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Clinical Trial News

Ropeginterferon Alfa-2b Shows Superior Efficacy Over Anagrelide in Phase III Essential Thrombocythemia Trial

  • Ropeginterferon alfa-2b achieved a 42.9% sustained response rate compared to 6.0% with anagrelide in patients with essential thrombocythemia resistant or intolerant to hydroxyurea.
  • The drug demonstrated superior molecular responses with 27.8% partial and 2.8% complete molecular responses, while anagrelide showed no molecular responses.
  • Ropeginterferon alfa-2b significantly reduced thrombotic events to 1.1% versus 8.8% with anagrelide and showed better safety profile with fewer treatment discontinuations.
  • The SURPASS-ET trial results support ropeginterferon as a promising disease-modifying therapy for high-risk essential thrombocythemia patients.

Zilia Ocular Receives World-First Health Canada Approval for Non-Invasive Retinal Oxygen Measurement

AI
  • Zilia's flagship device becomes the first in the world authorized to measure oxygen saturation in the eye, marking a breakthrough in non-invasive ocular diagnostics.
  • The revolutionary retinal camera uses patented optical technology to detect metabolic biomarkers, potentially enabling earlier detection of glaucoma, diabetic retinopathy, and age-related macular degeneration.
  • The Canadian health technology company plans to launch the device domestically before expanding to the United States and Europe, positioning itself at the forefront of the emerging oculomics field.

CNS Oligonucleotide Licensing Deals Surge 339% to $2.81 Billion in 2024

Precision Medicine
  • Licensing agreements for CNS-targeted oligonucleotide therapeutics increased 339% from 2023 to 2024, reaching $2.81 billion in total deal value according to GlobalData's analysis.
  • Antisense oligonucleotides (ASOs) dominated the market with $3.54 billion in cumulative deals from 2021-2025, while siRNAs accounted for $2.51 billion during the same period.
  • Major pharmaceutical companies including AbbVie, Lilly, Roche and Takeda represented 63% of licensing deals, with Ionis Pharmaceuticals leading the field through $13.4 billion in out-licensed assets over the past decade.
  • Arrowhead Pharmaceuticals secured 2025's largest deal worth up to $2.18 billion through an exclusive licensing agreement with Sarepta Therapeutics for rare genetic and CNS disease programs.

Beta Bionics Partners with Abbott to Integrate iLet Bionic Pancreas with Future Dual Glucose-Ketone Sensor

  • Beta Bionics announced plans to integrate its iLet Bionic Pancreas automated insulin delivery system with Abbott's future dual glucose-ketone sensor in the U.S.
  • The iLet represents the first and only automated insulin delivery system that makes 100% of insulin dosing decisions automatically without requiring carb counting or manual calculations.
  • This collaboration extends the existing partnership between Beta Bionics and Abbott, building on the current integration with Abbott's FreeStyle Libre 3 Plus continuous glucose monitoring sensor.
  • The integration aims to enhance real-time decision-making support for people living with diabetes through advanced automated insulin delivery technology.

Subcutaneous Immunoglobulin with Hyaluronidase Shows Promise for Idiopathic Inflammatory Myositis Treatment

  • A recent study demonstrates that subcutaneous immunoglobulin (SCIg) facilitated by recombinant human hyaluronidase effectively sustains clinical remission in patients with idiopathic inflammatory myositis.
  • The treatment offers comparable therapeutic benefits to intravenous immunoglobulin (IVIg) while minimizing adverse effects and reducing financial burdens associated with traditional therapy.
  • Patients experienced sustained remission without significant side effects commonly linked to IVIg therapy, with additional logistical advantages including reduced hospital dependency.
  • The subcutaneous administration presents improved accessibility and convenience for long-term management of this autoimmune condition.

Theranica's Nerivio REN Wearable Shows Sustained Migraine Efficacy Over Three Years in Real-World Studies

Real World Evidence
  • Theranica presented three real-world evidence studies at the 2025 American Headache Society meeting demonstrating the sustained efficacy of its FDA-cleared Nerivio REN wearable for migraine treatment.
  • A long-term study showed the REN wearable maintains consistent efficacy over three years without signs of tachyphylaxis or treatment fatigue, providing ongoing pain relief and functional recovery.
  • Analysis of nearly 32,000 patients revealed comparable treatment outcomes between migraine with aura and without aura subtypes, reinforcing the device's reliable performance across different migraine types.
  • The studies support REN as a non-drug alternative for patients seeking sustainable migraine management without the risks of systemic side effects or drug interactions.

