Over three years following the initial approval of a pharmaceutical by Deciphera Pharmaceuticals, the company now stands at the cusp of introducing a second product to the commercial market. This promising new addition, vimseltinib, has successfully met its primary endpoint in a pivotal phase 3 clinical trial, specifically designed for patients afflicted with tenosynovial giant cell tumors.
In this groundbreaking trial, vimseltinib exhibited its potential by producing a remarkable 40% objective response rate at the 25-week mark among patients with TGCTs who were unsuitable candidates for surgery and had not previously undergone anti-CSF1/CSF1R therapy. In stark contrast, the group administered a placebo showed a disheartening 0% objective response rate, highlighting the substantial efficacy of vimseltinib.
The trial involved more than 80 patients randomly receiving 30 mg of vimseltinib twice a week, while 39 patients were administered a placebo. Notably, eight patients experienced a grade 3 or 4 increase in creatine phosphokinase, a marker of potential muscle injury, with grade 3 being categorized as severe and grade 4 as life-threatening. While Deciphera reported that 6% of treated patients discontinued their treatment due to side effects that emerged during their treatment, the most commonly reported side effect was swelling around the eye.
Furthermore, Deciphera unveiled additional data from the open-label phase 1/2 trial, with patients from two phase 2 cohorts receiving the 30-mg dose of vimseltinib. It’s noteworthy that the median duration of response in these cohorts is yet to be determined, but the company has asserted that these data exhibit “clinically meaningful symptomatic benefit at week 25 across multiple secondary efficacy measures.” The discontinuation rate among patients in the phase 1/2 trial was noted at 9%.
The culmination of this data, particularly bolstered by the compelling phase 3 results, is scheduled to be meticulously compiled and submitted to the FDA in the second quarter of 2024. Additionally, the company is tentatively planning to submit a marketing application for European regulatory approval in the third quarter of the following year, underscoring their commitment to making vimseltinib available to a global patient population.
Steve Hoerter, the CEO of Deciphera, expressed his enthusiasm regarding vimseltinib, stating, “We are excited about the potential for vimseltinib to become our next approved medicine, supporting our continued evolution to a company with multiple marketed products.” This optimism is rooted in the success of the company’s first approval, Qinlock, granted in May 2020 for the treatment of advanced gastrointestinal stromal tumors in fourth-line patients. Qinlock has proven to be a commercial success, generating $41.8 million in revenue during the third quarter of the current year, marking a notable 29% increase compared to the same quarter in 2022.
With financial stability in mind, Deciphera boasts a substantial reserve of $376.9 million in cash and equivalents, ensuring that its operations can be sustained through the year 2026, providing ample resources for its ongoing research and development endeavors.