Exonate, an early stage biotechnology company, announced today that it has entered into a strategic collaboration agreement with Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson. Through the collaboration, Exonate will work with Janssen Research & Development, LLC scientists to develop an eye drop treatment for retinal vascular diseases such as wet AMD and DMO by using mRNA targeted therapies. Exonate has developed small molecules that inhibit the production of pro-angiogenic vascular endothelial growth factor (VEGF) through the selective inhibition of serine/threonine-protein kinase (SRPK1)-mediated VEGF splicing. The agreement was facilitated by Johnson & Johnson Innovation.

Commenting on the announcement, Dr. Catherine Beech CEO of Exonate, said: "I am absolutely delighted to enter this strategic collaboration with Janssen, we are looking forward to successfully developing a novel treatment for retinal neovascular diseases."

About Exonate:

Exonate is a privately held, early stage, biotech company spun out of the University of Nottingham that is focused on alternative splicing of Vascular Endothelial Growth Factor (VEGF) in ophthalmology. Exonate's lead programme is focused on Diabetic Macular Oedema (DMO). A consequence of diabetic retinopathy, DMO, is swelling in an area of the retina called the macula and wet Age-Related Macular Degeneration (wAMD), which is the leading cause of vision loss in people aged 60 and older.  The Company is founded on scientific excellence with strong links to Professor David Bates and his lab at Nottingham University specializing in the biology and biochemical pathways of VEGF splice variants.

Exonate has developed small molecules that inhibit production of pro-angiogenic VEGF through selective inhibition of serine/threonine-protein kinase 1 (SRPK1)-mediated VEGF splicing.  These inhibitors have already demonstrated superior efficacy as topical agents in preclinical models of wet AMD.  Through a Wellcome Trust funded project, Exonate will complete regulatory toxicology and safety pharmacology studies to support an application to the regulatory authorities for clinical evaluation.  Exonate expects to reach this milestone and enter the clinic in mid-2020.

Exonate is led by an experienced, international management team that has previously worked together with cross-disciplinary experience in medicine and drug development, as well as successful fundraising for early stage companies.

About Diabetic Macular Oedema (DMO)*:

DMO is the build-up of fluid (Oedema) in a region of the retina called the macula. The macula is important for the sharp, straight-ahead vision that is used for reading, recognizing faces, and driving. DMO is the most common cause of vision loss among people with diabetic retinopathy. About half of all people with diabetic retinopathy will develop DMO and although it is more likely to occur as diabetic retinopathy worsens, DMO can happen at any stage of the disease.

About wet Age-Related Macular Degeneration (wet AMD):

Today, wet AMD is a leading cause of vision loss in people aged 60 years or older and affects more than 30 million patients worldwide, over 200,000 of those in the UK alone. If untreated patients are likely to lose sight in the affected eye within 24 months of disease onset.

The main currently available treatment options for DMO and wet AMD are:

• anti-VEGF antibody drugs – to prevent the growth of new blood vessels in the eye. Unlike small molecule drugs or eye drops these treatments must be injected into the eye once every 1 or 2 months. Resistance can develop to these drugs causing the disease to progress anew.
• Laser surgery – to destroy abnormal blood vessels in the eye. This type of surgery is only suitable if blood vessel damage is not too extensive and if the abnormal blood vessels aren't close to the fovea, as performing surgery close to this part of the eye can cause permanent vision loss.
• With DMO, Corticosteroids either injected or implanted into the eye, may be used alone or in combination with other drugs or laser surgery to treat DMO.

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