Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy
News Article | May 4, 2017
Bercom International, the manufacturer of HANDy Paint Products, is searching for do-it-yourself painters (and film-making enthusiasts) to enter its 6th Annual HANDy Paint Products’ Online Video Contest. To submit, contestants must produce a short video (1 ½ - 4 minutes long) featuring at least one of the 15 HANDy Paint Products in some way, shape or form (as long as it’s legal). Any and all HANDy Paint Products are acceptable for use in a video, including the popular HANDy Paint Pail, Paint Tray, Paint Cup, Roller Cup, Pro Pail, Ladder Pail and Grid. For inspiration, check out these top entries from previous years, which received rave reviews. “It’s incredibly exciting to see the video contest enter its sixth year,” says Mark Bergman, CEO of Bercom International. “Every year we are blown away by the hard work that applicants put into their submissions and we look forward to seeing this year’s entries.” The first-place winner of the HANDy Paint Products Film Festival receives a $2,000 cash prize (along with a lifetime of bragging rights), with the second and third place winners taking $750 and $250 respectively. All contest entrants receive $50 worth of free HANDy Paint Products and there is an additional $5,000 prize awarded to whomever from the Top 10 finalists can make their video “go viral” on the HANDy Paint Products YouTube channel by the end of 2017 (e.g. with 100,000 views or more). The deadline for video submissions is September 3, 2017. Entry details can be downloaded online and questions about the contest can be directed to Bercom International at 877-464-1170. Each of the Top 10 finalists will have the opportunity to see their work on the big screen on September 9, 2017, during the HANDy Paint Products Film Festival at Bercom headquarters in Chanhassen, Minn. The top three winners will be chosen by local celebrity judges. The Bercom-sponsored event is open to the public and all donations made at the Film Festival will go directly to the Parent Project Muscular Dystrophy. Registration details are available at handypaintproducts.com. About HANDy Paint Products HANDy Paint Products is a painting supplies product line developed by Bercom International. Mark Bergman founded Bercom in 2002 after realizing the need for making painting easier. With his old Folgers® coffee can-turned-paint bucket as inspiration, the HANDy Paint Pail was born. Bercom is located in Chanhassen, Minn. Its line of 15 HANDy Paint Products can be found at most home centers, paint stores, and hardware stores across the country as well as in Canada, Australia and Europe. Learn more about HANDy Paint Products by visiting HandyPaintProducts.com. See demonstrations on YouTube and follow the brand on Facebook, Twitter, Pinterest and Instagram.
News Article | July 13, 2017
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 boys. Gillette Children's Specialty Healthcare Neuromuscular Clinic is led by Dr. Randall Richardson. Children who have disabilities and complex medical needs—and their families—have been at the center of Gillette's mission for more than 120 years. And like PPMD, Gillette believes in the importance of standards of care for a variety of challenging childhood conditions. What makes the Gillette clinic a stand out is their pediatric program, transition program, and an adult program, all led by highly qualified coordinators. Gillette believes that with innovative medical and surgical intervention, proven therapies and assistive technology, and the support of compassionate health care professionals, children who have these conditions can thrive and live happy, healthy, productive lives. "This serves not just as a wonderful recognition of the work that Gillette Children's Specialty Healthcare neuromuscular team has done, but also offers us the opportunity for continuing performance enhancement," Richardson said. "We are great, but this relationship allows us the opportunity to be even better." Kathi Kinnett, MSN, CNP, PPMD's Senior Vice President of Clinical Care and co-director of Transforming Duchenne Care Initiative (TDCI), is pleased that Gillette is receiving certification. Kinnett says, "Gillette has amassed an impressive team capable of providing comprehensive Duchenne care across the lifespan. We are excited to add this new center, with their expertise, compassion and enthusiasm, to our growing network of Certified Duchenne Care Centers." Kinnett says that PPMD receives numerous requests from centers wanting to be considered for certification. "When PPMD launched our certification program in 2014, we had no idea that the Duchenne community would respond so positively. Families have come to rely on our certification as an indication that these centers are the best of the best. We will continue to certify more centers across the country throughout 2017, recognizing teams of physicians for their leadership in Duchenne care, which in turn will enable families to make the best choice for the care of their child." In 2014, PPMD launched the Certified Duchenne Care Center Program as part of its robust Transforming Duchenne Care Initiative (TDCI). PPMD will continue to award qualified centers the title of Certified Duchenne Care Center in an effort to ensure centers maintain the highest standards in clinical and sub-specialty services, rapidly apply new evidence-based knowledge, minimize heterogeneity in clinical research outcomes, and comply with standards in clinical care that were established by the CDC Care Considerations. As part of its ongoing mission to end Duchenne, PPMD continues to insist that all people with Duchenne receive comprehensive care. To learn more about PPMD's Certified Duchenne Care Center Program, visit PPMD's website. Click here to learn more about the history of PPMD's Certified Duchenne Care Center Program and to access PPMD's first published article on the program. Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne. We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community. Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube. Founded in 1897, Gillette Children's Specialty Healthcare Neuromuscular Clinic (Gillette) was the first hospital in the nation for kids who have disabilities. Last year, nearly 25,000 families with children facing such complex medical conditions as cerebral palsy, epilepsy and traumatic brain injury, trusted Gillette to help their children thrive. Gillette tailors its care model specifically to the needs of its patient and family community so they can achieve their highest potential.
News Article | June 30, 2017
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 boys. Established more than seven years ago, PPMD's Duchenne Drug Development Roundtable (DDDR) is a group of committed innovators representing industry and relevant stakeholders that has the goal of accelerating the development of meaningful treatments for Duchenne through open discussion and working collaboratively in the precompetitive space to minimize duplication. This year's annual DDDR has never been more important or timely. There are now more than three dozen publicly and privately traded companies in Duchenne; more than a dozen therapies in late stage clinical trials; and a robust pipeline representing an array of therapeutic approaches not far behind. The Duchenne community also has crossed into the post-approval space with the accelerated approval of EXONDYS 51 in September of 2016 and the recent approval of EMFLAZA this past spring. But, according to PPMD Founding President and CEO, Pat Furlong, "The last two years – two years of intense product reviews for our Duchenne therapies – have provided us with new challenges and opportunities. These reviews – coupled with PPMD's policy engagement around the Duchenne community-led Draft Guidance, key provisions within 21st Century Cures, Benefit-Risk studies, and PDUFA VI discussions – have resulted in an opportunity to take what has been learned from all trial experiences and work together to become even more focused and efficient. PPMD and the leadership of our DDDR felt that this is a strategic inflection point for Duchenne; a moment to bring industry partners together to assess how these 'regulatory learnings' and new policies might best be applied back into our pipeline and into clinical trial design." To achieve this, the DDDR Steering Committee mapped out a series of three one-day meetings that were held earlier this year. Each included leaders and innovators from outside the Duchenne space to present on opportunities to enhance our existing trial designs, tools, and infrastructure. Furlong believes that the DDDR meeting at PPMD's Connect Conference is a unique moment in the Duchenne calendar each year, providing companies and stakeholders a chance to truly connect. Says Furlong, "We appreciate each company that has come to the table to lean in even further and we look forward to the opportunity to work together as a community to explore the innovations and opportunities that are raised through ongoing discussion and exploration. It is our hope that the DDDR will continue to assist PPMD and the Duchenne community, working together to drive forward collaborations and projects that will benefit all partners working in this space and yield effective therapies that are accessible to all who need them." PPMD's Annual Connect Conference is the largest, most comprehensive annual, international conference focused entirely on Duchenne. Each year nearly 500 families from around the world gather at PPMD's Connect Conference to learn the latest progress in the fight to end Duchenne. It is also an opportunity for families to gather for support, strength, and camaraderie. PPMD believes strongly in the power of connecting. The 2017 Connect Conference is sponsored, in part, by Elite Sponsors PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics. Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne. We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community. Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/parent-project-muscular-dystrophy-to-host-duchenne-drug-development-roundtable-during-23rd-annual-connect-conference-300481556.html
News Article | June 28, 2017
