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BioWorld - Friday, March 20, 2026
Home » Topics » Drugs » Small molecule

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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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Debate over Orange Book device listings heard on multiple fronts

May 1, 2024
By Mari Serebrov
Just a few days after the U.S. Congressional Research Service issued a report suggesting ways Congress could resolve the unanswered questions about patent listings in the FDA’s Orange Book, the FTC sent a second round of warning letters to eight biopharma companies and their subsidiaries, citing the listing of device patents for combination products.
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Female sitting on floor in dark room

Nrx plans suicidal bipolar depression study despite phase II/III trouble

May 1, 2024
By Lee Landenberger
While Nrx Pharmaceuticals Inc.’s oral antidepressant NRX-101 failed, producing a not statistically significant 33% reduction in suicidality along with a 70% reduction in the symptoms of akathisia, the company said it plans to forge ahead and conduct a registrational study.
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Drug capsule spilling onto brain

Newron hails ‘groundbreaking’ evenamide data in schizophrenia

April 30, 2024
By Nuala Moran
Newron Pharmaceuticals SpA added heft to the data supporting evenamide, reporting positive top-line results from a phase II/III placebo-controlled trial of the glutamate inhibitor in patients with chronic schizophrenia who were receiving a second-generation antipsychotic, but having an inadequate response to that treatment.
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Korea-US biotech Genosco passes technology review for Kosdaq IPO

April 30, 2024
By Marian (YoonJee) Chu
Boston-based South Korean biotech Genosco Inc. said on April 25 that it passed a technology review required for the special listing track on the tech-heavy Kosdaq market, as it readies an IPO on the Korea Exchange.
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Infection

Jun-12682, a SARS-CoV-2 PLpro inhibitor with potent antiviral activity in mice

April 30, 2024
Researchers from State University of New Jersey (Rutgers) and Oklahoma State University have published preclinical data for a novel a SARS-CoV-2 papain-like protease (PLpro) inhibitor being developed as an antiviral candidate for the treatment of COVID-19.
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