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NKGen Biotech, Inc. Reports Third Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, November 14, 2023
Alzheimer's disease, Business analyst, Microsoft PowerPoint, IND, Clinical trial, CSF, Patient, GFAP, Intellectual property, Research, Week, FDA, Glial fibrillary acidic protein, G&A, Conference, Cerebrospinal fluid, R, Loss, NK, Traffic barrier, Medical device, Pharmaceutical industry, Security (finance), Graf

SANTA ANA, Calif., Nov. 14, 2023 (GLOBE NEWSWIRE) -- NKGen Biotech Inc. (Nasdaq: NKGN) (NKGen or the Company), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous, allogeneic, and CAR-NK natural killer (NK) cell therapeutics, today announced its financial results for the third quarter ended September 30, 2023, and provided business highlights.

Key Points: 
  • 90% of patients demonstrated improvement or maintained stable cognitive function as per Alzheimer’s disease composite score (ADCOMS) following 11 weeks.
  • Research and development (R&D) expenses: R&D expenses for the third quarter of 2023 were $3.9 million compared to $4.1 million in the same period in 2022.
  • General and administrative (G&A) expenses: G&A expenses for the third quarter of 2023 were $3.0 million compared to $1.9 million in the same period in 2022.
  • The increase was primarily due to stock option grants made during the first quarter of 2023.

U.S. FDA Grants Orphan Drug Designation to JR-441 for the Treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA)

Retrieved on: 
Friday, December 15, 2023
FDA, Health, Science, Pharmaceutical, Research, Food, MPS, European Commission, ODD, Disease, Clinical trial, Civil service commission, Patient, BBB, JCR, NCT, TSE, ClinicalTrials.gov, Sanfilippo, Traffic barrier, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A).
  • JR-441 is a blood-brain barrier (BBB)-penetrating form of recombinant heparan N-sulfatase that was developed using JCR’s proprietary J-Brain Cargo® BBB-penetrating technology.
  • JCR is currently conducting a global Phase I/II clinical trial for JR-441, with the first patient with MPS IIIA dosed in October 2023.
  • We look forward to advancing JR-441 in clinical development and bringing this very important treatment to patients with MPS IIIA as quickly and safely as possible."

Alnylam Highlights Significant Progress with Platform Innovation and Clinical Pipeline at R&D Day

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CNS, Hypertension, Cerebral amyloid angiopathy, Hepatocellular carcinoma, Gene, ALN, Cardiomyopathy, RNA interference, IND, Tissue, Liver, Sanofi, Amyloidosis, Gene silencing, Overweight, KHK, Patient, FGF21, CAA, Part, Obesity, T2DM, Neoplasm, Investigational New Drug, Therapy, LNP, Development, Shanda, Growth, HCC, Central nervous system, Type, TTR, Immunotherapy, Polyneuropathy, Haemophilia, Medication, Biology, Coagulopathy, Traffic barrier, Adipose tissue, Vaccine, Medical device, Pharmaceutical industry, Video game, Alnylam Pharmaceuticals, Capella, ATTR

We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.

Key Points: 
  • We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.
  • In addition, Alnylam will share an update on delivery advances driving ALN-BCAT, which will soon begin clinical testing in patients with hepatocellular carcinoma (HCC).
  • A peak mean TTR reduction of 97% was achieved at Day 29 and a mean TTR reduction of 93% was sustained at Day 180.
  • The Company’s R&D Day event will be held today, Wednesday, Dec. 13, from 8:30 a.m. to 12:45 p.m.

Modalis and JCR Pharmaceuticals enter into joint research agreement

Retrieved on: 
Tuesday, December 12, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, CNS, Brain, Safety, DNA, Tokyo Stock Exchange, Patient, DSM-IV codes, Guide, BBB, JCR, Therapy, CRISPR, Cardiomyopathy, Central nervous system, ICM, Traffic barrier, Pharmaceutical industry

Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) today announced that it has entered into a joint research agreement with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) on joint evaluation of the drug delivery technology of the gene therapy to the central nervous system (CNS) assuming CNS diseases as a target.

Key Points: 
  • Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) today announced that it has entered into a joint research agreement with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) on joint evaluation of the drug delivery technology of the gene therapy to the central nervous system (CNS) assuming CNS diseases as a target.
  • Taking this agreement as an opportunity, Modalis and JCR Pharmaceuticals aim to establish the new gene therapies for undisclosed CNS diseases by applying J-Brain Cargo®, JCR Pharmaceuticals’ proprietary technology for crossing the blood-brain barrier (BBB), and CRISPR-GNDM® (Guide Nucleotide-Directed Modulation), Modalis proprietary epigenome modulation technology which does not require DNA cleavage.
  • Therefore, this joint research is an important step to explore the application of J-Brain Cargo®, with the aim of establishing new gene therapies in combination with CRISPR-GNDM®.
  • Haru Morita, CEO of Modalis, stated: “JCR Pharmaceuticals’ proprietary BBB penetrating technology, J-Brain Cargo®, has successfully and efficiently delivered biopharmaceutical candidates of various modalities to the CNS.

