Miplyffa (arimoclomol) / LadRx, Orphazyme, Zevra Therapeutics, Xoma - LARVOL DELTA

IP-OR01-1.5: Oral Presentations - Preserving Lean Mass with GLP-1R Therapy: Preclinical Obesity Studies (ADA 2026) - No abstract available

Preclinical • Metabolic Disorders • Obesity

CT-OR01-1.5: Oral Presentations - Preventing Type 1 Diabetes (ADA 2026) - No abstract available

Diabetes • Metabolic Disorders • Type 1 Diabetes Mellitus

IA-OR01-1.5: Oral Presentations - Molecular Transducers of Insulin Action and Insulin Resistance (ADA 2026) - No abstract available

Metabolic Disorders

AC-OR01-1.5: Oral Presentations - Hypoglycemia Across Contexts: Risk, Recognition, and Real-World Safety (With ADA Presidents' Select Abstract Presentation) (ADA 2026) - No abstract available

Clinical • Real-world • Real-world evidence • Hypoglycemia • Metabolic Disorders

IB-OR01-1.5: Oral Presentations - Molecular and Therapeutic Strategies for Diabetes (ADA 2026) - No abstract available

Diabetes • Metabolic Disorders

EG-OR01-1.5: Oral Presentations - From Plate to Pancreas: Mechanisms Linking Diet to Diabetes (With ADA Presidents' Select Abstract Presentation) (ADA 2026) - No abstract available

Diabetes • Metabolic Disorders

BM-OR01-1.5: Oral Presentations - Empowering Populations: Diabetes Self-Management Education in Practice (ADA 2026) - No abstract available

Clinical • Diabetes • Metabolic Disorders

Zevra Reports Third Quarter 2025 Financial Results and Corporate Update (The Manila Times) - "MIPLYFFA: Eight prescription enrollment forms during Q3 2025, bringing the total since product launch to 137; OLPRUVA: One prescription enrollment form during Q3 2025, bringing the total since product launch to 30...$26.1 million for Q3 2025, which includes $22.4 million of MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $2.4 million in net reimbursements from the French EAP for arimoclomol..."

Commercial • Genetic Disorders

OR01-1. Ambulatory Anesthesia - Oral Presentations (0 Credit) (ASA 2025) - No abstract available

Anesthesia

OR01 Oral abstracts: Maternal fetal medicine (FIGO 2025) - No abstract available

Q2 2025 Financial Highlights (Yahoo Finance) - "Revenue, Net: $25.9 million for Q2 2025, which includes $21.5 million of MIPLYFFA net revenue, $0.3 million of OLPRUVA net revenue, $2.6 million in net reimbursements from the French EAP for arimoclomol, $1.2 million in royalties and other reimbursements under the AZSTARYS license agreement, and an upfront payment of $0.3 million from the out-license of our dextrorphan prodrug. For Q2 2024, total net revenue was $4.4 million."

Commercial • CNS Disorders • Genetic Disorders

Zevra Therapeutics Submits Marketing Authorization Application to European Medicines Agency to Review Arimoclomol for the Treatment of Niemann-Pick Disease Type C (The Manila Times) - "Zevra Therapeutics, Inc...announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC)...The EMA will review the application under the centralized marketing authorization procedure...'The extensive data generated for arimoclomol has shown long-term, meaningful clinical outcomes with 5 to 7 years of patient experience across more than 270 NPC patients through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date.'"

EMA filing • Genetic Disorders

OR01. NEUROENDOCRINOLOGY AND PITUITARY: Mechanisms and Gene Changes (ENDO 2025) - No abstract available

Endocrine Disorders

Game Changers: Blockbuster Small-Molecule Drugs Approved by the FDA in 2024. (PubMed, Pharmaceuticals (Basel)) - "Notably, eight of these drugs (including Rezdiffra®, Voydeya®, Iqirvo®, Voranigo®, Livdelzi®, Miplyffa®, Revuforj®, and Crenessity®) are classified as "first-in-class" and have received breakthrough therapy designation. These agents not only exhibit distinct mechanisms of action but also offer substantial improvements in efficacy for patients compared to prior therapeutic options. This article offers a comprehensive analysis of the mechanisms of action, clinical trials, drug design, and synthetic methodologies related to representative drugs, aiming to provide crucial insights for future pharmaceutical development."

FDA event • Journal • Review • Alopecia • Brain Cancer • Breast Cancer • Cardiovascular • Chronic Kidney Disease • Chronic Obstructive Pulmonary Disease • CNS Disorders • Congenital Adrenal Hyperplasia • Cystic Fibrosis • Dermatology • Duchenne Muscular Dystrophy • Endocrine Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Glioma • Hematological Disorders • Hematological Malignancies • Hepatology • Hypertension • Immunology • Infectious Disease • Leukemia • Lung Cancer • Lysosomal Storage Diseases • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis • Muscular Dystrophy • Nephrology • Non Small Cell Lung Cancer • Oncology • Primary Biliary Cholangitis • Psychiatry • Pulmonary Disease • Renal Disease • Respiratory Diseases • Schizophrenia • Solid Tumor • Ventricular Tachycardia

Zevra Announces Publication of MIPLYFFA Mechanism of Action Manuscript in Molecular Genetics and Metabolism (GlobeNewswire) - "The publication presents data demonstrating that arimoclomol enters the cell and increases the translocation of translation factors EB and E3 (FTEB & TFE3) from the cytosol to the nucleus, a key initial step for triggering a cascade of downstream events. Specifically, TFEB & TFE3 upregulate the coordinated lysosomal expression and regulation (CLEAR) genes including NPC1, which is essential for the regulation of lysosomal function. Increased CLEAR gene expression then causes higher NPC1 protein levels in the lysosomes, leading to greater correction of aberrant cholesterol trafficking. Of note, in animal NPC models, greater correction of aberrant cholesterol trafficking was shown to correlate with the improvement of specific neurological behaviors, such as rearing and gait."

