Despite the success of COVID-19 vaccines and medicines, the biotech sector underperformed the broader market in 2021. However, the sector is expected to pick up in 2022 due to its strong fundamentals.
In this defensive sector,
Vertex Pharmaceuticals Incorporated ( VRTX Quick Quote VRTX - Free Report) is a good stock to own.
There are several reasons to own the stock.
Good Rank and Rising Estimates: Vertex has a Zacks Rank #2 (Buy). You can see . the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here
Vertex’s stock has declined 2.5% in the past year compared with the
industry’s decline of 37%. The Zacks Consensus Estimate for 2022 has gone up from $13.32 to $13.39 over the past 60 days.
Image Source: Zacks Investment Research
Strong CF Portfolio: Vertex’s main area of focus is cystic fibrosis (CF). The CF market represents a huge commercial potential. With its four CF medicines, Vertex is treating around half of the 83,000 patients living with CF in the United States, Europe, Canada, and Australia. Meanwhile, the company is making progress toward reaching 30,000 additional patients in these regions through label expansions of its medicines.
Trikafta’s (triple combo CF medicine) early approval and launch in 2019 in the United States was the most significant milestone for Vertex. In the EU, Kaftrio was approved in August 2020. New reimbursement agreements in ex-U.S. markets and label expansions in younger age groups in the United States are driving Trikafta/Kaftrio sales higher.
Trikafta/Kaftrio is crucial for Vertex’s long-term growth as it has the potential to treat up to 90% of CF patients. Meanwhile, 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.
Upside Potential from Non-CF Pipeline: While Vertex’s main focus is on the development and strengthening of its CF franchise, the company also has a rapidly advancing early-stage portfolio in five additional diseases beyond CF like pain, sickle cell disease, beta-thalassemia, APOL1-mediated kidney diseases and cell therapy for type I diabetes. Data from multiple programs are expected in the next six to nine months.
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. Preliminary safety and efficacy data from the studies were positive. Vertex and CRISPR Therapeutics plan to file regulatory applications for CTX001 for both indications by 2022 end.
In April, Vertex increased its investment in the collaboration with CRISPR Therapeutics. Under the amended deal, Vertex will lead the global development and future commercialization of CTX001.
Enrollment is complete in a phase II study on VX-147, its first oral small molecule medicine in APOL1-mediated focal segmental glomerulosclerosis (FSGS). Data from the study showed that VX-147 caused a reduction in proteinuria in patients with APOL1-mediated kidney disease. Vertex plans to push VX-147 into pivotal studies for APOL1-mediated kidney disease, including FSGS, in the first quarter of 2022.
VX-548, a selective NaV1.8 inhibitor, is being evaluated in two phase II acute pain studies, one following bunionectomy surgery and the other following abdominoplasty surgery. Data from the acute pain studies are expected in the first quarter of 2022.
A phase I/II study was initiated on VX-880 for the treatment of type I diabetes (T1D) in March. Data from the phase I/II study for the first T1D patient announced in October 2021 demonstrated that after 90 days of the single infusion of VX-880 at half the target dose along with immunosuppressive therapy, substantial improvements across multiple measures of islet cell function were observed that were rapid, robust, and durable through Day 90. 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.
Collaborations Broadening Pipeline: Vertex’s success in CF has given it the financial strength to invest in both internal and external innovation. Vertex has entered into multiple agreements in the past few years to provide it with access to new external scientific technologies, programs and expertise in multiple diseases to complement its internal pipeline. It has such collaborations with CRISPR Therapeutics, Moderna, Obsidian Therapeutics, Arbor Biotechnologies, and Kymera Therapeutics, among others. Vertex plans to pursue more business development transactions to bolster its pipeline for serious diseases with multiple modalities and technologies. Conclusion
Vertex’s dependence on just the CF franchise for commercial revenues is a concern. It needs a growth opportunity outside CF with recent disappointment for the alpha-1 antitrypsin deficiency candidate. Vertex’s two candidates for alpha-1 antitrypsin deficiency (AATD), VX-814 and VX-814, have failed.
Nonetheless, Vertex’s CF sales continue to grow despite the impact of the pandemic. 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.
Stock to Consider
A large biotech stock worth considering is
BioNTech ( BNTX Quick Quote BNTX - Free Report) which has a Zacks Rank of 1.
BioNTech’s stock has surged 64.0% in the past year. Estimates for BioNTech’s 2022 earnings have gone up from $31.14 to $32.52 over the past 60 days.
BioNTech topped earnings estimates in each of the last four quarters. BioNTech has a four-quarter earnings surprise of 132.44%, on average.