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]

Core Viewpoint - Oryzon Genomics has received a significant U.S. patent for iadademstat, a selective LSD1 inhibitor, which strengthens its position in the oncology market, particularly for treating acute myeloid leukemia (AML) in combination with existing therapies like venetoclax [1][5]. Patent Details - The U.S. patent US12,564,559 B2 covers therapeutic combinations of iadademstat for treating neoplastic diseases, including AML, and is set to expire in January 2039, with potential for further extensions [2][3]. - The patent includes claims for combinations with venetoclax, which is a standard treatment for first-line AML, enhancing the long-term value of Oryzon's clinical programs [5]. Clinical Development - Iadademstat is currently being evaluated in seven ongoing oncology clinical trials, including the Phase Ib ALICE-2 study, which has shown a 100% overall response rate (ORR) and a 90% strict complete remission (CR) rate in preliminary data [4][8]. - Updated data from approximately 15-16 patients in the ALICE-2 study are expected to be presented at the European Hematology Association Annual Congress in June 2026 [4]. Intellectual Property Portfolio - In addition to the U.S. patent, Oryzon has secured patent protection for iadademstat combinations in multiple countries, including Australia, Brazil, Canada, Europe, India, Israel, Japan, Korea, Malaysia, Mexico, New Zealand, and Russia, with additional applications pending [5]. - Oryzon also holds patents for iadademstat combinations with other AML therapies, such as azacitidine and decitabine, in the U.S. and other jurisdictions [5]. Company Overview - Founded in 2000 and headquartered in Barcelona, Spain, Oryzon is a clinical-stage biopharmaceutical company focused on epigenetics and personalized medicine for CNS disorders and oncology [6]. - The company has a robust clinical portfolio centered around two LSD1 inhibitors: iadademstat for oncology and vafidemstat for CNS disorders, with ongoing Phase I and II studies [6]. Mechanism of Action - Iadademstat is a small oral molecule that selectively inhibits the epigenetic enzyme LSD1, demonstrating significant potential in hematologic cancers [7]. - The drug has shown promising safety and clinical activity in combination therapies, including trials for relapsed/refractory AML and other hematological conditions [9].

Core Insights - Oryzon Genomics has received patent protection for its cancer therapy involving iadademstat in combination with PD-1 or PD-L1 inhibitors, specifically targeting small cell lung cancer (SCLC) [1][2] - The patent is expected to provide protection until at least 2040, enhancing Oryzon's intellectual property portfolio globally [2][3] - Iadademstat is currently undergoing clinical trials in combination with immune checkpoint inhibitors for extensive-stage SCLC, with promising preliminary results [3][5] Company Overview - Oryzon Genomics, founded in 2000 and headquartered in Barcelona, Spain, is a clinical-stage biopharmaceutical company focused on epigenetics and personalized medicine for oncology and CNS disorders [4] - The company has a robust clinical portfolio, including two LSD1 inhibitors: iadademstat for oncology and vafidemstat for CNS disorders, with ongoing Phase I and II studies [4][5] - Iadademstat has shown a 100% overall response rate in acute myeloid leukemia (AML) and is being evaluated in various hematological and solid tumors, including SCLC [5][6] Clinical Development - Iadademstat is being tested in two ongoing clinical trials for extensive-stage SCLC, one sponsored by the U.S. National Cancer Institute and the other by Yale University [3][6] - The drug has demonstrated safety and efficacy in combination with other therapies, including azacitidine and venetoclax for AML, and is also being explored for non-oncological indications [5][6] - Oryzon has received orphan drug designation for SCLC and AML in both the US and EU, indicating its potential significance in treating these conditions [6]

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 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 has received a patent grant from the Japanese Patent Office for its application related to combinations of iadademstat for cancer therapy, which is a significant step in enhancing its patent portfolio [1][2][3] Patent Protection - The granted patent is expected to remain effective until at least 2040, excluding any potential extensions, securing Oryzon's intellectual property in Europe, Japan, Australia, and Russia [2] - Oryzon is actively pursuing corresponding patent applications in other countries to further strengthen its global patent coverage [2] Clinical Development - Iadademstat is being evaluated in combination with immune checkpoint inhibitors, specifically atezolizumab and durvalumab, for the treatment of small cell lung cancer (SCLC) in an ongoing Phase I/II trial sponsored by the U.S. National Cancer Institute [3] - The trial is being conducted at over 30 clinical sites across the U.S., including prestigious institutions like Memorial Sloan Kettering Cancer Center and Johns Hopkins [3] Company Overview - Founded in 2000, Oryzon Genomics is a clinical-stage biopharmaceutical company based in Barcelona, Spain, focusing on epigenetics and personalized medicine in oncology and CNS disorders [4] - The company has a robust clinical portfolio, including two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [4] Iadademstat Details - Iadademstat (ORY-1001) is a selective inhibitor of the epigenetic enzyme LSD1, showing promising results in hematologic cancers and is currently in various clinical trials for conditions such as acute myeloid leukemia (AML) and small cell lung cancer [5][6] - The drug has demonstrated safety and preliminary efficacy in multiple trials, including a Phase IIa trial in combination with azacitidine for elderly AML patients [5]