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Clinical Trial News

Camurus' CAM2029 Shows Significant Liver Volume Reduction in Phase 2b Trial for Rare Polycystic Liver Disease

Rare DiseaseOrphan Drug
  • Camurus' POSITANO Phase 2b study demonstrates that CAM2029 significantly reduces liver volume by 4.3% and liver cyst volume by 8.7% compared to placebo in patients with polycystic liver disease.
  • The 12-month randomized trial enrolled 71 participants and met its primary endpoint with statistical significance, while also showing improvements in disease symptoms and patient-reported outcomes.
  • CAM2029 was well tolerated with a safety profile consistent with approved somatostatin receptor ligands, and Camurus plans to discuss Phase 3 study design with regulatory authorities.
  • The treatment addresses a significant unmet medical need for approximately 37,000 patients in the US and EU who currently have no approved pharmacological treatment options.

A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD

NCT05281328Active, Not RecruitingPhase 2
Camurus AB
Posted 6/28/2022

TOLREMO Therapeutics Strengthens Leadership with Key Appointments as TT125-802 Shows Clinical Promise

  • TOLREMO therapeutics has appointed Alessandra Cesano, MD, PhD, to its Board of Directors and named Florian D. Vogl, MD, PhD, as Chief Medical Officer to strengthen leadership capabilities.
  • The leadership changes follow promising clinical data presentation at ASCO 2025, where TT125-802 demonstrated significant clinical activity as the first CBP/p300 inhibitor to show efficacy in solid tumors.
  • TT125-802 showed rapid, deep and durable objective responses in KRAS-G12C and EGFR-mutant non-small cell lung cancer with a best-in-class safety profile without thrombocytopenia.
  • The company plans to initiate clinical trials evaluating TT125-802 in combination with targeted therapies for advanced tumor indications including EGFR- and KRAS-mutated lung cancer and multiple myeloma.

BrightSpring's Onco360 Pharmacy Selected as National Partner for Three New Cancer and Rare Disease Therapies

OncologyRare DiseaseDrug ApprovalTargeted Therapy
  • BrightSpring Health Services announced that its specialty pharmacy Onco360 has been selected as the national pharmacy partner for three newly approved therapies targeting advanced cancers and rare genetic disorders.
  • The partnership includes GOMEKLI for neurofibromatosis type 1 patients with plexiform neurofibromas, AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, and IBTROZI for ROS1-positive non-small cell lung cancer.
  • Onco360 will provide comprehensive patient support including access, education, data management, and expert clinical guidance for these innovative treatments across all 50 states.

AbbVie's Qulipta Demonstrates Superior Tolerability and Efficacy Over Topiramate in Head-to-Head Migraine Prevention Trial

  • AbbVie's migraine prevention drug Qulipta showed significantly lower discontinuation rates compared to topiramate in a late-stage head-to-head trial, with only 12.1% of patients stopping treatment versus 29.6% for the generic drug at 24 weeks.
  • The study met all secondary endpoints, with 64.1% of Qulipta patients achieving a 50% or greater reduction in monthly migraine days compared to 39.3% of topiramate patients.
  • This clinical success supports AbbVie's strategic expansion into neuroscience as the company seeks to diversify beyond Humira, which faces increasing biosimilar competition.
  • Qulipta generated $658 million in international sales last year, highlighting its commercial potential in the migraine prevention market.

Singlera Genomics' GutSeer Assay Achieves 81.5% Sensitivity in Detecting Early-Stage Gastrointestinal Cancers

  • Singlera Genomics and Fudan University's Zhongshan Hospital published results from the GUIDE prospective cohort study in Molecular Cancer, demonstrating their GutSeer assay's ability to detect five major gastrointestinal cancers through a simple blood test.
  • The DNA methylation-based assay achieved 81.5% sensitivity and 94.4% specificity for cancer detection, with 66.4% of cancer patients in the cohort being early-stage (stage I/II).
  • GutSeer outperformed a low-pass WGS fragmentomics assay with an AUC of 0.963 versus 0.887, while also determining tissue-of-origin in 80.7% of cases and detecting 21.4% to 47.1% of precancerous lesions.

