Keyword: Ionis Pharmaceuticals
Ionis signed on with Roche to develop its RNA drug for advanced dry AMD, in a deal worth up to $760 million, after being snubbed by GSK last year.
Akcea has its first FDA approval for hATTR therapy Tegsedi but will be burdened with a black box warning as it chases rival Alnylam.
The interim readout showed that two-fifths of infants who received PTC and Roche’s oral SMA drug were sitting eight months after starting treatment.
Akcea reported topline data showing its hyperlipoproteinemia drug lowered lipoprotein(a) levels in a phase 2 study in patients with cardiovascular disease.
The cuts come 10 days after the FDA rejected a filing for approval of ultrarare lipid disorder drug volanesorsen.
Swedish researchers have found that the rogue SOD1 protein can “transmit” protein misfolding in ALS.
The FDA rejected Akcea’s volanesorsen three months after the drug squeaked past an FDA advisory committee that voted 12-8 to recommend its approval.
Pfizer’s ATTR drug tafamidis has hit the mark in a phase 3 trial, setting up regulatory filings and a challenge to rivals Alnylam and Ionis/Akcea.
The week’s biotech news included a mutation-targeting treatment for ALS and a malaria vaccine targeting a transmission protein.
Orion Pharma is pressing ahead with a pivotal trial for its amyotrophic lateral sclerosis drug ODM-109, despite mixed results in a phase 2 trial.