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BioWorld - Thursday, April 23, 2026
Home » Topics » Clinical

Clinical
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Nex-z less sexy with second liver case; Intellia phase III paused

Oct. 27, 2025
By Randy Osborne
No Comments
Intellia Therapeutics Inc. followed up troubling news in May with a similar, and worse, update regarding the Magnitude and Magnitude-2 phase III trials with nexiguran ziclumeran, also known as nex-z, for patients with transthyretin amyloidosis with cardiomyopathy and polyneuropathy, respectively.
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‘Home run scenario’ for Bridgebio’s BBP-418 in limb-girdle phase III

Oct. 27, 2025
By Jennifer Boggs
No Comments
Impressive data from an interim readout of Bridgebio Pharma Inc.’s BBP-418 in limb-girdle muscular dystrophy type 2I/R9 has the company prepping to meet with the U.S. FDA to discuss plans for the upcoming NDA filing, including the possibility for seeking full approval for what could be the first therapy for the rare muscular disease.
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Health professional holding stethoscope with health icons
Biopharma clinical updates September 2025

Clinical trial activity jumps as BioWorld tracks 230 updates in September

Oct. 24, 2025
By Amanda Lanier
No Comments
In September 2025, BioWorld recorded 230 clinical trial updates spanning phases I through III, up sharply from 95 in August, 140 in July and just below 254 in June. Among these, 22 phase III trials reported positive outcomes, while four ended in failure and another three produced mixed results.
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Pancreas

Pancreatic Revolution due? Tango PRMT5 data satisfy

Oct. 24, 2025
By Randy Osborne
No Comments
Tango Therapeutics Inc. plans a pivotal study next year in second-line MTAP-deleted pancreatic ductal adenocarcinoma based on positive phase I/II data with next-generation MTA-cooperative PRMT5 inhibitor vopimetostat (TNG-462) in patients with tumors of that type.
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Ozekibart-(INBRX-109)

Inhibrx’s positive results in rare cancer prompts BLA talk

Oct. 24, 2025
By Lee Landenberger
No Comments
Inhibrx Biosciences Inc.’s monoclonal antibody for treating advanced or metastatic, unresectable chondrosarcoma hit its primary endpoint in a registrational phase II study, doubling the company’s stock on Oct. 24. Top-line data from the placebo-controlled study of ozekibart (INBRX-109) produced statistically significant and clinically meaningful improvement in median progression-free survival in a tough-to-treat patient population that has few remaining options.
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NLRP3 inflammasome

Risk intolerance: Ventyx CV bid shines in NLRP3 phase II

Oct. 23, 2025
By Randy Osborne
No Comments
Ventyx Biosciences Inc. rolled out positive results from the phase II study with oral, once-daily VTX-3232 in patients with obesity and cardiovascular (CV) risk factors.
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Acquisition target

Ipsen to acquire fellow French company Imcheck in €1B deal

Oct. 23, 2025
By Nuala Moran
No Comments
Ipsen SA is expanding its cancer portfolio with the €1 billion (US$1.16 billion) acquisition of immuno-oncology specialist Imcheck SAS, and will pay €350 million up front with the balance to come in regulatory and sales-based milestones for the lead program ICT-01.
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Moderna signage

Moderna’s cytomegalovirus vaccine fails a phase III

Oct. 23, 2025
By Lee Landenberger
No Comments
Moderna Inc. stopped its efforts to develop mRNA-1647 as a vaccine for preventing congenital cytomegalovirus infection, the most common infectious cause of birth defects in the U.S. The pivotal and placebo-controlled phase III CMVictory study of mRNA-1647 missed the primary efficacy endpoint, which was preventing the infection in seronegative females ages 16 to 40.
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PRIMA implant

Science Corp.'s Prima implant enables blind patients to read

Oct. 22, 2025
By Annette Boyle
Interim results from the PRIMAVERA study published in the New England Journal of Medicine demonstrated that Science Corp.'s Prima retinal implant restored vision, enabling 80% of patients to read at 12 months. Participants had age-related macular degeneration-related geographic atrophy resulting in total loss of central vision.
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Red arrow pointing downward on a stock market ticker

Alector stock and staff halved after phase III fail in dementia

Oct. 22, 2025
By Lee Landenberger
No Comments
The failure of Alector Inc.’s phase III study of latozinemab in treating dementia halved the company’s stock on Oct. 22. That is also about the same percentage of staff that Alector is letting go after the clinical trial stumble.
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