Sale of Cystic Fibrosis Therapy CTP-656 to Vertex Will Accelerate Its Development, Concert CEO Predicts

Concert Pharmaceuticals’ sale of its cystic fibrosis therapy CTP-656 to the larger Vertex Pharmaceuticals will lead to cystic fibrosis patients obtaining faster access to the therapy, Concert CEO Roger Tung predicted.

He also said that the $160 million that Concert obtained from the sale will allow the company to advance its hair-loss therapy alopecia areata CTP-543 into a pivotal clinical trial.

His comments came in a press release on Concert’s second-quarter earnings. Vertex acquired the worldwide rights to CTP-656 in late July.

“We are very pleased that we have closed the CTP-656 asset purchase agreement with Vertex, which resulted in a payment to Concert of $160 million in July,” Tung said. “We believe our transaction with Vertex provides the optimal pathway to rapidly advance the development of CTP-656 for the benefit of cystic fibrosis patients.”

The hair-loss disorder, alopecia areata, occurs when the immune system mistakenly attacks hair follicles, which is where hair growth begins. A pivotal clinical trial is one aimed at generating the kind of solid evidence of effectiveness that can lead to regulatory approval. It is typically a Phase 3 trial.

CTP-656 helps faulty versions of a protein called the CF transmembrane conductance regulator, or CFTR, do the job that the healthy protein was intended to do. That role makes it what scientists call a CFTR potentiator.

CFTR functions as a channel across the membrane of cells that produce mucus, sweat, saliva, tears, and digestive enzymes. Mutations of the CFTR gene, which provides instructions for making the protein, lead to the protein being unable to do its job properly. The mutations are a hallmark of CF.

Concert expects combinations of CTP-656 and another class of drug, CFTR modulators, to become future CF therapies. Modulators correct the function of the faulty version of the CFTR protein that the defective gene makes.

CTP-656’s pharmacokinetics profile is superior to that of Kalydeco (ivacaftor), an approved CFTR potentiator used to treat CF, studies have shown. Pharmacokinetics refers to the time it takes for a drug to be absorbed and distributed in the body before being excreted.

Among other pharmacokinetic performance measures, researchers said CTP-656 had a longer half-life than Kalydeco and its levels in the blood were higher than Kalydeco’s at 24 hours. Half-life is the time it takes for half of a drug to leave the body.

A Phase 2 clinical trial (NCT02971839) is under way to evaluate the effectiveness and safety of CTP-656, compared with Kalydeco, in 40 CF patients with gating mutations of the defective CFTR gene. The study is still recruiting patients. Results are expected at the year of the year.

The U.S. Food and Drug Administration has granted CTP0656 orphan drug status, a designation that gives companies incentives for developing drugs for rare diseases.