Clinical Development - CAP-1002 is in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD), with top-line data expected in Q4 2024[658] - The ongoing HOPE-2 open label extension study has shown statistically significant improvements in the Performance of the Upper Limb (PUL) scale at one year (p=0.02) and two years (p=0.021)[660] - Enrollment for Cohort B of the Phase 3 trial is underway, targeting approximately 44 participants randomized in a 1:1 ratio to receive either CAP-1002 or placebo, with completion expected in Q2 2024[661] - Cohort A of the Phase 3 trial has completed enrollment with 61 subjects, and an interim futility analysis resulted in a favorable recommendation to continue the trial[686] - The FDA Type-B meeting in Q3 2023 confirmed alignment on the Phase 3 HOPE-3 program and discussed plans for a Biologics License Application (BLA) submission[662] - The company plans to readout top-line data for Cohort A in the fourth quarter of 2024 and expects to complete enrollment for Cohort B in the second quarter of 2024[723] - The company plans to meet with the FDA in the first quarter of 2024 to discuss the pathway to BLA submission and further CMC plans for commercial launch[723] Financial Performance - Net losses for the year ended December 31, 2023, were $22.3 million, compared to $29.0 million for 2022, with an accumulated deficit of $159.4 million as of December 31, 2023[691] - Cash used in operating activities was approximately $25.6 million for the year ended December 31, 2023, compared to cash provided by operating activities of approximately $4.9 million for the year ended December 31, 2022, resulting in a net change of approximately $30.5 million[769] - The net loss for the year ended December 31, 2023, was $22.29 million, compared to a net loss of $29.02 million in 2022, indicating an improvement of 23.1%[817] - The company expects to continue incurring substantial losses, which will generate negative net cash flows from operating activities, as it develops product candidates and engages in further research and development activities[769] Funding and Capital - The company completed a registered direct offering in October 2023, raising gross proceeds of approximately $23.0 million[666] - The company anticipates needing substantial additional capital to fund operations and clinical trials, with potential dilution for existing shareholders if additional equity securities are issued[672] - The company expects to incur expenses of approximately $25.0 million to $35.0 million in 2024 for the ongoing HOPE-3 Phase 3 study and related activities for CAP-1002[707] - The company anticipates needing substantial additional funding to support ongoing operations and development of CAP-1002 and other product candidates[696] Research and Development - Research and development expenses increased by approximately $14.6 million, or 67%, in 2023, totaling $36.4 million, primarily driven by costs associated with the DMD program[701] - Research and development expenses increased by $11.2 million primarily due to the enrollment of the HOPE-3 clinical program and expanded manufacturing efforts for CAP-1002[729] - R&D expenses consist primarily of salaries, clinical trial costs, and other related expenses, with costs expensed as incurred except for certain capitalized intangible assets[761] Agreements and Partnerships - The company is developing exosome-based vaccines and therapeutics for various diseases, with a focus on securing partnerships for clinical development[665] - The company entered into a Commercialization and Distribution Agreement with Nippon Shinyaku, receiving an upfront payment of $12.0 million and potential milestone payments of up to approximately $89.0 