A Double-blind, Randomized, Placebo-controlled, Multicentric, Phase II Study in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) to Assess Efficacy, Safety, Tolerability and Pharmacokinetics of Multiple Intravenous Infusions of NX210c

This study will investigate the efficacy, safety, tolerability and pharmacokinetics (PK) of multiple intravenous infusions of NX210c, at two dose levels, in patients with Amyotrophic lateral sclerosis (ALS).

Start Date25 Oct 2024

Sponsor / Collaborator

Axoltis Pharma SA

[+1]

NL-OMON53584

/ CompletedNot Applicable

A double-blind, randomized, placebo-controlled, Multiple Ascending Dose (MAD) study in healthy elderly volunteers and Alzheimer's Disease (AD) and Parkinson's disease (PaD) patients to investigate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) effects of multiple intravenous infusions of NX210c - MAD study of NX210c

Start Date09 Dec 2022

Sponsor / Collaborator

Axoltis Pharma SA

NCT05827653

/ CompletedPhase 1

A Double-blind, Randomized, Placebo-controlled, Multiple Ascending Dose (MAD) Study in Healthy Elderly Volunteers and Alzheimer's Disease (AD) Patients to Investigate the Safety and Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) Effects of Multiple Intravenous Infusions of NX210c

This study will investigate the safety and tolerability of multiple intravenous infusions of NX210c with two ascending doses as well as NX210c pharmacokinetics (PK), and pharmacodynamics (PD) effects in healthy elderly subjects.

Start Date05 Dec 2022

Sponsor / Collaborator

Axoltis Pharma SA

[+1]

100 Clinical Results associated with NX-210c

100 Translational Medicine associated with NX-210c

100 Patents (Medical) associated with NX-210c

Literatures (Medical) associated with NX-210c

01 Feb 2025·Neurology and Therapy

Safety, Tolerability and Pharmacokinetic-Pharmacodynamic Relationship of NX210c Peptide in Healthy Elderly Volunteers: Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study

Article

Author: Lemarchant, Sighild ; Bambury, Pauline ; Le Douce, Juliette ; Marie, Sébastien ; Janus, Annette ; Pasculli, Giuseppe ; Dumas, Daniël ; Kremer, Philip ; Godfrin, Yann

INTRODUCTION:

Blood-brain barrier (BBB) integrity is fundamental to brain homeostasis, enabling control of substance exchange and safeguarding neurons against harmful toxins, pathogens, and immune cells that lead to dysregulation and inflammation involved in ageing and neurodegenerative diseases (NDD). The cyclized peptide NX210c is a thrombospondin type 1 repeat analogue derived from subcommissural organ-spondin. It exerts beneficial effects in animal models of NDD owing to its effects on neurons and endothelial cells. NX210c demonstrated a good safety profile in a single ascending dose phase 1a clinical study. The present multiple ascending dose phase 1b study was performed to evaluate the tolerability and pharmacological effects of repeated doses of NX210c in healthy elderly (age: > 55 years) volunteers.

METHODS:

This was a randomized, placebo-controlled, double-blind study (EudraCT No. 2022-002868-76), investigating safety/tolerability, pharmacokinetics, and pharmacodynamics (including blood and cerebrospinal fluid biomarkers). Participants received 5 or 10 mg/kg NX210c or placebo (10-min infusion) thrice weekly for 4 weeks in an ascending dose fashion. Follow-up was conducted 2 weeks after last dosing.

RESULTS:

The investigation included 29 participants. No serious adverse events were recorded and all adverse events were mild. Dedicated central nervous system testing did not reveal neurotoxicity. Biomarker evaluation showed a statistically significant reduction in blood claudin-5 and a trend toward reduction of blood homocysteine. In silico data modelling revealed salient pharmacokinetic-pharmacodynamic relationships, including reduction of claudin-5, neurofilament light chain, and SPARC-like protein 1 release, and degradation of homocysteine.

