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Lantern Pharma's LP-184 Demonstrates Significant Survival Benefits in Pediatric Brain Cancer Models

Rare DiseaseOrphan Drug
  • LP-184 showed remarkable survival improvements in mouse models of atypical teratoid rhabdoid tumors (ATRT), with median survival increasing by 345% in one model (p<0.0001).
  • Independent research from Johns Hopkins University validates Lantern Pharma's FDA Rare Pediatric Disease Designation for LP-184 in treating this aggressive pediatric brain cancer.
  • The drug demonstrated potent anti-tumor activity across multiple ATRT cell lines with strong blood-brain barrier penetrance and no apparent toxicity in preclinical studies.
  • Lantern Pharma plans to initiate a pediatric Phase I clinical trial for LP-184 in brain tumors in late 2025 or early 2026.

Study of LP-184 in Patients with Advanced Solid Tumors

NCT05933265RecruitingPhase 1
Lantern Pharma Inc.
Posted 6/9/2023

Palvella Therapeutics Appoints Rare Disease Commercial Expert Ashley Kline as Chief Commercial Officer

Rare DiseaseOrphan Drug
  • Palvella Therapeutics has appointed Ashley Kline as Chief Commercial Officer to lead the commercial buildout for QTORIN™ 3.9% rapamycin anhydrous gel, targeting microcystic lymphatic malformations affecting over 30,000 U.S. patients.
  • Kline previously led the successful launch of Oxervate® at Dompé Pharmaceuticals, scaling the rare disease therapy to over $500 million in annual U.S. sales by 2023.
  • The appointment positions Palvella for the potential standalone U.S. launch of what could be the first FDA-approved therapy for serious, rare genetic skin diseases with significant unmet medical need.

ITM Secures $262.5M Debt Financing to Support Commercial Launch of Radiopharmaceutical ITM-11 Following Positive Phase 3 Results

Orphan Drug
  • ITM Isotope Technologies Munich secured up to $262.5 million in debt financing from Blue Owl Capital to support commercial readiness and potential U.S. launch of ITM-11.
  • ITM-11 recently met the primary endpoint in the COMPETE Phase 3 study for treating gastroenteropancreatic neuroendocrine tumors (GEP-NETs).
  • The company plans to submit a New Drug Application (NDA) to the U.S. FDA in 2025 for ITM-11, which has received orphan drug and fast track designations.
  • The flexible funding structure will also enable continued advancement of ITM's targeted radiopharmaceutical pipeline and Actinium-225 manufacturing scale-up.

CDSCO Panel Approves Label Updates for Sanofi's Rare Disease Therapies Fabrazyme and Aldurazyme

Rare DiseaseDrug ApprovalOrphan Drug
  • India's CDSCO panel has approved prescribing information updates for Sanofi's Fabrazyme (agalsidase beta) for Fabry disease and Aldurazyme (laronidase) for Mucopolysaccharidosis I.
  • The Fabrazyme update aligns with global Company Core Data Sheet versions and requires additional EMA approval submission to CDSCO for further evaluation.
  • The Aldurazyme update harmonizes Indian prescribing information with the US Prescribing Information dated December 2023.
  • Both approvals were recommended during the Subject Expert Committee's Endocrinology and Metabolism meeting held on April 22, 2025.

Aptahem Advances Apta-1 to Phase II as European Pharma Company Expresses Strong Partnership Interest

Orphan Drug
  • Aptahem has received formal partnership interest from a well-reputed European pharmaceutical company for its RNA-based lead candidate Apta-1, which targets severe inflammatory conditions.
  • The company has finalized plans for a Phase II "basket trial" to evaluate Apta-1 in patients with acute urogenital, kidney, and lung disorders characterized by inflammatory and thrombotic pathologies.
  • Aptahem has initiated a strategic collaboration with Hongene Biotech to develop cost-effective and sustainable manufacturing methods for Apta-1, a critical step toward clinical advancement and commercialization.

Vicore Pharma's Buloxibutid Shows Superior Anti-Fibrotic Activity for IPF Treatment in New Data

Orphan Drug
  • Vicore Pharma presented promising Phase 2a trial data at the 2025 ATS Conference showing buloxibutid significantly improved lung function in idiopathic pulmonary fibrosis patients compared to existing treatments.
  • The novel angiotensin II type 2 receptor agonist demonstrated superior anti-fibrotic activity in laboratory studies, inhibiting key fibrosis biomarkers at clinically relevant concentrations where competitors nintedanib and nerandomilast showed limited effects.
  • Vicore's ongoing Phase 2b ASPIRE trial incorporates patient-friendly design elements based on input from IPF patients and caregivers, while their digital therapeutic Almee has shown improvements in quality of life for pulmonary fibrosis patients.

India's CDSCO Approves Two Cancer Drugs with Phase IV Trial Requirements

Drug ApprovalMonoclonal AntibodyOrphan Drug
  • India's drug regulatory authority has approved Eli Lilly's selpercatinib tablets in multiple strengths for RET fusion-positive non-small cell lung cancer treatment.
  • Intas Pharmaceuticals received approval to import and market serplulimab, a PD-1 inhibitor monoclonal antibody for cancer treatment.
  • Both approvals come with mandatory Phase IV clinical trial requirements to be submitted within three months of marketing authorization.
  • The approvals reflect India's regulatory alignment with global standards for orphan cancer drugs already approved in major markets.

Philikos Initiates Phase 1/2 Trial of T-Guard for Diffuse Cutaneous Systemic Sclerosis

Monoclonal AntibodyOrphan Drug
  • Philikos has enrolled the first patient in a Phase 1/2 trial evaluating T-Guard for diffuse cutaneous systemic sclerosis (dcSSc), a severe autoimmune disorder with limited treatment options.
  • The open-label study will assess safety and preliminary efficacy in 12 early-stage dcSSc patients whose disease remains refractory despite prior immunosuppressive therapy.
  • T-Guard, administered as four infusions over one week, aims to provide a safer alternative to hematopoietic stem cell transplantation by selectively depleting disease-associated T cells and NK cells.

FDA Greenlights Promontory Therapeutics' Phase 3 Trial Design for PT-112 in Metastatic Prostate Cancer

OncologyOrphan Drug
  • Promontory Therapeutics has successfully completed an End of Phase 2 meeting with the FDA, reaching agreement on key aspects of a registrational Phase 3 trial for PT-112 in metastatic castration-resistant prostate cancer.
  • The FDA approved the proposed dosing regimen, patient population, study comparator, and endpoints, with an interim analysis provision that could allow for drug approval before study completion.
  • Preliminary clinical outcomes from the Phase 2 trial of PT-112 will be presented at the upcoming ASCO 2025 Annual Meeting on June 2nd, following recent presentation of immune response biomarker data at AACR 2025.

A Study Evaluating the Safety, Pharmacokinetics, and Clinical Effects of Intravenously Administered PT-112 Injection in Subjects With Advanced Solid Tumors and Subsequent Dose Expansion Cohorts

NCT02266745Active, Not RecruitingPhase 2
Promontory Therapeutics Inc.
Posted 7/1/2014

PT-112 in Subjects With Thymoma and Thymic Carcinoma

NCT05104736RecruitingPhase 2
National Cancer Institute (NCI)
Posted 4/6/2022

Juvena Therapeutics Initiates First-in-Human Trial of JUV-161 for Muscle Regeneration in Myotonic Dystrophy

Orphan Drug
  • Juvena Therapeutics has begun enrolling participants in the first human clinical trial of JUV-161, a novel fusion protein designed to enhance muscle regeneration for treating Myotonic Dystrophy Type 1 and sarcopenia.
  • JUV-161, described as "insulin for muscle," works by restoring AKT signaling pathways that regulate muscle growth and metabolism, offering a unique approach compared to existing RNA-targeting or gene therapy strategies.
  • The therapy was discovered using Juvena's proprietary JuvNET platform, which combines AI and stem cell secretome biology to identify therapeutic proteins with regenerative potential.

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