Vertex Pharmaceuticals Inc - VRTX STOCK NEWS

Vertex Pharmaceuticals (Nasdaq: VRTX) has received FDA approval for expanded use of TRIKAFTA in treating cystic fibrosis (CF) patients aged 2 and older. The approval covers patients with at least one F508del mutation or a TRIKAFTA-responsive mutation in the CFTR gene. This expansion adds 94 non-F508del CFTR mutations to TRIKAFTA's label, making approximately 300 additional CF patients in the U.S. eligible for treatment. The FDA has also updated safety information, elevating liver injury and liver failure warnings to a boxed warning. TRIKAFTA has been treating the underlying cause of CF since its initial approval in 2019.

Vertex Pharmaceuticals announced results from its Phase 2 study of suzetrigine for painful lumbosacral radiculopathy (LSR). The study met its primary endpoint with a -2.02 point reduction in pain on the Numeric Pain Rating Scale (NPRS), though the placebo arm showed a similar reduction of -1.98 points.

The drug was generally well tolerated with lower adverse events (22.9%) compared to placebo (32.4%). No serious adverse events related to suzetrigine were reported. Post-hoc analyses revealed variability in placebo response across study sites, with better treatment separation in sites with lower placebo responses.

Vertex plans to advance to Phase 3 development for LSR pending regulatory discussions, incorporating learnings to better control placebo response in future studies.

Vertex Pharmaceuticals presented long-term data for CASGEVY™, their CRISPR/Cas9 gene-edited therapy, showing sustained benefits in treating severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Key findings include:

- 93% of SCD patients (39/42) remained free from vaso-occlusive crises for at least 12 months, with mean duration of 30.9 months

- 98% of TDT patients (53/54) achieved transfusion-independence for at least 12 months with adequate hemoglobin levels

The company has activated over 45 treatment centers globally and collected cells from more than 40 patients. CASGEVY is now approved in multiple countries, including the U.S., EU, and UK, with reimbursement agreements secured in several regions.

Vertex Pharmaceuticals reported strong Q3 2024 results with product revenue of $2.77 billion, up 12% year-over-year. The company raised its full-year 2024 product revenue guidance to $10.8-10.9 billion. Q3 performance was driven by TRIKAFTA/KAFTRIO's continued strong performance, with U.S. revenue up 10% to $1.71 billion and international revenue up 14% to $1.06 billion. The company is preparing for two potential near-term launches: vanzacaftor triple in CF and suzetrigine for acute pain. Three additional programs advanced to Phase 3: suzetrigine in DPN, povetacicept in IgAN, and VX-880 in T1D.

Vertex Pharmaceuticals (Nasdaq: VRTX) has announced its management's participation in two upcoming investor conferences in November 2024. Dr. Reshma Kewalramani, CEO and President, will join a fireside chat at the Guggenheim Inaugural Healthcare Innovation Conference on November 12 at 9:00 a.m. ET. Stuart A. Arbuckle, Executive Vice President and COO, will participate in the Stifel Healthcare Conference on November 19 at 8:00 a.m. ET. Both sessions will be available via live webcast on the company's website, with replays archived in the Investors section.

Vertex Pharmaceuticals shared updates on multiple kidney disease programs at ASN Kidney Week Congress. New data shows povetacicept achieved 66% mean UPCR reduction at 48 weeks in IgA nephropathy patients, with 63% reaching clinical remission. In primary membranous nephropathy, povetacicept demonstrated 62% mean UPCR reduction at 24 weeks. The company launched the global Phase 3 RAINIER trial for IgA nephropathy and continues enrollment in Phase 3 AMPLITUDE trial of inaxaplin for APOL1-mediated kidney disease. Both treatments were well-tolerated with mostly mild to moderate adverse events.

Vertex Pharmaceuticals (Nasdaq: VRTX) will present Phase 3 data on suzetrigine, an investigational oral NaV1.8 pain signal inhibitor, at the American Society of Anesthesiologists (ASA) Annual Meeting in October 2024. The data, selected for the "Best Abstract" session, demonstrates compelling efficacy and safety across multiple acute pain conditions. Two randomized, placebo-controlled trials and a single-arm safety study will be presented, marking the first public presentation of these positive results announced in January 2024.

Suzetrigine aims to be the first non-opioid acute pain treatment in over two decades. Vertex has also initiated a Phase 3 program for suzetrigine in painful diabetic peripheral neuropathy and expects results from a Phase 2 study in painful lumbosacral radiculopathy in late 2024. An investor event will be held on October 20, 2024, to discuss the Phase 3 results in acute pain.

Vertex Pharmaceuticals (Nasdaq: VRTX) has announced that it will release its third quarter 2024 financial results on Monday, November 4, 2024, after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET to discuss the results.

Interested parties can access the call by dialing (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and referencing the "Vertex Pharmaceuticals Third Quarter 2024 Earnings Call." A live webcast link will be available on Vertex's website in the "Investors" section. Participants are advised to register at least 15 minutes before the scheduled webcast to ensure a timely connection. An archived version of the webcast will be made available on the company's website for those unable to attend the live event.

Vertex Pharmaceuticals (Nasdaq: VRTX) announced the first oral presentation of Phase 3 data on their investigational vanzacaftor/tezacaftor/deutivacaftor (vanza triple) at the North American Cystic Fibrosis Conference (NACFC). The vanza triple demonstrated non-inferiority to TRIKAFTA® in ppFEV1 and further improvement in CFTR function as measured by sweat chloride. Vertex also presented data showing sustained long-term benefits of TRIKAFTA®, including improvement in pancreatic function in young patients.

The vanza triple has been submitted for regulatory approval in multiple countries, with an FDA PDUFA date of January 2, 2025. New data on TRIKAFTA® from long-term studies in patients ages 2-11 years old showed sustained improvements in lung function and potential improvement in exocrine pancreatic function over time.