Research and Pipeline

Vertex is focused on discovering, developing and commercializing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.

These studies are investigating treatments or outcomes that have not all received approval from a health authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a health authority. 

About Pain

What is pain? While pain can happen anywhere and anytime, chronic pain can be a debilitating disorder that affects millions of people worldwide. Pain can also be acute, meaning it can occur suddenly and may not last more than a few weeks. Chronic pain, on the other hand, can last several months or longer and can be anywhere from mild to very severe. Pain can develop from a variety of pathophysiological and psychological conditions. Some common causes of pain include arthritis, neuropathies, operations, musculoskeletal injuries, etc. Poorly managed pain can lead to a significant decrease in quality of life, and it’s often associated with complications, such as sleep interruption, immobility, inability to work and major depression (which can potentially lead to suicidal ideation). 

The management of pain is complex. Opioids are generally prescribed for acute pain but are often unsuitable for chronic use. Anti-inflammatory drugs can be used for both acute and chronic pain, but their use is limited due to side effects. Beyond these approaches there are limited options and there haven’t been any major innovations in pain treatment for decades. There is a significant unmet need for new treatments that can be effective for various types of pain. 

How is pain assessed? Pain can result from many types of conditions. It is one of the most common symptoms that causes people to go to a doctor. Pain is often assessed on a zero to 10 scale, with zero being no pain and 10 being the worst imaginable pain. For children, this scale is often translated into a range of happy to sad faces.

Because pain itself is a symptom, medical evaluations focus on both the source of the pain and the impact of the pain. During an evaluation, the health care professional will typically start with a physical exam and ask detailed questions about the pain and its impact on the person’s life. Other tests may include blood and imaging tests. Common causes, such as arthritis, may be diagnosed by a primary care provider. Less common causes, such as neuropathic pain, may only be diagnosed after evaluation and testing by a neurologist or a pain specialist.

What is the underlying cause of pain? Pain is usually caused by an injury or its consequences. There are many factors that contribute to developing a pain response, including the presence of an illness or injury, other coexisting conditions and/or specific genes that influence pain tolerance. While we cannot prevent the initial triggers of pain, we can influence how the human body processes pain signals to ease the burden of pain. The nervous system, through voltage-gated sodium channels (NaV), is responsible for how the body senses, transmits and interprets pain. These NaV channels help mediate activity in excitable cells throughout the body. Some channels, like NaV 1.7 and 1.8, which have been validated through human genetics, play an important role in transmitting pain signals from peripheral sensory neurons to the central nervous system.

The Vertex Approach

We’re focused on researching and discovering medicines aimed at selectively targeting the underlying cause of pain response, including acute or chronic severe pain caused by a variety of conditions. Although the role of voltage-gated sodium channels like NaV 1.7 and 1.8 in human pain sensation has been understood since the cloning of the NaV channels in the 1980s, no one has successfully developed a treatment that selectively uses these mechanisms to treat pain. Because of the significant prevalence of pain across the world and the complications it may cause, we believe there is a significant unmet need for potential new treatments.

We're investigating an approach to treat pain by targeting specific sodium channels in the NaV family that have been validated by human biology. Our aim is to interrupt pain signals and prevent them from traveling from the sensory nerves to the brain. We continue to discover, research and develop a portfolio of small molecule medicines as potential medicines for the treatment of both acute and chronic pain, including chronic neuropathic pain. By inhibiting the specific channels validated by human biology, we hope to alleviate pain.

Pipeline

Pain

VX-548
Phase 2

We continue to discover, research and develop a portfolio of small molecule medicines as potential non-opioid medicines for the treatment of both acute and neuropathic pain. Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain. 

ADDITIONAL SMALL MOLECULES
Preclinical

We continue to discover, research and develop a portfolio of small molecule medicines as potential non-opioid medicines for the treatment of both acute and neuropathic pain. Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain. 

Our Studies 

In 2021, Vertex initiated a Phase 2 study evaluating VX-548 in subjects with acute pain after a bunionectomy. To learn more, visit clinicaltrials.gov.

In 2021, Vertex initiated a Phase 2 study evaluating VX-548 in subjects with acute pain after an abdominoplasty. To learn more, visit clinicaltrials.gov.

News

For more information from Vertex on pain, please visit the Newsroom.