Vertex’s non-opioid painkiller eased pain intensity after surgery in two phase 2 clinical trials, giving the company the proof-of-concept needed to advance to a pivotal study in the second half of this year.
The eagerly awaited results for VX-548 came from two phase 2 studies in patients who had just had bunion-removal or tummy-tuck surgery. Patients in the high-dose arm received 100 mg initially, followed by 50 mg every 12 hours.
When compared to placebo, VX-548 offered rapid relief of pain intensity over 48 hours, meeting the studies' primary goal. The studies also included reference arms with patients receiving an opioid and acetaminophen.
RBC Capital Market analysts noted slight inconsistencies between the treatment arm and the opioid comparator group, but the results for VX-548 were clearly statistically significant over placebo and clinically meaningful.
As for safety, VX-548 was generally well tolerated, with mild to moderate adverse events reported. Vertex said fewer patients in the high- and mid-dose treatment arms discontinued compared to the placebo group.
With the mid-stage data, Vertex now has what it needs to move the therapy to more advanced studies. The company said the pivotal trials could begin in the second half after discussions with regulators.
Vertex is trying to develop a pain drug that can provide acute relief from surgical pain, without the addictive properties and other side effects of opioid painkillers. The company chose to advance VX-548 after trying and failing with other NaV1.8 inhibitor candidates.
“Our high expectations of achieving therapeutic pain control by inhibiting the NaV1.8 channel have been met with these results from the two acute pain studies,” said Vertex’s Carmen Bozic, M.D., CMO and executive VP for global medicines development and medical affairs.
Bozic noted the “remarkable consistency” on safety, tolerability and efficacy between the two studies.
“We are working with urgency to advance the program into phase 3 with the goal of bringing forward the first novel pain treatment in decades to address the unmet needs of patients suffering from acute pain,” Bozic said.
Full data from the two trials will be presented at a future medical meeting.
"We see a high unmet need for a non-opioid solution with a differentiated mechanism of action, though we think the bar for approval and use will likely be high and will watch for greater clarity on both relative gains vs. opioids and potential for opioid sparing," RBC said in a Thursday note.
VX-548 marks a turning point for Vertex, which has in recent years been focused on its successful cystic fibrosis franchise. While the company has a number of next-gen programs for that disease in the works, a new pain drug is high on its priority list. The company also saw a huge response last year when data for a single patient was released for a trial of a Type 1 diabetes cell therapy called VX880.