Autism Spectrum Disorder

Partnership underscores Baylor Genetics’ commitment to providing genetic testing as an important step to end the diagnostic odyssey for patients and families facing neurodevelopmental disordersHOUSTON, April 28, 2025 (GLOBE NEWSWIRE) -- Baylor Genetics, a clinical diagnostic laboratory at the forefront of genetic testing, today announced their collaboration with the Child Neurology Society (CNS) as an official 2025-2026 genetic testing partner. For patients and families facing neurodevelopmental disorders (NDD), epilepsy, and other neurological concerns, comprehensive genetic testing is an important step to ending the diagnostic odyssey. Many childhood-onset disorders lead providers to select comprehensive genetic testing like whole exome sequencing (WES) and whole genome sequencing (WGS). Indications for WES or WGS testing include autism spectrum disorders, developmental delay, intellectual disability, seizures and movement disorders. Baylor Genetics’ flagship rapid WGS and WES tests are among the fastest and most accurate precision diagnostic tools available, with written reports starting at five days from receipt of patient samples and high diagnostic yield of up to 40-60 percent. “Partnering with CNS underscores our commitment to helping children and families who are desperately seeking insights and answers to end their diagnostic odyssey,” said Christine Eng, M.D., Chief Medical Officer and Chief Quality Officer at Baylor Genetics. “As an official genetic testing partner for CNS, we are able to provide our comprehensive WES and WGS tests, Neurodevelopmental Disorders Panel, and other diagnostic tests to an extensive network of clinicians, patients and families, helping to deliver more answers and improve outcomes.” Baylor Genetics’ Neurodevelopmental Disorders Panel is designed for patients affected by a suspected or clinically diagnosed neurodevelopmental disorder, autism spectrum disorder, intellectual disability, and/or developmental delay. The panel analyzes 236 genes associated with these conditions, enabling robust detection of single nucleotide variants, insertions or deletions, and copy number variants at exceptional read depths. “We are delighted to welcome Baylor Genetics as a partner. Their expertise in WES, WGS, and neurodevelopmental diagnostics will be essential in delivering critical insights to patients and families who need them most,” said Monique Terrell, Executive Director and CEO of the Child Neurology Society. “As a CNS partner, we look forward to Baylor Genetics’ meaningful contributions at our Annual Meeting and other events as we collaborate to advance the care of children with neurological and developmental disorders through education, advocacy, and support for clinicians and researchers in child neurology.” Baylor Genetics’ partnership with the Child Neurology Society is part of a broader commitment to improving access to genetic testing across the U.S. The company is the core genetic sequencing partner for the National Institutes of Health’s Undiagnosed Disease Network, with more than 2,740 patient participants evaluated using WGS, WES, and other sequencing tests. Baylor Genetics also partners with the Texome Project and Project GIVE to increase access and reduce socioeconomic disparities in genetic testing. About Baylor Genetics As a pioneer of precision medicine for more than 45 years, Baylor Genetics is a leading diagnostic genomics partner offering a full spectrum of clinically relevant genetic testing, including Whole Genome Sequencing, Whole Exome Sequencing, and focused panels. Through a joint venture of H.U. Group Holdings, Inc. and Baylor College of Medicine, Baylor Genetics combines rapid and comprehensive precision diagnostics options with the support of genetic counselors to help clinicians avoid a lengthy diagnostic odyssey for their patients, guide medical management, and ensure no patient with a genetic disorder gets left behind. Baylor Genetics’ testing menu covers family planning, pregnancy, neonatal and pediatric testing, oncology, and many other specialized testing options. Located in Houston’s Texas Medical Center, Baylor Genetics serves clients in 50 states and 16 countries. Learn more about what’s next. PR Contact SVM PR baylorgenetics@svmpr.com (401) 490-9700

Issued 12,765,958 new sharesPriced at €2.35 per share, representing a 15.44% discount on the 5-day VWAPStrong investor demand, with the offering significantly oversubscribed€15 million anchored by a leading US institutional investorProceeds to fund clinical development and corporate initiatives MADRID and CAMBRIDGE, Mass., April 24, 2025 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a European leader in epigenetics, announced today the successful completion of a €30 million capital increase through the issuance of 12,765,958 new shares, priced at €2.35 per share. This pricing reflects a 15.44% discount to the 5-day volume-weighted average price (VWAP) of €2.779 per share prior to April 22, 2025, and a 19.38% discount to the April 22 closing price of €2.915. The offering, which was structured as straight equity with no warrants attached, attracted strong