Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ Quarterly Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 for the quarterly period ended March 31, 2021 or ☐ Transition Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 for the transition period from to Commission File Number: 001-34058 CAPRICOR THERAPEUTICS, INC. (Exact Name Of Registrant As Specified In Its Charter) Delaware 88-0363465 (State or o ...

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q1 2021 Earnings Conference Call May 13, 2021 4:30 PM ET Company Participants AJ Bergmann - Chief Financial Officer Linda Marbán - Chief Executive Officer Conference Call Participants Alan Leong - BioWatch News Operator Greetings. And welcome to Capricor Therapeutics First Quarter 2021 Earnings and Corporate Update Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions ...

Financial Performance - Grant income for the years ended December 31, 2020 and 2019 was approximately $0.2 million and $0.5 million, respectively, indicating a decrease of approximately 60% in 2020 compared to 2019[535]. - General and Administrative (G&A) expenses for the years ended December 31, 2020 and 2019 were approximately $5.5 million and $3.6 million, respectively, reflecting an increase of approximately 52.8% in 2020[537]. - The increase in G&A expenses in 2020 was primarily due to a $1.2 million increase in stock-based compensation expense and a $0.5 million increase in salaries and recruiting related expenses[537]. - Investment income decreased from $94,791 in 2019 to $32,943 in 2020, a decline of approximately 65.3% due to reduced interest rates[540]. - The company reported a net loss of approximately $13.7 million for the year ended December 31, 2020, compared to a net loss of $10.4 million in 2019[548]. - Cash used in operating activities rose to approximately $10.1 million in 2020 from $6.8 million in 2019, an increase of about 48.5%[548]. - The company raised approximately $33.0 million from the sale of common stock in 2020, compared to $9.2 million in 2019, marking an increase of about 258.7%[550]. Research and Development - Research and Development (R&D) expenses increased from approximately $5.1 million in 2019 to $8.5 million in 2020, a rise of about 66.7%[539]. - R&D expenses primarily include salaries, clinical trial costs, and manufacturing costs, with expenses recognized as incurred[583]. - The accrual policy for R&D activities aims to match expenses with actual services received, with variations in payment flows due to contract negotiations[585]. - The company monitors activity levels through communication with CROs and vendors, ensuring expenses align with budgeted amounts[584]. - The clinical trial accrual process is designed to reflect appropriate expenses in financial statements based on the progress of trials[592]. Clinical Trials and Product Development - The company has completed the Phase II clinical trial (HOPE-2) for CAP-1002, showing improvements in multiple measures of upper limb, cardiac, and respiratory functions[507]. - The FDA has encouraged the company to conduct a Phase III study for CAP-1002, although discussions regarding the pathway forward are still ongoing[507]. - The INSPIRE trial for CAP-1002 in severe or critical COVID-19 patients is a Phase II, randomized, double-blind, placebo-controlled study enrolling up to 60 patients[509]. - The company is developing two vaccine candidates for COVID-19, including a tripartite exosome-mRNA vaccine, with plans to file an IND by the third quarter of 2021[510]. - The company has submitted an IND for CAP-2003 to investigate its use in patients with Duchenne Muscular Dystrophy (DMD) and is evaluating the next steps for this program[514]. Funding and Grants - The Company received a CIRM Grant Award of approximately $3.4 million for its Phase I/II clinical trial, requiring a co-funding of about $2.3 million from the Company[565]. - As of December 31, 2020, the liability balance for the CIRM Award was approximately $3.4 million, with all milestones completed by June 2019[567]. - The NIH grant for CAP-2003 amounted to approximately $4.2 million, with $0.7 million incurred by June 30, 2019, and the award was closed in Q2 2019[568]. - The Department of Defense grant totaled approximately $2.4 million, with about $2.3 million utilized, and a no-cost extension granted until September 29, 2020[569]. Cash and Investments - Cash, cash equivalents, and marketable securities increased from approximately $9.9 million in 2019 to $32.7 million in 2020, a growth of about 230.5%[544][550]. - The net increase in cash and cash equivalents for 2020 was approximately $28.8 million, compared to a decrease of $646,000 in 2019[547]. - The fair value of cash and cash equivalents as of December 31, 2020, was approximately $32.7 million[598]. - The investment portfolio primarily consists of money market funds and short-term U.S. treasuries, aiming to limit credit exposure and default risk[598]. - The company does not hedge interest rate exposure and believes that a hypothetical 100 basis point change in interest rates would not significantly impact the fair value of its investment portfolio[599]. Stock-Based Compensation - Stock-based compensation expense is recognized over the vesting period, with significant future non-cash compensation expenses expected[591]. - The company accounts for stock options using the Black-Scholes option-pricing model, requiring assumptions about various variables[589].

