Yumanity Therapeutics (NASDAQ: YMTX), a clinical-stage
biopharmaceutical company focused on the discovery and development
of innovative, disease-modifying therapies for neurodegenerative
diseases, today announced results of a study of its lead program,
YTX-7739, that demonstrate pharmacological, physiological and
behavioral pre-clinical proof of concept in a Parkinson’s disease
(PD) mouse model. This oral presentation and two posters will be
presented at the 15th Annual International Conference on
Alzheimer’s and Parkinson’s Diseases (AD/PD™ 2021) Virtual
Conference, March 9 to 14, 2021.
“Evidence suggests that α-synuclein pathology is
a strong risk factor for Parkinson’s disease,” said Dan Tardiff,
Ph.D., interim Head of Research and Scientific Co-founder at
Yumanity Therapeutics. “Our proprietary discovery platform led us
to the target stearoyl-CoA desaturase (SCD), which when inhibited
decreases the toxicity associated with pathogenic α-synuclein.
Inhibition of SCD, an enzyme involved in lipid metabolism, has been
shown to prevent α-synuclein pathology in multiple models,
including patient-derived neurons, in vitro. The results from the
current study demonstrate that YTX-7739 has a similar effect in a
mouse model of Parkinson’s disease-related pathology. These results
lend additional evidence to support our ongoing clinical program in
Parkinson’s disease patients.”
The research entitled, YTX-7739, A Clinical
Stage Stearoyl-CoA Desaturase Inhibitor for Parkinson’s Disease
Improves Behavioral and Pathological Features in an a-Synuclein
Mouse Model, conducted in collaboration with the laboratories of
Silke Nuber, Ph.D., and Dennis Selkoe, M.D., at Brigham &
Women’s Hospital, will be presented at 12:30 p.m. [CET] on March 13
(live discussion March 13 from 5:30 to 6 p.m. CET) by Dr. Tardiff.
The study evaluated YTX-7739 vs. control in the PD mouse model. The
model was used to evaluate the effect of the drug on motor function
as well as survival of neurons and associated biochemical features.
The effects of the drug on lipid metabolism were also examined. The
investigators found:
- YTX-7739 prevented motor function
deficits in the diseased mice after four months of YTX-7739 oral
dosing compared to placebo-treated mice.
- YTX-7739 concentrations in the
brain reached a level that engaged and inhibited SCD activity
consistent with in vitro studies. In addition, SCD inhibition by
YTX-7739, which can be measured by a quantitative biomarker,
resulted in a decrease in monounsaturated fatty acids in both
plasma and brain. Monounsaturated fatty acids may contribute to
α-synuclein pathology.
- Multiple biochemical measures of
α-synuclein pathology were significantly improved in mice with
YTX-7739 as compared to controls, including levels of pathological
α-synuclein. The reduction in levels of pathological α-synuclein
was also confirmed via histopathological analysis.
- Mice treated with YTX-7739
exhibited enhanced survival of dopaminergic neurons, the type of
neuron that selectively dies in the brains of patients with
Parkinson’s disease.
The results indicate that YTX-7739 actively
engages with its intended target, SCD, in the mouse brain. The
observed improvements in pathology and neuron survival, and the
resultant correction of motor dysfunction in mice provided added
evidence for SCD inhibition as a potential treatment approach for
Parkinson’s disease, and further support for the on-going
evaluation of YTX-7739 in humans.
Posters to be presented at AD/PD
2021
Non-Clinical Pharmacokinetic and
Pharmacodynamic Properties of YTX-7739, a Clinical Stage
Stearoyl-CoA Desaturase Inhibitor for Parkinson’s
DiseasePoster #: P499 / #1463Topic: Theme C:
α-Synucleinopathies / C2.a. Therapeutic Targets, Mechanisms for
Treatment: Αlpha-synucleinAuthors: D. Tardiff, M. Lucas, I. Wrona,
B. Chang, C. Chung, B. Le Bourdonnec, K. Rhodes, R.
ScannevinSummary: YTX-7739 is an inhibitor of SCD, an enzyme that
desaturates fatty acids. The objective of the study was to
characterize the pharmacokinetic properties of YTX-7739 in rats and
cynomolgus macaques and the corresponding pharmacodynamic changes
in fatty acid profiles. YTX-7739 was found to be well-tolerated and
brain penetrant in both species. It was a potent inhibitor of SCD
in vivo where it reduced the levels of unsaturated fatty acids in a
dose dependent manner.
A Three-Dimensional Patient-Derived
Cortical Neurosphere Model of Parkinson’s DiseasePoster #:
P497 / #942Topic: Theme C: α-Synucleinopathies / C2.a. Therapeutic
Targets, Mechanisms for Treatment: Αlpha-synucleinAuthors: W. Raja,
E. Neves, C. Burke, X. Jiang, P. Xu, V. Khurana, C. Chung, R.
ScannevinSummary: The objective of the study was to develop a
patient-derived induced pluripotent stem cell PD model.
Three-dimensional neurospheres were created and exhibited relevant
PD phenotypes that responded to SCD inhibitors. These neurospheres
have been used by Yumanity to evaluate the potential of small
molecule therapeutics.
About YTX-7739YTX-7739 is
Yumanity Therapeutics’ proprietary lead small molecule
investigational therapy designed to penetrate the blood-brain
barrier and inhibit the activity of a novel target, stearoyl-CoA
desaturase (SCD), that plays an important and previously
unrecognized role in modulating neurotoxicity arising from the
alpha-synuclein protein, a major driver of Parkinson’s disease and
related neurodegenerative disorders. Misfolding and aggregation of
alpha-synuclein triggers a cascade of events, ultimately resulting
in neurotoxicity and the subsequent impairment of movement and
cognition that afflicts patients living with this disease. Through
inhibition of SCD, YTX-7739 modulates an upstream process in the
alpha-synuclein pathological cascade and has been shown to rescue
or prevent toxicity in preclinical models. The company is assessing
the potential utility of YTX-7739 in Parkinson’s disease.
