Semmelweis University

VIENNA, Va.--( BUSINESS WIRE )-- CEL-SCI Corporation (NYSE American: CVM) today reported it will report new data from its Phase 3 study of Multikine (Leukocyte Interleukin, Injection)* at the European Society for Medical Oncology (ESMO) 2024 Congress which takes place from September 13 – 17, 2024 in Barcelona, Spain. A poster titled “Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy” will be presented by the study’s co-author József Tímár MD, PhD, DSc, a prominent and highly respected pathologist. CEL-SCI has received the go-ahead from the U.S. Food and Drug Administration (FDA) to commence a confirmatory Registration Study of Multikine in the treatment of head and neck cancer based on strong safety and efficacy data from its IT-MATTERS completed Phase 3 study. Dr. Timar is Professor Department of Pathology, Forensic and Insurance Medicine at Semmelweis University in Budapest, Hungary, and served as the Director of the Central Pathology Laboratory for the IT-MATTERS study. With 174 peer reviewed studies published, Dr. Timar is a founding editor, editor in chief, or a member of the editorial board of four oncology journals. He is the recipient of a dozen honors and awards for excellence in cancer research and teaching. About CEL-SCI Corporation CEL-SCI believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival. Multikine is designed to help the immune system "target" the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor. Multikine (Leukocyte Interleukin, Injection), a true first-line cancer therapy, has been dosed in over 740 patients and received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck. Multikine significantly extended life in its target patient population demonstrating a 73% survival rate with Multikine vs. only 45% without Multikine at 5 years after treatment. Based on this very strong data, the FDA agreed to CEL-SCI’s target patient selection criteria and gave the go-ahead to conduct a small, focused, confirmatory Registration Study which will enroll 212 patients. CEL-SCI will enroll newly diagnosed locally advanced primary head and neck cancer patients who present with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy), representing over 100,000 patients (globally) annually. The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2023. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. * Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.

Dr. Scholl is a globally recognized leader in the field of ophthalmology and the coordinating principal investigator of the largest ever natural history study of Stargardt disease SAN DIEGO, Sept. 01, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), 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 its board of directors has appointed Hendrik P. N. Scholl, MD, MA, as the Chief Medical Officer of the Company, effective immediately. Dr. Scholl is the foremost globally recognized authority on Stargardt disease and age-related macular degeneration (AMD), bringing decades of expertise in treating retinal diseases, including the two key indications targeted by Belite Bio's lead drug candidate, Tinlarebant. “Dr. Scholl has pioneered some of the most significant ophthalmology advancements of our time, and we are incredibly honored and excited to welcome him as our Chief Medical Officer,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “Dr. Scholl has served as Chair of the Data and Safety Monitoring Board for both our Phase 2 and Phase 3 Stargardt disease trials, and his decision to join Belite further validates our belief in Tinlarebant’s immense potential to improve the lives of patients living with debilitating macular diseases. His leadership, expertise, and commitment to addressing this area of high unmet need has and will continue to be invaluable as we advance our clinical programs.” “I am excited to join Belite Bio at this pivotal time in the Company’s development,” said Dr. Scholl. “I have dedicated my career to improving the lives of people suffering from serious retinal and macular diseases, and I am deeply impressed by Belite Bio’s pioneering approach to developing transformative therapies for Stargardt disease and dry AMD, which have historically lacked effective treatment options. I look forward to working with the team to advance these programs and bring hope to patients around the world.” Dr. Scholl served as the founding and scientific co-director of the Institute of Molecular and Clinical Ophthalmology Basel (IOB) and Professor of Ophthalmology at the University of Basel, where he also led the Department of Ophthalmology as its Chairman. He currently serves as President of the European Vision Institute as well as Chairman of the largest clinical research network in ophthalmology in Europe, EVICR.net, and its Expert Committee on Retinal Dystrophies. He is also the Founder and President of the Swiss Association for Research in Vision and Ophthalmology (ARVO-SWISS). Dr. Scholl’s distinguished career in academia includes leadership positions at several key academic institutions. Recently, he served as Professor of Ophthalmology and Endowed Chair at the Wilmer Eye Institute of Johns Hopkins University Medical School. At the Johns Hopkins Hospital, he was the Head of the Retinal Degeneration Clinic and the Director of the Visual Neurophysiology Service. For the Wilmer Eye Institute, he also served as the Co-director of the Johns Hopkins Center for Stem Cells and Ophthalmic Regenerative Medicine. Dr. Scholl is the coordinating principal investigator of the largest natural history study of Stargardt disease (ProgStar Study), which enrolled 365 subjects. Throughout his career, he has participated in over 10 