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Home » Topics » Drugs » Small molecule

Small molecule
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Glycomine snags $115M to advance its PMM2-CDG treatment

April 17, 2025
By Brian Orelli
Glycomine Inc. secured $115 million in a series C financing round to support the advancement of GLM-101 into a phase IIb study in patients with phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG).
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Man piecing together a puzzle
Neurology/psychiatric

Discovery and preclinical results of a Rac1 inhibitor for Alzheimer’s

April 17, 2025
At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), researchers from Beijing Joekai Biotechnology LLC presented the discovery process and preclinical results for 50561.
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Pills, bottle on $100 bills

New Trump order zeroes in on drug prices

April 16, 2025
By Mari Serebrov
In a blast from the past, U.S. President Donald Trump signed an executive order April 15 to deliver on his 2016 campaign promises and strengthen or reinstate efforts of his first administration to drive down prescription drug prices. “My first term included numerous significant actions, including some of the most aggressive in recent history, to deliver lower prescription drug prices to American patients,” Trump noted in the order, which builds on many of those actions, including increased competition, re-importation, price transparency and a mandate to pass discounts through to patients.
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Tryptamine to test injectable psilocybin for binge eating disorder

April 15, 2025
By Tamra Sami
Tryptamine Therapeutics Ltd. is gearing up to enter the clinic with lead compound TRP-8803, an intravenous-infused psilocybin therapy, in patients with binge eating disorder in conjunction with psychotherapy.
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Pfizer’s loss offers boost to competitors in obesity space

April 14, 2025
By Jennifer Boggs
Pfizer Inc. is ending work on oral GLP-1 candidate danuglipron for weight loss following the report of a single potentially drug-induced liver injury, a move that appears to open the door for other firms working on oral therapies in the high-dollar obesity space, even as industry watchers seek further details to determine whether similar safety signals could emerge for those competitors.
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Cancer cell targeted in crosshairs
Cancer

Specifically targeting TUBG1 shows potential in RB1-deficient tumors

April 11, 2025
Researchers from Lund University and collaborators have investigated the potential of selectively targeting TUBG1 as a therapeutic strategy in cancer treatment.
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Female hands holding heart on cardiogram
Endocrine/metabolic

Northstrive partners with Yuva Biosciences on obesity and cardiometabolic diseases

April 11, 2025
Northstrive Biosciences Inc., a subsidiary of PMGC Holdings Inc., has entered into a development and license agreement with Yuva Biosciences Inc. to discover and develop novel pharmaceutical treatments for obesity, type 2 diabetes and other cardiometabolic conditions using Yuva’s mitochondrial science-focused platform Mitonova, powered by AI.
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Tryptamine to test injectable psilocybin for binge eating disorder

April 10, 2025
By Tamra Sami
Tryptamine Therapeutics Ltd. is gearing up to enter the clinic with lead compound TRP-8803, an intravenous-infused psilocybin therapy, in patients with binge eating disorder in conjunction with psychotherapy.
Read More
Elderly woman and jigsaw puzzle

Alzheon’s lead candidate misses a phase III in early Alzheimer’s

April 10, 2025
By Lee Landenberger
Alzheon Inc.’s oral treatment for people in the early stages of Alzheimer’s disease missed its phase III primary endpoint, adding yet another therapy to a long list by many developers that can’t beat dementia. The study also received grant money, which is in increasingly short supply.
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Neurosense phase II combo to hail new Paradigm in ALS?

April 9, 2025
By Randy Osborne
As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.
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