DMD-Associated Dilated Cardiomyopathy

Medera and Cardiovascular Disease National Collaborative Enterprise (CADENCE) announce the initiation of Asia's first cardiac gene therapy clinical trial for heart failure, with Singapore being the first and only site outside of the United States (US).Heart failure is a global pandemic with an estimated 64.3 million cases worldwide. Asian patients tend to present with heart failure at a younger age (<15-20 years) compared to those in the Western population.Having received an Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA), Medera’s SRD-001, an adeno-associated virus (AAV)-based gene therapy candidate, is intended to treat patients with heart failure with reduced ejection fraction (HFrEF), which accounts for half of all heart failure cases worldwide.This site initiation of Singapore as the first outside of the U.S. for the multi-centre trial of SRD-001 also marks the first collaboration between Singapore’s two national heart centres, namely National Heart Centre, Singapore (NHCS) and National University Heart Centre, Singapore (NUHCS), and Tan Tock Seng Hospital, to jointly conduct a gene therapy trial for heart failure. SINGAPORE and BOSTON, Nov. 19, 2024 (GLOBE NEWSWIRE) -- Medera Inc. (“Medera”), a clinical-stage biotechnology company focused on targeting difficult-to-treat or incurable cardiovascular diseases using a range of next-generation gene- and cell-based approaches, and The Cardiovascular Disease National Collaborative Enterprise (CADENCE) of Singapore are pleased to announce the initiation of Asia’s first cardiac gene therapy clinical trial for heart failure in Singapore. Singapore is the first and only site selected outside of the United States (US) where the trials have been ongoing. Heart failure is a global challenge with 64.3 million patients worldwide. Singapore, or Asia in general, is facing a particularly high burden. About 2.5% of Singaporeans aged 60 years old or above have heart failure1. The condition is often the result of ischaemic heart disease, which contributes to around 20% of deaths in Singapore2. Asian patients also tend to present with heart failure at a younger age (<15-20 years) than those in the Western population, and often with more severe conditions which require intensive care, longer length of hospital stay, and a higher hospital mortality rate3. Having received an Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA), SRD-001, developed by Medera’s subsidiary Sardocor, is intended to treat patients with heart failure with reduced ejection fraction (HFrEF), a prevalent form of heart disease that accounts for half of all heart failure cases worldwide. A clinical update of the ongoing MUSIC-HFrEF (NCT0470384) Phase 1/2a trial of SRD-001 in the U.S. was featured in a late-breaking presentation at the American Society of Gene & Cell Therapy (ASGCT) in May 2024. The Phase 1/2a trial is expected to complete its enrollment, followed by an interim data readout and the randomized Phase 2b in 2025. Medera’s Sardocor has also obtained IND clearances, as well as Fast Track Designation (SRD-002; NCT06061549)3 and Orphan Drug Designation (SRD-003; NCT0622466)4, for heart failure with preserved ejection fraction (HFpEF, which accounts for the remaining half of all HF cases) and Duchenne muscular dystrophy-associated cardiomyopathy, respectively. SRD-001/2/3 employ an adeno-associated virus (AAV)-based vector system, delivered directly to cardiac ventricular muscle cells via Sardocor’s proprietary intracoronary infusion system. Furthermore, clinical dosages have been optimised by the human mini-Heart® technology developed by Novoheart, a pre-clinical subsidiary of Medera, in accordance with the FDA Modernisation Act 2.0. On November 21, 2023, the National Heart Centre Singapore (NHCS) announced a strategic partnership with Novoheart to develop Asia’s first bioengineered human heart-in-a-jar with heart failure for precision medicine5. An Agency for Science, Technology and Research (A*STAR) award of $5 million has been granted to foster this partnership. The MUSIC-HFrEF trial in Singapore involves the country’s two national heart centres, namely NHCS and National University Heart Centre, Singapore (NUHCS), and Tan Tock Seng Hospital. Roger Hajjar, MD, President and co-founder of Medera, expressed enthusiasm about the initiation of the Singapore clinical site, "Medera and Sardocor are very excited to initiate the Singapore clinical site as the first outside of the US, by deploying this cutting-edge technology for the first time ever to Asian heart failure patients. Sardocor’s unique regulated intracoronary delivery methodology is a minimally invasive procedure that targets the heart directly, designed to minimise drug dosage and therefore side-effects while preserving efficacy. While the Singapore trial’s initial focus is on congestive heart failure, this foundation will enable subsequent expansion to other cardiac indications." Adding to the significance of this trial, Derek Hausenloy, MBBS/PhD, Executive Director of CADENCE, and Director of the National Heart Research Institute Singapore at NHCS, remarked, "The launch of the SRD-001 trial in Singapore is a landmark moment for cardiovascular cell and gene therapy research in Asia. It underscores our commitment to integrate cutting-edge research and technologies to combat cardiovascular diseases. By bringing together the best of our national research capabilities and expertise, we aim to pave the way for innovative treatments that can significantly improve patient outcomes. This trial not only highlights Singapore’s position as a leader in cardiovascular research but also showcases our potential to contribute to the global fight against heart failure." “Medera is extremely delighted to partner with Singapore’s cardiac experts in its first precision medicine efforts in Asia to comprehensively cover important areas from mini-Heart®-based drug discovery all the way to clinical gene therapy starting with SRD-001 for the benefit of the large number of Asian heart patients,” said Ronald Li, PhD, CEO and co-founder of Medera. “We are pleased that Medera is setting yet another milestone in further expanding its international footprints. I am confident that the potential merger between KVAC and Medera will provide a platform for efficiently assessing capital from global markets thereby accelerating the various exciting clinical programs for heart patients worldwide,” said Kenneth KC Wong, Chairman and CEO of KVAC. On September 5, 2024, Medera and Keen Vision Acquisition Corporation ("KVAC"), announced they had entered into a definitive merger agreement. About MederaMedera (www.medera.bio) is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat or currently incurable diseases with significant unmet needs, utilizing next-generation gene and cell-based approaches in combination with bioengineered human-based technology (including the mini-Heart platform). Medera operates via the two preclinical and clinical business units, Novoheart and Sardocor, respectively. Novoheart capitalizes on the world’s first and award-winning “mini-Heart” Technology for revolutionary disease modelling and drug discovery, uniquely enabling the modelling of human-specific diseases and discovery of therapeutic candidates free from species-specific differences in accordance to the FDA Modernization Act 2.0. Novoheart's versatile technology platform provides a range of state-of-the-art automation hardware and software as well as screening services, for human-specific disease modelling, therapeutic target discovery and validation, drug toxicity and efficacy screening, and dosage optimization carried out in the context of healthy and/or diseased human heart chambers and tissues. Global pharmaceutical and academic leaders are using Novoheart's technology platform their drug discovery and development purposes. The Novoheart platform has facilitated and accelerated the development of Sardocor's lead therapeutic candidates that are currently in clinical trials. Sardocor is dedicated to the clinical development of novel next-generation therapies for Medera. Leveraging Novoheart’s human-based drug discovery and validation platforms, Sardocor aims to expedite drug development and regulatory timelines for its gene and cell therapy pipeline. Sardocor has received Investigational New Drug (IND) clearances from the FDA for three ongoing AAV-based cardiac gene therapy clinical trials targeting Heart Failure with Reduced Ejection Fraction (HFrEF), Heart Failure with Preserved Ejection Fraction (HFpEF) with the Fast Track Designation, and Duchenne Muscular Dystrophy-induced Cardiomyopathy (DMD-CM) with the Orphan Drug Designation. Additionally, Sardocor's pipeline includes four preclinical gene therapy and three preclinical small molecule candidates targeting various cardiac, pulmonary, and vascular diseases. About CADENCEEstablished in February 2023, the Cardiovascular Disease National Collaborative Enterprise (CADENCE) is a national clinical translational programme aimed at synergising cardiovascular research and technology capabilities across Singapore. Its goal is to integrate existing research capabilities, and bring together the strongest basic, clinical, and translational talent and expertise across Singapore and institutions, to establish a focused and impactful national cardiovascular disease research and translational programme with world-class peaks of excellence. To achieve this, CADENCE has established four national joint platforms focused on data science, clinical trials, and artificial intelligence/digital health, together with a business intelligence and development unit to attract industry collaborations/external