Core Insights - Oryzon Genomics has received a Notice of Allowance from the USPTO for its patent application related to vafidemstat, an LSD1 inhibitor aimed at treating non-aggressive symptoms of Borderline Personality Disorder (BPD) [1][3] - The U.S. patent is expected to be valid until at least 2040, with corresponding patents granted or allowed in multiple countries, enhancing Oryzon's intellectual property portfolio [2][4] Company Overview - Oryzon Genomics, founded in 2000 and headquartered in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics and personalized medicine for CNS disorders and oncology [5] - The company has a clinical portfolio centered around two LSD1 inhibitors: iadademstat for oncology and vafidemstat for CNS disorders, with vafidemstat being Phase III-ready for BPD [5][6] Product Development - Vafidemstat (ORY-2001) is an oral LSD1 inhibitor that shows promise in reducing cognitive impairment and neuroinflammation, with positive results in various preclinical and clinical trials for psychiatric disorders and Alzheimer's disease [6][7] - The drug is also being investigated in a Phase IIb trial for negative symptoms of schizophrenia and is part of a precision medicine approach targeting genetically defined patient subpopulations [7]

Financial Overview - Oryzon Genomics reported a strong cash position of $33.3 million (€28.4 million) at the end of 2025, following a financial turnaround with over $60 million secured in the first half of 2025 [4][14]. - Research and development (R&D) expenses for Q4 2025 were $5.2 million, totaling $14.8 million for the year, compared to $2.1 million and $8.7 million in the same periods of 2024 [12]. - General and administrative expenses were $1.7 million for Q4 2025 and $5.6 million for the full year, up from $0.9 million and $3.7 million in 2024 [12]. - The net loss for Q4 2025 was $2.1 million, with a total net loss of $6.7 million for the year, compared to losses of $1.1 million and $4.6 million in 2024 [13][14]. Oncology Developments - Iadademstat, Oryzon's lead oncology program, is currently involved in seven ongoing trials, with six sponsored by the National Cancer Institute (NCI) or leading U.S. institutions [4][6]. - The drug has shown a 100% overall response rate (ORR) in first-line acute myeloid leukemia (AML) trials, with no dose-limiting toxicities reported [4][8]. - Preliminary data from the ALICE-2 Phase Ib trial indicated a 100% ORR and 90% strict complete remission (CR) in newly diagnosed AML patients [8]. - A new Phase Ib trial of iadademstat in combination with immune checkpoint inhibitors and radiotherapy for extensive-stage small cell lung cancer (ES-SCLC) has commenced [8]. CNS Program Updates - Oryzon has appointed Dr. Rolando Gutierrez as Chief Medical Officer for CNS programs, enhancing its medical and regulatory capabilities [4][7]. - The company is preparing for a protocol resubmission to the FDA for the Phase III PORTICO-2 trial in aggression in Borderline Personality Disorder (BPD) [13]. - Oryzon is expanding its Phase IIb schizophrenia trial into additional EU countries and finalizing preparations for a new Phase II trial in aggression in autism spectrum disorder (ASD) [7][13]. Intellectual Property and Future Trials - Oryzon has strengthened its intellectual property protection for iadademstat, with a patent application granted in Japan, expected to remain in force until at least 2040 [9]. - The company plans to initiate a clinical trial for iadademstat in essential thrombocythemia (ET) following EMA approval [8]. - Oryzon's earlier stage program, ORY-4001, a selective HDAC6 inhibitor, is progressing through IND enabling studies for neurological diseases [11].

Core Viewpoint - Oryzon Genomics has received authorization from the European Medicines Agency (EMA) to initiate a Phase II clinical trial for iadademstat, a selective LSD1 inhibitor, aimed at treating essential thrombocythemia (ET) [1][2]. Group 1: Clinical Trial Details - The Phase II study, named IDEAL, will be a multicenter, single-arm trial conducted in Spain, targeting adult ET patients who are resistant or intolerant to hydroxyurea [2]. - Primary objectives include evaluating the safety and tolerability of iadademstat and assessing its efficacy in reducing abnormal platelet counts in ET patients [2]. - The treatment duration for iadademstat will be up to 24 weeks, with an optional 24-week extension for patients benefiting from the treatment [3]. Group 2: Disease Background - Essential thrombocythemia is the most common type of myeloproliferative neoplasm, affecting approximately 200,000 people in the United States, and is associated with serious complications such as stroke and heart attack [4]. - Current treatments primarily focus on reducing platelet counts and controlling symptoms, but many patients develop resistance or intolerance to first-line therapies like hydroxyurea, indicating a need for new treatment options [4]. Group 3: Mechanism and Efficacy - LSD1 inhibition has been shown to prevent the differentiation of megakaryocytes into platelets, leading to a reduction in circulating platelet counts, supporting the use of LSD1 inhibitors in treating ET [5]. - Positive results from another LSD1 inhibitor in a Phase II trial for high-risk ET patients further validate this therapeutic approach [5]. Group 4: Company Insights - Dr. Carlos Buesa, CEO of Oryzon, emphasized that the IDEAL study aligns with the company's strategy to expand the clinical applications of iadademstat beyond acute leukemia to other hematologic conditions with significant unmet medical needs [6]. - Iadademstat is noted to be the most potent LSD1 inhibitor in clinical development, with over 100-fold greater potency than other inhibitors [6]. - The company is also exploring iadademstat in various oncology trials, including a Phase Ib study in combination with venetoclax and azacitidine for first-line acute myeloid leukemia (AML), which has shown a 100% overall response rate [6][8].

