Cf news14 issuu 10

10 Years Although we’ve come a long way, we’re not celebrating.

Yet.

MISSION: We direct

money into the hands of researchers who have the best shot at developing a treatment or cure for Duchenne muscular dystrophy. Our goal is to cure Duchenne in time to save Charley’s life and the lives of thousands of boys like him.

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CHARLEY: Basketball aficionado.

Indie-rock fan. Foodie. Fashion-forward dresser. Aspiring lawyer.

Charley was diagnosed with Duchenne in 2004, when he was three years old. Today he’s a teenager who listens to extremely loud music and obsessively follows NBA and college basketball. He wants to be a lawyer when he grows up. We want to give him the chance to get there.

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2004: Charley enjoys a carousel ride.

2005: Brothers Sam and Charley

2006: Charley, Sam and Maisy

Dear Friends and Supporters, When we started out ten years ago, Charley’s Fund was simply two determined parents with a couple of laptops and a spare bedroom. The division of labor was simple. Benjy scoured medical journals for leads on potential treatments. Tracy wrote her heart out to family, friends, colleagues, and neighbors seeking contributions. All we wanted to do was save our little boy.

participants in conversations with the FDA as the first therapies for Duchenne face regulatory approval. Yet despite this evolution into a highly effective organization creatively solving multiple problems, our stripped-down essence remains. We have one and only one goal: to develop treatments quickly enough to save Charley’s life and the lives of all kids with Duchenne.

Ten years into the business of driving research to treat and cure Duchenne muscular dystrophy, Charley’s Fund has grown into a sophisticated, efficient organization with global impact. We’ve directly funded more than $14 million in medical research and leveraged an additional $17 million from organizations that look to us for leadership in their funding decisions. We have spun out a biotech company that is developing three promising Duchenne therapies, the first of which is now in a clinical trial at five leading hospitals across the country. We have become active

We are decidedly not calling this ten-year milestone our “anniversary,” because we do not celebrate the passage of time. The celebration will come when we succeed. Instead, this is an opportunity to reflect on lessons learned so we can expand what we are doing right and fix what we need to improve. As we move forward, our order of the day is organize our efforts to amplify our impact. In June, we took a crucial first step toward bringing those words to life. We created the position of Chief Operating Officer and brought on Laura Dalle Pazze, formerly of

2009: Charley and a friend

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2010: Pancakes!

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2011: Charley poolside in Miami

2007: Maisy, Sam and Charley in Boston

2008: Charley gives Stella some love.

The Michael J. Fox Foundation for Parkinson’s Research, to fill the role. We took our time (10 years!) creating the right role and filling it with the right person. Based on the impact Laura has had in just four months, we are extremely bullish about this choice.

In the meantime, while we’ve been continuously self-assessing, making improvements, and finding ways to clear hurdles that stand in the way of new treatments for Duchenne, our “little boy” has become a teenager. And just like the fund that bears his name, Charley has matured but kept his essence. He still has that penchant for adventure, and this past year he was lucky enough to experience some incredible travel. He spent a week site seeing (and eating cheese) in Paris and was treated to a once-in-a lifetime safari in Tanzania! Charley’s on a tear to live a full life, and Duchenne is not gonna stop him.

While Charley’s main resources are inner reserves

and a positive outlook, the task at hand for Charley’s Fund requires more than that. Namely, millions of dollars to identify and spur the best research so that treatments become available in time to save this generation of kids with Duchenne. For that, we have you to thank. Your contributions to our effort have enabled us to achieve profound, meaningful impact in our first ten years.

Ambitious goals are the only way we will defeat Duchenne, and we are doubling down with all we’ve got. In honor of our tenth “un-anniversary”, we hope you will join us in doubling down your support to help us reach $10 million in the next three years. If you’ve got our backs, we’ve got this!

All our gratitude,

Benjy and Tracy

@RupjaniB

2012: Charley and Maisy

2013: Charley shows off his new shades.

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2014: Charley at his bar mitzvah

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Charley’s Fund Today Ten years in the business of driving research to develop treatments and a cure for Duchenne, our commitment to urgency, impact, and results is stronger than ever.

Our Mission: Accelerate the delivery of life-saving treatments in time to save Charley and all boys with Duchenne muscular dystrophy.