Preclinical • Genetic Disorders • Lysosomal Storage Diseases • Rare Diseases

Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease. (PubMed, Mol Genet Metab) - "Arimoclomol, an orally available small molecule, is the first FDA-approved treatment for NPC when used in combination with miglustat...By also amplifying expression of CLEAR genes associated with autophagy, arimoclomol has the potential to act on different pathways and improve cell viability independent of NPC1 protein levels and functionality. In summary, the findings presented illustrate how arimoclomol improves lysosomal function and potentially autophagy flux to decrease lipid burden in NPC patient fibroblasts."

Journal • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • TFE3 • TFEB

Zevra Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million (GlobeNewswire) - "Zevra Therapeutics, Inc...announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. 'We have reached another key milestone with the closing on the sale of our PRV, which we received following the successful approval of MIPLYFFA (arimoclomol) from the U.S. Food and Drug Administration (FDA)'...'The $150 million in gross proceeds is non-dilutive capital that further enhances our financial flexibility to support our strategic priorities, which includes executing on the commercial launches of MIPLYFFA and OLPRUVA, supporting our ongoing Phase 3 trial for celiprolol, and augmenting our solid balance sheet to support future investments that are in alignment with our strategic plan'."

Financing • CNS Disorders • Metabolic Disorders

FDA approval of Miplyffa and Aqneursa: A dual breakthrough for the treatment of Neimann-Pick disease type C. (PubMed, Alzheimers Dement (N Y)) - No abstract available

FDA event • Journal • Frontotemporal Lobar Degeneration

Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million (GlobeNewswire) - "Zevra Therapeutics, Inc...today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the transaction, which is expected to take place within 30 to 45 days, subject to customary closing conditions...'This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of MIPLYFFA and OLPRUVA, and advancing our pipeline of product candidates to address unmet needs within the rare disease community.'"

Financing • CNS Disorders • Metabolic Disorders

Arimoclomol: First Approval. (PubMed, Drugs) - "Arimoclomol (MIPLYFFA™), an oral small molecule that crosses the blood brain barrier and is thought to upregulate CLEAR (Coordinated Lysosomal Expression and Regulation) network genes and improve lysosomal function, is being developed by Zevra Therapeutics Inc., for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC). In September 2024, arimoclomol was approved for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older in the USA. This article summarizes the milestones in the development of arimoclomol leading to this first approval for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients aged ≥ 2 years."

Journal • Review • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics

Miplyffa (arimoclomol). (PubMed, Clin Ther) - No abstract available

Journal

OR01-1. Ambulatory Anesthesia - Oral Presentations (Non-CME) (ASA 2024) - No abstract available

Anesthesia

Zevra’s MIPLYFFA (arimoclomol) Receives Approval from U.S. Food and Drug Administration for Use in Patients with Niemann-Pick Disease Type C (NPC) (GlobeNewswire) - "XOMA Royalty Corporation...announced today Zevra Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) for MIPLYFFA (arimoclomol). MIPLYFFA is approved for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients two years of age and older. It is the sixth commercial asset in XOMA Royalty’s growing royalty and milestone portfolio."

FDA approval • CNS Disorders • Lysosomal Storage Diseases

FDA Advisory Committee Votes Favorably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C (GlobeNewswire) - "Zevra Therapeutics...announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC)....'Based on the totality of the clinical data, including data from the pivotal trial, the long-term data from the arimoclomol open label extension study, and data from our expanded access programs (EAP: NCT04316637), we remain confident in the clinical benefit offered by arimoclomol as a treatment for NPC, and are optimistic about its continued path to approval'....The arimoclomol NDA has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024."

FDA event • PDUFA • CNS Disorders • Lysosomal Storage Diseases

LadRx and ImmunityBio Mutually Agree to Terminate Aldoxorubicin License (Businesswire) - "LadRx Corporation...is pleased to announce that the Company and NantCell, Inc...together with NantCell’s parent company ImmunityBio, Inc. ('ImmunityBio'), have agreed to a mutual termination of the license of aldoxorubicin entered into in 2017...With the termination of the license agreement between LadRx and NantCell, LadRx regains control of aldoxorubicin...XOMA consented to the mutual termination of the LadRx-NantCell agreement in order to facilitate the return of the program to LadRx...The agreement between LadRx and XOMA regarding future royalties and milestones associated with arimoclomol is not affected by the termination of the aldoxorubicin license between LadRx and NantCell....'we continue to march LADR-7 towards the clinic and remain on track for filing an IND application for LADR-7 in the third or fourth quarter of 2024.'"

IND • Licensing / partnership • CNS Disorders • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor