2014年11月25日讯 /生物谷BIOON/ --最近，著名的罕见病药物研发公司BioMarin传出消息，公司以6亿8千万美元的价钱收购了荷兰的小型生物医药公司Prosensa公司，同时合约中还附带了价值1亿6千万美元的里程碑资金用于Prosensa公司的杜氏肌营养不良症药物的研发。这对于Prosensa公司来说无疑是雪中送炭。公司股价应声上扬。
此前Prosensa公司与制药巨头葛兰素史克公司合作开发治疗杜氏肌营养不良症的药物。由于三期对比临床研究结果不佳，Prosensa公司的这一药物此前在儿童患者六分钟独立行走测试中未能与对照组表现出明显差别，最终葛兰素史克公司决定结束这一合作项目。然而Prosensa公司最近的一些研究数据显示，在疾病出现症状的早期对患者进行用药能够显着延缓这一罕见疾病的发病流程。这也为Prosensa公司的DMD药物获批增大了一些把握。
目前，市面上关于治疗杜氏肌营养不良的药物还十分有限，尤其是专门针对患病男孩的疗法尚属空白。此次BioMarin公司收购Prosensa公司显然是经过深思熟虑，一方面，在DMD药物研发方面Sarepta和PTC公司等罕见病药物公司虎视眈眈；另一方面，BioMarin公司深知DMD药物研发时常伴随着大起大落，锦上添花总不如雪中送炭。
因此，BioMarin公司在与Prosensa公司的协议中规定了两项里程碑事件--Prosensa公司药物在2016年五月15日之前获得美国FDA的上市批准和2017年2月15日获得欧盟管理部门的上市批准，完成任意一项即可获得BioMarin公司8千万美元的里程碑资金。不过BioMarin公司的这一豪赌是否能收获回报，还需要时间来检验。
详细英文报道：
BioMarin ($BMRN) just bought itself a pricey ticket into a high-profile Phase III drug development race. The rare-disease specialist has stepped in to buy the Dutch biotech Prosensa for $680 million in cash while offering up to $160 million more in near-term milestones if it can land an early approval for its late-stage drug for Duchenne muscular dystrophy.
The buyout, which immediately triggered a 53% spike in Prosensa's share price, gives BioMarin control of drisapersen, a drug that failed a pivotal Phase III for DMD and lost its Big Pharma chaperone, GlaxoSmithKline ($GSK). With the deal BioMarin, which has enjoyed commercial success in rare diseases, finds itself competing with Sarepta ($SRPT) and PTC Therapeutics ($PTCT) in the competition to hustle ahead new DMD drugs to regulators.
BioMarin is paying $17.75 a share for Prosensa and will add two milestones of $80 million each if it can land a U.S. approval by a May 15, 2016, deadline and a Europe approval no later than February 15, 2017.
Prosensa's drug failed to beat a placebo in significantly improving boys' ability to complete a 6-minute walk test--the classic measure of success in this field. But left to its own devices the biotech, a 2012 Fierce 15 winner, completed fresh analysis of new extension study data that backed a theory that providing the drug earlier while extending treatment could delay disease progression. The biotech held what it called "positive" reviews with the FDA and began a rolling submission of the application just weeks ago. Evidently BioMarin has high hopes of success where GlaxoSmithKline saw nothing but failure.
"We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible," said BioMarin CEO Jean-Jacques Bienaime. "Further, if we are successful in advancing drisapersen to early regulatory approvals, we believe this transaction would be accretive to operating and GAAP profitability in 2017." If the transaction falls through, BioMarin is committed to acquire a $50 million equity stake in the biotech.
All three leading companies in DMD have been on an unending roller coaster ride. Sarepta's experienced a series of shocks and excitements, with the CEO advancing and then reeling back in his statements about the FDA's willingness to accept limited--though very encouraging--data sets on its DMD drug (which like drisapersen also relies on exon skipping to restore dystrophin and slow the rate of decline for boys with this lethal disease). And PTC, which has only reported failure in the clinic, was first slapped by European regulators after filing for early approval and then unexpectedly embraced.
Patient groups have played a big role in DMD. Boys afflicted with the disease have no current treatments to turn to, which has apparently persuaded regulators to take a more open-minded approach to the risk-benefit equation they use in evaluating new drug approvals. That may short-circuit the process in the U.S., which is why BioMarin may be making this gamble now after GlaxoSmithKline decided to walk.