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BioWorld - Tuesday, March 31, 2026
Home » Topics » Drugs » Small molecule

Small molecule
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Sanofi lever hoists Fulcrum in $1B-plus FSHD pact

May 13, 2024
By Randy Osborne
Fulcrum Therapeutics Inc.’s deal with Sanofi SA to develop and commercialize oral losmapimod shone more light on facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disease where Avidity Biosciences Inc. also has an earlier-stage but high-profile program.
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Endometriosis

Tiumbio’s Merigolix hits phase II endpoint for endometriosis pain

May 9, 2024
By Marian (YoonJee) Chu
Tiumbio Co. Ltd., of Seongnam-si, Gyeonggi-do, reported positive phase IIa top-line data on May 8 for Merigolix, its lead asset to reduce pain in female patients with moderate to severe endometriosis.
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Test tube, dropper
Cancer

MS-8535 is epigenetic SPIN1 inhibitor

May 9, 2024
Researchers from the Icahn School of Medicine at Mount Sinai and affiliated organizations presented the discovery and preclinical characterization of MS-8535, a novel spindlin-1 (SPIN1) inhibitor being developed as chemical tool anticancer agent.
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Justin Hanka, CEO, Mindbio
Newco news

Mindbio explores psychedelic potential of LSD for depression, PMS

May 7, 2024
By Tamra Sami
The use of psychedelics is seeing impressive results in treating psychiatric disorders like treatment-resistant depression and post-traumatic stress disorder, and Mindbio Therapeutics Ltd. is expanding the field to include microdoses of lysergic acid diethylamide (LSD) to treat depression and premenstrual syndrome.
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Jacobio seeks China approval of second-line KRAS lung cancer drug

May 7, 2024
By Marian (YoonJee) Chu
China and U.S. biotech Jacobio Pharmaceuticals Group Co. Ltd.’s shares (HKEX:1167) rose nearly 20% after it filed an NDA in China on May 6 for its independently developed KRAS G12C inhibitor, glecirasib (JAB-21822), to treat lung cancer.
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Musculoskeletal

Vyne’s BD2-selective BET inhibitor cleared by FDA for phase I

May 7, 2024
Vyne Therapeutics Inc. has received IND clearance from the FDA allowing it to initiate a first-in-human phase Ia study of VYN-202, an oral small-molecule BD2-selective BET inhibitor for autoimmune diseases.
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Acute myeloid leukemia

Glycomimetics wilts on missed acute myeloid leukemia phase III

May 6, 2024
By Lee Landenberger
Top-line data from Glycomimetics Inc.’s pivotal phase III study of uproleselan in 388 patients with relapsed/refractory acute myeloid leukemia missed its primary endpoint. The data crippled the stock, which closed at the low point of its 10-year existence.
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Cancer

Pan-TEAD palmitoylation inhibitor suppresses tumor growth in Hippo pathway-altered cancer models

May 3, 2024
The TEAD family of transcription factors are regulated by the Hippo tumor suppressor pathway and they act by binding the co-activators YAP and TAZ that drive the transcription of genes involved in cell survival, proliferation, migration, differentiation and resistance.
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Allarity’s stenoparib positive in late-stage ovarian cancer

May 2, 2024
By Karen Carey
Achieving proof of concept and a substantial clinical benefit with its PARP inhibitor stenoparib in advanced recurrent ovarian cancer, Allarity Therapeutics Inc. stopped a phase II trial and is preparing for a registrational study for what is now the company’s only internal drug candidate.
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Cancer

New TLR2/1 agonist induces pyroptosis in cancer cells

May 2, 2024
Researchers from the Beijing Institute of Radiation Medicine have presented the discovery of novel toll-like receptor (TLR)2/1 agonists as potential candidates for the treatment of lung cancer.
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