Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage
biotechnology company focused on Alzheimer’s disease, today
announced that The Journal of Prevention of Alzheimer’s Disease
(JPAD), a peer-reviewed journal for the research community,
published results from the Company’s Phase 2a study demonstrating
that its lead drug candidate, PTI-125, reduced biomarkers of
disease in Alzheimer’s patients.
Nadav Friedmann, MD, PhD, Chief Medical Officer
at Cassava Sciences commented, “We are pleased to share these study
results in JPAD. This publication provides clinical insights into
how our lead drug candidate, PTI-125, could make an important
difference for patients living with Alzheimer’s disease. In this
study, PTI-125 reduced multiple biomarkers of Alzheimer’s disease,
including neurodegeneration and neuroinflammation. To our
knowledge, no other drug has shown such effects in Alzheimer’s
patients.”
Published results from this study demonstrate
that biomarkers of Alzheimer’s disease pathology (P-tau, total tau
and Aβ42), neurodegeneration (NfL and neurogranin) and
neuroinflammation (YKL-40, IL-6, IL-1β and TNFα) improved
significantly after 28 days of treatment with PTI-125. Biomarker
reductions were at least p< 0.001 by paired t-test. Biomarkers
effects were seen in all patients in both cerebrospinal fluid (CSF)
and plasma. PTI-125 was safe and well tolerated, with no observable
drug-related adverse events.
Lindsay H. Burns, PhD, VP Neuroscience at
Cassava Sciences, added, “This publication supports PTI-125 as a
new and potentially disease-modifying drug treatment for
Alzheimer’s disease. Significant improvements observed across
multiple biomarkers imply a slower rate of neurodegeneration or a
suppression of disease processes. These treatment effects are
consistent with the drug’s mechanism of action and over ten years
of basic research and preclinical data.”
Key results published from the Phase 2a study
include:
- Total tau (T-tau) decreased 20% (p<0.001)
- Phosphorylated tau (P-tau) decreased 34% (p<0.0001)
- Neurofilament light chain (NfL), a marker for
neurodegeneration, decreased 22% (p<0.0001)
- Neurogranin, a marker for cognitive decline, decreased 32%
(p<0.0001)
- Neuroinflammatory marker YKL-40, an indicator of microglial
activation, decreased 9% (p<0.0001)
- Proinflammatory Interleukin 6 (IL-6) decreased 14%
(p<0.0001)
- Proinflammatory Interleukin 1 beta (IL-1β) decreased 11%
(p<0.0001)
- Proinflammatory Tumor Necrosis Factor alpha (TNFα) decreased 5%
(p<0.001)
- The ratio of CSF P-tau to Aβ42, a widely accepted biochemical
value of Alzheimer’s disease, improved in all patients
(p<0.001)
Although cognition and function were not
assessed in this small Phase 2a study, independent research has
shown that high levels of CSF biomarkers of P-tau and total
tau/Aβ42 ratio correlate with worse performance on a wide range of
memory and attention tests. Conversely, lowering biomarkers of
disease may benefit patients.
“We are now conducting a confirmatory Phase 2b
study of PTI-125 in Alzheimer’s disease,” said Remi Barbier,
President and CEO. “If positive, results of that study may be a
major inflection point for us as a company and for patients,
physicians and caregivers in the Alzheimer’s community. We look
forward to sharing results of our on-going Phase 2b study
approximately mid-2020.”
Results of On-going Phase 2b Study
Expected mid-2020Sixty-four (64) patients with
mild-to-moderate Alzheimer’s disease are enrolled in a randomized,
placebo-controlled, confirmatory Phase 2b study to assess the
safety, tolerability and biomarkers effects of PTI-125. More
information for this study is available on-line at
ClinicalTrials.gov: https://clinicaltrials.gov/ct2/show/NCT04079803
About the Published Phase 2a
Study Phase 2a was a first-in-patient, open-label,
multi-center, safety, pharmacokinetic and biomarker study of
PTI-125 in the U.S. Thirteen patients with mild-to-moderate
Alzheimer’s disease, age 50-85, received 100 mg oral PTI-125 twice
daily for 28 days. A diagnosis of Alzheimer’s disease was confirmed
with Mini-Mental State Examination (MMSE) ≥ 16 and ≤ 24 and a CSF
T-tau/Aβ42 ratio ≥ 0.30. Safety was assessed by ECGs, clinical
labs, adverse event monitoring and physical examinations. CSF was
drawn from patients before dosing started and again after 28
continuous days of dosing with PTI-125. CSF samples were then
analyzed for biomarkers of Alzheimer’s pathology (T-tau, P-tau,
Aβ42); neurodegeneration (NfL, neurogranin); and neuroinflammation
(YKL-40, IL-6, IL-1β and TNFα). A consulting biostatistician
conducted an independent analysis of the data set.
