ALS

Intravacc and DZNE awarded EU funding to develop vaccine against genetic ALS variant

Retrieved on: 
Wednesday, July 6, 2022 - 7:00am

The project aims to develop the vaccine candidate identified at DZNE to the point where it can be clinically tested in humans.

Key Points: 
  • The project aims to develop the vaccine candidate identified at DZNE to the point where it can be clinically tested in humans.
  • About 5-10% of all ALS cases are caused by a mutation in the C9orf72 gene, making it the most common genetic ALS variant.
  • DZNE developed an experimental vaccine that instructs the immune system to produce antibodies against these harmful poly-GA molecules.
  • Clinical trials for C9orf72 ALS, which is the most common genetic variant of ALS, are expected to commence in 2025.

Intravacc and DZNE awarded EU funding to develop vaccine against genetic ALS variant

Retrieved on: 
Wednesday, July 6, 2022 - 7:00am

The project aims to develop the vaccine candidate identified at DZNE to the point where it can be clinically tested in humans.

Key Points: 
  • The project aims to develop the vaccine candidate identified at DZNE to the point where it can be clinically tested in humans.
  • About 5-10% of all ALS cases are caused by a mutation in the C9orf72 gene, making it the most common genetic ALS variant.
  • DZNE developed an experimental vaccine that instructs the immune system to produce antibodies against these harmful poly-GA molecules.
  • Clinical trials for C9orf72 ALS, which is the most common genetic variant of ALS, are expected to commence in 2025.

Cytokinetics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4)

Retrieved on: 
Tuesday, July 5, 2022 - 9:00pm

Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which muscle performance is compromised.

Key Points: 
  • Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which muscle performance is compromised.
  • Cytokinetics continues its over 20-year history of pioneering innovation in muscle biology and related pharmacology focused to diseases of muscle dysfunction and conditions of muscle weakness.
  • Examples of such statements include, but are not limited to, statements relating toCytokinetics'and its partners' research and development activities of Cytokinetics product candidates.
  • CYTOKINETICS and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries.

Amylyx Pharmaceuticals Announces FDA Plan to Reconvene Advisory Committee to Review AMX0035 NDA for the Treatment of ALS on September 7, 2022

Retrieved on: 
Tuesday, July 5, 2022 - 2:00pm

The FDA will formally announce the scheduling of the planned PCNSDAC meeting in the Federal Register.

Key Points: 
  • The FDA will formally announce the scheduling of the planned PCNSDAC meeting in the Federal Register.
  • Discussions will focus on the additional analyses of data from the Companys clinical studies that were determined by the FDA to constitute a major amendment to the NDA.
  • The PCNSDAC previously met on March 30, 2022, to discuss the NDA for AMX0035 for the treatment of ALS.
  • We remain engaged with the FDA to advance AMX0035 through the review process as efficiently as possible, said Tammy Sarnelli, Global Head of Regulatory Affairs of Amylyx.

ReviR Therapeutics Appoints Paul August, Ph.D., as Chief Scientific Officer

Retrieved on: 
Friday, July 1, 2022 - 3:00pm

SOUTH SAN FRANCISCO, Calif., July 1, 2022 /PRNewswire/ --ReviR Therapeutics, a biotechnology company focused on developing RNA-targeting small molecule drugs, announced the appointment of Paul August, Ph.D., as the Chief Scientific Officer.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., July 1, 2022 /PRNewswire/ --ReviR Therapeutics, a biotechnology company focused on developing RNA-targeting small molecule drugs, announced the appointment of Paul August, Ph.D., as the Chief Scientific Officer.
  • "We are thrilled to welcome Dr. August to our team," said Peng Yue, Ph.D., Co-Founder, and Chief Executive Officer of ReviR Therapeutics.
  • Dr. August brings to ReviR Therapeutics more than two decades of senior experience in pharmaceutical research and development.
  • ReviR Therapeutics is a research and development company aiming to treat human diseases by harnessing advanced AI technologies and RNA biology.

Patients Who Use Medicines Made from Plasma Share How Their Lives Were Saved Thanks to Plasma Donors

Retrieved on: 
Thursday, June 30, 2022 - 2:00pm

IG therapy is created from source plasma donations, meaning the community relies on healthy people to donate their plasma in order for their medicines to be made.

