Capricor Therapeutics said on Wednesday its cell therapy for a heart condition related to a rare muscular disorder met the main goal of a late-stage study, months after U.S. regulators rejected its in... ...

Core Insights - Capricor Therapeutics announced positive topline results from the Phase 3 HOPE-3 trial for Deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) [1][2] - The trial demonstrated statistically significant improvements in both skeletal and cardiac function, reinforcing previous findings from the HOPE-2 trial [2][5] - The results are expected to support regulatory approval following a Complete Response Letter from the FDA earlier this year [2][5] Study Details - HOPE-3 was a randomized, double-blind, placebo-controlled trial involving 106 participants across 20 U.S. clinical sites, with an average age of approximately 15 years [2][11] - Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for 12 months [2][12] - The study maintained a favorable safety and tolerability profile consistent with prior clinical experience [2][5] Efficacy Results - The primary endpoint, Performance of Upper Limb (PUL v2.0), showed a 54% slowing of progression compared to placebo (p=0.029) [3][5] - The key secondary endpoint, Left Ventricular Ejection Fraction (LVEF), demonstrated a 91% improvement (p=0.041) [3][5] - Statistically significant results were achieved across all controlled secondary endpoints [5] Clinical Implications - The findings indicate meaningful treatment effects on both upper limb function and cardiomyopathy, addressing critical aspects of DMD [5][6] - The preservation of cardiac function is particularly significant, as cardiomyopathy is the leading cause of mortality in DMD [6][8] - The results provide renewed confidence for families seeking therapies that maintain functional ability and protect heart health [6][10] Company Background - Capricor Therapeutics focuses on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead candidate for DMD [13] - Deramiocel has received Orphan Drug Designation from the FDA and EMA, along with other designations that may facilitate regulatory approval [10][15] - The company is committed to advancing innovative therapies and has a proprietary platform for targeted delivery of therapeutics [13]

Core Insights - Premarket trading is showing notable activity with early price movements indicating potential opportunities before the market opens [1] Premarket Gainers - Mawson Infrastructure Group Inc. (MIGI) is up 10% at $5.65 [3] - Mingteng International Corporation Inc. (MTEN) is up 9% at $1.88 [3] - Barnwell Industries, Inc. (BRN) is up 9% at $1.20 [3] - Amentum Holdings, Inc. (AMTM) is up 8% at $27.57 [3] - Antelope Enterprise Holdings Limited (AEHL) is up 8% at $2.75 [3] - Protagenic Therapeutics, Inc. (PTIX) is up 8% at $2.43 [3] - Envirotech Vehicles, Inc. (EVTV) is up 8% at $1.18 [3] - Vir Biotechnology, Inc. (VIR) is up 6% at $6.49 [3] - Zoom Communications Inc. (ZM) is up 5% at $82.73 [3] - Core AI Holdings (CHAI) is up 5% at $2.68 [3] Premarket Losers - Foxx Development Holdings Inc. (FOXX) is down 13% at $4.00 [4] - Jaguar Health, Inc. (JAGX) is down 11% at $1.13 [4] - AlphaTON Capital Corp. (ATON) is down 7% at $2.54 [4] - Semtech Corporation (SMTC) is down 6% at $65.81 [4] - Cypherpunk Technologies Inc. (CYPH) is down 5% at $1.73 [4] - SunPower Inc. (SPWR) is down 5% at $1.59 [4] - Visionary Holdings Inc. (GV) is down 4% at $1.23 [4] - Gelteq Limited (GELS) is down 4% at $1.01 [4] - Capricor Therapeutics, Inc. (CAPR) is down 3% at $4.47 [4] - Mobile-Network Solutions (MNDR) is down 3% at $2.82 [4]

