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]

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 BIO-EUROPE from November 3-5 in Vienna, Austria, and the LSX Investival Showcase Europe 2025 on November 17 in London, UK [1][2]. - One-on-one meetings will be available during these conferences [2]. Company Overview - Founded in 2000 in Barcelona, Spain, Oryzon specializes in epigenetics and has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready for CNS) and iadademstat (Phase II for oncology) [3]. - The company is also developing ORY-4001, targeting HDAC-6 for potential applications in CMT and ALS [3]. - Oryzon has a robust platform for biomarker identification and target validation across various malignant and neurological diseases [3].

Core Insights - Oryzon Genomics has announced a strategic collaboration with Dr. Iman Barilero as Senior Advisor for Regulatory Affairs to advance its Phase III clinical program for vafidemstat in Borderline Personality Disorder (BPD) [1][4] Company Overview - Oryzon Genomics is a clinical-stage biopharmaceutical company founded in 2000 in Barcelona, Spain, and is a leader in epigenetics, focusing on personalized medicine for CNS disorders and oncology [5] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [5] Regulatory Expertise - Dr. Iman Barilero brings over 30 years of global regulatory leadership experience, particularly in CNS, neuropsychiatry, and rare diseases, with a successful track record in guiding Phase II-III programs and securing global approvals [2][3] - Her previous role as Vice President at Lundbeck involved leading global regulatory strategies for breakthrough therapies targeting various CNS disorders [3] Clinical Development - Vafidemstat is currently being evaluated in a double-blind, randomized, placebo-controlled Phase IIb trial (EVOLUTION) for its potential to improve negative symptoms in schizophrenia [4][8] - The company is also preparing a clinical trial for vafidemstat in Autism Spectrum Disorder (ASD) and has conducted several Phase IIa trials with positive results in psychiatric disorders [8] Strategic Goals - The collaboration with Dr. Barilero aims to enhance regulatory dialogue with the FDA and provide strategic guidance across clinical development programs [7] - Oryzon's pipeline includes programs targeting schizophrenia and ASD, with a focus on advancing its late-stage pipeline in BPD and schizophrenia [4][8]

Core Insights - Oryzon Genomics has received feedback from the FDA regarding its Phase III protocol for vafidemstat in borderline personality disorder (BPD), which will be revised and resubmitted [2][3] - The company views the FDA's feedback as an opportunity to enhance the trial design and improve the chances of bringing a first-in-class therapy to patients with BPD [3] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics, particularly in CNS disorders and oncology [5] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [5] - Oryzon is also developing other pipeline assets targeting various epigenetic mechanisms, including ORY-4001 for CMT and ALS [5] Product Details - Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 inhibitor that has shown potential in reducing cognitive impairment and neuroinflammation [6] - Preclinical studies indicate vafidemstat can restore memory and reduce aggressive behavior in models of Alzheimer's disease [6] - The drug has demonstrated efficacy in several Phase IIa trials for various psychiatric disorders and is currently being evaluated in a Phase IIb trial for schizophrenia [6][4]

Core Insights - Oryzon Genomics, a clinical-stage biopharmaceutical company, is a European leader in epigenetics, focusing on personalized medicine for CNS disorders and oncology [3] Company Events - Oryzon management will participate in several upcoming events, including the Drug Discovery Innovation Programme on September 25-26 in Barcelona, and the Paris Midcap Event from September 30 to October 1 [1][2] - Additional events include the BME Investor Access Event on October 7 in Paris, BIOSPAIN 2025 from October 7-9 in Barcelona, and the 25th Sachs Annual Biotech in Europe Forum on October 8-9 in Basel, where Oryzon will present on October 8 at 13:45 CET [2] Company Overview - Founded in 2000 in Barcelona, Oryzon has a strong clinical portfolio featuring two LSD1 inhibitors: vafidemstat, which is Phase III-ready for CNS, and iadademstat, currently in Phase II for oncology [3] - The company is also developing ORY-4001, a clinical candidate targeting HDAC-6 for potential applications in CMT and ALS, alongside a robust platform for biomarker identification and target validation for various diseases [3]

Core Insights - Oryzon Genomics is sponsoring the first Phelan-McDermid syndrome (PMS) burden of illness study, led by CureShank, to assess the economic impact of PMS and guide therapy development [1][2][3] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics, particularly in CNS disorders and oncology [6] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [6] Study Details - The PMS study aims to collect data from families, health insurance claims, and clinical experts to quantify the economic burden of PMS and inform future market access strategies [2][8] - The study will help identify key drivers of the burden, which can guide new interventions and accelerate treatment development [3] Disease Context - PMS is a neurodevelopmental disorder caused by mutations in the SHANK3 gene, leading to developmental delays, intellectual disabilities, and behavioral issues [4] - There are currently no approved pharmacological treatments for PMS, highlighting the need for effective therapies [4] Clinical Trials - Oryzon is preparing a Phase II trial named HOPE-2 to evaluate the safety and efficacy of vafidemstat in PMS, expected to start soon [5] - Vafidemstat has shown promise in preclinical models for reversing social-behavior and aggression phenotypes associated with ASD [5][7]

