IntraBio Announces U.S. FDA Accepts New Drug Application for IB1001 for the Treatment of Niemann-Pick disease Type C

IntraBio Inc today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC).

The application has been granted Priority Review and was given a Prescription Drug User Fee Act (PDUFA) target action date of September 24th, 2024.

The NDA is based on the results of a Pivotal Phase 3 trial (IB1001-301) for adult and pediatric patients with NPC that met all primary and key secondary endpoints and showed IB1001 improved neurological signs and symptoms, functioning, and quality of life versus placebo. The findings from the study were recently published in the New England Journal of Medicine on February 1st, 2024.

The NDA also included data from a positive multinational Phase IIb trial of IB1001 for NPC, which also met its primary and secondary endpoints and showed improvement in symptoms, functioning, and quality of life in pediatric and adult patients with NPC with the drug being well-tolerated.

Sean Kassen, Director of the Ara Parseghian Medical Research Fund commented: “We are absolutely thrilled and filled with hope with the news of IntraBio’s NDA filing acceptance. NPC has an extremely high unmet medical need and the data from the IB1001 clinical trials demonstrated that this therapy would bring relief and improved quality of life to those affected by Niemann-Pick Type C disease. We are excited and committed to working with and supporting the NPC community and IntraBio and look forward to the eventual approval of IB1001 so all patients will benefit from this therapy.”

“The FDA’s acceptance of IntraBio’s NDA submission for IB1001 and granting of priority review brings us one step closer to our ultimate goal of delivering new, effective treatments to patient communities like NPC who have extremely high unmet medical needs,” said Mallory Factor, IntraBio’s Executive Chairman. “IntraBio remains dedicated to advancing therapies and addressing unmet medical needs, and we are optimistic IB1001 will be approved and made rapidly available for all NPC patients.”

Professor Elizabeth Berry-Kravis, Rush University Medical Center, Chicago, IL, commented: “We are tremendously excited to have potential new treatment options for our NPC patient community. Once approved, we intend to treat all our NPC patients who meet the labelling criteria with IB1001 and to carefully track long-term improvement relative to baseline with whatever therapies they are on to help elucidate the long-term effects.”

IntraBio also today announced it has recently closed an equity financing round, in which it raised over $40 million US, to support the commercialization and launch of IB1001 subject to FDA approval.

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IntraBio Announces Positive Pivotal Trial Results of IB1001 for the Treatment of Niemann-Pick Disease Type C

June 29, 2023, 8:00 AM EDT

IntraBio Inc today announced positive topline results from the pivotal, Phase III IB1001-301 clinical trial (NCT05163288), showing IB1001 significantly improved symptoms, functioning, quality of life, and cognition in pediatric and adult patients with Niemann-Pick disease Type C (NPC).

The primary endpoint of the trial evaluated the impact of IB1001 on the Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo after 12 weeks. Treatment with IB1001 demonstrated a statistically significant and clinically meaningful 1.37-point reduction of the SARA score compared to placebo (-1.97 on IB1001 vs. -0.60 on placebo; p<0.001). The trial also met its secondary endpoints, the modified Scale for the Assessment and Rating of Ataxia (mSARA) (-1.66 on IB1001 vs. on -0.67 placebo; p<0.001) and the Investigator’s Clinical Global Impression of Change (CGI-C) (-0.7 on IB1001 vs. +0.1 on placebo; p<0.001). IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events, consistent with its established, benign safety profile.

The positive IB1001-301 results are consistent with the Phase IIb multinational clinical trial previously completed with IB1001 for NPC (IB1001-201 – NCT03759639), which also showed a statistically significant and clinically meaningful improvement on the SARA, mSARA, and Investigator’s CGI-I endpoints and a benign safety profile. Based on these positive trials, IntraBio will proceed with global regulatory submissions to the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other global regulators.

“This is a critical breakthrough for the NPC patient community,” said Dr. Marc Patterson, Professor of Neurology, Pediatrics, and Medical Genetics at Mayo Clinic. “Conducting clinical trials in ultra rare, clinically variable disorders such as Niemann-Pick disease, type C, is extremely challenging, owing to the limited pool of potential participants and difficulty in demonstrating an effect in patients with existing neurological impairment. The trial of IB1001 shows clinically meaningful effects at a high level of statistical significance in patients with NPC. There is currently no approved therapy for NPC in the US, and given this convincing evidence of efficacy coupled with the very benign safety profile, I am hopeful that IB1001 will be rapidly approved, to ensure that every NPC patient will be able to access safe and effective treatment for this devastating disease.”

The National Niemann-Pick Disease Foundation’s Executive Director Joslyn Crowe commented: “We are optimistic IB1001 is quickly on track to be an approved treatment for NPC. The strong efficacy and safety results of the IB1001 clinical trial bring new levels of hope to patients and families affected by NPC. We look forward to continuing to collaborate with IntraBio to help ensure IB1001 is made available for all patients in our community given their urgent need for effective, approved treatments.”