Tocilizumab Biosimilar CT-P47 Demonstrates Long-Term Efficacy and Safety in Rheumatoid Arthritis Treatment

Monoclonal AntibodyDrug Approval
  • CT-P47, a tocilizumab biosimilar, showed comparable efficacy and safety to reference Actemra over 52 weeks in a phase 3 trial involving 444 rheumatoid arthritis patients.
  • Patients who switched from Actemra to CT-P47 maintained similar disease control with no new safety concerns, supporting biosimilar interchangeability.
  • The biosimilar demonstrated successful self-administration via autoinjector with high patient satisfaction scores and minimal injection site reactions.
  • Multiple tocilizumab biosimilars have gained regulatory approval, offering potential cost savings of 16-26% compared to the originator biologic.

A Study to Evaluate Usability of Subcutaneous Auto-injector of CT-P47 in Patients With Active Rheumatoid Arthritis

NCT05725434Not Yet RecruitingPhase 3
Celltrion
Posted 2/6/2023

A Study to Compare Efficacy and Safety of CT-P47 and RoActemra in Patients With Rheumatoid Arthritis

NCT05489224CompletedPhase 3
Celltrion
Posted 9/14/2022

FDA Approves First Treatment for Alkaptonuria: Nitisinone Shows Clinical Benefits in Rare Metabolic Disorder

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved nitisinone (Harliku) as the first and only treatment for alkaptonuria, a rare genetic metabolic disorder affecting 1 in 250,000 to 1 million Americans.
  • A 3-year randomized clinical trial of 40 patients demonstrated that nitisinone improved pain, energy levels, and physical functioning compared to untreated controls.
  • Alkaptonuria causes buildup of homogentisic acid leading to joint problems, ochronosis, and complications in kidneys and heart, with the medication expected to launch in July 2025.
  • The approval represents a significant breakthrough for patients who previously had no approved therapeutic options for this debilitating condition.

Long-Term Study of Nitisinone to Treat Alkaptonuria

NCT00107783CompletedPhase 2
National Human Genome Research Institute (NHGRI)
Posted 1/1/2005

ExCellThera's Zemcelpro Receives Positive CHMP Opinion for Blood Cancer Patients Without Suitable Donors

Orphan Drug
  • The European Medicines Agency's CHMP has recommended conditional marketing authorization for Zemcelpro, a novel stem cell therapy for blood cancer patients lacking suitable donor cells.
  • If approved, Zemcelpro would become the first and only EU-authorized therapy for adults with haematological malignancies requiring stem cell transplantation when no suitable donor cells are available.
  • The therapy addresses a significant unmet need, as over 10,000 new cases of patients with blood cancers requiring bone marrow transplant occur annually in Europe.
  • European Commission approval is expected within two months, with additional regulatory filings planned for the US, Canada, UK, and Switzerland.

Expanded Cord Blood in Patients in Need of an Allogeneic Stem Cell Transplant

NCT02668315CompletedPhase 1
Maisonneuve-Rosemont Hospital
Posted 12/16/2015

US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia

NCT04103879Active, Not RecruitingPhase 2
ExCellThera inc.
Posted 11/13/2020

Brenus Pharma and InSphero Develop Advanced 3D Tumor Models for Colorectal Cancer Immunotherapy Testing

  • Brenus Pharma and InSphero presented promising data at the European Association for Cancer Research 2025 congress confirming the use of 3D tumor spheroids for potency testing of STC-1010, their lead colorectal cancer immunotherapy candidate.
  • The collaboration developed vitrified 3D colorectal cancer spheroids using tumor cell lines co-cultured with fibroblasts, preserving tumor morphology and stromal integrity to better mimic in vivo human tumor conditions.
  • Previous studies demonstrated that STC-1010 antigens were effectively processed by dendritic cells, activating CD8+ T cells that induced apoptosis in both 2D and 3D colorectal cancer models while releasing anti-tumor cytokines.
  • The European-funded collaboration represents a significant advancement in translational immunotherapy development, enabling more realistic evaluation of therapeutic potential through scalable 3D tumor models.

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