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BioWorld - Saturday, December 20, 2025
Home » Topics » Drugs » Small molecule

Small molecule
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Zevra’s arimoclomol on track as GeMDAC backs NPC drug by 11-5

Aug. 2, 2024
By Randy Osborne
As the Sept. 21 PDUFA date looms for arimoclomol from Zevra Therapeutics Inc. in Niemann-Pick type C (NPC), the U.S. FDA’s newly formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) decided in favor of the drug.
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Drug discovery illustration
Cancer

Deazaflavin analog reverses MRP1-mediated drug resistance

Aug. 2, 2024
Researchers from the University of Minnesota have presented the discovery and preclinical characterization of a novel potent inhibitor of multidrug resistance-associated protein 1 (MRP1), ZW-1226, that is being developed as a therapeutic candidate for the treatment of multidrug resistant (MDR) cancers.
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Musculoskeletal

Z-526 alleviates chemotherapy-induced cachexia muscle atrophy in vivo

Aug. 2, 2024
Researchers from East China Normal University and Fudan University presented the discovery of a new oral compound, Z-526, being developed as a treatment to alleviate chemotherapy-induced cachectic muscle loss.
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3D illustration of cancer in crosshairs
Cancer

Nur77 ligandNB-1 has anticancer activity in preclinical models

Aug. 1, 2024
Researchers from Xiamen University and affiliated organizations have presented the discovery of NB-1, a novel orally active Nur77 ligand being developed for the treatment of cancer.
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New FDA adcom to consider Niemann-Pick drug at debut meeting

July 31, 2024
By Mari Serebrov
Zevra Therapeutics Inc. will make its case Aug. 2 for its Niemann-Pick type C (NPC) candidate, arimoclomol, when the U.S. FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) meets for the first time.
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Hands holding holographic intestine
Inflammatory

Ensho debuts to target gut inflammation with oral α4β7 inhibitors

July 31, 2024
By Marian (YoonJee) Chu
Ensho Therapeutics Inc. launched in July after licensing a pipeline of four oral α4β7 inhibitors for inflammatory and gastrointestinal disorders, including inflammatory bowel disease (IBD), from EA Pharma Co. Ltd. “Millions of people worldwide are living with IBD,” Ensho founder, president and executive chair Neena Bitritto-Garg recently told BioWorld, “and while there are a number of approved medications to address the symptoms of IBD, it remains a difficult-to-treat disease with high relapse rates for a considerable proportion of patients.”
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3D illustration showing tumor inside prostate gland and closeup view of cancer cells
Cancer

BKIDC-1553 shows promise in prostate cancer models

July 31, 2024
Researchers from University of Washington presented the discovery of novel bumped kinase inhibitor (BKI) derived compound (BKIDC), BKIDC-1553, being developed for the treatment of prostate cancer.
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Illustration of brain in head highlighting the blood-brain barier.
Neurology/psychiatric

Montara secures seed funding for binary brain pharmacology

July 31, 2024
Montara Therapeutics Inc. has received $8 million seed financing from several investors to further its development of its ’binary pharmacology’ for diseases of the brain.
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Joint pain illustration
Musculoskeletal

STX-0119 shows therapeutic potential in models of osteoarthritis

July 31, 2024
Researchers from the Second Affiliated Hospital of Fujian Medical University and Shaoxing People's Hospital presented preclinical data for the signal transducer and activator of transcription 3 (STAT3) inhibitor STX-0119, which has been previously validated in models of cancer.
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Hands holding holographic intestine
Newco news

Ensho debuts to target gut inflammation with oral α4β7 inhibitors

July 30, 2024
By Marian (YoonJee) Chu
Ensho Therapeutics Inc. launched in July after licensing a pipeline of four oral α4β7 inhibitors for inflammatory and gastrointestinal disorders, including inflammatory bowel disease (IBD), from EA Pharma Co. Ltd. “Millions of people worldwide are living with IBD,” Ensho founder, president and executive chair Neena Bitritto-Garg recently told BioWorld, “and while there are a number of approved medications to address the symptoms of IBD, it remains a difficult-to-treat disease with high relapse rates for a considerable proportion of patients.”
Read More
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