Cone-Rod Dystrophies

LONDON, UK and CAMBRIDGE, MA, USA I October 15, 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 24-month interim safety and efficacy results of the Phase 2 SKYLINE trial in patients with XLRP at the American Academy of Ophthalmology’s Annual Meeting in Chicago, Illinois. The 24-month data showed a response rate of 57% (4/7) in study eyes treated with a high dose (6.8 E+11 vg/eye) of AGTC-501, defined as a patient who has an improvement in retinal sensitivity as assessed by microperimetry of at least 7 decibels (dB) in at least 5 loci. Patients in the high-dose cohort also showed a robust improvement in mean retinal sensitivity. Study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 showed a response rate of 25% (1/4). There were no clinically significant safety events associated with AGTC-501 treatment in this trial, and any treatment-emergent adverse events were mostly non-serious and mild to moderate in severity. XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects an estimated 3.4 – 4.4 per 100,000 young males with RPGR mutations in US, Europe and Australia. AGTC-501 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, making it uniquely well-suited to improve the lives of patients with XLRP as a potential treatment. Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics, stated, “Our Phase 2 SKYLINE 24-month data reinforces AGTC-501’s favorable safety profile and robust improvements in mean retinal sensitivity. We will continue to assess the long-term safety and durability of AGTC-501 but are encouraged by the results we’ve seen in the SKYLINE trial to date.” Beacon Therapeutics is also enrolling its pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials as the Company progresses toward the development of a treatment for XLRP. Poster – Subretinal AGTC-501 Gene Therapy for XLRP: 24-Month Interim Results of the Phase 2 SKYLINE Trial. Presenting Author – Robert Sisk, MD, FACS, FASRS, Associate Professor of Ophthalmology at the University of Cincinnati; Vitreoretinal Surgeon and partner at Cincinnati Eye Institute; Director of Pediatric Retinal Surgery and Director of Ophthalmic Genetics at Cincinnati Children’s Hospital. Contact: info@beacontx.com Media: beacontherapeutics@edelman.com 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), as well as 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 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. AGTC-501 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 Holdings

LONDON and CAMBRIDGE, Mass., Sept. 19, 2024 /PRNewswire/ -- Beacon Therapeutics Holdings Limited ('Beacon' 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 presented 36-month interim results from its Phase I/2 HORIZON trial of its lead asset, AGTC-501, in patients with X-linked retinitis pigmentosa (XLRP) at the 24th EURETINA Congress held in Barcelona, Spain. Key presentation highlights included: AGTC-501 was reported to be generally safe and well-tolerated in the 29 patients enrolled with no clinically significant safety events related to the therapy. Data also demonstrated that a difference in visual function between the treated and untreated eyes was still observed at month 36. The benefit-risk profile of AGTC-501 supports ongoing clinical development for the treatment of patients with XLRP caused by RPGR mutations. Lance Baldo, MD, Chief Executive Officer of Beacon, stated, "This emerging longer-term data is another clinical validation of the safety of AGTC-501 for the treatment of XLRP. We look forward to achieving several upcoming clinical milestones, including 24-month data from the Phase 2 SKYLINE trial in XLRP later this year, and continued enrollment into our open-label Phase 2 DAWN trial and Phase 2/3 VISTA trial." HORIZON is a Phase 1/2, open-label, dose-escalation study of patients with XLRP treated with subretinal AGTC-501, which has completed enrollment of 29 male participants and all participants are in long-term follow-up. Presentation – Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) Phase 1/2 Multicenter Study (HORIZON): 36-Month Interim Results Presenter – Paul Yang, MD, PhD, Chief, Paul H. Casey Ophthalmic Genetics Division, Casey Eye Institute, OHSU The presentation took place on Thursday, September 19th at 15:30 CEST. 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), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting 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 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. AGTC-501 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. 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

CAMBRIDGE, MA USA I September 3, 2024 I BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. “We are pleased with the immense progress we have made to date and thrilled that the FDA has cleared our IND application to initiate clinical testing for OpCT-001,” said Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics. “We believe that OpCT-001 has potential to restore vision in people living with primary photoreceptor diseases and look forward to working with the ophthalmology community in initiating our Phase 1/2a clinical study.” OpCT-001 is the first investigational iPSC-derived cell therapy to be clinically evaluated for the treatment of primary photoreceptor diseases. Initiation activities to support a Phase 1/2a study are underway. The Phase 1/2a study is designed as a first-in-human study to evaluate the safety and tolerability of subretinal administration of OpCT-001 in people with primary photoreceptor diseases as well as evaluate the effect of OpCT-001 on retinal structure, visual function and functional vision. The study will assess several dose levels of OpCT-001 and is expected to enroll participants in sites across the U.S. Primary photoreceptor diseases are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy. These diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. Primary photoreceptor diseases affect an estimated 110,000¹ people in the U.S. and only limited treatment options exist for this population. OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated cells in the retina with functional cells. OpCT-001 was exclusively licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January 2024 as part of the strategic R&D and clinical manufacturing collaboration between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics forged in 2021. As part of the collaboration, FUJIFILM Cellular Dynamics supported BlueRock Therapeutics via research, development and the execution of critical IND-enabling activities including the clinical manufacturing of OpCT-001 at their cGMP² facility in Madison, Wisconsin. OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. ¹https://www.ncbi.nlm.nih.gov/books/NBK519518/ ; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1808442/ ; https://www.nidcd.nih.gov/health/usher-syndrome#b ²Current good manufacturing practices are regulations enforced by the FDA. About BlueRock Therapeutics LP BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the U.S. FDA (Food and Drug Administration); has completed Phase 1 clinical trial and is progressing to the next stage of clinical development. OpCT-001 has been cleared by the FDA to begin Phase 1 clinical testing. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com . Find more information at https://pharma.bayer.com Follow us on Facebook: http://www.facebook.com/bayer SOURCE: Bluerock Therapeutics