Cenegermin-BKBJ

Positive proof-of-concept clinical data show substantial improvements in visual acuity in limbal stem cell deficiency ( LSCD) patients treated with CSB-001 Pivotal studies for CSB-001 in LSCD planned for 1H 2027 Series B funds Claris through pivotal development and supports pre-commercialization activities Stephen Brady appointed President and Chief Executive Officer, Brian Baum named Chief Commercial Officer, and Marc de Garidel appointed Chairman of the Board ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); JERSEY CITY, N.J., July 28, 2026 (GLOBE NEWSWIRE) -- Claris Biotherapeutics, Inc. , a late-stage biopharmaceutical company dedicated to advancing transformative therapies for sight-threatening conditions where limited or no treatment options exist, today announced multiple key milestones, including the close of a $118 million Series B financing and key executive and board appointments. These catalysts are designed to accelerate the company’s next phase of growth as it advances CSB-001 (oremepermin alfa ophthalmic solution) based on positive proof-of-concept data towards the planned initiation of pivotal studies in 1H 2027 in patients with limbal stem cell deficiency (LSCD), a blinding disease impacting the ocular surface, representing an area of high unmet need where no approved drug therapy exists. Financing Details The $118M Series B financing was co-led by new investors Samsara Biocapital and Catalio Capital Management, and joined by Adage Capital Management, Sofinnova Investments, Aisling Capital, and ADAR1 Capital Management, with continued support from all existing investors, Novo Holdings, Janus Henderson Investors, and Mass General Brigham Ventures. Proceeds from the fundraise will be used to complete Claris’ ongoing clinical trials in patients with LSCD, including a proof-of-concept study evaluating CSB-001 and a non-interventional natural history study. The financing will also fund the company through a planned pivotal program of CSB-001 in patients with LSCD, scheduled to start in 1H 2027, and enable pre-commercialization activities. CSB-001 targets the underlying pathophysiology of LSCD by promoting corneal epithelial regeneration while modulating inflammation and fibrosis. Clinical data generated to date have demonstrated substantial improvements in visual acuity. Claris expects to report its proof-of-concept results in LSCD during the second half of 2026. In connection with the financing, Stephen Brady, President and CEO of Claris, Mike Dybbs, Ph.D., Partner at Samsara Biocapital, Matthew Hobson, Ph.D., Principal at Catalio Capital Management, and Maha Radhakrishnan, M.D., Executive Partner at Sofinnova Investments, joined the board of directors. Claris Appointments Stephen Brady, President & CEO , has nearly 30 years of operational, transactional, and board experience spanning corporate strategy, M&A, business development, and finance across public and private life sciences companies. Prior to Claris, Mr. Brady was the Chairman of the Board and CEO of Tempest Therapeutics, and prior to this position, he was executive vice president of strategy and finance at Immune Design and played a key role in the company's IPO, financings, licensing deals, and eventual sale to Merck. Earlier in his career, he held progressively senior roles at several biopharmaceutical companies, including vice president of corporate development at Proteolix. In this position, Mr. Brady led Proteolix’s business development and strategic efforts that culminated in the company’s sale to Onyx Pharmaceuticals. Proteolix’s lead asset, Kyprolis ® (carfilzomib), subsequently became the cornerstone of Onyx and a principal driver of Amgen’s $10.4 billion acquisition of Onyx. Mr. Brady currently serves on the Board of Directors of Claris and the Biotechnology Innovation Organization (BIO). Brian Baum, CCO, brings to Claris more than 20 years of program and lifecycle management, commercial strategy, marketing, market access, commercial operations, and specialty product launch experience in biopharma. Prior to Claris, Mr. Baum served as Senior Vice President at Dompé Pharmaceuticals and led critical commercial initiatives that supported the successful U.S. launch and growth of Oxervate ® , a recombinant human nerve growth factor approved in 2018 for neurotrophic keratitis, a rare and serious eye disease with no prior FDA-approved therapies. As a senior commercial leader, Mr. Baum played a key role in driving Oxervate’s growth, with annual U.S. sales surpassing $1.1 billion in 2024. Prior to Dompé, Mr. Baum spent ten years