Hypobetalipoproteinemias

Company to host conference call on Wednesday, November 15, 2023 at 8:00 a.m. ETBOSTON and ATLANTA, Nov. 14, 2023 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease ("PD"), Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today reported financial results for the third quarter ended September 30, 2023 and highlighted recent developments. “We are very pleased with the progress of the last quarter,” noted Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. “Our efforts to improve drug delivery for protein kinase inhibitors brought IkT-001Pro to pre-NDA stage just 15 months after opening of the IND. Our neurodegenerative disease programs with risvodetinib are expanding, with 20% of the Phase 2 201 Trial in untreated Parkinson’s disease enrolled coupled with our efforts to initiate a Phase 2 program in Multiple System Atrophy. Finally, our internal and external medicinal chemistry programs are yielding important insights into the design of next generation Abl kinase inhibitors that could lead to a pipeline of beneficial products for Abl kinase-related diseases. Collectively, this has been a very productive period for the Company.” Recent Developments and Upcoming Milestones: Advancing Phase 2 '201' Trial of Risvodetinib (IkT-148009) in untreated Parkinson’s disease: As of November 10, 2023, 28 sites are open and actively evaluating prospective trial participants. Twenty-four participants have been enrolled, 7 prospective participants are in screening and 15 potential participants are going through informed consents.The 201 Trial patient portal (www.the201trial.com) has been visited by more than 20,000 unique people since launch in September, 2023. The pre-qualification process has led to 201 unique individuals to contact open clinical sites, the first step in the evaluation process that could lead to enrollment. Monthly site enrollments have increased month-over-month since this patient outreach program was initiated. Unblinded functional analysis from the 201 Trial are encouraging: In August 2023, unblinded functional assessments of 11 patients with untreated Parkinson’s disease were presented at the Movement Disorder Congress in Copenhagen, Denmark. These participants were withdrawn from the trial following the FDA’s temporary clinical hold in November, 2022, which was lifted January, 2023. These assessments showed that patients receiving the 200 mg dose of risvodetinib (N=3) saw a greater than 10 point improvement over placebo in the sum of motor and non-motor scores after once daily treatment for up to 11 weeks; by contrast, a typical patient with Parkinson’s disease would worsen by 3 to 6 points in the sum of motor and non-motor score assessments over a 12 month period. While the number of treated participants is too small for the Company to conclude a clinical benefit, these early data are cautiously encouraging.Received Orphan Drug Designation in Multiple System Atrophy (MSA): In October 2023, risvodetinib was granted Orphan Drug Designation by the FDA for the treatment of MSA. In animal model studies of MSA, risvodetinib was shown to be therapeutically active. The designation by the FDA underscores the need for innovative treatment options for patients afflicted with this rare and rapidly progressing Parkinson’s-related disorder. The Company is pursuing a comparable designation in the European Union, or E.U., as part of its efforts to initiate a Phase 2 clinical trial to evaluate risvodetinib in MSA. The Company is discussing conduct of the trial with private foundations, Federal and industry stakeholders in an effort to initiate this trial in the future.Completed the ‘501’ bioequivalence studies with IkT-001Pro: In October 2023, Inhibikase completed its bioequivalence studies of IkT-001Pro, measuring the bioequivalent dose to both 400 mg and 600 mg imatinib mesylate. The study enrolled a total of 66 healthy volunteers in three parts. In single dose studies, bioequivalent IkT-001Pro induced fewer neurological, musculoskeletal and gastrointestinal adverse events relative to 400 mg imatinib mesylate. Gastrointestinal adverse events were more persistent for imatinib mesylate in the study evaluating bioequivalence to 600 mg imatinib mesylate. Measures of bioequivalence along with safety and tolerability data are being submitted as briefing materials for a pre-NDA meeting with the FDA to reach agreement on the requirements for approval of IkT-001Pro under the 505(b)(2) statute.Initiated preclinical development of second-generation c-Abl inhibitors: In August 2023, Inhibikase initiated preclinical development of new molecules arising from internal medicinal chemistry and external collaborations identifying second generation molecules that could enhance suppression of neurodegeneration or address other diseases that could benefit from Abl kinase inhibition. Third Quarter Financial Results Net Loss: Net loss for the three months ended September 30, 2023 was $4.60 