IGFBP2

Biologic treatment that restores muscle fiber formation leads Juvena’s pipeline of assets to treat myopathies, metabolic diseases and obesity REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Juvena Therapeutics, a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to the company’s flagship candidate, JUV-161. JUV-161 is an investigational therapeutic for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare, multi-systemic, autosomal dominant inherited disease and the most common form of adult muscular dystrophy. “People living with DM1 deserve safe, effective, and rejuvenating treatments that can repair and restore tissue health to improve muscle function and metabolism,” said Dr. Hanadie Yousef, Co-Founder and CEO of Juvena Therapeutics. “As we advance Juvena’s first biologic initially focused on the treatment of DM1, we look forward to exploring potential treatments for an entire class of disorders that remain major unmet medical needs.” JUV-161, which was identified by Juvena’s AI-driven drug discovery and development platform, has demonstrated an ability to restore muscle fiber formation, counter muscle atrophy, enhance muscle strength and endurance, and improve metabolism in multiple animal models. Human trials are anticipated to commence this year. JUV-161 includes an engineered human IGF-2 protein developed for subcutaneous injection. “Until treatments are available, DM1 will continue to progressively limit individuals' strength and their ability to do everyday things like walking, talking, swallowing, and even breathing,” said Dr. Tanya Stevenson, CEO of the Myotonic Dystrophy Foundation. “We are excited about the unique approach Juvena Therapeutics is advancing because of its potential to safely promote muscle regeneration, improve metabolism, rejuvenate lives, and ultimately to more holistically address the disease.” Juvena’s platform mines thousands of proteins secreted by human stem cells, screens their potential to restore tissue homeostasis, and engineers novel biologic medicines into life-saving treatments. The company has identified an expanded proprietary multi-dimensional library of regenerative proteins. JUV-161 leads Juvena’s preclinical pipeline of engineered biologics for multiple myopathies and metabolic diseases. Additionally, Juvena has identified multiple secreted protein hits that induce disease-modifying effects across several metabolic organs and therapeutic areas. “FDA-approved biologics based on secreted proteins such as insulin, human growth hormone, and erythropoietin, can have life-changing impacts; however, the massive search power needed to systematically map this complex class of proteins has challenged the industry's ability to continue discovering proteins that reverse disease in humans,” said Dr. Jeremy O'Connell, Co-Founder and Chief Scientific Officer of Juvena Therapeutics. “We are thrilled with the recognition of our lead candidate, JUV-161, as a potential addition to this class of drugs, and the success of Juvena’s platform in revolutionizing the study of secreted proteins as therapeutics.” The FDA's Office of Orphan Products Development grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan Drug designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials. About DM1 DM1 affects an estimated 1 in 2,100 people and can present at any age. People living with DM1 experience a range of signs and symptoms which may include muscle wasting and weakness, myotonia (slow muscle relaxation), cataracts, heart conduction and rhythm abnormalities, abnormal blood sugar control, and breathing problems. Currently, there are no treatments for DM1. About Juvena Therapeutics Juvena Therapeutics is a computationally-driven biopharmaceutical company on a mission to develop disease-modifying biologics to treat chronic muscle and metabolic diseases. The company’s approach stems from unlocking the therapeutic potential of stem-cell secreted proteins and translating them into a growing pipeline of engineered biologics. In addition to JUV-161, Juvena is progressing multiple preclinical programs, including a novel obesity asset with a unique mechanism. Juvena is achieving this through a fully integrated, end-to-end Artificial Intelligence (AI)-enabled drug discovery and development platform that combines a compounding database mapping secreted proteins to specific disease phenotypes, in silico and in vitro human cell screening, pharmacology, and protein engineering capabilities. To learn more about Juvena Therapeutics, visit JuvenaTherapeutics.com or follow the company on LinkedIn @Juvena-Therapeutics.

