October 24, 2023

WATCH: Edgewise Therapeutics: A Novel Approach Designed to Protect Muscle in Becker (Webinar Recording)

Edgewise Therapeutics joined PPMD for a community webinar on October 18, 2023 to discuss the company’s investigational compound, EDG-5506. Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer at Edgewise, and Alan Russell, Ph.D., Edgewise’s Co-Founder and…

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October 14, 2023

WATCH: Affinity Duchenne™ and Affinity Beyond™: Updates on Investigational RGX-202 from REGENXBIO (Webinar Recording)

REGENXBIO joined PPMD for a community webinar on October 11, 2023 to discuss the company’s investigational gene therapy for Duchenne muscular dystrophy, AFFINITY DUCHENNE™, and AFFINITY BEYOND™, an AAV8 antibody assessment study. Jahannaz (Naz) Dastgir,…

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October 13, 2023

FibroGen announces discontinuation of LELANTOS-2 Open Label Extension

PPMD has recently learned that FibroGen has discontinued the LELANTOS-2 Open Label Extension. This news follows the August 2023 release of negative topline results of the Phase 3 LELANTOS-2 trial of pamrevlumab, FibroGen’s investigational anti-fibrotic…

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October 13, 2023

Santhera Receives Positive European Medicines Agency (EMA) CHMP Opinion Recommending Approval of Vamorolone in European Union

Santhera Pharmaceuticals has announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion in favor of approval of vamorolone (AGAMREE®) for the treatment of Duchenne muscular dystrophy…

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October 4, 2023

REGENXBIO announces interim clinical data from Phase I/II AFFINITY DUCHENNE™ trial of RGX-202

REGENXBIO Inc. has announced additional interim safety data and initial efficacy data from the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne. RGX-202 utilizes a…

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September 26, 2023

Edgewise Therapeutics Now Enrolling GRAND CANYON Study of EDG-5506 in Individuals with Becker Muscular Dystrophy

Edgewise Therapeutics has announced the start of enrollment of GRAND CANYON, the company’s global pivotal study of EDG-5506 in individuals with Becker. GRAND CANYON is an expansion of the CANYON study, which was over-enrolled. EDG-5506…

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September 21, 2023

Entrada Therapeutics starts dosing in ENTR-601-44 Phase 1 trial

Entrada Therapeutics has announced that the first participant has been dosed in its Phase 1 clinical trial evaluating ENTR-601-44, the company’s proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), for the potential treatment of…

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September 15, 2023

PTC Therapeutics Announces Negative European Medicines Agency Opinion Regarding Full Authorization of Translarna

PTC Therapeutics today shared that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not renewing the Marketing Authorization for Translarna™ (ataluren), a treatment for Duchenne patients…

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August 30, 2023

FibroGen Announces Topline Data from Phase 3 LELANTOS-2 Study

FibroGen has announced topline results of the Phase 3 LELANTOS-2 trial of pamrevlumab, their investigational anti-fibrotic monoclonal antibody, for ambulatory patients with Duchenne muscular dystrophy.  Although generally safe and well tolerated, pamrevlumab did not meet…

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June 29, 2023

Italfarmaco Group Announces NDA Accepted for Givinostat

PPMD is excited to learn that the FDA completed its filing review and accepted Italfarmaco Group’s New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the treatment of individuals with…

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