CAPRICOR THERAPEUTICS, INC.
For the period ended Jun 30, 2025. Filed Aug 11, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Aug 11, 2025
CAPR$9.00+4.7%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-14.
0001558370-25-007770
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-11.
0001558370-25-011084
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 106 previous paragraphs and 124 current paragraphs.
68 unchanged; 24 reworded; 26 added; 10 removed; 4 previous and 6 current paragraphs rearranged.
Tables are not compared: 33 previous and 42 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (26)
Current report
Deramiocel for the Treatment of DMD: Regulatory and Clinical Update
Current report
Capricor’s lead clinical program is centered on deramiocel, an allogeneic cell therapy comprised of cardiosphere-derived cells (CDCs), for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy. CDCs are a unique population of cardiac cells derived from healthy donor hearts, and they exert their therapeutic effect through immunomodulatory, anti-inflammatory, pro-angiogenic, and anti-fibrotic mechanisms—primarily mediated by secreted exosomes enriched with bioactive microRNAs. These non-coding RNA species influence gene expression in immune and tissue-resident cells, promoting regeneration and reducing inflammation, in contrast to dystrophin-restoring approaches like exon-skipping or gene therapy.
Current report
DMD is a progressive, fatal genetic disorder that results in significant skeletal and cardiac muscle deterioration. Over time, the lack of functional dystrophin leads to cardiomyocyte death and fibrosis, ultimately progressing to heart failure—the leading cause of death in DMD patients. There are currently no approved therapies specifically targeting DMD-associated cardiomyopathy, representing a substantial unmet medical and commercial opportunity for deramiocel.
Current report
Regulatory Progress and BLA Pathway
Current report
In the third quarter of 2024, we conducted a pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) to discuss key components of our submission strategy, including the use of data from our HOPE-2 and HOPE-2 open label extension (OLE) trials, commercial manufacturing readiness and potential label considerations. Following additional discussions with the FDA, we completed our rolling BLA submission in December 2024. In early 2025, the FDA accepted our application for review, granted it Priority Review, and assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.
Current report
At the May 2025 mid-cycle review meeting, the FDA noted no significant deficiencies. In May 2025, the FDA conducted a Pre-License Inspection (PLI) of our San Diego manufacturing facility for deramiocel. The inspection concluded with a Form 483 containing several observations. We were recently informed by the FDA that they have reviewed and found acceptable all responses to the 483 observations noted in the PLI.
Current report
In July 2025, we received a Complete Response Letter (CRL) stating that the current BLA does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data. The CRL also cited certain outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section, many of which we had previously addressed but were not reviewed by the FDA due to timing of the issuance of the CRL. At this time, we have internally addressed the remaining CMC issues noted in the CRL and have prepared formal responses which we plan to submit with our response to the CRL.
Current report
We are now actively preparing a formal written response to the CRL and have scheduled a Type A meeting with the FDA for August 2025. Pending regulatory guidance, Capricor seeks to resubmit its BLA based on its existing dataset and with HOPE-3 data (expected to be available in the fourth quarter of 2025) potentially serving as supportive and confirmatory evidence.
Current report
Clinical Development Overview
Current report
Building on these results, we initiated the HOPE-2 OLE study, in which, at this time, 12 patients are continuing treatment with deramiocel. In June 2025, we reported positive four-year outcomes in the HOPE-2 OLE study demonstrating sustained preservation of cardiac function and a slowing in skeletal muscle decline. These findings further underscore the potential of deramiocel to alter disease progression. Clinical benefit was also observed when comparing treated patients to a natural history cohort, including statistically significant differences in PUL v2.0 scores and cardiac function metrics.
Current report
Our ongoing Phase 3 clinical trial, HOPE-3, is a 1:1 randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of deramiocel in approximately 104 boys with DMD. Participants receive four doses over 12 months, and the trial includes two manufacturing cohorts: Cohort A (Los Angeles facility) and Cohort B (San Diego facility). In the fourth quarter of 2023, the Data Safety Monitoring Board (DSMB) reviewed a pre-specified futility analysis and recommended that the study continue as planned.
Current report
We recently completed the 12-month treatment period in HOPE-3 and expect to report topline results in the fourth quarter of 2025, although the study remains blinded at present. In alignment with FDA feedback and our evolving regulatory strategy, we amended the study protocol to designate LVEF as the primary endpoint, shifting the focus to cardiac efficacy. This change reflects the FDA’s emphasis on well-controlled cardiac outcome measures in DMD based on FDA feedback in the CRL and may, if the results are positive and accepted by the FDA, meaningfully support our BLA. Skeletal muscle function will remain a key secondary endpoint, assessed via PUL v2.0, which captures upper limb strength across a spectrum of functional domains.
Current report
Additionally, if positive, HOPE-3 may support international regulatory filings, and we have initiated discussions with health authorities in Europe and Japan to define potential approval pathways.
Current report
Designations and Commercial Planning
Current report
We have entered into exclusive Commercialization and Distribution Agreements with Nippon Shinyaku for the United States and Japan. We aim to efficiently scale commercial efforts in key markets upon potential approval.