CONCLUSION:

Multiple doses of NX210c exhibited a good safety profile, showed non-cumulative pharmacokinetics, and exerted pharmacodynamic effects on biomarkers linked to BBB integrity. The effects of NX210c on claudin-5 and biomarkers influencing BBB integrity-and the overarching brain protection it offers-provide a novel therapeutic strategy targeting an underlying driver of neurodegenerative conditions for which disease-modifying treatments are limited or not available.

01 May 2021·NeuroscienceQ3 · MEDICINE

SCO-spondin-derived Peptide Protects Neurons from Glutamate-induced Excitotoxicity

Q3 · MEDICINE

Article

Author: Le Douce, Juliette ; Lemarchant, Sighild ; Godfrin, Yann ; Delétage, Nathalie ; Callizot, Noëlle

Subcommissural organ (SCO)-spondin is a brain-specific glycoprotein produced during embryogenesis, that strongly contributes to neuronal development. The SCO becomes atrophic in adults, halting SCO-spondin production and its neuroprotective functions. Using rat and human neuronal cultures, we evaluated the neuroprotective effect of an innovative peptide derived from SCO-spondin against glutamate excitotoxicity. Primary neurons were exposed to glutamate and treated with the linear (NX210) and cyclic (NX210c) forms of the peptide. Neuronal survival and neurite networks were assessed using immunohistochemistry or biochemistry. The mechanism of action of both peptide forms was investigated by exposing neurons to inhibitors targeting receptors and intracellular mediators that trigger apoptosis, neuronal survival, or neurite growth. NX210c promoted neuronal survival and prevented neurite network retraction in rat cortical and hippocampal neurons, whereas NX210 was efficient only in neuronal survival (cortical neurons) or neurite networks (hippocampal neurons). They triggered neuroprotection via integrin receptors and γ-secretase substrate(s), activation of the PI3K/mTOR pathway and disruption of the apoptotic cascade. The neuroprotective effect of NX210c was confirmed in human cortical neurons via the reduction of lactate dehydrogenase release and recovery of normal basal levels of apoptotic cells. Together, these results show that NX210 and NX210c protect against glutamate neurotoxicity through common and distinct mechanisms of action and that, most often, NX210c is more efficient than NX210. Proof of concept in central nervous system animal models are under investigation to evaluate the neuroprotective action of SCO-spondin-derived peptide.

Fluids and Barriers of the CNS

NX210c drug candidate peptide strengthens mouse and human blood-brain barriers

Article

Author: Lemarchant, Sighild ; Godfrin, Yann ; Janigro, Damir ; Fewell, Gwen ; Rebergue, Nicolas ; Greene, Chris ; Campbell, Matthew

Abstract:

Background:

Alterations of blood-brain barrier (BBB) and blood-spinal cord barrier have been documented in various animal models of neurodegenerative diseases and in patients. Correlations of these alterations with functional deficits suggest that repairing barriers integrity may represent a disease-modifying approach to prevent neuroinflammation and neurodegeneration induced by the extravasation of blood components into the parenchyma. Here, we screened the effect of a subcommissural organ-spondin-derived peptide (NX210c), known to promote functional recovery in several models of neurological disorders, on BBB integrity in vitro and in vivo.

Methods:

In vitro, bEnd.3 endothelial cell (EC) monolayers and two different primary human BBB models containing EC, astrocytes and pericytes, in static and microfluidic conditions, were treated with NX210c (1-100 µM), or its vehicle, for 4 h and up to 5 days. Tight junction (TJ) protein levels, permeability to dextrans and transendothelial electrical resistance (TEER) were evaluated. In vivo, young and old mice (3- and 21-month-old, respectively) were treated daily intraperitoneally with NX210c at 10 mg/kg or its vehicle for 5 days and their brains collected at day 6 to measure TJ protein levels by immunohistochemistry.

