Latest news with #LUMINA
Yahoo
6 days ago
- Business
- Yahoo
Amylyx Pharmaceuticals Receives U.S. FDA Fast Track Designation for AMX0114 for the Treatment of Amyotrophic Lateral Sclerosis
- AMX0114 is an Amylyx-developed antisense oligonucleotide designed to target calpain-2, a key contributor to the axonal degeneration pathway in ALS - Phase 1 LUMINA trial of AMX0114 underway; early cohort data expected in 2025 CAMBRIDGE, Mass., June 03, 2025--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) ("Amylyx" or the "Company") today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the treatment of people living with amyotrophic lateral sclerosis (ALS). "Obtaining FDA Fast Track designation for AMX0114 is an important step forward in our mission to develop potential treatments for people living with ALS, a relentlessly progressive and fatal disease with limited therapeutic options," said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. "This designation from the FDA recognizes both the seriousness of this devastating disorder and the encouraging preclinical evidence supporting AMX0114's potential to target calpain-2, which has been found to be an important contributor to axonal degeneration, a critical driver in ALS progression. We are committed to advancing AMX0114 as quickly and efficiently as possible, and we continue to anticipate early cohort data from the Phase 1 LUMINA clinical trial later this year. We look forward to continued interaction with the FDA as we work to expeditiously advance the development of AMX0114, with the ultimate goal of addressing the urgent, unmet needs of the ALS community." The FDA's Fast Track designation is designed to facilitate the development and expedited review of therapies that are intended to treat serious and life-threatening conditions and demonstrate the potential to address an unmet medical need. A therapy that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA, as well as Priority Review if relevant criteria continue to be met. Amylyx designed AMX0114 to target calpain-2, a calcium-activated protease. Peer-reviewed research has demonstrated that overactive calpain-2 activity may be an important driver of disease progression in ALS and other neurodegenerative diseases by executing the degeneration of axons, the long tubular neuronal segments which carry signals from neurons to the muscle or other neurons. In preclinical studies, AMX0114 showed improved neuronal survival and reductions in extracellular neurofilament light (NfL) levels across multiple disease models. In April 2025, the Company announced the first participant was dosed in LUMINA, a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose Phase 1 clinical trial designed to evaluate the safety and biological activity of AMX0114 in people living with ALS. LUMINA will also assess broadly researched ALS biomarkers, including changes from baseline in NfL levels. Approximately 48 participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks, for up to four doses. Amylyx expects early cohort data from LUMINA in 2025. About AMX0114 AMX0114 is an investigational antisense oligonucleotide (ASO) targeting calpain-2 (CAPN2) for the potential treatment of ALS. The U.S. Food and Drug Administration (FDA) has granted AMX0114 Fast Track designation for the treatment of ALS. In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular neurofilament light (NfL) levels across multiple disease models and paradigms of neuronal injury. AMX0114 was well-tolerated in in vivo preclinical safety studies. About ALS Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease) is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history. About LUMINA The Phase 1 LUMINA clinical trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS. LUMINA is anticipated to enroll approximately 48 adult participants. LUMINA will also assess broadly researched ALS biomarkers, including change from baseline in neurofilament light (NfL) levels. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit and follow us on LinkedIn and X. For investors, please visit Forward-Looking Statements Statements contained in this press release 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, Amylyx' expectations regarding: the potential for AMX0114 as a treatment for ALS, the expected timeline for data readout, and the expectation for regulatory action. Any forward-looking statements in this press release and related comments in the Company's earnings conference call 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 success, cost, and timing of Amylyx' program development activities; Amylyx' ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; Amylyx' ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx' operations, as well as the risks and uncertainties set forth in Amylyx' United States Securities and Exchange Commission (SEC) filings, including Amylyx' Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. 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, except as required by law. View source version on Contacts Media Amylyx Media Team(857) 320-6191amylyxmediateam@ Investors Lindsey Allen(857) 320-6244Investors@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Globe and Mail
26-02-2025
- Business
- Globe and Mail
Merger Agreement with BioSymetrics to Accelerate Biomarker Identification, Enhance Drug Discovery and Expand Precision Medicine Applications in Advanced Cancer Diagnosis and Treatment
$RENB Also Secures Plus $15 Million in New Equity to Support its Operations Towards Revolutionizing Healthcare • Developing AI and Biotechnology Platforms for Early Diagnosis, Better-Targeted Treatments and Drug Discovery. • Pioneer Leader in Cancer Diagnostics and Therapeutics Powered by Artificial Intelligence (AI). • Definitive Merger Agreement with BioSymetrics to Advance AI-Driven Biomarker Discovery and Precision Medicine in Cancer and Beyond. • $15 Million in New Equity Committed to accelerate Focus on Revolutionizing Healthcare via the Company's Established Strategies. • 2025 Shareholder Update Issued from CEO. • Plans to Attend American Association for Cancer Research (AACR), The First and Largest Cancer Research Organization, Annual Meeting in April, 2025. • Funding Approval of LUMINA Minimal Residual Disease Detection Platform for Lung Cancer Harnessing Multi-omics Biomarkers and AI. • Entered Strategic Collaboration with Nebul to Advance a Paradigm Shift for Early Disease Detection. • Plans to Establish the Renovaro GEDi CUBE Next Generation