Capricor Therapeutics' BLA for deramiocel in DMD cardiomyopathy accepted by FDA; priority review set for August 31, 2025.
Quiver AI Summary
Capricor Therapeutics announced that the U.S. FDA has accepted its Biologics License Application (BLA) for deramiocel, aimed at treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), with a priority review and a target date of August 31, 2025. The latest trial results presented at the MDA Conference indicated a significant slowing of disease progression in patients treated with deramiocel, highlighting its potential effectiveness. The company received a $10 million milestone payment from Nippon Shinyaku and has a strong cash balance of approximately $152 million, expected to support operations until 2027. Capricor is preparing for the commercial launch of deramiocel while also expanding its manufacturing capabilities in anticipation of regulatory approval in the U.S. and Europe, where deramiocel has received Orphan Drug and Advanced Therapy designations from the EMA.
Potential Positives
- FDA acceptance of the Biologics License Application (BLA) for deramiocel marks a significant step towards obtaining full approval for a treatment addressing Duchenne muscular dystrophy cardiomyopathy, a critical need in the market.
- The BLA was granted priority review status, with a PDUFA target action date set for August 31, 2025, indicating the FDA's recognition of the urgency and importance of this potential therapy.
- New data presented at the 2025 MDA Conference demonstrated a 52% slowing in disease progression, reinforcing deramiocel's potential as a transformational therapy for patients with Duchenne muscular dystrophy.
- Strong financial position with approximately $151.5 million in cash as of the end of 2024, expected to support operations into 2027, providing stability for ongoing research and commercialization efforts.
Potential Negatives
- Net loss for the fourth quarter of 2024 increased significantly to approximately $7.1 million from $0.8 million in the same quarter of the previous year.
- Operating expenses for the fourth quarter of 2024 rose sharply to approximately $18.8 million, compared to $13.4 million for the same quarter in 2023, indicating a concerning increase in costs relative to revenue.
- Revenues for the fourth quarter and full year of 2024 decreased compared to 2023, showing a decline from $12.1 million to $11.1 million and from $25.2 million to $22.3 million respectively.
FAQ
What is the significance of the FDA acceptance of the BLA for deramiocel?
The FDA's acceptance indicates a step closer to full approval for treating DMD cardiomyopathy with deramiocel.
When is the target action date for the deramiocel BLA?
The target action date is set for August 31, 2025, under the Priority Review designation.
What were the findings from the HOPE-2 trial?
The HOPE-2 trial showed a 52% slowing of disease progression in patients treated with deramiocel over three years.
How much cash does Capricor expect to have by the end of 2024?
Capricor expects to have approximately $152 million in cash at the end of 2024, supporting operations into 2027.
What are the designations granted to deramiocel in the EU?
Deramiocel has received Orphan Drug and Advanced Therapy Medicinal Product designations from the European Medicines Agency.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$CAPR Insider Trading Activity
$CAPR insiders have traded $CAPR stock on the open market 1 times in the past 6 months. Of those trades, 1 have been purchases and 0 have been sales.
Here’s a breakdown of recent trading of $CAPR stock by insiders over the last 6 months:
- SHINYAKU CO LTD NIPPON purchased 2,798,507 shares for an estimated $14,999,997
To track insider transactions, check out Quiver Quantitative's insider trading dashboard.
$CAPR Hedge Fund Activity
We have seen 79 institutional investors add shares of $CAPR stock to their portfolio, and 39 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- FARALLON CAPITAL MANAGEMENT LLC added 2,250,400 shares (+inf%) to their portfolio in Q4 2024, for an estimated $31,055,520
- STATE STREET CORP added 1,696,055 shares (+331.1%) to their portfolio in Q4 2024, for an estimated $23,405,559
- BLACKROCK, INC. added 1,216,907 shares (+69.5%) to their portfolio in Q4 2024, for an estimated $16,793,316
- VANGUARD GROUP INC added 700,243 shares (+44.4%) to their portfolio in Q4 2024, for an estimated $9,663,353
- WOODLINE PARTNERS LP added 629,934 shares (+inf%) to their portfolio in Q4 2024, for an estimated $8,693,089
- MORGAN STANLEY added 500,238 shares (+380.4%) to their portfolio in Q4 2024, for an estimated $6,903,284
- IKARIAN CAPITAL, LLC removed 300,000 shares (-100.0%) from their portfolio in Q4 2024, for an estimated $4,140,000
To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.
