ARG1

NEW YORK, Oct. 04, 2024 (GLOBE NEWSWIRE) -- IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, off-the-shelf, immune-modulating therapeutic cancer vaccines based on its T-win® platform, today announced two abstracts accepted for poster presentation at the Society for Immunotherapy of Cancer’s 39th Annual Meeting (SITC 2024) taking place taking place in Houston, Texas on November 8-10, 2024. The first abstract accepted for poster presentation contains updated and expanded data from the complete cohort (n=31) from the first line (1L) cohort of patients with PD-L1 high metastatic non-small cell lung cancer (NSCLC) in the Company’s Phase 2 basket trial of investigational IO102-IO103 in combination with pembrolizumab (KEYTRUDA®), Merck’s anti-PD-1 therapy (IOB-022/KN-D38). The second abstract accepted for poster presentation contains new pre-clinical data for the next candidate in the IO Biotech pipeline, IO112, which targets Arginase 1. Poster Presentations Title: A phase 2 trial of the IO102-IO103 cancer vaccine plus pembrolizumab: results from the first-line (1L) cohort of PD-L1 high metastatic non-small cell lung cancer (NSCLC) Presenter: Jonathan W. Riess, MD, MS, UC Davis Comprehensive Cancer CenterPoster presentation number: 756Date: Saturday, November 9, 2024Location: Exhibit Halls AB - George R. Brown Convention CenterTimes: Poster hall: 9:00 a.m. - 8:30 p.m. CDT; Poster session 12:00 – 1:30 p.m.; Poster reception 7:00-8:30 p.m. Title: Immune modulating vaccine against arginase 1 controls tumor growth via modulation of tumor-associated macrophages Presenters: Evelina Martinenaite, PhD, Senior Scientist, Translational Research, and Inés Lecoq, PhD, Scientist, Translational Research, IO BiotechPoster presentation number: 1038Date: Saturday, November 9, 2024Location: Exhibit Halls AB - George R. Brown Convention CenterTimes: Poster hall: 9:00 a.m. - 8:30 p.m. CDT; Poster session 12:00 – 1:30 p.m.; Poster reception 7:00-8:30 p.m. The posters will be available on the “Posters & Publications” page of the IO Biotech website at the start of the meeting. About IO102-IO103 IO102-IO103 is an investigational off-the-shelf therapeutic cancer vaccine designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment (TME) by stimulating activation and expansion of T cells against indoleamine 2,3-dioxygenase (IDO) positive and programmed death-ligand 1 (PD-L1) positive cells. The company is currently conducting a pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) investigating IO102-IO103 in combination with pembrolizumab versus pembrolizumab alone in patients with advanced melanoma, a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709) investigating IO102-IO103 in combination with pembrolizumab as first line treatment in patients with solid tumors, and a Phase 2 basket trial (IOB-032/PN-E40; NCT05280314) investigating IO102-IO103 in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors. The clinical trials are sponsored by IO Biotech and conducted in collaboration with Merck, which is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103. KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. About IOB-022/KN-D38 Phase 2 Solid Tumor Basket Trial IOB-022/KN-D38 (NCT05077709) is a non-comparative, open-label trial to investigate the safety and efficacy of IO102-IO103 in combination with pembrolizumab in first-line advanced cancers in non-small cell lung cancer (NSCLC) and squamous cell carcinoma of the head and neck (SCCHN). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103. About IO Biotech IO Biotech is a clinical-stage biopharmaceutical company developing novel, immune-modulating therapeutic cancer vaccines based on its T-win® platform. The T-win platform is based on a novel approach to cancer vaccines designed to activate T cells to target the immunosuppressive cells in the tumor microenvironment. IO Biotech is advancing its lead cancer vaccine candidate, IO102-IO103, in clinical trials, and additional pipeline candidates through preclinical development. Based on positive Phase 1/2 first line metastatic melanoma data, IO102-IO103, in combination with pembrolizumab, has been granted a breakthrough therapy designation for the treatment of advanced melanoma by the US Food and Drug Administration. IO Biotech is headquartered in Copenhagen, Denmark and has US headquarters in New York, New York. For further information, please visit www.iobiotech.com. Follow us on our social media channels on LinkedIn and X (@IOBiotech). Forward-Looking Statement This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements, including regarding the timing or outcome of primary analysis of the company’s Phase 3 trial, other current or future clinical trials, their progress, enrollment or results, or the company’s financial position or cash runway, are based on IO Biotech’s current assumptions and expectations of future events and trends, which affect or may affect its business, strategy, operations or financial performance, and actual results and other events may differ materially from those expressed or implied in such statements due to numerous risks and uncertainties. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Because forward-looking statements are inherently subject to risks and uncertainties, you should not rely on these forward-looking statements as predictions of future events. These forward-looking statements speak only as of the date hereof and should not be unduly relied upon. Except to the extent required by law, IO Biotech undertakes no obligation to update these statements, whether as a result of any new information, future developments or otherwise. Contact: InvestorsMaryann Cimino, Director of Investor Relations IO Biotech, Inc.617-710-7305mci@iobiotech.com MediaJulie FunestiSalutem917-498-1967Julie.Funesti@salutemcomms.com

In H1 2024, Molecure focused on the development of two key molecules: OATD-01 and OATD-02, as well as expanding research activities based on, among other things, advanced artificial intelligence technologies In May, Molecure received approval to initiate a Phase II clinical trial (KITE) for clinical candidate OATD-01 for the treatment of pulmonary sarcoidosis in the European Union and Norway. Earlier, Molecure received approval from the U.S. FDA and the U.K. MHRA In the OATD-01 clinical trial, the Company currently has 13 sites activated in the US, the European Union, the UK and Norway. More will be activated by mid-November this year. Progress in the Phase I clinical trial of OATD-02, the first-in-class dual arginase inhibitor being developed for the treatment of cancer. Following analysis of clinical and pharmacokinetic data and Safety Review Committee (SRC) recommendations, recruitment continues with the addition of 3 additional patients in the fourth cohort at the 20 mg dose level, with the goal of expanding the amount and scope of data to inform decisions on further dose escalation Molecure has confirmed in in vitro tests the ability of the second molecule already being developed within the mRNA platform to effectively bind to the presumed mRNA fragment and block the translation process of the pathogenic protein. Reaching the PoC (in vitro proof-of-concept) stage for yet another molecule being developed in the mRNA platform is evidence of the effectiveness of Molecure’s strategy to identify mRNA-binding compounds with therapeutic potential. In July, Molecure and Avicenna Biosciences entered into a strategic research collaboration agreement to optimize USP7’s small-molecule drug discovery and development processes. The collaboration will combine Molecure’s extensive drug discovery and USP7 biology expertise with Avicenna’s machine learning-based medicinal chemistry platform to identify new drug candidates with superior pharmaceutical properties. In August, Dr Piotr Iwanowski, M.D., was appointed Chief Medical Officer (CMO) and Member of the Management Board of Molecure S.A., he will assume the above functions on September 30th this year. Warsaw, 27 September 2024 – Molecure S.A. (“Molecure”, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage and leverages its globally unique expertise in medicinal chemistry and biology to explore and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA function to treat a wide range of incurable diseases, has released its report for the first half of 2024. The report is available at: https://molecure.com/pl/informacje-dla-inwestorow/ Marcin Szumowski, CEO Molecure S.A. „In the past six months, our main focus has been on the continued clinical development of OATD-01 and OATD-02. We continued to recruit to clinical trial of OATD-01, a first-in-class chitotriosidase 1 (CHIT1) inhibitor, in patients with pulmonary sarcoidosis on both continents. A positive decision by regulatory authorities from European countries, following earlier approval in the US and UK, has given us the “green light” to conduct Phase II trials of OATD-01 also in Denmark, France, Greece, Germany and Norway. We still hope that we will be able to involve Polish centers and Polish patients in this study. We are planning the first readouts of intermediate signals of therapeutic efficacy and safety in patients with sarcoidosis at the first half of 2025. However, due to the double-blinding requirement of the study, the publication of the final results will occur after the completion of the study and is scheduled for 2026. Our attention in the OATD-01 program is increasingly focused on the area