AAO 2024: An update from Neurotech on its MacTel treatment candidate

Thomas Aaberg, Jr, MD, Neurotech's Chief Medical Officer, shared some insights on the company's recent presentation as well as their upcoming presentations at the annual American Academy of Ophthalmology meeting being held in Chicago, Illinois.

Video Transcript:

Editor's note: The below transcript has been lightly edited for clarity.

Sydney M. Crago: I'm Sydney Crago with Modern Retina and Ophthalmology Times, and we're here today with Dr Thomas Aaberg Jr, MD, to discuss NT-501 from Neurotech. Neurotech has made significant strides recently, especially with their presentations at Retina Society, EURETINA and the upcoming AAO meeting. Dr Aaberg can you walk us through these developments?

Thomas Aaberg Jr, MD: Hi Sydney, yes, thank you for having me. It's been an incredible few months with a lot of pivotal data coming to light at Neurotech, our focus is on Macular Telangiectasia Type 2, which is, as many people know, a rare progressive bilateral neurodegenerative disease of the central retina, the macular region. And the recent presentations from Retina Society and EURETINA as well as the upcoming ones that we are going to be presenting at AAO showcase, the hope of NT-501, encapsulated cell technology in both efficacy and safety.

Sydney M. Crago: Now you've returned from Retina Society and EURETINA. There are several key presentations that were delivered there. Can you summarize the main findings of these meetings?

Thomas Aaberg Jr, MD: We had a series of important presentations across both events that highlights the different aspects of NT-501's performance. And at the Retina Society, we had Tom Albini led the discussion on long term NT-501 CNTF delivery, where we saw over a decade of continuous ciliary neurotrophic factor, CNTF, release from the NT-501. So this suggests that we can offer patients more sustained therapy without frequent intraocular injections or repeated surgical interventions, and for a progressive disease like MacTel, where ongoing neuroprotection is critical, the ability to maintain this consistent treatment over a long period of time can potentially be a game changer for these patients.

Dean Elliot presented our bilateral safety data. So one of the critical challenges with MagTel is that it of course, impacts both eyes. So there's always a question about safety when you're treating bilaterally. The safety data generated from this phase 2 bilateral study is encouraging, as it suggests patients can be treated in both eyes without any additional risks.

And then Tony Capone shared our post-hoc efficacy data from our pivotal phase 3 trials. And what stood out in Tony's presentation was the identification of specific baseline characteristics that predicted a better outcome when you got the NT-501. For example, patients with a smaller baseline ellipsoid zone area loss, so they had less photoreceptor loss at baseline. Second younger age, less than 65 years of age, specifically. And lack of foveal involvement in the disease progression all showed a better response to treatment. The earlier you intervene, before the disease has progressed too far, could potentially lead to more favorable outcomes for patients and this is important for physician guidance on how to recommend use of NT-501, should it become approved.

EURETINA was another great platform for sharing additional data. Some of the key presentations included one by David Lally. He presented an audio free paper on the same bilateral safety study, and his findings mirrored those of Dean Elliott's at the Retina Society, confirming that the safety profile of NT 501, when implanted in both eyes was positive and we didn't see any additional safety signals. Catherine Egan also presented an audio narrated free paper on the pooled clinical trial safety analysis. Now her data provided a comprehensive look at NT-501's safety profile across our phase 1, our phase 2 and our phase 3 studies. Showing a very consistent safety record in a broader patient population as far out as 108 months or 9 years. This pooled analysis offers the, to date, broadest foundation for understanding ND-501's safety.

Finally, Barry Cooperman presented at the podium the post-hoc efficacy analysis from our phase 3 studies, which again focused on the responder status. His presentation highlighted how different patient subgroups responded to NT-501, and reinforced the importance of patient selection for optimizing outcomes in understanding which patients are likely to have the best response.

Sydney M. Crago: As we approach AAO in 2024 what can we expect from Neurotech?

Thomas Aaberg Jr, MD: First, we're excited that we're going to be able to present. We have several key presentations lined up that will build further on our data that we've shared at Retina Society and EURETINA. So Roger Goldberg is going to be presenting the pooled functionality data in the late breaking development session at retina subspecialty day. And this provides a broad view of how NT-501 impacts overall visual function, you know, across different functional endpoints for both our phase 2 and our 2 phase 3 studies, Emily Chu is going to be presenting again, the phase 3 study data for NT-501 specifically focusing on the drug's ability to to slow the progression of MacTel type 2. Charlie Wykoff will be presenting data from our extension study data. So this is a phase 1 and phase 2 extension study. This presentation looks at long term safety and efficacy beyond the initial trial periods, and it provides insight into how NT-501 continues to be safe for up to 9 years.

Sydney M. Crago: We're very much looking forward to those presentations at the meeting. Where does Neurotech go from here? What's the next big milestone for NT-501

Thomas Aaberg Jr, MD: Our next big milestone is our PDUFA goal date, which currently is December 17. If we receive approval, it'll mean that we can begin to offer this innovative therapy to our MacTel patients. But of course, we have to get through December 17, and then we can talk a bit more expansively about our future plans.