The prevalence of diabetes has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of Diabetes and the growing research and development activities to develop novel therapies to treat diabetes to drive the market. The companies developing the potential therapies in the last stage of development include Eli Lilly and Company, Daewoong Pharmaceutical, Janssen Biotech, and several others. LAS VEGAS, March 2, 2023 /PRNewswire/ -- DelveInsight's ' Diabetes Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline diabetes therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the diabetes pipeline domain. Key Takeaways from the Diabetes Pipeline Report DelveInsight's diabetes pipeline report depicts a robust space with 200+ active players working to develop 200+ pipeline therapies for diabetes treatment. Key diabetes companies such as Daewoong Pharmaceutical, Janssen Biotech, Zealand Pharma, BioRestorative Therapies, Elevian, Oramed Pharmaceuticals, ImCyse, Novo Nordisk, Enthera, Precigen, Inc., Japan Tobacco, Avotres, AstraZeneca, Landos Biopharma, Vertex Pharmaceuticals, REMD Biotherapeutics, Inc., Eledon Pharmaceuticals, Eli Lilly and Company, Novo Nordisk A/S, Diamyd Medical, NextCell Pharma, ViaCyte, Op-T LLC, Dompé Farmaceutici S.p.A, ILTOO Pharma, Throne Biotechnologies, Oramed Pharmaceuticals, Janssen Research & Development, LLC, Jaguar Gene Therapy, SQZ Biotechnologies, Genprex, Inc., CRISPR Therapeutics, Biora Therapeutics, Genprex, Inc., and others are evaluating new diabetes drugs to improve the treatment landscape. Promising diabetes pipeline therapies in various stages of development include Enavogliflozin, Golimumab, Dasiglucagon, ThermoStem, rGDF11, IMCY-0098, Insulin icodec, CagriSema, FDC Sema-OW GIP, SemaDapa FDC, ENT-001, AG019, JTT-662, AVT-001, AZD-0186, Cotadutide, LABP-111, VX-880, Encapsulated islet cell program, CPP-1X-T, REMD-477, AT-1501, LY 3209590, Orforglipron, LY3437943, Mazdutide, Peptide YY analogue agonist, GIPR agonist long acting, GIPR Agonist Long Acting II, LY 3493269, NNC0363-0845, Diamyd, ProTrans, VC-02, OPT101, Ladarixin, ILT-101, Stem Cell Educator Therapy, ORMD-0801, Golimumab, JAG301, SQZ TAC research program, VCTX 211, and others. In February 2023, Genprex, Inc. announced that data highlighting the potential of its gene therapy for Type 1 diabetes was being presented by its research collaborators at the University of Pittsburgh at the four-day International Conference on Advanced Technologies and Treatments for Diabetes (ATTD 2023).These results were compelling as they demonstrated the potential for this gene therapy to create newly formed beta-like cells that can produce insulin. They also validated earlier studies of this approach in diabetic mouse models that showed restoration of normal blood glucose levels for several months. In February 2023, Provention Bio, Inc. announced the closing of the $35 million equity investment from Sanofi US under the previously announced Co-Promotion Agreement and Securities Purchase Agreement (the "Purchase Agreement").Pursuant to the Purchase Agreement, Sanofi purchased 2,712,497 shares of the Company's common stock, par value $0.0001 per share, at a price of $12.90 per share, representing a total investment of $35 million. In January 2023, iTolerance, Inc. announced it has entered into a Master Services Agreement (MSA) with the Diabetes Research Institute (DRI) at the University of Miami Miller School of Medicine, for the advancement of its novel iTOL-100 platform technology as a potential cure for Type 1 Diabetes. In November 2022, Biocon Limited announced the signing of a semi-exclusive partnership agreement with Zentiva, a leading pharmaceutical company in Europe, for the commercialization of its vertically integrated, complex formulation, Liraglutide, a drug-device combination for the treatment and management of Type 2 diabetes and obesity.Under the terms of this agreement, Biocon is responsible for the manufacturing and supply of Liraglutide to Zentiva for its commercialization across 30 countries in Europe. Biocon also retained the right to commercialize this product under its own brand in the region. In November 2022, The US Food and Drug Administration (FDA) approved Provention Bio's biologics license application (BLA) for Tzield (teplizumab-mzwv) to treat type 1 diabetes (T1D) patients. The anti-CD3-directed antibody Tzield aims to delay the onset of Stage 3 T1D in adults and children aged eight years and above who are currently with stage 2 T1D. Request a sample and discover the recent advances in diabetes drug treatment @ Diabetes Pipeline Report The diabetes pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage diabetes drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the diabetes clinical trial landscape. Diabetes Overview Diabetes is a chronic disease that occurs when the pancreas does not produce enough insulin or when