investments. CADENCE is a programme of the Consortium for Clinical Research and Innovation, Singapore (CRIS). CADENCE is supported by the Singapore Ministry of Health through the National Medical Research Council (NMRC) Office, MOH Holdings Pte Ltd under the NMRC RIE2025 National Clinical Translational Programme Funding Initiative (MOH-001277). For more information, please visit https://cadence-cvd.sg Forward-Looking StatementsCertain statements included in this press release are not historical facts but are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release are forward-looking statements. Any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are also forward-looking statements. In some cases, you can identify forward-looking statements by words such as "estimate," "plan," "project," "forecast," "intend," "expect," "anticipate," "believe," "seek," "strategy," "future," "opportunity," "may," "target," "should," "will," "would," "will be," "will continue," "will likely result," "preliminary," or similar expressions that predict or indicate future events or trends or that are not statements of historical matters, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements include, without limitation, KVAC's, Medera's, or their respective management teams' expectations concerning the outlook for their or Medera's business, productivity, plans, and goals for future operational improvements and capital investments, operational performance, future market conditions, or economic performance and developments in the capital and credit markets and expected future financial performance, including expected net proceeds, expected additional funding, the percentage of redemptions of KVAC's public shareholders, growth prospects and outlook of Medera' operations, individually or in the aggregate, including the achievement of project milestones, commencement and completion of commercial operations of certain of Medera's projects, as well as any information concerning possible or assumed future results of operations of Medera. Forward-looking statements also include statements regarding the expected benefits of the transactions contemplated by the merger ("Transaction"). The forward-looking statements are based on the current expectations of the respective management teams of Medera and KVAC, as applicable, and are inherently subject to uncertainties and changes in circumstance and their potential effects. There can be no assurance that future developments will be those that have been anticipated. These forward-looking statements involve a number of risks, uncertainties or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to, (i) the risk that the Transaction may not be completed in a timely manner or at all, which may adversely affect the price of KVAC's securities; (ii) the risk that the Transaction may not be completed by KVAC's business combination deadline and the potential failure to obtain an extension of the business combination deadline if sought by KVAC; (iii) the failure to satisfy the conditions to the consummation of the Transaction, including the adoption of the Merger Agreement by the shareholders of KVAC and the receipt of certain regulatory approvals; (iv) market risks; (v) the occurrence of any event, change or other circumstance that could give rise to the termination of the Merger Agreement; (vi) the effect of the announcement or pendency of the Transaction on Medera's business relationships, performance, and business generally; (vii) the outcome of any legal proceedings that may be instituted against Medera or KVAC related to the Merger Agreement or the Transaction; (viii) failure to realize the anticipated benefits of the Transaction; (ix) the inability to maintain the listing of KVAC's securities or to meet listing requirements and maintain the listing of Medera's securities on Nasdaq; (x) the inability to implement business plans, forecasts, and other expectations after the completion of the Transaction, identify and realize additional opportunities, and manage its growth and expanding operations; (xi) risks related to Medera's ability to develop, license or acquire new therapeutics; (xii) the risk that Medera will need to raise additional capital to execute its business plan, which may not be available on acceptable terms or at all; (xiii) the risk of product liability or regulatory lawsuits or proceedings relating to Medera's business; (xiv) uncertainties inherent in the execution, cost, and completion of preclinical studies and clinical trials; (xv) risks related to regulatory review, and approval and commercial development; (xvi) risks associated with intellectual property protection; (xvii) Medera's limited operating history and risk that it may never successfully commercialise its products; (xviii) Medera expects to continue to incur significant losses and may never achieve