Hyperargininemia

-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

–Phase 3, HOPE-3 Trial (Cohort A) of CAP-1002 in Duchenne Muscular Dystrophy Fully Enrolled; On Track to Report Top-Line Data in Q4 2024– –Positive Type-B CMC FDA Meeting held in Q1; Company Aligned with FDA on Demonstration of Non-Clinical Comparability, A Major Milestone on Path to CMC Clearance for BLA– –Received First Milestone Payment of $10 Million under U.S. Distribution and Commercialization Agreement with Nippon Shinyaku in Q1– –Recent FDA Feedback Supports Requests for a Pre-BLA Meeting and Subsequent Rolling BLA Submission at Upcoming Type-B Clinical FDA Meeting in May 2024– –Plan to Report 3-Year Data from HOPE-2 Open-Label Extension (OLE) Trial in Q2 2024– –Conference Call and Webcast Today at 4:30 p.m. ET– SAN DIEGO, May 13, 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 first quarter ended March 31, 2024 and provided a corporate update. “Capricor continues to make tremendous progress across our pipeline marked by significant advancements in our CAP-1002 cell therapy program for the treatment of Duchenne muscular dystrophy (DMD),” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “With enrollment completed in Cohort A of our Phase 3 pivotal trial, we further aligned with the U.S. Food and Drug Administration (FDA) on key CMC (chemistry manufacturing and controls) deliverables necessary for the filing of a Biologics License Application (BLA) including the establishment of non-clinical comparability, a major milestone on the path to clearance for a BLA submission. We are appreciative of the FDA’s continued guidance under our RMAT designation and we will provide further updates on our plans as they become available. Furthermore, we continue to work diligently with our partner, Nippon Shinyaku (U.S. subsidiary: NS Pharma, Inc.) as we prepare for the potential launch of CAP-1002. Looking ahead, later this quarter, we plan to meet with FDA to continue to discuss options for potential expedited approval pathways as well as announce the 3-year HOPE-2 OLE results.” Dr. Marbán, continued, “We continue our efforts to advance our proprietary StealthX™ exosome platform to leverage exosomes for our vaccine program as well as for therapeutic development. This quarter, we announced a collaboration for our exosome-based multivalent vaccine for the prevention of SARS-CoV-2 with the National Institute of Allergy and Infectious Diseases (NIAID) where they will conduct and fully fund a Phase 1 clinical trial, subject to regulatory approval. We continue to remain focused on advancing this program through partnerships and other non-dilutive sources of funding.” First Quarter 2024 and Recent Operational Highlights CAP-1002 Duchenne Muscular Dystrophy Program: CAP-1002 is an investigational cell therapy in Phase 3 development for the treatment of DMD. CAP-1002 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. HOPE-3, our Phase 3 study, is a multi-center, randomized, double-blind, placebo-controlled clinical trial comprised of two cohorts evaluating the safety and efficacy of CAP-1002 in participants with DMD and impaired skeletal muscle function. The trial is being conducted in the United States. Approximately 102 eligible study subjects will participate in this dual-cohort study (Cohort A and B). CAP-1002 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 CAP-1002 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. Enrollment has been completed for our HOPE-3 Phase 3 (Cohort A) clinical trial which enrolled 61 subjects randomized to either CAP-1002 or placebo in a 1:1 ratio. Reported a positive outcome from the interim futility analysis for Cohort A which triggered the first milestone payment of $10.0 million received in the first quarter of 2024 under our U.S. Distribution and Commercialization Agreement with Nippon Shinyaku. Announced a positive outcome from a Type-B CMC FDA meeting held in the first quarter of 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 will now allow for the use of CAP-1002 drug product manufactured at our San Diego manufacturing facility upon potential product approval (subject to approval of the facility). Granted a Type-B clinical FDA meeting scheduled to be held in May 2024 to continue discussing our pathway to BLA. The FDA advised us to include discussion and a request for a pre-BLA meeting and rolling BLA schedule at this meeting. We also plan to share with FDA our HOPE-2 OLE 3-year safety and efficacy data. Announced further specifics under our U.S. Distribution and Commercialization Agreement with Nippon Shinyaku. Capricor will receive a meaningful mid-range double-digit share of product revenue. For clarity, mid-range falls in between 30-50%, which sum will be offset by the amounts paid to us as the transfer price for the purchase of the product. As previously disclosed, there is an additional $90.0 million in potential milestone payments up to the time of approval which are triggered upon certain regulatory-based achievements. Following potential approval, there is an additional $605.0 million in potential milestones payments which may be payable to Capricor based on various sales-based targets being