Tagged News
UK High Court Rules Amgen and Samsung Bioepis Soliris Biosimilars Do Not Infringe Alexion Patent
- The UK High Court has ruled that biosimilar versions of Soliris (eculizumab) developed by Amgen and Samsung Bioepis do not infringe on Alexion's patent for paroxysmal nocturnal hemoglobinuria treatment.
- This favorable UK ruling comes just days after a Canadian court barred Amgen's Soliris biosimilar until patent expiry in 2027, highlighting the complex global landscape for biosimilar litigation.
- The decision potentially opens the UK market for these biosimilar alternatives, which could increase treatment accessibility and reduce costs for patients with this rare blood disorder.
Citrin Foundation and University Children's Hospital Zürich Launch Pioneering Center for Urea Cycle Disorders Research
- The Citrin Foundation and University of Zürich have established the world's first center dedicated to translational research in urea cycle disorders with a CHF$10 million commitment over ten years.
- Led by Professor Johannes Häberle, the center will focus on biomarker discovery, liver metabolic function research, novel therapy development including gene therapy, and clinical trial readiness.
- The initiative aims to bridge the "valley of death" between laboratory discoveries and clinical applications for rare metabolic diseases that currently have no curative treatments beyond liver transplantation.
FDA Grants Rare Pediatric Disease Designation to Relief Therapeutics' EB Treatment RLF-TD011
- The FDA has granted Rare Pediatric Disease designation to Relief Therapeutics' RLF-TD011 for epidermolysis bullosa, a rare genetic skin disorder affecting approximately 500,000 people worldwide.
- RLF-TD011 is a hypotonic acid-oxidizing solution with hypochlorous acid that provides antimicrobial and anti-inflammatory properties while creating a wound microenvironment conducive to healing.
- The designation could potentially lead to a Priority Review Voucher upon approval, which has significant value as demonstrated by a recent EB treatment voucher that sold for $155 million.
Highlighted Clinical Trials:
Northwestern University
Posted 2/6/2023
AnnJi Pharmaceutical Reports Promising Phase 1/2a Results for AJ201 in SBMA, a Rare Neuromuscular Disease
- AnnJi Pharmaceutical's AJ201 demonstrated positive results in a Phase 1/2a trial for Spinal and Bulbar Muscular Atrophy (SBMA), showing improvements in physical function and muscle biomarkers after 12 weeks of treatment.
- The investigational drug reduced mutant androgen receptor levels by more than 50% in over half of treated patients and activated the Nrf2 pathway, supporting its proposed mechanism of action.
- With no FDA-approved treatments currently available for SBMA, AnnJi plans to advance AJ201 to Phase 3 trials, potentially offering hope for approximately 1 in 40,000 males affected by this rare genetic disorder.
Highlighted Clinical Trials:
AnnJi Pharmaceutical Co., Ltd.
Posted 2/28/2023
Rainbow Children's Hospital Completes Eighth Zolgensma Gene Therapy for Spinal Muscular Atrophy in India
- Rainbow Children's Hospital in Secunderabad, India has successfully administered its eighth Zolgensma gene therapy treatment for Spinal Muscular Atrophy, a rare genetic neuromuscular condition affecting approximately 1,000 children in India.
- The one-time gene therapy, which costs approximately Rs 14 crore (USD 1.7 million) per dose, works by replacing the defective SMN1 gene using adeno-associated viral vector technology to enable production of essential SMN protein.
- Following treatment, patients require continuous monitoring by a multidisciplinary team and weekly laboratory investigations to identify potential adverse events and enable early intervention.
Telomir Pharmaceuticals Secures $3 Million Investment to Advance Novel Age-Reversal Drug for Rare Diseases
- Telomir Pharmaceuticals has secured $3 million in premium equity financing from its largest shareholder to advance Telomir-1, a first-in-class age-reversal molecule, toward an IND submission for rare disease indications by year-end.
- The company's lead candidate, Telomir-1, targets five fundamental biological drivers of aging and has shown promising preclinical results across multiple conditions including Progeria, Wilson's disease, and Type 2 diabetes.
- Telomir is also developing Telomir-Ag2, a novel stabilized Silver(II) compound with broad-spectrum antimicrobial activity against drug-resistant pathogens, addressing a global market projected to exceed $30 billion.
Worldwide Clinical Trials Joins WCG's Avoca Quality Consortium to Enhance Clinical Trial Standards
- Worldwide Clinical Trials has joined WCG's Avoca Quality Consortium (AQC), a collaborative network of over 200 life sciences companies focused on elevating clinical trial quality.
- The strategic partnership aims to enhance quality, efficiency, and regulatory compliance through industry collaboration and best practices, ultimately delivering faster and more reliable outcomes for customers.
- This membership aligns with Worldwide's commitment to quality assurance across its services, which include bioanalytical laboratory services, Phase I-IV clinical trials, and post-approval studies.
AstraZeneca Showcases Groundbreaking Cancer Research at ASCO 2025 with Two Plenary Presentations
• AstraZeneca will present over 80 abstracts at ASCO 2025, including two plenary presentations featuring camizestrant for HR-positive breast cancer and IMFINZI for early gastric cancer, marking their seventh consecutive year with plenary data.
• The SERENA-6 trial is the first positive Phase III study for a next-generation oral SERD in first-line HR-positive breast cancer, pioneering the use of circulating tumor DNA to guide treatment decisions.
• DESTINY-Breast09 data shows ENHERTU plus pertuzumab is the first treatment in over a decade to demonstrate superiority over standard care in first-line HER2-positive metastatic breast cancer.
Highlighted Clinical Trials:
AstraZeneca
Posted 11/19/2021
Daiichi Sankyo Co., Ltd.
Posted 9/1/2015
New Genetic Findings Challenge Traditional Understanding of Coats' Disease
- Research led by Dr. David Abramson reveals that Coats' disease, traditionally considered unilateral, shows bilateral involvement when examined with fluorescein angiography, challenging long-held clinical beliefs.
- Genetic testing identified a variation in the LTBP2 gene associated with Coats' disease, providing new insights into the condition's genetic underpinnings and supporting the concept that single genes can influence multiple diseases.
- Treatment approaches combining laser photocoagulation and anti-VEGF therapy showed promising results in advanced cases, though researchers speculate effectiveness may be linked to specific telomere biology disorders.
Bristol Myers Squibb to Close Gene Therapy Manufacturing Facility in Libertyville, Illinois
- Bristol Myers Squibb has announced the closure of its viral vector production facility in Libertyville, Illinois, with operations being consolidated to Devens, Massachusetts, as part of a $2 billion cost-saving initiative.
- The facility, which was acquired by Bristol Myers Squibb two years ago and previously used by Novartis to manufacture the gene therapy Zolgensma, will begin layoffs on July 1 according to state WARN reports.
- Company representatives acknowledged the difficult decision to cease vector manufacturing at the Libertyville site, which they noted "has operated at the highest level for BMS Cell Therapy."