Givinostat Hydrochloride

SYDNEY, June 27, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specialising in oncology, is pleased to announce the presentation of new data from its lead program, paxalisib, at the upcoming 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) June 29 – July 2, 2024, in Philadelphia, PA. Kazia concurrently announces publication of an article in European Journal of Cancer highlighting the need for evaluating mutation-specific, CNS penetrant, inhibitors to treat pediatric patients with Diffuse Midline Glioma (DMG). There will be three paxalisib-related presentations in total at ISPNO, including data from the Phase 2 PNOC DMG-ACT (DMG-Adaptive Combination Trial, PNOC022) study evaluating the efficacy and safety of paxalisib in combination with ONC201. As a follow up to data presented last year at the Society for NeuroOncology, 28th Annual Meeting, lead researchers will discuss survival, pharmacokinetics, and tumor biomarkers from 132 diffuse midline glioma (DMG) patients enrolled in the Phase 2 study. Highlights of the abstract include median overall survival of 13.2 months in Cohort 1 (newly diagnosed, enrolled pre-radiation n=33), 15.8 months in Cohort 2 (newly diagnosed, enrolled post-radiation n=69) and 8.8 months in Cohort 3 (relapsed patients, enrolled after progression n=30). The second presentation is based on novel preclinical data utilizing the addition of a novel HDAC inhibitor to the backbone therapy of paxalisib in DMG models. The third presentation will highlight preclinical data results of the combination therapy of paxalisib and gemcitabine for patients with relapsed/recurrent atypical teratoid/rhabdoid tumors AT/RT by Johns Hopkins University researchers. Based on these findings, the Pacific Pediatric Neuro-Oncology Consortium is planning to include this combination therapy in its next AT/RT international clinical trial. Summary of Abstracts ( ) Clinical Trials; July 2, 2024; 8:15am TRLS-14: PNOC022 report: a combination therapy trial using an adaptive platform design for patients with diffuse midline glioma at initial diagnosis, post-radiation therapy, or progression Cassie Kline, Andrea Franson, Anuradha Banerjee, Alyssa T Reddy, et al Poster Session I; June 30, 2024; 5pm ATRT-15 Combining the PI3K inhibitor paxalisib with nucleoside analog gemcitabine to improve survival of atypical teratoid/rhabdoid tumors Tyler Findlay, Kristen Malebranche, Anupa Geethadevi, Charles Eberhart, Jeffrey Rubens, Eric Raabe DIPG-21 Preclinical assessment of a multimodal treatment approach with Givinostat, Paxalisib, and radiotherapy for Diffuse Midline Glioma (DMG) Aimée du Chatinier, Michaël H Meel, Piotr Waranecki, Dennis S Metselaar, Esther The European Journal of Cancer publication titled Paediatric Strategy Forum for Medicinal Product Development of PI3-K, mTOR, AKT and GSK3β Inhibitors in Children and Adolescents with Cancer is the output from a two-day forum in April 2023 at Dana Farber Cancer Institute. Consisting of patient advocates, regulators, researchers and pediatric clinicians, the publication concludes "Evaluation of mutation-specific, CNS-penetrant PI3-K inhibitors in children with DMG should be prioritised and innovative regulatory approaches are needed in view of the rarity of the population." The paper can be accessed at the following website: (24)00801-3/fulltext About Kazia Therapeutics Limited Kazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase 2 study in glioblastoma reported early signals of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, has completed enrollment, with final data expected imminently. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported encouraging interim data. Paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumours in June 2022 and July 2022, respectively. For more information, please visit or follow us on Twitter @KaziaTx. Forward-Looking Statements This announcement may contain forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for results and data related to Kazia's clinical and preclinical trials and investigator-initiated trials of Kazia's product candidates, and Kazia's strategy and plans with respect to its programs, including paxalisib and EVT801. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the United States Securities and Exchange Commission (SEC), and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement. This announcement was authorized for release by Dr John Friend, CEO. Company Codes: NASDAQ-SMALL:KZIA

Milestone event brings together record-breaking 1,300 plus families, physicians, researchers, caregivers, industry partners, and those living with Duchenne and Becker in person and virtually to connect, share information, and learn the latest progress in the fight for every future. ORLANDO, Fla., June 24, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its 30th Annual Conference in Orlando, Florida, June 27–29. Each year, the Duchenne and Becker community gathers to learn about the latest progress in the fight to end Duchenne and connect with leading experts. Now in its 30th year, PPMD's Annual Conference returns to Orlando, the location of the organization's very first Annual Conference in 1994, to engage more of the Duchenne and Becker community than ever before. PPMD expects a record-breaking 1,300 plus families, researchers, clinicians, and industry partners from all over the world for three days of informative and interactive sessions featuring the latest news in research, clinical trials, approved and emerging therapies, care initiatives, quality of life issues, and