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 third quarter of 2021 and provided an update on recent developments.
Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued to make vigorous progress on our clinical pipeline this quarter. In oncology, after the positive data from iadademstat’s Phase II trial in AML published at EHA in June, ALICE is now fully recruited. Also, we have unfolded our registrational strategy in AML and in ED-SCLC with two carefully designed trials, FRIDA and STELLAR, that we believe may merit accelerated approval.”
“Our CNS pipeline has reached important milestones. After FDA’s IND approval to start vafidemstat’s Phase IIb PORTICO in BPD, we have started a vigorous recruitment in the US that will complement the one being made in Europe. We have also activated multiple clinical sites for EVOLUTION, our Phase IIb in schizophrenia and we expect to start recruitment very soon. Also, together with the most prestigious KOLs in the space, we are progressing in the design of HOPE, the first randomized Phase I/II personalized medicine trial targeting LSD1, in particular in Kabuki Syndrome patients. The extent of the planned clinical activities highlight our strong commitment for US clinical and regulatory activities. We finished this third quarter with a solid cash position of $35.83 million, which provides funding for further development of our exciting pipeline until 1Q 2023.”
Third Quarter and Recent Highlights
Iadademstat in oncology:
Vafidemstat in large multifactorial CNS indications:
Vafidemstat in monogenic CNS indications:
Financial Update: Third Quarter 2021 Financial Results
Research and development (R&D) expenses were $4.0 and $11.1 million for the quarter and 9 months ended September 30, 2021, compared to $2.3 and $9.7 million for the quarter and 9 months ended September 30, 2020.
General and administrative expenses were $1.1 and $3.5 million for the quarter and 9 months ended September 30, 2021, compared to $0.7 and $2.6 million for the quarter and 9 months ended September 30, 2020.
Net losses were $1.8 and $5.6 million for the quarter and 9 months ended September 30, 2021, compared to net losses of $1.2 and $3.8 million for the quarter and 9 months ended September 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 $3.0 million (-$0.06 per share) for the 9 months ended September 30, 2021, compared to a negative net result of $2.7 million (- $0,06 per share) for the 9 months ended September 30, 2020.
Cash, cash equivalents and marketable securities totaled $35.8 million as of September 30, 2021, compared to $52.2 million as of September 30, 2020.
During the quarter, Oryzon has obtained a grant of $1 million to support a new clinical trial (the HOPE trial) in patients with Kabuki syndrome.
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 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 and 12 months of treatment, and the pilot, small scale SATEEN trial in Relapse-Remitting and Secondary Progressive MS, where antiinflammatory activity has also been observed. Vafidemstat has also been tested 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, where it showed significant anti-inflammatory effects in severe Covid-19 patients. Currently, vafidemstat is in two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION). The company is also deploying a CNS precision medicine approach with vafidemstat in genetically-defined patient subpopulations of certain CNS disorders.
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