A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Start Date17 Aug 2026

Sponsor / Collaborator

IntraBio Ltd.

NCT06673056

/ Active, not recruitingPhase 3

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Start Date18 Mar 2025

Sponsor / Collaborator

IntraBio Ltd.

NCT05163288

/ RecruitingPhase 3

Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.

Start Date30 Jun 2022

Sponsor / Collaborator

IntraBio Ltd.

100 Clinical Results associated with Levacetylleucine

100 Translational Medicine associated with Levacetylleucine

100 Patents (Medical) associated with Levacetylleucine

588

Literatures (Medical) associated with Levacetylleucine

03 Jul 2026·JOURNAL OF BIOMOLECULAR STRUCTURE & DYNAMICS

Amino acid osmolytes disrupting intradomain interactions reduce the amyloidogenicity of α-synuclein: studies with charged l -amino acids and their derivatives

Article

Author: Prakash, Jogadhenu S. S. ; Prabhu, N. Prakash ; Saurabh, Archi ; Chauhan, Suraj

Aggregation of α-synuclein (α-syn) is a hallmark of Parkinson's and dementia with Lewy bodies pathogenesis. The high plasticity and lack of stable tertiary structure make α-syn more susceptible to its surrounding environment. Under stress conditions, small organic molecules known as osmolytes accumulate inside the cells. They affect the conformational states and fibrillation pathways of proteins. Here, the effects of eight different amino acid osmolytes (charged: l-glutamate, l-aspartate, l-lysine, l-arginine; amide side chains: l-glutamine, l-asparagine; and N-acetylated: N-acetyl-l-glutamic acid, N-acetyl-l-lysine) on the fibrillation of human α-synuclein were examined. Arginine and N-acetyl-l-lysine inhibited the fibrillation at concentrations above 0.2 and 0.4 M, respectively. Lysine, asparagine, and glutamate accelerated the fibrillation by reducing lag time. N-acetyl-l-glutamic acid induced lag-independent fibrillation, whereas glutamine and asparagine showed concentration-dependent effects on the fibrillation with reduced lag time at higher concentrations. Molecular dynamics simulations revealed that interdomain contacts facilitated the fibrillation. The amino acids interacting predominantly through van der Waals interactions reduced the lag time of α-syn. However, the amino acids having strong electrostatic interactions with the protein disrupted intradomain contacts, favored extended conformation, and inhibited the fibril formation.

01 Jul 2026·LANCET NEUROLOGY

Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial

Article

Author: Skorvanek, Matej ; Foltan, Tomas ; Bremova-Ertl, Tatiana ; Thakur, Nivedita ; Del Mar Garcia Romero, Maria ; Bolton, Clare ; Gautschi, Matthias ; Schmahmann, Jeremey ; Kolnikova, Miriam ; Hoche, Franziska ; Han, Vladimir ; Hahn, Andreas ; Martakis, Kyriakos ; Prasad, Manish ; Perlman, Susan ; Panagioti, Ouliana ; Thiel, Moritz

BACKGROUND:

Ataxia-telangiectasia is a rare, autosomal recessive neurodegenerative disorder. Levacetylleucine (N-acetyl-L-leucine) has been shown to be efficacious for the treatment of neurological manifestations and to have a disease-modifying effect in lysosomal storage disorders such as Niemann-Pick disease type C. We aimed to assess the safety and efficacy of levacetylleucine for paediatric and adult patients with ataxia-telangiectasia.

METHODS:

In this phase 3, randomised, double-blind, placebo-controlled crossover trial, participants were enrolled across ten research hospitals in Germany, Slovakia, Spain, Switzerland, the UK, and the USA. Eligible patients aged 4 years or older with genetically confirmed ataxia-telangiectasia were randomly assigned (1:1) using interactive response technology to receive two or three times daily orally administered levacetylleucine or a matching placebo over two consecutive 12-week treatment periods (patients weighing 35 kg or more received 4 g per day of orally administered levacetylleucine or a matching placebo three times per day and patients weighing less than 35 kg received weight-tiered doses two or three times per day based on approximately 0·1 g/kg per day). All participants, investigators, and assessors were blinded to group assignment. The primary outcome was the mean change on the Scale for the Assessment and Rating of Ataxia (SARA), assessed at baseline and at the end of each 12-week treatment period of levacetylleucine or placebo. Safety and efficacy analyses were done in all randomly assigned patients who received at least one dose of study medication, and a linear mixed-effects model was used to account for data missing at random. The trial is registered with ClinicalTrials.gov, NCT06673056, and CTIS, 2024-517706-29; the open-label extension phase is ongoing.

FINDINGS:

Between March 18, 2025 and June 30, 2025, 77 participants with a genetically confirmed diagnosis of ataxia-telangiectasia were screened for inclusion. Four patients were excluded (not meeting inclusion criteria) and 73 were enrolled and randomly assigned (36 to levacetylleucine followed by placebo and 37 to placebo followed by levacetylleucine. 73 patients were included in the primary analysis and safety sets. 38 (52%) of 73 patients were female and 35 (48%) were male; 55 (75%) of 73 patients were White. 47 (64%) of 73 were younger than 18 years and 26 (36%) were aged 18 years or older. The mean change in the SARA total score with levacetylleucine was -1·92 (SD 2·81) versus -0·14 (2·38) with placebo (linear mixed model treatment effect -1·88 [SD 0·41], 95% CI -2·70 to -1·06; p<0·0001). 54 adverse events occurred in 29 patients receiving levacetylleucine versus 75 events in 25 patients receiving placebo. No treatment-related serious adverse events or deaths occurred.

INTERPRETATION:

Levacetylleucine showed a significant and clinically meaningful improvement in functioning and was safe and well-tolerated, providing a favourable benefit-risk profile for the treatment of ataxia-telangiectasia. An ongoing open-label extension phase of this trial will investigate potential long-term, neuroprotective and disease-modifying effects.

FUNDING:

IntraBio.

14 May 2026·ChemMedChem

Synthesis of N‐Acetyl‐D‐ and ‐L‐Leucine‐ ¹³ C ₆ Tool Compounds in Neurodegenerative Disease

Article

Author: Ribeiro, João Gabriel ; Crepin, Damien ; Pantoş, G. Dan ; Nafie, Jordan ; McGown, Andrew ; Churchill, Grant ; Spencer, John ; Shepherd, Dawn ; Sharma, Manvendra ; Braine, Rebecca ; Platt, Frances M.

N‐acetyl‐L‐leucine (levacetylleucine, ALL) is the neuroprotective enantiomer of the racemic antivertigo drug, Tanganil (acetyl‐DL‐leucine, ADLL). ALL has recently been clinically repurposed for the treatment of Niemann Pick disease type C1, a disorder characterised by lysosomal accumulation of glycolipids and cholesterol. Isotopically labelled ALL was required, as well as its negative control, D‐enantiomer (ADL), to monitor the in vivo metabolic fate of the drug and to distinguish it from endogenous leucine and associated metabolites. Here, we describe the synthesis of N‐acetyl‐D‐ and ‐L‐leucine‐ 13 C 6 , as well as N‐acetyl‐L‐leucine‐ 13 C 1 , by N‐acetylation of their amino acid precursors, their spectroscopic characterisation and stereochemical and stability studies. These investigations found that formation of the products, as well as their water‐soluble sodium salt formulation, occurs without compromising stereochemical integrity.

News (Medical) associated with Levacetylleucine

19 Aug 2026

·www.biospace.com

IntraBio’s Pivotal Phase III Trial in CACNA1A-Related Disorders Is Enrolling and Is Expected to Be Over-Enrolled and Complete Recruitment in October

IB1001-304 is the first pivotal clinical trial conducted specifically for CACNA1A-related disorders , for which there are currently no approved therapies anywhere in the world. Recruitment for IB1001-304 has commenced; IntraBio anticipates the study will over-enroll and complete recruitment in October 2026. CACNA1A-related disorders have an incidence of approximately 1 in 11,700, corresponding to more than 30,000 individuals in the United States alone and an estimated 690,000 worldwide. IB1001-304 uses the same pivotal trial design that supported FDA approval and European Commission authorization of levacetylleucine (AQNEURSA®) in Niemann-Pick disease type C. AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc., a global biopharmaceutical company developing and commercializing therapies for rare and common neurological diseases, today announced that its pivotal Phase III IB1001-304 trial evaluating levacetylleucine in individuals with CACNA1A-related disorders is enrolling. The company anticipates the study will over-enroll and complete recruitment in October 2026, with the trial designed to take 6-months to complete. IB1001-304 is the first pivotal clinical trial conducted specifically for CACNA1A-related disorders. The study uses the same design as the pivotal trial that supported U.S. FDA approval and European Commission authorization of levacetylleucine (AQNEURSA®) for the neurological manifestations of Niemann-Pick disease type C (NPC), and that supported IntraBio’s positive Phase III study in Ataxia-Telangiectasia (A-T). The supplemental New Drug Application for A-T is currently under FDA Priority Review, with a PDUFA target action date of September 19, 2026. “This trial marks a pivotal milestone for the CACNA1A community,” said Lisa Manaster, Co-Founder and President of the CACNA1A Foundation. “As the mother of a daughter living with a CACNA1A-related disorder, I know firsthand what it means to have real hope that we are moving closer to having an approved, safe, and effective treatment. Given the urgent unmet need our families face, we look forward to continuing to collaborate with IntraBio to help ensure this novel treatment is rapidly made available to patients in our community.” CACNA1A-related disorders refer to a group of rare, inherited neurological conditions. Despite the severity and lifelong course of these conditions, there are currently no approved therapies indicated for CACNA1A-related disorders anywhere in the world. The estimated incidence of CACNA1A-related disorders is approximately 1 in 11,700, corresponding to approximately 30,000 individuals in the United States alone and an estimated 690,000 worldwide. About IB1001-304 IB1001-304 is a multinational, randomized, double-blind, placebo-controlled crossover Phase III clinical trial evaluating levacetylleucine in individuals aged four years and older with genetically confirmed CACNA1A-related disorders. The trial is being conducted at multinational sites in Austria, Germany, Greece, Italy, Switzerland, the United Kingdom and the United States. Further information about IB1001-304, including participating locations and eligibility requirements, is available at ClinicalTrials.gov under study NCT07221292. The study is registered on ClinicalTrials.gov as NCT07221292 and under EU CT Number 2025-523828-51. Levacetylleucine is investigational for the treatment of CACNA1A-related disorders and has not been approved by the FDA, MHRA, European Commission, Swissmedic or any other regulatory authority for this indication. About CACNA1A-Related Disorders CACNA1A-related disorders are rare, inherited neurological conditions caused by pathogenic variants in the CACNA1A gene, which plays a central role in calcium-channel function within the nervous system. Symptoms can begin in infancy, childhood or adulthood. The estimated incidence of CACNA1A-related disorders is approximately one in 11,700. There are currently no approved therapies indicated for CACNA1A-related disorders. About AQNEURSA® (levacetylleucine) U.S. Indication AQNEURSA® (levacetylleucine) is indicated in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg. U.S. IMPORTANT SAFETY INFORMATION Embryo-Fetal Toxicity Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA during pregnancy should consider the patient’s clinical need, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease. Pregnancy and Lactation: For females of reproductive potential, confirm the patient is not pregnant prior to initiating treatment. Advise use of effective contraception during treatment and for 7 days after the last dose if AQNEURSA is discontinued. There are no data on the presence of levacetylleucine or its metabolites in human or animal milk; the developmental and health benefits of breastfeeding should be weighed against clinical need and potential risks to the infant. Adverse Reactions The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. Drug Interactions Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer competes with levacetylleucine for monocarboxylate transporter uptake, potentially reducing efficacy. Monitor more frequently for P-gp substrate-related adverse reactions when used concomitantly with AQNEURSA, as AQNEURSA inhibits P-gp; the clinical significance of this finding has not been fully characterized. To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or visit . Please see Full U.S. Prescribing Information for AQNEURSA at aqneursahcp.com . EU (EMA) Indication AQNEURSA® is authorized in the European Union for the treatment of neurological manifestations of Niemann-Pick disease type C, in combination with miglustat or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg. See EMA Indication and Important Safety Information: About IntraBio IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading institutions worldwide. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding the anticipated pace and completion of enrollment in IB1001-304, the expected timing on which the study will close to further participants, the potential of levacetylleucine in CACNA1A-related disorders and other indications, and the anticipated timing of regulatory action. These statements are based on IntraBio’s current expectations and are subject to risks and uncertainties, including the pace of patient identification and screening, site activation timelines, and regulatory and clinical developments. Actual results may differ materially. IntraBio undertakes no obligation to update any forward-looking statement except as required by law. Contacts MEDIA & INVESTOR CONTACT Cass Fields Vice President, External Affairs ccfields@intrabio.com

