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Clinical Trial News

Amgen's Pipeline Shows Promise with MariTide and Expanding Drug Portfolio

• Amgen is focusing on new drug launches, including the cancer drug Lumakras, to replace revenue from older medications. • Tepezza, acquired by Amgen last year, is the first FDA-approved treatment for thyroid eye disease (TED) and is expected to perform strongly, particularly in international markets. • MariTide, currently in phase two trials, shows potential in the weight loss market and may also have applications in treating diabetes, with analysts projecting significant revenue. • Amgen has a robust pipeline featuring over 30 phase 3 programs, indicating a strong focus on future growth and innovation in multiple therapeutic areas.

Beqvez Gene Therapy Shows Sustained Reduction in Bleeding for Hemophilia B Patients

  • A single infusion of Beqvez (fidanacogene elaparvovec) gene therapy resulted in a 71% reduction in bleeding episodes in hemophilia B patients.
  • Over half of the patients in the study experienced no bleeding episodes following the gene therapy treatment.
  • The gene therapy delivers a functional copy of the factor IX gene to the liver, enabling the body to produce the necessary clotting factor.
  • Beqvez, developed by Pfizer, has been approved by the FDA, offering a potentially life-changing, single-dose alternative to regular factor IX infusions.

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

NCT03861273Active, Not RecruitingPhase 3
Pfizer
Posted 7/29/2019

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291CompletedPhase 2
CSL Behring
Posted 7/24/2018

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

NCT03370913CompletedPhase 3
BioMarin Pharmaceutical
Posted 12/19/2017

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

NCT03587116CompletedPhase 3
Pfizer
Posted 7/26/2018

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891CompletedPhase 3
CSL Behring
Posted 6/27/2018

BioCity's SC0062 Receives Breakthrough Therapy Designation for IgA Nephropathy

  • BioCity Biopharma's SC0062, a selective endothelin type A (ETA) antagonist, has been granted Breakthrough Therapy Designation (BTD) by the NMPA.
  • The BTD was based on positive Phase 2 trial results in IgA nephropathy (IgAN) patients with proteinuria, demonstrating a statistically significant reduction in proteinuria.
  • SC0062 showed a favorable safety profile, including a lower incidence of peripheral edema, and is being advanced into Phase 3 trials for IgAN and other CKDs.
  • SC0062's high selectivity for ETA over ETB suggests potential advantages over non-selective ET antagonists in reducing CKD progression with improved safety.

Kuhnil Pharmaceutical Launches First Pediatric Insomnia Drug in Korea

  • Kuhnil Pharmaceutical has launched Slenyto SR Tablet, the first officially licensed insomnia treatment for children and adolescents in Korea.
  • Clinical trials showed Slenyto increased sleep duration by 40 minutes at week 3 compared to placebo, with sustained improvement over 52 weeks.
  • Parents reported improved sleep satisfaction and quality of life with Slenyto treatment, with no significant adverse events observed in trials.
  • Slenyto is approved for children and adolescents aged 2-18 with Autism Spectrum Disorders (ASD) and Smith-Magenis syndrome (SMS).

Cambridge University Hospitals Plays Key Role in International Cancer Studies

  • Cambridge University Hospitals (CUH) has played a crucial role in early-phase trials, contributing to advancements in gynecological cancer care and innovation.
  • The ESMO Congress, a globally recognized oncology platform, facilitates the dissemination of cutting-edge data and provides networking opportunities for oncologists worldwide.
  • CUH's involvement in multiple international conferences underscores its commitment to advancing cancer research and improving patient outcomes through collaborative efforts.
  • Positive results from these trials will support further development work at CUH, solidifying its position as a leader in gynecological care innovation.

BioAge Labs Raises $198 Million in IPO to Advance Azelaprag for Obesity

  • BioAge Labs successfully completed its IPO, raising $198 million to support the clinical development of azelaprag and other pipeline programs.
  • Azelaprag, an apelin receptor agonist, is being investigated in a Phase 2 trial in combination with Eli Lilly's Zepbound for obesity, with preliminary results expected in Q3 2025.
  • The company aims to develop an all-oral combination therapy for obesity and is also planning a Phase 2 study of azelaprag with Novo Nordisk's Wegovy.
  • BioAge's technology platform identifies drug targets related to aging, with a focus on preserving metabolic health and addressing neuroinflammation.

ARTHEx Biotech to Present at Chardan Genetic Medicines Conference

  • ARTHEx Biotech will participate in the 8th Annual Chardan Genetic Medicines Conference, focusing on genetic medicine advancements.
  • The company's CEO, Frédéric Legros, will engage in one-on-one meetings with investors during the conference in New York.
  • ARTHEx Biotech is currently evaluating ATX-01 for Myotonic Dystrophy Type 1 (DM1) in a Phase I-IIa trial.
  • ARTHEx Biotech is advancing its discovery engine to develop microRNA modulators for genetically-driven diseases with unmet needs.

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

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