U.S. Markets closed

Capricor Presents Additional Positive Data from Ongoing HOPE-2 Study of CAP-1002 in Duchenne Muscular Dystrophy at World Muscle Society: Data Demonstrates Improved PUL 2.0 Performance at 6 Months

--Company Exploring Potential for Accelerated Approval under RMAT Designation, an Expedited Program for Regenerative Therapies--

--Company to Host Conference Call Today at 5:30 AM PT / 8:30 AM ET--

LOS ANGELES, Oct. 07, 2019 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (CAPR), a clinical-stage biotechnology company, in its presentation at the 24th Annual International Congress of the World Muscle Society, presented additional six-month interim data from HOPE-2, its ongoing Phase 2 clinical trial of its lead investigational product, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is Capricor’s cell-based therapeutic candidate whose mechanism of action is immunomodulatory, anti-fibrotic and has been shown to generate new muscle cells.

Data from the pre-specified interim analysis demonstrated that teens and young men in the advanced stages of DMD saw improvements in skeletal, pulmonary, and cardiac measurements after receiving multiple doses of CAP-1002. Specifically, patients showed improvements in the Performance of the Upper Limb (PUL), a tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients. As previously reported, additional independent tests assessing grip strength and tip to tip pinch strength also showed positive results.   

The U.S. Food and Drug Administration (FDA) has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application (BLA). The PUL scale was designed to measure upper limb motor performance across the spectrum of severity of DMD and is specifically focused on patients that are unable to perform the six-minute walk test.

“We are very pleased that the interim analysis from this double-blind placebo-controlled study, has demonstrated meaningful improvements across three clinically relevant endpoints in older patients with limited remaining treatment options,” said Linda Marban, PhD, Chief Executive Officer of Capricor. “We continue to explore ways to utilize our FDA-granted Regenerative Medicine Advanced Therapy (RMAT) designation to pursue accelerated approval of CAP-1002, and we believe the agency’s written guidance on the matter supports our efforts to introduce this potentially groundbreaking therapy to physicians and patients as quickly as possible. We look forward to meeting with FDA later this year to further define our path to commercialization.”

For the interim analysis, data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 3- and 6-month time-point in the intent to treat (ITT) population. Approximately 80% of the patients were non-ambulant and all patients were on a stable regimen of steroids. Demographic and baseline characteristics were similar between the two treatment groups.

The late breaking podium presentation presented the pre-specified top-line, 6-month results from the HOPE-2 clinical trial of CAP-1002. By expanding the skeletal assessment beyond the mid-level and evaluating patients’ PUL “scores” at the upper and distal dimensions, Capricor’s CAP-1002 showed meaningful improvements, as shown in the summary table below. 

Skeletal Assessments at 3 and 6-month time-points (PUL 2.0)

Time-point 3 months 6 months
Treatment CAP-1002
p-value CAP-1002
Shoulder + Mid + Distal Level 0.5 (1.69) -1.2 (1.69) 0.0549 -0.3 (0.52) -2.3 (1.49) 0.0299
Mid + Distal Level 
0.4 (1.30) -0.4 (0.70) 0.1035 0.2 (1.47) -1.4 (0.92) 0.0177
0.1 (0.99) -0.4 (0.52) 0.2202 -0.2 (1.17) -1.1 (0.99) 0.0612

Mean Change from baseline (standard deviation) shown.
ITT (intent to treat) population shown
Comparisons treated vs. placebo using mixed model repeated ANOVA with covariates

To reduce the risk of future adverse events, Capricor initiated a commonly used pre-medication strategy including oral steroids and antihistamines to prevent or mitigate potential allergic reactions during the administration. Since the initiation of the pre-treatment regimen, approximately 40 infusions of investigational drug (CAP-1002 or placebo) have been administered to HOPE-2 patients with only one serious adverse event reported that required an overnight observation of the patient.

