A Multicenter, Multiple-Dose Study in Neovascular Age-related Macular Degeneration (nAMD) to Evaluate the Safety, Tolerability, Pharmacodynamics, Immunogenicity, and Clinical Effect of Repeat Intravitreal (IVT) Injections of GEM103 as an Adjunct to Standard of Care Aflibercept Therapy

This study is designed to investigate the safety and tolerability of GEM103 IVT injection + standard of care vs. sham + standard of care.

Start Date29 Dec 2020

Sponsor / Collaborator

Gemini Therapeutics, Inc.

NCT04643886

/ TerminatedPhase 2

A Multicenter, Open-label, Multiple Dose Study in Patients With Geographic Atrophy Secondary to Dry Age-related Macular Degeneration to Evaluate the Safety, Tolerability, Pharmacodynamics, and Immunogenicity of Repeat Intravitreal Injections of GEM103

This study is designed to investigate the safety, PK/PD, biomarker and early clinical effects of repeat GEM103 IVT injections.

Start Date30 Jul 2020

Sponsor / Collaborator

Gemini Therapeutics, Inc.

NCT04246866

/ CompletedPhase 1

A Phase 1, Multicenter, Open-label, Single-dose, Dose-escalation Study in Patients With Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD) to Evaluate the Safety, Tolerability, Pharmacodynamics, and Immunogenicity of Intravitreal Injections of GEM103

The study is designed to identify the maximum tolerated dose (MTD) for intravitreal (IVT) administration of GEM103 in subjects with geographic atrophy (GA) secondary to dry AMD. Safety and tolerability of a single dose of GEM103 will be assessed based on the occurrence of dose-limiting toxicities (DLTs). Each subject will be followed for safety, pharmacokinetic (PK), clinical, and biomarker evaluations. Three escalating dose cohorts are planned.

Start Date19 Dec 2019

Sponsor / Collaborator

Gemini Therapeutics, Inc.

100 Clinical Results associated with GEM-103

100 Translational Medicine associated with GEM-103

100 Patents (Medical) associated with GEM-103

Literatures (Medical) associated with GEM-103

28 Oct 2024·Medical review (Berlin, Germany)

Complement factor H in molecular regulation of angiogenesis

Review

Author: Li, Jiang ; Wang, Kaili ; Starodubtseva, Maria N. ; Nadyrov, Eldar ; Liu, Ju ; Kapron, Carolyn M. ; Hoh, Josephine

Abstract:

Angiogenesis, the process of formation of new capillaries from existing blood vessels, is required for multiple physiological and pathological processes. Complement factor H (CFH) is a plasma protein that inhibits the alternative pathway of the complement system. Loss of CFH enhances the alternative pathway and increases complement activation fragments with pro-angiogenic capacity, including complement 3a, complement 5a, and membrane attack complex. CFH protein contains binding sites for C-reactive protein, malondialdehyde, and endothelial heparan sulfates. Dysfunction of CFH prevents its interaction with these molecules and initiates pro-angiogenic events. Mutations in the CFH gene have been found in patients with age-related macular degeneration characterized by choroidal neovascularization. The Cfh-deficient mice show an increase in angiogenesis, which is decreased by administration of recombinant CFH protein. In this review, we summarize the molecular mechanisms of the anti-angiogenic effects of CFH and the regulatory mechanisms of CFH expression. The therapeutic potential of recombinant CFH protein in angiogenesis-related diseases has also been discussed.

03 Jul 2022·Current eye researchQ4 · MEDICINE

A Novel Full-Length Recombinant Human Complement Factor H (CFH; GEM103) for the Treatment of Age-Related Macular Degeneration Shows Similar In Vitro Functional Activity to Native CFH

Q4 · MEDICINE

Article

Author: Makou, Elisavet ; Lauder, Scott ; Herbert, Andrew P. ; Biggs, Robyn M. ; Barlow, Paul N. ; Katti, Suresh K.

PURPOSE:

GEM103 is a recombinantly produced full-length version of the human complement factor H (CFH) under clinical investigation for treatment of age-related macular degeneration (AMD) in individuals carrying an AMD risk-associated genetic variant of CFH. This study aimed to investigate the complement pathway-related functions of GEM103 in comparison with those of native human CFH.

