Based in Montreal, Canada, CMTA is a clinical stage biopharmaceutical company, specializing in the development of disease-modifying treatments for patients suffering from bone and other diseases with high unmet medical needs.
It’s lead candidate—palovarotene—is an oral molecule that activates retinoic acid receptor gamma (RARg agonist), which has shown potent activity in preventing abnormal new bone formation and scar tissue formation in a variety of tissues in animal models.
Palovarotene is being developed for the treatment of Fibrodysplasia Ossificans Progressiva (FOP) and Multiple Osteochondroma (MO). The company has a Phase 3 trial and a Phase 2/3 trail for two separate indications that are expected to begin in 2017 with readouts planned for 2019 and 2020. The company believes that a regulatory approval could pave the way for palovarotene to become the standard of care for FOP and/or MO.
The company estimates that the prevalence of FOP amounts to approximately 1.3 individuals per million, suggesting roughly 9,000 global cases. In October 2016, there were 800 known patients diagnosed with FOP worldwide. The prevalence of MO is estimated at 20 individuals per million, suggesting 150,000 cases worldwide.
Palovarotene was in-licensed from Roche Pharmaceuticals after the drug showed promise in animal subjects. This formed the basis of the company, which was granted an Orphan Drug Designation from the Food and Drug Administration in July 2014. In November 2014, the company’s product received the same designation from the European Medicines Agency. The designation gives the company seven years of market exclusivity in the United States and ten years in Europe.
CMTA is yet to generate any revenue through its products. For the first three months of 2017, the company incurred R&D expenses of $3.41 million, down from $3.67 million in 2016. For 2016, R&D expenses totaled $16.85 million, up from $14.40 million in 2015 and $7.80 million in 2014.
CMTA’s cash and short-term investments balance at the end of March 2017 totaled $43.72 million, up from $39.43 million at the end of 2016, but down from $58.11 million in 2015. At the end of 2014, the company had $5.50 million in cash and short-term investments.
The company used $5.94 million for operating activities in the first quarter of 2017, up from $5.65 million used one year ago. In 2016, $18.83 million was used for operating activities, up from $17.62 million used in 2015 and $9.66 million used in 2014.