Dwarfism

Investigator-Sponsored Phase 2 Study Demonstrated Positive Results for VOXZOGO in Children with Hypochondroplasia New Analyses Highlight VOXZOGO 7-year Data and Improvement on Quality of Life in Children with Achondroplasia SAN RAFAEL, Calif., March 12, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024. Researchers will also present data from Phase 2 and Phase 3 studies of the medicine in children with achondroplasia, including results that demonstrate VOXZOGO's positive impact on quality of life. Results in Hypochondroplasia Andrew Dauber, M.D., will present positive results from his investigator-sponsored Phase 2 study of VOXZOGO in children with hypochondroplasia. The annualized growth velocity (AGV) increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period (mean difference: 1.81 cm/year, p<0.0001). No new safety signals were reported. "We are encouraged by preliminary results from the first study evaluating VOXZOGO in children with hypochondroplasia, suggesting the medicine has the potential to impact growth outcomes for this group of patients," said Dr. Dauber, who is the lead study author and Chief of Endocrinology at Children's National Hospital in Washington, D.C. "There are currently no approved medicines for children with hypochondroplasia, so we look forward to gathering additional data to deepen our understanding of VOXZOGO in hypochondroplasia and other growth-related conditions." In late 2023, BioMarin launched the pivotal clinical trial program studying the safety and efficacy of VOXZOGO in children with hypochondroplasia. A multinational observational study in children with hypochondroplasia (111-902) is currently recruiting participants, and the company plans to enter the treatment phase (Phase 3 trial) later this year. New Data Highlights VOXZOGO Long-Term Efficacy, Safety and Quality of Life in Achondroplasia 7-year Phase 2 Results in Achondroplasia Data from two studies of VOXZOGO in children ≥5 years of age with achondroplasia demonstrated a sustained improvement in AGV for each year that the participants were treated. In the Phase 2 study with more than 7 years of follow up, the mean increase in growth across each year of age up to 16 years compared with untreated participants was 1.63 cm/year for boys and 1.33 cm/year for girls. Separate cross-sectional comparative analysis assessing increase in height over a 7-year period showed an additional height gain of 11.03 cm with use of VOXZOGO compared to matched untreated children over the same period. 4-year Phase 3 Results in Achondroplasia The same analyses were produced for the Phase 3 trial in which children with achondroplasia have a mean treatment follow-up of 4 years. The mean increase in growth across each year of age up to 17 years was 1.73 cm/year for boys and 1.46 cm/year for girls. A separate cross-sectional comparative analysis assessing increase in height over a 3-year period showed an additional height gain of 5.75 cm with use of VOXZOGO compared to matched untreated children over the same period. 3-year Phase 3 Quality of Life Results in Achondroplasia Results from a separate analysis of the Phase 3 study showed that VOXZOGO improved health-related quality of life (HRQoL) among children with achondroplasia, particularly those associated with physical activities. After 3 years, the mean increase in Quality of Life in Short Stature Youth (QoLISSY) physical domain score was 6.0 as reported by caregivers and 6.3 as reported by children. These improvements were even more pronounced in children who grew more (for those with ≥ 1 SD increase in height z-score, the mean increase in physical domain score was 11.4 as reported by caregivers and 8.5 as reported by children). "The clinical results at ACMG reinforce the long-term benefit we are seeing in children treated with VOXZOGO, with a meaningful median height gain of more than 11 cm in children with achondroplasia following 7 years of treatment," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. "We are particularly encouraged by new data demonstrating the potential impact height gains from VOXZOGO can have on quality of life, an outcome of incredible importance for children and families impacted by achondroplasia." "Achondroplasia can have a negative effect on a child's quality of life, including physical, social and emotional aspects," said Melita Irving, M.D., study investigator and consultant clinical geneticist at Guy's and St. Thomas' NHS Foundation Trust in London. "Our studies showed that increasing height with long-term administration of VOXZOGO can result in meaningful improvements in quality-of-life measures for children with achondroplasia." BioMarin's full list of presentations at ACMG includes: BioMarin Poster Presentations Persistence of Growth-Promoting Effects in Children with Achondroplasia Up to 7 Years: Update from a Phase 2 Extension Study with Vosoritide Poster #: P144 Friday, March 15, 2024, 10:30 a.m. – 12 p.m. ET Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia for Up to 4 Years: Update from Phase 3 Extension Study Poster #: P139 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia is Accompanied by Improvement in Physical Aspects of Quality of Life Poster #: P141 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Persistence of Growth-Promoting Effects in Infants and Toddlers with Achondroplasia: Results from a Phase 2 Extension Study with Vosoritide Poster #: P131 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Exploring the Landscape of Phenylketonuria Education and Learning Needs of Genetics Trainees Poster #: P049 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Reduction of Blood Phenylalanine in Participants Enrolled in OPAL, an Observational Study, Mirror Findings from the U.S.