clinical studies both in Stargardt disease and AMD, authored over 280 articles and reviews in peer-reviewed journals, and received numerous prestigious awards, including the European Vision Award, the President’s Award of the American Society of Retinal Specialists, the W. Richard Green Award and the Paul Henkind Memorial Award of the Macula Society, the Swiss Alfred-Vogt Award, and the Kupfer award of ARVO. He holds an honorary doctorate from Semmelweis University in Budapest, Hungary, and is Adjunct Professor at the Medical University of Vienna, Austria. Over the course of his 25 years of experience, Dr. Scholl has led and participated in numerous boards and advisory committees. He currently serves on the Scientific Advisory Board of Pro Retina Deutschland, Foundation Fighting Blindness, Erasmus University Medical Center, AIBILI, and the Institut de la Vision (Paris); on the Investment Advisory Board of Droia NV; and the Data Safety Monitoring Board of Roche Holding AG and ViGeneron GmbH. Dr. Scholl graduated from the University of Tübingen, Germany with a Doctor of Medicine (Dr. med.) and Master of Arts. He completed his specialist training at the University Eye Hospital in Tübingen before being awarded a fellowship from the German Research Foundation (DFG) at Moorfields Eye Hospital and Institute of Ophthalmology in London, and a DFG Heisenberg Professorship for Macular Diseases at the Eye Clinic of the University of Bonn, Germany. About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs such as Stargardt Disease and Geographic Atrophy 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 Stargardt subjects and a Phase 3 study (PHOENIX) in subjects with Geographic Atrophy. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. Media and Investor Relations Contact:Jennifer Wu : ir@belitebio.com Julie Fallon : belite@argotpartners.com

Late-breaking data from phase 3 KONFIDENT trial of sebetralstat published in NEJM and presented concurrently at the European Academy of Allergy and Clinical Immunology Congress 2024 Interim data from KONFIDENT-S open-label trial show median time to treatment of 9 minutes and median time to beginning of symptom relief for laryngeal attacks 1.3 hours CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)-- KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced that late-breaking data from the pivotal phase 3 KONFIDENT trial and open label KONFIDENT-S extension trial evaluating the efficacy and safety of sebetralstat were presented as late-breaking presentations at the European Academy of Allergy and Clinical Immunology Congress 2024. The results of KONFIDENT were simultaneously published in The New England Journal of Medicine. “The phase 3 KONFIDENT results represent a major advance for people living with HAE and reinforce our commitment to addressing critical gaps in the current standard of care,” said Paul Audhya, MD, MBA, KalVista’s Chief Medical Officer. “The efficacy and safety data presented as a late-breaker at EAACI and simultaneously published in The New England Journal of Medicine support the potential of sebetralstat as the first new treatment approach for the on-demand treatment of HAE in more than a decade, and the first potential oral therapy for this indication.” As presented and published, the KONFIDENT trial met its primary and key secondary endpoints and demonstrated a safety pro different from placebo. Phase 3 KONFIDENT Trial Results Median time from attack onset to treatment was 41 min (Q1, 6; Q3, 140) with 55.7% of participants treating in less than 1 hour 42.8% of attacks treated when still mild Primary endpoint: Time to beginning of symptom relief was significantly faster than placebo (p<0.0001 for 300 mg sebetralstat, p=0.0013 for 600 mg sebetralstat) Median time to beginning of symptom relief was 1.61 hours with sebetralstat 300 mg (IQR, 0.78-7.04), 1.79 hours with sebetralstat 600 mg (IQR, 1.02-3.79), and 6.72 hours with placebo (IQR, 1.34->12) Key secondary endpoints: Significantly faster time to a reduction in attack severity from baseline compared to placebo (p=0.0036 for 300 mg sebetralstat, p=0.0032 for 600 mg sebetralstat) Significantly faster time to complete attack resolution compared to placebo (p=0.0022 for 300 mg sebetralstat, p<0.0001 for 600 mg sebetralstat) Primary and key secondary endpoints were analyzed in a fixed, hierarchical sequence and adjusted for multiplicity. Subgroup analyses showed a consistency of treatment effect in among attacks regardless of attack severity, attack location, use of long-term prophylaxis, or geography. The safety of sebetralstat at both dose levels was no different than placebo. There were no patient withdrawals due to any adverse events and no treatment-related serious adverse events (SAEs) were observed. Treatment-related adverse event rates were 2.3% for 300 mg sebetralstat, 3.2% for 600 mg sebetralstat, and 4.8% for placebo. “For many years, HAE patients and their caregivers have sought an oral on-demand treatment option that would allow them to effectively and safely manage their attacks and take control of their disease. The KONFIDENT results demonstrate that sebetralstat has the potential to meet that need,” said Danny Cohn, MD, PhD, Department of Vascular Medicine, Amsterdam University Medical Centre, and principal investigator for the KONFIDENT phase 3 trial. “Sebetralstat provided rapid symptom relief with a safety pro different from placebo. It was also encouraging to see that efficacy was consistent regardless of attack severity, anatomic location or the use of long-term prophylaxis. These data reinforce that, if approved, sebetralstat would have the potential to transform the treatment landscape.” Phase 3 