Eye Diseases, Hereditary

SBT-589 showed mitochondrial protection across models of Friedreich's ataxia SBT-589 displayed cardioprotective effects in aggressive mouse model of FA cardiomyopathy Ongoing studies are expected to build on these insights as SBT-589 progresses through developmental stage-gates NEEDHAM, Mass., March 19, 2024 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced today the presentation of new SBT-589 data demonstrating cardioprotective effects across pre-clinical models of Friedreich's ataxia (FA). The data were presented at the Wellcome Trust Conference on Mitochondrial Medicine – Therapeutic Development, held March 18-20, 2024, in Cambridge, England. FA is a chronic disease of metabolic disruption that is typically diagnosed in childhood or adolescence, is inexorably progressive and usually results in premature death from cardiomyopathy or heart failure. There are no approved therapies for the progressive cardiomyopathy, which is linked to severe mitochondrial dysfunction, and is the leading cause of early death in this devastating disease. SBT-589 is a promising novel molecule that acts on mitochondrial pathways essential for cellular health and energy production that are impaired in FA cardiomyopathy. To evaluate the potential of SBT-589 in FA, a series of studies were conducted in FA patient-derived cells, isolated heart mitochondria, and an aggressive mouse model of FA cardiomyopathy. SBT-589 improved bioenergetics in FA patient-derived cells and mitochondria. In an FA mouse model with prominent cardiac hypertrophy and aggressive mortality, mice treated with once-daily SBT-589 displayed significantly reduced cardiac hypertrophy and a delay in the onset of mortality compared to vehicle-treated mice. "Mitochondrial dysfunction is a central contributor to pathology in Friedreich's ataxia, a disease in which heart failure and sudden cardiac events are among the leading causes of mortality," said David A. Brown, Senior Vice President, Discovery. "Mitigating heart disease in FA is central to Stealth's ongoing efforts to improve the lives of individuals with FA. The new findings presented today support continued development of SBT-589 as a potentially disease-modifying therapy to address the unmet need in cardiomyopathy associated with Friedreich's ataxia." The data were presented by Dr. Laura E. Kropp, Senior Manager of Discovery Biology at Stealth, and recipient of the Keith Michael Andrus Cardiac Award from the Friedreich's Ataxia Research Alliance. About Friedreich's Ataxia Friedreich's ataxia is a rare genetic disease affecting an estimated 1 in 50,000 individuals in the United States. The disease is caused by a defect in the frataxin gene resulting in a relative deficiency of frataxin, leading to mitochondrial iron accumulation and oxidative stress. More than 90% of patients with Friedreich's ataxia experience progressive cardiomyopathy which usually becomes fatal by early adulthood and is the leading cause of early mortality. About Stealth BioTherapeutics Stealth BioTherapeutics is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for age-related and rare genetic diseases involving mitochondrial dysfunction. The Company is initiating Phase 3 clinical trials of elamipretide, its lead investigational product candidate, in dry age-related macular degeneration. Elamipretide is also being tested in a fully enrolled Phase 3 clinical trial in primary mitochondrial myopathy, a rare skeletal myopathic disease caused by nuclear DNA mutations, and is the subject of a New Drug Application for Barth syndrome, an ultra-rare cardioskeletal disease. The Company is evaluating its second-generation clinical-stage candidate, SBT-272, for ophthalmic and neurological disease indications. The Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates. Several of these lead compounds, including SBT-589, have progressed through pre-clinical development stage-gates and toward IND-enabling studies. Investor Contact Kendall Investor Relations Adam Bero, Ph.D. [email protected] [email protected] Media Contact Anna Stallman Communications Anna Stallman [email protected] SOURCE Stealth BioTherapeutics Inc.

