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BioWorld - Wednesday, December 17, 2025
Home » Topics » Drugs » Small molecule

Small molecule
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Neil Miller, CEO, NRG Therapeutics
Neurology/psychiatric

NRG raises $67M to test mitochondrial pore inhibitor in ALS

Sep. 9, 2025
By Nuala Moran
No Comments
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
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Neil Miller, CEO, NRG Therapeutics

NRG raises $67M to test mitochondrial pore inhibitor in ALS

Sep. 8, 2025
By Nuala Moran
No Comments
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
Read More
Digital handshake

Servier in $450M deal for Medicxi-funded phase II fragile X program

Sep. 8, 2025
By Nuala Moran
No Comments
Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.
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Digital heart illustration

Braveheart licenses Hengrui's heart drug HRS-1893 in $1B deal

Sep. 5, 2025
By Marian (YoonJee) Chu
No Comments
Braveheart Bio Inc. is paying $65 million up front to license Jiangsu Hengrui Pharmaceuticals Co. Ltd.’s oral hypertrophic cardiomyopathy drug candidate called HRS-1893.
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Skyvaricella

South Korea INDs: Pimedbio, Sillajen, Genosco, SK Bio, Ami

Sep. 3, 2025
By Marian (YoonJee) Chu
No Comments

Several South Korean biotech and biopharmaceutical companies completed IND submissions or won nods to start clinical trials in either the U.S. or South Korea, including SK Bioscience Co. Ltd., Genosco Inc., Pimedbio Inc., Sillajen Inc. and Ami Pharm Co. Ltd.


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Stock table, gold arrow pointing down

Argenica’s stock drops 56% on mixed phase II results

Sep. 3, 2025
By Tamra Sami
No Comments
Argenica Therapeutics Ltd. saw its stock drop 56% following mixed results for its ARG-007 phase II Seancon trial in acute ischemic stroke patients undergoing endovascular revascularization, or thrombectomy.
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Liver and lungs

Wave Life says data are positive but stock wavers

Sep. 3, 2025
By Lee Landenberger
No Comments
As Wave Life Sciences Ltd. released more results from its ongoing phase Ib/IIa study of small interfering RNA editing oligonucleotide WVE-006 for treating alpha-1 antitrypsin deficiency, the company’s stock (NASDAQ:WVE) dropped by 16.8% to close at $8 on Sept. 3.
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Fosun out-licenses immunology candidate to Sitala in $675M deal

Sep. 2, 2025
By Marian (YoonJee) Chu
No Comments
Shanghai Fosun Pharmaceutical (Group) Co. Ltd.’s subsidiary, Shanghai Fosun Pharma Industrial, signed off ex-China rights to a phase II small-molecule inhibitor, FXS-6837, to Sitala Bio Ltd. in a potential $675 million deal.
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AI biotech research concept with lightbulb and digital background

Charm raises $80M series B to develop better menin inhibitors using AI

Sep. 2, 2025
By Nuala Moran
No Comments
Charm Therapeutics Ltd. is heading to the clinic after closing an oversubscribed series B, which will fund initial development of a menin inhibitor that is AI-designed to circumvent shortcomings of first-generation molecules.
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Tyvaso nebulizer

United’s Tyvaso surprise in IPF potentially opens $4B market

Sep. 2, 2025
By Karen Carey
No Comments
Following positive data from competitors earlier this year, United Therapeutics Corp. rolled out impressive and unexpected phase III results of nebulized Tyvaso (treprostinil) for idiopathic pulmonary fibrosis (IPF), setting the stage for an sNDA filing with the U.S. FDA next year.  If approved for the indication, United, of Silver Spring, Md., and Research Triangle Park, N.C., could tap into what analysts say is a $4 billion-plus market.
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