Ixinity (trenonacog alfa) / Medexus, Ligand - LARVOL DELTA

Subdomain Analysis of the NPC Clinical Severity Scale Evaluating Long-Term Efficacy of Levacetylleucine in NPC (SSIEM 2026) - "Background IB1001-301 is a Phase 3, double-blind, randomized, placebo-controlled, crossover trial evaluating levacetylleucine (also known as N-acetyl-L-leucine) for the treatment of neurological signs and symptoms in Niemann-Pick disease type C (NPC) encompassing a Parent Study and an ongoing open-label Extension Phase (EP). These findings are consistent with results from 24-months of treatment and are indicative of a disease-modifying, neuroprotective effect. The uniform improvement across all subdomains demonstrates the robust and positive efficacy of levacetylleucine across a wide spectrum of neurological functions."

Clinical • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

Long-Term Efficacy and Safety of Levacetylleucine in Adults and Children with Niemann-Pick Disease Type C (SSIEM 2026) - "IB1001-301 is a Phase 3, double-blind, randomized, placebo-controlled, crossover trial evaluating levacetylleucine (also known as N-acetyl-L-leucine) for the treatment of neurological signs and symptoms in NPC encompassing a Parent Study and an ongoing open-label Extension Phase (EP). Conclusion Long-term treatment with levacetylleucine was associated with a statistically significant and clinically meaningful reduction in disease progression and consistent with a neuro-protective, disease modifying effect. Levacetylleucine was well-tolerated with no treatment emergent events occurring in more than 15% of participants on treatment."

Clinical • Ataxia • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases

Results from the Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Ataxia-Telangiectasia (EAN 2026) - "Levacetylleucine demonstrated a significant benefit versus placebo, and clinically meaningful improvements in neurological manifestations of A-T, functioning, and quality of life, and a favorable benefit-risk profile for the treatment of A-T."

Clinical • P3 data • Ataxia • Immunology • Metabolic Disorders • Movement Disorders • Primary Immunodeficiency

Long-term (30 Months) Outcomes of N-Acetyl-L-Leucine in Adults and Children with Niemann-Pick Disease Type C (EAN 2026) - "IB1001-301 is a Phase 3, placebo-controlled, crossover trial encompassing a Parent Study and an ongoing Extension Phase (EP) evaluating the long-term effects of N-acetyl-L-leucine (NALL) in people with NPC. Treatment with NALL demonstrated significant and meaningful long-term improvement, i.e, disease-modifying effects. NALL was well tolerated, and no serious adverse events occurred."

Clinical • Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases

Patterns of help-seeking for mental health problems in 1001 self-identified neurodivergent adolescents who self-harm. (PubMed, JCPP Adv) - "These findings, instead, point to potential issues with the acceptability of support received. This highlights the need to better understand which forms and locations of support are most acceptable to meet the specific needs of this population."

Journal • ADHD (Impulsive Aggression) • Attention Deficit Hyperactivity Disorder • CNS Disorders • Psychiatry

Results from a Global Phase 3 Trial (IB1001-303) Evaluating N-acetyl-L-leucine in Ataxia-telangiectasia (AAN 2026) - "Topline results including baseline demographics, primary efficacy and safety data will be presented pending data availability. Conclusions IB1001-303, a global, phase 3, randomized, double-blind, crossover trial will generate important data to evaluate the efficacy and safety of NALL in people with A-T."

Clinical • P3 data • Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Primary Immunodeficiency

Long-Term 30-Month Results of N-Acetyl-L-Leucine for Niemann-Pick Disease Type C (AAN 2026) - "IB1001-301 is a Phase 3, double-blind, randomized, placebo-controlled, crossover trial evaluating NALL for the treatment of neurological signs and symptoms in NPC encompassing a Parent Study and open-label Extension Phase (EP)...Long-term 30-month data will be presented at the 2026 American Academy of Neurology pending data availability. Conclusions Long-term 24-month treatment with NALL has demonstrated statistically significant and clinically meaningful reduction in disease progression."

Ataxia • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases

Results from the Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Children and Adults with Ataxia-Telangiectasia (AAN 2026) - "Conclusions Levacetylleucine demonstrated a significant benefit versus placebo, and clinically meaningful improvements in neurological manifestations of A-T, functioning, and quality of life, and a favorable benefit-risk profile for the treatment of A-T. Levacetylleucine was observed to be safe and well-tolerated, consistent with its established safety profile."

Clinical • Late-breaking abstract • P3 data • Ataxia • Immunology • Metabolic Disorders • Movement Disorders • Primary Immunodeficiency

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (clinicaltrials.gov) - P3 | N=53 | Recruiting | Sponsor: IntraBio Inc | Trial completion date: Dec 2028 ➔ Dec 2030 | Trial primary completion date: Jun 2023 ➔ Jun 2030

Trial completion date • Trial primary completion date • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

A pivotal study of N-Acetyl-L-Leucine on CACNA1A Disorders (clinicaltrialsregister.eu) - P2/3 | N=10 | Not yet recruiting | Sponsor: Intrabio Limited, IntraBio Inc

New P2/3 trial • CACNA1A

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - P2 | N=17 | Terminated | Sponsor: IntraBio Inc | N=39 ➔ 17 | Trial completion date: Jul 2025 ➔ Oct 2025 | Active, not recruiting ➔ Terminated; Determined priority was to recruit directly to the pivotal Phase III double-blind, placebo-controlled, randomised study (NCT07221292).