About SCDSCD is an enzyme that
catalyzes fatty acid desaturation, the products of which are
incorporated into phospholipids, triglycerides, or cholesterol
esters. These lipid-related molecules regulate multiple diverse
cellular properties and processes, including membrane structure and
function, vesicle trafficking, intracellular signaling and
inflammation. SCD expression is regulated by a transcription factor
known as SREBF1, which has been identified in human genome-wide
association studies as a risk factor for Parkinson’s disease. In
preclinical models, SCD inhibition appears to normalize the dynamic
interaction of pathological alpha-synuclein with membranes, which
improves neuronal function and reduces toxicity, leading to
enhanced neuronal survival. Alpha-synuclein-dependent disruption of
membrane-related biological pathways, such as vesicle trafficking,
is closely linked to the formation of Lewy body protein/membrane
aggregations, a hallmark pathological feature of Parkinson’s
disease.
About Yumanity
TherapeuticsYumanity Therapeutics is a clinical-stage
biopharmaceutical company dedicated to accelerating the revolution
in the treatment of neurodegenerative diseases through its
scientific foundation and drug discovery platform. The Company’s
most advanced product candidate, YTX-7739, is currently in Phase 1
clinical development for Parkinson’s disease. Yumanity’s drug
discovery platform is designed to enable the Company to rapidly
screen for potential disease-modifying therapies by overcoming
toxicity of misfolded proteins in neurogenerative diseases.
Yumanity’s pipeline consists of additional programs focused on Lewy
body dementia, multi- system atrophy, amyotrophic lateral sclerosis
(ALS or Lou Gehrig’s disease), frontotemporal lobar dementia
(FTLD), and Alzheimer’s disease. For more information, please visit
www.yumanity.com.
Forward-Looking StatementsThis
press release contains forward-looking statements, including
statements made pursuant to the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995. These statements
may be identified by words and phrases such as “aims,”
“anticipates,” “believes,” “could,” “designed to,” “estimates,”
“expects,” “forecasts,” “goal,” “intends,” “may,” “plans,”
“possible,” “potential,” “seeks,” “will,” and variations of these
words and phrases or similar expressions that are intended to
identify forward-looking statements. These forward-looking
statements include, without limitation, statements regarding the
potential therapeutic benefits of our prospective product
candidates and results of preclinical studies, including YTX-7739,
and the design, commencement, enrollment, and timing of ongoing or
planned clinical trials, clinical trial results, product approvals
and regulatory pathways, and the anticipated benefits of our drug
discovery platform. Any such statements in this press release that
are not statements of historical fact may be deemed to be
forward-looking statements. Results in preclinical or early-stage
clinical trials may not be indicative of results from later stage
or larger scale clinical trials and do not ensure regulatory
approval. You should not place undue reliance on these statements,
or the scientific data presented.
Any forward-looking statements in this press
release are based on Yumanity Therapeutics’ current expectations,
estimates and projections about our industry as well as
management’s current beliefs and expectations of future events only
as of today and are subject to a number of risks and uncertainties
that could cause actual results to differ materially and adversely
from those set forth in or implied by such forward-looking
statements. These risks and uncertainties include, but are not
limited to, the risk that any one or more of our product candidates
will not be successfully developed or commercialized, the risk of
cessation or delay of any ongoing or planned clinical trials of
Yumanity Therapeutics or our collaborators, the risk that Yumanity
Therapeutics may not successfully recruit or enroll a sufficient
number of patients for our clinical trials, the risk that Yumanity
Therapeutics may not realize the intended benefits of its drug
discovery platform, the risk that our product candidates will not
have the safety or efficacy profile that we anticipate, the risk
that prior results, such as signals of safety, activity or
durability of effect, observed from preclinical or clinical trials,
will not be replicated or will not continue in ongoing or future
studies or trials involving Yumanity Therapeutics’ product
candidates, the risk that we will be unable to obtain and maintain
regulatory approval for our product candidates, the risk that the
size and growth potential of the market for our product candidates
will not materialize as expected, risks associated with our
dependence on third-party suppliers and manufacturers, risks
regarding the accuracy of our estimates of expenses and future
revenue, risks relating to our capital requirements and needs for
additional financing, risks relating to clinical trial and business
interruptions resulting from the COVID-19 outbreak or similar
public health crises, including that such interruptions may
materially delay our enrollment and development timelines and/or
increase our development costs or that data collection efforts may
be impaired or otherwise impacted by such crises, and risks
relating to our ability to obtain and maintain intellectual
property protection for our product candidates. For a discussion of
these and other risks and uncertainties, and other important
factors, any of which could cause Yumanity Therapeutics’ actual
results to differ materially and adversely from those contained in
the forward-looking statements, see the section entitled “Risk
Factors” in the definitive proxy statement/prospectus/information
statement filed with the Securities and Exchange
Commission on November 12, 2020, as well as discussions
of potential risks, uncertainties, and other important factors in
Yumanity Therapeutics’ subsequent filings with the Securities
and Exchange Commission. Yumanity Therapeutics explicitly disclaims
any obligation to update any forward-looking statements except to
the extent required by law.
Investors:Burns McClellan, Inc.John
Grimaldijgrimaldi@burnsmc.com (212) 213-0006
Media:Burns McClellan, Inc.Ryo Imai / Robert
Flamm, Ph.D.rimai@burnsmc.com / rflamm@burnsmc.com (212)
213-0006
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