FDA Accelerated Approval of Sarepta Therapeutics’ exon skipping therapy to treat Duchenne muscular dystrophy was marked by a “big day of celebration,” said Debra Miller, CEO of nonprofit group CureDuchenne.
The Exondys 51 eteplirsen injection from Sarepta is the first approved therapy for Duchenne, a rare genetic muscle-wasting disease that typically puts boys diagnosed with it in wheelchairs by their teens and kills them in their 20s.
Beyond the approval, CureDuchenne had good cause to celebrate as the organization contributed funding in the form of a grant to Sarepta.
“We funded Sarepta Therapeutics back in 2010 when they were on clinical hold and did not have the money to do toxicology studies necessary to move to clinical trials,” said Miller. CureDuchenne collaborated with Children’s National Medical Center in Washington, DC and the Foundation to Eradicate Duchenne to fund the studies so the company could move into clinical trials.
CureDuchenne Ventures, the investment arm of the organization, has scored three exits with drug development companies, generating profits that have been returned to invest in new projects.
Speaking with Miller, it is easy to forget that she is not a professional biotech business development executive. She is something more serious than that.
“I am a mom and I want to care for my son,” she said. Yet you want to tread carefully here, much as you would finding yourself walking between a mother grizzly bear and her cub.
Debra Miller takes drug development very seriously when it has anything to do with Duchenne Muscular Dystrophy. And if you want to learn how passion and persistence can pay off, you will want to hear her speak during the BIO-Europe® session, “An autobahn for orphan drug development,” on November 7, 2016 in Cologne, Germany.
Miller and her husband Paul founded CureDuchenne in 2003 after their son was diagnosed with Duchenne muscular dystrophy. They have built a fiscally-responsible business sharply focused on doing just what the group’s name states.
“I have learned things that I didn’t know even existed. Every day is continuing education,” she laughed.
In the rare disease space, CureDuchenne has a similar model to the Cystic Fibrosis Foundation, according to Miller.
“Our model is different than traditional nonprofits; I run CureDuchenne more like a business than a traditional nonprofit. The organizations that existed before had not funded significant translational research. Where there is a persistent problem you need to innovate to find the solution.”
One of Miller’s innovations in venture funding is what she calls the “Mom Filter.”
“I am not going into a project for a financial return, that is secondary. I want to go into these investments based on the science and whether this is really going to help the kids. It’s all about being a Mom, just a Mom. Going into a project with that motivation, success is going to follow,” she said, and is pleased to add, “We have had three successful exits.”
In 2004 she recalled, “We had USD 10,000 in the bank yet we committed to a USD 1.3 million contract with a Dutch company called Prosensa. It was ‘hold your nose and jump in the pool.’ We were going to do it, we didn’t know how. There were many sleepless nights, but our son has Duchenne, and when you are a parent, you just do it.”
The Prosensa molecule was subsequently licensed by GSK, which Miller said conducted a big but inconclusive trial. Prosensa was able to regain the rights to the molecule, then BioMarin Pharmaceutical bought the company.
In January 2016 Duchenne Ventures funded Bamboo Therapeutics, a gene therapy company spun out of the University of North Carolina.
“This project was so promising that Pfizer bought Bamboo in August, so that was a real quick turn-around,” Miller said.
Every company talks about their commitment to patients, and Miller finds that many pass the Mom Filter.
“I really respect our biotech partners,” she said. “Yes, most are public companies with shareholders, yet I have come to know their development teams really well over the years and there is no way these biotech executives do not fall in love with their patients and the kids. They have shown compassion and great emotion and sympathy for the disease group they serve. My hat is off to them because they go through a lot of ups and downs. And I see and admire their perseverance and dedication. ”
At BIO-Europe Miller will bring the Mom Filter to scan the landscape in rare disease drug development.
“One of the big changes is technology—being able to identify the root causes of disease and understand some of the peripheral effects to be dealt with. This creates a big advantage because when you know the problem, science can start working on the solution.
“And there is the Accelerated Approval designation, which is how we finally got the Sarepta approval. This marks a huge shift for rare diseases because it is always going to be difficult for companies to do large placebo controlled trials with a tiny patient population. And the Orphan Drug designation incentivizes companies to address these needs of rare disease community. That is very important.
“Then there is the pricing. It is going to always be quite an issue because these drugs can be upwards of USD 300,000 per year. It is going to be interesting to see how open or resistant the insurance companies and government payers will be, but so far, it seems that the payers will not want to refuse a drug when there are no other options.”
A major change, she said, is the groundswell of patient activism and advocacy, thanks in part to the awareness created by Duchenne organizations, but more importantly in the ways rare disease communities are coming together and being recognized.
“Unfortunately we do not have a movie star with Duchenne, like Michael J. Fox or athlete like Lou Gehrig. These are pediatric diseases and these kids don’t often live long enough to make a name for themselves. That leaves it up to parents to fight to get recognition.
“We have seen rare disease become more of a movement instead of a collection of separate diseases. These different disease communities are going to band together to get the awareness and the funding to get to treatment. Global Genes is an organization that provides valuable services to many rare disease groups and patients.
“It is important for families to know the social media activities are important, but that the work does not stop there. All the advocacy, lobbying and social media in the world is only effective if we have drugs before the FDA. We need to keep funding these companies, and we need the support of the patient community to raise the funds to keep the pipeline full so we have more shots on goal.”