Beacon Therapeutics (USA), Inc.

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

Purespring Therapeutics has reeled in £80 million ($105 million) to test two gene therapies in humans. The Sofinnova Partners-led Series B, disclosed Wednesday morning, will carry the London startup into an open-label Phase 1/2 next year for a kidney disease that has attracted a lot of attention in recent years from regulators and pharmaceutical acquirers. The startup’s lead gene therapy, PS-002, will be investigated for IgA nephropathy, or IgAN. The autoimmune and kidney disease, in which too much IgA protein stays in the kidneys, can lead to end-stage renal disease. Purespring anticipates it could snag an accelerated approval from its upcoming Phase 1/2, CEO Julian Hanak told Endpoints News . Travere , Novartis and Calliditas have received FDA nods in the past few years for their oral medicines. During that approval process, Purespring would plan to launch a confirmatory Phase 3 to get full approval, Hanak said. To fund that, Purespring would have a few options: raise a crossover round, go through an IPO and/or secure a commercialization partnership with a pharma company, the CEO said. Purespring’s experimental gene therapy goes after a particular cell type known as the podocyte, which is key to a kidney’s ability to operate. Judo Bio, a new siRNA startup that emerged earlier this week with $100 million, also plans to target podocytes to directly treat renal diseases. Purespring’s candidate “actually could work in conjunction and in complementation with some of the new therapies coming on the market,” Hanak said. “It’ll be a one-and-done therapy because it’s gene therapy and because podocytes are totally differentiated, non-dividing cells. Once the gene therapy is in them, we’ll get persistence of expression.” There’s been a wave of M&A in the IgAN field, with Novartis , Vertex and Biogen each dishing out more than $1 billion to buy clinical-stage drug developers Chinook, Alpine Immune Sciences and Human Immunology Biosciences, respectively, in the past 15 months. “I think the industry has woken up to the fact that this is a huge unmet medical need,” Hanak said. He initially joined Purespring as chief development officer in 2021, coming from Biogen, where he served as CMC leader for the gene therapy work acquired from Nightstar . Purespring could go into “any other complement-mediated kidney disease affecting podocytes, so lupus nephritis, ANCA vasculitis and so on,” Hanak said, but the initial focus is on IgAN and an undisclosed second indication that will enter the clinic before the Series B runs out in a few years. The startup could also ink partnerships over the coming two years, the CEO said. “Companies have been approaching us about access to our platform to deliver their genes to the kidney,” he added. It has an internal process development manufacturing setup, Hanak said, noting the company also works with Charles River for plasmid DNA and with Columbus, OH-based Andelyn Biosciences for vector supplies. “We have to get the cost of goods for the product down, which we’ve been able to do,” Hanak said. “Our dose is very low, 50-to-100-fold lower than a traditional, systemic gene therapy because of our platform.” Purespring derived from the work of University of Bristol professor Moin Saleem, who works in pediatric renal medicine. The company launched with a £45 million Series A from Syncona in November 2020. Syncona returned for the Series B alongside Forbion. The respective UK and Dutch investors have paired up with other gene therapy companies before, including a $170 million round for ophthalmology-focused Beacon Therapeutics in July. Other Purespring investors include Gilde Healthcare and British Patient Capital. The company has 46 employees and doesn’t plan to rapidly hire. “We have all the juice we need to deliver,” Hanak said.

Swiss anti-aging biotech Rejuveron's most advanced med is the early clinical stage EA-2353 program, in trials for retinitis pigmentosa, which can cause vision loss. Swiss anti-aging biotech Rejuveron is seeking its rejuvenation as it morphs into Centenara Labs.The biotech, as Rejuveron Life Sciences AG, raised $75 million in dual tranche series B and convertible loan financing one year ago this month and is focused on a small-molecule approach to regenerate endogenous stem cells.Its most advanced med is the early clinical stage EA-2353 program, in trials for retinitis pigmentosa (RP), which can cause vision loss. This month, new data for the med have also been released, which shows “encouraging preliminary results,” predominately in safety.While the startup is still at the beginning of its journey, the name change comes amid a “wider rebranding initiative” set up for “differentiation in the marketplace,” as well as boosting awareness of the company, it said in a statement.Several companies are in clinical trials are seeking to treat RP, including gene therapy biotech Beacon Therapeutics, which is working on AGTC-501, a phase 2/3 therapy for the treatment of X-linked RP.Nanoscope Therapeutics is also using a gene therapy approach with MCO-010, which saw positive data presented earlier this year.“Our transition to Centenara is a bold and forward-thinking step that aligns with our strategic vision and represents more than just a new name; it underscores a renewed focus on working together to build a business that can successfully develop therapies for regenerative medicine and age-related disorders,” said Aksana Labokha, CEO of Centenara.“Our updated visual identity demonstrates our evolution and sets us apart within the biotechnology sector putting a focus on the drivers of an extended quality of life.”Biotechs focusing on aging have reveled in the hype over the years, though some struggled as that initial hype failed to live up to expectations. This includes anti-aging biotech Unity Biotechnology, which was forced to ditch its leading knee pain program in 2020 after it failed a key midstage test.Another anti-aging biotech, AgeX Therapeutics, also saw trouble and appeared locked in limbo for years as it consistently warned that its cash was about to run out while its preclinical pipeline stalled. It had a reprieve last year when Serina Therapeutics decided to act on the opportunity to leverage AgeX’s public listing and merge with the company.