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BeOne Medicines Receives EMA PRIME Designation for BTK Degrader BGB-16673 in Waldenström's Macroglobulinemia

Orphan Drug
  • BeOne Medicines received PRIME designation from the European Medicines Agency for BGB-16673, an investigational BTK degrader for treating Waldenström's macroglobulinemia patients previously treated with BTK inhibitors.
  • The EMA's Committee for Medicinal Products for Human Use issued a positive opinion on the EU Orphan Drug Designation application for BGB-16673 in WM, with a final decision expected in the coming weeks.
  • BGB-16673 is designed to overcome resistance by degrading both wild-type and mutant forms of BTK, representing the most advanced BTK protein degrader currently in clinical development.
  • The FDA has also granted Fast Track Designation to BGB-16673 for treating relapsed or refractory chronic lymphocytic leukemia, small lymphocytic lymphoma, and mantle cell lymphoma.

A Dose-Escalation and Expansion Study of BGB-16673 in Participants With B-Cell Malignancies

NCT05006716RecruitingPhase 1
BeiGene
Posted 9/13/2021

Osteosarcoma Market Poised for Significant Growth Through 2034 as Novel Therapies Enter Pipeline

Monoclonal AntibodyOrphan DrugRare Disease
  • The osteosarcoma market in the 7MM is expected to grow significantly by 2034, driven by extensive R&D activities and the entry of novel therapies including OST-HER2, Naxitamab 15-096, and HALMB-0168.
  • OS Therapies plans to submit a Biologics License Application for OST-HER2 to the US FDA by 2025, with the potential to receive a Priority Review Voucher upon approval.
  • The market faces challenges including chemotherapy resistance in approximately half of patients and limited treatment options for relapsed or refractory cases.
  • Currently, MEPACT (mifamurtide) remains the only approved therapy specifically for osteosarcoma, highlighting significant unmet medical needs in this rare cancer.

Basilea Launches Phase 3 Trial for Novel Antifungal Fosmanogepix in Invasive Mold Infections

Orphan Drug
  • Basilea Pharmaceutica has initiated FORWARD-IM, a phase 3 registrational study evaluating fosmanogepix for treating invasive mold infections in adults, with completion expected in Q1 2028.
  • Fosmanogepix is a first-in-class broad-spectrum antifungal with novel mechanism of action, showing activity against multidrug-resistant molds including Aspergillus, Fusarium, and Mucorales fungi.
  • The study will enroll approximately 220 patients across two cohorts, with the first cohort randomizing 160 patients 2:1 to fosmanogepix versus standard-of-care therapy.
  • This marks the second phase 3 study for fosmanogepix, following the FAST-IC trial initiated in September 2024 for candidemia and invasive candidiasis treatment.

A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invasive Candidiasis.

NCT05421858RecruitingPhase 3
Basilea Pharmaceutica
Posted 12/11/2024

A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Patients With Invasive Mold Infections.

NCT06925321RecruitingPhase 3
Basilea Pharmaceutica
Posted 7/1/2025

Qualigen Therapeutics Secures $4.5 Million in Series A-3 Preferred Stock Financing to Advance Cancer Therapeutics Pipeline

OncologyOrphan Drug
  • Qualigen Therapeutics completed a $4.5 million private placement of Series A-3 Preferred Stock on July 28, 2025, with institutional and accredited investors.
  • The financing will support working capital needs for the clinical-stage biotechnology company's cancer therapeutics development programs.
  • Qualigen's pipeline includes QN-302 for pancreatic cancer, QN-247 for hematologic malignancies, and RAS oncogene inhibitors, all targeting high unmet medical needs.
  • The preferred stock is convertible into 1,607,143 common shares at $2.80 per share, with Univest LLC serving as exclusive placement agent.

AIM ImmunoTech Reports Promising Mid-Year Results from Phase 2 Pancreatic Cancer Combination Therapy Study

OncologyOrphan Drug
  • AIM ImmunoTech's Phase 2 DURIPANC study evaluating Ampligen combined with AstraZeneca's Imfinzi shows encouraging safety and efficacy signals in metastatic pancreatic cancer patients post-FOLFIRINOX treatment.
  • The combination therapy demonstrated no significant toxicity with 21% of patients achieving progression-free survival greater than 6 months and 64% of patients surviving beyond 6 months.
  • The study builds on previous monotherapy data from over 50 patients treated under compassionate use, representing a potential breakthrough in a disease with historically limited immunotherapy responsiveness.
  • Results suggest superior outcomes compared to historical data for maintenance or second-line immunotherapies in pancreatic cancer, a disease that kills over 500,000 people worldwide annually.