KONFIDENT-S Open-Label Extension Trial Interim Results (as of February 2, 2024) 112 participants enrolled (Europe 50.0%, US 27.7%, other 22.3%) 15.2% aged 12-<18 years, 81.3% aged 18-<65 years, and 2.7% aged ≥65 years 68.8% receiving on-demand treatment only; 31.3% receiving stable long-term prophylaxis 649 attacks treated with sebetralstat; median 5.0 attacks treated per participant (range, 1-39) Median time from attack onset to treatment was 9 minutes (IQR 1-69) Median time to beginning of symptom relief was 1.8 hours Median times to reduction in attack severity (6.57 hours [IQR 1.61->12]) and complete attack resolution (21.0 hours [IQR 7.22->24]) were consistent with results in the KONFIDENT Trial. KONFIDENT and KONFIDENT-S are the first and only clinical trials ever to include complete symptom relief as an endpoint; all other trials use near-complete relief, which is a much lower standard that does not capture the entire attack sequence in the same manner. Laryngeal attack subgroup (n=14), median time to beginning of symptom relief was 1.3 hours “Compared to KONFIDENT, which is a placebo-controlled crossover trial, KONFIDENT-S has many real-world elements which enabled patients to treat attacks even earlier as evidenced by a median time to treatment of 9 minutes. These results further validate the promise of an oral on-demand treatment. By acting quickly, patients may halt the progression of attacks before they become severe and thereby minimize the burden of their attacks and the time to improvement and resolution of their symptoms,” said Henriette Farkas, MD, PhD, Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary. “Importantly, we also learned that sebetralstat’s effect in laryngeal attacks was consistent with the results in all other attack locations.” “We want to thank the trial participants, their families, their advocates, and the investigator teams around the world who supported KONFIDENT and continue to support KONFIDENT-S. As reported at EAACI, as of May 7, 2024, KONFIDENT-S participants have treated over 1000 attacks with sebetralstat (including 24 laryngeal attacks). We look forward to submitting a new drug application for sebetralstat to the US FDA in June 2024 and in the EU and Japan later this year,” said Ben Palleiko, Chief Executive Officer of KalVista. The publication for NEJM can be found at the below link: Links to all posters and presentations can be found on the KalVista website under “Publications”. About the KONFIDENT Phase 3 Trial The KONFIDENT phase 3 trial was a randomized, double blind, event-driven, crossover clinical trial evaluating the efficacy and safety of sebetralstat 300 mg and 600 mg versus placebo for the on-demand treatment of HAE. The trial enrolled a total of 136 adult and adolescent HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE. In the trial, patients treated each eligible attack with up to two doses of study drug, and each patient could treat up to three attacks over the course of the study. The trial included type 1 and type 2 HAE patients who had at least two attacks in 90 days prior to enrollment. About the KONFIDENT-S Trial The KONFIDENT-S trial is an open label extension study evaluating the long-term safety of sebetralstat for on-demand treatment of HAE attacks in adolescents (aged 12-17) and adult patients with HAE Type I or Type II. The trial is also evaluating sebetralstat for use as a short-term prophylaxis treatment prior to medical procedures. About Sebetralstat Discovered by KalVista, sebetralstat is an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE). Sebetralstat received Fast Track and Orphan Drug designations from the U.S. FDA, as well as Orphan Drug Designation and an approved Pediatric Investigational Plan from the European Medicines Agency (EMA). About Hereditary Angioedema Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the location affected. All currently approved on-demand treatment options require either intravenous or subcutaneous administration. About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc. is a global pharmaceutical company focused on the development and delivery of oral medicines for diseases with significant unmet need. KalVista disclosed positive phase 3 data for the KONFIDENT trial for its oral, on-demand therapy sebetralstat in February 2024. The Company anticipates submitting a new drug application to the U.S. Food and Drug Administration (FDA) for sebetralstat in June 2024 and expects to approval in the UK, Europe, and Japan later in 2024. For more information about KalVista, please visit . Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA or other international regulatory agencies, our expectations about safety and efficacy of our product candidates, our ability to obtain regulatory approvals for sebetralstat and other candidates in development within our expected timelines or at all, our success in engaging with potential commercial partners, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, our ability to commence pediatric trials of sebetralstat and develop an ODT formulation, the future progress and potential success of our oral Factor XIIa program, our ability to reduce spending on discovery and preclinical activities, and our expectation to become cash flow positive. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2023, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. View source version on businesswire.com: Contacts KalVista Pharmaceuticals, Inc. Jarrod Aldom Vice President, Corporate Communications (201) 705-0254 jarrod.aldom@kalvista.com Ryan Baker Head, Investor Relations (617) 771-5001 ryan.baker@kalvista.com Source: KalVista Pharmaceuticals, Inc. View this news release online at: