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BioWorld - Monday, December 22, 2025
Home » Topics » Drugs » Small molecule

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Cerebellum, brain stem, spinal cord

Alterity’s ATH-434 shows early response in multiple system atrophy

July 18, 2024
By Tamra Sami
Aussie biotech Alterity Therapeutics Ltd. released interim data of an open-label phase II trial for lead candidate ATH-434 for treating multiple system atrophy that showed 43% of participants improved, with 29% showing either stable or improved neurological symptoms.
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Kidney disease illustration

Vivoryon eyes phase III after kidney win with varoglutamstat

July 18, 2024
By Randy Osborne
About four months after varoglutamstat failed in Alzheimer’s disease, Vivoryon Therapeutics NV disclosed data from the Viviad phase IIb study with the same compound in kidney disease that provided cause for hope – and Anne Doering, chief financial officer, said the new data “reinforce our strategic shift.”
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Blue dollar sign on white background

Cardurion’s novel cardiovascular pipeline draws $260M series B

July 17, 2024
By Jennifer Boggs
In one of the largest venture rounds for biopharma in 2024, Cardurion Pharmaceuticals Inc. closed a $260 million series B financing, with funds slated to advance and expand its pipeline for potentially first-in-class drugs targeting cardiovascular disease, including two programs in phase II development.
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SK Biopharm grows radiopharma reach with $571.5M Full-Life deal

July 17, 2024
By Marian (YoonJee) Chu
As the “most concrete achievement” since its entry into the radiopharmaceutical therapy space last year, SK Biopharmaceuticals Co. Ltd. picked up rights to Full-Life Technologies Ltd.’s RPT asset, FL-091, in a deal fetching up to $571.5 million.
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Renalys’ ¥6B series A to fund phase III IgAN trial in Japan

July 17, 2024
By Tamra Sami
Renalys Pharma Inc. completed a ¥6 billion (US$38.199 million) series A round that will enable it to complete a phase III trial for sparsentan for immunoglobulin A nephropathy (IgAN) in Japan. Tokyo-based Renalys in-licensed rights to sparsentan in January 2024 from Travere Therapeutics Inc. to develop the compound in Japan and 13 Asian countries to treat IgAN, a rare kidney disease.
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Illustration of tau accumulating in a neuron cell.

Asceneuron raises $100M for OGA inhibitor work in Alzheimer’s

July 16, 2024
By Nuala Moran
Asceneuron SA has raised $100 million in an oversubscribed series C to take its lead small molecule, ASN-51, into phase II, with aim of demonstrating it prevents the formation of tau tangles and slows the progression of Alzheimer’s disease.
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Brain scans

Kazia’s stock rockets on positive glioblastoma trial results

July 16, 2024
By Tamra Sami
Kazia Therapeutics Ltd.'s stock shot up 248% on July 10 after the Sydney-based company reported that paxalisib showed a 3.8-month improvement in overall survival, a 33% improvement, for newly diagnosed unmethylated patients with glioblastoma (GBM) compared to the concurrent standard-of-care arm in the pivotal GBM-Agile phase II/III study.
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Brain scans

Kazia’s stock rockets on positive glioblastoma trial results

July 11, 2024
By Tamra Sami
Kazia Therapeutics Ltd.'s stock shot up 248% on July 10 after the Sydney-based company reported that paxalisib showed a 3.8-month improvement in overall survival, a 33% improvement, for newly diagnosed unmethylated patients with glioblastoma (GBM) compared to the concurrent standard-of-care arm in the pivotal GBM-Agile phase II/III study.
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South Africa drops anticompetition complaint against J&J

July 10, 2024
By Mari Serebrov
Having secured a 40% price cut and a commitment to not enforce a patent protecting a tuberculosis drug, South Africa’s Competition Commission decided not to prosecute a complaint accusing Johnson & Johnson (J&J) and its subsidiary, Janssen Pharmaceutica (Pty) Ltd., of anticompetitive conduct.
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First Circuit: Frequency class action brushstrokes no ‘van Gogh’

July 9, 2024
By Mari Serebrov
Just because a statement turns out to be false doesn’t mean it was intended to be, the U.S. Court of Appeals for the First Circuit said in tossing a stockholder class action against Frequency Therapeutics Inc., now part of Korro Bio Inc.
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