Report
Karen Andersen
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Morningstar | Vertex's Cystic Fibrosis Franchise Fortifies Narrow Moat; Maintaining FVE After Solid 2Q

Vertex Pharmaceuticals' portfolio for cystic fibrosis treatment remains strong; it posted 46% total revenue growth in the second quarter, driven by next-generation molecule Symdeko, which was approved in February. Management raised its full-year revenue guidance to a midpoint of $2.95 billion from a midpoint of $2.73 billion, thanks to the better-than-anticipated adoption of the new combination regimen. After adjusting for this rapid uptake in our model, we do not expect a material change to our fair value estimate of $183 per share, and we continue to believe that the firm's position in cystic fibrosis warrants a narrow economic moat and positive moat trend.

Although the successful launch of Symdeko is cannibalizing some Orkambi sales as expected, Symdeko expands the eligible treatment population, capturing patients who previously discontinued treatment by Orkambi as well as patients naive to Vertex combinations. We expect the firm to further stretch the eligible population with new corrector molecules, VX-445 and VX-659, which we anticipate launching in the second half of 2019, if approved. Furthermore, results for Food and Drug Administration approval of Kalydeco and Orkambi in younger patients are anticipated in August. While the reimbursement environment remains tough with some non-U.S. payers (such as those in the United Kingdom, France, and Australia) due to the expensive price tag of Vertex treatments, we believe the firm will successfully negotiate reimbursement for the rare disease, leaning on the superior efficacy of the treatment regimen and the lack of alternative treatments. While its cystic fibrosis portfolio steals the spotlight, Vertex is well positioned to diversify with pipeline therapies in pain and partnership with CRISPR Therapeutics for sickle cell disease, in addition to a healthy accumulation of cash for worthy acquisitions.
Underlying
Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals is focused on developing and commercializing therapies for the treatment of cystic fibrosis (CF) and developing research and development programs in other indications. The company's marketed products are TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO/SYMKEVI (tezacaftor in combination with ivacaftor), ORKAMBI (lumacaftor in combination with ivacaftor) and KALYDECO (ivacaftor), which are approved to treat patients with CF who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene. The company is evaluating CTX001, a genetic therapy as a potential treatment for sickle cell disease and beta-thalassemia.

Provider
Morningstar
Morningstar

Morningstar, Inc. is a leading provider of independent investment research in North America, Europe, Australia, and Asia. The company offer an extensive line of products and services for individual investors, financial advisors, asset managers, and retirement plan providers and sponsors.

Morningstar provides data on approximately 530,000 investment offerings, including stocks, mutual funds, and similar vehicles, along with real-time global market data on more than 18 million equities, indexes, futures, options, commodities, and precious metals, in addition to foreign exchange and Treasury markets. Morningstar also offers investment management services through its investment advisory subsidiaries and had approximately $185 billion in assets under advisement and management as of June 30, 2016.

We have operations in 27 countries.

Analysts
Karen Andersen

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