AMT-162 / Apic Bio, uniQure - LARVOL DELTA

uniQure Announces First Quarter 2025 Financial Results and Highlights of Recent Company Progress (GlobeNewswire) - "AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – The Company continues to screen patients across 12 clinical sites, with plans to activate additional centers in the second half of the year. The Company expects to present initial safety and exploratory efficacy data from the first treated patient at the Epilepsy Therapies & Diagnostics Development Symposium on Thursday, May 29, 2025....AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – In January 2025, the Company announced a favorable recommendation from the IDMC based on the review of initial safety data from the first study cohort in the Phase I/II EPISOD1 study. The Company has completed enrollment in a second dose cohort and expects to initiate enrollment in the third dose cohort in the third quarter of 2025. The Company expects to present initial data from the study in the first half of 2026."

P1/2 data • Amyotrophic Lateral Sclerosis • Epilepsy

uniQure Announces 2024 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "A separate Type B meeting to discuss the pivotal statistical analysis plan for the BLA is anticipated to take place in the second quarter of 2025...the Company expects to provide a regulatory update in the second quarter of 2025, including the expected timing of a potential BLA submission. In February 2025, the Company completed enrollment of all 12 patients in the third cohort investigating an optimized immunosuppression regimen. The Company expects to provide an initial safety update on the third cohort in the second quarter of 2025. In the third quarter of 2025, the Company expects to present data from its ongoing Phase I/II studies of AMT-130...AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE)...The Company expects to present initial data from the study in the first half of 2026...AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS)...expects to present initial data from the study in the first half of 2026."

FDA event • FDA filing • P1/2 data • Amyotrophic Lateral Sclerosis • Epilepsy • Huntington's Disease

uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS (GlobeNewswire) - "Company Expects to Initiate Enrollment of Second Dose Cohort in the First Quarter of 2025...uniQure N.V...today announced that the Independent Data Monitoring Committee (IDMC) for EPISOD1, uniQure’s Phase I/II clinical trial of AMT-162, an investigational gene therapy for amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, has met and reviewed 28-day safety data from the first study cohort. The IDMC’s review identified no significant safety concerns and recommended proceeding with enrollment in the second cohort."

DSMB • Enrollment status • Amyotrophic Lateral Sclerosis

EPISOD1: A Phase 1/2, multicenter study to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162 in patients with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (ALS-MND 2024) - P1/2 | "The clinical study design (NCT06100276) presented here is the first-in-human clinical trial using the investigational gene therapy AMT-162 focused on safety, tolerability, and exploratory efficacy in ALS patients with SOD1 mutations."

Clinical • Gene therapy • P1/2 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • Plasma NfL • SOD1

uniQure Announces Dosing of First Patient in Phase I/II Clinical Trial of AMT-162 for the Treatment of SOD1-ALS (GlobeNewswire) - "uniQure...announced that the first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-ALS....There are currently four active sites in the U.S. with plans to activate seven additional sites by the first quarter of 2025."

Trial status • Amyotrophic Lateral Sclerosis

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - P1/2 | N=20 | Recruiting | Sponsor: UniQure Biopharma B.V. | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • SOD1

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - P1/2 | N=20 | Not yet recruiting | Sponsor: UniQure Biopharma B.V. | N=12 ➔ 20

Enrollment change • Gene therapy • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • SOD1

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: UniQure Biopharma B.V. | N=42 ➔ 12 | Trial completion date: Feb 2032 ➔ Mar 2031 | Initiation date: Feb 2024 ➔ May 2024 | Trial primary completion date: Jun 2026 ➔ Sep 2026

Enrollment change • Gene therapy • Trial completion date • Trial initiation date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • SOD1

uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "Patient dosing is ongoing in a third cohort of up to 12 patients to further evaluate both doses of AMT-130...Enrollment in this third cohort is expected to be completed in the second half of 2024....AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – Patient enrollment in a Phase I/II clinical trial is expected to begin in the third quarter of 2024."

Enrollment status • Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease

Safety, Tolerability, and Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Amyotrophic Lateral Sclerosis (ALS) Patients With SOD1 Mutations (clinicaltrials.gov) - P1/2 | N=42 | Not yet recruiting | Sponsor: UniQure Biopharma B.V. | Initiation date: Nov 2023 ➔ Feb 2024

Gene therapy • Trial initiation date • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • SOD1

uniQure Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "In the first quarter of 2024, the Company plans to request regulatory interactions with the Food and Drug Administration (FDA) to discuss the U.S. and EU data and potential late-stage development pathways for AMT-130....AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS)....The Company expects to initiate patient screening in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter of 2024."

European regulatory • FDA event • New trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease

Safety, Tolerability, and Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in ALS Patients With SOD1 Mutations (clinicaltrials.gov) - P1/2 | N=42 | Not yet recruiting | Sponsor: UniQure Biopharma B.V.

