European Medicines Agency

Harvard Apparatus Regenerative Technology Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Tuesday, April 2, 2024
Uterus, Gynaecology, Longevity, Reproductive Sciences, Patient, Collaboration, University, EMA, MD, European Medicines Agency, European, PIPE, Esophageal atresia, Yale school, OTCQB, Cryptocurrency

The EMA is the centralized regulatory agency for the review and approval of new medicines in the European Union.

Key Points: 
  • The EMA is the centralized regulatory agency for the review and approval of new medicines in the European Union.
  • Company’s subsidiary in Hong Kong launched its longevity product business in Asia, generating more than $100,000 in 2023 sales.
  • Entered into securities purchase agreements with accredited investors for a $6 million private investment in public equity (PIPE) financing.
  • Holliston, MA, April 02, 2024 (GLOBE NEWSWIRE) -- Harvard Apparatus Regenerative Technology, Inc. (OTCQB: HRGN) (“Harvard Apparatus Regenerative Technology” or the “Company”), a clinical-stage biotechnology company developing the technology to regenerate organs inside the body to treat severe diseases, today reported fourth quarter and full year 2023 financial results.

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.

4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis

Retrieved on: 
Thursday, March 28, 2024
CDMO, Conference, TDN, Patient, Lung, Cohort, RNA, EMA, Technology transfer, Cystic fibrosis, European Medicines Agency, GMP, Clinical trial, ECFS, Safety, CFTR, AMD, Food, FDA, VG, Pharmaceutical industry

Phase 3 clinical endpoints include changes after 4D-710 treatment in ppFEV1, quality-of-life (Cystic Fibrosis Questionnaire Revised Respiratory Domain, CFQ-R-RD) and frequency of pulmonary exacerbations.

Key Points: 
  • Phase 3 clinical endpoints include changes after 4D-710 treatment in ppFEV1, quality-of-life (Cystic Fibrosis Questionnaire Revised Respiratory Domain, CFQ-R-RD) and frequency of pulmonary exacerbations.
  • The Company anticipates initiation of technology transfer to a commercial contract development and manufacturing organization (CDMO) in H1 2025.
  • Our goal is to initiate Phase 3 development in H2 2025 with 4D-710 suspension GMP process clinical trial material.
  • We look forward to sharing interim clinical data from the AEROW Phase 1/2 clinical trial at the ECFS conference in June 2024.”

XORTX Announces New Clinical Advisory Board Member

Retrieved on: 
Wednesday, March 27, 2024
Sanofi, Database, Internal medicine, Critical Path Institute, XRX, TEMPO, Patient, Research, Biomarker, TAME, Polycystic kidney disease, National Institutes of Health, MD, Kidney disease, European Medicines Agency, Publication, PKD, Clinical trial, Boston Medical Center, Therapy, Grady Memorial Hospital, FDA, Food, Pharmaceutical industry

CALGARY, Alberta, March 27, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a biopharmaceutical company focused on developing innovative therapies to treat autosomal dominant polycystic kidney disease (“ADPKD”), is pleased to announce that Dr. Ronald Perrone has joined the Company’s Clinical Advisory Board.

Key Points: 
  • CALGARY, Alberta, March 27, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a biopharmaceutical company focused on developing innovative therapies to treat autosomal dominant polycystic kidney disease (“ADPKD”), is pleased to announce that Dr. Ronald Perrone has joined the Company’s Clinical Advisory Board.
  • Dr. Allen Davidoff stated, “We are excited that Dr. Ron Perrone has agreed to join XORTX’s Clinical Advisory Board.
  • Dr. Perrone brings substantial medical and professional experience as a thought leader, combined with clinical experience treating patients with ADPKD and kidney disease.
  • We are privileged to have Dr. Perrone join our esteemed Clinical Advisory Board alongside current members, Dr. Petter Bjornstad, Dr. Richard Johnson, Dr. Federico Maese, Dr. Anjay Rostogi and Dr. Charles Edelstein.

89bio Receives EMA PRIME Status for Pegozafermin in the Treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH) with Fibrosis and Compensated Cirrhosis

Retrieved on: 
Wednesday, March 27, 2024
MASH, Cirrhosis, Patient, SAN, European Medicines Agency, Marketing, FGF21, Liver, Fibrosis, PRIME, EMA, Pharmaceutical industry

SAN FRANCISCO, March 27, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) status to pegozafermin in patients with MASH. The PRIME status was supported by positive data from the Phase 2b ENLIVEN trial of pegozafermin in patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4).

Key Points: 
  • The PRIME status was supported by positive data from the Phase 2b ENLIVEN trial of pegozafermin in patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4).
  • “The EMA PRIME status further supports pegozafermin’s positioning as a leading FGF21 analog for the treatment of MASH, which has demonstrated robust anti-fibrotic and metabolic benefits as seen in our Phase 2b ENLIVEN trial,” said Rohan Palekar, Chief Executive Officer of 89bio.
  • “This status recognizes the urgent need for effective treatment options for MASH patients with advanced fibrosis and underscores pegozafermin’s potential to address the targeted unmet medical need.
  • For more information on the PRIME status, please visit the EMA website at www.ema.europa.eu .