Financial Data and Key Metrics Changes - As of December 31, 2020, the company's cash, cash equivalents, and marketable securities totaled approximately $32.7 million, compared to approximately $9.9 million on December 31, 2019, indicating significant growth in liquidity [47] - The net loss for the fourth quarter of 2020 was approximately $4.2 million, compared to a net loss of approximately $1.5 million for the fourth quarter of 2019, reflecting increased operational costs [49] - For the full year of 2020, the net loss was approximately $13.7 million, compared to a net loss of approximately $7.6 million for the full year 2019, highlighting a trend of increasing losses [49] Business Line Data and Key Metrics Changes - The research and development expense for Q4 2020 was approximately $2.7 million, up from approximately $800,000 in Q4 2019, indicating a substantial increase in investment in R&D [48] - General and administrative expenses also rose to approximately $1.1 million in Q4 2020 from approximately $800,000 in Q4 2019, reflecting higher operational costs [48] Market Data and Key Metrics Changes - The company is focused on becoming a leading exosome RNA company, with plans to develop engineered exosomes and synthetic mRNAs for therapeutic applications [13][35] - The exosome platform is positioned to address a broad spectrum of diseases, leveraging the success of mRNA vaccines as a market opportunity [9][10] Company Strategy and Development Direction - The company is pivoting from traditional cell therapy towards an exosome platform, which is expected to provide significant opportunities in vaccine and therapeutic development [72] - The strategy includes developing a multi-antigen exosome-mRNA vaccine, with plans to initiate a Phase 1 clinical trial in Q3 of 2021, pending FDA approval [37] - The company aims to optimize its exosome platform for both vaccine and therapeutic applications, with a focus on monogenic diseases and other infectious diseases [35][38] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in 2020 and the potential of the exosome platform to revolutionize therapeutic delivery [5][45] - The company is awaiting FDA feedback on its pre-IND package and is prepared to move into clinical trials based on that feedback [37][57] - Management emphasized the importance of CAP-1002 for Duchenne muscular dystrophy and the ongoing discussions with the FDA regarding its approval pathway [41][42] Other Important Information - The company has entered into a Sponsored Research Agreement with Johns Hopkins University to support its exosome program, enhancing its research capabilities [6] - The company is exploring partnerships for CAP-1002, targeting corporate partners with existing products in the Duchenne muscular dystrophy space [61] Q&A Session Summary Question: How should Capricor be viewed going forward? - Management sees Capricor positioned between traditional cell therapy companies and those focused on engineered exosomes, emphasizing the shift towards RNA medicines in exosomes [52] Question: Will the mRNA strategy focus on internal development or partnerships? - The plan includes both internal programs and attracting partners for collaborative development, aiming for a plug-and-play model [54] Question: What is the expected cold chain requirement for the exosome-based mRNA vaccine? - Current expectations are for similar cold chain requirements as existing mRNA vaccines, with potential for a single-dose option being explored [55] Question: What work is still required for entering the clinic with the vaccine? - The company is ready to proceed into the clinic based on FDA feedback, leveraging its experience with regulatory processes [57] Question: What is the regulatory pathway for exosome-based therapeutics? - Management believes the regulatory process will be relatively straightforward, drawing on previous experiences with biologics [59] Question: What is the ideal partner for CAP-1002 development? - An ideal partner would have activity or commercial products in the Duchenne muscular dystrophy space, with ongoing discussions with multiple parties [61] Question: Plans for label expansion on post-COVID patients in the INSPIRE trial? - The company is open to label expansion based on trial data and the evolving landscape of COVID-19 treatment [63] Question: What additional studies are being conducted with CAP-1002? - The company is working with consultants and statisticians to analyze the impact of CAP-1002 in Duchenne muscular dystrophy [65] Question: Insights on the exosome platform compared to lipid nanoparticles? - The exosome platform shows promise in safety, repeat dosing, and targeting capabilities, with ongoing research to optimize these aspects [68][70]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q x Quarterly Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 for the quarterly period ended September 30, 2020 or ¨ Transition Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 for the transition period from to Commission File Number: 001-34058 CAPRICOR THERAPEUTICS, INC. (Exact Name Of Registrant As Specified In Its Charter) Delaware 88-0363465 (State or other jurisdict ...

