2014年11月25日訊 /生物谷BIOON/ --最近，著名的罕見病藥物研發公司BioMarin傳出消息，公司以6億8千萬美元的價錢收購了荷蘭的小型生物醫藥公司Prosensa公司，同時合約中還附帶了價值1億6千萬美元的裡程碑資金用於Prosensa公司的杜氏肌營養不良症藥物的研發。這對於Prosensa公司來說無疑是雪中送炭。公司股價應聲上揚。
此前Prosensa公司與製藥巨頭葛蘭素史克公司合作開發治療杜氏肌營養不良症的藥物。由於三期對比臨床研究結果不佳，Prosensa公司的這一藥物此前在兒童患者六分鐘獨立行走測試中未能與對照組表現出明顯差別，最終葛蘭素史克公司決定結束這一合作項目。然而Prosensa公司最近的一些研究數據顯示，在疾病出現症狀的早期對患者進行用藥能夠顯著延緩這一罕見疾病的發病流程。這也為Prosensa公司的DMD藥物獲批增大了一些把握。
目前，市面上關於治療杜氏肌營養不良的藥物還十分有限，尤其是專門針對患病男孩的療法尚屬空白。此次BioMarin公司收購Prosensa公司顯然是經過深思熟慮，一方面，在DMD藥物研發方面Sarepta和PTC公司等罕見病藥物公司虎視眈眈；另一方面，BioMarin公司深知DMD藥物研發時常伴隨著大起大落，錦上添花總不如雪中送炭。
因此，BioMarin公司在與Prosensa公司的協議中規定了兩項裡程碑事件--Prosensa公司藥物在2016年五月15日之前獲得美國FDA的上市批准和2017年2月15日獲得歐盟管理部門的上市批准，完成任意一項即可獲得BioMarin公司8千萬美元的裡程碑資金。不過BioMarin公司的這一豪賭是否能收穫回報，還需要時間來檢驗。（生物谷Bioon.com）
詳細英文報導：
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.