Recording Available: Foundation Insights Forum – August 29, 2024

Foundation Fighting Blindness
Insights Forum Transcript
August 29, 2024

Amanda Bement, Development Associate, Admin & Ops:

Hello everyone, and thank you for joining today's Insights Forum. Before we get started, I would like to briefly review a few details for this call. Currently, all participant lines are muted and without video. Please be aware that the controls are at the bottom of your Zoom window. This control bar may disappear when it is not in use, so to prevent the controls from hiding automatically, you can use the following keyboard shortcuts to always show those meeting controls. You can press the Alt key if you are using Windows commands, and if you are using a Mac keyboard, press Command and Backslash at the same time. Today's presentation is being recorded and has closed captioning, which is available by selecting the show Captions option on the bottom toolbar. Please note that on today's call our speakers do have their videos live. However, all of their comments will be provided verbally, and there are no slides. Throughout the call you'll be able to ask questions via the Q&A feature and the chat feature, both of which are at the bottom of that Zoom window.

We'll address these questions towards the end of the call, and if we do not get to your question live, we will follow up over the next week or so. So, please do make sure to include your name in your question. You can also submit a question by sending an email to info@fightingblindness.org. I would now like to turn the call over to our Chief Executive Officer, Jason Menzo.

Jason Menzo, Chief Executive Officer:

Thank you so much, Amanda, and good morning everyone. Thank you for joining us today. We truly appreciate the opportunity to connect with you directly and provide updates on strategic initiatives here at the Foundation Fighting Blindness. And then also to share research and development progress that are happening across the broader inherited retinal disease and dry AMD landscape. We have many updates to share with you today, and a truly inspiring guest speaker towards the end of our call.

But first, let me run down our agenda for today. First off, Chris Adams, our Vice President of Marketing Communications, will share highlights of our outreach and engagement initiatives across the community.

Then Peter Ginsberg, our Chief Operating Officer, will highlight several new corporate partnerships and review our fiscal year 2024 financial performance through the end of June. And then also provide a snapshot of our fiscal year 2025 budget plans and projections.

Next, Dr. Todd Durham, our Senior Vice President of Clinical and Outcomes Research, will share a summary of a new report hot off the presses that summarizes the impact and statistics related to our My Retina Tracker Registry and Genetic Testing Program. Todd will also highlight a recently announced addition to our ongoing Uni-Rare natural history study.

Then following Todd's remarks, Dr. Amy Laster, who's our Senior Vice President of Science Strategy and Awards, will share details about our new five-year science strategic plan, which will span from fiscal year 2025, which just started last month, and go through fiscal year 2029, which is kind of amazing to think about something that's that far in advance. This plan opens a new groundbreaking chapter in our growth as an organization and will help guide our research funding and decision-making over the next five years.

And then finally, to conclude our remarks today, I will focus on the next chapter of the Foundation's Evolution and Growth and introduce our brand new Board Chair, Karen Petrou, who joins us today to share her vision for the future of this great organization. Of course, as always, following Karen's remarks, we will open the call for questions from you all. And in addition to the speakers that I've mentioned so far, we'll also at the Q&A session be joined by Dr. Rusty Kelley, our Managing Director of our venture fund, the RD Fund, and Jeff Klaas, our Chief of Strategy and Innovation. Both of them will be joining us for the Q&A session today. With that, I'd now like to turn the call over to Chris Adams.

Chris Adams, Vice President, Marketing & Communications:

Thank you Jason, and good morning everyone. As part of the Strategy and Innovation team here at the Foundation, we have many compelling activities and initiatives underway to expand our awareness of our mission, increase community engagement and strengthen our fundraising capabilities.

One of this year's highlights was the Visions 2024 conference held in June in Chicago, which brought together over 500 individuals and families affected by blinding diseases. The conference showcased significant advancements in optogenetics, stem cell therapies, genetic treatments, and more. In addition to the scientific sessions, we offered adaptive thriving sessions on technology, mental health, advocacy, and many other vital topics. With 45 exhibitors and inspiring keynote speakers like Sam Latif from Procter & Gamble and Kenyetta McCurdy-Byrd of Research Riverside Development Corporation, the event was a resounding success. Recordings are now available within the VISIONS Conference section of our website at fightingblindness.org, which can be found under the events tab.

