Ionis Pharmaceuticals, Inc. Common Stock IONS

Ionis Pharmaceuticals, Inc. se dedica a descubrir y desarrollar terapéuticas dirigidas al ácido ribonucleico (ARN). La Compañía, utilizando su plataforma de descubrimiento de medicamentos, ha desarrollado una cartera de medicamentos para pacientes con necesidades médicas no satisfechas. Los segmentos de la Compañía incluyen Ionis Core y Akcea Therapeutics. En el segmento Ionis Core, la Compañía se dedica a explotar una plataforma de descubrimiento de medicamentos para generar una cartera de medicamentos para la Compañía y sus socios. El segmento Akcea Therapeutics incluye las operaciones de la filial de la Compañía, Akcea Therapeutics, Inc. (Akcea Therapeutics). Akcea Therapeutics se enfoca en desarrollar y comercializar volanesorsen y otros medicamentos en etapa clínica para enfermedades cardiometabólicas graves causadas por trastornos lipídicos. La Compañía está desarrollando volanesorsen para tratar dos enfermedades severas y raras, genéticamente definidas, la quilomicronemia familiar (FCS) y la lipodistrofia parcial familiar (FPL). La Compañía ofrece SPINRAZA, un medicamento antisentido de Generación 2.0+.

• Zacks Investment Research 5 oct

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• Zacks Investment Research 23 sept

  Ionis Delivers Positive Late-Stage Results for Two Partnered Drugs

  Ionis Pharmaceuticals announced positive phase III results for two partnered programs: ulefnersen (FUSION study) for FUS-ALS met its primary endpoint with Otsuka planning regulatory discussions, and sefaxersen (IMAgINATION study) for IgAN showed statistically significant proteinuria reductions with Roche continuing the trial. These wins come after recent clinical setbacks in cardiovascular programs but follow FDA approval of Zanvastro for Alexander disease.

  

• The Motley Fool 20 sept

  Investors Dumped Ionis Pharmaceuticals Stock -- But Wall Street Hasn't Budged From Its Bullish Outlook

  Ionis Pharmaceuticals stock has plummeted over 40% this year due to late-stage clinical trial failures and ongoing losses, yet Wall Street analysts maintain a bullish outlook with an average price target of $83.82. Despite setbacks with pelacarsen and eplontersen programs, the company's strong ASO technology platform, solid $2.1 billion cash position, and recent FDA approval of Zanvastro for Alexander disease provide potential for long-term investors willing to wait several years for profitability.