Current report
Exosome Platform
Current report
Extracellular vesicles, including exosomes and microvesicles, are nanoscale, membrane-enclosed particles secreted by most cells. These vesicles carry proteins, lipids, and nucleic acids (such as mRNA and microRNAs) and play a key role in intercellular communication. They signal by binding to membrane receptors or delivering their cargo directly into target cells, influencing functions such as inflammation, survival, proliferation, and tissue regeneration. Due to their small size, low immunogenicity, and ability to interact in native cellular language, exosomes represent a promising new class of therapeutic agents capable of modulating complex biological systems. As cell-free products, they offer advantages in storage, handling, and delivery similar to traditional biologics like monoclonal antibodies.
Current report
Preclinical data from our StealthX™ platform demonstrated rapid development of a recombinant protein based vaccine against SARS-CoV-2. Building on advancements in RNA and protein science, targeting, and scalable manufacturing, the platform has potential applications across infectious diseases, monogenic disorders, and other indications.
Current report
In 2024, Capricor was selected to participate in Project NextGen, an initiative led by the U.S. Department of Health and Human Services aimed at advancing next generation COVID-19 vaccines with broader and longer lasting protection. Under this program, NIAID is sponsoring a Phase 1 clinical trial of our StealthX™ vaccine. The trial will be conducted and overseen by NIAID’s Division of Microbiology and Infectious Diseases (DMID), with Capricor supplying the clinical material.
Current report
Following the clearance of the Investigational New Drug (IND) application by the FDA, the Phase 1 study initiated in August 2025. The trial includes several dosing arms initially focused on the spike (S) protein, with an additional planned arm which will utilize the nucleocapsid (N) protein, pending separate FDA clearance. If the vaccine meets NIAID’s criteria for safety and efficacy, the program may advance to a funded Phase 2 study.
Current report
As we advance our exosome platform, our strategy is to identify partners who can provide capital and development expertise to support clinical advancement across targeted indications.
Current report
R&D expenses for the three months ended June 30, 2025 increased by approximately $9.5 million, or 76%, compared to the three months ended June 30, 2024. The increase was primarily driven by the following:
Current report
R&D expenses for the six months ended June 30, 2025 increased by approximately $17.4 million, or 74%, compared to the six months ended June 30, 2024. The increase was primarily driven by the following:
Current report
G&A expenses for the three months ended June 30, 2025 increased by approximately $2.6 million, or 85%, compared to the three months ended June 30, 2024. The increase was primarily driven by the following:
Current report
G&A expenses for the six months ended June 30, 2025 increased by approximately $4.6 million, or 65%, compared to the six months ended June 30, 2024. The increase was primarily driven by the following:
Current report
Investment Income. Investment income for the three months ended June 30, 2025 and 2024 was approximately $1.8 million and $0.6 million, respectively. Investment income for the six months ended June 30, 2025 and 2024 was
Removed paragraphs (10)
Previous report
annual cost of care for patients with DMD is very high and increases with disease progression. There is no currently approved treatment for DMD-cardiomyopathy, therefore, we believe that DMD represents a significant market opportunity for our product candidate, deramiocel.
Previous report
Biologics License Application (“BLA”): In the third quarter of 2024, we held a pre-BLA meeting with FDA where we discussed our rolling BLA submission schedule, potential label expansion, plans for commercial manufacturing as well as other topics. Subsequent to this meeting, we held several additional meetings with FDA and announced our intent to file a BLA based on existing data from our Phase 2 HOPE-2 and HOPE-2 OLE trials compared to patient-level natural history data. We completed the full submission of the BLA in December 2024 and in the first quarter of 2025, we were informed by the FDA, they have accepted for review our BLA seeking full approval for deramiocel as a treatment for patients diagnosed with DMD cardiomyopathy. Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (“PDUFA”) target action date of August 31, 2025. In May 2025, we announced the completion of a mid-cycle review meeting with the FDA. During the meeting, the FDA stated that no significant deficiencies have been identified by the Review Committee. The FDA also confirmed its intent to hold an advisory committee meeting, although an official date has not yet been finalized.
Previous report
Additionally, we are currently conducting an open label extension (“OLE”) study of the HOPE-2 trial in which 12 patients have elected to continue treatment of deramiocel. We announced positive one-year and two-year results from this ongoing OLE study. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). The study remains ongoing and the three-year data 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 order to evaluate the relevance of the data to disease progression as well as the chronic and progressive nature of DMD where cardiac function can decline year over year, a natural history data set was used to compare the trajectory of those treated with deramiocel to standard of care. In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. Deramiocel treatment during the OLE portion of the study continues to yield a consistent safety profile and has been well-tolerated throughout the study.
Previous report
The primary outcome measure of the HOPE-3 study will be the Performance of the Upper Limb (“PUL”) v2.0, a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) functions, with a conceptual framework reflecting weakness progression in upper limb function.
Previous report
HOPE-3 will also measure various secondary endpoints including cardiac function assessments. To support potential label expansion to treat DMD, we plan to provide clinical data on skeletal muscle myopathy by combining Cohorts A and B of the HOPE-3 clinical trial to serve as a post-approval study. Furthermore, if necessary, the HOPE-3 study will also be supporting ex-U.S. marketing authorizations. Currently, we have initiated regulatory activities in Europe and Japan and will be working with the various health authorities to develop the most efficient path for regulatory approval of deramiocel in these regions.
Previous report
R&D expenses for the three months ended March 31, 2025 increased by approximately $7.8 million, or 70%, compared to the three months ended March 31, 2024. The increase was primarily driven by the following:
Previous report
G&A expenses for the three months ended March 31, 2025 increased by approximately $2.0 million, or 49%, compared to the three months ended March 31, 2024. The increase was primarily driven by the following:
Previous report
This increase was partially offset by a $0.1 million decrease in professional service expenses, primarily due to a decrease in business development related expenses.