We continue to expand our international outreach by bringing a vision seminar to Paris. The event is on Saturday, October 12th, and will feature presentations from IRD researchers in France, as well as foundation representatives. In addition, the foundation will host our second UK Vision Seminar in London on Saturday, November 16th. Planning is in progress for both of these events, so please check out the Foundation's website in the coming weeks for further details. We hope you join us internationally.

Also, coming up is Blindness Awareness Month this October. We want everyone to come together to spread the word about blinding diseases and share their personal experiences with blindness and visual impairments through our social media campaign, Share Your Vision. The campaign invites individuals affected by vision loss to share their story through written word, audio, or video. There will also be special days highlighted during the month, including World Sight Day on October 10th, and White Cane Day on October 15th. Check out our social media pages to celebrate this October with us.

And lastly, we are very excited to roll out a new program called Vision Connection, which is an enriching and supportive gathering for the blind and low-vision community. Each session will consist of educational presentations on a variety of topics, Vision Walk kickoffs, and interactive Q&A sessions with guest speakers. You will be able to share your experiences, gain knowledge, and connect with others in our community. We're asking you to be part of Vision Connection and strengthen our community together. Join us by registering for one of the upcoming sessions at fightingblindness.org/vision-connection. Also, be sure to put that link in the chat. Your participation will make an enormous difference. With that, I will now turn the program over to Peter Ginsberg, our Chief Operating Officer. Peter?

Peter Ginsberg, Chief Operating Officer:

Chris, thank you very much. This is Peter Ginsberg. I wanted to talk first about some of the new corporate partnerships that the Foundation has completed recently. We continue to expand the number of corporate partners that help us fund new important Foundation initiatives and research. And we recently welcomed two new partners that are supporting our My Retina Tracker Registry and Genetic Testing Program. First, Sepul Bio, a new business unit of the leading European eye care group, Théa, is dedicated to developing treatments for IRDs using RNA therapy. Sepul is developing two cutting-edge ophthalmic products recently acquired from ProQR. The first one is sepofarsen, which aims to combat Leber Congenital Amaurosis 10 or LCA10, which is a rare genetic disease causing severe visual impairment. The second program is ultevursen, which is designed to stop vision loss in people with retinitis pigmentosa caused by the mutation in exon 13 of the USH2A gene.

Our other new partner is Octant, and Octant is a drug discovery company developing correctors for protein misfolding diseases. Octant's Navigator platform combines high-throughput, synthetic biology, generative chemistry and artificial intelligence to discover and develop therapeutics against complex cellular mechanisms in human cells. And importantly for us, Octant's pipeline includes a program for autosomal dominant retinitis pigmentosa. So, we're really appreciative of those two new My Retina Tracker Registry and Genetic Testing Partners.

In other partnership news, we began sponsoring genetic testing through PreventionGenetics in July, and that transition is proceeding well. Through our collaborations with PreventionGenetics and InformedDNA, which is our genetic counseling partner for the program, we're able to continue providing genetic testing and counseling at no cost to patients with a clinical diagnosis of an IRD who meet program eligibility criteria. It's a very important program for the Foundation and for our community. We believe supporting patient access to high-quality genetic testing and expanding our My Retina Tracker Registry are critical components in accelerating the development of therapies for inherited retinal diseases.

Now I'd like to provide a brief summary of our fiscal year 2024 financials and our budget outlook for fiscal 2025. Recall that the Foundation operates on the fiscal year that runs from July to June, so we just completed our fiscal 2024 on June 30th. I'm pleased to report for fiscal 2024 our preliminary unaudited financial results indicate that we exceeded our budgeted $13.4 million net fundraising surplus for the year. Overall, preliminarily, we invested more than $30 million in retinal disease research and education in fiscal 2024. Let me repeat that because it's important. Preliminarily, we invested more than $30 million in retinal disease research and education in this last fiscal year. Following our regular annual audit, we expect that our audited fiscal 2024 financial statements will be available this fall on the Foundation's website in the About Us section under financial reporting.

Finally, for fiscal 2025, which is the current fiscal year that we just started, we're targeting $36.4 million in unrestricted revenue against $22.9 million in operating expenses for a $13.5 million net fundraising surplus. I'll now turn the call over to Dr. Todd Durham to discuss the recent developments related to our patient registry and our ongoing natural history studies. Todd?

Dr. Todd Durham, Senior Vice President of Clinical and Outcomes Research

Thanks, Peter. The Foundation, as you know, hosts My Retina Tracker Registry, the largest retinal disease patient database connecting people to relevant clinical trials and natural history studies. Our Registry and related Genetic Testing Program aims to advance research focused on IRDs, while addressing critical barriers to genetic testing and counseling for individuals with IRDs across the United States. Our Registry now has more than 35,000 participants, including more than 23,000 members who have genetic testing information in their profiles. This large cohort of genetically tested individuals has become increasingly valuable as a source for identified research data that can be used to support clinical trial planning and development, estimate disease prevalence, and increase our understanding of the patient journey and more. On that note, we just completed production of the 2024 Registry Community Report, which highlights recent enhancements to our Registry program and includes summary statistics for 15 genetic variants. The data provided for each variant includes the number of participants in the registry with that particular genetic variation, the range of participant ages, the average age of onset and diagnosis, the average impact division, and the usage of glasses or contacts.

The report provides an excellent snapshot of the wide range of Registry members in their inherited retinal diseases based on participants tested in the My Retina Tracker Genetic Testing Program between April 2017 and May 2024. Here are some interesting takeaways. The most common clinical diagnoses are participants who undergo genetic testing through our program are retinitis pigmentosa (45%), Stargardt disease (11%), pattern dystrophy (7%), inherited macular dystrophy (7%), and cone rod dystrophy (6%). Of participants who receive genetic results, the most commonly implicated genes include ABCA4, USH2A, RPGR, PRPH2 and RHO. The Registry supported recruitment for over 35 clinical research studies over the past five years. And while the Registry has already accomplished so much over the last decade, we know there's room for continued growth and enhancements.

Over the next several years we expect to add even more validated surveys to increase the value of our data. We plan to increase the Registry diversity by translating materials into Spanish and working more closely with the Foundation's many community chapters and continue improving our already robust data privacy and security measures. We look forward to sharing this report with our community in the coming weeks. Registry participants will receive the first access to this report and it will also be posted on our website and featured in our social media channels.

One of the most important benefits of participation in the Registry is to help identify patients who may qualify for a clinical study. This includes our natural history studies, which help us to evaluate how a disease progresses, estimate the rate of disease progression, and how variable that progression is among groups of affected people.

The Foundation Fighting Blindness with funding from the Usher III Initiative is launching a natural history study of people with Usher syndrome type 3, known as USH3, caused by mutations in the CLRN1 gene. This four-year study of up to 20 patients with the N48K mutation in CLRN1 is part of the Foundation's Uni-Rare natural history study. We are targeting enrollment of as many as 1,500 patients with a broad range of IRDs associated with more than 300 genes. The Uni-Rare study is evaluating patients at more than 30 clinical research sites in the U.S., EU, and other countries. The Jaeb Center for Health Research is the study's Coordinating Center. For more information about the study, please go to clinicaltrials.gov.

Collaborations like these help us compile more patient data and ultimately provide insights to therapy developers to optimize the design of clinical trials for emerging IRD therapies. By understanding the progression of the disease in a real-world context, treatment approaches can be tailored to meet the needs of those affected. Overall, we are very encouraged by the continued progress in learning more about IRD progression through our Registry and natural history studies, and believe these initiatives will have numerous benefits for our community. I will now turn the program over to Dr. Amy Laster.

Dr. Amy Laster, Senior Vice President of Science Strategy and Awards:

Thank you Todd. Today I'm going to talk about how we fund cutting-edge research into the future. Every five years we launch the next iteration of our scientific strategic plan, which identifies gaps that need to be addressed and milestones that we can reach for in the pursuit of furthering our mission. We just completed the final year of our prior five-year strategic plan, which spanned from July 2020 through June 2024. Over the past nine months or so, we worked really closely with our global Scientific Advisory Board, or we like to call them the SAB, to develop a new plan spanning from July 2024 through June 2029. Our SAB is comprised of 60 of the world's leading retinal disease clinicians and researchers, chaired by Dr. Jackie Duncan, who's a Professor of Clinical Ophthalmology with the Beckman Vision Center at the University of California San Francisco. And each year we ask the Scientific Advisory Board to assess the gaps in priorities, and they're responsible for determining which research grants will be funded. They undertake a very rigorous multi-step evaluation process that typically begins about now, August, or September, and ends May / June.