-based PRISM Population Poster #: P034 Friday, March 15, 2024, 10:30 a.m. – 12 p.m. ET Investigator-Sponsored Oral Presentation Vosoritide Increases Growth Velocity in Hypochondroplasia: Phase 2 Trial Results Oral Session: O03 Thursday, March 14, 2024, 1:30 – 3 p.m. ET About VOXZOGO In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. VOXZOGO is approved in the U.S. and indicated to increase linear growth in children with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history. Patient Support Accessing VOXZOGO To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail VOXZOGOSupport@biomarin-rareconnections.com. For more information about VOXZOGO, please visit . For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com. About Achondroplasia Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face, and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth. More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category. VOXZOGO U.S. Important Safety Information What is VOXZOGO used for? VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO? VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO? The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken? VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO? Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Please see additional safety information in the full Prescribing Information and Patient Information. About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of the genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data to be presented at the 2024 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting, including the six poster presentations and the investor-sponsored oral presentation; the development of BioMarin's VOXZOGO program generally; the potential benefits of VOXZOGO for children with achondroplasia, including the duration of such benefits, potential improvement in annualized growth velocity (AGV), and potential improvement in health-related quality of life; the potential efficacy of VOXZOGO for children with hypochondroplasia, including potential to impact growth outcomes; and the continued clinical development of VOXZOGO, including BioMarin's plans for launching the treatment phase of the Phase 3 trial studying the safety and efficacy of VOXZOGO in children with hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2023 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. Company Codes: NASDAQ-NMS:BMRN

Bionano will host a pre-conference scientific session that will include a live product demonstration of the Stratys™ system for high throughput optical genome mapping (OGM) and findings from early access customersThe pre-conference event will also include a 2024 corporate overview from Bionano’s chief executive officer, Dr. Erik Holmlin, presentations from Dr. Brynn Levy at Columbia University Medical Center, Dr. Ulrich Broeckel at Medical College of Wisconsin, Dr. Susan Crocker at Kingston Health Sciences Centre, Dr. Liz McCready at Hamilton Health Science Center, and Dr. Zeid Hamadeh at Vancouver General Hospital covering the utility of OGM and VIA™ software and the adoption, implementation, and advanced research capabilities of the Stratys system, and will conclude with a fireside chat with panelists hosted by Bionano’s chief medical officer, Dr. Alka ChaubeyIn a sponsored session, Dr. Chaubey will cover findings from large multi-site clinical studies using OGM as well as features of the Stratys system. Dr. Tara Spence from Vancouver General Hospital will present insights into her laboratory’s adoption of Stratys and its potential impact on hematological malignancy analysis A scientific platform presentation will feature Drs. Hamadeh and Spence discussing OGM’s utility for the genotyping of hematological neoplasmsDr. Holmlin will participate with Dr. Nancy Mendelsohn, president of the ACMG Foundation, in the foundation’s educational and clinical laboratory genetics and genomics (LGG) awards ceremony by presenting the fellowship awards sponsored by BionanoEleven scientific posters featuring results from OGM applications in cancer, postnatal applications and genetic disorders will be presented at the conference SAN DIEGO, March 07, 2024 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) today announced its participation in the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting 2024 with a broad range of content covering optical genome mapping’s (OGM) utility for research areas including cancer, rare genetic disease and constitutional disorders. ACMG’s Annual Meeting brings together industry, medical, and academic professionals to discuss advances in clinical genetics research. The ACMG conference will be held March 12-16, 2024, in Toronto, Canada. All scientific posters will be presented in Exhibit Halls DE. Poster presentations and scientific workshop sessions on OGM include: Poster Number Title AuthorsPresentedP573 Genome-wide short tandem repeat expansion screening using optical genome mapping Yu J.March 14, 202410:30 AM-12:00 PM ETP583 Clinical utility of optical genome mapping as an additional test to standard cytogenetic workup of hematological malignancies Toruner G.March 14, 202410:30 AM- 12:00 PM ETP839 A curated research catalogue of structural variation detected by optical genome mapping Pang A.March 14, 202410:30 AM-12:00 PM ETP088 A comprehensive