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IONIS PHARMACEUTICALS, INC.

(IONS)
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Ionis Pharma Shares Drop 12% After Tofersen Study Misses Primary Endpoint

10/18/2021 | 10:41am EST

By Chris Wack

Ionis Pharmaceuticals Inc. shares fell 12% to $30.90 after the company said a study for tofersen missed its primary endpoint.

The company said its partner Biogen reported topline results from its placebo-controlled study of tofersen, an investigational antisense medicine being evaluated for people with superoxide dismutase 1 amyotrophic lateral sclerosis.

Tofersen didn't meet its primary endpoint, but trends favoring tofersen were seen across multiple secondary and exploratory measures of disease activity and clinical function.

Biogen said a pre-specified integration of data from the study and its ongoing open-label extension study reinforced the findings, and showed that early tofersen initiation led to less decline across multiple measures including motor function, respiratory function, muscle strength, and quality of life in people with SOD1-ALS.

Biogen, which licensed tofersen from Ionis in 2018, said it is actively engaging with regulators, the medical community, patient advocacy groups and other key stakeholders to determine next steps.

Biogen said it will expand eligibility for its ongoing early access program to all people with SOD1-ALS in countries where such programs are permitted by local regulations and future access may be secured. If a path forward for tofersen isn't established or if another controlled trial is required by regulators, Biogen said it could revise or discontinue the EAP.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

10-18-21 1040ET

Stocks mentioned in the article
ChangeLast1st jan.
BIOGEN INC. -2.89% 242.9 Delayed Quote.-0.80%
IONIS PHARMACEUTICALS, INC. -2.39% 29.05 Delayed Quote.-48.62%
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Financials (USD)
Sales 2021 605 M - -
Net income 2021 -254 M - -
Net Debt 2021 409 M - -
P/E ratio 2021 -15,6x
Yield 2021 -
Capitalization 4 102 M 4 102 M -
EV / Sales 2021 7,45x
EV / Sales 2022 7,77x
Nbr of Employees 757
Free-Float 99,2%
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Mean consensus OUTPERFORM
Number of Analysts 20
Last Close Price 29,05 $
Average target price 51,58 $
Spread / Average Target 77,6%
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Managers and Directors
Brett P. Monia Chief Executive Officer & Director
Elizabeth L. Hougen Chief Financial Officer & Executive VP-Finance
Joseph Loscalzo Chairman
C. Frank Bennett Chief Scientific Officer & Executive VP
Sanjay Bhanot Chief Medical Officer
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