at Genentech in a range of U.S. and global commercial leadership roles, including supporting the launch and commercialization of Lucentis ® across three major retinal indications: wAMD, RVO, and DME. Marc de Garidel, Chairman, is a recognized biotech entrepreneur and global pharmaceutical executive with a distinguished track record of building and scaling innovative healthcare companies, advancing breakthrough therapies, and creating substantial shareholder value through organic growth and strategic M&A across the biotechnology sector. Since May 2023, Mr. de Garidel has served as CEO of Abivax, leading the development of obefazimod for ulcerative colitis and driving exceptional corporate growth, from a $500 million to a $14 billion company in over three years. Prior to Abivax, he was CEO of CinCor Pharma from 2020 to 2023, where he successfully led the company through its acquisition by AstraZeneca. CinCor’s lead therapy was subsequently approved by the FDA as a first-in-class treatment for resistant hypertension. From 2018 to 2020, Mr. de Garidel served as CEO of Corvidia Therapeutics, overseeing its strategic growth and successful acquisition by Novo Nordisk. Earlier in his career, he served as CEO of Ipsen from 2010 to 2016, where he transformed the company into a leading global oncology player through the expansion of Somatuline and the landmark ex-U.S. licensing agreement for Cabometyx. Mr. de Garidel began his career at Eli Lilly and Company before spending 15 years at Amgen in senior international leadership roles. He has also served as Chairman of Ipsen since late 2010. “I’m thrilled to join Claris at such a pivotal moment in the company’s evolution,” said Mr. Brady, President and CEO of Claris. “The strong interest in CSB-001 reflects both its promise for patients and commercial potential. With the company financed through Phase 3 and the team in place to execute, we're ready to advance CSB-001 from a position of strength. The prospect of significantly improving vision with a convenient eye drop is a tremendous advance, and I'm energized by the opportunity to take on a blinding disease like LSCD.” Marc de Garidel, Chairman of Claris Bio, added, “Claris is entering a defining period in its growth. In addition to welcoming Steve and Brian to Claris’ leadership team, I’d like to thank our founding CEO, Clarke Atwell, for his years of diligence in building the team and advancing the science underlying CSB-001, which allows us to approach this watershed moment with confidence. We look forward to continuing our work to advance CSB-001 in patients with limbal stem cell deficiency and lead the company through its next phase as we approach commercialization.” About LSCD and CSB-001 Limbal stem cell deficiency (LSCD) is a disease of the eye in which specialized stem cells that continuously replenish and repair the corneal surface are damaged or destroyed, leading to severe vision loss or blindness. CSB-001 (oremepermin alfa ophthalmic solution) contains recombinant human deleted hepatocyte growth factor (dHGF) as its active ingredient. By promoting corneal epithelial regeneration while modulating inflammation and fibrosis, CSB-001 is intended to address the underlying pathophysiology of LSCD. Clinical data to date demonstrate substantial improvements in visual acuity in LSCD patients treated with CSB-001. If approved, CSB-001 has the potential to become the first pharmacologic treatment for LSCD, providing a reliable, accessible, and scalable non-surgical treatment option. Claris and Kringle Pharma, Inc., a company developing oremepermin alfa for non-ophthalmic indications, have a safety-data sharing collaboration that has enabled a more efficient development of oremepermin alfa. Estimates indicate that at least 30,000 LSCD patients in the U.S. are actively being managed by an eye care professional, although given the lack of diagnosis, the number is likely larger. Ongoing and Planned CSB-001 Clinical Studies Proof-of-Concept Study. An open-label clinical study evaluating the safety and efficacy of CSB-001 eyedrops in 63 LSCD subjects is ongoing. Subjects receive CSB-001 investigational eye drops in either one or both eyes over 20 weeks beginning on Day 0, or at Week 20 after a 20 week observational period. Completion of dosing is followed by an observational period. More information can be found at clinicaltrials.gov (NCT06452316): Pivotal Studies. In 1H 2027, Claris plans to initiate two pivotal studies evaluating the safety and efficacy of CSB-001 compared to vehicle in a total of approximately 400 LSCD subjects. The primary