“The results from this trial to date are very promising in that the cells appear to positively impact skeletal, pulmonary and cardiac assessments in older DMD patients who have few, if any, remaining treatment options. We are eager to meet with the FDA to discuss the next steps for this promising program,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor, Chair of its Department of Physical Medicine and Rehabilitation and Professor of Pediatrics.

The FDA has granted Capricor’s CAP-1002 RMAT and Orphan Drug Designation, for which the FDA has also granted a Rare Pediatric Disease Designation. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted, covers the broad treatment of DMD. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.

Capricor expects to announce further updates on its clinical development strategy for CAP-1002 in DMD later this year, including an update on guidance it receives from the FDA.

Conference Call and Webcast

Capricor will host a conference call and webcast with slides today, October 7, 2019, at 8:30 a.m. ET to discuss the updated interim results of the HOPE-2 clinical trial. To participate in the conference call, please dial 866-717-4562 (domestic) or 210-874-7812 (international) and reference the access code: 8074349.

To participate via a webcast, please visit: https://edge.media-server.com/mmc/p/uenq8x2j. The webcast will be archived for approximately 30 days and will be available at http://capricor.com/news/events/.

About HOPE-2

HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the company’s lead investigational therapy, CAP-1002 in steroid-treated boys and young men who are in advanced stages of DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immuno-modulatory activity, and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 150 human subjects across several clinical trials.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. The HOPE-Duchenne trial was funded in part by the California Institute for Regenerative Medicine. For more information, visit www.capricor.com.
Keep up with Capricor on social media: www.facebook.com/capricortherapeutics, www.instagram.com/capricortherapeutics/ and https://twitter.com/capricor

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; 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, 2018 as filed with the Securities and Exchange Commission on March 29, 2019, and as amended by its Amendment No. 1 to Annual Report on Form 10-K/A filed with the Securities and Exchange Commission on April 1, 2019, in its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, as filed with the Securities and Exchange Commission on August 8, 2019, and in its Registration Statement on Form S-3 as filed with the Securities and Exchange Commission on October 24, 2018, and as amended by its Amendment No. 1 to Form S-3 filed with the Securities and Exchange Commission on July 17, 2019, together with prospectus supplements thereto. 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. CAP-2003 has not yet been approved for clinical investigation.

For more information, please contact:

AJ Bergmann, Chief Financial Officer

  • Business

    If Coronavirus Is Killing Fewer People Than the Flu, Why Has Beijing Quarantined Millions?

    China's Leaders on the Hot Seat Market Intelligence Report by TIS Group Feb. 21: Over the past several days, we have detailed some, though not all, of the actions being taken by China's government to control the coronavirus outbreak. I was told today that roughly 50,000 people die in China each year from the flu. If the coronavirus is just another flu-like virus and the current death toll really is about 2,000, not 50,000, why then has Beijing locked down 750 million people?

  • Morgan Stanley is paying $13B for E-Trade, or $2,500 per customer. You can earn a $3,500 sign-up bonus for signing with a new broker — with one major catch

    Morgan Stanley is paying $13B for E-Trade, or $2,500 per customer. You can earn a $3,500 sign-up bonus for signing with a new broker — with one major catch

    If the latest Wall Street mega-deal doesn't make you want to switch online brokerage accounts for a lucrative sign-up bonus, maybe it should. Wall Street giant Morgan Stanley announced an agreement Thursday to pay $13 billion to acquire the online brokerage E-Trade which has 5.2 million customer accounts. “The combination will significantly increase the scale and breadth of Morgan Stanley's Wealth Management franchise, and positions Morgan Stanley to be an industry leader in Wealth Management across all channels and wealth segments,” Morgan Stanley said in a statement.

