NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, in an effort to advance gene therapy.

“Our investment into 4DMT will be the fourth investment by CureDuchenne Ventures, the investment arm of CureDuchenne, into gene therapy and gene editing. So far, we have had preclinical success in this area for Duchenne with our investments in duplication 2 exon skipping at Nationwide Children’s Hospital, Bamboo Therapeutics/Pfizer and Exonics Therapeutics, and we are very positive about the prospect of 4DMT’s work,” said Debra Miller, founder and CEO of CureDuchenne. “CureDuchenne’s investments are moving quickly through the drug development process and we look forward to safety and efficacy data for these potential therapies.”

The Duchenne community is aware of possible limitations of the current delivery system for gene therapy and gene editing due to pre-existing neutralizing antibodies, and manufacturing challenges due to the large amount of vector needed to treat the entire muscular system in the body.

4DMT focuses on the discovery and development of targeted, customized and proprietary next-generation AAV gene therapy products for use in patients with severe genetic diseases with high unmet medical need. Therapeutic Vector Evolution empowers 4DMT to create customized gene therapy products to deliver genes specifically to any tissue or organ in the body, by the optimal clinical route of administration for any given disease, at lower doses and with resistance to pre-existing antibodies.

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Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and to provide the seed funding necessary to accelerate his work in this company, we could not be more thrilled. This is a true testament to the power of venture philanthropy and I want to thank each and every donor and supporter who has believed in our work, and we are grateful to Dr. Olson for his dedication to Duchenne patients. Although we still have a long way to go before we confirm this therapy will be safe and effective in humans, this dramatic dystrophin restoration offers hope to all families affected by this disease.

The story began in 2015 when news of CRISPR technology first began surfacing. The CureDuchenne science and investment teams started to research and talk to different scientists. We met with a couple different groups before landing on Dr. Eric Olson, who had a long history of working in muscular disorders and had a true entrepreneurial spirit, which is crucial when working in this space.

After several meetings with UT Southwestern and Dr. Olson, we came to an agreement that the fastest way to accelerate CRISPR technology to human clinical trials was to form a company to fast track development. That’s when the three groups worked together in a truly collaborative partnership to form Exonics.

Within a matter of weeks, CureDuchenne committed $5 million in seed money to Exonics. We also put together an LLC to secure funding from other Duchenne organizations to help us to raise the $5 million, which we did not have, but every organization we talked to turned us down because they did not believe enough in the research project. Thankfully, after our first payment of $2 million, which was every penny we had, The Column Group came in with Series A funding and we no longer needed to fund the remaining $3 million.

With a mission to find, foster and fund investments in early stage projects, which many VCs and other investment groups avoid because of the high risk involved, this potential success story proves the incredible value in being a knowledgeable risk taker when it comes to finding cures for rare diseases.

On average, a drug takes 10 years to get to FDA approval after three years of clinic trials. With an average lifespan into the mid-20s, those with Duchenne need solutions faster. CureDuchenne Ventures’ goal is to identify the best science and to accelerate drug development timelines. We will continue to operate with the highest standards of ethics and integrity by re-investing the earnings back into research and programs that help support the whole journey of a Duchenne patient until we have a cure.

Read the full article HERE

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Dr. Victor, who is the associate director for clinical research at the Cedars-Sinai Heart Institute and a lead investigator for the HOPE trial, reported that those treated with CAP-1002 had improvement in cardiac muscle function and reduction in cardiac scarring that were statistically-significant in comparison to the control group, according to a pre-specified analysis. In addition, in a post-hoc analysis, 89% of the CAP-1002 treated patients who were more severely impaired demonstrated sustained or improved skeletal muscle function at 12 months, as compared to none of the participants in the control group.

“Because Duchenne muscular dystrophy is a devastating, muscle-wasting disease that causes physical debilitation and eventually heart failure, the improvements in heart and skeletal muscle in those treated with a single dose of CAP-1002 are very promising and show that a subsequent trial is warranted,” said Dr. Victor. “These early results provide hope for the Duchenne community, which is in urgent need of a major therapeutic breakthrough.”

