Vertex Pharmaceuticals Incorporated ( VRTX Quick Quote VRTX - Free Report) stock has risen 36.2% this year so far against the industry’s decline of 20.6%.
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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 CF sales rose22% in the first half of 2022 primarily driven by higher sales of Trikafta (marketed as Kaftrio in Europe). Trikafta sales in 2022 are being driven by strong uptakes in international markets as well additional patients starting treatment with Trikafta, most notably pediatric patients (6-11 years of age) in the United States.
In the second half of the year, Vertex expects continued robust sales of Kaftrio/Trikafta in international markets following new reimbursements recently secured in France, Spain and Italy.
Meanwhile, Vertex is evaluating its CF 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.
While Vertex’s main focus is on the development and strengthening of its CF franchise, the company also has a rapidly advancing mid- to late-stage pipeline in six additional diseases beyond CF like acute pain, sickle cell disease, beta-thalassemia, APOL1-mediated kidney diseases (AMKD) and cell therapy for type I diabetes. Many of these candidates represent multibillion-dollar opportunities. Programs in five disease areas are entering or progressing through late-stage clinical development. Multiple late-stage projects established proof-of-concept when data were released this year.
Vertex is co-developing a gene-editing treatment, exa-cel (formerly 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 exa-cel in adult transfusion-dependent b-thalassemia in Europe and sickle cell disease in the United States are ongoing. Vertex and CRISPR Therapeutics plan to file regulatory applications for exa-cel for both indications in Europe and the United Kingdom in the fourth quarter of 2022 while discussions with the FDA are ongoing. Vertex expects exa-cel to be its next commercial launch.
In April, Vertex increased its investment in collaboration with CRISPR Therapeutics. Per the amended deal, Vertex will lead the global development and future commercialization of CTX001.
Inaxaplin (formerly VX-147) is being developed in a single pivotal phase II/III study in patients with APOL1-mediated kidney disease, or AMKD with two APOL1 mutations and proteinuric kidney disease. Enrollment is ongoing, with more than 30 sites active in the United States. In June 2022, the FDA granted Breakthrough Therapy Designation (BTD) to inaxaplin for treating APOL1-mediated focal segmental glomerulosclerosis, or FSGS based on phase II proof-of-concept data, which showed a 47.6% reduction in proteinuria.
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 2022, Vertex announced positive data from the two phase II studies on VX-548. The studies demonstrated statistically significant and clinically meaningful relief of pain and established proof of concept for VX-548. Based on this data, the company plans to advance VX-548 into pivotal phase III development for acute pain in the fourth quarter of 2022.
A phase I/II study is ongoing on VX-880, Vertex’s stem cell-derived fully differentiated islet cell replacement therapy for the treatment of type I diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia. 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. In July, the FDA lifted the clinical hold and the study resumed enrollment in the United States. Three patients have been treated in the phase I/II study to date.
Vertex’s dependence on just the CF franchise for commercial revenues is a concern. It needs a growth opportunity outside CF. Vertex’s non-CF programs carry significant risk, which is a concern.
Nonetheless, a consistent rise in CF sales, the rapid progress of non-CF pipeline candidates, minimal competition in its core CF franchise and regular business development should keep the stock afloat going forward.
Zacks Rank & Stock to Consider
Vertex currently has a Zacks Rank #3 (Hold).
A better-ranked stock in the drug/biotech sector is
Agenus ( AGEN Quick Quote AGEN - Free Report) , with a Zacks Rank #1 (Strong Buy). You can see . the complete list of today’s Zacks #1 Rank stocks here
Agenus’ loss estimates for 2022 have narrowed from a loss of 89 cents to 70 cents per share, while that for 2023 have narrowed from 64 cents per share to 60 cents per share over the past 30 days. Agenus’ stock has declined 13.3% this year so far.