Five ophthalmology biotechnology companies to watch out for

There are hundreds of different eye diseases, with some of the most common major eye diseases including age-related macular degeneration (AMD) – an age-related eye disease that can cloud central vision – glaucoma – a condition in which the optic nerve becomes damaged – and diabetic retinopathy (DR). – a complication of diabetes in which high blood sugar levels damage the retina.

While some eye diseases are treatable, others are sorely lacking in effective treatment options, and there are no medications available that can reverse the effects of certain conditions.

There are many ophthalmology biotechnology companies focused on developing better treatment options for different eye ailments. In fact, in 2021, ophthalmology-focused startups in Europe had a record year for venture capital funding, with continued success well into 2022, and it seems major pharma has set its sights on the ophthalmology opportunity lately.

Here, in alphabetical order, we have listed the top five ophthalmology companies focused on creating therapies for various eye ailments.

Azura Ophthalmic

Azura Ophthalmics aims to create treatments for eye conditions by leveraging keratolytic properties, which are compounds typically used to break down the disulfide bonds that bind keratin into aggregates to treat skin conditions such as acne, seborrheic dermatitis, and psoriasis.

However, because abnormal keratin production, deposition, and aggregation are pathognomonic for many ocular surface diseases, Azura is developing a portfolio of keratolytic agents for the treatment of eyelid margin disease. and related conditions.

The ophthalmology company has a pipeline of new chemical entities, and its prime candidate is AZR-MD-001, for the treatment of meibomian gland dysfunction (MGD). MGD is a common condition that occurs when the meibomian glands don’t produce enough oil, called meibum, or poor quality oil, and is a common cause of dry eye syndrome and blepharitis.

Last year, Azura announced positive results from its phase 2b AZR-MD-001 trial for MGD, with the trial meeting the shared primary endpoint of increased meibomian gland producing fluid secretion and Ocular Surface Disease Index score compared to vehicle.

Bree’s Therapy

With the goal of addressing an unmet medical need to develop therapies that are more effective and less burdensome for patients and caregivers, Breye Therapeutics is developing new ophthalmology drugs that are orally administered to patients with vision loss. The focus is on DR and AMD.

To treat this condition, ophthalmology companies are developing danegaptide – a small molecule drug – as an oral therapy that targets retinal pathologies, to reverse the causes of retinal vascular disease. Danegaptide has a mode of action that protects against detachment of cells, retinal capillary damage, and vascular leakage caused by conditions of high glucose stress.

Last year, Breye Therapeutics closed a €4 million ($4.1 million) seed funding round, led by Novo Holdings and Sounds Bioventures.

EyeBio

Ophthalmology company EyeBio raised $65 million in series A funding last year to develop a new generation of eye therapy that will protect, restore, and enhance vision.

EyeBio recently announced details about Restoret, which is the company’s key asset in its pipeline of multispecific agonist antibodies. Restoret is a tri-specific agonist antibody that targets the Wnt pathway, and is designed to address the unmet medical need in patients with back of the eye disease. Wnt signaling in the retina plays a central role in the maintenance of vascular integrity, with defects in Wnt signaling leading to retinal vascular leakage.

Restoret is given by intravitreal injection to specifically treat retinal diseases characterized by leakage, including neovascular age-related macular degeneration (NVAMD), diabetic macular edema (DME), and familial exudative vitreal retinopathy (FEVR).

EyeBio plans to apply for a clinical trial authorization to enable Global Restoret’s phase 1b/2 clinical trials later this year.

Oxurion

Formerly known as ThromboGenics, ophthalmology company Oxurion’s focus is developing treatments for diabetic eye disease. The prime candidate is called THR-149, for the treatment of DME.

THR-149 is licensed from Bicycle Therapeutics and is a bicyclic peptide that selectively inhibits human plasma kallikrein (PKal) – a mediator of vascular leakage, inflammation, microbleeding, and neurodegeneration. By inhibiting the kallikrein-kinin system (KKS), it prevents the induction of retinal vascular permeability and inflammation.

Oxurion recently announced it has completed its target of enrolling 108 patients in a randomized phase 2b trial called KALAHARI, which aims to evaluate THR-149 for the treatment of DME against the current standard of care, aflibercept.

If the trials yield positive results, it could mean that THR-149 may provide an alternative for DME patients who respond less optimally to current anti-VEGF therapies.

SpliceBio

Last year, Spanish company SpliceBio was able to raise €50 million ($52.7 million) in oversubscribed series A financing, which will allow the company to concentrate on bringing its main program for Stargardt disease into phase 1 clinical trials.

Stargardt’s disease is a rare genetic eye disease that occurs when fatty material builds up in the macula – which is the small central part of the retina – and causes vision loss, especially in children and young adults. The disease itself is caused by a change in one gene called the ABCA4 gene.

However, the ABCA4 gene is too large at 6.8 kb for a single adeno-associated virus (AAV) vector to pack enough genetic material to treat it, because an AAV vector can pack only 4.7 kb of genetic material.

In an effort to overcome the capacity limitations presented by the AAV vector, SpliceBio seeks to harness the potential of inteins, which are a family of proteins that carry out a process called protein splicing, linking peptides together to form new proteins. By leveraging this process, company co-founders Miquel Vila-Perelló and Silvia Frutos were able to develop split inteins engineered for therapeutic use.

The biotech company is now aiming to find a treatment for Stargardt’s disease and other indications in ophthalmology – as well as other therapeutic areas – through its Protein Splicing platform.