Boston Business Journal

 

 

 Health Care

 

Vertex has built a CF empire. Next up: cell and gene therapy.

 By Rowan Walrath – Life Sciences Reporter, Boston Business Journal

 

 

Vertex Pharmaceuticals Inc. is one of the largest drug companies in the U.S. As of the end of 2022, the company had nearly $10.8 billion cash on hand, fueled almost entirely by its blockbuster cystic fibrosis (CF) drug, Trikafta.

During its annual earnings report Tuesday night, the company made clear that its next big play will be in cell and gene therapy. Much of that comes down to a drug called exa-cel, short for exagamglogene autotemcel. It's a CRISPR-based gene therapy designed to treat severe sickle-cell disease and transfusion-dependent beta thalassemia (TDT).

Exa-cel could see its first approvals later this year. Vertex recently finished regulatory submissions in the U.K. and Europe, and will wrap up its U.S. application by the end of March, the company said in its earnings release Tuesday.

"Exa-cel holds the promise to be the first CRISPR-based gene-editing treatment to be approved, and represents a near-term and significant market opportunity," CEO Reshma Kewalramani said on Tuesday's earnings call. She added that are 32,000 patients in the U.S. and Europe that Vertex has identified as experiencing the "most severe" forms of both sickle-cell disease and TDT.

To date, Vertex has mainly established itself as a cystic fibrosis company, and its CF empire is certainly formidable. Its blockbuster bug, Trikafta, brought in nearly $7.7 billion in sales in 2022 — 86% of Vertex's total product revenue.

Vertex now sits on nearly $10.8 billion in cash. Executives, however, aren't sharing much about how they plan to deploy that capital long-term.

In response to an analyst question, Charlier Wagner, Vertex's executive vice president and chief financial officer, said the company had not established a long-term goal for R&D investment.

The company did, however, authorize a $3 billion share repurchase program as of Feb. 1.

Vertex has been under some pressure to use its funds to make acquisitions. Last July, it spent $320 million on ViaCyte, a cell therapy startup specializing in diabetes. That's about 3% of the cash Vertex now has.

"I probably sound like a broken record, but our priorities are the same: investing in innovation, both internal and external, is the top priority," Wagner said. "We clearly see that as the best way to create value for patients and for shareholders."

Still, exa-cel offers some clues — and so does Vertex's Boston real estate portfolio.

The company's headquarters sit at 50 Northern Ave. in Boston's Seaport District. A mile away, the company has a 400-plus-person facility called the Jeffrey Leiden Center for Cell and Genetic Therapies. About two years from now, that center will expand with the opening of a new, 344,000-square-foot, 500-person facility right across the street. It, too, will be dedicated to genetic medicines.

In comments, Kewalramani remained mostly focused on the company's near-term launches — Vertex's "five in five goals," as she calls it. Under that plan, the company would launch five new medicines in the next five years.

After exa-cel, the first, comes another CF drug, a combination of vanzacaftor, tezacaftor and deutivacaftor, which Kewalramani believes could be even better than Trikafta.

"Those are just right in front of us," Kewalramani said.

Vertex is also working to expand its portfolio in other ways. A drug called VX-548 is now in Phase 3 trials that will determine the fate of the drug when results are expected this year. It's designed to manage pain, filling what Vertex executives describe as a "gap" left by current options. That includes over-the-counter painkillers like ibuprofen and acetaminophen, which have limited efficacy and can cause stomach and liver damage over time; and opioids, which are effective but have other limitations and are notorious for their abuse potential.

Other mid- and late-stage drugs include VX-147, which is designed to treat a type of kidney disease, and VX-864, which targets alpha-1 antitrypsin deficiency, or AATD, which plays a role in lung and liver disease. Earlier-stage programs include drugs being developed in partnership with Moderna Inc. (Nasdaq: MRNA) and CRISPR Therapeutics Inc. (Nasdaq: CRSP).