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 the combination approaches with iadademstat will increase therapeutic options for acute myeloid leukemia patients 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:
Vafidemstat in neurological and inflammatory disease:
Financial Update: First Half 2021 Financial Results
Research and development (R&D) expenses were $2.9 and $7.3 million for the 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.
Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European champion in Epigenetics. Oryzon has one of the strongest portfolios in the field. Oryzon’s LSD1 program has rendered two compounds, vafidemstat and iadademstat, in Phase II clinical trials. In addition, Oryzon has ongoing programs for developing inhibitors against other epigenetic targets. Oryzon has a strong technological platform for biomarker identification and performs biomarker and target validation for a variety of malignant and neurological diseases. Oryzon has offices in Spain and the United States. Oryzon is one of the most liquid biotech stocks in Europe with +90 M shares negotiated in 2020 (ORY:SM / ORY.MC / ORYZF US OTC mkt). For more information, visit
Iadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A first Phase I/IIa clinical trial with iadademstat in refractory and relapsed acute leukemia patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors, medulloblastoma and others. Iadademstat has been tested in four clinical trials (two in monotherapy in SCLC and AML, and two in combination, in SCLC and AML) in more than 100 patients. In the combination studies, ALICE (ongoing), a Phase IIa trial in combination with azacitidine in elderly or unfit AML patients, and CLEPSIDRA (finalized), a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients, preliminary efficacy results have been reported.
Vafidemstat (ORY-2001) is an oral, CNS optimized LSD1 inhibitor. The molecule acts on several levels: it reduces cognitive impairment, including memory loss and neuroinflammation, and at the same time has neuroprotective effects. In animal studies vafidemstat not only restores memory but reduces the exacerbated aggressiveness of SAMP8 mice, a model for accelerated aging and Alzheimer’s disease (AD), to normal levels and also reduces social avoidance and enhances sociability in murine models. In addition, vafidemstat exhibits fast, strong and durable efficacy in several preclinical models of multiple sclerosis (MS). Oryzon has performed two Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders (REIMAGINE) and in aggressive/agitated patients with moderate or severe AD (REIMAGINE-AD), with positive preliminary clinical results reported in both. Additional finalized Phase IIa clinical trials with vafidemstat include the ETHERAL trial in patients with Mild to Moderate AD, where a significant reduction of the inflammatory biomarker YKL40 has been observed after 6 months of treatment, and the pilot, small scale SATEEN trial in Relapse-Remitting and Secondary Progressive MS. Two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION) have been recently authorized. The company is also deploying a CNS precision medicine approach with vafidemstat in genetically-defined patient subpopulations of certain CNS disorders. Vafidemstat is also being explored in a Phase II in severe Covid-19 patients (ESCAPE) assessing the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection.
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