About IB1001

IB1001 is a novel, orally administered therapy with a unique mechanism of action offering neuroprotective and symptomatic benefits for rare and common neurological disorders. IB1001 is initially being developed as a potential treatment for rare lysosomal storage disorders, including NPC, GM1 and GM2 gangliosides, and inherited cerebellar ataxias.

IB1001 has been granted 13 Orphan Drug Designations from the FDA and EMA (for the treatment of NPC, GM1 gangliosides, GM2 gangliosides (GM2), Spinocerebellar Ataxias – 40+ subtypes, Ataxia-Telangiectasia (A-T), and Ataxia Ocular Motor Apraxia type 4) and three Rare Pediatric Disease Designations (RPDD) from the US FDA for the treatment of NPC, GM2, and A-T.  IB1001 has also received Fast Track designation from the US FDA for NPC and GM2.

About IB1001-301

IB1001-301 (NCT05163288) is a multinational, randomized, placebo-controlled, crossover trial that evaluates the safety and efficacy of IB1001 in pediatric and adult patients with NPC.  Patients aged 4 years and older were screened at trial sites in Australia, Europe, the United Kingdom, and the United States.

Patients were assessed during a baseline period and then randomly assigned (1:1) to receive orally administered IB1001 or placebo for 12 weeks. At the end of the 12-week treatment period, patients crossed over and initiated therapy with the alternate study drug (IB1001 or placebo) over the subsequent 12-week period.  Patients who completed the study had the option to participate in an open-label Extension Phase.

About IntraBio

IntraBio Inc, a US biopharmaceutical company, is focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio’s platform technologies result from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio’s management team and scientific founders have a successful track record of drug development and commercialization in the USA and Europe.  IntraBio’s team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.

IntraBio Reaches Target Enrollment for IB1001 NPC Pivotal Clinical Trial

OXFORD, UK / November 22, 2022 / IntraBio Inc announced today that it has enrolled 100% of the target number of patients for its multinational pivotal trial, IB1001-301, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study”.

Enrollment for IB1001-301 commenced in September 2022, and patients were screened at trial sites in Australia, the Czech Republic, Germany, the Netherlands, Slovakia, Switzerland, the United Kingdom, and the United States. To increase the study’s power, IntraBio plans to “over enroll” the trial by over 125% and expects this additional recruitment to be completed by year-end. Data readout is anticipated before the end of the second quarter 2023.

About IB1001-301

IB1001-301 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic and long-term neuroprotective effect of treatment of Niemann-Pick disease Type C (NPC), with the primary endpoint based on the Scale for the Assessment and Rating of Ataxia. The study was designed in consultation with neurologists engaged in the treatment of NPC and NPC patient organizations.  The study has been designed to support a label for the chronic treatment of NPC symptoms.

IB1001 Development Program / Approval Pathway

This pivotal trial was also designed in consultation with both the US Food and Drug Administration (FDA) and the European Medicine Agency (EMA) to support an accelerated approval pathway for IB1001. Upon approval of IB1001, IntraBio would be eligible for a “priority review voucher” by the FDA, which can be used or sold, with all sales of these vouchers in the past 2 years over $100 million each. IntraBio is also Phase III ready for three other disease indications.

About IntraBio

IntraBio Inc is a biopharmaceutical company with a late-stage drug pipeline, including novel treatments for common and rare neurodegenerative diseases.  IntraBio’s platform technologies result from decades of research and investment at premier universities and institutions worldwide.  Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signalling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio’s management team and consultants have a successful track record of drug development in the USA and Europe.  This team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients.

IntraBio Inc is a US corporation with its principal operations in Oxford, United Kingdom.

For further information please contact:

Cass Fields – ccfields@intrabio.com

IntraBio Completes Recruitment for IB1001 NPC Pivotal Clinical Trial

OXFORD, UK / December 08, 2022 / IntraBio Inc announced today that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288).

Enrolment for IB1001-301 commenced in September 2022.  Patients were screened across 13 multinational trial sites in Australia, Europe, the United Kingdom, and the United States. Given the NPC community’s extremely high interest in IB1001-301 and their high unmet medical need, IntraBio enrolled 130% of target patients for the trial, substantially increasing the power of the study.

“We are thrilled to have completed recruitment and thankful to the dedicated Principal Investigators and Study Teams, as well as NPC Patient Organizations worldwide, who helped us reach this milestone” said Taylor Fields, IntraBio’s Chief Product Development Officer. “We are also very appreciative for the NPC community’s tremendous enthusiasm and support of our development of IB1001, which enabled us to complete recruitment so quicky. We remain determined to get IB1001 approved as quickly as possible to help meet NPC patients’ urgent unmet medical need.”

