Hypolipoproteinemias

LONDON and CAMBRIDGE, Mass., April 24, 2025 /PRNewswire/ -- Beacon Therapeutics Holdings Limited ('Beacon Therapeutics' or 'the Company'), a leading clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases, today announces an upcoming presentation on 6-month interim safety and efficacy results from the Phase 2 DAWN trial of the Company's lead program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa (XLRP) at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting being held May 4-8, 2025 in Salt Lake City. Presentation details: Presentation t itle: Subretinal gene therapy laru-zova (AGTC-501) for X-linked retinitis pigmentosa (XLRP): Phase 2 DAWN preliminary month 6 results Session title: Clinical advances in cell and gene therapy Date: Tuesday, May 6, 2025 Presenter: Mark Pennesi, M.D., Ph.D., FARVO, Director, Ophthalmic Genetics at the Retina Foundation in Dallas, Texas; Professor of Ophthalmology and Professor of Molecular and Medical Genetics, and Chief of the Paul H. Casey Ophthalmic Genetics Division at the Casey Eye Institute, Oregon Health and Science University in Portland, Oregon Beacon Therapeutics is currently enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP. About laru-zova Laru-zova (laruparetigene zovaparvovec) is a potential best-in-class gene therapy currently being investigated for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Laru-zova has the potential to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene designed to produce the full-length protein. Laru-zova has Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA), Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Innovative Licensing and Access Pathway (ILAP) from the UK's Medicines and Healthcare products Regulatory Agency (MHRA), as well as Orphan Drug Designation (ODD) from the FDA and EMA. About the DAWN and VISTA Studies DAWN (NCT06275620) is a Phase 2, open-label study of laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The objective of DAWN is to assess two different dose levels of laru-zova for efficacy, safety and tolerability in the target population. DAWN is also evaluating the changes in visual function and functional vision, and is the first trial in the laru-zova clinical development program that is collecting and evaluating low luminance visual acuity (LLVA) data. VISTA (NCT04850118) is a Phase 2/3, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety and tolerability of laru-zova in two study groups compared to an untreated control group. The study will evaluate the proportion of participants with a 15 or more letter increase from baseline in LLVA and additional measures of functional vision. The VISTA study is currently enrolling. About XLRP X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease that predominantly affects males, typically caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The mutations, which affect approximately 1 in 25,000 males in the U.S., Europe and Australia, result in progressive photoreceptor loss over time and visual dysfunction beginning in childhood, eventually leading to blindness and impacting quality of life with no approved treatments. About Beacon Therapeutics Beacon Therapeutics is a clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases. The Company has an established scientific foundation that includes a late-stage clinical development candidate to treat XLRP and two preclinical programs targeting dry age-related macular degeneration (AMD) and an inherited cone-rod dystrophy (CRD). Beacon Therapeutics' investors include Syncona Limited, Forbion, Oxford Science Enterprises, TCGX and Advent Life Sciences, among others. Learn more about Beacon Therapeutics at beacontx.com. Contact: [email protected] Media: [email protected] SOURCE Beacon Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

LONDON, UK and CAMBRIDGE, MA, USA I December 6, 2024 I Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases, today announced the presentation of 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy. Key presentation highlights: DAWN is a non-randomized, open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting the RPGR protein. The purpose of DAWN is to assess two different doses of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP. XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects young males with an estimated prevalence of 1 in 25,000 males in the US, Europe and Australia having XLRP with RPGR mutations. Laru-zova expresses the full length RPGR protein and is therefore expected to address the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP. Lance Baldo, MD, chief executive officer of Beacon Therapeutics, stated, “We are encouraged by the early results from the Phase 2 DAWN study. The strong safety profile observed to date is complemented by promising early improvements in low luminance visual acuity – a meaningful and functional measure of vision in patients with XLRP. These data not only support the ongoing pivotal VISTA study, but also strengthen our commitment to this opportunity to bring hope to patients and families affected by this devastating disease.” Beacon Therapeutics is also enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP. Presentations – Presenting Author – Professor Paulo Eduardo Stanga, Founder and Chief Medical Officer, The Retina Clinic London About Beacon Therapeutics Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP) and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD). Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. Lead development candidate laru-zova (AGTC-501), is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss, primarily in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences and additional investors. Find out more about Beacon Therapeutics at beacontx.com . SOURCE: Beacon Therapeutics

To date, laru-zova has been well-tolerated by all participants in the Phase 2 DAWN study. Data show promising early improvements in low luminance visual acuity (LLVA), a critical measure of visual function. Data build confidence in laru-zova as a potential treatment for patients with XLRP. Pivotal Phase 2/3 VISTA trial for laru-zova in XLRP is currently enrolling. LONDON and CAMBRIDGE, Mass., Dec. 6, 2024 /PRNewswire/ -- Beacon Therapeutics Holdings Limited ('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases, today announced the presentation of 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy. Key presentation highlights: The three-month data show that laru-zova has been well-tolerated by all participants. No study agent-related treatment emergent adverse events (TEAEs) were reported, including no ocular inflammatory adverse events. Data also show promising early improvements in LLVA, a critical measure of visual function. The benefit-risk profile supports on-going clinical development for the treatment of patients with XLRP caused by RPGR mutations. DAWN is a non-randomized, open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting the RPGR protein. The purpose of DAWN is to assess two different doses of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP. XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects young males with an estimated prevalence of 1 in 25,000 males in the US, Europe and Australia having XLRP with RPGR mutations. Laru-zova expresses the full length RPGR protein and is therefore expected to address the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP. Lance Baldo, MD, chief executive officer of Beacon Therapeutics, stated, "We are encouraged by the early results from the Phase 2 DAWN study. The strong safety profile observed to date is complemented by promising early improvements in low luminance visual acuity - a meaningful and functional measure of vision in patients with XLRP. These data not only support the ongoing pivotal VISTA study, but also strengthen our commitment to this opportunity to bring hope to patients and families affected by this devastating disease." Beacon Therapeutics is also enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP. Presentations – Subretinal Gene Therapy laru-zova (AGTC-501) for X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study (DAWN): Preliminary Results RPGR gene therapy and the Beacon clinical trials: Beacon Therapeutics Subretinal Gene Therapy laru-zova (AGTC-501) for X-Linked Retinitis Pigmentosa (XLRP) Presenting Author – Professor Paulo Eduardo Stanga, Founder and Chief Medical Officer, The Retina Clinic London Contact: [email protected] Media: [email protected] About Beacon Therapeutics Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP) and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD). Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. Lead development candidate laru-zova (AGTC-501), is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss, primarily in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences and additional investors. Find out more about Beacon Therapeutics at beacontx.com. SOURCE Beacon Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED