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FDA Approves Expanded Pediatric Trial for Moleculin's Annamycin in Acute Myeloid Leukemia

Orphan Drug
  • FDA has approved Moleculin's pediatric study plan for Annamycin in combination with cytarabine as second-line therapy for children with relapsed/refractory acute myeloid leukemia.
  • The regulatory agency recommended including patients as young as 6 months old, younger than Moleculin's original proposal of 2 years, expanding potential treatment access.
  • Annamycin aims to eliminate cardiotoxicity associated with anthracyclines, which are used to treat approximately 60% of children with cancer.
  • The pediatric trial is planned to begin in the second half of 2027, while the ongoing Phase 3 MIRACLE trial in adults continues with initial data expected in late 2025.

L-Annamycin for Injection in Combination With Cytarabine Injection as Second Line Therapy for Remission Induction in Adult Subjects With Refractory/Relapsed AML

NCT06788756RecruitingPhase 2
Moleculin Biotech, Inc.
Posted 3/12/2025

Biogen Launches Phase 3 Pediatric Trial of Omaveloxolone for Friedreich Ataxia

Rare DiseaseOrphan Drug
  • Biogen has initiated the BRAVE study, a global Phase 3 clinical trial evaluating omaveloxolone in children aged 2 to 16 years with Friedreich ataxia.
  • The study will enroll approximately 255 pediatric participants and use the Upright Stability Score as the primary endpoint to measure disease progression.
  • This represents the first potential treatment option for pediatric Friedreich ataxia patients, addressing a critical unmet medical need in this vulnerable population.
  • Omaveloxolone is currently approved as SKYCLARYS for adults and adolescents aged 16 and older in over 40 countries worldwide.

A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

NCT06953583RecruitingPhase 3
Biogen
Posted 6/9/2025

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890Recruiting
Biogen
Posted 12/12/2024

Actio Biosciences Raises $66M Series B to Advance Rare Disease Programs Targeting Ion Channel Disorders

Rare DiseaseOrphan DrugPrecision Medicine
  • Actio Biosciences secured $66 million in Series B funding co-led by Regeneron Ventures and Deerfield Management to advance two lead programs targeting rare genetic diseases.
  • The company's most advanced program, ABS-0871, is currently in Phase 1 trials for Charcot-Marie-Tooth disease type 2C, with potential expansion to overactive bladder treatment.
  • ABS-1230, targeting KCNT1-related epilepsy affecting approximately 2,500 individuals in the U.S., received FDA rare pediatric and orphan drug designations and is expected to enter Phase 1 trials in late 2025.
  • The company's "inclusive precision medicine" approach aims to develop treatments that address multiple mutations within the same genetic pathway, potentially benefiting broader patient populations.

BiomX Achieves Breakthrough in Phage Therapy with Positive Phase 2 Results for Antibiotic-Resistant Infections

Orphan Drug
  • BiomX Inc. reported positive Phase 2 results for diabetic foot osteomyelitis, achieving statistically significant ulcer size reduction with p-values of 0.046 at week 12 and 0.052 at week 13.
  • The company's cystic fibrosis program showed 14.3% of patients completely cleared chronic P. aeruginosa infections after 10 days of treatment, compared to 0% in the placebo group.
  • BiomX has received $40 million in non-dilutive funding from the U.S. Defense Health Agency and FDA Fast Track and Orphan Drug designations for its phage therapy programs.
  • Wall Street analysts maintain Buy ratings with price targets of $15-16, representing potential upside of over 3000% from current trading levels around $0.4.

Intellia's CRISPR Gene Therapy Shows 98% Reduction in HAE Attacks After Three Years

Gene EditingOrphan DrugRare Disease
  • Intellia Therapeutics reported three-year follow-up data showing lonvoguran ziclumeran achieved a 98% mean reduction in monthly hereditary angioedema attack rates across all 10 Phase 1 patients.
  • All patients remained attack-free and treatment-free for a median of 23 months, demonstrating the potential for lonvo-z to become the first one-time therapy for HAE patients.
  • The company's Phase 3 HAELO trial completed screening ahead of schedule, with results expected in the first half of 2026 and a planned U.S. launch in 2027.
  • The CRISPR-based therapy targets the KLKB1 gene to prevent HAE attacks and has received multiple regulatory designations including FDA Orphan Drug and RMAT status.

