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BioWorld - Tuesday, December 16, 2025
Home » Topics » Drugs » Small molecule

Small molecule
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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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Daewoong researchers in the lab

Daewoong sees end of CS Pharma deal; new starts in cancer, DTx

April 1, 2025
By Marian (YoonJee) Chu
It was a mixed day for Daewoong Pharmaceutical Co. Ltd., which disclosed separately on March 28 both the termination of a $336 million licensing deal with CS Pharmaceuticals Ltd. and upbeat plans to unveil three new oncology assets at the upcoming American Association for Cancer Research in April.
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Phase III primary endpoint data disappoint Axsome but subgroup beckons

April 1, 2025
By Lee Landenberger

Despite missing the primary endpoint in a phase III study of solriamfetol in major depressive disorder, Axsome Therapeutics Inc. still sees a path to another phase III study. It’s a path some analysts and investors are concerned about, though its precision-medicine approach got plenty of nods of approval.


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Lungs

Street can a-GRI on phase IIb safety win in IPF with tazarotene

April 1, 2025
By Randy Osborne
GRI Bio Inc.’s oral version of tazarotene is showing phase IIa promise in idiopathic pulmonary fibrosis (IPF). The peek at interim safety with the compound, a RAR-βɣ dual agonist designed to inhibit the activity of human type 1 invariant natural killer T cells, consisted of a preplanned analysis of two-week results in the biomarker study. GRI-0621 at 4.5 mg once daily proved safe and well-tolerated in the first 12 patients.
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3d illustration of ovarian cancer

Corcept looks to NDA following strong phase III ovarian cancer data

March 31, 2025
By Lee Landenberger
Relacorilant, a cortisol modulator from Corcept Therapeutics Inc., produced new and strong phase III results in treating platinum-resistant ovarian cancer. The company also has relacorilant in studies that could eventually lead to approval in other indications. In the near term, though, the positive data led to the company’s stock (NASDAQ:CORT) closing 109% higher at $114.22 per share on March 31.
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Doctor measuring patient's waist
Endocrine/metabolic

Novo Nordisk licenses Lexicon’s preclinical obesity drug in $1B deal

March 31, 2025
By Karen Carey
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
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Daewoong researchers in the lab

Daewoong sees end of CS Pharma deal; new starts in cancer, DTx

March 28, 2025
By Marian (YoonJee) Chu
It was a mixed day for Daewoong Pharmaceutical Co. Ltd., which disclosed separately on March 28 both the termination of a $336 million licensing deal with CS Pharmaceuticals Ltd. and upbeat plans to unveil three new oncology assets at the upcoming American Association for Cancer Research in April.
Read More
Doctor measuring patient's waist

Novo Nordisk licenses Lexicon’s preclinical obesity drug in $1B deal

March 28, 2025
By Karen Carey
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
Read More

Myst it again: Milestone gets a CRL for its heart spray

March 28, 2025
By Lee Landenberger
Milestone Pharmaceuticals Inc.’s heart rhythm-restoring calcium channel blocker for paroxysmal supraventricular tachycardia (PSVT) has yet another obstacle in its way. 
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