of metabolic diseases, particularly MASH (metabolic dysfunction-associated steatohepatitis). We have very good preclinical results and there are a growing number of scientific publications from genetic studies and based on samples from MASH patients highlighting the key role of CHIT1 in disease escalation. The MASH market is projected to grow at more than 40% CAGR annually to a size of more than $25 billion in 2032. Molecure aims to play a significant role in treatment in MASH in partnership with a major company in the area of progressive metabolic liver disease. Scientific data and market projections support our assumptions about the therapeutic potential of blocking chitinases in multi-organ diseases. In the Phase I clinical trial of OATD-02, an innovative arginase inhibitor, we are continuing to recruit and add 3 more patients to this cohort after analyzing data from the fourth cohort at the 20 mg daily dose level and the Safety Review Committee’s recommendation. As recommended by the SRC, we want to collect more extensive pharmacokinetics and pharmacodynamics data at this stage in order to decide whether to further increase the dose. We are confident that this next phase will bring us significantly closer to bringing breakthrough therapies to market that can change the lives of patients around the world. We see great potential for a platform of small-molecule drug discovery that modulates mRNA function. In December 2023, we obtained positive in vitro results for the first of our molecules, and just a few months later we confirmed translation inhibition for another molecule targeting a different mRNA target. At the BIO International Convention in San Diego in early June, we had many meetings with representatives of Big Pharma companies interested in collaborating on the mRNA platform. In small molecule technology targeting mRNA, we are at the European and even global forefront, so we assume that establishing a commercially attractive collaboration with a large industry partner is likely within the next year. Also noteworthy is the establishment of a strategic collaboration with Avicenna Biosciences to support the discovery and development of new small-molecule drugs targeting ubiquitin-specific protease 7 (USP7), a deubiquitinating enzyme that promotes oncogenesis and whose high expression is observed in a number of cancers. By combining Molecure’s expertise on this attractive, in vivo validated target with Avicenna’s machine learning-driven medicinal chemistry platform, we intend to generate better drug candidates with optimal pharmacological profiles for further preclinical and clinical development.” Webinar with Molecure’s Management Board A meeting will be held on October 1, 2024 at 2:00 pm to discuss the Company’s operational and financial situation for 1H 2024, including the presentation of progress in ongoing research programs, as well as development plans for the coming quarters. The meeting will be held in online format, at the link: Original path – Polish:https://livingmedia.com.pl/live/molecure/1H2024-pl English: https://livingmedia.com.pl/live/molecure/1H2024-en Business Development activities In the first half of 2024, representatives of the Company participated in two important industry conferences: BIO Europe Spring in Barcelona, where they held 38 meetings with biotech and pharmaceutical industry representatives, mainly potential partners, investors and global pharmaceutical companies. The key topics of discussion were OATD-01 (more than 50% of the meetings), OATD-02 and the RNA platform, BIO International Convention in San Diego, where they held a total of a record 42 meetings, of which as many as 28 were related to OATD-01, demonstrating the growing and increasingly concrete interest in the results of preclinical and clinical research in lung diseases and also in metabolic diseases such as MASH. In addition, representatives of the Company participated in conferences: “Immunometabolism in Health and Disease” organized by Gordon Research Conferences at the American Bryant University in Smithfield, congress The European Congress of Life Sciences EUROBIOTECH in Krakow, Poland, International Conference on Medicinal Chemistry in Bordeaux, France, PHASE2024 in Warsaw, Americas Association of Sarcoidosis and Other Granulomatous Disorders (AASOG) in Baltimore, USA, IPF Summit in Boston, USA, and many others.. The company will participate in more industry conferences this year, including but not limited to major global events i.e. Bio Europe in Stockholm, as well as WASOG in India. Summary of financial data in 1H 2024* Operating income of PLN 0.4 million, up from PLN 1.8 million in 1H 2023. Operating expenses amounted to PLN 15.6 million, compared to PLN 17.2 million in 1Q 2023. 