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Vertex Pharmaceuticals Incorporated, a global biotechnology company investing in scientific innovation to create transformative medicines for people with serious diseases, has announced important advancements across its suzetrigine pain programme, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades. Suzetrigine is an oral selective NaV1.8 pain signal inhibitor (formerly known as VX-548).
Following the positive phase 3 results in acute pain announced in January 2024, the US Food and Drug Administration (FDA) has granted a rolling New Drug Application (NDA) submission for suzetrigine in moderate-to-severe acute pain. Vertex has started the rolling submission process and is on track to complete the submission in the second quarter of 2024. Suzetrigine was previously granted FDA Fast Track and Breakthrough Therapy designations in moderate-to-severe acute pain.
In neuropathic pain, Vertex released positive results from its phase 2 study in December 2023 and recently completed a successful end-of-phase 2 meeting with the FDA. Vertex is now preparing to initiate a phase 3 pivotal program of suzetrigine in patients with DPN in 2H 2024. In addition, the FDA recently granted suzetrigine breakthrough therapy designation for the treatment of pain associated with DPN.
The phase 3 program will include two identical 12-week randomized, double-blind, placebo-controlled studies evaluating the efficacy and safety of suzetrigine (70 mg once daily) in patients with DPN. The primary endpoint for both studies will be the change from baseline in weekly average of daily pain intensity on the numeric pain rating scale (NPRS) assessed at week 12 compared to placebo. Both studies will also include a key secondary endpoint of change from baseline in the weekly average of daily pain intensity on the NPRS at week 12 compared to pregabalin. Approximately 1,100 patients are expected to enroll in each Phase 3 study. After completing participation in the randomized controlled studies, patients may roll over into an open-label study to evaluate the long-term safety and effectiveness of suzetrigine in DPN.
Additionally, Vertex continues to enroll its phase 2 study of suzetrigine in patients with lumbosacral radiculopathy, or LSR, which is pain caused by impairment or injury to nerve roots in the area of the lumbar spine. The company is on track to complete enrollment in the phase 2 LSR study by the end of the year.
“Today marks a significant milestone on our journey to redefine the treatment of pain,” said Carmen Bozic, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex. “Given the favorable benefit/risk profile demonstrated by suzetrigine across the entire clinical program and the positive interactions with regulators, we are excited by the opportunity to rapidly advance suzetrigine, a new non-opioid potential treatment, for the millions of patients suffering from acute and peripheral neuropathic pain.”
In line with its pain portfolio serial innovation strategy, Vertex continues to advance preclinical and clinical development of additional NaV1.8 and NaV1.7 pain signal inhibitors, for use alone or in combination, in acute and neuropathic pain. The company intends to advance its next generation NaV1.8 pain signal inhibitor VX-993 oral formulation into phase 2 acute pain and peripheral neuropathic pain studies later this year. Vertex also anticipates initiating a phase 1 study of an intravenous formulation of VX-993 later this year.
Acute pain is a disabling condition and is defined as pain lasting less than 3 months. It is estimated that over 80 million people are prescribed a medicine for acute pain every year in the US. Due to limited treatment options, there is an unmet need in acute pain management to improve the patient experience and reduce the economic and societal burden.
Peripheral neuropathic pain, or PNP, is a significant area of unmet need for patients suffering from pain. PNP is a collection of chronic conditions including pain associated with diabetic peripheral neuropathy (DPN), lumbosacral radiculopathy (LSR), small fiber neuropathy and trigeminal neuralgia. DPN and LSR are two of the largest segments within the estimated 10 million patients who are prescribed a medicine for PNP every year in the US.
Suzetrigine (formerly VX-548) is an investigational oral, selective NaV1.8 pain signal inhibitor that is highly selective for NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. NaV1.8 is a genetically validated target for the treatment of pain, and suzetrigine has demonstrated a favorable benefit/risk profile in three phase 3 studies and two phase 2 studies in moderate-to-severe acute pain. Suzetrigine also demonstrated positive results and a well-tolerated profile in a phase 2 study in patients with pain associated with diabetic peripheral neuropathy, a type of peripheral neuropathic pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of pain signal inhibitors that have the potential to provide effective relief of pain without the limitations of currently available therapies, including the addictive potential of opioids.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programmes in these diseases.
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