Dr. Victor, who has worked in the Duchenne muscular dystrophy field for 20 years, presented the 12-month results of the randomized, open-label, early stage Phase I/II HOPE clinical trial. The trial was designed to evaluate safety and explore efficacy. It enrolled 25 boys and young men in advanced stages of Duchenne muscular dystrophy and was conducted at three U.S. centers. All participants had significant cardiac scarring and approximately two-thirds were wheelchair-dependent at the time they began the trial.

During the 12-month course of the trial, all patients received standard-of-care for Duchenne muscular dystrophy, including oral steroids, and 13 also received one dose of intracoronary CAP-1002 upon randomization. CAP-1002 consists of allogeneic cardiosphere-derived cells which have been reported to improve muscle function and increase new muscle cell generation in preclinical models of Duchenne muscular dystrophy.

To assess skeletal muscle function, investigators used the Performance of the Upper Limb test (PUL). The PUL tests manual tasks that relate to activities of daily living that are very important for quality of life. PUL has been validated for the assessment of upper limb motor function in individuals with Duchenne muscular dystrophy. The functional tasks of the PUL are subdivided into three subscales reflecting disease progression from proximal to distal (from the middle of the body outward): (1) High-level: shoulder dimension; (2) Mid-level: elbow dimension; and (3) Distal-level: wrist and finger dimension.

As shoulder function had already been lost in most of the HOPE participants, investigators used the combined Mid-Distal PUL subscales to assess changes in skeletal muscle function and found significant improvement in those treated with CAP-1002 (defined post-hoc). Among the lower-functioning patients (baseline Mid-Distal PUL < 55 out of 58), investigators reported sustained or improved motor function in 8/9 (89%) of the CAP-1002 treated patients as compared to 0/4 (0%) of the usual care participants, at 12 months (p=0.007).

To assess cardiac structure and function, investigators used magnetic resonance imaging (MRI). They found significant improvements in cardiac muscle function among those treated with CAP-1002, according to measures of systolic thickening of the left ventricular wall. Systolic thickening is thought to be a principal mechanism of cardiac output generation in people with Duchenne muscular dystrophy.

In the inferior wall, they recorded a mean (SD) 31.2% (47.0%) increase in thickening six months after treatment and a mean 25.8% (46.7%) increase in thickening 12 months after treatment. In comparison, the usual care group showed a mean 8.8% (27.7%) decrease at six months and a mean 1.6% (37.9%) increase at 12 months in the systolic thickening of the inferior wall. The difference between the groups at six months achieved statistical significance (p=0.04; p=0.09 at 12 months).

Investigators also found that scarring of the heart muscle among those treated with CAP-1002 decreased relative to the control group. Progressive cardiac scarring eventually impairs the heart’s pumping ability and is currently the leading cause of death in Duchenne muscular dystrophy. At the 12-month follow-up, those treated with CAP-1002 had a mean 7.1% (10.3%) reduction in scar size, in contrast to a mean 4.8% (22.3%) increase in scar size in the usual care group, a difference that achieved statistical significance (p=0.03).

CAP-1002 was generally safe and well-tolerated in the HOPE trial. There was no significant difference in the incidence of treatment-emergent adverse events in either group. There were no early study discontinuations due to adverse events.

“These 12-month results extend our prior findings with CAP-1002 and further support its potential to serve those with Duchenne muscular dystrophy,” Linda Marbán, Ph.D., Capricor president and CEO, said. “Pending regulatory clearance, we plan to initiate the randomized, double-blind, placebo-controlled HOPE-2 clinical trial in the first quarter of 2018. HOPE-2 will evaluate multiple doses of CAP-1002 given intravenously, and the primary efficacy analysis will be driven by changes in skeletal muscle function as assessed by the PUL.”

Drs. Victor and Marbán are scheduled to participate in a conference call and webcast at 4:30 p.m. ET today to review the results presented at the AHA meeting. To participate, please dial (866) 939-3921 (domestic) or (678) 302-3550 (international) and reference the access code 45894703. Slides to accompany the call may be viewed via the webcast link at http://wsw.com/webcast/cc/capr5. Access to the live webcast as well as the link to the replay of the call can be found at http://capricor.com/news/events/. The webcast will be archived for approximately 30 days.

