National Eye Institute

FORT LEE, N.J.--(BUSINESS WIRE)--Luxa Biotechnology LLC (Luxa), a clinical-stage biotechnology company developing novel adult retinal pigment epithelial stem cell (RPESC) therapies for dry age-related macular degeneration (dry AMD), today announced the company will present Phase 1/2a clinical and preclinical data on the company’s adult retinal pigment epithelial stem cell (RPESC) therapy for dry age-related macular degeneration (AMD) at the 2025 ARVO Annual Meeting taking place May 4-8, 2025 in Salt Lake City, Utah. Clinical outcomes demonstrating vision improvement from the low-dose cohort of the ongoing first-in-human Phase 1/2a clinical trial (NCT04627428) will be shared. “At ARVO, we will present data on our low-dose clinical outcomes and the rigorous preclinical data that laid the foundation for our therapy,” said Dr. Keith Dionne, CEO of Luxa. “The interim clinical data reinforces the potential of our RPESC-RPE-4W cell therapy to restore vision and potentially alter the course of dry AMD. These findings represent an important step toward addressing the significant unmet needs of patients affected by this debilitating condition.” Details of the two podium presentations are as follows: Title: Safety and Tolerability of RPE Stem Cell-derived RPE (RPESC-RPE) Implantation in Patients with Dry AMD: Early Low Dose Clinical Outcomes Presenter: Dr. Jeffrey Stern Session: Clinical Advances in Cell and Gene Therapy Number: Paper Session, Presentation #2701 Date/Time: Tuesday, May 6, 2025 | 9:00–9:15 AM MT Location: Ballroom B Title: Preclinical Safety and Efficacy of an Adult Progenitor Cell Product, RPESC-RPE-4W, as Therapy for Dry AMD Presenter: Dr. Brigitte Arduini Session: Stem Cell and Gene Therapy Number: Paper Session, Presentation #876 Date/Time: Sunday, May 4, 2025 | 3:15–3:30 PM MT Location: Room 255E About Dry AMD Age-related macular degeneration (AMD) is the leading cause of adult-onset blindness in the Western world, affecting approximately 20 million Americans and 200 million people globally, with over 80% of cases classified as the dry form. Dry AMD causes diminished vision accompanied by loss of central retinal pigmented epithelium (RPE) cells in individuals aged 55 and older. There are no approved vision-improving treatments for dry AMD. About Luxa Biotechnology Luxa Biotechnology is a joint venture of the Korean technology company Y2 Solution and the Neural Stem Cell Institute (NSCI) in Albany, New York. Luxa licensed RPESC-RPE-4W from NSCI and maintains a robust research program at NSCI to advance RPESC-RPE-4W as an effective, commercially viable therapy to restore the lost vision of AMD patients. Luxa acknowledges the support and critical contributions of the clinical team at the University of Michigan Kellogg Eye Center, the manufacturing team at the Cedars Sinai Biomanufacturing Center, the California Institute for Regenerative Medicine, the U.S. National Institutes of Health, and the National Eye Institute. For more information, visit our website and follow us on LinkedIn.

FORT LEE, NJ, USA I March 06, 2025 I Luxa Biotechnology LLC (Luxa), a clinical-stage biotechnology company developing a novel adult retinal pigment epithelial stem cell (RPESC) therapy for dry age-related macular degeneration (AMD), today announced clinical data from its Phase 1/2a clinical trial evaluating RPESC-RPE-4W, a proprietary retinal pigment epithelium (RPE) cell therapy for patients with dry age-related macular degeneration (dry AMD). The data were presented today at the 77th Annual Wills Eye Conference in Philadelphia, Pennsylvania. Findings from six patients in the first low-dose cohort of the ongoing first-in-human trial were presented ( NCT04627428 ). Each patient received a 50,000-cell suspension of RPESC-RPE-4W implanted under the macula. Post-implant clinical outcomes were assessed over 12 months for the worse-seeing Group I and over 3 months for the better-seeing Group II. No serious adverse events (SAEs) related to the investigational therapy were observed. Best Corrected Visual Acuity (BCVA) improvements were substantial, with Group I experiencing an average gain of +21.67 ETDRS letters at 12 months and Group II showing a +3.3-letter improvement at 3 months. These gains are particularly meaningful given that even a 5- to 10-letter improvement can translate into a real-world functional benefit for patients, such as improved reading ability, better recognition of faces, and enhanced mobility. The patients with the most severe baseline vision impairment and the greatest clinical need achieved the most significant vision restoration, reinforcing the potential of this therapeutic approach to address the substantial unmet need in advanced dry AMD. Meanwhile, individuals who began with better baseline vision saw smaller, yet still clinically relevant, improvements that counteracted the natural decline expected in dry AMD progression. This reversal of disease trajectory underscores the potential for preserving vision in earlier-stage patients, possibly delaying or even preventing progression to severe visual impairment. “These findings are encouraging, as they demonstrate not only meaningful vision improvements in patients with the greatest need but also the potential to alter the trajectory of dry AMD in those with better baseline vision,” said Dr. Jeffrey Stern, Chief Medical Officer of Luxa. “The substantial BCVA gains, particularly in worse-seeing patients, highlight the promise of our approach in restoring vision, while the modest improvements in better-seeing individuals suggest a beneficial impact in slowing disease progression. This reinforces our commitment to advancing innovative treatments that address the urgent unmet needs in dry AMD.” Last month, Luxa announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation for RPESC-RPE-4W in the treatment of dry AMD. This designation will further facilitate the development and regulatory progress of the novel adult stem cell therapy. “We are thrilled with these promising clinical outcomes, which mark a significant step forward in our progress towards addressing a major unmet need in dry AMD,” said Dr. Keith Dionne, CEO of Luxa. “This achievement underscores our commitment to reversing vision loss for millions of adults affected by dry AMD and geographic atrophy. We look forward to advancing our Phase 1/2a trial and evaluating higher dose cohorts.” About Dry AMD Age-related macular degeneration (AMD) is the leading cause of adult-onset blindness in the Western world, affecting approximately 20 million Americans and 200 million people globally, with over 80% of cases classified as the dry form. Dry AMD causes diminished vision accompanied by loss of central retinal pigmented epithelium (RPE) cells in individuals aged 55 and older. There are no approved vision-improving treatments for dry AMD. About Luxa Biotechnology Luxa Biotechnology is a joint venture of the Korean technology company Y2 Solution and the Neural Stem Cell Institute (NSCI) in Albany, New York. Luxa licensed RPESC-RPE-4W from NSCI and maintains a robust research program at NSCI to advance RPESC-RPE-4W as an effective, commercially viable therapy to restore the lost vision of AMD patients. Luxa acknowledges the support and critical contributions of the clinical team at the University of Michigan Kellogg Eye Center, the manufacturing team at the Cedars Sinai Biomanufacturing Center, the California Institute for Regenerative Medicine and the U.S. National Institutes of Health and the National Eye Institute. For more information, visit our website and follow us on LinkedIn . SOURCE: Luxa Biotechnology