Menarini Group Presented Initial Safety and Efficacy Data from Phase 1b/2 ELEVATE and ELECTRA Combination Studies of ORSERDU® (Elacestrant) in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the 2023 San Antonio Breast Cancer Symposium

Retrieved on: 
Monday, December 11, 2023
Oncology, Health, Diabetes, Infectious Diseases, Clinical Trials, Pharmaceutical, Cardiology, Medicine, Observation, Research, Safety, Therapy, Toxicity, Cohort, University, Everolimus, Learning, Brain, Patient, Ribociclib, Division, Pharmacokinetics, Hematology, Neutropenia, Diarrhea, Nausea, Cancer, Financial toxicity, Menarini, Traffic barrier, Pharmaceutical industry, MD

Both the ELEVATE and ELECTRA studies were designed with the objective to overcome different resistance mechanisms and enhance patient outcomes with combination treatment options.

Key Points: 
  • Both the ELEVATE and ELECTRA studies were designed with the objective to overcome different resistance mechanisms and enhance patient outcomes with combination treatment options.
  • Data was presented at the 2023 San Antonio Breast Cancer Symposium (SABCS), December 5-9, 2023.
  • The ELEVATE study is evaluating elacestrant in combination with inhibitors of the PI3K/AKT/mTOR pathway (everolimus and alpelisib) and cell-cycle pathway inhibitors (palbociclib, ribociclib, and abemaciclib).
  • Additional cohorts are currently under evaluation to assess pharmacokinetics (PK) and determine the recommended phase 2 dose (RP2D) for each combination.

Delix Presents Interim Data From Phase I Trial of Novel Neuroplastogen at ACNP Annual Meeting

Retrieved on: 
Wednesday, December 6, 2023
Biotechnology, Pharmaceutical, General Health, Health, Mental Health, Genetics, Clinical Trials, Other Health, CNS, Psilocybin, Bowdle, South Dakota, DMT, Phase 1, Therapy, DSM-IV codes, Prefrontal cortex, Plasma, Food, CSF, SAD, Hallucination, Patient, Memantine, Neuron, American College, Pharmacokinetics, Science, Cardiotoxicity, Mysticism, MD, Drug discovery, Psychiatry, Coronavirus Scientific Advisory Board, Ketamine, Drug development, Traffic barrier, Pharmaceutical industry, Generic drug, Vaccine, Medical imaging, Ramipril, Allopregnanolone, Safety

Delix Therapeutics , a clinical-stage neuroscience company developing novel neuroplasticity-promoting therapeutics for psychiatric and neurological disorders, today announced interim results from the Phase 1 trial evaluating DLX-001, a novel neuroplastogen, in healthy volunteers, which were presented at the 2023 American College of Neuropsychopharmacology (ACNP) Annual Meeting in Tampa, Florida.

Key Points: 
  • Delix Therapeutics , a clinical-stage neuroscience company developing novel neuroplasticity-promoting therapeutics for psychiatric and neurological disorders, today announced interim results from the Phase 1 trial evaluating DLX-001, a novel neuroplastogen, in healthy volunteers, which were presented at the 2023 American College of Neuropsychopharmacology (ACNP) Annual Meeting in Tampa, Florida.
  • “Delix is excited to share these data from DLX-001, the first novel neuroplastogen to enter clinical testing.
  • These data reinforce the potential of our broader neuroplastogen platform to develop therapies that can repair damaged neurons,” said Delix CEO Mark Rus.
  • I look forward to leveraging his considerable experiences as we progress our novel neuroplastogen platform and build Delix into a leading neuroscience company."

Enveric Biosciences Selects Development Candidates from EVM301 Series Based on Potential to Minimize or Eliminate the Hallucinogenic Effect of Psychedelic-Derived Compounds

Retrieved on: 
Tuesday, December 5, 2023
Biotechnology, Mental Health, Health, Pharmaceutical, Clinical Trials, Depression, Research, IND, Hallucination, Clinical trial, Serum, Mouse, Addiction, Artificial intelligence, Neuroplasticity, Investigational New Drug, Therapy, Anxiety, Permeability, HTR, Traffic barrier, Pharmaceutical industry

Enveric plans to select one of the compounds for preclinical development early in 2024 in anticipation of Investigational New Drug (IND) application for a first-in-human clinical trial.

