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Importantly, in this evaluation, one patient included in the analysis experienced multiple flare-ups which were not treated with the 20/10mg regimen because they did not meet the requirements for flare-up treatment according to the Part B protocol. Utilizing the MOVE Trial flare-up definition and treatment criteria, which require only one flare-up symptom, this patient would have qualified for high-dose treatment in the Phase 3 MOVE Trial. When we analyze these data without this one patient, treatment with palovarotene resulted in a 65 percent mean reduction of new HO by WBCT (10,682mm3) compared to the untreated control group (29,731mm3).

We believe that the MOVE Trial has optimized the flare-up definition and treatment regimen to evaluate the potential for benefit in patients, said Donna Grogan, M.D., chief medical officer of Clementia. The findings in the Part B support our design of the MOVE Trial, and we look forward to continuing to advance the study so that we can define the potential benefit of palovarotene as quickly as possible. In addition, we would like to thank all the patients, caregivers and site personnel for their dedication all along the way.

Safety Analysis

Palovarotene has been generally well-tolerated in this ongoing trial, with the majority of adverse events being mild to moderate mucocutaneous events, consistent with retinoid-associated side effects and the experience across palovarotene clinical studies. These events are primarily managed with prophylactic treatments or dose reductions. The majority of dose reductions occurred when patients were receiving the 20mg flare-up dose. No patients have discontinued treatment in the trial due to a palovarotene-related-adverse event.

Phase 3 MOVE Trial Update

Enrollment is well underway in Clementias Phase 3 MOVE Trial of palovarotene for the treatment of FOP. The MOVE Trial will enroll 80 patients age 4 and older and utilizing the chronic/flare-up dosing regimen. Data from patients in Clementias natural history study are serving as the external control. The company has enrolled more than 35 patients in the trial and is on track to complete enrollment by the end of the year. The MOVE Trial is designed with three interim analyses: the first when 35 patients have 12-month WBCT scans, and the next two interim analyses coming six and 12 months thereafter. Based on current enrollment, Clementia is on track to report the first interim analysis for the MOVE Trial in mid-2019.

Conference Call Information

To participate in the conference call, please dial (866) 916-2014 (domestic) or (636) 812-6655 (international) and refer to conference ID 7898867. The webcast can be accessed in the Investor Relations section of the company's website at The replay of the webcast will be available in the investor section of the companys website at 60 days following the call.

About the Phase 3 MOVE Trial

All patients in the MOVE Trial receive a single daily dose of palovarotene, with increased dosing at the time of a flare-up (the chronic/flare-up regimen). Data from Clementias natural history study (NHS) will serve as the control. Patients are treated with palovarotene for 24 months, with three planned interim analyses. The primary efficacy endpoint of the MOVE Trial is the annualized change in new heterotopic ossification (HO) volume as assessed by low-dose whole body CT (WBCT) scan (excluding head) compared to untreated patients from the NHS. Secondary endpoints include the proportion of patients with any new HO, the change in the number of body regions with new HO, the proportion of patients reporting flare-ups and the rate of flare-up occurrence. Exploratory endpoints include functional assessments of joint function and changes in patient-reported physical function. Safety evaluations include adverse events, assessments of growth in children, clinical laboratory tests and vital signs. Full details of the study can be found at, NCT03312634.

About Palovarotene

Palovarotene is an RAR agonist being developed as a treatment for patients with ultra-rare and debilitating bone diseases, including fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), as well as other diseases. Data from the palovarotene Phase 2 program suggest that palovarotene has the ability to significantly reduce the development of new bone growth in abnormal places, or heterotopic ossification (HO) in patients with FOP. Palovarotene was also found to inhibit the formation of osteochondromas (OCs) in preclinical models of multiple exostoses, supporting the initiation of the MO-Ped Trial in the MO indication. Palovarotene has received Orphan Drug status for FOP and MO from the U.S. Food and Drug Administration (FDA), and orphan status for the treatment of FOP in the EU. In addition, palovarotene has been granted Fast Track and Breakthrough Therapy designations for FOP from the FDA.

About Fibrodysplasia Ossificans Progressiva (FOP)