Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) holds a commanding position in the realm of cystic fibrosis (CF) therapeutics. The company’s progress in CF sales is on an upward trajectory, primarily due to the success of its triple therapy, Trikafta/Kaftrio. The surge in Trikafta/Kaftrio sales can be attributed to fresh reimbursement agreements in international markets and extensions of its approved use to younger age cohorts. This trend is projected to persist throughout the latter half of the current year.
While the focus remains firmly on CF, Vertex is concurrently advancing treatments for various conditions, including sickle cell disease (SCD), beta-thalassemia (TDT), acute and neuropathic pain, APOL1-mediated kidney disease, type I diabetes, alpha-1 antitrypsin deficiency. The company also has ongoing initiatives in earlier-stage programs related to disorders like muscular dystrophies.
The current year is anticipated to bring forth numerous pivotal moments for Vertex. Over the next twelve months, significant clinical milestones are in the pipeline for both its CF and non-CF portfolios.
To date, Vertex’s stock has exhibited a commendable 21.6% increase, diverging significantly from the industry’s 12.8% decline.
Vertex is actively exploring the application of its CF medications among younger patient demographics, with the overarching goal of establishing small-molecule therapies accessible to most individuals with CF.
In the CF domain, particular attention is directed toward the triple combination consisting of vanzacaftor (a CFTR potentiator), deutivacaftor (a CFTR corrector), and tezacaftor. This triple combination regimen is being developed for CF patients aged 12 and above. Vertex has already completed patient enrollment in two pivotal phase III trials focusing on this triple combination. The novel once-daily medication carries the potential to offer improved advantages to patients compared to Trikafta. It might address CF patients who have previously discontinued Trikafta or other Vertex CF treatments, while also simplifying dosing to once daily and reducing financial obligations. The conclusion of these studies is anticipated by the conclusion of 2023, and phase III data outcomes are expected in early 2024.
Furthermore, Vertex is collaborating with Moderna (MRNA) on the development of a mRNA therapeutic named VX-522. This collaboration targets around 5,000 CF patients who do not derive benefits from the CFTR modulators developed by Vertex. VX-522 is currently undergoing a single ascending dose (SAD) clinical study, following clearance from the FDA for an investigational new drug application in December 2022. The plan is to finalize the SAD study and initiate the multiple ascending dose study in the course of 2023.
Several of Vertex’s non-CF programs signify substantial market opportunities, with six of these initiatives having progressed beyond the proof-of-concept stage.
A noteworthy achievement from Vertex’s collaboration with CRISPR Therapeutics (CRSP) is the gene-editing treatment exa-cel, targeting sickle cell disease (SCD) and beta-thalassemia (TDT). Submissions for exa-cel in SCD and TDT indications have been made to the FDA, with a priority review decision anticipated by December 8, 2023, and March 30, 2024, respectively. Similar submissions for exa-cel in the European Union and the United Kingdom are currently under evaluation. Vertex anticipates exa-cel to mark its next commercial launch, potentially providing a one-time functional cure for SCD and TDT patients, encompassing approximately 32,000 individuals.
Investor attention is notably drawn to VX-548, a potential blockbuster in the realm of pain management. VX-548, a pioneering non-opioid NaV1.8 inhibitor, is undergoing evaluation in two pivotal phase III studies focusing on acute pain, one involving bunionectomy surgery and the other abdominoplasty surgery. These pivotal trials are projected to conclude either in late 2023 or early 2024. Additionally, Vertex has commenced a phase II study of VX-548 in diabetic peripheral neuropathy, a form of peripheral neuropathic pain, with an expected completion by the end of 2023. Data stemming from these studies is foreseen to emerge in late 2023 or early 2024.
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Author Faith Yakubu 
