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LIFE CARE IS REGISTERED MAGAZINE IN RNI, NO.GUJGUJ/2015/71283
Copyright © 2015 - 2022 Lifecarenews.in
LIFE CARE IS REGISTERED MAGAZINE IN RNI, NO.GUJGUJ/2015/71283
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Strong clinical progress in both oncology and CNS
Cash, cash equivalents and marketable securities totaled $22.7 million as of December 31, 2022
MADRID, Spain and BOSTON, Feb. 17, 2023 (GLOBE NEWSWIRE) — 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 fourth quarter ended December 31, 2022 and provided an update on recent developments.
Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “We continued to make robust progress on our clinical development this quarter. The final data from iadademstat’s Phase II trial in acute myeloid leukemia (AML) reported at ASH confirmed not only a robust percentage of rapid and durable responses alongside good tolerability but, interestingly we also observed clinical responses in myelomonocytic AML patients, and in other patients harboring adverse prognose mutations such as p53+ who usually respond poorly to current therapies. We believe combination approaches with iadademstat will increase therapeutic options for AML patients both in first and second line. Our new FRIDA trial with iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutant AML patients is now the company’s central strategy and, we believe, our fastest route to market. We are also extremely excited with the recent initiation of a collaborative trial with the Fox Chase Cancer Center (FCCC) in the US in neuroendocrine tumors. This collaboration with the Cancer Epigenetics Institute at Fox Chase Cancer Center, a center of excellence for research in both Neuroendocrine Cancers and epigenetics, is part of an ambitious project to explore LSD1 therapeutic potential in neuroendocrine tumors.”
Dr Buesa continued, “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 US and Europe, and expect to conclude the planned interim analysis by end of the first quarter this year. BPD is a highly unmet medical need and an enormous commercial opportunity with limited competition. Enrollment continues to progress in our second Phase IIb EVOLUTION trial in schizophrenia. Furthermore, we continue with our plan to initiate HOPE this year, the first randomized Phase I/II personalized medicine trial with an LSD1 inhibitor, in Kabuki Syndrome patients.”
Fourth Quarter and Recent Highlights
Iadademstat in oncology:
Vafidemstat in large multifactorial CNS indications:
Vafidemstat in monogenic CNS indications:
Financial Update: Fourth Quarter 2022 Financial Results
Research and development (R&D) expenses were $5.0 and $18.1 million for the quarter and twelve months ended December 31, 2022, compared to $3.9 and $14.8 million for the quarter and twelve months ended December 31, 2021.
General and administrative expenses were $1.2 and $4.8 million for the quarter and twelve months ended December 31, 2022, compared to $2.0 and $5.4 million for the quarter and twelve months ended December 31, 2021.
Net losses were $1.6 and $5.9 million for the quarter and twelve months ended December 31, 2022, compared to $2.4 and $7.9 million for the quarter and twelve months ended December 31, 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 $4.5 million (-$0.085 per share) for the twelve months ended December 31, 2022, compared to a negative net result of $5.3 million (-$0.101 per share) for the twelve months ended December 31, 2021.
Cash, cash equivalents and marketable securities totaled $22.7 million as of December 31, 2022.
|ORYZON GENOMICS, S.A.|
|BALANCE SHEET DATA (AUDITED)1|
|(Amounts in thousands US $)|
|December 31st, 2022||December 31st, 2021|
|Cash and cash equivalents||22,737||32,534|
|Total Stockholders’ equity||77,405||80,711|
|ORYZON GENOMICS, S.A.|
|STATEMENTS OF OPERATIONS (AUDITED)1|
|(US $, amounts in thousands except per share data)|
|Three Months Ended December 31st||Twelve Months Ended December 31st|
|Research and Development||5,033||3,930||18,050||14,748|
|General and administrative||1,249||1,957||4,822||5,405|
|Total operating expenses||6,282||5,887||22,872||20,153|
|Loss from Operations||-6,282||-5,887||-22,872||-20,153|
|Other income, net||4,693||3,466||17,016||12,213|
|Net Financial & Tax||-863||62||1,342||2,632|
|Loss per share allocable to common stockholders:|
|Weighted average Shares outstanding|
|1 Spanish GAAP|
|* Exchange Euro/Dollar (1.0666 for 2022 and 1.1326 in 2021)|
Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European leader in epigenetics. Oryzon has one of the strongest portfolios in the field, with two LSD1 inhibitors, iadademstat and vafidemstat, in Phase II clinical trials, and other pipeline assets directed against other epigenetic targets. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit www.oryzon.com
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 FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). In a recently completed Phase IIa trial in elder 1L-AML patients (ALICE trial), iadademstat has shown encouraging safety and efficacy data in combination with azacitidine (see Salamero et al., ASH 2022 oral presentation). The company has obtained approval from the U.S. FDA for its IND for FRIDA, a Phase Ib trial of iadademstat plus gilteritinib in patients with relapsed/refractory AML with FLT3 mutations. 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 (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). Iadademstat is being evaluated in a collaborative Phase II basket study with the Fox Chase Cancer Center in combination with paclitaxel in R/R neuroendocrine carcinomas, and the company is preparing a new trial in combination in SCLC. Oryzon has entered into a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI) to collaborate on potential further clinical development of iadademstat in different types of solid and hematological cancers. In total iadademstat has been dosed so far to more than 100 cancer patients in four clinical trials. Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU.
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 and is preparing a clinical trial in Kabuki Syndrome patients. The company is also exploring the clinical development of vafidemstat in other neurodevelopmental syndromes.
This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events or otherwise. This press release is not an offer of securities for sale in the United States or any other jurisdiction. Oryzon’s securities may not be offered or sold in the United States absent registration or an exemption from registration. Any public offering of Oryzon’s securities to be made in the United States will be made by means of a prospectus that may be obtained from Oryzon or the selling security holder, as applicable, that will contain detailed information about Oryzon and management, as well as financial statements.
|IR, US||IR & Media, Europe||Spain||Oryzon|
|Ashley R. Robinson||Sandya von der Weid||Patricia Cobo/
|LifeSci Advisors, LLC||LifeSci Advisors, LLC||Atrevia||Chief Business Officer|
|+1 617 430 7577||+41 78 680 05 38||+34 91 564 07 25
+ 34 672 447 094
|+1 917 208 8293|
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