METHODS:

Key biological activities of GEM103 and human serum-derived CFH (sdCFH) were compared using four independent functional assays. Assays of C3b binding and C3 convertase decay-accelerating activity (DAA) were performed by surface plasmon resonance (SPR). Cofactor activity (CA) was measured using 8-anilinonaphthalene-1-sulfonic acid as a fluorescent probe of C3b integrity. The abilities of GEM103 and sdCFH to protect sheep erythrocytes from hemolysis by CFH-depleted normal human serum were assessed colorimetrically.

RESULTS:

In multiple SPR-based assays of C3b binding and DAA, the performance of GEM103 was consistently comparable to that of sdCFH across a range of matching concentrations. The EC₅₀ ± SD in the fluorescence-based fluid-phase CA assay was 0.21 ± 0.06 µM for GEM103 compared to 0.20 ± 0.09 µM for sdCFH. In hemolysis assays, the EC₅₀ value of 0.33 ± 0.16 µM for GEM103 versus 0.46 ± 0.06 µM for sdCFH were not significantly different (p = 0.81).

CONCLUSIONS:

GEM103, a recombinant CFH developed by Gemini Therapeutics, shows activity profiles comparable to sdCFH in all complement-related assays employed in this study, suggesting that GEM103 is equivalent to the native glycoprotein in terms of its in vitro functional activity. These results support further study of GEM103 as a potential therapy for AMD.

01 Jun 2022·Ophthalmology science

A Phase I, Single Ascending Dose Study of GEM103 (Recombinant Human Complement Factor H) in Patients with Geographic Atrophy

Article

Author: Bakall, Benjamin ; Marcus, Dennis M ; Barone, Samuel B ; Pieramici, Dante ; Jamal, Karim N ; Couvillion, Stephen S ; Patki, Kiran C ; Dhoot, Dilsher S ; Boyer, David S ; Maturi, Raj K ; Bridges, William Z ; Aziz, Aamir A ; Khanani, Arshad M ; Bagheri, Nika ; Holekamp, Nancy M

Purpose:

To establish the safety, tolerability, pharmacokinetics, and pharmacodynamics of an intravitreal injection of recombinant human complement factor H (CFH), GEM103, in individuals with genetically defined age-related macular degeneration (AMD) and geographic atrophy (GA).

Design:

Phase I single ascending-dose, open-label clinical trial (ClinicalTrials.gov identifier, NCT04246866).

Participants:

Twelve individuals 50 years of age or older with a confirmed diagnosis of foveal GA in the study eye.

Methods:

Participants were assigned to the increasing dose cohorts and received 1 50-μl intravitreal injection of GEM103 at doses of 50 μg/eye, 100 μg/eye, 250 μg/eye, or 500 μg/eye; dose escalation was dependent on the occurrence of dose-limiting toxicities.

Main Outcome Measures:

Safety assessments included ocular and systemic adverse events (AEs), ocular examinations, clinical laboratory and vital signs, and serum antidrug antibody levels. Biomarkers, measured in the aqueous humor (AH), included CFH and complement activation biomarkers factor Ba and complement component 3a.

Results:

No dose-limiting toxicities were reported, enabling escalation to the maximum study dose. No anti-GEM103 antidrug antibodies were detected during the study. Four participants experienced AEs; these were nonserious, mild or moderate in severity, and unrelated to GEM103. The AEs in 2 of these participants were related to the intravitreal injection procedure. No clinically significant ophthalmic changes and no ocular inflammation were observed. Visual acuity was maintained and stable throughout the 8-week follow-up period. No choroidal neovascularization occurred. CFH levels increased in a dose-dependent manner after GEM103 administration with supraphysiological levels observed at week 1; levels were more than baseline for 8 weeks or more in all participants receiving single doses of 100 μg or more. Complement activation biomarkers were reduced 7 days after dose administration.

Conclusions:

A single intravitreal administration of GEM103 (up to 500 μg/eye) was well tolerated in individuals with GA. Of the few mild or moderate AEs reported, none were determined to be related to GEM103. No intraocular inflammation or choroidal neovascularization developed. CFH levels in AH were increased and stable for 8 weeks, with pharmacodynamic data suggesting that GEM103 restored complement regulation. These results support further development in a repeat-dose trial in patients with GA with AMD.

News (Medical) associated with GEM-103

10 Aug 2022

·www.biopharmadive.com

Gemini’s fall gives Disc a way around biotech’s IPO slump

Dive Brief:Disc Medicine, a privately held startup specializing in drugs for blood disorders, announced Wednesday it will go public through a reverse merger with struggling biotechnology company Gemini Therapeutics.The new company will use Discs name, be run by its existing management team and advance a portfolio of medicines led by a candidate thats newly in Phase 2 testing. Disc shareholders will own 72% of the company, while existing Gemini stockholders will hold the remaining 28%.In a challenging year for biotechs planning to go public, the reverse merger gives Disc an alternative route to Wall Street, while a concurrent round of financing hands it access to new funds.Dive Insight:Current market conditions are preventing many private biotech companies from going public, but where there's a will, there's a way. The reverse merger will give Watertown, Mass.-based Disc an infusion of much-needed cash while allowing the troubled Gemini to capitalize on the value of its public listing.By the time the companies close the deal in the last quarter of 2022, there will be approximately $92 million left in Gemini's coffers, said Jonathan Yu, Discs chief business officer.Combined with roughly $54 million in new financing from investors including OrbiMed, Atlas Venture and 5 AM Ventures, plus whats left of a $90 million round from 2021, Disc will have $175 million available by the end of the year, enough to keep the company afloat until 2025.Disc CEO John Quisel said the company decided against waiting for the right time to file for an initial public offering once the offer from Gemini was on the table.We can now control our future and ensure that our molecules are able to complete their clinical course, Quisel said.Disc Medicines portfolio includes bitopertin, an experimental drug licensed from Roche that turned up poor results against psychiatric disorders like schizophrenia, but showed potential for treating certain rare genetic blood disorders. Bitopertin is now in Phase 2 clinical testing for erythropoietic porphyria, a potentially fatal inherited condition, with Disc announcing the trials initiation on Wednesday.It's also working on an antibody drug acquired from AbbVie and designed to treat some forms of anemia. One Phase 1b/2 trial evaluating the drug in myelofibrosis patients with anemia is already underway. Disc plans to start a second study in patients with chronic kidney disease later this year.Funding those clinical trials requires a lot of capital, and thats where the funds from Gemini come in.Gemini went public five years after its launch, via an October 2020 merger with a special purpose acquisition company created by investment firm Foresite Capital. Shares began trading in February 2021. The company was working on GEM103, a candidate to treat age-related macular degeneration, among other drug prospects.But Gemini fell on hard times during the downturn. In October, it cut 20% of its workforce and announced it would focus exclusively on late-stage development of GEM103, ending its early drug research.That trial never began, however. A strategic review was launched in January and a month later, Gemini's board of directors appointed a new CEO. The company then let go of 80% of its remaining employees and terminated its work on GEM103.The new board will include eight directors from Disc and one from Gemini.Over the past few years, we've learned that there's a lot of uncertainty in the world, Quisel said. This transaction presenting itself now is a great opportunity for us. '

