Capricor Therapeutics Reports Third Quarter 2024 Financial Results and Provides Corporate Update
Capricor Therapeutics initiated a rolling BLA submission for deramiocel to treat DMD-cardiomyopathy, with full submission expected by year-end 2024. The company signed a binding term sheet with Nippon Shinyaku for European commercialization, potentially worth up to $715 million in milestones plus a $20 million upfront payment. Q3 2024 financial results showed revenues of $2.3 million and a net loss of $12.6 million. The company completed an $86 million public offering and expects cash runway into 2027. Positive 3-year data from HOPE-2 OLE trial demonstrated improvements in cardiac function and PUL v2.0 scores.
Capricor Therapeutics ha avviato una presentazione BLA a rilascio graduale per deramiocel per trattare la cardiomiopatia da DMD, con la presentazione completa prevista entro la fine del 2024. L'azienda ha firmato un accordo vincolante con Nippon Shinyaku per la commercializzazione in Europa, il cui valore potenziale può raggiungere i 715 milioni di dollari in traguardi, oltre a un pagamento iniziale di 20 milioni di dollari. I risultati finanziari del Q3 2024 hanno mostrato ricavi di 2,3 milioni di dollari e una perdita netta di 12,6 milioni di dollari. L'azienda ha completato un'offerta pubblica di 86 milioni di dollari e prevede di avere liquidità fino al 2027. Dati positivi a 3 anni dallo studio HOPE-2 OLE hanno dimostrato miglioramenti nella funzione cardiaca e nei punteggi PUL v2.0.
Capricor Therapeutics inició una presentación de BLA de forma gradual para deramiocel con el fin de tratar la cardiomiopatía relacionada con la DMD, con la presentación completa esperada para finales de 2024. La empresa firmó un acuerdo vinculante con Nippon Shinyaku para la comercialización en Europa, lo que podría valer hasta 715 millones de dólares en hitos más un pago inicial de 20 millones de dólares. Los resultados financieros del Q3 2024 mostraron ingresos de 2,3 millones de dólares y una pérdida neta de 12,6 millones de dólares. La empresa completó una oferta pública de 86 millones de dólares y espera tener liquidez hasta 2027. Los datos positivos a 3 años del ensayo HOPE-2 OLE demostraron mejoras en la función cardíaca y en las puntuaciones PUL v2.0.
Capricor Therapeutics는 DMD 심근병 치료를 위한 deramiocel의 단계적 BLA 제출을 시작했으며, 전체 제출은 2024년 연말까지 완료될 예정입니다. 이 회사는 일본 신약(Nippon Shinyaku)과 유럽 상업화를 위한 구속력 있는 조건서에 서명했으며, 이로 인해 최대 7억 1500만 달러의 이정표와 2000만 달러의 선불 지급이 있을 것으로 예상됩니다. 2024년 3분기 재무 결과는 230만 달러의 수익과 1260만 달러의 순손실을 보여주었습니다. 이 회사는 8천600만 달러 규모의 공모를 완료했으며, 2027년까지 현금 흐름을 예상하고 있습니다. HOPE-2 OLE 시험의 3년 긍정적 데이터는 심장 기능 및 PUL v2.0 점수의 개선을 보여주었습니다.
Capricor Therapeutics a lancé une soumission BLA progressive pour deramiocel afin de traiter la cardiomyopathie liée à la DMD, avec une soumission complète prévue d'ici fin 2024. L'entreprise a signé une feuille de termes contraignante avec Nippon Shinyaku pour la commercialisation en Europe, pouvant valoir jusqu'à 715 millions de dollars en jalons, plus un paiement initial de 20 millions de dollars. Les résultats financiers du Q3 2024 ont montré des revenus de 2,3 millions de dollars et une perte nette de 12,6 millions de dollars. L'entreprise a réalisé une offre publique de 86 millions de dollars et prévoit des liquidités jusqu'en 2027. Des données positives sur 3 ans de l'essai HOPE-2 OLE ont montré des améliorations de la fonction cardiaque et des scores PUL v2.0.
