Dr
“Our CNS pipeline reached important milestones with Investigational New Drug approval from the
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 bothU.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 submitted an 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 andHealth 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 theSpanish 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 theIcahn School of Medicine atMount Sinai Hospital inNew York and theInstitute of Medical and Molecular Genetics (INGEMM) at Hospital Universitario La Paz ofMadrid continue to advance. The collaboration in precision medicine in schizophrenia with researchers fromColumbia University inNew 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 , ECCMID-2021. 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
General and administrative expenses were
Net losses were
Negative net result of
Cash, cash equivalents and marketable securities totaled
BALANCE SHEET DATA (AUDITED)1 | |||
(Amounts in thousands US $) | |||
2021 | 2020 | ||
Cash and cash equivalents | 40,083 | 54,782 | |
Marketable securities | 0 | 159 | |
Total Assets | 113,226 | 112,122 | |
Deferred revenue | 0 | 0 | |
Total Stockholders' equity | 89,047 | 86,995 |
STATEMENTS OF OPERATIONS (AUDITED)1 | ||||||
(US $, amounts in thousands except per share data) | ||||||
Three Months Ended | Six Months Ended | |||||
2021 | 2020 | 2021 | 2020 | |||
Collaboration Revenue | 0 | 0 | 0 | 0 | ||
Operating expenses: | ||||||
Research and Development | 2,928 | 2,731 | 7,264 | 7,142 | ||
General and administrative | 1,200 | 906 | 2,520 | 1,770 | ||
Total operating expenses | 4,128 | 3,636 | 9,784 | 8,912 | ||
Loss from Operations | -4,128 | -3,636 | -9,784 | -8,912 | ||
Other income, net | 2,256 | 2,312 | 5,840 | 6,414 | ||
Net Loss | -1,872 | -1,324 | -3,944 | -2,498 | ||
Net Financial & Tax | 2,823 | 1,102 | 2,733 | 984 | ||
Net Result | 951 | -222 | -1,211 | -1,515 | ||
Loss per share allocable to common stockholders: | ||||||
Basic | 0.02 | -0.00 | -0.02 | -0.03 | ||
Diluted | 0.02 | -0.00 | -0.02 | -0.03 | ||
Weighted average Shares outstanding | ||||||
Basic | 52,761,554 | 45,808,246 | 52,761,554 | 45,648,400 | ||
Diluted | 52,761,554 | 45,808,246 | 52,761,554 | 45,648,400 | ||
1 Spanish GAAP | ||||||
* Exchange Euro/Dollar (1.1884 for 2021 and 1.1198 in 2020) |
About Oryzon
Founded in 2000 in
About 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 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.
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 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.
FORWARD-LOOKING STATEMENTS
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
IR, US | IR & Media, | Oryzon | |
/ Carlos C. Ungría | BD Director | ||
+1 617 430 7577 | +41 78 680 05 38 | +34 91 564 07 25 | +34 93 515 13 13 |
arr@lifesciadvisors.com | svonderweid@lifesciadvisors.com | pcobo@atrevia.com cungria@atrevia.com | etorrell@oryzon.com |
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