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q3 2020 Earnings Conference Call November 12, 2020 4:30 PM ET Company Participants A.J. Bergmann - Chief Financial Officer Linda Marbán - President, Chief Executive Officer and Director Steve Gould - Executive Consultant Conference Call Participants Emanuela Branchetti - H.C. Wainwright Jason McCarthy - Maxim Group Alan Leong - BioWatch News Operator Greetings, and welcome to the Capricor Therapeutics Inc Third Quarter 2020 Earnings Call. [Operator Instructions]. As ...

Corporate & Investor Presentation Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside NASDAQ: CAPR August 2020 Forward-Looking Statements Statements in this presentation 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; regulator ...

Clinical Trials and Drug Development - Capricor Therapeutics completed the HOPE-2 Phase II clinical trial for CAP-1002, showing statistically significant improvements in upper limb, cardiac, and respiratory function with p-values less than 0.05[203] - The 12-month data from the HOPE-2 trial indicated a mean change of 2.4 points in the PUL 2.0 for CAP-1002 treated patients compared to placebo, with a p-value of 0.05[203] - In the same trial, CAP-1002 demonstrated a reduction in CK-MB levels (p=0.006), indicating a decrease in cardiac muscle cell damage[204] - The primary efficacy endpoint of the HOPE-2 trial was the relative change in patients' abilities to perform manual tasks, measured through the Performance of the Upper Limb (PUL) test[200] - CAP-1002 was generally safe and well tolerated, with no significant safety signals identified during the HOPE-2 trial[208] - The Phase I/II HOPE-Duchenne clinical trial involved 25 patients, with a total dose of 75 million cells infused, showing significant improvement in motor function in 89% of lower-functioning patients treated with CAP-1002[216][217] - The FDA has granted CAP-1002 Orphan Drug Designation and Rare Pediatric Disease Designation, providing incentives such as tax credits and potential market exclusivity upon approval[219][220] - CAP-1002 is one of the few therapies in development for non-ambulant patients with DMD, with the FDA encouraging a Phase III study for its approval pathway[214][222] - Capricor has initiated a Phase II, randomized, placebo-controlled study for CAP-1002 in severe or critical COVID-19 patients, aiming to treat up to 60 patients[183] - The company is developing two vaccine candidates using its exosomes platform technology for potential COVID-19 prevention, with pre-clinical studies showing an antibody response[184] Financial Performance and Funding - Grant income for Q2 2020 was approximately $50,000, down from $144,000 in Q2 2019, primarily due to the timing of the DoD Grant Award expected to end in September 2020[237] - Miscellaneous income for Q2 2020 was zero, compared to approximately $300,000 in Q2 2019, attributed to delays in clinical trials sponsored by CSMC due to the COVID-19 pandemic[238] - The company has no commercial product sales to date and relies on grants and equity sales for funding its development programs[232] - The company reported a net loss of approximately $5.6 million for the six months ended June 30, 2020[249] - The company has raised approximately $20.2 million from the sale of 3,059,959 shares under the May 2020 ATM Program at an average price of approximately $6.59 per share[258] - The company has not generated any revenue from the commercial sale of its products to date and does not expect to generate revenue for several years, necessitating the need for substantial additional capital[254] - The company expects to spend approximately $4.0 million to $6.0 million in 2020 on the further development of CAP-1002 for DMD and COVID-19[244] - The company anticipates spending approximately $3.0 million to $6.0 million in 2020 on pre-clinical and other research expenses related to its exosomes program[246] Expenses and Financial Management - Research and development expenses primarily consist of clinical trial costs, personnel costs, and other related expenses, which are expected to increase as clinical development progresses[233] - General and