Bristol bets big on Myokardia

The $13.1bn price suggests that Bristol could be banking on mavacamten’s potential outside rare diseases.

Myokardia’s mavacamten has undoubtedly yielded impressive data in its lead indication, obstructive hypertrophic cardiomyopathy. Whether this is enough to justify the $13.1bn that Bristol Myers Squibb dropped on the company today is another matter.

Mavacamten could also have use in some patients with heart failure with preserved ejection fraction (HFpEF), Bristol execs said today; however, they admitted that it was early days here. For now, EvaluatePharma sellside consensus puts the project’s 2026 sales at nearly $2bn, giving a net present value of around $4.5bn.


Bristol’s chief executive, Giovanni Caforio, insisted during a conference call today that mavacamten’s promise in obstructive hypertrophic cardiomyopathy (HCM) alone justified the acquisition of Myokardia, calling the indication a “multibillion opportunity”.

HCM affects around one in 500 people, making it the most common genetic cardiovascular disease. Around two thirds of these have the obstructive form, and around 25% of these are symptomatic – the population that Bristol will initially be targeting, which numbers around 80,000-100,000 patients apiece in the US and Europe.

Mavacamten, a cardiac myosin inhibitor, looks likely to get approved here based on Explorer HCM study results, which were presented at the recent ESC Congress (ESC 2020 – Myokardia odds-on for first approval in heart muscle disease, August 29, 2020).

Bristol plans to file mavacamten, which has breakthrough status, in the US in the first quarter of 2021.

Should the project get the go-ahead, Bristol will have the market to itself for a while. Currently, no specific therapies are approved for HCM and the competition is some way behind: Celltrion and Cytokinetics both have rival cardiac myosin inhibitors in the shape of CT-G20 and CK-274, but these are in phase I and phase II respectively.  

Eliquis’s footsteps

And Bristol knows what it is doing in the cardiovascular arena. The company plans to use the same playbook it employed with the anticoagulant Eliquis, which is its best-selling drug, ahead of Opdivo.

The company needs something to replace the blood thinner, which was set to go off patent by 2026, but might now cling on to exclusivity a little longer after a recent court ruling.

With mavacamten, Bristol initially intends to target around 60 specialist centres that treat around 20% of HCM patients, and then gradually expand this to include the broader cardiology setting, then primary care providers.  

On today’s call several analysts noted that heart failure therapies often suffered from a slow ramp-up in sales, but Bristol execs said they had a “strong story” to tell to payers and healthcare providers regarding mavacamten.

However, they declined to give any clues about pricing, saying it was too early to discuss this. Bristol’s ability to charge rare disease rates could take a hit if the project is approved in both HCM and a more widespread disease like HFpEF.

The more immediate next step for the project will be non-obstructive HCM, where it has completed a phase II trial, Maverick-HCM. The non-obstructive form accounts for the remaining third of HCM patients.

Bristol also highlighted other assets in Myokardia’s clinical pipeline: danicamtiv and MYK-224, a next-generation cardiac myosin inhibitor designed to have reduced pharmacokinetic variability.

Still, the main draw for Bristol is undoubtedly mavacamten. Mr Caforio would not say whether the bidding process for Myokardia was competitive, but investors will have to hope that Bristol was not pressured into overpaying.

Meanwhile, Myokardia's backers will be delighted at seeing the company bought at a 61% premium to its share price on Friday – and a whopping 2,150% premium over its listing price.

Myokardia's clinical pipeline
Project Description Indication Status 2026e sales ($m) NPV ($m)
Mavacamten  Cardiac myosin inhibitor Obstructive hypertrophic cardiomyopathy Filing due Q1 2021 1,923 4,566
    Non-obstructive hypertrophic cardiomyopathy Phase II
Danicamtiv (MYK-491/SAR440181) Cardiac myosin activator Dilated cardiomyopathy Phase II 248 622
MYK-224 Cardiac myosin inhibitor Hypertrophic cardiomyopathy Phase I  -
Source: EvaluatePharma.

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