Previous report
Grant Income
Previous report
The determination as to when income is earned is dependent on the language in each specific grant. Generally, we recognize grant income in the period in which the expense is incurred for those expenses that are deemed reimbursable under the terms of the grant. Grant income is due upon submission of reimbursement request. The transaction price varies for grant income based on the expenses incurred under the awards.
Reworded paragraphs (24)
Previous wording
To date, we have completed two promising clinical trials investigating deramiocel for DMD. Data from the first trial, a Phase I/II trial named HOPE-Duchenne, suggested improvements in skeletal and cardiac endpoints. In HOPE-2, a Phase II clinical trial conducted in the United States, deramiocel was used to treat patients with late-stage DMD. In March 2022, we announced that the final one-year results from HOPE-2 were published inThe Lancet showing that the trial met its primary efficacy endpoint of the mid-level dimension of the Performance of the Upper Limb (“PUL”) v1.2 (p=0.01) and additional positive endpoints of full PUL v2.0 (p=0.04) and a cardiac endpoint of left ventricular ejection fraction (p=0.002). deramiocel was generally safe and well-tolerated throughout the studies.
Current wording
To date, we have completed two clinical trials evaluating deramiocel in DMD patients. The Phase I/II HOPE-Duchenne study showed encouraging signals in both skeletal and cardiac endpoints. In our randomized, placebo-controlled Phase II HOPE-2 trial, deramiocel met its primary endpoint (mid-level PUL v1.2; p=0.01) and secondary endpoints, including full PUL v2.0 (p=0.04) and left ventricular ejection fraction (LVEF; p=0.002). These results were published in The Lancet in March 2022, and the therapy was generally safe and well-tolerated.
Previous wording
Phase 3 (HOPE-3) Clinical Trial: HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial comprised of two cohorts evaluating the safety and efficacy of deramiocel in participants with DMD and impaired skeletal muscle function who are on a stable regimen of systemic glucocorticoids. Non-ambulatory and ambulatory boys who meet eligibility criteria are randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12-months of the study. Approximately 105 eligible study subjects are currently enrolled in the dual-cohort study (comprised of Cohorts A and B). Cohort A uses product manufactured at our Los Angeles facility and Cohort B uses product manufactured at our San Diego facility. Subjects are randomized to either deramiocel or placebo in a 1:1 ratio. In the fourth quarter of 2023, we announced a positive outcome of the futility analysis for Cohort A of HOPE-3, which was reviewed by the Data Safety Monitoring Board (“DSMB”). This resulted in a favorable recommendation to continue the HOPE-3 trial as planned.
Current wording
Phase 3 HOPE-3 Clinical Trial and Next Steps
Previous wording
The regulatory pathway for deramiocel is supported by RMAT designation as well as orphan drug designation. In addition, if Capricor were to receive FDA marketing approval for deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (“PRV”) based on its previous receipt of a rare pediatric disease designation. Capricor retains full rights to the PRV, if received. Further, Capricor has entered into two Commercialization and Distribution Agreements with Nippon Shinyaku appointing Nippon Shinyaku as its exclusive distributor of deramiocel in the United States and Japan.
Current wording
Deramiocel has received RMAT and orphan drug designations, and, if approved, Capricor is eligible to receive a Priority Review Voucher (PRV) under its rare pediatric disease designation. Capricor retains full rights to the PRV, if granted.
Previous wording
We are focused on developing a precision-engineered exosome platform technology that has the ability to deliver defined sets of effector molecules that exert their effects through defined mechanisms of action. Aspects of our exosome pipeline have been supported through collaborations and alliances. Our collaborations and research around exosomes include the National Institutes of Health (“NIH”), the National Institute of Allergy and Infectious Diseases (“NIAID”), Johns Hopkins University (“JHU”), the Department of Defense (“DoD”), the U.S. Army Institute of Surgical Research (“USAISR”), and Cedars-Sinai Medical Center (“CSMC”). We have published preclinical data on our StealthX™ platform showing the rapid development of a recombinant protein-based vaccine for immunization and prevention against SARS-CoV-2, the virus causing COVID-19. Our platform builds on advances in fundamental RNA and protein science, targeting technology and manufacturing, providing us the opportunity to potentially build a broad pipeline of new therapeutic candidates. Recently, we were 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 providing broader and more durable protection for COVID-19. As part of Project NextGen, the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, will conduct a Phase 1 clinical study with our StealthX™ vaccine, subject to regulatory approval. At this time, manufacturing is underway for our StealthX™ vaccine and we have submitted an Investigational New Drug Application (“IND”) to the FDA, which is currently under review. At this time, NIAID is planning for regulatory approval in the third quarter of 2025 with the clinical study initiated soon thereafter. NIAID's Division of Microbiology and Infectious Diseases (“DMID”) would oversee the study. If NIAID finds that our StealthX™ vaccine meets its criteria for safety and efficacy, they may consider our program for a funded Phase 2. At this time, we are developing exosome-based vaccines and therapeutics for infectious diseases, monogenic diseases and other potential indications. Our current strategy is focused on securing partners who will provide capital and additional resources to enable us to bring this program into the clinic.