Core Insights - Whales have adopted a bullish stance on Capricor Therapeutics, with 61% of trades being bullish and 23% bearish [1] - The expected price movement for Capricor Therapeutics is projected between $1.0 and $10.0 [2] - The average open interest for options stands at 8,498.25, with a total volume of 54,025.00 in the last 30 days [3] Options Activity - Significant options activity includes 13 trades, with a total of 7 puts amounting to $498,769 and 6 calls totaling $468,675 [1] - The largest spotted options include a bullish put sweep with a total trade price of $146.9K and a strike price of $2.50 [6] - A bullish call trade was noted with a total trade price of $115.0K and a strike price of $10.00 [6] Company Overview - Capricor Therapeutics is a clinical-stage biotechnology company focused on developing cell and exosome-based therapeutics for Duchenne muscular dystrophy (DMD) and other diseases [7] - The company's product pipeline includes CAP-1002 and deramiocel, with deramiocel currently in Phase 3 clinical development for DMD [7] Market Status - An industry analyst has set an average target price of $13.0 for Capricor Therapeutics [8] - Roth Capital maintains a Buy rating on the stock, also targeting a price of $13 [9] - The current stock price is $5.6, reflecting a decrease of -2.61% [11]

Core Viewpoint - Capricor Therapeutics, Inc. (NASDAQ:CAPR) experienced a significant decline in stock price following negative commentary from former pharmaceutical executive Martin Shkreli, who publicly identified the company as a short target and expressed skepticism about its clinical trial data and cell therapy approach [1][2][3]. Company Analysis - Shkreli criticized Capricor's lead candidate, deramiocel, and predicted that the upcoming HOPE-3 (COPE-3) study would report unfavorable data, stating that it is the company's only asset [2][3]. - He raised concerns regarding challenges with cell trafficking and potential safety issues related to donor heart-derived cells [3]. - Following Shkreli's comments, Capricor's stock price dropped over 17%, settling at approximately $4.75 per share [4][5]. Recent Developments - On the same day, Capricor announced a scalable framework for loading therapeutic oligonucleotides into exosomes, which management believes could facilitate the manufacturing of clinically relevant quantities of loaded exosomes, a critical step for advancing their platform into later-stage trials [4][5]. - CEO Linda Marbán emphasized the strength and versatility of Capricor's exosome technology and its potential applications across a broad range of diseases [5].

Core Insights - Capricor Therapeutics has announced new data on a scalable framework for loading therapeutic siRNAs and PMOs into exosomes, which is crucial for future clinical development [1][2][4] Group 1: Exosome Technology - The poster presented at the AAEV 2025 highlighted Capricor's proprietary exosome-based technology, showcasing strategies for scale-up and scale-out electroporation to achieve larger yields of engineered exosomes [2][4] - Key findings indicate that engineered exosomes derived from 293F cells can be successfully loaded with therapeutic siRNA and PMO using optimized electroporation conditions, with comparable loading efficiencies to standard methods [7] Group 2: Clinical Development - Establishing a scalable and reproducible loading process is essential for advancing Capricor's exosome pipeline for therapeutic development [4] - The framework developed provides a feasible pathway for producing clinically relevant quantities of loaded exosomes to support future clinical development [7] Group 3: Company Overview - Capricor Therapeutics is focused on advancing cell and exosome-based therapeutics, with its lead product candidate, Deramiocel, in late-stage clinical development for Duchenne muscular dystrophy (DMD) [5][9] - The company is leveraging its proprietary StealthX™ platform for preclinical development aimed at targeted delivery of therapeutics, with potential applications across a wide range of diseases [5]

Financial Data and Key Metrics Changes - As of September 30, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $98.6 million, which is expected to cover anticipated expenses into the fourth quarter of 2026 [17] - Revenue for Q3 2025 was zero, compared to approximately $2.3 million for Q3 2024, and revenue for the first three quarters of 2025 was zero, compared to approximately $11.1 million for the same period in 2024 [18] - The net loss for Q3 2025 was approximately $24.6 million, compared to a net loss of approximately $12.6 million for Q3 2024, and the net loss for the first three quarters of 2025 was approximately $74.9 million, compared to approximately $33.4 million for the same period in 2024 [19] Business Line Data and Key Metrics Changes - The company has administered over 800 infusions to approximately 150 boys and young men with Duchenne muscular dystrophy, demonstrating a strong and consistent safety profile for deramiocel [8] - The HOPE-3 phase 3 clinical study focuses on non-ambulant individuals, a patient population that has historically had limited clinical research dedicated to it [4] Market Data and Key Metrics Changes - The company is preparing for potential global expansion while closely monitoring evolving U.S. and international pricing policies [15] - The FDA has classified the resubmission of the BLA as Type 2, which means the review period can be up to six months, but there is precedent for faster review times [11] Company Strategy and Development Direction - The company aims to bring forward the first therapy that directly addresses Duchenne muscular dystrophy-associated cardiomyopathy, emphasizing the life-limiting cardiovascular impact of the disease [8] - The company is engaging both neurology and cardiology specialists to ensure an integrated approach to patient care should deramiocel receive approval [15] - The company is advancing its StealthX program under Project NextGen to develop next-generation vaccines for COVID-19 and other infectious threats [16] Management's Comments on Operating Environment and Future Outlook - Management remains highly motivated to achieve approval for deramiocel as early as possible in 2026, well ahead of the September 30, 2026 deadline for priority review voucher eligibility [11] - Management expressed confidence in the potential of deramiocel to stabilize disease progression and preserve both muscle and heart function [12] Other Important Information - The company has published a peer-reviewed paper detailing new mechanistic insights into deramiocel's mechanism of action, reinforcing the biologic rationale for its development program [12] - The San Diego commercial facility is fully operational and preparing for GMP production activities, addressing all CMC-related items cited in the CRL [14] Q&A Session Summary Question: What to expect from the upcoming HOPE-3 data? - The company will release top-line data including primary and key secondary endpoints as soon as available and will host a conference call to explain the implications of the data [22] Question: Are there any statistical changes in the study regarding left ventricular ejection fraction? - The study was always powered to measure ejection fraction, and the results from previous studies provide strong support for this analysis [23] Question: How will the statistical analysis plan handle the primary and secondary endpoints? - The primary endpoint remains the performance of the upper limb, and the analysis will focus on both combined cohorts and specifically on Cohort B [25] Question: What is the FDA's view on cohort B? - The focus on cohort B is due to its importance in addressing manufacturing concerns and ensuring the efficacy data is tied to the approved facility [41] Question: What is the potential for label expansion upon approval? - The company will discuss potential label expansion during conversations with the FDA, contingent on the data outcomes [50]

