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BioWorld - Tuesday, June 23, 2026
Home » Topics » Drugs » Small molecule

Small molecule
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Parkinson's disease illustration showing neurons containing alpha-synuclein

Cerevel undersell? Bombshell Parkinson’s phase III with tavapadon

April 18, 2024
By Randy Osborne
Cerevel Therapeutics Inc.’s positive results from the long-shot pivotal phase III Tempo-3 trial with tavapadon – the first D1/D5 receptor partial agonist being studied as a once-daily treatment for Parkinson’s disease – added gravy to the $8.7 billion takeover by Abbvie Inc., disclosed late last year. The buyout’s centerpiece was the late-stage asset emraclidine, a positive allosteric modulator of the muscarinic M4 receptor, touted as a potential best-in-class, next-generation antipsychotic for schizophrenia, which strikes more than 5 million people in the G7 (U.S., France, Germany, Italy, Spain, U.K. and Japan).
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Neurocrine advances Nxera’s NBI-1117568 in schizophrenia

April 16, 2024
By Tamra Sami
Nxera Pharma Co. Ltd. announced that its partner, Neurocrine Biosciences Inc., is advancing NBI-1117568 to phase II trials for treatment of schizophrenia and other neuropsychiatric disorders.
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Closeup of wheelchair

Edgewise phase Ib data promising in Becker muscular dystrophy

April 16, 2024
By Lee Landenberger
With no drugs approved by the U.S. FDA for treating Becker muscular dystrophy, Edgewise Therapeutics Inc. reported positive two-year, phase Ib data looking at patients’ ability to physically function, plus biomarker data.
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Pill over molecule structures

Preclinical convulsions in rabbits halt Neumora’s phase I

April 15, 2024
By Lee Landenberger
Preclinical data of rabbits having convulsions has prompted the U.S. FDA to place a clinical hold on Neumora Therapeutics Inc.’s phase I study of NMRA-266 in healthy adults. Neumora said about 30 participants had been dosed so far in the single ascending and multiple ascending dose study, with no evidence of convulsions seen.
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Amylyx’s AMX-0035 promising in ultrarare Wolfram syndrome

April 10, 2024
By Jennifer Boggs
With the recent removal of its amyotrophic lateral sclerosis drug from the market, Amylyx Pharmaceuticals Inc. is looking to revive investor interest with interim data from its phase II Helios study testing the same drug, AMX-0035 (sodium phenylbutyrate plus taurursodiol), in Wolfram syndrome, a rare indication in which Amylyx could be leading the charge.
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Business, data, dollars illustration

VC firm Medicxi invests $40M in China’s D3 Bio

April 9, 2024
By Nuala Moran
Medicxi has made its first ever investment in China – and its biggest single commitment to date – putting $40 million into D3 Bio Inc. The investment by the London-based venture capital firm will accelerate development of D3’s lead program D3S-001, a second generation KRAS G12C inhibitor, which is in phase II development in advanced solid tumors.
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Ascletis quits development of farnesoid X receptor agonist ASC-42

April 9, 2024
By Tamra Sami
After reviewing data from its phase II trial of farnesoid X receptor agonist ASC-42 in primary biliary cholangitis, Ascletis Pharma Inc. said it was quitting development of the molecule in all indications.
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Business, data, dollars illustration

VC firm Medicxi invests $40M in China’s D3 Bio

April 8, 2024
By Nuala Moran
Medicxi has made its first ever investment in China – and its biggest single commitment to date – putting $40 million into D3 Bio Inc. The investment by the London-based venture capital firm will accelerate development of D3’s lead program D3S-001, a second generation KRAS G12C inhibitor, which is in phase II development in advanced solid tumors.
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IPO stock market ticker

Contineum prices $110M IPO to advance LPA1R antagonists

April 5, 2024
By Caroline Richards
With high hopes for its LPA1R antagonist program, Contineum Therapeutics Inc. has priced an IPO of 6.9 million shares of its class A common stock at $16 per share as it seeks to generate $110 million in gross proceeds. The San Diego-based company began trading on Nasdaq under the ticker CTNM on April 5, with shares ending the day at $15.40, down 3.8%. There have been nine other biopharma IPOs so far in 2024.
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Ascletis quits development of farnesoid X receptor agonist ASC-42

April 5, 2024
By Tamra Sami
After reviewing data from its phase II trial of farnesoid X receptor agonist ASC-42 in primary biliary cholangitis, Ascletis Pharma Inc. said it was quitting development of the molecule in all indications.
Read More
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