– Biomarker results indicate GEM103’s ability
to regulate complement and complement factor H therapeutic approach
in geographic atrophy –
– GEM103 continues to be well-tolerated and
demonstrates a differentiated safety profile with no increased risk
of CNV and minimal inflammation –
– GEM103 program for GA advancing in
discussions with regulators to seek alignment on late-stage trial
design including every other month dosing –
Gemini Therapeutics, Inc. (Nasdaq:GMTX), a clinical stage
precision medicine company developing innovative treatments for
genetically defined age-related macular degeneration (AMD), today
announced initial data from its Phase 2a ReGAtta study of GEM103 as
of May 2021 in patients with geographic atrophy (GA) secondary to
dry AMD. ReGAtta is a dose escalation trial of GEM103, which is
intravitreally administered recombinant human complement factor H
(CFH), in dry AMD patients. The trial, which remains ongoing, is
designed to evaluate safety and tolerability, as well as measures
of intraocular pharmacokinetics (PK) and disease-relevant
biomarkers, to inform the late-stage development program.
“We are very excited these results demonstrate that GEM103 has
biological activity and support CFH’s mechanism of action to
regulate complement activity in patients with dry AMD. Critically,
GEM103 also continues to show a positive safety profile in the
setting of repeat dosing for up to six months,” said Samuel Barone,
M.D., Chief Medical Officer of Gemini Therapeutics. “The ReGAtta
study includes novel analysis of the biological pathways involved
in GA and we are encouraged by GEM103’s activity seen at this early
point and look forward to the additional analyses expected later
this year.”
ReGAtta was designed to evaluate repeat dosing of GEM103 and
assess its safety in an open-label study that enrolled 62 patients
with GA secondary to dry AMD. The first 36 patients enrolled
received monthly 250µg intravitreally administered doses of GEM103.
After evaluating the safety profile of repeated dosing over three
months, patients were dose escalated to 500µg and an additional 26
patients enrolled and received monthly 500µg doses. After
completing the first six months of dosing, each patient will have
the option to continue receiving GEM103 for up to an additional 12
months.
Patients enrolled in ReGAtta had a mean age of 78 and GA
secondary to dry AMD in the study eye with 63% of patients also
having GA in the fellow eye. Choroidal neovascularization (CNV) in
the study eye was an exclusion criterion, however 30% of patients
had a history of CNV in the fellow eye at baseline. Among the
baseline characteristics in the study eye, mean best corrected
visual acuity (BCVA) score, as measured by Early Treatment Diabetic
Retinopathy Study (ETDRS) letters, at enrollment was 61.5 (with a
range of 14-86). Average GA size was 8.1 mm2. The GA was foveal in
68% of patients and multifocal in 63% of patients. Loss of function
variants in the CFH gene were confirmed in 55 of the 62 patients
enrolled. A total of 43 patients carry a homozygous AMD risk
variation at the 402 locus of the CFH gene and six patients carry a
rare heterozygous variant in CFH.
Summarized results observed to date in the ongoing Phase 2a
ReGAtta study include the following:
Demonstration of Biological Activity,
Complement Regulation and Dosing Frequency
Intraocular measures of CFH and biomarkers demonstrated GEM103’s
biological activity to regulate complement and support every other
month dosing.
- Both 250µg and 500µg doses of GEM103 resulted in sustained,
elevated CFH levels from the first evaluated time point of one
month (at least 6-fold and 12-fold above baseline, respectively)
that continued to increase dose dependently.
- Changes in biomarkers of complement activation indicated that
GEM103 has the ability to regulate the complement system and
overall disease-related inflammation, with an ~40% reduction in Ba,
~20% reduction in C3a and an increase in CFB, consistent across all
genotypes.
GEM103 Continued to be Well-tolerated with
a Differentiated Safety Profile
- GEM103 was well-tolerated with no serious adverse events
related to study drug and no serious ocular adverse events as of
May. There were no early discontinuations due to the study
drug.
