Ataxia–telangiectasia

Newly Published Phase II Clinical Study Demonstrates that Supplementation with Niagen®, Patented Nicotinamide Riboside (NR), Elevates NAD+ Up to Fourfold, Improving Motor Coordination and Eye Movement in Ataxia Telangiectasia (AT) Patients

Retrieved on: 
Wednesday, November 15, 2023
Science, Other Science, Research, Public Relations, Investor Relations, General Health, Health, Communications, Clinical Trials, De Veenhuis, Movement disorder, DNA, CERP, Oculomotor apraxia, ChromaDex, Liver, Clinical trial, Cerebellar degeneration, Patient, Multimedia, Nicotinamide adenine dinucleotide, Immunodeficiency, Movement, DNA repair, Cerebellar ataxia, Cognition, Eye, University, Trial of the century, University of Oslo, Biomarker, NAD, Medical device, Ataxia–telangiectasia

The promising results demonstrate that long-term supplementation with Niagen NR effectively increased whole blood NAD+ levels up to fourfold, improved coordination, and enhanced eye movement while maintaining biomarkers of stable liver and kidney function in Ataxia Telangiectasia (AT) patients.

Key Points: 
  • The promising results demonstrate that long-term supplementation with Niagen NR effectively increased whole blood NAD+ levels up to fourfold, improved coordination, and enhanced eye movement while maintaining biomarkers of stable liver and kidney function in Ataxia Telangiectasia (AT) patients.
  • AT is a rare, inherited neurodegenerative disorder characterized by premature aging, cerebellar degeneration, immunodeficiency, and cancer predisposition.
  • View the full release here: https://www.businesswire.com/news/home/20231115900463/en/
    Newly published phase II clinical study demonstrates that supplementation with Niagen, patented nicotinamide riboside (NR), elevates NAD+ up to fourfold, improving motor coordination and eye movement in Ataxia Telangiectasia (AT) patients (Graphic: Business Wire)
    Building on a growing body of research, this is the second published study ( Veenhuis et al.
  • This growing body of research is promising as it demonstrates that NR supplementation may be a potential therapeutic strategy for AT patients.”

Global ATR Protein Inhibitors Market Set to Revolutionize Cancer Treatment and Expand into Neurodegenerative and Infectious Diseases - ResearchAndMarkets.com

Retrieved on: 
Wednesday, November 8, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, BAY, Alzheimer's disease, AstraZeneca, Cholangiocarcinoma, Genetic testing, Ovarian cancer, Therapy, Parkinson's disease, Breast, Emotional dysregulation, Research, Virus, Clinical trial, Berzosertib, COVID-19, Viral, Ataxia telangiectasia and Rad3 related, Radiation, HIV-1, DNA repair, Vertex Pharmaceuticals, Bone marrow, Cancer, Investment, ATR, Patient, Ataxia–telangiectasia, DNA, Pharmaceutical industry, Vaccine, Bayer, Merck

A promising frontier in cancer pharmaceutical research, the inhibition of Ataxia telangiectasia and Rad3 related kinase (ATR kinase) is poised to revolutionize cancer treatment and extend its reach to neurodegenerative and infectious diseases.

Key Points: 
  • A promising frontier in cancer pharmaceutical research, the inhibition of Ataxia telangiectasia and Rad3 related kinase (ATR kinase) is poised to revolutionize cancer treatment and extend its reach to neurodegenerative and infectious diseases.
  • Pipeline Analysis: The report offers insights into the global ATR protein inhibitors clinical pipeline, categorized by company, indication, and phase.
  • Development Trends: ATR inhibitors are expanding their scope to include neurodegenerative diseases and viral infections, opening new avenues for treatment.
  • This innovative approach to targeting DNA damage response pathways has the potential to revolutionize cancer treatment and extend its reach to neurodegenerative and infectious diseases.

Aprea Therapeutics to Host a Key Opinion Leader (KOL) Event on its Synthetic Lethality (SL) and DNA Damage Response (DDR) Pathways

Retrieved on: 
Tuesday, October 24, 2023
Aprea-Cuccaro clan, IND, DDR, APRE, WEE1, MD Anderson Cancer Center, Ataxia–telangiectasia, University of Pennsylvania, News, KOL, Perelman School of Medicine at the University of Pennsylvania, University, Therapy, ATR, Patient, Pharmaceutical industry

DOYLESTOWN, Pa., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE) (“Aprea”, or the “Company”), a clinical stage biopharmaceutical company focused on precision oncology through synthetic lethality, today announced it will host a KOL event on Tuesday, October 31, 2023 at 11:00 am ET.

