Updated Authoritative Action Based on 12-Week Blast Abstracts from Completed Phase 2 Affairs and Consultation with the FDA
NDA Acquiescence Planned for the Added Bisected of 2019
MONTREAL, Oct. 23, 2018 (GLOBE NEWSWIRE) — Clementia Pharmaceuticals Inc. (Nasdaq: CMTA), a clinical-stage biopharmaceutical aggregation innovating treatments for bodies with ultra-rare cartilage disorders and added diseases, today appear that it affairs to abide a New Drug Application (NDA) for palovarotene to the U.S. Food and Drug Administration (FDA) in the added bisected of 2019. Based on contempo interactions amid Clementia and the FDA, including a Type B affair captivated beforehand today as allotment of palovarotene’s Breakthrough Analysis Designation, the FDA has agreed that accessible abstracts would abutment filing of an NDA for palovarotene for the blockage of heterotopic ossification (HO) associated with blaze up affection in patients with fibrodysplasia ossificans progressiva (FOP). The FDA based its appraisal on the adeptness and assurance abstracts accessible from the completed Phase 2 analytic program, which showed a statistically cogent abridgement in beggarly new HO volume, or cartilage volume, associated with flare-ups of FOP at 12 weeks as compared to placebo or basic flare-ups.
The abstracts provided to the FDA included the after-effects from Clementia’s Phase 2 studies, which were afresh presented at ASBMR 2018. In these studies, 92 palovarotene-treated flare-ups in 62 patients beyond three altered dosing regimens were evaluated compared to 46 placebo or basic flare-ups in 41 patients from the Company’s accustomed history study. Patients advised with palovarotene at the time of a blast accustomed a greater than 70 percent abridgement in beggarly new HO aggregate at 12 weeks compared to the basic group. Further, the abridgement in beggarly new HO aggregate amid those advised with the anecdotal 20/10 mg dosing dieting (20 mg for 4 weeks starting at the time of a blast followed by 10 mg for 8 weeks) was statistically cogent (p=0.02). Palovarotene was about able-bodied acceptable beyond all dosing regimens of the Phase 2 analytic program. There were dose-related increases in retinoid-associated adverse contest (AEs) with best actuality balmy or abstinent in severity, and alone one accommodating discontinued accord in the abstraction because of an AE.
Following the achievement of accepted non-clinical, analytic pharmacology and CMC studies to be agreed aloft with the FDA, Clementia affairs to abide its NDA in the added bisected of 2019 to seek approval of the palovarotene 20/10 mg anecdotal dosing regimen.
“The identification of a aisle to an NDA acquiescence in the added bisected of 2019 is a cogent anniversary for Clementia and for patients with this ultra-rare and adverse abiogenetic cartilage disease,” said Clarissa Desjardins, Ph.D., architect and arch authoritative administrator of Clementia. “We are beholden for the accord with the FDA’s Division of Bone, Reproductive and Urologic Products, potentially bringing the aboriginal accustomed analysis advantage to individuals afflicted by FOP. We are additionally beholden to the patients and their families, as able-bodied as the board and analytic sites, after whom none of this assignment would accept been possible.”
Clementia’s advancing Phase 3 MOVE Balloon will abide as planned, evaluating a dosing dieting of palovarotene which includes a abiding 5 mg circadian dosage in accession to the anecdotal 20/10 mg dosing dieting at the time of a flare-up. The Aggregation believes that, if successful, the abstracts from the MOVE Balloon may accommodate the base for a added NDA for an added analysis dieting advantage for patients with FOP.
Clementia additionally affairs to appoint all-embracing authoritative authorities in 2019 to altercate the allotment action for palovarotene and anticipates accouterment an amend at a after stage.
About Palovarotene Palovarotene is an RARγ agonist actuality developed as a analysis for patients with ultra-rare and debilitating cartilage diseases, including fibrodysplasia ossificans progressiva (FOP) and assorted osteochondromas (MO), as able-bodied as added diseases. Palovarotene was in-licensed from Roche Pharmaceuticals, area it was ahead evaluated in added than 800 subjects, including 450 patients advised for up to two years. Palovarotene has accustomed Orphan Drug cachet for FOP and MO from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In addition, palovarotene has been accepted Fast Track and Breakthrough Analysis designations for FOP from the FDA.
