MHRA

Molecure has published its financial report for 2023 - the company has significantly accelerated the development of its clinical and pre-clinical programmes and plans to make strong progress in research in 2024 and 2025

Retrieved on: 
Wednesday, April 3, 2024
Medical device, Annual report, ARG1, Documentation, Biotechnology, Sarcoidosis, Partnership, Patient, Scientific Research Publishing, USP7, Ethics, SPO, Biomarker, Research, Health care, Conception, Cancer, Messenger, POC, PLN, Biology, Royal, MHRA, Polish, Clinical trial, Safety, MOC, Phase, ARG2, Food, FDA, SMART, CDA, Pharmaceutical industry

We initiated clinical trials of our drug candidate, which was discovered and brought into clinical trials for cancer patients by Molecure.

Key Points: 
  • We initiated clinical trials of our drug candidate, which was discovered and brought into clinical trials for cancer patients by Molecure.
  • In 2023, our focus was also on preparing for a Phase II clinical trial for OATD 01, Molecure's flagship clinical programme.
  • We are very pleased to report that the first patient has started dosing in a clinical trial at a clinical site in the UK.
  • The Company plans to obtain further grants for projects within the framework of the pipeline developed so far.

Autolus Therapeutics announces acceptance of Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for obecabtagene autoleucel (obe-cel) for Patients with Relapsed/refractory (r/r) Adult B-Cell Acute Lymphoblastic Leukemia (B-AL

Retrieved on: 
Tuesday, April 2, 2024
Certification, MAA, B-ALL, Patient, Orphan, ALL, EMA, GMP, European Medicines Agency, MHRA, Therapy, Priority, Marketing, FDA, BLA, PDUFA, Pharmaceutical industry

Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL).

Key Points: 
  • Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL).
  • The MAA submission was based on data from the pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL.
  • Autolus’ Nucleus site has recently received the formal certification from the MHRA following a full inspection of the site in February 2024.
  • The MHRA issued two new GMP certificates to cover both clinical and commercial manufacture from the site.

Verve Therapeutics Announces Updates on its PCSK9 Program

Retrieved on: 
Tuesday, April 2, 2024
LDLR, Cardiovascular disease, Gene editing, PCSK9, ALT, Patient, RNA, Health care, Health Canada, IND, N-Acetylgalactosamine, MHRA, Asialoglycoprotein receptor, Clinical trial, CTAS, Therapy, Bleeding, Safety, Moyamoya disease, DSMB, FDA, Liver, ASCVD, LNP, Medsafe, Hypercholesterolemia, Food, Pharmaceutical industry

BOSTON, April 02, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced updates from the Heart-1 Phase 1b clinical trial of VERVE-101 and clearance of its Clinical Trial Applications (CTAs) by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and Health Canada for VERVE-102, with the Heart-2 Phase 1b clinical trial expected to initiate in the second quarter of this year.

Key Points: 
  • Six participants have been dosed at 0.45 mg/kg of VERVE-101, with a total of 13 participants dosed in the study.
  • Verve is conducting an investigation into the laboratory abnormalities and based on those results, expects to work with regulatory authorities to define a path forward for VERVE-101.
  • Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial.
  • VERVE-102 uses the same base editor and guide RNA for PCSK9 but a different lipid nanoparticle (LNP) delivery system than VERVE-101.

Qnovia, Inc. Announces the Appointment of Four New Members to its Scientific Advisory Board

Retrieved on: 
Tuesday, April 9, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Tobacco, Clinical Trials, Retail, Disease, Cardiovascular Research, Society, IND, Psychology, Therapy, Senior counsel, Population health, School, Dean (education), Partnership, Nicotine, Policy, Smoker, Behavior, NIH, Brown University, Trial of the century, University, SAB, Pharmacology, Homelessness, San Francisco General Hospital, UCSF, Public, Cancer, MD, Publication, Heated tobacco product, Hospital, Juul, FDA, Alpert Medical School, Pregnancy, Chair, Public health, University of Kansas, Cardiology, CTA, Mortality, Louisiana State University, Doctor of Philosophy, MHRA, Smoking, Toxicology, Division, Brown, Social science, Pharmaceutical industry

“We believe our proprietary drug-device combination platform has the potential to be a first-in-class and best-in-class treatment for smoking cessation.

Key Points: 
  • “We believe our proprietary drug-device combination platform has the potential to be a first-in-class and best-in-class treatment for smoking cessation.
  • Last fall, QN-01 demonstrated a superior pharmacokinetic profile compared to existing nicotine replacement therapies in our first-in-human Phase 1 clinical trial.
  • We plan to submit our IND and CTA to the regulatory bodies and look forward to commencing our Phase 1/2 clinical study this year.
  • He also served a three-year term on the Board of the Society for Research on Nicotine and Tobacco.

Fennec Pharmaceuticals Reports Full Year and Fourth Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 21, 2024
AYA, Research, Remuneration, Civil service commission, Administration, Conference, Net, Growth, Young adult, Fennec, FENC, Patient, Into, G&A, NCCN, Adolescence, Marketing, MHRA, TSX, European Commission, FDA, Pharmaceutical industry

RESEARCH TRIANGLE PARK, N.C., March 21, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, today reported its financial results for the fiscal year ended December 31, 2023 and provided a business update.

Key Points: 
  • ET ~
    RESEARCH TRIANGLE PARK, N.C., March 21, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, today reported its financial results for the fiscal year ended December 31, 2023 and provided a business update.
  • PEDMARQSI was granted EU marketing authorization by the European Commission in June 2023, and received UK approval from the MHRA in October 2023.
  • Achieved PEDMARK net product revenue of approximately $9 million and $21 million for the fourth quarter and full year 2023, respectively.
  • Financial Results for the Fourth Quarter and Fiscal Year Ended December 31, 2023
    Net Sales – Net product sales of $21.3 million in fiscal 2023 compared to $1.5 million in 2022.

Taysha Gene Therapies Reports Full Year 2023 Financial Results and Provides Corporate and Clinical Updates

Retrieved on: 
Tuesday, March 19, 2024
Diary, Patient, IND, Adolescence, Pediatrics, Therapy, FAAP, Rett syndrome, Research, Central nervous system, Risk, Webcast, CNS, Ageing, CHU, Safety, AAV, Mutation, Development, MECP2, Disease, Associate, Sleep, Șaeș, Dysrhythmia, FRCP, MTD, Movement, IDMC, VG, Hyperventilation, Principal, Administration, Index, Seizure, CTA, Female, MHRA, Phenotype, ID, Frasier, Pharmaceutical industry

Notably, she has vastly increased interest in social communication and activities at week 35 compared to earlier post-treatment assessments.

Key Points: 
  • Notably, she has vastly increased interest in social communication and activities at week 35 compared to earlier post-treatment assessments.
  • Dr. Schultz is a board-certified, licensed pediatric neurologist experienced in treating patients with Rett syndrome and leading gene therapy clinical trials.
  • She brings more than 17 years of clinical experience and will lead the Company’s clinical development, clinical operations, medical affairs and safety activities.
  • ET to review its financial and operating results and to provide corporate and clinical updates.

Fennec Pharmaceuticals and Norgine Enter into Exclusive Licensing Agreement to Commercialize PEDMARQSI in Europe, Australia, and New Zealand

Retrieved on: 
Monday, March 18, 2024
Civil service commission, Neuroblastoma, Ototoxicity, Partnership, Central nervous system, ANZ, Risk, FENC, Patient, Hepatoblastoma, Neoplasm, Cancer, MHRA, TSX, Hearing loss, European, Cisplatin, Safety, European Commission, Liver, Arnold & Porter, Pharmaceutical industry

RESEARCH TRIANGLE PARK, N.C. and UXBRIDGE, United Kingdom, March 17, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ: FENC; TSX: FRX), a commercial stage specialty pharmaceutical company, and Norgine, a leading European specialist pharmaceutical company, today announced an exclusive licensing agreement under which Norgine will commercialize PEDMARQSI® in Europe, Australia and New Zealand. PEDMARQSI is the first and only approved therapy in the EU and U.K. for the prevention of ototoxicity (hearing loss) induced by cisplatin chemotherapy in patients 1 month to

Key Points: 
  • Norgine will be responsible for all commercialization activities in the licensed territories and will hold all marketing authorizations in the licensed territories.
  • It is estimated that more than 5,000 pediatric patients annually are eligible for platinum-based chemotherapy in Europe.
  • PEDMARQSI was granted EU marketing authorization by the European Commission in June 2023, and received UK approval from the MHRA in October 2023.
  • The studies compared PEDMARQSI plus cisplatin-based regimens to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients.

Autolus Therapeutics Reports Full Year 2023 Financial Results and Business Updates

Retrieved on: 
Thursday, March 14, 2024
Nucleus, Bone marrow, BCMA, BioNTech, B-ALL, Patient, ALL, SLE, Engineering, Minocycline, European Medicines Agency, Therapy, Research, Canadian Aviation Regulations, Flow cytometry, ASH, General counsel, Immunotherapy, SVP, EMA, Legal Affairs, Trial of the century, GMP, SME, BLA, Nature Communications, Development, UCL, Biologics license application, Multiple myeloma, MIA, University College London, Senior, TRBC, GD2, ASCO, FDA, CD19, Conference call, Conference, MAA, EHA, ADS, MHRA, T cell, ACS Chemical Biology, PDUFA, Autoimmune disease, Pharmaceutical industry

“For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.

Key Points: 
  • “For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.
  • In February 2024, Autolus promoted Dr. Chris Williams to Chief Business Officer and Alex Driggs to Senior Vice President, Legal Affairs and General Counsel.
  • Dr. Leiderman brings extensive transactional and financial expertise, and Mr. Azelby brings more than 30 years of biopharmaceutical leadership and commercial experience to Autolus’ Board.
  • Financial Results for the Year Ended December 31, 2023

Pharming Group reports fourth quarter and full year 2023 financial results

Retrieved on: 
Thursday, March 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: 
Wednesday, March 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.