Data readout is anticipated before the end of the second quarter 2023.

About IB1001-301

IB1001-301 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic and long-term neuroprotective effect of treatment of Niemann-Pick disease Type C (NPC), with the primary endpoint based on the Scale for the Assessment and Rating of Ataxia. The study was designed in consultation with neurologists engaged in the treatment of NPC and NPC patient organizations. The study has been designed to support a label for the chronic treatment of NPC symptoms.

IB1001 Development Program / Approval Pathway

This pivotal trial was also designed in consultation with both the US Food and Drug Administration (FDA) and the European Medicine Agency (EMA) to support an accelerated approval pathway for IB1001. Upon approval of IB1001, IntraBio would be eligible for a “priority review voucher” by the FDA, which can be used or sold, with all sales of these vouchers in the past 2 years over $100 million each. IntraBio is also Phase III ready for three other disease indications.

About IntraBio

IntraBio Inc is a biopharmaceutical company with a late-stage drug pipeline, including novel treatments for common and rare neurodegenerative diseases. IntraBio’s platform technologies result from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signalling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio’s management team and consultants have a successful track record of drug development in the USA and Europe. This team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients.

IntraBio Inc is a US corporation with its principal operations in Oxford, United Kingdom. For further information, please contact:

For further information please contact:

Cass Fields
Vice-President of External Affairs
ccfields@intrabio.com
www.intrabio.com

OXFORD, UK / IntraBio Inc announced positive results from the full data set for its multinational clinical trial of IB1001 (N-acetyl-L-leucine) for the treatment of GM2 Gangliosidosis (“GM2”; Tay-Sachs and Sandhoff disease). In total, 30 subjects aged 6 to 55 years with a confirmed genetic diagnosis of GM2 were enrolled across 8 clinical trial sites in the United States, United Kingdom and Europe.

Key Findings

Efficacy

A total of 30 patients were recruited into the study and 29 patients were included in the primary modified intention-to-treat (mITT) analysis set. IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life for pediatric and adult patients with GM2 Gangliosidosis. Treatment with IB1001 resulted in a statistically significant change in the Clinical Impression of Change in Severity (CI-CS) (90% CI: 0.00, 1.50, p-value = 0.039) assessed by blinded, independent raters (professors of neurology with expertise in movement and neurological disorders).

The trial also met its secondary endpoints, including the Scale for the Assessment and Rating of Ataxia (SARA), the Investigator’s Clinical Global Impression of Change (CGI-C), and the Modified Disability Rating Scale (mDRS), demonstrating a statistically significant, clinical improvement with IB1001 treatment (SARA 90% CI: -1.75, -0.75, p<0.001; CGI-C 90% CI: -1.0, -0.5 p <0.001; mDRS 90% CI: -1.5, 0.0, p=0.02) and a statistically significant, clinical deterioration during post-treatment washout from IB1001 (SARA 90% CI: 0.5, 2.0, p<0.001; CGI-C 90% CI: 0.5, 1.0 p<0.001; mDRS 90% CI: 0.5, 1.5, p<0.001)).

Subgroup analysis of the endpoints demonstrates consistent clinical effects across all demographics (age, gender, disease severity, age of symptom onset, etc.). This positive data provides a strong rationale for IB1001 to be used in the treatment of all patients with GM2 Gangliosidosis.

Safety

IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.

Summary

The positive results of this IB1001-202 study are reinforced by the efficacy and safety profile of IB1001 already demonstrated in IntraBio’s successful IB1001-201 study for Niemann-Pick disease Type C (NPC). As is the case in the IB1001-202 clinical trial for GM2 Gangliosidosis, IB1001-201 was the first clinical trial to demonstrate statistical significance and a clinically meaningful effect in patients with NPC. These results provide further momentum for the broad clinical development program planned for IB1001 which will address high unmet medical needs for the treatment of both rare and common neurological disorders.

Impact of the GM2 Gangliosidosis Clinical Study

“The results of this study are hugely important for the GM2 community,” said Dr. Susanne Schneider, Principal Investigator and Professor of Neurology from the Ludwig Maximilian University of Munich. “IB1001 is the first drug to demonstrate a statistically significant and clinically meaningful effect for the treatment of GM2 Gangliosidosis. IB1001 has a very compelling safety profile, easy oral administration [sachet mixed with water], affirming its very favourable risk/benefit profile as a treatment for this devastating disease.”