Current wording
Our focus is on developing a precision engineered exosome platform capable of delivering defined sets of effector molecules through well characterized mechanisms of action. Our exosome platform, has advanced through internal research and external collaborations, including with the National Institutes of Health (NIH), National Institute of Allergy and Infectious Diseases (NIAID), Johns Hopkins University, the Department of Defense (DoD), U.S. Army Institute of Surgical Research (USAISR), and Cedars-Sinai Medical Center.
Previous wording
As of March 31, 2025, we had cash, cash equivalents, and marketable securities totaling approximately $144.8 million. In the fourth quarter of 2024, we submitted our BLA to the FDA, which triggered our second milestone pursuant to the terms of our U.S. Distribution Agreement with Nippon Shinyaku. In January 2025, we received the $10.0 million milestone payment.
Current wording
As of June 30, 2025, we had cash, cash equivalents, and marketable securities totaling approximately $122.8 million. In the fourth quarter of 2024, we submitted our BLA to the FDA, which triggered our second milestone pursuant to the terms of our U.S. Distribution Agreement with Nippon Shinyaku. In January 2025, we received the $10.0 million milestone payment.
Previous wording
Due to our significant research and development expenditures, and general administrative costs associated with our operations, we have generated substantial operating losses in each period since our inception. Our net losses were $24.4 million and $9.8 million, for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $224.2 million. We expect to incur significant expenses and operating losses for the foreseeable future.
Current wording
Due to our significant research and development expenditures, and general administrative costs associated with our operations, we have generated substantial operating losses in each period since our inception. Our net losses were
Previous wording
Clinical Development Income. Clinical development income for the three months ended March 31, 2025 and 2024 was $0 and approximately $4.9 million, respectively. As of March 31, 2025, the Company has fully recognized $50.0 million in development milestone payments received from Nippon Shinyaku related to the Exclusive Commercialization and Distribution Agreement (the “U.S. Distribution Agreement”). The upfront payment of $30.0 million and the first milestone payment of $10.0 million was ratably recognized as revenue using a proportional performance method in relation to the completion of the HOPE-3 clinical trial (Cohort A) whereas the $10.0 million related to the second milestone payment was recognized as revenue at the point in time when the BLA was submitted in December 2024.
Current wording
Clinical Development Income. Clinical development income for the three months ended June 30, 2025 and 2024 was $0 and approximately $4.0 million, respectively. Clinical Development income for the six months ended June 30, 2025 and 2024 was approximately $0 and $8.9 million. As of June 30, 2025, the Company has fully recognized $50.0 million in development milestone payments received from Nippon Shinyaku related to the Exclusive Commercialization and Distribution Agreement (the “U.S. Distribution Agreement”). The upfront payment of $30.0 million and the first milestone payment of $10.0 million was ratably recognized as revenue using a proportional performance method in relation to the completion of the HOPE-3 clinical trial (Cohort A) whereas the $10.0 million related to the second milestone payment was recognized as revenue at the point in time when the BLA was submitted in December 2024.
Previous wording
Investment Income. Investment income for the three months ended March 31, 2025 and 2024 was approximately $0.7 million and $0.5 million, respectively. The increase in investment income for the three months ended March 31, 2025 as compared to the three months ended March 31, 2024 is due to a higher principal balance in our marketable securities, savings and money market fund accounts.
Current wording
approximately $2.5 million and $1.1 million, respectively. The increase in investment income for the three and six months ended June 30, 2025 as compared to the three and six months ended June 30, 2024 is due to a higher principal balance in our marketable securities, savings and money market fund accounts.
Previous wording
Deramiocel for the treatment of DMD– The expenses for our DMD program include costs for personnel, clinical, regulatory and manufacturing-related expenses, including expenses related to the scale-up for potential commercial scale manufacturing if our deramiocel product is approved. In 2025, we expect to spend approximately $40.0 million to $50.0 million primarily consisting of CMC expansion, product inventory buildout, clinical, regulatory and pre-commercial expenses for our deramiocel program.
Current wording
Deramiocel for the treatment of DMD– The expenses for our DMD program include costs for personnel, clinical, regulatory and manufacturing-related expenses, including expenses related to the scale-up for potential commercial scale manufacturing if our deramiocel product is approved. In 2025, we expect to spend approximately $45.0 million to $50.0 million primarily consisting of CMC expansion, product inventory buildout, clinical, regulatory and pre-commercial expenses for our deramiocel program.
Previous wording
Exosome-Based Therapeutics and Vaccines – Our exosome platform is in early-stage preclinical development. We expect to spend approximately $5.0 million to $7.5 million during 2025 on development expenses related to our exosomes program, which includes personnel, preclinical studies and manufacturing related expenses for these technologies. Our expenses for this program are primarily focused on the expansion of our engineered exosomes platform including the manufacturing of our StealthX™ vaccine to be used in connection with our collaboration with NIAID.
Current wording
Exosome-Based Therapeutics and Vaccines – Our exosome platform is in early-stage preclinical development. We expect to spend approximately $5.0 million to $7.0 million during 2025 on development expenses related to our exosomes program, which includes personnel, preclinical studies and manufacturing related expenses for these technologies. Our expenses for this program are primarily focused on the expansion of our engineered exosomes platform including the manufacturing of our StealthX™ vaccine to be used in connection with our collaboration with NIAID.