About Fibrodysplasia Ossificans Progressiva (FOP)FOP is a rare, acutely disabling ataxia characterized by heterotopic ossification (HO), or cartilage that forms alfresco the accustomed skeleton, in muscles, tendons or bendable tissue. In FOP, HO progressively restricts movement by locking joints, arch to a accumulative accident of function, accelerating disability, and added accident of aboriginal death. FOP is acquired by a alteration in the ACVR1 gene, consistent in balance signaling in the cartilage morphogenetic pathway, a key alleyway authoritative cartilage advance and development, by way of both ligand-dependent and absolute mechanisms. There are currently no accustomed treatments for FOP.
About Clementia Pharmaceuticals Inc.Clementia is a clinical-stage aggregation innovating treatments for bodies with ultra-rare cartilage disorders and added diseases with aerial medical need. The Aggregation is advancing for a 2019 NDA acquiescence to the FDA to seek approval of its advance artefact candidate, palovarotene, a atypical RARγ agonist, for the analysis of fibrodysplasia ossificans progressiva (FOP). The advancing Phase 3 MOVE Balloon is evaluating an added dosing dieting of palovarotene which includes a abiding 5 mg circadian dosage in accession to the anecdotal 20/10 mg dosing dieting at the time of a flare-up. Palovarotene is additionally in a Phase 2 trial, the MO-Ped Trial, for the analysis of assorted osteochondromas (MO, additionally accepted as assorted ancestral exostoses, or MHE). In addition, Clementia has commenced a Phase 1 balloon for an eye bead conception of palovarotene for the abeyant analysis of dry eye ache and is additionally investigating added altitude that may account from RARγ therapy. For added information, amuse appointment www.clementiapharma.com and affix with us on Twitter @ClementiaPharma.
Cautionary Note Regarding Forward-Looking StatementsThis columnist absolution may accommodate “forward-looking statements” aural the acceptation of the applicative balance laws, including with account to the proposed timing of acquiescence of the NDA for palovarotene. Each advanced account independent in this columnist absolution is accountable to accepted and alien risks and uncertainties and added alien factors that could account absolute after-effects to alter materially from actual after-effects and those bidding or adumbrated by such statement. In accession to statements which absolutely call such risks and uncertainties, readers are apprenticed to accede statements labeled with the agreement “believes,” “belief,” “expects,” “intends,” “anticipates,” “will,” or “plans” to be ambiguous and forward-looking. Applicative risks and uncertainties include, amid others, the Company’s adeptness to auspiciously complete in a appropriate address the studies appropriate to be completed in adjustment to abide the NDA, the Company’s adeptness to accomplish acquirement and become profitable; the risks accompanying to its abundant assurance on palovarotene, its alone accepted artefact candidate; the risks associated with the development of palovarotene and any approaching artefact candidate, including the affirmation of adeptness and safety; its assurance on accountant bookish property, including the adeptness to antecedent and advance licenses from third-party owners; as able-bodied as the risks articular beneath the branch “Risk Factors” in the Company’s Annual Report on Form 20-F filed with the Balance and Exchange Commission (“SEC”), as able-bodied as the added advice its book with the SEC or on SEDAR. Clementia cautions investors not to await on the advanced statements independent in this columnist absolution back authoritative an advance accommodation in its securities. Investors are encouraged to apprehend the Company’s filings with the SEC or on SEDAR, accessible at www.sec.gov or www.sedar.com, for a altercation of these and added risks and uncertainties. The advanced statements in this columnist absolution allege alone as of the date of this columnist release, and the Aggregation undertakes no obligation to amend or alter any of these statements, whether as a aftereffect of new information, approaching contest or otherwise, except as appropriate by law.
Investor/Media Contacts:Joseph WalewiczClementia Pharmaceuticals Inc. 1-514-940-1080
Alicia DavisTHRUST Strategic Communications [email protected]
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