Myokardia Inc (MYOK) Heart Drug Very Likely to Achieve FDA Breakthrough Status: Cowen

It is a nice time to be a Myokardia Inc (NASDAQ:MYOK) investor, particularly on back of the biotech firm’s successful Phase 2a PIONEER trial read-out from lead drug candidate MYK-461 (Mavacamten), which aims to effectively treat obstructive and non-obstructive hypertrophic cardiomyopathy (oHCM). The drug’s major clinical stride has certainly pulled on investors’ heart strings, sending shares shooting through the roof 56% over the course of last week, closing at $36.70 on Friday.

Analyst Ritu Baral of Cowen is out with a bullish research note after having the chance to meet recently with MYOK management at their headquarters in San Fransisco to probe the lead drug candidate’s “upcoming catalysts and milestones as well as future clinical and regulatory strategies.” As such, the analyst has emerged with key highlights from her discussion that have left her enthusiastic, eagerly awaiting forthcoming data next month at the Heart Failure Society of America (HFSA) conference in Dallas.

A meaningful highlight for the analyst lies in momentum for ‘461, as all signs point to impending strong PIONEER data and the drug continuing to make headway towards earning FDA breakthrough status. Baral writes: “MYOK will hold a meeting with FDA in the near-term to discuss the design of the upcoming pivotal oHCM EXPLORE study. MYOK reiterated to us this trial would likely be sized at 200-250 patients with a 3-6 month treatment duration. We model a 6-month treatment duration given potential drug exposure requirements for regulatory safety hurdles.” Touching upon the safety of the drug the analyst additionally notes, “We will be particularly watching for biomarkers that could indicate cardiac muscle tox including troponin levels as well as proBNP. Although we currently have no figures, based on our conversation with management last week, we believe we will see no problematic signal or elevation in these figures.”

Baral expects that the upcoming HFSA presentation in September will include important data on “The two most important secondary efficacy endpoints […] left ventricular ejection fraction (LVEF) and dynspnea,” opining that the latter endpoint “not only is it considered clinically meaningful and supportive of gradient and VO2max benefit, but it could also have read through on chance of success for the non-obstructive program.”

Contending there is a highly probably chance “MYOK has applied for FDA breakthrough status on the PIONEER data,” the analyst contends approval is “very likely” thanks to such robust data. Looking ahead, Baral anticipates that the EXPLORE trial will begin as early as the fourth quarter of this year with a top-line data read-out available within two years for the heart drug.

Baral maintains an Outperform rating on MYOK stock with a price target of $56.00 representing a 52% rise above Friday’s close. (To watch Baral’s track record, click here)

TipRanks analytics demonstrate MYOK as a Strong Buy. Out of 3 analysts polled by TipRanks in the last 3 months, all 3 are bullish on Myokardia stock. With an upside potential of 35%, the stock’s consensus target price stands at $49.67.

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