Ophthalmoplegia

NEW YORK, March 14, 2024 /PRNewswire/ -- CurePSP has awarded its latest Pathway and Pipeline Grants, totaling approximately $400,000. Recipients of the grants are: Dr. Kathryn Bowles, group leader at University of Edinburgh; Dr. Maura Malpetti, research fellow at University of Cambridge; Dr. Jessica Rexach, assistant professor at UCLA; and Dr. Yang You, assistant professor at Mayo Clinic Jacksonville. The studies will make progress in understanding the basic neurobiology of progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) and in developing biomarkers to improve diagnosis and treatment. The latest Pathway grants will fund two projects: "Investigating Mitochondrial Dysfunction as an Early Pathogenic Mechanism of Primary Tauopathies, Using MAPT Splicing Mutation iPSC-organoids" by Dr. Kathryn Bowles, and "Defining the Unique Immunogenetic Landscape of PSP Compared to Related Dementias" by Dr. Jessica Rexach. The newest Pipeline grants will fund two projects: "Blood-based Fingerprints of Neuroinflammation in People with PSP and CBD" by Dr. Maura Malpetti, and "Characterization of Plasma-derived Extracellular Vesicle Proteomics for Biomarker Development of PSP and CBD" by Dr. Yang You. "Together, these grants reflect CurePSP's commitment to funding junior investigators working in high-priority research areas that will help advance our understanding and treatment of PSP and CBD," said Dr. Jennifer Brummet, associate director of scientific affairs and partnerships at CurePSP. "There is a critical need to improve our understanding of PSP and CBD and to improve diagnosis, and these grants will help move the field forward." About CurePSP's Research Grants Pathway and Pipeline Grants provide seed funding to support innovative projects focused on the fundamental neurobiology and mechanisms of tauopathies ("Pathway"), and translational projects focusing on new therapeutics and biomarkers for tauopathies ("Pipeline"). The next deadline for Pathway and Pipeline Grant applications is March 28, 2024. Learn more about our research grants here. About CurePSP CurePSP is the leading nonprofit organization dedicated to the awareness, care and cure for three neurodegenerative diseases: progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and multiple system atrophy (MSA). As a catalyst for new treatments and a cure, CurePSP establishes important partnerships and funds critical research internationally. Through its advocacy and support efforts, CurePSP enhances education, care delivery and quality of life for people living with PSP, CBD and MSA and their families. Science, community and hope are at the heart of CurePSP's mission and all its services. CurePSP is a registered 501(c)(3) charity within the United States (EIN: 52-1704978). For more information, please visit . Contact: Kristophe Diaz, PhD Executive Director and Chief Science Officer 646-725-1453 [email protected] SOURCE CurePSP, Inc.

