• ETHERAL has recruited more than 100 patients to date
  • Safety data so far show that vafidemstat is safe and well tolerated
  • Parametric functional evolution of the first (blinded) 33 patients that have been treated for 6 months show changes
  • Changes also detected in some biomarkers evolution (blinded)

MADRID, Spain and CAMBRIDGE, Mass., July 16, 2019 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a public clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today that the company presented yesterday data from the ongoing Phase IIa clinical trial with vafidemstat in Alzheimer’s disease (AD) named ETHERAL (Epigenetic THERapy in ALzheimer’s Disease) at the 2019 Alzheimer's Association International Conference (AAIC-2019) being held in Los Angeles (USA) on July 14-18.

Dr. Roger Bullock, Oryzon’s Medical Director, and Dr. Michael Ropacki, Oryzon’s Vice-President of Clinical Development, are attending the Conference. Dr. Bullock presented a Poster entitled “P#31419. Safety Evaluation of Vafidemstat on Mild to Moderate Alzheimer’s Subjects” at the session “Therapeutics: Clinical” yesterday, July 15.

Positive safety data from the first 104 patients recruited in ETHERAL were presented, suggesting that the drug is safe and well tolerated in AD patients. With 87.5% (91/104) patients having completed at least one month of treatment, no clinically relevant effects on platelets, neutrophils and other hematological parameters have arisen, in keeping with the previous vafidemstat safety data from other clinical studies. In addition, 36 patients have already passed the 6-month threshold and no significant safety issues have been reported.

The initial blind analysis performed in certain functional parameters in the first 33 individual patients that had completed the first 24 weeks of treatment, showed that while some patients show disease progression, in others the baseline values are maintained or even improved, as for example in memory performance values measured by MMSE or aggressivity values measured by CMAI.

As regards biomarker analysis (also blind), CSF levels of S100A9, a well know proinflammatory biomarker highly abundant in the AD brain, follow a similar pattern after the 24 first weeks of treatment, with only six patients showing a strong increase, while the others remain stable or show a significant decrease.

Dr. Bullock has stated, ”We are happy with these first ETHERAL results, the drug is showing a very good safety profile in this aged and frail population often with comorbidities. This reinforces the safety data obtained in psychiatric younger patients and suggests that vafidemstat is well tolerated and with few adverse events. Although it is obviously premature to speculate about efficacy, as the study remains blind, the evolution of the functional parameters in these first 33 patients allow us to expect meaningful clinical readouts at the end of the study.”

For more information about the Conference please visit https://www.alz.org/aaic/overview.asp

For a complete view of the poster please visit https://www.oryzon.com/sites/default/files/20190716 ORYZON AAIC poster.pdf

The ongoing ETHERAL Phase IIa study is being conducted in 17 European hospitals in UK, France and Spain and 3 additional sites in the US. It is a randomized, double-blind, 3-arm, parallel-group study with a 24-week placebo-controlled period followed by a 24-week extension where placebo patients are randomized to vafidemstat therapy, to evaluate the safety, tolerability and preliminary efficacy of vafidemstat in patients with mild to moderate AD. Secondary endpoints include measures of cognition, function and behavior. Several traditional and novel CSF biomarkers are also measured. The European arm of the study plans to enroll up to 125 patients, with more than 100 patients already randomized. The US arm of the trial plans to enroll up to 30 patients, to complete a minimum of 150 patients on the aggregate. The company has received a grant of $1.5 million from the Alzheimer’s Drug Discovery Foundation (ADDF) to support the US-arm of ETHERAL clinical trial.

About Oryzon
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 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 neurodegenerative diseases. Oryzon has offices in Spain and the United States. For more information, visit www.oryzon.com

About Vafidemstat
Vafidemstat (ORY-2001) is an oral, brain penetrant drug that selectively inhibits LSD1 and MAOB. 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, 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). Vafidemstat is in Phase IIa clinical studies in patients with Relapse-Remitting and Secondary Progressive MS (SATEEN), in patients with Mild to Moderate Alzheimer’s disease (ETHERAL) and in aggressiveness in patients with different psychiatric or neurodegenerative disorders (REIMAGINE, a basket trial).

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 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 & Media, US & Europe:Spain:Oryzon:
LifeSci Advisors LLC ATREVIAEmili Torrell
Hans HerklotsPatricia Cobo/Idoia RevueltaBD Director
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