Vertex, Concert orchestrate $250M phase 2 CF drug deal

Concert posted data showing its modified version of Kalydeco could outdo its reference med in a small test back in 2015.

Vertex could pay out as much as $250 million to gain full access to Concert Pharmaceuticals’ midstage cystic fibrosis (CF) candidate CTP-656, a tweaked version of its own Kalydeco, as it eyes combo uses that target the underlying causes of the fatal disease while taking over a potential competitor product.  

The deal sees Vertex, which is looking to build up its marketed CF franchise, pay $160 million in cash with $90 million in biobucks also lined up for the future.

The pharma gets CTP-656 for its moneyan investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that the company hopes it can use as part of a once-daily combo of CFTR modulators that treat the underlying cause of CF.

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This will dovetail with its CF meds: Kalydeco (ivacaftor) and more recently its combo therapy Orkambi (lumacaftor/ivacaftor).

Kalydeco is suitable for CF patients with the Gly551Asp mutation in the CFTR generepresenting just 4% to 5% of the total patient populationbut adding lumacaftor means that it can be prescribed for about 45% of patients. The second ingredient targets those carrying two copies of the much more common F508del mutation.

CTP-656 was in fact developed by Concert through the use of its deuterium chemistry to modify ivacaftor as it looked to make a better version of the drug. The big idea at Concert is taking approved or previously studied drugs and reformulating them with deuterium to boost their safety, tolerability and efficacy, creating new, and patentable, products in the process.

Back in 2015, the biotech posted data showing its treatment lasted longer than Vertex’ med in a small, early-stage trial. Clearly, Vertex wants to get its hands fully on this boosted version of its drug.

“With Vertex’s clinical and commercial expertise in CF, this agreement provides the optimal pathway to rapidly advance the development of CTP-656 for the benefit of cystic fibrosis patients,” said Roger Tung, president and CEO of Concert Pharmaceuticals.

“The financial strength provided to Concert by this agreement will allow us to advance [autoimmune med] CTP-543 into pivotal testing and broaden our proprietary development pipeline.”

Concert is currently conducting a phase 2 study of CTP-656 in people with CF who have gating mutations. As part of the agreement, Vertex gains all rights to Concert’s other CF research and preclinical programs.

Analysts at Leerink said they saw the deal as a positive for Vertex, saying in a note: "First, most obviously it removes a potential competitor, and, more importantly, a potential combination partner for other entrants who may possess a CFTR corrector. It signals Vertex's willingness to enhance and improve its existing franchise and portfolio in CF, rather than fret over "not-invented-here" considerations.

"Second, it offers a potentially improved profile for future multi-drug CF combinations. Third, it can extend the duration of Vertex's CF combinations even longer, given likely composition-of-matter exclusivity for Kalydeco of 2027 in the U.S. and 2025 in Europe, compared to 2032-plus in the U.S. and Europe for CTP-656.

"CTP-656 was one of numerous CF competitors in early clinical trials that have been advanced in parallel with Vertex’s race to concoct a triple-therapy regimen for the majority of CF patients, and today’s news signals, in our view, the willingness of Vertex to defend its dominant and valuable position in the market category."

Concert rocketed up more than 77% on the news while Vertex edged down 0.5%.