NASH

Corcept Therapeutics Announces Fourth Quarter and Full-Year 2023 Audited Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, February 15, 2024
Woman, ALS, Oncology, PIN, MD, Investment, Physician, Neuroinflammation, Syndrome, Exercise, GRACE, Doctor of Pharmacy, Prevalence, Ovarian cancer, Patient, Journal, NASH, Atrophy, NDA, Glomus tumor, Pharmaceutical industry

Our strong 2023 commercial results reflect that physicians are more regularly screening for hypercortisolism.

Key Points: 
  • Our strong 2023 commercial results reflect that physicians are more regularly screening for hypercortisolism.
  • Corcept’s fourth quarter 2023 revenue was $135.4 million, compared to $103.1 million in the fourth quarter of 2022.
  • Diluted net income per common share was $0.28 in the fourth quarter of 2023, compared to $0.14 in the fourth quarter of 2022.
  • Cash and investments were $425.4 million at December 31, 2023 compared to $436.6 million at December 31, 2022.

Poxel Reports Cash and Revenue for the Full Year 2023 and Provides Corporate Update

Retrieved on: 
Thursday, February 15, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Merck, Kidney, USD, POC, Adrenoleukodystrophy, Merck Serono, EPO, JPY, AMN, Sumitomo Pharma, European Patent Convention, Safety, Biomarker, EUR, ALD, ADPKD, Serono, Adenosine, Pharmacokinetics, Patent, Fatty liver disease, Patient, Diabetes, Disease, NASH, Plasma, Male, DSM-IV codes, VLCFA, Pharmaceutical industry

Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We remain focused and continue to concentrate all our efforts at securing financing options, prioritizing non-dilutive solution, and partnerships.

Key Points: 
  • Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We remain focused and continue to concentrate all our efforts at securing financing options, prioritizing non-dilutive solution, and partnerships.
  • We’ve also progressed our advanced discussions – in some cases on an exclusive basis – with several potential partners for each of our three proprietary products.
  • Based on this conservative assumption, Poxel expects to receive 8% royalties on TWYMEEG® net sales.
  • As of December 31, 2023, total cash and cash equivalents were EUR 2.3 million (USD 2.6 million)1, as compared to EUR 13.1 million (USD 14.0 million) as of December 31, 2022.

Reflection paper on investigation of pharmacokinetics in the obese population - Scientific guideline

Retrieved on: 
Wednesday, February 14, 2024
Leptin, PD, Metabolism, Toxicity, Body composition, Ciprofloxacin, Clinical Pharmacokinetics, Water, LBW, Perfusion, LBM, Canadian International Council, AUC, PLOS One, Bariatric surgery, Clopidogrel, CYP, Breast, Health, DNA-binding protein, BMC Public Health, Pharmacology, BMI, CRP, Pharmaceutical Research (journal), Organ, Draft, Guideline, Bypass surgery, WHO, Ageing, INT, Pharmacokinetics, Liver, Bioavailability, Chronic liver disease, Human, Incidence, Committee, Absorption, TBW, Cardiovascular disease, Anatomy, CYP2C19, PBPK, Cmax, Factorization of polynomials, European, Lancet, CHMP, Journal, NASH, Pharmacotherapy, Adipose tissue, Non-alcoholic fatty liver disease, Journal of Clinical Investigation, Medication package insert, Overweight, NPS MedicineWise, Permeability, Benzodiazepine, Meta-analysis, Liver disease, Half-life, Acetylcholine, Ciclosporin, AAG, Kinetics, Midazolam, Kidney, Safety, Biomarker, Clinical Pharmacology & Therapeutics, Peptide, Classification, Hypertension, Metabolic syndrome, Acute coronary syndrome, Insulin resistance, Fatty liver disease, CYP2E1, Lithium, Body mass index, Reproduction, EMA, IBW, Injection, Anker, NCA, Enzyme, Inflammation, Cancer, Weight loss, Gastrointestinal tract, Cardiac output, Public health, Adipokine, CYP2C9, E pluribus unum, Physiology, Agency, Risk, Steroid, Lymph, CYP2D6, BSA, GI, Cytokine, Systematic review, Vaccine

Reflection paper on investigations of pharmacokinetics in

Key Points: 
    • Reflection paper on investigations of pharmacokinetics in
      the obese population
      Table of contents
      1.
    • References .............................................................................................. 9

      Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 2/12

      1.

    • This is considered
      a shortcoming that is potentially compounded by obese patients often being poorly represented in
      clinical studies.
    • The specific aims of this reflection paper are to:
      ?

      describe how the effects of obesity can be investigated during clinical medicinal product
      development.

    • ?

      provide recommendations on when investigations of the effect of obesity on the PK of a
      medicinal product should be particularly considered.

