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Clinical Trial News

PANORAMIC Trial Launches to Evaluate Oral Antivirals for Early COVID-19 Treatment

  • The PANORAMIC trial, a UK-wide study, is assessing the effectiveness of novel oral antiviral treatments for COVID-19 in non-hospitalized patients.
  • Participants must have a positive COVID-19 test and be within five days of symptom onset, with specific age and health condition criteria.
  • The trial will evaluate antivirals like molnupiravir against standard care to understand their impact in a highly vaccinated population.
  • The study aims to provide evidence for the NHS to optimize the use of COVID-19 antivirals, potentially reducing hospitalizations and easing NHS burden.

Caribou Biosciences to Present Preclinical Data on CB-010 for Lupus at ACR Convergence 2024

Caribou Biosciences, Inc. will showcase preclinical data supporting the development of CB-010, an allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout, for treating lupus nephritis and extrarenal lupus at the American College of Rheumatology Convergence 2024.

CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy for Relapsed/Refractory B Cell Non-Hodgkin Lymphoma (ANTLER)

NCT04637763RecruitingPhase 1
Caribou Biosciences, Inc.
Posted 5/26/2021

Acelyrin Prioritizes Lonigutamab for Thyroid Eye Disease After Positive Phase 1/2 Data

• Acelyrin focuses on lonigutamab for Thyroid Eye Disease (TED) after promising Phase 1/2 results, with Phase 3 trials expected to begin in Q1 2025. • Lonigutamab shows advantages over anti-IGF-1R therapies, including no hyperglycemia, lower Cmax for reduced hearing loss risk, and higher potency at lower doses. • Acelyrin halts izokibep development for Hidradenitis Suppurativa (HS) and Psoriatic Arthritis (PsA), and reduces workforce by 33% to streamline operations. • Piper Sandler maintains an Overweight rating with a $20.00 price target for Acelyrin, citing lonigutamab's potential and validated anti-IGF-1R mechanism.

IDHIFA Clinical Trial Induces Remission in AML Patient with IDH2 Mutation

  • A 33-year-old patient with acute myeloid leukemia (AML) achieved remission through a clinical trial using IDHIFA, an oral medication targeting the IDH2 mutation.
  • The patient, who initially underwent intensive remission induction therapy with chemotherapy, opted for the IDHIFA trial as an alternative to a bone marrow transplant.
  • IDHIFA works by turning off the mutated IDH2 gene, restoring normal cell function and offering a targeted approach to treating AML in patients with this specific mutation.
  • The patient experienced minimal side effects and has returned to work and normal activities, highlighting the potential of targeted gene therapy in leukemia treatment.

RHEACELL's Allo-APZ2-CVU Receives FDA Approval for Phase 3 Trial in Refractory Chronic Venous Ulcers

  • RHEACELL's allo-APZ2-CVU, a first-in-class stem cell therapy, has received FDA approval for a Phase 3 trial (NCT06489028) to treat chronic venous ulcers (CVU).
  • The pivotal, multi-center trial will enroll 250 patients across 100 sites to assess the efficacy and safety of allo-APZ2-CVU in therapy-resistant, non-healing CVU.
  • Allo-APZ2-CVU leverages ABCB5-positive mesenchymal stem cells to modulate the immune response and promote wound healing in patients with severe, inflammation-driven CVU.
  • The Phase 3 approval follows positive Phase 2b results, which demonstrated a favorable safety profile and preliminary efficacy compared to placebo in refractory CVU patients.

CLINICAL STUDY TO TEST STEM CELLS FOR THE TREATMENT OF LONG-TERM, NON-HEALING VENOUS LEG ULCERS

2024-512720-11-00RecruitingPhase 3
Rheacell GmbH & Co. KG

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of CVU (Phase IIb)

NCT04971161RecruitingPhase 2
RHEACELL GmbH & Co. KG
Posted 8/18/2021

Streamlined Regulatory Pathways Enhance Clinical Trial Efficiency in the UK

  • The UK's Health Research Authority (HRA) streamlines research approvals through the Integrated Research Application System (IRAS), reducing administrative burden for clinical trials.
  • Standardized protocol templates, aligned with the SPIRIT Statement, ensure comprehensive and consistent trial design, facilitating ethical review and regulatory compliance.
  • The Research Passport system expedites multicenter studies by simplifying researcher movement between NHS Trusts, fostering collaboration and accelerating research progress.

UroGen Pharma Secures $25M Loan as UGN-102 Awaits Potential FDA Approval

• UroGen Pharma obtained a $25 million loan to support its corporate and working capital needs, with the loan's terms tied to FDA approval of UGN-102. • The loan's interest rate is based on the three-month Secured Overnight Financing Rate (SOFR) plus 7.25%, with potential for term extension upon FDA approval. • UroGen Pharma has completed its New Drug Application (NDA) to the FDA for UGN-102, a treatment for non-muscle invasive bladder cancer, after positive Phase 3 trial results. • Analysts remain confident in UroGen's prospects, anticipating UGN-102 approval and launch, projecting potential sales exceeding $1 billion.

FDA Approves Pfizer's Hympavzi (Marstacimab) for Hemophilia A and B

  • The FDA has approved Hympavzi (marstacimab) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in hemophilia A and B.
  • Hympavzi is the first anti-tissue factor pathway inhibitor approved in the U.S. for hemophilia A or B, offering a novel mechanism of action.
  • Clinical trials demonstrated Hympavzi significantly reduced annualized bleeding rates compared to both on-demand and routine prophylactic treatments.
  • The drug is administered via a convenient once-weekly subcutaneous injection, providing an alternative to frequent intravenous infusions.

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

NCT03938792CompletedPhase 3
Pfizer
Posted 3/9/2020

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

NCT05145127RecruitingPhase 3
Pfizer
Posted 11/17/2021

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

NCT05611801RecruitingPhase 3
Pfizer
Posted 12/9/2022

Lupin Receives FDA Approval for Generic Bumetanide Injection

  • Lupin has secured FDA approval for its bumetanide injection, available in single and multiple-dose vials, offering a generic alternative to Validus Pharmaceuticals' Bumex.
  • The bumetanide injection is indicated for treating edema linked to congestive heart failure, hepatic, and renal diseases, including nephrotic syndrome.
  • Manufactured at Lupin's Nagpur facility in India, the generic bumetanide injection addresses a market valued at approximately $20 million, according to IQVIA data from July 2024.

Nivolumab and Ipilimumab Combination Therapy Shows Promise in Advanced Merkel Cell Carcinoma

  • A phase II clinical trial demonstrated that combined nivolumab and ipilimumab, with or without stereotactic body radiotherapy, halted the progression of advanced Merkel cell carcinoma.
  • The study showed a 41% complete response rate in patients treated with the combination immunotherapy and stereotactic radiation arm.
  • The treatment offers a potential first-line therapy or an option after previous anti-PD-1 and anti-PD-L1 monotherapy failure for this aggressive skin cancer.
  • Patients in the trial experienced improved quality of life, allowing them to pursue personal interests and contribute to their communities.

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