dry age-related macular degeneration

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.

ROCKVILLIE, Md. and SUZHOU, China, March 18, 2024 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, today announced that the primary endpoint has been met in the second Phase 2 clinical study of efdamrofusp alfa high-dose, a recombinant human VEGFR-Fc-Human CR1 fusion protein injection (R&D code: IBI302), in Chinese subjects with neovascular age-related macular degeneration (nAMD). According to the results of the two Phase 2 clinical studies conducted in more than 360 subjects of nAMD, compared with Aflibercept, IBI302 can be administrated in long-interval (every 12 weeks), while providing a stable and robust visual benefit and anatomic improvements, as well as potential inhibition effect in macular atrophy. Based on those results, Innovent advanced IBI302 8mg into a Phase 3 clinical study STAR in October 2023. This was a randomized, double-masked, active-controlled Phase 2 clinical study (NCT05403749), evaluating the longer interval of intravitreal injection of high-dose IBI302 in subjects with nAMD. A total of 132 subjects were randomized 1: 1: 1 to IBI302 6.4 mg group, IBI302 8.0 mg group, or Aflibercept 2.0 mg group. After the loading therapy, subjects in IBI302 6.4 mg group and 8.0 mg group were dosed with adjusted intervals of every 8 weeks (Q8W) or every 12 weeks (Q12W), depending on response to loading therapy. Subjects in Aflibercept 2.0 mg group were dosed Q8W after the loading therapy. The primary endpoint was the change in best corrected visual acuity (BCVA) in the study eye from baseline to week 40. The study lasted for 52 weeks. The results showed that the primary endpoint was successfully met: at week 40, the IBI302 6.4 mg and 8.0 mg groups showed non-inferior BCVA gains to the Aflibercept 2.0 mg group. The mean BCVA improvement from baseline was 10.5 ETDRS letters for the IBI302 6.4 mg group, 11.0 ETDRS letters for the IBI302 8.0 mg group, and 9.8 ETDRS letters for the Aflibercept 2.0 mg group at week 40. The mean change from baseline in central subfield thickness (CST) was -163.19 μm for the IBI302 6.4 mg group, -184.46 μm for the IBI302 8.0 mg group, and -108.23 μm for the Aflibercept 2.0 mg group at week 40. In addition, approximately 81%, 88% of subjects in 6.4 mg IBI302 groups and 8.0mg IBI302 groups respectively were able to extend dosing interval to Q12W, similar to that in the proportion of subjects dosed Q12W or longer with Aflibercept 8.0 mg (83% in PULSAR trial)1 or Faricimab (TENAYA & LUCERNE trial, with 79.7% and 77.8% respectively)2 by indirect comparison. Based on the excellent long-interval dosing performance in the Phase 2 studies, the Phase 3 study STAR added Q16W dosing interval regimen for IBI302. The overall safety profile of IBI302 was favorable, comparable to Aflibercept 2.0 mg, and consistent with previous studies. No new safety signals were identified. Detailed study data will be further analyzed and published in the near future. Professor Xiaodong Sun, Principal Investigator of the Study, Head of National Center for Clinical Ophthalmology, Shanghai General Hospital, stated: "Intravitreal injection of anti-VEGF drugs is currently the first-line treatment for nAMD, but there are still unmet clinical needs given their frequent intravitreal injection and gradual loss of visual benefits. Exploring longer interval dosing and anti-macular atrophy are necessary and urgent. IBI302 is a global first-in-class anti-VEGF-anti-complement bispecific molecule. As the principal investigator for IBI302 trials, I am very pleased to see that this Phase 2 study met the primary endpoint and demonstrated the potential for long-interval dosing. These results will be further validated in the pivotal trial of IBI302. I look forward to providing a new treatment option for nAMD patients." Dr. Lei Qian, Vice President of Clinical Development of Innovent, stated: "There are two major trends in drug development for nAMD: extending dosing intervals and reducing the occurrence of macular atrophy. In the results of two Phase 2 studies, which enrolled over 360 subjects, IBI302 improved BCVA and macular edema in patients with nAMD significantly, extended dosing intervals, and had the potential to prevent the development of macular atrophy. Next, we will further investigate the long-interval dosing efficacy and safety of high-dose IBI302 in the Phase 3 STAR trial, hoping to bring a new generation of anti-VEGF agents to patients with nAMD." About neovascular age-related macular degeneration (nAMD) Age-related macular degeneration (AMD) is a progressive ocular disease involving the macular retina, leading to central visual impairment, the incidence of which increases with age. Neovascular age-related macular degeneration (nAMD) is one of the major forms of AMD, accounting for 15% to 20% of all AMD patients and is the leading cause of central vision loss in AMD patients over 65 years3. The incidence of AMD is increasing year by year in China, and it has become the third leading cause of blindness in China. The pathogenesis of nAMD has not been fully elucidated. It is generally accepted that angiogenesis induced by increased expression of VEGF is the main cause of nAMD, and inflammatory reaction mediated by abnormal activation of complement is also considered to be an important cause of AMD. Ocular anti-VEGF agents have led to significant visual benefits and changed the course of nAMD, but the frequent dosing (every 4 or 8 weeks) currently places a heavy burden on patients, families and society. In addition, the visual benefits of anti-VEGF drug therapy are gradually diminished year by year. In approximately two thirds of nAMD patients with a follow-up for over 7 years, the visual gains from anti-VEGF treatment significantly diminish4. Macular atrophy or retinal fibrosis are important causes of vision loss after long-term anti-VEGF therapy. Currently, drug development for nAMD is mainly focusing on extending the dosing intervals, and there are few drugs under investigation for macular atrophy or retinal fibrosis. Two drugs targeting complement have been approved by the US FDA in 20235,6 for the treatment of geographic atrophy secondary to dry AMD. About Efdamrofusp Alfa (IBI302) IBI302 is a recombinant fully human bispecific fusion protein of Innovent Biologics with global proprietary rights. The N-terminus is a VEGF domain that can bind to the VEGF family, block VEGF-mediated signaling pathway, inhibit vascular epithelium proliferation and angiogenesis, and improve vasopermeability and reduce leakage. The C-terminus of IBI302 is the complement binding domain that can inhibit the activation of the classic pathway and alternative pathway of complement through the specific binding of C3b and C4b, and reduce the inflammatory response mediated by the complement. IBI302 may exert its therapeutic effect by inhibiting both VEGF-mediated angiogenesis and complement activation pathways. About Innovent Innovent is a leading biopharmaceutical company founded in 2011 with the mission to provide high-quality biologics that are affordable to all. The company discovers, develops, manufactures and commercializes innovative medicines that treat some of the most intractable diseases. Its pioneering therapies to treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has 10 products in the market, 3 new drug applications under the NMPA review, 5 assets in Phase 3 or pivotal clinical trials and 18 more molecules in early clinical stage. Innovent partners with over 30 global healthcare leaders, including Eli Lilly, Roche, Sanofi, Adimab, Incyte and MD Anderson Cancer Center. Guided by the motto, "Start with Integrity, Succeed through Action," Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit , or follow Innovent on Facebook and LinkedIn. Statement: Innovent does not recommend the use of any unapproved drug (s)/indication (s). Forward-looking statement This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent Biologics ("Innovent"), are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialize or is otherwise inaccurate. SOURCE Innovent Biologics