Our Values:

Focus

Efficiency

Creativity

Our sole focus is to identify and advance medical research and drug development projects that are the best shots on goal to treat and cure Duchenne. Our definition of success is impact on the lives of children with the disease. Everything we do must have a yes in answer to “Will this help get medicines to kids who need them faster and more efficiently?”

We live by the mantra “lean and mean.” This no-frills approach applies to everything we do — our timelines, our funding, and our strategies to move research forward.

Since inception, $0.95 of every dollar spent has gone directly to our programmatic efforts.

We inject creative thinking and nimble problemsolving for faster results.

The traditional drug development process is slow, unwieldy, and subject to entrenched bureaucratic constraints. This businessas-usual approach will not defeat Duchenne. Drastic circumstances call for bold measures. We identify bottlenecks and create solutions, rather than do what’s usually done.

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Leverage Our organization is small but mighty. We make our investments count extra by aggressively pursuing leverage in two ways: 1) Strategic funding points where our dollars have extra impact because other stakeholders — like the NIH or big pharma — are not injecting funds.

2) Leadership opportunities where our funding rallies more money from other patient organizations.

Faces of Duchenne:

Our Inspiration

Gus: Animal enthusiast. Music lover. Second-grade clown.

Budding wildlife ecologist.

Nash: Art aficionado. Long-time admirer of Jean Michel Basquiat. Favorite activity: chilling with family and seeing movies with dad.

Nash is is an artist. He loves to visit the world’s top museums, especially his favorite – the MOMA in New York City. Although Duchenne has put Nash in a power chair, he still has full use of his upper body. We are determined to make sure Nash maintains this strength so he can keep sketching and painting until his work is hanging in a famous gallery.

Gus was diagnosed with Duchenne in 2009, when he was four years old. Now a seven-year old boy, Gus is eagerly soaking up every minute of being a second-grader. He loves music, dancing, swimming, and animals, and when he grows up, he wants to be a wildlife ecologist. Our team hopes one day our research will enable him to do some of his own.

Javier: Quirky. Bacon-lover. Wii bowling champion. Head over heels for Katy Perry and wants to marry a girl just like her.

Joseph: Daredevil. Snuggler. Football fan. Master sledder.

Magic-trick master. Doesn’t ever want to slow down.

Joseph was diagnosed with Duchenne in 2008 at age five. He’s an easy-going middle-schooler who spends as much time as possible adapting to his body’s limits by taking photos of his middle school football team rather than playing, learning how to play sled hockey, playing catch with his dog, and cheering on his favorite pro teams. He’s a little boy with big determination not to be defined by Duchenne. We are determined to develop treatments that will help Joseph live a life with no limits.

Javier was diagnosed with Duchenne in 2007 at age three. He’s grown into a hysterical, quirky kid who loves life and proudly marches to the beat of his own drum. He loves his Nintendo Wii and Katy Perry almost as much as he loves writing risqué mad-libs and eating bacon in any culinary format. He’ll never outgrow the bacon, but he dreams of becoming a young man and starting a family with the love of his life. Our goal is to help him outgrow Duchenne, too.

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Research

Q&A with Benjamin Seckler, MD Benjamin Seckler, MD (a.k.a. Charley’s father) is President of the Board of Directors. He leads the organization’s efforts to identify the most promising opportunities for Charley’s Fund to accelerate research. Ten years in, Dr. Seckler shares his thoughts on the role Charley’s Fund plays within the changing Duchenne landscape and how he thinks about achieving results.

Question: Charley’s Fund completes its tenth year of operation this November. Why is this an important moment?

Answer: Over the course of 10 years, you gain knowledge, experience, resources, connections. You see surrounding circumstances evolve and new opportunities emerge. But for all we’ve gained, we’re still racing against the clock as we lose our most important resource: time. We are seizing this milestone as an opportunity to reflect on the decade of experience under our belts and refresh our strategy to ensure we make the best possible choices and lay the best possible plans.

Q: What are some of the things you have learned over these ten years?

A: Most importantly, we’ve learned that what we’re doing is working. When Charley was diagnosed, there was not even one clinical trial for a hopeful drug to treat Duchenne. Today, the first treatments ever — two genetic treatments that could benefit about 13% of boys with DMD — are just months away from applying to the FDA for approval. We funded both drug companies early on, and we also played a major role in influencing the FDA’s decision to consider accelerated approval. We created our own biotech company that is conducting clinical testing for an antifibrotic, anti-inflammatory treatment that could benefit all boys with Duchenne, and the initial clinical data looks positive. We partnered with a group of scientists at the University of Buffalo to translate their exciting discovery — spider venom might help kids with Duchenne! — into a treatment. We now can hope that boys with Duchenne will have more time, and we can use that time

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to continue building the cocktail of drugs that will turn Duchenne from an aggressive killer into a manageable chronic condition.

But we’ve also learned that we have a lot more to do, and the role our organization plays is critical. Early success means research moves forward to the more expensive stages of development, which means a lot more money is needed. And more research approaching clinical trials means we desperately need more effective, consistent tools to measure results.

The importance of our role as a creative problem-solver can’t be overstated. Drug companies and the FDA are goliaths that have every incentive to follow established processes and procedures.

But established processes and procedures are too slow and too risk-averse for Duchenne. We have to be the ones who challenge the status quo and identify and enact opportunities to do better.

Q: How does Charley’s Fund make a difference when the government and the pharmaceutical industry have such deep pockets for funding research and drug development?

A: As a patient-founded organization, we play a unique role in making research happen better and faster. The amount of capital we can raise and fund into research is small compared to what the government contributes to basic science and what pharma companies invest in later-stage clinical trials. But we step in at a point so critical it is often referred to as “the valley of death.”

This is the point in development that usually comes after the government funding stops, but before the research is de-risked enough for a pharmaceutical company to step in. Sadly, many potential therapies languish there and never make it to patients.

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Q: What’s next in store for Charley’s Fund?

A: Ten years ago, we decided to plant a large number of seeds in various therapeutic areas. The individual seeds we planted have either flourished (like exon-skipping) or floundered. Additional players have taken up ploughshares and are effectively nurturing certain sectors. This enables us to tighten our focus on the best research opportunities and develop tools that will help all new drugs move faster.

A therapy that promotes healthy muscle regeneration will be an important component of the Duchenne medicinal “cocktail.” We recently partnered with Stanford University to support a stem cell program, and we will be delving further into this exciting area of muscle regeneration in the next couple of years. We also need better tools to accelerate clinical research. More therapies are approaching the clinical stage, which is hugely encouraging.

But we need to ensure that new treatments move through clinical trials as quickly, efficiently, and effectively as possible. There’s little financial upside in developing these tools, so it is a prime opportunity for us to have a major, meaningful impact. The same applies to working with the FDA to identify creative solutions to the regulatory path all therapies will face before they can be approved.

We don’t have one company’s interest in mind — we have long, healthy lives for all boys with Duchenne in mind. So we have an opportunity as a neutral party to drive that conversation.

“The importance of our role as a creative problemsolver cannot be overstated.

We have to be the ones who challenge the status quo and identify and enact opportunities to do better.”

Q: How does starting a biotechnology company figure into this equation?

A: It may sound cliché, but when we say “do whatever it takes” we mean it — including founding a new company and assembling a veteran management team to run it. As parents, we just could not live with the fact that certain assets were languishing undeveloped despite serious potential to help our son and other children with his disease. Akashi Therapeutics picked up three of those assets and has a growing pipeline of promising drugs.

Q: How is Charley doing?

A: Under the circumstances, I would say he’s terrific. Duchenne is a beast and there are certainly times when he gets down.

Participating in a clinical trial with the exhausting travel schedule and many doctor visits is a drain on the entire family. But he has an amazing ability to put the grief aside and get back to the business of enjoying life. At the moment his top three “things I love” would probably be the alt-rock band Arctic Monkeys, Key and Peele comedy skits, and coconut Chobani yogurt.

Charley (left) and Benjy.

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Innovation in Action:

Meet Akashi Therapeutics N

othing’s more frustrating than shelved potential. But in 2010, Charley’s Fund saw just that when Dr. Benjy Seckler read about a compound showing promise in animal models of Duchenne. The therapy, initially created for a different disease, hit a dead-end when tested in humans: it triggered nausea and vomiting. This and other roadblocks proved too daunting for the Israeli biotech that owned the asset, and development was halted.

