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BioWorld - Monday, April 20, 2026
Home » Topics » Small molecule, BioWorld

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Close up of man's eye

Qlaris’ QLS-111 reduces eye pressure in two phase II trials

Feb. 5, 2025
By Jennifer Boggs
Touting its novel approach for treating glaucoma, Qlaris Bio Inc. reported promising top-line data from two phase II studies testing QLS-111 in patients with primary open-angle glaucoma and ocular hypertension, showing the drug met all primary and secondary endpoints, reducing intraocular pressure with a clean safety profile that could encourage patients to remain on treatment.
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DMD space takes heart from Cumberland’s phase II ifetroban data

Feb. 4, 2025
By Randy Osborne
The first successful phase II trial in Duchenne muscular dystrophy (DMD) heart disease rolled out from Cumberland Pharmaceuticals Inc., which said top-line findings from the experiment called Fight DMD showed promising results in the indication that represents the main cause of death for such patients.
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Updated Maia phase II data nudges its stock higher

Feb. 4, 2025
By Lee Landenberger
New and positive data for Maia Biotechnology Inc.’s lead candidate for a particularly deadly and advanced lung cancer propelled the stock modestly higher on Feb. 4. The pivotal phase II THIO-101 study of Thio, a telomere-targeting agent sequenced with Regeneron Inc.’s immune checkpoint inhibitor, Libtayo (cemiplimab), as a third-line treatment for advanced non-small-cell lung cancer (NSCLC) showed median overall survival of 16.9 months for the 22 NSCLC patients who received at least one dose of Thio in parts A and B.
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Going public ticker

Metsera and Maze IPOs look to bring $415M to Nasdaq

Jan. 31, 2025
By Lee Landenberger
A week after the first IPO of the year was priced, obesity treatment developer Metsera Inc. and renal specialist Maze Therapeutics Inc. have begun trading on Nasdaq. Metsera (NASDAQ:MTSR) surged 47% on Jan. 31 to close at $26.50 per share while Maze (NASDAQ:MAZE) barely budged, closing three-tenths of a percentage point lower on the day.
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Journavx bottle and tablet

Vertex wins FDA nod for Journavx, new mechanism for acute pain

Jan. 31, 2025
By Jennifer Boggs
An effort two decades in the making, Vertex Pharmaceuticals Inc.’s suzetrigine gained U.S. FDA approval as the first drug targeting the NaV1.8 pain signal. Branded Journavx, the oral small molecule is cleared for use as a non-opioid option for treating moderate to severe acute pain.
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Central nervous system

Alterity’s ATH-434 looks to be disease modifying in multiple system atrophy

Jan. 30, 2025
By Tamra Sami
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
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Zentalis creates a path for its cancer study with layoffs

Jan. 29, 2025
By Lee Landenberger
It’s one step backward in order to take two steps forward at Zentalis Pharmaceuticals Inc. In a restructuring to help fund the second part of its potentially registration-enabling Denali study, Zentalis has laid off 40% of its workforce. The clinical trial of azenosertib, Zentalis’ lead candidate and a WEE1 kinase inhibitor, is for treating advanced solid tumors and hematologic malignancies. The therapy is designed to make cancer cells self-destruct.
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Gavel and pill blister packs

US Claims Court rules Vanda’s trade secrets not so secret

Jan. 24, 2025
By Mari Serebrov
A U.S. Federal Claims Court judge shot down Vanda Pharmaceuticals Inc.’s accusations that the FDA disclosed the company’s trade secrets to generic competitors, finding that the trade secrets weren’t really secret or necessarily proprietary to Vanda.
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Hand holding white pill

Aussie spinout could change blood pressure paradigm with low-dose poly pill

Jan. 23, 2025
By Tamra Sami
George Medicines, a new spinout from Australia’s George Institute for Global Health, could offer patients better control of their blood pressure as well as fewer side effects thanks to an ultra-low-dose triple combination. A polypill that combines multiple medicines into a single tablet, GMRx2 was developed out of a 20-year research program at The George Institute for Global Health. The single pill is a combination of three best-in-class medicines: telmisartan, amlodipine and indapamide.
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Neomorph, Abbvie to develop molecular glue degraders in $1.64B deal

Jan. 23, 2025
By Lee Landenberger
Neomorph Inc. has signed another billion-dollar deal to develop molecular glue degraders, this time with Abbvie Inc. It’s the third agreement in the past year the five-year-old company has signed with big pharma companies in a space with lots of collaborative deals.
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