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BioWorld - Friday, February 27, 2026
Home » Topics » Small molecule, BioWorld

Small molecule, BioWorld
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Artistic rendering of the KRAS protein.

AACR 2025: Revolution has early stage KRAS success in NSCLC

April 28, 2025
By Lee Landenberger
Early phase I data for Revolution Medicines Inc.’s RAS(ON) G12D-selective inhibitor zoldonrasib encouraged both the company and the analysts. Initial study data produced a 61% objective response rate in 11 solid tumor patients receiving the 1,200-mg dose once a day. The disease control rate for 16 patients came in at 89%.
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China adds Ascentage's lisaftoclax, olverembatinib to guidelines

April 23, 2025
By Tamra Sami
Ascentage Pharma Group Corp. Ltd. said two of its novel candidates have been included in the 2025 Chinese Society of Clinical Oncology Guidelines, including its in-house developed Bcl-2 selective inhibitor, lisaftoclax (APG-2575), which is under review in China and could be the first domestically approved Bcl-2 inhibitor and the second global approval.
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Brain cancer illustration

Telix’s brain cancer radiotherapy shows phase II survival benefit

April 17, 2025
By Tamra Sami
Telix Pharmaceuticals Ltd.’s glioblastoma theranostic, TLX-101, is showing promising overall survival in a phase II trial in recurrent high-grade glioma. The targeted radiation therapy, combined with external beam radiation therapy, demonstrated a median overall survival of 12.4 months from treatment initiation and 32.2 months from initial diagnosis. The results significantly outperform the 9.9-month median survival typically observed with radiation therapy alone in recurrent glioblastoma patients.
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Person holding pills and glass of water

Post-Pfizer dropout, Lilly Achieves GLP-1 win in phase III

April 17, 2025
By Randy Osborne
Days after Pfizer Inc. pulled the plug on its oral GLP-1 candidate danuglipron, Eli Lilly and Co. aired positive top-line data from the phase III trial called Achieve-1 testing orforglipron vs. placebo in adults with type 2 diabetes and inadequate glycemic control with diet and exercise alone.
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Glycomine snags $115M to advance its PMM2-CDG treatment

April 17, 2025
By Brian Orelli
Glycomine Inc. secured $115 million in a series C financing round to support the advancement of GLM-101 into a phase IIb study in patients with phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG).
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Pills, bottle on $100 bills

New Trump order zeroes in on drug prices

April 16, 2025
By Mari Serebrov
In a blast from the past, U.S. President Donald Trump signed an executive order April 15 to deliver on his 2016 campaign promises and strengthen or reinstate efforts of his first administration to drive down prescription drug prices. “My first term included numerous significant actions, including some of the most aggressive in recent history, to deliver lower prescription drug prices to American patients,” Trump noted in the order, which builds on many of those actions, including increased competition, re-importation, price transparency and a mandate to pass discounts through to patients.
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Pfizer’s loss offers boost to competitors in obesity space

April 14, 2025
By Jennifer Boggs
Pfizer Inc. is ending work on oral GLP-1 candidate danuglipron for weight loss following the report of a single potentially drug-induced liver injury, a move that appears to open the door for other firms working on oral therapies in the high-dollar obesity space, even as industry watchers seek further details to determine whether similar safety signals could emerge for those competitors.
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Tryptamine to test injectable psilocybin for binge eating disorder

April 10, 2025
By Tamra Sami
Tryptamine Therapeutics Ltd. is gearing up to enter the clinic with lead compound TRP-8803, an intravenous-infused psilocybin therapy, in patients with binge eating disorder in conjunction with psychotherapy.
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Elderly woman and jigsaw puzzle

Alzheon’s lead candidate misses a phase III in early Alzheimer’s

April 10, 2025
By Lee Landenberger
Alzheon Inc.’s oral treatment for people in the early stages of Alzheimer’s disease missed its phase III primary endpoint, adding yet another therapy to a long list by many developers that can’t beat dementia. The study also received grant money, which is in increasingly short supply.
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Neurosense phase II combo to hail new Paradigm in ALS?

April 9, 2025
By Randy Osborne
As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.
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