Neurodegeneration

KemPharm Announces Promotion of Sven Guenther, Ph.D., to Chief Scientific Officer and Christal Mickle, M.A., to Chief Product Development Officer

Retrieved on: 
Wednesday, January 11, 2023
Master of Arts, Disease, New Drug Application, University, Virginia Tech, CNS, Regulation of tobacco by the U.S. Food and Drug Administration, Akrofi-Christaller Institute, NDAS, FDA, Senior, Drug development, State university system, NDA, Research and development, Medicinal chemistry, Central nervous system, Food, M.A, KMPH, Therapy, Glycogen storage disease, Neurodegeneration, Pain, Pharmaceutical industry, Research, ADHD, Biochemistry, Chemistry

CELEBRATION, Fla., Jan. 11, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS) disorders, neurodegenerative diseases, lysosomal storage disorders and related treatment areas, announced the promotions of Sven Guenther, Ph.D., to Chief Scientific Officer, and Christal M.M. Mickle, M.A., to Chief Product Development Officer. Ms. Mickle, a co-founder of KemPharm, and Dr. Guenther have been members of KemPharm’s leadership team since the early days of the Company.

Key Points: 
  • Mickle, M.A., to Chief Product Development Officer.
  • Ms. Mickle, a co-founder of KemPharm, and Dr. Guenther have been members of KemPharm’s leadership team since the early days of the Company.
  • Dr. Guenther was one of the first members of KemPharm, most recently serving as the company’s Executive Vice President of Research and Development.
  • As Chief Scientific Officer, he will continue to lead KemPharm’s research team and play a central role in the advancement of the company’s pipeline.

NKGen Biotech Appoints Paul Y. Song, MD as Chief Executive Officer

Retrieved on: 
Tuesday, January 10, 2023
Disease, Neurodegeneration, University, The Institute, Therapy, Cedars-Sinai Medical Center, Biotechnology, MD, Radiation, Patient, George Washington University, Institut Gustave Roussy, Research, Cancer, ASTRO, CMO, Carl Berg, Chicago (franchise), Pharmaceutical industry, NK

Song, MD as Chief Executive Officer (CEO) and Vice Chairman of the Board of Directors, effective January 10, 2023.

Key Points: 
  • Song, MD as Chief Executive Officer (CEO) and Vice Chairman of the Board of Directors, effective January 10, 2023.
  • “We are thrilled to welcome Paul as our new CEO and the experience, vision and leadership Paul brings to NKGen Biotech,” said Sangwoo Park, Founder and Chairman of NKGen Biotech.
  • “Paul will play a critical role in leading our company’s corporate strategy and the advancement of our potentially life-changing clinical portfolio in oncology and neurodegenerative disease.
  • Song, MD, CEO of NKGen Biotech.

InMed Provides Business Update and Milestones for 2023

Retrieved on: 
Tuesday, January 10, 2023
Database, Neurodegeneration, CBC, Molecule, Itch, THCV, Safety, Cannabinoid, FDA, Health, INMED, Glaucoma, BMI, Drug development, Radicle, CBDV, Patient, Appetite, CBT, Research, Clinical trial, Wound healing, Epidermolysis bullosa, Acquisition, INM, GLP, Pain, Pharmacy, Fine chemical, Pharmaceutical industry

Advance research using rare cannabinoids in treating neurodegenerative diseases such as Alzheimer’s, Huntington’s and Parkinson’s

Key Points: 
  • Advance research using rare cannabinoids in treating neurodegenerative diseases such as Alzheimer’s, Huntington’s and Parkinson’s
    VANCOUVER, British Columbia, Jan. 10, 2023 (GLOBE NEWSWIRE) -- InMed Pharmaceuticals Inc. (“InMed” or the “Company”) (Nasdaq: INM), a leader in the pharmaceutical research, development and manufacturing of rare cannabinoids and cannabinoid analogs, today outlines key accomplishments from 2022 and provides business update and catalysts for 2023.
  • Despite the many economic pressures affecting businesses on a global scale, including challenging capital markets, particularly in biotech, InMed was able to advance its programs and achieve a number of key milestones.
  • As we move forward into 2023, we are very encouraged by the strength of our pharmaceutical programs, with several material milestones anticipated in the coming quarters,” commented Eric A. Adams, InMed President and CEO.
  • As we enter 2023, we will continue to evaluate strategic options and long-term supply agreements for this business segment.

CENTOGENE and Denali Therapeutics Extend World’s Largest Observational Study on Parkinson’s Disease Genetics

Retrieved on: 
Monday, January 9, 2023
Disease, Neurodegeneration, Safety, PD, Genetics, Biogen, Partnership, Patient, Diagnosis, Therapy, Drug discovery, HIV disease progression rates, ClinicalTrials.gov, Denali, Pharmaceutical industry, LRRK2

In 2018, CENTOGENE entered a strategic collaboration with Denali Therapeutics for the targeted global identification of PD patients with genetic variations in the LRRK2 gene.

Key Points: 
  • In 2018, CENTOGENE entered a strategic collaboration with Denali Therapeutics for the targeted global identification of PD patients with genetic variations in the LRRK2 gene.
  • CENTOGENE conducts clinical studies with pharma partners around the world, such as Denali Therapeutics.
  • “Parkinson’s disease is a devastating neurodegenerative disease – affecting over 10 million people worldwide from all walks of life.
  • There is an urgent medical need to unveil multidimensional data,” said Kim Stratton, Chief Executive Officer at CENTOGENE.

Vaccinex Announces First Patient Dosed with Anti-CCR8 Antibody Licensed to Surface Oncology

Retrieved on: 
Monday, January 9, 2023
Regulatory T cell, Neurodegeneration, CCR8, Trial of the century, Surface, Cancer, GPCR, SEMA4D, Doctor of Philosophy, Vaccine

ROCHESTER, N.Y., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, today announced that its licensee, Surface Oncology (NASDAQ: SURF, “Surface”) dosed the first patient in its Phase 1/2 clinical study investigating SRF114 , an antibody discovered using Vaccinex’s ActivMAb® antibody discovery platform and licensed to Surface Oncology in 2021.

Key Points: 
  • ROCHESTER, N.Y., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, today announced that its licensee, Surface Oncology (NASDAQ: SURF, “Surface”) dosed the first patient in its Phase 1/2 clinical study investigating SRF114 , an antibody discovered using Vaccinex’s ActivMAb® antibody discovery platform and licensed to Surface Oncology in 2021.
  • “We are very pleased that Surface has progressed SRF114 into a Phase 1/2 clinical study.
  • Advancing this promising drug candidate into the clinic provides positive validation of our proprietary ActivMAb® antibody discovery platform,” said Ernest Smith, PhD, Chief Scientific Officer of Vaccinex.
  • Under the terms of the antibody discovery agreement, Vaccinex has the potential to receive progress-related clinical milestone payments and royalties on sales.

KemPharm Announces Board and Leadership Changes to Support its Transformation into a Leading Rare Disease Company

Retrieved on: 
Monday, January 9, 2023
Disease, Chief business officer, Cognia, University, New Drug Application, CNS, Notre Dame, Financial compensation, Shareholder, Autoimmunity, NDA, Rich, Central nervous system, KMPH, Therapy, Business development, Science, Takeda Pharmaceutical Company, Glycogen storage disease, Acquisition, Employment, Neurodegeneration, Management, Mallinckrodt, Northwestern University, Biotechnology, Biology, Pfizer, Accenture

CELEBRATION, Fla., Jan. 09, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS), neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced changes to its Board of Directors (Board) and executive leadership team as part of the Company’s ongoing business transformation initiatives. Effective immediately, Matthew R. Plooster, who has been serving as an independent member of the Board of Directors, has been appointed to serve as Chairman of the Board of Directors. Richard W. Pascoe, who has been serving as Executive Chairman, will transition into the role of Chief Executive Officer (CEO) and remains a member of the Board of Directors. Travis C. Mickle, Ph.D., who is co-founder and has been serving as President and CEO, will transition to the role of President and will also remain a member of the Board of Directors until the Company’s 2023 Annual Meeting of Stockholders. Thereafter, Dr. Mickle will continue to support the Company’s pipeline build and the planned resubmission of the arimoclomol New Drug Application (NDA) as a scientific advisor. Additionally, Joshua Schafer has been appointed to the newly created role of Chief Commercial Officer and Executive Vice President of Business Development.

Key Points: 
  • Thereafter, Dr. Mickle will continue to support the Company’s pipeline build and the planned resubmission of the arimoclomol New Drug Application (NDA) as a scientific advisor.
  • Additionally, Joshua Schafer has been appointed to the newly created role of Chief Commercial Officer and Executive Vice President of Business Development.
  • “With these changes, we are continuing to execute the strategic plan embarked upon two years ago to become a commercially-focused rare disease company with multiple value-creating programs,” said Matthew R. Plooster, Chairman of KemPharm’s Board of Directors.
  • “With this announcement of our senior leadership transition plan, we are excited to enter the next chapter of our corporate evolution.

Cyclo Therapeutics Announces First Patient Dosed in Phase 2b Study of Trappsol® Cyclo™ for the Treatment of Early Alzheimer’s Disease

Retrieved on: 
Monday, January 9, 2023
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Transport, NPC1, Cell, CYTH, Brain, Therapy, Safety, Dementia, AD, Cholesterol, C1, Risk, Patient, Physician, Niemann–Pick disease, Science, Cerebrospinal fluid, Genetic disorder, Neurodegeneration, Pharmaceutical industry

“The start of this study is a significant milestone for our Company and the Alzheimer’s disease community.

Key Points: 
  • “The start of this study is a significant milestone for our Company and the Alzheimer’s disease community.
  • The Phase 2b study is a U.S. multicenter, randomized, placebo-controlled, double-blind, parallel group, 6-month study evaluating the safety, tolerability, and potential efficacy of monthly Trappsol® Cyclo™ infusions in patients with early Alzheimer’s disease.
  • Enrolled patients will be randomized across three study arms: 500 mg/kg or 1000 mg/kg of Trappsol® Cyclo™ and placebo.
  • The study will consist of a screening period, treatment period up to 24 weeks, and a safety follow-up period.

Quanterix Expands Laboratory Developed Test Menu with Launch of Neurofilament Light Chain Test

Retrieved on: 
Monday, January 9, 2023
Health, Neurology, Other Science, Research, Science, Biotechnology, Simoa, Multiple sclerosis, RUO, Blood, Neurodegeneration, CSF, CLIA, Ageing, RRMS, SLAC National Accelerator Laboratory, MS, Laboratory developed test, AFL, HIV, Amyotrophic lateral sclerosis, Corporation, Microangiopathy, LDT, FDA, Risk, Neurology, Plasma, TBI, ISO 15189, Protein, Diagnosis, Central nervous system, CAR, Therapy, Patient, HIV/AIDS, Research, ISO, Stroke, Serum, Nerve, Concussion, Traumatic brain injury, Clinical trial, Cerebrospinal fluid, ALS, Medical device, Pharmaceutical industry, Medical imaging, Parkinsonism

NfL is a well-studied biomarker for neuro-axonal injury with wide applicability to different neurological disorders.

Key Points: 
  • NfL is a well-studied biomarker for neuro-axonal injury with wide applicability to different neurological disorders.
  • Any disorder or injuring force resulting in neuronal damage can lead to the release of NfL into the interstitial fluid and cerebrospinal fluid (CSF).
  • A fraction of these proteins diffuses into the blood, where concentrations are typically 50- to 100-fold lower than in CSF and difficult to measure with conventional immunoassay technologies.
  • “Assessment and monitoring of brain health is incredibly difficult and typically performed in limited fashion by surgery or imaging.

FSD Pharma Inc Incorporates New Subsidiary to Capitalize on Drug Development Incentives in Australia

Retrieved on: 
Monday, January 9, 2023
Mental Health, Health, Research, Pharmaceutical, Science, Biotechnology, Government, Mental health, Multiple sclerosis, Royal College of Physicians and Surgeons of Canada, Faculty, Associate, CAMH, Drug development, London Olympics, FSD, Neurodegeneration, University Hospitals Plymouth NHS Trust, Neurology, University, Liaquat National Hospital, University of Oxford, Science, Nicotine, DRS, CSE, Neuroscience, University of Bristol, Liver transplantation, Special interest group, Medicine, Scientist, Interest group, European Association for the Study of the Liver, University of Melbourne Faculty of Science, Royal college, Senior, Bachelor of Medicine, Bachelor of Surgery, British, University of Cambridge, FRA, University College London, National Hospital for Neurology and Neurosurgery, Fine chemical, Medical device, Pharmaceutical industry, Philosophy, Neurosurgery, Pharmacology

“We are excited about the incorporation of FSD Pharma Australia, where we have an excellent opportunity to advance our pipeline,” said Mr. Zeeshan Saeed, President of FSD Pharma.

Key Points: 
  • “We are excited about the incorporation of FSD Pharma Australia, where we have an excellent opportunity to advance our pipeline,” said Mr. Zeeshan Saeed, President of FSD Pharma.
  • “Australia is a hotbed for clinical stage biotech companies, and our drug development programs will be able to take advantage of facilities offered by various government incentives in Australia,” added Mr. Saeed.
  • Chataway, Dhanda and Lê have agreed to join our international panel to contribute to our current and new programs,” said Dr. Lakshmi Kotra, CEO of Lucid Psycheceuticals, a wholly owned subsidiary of FSD Pharma.
  • Dr. Chataway received his Bachelor of Medicine & Surgery from Oxford University, and Doctor of Philosophy from Cambridge University.

Myrtelle Announces Positive Interim Data in Phase 1/2 Clinical Trial of Its Proprietary Investigational Gene Therapy rAAV-Olig001-ASPA in Canavan Disease

Retrieved on: 
Monday, January 9, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, White matter, CSF, Clinical trial, MRI, Associate, Gross, N-Acetylaspartic acid, Food, Cerebrospinal fluid, Wright, European Medicines Agency, MD, Boonshoft School of Medicine, Inc., ASPA, CD, Metabolism, FIH, NAA, Patient, Myrtelle Canavan, Pediatrics, Classification, US Foods, Mutation, Canavan disease, Magnetic resonance imaging, Rita Raave, Orphan drug, Hospital, Fast Track, Neurodegeneration, Pharmaceutical industry, Medical imaging

Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.

Key Points: 
  • Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.
  • The observed improvements following gene therapy are in contrast to the continuous clinical decline expected for untreated CD patients.
  • The first three patients treated with the gene therapy in cohort one are now at least 18 months post therapy.
  • “The early results to date in patients treated in Myrtelle’s Phase 1/2 clinical trial are encouraging.