It appears Myokardia Inc. (NASDAQ: MYOK) has cleared a major hurdle to continue with the development of its lead candidate MYK-461. The company reported positive topline results from Phase 1 studies of the molecule, which effectively pave the way for Phase 2 testing of the drug.
The Phase 1 trials were mainly designed to evaluate the safety of MYK-461, but it also ended up offering favorable glimpse into the drug’s efficacy. Myokardia Inc (NASDAQ: MYOK) expects to launch Phase 2 trial of the drug in 2H2016. But no details have been provided about when exactly the study will begin and the number of subjected expected to be enrolled.
In the studies whose early results have just been shared, MYK-461 was testing in patients with hypertrophic cardiomyopathy (HCM) as well as healthy volunteers. The Phase 2 trial to follow will be targeted at evaluating the efficacy of the molecule in symptomatic obstructive HCM or oHCM. It is worth mentioning at this juncture that the FDA has already granted the candidate Orphan Drug Designation (ODD) for oHCM indication.
The FDA’s ODD means that Myokardia will enjoy certain privileges in the development of MYK-461. The ODD also points to a potential strong demand for the drug once it comes to market.
Partnership with Sanofi
Myokardia Inc. (NASDAQ: MYOK) is partnering with Sanofi SA (ADR) (NYSE: SNY) in the development of MYK-461. The biotech has the opportunity to access up to $200 million under the agreement with Sanofi with part of the money going into funding R&D. Myokardia has already received a portion of the Sanofi’s pledged funding.
The funding together with the collaboration is will give Sanofi ex-US commercialization rights to MYK-461. But the companies have not provided estimates of the revenue they expect from the sale of the drug if it launches successfully.
Analysts react to the positive data from Myokardia Inc. (NASDAQ: MYOK)
Some analysts on Wall Street reacted positively to Myokardia Inc. (NASDAQ: MYOK)’s positive topline results from the Phase 1 studies of MYK-461. For instance, analysts at Credit Suisse have reiterated their Outperform rating on the stock of Myokardia and lifted their price target on the stock to $20 from $16.