Results:

NX210c induced an increase in claudin-5 protein expression after 24-h and 72-h treatments in mouse EC. Occludin level was also increased after a 24-h treatment. Accordingly, NX210c decreased by half the permeability of EC to a 40-kDa FITC-dextran and increased TEER. In the human static BBB model, NX210c increased by ∼ 25% the TEER from 3 to 5 days. NX210c also increased TEER in the human 3D dynamic BBB model after 4 h, which was associated with a reduced permeability to a 4-kDa FITC-dextran. In line with in vitro results, after only 5 days of daily treatments in mice, NX210c restored aging-induced reduction of claudin-5 and occludin levels in the hippocampus, and also in the cortex for occludin.

Conclusions:

In summary, we have gathered preclinical data showing the capacity of NX210c to strengthen BBB integrity. Through this property, NX210c holds great promises of being a disease-modifying treatment for several neurological disorders with high unmet medical needs.

News (Medical) associated with NX-210c

13 Nov 2024

·www.prnewswire.com

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis 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, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultFast TrackCell TherapyImmunotherapy

12 Dec 2023

·pipelinereview.com

Axoltis Pharma presents promising results from phase 1b clinical trial of innovative drug candidate for neurodegenerative diseases

CLERMONT-FERRAND and LYON, France I December 12, 2023 I Axoltis Pharma, a French biopharmaceutical company dedicated to developing therapeutic solutions for neurodegenerative diseases, today announces promising results from its phase 1b clinical trial evaluating NX210c, a patented peptide drug candidate. These results follow a single-ascending dose phase 1a study carried out in 2020, which had demonstrated a good safety profile for NX210c in healthy volunteers. The phase 1b trial was a multiple ascending-dose study aimed at evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NX210c in healthy volunteers aged over 55 years (NCT05827653). During Q1, 2023, Axoltis enrolled 29 healthy volunteers (12 with 5mg/kg, 11 with 10mg/kg and 6 receiving a placebo) in a single-center, randomized, double-blind, placebo controlled, dose escalation study conducted at the Centre for Human Drug Research, Leiden (The Netherlands). NX210c was administrated in a ten-minute infusion via an intravenous route, three times a week for four weeks. The findings confirm that NX210c is safe and well-tolerated at all tested doses, any related adverse events observed were mild. Additionally, the specific neurologic safety profile of NX210c was strengthened by applying Neurocart® (a series of central nervous system tests) and included an extensive EEG signal review. “This trial confirms the excellent safety and tolerability profile of NX210c. Moreover, we are excited to observe significant pharmacodynamic effects on blood and cerebrospinal fluid biomarkers, especially those important for BBB integrity, neuroprotection and neurotransmission. The results are very promising and provide a strong foundation to build on the NX210c drug development strategy,” said Dr. Annette Janus, neurologist and chief medical officer at Axoltis. Axoltis recently presented these phase 1b results, combined with encouraging findings from the company’s preclinical studies, at Neuroscience 2023, the annual meeting of the Society for Neuroscience (SfN) in Washington DC (USA). In addition, Axoltis has been selected to deliver an oral presentation at AD/PD™ in Lisbon (Portugal) in March 2024. By using a computational modeling approach based on the clinical dataset, Axoltis was also able to show an NX210c pharmacokinetics-pharmacodynamics (PKPD) relationship for several biomarkers, strengthening its therapeutic approach. “We are very pleased with our preclinical and clinical studies, which strengthen Axoltis’ position to become a key leader in BBB repair. Among our target applications is the initiation of a phase 2 trial for NX210c in patients living with amyotrophic lateral sclerosis, as BBB deterioration may be a trigger in the progression of this terrible disease,” said Dr. Yann Godfrin, chief executive officer of Axoltis. In parallel to this study in ALS, Axoltis Pharma has already obtained authorization to extend its phase 1b study in the Netherlands with a cohort of Parkinson’s Disease patients. Additionally, Axoltis is partnered with InSilicoTrials, Milan (Italy), experts in in silico modeling, to explore the potential effects of NX210c in neurodegenerative disease models. About the Blood Brain Barrier (BBB) On a physiological level, the BBB separates the brain from the blood compartment, protecting it from circulating toxins and inflammatory blood components. When the BBB is no longer fully functional, these undesirable blood components can permeate the barrier and reach the brain. This plays a role in the progression of neurodegenerative diseases; it can even trigger them. For example, it has been recently shown in ALS that the BBB is damaged at the very early stage of the disease, probably even before clinical signs have become clear. About NX210c A large glycoprotein (SCO-spondin) produced by the subcommissural organ (SCO) plays crucial roles in neurogenesis and axonal guidance during embryogenesis. Only remnants of the SCO subsist in adults, thereby halting the production of SCO-spondin, which may account for a lack of regeneration and recovery in patients with neurological disorders. In this context, Axoltis is developing NX210c, a cyclic peptide of 12 amino acids designed from the most conserved sequence of the type 1 thrombospondin repeats of the SCO-spondin, as an innovative therapy for neurodegenerative diseases and traumas. NX210c is protected by seven patents fully owned by Axoltis Pharma, including one for composition of matter. In 2022, the FDA granted Orphan Drug Designation for NX210c in ALS. For more details: https://www.axoltis.com/our-product/properties/ About Axoltis Pharma Axoltis Pharma, a French biopharma company, develops innovative drugs for neurodegenerative and neurotraumatic diseases with a high unmet medical need. Headquartered in Clermont-Ferrand, with offices in Lyon, (France), Axoltis has established several partnerships with internationally recognized academic and private labs to develop its lead product, NX210c. The team is highly experienced in drug development, especially for neurological applications. The company is supported by significant grants from Bpifrance and Région Auvergne-Rhône-Alpes. www.axoltis.com