Core Insights - Oryzon Genomics is expanding its patent portfolio for its clinical-stage LSD1 inhibitors, iadademstat and vafidemstat, with recent patent grants from the Australian and European patent offices [1][4]. Patent Developments - The Australian Patent Office granted a patent for iadademstat, covering its use in combination with PD1 or PD-L1 inhibitors for cancer treatment, including small cell lung cancer (SCLC), valid until at least 2040 [2]. - A corresponding patent for iadademstat has also been granted in Russia, with pending applications in Europe, the U.S., Japan, China, and other countries [2]. - The European Patent Office granted a patent for vafidemstat, which covers its use in treating aggressiveness and social withdrawal associated with CNS diseases, valid until at least 2038 [4]. - Additional patents for vafidemstat have been granted in various countries, with pending applications in others [4]. Clinical Trials and Research - Iadademstat is being evaluated in a Phase I/II trial in combination with PD-L1 inhibitors for extensive disease SCLC, sponsored by the U.S. National Cancer Institute [3]. - Vafidemstat is in advanced clinical development for treating aggression in psychiatric disorders, with a Phase III trial for Borderline Personality Disorder (BPD) and ongoing Phase II trials for Autism Spectrum Disorder (ASD) and schizophrenia [5][10]. - Iadademstat has shown promising results in various trials, including a Phase IIa trial in combination with azacitidine for acute myeloid leukemia (AML) [9]. Company Overview - Oryzon Genomics, founded in 2000, is a clinical-stage biopharmaceutical company based in Barcelona, Spain, focusing on epigenetics and personalized medicine in CNS disorders and oncology [7]. - The company has a robust clinical portfolio, including two LSD1 inhibitors, with ongoing research into other epigenetic targets [7].

Core Points - Oryzon Genomics has been selected as a Finalist in the 2025 European Lifestars Awards for the category Post-IPO Raise of the Year in the European Mediterranean region [1][2] - The nomination is attributed to the successful completion of a €30 million capital increase, which involved issuing 12.76 million new shares at €2.35 per share, reflecting a 15.44% discount to the 5-day volume-weighted average price and a 19.38% discount to the April 22, 2025 closing price [3] - The capital raise was significantly oversubscribed, with a U.S.-based institutional investor contributing €15 million, and additional participation from investors across the U.S., Europe, and Spain [3] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics and personalized medicine, particularly in CNS disorders and oncology [5] - The company has an advanced clinical portfolio that includes two LSD1 inhibitors, vafidemstat (Phase III-ready) and iadademstat (Phase II), along with other pipeline assets targeting various epigenetic mechanisms [5] - Oryzon has established a strong platform for biomarker identification and target validation for a range of malignant and neurological diseases [5]

Core Insights - Oryzon Genomics, a clinical-stage biopharmaceutical company, reported financial results for the first half of 2025 and highlighted significant advancements in its clinical pipeline, particularly with its LSD1 inhibitors, vafidemstat and iadademstat [1][3][9] Financial Overview - The company secured a total of $61 million in funding since December 2024, including €30 million from a capital raise in April 2025, which was oversubscribed despite challenging market conditions [2][4][13] - Research and development expenses for the first half of 2025 were $5.8 million, compared to $4.9 million in the same period of 2024 [9] - The net loss for the first half of 2025 was $3.4 million, an increase from $2.6 million in the first half of 2024, reflecting the typical financial model of biotechnology companies in development phases [10][11] Clinical Developments - The Phase III clinical trial protocol for vafidemstat in treating agitation and aggression in Borderline Personality Disorder (BPD) was submitted to the FDA, with the trial expected to enroll approximately 350 patients [3][7] - Oryzon is expanding its ongoing Phase IIb EVOLUTION study of vafidemstat in schizophrenia into additional European countries to enhance recruitment [4][7] - A new Phase II trial is being prepared to evaluate vafidemstat for aggression in Autism Spectrum Disorder (ASD), supported by a recent EU-IPCEI grant [3][4][7] Oncology Progress - Oryzon reported solid progress in ongoing iadademstat clinical trials, including the FRIDA trial in relapsed/refractory Acute Myeloid Leukemia (AML) and studies in first-line AML, Myelodysplastic Syndromes (MDS), and small cell lung cancer (SCLC) [3][4][6] - The company plans to present updates from these oncology programs at the ASH-2025 conference in December [3][4] Expansion into New Indications - Iadademstat is being evaluated for non-malignant hematological indications, with a clinical trial for sickle cell disease (SCD) in preparation, following promising preliminary data [3][4][12] - The company is also preparing a trial for iadademstat in essential thrombocythemia (ET), with submission planned for the second half of 2025 [12] Patent and Intellectual Property - Oryzon has strengthened its patent portfolio for vafidemstat, with new patents expected to cover the treatment of aggression and social withdrawal, set to last until at least 2038 [7][9]

Core Insights - Oryzon Genomics is hosting a virtual KOL event to discuss the unmet medical needs in Borderline Personality Disorder (BPD) and the design of vafidemstat's Phase III PORTICO-2 trial [1] - The event will address the lack of approved pharmacological treatments for BPD and the limitations of off-label medications, emphasizing the importance of addressing agitation and aggression in BPD and other psychiatric conditions [1] - Vafidemstat is highlighted as a promising treatment option due to its novel epigenetic mechanism of action, with ongoing discussions about its potential applications in BPD, schizophrenia, and autism spectrum disorder (ASD) [1] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics and personalized medicine for CNS disorders and oncology [9] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [9] - Oryzon is also focused on biomarker identification and target validation for various malignant and neurological diseases [9] Vafidemstat Details - Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 inhibitor that has shown efficacy in reducing cognitive impairment and neuroinflammation, as well as having neuroprotective effects [10] - The drug has demonstrated positive results in multiple Phase IIa clinical trials, including studies on aggression in psychiatric disorders and Alzheimer's disease [10] - Vafidemstat is advancing towards a Phase III trial in BPD following the completion of the Phase IIb PORTICO trial, with plans to investigate its effects on agitation and aggression [10]