LEXINGTON, MA, USA I November 12, 2015 I Concert Pharmaceuticals, Inc. (CNCE) today announced that it has initiated a Phase 1 multiple ascending dose clinical trial with CTP-656. CTP-656 (deuterium-modified ivacaftor) is a novel, potentially disease-modifying treatment for cystic fibrosis for use as monotherapy as well as in combination with other CFTR modulators. Ivacaftor is commercially available under the name Kalydeco®.
“CTP-656 has the potential to be an important new treatment, expanding therapeutic options for the cystic fibrosis community,” said James Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals. “We believe the development of CTP-656 as a once-daily dose could help address adherence issues documented with the current standard of care by offering a simplified treatment option.”
The multiple ascending dose Phase 1 study will be conducted in two parts and enroll 38 healthy volunteers to assess safety, tolerability and pharmacokinetics of CTP-656 in a tablet formulation. The first part will assess a single dose pharmacokinetic comparison of 150 mg of CTP-656 versus 150 mg of Kalydeco. The second part will assess three doses of CTP-656, starting at 75 mg and up to 300 mg daily for seven days compared to placebo. Additional information is available on clinicaltrials.gov.
Concert expects to report top-line results from the Phase 1 multiple ascending dose study in the first half of 2016. The Company intends to initiate a single Phase 2 trial in patients with cystic fibrosis associated with gating or minimal function CFTR mutations in the second half of 2016.
About CTP-656 and Cystic Fibrosis
CTP-656 is a novel potentiator that may enable once-daily dosing that was developed by applying deuterium chemistry to modify ivacaftor. Concert is initially developing CTP-656 as a potential monotherapy treatment for cystic fibrosis gating and minimal-function mutations of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein which regulates components of sweat, mucus clearance and digestion. Cystic fibrosis is a life-threatening, hereditary genetic disease that has systemic effects and can cause significantly reduced lung and digestive system function. According to the Cystic Fibrosis Foundation, an estimated 70,000 people worldwide have cystic fibrosis.
About Concert
Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform® (deuterated chemical entity platform) to create novel small molecule drugs. This approach starts with approved drugs, advanced clinical candidates or previously studied compounds that have the potential to be improved with deuterium substitution to enhance clinical safety, tolerability and efficacy. The Company is developing a broad pipeline targeting CNS disorders, genetic diseases, renal disease, inflammatory diseases and cancer. For more information, please visit www.concertpharma.com.
SOURCE: Concert Pharmaceuticals
Post Views: 103
LEXINGTON, MA, USA I November 12, 2015 I Concert Pharmaceuticals, Inc. (CNCE) today announced that it has initiated a Phase 1 multiple ascending dose clinical trial with CTP-656. CTP-656 (deuterium-modified ivacaftor) is a novel, potentially disease-modifying treatment for cystic fibrosis for use as monotherapy as well as in combination with other CFTR modulators. Ivacaftor is commercially available under the name Kalydeco®.
“CTP-656 has the potential to be an important new treatment, expanding therapeutic options for the cystic fibrosis community,” said James Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals. “We believe the development of CTP-656 as a once-daily dose could help address adherence issues documented with the current standard of care by offering a simplified treatment option.”
The multiple ascending dose Phase 1 study will be conducted in two parts and enroll 38 healthy volunteers to assess safety, tolerability and pharmacokinetics of CTP-656 in a tablet formulation. The first part will assess a single dose pharmacokinetic comparison of 150 mg of CTP-656 versus 150 mg of Kalydeco. The second part will assess three doses of CTP-656, starting at 75 mg and up to 300 mg daily for seven days compared to placebo. Additional information is available on clinicaltrials.gov.
Concert expects to report top-line results from the Phase 1 multiple ascending dose study in the first half of 2016. The Company intends to initiate a single Phase 2 trial in patients with cystic fibrosis associated with gating or minimal function CFTR mutations in the second half of 2016.
About CTP-656 and Cystic Fibrosis
CTP-656 is a novel potentiator that may enable once-daily dosing that was developed by applying deuterium chemistry to modify ivacaftor. Concert is initially developing CTP-656 as a potential monotherapy treatment for cystic fibrosis gating and minimal-function mutations of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein which regulates components of sweat, mucus clearance and digestion. Cystic fibrosis is a life-threatening, hereditary genetic disease that has systemic effects and can cause significantly reduced lung and digestive system function. According to the Cystic Fibrosis Foundation, an estimated 70,000 people worldwide have cystic fibrosis.
About Concert
Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform® (deuterated chemical entity platform) to create novel small molecule drugs. This approach starts with approved drugs, advanced clinical candidates or previously studied compounds that have the potential to be improved with deuterium substitution to enhance clinical safety, tolerability and efficacy. The Company is developing a broad pipeline targeting CNS disorders, genetic diseases, renal disease, inflammatory diseases and cancer. For more information, please visit www.concertpharma.com.
SOURCE: Concert Pharmaceuticals
Post Views: 103