As part of the development of our new scientific strategic plan, the SAB reviewed the achievements in the field over the last five years and identified key knowledge gaps to inform the Foundation's research plans going forward. These insights were organized around our six research priority areas, which include novel medical therapies, gene therapy, regenerative medicine, cell and molecular mechanisms, clinical structure function, and genetics. We also have a subcommittee specifically focused on age-related macular degeneration, which also conducted a similar assessment.

We compiled these findings to provide the necessary context as our team set the goals and investment levels for the next five years. Our new plan will target the investment of a record $139 million over the next five years. This will include a wide range of research projects and activities for translating preclinical research towards the clinic, funding natural history studies and advancing clinical programs through the RD Fund investment.

Highlights of our plan include:

• a focus on therapy for people with the most advanced vision loss. This includes evaluation of emerging retinal prosthetics such as artificial retinas.
• The development of clinical trial outcome measures that the FDA and other regulators will accept for approval of emergent therapies.
• Another highlight are specific activities leading to an updated commercially available IRD, or Inherited Retinal Degeneration, gene testing panel, which will be inclusive of what we'll learn to be the most current comprehensive and diverse genetic insights for diagnosis and treatment planning for patients.
• We are renaming two of our priority areas. Formerly Gene Therapy will now be Genetic Technologies, and this is to recognize the expansion of technology and editing genes and RNA. And also, Regenerative Medicine will be renamed to Restorative Therapies to include not only cell-based therapies and optogenetics that we've always focused on, but to also bring in retinal prosthetics.

We are ready to embark on this new chapter and we're grateful for the tremendous input and expertise provided by Dr. Duncan and the rest of the SAB to position the Foundation for tremendous future progress and impact.

Before I wrap up, I'd like to briefly mention a few recent science and research developments within our community.

There's a new analysis of data from the National Eye Institute's Age-Related Eye Disease Study, that was recently published in the journal Ophthalmology. The studies, which are referred to as AREDS and AREDS2, which is the acronym for Age-Related Eye Disease Study, show that the AREDS nutritional supplements slow the progression of geographic atrophy, or GA in both early and late stage dry age-related macular degeneration. The AREDS2 supplement, which is available over the counter, contains vitamin C and E, lutein, zinc and copper. Now, as a reminder, always consult your physician when considering a new treatment or a change in treatment.

In other news, Beacon Therapeutics, which is an ophthalmic gene therapy company, recently announced they've dosed their first patient in its Phase 2/3 VISTA clinical trial for AGTC-501. This is an emerging gene therapy for X-linked retinitis pigmentosa, or XLRP. It's caused by mutations in the gene RPGR. Since the RPGR gene is on the X chromosome, males who have only one X chromosome are more severely affected. Females with two X chromosomes can be carriers and may have mild symptoms. So, the VISTA trial will enroll 75 males between the ages of 12 and 50 at the sites in the U.S. with additional sites expected to open in other countries. More information about this trial is available on clinicaltrials.gov.

There are two new clinical trials underway that I also want to talk about. Both of these, they're different trials on testing disulfiram for potentially improving vision in people with retinitis pigmentosa. Clinicians at the University of Washington and Seattle and clinicians also at the University of Rochester Medical Center have initiated clinical trials to test the ability of disulfiram to improve vision in patients with macular degeneration and other forms of photoreceptor dystrophy, who's treated with disulfiram for alcohol abuse disorder. Now, disulfiram also referred to as Antabuse, is an FDA approved medication for deterring alcohol consumption. The preclinical research has indicated that disulfiram not only inhibits enzymes that break down alcohol, but it also inhibits the enzymes that produce retinoic acid, which interferes with the transfer of visual information from photoreceptors to the brain. This may help patients make the most of their remaining photoreceptors, so we'll continue to track these very early studies and keep you informed of further development.

