Vertex Pain Product VX-150 Comes Out Of The Shadows
Executive Summary
Solid Phase II efficacy data for Vertex’s pain product have brought it blinking into the light. Analysts are hoping it will lead to an additional revenue stream for Vertex behind its all-conquering cystic fibrosis franchise.
Significant and clinically meaningful reductions in pain scores in small fiber neuropathy and a breakthrough designation for Vertex Pharmaceuticals Inc.’s VX-150 look set to put the product on a fast path towards the regulators.
Data just reported from the third positive Phase II proof-of-concept study for VX-150 validate the potential role of NaV1.8 inhibition in treating a number of pain conditions, the company said. The product is now in a Phase IIb dose-ranging study in acute pain following bunionectomy.
Vertex chairman, president and CEO Jeffrey Leiden said, “These results show a potential path for the treatment of pain at a time when there is great clinical and societal need for new medicines.” He added that the firm was progressing towards pivotal development with the product as well as taking additional NaV1.8 pain candidates into clinical development beginning in 2019.
Vertex also announced that the US FDA had granted breakthrough therapy designation to VX-150 for the treatment of moderate-to-severe acute pain. With the latest results, Vertex now has positive Phase II data for VX-150 in multiple pain conditions, also including chronic pain caused by osteoarthritis and acute pain following bunionectomy.
Analysts at BMO said they believed Vertex's pain program could provide “another layer of revenue growth,” but other analysts were keen to hear the company’s strategy for the product in its various potential pain indications.
The six-week study evaluated oral treatment with 1,250mg of VX-150 (n = 46) or placebo (n = 43) every 24 hours in patients with chronic pain caused by small fiber neuropathy.
The NaV1.8 inhibitor study met its primary endpoint, showing that treatment with VX-150 produced statistically significant and clinically meaningful pain reduction, as measured by the within-group change from baseline in the weekly average of daily pain intensity on the 11-point numeric rating scale (NRS) at Week 6. VX-150 was generally well tolerated.
A further pre-specified endpoint looked at the difference between the two groups and also found a treatment difference in the mean change from baseline of -1.09 (95% CI: -1.88 to -0.29) in the weekly average of daily pain intensity on the NRS, as reported in the daily diary, at Week 6. This difference was observed as early as Week 1, and was sustained through the six-week treatment period. VX-150 was also well tolerated.
Vertex Pharmaceuticals, Dec. 2018
The data have made analysts sit up and taking notice of the product, long overshadowed by Vertex’s cystic fibrosis franchise, and they are keen to hear more of the company’s plans in the new year, particularly in terms of partnering in the larger pain indications.
Vertex is considering its options for VX-150’s various potential indications, said analysts at JMP in a Dec. 18 research note.
“Although no final decisions have been made, Vertex feels confident that it has the infrastructure in place to develop VX-150 for this treatment group internally whereas the company likely would need a partner for its chronic pain indication due to the larger sales force requirements for this population with a hybrid in-between approach for the neuropathic pain group,” they said. “We think the area of non-opioid pain control is a public health topic of current interest, and therefore we believe that Vertex would easily find partners for the program if that is the route it chooses.”
Morgan Stanley analysts said they were awaiting news from management on its strategic priorities. “While we understand that management wants to be prudent with its plans to invest in VX-150, including confirming dose ranging and safety in the upcoming acute pain study, we see 2019 as the year in which management needs to lay out its plans on how to advance VX-150 into a pivotal program. We believe a JV with a large pharma company would be a major upside driver which could cause investors to recognize the value of this drug.”
NaV1.8 is a voltage-gated sodium channel expressed in peripheral neurons that transmit pain. According to Informa’s drug database Pharmaprojects, there are two other investigational pain products acting on the NaV1.8 channel, Vertex’s own VX-128 and Sunovion Pharmaceuticals Inc.’s DSP-2230 (both listed as being in Phase I). DSP-2230 is also a NaV1.7 inhibitor.