Yumanity Therapeutics (NASDAQ: YMTX), a clinical-stage
biopharmaceutical company focused on the discovery and development
of innovative, disease-modifying therapies for neurodegenerative
diseases, today reported that its lead product candidate, YTX-7739,
in development for the treatment of Parkinson’s disease (PD),
demonstrated dose-dependent decreases in target fatty acids, which
can be interpreted as evidence of in vivo target engagement, in a
Phase 1a multiple ascending dose (MAD) clinical trial in healthy
volunteers. Inhibition of stearoyl-CoA desaturase (SCD) has been
shown to improve motor function and other disease markers in
transgenic mouse models of PD. YTX-7739 was also observed to be
generally well tolerated in the Phase 1a study.
YTX-7739 was discovered through Yumanity’s target
discovery and drug development platform that is designed to
identify disease-modifying therapeutics against newly identified
targets for neurodegenerative diseases. Preclinical work has shown
that SCD inhibition by YTX-7739, an orally available, brain
penetrant small molecule, can overcome the toxicity of pathogenic
α-synuclein, a known risk factor for PD, both in vitro and in
animal models. Further, the fatty acid desaturation index (FA-DI)
which measures the ratio of SCD’s product to its substrate has
demonstrated utility as a biomarker to gauge SCD inhibition in
animals treated with YTX-7739.
“We are very pleased that repeated dosing with YTX-7739 was
observed to be generally well tolerated in these healthy
volunteers,” said Brigitte Robertson, M.D., Chief Medical Officer
of Yumanity Therapeutics. “Additionally, the biomarker analysis
revealed decreased levels of the enzyme’s product relative to
levels of its substrate in plasma. This finding supports that
YTX-7739 inhibits SCD and achieved target engagement.
The pharmacology of YTX-7739 observed preclinically has
translated nicely to the clinical experience observed in this part
of the study. Most importantly, we know from preclinical work that
a response in the plasma is associated with response in the brain.
Furthermore, the potential to correlate changes in the plasma FA-DI
with drug exposures support the use of FA-DI as a biomarker to help
assess clinical response to YTX-7739 and potentially identify
patients most likely to benefit, for inclusion in later phase
trials. We look forward to the results of our ongoing Phase 1b
safety and biomarker study to assess the effect of chronic dosing
in patients with Parkinson’s disease. Topline results from the
Phase 1b part of the study are expected in mid-2021.”
This part of the Phase 1, placebo-controlled, randomized,
double-blind study, investigated the safety, tolerability,
pharmacokinetics, and pharmacodynamics of once daily oral
administration of two doses of YTX-7739 (15 mg and 25 mg) for 14 to
28 days in 16 healthy male and female volunteers. The study
included two cohorts of eight subjects each, randomized to
treatment or placebo in a 6:2 ratio. YTX-7739 was observed to be
generally well tolerated in this part of the study. There were no
serious adverse events and all treatment emergent adverse events
were either mild or moderate in severity. Plasma pharmacokinetics
support once daily dosing, and relevant drug concentrations were
measured in the cerebrospinal fluid. Target engagement was
achieved, as evidenced by dose dependent decreases in the plasma
FA-DI, within the range associated with restoration of motor
function in animal models of Parkinson’s disease.
Study data will be presented at Yumanity’s upcoming R&D Day
on May 17, 2021 and at a future medical conference.
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 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 enables the Company to rapidly screen for
potential disease-modifying therapies by overcoming the toxicity of
misfolded proteins associated with 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 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, including YTX-7739, 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 Yumanity Therapeutics’ most recent Annual or Quarterly
Report, 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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