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Clinical Trial News

Alebund's AP301 Meets Primary Endpoint in Phase 3 Trial for Hyperphosphatemia in Dialysis Patients

  • Alebund Pharmaceuticals announced that its investigational drug AP301, a novel fiber-iron-based phosphate binder, met its primary endpoint in a pivotal phase 3 study for controlling serum phosphorus in dialysis patients with hyperphosphatemia.
  • The 52-week randomized, open-label study enrolled 474 participants across 50 sites in China and demonstrated statistically significant and clinically meaningful improvement in serum phosphorus control compared to sevelamer carbonate.
  • AP301 offers advantages including high phosphate-binding capability, no need to chew before swallowing, no volume expansion in gastric fluid, and no systemic absorption, potentially addressing the low patient compliance issue where less than 50% of patients achieve good phosphate control.
  • The company plans to engage with China's NMPA regarding its new drug application in Q3 2025, targeting a market expected to reach 10 billion RMB by 2035.

AP301 Efficacy and Safety in Chinese Dialysis Patients With Hyperphosphatemia

NCT07030595CompletedPhase 3
Alebund Pharmaceuticals
Posted 6/12/2023

Liposomal Lipid Nanoparticles Show Enhanced Extrahepatic mRNA Delivery and Improved Stability

  • Researchers developed liposomal lipid nanoparticles (LNPs) with high bilayer lipid content that exhibit enhanced mRNA encapsulation and transfection potency compared to conventional Onpattro-like formulations.
  • The liposomal LNPs demonstrated 50-fold and 150-fold increased protein expression in spleen and lymph nodes respectively, with significantly longer circulation times due to reduced protein corona formation.
  • These novel formulations showed superior stability over 63 weeks of storage and achieved efficient extrahepatic delivery to organs including pancreas, heart, and immune tissues through a unique pH-responsive mechanism.

Plus Therapeutics Prepares CNSide Diagnostics Platform for U.S. Commercial Launch in Second Half 2025

  • Plus Therapeutics announced plans to commercially launch its CNSide CSF Assay Platform in the second half of 2025, targeting an underserved CNS cancer diagnostic market estimated at over $6 billion in the U.S.
  • The CNSide platform demonstrates superior performance compared to current standard of care, with 92% sensitivity and 95% specificity, having influenced treatment decisions in 90% of cases across more than 11,000 tests performed.
  • The company has established commercial infrastructure including a centralized testing laboratory in Houston and hired experienced leadership to support the rollout, with meaningful revenue contributions expected in fiscal year 2026.
  • CNS metastases affect up to 30% of adult cancer patients, with leptomeningeal metastases occurring in approximately 5% of cancer patients and carrying a terminal prognosis with 1-year survival of just 7%.

Sysmex America Secures FDA Clearance for CN-6000 Automated Blood Coagulation Analyzer

  • Sysmex America received FDA clearance for the CN-6000™ Automated Blood Coagulation Analyzer, enabling high-throughput hemostasis testing for bleeding and clotting disorders.
  • The clearance includes five commonly performed hemostasis tests with specific reagent products, including PT/INR, APTT, fibrinogen, antithrombin, and D-dimer assays.
  • The analyzer offers fully automated testing in a space-saving footprint with expandable capacity to match different laboratory needs and workloads.
  • Sysmex continues pursuing market authorization for the CN-3000 analyzer and additional reagent applications to expand their hemostasis testing portfolio.

Portable Gene Therapy Device Could Revolutionize On-Demand Personalized Medicine for Rare Diseases

  • The NANOSPRESSO device enables hospital pharmacists to create personalized nanomedicines on-demand using portable cartridges containing genetic material and lipid components.
  • This breakthrough could address the treatment gap for 300 million people worldwide affected by rare diseases, where traditional centralized drug manufacturing is economically unfeasible.
  • The device combines nucleic acid therapeutics with lipid nanoparticles in a microfluidic system, allowing precise formulation of targeted medicines ready for injection.
  • Significant regulatory and safety hurdles remain before clinical implementation, as individualized treatments will require new approval and monitoring frameworks.

