Alnylam: ALN-AS1 Phase 1 Study Shows Positive Data, Gets FDA Orphan Drug Status

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY), a RNAi therapeutics company, announced Wednesday positive interim data from its ongoing Phase 1 study with ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 or ALAS1 for the treatment of acute hepatic porphyrias.

Further, Alnylam announced that the United States Food and Drug Administration or FDA has granted Orphan Drug Designation to ALN-AS1 for the treatment of acute hepatic porphyrias. The Orphan Drug Act provides incentives for sponsors to develop products for rare diseases. In July 2016, the European Medicines Agency granted Orphan Drug Designation to ALN-AS1 for the treatment of acute hepatic porphyrias.

The company noted that interim data from the ongoing study demonstrated that single and once-monthly, subcutaneous administration of ALN-AS1 achieved rapid, dose-dependent, and durable lowering of ALA and PBG. Further, ALN-AS1 was found to be generally well tolerated.

The new data were from Parts A and B of the ongoing Phase 1 study, which were conducted in asymptomatic "high excreter" or ASHE subjects.

ASHE subjects have a mutation in the porphobilinogen deaminase gene as found in acute intermittent porphyria and have elevated levels of upstream toxic heme intermediates aminolevulinic acid and porphobilinogen that mediate porphyria attacks.

These results were presented today during an oral presentation at the 2016 Society for the Study of Inborn Errors of Metabolism Annual Symposium, being held in Rome, Italy.

Alnylam is currently conducting Part C of the Phase 1 study in symptomatic AIP patients with recurrent porphyria attacks.

Consistent with previous guidance, the Company plans to present initial porphyria biomarker data from Part C in late 2016, with potential clinical efficacy data on the frequency and severity of recurrent attacks expected in 2017.

Akshay Vaishnaw, Chief Medical Officer at Alnylam, said, "The acute hepatic porphyrias are a group of ultra-rare orphan diseases with enormous unmet medical need, where novel therapies are clearly warranted. Accordingly, we're very encouraged by these interim Phase 1 data in ASHE subjects, showing robust lowering of the toxic heme synthesis intermediates that mediate porphyria attacks."