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Lexeo Therapeutics Secures $80M Financing to Advance Genetic Heart Disease Therapies

Rare Disease
  • Lexeo Therapeutics raised $80 million in private placement financing led by Frazier Life Sciences and Janus Henderson Investors to advance its genetic medicine pipeline for cardiovascular diseases.
  • The funding extends the company's cash runway into 2028 and will support operations through the potential 2027 efficacy readout for LX2006 in Friedreich ataxia cardiomyopathy.
  • The clinical-stage company is developing therapeutic candidates targeting genetic causes of cardiovascular conditions, including LX2006 for Friedreich ataxia cardiomyopathy and LX2020 for plakophilin-2 arrhythmogenic cardiomyopathy.

GRIN Therapeutics Secures $140M Series D and Global Partnership with Angelini Pharma for Radiprodil Development

Rare Disease
  • GRIN Therapeutics closed a $140 million Series D financing round with strategic investments from Angelini Pharma ($65 million) and Blackstone Life Sciences ($75 million).
  • The company entered an exclusive collaboration with Angelini Pharma for radiprodil development and commercialization outside North America, receiving $50 million upfront with potential for up to $520 million in milestones.
  • Radiprodil, targeting GRIN-related neurodevelopmental disorder, has received FDA Breakthrough Therapy designation and is advancing to a global Phase 3 trial in Q3 2025.
  • The partnership positions radiprodil as a potential first-in-class treatment for GRIN-NDD, addressing a significant unmet medical need in rare pediatric neurological diseases.

Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder

NCT05818943Active, Not RecruitingPhase 1
GRIN Therapeutics, Inc.
Posted 3/7/2023

Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

NCT06392009RecruitingPhase 1
GRIN Therapeutics, Inc.
Posted 7/10/2024

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.

RHEACELL and AOP Health Partner to Advance Stem Cell Therapy for Epidermolysis Bullosa and Chronic Venous Wounds

Rare Disease
  • RHEACELL and AOP Health have formed a strategic partnership to commercialize breakthrough stem cell therapies for epidermolysis bullosa and chronic venous wounds in Europe.
  • The collaboration builds on positive Phase 2 results, with two Phase 3 clinical trials currently underway and first EMA submission expected in 2026.
  • RHEACELL's ABCB5+ mesenchymal stromal cells offer a systemic approach to wound healing for patients with limited treatment options.
  • Epidermolysis bullosa affects approximately 500,000 people worldwide, causing skin fragility and chronic wounds with significant quality of life impact.

KU Leuven Spin-off Hemastatx Develops First Therapy Targeting Root Cause of Severe Bleeding Disorder

Monoclonal AntibodyRare Disease
  • Hemastatx, a new KU Leuven spin-off, has developed the first therapy to address the underlying mechanism of von Willebrand factor defects, a severe bleeding disorder affecting 100,000 hospitalized patients annually worldwide.
  • The company's novel antibody therapy targets ADAMTS13, a molecular scissor that excessively degrades von Willebrand factor in affected patients, restoring blood clotting function and halting bleeding episodes.
  • The spin-off secured seed funding from international investors including Swiss accelerator BaseLaunch, the Butterfly Fund, and KU Leuven's Gemma Frisius Fund to advance preclinical development and prepare for clinical trials.

FDA Places Clinical Hold on Rocket Pharmaceuticals' Danon Disease Gene Therapy After Patient Death

Rare Disease
  • The FDA has placed a clinical hold on Rocket Pharmaceuticals' Phase II gene therapy trial for RP-A501 after a patient developed capillary leak syndrome and subsequently died.
  • The patient experienced serious complications including fluid leaking from blood vessels into surrounding tissues, causing swelling and low blood pressure, followed by an acute systemic infection.
  • Rocket is investigating whether a novel immune suppression agent recently added to the pre-treatment regimen may have contributed to the adverse event.
  • The company's stock tumbled 63% in premarket trading, and analysts estimate the clinical hold could take weeks to months to resolve.

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437Active, Not RecruitingPhase 1
Rocket Pharmaceuticals Inc.
Posted 4/17/2019

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034RecruitingPhase 2
Rocket Pharmaceuticals Inc.
Posted 9/5/2023

Poxel's PXL065 Shows Promise in Preclinical Hypertrophic Cardiomyopathy Study, Data to be Presented at ESC 2025

Rare Disease
  • Poxel's PXL065, a deuterium-stabilized R-enantiomer of pioglitazone, demonstrated significant benefits in preventing pathological myocardial remodeling in a mouse model of hypertrophic cardiomyopathy.
  • The preclinical study showed PXL065 prevented hypertrophy and fibrosis in the heart, supporting its potential as a disease-modifying treatment for both symptomatic and asymptomatic HCM patients.
  • Results will be presented at the European Society of Cardiology Congress on September 1st, 2025, highlighting the compound's dual mechanism targeting mitochondrial dysfunction and oxidative stress.
  • The findings address a significant unmet medical need in HCM, which affects approximately 1 in 500 adults and currently has limited effective treatment options.

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.

Ruxoprubart Achieves Primary Endpoints in Phase 2 Trial for Paroxysmal Nocturnal Hemoglobinuria

Rare Disease
  • NovelMed's ruxoprubart, a novel complement-targeting immunotherapy, met all primary efficacy endpoints in interim Phase 2 trial results for adults with paroxysmal nocturnal hemoglobinuria at 12 weeks.
  • The therapy demonstrated clinically meaningful benefits including transfusion avoidance, increased hemoglobin levels, reduced lactate dehydrogenase, and increased PNH clone size.
  • These positive results represent a significant advancement in PNH treatment, offering potential for improved patient outcomes in this rare hematologic disorder.

Prothena Discontinues Birtamimab Development After Phase 3 Failure in AL Amyloidosis

Rare DiseaseMonoclonal Antibody
  • Prothena's Phase 3 AFFIRM-AL trial of birtamimab for AL amyloidosis failed to meet its primary endpoint of time to all-cause mortality (HR=0.915, p-value=0.7680).
  • The company has announced complete discontinuation of birtamimab development, including stopping the open-label extension trial, following the disappointing clinical results.
  • Prothena plans substantial organizational restructuring with workforce reductions and will provide detailed plans to decrease operating expenses in June 2025.

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