The HOPE trial was funded in part by the California Institute for Regenerative Medicine.

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CAMBRIDGE, Mass. – November 8, 2017 – Exonics Therapeutics, Inc., a biotechnology company focused on developing SingleCut CRISPR technology to repair mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, today announced it has secured a $40 million Series A financing to advance its lead gene editing program in Duchenne from The Column Group (TCG), a science-driven venture capital firm. Exonics also announced that TCG’s David Goeddel, Ph.D., managing partner, and JJ Kang, Ph.D., principal, will be joining the company’s board of directors.

Exonics is generating data to support the development of a safe and efficacious one-time gene editing treatment leveraging SingleCut CRISPR to potentially provide lifelong benefit for patients with Duchenne, a devastating muscle disease in children for which there is no cure.

“Exonics’ transformative gene repair technology presents a promising opportunity to create novel therapies that address the significant unmet need in the treatment of serious genetic neuromuscular diseases,” said Dr. Goeddel. “The company has generated compelling early data, and we are pleased to support Exonics as it advances its preclinical development program in Duchenne closer to the clinic.”

Published preclinical data in mice suggest the therapy has the potential to identify and repair exon mutations that prevent the production of dystrophin, the key protein missing in children with Duchenne that helps stabilize and protect muscle fibers. The technology underlying Exonics’ gene editing approach is licensed from the University of Texas Southwestern Medical Center and is based on research from the laboratory of Eric Olson, Ph.D., Exonics’ founder and chief science advisor.

“This funding from a leading healthcare venture capital firm further validates the potential for Exonics’ novel gene editing technology to help correct many of the mutations that cause Duchenne and other neuromuscular diseases,” said John Ripple, chief executive officer of Exonics. “We look forward to working with TCG to translate our science into a meaningful treatment for the many Duchenne patients and their families. We’re grateful to CureDuchenne Ventures for its support in founding the company and providing the seed financing, which has enabled Exonics to establish a strong scientific and corporate foundation to build upon.”

“As a leading research organization focused on Duchenne muscular dystrophy, CureDuchenne, through CureDuchenne Ventures, is proud to support the work of Dr. Eric Olson, and we are hopeful that Exonics’ CRISPR/Cas9 gene editing technology will ultimately offer a significant and impactful treatment for those affected by Duchenne,” said Debra Miller, chief executive officer and founder of CureDuchenne.

About Exonics Therapeutics

Exonics Therapeutics has developed SingleCut CRISPR, a gene repair technology that has the potential to effectively halt the progression of certain genetic neuromuscular diseases. In multiple Duchenne muscular dystrophy preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with Duchenne. Exonics is initially focused on correcting mutations that cause Duchenne in order to develop a therapy to treat many children with the devastating disease, for which there is no cure. Exonics’ technology is licensed from the University of Texas Southwestern Medical Center and is based on the research of Eric Olson, Ph.D., Exonics’ founder and chief science advisor. Exonics is located in Cambridge, Mass. For more information, please visit www.exonicstx.com.

About The Column Group
The Column Group (TCG) is a leading venture capital firm based in San Francisco that seeks to partner with exceptional scientific founders, entrepreneurs, executives, and investment organizations with a shared vision for building the next generation of drug discovery and development companies. TCG invests in disease-focused drug discovery companies with the potential to become leaders in their respective fields. These companies are strongly supported by the unique and complementary skill sets of TCG’s team, which includes prominent authorities in the scientific, operational, and financial arenas. For more information, please visit www.thecolumngroup.com.

About CureDuchenne

CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne is also the innovator in bringing physical therapy and standard of care to local communities around the country through CureDuchenne Cares. For more information, please visit www.CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.

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The late breaking abstract and results presented at the late breaking poster session describe the first six months of follow-up data from the randomized 12-month Phase I/II HOPE clinical trial of CAP-1002. CAP-1002 is a cell-based therapeutic candidate and consists of allogeneic cardiosphere-derived cells, whose mechanism of action is immunomodulatory and anti-fibrotic, and which have been shown to generate new muscle cells in preclinical models.

