ORYZON Reports Financial Results and Corporate Update for Quarter Ended September 30, 2024
-- Final data from PORTICO, global Phase IIb vafidemstat trial in BPD,
presented at the 37th ECNP annual conference in September
-- Company moving forward with PORTICO-2 Phase III trial preparations
following positive feedback from End-of-Phase II meeting with the FDA
-- Company continues to strengthen IP position for vafidemstat with
additional "intention to grant" communications
-- First cohort dosed in Investigator-initiated Phase Ib study of
iadademstat with venetoclax and azacitidine in first-line AML
-- Reduction in R&D expenses for the nine months ended September 30, 2024 as
a result of completion of the PORTICO clinical trial; savings of $5.1M
with respect to the nine months ended September 30, 2023
MADRID and CAMBRIDGE, Mass., Oct. 24, 2024 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, today reported financial results for the nine months ending September 30, 2024 and provided a corporate update on recent developments.
Dr Carlos Buesa, Oryzon's Chief Executive Officer said, "This quarter marked a pivotal moment for our CNS program. At the ECNP 2024 Conference, we presented the full dataset from our Phase IIb PORTICO trial of vafidemstat for Borderline Personality Disorder (BPD), and we observed notable improvements across most study endpoints compared to the topline data released in January. We also received the official minutes from our End-of-Phase II meeting with the FDA, confirming that we can proceed to a Phase III. The FDA indicated that agitation/aggression in BPD may be an acceptable indication, and agreed that we may use the same aggression scale that showed the strongest signal in Phase II. With this positive feedback, we will now begin preparations to submit the full Phase III protocol to the FDA. Careful analysis of the effect sizes in BPD has allowed us to reevaluate and increase the recruitment target for the EVOLUTION trial in schizophrenia, which continues to enroll patients in Spain. Meanwhile, we have strengthened our CNS intellectual property portfolio with additional "intention to grant" communications in two key patent families covering the use of vafidemstat in treating aggression, social withdrawal and BPD. These advancements significantly bolster our IP position for vafidemstat."
Dr Buesa continued, "In oncology, following the very positive results of our ALICE trial with iadademstat in first-line unfit AML patients, we are expanding our efforts to further evaluate therapeutic efficacy in this patient population through two additional trials. One is conducted under our CRADA agreement with the NCI, while the other is 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 enrolled the first cohort, representing a significant advancement in our oncology program and, if positive, could open additional options for our clinical development strategy. In addition, a new IIS sponsored by the Medical College Wisconsin in combination with azacitidine in patients with myelodysplastic syndrome is being prepared. In June, we presented promising initial data from our ongoing FRIDA Phase Ib trial, evaluating iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutant AML patients, at the EHA Conference. The 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. We have since completed enrollment of the third cohort, and as the data matures, we plan to present additional results at the EHA Conference next June."
Dr Buesa added, "The positive outcome of the End-of-Phase II meeting with the FDA has been a turning point for the company, as it transitions into a Phase III company for the first time. While we remain committed to maintaining strict budgetary discipline and leveraging access to our Convertible Notes program, we anticipate receiving further financial support from the recently approved IPCEI grant from the EU, which will provide crucial resources to advance our R&D in personalized medicine for both CNS and oncology. The company is also actively engaged in discussions with corporate partners to explore potential partnerships and will continue evaluating additional financing opportunities."
Third Quarter and Recent Highlights
Vafidemstat in large multifactorial CNS indications:
-- Final data from vafidemstat's Phase IIb PORTICO trial in BPD were
presented at the New Medications Symposium, a special symposium focused
on clinical trials of new compounds within the 37th European College of
Neuropsychopharmacology (ECNP-2024) congress held in September in Milan,
Italy. A strong improvement in State-Trait Anger Expression Inventory 2
(STAXI-2) Trait Anger, a measure of agitation and aggression, at Weeks
8--12 was observed compared to previous top line data (TLD); nominal
statistical significance now of p = 0.0071 (previously 0.0259). An
improvement in Borderline Evaluation of Severity $(BEST)$, an overall
measure of BPD disease severity, at Weeks 8--12 compared to TLD was also
achieved; nominal statistical significance now of p = 0.0260 (previously
0.0423). Vafidemstat showed favorable results over placebo in all primary
and secondary efficacy endpoints, as demonstrated by T-Forest plot
analysis. The final analysis confirmed now a global treatment effect
favoring vafidemstat by the Global Statistical Test $(GST)$, with the GST
p-value showing a statistical significance, particularly when considering
global improvement in the severity of the disease and in
agitation/aggression (p = 0.0362). Vafidemstat was, as in all previous
clinical studies, safe and well-tolerated.
-- Oryzon has received the minutes from the end-of-Phase II meeting with the
U.S. Food and Drug Administration (FDA). Based on the positive feedback
received, ORYZON has started preparations for Phase III, including the
preparation of a full protocol for the PORTICO-2 Phase III trial to
submit to the FDA for study approval. The trial will use STAXI-2 Trait
anger as a primary efficacy endpoint measure. Secondary endpoints will
include both patient-rated and clinician-rated scales, such as CGI-S A/A
to assess agitation/aggression, and BEST and CGI-S to assess overall BPD
improvement.The estimated total sample size for PORTICO-2 is 350 patients
(randomized 1:1 vafidemstat or control), with a trial duration of 18
weeks in total. Subject to FDA's review of the final data, the PORTICO-2
Phase III study has the potential to be one of the two registrational
trials required by the FDA. The company expects to obtain FDA's approval
for PORTICO-2 by end of 1Q2025.
-- Oryzon has continued to strengthen its patent portfolio for vafidemstat
during this quarter. The European Patent Office (EPO) recently granted
Oryzon's European patent EP3661510B1, titled "Methods of treating
behavior alterations"; the granted claims cover the use of vafidemstat
for the treatment of aggression and social withdrawal. A corresponding
patent has also been granted in South Korea and we have received
"intention to grant" communications in Australia and Malaysia. These
patents will not expire until at least 2038, excluding any potential
patent term extension that may provide additional years of protection. In
addition, Oryzon has received "intention to grant" communications in
Europe and Mexico for another key patent family related to vafidemstat
titled "Methods of treating borderline personality disorder", and a
corresponding patent has been granted in Japan. These patents will not
expire until at least 2040, excluding potential patent term extensions
that may provide additional protection. Another patent, covering the use
of vafidemstat to treat ADHD, has also been granted in Japan during this
period.
-- The EVOLUTION Phase IIb clinical trial evaluating vafidemstat in patients
with schizophrenia continues to enroll participants. This study aims to
assess the efficacy of vafidemstat, with a primary focus on improving
negative symptoms. Following a careful analysis of effect sizes observed
in BPD, we have reevaluated and increased the recruitment target for the
trial. In addition to negative symptoms, the trial is also exploring as
secondary endpoints vafidemstat's efficacy in improving cognitive
impairment and positive symptoms in schizophrenia. The project is
partially funded by the Spanish Ministry of Science and Innovation and is
being conducted at multiple hospitals across Spain.
Vafidemstat in monogenic CNS indications:
-- We continue to evaluate the feasibility of a new precision medicine trial
in Kabuki Syndrome. The company will decide on a possible submission of
an IND for HOPE to the FDA in 2025.
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October 24, 2024 12:08 ET (16:08 GMT)
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