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 boys. In January, PPMD organized a Critical Path Innovation Meeting with the FDA focused on "Inflammation and Immune Response in Duchenne." The goal of the meeting was to begin to explore the role that inflammation plays in Duchenne and how it might be measured. Biomarkers of inflammation were also discussed in terms of differences resulting from age and stage of disease, variability of biopsied muscle site, use of less invasive techniques and tissue for biomarker analyses, the need for robust validation of novel biomarkers, and challenges of differentiating steroid myopathy from underlying disease. Also discussed was the concept that there may not be a single treatment or dose that is consistently needed throughout the stages of disease or even within an individual with Duchenne. It may be important to tailor therapeutic options. What is needed is a clearer understanding of the inflammation related pathways at a molecular level so that specific inflammatory processes can be targeted at the right time. This 90-minute meeting helped identify the need for PPMD to convene a more involved workshop to further discuss what is known and unknown, about the role of inflammation and the immune response in Duchenne. According to Abby Bronson, PPMD's Senior Vice President of Research Strategy, "The agenda for this upcoming meeting will include Duchenne experts, as well as leaders from other disease areas bringing their expertise and experience to the Duchenne community. We look forward to better understanding the role that inflammation and immunity play in Duchenne, and we are anxious to continue discussing potential therapeutic options that will help every single person living with this disease." PPMD's Annual Connect Conference is the largest, most comprehensive annual, international conference focused entirely on Duchenne. Each year nearly 500 families from around the world gather at PPMD's Connect Conference to learn the latest progress in the fight to end Duchenne. It is also an opportunity for families to gather for support, strength, and camaraderie. Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne. We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community. Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/parent-project-muscular-dystrophy-to-convene-international-experts-for-inflammation-and-immunity-in-duchenne-muscular-dystrophy-workshop-300481258.html
News Article | September 25, 2017
"PPMD is extraordinarily proud of our Certified Duchenne Care Center Program, started in 2014, that recognizes outstanding centers capable of providing comprehensive care and services to people and families living with Duchenne in agreement with the Centers for Disease Control and Prevention's care guidelines," said Pat Furlong, PPMD's Founding President and CEO. "This continuation grant will allow us to continue our research liaison program, a program that has successfully increased enrollment in DuchenneConnect (PPMD's patient reported outcomes registry) and integrated clinical trial awareness into patient visits. In addition, this grant has given rise to young adult/transition seminars, family support groups, the integration of Child Life into patient visits, enhanced electronic medical records to include real time physical therapy data, and many other institutional projects. It is one thing for PPMD to certify these outstanding clinics, but we also have the responsibility to ensure that they can maintain their high standard of care. This grant will help support the compassion and care that these dedicated centers expertly provide to our patients and families. " Highly regarded in the Duchenne community, the CDCC Program has created a network of centers across the United States capable of providing increased access to comprehensive care and services to patients and families living with Duchenne. PPMD's CDCC Program recently announced its 17th Certified Duchenne Care Center, with plans to certify additional centers throughout 2018. The program includes more than 400 medical providers, serving over 3,000 patients, and active involvement in 44 clinical trials that account for over 850 patients within the CDCC network enrolled. Plans are also underway to start providing tools to implement the certification of centers internationally by partnering with global advocacy partners and using the strict guidelines of the CDCC Program. In 2012, PPMD convened a group of 17 institutions, parents, and representatives from advocacy and legislation, in its first Transforming Duchenne Care Initiative (TDCI) meeting, which set out to define Duchenne comprehensive care and services. Attendees discussed components of comprehensive care, discrepancies in care and services, and ways to reduce those discrepancies. In 2014, PPMD launched the Certified Duchenne Care Center Program, the natural evolution of its robust TDCI meeting, through which PPMD awards qualified centers the title of Certified Duchenne Care Center. This designation is an effort to ensure centers: As part of its ongoing mission to end Duchenne, PPMD continues to insist that all people with Duchenne receive comprehensive care. "The response to the CDCC program from the community has been tremendous," said Kathi Kinnett, MSN, CNP, PPMD's Senior Vice President of Clinical Care and co-director of Transforming Duchenne Care Initiative (TDCI). "All stakeholders in the Duchenne community, including families, industry, and providers, have come to value the standardized care and services that the CDCCs offer. In addition to improving patient and family access to comprehensive care and services, many of these CDCCs are major clinical trial sites. By providing CDCC's with the resources to secure a research liaison, we are providing families with an informed guide to help them navigate the diagnostic odyssey of Duchenne." To learn more about PPMD's 17 Certified Duchenne Care Center Program and a map of currently certified centers, visit PPMD's website. Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne. We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community. Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.
