NDD

AB Science: New research shows that masitinib limits neuronal damage in a model of neuroimmune-driven neurodegenerative disease

Retrieved on: 
Wednesday, March 13, 2024
Neurology, Serum, EAE, Multiple sclerosis, ALS, Disease, Patient, Biomarker, Masitinib, Research, Mouse, Science, University, MD, Euronext, Alzheimer's disease, Cytokine, AN, BioRxiv, FDA, NFL, Nerve, FIRST, AFL, NDD, Guideline, Pharmaceutical industry

Findings have been published on the bioRxiv preprint service as an article entitled, ‘Masitinib limits neuronal damage, as measured by serum neurofilament light chain concentration, in a model of neuroimmune-driven neurodegenerative disease’.

Key Points: 
  • Findings have been published on the bioRxiv preprint service as an article entitled, ‘Masitinib limits neuronal damage, as measured by serum neurofilament light chain concentration, in a model of neuroimmune-driven neurodegenerative disease’.
  • The neuroprotective action of masitinib was studied in an animal model of experimental autoimmune encephalitis (EAE).
  • Results showed that masitinib can significantly lower serum NfL levels, and by extension therefore, neuronal damage, in a neuroimmune-driven neurodegenerative disease model, with concomitant reduction in pro-inflammatory cytokines and slowing of clinical symptoms.
  • Masitinib limits neuronal damage, as measured by serum neurofilament light chain concentration, in a model of neuroimmune-driven neurodegenerative disease.

Vaccinex to Present Update on PDAC/Pepinemab Study; New ActivMAb® Application

Retrieved on: 
Wednesday, September 27, 2023
PDAC, AACR, Neurodegenerative disease, Cancer, NDD, RESEARCH, Patient, Observation, SEMA4D, Tumor microenvironment, Safety, Pharmaceutical industry

ROCHESTER, N.Y., Sept. 27, 2023 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease (NDD) through the inhibition of SEMA4D, today announced that it will present data on a new ActivMAb® application on September 28th and an update on the Phase 1b/2 pepinemab study in patients with PDAC on September 29th.

Key Points: 
  • Vaccinex will provide an overview of the design and enrollment for the pancreatic ductal adenocarcinoma (PDAC) study ( NCT05102721 ).
  • Treatment has been well-tolerated by these patients to date and a mixed response with some lesions showing radiologic partial and even complete response has been observed in one patient.
  • The study is being conducted with grant support from the Gateway Discovery Award (Conquer Cancer Foundation / ASCO).
  • AACR SPECIAL CONFERENCE IN CANCER RESEARCH: PANCREATIC CANCER

Vaccinex Reports First Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Monday, May 15, 2023
PDAC, Neoplasm, Merck Group, Cash, Patient, H. Lee Moffitt Cancer Center & Research Institute, American College, Three Months, Eurosport 1, University, Disease, MRK, Immunity, Research, GPCR, Security (finance), Drug discovery, FDA, Merck Sharp & Dohme Federal Credit Union, B cell, Abstract, G protein-coupled receptor, CCD8, Cancer, Immunotherapy, NDD, SEMA4D, Security, HD, ICIS, SIGNAL, Nature Medicine, Neurodegenerative disease, Nature Communications, Pfizer, Type, ASCO, CO, State Administrative Expenses, Annual general meeting, Tumor microenvironment, Avelumab, Breast cancer, Pharmaceutical industry, Vaccine, Cryptocurrency, Merck

ROCHESTER, N.Y., May 15, 2023 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease (NDD) and cancer through the inhibition of SEMA4D today announced financial results for the first quarter ended March 31, 2023 and provided a corporate update on key programs and financing in the first five months of the year.

Key Points: 
  • We expect to meet with our collaborator, Merck, in early June to review the results and consider plans for continued development of this novel immunotherapy combination.
  • Vaccinex plans to complete the interim analysis in May and to publicize results in June, 2023 after meeting with Merck to discuss the next steps.
  • Financial Results for the Three Months Ended March 31, 2023:
    Cash and Cash Equivalents and Marketable Securities.
  • Research and development expenses for the quarter ended March 31, 2023 were $3.8 million as compared to $3.0 million for the comparable period in 2022.

