Vertex Pharmaceuticals has reshaped itself into a cystic fibrosis powerhouse the past few years. Today, it’s trying to add to that position by teaming up with a biotech out of Durham, NC, on some experimental drugs for the devastating disorder—and perhaps for other lung diseases as well.
Boston-based Vertex (NASDAQ: [[ticker:VRTX]]) said this afternoon it’ll collaborate with privately-held Parion Sciences to develop drugs for cystic fibrosis and other unnamed pulmonary diseases. Vertex has paid $80 million initially for rights to two so-called epithelial sodium channel (ENaC) inhibitors, named P-1037 and P-1055, which are already in mid-stage testing for cystic fibrosis. Vertex also has the option to license a third ENaC drug, for which it would pay another $230 million.
The deal includes significant downstream payments as well. Parion could get another $490 million should the ENaC drugs progress forward as CF treatments, plus another $370 million if those drugs start panning out as therapies for unspecified “non-CF pulmonary” diseases. All told, Vertex could potentially shell out nearly $1.2 billion in the partnership, not including royalties to Parion if any of the drugs hit the market.
The move is the latest step in Vertex’s makeover. The company had a brief run as a dominant player in hepatitis C, which began with the approval of telaprevir (Incivek) in 2011. That quickly ended when a new wave of superior treatments came to market, led by sofosbuvir (Sovaldi) from Gilead Sciences (NASDAQ: [[ticker:GILD]]). The company has since pivoted and rallied around its emerging CF franchise, starting with the landmark FDA approval of ivacaftor (Kalydeco) in 2012, the first drug to treat underlying molecular abnormalities present in some patients. (Previous cystic fibrosis treatments only managed symptoms.)
Ivacaftor is just one piece of the puzzle for Vertex. The drug is only approved to treat patients with genetic mutations, and they make up about 4 percent of the overall CF population. So Vertex is trying to combine the drug with others, expand its reach, and treat more patients.
The company took a big step in that direction last month, when an FDA advisory panel recommended approval of a combination pill of ivacaftor and lumacaftor. The combined therapy, known as Orkambi, could become an option for patients 12 years or older with the most common form of cystic fibrosis—two copies of the genetic mutation known as F508del—should the FDA approve the drug.
The agency is expected to make a decision by July 5. Should that happen, Vertex could have a multi-billion dollar franchise on its hands.
Even with those drugs in tow, Vertex is still trying to develop better combinations with better results and that reach more patients. The ivacaftor/lumacaftor combination led to a roughly 3 percent improvement in lung function after six months of treatment in a Phase 3 trial.
One of those plans is a triple therapy—ivacaftor, lumacaftor, and a third drug. Vertex has been trying to do this with other drugs in-house, but now it’s reaching out to Parion for help in that area. P-1037 is in a Phase 2a trial as a monotherapy, but Vertex aims to start a mid-stage study to test the drug in combination with both ivacaftor and lumacaftor in patients with two copies of the F508del mutation.
The idea here is to use complementary mechanisms and overcome genetic mutations that stymie some of Vertex’s drugs. CF causes a buildup of thick mucus in the lungs which occurs when cellular channels meant to shuttle salt and water across cell membranes don’t function properly. Ivacaftor helps prop open those channels, while lumacaftor helps transport a key protein to the surface of cells so ivacaftor can have a greater impact.
With the Parion deal, Vertex is betting that a third mechanism—ENaC inhibition—can help as well. ENaC is an ion channel that helps maintain the balance of salt and water. When it’s hyperactive, as it is in CF, the mucus of the lungs gets dehydrated and thick, and harder to clear out. Blocking ENaC is supposed to hydrate the mucus and make it easier to clear from the lungs.
“This collaboration with Parion complements our ongoing work in CF and supports our two key goals in this disease: to increase the number of people eligible for new CF medicines and to enhance the benefit of treatment,” said Jeffrey Chodakewitz, Vertex’s chief medical officer and executive vice president, in a statement. “The goal of these planned studies of P-1037 is to determine whether ENaC inhibition can improve lung function in people with CF, including those with mutations unlikely to respond to treatment with the investigational combination of lumacaftor and ivacaftor.”
