Toxic leukoencephalopathy

Aclaris Therapeutics Announces Top-line Results from 12-Week Phase 2b Trial of Oral Zunsemetinib (ATI-450) for Moderate to Severe Rheumatoid Arthritis and Provides Corporate Update

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Monday, November 13, 2023
Webcast, Pancreatic cancer, MK2, Safety, Therapy, Clinical trial, Patient, ACR20, Rheumatoid arthritis, ACRS, Pharmacokinetics, Ulcerative colitis, Atopic dermatitis, Topical medication, Toxic leukoencephalopathy, Osteoporosis, MTX, Pharmaceutical industry, Czech Republic

WAYNE, Pa., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drugs for immuno-inflammatory diseases, today announced top-line results from a Phase 2b study of zunsemetinib (ATI-450), an investigational oral MK2 inhibitor, in subjects with moderate to severe rheumatoid arthritis (RA; ATI-450-RA-202).

Key Points: 
  • The primary efficacy endpoint is the proportion of patients achieving an ACR20 response following 12 weeks of treatment.
  • Based on the overall program results, Aclaris will discontinue further development of the ATI-450 program, including halting enrollment of Aclaris’ ongoing Phase 2a trial of zunsemetinib in psoriatic arthritis.
  • “We are deeply disappointed with the results of this trial and for patients suffering from rheumatoid arthritis.
  • Aclaris is supporting Washington University in a first-in-human investigator-initiated Phase 1a trial of ATI-2231 in patients with advanced solid tumor malignancies.

Aptose Reports Results for the Third Quarter 2023

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Thursday, November 9, 2023
Apoptosis, ASH, University, Acute myeloid leukemia, APS, Patient, Poster, VEN, SYK, TUS, FLT3, ORR, KIT, CMML, Toxic leukoencephalopathy, AML, MD Anderson Cancer Center, Society, TSX, Cell, European School, Pharmaceutical industry, Airline, Venetoclax

“Investigator enthusiasm for our tuspetinib/venetoclax (TUS/VEN) doublet has led to brisk enrollment in the APTIVATE trial.

Key Points: 
  • “Investigator enthusiasm for our tuspetinib/venetoclax (TUS/VEN) doublet has led to brisk enrollment in the APTIVATE trial.
  • In the most recent data cut (October 23, 2023), the favorable safety profile remained consistent for TUS and TUS/VEN treated R/R AML patients.
  • Aptose researchers investigated the effects of TUS on key elements of the phospho-kinome and apoptotic proteome in both parental and TUS-resistant AML cells.
  • APTIVATE oral presentation at ASH 2023 by Dr.

Bicycle Therapeutics Provides Data Updates for Three Clinical Programs and Strategy Overview at First R&D Day

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Thursday, December 14, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Webcast, MT1-MMP, Peripheral neuropathy, Brain, Safety, Immune system, Cervical cancer, Food, Therapy, Toxicity, Head, Diabetic neuropathy, Nobel Prize, Patient, CD137, Bicycle, FDA, Non-small-cell lung cancer, Pembrolizumab, Neoplasm, Ionis, Investigational New Drug, Brentuximab vedotin, MUC, ORR, Ovary, Anal cancer, Cancer, Ovarian cancer, Toxic leukoencephalopathy, Pain, R, Drug combination, EV, Central nervous system, TNBC, NSCLC, BRC, Pharmaceutical industry, Vaccine, Birth control, Medical imaging, Thromboxane A2

“In totality, the data support the emerging differentiated profile of our Bicycle® molecules, paving the way to deliver best-in-class or first-in-class therapies for many cancers.

Key Points: 
  • “In totality, the data support the emerging differentiated profile of our Bicycle® molecules, paving the way to deliver best-in-class or first-in-class therapies for many cancers.
  • BT8009 is a Nectin-4 Bicycle toxin conjugate (BTC®) designed to overcome the significant toxicity associated with other toxin conjugate approaches.
  • Over 2024, Bicycle Therapeutics intends to generate early human imaging data from its wholly owned BRC pipeline.
  • Bicycle Therapeutics will continue to develop Bicycle® molecules to address disease outside of oncology through innovative partnerships.

Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome

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Thursday, November 30, 2023
Health, Neurology, Surgery, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Courage, Associate, Brain, Research, Safety, GMP, Food, Hope, Pediatrics, Toxicity, Baylor University, Clinical trial, MECP2, DSM-IV codes, Patient, FDA, Baylor College of Medicine, Data monitoring committee, McMinn, Toxic leukoencephalopathy, Epilepsy surgery, Rett syndrome, ICV, Orphan, Female, Inc., Șaeș, Neurosurgery, Construct, AAV, Hospital, Spinal cord, Neurodevelopmental disorder, Pharmaceutical industry, Vaccine

NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize the therapeutic activity while averting transgene overexpression toxicities.

