Orphan

Lisata Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to LSTA1 for the Treatment of Osteosarcoma

Retrieved on: 
Tuesday, April 9, 2024
Disease, Osteosarcoma, Patient, Orphan, Food and Drug Administration, Cancer, Therapy, FDA, Food, Pharmaceutical industry

LSTA1 recently received Rare Pediatric Disease Designation for osteosarcoma, as announced by the Company on March 21, 2024.

Key Points: 
  • LSTA1 recently received Rare Pediatric Disease Designation for osteosarcoma, as announced by the Company on March 21, 2024.
  • “We are thrilled to have received another favorable regulatory designation from the FDA.
  • “Osteosarcoma, while rare, is a type of bone cancer that is often associated with high morbidity, early metastasis, rapid progression, and poor prognosis.
  • In addition to the financial benefits, it also may potentially shorten clinical development due to closer collaboration with the FDA.

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

Retrieved on: 
Wednesday, April 3, 2024
DFO, OrbiMed, NESS, Cystic Fibrosis Foundation, APT, FEV1, Part, Phage therapy, Patient, Orphan, KOL, Acquisition, Șaeș, Cystic fibrosis, Infection, Therapy, Pseudomonas aeruginosa, Pharmaceutical industry

CAMBRIDGE, Mass. and NESS ZIONA, Israel, April 03, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.

Key Points: 
  • and NESS ZIONA, Israel, April 03, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.
  • Initial top line results of the Phase 2 trial are expected in the first quarter of 2025.
  • However, our financial statements contain an explanatory paragraph regarding substantial doubt about our ability to continue as a going concern.
  • ET to discuss its fourth quarter and full year 2023 financial results and to provide a corporate update.

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.

Ligand Pharmaceuticals Announces New Topiramate Injection Data Presented at 9th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures Conference

Retrieved on: 
Monday, April 8, 2024
Health, Neurology, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Epilepsy, Plasma, LGND, Conference, UMN, Seizure, Status epilepticus, Patient, Topiramate, Orphan, Pharming, Concomitant, Pharmacokinetics, University, Faculty, Safety, Poster, Food, Pharmaceutical industry

The poster was featured at the 9th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures Conference in London, England.

Key Points: 
  • The poster was featured at the 9th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures Conference in London, England.
  • The authors presented modeling and simulations of IV topiramate dosing based on previous studies conducted by UMN faculty in healthy research participants and patients.
  • Ligand licensed the rights to the IV formulation of topiramate from UMN and entered into a global license agreement with CURx Pharmaceuticals to further develop and commercialize the product.
  • “The availability of an injectable topiramate formulation will make it possible to ensure continuity of therapy.

Regeneron Provides Update on Biologics License Application for Odronextamab

Retrieved on: 
Monday, March 25, 2024
Regeneron Pharmaceuticals, Biologics license application, Patient, Orphan, EMA, European Medicines Agency, European, Safety, FDA, BLA, DLBCL, Lymphoma, Food, Pharmaceutical industry

Regeneron has been actively enrolling patients in multiple Phase 3 trials for odronextamab as part of the OLYMPIA program – one of the largest clinical programs in lymphoma.

Key Points: 
  • Regeneron has been actively enrolling patients in multiple Phase 3 trials for odronextamab as part of the OLYMPIA program – one of the largest clinical programs in lymphoma.
  • Regeneron is committed to working closely with the FDA and investigators to bring odronextamab to patients with R/R FL and R/R DLBCL as quickly as possible.
  • Regulatory review of odronextamab remains ongoing by the European Medicines Agency (EMA) for the treatment of R/R DLBCL and R/R FL.
  • The potential use of odronextamab in R/R DLBCL and R/R FL is currently under clinical development and has not been approved by any regulatory authority.