“These findings are especially significant because the patients in the HOPE trial were preteens or young men who were in advanced stages of Duchenne muscle disease,” said Ron Victor, M.D., professor of medicine and Burns and Allen Chair in Cardiology Research at Cedars-Sinai Heart Institute and a principal investigator for the HOPE Trial. “Most other studies in DMD have focused on pediatric patients in earlier stages of the disease. To see such positive results in a clinical trial with just one dose of CAP-1002 sets the stage for the next step of evaluating multiple doses of this innovative cellular therapy in a larger trial.”

The HOPE Trial was a randomized, open label trial of 25 males with DMD, of ages 12 to 25 years (mean 17.8). For 17 of them, the disease had progressed to the point of wheelchair dependence for mobility. Cardiomyopathy, or heart disease, secondary to DMD was an eligibility criterion and was evidenced by scar in four or more left ventricular segments. All participants had been receiving chronic corticosteroid therapy at entry. Thirteen received a single dose of CAP-1002, while the others received the standard of care, and all participants were to be followed for 12 months. CAP-1002 was administered by infusion into each of the three main coronary arteries for a total dose of 75 million cells.

In the trial, cardiac muscle was assessed by magnetic resonance imaging (MRI) studies performed at baseline, six months, and 12 months. All MRI interpretations were conducted in a manner blinded to treatment assignment and clinical outcomes.

Regional left ventricular (LV) function significantly differed between treatment groups following a single dose of CAP-1002, as determined by assessments of systolic thickening of LV wall segments. At six months, a statistically-significant increase in mean (standard deviation, or SD) change from baseline in inferior wall segments was observed with CAP-1002 (+31.2% (46.9)) compared to the usual care group (-8.8% (27.7)) (p=0.02). Six-month mean changes in anterior (+16.3 (46.5)) and lateral (+24.5 (51.2)) wall segments numerically favored CAP-1002 as compared to usual care ((-14.1 (24.9)) (p=0.11) and (-4.5 (35.0)) (p=0.24), respectively).

Differences observed in the change from baseline in cardiac scar size are consistent with a treatment effect on the heart. At six months, the mean (SD) percent change from baseline in observed scar size was -5.1 (8.5) in the CAP-1002 group (p=0.04) and -0.2 (11.5) in the usual care group (p=0.71) (p=0.09 for treatment group difference).

Skeletal muscle was assessed by the Performance of the Upper Limb test (PUL), a validated instrument for the assessment of upper limb motor function in individuals with DMD and consists of manual tasks that relate to activities of daily living. Scoring on the PUL was evaluated at baseline and at six weeks, and then at three, six, and 12 months.

Following a single dose of CAP-1002, the mean (SD) percent changes from baseline to six weeks and three months, respectively, in combined middle-plus-distal PUL dimension were +8.8 (15.0) and +8.9 (15.4) in the CAP-1002 group and -1.7 (3.7) and +0.8 (3.7) in the usual care group. By a post hoc responder analysis, mid-distal PUL score increased at six weeks by ? 10% (or maximum possible) in 42% of CAP-1002 participants compared to none of the usual-care participants (p=0.045). At three months, the group difference in response was 33% CAP-1002 vs. 10% usual care (p=0.32). Given the ages of the trial participants, shoulder function (upper PUL scoring) had been essentially lost prior to entry.

Treatment with CAP-1002 was generally safe and well-tolerated over the initial six-month follow-up period of the HOPE Trial. There was no significant difference in the incidence of treatment-emergent adverse events of either group.

“These exciting findings propel us into our next phase of development of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer. “Subject to regulatory approvals, we expect to initiate the randomized, double-blind, placebo-controlled HOPE-2 clinical trial of intravenous (IV), repeat-dose CAP-1002 in the first quarter of 2018. The primary efficacy endpoint will be based on the PUL, and the HOPE-2 Trial may potentially serve as a registration study. We also look forward to presenting 12-month follow-up results from the HOPE Trial at a major medical conference later this quarter.”

The poster is available at the Events & Presentations section of Capricor’s website.

