ORYZON Reports Financial Results and Corporate Update for the 4th Quarter and Year Ended December 31, 2017

MADRID, SPAIN and CAMBRIDGE, MA--(Marketwired - February 20, 2018) - Oryzon Genomics, S.A. (ISIN Code: ES0167733015) (MAD: ORY), a public clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, has reported today financial results for the fourth quarter of 2017 and provided an update on the Company's recent developments.

The Company has regained full control of ORY-1001 in January 2018 following the termination of the license agreement with Roche. The Company is working with KOLs and regulatory experts in US and Europe to define the best clinical positioning for ORY-1001 in the onco-hematological cancers and solid tumors and is planning to start new clinical trials in leukemia and small cell lung cancer in H1 2018.

On October 31^st, 2017 Oryzon obtained the approval from the Spanish Drug Agency (AEMPS) to start a Phase IIA trial with ORY-2001 in MS patients. This study, named SATEEN (SAfety, Tolerability and Efficacy in an EPIGENETIC approach to treat Multiple Sclerosis), will be conducted in different Spanish hospitals, and is designed as a randomized, double-blind, placebo-controlled, 3-arm, 36 weeks parallel-group study to evaluate the safety and tolerability of ORY-2001 in patients with Relapsing-Remitting Multiple Sclerosis (RRMS) and Secondary Progressive Multiple Sclerosis (SPMS). This PhIIA is currently ongoing and patient recruitment has recently started.

In October 2017 the Company presented new data on preclinical efficacy of ORY-2001 in MS at MSParis2017, the joint meeting of ECTRIMS and ACTRIMS, the European and Americas Committees for Treatment and Research in Multiple Sclerosis, held in Paris, France. The company presented a poster entitled "Characterization of the efficacy of ORY-2001, a novel epigenetic drug for the treatment of multiple sclerosis, during the effector phase of the EAE model". Among other data, the Company reported that in the acute effector phase of the autoimmune attack in this animal model, side to side experiments showed a deeper and/or faster protection on the animals treated with ORY-2001 than the ones treated with fingolimod.

Additionally, the Company reported new data on ORY-2001 in preclinical models of Alzheimer's disease (AD) and other CNS indications at the SfN's 47^th annual meeting (Neuroscience 2017), the world's largest conference in Neuroscience, which was held in Washington DC in November 2017. Data presented showed that ORY-2001 reduces the exacerbated aggressiveness of SAMP8 animals, a model of sporadic AD, to normal levels, and also reduces social avoidance in rat models maintained in isolation.

Preclinical efficacy data on Sickle Cell Disease (SCD) of Oryzon's third LSD1 inhibitor, ORY-3001, were presented at the American Society of Hematology (ASH) 59th Annual Meeting and Exposition in December 2017 by Professor Donald Lavelle from the Department of Medicine, University of Illinois at Chicago, who has led the collaborative research with Oryzon. Data presented confirmed that LSD1 inhibition upregulates fetal hemoglobin gene expression which are normal and may replace partially the function of the mutated adult versions, causing a general improvement in the animals.

SCD affected approximately 150,000 people in the US in 2014, and there is no cure but palliative treatments. From 1989 through 1993, an average of 75,000 hospitalizations due to SCD occurred in the US, costing approximately $475 million.

The Company currently has two "first-in-class" epigenetic drugs in clinical trials in humans, ORY-1001 and ORY-2001, and a third one in the last stages of preclinical development, ORY-3001.

Fourth Quarter Highlights

• In October 2017 Oryzon received the approval from AEMPS to start SATEEN: a Phase IIA clinical trial with ORY-2001 in Multiple Sclerosis.
• Oryzon presented new preclinical data of ORY-2001 at the Neuroscience 2017 meeting organized by the Society for Neuroscience.
• Oryzon presented new preclinical data of ORY-2001 in MS at the International Conference on Multiple Sclerosis ECTRIMS-ACTRIMS.
• Oryzon presented preclinical efficacy data of ORY-3001 in SCD at ASH 2017.

Financial Update: 2017 Fourth Quarter Financial Results

Collaboration revenue was $0.0 and $0.2 million for the 3 and 12 months ended on December 31^st, 2017 and $0.03 and $0.8 million for the 3 and 12 months ended on December 31^st, 2016. The Company does not have recognitions of deferred revenues from upfront payments.

Research and development (R&D) expenses established themselves at $1.3 and $6.4 million for the 3 and 12 months ended on December 31^st, 2017 compared to the $1.7 and $5.5 million for the 3 and 12 months ended on December 31^st, 2016. The $0.9 million increase was driven primarily by accelerated R&D efforts in the ORY-2001 program.

General and administrative expenses were $1.2 and 4.5 million for the 3 and 12 months ended on December 31^st, 2017 and $1.0 and $5.0 million for the 3 and 12 months ended on December 31^st, 2016. This decrease is primarily due to the fact that during 2016 the Company incurred in specific non-recurring expenses related with the activities to list the Company in the Spanish stock market.

Net loss for the last 3 and 12 months ended December 31, 2017 was $1.6 and $6.2 million (-$0.18 and -$0.20 per share) respectively, compared to a net loss of $1.4 and $5.7 million for the 3 and 12 months ended December, 2016 (-$0.20 and -$0.21 per share) respectively.

Cash, cash equivalents and marketable securities amounted to $42.1 million as of December 31^st, 2017, compared to $28.7 million as of December 31^st, 2016.

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. The company has one of the strongest portfolios in the field. Oryzon's LSD1 program has resulted in + 20 patent families and has rendered two compounds in clinical trials. In addition, Oryzon has ongoing programs for developing inhibitors against other epigenetic targets. The company has a strong technological platform for biomarker identification and performs biomarker and target validation for a variety of malignant and neurodegenerative diseases. Oryzon's strategy is to develop first in class compounds against novel epigenetic targets through Phase II clinical trials, at which point it is decided on a case by-case basis to either keep the development in-house or to partner or outlicense the compound for late stage development and commercialization. The company has offices in Spain and USA. For more information, visit www.oryzon.com.

FORWARD-LOOKING STATEMENTS
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