X4 Pharmaceuticals Reports Fourth-Quarter and Full-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, March 21, 2024
Security (finance), WHIM, WHIM syndrome, Conference call, Conference, RPD, New Drug Application, Ageing, Orphan, Wart, NDA, Priority review, Research and development, Immune system, File, ID, FDA, X4, PDUFA, Pharmaceutical industry

BOSTON, March 21, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the fourth quarter and full year ended December 31, 2023, and highlighted key 2023 events and expected upcoming milestones.

Key Points: 
  • “Following an incredibly productive 2023, we are expecting a transformative year in 2024,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights.
  • The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .
  • Following the completion of the call, a webcast replay of the conference call will be available on the company website.

XORTX Highlights Achievements of 2023 and Preparation for Registration Clinical Trial

Retrieved on: 
Tuesday, March 19, 2024
Consultant, Nephrology, Provisional application, XRX, Partnership, Chemistry, Kidney disease, Tablet, Orphan, Bryant University, University, EMA, Polycystic kidney disease, Trial of the century, Oxipurinol, MBA, GMP, Diet, PKD, Treatment, Society, Therapy, Patent, Rensselaer Polytechnic Institute, FDA, Marketing, Diagnosis, American Society of Nephrology, Uric acid, Pharmaceutical industry

Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals.

Key Points: 
  • Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals.
  • Each of these milestones permit the next step in the Company’s clinical development plan, being a “registration” clinical trial – XRX-OXY-201 in pursuit of accelerated approval and support of the Company’s lead program XRx-008 program for ADPKD.
  • Chemistry and Manufacturing, Clinical and Pre-Clinical Highlights – Produced drug substance for oxypurinol production and produced GMP drug substance; confirmed XORLO™ formulation, produced enhanced bioavailability and produced clinical supply of tablets for clinical trials.
  • August 29, 2023 – XORTX submitted an Orphan Drug Designation application to the EMA for the treatment of ADPKD.

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

Retrieved on: 
Tuesday, March 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.

VOYDEYA™ approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular hemolysis in adults with the rare disease PNH

Retrieved on: 
Monday, April 1, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Factor D, Arthralgia, Chest pain, Confusion, Neuropsychological test, Infection, Eculizumab, Risk, Headache, ALPHA, PNH, C5, Patient, Orphan, Mitigation, Vaccine, Light, Paroxysmal nocturnal hemoglobinuria, EVH, Bacteria, University, Chills, MD, Breakthrough therapy, Cough, European Medicines Agency, Fatigue, Nausea, Rash, Fever, Priority, Safety, Immune system, REMS, Division, Vomiting, Hematology, Patient safety, Medicine

VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.

Key Points: 
  • VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.
  • You must complete or update meningococcal vaccine(s) and streptococcus vaccine(s) at least 2 weeks before your first dose of VOYDEYA.
  • If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting VOYDEYA.
  • Carry it with you at all times during treatment and for 1 week after your last VOYDEYA dose.

Tango Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Highlights

Retrieved on: 
Monday, March 18, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, ODD, Cancer, Research, PRMT5, Brain, Mutation, BRCA, NSCLC, NYU Langone Health, Breast, General counsel, Patient, GBM, Orphan, Collaboration, DNA, DevOps, MTAP, Sarcoma, Head, MTA, AACR, Glioblastoma, Clinical trial, PARP, Therapy, Drug development, ATM, ACS, Safety, Pharmacokinetics, Carcinoma, Corporate governance, FDA, American Chemical Society, Food, Pharmaceutical industry

Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to discovering and delivering the next generation of precision cancer medicines, reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided business highlights.

Key Points: 
  • Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to discovering and delivering the next generation of precision cancer medicines, reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided business highlights.
  • “In 2023, we made meaningful progress developing precision oncology treatments and now have four ongoing phase 1/2 clinical trials.
  • The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to TNG462 in December 2023 for the treatment of soft tissue sarcomas.
  • The increase is the result of out-licensing a program to Gilead for $5.0 million during the second quarter of 2023.