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BioWorld - Wednesday, June 24, 2026
Home » Topics » Drugs » Small molecule

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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.
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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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IgAN again: Novartis gets second approval in rare kidney disease

April 3, 2025
By Lee Landenberger
The U.S. FDA’s accelerated approval of Vanrafia (atrasentan) from Novartis AG for primary immunoglobulin A nephropathy (IgAN) is the company’s second approval for the indication in the past year and a half. The nod also came without a required safety program through a black box warning.
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Face with digital focus on eye

Reproxalap rapped again in DED; second Aldeyra CRL

April 3, 2025
By Randy Osborne
Aldeyra Therapeutics Inc. is hoping that research already underway will satisfy the U.S. FDA, which delivered to the company another complete response letter (CRL) related to the NDA for reproxalap in dry eye disease (DED).
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Blue heart and data grid

Edgewise down on atrial fibrillation; cardiomyopathy data positive

April 2, 2025
By Lee Landenberger
Top-line data from the second and third parts of Edgewise Therapeutics Inc.’s four-part phase II Cirrus-HCM study in hypertrophic cardiomyopathy (HCM) has produced more positive data. There was however less enthusiasm for the results than there had been in September for the first batch of data.
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Gastrointestinal

PANX1 inhibitor for colitis management presented at ACS Spring 2025

April 2, 2025
Pannexin 1 (PANX1) forms channels that may release signaling metabolites that are involved in a variety of pathophysiological processes, such as asthma, diabetes, hypertension or inflammatory bowel disease (IBD), among others. Inhibition of PANX1 when dysregulated has been proposed as a therapeutic approach in the treatment of IBD.
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Beta cells
Endocrine/metabolic

Stabilizing ‘molecular glues’ protect β cells in type 2 diabetes

April 2, 2025
In type 2 diabetes patients, inadequate hyperglycemic control can lead to insulin-secreting β-cell exhaustion, dedifferentiation and eventual loss. Since adult β cells have limited proliferative capacity, this final stage cannot be reversed.
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Neurology/psychiatric

Cryptic pocket in CB1 receptor is better target for analgesia

April 2, 2025
Cannabinoid CB1 receptors have been a potential target for nonopioid-based pain treatment, but actually targeting the pathway has been hindered by issues with tolerance and unwanted CNS side effects. Peripherally selective CB1 agonists developed to overcome these problems have not fully resolved these issues, meaning the peripheral selectivity has to be substantially enhanced.
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