IntraBio Inc

01/21/2026

IntraBio today announced positive pivotal Phase III trial results of levacetylleucine for the treatment of Ataxia-Telangiectasia, with statistically significant and clinically meaningful improvements across primary and key secondary endpoints. These data support planned regulatory submissions in the United States, Europe, and additional jurisdictions.

IntraBio Inc. today announced positive topline results from its pivotal Phase III IB1001-303 clinical trial, “Effects of N-Acetyl-L-Leucine on Ataxia-Telangi...

01/21/2026

IntraBio is proud to announce European Commission approval of AQNEURSA® for the treatment of Niemann-Pick Type C disease.

IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA® (levacetylleucine) for the treatment of neurological ...

10/16/2025

At IntraBio the true measure of progress is the impact on patients and families.

We are deeply grateful to Alec Koujaian and his mom, Gail, for sharing their journey with Niemann-Pick disease Type C (NPC) - from the uncertainty of diagnosis to today, living with treatment on AQNEURSA (levacetylleucine).

Their story highlights the importance of patient voices and the difference AQNEURSA can make within the NPC community - bringing hope and meaningful change to patients and families.
INDICATION
AQNEURSA (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

IMPORTANT SAFETY INFORMATION
Do not take AQNEURSA if you:
• have not had a negative pregnancy test. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments
Before taking AQNEURSA, discuss with your doctor if you:
• are pregnant or planning to become pregnant. Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments.
• require the need for effective contraception during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
• are breastfeeding or plan to breastfeed. It is not known if AQNEURSA or its metabolites pass into human or animal milk or effects on breastmilk production
• are taking P-gp substrate. Your doctor will monitor you for related adverse reactions
• have any other medical conditions
• are taking any medications and supplements, including N-acetyl-DL-leucine or N-acetyl-D-leucine, as some of these may impact the effectiveness of AQNEURSA
The most common side effects of AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections and vomiting. Speak with your doctor if these side effects persist or worsen.

Take AQNEURSA exactly as prescribed by your doctor.

If a dose of AQNEURSA is missed, skip the missed dose and take the next dose at the scheduled time. Do not take 2 doses at the same time to make up for a missed dose.

AQNEURSA can be taken with or without food.

You are encouraged to report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please click here for Full Prescribing Information for AQNEURSA:https://www.aqneursa.com/wp-content/prescribing-information.pdf

MAT-0153
10/2025

10/08/2025

Niemann-Pick Disease Awareness Month reminds us that while rare diseases may affect only a few individuals, together their voices create a powerful impact.

The INPDA International Niemann-Pick Disease Alliance and its member organizations play a vital role in amplifying those voices, uniting the community, and driving forward awareness.

At IntraBio, we are proud to stand alongside these organizations in raising awareness of Niemann-Pick disease and highlighting the importance of collaboration to improve the lives of those affected.

10/01/2025

October is Niemann-Pick Disease Awareness Month.

At IntraBio, we are proud to celebrate and stand with the global Niemann-Pick community. Throughout this month, we honor the patients, families, clinicians, and advocates whose strength and dedication continue to shine a light on Niemann-Pick disease and its impact on the lives of those affected.

We are grateful to be part of this community and remain committed to supporting awareness alongside those who work tirelessly to make a difference.

09/24/2025

Today we celebrate one year since the FDA approval of AQNEURSA® (levacetylleucine) for Niemann-Pick disease Type C (NPC) on September 24, 2024.

This milestone marked an important step forward for patients and families living with NPC. Over the past year, we have been energized by the strong engagement from healthcare providers and the dedication of the NPC advocacy community. Most importantly, we are inspired by the voices of patients, families, and advocates who remind us what this community means.

We are proud to celebrate this first year together and remain committed to supporting patient access and advancing care for everyone affected by NPC in the US.

INDICATION
AQNEURSA® (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

IMPORTANT SAFETY INFORMATION
Do not take AQNEURSA if you:
· have not had a negative pregnancy test. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments
Before taking AQNEURSA, discuss with your doctor if you:
· are pregnant or planning to become pregnant. Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments.
· require the need for effective contraception during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
· are breastfeeding or plan to breastfeed. It is not known if AQNEURSA or its metabolites pass into human or animal milk or effects on breastmilk production
· are taking P-gp substrate. Your doctor will monitor you for related adverse reactions
· have any other medical conditions
· are taking any medications and supplements, including N-acetyl-DL-leucine or N-acetyl-D-leucine, as some of these may impact the effectiveness of AQNEURSA
The most common side effects of AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections and vomiting. Speak with your doctor if these side effects persist or worsen.

Take AQNEURSA exactly as prescribed by your doctor.

If a dose of AQNEURSA is missed, skip the missed dose and take the next dose at the scheduled time. Do not take 2 doses at the same time to make up for a missed dose.

AQNEURSA can be taken with or without food.

You are encouraged to report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please click here for Full Prescribing Information for AQNEURSA: https://lnkd.in/eZaW2t2R

08/19/2021

IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease)

Multinational clinical trial is the first successful clinical trial for GM2 Gangliosidosis; favourable safety and efficacy data consistent with previously announced IB1001 clinical trial results for NPC OXFORD, UK / ACCESSWIRE / August 19, 2021 / IntraBio Inc today announced positive data from its m...

09/30/2020

IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C

01/16/2020

IntaBio Neuroprotection and Disease Modification for Niemann-Pick C Extension Phase Approved in Europe

OXFORD, UK / ACCESSWIRE / January 15, 2020 / IntraBio Inc., a late-stage biopharmaceutical company, is pleased to share that the Extension Phase for the IB1001-201 Clinical Trial has been approved in all European countries where the trial is being conducted. IB1001-201 is a multinational, clinical t...

03/13/2019

IntraBio Investigational New Drug Application Approved by the FDA for the Treatment of Ataxia-Telangiectasia

OXFORD, UK / ACCESSWIRE / March 13, 2019 / IntraBio Inc., a late-stage biopharmaceutical company, today announced that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) Application for Clinical Trial IB1001-203 with its lead compound (IB1001) for the treatment...

03/11/2019

IntraBio Clinical Trial Authorisation Application Approved by the MHRA for the treatment of Niemann-Pick Disease Type C

OXFORD, UK / ACCESSWIRE / March 11, 2019 / IntraBio Inc., a late-stage biopharmaceutical company, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved its Clinical Trial Authorisation (CTA) Application for Clinical Trial IB1001-201 with its lead compoun...