Financial Position - As of September 30, 2025, Capricor Therapeutics had cash, cash equivalents, and marketable securities totaling approximately $98.6 million[176]. - Capricor's accumulated deficit as of September 30, 2025, was approximately $274.7 million[177]. - Total cash, cash equivalents, and marketable securities as of September 30, 2025, were approximately $98.6 million, down from $151.5 million as of December 31, 2024[200]. - As of September 30, 2025, total liabilities were approximately $42.6 million, with net working capital of approximately $72.8 million[200]. - As of September 30, 2025, the principal liability balance for the CIRM Award was approximately $3.4 million, with potential accrued interest reaching up to approximately $7.9 million[224][225]. - The fair value of the company's cash, cash equivalents, and marketable securities was approximately $98.6 million[257]. Revenue and Income - The company has no commercial product sales to date and will not generate any commercial product revenue until FDA approval is obtained[179]. - Clinical development income for Q3 2025 was $0, compared to approximately $2.3 million in Q3 2024, with total income for the nine months ended September 30, 2025 being approximately $0 versus $11.1 million in 2024[181]. - The company has not generated any revenue from commercial sales and does not expect to do so for several years, necessitating substantial additional capital for research and development[205]. - The company has not yet achieved commercial sales of its drug candidates, but applies ASU 606 for revenue recognition in distribution agreements[235]. - The company recognizes revenue based on a five-step framework, including identifying contracts and performance obligations, determining transaction prices, and recognizing revenue as obligations are satisfied[236]. Expenses and Losses - For the three months ended September 30, 2025, the net loss was approximately $24.6 million, compared to a net loss of approximately $12.6 million for the same period in 2024[177]. - For the nine months ended September 30, 2025, the net loss was approximately $74.9 million, compared to approximately $33.4 million for the same period in 2024[177]. - The company anticipates significant increases in expenses and operating losses as it seeks to develop and commercialize Deramiocel and other product candidates[178]. - R&D expenses for Q3 2025 increased by approximately $8.6 million, or 72%, to $20.4 million compared to $11.8 million in Q3 2024, driven by increases in personnel costs and clinical trial expenses[184]. - Total G&A expenses for Q3 2025 rose by approximately $2.5 million, or 71%, to $5.9 million compared to $3.5 million in Q3 2024, primarily due to increased personnel and professional service costs[189]. - The company expects to spend approximately $50.0 million to $55.0 million in 2025 on the Deramiocel program, focusing on CMC expansion and pre-commercial expenses[195]. - The exosome platform is projected to incur expenses of approximately $7.0 million to $9.0 million in 2025, primarily for preclinical studies and manufacturing[196]. Cash Flow - Cash used in operating activities for the nine months ended September 30, 2025, was approximately $46.2 million, an increase of $21.0 million compared to $25.2 million in the same period of 2024[202]. - The company had cash flow from investing activities of approximately $54.4 million for the nine months ended September 30, 2025, compared to $7.2 million in 2024, due to marketable securities transactions and capital expenditures[203]. - Cash flow provided by financing activities was $76.884 million for the nine months ended September 30, 2025, down from approximately $71.7 million in the same period of 2024[204]. Funding and Agreements - Capricor received an upfront payment of $30 million in 2022 under a U.S. Distribution Agreement with Nippon Shinyaku, with potential milestone payments totaling $100 million[211]. - Under the Japan Distribution Agreement, Capricor received an upfront payment of $12 million and may receive additional milestone payments of up to approximately $89 million[213]. - A Binding Term Sheet with Nippon Shinyaku for Europe includes potential milestone payments of up to $715 million, subject to finalization of a definitive agreement[214]. - The company raised approximately $86.3 million from a public offering in October 2024 at a price of $17.00 per share[216]. - In a private placement in September 2024, the company sold 2,798,507 shares at $5.36 per share for approximately $15 million[217]. - The company established an at-the-market offering program in September 2025 for aggregate sales proceeds of up to $150 million[219]. Going Concern - The company has substantial doubt about its ability to continue as a going concern, lacking sufficient funds to support operations for at least twelve months from the filing date[207]. Clinical Trials and Product Development - The Phase II HOPE-2 trial of Deramiocel met its primary endpoint with a p-value of 0.01 and secondary endpoints including full PUL v2.0 with a p-value of 0.04[166]. - The HOPE-3 Phase 3 clinical trial is evaluating the safety and efficacy of Deramiocel in 105 boys with DMD, with topline data expected in the fourth quarter of 2025[164]. - Deramiocel has received Orphan Drug Designation from both the U.S. FDA and the European Medicines Agency (EMA) for the treatment of DMD[168]. - Capricor has entered into exclusive commercialization and distribution agreements with Nippon Shinyaku for the United States and Japan[169]. Accounting and Financial Reporting - R&D expenses primarily consist of salaries, clinical trial costs, and other related expenses, with costs expensed as incurred[243]. - The company utilizes estimates for clinical trial accruals based on contracts with various vendors, with adjustments made as necessary[244]. - Stock-based compensation is recorded as non-cash expense, with fair value determined using the Black-Scholes model for stock options[250]. - The company has issued stock options and restricted stock awards under multiple plans, with expenses included in general and administrative or R&D expenses[251]. - The company aims to mitigate market risk by investing in high credit quality securities, with no current hedging of interest rate exposure[258]. - The company expects to record additional non-cash compensation expense in the future, which may be significant[251]. - The company evaluates the impact of newly issued accounting standards on its financial statements, including ASU 2025-06 and ASU 2024-03[254][255].

