Embed Size (px)
Citation preview
NOVEMBER 2016
RESEARCH
CHMP Recommends Renewal Of Translarna's™Marketing Authorization For Nonsense MutationDuchenne Muscular Dystrophy Based On ContinuedPositive Benefit-Risk Assessment
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced November 11 that theCommittee for Medicinal Products for Human Use (CHMP) of the EuropeanMedicines Agency (EMA) has recommended the renewal of the conditionalmarketing authorization of Translarna™ (ataluren) for the treatment of nonsensemutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients fiveyears and older...
Read more.
Capricor Therapeutics Announces Plans To ExpandClinical Development Program In Duchenne MuscularDystrophy To Evaluate Peripheral And RespiratoryMuscle Function
Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnologycompany developing first-in-class biological therapies for cardiac and otherserious medical conditions, announced on November 10 that it intends to expandits CAP-1002 clinical development program in Duchenne muscular dystrophy(DMD) to encompass the skeletal muscle aspects of the disease, in addition tothe cardiac complications...
Read more.
Summit Enrolls Patients In The US Into PhaseOutDMD, A Phase 2 Clinical Trial Of Ezutromid
Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery
and development company advancing therapies for Duchenne musculardystrophy ('DMD') and Clostridium difficile infection, on November 16 announcesthat it has enrolled its first patients at trial sites in the US into PhaseOut DMD, aPhase 2 proof of concept clinical trial of ezutromid in patients with DMD.Ezutromid dosing is expected to follow within a screening period of up to 28days...
Read more.
CUREDUCHENNE CARES
CHLA Cares Workshop
On Saturday, November 5, CureDuchenne Cares held its final Family Workshopof 2016. The successful workshop was hosted by Children’s Hospital LosAngeles, which was a welcome collaboration between CureDuchenne and theCHLA Neuromuscular Clinic led by Clinic Director/Neurologist Dr. Leigh Ramos-Platt...
Read more.
Video On Multiple Disciplinary Care Model
Dr. Leigh Ramos-Platt from Children's Hospital Los Angeles recently presentedduring the CureDuchenne Cares Family Summit on the multiple disciplinary caremodel. Watch the video to learn more...
Read more.
Oral Health Consideration Video
Those with Duchenne muscular dystrophy may be at increased risk for oralhealth complications. This video with Dr. Parish Sedghizadeh of USC School ofDentistry helps to explain some of the conditions, causes and symptoms to beaware of...
Read more.
Spotlight On Sandi Preston, PT, CureDuchenne Cares
Certified Physical Therapist
Understanding a patient’s motivation for treatment is the key for a physicaltherapist to empower that patient for the best outcome. For a patient withDuchenne muscular dystrophy, a terminal disease that deteriorates muscletissue, this empowerment can be the difference between more cherishedambulatory time or losing their drive for therapy altogether...
Read more.
NEWS
Chef Amar Santana Comes On As The Celebrity ChefFor The Third Annual Napa In Newport
Chef Amar Santana of the celebrated Broadway by Amar Santana and Vacarestaurants will be the celebrity chef for the much anticipated third annual Napain Newport Wine Auction. Chef Amar Santana is one of Orange County’s hottestrising stars who is undeniably transforming the OC culinary scene into a fooddestination...
Read more.
Webinar: Update On Deflazacort Access AndRegulatory Status
CureDuchenne was pleased to host a webinar with Marathon Pharmaceuticalson November 1. Marathon provided updated information about deflazacortaccess and regulatory status...
Read more.
CTAP Announces Two Research PublicationsCategorizing And Predicting Disease Progression InDuchenne Muscular Dystrophy
The Collaborative Trajectory Analysis Project (cTAP), a public-private partnershipto accelerate data science solutions to critical problems in drug development forDuchenne Muscular Dystrophy (DMD), today announced the publication of tworesearch studies with important implications for the design of effective clinicaltrials...
Read more.
Give While You Shop
Give a percent of your Amazon purchase to CureDuchenne this holiday seasonby making your purchases on Amazon Smile. Amazon Smile donates 0.5% of allqualifying purchases to the charity chosen by buyer. Millions of product qualify,and will be indicated on the item pages...
Read more.
RECENT SUCCESS
CBS Courage In Sports: Climb To CureDuchenne
Mountain climber Tyler Armstrong was featured in CBS Courage in Sportsshowcasing how he climbs mountains to raise awareness and fund research tohelp find a cure for Duchenne muscular dystrophy...
Read more.
