Periodic Reporting for period 1 - IVI-Boost (IntraVenous Immune Boost)
Okres sprawozdawczy: 2024-05-01 do 2025-03-31
Immunotherapies revolutionized cancer treatment and gave new hope towards cancer cure. However, they remain ineffective in a wide range of cancers due to the lack of immune cell activation and infiltration so-called “cold” tumors. There is an urgent need to identify new drugs able to increase the cure rates with immunotherapies and offer treatment options to patients with high unmet medical needs, notably metastatic patients.
Innate immune modulators have shown promise in addressing this challenge but most of them failed in clinical phases due to toxicity. Only TLR4 agonists have proven efficacy in oncology with one product commercialized, and another having demonstrated significant clinical results to potentiate the efficacy of immunotherapies. Unfortunately, current TLR4 agonists are all limited to local injection due to toxicity issues. They cannot generate a systemic stimulation of the immune system to target hard-to-reach and metastatic tumors affecting 90% of patients.
HEPHAISTOS is revolutionizing cancer immunotherapy
Developed by HEPHAISTOS, ONCO-Boost is the first TLR4 immune modulator that can be safely administered intravenously. Thanks to intravenous injection, ONCO-Boost can stimulate systemic immune responses and transform “cold” tumors into “hot” tumors that can be recognized and eliminated by the immune system. ONCO-Boost can thus address metastatic patients and hardly accessible tumors which represent 90% of the market. It opens a market 10x times larger than current competitors and has the potential to save millions of lives. HEPHAISTOS offers a breakthrough platform with broad applications, both as monotherapy and in combination with approved biotherapies.
The IVI-Boost project aims to accelerate HEPHAISTOS development plan to become a clinical stage company by 2026. The project focuses on the completion of 3 key value milestones:
1/Industrialization of the manufacturing process
2/ Regulatory validation with Clinical Trial Authorization (CTA)
3/ Validation of clinical safety and efficacy in a phase 1/2 clinical trials.
IVI-Boost project will bring ONCO-Boost to its next inflection point with a first market access (osteosarcoma monotherapy) and conduct significant deals with big pharma companies for combination therapies
Innate immune modulators have shown promise in addressing this challenge but most of them failed in clinical phases due to toxicity. Only TLR4 agonists have proven efficacy in oncology with one product commercialized, and another having demonstrated significant clinical results to potentiate the efficacy of immunotherapies. Unfortunately, current TLR4 agonists are all limited to local injection due to toxicity issues. They cannot generate a systemic stimulation of the immune system to target hard-to-reach and metastatic tumors affecting 90% of patients.
HEPHAISTOS is revolutionizing cancer immunotherapy
Developed by HEPHAISTOS, ONCO-Boost is the first TLR4 immune modulator that can be safely administered intravenously. Thanks to intravenous injection, ONCO-Boost can stimulate systemic immune responses and transform “cold” tumors into “hot” tumors that can be recognized and eliminated by the immune system. ONCO-Boost can thus address metastatic patients and hardly accessible tumors which represent 90% of the market. It opens a market 10x times larger than current competitors and has the potential to save millions of lives. HEPHAISTOS offers a breakthrough platform with broad applications, both as monotherapy and in combination with approved biotherapies.
The IVI-Boost project aims to accelerate HEPHAISTOS development plan to become a clinical stage company by 2026. The project focuses on the completion of 3 key value milestones:
1/Industrialization of the manufacturing process
2/ Regulatory validation with Clinical Trial Authorization (CTA)
3/ Validation of clinical safety and efficacy in a phase 1/2 clinical trials.
IVI-Boost project will bring ONCO-Boost to its next inflection point with a first market access (osteosarcoma monotherapy) and conduct significant deals with big pharma companies for combination therapies
HEPHAISTOS technology has reached TRL6 and the preclinical regulatory phases through validation of the feasibility of its industrialization and the best safety and efficacy (in monotherapy and in combination) of ONCO-Boost in simulated environments.
The first period of the IVI-Boost project was marked by major achievements in the industrialization of our manufacturing process and the preparation of regulatory validation and clinical trials
Manufacturing process industrialization
- Creation of a Belgian subsidiary dedicated to industrialisation with the recruitment of 2 experts
- Adaptation and validation of our manufacturing process to industrial conditions (GMP)
- Technical transfer - Update technical documentation and selection of lead and back-up CDMOs
- Transfer of production documentation to CDMO (December 2024) and start of production runs (Transfer Runs - February 2025)
- Analytical Transfer - Validation Quality Controls (QC) and specifications, and work on Potency assay optimization and development of additional QC analysis method (HPLC-CAD)
Regulatory Validation
- Validation of our non-clinical regulatory toxicity studies protocol with external experts and the French Medicines Agency (ANSM)
- Selection of accredited subcontractors for the conduct of regulatory toxicity studies
- Development of 2 dosage methods for PK studies to minimize the risk of failure
Clinical trials preparation
- Development of a detailed plan for clinical studies with external experts and clinicians, including inclusion and exclusion criteria, immunomonitoring, PK/PD, biological and statistical analyses.
- Identification of potential markers of efficacy that will be assessed in the first clinical trials
The first period of the IVI-Boost project was marked by major achievements in the industrialization of our manufacturing process and the preparation of regulatory validation and clinical trials
Manufacturing process industrialization
- Creation of a Belgian subsidiary dedicated to industrialisation with the recruitment of 2 experts
- Adaptation and validation of our manufacturing process to industrial conditions (GMP)
- Technical transfer - Update technical documentation and selection of lead and back-up CDMOs
- Transfer of production documentation to CDMO (December 2024) and start of production runs (Transfer Runs - February 2025)
- Analytical Transfer - Validation Quality Controls (QC) and specifications, and work on Potency assay optimization and development of additional QC analysis method (HPLC-CAD)
Regulatory Validation
- Validation of our non-clinical regulatory toxicity studies protocol with external experts and the French Medicines Agency (ANSM)
- Selection of accredited subcontractors for the conduct of regulatory toxicity studies
- Development of 2 dosage methods for PK studies to minimize the risk of failure
Clinical trials preparation
- Development of a detailed plan for clinical studies with external experts and clinicians, including inclusion and exclusion criteria, immunomonitoring, PK/PD, biological and statistical analyses.
- Identification of potential markers of efficacy that will be assessed in the first clinical trials
In December 2023, HEPHAISTOS completed a seed round of 4.5M€ with a European syndicate of VCs to finance its development so far. To support clinical development, HEPHAISTOS has launched a series A round of 20M€, with the support of historical VCs for 4M€ and a 5M€ ticket from the EIC fund. The objective is to close the round by the end of 2025.
Since the kickoff of the IVI-Boost project, HEPHAISTOS has been working on promoting its technology and raising awareness in the scientific, medical, industrial, investment and civil societies.
- 17 events have been covered since the beginning of IVI-Boost, with participation in round tables, pitch sessions and partnerings
- 2 scientific publications were made
- 3 communications were done - 2 interviews in newspapers + 1 press release
- 3 awards were received - Golden ticket from Amgen, Biotech winner at Hello tomorrow Global Summit, winner of Hack4Hope pediatric cancer hackathon
HEPHAISTOS also developed its network of partners with academics and industries:
- 2 Golden tickets with Amgen and Sanofi
- 2 established collaborations with pharmas
- 2 discussions for collaborations with academic units
- 4 established collaborations with European academic units
HEPHAISTOS is actively strengthening is IPR.
- Preparation of a new patent to protect our innovative manufacturing process on large-scale based on the work done in the frame of the IVI-Boost project (filing planned for Q3 2025)
- Geographic extension of our Composition of Matter and Formulation patents
Since the kickoff of the IVI-Boost project, HEPHAISTOS has been working on promoting its technology and raising awareness in the scientific, medical, industrial, investment and civil societies.
- 17 events have been covered since the beginning of IVI-Boost, with participation in round tables, pitch sessions and partnerings
- 2 scientific publications were made
- 3 communications were done - 2 interviews in newspapers + 1 press release
- 3 awards were received - Golden ticket from Amgen, Biotech winner at Hello tomorrow Global Summit, winner of Hack4Hope pediatric cancer hackathon
HEPHAISTOS also developed its network of partners with academics and industries:
- 2 Golden tickets with Amgen and Sanofi
- 2 established collaborations with pharmas
- 2 discussions for collaborations with academic units
- 4 established collaborations with European academic units
HEPHAISTOS is actively strengthening is IPR.
- Preparation of a new patent to protect our innovative manufacturing process on large-scale based on the work done in the frame of the IVI-Boost project (filing planned for Q3 2025)
- Geographic extension of our Composition of Matter and Formulation patents