or maintain profitability; and (xix) the risk that additional financing in connection with the Transaction may not be raised on favorable terms. The foregoing list is not exhaustive, and there may be additional risks that neither KVAC nor Medera presently knows or that KVAC and Medera currently believe are immaterial. You should carefully consider the foregoing factors, any other factors discussed in this press release and the other risks and uncertainties described in the "Risk Factors" section of KVAC's Annual Report on Form 10-K for the year ended December 31, 2023, which was filed with the SEC on March 29, 2024, the risks to be described in the registration statement, which will include a preliminary proxy statement/prospectus, and those discussed and identified in filings made with the SEC by KVAC from time to time. Medera and KVAC caution you against placing undue reliance on forward-looking statements, which reflect current beliefs and are based on information currently available as of the date a forward-looking statement is made. Forward-looking statements set forth in this press release speak only as of the date of this press release. Neither Medera nor KVAC undertakes any obligation to revise forward-looking statements to reflect future events, changes in circumstances, or changes in beliefs. In the event that any forward-looking statement is updated, no inference should be made that Medera or KVAC will make additional updates with respect to that statement, related matters, or any other forward-looking statements. Any corrections or revisions and other important assumptions and factors that could cause actual results to differ materially from forward-looking statements, including discussions of significant risk factors, may appear, up to the consummation of the Transaction, in KVAC's public filings with the SEC, and which you are advised to review carefully. Important Information for Investors and Shareholders In connection with the Transaction, KVAC and Medera filed a registration statement with the SEC, which includes a prospectus with respect to the securities to be issued in connection with the Transaction and a proxy statement to be distributed to holders of KVAC's common shares in connection with KVAC's solicitation of proxies for the vote by KVAC's shareholders with respect to the Transaction and other matters to be described in the Registration Statement (the "Proxy Statement"). After the SEC declares the registration statement effective, KVAC plans to mail copies to shareholders of KVAC as of a record date to be established for voting on the Transaction. This press release does not contain all the information that should be considered concerning the Transaction and is not a substitute for the registration statement, Proxy Statement or for any other document that KVAC may file with the SEC. Before making any investment or voting decision, investors and security holders of KVAC are urged to read the registration statement and the Proxy Statement, and any amendments or supplements thereto, as well as all other relevant materials filed or that will be filed with the SEC in connection with the Transaction as they become available because they will contain important information about, Medera, KVAC and the Transaction. Investors and security holders will be able to obtain free copies of the registration statement, the Proxy Statement and all other relevant documents filed or that will be filed with the SEC by KVAC through the website maintained by the SEC at www.sec.gov. In addition, the documents filed by KVAC may be obtained free of charge from KVAC's website at https://www.kv-ac.com or by directing a request to info@kv-ac.com. The information contained on, or that may be accessed through, the websites referenced in this press release is not incorporated by reference into, and is not a part of, this press release. Participants in the Solicitation KVAC, Medera and their respective directors, executive officers and other members of management and employees may, under the rules of the SEC, be deemed to be participants in the solicitations of proxies in connection with the Transaction. For more information about the names, affiliations and interests of KVAC's directors and executive officers, please refer to KVAC's annual report on Form 10-K filed with the SEC on March 29, 2024, which can be found at https://www.sec.gov/ix?doc=/Archives/edgar/data/1889983/000121390024027973/ea0201104-10k_keenvision.htm and registration statement, Proxy Statement and other relevant materials filed with the SEC in connection with the Transaction when they become available. Additional information regarding the participants in the proxy solicitation and a description of their direct and indirect interests, which may, in some cases, be different than those of KVAC's shareholders generally, will be included in