Key Points: 
  • DOYLESTOWN, Pa., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE) (“Aprea”, or the “Company”), a clinical stage biopharmaceutical company focused on precision oncology through synthetic lethality, today announced it will host a KOL event on Tuesday, October 31, 2023 at 11:00 am ET.
  • The Aprea Therapeutics team will discuss the Phase 1/2a study evaluating its lead program of a highly potent and selective macrocyclic inhibitor of ataxia telangiectasia and Rad3-related (ATR), ATRN-119, in patients with advanced solid tumors having mutations in defined DDR genes.
  • The session will be available for replay on the News and Events portion of the Investor Relations section of the company’s website.
  • To register for the event, please click here .

Artios Presents Positive Initial Phase 1/1b Clinical Monotherapy Data for ATR Inhibitor ART0380 in Advanced Solid Tumors at the European Society of Medical Oncology Congress 2023

Retrieved on: 
Monday, October 23, 2023
Irinotecan, Phase II, DDR, ATM, Ovarian cancer, ESMO, MD, Ataxia–telangiectasia, Drug development, Pharmacokinetics, PK, Biology, Cancer, DNA repair, Platinum, University, Safety, European Society for Medical Oncology, ATR, MD Anderson Cancer Center, Patient, Toxicity, DNA, Medical imaging, Pharmaceutical industry, Casting Society of America

CAMBRIDGE, United Kingdom and NEW YORK, Oct. 23, 2023 (GLOBE NEWSWIRE) -- Artios Pharma Limited (Artios), a clinical-stage biotech company led by pioneers of DNA damage response (“DDR”) drug development, presented promising monotherapy data from the Phase 1/1b portion of the ongoing Phase 1/2a study (NCT04657068) of its ataxia telangiectasia and Rad-3 related (“ATR”) kinase inhibitor ART0380 in advanced or metastatic solid tumors as part of a poster presentation at the European Society of Medical Oncology Congress (ESMO) 2023. ART0380 is a clinically advanced, oral, highly potent, and selective ATR inhibitor with best-in-class potential. The Phase 1/1b portion of the trial presented at ESMO assessed the safety and tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of ART0380 monotherapy in patients with advanced or metastatic solid tumors. The patient population was enriched for cancers harboring DNA damage response deficiencies as identified by the Artios DcoDeR platform (n = 49).

Key Points: 
  • ART0380 is a clinically advanced, oral, highly potent, and selective ATR inhibitor with best-in-class potential.
  • The Phase 1/1b portion of the trial presented at ESMO assessed the safety and tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of ART0380 monotherapy in patients with advanced or metastatic solid tumors.
  • The patient population was enriched for cancers harboring DNA damage response deficiencies as identified by the Artios DcoDeR platform (n = 49).
  • Our next-generation ATR inhibitor ART0380 has demonstrated highly encouraging molecular and clinical responses as a monotherapy particularly in patients with DDR deficiency and high replication stress alterations.

Artios to Present Initial Phase 1 Clinical Monotherapy Data for ATR Inhibitor ART0380 in Advanced Solid Tumors at the European Society of Medical Oncology Congress 2023

Retrieved on: 
Thursday, October 5, 2023
Ataxia–telangiectasia, Cancer, University, Pharmacokinetics, Drug development, DNA, Safety, ESMO, ATR, European Society for Medical Oncology, Patient, DDR, Combination therapy, Pharmaceutical industry

CAMBRIDGE, United Kingdom and NEW YORK, Oct. 05, 2023 (GLOBE NEWSWIRE) -- Artios Pharma Limited (Artios), a clinical-stage biotech company led by pioneers of DNA damage response (“DDR”) drug development, announces the Company will unveil data from the initial Phase 1 study of its ataxia telangiectasia and Rad-3 related (“ATR”) kinase inhibitor ART0380 as part of a poster presentation at the European Society of Medical Oncology Congress (ESMO) 2023 taking place October 20 to 24, 2023 in Madrid, Spain.