In a joint statement, Rick Karl, President of the Cure Tay-Sachs Foundation and Dan Lewi, Chief Executive Officer of the Cure Action for Tay-Sachs Foundation, commented: “This treatment is a major breakthrough for the GM2 Gangliosidosis community that includes Tay-Sachs and Sandhoff. It is the first drug to offer hope to the patients and families affected by these devastating diseases. They are progressive, life-threatening conditions with no approved medicinal treatments. There is an urgent need for this effective treatment to be approved and made available for patients in our community before the window of therapeutic opportunity is lost.”

“IntraBio understands the importance and urgency of making IB1001 available to GM2 patients as quickly as possible”, said Jim Meyers, President & CEO of IntraBio. “We will continue to engage with regulatory authorities with respect to expediting the pathway to approval”.

IntraBio IB1001 Development

In addition to Clinical Study IB1001-202, IntraBio has completed a parallel multinational clinical trial with IB1001 for the treatment of Niemann-Pick disease Type C (NPC; NCT03759639). In September 2020, IntraBio announced the positive results of this trial (IB1001-201), which met both its primary and secondary endpoints and demonstrated a statistically significant and meaningful improvement in patients with NPC. The results of the trial have been peer-reviewed and published in the Journal of Neurology.

IntraBio is currently conducting a parallel clinical trial for IB1001 for Ataxia-Telangiectasia (A-T; NCT03759678).

Mallory Factor, IntraBio Chairman, said: “IB1001 is part of IntraBio’s broad platform of novel treatments to provide neuroprotection, disease modification and symptomatic relief from multiple neurodegenerative and lysosomal storage diseases. In addition to our immediate priority of making IB1001 available for patients with GM2 Gangliosidosis, Niemann-Pick disease Type C, and Ataxia-Telangiectasia, we will continue to investigate IB1001 for other neurological disorders with high unmet medical needs.”

About GM2 Gangliosidosis

GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and are caused by mutations in the HEXA gene, which disrupts the activity of the enzyme beta-hexosaminidase A, preventing the enzyme from breaking down GM2 gangliosides. As a result, GM2 gangliosides accumulate to toxic levels, particularly in neurons in the brain and spinal cord, leading to cell death and resulting in the signs and symptoms of Tay-Sachs and Sandhoff disease. There is nothing medically available for the treatment of GM2 Gangliosidosis at this time.

About IB1001-202 Trial

IB1001-202 (NCT03759665) is a multinational clinical trial evaluating IB1001 for the treatment of adult and pediatric patients with GM2 Gangliosidosis. Patients aged 6 years and older were enrolled at trial sites in the United States, the United Kingdom, the European Union.

IB1001 was assessed during a “Parent Study” consisting of a baseline period (with or without a study-run in), a 6-week treatment period, followed by a 6-week post-treatment washout period for examining symptomatic relief. In the “Extension Phase”, patients receive treatment with IB1001 for 1 year. Both the symptomatic and long-term benefits of treatment have previously been observed in observational clinical studies and are consistent with the pharmacological action of IB1001 demonstrated in in vitro and in vivo non-clinical studies.

IntraBio Inc announced positive data from its multinational clinical trial of IB1001 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease).

IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both the primary and secondary endpoints for pediatric and adult patients with GM2 Gangliosidosis.

The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was assessed by blinded, centralized raters (professors of neurology with expertise in movement and neurological disorders).

The trial also met secondary endpoints including the Scale for the Assessment and Rating of Ataxia (SARA), the Modified Disability Rating Scale (mDRS), the Investigators’, Caregivers’, and Patients’ Clinical Global Impression of Change (CGI-C) assessment.

IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.

“The results of this study are hugely important for the GM2 community,” said Dr. Susanne Schneider, Principal Investigator and Professor of Neurology from Ludwig Maximilian University of Munich. “IB1001 is the first drug to demonstrate a statistically significant and clinically meaningful effect for the treatment of GM2 Gangliosidosis. IB1001 has a very compelling safety profile, easy oral administration [sachet mixed with water], affirming its very favourable risk/benefit profile as a treatment for this devastating disease.”

Professor Antony Galione, FRS, FMedSci, Statutory Professor of Pharmacology, University of Oxford commented, “GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease) is a devastating disease that has never had any available treatment. We are very excited that IB1001 is the first drug that is effective for this disorder and will improve the lives of so many patients and their families. Given what is known about IB1001’s mechanism, and its multiple successful clinical trials, we will continue to investigate this drug for other rare genetic neurological diseases and for more common neurodegenerative diseases prevalent in society with large unmet medical needs.”

In a joint statement, Rick Karl, President of the Cure Tay-Sachs Foundation and Dan Lewi, Chief Executive Officer of the Cure Action for Tay-Sachs Foundation, commented: “This treatment is a major breakthrough for the GM2 Gangliosidosis community that includes Tay-Sachs and Sandhoff. It is the first drug to offer hope to the patients and families affected by these devastating diseases. They are progressive, life-threatening conditions with no approved medicinal treatments. There is an urgent need for this effective treatment to be approved and made available for patients in our community before the window of therapeutic opportunity is lost.”

