Yumanity Therapeutics (NASDAQ: YMTX), a clinical-stage
biopharmaceutical company focused on the discovery and development
of innovative, disease-modifying therapies for neurodegenerative
diseases, today provided several updates on its lead clinical-stage
program, YTX-7739, in development for the treatment of Parkinson’s
Disease.
“Recent advances in our understanding of the
pathological processes underlying neurodegenerative diseases are
providing the opportunities for innovative companies such as
Yumanity to address this challenge with therapies that attack known
disease pathways,” said Robert Scannevin, Ph.D., Head of Discovery
at Yumanity Therapeutics. “Multiple lines of evidence indicate that
misfolded alpha-synuclein is a strong risk factor for Parkinson’s
disease. However, rather than targeting alpha-synuclein directly,
we discovered that inhibiting the enzyme stearoyl-CoA desaturase
(SCD) could overcome alpha-synuclein toxicity, suggesting its
potential as a therapeutic target.”
Brigitte Robertson, M.D., Chief Medical Officer
at Yumanity Therapeutics, added, “YTX-7739 is a novel small
molecule SCD inhibitor, which can be administered orally and was
shown to be brain penetrant in preclinical models. We have
completed the single ascending dose (SAD) study in healthy
volunteers, which provides early evidence of the drug candidate’s
safety and tolerability profile. We look forward to reporting on
the data from the ongoing Multiple Ascending dose (MAD) study in
healthy volunteers and the preliminary results of our first
clinical trial in patients with Parkinson’s disease, later this
year. We wish to also thank our investigators, their staff, study
volunteers and patients for their dedication in continuing this
important research through the challenges of the COVID-19
pandemic.”
Results from the single ascending dose
study in healthy volunteers: This was a Phase 1,
single-ascending dose study of YTX-7739, a novel SCD inhibitor
being developed for the treatment of Parkinson’s disease. Fifty-six
healthy volunteers (aged 19-39 years of age; 22 males; 34 females)
were administered single oral doses of YTX-7739, from 5 mg to 400
mg. Forty subjects participated in the placebo controlled,
randomized, double blind part of the study which included 7 cohorts
of 8 subjects each, randomized to treatment or placebo in a 6:2
ratio. Sixteen of these subjects also participated in 2 cohorts
where YTX-7739 was administered with food. In addition, 2 cohorts
of 8 subjects each (16 subjects) were conducted in an open label
fashion to further inform dose selection for the MAD study. There
were no safety concerns identified and YTX-7739 was found to be
well tolerated with most adverse events being mild or moderate in
severity. The half-life of YTX-7739 combined with a favorable
dose-proportional pharmacokinetic (PK) profile, in the fed state,
supports that low daily doses administered with food will sustain
the target range of exposure. Drug plasma concentrations in the
study exceeded levels of exposure estimated to be sufficient for
target engagement based on pharmacodynamic modeling. Consistent
with preclinical data, YTX-7739 also demonstrated clinically
relevant drug concentrations in the cerebral spinal fluid (CSF).
The results of this SAD study supported progression to the multiple
ascending dose study.
Completion of enrollment in the multiple
ascending dose study (MAD) in healthy volunteers: This is
a Phase 1, placebo-controlled, randomized, double-blind study,
investigating the safety, tolerability, and pharmacokinetics of
once daily oral administration of 2 doses of YTX-7739 (15 mg and 25
mg) for 14 to 28 days in 16 healthy male and female volunteers. The
study includes 2 cohorts of 8 subjects each, randomized to
treatment or placebo in a 6:2 ratio. The study is also exploring
plasma and CSF biomarker measures of pharmacodynamic activity. The
company expects to report the results of this MAD study by the end
of the current quarter. Detailed clinical data from these Phase 1
studies in healthy volunteers with YTX-7739 will be presented at a
future medical conference.
Dosing initiated in a Phase 1b study in
Parkinson’s disease patients: The company also announced
the start of a placebo controlled, randomized double blind, MAD
Phase 1b study of YTX-7739 in patients with Parkinson’s disease.
This study is expected to enroll 30 subjects and will collect
safety, tolerability, pharmacokinetic and pharmacodynamic
parameters including potential biomarkers of SCD activity and
target engagement in the CSF, plasma, and other fluids or tissues.
Preliminary results are expected by mid-year 2021.
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 dysregulation of movement and
cognition that afflicts patients living with these diseases.
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
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may be identified by words and phrases such as “aims,”
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identify forward-looking statements. These forward-looking
statements include, without limitation, statements regarding our
business strategy for and the potential therapeutic benefits of our
prospective product candidates, results of preclinical studies, 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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