ATLANTA, March 19, 2024 (GLOBE NEWSWIRE) -- Alimera Sciences, Inc. (Nasdaq: ALIM) (“Alimera”), a global pharmaceutical company whose mission is to be invaluable to patients, physicians and partners concerned with retinal health and maintaining better vision longer, announces that the first patient has been randomized in the DRCR Retina Network’s Protocol AL. The study is titled, “A Randomized Clinical Trial Evaluating Intravitreal Faricimab (6.0 mg) Injections or Fluocinolone Acetonide (0.19 mg) Intravitreal Implants vs Observation for Prevention of Visual Acuity Loss due to Radiation Retinopathy.” The study, chaired by Arun Singh, M.D. (Cole Eye Institute, Cleveland Clinic, Cleveland, Ohio), plans to include 600 participants with primary choroidal melanoma receiving treatment with plaque brachytherapy. It will assess development of macular edema and associated long-term visual acuity effects of consistent and continuous release of corticosteroid or repeated injections of anti-VEGF initiated near the time of radiation therapy compared to observation until macular edema develops in patients at risk for radiation retinopathy. “We are very excited that this study is underway as it has the potential to show ILUVIEN addressing another unmet need affecting the retina,” said Rick Eiswirth, President and Chief Executive Officer of Alimera. “We believe ILUVIEN with CONTINUOUS MICRODOSING™ delivery may prevent, delay or reduce the occurrence of the complication of radiation retinopathy and consequent vision loss when used in patients treated with plaque brachytherapy and look forward to the continuing progress of this study.” About Radiation Retinopathy Radiation retinopathy (RR) is a common complication after Iodine-125 plaque brachytherapy for choroidal melanoma. Although the initial radiation insult is immediate, clinical onset of RR is not seen until many months later and RR frequently progresses over time to profound vision loss. The Collaborative Ocular Melanoma Study reported that at three years, 45% of patients have visual acuity 20/200 or worse. A treatment that prevents the development of RR, or dampens its severity, would have important consequences for many of these eyes that may be successfully treated for choroidal melanoma but will likely develop severe RR and loss of vision over time. About the DRCR Retina Network (DRCR.net) The DRCR Retina Network was formed in 2002 and includes clinical sites and physicians throughout the United States and Canada. It is funded by the National Eye Institute (NEI), part of the National Institutes of Health, which is the federal government agency that funds medical research. The DRCR Retina Network is supported in overall operation and leadership by the office of the Network Chairs (Daniel F. Martin, M.D., Cole Eye Institute, Cleveland Clinic, Cleveland, Ohio and Jennifer K. Sun, M.D., M.P.H., Joslin Diabetes Center, Harvard Medical School, Boston, Massachusetts) and the Coordinating Center (Executive Director Adam R. Glassman, M.S., Jaeb Center for Health Research, Tampa, FL), and the National Eye Institute (Project Officer, Sangeeta Bhargava, Ph.D.). About ILUVIEN The Company’s primary product is ILUVIEN (fluocinolone acetonide intravitreal implant) 0.19 mg sustained release intravitreal implant, injected into the back of the eye. With its CONTINUOUS MICRODOSING™ technology, ILUVIEN is designed to release sub-microgram levels of fluocinolone acetonide, a corticosteroid, for 36 months, to reduce the recurrence of disease, enabling patients to maintain vision longer with fewer injections. ILUVIEN is approved in the U.S., Kuwait, Lebanon and the U.A.E. to treat diabetic macular edema (DME) in patients who have been previously treated with a course of corticosteroids and did not have a clinically significant rise in intraocular pressure. In 17 European countries, ILUVIEN is indicated for the treatment of vision impairment associated with chronic DME considered insufficiently responsive to available therapies. In March 2019, ILUVIEN received approval in the 17 countries under the Mutual Recognition Procedure for prevention of relapse in recurrent non-infectious uveitis affecting the posterior segment of the eye. The 17 European countries include the U.K., Germany, France, Italy, Spain, Portugal, Ireland, Austria, Belgium, Denmark, Norway, Finland, Sweden, Poland, Czechia, the Netherlands, and Luxembourg. The non-infectious posterior uveitis indication for ILUVIEN was launched in Germany and the U.K. in late 2019, Belgium in 2021 and Spain and Italy in 2022. Alimera markets YUTIQ® (fluocinolone acetonide intravitreal implant) 0.18mg for treatment of uveitis in the United States. About Alimera Sciences, Inc. Alimera Sciences is a global pharmaceutical company whose mission is to be invaluable to patients, physicians and partners concerned with retinal health and maintaining better vision longer. For more information, please visit . Forward Looking Statements This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, Alimera’s expectations with respect to ILUVIEN’s potential to address another unmet need affecting the retina. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, ILUVIEN’s ability to prevent or reduce the occurrence of the complication of radiation retinopathy and consequent vision loss when used in patients treated with plaque brachytherapy, as well as other factors discussed in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Alimera’s Annual Report on Form 10-K for the year ended December 31, 2023 which is on the Securities and Exchange Commission (SEC) and available on the SEC’s website at All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. Alimera cautions investors not to rely too heavily on the forward-looking statements Alimera makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). Alimera undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise. For press inquiries: Cassy Dump for Alimera Sciences 619-971-1887 cassydoespr@gmail.com For investor inquiries: Scott Gordon for Alimera Sciences scottg@coreir.com

Pictured: Sign above the entrance of a U.S. courthouse/iStock, Mariakray The U.S. Court of Appeals for the Second Circuit on Monday overturned a lower court’s decision on the antitrust case between Regeneron and Novartis regarding their respective anti-VEGF eye treatments Eylea (aflibercept) and Lucentis (ranibizumab). The appellate court sided with Regeneron, agreeing with the pharma that a district court had wrongly dismissed its antitrust claims against Novartis because anti-VEGF therapies sold in pre-filled syringes (PFS) constitute their own and distinct market versus therapies sold in vials. The lower court approached the case believing that vials and PFSs operated in the same market and therefore applied the wrong legal standard, Circuit Judge Barrington Parker wrote in Monday’s decision. “The district court focused on the functional similarities between the PFS and vial versions of Regeneron’s and Novartis’s respective anti-VEGF treatments and concluded that, because of those similarities, both versions compete in the same relevant market,” Barrington wrote. “The pivotal issue in this appeal is whether anti-VEGF medications in vials and PFSs compete in the same or different product market,” Barrington wrote, adding that the court agrees with Regeneron’s assertion that anti-VEGF therapies administered through PSFs “constitute their own product market—distinct from the market for vials.” A Regeneron spokesperson in a statement to Reuters said that the company is “encouraged by the Second Circuit’s decision.” Novartis, meanwhile, said it was “disappointed with the court’s ruling,” adding that it continues “to believe Regeneron’s claims lack merit.” Regeneron’s Eylea and Novartis’ Lucentis are anti-VEGF therapies that work by slowing the growth of abnormal blood vessels in the eye, targeting several ophthalmic diseases such as wet age-related macular degeneration and retinal vein occlusion. Originally, anti-VEGF therapies were dispensed in vials and would be administered via an injection. According to Monday’s court document, this process was “cumbersome” and could aggravate the risk of complications when done incorrectly. Regeneron in 2005 collaborated with contract manufacturing company Vetter to develop a PFS syringe of Eylea, which would make administration safer and more convenient. However, the pharma alleges that Vetter “confidentially entered into a similar agreement with Novartis” in 2009, which eventually culminated in a patent covering a PFS product containing an anti-VEGF therapy. Novartis and Vetter then allegedly engaged in other “anti-competitive steps” to “keep Regeneron out of the anti-VEGF PFS market,” which pushed back the launch of Eylea’s PFS product to 2019. Regeneron sued Novartis in July 2020 based on these allegations, according to the court document on Monday. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.