Financial Performance - Revenues for Q3 2025 were $0, a decrease from approximately $2.3 million in Q3 2024; revenues for the first nine months of 2025 were also $0, down from approximately $11.1 million in the same period of 2024[10]. - Total operating expenses for Q3 2025 were approximately $26.3 million, compared to approximately $15.3 million for Q3 2024; total operating expenses for the first nine months of 2025 were approximately $79.0 million, up from approximately $46.0 million in the same period of 2024[11]. - The net loss for Q3 2025 was approximately $24.6 million, or $0.54 per share, compared to a net loss of approximately $12.6 million, or $0.38 per share, for Q3 2024[12]. - Total revenue for Q3 2025 was $0, compared to $2,261,642 in Q3 2024, representing a decrease of 100% year-over-year[26]. - The net loss for Q3 2025 was $24,570,647, compared to a net loss of $12,556,728 in Q3 2024, indicating a 96% increase in losses[26]. - The net loss per share for Q3 2025 was $0.54, compared to $0.38 in Q3 2024, reflecting a 42% increase in loss per share[26]. - Comprehensive loss for Q3 2025 was $25,114,099, compared to $12,615,494 in Q3 2024, indicating a 99% increase in comprehensive losses[26]. Cash and Assets - Cash, cash equivalents, and marketable securities totaled approximately $98.6 million as of September 30, 2025, down from approximately $151.5 million as of December 31, 2024[9]. - Cash, cash equivalents, and marketable securities decreased to $98,565,971 as of September 30, 2025, down from $151,515,877 at the end of 2024, a decline of 35%[28]. - Total assets decreased to $126,438,207 as of September 30, 2025, from $170,481,086 at the end of 2024, a reduction of 26%[28]. - Total liabilities increased to $42,570,952 as of September 30, 2025, compared to $25,018,750 at the end of 2024, an increase of 70%[28]. Research and Development - A Phase 1 clinical trial for the StealthX™ exosome-based vaccine is ongoing, with initial topline data expected in Q1 2026[5]. - Topline results from the pivotal HOPE-3 Phase 3 study (n=105) of Deramiocel for Duchenne muscular dystrophy are expected in Q4 2025[5]. - Research and development expenses increased to $20,359,098 in Q3 2025, up from $11,807,867 in Q3 2024, reflecting a 72% increase[26]. Regulatory and Operational Updates - Capricor plans to resubmit its Biologics License Application (BLA) pending the results of the HOPE-3 study, with a review period anticipated to be up to six months[6]. - The company has completed its FDA Pre-License Inspection for its GMP facility, which is now operational and ready for initial commercial launch upon approval[6]. - Deramiocel has received Orphan Drug Designation from both the U.S. FDA and the European Medicines Agency for the treatment of Duchenne muscular dystrophy[18]. Shareholder Information - The weighted average number of shares outstanding increased to 45,716,151 in Q3 2025 from 33,090,063 in Q3 2024[26]. - Capricor's available cash is expected to cover anticipated expenses into Q4 2026, excluding any additional potential milestone payments[13].

Core Insights - Capricor Therapeutics is approaching a critical phase in its development with the upcoming topline results from the HOPE-3 Phase 3 trial of Deramiocel for Duchenne muscular dystrophy (DMD) [2][4][5] - The company has prepared for a potential commercial launch of Deramiocel in 2026, contingent on the trial results and regulatory approval [4][5] - Financial results indicate a significant increase in operating expenses and a net loss for the third quarter of 2025 compared to the same period in 2024 [9][10] Company Updates - The topline results from the HOPE-3 Phase 3 study, which includes 105 participants, are expected in Q4 2025 [4][5] - Following a recent Type A meeting with the FDA, Capricor plans to resubmit its Biologics License Application (BLA) using the HOPE-3 results to address previous concerns raised in a Complete Response Letter [5] - The company has completed its FDA Pre-License Inspection for its commercial manufacturing facility, which is now operational and ready for initial product launch [5] Financial Performance - As of September 30, 2025, Capricor reported cash, cash equivalents, and marketable securities totaling approximately $98.6 million, down from $151.5 million at the end of 2024 [7][25] - Revenues for Q3 2025 were $0, a decrease from approximately $2.3 million in Q3 2024, with total revenues for the first nine months of 2025 also at $0 compared to approximately $11.1 million in the same period of 2024 [8] - Total operating expenses for Q3 2025 were approximately $26.3 million, up from approximately $15.3 million in Q3 2024, leading to a net loss of approximately $24.6 million for Q3 2025 compared to a net loss of approximately $12.6 million in Q3 2024 [9][10] Upcoming Developments - Capricor is preparing for a commercial launch of Deramiocel in 2026, pending the results of the HOPE-3 trial [4][5] - The company is also advancing its StealthX™ exosome-based vaccine platform, with initial data from a Phase 1 clinical trial expected in Q1 2026 [4][6]