Australian Immunotherapy Selected for Revolutionary US Clinical Trial Platform Debut

An Australian immunotherapy drug will receive a huge boost when it is tested with four other new concepts across 54 clinical sites across the USA in a search for an effective treatment for ALS (a motor neuron disease).

This bold initiative will see Implicit Bioscience’s IC14 entering an elite pivotal clinical study in the first Platform Trial concept to be rolled from early 2020 by the Sean M. Healey & AMG Center for ALS at Mass General (Healey Center), subject to FDA approval. 

“We brought together top leaders from around the world to dramatically rethink how we design ALS trials and create a groundbreaking platform trial program,” says Merit Cudkowicz, MD, MSc, director of the Healey Center. “Our new platform approach cuts the time to find an effective treatment in half, decreases costs by a third or more, and is supported by our patients, the FDA, ALS clinicians, ALS foundations and scientists and our pharma colleagues.”

From more than two dozen international applications, Implicit’s IC14 immunotherapy was selected for inclusion in the initial five therapeutic concepts to be tested.

“The concept of treating this devastating disease with an immunotherapy is new and builds on an encouraging pilot study in Brisbane that Dr Rob Henderson ran last year with funding from Australia’s FightMND,” said Implicit Bioscience CEO, Garry Redlich.

“This visionary design from the Healey Center at Mass General for the next phase of testing will accelerate the ability of clinical researchers to identify and test the most promising new therapies for ALS.

“The molecular target of IC14, measured in a patients’ blood, has recently been identified by our colleagues at Houston Methodist as highly associated with rapid disease progression. This potentially makes IC14 a personalized treatment for people with the most aggressive forms of this relentless disease,” said Mr Redlich.

For more information

Implicit Bioscience:
Garry Redlich D.Univ.
Tel: +1 303 775 8210 during AU morning hours / US Pacific afternoon and evening hours.

Healey Center

About IC14 IC14 is a biologic monoclonal antibody drug that inactivates CD14 on immune cells and in circulation. CD14 is a master regulator of the immune response that is also a key organizer of responses by the brain’s most common immune cell to infection and injury. Hyper-activated CD14 results in damaging inflammation that kills healthy brain and other cells in diseases such as ALS. Implicit Bioscience holds owns all worldwide rights to IC14 and is developing it in the new field of immunoneurology for a range of neurodegenerative and neurotraumatic disease indications.

About Implicit Bioscience Ltd.
Implicit Bioscience is an unlisted public company based in Brisbane and Seattle. Founded in 2005 by a number of biopharma veterans including Prof. Peter Andrews AOand Prof. Ian Frazer AC FRS, the Company’s mission is to apply groundbreaking immunotherapy to treat serious and life-threatening diseases with few or no effective treatments.

About ALS
Amyotrophic lateral sclerosis is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently three FDA therapies approved specifically for treating ALS—riluzole, nuedexta and edaravone. 

About Platform Trials
A “platform trial” is a clinical trial in which multiple treatments are tested and evaluated simultaneously. New treatments are added to the platform as they become available, thereby decreasing the gap in time from identification of a rational therapy to testing. This model, already proven successful in the cancer field, will greatly accelerate therapy development by allowing investigators to test more drugs, increase patient access to trials, and reduce the cost by quickly and efficiently evaluating the effectiveness of multiple therapies. 

In addition, because different treatments are tested using a shared infrastructure and a common Master Protocol, data from participants in all placebo groups can be pooled resulting in high statistical power to see a treatment effect between each treatment and the pooled placebo group, increasing the participant’s individual chance of being on active treatments much higher than in traditional trials. 

“This design optimizes chances for people to receive active treatment, provides answers faster, and ensures we keep learning about the disease”, says Prof. Cudkowicz. 

The HEALEY ALS Platform Trial will include several promising biomarkers and novel outcome measures, such as measures of muscle strength and speech analysis and biofluid markers and will be an engine to further develop these exciting novel tools. “We are thrilled to be in a position to test several treatments for ALS and, at the same time, facilitate the development of biomarkers that can more quickly predict therapeutic success”, says Sabrina Paganoni, MD, a physician-scientist and faculty member at the Healey Center. “In this new paradigm, more patients will have access to promising therapies and the trial will provide important contributions to our knowledge about ALS.”

Industry partners from these first five companies are working closely with the Healey Center Trial Design Team, the Northeast ALS Consortium (NEALS) and Berry Consultants to tailor the arm of the Platform Trial to their experimental treatment with the goal of initiating participant enrollment beginning in early 2020. 54 NEALS trial-ready sites across the US were selected for participation in the HEALEY ALS Platform Trial.   

Partial financial support to initiate these first treatments is made possible thanks to the generosity of the Healey family and friends and the AMG Charitable Foundation along with partners at TackleALS. The Healey Center at Mass General is working closely with industry partners to finalize contracting in the coming weeks, while continuing to seek additional partnerships with foundations to sustain and expand the program over time.  Several other exciting therapies for people with ALS are in the pipeline and the Healey Center’s goal is to add new treatments to the platform each year until effective treatments are found.