MedPath

Tagged News

Calico's ABBV-CLS-628 Receives FDA Fast Track Designation for Autosomal Dominant Polycystic Kidney Disease

Monoclonal AntibodyRare Disease
  • The U.S. FDA has granted Fast Track Designation to ABBV-CLS-628, an investigational anti-PAPP-A monoclonal antibody developed by Calico and AbbVie for treating ADPKD.
  • ADPKD is the most common inherited kidney disease worldwide, leading to kidney failure in more than 50% of patients by age 60.
  • The therapy is currently being evaluated in a global Phase 2 clinical trial across approximately 95 sites, with participants receiving intravenous treatment every 4 weeks for 92 weeks.
  • Fast Track Designation facilitates development and expedites FDA review for drugs addressing serious conditions with unmet medical needs.

A Study to Assess Adverse Events and Effectiveness of IntraVenous Infusions of ABBV-CLS-628 in Adult Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT06902558RecruitingPhase 2
AbbVie
Posted 6/9/2025

Lundbeck Advances Innovative Phase 3 Trial Design for Amlenetug in Multiple System Atrophy

Monoclonal AntibodyOrphan DrugRare Disease
  • Lundbeck will present details of its Phase 3 MASCOT trial for amlenetug, a first-in-class monoclonal antibody targeting α-synuclein aggregation in Multiple System Atrophy patients.
  • The innovative trial design employs Bayesian progression modeling methods to address unique challenges in rare disease drug development with limited patient populations.
  • Multiple System Atrophy is a rapidly progressing neurodegenerative disease with no approved treatments, affecting patients who typically live 6-9 years after symptom onset.
  • Amlenetug has received Orphan Drug Designation from FDA and EMA, plus SAKIGAKE designation in Japan, highlighting its potential therapeutic significance.

A Trial of Lu AF82422 in Participants With Multiple System Atrophy (MSA)

NCT06706622RecruitingPhase 3
H. Lundbeck A/S
Posted 12/3/2024

A Study of Lu AF82422 in Participants With Multiple System Atrophy

NCT05104476Active, Not RecruitingPhase 2
H. Lundbeck A/S
Posted 11/16/2021

Spur Therapeutics to Present Long-Term Durability Data for Phase 3-Ready Gaucher Disease Gene Therapy

Rare Disease
  • Spur Therapeutics will present new clinical data for avigbagene parvec (FLT201), its Phase 3-ready gene therapy candidate for Gaucher disease, at the ESGCT 32nd Annual Congress in October 2025.
  • The poster presentation will focus on the long-term durability of FLT201, which encodes an engineered variant of the GCase enzyme for treating Gaucher disease Type 1.
  • The data presentation represents a significant milestone for the clinical-stage biotechnology company as it advances toward Phase 3 trials for this debilitating metabolic disorder.

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943Active, Not RecruitingPhase 1
Spur Therapeutics
Posted 4/15/2022

OmniaBio Partners with BrainChild Bio to Manufacture CAR-T Therapy for Fatal Pediatric Brain Cancer

CAR-TRare Disease
  • OmniaBio Inc. and BrainChild Bio announced a manufacturing collaboration for BCB-276, an autologous CAR-T therapy targeting pediatric diffuse intrinsic pontine glioma (DIPG).
  • BCB-276 addresses a critical unmet need in DIPG, a universally fatal brain cancer with median overall survival of less than one year.
  • The partnership will support BrainChild Bio's Phase 2 pivotal registration trial aimed at securing FDA approval for treating children and young adults with DIPG.
  • Manufacturing will utilize OmniaBio's advanced robotics and AI-powered facility in Hamilton, Ontario, emphasizing scalable production and patient access.

FDA Issues Complete Response Letter for CUTX-101 Menkes Disease Treatment Due to Manufacturing Deficiencies

Rare DiseaseOrphan Drug
  • The FDA issued a Complete Response Letter for CUTX-101, a potential treatment for Menkes disease, citing manufacturing facility deficiencies but no concerns about the drug's safety or efficacy data.
  • CUTX-101 demonstrated significant improvement in overall survival for Menkes disease patients who received early treatment, supporting its Priority Review designation from the FDA.
  • Sentynl Therapeutics, which assumed development responsibility in 2023, plans to address the manufacturing issues and resubmit the application promptly.
  • Menkes disease affects between 1 in 8,664 to 1 in 34,810 live male births and currently has no FDA-approved treatment options.