News Article | February 16, 2017
HACKENSACK, N.J., Feb. 16, 2017 /PRNewswire-USNewswire/ -- Ann & Robert H. Lurie Children's Hospital of Chicago (Lurie Children's) became the 14th clinic named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to...
News Article | February 15, 2017
In response to outrage from patients and lawmakers, Marathon Pharmaceuticals has delayed the launch of an $89,000 drug for Duchenne muscular dystrophy. The company had announced the annual list price for Emflaza, which is a steroid, after the Food and Drug Administration approved the drug Thursday. Emflaza is approved as an orphan drug, which means it is intended to treat a rare disease. Duchenne is an inherited disorder that causes muscles to become weak. There is no cure for the condition, which mainly affects boys, but some drugs, including Emflaza, are used to lessen symptoms. For years, many American patients have imported deflazacort, the generic version of Emflaza, for about $1,200 a year. But because the medicine wasn't approved in the U.S., the cost of the medicine wasn't typically covered by insurers. That contrast in price between became a flash point Monday as Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., sent a letter to Marathon on Monday morning demanding answers about the $89,000 price for a drug that isn't new. It has been used routinely by Duchenne patients in the U.S. since at least 2005. "We believe Marathon is abusing our nation's 'orphan drug' program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades," Sanders and Cummings wrote. Marathon said FDA approval would help more patients get the drug. "Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who prior to FDA approval have not had access to the therapy," said a statement attributed to Marathon CEO Jeffrey Aronin that was read Monday to a meeting of parents, patients and advocates in Washington, D.C. "We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process." But patients and their families cried foul. "What you're doing is robbing my insurance company," said Dana Edwards, a mother from New Jersey whose 12-year-old has taken deflazacort, the generic version of the drug, since he was 5. Pat Furlong, the president and founder of Parent Project Muscular Dystrophy, which sponsored the Monday conference, read the statement to an outraged crowd in a conference room at the Mayflower Hotel. "Since last week's approval, they've heard from all of us," he said. Furlong told the audience that complaints from patients helped to prompt Marathon's action. The company, Furlong read, will continue to offer patients an expanded access program, which allows about 800 patients to receive the drug from the company. More can join that program free, and patients can continue importing the drug from Canada or "wherever they are getting it," the statement said. In January, Kaiser Health News and NPR published an investigation that found the orphan drug law intended to help desperate patients is being manipulated by drugmakers for financial gain. Republican Rep. Robert Aderholt, the chairman of the appropriations subcommittee that funds the FDA, called Marathon's announcement a "tipping point." And he said the astronomical increase in deflazacort's price "makes me question whether the current construct of how the FDA approves orphan drugs does more harm than good if companies have found a way to game the system." At Monday's meeting, Marathon executives presented a slideshow on financial assistance for patients, as well as details on the drug's expected availability. Emflaza was set to hit the market by mid-March, said Eric Messner, vice president of sales and marketing for Marathon. Messner said the drug would cost $50,000 to $54,000 after rebates and discounts to insurers. "I know there has been a lot of discussion about that. How did we arrive at the price?" Messner said. An audience member interrupted Messner asking if the company had talked to patients and families about the price. He offered assurance that they did and said there would be an ongoing dialogue with patient groups. "We expect that patients will experience a low or zero out-of-pocket experience," he said. Late Monday, Marathon released an official statement and open letter to the Duchenne community, promising that "price should not be a barrier" for patients. "Put simply we expect patients will pay a standard copay of typically $20 or less per prescription. Edwards, the New Jersey mother, said she hopes President Trump will fulfill his vow to bring down high drug prices. Kaiser Health News' coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.
