Clementia Pharmaceuticals Inc.’s (CMTA) shares were up 35.40% in pre-market trading after the Canada-based biotech firm announced plans to file a new drug application (NDA) supported by encouraging testing results.
In a press release, Clementia said it is seeking approval of palovarotene to prevent heterotopic ossification (HO), the abnormal growth of bone in non-skeletal tissues, in patients that suffer from fibrodysplasia ossificans progressive (FOP), a rare and dangerous genetic bone disease.
The biotech firm added that the Food and Drug Administration (FDA) agreed to let Clementia use Phase 2 data to support its application. Those studies, which were presented earlier this year, point to palovarotene being an effective remedy.
Patients treated with palovarotene at the time of a flare-up demonstrated a greater than 70% reduction in mean new HO volume at 12 weeks compared to the untreated group, according to the company’s Phase 2 data. Clementia is set to submit its full application to the FDA in the second half of 2019.
In the press release, the biotech firm heralded the NDA submission as a “significant milestone,” adding that its drug is well-placed to become the first of its kind to treat cases of HO in FOP sufferers.
“The identification of a path to an NDA submission in the second half of 2019 is a significant milestone for Clementia and for patients with this ultra-rare and devastating genetic bone disease,” said Clarissa Desjardins, founder and CEO of Clementia. “We are thankful for the collaboration with the FDA’s Division of Bone, Reproductive and Urologic Products, potentially bringing the first approved treatment option to individuals affected by FOP. We are also grateful to the patients and their families, as well as the investigators and clinical sites, without whom none of this work would have been possible.”
Clementia confirmed that its ongoing “Phase 3 MOVE Trial”, which consists of giving patients “a chronic 5 mg daily dose” in addition to the episodic 20/10 mg dosing regimen at the time of a flare-up, will continue as planned. If these trials are successful, the company added that they could later be used for a supplemental NDA.
Clementia also said it will approach international regulatory authorities next year about how to register palovarotene.