$CAPR Analyst Ratings
Wall Street analysts have issued reports on $CAPR in the last several months. We have seen 5 firms issue buy ratings on the stock, and 0 firms issue sell ratings.
Here are some recent analyst ratings:
- Piper Sandler issued a "Overweight" rating on 10/21/2024
- Oppenheimer issued a "Outperform" rating on 10/14/2024
- H.C. Wainwright issued a "Buy" rating on 10/11/2024
- Maxim Group issued a "Buy" rating on 09/25/2024
- Cantor Fitzgerald issued a "Overweight" rating on 09/24/2024
To track analyst ratings and price targets for $CAPR, check out Quiver Quantitative's $CAPR forecast page.
$CAPR Price Targets
Multiple analysts have issued price targets for $CAPR recently. We have seen 4 analysts offer price targets for $CAPR in the last 6 months, with a median target of $15.0.
Here are some recent targets:
- Edward Tenthoff from Piper Sandler set a target price of $17.5 on 10/21/2024
- Leland Gershall from Oppenheimer set a target price of $21.5 on 10/14/2024
- Jason McCarthy from Maxim Group set a target price of $12.5 on 09/25/2024
- Joseph Pantginis from H.C. Wainwright set a target price of $10.0 on 09/25/2024
Full Release
Announced the U.S. FDA has accepted our Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy
BLA granted priority review with a Prescription Drug User Fee Act (PDUFA) target action date set for August 31, 2025
Reported positive data from HOPE-2 open label extension (OLE) trial at 2025 MDA Conference showing preservation of skeletal muscle function over 3 years resulting in 52% slowing of disease
Received $10 million milestone payment from Nippon Shinyaku; cash balance of approximately $152 million at year-end 2024 expected to support current planned operations into 2027
Capricor to host conference call and webcast today at 4:30 p.m. ET
SAN DIEGO, March 19, 2025 (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 fourth quarter and full year ended December 31, 2024 and provided a corporate update.
“2024 was a transformational year for Capricor and the patients we serve as we move closer to our goal of bringing the first cellular therapy to market for the treatment of Duchenne-cardiomyopathy, a condition for which there are no approved therapies" said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “We continue to work diligently towards our August 31, 2025 action date for our deramiocel Biologics License Application, directly engaging with the FDA, preparing for pre-approval licensure inspection and preparing for potential commercial launch with our partner Nippon Shinyaku Co. (U.S. subsidiary:
NS Pharma Inc
). Our BLA is the culmination of a body of work that has been focused on bringing this transformational therapy to those patients in need with the potential to alter the trajectory of this degenerative disease
.
In addition to our operational achievements, we ended the year with over $150 million on our balance sheet allowing us to invest diligently in manufacturing expansion and commercial endeavors as we work to bring deramiocel to the Duchenne community in the United States and abroad.”
Fourth Quarter 2024 and Recent Developments
In March 2025, the FDA accepted Capricor’s BLA seeking full approval of deramiocel for the treatment of individuals with Duchenne muscular dystrophy cardiomyopathy.