the registration statement and the Proxy Statement and other relevant materials when they are filed with the SEC when they become available. Shareholders, potential investors and other interested persons should read the registration statement and the Proxy Statement and other such documents carefully, when they become available, before making any voting or investment decisions. You may obtain free copies of these documents from the sources indicated above. No Offer or Solicitation This communication shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities in the Transaction shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended. Contacts: Investor RelationsStephanie CarringtonICR HealthcareStephanie.Carrington@icrhealthcare.com(646) 277-1282 Media RelationsSean LeousICR HealthcareSean.Leous@icrhealthcare.com(646) 866-4012 1 SingCLOUD Data 2012-2021.2 https://www.moh.gov.sg/resources-statistics/singapore-health-facts/principal-causes-of-death 3 https://www.ktalnews.com/business/press-releases/ein-presswire/688652471/mederas-sardocor-announces-fast-track-designation-and-dosing-of-3-patients-in-first-in-human-hfpef-gene-therapy-trial/4 https://www.kxan.com/business/press-releases/ein-presswire/691834036/mederas-sardocor-granted-fda-orphan-drug-designation-for-cardiomyopathy-secondary-to-dmd-associated-cardiomyopathy/5 https://www.singhealthdukenus.com.sg/news/research/national-heart-centre-singapore-partners-mederas-novoheart-to-develop-asias-first-bioengineered-human-heart-in-a-jar-with-heart-failure-for-precision-medicine

Announced intent to file a biologics license application (BLA) for deramiocel to treat all patients with DMD-cardiomyopathy Rolling BLA submission initiated in October 2024 with full submission expected by year-end 2024Company anticipates potential PDUFA date in second half of 2025Announced signing of binding term sheet with Nippon Shinyaku for European expansion and commercialization of deramiocel; potential milestones from combined agreements would total approximately $1.5 billion payable to Capricor Reported positive long-term data from HOPE-2 OLE trial at 2024 World Muscle Society CongressStealthX™ exosome platform expanded to include PMO loading and targeting for the treatment of DMD presented at AAEV Annual MeetingCompleted public offering of common stock for gross proceeds of approximately $86 million; cash runway is expected to support current planned operations into 2027Capricor to host conference call and webcast today at 4:30 p.m. ET SAN DIEGO, Nov. 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 third quarter ended September 30, 2024 and provided a corporate update. “This has been a transformational quarter for Capricor as we move towards potential commercialization of deramiocel for the treatment of DMD. We have commenced the submission of our BLA which we expect to be complete by year end and we have significantly strengthened our balance sheet in order to scale up manufacturing as we anticipate a strong launch, pending FDA approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “In addition, we continue to advance our proprietary StealthX™ platform technology as part of our long-term strategy to leverage exosomes as vehicles for targeted delivery of payloads for therapeutic development. We continue to explore partnership opportunities and other non-dilutive sources of funding to advance this program.” Recent Updates and Upcoming Milestones Deramiocel DMD Program: Deramiocel is an investigational cell therapy in late-stage development for the treatment of DMD. Deramiocel for the treatment of DMD has received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT) from the FDA in the U.S. In addition, if deramiocel is approved, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on our previous receipt of a rare pediatric disease designation. Based on FDA feedback and following Capricor’s recent pre-BLA meeting in August, Capricor initiated the rolling BLA submission in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected to be complete by year end 2024. The BLA submission will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 open label extension (OLE) trials compared to patient-level natural history data. To support potential label expansion to treat DMD, Capricor plans to provide clinical data on skeletal muscle myopathy by combining Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was originally planned to occur in the fourth quarter of 2024. Furthermore, if required, the HOPE 3 study may also potentially support ex-U.S. marketing authorizations. Currently, Capricor has initiated regulatory activities in Europe and Japan and will be working with the various regional health authorities to develop the most efficient path forward for regulatory approval of deramiocel in these regions. Capricor is actively working towards expansion of our