demand and was significantly oversubscribed. A US-based institutional investor anchored the round with a €15 million order, with the remaining demand filled by investors across the US, Europe, and Spain. The capital increase was capped at €30 million by the company’s Board of Directors. Maxim (U.S.), All-Invest (EU/UK), and Singular Bank (Spain) acted as bookrunners. Banco Sabadell, S.A. acted as the Settlement and Technical Pre-financing Agent for the Capital Increase, while Gómez-Acebo & Pombo Abogados, S.L.P. (Spanish law) and Gibson, Dunn & Crutcher LLP (U.S. law) served as legal advisors to the Company, and Loeb & Loeb LLP acted as U.S. legal counsel to Maxim Group LLC. The funds raised will be used to: Advance the clinical development of vafidemstat for the treatment of aggression in CNS disorders such as Borderline Personality Disorder (BPD) and Autism Spectrum Disorder (ASD)Expand ongoing clinical programs in oncology and hematologySupport CMC activities and preparations for Phase IIb-III trials, as well as other regulatory initiativesStrengthen the company's balance sheet to support future partnership or M&A negotiationsCover general administrative expenses and financial obligationsEnhance legal and audit readiness for a potential dual listing on Nasdaq Dr. Carlos Buesa, CEO of Oryzon, commented: “This financing marks a key milestone that enables us to continue progressing our clinical programs with vafidemstat in aggression-related CNS disorders and to further develop our oncology and hematology pipeline. It also reinforces our financial position, supporting strategic conversations with investors and potential partners.” About OryzonFounded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company and the European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. Oryzon’s team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston, and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS (Phase III-ready) and iadademstat in oncology (Phase II). The company has other pipeline assets directed against other epigenetic targets like HDAC-6 where a clinical candidate ORY-4001, has been nominated for its possible development in CMT and ALS. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit www.oryzon.com About IadademstatIadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L AML patients (ALICE trial) (see Salamero et al., ASH 2022 oral presentation & The Lancet Haematology, 2024, 11(7):e487-e498). Iadademstat is currently being evaluated in combination with gilteritinib in the ongoing Phase Ib FRIDA trial in patients with relapsed/refractory AML with FLT3 mutations, and in combination with azacitidine and venetoclax in 1L AML in an investigator-initiated study led by OHSU and in a trial sponsored by the U.S. National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) signed between Oryzon and the NCI to collaborate on further clinical development of iadademstat in different types of hematologic and solid cancers. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). Iadademstat is in a collaborative Phase II trial with the Fox Chase Cancer Center (FCCC) in combination with paclitaxel in R/R neuroendocrine carcinomas, and in a Phase I/II randomized trial in 1L ED-SCLC in combination with ICI sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center. Oryzon is further expanding the clinical development of iadademstat through additional investigator-initiated studies. Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU. About Vafidemstat Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 inhibitor. The molecule acts on several levels: it reduces cognitive impairment, including memory loss and neuroinflammation, and at the same time has neuroprotective effects. In animal studies vafidemstat not only restores memory but reduces the exacerbated aggressiveness of SAMP8 mice, a model for accelerated aging and Alzheimer’s disease (AD), to normal levels and also reduces social avoidance and enhances sociability in murine models. In addition, vafidemstat exhibits fast, strong, and durable efficacy in several preclinical models of multiple sclerosis (MS). Oryzon has performed two Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders (REIMAGINE, see Ferrer et al, Psychiatry & Clin Neurosci, 2025, doi.org/10.1111/pcn.13800) and in aggressive/agitated patients with moderate or severe AD (REIMAGINE-AD), with positive clinical results reported in both. Additional finalized Phase IIa clinical trials with vafidemstat include the ETHERAL trial in patients with Mild to Moderate AD, where a significant reduction of the inflammatory biomarker YKL40 was observed after 6 and 12 months of treatment, and the pilot, small-scale SATEEN trial in Relapse-Remitting and Secondary Progressive MS, where anti-inflammatory activity was also observed. Vafidemstat has also been tested in a Phase II in severe Covid-19 patients (ESCAPE) assessing the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection, where it showed significant anti-inflammatory effects in severe Covid-19 patients. Vafidemstat is currently advancing as a Phase III-ready asset in Borderline Personality disorder (BPD) following