the body does not use the insulin that is produced effectively. The vast majority of diabetes can be divided into two types: type 1 and type 2. Diabetes symptoms include polyuria, polydipsia, weight loss, sometimes accompanied by polyphagia and blurred vision. Chronic hyperglycemia may also cause growth impairment and susceptibility to certain infections. Diabetes long-term complications include retinopathy, which can lead to vision loss, nephropathy, which can lead to renal failure, peripheral neuropathy, which can lead to foot ulcers, amputations, and Charcot joints, and autonomic neuropathy, which can cause gastrointestinal, genitourinary, and cardiovascular symptoms. Diabetes is diagnosed through a thorough physical examination, medical history, and a battery of specialized tests. The A1C, or glycated hemoglobin test, is the primary test used to diagnose both type 1 and type 2 diabetes. Diabetes treatment typically consists of diet control, exercise, home blood glucose testing, oral medication, and insulin. Insulin is the primary treatment for type 1 diabetes, and people with type 2 diabetes can be treated with oral medications but may need insulin in some cases. Find out more about drugs for diabetes @ New Diabetes Drugs A snapshot of the Diabetes Pipeline Drugs mentioned in the report: Learn more about the emerging diabetes pipeline therapies @ Diabetes Clinical Trials Diabetes Therapeutics Assessment The diabetes pipeline report proffers an integral view of diabetes emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Diabetes Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: Tumor necrosis factor alpha inhibitors, Glucagon receptor agonists, Cytotoxic T lymphocyte stimulants, Apoptosis inhibitors, Caspase inhibitors, Insulin-like growth factor binding protein inhibitors, Gene transference, Interleukin-10 expression modulators, CD40 antigen modulators Key Diabetes Companies: Daewoong Pharmaceutical, Janssen Biotech, Zealand Pharma, BioRestorative Therapies, Elevian, Oramed Pharmaceuticals, ImCyse, Novo Nordisk, Enthera, Precigen, Inc., Japan Tobacco, Avotres, AstraZeneca, Landos Biopharma, Vertex Pharmaceuticals, REMD Biotherapeutics, Inc., Eledon Pharmaceuticals, Eli Lilly and Company, Novo Nordisk A/S, Diamyd Medical, NextCell Pharma, ViaCyte, Op-T LLC, Dompé Farmaceutici S.p.A, ILTOO Pharma, Throne Biotechnologies, Oramed Pharmaceuticals, Janssen Research & Development, LLC, Jaguar Gene Therapy, SQZ Biotechnologies, Genprex, Inc., CRISPR Therapeutics, Biora Therapeutics, Genprex, Inc., and others. Key Diabetes Pipeline Therapies: Enavogliflozin, Golimumab, Dasiglucagon, ThermoStem, rGDF11, IMCY-0098, Insulin icodec, CagriSema, FDC Sema-OW GIP, SemaDapa FDC, ENT-001, AG019, JTT-662, AVT-001, AZD-0186, Cotadutide, LABP-111, VX-880, Encapsulated islet cell program, CPP-1X-T, REMD-477, AT-1501, LY 3209590, Orforglipron, LY3437943, Mazdutide, Peptide YY analogue agonist, GIPR agonist long acting, GIPR Agonist Long Acting II, LY 3493269, NNC0363-0845, Diamyd, ProTrans, VC-02, OPT101, Ladarixin, ILT-101, Stem Cell Educator Therapy, ORMD-0801, Golimumab, JAG301, SQZ TAC research program, VCTX 211 and others. Dive deep into rich insights for new drugs for diabetes treatment; visit @ Diabetes Medications Table of Contents For further information on the diabetes pipeline therapeutics, reach out @ Diabetes Drug Treatment Related Reports Type 1 Diabetes Market Type 1 Diabetes Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key type 1 diabetes companies, including Histogen , Novo Nordisk, Prevention Bio, among others. Type 1 Diabetes Epidemiology Forecast Type 1 Diabetes Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted type 1 diabetes epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Diabetes Market Diabetes Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key diabetes companies, including TikoMed, Avotres, REMD Biotherapeutics, Novo Nordisk, among others. Type 2 Diabetes Market Type 2 Diabetes Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key type 2 diabetes companies, including Sanofi, Eli Lilly and Company, AstraZeneca, Novartis, among others. Type 2 Diabetes Pipeline Type 2 Diabetes Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key type 2 diabetes companies, including Sanofi, Eli Lilly and Company, Novartis, among others. 