Study exemplifies how ADDF funding fuels
progress of innovative science guided by the biology of aging for
future of Alzheimer's treatment.
NEW
YORK, May 17, 2024 /PRNewswire/ -- A study
published today in Nature Medicine found that a novel
neuroprotective drug candidate, LM11A-31, was safe and
well-tolerated following dosing for 26 weeks in patients with mild
to moderate Alzheimer's disease. LM11A-31 also slowed the
progression of multiple spinal fluid and imaging biomarkers. These
encouraging results support LM11A-31 being studied in a larger
trial to further evaluate its safety and efficacy. The study, which
received funding by the Alzheimer's Drug Discovery Foundation
(ADDF), reflects ongoing progress as the field moves towards a
broader approach guided by the biology of aging, building the
arsenal of Alzheimer's treatments needed to complement existing
anti-amyloid therapies for combination treatment.
"Studies like this one are moving us closer to the day when we
could have a pill to help treat Alzheimer's," says
Howard Fillit, MD, Co-Founder and
Chief Science Officer of the ADDF. "A decade ago, it was
unimaginable to see a novel drug advance to this stage in the
pipeline. These results are indicative of the rapid evolution
of the field. Neuroprotective drugs represent the type of therapies
that will define the next generation of Alzheimer's treatments,
which will target the underlying biology of the disease in a
combination therapy and precision medicine approach, similar to
cancer care."
Phase 2a exploratory studies, such as this one, that meet their
primary endpoint are valuable for generating safety and
tolerability data in the target population and providing clinical
and biomarker data to design well-informed and rigorous phase
2b and 3 studies.
"We are greatly encouraged by the findings of this study and
look forward to uncovering how this drug, which has downstream
effects on several aging- and resilience-related pathways, will
impact the slowing of cognitive decline in the study's next phase,"
says Frank Longo, MD, PhD, Founder
of PharmatrophiX, George E. and Lucy Becker Professor of Medicine
and Neurology at Stanford University,
and inaugural Goodes Prize honoree. "Without ADDF funding, LM11A-31
would not have reached this milestone. My 25-year-long relationship
with the foundation began when this molecule was first discovered
in the lab and has catalyzed this drug through basic mechanism and
preclinical studies to the clinical phase, showing the importance
of advancing translational research that will pave the way for new
treatments and prevention strategies for the millions of patients
living with this disease."
LM11A-31 works by activating survival pathways and inhibiting
degenerative pathways of nerve cells, addressing several
Alzheimer's-related pathologies including resilience against
amyloid, reduction of tau pathology, and reduction of microglial
pathology. The compound is being developed by PharmatrophiX, a
clinical-stage biopharmaceutical company focused on the development
of disease-modifying small molecule drugs targeting
neurodegenerative disorders.
These findings are reflective of the broadening and diversifying
drug pipeline that looks to address the plethora of causes that
lead to Alzheimer's. Today, nearly 75% of drugs are exploring novel
targets like inflammation, metabolic disturbances, and vascular
dysfunction. With the advent of new, novel drugs, the next step is
to conduct combination therapy trials that explore combining
multiple drugs into one treatment regimen.
"It's harvest time for Alzheimer's research and the advancement
of novel, pleiotropic drugs, like LM11A-31, are the future,
showcasing our ability to develop therapies that have the potential
to target not one, but several underlying disease mechanisms. This
trial underscores the impact of the ADDF's investments, helping to
transform the treatment landscape by catalyzing cutting-edge
science," notes Dr. Fillit.
About The Alzheimer's Drug Discovery Foundation
(ADDF)
Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer's Drug Discovery
Foundation is dedicated to rapidly accelerating the discovery of
drugs to prevent, treat and cure Alzheimer's disease. The ADDF is
the only public charity solely focused on funding the development
of drugs for Alzheimer's, employing a venture philanthropy model to
support research in academia and the biotech industry. The ADDF's
leadership and contributions to the field have played a pivotal
role in bringing the first Alzheimer's PET scan
(Amyvid®) and blood test (PrecivityAD®) to
market, as well as fueling the current robust and diverse drug
pipeline. Through the generosity of its donors, the ADDF has
awarded more than $290 million to
fund over 750 Alzheimer's drug discovery programs, biomarker
programs and clinical trials in 20 countries. To learn more, please
visit: http://www.alzdiscovery.org/.
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SOURCE Alzheimer's Drug Discovery Foundation