Oculis Strengthens Pipeline by In-Licensing Neuroprotective Drug Candidate for Glaucoma

Oculis Strengthens Leading Ophthalmology Pipeline by In-Licensing Neuroprotective Drug Candidate for Glaucoma from Accure Therapeutics


  • First-in-class peptidomimetic with distinct MoA activating trophic signaling pathways to protect and prevent damage to the optic nerve and retina
  • Potential to be disease modifying in lead indications such as glaucoma, geographic atrophy, acute optic neuritis (AON) and other optic neuropathies
  • Proof of concept (PoC) clinical trial in progress for AON with results due 2H23
  • Agreement supports Oculis’s mission to become a global ophthalmology leader bringing breakthrough innovations to the top three segments of the market: retina, dry eye and glaucoma
  • Deal validates Accure’s strategy as a translational R&D engine in neuroscience


Lausanne, Switzerland, and Barcelona, Spain, March 2, 2022 – 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 Pitié-Salpétrière 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.”




About Oculis

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. 

To learn more 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, Parkinsons 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/


Oculis Media & Investor Relations

SPRG (China / Asia)
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