Key Points: 
  • ART0380 is a highly competitive, wholly owned ATR inhibitor with broad clinical application alone or in combination for DDR deficient and high replication stress tumors
    CAMBRIDGE, United Kingdom and NEW YORK, Oct. 05, 2023 (GLOBE NEWSWIRE) -- Artios Pharma Limited (Artios), a clinical-stage biotech company led by pioneers of DNA damage response (“DDR”) drug development, announces the Company will unveil data from the initial Phase 1 study of its ataxia telangiectasia and Rad-3 related (“ATR”) kinase inhibitor ART0380 as part of a poster presentation at the European Society of Medical Oncology Congress (ESMO) 2023 taking place October 20 to 24, 2023 in Madrid, Spain.
  • ART0380 is a competitive, oral, highly potent, and selective ATR inhibitor undergoing clinical evaluation as monotherapy in patients with solid tumors as well as in combination with chemotherapies in patients with molecularly selected cancers.
  • Monotherapy data presented will include safety and tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy.
  • First results from the phase I trial of the ATR inhibitor, ART0380, in advanced solid tumors
    Presenter: Kathleen Moore, Stephenson Cancer Center at the University of Oklahoma, Oklahoma City, United States of America
    For more information about Artios Pharma Ltd., please contact:
    Media & Investor Relations Contact:

Ataxia Telangiectasia (AT) Market and Epidemiology Landscape 2023-2032: Unmet Medical Needs, Reimbursement Strategies, Competitive Intelligence Analysis - ResearchAndMarkets.com

Retrieved on: 
Friday, August 11, 2023
Health, Pharmaceutical, KOL, PESTLE, AT, Epidemiology, Research, Forecasting, SWOT, Porter's five forces analysis, United Kingdom, Growth, Nursing, Pharmaceutical industry, Retail, Ataxia–telangiectasia

The "Ataxia Telangiectasia (AT) - Market Insight, Epidemiology and Market Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Ataxia Telangiectasia (AT) - Market Insight, Epidemiology and Market Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.
  • Market Share Analysis: The report presents market share insights of individual therapies, enabling stakeholders to strategize and make informed decisions based on the competitive landscape.
  • Unmet Medical Needs: By identifying the unmet medical needs in the Ataxia Telangiectasia (AT) market, the report helps pharmaceutical companies identify lucrative opportunities for research and development, with the ultimate goal of addressing critical patient needs.
  • Competitive Intelligence Analysis: Utilizing advanced tools like SWOT analysis, PESTLE analysis, and Porter's five forces, the report assesses the competitive landscape, enabling companies to develop robust strategies for market success.

New Journal of Pharmaceutical Analysis Articles Showcase Promising Therapeutic Candidates for Treating Cancer and Hearing Loss

Retrieved on: 
Wednesday, August 9, 2023
Neuron, DNA, Non-small-cell lung cancer, Hearing loss, JPA, XPO1, Cancer, Quality of life, Research, NSCLC, Cisplatin, Liver, DNA repair, HIV disease progression rates, Patient, Apoptosis, Cell cycle, METTL3, Dick's Picks Volume 13, Ataxia–telangiectasia, Leukemia, Kinetics, ATR, PRMT5, Pharmaceutical industry

XI'AN, China, Aug. 9, 2023 /PRNewswire/ -- Although cancer treatment has advanced significantly over the last decade, some fairly common cancers remain extremely difficult to treat. Besides, some chemotherapeutics or anti-cancer drugs can have serious, irreversible side-effects. The latest issue of the JPA features three articles that highlight novel treatment strategies based on cutting-edge research on cancer and chemotherapeutic drug-induced adverse events.

Key Points: 
  • The latest issue of the JPA features three articles that highlight novel treatment strategies based on cutting-edge research on cancer and chemotherapeutic drug-induced adverse events.
  • The first study reports a new approach to prevent hearing loss induced by the widely used chemotherapeutic drug "cisplatin".
  • The article was available online in April 2023 and published in Volume 13, Issue 6 in June 2023 .
  • A better understanding of the role of PRMT5 in hearing loss could help scientists develop effective hearing protection drugs and prevent this unwanted side-effect.