1H 2024 saw a decrease of PLN 1.6 million in operating expenses compared to 1H 2023. The main factor for the change was a decrease of PLN 0.5 million in program expenses, which, in accordance with the new accounting policy, are no longer capitalized under uncompleted development work. In addition, costs of legal and consulting services decreased by about PLN 0.9 million in 2024 compared to 2023. Net loss amounted to PLN 14.6 million compared to PLN 12.9 million in the same period in 2023. As of June 30, 2024, Molecure had cash of more than PLN 38 million, and the end of September this year. PLN 27 million. In addition, contracted grant funding for the coming years amounts to PLN 32.5 million. The company plans to raise further project grants as part of its expanding pipeline of clinical and preclinical projects. * Comparative figures for the first half of 2024 have been restated for comparability due to a change in accounting policy. *** About Molecure S.A. Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical stage, leveraging its own unique expertise in medicinal chemistry and biology to explore and develop first-in-class small-molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, could provide therapies for many incurable diseases. Molecure has generated a diverse portfolio of seven distinct programs with the support of leading academic research institutions around the world, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan and the International Institute of Molecular and Cell Biology in Warsaw, Poland (MIBMiK). The most advanced drug candidate developed by Molecure is OATD-01, a first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, and MASH, which is in phase II clinical trials. The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer, whose Phase I clinical trial has begun with first patient administration in Q1 2023. Molecure’s headquarters and laboratories are located in Warsaw and Lodz. The company is listed on the Warsaw Stock Exchange (ticker: MOC). For more detailed information, please visit: https://molecure.com/pl/ LinkedIn: Molecure | Twitter: @molecure_sa | YouTube: Molecure SA

-Recently Held Positive Pre-BLA Meeting with FDA with Aim to Accelerate Approval Pathway of Deramiocel for the Treatment of Duchenne Muscular Dystrophy- -Phase 3, HOPE-3 Trial of Deramiocel in DMD Fully Enrolled; On Track to Report Top-Line Data from Cohort A in Q4 2024- -Reported Positive 3-Year Skeletal and Cardiac Data from HOPE-2 Open-Label Extension Trial- -Conference Call and Webcast Today at 4:30 p.m. ET- SAN DIEGO, Aug. 07, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced its financial results for the second quarter ended June 30, 2024 and provided a corporate update. “We continue to make significant progress across our pipeline as we move closer to the filing of our Biologics License Application (BLA) of our lead asset, deramiocel (also referred to as CAP-1002), for the treatment of Duchenne muscular dystrophy (DMD),” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We recently conducted a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) and we will provide further updates on our plans as they become available. Furthermore, our recently announced 3-year HOPE-2 open label extension (OLE) data gives us confidence in the totality of safety and efficacy data we have seen year over year and the potential benefit that deramiocel can provide to patients with DMD. Lastly, on the corporate front, we remain focused on securing a partnership in Europe in order to support our balance sheet as we move towards major inflection points over the next several quarters.” Dr. Marbán continued, “In addition, we continue to progress our proprietary StealthX™ platform technology as part of our long-term strategy to leverage exosomes for therapeutic development. We continue to explore partnership opportunities and other non-dilutive sources of funding to advance this program.” Second Quarter 2024 and Recent Operational Highlights Deramiocel DMD Program: Deramiocel is an investigational cell therapy in Phase 3 development for the treatment of DMD. Deramiocel aims to slow disease progression through immunomodulatory, anti-inflammatory, and anti-fibrotic actions, with the goal of potentially improving skeletal and cardiac muscle function in patients with DMD. Deramiocel for the treatment of DMD has received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT). In addition, if Capricor receives FDA marketing approval for deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on our previous receipt of a rare pediatric disease designation. Announced the completion of a positive pre-BLA meeting with FDA that occurred in Q3 2024, where we discussed