DMD is a devastating genetic disorder that causes muscle degeneration and leads to death generally before the age of 30, most commonly from heart failure. DMD occurs in one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 15,000 to 20,000 boys and young men in the U.S. Treatment options are limited and there is no cure.

The HOPE trial was funded in part by the California Institute for Regenerative Medicine.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and alters 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 140 human subjects across several clinical trials.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases. Capricor’s lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of DMD. Capricor is also exploring the potential of CAP-2003, a cell-free, extracellular vesicle-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.

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Additionally, supportive changes in measures of muscle health were seen, consistent with positive edasalonexent treatment effects. Muscle enzymes significantly decreased compared to baseline at 12 weeks and later time points (p<0.05) and lower leg muscle MRI T2 rate of change was significantly improved in comparison to progression during the control period (p≤0.05). Edasalonexent continued to be well tolerated with no safety signals observed in the trial.

Based on the consistency of the MoveDMD results and supportive regulatory input from FDA, Catabasis plans to initiate a single global Phase 3 trial with edasalonexent in patients with DMD regardless of mutation type in the first half of 2018 with top-line results expected in 2020. The data were presented today in a late breaking session at the World Muscle Society Conference and will be discussed, along with the Phase 3 clinical trial plan, on a conference call today, October 4, 2017.

“We are extremely excited to see edasalonexent change the trajectory of disease in the MoveDMD trial with substantially slowed disease progression,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. “Boys treated with edasalonexent stabilized; they experienced meaningful improvements in muscle function compared to the rates of change observed during the control period. Importantly, other supportive positive measures of muscle health were observed. We look forward to advancing edasalonexent as a disease-modifying therapy in a single Phase 3 pivotal trial as soon as possible with the goal of providing a meaningful impact on disease progression for all boys affected by Duchenne.”

“Our goal in treating boys with Duchenne is to slow the progression of the disease. It is tremendously encouraging to see boys taking edasalonexent stabilize in their functional abilities and MRI T2 measures along with its continued safety profile,” said Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System and a Principal Investigator for the study. “I look forward to continuing to investigate edasalonexent as a potential therapy for the many boys affected by this devastating disease.”

In the MoveDMD trial, a substantial slowing of the disease progression of DMD was seen in boys treated with edasalonexent compared to the rates of change during the control period. Through 36 weeks of treatment, the 100 mg/kg/day treatment group showed clinically meaningful numerical improvements in rates of decline compared to rates of change during the control period across all three timed function tests (10-meter walk/run, 4-stair climb and time to stand), as well as the North Star Ambulatory Assessment (NSAA), an integrated global assessment of muscle function. Control period changes were measured prior to boys receiving edasalonexent, either prior to Phase 2 or in the placebo group, for time periods averaging 39 weeks. In the 100 mg/kg/day treatment group, 16 boys commenced edasalonexent either at the beginning of Phase 2 or at the beginning of the open-label extension. At the time of the open-label extension data analysis, all 14 boys continuing to participate had received 100 mg/kg/day for 24 weeks and 11 had completed 36 weeks of 100 mg/kg/day edasalonexent treatment. Results are detailed for the 100 mg/kg/day treatment group as all boys are now taking this dose in the open-label extension.

Additional supportive measures of muscle health were also consistent with a positive edasalonexent treatment effect. Four muscle enzymes (creatine kinase, alanine aminotransferase, aspartate aminotransferase and lactate dehydrogenase) were significantly decreased compared to baseline following edasalonexent treatment at 12 weeks and later time points (p<0.05), consistent with the ability to slow muscle degeneration and improve muscle integrity. Rate of change in lower leg MRI T2 significantly improved at 12 weeks and at last observation on treatment compared to control period (p≤0.05), consistent with a reduction of inflammation in the muscle.

Edasalonexent continued to be well tolerated with no safety signals observed to date in the MoveDMD trial. The majority of adverse events (AEs) have been mild in nature with no serious AEs. There have been no dose reductions and no drug-related discontinuations. The most common AEs were gastrointestinal, primarily mild and transient diarrhea. Height, weight and BMI growth patterns were similar to standard growth curves for unaffected boys in the age range of MoveDMD subjects. Boys with DMD in this age range typically have resting tachycardia, a heart rate that exceeds the normal resting rate, and heart rate of the boys treated with edasalonexent decreased toward age-normative values during treatment. We believe these clinical heart rate observations are intriguing and warrant follow-up.