approach to evaluate genetic abnormalities in plasma cell neoplasms using optical genome mapping and next-generation sequencing Zou Y.March 15, 202410:30 AM-12:00 PM ETP608 Optical genome mapping for genome-wide structural variation analysis in hematologic malignancies: results of a prospective study and potential impact on diagnosis and management Sahoo T.March 15, 202410:30 AM-12:00 PM ETP656 Improved diagnostic paradigm using optical genome mapping (OGM) for cytogenomic testing for recurrent pregnancy loss and infertility Crocker S.March 15, 202410:30 AM-12:00 PM ETP694 Case report: unraveling a complex chromosomal rearrangement case using optical genome mapping Ozcan Z.March 15, 202410:30 AM- 12:00 PM ETP742 Assessing stability of frozen samples for Bionano optical single DNA mapping for diagnosis of Facioscapulohumeral Muscular Dystrophy Type 1 Cook S.March 15, 202410:30 AM-12:00 PM ETP746 Genome wide, high-throughput, high-resolution structural variation detection at low variant allele fraction for oncology samples Hastie A.March 15, 202410:30 AM-12:00 PM ETP842 Unified comprehensive analysis of NGS and optical genome mapping data for constitutional applications using Bionano VIA™ software Norgaard Z.March 15, 202410:30 AM-12:00 PM ETP850 Accelerated optical genome mapping analysis with Stratys Compute and Guided Assembly Senol Cali D.March 15, 202410:30 AM-12:00 PM ETSession Title PresenterPresentedSponsored Workshop LIVE Bionano product showcase – Stratys™ revealed: exclusive first look at the future of OGM Chaubey A., Broeckel U., Crocker S., Hamadeh Z., Holmlin E., Jiandani D., Levy B., McCready L., Sahoo T.March 12, 20248:00 AM- 12:30 PM ETDelta Hotel by Marriott- Kensington BallroomPlenary Session 2024 ACMG Foundation Awards and Presidential Plenary Session Holmlin E.March 13, 202410:00-12:00 PM ETExhibit Hall FGPlatform Presentation A Canadian lab’s clinical validation experience with optical genome mapping as a front-line diagnostic test for hematological neoplasms Hamadeh Z., Spence T.March 14, 20244:15-5:45 PM ETMTCC- 714/716Sponsored Workshop Revolutionizing cytogenomics with Stratys™: unveiling a new frontier in structural variant assessment through optical genome mapping at scale Chaubey A., Spence T.,March 15, 202410:45-11:15 AM ETExhibit Theater 2 Erik Holmlin, PhD, president and chief executive officer of Bionano, added, “We are proud to debut our new high throughput system for OGM, Stratys, at ACMG this year, and to host sessions featuring customers from our Stratys early access program presenting data from work conducted on the system. Researchers and scientists continue to push forward cutting-edge research in the human genetics space and we believe the capabilities of the Stratys system will unlock even more exciting research advancements. We look forward to these customers sharing their findings with the ACMG community.” More details on Bionano’s conference events can be found here. About Bionano Bionano is a provider of genome analysis solutions that can enable researchers and clinicians to reveal answers to challenging questions in biology and medicine. Bionano’s mission is to transform the way the world sees the genome through OGM solutions, diagnostic services and software. Bionano offers OGM solutions for applications across basic, translational and clinical research. Through its Lineagen, Inc. d/b/a Bionano Laboratories business, Bionano also provides diagnostic testing for patients with clinical presentations consistent with autism spectrum disorder and other neurodevelopmental disabilities. Bionano also offers an industry-leading, platform-agnostic software solution, which integrates next-generation sequencing and microarray data designed to provide analysis, visualization, interpretation and reporting of copy number variants, single-nucleotide variants and absence of heterozygosity across the genome in one consolidated view. Bionano additionally offers nucleic acid extraction and purification solutions using proprietary isotachophoresis technology. Forward-Looking Statements of Bionano This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “believe,” “potential,” “will,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: OGM’s utility for applications in cancer, rare genetic disease and constitutional disorder research and in the areas reported in the presentations given and the posters made available at ACMG’s 2024 Annual Meeting; the ability of the Stratys system to unlock research advancements; and the growth and adoption of OGM. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks and uncertainties associated with: the impact of adverse geopolitical and macroeconomic events, such as the ongoing conflicts between Ukraine and Russia and Israel and Gaza and uncertain market conditions, including bank failures, inflation and supply chain disruptions, on our business and the global economy; general market conditions; changes in the competitive landscape and the introduction of competitive technologies or improvements to existing technologies; failure of OGM to prove useful in areas including applications in cancer, rare genetic disease and constitutional disorder research and in the areas reported in the presentations given and the posters made available at ACMG’s Annual Meeting; failure of the Stratys system to unlock research advancements; failure of laboratories to adopt OGM; the ability of our OGM solutions to offer the anticipated benefits for and contributions to the areas reported in the presentations given and posters made available at the ACMG’s 2024 Annual Meeting; future study results contradicting the results reported in the presentations given and posters made available at the ACMG’s 2024 Annual Meeting; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of our technologies; and the risks and uncertainties associated with our business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2023 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise. CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionano.com Investor Relations:David HolmesGilmartin Group+1 (858) 888-7625IR@bionano.com

