Vertex Pharmaceuticals Incorporated ( VRTX Quick Quote VRTX - Free Report) stock has risen 19.2% this year so far against the industry’s decline of 25%. Image Source: Zacks Investment Research
Vertex’s main area of focus is cystic fibrosis (CF). With its four CF medicines, Vertex is treating the majority of the 83,000 patients living with CF in the United States, Europe, Canada and Australia.
Vertex’s cystic franchise sales continue to grow driven by its triple therapy, Trikafta/Kaftrio. New reimbursement agreements in ex-U.S. markets and label expansions to younger age groups in the United States are driving Trikafta/Kaftrio sales higher.
Vertex is evaluating its medicines in younger patient populations and aims to have small molecule treatments for approximately 90% of people with CF. Additionally, Vertex is pursuing genetic therapies to address the remaining 10% of CF patients, including an mRNA approach in partnership with
Moderna ( MRNA Quick Quote MRNA - Free Report) . Vertex and Moderna plan to begin clinical development of their CFTR mRNA therapy in 2022/2023.
Vertex is also evaluating a triple combination of VX-561, a CFTR potentiator, VX-121, a CFTR corrector, and tezacaftor in two pivotal phase III studies. This new once-a-day combination medicine has the potential for enhanced patient benefit than Trikafta. Enrollment in the studies is expected to be completed in late 2022 or early 2023.
Meanwhile, Vertex has a broad clinical non-CF pipeline across six disease areas like pain, sickle cell disease, beta-thalassemia, APOL1-mediated kidney diseases (AMKD) and cell therapy for type I diabetes. Clinical studies on these candidates are progressing rapidly with impressive data presented from multiple programs this year that established proof-of-concept.
Vertex is co-developing a gene-editing treatment, CTX001 in partnership with
CRISPR Therapeutics ( CRSP Quick Quote CRSP - Free Report) in two devastating diseases — sickle cell disease and thalassemia. Phase I/II studies of CTX001 in adult transfusion-dependent b-thalassemia in Europe and sickle cell disease in the United States are ongoing. The preliminary safety and efficacy data from the studies were positive.
Vertex and CRISPR Therapeutics plan to file regulatory applications for CTX001 for both indications in late 2022. Vertex expects CTX001 to be its next commercial launch.
In March, Vertex initiated the pivotal development of VX-147 in a single phase II/III study in patients with AMKD with two APOL1 mutations and proteinuric kidney disease. In patients with APOL1-mediated focal segmental glomerulosclerosis, or FSGS, a particular kind of AMKD, treatment with VX-147 led to a 47.6% reduction in proteinuria compared to baseline in a phase II study.
VX-548, a novel first-in-class, non-opioid NaV1.8 inhibitor, is being evaluated in two phase II acute pain studies, one following bunionectomy surgery and the other following abdominoplasty surgery. In March, Vertex announced positive data from the two phase II studies on VX-548. The studies met their primary endpoint and established proof of concept for VX-548. Based on this data, the company plans to advance VX-548 into pivotal development for acute pain in the second half of 2022.
A phase I/II study is ongoing on VX-880, Vertex’s stem cell-derived fully differentiated islet cell replacement therapy that could offer a functional cure for people living with type I diabetes. While the program was placed on a clinical hold by the FDA in May based on insufficient information for dose escalation, data available from the two patients treated at half-dose demonstrated proof of concept. VX-880 is Vertex’s first of the two investigational programs for the transplant of functional islets into patients. VX-880 is for the transplantation of islet cells alone, using immunosuppression to protect the implanted cells. The second program will involve the implantation of the islet cells inside an immunoprotective device. Clinical development on the second program is expected to begin in 2022.
Vertex faces minimal competition in its core CF franchise.
AbbVie ( ABBV Quick Quote ABBV - Free Report) completed an interim analysis of a phase II proof-of-concept study on its triple combination CF therapy. However, the interim efficacy results did not meet the pre-specified criteria for advancing this triple therapy in development.
The failure of AbbVie’s CF therapy has removed a significant overhang on Vertex’s shares regarding competitive data.
However, Vertex’s dependence on just the CF franchise for commercial revenues is a concern. Vertex’s non-CF programs carry significant risk, which is a concern.
Overall, a consistent rise in CF sales, the rapid progress of the non-CF pipeline candidates, minimal competition in its core CF franchise, and regular business development should keep the stock afloat going forward.
Vertex currently has a Zacks Rank #3 (Hold). You can see
. the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here