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

NCT06634420Active, Not RecruitingPhase 3
Intellia Therapeutics
Posted 1/15/2025

Silence Therapeutics Reports Promising Phase 1 Data for Divesiran in Polycythemia Vera at EHA 2025

Orphan DrugRare Disease
  • Silence Therapeutics presented updated Phase 1 data for divesiran at EHA 2025, showing the siRNA therapy essentially eliminated the need for phlebotomies in 21 polycythemia vera patients with a combined history of 79 prior phlebotomies.
  • The SANRECO Phase 1 study demonstrated that divesiran maintained hematocrit levels at ≤45% across all dose cohorts while increasing hepcidin and ferritin levels, with no dose-limiting toxicities observed.
  • The company announced that its Phase 2 SANRECO study has exceeded 50% enrollment and remains on track for full enrollment by year-end 2025.
  • Divesiran represents a first-in-class siRNA therapy targeting TMPRSS6 to regulate iron metabolism and reduce excessive red blood cell production in polycythemia vera patients.

Hoth Therapeutics Secures Japanese Patent for Novel Mast Cell-Targeting Platform HT-KIT

Orphan DrugOncology
  • Hoth Therapeutics received Japan Patent No. 7677628 for its HT-KIT platform, providing exclusive protection until August 27, 2039.
  • The patented technology uses splice-switching oligonucleotides to selectively disrupt KIT gene expression in mast cells implicated in chronic hives and rare cancers.
  • The patent positions HT-KIT for orphan indications and expedited regulatory pathways, with potential applications in mast cell-driven inflammatory and oncologic conditions.
  • The company sees strategic licensing opportunities across Asia as it advances this novel therapeutic platform.

AlzeCure Receives FDA Support for ACD440 Development in Rare Pain Disorder Erythromelalgia

Orphan DrugRare Disease
  • AlzeCure Pharma received positive FDA guidance for advancing ACD440, a first-in-class TRPV1 antagonist, into Phase II/III studies for treating erythromelalgia, a rare chronic pain disorder affecting 1-4 per 100,000 people.
  • The FDA confirmed significant medical need for erythromelalgia treatment, which currently has no approved therapies and causes intense burning pain and severe skin redness in extremities.
  • ACD440 is being developed as a topical gel that maintains low systemic exposure while achieving high local concentrations for maximum analgesic effect, with potential for orphan drug designation and accelerated market access.
  • The drug candidate previously completed positive Phase IIa trials in chronic peripheral neuropathic pain and is based on Nobel Prize-winning scientific research from 2021.

FDA Grants Fast Track Designation to Sumitomo's Nuvisertib for Myelofibrosis Treatment

Orphan DrugRare Disease
  • The FDA has granted Fast Track Designation to nuvisertib (TP-3654), Sumitomo Pharma America's investigational PIM1 kinase inhibitor, for treating intermediate or high-risk myelofibrosis patients.
  • Updated Phase 1/2 data presented at EHA 2025 showed nuvisertib achieved 22.2% spleen volume reduction and 44.4% symptom score improvement in evaluable patients with relapsed/refractory myelofibrosis.
  • The oral selective inhibitor demonstrated significant cytokine modulation with p<0.001 correlation between anti-inflammatory effects and clinical responses, supporting its potential as monotherapy or combination treatment.
  • Myelofibrosis affects 1 in 500,000 people worldwide and represents a serious unmet medical need with limited treatment options for patients facing poor prognosis.

A Study of Oral Nuvisertib (TP-3654) in Patients With Myelofibrosis

NCT04176198RecruitingPhase 1
Sumitomo Pharma America, Inc.
Posted 12/16/2019

Cellectar's Iopofosine I 131 Doubles Survival in Pediatric Brain Cancer Trial

Rare DiseaseOrphan Drug
  • Cellectar Biosciences reported that iopofosine I 131 achieved 5.4 months average progression-free survival in pediatric high-grade glioma patients, doubling the reported median of 2.25 months.
  • Patients receiving higher doses (minimum 55 mCi) showed 8.6 months overall survival, with three patients receiving additional cycles achieving 11.5 months average overall survival.
  • The radioligand therapy demonstrated a favorable safety profile with no cardiovascular, renal, or liver toxicities, and all hematologic adverse events were manageable and reversible.
  • The CLOVER-2 Phase 1 trial enrolled 14 pediatric patients with relapsed/refractory high-grade gliomas, including diffuse midline gliomas and diffuse intrinsic pontine gliomas.

Study to Compare Tivozanib in Combination With Nivolumab to Tivozanib Monotherapy in Subjects With Renal Cell Carcinoma

NCT04987203Active, Not RecruitingPhase 3
AVEO Pharmaceuticals, Inc.
Posted 9/9/2021

Novel Targeted Radiotherapy in Pediatric Patients With Inoperable Relapsed or Refractory HGG

NCT05610891Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 10/1/2023

Dose Escalation Study of CLR 131 in Children, Adolescents, and Young Adults With Relapsed or Refractory Malignant Tumors Including But Not Limited to Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma

NCT03478462Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 4/30/2019

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