Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Adds Hepatic Porphyria Drug to Official Pipeline

Premium

Alnylam Pharmaceuticals said this week that it has formally advanced its preclinical hepatic porphyria treatment into its development pipeline.

The drug, called ALN-AS1, is designed to silence ALAS-1, a liver-expressed rate-limiting enzyme upstream of porphobilinogen deaminase. Inhibition of ALAS-1 has been shown to reduce the accumulation of heme precursors that cause the clinical manifestations of acute intermittent porphyria — a form of porphyria Alnylam is focusing on.

The company said that non-human primate data show that multiple subcutaneous doses of a GalNAc conjugate formulation of the drug led to rapid, dose-dependent, and long-lasting knockdown of the ALAS-1 mRNA, with an ED50 of approximately 1.25 mg/kg.

In rodent studies, doses as low as 2.5 mg/kg resulted in a complete blunting of phenobarbital-induced over-production of the toxic heme intermediates PBG and ALA.

Alnylam said that it will now begin investigational new drug application-enabling studies of ALN-AS1, with the goal of filing an IND next year.

The Scan

Support for Moderna Booster

An FDA advisory committee supports authorizing a booster for Moderna's SARS-CoV-2 vaccine, CNN reports.

Testing at UK Lab Suspended

SARS-CoV-2 testing at a UK lab has been suspended following a number of false negative results.

J&J CSO to Step Down

The Wall Street Journal reports that Paul Stoffels will be stepping down as chief scientific officer at Johnson & Johnson by the end of the year.

Science Papers Present Proteo-Genomic Map of Human Health, Brain Tumor Target, Tool to Infer CNVs

In Science this week: gene-protein-disease map, epigenomic and transcriptomic approach highlights potential therapeutic target for gliomas, and more