20 Jul 2026

·www.biospace.com

CORRECTING and REPLACING IntraBio Announces Publication of New Research Showing a Direct, Bidirectional Effect on Lysosomal Function with Levacetylleucine

AUSTIN, Texas--(BUSINESS WIRE)--The last paragraph in the release after the boilerplate has been removed. The updated release reads: INTRABIO ANNOUNCES PUBLICATION OF NEW RESEARCH SHOWING A DIRECT, BIDIRECTIONAL EFFECT ON LYSOSOMAL FUNCTION WITH LEVACETYLLEUCINE IntraBio today announced the publication of new research in PLOS ONE describing a newly identified way that levacetylleucine, the active ingredient in AQNEURSA®, acts directly on cellular lysosomal function. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); The study, conducted by researchers at the University of Oxford, examined the drug's effect on Transcription Factor EB (TFEB), the master transcriptional regulator of lysosomal biogenesis and autophagy - the cellular systems responsible for clearing damaged proteins and lipids. Uniquely, a biphasic action of levacetylleucine on TFEB activity was identified, whereby levacetylleucine normalizes TFEB, activating its translocation in disease models when there is a deficiency, but also downregulating TFEB in models where it is overstimulated, thereby maintaining cellular homeostasis. This biphasic action is essential for a therapeutic effect and to ensure TFEB is not persistently overexpressed. The effect was also shown to be stereospecific and rapid in onset, appearing within approximately one hour. Only the purified L-enantiomer (levacetylleucine) was active; the D-enantiomer and the racemic mixture produced no effect, reinforcing the distinct pharmacology of the purified L-enantiomer used in AQNEURSA. Levacetyllecuine has been previously identified to have polypharmacolohical effects, with direct action on other cellular organelles such as the mitochondria, where it normalizes energy metabolism (cellular energy production). These novel findings provide the first evidence that levacetylleucine directly modulates lysosomal function. As TFEB dysregulation is implicated in a range of neurodegenerative and neurodevelopmental conditions, these findings provide further evidence of the potential applicability of levacetylleucine as a broad treatment for rare and common neurological conditions. “We have not previously observed a mechanism that regulates TFEB in this bidirectional, homeostasis-restoring manner,” said Professor Antony Galione, FRS FMedSci, senior and corresponding author and Professor of Pharmacology at the University of Oxford. “Rather than simply activating or inhibiting the pathway, levacetylleucine appears to recalibrate lysosomal activity according to the state of the cell to match its needs – in effect, acting as a molecular thermostat. These findings are remarkable and may have important implications for diseases characterized by lysosomal and mitochondrial dysfunction.” The full publication, “N-acetyl-L-leucine normalizes Transcription Factor EB activity by stereospecific bidirectional modulation in a HeLa cell model of Niemann-Pick disease type C,” can be accessed here: ABOUT INTRABIO IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading institutions worldwide, including the University of Oxford and the University of Munich. Contacts MEDIA & INVESTOR CONTACT Cass Fields | Vice President, External Affairs & Marketing ccfields@intrabio.com intrabio.com