efficacy endpoint will be visual acuity supported by anatomical endpoints. Non-Interventional Study. To prepare for the planned pivotal studies, Claris is currently conducting a prospective non-interventional study in subjects with LSCD. This study will provide insights into the natural history of LSCD and characterize real-world LSCD management patterns, and identify and qualify investigative sites and potential study participants for the planned pivotal studies. More information can be found at clinicaltrials.gov (NCT07636590): About Claris Bio Claris Biotherapeutics is a late-stage biopharmaceutical company dedicated to advancing transformative therapies for sight-threatening conditions where no or limited treatment options exist. With an initial focus on limbal stem cell deficiency (LSCD), a blinding disease, we are developing the first pharmacologic treatment with the potential to provide a reliable, accessible, and scalable non-surgical treatment option, while moving medical management beyond today's palliative care and towards meaningful improvements in vision and ocular surface health. Our near-term plan is to advance our lead therapeutic candidate, CSB-001 (oremepermin alfa ophthalmic solution), into LSCD pivotal studies in the first half of 2027, with a longer-term goal to become a leader in developing new therapies that improve vision and change patients’ lives. Foundational intellectual property for Claris was based on the scientific work of Drs. Reza Dana, MD, MSc, MPH, and Sunil Chauhan, DVM, PhD., both from the Massachusetts Eye and Ear and the Harvard Medical School Department of Ophthalmology. To learn more, visit . Media Contact Jordyn Temperato LifeSci Communications jtemperato@lifescicomms.com

Non-arteritic anterior ischemic optic neuropathy (NAION) is a loss of blood flow to the optic nerve which causes spontaneous and rapid vision loss, for which there are currently no FDA-approved treatments. Given the substantial unmet need for effective treatments in NAION, in October 2025, Dompé was awarded a Commissioner’s National Priority Review Voucher (CNPV) by the US FDA to accelerate a potential Biologics License Application for intranasal nerve growth factor (NGF) in NAION. MILAN & SAN MATEO, Calif.--(BUSINESS WIRE)--Dompé, a biopharmaceutical company with operations in Italy and the US, announced the first patient has been enrolled in a Phase 3 study of cenegermin-bkbj for the treatment of non-arteritic anterior ischemic neuropathy (NAION). Involving more than 130 centers globally, the Phase 3 trial, “Galassia-NAION-301” is a multicenter, double-blind study comparing cenegermin-bkbj against placebo in improving vision loss caused by NAION. “Our NGF R&D program continues to accelerate with our third clinical study of 2026,” said Marcello Allegretti, Chief Scientific Officer at Dompé. “Dosing the first patient in our NAION trial marks a critical step in our mission to unlock the full potential of NGF. By advancing our proprietary intranasal NGF in clinical investigation, we are taking a bold, non-invasive approach that bypasses the blood-brain barrier and has the potential to redefine the standard of care.” NAION refers to a loss of blood flow to the optic nerve which causes rapid vision loss that usually occurs spontaneously. There are approximately 6,000 new cases per year in the US and 10,000-46,000 new cases in the EU per year. The number of cases is growing because of the aging population and rising prevalence of risk factors such as diabetes, hypertension, and other obesity-related conditions like sleep apnea. As many as three out of four patients experience substantial visual impairment, and approximately one in 10 patients experience profound loss of vision and legal blindness. “With no FDA-approved treatments currently available for NAION, there is an urgent need for novel therapies,” said Ahmed Enayetallah, Chief Development Officer at Dompé. “Dompé’s intranasal NGF therapy represents a promising approach, leveraging NGF’s ability to support the survival and repair of optic nerve cells, with the potential to reverse and/or prevent vision loss associated with NAION if diagnosed and treated quickly.” The study will enroll approximately 272 people globally with NAION aged 50 to 80 years. The study endpoints will assess functional and structural changes in the affected eye, as measured by Best Corrected Visual Acuity (BCVA), visual field testing, and Optical Coherence Tomography (OCT). Cenegermin-bkbj is an investigational product for NAION, and its safety and efficacy for this indication have not