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BioWorld - Sunday, December 14, 2025
Home » Topics » Clinical

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Brain tumor illustration

Servier vorasidenib PDUFA near, Agios wins in thalassemia

June 3, 2024
By Randy Osborne
While Servier Pharmaceuticals LLC awaits this summer’s PDUFA date for the IDH-mutant glioma drug vorasidenib, acquired in the buyout of oncology assets from Agios Pharmaceuticals Inc., the latter unveiled positive data from a global phase III study with oral mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.
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ADCs lead the data charge at ASCO while AI shapes the landscape

June 3, 2024
By Lee Landenberger
While there was much talk about the vast potential of artificial intelligence (AI) during one of the world’s largest research cancer conferences, antibody-drug conjugates (ADCs) continued to grab the lion’s share of attention as Corbus Pharmaceuticals Holdings Inc. and Takeda Pharmaceutical Co. Ltd. released new and positive data.
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ASCO: KRAS and lung cancer data from Lilly, Astrazeneca and Catalym

June 3, 2024
By Lee Landenberger
Eli Lilly and Co.’s updated phase I/II study of olomorasib as a monotherapy in patients with KRAS G12C-mutant advanced solid tumors yielded more promise in data released over the weekend at the American Society of Clinical Oncology (ASCO) annual meeting.
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Lung cancer illustration

Akeso’s ivonescimab bests Keytruda in PD-L1-positive NSCLC trial

May 31, 2024
By Tamra Sami
Although details are scant, Akeso Pharmaceuticals Inc. reported that its PD-1/VEGF bispecific antibody, ivonescimab (AK-112), demonstrated statistically significant superiority as a first-line therapy for PD-L1-positive non-small-cell lung cancer (NSCLC) compared to Merck and Co. Inc.’s Keytruda (pembrolizumab).
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Ultragenyx gene therapy hits phase III goal in rare disease GSD1a

May 31, 2024
By Jennifer Boggs
Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage disease type 1a (GSD1a) after the phase III study hit its primary endpoint and two key secondary endpoints.
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Merus raising $400M; petosemtamab scores in head and neck cancer

May 30, 2024
By Karen Carey
After shares climbed 36% on May 24 following the release of an American Society of Clinical Oncology abstract detailing an impressive phase II overall response rate in first-line head and neck cancer with bispecific antibody petosemtamab in combination with pembrolizumab, Merus NV is raising $400.2 million in an upsized follow-on offering.
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ASCO 2023 poster session

ASCO by the numbers: Massive cancer conference begins Friday

May 30, 2024
By Lee Landenberger
The American Society of Clinical Oncology (ASCO) begins its 2024 annual meeting at the cavernous and labyrinthine McCormick Place convention center in Chicago Friday, May 31. It’s one of the world’s largest cancer research conferences and can be daunting to follow.  More than 400 organizations will participate this year, with about 200 sessions ready to convene. The vast majority of the 5,000 abstracts that cover all aspects of cancer treatment have already been released, and they will be scrutinized by the more than 40,000 attendees from around the world.
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Biohaven’s phase I protein degrader results stun the stock

May 29, 2024
By Lee Landenberger
Interim data from Biohaven Ltd.’s phase I study of BHV-1300, an IgG degrader using an ASGRP-bispecific, produced dose-dependent results in its first four cohorts, though not enough to satisfy investors.
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Brain clay model

Neuren’s NNZ-2591 meets phase II endpoints in Pitt Hopkins syndrome

May 29, 2024
By Tamra Sami
Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Pitt Hopkins syndrome, with improvements seen in communication, social interaction, cognition and motor abilities, according to top-line results.
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Lungs

Insmed stock soars on phase III lung disease data

May 28, 2024
By Lee Landenberger
Insmed Inc.’s phase III study of brensocatib in treating noncystic fibrosis bronchiectasis hit its primary and multiple secondary endpoints, impressing investors with statistically significant results. The positive data could lead to the first drug approval for the treating the deadly lung disease.
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