Yahoo Finance Vertex (VRTX) Continues to Diversify Beyond CF Franchise
Vertex Pharmaceuticals Incorporated VRTX enjoys a dominant position in the cystic fibrosis (CF) market. Vertex’s CF sales continue to grow, driven by its triple therapy, Trikafta (marketed as Kaftrio in Europe). In the United States, Trikafta sales are being fueled by label expansions to younger age groups (two to five years old). In the ex-U.S. markets, the drug continues to witness strong uptake with recently achieved reimbursements and expanded use in young age groups.
Vertex is also evaluating its CF medicines in younger patient populations and aims to have small-molecule treatments for most people with CF.
In the CF franchise, all eyes are on the triple combination of vanzacaftor, a CFTR potentiator, deutivacaftor, a CFTR corrector and tezacaftor. Vertex has completed two pivotal phase III studies on the vanza triple in CF patients 12 year and older. Data from the studies, released in February 2024, demonstrated a strong benefit-risk profile and the ability to deliver greater restoration of CFTR function than even Trikafta. Vertex has also completed pivotal studies of vanzacaftor/tezacaftor/deutivacaftor in patients 6 to 11 years old.
Based on these data, VRTX plans to submit regulatory applications for the vanza triple for treating people with CF aged six years and older in the United States and EU by mid-2024. This new once-a-day oral combination medicine has the potential for enhanced patient benefit than Trikafta patients and can potentially treat CF patients who have discontinued Trikafta or other Vertex CF medicines. It can also improve dosing (once daily) and lower the royalty burden.
Additionally, Vertex is developing a mRNA therapeutic, VX-522, in partnership with Moderna MRNA for approximately 5,000 people with CF who cannot benefit from its CFTR modulators. Vertex and Moderna have completed a single ascending dose clinical study on VX-522, while a multiple ascending dose (MAD) portion of the study has been initiated. Data from the MAD study is expected in late 2024 or early 2025.
While CF remains the main area of focus, Vertex saw rapid success in its non-CF pipeline candidates’ development in 2023. Vertex and partner CRISPR Therapeutics’ CRSP one-shot gene therapy, Casgevy, was approved for two blood disorders, sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), in multiple regions. These approvals have diversified its commercial opportunity.
Vertex and CRISPR Therapeutics’ Casgevy is the first-ever CRISPR/Cas9-based therapy to be approved anywhere in the world. Vertex and CRSP believe Casgevy has the potential to be a one-time functional cure for SCD and TDT patients, with an estimated patient population of approximately 35,000 across the United States and Europe.
Vertex is also developing treatments for acute and neuropathic pain, APOL1-mediated kidney disease, type I diabetes and alpha-1 antitrypsin deficiency. It has earlier-stage programs in diseases such as muscular dystrophy.
Many of its non-CF candidates represent multibillion-dollar opportunities.
Investors are paying a lot of attention to pain asset VX-548, which, they believe, has blockbuster potential.
Vertex has completed three pivotal phase III acute pain studies on VX-548, a novel first-in-class, non-opioid NaV1.8 inhibitor. The studies were in two different pain models — abdominoplasty, a soft-tissue pain model, and bunionectomy, a hard-tissue pain model, and a single-arm safety and effectiveness study in a broad range of surgical and non-surgical pain conditions.
Data from the studies, announced in January 2024, showed that treatment with VX-548 led to a significant reduction in pain intensity across a range of pain conditions, both surgical and non-surgical, and across a range of settings. Based on the above data, Vertex plans to submit an NDA with the FDA for VX-548 across a broad label in moderate-to-severe acute pain by mid-2024.
In December 2023, Vertex announced encouraging data from a phase II study of VX-548 for painful diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain caused by damage to nerves. The data demonstrated a promising safety and efficacy profile of VX-548, with consistent efficacy seen across all doses studied. Vertex believes VX-548 has blockbuster potential as it can change the standard of care for neuropathic pain, an area with limited treatment options, mostly highly addictive opioid-based medications. Vertex has also initiated a second phase study of VX-548 in patients with painful lumbosacral radiculopathy, another form of peripheral neuropathic pain. Vertex expects to advance VX-548 into pivotal development for DPN in 2024.
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