Chylomicron Retention Disease

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.

Montrouge, France, March 7, 2023 DBV Technologies Announces First Patient Screened in VITESSE Phase 3 Clinical Trial in Peanut-Allergic Children 4 – 7 Years Old First patient screened at Midwest Allergy Sinus Asthma in Normal, Illinois VITESSE is DBV’s Phase 3 clinical study evaluating the modified Viaskin Peanut patch in peanut-allergic children ages 4 – 7 years DBV Technologies (Euronext: DBV – ISIN: FR0010417345 – Nasdaq Market: DBVT), a clinical-stage biopharmaceutical company, today announced that the first patient has been screened in the Company’s VITESSE (Viaskin Peanut Immunotherapy Trial to Evaluate Safety Simplicity and Efficacy) Phase 3 clinical trial that will evaluate the modified Viaskin™ Peanut 250 μg patch (DBV712) in peanut-allergic children ages 4 through 7 years. Viaskin Peanut 250 μg is a novel treatment designed to re-educate the immune system by introducing microgram amounts of peanut allergen to the immune system through intact skin. The VITESSE trial will use the modified Viaskin Peanut 250 μg patch and enroll 600 subjects, randomized 2:1 active versus placebo. The study will involve approximately 80 trial sites across the United States, Canada, Australia, and Europe. The primary efficacy endpoint of the study is the percentage of treatment responders in the active versus placebo arms at Month 12. Secondary efficacy endpoints include changes in cumulative reactive dose (CRD), eliciting dose (ED) and severity of allergic reaction at Month 12 food challenge. “I am pleased that the first trial participant has been screened in the VITESSE study,” said David Fleischer, MD, FAAAAI, FACAAI, Children’s Hospital Colorado and Global Principal Investigator for the VITESSE study, “These peanut-allergic children represent a cohort of patients that are eagerly awaiting potential treatment options. I am looking forward to leading the team of highly skilled VITESSE investigators and their staff as we generate these critical data that may one day potentially support a BLA submission for Viaskin Peanut.” Last year, following productive exchanges with the U.S. Food and Drug Administration (FDA), DBV finalized the VITESSE protocol and received the FDA’s support to advance the study. Previously, the FDA had requested changes to certain elements of the VITESSE protocol with the intent for the trial to support a future Biologics License Application (BLA). "The initiation of the VITESSE Phase 3 Study in peanut-allergic children ages 4 - 7 years represents a culmination of years of research and collaboration between DBV, FDA, patient groups, site investigators, trial centers, and, of course, the support and unwavering encouragement from food-allergic families,” said Pharis Mohideen, MD, Chief Medical Officer, DBV Technologies. “DBV is proud to see that patient screening has begun in Q1 2023. This important milestone reflects incredible work from the DBV team and swift action in beginning enrollment from Principal Investigators like Dr. Dareen Siri and others. We are grateful for the strong collaboration that will continue throughout the VITESSE study.” Principal Investigator, Dr. Dareen Siri, FAAAAI, FACAAI from Midwest Allergy Sinus Asthma, in Normal and Springfield, Illinois was the first to conduct a screening visit for a potential new enrollee. "I am thrilled that our talented team of clinicians was the first to screen a patient for the VITESSE clinical study,” said Dareen Siri, MD, Midwest Allergy Sinus Asthma, Normal, IL. “The initiation of patient enrollment in VITESSE reinforces our commitment to peanut-allergic children and their families and is an important step in generating the data needed to potentially advance Viaskin Peanut to market. I am grateful that peanut-allergic children have the opportunity to participate in a trial that may one day help other kids just like them living with peanut allergy. There is a lot of excitement around the VITESSE study, and I am proud our team will be an integral part of it.” DBV anticipates screening the last patient in 1H 2024 and announcing topline results in 1H 2025. About VITESSEThe VITESSE trial will enroll 600 subjects, randomized 2:1 active versus placebo. The study will involve approximately 80 trial sites across the United States, Canada, Australia and Europe. Dr. David Fleischer, Colorado Children’s