REDWOOD CITY, Calif., March 06, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today provided a corporate update, and reported financial results for the fourth quarter and full-year ended December 31, 2023. Full Year 2023 and Recent Corporate Highlights Announced positive statistically significant top-line data from the randomized withdrawal period of Study C602, a long-term treatment study of DCCR (Diazoxide Choline) Extended-Release tablets for the treatment of Prader-Willi syndrome (PWS) in September 2023. The study met its primary endpoint, demonstrating a highly statistically significant difference in change from baseline in HQ-CT total score for DCCR compared to placebo (p=0.0022). Secondary endpoints of Clinical Global Impression of Severity (CGI-S) and Clinical Global Impression of Improvement (CGI-I) both showed strong trends towards worsening in the placebo group compared to DCCR (p=0.08 and 0.09), respectively. DCCR continued to be generally well-tolerated in the randomized withdrawal period with no new or unexpected safety signals. Planned submission of a New Drug Application (NDA) for DCCR in PWS remains on track for mid-2024, after receipt of pre-NDA meeting minutes from the U.S. Food and Drug Administration. The FDA stated that the potential for data from the DCCR clinical program to provide substantial evidence of effectiveness will be a matter of review following the submission of an NDA. Closed on gross proceeds of approximately $129 million from an underwritten public offering of common stock and concurrent private placement of common stock and pre-funded warrants in October 2023, and received $43.1 million from the sale and exercise of warrants issued in connection with the December 2022 Securities Purchase Agreement. Strengthened leadership team with appointments of Meredith Manning, M.B.A as Chief Commercial Officer, Dairine Dempsey, Ph.D. as Vice President, Europe and Lauren Budesheim, M.S. as Vice President of Human Resources. “Following recent feedback from the FDA, Soleno remains focused on preparing an NDA submission for DCCR in PWS mid-2024,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “We believe data from our extensive clinical program, including highly statistically significant top-line results from the randomized withdrawal period of Study C602, supports that DCCR has the potential to be a safe and effective therapy for patients with PWS. With our strengthened leadership team and balance sheet, we are ideally positioned to carry out our corporate strategy and, if approved, deliver a new treatment option for PWS patients.” Financial Results Soleno’s current research and development efforts are primarily focused on advancing its lead product candidate, DCCR, for the treatment of PWS, through late-stage clinical development. Fourth Quarter and Full Year Ended December 31, 2023 Financial Results As of December 31, 2023, Soleno had cash and cash equivalents of approximately $169.7 million, which includes $129 million received upon the closing of the public offering and concurrent private placement in October 2023, and $43.1 million received from the sale and exercise of warrants issued in connection with the December 2022 Securities Purchase Agreement. The Company may receive an additional $16.9 million from the exercise of the remaining warrants issued pursuant to the December 2022 Securities Purchase Agreement. Research and development expenses were $8.7 million for the quarter ended December 31, 2023, compared to $3.8 million in the same period of 2022, and $25.2 million for the year ended December 31, 2023, compared to $15.3 million for the year ended 2022. The increase was primarily due to increased clinical trials costs, manufacturing efforts, and expenditures in support of our NDA submission. General and administrative expenses were $4.1 million for the quarter ended December 31, 2023, compared to $2.4 million in the same period of 2022, and $13.5 million for the year ended December 31, 2023, compared to $9.8 million for the year ended 2022. The increase was primarily related to higher stock-based compensation expense, higher costs because of an increase in headcount and higher professional and consulting expenses in 2023. Soleno is obligated to make cash payments of up to a maximum of $21.2 million to the former Essentialis stockholders upon the achievement of certain commercial milestones associated with the future sales of DCCR in accordance with the terms of Soleno’s 2016 merger agreement with Essentialis. The fair value of the liability for the contingent consideration payable by the Company upon achieving two commercial sales milestones of $100 million and $200 million in revenue in future years was estimated to be $11.5 million as of December 31, 2023, a $1.1 million increase from the estimate as of September 30, 2023. Total other income (expense), net, was $2.6 million for the quarter ended December 31, 2023, and $0.1 million in the same period of 2022. For