FDA Grants Priority Review for Leniolisib in Children with Rare Immunodeficiency APDS

Drug ApprovalRare Disease
  • The FDA has accepted Pharming Group's supplemental New Drug Application for leniolisib in children aged 4-11 years with APDS, granting Priority Review with a target decision date of January 31, 2026.
  • If approved, leniolisib would become the first and only treatment specifically indicated for children with activated phosphoinositide 3-kinase delta syndrome, a rare primary immunodeficiency affecting 1-2 people per million worldwide.
  • The application is based on positive Phase III data showing improvements in lymphadenopathy and naïve B cell counts over 12 weeks, indicating correction of the underlying immune defect.
  • APDS is a progressive disease that typically begins in early childhood, causing immune dysregulation, recurrent infections, and potentially permanent lung damage and lymphoma.

Ultragenyx Appoints Eric Olson as Chief Business Officer to Lead Rare Disease Pipeline Expansion

Rare Disease
  • Ultragenyx Pharmaceutical appointed Eric Olson as Chief Business Officer and Executive Vice President effective September 22, 2025, following Thomas Kassberg's planned retirement after 14 years with the company.
  • Olson brings nearly two decades of biopharma business development experience, having led or supported over $15 billion in aggregate transaction value across rare disease and other therapeutic areas.
  • The appointment positions Ultragenyx to continue expanding what the company describes as the largest clinical pipeline in rare disease, building on four commercial therapies already approved.
  • Olson's previous roles include Chief Business Officer at Stoke Therapeutics and leadership positions at Alnylam Pharmaceuticals, Takeda, and Genzyme Corporation, with notable deals including partnerships worth billions of dollars.

JR-446 Receives Japanese Orphan Drug Designation for Rare Sanfilippo Syndrome Type B

Orphan DrugRare Disease
  • Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to JR-446 for treating mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B), joining similar designations from the FDA and European Commission.
  • The investigational therapy targets a devastating rare disease affecting 500-1,000 individuals worldwide, causing severe neurological decline with no currently approved treatments available.
  • JR-446 utilizes JCR's proprietary J-Brain Cargo® technology and has demonstrated promising non-clinical results in addressing central nervous system symptoms.
  • The drug is currently being evaluated in a Phase I/II clinical trial in Japan under a collaboration between MEDIPAL Holdings and JCR Pharmaceuticals.

ReCode Therapeutics Secures $29M Financing to Advance Inhaled mRNA Therapy for Cystic Fibrosis

Rare Disease
  • ReCode Therapeutics raised over $29 million in additional financing to advance its genetic medicines pipeline, including investigational therapies for cystic fibrosis.
  • The Cystic Fibrosis Foundation committed an additional $3 million to support the ongoing Phase 2 clinical trial of RCT2100, bringing total CF Foundation investment to up to $33 million.
  • RCT2100 is designed to deliver functional CFTR protein via inhaled mRNA therapy, potentially benefiting all CF patients including those with rare mutations who don't respond to existing modulator therapies.
  • The company also announced a research collaboration with Praxis Precision Medicines to develop lipid nanoparticle formulations for enhanced antisense oligonucleotide delivery to brain regions.

Tonix Pharmaceuticals Advances TNX-2900 to Phase 2 Trial for Prader-Willi Syndrome Following FDA Clearance

Rare Disease
  • Tonix Pharmaceuticals received FDA clearance to initiate a Phase 2 trial of TNX-2900, a magnesium-potentiated intranasal oxytocin formulation for Prader-Willi syndrome in children and adolescents aged 8 to 17.5 years.
  • The drug received both Orphan Drug and Rare Pediatric Disease designations from the FDA, potentially qualifying Tonix for a Priority Review Voucher upon approval.
  • The 12-week randomized, double-blind, placebo-controlled study will evaluate TNX-2900's efficacy using the Hyperphagia Questionnaire for Clinical Trials as the primary endpoint.
  • Prader-Willi syndrome affects 1 in 10,000 to 1 in 30,000 births and has an average life expectancy of 22.1 years, representing a significant unmet medical need.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.