Oryzon Genomics, a clinical-stage biopharmaceutical company, has announced significant advancements in its Central Nervous System (CNS) and oncology programs. The company is moving forward with preparations for a Phase III trial of vafidemstat in Borderline Personality Disorder (BPD) following positive feedback from the FDA. Additionally, Oryzon is expanding its efforts to evaluate iadademstat in first-line Acute Myeloid Leukemia (AML) through multiple clinical trials.
Vafidemstat Shows Promise in Borderline Personality Disorder
At the 37th European College of Neuropsychopharmacology (ECNP) annual conference, Oryzon presented final data from the Phase IIb PORTICO trial of vafidemstat in BPD. The results showed notable improvements across most study endpoints compared to the topline data released earlier. Specifically, a strong improvement was observed in State-Trait Anger Expression Inventory 2 (STAXI-2) Trait Anger, a measure of agitation and aggression, with a nominal statistical significance of p = 0.0071 at Weeks 8–12.
"This quarter marked a pivotal moment for our CNS program," said Dr. Carlos Buesa, Oryzon’s Chief Executive Officer. "We observed notable improvements across most study endpoints compared to the topline data released in January."
The company also received official minutes from its End-of-Phase II meeting with the FDA, confirming the path to a Phase III trial. The FDA indicated that agitation/aggression in BPD may be an acceptable indication and agreed to the use of the same aggression scale that showed the strongest signal in Phase II. The planned PORTICO-2 Phase III trial will enroll approximately 350 patients, randomized 1:1 to vafidemstat or control, with a total trial duration of 18 weeks.
Strengthening Intellectual Property for Vafidemstat
Oryzon has continued to strengthen its patent portfolio for vafidemstat. The European Patent Office (EPO) recently granted Oryzon’s European patent EP3661510B1, titled “Methods of treating behavior alterations,” covering the use of vafidemstat for the treatment of aggression and social withdrawal. Similar patents have been granted in South Korea, with “intention to grant” communications received in Australia and Malaysia. These patents will not expire until at least 2038.
Expanding Iadademstat Development in Oncology
Following positive results from the ALICE trial with iadademstat in first-line unfit AML patients, Oryzon is expanding its efforts to further evaluate therapeutic efficacy in this patient population. This includes trials conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) and in collaboration with Oregon Health & Science University (OHSU) as an investigator-initiated study (IIS). Both trials will assess iadademstat in combination with azacitidine and venetoclax in first-line AML.
The OHSU-led IIS trial has already dosed the first cohort. Additionally, a new IIS sponsored by the Medical College Wisconsin in combination with azacitidine in patients with myelodysplastic syndrome is being prepared.
Oryzon also presented promising initial data from its ongoing FRIDA Phase Ib trial, evaluating iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutant AML patients, at the EHA Conference. Data from the first two cohorts demonstrated that the combination was safe and showed strong antileukemic activity, with encouraging response rates and a shorter time to response compared to historical data on gilteritinib alone.
Financial Update
Research and development (R&D) expenses were $1.9 million for the quarter ended September 30, 2024, compared to $3.8 million for the same period in 2023. Cash, cash equivalents, and marketable securities totaled $8.4 million as of September 30, 2024.