million, or $0.86 per share, compared to a net loss of $4.49 million, or $1.06 per share in the quarter ended September 30, 2022. R&D Expenses: Research and development expenses were $3.23 million for the quarter ended September 30, 2023 compared to $2.98 million in the quarter ended September 30 2022. The increase was primarily due to the Company's ongoing Phase 2 ‘201’ PD clinical trial costs. SG&A Expenses: Selling, general and administrative expenses for the quarter ended September 30, 2023 were $1.62 million compared to $1.54 million for the quarter ended September 30, 2022. The increase was driven by net increase in normal selling, general and administrative expenses. Cash Position: Cash, cash equivalents and marketable securities were $16.83 million as of September 30, 2023. The Company expects that existing cash and cash equivalents will be sufficient to fund operations into the fourth quarter of 2024. Conference Call InformationThe conference call can be accessed by dialing 1-833-816-1414 (United States) or 1-412-317-0506 (International) with the conference code 0866324. A live webcast may be accessed using the link here, or by visiting the investors section of the Company's website at www.inhibikase.com. After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call. About Inhibikase (www.inhibikase.com) Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing therapeutics for Parkinson's disease and related disorders. Inhibikase's multi-therapeutic pipeline has a primary focus on neurodegeneration and its lead program risvodetinib, an Abelson Tyrosine Kinase (c-Abl) inhibitor, targets the treatment of Parkinson's disease inside and outside the brain as well as other diseases that arise from Abelson Tyrosine Kinases. Its multi-therapeutic pipeline is pursuing Parkinson's-related disorders of the brain and GI tract, orphan indications related to Parkinson's disease such as Multiple System Atrophy, and drug delivery technologies for kinase inhibitors such as IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate that the Company believes will provide a better patient experience with fewer on-dosing side-effects. The Company's RAMP™ medicinal chemistry program has identified a number of follow-on compounds to risvodetinib to be potentially applied to other cognitive and motor function diseases of the brain. Inhibikase is headquartered in Atlanta, Georgia with an office in Lexington, Massachusetts. Social Media DisclaimerInvestors and others should note that we announce material financial information to our investors using our investor relations website, press releases, SEC filings and public conference calls and webcasts. The Company intends to also use X, Facebook, LinkedIn and YouTube as a means of disclosing information about the Company, its services and other matters and for complying with its disclosure obligations under Regulation FD. Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking terminology such as "believes," "expects," "may," "will," "should," "anticipates," "plans," or similar expressions or the negative of these terms and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Inhibikase's current expectations and assumptions. Such statements are subject to certain risks and uncertainties, which could cause Inhibikase's actual results to differ materially from those anticipated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include our ability to successfully conduct clinical trials that are statistically significant and that results from our animal studies may not be replicated in humans, as well as such other factors that are included in our periodic reports on Form 10-K and Form 10-Q that we file with the U.S. Securities and Exchange Commission. Any forward-looking statement in this release speaks only as of the date of this release. Inhibikase undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contacts: Company Contact:Milton H. Werner, PhDPresident & CEO678-392-3419info@inhibikase.com Investor Relations:Alex LoboSternIR, Inc.alex.lobo@sternir.com Inhibikase Therapeutics, Inc.Condensed Consolidated Balance Sheets September 30, 2023 December 31, 2022 (unaudited) (Note 3) Assets ​ Current assets: ​ Cash and cash equivalents$14,861,309 $7,188,553 Marketable securities 1,970,260 15,861,620 Accounts receivable — 39,881 Prepaid research and development 347,565 1,117,616 Prepaid expenses and other current assets 371,538 163,452 Total current assets 17,550,672 24,371,122 Equipment and improvements, net 79,940 236,532 Right-of-use asset 250,090 328,643 Total assets$17,880,702 $24,936,297 Liabilities and stockholders’ equity ​ Current liabilities: ​ Accounts payable$734,561 $1,151,173 Lease obligation, current 149,030 145,836 Accrued expenses and other current liabilities 1,858,215 2,398,436 Total current liabilities 2,741,806 3,695,445 Lease obligation, net of current portion 121,013 205,451 Total liabilities 2,862,819 3,900,896 Commitments and contingencies (see Note 