Preclinical neuroscience firm Delix Therapeutics Inc., a startup developing non-hallucinogenic analogues of psychedelics, has closed a $70 million series A financing. The new funds will help the Boston-based company advance two lead candidates through phase I trials, DLX-1 and DLX-7, with studies planned for starts in late 2022 or early 2023.
DUBLIN – Treefrog Therapeutics SA closed a $75 million series B round this week, which will help to increase its reach and its profile, as it pursues its highly ambitious objective to drive the adoption of a new way of making induced pluripotent stem cells (iPSCs) at scale. The Bordeaux, France-based firm is not a CDMO in any sense, however. It is a fully fledged biotech, with early stage iPSC-based programs in Parkinson’s disease, cardiovascular disease and bone marrow transplant, among others. It’s just that it is also attempting to revolutionize how those cells are cultivated before it administers them as therapies.
European regulators have rejected Pfizer Inc.’s tanezumab, casting further doubts on whether the FDA will okay the troubled drug that is intended as a non-opioid alternative for osteoarthritis pain.
Rune Labs Inc. is on a mission to make the mysteries of the brain easier to read for both clinicians and biopharma companies. Having a fresh $22.8 million in a series A financing on the books will no doubt make the goal easier to reach.
Chicago-based Vanqua Bio Inc. has raised $85 million in series B financing to accelerate development of its therapies tackling Parkinson’s and other neurodegenerative diseases. Founded in 2019, Vanqua’s technology is focused on developing small-molecule activators of glucocerebrosidase, an enzyme that regulates levels of lipids in cells.
A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy.
Proqr Therapeutics NV sealed a deal for genetic disorders in the liver and nervous system with Eli Lilly and Co., collecting $50 million in the form of an up-front payment ($20 million) and an equity investment ($30 million), with the prospect of about $1.25 billion more if the arrangement hits research, development and commercialization goals. “The milestones are pretty evenly spread out,” said Smital Shah, Proqr’s chief business and financial officer, though details weren’t provided.
The phase III fizzle in autism spectrum disorder (ASD) by French firms Les Laboratoires Servier SAS and Neurochlore SAS was the latest in a disease that has proved challenging because of its heterogeneity, which – in the words of Yehezkel Ben-Ari, president of Neurochlore – “probably makes it impossible to offer a sole treatment for all autistic children.”
The FDA has approved Trudhesa (dihydroergotamine mesylate [DHE]; INP-104) from Impel Neuropharma Inc. for treating acute migraine headaches with or without aura in adults. The approval comes as several competitors, including Axsome Therapeutics Inc and Abbvie Inc., have NDAs submitted or approved for new therapies to treat migraine.
Astellas Pharma Inc. has halted further dosing of the experimental X-linked myotubular myopathy (XLMTM) gene therapy AT-132 (resamirigene bilparvovec) after one participant in the ongoing Aspiro study experienced a serious adverse event (SAE) of abnormal liver function.