-Enrollment Complete in Cohort A of Phase 3, Pivotal, HOPE-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy; Announced Successful Interim Futility Analysis; On Track to Report Top-Line Data in Q4 2024-
-Upcoming Type-B Meeting with FDA in Q1 2024 to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy-
-CAP-1002 Scale-Up Expansion Underway at New San Diego Manufacturing Facility in Preparation for Commercial Launch-
-Plan to Report 3-Year Data from HOPE-2 Open-Label Extension (OLE) Trial in Q2 2024-
-Announced Selection by Project NextGen for a Clinical Trial of Our Novel Exosome-Based Multivalent Vaccine in Collaboration with the National Institutes of Health-
-Conference Call and Webcast Today at
“2023 was a pivotal year for
Dr. Marbán, continued, “We continue to progress our proprietary StealthX™ exosome platform technology as part of our strategy to leverage exosomes for therapeutic development. We recently announced a collaboration for our exosome-based multivalent vaccine for the prevention of SARS-CoV-2 with the
Fourth Quarter 2023 and Recent Operational Highlights
CAP-1002 Duchenne Muscular Dystrophy Program: CAP-1002 is an investigational cell therapy in Phase 3 development for the treatment of DMD. CAP-1002 aims to slow disease progression through immunomodulatory, anti-inflammatory, and anti-fibrotic actions, with the goal of potentially improving skeletal and cardiac muscle function in patients with DMD. HOPE-3, our Phase 3 study, is a multi-center, randomized, double-blind, placebo-controlled clinical trial comprised of two cohorts evaluating the safety and efficacy of CAP-1002 in participants with DMD and impaired skeletal muscle function. The trial is being conducted in
- Enrollment has been completed for Cohort A in our Phase 3 trial which enrolled 61 subjects randomized to either CAP-1002 or placebo in a 1:1 ratio.
- Reported a positive outcome from the interim futility analysis for Cohort A which triggered the first milestone payment of
$10.0 million under ourU.S. Distribution and Commercialization Agreement with Nippon Shinyaku. There is an additional$90.0 million in potential milestone payments up to the time of approval which are triggered upon certain regulatory-based achievements. Following potential approval, there is an additional$605.0 million in potential milestones payments which may be payable toCapricor based on various sales-based targets being met. Further,Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement. - Next steps for Cohort A: plan to report top-line data in the fourth quarter of 2024.
- Enrollment is underway for Cohort B designed to enroll approximately 44 subjects randomized to either CAP-1002 or placebo in a 1:1 ratio.
- Next steps for Cohort B: expect to complete enrollment in the second quarter of 2024.
- Announced the scale-up to expand manufacturing capacity of CAP-1002 to our new
San Diego facility, intended for commercial use, subject to regulatory approval. This facility was designed to be a versatile and cost-effective way to bring CAP-1002 to market efficiently and it is expected that our enhanced manufacturing capacity will increase our supply capabilities and improve our margins on ultimate product sales, if any. We are currently producing CAP-1002 doses at ourSan Diego facility for use in Cohort B. - Announced a positive outcome from a Type-B meeting held with FDA in the third quarter of 2023. In the meeting, the FDA affirmed alignment on the current HOPE-3 clinical trial design comprised of two cohorts and our plan to submit a BLA supported by results from Cohort A which uses product manufactured from our
Los Angeles manufacturing facility. - We plan to meet with FDA in the first quarter of 2024 to continue discussing our pathway to BLA. In the upcoming Type-B meeting, we intend to discuss our further CMC plans for commercial launch, if approved, with an aim to expedite the approval pathway to our BLA filing. Our ultimate goal is to transition to our
San Diego manufacturing facility for commercial manufacturing as quickly as possible. - Hosted a webinar in conjunction with Parent Project Muscular Dystrophy (PPMD) where key updates on our DMD program were outlined.
- Presented a late-breaking poster at the 28th International Annual Congress of the
World Muscle Society (WMS). Highlights from the poster included data from the HOPE-2 OLE trial measured by the Performance of the Upper Limb (PUL 2.0) showing a delta change=4.9 points, p=0.021 after 24-months of treatment, compared with the placebo patient group.
Exosome Program: Exosomes are membrane-bound extracellular vesicles which are secreted by most cells and contain characteristic lipids, proteins and nucleic acids such as mRNA and microRNAs. Exosomes act as messengers to regulate the functions of neighboring or distant cells and have been shown to regulate functions such as cell survival, proliferation, inflammation and tissue regeneration. We are developing our exosome technology, using our proprietary StealthX™ platform which is 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.
- Announced that our proprietary StealthX™ exosome-based multivalent vaccine (StealthX™ vaccine) for the prevention of SARS-CoV-2 was selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines providing broader and more durable protection for COVID-19. As part of Project NextGen, NIAID, part of the
National Institutes of Health , will conduct and fund a Phase 1 clinical trial with our StealthX™ vaccine, subject to regulatory approval. Under the terms of the collaboration,Capricor will supply the investigational product andNIAID's Division of Microbiology and Infectious Diseases (DMID) will oversee the trial. - Next steps for this project: NIAID plans to initiate the Phase 1 clinical trial in late 2024, subject to regulatory approval. If NIAID finds that our StealthX™ vaccine meets its criteria for safety and efficacy, they may consider our program for a funded Phase 2.
- Currently, in collaboration with an undisclosed pharmaceutical company, we are also investigating the therapeutic application of our StealthX™ exosome platform.
- Presented a late-breaking poster at the WMS on the application of our StealthX™ exosome platform for the delivery of antisense oligonucleotides (ASO). Highlights from the poster included data showing the presence of exosomes loaded with a labeled ASO in the lower limbs of mice 24 hours post-intravenous (IV) injection. Notably, the exosomes carrying the muscle-targeting moiety were not detected in any other tissues except for the expected clearance pathways (kidney and liver) with a single dose.
Corporate Updates
- Announced receipt of our first milestone payment of
$10.0 million under ourU.S. Distribution and Commercialization Agreement with Nippon Shinyaku. - Announced completion of a registered direct offering with participation from Nippon Shinyaku for gross proceeds of approximately
$23.0 million .
Anticipated Upcoming Milestones
CAP-1002 Duchenne Muscular Dystrophy Program
- Plan to have a Type-B meeting with FDA in the first quarter of 2024 to discuss commercial manufacturing planning with an aim to expedite the BLA pathway.
- Plan to report 3-year HOPE-2 OLE data in the second quarter of 2024.
- Plan to complete HOPE-3 (Cohort B) enrollment in the second quarter of 2024.
- Plan to report topline data from HOPE-3 (Cohort A) in the fourth quarter of 2024.
- Continue to explore opportunities for additional partnerships outside of the
U.S. andJapan to support the potential commercialization of CAP-1002 in DMD.
Exosome Program
- Subject to regulatory approval, plan to announce IND clearance for our StealthX™ vaccine for the prevention of SARS-CoV-2.
- Plan to provide updates on our NIAID collaboration for our StealthX™ vaccine as they become available.
- Continue to explore opportunities for partnerships and non-dilutive sources of funding to support advancement of our StealthX™ exosome platform technology.
Fourth Quarter and Full Year 2023 Financial Results
Cash position: The Company’s cash, cash equivalents and marketable securities totaled approximately
Revenues: Capricor’s primary source of revenue was from the ratable recognition of the
Operating expenses: Total operating expenses for the fourth quarter of 2023 were approximately
Net loss: The Company reported a net loss of approximately
Financial Outlook
Conference Call and Webcast
To participate in the conference call, please dial 1-888-886-7786 (Domestic/Toll-Free) or 1-416-764-8658 (International) and reference the conference ID: 83986877. 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 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
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; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue 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
For more information, please contact:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||||||||||
(UNAUDITED) | ||||||||||||||||
Three months ended | Year ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
REVENUE | ||||||||||||||||
Revenue | $ | 12,088,089 | $ | 959,903 | $ | 25,178,066 | $ | 2,551,469 | ||||||||
TOTAL REVENUE | 12,088,089 | 959,903 | 25,178,066 | 2,551,469 | ||||||||||||
OPERATING EXPENSES | ||||||||||||||||
Research and development | 9,940,167 | 6,231,806 | 36,448,039 | 21,816,949 | ||||||||||||
General and administrative | 3,210,947 | 2,794,442 | 12,589,619 | 10,431,903 | ||||||||||||
TOTAL OPERATING EXPENSES | 13,151,114 | 9,026,248 | 49,037,658 | 32,248,852 | ||||||||||||
LOSS FROM OPERATIONS | (1,063,025 | ) | (8,066,345 | ) | (23,859,592 | ) | (29,697,383 | ) | ||||||||
OTHER INCOME (EXPENSE) | ||||||||||||||||
Other income | 67,657 | - | 67,657 | 190,582 | ||||||||||||
Investment income | 233,932 | 379,699 | 1,510,434 | 521,535 | ||||||||||||
Loss on disposal of fixed assets | (653 | ) | (34,266 | ) | (6,041 | ) | (34,266 | ) | ||||||||
TOTAL OTHER INCOME (EXPENSE) | 300,936 | 345,433 | 1,572,050 | 677,851 | ||||||||||||
NET LOSS | (762,089 | ) | (7,720,912 | ) | (22,287,542 | ) | (29,019,532 | ) | ||||||||
OTHER COMPREHENSIVE INCOME (LOSS) | ||||||||||||||||
Net unrealized gain (loss) on marketable securities | 122,605 | (17,223 | ) | 130,569 | 105,244 | |||||||||||
COMPREHENSIVE LOSS | $ | (639,484 | ) | $ | (7,738,135 | ) | $ | (22,156,973 | ) | $ | (28,914,288 | ) | ||||
Net loss per share, basic and diluted | $ | (0.02 | ) | $ | (0.31 | ) | $ | (0.83 | ) | $ | (1.18 | ) | ||||
Weighted average number of shares, basic and diluted | 30,664,100 | 25,163,711 | 26,778,360 | 24,552,688 | ||||||||||||
SUMMARY BALANCE SHEETS | ||||||||
Cash, cash equivalents and marketable securities | $ | 39,487,703 | $ | 41,421,262 | ||||
Total assets | $ | 58,734,327 | $ | 50,094,910 | ||||
Total liabilities | $ | 36,132,860 | $ | 38,308,816 | ||||
Total stockholders' equity - 31,148,320 and 25,241,402 common shares issued and | ||||||||
outstanding at | 22,601,467 | 11,786,094 | ||||||
Total liabilities and stockholders' equity | $ | 58,734,327 | $ | 50,094,910 | ||||
Source:
2024 GlobeNewswire, Inc., source