Core Insights - Oryzon Genomics, a clinical-stage biopharmaceutical company, is a leader in epigenetics and focuses on personalized medicine for CNS disorders and oncology [3] Upcoming Events - Oryzon management will participate in several upcoming events, including: - Bio-Neuroscience 2026 from February 24-27 in Amsterdam - SmallCap Event 20 Edition on March 17 in Paris - BIO-Europe Spring 2026 from March 23-25 in Lisbon - LSX World Congress Europe on March 25-26 in Lisbon, where a panel discussion is scheduled for March 25 at 14:00 GMT [1][2] Company Overview - Founded in 2000 and headquartered in Barcelona, Oryzon has a strong clinical portfolio centered around two LSD1 inhibitors: - Vafidemstat, which is Phase III-ready for CNS programs - Iadademstat, which is in ongoing Phase I and II studies for oncology/hematology, showing a 100% overall response rate in first-line acute myeloid leukemia [3] - The company is also advancing a broader epigenetics pipeline, including a clinical candidate ORY-4001 for Charcot-Marie-Tooth disease and amyotrophic lateral sclerosis [3] - Oryzon has a robust platform for biomarker identification and target validation across various malignant and neurological diseases [3]

Core Insights - Oryzon Genomics has appointed Dr. Rolando Gutierrez-Esteinou as Chief Medical Officer for CNS Programs, bringing over 20 years of experience in neuroscience and psychiatry drug development [1][2][3] - Dr. Gutierrez-Esteinou will lead the Phase III clinical development of vafidemstat, targeting agitation and aggression in borderline personality disorder, while also expanding its use in autism spectrum disorder and schizophrenia [2][4] - Oryzon is recognized as a leader in epigenetics, focusing on personalized medicine for CNS disorders and oncology, with a clinical portfolio that includes two LSD1 inhibitors [5][6] Company Overview - Oryzon Genomics, founded in 2000 and headquartered in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics [5] - The company has a strong clinical portfolio, including vafidemstat, which is Phase III-ready, and iadademstat, which is in ongoing Phase I and II studies with promising results in acute myeloid leukemia [5][6] - Oryzon is advancing a broader epigenetics pipeline, including a clinical candidate for Charcot-Marie-Tooth disease and amyotrophic lateral sclerosis [6]

Core Insights - Oryzon Genomics has initiated a Phase Ib trial for iadademstat, a selective LSD1 inhibitor, in combination with radiotherapy and an immune checkpoint inhibitor for extensive stage small cell lung cancer (ES-SCLC) patients [1][2][3] Group 1: Trial Details - The trial, titled "Iadademstat and Radiation Therapy With Atezolizumab in Extensive Stage Small-cell Lung Cancer (ES-SCLC) Patients With Persistent, Recurrent or Progressive Disease After First Line Systemic Therapy," is an open-label, non-randomized study [2] - It will evaluate the safety, tolerability, and efficacy of iadademstat combined with atezolizumab and stereotactic body radiation therapy (SBRT) [2] - The study will enroll patients who have previously received platinum-based chemotherapy with or without immune checkpoint inhibitor therapy [2] Group 2: Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company focused on epigenetics and personalized medicine in CNS disorders and oncology [4] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [4] - Oryzon is also developing other pipeline assets targeting different epigenetic mechanisms, including HDAC-6 [4] Group 3: Iadademstat Profile - Iadademstat (ORY-1001) is a small oral molecule that selectively inhibits the epigenetic enzyme LSD1, showing promising results in hematologic cancers [5] - Previous trials have demonstrated its safety and preliminary antileukemic activity, with encouraging results in combination with azacitidine [5] - The drug is currently being evaluated in various studies, including combinations with azacitidine and venetoclax in first-line acute myeloid leukemia (AML) [5][7] Group 4: Research and Development Focus - The combination of LSD1 inhibition with immunotherapy and radiotherapy is seen as a compelling strategy for treating aggressive cancers like SCLC [3] - Oryzon is expanding iadademstat's clinical development into non-oncological hematology indications, with trials in sickle cell disease and essential thrombocythemia [7] - Iadademstat has received orphan drug designation for SCLC and AML in both the US and EU [7]

