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Clinical Trial News

UGN-102 Shows Durable Response in Low-Grade Bladder Cancer

• UroGen's UGN-102 demonstrates robust durability of response in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). • The ATLAS trial data showed high 12-month duration of response rates in both new (87.5%) and recurrent (69.1%) LG-IR-NMIBC patients treated with UGN-102. • Disease-free survival rates at 15 months post-randomization were 77.4% in new and 63.2% in recurrent patients treated with UGN-102 in the ATLAS trial. • UroGen plans to submit a New Drug Application (NDA) for UGN-102 in September 2024, with potential FDA decision in early 2025, pending positive data from the ENVISION trial.

A Phase 3 Study of UGN-102 for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

NCT04688931TerminatedPhase 3
UroGen Pharma Ltd.
Posted 2/19/2021

A Phase 3 Single-Arm Study of UGN-102 for Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

NCT05243550Active, Not RecruitingPhase 3
UroGen Pharma Ltd.
Posted 3/1/2022

Fresenius Kabi Launches First Actemra® Biosimilar in U.S. Market as Biosimilar Landscape Expands

Drug ApprovalOncologyMonoclonal Antibody
• Fresenius Kabi has launched Tyenne® (tocilizumab-aazg), the first biosimilar of Actemra® to enter the U.S. market, following FDA approval in March 2024 for both intravenous and subcutaneous formulations.
• Accord BioPharma received FDA approval for Hercessi™ (trastuzumab-strf), becoming the sixth Herceptin® biosimilar approved in the U.S., while Xbrane's Xlucane™ (ranibizumab) received a Complete Response Letter from the FDA.
• The expanding biosimilar market continues to challenge reference products with significant annual sales, with Actemra® generating approximately $3 billion, Herceptin® $1.77 billion, and Lucentis® $1.475 billion in 2023.

NHS Approves Oxbryta for Sickle Cell Disease Following Price Reduction Agreement

• NICE has approved Pfizer's Oxbryta (voxelotor) for treating sickle cell disease-related anemia in patients aged 12 and over, potentially benefiting 4,000 people in England.
• The breakthrough approval comes after Pfizer agreed to reduce the drug's price, making it cost-effective for NHS funding through the Innovative Medicines Fund.
• Oxbryta represents a significant advancement as the first European-approved medicine that directly prevents sickle hemoglobin polymerization, addressing the root cause of red blood cell destruction.

FDA Approves Lifileucel (Amtagvi): First TIL Therapy for Melanoma

  • The FDA has granted accelerated approval to lifileucel (Amtagvi) for advanced melanoma after prior therapies, marking the first tumor-infiltrating lymphocyte (TIL) therapy approval.
  • Lifileucel is a personalized immunotherapy that uses a patient's own T cells extracted from their tumor to target and destroy cancer cells.
  • Clinical trials demonstrated that nearly one-third of patients treated with lifileucel experienced tumor reduction, with some achieving complete remission and durable responses.
  • Ongoing research focuses on combining lifileucel with other immunotherapies and expanding its use to other solid tumors, such as lung and ovarian cancers.

Study of Lifileucel (LN-144), Autologous Tumor Infiltrating Lymphocytes, in the Treatment of Patients With Metastatic Melanoma

NCT02360579CompletedPhase 2
Iovance Biotherapeutics, Inc.
Posted 9/24/2015

Study to Investigate Lifileucel Regimen Plus Pembrolizumab Compared With Pembrolizumab Alone in Participants With Untreated Advanced Melanoma.

NCT05727904RecruitingPhase 3
Iovance Biotherapeutics, Inc.
Posted 3/30/2023

Sarepta's DMD Gene Therapy Approval Faces Scrutiny Over Efficacy and Pricing

  • An article in JAMA questions the FDA's accelerated approval of Sarepta's Elevidys for Duchenne muscular dystrophy (DMD) due to efficacy concerns.
  • Elevidys, priced at $3.1 million per dose, failed to meet its primary endpoint in Phase I and Phase III EMBARK studies, raising cost-effectiveness questions.
  • The FDA's reliance on micro-dystrophin expression as a surrogate marker for clinical benefit is debated, as it may not directly correlate with functional improvements.
  • The FDA is set to decide on Sarepta's supplemental Biologics License Application for Elevidys by June 21, 2024, amidst ongoing controversy.

ICH E6(R3) Guidelines Set to Transform Clinical Trial Quality Management in 2025

  • The International Council for Harmonisation (ICH) has released draft E6(R3) guidelines aimed at modernizing Good Clinical Practice standards, with final implementation expected in 2025.
  • The new guidelines introduce a risk-proportionate approach to clinical trial management, replacing rigid Quality Tolerance Limits with more flexible 'Acceptable Ranges' to enhance trial efficiency.
  • E6(R3) addresses the challenges of managing increasing data volumes in clinical trials, with studies now handling up to 6 million data points and multiple data sources.

Immutep's Triple Combination Therapy Shows Promising Results in Soft Tissue Sarcoma Phase II Trial

  • Immutep's EFTISARC-NEO Phase II trial demonstrates that 67% of patients achieved near-complete pathologic responses with eftilagimod alpha combined with radiotherapy and pembrolizumab.
  • The novel triple combination therapy was well tolerated with no new safety findings in the first six patients who completed 10 weeks of treatment.
  • This represents the first clinical evaluation of efti in a neoadjuvant setting and the first combination with radiotherapy for soft tissue sarcoma patients.
  • The encouraging results address a significant unmet medical need in soft tissue sarcoma, an orphan disease with poor prognosis affecting approximately 13,400 new patients annually in the United States.

Pembrolizumab in Combination with Eftilagimod Alpha and Radiotherapy in Neoadjuvant Treatment of Patients with Soft Tissue Sarcoma - EFTISARC-NEO Trial

NCT06128863Active, Not RecruitingPhase 2
Maria Sklodowska-Curie National Research Institute of Oncology
Posted 7/17/2023

Atsena Therapeutics Reports Promising Results for XLRS Gene Therapy Using Novel Spreading Capsid

Rare Disease
  • Atsena Therapeutics' ATSN-201 gene therapy showed positive safety and early efficacy in the first cohort of XLRS patients, with two of three patients experiencing extensive schisis resolution beginning at 8 weeks post-treatment.
  • The trial demonstrated clinical validation of AAV.SPR's ability to spread laterally beyond subretinal injection sites, achieving therapeutic effects without requiring risky foveal detachment procedures.
  • Functional improvements were observed using microperimetry, with one patient showing improvements up to 14 dB and 38 loci improving by more than 7 dB, exceeding the FDA's threshold for clinical significance.

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

NCT05878860RecruitingPhase 1
Atsena Therapeutics Inc.
Posted 8/22/2023

FDA Issues New Draft Guidance on Allogeneic Cell-Based Medical Products

The FDA has released a new draft guidance titled 'Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products', aimed at assisting sponsors in determining appropriate cell safety testing for investigational new drug applications or biologics license applications. The guidance applies to allogeneic cell-based products regulated by CBER's Office of Therapeutic Products, including genetically modified cells, and excludes cell substrates used in manufacturing non-cell-based products. Comments on the draft are accepted until July 29, 2024.

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