    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 3/12

      ?

      discuss how to reflect PK (and/or PK/PD) findings in weight/weight-based dosing
      recommendations.

    • Absorption
      Reduced rate of absorption linked to locally reduced blood flow (8) is reported for the subcutaneous
      and transdermal routes in obese subjects.
    • Distribution
      The distribution of medicinal products is driven by body composition, regional blood flow and binding to
      tissue and plasma proteins.
    • Obese subjects have a larger absolute lean body weight (LBW) as well as fat mass.
    • The physicochemical properties of a medicinal product (lipophilicity, polarity, molecular size, and
      degree of ionization) influence its distribution in the body.
    • In BMI class III obese
      subjects, the blood flow per gram of fat is significantly lower than that observed in class I obese or
      lean subjects (4).
    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 4/12

      An increased amount of alpha-1-acid-glycoprotein (AAG), linked to a chronic inflammatory state, is
      reported in obese individuals.

    • Fatty infiltrations are present in the liver for 90% of obese subjects, with the extent of the infiltrations
      being proportional to the degree of obesity.
    • In some cases, in particular for CYP3A4 metabolized medicinal products,
      bodyweight normalized clearance can be lower in obese patients (23).
    • Based on presently available data, it has been suggested that uptake transporters

      Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 5/12

      are downregulated while efflux transporters may be upregulated (31).

    • Platelet hyper-reactivity is also observed,
      which can impair the response to anti-platelet medicinal products in obese patients (42, 43).
    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 6/12

      3.

      the medicinal product properties and scientific literature indicate that obesity may lead to a
      marked effect on elimination and/or distribution or on the PK/PD relationship.

    • These
      models may aid in extrapolating the known efficacy and safety in the non-obese population to the
      obese population.
    • The Pharmacokinetics of the CYP3A Substrate Midazolam in Morbidly Obese Patients
      Before and One Year After Bariatric Surgery.
    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 11/12

      41.

    • Reflection paper on investigations of pharmacokinetics in the obese population
      EMA/CHMP/535116/2016

      Page 12/12

OliX Pharmaceuticals Commences Patient Dosing in Phase 1 Clinical Trial of OLX75016 for MASH

Retrieved on: 
Tuesday, February 13, 2024
Health, Genetics, Fitness & Nutrition, Clinical Trials, Pharmaceutical, Biotechnology, Therapy, KOSDAQ, Genome-wide association study, Inflammation, Obesity, Hope, GWAS, Safety, MASH, Patient, Gene, Clinical trial, Cirrhosis, NASH, RNA interference, Non-alcoholic fatty liver disease

OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced that the first participant was dosed today with OLX75016.

Key Points: 
  • OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced that the first participant was dosed today with OLX75016.
  • OLX75016 is being developed to provide a novel treatment approach for metabolic dysfunctions associated with non-alcoholic fatty liver disease linked to obesity.
  • The phase 1 trial is ongoing in Brisbane, Australia.
  • This is how we believe OLX75016 is set apart, and we anticipate this novel therapeutic to bring hope to patients with multiple metabolic diseases, starting in phase 1 clinical trial."

Madrigal Pharmaceuticals Announces Publication of the Phase 3 MAESTRO-NASH Trial of Resmetirom in the New England Journal of Medicine

Retrieved on: 
Thursday, February 8, 2024
Principal, ALT, Liver failure, Fatty liver disease, Cirrhosis, Hepatotoxicity, Nausea, GGT, Death, Therapy, Biomarker, Patient, Breakthrough therapy, Safety, Incidence, FDA, NAFLD, Lipoprotein, Priority review, Diarrhea, NAS, Woman, University of Oxford, Disease, Drug development, Fibrosis, Biopsy, Risk, Week, Heart rate, PDUFA, CAP, NASH, Medicine, Liver cancer, Liver, Pharmaceutical industry

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis.
  • Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement.
  • More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.

Vandria Awarded €3.8M in Prestigious Innosuisse and Eurostars Grants to Advance Innovative Drug Programs for Age-Related Diseases of the CNS and Muscle

Retrieved on: 
Thursday, February 8, 2024
Partnership, Mitophagy, CHF, Lead, European, Conditional sentence, Science, Research, Deficit spending, AP-HP, MD, Mitochondrion, Therapy, Myopathy, Lung, CNS, Dementia, NASH, Liver, Pharmaceutical industry

Vandria contributes CHF 4.6M to the grant alongside the CHF 2.5M non-dilutive funding from Innosuisse.