Thirteen out of 14 people with eye damage experienced at least some improvements to their cornea after receiving a stem cell transplant in one eye in an early-stage study. The National Institutes of Health-funded Phase 1/2 trial was led by ophthalmologist Ula Jurkunas at Massachusetts Eye and Ear, and reported in Nature Communications. The patients in the study mostly had chemical or thermal burns to one eye, such as from a fireworks accident. There are stem cell treatments approved in other countries, such as Holoclar in the EU, to treat damage on the outer layer of the eye, or what’s known as limbal stem cell deficiency, when the eye can no longer properly regenerate its cornea usually after an injury. But the study authors said that the manufacturing procedures used in those treatments include methods that may not be FDA-compliant. “The FDA doesn’t like us to use antibiotics in the media, because that could cover up a potential infection,” said Dana-Farber Cancer Institute’s Jerome Ritz, who led the manufacturing of the stem cell therapies in the study. “The other procedures will grow the cells on ‘feeder cells.’ So, they actually have mouse cells that they grow first, and then they put the human cells on these mouse feeder cells. And the FDA doesn’t like that either,” Ritz added. “Then, the other thing is the FDA doesn’t like using serum,” he said, referring to a kind of supplement added to cell cultures that often come from animals. Jurkunas is a co-founder of the biotech OcuCell, which is working to develop cell therapies for eye conditions including limbal stem cell deficiency. Other biotechs such as Aurion Biotech (which is currently in a legal spat with its investor, the eye giant Alcon) are likewise developing cell therapies for conditions related to the cornea. The researchers previously reported early results from four people in this trial in 2023. Fifteen people were enrolled in the Mass Eye and Ear study and had a biopsy to obtain cells from their healthy eye. Of those patients, stem cell therapies were successfully manufactured for 14 patients. One patient needed two biopsies for their stem cell treatment to be successfully made. A key limitation of the study was that patients needed one eye that was healthy enough so the researchers could take a biopsy to make the stem cell therapy. Of the 14 participants who received transplants, 11 saw improvement in the outermost layer of their cornea and were considered “complete success” at one year. Two more were deemed a “partial success,” meaning they had improvements in their symptoms or fewer blood vessel formations around their eye, which can cause the cornea to become opaque. One patient saw no improvements. Jurkunas noted that vision wasn’t part of the key endpoint for the study, because the outer layer of the cornea is only one of the layers that is impacted when someone has a major eye injury. “There are many other layers in the cornea and the eye can get affected. And even though we restore the surface of the cornea, that does not mean that we fully restore all other layers, and the vision can still be impaired. However, when we restore epithelial cells, we can do other surgeries to improve the vision,” she said. “In some patients, it’s the first step towards next procedures like a full corneal transplant,” she added, noting that two patients went on to receive full corneal transplants after receiving the stem cell transplant. Jurkunas said although vision improvements were not the goal of the study, 71% saw vision improvements. In addition, there were no serious safety events in the donor or recipient’s eye. The researchers wrote in their paper that the next steps include developing an allogeneic stem cell therapy, where the cells would be from a donor and not from the patient’s other eye. That could enable treating patients with injuries to both eyes and at a greater scale. The clinical trial results are coming out at a time when NIH funding is coming under attack from the Trump administration. “I would point out that not only was the clinical trial funded — that was funded by the National Eye Institute — but also the pre-clinical development going back 10, 12 years, that was also funded by the NIH,” Ritz said. “We’ve been very fortunate to have federal support. We haven’t had commercial support for any of this. That’s all been done through funding from the NIH.” “The important part is that commercial partners probably wouldn’t necessarily find it appealing to fund these early-stage trials because they just don’t have as much evidence of the feasibility,” Jurkunas said. “So it was really critical for NIH to fund our trial in this early stage.”