June 1, 2016
By Alex Keown, BioSpace.com Breaking News Staff
CAMBRIDGE, Mass. – One company’s loss is another company’s gain. That’s certainly the case when looking at California-based Ionis Pharmaceuticals and Massachusetts-based Alnylam .
Last week, Ionis was dealt a serious blow after its partner GlaxoSmithKline opted to not initiate a Phase III study of its IONIS-TTR drug program for the treatment of patients with TTR amyloidosis. GSK’s move caused Ionis stock to plummet more than 35 percent. GSK’s move also came after the U.S. Food and Drug Administration announced a delay in Ionis’ late-stage trial, citing safety concerns.
But, Ionis’ loss has been beneficial to Alnylam, which is also developing an RNAi-based therapy for TTR amyloidosis. Since Ionis’ stock has fallen, Alnylam has seen shares of its stock increase nearly $20 per share—meaning a gain of about $1.5 billion in market cap, the Boston Business Journal noted Tuesday afternoon.
Alnylam is currently developing its Phase III lead candidate Patisiran for TTR amyloidosis at a small lab in Cambridge. The company is anticipating final data for Patisiran by next year and hopes the path for regulatory approval is clear. In fact, by 2020, John Maraganore, Alnylam’s chief executive officer, told BioSpace in an exclusive interview, that he anticipates Alnylam will be a multi-product company. He said the company has a “real engine” to develop mew medications to treat various diseases.
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. Therapeutic options for the treatment of patients with TTR amyloidosis are very limited.
While Ionis-TTR had some safety concerns, Alnylam’s Patisaran does not seem to have the same issues, analysts at J.P. Morgan told the Journal.
“With (Ionis’ trial) on pause indefinitely, we believe Alnylam’s phase III ENDEAVOR trial benefits from an execution standpoint, via lower competition for patients,” J.P. Morgan analyst Anupam Rama said, according to the Journal.
Not only is Alnylam benefitting from Ionis’ setback, but the company is also experiencing explosive growth, anticipating the hiring of more than 1,000 employees over the next few years and the opening of a new $200 million manufacturing center. The new site is expected to manufacture Alnylam’s RNAi-based therapeutics for clinical trials, as well as drugs for commercialization when the company ushers its first product to market. In addition to the new manufacturing site, Alnylam also leased an additional 100,000 square feet in Kendall Square, with plans to expand their headquarters by 2019.
Additionally, there are talks that French drugmaker Sanofi could be interested in acquiring Alnylam. The Business Journal reported that Sanofi already owns 12 percent of Alnylam’s stock. Alnylam’s chief business officer, David-Alexandre Gros, is a former Sanofi executive, who was instrumental in the French company’s investment in Alnylam, the Journal noted.