Combining Anti-PD-L1 Immune Checkpoint Inhibitor Durvalumab With TLR-3 Agonist Rintatolimod in Patients With Metastatic Pancreatic Ductal Adenocarcinoma for Therapy Efficacy

NCT05927142RecruitingPhase 1
Joachim Aerts, MD PhD
Posted 1/9/2024

EMA Recommends Approval of Aqneursa for Niemann-Pick Type C Disease Treatment

Drug ApprovalRare DiseaseOrphan Drug
  • The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending approval of Aqneursa (levacetylleucine) for treating neurological manifestations of Niemann-Pick type C disease in adults and children.
  • The recommendation is based on Phase III trial data showing statistically significant improvement in neurological symptoms within 12 weeks, with patients demonstrating better SARA scores compared to placebo.
  • Aqneursa represents a potential new frontline treatment option for NPC patients in Europe, following its FDA approval in September 2024.
  • The drug targets underlying neurological dysfunction processes and can be used in combination with miglustat or as monotherapy when miglustat is not tolerated.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288RecruitingPhase 3
IntraBio Inc
Posted 6/30/2022

MediciNova Nears Completion of Phase 2/3 ALS Trial as COMBAT-ALS Study Approaches Full Enrollment

Orphan Drug
  • MediciNova's Phase 2/3 COMBAT-ALS trial for MN-166 (ibudilast) is down to single-digit enrollment requirements, marking a critical milestone in ALS treatment development.
  • The company's MN-001 (tipelukast) Phase 2 trial for dyslipidemia and fatty liver disease in type 2 diabetes patients needs only two more subjects to complete enrollment.
  • Top-line data from the COMBAT-ALS trial is anticipated by the end of next year, with regulatory discussions with the FDA being actively prepared.
  • A parallel $22 million NIH-funded Expanded Access Program is steadily enrolling patients, demonstrating significant interest from the ALS community.

AI-Guided Drug LP-284 Achieves Complete Response in Treatment-Resistant Lymphoma Patient

AIOrphan Drug
  • Lantern Pharma's LP-284 achieved complete metabolic response in a heavily pretreated diffuse large B-cell lymphoma patient after just two treatment cycles.
  • The 41-year-old patient had previously failed three aggressive treatment regimens including CAR-T therapy and bispecific antibody therapy over 18 months.
  • LP-284 was developed using Lantern's RADR® AI platform in under three years at approximately $3 million, demonstrating computational efficiency in drug development.
  • The synthetic lethal therapy targets cancer cells with DNA damage repair deficiencies while preserving healthy tissue function.

Safety, Pharmacokinetics, and Clinical Activity of LP-284 in Adult Patients with Relapsed or Refractory Lymphomas and Solid Tumors

NCT06132503RecruitingPhase 1
Lantern Pharma Inc.
Posted 1/3/2023

FDA Grants Breakthrough Therapy Designation to Avidity's Del-zota for Duchenne Muscular Dystrophy

Orphan DrugRare Disease
  • The FDA has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for treating Duchenne muscular dystrophy in patients with mutations amenable to exon 44 skipping.
  • Del-zota demonstrated statistically significant increases in exon skipping, substantial dystrophin production increases, and significant creatine kinase reduction to near-normal levels in Phase 1/2 trials.
  • Avidity Biosciences remains on track for a planned Biologics License Application submission at year-end 2025, with topline data from the ongoing Phase 2 extension trial expected in Q4 2025.
  • The therapy represents a novel Antibody Oligonucleotide Conjugate approach that combines monoclonal antibody specificity with oligonucleotide precision to deliver treatment directly to muscle tissue.

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730CompletedPhase 1
Avidity Biosciences, Inc.
Posted 11/9/2022

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

NCT06244082Active, Not RecruitingPhase 2
Avidity Biosciences, Inc.
Posted 1/22/2024

RedHill Biopharma Receives FDA Approval for Groundbreaking MAP-Targeted Crohn's Disease Study

FDA ApprovalOrphan Drug
  • RedHill Biopharma received positive FDA feedback for the first-ever clinical study targeting Mycobacterium avium subspecies paratuberculosis (MAP) infected Crohn's disease patients with RHB-204.
  • The novel Phase 2 study will test MAP as a root cause of Crohn's disease, potentially making RHB-204 a paradigm-shifting therapy treating both the suspected cause and symptoms.
  • RHB-204 is supported by positive Phase 3 data from RHB-104 showing 64% improvement in efficacy and offers a 40% pill burden reduction with patent protection through 2041.
  • The Crohn's disease market is expected to grow from $13.6 billion in 2024 to over $19 billion by 2033, presenting significant commercial potential for new therapies.

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