the year, total other income (expense), net, was $2.4 million for 2023, and $0.3 million for 2022. Other income (expense) consists of interest income and changes in the fair value of the Company’s warrant liability. Net loss was approximately $11.3 million, or $0.33 per basic and diluted share, for the quarter ended December 31, 2023, and $5.5 million, or $0.58 per basic and diluted share, in the same period of 2022. Net loss for the year ended December 31, 2023, was approximately $39.0 million, or $2.36 per basic and diluted share, compared to a net loss of approximately $24.1 million, or $2.87 per basic and diluted share, for the year ended December 31, 2022. About PWS The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening feeling of intense, persistent hunger, food pre-occupation, extreme drive to food seek and consume food that severely diminish the quality of life for patients with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant morbidities (e.g., obesity, diabetes, cardiovascular disease) and mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior). In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parent and caregivers) rated hyperphagia and 92.9% rated body composition as either the most important or a very important symptom to be relieved by a new medicine. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder. About DCCR (Diazoxide Choline) Extended-Release Tablets DCCR is a novel, proprietary extended-release dosage form containing the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has not been approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in patients with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in patients with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters. Diazoxide choline has received Orphan Drug Designation for the treatment of PWS in the U.S. and E.U., and Fast Track Designation in the U.S. About Soleno Therapeutics, Inc. Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s lead candidate, DCCR extended-release tablets, a once-daily oral tablet for the treatment of PWS, recently completed its Phase 3 development program to support a planned NDA submission. For more information, please visit . Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the timing of any regulatory process or ultimate approvals and determining a path forward for DCCR for the treatment of PWS. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including the risks and uncertainties associated with the projected timeline of our NDA submission, whether FDA will agree with our interpretation of the data or the adequacy of data to support an NDA, the FDA’s review of our NDA, market conditions, as well as risks and uncertainties inherent in Soleno’s business, including those described in the company's prior press releases and in the periodic reports it files with the SEC. The events and circumstances reflected in the company's forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, the company does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Corporate Contact: Brian Ritchie LifeSci Advisors, LLC 212-915-2578 Soleno Therapeutics, Inc. Condensed Consolidated Balance Sheets (In thousands except share and per share data) December 31, 2023 December 31, 2022 Assets Current assets Cash and cash equivalents $ 169,681 $ 14,602 Prepaid expenses and other current assets 1,677 1,045 Total current assets 171,358 15,647 Long-term assets Property and equipment, net 12 26 Operating lease right-of-use assets 407 131 Intangible assets, net 8,749 10,693 Other long-term assets 165 — Total assets $ 180,691 $ 26,497 Liabilities and stockholders’ equity Current liabilities Accounts payable, net $ 3,149 $ 1,777 Accrued compensation 3,135 1,675 Accrued clinical trial site costs 3,393 3,222 Operating lease liabilities 273 155 Other current liabilities 1,555 484 Total current liabilities 11,505 7,313 Long-term liabilities 2018 PIPE Warrant liability - 1 Contingent liability for Essentialis purchase price 11,549 8,835 Long-term lease liabilities 130 — Total liabilities 23,184 16,149 Commitments and contingencies (Note 8) Stockholders’ equity Preferred stock, $0.001 par value; 10,000,000 shares authorized, no shares issued and outstanding — — Common stock, $0.001 par value, 100,000,000 shares authorized, 31,678,159 and 8,159,382 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively 32 8 Additional paid-in-capital 433,885 247,762 Accumulated deficit (276,410 ) (237,422 ) Total stockholders’ equity 157,507 10,348 Total liabilities and stockholders’ equity $ 180,691 $ 26,497 Soleno Therapeutics, Inc. Condensed Consolidated Statements of Operations (In thousands except share and per share data) Three Months Ended December 31, For the Years Ended December 31, 2023 2022 2023 2022 Operating expenses Research and development $ 8,689 $ 3,810 $ 25,189 $ 15,265 General and administrative 4,410 2,402 13,481 9,844 Change in fair value of contingent consideration 1,081 (602 ) 2,714 (712 ) Total operating expenses 13,910 5,610 41,384 24,397 Operating loss (13,910 ) (5,610 ) (41,384 ) (24,397 ) Other income (expense), net Change in fair value of warrant liability 470 (1 ) (182 ) 30 Interest income 2,144 125 2,578 300 Total other income (expense), net 2,614 124 2,396 330 Net loss $ (11,296 ) $ (5,486 ) $ (38,988 ) $ (24,067 ) Net loss per common share, basic and diluted $ (0.33 ) $ (0.58 ) $ (2.36 ) $ (2.87 ) Weighted-average common shares outstanding used to calculate basic and diluted net loss per common share 34,441,721 9,440,347 16,492,132 8,397,088