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BioWorld - Tuesday, April 21, 2026
Home » Topics » Drugs » Small molecule

Small molecule
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Myst it again: Milestone gets a CRL for its heart spray

March 28, 2025
By Lee Landenberger
Milestone Pharmaceuticals Inc.’s heart rhythm-restoring calcium channel blocker for paroxysmal supraventricular tachycardia (PSVT) has yet another obstacle in its way. 
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Illustration of ovary, uterus and endometrial cancer.

Acrivon narrows focus for ACR-368 in endometrial cancer

March 27, 2025
By Jennifer Boggs
Acrivon Therapeutics Inc. provided updated phase II data for checkpoint kinase candidate ACR-368, highlighting promising response rates for biomarker-positive patients with endometrial cancer and raising the possibility of an accelerated pathway in the second-line setting.
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Acrivon narrows focus for ACR-368 in endometrial cancer

March 26, 2025
By Jennifer Boggs
Acrivon Therapeutics Inc. provided updated phase II data for checkpoint kinase candidate ACR-368, highlighting promising response rates for biomarker-positive patients with endometrial cancer and raising the possibility of an accelerated pathway in the second-line setting.
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Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia
Cancer

Pleco holds pre-IND meeting with FDA on PTX-252 for AML

March 26, 2025
Pleco Therapeutics BV has held a successful pre-IND meeting with the FDA to discuss the continued development of PTX-252, an intravenous formulation designed to treat patients with acute myeloid leukemia (AML).
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3D dollar sign
Neurology/psychiatric

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 26, 2025
By Nuala Moran
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
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Heart scientific overlay

For nearly $2B, Jiangsu Hengrui licenses heart drug to Merck

March 25, 2025
By Lee Landenberger
In a deal that could reach nearly $2 billion, Jiangsu Hengrui Pharmaceuticals Co. Ltd. has licensed a small molecule for treating cardiovascular disease to Merck & Co. Inc., dropping the China-based company into a space with lots of competitors in varying stages of development.
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3D dollar sign

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 24, 2025
By Nuala Moran
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
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Unity eyes path forward for UBX-1325 despite missed DME endpoint

March 24, 2025
By Jennifer Boggs
Shares of Unity Biotechnology Inc. (NASDAQ:UBX) sank 28.8% to close at $1.30 March 24 as a top-line readout of its phase IIb Aspire study testing UBX-1325 head-to-head against aflibercept in diabetic macular edema (DME) fell short of statistical noninferiority on the primary analysis endpoint. But that’s not the full story, according to company executives, who have plans for moving the senolytic Bcl-xL inhibitor into late-stage studies.
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Black Diamond lands $780M Servier deal for RAS/RAF candidate

March 19, 2025
By Jennifer Boggs
Black Diamond Therapeutics Inc. is getting $70 million up front and could earn up to $710 million in milestone payments in a deal with Servier for phase I-stage BDTX-4933, a small-molecule candidate targeting both RAS mutations and RAF alternations for treating solid tumors, including non-small-cell lung cancer.
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Torqur’s actinic keratosis data catches pharma eye, says CEO

March 14, 2025
By Nuala Moran
Term sheets are being drawn up with potential pharma partners after Torqur AG reported positive interim results from an ongoing phase II trial of bimiralisib topical gel in the treatment of actinic keratosis.
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