Administrative (G&A) expenses for Q2 2020 were approximately $1.6 million, an increase of 100% compared to $0.8 million in Q2 2019, primarily due to a $0.5 million rise in stock-based compensation[240] - Research and Development (R&D) expenses for Q2 2020 were approximately $1.9 million, up 18.75% from $1.6 million in Q2 2019, driven by costs related to the COVID-19 exosome vaccine program[242] - Cash used in operating activities for the six months ended June 30, 2020, was approximately $3.2 million, a slight decrease from $3.4 million in the same period of 2019[250] - The company expects to record additional non-cash compensation expense in the future, which may be significant[292] Cash and Investments - Total cash, cash equivalents, and marketable securities as of June 30, 2020, were approximately $36.3 million, a significant increase from $9.9 million as of December 31, 2019, primarily due to net financing activities of approximately $29.7 million[249] - Cash flow from financing activities for the six months ended June 30, 2020, was approximately $29.7 million, compared to $2.0 million for the same period in 2019, reflecting a substantial increase due to the May 2020 ATM Program[253] - As of June 30, 2020, the fair value of the company's cash and cash equivalents was approximately $36.3 million[300] - The company issued a letter of credit amounting to $232,803 for its lease agreement, which was subsequently canceled and funds returned[294] Grants and Awards - The Company received a CIRM Grant Award of approximately $3.4 million for a clinical trial, requiring co-funding of approximately $2.3 million from the Company[267] - As of June 30, 2020, the liability balance for the CIRM Award was approximately $3.4 million, with all milestones completed in June 2019[269] - The Company was awarded approximately $2.4 million from the U.S. Department of Defense to develop a scalable process for manufacturing CAP-2003, with approximately $2.3 million incurred under the award as of June 30, 2020[270] - The Company accounts for the CIRM Award as a liability rather than income, as it may be required to repay some or all of the awarded amounts[283] Risk Management and Accounting - The company manages market risk by investing in highly rated credit issuers and does not hedge interest rate exposure[301] - The company's investment policy aims to limit credit exposure and improve the safety of invested funds[301] - The company calculates the fair value of stock options using the Black-Scholes option-pricing model, accounting for various assumptions[290] - Clinical trial expenses are accrued based on contracts with vendors and are adjusted as actual results differ from estimates[295] - The Company utilizes estimates for clinical trial costs based on contracts with various vendors, which may lead to significant differences between estimated and actual costs[285] - The Company has not recognized any adjustments for material changes in estimates in any presented period[287] - The company accounts for stock-based compensation expense in general and administrative or research and development expenses[292]

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q2 2020 Earnings Conference Call August 6, 2020 4:30 PM ET Company Participants Anthony Bergmann - CFO & Corporate Treasurer Linda Marbán - Co-Founder, President, CEO & Director Conference Call Participants Naureen Quibria - Maxim Group Alan Leong - BioWatch Operator Greetings, and welcome to the Capricor Therapeutics Second Quarter 2020 Earnings Call. [Operator Instructions]. As a reminder, this conference is being recorded Thursday, August 6, 2020. I would now lik ...

Capricor Therapeutics Inc. (NASDAQ:CAPR) Q1 2020 Earnings Conference Call May 14, 2020 4:30 PM ET Corporate Participants AJ Bergmann - CFO Linda Marban - CEO Conference Call Participants Emanuela Branchetti - H.C. Wainwright Alan Leong - BioWatch Operator Greetings and welcome to the Capricor Therapeutics Inc. First Quarter 2020 Earnings Conference Call. During the presentation, all participations will be in a listen-only mode. Afterward, we will conduct a question-and-answer session. [Operator Instructions ...