Catabasis plans to commence a single global Phase 3 trial in DMD in the first half of 2018 to evaluate the efficacy and safety of edasalonexent for registration purposes. The planned design of the randomized, double-blind, placebo-controlled trial is informed by discussions with FDA. Catabasis plans for the Phase 3 trial to have many elements in common with the Phase 2 trial including the patient population and endpoints. The trial is anticipated to enroll approximately 125 patients ages 4 to 7 who have not been on steroids for at least 6 months. The primary efficacy endpoint will be change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints are planned to include age-appropriate timed function tests. Catabasis expects to report top-line results from this trial in 2020.

“We are excited to see positive effects on muscle function with edasalonexent, as we know from research that effects on muscle function are the most important aspect of a therapy for Duchenne for the affected patients and their families,” said Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD). “We look forward to learning more about edasalonexent as Catabasis begins its Phase 3 trial. With the disease-modifying effects and safety and tolerability profile observed to date for edasalonexent, it has the potential to be a foundational therapy for all people affected by Duchenne.”

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CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced the publication of data validating Genethon’s micro-dystrophin gene therapy approach in an animal model for Duchenne muscular dystrophy (DMD). The results were featured in the July 25, 2017 online issue of Nature Communications. Sarepta announced in June 2017 that it entered into an exclusive gene therapy research collaboration with Genethon to jointly develop treatments for DMD.

“While early, these data highlight the potential for Genethon’s micro-dystrophin gene therapy program and once again underscore the significance of dystrophin production in the treatment of DMD,” said Douglas Ingram, Sarepta’s president and chief executive officer. “As the leader in the research and development of new treatments for DMD, we are taking a multi-front approach to advancing therapies for those afflicted with this debilitating disease.”

The study was conducted in 12 dogs naturally affected by DMD and treated with intravenous micro-dystrophin, a shortened version of the dystrophin protein combined with an AAV-type viral vector. At two-year follow-up, muscle function was significantly restored and clinical symptoms had stabilized. Additionally, researchers noted that dystrophin expression had returned to a high level in the high-dose group. No immunosuppressive treatment was administered beforehand, and no side-effects were observed.

Under the terms of the previously announced collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethon’s micro-dystrophin program, which includes exclusive U.S. commercial rights. For more information, and to see a video illustrating the results of the aforementioned study, please click here.

Le Guiner, C. et al.  Long-term micro-dystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nature Communications. 2017: Accepted Article, ahead of print. DOI: 10.1038/ncomms161.

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The aim of the EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.¹ The MHRA decision allows patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product currently under review for DMD for Marketing Authorization by the European Medicines Agency (EMA).

Under the EAMS, and as shown in the public assessment report,² Raxone is indicated for slowing the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids. The decline of respiratory function must be confirmed by repeated measurements prior to initiation of treatment. Raxone can be used in patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.

“We`re proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD,” said Thomas Meier, PhD, CEO of Santhera. “This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options.”

“This is excellent news for patients with respiratory decline in Duchenne muscular dystrophy,” said Janet Bloor, Chair of the Board of Trustees at Action Duchenne. “The need for new treatments in DMD is very great and the EAMS can help to accelerate access for patients. Action Duchenne was pleased to provide advice during the development of the EAMS program and we are delighted to see this first positive opinion in DMD.”

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Genethon Logo

Under the terms of the collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethon’s micro-dystrophin program, which includes exclusive U.S. commercial rights. Financial terms of the collaboration have not been disclosed.

Genethon has made significant investment in the development of gene therapies for neuromuscular diseases and employs one of the largest research and clinical groups in the world working to advance rare disease therapies. The Company’s European-based research laboratory has a long-term commitment to neuromuscular diseases with a central focus on DMD. In addition, Genethon is affiliated with Europe’s largest cGMP vector manufacturing facility, YposKesi, located in Evry (Essonne). YposKesi employs approximately 150 experts in bio-production at its current 54,000 square feet manufacturing facility, and plans significant future expansion to meet the growing demand of gene therapy products.

“Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industry’s most comprehensive franchise in DMD,” stated Edward Kaye, Sarepta’s chief executive officer. “This partnership brings together our collective experience in Duchenne drug development and Genethon’s particular expertise in gene therapy for rare diseases.  We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.”

“Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy. This partnership brings together the highly complementary and synergistic expertises of Sarepta and Genethon, to the benefit of the patients,” said Frederic Revah, chief executive officer of Genethon.

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LOS ANGELES, April 25, 2017 /PRNewswire/ — Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions, today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne muscular dystrophy, or DMD. Duchenne muscular dystrophy is a rare, life-threatening genetic disorder for which treatment options are limited.

Highlights from the Phase I/II HOPE Clinical Trial six-month results:

• In a 25-patient, randomized, single-dose, three-center clinical trial being conducted in a DMD population with advanced cardiac disease, patients treated with CAP-1002 demonstrated statistically-significant (p<0.05) improvement compared to usual care controls in certain measures of cardiac and upper limb function.
• CAP-1002 was generally safe and well-tolerated over the initial six-month follow-up period.
• Clinical results corroborate a large body of preclinical data from studies in DMD models.
• Capricor has submitted a meeting request to the U.S. Food and Drug Administration, or FDA, to discuss potential product registration strategies for CAP-1002 in the DMD indication.
• Capricor intends to submit HOPE-Duchenne data to FDA and to request Breakthrough Therapy or Regenerative Medicine Advanced Technology, or RMAT, designations for CAP-1002. The RMAT designation, intended to expedite the approval of safe and effective cell therapies, was created by the U.S. Congress as part of the recently-enacted 21st Century Cures Act.

John L. Jefferies, M.D., Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at the University of Cincinnati and Director, Advanced Heart Failure and Cardiomyopathy, and Principal Investigator of the HOPE Trial, said, “In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial. These results clearly support the conduct of a confirmatory clinical trial in DMD to further evaluate the potential of CAP-1002. We look forward to an effective medication becoming available for people with this progressive and fatal disease, one that is poorly met by current options.”

Joao A. C. Lima, M.D., Professor of Medicine and Director of the Magnetic Resonance Imaging, or MRI, Core Lab at The Johns Hopkins University School of Medicine, said, “The observed signal in global cardiac scar reduction and the increase in the thickening of the left ventricle during contraction are very encouraging. The population treated in HOPE had very advanced cardiac involvement, and to see such positive results following just a single dose of CAP-1002 is remarkable. Cardiac disease is the most common cause of mortality among those with DMD.”

Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the largest nonprofit organization in the U.S. solely focused on DMD, said, “I’m excited to see these data, especially given the advanced nature of the patients in the HOPE trial. It is also gratifying to see the field of cell therapy making progress after more than two decades in development. It is our hope that CAP-1002 will have broad potential to improve the lives of patients with Duchenne muscular dystrophy.”

The randomized HOPE (Halt cardiomyOPathy progrEssion in Duchenne) Clinical Trial was designed to evaluate the safety and exploratory efficacy of CAP-1002 in patients 12 years and older with DMD who had cardiomyopathy, or heart disease, secondary to DMD as evidenced by scar in four or more left ventricular segments as detected by late gadolinium-enhancement MRI. Twenty-five patients were randomized to receive either a single dose of CAP-1002 (13 patients) or usual care (12 patients). CAP-1002 was infused into each of the three main coronary arteries at a total dose of 75 million cells.

All cardiac assessments were performed by MRI. A validated test of upper limb function, the Performance of the Upper Limb test, or PUL, was used to assess skeletal muscle performance. The PUL was designed specifically for use in the DMD setting, and evaluates a variety of manual tasks, such as lifting cans, tearing paper, and removing a container lid, which simulate activities of daily living. Boys and young men with advanced DMD have difficulty with such common activities as feeding themselves and brushing their teeth.

