ORYZON reports results and corporate update for half-year ending June 30, 2022

Oryzon invests $8.1 million in R&D in the first half 2022

Madrid (España) y Boston (EEUU)

Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, today reported financial results for the first half of 2022 and provided an update on recent developments.

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “We continued to make strong progress on our clinical pipeline this quarter, with very positive data from iadademstat’s Phase II trial in acute myeloid leukemia (AML). The 42-month data we recently reported continued to confirm a very robust percentage of responses, which are rapid and durable, alongside good tolerability. We believe combination approaches with iadademstat will increase therapeutic options for AML patients both in first and second line. We expect to start recruiting patients in our new FRIDA trial with iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutant AML patients in the summer. We are also thrilled to have entered into a collaboration with the U.S. National Cancer Institute under the CRADA scheme, which will allow us to significantly expand iadademstat’s clinical development.”

“In CNS, we also made strong progress. We continue to actively recruit patients in the Phase IIb PORTICO trial with vafidemstat in Borderline Personality Disorder in the USA, Spain, Germany, Bulgaria and Serbia. Vafidemstat’s Phase IIb trial in schizophrenia, called EVOLUTION, has continued to enroll patients. Furthermore, we are finalizing the design of HOPE, the first randomized Phase I/II personalized medicine trial with an LSD1 inhibitor, in Kabuki Syndrome patients, which we expect to start in the second half of 2022. Further, we have strengthened our balance sheet and runway with the recent signature of a convertible bond program for a total amount up to €20 million over 30 months which provides funding for further development of our exciting pipeline until 1H2024.”

First Half and Recent Highlights

Iadademstat in oncology:

  • The Phase II ALICE trial, investigating iadademstat in combination with azacitidine in AML, is fully enrolled, with a total of 36 patients. Preliminary data corresponding to the 42 months of the study were presented at the EHA-2022 congress in June, showing robust signs of clinical efficacy, with ORR of 81% where 64% of the responders showed a CR/CRi, as well as a good safety profile for the combination of iadademstat and azacitidine. Responses were rapid, with 91% of patients responding by cycle 2, and durable, with 64% of the CR/CRi lasting over 6 months. Three patients remained on 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 data from ALICE at ASH-2022.
  • Oryzon is completing preparations to start FRIDA, a Phase Ib clinical trial in patients with relapsed/refractory (R/R) Acute Myeloid Leukemia (AML) harboring a FMS-like tyrosine kinase mutation (FLT3mut+). The trial received IND approval from the FDA in March. 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 evaluate the safety and tolerability of iadademstat in combination with gilteritinib in patients with FLT3mut+ R/R AML and to establish the Recommended Phase 2 Dose (RP2D) for this combination. Secondary objectives include evaluation of the treatment efficacy, measured as the rate of complete remission and complete remission with partial hematological recovery (CR/CRh), the Duration of Responses (DoR) and the assessment of Measurable Residual Disease. The study will accrue up to approximately 45 patients and if successful, Oryzon and the FDA have agreed to hold a meeting to discuss the best plan to further develop this combination in this much in need AML population. To complement the financial support for this study, the company recently secured a non-refundable public grant from the EU of €1.87 million, having also been awarded the Seal of Excellence, a quality label awarded by the European Commission.
  • Orphan Drug Designation from the FDA was granted to iadademstat for the treatment of small cell lung cancer (SCLC) in June.
  • Preparations for new trials in combination in solid tumors have continued. In SCLC, the STELLAR trial is in preparation. STELLAR is a randomized, multicenter Phase Ib/II study of iadademstat plus a checkpoint inhibitor in first line extensive-stage SCLC. The company believes that STELLAR could potentially support an application for accelerated approval. In addition, the company is preparing a collaborative Phase Ib/II basket trial of iadademstat in combination with synergistic agents in platinum R/R SCLC and extrapulmonary high grade neuroendocrine tumors (NET) expected to start in 2H 2022. Both trials will be conducted in the US.

Vafidemstat in large multifactorial CNS indications:

  • The PORTICO Phase IIb clinical trial with vafidemstat in patients with Borderline Personality Disorder (BPD) has continued to actively enroll patients in Europe and the US. PORTICO is a multicenter, double-blind, randomized, placebo-controlled Phase IIb to evaluate the efficacy and safety of vafidemstat in BPD patients. The trial has two independent primary objectives: reduction of aggression/agitation and overall BPD improvement. The study will include 156 patients, with 78 patients in each arm, and has a pre-defined interim analysis to adjust the sample size in case of excessive variability around the endpoints or an unexpectedly high placebo rate. The trial will be conducted in 15-20 sites in Europe and US.
  • The EVOLUTION Phase IIb clinical trial with vafidemstat in patients with schizophrenia has continued to enroll patients. This Phase IIb study aims to evaluate the efficacy of vafidemstat on negative symptoms and cognitive impairment in patients with schizophrenia. This project is partially financed with public funds from the Spanish Ministry of Science and Innovation and is being carried out in various Spanish hospitals.

Vafidemstat in monogenic CNS indications:

  • We are finalizing the preparation of a new precision medicine trial in Kabuki Syndrome (KS). This Phase I/II trial, named HOPE, will be a multicenter, multi-arm, randomized, double-blind and placebo-controlled trial to explore the safety and efficacy of vafidemstat in improving several impairments described in KS patients. The trial plans to enroll 50-60 patients and will be carried out in children older than 12 years and in young adults. The company expects to start HOPE in the second half of 2022 in several hospitals and sites in the United States and, possibly, in Europe. The company is in a dialogue with the regulatory agencies to refine the final design of this trial. We also started in May a preclinical collaboration on KS with researchers from Kennedy Krieger Institute and Johns Hopkins University to evaluate the molecular effects of LSD1 inhibition with vafidemstat in samples of KS patients.
  • Our precision medicine programs in psychiatric disease continue to progress. We have collaborations in autism with researchers at the Seaver Autism Center for Research and Treatment at Icahn School of Medicine at Mount Sinai Hospital in New York and the Institute of Medical and Molecular Genetics (INGEMM) at Hospital Universitario La Paz of Madrid and in schizophrenia with researchers from Columbia University in New York. The results of the ongoing pilot studies to characterize patients with specific mutations to inform subsequent precision psychiatry clinical trials with vafidemstat are expected to conclude in 2022.

Financial Update: First Half 2022 Financial Results

Research and development (R&D) expenses were $4.2 and $8.1 million for the quarter and six months ended June 30, 2022, compared to $2.9 and $7.3 million for the quarter and six months ended June 30, 2021.

General and administrative expenses were $1.5 and $2.8 million for the quarter and six months ended June 30, 2022, compared to $1.2 and $2.5 million for the quarter and six months ended June 30, 2021.

Net losses were $1.8 and $3.4 million for the quarter and six months ended June 30, 2022, compared to $1.9 and $3.9 million for the quarter and six months ended June 30, 2021. The result is as expected, given the biotechnology business model where companies in the development phase typically have a long-term maturation period for products, and do not have recurrent income.

Negative net result was $1.3 million (-$0.03 per share) for the first half ended June 30, 2022, compared to a negative net result of $1.2 million (- $0.02 per share) for the first half ended June 30, 2021.

Cash, cash equivalents and marketable securities totaled $23.6 million as of June 30, 2022.


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