RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union)

Structure/Sequence

Molecular FormulaC18H24ClN3O7

InChIKeyOHUSJUJCPWMZKR-FEGZNKODSA-N

CAS Registry289893-26-1

Clinical Trials associated with Arimoclomol

NL-OMON49378

/ CompletedNot Applicable

Interventional, randomised, partial double-blind, placebo- and positive controlled, multiple-dose, 4-way-cross-over trial Investigating the effect of arimoclomol on cardiac repolarization in healthy men. - CS0344 Orphazyme TQT

Start Date02 Jun 2020

Sponsor / Collaborator-

NCT03836716

/ TerminatedPhase 3

Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.

Start Date19 Sep 2019

Sponsor / Collaborator-

NCT04049097

/ TerminatedPhase 3

An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial

A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)

Start Date20 May 2019

Sponsor / Collaborator

University College London

[+1]

100 Clinical Results associated with Arimoclomol

100 Translational Medicine associated with Arimoclomol

100 Patents (Medical) associated with Arimoclomol

Literatures (Medical) associated with Arimoclomol

01 Aug 2025·MOLECULAR GENETICS AND METABOLISM

Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial

Article

Author: Harmatz, Paul ; Dali, Christine Í ; Himmelstrup, Louise ; Grønborg, Sabine Weller ; Héron, Bénédicte ; Hennermann, Julia B ; Grunewald, Stephanie ; Mengel, Eugen ; Maier, Esther M ; Sharma, Reena ; Guenther, Sven ; Gautschi, Matthias ; Cording, Malene ; Da Riol, Rosalia M ; Deodato, Federica ; Tylki-Szymanska, Anna ; Santra, Saikat ; Del Toro, Mireia

BACKGROUND:

This paper presents efficacy and safety outcomes from the 48-month open-label extension (OLE) of the phase 2/3 NPC-002 trial (NCT02612129) which evaluated arimoclomol treatment in patients with Niemann-Pick disease type C (NPC). Arimoclomol was recently approved by the US Food and Drug Administration for treatment of NPC in combination with miglustat.

METHODS:

Patients with NPC who completed the double-blind (DB) phase of the randomized controlled NPC-002 trial were eligible to continue in the OLE, during which all patients received arimoclomol in addition to routine clinical care. Primary efficacy outcomes were the 5-domain NPC Clinical Severity Scale (5DNPCCSS), and the rescored 4-domain NPCCSS (R4DNPCCSS), which was introduced post-hoc. Additional outcomes included NPC-specific measures (full scale NPCCSS, and NPC clinical database [NPC-cdb] score), and safety evaluations.

RESULTS:

Of the 50 patients who started the DB phase, 41 entered the OLE phase, with 29 completing 48 months. During the OLE, mean (SD) 5DNPCCS and R4DNPCCSS scores increased by 3.2 (4.8) and 2.7 (4.2) over 48 months, respectively. Among patients switching from placebo to arimoclomol after the DB phase, mean annual change in 5DNPCCSS decreased from 2.0 (on placebo) to 0.1 in the first year of receiving arimoclomol and mean annual change in R4DNPCCSS decreased from 1.9 to 0.2, indicating slowing of disease progression. Annual scores for both endpoints remained numerically smaller throughout the OLE than during the DB phase. The score pattern in the subset of patients who received miglustat as part of their standard care regime in addition to arimoclomol (N = 33) was similar to that seen in the total population. 17-domain NPCCSS (excluding hearing domains) and NPC-cdb results further supported sustained efficacy of arimoclomol. Arimoclomol was well-tolerated over 48 months, with no new safety concerns identified.

CONCLUSION:

The OLE of the NPC-002 trial provides evidence for a sustained reduction in disease progression for at least 5 years in a heterogeneous population of NPC patients receiving arimoclomol in addition to routine clinical care, with no new safety concerns. These results align with the statistically significant and clinically meaningful reduction in disease progression observed over 12-months in the DB phase, further highlighting the potential of arimoclomol as an effective and well tolerated disease modifying treatment for NPC.

01 Jul 2025·NEUROLOGICAL SCIENCES

Safety and efficacy of arimoclomol in amyotrophic lateral sclerosis: a systematic review and meta-analysis

Review

Author: Fiaz, Muhammad Hamza ; Zafar, Hafiz Muhammad Qasim ; Masood, Musa ; Minhas, Umm E Aimen ; Tahira, Sameen ; Almas, Muhammad Sameer ; Masood, Saad ; Hassan, Syed Saad Ul

OBJECTIVES:

Amyotrophic Lateral Sclerosis (ALS) is a debilitating motor neuron disorder characterized by muscle weakness, atrophy, and spasticity. This meta-analysis aims to assess the safety and efficacy of Arimoclomol in patients with ALS.

METHOD:

A comprehensive literature search was conducted on 3 databases to discover articles published up to August 2024. Included studies were randomized controlled trials (RCTs). Data was analysed using Review Manager (v5.4). Cochrane Risk of Bias-2 (RoB-2) was adopted to assess the quality of RCTs.

RESULTS:

A total of 359 patients were analysed, with 239 individuals in the Arimoclomol group and 120 individuals in the placebo group. The pooled analysis of the primary outcome, change in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score from baseline, did not demonstrate a statistically significant difference favoring the Arimoclomol group (MD = 0.4495; 95% CI: -0.39, 1.27; p = 0.30). Similarly, secondary outcomes, including the Combined Assessment of Function and Survival (CAFS) rank score (MD = 1.00; 95% CI: -2.68, 4.67; p = 0.60), increase in transaminases (RR = 1.05; 95% CI: 0.19, 5.70; p = 0.95), mortality rate (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), and adverse events (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), showed no statistically significant differences between the groups.

CONCLUSION:

This study does not conclusively demonstrate that Arimoclomol has beneficial effects on ALS patients' physical functionality but shows promise for safety. Further clinical trials are needed to explore the neuroprotective effects of Arimoclomol in the treatment of ALS.

01 Jun 2025·Molecular Genetics and Metabolism Reports

Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Article

Author: Harmatz, Paul ; Héron, Bénédicte ; Roubertie, Agathe ; LaGorio, Lisa ; Porter, Forbes D ; Grunewald, Stephanie ; Mickle, Travis ; Maier, Esther M ; Dali, Christine Í ; Mengel, Eugen ; Berry-Kravis, Elizabeth ; Gautschi, Matthias ; Deodato, Federica ; Guenther, Sven ; Del Toro, Mireia ; Da Riol, Rosalia M ; Tylki-Szymanska, Anna ; Grønborg, Sabine Weller ; Santra, Saikat ; Patterson, Marc C ; Solomon, Beth ; Himmelstrup, Louise ; Hennermann, Julia B

Background:

In the 12-month, randomized, double-blind, placebo-controlled Phase 2/3 NPC-002 study (NCT02612129), arimoclomol significantly reduced annual disease progression versus placebo, measured by the 5-domain NPC Clinical Severity Scale (5DNPCCSS). Arimoclomol has been approved in the US for treatment of Niemann-Pick disease type C (NPC) in combination with miglustat. This paper introduces the rescored 4-domain NPCCSS (R4DNPCCSS) as a post-hoc primary endpoint in NPC-002, discusses its validation, and presents the results of the post-hoc primary analysis.

Methods:

To more accurately assess changes in disease course over a 12-month time period in a heterogeneous group of patients, the Cognition domain was removed from the 5DNPCCSS and the Swallow domain was rescored to reflect linearity in disease progression. Rescoring of the Swallow domain was based on input from clinical NPC and swallow experts from a qualitative interview-based study (N = 12), resulting in the R4DNPCCSS. To supplement prior validation analyses, data supporting the overall validity and reliability of the R4DNPCCSS was gathered through additional analyses of construct and convergent validity. The NPC-002 prespecified primary efficacy endpoint analysis based on the 5DNPCCSS score change from baseline to 12 months was repeated with R4DNPCCSS.

Results:

Construct validity analysis demonstrated high agreement between the R4DNPCCSS domain scores and the Clinical Global Impression Scale of Severity (CGI-S) and NPC Clinical Database (NPC-cdb) scores. Convergent validity was confirmed by strong correlations between the R4DNPCCSS domains and corresponding items on the Scale for Assessment and Rating of Ataxia (SARA), 9-hole peg test (9-HPT), and Video Fluoroscopic Swallowing Study (VFSS) performance tests. The NPC-002 post-hoc primary analysis showed a mean standard error (SE) change in R4DNPCCSS score of 0.35 (0.40) with arimoclomol (N = 34) versus 2.05 (0.54) with placebo (N = 16), and a treatment effect in favor of arimoclomol over placebo of -1.70 (p = 0.0155). In the miglustat subgroup analysis, mean (SE) change in R4DNPCCSS score was -0.23 (1.02) with arimoclomol (N = 22) versus 1.92 (3.37) with placebo (N = 12), representing a treatment effect of -2.21 (p = 0.0077).

Conclusion:

The R4DNPCCSS is a valid and reliable measure of disease progression demonstrating consistent outcomes with the prespecified 5DNPCCSS endpoint. Arimoclomol significantly slowed disease progression through 12 months as measured by the R4DNPCCSS versus placebo.

190

News (Medical) associated with Arimoclomol

22 Sep 2025

·www.globenewswire.com

XOMA Royalty Declares Quarterly Preferred Stock Dividends

EMERYVILLE, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (Nasdaq: XOMA) today announced its Board of Directors has authorized the following cash dividends to holders of XOMA Royalty’s Series A and Series B Cumulative Preferred Stock: Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share. Holders of depositary shares, each representing 1/1000 of a share of XOMA Royalty’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share. The preferred dividends will be paid on or about October 15, 2025, to respective holders of record at the close of business on October 3, 2025. About XOMA Royalty CorporationXOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the sellers receive non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. XOMA Royalty has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about XOMA Royalty and its portfolio, please visit www.xoma.com or follow XOMA Royalty Corporation on LinkedIn. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. Investor Contact Juliane Snowden XOMA Royalty Corporation +1-646-438-9754 juilane.snowden@xoma.comXOMA Royalty Media ContactKathy VincentKV Consulting & Managementkathy@kathyvincent.com

04 Sep 2025

·www.globenewswire.com

Zevra Therapeutics Presents Positive New Data Supporting Foundational Role of MIPLYFFA® (arimoclomol) for the Treatment of Niemann-Pick Disease Type C at the International Congress of Inborn Errors of Metabolism (ICIEM)

New data from pre-specified analysis shows patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression Nomination for Best Poster Award received for poster highlighting MIPLYFFA’s differentiated mechanism of action targets underlying pathology of NPC CELEBRATION, Fla., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the presentation of several posters highlighting new positive data on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) for the treatment of Niemann-Pick disease type C (NPC) at the International Congress of Inborn Errors of Metabolism (ICIEM). “The efficacy and safety of MIPLYFFA have been substantiated by the most extensive clinical dataset currently available for patients with NPC,” said Adrian Quartel, M.D., FFPM, Zevra’s Chief Medical Officer. “Our commitment to the NPC community drives our ongoing efforts to publish additional data demonstrating the impact of MIPLYFFA across a heterogeneous population of NPC patients, including new findings in patients under the age of two and in patients who transitioned to MIPLYFFA after previously receiving placebo in our pivotal double-blind trial. We are also pleased that our poster detailing the pathways by which MIPLYFFA targets the underlying pathophysiology of NPC has been nominated for the Best Poster Award by ICIEM.” Data Highlights In a poster (BP-19) titled “Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network,” it was demonstrated that MIPLYFFA activates transcription factors leading to the amplification of NPC1 protein levels and more successful NPC1 processing; thereby addressing the underlying NPC etiology through multiple mechanistic pathways. In a poster (P-264) titled “Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for the treatment of Niemann-Pick disease type C in patients treated with miglustat,” data demonstrated that patients who switched from placebo in the double-blind phase to MIPLYFFA in the open-label extension phase, while on continued concomitant miglustat treatment, experienced a decline in annual disease progression. In a poster (P-261) titled “Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to <24 months at study enrollment,” data evaluating five patients for up to 36 months of treatment showed that MIPLYFFA was well tolerated in children in this age group with no new safety signals observed. In a poster (P-76) titled “Arimoclomol for the treatment of Niemann-Pick disease type C in a real-world setting: long-term outcomes from an expanded access program in the United States,” the effectiveness and safety of MIPLYFFA for the treatment of NPC, including with and without miglustat as a component of routine clinical care, were further confirmed and were consistent with the benefits demonstrated in the pivotal, double-blind, placebo-controlled Phase 3 study. About MIPLYFFA® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal Phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. MIPLYFFA, used in conjunction with miglustat, is the only treatment shown to halt disease progression by addressing the underlying pathology of NPC with improvement seen at the first evaluation at week 12, and durable effect for more than 5 years. More than 270 NPC patients worldwide have been treated with MIPLYFFA, including in a pivotal Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP) for up to 7 years, and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. A Marketing Authorization Application for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C is under review by the European Medicines Agency. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Caution Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data; or the potential benefits of any of our products or product candidates for any specific disease or at any dosage. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2024, filed on March 12, 2025, and Zevra’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed on August 12, 2025, and Zevra’s other filings with the SEC. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Zevra Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com

