- Studies show mechanism of action and
biodistribution of GEM103 and support continued development as a
potential treatment for AMD
Gemini Therapeutics, Inc. (Nasdaq: GMTX), a clinical stage
precision medicine company developing innovative treatments for
genetically defined age-related macular degeneration (AMD), today
announced one oral presentation and the presentation of three
posters at the 13th International Conference on Complement
Therapeutics held in Ioannina, Greece from September 8-13,
2021.
“The results presented today underscore the potential of GEM103
as the next class of complement therapeutic for the treatment of
AMD by regulating complement. These results to date add to our
understanding of GEM103’s mechanism of action, points of
intervention in the pathway, duration and the ability of complement
factor H to restore regulation of complement activity in the eye,”
said Walter Strapps, Ph.D., Chief Scientific Officer of Gemini
Therapeutics. “Additionally, these data contribute to the growing
body of evidence that gives us confidence in this potential novel
intravitreal treatment and the importance of the restoration of
complement regulation, rather than indiscriminate inhibition, as a
therapeutic approach.”
Gemini is currently conducting the ReGAtta study, an ongoing
Phase 2a open-label, single-arm study of GEM103 in
genetically-defined patients with geographic atrophy (GA) secondary
to dry AMD. ReGAtta is designed to investigate the safety and
tolerability, PK, and evidence of complement regulation through
ocular biomarkers following repeat intravitreal administration of
GEM103. Based on the previously announced initial results of the
ReGAtta study, Gemini plans to advance GEM103 into late-stage
studies that will be designed to assess efficacy and safety of
GEM103 in the treatment of GA.
The oral presentation was given by Dr. Robyn Biggs, Associate
Director – Research on September 10, 2021 at 9:35 am (GMT+3) and
was titled “Mechanism of Action of Complement Factor H as a
Therapeutic in AMD”. The presentation covered a study designed
to understand the non-canonical complement pathway mechanisms of
action (MOA) as it relates to the RPE cell health and homeostasis,
and GEM103’s demonstration of several advantages as a potential
therapeutic for the treatment of AMD. Notably, the study results
show that GEM103 localized to “self cell” surfaces, including the
RPE, using endogenous mechanisms and remained bound on the cell
surface for an extended duration. GEM103 also inhibited
inappropriate complement activation selectively on host cells by
inhibiting deposition of C3-convertase while leading to a reduction
in inflammation markers C3a and Ba levels.
Posters presented include the following:
“Recombinant Complement Factor H (GEM103) Ocular
Biodistribution and Activity Following Intravitreal Administration
in Cynomolgus Monkeys” Prior to initiating its ongoing Phase 2a
clinical program of GEM103, researchers studied the biodistribution
and half-life of GEM103 in the ocular tissue of non-human primates
following a single IVT dose. Results demonstrate that GEM103
rapidly biodistributed to key tissues of interest including the
retina, choroid and retinal pigment epithelium (RPE), and was
retained on the RPE cell surface for an extended duration and
remains functionally active in the eye following
IVT-administration. Additionally, the pharmacokinetic profile for
GEM103 was consistent across retinal tissues, with 24-hour Cmax
observed in the vitreous humor, retina, RPE, choroid and aqueous
humor post IVT dose.
“Complement Factor H (CFH) Potentiating Antibody (PoAb)
Enhances Cell Surface FH Localization and Activity and Maintains
Fluid-phase Regulation in vivo” In-vitro assays demonstrate the
ability of a novel CFH PoAb to promote CFH localization to cell
surfaces and thereby enhance CFH complement regulatory function at
the activation site. Subsequent treatment of the PoAb in a
preclinical renal disease model showed similar behavior in vivo
leading to reduction in the pathologic C3 deposits on kidney while
maintaining fluid phase regulation and endogenous complement driven
encapsulated bacteria clearance mechanisms.
“AMD Associated Variants in Complement Factor I (CFI) Hinder
CFI Expression, Function or Both” In a preclinical study
evaluating the AMD risk-associated CFI protein variants for their
effect on CFI expression and function as compared to a non-risk
form in a panel of in vitro assays, risk variants were observed to
have insufficient functional activity, reduced expression level, or
both. Overall, the level of CFI functional deficiency correlated
with AMD disease risk for the variants studied.
Information on Gemini Therapeutics, including GEM103 and initial
ReGAtta data, and posters and presentations made by the company at
the 13th International Conference on Complement Therapeutics are
available on Gemini Therapeutics’ website under the Investors &
Media section: Events and Presentations.
About the Phase 2a ReGAtta Study
The ongoing Phase 2a, multi-center, open-label, multiple
ascending dose study of GEM103 in genetically-defined patients with
GA secondary to dry AMD is designed to investigate safety and
tolerability, PK, exploratory ocular biomarkers, and measures of
retinal anatomy and function and not assess GA efficacy of GEM103.
GEM103 is delivered monthly by an intravitreal injection and PK and
biomarkers of complement regulation are determined from aqueous
humor sampling. To date, the study has enrolled 62 patients with
gene variants that have been linked to the progression of dry AMD
from early to late-stage. The study’s design allowed for imbalances
in GA baseline characteristics and does not inform GA efficacy and
does not allow comparisons with uncontrolled fellow eye with
similar imbalances.
