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

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

Metastasis-free survival of uveal melanoma by tumour size category based on The Cancer Genome Atlas (TCGA) classification in 1001 cases. (PubMed, Clin Exp Ophthalmol) - "By tumour size category, favourable cytogenetics (Groups A or B) is found in 86% of small tumours, 60% of medium tumours, and 58% of large tumours. The MFS at 10 years for favourable cytogenetics was 98% for small tumours, 92% for medium tumours, and 54% for large tumours. Tumour size category can serve as a surrogate for TCGA."

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

LONG-TERM FINDINGS OF N-ACETYL-L-LEUCINE FOR NIEMANN-PICK DISEASE TYPE C (SSIEM 2024) - "Background: The IB1001-301 clinical trial was a Phase III, double-blind, randomized, placebo-controlled trial comparing N-acetyl-L-leucine (NALL) with placebo for the treatment of neurological signs and symptoms in Niemann-Pick disease type C (NPC) after 12 weeks... In patients with NPC, treatment with NALL after 1 year was associated with a statistically and clinically significant reduction in disease progression and consistent with a neuroprotective and disease-modifying effect."

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

Assessment of the reliability, responsiveness, and meaningfulness of the scale for the assessment and rating of ataxia (SARA) for lysosomal storage disorders. (PubMed, J Neurol) - "Qualitative and quantitative data demonstrate the reliability and responsiveness of the SARA score as a valid measure of neurological signs and symptoms in LSDs with CNS involvement, such as NPC and GM2 Gangliosidoses. A 1-point change represents a clinically meaningful transition reflecting the gain or loss of complex function."

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

Long-term findings of N-acetyl-L-leucine for Niemann-Pick disease type C (EAN 2024) - "Background and Aims: The IB1001- 301 clinical trial was a Phase II, double- blind, randomized, placebo- controlled trial comparing N- acetyl- L- leucine (NALL) with placebo for the treatment of neurological signs and symptoms in Niemann- Pick disease type C (NPC) after 12weeks... In NPC, treatment with NALL after 1year was associated with a statistically and clinically significant reduction in disease progression and consistent with a neuroprotective, diseasemodifying effect."

Late-breaking abstract • Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders

Results of a Phase III, Randomized, Placebo-controlled Crossover Trial with N-acetyl-L-leucine for Niemann-Pick Disease Type C (AAN 2024) - P3 | "The secondary endpoints (CGI, SCAFI, mDRS) were also improved with IB1001 as compared to placebo (p<0.001)...NALL demonstrated a statistically significant improvement over placebo with a clinically meaningful benefit in symptoms (motor and cognitive), functioning, cognition, and quality of life. NALL was safe and well-tolerated, providing a favorable benefit-risk profile for the treatment of NPC."

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

XOMA Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent and Upcoming Events Expected to Drive Shareholder Value (GlobeNewswire) - "Raised up to $140 million of non-dilutive non-recourse capital through a royalty-backed loan related to VABYSMO from funds managed by Blue Owl Capital...On December 31, 2023, XOMA had cash and cash equivalents of $159.6 million (including $6.3 million in restricted cash). In 2023, XOMA’s royalty interests generated cash payments of $7.3 million from Roche related to VABYSMO sales and $1.7 million from Medexus related to IXINITY sales."

Commercial • Sales • Age-related Macular Degeneration • Diabetic Macular Edema • Diabetic Retinopathy • Genetic Disorders • Ophthalmology • Retinal Vein Occlusion • Wet Age-related Macular Degeneration

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (clinicaltrials.gov) - P2 | N=33 | Completed | Sponsor: IntraBio Inc | Active, not recruiting ➔ Completed

Trial completion • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • NPC1

CT findings as prediction of long covid (ERS 2023) - "Lung changes, most fibrosis, were found in 1001 patients with long covid... It is possible to predict long covid in patient with early subpleural fibrosis. Patients with chronic fatigue syndrome were often with Omicron strain.; Imaging; Endoscopy and interventional pulmonology; Epidemiology; Public health; Physiology; Pulmonary function testing; Respiratory intensive care; Surgery"

Cardiovascular • Critical care • Fatigue • Fibrosis • Immunology • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (clinicaltrials.gov) - P2 | N=39 | Recruiting | Sponsor: IntraBio Inc | Trial completion date: Jul 2023 ➔ Mar 2025 | Trial primary completion date: Jul 2023 ➔ Dec 2024

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

N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study. (PubMed, Trials) - P3 | "Pre-clinical as well as observational and phase IIb clinical trials have previously demonstrated that IB1001 rapidly improved symptoms, functioning, and quality of life for pediatric and adult NPC patients and is safe and well tolerated. In this placebo-controlled cross-over trial, the risk/benefit profile of IB1001 for NPC will be evaluated. It will also give information about the applicability of IB1001 as a therapeutic paradigm for other rare and common neurological disorders."

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

N-acetyl-L-leucine Improves Symptoms and Functioning in Niemann-Pick disease type C (NPC) and GM2 Gangliosidosis (Tay-Sachs & Sandhoff): Results from Two Parallel, Multi-National, Rater-Blinded Clinical Trials (EPNS 2023) - " We conducted two multicenter Phase IIb studies (IB1001-201 and IB1001-202)... NALL rapidly improved symptoms, functioning and quality of life in 6-weeks in patients with NPC and GM2 gangliosidosis. The effect was reversed after a 6-week, wash-out period. High consistency and statistical significance between the primary and secondary endpoints demonstrate a clinically meaningful improvement with NALL."

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

Acetyl-L-Leucine for Niemann-Pick type C: A Multinational Double-blind Randomized Placebo-controlled Crossover Study (AAN 2023) - P3 | "Patients are assessed during a baseline period and then randomized (1:1) to one of two treatment sequences: IB1001 followed by placebo or vice versa...Conclusions Given the lack of global symptomatic or disease-modifying therapies for NPC and other lysosomal diseases there is an urgent need for effective and well-tolerated drug treatments. This clinical trial was designed through a collaboration between National Regulatory Agencies, leading Clinical Experts, Patient Organizations, and the industry Sponsored."

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