Vaccinex Completes Enrollment in Phase 1b/2 SIGNAL-AD (Alzheimer’s Disease) Study of Pepinemab

Retrieved on: 
Tuesday, April 25, 2023
Cognition, Neuron, SIGNAL, Neurodegenerative disease, Pathology, Cancer, Brain, Research, Nature Medicine, NDD, Dementia, HIV disease progression rates, Patient, AD, FDA, SEMA4D, Safety, Disease, Pharmaceutical industry, Medical imaging, HD

ROCHESTER, N.Y., April 25, 2023 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the inhibition of SEMA4D, today announced that it has completed enrollment goal in the SIGNAL-AD clinical trial for people with mild dementia due to Alzheimer’s Disease (NCT04381468). Topline data from the study are expected in mid-2024, after the last enrolled patients will have received 12 months of treatment.

Key Points: 
  • Topline data from the study are expected in mid-2024, after the last enrolled patients will have received 12 months of treatment.
  • The randomized, double-blinded, placebo-controlled Phase 1b/2 SIGNAL-AD study was designed to evaluate the safety, tolerability and effects on cognition and brain metabolism of the SEMA4D inhibitor, pepinemab.
  • The SIGNAL-AD study was designed to build on the exciting data obtained in the Phase 2 SIGNAL study of pepinemab in Huntington’s disease (HD), another neurodegenerative disease with many similarities in pathology to AD.
  • “While the treatment phase of the SIGNAL-AD study is ongoing, we are expecting FDA response to proposed plans for the design of a Phase 3 study in HD.

STALICLA and Firefly Neuroscience enter Partnership Agreement for the Development of Electroencephalogram (EEG) as a Biomarker for Subgroups of Patients with ASD

Retrieved on: 
Monday, March 27, 2023
Cognition, DEPI, Partnership, Autism spectrum, Neuroscience, DSM-IV codes, Biomarker, Firefly, CNS, NDD, Patient, ASD, EEG, Medicine, AI, FDA, Pharmaceutical industry, Medical device, Medical imaging

Geneva, Switzerland – 27 March 2023, STALICLA SA and Firefly Neuroscience today announced a partnership to further validate EEG-based biomarkers in biologically enriched subgroups of patients with Autism Spectrum Disorder (ASD).

Key Points: 
  • Geneva, Switzerland – 27 March 2023, STALICLA SA and Firefly Neuroscience today announced a partnership to further validate EEG-based biomarkers in biologically enriched subgroups of patients with Autism Spectrum Disorder (ASD).
  • Applying BNA™ to the DEPI-driven biologically enriched subgroups of patients with ASD is expected to establish and expand EEG as a biomarker in the field of ASD.
  • EEG measurements will reinforce the DEPI stratification capacity and prediction of treatment response in patients with a behavioral diagnosis of ASD.
  • Lynn Durham, STALICLA’s CEO & Founder, commented: “STALICLA is thrilled to partner with Firefly Neuroscience to advance an EEG-based treatment response marker in the NDD population.

Cara Therapeutics Announces Fourth Quarter and Full Year 2022 Financial Results

Retrieved on: 
Monday, March 6, 2023
New Drug Application, Nephrology, Fresenius, Dermatology, Dialysis, Civil service commission, Health care, Chronic kidney disease, Travel, Sale, Patient, KICK, NDD, AD, Itch, Medication, American Academy, Conference call, Research and development, Notalgia paresthetica, Atopic dermatitis, CSL, NP, G&A, CSL Vifor, European Commission, Investment, R, PMDA, KIND, Administration, CKD, Medicines and Healthcare products Regulatory Agency, Cara, Growth, Therapy, Conference, Acceleration, EST, Pharmaceuticals and Medical Devices Agency, Pharmaceutical industry, Nursing, Medical device, Cryptocurrency, Music, Difelikefalin, EU

STAMFORD, Conn., March 06, 2023 (GLOBE NEWSWIRE) -- Cara Therapeutics, Inc. (Nasdaq: CARA), a commercial-stage biopharmaceutical company leading a new treatment paradigm to improve the lives of patients suffering from pruritus, today announced financial results and operational highlights for the fourth quarter and full year ended December 31, 2022.