Key Points: 
  • IG therapy is created from source plasma donations, meaning the community relies on healthy people to donate their plasma in order for their medicines to be made.
  • For patients living with these conditions, plasma donors are everyday heroes.
  • Jessica, living with MMN, noted "[the plasma given by plasma donors] is a gift of life, literally giving me the ability to walk and use my hands.
  • If you are interested in becoming an everyday hero for the thousands of patients who rely on source plasma for their medicine, please visit https://www.donatingplasma.org/ .

MediciNova Announces Extension of BARDA Contract to Develop MN-166 (ibudilast) as a Medical Countermeasure Against Chlorine Gas-induced Lung Injury

Retrieved on: 
Thursday, June 30, 2022 - 12:00am

Department of Health and Human Services, to repurpose MN-166 (ibudilast) as a potential medical countermeasure (MCM) against chlorine gas-induced lung damage such as acute respiratory distress syndrome (ARDS) and acute lung injury (ALI).

Key Points: 
  • Department of Health and Human Services, to repurpose MN-166 (ibudilast) as a potential medical countermeasure (MCM) against chlorine gas-induced lung damage such as acute respiratory distress syndrome (ARDS) and acute lung injury (ALI).
  • MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF).
  • Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof.
  • MediciNova disclaims any intent or obligation to revise or update these forward-looking statements.

The LGMD Community Celebrates Adoption of ICD-10 Diagnostic Codes for LGMD

Retrieved on: 
Wednesday, June 29, 2022 - 10:52pm

The implementation of these ICD-10 codes represents a culmination of two-years of collaborative efforts within the LGMD patient, clinical, and research communities.

Key Points: 
  • The implementation of these ICD-10 codes represents a culmination of two-years of collaborative efforts within the LGMD patient, clinical, and research communities.
  • These diagnostic codes will be fully implemented and ready for use starting this October.
  • This significant advance could well shorten the diagnostic odyssey for individuals with LGMD, facilitate the targeted care the community needs, and accelerate surveillance and epidemiological research.
  • This milestone is the culmination of over two years of collaborative efforts between LGMD advocacy organizations and leading LGMD clinicians and researchers.

Mitsubishi Tanabe Pharma America Announces Long-Term Findings from Post-Marketing Safety Study of RADICAVA® (edaravone)

Retrieved on: 
Wednesday, June 29, 2022 - 2:00pm

JERSEY CITY, N.J., June 29, 2022 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced findings from an analysis of post-marketing safety data evaluating RADICAVA® (edaravone) in a real-world setting during the first three years of availability in the United States (U.S.) for the treatment of amyotrophic lateral sclerosis (ALS). RADICAVA maintained a similar safety profile as seen in clinical trials, with no new safety signals or inconsistencies with the clinical trials identified over the treatment period. Study findings were published in a paper entitled, "Analysis of the U.S. Safety Data for Edaravone (Radicava®) From the Third Year After Launch," in Drugs in R&D.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) approved RADICAVA (edaravone) on May 5, 2017 as a treatment for amyotrophic lateral sclerosis (ALS).2 Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc. (MTDA), commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc.
  • About Mitsubishi Tanabe Pharma America, Inc.
    Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation's (MTPC) 100 percent owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc.
  • The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) is located in Jersey City, New Jersey.
  • MTDA is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation's 100 percent-owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. For more information, please visit https://mt-pharma-development-america.com/ .

Initial Assay Results from 2022 Drill Program at Calico Silver Project Show Broad, High-Grade Silver and Expanded Gold Horizon

Retrieved on: 
Wednesday, June 29, 2022 - 12:00pm

Representative chip samples were also collected for logging purposes (lithology, alteration, mineralization), detailed photography and analysis by portable X-Ray Fluorescence.

Key Points: 
  • Representative chip samples were also collected for logging purposes (lithology, alteration, mineralization), detailed photography and analysis by portable X-Ray Fluorescence.
  • Splits of prepared pulps are securely shipped from ALS Reno to ALS Vancouver, British Columbia for further analysis.
  • Gold was analyzed by fire assay with atomic absorption finish (method Au-AA26) with a reportable range of 0.01-100 ppm Au.
  • All analyses were completed at ALS Vancouver except for gold by fire assay, which was completed at ALS Reno.