Session Recap: Advances in Ophthalmology: Therapies for Saving Sight

Monday at the BIO CEO & Investor Conference featured a 9:30am panel that offered a comprehensive perspective on what is happening in the world of ophthalmology today.  The panel was moderated by Tony Gibney, Managing Director, Leerink Partners.

The panelists were:

  • Jeffrey L. Cleland, PhD, Interim Chief Executive Officer, GrayBug
  • Guy Eakins, PhD, Vice President, Scientific Affairs, BrightFocus Foundation
  • Nouhad Husseini, Vice President, Business Development, Regeneron
  • David P. Southwell, President and Chief Executive Officer, Inotek Pharmaceuticals

Gibney introduced the panelists and asked them to briefly introduce their companies.

Husseini started by talking about Eylea, which was approved for macular degeneration, and rocketed Regeneron to success. This success, however, led to them being thought of as an ophthalmology company by accident. They consider themselves technology platform company whose goal is to focus on unmet needs and innovative therapies, and there happens to be a lot of unmet needs in opthomology.

Southwell spoke about how Inotek is working on new mechanisms for the treatment of glaucoma, which has not seen new treatments since the 1990s. They have an eye drop that targets a subreceptor to lower eye pressure; this drug has gone through phase 1 and 2 studies, and is currently in the middle of a phase 3 program. Inotek hopes the drug will get better efficacy without side effects.

Eakins talked about how BrightFocus funds research for mind and sight diseases like Alzheimer’s, glaucoma, and macular degeneration.  He manages their portfolio of research grants. The organization also focuses on patient advocacy programs, webinars, events, outreach in patient community, and recruitment for clinical trials. He said that if done right, BrightFocus will give the other panelists something new to talk about one day.

Cleland talked about how Graybug is working to develop glaucoma drugs to lower eye pressure and neuroprotection. He discussed working on a competitor to Eylea that will work as well, but will come in a dosage form that lasts 6 months without side effects.

After all the panelists finished introducing themselves and their companies, Gibney identified three major segments in opthomology: 1. Back of eye, which has had the most intense innovation recently; 2. Front of eye, in which there are only 2 mechanisms of actions in last 40 years; and 3. Drug delivery innovations, where new techniques can make conditions druggable.

Eakins said that large number of people have these diseases. Specifically, there are 12 million people in the U.S. with macular degeneration, 4 million with glaucoma, and as a rule of thumb, we can multiply these numbers by 10 to estimate the global disease burden. The surgical treatment for glaucoma has a failure rate of 30-40%. Patients are excited by stem cell therapies and other new therapies that are in trials. He mentioned ciliary neurotrophic factor (CNTF) from Neurotech as an example of a promising treatment for macular degeneration. He also discussed another company using a vitamin A dimerizer in swine models, although he jokingly pointed out that ancient Egyptians rubbed ox liver on eyes to accomplish the same thing, so it’s perhaps better thought of as a forgotten mechanism of action than a new one. Eakins also said that exosomes were an “open frontier,” ripe for exploration. He also said that although he organizations deals a lot with the basic sciences, there is a lot of promise shown in some of data-oriented approaches; querying electronic medical records along with basic science records can help find human health relevance.

Southwell was asked to talk about the regulatory environment and combination therapies. He said that the FDA had taken different approach than European regulators for Timolol. European medicine regulatory authorities agreed to fixed dose combination, but the FDA didn’t approve the combination of drugs, saying that it added side effects without justifying them. In addition, compliance for Timolol eye drops is low; many patients don’t take them because of the red eye side effect. Therefore, there was a need to develop a drug without the side effects. Southwell said that the side effects of a new Inotek drug that is currently in development compares well to placebo. He also pointed out that theirs was the first new eye drop therapy to be allowed to be compared to placebo, rather than compared to Timolol.

Husseini described Eylea as a “miracle drug.” He said that others will have to look at the margins for improvements in meeting the remaining unmet needs. He admitted that injections into the eye are unpleasant and a barrier to compliance, so there is room for improvement (e.g. reducing the frequency of injection). He also pointed out that Eyelea is not a cure, and not everyone responds to it. Still, he says that Eylea has set a “high bar” for others. He said he has seen about 45 presentations showing slides that claim that combining Eylea with another drug will improve treatment. Husseini says his response is to wait and see the clinical data, and that while animal models may be promising, they do not always translate well in human trials.

Cleland then said that getting a needle in the eye, as is required by Eyelea, is a high burden. People frequently drop off after a year, unable to take it anymore, even when the patients know they might lose their vision. Patients only had a 72% compliance rate in a clinical setting, which is sure to be much lower in practice. Patients also risk irreversible scarring from these injections. He says we need a better delivery platform that reduces how many times a year the treatment is needed. He said that there are other promising approaches, such as injection around the eye rather than through the eye, or lenses that you can apply to the eye.

Another major topic of discussion was the gene therapy approach to ophthalmology. Gibney asked Husseini to comment, and he said that one of the reasons that gene therapy has taken off for ophthalmology is the importance of delivery and dosage issues. Gene therapy relies on virus delivery of genes for therapeutic effect. There are still many hurdles to be overcome; producing a virus for gene therapy is no small feat. He points out that although this technology is not new, what is new is that there are big investors in this space who have decided that now is the time to invest in this technology.

Cleland sounded more skeptical on gene therapies, pointing out that what is needed is consistent, high level expression of therapeutic benefits. He said he has not seen data that shows this.

Gibney ended by asking the panel to look into their crystal balls and predict what the next major disease will be to be positively impacted by breakthroughs. Southwell speculated that cancer was a promising field for new developements in ophthalmology, citing a book he recently read called, “The Death of Cancer”. Eakins suggested diabetes may see developments in the next decade. Husseini suggested genetic diseases, due to our rapidly-growing understanding of them. Finally, Cleland suggested orphan disease and dry eye will show promise.

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