The Jett Foundation Fighting Duchenne Muscular Dystrophy

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Sarepta Community Update on ESSENCE Study

June 23, 2016

Sarepta Therapeutics is updating the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301), as posted on clinicaltrials.gov. [Identifier: NCT02500381]

ESSENCE will serve as a confirmatory study for eteplirsen if it is granted accelerated approval. Post-marketing confirmatory studies are a requirement for drugs that are granted accelerated approval in order to verify the clinical benefit of the drug.

Additionally, we believe that data generated from the ESSENCE study will serve as a foundation to the advancement of our future DMD development programs and to demonstrating the potential safety and efficacy of SRP-4045 and SRP-4053 in boys with mutations amenable to skipping exons 45 and 53, respectively.

We acknowledge the community’s concerns regarding a 96 week placebo-controlled trial and hope to provide clarity regarding recent changes to the clinical study design in this communication.

Why is Sarepta conducting a placebo-controlled trial?

In addition to fulfilling confirmatory study requirements for eteplirsen, we believe that following FDA guidance will provide the most efficient pathway to approval for SRP-4045 and SRP-4053.

Why has the trial been extended from 48 weeks to 96 weeks?

The primary endpoint for the ESSENCE study is 6MWT. Data reported from Sarepta’s “201/202” study revealed that a statistically significant divergence (or separation) in 6MWT between patients on eteplirsen vs. patients in the external control who were not receiving eteplirsen, was not demonstrated at 48 weeks of treatment.

As stated in the FDA draft guidance: “efficacy studies of 18 to 24 months’ duration may substantially increase statistical power, while only modestly increasing overall development time.” We believe that extending this trial to 96 weeks will increase the likelihood of meeting the primary endpoint and generating data that may lead to broader access across the Duchenne populations amenable to these investigational candidates.

What is an interim analysis and what are the implications?

An interim analysis provides an opportunity to look at data at a pre-defined point in time. Sarepta has included a planned interim analysis of the 6MWT in the ESSENCE study when 75% of the study population have reached 48 weeks of participation in the study. A Data Safety Monitoring Board (DSMB) will review these interim data collected and make a recommendation based on their findings. At that point, the study may:

• continue as planned

• be stopped early due to positive efficacy results

If the trial is stopped early due to positive efficacy results, patients in the treatment arm will continue to receive investigational treatment and those in the placebo arm of the study will roll over onto active investigational treatment with either SRP-4045 or SRP-4053. It is important to point out that if the study is stopped early due to positive results, this potentially would be a shorter time period than a standard 48 week study where you would not look at the data until every patient had completed the 48 week time point.

Why has the age range changed from 7-16 years to 7-13 years?

Studies can fail if they are not designed appropriately. We need to use the most current understanding of this complex disease to ensure the data are interpretable.

Based on our review of Duchenne natural history data, some young men over the age of 13 who walk further than 300 meters on a 6MWT seem to behave differently than what is typically observed in the progression of the disease. Because of the less predictable disease course in such patients, they could introduce variability that would make it harder to see a difference between the patients receiving treatment and the patients receiving placebo and thus could jeopardize the overall results of the study. By studying a homogenous (or, very similar) population of patients, we believe there is an increased chance that the primary endpoint of the study will be met.

When will the study start enrolling?

We will work with participating trial sites and patient advocacy organizations to notify the patient community when sites are open for enrollment. You may also check www.clinicaltrials.gov [Identifier: NCT02500381] for the most up-to-date information on the enrollment status of study sites.

What can the community do?

Stay informed by keeping in touch with your doctor and other members of the community. Sarepta will be working with ESSENCE investigators, study staff, and advocacy organizations to expedite enrollment of the study and to provide updates as they become available.

Genetic testing is always required to determine eligibility for investigational exon-skipping drugs targeted to specific mutations. We recommend that patients and families who have general questions about genetic testing or genetic test results contact their physician or a genetic counselor.

Sarepta remains committed to the Duchenne community and thanks Duchenne patients and families, clinical investigators, and advocacy organizations for your support of and participation in the clinical trial process. Kindly submit your questions regarding the ESSENCE study via email to trialinfo@sarepta.com.

Forward Looking-Statements:

This patient community update contains various forward-looking statements regarding regulatory, clinical and other Sarepta matters. Each forward-looking statement contained in this update is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include those identified under the heading “Risk Factors” in Sarepta’s Quarterly Report on Form 10-Q for the Quarter ended March 1, 2016 (https://www.sec.gov/Archives/edgar/data/873303/000156459016017783/srpt- 10q_20160331.htm) and Annual Report on Form 10-K for the year ending December 31, 2015 (https://www.sec.gov/Archives/edgar/data/873303/000156459016013358/srpt-10k_20151231.htm) and other documents filed by Sarepta with the Securities and Exchange Commission.

The Right to Try – Why We Need Accelerated Approval

December 2, 2015

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Darcy Olsen, author of The Right To Try was a guest speaker on a recent NPR Diane Rehm’s show, talking about Jenn McNary’s story. Guest speakers included FDA representative Dr. Peter Laurie who speaks on the role of Expanded Access programs. The show highlights how difficult access (compassionate Use, clinical trials, expanded access) is to potentially life saving drugs in our country today.  It also walks through how some decisions are made providing that access, what role pharmaceutical companies can have, how patients and families also can play a role and what role the FDA is trying fulfill. This is a hot topic in healthcare today. Given it takes about 15 years to get a drug to market, this debate seems to beg the question, would faster approvals, via paths like accelerated approval, solve some of these issues?

 

Christine McSherry’s ADCOM Speech to FDA/Panel on Drisapersen Side Effects

November 27, 2015

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My name is Christine McSherry – for disclosure Jett Foundation has received educational program funding from the sponsor. I am the founder of the Jett Foundation, a leading Duchenne non profit started 16 years ago when my now 20 year old son Jett – was diagnosed with Duchenne.
I am here today to tell you about my family’s experience with drisapersen and why it is not an option for Jett.
He, like others — but not all — experienced side effects, which you have heard about today from both the company and the agency.
When Jett was diagnosed in 2001, there were no options. Duchenne was terminal and there were no drugs in the pipeline.
When I heard of Prosensa’s safety and efficacy trial, we agreed to participate. The year was 2010 and the site at Nationwide Children’s Hospital in Columbus, Ohio was 17 hours by car from our home in Boston.
We drove those 17 hours three times between Thanksgiving and Christmas leaving 4 other young children at home.
During a 2 week stay Jett received three doses over three days by subcutaneous injection in his belly. By the third injection, we knew he was getting drug as the sites were inflamed and incredibly painful.
Even if this was an effective treatment, there was no way Jett could tolerate it and at 15 he said so.
It was a long drive home.
And while we awaited word of a promised extension study, it never came. We knew that even if it did, the risks would not be manageable for Jett.
By Christmas, Jett could no longer walk on his own.
For Jett, what did an injection site reaction actually mean?
-it meant it was extremely painful when the drug was injected.
-it meant the sites immediately turned red and swelled
-they stayed that way for months
-then, they turned purple
-it meant interference with his bathing, dressing, transferring, sleeping, and worse of all, his embarrassment during the summer months
Still today, 5 years later, Jett has three large hardened welts on his belly.
Had Jett remained on drug, by my own estimate, he would have 250 of these injection sites. There is no way Jett could “manage” this risk and in the context of all of the safety risks you heard about today, these are the least severe.
So as you deliberate, please remember:
-the risk to patients like Jett is not manageable
-for patients like Jett who are exon 51 amenable, we need other options
-and for all those patients in our community who are not exon 51 amenable, the unmet need remains and is significant.
Thank you.

Help get this film funded!

May 7, 2015

We have launched a kick-starter campaign to finish a documentary that follows four families in their quest to get access to drug(s) that could save the lives of children with Duchenne.

 Please watch and share To the Edge of the Sky.  

Share the link that contains the trailer – through social media, by email – however you can. #totheedgeofthesky

The more eyes on the trailer, the faster we will spread our collective stories of how much we love our children, and how we won’t stop until we succeed at finding a way to save their lives.

We hope that this film will have an important impact on how people see Duchenne Muscular Dystrophy and the struggles that the boys and their families face every day as they seek access to medication. Additionally, we hope it will help convince the FDA to pay more attention to the special needs of people that are suffering from rare diseases. Ultimately, we want to rally the masses and get the global community involved in reform–the more people who get involved, the more attention this rare disease will receive.

 Please watch the trailer and help us spread the message.