million[744] - The U.S. Distribution Agreement includes potential milestones totaling up to $90.0 million leading to BLA approval and sales-based milestones tied to annual net sales of CAP-1002 of up to $605.0 million[715] - Capricor entered into a Commercialization and Distribution Agreement with Nippon Shinyaku on February 10, 2023, appointing Nippon Shinyaku as its exclusive distributor in Japan for CAP-1002[773] Cash and Assets - Cash and cash equivalents as of December 31, 2023, were $14.7 million, with total cash, cash equivalents, and marketable securities at approximately $39.5 million, down from $41.4 million in 2022[712] - As of December 31, 2023, the company had cash, cash equivalents, and marketable securities totaling approximately $39.5 million[720] - Total current assets increased to $50.86 million as of December 31, 2023, compared to $42.89 million in the previous year, representing a growth of 18.5%[814] - Cash and cash equivalents rose to $14.69 million from $9.60 million, marking an increase of 53.5% year-over-year[814] - Marketable securities decreased to $24.79 million from $31.82 million, a decline of 22.1%[814] - Total liabilities decreased to $36.13 million from $38.31 million, a reduction of 5.7%[814] - Stockholders' equity increased significantly to $22.60 million from $11.79 million, reflecting an increase of 92.5%[814] Income and Revenue - The company reported clinical development income of approximately $25.2 million for the year ended December 31, 2023, compared to $2.6 million in 2022[727] - Investment income for 2023 was approximately $1.7 million, up from $0.5 million in 2022, attributed to increased interest rates and higher principal balances[706] - Deferred revenue increased to $24.27 million from $17.98 million, a rise of 35.0% year-over-year[814] - The company has not generated any revenues from the commercial sale of products and will not be able to do so until receiving FDA approval for its drug candidates[720] - The company has not yet achieved commercial sales of its drug candidates, but the new revenue recognition standard is applicable to its distribution agreements[786] Regulatory and Compliance - The proprietary StealthX™ exosome-based vaccine for SARS-CoV-2 is part of Project NextGen, with a Phase 1 clinical trial planned for late 2024, pending regulatory approval[669] - The company is expanding the manufacturing capacity of CAP-1002 at its new San Diego facility, which is intended for commercial use, subject to regulatory approval[723] - The company accounts for the CIRM grant disbursements as long-term liabilities, recognizing them as a liability rather than income until certain conditions are met[760] - The Financial Accounting Standards Board issued ASU 2023-06, which affects various topics within the Accounting Standards Codification, with the company currently evaluating its impact on financial statement disclosures[827] Investment and Risk Management - The company’s investment portfolio primarily consists of money market funds and short-term U.S. treasuries[799] - The investment policy aims to limit credit exposure by investing in highly rated credit issuers, focusing on preserving invested funds and mitigating default risk[829] - The company does not hedge interest rate exposure and believes that a hypothetical 100 basis point change in interest rates would not materially impact the fair value of its investment portfolio[829] - The company has been actively monitoring clinical trial costs through detailed invoice reviews and budget analyses[821] - The company is required to estimate accrued expenses related to clinical trials, reflecting appropriate expenses based on patient progression and trial completion[825]