- Dr. Zeiher brings more than 20 years of drug development experience in areas with serious unmet treatment needs at companies including Astellas Pharma, Pfizer, Eli Lilly and Company, and Merck CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the addition of Bernhardt G. Zeiher, MD, FCCP, FACP, to its Board of Directors (“Board”). Dr. Zeiher brings more than 20 years of drug development experience where, in his various roles, he oversaw the approval of 15 new treatments that addressed people’s unmet needs in serious diseases with few to no treatment options. Most recently, he served as Chief Medical Officer (“CMO”) of Astellas Pharma, where he led early- and late-stage drug development, medical and regulatory affairs, pharmacovigilance, and quality assurance. “This is a pivotal time for Amylyx, and we are thrilled to welcome Dr. Zeiher to our Board to share his perspectives and expertise as the company continues to build a pipeline supporting our collective mission to one day end the suffering of neurodegenerative diseases,” said George M. Milne, Jr., PhD, Chairman of Amylyx’ Board. “Dr. Zeiher's insights of the intricacies and complexities of drug development and innovation in diseases with clear unmet need will be valuable as we continue to serve neurodegenerative disease communities impacted by these devastating diseases.” Dr. Zeiher is a recognized industry leader who spent more than 10 years at Astellas Pharma, holding multiple roles of increasing responsibility in the drug development division, leading up to his role as CMO from 2018 to 2022. Prior to his time at Astellas, Dr. Zeiher held various roles leading drug development at other pharmaceutical companies including Pfizer, Eli Lilly and Company, and Merck. Dr. Zeiher also practiced medicine at a tertiary medical center in Indianapolis and has held a range of board appointments since 2014, including positions at PhRMA Biomedical Advisory Committee, TransCelerate Biopharma, Biotechnology Innovation Organization, Astellas Global Health Foundation, and most recently, Entrada Therapeutics, which he joined in 2023. “We are seeing a transformation in neurodegenerative diseases, both in potential new treatments and what we’re learning through research, and I believe Amylyx is a catalyst in that change,” said Dr. Zeiher. “The company’s community-centric values, steadfast commitment to following the science, and beyond-therapy approaches and support for the neurodegenerative disease community are a few examples of why I am excited for the future and what we can accomplish to support those living with these devastating diseases and their loved ones.” “Dr. Zeiher brings to the Board his distinguished drug development background in overseeing the approval of 15 medicines, and his first-hand experience as a physician. On behalf of the entire Amylyx team, we are delighted to welcome him onboard as we continue to advance our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “We have several important milestones that we’re working toward this year where Dr. Zeiher’s expertise will be key, including our goal to bring our antisense oligonucleotide AMX0114, developed in-house, to the clinic for ALS, continuing to research the potential of AMX0035 in diseases like Wolfram syndrome and progressive supranuclear palsy, and sharing additional insights, learnings, and next steps from our Phase 3 PHOENIX trial in ALS.” Dr. Zeiher received a B.S. in biology from the University of Toledo and an MD from Case Western Reserve University School of Medicine. He completed his internal medicine residency and chief residency at University Hospitals of Cleveland and then finished his physician training as a Pulmonary and Critical Care Fellow at University of Iowa Hospitals and Clinics. About Amylyx Pharmaceuticals Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative disease community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada, EMEA, and Japan. For more information, visit amylyx.com and follow us on LinkedIn and X, formerly known as Twitter. For investors, please visit investors.amylyx.com. Forward-Looking Statements Statements contained in this press release and related comments in our earnings conference call regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, the Company’s expectations of contributions from its board of directors; the Company’s beliefs regarding the benefits of AMX0035 in neurodegenerative diseases; expectations regarding the continued research of AMX0035 in progressive supranuclear palsy and Wolfram syndrome; the Company’s potential announcement of additional insights, learnings, and next steps from the Phase 3 PHOENIX trial; the Company’s expectations with respect to its progress through IND-enabling studies of AMX0114; the Company’s expectations with respect to its early-stage ALS diagnostic test; and expectations regarding the Company’s longer-term strategy. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include the risks and uncertainties set forth in Amylyx’ filings with the Securities and Exchange Commission (“SEC”), including its Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Subject to any obligations under applicable law, Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

LILLE, France--(BUSINESS WIRE)-- ALZPROTECT is thrilled to announce that it has received favorable feedback from both the American (FDA) and European (EMA) authorities regarding the regulatory path for advancing the clinical development of Ezeprogind/AZP2006 for patients with PSP. This significant milestone follows the already very promising results of the phase 2a study in PSP and marks a decisive advancement. It will provide a clear path for the further development of Ezeprogind/AZP2006 within both scientific and regulatory expectations. This press release features multimedia. View the full release here: Regulatory authorities have provided crucial insights into various design elements, with a particular focus on selecting study endpoints pertinent to the PSP indication and ensuring that the demographic and characteristic pro the study sample closely mirrors the global PSP population. This feedback has armed Alzprotect with a valuable roadmap, enabling the refinement of the intended Phase 2/3 study protocol to ensure seamless alignment with scientific standards and regulatory requirements. With well-defined regulatory pathways for the PSP indication, Alzprotect's forthcoming Proof of concept randomized controlled PROMISE-PSP trial, scheduled to commence in late 2024, is set to deliver outcomes that align with scientific and regulatory expectations across the United States and the European Union. Dr. Artin Karapet, our Chief Medical Officer, expressed his heartfelt joy stating, "I am truly delighted to witness this alignment between agencies from different shores on critical program elements. Their guidance not only paves a feasible path forward but also brings AZP2006 one step closer to our patient community." Phil Verwaerde, Chief Executive Officer, added, "The alignment with regulatory authorities marks a pivotal moment for ALZPROTECT. It validates our vision and the potential of Ezeprogind/AZP2006 not only for PSP but also for a broader neurodegenerative disease spectrum. We are dedicated to accelerating our efforts to meet the urgent needs of patients." The molecule, AZP2006, proposes a fundamentally distinct mechanism of action compared to existing treatments: Targeting the root causes of neurodegeneration by stimulating the action of a protein essential to the functioning of the brain, Progranulin. AZP2006 has shown excellent safety and promising efficacy in human trials, including 102 healthy subjects across three Phase I trials and 36 PSP patients in a Phase 2a trial. Alzprotect plans to initiate a randomized Phase 2b/3 proof of concept for PSP and expand its drug development to include Parkinson's disease, Alzheimer's disease, and amyotrophic lateral sclerosis in the coming years. View source version on businesswire.com: Contacts p.verwaerde@alzprotect.com Source: Alzprotect Smart Multimedia Gallery Video Alzprotect's corporate video Logo View this news release online at: