Vertex's Success Spurs Startups Corbus and Pulmatrix to Enter Cystic Fibrosis Market

August 18, 2015
By Alex Keown, BioSpace.com Breaking News Staff

BOSTON – Vertex Pharmaceuticals ’ dominance of the cystic fibrosis medication market is being challenged by two startup companies working on their own treatments for the pulmonary disease.

Vertex has dominated the cystic fibrosis market with its blockbuster drugs Kalydeco and Orkambi, which was approved by the U.S. Food and Drug Administration (FDA) in July. Kalydeco was approved in 2012 and is aimed at a “select few” of the genetic mutations that can cause cystic fibrosis. Orkambi is the combination of Kalydeco and lumacaftor, which will be used to treat patients with the F508del mutation of cystic fibrosis, a mutation the lead drug cannot treat on its own. The F508del mutation is the most common genetic mutation in cystic fibrosis. Analysts predict Orkambi will push the company to profitability, in large part because of the reach the drug is expected to have to cystic fibrosis patients. Kalydeco is expected to reach about 3,900 patients by the end of this year, while Orkambi is expected to be able to treat more than 20,500 patients in the United States and the European Union, if European regulators approve the drug. European regulators are expected to rule on the drug later this year. Orkambi’s reach could be increased if the drug is further approved for younger patients. If that occurs, it’s estimated Orkambi could reach another 5,000 patients.

But while those drugs are dominating the market, other companies are wedging their toes in the door, spurred on by the success of Vertex, the Boston Business Journal reported this morning. The drugs being developed by Corbus Pharmaceuticals Holdings, Inc. and Pulmatrix Inc. could work in combination with Vertex’ drugs, as well as potentially have a broader reach than Orkambi and Kalydeco, the Journal said.

Norwood, Mass.-based Corbus Pharmaceuticals Holdings Inc.’s lead drug candidate Resunab was granted Orphan Drug Designation for the treatment of systemic sclerosis by the FDA in June. Resunab, synthetic oral drug that is a preferential agonist to the CB2 receptor expressed on activated immune cells, is set to begin Phase II clinical trials soon. Systemic sclerosis affects around 70,000 people in the United States. About four or five of every ten patients in the Unites States with systemic sclerosis die within 10 years of disease onset, usually from cardiopulmonary disease such as restrictive (fibrotic) lung disease, pulmonary hypertension or sudden death.

Like Corbus, Lexington, Mass.-based Pulmatrix is working on its own treatment for cystic fibrosis. The company is developing PUR1900, an inhaled anti-infective to treat fungal infections associated with cystic fibrosis. PUR1900 is a dry powder anti-fungal compound that can be administered at high therapeutic dose to the lung while minimizing systemic side effects. Robert Clarke, chief executive officer of Pulmatrix, told the Journal infections caused by fungus are “treated with high doses of medicine that have serious gastrointestinal side effects.” He said the inhaled medication goes straight into the lungs, which means the dosage can be lowered so as not to cause any side effects, but still kill off the infection.

But, while Corbus and Pulmatrix are entering the cystic fibrosis market, Vertex is not sitting idly by. The company is continuing to develop other cystic fibrosis drugs for its pipeline, including VX-661. In May the company reported the benefits to lung function in the 39-patient trial seemed to decline slightly over time.