Shares of Capricor Therapeutics (CAPR) have gained 32.6% over the past four weeks to close the last trading session at $5.19, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $18.75 indicates a potential upside of 261.3%.The mean estimate comprises four short-term price targets with a standard deviation of $14.45. While the lowest estimate of $8 indicates a 54.1% increase from th ...

SAN DIEGO, March 06, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company will present the positive 24-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at this year's MDA Clinical and Scientific Conference which is taking place in Orland ...

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q4 2023 Earnings Conference Call February 29, 2024 4:30 PM ET Company Participants AJ Bergmann - Chief Financial Officer Linda Marban - Chief Executive Officer Conference Call Participants Joseph Pantginis - H.C. Wainright Kristen Kluska - Cantor Fitzgerald Aydin Huseynov - Ladenburg Operator Good afternoon, ladies and gentlemen, and welcome to Capricor Therapeutics Fourth Quarter 2023 Earnings Call. At this time, all lines are in listen-only mode. Following the pre ...

Financial Performance - Capricor reported total revenue of approximately $12.1 million for Q4 2023, a significant increase from approximately $1.0 million in Q4 2022, representing a growth of over 1,100%[10] - The company’s total operating expenses for Q4 2023 were approximately $13.2 million, compared to approximately $9.0 million in Q4 2022, reflecting a year-over-year increase of about 46%[14] - Capricor's net loss for Q4 2023 was approximately $0.8 million, or $0.02 per share, a substantial improvement from a net loss of approximately $7.7 million, or $0.31 per share, in Q4 2022[15] - For the full year 2023, Capricor reported a net loss of approximately $22.3 million, or $0.83 per share, compared to a net loss of approximately $29.0 million, or $1.18 per share, for the full year 2022[15] Cash and Milestone Payments - The company’s cash, cash equivalents, and marketable securities totaled approximately $39.5 million as of December 31, 2023, down from approximately $41.4 million on December 31, 2022[13] - Capricor received its first milestone payment of $10.0 million under its U.S. Distribution and Commercialization Agreement with Nippon Shinyaku in January 2024[13] - Positive outcome from interim futility analysis triggered a $10 million milestone payment under the U.S. Distribution and Commercialization Agreement with Nippon Shinyaku[32] - Potential for $90 million in additional milestone payments prior to approval and $605 million post-approval based on sales targets[32] Clinical Trials and FDA Meetings - The company plans to have a Type-B meeting with the FDA in Q1 2024 to discuss commercial manufacturing planning for CAP-1002[1] - Capricor plans to report topline data from the HOPE-3 (Cohort A) study in Q4 2024[1] - Enrollment completed in Cohort A of Phase 3 HOPE-3 trial with 61 subjects randomized in a 1:1 ratio[32] - Cohort B enrollment underway, aiming for approximately 44 subjects, expected to complete by Q2 2024[32] - FDA Type-B meeting in Q1 2024 to discuss expedited BLA pathway for CAP-1002[26] Partnerships and Collaborations - The company is exploring additional partnerships outside of the U.S. and Japan to support the commercialization of CAP-1002 for DMD[1] - Collaboration with NIAID for a Phase 1 clinical trial of an exosome-based multivalent vaccine for SARS-CoV-2[26] Product Development - Capricor's proprietary StealthX™ exosome-based vaccine for SARS-CoV-2 is part of Project NextGen, with a Phase 1 clinical trial to be conducted by NIAID[23] - New San Diego manufacturing facility established to scale up CAP-1002 production for commercial use[32] - Capricor's proprietary StealthX™ platform technology is being developed for targeted delivery of therapeutics[33] Trial Outcomes - Positive data from HOPE-2 OLE trial showing a delta change of 4.9 points (p=0.021) after 24 months of treatment[32] - Anticipated top-line data from Cohort A to be reported in Q4 2024[32]

SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- – Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March. In the upcoming meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for comm ...

-Project NextGen Collaboration will Support Capricor's StealthX™ Exosome Platform and Provide Non-Dilutive Support for Advancement into the Clinic- SAN DIEGO, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, today announced that Capricor's proprietary StealthX™ exosome-based multivalent vaccine (StealthX™ vaccine) for the prevention of SARS-CoV-2 has ...

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q3 2023 Results Conference Call November 14, 2023 4:30 PM ET Company Participants AJ Bergmann - CFO Linda Marbán - CEO Conference Call Participants Joe Pantginis - H.C. Wainwright Aydin Huseynov - Ladenburg Operator Good afternoon, ladies and gentlemen and welcome to the Capricor’s Third Quarter 2023 Financial Results and Corporate Update Call. This conference call is being recorded. I would now like to turn the conference call over to our host, Mr. AJ Bergmann, Cap ...

