Vanzacaftor

Pictured: Vertex Pharmaceuticals' office in Massachusetts/iStock, hapabapa Vertex Pharmaceuticals on Monday released its first-quarter 2024 earnings results, reporting a 13% year-over-year increase in product revenue driven by the continued growth of its cystic fibrosis treatments. In the three months ending March 31, Vertex brought in $2.69 billion, up from its product revenue of nearly $2.38 billion during the same period a year prior. The biotech’s adjusted earnings per share was $4.76 in the first quarter, beating analyst estimates of $4.06, according to LSEG data. Experts had also forecasted a quarterly revenue of $2.58 billion. Vertex’s net income surged 56% from $794 million in the first quarter of 2023 to over $1.24 billion in the most recent quarter. The cystic fibrosis (CF) treatment Trikafta (elexacaftor/tezacaftor/ivacaftor)—which is sold as Kaftrio in territories outside the U.S.—continued to be Vertex’s strongest asset and primary driver of growth, bringing in $2.483 billion in the first quarter, up from $2.097 billion during the same time period last year. In the U.S., Vertex attributed the spike in Trikafta earnings to its April 2023 approval for use in patients aged two to five years. A similar pediatric approval for Kaftrio from the European Commission in November 2023 bolstered the biotech’s CF business internationally. “Our outlook in CF is bright in the short, medium, and long term,” COO Stuart Arbuckle said in an investor call. “We will drive growth in the near term by reaching more eligible patients, including younger age groups and additional geographies.” At the end of the first quarter, the biotech had total current assets amounting to nearly $24 billion. Looking ahead to the rest of the year, Vertex reiterated its 2024 revenue guidance of $10.55 billion to $10.75 billion. The biotech has lined up several near-term clinical milestones and product launch opportunities to help it meet its sales forecast. For instance, Vertex is awaiting the FDA’s verdict on its investigational CF treatment called vanza triple—consisting of vanzacaftor, tezacaftor and deutivacaftor—which it is proposing as a once-daily therapy for patients aged 6 years and above. In February 2024, the biotech released data from the Phase III SKYLINE 102 and SKYLINE 103 studies, which showed that the drug combo could match Trikafta in terms of improving forced expiratory volume in CF patients aged 12 years and above. Vertex also published data from the Phase III RIDGELINE 105 trial demonstrating a sharp reduction in mean sweat chloride levels in patients 6 to 11 years of age. Beyond CF, Vertex is also ramping up its commercial operations for the newly approved Casgevy (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited cell therapy for sickle cell disease and transfusion-dependent beta-thalassemia. The biotech, with partner CRISPR Therapeutics, has so far activated more than 25 treatment centers globally and several patients have started cell collection. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Vertex Pharmaceuticals has kicked off 2024 by staying busy, submitting a new drug application to the FDA for its cystic fibrosis (CF) therapy, vanzacaftor triple combination, and a corresponding marketing authorization application to the European Medicines Agency. The vanzacaftor/tezacaftor/deutivacaftor combination has received Fast Track and Orphan Drug designations from the FDA for CF treatment. Vertex submitted its NDA using a priority review voucher. In CF, mutations reduce the amount and function of the CFTR protein on cell surfaces. The medications vanzacaftor and tezacaftor help increase CFTR protein levels by improving its processing and transport to the cell surface. Deutivacaftor, meanwhile, enhances the protein's function, boosting salt and water flow across cell membranes. In addition to its cystic fibrosis advancements, Vertex also recently reported a strong financial performance for the first quarter with product revenues rising to $2.69 billion, a 13% increase from the first quarter of 2023. This growth is attributed to the continued success of blockbuster Trikafta in both U.S. and international markets, especially following its approval for younger cystic fibrosis patients. The drugmaker has also initiated a rolling NDA submission for suzetrigine (VX-548) for the treatment of moderate-to-severe acute pain, expected to be completed within the quarter. Suzetrigine represents a new class of pain management solutions that could potentially offer an alternative to traditional opioids. Recently, Vertex also announced the acquisition of Alpine Immune Sciences. The deal includes povetacicept, a phase 3-ready asset for IgA nephropathy (IgAN), poised to become a leading treatment option for this chronic kidney disease.