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Safety And Tolerability Issues Continue To Plague Ionis Pharmaceuticals

Safety And Tolerability Issues Continue To Plague Ionis Pharmaceuticals - IONS

Safety and tolerability have been recurrent concerns regarding the drugs produced by Ionis Pharmaceuticals Inc IONS 1.91%, according to Morgan Stanley's David N. Lebowitz, which has kept the stock valuation in check.

Lebowitz initiated coverage of the company with an Equal-Weight rating and price target of $37.

Safety And Tolerability Concerns

“While nusinersen is a likely blockbuster, safety & tolerability concerns for IONIS-TTRRx and volanesorsen likely to remain near-term overhang,” the analyst said.

Lebowitz pointed out that the platelet declines seen in the two lead candidates are the most recent examples of the safety overhang.

Related Link: Needham Raises Target On Ionis Pharam From $55 To $64, Maintains Buy Rating

“That said, we see nusinersen, a drug for babies with a deadly form of muscle atrophy, as a likely blockbuster and a backstop to valuation.”

Ionis Pharma’s two Phase III agents, IONIS-TTR for TTR amyloidosis treatment, and volanesorsen for high triglyceride levels showed serious thrombocytopenia, which led to the CARDIO-TTR trial being put on hold.


“While improving chemistry could address these issues, the potential for these AEs to be a class effect can't be ruled out increasing the risk across the pipeline,” the analyst mentioned.

Management has made improvements to antisense, using next generation ligand-conjugated antisense chemistry, which the analyst believes could represent a long-term solution for any significant issues.

Lebowitz, however, expressed optimism regarding nusinersen, which is estimated to have sales potential of over $1.3 billion in 2020.

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Aug 2016Morgan StanleyInitiates Coverage onEqual-weight
Aug 2016BarclaysMaintainsEqual-weight
May 2016BMO CapitalDowngradesOutperformMarket Perform

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