Jeffrey Leiden, who in seven years has taken Vertex Pharmaceuticals to the cusp of developing treatments for about 90% of people with cystic fibrosis (CF), is exchanging his day-to-day leadership role as CEO for an elder statesman position.
Leiden — who finished high school at 15, earned a PhD in virology from the University of Chicago at 21, and a medical degree there at 23 — will become executive chairman of Vertex’s board effective April 1, 2020.
Reshma Kewalramani, a physician and scientist who left Amgen to become Vertex’s chief medical officer in 2017, will succeed Leiden. Analysts say Kewalramani — one of the first women to run a major biotech firm — is unlikely to change the direction of Vertex, whose CF therapies now generate $3 billion in annual revenues.
“Jeff’s leadership has transformed Vertex,” Bruce I. Sachs, lead independent director of the company’s board, said in a press release. “Vertex has never been stronger, and is well positioned for the future.”
Two years ago, Leiden said his major goals were to maintain Vertex’s dominance in the CF market, develop treatments for other diseases, and strengthen the company’s financial picture. By all accounts, he has succeeded.
Vertex has approved therapies for about 40% of all CF patients, and recently submitted a request for U.S. approval of a triple-combination that potentially extends that reach to 90%. Triple combinations include “next generation” correctors being developed for people whose underlying mutations don’t respond or respond well to the company’s existing CF therapies.
Although Vertex faces rivals in the CF market, it’s way ahead of them, analysts say.
Vertex had $2 billion in cash at the beginning of 2018 for projects and acquisitions, according to Leiden. This allowed it to buy Exonics Therapeutics — which develops therapies for Duchenne muscular dystrophy using CRISPR/Cas9 gene editing technology — for $245 million in June 2019.
Vertex expects to earn $3.5 billion in revenue this year.
Leiden, who was an executive at Abbott Laboratories and other biotech firms before becoming Vertex’s CEO in 2012, quickly brought the Boston-based company back from the abyss.
At the time, Vertex had been making more than $1 billion in annual revenue from its first hit treatment — Incevik (telaprevir) for hepatitis C. But competitors introduced better therapies, and Incevik sales tanked to the point that Vertex abandoned the hepatitis C market in 2014.
In timing that couldn’t have been better, a CF treatment that Vertex was developing won U.S. Food and Drug Administration (FDA) approval in January 2012, just as Incevik sales were plunging. Sales of Kalydeco (ivacaftor) — the first therapy to address one of the genetic malfunctions that cause CF — skyrocketed, replacing much of the lost Incevik revenue.
Vertex got into CF research in the late 1990s, when the Cystic Fibrosis Foundation persuaded the company’s predecessor, Aurora Biosciences, to join it in a treatment development partnership. Over the ensuing two decades, the foundation invested more than $225 million in the joint research effort.
The partnership has produced three CF treatments, with a fourth on the way. Kalydeco covers a genetic malfunction that affects about 3% of patients. The other approved Vertex treatments — Orkambi (ivacaftor/lumacaftor) and Symdeko (tezacaftor/ivacaftor and ivacaftor) — address malfunctions that affect 40%.
The FDA approved Orkambi in 2015, and Symdeko in 2018. On July 22, three days before Vertex announced Leiden’s reassignment, it asked the FDA to approve a triple-combo CF treatment. Vertex based its submission on two Phase 3 clinical trials showing that its therapeutic combo — elexacaftor (VX-445) plus tezacaftor, and ivacaftor (Kalydeco) — resulted in significant improvements in patients’ lung function.
The new combo chosen treats those with one F508del and one minimal function mutation or two F508del mutations, and could extend Vertex therapies to about 90% of all CF patients, Leiden has said.