Vertex Pharmaceuticals’ breakthrough in cystic fibrosis was a medicine that treats just 4% of patients who have the lung disorder. Today, Vertex’s portfolio of blockbuster CF products spans multiple drugs and drug combinations that can treat 90% of CF patients.
CEO Reshma Kewalramani says Vertex aims to eventually reach all CF patients. But as the company moves closer to this goal, it brings up another part of the company’s strategy: pipeline diversification. The lion’s share of Vertex’s revenue comes from its CF portfolio, so revenue growth will have to come from adding on new therapeutic areas. Kewalramani sees this effort as building on the company’s CF work.
“The real drive for Vertex is to invest in serial innovation, not just do it once, but do it repeatedly,” Kewalramani said, speaking in a fireside chat Monday at the World Medical Innovation Forum in Boston. “The goal here is to learn from all the lessons we have from CF and to invest in other disease areas.”
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While Vertex is known for CF drugs, its research in pain medicines actually began even earlier. Vertex began its CF work about 20 years ago, Kewalramani said. The pain program started 25 years ago. The same scientists who worked on CF are the exact ones who now work on the pain program, she said.
The past two decades have not seen new medications for acute pain or chronic pain. With innovation lacking in this therapeutic area, opioids filed the gap. Vertex is trying to change the pain treatment paradigm with a different approach. Opioids treat pain by working in the central nervous system (CNS) to alter the perception of pain, Kewalramani said. By developing drugs that act on the peripheral nervous system, Vertex aims to avoid the safety, tolerability, and addictive problems of drugs that act on the CNS.
Vertex’s most advanced pain product candidate is Suzetrigine, formerly known as VX-458. This non-opioid drug is a small molecule that stops pain signals from reaching the brain by blocking NaV1.8, a sodium channel in peripheral nerves. Kewalramani acknowledges that other companies are also developing sodium channel-blocking drugs. But Vertex is in position to bring one to patients sooner. The FDA accepted Vertex’s new drug application for Suzetrigine in July, setting a Jan. 30, 2025 target date for a regulatory decision.
Vertex separates the pain market into three segments: acute, neuropathic, and musculoskeletal. The latter two segments are what many people would call chronic pain, Kewalramani said. Vertex aims to discover, develop, and commercialize drugs for acute and neuropathic pain. These medicines may also work on musculoskeletal pain, but Kewalramani said that if the company enters the market for such drugs, it will be through a partnership.
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When Vertex launched 35 years ago, the company’s focus was developing small molecule drugs. With that in mind, Kewalramani said it’s interesting to note that Vertex is now the first to bring patients a CRISPR-Cas9 therapy, Casgevy. This genetic medicine won FDA approval last winter for treating the rare blood disorders sickle cell disease and beta thalassemia.
Vertex’s broadening scope includes cell therapies for type 1 diabetes. VX-880 is what Kewalramani describes as a “naked cell” program. This therapy involves taking stem cells and coaxing them into differentiating into pancreatic islet cells that sense glucose levels and release insulin. But this therapy requires chronic immunosuppression to prevent the immune system from destroying these cells. The next Vertex type 1 diabetes cell therapy, VX-264, takes these same cells and encapsulates them in a proprietary device that shields them from the immune system so no immunosuppression is needed. Both programs are in the clinic.
The third type 1 diabetes program is still preclinical, but here, Vertex is using CRISPR technology to edit cells to avoid sparking an immune response, obviating the need for immunosuppressants. Kewalramani said the potential to move from chronic therapies to a potentially curative therapy makes this one of the programs she is most excited about. Rather than focusing on specific technologies, Vertex is a disease-first company, she said.
“We use whatever modality is needed to transform if not cure those diseases of interest,” Kewalramani explained. “One of the advantages to having that approach is that we can combine modalities.”
Photo by the World Medical Innovation Forum