Periodic Reporting for period 1 - Gate2Brain (Gate2Brain. Medicines beyond barriers.)
Periodo di rendicontazione: 2023-04-01 al 2024-03-31
One every four of us will experience a brain disorder during our life. Despite ongoing efforts by research institutions and companies worldwide to develop drug candidates for nearly all these conditions, a significant bottleneck exists: the blood-brain barrier (BBB). While the BBB naturally safeguards the brain, it also poses a challenge by preventing approximately 98% of drug candidates from crossing, leading to their failure in development and denying potential treatments for affected patients.
Gate2Brain’s mission is to overcome this hurdle by efficiently transporting therapeutics across biological barriers like the BBB. For instance, our flagship product, G2B-002, aims to address the delivery obstacles in treating rare paediatric brain tumours using our innovative drug delivery technology. Given the incurable nature of these tumours, there exists a pressing and unmet medical need.
Our platform technology relies on three patented families of peptide shuttles, which enhance the biopharmaceutical properties of cargoes such as small molecules, peptides, nanoparticles, proteins and antibodies, both in vitro and in vivo when conjugated with them.
With G2B-002, our objective is to initiate clinical trials in paediatric brain tumours with an intact BBB, such as Diffuse Midline Glioma or paediatric glioblastoma, both considered high-grade gliomas, by 2026. The EIC Accelerator program is crucial in advancing our preclinical regulatory studies, bringing closer to achieving the Investigational Medicinal Product Dossier (IMPD) and phase I/IIa clinical trial.
These advancements will not only validate the Gate2Brain technology but also position it for broader application in other drug candidates requiring enhanced transport mechanisms.
Gate2Brain’s mission is to overcome this hurdle by efficiently transporting therapeutics across biological barriers like the BBB. For instance, our flagship product, G2B-002, aims to address the delivery obstacles in treating rare paediatric brain tumours using our innovative drug delivery technology. Given the incurable nature of these tumours, there exists a pressing and unmet medical need.
Our platform technology relies on three patented families of peptide shuttles, which enhance the biopharmaceutical properties of cargoes such as small molecules, peptides, nanoparticles, proteins and antibodies, both in vitro and in vivo when conjugated with them.
With G2B-002, our objective is to initiate clinical trials in paediatric brain tumours with an intact BBB, such as Diffuse Midline Glioma or paediatric glioblastoma, both considered high-grade gliomas, by 2026. The EIC Accelerator program is crucial in advancing our preclinical regulatory studies, bringing closer to achieving the Investigational Medicinal Product Dossier (IMPD) and phase I/IIa clinical trial.
These advancements will not only validate the Gate2Brain technology but also position it for broader application in other drug candidates requiring enhanced transport mechanisms.
Gate2Brain objective is to develop an innovative drug delivery technology capable of efficiently transporting therapeutic across biological barriers, particularly the Blood-Brain Barrier (BBB). Through this technology, we aim to address the significant challenge of delivering drugs to the Central Nervous System (CNS), thus unlocking new treatment possibilities for devastating paediatric brain diseases such as paediatric high-grade gliomas using our flagship product G2B-002.
In the manufacturing development, thorough evaluations were conducted on the active pharmaceutical ingredient (API) and raw materials essentials for G2B-002 production. A reliable supplier of key components was secured and Gate2Brain established a contingency plan for alternative raw material providers to ensure uninterrupted production. A new patent will be filled during Q2 regarding the manufacturing process.
In preclinical studies using animal testing, preliminary studies were completed to establish crucial parameters such as the maximum tolerated dose and repeated-dose toxicity. Gate2Brain has also initiated the development of a full non-clinical toxicology and safety study for rodents and non-rodents in order to ensure safety during the clinical phase with paediatric patient population with high-grade gliomas.
On the regulatory front, Gate2Brain achieved recognition as a small- and medium-sized enterprise (SME) by the EMA and developed a comprehensive regulatory roadmap for G2B-002. Preparations for a Pre-submission Meeting with the Committee for Orphan Drug Product are well underway, with all necessary documentation and presentations prepared.
Overall, Gate2Brain’s concerted efforts across manufacturing development, preclinical studies and regulatory affair have positioned G2B-002 for continued advancement towards clinical trials and eventual market approval.
In the manufacturing development, thorough evaluations were conducted on the active pharmaceutical ingredient (API) and raw materials essentials for G2B-002 production. A reliable supplier of key components was secured and Gate2Brain established a contingency plan for alternative raw material providers to ensure uninterrupted production. A new patent will be filled during Q2 regarding the manufacturing process.
In preclinical studies using animal testing, preliminary studies were completed to establish crucial parameters such as the maximum tolerated dose and repeated-dose toxicity. Gate2Brain has also initiated the development of a full non-clinical toxicology and safety study for rodents and non-rodents in order to ensure safety during the clinical phase with paediatric patient population with high-grade gliomas.
On the regulatory front, Gate2Brain achieved recognition as a small- and medium-sized enterprise (SME) by the EMA and developed a comprehensive regulatory roadmap for G2B-002. Preparations for a Pre-submission Meeting with the Committee for Orphan Drug Product are well underway, with all necessary documentation and presentations prepared.
Overall, Gate2Brain’s concerted efforts across manufacturing development, preclinical studies and regulatory affair have positioned G2B-002 for continued advancement towards clinical trials and eventual market approval.
Gate2Brain aims to enhance patients’ quality of life by offering a technology that improves drug transport effectiveness while minimizing side effects. Through extensive testing in rodent, we have confirmed the efficacy and safety approach and now poised to transition to non-rodent models, bringing us closer to human application in clinical trials.
Our technology exhibits several key advantages, including protease resistance across various species, the ability to transport a diverse range of cargoes, low production costs, and minimal immunogenicity. By overcoming societal and economic barriers, we strive to make medicines accessible to all, particularly paediatric patients worldwide.
A significant milestone in this direction is the optimization and patenting of a cost-effective scale-up manufacturing process, paving the way for an affordable treatment option while the potential to impact paediatric patients globally.
In essence, Gate2Brain offers a groundbreaking delivery technology that enables non-invasive, non-antigenic, permeable, stable, soluble, and receptor-specific drug transport across the blood brain barrier (BBB) and into the central nervous system (CNS), positioning us on the path to clinical trials.
To expedite this journey to patients, we are actively addressing regulatory considerations, including seeking Orphan Drug Designation (ODD) for G2B-002 and engaging with the European Medicines Agency (EMA) through Scientific Advice. Additionally, we are strategizing our approach to market access for G2B-002 to ensure its availability and affordability for those in need.
Our technology exhibits several key advantages, including protease resistance across various species, the ability to transport a diverse range of cargoes, low production costs, and minimal immunogenicity. By overcoming societal and economic barriers, we strive to make medicines accessible to all, particularly paediatric patients worldwide.
A significant milestone in this direction is the optimization and patenting of a cost-effective scale-up manufacturing process, paving the way for an affordable treatment option while the potential to impact paediatric patients globally.
In essence, Gate2Brain offers a groundbreaking delivery technology that enables non-invasive, non-antigenic, permeable, stable, soluble, and receptor-specific drug transport across the blood brain barrier (BBB) and into the central nervous system (CNS), positioning us on the path to clinical trials.
To expedite this journey to patients, we are actively addressing regulatory considerations, including seeking Orphan Drug Designation (ODD) for G2B-002 and engaging with the European Medicines Agency (EMA) through Scientific Advice. Additionally, we are strategizing our approach to market access for G2B-002 to ensure its availability and affordability for those in need.