Cowen Downgrades Ionis After Thrombocytopenia Issues

Ionis Pharmaceuticals Inc IONS shares are tanking nearly 40 percent on Thursday following the company’s disclosure that severe cases of thrombocytopenia have been associated with two of its major drug programs: TTR in FAP and APOC3 in FCS. Cowen & Co analyst Eric Schmidt has downgraded the stock on the uncertainty surrounding the incidents and their potential impact on the future of the company.

GlaxoSmithKline plc (ADR) GSK has already terminated its planned Phase III outcome study on IONIS-TTR-Rx in patients with TTR amyloid cardiomyopathy, but Schmidt believes that Ionis’ Phase III trial of volanesorsen in familial chylomicronemia syndrome may also soon be placed on hold.

“Though the company refrained from confirming whether any death associated with thrombocytopenia has occurred in the [TTR] trial, we suspect the speculation that there has been a death is true,” Schmidt writes.

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Schmidt says the revelation of a case of severe thrombocytopenia associated with APOC3 is the real bombshell for investors.

“This has implications for (1) the drug’s ongoing development in FCS and FPL (programs that we have considered second in importance after nusinersen in SMA) and (2) the company’s 2.0 generation 2’-o-methoxy platform chemistry,” Schmidt explains.

Cowen has downgraded Ionis from Outperform to Market Perform following the news.

Disclosure: the author holds no position in the stocks mentioned.