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Clinical Trial News

Prothena Cuts 63% of Workforce as Company Restructures Operations Amid Pipeline Milestones

  • Prothena Corporation announced a 63% workforce reduction to substantially reduce operating costs while maintaining support for remaining wholly owned programs and partnered collaborations.
  • The restructuring comes as multiple pipeline milestones approach, including Roche advancing prasinezumab to Phase 3 for Parkinson's disease and expected PRX012 Alzheimer's data in August 2025.
  • The company revised its 2025 financial guidance, expecting net cash burn of $170-178 million and ending the year with approximately $298 million in cash reserves.
  • Prothena's board and management are evaluating comprehensive business options while maintaining obligations to partnered programs that could generate up to $105 million in clinical milestones by 2026.

CD5-Targeted Therapies Show Promise Across Cancer, Autoimmunity, and Transplantation with First Commercial Launch Expected by 2030

CAR-TMonoclonal Antibody
  • CD5-targeted therapies are advancing through clinical development with fewer than 10 therapies currently in trials and the highest phase reaching Phase II, with first commercial approval expected by 2030.
  • Leading CAR-T candidates MB-105 and VIPER-101 demonstrate acceptable safety profiles and encouraging antitumor activity in relapsed and refractory T-cell malignancies.
  • CD5's therapeutic versatility extends beyond oncology to autoimmune disorders and transplantation medicine, offering potential to modulate immune responses across multiple disease contexts.
  • Despite promising early results, challenges remain including fratricide effects in cancer therapy, immune balance in autoimmunity, and limited long-term safety data for novel approaches.

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

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