Walker-Warburg Syndrome

GAL-201 specifically targets and binds to misfolded Aβ monomers, preventing their aggregation to toxic Aβ oligomers and protofibrilsAs a consequence, consistent beneficial effects have been demonstrated on synaptic spine density and on cognitive performance after single injection of GAL-201 in transgenic models of Alzheimer’s diseaseThese results warrant initiation of an IND-enabling program as next step towards clinical development KENSINGTON, Md., March 08, 2024 (GLOBE NEWSWIRE) -- Galimedix Therapeutics, Inc. (“Galimedix”), a Phase 2 clinical-stage biotechnology company developing novel oral and topical neuroprotective therapies with the potential to revolutionize the treatment of serious eye and brain diseases, announces encouraging data with orally available small molecule GAL-201, showing its neuroprotective and symptomatic alleviation potential in models of Alzheimer’s disease (AD). These new findings strongly support previous studies where GAL-201 was characterized as a promising development candidate for the treatment of AD (Russ et. al. 2022). The data was presented this week at the International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD™ 2024: advances in science and therapy). “The small molecule GAL-201 belongs to a new pharmacological class of amyloid beta aggregation modulators that acts upstream of other known Aβ-targeting agents,” explained Hermann Russ, M.D., Ph.D., Co-founder, and Chief Scientific Officer of Galimedix. “The positive results presented at AD/PD™ 2024 demonstrate how treatment with GAL-201 has a potential neuroprotective effect and may also improve cognitive function. Furthermore, they validate that toxic Aβ oligomers and protofibrils are a major underlying cause of this devastating disease, and not the deposited forms of Aβ. We look forward to advancing this promising new drug candidate into the clinic.” Data from the poster, entitled “The amyloid beta aggregation modulator GAL-201 is under development for oral AD treatment: Cognitive improvement in a transgenic AD model”, showed a high affinity of GAL-201 to misfolded Aβ monomers, the precursors of the neurotoxic forms of Aβ oligomers and protofibrils. Binding of GAL-201 to the Aβ monomers significantly prevented Aβ-induced suppression of long-term potentiation (LTP), considered a neuronal correlate of learning and memory. These results mirrored morphological changes observed in neuronal cells (dendrites), where Aβ-induced spine loss was also prevented. Preserving dendritic spines is essential, as they are correlated with synaptic plasticity. Additionally, in behavioral experiments using AD animal models, a single injection of GAL-201 resulted in improved cognitive performance, both in duration (escape latency) and accuracy (left and right decisions) performing a task (water-cross maze) and were comparable to healthy mice. The presented results further substantiate GAL-201’s role as a promising future Aβ-targeting treatment for AD, with an advanced mechanism of action and a patient-friendly application route as an oral tablet. The next step is to move GAL-201 towards IND submission and clinical development. The poster can be accessed here: “The amyloid beta aggregation modulator GAL-201 is under development for oral AD treatment: Cognitive improvement in a transgenic AD model” About GAL-201GAL-201 is an orally administered small molecule specifically designed to target amyloid beta (Aβ) and suppress the formation of toxic forms of Aβ aggregates in the brain of people with AD. GAL-201 has been shown to be able to bind with high affinity to the misfolded forms of Aβ, and thereby prevent their aggregation to toxic Aβ oligomers and protofibrils. GAL-201 is believed to be a promising development candidate for the treatment of AD with the potential to slow or stop disease progression and is currently in pre-clinical testing. Galimedix holds global rights to GAL-201. About Galimedix Therapeutics, Inc. Galimedix is a Phase 2 clinical-stage private company developing novel oral and topical neuroprotective therapies with the potential to revolutionize the treatment of serious eye and brain diseases. Founded by a seasoned and highly dedicated team of bio-entrepreneurs, pharmaceutical executives, and scientists, Galimedix’s groundbreaking small molecules offer the hope of changing the course of diseases where amyloid beta (Aβ) plays a role, such as in dry age-related macular degeneration (AMD), glaucoma and Alzheimer’s disease - Galimedix’s initial areas of focus. The Company’s approach targets toxic Aβ oligomers and protofibrils. Many studies have indicated that these oligomers and