PURCHASE, N.Y., March 15, 2024 (GLOBE NEWSWIRE) -- Cognition Therapeutics Inc. (Nasdaq: CGTX), a clinical-stage neuroscience company developing product candidates designed to treat neurodegenerative disorders by regulating cellular damage response pathways, today announced that the Company will release financial results for the fourth quarter and full year ended December 31, 2023 on Tuesday, March 26, 2024 before the market open. Following the release, management will host a conference call at 8:00 a.m. ET on the same day to review the financial and operating results and provide a business update. Conference Call Information: Telephone Access: US/Canada Participant Toll-Free Dial-in Number: (800) 715-9871 US/Canada Participant International Dial-In Number: (646) 307-1963 Conference ID Number: 6295507 Webcast Access: The audio webcast with live Q&A will be accessible at or via the Investor Relations section of Cognition’s website. An archive of the webcast and presentation will be available for 90 days beginning at approximately 10:00 a.m. ET on March 26, 2024. About Cognition Therapeutics: Cognition Therapeutics, Inc. is a clinical-stage biopharmaceutical company engaged in the discovery and development of innovative, small molecule therapeutics targeting age-related degenerative disorders of the central nervous system and retina. We are currently investigating our lead candidate CT1812 in clinical programs in Alzheimer’s disease, dementia with Lewy bodies (DLB) and dry age-related macular degeneration (dry AMD). We believe CT1812 and our pipeline of σ-2 receptor modulators can regulate pathways that are impaired in these diseases. We believe that targeting the σ-2 receptor with CT1812 represents a mechanism functionally distinct from other current approaches in clinical development for the treatment of degenerative diseases. More about Cognition Therapeutics and its pipeline can be found at Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this press release, other than statements of historical facts or statements that relate to present facts or current conditions, are forward-looking statements. These statements, including statements relating to the timing and expected results of our clinical trials, and cash runway, involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” might,” “will,” “should,” “expect,” “plan,” “aim,” “seek,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “forecast,” “potential” or “continue” or the negative of these terms or other similar expressions. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition, and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond our control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: competition; our ability to secure new (and retain existing) grant funding; our ability to grow and manage growth, maintain relationships with suppliers and retain our management and key employees; our ability to successfully advance our current and future product candidates through development activities, preclinical studies and clinical trials and costs related thereto; uncertainties inherent in the results of preliminary data and pre-clinical studies and earlier-stage clinical trials being predictive of the results of early or later-stage clinical trials; the timing, scope and likelihood of regulatory filings and approvals, including regulatory approval of our product candidates; changes in applicable laws or regulations; the possibility that the we may be adversely affected by other economic, business or competitive factors, including ongoing economic uncertainty; our estimates of expenses and profitability; the evolution of the markets in which we compete; our ability to implement our strategic initiatives and continue to innovate our existing products; our ability to defend our intellectual property; the impact of the COVID-19 pandemic on our business, supply chain and labor force;the impacts of ongoing global and regional conflicts; and the risks and uncertainties described more fully in the “Risk Factors” section of our annual and quarterly reports filed with the Securities Exchange Commission and are available at . These risks are not exhaustive and we face both known and unknown risks. You should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. Moreover, we operate in a dynamic industry and economy. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that we may face. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact Information: Cognition Therapeutics, Inc. info@cogrx.com Casey McDonald (media) Tiberend Strategic Advisors, Inc. cmcdonald@tiberend.com Mike Moyer (investors) LifeSci Advisors mmoyer@lifesciadvisors.com