met. Announced we are on track to enroll 44 U.S. based patients by the end of second quarter in HOPE-3 (Cohort B) clinical trial, however at this time, we are evaluating various options, with one of such options being the opportunity for an expansion of Cohort B to include European patients. Announced the scale-up of manufacturing capacity of CAP-1002 in our new San Diego facility, intended for commercial use, subject to regulatory approval. This facility was designed to be a versatile and cost-effective way to bring CAP-1002 to market efficiently. Currently, our San Diego facility is fully operational, staffed and producing doses for clinical use. Along with BLA readiness activities, we are also actively preparing for commercial runs. Presented at H.C. Wainwright 2nd Annual Cell Therapy Conference. Presented at the Cantor Fitzgerald’s Muscular Dystrophy Symposium. Presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference sharing the previously presented positive 24-month results from our HOPE-2 OLE study. Key results included skeletal muscle function as measured by the Performance of the Upper Limb (PUL v2.0) showed a mean PUL v2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7 point decline on average observed over 24-months in the placebo patient group. Additionally, CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function Presented at the Parent Project Muscular Dystrophy (PPMD) Cardiac Workshop III. Capricor was featured in a panel on industry perspectives on cardiac monitoring in DMD clinical trials. StealthX™ Exosome Platform: Exosomes are membrane-bound extracellular vesicles which are secreted by most cells and contain characteristic lipids, proteins and nucleic acids such as mRNA and microRNAs. 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. Announced that 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. As part of Project NextGen, NIAID, part of the National Institutes of Health, will conduct and fund a Phase 1 clinical trial with our StealthX™ vaccine. Under the terms of the collaboration, Capricor will supply the investigational product and NIAID's Division of Microbiology and Infectious Diseases (DMID) will conduct the trial. If NIAID finds that our StealthX™ vaccine meets its criteria for safety and efficacy, they may consider our program for a funded Phase 2. Currently, in collaboration with an undisclosed pharmaceutical company, we are also investigating the therapeutic application of our StealthX™ exosome platform. Presented data in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting. The findings highlight 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. The data from this preclinical study further characterize our StealthX™ exosome platform. Presented data at the International Society of Extracellular Vesicles (ISEV) Annual Meeting 2024. Highlights from the abstract included data showing targeted cargo delivery to mouse lower limbs by exosomes carrying a muscle targeting moiety by intravenous injection. Anticipated Upcoming Milestones CAP-1002 DMD Program Plan to have a Type-B clinical FDA meeting in May 2024 to discuss requests for a pre-BLA meeting and rolling BLA schedule. Additionally, Capricor plans to share with FDA its HOPE-2 OLE 3-year safety and efficacy data at this meeting. Plan to report 3-year HOPE-2 OLE data in the second quarter of 2024. Plan to announce further updates with respect to next steps for HOPE-3 (Cohort B), when available. 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 commercialization of CAP-1002 in DMD. StealthX™Exosome Platform Plan to provide updates on our NIAID collaboration for our StealthX™ vaccine as they become available. NIAID plans to initiate the Phase 1 clinical trial in late 2024, subject to regulatory approval. Continue to explore opportunities for partnerships and non-dilutive sources of funding to support advancement of our StealthX™ exosome platform technology. First Quarter 2024 Financial Results Cash position: Cash, cash equivalents and marketable securities totaled approximately $39.9 million as of March 31, 2024 compared to approximately $39.5 million as of December 31, 2023. In the first quarter of 2024, the Company received $10.0 million from the first milestone payment under our U.S. Distribution and Commercialization Agreement with Nippon Shinyaku and raised approximately $2.3 million in net proceeds through issuances of common stock at an average price of approximately $5.33 per share under its at-the-market offering program. Revenues: Revenues for the first quarter of 2024 were approximately $4.9 million compared with approximately $3.0 million for the first quarter 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: Operating expenses for the first quarter of 2024 were approximately $15.2 million compared with approximately $11.2 million for the first quarter of 2023. Net loss: The Company reported a net loss of approximately $9.8 million, or $0.31 per share, for the first quarter of 2024, compared to a net loss of approximately $7.8 million, or $0.31 per share, for the first quarter 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 its 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: CureDuchenne 2024 FUTURES National Conference, May 23-26, 2024, Orlando International Society for Cell & Gene Therapy (ISCT) 2024 Meeting, May 29-June 1, 2024, Vancouver BIO International Convention 2024, June 3-6, 2024, San Diego Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference, June 27-29, 2024, Orlando Conference Call and Webcast To participate in the conference call, please dial 1-888-886-7786 (Domestic/Toll-Free) or 1-416-764-8658 (International) and reference the conference ID: 01529679. 