Emalex Biosciences Inc. closed an upsized and oversubscribed $250 million series D funding round intended to fund a phase III trial and potential commercialization of ecopipam, its dopamine-1 receptor antagonist for Tourette syndrome, a neurological disorder characterized by involuntary motor and vocal tics. The company was created by Paragon Biosciences in 2018 to develop novel treatments for central nervous system disorders.

Abeona’s positive phase III data and $35M private placement can’t support the stock

Abeona Therapeutics Inc. is on the road to filing a BLA with the U.S. FDA after posting positive top-line phase III data in wound healing and also to the bank with a new $35 million private placement financing. The data for EB-101, an autologous cell therapy, came from a pivotal study of treating recessive dystrophic epidermolysis bullosa, a rare connective tissue disorder. Results showed the study met its two co-primary endpoints in wound healing and for reducing pains in large, chronic wounds caused by the disorder. Abeona said it will sell about 7 million shares of its common stock as it looks for about $35 million in gross proceeds. Investors stepped away from the shares Nov. 3 as the stock (NASDAQ:ABEO) had dropped 20% at midday to about $3.50 each.

No more surprises, please! COVID-19 loosens grip on humanity

While weekly global and U.S. confirmed cases of COVID-19 are below each of the last two years, infectious disease experts remain on guard. There are still about 1,500 people dying around the world each day, including 350 in the U.S., and the SARS-CoV-2 virus may continue to find ways to outmaneuver current treatments and vaccines. While COVID-19 is loosening its grip, the biopharma industry continues to research some of the 1,093 therapeutics and vaccines brought forward since the pandemic began. Of those, 269 have had news this year, suggesting development is inactive or stalled for hundreds of others.

Affimed stock rises on AFM13 lymphoma data, NK cell therapy pact with Artiva

Shares in Affimed AG gained as much as 28% during trading on Nov. 3 as the company unveiled continued good news from a phase I/II combination trial in CD30-positive lymphoma of its CD30-directed innate cell engager AFM13 and allogeneic natural killer (NK) cell therapy, as well as a clinical development partnership with Artiva Biotherapeutics Inc., which will provide it with access to a commercially scalable source of NK cells as the program matures.

Compass readies phase III trial of psilocybin formulation in treatment-resistant depression

Compass Pathways plc is poised to start the first ever phase III trial of the psychedelic drug psilocybin, after getting U.S. FDA backing for a study in treatment-resistant depression. The phase III, due to start before the end of 2022, will comprise two pivotal trials and a long-term follow-up study. In the first pivotal trial, 378 patients will be randomized 2-to-1 to receive 25 mg of Comp360, a proprietary synthetic formulation of psilocybin, a drug that occurs naturally in “magic” mushrooms, or a placebo.

GSK forges on with antibiotic R&D as gepotidacin hits targets in phase III

GSK plc has announced it has stopped two pivotal phase III trials of its urinary tract infection drug, gepotidacin, early for efficacy and is preparing regulatory filings for what could be the first new oral antibiotic for the disease in more than 20 years. London-based GSK made the decision based on findings of the independent data monitoring committee (IDMC) and was based on a prespecified interim analysis of more than 3,000 patients in the trials in uncomplicated urinary tract infection (uUTI). Giving only top-line data, GSK said the Eagle-2 and Eagle-3 trials met the primary efficacy endpoint of combined clinical and microbiological resolution following treatment at the test-of-cure visit for gepotidacin vs. nitrofurantoin in patients with a confirmed uUTI and a uropathogen sensitive to nitrofurantoin. The IDMC review did not identify any safety concerns.

‘On-demand’ epilepsy gene therapy selectively calms hyperactive cells

By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures. The channels are expressed when the promoter is turned on by excessive neuronal activity, and so “we can’t stop the first seizures,” Dimitri Kullmann told BioWorld. But for many patients with poorly controlled epilepsy, seizures are not one and done. “Forty percent of patients will get multiple seizures within 24 hours,” he said. An on-demand gene therapy would be able to stop those repeat seizures. Kullmann is professor of neurology at University College London’s Queen Square Institute of Neurology and co-corresponding author of the paper reporting the results, which appeared in the Nov. 4, 2022, issue of Science.

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Abeona, Acrivon, ADC, Aelix, Aim, Albireo, Antengene, Aptinyx, Arbutus, Ascletis, Beigene, Blaze, Brexogen, Bristol Myers Squibb, Catalent, CNS, Codagenix, Crispr, Daewoong, Decibel, Edesa, Eisai, Eloxx, Emergent, Equillium, Exelixis, G1, Gilead, Hansa, IGM, Intas, IO Biotech, Kura, Lianbio, Meletios, Merck, Neurotech, Omega, Pharmala, Regulus, Renovion, Rhythm, Rigel, Sarepta, Sonnet, Surface Oncology, Syna, TFF, Tricida, Uniqure