News Article | February 28, 2017
HACKENSACK, N.J., Feb. 28, 2017 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced the 500th patient application to their highly successful Decode Duchenne program. Decode Duchenne is a nationwide program to assist individuals with Duchenne muscular dystrophy in...
News Article | February 24, 2017
BURLINGTON, Mass. & LIESTAL, Switzerland--(BUSINESS WIRE)--Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announced that it will be establishing U.S. operations in Burlington, Mass. Santhera’s U.S. operations are being led by Todd Bazemore, Chief Operating Officer of Santhera Pharmaceuticals (USA), Inc. The U.S. operations will initially be staffed to focus on regulatory and clinical operations support, medical affairs, patient advocacy liaison and commercial strategy. “A strong presence in the U.S. is an important next step in our mission to advance novel treatments for Duchenne muscular dystrophy and other rare neuromuscular and mitochondrial diseases,” said Todd Bazemore, COO of Santhera U.S. “We chose the metropolitan Boston area for our U.S. operations because it is one of the main centers for the biotech and pharma business globally with a unique confluence of academia, life science companies, and clinical expertise.” Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and leads to progressive muscle weakness starting at an early age. DMD is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of up to 1 in 3,500 live male births worldwide.1 DMD is characterized by a loss of the protein dystrophin, ultimately resulting in muscle weakness and wasting and early morbidity and mortality due to respiratory failure. In DMD, the progressive respiratory muscle weakness results in respiratory symptoms such as inability to cough and clear mucus, frequent airway infections, sleep-disordered breathing and the need for assisted ventilation. Addressing the progressive loss of respiratory function is a hallmark in DMD and an urgent unmet medical need for these patients. In a Phase III clinical trial (DELOS) conducted in DMD patients not taking glucocorticoids, Santhera’s lead therapeutic candidate, idebenone, demonstrated a clinically significant slowing in the loss of respiratory function, regardless of the underlying genetic mutation. Santhera is now enrolling patients in a new Phase III trial (SIDEROS) in 60 centers across the United States and Europe to confirm the efficacy of idebenone in 266 patients who are currently taking a stable dose of glucocorticoids. The first patient was enrolled at the University of Kansas Medical Center (KUMC), Department of Neurology in September with Jeffrey Statland, MD. In the U.S., the SIDEROS trial will be conducted in 24 study centers with the goal of having this trial fully enrolled by end of 2017. If successful, this study will provide data that supports the use of idebenone in all DMD patients experiencing respiratory decline irrespective of their glucocorticoid use or genetic mutation. “Idebenone is generating a significant interest among researchers, physicians and the patient community. In slowing the loss of respiratory function in all patients irrespective of their mutational status, this drug candidate has the potential to expand the current treatment paradigm for DMD,” said Thomas Meier, PhD, CEO of Santhera. “Our presence here in the U.S. will enable us to expand our engagement with physicians, their patients, and families as the SIDEROS trial moves forward while we continue our efforts to find a regulatory path for early approval.” “There is high interest from investigators and the patient community in therapies that hold the promise of maintaining respiratory function in patients with DMD,” added Oscar Henry Mayer, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children’s Hospital of Philadelphia and Lead SIDEROS Investigator for the U.S. “A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values