Yumanity Therapeutics (Nasdaq: YMTX) is a clinical-stage
biopharmaceutical company focused on the discovery and development
of innovative, disease-modifying therapies for neurodegenerative
disease. The Company today reported that its lead product
candidate, YTX-7739, in development for the treatment of
Parkinson’s disease, achieved its primary endpoints in a
randomized, placebo-controlled Phase 1b clinical trial in patients
with mild-to-moderate Parkinson’s disease.
YTX-7739 is an investigational drug with a
potentially first-in-class disease-modifying mechanism of action.
There are no currently available disease-modifying treatments for
Parkinson’s disease.
YTX-7739 was generally well tolerated and
demonstrated favorable pharmacokinetic/pharmacodynamic (PK/PD)
profiles and a safety profile with no serious adverse events.
YTX-7739 achieved the expected target engagement in the patients
studied and results were consistent with earlier studies in healthy
volunteers and preclinical models.
Notably, YTX-7739 was shown to inhibit its
primary target, stearoyl-CoA desaturase (SCD), an enzyme whose
inhibition has been closely linked to neuronal survival and
improved motor function in a Parkinson’s disease model. After 28
days of treatment, the 20 mg dose given once-daily reduced the
fatty acid desaturation index (FA-DI), a biomarker of SCD
inhibition, by approximately 20%-40%, the range expected to be
clinically relevant based on preclinical studies. Target engagement
in the cerebrospinal fluid suggested that YTX-7739 effectively
crossed the blood-brain barrier. Additionally, the PK/PD profile of
YTX-7739 was consistent with previous studies and informs dose
selection for future studies.
YTX-7739 was generally well tolerated with all
treatment emergent adverse events being mild to moderate in
severity. There were no serious adverse events. Moderate adverse
events (AEs) in the active treatment group consisted of 2 patients
with increased Parkinson’s symptoms, 2 patients with lower back
pain, 1 patient with headache, 1 patient with myalgia, 1 patient
with insomnia, 1 patient with ligament sprain, and 1 patient with
vaccination complication. One patient on placebo had moderate
worsening of tremors and Parkinsonism, which led to
discontinuation. AEs occurring at a higher percentage in 2 or more
patients administered YTX-7739 compared to placebo were procedural
pain, myalgia, dry eye, hyperbilirubinemia, hypesthesia, lower back
pain, and constipation. AEs occurring at a higher percentage with
placebo included orthostatic hypotension, headache, tremor, fatigue
and dizziness.
As expected, after only 28 days of dosing, there
were no statistically significant differences in clinical
assessments (UPDRS III, MoCA) or most exploratory biomarkers.
Quantitative electroencephalogram (qEEG) assessments of the effect
of YTX-7739 on brain activity were completed in a subset of 8
patients and demonstrated a statistically significant change
compared to baseline, suggestive of a potential improvement in
synaptic function. The company expects to further validate the role
of this diagnostic marker in future clinical studies.
“Today’s announcement provides yet another
strong validation of our proprietary approach to the development of
disease-modifying drugs for neurodegenerative diseases,” said
Richard Peters, M.D., Ph.D., President and CEO of Yumanity
Therapeutics. “Together with our ongoing collaboration with Merck
in amyotrophic lateral sclerosis and frontotemporal lobar dementia,
which was announced last year, Yumanity has demonstrated that it
continues to lead in identifying new targets and new drug
candidates that have the potential for disease modification in
these terrible diseases and remains one of the few select companies
with an asset to be advanced into Phase 2 clinical development for
the potential treatment of Parkinson’s disease.”
“Parkinson’s disease is a devastating
neurodegenerative disease for which treatments address only
temporarily symptoms without any impact on the inevitable disease
progression,” said Dr. Geert Jan Groeneveld, Chief Scientific and
Medical Officer at the Center for Human Drug Research in Leiden and
Professor of Clinical Neuropharmacology at the Leiden University
Medical Center. “To truly alter the course of the disease, we need
new insights into the underlying cellular pathology and novel
treatments that interfere with those processes. Recent advances
made on the role of lipids in the normal and pathological activity
of alpha synuclein have provided a new therapeutic avenue to pursue
in Parkinson’s with SCD inhibition emerging as a rational approach.
The results of this trial are a very encouraging outcome and
position YTX-7739 as potentially the first therapeutic candidate to
test the lipidopathy hypothesis of synuclein.”