Nvidia Blackwell Cluster with Nebul's Certified AI Healthcare Cloud Solutions. Renovaro, Inc. (Nasdaq: RENB) aims to accelerate precision and personalized medicine for longevity powered by mutually reinforcing AI and biotechnology platforms for early diagnosis, better-targeted treatments, and drug discovery. Renovaro Inc. includes RenovaroBio with its advanced cell-gene immunotherapy company and RenovaroCube that is leveraging AI for multi-omic diagnostics and drug development. RENB RenovaroCube has developed an award-winning AI platform that is committed to the early detection of cancer and its recurrence and monitoring subsequent treatments. RenovaroCube intervenes at a stage where potential therapy can be most effective. RENB RenovaroCube is a molecular data science company with a background in FinTech and a 12-year history. It brings together proprietary artificial intelligence (AI) technology, multi-omics, multi-modal data, and the expertise of a carefully selected multidisciplinary team to radically accelerate precision medicine and enable breakthrough changes in cancer care. Renovaro and BioSymetrics Announce Definitive Merger Agreement to Advance AI-Driven Biomarker Discovery and Precision Medicine On February 26th RENB announced a definitive agreement to merge with BioSymetrics, an artificial intelligence (AI)-driven drug discovery and biomarker identification company. This transformative partnership is designed to enhance Renovaro's data repository, biomarker discovery capabilities, accelerate translational research, and bring precision medicine solutions to cancer and other critical disease areas. At the core of this collaboration is BioSymetrics' proprietary Elion platform, a cutting-edge AI and machine learning engine that uncovers complex biological relationships to accelerate the discovery of diagnostics and therapeutics. BioSymetrics' Phenograph provides a translational engine that maps human clinical signals to prioritized therapeutic targets and is designed to expedite and improve target and biomarker identification and enable patient stratification and drug repurposing. BioSymetrics' advanced AI in vivo modeling and machine vision systems enable high throughput phenotypic screening, leveraging AI-powered analysis to detect subtle biological responses with unprecedented accuracy. Through in vivo modeling BioSymetrics has amassed an incredible database of proprietary in vivo experimentation, with associated behavioral and morphological analysis. This integrated approach hastens the discovery and validation of transformative therapeutics by bridging computational insights with real-world biological validation, and has enabled platform and analytic partnerships with Janssen, Pfizer, Merck, Supernus Pharma, and Deerfield Cures. By integrating Elion into the RENB workflow, the combined entity aims to streamline the translation of biomarker insights into accelerated discovery timelines, enhancing precision in target identification and improving overall research efficiency, ultimately enabling faster and more effective drug discovery and therapeutic development. BioSymetrics' AI technology has been instrumental in uncovering novel disease signatures and optimizing precision medicine strategies. Through this merger, RENB will gain access to cutting-edge computational tools that enhance the ability to stratify patients, predict treatment responses, and drive more effective therapeutic interventions. For more information on BioSymetrics visit: $15 Million in New Equity Committed RENB has also announced that it has $15 million in additional equity committed at a price per share of $1.00 and one privately placed cash warrant exercisable at $1.50 with a one year term. David Weinstein, Chief Executive Officer of Renovaro, commented, 'We believe this new capital now enables the company to accelerate our focus on revolutionizing healthcare by combining advanced diagnostic and personalized immunotherapy for early diagnosis, better-targeted treatments, and drug discovery.' Shareholder Letter and Corporate Update On January 7th RENB issued a letter to shareholders from Chief Executive Officer David Weinstein. The letter contained the following commentary: RENB recently signed a binding letter of intent to acquire Predictive Oncology (NASDAQ: POAI) that will provide with a tremendous company-owned database consisting of 150,000 frozen / live tumor samples, 20 years of drug response data, 40,000 FFPE tissue blocks, 200,000 pathology slides and digital library, and a fully certified CLIA laboratory. RENB plans to commercialize Predictive's clinical support test that assists oncologists in selecting the best therapy for their patients, and deploy their multi-omic AI system for drug discovery known as PEDAL (Patient-centric Discovery by Active Learning), a platform that combines AI technology, drug response data, and a large biobank of tumor samples to predict how different tumors will respond to various drugs. When RENB acquired GEDiCube, the rationale was that these two business segments were synergistic. This remains a core value proposition but for synergies to materialize requires structured collaboration between the two verticals: AI cancer detection and genetically enhanced cancer vaccines. There are two business developments that RENB is pursuing. Identifying a whole genome sequencer that can serve as the front end of RENB point of care cancer detection platform. GEDiCube to deploy its AI/ML analysis to discover more genes and factors in RENB Dendritic Cancer Cell Vaccine (DCCV). Incorporating these new discoveries into RENB patent filings will build shareholder value by ringfencing the DCCV platform and providing the ability to partner or license specific cancer applications. This has the potential to enhance the core three elements RENB infuses into DCCV now, allowing it to become specific for cancer types, and ultimately be available as an off the shelf solution. In the future this could facilitate early cancer detection in a doctor's office followed by the treatment of a patient with our vaccine. To gain exposure for DCCV RENB will attend the American Association for Cancer Research Annual Meeting in April 2025. For more information on $RENB visit: and Disclosure listed on the CorporateAds website Media Contact Company Name: Renovaro Inc. Contact Person: David Weinstein, CEO Email: Send Email Phone: 732-780-5036 Address: 2080 Century Park East Suite 906 City: Los Angeles State: California Country: United States Website:
