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Clinical Trial News

Cabaletta Bio to Present New CABA-201 Clinical Data at ACR Convergence 2024

  • Cabaletta Bio will present new clinical data on CABA-201, a CD19-CAR T cell therapy, at the ACR Convergence 2024 in Washington, D.C.
  • The presentations will cover the safety and efficacy of CABA-201 in patients with immune-mediated necrotizing myopathy and systemic lupus erythematosus.
  • Correlative studies of CABA-201 in myositis and SLE will also be presented, offering insights into the therapy's mechanism of action.
  • CABA-201 is being evaluated across multiple autoimmune conditions, holding promise for durable remission without chronic therapy.

RenovoRx Boosts RenovoCath Production Amid Rising Demand for Targeted Oncology Therapies

  • RenovoRx increases RenovoCath production due to high demand for targeted diagnostic and therapeutic agent delivery by oncologists and interventional radiologists.
  • A new agreement with Medical Murray expands manufacturing capacity, including potential stock warrants based on milestone achievements.
  • RenovoRx remains focused on the Phase III TIGeR-PaC trial for locally advanced pancreatic cancer (LAPC) using TAMP therapy with gemcitabine HCl.
  • The company is exploring commercial opportunities for RenovoCath as a standalone device, with potential revenue generation expected in 2025.

WHO Guidance Aims to Broaden Diversity in Clinical Trials Beyond Race and Gender

  • The WHO has released new guidance to broaden diversity in clinical trials, moving beyond race and gender to include other underrepresented populations.
  • The guidance emphasizes the inclusion of pregnant and lactating women, patients from lower socioeconomic backgrounds, and those facing language barriers.
  • WHO's recommendations include strengthening clinical trial infrastructure in low- and middle-income countries to promote more equitable access.
  • Experts highlight the need for regulatory bodies like the FDA and EMA to adopt similar guidelines to ensure a significant impact on trial design.

Pre-Exposure Prophylaxis Study of Lenacapavir and Emtricitabine/Tenofovir Alafenamide in Adolescent Girls and Young Women at Risk of HIV Infection

NCT04994509Active, Not RecruitingPhase 3
Gilead Sciences
Posted 8/30/2021

Pfizer Withdraws Sickle Cell Disease Drug Oxbryta Globally Due to Safety Concerns

  • Pfizer has voluntarily withdrawn Oxbryta (voxelotor) from all global markets due to safety concerns, citing an imbalance in vaso-occlusive crises and fatal events.
  • The decision follows a review of clinical data that indicated the overall benefit of Oxbryta no longer outweighs the risks for sickle cell disease patients.
  • Regulatory agencies, including the EMA and FDA, have alerted healthcare professionals and patients about the withdrawal, advising discussions on alternative treatments.
  • The withdrawal impacts ongoing clinical trials and expanded access programs, leaving the sickle cell community seeking alternative treatment options and raising concerns about trust.

Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

NCT03036813CompletedPhase 3
Pfizer
Posted 12/1/2016

Resolution of Sickle Cell Leg Ulcers With Voxelotor

NCT05561140TerminatedPhase 3
Pfizer
Posted 5/30/2022

A Phase 2/3 Study in Adult and Pediatric Participants With SCD

NCT05431088Active, Not RecruitingPhase 2
Pfizer
Posted 9/22/2022

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

NCT04218084TerminatedPhase 3
Pfizer
Posted 11/11/2020

A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

NCT04927247TerminatedPhase 3
Pfizer
Posted 12/9/2021

Synairgen Advances SNG001 Towards Phase II Trial for COPD

  • Synairgen is progressing towards a Phase II trial of SNG001, its lead asset, for the treatment of COPD, marking a significant step in its development program.
  • The company reported advancements in its clinical and manufacturing strategies during the first half of the year, supporting the upcoming trial.
  • SNG001, an inhaled formulation of interferon beta, aims to reduce exacerbations and improve lung function in COPD patients by addressing viral-induced lung damage.
  • Synairgen is focused on securing partnerships and funding to support the Phase II trial and further development of SNG001.

Elixirgen Therapeutics Receives FDA Rare Pediatric Disease Designation for EXG-34217 in Dyskeratosis Congenita

  • Elixirgen Therapeutics received FDA Rare Pediatric Disease Designation for EXG-34217, a treatment for dyskeratosis congenita and related telomere biology disorders (DC/TBD).
  • EXG-34217 consists of autologous CD34+ hematopoietic stem cells treated with EXG-001, encoding human ZSCAN4, using a Sendai virus vector.
  • The FDA's designation acknowledges the significant unmet need for DC/TBD therapies and the potential of EXG-34217 to address this rare condition.
  • Elixirgen is currently enrolling patients in a Phase 1/2 trial at Cincinnati Children’s Hospital Medical Center to evaluate EXG-34217.

Safety and Immunogenicity of EXG-5003

NCT04863131CompletedPhase 1
Fujita Health University
Posted 4/28/2021

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

NCT04211714RecruitingPhase 1
Elixirgen Therapeutics, Inc.
Posted 4/8/2021

Cidara Therapeutics Presents Positive Clinical Data on CD388 for Influenza Prevention

  • Cidara Therapeutics presented safety and pharmacokinetic data from CD388 clinical trials at the OPTIONS XII conference.
  • The data supports CD388's safety profile across various doses and administration routes in healthy subjects.
  • Pharmacokinetic results suggest CD388 could provide season-long influenza A and B protection in diverse populations.
  • Cidara plans to advance CD388 into a Phase 2b trial for the Northern Hemisphere influenza season in 2024.

Palisade Bio Advances PALI-2108 for Ulcerative Colitis with Phase 1 Trial Imminent

  • Palisade Bio is set to initiate a Phase 1 clinical trial for PALI-2108, a PDE4 inhibitor prodrug, targeting moderate to severely active ulcerative colitis (UC).
  • The Phase 1 trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of PALI-2108 in healthy volunteers and UC patients.
  • Palisade Bio is developing precision medicine tests using PDE4B expression to identify patients most likely to respond to PALI-2108, potentially improving treatment outcomes.
  • A pre-Clinical Trial Application meeting with Health Canada has been completed, and the CTA for the Phase 1 study has been submitted.

Personalized Medicine and Genetic Markers Drive Major Advances in Pediatric Cancer Treatment

  • Pediatric cancer treatment has achieved remarkable success with cure rates reaching 85%, thanks to nationwide institutional collaboration and development of cooperative treatment groups.
  • Advanced genetic marker analysis enables personalized treatment approaches for pediatric cancers, leading to improved outcomes in brain tumors, kidney cancers, and hematologic malignancies.
  • Implementation of immunotherapy has allowed reduction in conventional therapy usage, with leukemia cure rates increasing from 85% to over 95% while minimizing long-term adverse effects.

CellBion's Lu-177-DGUL Shows Promise in Metastatic Prostate Cancer Treatment

  • CellBion is advancing Lu-177-DGUL, a radiopharmaceutical, for metastatic castration-resistant prostate cancer (mCRPC), aiming for conditional approval in Korea by late 2025.
  • Phase 2 trial interim results indicate a 38.5% objective response rate for Lu-177-DGUL, potentially surpassing Novartis' Pluvicto, which reported 28.9%.
  • CellBion emphasizes Lu-177-DGUL's improved safety profile due to its enhanced metabolic stability and faster elimination from the body via kidney excretion.
  • The company plans global expansion through technology exports and is strategically securing patents in multiple countries to solidify its market position.

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