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Clinical Trial News

Innovent's Picankibart NDA Accepted in China for Plaque Psoriasis Treatment

  • Innovent's New Drug Application (NDA) for picankibart, an anti-IL-23p19 antibody, has been accepted by China's NMPA for treating moderate to severe plaque psoriasis.
  • Phase 3 trial data showed over 80% of subjects achieved PASI 90 after 16 weeks, with a convenient maintenance dosing interval of once every 12 weeks.
  • The NDA acceptance is based on the CLEAR-1 study, which demonstrated significantly higher rates of skin lesion clearance and improved quality of life compared to placebo.
  • Picankibart represents the first IL-23p19 antibody drug independently developed by a Chinese enterprise to submit an NDA in China.

A Study to Evaluate IBI112 in the Treatment of Psoriasis With Moderate to Severe Plaque Psoriasis

NCT05645627CompletedPhase 3
Innovent Biologics (Suzhou) Co. Ltd.
Posted 2/10/2023

Calliditas Therapeutics Pioneered Novel Endpoint for IgAN Drug Approval

  • Calliditas Therapeutics utilized a novel renal primary surrogate endpoint, proteinuria reduction, in a Phase III trial for IgA nephropathy (IgAN).
  • This approach enabled accelerated FDA approval for Calliditas's nephrology drug, a first in the field, setting a new precedent for rare renal disease treatments.
  • CEO Renee Aguiar-Lucander emphasized the importance of this novel endpoint in overcoming the challenges of traditional endpoints like kidney failure in rare diseases.
  • Calliditas has expanded globally, growing into a fully integrated biopharma company with a strong commercial presence in the U.S. and partnerships in key markets.

Inbiome's Molecular Culture ID Receives FDA Breakthrough Device Designation for Rapid Bacterial Diagnostics

  • Inbiome's Molecular Culture ID has received FDA Breakthrough Device Designation, recognizing its potential to transform infectious disease diagnostics.
  • The diagnostic test combines advanced chemistry and AI to rapidly detect over 200 bacterial species from bodily samples, providing same-day results.
  • Inbiome plans to introduce Molecular Culture ID to the U.S. market by early 2026, partnering with leading hospitals for implementation studies.
  • The technology aims to combat antimicrobial resistance by ensuring appropriate and effective antibiotic use through enhanced diagnostics.

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.

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

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291CompletedPhase 2
CSL Behring
Posted 7/24/2018

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

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

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.

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