13) ​ Stockholders’ equity:​ ​ Preferred stock, $0.001 par value; 10,000,000 shares authorized; 0 shares issued and outstanding at September 30, 2023 and December 31, 2022 — — Common stock, $0.001 par value; 100,000,000 shares authorized; 5,360,326 and 4,224,294 shares issued and outstanding at September 30, 2023 and December 31, 2022 5,361 4,224 Additional paid-in capital 77,735,450 68,798,301 Accumulated other comprehensive income (loss) (143) 104,718 Accumulated deficit (62,722,785) (47,871,842)Total stockholders' equity 15,017,883 21,035,401 Total liabilities and stockholders’ equity$17,880,702 $24,936,297 Inhibikase Therapeutics, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Revenue Grant revenue$79,569 $7,291 $260,500 $59,874 Total revenue 79,569 7,291 260,500 59,874 Costs and expenses: Research and development 3,225,551 2,981,653 10,615,368 8,980,827 Selling, general and administrative 1,622,894 1,538,737 5,331,358 4,872,681 Total costs and expenses 4,848,445 4,520,390 15,946,726 13,853,508 Loss from operations (4,768,876) (4,513,099) (15,686,226) (13,793,634)Interest income 173,677 18,536 835,283 18,531 Net loss (4,595,199) (4,494,563) (14,850,943) (13,775,103)Other comprehensive income (loss), net of tax Unrealized gain (loss) on marketable securities 1,571 26,828 (104,861) 26,828 Comprehensive loss$(4,593,628) $(4,467,735) $(14,955,804) $(13,748,275)Net loss per share – basic and diluted$(0.86) $(1.06) $(2.93) $(3.26)Weighted-average number of common shares – basic and diluted 5,342,337 4,224,294 5,060,447 4,223,099

Company to host conference call on Wednesday, November 15, 2023 at 8:00 a.m. ET BOSTON and ATLANTA, Nov. 14, 2023 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease ("PD"), Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today reported financial results for the third quarter ended September 30, 2023 and highlighted recent developments. “We are very pleased with the progress of the last quarter,” noted Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. “Our efforts to improve drug delivery for protein kinase inhibitors brought IkT-001Pro to pre-NDA stage just 15 months after opening of the IND. Our neurodegenerative disease programs with risvodetinib are expanding, with 20% of the Phase 2 201 Trial in untreated Parkinson’s disease enrolled coupled with our efforts to initiate a Phase 2 program in Multiple System Atrophy. Finally, our internal and external medicinal chemistry programs are yielding important insights into the design of next generation Abl kinase inhibitors that could lead to a pipeline of beneficial products for Abl kinase-related diseases. Collectively, this has been a very productive period for the Company.” Recent Developments and Upcoming Milestones: Advancing Phase 2 '201' Trial of Risvodetinib (IkT-148009) in untreated Parkinson’s disease: As of November 10, 2023, 28 sites are open and actively evaluating prospective trial participants. Twenty-four participants have been enrolled, 7 prospective participants are in screening and 15 potential participants are going through informed consents. The 201 Trial patient portal ( ) has been visited by more than 20,000 unique people since launch in September, 2023. The pre-qualification process has led to 201 unique individuals to contact open clinical sites, the first step in the evaluation process that could lead to enrollment. Monthly site enrollments have increased month-over-month since this patient outreach program was initiated. Unblinded functional analysis from the 201 Trial are encouraging: In August 2023, unblinded functional assessments of 11 patients with untreated Parkinson’s disease were presented at the Movement Disorder Congress in Copenhagen, Denmark. These participants were withdrawn from the trial following the FDA’s temporary clinical hold in November, 2022, which was lifted January, 2023. These assessments showed that patients receiving the 200 mg dose of risvodetinib (N=3) saw a greater than 10 point improvement over placebo in the sum of motor and non-motor scores after once daily treatment for up to 11 weeks; by contrast, a typical patient with Parkinson’s disease would worsen by 3 to 6 points in the sum of motor and non-motor score assessments over a 12 month period. While the number of treated participants is too small for the Company to conclude a clinical benefit, these early data are cautiously encouraging. Received Orphan Drug Designation in Multiple System Atrophy (MSA): In October 2023, risvodetinib was granted Orphan Drug Designation by the FDA for the treatment of MSA. In animal model studies of MSA, risvodetinib was shown to be therapeutically active. The designation by the FDA underscores the need for innovative treatment options for patients afflicted with this rare and rapidly progressing Parkinson’s-related disorder. The Company is pursuing a comparable designation in the European Union, or E.U., as part of its efforts to initiate a Phase 2 clinical trial to