Neurological disorders

Pinteon Therapeutics Announces Positive Phase 1 Data for its Novel Tau Antibody PNT001

Retrieved on: 
Monday, June 28, 2021

Pinteon Therapeutics, a biotechnology company focused on protecting neuronal health by targeting neurotoxic forms of tau, today announced positive results from its Phase 1 study of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease.

Key Points: 
  • Pinteon Therapeutics, a biotechnology company focused on protecting neuronal health by targeting neurotoxic forms of tau, today announced positive results from its Phase 1 study of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease.
  • PNT001 is a monoclonal antibody targeting an epitope on a highly neurotoxic conformation of the tau protein called cis-pT231.
  • This conformation of tau is both acutely neurotoxic, but also contributes to the initiation and spread of tau pathology in neurodegenerative tauopathies.
  • Pinteon Therapeutics is advancing clinical studies of a novel antibody that aims to interrupt the spread of toxic tau and protect and preserve brain function in patients with neurodegenerative disease.

MTPA Announces PANTHERx® Selected as Exclusive Provider of U.S. Specialty Pharmacy Services for EXSERVAN™ (riluzole) Oral Film

Retrieved on: 
Friday, June 25, 2021

JERSEY CITY, N.J., June 25, 2021 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced PANTHERx Rare Pharmacy has been selected as the exclusive provider of specialty pharmacy services for EXSERVAN(riluzole), an oral film formulation of riluzole for the treatment of amyotrophic lateral sclerosis (ALS).

Key Points: 
  • JERSEY CITY, N.J., June 25, 2021 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced PANTHERx Rare Pharmacy has been selected as the exclusive provider of specialty pharmacy services for EXSERVAN(riluzole), an oral film formulation of riluzole for the treatment of amyotrophic lateral sclerosis (ALS).
  • Upon enrollment, PANTHERx will provide patients with a review and summary of insurance benefit options, including information about eligibility for the Out-of-Pocket Assistance Program for EXSERVAN.
  • "PANTHERx prides itself on being focused on people living with the implications of rare disease," said Rob Snyder, President of PANTHERx.
  • EXSERVAN, anoral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) in November 2019.

Otolith Labs Announces Closing of $3.3 Million in Seed Financing; Receives “Breakthrough Device” Designation from FDA for Its Prescription Treatment for Vertigo

Retrieved on: 
Thursday, June 24, 2021

Otolith Labs , launched at the Halcyon Incubator, announced today it has received U.S. Food and Drug Administration (FDA) BREAKTHROUGH DEVICE designation for its medical device that is designed to be prescribed by physicians for the treatment of vertigo.

Key Points: 
  • Otolith Labs , launched at the Halcyon Incubator, announced today it has received U.S. Food and Drug Administration (FDA) BREAKTHROUGH DEVICE designation for its medical device that is designed to be prescribed by physicians for the treatment of vertigo.
  • View the full release here: https://www.businesswire.com/news/home/20210624005166/en/
    Otolith Labs' vertigo treatment device (Photo: Business Wire)
    Otolith Labs noninvasive VESTIBULAR SYSTEM MASKING (nVSM) technology is designed to provide acute treatment for vestibulogenic vertigo.
  • The FDA Breakthrough Device designation will empower us to expedite the availability of our technology for the more than four million people with chronic vertigo who currently lack any viable alternative.
  • Under the program, the FDA will provide Otolith Labs with priority review and interactive communication regarding clinical trial designs and commercialization decisions.

Major shareholder announcement

Retrieved on: 
Thursday, June 24, 2021

Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases.

Key Points: 
  • Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases.
  • The company is harnessing amplification of heat shock proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction.
  • Arimoclomol, the companys lead candidate, is in clinical development for rare diseases including Niemann-Pick disease type C (NPC) and Gaucher disease.
  • Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland.

Alnylam Announces U.S. Food and Drug Administration Acceptance of New Drug Application for Investigational Vutrisiran for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis

Retrieved on: 
Thursday, June 24, 2021

If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.

Key Points: 
  • If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.
  • Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis.
  • Vutrisiran has also been granted a Fast-Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
  • Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis.

June is Migraine and Headache Awareness Month

Retrieved on: 
Wednesday, June 23, 2021

"I get nausea both from my migraine aura and from the extreme pain of the migraines themselves," says one migraine patient who suffers from migraine-related nausea.

Key Points: 
  • "I get nausea both from my migraine aura and from the extreme pain of the migraines themselves," says one migraine patient who suffers from migraine-related nausea.
  • According to the Migraine Research Foundation, migraine affects approximately 39 million men, women, and children in the United States and 1 billion worldwide.
  • "We're proud to support the American Migraine Foundation in helping to raise awareness for Migraine and Headache Awareness Month," Nihan says.
  • Learn more about Migraine and Headache Awareness Month at americanmigrainefoundation.org .

Major shareholder announcement

Retrieved on: 
Wednesday, June 23, 2021

Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases.

Key Points: 
  • Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases.
  • The company is harnessing amplification of heat shock proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction.
  • Arimoclomol, the companys lead candidate, is in clinical development for rare diseases including Niemann-Pick disease type C (NPC) and Gaucher disease.
  • Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland.

InFlectis BioScience Licenses Rights to a Potential New Class of Therapies for Demyelinating Diseases from The University of Chicago

Retrieved on: 
Tuesday, June 22, 2021

Nantes, France and Chicago, Illinois--(Newsfile Corp. - June 22, 2021) - InFlectis BioScience has been granted exclusive patent rights from the University of Chicago to use a family of small molecules for the treatment of demyelinating diseases.

Key Points: 
  • Nantes, France and Chicago, Illinois--(Newsfile Corp. - June 22, 2021) - InFlectis BioScience has been granted exclusive patent rights from the University of Chicago to use a family of small molecules for the treatment of demyelinating diseases.
  • The license covers the use of InFlectis BioSciences' lead compounds, IFB-088 and IFB-048, for the treatment of CMT and MS, two highly prevalent demyelinating diseases.
  • InFlectis BioScience has licensed rights from the University of Chicago for the use of a family of molecules to treat demyelinating diseases.
  • InFlectis BioScience is a France-based clinical stage company developing first-in-class therapies for neuromuscular diseases.

Anavex Life Sciences Announces ANAVEX®2-73 (Blarcamesine) Biomarker Correlated with Efficacy Endpoints in Placebo-Controlled U.S. Phase 2 Clinical Trial for the Treatment of Adult Patients with Rett Syndrome

Retrieved on: 
Monday, June 21, 2021

Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups.

Key Points: 
  • Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups.
  • The population of patients with Rett syndrome is estimated to be approximately 11,000 patients in the U.S.
  • The Phase 2 trial is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX2-73 to treat Rett syndrome in a total of 31 adult patients with Rett syndrome over a 7-weeks treatment period (End of Trial, EOT) were evaluated incorporating precision medicine biomarkers.
  • Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Taysha Gene Therapies Announces Publication of Natural History Data for TSHA-120 in Giant Axonal Neuropathy in the Journal, Brain

Retrieved on: 
Monday, June 21, 2021

The data were published online and will be included in the June edition of Brain, a highly esteemed neurological science peer-reviewed journal.

Key Points: 
  • The data were published online and will be included in the June edition of Brain, a highly esteemed neurological science peer-reviewed journal.
  • Symptoms worsen over time and children develop scoliosis, contractures, atrophy of the spinal cord and abnormalities of the white matter in the brain.
  • Currently, there are no approved treatments for GAN, which results in death for patients in their late teens or early twenties.
  • In this natural history study, 45 patients, age 3 years to 21 years old, with genetically confirmed GAN were enrolled at NIH and evaluated at their first enrollment visit.