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BioWorld - Monday, March 23, 2026
Home » Topics » Drugs » Small molecule

Small molecule
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Verastem seesaws on ASCO avutometinib/defactinib pancreatic data

May 24, 2024
By Randy Osborne
As the company began a rolling NDA submission to the U.S. FDA for its drug combo in low-grade serous ovarian cancer, Verastem Oncology Inc. popped the lid off phase I/II data in pancreatic tumors, but Wall Street seemed uncertain about the news. Boston-based Verastem disclosed upbeat outcomes from the Ramp 205 study testing the RAF/MEK clamp avutometinib when paired with focal adhesion kinase inhibitor defactinib in combination with gemcitabine plus nab-paclitaxel as first-line therapy for metastatic pancreatic cancer.
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AI-generated image of illustration of MRI of lungs with fibrosis
Respiratory

GTX-011 ameliorates symptoms in murine models of lung fibrosis

May 24, 2024
Interstitial lung fibrotic disease (ILD) is characterized by inflammation and fibrosis of lung tissue and is associated with poor prognosis. Gat Therapeutics SL has developed GTX-011, an orally available small molecule for treating fibrotic diseases.
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Molecule illustration

Degron, Takeda bind $1.2B deal for molecular glue degraders

May 23, 2024
By Marian (YoonJee) Chu
Shanghai- and San Diego-based Degron Therapeutics Inc. secured a potential $1.2 billion deal with Tokyo-headquartered Takeda Pharmaceutical Co. Ltd. May 23 for a multitarget collaboration and exclusive licensing agreement for molecular glue degraders. “It is a breakthrough technology in the small-molecule drug discovery field,” Degron CEO Lily Zou told BioWorld. “People talk about cell and gene therapy, but small molecules are still the mainstream of drug discovery, [with] more reach.”
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Molecule illustration

Degron, Takeda bind $1.2B deal for molecular glue degraders

May 23, 2024
By Marian (YoonJee) Chu
Shanghai- and San Diego-based Degron Therapeutics Inc. secured a potential $1.2 billion deal with Tokyo-headquartered Takeda Pharmaceutical Co. Ltd. May 23 for a multitarget collaboration and exclusive licensing agreement for molecular glue degraders. “It is a breakthrough technology in the small-molecule drug discovery field,” Degron CEO Lily Zou told BioWorld. “People talk about cell and gene therapy, but small molecules are still the mainstream of drug discovery, [with] more reach.”
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Promising data help Grey Wolf land $50M series B expansion

May 23, 2024
By Nuala Moran
Grey Wolf Therapeutics Ltd. added $50 million to its series B, bringing the total for the round to $99 million and providing funding to expand the scope of an ongoing phase I/II trial of its lead antigen modulation program.
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Contera’s JM-101 missed phase IIb endpoint in Parkinson’s

May 22, 2024
By Marian (YoonJee) Chu
Hørsholm, Denmark-based Contera Pharma A/S’ lead asset for levodopa-induced dyskinesia in Parkinson’s disease, JM-010, failed to meet its primary endpoint in the late-stage phase IIb Astoria trial. JM-010 is a novel drug formulation and 5-HT receptor modulator that combines existing medications of immediate-release buspirone and extended-release zolmitriptan.
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Senior eye exam

Rezolute’s oral diabetic macular edema drug hits phase II goal

May 22, 2024
By Marian (YoonJee) Chu
Shares of Redwood City, Calif.-based Rezolute Inc., and Korean parent company Handok Inc., rose May 22 on positive results of a phase II proof-of-concept study for its investigative oral diabetic macular edema (DME) drug, RZ-402. RZ-402 is an oral small-molecule plasma kallikrein inhibitor designed to block vascular leakage and inflammation for treating chronic DME.
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Théa returns rights to Curacle’s oral diabetic macular edema drug

May 21, 2024
By Marian (YoonJee) Chu
Théa Open Innovation, a subsidiary of France’s Laboratoires Théa SAS, returned rights to South Korea’s Curacle Co. Ltd.’s CU-06, an oral diabetic macular edema drug candidate. Curacle posted positive top-line phase IIa data of CU-06 just three months prior.
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Concept of business partnership

Erasca buys-in assets from two China biotechs, slashes workforce

May 21, 2024
By Marian (YoonJee) Chu
Shaking up corporate and pipeline structure, San Diego-based cancer developer Erasca Inc. in-licensed two assets from China-based biopharmas in all-cash deals, while laying off 18% of its workforce, primarily in drug discovery. The flurry of announcements made on May 16, which included $160 million raised in private placement, showed that Erasca would scrap three existing pipeline assets – ERAS-007, ERAS-801 and ERAS-4 – and reshape development to a RAS-targeting franchise.
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Hitting reverse: Endeavor meets phase IIa goals in IPF

May 20, 2024
By Lee Landenberger
With positive phase IIa data for ENV-101 in idiopathic pulmonary fibrosis (IPF) in hand, showing some lung damage was reversed, Endeavor Biomedicines Inc. said it’s ready to begin further phase II studies in the indication.
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