据赛诺菲公司透露，此次协议至少涉及2亿美元，其中包括了研发过程中的里程碑资金，赛诺菲公司将先期投入4500万美元作为早期研究之用。MyoKardia公司是由著名风投公司Third Rock公司投资成立的，在这一合作中，公司将主要负责早期人类功效数据处理工作，而赛诺菲公司将主要提供资金支持。
此次合作协议的范围涉及了MyoKardia公司的三个研究项目，其中包括了两个关于肥厚性心脏病药物研发项目和一个扩张性心脏病药物研发项目。双方计划到2018年完成这一协议研发。
2012年Third Rock公司投入3800万美元资金创立MyoKardia公司，公司主要专注于治疗肥厚性心脏病和扩张性药物的研发工作，在美国有数百万人受这两种疾病的折磨。公司的主要研究着眼于开发治疗心肌肌节蛋白突变引发疾病的个性化小分子药物疗法。
不久之前，公司刚刚宣布开始进行总额为1000万美元的融资项目。而值得注意的是，由于心脏病药物研发需要巨大的投入，长期以来心脏病药物的研发一直被世界大型生物医药公司所把持，此次赛诺菲的投资无疑是雪中送炭为MyoKardia公司提供了强有力的资金支持。而赛诺菲公司的高层也对这一合作寄予了厚望。如果MyoKardia公司的研究能获得突破，则有望打破世界医药巨头对这一领域的垄断。
详细英文报道：
Sanofi ($SNY) has stepped up with a $45 million down payment on a potential $200 million early-stage deal to develop new drugs for heart muscle disease. The pharma giant inked one if its rare small-biotech collaboration pacts with MyoKardia, a South San Francisco-based biotech that was funded by Third Rock a couple of years ago.
Now MyoKardia will take the lead on the work through early human efficacy data, with Sanofi offering financial support through 2018.
The $200 million deal--which involves milestones, an equity investment and research support--covers three MyoKardia programs; two for hypertrophic cardiopathy and one for dilated cardiomyopathy. Pushing ahead past proof-of-concept work in humans, the two partners plan to split the development costs on the hypertrophic work with Sanofi paying the full freight on the third dilated cardiomyopathy program.
MyoKardia filed a Form D with the SEC a few weeks ago outlining a $10 million raise. CEO Tassos Gianakakos confirmed that that was Sanofi's equity stake, with the rest of the initial round of cash coming in an upfront. The full deal positions the biotech to achieve its full 5-year vision for the company, says the CEO, who joined MyoKardia a year ago.
In many ways, MyoKardia is a classic Third Rock creation. Helmed initially by Charles Homcy, a partner in the venture group, it gathered together some marquee scientific talent to go after the root causes of hypertrophic and dilated cardiomyopathy, a condition that afflicts a million people in the U.S. The biotech set out to target personalized small-molecule treatments that can address protein mutations in the sarcomere--part of the heart muscle--for small "buckets" of patients. Third Rock provided a $38 million A round in 2012 to get the company off the launch pad.
Cardiovascular disease is a field dominated by giant pharmas which can afford to do the kind of huge late-stage studies needed to gain an approval for a large patient population. MyoKardia, though, is out to "break down heart failure into genetic subtypes," says the CEO--a rare disease approach that Sanofi's Genzyme used to build the company.
"We're doing something totally different. It's totally new," says Gianakakos. "We know exactly what is driving the disease."
The CEO added that the company has already built a staff of 45 and plans to add more researchers to the mix as it expands its efforts.
Sanofi is better known in the industry for its big R&D partnerships with the likes of Regeneron ($REGN) and Alnylam ($ALNY). This much smaller, early-stage pact is part of its "Sunrise initiative," which is aimed at licensing rights to very early-stage work. Sanofi joined hands on another Third Rock startup in 2012, when the pharma giant participated in the $125 million launch of Warp Drive Bio in Cambridge, MA.
"We're going to focus on really getting this relationship off to the right start," adds Gianakakos, emphasizing that the biotech remains in charge of the research during the first stage of the pact. "Financially, this partnership significantly derisks the company."
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