The positive results of this IB1001-202 study are reinforced by the efficacy and safety profile of IB1001 already demonstrated in IntraBio’s successful IB1001-201 study for Niemann-Pick disease Type C (NPC). As is the case in the IB1001-202 clinical trial for GM2 Gangliosidosis, IB1001-201 was the first clinical trial to demonstrate statistical significance and a clinically meaningful effect in patients with NPC. These results provide further momentum for the broad clinical development program planned for IB1001 which will address high unmet medical needs for the treatment of both rare and common neurological disorders.

About IB1001-202 Trial

IB1001-202 (NCT03759665) is a multinational clinical trial evaluating IB1001 for both symptomatic and neuroprotective, disease-modifying treatment for adult and pediatric patients with GM2 Gangliosidosis. Patients aged 6 years and older were enrolled at trial sites in the United States, the United Kingdom, the European Union.

To investigate its symptomatic effects, IB1001 was assessed during a “Parent Study” consisting of a baseline period (with or without a study-run in), a 6-week treatment period, followed by a 6-week post-treatment washout period for examining symptomatic relief. In the “Extension Phase”, patients receive treatment with IB1001 for 1 year to study the neuroprotective, disease-modifying effects. Both the symptomatic and long-term benefits of treatment have previously been observed in observational clinical studies and are consistent with the pharmacological action of IB1001 demonstrated in in vitro and in vivo non-clinical studies.

In addition to Clinical Study IB1001-202, IntraBio has completed a parallel multinational clinical trial with IB1001 for the treatment of Niemann-Pick disease Type C (NPC; NCT03759639). In September 2020, IntraBio announced the positive results of this trial (IB1001-201), which met both its primary and secondary endpoints and demonstrated a statistically significant and meaningful improvement in patients with NPC.

IntraBio is currently conducting a parallel clinical trial for IB1001 for Ataxia-Telangiectasia (A-T; NCT03759678).

About IB1001

IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine that can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain. The mechanism of N-acetyl-L-leucine is multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death.

IntraBio has received Orphan Drug Designations for Acetyl-Leucine from the US Food and Drug Administration (FDA) and the European Commission for the treatment of NPC, GM2, A-T, and Spinocerebellar Ataxias (40+ subtypes). In addition, Acetyl-Leucine has been granted Rare Pediatric Disease Designations for NPC, GM2, and A-T, and Fast Track Designations for NPC and GM2 by the US FDA.

About GM2 Gangliosidosis

GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and are caused by mutations in the HEXA gene, which disrupts the activity of the enzyme beta-hexosaminidase A, preventing the enzyme from breaking down GM2 gangliosides. As a result, GM2 gangliosides accumulate to toxic levels, particularly in neurons in the brain and spinal cord, leading to cell death and resulting in the signs and symptoms of Tay-Sachs and Sandhoff disease. There is nothing medically available for the treatment of GM2 Gangliosidosis at this time.

We are delighted to announce today that IntraBio’s Co-Founding Scientist Professor Frances Platt has been elected a Fellow of The Royal Society. Professor Platt joins 52 distinguished scientists who were honoured today with their election for their exceptional contributions to science.

Professor Platt is a Professor of Biochemistry and Pharmacology and the Head of the Department of Pharmacology at the University of Oxford. Oxford’s Department of Pharmacology has been ranked the #1 in the world by QS World University Rankings since 2018.

Professor Platt’s research focuses on the biology and pathobiology of glycosphingolipids and lysosomal storage disorders. This research led to the development of miglustat for the treatment of glycosphingolipid lysosomal storage diseases including Gaucher disease and Niemann-Pick disease type C. Her research has also led to the development of IntraBio’s lead asset IB1001 as a novel treatment for rare and common neurodegenerative diseases and lysosomal storage disorders. Three multinational clinical trials with IB1001 are ongoing for Niemann-Pick disease Type C (NPC, positive data recently reported), GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Ataxia Telangiectasia (A-T).

Professor Platt commented, “It’s a great honour to have been elected and I hope this will help raise awareness about the devastating diseases we work on. I am truly indebted to past and present members of my lab and our collaborators for their outstanding work over the years”.

In addition to being elected to The Royal Society, Professor Platt was elected a Fellow of the Academy of Medical Sciences in 2011, received a Royal Society Wolfson Merit Award in 2013, and in 2016 she became a Wellcome Trust Investigator in Science. She has been awarded the Alan Gordon Memorial Award and the Horst Bickel Award for advances in metabolic disease therapy.

We warmly congratulate Professor Platt on this distinguished and well-deserved honour.