  • ‘Overprotected’ investors could get stung in the next recession, warns top Barclays strategist

    ‘Overprotected’ investors could get stung in the next recession, warns top Barclays strategist

    Rattled by rising non-China coronavirus cases — notably in South Korea — investors appear unwilling to load up on stocks heading into the weekend. Welcome to the world of helicopter investing and our call of the day from Barclays Wealth Management's chief investment officer William Hobbs, who finds overly anxious investors living in the “long shadow” caused by the financial crisis. “The most common narrative in markets is what to do when the next recession comes along — own Treasurys, own gold, own quality to such an extent that my concern would almost be that if the next recession is of a more normal variety, such as a stock retracement of 10% to 15% and moderate declines in unemployment, you'll find that you have overprotected yourself and the underperformance could actually be in the areas which you thought were giving you safety,” Hobbs told MarketWatch.

  • How Does My Spousal Social Security Benefit Work?

    How Does My Spousal Social Security Benefit Work?

    If you have never worked or paid Social Security taxes (or not paid them for long enough), you will not be eligible to receive Social Security retirement benefits on your own account. You will also be able to apply for Medicare at age 65 as long as your spouse is at least 62. If you apply before you have reached full retirement age and your own benefit is larger, you will automatically be paid that amount.

  • Opinion: Bernie Sanders and the Missing $25 Trillion

    Opinion: Bernie Sanders and the Missing $25 Trillion

    During the Democratic debate on Feb. 19, 2020, Pete Buttigieg skewered Bernie Sanders over the high cost of his plans. Image: Mike Blake/Reuters

  • Buffett defends investments in stocks, which fueled record Berkshire profit

    Buffett defends investments in stocks, which fueled record Berkshire profit

    Warren Buffett on Saturday defended Berkshire Hathaway Inc's decision to invest heavily in the stocks of companies such as Apple Inc, as the conglomerate struggled to find whole businesses to buy. Buffett made his assessment in his annual letter to Berkshire shareholders, where the 89-year-old also assured that Berkshire is "100% prepared" for the eventual departures of himself and Vice Chairman Charlie Munger, 96. Berkshire also reported record net income for the year of $81.42 billion, reflecting enormous gains in its common stock holdings, though operating profit fell 3%.

  • Business

    Buffett Spends Record $2.2 Billion Buying Up Berkshire Shares

    Since then, Berkshire has taken a cautious approach to repurchases, buying back only $6.3 billion of stock. JPMorgan Chase & Co., which counts Berkshire among its investors, bought back $6.7 billion on a net basis in the fourth quarter alone. Buffett, Berkshire's chairman and chief executive officer, has sought to redeploy those funds into higher-returning deals or stock purchases, but has been stymied by what he's said are “sky-high” prices for good businesses.Kraft Heinz Co., which counts Berkshire as its largest shareholder, had a tumultuous 2019, with writedowns, management shakeups and downgrades to junk.

  • Business

    Stocks Drop on the Week but Still Look Bubbly. Time to Prepare for a Correction.

    The last time we got to these levels, the bubble burst and a 50%-plus global bear market began,” Consider the trading action in the market's most popular stocks. The FAANGs— (FB) (ticker: FB), (AAPL) (AAPL), (AMZN) (AMZN), (NFLX) (NFLX), and Google parent (GOOGL) (GOOGL)—are up 10% year to date, adding more than $330 billion in market value to the S&P 500. What's more, the average price/earnings ratio for FAANG stocks has jumped to 35 times estimated earnings from 21 times last year.

  • How Much Are Taxes on an IRA Withdrawal?

    How Much Are Taxes on an IRA Withdrawal?

    How much you will pay in taxes on an individual retirement account (IRA) withdrawal depends on the type of IRA, your age, and the purpose of the withdrawal. In other cases, you owe income tax on the money you withdraw and sometimes an additional penalty if you withdraw funds before age 59½. There are a number of IRA options and a variety of places to open these accounts, but the Roth IRA and the traditional IRA are by far the most widely held types.

  • Kraft Heinz Stock Is Not Worth Chasing

    Kraft Heinz Stock Is Not Worth Chasing

    Berkshire Hathaway (NYSE:BRK.A,NYSE:BRK.B), led by famed investor Warren Buffett, remains a major shareholder, and in fact helped lead the merger between Kraft and Heinz. It doesn't take that much of a close look to see what's wrong with Kraft Heinz and with Kraft Heinz stock.

