The Muscular Dystrophy Association has awarded an MDA Venture Philanthropy (MVP) grant totaling $96,360 to Lexington, Mass.-based Izumi Biosciences, Inc., to fund early-stage development of IZ10023, a type of drug called a “pharmacokinetic (PK) enhancer,” for use in people with ALS who are taking riluzole. (Riluzole, marketed under the brand name Rilutek, was developed based on MDA-supported research on the neurotransmitter glutamate, and is the only drug approved by the U.S. Food and Drug Administration to treat ALS.)
MVP is MDA’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular disorders. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics.
“MDA is excited to enable development of a drug that could improve the effectiveness and safety of riluzole, the only drug currently approved for ALS,” said MDA Scientific Program Officer Amanda Haidet-Phillips. “The work funded through the MVP program will help not only to push this drug combination toward the clinic but also to provide insight into who among ALS patients may benefit from treatment with the drug combination.”
IZ10023 may eliminate a key clinical bottleneck
IZ10023 targets and blocks two proteins in the brain and spinal cord that work to protect the central nervous system (CNS) by acting as pumps that remove foreign or toxic substances. In mouse studies it’s been shown that these pumps remove drugs such as riluzole, reducing levels of the drug in the CNS and thereby limiting its effectiveness.
“Blockade of these pumps maintains effective levels of riluzole in the diseased tissues and leads to improved outcomes in animal models of ALS and other brain diseases where multi-drug resistance is a key clinical bottleneck,” President and CEO of Izumi Biosciences Antonius Bunt explained. He will serve as the principal investigator on the project.
Confirmation that the pumps’ activity is increased in most ALS patients and demonstration that sufficient blood levels of IZ10023 can be achieved to block their activity are important milestones that must be achieved before moving IZ10023 into clinical trials in patients with ALS, Bunt noted. If the work is successful, Izumi Biosciences intends to open an Investigational New Drug Application and move into clinical trials — possibly as early as 2019.
Building on the work of others
In addition to MDA support for development of IZ10023, Izumi recently received a nearly $1.5 million therapeutic development award from the Department of Defense.
“Our hope,” Haidet-Phillips said, “is that MDA’s funding will synergize with the recent award from the Department of Defense, accelerating Izumi’s drug toward the clinic for use in ALS patients.”
“This MDA grant is important for moving our IZ10023 platform technology forward, with potential benefits for not only people with ALS but for people with other neuromuscular diseases as well,” Bunt said. “We are delighted with the recognition and tangible support from MDA at this early stage of product development.”
MDA is committed to ALS research
MDA has funded more than $156 million in ALS research since 1950, and including this most recent award, is currently funding 44 ALS grants with a total funding commitment of more than $11 million.
The new grant was approved by MDA’s Board of Directors after careful deliberations and analysis by MDA’s Venture Philanthropy Advisory Committee. This year, MDA is funding 150 research projects around the world.