Core Viewpoint - Oryzon Genomics has received a "Decision to grant" communication from the Japanese Patent Office for its patent application related to vafidemstat, an LSD1 inhibitor in clinical development for treating psychiatric disorders, specifically targeting aggression and social withdrawal [1][2][3] Patent and Intellectual Property - The allowed claims cover the use of vafidemstat for treating aggression and social withdrawal, symptoms associated with various CNS disorders [2] - The Japanese patent will remain in force until at least 2038, with potential extensions, and corresponding patents have been granted in multiple countries including Europe, Australia, and Canada [3][4] - Oryzon holds additional patents for vafidemstat targeting CNS disorders, expected to remain valid until at least 2040, enhancing the company's intellectual property position [5] Clinical Development - Oryzon is preparing a Phase III trial for vafidemstat to evaluate its efficacy in treating aggression in borderline personality disorder (BPD) and has ongoing Phase II trials in autism spectrum disorder (ASD) and schizophrenia [2][8] - Vafidemstat has shown positive results in previous Phase IIa trials for aggression in psychiatric disorders and has demonstrated anti-inflammatory effects in severe Covid-19 patients [7][8] Company Overview - Founded in 2000, Oryzon Genomics is a clinical-stage biopharmaceutical company focused on epigenetics and personalized medicine in CNS disorders and oncology, with a strong clinical portfolio including two LSD1 inhibitors [6] - The company is advancing a precision medicine approach in CNS disorders, preparing trials for genetically defined patient subpopulations [8]

Core Insights - Oryzon Genomics, a clinical-stage biopharmaceutical company, is a leader in epigenetics and focuses on personalized medicine for CNS disorders and oncology [3] Group 1: Upcoming Events - Oryzon management will participate in the 9th Sachs Annual Neuroscience Innovation Forum on January 11, 2026, in San Francisco, with a presentation scheduled at 10:35 am (PST) [1] - The company will also attend the 44th Annual J.P. Morgan Healthcare Conference from January 12-15, 2026, in San Francisco, and the Allinvest Securities Biomed Forum on January 29, 2026, in Paris, where one-on-one meetings will be available [2] Group 2: Company Overview - Founded in 2000 in Barcelona, Oryzon is recognized as the European leader in epigenetics, with a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) for CNS and iadademstat (Phase II) for oncology/hematology [3] - The company is developing other pipeline assets targeting epigenetic mechanisms, including ORY-4001 for potential use in CMT and ALS, and has a robust platform for biomarker identification and target validation across various malignant and neurological diseases [3]

Core Insights - Oryzon Genomics S.A. held an Extraordinary General Shareholders' Meeting where 33,581,589 voting rights were represented, accounting for 37.7938% of the share capital [1][6]. Resolutions Approved - All resolutions proposed by the Board of Directors received favorable votes ranging from 93% to 98% [2]. - Key resolutions included amendments to the Company's Bylaws, approval of a new Remuneration Policy for directors, and a Long-Term Incentive Plan for executives [7]. Financial and Corporate Actions - A capital increase of up to €125 million was approved, with the exclusion of preemptive subscription rights [7]. - The Company is focused on personalized medicine in CNS disorders and oncology/hematology, with a clinical portfolio that includes two LSD1 inhibitors [4].

Core Insights - Oryzon Genomics reported a significant financial turnaround, securing over $60 million in the first half of 2025, enabling a renewed focus on CNS clinical programs, particularly in Borderline Personality Disorder (BPD), schizophrenia, and Autism Spectrum Disorder (ASD) [3][4][8] - The company is advancing its clinical pipeline with promising data from its oncology asset iadademstat, showing a 100% overall response rate in a Phase I trial for acute myeloid leukemia (AML) [7][11] - Oryzon is enhancing its clinical and regulatory teams by incorporating experienced professionals to strengthen its late-stage pipeline and improve interactions with regulatory bodies [6][8] Financial Performance - For the nine months ended September 30, 2025, research and development expenses were $9.6 million, up from $7.1 million in the same period of 2024, while general and administrative expenses increased to $3.9 million from $3.1 million [12][13] - The net loss for the quarter was $1.2 million, compared to a loss of $1.1 million in the same quarter of 2024, reflecting the typical financial profile of a biotechnology company in the development phase [13][14] - Cash, cash equivalents, and marketable securities totaled $40.4 million as of September 30, 2025, a significant increase from $8.4 million in the previous year [14][16] Clinical Developments - The company is preparing to resubmit the revised Phase III protocol for vafidemstat in BPD following FDA feedback, which included guidance on study endpoints and non-clinical considerations [4][5] - Oryzon is expanding its clinical trials for vafidemstat in schizophrenia to additional EU countries and is preparing a new Phase II trial for aggression in ASD [5][8] - The iadademstat combination therapy has shown promising results in ongoing trials for various hematologic malignancies, with additional studies planned for sickle cell disease and essential thrombocythemia [11][22] Strategic Partnerships and Collaborations - Oryzon is exploring potential partnerships for its oncology asset iadademstat to enhance its value and ensure the drug reaches patients [3][8] - The company is collaborating with the National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) to further develop iadademstat in different cancer types [11][21] Patent Portfolio - Oryzon has strengthened its patent portfolio for both vafidemstat and iadademstat, with new patents granted or allowed in multiple jurisdictions, ensuring protection for their innovative therapies until at least 2038 and 2040, respectively [8][11][22]