Vertex Pharmaceuticals is best known for its cystic fibrosis franchise. But over the past several years Vertex has been making inroads elsewhere, and a deal it is announcing this morning continues that strategy.
Vertex (NASDAQ: VRTX), of Boston, will pay privately held startup Kymera Therapeutics $70 million to form an alliance to develop a group of drugs for “serious specialty diseases.” Vertex won’t say which ones, but beyond cystic fibrosis (CF), where it has three FDA-approved drugs that generated $3 billion in sales last year, the company has largely been focusing on rare diseases that are caused by easily identifiable genetic mutations.
The $70 million includes an equity investment in Cambridge, MA-based Kymera, and Vertex could significantly add to the payout if it chooses to license drugs from the collaboration and they progress forward. All told, the deal includes another $1 billion in potential downstream payments for Kymera, though such “bio bucks,” as they are known, may or may not materialize.
The deal gives Vertex an entry to the competitive field of “protein degradation,” a way of using drugs to make the cell’s internal garbage disposal system, the proteasome, get rid of harmful proteins. The method is a way for drugmakers to use tools they know well—small-molecule, chemical drugs—to go after disease-causing proteins they previously couldn’t touch. In addition to Kymera, a slew of biotechs are using protein degradation to develop drugs, among them Cedilla Therapeutics, C4 Therapeutics, Nurix, and Arvinas. Vertex aims to explore the potential of protein degradation “across multiple disease areas,” says spokesperson Heather Nichols, though she wouldn’t be more specific. Kymera has been focusing on cancer and autoimmune and inflammatory diseases, and it already has a partnership with GlaxoSmithKline (NYSE: GSK).
Vertex saw its once-promising hepatitis C drug franchise quickly wiped out in the face of competition, so it has been deploying a multi-pronged strategy to both defend its turf in CF and diversify. For instance, a few experimental in-house drugs for pain and the rare genetic disease alpha-1 antitrypsin (AAT) deficiency, which causes liver and lung damage, are in human testing. So is a gene editing treatment for sickle cell disease and beta thalassemia that the company is developing with the help of CRISPR Therapeutics (NASDAQ: CRSP).
Vertex is trying to focus on diseases where it has a good understanding of the biology underlying the disease, and hoping that a strong signal from small, early studies will lead to “rapid development timelines,” CEO Jeff Leiden said on a conference call last month. Vertex is trying to recreate its approach in CF, where it has developed multiple drugs for the disease, some of which overlap. It has several drug candidates for AAT deficiency and focal segmental glomerulosclerosis, a debilitating kidney disorder, in development.
“You won’t see us take rifle shots in these diseases,” Leiden (pictured above) said on the April conference call. “We’ll take multiple molecules into the clinic, which I think is a very important way to reduce risk.”
As part of that effort, the company has formed alliances over the past few years with Arbor Biotechnologies, Merck KGaA, Genomics, X-Chem, and now with Kymera. Here’s more on Vertex and its strategy in CF and beyond.