Oryzon Genomics Enters High-Stakes January with Key Pipeline Data

MADRID, Spain – January 08, 2026 – Oryzon Genomics, a Spanish biopharmaceutical firm at the forefront of epigenetic research, is poised for a pivotal month as its leadership prepares for a series of high-profile presentations across the United States and Europe. The company announced it will participate in the 9th Sachs Annual Neuroscience Innovation Forum and the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, followed by the Allinvest Securities Biomed Forum in Paris.

For the clinical-stage company, this intensive tour is more than a routine update; it represents a crucial campaign to attract the strategic partners and capital necessary to advance its promising drug candidates through late-stage development and toward commercialization. With a pipeline featuring a Phase III-ready asset for central nervous system (CNS) disorders and a compelling oncology drug in Phase II trials, the stakes for Oryzon have never been higher.

A Packed Itinerary for a Pipeline in Focus

The January conference circuit is a critical rite of passage for biotech firms, and Oryzon's schedule is strategically targeted. The month kicks off with a company presentation at the Sachs Neuroscience Innovation Forum on January 11, an event tailored to showcasing its lead CNS asset, vafidemstat, to a specialized audience of neuroscientists, investors, and potential partners.

Immediately following is the J.P. Morgan Healthcare Conference, widely considered the industry's most significant investment symposium. While Oryzon does not have a main-stage presentation slot, its management's availability for one-on-one meetings is arguably more important. These private discussions, held away from the public glare, are where foundational conversations about licensing deals, collaborations, and major investments take place. For a company with a market capitalization of approximately €250 million, securing a partnership with a large pharmaceutical player at an event like J.P. Morgan could be transformative.

The month concludes with the Allinvest Securities Biomed Forum in Paris on January 29, reinforcing Oryzon’s standing as a European leader in epigenetics and allowing it to engage directly with its home continent's investment community.

Vafidemstat: The Centerpiece of Oryzon's CNS Strategy

At the heart of Oryzon's pitch to investors is vafidemstat, a first-in-class, brain-penetrant inhibitor of the epigenetic enzyme LSD1. The drug candidate is considered "Phase III-ready" for treating Borderline Personality Disorder (BPD), a severe psychiatric condition with no currently approved therapies. This advanced status was bolstered in October 2025 when the company received supportive written feedback from the U.S. Food and Drug Administration (FDA) on its proposed Phase III trial protocol, a key de-risking milestone that will feature prominently in partnership discussions.

Confidence in the BPD program is supported by data from the recently finalized Phase IIb PORTICO trial. While full data analysis is ongoing, earlier Phase IIa results from the REIMAGINE study demonstrated that vafidemstat was safe and led to significant reductions in agitation and aggression across patients with BPD, ADHD, and Autism Spectrum Disorder. This ability to address behavioral symptoms that cut across multiple psychiatric conditions highlights a substantial unmet medical need and a large potential market.

Beyond BPD, vafidemstat is also being evaluated in an ongoing Phase IIb trial, EVOLUTION, for schizophrenia. Previous studies in Alzheimer's disease, while not meeting cognitive endpoints, showed a promising reduction in inflammatory biomarkers and ameliorated aggression in a subset of patients, suggesting a broader potential for the drug as an anti-inflammatory and behavior-modulating agent in neurology.

Beyond CNS: Oncology and Rare Disease Ambitions

While vafidemstat is the lead story, Oryzon's pipeline offers significant diversification. The company will also be showcasing iadademstat, another potent LSD1 inhibitor, for hematological cancers. The drug has received Orphan Drug Designation from the FDA for the treatment of Acute Myeloid Leukemia (AML), a notoriously difficult-to-treat blood cancer.

In its Phase IIa ALICE trial, iadademstat, in combination with the standard-of-care agent azacitidine, produced an impressive overall response rate of 85% in one evaluable cohort of elderly, unfit AML patients. Such a high response rate, coupled with durable responses and a good safety profile, makes iadademstat a highly attractive asset for potential partners in the competitive oncology space.

Further broadening its technological base, Oryzon is advancing ORY-4001, a selective HDAC-6 inhibitor, for rare neurological diseases. The candidate has shown strong preclinical efficacy in models of Charcot-Marie-Tooth disease (CMT), a debilitating hereditary neuropathy, by improving myelination and restoring nerve integrity. The program's credibility was recently enhanced by a nearly $500,000 grant from the ALS Association to support its preclinical development for Amyotrophic Lateral Sclerosis (ALS), providing valuable non-dilutive funding and third-party validation of its scientific approach.

Translating Clinical Promise into Shareholder Value

Oryzon arrives at this crucial juncture on the back of strong momentum. The company's stock has surged over 114% in the past year, and a consensus of market analysts holds a "Strong Buy" rating with average price targets suggesting a potential upside of over 200%. This optimism reflects a growing appreciation for the company's deep epigenetic pipeline and key clinical milestones.

However, like most clinical-stage biotechs, Oryzon has a history of operating losses and has relied on capital raises that have diluted shareholders. This financial reality underscores the company's core strategy: to use its expertise to develop and de-risk novel drug candidates through mid-stage clinical trials before seeking well-resourced partners for the costly final phases of development and global commercialization.

Vafidemstat is now squarely at that inflection point. The upcoming meetings in San Francisco and Paris are the proving ground for this strategy. The outcomes of these closed-door discussions will likely determine Oryzon's ability to fund its ambitious clinical plans and could very well define its growth trajectory for years to come, testing whether the market's current bullish sentiment can be converted into tangible, long-term value.