evaluate risvodetinib in MSA. The Company is discussing conduct of the trial with private foundations, Federal and industry stakeholders in an effort to initiate this trial in the future. Completed the ‘501’ bioequivalence studies with IkT-001Pro: In October 2023, Inhibikase completed its bioequivalence studies of IkT-001Pro, measuring the bioequivalent dose to both 400 mg and 600 mg imatinib mesylate. The study enrolled a total of 66 healthy volunteers in three parts. In single dose studies, bioequivalent IkT-001Pro induced fewer neurological, musculoskeletal and gastrointestinal adverse events relative to 400 mg imatinib mesylate. Gastrointestinal adverse events were more persistent for imatinib mesylate in the study evaluating bioequivalence to 600 mg imatinib mesylate. Measures of bioequivalence along with safety and tolerability data are being submitted as briefing materials for a pre-NDA meeting with the FDA to reach agreement on the requirements for approval of IkT-001Pro under the 505(b)(2) statute. Initiated preclinical development of second-generation c-Abl inhibitors: In August 2023, Inhibikase initiated preclinical development of new molecules arising from internal medicinal chemistry and external collaborations identifying second generation molecules that could enhance suppression of neurodegeneration or address other diseases that could benefit from Abl kinase inhibition. Third Quarter Financial Results Net Loss: Net loss for the three months ended September 30, 2023 was $4.60 million, or $0.86 per share, compared to a net loss of $4.49 million, or $1.06 per share in the quarter ended September 30, 2022. R&D Expenses: Research and development expenses were $3.23 million for the quarter ended September 30, 2023 compared to $2.98 million in the quarter ended September 30 2022. The increase was primarily due to the Company's ongoing Phase 2 ‘201’ PD clinical trial costs. SG&A Expenses: Selling, general and administrative expenses for the quarter ended September 30, 2023 were $1.62 million compared to $1.54 million for the quarter ended September 30, 2022. The increase was driven by net increase in normal selling, general and administrative expenses. Cash Position: Cash, cash equivalents and marketable securities were $16.83 million as of September 30, 2023. The Company expects that existing cash and cash equivalents will be sufficient to fund operations into the fourth quarter of 2024. Conference Call Information The conference call can be accessed by dialing 1-833-816-1414 (United States) or 1-412-317-0506 (International) with the conference code 0866324. A live webcast may be accessed using the link here, or by visiting the investors section of the Company's website at . After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call. About Inhibikase ( ) Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing therapeutics for Parkinson's disease and related disorders. Inhibikase's multi-therapeutic pipeline has a primary focus on neurodegeneration and its lead program risvodetinib, an Abelson Tyrosine Kinase (c-Abl) inhibitor, targets the treatment of Parkinson's disease inside and outside the brain as well as other diseases that arise from Abelson Tyrosine Kinases. Its multi-therapeutic pipeline is pursuing Parkinson's-related disorders of the brain and GI tract, orphan indications related to Parkinson's disease such as Multiple System Atrophy, and drug delivery technologies for kinase inhibitors such as IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate that the Company believes will provide a better patient experience with fewer on-dosing side-effects. The Company's RAMP™ medicinal chemistry program has identified a number of follow-on compounds to risvodetinib to be potentially applied to other cognitive and motor function diseases of the brain. Inhibikase is headquartered in Atlanta, Georgia with an office in Lexington, Massachusetts. Social Media Disclaimer Investors and others should note that we announce material financial information to our investors using our investor relations website, press releases, SEC filings and public conference calls and webcasts. The Company intends to also use X, Facebook, LinkedIn and YouTube as a means of disclosing information about the Company, its services and other matters and for complying with its disclosure obligations under Regulation FD. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking terminology such as "believes," "expects," "may," "will," "should," "anticipates," "plans," or similar expressions or the negative of these terms and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Inhibikase's current expectations and assumptions. Such statements are subject to certain risks and uncertainties, which could cause Inhibikase's actual results to differ materially from those anticipated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include our ability to successfully conduct clinical trials that are statistically significant and that results from our animal studies may not be replicated in humans, as well as such other factors that are included in our periodic reports on Form 10-K and Form 10-Q that we the U.S. Securities and Exchange Commission. Any forward-looking statement in this release speaks only as of the date of this release. Inhibikase undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contacts: Company Contact: Milton H. Werner, PhD President & CEO 678-392-3419 info@inhibikase.com Investor Relations: Alex Lobo SternIR, Inc. alex.lobo@sternir.com Inhibikase Therapeutics, Inc. Condensed Consolidated Balance Sheets September 30, 2023 December 31, 2022 (unaudited) (Note 3) Assets ​ Current assets: ​ Cash and cash equivalents $ 14,861,309 $ 7,188,553 Marketable securities 1,970,260 15,861,620 Accounts receivable — 39,881 Prepaid research and development 347,565 1,117,616 Prepaid expenses and other current assets 371,538 163,452 Total current assets 17,550,672 24,371,122 Equipment and improvements, net 79,940 236,532 Right-of-use asset 250,090 328,643 Total assets $ 17,880,702 $ 24,936,297 Liabilities and stockholders’ equity ​ Current liabilities: ​ Accounts payable $ 734,561 $ 1,151,173 Lease obligation, current 149,030 145,836 Accrued expenses and other current liabilities 1,858,215 2,398,436 Total current liabilities 2,741,806 3,695,445 Lease obligation, net of current portion 121,013 205,451 Total liabilities 2,862,819 3,900,896 Commitments and contingencies (see Note 13) ​ Stockholders’ equity: ​ ​ Preferred stock, $0.001 par value; 10,000,000 shares authorized; 0 shares issued and outstanding at September 30, 2023 and December 31, 2022 — — Common stock, $0.001 par value; 100,000,000 shares authorized; 5,360,326 and 4,224,294 shares issued and outstanding at September 30, 2023 and December 31, 2022 5,361 4,224 Additional paid-in capital 77,735,450 68,798,301 Accumulated other comprehensive income (loss) (143 ) 104,718 Accumulated deficit (62,722,785 ) (47,871,842 ) Total stockholders' equity 15,017,883 21,035,401 Total liabilities and stockholders’ equity $ 17,880,702 $ 24,936,297 Inhibikase Therapeutics, Inc. Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Revenue Grant revenue $ 79,569 $ 7,291 $ 260,500 $ 59,874 Total revenue 79,569 7,291 260,500 59,874 Costs and expenses: Research and development 3,225,551 2,981,653 10,615,368 8,980,827 Selling, general and administrative 1,622,894 1,538,737 5,331,358 4,872,681 Total costs and expenses 4,848,445 4,520,390 15,946,726 13,853,508 Loss from operations (4,768,876 ) (4,513,099 ) (15,686,226 ) (13,793,634 ) Interest income 173,677 18,536 835,283 18,531 Net loss (4,595,199 ) (4,494,563 ) (14,850,943 ) (13,775,103 ) Other comprehensive income (loss), net of tax Unrealized gain (loss) on marketable securities 1,571 26,828 (104,861 ) 26,828 Comprehensive loss $ (4,593,628 ) $ (4,467,735 ) $ (14,955,804 ) $ (13,748,275 ) Net loss per share – basic and diluted $ (0.86 ) $ (1.06 ) $ (2.93 ) $ (3.26 ) Weighted-average number of common shares – basic and diluted 5,342,337 4,224,294 5,060,447 4,223,099

PRESS RELEASE Beacon Therapeutics launches with £96 million ($120 million) to develop a new generation of gene therapies for retinal diseases resulting in blindness New leading gene therapy company created combining late-stage X-linked retinitis pigmentosa (XLRP) program from AGTC, a preclinical program spun out from the University of Oxford and a second preclinical program to deliver a broad ophthalmic development pipelineLed by a highly experienced leadership team including David Fellows, Dr. Nadia Waheed, and Dr. Abraham Scaria, each with decades of ophthalmic gene therapy experience£96m ($120 million) in funding from lead investor Syncona and additional investors including Oxford Science Enterprises (OSE) to progress pipeline to key value inflection points London and Oxford, UK, 12 June 2023 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a purpose to restore and improve the vision of patients with retinal diseases, launches today and will develop a new generation of gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness. Syncona Limited, with additional investors including OSE, has provided £96 million ($120 million) to fund the acquisition of AGTC and provide capital to take each of Beacon Therapeutics’s development candidates through to value inflection points. Beacon Therapeutics’s lead clinical asset is AGTC-501, a gene therapy program currently in Phase II clinical trials for the treatment of XLRP that was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501 correctly expresses the full length RPGR protein, thereby addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics’s first pre-clinical asset is an intravitreally (IVT) delivered novel AAV based program for dry Age-related Macular Degeneration (dry AMD). Dry AMD is a leading cause of irreversible vision loss in people over 60, if left untreated. Around 20 million people in the United