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Clinical Trial News

Viral Infections Emerge as Critical Consideration in Rheumatic Disease Management

  • Dr. Leonard Calabrese highlights the increasing intersection between virology and rheumatology, emphasizing the importance of recognizing viral forms of arthritis in clinical practice.
  • Viral infections, including parvovirus and arboviruses, can trigger rheumatic symptoms, while biologics may reactivate dormant viruses like hepatitis B, necessitating careful screening protocols.
  • Healthcare providers must maintain vigilance in screening rheumatic disease patients for HIV, hepatitis B, and hepatitis C before initiating immunosuppressive or targeted therapies.

Coverage Disputes Arise for Sarepta's Duchenne Gene Therapy Elevidys Despite FDA Approval

  • Insurers are denying coverage for Sarepta's Elevidys, a gene therapy for Duchenne muscular dystrophy, despite its FDA approval for a broad patient population.
  • Coverage denials often stem from insurers aligning policies with clinical trial enrollment criteria, which focused on ambulatory patients, creating access barriers for non-ambulatory individuals.
  • The high cost of Elevidys, priced at $3.2 million, is a significant factor in coverage disputes, with insurers questioning the cost-effectiveness for patients with limited mobility.
  • Patient advocacy groups and medical professionals are pushing back against restrictive insurance policies, emphasizing the potential for Elevidys to prolong life and preserve function, even in wheelchair-bound patients.

Urteste Expands Cancer Diagnostic Pipeline with Focus on Early Detection

  • Urteste is expanding its diagnostic projects, leveraging its technology to detect cancer from urine samples with high sensitivity and specificity.
  • The company received a $3 million grant to develop NASTRO, a breast cancer diagnostic test, aiming for a non-invasive early detection method.
  • Urteste's PANURI test for pancreatic cancer demonstrated 95.6% sensitivity and 95.5% specificity in a proof-of-concept study.
  • Urteste is developing a portfolio of 12 diagnostic tests for various cancers, including pancreatic, breast, lung and colorectal cancers.

Genprex Advances Reqorsa Gene Therapy for Mesothelioma Treatment

  • Genprex is expanding its research program for Reqorsa, an investigational immunogene therapy, to include mesothelioma, a cancer with limited treatment options.
  • A Mesothelioma Clinical Advisory Board comprising world-renowned researchers has been formed to guide the development of Reqorsa for mesothelioma.
  • Reqorsa delivers the TUSC2 tumor suppressor gene via intravenous injection, showing promise in preclinical studies for mesothelioma, where TUSC2 expression is often reduced.
  • While expanding into mesothelioma, Genprex discontinued enrollment in the Acclaim-2 trial for NSCLC due to enrollment challenges amid a competitive clinical trial landscape.

Quaratusugene Ozeplasmid (Reqorsa) and Atezolizumab Maintenance Therapy in ES-SCLC Patients

NCT05703971RecruitingPhase 1
Genprex, Inc.
Posted 5/9/2024

Quaratusugene Ozeplasmid (Reqorsa) and Osimertinib in Patients With Advanced Lung Cancer Who Progressed on Osimertinib

NCT04486833RecruitingPhase 1
Genprex, Inc.
Posted 9/3/2021

Quaratusugene Ozeplasmid (Reqorsa) in Combination With Pembrolizumab in Previously Treated Non-Small Lung Cancer

NCT05062980TerminatedPhase 1
Genprex, Inc.
Posted 3/30/2022

FDA Approves Otulfi, a Stelara Biosimilar, for Inflammatory Conditions

  • The FDA has approved Otulfi (ustekinumab-aauz) as a biosimilar to Stelara, offering a new treatment option for Crohn's disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis.
  • Otulfi, developed by Formycon and Fresenius Kabi, demonstrated comparable efficacy, safety, and immunogenicity to Stelara in clinical trials.
  • Fresenius Kabi plans to launch Otulfi in the U.S. by February 2025, following a settlement agreement with Johnson & Johnson, potentially increasing access to treatment.
  • The approval expands Fresenius Kabi's biosimilar portfolio and aligns with their strategy to become a significant player in the biopharmaceutical field globally.