Reference“PTI-125 Reduces
Biomarkers of Alzheimer’s Disease In Patients”, The
Journal of Prevention of Alzheimer’s Disease (2020) (DOI:
10.14283); H.-Y. Wang1,2, Z. Pei1, K.-C. Lee1, E.
Lopez-Brignoni3, B. Nikolov3, C.A. Crowley4, M.R. Marsman4, R.
Barbier4, N. Friedmann4, L.H. Burns4The publication is available
on-line: http://link.springer.com/article/10.14283/jpad.2020.6
About PTI-125The target
of PTI-125 is an altered form of filamin A (FLNA), a scaffolding
protein. Altered FLNA in the brain disrupts the normal function of
neurons, leading to Alzheimer’s pathology, neurodegeneration and
neuroinflammation. Lead drug candidate PTI-125 is a proprietary
small molecule that restores the normal shape and function of FLNA
in the brain. This action improves the function of certain
receptors in the brain, slows neurodegeneration and exerts powerful
anti-neuroinflammatory effects. The underlying science for PTI-125
is published in peer-reviewed scientific journals, including
Journal of Neuroscience, Neurobiology of Aging, and Journal of
Biological Chemistry.
Cassava Sciences is also developing an
investigational diagnostic, called PTI-125Dx, to detect Alzheimer’s
disease with a simple blood test.
About Alzheimer's
Disease Alzheimer’s disease is a progressive brain
disorder that destroys memory and thinking skills. Currently, there
are no drug therapies to halt Alzheimer’s disease, much less
reverse its course. In the U.S. alone, approximately 5.8 million
people are currently living with Alzheimer’s disease, and
approximately 487,000 people age 65 or older developed Alzheimer’s
in 2019.5 The number of people living with Alzheimer’s disease is
expected to grow dramatically in the years ahead, which may also
result in a growing social and economic burden.6
About Cassava Sciences,
Inc.The mission of Cassava Sciences is to detect and treat
neurodegenerative diseases, such as Alzheimer’s disease. Over
the past 10 years, Cassava Sciences has combined state-of-the-art
technology with new insights in neurobiology to develop novel
solutions for Alzheimer’s disease. Cassava Sciences owns worldwide
development and commercial rights to its research programs in
Alzheimer’s disease, and related technology, without royalty
obligations to any third-party.
For More Information Contact:
Kirsten Thomas, SVP The Ruth Groupkthomas@TheRuthGroup.com (508)
280-6592
Acknowledgment and
DisclaimerResearch reported in this press release
is supported by the National Institute of Aging of the NIH under
award AG060878. The content of this press release is solely the
responsibility of Cassava Sciences and does not necessarily
represent any official views of NIH.
Cautionary Note Regarding
Forward-Looking Statements: This press release contains
“forward-looking statements” for purposes of the Private Securities
Litigation Reform Act of 1995 (the Act). Cassava Sciences
claims the protection of the Safe Harbor for forward-looking
statements contained in the Act. All statements other than
statements of historical fact contained in this press release
including, but not limited to, statements regarding the status of
clinical studies with PTI-125; the interpretation of results of
clinical studies, including target engagement and potential health
benefits, if any, of changes in levels of biomarkers; verbal
commentaries made by Cassava Sciences’ employees; and other
potential benefits, if any, of the Company’s product candidates for
Alzheimer’s disease, are forward-looking statements. Such
statements are based largely on the Company’s current expectations
and projections about future events. Such statements speak only as
of the date of this press release and are subject to a number of
risks, uncertainties and assumptions, including, but not limited
to, those risks relating to the ability to conduct or complete
clinical trials on expected timelines, to demonstrate the
specificity, safety, efficacy or potential health benefits of our
product candidates and including those described in the section
entitled “Risk Factors” in Cassava Sciences’ Annual Report on Form
10-K for the year ended December 31, 2018 and future reports to be
filed with the SEC. In light of these risks, uncertainties and
assumptions, the forward-looking statements and events discussed in
this press release are inherently uncertain and may not occur, and
actual results could differ materially and adversely from those
anticipated or implied in the forward-looking statements.
Accordingly, you should not rely upon forward-looking statements as
predictions of future events. Except as required by law, the
Company disclaims any intention or responsibility for updating or
revising any forward-looking statements contained in this press
release.
For further information regarding these and
other risks related to our business, investors should consult our
filings with the SEC, which are available on the SEC's website at
www.sec.gov.
1 Department of Molecular, Cellular and Biomedical Sciences,
City University of NY School of Medicine
2 Department of Biology and Neuroscience, Graduate School of the
City University of New York
3 IMIC, Inc., Palmetto Bay, FL
4 Cassava Sciences, Inc., Austin, TX
5, 6 Source: Alzheimer’s Association. 2019 Alzheimer’s Disease
Facts and Figures. Available online at:
https://www.alz.org/media/documents/alzheimers-facts-and-figures-2019-r.pdf
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