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Positive Interim Results from Clinical Trial Offers Hope to Patients with Duchenne Muscular Dystrophy (DMD)

18 Jul Positive Interim Results from Clinical Trial Offers Hope to Patients with Duchenne Muscular Dystrophy (DMD)

Earlier this year, we had covered some path-breaking companies engaged in clinical trials, to test the efficacy and safety of novel therapeutics, which could help in the treatment and management of Duchenne Muscular Dystrophy (DMD).

Capricor Therapeutics, (NASDAQ: CAPR) a clinical-stage biotechnology company at the forefront of innovation in the biological therapeutics space, with special focus on DMD and other rare disorders, was one of them.

In line with our expectations the company has announced positive results from an interim analysis on 6-month data from its ongoing HOPE-2 trial, with statistically significant results for various clinical measures.

Capricor Therapeutics, Inc, (NASDAQ: CAPRChart
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Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial states

“I am incredibly pleased with the outcome of the interim analysis as it has demonstrated the biologic activity of CAP-1002 that has resulted in changes of clinically relevant outcomes including the upper limb, the hand and diaphragmatic function,” He further adds that “For these older boys who have no further therapeutic options, these data support the hope that CAP-1002 may one day become an important therapeutic option and possibly slow the advancement of the disease.

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CAP-1002, its lead product candidate for the treatment of DMD, is an “off-the-shelf” cardiac cell therapy that consists of allogeneic cardiosphere-derived cells (CDC’s) that have progenitor cells that exhibit immunomodulatory activity and have the potential to stimulate cellular regeneration. It intends to focus on developing Exosome technology as the platform for the future to treat a variety of inflammatory disorders.

CAP-1002 has been granted an orphan drug designation and a Regenerative Medicine Advanced Therapy Designation (RMAT) by the FDA for the treatment of DMD. In addition, it has also received a rare Paediatric Disease Designation, which will make it eligible to receive a Priority Review Voucher, in case of receiving market approval for CAP-1002.

The results from HOPE-2, a randomized, double-blind, placebo-controlled, Phase II clinical trial, demonstrate significant improvement in aspects such as skeletal muscle function, pulmonary function, cardiac structure and function and safety.

“We are extremely pleased and it is truly extraordinary that even in such as small sample size, we achieved statistically significant improvements in several clinically relevant parameters. In these older patients, functional improvement in the upper limb is highly meaningful for their quality of life. To our knowledge, this is the first randomized double-blind, placebo-controlled study in DMD that has shown statistically significant functional improvement in steroid treated boys”

Linda Marbán, president and CEO of Capricor opines.

The Muscular Dystrophy Association describes Duchenne Muscular Dystrophy (DMD) as a fatal, X-linked genetic neuromuscular disorder that leads to progressive, irreversible loss of muscle function, including the heart and lungs. It has an X-linked recessive inheritance pattern and is acquired by a “Carrier” mother. Globally, 1 in 3500 male births is affected by this rare neuromuscular disease. There are over 250,000 people in the United States alone who are afflicted by this disorder.

The growing awareness about this disease and increasing research efforts to find a cure, along with improved insurance coverage and favourable government regulations will drive the growth in the market. According to a report by Grand View Research, Inc the market of DMD therapeutics will grow at a CAGR of 41.3% and reach USD 4.11 billion by 2023.

Researchers have identified the lack of dystrophin, a protein that helps regulate the functioning of membrane (sarcolemma) of muscle cells as the cause of this disorder. Exon skipping is gaining credence as a potential treatment approach to correct for specific genetic mutations and restore production of dystrophin protein, with an increasing number of companies coming up with first-in-class therapeutics based on this approach.

The disease has no cure and the treatment is aimed at alleviating symptoms and arresting the progress of the disease by use of glucocorticoid steroid treatment. Steroids can help reduce the inflammation and assist in the repair of the weakened muscle cell membranes, however they come with multiple side-effects such as weakening of bones , hypertension, weight gain and muscle atrophy.

However the emergence of new mechanisms of action such as NF-κB inhibition, Gene therapy, and Exosome technology can redefine the therapeutic options for those suffering from DMD.

Capricor aims to develop first-in class cellular and Exosome therapeutics, for improving the quality of life of people suffering from rare genetic disorders and orphan diseases, such as Duchenne Muscular Dystrophy. Its research and development efforts are focussed on Exosomes, an emerging field in science, which are primarily nanosized particles with biologically-active contents that can regulate the functioning of other cells.

Besides DMD, it is also evaluating the efficacy of CAP-1002 for treatment of cardiomyopathy through its DYNAMIC clinical trials. Its pipeline also consists of CAP-2003, a CDC –Exosome, intended for the treatment of Inflammatory and Fibrotic disorders.

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Capricor’s proprietary cardiosphere-derived cell (CDC) technology is based on the work of Eduardo Marbán, M.D., Ph.D., company Co-Founder and Scientific Advisory Board Chairman, and Director of the Cedars-Sinai Heart Institute. He has pioneered the isolation of stem cells from heart tissue, which is the fundamental principle behind CDC technology. It has licensed its CDC Exosome technology from Cedars-Sinai Medical Center, and enjoys worldwide rights to the same. Furthermore the company has entered into collaboration with the U.S. Army Institute of Surgical Research (USAISR) to study the use of CAP-2003 to address trauma-related injuries and conditions.

Risk Assessment

    • Capricor has been battling a paucity of funds, forcing it to cut down its workforce and temporarily halt patient enrolment in DMD, after a failed 142-patient Phase II stem cell study in patients with a heart anomaly.


    • The company previously faced a setback in its ALLSTAR trial that intended to use Stem Cells to treat patients with heart conditions. The study was backed by a $12.5 million upfront payment by Jannsen, with a promise of further milestone-related payments. However all its efforts came to a naught, as Janssen walked away from the deal, owing to failure to achieve the desired results. This made the company revaluate its strategy and realign its focus on the DMD program.


    • The company has all its hopes hinged on the success of CAP-1002, which could prove to be a double-edged sword, though a successful completion of the trial will translate into a great news for the company , any failure to achieve the desired clinical endpoints may have an adverse impact on its operations. It has already faced a setback in its stem cell program and any such occurrence for the second time will have a profoundly negative impact on the company.


    • There is a burgeoning demand for Exosome Technology, for numerous applications such as treatment of cancer, inflammatory and infectious diseases, neurogenerative disorders and cardiovascular diseases. If the company can translate its clinical efforts into positive results, then it is on the right path to great success in the future.


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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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