Ionis Pharma Investors Still Nervous Despite Upbeat Trial Data Presented Earlier This Week

Ionis Pharma Investors Still Nervous Despite Upbeat Trial Data Presented Earlier This Week August 5, 2016
By Alex Keown, BioSpace.com Breaking News Staff

CARLSBAD, Calif. – Despite a $75 million payment from Biogen to Ionis Pharmaceuticals for successful Phase III data of their joint investigational treatment for spinal muscular atrophy, the California company still faces an uphill battle regarding trust issues for The Street’s Adam Feuerstein.

In several Tweets, Feuerstein questioned the financial impact of nusinersen, which some have speculated could hit $2 billion in annual revenue. Hitting that mark would make nusinersen one of the biggest anti-sense orphan drugs on the market, he said. Feuerstein also said the drug will be a better revenue driver for Biogen than Ionis.

“That's what happens when you sign away most of the drug's commercial rights to a partner,” Feuerstein said.

Nusinersen is the first antisense drug from Ionis’ neurological disease franchise to advance to regulatory review, the company said.

Joint drug development is nothing new for Ionis. In July, the company struck a deal worth up to $810 million with Janssen Biotech for the development of its oral gastrointestinal treatment, IONIS-JBI1-2.5. IONIS-JBI1-2.5, is an antisense drug designed to locally inhibit an undisclosed target in the gastrointestinal (GI) tract. This is the first time the two companies have collaborated.

Feuerstein said that he believes Ionis’ pipeline is overvalued “given the questions about the safety and tolerability of its technology platform.” He said that opinion has been validated by unnamed Wall Street investors and analysts. For a point of reference, Feuerstein said people should look no further than Ionis’ approved drug Kynamro, which he called a commercial flop due to safety issues. He also cited a slide presentation the company has since removed from its website about the safety inquiry into the drug that held the claim the development of the drug showed “no clinically relevant findings.

Feuerstein’s piece, which published this morning may be resonating with some investors as the company’s stock is trading lower than its opening price this morning. Shares are down to $36.20 as of 10:40.

Ionis, formerly known as Isis, has several RNA-targeting drugs in late-stage development, including volanesorsen for patients with either familial chylomicronemia syndrome or familial partial lipodystrophy.

The company has had some setbacks this year, particularly after its partner GlaxoSmithKline opted to not initiate a Phase III study of its CARDIO-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 followed an April decision by the U.S. Food and Drug Administration to place an Ionis study of CARDIO-TTR on clinical hold as a result of safety findings. Again, safety concerns were something Feuerstein pointed to in his article this morning as to why he is not bullish on Ionis.

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