CDC.govSwiss clinical-stage biotech Vaderis Therapeutics has raised $152 million in a Series B round to advance engasertib into Phase 3 for hereditary hemorrhagic telangiectasia (HHT). The financing, backed by a broad syndicate of specialist investors, gives the company a potentially decisive runway toward registration for a disease with no approved therapy.
Tag Archive for: Rare disease
Unsplash+Ipsen has announced the immediate withdrawal of its cancer drug Tazverik across all indications after serious safety issues emerged in an ongoing Phase III trial.
argenx delivered two significant updates on the same day: positive Phase 3 results in ocular myasthenia gravis (oMG) and its first full year of operating profitability. Together, the announcements show a company expanding its lead medicine into a new patient population while doing so from a position of financial strength.
The European Commission has approved Amgen’s Uplizna in generalized myasthenia gravis, moving the big biotech into a market served by argenx, Johnson & Johnson and UCB.
Researchers at NDM Pharma A/S and Aarhus University have demonstrated that their oral CIC-1 blocker NMD670 restores crosstalk between neurons and muscles in Myasthenia gravis.
Rare disease specialist SynaptixBio Ltd has got its second FDA orphan drug designation for a subtype of TUBB4A leukodystrophy.