AntibodyIPOAcquisition

13 Jun 2022

·www.prnewswire.com

The Age-related macular degeneration market growth is expected to boost owing to the expected launch of therapies, increasing prevalence of Age-related macular degeneration, development in the field of gene therapy, patient-friendly dosage regimes, and several other factors during the forecast period. LAS VEGAS, June 13, 2022 /PRNewswire/ -- DelveInsight's Age-related Macular Degeneration Market Insights report includes a comprehensive understanding of current treatment practices, Age-related macular degeneration emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM (the United States, the EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. Key Takeaways from the Age-related Macular Degeneration Market Report As per DelveInsight analysis, the Age-related macular degeneration market size in the 7MM was approximately USD 9.8 billion in 2021. As per DelveInsight estimation, the Dry AMD market size was approximately USD 1,377 million, and the Wet AMD market size was approximately USD 8,463 million, which is further expected to increase by 2032. As per the estimates, the total Age-related macular degeneration prevalent cases in the 7MM was approximately 51.7 million in 2021. Leading Age-related macular degeneration companies Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc., Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics, and others are developing novel Age-related Macular Degeneration drugs which can be available in the Age-related Macular Degeneration market in the upcoming years. The Wet Age-related macular degeneration therapies in the pipeline include OPT-302, KSI-301, RGX-314, AKST4290, GB-102 (Sunitinib Malate), RBM-007, Lytenava (Bevacizumab-vikg), UBX1325, PAN-90806, CLS-AX (Axitinib), and others. The Dry Age-related Macular Degeneration therapies in the pipeline include Danicopan (ALXN2040), EG-301, RG6147 (RO7171009), ALK-001, Elamipretide, OpRegen, CPCB-RPE1, Luminate (Risuteganib), ANX007, NGM621, LNP023 (Iptacopan), IONIS-FB-LRx, Pegcetacoplan, Zimura (Avacincaptad Pegol), GT005, RPESC-RPE-4W, GEM103, and others. The expected launch of therapies and the increasing prevalence will lead to Age-related macular degeneration market growth during the forecast period. Discover more about therapies set to grab major age-related macular Degeneration market share @ Age-related Macular Degeneration Market Landscape Age-related Macular Degeneration Overview Age-related macular degeneration (AMD) is a leading cause of severe, irreversible blindness in developed countries around the world, with a significant impact on the quality of life of the affected people. With the current expensive standard of care, the increase in Age-related macular degeneration life expectancy brings an increase in the number of reported cases, putting major stress on health care systems. Age-related macular degeneration (AMD) can also be characterized as either dry Age-related macular degeneration (atrophic or non-neovascular) or wet Age-related macular degeneration (exudative or neovascular). The most common Age-related macular degeneration symptoms include blurry or fuzzy vision, loss of central vision, difficulty in recognizing familiar faces, and others. Visual acuity test, pupil dilation, fluorescein angiography, Amsler grid, and others are the following tests used for Age-related macular degeneration diagnosis. Age-related Macular Degeneration Epidemiology Segmentation As per DelveInsight, there were approx 51.7 million Age-related macular degeneration prevalent cases in the 7MM in 2021. Among the total diagnosed cases of Age-related macular degeneration, the highest cases were observed for dry Age-related macular degeneration in 2021. The Age-related macular degeneration market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Total Prevalent Cases of Age-related Macular Degeneration Total Diagnosed Prevalent Cases of Age-related Macular Degeneration Type-specific Diagnosed Prevalent Cases of AMD Total Age-specific Cases of Wet AMD, and Dry AMD Total Diagnosed Dry AMD Cases by Stages Total Geographic Atrophy Cases Associated With Dry AMD by Visual Impairment Download the report to understand which factors are driving Age-related macular degeneration epidemiology trends @ Age-related Macular Degeneration Epidemiological Insights Wet Age-related Macular Degeneration Treatment Market Photodynamic therapy, which employed an intravenous drug (injected into a vein) and laser to seal off bleeding blood vessels, was the best available Age-related macular degeneration treatment fifteen years ago. However, improvements in