Cerebrospinal fluid

Nurix Therapeutics Reports First Clinical Evidence of CNS Activity of NX-5948, a Brain-Penetrant, Orally Available, BTK Degrader in Development for B Cell Malignancies

Retrieved on: 
Tuesday, April 9, 2024

SAN FRANCISCO, April 09, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced the presentation of the first findings of clinical responses in the brain for NX-5948, an orally available, selective degrader of Bruton’s tyrosine kinase (BTK). The presentation included case studies for two patients, one with CLL with CNS involvement and the other with PCNSL, each demonstrating clinically meaningful responses. The presentation also provided evidence of measurable drug levels in the CNS of multiple patients in the ongoing Phase 1 trial who had CNS tumor involvement. These data were presented by Gwenn M. Hansen, Ph.D., chief scientific officer of Nurix, as part of the Major Symposium session Molecular Glues, PROTACs, and Next-Gen Degraders: Discovery and Early Preclinical Advances at the AACR 2024 Annual Meeting, which is being held from April 5-10, 2024, in San Diego, CA.

Key Points: 
  • The presentation included case studies for two patients, one with CLL with CNS involvement and the other with PCNSL, each demonstrating clinically meaningful responses.
  • The presentation also provided evidence of measurable drug levels in the CNS of multiple patients in the ongoing Phase 1 trial who had CNS tumor involvement.
  • “These data are the first demonstration of clinical activity in the brain of a targeted protein degrader, opening the door for new therapeutic strategies to treat leukemias and lymphomas with CNS involvement,” said Dr. Hansen.
  • “The CLL patient with CNS involvement showed an impressive durable response with NX-5948 as single agent therapy in this setting.

Brain & Behavior Research Foundation Awards Distinguished Investigator Grants Valued at $1 Million to 10 Scientists Pursuing Innovative Mental Health Research

Retrieved on: 
Thursday, April 4, 2024

New York, April 04, 2024 (GLOBE NEWSWIRE) -- The Brain & Behavior Research Foundation (BBRF) today announced it is awarding Distinguished Investigator Grants valued at $1 million to 10 senior-level scientists who are conducting innovative projects in neurobiological and behavioral research.

Key Points: 
  • New York, April 04, 2024 (GLOBE NEWSWIRE) -- The Brain & Behavior Research Foundation (BBRF) today announced it is awarding Distinguished Investigator Grants valued at $1 million to 10 senior-level scientists who are conducting innovative projects in neurobiological and behavioral research.
  • By funding transformative research focused on new ways to prevent, diagnose, and treat psychiatric disorders, our Distinguished Investigator Grants are encouraging established scientists to continue advancing our understanding of mental illness and disorders of brain and behavior,” says Jeffrey Borenstein, M.D., President and CEO of the Brain & Behavior Research Foundation.
  • The ground-breaking work of the Distinguished Investigator Grant recipients will bring hope and healing to people and families impacted by mental illness.
  • We applaud these scientists for their extraordinary dedication, innovation, and leadership.”
    Recipients of the Distinguished Investigator Grants are full professors at research institutions in the United States and abroad.

ProMIS Neurosciences Announces Full Year 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, April 1, 2024

This is a very exciting opportunity for both ProMIS and the AD patients we aim to serve.

Key Points: 
  • This is a very exciting opportunity for both ProMIS and the AD patients we aim to serve.
  • ProMIS dosed the first participants in a first-in-human Phase 1a clinical trial of PMN310 as a potential treatment for AD in November 2023 (Study NCT06105528 ).
  • Using a proprietary computational platform, ProMIS identified potential conformational epitopes (misfolded portions) unique to toxic alpha-synuclein involved in synucleinopathies.
  • Cash and cash equivalents were $12.6 million as of December 31, 2023, compared to $5.9 million as of December 31, 2022.

