ORYZON reports results and corporate update for first half ended June 30, 2021

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 2021 and provided an update on recent developments.

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon 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. The 30-month data we recently reported continued to confirm a very robust percentage of responses, with responses also maturing in longer times of remission, extending survival. We believe thecombination approaches with iadademstat will increase therapeutic options for acute myeloid leukemiapatients in first line, as well as for refractory or intolerant patients who have received BCL2 inhibitors as first line. 

“Our CNS pipeline reached important milestones with Investigational New Drug approval from the U.S.Food and Drug Administration for our Phase IIb trial with vafidemstat in Borderline Personality Disorder, PORTICO and Clinical Trial Application approval of our Phase IIb trial with vafidemstat in Schizophrenia, EVOLUTION, in Europe. The initiation of PORTICO also highlights the importance of our growing U.S. clinical activities. Furthermore, vafidemstat also showed clear anti-inflammatory responses in moderate and severe CoVID-19 patients in our two-arm, randomized Phase II study, ESCAPE. We finished this second quarter with a reinforced cash position of $40.1 million, which provides funding for further development of our exciting pipeline until 1Q 2023.”

First Half and Recent Highlights 

Iadademstat in oncology:

• Phase II ALICE trial, investigating iadademstat in combination with azacitidine in acute myeloid leukemia (AML), continues recruitment. The preliminary results corresponding to the 30 months of the study, presented at the EHA 2021 congress, show robust signs of clinical efficacy, with ORR of 83%, of which 67% are CR/CRi. Five patients with remissions greater than one year were reported and the longest remission of 858 days, is still ongoing. The responses appear early. The combination of iadademstat and azacitidine continues to show a good safety profile. The company plans to present a new clinical update on ALICE at the ASH 2021 congress.
• U.S. Food and Drug Administration (FDA) Orphan Drug Designation granted to iadademstat for the treatment of AML. The drug now has orphan designation in both U.S. and EU.
• New trials in combination in AML and solid tumors are under preparation. The company believes that there is potential for fast market regulatory paths in both areas. Oryzon expects to announce further details in 2H 2021.

Vafidemstat in neurological and inflammatory disease:

• Actively recruiting patients in the Phase IIb clinical trial with vafidemstat in patients with Borderline Personality Disorder (BPD). The study, named 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 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, with three Spanish hospitals activated in the first stage.
• Following a successful pre-Investigational New Drug (IND) meeting with the FDA, Oryzon submittedan IND application for vafidemstat to perform PORTICO in the second quarter of 2021. The company has received confirmation from the FDA of IND authorization to conduct this trial.
• Received Clinical Trial Application (CTA) approval from the Spanish Agency for Medicines and Health Products (AEMPS) to carry out a Phase IIb clinical trial with vafidemstat in patients with schizophrenia. This Phase IIb study, called EVOLUTION, aims to evaluate the efficacy of vafidemstat on negative symptoms and cognitive impairment in patients with schizophrenia. Recruitment is expected to begin during the summer. This project is partially financed with public funds from the Spanish Ministry of Science and Innovation and will be carried out in various Spanish hospitals.
• The collaborations in the field of 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 the Institute of Medical and Molecular Genetics (INGEMM) at Hospital Universitario La Paz of Madridcontinue to advance. The collaboration in precision medicine in schizophrenia with researchers from Columbia University in New York has also made progress. The results of the ongoing pilot studies to characterize these patients with specific mutations to inform subsequent precision psychiatry clinical trials with vafidemstat are expected in the second half of 2021.
• Preliminary data from vafidemstat’s clinical study in seriously ill patients with CoVID-19, ESCAPE,presented at the 31st European Congress of Clinical Microbiology and Infectious Diseases, ECCMID2021. This open-label, randomized, double-arm Phase II trial was aimed to evaluate the efficacy and tolerability of vafidemstat in combination with standard treatment used in hospitals to prevent progression to acute Respiratory Distress Syndrome. The trial recruited 60 patients. Vafidemstat was safe and well tolerated in severe CoVID-19 patients. The anti-inflammatory effects of vafidemstat have been confirmed in severe CoVID-19 patients. Vafidemstat reduced the exacerbated activation of CD4 + T cells and reduced the release of key inflammatory cytokines. There were no significant differences in the number of deaths between the two arms of the study and the patients in both arms of the study recovered quickly.

Financial Update: First Half 2021 Financial Results

Research and development (R&D) expenses were $2.9 and $7.3 million forthe quarter and 6 months ended June 30, 2021, compared to $2.7 and $7.1 million for the quarter and 6 months ended June 30, 2020.General and administrative expenses were $1.20 and $2.5 million for the quarter and 6 months ended June 30, 2021, compared to $0.9 and $1.8 million for the quarter and 6 months ended June 30, 2020. 

Net losses were $1.9 and $3.9 million for the quarter and 6 months ended June 30, 2021, compared to net losses of $1.3 and $2.5 million for the quarter and 6 months ended June 30, 2020. This is due to a higher investment in research and non-capitalized development of the ESCAPE clinical trial and non-recurring expenses. The result is in accordance with the specificity of the biotechnology business model, in the development phase of the Company, with a long-term maturation period for its products, and without recurrent income.

Negative net result of $1.2 million (-$0.02 per share) for the first 6 months ended June 30, 2021, compared to a negative net result of $1.5 million (- $0,03 per share) for the first 6 months ended June 30, 2020.Cash, cash equivalents and marketable securities totaled $40.1 million as of June 30, 2021, compared to $54.9 million as of June 30, 2020

More information on Oryzon's results can be found in the attached document