treatment options for respiratory complications.” SIDEROS is a phase III, double-blind, randomized, placebo-controlled trial with idebenone in approximately 266 DMD patients receiving concomitant glucocorticoids. Patients with declining respiratory function on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status will be eligible. Study participants will receive either idebenone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 months). The primary endpoint of the trial is the change from baseline to week 78 in forced vital capacity % predicted (FVC%p). Secondary endpoints include changes from baseline in % predicted peak expiratory flow (PEF%p), time to first 10% decline in FVC and change from baseline in inspiratory flow reserve. Patients completing the trial will be offered the opportunity to enroll in an open label extension study in which all patients receive idebenone. The study will be conducted at about 60 centers in the United States and Europe. Patients wishing to enroll in the study should contact their neuromuscular clinic physician. Further information about the study is available under www.clinicaltrials.gov. Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and results in rapidly progressive muscle weakness. DMD is characterized by a loss of the protein dystrophin, leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy production in muscle cells. This results in progressive muscle weakness and wasting and early morbidity and mortality due to respiratory failure. Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS) and supplementing cellular energy levels. DELOS was a phase III, double-blind, placebo-controlled trial which randomized 64 patients, not taking concomitant glucocorticoids, to receive either idebenone (900 mg/day) or matching placebo. The trial met its primary endpoint and demonstrated that idebenone can slow the loss of respiratory function and reduces bronchopulmonary complications. The statistically significant and clinically relevant outcomes of the phase III DELOS study were published: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26: 473–480 and Buyse et al., Pediatric Pulmonology 2016: http://dx.doi.org/10.1002/ppul.23547. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and the Swiss regulatory authorities Swissmedic are currently assessing a Marketing Authorization Application (MAA) for idebenone under the name RAXONE® in DMD patients with respiratory function decline who are not taking concomitant glucocorticoids. The indication would include patients who previously were treated with glucocorticoids or in whom glucocorticoid treatment is not desired, not tolerated or is contraindicated. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera's lead product RAXONE® (idebenone) is authorized in the European Union, Norway, Iceland and Liechtenstein for the treatment of Leber's hereditary optic neuropathy (LHON). For Duchenne muscular dystrophy (DMD), the second indication for RAXONE®, Santhera has filed a Marketing Authorization Application (MAA) in the European Union and Switzerland. In collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing RAXONE® in a third indication, primary progressive multiple sclerosis (PPMS), and omigapil for congenital muscular dystrophy (CMD), all areas of high unmet medical need. For further information, please visit the Company's website www.santhera.com. This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. These statements could be affected by, among other things, risks and factors referred to in the Risk Factors section on Santhera’s web site (http://www.santhera.com/investors-and-media/investor-toolbox/risk-factors). Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. 1 National Institutes of Health, U.S. National Library of Medicine, Genetics Home Reference, Duchenne and Becker muscular dystrophy, https://ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy#statistics