Our BLA has been granted Priority Review by the FDA, with a Prescription Drug User Fee Act (PDUFA) action date set for August 31, 2025. Deramiocel is a cellular therapy that consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in preservation of cardiac and skeletal muscle function in DMD. CDCs act by secreting exosomes, which reduce fibrosis in muscle resulting in a reduction in myocardial scarring and cardiac inflammation by targeting macrophages to adopt a healing, rather than a pro-inflammatory phenotype. The BLA submission for deramiocel included safety and efficacy data from Capricor’s Phase 2 HOPE-2 placebo-controlled trial and the HOPE-2 open label extension (OLE) trial compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. The results from these clinical studies demonstrated statistically significant and clinically relevant improvements in cardiac function up to three-years after treatment as well as a consistent safety profile. Capricor’s ongoing HOPE-3 Phase 3 study which is assessing skeletal muscle function has not been requested for review by the FDA for this application.
Expanded internal manufacturing capacity for deramiocel production:
In February 2025, Capricor entered into an amendment to its current lease for additional GMP space in its headquarters located in San Diego, California to support additional commercial manufacturing capacity and throughput.
The European Medicines Agency (EMA) granted deramiocel both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations for the treatment of DMD.
The Orphan Drug designation provides Capricor with several benefits that support the development of deramiocel in Europe, including market exclusivity for 10 years if approval is granted and substantially reduced regulatory fees. The ATMP designation provides substantial regulatory support to assist in the development of cell-based therapies. The additional support can potentially reduce time to market, streamline development, and open up access to critical resources. The ongoing HOPE-3 study may also support ex-U.S. marketing authorizations.
Capricor presented new positive data at the 2025 Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference.
The late-breaking poster showed that patients treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb (PUL v2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). This equates to a 52 percent slowing of disease progression, reinforcing deramiocel’s potential long-term therapeutic durability.
Capricor presented
preclinical data
at the 2024 American Association of Extracellular Vesicles (AAEV) Annual Meeting
.
The data highlighted a potential exosome-based approach for delivering phosphorodiamidate morpholino oligomers (PMOs) to muscle for the treatment of DMD.
The Company’s StealthX™ exosome-based vaccine for the prevention of SARS-CoV-2 was selected to be part of Project NextGen.
This initiative led by the U.S. Department of Health and Human Services is to advance a pipeline of new, innovative vaccines for future pandemics. Currently, manufacturing is underway for our StealthX™ vaccine and the National Institute of Allergy and Infectious Diseases (NIAID) is planning for regulatory approval in the second quarter of 2025 with the clinical study initiated soon thereafter.
Fourth Quarter and Full Year 2024 Financial Results
Cash position:
Cash, cash equivalents and marketable securities totaled approximately $151.5 million as of December 31, 2024 compared to approximately $39.5 million as of December 31, 2023. In October 2024, the Company completed a public offering for net proceeds of approximately $80.8 million. Additionally, in January 2025, the Company received $10.0 million from the second development milestone payment under our U.S. Distribution and Commercialization Agreement with Nippon Shinyaku.
Revenues:
Revenues for the fourth quarter of 2024 were approximately $11.1 million compared to $12.1 million for the fourth quarter of 2023. Additionally, revenues for the year ended December 31, 2024 and 2023 were approximately $22.3 million and $25.2 million, respectively. Capricor’s primary source of revenue was from the ratable recognition of the $40.0 million (upfront and first development milestone payments) and the recognition of the $10.0 million second development milestone payment in accordance with its U.S. Commercialization and Distribution Agreement with Nippon Shinyaku.
Costs and Expenses:
Total operating expenses for the fourth quarter of 2024 were approximately $18.8 million compared to $13.4 million for the fourth quarter of 2023. Total operating expenses for the year ended December 31, 2024 and 2023 were approximately $64.8 million and $49.3 million, respectively.
Net loss:
The Company reported a net loss of approximately $7.1 million, or $0.16 per share, for the fourth quarter of 2024, compared to a net loss of $0.8 million, or $0.02 per share, for the fourth quarter of 2023. Capricor reported a net loss of approximately $40.5 million, or $1.15 per share, and $22.3 million, or $0.83 per share, for the year ended December 31, 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:
Piper Sandler & Co. Cardio Day, April 1-2, 2025, Virtual
JonesTrading Technology and Innovation Conference, April 8-9, 2025, Las Vegas
Conference Call and Webcast
To participate in the conference call, please dial 1-800-717-1738 (Domestic) or 1-646-307-1865 (International) and reference the conference ID: 98172. 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 Duchenne Muscular Dystrophy
DMD is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. Treatment options are limited and there is no cure.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been the subject of over 200 peer-reviewed scientific publications and have been administered to over 250 human subjects across several clinical trials.
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, an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to exert potent immunomodulatory and anti-fibrotic actions in preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. Deramiocel is currently in late-stage 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 September 30, 2024, as filed with the Securities and Exchange Commission on November 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 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 Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
CAPRICOR THEAPEUTICS, INC. | ||||||||||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||||||||||
(UNAUDITED) | ||||||||||||||||
Three months ended December 31, | Year ended December 31, | |||||||||||||||
2024 | 2023 | 2024 | 2023 | |||||||||||||
REVENUE | ||||||||||||||||
Revenue | $ | 11,130,509 | $ | 12,088,089 | $ | 22,270,465 | $ | 25,178,066 | ||||||||
TOTAL REVENUE | 11,130,509 | 12,088,089 | 22,270,465 | 25,178,066 | ||||||||||||
OPERATING EXPENSES | ||||||||||||||||
Research and development | 14,554,936 | 9,940,167 | 49,968,585 | 36,448,039 | ||||||||||||
General and administrative | 4,273,414 | 3,429,214 | 14,866,722 | 12,807,886 | ||||||||||||
TOTAL OPERATING EXPENSES | 18,828,350 | 13,369,381 | 64,835,307 | 49,255,925 | ||||||||||||
LOSS FROM OPERATIONS | (7,697,841 | ) | (1,281,292 | ) | (42,564,842 | ) | (24,077,859 | ) | ||||||||
OTHER INCOME (EXPENSE) | ||||||||||||||||
Other income | 7,471 | 67,657 | 7,471 | 67,657 | ||||||||||||
Investment income | 686,572 | 452,199 | 2,202,990 | 1,728,701 | ||||||||||||
Loss on disposal of fixed assets | (112,805 | ) | (653 | ) | (112,805 | ) | (6,041 | ) | ||||||||
TOTAL OTHER INCOME (EXPENSE) | 581,238 | 519,203 | 2,097,656 | 1,790,317 | ||||||||||||
NET LOSS | (7,116,603 | ) | (762,089 | ) | (40,467,186 | ) | (22,287,542 | ) | ||||||||
OTHER COMPREHENSIVE INCOME (LOSS) | ||||||||||||||||
Net unrealized gain on marketable securities | 930,734 | 122,605 | 791,142 | 130,569 | ||||||||||||
COMPREHENSIVE LOSS | $ | (6,185,869 | ) | $ | (639,484 | ) | $ | (39,676,044 | ) | $ | (22,156,973 | ) | ||||
Net loss per share, basic and diluted | $ | (0.16 | ) | $ | (0.02 | ) | $ | (1.15 | ) | $ | (0.83 | ) | ||||
Weighted average number of shares, basic and diluted | 44,509,154 | 30,664,100 | 35,218,628 | 26,778,360 | ||||||||||||
CAPRICOR THEAPEUTICS, INC. | ||||||||
SUMMARY BALANCE SHEETS | ||||||||
December 31, 2024 | December 31, 2023 | |||||||
Cash, cash equivalents and marketable securities | $ | 151,515,877 | $ | 39,487,703 | ||||
Total assets | $ | 170,481,086 | $ | 58,734,327 | ||||
Total liabilities | $ | 25,018,750 | $ | 36,132,860 | ||||
Total stockholders' equity – 45,582,288 and 31,148,320 common shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively | 145,462,336 | 22,601,467 | ||||||
Total liabilities and stockholders' equity | $ | 170,481,086 | $ | 58,734,327 | ||||
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