Yumanity Therapeutics (NASDAQ: YMTX), a biopharmaceutical company
focused on the development of innovative, disease-modifying
therapies for neurodegenerative diseases today held a virtual
R&D Day. Management and David S. Russell, M.D., Ph.D., a guest
researcher in neurodegenerative disorders from Yale University
School of Medicine, discussed the cellular pathology underlying
Parkinson’s disease (PD) and related disorders, how Yumanity’s lead
clinical program, YTX-7739, has the potential to address a toxic
biochemical pathway associated with PD, and how Yumanity’s
discovery engine reveals additional novel biological targets with
the potential to change the course of other neurodegenerative
diseases.
“Yumanity has been a public company for about five months, and
in that time the Company has made significant strides in advancing
its pipeline,” said Richard Peters, M.D., Ph.D., President, Chief
Executive Officer and Director of Yumanity Therapeutics. “Not
only have we advanced our orally administered SCD inhibitor,
YTX-7739, in clinical development but we have also demonstrated
target engagement in humans at levels that reduced motor function
deficits in a PD mouse model. Our first data from the Phase 1b part
of our Phase 1 program in patients with PD are expected in the
middle of this year. We expect to initiate clinical development of
a second asset, YTX-9184 this year, honoring our commitment to
introduce one new clinical program per year.”
“We are also excited to announce plans to initiate a third
clinical program in early 2022 with a window of opportunity study
targeting glioblastoma multiforme. This decision has been informed
by compelling results of our SCD inhibitors in a preclinical animal
model of the disease. Importantly, we expect our three clinical
programs to achieve data read-outs within our expected current cash
runway, which is into the third quarter of 2022.”
The R&D Day focused primarily on an up-to-date review of the
clinical and pre-clinical data on YTX-7739.
- Phase 1 multiple ascending dose (MAD) study. This randomized,
double-blind, placebo-controlled study with oral once-daily dosing
of 15 mg or 25 mg for up to 28 days demonstrated that YTX-7739 was
generally well tolerated in healthy volunteers. The drug was
detected in the cerebrospinal fluid (CSF) at concentrations
consistent with all pre-clinical animal models. YTX-7739
demonstrated dose dependent target engagement. After 14 days, the
15 mg dose reduced the plasma target engagement biomarker by about
20% while the 25 mg dose reduced the index by about 40%. Reductions
in this range resulted in motor function improvement in animal
models of PD.
- Target engagement is measured by a validated biomarker called
the fatty acid desaturation index (FA-DI). YTX-7739 inhibits the
enzyme SCD, which catalyzes the conversion of two saturated fatty
acids to unsaturated fatty acids. Increased concentrations of
unsaturated fatty acids alter cell membrane structure, resulting in
alpha-synuclein aggregation, neuronal toxicity, and
neurodegeneration. Inhibition of SCD by YTX-7739 reduces the
concentration of unsaturated fatty acids, which in turn is
reflected in reductions of the FA-DI biomarker.
- Phase 1 single ascending dose (SAD) study in healthy volunteers
demonstrated that YTX-7739 is generally well tolerated. The drug
candidate demonstrated a half-life of 47 to 72 hours suggesting
once-daily dosing. YTX-7739 was also shown to cross the blood brain
barrier with a single, oral dose. Despite evaluating doses of up to
400 mg, which is 10-fold greater than the modeled therapeutic dose
expected to be evaluated in humans, no maximum tolerated dose was
reached.
- The ongoing Phase 1b portion of the
Phase 1 program is a randomized, double-blind, placebo-controlled
study enrolling 30 PD patients with mild-to-moderate symptoms who
are either treatment naïve or satisfactorily controlled with
L-dopa. Two doses of YTX-7739, 20 mg and a second dose to be
determined, will be dosed orally once daily for 28 days. Study
endpoints include tolerability, pharmacokinetics and target
engagement as measured by FA-DI. Several exploratory biomarkers
will also be evaluated. Initial results from the study are expected
to be announced in mid-2021.
“Today’s presentations highlight the progress Yumanity has made
with YTX-7739 and demonstrates our improved understanding of
neurodegenerative disease pathologies,” said Ajay Verma, Executive
Vice President, Head of R&D of Yumanity Therapeutics.
“Concurrently, we validated the potential of our unbiased drug
discovery engine’s ability to identify multiple novel targets for
drug development. With our Merck collaboration signed last year and
multiple novel targets against different diseases of the central
nervous system ready for development, we are extremely well
positioned to build a multi-asset neurology product pipeline in the
coming years.”
A replay of a webcast of the R&D Day is available for 60
days following the event. It can be accessed under "Events &
Presentations" in the Investor Relations section of the Company's
website at
https://www.yumanity.com/investor-relations/events-presentations/.
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). SCD
appears to play an important and previously unrecognized role in
mitigating neurotoxicity arising from the effects of pathogenic
alpha-synuclein protein aggregation and accumulation, which
ultimately results in the death of neurons 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
cellular and preclinical models. The company is assessing the
potential utility of YTX-7739 as a disease modifying therapy for
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.Lee
Rothlroth@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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