Lipocine Initiates Phase 3 Trial for Oral Brexanolone in Postpartum Depression

  • Lipocine has dosed the first patient in its pivotal Phase 3 clinical trial of LPCN 1154, an oral formulation of brexanolone for treating postpartum depression.
  • The randomized, blinded study will evaluate LPCN 1154 versus placebo in women aged 15 and older with severe postpartum depression, using a 48-hour dosing regimen.
  • The company anticipates submitting a New Drug Application to the FDA in mid-2026, with Phase 3 topline results expected in Q2 2026.
  • LPCN 1154 represents a potential breakthrough as an oral alternative to injectable brexanolone, offering rapid relief without requiring medical monitoring.

A Study to Assess the Safety and Efficacy of Oral LPCN 1154A in Women With Severe PPD

NCT06979544RecruitingPhase 3
Lipocine Inc.
Posted 6/16/2025

XEOMIN Receives First-Ever Approval in Japan for Chronic Sialorrhea Treatment

Drug ApprovalRare Disease
  • Teijin Pharma has received approval from Japan's Ministry of Health, Labor and Welfare for XEOMIN (incobotulinumtoxinA) to treat chronic sialorrhea, marking the first drug approved for this indication in Japan.
  • The approval was based on Phase III clinical trials conducted by Merz Therapeutics in Germany and Poland, as well as trials conducted by Teijin in Japan.
  • XEOMIN works by inhibiting acetylcholine release from cholinergic nerve endings, effectively suppressing saliva secretion and reducing water and electrolyte secretion from salivary glands.
  • Chronic sialorrhea significantly impacts patients' quality of life, causing social isolation and difficulties with speaking and eating, while placing heavy burdens on families and caregivers.

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Episodic Migraine

NCT07018700RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Chronic Migraine

NCT07018713RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

FDA Approves Enhanced Labeling for Endari Based on Post-Marketing Pharmacokinetic Data

Drug ApprovalRare Disease
  • Emmaus Life Sciences received FDA approval for enhanced labeling of Endari (L-glutamine oral powder) based on additional post-marketing pharmacokinetic study data.
  • The updated label provides more comprehensive prescribing information, including confirmation of body weight-based dosing, no unwanted accumulation with twice-daily dosing, and flexible administration with or without food.
  • Endari remains approved to reduce acute complications of sickle cell disease in adult and pediatric patients five years and older, addressing a condition affecting approximately 100,000 people in the United States.
  • The label enhancement represents a significant improvement in clinical guidance for healthcare providers treating sickle cell disease patients with this prescription-grade L-glutamine therapy.

IMCD Acquires Trichem to Strengthen Pharmaceutical Supply Chain in India's Generic Drug Market

  • IMCD N.V. has acquired 100% of Trichem Healthcare Private Limited and its subsidiaries to expand its pharmaceutical distribution capabilities in India and the Middle East.
  • Trichem, founded in 1998 and based in Mumbai, generates approximately INR 1.7 billion (EUR 18 million) annually through supplying active pharmaceutical ingredients and pharmaceutical intermediates.
  • The acquisition positions IMCD to capitalize on India's role as a major supplier of generic drugs globally while adding regulatory expertise for highly regulated pharmaceutical markets.

ZyVersa Therapeutics Initiates Phase 2a Trial for First-in-Class Diabetic Kidney Disease Treatment VAR 200

  • ZyVersa Therapeutics has activated its first clinical site in San Antonio, Texas, to begin patient recruitment for a Phase 2a trial of VAR 200, a potential first-in-class treatment targeting renal lipotoxicity in diabetic kidney disease.
  • VAR 200 addresses an unmet medical need by targeting excess cholesterol and lipid accumulation in kidney glomeruli, a neglected pathogenic pathway with no currently available therapies.
  • The 16-week open-label study will evaluate VAR 200's efficacy and safety in patients with type 2 diabetes and diabetic kidney disease, with preliminary data expected in H2-2025.
  • Preclinical studies demonstrated VAR 200's ability to reduce cholesterol and lipid levels, protect against renal injury and fibrosis, and improve proteinuria across multiple kidney disease models.

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