Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with a strong unmet medical need, today reported financial results for the half-year ended June 30, 2023 and provided a corporate update on recent developments.
Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in 2Q23. In CNS, after obtaining positive results from the predefined interim analysis in the Phase IIb PORTICO trial with vafidemstat in BPD, we reported positive blinded aggregate safety data from 167 patients. BPD is a highly unmet medical need and an enormous commercial opportunity with limited competition and a low risk of generification. Recruitment of patients in PORTICO in the U.S. and Europe has proceeded at a very high rate, and we expect to analyze top-line data at the end of this year or early next year. Our Phase IIb trial with vafidemstat in schizophrenia, EVOLUTION, has also continued to enroll patients. We remain on track to submit the IND to initiate HOPE this year, the first randomized Phase I/II personalized medicine trial with an LSD1 inhibitor, in Kabuki Syndrome patients.”
Dr Buesa continued: “In oncology, we also continued to make progress this quarter, with both iadademstat’s ongoing clinical trials, the FRIDA trial in combination with gilteritinib in relapsed/refractory FLT3-mutant AML patients and the collaborative trial with the Fox Chase Cancer Center (FCCC) in combination with paclitaxel in neuroendocrine tumors, actively recruiting patients. In addition, and broadening our epigenetic pipeline, we reported positive preclinical efficacy data of ORY-4001, our selective HDAC-6 inhibitor recently nominated as a clinical development candidate, in Charcot-Marie-Tooth (CMT) disease. CMT is a progressive, degenerative disease involving the peripheral nerves, affecting 150,000 Americans and more than 3 million people around the world. It is one of the most prevalent conditions among rare diseases and currently lacks effective treatments or cures.”
Second Quarter and Recent Highlights
Vafidemstat in large multifactorial CNS indications:
Vafidemstat in monogenic CNS indications:
Iadademstat in oncology:
Earlier stage programs:
Financial Update: First Half 2023 Financial Results
Research and development (R&D) expenses were $4.3 and $8.6 million for the quarter and six months ended June 30, 2023, compared to $4.2 and $8.1 million for the quarter and six months ended June 30, 2022.
General and administrative expenses were $1.1 and $2.3 million for the quarter and six months ended June 30, 2023, compared to $1.5 and $2.8 million for the quarter and six months ended June 30, 2022.
Net losses were $1.3 and $2.7 million for the quarter and six months ended June 30, 2023, compared to $1.8 and $3.4 million for the quarter and six months ended June 30, 2022. 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 $0.6 million (-$0.01 per share) for the six months ended June 30, 2023, compared to a negative net result of $1.3 million (-$0.03 per share) for the six months ended June 30, 2022
Cash, cash equivalents and marketable securities totaled $14.6 million as of June 30, 2023.
About Oryzon
Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company and the European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. Oryzon’s team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston, NYC and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS and iadademstat in oncology, in several Phase II clinical trials. The company has 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, visitAbout Iadademstat
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). Iadademstatis currently being evaluated in combination with gilteritinib in the Phase Ib FRIDA trial 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.
About Vafidemstat
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 anti-inflammatory 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.
FORWARD-LOOKING STATEMENTS
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