Acasti Pharma Reports Third Quarter 2023 Operational Results

Retrieved on: 
Tuesday, February 14, 2023
Bank statement, Subarachnoid hemorrhage, Toxicity, Caregiver, Brain, Dizziness, Nimodipine, NDA, AUC, Bupivacaine, Trial of the century, OS, Human, Blood, Postherpetic neuralgia, PK, Cmax, TSX, Hypoesthesia, Aes, Investment, SAH, Safety, Literature, Acquisition, Tmax, Anesthesiology, Patient, PHN, Telephone, AE, Skin, Food, Skull, Research, Government, Aneurysm, Risk, IM, SubArachnoid Space, Betamethasone, Plasma, Tongue, FDA, Nausea, A-T, Food and Drug Administration Amendments Act of 2007, Calendar, Bleeding, Type, Ataxia–telangiectasia, ACST, Pharmacokinetics, Rupture, Headache, Pharmaceutical industry, Vaccine, Cryptocurrency

LAVAL, Québec, Feb. 14, 2023 (GLOBE NEWSWIRE) -- Acasti Pharma Inc. (“Acasti” or the “Company”) (Nasdaq: ACST and TSX-V: ACST), a late-stage, specialty pharma company advancing three clinical stage drug candidates addressing rare and orphan diseases, today announced financial and operational results for the third quarter ended December 31, 2022

Key Points: 
  • These positive results allow us to advance both programs to the next stage of clinical development in 2023.
  • We expect 2023 to be very exciting for Acasti with two of our drug candidates ready to enter Phase 3.
  • Topline results from this study were reported on December 23, 2022, and the results met all primary outcome measures.
  • Acasti will host a conference call on Tuesday, February 14, 2023, at 1:00 PM Eastern Time to discuss the Company’s corporate progress and other developments, as well as financial results for its quarter ended December 31, 2022.

Adrestia Therapeutics Partners with Leading Patient Advocacy Groups to Form Ataxia Telangiectasia Research Consortium

Retrieved on: 
Wednesday, February 8, 2023
Health, Medical Devices, Genetics, Research, Science, Pharmaceutical, Biotechnology, DNA, Biology, Infectious Diseases Society of America, DSM-IV codes, American Cancer Society Cancer Action Network, Diabetes, Therapy, Disease, Trustee, DNA repair, A-T, AT, Conditional sentence, Cardiovascular disease, Ataxia–telangiectasia, Genome, Pharmaceutical industry, Vaccine

Adrestia Therapeutics , a leader in synthetic rescue therapies for genetic diseases, today announced it has co-founded a research consortium to find new treatments for ataxia telangiectasia (AT), a fatal, inherited, progressive neurodegenerative disorder that is typically diagnosed in young children.

Key Points: 
  • Adrestia Therapeutics , a leader in synthetic rescue therapies for genetic diseases, today announced it has co-founded a research consortium to find new treatments for ataxia telangiectasia (AT), a fatal, inherited, progressive neurodegenerative disorder that is typically diagnosed in young children.
  • The consortium’s other founding members include the A-T Children’s Project, the AT Society and Action for AT.
  • Adrestia is applying its synthetic rescue technology to systematically mine the human genome for novel targets which could be used as the basis for therapies for AT and related diseases.
  • Our ultimate goal is to develop effective medicines which rebalance the underlying biology, unlocking new ways of treating intractable genetic diseases.

Artios Announces Initiation of Phase 2 Randomized Trial for ATR Inhibitor ART0380 plus Gemcitabine in Patients with Platinum Resistant Ovarian Cancer

Retrieved on: 
Thursday, February 9, 2023
DNA, MD Anderson Cancer Center, Safety, MD, DNA repair, DDR, Cancer, GEICO, Pharmacokinetics, Irinotecan, Ataxia–telangiectasia, Therapy, University, Founding, Patient, Ovarian cancer, ATR, Neoplasm, Doctor of Philosophy, RECIST, Platinum, Pharmaceutical industry, Casting Society of America

The recommended Phase 2 dose of ART0380 monotherapy has been defined for both intermittent and continuous daily dosing schedules together with the regimen of ART0380 in combination with gemcitabine.

Key Points: 
  • The recommended Phase 2 dose of ART0380 monotherapy has been defined for both intermittent and continuous daily dosing schedules together with the regimen of ART0380 in combination with gemcitabine.
  • Clinically active RECIST confirmed responses occurred across doses and in tumors harboring DDR deficiencies as predicted through Artios’ DcoDeR platform.
  • The favorable pharmacokinetic profile enabling combination with DNA damaging agents offers the potential of a new treatment option for patients with platinum resistant ovarian cancer.
  • ART0380 was originally in-licensed by Artios from The University of Texas MD Anderson Cancer Center and ShangPharma Innovation in 2019.