Key Points: 
  • NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize the therapeutic activity while averting transgene overexpression toxicities.
  • NGN-401 delivers the full-length human methyl cytosine binding protein 2 (MECP2) gene, providing an optimal gene replacement approach.
  • In non-clinical studies with NGN-401 at clinically relevant doses, cardinal features of Rett syndrome were ameliorated, and no overexpression toxicity was observed.
  • Your resilience, courage, and support not only contribute to the progress of this research, but also inspire hope within the entire Rett syndrome community.

BioMimetix Presents Data from Phase 2 Study of BMX-001 Demonstrating Increased Survival of 6.6 Months in High Grade Glioma Patients at the 2023 Society for Neuro-Oncology Annual Meeting

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Friday, November 17, 2023
Oncology, Health, Neurology, Clinical Trials, Research, Science, Biotechnology, Temozolomide, Annual general meeting, Radiation, Porphyrin, Pakistan Society of Neuro-Oncology, HGG, Disease, Patient, TMZ, White matter, Neuro-Oncology (journal), Prognosis, Standard of care, MD, RT, Cancer, Survival, Toxic leukoencephalopathy, Growth, Neoplasm, Quality of life, SNO, Lead, Medical imaging, Cognition

“Patients with high grade glioma are challenging to treat because the disease grows quickly, ultimately outcomes are very poor for these patients and often carries an unfavorable prognosis.

Key Points: 
  • “Patients with high grade glioma are challenging to treat because the disease grows quickly, ultimately outcomes are very poor for these patients and often carries an unfavorable prognosis.
  • This is demonstrated by the promising effects seen in this trial with BMX-001 on patients with high grade glioma receiving concurrent radiation and chemotherapies,” said James Crapo, M.D., Chief Executive Officer at BioMimetix.
  • “High grade glioma is an aggressive brain cancer with a low survival rate that affects 10,000-15,000 people in the US per year.
  • Patients on concurrent RT/TMZ who also received BMX-001 were found to have an increased median survival of 6.6 months.

Menarini Group Presented Initial Safety and Efficacy Data from Phase 1b/2 ELEVATE and ELECTRA Combination Studies of ORSERDU® (Elacestrant) in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the 2023 San Antonio Breast Cancer Symposium

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Monday, December 11, 2023
Medicine, Observation, Research, Safety, CDK, Therapy, Toxicity, Cohort, University, Disease, Everolimus, Learning, Brain, Patient, Ribociclib, Division, Pharmacokinetics, Hematology, Neutropenia, Menarini, Diarrhea, Nausea, Cancer, Toxic leukoencephalopathy, Traffic barrier, Pharmaceutical industry, MD

Encouraging results from phase 1 ELECTRA, evaluating ORSERDU in combination with abemaciclib regardless of metastatic sites, show a tolerable and manageable safety profile with favorable preliminary efficacy.

Key Points: 
  • Encouraging results from phase 1 ELECTRA, evaluating ORSERDU in combination with abemaciclib regardless of metastatic sites, show a tolerable and manageable safety profile with favorable preliminary efficacy.
  • Both the ELEVATE and ELECTRA studies were designed with the objective to overcome different resistance mechanisms and enhance patient outcomes with combination treatment options.
  • Data was presented at the 2023 San Antonio Breast Cancer Symposium (SABCS), December 5-9, 2023.
  • "We look forward to learning more about ORSERDU's role in combination settings and its potential across the spectrum of metastatic breast cancer."

Menarini Group Presented Initial Safety and Efficacy Data from Phase 1b/2 ELEVATE and ELECTRA Combination Studies of ORSERDU® (Elacestrant) in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the 2023 San Antonio Breast Cancer Symposium

Retrieved on: 
Monday, December 11, 2023
Medicine, Observation, Research, Safety, CDK, Therapy, Toxicity, Cohort, University, Disease, Everolimus, Learning, Brain, Patient, Ribociclib, Division, Pharmacokinetics, Hematology, Neutropenia, Menarini, Diarrhea, Nausea, Cancer, Toxic leukoencephalopathy, Traffic barrier, Pharmaceutical industry, MD

Encouraging results from phase 1 ELECTRA, evaluating ORSERDU in combination with abemaciclib regardless of metastatic sites, show a tolerable and manageable safety profile with favorable preliminary efficacy.