Phase 1Phase 2Orphan Drug

27 Jul 2023

·www.prnewswire.com

InSilicoTrials and Axoltis Pharma Collaborate to Advance Therapeutic Solutions for Neurological Disorders

MILAN, July 27, 2023 /PRNewswire/ -- InSilicoTrials, a leading provider of artificial intelligence (AI) and simulation tools for drug and medical device development, has embarked on a collaborative partnership with Axoltis Pharma, a biopharmaceutical company committed to developing disease-modifying drugs for patients with high unmet medical needs in neurodegenerative and traumatic neurologic impairments. This strategic collaboration works to revolutionize our understanding of Central Nervous System (CNS) diseases and to optimize the clinical development plan of Axoltis' drug candidate, NX210c, a promising 12-amino acid peptide derived from SCO-spondin. Continue Reading Collaboration between InSilicoTrials and Axoltis Pharma The primary focus of this partnership is to utilize the InSilicoTrials' platform and leverage its InSilicoNEURO suite, which encompasses a variety of mechanistic CNS models. More specifically, the models within InSilicoNEURO will be used to assess the impact of Axoltis' drug candidate on virtual patients with different conditions, including Amyotrophic Lateral Sclerosis (ALS), Parkinson's Disease and Multiple sclerosis (MS). Mechanistic CNS models are designed to simulate the physiological and pathological aspects of CNS disorders, providing insights into the disease mechanisms and potential therapeutic interventions. Integrating knowledge from various data sources, these models help researchers and clinicians understand the complex interactions within the CNS and contribute to the development of improved diagnostics and disease-modifying treatments for CNS disorders. Neurodegenerative conditions are often characterized by several common features, including blood-brain barrier leakage, synaptic transmission deteriorations, and neuron apoptosis. Given that Axoltis drug candidate NX210c targets these features, the collaboration with InSilicoTrials holds great promise for supporting CNS functions in a wide range of neurological and neurodegenerative conditions. By utilizing the InSilicoTrials' digital platform and leveraging Phase Ib clinical trial data and the preclinical package, the collaboration works to assess the relationship between NX210c dosing regimens, blood brain barrier repair and neurological function, biomarker concentrations, and ultimately clinical efficacy and safety endpoints, through computational modeling and simulation (CM&S) and AI techniques Unlocking the potential of artificial intelligence and computational modeling, the two companies work to accelerate research and development, minimize costs, and optimize time utilization. "We are thrilled to collaborate with Axoltis in our shared mission to advance therapeutic solutions for neurological disorders," said Luca Emili, CEO of InSilicoTrials. "This partnership represents an exciting synergy between cutting-edge simulation technologies and innovative biotechnological advancements. Together, we aim to unlock new insights and develop groundbreaking treatments that will positively impact the lives of patients with neurologic impairments worldwide." Dr. Yann Godfrin, CEO of Axoltis stated: "We are very