Us in Action

We’re not sure if you noticed, but in 2014 we updated our mission statement to add a critical phrase. In addition to supporting the most compelling research, we “drive new solutions to translate promise into results.” Here are some concrete examples of what we mean by that:

A promising therapy clutched from the notorious “valley of death” and given new life

Background

Summit Therapeutics is a UK-based company that Charley’s Fund has been supporting since 2007. The company made good progress on its “utrophin modulator,” and even struck a deal with a deep-pocketed pharma partner to conduct the costly human clinical trials that would be needed to determine if the drug is a safe and effective treatment for Duchenne.

Problem

The first clinical trial in humans didn’t go so well. The drug didn’t have great uptake in healthy adult volunteers. If a drug can’t get where it needs to go, of course it can’t do what it needs to do. The pharma partner gave up, and Summit did not have the funds to keep the program alive.

Solution + Status

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. He advised that if Summit 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 of preclinical research at a large pharmaceutical company for more than a decade.

Charley’s Fund — together with three nonprofit partners — provided the funds to reformulate the compound and try again in healthy adult volunteers. The change improved the drug’s bioavailability, and the company proceeded to the next step: testing the drug in boys with Duchenne. Meanwhile, Summit RAISED $XX IN FOLLOW ON FUNDING FROM AN IPO, which has enabled them to build out their pipeline of Duchenne drugs. Today, Summit has multiple clinical trials going on for boys with Duchenne in Europe and the US.

Using technology to help get clear, objective results from clinical trials

Background

We have been funding promising research since 2005, just six months after Charleyt was diagnosed. We started out supplying funds to scientists and companies who were already working on DMD treatments, but needed money to go faster. As we dug into the landscape, it became clear that some potential therapies weren’t just moving slowly -– they weren’t moving at all.

Problem

In one of his late-night internet research sessions years ago, our co-founder and President Dr. Benjy Seckler read about a compound that showed promise in animal models of Duchenne. An Israeli biotech company had planned to develop the compound to treat cancer-induced muscle wasting, but the roadblocks proved too daunting and development was halted.

Solution and Status

Knowing this compound had potential to benefit children with Duchenne, Charley’s Fund would not take no for an answer. We hired a team to conduct thorough due diligence on the molecule, and when all agreed that this compound did indeed have significant promise, we formed a biotech company to acquire the rights and develop the drug as  a treatment for Duchenne. Akashi Therapeutics got the treatment into a clinical trial in just 24 months, proving its ability to bridge the treacherous gap between the laboratory and the bedside.

The drug (HT-100) is presently in a Phase 1B/2a trial in which Charley is participating. Early data was released this summer at a scientific conference, indicates the treatment increases muscle strength in boys with Duchenne. Akashi has partnered with Grunenthal, a European family-owned pharmaceutical company, to finance HT-100’s full clinical development program and commercialization. Meanwhile, Akashi acquired two more compounds that were stuck in neutral despite their potential to help kids with Duchenne. DT-200 is a powerful muscle builder with positive early data that was dropped by its original owner for business strategy reasons. AT-300 is a calcium-channel inhibitor discovered by a scientific team that did not have the expertise or the means to take their exciting discovery from the lab to the bedside.