SOURCE: Clementia Pharmaceuticals, Inc.

Clementia Pharmaceuticals, Inc.

July 24, 2014 07:35 ET

Clementia Pharmaceuticals Receives U.S. Orphan Drug Designation for Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva

MONTREAL, QC and BOSTON, MA--(Marketwired - July 24, 2014) - Clementia Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted the Company's lead product candidate, palovarotene, Orphan Drug Designation for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). FOP is a rare, severely disabling genetic disease characterized by painful, recurrent flare-ups that lead to abnormal bone formation in muscles, tendons, and ligaments. The new, abnormal bone formation, or heterotopic ossification, causes morbidities and progressive disability.

Jeff Packman, Chief Development Officer of Clementia, stated, "Following the recent initiation of our Phase 2 clinical study, we are pleased to receive Orphan Drug Designation for palovarotene and believe this represents an important regulatory milestone for our development program."

Palovarotene is a retinoic acid receptor gamma agonist that has been shown to block bone formation in a variety of mouse models of heterotopic ossification. The ongoing Phase 2 clinical trial of palovarotene will determine whether the effects in the mouse models can be recapitulated in patients with FOP. The Company expects to complete the trial during 2015. For more information reference study protocol NCT02190747 on clinicaltrials.gov.

"There is a dire need for a treatment for people living with FOP," stated Clarissa Desjardins, Ph.D., Chief Executive Officer of Clementia. "The FDA's Orphan Drug Designation enables small companies like Clementia to take on the challenge of drug development with the ultimate goal of making palovarotene available to the FOP community as a treatment option."

The FDA Orphan Drug Designation program provides orphan status to drugs and biologics intended to safely and effectively treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the U.S. The granting of orphan drug designation does not alter the standard U.S. regulatory requirements and processes for obtaining marketing approval for an investigational drug. Safety and efficacy must be established in the treatment of a disease through adequate and well-controlled studies. Orphan Drug Designation affords the sponsoring drug development company certain incentives, including federal grants, tax credits, waiver of PDUFA filing fees and seven-year marketing exclusivity once the product is approved.

About Fibrodysplasia Ossificans Progressiva (FOP)
FOP is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in new, abnormal bone formation in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints leading to cumulative loss of function and disability. FOP is caused by a point mutation in the ALK2/BMP Type I receptor rendering it overactive. Virtually all known patients have the same point mutation and have congenital malformations of the great toes at birth. FOP is thought to affect less than one individual for every million lives.

About Palovarotene
Palovarotene is a retinoic acid receptor gamma agonist in-licensed from Roche in 2013 where it was previously evaluated in over 800 individuals including healthy volunteers and patients with chronic obstructive pulmonary disease. Palovarotene has been shown to block bone formation in a variety of mouse models of heterotopic ossification and is being investigated as a potential treatment for FOP.

About Clementia Pharmaceuticals, Inc.
Clementia is a privately held clinical stage biopharmaceutical company dedicated to the development and commercialization of treatments for people living with rare diseases by exploiting the science of novel retinoic acid receptor gamma agonists to address diseases of heterotopic ossification, including Fibrodysplasia Ossificans Progressiva. For more information, please visit www.clementiapharma.com.

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