delighted to initiate this collaboration with InSilicoTrials and have the opportunity to optimize and accelerate our next clinical steps. Integrating their industry-leading AI and computational modeling solutions in the development plan of our promising disease-modifying drug will allow us to address the pressing challenges faced by individuals suffering from neurological disorders." InSilicoTrials is an emerging startup founded by a team of life science, cybersecurity and digital innovation experts, which aims to revolutionize healthcare through an innovative modeling & simulation platform. InSilicoTrials has created a vertical software solution that integrates cutting-edge AI and simulation tools with the goal of helping pharmaceutical, medtech companies and researchers develop new drugs more efficiently and at a lower cost by reducing the need for traditional, time-consuming, and expensive clinical trials. Additionally, the solution contributes to the reduction, refinement, and replacement of in vitro, animal, and human testing methods. InSilicoTrials built up an ecosystem of more than 70 scientific collaborations, where computational models are developed with internationally recognized universities and research centers, offering access to the highest level of data security. With its cloud-based platform offering advanced M&S tools to perform in silico trials analyses, InSilicoTrials supports companies to integrate AI and simulation technology into their drug development workflows. Axoltis Pharma is a French biopharmaceutical company dedicated to developing a first-in-class disease-modifying drug to treat patients with neurodegenerative or traumatic neurological disorders with high unmet medical needs. NX210c, an innovative and promising drug-candidate, is a small peptide derived from SCO-spondin, a glycoprotein essential for neurogenesis during embryogenesis. This disease-modifying drug candidate has three main properties: recovery of blood brain barrier integrity, neuroprotection, and enhancement of neurotransmission. These properties are very useful in the treatment of multiple neurodegenerative diseases, especially as there is strong evidence that blood brain barrier dysfunction promotes disease progression from early stages of the disease. Photo - SOURCE InSilicoTrials

100 Deals associated with NX-210c

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Phase 2	France	Axoltis Pharma SA	25 Oct 2024
Alzheimer Disease	Phase 1	Netherlands	Axoltis Pharma SA	05 Dec 2022
Multiple Sclerosis	Phase 1	France	Axoltis Pharma SA	-
Parkinson Disease	Phase 1	-	Axoltis Pharma SA	-
Cerebrovascular Disorders	Discovery	France	Axoltis Pharma SA	17 Jun 2024

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05827653 (PipelineReview) Manual	Phase 1	-	29	NX210c (12 with 5mg/kg, 11 with 10mg/kg)	wxeztalynd(rzsstlghki) = The findings confirm that NX210c is safe and well-tolerated at all tested doses, any related adverse events observed were mild. ppjoxxllbp (bkjymzsoom )	Positive	12 Dec 2023
				Placebo
SFN2023	Not Applicable	-	-	NX210c 5 mg/kg	tyuxaduiwd(vgckqncckc) = The most common TEAE concerned nervous system disorders (51%, headache, somnolence) sosymbrook (qxznvacxit )	-	13 Nov 2023
				NX210c 10 mg/kg

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