Core Insights - The webinar focuses on Capricor Therapeutics' Phase 3 trial, HOPE-3, which is assessing the investigational product deramiocel for treating Duchenne muscular dystrophy [1]. Group 1: Company Overview - Capricor Therapeutics is represented by its CEO Linda Marban, Chief Medical Officer Michael Binks, and Chief Development Officer Mark Awadalla during the webinar [2]. Group 2: Event Structure - The presentation includes a Q&A session at the end, allowing participants to submit questions via a chat function [3]. - The webinar is recorded and will be available on PPMD's website for future reference [3].

Core Insights - Capricor Therapeutics (CAPR) shares surged 281.9% this week following positive results from a late-stage study of deramiocel for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) [1][6] - Deramiocel is the company's lead product candidate and the only asset in its clinical pipeline, representing an investigational, allogeneic cardiac-derived cell therapy [1] Study Details - The phase III HOPE-3 study involved 106 boys and young men with DMD, averaging 15 years old, who received either intravenous deramiocel at 150 million cells per infusion or placebo every three months for a year while on stable corticosteroid therapy [2] - The study demonstrated a 54% slowing of disease progression in upper-limb function compared to placebo, indicating significant preservation of daily functional abilities [3] - The study also achieved a 91% slowing in the deterioration of left-ventricular ejection fraction, addressing a major unmet need in DMD patients [4] Market Performance - Over the past year, Capricor Therapeutics shares have increased by 71%, contrasting with a 0.8% decline in the industry [5] Regulatory Context - In July 2025, Capricor faced a regulatory setback when the FDA issued a complete response letter (CRL) regarding the biologics license application (BLA) for deramiocel, citing insufficient clinical evidence [9][10] - The company believes the new phase III HOPE-3 results strengthen deramiocel's clinical profile and plans to submit a formal response to the FDA, which had indicated that positive findings could support resubmission [11][12] Disease Context - DMD is a severe genetic disorder affecting approximately 15,000 individuals in the U.S., primarily boys, leading to progressive muscle loss and often resulting in cardiomyopathy and heart failure [13]

Capricorn Therapeutics (CAPR) shares more than quadrupled on Dec. 3 after the biotech company announced late-stage HOPE-3 clinical trial results for deramiocel, its investigational treatment for Duchenne Muscular Dystrophy. The positive results prompted notable short-seller Martin Shkreli to publicly acknowledge his “bad call” on CAPR, reversing his earlier prediction that the HOPE-3 trial would fail. More News from Barchart His admission underscores the binary nature of biotech investments, where succe ...

Core Insights - Capricor Therapeutics, Inc. experienced a significant stock surge of 356%, reaching $29.02, following the announcement of positive topline data from the Phase 3 HOPE-3 trial for its lead cell therapy candidate, deramiocel [1][10] - The stock rally resulted in a "short squeeze," leading to substantial losses for short sellers, particularly Martin Shkreli, who had publicly bet against the company's success [2][9] Company Developments - Capricor's CEO, Linda Marbán, stated that the HOPE-3 results provide "strong and definitive evidence" that deramiocel can significantly improve the course of Duchenne muscular dystrophy, and the company plans to use this data to address a previous Complete Response Letter (CRL) from the FDA [8] - The positive data release has been characterized as a game-changer for Capricor, marking a major defeat for those who had shorted the stock [9] Market Reactions - Martin Shkreli, a prominent short-seller, had previously identified Capricor as a target for shorting, predicting the stock would fall to around $2 per share, but the positive trial results contradicted his expectations [3][4] - Following the data release, Shkreli criticized the company, alleging that it did not meet a "prespecified primary endpoint" and suggesting that the reported results were based on "post-hoc analyses" [6][7]

Summary of Capricor Therapeutics FY Conference Call Company Overview - **Company**: Capricor Therapeutics (NasdaqCM: CAPR) - **Focus**: Development of deramiocel for treating cardiomyopathy caused by Duchenne muscular dystrophy (DMD) [1][2] Key Points and Arguments Clinical Data and Efficacy - **HOPE-3 Trial**: Positive results reported from a randomized double-blind placebo-controlled trial involving 106 patients, with 105 completing the study [4][5] - **Primary Endpoint**: Achieved statistical significance with a p-value of 0.03 for the Performance of the Upper Limb 2.0, indicating a clinically relevant 1.2-point change [5][6] - **Secondary Endpoint**: Left ventricular ejection fraction also showed statistical significance with a p-value of 0.04, indicating potential for treating heart disease in DMD patients [6][7] - **Patient Response**: 40% of patients showed improvement in both cardiac and skeletal muscle function, while over 70% had improvement in either [14] Regulatory and Approval Process - **Complete Response Letter (CRL)**: The FDA previously issued a CRL citing insufficient data from earlier studies; however, the new HOPE-3 data is expected to address these concerns [10][11] - **Resubmission Timeline**: Capricor plans to respond to the CRL by the end of the calendar year, anticipating a PDUFA date around July of the following year [11][12] Market Opportunity - **Partnership with Nippon Shinyaku**: Capricor has a sales and distribution agreement, which includes an $80 million milestone payment upon approval and royalties between 30%-50% [16][17] - **Target Population**: DMD affects approximately 15,000 to 20,000 boys and young men in the U.S., presenting a significant market opportunity [17] - **Pricing Strategy**: Capricor aims to price deramiocel competitively within the range of existing exon-skipping therapies, which could lead to a robust revenue model [17] Manufacturing and Expansion Plans - **Manufacturing Facility**: A new commercial-scale facility in San Diego is ready to meet initial demand for about 500 patients, with plans to expand capacity for 2,500 patients annually [21][25] - **Potential for Exosome Development**: Capricor is exploring the use of exosomes for advanced genetic medicine, leveraging their manufacturing capabilities to scale up production [33][34] Future Indications - **Expansion into Becker's Muscular Dystrophy**: Plans to seek accelerated approval for Becker's, which shares similar cardiomyopathy characteristics with DMD, are in development [27][28] - **Broader Neuromuscular Disease Applications**: Capricor is considering expanding deramiocel's application to other neuromuscular diseases with cardiac components [29] Additional Important Information - **Financial Position**: Capricor ended the third quarter with nearly $100 million in cash, with potential additional funding from milestone payments and sales [31] - **Clean Capital Structure**: The company has no debt, positioning it well for future growth and development [31] This summary encapsulates the critical insights from the conference call, highlighting Capricor Therapeutics' advancements, market potential, and strategic plans for the future.

Capricor Therapeutics Conference Call Summary Company Overview - **Company**: Capricor Therapeutics (NasdaqCM: CAPR) - **Product**: Deramiocel, a therapy for Duchenne muscular dystrophy (DMD) Key Industry Insights - **Duchenne Muscular Dystrophy (DMD)**: An X-linked disease primarily affecting boys and young men, characterized by the absence of dystrophin, leading to muscle degeneration and early death, typically within 25-30 years. - **Current Treatment Landscape**: There are no approved therapies specifically for the non-ambulant DMD patient population, highlighting a significant unmet medical need. Core Findings from HOPE-3 Trial - **Trial Design**: - Phase 3, randomized, double-blind, placebo-controlled trial involving 106 patients across 20 sites in the U.S. - Primary endpoint: Performance of the Upper Limb (PUL) version 2.0. - Key secondary endpoint: Left ventricular ejection fraction (LVEF). - **Efficacy Results**: - **PUL Improvement**: A 54% slowing of disease progression with a 1.2-point change on an absolute scale, achieving statistical significance (p-value = 0.029) [22][24]. - **LVEF Improvement**: A 91% slowing of disease progression, with a statistically significant p-value of 0.041 [24][26]. - **Statistical Significance**: All Type 1 error controlled secondary endpoints were met, indicating robust efficacy data [27]. - **Safety Profile**: - Mild flu-like symptoms reported in 25%-35% of patients, easily managed with antihistamines and Tylenol. - No serious adverse events reported, contributing to low dropout rates in trials [20][21]. Mechanism of Action - **Deramiocel's Mechanism**: - Exhibits anti-fibrotic and immunomodulatory activities, reducing inflammation and promoting muscle repair. - Validated by FDA-approved potency assays, ensuring consistency in manufacturing [14][34]. Regulatory Path and Future Plans - **Response to FDA**: - Following a Complete Response Letter (CRL) from the FDA, Capricor plans to submit new data from the HOPE-3 trial to address previous concerns regarding efficacy and safety [35][48]. - Anticipated PDUFA date for resubmission is around July, with hopes for accelerated review based on the new data [35][49]. - **Market Potential**: - The therapy is positioned to address both cardiac and skeletal muscle aspects of DMD, with over 70% of patients showing improvement in either domain [60]. - Capricor aims to maintain the cardiomyopathy indication while also seeking to expand labeling to include skeletal muscle myopathy based on trial results [60]. Additional Insights - **Community Impact**: The data has generated significant hope among families affected by DMD, with potential to improve both quality and quantity of life for patients [6][37]. - **Collaboration with Other Therapies**: Deramiocel can be used in conjunction with existing treatments, potentially enhancing overall therapeutic outcomes for DMD patients [32][33]. Conclusion - Capricor Therapeutics has made significant strides in developing deramiocel for DMD, with promising trial results that could reshape treatment options for this underserved patient population. The company is preparing for regulatory submissions and is optimistic about the future of deramiocel in clinical practice.

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].

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]

Core Insights - Kuehn Law, PLLC is investigating potential breaches of fiduciary duties by certain officers and directors of Capricor Therapeutics, Inc. (NASDAQ: CAPR) [1] - The investigation is based on a federal securities lawsuit alleging that insiders provided misleading information regarding the company's lead cell therapy candidate, deramiocel, for treating cardiomyopathy associated with Duchenne muscular dystrophy [1] - The lawsuit claims that while Capricor communicated its ability to obtain a Biologics License Application (BLA) from the FDA, it also concealed adverse facts related to the safety and efficacy data from its Phase 2 HOPE-2 trial [1] Company Investigation - The investigation focuses on whether Capricor's management misled investors about the prospects of deramiocel [1] - The allegations include the dissemination of false statements and the concealment of material adverse facts regarding the drug's four-year safety and efficacy data [1] Shareholder Actions - Shareholders who purchased CAPR shares prior to October 9, 2024, are encouraged to contact Kuehn Law for potential legal action [2] - Kuehn Law offers to cover all case costs and does not charge its investor clients, emphasizing the importance of shareholder participation in maintaining market integrity [3]