Annovis Announces Publication That Supports Understanding of Buntanetap’s Mechanism of Action in Humans

Retrieved on: 
Monday, April 1, 2024

MALVERN, Pa., April 01, 2024 (GLOBE NEWSWIRE) -- Annovis Bio, Inc. (NYSE: ANVS), a clinical-stage drug platform company developing novel therapies for neurodegenerative diseases, today announced the publication of new data from an earlier study supporting buntanetap as a translational inhibitor of amyloid precursor protein (APP) in patients with early Alzheimer’s Disease (AD).

Key Points: 
  • Buntanetap is an oral molecule that selectively binds to an iron-responsive element in the mRNA of APP and other neurotoxic proteins and inhibits their translation.
  • Through this mechanism, buntanetap was shown to decrease the production of amyloid beta (Aβ), a key hallmark in AD.
  • Here, SILK was used to measure the kinetics of APP in early AD patients and to quantify Aβ40 in CSF.
  • Notably, the analysis of adverse events demonstrated no dose-dependent effect of buntanetap compared to placebo, affirming the drug’s safety and tolerability.

Inhibikase Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Activity

Retrieved on: 
Wednesday, March 27, 2024

The Meeting Minutes confirmed that the 505(b)(2) pathway appears to be appropriate for approval of IkT-001Pro.

Key Points: 
  • The Meeting Minutes confirmed that the 505(b)(2) pathway appears to be appropriate for approval of IkT-001Pro.
  • Inhibikase will host a conference call and webcast to discuss its full-year 2023 financial results and business highlights tomorrow, March 28, 2024, at 8:00am ET.
  • The conference call can be accessed by dialing 1-877-407-0789 (United States) or 1-201-689-8562 (International) and referencing Inhibikase Therapeutics.
  • After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call.

BrainStorm Cell Therapeutics Announces Agreement with FDA on a Special Protocol Assessment (SPA) for Phase 3b Trial in ALS

Retrieved on: 
Tuesday, April 9, 2024

NEW YORK, April 9, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it received written agreement from the U.S. Food and Drug Administration (FDA), under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn® in amyotrophic lateral sclerosis (ALS). 

Key Points: 
  • (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it received written agreement from the U.S. Food and Drug Administration (FDA), under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn® in amyotrophic lateral sclerosis (ALS).
  • The SPA agreement with the FDA validates the clinical trial protocol and statistical analysis of the planned Phase 3b trial of NurOwn, demonstrating their adequacy for addressing objectives that support a future BLA (Biologics License Application) in ALS.
  • We appreciate the Agency's engagement and guidance during the SPA process and look forward to moving forward with the study."
  • The Phase 3b trial (Study BCT-006-US) will be a two-part, multicenter, study designed to assess the efficacy and safety of NurOwn in patients with ALS.

Study Finds C₂N Diagnostics’ PrecivityAD® Blood Test Provides Opportunities for Robust Cost Savings in the Evaluation of Patients with Cognitive Impairment

Retrieved on: 
Monday, April 1, 2024

The test helps healthcare providers better determine the presence of amyloid plaques in the brain, a hallmark sign of Alzheimer’s disease.

Key Points: 
  • The test helps healthcare providers better determine the presence of amyloid plaques in the brain, a hallmark sign of Alzheimer’s disease.
  • The new research, “ Use of a Blood Biomarker Test Improves Economic Utility in the Evaluation of Older Patients Presenting with Cognitive Impairment ,” used a budget impact model in a hypothetical 1 million-member health plan to assess the economic utility of the PrecivityAD blood test.
  • Overall, the PrecivityAD blood test was associated with a cost savings of $643 per Alzheimer’s disease case (9% cost savings) identified.
  • We believe the use of the PrecivityAD blood test in the clinical care pathway may prevent unnecessary testing, provide cost savings and reduce the burden on both patients and health plans.”

New Findings From Sunbird Bio Reveal Significant Potential of Specific Tau Proteins to Serve as Blood-Based Biomarker for Alzheimer’s Disease

Retrieved on: 
Monday, March 25, 2024

“These results validate the potential of EV-bound tau proteins to serve as reliable blood-based biomarkers in accurately detecting Alzheimer’s disease,” said John McDonough, executive chair and CEO of Sunbird Bio.