About GEM103
Gemini’s lead program, GEM103, is a pioneering precision
medicine approach, targeting trial enrichment with genetically
defined patients. GEM103 targets a genetically defined subset of
age-related macular degeneration (AMD) patients with complement
dysregulation. Of the 15 million dry AMD patients in the United
States, approximately 40% (or six million) have variants in the
complement factor H (CFH) gene. Such loss of function variants are
associated with increased dry AMD disease risk. GEM103 is believed
to be the first ever recombinant complement regulator and is a
full-length and human, recombinant complement factor H (rCFH)
protein. When delivered by intravitreal injection, we believe
GEM103 has the potential to address unmet medical need in
genetically defined AMD patients by circumventing the complement
dysfunction resulting from CFH loss of function variants and
slowing the progression of their retina disease. The U.S. Food and
Drug Administration (FDA) granted Fast Track Designation for GEM103
for the treatment of dry AMD in patients with CFH loss of function
gene variants.
About Dry Age-Related Macular Degeneration (AMD)
Age-related macular degeneration (AMD) is a progressive retinal
disease affecting millions of older adults, and the leading cause
of irreversible blindness in the western world. Symptoms, which
include blurry vision, loss of night vision and loss of central
vision, make activities of daily living such as reading, driving
and even recognizing faces progressively more difficult.
Third-party reports indicate there are approximately 16 million
patients with AMD in the United States alone. Dry AMD, which
results from an interaction of environmental and genetic risk
factors, represents about 90% of that population (or about 15
million) in the US compared to about 1.4 million with wet AMD.
Genetic risk of developing dry AMD is significant, with
approximately 70% attributable risk of advanced disease to
heritability, while aging and smoking confer the strongest
non-genetic risk. CFH risk variants occur in approximately 40% of
patients with dry AMD and these patients have a significantly
increased risk of developing the disease as well as progression
from intermediate AMD to GA. The complement system, of which CFH is
a regulator, is dysregulated in patients with these risk variants,
and results in amplification of aberrant inflammatory responses in
the eye. Over time, this dysregulation leads to damage to the
macular region of the retina.
About Gemini Therapeutics
Gemini Therapeutics is a clinical stage precision medicine
company developing novel therapeutic compounds to treat genetically
defined age-related macular degeneration (AMD). Gemini’s lead
candidate, GEM103, is a recombinant form of human complement factor
H protein (CFH) and is designed to address both complement
hyperactivity and restore retinal health in patients with AMD.
GEM103 is currently in a Phase 2a trial in dry AMD patients with a
CFH risk variant and a Phase 1/2a study in patients with
neovascular age-related macular degeneration with or at risk for
macular atrophy. Gemini has generated a rich pipeline including
recombinant proteins, gene therapies, and monoclonal antibodies and
is advancing a potentiating antibody for CFH, GEM307, into clinical
development for treatment of systemic diseases.
For more information, visit www.geminitherapeutics.com.
Availability of Other Information About Gemini
Therapeutics
Investors and others should note that we communicate with our
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As a result, Gemini encourages investors, the media, and others
interested in Gemini to review the information that is posted on
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regular basis. This list of channels may be updated from time to
time on Gemini’s investor relations website and may include
additional social media channels. The contents of Gemini’s website
or these channels, or any other website that may be accessed from
its website or these channels, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933, as
amended.
Gemini’s Forward-Looking Statements
Certain statements in this press release and the information
incorporated herein by reference may constitute “forward-looking
statements” for purposes of the federal securities laws. Our
forward-looking statements include, but are not limited to,
statements regarding our or our management team’s expectations,
hopes, beliefs, intentions or strategies regarding the future,
including those relating to the success, cost and timing of our
product development activities and clinical trials, whether such
data, when final, will be consistent with interim reported data,
the timing to commence future clinical trials, the potential
attributes and benefits of our product candidates, including
GEM103, the reliability of the interim or final results of studies
relating to safety and possible adverse effects resulting from the
administration of our product candidates, our ability to obtain and
maintain regulatory approval for our product candidates, our
projected cash runway and our ability to obtain funding for our
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uncertainties include, but are not limited to, those factors
described under the heading “Risk Factors” in Gemini’s most recent
Annual Report on Form 10-K filed with the SEC, as well as
discussions of potential risks, uncertainties, and other important
factors included in any of our future filings with the SEC. Should
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any of our assumptions prove incorrect, actual results may vary in
material respects from those projected in these forward-looking
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be amplified by the ongoing COVID-19 pandemic and there may be
additional risks that we consider immaterial, or which are unknown.
It is not possible to predict or identify all such risks. Our
forward-looking statements only speak as of the date they are made,
and we do not undertake any obligation to update or revise any
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version on businesswire.com: https://www.businesswire.com/news/home/20210910005013/en/
Investor:
Argot Partners Sherri Spear 212-600-1902
gemini@argotpartners.com
Media:
Argot Partners Joshua R. Mansbach 212-600-1902
gemini@argotpartners.com
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