Gemini Therapeutics Announces FDA Fast Track Designation Granted for GEM103 for the Treatment of Dry Age-Related Macular Degeneration (AMD) in Patients With Complement Factor H (CFH) Loss of Function Gene Variants

Jan. 7, 2021 12:00 UTC



CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Gemini Therapeutics, a clinical stage precision medicine company developing innovative treatments for genetically defined age-related macular degeneration (AMD), today announced that GEM103, the Company’s investigational treatment for dry AMD, has been granted Fast Track designation by the United States Food and Drug Administration (FDA). Gemini is evaluating GEM103, a recombinant, human complement factor H (CFH), for its ability to address the multiple AMD pathobiologies in genetically-defined subsets of AMD patients caused by CFH loss-of-function variants and slow the progression of their disease. CFH loss-of-function variants are present in approximately 40% of patients with AMD. The ongoing Phase 2a ReGAtta study is evaluating GEM103 in patients with geographic atrophy (GA) secondary to dry AMD.

“We are pleased that the FDA has granted Fast Track designation to GEM103, which has the potential to be an important therapeutic option for patients with dry AMD and CFH loss-of- function gene variants,” said Jason Meyenburg, CEO of Gemini Therapeutics. “The FDA’s recognition of the existing unmet need in this patient population and this decision underscores the potential of GEM103 and its unique mechanism of action to treat this large population of AMD patients.”

Fast Track is an FDA designation intended to facilitate development and expedite review of investigational therapies to address unmet medical needs in the treatment of conditions for which non-clinical or clinical data has demonstrated the potential of the therapy to address this medical need. Benefits of this designation include more frequent engagements with the FDA to discuss the drug’s clinical development plan, eligibility for Accelerated Approval and Priority Review, and a Rolling Review, in which Gemini has the opportunity to submit completed sections of a Biologics Licensing Application (BLA) for review by the FDA, rather than waiting to complete the regulatory submission before it can be submitted for review.

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.

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. The company has generated a rich pipeline including recombinant proteins, gene therapies, and monoclonal antibodies.

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Forward-Looking Statements

This press release contains forward-looking statements that are based on beliefs and assumptions and on information currently available. In some cases, you can identify forward-looking statements by the following words: “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we caution you that these statements are based on a combination of facts and factors currently known by us and our projections of the future, about which we cannot be certain. Forward-looking statements in this press release include, but are not limited to, statements regarding the proposed business combination, including the timing and structure of the business combination, the proceeds of the business combination, the initial market capitalization of the Combined Company and the benefits of the business combination, as well as statements about the potential attributes and benefits of Gemini’s product candidates and the format and timing of Gemini’s product development activities and clinical trials. We cannot assure you that the forward-looking statements in this press release will prove to be accurate. These forward-looking statements are subject to a number of significant risks and uncertainties that could cause actual results to differ materially from expected results, including, among others, the ability to complete the business combination due to the failure to obtain approval from FS Development Corp.’s shareholders or satisfy other closing conditions in the Merger Agreement, the occurrence of any event that could give rise to the termination of the Merger Agreement, the ability to recognize the anticipated benefits of the business combination, the outcome of any legal proceedings that may be instituted against FS Development Corp. or Gemini following announcement of the proposed business combination and related transactions, the impact of COVID-19 on Gemini’s business and/or the ability of the parties to complete the business combination, the ability to obtain or maintain the listing of FS Development Corp.’s common stock on Nasdaq following the proposed business combination, costs related to the proposed business combination, changes in applicable laws or regulations, the possibility that FS Development Corp. or Gemini may be adversely affected by other economic, business, and/or competitive factors, and other risks and uncertainties, including those to be included under the header “Risk Factors” in the registration statement on Form S-4 to be filed by FS Development Corp. with the SEC and those included under the header “Risk Factors” in the final prospectus of FS Development Corp. related to its initial public offering. Most of these factors are outside of FS Development Corp.’s and Gemini’s control and are difficult to predict. Furthermore, if the forward-looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified time frame, or at all. The forward-looking statements in this press release represent our views as of the date of this press release. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.


Gemini Investor Contact:
Argot Partners
Sherri Spear

Gemini Media Contact:
Argot Partners
Joshua R. Mansbach


Source: Gemini Therapeutics

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