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Clinical Trial News

Keros Therapeutics Discontinues PAH Drug Development Following Safety Concerns in Phase 2 Trial

  • Keros Therapeutics has discontinued development of cibotercept (KER-012) for pulmonary arterial hypertension after observing dose-dependent pericardial effusions in the TROPOS Phase 2 trial.
  • The company halted all dosing in the trial following safety reviews that revealed higher incidence rates of pericardial effusions compared to placebo and existing PAH treatments.
  • No clinically meaningful improvements in pulmonary vascular resistance or 6-minute walk distance were observed, leading to complete program termination.
  • The company is reducing its workforce by 45% to 85 employees, expecting $17 million in annual cost savings while exploring alternative development strategies.

A Study to Investigate the Safety and Efficacy of KER-012 in Combination With Background Therapy in Adult Participants With Pulmonary Arterial Hypertension (PAH) (TROPOS Study).

NCT05975905CompletedPhase 2
Keros Therapeutics, Inc.
Posted 10/17/2023

SKYTROFA Receives Australian Approval as First Once-Weekly Growth Hormone Therapy for Children

Rare DiseaseDrug Approval
  • The Therapeutic Goods Administration has approved SKYTROFA (lonapegsomatropin) as a once-weekly injectable treatment for pediatric growth hormone deficiency in children aged 3-18 years.
  • The therapy addresses a rare condition affecting approximately 2,000 Australian children, offering a significant improvement over daily injection regimens previously required.
  • SKYTROFA utilizes Ascendis Pharma's TransCon technology platform to provide sustained release of unmodified growth hormone over one week.
  • The approval was based on three pivotal Phase 3 clinical trials that collectively treated over 300 pediatric patients with growth hormone deficiency.

A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency

NCT04615273CompletedPhase 3
Ascendis Pharma Endocrinology Division A/S
Posted 12/3/2020

Rune Labs and Parkinson's Foundation Launch Groundbreaking Study Combining Genetic Testing with AI-Powered Digital Biomarkers

Precision MedicineAI
  • Rune Labs and Parkinson's Foundation have launched the first clinical program to correlate genetic and digital biomarkers for Parkinson's disease, supported by the Global Parkinson's Genetics Program.
  • The study combines StrivePD AI software with Apple Watch monitoring and PD GENEration genetic testing to map gene variants to real-world symptom data in Parkinson's patients.
  • Researchers aim to identify biological and digital signatures that could enable earlier diagnosis, more precise patient stratification for clinical trials, and development of personalized treatment strategies.
  • Initial genotype-response findings are expected in 2026, with the goal of moving from average-patient dosing to genome-matched therapy recommendations for the 25 million people expected to have Parkinson's by 2050.

Australian Federal Court Streamlines Preliminary Discovery Process for Pharmaceutical Patent Disputes

  • The Australian Federal Court has streamlined Rule 7.23 applications, making preliminary discovery a routinely ordered tool for pharmaceutical patent holders to investigate potential infringement cases.
  • Pharmaceutical patents particularly benefit from preliminary discovery orders since formulation and manufacturing process details are typically not publicly available, requiring court-ordered document disclosure.
  • Patent holders must demonstrate reasonable belief of infringement and insufficient information to proceed, with applications rarely refused despite potential respondents' defense options.
  • The process includes confidentiality protections limiting document access to external lawyers and independent experts, while allowing targeted discovery of documents necessary to assess infringement claims.

NIH Scientists Report Breakthrough Plant-Derived Therapy Reduces Intractable Cancer Pain by 38%

  • NIH scientists conducted a first-in-human clinical trial of resiniferatoxin (RTX), a plant-derived molecule that reduced worst pain intensity by 38% and opioid use by 57% in advanced cancer patients.
  • The therapy targets TRPV1 ion channels to selectively block pain-specific nerve fibers while preserving other sensations like touch and motor function.
  • RTX is derived from the Euphorbia resinifera plant and offers a non-addictive alternative for the 15% of cancer patients who cannot find relief from standard pain interventions.
  • The treatment shows potential for treating multiple pain conditions including neuromas, trigeminal neuralgia, and post-surgical pain, with larger clinical trials planned for FDA approval.

mRNA Delivery System Successfully Reactivates Latent HIV in Resting T Cells

  • Researchers developed an optimized lipid nanoparticle platform that efficiently delivers mRNA to resting T cells, overcoming a major technical barrier in HIV latency reversal strategies.
  • The mRNA delivery system successfully reactivated latent HIV genomes in ex vivo models from HIV-positive individuals without causing widespread immune activation or cellular toxicity.
  • This precision approach contrasts with previous latency reversal agents that induced detrimental systemic inflammation, offering a safer pathway toward HIV cure strategies.
  • The modular mRNA constructs allow fine-tuning of reactivation strength and duration, potentially enabling personalized HIV therapy approaches.

Teva Unveils $5 Billion Growth Strategy with Five Potential Blockbuster Drugs in Late-Stage Pipeline

  • Teva Pharmaceutical announced its acceleration phase strategy targeting a $5 billion innovative medicines franchise by 2030, driven by existing products AUSTEDO, AJOVY, and UZEDY plus five late-stage pipeline assets.
  • The company's late-stage pipeline includes five potential blockbusters: duvakitug for inflammatory bowel disease ($2-5 billion peak sales), DARI asthma inhaler (~$1 billion), emrusolmin for Multiple System Atrophy ($2+ billion), olanzapine LAI for schizophrenia, and TEV-408 for celiac disease ($1+ billion).
  • Teva reported strong momentum with nine consecutive quarters of growth and more than $2.3 billion in 2024 revenue from its innovative portfolio, while reaffirming 2027 financial targets including 30% operating margin expansion.

Hedera Dx Secures €15 Million Series A to Expand Decentralized Liquid Biopsy Platform Across Europe

Real World Evidence
  • Hedera Dx raised €15 million in Series A funding led by Vsquared Ventures to expand its hospital-based liquid biopsy testing platform across Europe.
  • The company's decentralized model enables hospitals to perform advanced blood-based cancer diagnostics in-house, reducing turnaround time from 10-14 days to under 48 hours.
  • Hedera's platform is already deployed in hospital labs across 11 European countries and includes a comprehensive 32-gene circulating tumor DNA panel with cloud-based analytics.
  • The funding will accelerate commercial expansion and development of new assays for minimal residual disease and early detection while enhancing the company's real-world data capabilities.

Cornell Develops 'Stealthy' Lipid Nanoparticles to Enhance mRNA Vaccine Delivery and Reduce Immune Side Effects

  • Cornell researchers have developed a new material that could significantly improve mRNA vaccine delivery and effectiveness by replacing ingredients that may trigger unwanted immune responses.
  • The innovation focuses on creating 'stealthy' lipid nanoparticles that enhance the therapeutic potential of mRNA vaccines like those used against COVID-19.
  • This breakthrough addresses a key challenge in mRNA vaccine technology by potentially reducing adverse immune reactions while maintaining vaccine efficacy.

Medicilon Successfully Passes FDA Inspection, Strengthening Global Preclinical R&D Capabilities

  • Medicilon Preclinical Research successfully passed its first FDA on-site inspection in seven years, receiving an official Establishment Inspection Report that validates its operational excellence and regulatory compliance.
  • The comprehensive FDA evaluation covered organizational structure, personnel qualifications, SOPs, facilities, documentation processes, and quality assurance systems, with inspectors also reviewing multiple research projects submitted to the agency.
  • By the end of 2024, Medicilon has supported 520 INDs entering clinical trials across multiple global regulatory agencies, including 34 antibodies, 28 ADC drugs, 8 GLP-1 drugs, and 6 PROTAC drugs.
  • The successful inspection significantly enhances Medicilon's regulatory credibility and reinforces its position as a trusted leader in preclinical R&D services for the global pharmaceutical industry.

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