Key Points: 
  • Vandria contributes CHF 4.6M to the grant alongside the CHF 2.5M non-dilutive funding from Innosuisse.
  • Innosuisse is the Swiss Innovation Agency that helps to fund science-based pre-commercial projects of start-ups with significant innovation potential.
  • Klaus Dugi, MD, CEO of Vandria, said, “These prestigious grant awards are an endorsement of the science and the team behind Vandria.
  • The Eurostars grant significantly helps us to advance, in parallel, our program in muscle disease.

Genflow Biosciences Releases 2023 Year-End Review and Highlights Key Priorities for 2024

Retrieved on: 
Wednesday, February 7, 2024
SIRT6, Fatty liver disease, Investigational New Drug, Research, Therapy, CDMO, Patient, IND, Longevity, EMA, Patent Cooperation Treaty, QMS, Medication, European Medicines Agency, Werner syndrome, NASH, GMP, PCT, Pharmaceutical industry

LONDON, Feb. 07, 2024 (GLOBE NEWSWIRE) -- Genflow Biosciences Plc (Genflow or the "Company”) (LSE:GENF) (OTCQB:GENFF), an emerging leader in the field of longevity research developing therapeutics that potentially halt or slow the ageing process is pleased to provide a year-end review and summary of its progress during 2023 and defined strategic priorities for 2024.

Key Points: 
  • LONDON, Feb. 07, 2024 (GLOBE NEWSWIRE) -- Genflow Biosciences Plc (Genflow or the "Company”) (LSE:GENF) (OTCQB:GENFF), an emerging leader in the field of longevity research developing therapeutics that potentially halt or slow the ageing process is pleased to provide a year-end review and summary of its progress during 2023 and defined strategic priorities for 2024.
  • Genflow expanded its intellectual property portfolio with a provisional patent application for editing the SIRT6 gene, linked to longevity and age-related diseases.
  • Began trading on the OTCQB Venture Market in the US under the ticker GENFF, aligned to its US-focused strategy.
  • Dr. Eric Leire, CEO of Genflow commented: “The milestones we reached in 2023 were numerous, and I commend our team for their exceptional commitment to execution and delivery.

Madrigal Pharmaceuticals Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Tuesday, February 6, 2024
Therapy, NASH, Fatty liver disease, Employment, Certification, Pharmaceutical industry

The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

Key Points: 
  • The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The equity awards were granted as inducement material to the employees’ acceptance of employment with the company.
  • All time-based restricted stock units granted vest in a 25% increment on each of the first through fourth anniversaries of the grant date.
  • The vesting of all awards described above shall be subject to each such employee’s continued employment as of the vesting date.

Nordic Bioscience to make its precision medicine biomarker PRO-C3 available in China for research use only (RUO) in the first quarter of 2024.

Retrieved on: 
Tuesday, February 6, 2024
Support, Fatty liver disease, Conditional sentence, Research, Biomarker, Patient, Labcorp, FDA, Idiopathic pulmonary fibrosis, IPF, Drug development, Letter, RUO, Neoplasm, MASH, ECM, NASH, Fine chemical

All testing outside China continues to be offered exclusively at Nordic Bioscience's laboratories in Herlev, Denmark.

Key Points: 
  • All testing outside China continues to be offered exclusively at Nordic Bioscience's laboratories in Herlev, Denmark.
  • The availability of PRO-C3 as RUO in China will allow drug developers and scientists in China pre-IVD access to PRO-C3, which will be offered via LabCorp in Shanghai.
  • "This collaboration will allow faster and broader access to Nordic's precision medicine enabling biomarker PRO-C3.
  • The license agreement between Nordic Bioscience and Roche will put multiple Nordic Bioscience biomarkers on the fully automated cobas® e platform from Roche over the coming years.

Nordic Bioscience to make its precision medicine biomarker PRO-C3 available in China for research use only (RUO) in the first quarter of 2024.

Retrieved on: 
Tuesday, February 6, 2024
Support, Fatty liver disease, Conditional sentence, Research, Biomarker, Patient, Labcorp, FDA, Idiopathic pulmonary fibrosis, IPF, Drug development, Letter, RUO, Neoplasm, MASH, ECM, NASH, Fine chemical

All testing outside China continues to be offered exclusively at Nordic Bioscience's laboratories in Herlev, Denmark.

Key Points: 
  • All testing outside China continues to be offered exclusively at Nordic Bioscience's laboratories in Herlev, Denmark.
  • The availability of PRO-C3 as RUO in China will allow drug developers and scientists in China pre-IVD access to PRO-C3, which will be offered via LabCorp in Shanghai.
  • "This collaboration will allow faster and broader access to Nordic's precision medicine enabling biomarker PRO-C3.
  • The license agreement between Nordic Bioscience and Roche will put multiple Nordic Bioscience biomarkers on the fully automated cobas® e platform from Roche over the coming years.