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BioWorld - Monday, April 13, 2026
Home » Topics » Drugs » Small molecule

Small molecule
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FDA Approved stamp

Blockbuster bound? Neurocrine’s hyperplasia drug approved

Dec. 16, 2024
By Lee Landenberger
While Spruce Biosciences Inc.’s tildacerfont missed its phase II primary endpoint in classic congenital adrenal hyperplasia (CAH) last March, the U.S. FDA approved Crenessity (crinecerfont) from Neurocrine Biosciences Inc. for treating pediatric and adult CAH patients. The nod could lead the drug to become a blockbuster, analysts said.
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Brain illustrated with pills

Newron stock soars on €117M EA Pharma deal for schizophrenia drug

Dec. 16, 2024
By Marian (YoonJee) Chu
Newron Pharmaceuticals SpA scored €44 million (US$46.26 million) up front in a potential €117 million licensing deal with EA Pharma Co. Ltd. to pad the clinical runway of its late-stage oral schizophrenia asset, evenamide (NW-3509), sending company stock prices up near 20%.
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Doctor with brain illustration, businessman with dollar sign illustration

Beigene, CSPC strike $1.5B deal for phase I MAT2A inhibitor

Dec. 13, 2024
By Tamra Sami
Beigene Ltd. struck a global licensing deal with CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co. Ltd. worth up to $1.5 billion for its phase I selective methionine adenosyltransferase 2A (MAT2A) inhibitor, SYH-2039, which is being explored for solid tumors.
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New cancer data from Bicycle precedes stock drop

Dec. 13, 2024
By Lee Landenberger
Mixed results for three of Bicycle Therapeutics plc’s zelenectide pevedotin development programs preceded a strong drop in the company’s stock. Bicycle shares (NASDAQ:BCYC) dropped 31.3% on Dec. 13 to close at $13.81 each, the stock’s lowest price in the past 12 months.
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Ovarian cancer illustration

Drug duo to Repare lack in gyno cancers? Phase I shows promise

Dec. 13, 2024
By Randy Osborne
Wall Street wasn’t jumping up and down about Repare Therapeutics Inc.’s early data in gynecological cancers, though the findings look positive and the company has big plans for the combo therapy.
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Man measuring waist

Caliway’s CBL-514 meets endpoints in reducing abdominal fat

Dec. 12, 2024
By Tamra Sami
Caliway Biopharmaceuticals’ lipolysis candidate, CBL-514, met all the primary and secondary efficacy endpoints in a phase IIb study for subcutaneous fat reduction, paving the way for the company to begin a global pivotal phase III study in 2025.
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Lungs

Trial’d on for size, Trevi’s Haduvio fits nice in IPF cough

Dec. 12, 2024
By Randy Osborne
News that Trevi Therapeutics Inc. got the size just right of the phase IIb Coral trial with Haduvio (oral nalbuphine extended release) led the company’s shares (NASDAQ:TRVI) to close Dec. 12 at $3.60, up $1.11, or 45%, after trading as high as $4.60.
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Acute myeloid leukemia

ASH 2024: Kura, Kyowa highlight positive combo data for ziftomenib

Dec. 11, 2024
By Tamra Sami
Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged  acute myeloid leukemia.
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Noema adds new investor as it nears phase II readouts

Dec. 11, 2024
By Nuala Moran
CNS specialist Noema Pharma AG has added a further $44 million to its series B, bringing the total for the round to $147 million. The new financing bolsters the balance sheet as the Basel, Switzerland-based company progresses four phase II trials, with key data readouts expected in 2025.
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Ear illustration

Loud and clear: Sound Pharma has phase III success in Meniere’s

Dec. 11, 2024
By Lee Landenberger
Sound Pharmaceuticals Inc.’s lead candidate, the anti-inflammatory SPI-1005 (ebselen), has produced positive pivotal phase III study data in treating Meniere’s disease, an inner-ear disorder that can lead to vertigo and deafness.
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