our rolling BLA submission schedule, potential label expansion, plans for commercial manufacturing as well as other topics. We plan to announce further details from this meeting once the final meeting minutes are made available. Announced positive results from our 36-month HOPE-2 OLE study at the Parent Project Muscular Dystrophy (PPMD) Annual Conference. The 3-year results showed a statistically significant benefit (+3.7 points, p<0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. Data also showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed volumes (left ventricular end systolic volume and left ventricular end diastolic volume). Announced enrollment has been completed for our HOPE-3 Phase 3 (Cohorts A and B) clinical trial which enrolled 105 subjects randomized to either deramiocel or placebo in a 1:1 ratio. Next steps for Cohort A: plan to readout top-line data in the fourth quarter of 2024.Next steps for Cohort B are under consideration. Announced positive results from a Type-B clinical FDA meeting held in Q2 2024 where FDA granted our request for a pre-BLA meeting.Announced positive results from a Type-B CMC FDA meeting held in Q1 2024. In the meeting, the FDA affirmed alignment on the following topics: Comparability between drug product manufactured at our two different facilities (Los Angeles and San Diego) has been demonstrated using the provided analytical comparability data.This allowed for the use of deramiocel drug product manufactured at our San Diego manufacturing facility upon potential product approval (subject to approval of the facility). Presented at the PPMD Cardiac Workshop III. Capricor was featured in a panel on industry perspectives on cardiac monitoring in DMD clinical trials.Presented at the Cure Duchenne 2024 Futures National Conference. Capricor presented the 24-month safety and efficacy results from the HOPE-2 OLE study. StealthX™Exosome Platform: Exosomes are membrane-bound extracellular vesicles which are secreted by most cells and contain characteristic lipids, proteins and nucleic acids. They act as messengers to regulate the functions of neighboring or distant cells and have been shown to regulate functions such as cell survival, proliferation, inflammation and tissue regeneration. We are developing our exosome technology using our proprietary StealthX™ platform focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. Our proprietary StealthX™ exosome-based multivalent vaccine (StealthX™ vaccine) for the prevention of SARS-CoV-2 was selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines. Under the terms of the collaboration, Capricor will supply the investigational product and NIAID's Division of Microbiology and Infectious Diseases will conduct the trial.Currently, our vaccine candidate is in the manufacturing phase with plans to deliver it to NIAID by the end of 2024. Presented data at the American Society of Gene and Cell Therapy 27th Annual Meeting. Highlights from the presentation included preclinical data showing a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.Presented data at the International Society of Extracellular Vesicles Annual Meeting 2024. Highlights from the abstract included preclinical data showing targeted cargo delivery to the lower limbs of mice by exosomes carrying a muscle targeting moiety by intravenous injection.Presented data at the International Society for Cell & Gene Therapy 2024 Meeting. Highlights from the abstract included preclinical data showing targeted delivery of ASOs using exosomes leads to exon skipping. Corporate Updates Announced that the Company has been added to the Russell 2000 and 3000 Indexes.Presented at the Cantor Fitzgerald’s Muscular Dystrophy Symposium. Anticipated Upcoming Milestones Plan to announce the outcome from our pre-BLA meeting in the third quarter of 2024.Plan to present additional 3-year data from our HOPE-2 OLE study at a medical meeting in the fourth quarter of 2024.Plan to report topline data from HOPE-3 (Cohort A) in the fourth quarter of 2024.Continue to explore opportunities for additional partnerships outside of the U.S. and Japan to support the potential approval and commercialization of deramiocel in DMD.Plan to provide updates on our NIAID collaboration for our StealthX™ vaccine as they become available.Continue to explore opportunities for partnerships and non-dilutive sources of funding to support advancement of our StealthX™ exosome platform technology. Second Quarter 2024 Financial Results Cash position: Cash, cash equivalents and marketable securities totaled approximately $29.5 million as of June 30, 2024 compared to approximately $39.5 million as of December 31, 2023. Additionally, in the second quarter of 2024, Capricor raised approximately $2.1 million in net proceeds through issuances of common stock at an average price of approximately $6.46 per share under its at-the-market offering program. Revenues: Revenues for the second quarter of 2024 were approximately $4.0 million compared to approximately $3.9 million for the second quarter of 2023. Additionally, revenues for the first half of 2024 were approximately $8.9 million compared to approximately $6.9 million for the first half of 2023. Capricor’s primary source of revenue was from the ratable recognition of the $40.0 million (upfront and milestone payments) in accordance with its U.S. Commercialization and Distribution Agreement with Nippon Shinyaku. Expenses: Total operating expenses for the second quarter of 2024 were approximately $15.6 million compared to approximately $11.7 million for the second quarter of 2023. Total operating expenses for the first half of 2024 were approximately $30.7 million compared to approximately $22.8 million for the first half of 2023. Net loss: The Company reported a net loss of approximately $11.0 million, or $0.35 per share, for the second quarter of 2024, compared to a net loss of approximately $7.4 million, or $0.29 per share, for the second quarter of 2023. The Company reported a net loss of approximately $20.8 million, or $0.66 per share, for the first half of 2024, compared to a net loss of approximately $15.1 million, or $0.60 per share, for the first half of 2023. Financial Outlook: We believe that based on the current operating plan and financial resources, Capricor’s available cash, cash equivalents and marketable securities will be sufficient to cover anticipated expenses and capital requirements into the first quarter of 2025. This expectation excludes any additional potential milestone payments under our Commercialization and Distribution Agreements with Nippon Shinyaku, as well as any strategic use of capital not currently in the Company’s base-case planning assumptions. Upcoming Events The Company plans to participate in the following upcoming events: H.C. Wainwright 26th Annual Global Investment Conference, September 9-11, 2024, New York, NY6th Annual Exosome-Based Therapeutic Development Summit, September 17-19, 2024, Boston, MA2024 Cantor Fitzgerald Global Healthcare Conference, September 17-19, 2024, New York, NY2024 Cell & Gene Meeting on the Mesa, October 7-9, 2024, Phoenix, AZ29th Annual Congress of the World Muscle Society, October 8-12, 2024, Prague, Czech Republic2024 Maxim Healthcare Summit, October 15-17, 2024, Virtual Conference Call and Webcast To participate in the conference call, please dial 1-800-717-1738 (Domestic/Toll-Free) or 1-646-307-1865 (International) and reference the conference ID: 30827. Participants can use guest dial-in numbers above and be answered by an operator or click the Call me™ link for instant telephone access. To participate via a webcast, please click here. A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the Company’s website. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as filed with the Securities and Exchange Commission on May 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com 858.727.1755 CAPRICOR THEAPEUTICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (UNAUDITED) Three months ended June 30, Six months ended June 30, 2024 2023 2024 2023 REVENUE Revenue$3,971,438 $3,917,467 $8,878,315 $6,904,163 TOTAL REVENUE 3,971,438 3,917,467 8,878,315 6,904,163 OPERATING EXPENSES Research and development 12,504,769 8,817,389 23,605,782 16,478,908 General and administrative 3,057,888 2,847,337 7,129,654 6,357,222 TOTAL OPERATING EXPENSES 15,562,657 11,664,726 30,735,436 22,836,130 LOSS FROM OPERATIONS (11,591,219) (7,747,259) (21,857,121) (15,931,967) OTHER INCOME (EXPENSE) Investment income 591,437 380,680 1,063,266 797,122 TOTAL OTHER INCOME (EXPENSE) 591,437 380,680 1,063,266 797,122 NET LOSS (10,999,782) (7,366,579) (20,793,855) (15,134,845) OTHER COMPREHENSIVE INCOME (LOSS) Net unrealized gain (loss) on marketable securities (152,714) 84,707 (80,826) 74,449 COMPREHENSIVE LOSS$(11,152,496) $(7,281,872) $(20,874,681) $(15,060,396) Net loss per share, basic and diluted$(0.35) $(0.29) $(0.66) $(0.60)Weighted average number of shares, basic and diluted 31,841,964 25,335,342 31,598,296 25,291,591 CAPRICOR THEAPEUTICS, INC. SUMMARY BALANCE SHEETS June 30, 2024(unaudited) December 31, 2023Cash, cash equivalents and marketable securities $29,462,030 $39,487,703 Total assets $38,277,453 $58,734,327 Total liabilities $26,775,147 $36,132,860 Total stockholders' equity - 31,983,927 and 31,148,320 common shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively 11,502,306 22,601,467 Total liabilities and stockholders' equity $38,277,453 $58,734,327