GATE2BRAIN TOWARDS SME DESIGNATION AND PAEDIATRIC INVESTIGATIONAL PLAN

Project Information

G2B-002

Grant agreement ID: 101113568

DOI

10.3030/101113568

Project closed

EC signature date 20 July 2023

Start date 1 August 2023

End date 31 July 2024

Funded under

• European innovation ecosystems

Total cost

No data

EU contribution

€ 75 000,00

Coordinated by

GATE2BRAIN SL
Spain

Periodic Reporting for period 1 - G2B-002 (GATE2BRAIN TOWARDS SME DESIGNATION AND PAEDIATRIC INVESTIGATIONAL PLAN)

Reporting period: 2023-08-01 to 2024-07-31

Summary of the context and overall objectives of the project

Gate2Brain is a pioneering biotech company focused on developing therapeutics capable of efficiently crossing biological barriers, such as the blood-brain barrier (BBB). Utilizing a cutting-edge peptide-based patented technology platform, Gate2Brain has developed G2B-002, a novel therapy aimed at treating paediatric high-grade brain tumours, such as Diffuse Midline Glioma and paediatric glioblastoma. These conditions currently have no cure and a poor prognosis, representing a clear significant unmet medical need. Gate2Brain’s primary goal is to transform the treatment landscape for paediatric brain tumours through the therapeutic validation of its platform, demonstrating safety and efficacy. In the context of WomenTech EU, the company aimed to achieve Small and Medium-Sized Enterprise (SME) designation within the European Union (EU) to leverage various financial supports, reduced administrative compliance requirements, and other incentives crucial for accelerating research and innovation. Post-SME designation, Gate2Brain planned to develop a comprehensive Scientific Advice Briefing Documentation. This document will clearly present regulatory questions and scientific issues to be addressed by the European Medicines Agency (EMA). It will serve as the foundation for obtaining targeted feedback and guidance from the EMA, aimed at optimizing the development strategy, addressing potential regulatory hurdles, and ensuring that the preclinical and clinical development plan aligns with regulatory expectations. By evaluating these factors, Gate2Brain aims to enhance its market access potential and ensure economic viability and accessibility of G2B-002, thereby maximizing its impact on patient outcomes and broader healthcare landscape.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

Gate2Brain has successfully obtained its EMA-SME designation with the number EMA/SME/162/23. This achievement opens access to various EU support programs and funding opportunities, thereby facilitating Gate2Brain’s research and development efforts. Additionally, G2B-002, the company’s flagship product, has been validated with the Research Product Identifier (RP). This validation marks a significant milestone, ensuring the product is on track for further development and regulatory approval. A comprehensive Scientific Advice Briefing Document was prepared to address regulatory questions and scientific issues. This document will facilitate targeted feedback from the EMA, optimizing the development strategy, addressing regulatory hurdles, and aligning the development plan of G2B-002 with EMA regulatory expectations. Furthermore, Gate2Brain conducted an Early Access Program (EAP) and PICO (Population, Intervention, Comparator and Outcome) landscape analysis, along with Fit-Gap Analysis, to prioritize the activities for G2B-002’s development and lunch. The analysis led to a strategic roadmap, prioritizing several key activities: co-creation of the clinical trial design with patients, horizon scanning and competitive landscape assessment, market opportunity evaluation, development of a high-level roadmap to support clinical development, and stakeholder mapping of G2B-002.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

Gate2Brain’s innovative approach to crossing the BBB with its peptide-based technology platform represents a significant advancement in therapeutic delivery mechanisms, particularly for brain tumours. The development of G2B-002 exemplifies several results that surpass the current state of the art of paediatric high-grade brain tumours. G2B-002 leverages a unique peptide-based method to cross the BBB, enabling direct delivery of the therapeutic agent to the brain. This represents a breakthrough in treating central nervous system diseases, where drug delivery has historically been a major challenge. By targeting paediatric brain tumours, such as Diffuse Midline Glioma and paediatric Glioblastoma, G2B-002 addresses conditions with critical unmet medical needs. The innovative therapy has the potential to significantly improve survival rates and the quality of life for affected children, offering hope where there currently is none. By securing SME designation and developing a comprehensive Scientific Advice Briefing Document, Gate2Brain is well-positioned to navigate the regulatory landscape efficiently. The prioritized activities and strategic roadmap developed through extensive analysis ensure that G2B-002’s preclinical and clinical development is robust, resource-optimized, and aligned with patient and market needs. This holistic approach maximizes the likelihood of successful clinical trials and market entry. Gate2Brain’s HE&MAOS, including willingness-to-pay and product-market fit analysis, underpins the economic value proposition of G2B-002. This strategy not only reinforces the therapeutic potential of G2B-02 but also highlights its value to the healthcare system and pharmaceutical industry, paving the way for substantial societal impact.