States suffer from AMD. The second pre-clinical asset is targeting cone-rod dystrophy (CRD) which is caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene. The program has been licensed from the laboratory of Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford. Prof. MacLaren, a world leading retinal expert and an experienced biotech company founder and a Co-Founder of Beacon Therapeutics, will become a key scientific advisor to the company and will join the board of directors. To bolster its pipeline in the future, Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. Beacon Therapeutics will be led by David Fellows, the former Chief Executive of Nightstar Therapeutics with over 40 years’ experience in the ophthalmology field. He will be joined by retinal expert Dr. Nadia Waheed as Chief Medical Officer, formerly from Gyroscope Therapeutics. Dr. Abraham Scaria also joins the team from AGTC as Chief Scientific Officer bringing 25 years of experience in gene therapy, ranging from discovery research to early-stage clinical trials. David Fellows, Chief Executive Officer of Beacon Therapeutics commented: “Beacon Therapeutics combines a broad development pipeline, a deep scientific foundation, a strong clinical network, and a highly experienced management team to drive forward a unique late-stage clinical and pre-clinical pipeline. With the 12-month data from our Phase II SKYLINE trial for AGTC-501 expected shortly and two highly innovative and differentiated pipeline assets for prevalent and rare blinding diseases, we are excited to be building a new leader in the ophthalmic gene therapy space.” Chris Hollowood, CEO of Syncona Limited, the lead investor in Beacon Therapeutics commented: “By acquiring AGTC and combining its late-stage program with highly complementary and innovative science from the University of Oxford and elsewhere, we have created an exciting new gene therapy company in an area where we have a great breadth and depth of knowledge and expertise. We look forward to working closely with Beacon Therapeutics’ exceptional management team to advance these assets through the clinic and towards people suffering from debilitating retinal diseases.” Professor Robert MacLaren, Professor of Ophthalmology, University of Oxford, and Co-Founder of Beacon Therapeutics commented: “I have dedicated many years to translating pioneering gene therapies from the lab through to clinical trial phase with the support of my team in the Nuffield Laboratory of Ophthalmology and NIHR Oxford Biomedical Research Centre (BRC). Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data. The company has both highly innovative retinal gene therapy programmes and the means to target them to the main causes of blindness in both the young and old.” Katya Smirnyagina, Senior Partner, Life Sciences, Oxford Science Enterprises (OSE) added: “Beacon Therapeutics is truly an exciting new company built around an established scientific foundation and a wealth of experience in the gene therapy and ophthalmology space. We are delighted that expertise spun out from the University of Oxford will become a key component to drive the future success of the business that could bring significant change in the treatment of a broad range of retinal diseases that cause blindness.”Broad ophthalmic development pipelineThe company’s lead clinical gene therapy program AGTC-501 has a strong body of clinical evidence targeting XLRP, an inherited rare form of retinitis pigmentosa that causes progressive vision loss in boys and young men. AGTC-501 demonstrated meaningful efficacy and a good safety profile in the recent Phase I/II HORIZON trial and expects to be publishing 12-month data from its Phase II SKYLINE trial in the second half of 2023. In addition, Beacon Therapeutics is awaiting feedback from the US Food and Drug Administration (FDA) regarding the study design of its upcoming VISTA clinical trial, a Phase II/III study to assess the effect of AGTC-501 on the symptoms of retinitis pigmentosa in additional patients with XLRP. The first preclinical program features intravitreally (IVT) delivered gene therapy for dry AMD. IVT delivery provides access to a significantly greater number of patients than sub-retinal delivery as it can be administered within an outpatient clinic rather than requiring surgery. The second preclinical program, CRD, targets mutations in the CDHR1 gene, which may cause central vision loss similar to dry AMD in older people and severe sight loss in younger patients. The Company has a new location in London, UK, complementing existing facilities in Boston, Massachusetts and Alachua, Florida, US. Contacts:Beacon Therapeutics:David Fellows, CEOEmail: info@beacontx.com Consilium Strategic Communications:Alex Harrison, Ashley TappEmail: Beacon@consilium-comms.com Tel: +44 (0)20 3709 5700 About Beacon Therapeutics Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises. Find out more about us online at: www.beacontx.com.