MHRA

Pharming Group reports fourth quarter and full year 2023 financial results

Retrieved on: 
torsdag, mars 14, 2024
Patient, Health care, Epidemiology, Executive Committee, Laboratory, Microsoft Access, Lymphocyte, Clinical trial, European Commission, APDS, MHLW, AEG, Welfare, Ageing, Committee, EMA, Trial of the century, PID, CTLA4, Safety, Pharmacokinetics, Ministry, CHMP, ODD, U.S. FDA, Prevalence, PTEN, Disease, HAE, Immunodeficiency, VUS, Literature, PIK3R1, PMDA, FDA, DSM-IV codes, Diagnosis, Civil service commission, IRP, Haploinsufficiency, Genetic testing, MAA, Autoimmunity, World Games, Iberian languages, Marketing, MHRA, Euronext Amsterdam, PIK3CD, Physician, Pharmaceutical industry

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Entrada Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
onsdag, mars 13, 2024
Administration, Patient, Regulatory affairs, Research, G&A, EMA, Doctor of Philosophy, DM1, Completeness, Health Canada, Senior, Net operating loss, IND, MHRA, Research and development, Duchenne muscular dystrophy, Clinical trial, Data management, Therapy, Vertex Pharmaceuticals, FDA, DMD, Medication, Pharmaceutical industry

BOSTON, March 13, 2024 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. The Company today reported financial results for the fourth quarter and full year ending December 31, 2023, and highlighted recent business updates.

Key Points: 
  • The Company today reported financial results for the fourth quarter and full year ending December 31, 2023, and highlighted recent business updates.
  • “We continue to make significant progress advancing our growing pipeline of intracellular therapeutics,” said Dipal Doshi, Chief Executive Officer of Entrada Therapeutics.
  • Collaboration Revenue: Collaboration revenue was $41.8 million for the fourth quarter of 2023 and $129.0 million for the full year of 2023.
  • The G&A expenses in the fourth quarter of 2023 were lower than the fourth quarter of 2022 primarily due to a decrease in legal and consulting fees.

Autolus Therapeutics receives Medicines and Healthcare products Regulatory Agency (MHRA) certification for Nucleus commercial manufacturing site

Retrieved on: 
tisdag, mars 12, 2024
Certification, Nucleus, Patient, Orphan, ALL, MIA, EMA, GMP, MHRA, Therapy, Priority, FDA, Pharmaceutical industry

This authorisation enables Autolus to manufacture for global commercial and clinical product supply from the Nucleus effective as of March 18, 2024.

Key Points: 
  • This authorisation enables Autolus to manufacture for global commercial and clinical product supply from the Nucleus effective as of March 18, 2024.
  • Following a full site inspection in February 2024, Autolus’s Nucleus site has recently received the formal certification from the MHRA.
  • The MHRA issued two new licenses to cover both clinical and commercial manufacture from the site and found no major or critical observations in their summary report.
  • Our manufacturing team did an outstanding job qualifying, validating and taking into operation the Nucleus in record time and establishing the foundation for high quality product supply,” commented Dave Brochu, Chief Technology Officer of Autolus.

GenSight Biologics Reports Cash Position as of March 31, 2024, and Provides Business Update

Retrieved on: 
torsdag, april 4, 2024
Health, Neurology, Genetics, Pharmaceutical, Optical, Biotechnology, PIII, URD, Three-body problem, Patient, Good manufacturing practice, Health care, EAP, Acquisition, EMA, Risk, Chapter, GMP, MHRA, DS, ATU, AMF, Good, Phase, AAP, Ethics, FDA, Amendment, AAC, Medication, Pharmaceutical industry

GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported its cash position as of March 31, 2024 and provided a business update.

Key Points: 
  • GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported its cash position as of March 31, 2024 and provided a business update.
  • Once the AAC/AAP program resumes, the Company estimates that the cash runway would be extended to the end of Q1 2025.
  • GenSight successfully manufactured two Drug Substance (DS) batches of LUMEVOQ® meeting Good Manufacturing Practice (GMP) standards in September and November 2023.
  • As of March 31, 2024, GenSight Biologics’ number of outstanding shares was 78,370,724 ordinary shares.

iECURE Announces FDA Clearance of Investigational New Drug Application for ECUR-506 to Initiate OTC-HOPE Trial for Treatment of Neonatal Onset Ornithine Transcarbamylase Deficiency in the U.S.

Retrieved on: 
torsdag, april 4, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Gene editing, Therapeutic Goods Administration, University, Patient, Gene, MD, Quality of life, IND, GTP, Liver, MHRA, OTC, Pediatrics, Safety, Pharmacokinetics, TGA, Food, Medication, Pharmaceutical industry

ECUR-506 will be evaluated in the OTC-HOPE study in newborn males with genetically confirmed neonatal onset OTC deficiency.