- Over 390 injections of GEM103 were administered, which equates
to a total of 28 patient-years of exposure. Treatment was
well-tolerated with only 16 patients (26%) experiencing adverse
events in the study eye. Among these events, 12 patients’ adverse
events were related to the intravitreal administration procedure
with conjunctival hemorrhage as the most common reported adverse
event.
- Active monitoring was conducted for retina-specific safety
including inflammation and CNV. Dilated retina exams were conducted
at every study visit and retinal imaging was performed every three
months. An independent reading center reviewed such data.
- There were no endophthalmitis and no vitritis, retinal
vasculitis or vascular occlusive events. Mild iritis (< 1+ cell) was observed in the study eye in
three patients (4.8%); all cases resolved with either observation
only or topical therapy. One case was reported as related to
GEM103, and all patients continued on study without disruption of
GEM103 dosing schedule.
- One case of CNV in a study eye presented as a macular
hemorrhage at month 1 in the 500µg cohort was determined by the
investigator not to be related to GEM103 or the intravitreal
administration procedure. The patient is receiving anti-VEGF
treatment and has continued on study.
- There were no cases of CNV confirmed in the study eye by the
independent reading center’s analysis of the retinal imaging.
- Visual acuity remained stable (±5 EDTRS letters) throughout the
study.
- GA progression at three months and six months in the study eye
compared to fellow eyes that also meet the inclusion criteria was
statistically indistinguishable.
“This is an important milestone for the GEM103 program that
supports the potential advantages of a well-tolerated safety
profile without an increased risk for CNV and every other month
dosing,” said Jason Meyenburg, Chief Executive Officer of Gemini
Therapeutics. “We continue to evaluate the data coming out of
ReGAtta while seeking alignment with regulators on GEM103’s
late-stage trial designs.”
Information on Gemini Therapeutics, including GEM103 and ReGAtta
initial data, are included in its corporate presentation which is
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. 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.
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
investors and the public using our website
(www.geminitherapeutics.com), the investor relations website
(https://investors.geminitherapeutics.com/), and on social media
(Twitter and LinkedIn), including but not limited to investor
presentations and investor fact sheets, U.S. Securities and
Exchange Commission filings, press releases, public conference
calls and webcasts. The information that Gemini posts on these
channels and websites could be deemed to be material information.
As a result, Gemini encourages investors, the media, and others
interested in Gemini to review the information that is posted on
these channels, including the investor relations website, on a
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
operations when needed. Forward-looking statements include
statements relating to our management team’s expectations, hopes,
beliefs, intentions or strategies regarding the future. In
addition, any statements that refer to projections, forecasts or
other characterizations of future events or circumstances,
including any underlying assumptions, are forward-looking
statements. The words “anticipate,” “believe,” “contemplate,”
“continue,” “could,” “estimate,” “expect,” “intends,” “may,”
“might,” “plan,” “possible,” “potential,” “predict,” “project,”
“should,” “will,” “would” and similar expressions may identify
forward-looking statements, but the absence of these words does not
mean that a statement is not forward-looking. These forward-looking
statements are based on current expectations and beliefs concerning
future developments and their potential effects. There can be no
assurance that future developments affecting us will be those that
we have anticipated. These forward-looking statements involve a
number of risks, uncertainties (some of which are beyond our
control) or other assumptions that may cause actual results or
performance to be materially different from those expressed or
implied by these forward-looking statements. These risks and
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
one or more of these risks or uncertainties materialize, or should
any of our assumptions prove incorrect, actual results may vary in
material respects from those projected in these forward-looking
statements. Some of these risks and uncertainties may in the future
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
forward-looking statements, whether as a result of new information,
future events or otherwise, except as may be required under
applicable securities laws.
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version on businesswire.com: https://www.businesswire.com/news/home/20210622005988/en/
Investors: 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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