Previous wording
The following table summarizes our liquidity and capital resources as of March 31, 2025 and December 31, 2024 and our net increase (decrease) in cash, cash equivalents, and marketable securities for the three months ended March 31, 2025 and 2024 and is intended to supplement the more detailed discussion that follows. The amounts stated in
Current wording
The following table summarizes our liquidity and capital resources as of June 30, 2025 and December 31, 2024 and our net increase (decrease) in cash, cash equivalents, and marketable securities for the six months ended June 30, 2025 and 2024 and is intended to supplement the more detailed discussion that follows. The amounts stated in the tables below are expressed in thousands. We estimate our current cash, cash equivalents, and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements into the fourth quarter of 2026.
Previous wording
Our total cash, cash equivalents and marketable securities as of March 31, 2025 were approximately $144.8 million compared to approximately $151.5 million as of December 31, 2024. The decrease in cash, cash equivalents and marketable securities from December 31, 2024 to March 31, 2025 is primarily due to our continuing efforts in preparing the Company for potential commercialization. As of March 31, 2025, we had approximately $26.1 million in total liabilities, of which approximately $12.0 million relates to deferred revenue, and approximately $123.9 million in net working capital.
Current wording
Our total cash, cash equivalents and marketable securities as of June 30, 2025 were approximately $122.8 million compared to approximately $151.5 million as of December 31, 2024. The decrease in cash, cash equivalents and marketable securities from December 31, 2024 to June 30, 2025 is primarily due to our continuing efforts in preparing the Company for potential commercialization. As of June 30, 2025, we had approximately $28.6 million in total liabilities, of which approximately $12.0 million relates to deferred revenue, and approximately $95.7 million in net working capital.
Previous wording
Cash used in operating activities was approximately $6.4 million and approximately $1.3 million for the three months ended March 31, 2025 and 2024, respectively. The increase of approximately $5.2 million in cash used in operating activities is due to an approximately $14.6 million increase in net loss for the three months ended March 31, 2025 as compared to the same period in 2024. Furthermore, there was an increase of approximately $2.2 million in stock-based compensation, approximately $4.9 million in deferred revenue, and approximately $1.3 million in accounts payable and accrued expenses for the three months ended March 31, 2025 as compared to the same period in 2024. To the extent we obtain sufficient capital and/or long-term debt funding and are able to continue developing our product candidates, including if we expand our platform technology portfolio, engage in further research and development activities, and, in particular, conduct preclinical studies and clinical trials, we expect to continue incurring substantial losses.
Current wording
Cash used in operating activities was approximately $26.6 million and approximately $13.6 million for the six months ended June 30, 2025 and 2024, respectively. The increase of approximately $13.0 million in cash used in operating activities is due to an approximately $29.5 million increase in net loss for the six months ended June 30, 2025 as compared to the same period in 2024. Furthermore, there was an increase of approximately $3.7 million in stock-based compensation, approximately $8.9 million in deferred revenue, and approximately $2.9 million in accounts payable and accrued expenses for the six months ended June 30, 2025 as compared to the same period in 2024. To the extent we obtain sufficient capital and/or long-term debt funding and are able to continue developing our product candidates, including if we expand our platform technology portfolio, engage in further research and development activities, and, in particular, conduct preclinical studies and clinical trials, we expect to continue incurring substantial losses.
Previous wording
We had cash flow provided by investing activities of approximately $23.9 million for the three months ended March 31, 2025 and cash flow used in investing activities of approximately $9.5 million for the three months ended March 31, 2024, respectively. The change in investing activities for the three months ended March 31, 2025 as compared to the same period of 2024 is due to the net effect from purchases, sales and maturities of marketable securities and the purchase of approximately $0.5 million in property and equipment and leasehold improvements.
Current wording
We had cash flow provided by investing activities of approximately $38.4 million for the six months ended June 30, 2025 and cash flow used in investing activities of approximately $5.2 million for the six months ended June 30, 2024, respectively. The change in investing activities for the six months ended June 30, 2025 as compared to the same period of 2024 is due to the net effect from purchases, sales and maturities of marketable securities and the purchase of approximately $2.6 million in property and equipment, leasehold improvements and construction in progress.
Previous wording
We had cash flow provided by financing activities of approximately $50.0 thousand and approximately $2.3 million for the three months ended March 31, 2025 and 2024, respectively. The decrease in cash provided by financing activities for the three months ended March 31, 2025 as compared to the three months ended March 31, 2024 is primarily due to the net proceeds from the sale of common stock during 2024.
Current wording
We had cash flow provided by financing activities of $69,934 and approximately $4.4 million for the six months ended June 30, 2025 and 2024, respectively. The decrease in cash provided by financing activities for the six months ended June 30, 2025 as compared to the six months ended June 30, 2024 is primarily due to the net proceeds from the sale of common stock during 2024.