medical research have revealed VEGF as a critical pathophysiological factor in the development of neovascular AMD, with an important involvement in angiogenesis, vascular permeability, and inflammatory response. Anti-VEGF intravitreal injections have provided a new therapeutic window in the wet AMD treatment, effectively stopping the pathophysiological process of AMD and restoring retinal shape and function. Anti-VEGF therapies such as ranibizumab, aflibercept (Eylea), brolucizumab (Beovu), and faricimab (Vabysmo) are intravitreal injections given for wet AMD treatment. Though existing anti-VEGF therapies have transformed the way wet AMD is treated and greatly improved the lives of many patients, they will face stiff competition from new biosimilars and off-label drugs when their patents expire in the coming years. Furthermore, current anti-VEGF therapies have several unmet needs. Dry Age-related Macular Degeneration Treatment Market Currently, there is no approved dry AMD treatment, and there is a significant unmet need for a therapy to decrease its progression. Dry AMD's pathogenesis is poorly known. On the other hand, failure to achieve the primary outcomes may be due to the advanced stage of the disease rather than a lack of action on appropriate targets. Several pharmaceutical companies are currently working on developing novel therapies for dry AMD treatment. The therapies in the dry AMD pipeline include Pegcetacoplan (Apellis Pharmaceuticals), Zimura (Iveric bio), RO7171009; RG6147 (Genentech), IONIS-FB-LRx (Ionis Pharmaceuticals/ Roche), Luminate (Allegro Ophthalmics), NGM621 (NGM Biopharmaceuticals), Elamipretide (Stealth BioTherapeutics), CPCB-RPE1 (Regenerative Patch Technologies), OpRegen (CellCure Neurosciences/Lineage Cell Therapeutics/ Roche), GT005 (Gyroscope Therapeutics), and others. These therapies come with different types of mechanisms of action, including C3 inhibitor, C5 inhibitor, Integrin Inhibitors, Anti-C1q antibody, Anti-C3 antibody, Factor D inhibitor, HtrA1, ASO Complement Factor B, and others. Two C3 inhibitors, NGM621 (NGM Biopharmaceuticals) and pegcetacoplan (Apellis Pharmaceuticals), and one C5 inhibitor, Zimura (Iveric Bio), are showing promise as prospective treatments to slow the course of geographic atrophy. To know more about Age-related macular degeneration treatment visit @ Age-related Macular Degeneration Treatment Guidelines Wet Age-related Macular Degeneration Pipeline Therapies and Key Companies OPT-302: Opthea Limited KSI-301: Kodiak Sciences Inc. RGX-314: REGENXBIO AKST4290: Alkahest Inc GB-102 (Sunitinib Malate): Graybug Vision RBM-007: Ribomic USA Inc Lytenava (Bevacizumab-vikg): Outlook Therapeutics, Inc. UBX1325: Unity Biotechnology, Inc PAN-90806: PanOptica, Inc. CLS-AX (Axitinib): Clearside Biomedical Others Dry Age-related Macular Degeneration Pipeline Therapies and Key Companies Danicopan (ALXN2040): Alexion Pharmaceuticals and AstraZeneca EG-301: Evergreen Therapeutics RG6147 (RO7171009): Roche (Genentech) ALK-001: Alkeus Pharmaceuticals Elamipretide: Stealth BioTherapeutics OpRegen: CellCure Neurosciences CPCB-RPE1: Regenerative Patch Technologies Luminate (Risuteganib): Allegro Ophthalmics ANX007: Annexon Biosciences NGM621: NGM Biopharmaceuticals LNP023 (Iptacopan): Novartis IONIS-FB-LRx: Ionis Pharmaceuticals Pegcetacoplan: Apellis Pharmaceuticals Zimura (Avacincaptad Pegol): Iveric Bio GT005: Gyroscope Therapeutics and Novartis RPESC-RPE-4W: Luxa Biotechnology GEM103: Gemini Therapeutics Others Learn more about the dry AMD treatments in clinical trials @ New Treatment for Dry AMD Wet Age-related Macular Degeneration Market Dynamics The rising prevalence of wet AMD and the growing geriatric population are two significant factors driving the growth of the wet AMD market. Moreover, the wet AMD market dynamics are changing as extensive research and development have helped understand the histopathology and pathophysiology of wet AMD. Development in the field of gene therapy and a robust pipeline are also propelling the wet AMD market growth. However, some factors restrain the growth of the wet AMD market. Patients do not contact a physician until they have intermediate to severe AMD symptoms, making timely diagnosis extremely difficult. Moreover, the high cost of therapy, with gene therapy costing over USD 400,000, presents a significant barrier to treatment availability. In addition, the competitive treatment landscape and emerging biosimilars may also limit the growth of the wet AMD market. Dry Age-related Macular Degeneration Market Dynamics The dry AMD market is still untapped. The enormous and growing patient pool