On a final note, two RD-Fund portfolio companies recently received the Rare Pediatric Disease designation from the U.S. FDA. Opus Genetics for their LCA5 program, and Atsena Therapeutics for their X-linked retinoschisis program. As an incentive for companies and investors to focus on rare diseases, this designation qualifies these programs to receive a priority review voucher, or PRV, when their products are eventually approved for commercial sale by the FDA. This is considered very valuable in the industry, since a priority review designation means the FDA's goal is to take action on an application within six months to 10 months under standard review.

In summary, we look forward to updating you on the breadth of research that is occurring globally, and thank you for your attention and support for our science and research initiatives. At this time, I'd like to turn the program back over to Jason.

Jason Menzo, Chief Executive Officer:

Thank you so much, Amy, and I just want to personally thank you, Amy, for all that you've done in leading the development of what you just spoke of, this incredible next chapter of our science programs here, the Foundation, the next five-year Science Strategic Plan. As you can tell, we're all very excited about how aggressive and robust the plan is for the next five years. And this is really an exciting new chapter here of growth at the Foundation. The Science Plan is of course a key part of that vision, as being really the most aggressive and robust funding plan that we've ever undertaken. Just to take a second and put it in perspective, as Amy shared just a few minutes ago, we plan on committing at least $139 million in the next five years towards advancing treatments and cures for blinding inherited retinal diseases and dry age-related macular degeneration. That converts to targeting an investment of about $28 million every year going directly towards research, including our grants and awards, natural history studies, Genetic Testing Program, clinical trials, and of course really importantly, new investments through the Retinal Degeneration Fund.

It is really incredible, and of course, this is only possible due to the generous contributions of many of you who are on this call today, and all of our donors that are supporting this mission from across the globe.

The strategies detailed in our plan aim to build on the momentum of the Foundation's previous work and provide guidance to our team in allocating resources and measuring organizational effectiveness as we go forward. As Amy mentioned, the plan's milestones include a couple really important things.

• Number one, establishing validated clinical endpoints for clinical trials.
• Number two, funding projects aimed at restorative therapies created innovative disease models that'll inform and improve the likelihood of success of emerging clinical programs.
• Publishing regulatory guidance, advancing genetic insights, nurturing our talent in the field of the emerging crop of talented researchers that are coming into this field today.
• Fostering strategic collaborations with other like-minded entities and organizations here in the U.S. and of course abroad.
• And promoting awareness of our important mission to communities, again, both here in the United States and globally.

Another important strategic initiative well underway is the development of the next five-year strategic plan for the RD Fund, the venture arm of the Foundation. The RD Fund, as many of you know, was established in 2018 to be a natural complement to the work of the Foundation, driving promising treatments from the lab and to and through the clinic. The RD Fund operates as a venture philanthropy model, advancing mission-related investments in companies with projects nearing the clinical stage of development, and is led by Dr. Rusty Kelley and a stellar board of internationally recognized experts in our field. And as a quick recap, in its first five years the RD Fund has provided significant clinical returns with dozens of research development, regulatory and clinical milestone achievements. Including from our current portfolio of six clinically staged companies, two companies that are progressing through IND readiness studies, and two entering into pre-IND readiness studies.

Despite the challenges that exist in our funding environment in rare disease and in a greater biotech market as a whole, the Fund is expected to provide significant clinical and financial returns to our mission over the next five years. And as we look to the future, the RD Fund will continue to be a valuable catalyst in funding research and advancing our mission. Under Dr. Kelly's leadership the team is developing and has done an excellent job of a lot of work so far. What does the next five years look like for the RD Fund to continue to build upon the momentum of the first five years, and really continue to drive the mission forward through this unique and really special channel? There's nothing like the RD Fund anywhere else in the world, and we're certainly proud that it's part of our overall strategy to advance our mission.

