Vertex, riding high on Trikafta, isn't just a rare disease company, execs say

Cystic fibrosis may be Vertex Pharmaceuticals’ commercial bread and butter, but the drugmaker shouldn’t be pigeonholed as a rare disease company, executives said on a call with analysts Thursday.

The company's strategy is more about leveraging niche markets, they explained, flagging an “enormous” opportunity posed by the company’s closest commercial prospects in blood disorders and pain management.

“We see acute pain and neuropathic pain as fully ‘Vertexian,’” CEO Reshma Kewalramani said after an analyst questioned how a pain program would fit strategically with Vertex’s current business.

As it stands, all of Vertex’s product revenues come from its stable of cystic fibrosis medicines led by relative newcomer Trikafta, which generated sales of $1.76 billion in the first quarter of 2022. Vertex’s overall product revenues for the first three months of the year clocked in at about $2.1 billion, climbing 22% over the same period in 2021.

“I think there is a bit of a misconception about us as a company that we’re a rare or an orphan [disease] company,” Stuart Arbuckle, executive vice president and chief operating officer at Vertex, added on the call. “That’s not how we define ourselves.”

“Our research strategy is to focus on specific diseases where we understand the human biology … and they’re in markets where we can access them with a specialty infrastructure,” the COO said, adding, “we think pain fits that description perfectly.”

The executives’ comments came as Vertex shined a “commercialization spotlight” on its most advanced drug prospects: CTX001, a CRISPR cas9-based gene editing therapy for transfusion-dependent thalassemia (TDT) and severe sickle cell disease, plus non-opioid acute pain candidate VX-548.

For its blood disorder gene therapy, which Vertex plans to file for approval before the end of the year, the company sees an initial multibillion dollar opportunity in roughly 32,000 patients with severe sickle cell disease and beta thalassemia, the company noted in its earnings presentation.

Marketing and launch preparations for the med are already “well underway,” Arbuckle added.

“Over the past year, we have developed a deep understanding of the sickle cell and TDT markets, including where patients with these diseases are concentrated, the physicians who would refer them for treatment and the key treatment centers that will facilitate the patient journey,” the COO said.

Moving “rapidly” toward a potential launch, Vertex has locked up key leadership positions and established teams across multiple functions, including medical, commercial and manufacturing, Arbuckle continued. Vertex is also in talks with public and commercial payers in the EU and the U.S., and it’s developing “robust patient service programs to support patients throughout the treatment journey,” he said.

Vertex’s pain prospect VX-548, for its part, is headed for a pivotal trial start in the back half of the year, the company said.

“There is a vast unmet need in the treatment of moderate to severe pain, and especially for medicines with an improved benefit-risk profile, including avoiding side effects and the addictive qualities of standard-of-care opioids,” Arbuckle said.

The first indication Vertex is targeting with VX-548 is moderate to severe acute pain. Vertex sees “this segment of the market as a specialty market that fits the Vertex commercialization model well,” Arbuckle said.

That’s not to say Vertex shows any signs of slowing down in cystic fibrosis, where its blockbuster Trikafta continues to pick up steam.

As Vertex garners more real-world data on its triple combination therapy, Trikafta continues to “[raise] the bar” for any cystic fibrosis regimens in development, Kewalramani said on the company’s earnings call. If it’s possible to outperform Trikafta, “we’re determined to be the ones who do so,” she added.

Meanwhile, Vertex is sticking to the guidance it deployed earlier this year. The company expects to generate product sales between $8.4 billion and $8.6 billion in 2022.