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, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 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. 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 a partnership for the exclusive commercialization and distribution of 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. CAP-1002 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 Officer abergmann@capricor.com 858.727.1755 CAPRICOR THERAPEUTICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (UNAUDITED) Three months ended March 31, 2024 2023 REVENUE Revenue $ 4,906,877 $ 2,986,696 TOTAL REVENUE 4,906,877 2,986,696 OPERATING EXPENSES Research and development 11,101,013 7,661,519 General and administrative 4,071,766 3,509,885 TOTAL OPERATING EXPENSES 15,172,779 11,171,404 LOSS FROM OPERATIONS (10,265,902 ) (8,184,708 ) OTHER INCOME (EXPENSE) Investment income 471,829 416,442 TOTAL OTHER INCOME (EXPENSE) 471,829 416,442 NET LOSS (9,794,073 ) (7,768,266 ) OTHER COMPREHENSIVE INCOME (LOSS) Net unrealized gain (loss) on marketable securities 71,888 (10,258 ) COMPREHENSIVE LOSS $ (9,722,185 ) $ (7,778,524 ) Net loss per share, basic and diluted $ (0.31 ) $ (0.31 ) Weighted average number of shares, basic and diluted 31,354,629 25,247,354 CAPRICOR THERAPEUTICS, INC. SUMMARY BALANCE SHEETS March 31, 2024 (unaudited) December 31, 2023 Cash, cash equivalents and marketable securities $ 39,917,078 $ 39,487,703 Total assets $ 49,444,442 $ 58,734,327 Total liabilities $ 31,009,504 $ 36,132,860 Total stockholders' equity – 31,600,183 and 31,148,320 common shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively 18,434,938 22,601,467 Total liabilities and stockholders' equity $ 49,444,442 $ 58,734,327

-Preclinical findings show exosome-based therapeutic approach for the treatment of arginase-1 deficiency (ARG1-D)- -Presentation to be featured in an oral session being held on May 9, 2024- SAN DIEGO, May 09, 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 that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7–11, 2024. The findings highlight 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. “The findings from this preclinical study have further characterized our StealthX™ exosome platform and provide a novel approach for exosome-based enzyme-replacement therapies which aim to replace a deficient or absent enzyme,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We believe our StealthX™ platform provides an opportunity with potentially broad applications and our data continues to support the concept of exosomes as a suitable delivery vehicle for a variety of payloads. We continue to focus our exosome platform in two main areas, the development of therapeutics and vaccines, and our goal is to leverage partnership opportunities to further expand and advance these opportunities.” Study Overview and Findings: In this study, exosomes were engineered to express human Arg1 enzyme inside of the exosomes and were evaluated for their in vitro functionality as an approach for the potential treatment of ARG1-D.Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro. In addition, the Arg1-exosomes were capable of delivering the Arg1 protein into 293F and HepG2 cells in a time and dose-dependent manner, contrary to human recombinant Arg1 protein alone at the same or higher dose.The data suggests that the Arg1-exosomes were able to not only catalyze arginine efficiently at a lower delivered dose to cells in vitro but also protect the Arg1 protein to be delivered into target cells and retain its enzymatic activity in cells.Taken together, Capricor’s StealthX™ platform of engineered Arg1-exosomes as tested in vitro have the potential to serve as an enzyme replacement therapy to deliver Arg1 to hepatocytes and possibly have potential clinical benefits for the treatment of ARG1-D. Presentation details: Title: Exosome-mediated intracellular delivery of arginase-1: a potential application for the treatment of arginase-1 deficiencyOral session: Exosomes, Virus like Particles and LNPsPresenting author: Li-En Hsieh, Ph.D., Principal ScientistDate: May 9, 2024 at 4:45 p.m. ETAbstract number: 223 The abstract will be made available on the publications section of the Capricor website following the conclusion of the meeting. 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, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 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. 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 a partnership for the exclusive commercialization and distribution of 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. CAP-1002 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