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New Data Demonstrate Sunbird Bio’s APEX Diagnostic Test Accurately Differentiates Key Alzheimer’s Disease Biomarker in Blood

Retrieved on: 
Tuesday, March 5, 2024
Mental Health, Research, Neurology, Clinical Trials, Biotechnology, Health, General Health, Other Science, Science, Traffic barrier, EV, System, Blood, Cerebrospinal fluid, Neurological disorder, AUC, Amplification, Conference, Seminal vesicles, Biomarker, Patient, Dementia, DSM-IV codes, CSF, Disease, Diagnosis, PET, APEX, Brain, Medical imaging

The data will be shared this week in a poster presentation at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Disease.

Key Points: 
  • The data will be shared this week in a poster presentation at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Disease.
  • “These results provide further evidence that the APEX platform has the potential to offer highly sensitive and accurate Alzheimer’s disease diagnosis equivalent to PET scans from a simple blood draw,” said John McDonough, executive chair and CEO of Sunbird Bio.
  • The APEX Aβ test is a blood-based diagnostic that uniquely detects very low levels of disease-specific proteins in blood with high accuracy.
  • EV-associated Aβ in blood are highly correlative and a direct reflection of Aβ build-up in the brain, a key abnormality in Alzheimer’s disease.

J INTS BIO, Phase 1/2 study of 'JIN-A02', a Novel Oral 4th Generation EGFR TKI, accepted for presentation at the upcoming American Association for Cancer Research 2024 meeting in USA (AACR 2024)

Retrieved on: 
Monday, February 26, 2024
NSCLC, Traffic barrier, Severance Hospital, EGFR, ESMO, ASCO, Permeability, Catholic University of Korea, National Cancer Center, Hospital, Asan Medical Center, AACR, Society, University, Seoul National University Hospital, Patient, Clinical trial, Oncology, Mutation, Trial of the century, Cancer, Pharmaceutical industry

J INTS BIO will present a poster during the "Phase I Clinical Trials in Progress" session, which will be held on April 8 from 1:30 to 5:00 p.m.

Key Points: 
  • J INTS BIO will present a poster during the "Phase I Clinical Trials in Progress" session, which will be held on April 8 from 1:30 to 5:00 p.m.
  • The topic of the presentation is "Phase 1/2 clinical trial of JIN-A02, a 4th generation EGFR-TKI for 3rd generation EGFR-TKI resistant patients in EGFR mutated advanced/metastatic non-small cell lung cancer".
  • In August last year, J INTS BIO registered the first patient for the global Phase 1/2 clinical trial of 'JIN-A02' at Severance Hospital in Korea.
  • A J INTS BIO official said, "The global clinical trial for 'JIN-A02' is progressing well and is receiving interest in the global market."

J INTS BIO, Phase 1/2 study of 'JIN-A02', a Novel Oral 4th Generation EGFR TKI, accepted for presentation at the upcoming American Association for Cancer Research 2024 meeting in USA (AACR 2024)

Retrieved on: 
Monday, February 26, 2024
NSCLC, Traffic barrier, Severance Hospital, EGFR, ESMO, Multimedia, ASCO, Permeability, Catholic University of Korea, National Cancer Center, Hospital, Asan Medical Center, AACR, Society, University, Seoul National University Hospital, Patient, Clinical trial, Oncology, Mutation, Trial of the century, Cancer, Pharmaceutical industry

J INTS BIO will present a poster during the "Phase I Clinical Trials in Progress" session, which will be held on April 8 from 1:30 to 5:00 p.m.

Key Points: 
  • J INTS BIO will present a poster during the "Phase I Clinical Trials in Progress" session, which will be held on April 8 from 1:30 to 5:00 p.m.
  • The topic of the presentation is "Phase 1/2 clinical trial of JIN-A02, a 4th generation EGFR-TKI for 3rd generation EGFR-TKI resistant patients in EGFR mutated advanced/metastatic non-small cell lung cancer".
  • In August last year, J INTS BIO registered the first patient for the global Phase 1/2 clinical trial of 'JIN-A02' at Severance Hospital in Korea.
  • A J INTS BIO official said, "The global clinical trial for 'JIN-A02' is progressing well and is receiving interest in the global market."

