Clinical stage Concert Pharmaceuticals Inc. has initiated a phase 1 clinical study to evaluate a deuterium-modified version of ivacaftor, as a potential therapy for cystic fibrosis (CF). The company is planning on studying the drug that is commercialized under the name Kalydeco in order to improve it and offer better treatment options for patients with this debilitating, chronic disease.
Concert will compare two proprietary deuterium-modified compounds in the phase 1 trial, which is a crossover study designed to choose the most promising compound for future clinical trials. In addition, the phase 1 study will also include single- and multiple-ascending doses to enable the assessment of both safety and pharmacokinetics of the candidate drug, as announced in a press release.
“This new program exemplifies Concert’s platform capability of advancing deuterium-modified versions of marketed or late-stage development compounds into Phase 1 testing in an expedited manner,” stated the president and CEO of Concert Pharmaceuticals, Roger Tung, PhD. “Our goal with this Phase 1 evaluation is to efficiently evaluate the early clinical properties of two novel deuterium-modified analogs of ivacaftor in order to select the one with the best potential to benefit patients.”
“Treatments for cystic fibrosis are evolving rapidly with multiple exploratory agents and mechanisms for CFTR modulation that we believe may be beneficial and we are excited to develop our potentiator both as a single agent and to enable new combinations to expand patient options,” added Tung.
The clinical program is designed to include a total of 45 healthy participants, and the company has already started single dosing the volunteers in the first trial with two deuterium-modified compounds. The compounds revealed great metabolic stability compared to preclinical results from Kalydeco and the company expects to advance to further studies with single ascending dosages, based on these results.
“We are encouraged by our preclinical findings with deuterium-modified ivacaftor compounds which support the advancement of this program into clinical evaluation”said the vice president of Translational Research and Program Director of Cystic Fibrosis at Concert, Scott Harbeson PhD. “We look forward to executing our Phase 1 clinical development plan as we believe that deuterium modification has the potential to offer an important new treatment option for cystic fibrosis patients.”
The study is expected to result in a final phase, to be initiated by the second half of the year, designed to evaluate multiple ascending doses of the selected compound, and compare it to placebo. Concert plans to present top-line data about the program upon its completion.
In other developments in cystic fibrosis, Vertex Pharmaceuticals Inc. also recently announced its submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicine’s Agency (EMA) for a fully co-formulated combination of ivacaftor and lumacaftor to treat CF in patients with two copies of the F508del mutation in the CFTR gene.