completion of the global, randomized, double blind Phase IIb PORTICO trial (final data presented at ECNP-2024). Following receipt of the minutes from the End-of-Phase II meeting with the FDA to discuss PORTICO’s results, the company announced plans to move forward with a Phase III PORTICO-2 trial in agitation/aggression in BPD (FDA submission planned in 1H2025). Vafidemstat is also being investigated in a double-blind, randomized, placebo-controlled Phase IIb trial in negative symptoms of schizophrenia (EVOLUTION trial, recruitment ongoing). The company is also deploying a CNS precision medicine approach with vafidemstat in genetically-defined patient subpopulations of certain CNS disorders and is evaluating a clinical trial in Kabuki Syndrome patients. The company is also exploring the clinical development of vafidemstat in other neurodevelopmental syndromes. FORWARD-LOOKING STATEMENTS This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration.. Spain Oryzon IR & Media, Europe & US Patricia Cobo/Mario Cordera Emili Torrell Sandya von der Weid Atrevia Chief BD Officer LifeSci Advisors, LLC +34 91 564 07 25 +34 673 33 97 65 +34 93 515 1313 +41 78 680 05 38 pcobo@atrevia.com mcordera@atrevia.com etorrell@oryzon.com svonderweid@lifesciadvisors.com

A new, closely watched schizophrenia medication from Bristol Myers Squibb sold well in its first full quarter on the market, according to both the company and analysts.In its latest earnings report, Bristol Myers recorded $27 million in net sales from Cobenfy during the three-month period between January and March. Chief Financial Officer David Elkins called that a solid start and said the weekly number of Cobenfy prescriptions remains strong, tracking ahead of all branded specific benchmarks. As of April 11, that number was 1,655.Matt Phipps, an analyst at the investment firm William Blair, echoed those sentiments in a note to clients, describing the results as a nice start. David Risinger, of Leerink Partners, noted that the sales total was 48% above the average analyst estimate.The drugs success is imperative for Bristol Myers, which has watched generic competition erode one of its most lucrative products, the blood cancer treatment Revlimid. Two of its other top-selling medicines,the blood thinner Eliquis and the cancer immunotherapy Opdivo, are also poised to lose patent protection in a few years. Across its whole business, Bristol Myers recorded $11.6 billion in revenue in the first quarter, a 6% decline from the same period last year.Bristol Myers sees Cobenfy as a major product, hence why the company recently shelled out $14 billion to acquire its developer, Karuna Therapeutics. Analysts also have blockbuster expectations, though those were tempered a bit this week when the drug stumbled in testing.Having already secured an approval in schizophrenia, Bristol Myers has been trying to show Cobenfy has a range of other uses. Those include treating bipolar disorder, autism spectrum disorder, symptoms associated with Alzheimers disease, or even being layered onto other antipsychotics for schizophrenia patients.In that last category, however, a nearly 400-person clinical trial just found Cobenfy was not significantly better than a placebo. To some analysts, the setback erases billions of dollars in potential revenue.Perhaps expectedly, Bristol Myers leadership talked down the study failure on a call with investors Thursday. Chief Commercialization Officer Adam Lenkowsky said schizophrenia monotherapy remains the most significant opportunity, accounting for 70% to 80% of the patients treated with Cobenfy.What happens in the real world? Psychiatrists exhaust monotherapy options before trying adjunctiveuse, Lenkowsky said. That's exactly what we've been seeing in the market.Physicians have also told us, both in research and through advisory boards, that missing the endpoint of the study would have no impact on monotherapy usage or their willingness to use Cobenfy, he added.More broadly, Bristol Myers upped its revenue forecast for the year by 2%, to a range of about $45.8 billion to $46.8 billion. The raise was based on operational sales as well as the impact of foreign exchange rates.During the investor call, analysts also pressed the company about its plans to navigate the current political environment, especially Trump administration tariff policies. The U.S. pharmaceutical industry has so far avoided the worst of the tariff impact, but there are strong signs it could get caught in the crosshairs before too long.We have been up investing in core infrastructure in the U.S. for many years. So we need to ensure that, ultimately, our trade policies enhance the sector and support efforts like the ones we've been making, CEO Chris Boerner told investors.In terms of our exposure, again, what we've said is the tariffs that have gone into place, namely around China, have been reflected in the guidance that we provided today, Boerner added.It really is simply too early to provide a lot more on pharma-specific sectors, so we'll have to wait for the specifics there.Bristol Myers shares were about flat as of late Thursday morning, trading a little above $48 apiece. '