Hospital, will act as the global study Principal Investigator. The primary efficacy endpoint is the percentage of treatment responders in the active versus placebo arms at Month 12. The primary efficacy analysis includes the success criterion of a lower bound of the confidence interval of the difference in responder rates between active and placebo groups being greater than or equal to 15%. A treatment responder is defined as either a subject with a baseline ED ≤30 mg who reaches an ED ≥300 mg of peanut protein at Month 12, or a subject with a baseline ED = 100 mg who reaches an ED ≥600 mg of peanut protein at Month 12. A double-blind, placebo-controlled food challenge (DBPCFC) will be administered at baseline and Month 12 to determine a subject’s ED at both timepoints. During the screening period, subjects will undergo an initial visit with assessment for eligibility according to peanut skin prick test (SPT) and serum peanut IgE. Those meeting these criteria will proceed to a DBPCFC to confirm their peanut allergy and establish an entry peanut ED. The entry DBPCFC will be 1 mg peanut protein, and will escalate up to a highest single dose of 100 mg peanut protein. Subjects who react with an ED at or below the dose of 100 mg peanut protein and meet all other inclusion and no exclusion criteria are considered eligible. At Month 12, a post-treatment DBPCFC will be performed, with a starting dose of 3 mg peanut protein, escalating to a highest dose of 1,000 mg peanut protein according to the following schedule: 3, 10, 30, 100, 300, 600, 1,000 mg. Secondary efficacy endpoints include changes in CRD, ED and severity of allergic reaction at Month 12 food challenge. About DBV TechnologiesDBV Technologies is developing Viaskin™, an investigational proprietary technology platform with broad potential applications in immunotherapy. Viaskin is based on epicutaneous immunotherapy, or EPIT™, and is DBV Technologies’ method of delivering biologically active compounds to the immune system through intact skin. With this new class of non-invasive product candidates, the Company is dedicated to safely transforming the care of food-allergic patients. DBV Technologies’ food allergies programs include ongoing clinical trials of Viaskin Peanut. DBV Technologies has global headquarters in Montrouge, France, and North American operations in Basking Ridge, NJ. The Company’s ordinary shares are traded on segment B of Euronext Paris (Ticker: DBV, ISIN code: FR0010417345) and the Company’s ADSs (each representing one-half of one ordinary share) are traded on the Nasdaq Global Select Market (Ticker: DBVT). Forward Looking Statements This press release may contain forward-looking statements and estimates, including statements regarding the therapeutic potential of Viaskin™ Peanut as a treatment for peanut-allergic children and the potential benefits of EPIT™, DBV Technologies’ clinical development and regulatory plans, timing and projections of VITESSE study milestones, and timing and anticipated results of interactions with regulatory agencies. All statements about VITESSE study milestones, enrollment and anticipated results contained herein are DBV’s best estimates and projections are based on performance of previous studies and are subject to known and unknown risks, uncertainties and other factors that may cause actual results, performance and achievements with respect to the VITESSE study to differ materially from the estimates and projections contained herein. These forward-looking statements and estimates are not promises or guarantees and involve substantial risks and uncertainties and may be impacted by market conditions as well as other risks and uncertainties set forth in DBV Technologies’ regulatory filings with the Autorité des Marchés Financiers (“AMF”), DBV Technologies’ filings and reports with the U.S. Securities and Exchange Commission (“SEC”) and future filings and reports made with the AMF and SEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. Other than as required by applicable law, DBV Technologies undertakes no obligation to update or revise the information contained in this Press Release. Investor Contact Anne PollakDBV Technologies+1 857-529-2363anne.pollak@dbv-technologies.com Media Contact Angela MarcucciDBV Technologies+1 646-842-2393angela.marcucci@dbv-technologies.com Viaskin and EPIT are trademarks of DBV Technologies. Attachment PDF Version