CAP-1002 has been well-tolerated in the HOPE trial. During and immediately following CAP-1002 infusion, no treated subjects experienced a pre-specified composite safety endpoint, which included the major adverse cardiac events of death, myocardial infarction, or hospitalization for a cardiovascular event. There were no early study discontinuations due to adverse events.

In exploratory efficacy analyses, statistically-significant improvements in systolic thickening of the inferior wall of the heart (p=0.030), and in the function of the middle and distal upper limb according to a PUL responder analysis (p=0.045), were observed in patients treated with CAP-1002 as compared to usual care control patients. In addition, differences observed in several other cardiac and skeletal muscle measures, including cardiac scar (p=0.09), are consistent with a treatment effect. At three months, a statistically-significant difference in quality-of-life (p=0.03), according to the PODCI Adolescent Questionnaire, that favored the CAP-1002 arm was also observed. A more comprehensive summary of the top-line data will be provided in slides to be presented during today’s webcast.

“These initial positive clinical results build upon a large body of preclinical data which illustrate CAP-1002’s potential to broadly improve the condition of those afflicted by DMD, as they show that cardiosphere-derived cells exert salutary effects on cardiac and skeletal muscle,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer.

“We have submitted an FDA meeting request to discuss these results as well as next steps in our development of CAP-1002 for Duchenne muscular dystrophy, which includes our plan to begin a clinical trial of intravenously-administered CAP-1002 in the latter half of this year. We believe the interim HOPE results may enable us to pursue one of the FDA’s Expedited Programs for Serious Conditions, and we will apply for either or both of the Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations for CAP-1002,” added Dr. Marbán.

Capricor expects to report top-line 12-month results from the HOPE-Duchenne Trial in the fourth quarter of 2017.

A pre-publication manuscript reporting on academic studies of cardiosphere-derived cells in animal models of DMD may be accessed at http://biorxiv.org/content/early/2017/04/20/128900.

The HOPE-Duchenne trial is funded in part by the California Institute for Regenerative Medicine.

Conference Call and Webcast Information

Capricor will host a conference call and webcast with slides today, April 25, 2017, at 8:00 a.m. Eastern Time to discuss the top-line six-month HOPE-Duchenne clinical trial results. Capricor’s executive management team will be joined on the call by Dr. Lima. The conference call can be accessed by dialing (866) 901-2585 for participants in the U.S. and Canada and (404) 835-7099 for international callers (reference passcode 44797929). The conference call will be webcast live and can be accessed at Capricor’s website or by clicking this link (www.wsw.com/webcast/cc/capr). The webcast will be archived on the Capricor website for approximately 90 days.

About Duchenne Muscular Dystrophy

DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by an abnormality in the dystrophin complex, a structural element that plays a critical role in muscle fiber integrity, which leads to chronic skeletal and cardiac muscle damage. Patients with DMD typically die in their twenties, most commonly due to heart disease. The incidence of DMD is estimated to be one in every 3,600 live male births, and DMD is believed to afflict approximately 15,000 to 20,000 boys and young men in the U.S.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac progenitor cell. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. CAP-1002 is currently being evaluated in the randomized, double-blind, placebo-controlled Phase II ALLSTAR Clinical Trial in adults who have suffered a large heart attack and in the Phase I/II HOPE Clinical Trial in boys and young men with DMD.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions. Capricor’s lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of Duchenne muscular dystrophy, myocardial infarction (heart attack), and heart failure. Capricor is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.

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; 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, 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, 2016, as filed with the Securities and Exchange Commission on March 16, 2017, and in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, 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. Capricor’s exosomes technology, including CAP-2003, has not yet been approved for clinical investigation.

For more information, please contact:

Corporate
Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
+1-310-358-3200
abergmann@capricor.com

Investors
Argot Partners
Kimberly Minarovich
+1-212-600-1902
kimberly@argotpartners.com

Media
Argot Partners
Eliza Schleifstein
+1-917-763-8106
eliza@argotpartners.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/capricor-therapeutics-announces-positive-six-month-results-from-the-randomized-phase-iii-hope-clinical-trial-in-duchenne-muscular-dystrophy-300444967.html

SOURCE Capricor Therapeutics, Inc.

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