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May 23, 2018 06:30ET|Source:Clementia Pharmaceuticals Inc.

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Statistically Significant 91% Reduction of New HO at Flare-up Sites at 12 Weeks; 65% Reduction of New HO at 12-Month WBCT Scan

Evaluable Patients with No New Bone at 12 Weeks Had No New Bone at 12 Months

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MONTREAL, May 23, 2018 (GLOBE NEWSWIRE) --Clementia Pharmaceuticals Inc.(NASDAQ:CMTA), a clinical-stage biopharmaceutical company innovating new treatments for people with ultra-rare bone disorders and other diseases, today reported positive data from Part B of the companys ongoing Phase 2 clinical trial evaluating palovarotene, an RAR agonist, for the treatment of patients with fibrodysplasia ossificans progressiva, or FOP. Treatment with palovarotene resulted in meaningful reductions in new heterotopic ossification volume (HO, bone growth in abnormal places) as measured by both 12-week flare-up imaging and 12-month whole body CT (WBCT) scan. Data include a statistically significant 91 percent reduction in new HO at flare-up sites at 12 weeks as compared to those untreated flare-up sites from patients in Clementias natural history study and Phase 2 placebo group, and showed that, in evaluable patients, those who had no new HO at 12 weeks also had no new HO at 12 months. These data suggest that flare-up data could potentially be predictive of longer term outcomes. Palovarotene was generally well-tolerated in the trial, consistent with the experience across palovarotene clinical studies.

We are very pleased to report these preliminary positive data from Part B of our Phase 2 trial and are encouraged by palovarotenes potential to be the first-ever approved treatment for patients with FOP, said Clarissa Desjardins, Ph.D., co-founder and chief executive officer of Clementia. We recently surpassed enrollment of the first 35 patients in our Phase 3 MOVE Trial, and are on track to complete study enrollment by the end of the year. Based on this, we are also on-track to reach our first interim analysis in mid-2019, bringing us one step closer to being able to provide this important potential treatment to the many patients in need.

FOP is a rare, genetic bone disease that results in uncontrolled extra-skeletal bone growth for which there are no approved treatments, said study principal investigator Frederick Kaplan, M.D., the Isaac & Rose Nassau professor of orthopaedic molecular medicine and chief of the Division of Molecular Orthopaedic Medicine in the Perelman School of Medicine at the University of Pennsylvania. The preliminary data presented from this Phase 2 trial suggest that treatment with palovarotene may significantly reduce the development of new HO from flare-ups in patients with FOP. Palovarotene's reported safety profile to date is also promising. I look forward to continuing to work with Clementia and participating in the ongoing Phase 3 MOVE Trial.

Phase 2 Part B Dosing Regimens and Patient Population

The Part B portion of Clementias ongoing open-label Phase 2 trial of palovarotene for FOP enrolled 53 patients, including 41 adults and skeletally mature children (>13 years old) and 12 pediatric patients. Adults and skeletally mature children receive the chronic/flare-up dosing regimen, which includes 5mg of palovarotene daily in addition to increased dosing to 20mg for four weeks followed by 10mg for eight weeks when a flare-up is reported. Pediatric patients in Part B receive 20mg for four weeks followed by 10mg for eight weeks only when a flare-up is reported, a dosing regimen that is not used in the MOVE Trial.

As of May 15, 2018, a total of 29 flare-ups have been treated with the chronic/flare-up dosing regimen. The control group for the 12-week flare-up analysis pools data from 10 flare-ups from placebo-treated patients in the first Phase 2 trial and 50 untreated flare-ups from Clementias natural history study.

12-Week Flare-up Imaging Data

New HO volume at the site of a flare-up at 12 weeks was a pre-specified secondary endpoint in the Phase 2 study, and is an objective measure that is analyzed using standardized procedures by blinded, independent central imaging readers. In a preliminary analysis of adults and skeletally mature children, we observed a statistically significant 91 percent reduction (p=0.01) in mean volume of new HO for the 29 flare-ups treated with the chronic/flare-up dosing regimen (719mm3) in Part B as compared to the 60 untreated flare-ups (8,001mm3).

In addition to these data, Clementia analyzed data from those evaluable patients who had both 12-week flare-up scans and 12-month WBCT scans. For the 9 patients with no new HO at 12-weeks at the flare-up location, there was no new HO at 12-months anywhere in the body, including the assessed flare-up location. We believe these data suggest that flare-up data could potentially be predictive of longer term outcomes.

12-Month WBCT Scan Data

In addition to the 12-week flare-up scans, new HO in adults and skeletally mature children receiving the chronic/flare-up dosing regimen was assessed by WBCT scan at 12 months compared to untreated patients in the natural history study, regardless of whether or not flare-up symptoms were present. This preliminary per protocol assessment included 33 evaluable patients, and a 28 percent reduction in whole body volume of new HO in treated patients (21,567mm3) was observed as compared to the 55 untreated patients (29,731mm3).