(Reuters) – Corbus Pharmaceuticals Holdings Inc’s experimental drug for cystic fibrosis, a rare lung-scarring disease, could be a real money spinner and pose a challenge to rival Vertex Pharmaceuticals Inc’s successful treatment, Kalydeco.

Corbus, a tiny biopharmaceutical company, received $5 million from the Cystic Fibrosis Foundation last month to develop the drug, Resunab, and expects to begin a mid-stage study this quarter.

The company hopes Resunab will treat every patient suffering from the disease, compared with Kalydeco, which has approval to treat a small section of patients. The disease currently affects 70,000 people worldwide.

Kalydeco treats certain underlying genetic mutations behind cystic fibrosis (CF), while Resunab aims to manage the inflammation the disease causes.

“We’re not focusing on a particular subset of patients, like Vertex’s drug,” Chief Executive Yuval Cohen said.

Kalydeco, which was granted fast-track status by the U.S. Food and Drug Administration, received approval in 2012, six years after the company started an early stage trial.

Kalydeco, which had sales of about $464 million in 2014, is being tested in combination with other drugs to expand its use.

It is expected to generate peak sales of $1.1 billion in 2020, according to Thomson Reuters Cortellis.

Resunab could bring in more than $2 billion in U.S. sales treating CF patients, Zacks Investment Research analyst Jason Napodano said.

Still, Corbus has a long way to go.

“It’s too soon to know how significant Resunab will be …,” said a CF Foundation spokeswoman.

CF is caused by a missing or defective gene that makes the body produce a thick, sticky mucus resulting in inflammation and bacterial infections.

This then causes “abnormal inflammation,” which leads to fibrosis, and, eventually, death.

“Theoretically, we’re targeting all CF patients, because the one thing that all these mutations share is that every single one of them exhibits abnormal inflammation,” Cohen said.

Cohen had helped develop five anti-inflammatory drug candidates as co-founder of Celsus Therapeutics Plc.

Since Resunab aims to bring down the abnormal inflammation by mimicking a natural process, Cohen expects it will have a “good safety profile”. Existing anti-inflammatory drugs are typically associated with serious side-effects and suppress the immune system.

Resunab is also being tested to treat scleroderma, an inflammatory skin disorder. A scleroderma approval could add another $2 billion in peak sales, Zacks’ Napodano said.

Shares of the company, which has a market value of about $80 million, traded over the counter before listing on the Nasdaq in April.


(Editing by Sayantani Ghosh and Sriraj Kalluvila)

Source: Reuters