Oligonucleotide

18 Jun 2026

·www.biospace.com

IntraBio Announces Publication of Pivotal Phase III Data with Levacetylleucine for Ataxia-Telangiectasia in The Lancet Neurology

AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc. today announced that results from its pivotal Phase III clinical trial evaluating levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T) have been published in The Lancet Neurology. The peer-reviewed publication, titled “Safety and efficacy of levacetylleucine in ataxia telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial” reports findings from IntraBio’s pivotal randomized, double-blind placebo-controlled Phase III study of levacetylleucine in patients with A-T, a rare, inherited, progressive neurological disease. This pivotal study met its primary endpoint, demonstrating statistically significant improvement in neurological symptoms and function compared with placebo, as measured by the Scale for the Assessment and Rating of Ataxia. Treatment with levacetylleucine resulted in a mean change in SARA score of -1.92 compared with -0.14 for placebo, representing a treatment difference of -1.88 points (p<0.001). Levacetylleucine also demonstrated statistically significant improvements on key secondary endpoints, including the International Cooperative Ataxia Rating Scale and Investigator Clinical Global Impression. Levacetylleucine was generally well tolerated, with no drug-related serious adverse events reported. “These findings provide important clinical evidence for levacetylleucine in Ataxia-Telangiectasia, a rare and progressive disease that can profoundly impact movement, coordination, speech, swallowing, and daily functioning,” said PD Dr. med. Tatiana Brémovà-Ertl, PhD, Principal Investigator from the University of Bern. “The results published in The Lancet Neurology represent an important contribution to the understanding of A-T and the potential role of levacetylleucine in addressing the neurological manifestations of the disease.” The full publication is available at: (26)00158-4/fulltext A supplementary New Drug Application (sNDA) with Levacetylleucine is currently under review by the U.S. Food and Drug Administration for the treatment of Ataxia-Telangiectasia, with a Prescription Drug User Fee Act target action date of September 19, 2026. A variation application has been submitted to the European Medicines Agency to expand the approved marketing authorization for levacetylleucine to include the treatment of Ataxia-Telangiectasia. Levacetylleucine is an experimental therapy for A-T and has not been approved by any jurisdiction for this indication. ABOUT ATAXIA-TELANGIECTASIA (A-T) Ataxia-Telangiectasia is a rare, inherited, progressive neurodegenerative disorder that typically manifests in early childhood. A-T is estimated to affect 1 in 40,000-100,000 people. It is characterized by cerebellar degeneration leading to worsening loss of coordination, impaired speech, abnormal eye movements, and eventual wheelchair dependence. Many patients also develop telangiectasia, immune deficiency with recurrent and potentially life-threatening infections, pulmonary disease, and a significantly elevated risk of cancer. There are currently no FDA-approved therapies for A-T. ABOUT INTRABIO IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading institutions worldwide, including the University of Oxford and the University of Munich. Contacts MEDIA & INVESTOR CONTACT Cass Fields | Vice President, External Affairs & Marketing ccfields@intrabio.com |

