in cancer research, typically don’t happen that quickly. In fact, chemotherapy remains a standard of care for many cancers that have few effective and safe treatment choices. For example, despite advances in ovarian cancer treatment, patients diagnosed with low-grade serous ovarian cancer (LGSOC) have very few effective and safe treatment options.

Therefore, we are immensely proud that this week the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK inhibitor VS-6766, with defactinib, its FAK inhibitor.

A few months ago, Victoria Clare, CEO of UK-based patient advocacy group, Ovacome, described to our team how patients with LGSOC urgently need better solutions due to low response rates and tolerability issues associated with current therapies. LGSOC is most often diagnosed in women between the ages of 45 to 55 years and it is extremely difficult to treat. The majority of patients experience a significant amount of pain and impact on their lives over a long period of time. Unfortunately, response rates with current therapies have historically been low, and the toxicity profiles of these agents make it difficult to keep patients on therapy. Compared to other cancer types, patients with LGSOC do not have the same hope for new treatments.

Victoria painted a grueling portrait of the lengthy battle that women experience – often diagnosed at a relatively young age – with this highly recurrent and impactful disease, and told us in no uncertain terms to “hurry up.”

Therefore, words cannot begin to express the tremendous respect and appreciation Verastem Oncology owes the oncology doctors, nurses and trial sites. And above all, we are grateful to the women with recurrent LGSOC who have the courage to participate in the studies that achieved this important milestone and are continuing to help us get closer to bringing VS-6766 to patients through our RAMP 201 study.

Hearing from Victoria and other patient advocates, as well as patients, inspires us to work harder and explore collaborations that might finally provide new solutions for other cancer patients in need of better options. Under ordinary circumstances, patients summon tremendous bravery to participate in clinical trials. And over the past year, this resolve was further tested by a global pandemic that impeded many patients from seeking care.

When I arrived at Verastem Oncology less than two years ago, we evaluated where our development efforts could make the most impact moving forward. We made a bold decision to strategically transform our research program around the development of VS-6766 – what is thought to be the only complete vertical blocker of the RAS pathway in clinical development. We are still early in our development of VS-6766, but we have seen enough promising data to wholly commit to exploring and understanding its potential for blocking other RAS-driven cancers beyond LGSOC.

As my colleague Jon Pachter, Chief Scientific Officer at Verastem Oncology noted, it is a remarkable time for biopharmaceutical research targeting RAS-pathway driven cancers such as LGSOC. We still have much work to do, and this story is still being written, but we must all remain relentlessly committed to making the bold choices and courageous decisions that we believe could truly bring the right treatments to the right patients.

Brian Stuglik is CEO of Verastem Oncology. Additional information about the RAMP 201 study sponsored by Verastem Oncology can be found here or on on (NCT04625270).