Enrollment change • Trial completion date • Trial termination • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

A Global Phase 3 Trial (IB1001-303) and an Expanded Access Program of Levacetylleucine in Ataxia-Telangiectasia (ACMG 2026) - "IB1001-303, a global, Phase 3, randomized, double-blind, crossover trial will generate important data to evaluate the efficacy and safety of NALL in people with A-T. A US EAP will be launched to provide access to levacetylleucine to eligible people with A-T who are otherwise ineligible for any clinical trials and have no satisfactory or comparable alternative therapy options available."

Clinical • P3 data • Ataxia • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Immunology • Inflammation • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Primary Immunodeficiency

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - P3 | N=60 | Active, not recruiting | Sponsor: IntraBio Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (clinicaltrials.gov) - P3 | N=53 | Recruiting | Sponsor: IntraBio Inc | Active, not recruiting ➔ Recruiting | Trial completion date: Dec 2026 ➔ Dec 2028

Enrollment open • Trial completion date • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

Tumor Location of Uveal Melanoma and Impact on Metastasis-Free Survival in 1001 Cases. (PubMed, Semin Ophthalmol) - "Uveal melanoma in the central region was smaller in thickness and base and demonstrated more favorable MFS compared to other quadrants, clock hours, and anteroposterior zones. Analysis of the 49 zones showed no benefit of tumor location alone after adjusting for tumor size and proximity to foveola and optic disc."

Journal • Eye Cancer • Melanoma • Oncology • Solid Tumor • Uveal Melanoma

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (clinicaltrials.gov) - P3 | N=53 | Active, not recruiting | Sponsor: IntraBio Inc | Trial completion date: Nov 2024 ➔ Dec 2026

Trial completion date • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - P2 | N=39 | Active, not recruiting | Sponsor: IntraBio Inc | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2025 ➔ Jul 2025 | Trial primary completion date: Dec 2024 ➔ Mar 2025

Enrollment closed • Trial completion date • Trial primary completion date • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

Trenonacog alfa safety, efficacy, and pharmacokinetics in previously treated pediatric hemophilia B. (PubMed, Res Pract Thromb Haemost) - "There appeared to be no clinically important differences between the results for participants aged <6 years and those aged 6 to <12 years. Trenonacog alfa is a suitable option for the management of pediatric persons with hemophilia B."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia B • Immunology • Pediatrics • Rare Diseases

Subgroup and Subdomain Analysis of Patients with Long Term Follow-Up In a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C (S31.004). (PubMed, Neurology) - "Billington has stock in IntraBio Inc. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Clinical • Journal • P3 data • Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Pediatrics • Rare Diseases

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - P3 | N=60 | Recruiting | Sponsor: IntraBio Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

Subgroup and Subdomain Analysis of Patients with Long Term Follow-Up In a Phase III, Randomized, Placebo-Controlled Crossover Trial with Levacetylleucine (IB1001) for Niemann-Pick Disease Type C (AAN 2025) - "Levacetylleucine demonstrated a significant improvement over placebo in pediatric and adult patients on the SARA and consistent improvement across mDRS subdomains. Treatment with levacetylleucine after 18 months was associated with a clinically significant reduction in disease progression consistent with a neuroprotective and disease-modifying effect."

Clinical • P3 data • Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Pediatrics • Rare Diseases

SARA Eight-Item Subgroup Analysis of N-acetyl-L-leucine for Niemann-Pick Disease Type C (AAN 2025) - "The IB1001-301 clinical trial was a Phase III, double-blind, randomized, placebo-controlled cross-over trial comparing NALL with placebo for treating neurological signs and symptoms in NPC.SARA is a validated eight-item clinical rating scale that incorporates assessments of gait, stance, sitting, speech disturbance, finger-chase, nose-to-finger, fast-alternating-hand movements, and heel-along-shin slide.Design/Patients received treatment with orally administered NALL 2-3 times per day as weight-based dosing...Analysis of the SARA subdomains demonstrated consistent improvement across each domain and the associated functional area—including ambulation, postural balance, speech, fine motor skills, and upper and lower extremity function. There was a consistent improvement with NALL versus placebo demonstrating the positive effects of NALL across a broad range of neurological functions."

Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases

Medexus Announces Strong Fiscal Q2 2025 Results (Medexus Pharma Press Release) - "IXINITY (US): Unit demand in the United States decreased by 4% over the trailing 12-month period ended September 30, 2024. (Source: customer-reported dispensing data.) Demand continues to reflect the effects of lower observed average quantities of IXINITY consumed by newer patients and a greater than expected impact of other developments in the broader hemophilia B treatment solutions market specifically relating to greater availability and use of extended half-life products that compete with IXINITY. Medexus will continue seeking to maintain existing demand, and to improve management of product supply to the market primarily by means of an August 2024 agreement with a national operator of specialty pharmacies. Medexus's investments in its IXINITY manufacturing process improvement initiative have had a positive impact on batch yield and manufacturing costs over fiscal year 2024 and now extending into fiscal year 2025."

Commercial • Genetic Disorders • Hemophilia B

Long-Term Effects of Empagliflozin in CKD (KIDNEY WEEK 2024) - "Over the entirety of follow-up a primary outcome occurred in 865/3304 participants (26.2%) in the empagliflozin group and in 1001/3305 (30.3%) in the placebo group (HR=0.79, 95%CI 0.72-0.87). EMPA-KIDNEY PTFU quantifies more completely the total effects of the short period of 2 years of empagliflozin. Study empagliflozin continued to exert benefit on cardiorenal outcomes for a period after its discontinuation. The post-trial benefit was smaller than the benefit when taking study treatment and appeared to be temporary."

Late-breaking abstract • Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Metabolic Disorders • Nephrology

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - P3 | N=60 | Not yet recruiting | Sponsor: IntraBio Inc

New P3 trial • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency