Usher Syndrome, Type 2A

Webinar, moderated by Cantor Fitzgerald, will be held on Monday, May 13, 2024, at 2:00 p.m. ET Discussion will feature Dr. Michel Michaelides, leading expert in the field of inherited eye disease in adults and children SAN DIEGO, May 06, 2024 (GLOBE NEWSWIRE) --Belite Bio, LLC. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the executive management team will participate in a Key Opinion Leader Webinar hosted by Jennifer Kim, Director, Equity Research, of Cantor Fitzgerald on Monday, May 13, 2024, at 2:00 p.m. ET. The event will feature Michel Michaelides, BSc, MB BS, MD(Res), FRCOphth, FACS (University College London Institute of Ophthalmology and Moorfields Eye Hospital), who will discuss an overview of the unmet need for treatments in Stargardt disease (STGD1) and Geographic Atrophy (GA). Dr. Michaelides will also review the competitive landscape, real world applications for Tinlarebant in STGD1 and GA, and the additional analysis on data from Belite’s Phase 2 clinical trial in STGD1 recently published at ARVO. Tinlarebant (LBS-008) is a novel oral, once daily retinol binding protein 4 (RBP4) antagonist designed to reduce the accumulation of vitamin-A based toxins in the eye, which have been implicated in the onset and progression of STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S., Europe, and Japan for the treatment of STGD1. To register for the event, please click here. A replay of the webinar will be available approximately one week after the event for 90 days under the "Events" tab on the investor relations section of the Belite Bio website at: . About Michel Michaelides, BSc, MB BS, MD(Res), FRCOphth, FACS Dr. Michaelides is a Professor of Ophthalmology at the UCL Institute of Ophthalmology and a Consultant Ophthalmologist at Moorfields Eye Hospital in the departments of Medical Retina, Inherited Eye Disease and Pediatric Ophthalmology. He is a key member of a team developing gene and stem cell therapy at UCL and is a founding member and Head of Clinical Ophthalmology of a UCL gene therapy spin-out company, which was acquired by MeiraGTx and was till recently in partnership with Janssen to develop gene therapies for inherited retinal diseases. He has received a Career Development Award from the Foundation Fighting Blindness (USA), as well as the Department of Health/HEFCE ‘New Blood’ Clinical Senior Lectureship Award and is a member of Macula Society and Retina Society. With over 400 peer-reviewed publications and 35 book chapters. His research portfolio incorporates 8 on-going studies, including multi-center international natural history studies for USH2A-retinopathy, and he is also a principal investigator in 8 on-going retinal clinical trials investigating novel and established therapies. About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt Disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook, or visit us at . Media and Investor Relations Contact: Jennifer Wu ir@belitebio.com Julie Fallon belite@argotpartners.com

ProQR and Laboratoires Théa have finally reached a pact that fits both companies' needs. After an earlier version of the deal crumbled, ProQR has finally managed to sell off its ophthalmology assets to Laboratoires Théa, this time for €8 million ($8.6 million) with the potential to make up to €165 million ($177 million) in biobucks. Dutch biotech ProQR has been trying to offload RNA therapies sepofarsen and ultevursen for more than a year now, with hopes of turning its focus to targets for cholestatic and cardiovascular diseases. In August, the company seemed to have found a taker with eye care-focsed Théa. The two biotechs announced a deal in which Théa would pay €12.5 million ($13.7 million) upfront for the two assets, while also giving ProQR the chance to make up to €135 million ($148 million) in biobucks. The offer was short-lived though, with Théa canceling the pact at the end of September after ProQR failed to meet the conditions. The sale depended on Théa hiring several key ophthalmology personnel from ProQR to help build out a team specializing in inherited retinal disorders. However, some of the ProQR employees decided not to work at Théa, so the conditions weren’t met. Now, though, the two have closed on a revised agreement. ProQR plans to ask for confidential treatment from the Securities and Exchange Commission for certain parts of the amended deal, according to SEC documents. Théa now owns the rights to sepofarsen, which is designed to restore vision in people with the severe inherited retinal disease Leber congenital amaurosis 10, and ultevursen, a therapy to treat vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa. Sepofarsen previously failed to improve vision in a phase 2/3 trial and European regulators had asked for an additional pivotal study before ProQR could seek approval. The regulatory feedback prompted ProQR to halt trials of both sepofarsen and ultevursen, followed by a restructuring that included a 30% workforce reduction and eventually, a shift away from genetic eye diseases.

In a reversal of fortunes, ProQR Therapeutics’ ophthalmology asset sale to Laboratoires Théa has been canceled after ProQR failed to meet the conditions of the deal. In August, Dutch biotech ProQR announced its plan to offload RNA therapies sepofarsen and ultevursen to eye care company Théa for 12.5 million euros ($13.7 million) upfront and up to 135 million euros ($141.9 million) in biobucks. ProQR had initially put the two eye disease candidates up for sale about a year ago. Sepofarsen is designed to restore vision in people with the severe inherited retinal disease Leber congenital amaurosis 10. The candidate failed to improve vision in a phase 2/3 trial, and European regulators asked the biotech to run an additional pivotal study before seeking approval. The regulatory feedback prompted ProQR to halt trials of sepofarsen and ultevursen, a therapy designed to treat vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa. A two-phase reorganization saw ProQR lay off 30% of staff and later pull back from genetic eye diseases to go all in on its RNA-editing tech. However, Théa has now terminated its purchase of the eye disease candidates, according to a Sept. 27 release from ProQR. The deal had been expected to close this quarter. The sale depended on Théa hiring several key ophthalmology personnel from ProQR to help build out a team specializing in inherited retinal disorders. However, some of the ProQR employees decided not to work at Théa, so the conditions weren’t met. It’s unclear if ProQR will continue to try to sell the eye disease candidates, though the company’s focus is currently on targets for cholestatic and cardiovascular diseases, alongside its genetic medicine partnership with Eli Lilly, ProQR founder and CEO Daniel de Boer said in the release.