Oryzon Genomics has presented the results corresponding to the third quarter of the year, a period in which the Continuous Market company has achieved a net result of -1.9 million euros compared to the third quarter of the previous year of -2.6 million euros, which represents an improvement of 26% as a result of a reduction in operating costs. Carlos Buesa’s company also points out that “the €1.9 million loss is consistent with the specificity of the biotechnology business model, in the development phase in which the Company is, with a maturation period of their products for the long term, and no recurrence from a revenue perspective.
At the end of September, Oryzon maintains a financial position with available resources amounting to 27.8 million euros, which allows it to continue with the development of its R&D activities and the development of its clinical studies.
Oryzon has intensified its R&D investments in the third quarter of 2022, reaching a figure of 12.2 million euros, which represents a variation of +27% compared to the same period of the previous year. Investments in R&D of 12.2 million euros correspond to 11.3 million euros in development activities and 0.9 million euros in research activities.
At the end of September, Oryzon presents a net worth of 71.0 million euros.
The company has continued to make progress in this third quarter in the clinical development of its iadademstat oncology and vafidemstat neurology programs.
Iadademstat in oncology
- In July, the company signed a Cooperation Research and Development Agreement (CRADA) with the United States National Cancer Institute (NCI), which is part of the National Institutes of Health. Under this agreement, Oryzon and NCI will collaborate to evaluate the safety and efficacy of iadademstat in cancer patients with different hematologic and solid tumor types.
- The Phase II ALICE trial, investigating iadademstat in combination with azacitidine in acute myeloid leukemia (AML), has completed recruitment, with a total of 36 patients. The PRESS RELEASE 2022 Pioneering Personalized Medicine in Epigenetics preliminary results corresponding to the 42 months of the study were presented at the EHA-2022 congress in June, showing robust signs of clinical efficacy, with an ORR rate of 81%, of which 64% were CR/CRi, as well as a good safety profile of the combination of iadademstat and azacitidine. Responses were rapid, with 91% of patients responding at the end of cycle 2, and durable, with 64% of CR/CRi lasting more than 6 months. Three patients remained in the study for more than 1 year, 2 patients for more than 2 years, and 1 patient for more than 3 years. The company plans to present preliminary final ALICE data at ASH-2022 in December.
- Oryzon is completing preparations to initiate FRIDA, a Phase Ib clinical trial in patients with relapsed/refractory (R/R) AML harboring an FMS-like tyrosine kinase mutation (FLT3mut+). The IND of this study has already been approved by the FDA. FRIDA is an open-label, multicenter study of iadademstat plus gilteritinib for the treatment of patients with R/R AML with FLT3 mutations. The primary objectives are to assess the safety and tolerability of iadademstat in combination with gilteritinib in patients with R/R AML with FLT3 mutations and to establish the recommended Phase 2 dose (RP2D) for this combination. Secondary objectives include assessment of treatment efficacy, measured as complete remission rate and complete remission with partial haematological recovery (CR/CRh), duration of responses (DoR), and assessment of measurable residual disease. The study will include up to approximately 45 patients, and if the results are favorable, the company and the FDA have agreed to hold a meeting to discuss the best plan for further development of this combination in this high-need AML population.
- Preparations for new combination trials in solid tumors have continued. In small cell lung cancer (SCLC), the company is preparing the STELLAR trial. STELLAR is a multicenter, randomized Phase Ib/II trial of iadademstat with an immune checkpoint inhibitor in patients with first-line metastatic SCLC. The company believes that STELLAR could potentially support an accelerated marketing approval application. In addition, the company is preparing a Phase II collaborative trial of iadademstat in combination with synergists in platinum R/R SCLC and extrapulmonary high-grade neuroendocrine tumors (NETs) expected to start in the second half of 2022. Both trials will be held in the US
Vafidemstat in major multifactorial CNS disorders
- The PORTICO Phase IIb clinical trial with vafidemstat in patients with Borderline Personality Disorder (BPD) has continued to actively recruit patients in Europe and the US. PORTICO is a multicenter, double-blind, randomized, placebo-controlled Phase IIb study. placebo to evaluate the efficacy and safety of vafidemstat in patients with BPD. The trial has two independent primary objectives: the reduction of aggressiveness/agitation and the global improvement of the disease. The study will include 156 patients, with 78 patients in each arm. Preliminary blinded, aggregated safety data from the first 43 randomized patients of PORTICO were presented in an oral communication at the 10th European Conference on Mental Health (ECMH) in September. No serious adverse events were reported. PRESS RELEASE 2022 Pioneering Personalized Medicine in Epigenetics 41 adverse reactions were reported, affecting 12 patients treated with vafidemstat or placebo, most of them mild and none reported as serious, with none leading to treatment discontinuation or withdrawal of the drug. study patient. The safety data for PORTICO are aligned with the aggregated safety data obtained in 7 completed clinical trials of vafidemstat, in which more than 300 subjects have been treated with the drug. Current data from PORTICO continue to support that vafidemstat is safe and well tolerated. A predefined interim analysis to assess signal size and futility is expected to be performed in the first quarter of 2023 using data from the first 90 patients who have completed at least 2/3 of the trial.
- The Phase IIb EVOLUTION clinical trial with vafidemstat in patients with schizophrenia has continued to recruit patients. This Phase IIb study will evaluate the efficacy of vafidemstat on negative symptoms and cognition in patients with schizophrenia. This project is partially financed with public funds from the Ministry of Science and Innovation and is carried out in various Spanish hospitals.
Vafidemstat in monogenic CNS disorders
- The company is finalizing the preparation of a new precision medicine trial in patients with Kabuki syndrome (KS). This Phase I/II trial, called HOPE, will be a multicenter, multiarm, randomized, double-blind, placebo-controlled trial to explore the safety and efficacy of vafidemstat in ameliorating several impairments described in KS patients. The trial plans to recruit 50-60 patients and will be conducted at various hospitals and centers in the US and possibly Europe. The company is in dialogue with regulatory agencies to refine the final design of this trial and expects to submit the IND for HOPE to the FDA during the second half of 2022.
- Our programs in precision medicine in psychiatric illness continue to advance. We have collaborations in autism with researchers at the Seaver Center for Autism Research and Treatment at the Icahn School of Medicine at Mount Sinai Hospital in New York and with the Institute of Medical and Molecular Genetics (INGEMM) at Hospital Universitario La Paz in Madrid, and in schizophrenia with researchers at Columbia University in New York. The results of ongoing pilot studies characterizing patients with specific mutations, which will serve to inform further precision psychiatry clinical studies with vafidemstat, are expected to be completed in 2022.