more. Before the Conference officially kicks off, PPMD will host a Becker Care Professional Meeting, June 26–27, aimed at initiating discussions within the healthcare professional community regarding the creation of consensus-based standards of care for Becker muscular dystrophy. Founded in 1994 by a group of parents and grandparents, and led by Founding President and CEO Pat Furlong, PPMD is the largest, most comprehensive nonprofit organization in the United States focused on ending Duchenne. Since its first meeting 30 years ago, PPMD has convened families with researchers, clinicians, industry partners, advocacy organizations, and most importantly, people with Duchenne and Becker and their families at the largest Duchenne-specific conference held in the U.S. Furlong is excited about this year's milestone Conference and looks forward to bringing the Duchenne and Becker community together: "From PPMD's first Annual Conference 30 years ago to our momentous return to Orlando this week, this community's unwavering commitment, passion, and resilience have been the driving force behind our fight for every future. We have faced challenges and celebrated victories and we have motivation to keep progressing. PPMD's 30th Annual Conference will be filled with a variety of interactive experiences to spark conversation, foster new and familiar relationships, learn from one another, and continue to drive change in the fight to end Duchenne." PPMD is grateful to all of our industry sponsors, whose support helps make the Annual Conference possible, especially our 30th Anniversary Star sponsor Sarepta Therapeutics, our Platinum sponsors Catalyst Pharmaceuticals and Pfizer, Inc., and our Gold sponsors Avidity Biosciences, Dyne Therapeutics, Edgewise Therapeutics, ITF Therapeutics, NS Pharma, Inc., Upsher-Smith Laboratories, and Wave Life Sciences. For sponsorship opportunities, please click here. PPMD's 30th Annual Conference agenda will feature stories from multiple keynote speakers, and interactive discussion panels addressing comprehensive, multi-dimensional approaches to care for Duchenne and Becker. Unique topics to this year's conference include: Approved therapies in Duchenne, including presentations on the recently expanded label for ELEVIDYS and newly-approved therapies AGAMREE and DUVYZAT, as well as a clinician panel discussion on the eight currently approved therapies. Becker-specific breakout sessions, including discussions on neuromuscular care, cardiology, genetic counseling, and physical therapy, as well as industry updates about emerging therapies for Becker. What we have learned over time about outcome measures, including Functional Reach, Stride Velocity, Dynamic Visual Acuity (DVA), and Northstar and Performance of Upper Limb (PUL). Cardiac, neuromuscular, and adolescent care for carriers, as well as physical therapy implications and research in progress in the field of Duchenne carriers. Unique experiences and perspectives on life, living, and hope, as well as daily life care such as sleep and behavior; decision making around steroids, weight, and nutrition; pain; exercise; social connection; and more. This year's Conference will also feature dedicated updates from more than 20 partners currently in clinical trials, with panels including: Strategies to restore dystrophin The current and evolving landscape of gene therapy Approaches to alter disease progression PPMD's role in moving the needle in clinical research A variety of breakout sessions and social gatherings will also be offered during PPMD's 30th Annual Conference, encouraging attendees to create their own, unique experiences, in-person or virtually, by participating in: Special programming for Newly Diagnosed Families A dedicated track for Teens and Adults with Duchenne/Becker Expanded Sibs Track sessions, offering a safe place for siblings to be together, talk, and open up about topics that they face Breakout sessions according to age, covering multiple aspects of care One-on-one private appointments with Physical Therapists, Genetic Counselors, and behavior, speech, and language experts to help provide guidance on every family's unique needs Small group Lighthouse Mini-Sessions focused on self-care, effective communication, asking for help, and staying connected and finding belonging A Build Your Own Care Binder station for families to create personalized binders filled with important information regarding Duchenne/Becker care to help navigate routine care and emergencies A robust Resource Fair enabling resource providers and the community to connect about practical services, research and more A meet and greet for diverse families in Duchenne to share their unique experiences and foster connection Social activities for moms, dads, grandparents, siblings, and, of course, tweens, teens, and adults with Duchenne and Becker Fun and interactive sessions for kids Ahead of the Conference, all registered attendees were given access to PPMD's online Conference Hub & App to stream sessions and personalize their agenda and Conference experience. This year's Hub & App offers an enhanced experience for both in-person and virtual attendees, allowing for engagement with exhibitors and increased interaction during live sessions. AI-powered live translation will also be available for main sessions in dozens of languages. It is not too late to attend this year's meeting, in-person or virtually. To register for PPMD's 30th Annual Conference, click here. To learn more about PPMD, please visit our website. SOURCE Parent Project Muscular Dystrophy (PPMD)