protofibrils are an underlying cause of neurodegenerative diseases of the eye. Recent approvals and promising Phase 3 results of anti-Aβ drugs also have validated them as a key target in Alzheimer’s disease. Compelling pre-clinical data support the potential of Galimedix’s product candidates to slow or stop neurodegeneration and restore lost neuronal function. A Phase 2 proof-of-concept study in dry AMD with lead program, topical GAL-101, is in preparation with strong support from partner, Théa Open Innovation (TOI). Clinical studies in other indications are planned. Contact Alexander Gebauer, MD, PhD Galimedix Therapeutics, Inc. Co-founder and Executive Chairman info@galimedix.com Media inquiries: Anne Hennecke / Lydia Robinson-GarciaU.S.MC Services AGLaurie DoyleTel: +49 (0)170 7134018Tel: +1-339-832-0752galimedix@mc-services.eu

Bristol Myers Squibb has agreed to spend at least $100 million to expand a partnership aimed at creating RNA-based medicines for cardiovascular diseases.The deal, announced Tuesday, shows Bristol Myers is willing to bet more on drugmaking technology developed by Avidity Biosciences. Founded in 2012, Avidity is trying to combine elements from two kinds of medicines monoclonal antibodies and oligonucleotide therapies to make new treatments that are both precise and target the root cause of diseases.The most advanced of Aviditys so-called AOCs, or antibody oligonucleotide conjugates, are in early-stage human trials, where researchers are evaluating their ability to treat three kinds of rare, muscle-eroding diseases. The company has other experimental therapies in preclinical testing, and is partnered with Eli Lilly on AOCs for immune system disorders and other select indications.Those programs are separate from the Bristol Myers collaboration, which will now include the discovery, development and commercialization of AOCs for as many as five cardiovascular targets. Deal terms hold that Avidity will initially receive $60 million in cash and an approximately $40 million equity investment. Bristol Myers has also agreed to pay as much as $2.2 billion more, provided Avidity's programs hit certain milestones.The pharmaceutical giant said it will fund all future clinical development, regulatory and commercialization activities tied to the deal. Avidity, meanwhile, could earn royalties up to low double-digits on net sales from any resulting products.This strategic collaboration solidifies our commitment in cardiology as we continue to advance our own research and development programs in cardiac indications,“ said Sarah Boyce, Aviditys CEO, in a statement.The two companies originally linked up in late 2020, when Avidity entered a research partnership with the Bristol Myers subsidiary MyoKardia to demonstrate that AOCs can be useful in cardiac tissue. That deal came just after Bristol Myers completed its $13 billion acquisition of MyoKardia, which was advancing medicines for genetic forms of heart disease. One of those medicines has since received regulatory approval, and is sold under the brand name Camzyos.To Steven Seedhouse, an analyst at the investment firm Raymond James, the new collaboration is positive for Avidity in several ways. First, it provides a meaningful extension to the companys already very strong cash runway, which was at $542 million by the end of September.Second, its a vote of confidence in Aviditys platform from a large, established drugmaker. Bristol Myers must have liked what it saw preclinically over the last two or so years from the existing MyoKardia collaboration, Seedhouse wrote in a note to clients.Avidity shares rose more than 11% Tuesday morning, to trade at $6.87 apiece. The companys stock was valued at more than $25 per share in February, but has ticked down in the months since.In September 2022, the Food and Drug Administration placed a partial clinical hold on a trial testing one of Aviditys muscular dystrophy medicines, due to a rare serious adverse event. Following discussions between the agency and the company, the hold was eased in May.In his note, Seedhouse wrote that his team believes the newly expanded collaboration implies a high likelihood (or at least Bristols lack of concern about) the partial clinical hold ... is lifted soon. '