with no approved or adequate therapies provides companies with attractive prospects; the estimated potential is similar to wet AMD. Moreover, the dry AMD pipeline includes peptide derivatives, antibodies, antisense oligonucleotides, stem cell treatment, and gene therapy that function on various mechanisms such as C1q, C3, and C5 inhibitors, cell replacements, HtrA Serine Peptidase 1 inhibitor, and others. The launch of these therapies will boost the dry AMD market growth. Furthermore, the increasing prevalence of dry AMD is also driving the dry AMD market forward. However, limited knowledge of pathophysiology, disease severity, and consequences affect the development of efficacious treatment for dry AMD. Moreover, emerging gene therapies and stem cell therapies for dry AMD may face hurdles in access and reimbursement due to their anticipated high cost and will restrain the growth of the dry AMD market. Scope of the Age-related Macular Degeneration Market Report Study Period: 2019–2032 Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan] Key Age-related Macular Degeneration Companies: Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc., Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics, and others Key Wet Age-related Macular Degeneration Therapies: OPT-302, KSI-301, RGX-314, AKST4290, GB-102 (Sunitinib Malate), RBM-007, Lytenava (Bevacizumab-vikg), UBX1325, PAN-90806, CLS-AX (Axitinib), and others Key Dry Age-related Macular Degeneration Therapies: Danicopan (ALXN2040), EG-301, RG6147 (RO7171009), ALK-001, Elamipretide, OpRegen, CPCB-RPE1, Luminate (Risuteganib), ANX007, NGM621, LNP023 (Iptacopan), IONIS-FB-LRx, Pegcetacoplan, Zimura (Avacincaptad Pegol), GT005, RPESC-RPE-4W, GEM103, and others Therapeutic Assessment: Age-related Macular Degeneration current marketed and emerging therapies Age-related Macular Degeneration Market Dynamics: Age-related Macular Degeneration market drivers and barriers Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies Unmet Needs, KOL's views, Analyst's views, Age-related Macular Degeneration Market Access and Reimbursement Discover more about Age-related macular degeneration drugs in development @ Age-related Macular Degeneration Clinical Trials Table of Contents Get in touch with our Business executive @ Healthcare Due Diligence Services Related Reports Age-related Macular Degeneration Epidemiology Age-related Macular Degeneration Epidemiology Forecast to 2032 report delivers an in-depth understanding of the disease, historical, and forecasted Age-related Macular Degeneration epidemiology in the 7MM. Age-related Macular Degeneration Pipeline Age-related Macular Degeneration Pipeline Insight, 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Age-related Macular Degeneration companies including Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc. , among others. Wet Age-related Macular Degeneration Market Wet Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wet Age-related Macular Degeneration companies including Alkahest Inc, Graybug Vision, Clearside Biomedical, among others. Dry Age-related Macular Degeneration Market Dry Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast-2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Dry Age-related Macular Degeneration companies including Alkeus Pharmaceuticals, Dobecure, Iveric Bio, among others. Wet Age-related Macular Degeneration Pipeline Wet Age-related Macular Degeneration Pipeline Insight, 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Wet Age-related Macular Degeneration companies including Opthea Limited, Kodiak Sciences Inc., REGENXBIO, among others. Dry Age-related Macular Degeneration Pipeline Dry Age-related Macular Degeneration Pipeline Insight, 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Dry Age-related Macular Degeneration companies including Allergo Ophthalmics, Gyroscope Therapeutics, Gemini Therapeutics, among others. Other Trending Reports Periodontal Disease Market | Open-Angle Glaucoma Market | Obsessive-Compulsive Disorder Market | Nicotine Addiction Market | Nephrosclerosis Market | Muscle Invasive Bladder cancer Market | Mucinous cystic neoplasms Market | Metastatic Uveal Melanoma Market | ICOS-Next Generation Immunotherapy Market | Herpes Simplex Market | Hepatitis C Market | Guillain-Barre Syndrome Market | Graft Versus Host Disease Market | Goitre Market Related Healthcare Blogs Dry AMD Market: Pegcetacoplan vs Zimura Wet Age-related Macular Degeneration Market Outlook Eye Disorders Treatment Market About DelveInsight DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Gene TherapyAntibodyBiosimilarOligonucleotideCell Therapy