Key Points: 
  • Encouraging results from phase 1 ELECTRA, evaluating ORSERDU in combination with abemaciclib regardless of metastatic sites, show a tolerable and manageable safety profile with favorable preliminary efficacy.
  • Both the ELEVATE and ELECTRA studies were designed with the objective to overcome different resistance mechanisms and enhance patient outcomes with combination treatment options.
  • Data was presented at the 2023 San Antonio Breast Cancer Symposium (SABCS), December 5-9, 2023.
  • "We look forward to learning more about ORSERDU's role in combination settings and its potential across the spectrum of metastatic breast cancer."

TC BioPharm Announces Successful Completion of Safety Cohort and Positive DSMB Results

Retrieved on: 
Tuesday, November 14, 2023
Safety, Acute myeloid leukemia, PLC, Data monitoring committee, Clinical trial, Patient, DSMB, TC, Cancer, Toxic leukoencephalopathy, Holding, Pharmaceutical industry

EDINBURGH, Scotland, Nov. 14, 2023 /PRNewswire/ -- TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer, announces today that the Company has successfully completed its Safety Cohort and received positive review from the Data Safety Monitoring Board (DSMB).

Key Points: 
  • EDINBURGH, Scotland, Nov. 14, 2023 /PRNewswire/ -- TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer, announces today that the Company has successfully completed its Safety Cohort and received positive review from the Data Safety Monitoring Board (DSMB).
  • The DSMB review was held after completion of enrollment of the initial cohort of the ACHIEVE trial and examined the safety data from all patients enrolled to ensure of no dose limiting toxicities or drug related serious adverse events.
  • TC BioPharm's trial involves administering TCB008, dubbed OmnImmune® in the UK, to treat Acute Myeloid Leukemia in a large swathe of patient population.
  • "This positive safety review marks an important milestone in the development of our allogeneic gamma delta platform, building on the strong safety profile that we previously established for our novel therapeutic," said Bryan Kobel, Chief Executive Officer of TC BioPharm.

SFA Therapeutics Announces Updated Psoriasis Data for SFA-002, Bridge Financing, and Foundational Patent

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Thursday, November 9, 2023
FRSC, Psoriasis, MD–PhD, Patent, Composition of matter, SFA, TB, Cytokine, FRCPC, University, Research, Psoriasis Area and Severity Index, Conditional sentence, IGA, Disease, Cancer, Autoimmune disease, OC, Infection, Safety, Therapy, Patient, PASI, Immunosuppression, Toxic leukoencephalopathy, Pharmaceutical industry

Additional participants in this raise were the Ben Franklin Technology Partners of Pennsylvania, Savantus Ventures and Asymmetry Ventures.

Key Points: 
  • Additional participants in this raise were the Ben Franklin Technology Partners of Pennsylvania, Savantus Ventures and Asymmetry Ventures.
  • SFA-002 is an oral pill that simultaneously acts on multiple therapeutic pathways, unlike other drugs on the market which target a single channel.
  • SFA Therapeutics is also pleased to announce that North South Ventures Managing Partner, Dr. David Naylor, MD/PhD, OC, FRCPC, FRSC, has joined SFA Therapeutics’ Board of Directors.
  • Psoriasis is an incurable chronic disease that affects 125 million people worldwide, with the global psoriasis treatment market size projected to grow at a CAGR of 9.2%.

Matinas BioPharma Announces Positive in vivo Efficacy Results of Oral LNC Docetaxel Formulation in a Melanoma Model

Retrieved on: 
Tuesday, November 7, 2023
Neoplasm, LNC, Docetaxel, Lecture, NYSE, Toxicity, Infection, IV, Therapy, Toxic leukoencephalopathy, Pharmaceutical industry, Vaccine

Currently, docetaxel is administered intravenously and can be associated with significant side effects and toxicities.

Key Points: 
  • Currently, docetaxel is administered intravenously and can be associated with significant side effects and toxicities.
  • “We are excited to report new in vivo data demonstrating the therapeutic efficacy of an oral LNC formulation of docetaxel,” said Dr. James J. Ferguson, Chief Medical Officer of Matinas .
  • “The study shows reductions in tumor size comparable to systemic intravenous docetaxel in a well-validated mouse melanoma model,” he added.
  • Additional analyses of the data are ongoing, and we are preparing them for presentation at upcoming scientific meetings.”
    Purpose: Document the efficacy of an oral LNC docetaxel formulation to target and treat tumors, with both negative (untreated) and positive (IV docetaxel) controls.