Neurolixis

Global charities join forces to drive forward new drug for Parkinson's

Retrieved on: 
Monday, November 23, 2020

This drug has shown potential in the lab for reducing dyskinesia a common side effect of current Parkinson's disease medications.

Key Points: 
  • This drug has shown potential in the lab for reducing dyskinesia a common side effect of current Parkinson's disease medications.
  • This trial builds on previous research supported by the two charities, which enabled Neurolixis to develop NLX-112 and complete the final lab testing.
  • Fox Foundation to drive forward a potentially life-changing new treatment for people with Parkinson's.
  • Neurolixis is developing a platform of early-stage drug candidates targeting serious CNS diseases with unmet medical needs and sizeable market opportunity.

ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Rare Pediatric Disease Designation for Trofinetide for the Treatment of Rett Syndrome

Retrieved on: 
Tuesday, March 3, 2020

ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) and Neuren Pharmaceuticals Limited (ASX: NEU) announced today that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder.

Key Points: 
  • ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) and Neuren Pharmaceuticals Limited (ASX: NEU) announced today that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to trofinetide for the treatment of Rett syndrome, a serious and rare neurological disorder.
  • Trofinetide was previously granted Fast Track Status and Orphan Drug Designation for Rett syndrome in the U.S. and Orphan Drug Designation for Rett syndrome in Europe.
  • Trofinetide has been granted Fast Track Status and Rare Pediatric Disease Designation for the treatment of Rett Syndrome in the U.S. and Orphan Drug Designation in the U.S. and Europe for both Rett syndrome and Fragile X syndrome.
  • Neuren has completed Phase 2 development of trofinetide for Rett syndrome and has completed a Phase 2 clinical trial in Fragile X syndrome.

BlackThorn Therapeutics Reports Data Supporting Phase 2 Development of KOR Antagonist, BTRX-335140, in Depression and Other CNS Disorders

Retrieved on: 
Wednesday, December 11, 2019

BlackThorn Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing targeted medicines to treat central nervous system (CNS) disorders, today announced data supporting Phase 2 development of its lead drug candidate, BTRX-335140 (BTRX-140), a selective kappa opioid receptor (KOR) antagonist.

Key Points: 
  • BlackThorn Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing targeted medicines to treat central nervous system (CNS) disorders, today announced data supporting Phase 2 development of its lead drug candidate, BTRX-335140 (BTRX-140), a selective kappa opioid receptor (KOR) antagonist.
  • These clinical symptoms commonly occur in multiple CNS disorders, but are not adequately addressed by currently available medications for many people seeking therapy.
  • Today, more people are suffering from brain disorders than ever before, but effective, therapeutic interventions for many of these disorders are still limited.
  • BlackThorn Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery and development of targeted medicines for the treatment of central nervous system (CNS) disorders.

Anavex Life Sciences Receives Rare Pediatric Disease Designation from FDA for ANAVEX®2-73 (blarcamesine) for the Treatment of Rett Syndrome

Retrieved on: 
Thursday, November 14, 2019

The RPD designation provides priority review by theFDAto encourage treatments for rare pediatric diseases.

Key Points: 
  • The RPD designation provides priority review by theFDAto encourage treatments for rare pediatric diseases.
  • We are pleased to have received the rare pediatric designation from the U.S. FDA for ANAVEX2-73 (blarcamesine) for young patients with Rett syndrome, said Christopher U. Missling, Ph.D., Chief Executive Officer of Anavex.
  • Our goal is to advance a potential treatment for Rett syndrome in order to bring medicines to patients as soon as possible.
  • Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups.

Global Central Nervous System Diseases Drug Delivery Report 2019: Profiles of 72 Companies with Their Technologies, Products and 100 Collaborations

Retrieved on: 
Monday, February 4, 2019

The "Drug Delivery in Central Nervous System Diseases - Technologies, Markets & Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Drug Delivery in Central Nervous System Diseases - Technologies, Markets & Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.
  • The delivery of drugs to central nervous system (CNS) is a challenge in the treatment of neurological disorders.
  • Profiles of 72 companies involved in drug delivery for CNS disorders are presented along with their technologies, products and 100 collaborations.
  • These include pharmaceutical companies that develop CNS drugs and biotechnology companies that provide technologies for drug delivery.