15 Nov 2021

·www.biospace.com

Gemini Therapeutics Reports Third Quarter 2021 Financial Results and Provides Business Update

Nov. 15, 2021 12:00 UTC Gemini Therapeutics Reports Third Quarter 2021 Financial Results and Provides Business Update GEM103 updated safety information presented during AAO including no increased risk for CNV observed to date in ongoing ReGAtta Phase 2a study Expect to provide six-month update from GEM103 ReGAtta study by year-end Scheduled to meet with the FDA in 4Q’21 and expect to initiate late-stage trial for GEM103 in patients with GA in 1Q’22 CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Gemini Therapeutics (Nasdaq: GMTX), a clinical stage precision medicine company developing innovative treatments for genetically-defined age-related macular degeneration (AMD) today reported its financial results for the third quarter ended September 30, 2021 and provided a business update. “This weekend at the American Academy of Ophthalmology (AAO) annual meeting, Dr. Maturi provided a safety update on our ongoing Phase 2a ReGAtta study of GEM103 which shows GEM103 is generally well-tolerated with no increased risk of CNV in over 500 intravitreal injections of GEM103. We look forward to presenting a six-month update for the study before year end. We are meeting with the FDA this quarter and anticipate initiating our late-stage study of GEM103 for geographic atrophy (GA) secondary to dry AMD in the first quarter of 2022,” said Jason Meyenburg, Chief Executive Officer of Gemini Therapeutics, Inc. Recent Business and Clinical Highlights Presented newly available safety data from ongoing ReGAtta Phase 2a study of GEM103 at AAO 2021. During the AAO Meeting in November Dr. Raj Maturi, M.D., Adjunct Clinical Assistant Professor of Ophthalmology, at Indiana University School of Medicine and an investigator in the ReGAtta study, presented an e-poster with newly available safety data that updated the May 2021 initial data from the ongoing ReGAtta Phase 2a study of GEM103 in GA. The most recent safety update from the ReGAtta study showed that across 510 intravitreal injections, GEM103 was generally well-tolerated with no ocular serious adverse events and no interruption in GEM103 therapy. 17 patients (27.4%) experienced mild to moderate ocular adverse events in the study eye and there continued to be no increased risk of CNV in the study eyes treated with GEM103. Presented GEM103 biology and development at 2nd Annual Dry AMD Therapeutic Development Summit. In October 2021 the Company further discussed the previously released initial data from its ongoing Phase 2a ReGAtta study in a presentation titled “Spotlight on GEM103 – Restoring Physiologic Complement Activity with Complement Factor H (CFH) for GA” at the 2nd Annual Dry AMD Therapeutic Development Summit: Uncovering the Largest Untapped Eyecare Market. Announced corporate restructuring to prioritize late-stage clinical development of GEM103 for GA. In October 2021, Gemini announced a corporate restructuring including several executive officer transitions to prioritize assets and focus on initiating and executing GEM103’s resource-intensive pivotal trial in GA. Presented preclinical data on GEM103 at the 13th International Conference on Complement Therapeutics. In September 2021 at the 13th International Conference on Complement Therapeutics, an oral presentation and three poster presentations highlighted the potential of GEM103 as the next class of complement therapeutics for the treatment of AMD by regulating complement. 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 intravitreal administration. Presented data from its ongoing Phase 2a study of GEM103 at EURETINA 2021 Virtual. In September 2021, Dr. Maturi presented Gemini’s previously released initial results from its ongoing Phase 2a ReGAtta study that GEM103 demonstrated the ability to regulate complement in GA patients with treatment resulting in a reduction of elevated complement biomarkers and a dose dependent reduction in the overall inflammatory state. Presented data from its ongoing Phase 2a study of GEM103 at Clinical Trials at the Summit. In August 2021, Gemini participated in panel discussions at Clinical Trials at the Summit and presented an analysis of the baseline GA lesion sizes for patients enrolled in the ongoing Phase 2a ReGAtta study that showed imbalances in GA baseline characteristics prevent efficacy assessment in a non-controlled open-label study. Financial Results Research and development expenses totaled $13.5 million for the three months ended September 30, 2021 compared to $6.7 million for the same period in 2020. The increase was primarily due to increased expenses related to clinical studies and headcount costs. General and administrative expenses were $5.0 million for the three months ended September 30, 2021 compared to $1.2 million for the same period in 2020. The increase was primarily due to increased expenses related to professional fees associated with being a public company as well as headcount costs. For the three months ended September 30, 2021 Gemini reported a net loss of $18.6 million, or $0.43 per share compared to a net loss of $10.0 million, or $0.65 per share, in the corresponding period in 2020. The increase in net loss for the three months ended September 30, 2021 was primarily due to an increase in operating expenses. At September 30, 2021, Gemini held $150.1 million in cash, $6.7 million of principal outstanding debt and 43.1 million shares outstanding. 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 Geographic Atrophy secondary to dry AMD is designed to investigate safety and tolerability, pharmacokinetics (PK), exploratory ocular biomarkers, and measures of retinal anatomy and function. This study is not designed to assess the efficacy of GEM103. GEM103 is delivered monthly by an intravitreal injection, and PK and biomarkers of complement regulation are determined from aqueous humor sampling. The study 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 candidate, GEM103, is a pioneering precision medicine approach, targeting trial enrichment with genetically defined patients. GEM103 targets a genetically defined subset of 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 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, Gemini believes 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 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) AMD is a progressive retinal disease affecting millions of older adults, and is 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 million with wet AMD. The genetic risk of developing dry AMD is significant, with approximately 70% of the risk of advanced disease being attributable 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 the CFH gene 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 is also working to advance a potentiating antibody for CFH, GEM307, towards clinical development for treatment of systemic diseases. For more information, visit . Availability of Other Information About Gemini Therapeutics Investors and others should note that we communicate with our investors and the public using our website ( ), the investor relations website ( ), 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 timing of and costs associated with our restructuring, and the benefits we expect to receive from the restructuring, 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 the 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. Gemini Therapeutics, Inc. Statements of Operations (In Thousands) Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Operating expenses: Research and development $ 13,455 $ 6,727 $ 36,083 $ 20,472 General and administrative 4,995 1,222 15,177 3,774 Total operating expenses 18,450 7,949 51,260 24,246 Loss from operations (18,450 ) (7,949 ) (51,260 ) (24,246 ) Other income (expense): Interest expense (104 ) (2,047 ) (2,073 ) (2,307 ) Interest income 5 1 11 37 Loss on conversion of convertible notes - - (711 ) - Change in fair value of warrant liability - (8 ) - (6 ) Other expense (2 ) - (13 ) - Net loss and comprehensive loss $ (18,551 ) $ (10,003 ) $ (54,046 ) $ (26,522 ) Net loss per share, basic and diluted $ (0.43 ) $ (0.65 ) $ (1.37 ) $ (1.77 ) Weighted average common shares outstanding, basic and diluted 43,091,822 15,282,987 39,427,476 15,016,038 Gemini Therapeutics, Inc. Balance Sheets (In Thousands) September 30, 2021 December 31, 2020 Assets Cash and cash equivalents $ 150,069 $ 4,503 Other current assets 4,731 562 Total current assets 154,800 5,065 Other assets 735 3,254 Total assets $ 155,535 $ 8,319 Liabilities and stockholders' equity (deficit) Current liabilities $ 12,661 $ 24,876 Long-term liabilities 2,013 5,304 Total liabilities 14,674 30,180 Total stockholders' equity (deficit) 140,861 (21,861 ) Total liabilities and stockholders' equity (deficit) $ 155,535 $ 8,319