Phase 3Clinical ResultDrug Approval

100 Deals associated with Levacetylleucine

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Neurologic Manifestations	European Union	Intrabio Ireland Ltd.	19 Jan 2026
Neurologic Manifestations	Iceland	Intrabio Ireland Ltd.	19 Jan 2026
Neurologic Manifestations	Liechtenstein	Intrabio Ireland Ltd.	19 Jan 2026
Neurologic Manifestations	Norway	Intrabio Ireland Ltd.	19 Jan 2026
Niemann-Pick Disease, Type C	United States	Intrabio Ireland Ltd.	24 Sep 2024
Niemann-Pick Disease, Type C	United States	IntraBio, Inc.	24 Sep 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Ataxia Telangiectasia	NDA/BLA	United States	IntraBio, Inc.	20 Mar 2026
Episodic Ataxia, Type 2	Phase 3	United States	IntraBio Ltd.	17 Aug 2026
Episodic Ataxia, Type 2	Phase 3	Austria	IntraBio Ltd.	17 Aug 2026
Episodic Ataxia, Type 2	Phase 3	Germany	IntraBio Ltd.	17 Aug 2026
Episodic Ataxia, Type 2	Phase 3	Greece	IntraBio Ltd.	17 Aug 2026
Episodic Ataxia, Type 2	Phase 3	Italy	IntraBio Ltd.	17 Aug 2026
Episodic Ataxia, Type 2	Phase 3	Switzerland	IntraBio Ltd.	17 Aug 2026
Episodic Ataxia, Type 2	Phase 3	United Kingdom	IntraBio Ltd.	17 Aug 2026
Hemiplegic Migraine, Familial Type 1	Phase 3	United States	IntraBio Ltd.	17 Aug 2026
Hemiplegic Migraine, Familial Type 1	Phase 3	Austria	IntraBio Ltd.	17 Aug 2026

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


IB1001-301 (EAN2026)	Phase 3	Niemann-Pick Disease, Type C	40	N-acetyl-L-leucine (NALL) (Extension Phase (open-label))	lonhlvmqvx(aywfgelmdh) = jzzmhpwthm djhwnznaom (irkarexffb, 4.0)	Positive	26 Jun 2026
				Placebo	rjumaushnw(khhiiumvrx) = cyxtlbvvyp vbwinpsche (succduitok, 7.9) View more
S31.004 (AAN2025)	Phase 3	Niemann-Pick Disease, Type C	60	Levacetylleucine	ikibvzfykt(vefidjhjmn) = On each mDRS domain there was improvement from baseline when patients received levacetylleucine and a greater improvement in each domain when patients were treated with levacetylleucine versus placebo njwwpnozig (iopkziwcmp ) View more	Positive	07 Apr 2025
				Placebo
NCT05163288 (FDA_CDER) Manual	Phase 3	Niemann-Pick Disease, Type C	60	AQNEURSA	wcydbgwkzd(ncpjgvbxti) = reizxilqpa qskunhwvzq (knqyvscoxm, 0.1)	Positive	24 Sep 2024
				Placebo	wcydbgwkzd(ncpjgvbxti) = evlswtydpg qskunhwvzq (knqyvscoxm, 0.1)
NCT03759665 (Accesswire) Manual	Phase 2	Gangliosidoses, GM2	30	IB1001	hanyutzcii(wivaeppmss) = statistically significant change. 90% CI: 0.00, 1.50, p-value = 0.039 suokeujbxo (cabyqmzswx ) View more	Positive	14 Sep 2021
NCT03759639 (Pubmed \| J Neurol) Manual	Phase 2	Niemann-Pick Disease, Type C	32	N-acetyl-L-leucine	fuvdhryxxy(swhsrlwoos) = NALL met the CI-CS primary endpoint (mean difference 0.86, SD = 2.52, 90% CI 0.25, 1.75, p = 0.029) fofwxsicba (qbmgwftvlg )	Positive	13 Aug 2021
NCT00768287 (CTgov)	Phase 2/3	Hemophilia B	77	IB1001 (IB1001)	vpcuzuxwtz(wpmegdyytb) = uzalvwnbmp uxbwwzxgly (zlewszbmza, 451.5) View more	-	10 Sep 2018
				nonacog alfa (Nonacog Alfa)	vpcuzuxwtz(wpmegdyytb) = dhheodqkyj uxbwwzxgly (zlewszbmza, 468.6) View more

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