Knowing this compound had potential to benefit children with Duchenne, Charley’s Fund would not take no for an answer. We had to figure out a way to take the molecule, address the side effects, and get it into a clinical trial for kids with Duchenne.

The solution turned out to be creating a new biotech company, which we called DART Therapeutics.

Fast-forward 3 years. The drug is now called HT-100 (affectionately referred to as “Halo” because its main ingredient is halofuginone), and is presently completing a Phase 1B/2a trial in which Charley is participating. Early data was released this summer at a scientific conference, and it is encouraging. If DART could do this for Halo, why not for other therapies?

Our company got Halo into a clinical trial in just 24 months, proving its ability to bridge the treacherous gap between the laboratory and the bedside. To highlight this expertise, we rebranded the company as Akashi Therapeutics (see “What’s with the name?” sidebar). Akashi is now a clinical-stage biopharmaceutical company with a growing

pipeline of promising drugs and the talent and track record to take them into boys with Duchenne as fast as possible.

The track record: • Identified and acquired promising drug from Israeli biotech • Reformulated HT-100 to address GI intolerability • Started clinical trial for Duchenne patients at five hospitals across the U.S. • Validated a novel biomarker to help determine if the drug is working • Earned Fast-Track status from FDA, which will expedite the review process The therapies: HT-100: An anti-fibrotic, anti-inflammatory oral medication completing Phase 1b/2a testing later this year. Long-term toxicology studies underway to support an FDA filing for approval.

DT-200: A powerful muscle builder with positive early data and potential to treat multiple neuromuscular diseases. Ready to enter testing in healthy volunteers.

AT-300: A calcium-channel inhibitor derived from the venom of the Chilean Rose Tarantula with demonstrated potential to positively affect calcium homeostasis, a known problem in Duchenne that is not being addressed by other companies.

What’s with the name?

Akashi Therapeutics borrows its name from the longest, strongest suspension bridge in the world. It was created sixty years ago in Japan after a ferryboat disaster killed 168 children. Public outcry inspired the government to accomplish something it had never done before: build a passageway to enable people to cross the notoriously treacherous waters safely. For Akashi Therapeutics, the name signifies the mission: boldly bridge the chasm from promising science to safe and effective therapies to save our boys.

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Michael Staten Island, NY

Nathan Highland Village, TX

Our Team Meet some of the boys participating in the HT-100 trial. Their dedication and bravery is helping us determine whether this anti-fibrotic medicine is a safe and effective treatment for Duchenne.

Nicolas Bel Air, MD Joey Troy, MO

Jasdeep Cleveland, OH Mark Havre de Grace, MD

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Innovation in Action:

Summit Therapeutics Update

S

ummit Therapeutics is a UK-based company that is developing a treatment for Duchenne.Charley’s Fund supported Summit’s research back in 2008, and things moved along so well that Summit struck a deal with BioMarin, a leading pharmaceutical company with the resources to finance clinical trials. But when the compound was tested in a phase 1 trial with healthy adult volunteers, it didn’t get distributed effectively throughout the body. Biomarin backed out.

of preclinical research at a large pharmaceutical company for more than a decade. We joined five other nonprofit foundations to supply Summit with the funds needed to reformulate the compound and redo the phase 1 trial in healthy adult volunteers. The trial was a success, and the company moved the drug into the next phase, a clinical trial in boys with Duchenne.

Summit wanted to keep the promising compound moving forward, but traditional scientific advisors cautioned potential funders that if pharma wasn’t interested, the asset must not be viable. Before we allowed this drug to succumb to the notorious Valley of Death, we wanted to make sure it truly was a dead end. We hired a preclinical research expert to evaluate the data and share his opinion with several partner organizations.

He agreed with Summit that if they reformulated the drug, there was a good chance they could overcome the bioavailability problem. Our advisor had seen this work several times before in his role as director

Today, Summit is preparing to build on that success and initiate two more clinical trials for Duchenne boys. The data gathered from the trial we financed is also informing the development of Summit’s other drugs to treat Duchenne.

Our support of Summit’s drug development program illustrates two of our core tenets in action.

LEVERAGE – We formed a partnership with three other foundaCREATIVITY – Charley’s Fund makes sure that the advisors we tions so we could all contribute funding but Summit wouldn’t have hire have diverse backgrounds and varied perspectives. When they to maintain four separate points of contact. work together to exchange data and ideas, it brings about We injected funding when Summit needed a professional cross-pollination for innovative results. In it most to prove that their drug was still this case, a key advisor was not a Duchenne expert but viable. We knew that if they succeeded, forhad decades of experience directing preclinical developprofit investors would follow. Sure enough, ment at a large pharmaceutical company. When we put Summit soon raised more than $25 million to his head together with the heads of experts in the field of continue developing the pipeline. The funds Duchenne, together they forged a path forward for a promising drug we provided to Summit were returned to us so we could reinvest the that was almost halted in its tracks. cash in other promising therapeutics. To boot, if and when Summit starts making money from sales of the drug, our group will get an extra ROI to put back into advancing additional medical research.

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Innovation in Action:

The Race to Yes A

t the risk of sounding like a broken record, we’re going to start this section with a statement you’ve definitely heard by now: Charley’s Fund’s sole mission is to get new treatments to children with Duchenne as quickly as possible. Everything we do comes back to our guiding principal: Will this expedite access to new treatments?

Last year we realized that the regulatory process was a rate limiting step for an exciting new therapy, and we needed to get involved to speed up the process. One of the companies developing a suite of exon skipping treatments had promising data from a two-year clinical trial with 12 children. The company was waiting for FDA guidance on how to proceed toward regulatory approval. They asked FDA for input on what additional data they would need and whether the Agency would consider accelerated approval (see sidebar) based on the fact that the drug produces dystrophin, the protein boys with Duchenne are missing. But months and months went by, and that guidance was not forthcoming.

What is “Accelerated Approval?”

The FDA can grant conditional approval to a drug if it acts on a biomarker that is reasonably likely to lead to clinical benefit. Accelerated approval is especially important in diseases where time is of the essence. For example, a cancer drug may shrink a tumor in 3 months, but whether that leads to a longer and/or healthier life would take many more months and even years to determine. Accelerated approval allows FDA to approve the drug based on the fact that it shrink the tumor. The drug company has to confirm that this leads to clinical benefit, and if they fail to do so the approval can be revoked.

FDA

Race to Yes Elected Officials

General Public

We formulated and executed a highly effective advocacy plan that cleared the way for progress. The Race to Yes (R2Y) came into being to help eliminate unnecessary regulatory delays that get in the way of access to safe and effective treatments. R2Y has three prongs: communicate directly with the FDA, garner support from elected officials, and rally the public to demand justice.

In 2014, the Race to Yes momentum brought experts from around the world to Washington to meet with FDA officials and speak at a congressional hearing about Duchenne and the promise of this new exon skipping platform. We collected 106,000+ signatures on a petition that got the White House to weigh in, signaling to the world that the commitment to saving our children comes from the top! We organized families across the nation to speak to the media so the FDA and drug companies know that the world is watching and will not tolerate unnecessary waiting. The Race to Yes remains active on all fronts. We will not let up until our children have access to safe and effective treatments.

Cool Fact:

The Race to Yes garnered incredible star power. Celebs with heart (and tons of Twitter followers) like Sarah Jessica Parker, LL Cool J and Mark Cuban helped get the word out until our petition reached 100,000 signatures and President Obama’s desk.

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Fundraising Race Against Time

The Race Against Time has grown to be a HUGE annual event in NYC with hundreds of participants every year and a spinout version in Great Barrington. You may be surprised to learn about this incredible event’s humble beginnings.

SETTING: Charley’s Fund cramped basement office, July 2009. Phone rings.

Tracy: “Hello, Charley’s Fund. This is Tracy.”

Friend of Tracy’s sister: “Hi Tracy, this is Ellen. I’m thinking about trying my hand at a fundraising event.

What do you think about doing a run?”

And that is how it all began…one energetic supporter giving it a shot and sticking with it over time to create the fabulous Race Against Time! To date, the “RAGT” has raised six hundred thousand dollars to help us in the fight to end Duchenne.

A Yearly Gala Event:

Matt’s Promise Benefit At left, participants in the Race Against Time in New York, 2014, with event organizer, Ellen Wilner (back row, second from left in sunglasses).

Above right, the Race Against Time event in Great Barrington, Mass., was another successful day for Charley’s Fund.

Spread the Smile: It All Adds Up! Simple acts of generosity add up in a big way. Supporters across the country have dedicated their special occasions to raising funds for DMD research by requesting donations in lieu of gifts.

Check out these stats: Donations in honor of bar or bat mitzvahs................ $350,000+ Donations in lieu of birthday gifts..............................$100,000+ Donations in honor of or in memory of someone........ $110,000+ TOTAL........................................ More than half a million bucks!

Do you have a special occasion coming up? We can help you do all of the above with personalized cards including a cute pic of Charley and a brief explanation of the Fund. Contact us at sarah@charleysfund.org to get started.

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Charley’s Fund is the recipient of proceeds from the Matt’s Promise gala each year in May at Cipriani Wall Street. The glitter, the sparkle, the lively auction and the big-name performer always draw a huge crowd. This year, Chicago performed an incredible show.

Through this spectacular event, Matt’s Promise has raised millions to help Charley’s Fund drive forward confidently and aggressively.

We’re doubling down our efforts to defeat Duchenne in time to save Charley and thousands of boys living with DMD. We’d like to double our speed. Our creativity. Could it even be possible that we double our determination?

In honor of the milestone of our tenth anniversary, we’re asking our supporters to help us double down to reach $10 million over the next three years. If all our supporters pick one way to double down, we can do it.

Here’s how you can double down: Double down your giving: take your usual gift and double it this year.

Double down your time: in addition to giving a gift this year, host a grassroots fundraiser for Charley’s Fund — a walk, a cookie drive, a dinner party… it all adds up!

Double down with a friend: introduce Charley’s Fund to a friend, and ask them to be your partner in doubling down to defeat Duchenne.

Double down over time: Want to double down but not all at once? Set a date and give some now, give some later.

Best buds (and cousins) Theo and Charley double down the fun at a game (2014).

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“

I have been impressed with the fierce moms who have advocated for DMD drug development and treatment. Their advocacy is inspiring.

I consider them effective because they have combined personal experience with a discussion of scientific data. Indeed, they have in many ways become experts in the field.

John Whyte, MD, MPH Director, Professional Affairs and Stakeholder Engagement, Food and Drug Administration

Over the past 10 years, Tracy and Benjy have built Charley’s Fund into an organization that has advanced the field of Duchenne research by maintaining a focus on funding projects that are impactful and relevant to patients. Charley’s Fund first collaborated with Sarepta in 2007 to help advance our exon skipping platform and brought intelligence and passion to ensure a successful partnership. Their effort goes beyond the millions of dollars they bring to projects and includes a strong and credible voice to advocacy campaigns that will surely lead to faster development and access to new Duchenne treatments.

Chris Garabedian, CEO Sarepta Therapeutics It is always hard to implement change in government policy and procedures, but you are all doing a wonderful job and your efforts are greatly appreciated, they really are. At least FDA is listening! Thanks for all that you have done for awareness, research and FDA approval.

I am looking forward to seeing great progress in Duchenne treatment and therapy in 2015.

Melissa Spencer, PhD David Geffen School of Medicine at UCLA Co-Director, Center for Duchenne Muscular Dystrophy at UCLA I am so grateful for Charley’s Fund and Akashi Therapeutics who have brought the HT-100 (Halo) trial my son is currently a part of to our community. With this drug and a few others currently in the pipeline, I hope we can turn things around for these beautiful innocent children who deserve to have healthy and full lives.

Mom of an 11-year-old with DMD, Texas In the next 12 months, heads will be turning toward rare disease space & breakthroughs in DMD- @CharleysFund Seckler. #MAPavilion #BIO2014 @MassBio @CharleysFund Way to go Akashi Therapeutics! This is what commitment means. @RupjaniB

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@charleysfund This has become so much more than an investment for me. You are amazing & I pray you will benefit from your incredible efforts.

Biotech investor

To read the news on all of our projects, make a donation or shop our fun Charley’s Fund products visit charleysfund.org.

Charley’s Fund, Inc. 635 Main Street, Suite 3 Great Barrington, MA 01230

BY THE NUMBERS:

$30,000,000 directed to research since 2004

100,000+ supporters engaged to defeat DMD

30,000+ boys living with DMD in the United States

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cents of every dollar spent goes to research efforts

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nonprofit community partners galvanized for action

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treatments in development with our support

clear goal: accelerate research to save our boys