Clinical ResultPhase 3Drug ApprovalOrphan Drug

28 Aug 2025

·www.globenewswire.com

Zevra Therapeutics Announces Multiple Datasets on MIPLYFFA® (arimoclomol) to be Presented at the International Congress of Inborn Errors of Metabolism (ICIEM)

CELEBRATION, Fla., Aug. 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced four posters on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM), taking place September 2-6, 2025, in Kyoto, Japan. MIPLYFFA is approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC). A poster (# BP-19) detailing that MIPLYFFA’s mechanism of action uniquely targets the underlying pathophysiology of NPC was selected for a Best Poster award. Additionally, positive, new data from a multi-center pediatric substudy in patients younger than two years old, as well as a new prespecified efficacy analysis of patients on routine clinical care with miglustat who switched from placebo to MIPLYFFA in their treatment regimen, will be presented. Poster Details Title: Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to <24 months at study enrollmentPresentation No:P-261Date/Time: Wednesday, September 3, 2025; 6:00pm JST Presenter: Laila Arash-Kaps, M.D.; SpinCS, Clinical Science for LSD Title: Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network Best Poster AwardPresentation No:BP-19Date/Time: Thursday, September 4, 2025; 5:30pm JST Presenter: Hadeel Shammas, Ph.D.; Zevra Therapeutics Title: Arimoclomol for the treatment of Niemann-Pick disease type C in a real-world setting: long-term outcomes from an expanded access program in the United StatesPresentation No:P-76Date/Time: Thursday, September 4, 2025; 5:30pm JST Presenter: Caroline Hastings, M.D.; UCSF Benioff Children’s Hospitals Title: Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for treatment of Niemann-Pick disease type C in patients treated with miglustatPresentation No:P-264Date/Time: Thursday, September 4, 2025; 5:30pm JST Presenter: Laila Arash-Kaps, M.D.; SpinCS, Clinical Science for LSD About MIPLYFFA® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. The extensive data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with 5 and in some patients 7 years of patient experience across more than 270 NPC patients worldwide 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. A Marketing Authorization Application for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C has been validated and is under review by the European Medicines Agency. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch. Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate. Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Caution Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the promise and potential impact of our preclinical or clinical trial data; or the potential benefits of any of our products or product candidates for any specific disease or at any dosage. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2024, filed on March 12, 2025, and Zevra’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed on August 12, 2025, and Zevra’s other filings with the SEC. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Zevra Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com

Clinical ResultDrug ApprovalPhase 3Orphan Drug

100 Deals associated with Arimoclomol

External Link

KEGG	Wiki	ATC	Drug Bank
-	Arimoclomol	-	DB05025

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Niemann-Pick Disease, Type C	United States	Zevra Therapeutics, Inc.	20 Sep 2024
Niemann-Pick Disease, Type C	United States	Zevra Denmark as	20 Sep 2024

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Myositis, Inclusion Body	Phase 3	United States	University College London	20 May 2019
Myositis, Inclusion Body	Phase 3	United Kingdom	University College London	20 May 2019
Amyotrophic Lateral Sclerosis	Phase 3	United States	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Belgium	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Canada	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	France	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Germany	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Italy	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Netherlands	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Poland	Zevra Denmark as	31 Jul 2018