Financial Performance - For the three months ended September 30, 2023, clinical development income was approximately $6.2 million, up from $1.6 million in the same period of 2022, representing a significant increase [112]. - The company reported net losses of approximately $6.4 million for both the three months ended September 30, 2023, and 2022, and approximately $21.5 million for the nine months ended September 30, 2023, compared to $21.3 million in 2022 [92]. - Net loss for the nine months ended September 30, 2023, was $21,525,453 compared to a net loss of $21,298,620 for the same period in 2022 [191]. - The company recorded a comprehensive loss of approximately $6.5 million for the three months ended September 30, 2023, compared to $6.3 million for the same period in 2022 [226]. Research and Development Expenses - Research and development (R&D) expenses for the nine months ended September 30, 2023, increased by approximately $10.9 million, or 70%, compared to the same period in 2022, primarily driven by ongoing clinical trials and expanded manufacturing efforts [114]. - Research and development (R&D) expenses for the three months ended September 30, 2023 increased by approximately $4.5 million, or 82%, compared to the same period in 2022, totaling $10,028,964 [126]. - Research and development costs for the three months ended September 30, 2023, were approximately $10.0 million, compared to approximately $5.5 million for the same period in 2022 [233]. - For the nine months ended September 30, 2023, research and development costs totaled approximately $26.5 million, up from approximately $15.6 million in 2022 [233]. - CAP-1002 program for Duchenne muscular dystrophy (DMD) incurred expenses of $5,616,093 for the nine months ended September 30, 2023, representing a 174% increase compared to $2,045,997 in the same period in 2022 [126]. Cash Flow and Liquidity - Total cash, cash equivalents, and marketable securities as of September 30, 2023 were approximately $28.5 million, down from approximately $41.4 million as of December 31, 2022 [144]. - Cash used in operating activities for the nine months ended September 30, 2023 was approximately $14.0 million, compared to cash provided of approximately $11.8 million in the same period in 2022 [136]. - Cash and cash equivalents balance at the end of the period was $8,984,667, a decrease from $34,885,274 at the beginning of the period [191]. - The company’s principal uses of cash include research and development expenses, general and administrative expenses, and capital expenditures [185]. - The company’s future capital requirements may be substantial, with potential financing options including equity or debt securities and government grants [198]. Offerings and Financing - The company completed a registered direct offering on October 3, 2023, raising approximately $23.0 million through the sale of 4,935,621 shares at a price of $4.66 per share [107]. - On October 3, 2023, the company entered into Securities Purchase Agreements to issue 4,935,621 shares at a price of $4.66 per share, raising approximately $23.0 million [151]. - The company has sold an aggregate of 2,620,703 shares under its ATM Program at an average price of approximately $5.74 per share for gross proceeds of approximately $15.0 million [152]. - Capricor received an upfront payment of $30.0 million from Nippon Shinyaku under the U.S. Distribution Agreement for CAP-1002, with potential milestones totaling up to $100.0 million [157]. Clinical Trials and Development - CAP-1002's HOPE-3 clinical trial has reached its target enrollment of 58 patients, with Cohort B expected to commence enrollment in the fourth quarter of 2023 [102]. - The company plans to report the outcome from the interim futility analysis of the CAP-1002 program in the fourth quarter of 2023 and top-line data from Cohort A in the fourth quarter of 2024 [93]. - The FDA affirmed alignment on the current HOPE-3 clinical trial design during a Type-B clinical meeting held in the third quarter of 2023, supporting the submission of a Biologics License Application (BLA) for CAP-1002 [94]. General and Administrative Expenses - General and administrative (G&A) expenses for the three months ended September 30, 2023 increased by approximately $0.5 million, or 18%, compared to the same period in 2022, totaling $3,021,450 [122]. - Total general and administrative expenses for Q3 2023 were $1,235,219, compared to $943,075 in Q3 2022, representing a 31% increase [276]. Other Financial Metrics - The company has an accumulated deficit of approximately $158.6 million as of September 30, 2023, and expects to incur significant expenses and operating losses for the foreseeable future [92]. - Total liabilities as of September 30, 2023 were approximately $38.9 million, with $26.4 million related to deferred revenue [144]. - The company has a liability balance of approximately $3.4 million related to the CIRM Award as of September 30, 2023 [154]. - The company has not recognized an income tax benefit due to uncertainty regarding realization [260].

Capricor Therapeutics, Inc. (NASDAQ:CAPR) Q2 2023 Earnings Call Transcript August 7, 2023 4:20 PM ET Company Participants AJ Bergmann - CFO Linda Marban - CEO Conference Call Participants Joe Pantginis - H.C. Wainwright Operator Good afternoon, ladies and gentlemen and welcome to the Capricor Therapeutics Second Quarter 2023 Financial Results and Corporate Update Call. At this time all lines are in a listen-only mode. Following the presentation we’ll conduct a question-and-answer session. [Operator Instruct ...