 Professor Platt joins IntraBio Co-Founding Scientist Professor Antony Galione as a Fellow of the Royal Society (FRS).

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

Oxford, U.K. — IntraBio Inc today announced positive results from the full data set for its multinational clinical trial of IB1001 (N-acetyl-L-leucine) for the treatment of Niemann-Pick disease Type C (NPC).  In total, 33 subjects aged 7 to 64 years with a confirmed diagnosis of NPC were enrolled across 9 clinical trial sites in the United States, United Kingdom, and Europe.

Key Findings

Efficacy

A total of 33 patients were recruited into the study and 32 patients were included in the mITT analysis set. IB1001 demonstrated a statistically significant and clear clinically meaningful improvement in symptoms, functioning, and quality of life for pediatric and adult patients with NPC.  Treatment with IB1001 resulted in a statistically significant change in the Clinical Impression of Change in Severity (CI-CS) (90% CI: 0.25, 1.75, p-value = 0.029) assessed by blinded, independent raters.

The trial also met its secondary endpoints, including the Scale for the Assessment and Rating of Ataxia (SARA), Investigator’s Clinical Global Impression of Change (CGI-C) demonstrating both a statistically significant change during treatment (clinical improvement; SARA 90% CI: -1.8, -0.5,  p=0.001; CGI-C 90% CI: 3.0, 3.5, p <0.001) and during post-treatment washout (clinical deterioration; SARA 90% CI: 0.5, 2.0, p=0.002; CGI-C 90% CI: 4.0, 5.0, p=0.006). There was no meaningful difference in these endpoints detected between the baseline and washout period, bolstering the statistical significance and clinically meaningful effect of IB1001 established by the primary and secondary endpoints.

Subgroup analysis of the primary and secondary endpoints demonstrates consistent clinical effects across all demographics (age, gender, disease severity, age of symptom onset, etc.). This positive data provide a strong rationale for IB1001 to be used in the treatment of all patients with NPC.

Safety

IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.

Next Steps for NPC Clinical Study – IB1001-201

An Extension Phase with IB1001 is ongoing to confirm the treatment’s long-term neuroprotective effects. The rationale for IB1001’s disease-modifying effect is strongly supported by earlier non-clinical studies in the NPC mouse model and results from long-term clinical case-series for NPC. IntraBio understands the urgency of making IB1001 available to NPC patients as soon as possible and will continue to engage with regulatory agencies with respect to an accelerated approval path.

Professor Susanne Schneider, MD, Principal Investigator, Ludwig Maximilians University of Munich said: “This treatment is a breakthrough for the NPC patient community. The statistically significant and clinically meaningful response in primary and topline secondary endpoints with IB1001, together with its compelling safety profile, easy oral administration [sachet mixed with water] affirm the very favorable risk/benefit profile of IB1001 as a treatment for this devastating disease.”

The National Niemann-Pick Disease Foundation’s Executive Director Joslyn Crowe commented: “The exciting results of the IB1001 clinical trial bring new levels of hope and optimism to patients and families affected by Niemann-Pick disease type C.  There continues to be a critical need for new therapies to treat NPC and we are so grateful to our partners like IntraBio for their continued commitment to finding new treatments like IB1001.”

Next Steps for IntraBio

In addition to Clinical Study IB1001-201, IntraBio is completing parallel multinational clinical trials with IB1001 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease; NCT03759665) and Ataxia-Telangiectasia (A-T; NCT03759678). The IB1000 series has demonstrated efficacy (non-clinical and/or clinical) for a broad series of rare, common, and acquired neurological disorders including Alzheimer’s Traumatic Brain Injury, and Dementias, which IntraBio is preparing to initiate clinical trials for with IB1001.

Mallory Factor, Chairman, said: “IB1001 is part of IntraBio’s broad platform of novel treatments to provide neuroprotection, disease modification and symptomatic relief from multiple neurodegenerative and lysosomal storage diseases. In addition to our immediate priority of making IB1001 available for patients with NPC, GM2 Gangliosidosis, and Ataxia-Telangiectasia, we will continue to investigate the neuroprotective, disease-modifying, and symptomatic treatment effects for other neurological disorders with unmet medical needs.”

About Niemann-Pick Disease Type C

Niemann-Pick disease Type C (NPC) is a rare (1:100,000 live births), prematurely fatal, autosomal recessive, lysosomal storage disorder.  The disease begins in early childhood and presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia.  NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life.  There is no approved treatment for NPC in the United States.  Treatment of NPC in the European Union and several other countries is limited to substrate reduction therapy drug miglustat (Zavesca™).

About IB1001-201 Trial

IB1001-201 (NCT03759639) is a multinational clinical trial evaluating IB1001 for both the symptomatic and neuroprotective, disease-modifying treatment for adult and pediatric patients with NPC.  Patients aged 6 years and older were enrolled at trial sites in the United States, the United Kingdom, the European Union.

