Vertex Made Its Fortune Pushing a Rare Disease Toward Extinction. Here’s Its Next Act. – The Average Joe
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    Vertex Made Its Fortune Pushing a Rare Disease Toward Extinction. Here’s Its Next Act.

    Noah Weidner

    January 30, 2024

    There’s more to pharma than just COVID vaccines and weight-loss medications. Thirty-five years ago, Vertex Pharmaceuticals (NASDAQ:VRTX) took a fresh approach to treating illnesses by targeting human cells and genes. Except it spent 20 years with bleak growth prospects before scoring its first blockbuster drug approval in 2012.

    Since then, Vertex has generated over $20B in profits attacking cystic fibrosis at the source. And last year, it became the first company to bring a gene-editing therapy to market, a generational moment for the biopharma industry that pushed Vertex into the top 10 US pharma companies.

    We feel heal your pain: Yesterday, Vertex announced positive clinical trial results from its experimental non-addictive alternative to opioid painkillers, VX-548, which blocks pain signals from reaching the brain. This opens the door for the “first new acute pain medicine in 25 years,” Vertex Chief Scientific Officer Dr. David Altshuler tells Barron’s.

    • While VX-548 might not match the effectiveness of Vicodin (opioid med), analysts predict it could replace addictive opioids for cases of “moderate-to-severe pain” once approved in early 2025.
    • The drug could cater to over 80M patients experiencing acute pain, a vast market that analysts at Leerink Partners forecast will help VX-548 generate $5.1B in sales by 2030.

    Vertex’s next act

    Altogether, Vertex has over a dozen treatments in research or studies — a strategy deemed “more important than ever” by RBC Capital Markets analyst Brian Abrahams, as it will drive the next leg of the company’s growth.

    • Vertex plans to expand its pain and blood disorder franchises but boasts a pipeline of gene-editing treatments with CRISPR Therapeutics (NASDAQ:CRSP), which could eventually treat Muscular Dystrophy and Type 1 Diabetes.
    • Focusing on rare diseases has also allowed Vertex to skip some costly drug filing fees and secure fast-track approval — all while charging hefty premiums for its orphan drugs.

    Forward-looking: Unlike many other pharma giants facing patent expirations, Vertex doesn’t have any of its major drug patents reaching a patent cliff anytime soon, leaving the company ample time to commercialize its new cures.

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