Pharmacokinetics, Safety and Tolerability Study of AVT04 to EU Approved and US Licensed Stelara (Ustekinumab)

NCT04744363CompletedPhase 1
Alvotech Swiss AG
Posted 5/25/2021

A Double-blind Study to Compare the Efficacy, Safety, and Immunogenicity of the Proposed Biosimilar Ustekinumab FYB202 to Stelara® in Patients With Moderate-to-Severe Plaque Psoriasis

NCT04595409CompletedPhase 3
Bioeq GmbH
Posted 11/9/2020

Xbrane Receives FDA Concurrence on Streamlined Development Plan for Opdivo Biosimilar, Xdivane

  • Xbrane Biopharma received positive scientific advice from the FDA regarding its Opdivo biosimilar candidate, Xdivane, aligning with EMA feedback.
  • The FDA supports Xbrane's streamlined clinical development plan, which includes a single clinical trial, significantly reducing the development budget by at least 60%.
  • Xbrane is focusing on a global market strategy for Xdivane, targeting US launch by December 2028, and is actively seeking commercialization partners.
  • Opdivo, an immune-oncology drug (PD-1 inhibitor), had global sales of approximately $8 billion in 2023, projected to reach $14 billion by 2028.

PSMA-Targeted Radiopharmaceuticals Transform Advanced Prostate Cancer Treatment Landscape

  • Radiopharmaceuticals, particularly PSMA-targeted therapies like Pluvicto (lutetium-177), are showing promise in treating advanced prostate cancer by delivering targeted radiation to cancer cells while minimizing damage to healthy tissue.
  • PSMA PET imaging has revolutionized prostate cancer detection, offering superior sensitivity in identifying metastatic sites and helping clinicians make more informed treatment decisions.
  • Clinical trials are exploring the potential of new alpha-emitting radiopharmaceuticals like actinium-225, which may overcome tumor resistance mechanisms and provide treatment options for PSMA-negative patients.

Shionogi Submits Zuranolone NDA in Japan for Major Depressive Disorder

  • Shionogi has submitted a New Drug Application (NDA) in Japan for zuranolone, targeting the treatment of major depressive disorder (MDD).
  • The NDA is supported by Phase 3 trial data in Japan, which demonstrated statistically significant improvements in MDD symptoms.
  • Zuranolone, a neuroactive steroid GABA-A receptor modulator, has shown rapid efficacy, potentially addressing the need for faster-acting treatments.
  • Zuranolone is already approved in the U.S. for postpartum depression and could offer a novel treatment option for MDD in Japan.

Anakinra Shows Promise in Reducing Endometriosis-Related Pain and Inflammation in Pilot Study

  • A pilot study suggests that anakinra, an IL-1 antagonist, may reduce dysmenorrhea pain scores in women with endometriosis.
  • The study also found statistically significant improvements in quality of life, specifically in the domains of powerlessness and self-image.
  • Anakinra did not disrupt menstrual cycles, suggesting it may preserve fertility potential, a key advantage over current treatments.
  • Biomarker analysis revealed significantly lower levels of BDNF, an inflammatory marker, indicating a potential anti-inflammatory effect.

Pilot Study of the IL-1 Antagonist Anakinra for the Treatment of Endometriosis Related Symptoms

NCT03991520Unknown StatusEarly Phase 1
University of California, San Diego
Posted 6/16/2020

Travere Therapeutics Pauses Enrollment in Phase 3 Pegtibatinase Trial for Homocystinuria Due to Manufacturing Issues

  • Travere Therapeutics has voluntarily paused enrollment in its Phase 3 HARMONY study of pegtibatinase for classical homocystinuria (HCU).
  • The pause is due to the company addressing manufacturing scale-up to support commercial production and full enrollment in the HARMONY study.
  • Enrollment is expected to resume in 2026, with R&D expenses projected to decrease by over $30 million in 2025 compared to 2024.
  • Patients currently enrolled in pegtibatinase studies will continue to receive medication from unaffected small-scale batches.

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