Capricor Therapeutics Reports Second Quarter 2022 Financial Results and Provides Corporate Update

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) has initiated the pivotal Phase 3 HOPE-3 clinical trial for CAP-1002, targeting patients with late-stage Duchenne Muscular Dystrophy (DMD). Positive results from the HOPE-2 open label extension study showcase significant improvements in muscle function. The company is pursuing FDA discussions for CAP-1002's development. Financially, Capricor reported a net loss of $7.1 million for Q2 2022, but has sufficient cash to fund operations into Q2 2024. A partnership with Nippon Shinyaku supports HOPE-3 with a $30 million upfront payment.

Positive

• Initiation of pivotal Phase 3 trial HOPE-3 for CAP-1002 in late-stage DMD.
• Positive one-year results from HOPE-2 study showing significant muscle function improvement (p=0.02).
• Partnership with Nippon Shinyaku includes $30M upfront payment and potential milestone payments up to $705M.
• Sufficient cash reserves of $51.4 million to cover expenses into Q2 2024.

Negative

• Net loss increased to $7.1 million in Q2 2022 from $4.7 million in Q2 2021.

-First Patient Treated in HOPE-3, the Pivotal Phase 3 Clinical Trial of CAP-1002 in Duchenne Muscular Dystrophy-

-Positive One-Year Safety and Efficacy Results From HOPE-2 Open Label Extension Study of CAP-1002 in Non-Ambulant Duchenne Muscular Dystrophy Patients Presented at PPMD’s 2022 Annual Conference-

-Requesting Meeting with FDA to Discuss Next Steps in Development of CAP-1002 for Duchenne Muscular Dystrophy-

-Pipeline Expansion Underway Using Engineered Exosomes-

-To Host Conference Call and Webcast Today at 4:30 p.m. ET-

SAN DIEGO, Aug. 10, 2022 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, announced today its financial results for the second quarter ended June 30, 2022 and provided a corporate update.

“The second quarter was marked by important progress across our ongoing development program for CAP-1002 in patients with late-stage Duchenne Muscular Dystrophy (DMD), highlighted by the initiation of our pivotal Phase 3 trial, HOPE-3, as well as the positive one-year results from our HOPE-2 open label extension (OLE) study,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Positive data from the HOPE-2 OLE study were recently presented at this year’s Parent Project Muscular Dystrophy (PPMD) Annual Conference and further validate the statistically significant and clinically relevant evidence of CAP-1002’s ability to improve skeletal muscle function, with disease modification and long-term safety potential in patients with later stages of DMD. Together, with the data generated to date, we believe CAP-1002 is positioned to become an anchor therapy for non-ambulant DMD patients. Based on this recently announced data and our RMAT designation, we are requesting a meeting with FDA later this year to discuss the best path forward with the objective of getting CAP-1002 to approval as quickly as possible.”

Dr. Marbán continued, “In parallel, our engineered exosome platform continues to advance in several areas. We are internally developing a streamlined manufacturing paradigm to support future development efforts. This foundational work will drive our platform strategy to leverage the development of external partnerships where companies have therapeutics that can benefit from a targeted delivery vehicle which can be obtained using engineered exosomes. We look forward to sharing further updates, including the expansion into new indications.”

Second Quarter Highlights and Recent Operational Highlights

• Treated first patient in HOPE-3, our pivotal Phase 3 clinical trial of CAP-1002 in late-stage DMD. The randomized, double-blind, placebo-controlled study is designed to enroll approximately 70 patients across approximately 15 to 20 sites in the United States (NCT05126758).
  • Partnership with Nippon Shinyaku Co., Ltd., which has commercialization and distribution rights in the United States, provides funding for the support of HOPE-3 ($30M upfront payment with potential for additional milestone payments of up to $705M).
• Reported positive one-year results from our HOPE-2 open label extension study in patients with later-stage DMD. Treatment with CAP-1002 resulted in statistically significant clinical benefits in skeletal muscle function, demonstrated by improvements on the Performance of the Upper Limb (PUL version 2.0) scale (p=0.02). Additionally, results underscore the disease modifying and long-term safety potential of CAP-1002 in patients with DMD.
  • Results were presented at the PPMD Annual Conference on June 25, 2022 in Scottsdale, Arizona.
• Appointed Xavier Avat as Chief Business Officer, who brings deep industry knowledge, to oversee Capricor’s strategic priorities, the development and potential commercialization of its pipeline products and business development activities.
  

Anticipated Milestones and Events

The Company has set forth the following guidance for pipeline progression:

• Requesting meeting with FDA to discuss next steps in our CAP-1002 DMD program
• Publication of additional preclinical work related to our engineered exosomes platform
• Full enrollment of HOPE-3 expected by the third quarter of 2023
  

Upcoming Events

The Company plans to participate in the following events:

• H.C. Wainwright 24^th Annual Global Investment Conference: September 12-14
• Cantor Fitzgerald Cell and Genetic Medicines Conference: September 15
• 4^th Extracellular Vesicle Based Therapeutic Development: October 4-6
  

Financial Results for Second Quarter 2022

The Company reported a net loss of approximately $7.1 million, or $0.29 per share, for the second quarter of 2022, compared to a net loss of approximately $4.7 million, or $0.21 per share, for the second quarter of 2021.

As of June 30, 2022, the Company’s cash, cash equivalents and marketable securities totaled approximately $51.4 million, compared to approximately $34.9 million on December 31, 2021. No shares were sold under the Company’s at-the-market program in the second quarter of 2022.

Financial Outlook

Capricor believes that based on the current operating plan and financial resources, the Company expects that its available cash and cash equivalents will be sufficient to cover anticipated expenses and capital requirements into the second quarter of 2024. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.

Conference Call and Webcast

To participate in the conference call, please dial 800-289-0571 (Domestic/Toll-Free) or 929-477-0324 (International) and reference the conference ID: 6015762. To participate via a webcast, please click here. The webcast will be archived for approximately 30 days and will be available at http://capricor.com/news/events/

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Capricor is also developing its exosome technology as a next-generation therapeutic platform. The Company’s current focus is on developing exosomes capable of delivering nucleic acids, including mRNA, as well as proteins to treat or prevent a variety of diseases. For more information, visit capricor.com, and follow the Company 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; 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, 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 December 31, 2021, as filed with the Securities and Exchange Commission on March 11, 2022 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as filed with the Securities and Exchange Commission on May 11, 2022. 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.

CAP-1002 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 / Shana Marino
KCSA Strategic Communications
rcona@kcsa.com / smarino@kcsa.com
212.896.1204 / 347.487.6189

Capricor Investor Contact:
Joyce Allaire
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com
617.435.6602

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
310.358.3200

CAPRICOR THERAPEUTICS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(UNAUDITED)

	Three months ended June 30,								Six months ended June 30,
		2022				2021				2022				2021
REVENUE
Revenue	$	-			$	204,082			$	-			$	244,898
TOTAL REVENUE		-				204,082				-				244,898
OPERATING EXPENSES
Research and development		4,965,088				3,497,275				10,080,787				6,793,597
General and administrative		2,356,666				1,789,974				5,072,501				3,695,556
TOTAL OPERATING EXPENSES		7,321,754				5,287,249				15,153,288				10,489,153
LOSS FROM OPERATIONS		(7,321,754	)			(5,083,167	)			(15,153,288	)			(10,244,255	)
OTHER INCOME (EXPENSE)
Other income		190,582				-				190,582				-
Investment income		21,761				16,741				35,201				25,906
Forgiveness of debt		-				318,160				-				318,160
TOTAL OTHER INCOME (EXPENSE)		212,343				334,901				225,783				344,066
NET LOSS		(7,109,411	)			(4,748,266	)			(14,927,505	)			(9,900,189	)
OTHER COMPREHENSIVE INCOME (LOSS)
Net unrealized gain on marketable securities		22,830				-				22,830				-
COMPREHENSIVE LOSS	$	(7,086,581	)		$	(4,748,266	)		$	(14,904,675	)		$	(9,900,189	)
Net loss per share, basic and diluted	$	(0.29	)		$	(0.21	)		$	(0.61	)		$	(0.44	)
Weighted average number of shares, basic and diluted		24,324,156				22,861,051				24,303,564				22,546,634

CAPRICOR THERAPEUTICS, INC.
SUMMARY BALANCE SHEETS

		June 30, 2022 (unaudited)			December 31, 2021
Cash, cash equivalents and marketable securities		$	51,416,950		$	34,885,274
Total assets		$	58,065,574		$	41,330,323
Total liabilities		$	39,336,579		$	9,962,357
Total stockholders’ equity - 24,334,952 and 24,185,001 common shares issued
and outstanding at June 30, 2022 and December 31, 2021, respectively			18,728,995			31,367,966
Total liabilities and stockholders’ equity		$	58,065,574		$	41,330,323

FAQ

What are the recent developments for Capricor's CAP-1002?

Capricor has initiated the pivotal Phase 3 HOPE-3 trial and reported positive one-year results from the HOPE-2 study.

What financial results did Capricor report for Q2 2022?

Capricor reported a net loss of approximately $7.1 million for Q2 2022, an increase from $4.7 million in Q2 2021.

What is the duration of Capricor's financial resources?

Capricor expects its available cash to cover anticipated expenses into the second quarter of 2024.

What is the significance of the partnership with Nippon Shinyaku for Capricor?

The partnership provides $30 million upfront and potential milestone payments up to $705 million, supporting the HOPE-3 trial.

When is Capricor hosting their conference call regarding recent updates?

Capricor hosted a conference call on August 10, 2022, at 4:30 p.m. ET.