Previous wording
From inception through March 31, 2025, we financed our operations primarily through private and public sales of our equity securities, and payments from distribution agreements and collaboration partners. As we have not generated any revenue from the commercial sale of our products to date, and we do not expect to generate revenue for several years, if ever, we will need to raise substantial additional capital to fund our research and development, including our long-term plans for clinical trials and new product development. We may seek to raise additional funds through various potential sources, such as equity and debt financings, government grants, or through strategic collaborations and license agreements or other distribution agreements. We can give no assurances that we will be able to secure such additional sources of funds to support our operations, complete our clinical trials or if such funds become available to us, that such additional financing will be sufficient to meet our needs. Moreover, to the extent that we raise additional funds by issuing equity securities, our
Current wording
From inception through June 30, 2025, we financed our operations primarily through private and public sales of our equity securities, and payments from distribution agreements and collaboration partners. As we have not generated any revenue from the commercial sale of our products to date, and we do not expect to generate revenue for several years, if ever, we will need to raise substantial additional capital to fund our research and development, including our long-term plans for clinical trials and new product development. We may seek to raise additional funds through various potential sources, such as equity and debt financings, government grants, or through strategic collaborations and license agreements or other distribution agreements. We can give no assurances that we will be able to secure such additional sources of funds to support our operations, complete our clinical trials or if such funds become available to us, that such additional financing will be sufficient to meet our needs. Moreover, to the extent that we raise additional funds by issuing equity securities, our stockholders may experience significant dilution, and debt financing, if available, may involve restrictive covenants. To the extent that we raise additional funds through collaboration and licensing arrangements, it may be necessary to relinquish some rights to our technologies or our product candidates or grant licenses on terms that may not be favorable to us.
Previous wording
Under the terms of the Japan Distribution Agreement, Capricor received an upfront payment of $12.0 million in the first quarter of 2023 and in addition, Capricor will potentially receive additional development and sales-based milestone payments of up to approximately $89.0 million, subject to foreign currency exchange rates, and a meaningful double-digit share of product revenue. Nippon Shinyaku will be responsible for the distribution of deramiocel in Japan. Capricor will be responsible for the conduct of clinical development and regulatory approval in Japan, as may be required, as well as the manufacturing of deramiocel. Subject to regulatory approval, Capricor or its designee will hold the Marketing Authorization in Japan if the product is approved in that territory.
Current wording
Under the terms of the Japan Distribution Agreement, Capricor received an upfront payment of $12.0 million in the first quarter of 2023 and in addition, Capricor will potentially receive additional development and sales-based milestone payments of up to approximately $89.0 million, subject to foreign currency exchange rates, and a meaningful double-digit share of product revenue. Nippon Shinyaku will be responsible for the distribution of deramiocel in Japan. Capricor will be responsible for the conduct of clinical development and regulatory approval in Japan, as may be required, as well as the
Previous wording
On September 16, 2024, the Company entered into a Subscription Agreement with Nippon Shinyaku pursuant to which the Company agreed to issue and sell to Nippon Shinyaku in a private placement (the “Private Placement”), an aggregate of 2,798,507 shares of the common stock of the Company at a price per Share of $5.36, which was issued at a 20% premium to the 60-day volume-weighted average price, for an aggregate purchase price of approximately $15.0 million. The Subscription Agreement also includes lock-up provisions restricting Nippon Shinyaku from selling or otherwise disposing of shares of Common Stock until the six-month anniversary of the Closing Date.
Current wording
On September 16, 2024, the Company entered into a Subscription Agreement with Nippon Shinyaku pursuant to which the Company agreed to issue and sell to Nippon Shinyaku in a private placement (the “Private Placement”), an aggregate of 2,798,507 shares of the common stock of the Company at a price per Share of $5.36, which was issued at a 20% premium to the 60-day volume-weighted average price, for an aggregate purchase price of approximately $15.0 million. The Subscription Agreement also includes lock-up provisions restricting Nippon Shinyaku from selling or otherwise disposing of shares of the Company’s common stock until the six-month anniversary of the Closing Date.
Previous wording
Agreement,”), with H.C. Wainwright & Co. LLC (“Wainwright”), under which the Company issued and sold shares of our common stock through Wainwright as sales agent. The Sales Agreement provided that Wainwright would be entitled to compensation for its services at a commission rate of 3.0% of the gross sales price per share of common stock sold. All shares issued pursuant to the ATM Program were issued pursuant to our shelf registration statement on Form S-3 (File No. 333-254363), which was initially filed with the Securities and Exchange Commission (the “SEC”), on March 16, 2021, amended on June 15, 2021 and declared effective by the SEC on June 16, 2021. From June 21, 2021 through October 1, 2024, the Company sold an aggregate of 9,228,383 shares of common stock under the ATM Program at an average price of approximately $8.13 per share for gross proceeds of approximately $75.0 million which represents all amounts that were available to be sold. Effective October 1, 2024, the ATM Program was closed and terminated. The Company paid cash commissions on the gross proceeds, plus reimbursement of expenses to Wainwright, as well as legal and accounting fees, in the aggregate amount of approximately $2.4 million.
Current wording
On June 21, 2021, the Company initiated an at-the-market offering under a prospectus supplement for aggregate sales proceeds of up to $75.0 million (the “ATM Program”), with the common stock to be distributed at the market prices prevailing at the time of sale. The ATM Program was established under a Common Stock Sales Agreement (the “Sales Agreement,”), with H.C. Wainwright & Co. LLC (“Wainwright”), under which the Company issued and sold shares of our common stock through Wainwright as sales agent. The Sales Agreement provided that Wainwright would be entitled to compensation for its services at a commission rate of 3.0% of the gross sales price per share of common stock sold. All shares issued pursuant to the ATM Program were issued pursuant to our shelf registration statement on Form S-3 (File No. 333-254363), which was initially filed with the Securities and Exchange Commission (the “SEC”), on March 16,
Previous wording
On February 26, 2025, Capricor notified CIRM of its election to convert the CIRM Award into a loan. As a result, certain requirements of the CIRM Award will no longer be applicable, including the revenue sharing requirements. The terms of the loan agreement are currently under discussion with CIRM. Depending on these discussions, accrued interest on the CIRM Award could reach up to approximately $7.1 million and will continue to accrue over time until the final payout, if it is determined that interest is due. There was no accrued interest recorded as of March 31, 2025.