UPCOMING EVENTS
Napa In Newport, March 4, 2017, Laguna, Nigel, CA
Reserve the date for Napa In Newport on March 4, 2017 at the Ritz-Carlton,Laguna Niguel. Napa In Newport brings together 30 of Napa’s most distinguishedwineries to Orange County for an extraordinary wine and culinary experience...
Read more.
Champions To CureDuchenne, April 8, 2017, Austin,TX
Save the date for the Champions to CureDuchenne gala on April 8 at theUniversity of Texas Golf Club. Join us for a magical evening to find a cure forDuchenne muscular dystrophy. The event includes cocktails, dinner, casino, liveand silent auction...
Read more.
Unsubscribe from receiving email, or change your email preferences.
nonprofit software
◄ RETURN TO NEWSLETTER
CHMP Recommends Renewal Of Translarna's™ MarketingAuthorization For Nonsense Mutation Duchenne MuscularDystrophy Based On Continued Positive Benefit-Risk Assessment
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced November 11 that the Committee for Medicinal Products forHuman Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditionalmarketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne musculardystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketingauthorization will include a specific obligation to conduct an additional long-term post-authorization trial.
"We are pleased with this outcome which took into account all available data for Translarna," said Stuart W. Peltz,Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "This decision reflects the benefit that Translarna is having forpatients suffering from nonsense mutation Duchenne muscular dystrophy."
The CHMP opinion forms the basis for a European Commission decision on the renewal of the marketingauthorization. The European Commission generally delivers its decision within three months.
"Translarna has shown clinically meaningful benefits for patients," said Eugenio Mercuri, M.D., Professor of PediatricNeurology at the Catholic University, Rome, Italy. "Duchenne is a devastating disease with a progressive loss offunction. Maintaining function is of the utmost importance to patients."
"The consistency of Translarna's benefit shown across key endpoints is impressive for a dystrophin replacementtherapy," said Craig McDonald, M.D., Professor of Pediatrics and Chair of the Department of Physical Medicine &Rehabilitation at University of California. "I am encouraged for the DMD community by the CHMP'srecommendation."
The CHMP has requested that PTC conduct a new 18-month randomized, placebo-controlled study in patients withnonsense mutation Duchenne muscular dystrophy, as a specific post-authorization obligation, with results expectedto be available in the first quarter of 2021. This study will be followed by an 18-month open-label extension periodwhere all patients will be switched to Translarna. PTC has proposed a trial similar in size to ACT DMD and details of
NOVEMBER 2016
the protocol are expected to be finalized in future interactions with the EMA. Conditional marketing authorizations aresubject to annual reassessment and renewal.
Read more.
◄ RETURN TO NEWSLETTER
Capricor Therapeutics Announces Plans To Expand ClinicalDevelopment Program In Duchenne Muscular Dystrophy ToEvaluate Peripheral And Respiratory Muscle Function
Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-classbiological therapies for cardiac and other serious medical conditions, announced on November 10 that it intends toexpand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass theskeletal muscle aspects of the disease, in addition to the cardiac complications. Based on preclinical data in DMDmodels that show significant improvement in skeletal, including diaphragmatic, muscle function with CAP-1002(allogeneic cardiosphere-derived cells), Capricor is designing a clinical trial to evaluate the potential ability of CAP-1002 to benefit skeletal muscle function in boys and young men with DMD. In the planned study, the medication willbe given by systemic intra-vascular administration. This trial is expected to begin in 2017 subject to regulatoryapproval, and is intended to enroll people with DMD irrespective of their mutation or ambulatory status.
Linda Marbán, Ph.D., president and chief executive officer, said, "In a standard preclinical model of DMD, CAP-1002is able to positively affect peripheral and respiratory muscles and to partially restore muscle cell energetics andfunction. These encouraging results indicate that CAP-1002 has the potential to not only address the heart disease inDuchenne, but to also treat the progressive failure that occurs in the peripheral and respiratory musculature. Towardthis goal, we have developed a formulation of CAP-1002 that can be infused into the systemic circulation. We arehopeful that the muscle function improvements we have observed in preclinical models can be recapitulated in boysand young men with DMD."
Capricor is currently conducting the randomized, controlled Phase I/II HOPE clinical trial of CAP-1002 in DMD-associated cardiomyopathy. In HOPE, 13 patients received a single dose of CAP-1002 via intra-coronary triple-vessel infusion and 12 patients are receiving usual care, with those randomized to the control arm to be eligible toreceive CAP-1002 following the completion of their 12-month follow-up. Subject to regulatory review, Capricor isexploring the possibility of re-dosing patients in the active arm who complete the 12-month trial period. It is hopedthat patients who initially respond to CAP-1002 will become eligible for repeat dosing.
