Remix Therapeutics Inc., a company developing small molecules to manipulate RNA processing, stands to earn upward of $1 billion through a new strategic collaboration with Janssen Pharmaceutica NV. A Cambridge, Mass.-based startup, Remix last year patented new RNA splicing modulators. It will receive an initial payment of $45 million for research funding plus potential preclinical, clinical, commercial and sales milestone payments, and royalties. Janssen gains exclusive rights to three targets with applications in immunology and oncology.
Third Harmonic Bio Inc. CEO Natalie Holles said the company’s $105 million in series B money will push THB-001 – a first-in-class, highly selective, oral inhibitor of wild-type KIT – “well past the first proof-of-concept study in inducible urticaria [hives].”
LONDON – Neuromuscular disease specialist NMD Pharma A/S has raised €35 million (US$39.7 million) in a new financing, as it awaits initial data from its first clinical trial, in the treatment of myasthenia gravis. The new money enables NMD to complete that phase IIa study and to launch another trial of the same compound, NMD-670, in spinal muscular atrophy. The Aarhus, Denmark-based company is preparing the IND and aims to treat the first patient before the end of 2022.
In the bad old days of cancer treatment, it was the nasty side effects of chemotherapy that often ended up limiting treatment – and while checkpoint inhibitors have raised the bar in terms of efficacy and safety, they can have dangerous and unpleasant off-target consequences, too. Finding ways to focus the immune system on cancer and limiting any off-target effects will not only make checkpoint inhibitor therapy more tolerable but could also improve survival rates – and Sweden’s Ilya Pharma AB aims to do this with a novel approach combining synthetic biology with cell therapy.
These days it’s nearly impossible to turn around in the biopharma world without hearing about how some company is going to use machine learning to revolutionize drug development. “It really is a catchphrase,” acknowledged Jo Viney, whose latest startup, Seismic Therapeutic Inc. launched with a $101 million series A round to advance a platform incorporating machine learning capabilities to find new drugs for autoimmune diseases.
Ventus Therapeutics Inc. closed a $140 million series C financing to continue scaling its platform to address previously undruggable targets. The company said it plans to advance programs targeting key modulators of the innate immune system and other therapies, including two targeting NLRP3 (NLR pyrin domain-containing 3) and one targeting cGAS, into the clinic. It plans to submit INDs for three programs in 2023.
The U.S. FDA has approved Sanofi SA’s treatment for cold agglutinin disease (CAD), sutimlimab, after the drug was initially rejected by the regulator for technical reasons in 2020. Paris-based Sanofi’s drug will be branded as Enjaymo.