Key Points: 
  • ECUR-506 will be evaluated in the OTC-HOPE study in newborn males with genetically confirmed neonatal onset OTC deficiency.
  • The OTC-HOPE study was previously cleared to begin in the United Kingdom by the Medicines & Healthcare Products Regulatory Agency (MHRA) and Australia by the Therapeutic Goods Administration (TGA).
  • “There is a significant need for clinical research and treatment options for newborns with severe, neonatal onset OTC deficiency,” said Gabriel M. Cohn, MD, Chief Medical Officer of iECURE.
  • “We hope that treatment with this investigational therapeutic will show promise for clinically meaningful improvements for infants with neonatal onset OTC deficiency and their families.”

Bristol Myers Squibb Announces Pivotal KRYSTAL-12 Confirmatory Trial Evaluating KRAZATI (adagrasib) Meets Primary Endpoint of Progression-Free Survival for Patients with Pretreated KRASG12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung…

Retrieved on: 
torsdag, mars 28, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Civil service commission, BMY, NSCLC, Cetuximab, Progression-free survival, Patient, Neoplasm, Shanda, Health care, News, Bristol Myers Squibb, MHRA, Pancreatic cancer, CRC, Safety, European Commission, PFS, PDUFA, Medication, Pharmaceutical industry

The study remains ongoing to assess the additional key secondary endpoint of overall survival.

Key Points: 
  • The study remains ongoing to assess the additional key secondary endpoint of overall survival.
  • Results of the confirmatory trial showed that KRAZATI demonstrated a statistically significant and clinically meaningful benefit in PFS and ORR compared to standard-of-care chemotherapy as a second-line or later treatment for these patients.
  • “Today’s news is an important reinforcement of the power of a targeted therapy for patients with locally advanced or metastatic KRASG12C-mutated lung cancer.
  • Bristol Myers Squibb thanks the patients and investigators involved in the KRYSTAL-12 clinical trial.

Precision BioSciences Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides Business Update

Retrieved on: 
onsdag, mars 27, 2024
Technology, Biotechnology, FDA, Nanotechnology, Health, Pharmaceutical, Stem Cells, Other Science, Research, Infectious Diseases, Genetics, Fitness & Nutrition, Science, Clinical Trials, Janus, Vivo, Gene editing, Precision BioSciences, Patient, Cash, Mitochondrion, Health care, Infant, NHP, IND, Duchenne muscular dystrophy, Investment, Therapy, ATM, Lilly, Precision, Cell, Skeletal muscle, Hepatitis B, Rachel Haurwitz, Central nervous system, Novartis, Primate, RNA, DNA, Mitochondrial disease, Hepatology, CRISPR, Safety, VWAP, PMM, Food, AAV, Development, Prevail, Publishing, Gene, ASX, University, Diaphragm, OTC, Hemoglobinopathy, IMU, Economics, Administration, Sickle cell disease, PCSK9, CTA, MHRA, Reindeer, Heart, Autoimmune disease, Eli Lilly and Company, DMD

PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B.

Key Points: 
  • PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B.
  • In November 2023, Precision presented preclinical efficacy and safety data at the 2023 American Association for the Study of Liver Diseases Annual Meeting.
  • Cash and Cash Equivalents: As of December 31, 2023, Precision had $116.7 million in cash and cash equivalents.
  • Revenues: Total revenues for the quarter ended December 31, 2023 were $7.0 million, as compared to $10.6 million for the quarter ended December 31, 2022.

GenSight Biologics Reports Full Year 2023 Consolidated Financial Results

Retrieved on: 
fredag, mars 22, 2024
Health, Neurology, Genetics, Pharmaceutical, Optical, Biotechnology, Control, URD, Three-body problem, Safety, Partnership, Research, CIR, ANSM, CMC, EMA, BofA Securities, Risk, Chapter, MHRA, Clinical trial, European Investment Bank, ATU, AMF, Workforce, EIB, AAP, Marketing, FDA, Amendment, AAC, Cryptocurrency

From 2022 to 2023, the Company's research and development expenses remained stable, at €19.3 million in 2022 and €19.4 million in 2023.

Key Points: 
  • From 2022 to 2023, the Company's research and development expenses remained stable, at €19.3 million in 2022 and €19.4 million in 2023.
  • The Company's general and administrative expenses also remained stable and amounted to €5.4 million in 2022 and 2023.
  • The weighted average number of shares outstanding increased from 46.3 million in 2022 to 48.3 million in 2023, thereby reducing the loss per share from (€0.54) in 2023 to (€0.60) in 2023.
  • GenSight Biologics will report its cash position as of March 31, 2024 on April 4 2024.

Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation

Retrieved on: 
tisdag, mars 19, 2024
Biotechnology, Pharmaceutical, Genetics, Health, Infant, AAV, MAA, Disease, Patient, FBX, Innovation, HSCT, EMA, Krabbe disease, GMP, European Medicines Agency, MHRA, Education minister, Clinical trial, Safety, FDA, Neurodegenerative disease, Hematopoietic stem cell transplantation, Medication, Pharmaceutical industry

FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two.

Key Points: 
  • FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two.
  • FBX-101 has been granted Orphan Drug Designation and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), allowing Forge to advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease.
  • In the US, the FDA has granted FBX-101 Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation.
  • Through this designation Forge has expanded global regulatory experience to include interactions with the MHRA in the UK.

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
onsdag, mars 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.