Gene therapy • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • SOD1

uniQure Announces Major Presence at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting (GlobeNewswire) - "uniQure N.V...today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium."

P1/2 data • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease

uniQure Announces Strategic Reorganization to Reduce Operating Expenses and Support Advancement of Multiple Clinical-Stage Programs (GlobeNewswire) - "Prioritizing continued development of AMT-130 in Huntington’s disease and near-term...uniQure N.V...today announced a strategic reorganization that will significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept....The company will focus its research efforts on a limited number of projects believed to have optimal risk, value and speed attributes, including AMT-161 for c9orf72 amyotrophic lateral sclerosis (ALS), AMT-240 for autosomal dominant Alzheimer's disease, and next-generation AAV capsid development. As a result of the reprioritization, the Company will be closing a research lab in Lexington and plans to sublease this space."

Pipeline update • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease

uniQure Announces First Quarter 2023 Financial Results and Highlights Recent Company Progress (GlobeNewswire) - "Expanding the Pipeline and Progress Towards Investigational New Drug (IND) Applications: AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis...The Company expects to initiate a Phase I/II clinical study of AMT-162 in the second half of 2023; AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) – In the third quarter of 2022, the Company initiated a GLP toxicology study of AMT-260 in non-human primates. The Company expects to submit an investigational new drug (IND) application and initiate a Phase I/II clinical study of AMT-260 in the second half of 2023; AMT-191 for the treatment of Fabry disease – In the third quarter of 2022, the Company initiated a GLP toxicology study of AMT-191 in non-human primates which is expected to support an IND submission in 2023."

IND • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Epilepsy • Fabry Disease • Genetic Disorders

uniQure to Participate in Multiple Upcoming Industry Conferences in March (GlobeNewswire) - “uniQure N.V…announced its participation in the following upcoming investor and scientific conferences: Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19 – 22, 2023 Austin, TX: Meg Bradbury, M.S., CGC, MSHS, director of clinical translation, will present a pre-recorded overview on both AMT-162, a clinical-stage gene therapy candidate in SOD1 amyotrophic lateral sclerosis (ALS), and AMT-161, a preclinical gene therapy candidate in c9orf72 ALS mutation on Tuesday, March 21....International Conference on Alzheimer’s & Parkinson’s Disease (AD/PD), March 28 – April 1, 2023 Gothenburg, Sweden: Amila Zuko...will deliver an oral presentation on preclinical mouse data on an intrastriatally delivered AAV product candidate focused on a combination approach of increasing protective APOE variants and lowering toxic APOE variants as a therapy for Alzheimer Disease.”

Clinical • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders

uniQure and Apic Bio enter into global licensing agreement for APB-102, a clinical stage gene therapy for patients with ALS caused by mutations in SOD1 (GlobeNewswire) - "uniQure N.V...and Apic Bio...today announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders....Under the terms of the agreement, uniQure will make an initial cash payment of $10 million. In addition, uniQure will pay Apic Bio up to $45 million in milestones upon achievement of regulatory approvals in the U.S. and Europe and pre-specified annual net sales, and a tiered royalty on net sales ranging from the mid-single digits to low double digits."

Licensing / partnership • Amyotrophic Lateral Sclerosis • CNS Disorders

Development of a ddPCR Based Relative Potency Assay for APB-102 (ASGCT 2022) - "An accelerated stability study showed that this potency assay is stability indicating and is more sensitive than a traditional TCID50 infectivity assay. The assay has been revised and streamlined to simplify the method after transfer to Quality Control for use as a release assay."

Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • Respiratory Diseases • SOD1

Apic Bio Receives FDA Fast Track Designation for APB-102 for the Treatment of Patients with SOD1 ALS (Businesswire) - “Apic Bio…announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to APB-102, the Company’s lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS). Apic plans to commence a Phase 1/2 clinical trial in early 2022.”

Fast track designation • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Apic Bio Announces FDA Clearance of IND Application for Lead Gene Therapy Candidate APB-102 for the Treatment of SOD1 ALS (Businesswire) - “Apic Bio…announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for APB-102, the Company’s lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS)...The Phase 1/2 clinical trial which will be initiated in late 2021/early 2022 is a multi-center, three-part study to evaluate the safety, tolerability, and efficacy of intrathecally administered APB-102 in patients with SOD1 ALS mutations: part I, single ascending dose; part II, randomized, double-blind, placebo-controlled; and part III, extended follow-up.”

IND • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Apic Bio’s APB-102 receives orphan drug designation from the FDA for the treatment of genetic SOD1 ALS (Yahoo Finance) - “Apic Bio…announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to APB-102, a gene therapy soon to be in clinical development for the treatment of genetic SOD1 amyotrophic lateral sclerosis (ALS).”

Orphan drug