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Phase 3 study shows strong efficacy
Original title: “Definium reports positive results for anxiety treatment”
Rigel makes Veppanu available across US
Original title: “Veppanu launch brings new option for advanced breast cancer”
The discontinuation of the uveitis study adds to Regeneron’s series of clinical setbacks, including the Phase 3 failures of the Dupixent follow-up itepekimab and an investigational combo regimen for melanoma.
Original title: “Regeneron ends eye disease study due to antibody's safety profile”
The discontinuation of the uveitis study adds to Regeneron’s series of clinical setbacks, including the Phase 3 failures of the Dupixent follow-up itepekimab and an investigational combo regimen for melanoma.
Original title: “Regeneron ends eye disease study after antibody shows ‘unfavorable’ risk/benefit profile”
Though its drug missed the main goal of a wet AMD study, EyePoint, as well as analysts, still see a path toward approval. Investors may be harder to win over, however.
Original title: “EyePoint plummets on pivotal data for main drug”
EyePoint’s quest to reduce treatment burden for patients with age-related macular degeneration has hit a bump in the road, with a clinical miss driving the biotech’s stock down by 70%.
Original title: “EyePoint’s Phase 3 fail sends stock spiraling amid next-gen AMD race”
Ivonescimab notched its third approval in China last week, but the label update has tempered expectations for the Summit Therapeutics/Akeso drug, showing that progression-free survival declines over time.
Original title: “Summit, Akeso’s China approval celebration cut short as survival benefit degrades”
AstraZeneca will push through with its other Phase 3 studies for volrustomig, including in cervical cancer and mesothelioma.
Original title: “AstraZeneca pulls plug on late-stage lung cancer trial, extending losing streak”
AstraZeneca will push through with its other Phase 3 studies for volrustomig, including in cervical cancer and mesothelioma.
Original title: “AstraZeneca pulls plug on late-stage lung cancer trial, taking another clinical blow”
Otsuka Pharmaceuticals’ Voyxact is not the only IgA nephropathy drug that can slow the decline of kidney function. Vera Therapeutics’ Trutakna, which the FDA granted accelerated approval last month, showed “impressive” stabilization, analysts say.
Original title: “Vera’s newly approved IgA nephropathy drug supports kidney function, like Otsuka’s Voyxact”
Study begins testing KJ‑103 in TROP2‑positive cancers
Original title: “First patient treated in new heavy‑chain‑only antibody trial”
In this Q&A, Minsu Kang, vice president ... experience engaging with the FDA in the context of rare disease therapies. Kang has over 10 years in CNS development, and extensive experience working on late-stage clinical trials and regulatory affairs for a number of neurodegenerative ... In this Q&A, Minsu Kang, vice president of clinical development and regulatory affairs at Polaryx Therapeutics, shares his insights from his firsthand experience engaging with the FDA in the context of rare disease therapies. Kang has over 10 years in CNS development, and extensive experience working on late-stage clinical trials and regulatory affairs for a number of neurodegenerative diseases. Polaryx, a publicly-traded, clinical-stage biotech is developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (LSD). Recent funding will allow the initiation of its Phase II SOTERIA trial, evaluating its lead compound across multiple LSD indications. Minsu Kang of Polaryx Therapeutics explains how to align with regulators on study design and maintain scientific rigor Many factors go into achieving alignment with the FDA on this topic, such as off-label uses of existing medications, disease severity, and availability of natural history data that may serve as an external control arm. Having a good scientific foundation and supporting data, beyond simply referring to the unmet medical needs, can be helpful in achieving regulatory alignment. BX: In your experience, has this alignment caused any friction with the clinical development plans or maybe trial amendments?
Original title: “Navigating FDA talks in rare disease programs”
In this Q&A, Minsu Kang, vice president ... experience engaging with the FDA in the context of rare disease therapies. Kang has over 10 years in CNS development, and extensive experience working on late-stage clinical trials and regulatory affairs for a number of neurodegenerative ... In this Q&A, Minsu Kang, vice president of clinical development and regulatory affairs at Polaryx Therapeutics, shares his insights from his firsthand experience engaging with the FDA in the context of rare disease therapies. Kang has over 10 years in CNS development, and extensive experience working on late-stage clinical trials and regulatory affairs for a number of neurodegenerative diseases. Polaryx, a publicly-traded, clinical-stage biotech is developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (LSD). Recent funding will allow the initiation of its Phase II SOTERIA trial, evaluating its lead compound across multiple LSD indications. Minsu Kang of Polaryx Therapeutics explains how to align with regulators on study design and maintain scientific rigor Many factors go into achieving alignment with the FDA on this topic, such as off-label uses of existing medications, disease severity, and availability of natural history data that may serve as an external control arm. Having a good scientific foundation and supporting data, beyond simply referring to the unmet medical needs, can be helpful in achieving regulatory alignment. BX: In your experience, has this alignment caused any friction with the clinical development plans or maybe trial amendments?
Original title: “Navigating FDA talks in rare disease programs”
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
Original title: “Capricor soars on potential FDA review extension, filing amendments”