Small molecular drugFinancial StatementAntibodyFast Track

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R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Wet age-related macular degeneration	Phase 2	United States	Gemini Therapeutics, Inc.	29 Dec 2020
dry age-related macular degeneration	Phase 2	United States	Gemini Therapeutics, Inc.	30 Jul 2020
Geographic Atrophy	Phase 2	United States	Gemini Therapeutics, Inc.	30 Jul 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04684394 (CTgov)	Phase 2	Wet age-related macular degeneration	50	aflibercept+GEM103 (SoC + GEM103)	grnhwhckfb(imtnmflxcy) = furaqdoukf mbanavjcgv (bjpcjcekyu, xgzqtcrvrr - cgtkppxuhw) View more	-	31 Oct 2022
				Sham (SoC + Sham)	grnhwhckfb(imtnmflxcy) = ydgyuhocli mbanavjcgv (bjpcjcekyu, uysvfgxssx - dolxonuseg) View more
NCT04246866 (Pubmed) Manual	Phase 1	Geographic Atrophy	12	GEM103 (50 μg/eye, 100 μg/eye, 250 μg/eye, or 500 μg/eye)	ilymjdxjly(ieusznxyiu) = None yhglgfwxix (baqchzmehe ) View more	Positive	11 Apr 2022
ReGAtta (AAO2021)	Phase 2	Geographic Atrophy complement factor H (CFH) levels \| PD biomarkers in AH	62	GEM103 at 250 µg	kqnotqgotz(uuakbnjhqo) = bvrgfrfawk ephykhnqpj (zkkwnsfhlg, 18.92) View more	Positive	13 Nov 2021
				GEM103 at 500 µg	kqnotqgotz(uuakbnjhqo) = iksjwrrvuu ephykhnqpj (zkkwnsfhlg, 18.92) View more

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