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Sept. 29, 2022 12:00 UTC New publication highlights novel and differentiated NMDA receptor mechanism, and rapid and long-lasting antidepressant activity with lead clinical program, Zelquistinel Supports further development of Zelquistinel in a Phase 2 confirmatory study in Major Depressive Disorder, with expected initiation in early 2023 Company highlights business update, recent and upcoming clinical milestones, and additions to advisory network, after emerging from stealth last month INDIANAPOLIS--(BUSINESS WIRE)-- Gate Neurosciences, Inc. today announced the results of research, published in the International Journal of Neuropsychopharmacology, supporting the mechanism of action and clinical foundation of Zelquistinel – the company’s lead oral program in Phase 2 development – as a rapid-acting, long-lasting and safe antidepressant. Publication highlights robust preclinical pro novel mechanism for lead Phase 2 clinical program, Zelquistinel The peer-reviewed manuscript spotlights Zelquistinel’s novel mechanism and pharmacology by which it elicits rapid and long-lasting antidepressant effects. Zelquistinel was shown to positively modulate NMDA receptors (NMDARs) and enhance synaptic plasticity in the hippocampus and medial prefrontal cortex. A single dose of the molecule demonstrated a dose-dependent antidepressant-like response within one hour of administration that was sustained for more than 7 days. The studies highlighted in the publication build upon a robust underlying translational data package and predictive dose model that is consistent across Gate’s class of NMDAR modulators. Zelquistinel’s potential as an oral pill was confirmed by its high bioavailability and brain exposure, and also shows 1000-fold greater potency and a longer plasma half-life compared to its precursor molecule Rapastinel (GLYX-13). In terms of safety, Zelquistinel lacked the sedative, ataxic, and motor impairment effects that are typically observed with NMDAR antagonists and notably, it reversed NMDAR antagonist side effects upon co-administration. These data highlight Zelquistinel’s novel, potent, and highly differentiated mechanism of action with potential for a significantly improved safety pro to other NMDAR-targeted antidepressants in development for depressive disorders. “Zelquistinel’s mechanism of action represents a novel pharmacological approach which may help address patient needs in the treatment of depression,” said Maurizio Fava M.D., Psychiatrist-in-Chief at Massachusetts General Hospital, and a key advisor to Gate Neurosciences. “The upcoming Phase 2 study seeks to navigate the known challenges of conducting trials in psychiatry to confirm efficacy in Major Depressive Disorder.” Business Update Since its inception in 2019, Gate Neurosciences has assembled and advanced a clinical-stage pipeline of next-generation central nervous system therapies, as previously highlighted when the company emerged from stealth-mode in August 2022. Alongside the previously detailed Zelquistinel, Apimostinel and Rapastinel programs acquired from AbbVie, the company has also acquired exclusive rights to preclinical mGlu2/3 receptor antagonists from Eli Lilly which it is currently developing for sleep disorders, and is advancing a preclinical-stage Insulin Growth Factor 2 Receptor (IGF2R) program for cognitive disorders in collaboration with Northwestern University. Recent Milestones Completed financings totaling $25M from life science-focused investors, including Innoviva, Luson Bioventures, Biocrossroads, and ThornApple Capital, among others. Appointed new Board of Directors member Sarah Schlesinger M.D., a leading clinical investigator and chair of the Institutional Review Board at Rockefeller University, and member of the Board of Directors of Innoviva, Inc. Successfully advanced Investigational New Drug (IND) Applications for lead clinical programs. Initiated a Phase 1b clinical study exploring human target engagement biomarkers with 2nd generation injectable NMDAR program Apimostinel, in development for acute severe depression. Established extensive scientific and clinical advisor network, including leading psychiatrists across top institutions in the US. Hosted an R&D Day event at the 2022 ASCP Annual Meeting: “Update on NMDAR Modulators for Depressive Disorders.” Published in several peer-reviewed journals highlighting novel NMDAR mechanism and antidepressant activity of lead programs. The full list of publication can be found at gateneuro.com/publications. Upcoming Milestones First patient dosed in a single- and repeat-dose Phase 1b safety and EEG (electroencephalography) target engagement biomarker study of Apimostinel in 40 healthy volunteers in Q4 2022 with topline readouts expected in Q1 2023. Initiation of a confirmatory Phase 2 study of Zelquistinel in subjects with major depressive disorder (MDD) in early 2023, with topline readout expected mid-2024. Advancement of Rapastinel (GLYX-13) into investigator-led studies in collaboration with multiple leading research hospitals, under the FDA’s Expanded Access Program, beginning in 2023. Key Additions to Advisory Network Gate Neurosciences has continued expanding its network of 20+ scientific and clinical advisors from top psychiatry institutions across the US, who are providing encouraging perspectives on the company’s NMDA receptor modulator portfolio. Recent key additions include Maurizio