Vertex Pharmaceuticals Incorporated - VRTX STOCK NEWS

Orum Therapeutics has entered a global, multi-target license and option agreement with Vertex Pharmaceuticals (VRTX) for the use of Orum's TPD²® technology to develop novel degrader-antibody conjugates (DACs). The agreement focuses on discovering targeted conditioning agents for use with gene editing.

Key financial terms include:

• $15 million upfront payment to Orum
• Up to $310 million in potential option fees and milestone payments per target
• Tiered royalties on potential future global annual net sales

Vertex will be responsible for all research, development, and commercialization activities. The agreement covers up to three targets, with Vertex having the option to obtain worldwide, exclusive licenses for each target after the research period.

Vertex Pharmaceuticals announced that the FDA has accepted its New Drug Application (NDA) for vanzacaftor/tezacaftor/deutivacaftor (vanza triple). This triple combination therapy is for cystic fibrosis (CF) patients aged 6 years and older with at least one F508del mutation or other responsive mutations in the CFTR gene. The application used a priority review voucher, which shortened the review period to six months, setting a PDUFA target action date of January 2, 2025. Additionally, the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for the same treatment, applicable to CF patients in the EU. Submissions have also been made in Canada, Australia, Switzerland, and the U.K.

Vertex Pharmaceuticals (Nasdaq: VRTX) will announce its second quarter 2024 financial results on August 1, 2024, after market close. A conference call and webcast will be held at 4:30 p.m. ET to discuss the results.

U.S. participants can dial (833) 630-2124, while international participants can call +1 (412) 317-0651, referencing the “Vertex Pharmaceuticals Second Quarter 2024 Earnings Call.” The webcast will be accessible via Vertex's website in the 'Investors' section, with an archived version available post-call.

Vertex Pharmaceuticals announced positive results from its ongoing Phase 1/2 clinical trial of VX-880, a stem cell-derived islet cell therapy for Type 1 Diabetes (T1D), during the American Diabetes Association 84th Scientific Sessions. Data from 12 patients who received the full dose showed islet cell engraftment and glucose-responsive insulin production by Day 90.

All patients achieved HbA1c levels below 7.0% and more than 70% time-in-range (70-180 mg/dL). Eleven out of twelve patients reduced or eliminated their use of exogenous insulin. Three patients with at least 12 months of follow-up met the primary endpoint of eliminating severe hypoglycemic events and achieving HbA1c below 7.0%, as well as the secondary endpoint of insulin independence.

VX-880 has been generally well tolerated, with no serious adverse events related to the treatment. Vertex plans to expand the trial to enroll approximately 37 participants as it moves toward pivotal development.

Vertex Pharmaceuticals announced new long-term data for CASGEVY™ (exa-cel) at the 2024 EHA Congress. Data from the CLIMB-111, -121, and -131 trials, with follow-up over five years, show CASGEVY as a transformative and durable treatment for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The efficacy results showed that 92.3% of patients with SCD were free from vaso-occlusive crises for at least 12 months, and 94.2% of TDT patients were transfusion-independent for at least 12 months. The safety profile was consistent with busulfan conditioning and autologous stem cell transplant.

Vertex Pharmaceuticals presented new data at the European Cystic Fibrosis Conference highlighting significant benefits of TRIKAFTA® for cystic fibrosis (CF) patients.

The Phase 3 study showed that TRIKAFTA® significantly improved lung function and reduced pulmonary exacerbations by 72% among patients with rare, non-F508del CFTR mutations. Additionally, the interim analysis of the largest real-world study reported sustained lung function improvement over three years, as well as considerable reductions in rates of lung transplants and death in both the U.S. and Germany.

No new safety concerns were identified.

Vertex Pharmaceuticals (Nasdaq: VRTX) will take part in two upcoming investor conferences in June 2024. CEO Dr. Reshma Kewalramani will present at the William Blair 44th Annual Growth Stock Conference on June 4 at 4:00 p.m. CT. Executives Charles Wagner and Dr. David Altshuler will join a fireside chat at the Goldman Sachs 45th Annual Global Healthcare Conference on June 11 at 11:20 a.m. ET. Both events will be webcast live on the Vertex website, with replays available afterward.

Vertex Pharmaceuticals (Nasdaq: VRTX) has elected Jennifer Schneider, M.D., M.S., to its Board of Directors as an independent director. Dr. Schneider brings over 20 years of expertise in the healthcare sector. She co-founded and leads Homeward Health, targeting healthcare delivery for rural Americans. Previously, she was President and Chief Medical Officer of Livongo Health, where she steered the company through a record-breaking IPO and a major merger with Teladoc Health. Dr. Schneider's background also includes roles at Castlight Health and research at Stanford University. Her addition brings Vertex's board to 11 members, 45% of whom are women. Dr. Schneider's experience is poised to aid Vertex's growth and innovation, particularly in cystic fibrosis, sickle cell disease, and beta thalassemia.

Alpine Immune Sciences reported its financial results for the first quarter of 2024, with a cash position of $362.4 million. Vertex Pharmaceuticals will acquire Alpine for $65 per share, expected to close in Q2 2024. Collaboration revenue decreased to $7.0 million, with an increase in research and development expenses to $22.5 million. Net loss for Q1 2024 was $17.9 million.