The Phase 1b clinical trial was a randomized,
placebo-controlled, double-blind multi dose study to investigate
the safety, tolerability, pharmacokinetics and pharmacodynamics of
YTX-7739. Data were reported from 20 patients with mild-to-moderate
Parkinson’s disease. Patients received once-daily oral doses of
YTX-7739 (20 mg or placebo) for 28 days. More information on the
design of the trial can be found on the Netherlands Clinical trial
Registry, Trial NL9172.
“We are very encouraged by the results from this
Phase 1b trial, which are consistent with the data from the Phase
1a portion of the trial in healthy volunteers,” said Ajay Verma,
M.D., Ph.D., Executive Vice President of Research and Development
of Yumanity Therapeutics. “The favorable tolerability and PK/PD
profiles, absence of serious adverse events and evidence of target
engagement in patients support the continued development of
YTX-7739 as a potential treatment for Parkinson’s disease. In
addition, the observed qEEG changes, even after only 28 days of
treatment, increase our enthusiasm for the potential of YTX-7739.
We believe we are well-positioned to test the SCD hypothesis in a
Phase 2 randomized clinical trial in Parkinson’s disease, which we
expect to start in 2022. In the meantime, we anticipate presenting
the full data set at an upcoming medical conference.”
Management will host a conference call to
discuss the results of the Phase 1b clinical trial at 8:30 a.m.
EST, Wednesday, November 10. To participate in the conference call,
dial +1 (833) 562-0153 (U.S.) or +1 (661) 567-1235 (International),
confirmation code 1261348 shortly before 8:30 a.m. EST. The
webcast can be accessed
at https://www.yumanity.com/investor-relations/events-presentations/
and will be archived for at least 30 days. A replay of the call
will be available from 11:00 a.m. EST, November 10,
2021 to 7:30 p.m. ET, November 17, 2021. The replay
number is +1 (855) 859-2056 (U.S.) or +1 (404) 537-3406
(International), confirmation code 1261348.
About YTX-7739
YTX-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 preclinical cellular and animal models. The Company is
assessing the potential utility of YTX-7739 as a disease modifying
therapy for Parkinson’s disease.
About SCD
SCD is an enzyme that catalyzes fatty acid
desaturation, the products of which are incorporated into
phospholipids, triglycerides, or cholesterol esters. These classes
of lipid molecules regulate multiple diverse cellular properties
and processes, including membrane structure and function, vesicle
and organelle 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. Following the initial discovery of
SCD’s role in synucleinopathy by Yumanity’s unbiased discovery
engine, several prominent academic laboratories have independently
focused on SCD as a promising upstream target for mitigating
alpha-synuclein mediated neurodegeneration.
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, Lewy body dementia and
other neurodegenerative diseases.
About Yumanity Therapeutics
Yumanity 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 in 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 initiatives focused on multi-system
atrophy, Lewy body dementia, amyotrophic lateral sclerosis (ALS or
Lou Gehrig’s disease), frontotemporal lobar dementia, and
Alzheimer’s disease. For more information, please
visit www.yumanity.com.
Forward Looking Statements
This 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
YTX-7739 and the design, commencement, enrollment, and timing of
planned clinical trials, clinical trial results, product approvals
and regulatory pathways, the anticipated benefits of our drug
discovery platform, and statements regarding our financial and cash
position and expected cash runway. 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 and
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 the Company’s current expectations, estimates
and projections about its 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
the Company is unable to execute on its business strategy; the risk
that YTX-7739 will not have the safety or efficacy profile that the
Company anticipates; the risk that prior results, such as signals
of safety, activity or durability of effect, observed from
preclinical studies and clinical trials of YTX-7739 will not be
replicated or will not continue in ongoing or future studies or
trials; and the risk that the Company will be unable to obtain and
maintain regulatory approval for its product candidates. For a
discussion of these and other risks and uncertainties, and other
important factors, any of which could cause the Company’s actual
results to differ materially and adversely from those contained in
the forward-looking statements, see the section entitled “Risk
Factors” in the Company’s most recent Annual or Quarterly Report,
as well as discussions of potential risks, uncertainties, and other
important factors in the Company’s subsequent filings with
the Securities and Exchange Commission. The Company 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.Robert Flamm, Ph.D.
/ Harrison Wongrflamm@burnsmc.com / Hwong@burnsmc.com (212)
213-0006 ext. 364 / 316
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