Agios Pharmaceuticals is basking in the “tremendous progress” of its PK activator pipeline, but that hasn’t stopped the biotech from looking for ways to freshen up its hematology portfolio courtesy of a preclinical siRNA program from Alnylam.

Agios Pharmaceuticals正致力于其PK激活剂管道的“巨大进步”，但这并未阻止生物技术公司寻找通过Alnylam的临床前siRNA计划来更新其血液学组合的方法。

Agios is paying $17.5 million upfront for the program with up to $130 million in development and regulatory milestones potentially to follow. In return, Agios is taking on sole responsibility for ushering the TMPRSS6 program through the clinic and, hopefully, to market, with Alnylam providing manufacturing support for phase 1 only..

Agios为该计划支付1750万美元的前期费用，开发和监管里程碑可能会达到1.3亿美元。作为回报，Agios全权负责通过诊所推出TMPRSS6计划，并有望推向市场，Alnylam仅为第1阶段提供制造支持。。

The program is being developed for patients with polycythemia vera, a disease characterized by excessive production of red blood cells that can ultimately prove fatal. Despite affecting around 100,000 patients in the U.S., according to Agios, there are no disease-modifying treatments available.

该计划正在为真性红细胞增多症患者开发，这种疾病的特点是红细胞过量产生，最终可能致命。根据Agios的说法，尽管在美国影响了大约100000名患者，但尚无可改善疾病的治疗方法。

The aim of the siRNA program is to target knockdown of TMPRSS6, which is a key driver of red blood cell production. So far, the program has demonstrated a 90% knockdown of TMPRSS6 mRNA over three months in nonhuman primates, which Agios said supports “the potential for an infrequent dosing regimen.”.

siRNA程序的目的是靶向TMPRSS6的敲低，TMPRSS6是红细胞生成的关键驱动力。到目前为止，该计划已证明在非人类灵长类动物中三个月内TMPRSS6 mRNA的敲低率为90%，Agios说这支持“罕见给药方案的潜力”。

“This program is highly aligned with our core scientific expertise, clinical development and commercial capabilities in rare hematology as well as our business development strategy to expand beyond our industry-leading pipeline of PK activators,” Agios CEO Brian Goff said in the release. “We look forward to initiating IND-enabling studies this year with the aim of delivering a transformative treatment option for this patient community with profound unmet need.”.

Agios首席执行官Brian Goff在该版本中说：“该计划与我们在稀有血液学方面的核心科学专业知识，临床开发和商业能力以及我们的业务发展战略高度一致，以超越我们行业领先的PK激活剂管道。“我们期待今年启动IND支持性研究，旨在为这个患者群体提供一种变革性的治疗方案，并且需求尚未得到满足。”。

Agios describes itself as the “pioneering leader in PK activation.” In the company’s second-quarter earnings release, which coincided with the licensing announcement, Goff pointed to positive phase 2 data from the biotech’s approved PTK inhibitor Pyrukynd in sickle cell disease last month.

Agios将自己描述为“PK激活的先驱领导者”。在公司第二季度的收益发布中，与许可公告同时，Goff指出了生物技术公司上个月批准的PTK抑制剂Pyrukynd在镰状细胞病中的第二阶段数据。

But rather than double down on the “tremendous progress executing across our industry-leading pipeline of PK activators,” Goff used the earnings release to announce that the company is “further expanding our portfolio beyond PK activation through focused business development.”

但Goff并没有加倍“在我们行业领先的PK激活剂管道中执行的巨大进步”，而是利用盈利发布来宣布该公司“通过集中业务开发进一步扩大我们的产品组合，超越PK激活”

The latest licensing deal also marks a good summer for Alnylam, which only last week pocketed $310 million from Roche in exchange for most of the rights to the midstage hypertension med zilebesiran.

最新的许可协议也标志着Alnylam的美好夏天，Alnylam上周才从罗氏（Roche）购买了3.1亿美元，以换取中期高血压med zilebesiran的大部分权利。