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BioWorld - Friday, July 31, 2026
Home » Topics » Clinical

Clinical
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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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Sleep apnea illustration

Lilly’s GLP-1/GIP tirzepatide clears sleep apnea phase III hurdle

April 17, 2024
By Jennifer Boggs
Eli Lilly and Co. is planning to file for U.S. FDA approval later this year after reporting that tirzepatide met all primary and key secondary endpoints in two phase III trials in obstructive sleep apnea (OSA). Assuming approval, tirzepatide could become the first drug approved specifically for OSA, while providing potential entry access for Medicare Part D coverage, which is denied for the GLP-1 class of drugs approved as obesity medications.
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Elderly hands holding broken brain structure

Phase II PD data from Sage: Precedent set for dalzanemdor?

April 17, 2024
By Randy Osborne
After Sage Therapeutics Inc. reported a phase II failure with oral dalzanemdor, also known as SAGE-718, in mild cognitive impairment related to Parkinson’s disease (PD), Wall Street’s eyes turned to ongoing mid-stage efforts with the same N-methyl-D-aspartate receptor-positive allosteric modulator in Huntington’s disease and Alzheimer’s disease.
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Child drawing with pencil

Neurotech’s NTI-164 meets endpoints in pediatric autism trial

April 17, 2024
By Tamra Sami
Neurotech International Ltd.’s cannabinoid NTI-164 met the primary and secondary endpoints in a phase II/III trial in children with autism spectrum disorder. The company also reported positive top-line data for Rett syndrome in a phase I/II trial.
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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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Hey Cullinan plan: CLN-978’s new focus watering shares

April 16, 2024
By Randy Osborne
With an IND filing in systemic lupus erythematosus expected during the third quarter of 2024, Cullinan Therapeutics Inc. changed its name from Cullinan Oncology Inc. and detailed its plans to target autoimmune diseases with CLN-978, a CD19xCD3 T-cell engager.
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Man sitting on bed holding head

Phase III win for Intra-Cellular's Caplyta for major depressive disorder

April 16, 2024
By Karen Carey
Robust top-line phase III data showed Intra-Cellular Therapies Inc.’s marketed oral atypical antipsychotic drug, Caplyta (lumateperone), achieved statistically significant and clinically meaningful best-in-class results, potentially expanding its reach into the billion-dollar major depressive disorder market. The company’s shares (NASDAQ:ITCI) surged to a 52-week high of $84.89 throughout the day, closing at $79.84, up 23.3%, or $15.08, on April 16.
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Ultragenyx’s Angelman prospect scores in phase I/II

April 15, 2024
By Randy Osborne
Wall Street may not have responded as positively as Ultragenyx Pharmaceutical Inc. would have liked after the firm unveiled new data from the phase I/II study with GTX-102 for the treatment of Angelman syndrome (AS). Patients in expansion cohorts A & B treated with a set dose and regimen of the intrathecally delivered antisense oligonucleotide (ASO) showed rapid and clinically meaningful improvement across multiple domains.
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Stock chart, red down arrow

No early stop for status epilepticus phase III; Marinus falls

April 15, 2024
By Jennifer Boggs
Investors anxious to see data from Marinus Pharmaceuticals Inc.’s phase III Raise study testing intravenous ganaxolone in refractory status epilepticus (RSE) will have to wait a little longer, as the trial failed to meet the predefined criteria for stopping at the interim analysis, sending shares (NASDAQ:MRNS) down nearly 83%.
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