Servier Receives Regulatory Filing Acceptances from FDA and EMA for Vorasidenib in the Treatment of IDH-Mutant Diffuse Glioma

Retrieved on: 
Wednesday, February 21, 2024
Orbis, Classification, PFS, European Commission, EMA, Traffic barrier, Progression-free survival, Confidence interval, Committee, Enzyme, MAA, Research and development, ASCO, Malignancy, Physician, FDA, PDUFA, Society, Glioma, TGR, Microsoft Access, Safety, INDIGO, Prognosis, Civil service commission, HR, CHMP, SNO, IDH, Patient, Medicine, BIRC, Public, Disease, Diagnosis, Mutation, Development, NDA, Priority review, Pharmaceutical industry

BOSTON and SURESNES, France, Feb. 21, 2024 /PRNewswire/ -- Servier, a global leader in oncology focused on delivering meaningful therapeutic progress for the patients it serves, today announced the FDA filing acceptance and priority review for a New Drug Application (NDA) for vorasidenib, as well as the EMA granting accelerated assessment for the vorasidenib Marketing Authorization Application (MAA). This innovative targeted therapy is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes for the treatment of IDH-mutant diffuse glioma. If approved, vorasidenib would become a first-in-class targeted therapy for patients with IDH-mutant gliomas and would mark Servier's sixth approval across IDH-mutant cancers. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of August 20, 2024, while the European Commission approval is anticipated in the second half of 2024.

Key Points: 
  • This innovative targeted therapy is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes for the treatment of IDH-mutant diffuse glioma.
  • If approved, vorasidenib would become a first-in-class targeted therapy for patients with IDH-mutant gliomas and would mark Servier's sixth approval across IDH-mutant cancers.
  • "In the realm of glioma treatment, innovation has been stagnant for nearly a quarter-century, posing challenges for patients who, post-surgery, may opt to defer treatment due to concerns around potential toxic side effects.
  • "This promising outcome brings hope to patients grappling with IDH-mutant diffuse gliomas, offering a potential breakthrough for those eagerly awaiting a new therapeutic option."

Rgenta Therapeutics to Present at the 19th Annual Huntington's Disease Therapeutics Conference

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Wednesday, February 21, 2024
Traffic barrier, MLH1, PMS2, Huntington's disease, GWAS, FXS, Conference, DM1, MSH3, PMS1, RNA, Therapy, FAN1, Gene, FRDA, CSO, Fragile X syndrome, Friedreich's ataxia, DNA, Vaccine

CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.
  • As part of the annual conference, Travis Wager, Ph.D., President & CSO of Rgenta will participate in the Targeting the DNA repair machinery to modulate somatic instability session on Tuesday, February 27, 2024 at 2:00 PM PT.
  • To date, there has been a lack of successful strategies for directly targeting the PMS1 protein with small molecule inhibitors.
  • For more information about the event, please visit the conference website .

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

Retrieved on: 
Wednesday, February 14, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Enzyme replacement therapy, BBB, JCR, MPS II, Traffic barrier, Glycogen storage disease, Ministry, MHLW, ERT, Hunter syndrome, Syndrome, Scheie syndrome, MPS, Safety, Developmental psychology, Hurler syndrome, Child, Patient, Hurler–Scheie syndrome, Trial of the century, Consciousness, DSM-IV codes, Hypotension, Mouse, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™

Retrieved on: 
Wednesday, February 7, 2024
Traffic barrier, Sanfilippo, Genomics, Hearing, Growth, BBB, AFL, Metabolism, Feinberg School of Medicine, Spleen, Dermatan sulfate, Patient, Cerebrospinal fluid, University of North Carolina, FDA, Cognition, COMPASS, Mouse, CSF, Physician, Hunter syndrome, MPS II, Brain, Behavior, MPS, Mucopolysaccharidosis, Hunting, University, SGSH, ETV, Liver, Alfa, Glycogen storage disease, Pharmaceutical industry

Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.

Key Points: 
  • Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.
  • New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity.
  • Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
  • “Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali.