been established or approved by any regulatory authority. About Dompé Dompé farmaceutici S.p.A. is a privately held, global biopharmaceutical company on a mission to bring the full potential of nerve growth factor (NGF) to patients. As the first company to unlock the therapeutic potential of NGF, Dompé developed the first FDA-approved NGF treatment. Today, our clinical pipeline reflects our commitment to redefining treatments across ophthalmic, neurological and pain-related conditions. Building on 130 years of independence, we are embracing the challenge to transform our science and ourselves by delivering first-in-class, disease-modifying therapies through NGF and other breakthrough molecules to help people improve their sight, support neurological recovery and manage pain effectively. Today, Dompé employs more than 1,100 employees worldwide and maintains a US commercial operations hub in the San Francisco Bay Area. More information about the current Galassia-NAION-301 clinical trial can be found at (NCT07453888) or at: . About Nerve Growth Factor (NGF) Nerve Growth Factor (NGF) is a neurotrophin, a member of a family of proteins essential for the growth, maintenance and survival of neurons, with broad therapeutic potential. NGF was discovered by the Italian neurobiologist Professor Rita Levi-Montalcini and American biochemist Dr. Stanley Cohen, earning them the Nobel Prize in Physiology or Medicine in 1986. In their research, Drs. Levi-Montalcini and Cohen uncovered that NGF plays a crucial role in the proliferation, differentiation, and survival of sympathetic and sensory neurons. Dompé is the first company to unlock the therapeutic potential of NGF, building upon a Nobel Prize-winning discovery to deliver a breakthrough treatment for a rare neurotrophic eye disease. About Non-Arteritic Anterior Ischemic Neuropathy (NAION) Optic neuropathies are disorders caused by damage to the optic nerve, disrupting visual signals from the retina to the brain due to injury, inflammation, poor blood flow, or genetic and degenerative factors. The result is progressive or rapid loss of vision, often affecting central or peripheral sight, color discrimination, and in severe cases, leading to permanent blindness. Non-arteritic anterior ischemic optic neuropathy (NAION) is the most common cause of acute optic nerve injury and optic neuropathy in people over 50. It affects men and women equally, usually starting around age 66. While the exact cause isn’t fully understood, common risk factors include diabetes, high blood pressure, high cholesterol, and smoking. Despite its frequency, and risk of severe permanent vision loss, there is no proven treatment to improve visual outcomes. About the Commissioner's National Priority Voucher (CNPV) Pilot Program The U.S. Food and Drug Administration (FDA) launched the Commissioner’s National Priority Voucher (CNPV) pilot program to accelerate the development and review of prescription drugs and biological products that address critical U.S. national health priorities. Announced in June 2025, the program offers companies the opportunity to reduce standard application review times from 10–12 months to just 1–2 months through a collaborative, multidisciplinary review process. Selected companies receive a voucher that provides enhanced communication with the FDA and a rolling review to facilitate a faster timeline, while maintaining the agency’s rigorous standards for safety and efficacy. The program focuses on transformative therapies that tackle major public health challenges and large unmet medical needs. About Oxervate OXERVATE ® (cenegermin-bkbj) ophthalmic solution 0.002% is indicated for the treatment of neurotrophic keratitis. Important Safety Information: Contact lenses, either therapeutic or corrective, should be removed before applying OXERVATE. Contact lenses may be reinserted 15 minutes after OXERVATE administration. Eye Discomfort, such as eye pain, that can be mild to moderate can occur with OXERVATE. Patients should contact their health care provider if a more serious eye reaction occurs. The most common adverse reaction with OXERVATE (~16%) was eye pain. Other adverse reactions with OXERVATE (1% to 10%) included corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation, photophobia, tearing, and headache. Contacts Media: Guido Romeo – Head of Corporate Communications and Public Affairs +39 349 4154010 / guido.romeo@dompe.com Lyndsay Meyer – Head of US Corporate Communications +1 650 477 9848 / lyndsay.meyer@dompe.com Charlie Gould – Ruder Finn +1 917 626 7968 / Charlie.Gould@ruderfinn.com