Key Points: 
  • During the fourth quarter of 2022, we saw both new clinic and repeat orders driving product uptake, bolstering our confidence in KORSUVA’s long-term potential,” said Christopher Posner, President and Chief Executive Officer of Cara Therapeutics.
  • In the fourth quarter of 2022, wholesalers shipped 20,844 vials to dialysis centers following initial inventory building in the third quarter of 2022, predominantly at Fresenius clinics.
  • The Company expects launches in countries in Europe to continue in 2023, following launches of Kapruvia in Austria, Germany, and Sweden during the fourth quarter of 2022.
  • With the approval of Kapruvia in Australia in the fourth quarter of 2022, all four Access Consortium countries have approved the product.

Disc Medicine Initiates a Phase 1b/2 Clinical Study of DISC-0974 in Adults with Non-Dialysis Dependent Chronic Kidney Disease (NDD-CKD) and Anemia

Retrieved on: 
Thursday, February 16, 2023
Erythropoiesis, NDD, PK, HB, Safety, CKD, Patient, JD, Clinical trial, Anemia, Dialysis, Chronic kidney disease, Connective tissue, Ruxolitinib, Expansion, Rollcage Stage II, Iron, Doctor of Philosophy, Pharmaceutical industry, HJV

DISC-0974 is a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, and thereby address anemia by enhancing the availability of iron for erythropoiesis.

Key Points: 
  • DISC-0974 is a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, and thereby address anemia by enhancing the availability of iron for erythropoiesis.
  • DISC-0974 is also currently being studied in a Phase 1b/2 clinical study in patients with myelofibrosis and anemia.
  • “We are excited to initiate this clinical trial of DISC-0974 in chronic kidney disease, where hepcidin plays a key role in the pathophysiology of anemia.
  • The study endpoints will include hepcidin levels, serum iron and markers of iron mobilization and changes in hemoglobin.

$25 Million Gift to Penn Medicine and Children's Hospital of Philadelphia Establishes Center for Epilepsy and Neurodevelopmental Disorders

Retrieved on: 
Tuesday, February 7, 2023
Health system, Department of Neurology, Xuanwu hospital, Mutation, Pathology, Trustee, NDD, Perelman School of Medicine at the University of Pennsylvania, Erlanger Health System, Epilepsy, Hope, ENGIN, DSM-IV codes, Diagnosis, Disorder, Neurodevelopmental disorder, Seizure, Physiology, Patient, Hospital, CRISPR, Division, Penn Presbyterian Medical Center, University, Research, Neurology, Intellectual, Genomics, Therapy, CHOP, Children's Hospital of Philadelphia, SYNGAP1, Raymond, STXBP1, ASO, Partnership, Penn, Davidson, Multimedia, Intellectual disability, Doctor of Philosophy, MD, Pharmaceutical industry, Medicine

PHILADELPHIA, Feb. 7, 2023 /PRNewswire/ -- A $25 million gift from an anonymous donor to Penn Medicine and Children's Hospital of Philadelphia (CHOP) will establish the Center for Epilepsy and Neurodevelopmental Disorders (ENDD), accelerating collaborative research in genetic therapies for neurodevelopmental disorders.

Key Points: 
  • PHILADELPHIA, Feb. 7, 2023 /PRNewswire/ -- A $25 million gift from an anonymous donor to Penn Medicine and Children's Hospital of Philadelphia (CHOP) will establish the Center for Epilepsy and Neurodevelopmental Disorders (ENDD), accelerating collaborative research in genetic therapies for neurodevelopmental disorders.
  • "This important gift will accelerate the progress of an extraordinary partnership between Penn and CHOP that demonstrates the power of collaboration and a commitment to finding cures.
  • "CHOP and Penn have systematically invested in integrated care programs for genetic epilepsies and neurodevelopmental disorders.
  • View original content to download multimedia: https://www.prnewswire.com/news-releases/25-million-gift-to-penn-medicin...
    SOURCE Children's Hospital of Philadelphia; University of Pennsylvania School of Medicine

AC Immune Awarded New Grants from MJFF and Target ALS Supporting Programs Targeting TDP-43

Retrieved on: 
Tuesday, February 7, 2023
Amyotrophic lateral sclerosis, Barrow Neurological Institute, Target, Alpha-synuclein, Disease, Research, Diagnosis, Neurodegeneration, Pathology, The Michael J. Fox Foundation, NDD, MJFF, Frontotemporal lobar degeneration, Precision medicine, International Center, PET, FTLD, Patient, Drug discovery, ALS, Kansas City University, National Center for Research Resources, Senior, AC, TAR, Engineering, Pharmaceutical industry, Medical imaging, Biotechnology

Fox Foundation for Parkinson’s Research (MJFF) and Target ALS Foundation (Target ALS) supporting research programs to enable diagnosis of TDP-43 (TAR DNA-binding protein 43), recognized as an important target in multiple neurodegenerative diseases (NDDs) such as amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) and as a prominent co-pathology in Alzheimer’s and Parkinson’s diseases.

Key Points: 
  • Fox Foundation for Parkinson’s Research (MJFF) and Target ALS Foundation (Target ALS) supporting research programs to enable diagnosis of TDP-43 (TAR DNA-binding protein 43), recognized as an important target in multiple neurodegenerative diseases (NDDs) such as amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) and as a prominent co-pathology in Alzheimer’s and Parkinson’s diseases.
  • Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “It is an honor to have the support of MJFF and Target ALS, two leading international organizations that recognize the pressing need for diagnostics to detect pathological TDP-43.
  • AC Immune and its collaborators recently demonstrated their expertise in developing cutting-edge PET imaging agents by providing the first images of alpha-synuclein.
  • The development of a TDP-43 specific biofluid-based diagnostic test has the potential to more rapidly enable confirmed early diagnosis.”
    The MJFF and Target ALS grants collectively provide more than USD 500,000 in additional non-dilutive capital to support the advancement of diagnostic programs targeting TDP-43.

STALICLA signs exclusive in-licensing agreement for late-stage clinical neuropsychiatric and neurodevelopmental disorder treatment

Retrieved on: 
Monday, January 9, 2023
Substance use disorder, Novartis, Safety, Patient, CUD, DEPI, NDD, Mavoglurant, Pharmaceutical industry

Geneva, SWITZERLAND – 9 January 2023, STALICLA SA, a clinical-stage biotech company advancing the first precision neurobiology platform for patients with neurodevelopmental disorders, today announced that it has entered into an exclusive in-licensing agreement with Novartis to develop mavoglurant as a treatment for substance-use disorder and neurodevelopmental disorders (NDDs).

Key Points: 
  • Geneva, SWITZERLAND – 9 January 2023, STALICLA SA, a clinical-stage biotech company advancing the first precision neurobiology platform for patients with neurodevelopmental disorders, today announced that it has entered into an exclusive in-licensing agreement with Novartis to develop mavoglurant as a treatment for substance-use disorder and neurodevelopmental disorders (NDDs).
  • Mavoglurant has previously been tested in more than 1,800 patients and demonstrated a good safety and tolerability profile.
  • Following highly promising results from a Phase 2 study conducted with mavoglurant in cocaine-use disorder (CUD; NCT03242928), STALICLA is now preparing to advance mavoglurant into Phase 3 development for the treatment of CUD.
  • In parallel, STALICLA will leverage its precision neurobiology drug development platform (DEPI), which has demonstrated proof-of-concept in other neurodevelopmental programs, to detect subgroups of high responder patients with rare and common neurodevelopmental disorders where mavoglurant can be an effective treatment, as guided by earlier clinical studies.