InFlectis BioScience Completes Enrollment of Its Phase 2 Trial of IFB-088 for the Treatment of Amyotrophic Lateral Sclerosis; Anticipates Initial Results Later This Year

Retrieved on: 
Wednesday, January 31, 2024
Traffic barrier, INN, Amyotrophy, Telescopic sight

Nantes, France--(Newsfile Corp. - January 31, 2024) - InFlectis BioScience, a pioneer in developing first-in-class therapies for neuromuscular diseases, announced that it has completed enrollment in its Phase 2 clinical trial of IFB-088 (INN: icerguastat), a novel investigational treatment of Amyotrophic Lateral Sclerosis (ALS).

Key Points: 
  • Nantes, France--(Newsfile Corp. - January 31, 2024) - InFlectis BioScience, a pioneer in developing first-in-class therapies for neuromuscular diseases, announced that it has completed enrollment in its Phase 2 clinical trial of IFB-088 (INN: icerguastat), a novel investigational treatment of Amyotrophic Lateral Sclerosis (ALS).
  • To view the full announcement, including downloadable images, bios, and more, click here .
  • InFlectis completed enrollment in its Phase 2 clinical trial of IFB-088 in ALS.
  • Initial findings to be reported as early as the end of 2024.

Silo Pharma Announces Pipeline Prioritization for 2024 Targeting Mental Health, Chronic Pain, and Neurology

Retrieved on: 
Thursday, February 1, 2024
Traffic barrier, PTSD, Multiple sclerosis, Inflammation, Fibromyalgia, Chronic pain, Research, Plasma protein binding, Therapy, SILO, Neurology, IND, New Drug Application, UMB, Central nervous system, FDA, Columbia University, Ketamine, GLP, Food, Mental health, CNS, Social media, Post-traumatic stress disorder, Administration, University, Pharmaceutical industry

SARASOTA, FL, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Silo Pharma, Inc. (Nasdaq: SILO) (“Silo” or the “Company”), a developmental stage biopharmaceutical company focused on developing novel formulations and drug delivery systems for traditional therapeutics and psychedelic treatments, today announced updates for its strategic 2024 focus for its clinical pipeline. The Company is advancing a diversified portfolio of four drugs targeting three health areas: mental health, chronic pain, and neurology.

Key Points: 
  • The Company is advancing a diversified portfolio of four drugs targeting three health areas: mental health, chronic pain, and neurology.
  • A dose-ranging non GLP study of SPC-15 is in progress, with a final validation report expected in the first half of 2024.
  • As a self-administered treatment, SP-26 holds the potential to be the first at-home ketamine treatment approved for chronic pain management.
  • Silo Pharma Inc. (Nasdaq: SILO) is a developmental stage biopharmaceutical company developing novel therapeutics that address underserved conditions including stress-induced psychiatric disorders, chronic pain conditions, and central nervous system (CNS) diseases.

Denali Therapeutics Announces Presentations on Its Investigational Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapies at the Upcoming 2024 WORLDSymposium™

Retrieved on: 
Thursday, February 1, 2024
Traffic barrier, SAN, BBB, Biomarker, Poster, Degeneracy, CNS, MPS II, Therapy, MPS, Interim analysis, SGSH, ETV, Glycogen storage disease

SOUTH SAN FRANCISCO, Calif., Feb. 01, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Feb. 01, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.
  • Presentations on DNL310 will include new two-year data on clinical outcomes and previously presented data on biomarkers from the ongoing Phase 1/2 study in MPS II.
  • In addition, an oral presentation will highlight data from preclinical studies of DNL126 in an MPS IIIA mouse model.
  • The presentation schedule at WORLDSymposium™ is as follows:
    Title: DNL310 Phase 1/2 Case Study Demonstrates Properties of Raw, Standard and Growth Scale Scores for Adaptive Behavior Scales (Poster #80)
    Title: DNL310 Normalizes Primary Storage Substrates, Corrects Biomarkers of Lysosomal Dysfunction and Reduces Biomarkers of Neuronal Injury (Neurofilament Light Chain) in MPS II: 2-Year Interim Analysis of a Phase 1/2 Study (Poster #34)
    Title: ETV:SGSH, a Brain-Penetrant Enzyme Transport Vehicle for SGSH, Improves Lysosomal and Microglial Morphology, Degeneration and Cognitive Behavior in MPS IIIA Mice
    Title: 3rd Annual Robert J. Gorlin Symposium – Beyond the Blood Brain barrier: Strategies for Treating the CNS