Brings over 20 years of clinical experience, including management of Phase 1-2 clinical trials in a variety of indications such as ophthalmology Will oversee all R&D operations with focus on clinical development and operations KENSINGTON, MD / ACCESSWIRE / October 25, 2023 / Galimedix Therapeutics, Inc. ("Galimedix"), a Phase 2 clinical-stage biotechnology company developing novel oral and topical neuroprotective therapies with the potential to revolutionize the treatment of serious eye and brain diseases, today announced the appointment of Luciana Summo, PhD, as Vice President, R&D Operations. In this newly created position, she will oversee all activities related to R&D operations, with an emphasis on clinical development and operations. This includes spearheading the design, planning, and execution of clinical trials. Dr. Summo reports to Hermann Russ, MD, PhD, Co-founder and Chief Scientific Officer. "We are delighted to welcome Luciana to Galimedix," said Alexander Gebauer, MD, PhD, Co-founder and Executive Chairman . "She brings strong experience setting up and running clinical trials and ensuring that all of the proper support structures are put in place. As we move our lead product candidate, GAL-101, into Phase 2 testing for the treatment of dry age-related macular degeneration, her skill set is the perfect fit for this next stage in our development. Luciana will play a pivotal role in defining Galimedix's overall R&D strategy and driving our R&D initiatives forward. All of us very much look forward to working with her as we contribute to advancing innovative healthcare solutions that have the potential to improve the lives of patients." Dr. Summo has over 20 years of experience in clinical development and operations, including the management of Phase 1-2 clinical trials in several indications such as ophthalmology, cardiology, and oncology. Prior to joining Galimedix, she worked in positions of increasing responsibility in the clinical departments at OMEICOS Therapeutics GmbH, NOXXON Pharma AG, and Berlin-Chemie AG/ Menarini Group. She is well versed in setting up and overseeing clinical trials on a global scale and has played a key role in transitioning programs from pre-clinical to clinical stage. She has experience with the entire spectrum of activities related to R&D operations, with a special emphasis on clinical development and clinical operations, including also quality management and CMC. She works closely with cross-functional teams, including CROs, scientists, clinicians, regulatory affairs specialists, and different stakeholders, to ensure that trials are conducted in accordance with rigorous scientific standards and regulatory guidelines. Dr. Summo has experience in submitting Clinical Trial Applications (CTAs) in Europe and Investigational New Drug (IND) applications in the US and prepared for and participated in pre-IND meetings with the US Food and Drug Administration (FDA), as well as regulatory agency meetings in Europe. She also has set up from scratch and run a clinical operations department. Luciana Summo, PhD, Vice President, R&D Operations , added: "I am truly excited to be joining Galimedix at this important point in the Company's growth. The programs in ophthalmology and Alzheimer's disease have the potential to change the treatment paradigm, and I look forward to working with the rest of the team to bring them forward in the clinic." About Galimedix Therapeutics, Inc. Galimedix is a Phase 2 clinical-stage private company developing novel oral and topical neuroprotective therapies with the potential to revolutionize the treatment of serious eye and brain diseases. Founded by a seasoned and highly dedicated team of bio-entrepreneurs, pharmaceutical executives and scientists, Galimedix's groundbreaking small molecules offer the hope of changing the course of disease where amyloid beta (Aβ) plays a role, such as in dry age-related macular degeneration (AMD), glaucoma and Alzheimer's disease - Galimedix's initial areas of focus. The Company's approach targets toxic Aβ oligomers and protofibrils. Many studies have indicated that these oligomers and protofibrils are an underlying cause of neurodegenerative diseases of the eye. And, recent approvals and promising Phase 3 results of anti-Aβ drugs also have validated them as a key target in Alzheimer's disease. Compelling pre-clinical data support the potential of Galimedix's product candidates to slow or stop neurodegeneration and also restore lost neuronal function. A Phase 2 proof-of-concept study in dry AMD with lead program, topical GAL-101, is in preparation with strong support from partner, Théa Open Innovation (TOI). Clinical studies in other indications are planned. Contact Alexander Gebauer, MD, PhD Galimedix Therapeutics, Inc. Co-founder and Executive Chairman info@galimedix.com Media inquiries: Anne Hennecke / Lydia Robinson-Garcia U.S. MC Services AG Laurie Doyle Tel: +49 (0)170 7134018 Tel: +1-339-832-0752 galimedix@mc-services.eu SOURCE: Galimedix Therapeutics, Inc. View source version on accesswire.com: