8亿美元到手，Alnylam与Regeneron扩大RNAi疗法的合作范围

Pharmews · 公众号 · · 2019-04-08 21:20

正文

#RNAi

去年三月

Regeneron与Alnylam达成合作协议

旨在开发用于NASH治疗的 RNAi疗法

Regeneron负责对HSD17B13靶点的研究

该靶点或可降低慢性肝病风险

而Alnylam则将利用RNAi疗法开发平台

来识别针对该靶点的化合物

而此次合作范围

将进一步扩大

用于包括CNS、眼部和肝脏疾病

RNAi单药和联合疗法的开发

Regeneron给出4亿美元首付款

以及

外加4亿美元的股权投资

另外还将有可能拿到

高达2亿美元的早期临床里程碑款

该临床用于眼部和中枢神经系统疾病

五年合作开发项目中

多达30个研发项目

每年也将有1-2个新的INDs

包括CNS、眼部和肝脏疾病

Alnylam依旧掌控Cemdisiran单药疗法

Regeneron则主导组合疗法

包括Cemdisiran和Pozelimab

“

At Regeneron we believe the best use of our resources is to invest in potentially game-changing science that will yield innovative medicines for patients with serious diseases. This collaboration couples proven and emerging RNAi technology, which holds important promise in many diseases, with Regeneron’s world-leading genetics research and target discovery engine. This collaboration enables us to reach targets inside the cell complementing our expertise in antibodies, which are ideal for extracellular targets and those on the cell surface. Through the RGC and our other research groups, we are already identifying additional targets that may be well-suited for RNAi-based drug development, particularly in the eye and CNS.

—— George D. Yancopoulos, M.D., Ph.D.

President and Chief Scientific Officer of Regeneron

”

“

This new industry-leading alliance is aimed at realizing what we believe to be a significant opportunity for RNAi therapeutics as potentially transformative medicines for ocular and CNS diseases. We are thrilled to collaborate with Regeneron, a like-minded science-based organization, to significantly accelerate our efforts to bring RNAi therapeutics to patients. Importantly, the alliance structure enables Alnylam to continue to build its industry-leading pipeline of RNAi therapeutics while retaining significant product rights. In addition, the near-term payments under this new agreement will strengthen Alnylam’s balance sheet with over $2 billion in pro forma cash upon closing of the transaction, supporting our global efforts to develop and commercialize multiple products as potentially breakthrough medicines and advance our profile toward sustainable profitability.

—— John Maraganore, Ph.D.

Chief Executive Officer of Alnylam