Another essential way in which we're creating more connections in our community is through our VISIONS Conference, which as many of you know is an every other year conference where we bring the global inherited retinal disease community together. As Chris highlighted a few minutes ago, this year's event, the VISIONS 2024 Conference in Chicago, was our largest ever, bringing together more than 500 members of our community for an educational and truly inspiring program. As we plan for our next VISIONS Conference, we're pioneering a new approach unlike anything that we've ever done before, bringing together VISIONS in conjunction with the Retina International World Congress. So, VISIONS 2026 is going to be a completely different level in terms of bringing together the global inherited retinal disease community in partnership with this amazing organization, Retina International. And for those of you who don't know, Retina International is a global member organization dedicated to fostering research and ensuring universal access to diagnosis, treatment, and care for people affected by retinal degenerative diseases.

As a combined effort we expect to attract as many as 2,000 potential attendees to the Retina International Visions World Congress in 2026, which would make it the largest gathering of its kind in the history of our space. As you can tell, we're very excited about this, and there's a lot more details to come and formal announcements around location, dates, et cetera. But you can plan on it being the summer of 2026 and more details will come out in the next couple of weeks, or months, maybe months.

Finally, I look forward to speaking about an important transition which occurred at the beginning of last month. We've talked a little bit about this on the Insights Forum in the past. And certainly we've been planning and discussing the fact that David Brent was going to be retiring as the Chair of the Foundation after eight years of incredible service to this cause. His significant impact on our mission and our organization and his contributions are truly too many to list. And on behalf of the Foundation and everyone in our community, I want to extend a heartfelt thanks and our gratitude to David for his exceptional leadership and the lasting impact that he has certainly made on the organization.

To facilitate a smooth transition a special committee was formed two years ago and identified our next speaker, Karen Petrou, as David's successor, and that went into effect last month, early last month, July 1st, 2024, Karen became the Chair of the Foundation Fighting Blindness and I, for one am thrilled. I get to work with Karen very closely every day, and I mean that sincerely, multiple times a day. There's so much that she's helping with and bringing so much value into so much that we're doing. Her appointment marks a significant milestone in the history of this organization as she's only the third Chair of the Board in our 52-year history, and she's our first female board chair as well.

She's a seasoned leader and an advocate who brings a wealth of experiences to the chair position. She's personally diagnosed with retinitis pigmentosa when she was 18 and has been a passionate supporter and leader within the Foundation community for decades. And that culminated in her joining the Board back in 2006. She's also professionally in her day job, as she likes to say, although I kind of think that the Foundation's becoming more like her day job, because she's spending an incredible amount of time advancing this mission. But in her day job, she's the co-founder and managing partner of Federal Financial Analytics, which is a firm providing analytical and advisory services on legislative, regulatory, and public policy issues affecting the financial services industry here in the U.S. and abroad. And her extensive experience both in the financial services industry and her deep personal connection to our mission truly makes her the ideal leader for this next chapter and the organization's growth.

She served as Executive Vice Chair of the Board for the last year, working closely with me, with David, with the Board of Directors, and she's all set up and making impact obviously, as you can tell, from day one. And with that said, it is my absolute pleasure to introduce and turn the call over to our new Board Chair and today's guest speaker, Karen Petrou.

Karen Petrou, Board Chair, Foundation Fighting Blindness

Thank you so much Jason, and hi to everyone on this call. I have to say that if this were the first time I had ever thought to learn a bit more about the Foundation, the presentations by Jason and the staff team would get me really psyched. But of course, it's not the first time, and I'm thrilled to be leading the Foundation on what Jason sometimes calls FFB 3.0 after Gordon's, immense contribution, Gordon Gund, many of you know. And David, as Jason has rightly said. And even if I weren't psyched and thrilled by all of that, going to the VISIONS Conference in June really was exciting. It was fabulous to meet so many members of the community focused on treatments and cures for themselves, for their kids, for all of us, as well as ensuring that those of us with vision impairment continue to lead full, successful, and loving lives.

I really want us to focus on treatments and cures in the context of a positive understanding that while we work hard for the vision that I know I miss and all of us want, that doesn't mean we have to hold back. We can and we will each of us find our way in this difficult time. And FFB is very focused on that as well as our truly ambitious scientific mission. I won't repeat what Todd and Amy said or what Rusty is going to say, Rusty Kelley about the venture capital fund we have, which is, as Jason said, groundbreaking.