commercial manufacturing capacity and throughput. In addition, Capricor is exploring expansion opportunities for its GMP production for additional capacity that may be necessary to meet product demand in the U.S. and other regions. Capricor signed a binding term sheet with Nippon Shinyaku for European expansion and commercialization of deramiocel for the treatment of DMD. The potential transaction covered by the term sheet would be similar to the existing Commercialization and Distribution Agreements with Nippon Shinyaku in the U.S. and Japan with an opportunity for increased global product reach. Subject to finalization of the Definitive Agreement, Capricor will receive an upfront payment of $20 million along with potential additional development and sales-based milestone payments to Capricor of up to $715 million and a double-digit share of product revenue. In September 2024, Nippon Shinyaku purchased approximately $15 million of Capricor common stock at a 20% premium to the 60-day volume weighted average price of Capricor’s common stock.Capricor presented positive 3-year safety and efficacy results from its ongoing HOPE-2 OLE in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society. The 3-year data from HOPE-2 OLE demonstrated improvements in multiple measures of cardiac function, including left ventricular ejection fraction (LVEF%), as well as indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes.In addition to the cardiac data, patients demonstrated a statistically and clinically relevant 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. StealthX™Exosome Platform: Exosomes are membrane-bound extracellular vesicles and contain lipids, proteins and nucleic acids. They act as messengers to regulate the functions of neighboring or distant cells. Capricor is developing our engineered exosome technology using our proprietary StealthX™ platform focused on the areas of targeted therapeutics and vaccines to potentially treat and prevent a diverse array of diseases. The Company’s proprietary StealthX™ exosome-based 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 initiative, Capricor will supply its vaccine candidate for clinical use and NIAID's Division of Microbiology and Infectious Diseases will conduct the trial.Currently, manufacturing is underway for our StealthX™ vaccine with current plans to deliver it to NIAID in the first quarter of 2025.NIAID plans to initiate the trial in the first quarter of 2025, and Capricor expects that it will have preliminary data available in the second quarter of 2025, subject to FDA approval of the NIAID’s IND.If NIAID finds that our StealthX™ vaccine meets its criteria for safety and efficacy, they may consider funding our program for a Phase 2 study. Capricor presented preclinical data at the 2024 American Association of Extracellular Vesicles (AAEV) Annual Meeting highlighting a potential exosome-based approach by delivering phosphorodiamidate morpholino oligomers (PMOs) for the treatment of DMD. Third Quarter 2024 Financial Results Cash position: Cash, cash equivalents and marketable securities totaled approximately $85.0 million as of September 30, 2024 compared to $39.5 million as of December 31, 2023. In the third quarter of 2024, Capricor raised approximately $52.2 million in net proceeds through issuances of common stock at an average price of approximately $9.84 per share under its at-the-market (ATM) Program. Effective October 1, 2024, the ATM Program was closed and terminated. Additionally, on October 18, 2024, the Company completed a public offering of 5,073,800 shares of common stock and received gross proceeds of approximately $86.3 million before deducting underwriting discounts, commissions and offering expenses of approximately $5.5 million, for net proceeds of approximately $80.8 million. Revenues: Revenues for the third quarter of 2024 were approximately $2.3 million compared to $6.2 million for the third quarter of 2023. Additionally, revenues for the nine months ended September 30, 2024 and 2023 were approximately $11.1 million and $13.1 million, respectively. 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 third quarter of 2024 were approximately $15.3 million compared to $13.1 million for the third quarter of 2023. Total operating expenses for the nine months ended September 30, 2024 and 2023 were approximately $46.0 million and $35.9 million, respectively. Net loss: The Company reported a net loss of approximately $12.6 million, or $0.38 per share, for the third quarter of 2024, compared to a net loss of $6.4 million, or $0.25 per share, for the third quarter of 2024. Capricor reported a net loss of approximately $33.4 million, or $1.04 per share, and $21.5 million, or $0.85 per share, for the nine months ended September 30, 2024 and 2023, respectively. Financial Outlook: The Company believes that based on the current operating plan and financial resources, our available cash, cash equivalents and marketable securities will be sufficient to cover anticipated expenses and capital requirements into 2027. This expectation excludes any additional potential milestone payments under the 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 present at the following upcoming events: B. Riley Securities NextGen Tissue Delivery Modalities Summit, November 14, 2024, VirtualPiper Sandler 36th Annual Healthcare Conference, December 3-5, 2024, New York, NYOppenheimer Movers in Rare Disease Summit, December 12, 2024, New York, NY 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: 68076. 