Vertex Pharmaceuticals agreed a potentially $250 million deal to acquire global rights to Concert Pharmaceuticals’ mid-stage cystic fibrosis (CF) therapy candidate CTP-656. Under terms of the proposed deal, Vertex would pay Concert $160 million upfront and potentially another $90 million in milestones, dependent on regulatory approval of CTP-656 in the U.S. and reimbursement in the U.K., Germany, or France. Vertex would also acquire rights to Concert’s other CF research and preclinical programs. The proposed terms have been approved by Concert’s board but are still subject to approval by the firm’s shareholders,

Concert has used its DCE® deuterium chemistry platform to develop the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator CTP-656 as a deuterated form of Vertex’s existing CF therapy, Kalydeco® (ivacaftor), which is approved in the U.S., Europe, Canada, and Australia for treating CF patients with specific CFTR gene mutations. Concert claims CTP-656 could potentially be used as part of once-daily combination CFTR modulator therapies.

“Our vision is to develop the most effective and convenient medicines for people with CF,” said Jeffrey Chodakewitz, M.D., evp and CMO at Vertex. “We look forward to exploring once-daily regimens that combine CTP-656 with other potential medicines from our broad CF pipeline that treat the underlying cause of the disease.”

Vertex’s approved CF therapies include Kalydeco and Orkambi® (lumacaftor/ivacaftor). Clinical development of Orkambi in children from aged 2 years and over is ongoing, and the firm has prevously said it expects to file a marketing approval application for the use of Orkambi in CF patients aged 6 to 11 years during the first half of 2017.

Vertex reported total CF product revenues of $1.68 billion in 2016, compared with $982.3 million for 2015. Net product revenues from sales of Orkambi were $979.6 million in 2016, compared with $350.7 million in 2015. Orkambi was launched in the U.S. in July 2015. Net product revenues from Kalydeco were $703.4 million in 2016, up from $631.7 million in 2015.

Vertex has five CF candidates in its clinical pipeline. An NDA filing for lead candidate tezacaftor (VX-661) is projected for later in 2017, subject to results from Phase III studies, which are expected during the first half of the year. A Phase III trial with tezacaftor in children aged 6 to 11 years is also ongoing.

In October 2016, Vertex expanded its long-term collaboration with Cystic Fibrosis Foundation Therapeutics, and in mid-2016 announced a potentially $315 million deal with Moderna, to develop CF treatments based on Moderna’s messenger Ribonucleic Acid (mRNA) Therapeutics™. In January 2017, Vertex reported the acquisition by Merck KGaA of two clinical, and two preclinical anticancer programs for $230 million upfront.

Concert says the Vertex deal would give it the financial resources to fully fund the development of its proprietary candidate CTP-543 into pivotal clinical trials for treating alopecia areata. CTP-543 is an oral JAK1/JAK2 inhibitor that Concert has developed using its deuterium chemistry as a modified form of Incyte’s Jakafi® (ruxolitinib). Jakafi is approved in the U.S. for treating myelofibrosis and polycthemia vera, and in June 2016 received breakthrough designation by FDA for treating acute graft-versus-host disease (GvHD). Concert says it projects starting a Phase IIa study with CTP-543 for the alopecia areata indication during the first quarter of 2017.

The firm has ongoing collaborations with Jazz Pharmaceuticals for development of the clinical candidate JZP-386 (deuterated sodium oxybate) as a treatment for narcolepsy and with Celgene for the development of CTP-730 (deuterated apremilast) for treating inflammatory diseases. Deuterated dextromethorphan (AVP-786) is being developed in partnership with Teva (previously Avanir), and is in Phase III development for the indication of Alzheimer’s disease agitation and in Phase II development for multiple neurologic/psychiatric indications.








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