News Article | November 3, 2016
ALLSCHWIL, SCHWEIZ und ROCKVILLE (MD), USA - 3. November 2016 - Actelion Ltd (SIX: ATLN) und ReveraGen BioPharma, Inc., ein privates Unternehmen, das proprietäre therapeutische Produkte im Bereich der neuromuskulären und entzündlichen Krankheiten erforscht und entwickelt, gaben heute den Abschluss einer Vereinbarung bekannt. Im Rahmen dieser Vereinbarung erhält Actelion eine Exklusivoption zur Einlizensierung des Leitkandidaten von ReveraGen, Vamorolone, zur Behandlung von Muskeldystrophie Duchenne. Die Option kann in zwei verschiedenen Entwicklungsphasen des Wirkstoffs ausgeübt werden. ReveraGen entwickelt Vamorolone in klinischen Studienprogrammen parallel in den USA und in Europa. Die klinischen Studien der Phase I wurden Ende 2015 abgeschlossen - sie wurden im Rahmen von Venture-Philanthropy-Verträgen von Organisationen wie der Muscular Dystrophy Association (USA), Joining Jack (UK), Duchenne Research Fund (UK) und Duchenne Children's Trust (UK) finanziert. Actelion hat mit ReveraGen BioPharma, Inc., einer Gesellschaft nach dem Recht des Staates Delaware, USA, eine Lizenz-, kooperative Entwicklungs- und Kommerzialisierungsvereinbarung zur Erforschung und gemeinsamen Entwicklung von Vamorolone, einem nicht-hormonalen Steroidmodulator primär zur Behandlung von Muskeldystrophie Duchenne (DMD) abgeschlossen. Bislang hat Actelion insgesamt USD 10 Millionen an ReveraGen gezahlt, welche als F&E-Ausgaben ausgewiesen wurden. Gemäss der Vertragsbedingungen wird Actelion in den nächsten drei Jahren auch F&E-Aktivitäten unterstützen, wobei der Höchstbetrag dafür bei USD 1 Million pro Jahr liegt. Weiterhin erwarb Actelion eine Option auf die globalen Exklusivrechte zur Einlizensierung von Vamorolone zu einem beliebigen Zeitpunkt, jedoch nicht später als nach Erhalt der Ergebnisse der IIb-Studie. Wenn die Option ausgeübt wird, hat ReveraGen einen Anspruch auf Milestone-Zahlungen in Höhe von USD 165 Millionen für die Indikation DMD, und auf weitere USD 190 Millionen für drei weitere Indikationen, abhängig vom Erreichen bestimmter Meilensteine in den Bereichen F&E, Registrierung und Kommerzialisierung. Desweiteren wird Actelion gestaffelte Lizenzgebühren im ein- bis zweistelligen Bereich auf den Nettoumsatz von Vamorolone zahlen. Vamorolone hat sowohl in den USA als auch in Europa den Status als Orphan Drug erhalten. ReveraGen entwickelt Vamorolone in parallelen klinischen Studienprogrammen in den USA und in Europa. Die klinischen Studien der Phase I wurden Ende 2015 abgeschlossen. Gegenwärtig läuft ein Programm der Phase IIa, das die Sicherheit und Wirksamkeit von Vamorolone bei vier- bis siebenjährigen steroidnaiven Jungen mit Duchenne untersucht, d.h. von Patienten, die noch nicht mit Prednison oder Deflazacort behandelt wurden. Das klinische DMD-Programm wird in Zusammenarbeit zwischen CINRG Group und Newcastle University (Kate Bushby und Michela Guglieri) entwickelt und durchgeführt. Ein Programm der Phase IIb ist in Planung. ReveraGen BioPharma, Inc., ist ein privates pharmazeutisches Unternehmen, das 2008 gegründet wurde und seinen Sitz in Rockville (MD), USA hat. Der einzige Vermögenswert von ReveraGen, Vamorolone, ist ein proprietärer Wirkstoff für die Behandlung von Muskeldystrophie Duchenne. Die Entwicklung von Vamorolone erfolgt auf der Basis von Venture Philanthropy, in Zusammenarbeit mit staatlichen Institutionen wie den US National Institutes of Health, der Europäischen Gemeinschaft und internationalen Organisationen, einschliesslich der Foundation to Eradicate Duchenne, Parent Project Muscular Dystrophy, Save our Sons und anderen. Insgesamt wurden bis heute von Regierungsstellen und Interessengruppen USD 20 Millionen zur Verfügung gestellt. Actelion: Andrew C. Weiss Senior Vice President, Head of Investor Relations & Corporate Communications Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil +41 61 565 62 62 http://www.actelion.com