Alnylam Spikes Prodding Ionis' Pitfall On Rare-Disease Drug Trial

Alnylam Pharmaceuticals ( ALNY ) spiked to a record high Thursday after releasing positive Phase 3 data for a rare-disease drug that would rival Ionis Pharmaceuticals ( IONS ) - helping prod the latter into a pitfall.

[ibd-display-video id=2479411 width=50 float=left autostart=true]In morning trading on the stock market today , Alnylam rocketed 14.3%, near 138.40, as shares of Ionis toppled 7.5%, near 53.40.

The results were for a Phase 3 trial of a drug called patisiran, which would treat hereditary ATTR amyloidosis in patients with polyneuropathy. Based on the results, Alnylam expects to file for approval in the U.S. by the end of 2017 and in Europe shortly after.

Hereditary ATTR amyloidosis is life-threatening and rapidly progressive. It is caused by a mutation in a specific gene that results in misfolded protein accumulating throughout the body. Symptoms can affect sensory, motor, autonomic and cardiac systems.

In a Phase 3 trial dubbed Apollo, patisiran met a series of goals. At 18 months, 148 patients treated with patisiran experienced an average 34-point improvement in symptoms of neuropathy relative to a placebo, Alnylam said in a press release.

Patisiran improved sensory, motor and autonomic neuropathy symptoms. The drug also demonstrated positive effects in a population of patients with cardiac symptoms.

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The most common side effects included swelling in the extremities and infusion-related reactions. Deaths and serious adverse events were similar in patisiran and placebo groups. None of the deaths were considered related to the drug.

Alnylam's patisiran rivals a drug from Ionis called inotersen. Also Thursday, Ionis presented Phase 3 data from a trial of inotersen in hereditary ATTR amyloidosis. Ionis' study measured results at eight months and 15 months.

Inotersen met its key goal of improving symptoms of neuropathy compared with a placebo. Statistically significant benefit was also observed in a health survey and cardiac measures.

The study discovered two key safety issues: low blood platelet count and safety signals tied to renal function, Ionis said. Enhanced monitoring was implemented during the study to detect and manage issues early.

Adverse events occurred in about 10% of patients and twice as frequently in patients treated with inotersen, Ionis said. These events included low platelet count, nausea, fever, chills, vomiting and anemia. Injection site reactions occurred in less than 1% of patients.

Overall mortality was 2.9% and lower than mortality rates reported in other studies of the same population of patients. Five patients treated with inotersen died. Of those, four deaths were associated with disease progression. The fifth resulted from a brain hemorrhage.

Leerink analyst Paul Matteis expects Alnylam's patisiran to be the market leader in treating the condition. He has a market perform rating on Alnylam.

"With all the caveats of cross-trial comparisons, the Alnylam population was actually sicker on average therefore rendering the relative delta arguably more impressive," he wrote in a note to clients.

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