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BioWorld - Friday, April 10, 2026
Home » Topics » Clinical

Clinical
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Brain and DNA

Neuren’s NNZ-2591 meets endpoints in phase II trial in Phelan-McDermid syndrome

Dec. 19, 2023
By Tamra Sami
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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Brain and DNA

Neuren’s NNZ-2591 meets endpoints in phase II trial in Phelan-McDermid syndrome

Dec. 18, 2023
By Tamra Sami
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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Invivyd stock spikes as MAb shows strength against COVID

Dec. 18, 2023
By Lee Landenberger
Invivyd Inc.’s VYD-222 produced positive initial top-line results in the ongoing pivotal phase III Canopy study for preventing symptomatic COVID-19. The results could reach a vulnerable population of patients who are immunocompromised and don’t get the same protection as other patients, the company’s CEO, Dave Hering, told BioWorld.
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Stock chart with falling red arrow

Phase IIa data re-Structure hopes in oral GLP-1 effort

Dec. 18, 2023
By Randy Osborne
Given what CEO Raymond Stevens called a space that’s “evolving extremely rapidly,” Structure Therapeutics Inc. chose – rather than wait for next year’s 12-week data – to unblind the eight-week obesity findings with GSBR-1290, an oral glucagon-like peptide-1 (GLP-1) agonist for which the firm also provided a phase IIa update in patients with type 2 diabetes.
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Marrow margin: Gilead CD47 prospect to boneyard, Shattuck’s wins in phase I

Dec. 13, 2023
By Randy Osborne
Wall Street will be watching closely for such adverse effects as anemia that foiled Gilead Sciences Inc.’s CD47-binding magrolimab earlier this year, but so far Shattuck Labs Inc.’s SL-172154 looks strong in combination with azacitidine to treat front-line higher-risk myelodysplastic syndrome (HR-MDS) and TP53-mutant (TP53m) acute myeloid leukemia (AML).
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3D illustration of a nerve cell

Feeling no pain: Vertex’s non-opioid posts positive phase II data

Dec. 13, 2023
By Lee Landenberger
All treatment groups in Vertex Pharmaceuticals Inc.’s phase II study of the non-opioid VX-548 showed meaningful reductions in pain from baseline in treating diabetic peripheral neuropathy (DPN), pointing the company toward pivotal phase III studies and prompting analysts to applaud enthusiastically.
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ASH 2023: Multiple new dosing regimens for multiple myeloma

Dec. 12, 2023
By Brian Orelli
Multiple studies at the 65th American Society of Hematology Annual Meeting 2023 have the potential to change the treatment paradigm for first-line treatment of multiple myeloma.
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ASH 2023: Syndax makes splash in cGVHD and leukemia

Dec. 12, 2023
By Brian Orelli
It’s not every day you see a small drug company’s presentations get picked for both the plenary session and the late-breaker session at a conference, but Syndax Pharmaceuticals Inc. managed to do just that at the 65th American Society of Hematology Annual Meeting 2023 – with a little help from a friend.
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ASH 2023: Gene therapy success is ‘historic’ but small molecules still mean more drugs in more places

Dec. 12, 2023
By Anette Breindl
Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by the U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. The addition of gene therapy to the therapeutic arsenal for SCD is “phenomenal,” Adetola Kassim, director of the Adult Sickle Cell Disease Program and professor of medicine at the Vanderbilt-Ingram Cancer Center, told BioWorld. Nevertheless, at a Saturday, Dec. 9, session titled, “Improving Outcomes for Individuals with Sickle Cell Disease: Are We Moving the Needle?,” which Kassim chaired, the answer remained “maybe.”
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Non-Hodgkin lymphoma cells in the blood flow

Prescient to advance PTX-100 to phase II for refractory T-cell lymphomas

Dec. 12, 2023
By Tamra Sami
Prescient Therapeutics Pty Ltd.’s PTX-100 met primary safety endpoints and showed preliminary efficacy in a phase Ib trial in patients with relapsed and refractory T-cell lymphomas that exceeded the standard of care, and the company hopes to advance to a phase II registrational study in 2024, Prescient CEO Steven Yatomi-Clarke told BioWorld.
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