Barry J. Byrne, M.D., Ph.D., Director of the Powell Gene Therapy Center and Professor of Pediatrics and MolecularGenetics & Microbiology at the University of Florida, and Principal Investigator of the HOPE-Duchenne clinical trial,stated, "The prospect of sustainably preserving heart function in DMD via repeated dosing of CAP-1002 is supported
NOVEMBER 2016
by studies in preclinical models. We look forward to learning more about the treatment potential of CAP-1002 throughan amendment to the HOPE trial, as well as through the anticipated trial of CAP-1002 to be given by systemic intra-vascular administration."
Read more.
◄ RETURN TO NEWSLETTER
Summit Enrolls Patients In The US Into PhaseOut DMD, A Phase 2Clinical Trial Of Ezutromid
Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancingtherapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, on November 16 announcesthat it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 proof of concept clinical trialof ezutromid in patients with DMD. Ezutromid dosing is expected to follow within a screening period of up to 28 days.Enrolment and dosing of patients into PhaseOut DMD in the UK is ongoing. Ezutromid is a utrophin modulator andrepresents a potential disease modifying treatment for all patients with DMD.
"Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow orstop disease progression in all patients with DMD, regardless of their underlying dystrophin gene mutation," saidJohn Jefferies, MD, of Cincinnati Children's Hospital Medical Center, and the US coordinating investigator inPhaseOut DMD. "We are excited to participate in PhaseOut DMD and contribute to the clinical development of thisutrophin modulator."
Ralf Rosskamp, MD, Chief Medical Officer of Summit added, "Our PhaseOut DMD clinical trial is an importantcomponent of bringing ezutromid to patients and families who are in urgent need of a disease modifying therapy, andwe are making progress with patient enrolment in this clinical trial, with enrolment ongoing in the UK and now in theUS."
Read more.
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
CHLA Cares Workshop
On Saturday, November 5, CureDuchenne Cares held its final Family Workshop of 2016. The successful workshopwas hosted by Children’s Hospital Los Angeles, which was a welcome collaboration between CureDuchenne and theCHLA Neuromuscular Clinic led by Clinic Director/Neurologist Dr. Leigh Ramos-Platt.
The event drew in families from Los Angeles and surrounding southern California counties. There were more than 50attendees, which included parents, grandparents, family members and friends of those with Duchenne, along withboys and young adults with Duchenne themselves.
Collaboration between the organizations included a joint presentation and demonstration of stretching for those withDuchenne by CHLA physical and occupational therapists (Jed David, Rebecca Jorne, and Marielle Pascual)alongside CureDuchenne’s Director of Physical Therapy, Celeste Graham. Participants were shown common durablemedical equipment options by Kevin Phillips, local Assistive Technology Professional, and had the opportunity to try
NOVEMBER 2016
out the equipment themselves! Clinical trial and research updates were provided by Dr. Michael Kelly, Chief ScientificAdvisor for CureDuchenne, along with representatives from sponsoring pharmaceutical and biotech companies. Dr.Parish Sedghizadeh, Doctor of Dental Surgery, gave an important presentation on oral considerations—a new topicto many of the families.
CHLA specialists in Duchenne provided vital information in the areas of: neurology (Dr. Leigh Ramos-Platt);orthopedics (Dr. Lindsay Andras); pulmonology (Dr. Ajay Kasi); cardiology (Dr. Jennifer Su); social, community andfunding resources (Laurie Miller, clinical social worker). The event offered two tracks—one for those that are newlydiagnosed and the other for those seeking more information for medical considerations in the later stages ofDuchenne.
A mother with a young son with Duchenne shared, “As a patient advocate as well as a parent, your organizationgives me hope. Too many DMD families are lost in the community.” Another mother stated she would recommendthis workshop to others, as we gave “important information given about different topics like orthopedics.”
Do you want CureDuchenne Cares to come to your city? Please contact [email protected] or 949-872-2552. We are now planning for locations for 2017. Be sure to check back at www.CureDuchenneCares.org oftenfor updates on the 2017.
CureDuchenne Cares would not have been possible without the support of our sponsors: BioMarin, Bristol-MyersSquibb, Catabasis, FibroGen, Idera Pharmaceuticals, Invitae, Marathon Pharmaceuticals, Pfizer, PhaseBio, PTCTherapeutics, Sarepta Therapeutics, and SITE SoCal.
