Capricor Therapeutics Reports Second Quarter 2024 Financial Results and Provides Corporate Update -Recently Held Positive Pre-BLA Meeting with FDA with Aim to Accelerate Approval Pathway of Deramiocel for the Treatment o

Capricor Therapeutics Reports Second Quarter 2024 Financial Results and Provides Corporate Update

-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, August 7, 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 Marb n, 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. Marb n 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.

StealthXTM 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.

Anticipated Upcoming Milestones

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.

The Company plans to participate in the following upcoming events:

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 meTM 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 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 Officer

CAPRICOR THERAPEUTICS, INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

CAPRICOR THERAPEUTICS, INC.

SUMMARY BALANCE SHEETS