  • Analysts: 2 Big 8% Dividend Stocks to Buy (And 1 to Sell)

    Analysts: 2 Big 8% Dividend Stocks to Buy (And 1 to Sell)

    The company has been making reliable dividend payments since 2014, and has raised the payment modestly in the past two years. At $1.44 annualized, the yield is a generous 8.54%. Barclays analyst Christine Cho sees PAGP growing going forward, resting as it does on a sound foundation.

  • Justice Department announces $3 billion settlement with Wells Fargo
    ABC News Videos

    Justice Department announces $3 billion settlement with Wells Fargo

    Over 15 years, Wells Fargo employees made 2 million fake accounts, and the government said $500 million of the settlement will go to investors.

  • Dividend ETFs To Buy And Watch For 2020
    Investor's Business Daily

    Dividend ETFs To Buy And Watch For 2020

    Dividends have long been a favorite of investment gurus from Warren Buffett to late Vanguard Group founder John Bogle, and it's easy to see why. Individual stocks can provide income, but dividend ETFs are an easy way to gain exposure to a basket of dividend-paying stocks. With a stock dividend, a company distributes additional shares instead of cash.

  • Michael Bloomberg says it’s not so ‘simple’ to produce his tax returns — here’s what most high-income tax returns have in common

    Michael Bloomberg says it’s not so ‘simple’ to produce his tax returns — here’s what most high-income tax returns have in common

    Michael Bloomberg, the billionaire former New York City mayor running for the Democratic presidential nod, says he will release his tax returns — but that's no easy feat. “I can't go to TurboTax,” Bloomberg, the founder and CEO of the global media and financial data company Bloomberg L.P., told Democratic debate moderators on Wednesday night. Bloomberg, who is worth $65.2 billion according to Forbes, said he makes money from all over the globe, so his tax payments are complex.

  • Warren Buffett’s annual letter: Here’s what investors are looking for

    Warren Buffett’s annual letter: Here’s what investors are looking for

    Berkshire Hathaway Inc. (BRK)(BRK) will release Warren Buffett's eagerly awaited annual shareholder letter, the company's annual report and its latest earnings around 8 a.m. Eastern on its website, where investors can also peruse the chairman and chief executive's past missives. Mark Hulbert: Warren Buffett had a tough year—how might he explain it? Investors of all stripes have dived into the lengthy letters over the years to pick up Buffett's insights on a range of topics beyond Berkshire's performance.

  • Avoid the Social Security Tax Trap

    Avoid the Social Security Tax Trap

    In the good old days, Social Security was tax-free. Below, we'll explore why and how Social Security benefits become taxable and provide some tips to help you dig your way out of the trap. There are two separate income thresholds for filers that will determine whether they have to pay tax on their Social Security benefits.

  • 3 Marijuana Stocks Poised to Lead a Sector Rebound

    3 Marijuana Stocks Poised to Lead a Sector Rebound

    As the Canadian cannabis market continues to fail to meet sales projections, the licensed producers (LPs) with the best balance sheets are poised to lead a market rebound. With both Aurora Cannabis and Tilray implementing restructurings, the industry could see a void in certain markets providing opportunities for companies with the ability to fund growth initiatives. Based on the Aurora restructuring, the company is exiting several international markets along with shifting a focus to a value brand.

  • Mason Hawkins' Southeastern Buys 4 Stocks in 4th Quarter

    Mason Hawkins' Southeastern Buys 4 Stocks in 4th Quarter

    Southeastern Asset Management recently disclosed its portfolio updates for the third quarter of 2019. Founded by Mason Hawkins (Trades, Portfolio) in 1975, the Memphis, Tennessee-based firm manages the Longleaf Partners Funds. GuruFocus has detected 4 Warning Sign with WMB.

  • Biotech Stock Crumbles Despite Esperion Nabbing A Key Drug Approval
    Investor's Business Daily

    Biotech Stock Crumbles Despite Esperion Nabbing A Key Drug Approval

    Shares of Esperion Therapeutics crumbled Friday after the biotech company's cholesterol medicine gained Food and Drug Administration approval — with some caveats. The cholesterol medicine formerly known as bempedoic acid will sell as Nexletol, a once-daily oral treatment for high LDL cholesterol. The drug is specifically approved for patients with a genetic form of high cholesterol or a type of cardiovascular disease.

  • The Top 5 IBM Shareholders

    The Top 5 IBM Shareholders

    Founded in 1911 as the Computing-Tabulating-Recording Company (C-T-R), the company now known as International Business Machines Corp. (IBM) can be traced back even farther, to the 1880s, when Dr. Alexander Dey invented the first dial recorder. More recently, IBM has become a global information technology company focused on software, cloud computing, and consulting services. Not surprisingly, all of the top individual shareholders of IBM are members of its senior management team.

  • Bernie Sanders possibly running against President Trump is starting to scare investors: strategist
    Yahoo Finance

    Bernie Sanders possibly running against President Trump is starting to scare investors: strategist

    There is a growing undercurrent in markets that say a battle this fall between self-described socialist Senator Bernie Sanders and President Donald Trump could be bad news for stock prices. As evidenced by the market's strong run-up in 2020, investors have yet to exit stocks on fears of a big government Bernie winning the Oval Office. But his continued strong showing in debates and national polls may lead to some profit-taking sooner rather than later.

  • New Victoria’s Secret Owner, Sycamore Partners, Has a Storied History in Footwear: 4 Things to Know
    Footwear News

    New Victoria’s Secret Owner, Sycamore Partners, Has a Storied History in Footwear: 4 Things to Know

    It's the end of an era for Victoria's Secret, the lingerie giant that for 28 years has been under the wing of mall retail group L Brands. On Thursday, private equity firm Sycamore Partners announced that it would acquire a 55% stake in the company for $525 million. The deal, which takes Victoria's Secret off the public market at a valuation of $1.1 billion, is notable not only for its relatively modest size (L Brands' market capitalization has fallen from $29 billion at its peak in 2015 to around $6.5 billion today) but also for the brand's new owner.

  • Chevron’s Future Growth Prospects Dwindle With Reserves Decline

    Chevron’s Future Growth Prospects Dwindle With Reserves Decline

    New finds, acquisitions and expansions of existing oil and natural gas holdings were equivalent to just 44% of the company's 2019 production, according to a regulatory filing on Friday. The measure, known as the reserves-replacement ratio, is key for investors because it helps them gauge whether an oil driller is doing enough to sustain future production that underpins everything from dividends to buybacks to acquisitions. For Chevron, whose stock outperformed all other supermajor oil producers last year, the reserves data signals the company may be struggling to locate untouched caches of oil and gas as investors turn increasingly skeptical of the industry's sustainability.

  • Here’s three reasons why the 30-year Treasury yield plunged to a record low

    Here’s three reasons why the 30-year Treasury yield plunged to a record low

    The U.S. 30-year Treasury bond yield dropped to an all-time low on Friday, sparking worries among investors who wonder what it's saying about the economy's future prospects. Analysts say the slump in the long bond's yield this week reflects a confluence of factors including easy Federal Reserve monetary policy, concerns about the COVID-19 epidemic's impact on economic growth, and an absence of inflationary pressures. Historically, a slump in long-term yields can indicate fears that growth will start to disappoint and inflation will fall which may make the real return on bonds more attractive.

  • Business

    The Dow Closes Down 228 Points. Don’t Just Blame Coronavirus, Blame the U.S. Service Economy.

    The Dow Jones Industrial Average dropped again on Friday—and yields on long-term Treasury debt fell to record lows—but investors can't simply blame the coronavirus outbreak in China this time. A key U.S. economic indicator registered its weakest reading since 2013. The Markit Services PMI, or purchasing manager index, came in under 50—the level that divides growth from contraction.