Vertex Pharmaceuticals Inc Stock Price, News & Analysis

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 An archived version of the webcast will be made available on the company's website for those unable to attend the live event.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.

Vertex Pharmaceuticals (Nasdaq: VRTX) has announced its participation in two upcoming investor conferences in September 2024. Dr. Reshma Kewalramani, CEO and President, and Charles Wagner, EVP and CFO, will engage in a fireside chat at the Morgan Stanley 22nd Annual Global Healthcare Conference on September 5 at 11:30 a.m. ET. Additionally, Susie Lisa, SVP of Investor Relations, and Manisha Pai, Executive Director of Investor Relations, will participate in a fireside chat at the Cantor Global Healthcare Conference on September 17 at 9:10 a.m. ET.

Both events will be webcast live through the Vertex website's Investors section. Replays of the conference webcasts will be archived on the company's website for future reference.

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.