Current wording
On February 26, 2025, Capricor notified CIRM of its election to convert the CIRM Award into a loan. As a result, certain requirements of the CIRM Award will no longer be applicable, including the revenue sharing requirements. The terms of the loan agreement are currently under discussion with CIRM. Depending on the results of these discussions, accrued interest on the CIRM Award could reach up to approximately $7.1 million and may continue to accrue over time until the final payout. There was no accrued interest recorded as of June 30, 2025.
Previous wording
obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. At contract inception, the Company assesses whether the goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation, or whether they are not distinct and are combined with other goods and services until a distinct bundle is identified. The Company then determines the transaction price, which typically includes upfront payments and any variable consideration that the Company determines is probable to not cause a significant reversal in the amount of cumulative revenue recognized when the uncertainty associated with the variable consideration is resolved. The Company then allocates the transaction price to each performance obligation and recognizes the associated revenue when, or as, each performance obligation is satisfied.
Current wording
The revenue standard provides a five-step framework for recognizing revenue as control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that it determines are within the scope of the revenue standard, the Company performs the following five steps: (i) identify the contract; (ii) identify the performance obligations; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. At contract inception, the Company assesses whether the goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation, or whether they are not distinct and are combined with other goods and services until a distinct bundle is identified. The Company then determines the transaction price, which typically includes upfront payments and any variable consideration that the Company determines is probable to not cause a
Previous wording
Our cost accruals for clinical trials and other R&D activities are based on estimates of the services received and efforts expended pursuant to contracts with numerous clinical trial centers and contract research organizations (“CROs”), clinical study sites, laboratories, consultants or other clinical trial vendors that perform activities in connection with a trial. Related contracts vary significantly in length and may be for a fixed amount, a variable amount based on actual costs incurred, capped at a certain limit, or for a combination of fixed, variable and capped amounts. Activity levels are monitored through close communication with the CROs and other clinical trial vendors, including detailed invoice and task completion review, analysis of expenses against budgeted amounts, analysis of work performed against approved contract budgets and payment schedules, and recognition of any changes in scope of the services to be performed. Certain CRO and significant clinical trial vendors provide an estimate of costs incurred but not invoiced at the end of each quarter for each individual trial. These estimates are reviewed and discussed with the CRO or vendor as necessary, and are included in R&D expenses for the related period. For clinical study sites which are paid periodically on a per-subject basis to the institutions performing the clinical study, we accrue an estimated amount based on subject screening and enrollment in each quarter. All estimates may differ significantly from the actual amount subsequently invoiced, which may occur several months after the related services were performed.
Current wording
clinical study sites, laboratories, consultants or other clinical trial vendors that perform activities in connection with a trial. Related contracts vary significantly in length and may be for a fixed amount, a variable amount based on actual costs incurred, capped at a certain limit, or for a combination of fixed, variable and capped amounts. Activity levels are monitored through close communication with the CROs and other clinical trial vendors, including detailed invoice and task completion review, analysis of expenses against budgeted amounts, analysis of work performed against approved contract budgets and payment schedules, and recognition of any changes in scope of the services to be performed. Certain CRO and significant clinical trial vendors provide an estimate of costs incurred but not invoiced at the end of each quarter for each individual trial. These estimates are reviewed and discussed with the CRO or vendor as necessary, and are included in R&D expenses for the related period. For clinical study sites which are paid periodically on a per-subject basis to the institutions performing the clinical study, we accrue an estimated amount based on subject screening and enrollment in each quarter. All estimates may differ significantly from the actual amount subsequently invoiced, which may occur several months after the related services were performed.
Previous wording
Our results include non-cash compensation expense as a result of the issuance of stock options and restricted stock awards, as applicable. We have issued stock options and restricted stock awards to employees, directors and consultants under our five stock option plans: (i) the 2006 Stock Option Plan, (ii) the 2012 Restated Equity Incentive Plan (which superseded the 2006 Stock Option Plan), (the “2012 Plan”), (iii) the 2012 Non-Employee Director Stock Option Plan (the “2012 Non-Employee Director Plan”), (iv) the 2020 Equity Incentive Plan (the “2020 Plan”), and (v) the 2021 Equity Incentive Plan (the “2021 Plan”). At this time, the Company only issues stock options and restricted stock awards under the 2020 Plan and the 2021 Plan and no longer issues stock awards under the 2006 Stock Option Plan, the 2012 Plan, or the 2012 Non-Employee Director Plan.
Current wording
Our results include non-cash compensation expense as a result of the issuance of stock options and restricted stock awards, as applicable. We have issued stock options and restricted stock awards to employees, directors and consultants under our five stock option plans: (i) the 2006 Stock Option Plan, (ii) the 2012 Restated Equity Incentive Plan (which superseded the 2006 Stock Option Plan), (the “2012 Plan”), (iii) the 2012 Non-Employee Director Stock Option Plan (the “2012 Non-Employee Director Plan”), (iv) the 2020 Equity Incentive Plan (the “2020 Plan”), and (v) the 2021 Equity Incentive Plan (the “2021 Plan”). At this time, the Company only issues stock options and restricted stock awards under the 2020 Plan, the 2021 Plan, and the 2025 Plan, and no longer issues stock awards under the 2006 Stock Option Plan, the 2012 Plan, or the 2012 Non-Employee Director Plan.