Annexon Reports Fourth Quarter and Year-End 2023 Financial Results and Key Anticipated Milestones

Retrieved on: 
Tuesday, March 26, 2024
Diagnosis, Research, Brain, Geographic atrophy, Disease, RWE, International, Paratriathlon, Patient, ARCHER, G&A, Pivotal, EMA, Meta-analysis, Eye, European Medicines Agency, Medicine, Visual acuity, Immunoglobulin therapy, GBS, Facial muscles, Clinical trial, POC, Safety, Pharmacokinetics, Investment, SAD, FDA, BLA, AFL, CAD, Food, Pharmaceutical industry

BRISBANE, Calif., March 26, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today highlighted portfolio progress and reported fourth quarter and full year 2023 financial results.

Key Points: 
  • Finally, we anticipate clinical proof-of-concept data from our novel oral inhibitor ANX1502 later in the year.
  • ANX005 in GBS: First-in-class monoclonal antibody designed to block C1q and the entire classical complement pathway in both the body and the brain.
  • ANX005 in GBS: Topline data from pivotal, randomized, placebo-controlled Phase 3 trial expected in the second quarter of 2024.
  • Cash and operating runway: Cash and cash equivalents and short-term investments were $259.7 million as of December 31, 2023.

Marker Therapeutics Reports Year-End 2023 Corporate and Financial Results

Retrieved on: 
Monday, March 25, 2024
ODD, Research, Food and Drug Administration, U.S. FDA, Drug discovery, AML, International, Patient, Committee, SBIR, Female, Acute myeloid leukemia, Sale, INN, Marker, EMA, OTS, NIH, IND, Myelodysplastic syndrome, European Medicines Agency, Counsel, Therapy, MRD, Bone marrow, USAN, MDS, APOLLO, FDA, DLBCL, Press release, CD19, Pharmaceutical industry

Marker also executed a comprehensive non-dilutive agreement with Cell Ready which included a sale of select cell manufacturing assets from Marker for approximately $19 million in cash.

Key Points: 
  • Marker also executed a comprehensive non-dilutive agreement with Cell Ready which included a sale of select cell manufacturing assets from Marker for approximately $19 million in cash.
  • Granted ODD from the Committee for Orphan Medicinal Products of the EMA for the treatment of patients with AML in 2023.
  • On June 26, 2023, Marker completed a non-dilutive transaction with Cell Ready, under which Cell Ready purchased certain cell manufacturing assets from Marker for approximately $19 million in cash.
  • Cash Position and Guidance: At December 31, 2023, Marker had cash and cash equivalents of $15.1 million.

LIB Therapeutics Announces Positive Results from LIBerate-HR Study: A 52-Week, Placebo-Controlled Registration-Enabling Trial of Lerodalcibep at Late-Breaking Session at American College of Cardiology 2024

Retrieved on: 
Monday, April 8, 2024
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Week, Temperature, Cardiovascular disease, PCSK9, CVD, ADAS, FH, Patient, Travel, Female, Refrigeration, OLE, MBBS, Marketing, European Medicines Agency, American College, Therapy, Liberation, ITT, Safety, Food, ACC, Apolipoprotein B, Apolipoprotein, Pharmaceutical industry

Patients were randomized 2:1 to a single 300 mg (1.2 mL subcutaneous) once-monthly dose of Lerodalcibep or placebo for 52 weeks.

Key Points: 
  • Patients were randomized 2:1 to a single 300 mg (1.2 mL subcutaneous) once-monthly dose of Lerodalcibep or placebo for 52 weeks.
  • Of 478 participants in the base LIBerate-HeFH trial, 421 (88%) continued into the OLE trial: 281 patients on Lerodalcibep and 140 on placebo.
  • All participants received open-label Lerodalcibep 300 mg in a subcutaneous 1.2 mL once-monthly dose starting immediately upon completion of the base trial.
  • After 48 weeks of OLE treatment, mean % LDL-C reductions with Lerodalcibep from baseline in the base trial was 48.5% (ITT population).

Third European Medicines Agency and Affordable Medicines Europe bilateral meeting, Online, European Medicines Agency, Amsterdam, the Netherlands, 10 April 2024

Retrieved on: 
Tuesday, April 9, 2024
European Medicines Agency, EMA

Third European Medicines Agency and Affordable Medicines Europe bilateral meeting

Key Points: 
  • Third European Medicines Agency and Affordable Medicines Europe bilateral meeting
    EventHuman
    Date
    Location
    This meeting between European Medicines Agency (EMA) and Affordable Medicines Europe is being organised in the context of the EMA annual bilateral meetings with industry stakeholder organisations.
  • The purpose of the meeting is to exchange views and promote dialogue on strategic topics of common interest.

Focus group on veterinary pharmacovigilance reporting in poultry, European Medicines Agency, Amsterdam, the Netherlands, 11 October 2023

Retrieved on: 
Tuesday, April 9, 2024
Focus, Focus group, Veterinarian, Pharmacovigilance, Location, European Medicines Agency, Public, Knowledge, Science, Animal, Agency, Environment, Poultry, Veterinary medicine

Date

Key Points: 
  • Date
    - Wednesday, 11 October 2023
    Location
    - European Medicines Agency, Amsterdam, the Netherlands
    Event summary
    The European Medicines Agency has a coordinating role in the EU pharmacovigilance system and operates services and processes to support veterinary pharmacovigilance activities.
  • This entails surveillance of adverse events, including lack of expected efficacy reported after veterinary medicinal products are used in practice, to safeguard animal and public health and the environment.
  • The Agency is hosting a Focus Group meeting with specialists in poultry veterinary science (veterinarians or other healthcare professionals) to facilitate reporting of adverse events.
  • This meeting is a follow-up to the 2016 focus group meeting held with EU veterinarians specialised in food-producing animals, on the promotion of pharmacovigilance reporting.