Vertex Pharmaceuticals Incorporated (VRTX Quick QuoteVRTX - Free Report) has been benefiting from strong revenue growth, regulatory approvals and commercial launches in the past year.

Vertex enjoys a dominant position in the cystic fibrosis (CF) market. Vertex’s CF sales continue to grow, driven by its triple therapy, Trikafta (marketed as Kaftrio in Europe). In the United States, Trikafta sales are being fueled by label expansions to younger age groups (two to five years old). In the ex-U.S. markets, the drug continues to witness strong uptake with recently achieved reimbursements and expanded use in young age groups. The trend is expected to continue for the rest of the year.

While CF remains the main area of focus, Vertex has seen rapid success in its non-CF pipeline candidates’ development in the past year. Vertex and partner CRISPR Therapeutics’ (CRSP Quick QuoteCRSP - Free Report) one-shot gene therapy, Casgevy, was approved for two blood disorders, sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), in multiple regions in 2023 and early 2024. These approvals have diversified its commercial opportunity.

In the past year, the stock has risen 31.2% against the industry’s 10.0% decline.

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Vertex and CRISPR Therapeutics’ Casgevy is the first-ever CRISPR/Cas9-based therapy to be approved anywhere in the world. Vertex and CRSP believe Casgevy has the potential to be a one-time functional cure for SCD and TDT patients, with an estimated patient population of approximately 35,000 across the United States and Europe.

Vertex is making rapid progress with the launch of Casgevy. Vertex has more than 25 activated authorized treatment centers or ATCs in all regions where the therapy is approved. Multiple patients have initiated cell collection. Vertex said it is also making great progress with payers. It has established contracts and policies in the U.S. commercial market. In outside U.S. markets, it is progressing for reimbursement and access, either through reimbursement agreements or early access programs. Vertex expects to record Casgevy sales from the second half of 2024.

Vertex has additional near-term launches planned. These include suzetrigine for acute pain and vanzacaftor triple for CF. Vertex is optimistic that both products can be launched this year.

For suzetrigine (formerly known as VX-548), Vertex has initiated a rolling new drug application (NDA) submission process across a broad label in moderate-to-severe acute pain and is on track to complete the NDA submission in the second quarter. Vertex plans to initiate a pivotal phase III program of suzetrigine in diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain caused by damage to nerves, in the second half of 2024.

Data from the phase II study on suzetrigine in DPN demonstrated a promising safety and efficacy profile of suzetrigine with consistent efficacy seen across all doses studied. Vertex has also initiated a phase II study of VX-548 in patients with painful lumbosacral radiculopathy, another form of peripheral neuropathic pain.  Vertex believes suzetrigine has the potential to transform the treatment paradigm of pain, both acute and neuropathic. Pain is an area with limited treatment options, mostly highly addictive opioid-based medications.

VRTX filed regulatory applications for vanza triple for treating people with CF aged six years and older in the United States (with priority voucher) and EU in mid-2024. Vanza triple is a combination of vanzacaftor, a CFTR potentiator, deutivacaftor, a CFTR corrector and tezacaftor. This new once-a-day oral combination medicine has the potential for enhanced patient benefit than Trikafta patients and can potentially treat CF patients who have discontinued Trikafta or other Vertex CF medicines. It can also improve dosing (once daily) and lower the royalty burden.

Vertex has a rapidly advancing mid- to late-stage pipeline in other disease areas like APOL1-mediated kidney diseases (AMKD), alpha-1 antitrypsin (AAT) deficiency and cell therapy for type I diabetes. Many of these candidates represent multibillion-dollar opportunities. This year is expected to be a catalyst-rich year for Vertex. Several important clinical milestones are expected over the next 12 months in its CF and non-CF portfolio.

Importantly, in April, Vertex announced a $4.9 billion definitive agreement to acquire Alpine Immune Sciences which looks like a strategic fit for Vertex. The acquisition will add Alpine’s lead pipeline candidate, povetacicept, to Vertex’s pipeline. Povetacicept is designed to target two proteins, namely BAFF and APRIL, which are jointly responsible for the cause of multiple serious autoimmune diseases. This drug is set to enter into late-stage development for the treatment of IgA nephropathy (IgAN) in the second half of 2024. Vertex believes povetacicept holds a “pipeline in a product” potential in a number of other serious autoimmune renal diseases and cytopenias in phase II development.

Zacks Rank and Stock to Consider

Vertex currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Vertex Pharmaceuticals Incorporated Price and Consensus

Vertex Pharmaceuticals Incorporated price-consensus-chart | Vertex Pharmaceuticals Incorporated Quote

A better-ranked biotech stock is Ligand Pharmaceuticals , carrying a Zacks Rank #2 (Buy).

In the past 90 days, the Zacks Consensus Estimate for Ligand’s 2024 earnings per share has increased from $4.42 to $4.56. During the same time frame, the consensus estimate for Ligand’s 2025 earnings per share has increased from $5.11 to $5.27. Year to date, shares of LGND have gained 21.3%.

Ligand beat estimates in each of the trailing four quarters, delivering an average surprise of 56.02%.