◄ RETURN TO NEWSLETTER
Video On Multiple Disciplinary Care Model
CureDuchenne Family Summit - Multiple Disciplinary Care Model with Leigh Ramos Platt MD CHLA
Dr. Leigh Ramos-Platt from Children's Hospital Los Angeles recently presented during the CureDuchenne CaresFamily Summit on the multiple disciplinary care model. Watch the video to learn more.
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
Oral Health Consideration Video
Video Blog 19 Oral Health Considerations for Those With Duchenne Muscular Dystrophy
Those with Duchenne muscular dystrophy may be at increased risk for oral health complications. This video with Dr.Parish Sedghizadeh of USC School of Dentistry helps to explain some of the conditions, causes and symptoms to beaware of.
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
Spotlight On Sandi Preston, PT, CureDuchenne Cares CertifiedPhysical Therapist
Understanding a patient’s motivation for treatment is the key for a physical therapist to empower that patient for thebest outcome. For a patient with Duchenne muscular dystrophy, a terminal disease that deteriorates muscle tissue,this empowerment can be the difference between more cherished ambulatory time or losing their drive for therapyaltogether.
In working with Duchenne patients over her 30-year physical therapy career, Sandi Preston has learned theimportance of listening to her patients and truly hearing what inspires them. This knowledge helps her guide theirtherapy and maintain their enthusiasm as much as possible.
NOVEMBER 2016
Preston knows a bit about motivation during physical treatment. She was born with feet alignment issues thatrequired physical therapy in order to walk better. Later on, with the encouragement of her mother, she observedphysical therapists treating patients — doing for others what they at one time did for her. She saw how much aphysical therapist enhances their patient’s life and decided then that she wanted to spend her life helping othersimprove their lives.
With a rural New York practice, she rarely sees Duchenne patients but wanted to provide them with the best carepossible. There is currently no cure for Duchenne, and physical therapy is one of the primary treatment options.
After an experience with a later stage Duchenne patient, Sandi realized her knowledge base needed to go beyondthe therapy to include helping patients acquire the resources they need. She enrolled in the CureDuchenne CaresPhysical Therapist certification program to learn how to better discern the subtle symptoms of Duchenne that canalert a therapist as to when it’s necessary to act, treat, or order medical equipment quickly.
Read more.
◄ RETURN TO NEWSLETTER
Chef Amar Santana Comes On As The Celebrity Chef For TheThird Annual Napa In Newport
Chef Amar Santana of the celebrated Broadway by Amar Santana and Vaca restaurants will be the celebrity chef forthe much anticipated third annual Napa in Newport Wine Auction. Chef Amar Santana is one of Orange County’shottest rising stars who is undeniably transforming the OC culinary scene into a food destination. His impressivecareer crescendo in 2012 occurred at the age of 30, when he opened his widely successful flagship restaurant,Broadway By Amar Santana in Laguna Beach. His globally influenced menu includes unexpected flavor profiles andartful presentations, reflecting his zeal for food and for life.
Southern California’s premier annual wine event, Napa in Newport, hosted by the deLeuze family of ZD Wines, is setfor March 4 at the Ritz-Carlton, Laguna Niguel. Napa in Newport unites more than 30 renowned vintners and wineenthusiasts to help find a cure for Duchenne muscular dystrophy, a fatal genetic disease that robs children andyoung adults of their muscle strength and claims their lives by their mid-20s. All proceeds from the evening willbenefit CureDuchenne, the national organization committed to finding a cure for Duchenne muscular dystrophy.
Napa in Newport attendees will enjoy a selection of fine, rare and reserve wines from 30 select Napa Valley wineries,curated by Master Sommelier, Michael Jordan, MS, CWE, including ZD Wines, Anomaly Vineyards, Arkenstone,
NOVEMBER 2016
Arrow & Branch, Blackbird Vineyards, BOND Estates, Bure Family Wines, Continuum Estate, Crocker &Starr, Darioush, Davis Estates, Fairchild Napa Valley, Freemark Abbey Winery, Handwritten Wines, HillFamily Estate, Hundred Acre, JCB by Jean-Charles Boisset, Joseph Phelps Vineyards, Melka Wines, MorletFamily Vineyards, Nellcôte, PATEL Napa Valley, Realm Cellars, Relic Wine Cellars, Reynolds Family Winery,Shadybrook Estate, Sojourn Cellars, St. Supéry Estate Vineyards & Winery, Truchard Vineyards, Vineyard 29and Zakin Wines
Napa in Newport kicks off with a wine tasting reception and silent auction at 4:30 p.m., with a three-course dinnerprepared especially for the event by Chef Amar Santana, wine pairings and live auction festivities beginning at 7:00p.m. Tables and sponsorships are available now. For more information and to purchase tickets visit:www.NapaInNewport.org.