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02612129 (NPC-002, Pubmed) Manual	Phase 2/3	Niemann-Pick Disease, Type C	50	arimoclomol (DB phase)	-	Positive	01 Aug 2025
				arimoclomol (OLE phase)	jfsdcboelo(rgkgujyhba) = luwqnlkflb palsygygip (apbxrqacab, 4.8) View more
NCT02612129 (FDA_CDER) Manual	Phase 3	Niemann-Pick Disease, Type C	50	MIPLYFFA with miglustatMIPLYFFA with miglustat	wtbbqapcex(exprussdya) = dvozirgehs sciatxewny (fxzjdznoif, 1)	Positive	20 Sep 2024
				Placebo with miglustat	wtbbqapcex(exprussdya) = dvmzcexviv sciatxewny (fxzjdznoif, 3.4)
NCT04049097 (CTgov)	Phase 3	Myositis, Inclusion Body	121	Arimoclomol	jqygprbdsr(klsurhjreq) = vyfgvuazfz hvofcjbrph (fyndbueurw, 2.60) View more	-	15 Sep 2023
NCT03491462 (ORARIALS-01, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	245	Arimoclomol (Arimoclomol (up to 76 Weeks))	twmkieqoan(orgydafsif) = cwmajowgwo jwbtqmdzpy (ohdmbtdxzy, 0.291) View more	-	24 Aug 2023
				Placebo (Placebo (up to 76 Weeks))	twmkieqoan(orgydafsif) = zqsnhyvtjg jwbtqmdzpy (ohdmbtdxzy, 0.283) View more
NCT03836716 (CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	120	Arimoclomol	auqehwiknw = qwopznsbqn reoqpjzngb (bamiszjjmt, bqcbpeqvua - vlrbihaxln) View more	-	24 Aug 2023
NCT02612129 (NPC-002, CTgov)	Phase 2/3	Niemann-Pick Disease, Type C	50	arimoclomol (Arimoclomol (12-month Double-blind Phase))	bczwmonwqo(jwmcbloxbr) = yfwnbreotz nikgxmnkxn (obusoaziyv, shuiofrxqw - dxyqnpreud) View more	-	09 Jun 2023
				Placebo (Placebo (12-month Double-blind Phase))	bczwmonwqo(jwmcbloxbr) = heaxuqydzs nikgxmnkxn (obusoaziyv, rrxdiymmba - qgvywelxop) View more
NCT02753530 (CTgov)	Phase 2	Myositis, Inclusion Body	152	Arimoclomol (Arimoclomol (20 Months))	vibfsoqctt(rjyxgalpce) = sgnsxzfdbd isruavdpdi (raydgaftys, ynkhxanoec - yfzfbfjijl) View more	-	10 May 2023
				Placebo (Placebo (20 Months))	vibfsoqctt(rjyxgalpce) = ylkddrupux isruavdpdi (raydgaftys, tcycfwiujz - xkkwyubtkg) View more
NCT02753530 (ACR2021) Manual	Phase 2	Myositis, Inclusion Body	152	Arimoclomol	jkftgcbgue(xuqrbnphzd) = sskxsunrip brbpthkjfg (yergoawcih ) View more	Negative	01 Sep 2021
				Placebo	jkftgcbgue(xuqrbnphzd) = uicvvefkws brbpthkjfg (yergoawcih ) View more
NCT03491462 (ORARIALS-01, GlobeNewswire) Manual	Phase 3	Amyotrophic Lateral Sclerosis	245	arimoclomol 248 mg	rqtgwkqqgp(uordkgxbzg) = did not meet its primary endpoints tonuyumogv (kdhifpfatw ) View more	Negative	07 May 2021
				Placebo
NCT00706147 (Pubmed \| Ann Neurol \| Neurology) Manual	Phase 2/3	Amyotrophic Lateral Sclerosis	36	arimoclomol	vhcnsrexnc(vjszccjnsx) = zowvzrjyqj vdzrjfyimp (omykriddgv, -0.63 to 1.63) View more	Positive	13 Feb 2018
				Placebo	-

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