Key Points: 
  • “These results validate the potential of EV-bound tau proteins to serve as reliable blood-based biomarkers in accurately detecting Alzheimer’s disease,” said John McDonough, executive chair and CEO of Sunbird Bio.
  • “This confirmed correlation between EV-bound tau in the blood and tau tangles in the brain combined with the early validation of our new tau assays is incredibly exciting.
  • The Sunbird Bio study being shared at Tau2024 evaluated the potential of EV-bound tau proteins in the blood as a method of directly detecting the tau proteins that aggregate in the brain.
  • They then used Sunbird Bio’s new proprietary tau assays to distinguish between EV-bound and unbound soluble forms of tau in plasma.

QuantalX Secures Spot in FDA's Prestigious Total Life Cycle Advisory Program (TAP)

Retrieved on: 
Thursday, April 4, 2024

NEW YORK, April 4, 2024 /PRNewswire/ -- QuantalX Neuroscience, a pioneering healthcare company dedicated to early detection of brain disorders using the Delphi-MD medical device, announced today its selection to participate in the highly prestigious Total Life Cycle Advisory Program (TAP) initiated by the U.S. Food and Drug Administration (FDA). The program will focus on advancing Delphi-MD's unique Direct Electrophysiology Imaging technology, enabling the early detection of Normal Pressure Hydrocephalus (NPH) disease, with high accuracy rates, and the prediction of patients' response to ventriculoperitoneal shunting (VPS). The TAP program brings together the FDA, major health insurers, healthcare organizations, and industry leaders in a collaborative effort to construct a comprehensive strategy for technologies that can benefit the general population. This strategic framework spans crucial elements, such as value creation in the U.S. market, meticulous preparation for regulatory approval, meaningful engagement with insurers, and the establishment of a robust market presence with customers.

Key Points: 
  • NEW YORK, April 4, 2024 /PRNewswire/ -- QuantalX Neuroscience, a pioneering healthcare company dedicated to early detection of brain disorders using the Delphi-MD medical device, announced today its selection to participate in the highly prestigious Total Life Cycle Advisory Program (TAP) initiated by the U.S. Food and Drug Administration (FDA).
  • "The acceptance into the Total Life Cycle Advisory Program is a truly prestigious and significant opportunity for QuantalX," remarked Iftach Dolev.
  • The Total Life Cycle Advisory Program aims to guide innovative technologies through the entire product life cycle, ensuring regulatory compliance, market adoption, and enhanced positive patient impact.
  • The valuable experience and insights within this program are expected to accelerate QuantalX's market entry and establish a solid foundation for future growth.

San Diego Alzheimer's Partners Come Together

Retrieved on: 
Monday, March 18, 2024

SAN DIEGO, March 18, 2024 /PRNewswire/ -- The Alzheimer's Therapeutic Research Institute (ATRI) and over a half-dozen of San Diego's Alzheimer's partners invite the community to a free event on March 23, 2024. A Day of SCervice, in support of accelerating Alzheimer's disease (AD) research through blood testing. Though the entire San Diego community is invited to visit ATRI and learn about new advances in AD research, those attending who are over the age of 55 and interested in participating in AD clinical trials can consent and, if eligible, have their blood drawn that day.

Key Points: 
  • SAN DIEGO, March 18, 2024 /PRNewswire/ -- The Alzheimer's Therapeutic Research Institute (ATRI) and over a half-dozen of San Diego's Alzheimer's partners invite the community to a free event on March 23, 2024.
  • Local CBS 8 news anchor and Alzheimer's San Diego Board Chair, Carlo Cecchetto, will moderate the morning panel.
  • "March 23 is also Day of SCervice, and the San Diego Trojan Family will be making a difference," said Shelley Moore, President-Elect of the San Diego Trojan League of San Diego County.
  • These clinical trials are vital to the work we do," said Janet Hamada-Kelley, Executive Director of the Alzheimer's Association San Diego & Imperial County an one of the event partners.