Not only does this critical step signal the continuation of the phase 1 human safety trial and the first achieved milestone for AT-1501, it is the first time a nonprofit drug discovery center has successfully brought a new molecular entity, this type of experimental treatment for ALS, from inception through to the clinical trial phase.
"Our disruptive nonprofit, and its laser-focused ALS research model, is paying off. Today I have new hope we will uncover effective treatments and an ultimate cure for ALS – and quite possibly, in my lifetime," says Augie Nieto, Chairman of both ALS TDI and Augie's Quest. The business leader and fitness industry icon, who has lived with ALS for more than a decade, is instrumental in advancing the two organizations' collective vision: to fast-track the cutting-edge science needed to fight ALS by urgently funding more and better drug developments, like AT-1501, and move them into clinical trials to benefit people with ALS.
"ALS isn't incurable, it is underfunded. So that's where we've devoted our energy this last decade, inspiring tens of thousands globally, particularly our friends in the fitness industry, to join our quest and #EndALS. Collectively, Augie's Quest and ALS TDI have raised more than $150 million for our shared cause. These continued gifts from our committed community are making this clinical trial – and someday an ALS cure – a reality," Nieto adds.
Following the acceptance of the Investigation New Drug (IND) application with the Food & Drug Administration (FDA), this phase 1 safety trial is now enrolling both healthy volunteers and a total of eight people with ALS to determine the overall safety and tolerability of AT-1501. This experimental antibody is designed to bind to and block a protein target called CD40 ligand (CD40L), shown to be overactive in ALS patients. The trial will also be monitoring the drug's pharmacokinetics, or in other words, how long the AT-1501 drug circulates within the body.
"This is an important victory for the ALS community," said Steve Perrin, PhD., Chief Executive Officer, ALS TDI. "Because of the complex nature of the disease, it is extremely challenging to take ALS lab discoveries and translate our research into well-designed clinical drug trials. But that is what we're dedicated to doing. Seeing our lead candidate, AT-1501, move forward is a great sign our approach to ALS drug development is working and a strong indication we will be able to advance other promising discoveries from ALS TDI's deep pipeline, as well."
Augie's Quest, together with its many generous donors and corporate cause marketing partners, have raised more than $5 million over the past five years and is a leading source of support in the development of AT-1501. Fitness industry efforts to raise urgent funds and awareness on behalf of Augie's Quest, including Orangetheory Fitness' system-wide annual #IBurn4ALS challenge, Zumba and Strong by Zumba's upcoming January 2019 global campaign, ClubCorp's Charity Classic events, Shea Homes' commitment, countless IHRSA member activities (a global community of health and fitness professionals), and many more, are helping to fast-track this critical ALS research.
About Augie's Quest to Cure ALS Augie and Lynne Nieto have been taking on ALS the only way they know how – with honest humor, fierce friendship and one-of-a-kind business savvy. Augie is known as the "Steve Jobs" of the fitness industry, a business icon finding career success building the Life Fitness and Life Cycle brands, ushering in popular work outs and new forms of cardio, and getting the masses to hit the gym. Since their ALS diagnosis nearly 14 years ago, the couple has been on a unique quest, rallying family, friends and the entire fitness industry to fund innovative science, effective treatments and an ultimate cure through their nonprofit, Augie's Quest to Cure ALS. In collaboration with the ALS Therapy Development Institute, they have raised more than $150 million to fund essential research and develop treatments and cures. Because ALS isn't an incurable disease; it's only underfunded. Augie's Quest's tireless work has driven innovative science forward at an accelerated pace–including one of the most promising drug treatments yet, AT-1501. The quest for a cure is not yet over, but this incredible community is still fighting to end ALS once and for all. Join us at www.augiesquest.org.
About Anelixis Therapeutics & ALS TDI Anelixis Therapeutics entered into an exclusive licensing agreement with the ALS Therapy Development Institute (ALS TDI) in 2015 to develop AT-1501 and other anti CD40L antibodies as a potential treatment for ALS and other disease indications. ALS TDI published its initial discovery of a link between ALS disease progression and the innate immune system in the premier science journal, Nature Genetics, in 2009. In that paper, ALS TDI scientists described the robust preclinical validation work they did to show disease progression was significantly slowed with the administration of a treatment to block the activity of a specific receptor on specific cells called CD40.
ALS TDI created Anelixis Therapeutics in 2012 as a for-profit subsidiary to help bridge the gap in funding needed to further develop a monoclonal antibody based on original research done in its labs by attracting private investments. Anelixis has been successful at raising funds, managing the manufacturing of large-scale quantities of GMP and clinical grade AT-1501 in partnership with Lonza in the United Kingdom, and working with more than a dozen experts to gain Orphan Drug Designation from the FDA in April 2018 for AT-1501 and submit a successfulInvestigation New Drug (IND) application to the Food & Drug Administration (FDA); leading to this phase 1 trial being launched. A more comprehensive timeline tracking the development of AT-1501 can be viewed online here.