◄ RETURN TO NEWSLETTER
Webinar: Update On Deflazacort Access And Regulatory Status
CureDuchenne was pleased to host a webinar with Marathon Pharmaceuticals on November 1. Marathon providedupdated information about deflazacort access and regulatory status. The webinar addressed Marathon’s ExpandedAccess Program for deflazacort and what a potential FDA approval of deflazacort would mean in terms of access tothe drug in the U.S. Please click here to view the recording.
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
CTAP Announces Two Research Publications Categorizing AndPredicting Disease Progression In Duchenne Muscular Dystrophy
The Collaborative Trajectory Analysis Project (cTAP), a public-private partnership to accelerate data sciencesolutions to critical problems in drug development for Duchenne Muscular Dystrophy (DMD), today announced thepublication of two research studies with important implications for the design of effective clinical trials.
All patients with Duchenne eventually lose the ability to walk, but the rate at which ambulatory function declines canvary greatly between patients. This variability can cloud interpretation of clinical trials making it difficult to discernwhether or not a drug is effective. The published studies announced here explain approximately half of the variabilityin disease progression in Duchenne, more than double that explained previously with conventional analyses.
“Without this understanding of the natural clinical progression of the various genetic causes for DMD, it would beextremely difficult to design the clinical trials or choose the appropriate endpoints necessary to develop novel drugsto use for DMD,” said Dr. Edward Kaye, President, CEO and Chief Medical Officer of Sarepta Therapeutics. “cTAP isone of the best examples of international academic collaboration that has advanced the clinical understanding ofDuchenne Muscular Dystrophy.”
cTAP has connected leading clinical experts in Duchenne, drug developers and analytical experts with the sharedgoal of improving clinical trials in Duchenne by learning from patient data. The two published studies used statisticalmethods to quantify and predict disease progression in Duchenne, drawing from a growing database of more than1,000 boys with Duchenne that, in total, includes functional assessments at more than 10,000 clinic visits.
Read more.
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
Give While You Shop
Give a percent of your Amazon purchase to CureDuchenne this holiday season by making your purchases onAmazon Smile. Amazon Smile donates 0.5% of all qualifying purchases to the charity chosen by buyer. Millions ofproduct qualify, and will be indicated on the item pages. There is no additional cost to you! Remember, each time youmake a purchase from Amazon, you must start from smile.amazon.com.
If you have not selected CureDuchenne as your charity you can do that by logging into your Amazon account. If youhave not chosen a charity yet, you can search for CureDuchenne in the search bar at the bottom. SelectCureDuchenne as your organization. CureDuchenne will show as your organization at top of the page next to theAmazon Smile logo. Make your purchase as usual. Amazon Smile gives you the same great benefits of Amazon, butyou’re supporting CureDuchenne.
Click here for Amazon Smile FAQ
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
CBS Courage In Sports: Climb To CureDuchenneYour browser does not support the video tag.
Mountain climber Tyler Armstrong was featured in CBS Courage in Sports showcasing how he climbs mountains toraise awareness and fund research to help find a cure for Duchenne muscular dystrophy. To help Tyler Armstrong inhis quest to help find a cure for Duchenne, please donate: https://www.crowdrise.com/climbtocureduchenne
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
Napa In Newport, March 4, 2017, Laguna, Nigel, CA
Reserve the date for Napa In Newport on March 4, 2017 at the Ritz-Carlton, Laguna Niguel. Napa In Newport bringstogether 30 of Napa’s most distinguished wineries to Orange County for an extraordinary wine and culinaryexperience. For more information, go to www.NapaInNewport.org.
NOVEMBER 2016
◄ RETURN TO NEWSLETTER
Champions To CureDuchenne, April 8, 2017, Austin, TX
Save the date for the Champions to CureDuchenne gala on April 8 at the University of Texas Golf Club. Join us for amagical evening to find a cure for Duchenne muscular dystrophy. The event includes cocktails, dinner, casino, liveand silent auction. For more information, go to www.ChampionsForACure.org
NOVEMBER 2016
