Why All Eyes Will be on Alnylam and Any Updates of Its APOLLO Phase III Trial This Friday

May 11, 2017
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – Alnylam is expected to release data from the Phase III Apollo trial of its RNAi therapeutic, patisiran, in September, which is something that investors are closely watching, particularly as the company plans to host its investor conference on May 18.

When Alnylam released its quarterly financial report last week, the company pointed to how pivotal the Apollo trial is for the patisiran program. John Maraganore, Alnylam’s chief executive officer, said the company could file for regulatory approval by the end of the year if data is positive. The Apollo trial is studying patisiran for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP).

“In addition, we expect to advance three additional programs into Phase III trials: fitusiran, givosiran, and—with our partners at The Medicines Company —inclisiran,” Maraganore said.

In the company’s quarterly report, Maraganore said the company could be well on its way to be a multi-product company by 2020.

In a statement, Maraganore said the company is seeing “great progress” with each of these programs.

Brian Orelli, an analyst writing for The Motley Fool, noted that earlier this year Alnylam presented encouraging data for fitusiran in patients with hemophilia A or B at the meeting of the European Association for Haemophilia and Allied Disorders. He also said that The Medicines Company presented positive data from its Phase II trial of inclisiran, which reduced LDL cholesterol by more than 50 percent for a six-month period. And last month, Orelli noted that data from Alnylam’s Phase II trial of patisiran “showed a mean decrease in the modified neuropathy impairment score (mNIS+7) of seven points” at 24 months.

Regarding its Apollo data, Orelli said Maraganore did not reveal much during an investor call, but instead focused on the potential of failure in rival drugmaker Ionis Pharmaceutical’s TTR-mediated amyloidosis treatment, IONIS-TTRRx. In his discussion, Maraganore said he expects Ionis’ study to confirm the “TTR-lowering hypothesis,” but said there is a chance the Ionis data will reveal “mixed or even false-negative results based on their smaller sample size, shorter study duration, use of a co-primary endpoint structure that includes quality of life, potentially higher discontinuation rates due to adverse events or other factors, and slower, less robust TTR lowering effect, among others.”

TTR amyloidosis is a severe, progressive and fatal disease with multiple overlapping clinical manifestations. There are three forms of TTR amyloidosis: familial amyloid polyneuropathy (FAP), familial amyloid cardiomyopathy (FAC), and wild type (wt)-TTR amyloidosis. The disease is caused by the accumulation of misfolded TTR protein in a broad range of tissues and organs, including peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to organ failure and eventually death.