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 the event. 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. Capricor is also harnessing the power of its exosome technology, using its 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; potential future agreements; 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 June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 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 Media Contact:Raquel ConaKCSA Strategic Communications rcona@kcsa.com212.896.1204 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com858.727.1755 CAPRICOR THERAPEUTICS, INC.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(UNAUDITED) Three months ended September 30, Nine months ended September 30, 2024 2023 2024 2023 REVENUE Revenue$2,261,642 $6,185,814 $11,139,956 $13,089,977 TOTAL REVENUE 2,261,642 6,185,814 11,139,956 13,089,977 OPERATING EXPENSES Research and development 11,807,867 10,028,964 35,413,649 26,507,872 General and administrative 3,463,655 3,021,450 10,593,308 9,378,672 TOTAL OPERATING EXPENSES 15,271,522 13,050,414 46,006,957 35,886,544 LOSS FROM OPERATIONS (13,009,880) (6,864,600) (34,867,001) (22,796,567) OTHER INCOME (EXPENSE) Investment income 453,152 479,380 1,516,418 1,276,502 Loss on disposal of fixed assets - (5,388) - (5,388) TOTAL OTHER INCOME (EXPENSE) 453,152 473,992 1,516,418 1,271,114 NET LOSS (12,556,728) (6,390,608) (33,350,583) (21,525,453) OTHER COMPREHENSIVE INCOME (LOSS) Net unrealized gain (loss) on marketable securities (58,766) (66,485) (139,592) 7,964 COMPREHENSIVE LOSS$(12,615,494) $(6,457,093) $(33,490,175) $(21,517,489) Net loss per share, basic and diluted$(0.38) $(0.25) $(1.04) $(0.85)Weighted average number of shares, basic and diluted 33,090,063 25,817,676 32,099,181 25,468,880 CAPRICOR THERAPEUTICS, INC.SUMMARY BALANCE SHEETS September 30, 2024 (unaudited) December 31, 2023Cash, cash equivalents and marketable securities $85,028,624 $39,487,703 Total assets $92,951,613 $58,734,327 Total liabilities $24,686,654 $36,132,860 Total stockholders' equity - 40,332,392 and 31,148,320 common shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively 68,264,959 22,601,467 Total liabilities and stockholders' equity $92,951,613 $58,734,327

NASHVILLE, Tenn., Nov. 6, 2024 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Cumberland is completing the FIGHT DMD™ trial, a multicenter, double-blind, placebo-controlled Phase II study investigating the pharmacokinetics, safety and efficacy of once daily oral Ifetroban in patients with DMD. Results will be announced later this year. "For Duchenne muscular dystrophy, a devastating genetic disorder affecting young boys, securing both Orphan Drug and Rare Pediatric Disease Designations for Ifetroban from the FDA is a critical step forward," said A.J. Kazimi, chief executive officer of Cumberland Pharmaceuticals. "These designations not only recognize the urgent need for effective treatments but also provide vital support to accelerate research and development. These important regulatory milestones represent hope for families and a pathway to bring transformative medicines to a vulnerable patient population more quickly and efficiently." The U.S. FDA grants Rare Pediatric Disease Designation to incentivize drug development for life-threatening diseases affecting less than 200,000 children in the U.S. As these diseases pose unique challenges to drug development, special focus is needed to provide therapeutic options to these children. Companies that receive approval for a drug or biologic with this designation may be eligible for a voucher which may be redeemed for priority review of a different product. Additionally, this voucher may be transferred or sold to another sponsor. Orphan Drug Designation is granted by the U.S. FDA to encourage development of new therapies for rare diseases or conditions affecting fewer than 200,000 people in the U.S. This designation offers sponsors multiple incentives, which include exemption from user fees, tax credits for qualified clinical trials and potential market exclusivity for seven years following approval of the product. About Duchenne Muscular Dystrophy DMD is a rare and fatal