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Frontotemporal Dementia Therapeutics Enter New Era as Gene and Antibody Therapies Advance Toward Market

Monoclonal AntibodyOrphan DrugPrecision Medicine
  • Alector's Latozinemab (AL001) leads the frontotemporal dementia pipeline as a monoclonal antibody targeting progranulin deficiency, currently in pivotal Phase III trials with potential approval by 2026-2027.
  • Gene therapy companies AviadoBio and Passage Bio are developing AAV-based treatments AVB-101 and PBFT02 to restore progranulin expression through one-time administration, with readouts expected in late 2027 or early 2028.
  • The FTD therapeutic landscape is shifting from symptom management to disease modification, with multiple approaches including monoclonal antibodies, gene therapies, and oral small molecules targeting genetic biomarkers.
  • By 2028, the FTD market may include personalized therapies aligned with genetic profiles, potentially transforming clinical outcomes for patients aged 45-64 who currently lack disease-modifying treatment options.

Neuronata-R Stem Cell Therapy Shows Promise in ALS Subgroup, Targets FDA Accelerated Approval

Orphan Drug
  • CorestemChemon's Neuronata-R demonstrated statistically significant improvements in slow-progressing ALS patients across multiple efficacy measures including CAFS, ALSFRS-R scores, and respiratory function in Phase 3 subgroup analysis.
  • The autologous mesenchymal stem cell therapy showed consistent reductions in neurofilament light chain (NfL) biomarker levels, mirroring the regulatory pathway used for Tofersen's FDA accelerated approval.
  • The company plans to submit a biologics license application by Q4 2025 and target regulatory approval by mid-2026, following FDA meetings scheduled for Q3 2025.
  • While the overall Phase 3 trial missed its primary endpoint, post hoc analysis revealed meaningful clinical benefits in patients with slower disease progression, supporting a targeted therapeutic approach.

Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients with ALS

NCT04745299Active, Not RecruitingPhase 3
Corestemchemon, Inc.
Posted 3/23/2021

Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis

NCT01363401CompletedPhase 1
Corestemchemon, Inc.
Posted 2/1/2011

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.

European Commission Approves First MEK Inhibitor for NF1-Associated Plexiform Neurofibromas in Adults and Children

Drug ApprovalRare DiseaseOrphan Drug
  • The European Commission has granted conditional approval for Ezmekly (mirdametinib), the first therapy approved for both adults and children with neurofibromatosis type 1-associated plexiform neurofibromas.
  • The approval is based on the ReNeu Phase 2b trial results showing objective response rates of 41% in adults and 52% in children, with median tumor volume reductions of approximately 40% in both populations.
  • Ezmekly is a selective MEK 1/2 inhibitor that blocks the RAF-MEK-ERK pathway, addressing a significant unmet need for patients with symptomatic, inoperable plexiform neurofibromas aged 2 years and above.
  • The drug demonstrated a manageable safety profile with the most common adverse reactions including dermatitis acneiform, diarrhea, and elevated blood creatine phosphokinase levels.

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

NCT03962543Active, Not RecruitingPhase 2
SpringWorks Therapeutics, Inc.
Posted 9/29/2019

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.

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