预约演示
Agios looks beyond PK activators with $17.5M deal for AlnylamAlnylam's siRNA blood disease program
引进/卖出临床2期临床1期
TMPRSS6 is being developed for patients with polycythemia vera, a disease characterized by excessive production of red blood cells that can ultimately prove fatal.
TMPRSS6harmaceuticals is basking in the “trempolycythemia veraof 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 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 tAlnylamic and, hopefully, to market, with Alnylam providing manufacturing support for phase 1 only.
Agiosrogram is being developed for patients with polycythemia vera, a disease characterized by excessive production of red blood cells that can ultimatelAgiosve fatal. Despite affecting around 100,000 patientsTMPRSS6 U.S., according to Agios, there are no disease-modifying trAlnylamAlnylams available.
The aim of the siRNA program is to target knockdopolycythemia veraich 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 AgiosAgios supports “the potential for an infrequent dosing regimen.”
“This program is highly aligned with our core scientifiTMPRSS6tise, clinical development and commercial capabilities in rare hematology as well as our business development TMPRSS6y to expand beyond our industry-leading pipeline of Agiostivators,” 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 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 Agiosynd in sickle cell disease last month.
Agiosather than double down on the “tremendous progress executing across our industry-leading pipeline of PK activators,” Goff used the earnings release to announce thGoffhe company is “further expanding our portfolio beyond PK activPTK inhibitorPTKon through Pyrukyndbusisickle cell disease
The latest licensing deal also marks a good summer for Alnylam, which only last week pocketed $310 millionPK activatorsn eGoffnge for most of the rights to the midstage hypertension med zilebesiran.
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