Capricor Therapeutics hat eine schrittweise BLA-Einreichung für deramiocel zur Behandlung der DMD-Kardiomyopathie initiiert, wobei die vollständige Einreichung bis Ende 2024 erwartet wird. Das Unternehmen hat ein verbindliches Vertragsblatt mit Nippon Shinyaku für die europäische Vermarktung unterzeichnet, das potenziell bis zu 715 Millionen Dollar in Meilensteinen sowie eine Vorauszahlung von 20 Millionen Dollar wert sein könnte. Die finanziellen Ergebnisse des Q3 2024 zeigten Einnahmen von 2,3 Millionen Dollar und einen Nettoverlust von 12,6 Millionen Dollar. Das Unternehmen hat ein öffentliches Angebot von 86 Millionen Dollar abgeschlossen und erwartet, bis 2027 über ausreichend Liquidität zu verfügen. Positive 3-Jahres-Daten aus der HOPE-2 OLE-Studie zeigten Verbesserungen in der Herzfunktion und bei den PUL v2.0-Werten.
- Initiated rolling BLA submission for deramiocel with potential PDUFA date in H2 2025
- Secured European commercialization deal with Nippon Shinyaku worth potential $715M in milestones plus $20M upfront
- Strengthened balance sheet with $86M public offering, extending cash runway into 2027
- Positive 3-year HOPE-2 OLE trial data showing improved cardiac function
- Potential Priority Review Voucher eligibility upon deramiocel approval
- Revenue decreased to $2.3M in Q3 2024 from $6.2M in Q3 2023
- Net loss increased to $12.6M in Q3 2024 from $6.4M in Q3 2023
- Operating expenses increased to $15.3M from $13.1M year-over-year
Insights
The Q3 2024 results and corporate updates reveal significant positive momentum for Capricor Therapeutics. Key highlights include:
- Strong cash position of
$85.0 million , bolstered by a successful public offering raising$86.3 million gross proceeds - Strategic partnership with Nippon Shinyaku for European expansion, potentially worth
$1.5 billion in milestone payments - Advancement of deramiocel BLA submission with potential PDUFA date in H2 2025
- Extended cash runway into 2027
However, revenues decreased to
The clinical progress of deramiocel shows promising developments in treating DMD-cardiomyopathy. The 3-year HOPE-2 OLE data demonstrated significant improvements in cardiac function, particularly in left ventricular ejection fraction. The PUL v2.0 total score showed a statistically significant benefit of +3.7 points (p<0.001) compared to external controls, indicating meaningful clinical improvement.
The strategic decision to combine Cohorts A and B of Phase 3 HOPE-3 for post-approval study demonstrates confidence in existing data while potentially streamlining the path to additional indications. The receipt of RMAT designation and potential Priority Review Voucher eligibility further validates the therapy's promise. The expansion of manufacturing capacity suggests preparation for anticipated commercial demand, though execution will be critical.
- Announced intent to file a biologics license application (BLA) for deramiocel to treat all patients with DMD-cardiomyopathy
- Rolling BLA submission initiated in October 2024 with full submission expected by year-end 2024
- Company anticipates potential PDUFA date in second half of 2025
- Announced signing of binding term sheet with Nippon Shinyaku for European expansion and commercialization of deramiocel; potential milestones from combined agreements would total approximately
$1.5 billion payable to Capricor - Reported positive long-term data from HOPE-2 OLE trial at 2024 World Muscle Society Congress
- StealthX™ exosome platform expanded to include PMO loading and targeting for the treatment of DMD presented at AAEV Annual Meeting
- Completed public offering of common stock for gross proceeds of approximately
$86 million ; cash runway is expected to support current planned operations into 2027 - Capricor to host conference call and webcast today at 4:30 p.m. ET
SAN DIEGO, Nov. 13, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced its financial results for the third quarter ended September 30, 2024 and provided a corporate update.
“This has been a transformational quarter for Capricor as we move towards potential commercialization of deramiocel for the treatment of DMD. We have commenced the submission of our BLA which we expect to be complete by year end and we have significantly strengthened our balance sheet in order to scale up manufacturing as we anticipate a strong launch, pending FDA approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “In addition, we continue to advance our proprietary StealthX™ platform technology as part of our long-term strategy to leverage exosomes as vehicles for targeted delivery of payloads for therapeutic development. We continue to explore partnership opportunities and other non-dilutive sources of funding to advance this program.”
Recent Updates and Upcoming Milestones
Deramiocel DMD Program: Deramiocel is an investigational cell therapy in late-stage development for the treatment of DMD. Deramiocel for the treatment of DMD has received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT) from the FDA in the U.S. In addition, if deramiocel is approved, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on our previous receipt of a rare pediatric disease designation.
- Based on FDA feedback and following Capricor’s recent pre-BLA meeting in August, Capricor initiated the rolling BLA submission in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected to be complete by year end 2024.
- The BLA submission will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 open label extension (OLE) trials compared to patient-level natural history data.
- To support potential label expansion to treat DMD, Capricor plans to provide clinical data on skeletal muscle myopathy by combining Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was originally planned to occur in the fourth quarter of 2024.
- Furthermore, if required, the HOPE 3 study may also potentially support ex-U.S. marketing authorizations. Currently, Capricor has initiated regulatory activities in Europe and Japan and will be working with the various regional health authorities to develop the most efficient path forward for regulatory approval of deramiocel in these regions.
- Capricor is actively working towards expansion of our commercial manufacturing capacity and throughput.
- In addition, Capricor is exploring expansion opportunities for its GMP production for additional capacity that may be necessary to meet product demand in the U.S. and other regions.
- Capricor signed a binding term sheet with Nippon Shinyaku for European expansion and commercialization of deramiocel for the treatment of DMD. The potential transaction covered by the term sheet would be similar to the existing Commercialization and Distribution Agreements with Nippon Shinyaku in the U.S. and Japan with an opportunity for increased global product reach.
- Subject to finalization of the Definitive Agreement, Capricor will receive an upfront payment of
$20 million along with potential additional development and sales-based milestone payments to Capricor of up to$715 million and a double-digit share of product revenue.
- Subject to finalization of the Definitive Agreement, Capricor will receive an upfront payment of
- In September 2024, Nippon Shinyaku purchased approximately
$15 million of Capricor common stock at a20% premium to the 60-day volume weighted average price of Capricor’s common stock. - Capricor presented positive 3-year safety and efficacy results from its ongoing HOPE-2 OLE in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society.
- The 3-year data from HOPE-2 OLE demonstrated improvements in multiple measures of cardiac function, including left ventricular ejection fraction (LVEF%), as well as indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes.
- In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients.
StealthX™Exosome Platform: Exosomes are membrane-bound extracellular vesicles and contain lipids, proteins and nucleic acids. They act as messengers to regulate the functions of neighboring or distant cells. Capricor is developing our engineered exosome technology using our proprietary StealthX™ platform focused on the areas of targeted therapeutics and vaccines to potentially treat and prevent a diverse array of diseases.
- The Company’s proprietary StealthX™ exosome-based 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.
- Under the terms of the initiative, Capricor will supply its vaccine candidate for clinical use and NIAID's Division of Microbiology and Infectious Diseases will conduct the trial.
- Currently, manufacturing is underway for our StealthX™ vaccine with current plans to deliver it to NIAID in the first quarter of 2025.
- NIAID plans to initiate the trial in the first quarter of 2025, and Capricor expects that it will have preliminary data available in the second quarter of 2025, subject to FDA approval of the NIAID’s IND.
- If NIAID finds that our StealthX™ vaccine meets its criteria for safety and efficacy, they may consider funding our program for a Phase 2 study.
- Capricor presented preclinical data at the 2024 American Association of Extracellular Vesicles (AAEV) Annual Meeting highlighting a potential exosome-based approach by delivering phosphorodiamidate morpholino oligomers (PMOs) for the treatment of DMD.
Third Quarter 2024 Financial Results
Cash position: Cash, cash equivalents and marketable securities totaled approximately
Revenues: Revenues for the third quarter of 2024 were approximately
Expenses: Total operating expenses for the third quarter of 2024 were approximately
Net loss: The Company reported a net loss of approximately
Financial Outlook: The Company believes that based on the current operating plan and financial resources, our available cash, cash equivalents and marketable securities will be sufficient to cover anticipated expenses and capital requirements into 2027. This expectation excludes any additional potential milestone payments under the Commercialization and Distribution Agreements with Nippon Shinyaku, as well as any strategic use of capital not currently in the Company’s base-case planning assumptions.
Upcoming Events
The Company plans to present at the following upcoming events:
- B. Riley Securities NextGen Tissue Delivery Modalities Summit, November 14, 2024, Virtual
- Piper Sandler 36th Annual Healthcare Conference, December 3-5, 2024, New York, NY
- Oppenheimer Movers in Rare Disease Summit, December 12, 2024, New York, NY
Conference Call and Webcast
To participate in the conference call, please dial 1-800-717-1738 (Domestic/Toll-Free) or 1-646-307-1865 (International) and reference the conference ID: 68076. 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 the event. 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 Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development 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. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
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; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and 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 December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
For more information, please contact:
Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
| CAPRICOR THERAPEUTICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (UNAUDITED) | |||||||||||||||
| Three months ended September 30, | Nine months ended September 30, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| REVENUE | |||||||||||||||
| Revenue | $ | 2,261,642 | $ | 6,185,814 | $ | 11,139,956 | $ | 13,089,977 | |||||||
| TOTAL REVENUE | 2,261,642 | 6,185,814 | 11,139,956 | 13,089,977 | |||||||||||
| OPERATING EXPENSES | |||||||||||||||
| Research and development | 11,807,867 | 10,028,964 | 35,413,649 | 26,507,872 | |||||||||||
| General and administrative | 3,463,655 | 3,021,450 | 10,593,308 | 9,378,672 | |||||||||||
| TOTAL OPERATING EXPENSES | 15,271,522 | 13,050,414 | 46,006,957 | 35,886,544 | |||||||||||
| LOSS FROM OPERATIONS | (13,009,880 | ) | (6,864,600 | ) | (34,867,001 | ) | (22,796,567 | ) | |||||||
| OTHER INCOME (EXPENSE) | |||||||||||||||
| Investment income | 453,152 | 479,380 | 1,516,418 | 1,276,502 | |||||||||||
| Loss on disposal of fixed assets | - | (5,388 | ) | - | (5,388 | ) | |||||||||
| TOTAL OTHER INCOME (EXPENSE) | 453,152 | 473,992 | 1,516,418 | 1,271,114 | |||||||||||
| NET LOSS | (12,556,728 | ) | (6,390,608 | ) | (33,350,583 | ) | (21,525,453 | ) | |||||||
| OTHER COMPREHENSIVE INCOME (LOSS) | |||||||||||||||
| Net unrealized gain (loss) on marketable securities | (58,766 | ) | (66,485 | ) | (139,592 | ) | 7,964 | ||||||||
| COMPREHENSIVE LOSS | $ | (12,615,494 | ) | $ | (6,457,093 | ) | $ | (33,490,175 | ) | $ | (21,517,489 | ) | |||
| Net loss per share, basic and diluted | $ | (0.38 | ) | $ | (0.25 | ) | $ | (1.04 | ) | $ | (0.85 | ) | |||
| Weighted average number of shares, basic and diluted | 33,090,063 | 25,817,676 | 32,099,181 | 25,468,880 | |||||||||||
| CAPRICOR THERAPEUTICS, INC. SUMMARY BALANCE SHEETS | ||||||||
| September 30, 2024 (unaudited) | December 31, 2023 | |||||||
| Cash, cash equivalents and marketable securities | $ | 85,028,624 | $ | 39,487,703 | ||||
| Total assets | $ | 92,951,613 | $ | 58,734,327 | ||||
| Total liabilities | $ | 24,686,654 | $ | 36,132,860 | ||||
| Total stockholders' equity - 40,332,392 and 31,148,320 common shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively | 68,264,959 | 22,601,467 | ||||||
| Total liabilities and stockholders' equity | $ | 92,951,613 | $ | 58,734,327 | ||||
FAQ
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