Gemini Therapeutics Completes Enrollment in Phase 2a Trial of GEM103 in Dry Age-Related Macular Degeneration (AMD) in Patients with High-Risk Genetic Variants

Rhea-AI Summary

Gemini Therapeutics, Inc. (Nasdaq: GMTX) has completed enrollment in its Phase 2a ReGAtta study, a trial assessing GEM103 for dry age-related macular degeneration (AMD) patients with CFH loss-of-function gene variants. This study aims to evaluate safety, tolerability, pharmacokinetics, and disease-relevant biomarkers. Approximately 40% of dry AMD patients carry CFH variants, and GEM103 is designed to specifically target these genetic markers. The company expects to release topline data in the first half of 2021, marking a significant step towards potential new treatment options for patients with geographic atrophy.

Positive

• Completion of enrollment in Phase 2a ReGAtta trial for GEM103, indicating progress in clinical development.
• GEM103 targets genetically-defined patients, addressing a significant unmet medical need in dry AMD.
• FDA granted Fast Track Designation for GEM103, which may expedite the development process.

Negative

• None.

Gemini Therapeutics, Inc. (Nasdaq: GMTX), a clinical stage precision medicine company developing innovative treatments for genetically defined age-related macular degeneration (AMD) and linked ocular disorders, today announced the completion of enrollment in its Phase 2a “ReGAtta” study. ReGAtta is a dose escalation trial of GEM103, a recombinant human complement factor H (CFH), in dry AMD patients with CFH loss-of-function gene variants. The trial 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 pleased to have completed enrollment in our Phase 2a ReGAtta trial of GEM103. Our strategy is to provide an intravitreal recombinant human factor H in patients with gene variants resulting in loss of function of their own CFH. There is no approved therapy for the treatment of geographic atrophy (GA) and we know that approximately 40% of dry AMD patients have variants in the CFH gene linked to progression from early AMD to late stage GA,” said Marc E. Uknis, M.D., F.A.C.S., Chief Medical Officer of Gemini Therapeutics. “We believe that GEM103 offers hope for patients with dry AMD as it precisely targets the underlying genetics of the disease. We look forward to sharing topline data from the trial in the first half of 2021.”

“This is an important achievement for Gemini in our efforts to approach complement-mediated diseases in a novel manner, by restoring regulation of complement system. We want to thank the patients, the opthalmology community of investigators and the Gemini team for their commitment to this important mission,” said Jason Meyenburg, Chief Executive Officer of Gemini Therapeutics. “We believe that our work in biology and genetics, coupled with the novel mechanism of action of GEM103, can enable us to realize the potential of precision medicine in the treatment of AMD.”

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. The study was designed to enroll approximately 60 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, 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 native complement modulator, full-length recombinant complement factor H (rCFH) protein. When delivered by intravitreal injection, GEM103 has the potential to address unmet medical need in genetically-defined subsets of AMD patients by circumventing dysfunctional CFH loss-of-function variants and slowing the progression of their retinal 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 modulator, 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 the 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. The company has generated a rich pipeline including recombinant proteins, gene therapies, and monoclonal antibodies.

For more information, visit www.geminitherapeutics.com.

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, including our estimates regarding when data will be reported from ongoing clinical trials and the timing to commence future clinical trials, the potential attributes and benefits of our product candidates, including GEM103, our ability to obtain and maintain regulatory approval for our product candidates 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 the final proxy/prospectus for our recently completed business combination, and those that are 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 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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FAQ

What is the purpose of the Phase 2a ReGAtta study for GEM103?

The Phase 2a ReGAtta study evaluates the safety, tolerability, and pharmacokinetics of GEM103 in dry AMD patients with CFH gene variants.

When will topline data from the ReGAtta trial be released?

Topline data from the ReGAtta trial is expected to be shared in the first half of 2021.

How many patients are enrolled in the ReGAtta study?

Approximately 60 patients with CFH loss-of-function gene variants have been enrolled in the ReGAtta study.

What is GEM103 and its significance in treating AMD?

GEM103 is a recombinant human complement factor H designed to treat genetically-defined subsets of dry AMD patients, specifically those with CFH gene variants.

What designation has the FDA granted to GEM103?

The FDA has granted Fast Track Designation to GEM103 for the treatment of dry AMD in patients with CFH loss-of-function gene variants.