Fava, M.D., Psychiatrist-in-Chief at Massachusetts General Hospital, and Thomas Laughren, M.D., Former Director for the Division of Psychiatry Products at the FDA. NMDAR Modulator Portfolio: Data Highlights & Mechanistic Understanding The core scientific focus of Gate’s pipeline is addressing synaptic dysfunction, the hallmark disease biology of many cognitive and psychiatric disorders, by enhancing synaptic plasticity and normalizing brain signaling. NMDARs play an essential role in synaptic plasticity and are the targets of an emerging class of rapid-acting antidepressants. Considerable recent progress has been made, along with a suite of publications, clarifying the underlying mechanisms various NMDAR-targeted molecules. Gate Neurosciences’ lead programs are highly differentiated from other NMDAR-targeted antidepressants by acting through positive modulation instead of antagonism. Gate’s molecules positively modulate NMDAR activity on glutamatergic neurons to enhance synaptic plasticity and elicit antidepressant activity. In contrast, antagonists act through NMDARs located on GABA interneurons, causing a profound ‘burst’ of neurotransmitters and accompanying dissociative side effects, before indirectly stimulating downstream effects on synaptic plasticity. Gate’s novel approach represents a more direct mechanism for enhancing natural neuronal plasticity processes, with the potential for a significantly improved safety profile. Recent literature highlighting Gate’s novel mechanism can be found on its website. Key data highlights from the company’s lead NMDAR programs include: Zelquistinel: 3rd generation oral NMDAR modulator Development as a rapid, durable, and well-tolerated chronic oral therapy to address more than 17 million Americans with MDD Supported by a robust, de-risking translational data package across ~350 patients, including consistent target engagement and EEG biomarkers, safety, and clinical antidepressant efficacy A Phase 2a exploratory dose-finding study in MDD (n=251) showed that Zelquistinel 10mg delivered once-weekly was generally safe and well-tolerated, and demonstrated statistically significant differences from placebo on MADRS change from baseline at multiple timepoints over the study Apimostinel: 2nd generation IV NMDAR modulator Development as an acute injectable procedure for the rapid reduction of depressive symptoms in millions of Americans with acute depressive episodes Molecule is 1000-fold more potent than precursor compound Rapastinel, and is the most potent compound in Gate’s class of NMDAR modulators A Phase 2a proof-of-concept study in MDD (n=151) showed that a single dose of Apimostinel 10mg was generally safe and well-tolerated with no ketamine-like side effects, and demonstrated a 5.4-point difference from placebo on HDRS-17 change from baseline at 24-hours (p=0.0034) and a 74% response rate (p=0.0067), among the largest seen to-date within the class of rapid antidepressants Early Stage Research and Development Highlights Company continues to advance preclinical programs towards IND milestones in 2023 GATE-102: oral mGlu2/3 receptor antagonist for hypersomnia GATE-301: IGFBP2 mimetic for neurodegenerative and neurodevelopmental disorders GATE-252: 4th generation oral NMDAR modulator for cognitive disorders Company established cutting-edge research operations at Northwestern University’s Falk Center for Molecular Therapeutics led by Dr. Joseph Moskal Ph.D., the original inventor of Gate’s NMDAR platform Collaborating with leading research institutions with access to world-class laboratory facilities, equipment, and capabilities Advancing development of objective biomarkers for mood disorders, including novel methods to identify and predict patient responder subsets Advancing early research into growth-factor signaling and other novel drug targets of interest that enhance neuronal function and synaptic plasticity “We are excited about the momentum Gate Neurosciences has gathered heading into the remainder of the year, along with our upcoming clinical milestones for our lead programs,” said Mike McCully, President & CEO of Gate Neurosciences. “I believe our next-generation portfolio has true transformative potential for patients suffering from neuropsychiatric and cognitive disorders.” About Gate Neurosciences Gate Neurosciences is a precision medicine biotechnology company focused on advancing next-generation CNS treatments that address growing needs in mental health. The company is developing a portfolio of novel mechanisms of action that enhance synaptic function to address neuropsychiatric and neurocognitive diseases, including major depressive disorder (MDD). Using learnings from extensive clinical, preclinical, and translational data and a better understanding of CNS development challenges, the company is advancing its clinical pipeline using evidence-driven, precision psychiatry approaches.