Vertex Pharmaceuticals Inc - VRTX STOCK NEWS

Vertex Pharmaceuticals has secured a reimbursement agreement with NHS England for eligible transfusion-dependent beta thalassemia (TDT) patients to access CASGEVY™, a CRISPR/Cas9 gene-edited therapy. This agreement follows positive guidance from NICE recommending CASGEVY's use in the NHS. The therapy received the world's first authorization for a CRISPR-based gene-editing treatment from the UK's MHRA on November 15, 2023.

Vertex is establishing a network of authorized treatment centers throughout England for therapy administration. The company is also working to ensure access for eligible sickle cell disease patients in England and collaborating with reimbursement authorities in the EU to bring this innovative therapy to eligible SCD and TDT patients in other regions.

Vertex Pharmaceuticals (Nasdaq: VRTX) announced its Q2 2024 financial results, reporting a 6% increase in product revenue to $2.65 billion compared to Q2 2023. The company has raised its full-year product revenue guidance to $10.65-$10.85 billion. Significant regulatory milestones were achieved, including FDA Priority Reviews for Vanzacaftor triple therapy in cystic fibrosis (CF) and Suzetrigine for acute pain with target action dates in January 2025.

Net product revenue rose 7% in the U.S. and 5% internationally. However, Vertex reported GAAP and non-GAAP net losses of $3.6 billion and $3.3 billion, respectively, due to $4.4 billion in acquired in-process R&D expenses linked to its acquisition of Alpine Immune Sciences. Despite this, the company remains committed to advancing a robust clinical pipeline, with multiple milestones expected in H2 2024, including the launch of CASGEVY for sickle cell disease and transfusion-dependent beta thalassemia.

Vertex's cash and equivalents decreased to $10.2 billion, reflecting the Alpine acquisition cost. The company expects combined GAAP R&D and SG&A expenses to range from $5.0-$5.2 billion for 2024.

Vertex Pharmaceuticals (Nasdaq: VRTX) announced that the FDA has accepted its New Drug Application (NDA) for suzetrigine, an investigational non-opioid pain signal inhibitor for moderate-to-severe acute pain. The FDA granted priority review with a PDUFA target action date of January 30, 2025. Suzetrigine, already granted Fast Track and Breakthrough Therapy designations, could be the first new class of acute pain medicine in over 20 years.

The drug aims to fill the gap between well-tolerated but less effective medicines and opioids with known risks. It has the potential to treat millions of patients suffering from acute pain annually in the U.S., addressing a critical unmet need in pain management.

Genomics plc and Vertex Pharmaceuticals (NASDAQ: VRTX) have extended their drug discovery collaboration for three years, now running until 2026. The partnership, which began in 2018, aims to accelerate drug discovery using genomics and machine learning. Key points of the extension include:

1. Expanding the range of genomic insights and therapeutic areas under study
2. Exploring the use of genetic tools to improve patient population identification
3. De-risking the selection of biomarkers for early drug efficacy readouts

The collaboration has already focused on using human genetics to identify causal disease pathways and novel targets. Both companies express excitement about continuing their work to develop transformative medicines for serious diseases with high unmet medical needs.

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.