Oculis S.A., (‘Oculis’) a global ophthalmology company developing life-changing treatments to save sight and improve eye care with breakthrough innovations, announces that the Phase 2 DX-211, a randomized, double blinded, multi-center and vehicle controlled clinical trial assessing the safety and efficacy of topical OCS-01 in patients with Diabetic Macular Edema (DME) (NCT05343156) has been published by the Acta Ophthalmologica journal. The publication is accessible on the National Institutes of Health (NIH) website here ( https://pubmed.ncbi.nlm.nih.gov/35848336/ ). In addition, a new subgroup analysis of this study was also recently presented by Pr R. Tadayoni at the 22nd EURETINA Congress on 2nd September in Hamburg, Germany.
— Positive Phase 2 data, published in journal ACTA, provides the first proof-of-concept for a topical drug effect in diabetic macular edema (DME)
— Dataset shows that OCS-01 eye drops were more effective than vehicle in reducing central macular thickness and improving visual acuity in patients with DME as per the pre-defined criteria for statistical superiority in the study protocol
— Subgroup analysis, presented by Pr Ramin Tadayoni at EURETINA, suggests even greater improvement in patients with lower baseline visual acuity
— If approved, OCS-01 has the potential to provide a new and potentially the FIRST non-invasive eye drop option for DME patients
The dataset published shows OCS-01 to be superior to vehicle in the primary efficacy analysis of BCVA change from baseline with a between group difference of +1.58 ETDRS letters at the pre-specified significance level of 0.15. Mean CMT showed a statistically significant greater decrease from baseline in the OCS-01 group compared to vehicle at all post-baseline time points up to and including Week 12 (-54micrometer OCS-01 vs. -17micrometer vehicle) and reversed towards baseline when treatment stopped at 12 weeks, demonstrating the pharmacological effect of OCS-01 on retinal edema. OCS-01 was well tolerated and no significant or unanticipated ocular adverse events were observed.
Subgroup analysis in patients with BCVA baseline is less than or equal to 65 letters, presented at EURETINA 2022, showed greater improvements in both CMT (-77.4micrometer OCS-01 vs. -23.1micrometer vehicle) and BCVA (+2.9 ETDRS letters in OCS-01 vs vehicle) at week 12.
Approximately 37 million people are affected by DME worldwide, representing around 7% of the large and growing diabetes patient population. DME is the build-up of fluid (edema) in the macula (the macula is important for the sharp, straight-ahead vision that is used for reading, recognizing faces, and driving). DME is the most common cause of vision loss among people with diabetic retinopathy and a leading cause of new cases of blindness in US adults. Although treatment options currently exist, all of them are invasive (injections or implants) and represent a significant burden for patients and caregivers.
OCS-01 is a novel, high concentration, preservative free, topical formulation of dexamethasone that has the potential to be the first topical eye drop and non-invasive treatment for DME. OCS-01 opens up the possibility of treating DME patients at all stages of disease with an eye drop. In addition, OCS-01 could increase the accessibility to treatment of those patients without easy access to specialists; together, this could significantly reduce the burden to the health care system and improve patient outcomes and quality of life.
OCS-01 is currently under investigation for the treatment of DME in the ongoing Phase 3 DIAMOND trial to confirm these findings in a larger patient population.
Riad Sherif, M.D., CEO of Oculis, said: “We are delighted by these data from the Phase 2 DX-211 study, which further reinforce the potential of OCS-01 to provide patients with a potentially transformative treatment of DME as the first non-invasive, eye drop, option. An effective topical treatment for DME could expand the proportion of patients treated, especially those with recent onset DME, as well as provide a treatment option to general ophthalmologists prior to referral to a retina specialist and thereby, improving access to treatment. We look forward to the results of the ongoing Phase 3 DIAMOND trial as Oculis seeks to deliver life-changing treatments that can be accessed globally for the world’s most prevalent ocular diseases.”
Pr Ramin Tadayoni, MD, PhD, Professor of Ophthalmology at Universite de Paris, said: “The development of effective topical eye drops for posterior segment disorders such as DME would offer easier, non-invasive options than those currently available, and could be widely adopted across all socioeconomic and geographic boundaries. This would allow earlier treatment in DME, reduce the burden on both patients and their caregivers with potentially less injections per year if combined with current standard of care, increase adherence and ultimately, improve outcomes worldwide. OCS-01 is the most developed and promising topical therapy for DME to date, and I look forward to its further development as a potentially transformative treatment for patients in need.”
Oculis is a global biopharmaceutical company purposefully driven to save sight, improve eye care and address significant unmet medical needs with breakthrough innovations. Oculis’s highly differentiated pipeline includes candidates for topical retinal treatments, topical biologics and disease modifying treatments. With a presence in key international markets, Oculis is poised to deliver life-changing treatments to patients worldwide. Headquartered in Lausanne, Switzerland and with operations in Europe, the U.S. and China, Oculis is led by an experienced management team with a successful track record and supported by leading international healthcare investors.
For more information, please visit: www.oculis.com
Topic: Press release summary
Oculis S.A., (‘Oculis’) a global ophthalmology company developing life-changing treatments to save sight and improve eye care with breakthrough innovations, announces that the results of the double blinded, multicenter and placebo controlled Phase 2 clinical trial assessing the effect of topical licaminlimab (OCS-02) on global ocular discomfort in patients with severe dry eye disease (DED) (NCT02365519) has been published by the Clinical Ophthalmology journal. The publication is accessible on the National Institutes of Health (NIH) website here. ( https://pubmed.ncbi.nlm.nih.gov/35821785/ )
The results from the study show that the change from baseline to Day 29 in the global ocular discomfort score, the primary efficacy endpoint, was statistically significantly greater for topical ocular licaminlimab (OCS-02) (-7.9) than for vehicle (-3.6) (90% CI -7.7, -0.8; p = 0.041). The percentage of patients with an improvement in global ocular discomfort score >20 from baseline to treatment day 29, one of the main secondary efficacy endpoints, was statistically greater for licaminlimab (17.9%) compared to vehicle (4.7%) (p=0.018).
Licaminlimab (OCS-02) was well tolerated in this study, with no major safety differences between licaminlimab (OCS-02) and vehicle treatment groups, and no increase in intra-ocular pressure was observed.
Licaminlimab (OCS-02) is a single-chain antibody fragment (scFv) that binds to and neutralizes the activity of human TNFa, with dual mechanism of action (MoA), anti-inflammation and anti-necrosis. Unlike full-length monoclonal antibodies, scFv fragments can penetrate ocular surface tissues when used as eye drops, due to the smaller size of the molecule giving it the potential to become the first approved topical biologic for DED.
Dry Eye is a multifactorial disease in which inflammation rapidly takes on a central role in sustaining the pathological state(1). The global prevalence of DED has been reported at 11.59%(2) , representing approximately 900 million people worldwide. In the US alone, there is currently between 16 million and 49 million people who have dry eye disease(3) . Significant unmet medical needs remain for this large and growing patient population with only 9% of diagnosed patients in the US receiving treatment(4) and despite current options, only 13% of patients are achieving lasting relief(5) .
Licaminlimab (OCS-02) is currently being investigated by Oculis in Phase 2 clinical trials for the treatment of dry eye disease and uveitis.
Pr Christophe Baudouin, M.D., Professor of Ophthalmology and Chairman of Ophthalmology III in Quinze-vingts National Ophthalmology Hospital, Paris, commented: “There is a true unmet medical need for the development of drugs with new MOA to treat the inflammation involved in the pathogenesis of DED. OCS-02 is certainly one that could fill that gap. As compared to currently available treatment options and other product candidates in the DED pipeline, OCS-02 could play a role in the treatment of the underlying causes of the disease given its anti-inflammatory and anti-necrosis benefits. I certainly look forward to the continued development and approval of OCS-02 in severe dry eye disease as these patients currently have limited treatment options.”
Riad Sherif, M.D., CEO of Oculis, said: “The Phase 2 data further reinforce our confidence in the potential of OCS-02 as a novel anti-inflammatory treatment for the effective management of Dry Eye Disease, but also for other inflammatory eye diseases. With Phase 2b clinical trials of OCS-02 for the treatment of dry eye disease and uveitis which are planned to start in the coming months, along with on-going Phase 3 clinical trials of OCS-01 in diabetic macular edema and inflammation and pain following ocular surgery and a proof-of-concept (POC) trial of OCS-05 in acute optic neuritis, we look forward with confidence to the further development of the Oculis Ophthalmology Franchise to address significant unmet medical needs in key areas of ophthalmology.”
Oculis is a global biopharmaceutical company purposefully driven to save sight, improve eye care and address significant unmet medical needs with breakthrough innovations. Oculis’s highly differentiated pipeline includescandidates for topical retinal treatments, topical biologics and disease modifying treatments. With a presence in key international markets, Oculis is poised to deliver life-changing treatments to patients worldwide. Headquartered in Lausanne, Switzerland and with operations in Europe, the U.S. and China, Oculis is led by an experienced management team with a successful track record and supported by leading international healthcare investors.
For more information, please visit: www.oculis.com
(1) Baudoin C. Dry Eye Disease, the complex interactions of vicious cycles. EuDESEuropean Dry Eye Society https://www.dryeye-society.com/resources/dry-eye-disease-complex-interactions-vicious-cycles
(2) Eric B Papas “The global prevalence of dry eye disease: A Bayesian view” 2021
(4) DRG Dry Eye Disease Landscape and Forecast 2020
(5) Mukamal, R. Why is Dry Eye So Difficult to Treat? 2021 https://www.aao.org/eye-health/tips-prevention/fix-dry-eye-treatment-eyedrops
Topic: Press release summary
Oculis S.A., (Oculis) a global ophthalmology company developing life-changing treatments to save sight and improve eye care with breakthrough innovations, and Accure Therapeutics, a private translational neuroscience R&D company, today announced a licensing agreement granting Oculis exclusive global rights to develop and commercialize ACT-01, a potentially disease-modifying therapy to protect and prevent damage to the optic nerve and retina.
ACT-01, which is being renamed OCS-05, is a first-in-class small molecule with neuroprotective activity that has shown positive results in animal models of neuroinflammation and neurodegeneration. Its mechanism of action is related to the activation of the trophic factor pathways such as IGF-1 and BDNF. In ophthalmology, this action can protect the nerve axons in conditions where the optic nerve is affected, such as in acute optic neuritis and glaucoma, where the OCS-05 could prevent chronic vision loss.
Based on positive preclinical data and results of a phase 1 safety and PK study in healthy volunteers, a phase 2a study was initiated (the ACUITY study).
ACUITY is a two-arm, randomized, double-blind, placebo-controlled, monocentric study to evaluate the safety and tolerability of OCS-05 compared to placebo in patients with acute optic neuritis. In addition to safety, secondary outcome measures will include optic nerve anatomical measures, as well as visual function measures. The study is ongoing at the La Pitie-Salpetriere hospital in Paris within the neurology-ophthalmology network of the Public University Hospital Group in Paris (APHP). One third of the planned sample size has been enrolled.
Oculis is currently planning the expansion of the program in ophthalmology working with the regulatory agencies in the US, EU and China amongst others.
Riad Sherif, M.D., CEO of Oculis, said: “We are excited about this agreement as it combines Oculis’s ophthalmology expertise with Accure’s unique neuroprotective approach to help transform the treatment of neurodegenerative diseases in ophthalmology. At Oculis, our focus is on building a highly innovative and differentiated pipeline providing life changing treatments for ocular unmet medical needs, and OCS-05 is a perfect fit for that ambition. Glaucoma is a leading global cause of irreversible blindness, and despite IOP lowering treatments, a significant proportion of patients still go blind. With OCS-05, we have the potential to bring to market the first neuroprotective for glaucoma and other optic neuropathies.”
Laurent Nguyen, M.D., Co-founder and Chief Executive Officer at Accure Therapeutics, said: “Having shown promising early data and potential in a number of ocular disease indications, the timing is right for a partnership between two like-minded companies committed to patients and driven by a passion for neuroscience. By leveraging its expertise in ophthalmology, Oculis has the potential to fulfill the promise of this exciting asset in a wide range of neurodegenerative diseases.”
In reference experimental animal models of acute optic neuritis (acute inflammatory demyelinating disorder of the optic nerve) and high-pressure glaucoma (high eye pressure damaging the optic nerve and leading to permanent vision loss), OCS-05 reduces damage to the optic nerve and retina. It also decreases paralysis progression in an autoimmune encephalomyelitis animal model for multiple sclerosis (inflammation caused by the body’s immune system, which destroys nerve cell processes and myelin in the brain and spinal cord).
Data from a completed phase 1 study show the safety and tolerability of single and multiple doses of OCS-05 in healthy volunteers.
Under the terms of the agreement, Accure Therapeutics will receive an upfront payment, potential milestone payments upon the achievement of certain development and commercial milestones, and tiered royalties on sales.
Anthony Rosenberg, Chairman of Oculis’s Board of Directors, said: “The addition of OCS-05 to Oculis’s pipeline is very much in line with the Company’s strategy to develop transformative therapies that address the root cause of ocular disease to improve patients’ sight and quality of life. There is a clear need for first-in-class therapies that can protect and prevent damage to the optic nerve and retina. Oculis has the team and expertise to bring OCS-05 through clinical trials and ultimately to patients around the world.”
Montserrat Vendrell, Chairman of Accure Therapeutics and Partner at Alta Life Sciences added: “This licensing deal with Oculis is a proof-of-concept of the value creation that Accure Therapeutics brings as a translational R&D engine, taking forward truly unique science that spun-off from the University of Barcelona. This licensing agreement will enhance Accure’s capabilities to accelerate and expand value potential for the rest of its pipeline.”
Oculis is a global biopharmaceutical company purposefully driven to save sight, improve eye care and address significant unmet medical needs with breakthrough innovations. Oculis’s highly differentiated pipeline includes candidates for topical retinal treatments, topical biologics and disease modifying treatments. With a presence in key international markets, Oculis is poised to deliver life-changing treatments to patients worldwide.
Headquartered in Lausanne, Switzerland and with operations in Europe, the U.S. and China, Oculis is led by an experienced management team with a successful track record and supported by leading international healthcare investors.
For more information, please visit: www.oculis.com
About Accure Therapeutics
Accure Therapeutics is a private translational neuroscience R&D company. Based in Barcelona (Spain), it was launched in 2020 with a Series A funding led by Alta Life Sciences Spain I and supported by the Centre for Technological and Industrial Development (CDTI). This European company with an international mindset boasts a unique portfolio of three new chemical entity programs pursuing innovative targets – with potential to accommodate others. Accure aims to develop new disease modifying drugs to treat serious conditions such as optic neuritis, multiple sclerosis, Parkinson’s disease and epilepsy. With an experienced business and scientific team, Accure Therapeutics is one of the few companies that operate in an agnostic fashion on initial science to deliver cutting-edge drugs in CNS.
To learn more visit https://accure.health/
Topic: Press release summary