genetic disorder which affects about 1 in every 3,300 male births worldwide. Characterized by mutations in the gene responsible for producing dystrophin, DMD causes damage to the skeletal and cardiac muscle of DMD patients, with cardiomyopathy recognized as the primary cause of death. Ifetroban is being evaluated to treat the heart disease associated with DMD, which has received limited attention with current therapies. About Ifetroban Ifetroban is a potent and selective thromboxane-prostanoid receptor (TPr) antagonist. Ifetroban exhibits high affinity for TPr on many cell types including cardiomyocytes, platelets, vascular and airway smooth muscle, and fibroblasts, and lacks agonistic activity. In several preclinical models of muscular dystrophy, including limb-girdle and Duchenne, ifetroban prevented cardiac fibrosis and cardiac dysfunction and improved mortality. Cumberland previously announced the acquisition of the ifetroban program in collaboration with Vanderbilt University and Cumberland Emerging Technologies. Cumberland is also evaluating ifetroban for systemic sclerosis and pulmonary fibrosis. The ongoing FIGHTING FIBROSIS Trial is a multicenter, randomized placebo-controlled Phase II study in patients with idiopathic pulmonary fibrosis (IPF), a progressive interstitial lung disease. IPF is marked by inflammation and fibrosis of the lungs, resulting in rapidly declining lung function and reduced survival within five years of diagnosis. The study is evaluating the safety and efficacy of once daily oral Ifetroban for 52 weeks. About Cumberland Pharmaceuticals Cumberland Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the delivery of high-quality prescription brands to improve patient care. The Company develops, acquires, and commercializes brands for the hospital acute care, gastroenterology and rheumatology market segments. The Company's portfolio of FDA-approved brands includes: Acetadote® (acetylcysteine) Injection, for the treatment of acetaminophen poisoning; Caldolor® (ibuprofen) Injection, for the treatment of pain and fever; Vaprisol® (conivaptan) Injection, to raise serum sodium levels in hospitalized patients with euvolemic and hypervolemic hyponatremia; Vibativ® (telavancin) Injection, for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections; Kristalose® (lactulose) for Oral Solution, a prescription laxative, for the treatment of chronic and acute constipation; and Sancuso® (granisetron) Transdermal delivery system, for the treatment of nausea and vomiting associated with chemotherapy. For more information on Cumberland's approved products, including full prescribing information, please visit the individual product websites, links to which can be found on the Company's website . About Cumberland Emerging Technologies Cumberland Emerging Technologies, Inc. () is a joint initiative between Cumberland Pharmaceuticals Inc., Vanderbilt University, Launch Tennessee and WinHealth Pharma. The mission of CET is to bring biomedical technologies and products conceived at Vanderbilt and other regional research centers to the marketplace. CET helps manage the development and commercialization process for select projects and provides expertise on intellectual property, regulatory, manufacturing and marketing issues that are critical to successful new biomedical products. CET's Life Sciences Center, located in Nashville, Tennessee, provides laboratory space, equipment and infrastructure to early-stage life sciences companies. Forward-Looking Statements This press release contains forward-looking statements, which are subject to certain risks and reflect Cumberland's current views on future events based on what it believes are reasonable assumptions. No assurance can be given that these events will occur. As with any business, all phases of Cumberland's operations are subject to factors outside of its control, and any one or combination of these factors could materially affect Cumberland's results of operations. These factors include market conditions, competition, an inability of manufacturers to produce Cumberland's products on a timely basis or failure of manufacturers to comply with regulations applicable to pharmaceutical manufacturers, maintaining an effective sales and marketing infrastructure, natural disasters, public health epidemics, and other events beyond our control, as more fully discussed in the Company's most recent Form 10-K and subsequent 10-Qs as filed with the SEC. There can be no assurance that results anticipated by the Company will be realized or that they will have the expected effects. Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date hereof. The Company does not undertake any obligation to publicly revise these statements to reflect events after the date hereof. SOURCE Cumberland Pharmaceuticals Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED