Oryzon Genomics Secures €13.26 Million EU Grant for Rare Disease Research
核心洞察
Oryzon Genomics (搜索) has been awarded €13.26 million through the Med4Cure initiative, a pan-European project focused on developing therapies for rare diseases with no current treatment options.
The grant will fund the 44-month VANDAM (搜索) project, which aims to develop personalized epigenetic therapies for rare neurodevelopmental disorders and neuroendocrine tumors (搜索) caused by chromatin regulation gene mutations.
Combined with recent €30 million equity financing, this non-dilutive funding strengthens Oryzon's financial position as it advances its clinical pipeline, including Phase III-ready vafidemstat for borderline personality disorder (搜索).
Spanish biopharmaceutical company Oryzon Genomics (搜索) has secured a €13.26 million ($15 million) grant through the first Important Project of Common European Interest (IPCEI) in the health sector, known as the Med4Cure initiative. The funding will support the company's VANDAM (搜索) project, which focuses on developing personalized medicine approaches for rare neurodevelopmental disorders and neuroendocrine tumors (搜索).
The grant represents approximately 64% of the €20.68 million accepted budget for the 44-month program, which began in January 2023 and will continue until August 2026. This non-dilutive funding, combined with the €30 million recently raised through equity financing, significantly strengthens Oryzon's financial position as it advances its clinical pipeline.
The VANDAM Project
VANDAM (搜索), which stands for "Validation of epigenetic Agents for Neuro-related rare Diseases Applying a personalized Medicine approach," aims to develop effective therapies for serious rare diseases that currently have no treatment options. The project specifically targets rare neurodevelopmental disorders and neuroendocrine tumors (搜索) caused by mutations or loss of function in genes involved in chromatin regulation.
Dr. Jordi Xaus, Chief Scientific Officer of Oryzon, expressed gratitude for the support, stating, "We wish to express our public gratitude to the EU officials and Spain's Governmental agency CDTI for their support and valuable collaboration."
The initial focus of the project will be on aggression in subtypes of autism spectrum disorder (搜索) (ASD) and neuroendocrine tumors (搜索), aligning with Oryzon's strategic emphasis on precision medicine in central nervous system (CNS) disorders and oncology.
Med4Cure: A Pan-European Initiative
Med4Cure is a comprehensive European project comprising 14 scientific initiatives developed by 13 companies as Direct Partners and 11 as Associated Partners across six EU member states: Belgium, France, Hungary, Italy, Slovakia, and Spain. Oryzon is participating as an Associated Partner within the consortium.
In Spain, the project is coordinated by the Centro para el Desarrollo Tecnológico y la Innovación (CDTI) under the General Block Exemption Regulation framework. Spain has integrated its participation in the IPCEI Med4Cure into the national Recovery and Resilience Plan, with funding from the Spanish Ministry of Science, Innovation and Universities and CDTI, potentially supplemented by the EU's Recovery and Resilience Facility.
Following the publication of the provisional resolution, the final resolution is expected within 3-4 weeks, with the grant to be disbursed in a single installment shortly thereafter.
Oryzon's Clinical Pipeline
Founded in 2000 in Barcelona, Oryzon has established itself as a European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. The company has an advanced clinical portfolio centered around two LSD1 (搜索) inhibitors: vafidemstat for CNS applications and iadademstat for oncology.
Vafidemstat Program
Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 (搜索) inhibitor that has shown promise in reducing cognitive impairment, neuroinflammation, and providing neuroprotective effects. The compound is currently advancing as a Phase III-ready asset in Borderline Personality Disorder (搜索) (BPD) following the completion of the global, randomized, double-blind Phase IIb PORTICO trial.
After receiving feedback from an End-of-Phase II meeting with the FDA, Oryzon plans to move forward with a Phase III PORTICO-2 trial in agitation/aggression in BPD, with FDA submission planned for the first half of 2025. The company is also investigating vafidemstat in a Phase IIb trial for negative symptoms of schizophrenia (搜索) (EVOLUTION trial) and is evaluating a clinical trial in Kabuki Syndrome (搜索) patients.
Iadademstat Program
Iadademstat (ORY-1001) is a small oral molecule that acts as a highly selective inhibitor of the epigenetic enzyme LSD1 (搜索) and has shown differentiating effects in hematologic cancers. The compound has demonstrated encouraging safety and clinical activity in combination with azacitidine in elderly patients with acute myeloid leukemia (搜索) (AML) in the ALICE trial.
Currently, iadademstat is being evaluated in combination with gilteritinib in the ongoing Phase Ib FRIDA trial in patients with relapsed/refractory AML with FLT3 mutations. It is also being studied in combination with azacitidine and venetoclax in first-line AML in an investigator-initiated study led by Oregon Health & Science University (OHSU) and in a trial sponsored by the U.S. National Cancer Institute (NCI).
Beyond hematological cancers, iadademstat is being investigated in solid tumors, including small cell lung cancer (搜索) (SCLC) and neuroendocrine tumors (搜索) (NET).
Financial Implications
The €13.26 million grant significantly improves Oryzon's operating headroom, providing additional financial flexibility to advance its clinical pipeline. When combined with the recent €30 million equity financing, the company is well-positioned to continue its development programs without immediate capital concerns.
The funding is particularly valuable as Oryzon prepares for the resource-intensive Phase III trial of vafidemstat in BPD and continues to advance its other clinical programs. The non-dilutive nature of the grant means the company can access these funds without issuing additional shares, preserving value for existing shareholders.
Strategic Alignment
The VANDAM (搜索) project aligns perfectly with Oryzon's strategic focus on epigenetic approaches to CNS disorders and oncology. By targeting rare neurodevelopmental disorders and neuroendocrine tumors (搜索), the company can leverage its expertise in LSD1 (搜索) inhibition and other epigenetic mechanisms to address significant unmet medical needs.
The emphasis on personalized medicine approaches also fits with Oryzon's broader strategy of developing targeted therapies based on genetic and molecular profiles, potentially leading to more effective treatments for patients with limited or no current options.
As Oryzon continues to advance its clinical pipeline and expand its research into rare diseases through the VANDAM (搜索) project, the company is strengthening its position as a leader in epigenetic medicine, with potential applications across a range of serious conditions with high unmet needs.