To investigate both the symptomatic and neuroprotective, disease-modifying effects of treatment, IB1001 is assessed during two treatment sequences: a “Parent Study” consisting of a baseline period (with or without a study-run in), a 6-week treatment period, followed by a 6-week post-treatment washout period as an internal control for examining symptomatic relief, and an “Extension Phase”, during which patients receive treatment with IB1001 for 1 year to study the neuroprotective, disease-modifying effects.  Both the symptomatic and long-term benefits of treatment have previously been observed in observational clinical studies and are consistent with the pharmacological action of IB1001 demonstrated in in vitro and in vivo non-clinical studies.

About IntraBio

IntraBio Inc is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases.  IntraBio’s platform technologies result from decades of research and investment at premier universities and institutions worldwide.  Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio’s management team and consultants have a successful track record of drug development in the USA and Europe. IntraBio’s team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.

IntraBio Inc is a US corporation with its principal operations in Oxford, United Kingdom.

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

Oxford, U.K. — IntraBio Inc today announced positive data from its multinational clinical trial of IB1001 for the treatment of Niemann-Pick disease Type C (NPC).

IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC.

The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was assessed by blinded, centralized raters.

The trial also met topline secondary endpoints, including the Scale for the Assessment and Rating of Ataxia (SARA) and the Investigators’ Clinical Global Impression of Change (CGI-C) assessment.

IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.

“This treatment is a breakthrough for the NPC patient community,” said Professor Susanne Schneider, MD, Principal Investigator, Ludwig Maximilians University of Munich.  “The statistically significant and clinically meaningful response in primary and topline secondary endpoints with IB1001, together with its compelling safety profile, easy oral administration [sachet mixed with water] affirm the very favorable risk/benefit profile of IB1001 as a treatment for this devastating disease.”

“IB1001 is part of IntraBio’s broad platform of novel treatments to provide neuroprotection, disease modification, and symptomatic relief from multiple neurodegenerative and lysosomal storage diseases,” noted Mallory Factor, Chairman of IntraBio Inc.  “We are currently trialing IB1001 treatment of GM2 (Tay-Sachs and Sandhoff disease) and Ataxia-Telangiectasia (AT).  IntraBio is preparing to initiate clinical trials with IB1001 for other rare and neurodegenerative diseases.”

 The National Niemann-Pick Disease Foundation’s Executive Director Joslyn Crowe commented: “The exciting results of the IB1001 clinical trial bring new levels of hope and optimism to patients and families affected by Niemann-Pick disease type C.  There continues to be a critical need for new therapies to treat NPC and we are so grateful to our partners like IntraBio for their continued commitment to finding new treatments like IB1001.”

About IB1001-201 Trial
IB1001-201 (NCT03759639) is a multinational clinical trial evaluating IB1001 for both the symptomatic and neuroprotective, disease-modifying treatment for adult and pediatric patients with NPC.  Patients aged 6 years and older were enrolled at trial sites in the United States, the United Kingdom, the European Union.

To investigate both the symptomatic and neuroprotective, disease-modifying effects of treatment, IB1001 is assessed during two treatment sequences: a “Parent Study” consisting of a baseline period (with or without a study-run in), a 6-week treatment period, followed by a 6-week post-treatment washout period for examining symptomatic relief, and an “Extension Phase”, during which patients receive treatment with IB1001 for 1 year to study the neuroprotective, disease-modifying effects.  Both the symptomatic and long-term benefits of treatment have previously been observed in observational clinical studies and are consistent with the pharmacological action of IB1001 demonstrated in in vitro and in vivo non-clinical studies.

In addition to Clinical Study IB1001-201, IntraBio is completing parallel multinational clinical trials with IB1001 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease; NCT03759665) and Ataxia-Telangiectasia (A-T; NCT03759678).  IntraBio is also preparing to initiate clinical trials of  IB1001 for other rare and neurodegenerative diseases with high unmet medical needs.

About IB1001
IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid.  In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine and can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain.  The mechanism of N-acetyl-L-leucine is known to be multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death.

Professor Antony Galione, FRS, FMedSci, Statutory Professor of Pharmacology, University of Oxford commented, “We are particularly excited from what we know from in vitro and in vivo studies into the cellular mechanisms that this drug has the potential to ameliorate not only rare genetic neurological diseases like NPC, but also some of the more common neurodegenerative diseases prevalent in society with large unmet medical needs.”

IntraBio has received Orphan Drug Designations for Acetyl-Leucine from the US Food and Drug Administration (FDA) and the European Commission for the treatment of NPC, GM2, A-T, and Spinocerebellar Ataxias (40+ subtypes).  In addition, Acetyl-Leucine has been granted Rare Pediatric Disease Designations for NPC, GM2, and A-T, and Fast Track Designations for NPC and GM2 by the US FDA.

About Niemann-Pick disease Type C
Niemann-Pick disease Type C (NPC) is a rare (1:120,000 live births), prematurely fatal, autosomal recessive, lysosomal storage disorder.  The disease begins in early childhood and presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia.  NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life.  There is no approved treatment for NPC in the United States.  Treatment of NPC in the European Union and several other countries is limited to substrate reduction therapy drug miglustat (Zavesca™).

About IntraBio
IntraBio Inc is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases.  IntraBio’s platform technologies result from decades of research and investment at premier universities and institutions worldwide.  Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio’s management team and consultants have a successful track record of drug development in the USA and Europe.  IntraBio’s team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.

IntraBio Inc is a US corporation with its principal operations in Oxford, United Kingdom.

OXFORD, UK — IntraBio Inc announced today that their Chief Clinician and Co-Founding Scientist Professor Michael Strupp MD, FRCP, FAAN, FANA, FEAN received the 2020 “Galenus-von-Pergamon” Basic Research Award for his groundbreaking work on a new therapeutic principle for lysosomal storage disorders (LSDs).

The Basic Research Galenus-von-Pergamon Award is granted to an individual or team for their outstanding scientific achievements in the understanding and advancement of pharmacological research. Professor Strupp received the 2020 Award for his clinical and pre-clinical work with N-Acetyl-Leucine. This was done in close collaboration with Prof. Frances Platt, Dr. Ecem Kaya, Dr. Grant Churchill, Prof. Antony Galione and others from the Department of Pharmacology at the University of Oxford, UK as well as Dr. Tatiana Bremova-Ertl, now Department of Neurology at the University of Bern, CH.

N-acetyl-L-leucine (IB1001) is being developed by IntraBio as a novel treatment for rare and common neurodegenerative diseases and LSDs. IntraBio was founded to develop new therapeutic principles for LSDs, and Professor Strupp’s and his colleagues’ research serves as the scientific basis for their platform. Three multinational clinical trials with IB1001 are ongoing for Niemann-Pick disease Type C (NPC, positive data recently reported), GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Ataxia Telangiectasia (A-T).

“We are very pleased to congratulate Michael Strupp and his team on his well-deserved award for his innovative research and development of IB1001,” said IntraBio’s Chairman Mallory Factor. “We are encouraged by the international scientific community’s recognition of Professor Strupp’s scientific breakthrough with IB1001 and are committed to moving forward to bring this treatment to the patients who suffer from these rare and devastating diseases.”

IntraBio recently reported positive data from its multinational clinical trial with IB1001 for the treatment of NPC, where IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and secondary endpoints for both pediatric and adult patients with NPC.

About Professor Michael Strupp, MD, FRCP, FANA, FEAN
Professor Strupp is a Scientific Co-Founder of IntraBio Inc, and a Professor at the Ludwig Maximilians University of Munich, Germany in the Department of Neurology and German Centre for Vertigo and Balance Disorders. He has authored 407 PubMed listed papers (h-index 75; i-10 index 269) and five books. He is Joint Chief Editor of the Journal of Neurology, Editor in Chief of Frontiers of Neuro-otology, and Section Editor of F1000.

 

About IB1001
IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine and can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain. The mechanism of N-acetyl-L-leucine is known to be multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death.

IntraBio has received Orphan Drug Designations for Acetyl-Leucine from the US Food and Drug Administration (FDA) and the European Commission for the treatment of NPC, GM2, A-T, and Spinocerebellar Ataxias (40+ subtypes). In addition, Acetyl-Leucine has been granted Rare Pediatric Disease Designations for NPC, GM2, and A-T, and Fast Track Designations for NPC and GM2 by the US FDA.

About IB1001 Clinical Trials
Clinical Study IB1001-201 (NCT03759639) is a multinational clinical trial evaluating IB1001 for both the symptomatic and neuroprotective, disease-modifying treatment for adult and pediatric patients with NPC. Patients aged 6 years and older were enrolled at trial sites in the United States, the United Kingdom, the European Union.

In addition to Clinical Study IB1001-201, IntraBio is completing parallel multinational clinical trials with IB1001 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease; NCT03759665) and Ataxia-Telangiectasia (A-T; NCT03759678). IntraBio is also preparing to initiate clinical trials of IB1001 for other rare and neurodegenerative diseases with high unmet medical needs.

 

About IntraBio
IntraBio Inc is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases. IntraBio’s platform technologies result from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio’s management team and consultants have a successful track record of drug development in the USA and Europe. IntraBio’s team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.

IntraBio Inc is a US corporation with its principal operations in Oxford, United Kingdom

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