Establishment of Molecular Classification Models for Early Diagnosis of Digestive System Cancers

NCT05431621Completed
Shanghai Zhongshan Hospital
Posted 11/15/2020

Abbott Partners with MSD to Expand Distribution of Diabetes Drug Portfolio in India

  • Abbott and MSD Pharmaceuticals have formed a strategic distribution partnership to expand access to MSD's oral anti-diabetic portfolio in India, including sitagliptin and its fixed-dose combinations.
  • The agreement covers the distribution of sitagliptin-based brands Januvia, Janumet, and Janumet XR, which remain category leaders despite patent expiration in July 2022.
  • India's oral anti-diabetic drug market is valued at Rs 12,500 crore, with DPP-4 inhibitors accounting for Rs 4,000 crore, addressing a critical need for over 101 million diabetic patients.
  • The partnership represents a strategic response to post-patent landscapes, allowing multinational firms to maintain market presence through local distribution networks.

NervGen Appoints Randall Kaye as Chief Medical Advisor Following Positive Spinal Cord Injury Trial Results

  • NervGen Pharma has appointed Dr. Randall Kaye as Chief Medical Advisor to guide the clinical and regulatory strategy for NVG-291, following positive topline results from the chronic cohort of their Phase 1b/2a spinal cord injury trial.
  • Dr. Kaye brings extensive CNS therapeutic development experience, having previously served as CMO at Longboard Pharmaceuticals through its $2.6 billion acquisition by H. Lundbeck A/S.
  • The appointment positions NervGen to advance NVG-291, a first-in-class therapeutic peptide with FDA Fast Track designation, toward an anticipated FDA meeting in the second half of 2025.
  • NVG-291 met its primary endpoint in the chronic cohort and demonstrated promising changes in hand function assessment, representing a potential breakthrough in neuroreparative therapeutics.

Curasight Advances uTREAT Brain Cancer Therapy with Phase 1 Trial Submission to EMA

Targeted Therapy
  • Curasight A/S has submitted a Clinical Trial Application to the European Medicines Agency for a Phase 1 trial of uTREAT, a targeted radiopharmaceutical therapy for glioblastoma patients.
  • The therapy targets the uPAR receptor, with recent Phase II data showing 94% of Grade 4 gliomas were uPAR-positive, supporting the treatment's potential in this aggressive brain cancer.
  • Glioblastoma affects approximately 30,000 patients annually in the US and EU, with a devastating prognosis where only 5% of patients survive five years after diagnosis.
  • The company has partnered with Premier Research as the contract research organization to conduct the international Phase 1 trial, with first patient dosing expected in the second half of 2025.

Akari Therapeutics Secures India Patent for Novel Spliceosome-Inhibiting ADC Payload Technology

  • Akari Therapeutics received Patent No. 562,919 from India's Intellectual Property Office for its novel PH1 payload, a spliceosome inhibitor that disrupts RNA splicing to kill cancer cells and activate immune responses.
  • The patent covers the company's immuno-oncology PH1 payload, proprietary linkers, and ADC technology with applications across various cancer targets, strengthening global IP protection.
  • India represents a strategic market with cancer cases projected to reach 2.08 million by 2040, highlighting the growing need for innovative cancer therapies.
  • The company's lead candidate AKTX-101 targets Trop2 receptors and has demonstrated significant tumor-killing activity and prolonged survival in preclinical studies compared to traditional ADC payloads.

Respiratorius Secures FDA Pre-IND Meeting for VAL001 Cancer Treatment Following Positive Physician Survey

Oncology
  • Respiratorius has been granted a pre-IND meeting with the FDA for VAL001, an innovative oral formulation of sodium valproate designed as pre-treatment for cancer therapy, particularly Diffuse Large B-cell Lymphoma.
  • A comprehensive physician survey across the U.S., Europe, and Japan involving 135 treating physicians and key opinion leaders found that over 70% would be willing to prescribe VAL001, citing favorable clinical efficacy and established safety profile.
  • Commercial analysis projects VAL001 could achieve annual sales of USD 190 million at estimated market penetration, with cumulative revenue of USD 2.3 billion over the period of market exclusivity.
  • The company has secured patent protection for VAL001's formulation across key markets including the U.S., Europe, Japan, and Canada, with the European patent recently granted providing at least 20 years of market exclusivity.

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