Previous wording
As part of the process of preparing our condensed consolidated financial statements, we are required to estimate our accrued expenses. Our clinical trial accrual process is designed to account for expenses resulting from our obligations under contracts with vendors, consultants, CROs and clinical site agreements in connection with conducting clinical trials. The financial terms of these contracts are subject to negotiations, which vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided to us under such contracts. Our objective is to reflect the appropriate clinical trial expenses in our condensed consolidated financial statements by matching the appropriate expenses with the period in which services are provided and efforts are expended. We account for these expenses according to the progress of the trial as measured by patient progression and the timing of various aspects of the trial. We determine accrual estimates through financial models that take into account discussions with applicable personnel and outside service providers as to the progress or state of completion of trials, or the services completed. During the course of a clinical trial, we adjust our clinical expense recognition if actual results differ from our estimates. We make estimates of our accrued expenses as of each balance sheet date in our condensed consolidated financial statements based on the facts and circumstances known to us at that time. Our clinical trial accrual and prepaid assets are dependent, in part, upon the receipt of timely and accurate reporting from CROs and other third-party vendors. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in us reporting amounts that are too high or too low for any particular period.
Current wording
payment flows that do not match the periods over which materials or services are provided to us under such contracts. Our objective is to reflect the appropriate clinical trial expenses in our condensed consolidated financial statements by matching the appropriate expenses with the period in which services are provided and efforts are expended. We account for these expenses according to the progress of the trial as measured by patient progression and the timing of various aspects of the trial. We determine accrual estimates through financial models that take into account discussions with applicable personnel and outside service providers as to the progress or state of completion of trials, or the services completed. During the course of a clinical trial, we adjust our clinical expense recognition if actual results differ from our estimates. We make estimates of our accrued expenses as of each balance sheet date in our condensed consolidated financial statements based on the facts and circumstances known to us at that time. Our clinical trial accrual and prepaid assets are dependent, in part, upon the receipt of timely and accurate reporting from CROs and other third-party vendors. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in us reporting amounts that are too high or too low for any particular period.
Rearranged current paragraphs (6)
Current report — wording carried from elsewhere
approximately $25.9 million and approximately $11.0 million, for the three months ended June 30, 2025 and 2024, respectively. Our net losses were approximately $50.3 million and approximately $20.8 million, for the six months ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had an accumulated deficit of approximately $250.1 million. We expect to incur significant expenses and operating losses for the foreseeable future.
Current report — wording carried from elsewhere
manufacturing of deramiocel. Subject to regulatory approval, Capricor or its designee will hold the Marketing Authorization in Japan if the product is approved in that territory.
Current report — wording carried from elsewhere
2021, amended on June 15, 2021 and declared effective by the SEC on June 16, 2021. From June 21, 2021 through October 1, 2024, the Company sold an aggregate of 9,228,383 shares of common stock under the ATM Program at an average price of approximately $8.13 per share for gross proceeds of approximately $75.0 million which represents all amounts that were available to be sold. Effective October 1, 2024, the ATM Program was closed and terminated. The Company paid cash commissions on the gross proceeds, plus reimbursement of expenses to Wainwright, as well as legal and accounting fees, in the aggregate amount of approximately $2.4 million.
Current report — wording carried from elsewhere
significant reversal in the amount of cumulative revenue recognized when the uncertainty associated with the variable consideration is resolved. The Company then allocates the transaction price to each performance obligation and recognizes the associated revenue when, or as, each performance obligation is satisfied.
Current report — wording carried from elsewhere
Our cost accruals for clinical trials and other R&D activities are based on estimates of the services received and efforts expended pursuant to contracts with numerous clinical trial centers and contract research organizations (“CROs”),
Current report — wording carried from elsewhere
As part of the process of preparing our condensed consolidated financial statements, we are required to estimate our accrued expenses. Our clinical trial accrual process is designed to account for expenses resulting from our obligations under contracts with vendors, consultants, CROs and clinical site agreements in connection with conducting clinical trials. The financial terms of these contracts are subject to negotiations, which vary from contract to contract and may result in
Rearranged previous paragraphs (4)
Previous report — wording carried elsewhere
the tables below are expressed in thousands. We estimate our current cash, cash equivalents, and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements into 2027.
Previous report — wording carried elsewhere
stockholders may experience significant dilution, and debt financing, if available, may involve restrictive covenants. To the extent that we raise additional funds through collaboration and licensing arrangements, it may be necessary to relinquish some rights to our technologies or our product candidates or grant licenses on terms that may not be favorable to us.
Previous report — wording carried elsewhere
On June 21, 2021, the Company initiated an at-the-market offering under a prospectus supplement for aggregate sales proceeds of up to $75.0 million (the “ATM Program”), with the common stock to be distributed at the market prices prevailing at the time of sale. The ATM Program was established under a Common Stock Sales Agreement (the “Sales
Previous report — wording carried elsewhere
The revenue standard provides a five-step framework for recognizing revenue as control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that it determines are within the scope of the revenue standard, the Company performs the following five steps: (i) identify the contract; (ii) identify the performance obligations; (iii) determine the transaction price; (iv) allocate the transaction price to the performance