Key Points: 
  • Enveric plans to select one of the compounds for preclinical development early in 2024 in anticipation of Investigational New Drug (IND) application for a first-in-human clinical trial.
  • Enveric’s research team successfully narrowed the EVM301 Series to three candidates with preclinical data indicating 5HT2A receptor binding, induction of neuroplasticity, and positive stress recovery outcomes in animal models showing no indication of hallucinations.
  • All three candidates demonstrated acceptable assessments of cellular toxicity, blood-brain barrier permeability, and differential binding to other serotonin receptors and transporters.
  • We are committed to aggressively pursuing a thorough and expedited preclinical program in 2024 in preparation for an IND application.”

Global CNS Therapeutics Strategic Research Report 2023 - ResearchAndMarkets.com

Retrieved on: 
Friday, December 1, 2023
General Health, Health, Other Health, CNS, Neurodegenerative disease, Pressure, Research, Therapy, Pathology, Incidence, Patient, Growth, Mental health, BBB, Music, Alzheimer's disease, Dementia, Epilepsy, Plasma protein binding, Biology, Traffic barrier, Central nervous system, Pharmaceutical industry, Pet industry, Video game

The "CNS Therapeutics - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "CNS Therapeutics - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering.
  • The global CNS (Central Nervous System) therapeutics market is witnessing significant growth due to various factors.
  • Moreover, increased spending on prescription drugs and rising healthcare expenditure are contributing to the expansion of the CNS therapeutics market.
  • Despite the complexities of research and development in this field, the future outlook for CNS therapeutics appears promising, with novel drug-delivery options and advances in drug discovery methods for rare diseases.

VBI Vaccines Presents Additional Biomarker Data from Phase 1/2a Study of VBI-1901 in Recurrent GBM at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting

Retrieved on: 
Monday, November 20, 2023
Biotechnology, FDA, Other Health, Health, Pharmaceutical, Oncology, Neurology, Research, Infectious Diseases, Science, Clinical Trials, Education, Toxicity, Progression-free survival, Disease, GBM, Vaccination, Patient, Tumor microenvironment, VBI, Vaccine, Survival, Society, SNO, HIV disease progression rates, Traffic barrier, Pharmaceutical industry

New proof-of-mechanism data demonstrated that vaccination with VBI-1901 was associated with T-cell activation capable of trafficking across the blood-brain barrier to the tumor microenvironment, an area which is known to be highly immunosuppressive and difficult to infiltrate.

Key Points: 
  • New proof-of-mechanism data demonstrated that vaccination with VBI-1901 was associated with T-cell activation capable of trafficking across the blood-brain barrier to the tumor microenvironment, an area which is known to be highly immunosuppressive and difficult to infiltrate.
  • Additionally, higher levels of C4G after vaccination with VBI-1901 have been associated with longer progression-free survival.
  • “We are very pleased to share these significant mechanistic findings at SNO,” said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer.
  • “The GBM tumor microenvironment is notoriously immunosuppressive and difficult to penetrate, which is why most treatment needs to be administered intratumorally, and why there are currently so few effective treatment options for patients.

Menarini Group Presented Initial Safety and Efficacy Data from Phase 1b/2 ELEVATE and ELECTRA Combination Studies of ORSERDU® (Elacestrant) in Patients with ER+, HER2- Metastatic Breast Cancer (mBC) at the 2023 San Antonio Breast Cancer Symposium

Retrieved on: 
Monday, December 11, 2023
Medicine, Observation, Research, Safety, CDK, Therapy, Toxicity, Cohort, University, Disease, Everolimus, Learning, Brain, Patient, Ribociclib, Division, Pharmacokinetics, Hematology, Neutropenia, Menarini, Diarrhea, Nausea, Cancer, Toxic leukoencephalopathy, Traffic barrier, Pharmaceutical industry, MD

Encouraging results from phase 1 ELECTRA, evaluating ORSERDU in combination with abemaciclib regardless of metastatic sites, show a tolerable and manageable safety profile with favorable preliminary efficacy.

Key Points: 
  • Encouraging results from phase 1 ELECTRA, evaluating ORSERDU in combination with abemaciclib regardless of metastatic sites, show a tolerable and manageable safety profile with favorable preliminary efficacy.
  • Both the ELEVATE and ELECTRA studies were designed with the objective to overcome different resistance mechanisms and enhance patient outcomes with combination treatment options.
  • Data was presented at the 2023 San Antonio Breast Cancer Symposium (SABCS), December 5-9, 2023.
  • "We look forward to learning more about ORSERDU's role in combination settings and its potential across the spectrum of metastatic breast cancer."