But I do want to highlight several key initiatives. One, as Todd said, is the patient registry, the genetic registry. We've done this for years. It's critical. As Peter pointed out, it's vital to companies in our space, because with rare diseases makes it hard to find rare people, particularly with some of the smaller indications in our field, and we have to have this registry.

But we are conscious that the Registry is not fully inclusive, and we are working very hard to ensure that populations such as persons of African descent are fully incorporated, not just in the Registry, but also in our clinical trials. Also, people of different ages, different demographics, other key criteria, so our clinical trials not only reflect the community as a whole, but frankly have a much better chance of success.

The FDA is, thanks to a lot of the hard work that Todd and his group are doing, is beginning to better understand that regaining or preserving vision doesn't just mean a line on an eye chart. It can be a much more subjective and complicated thing to measure. And for our clinical trials, we have to persuade the FDA and other global regulators to understand how complicated vision is. And that what a sighted person might think is not much is a lot to each of us dealing with vision loss or fearing it.

Amy mentioned we're really focused on late-stage restoration. That's not because we weren't before. I want to really emphasize, this is something we've always known we needed to do, but we've moved it up and we've made it more of a priority, because there's a lot of very exciting work in this area. Vision prostheses, yes, things that artificial intelligence is opening up in a new way remarkably quickly, and other potential treatments and cures across our broader populations of patients on a gene agnostic and even late-stage basis. That's going to be vital and I think it's going to make a huge difference in the field and then ultimately to many, if not all of us.

There's a very exciting initiative we can't talk much about, but I just want to get to you, watch your computers, watch your phones. FFB is poised, we're the last stages of being asked to lead a truly exciting effort by the U.S. federal government for a revolutionary new eye treatment.

This is going to make global news. It's extremely exciting, and that FFB is likely to be the group chosen to lead this effort is truly, truly an impressive testament to the hard work of all of the staff and all of you.

As Jason said, none of this comes cheap. It’s vital as we're getting towards some of the late stage treatments and cures, not just for vision restoration, but for much else, we're facing a serious problem. The private sector funding essential to moving the literally hundreds of millions we need to move treatments and cures to approval is harder and harder to get. That makes the work of our Venture Philanthropy fund and our partnerships with private sector entities even more important than ever before. But we can't do it without you, because this is all philanthropic funding. It's a unique model. It's been successful, but it's even more urgent now than it's ever been.

We do have challenges, but we have an enormously talented staff, and thanks to all of you, a great community. I am totally committed to transparent leadership. We've taken a lot of steps internally in the last year to open some key aspects of our governance up, but I am excited to be here for all of you. You can find me through the foundation. You can find me on LinkedIn or X or Twitter or whatever you want to call it. And I will answer any questions you have, or take any concerns you may offer to heart and deal with them as quickly and as thoroughly as I can. Thank you all for everything. This is a thrill for me personally and truly an honor, and I can't wait to report back to all of you about some more of the progress I am positive we'll be making over my term as Chair and in years to come. Thank you all.

Jason Menzo, Chief Executive Officer:

Thank you so much, Karen, and everyone who has not heard you speak prior to this call certainly has a jolt of energy and excitement now the way that we all as a staff feel by getting to work closely with you every day. I think it's clear the impact that you will absolutely make leading as Board Chair over the next several years of the foundation to this next chapter, FFB 3.0. Love it.

We're at the time, we've got about 17 minutes available now for Q&A. We've already got probably a dozen or so questions that have been chatted in. For those who have chatted in questions using the Zoom interface, if it says question answered live, that just means it's in the queue to be answered live, so don't be alarmed. Before we start answering questions, I'm going to turn it back to Amanda just to briefly remind our audience how they can ask questions, and then we'll jump in.

Amanda Bement, Development Associate, Admin & Ops:

Thank you, Jason. There are several methods that you can use to answer or to ask questions. You can submit them through the Q&A or the chat features, both of which are at the bottom of your Zoom window. And do make sure to include your name please, so that we can follow up afterwards. And you can also send an email to info@fightingblindness.org and we will follow up in the next week or so through there as well.

Jason Menzo, Chief Executive Officer:

All right, thank you very much. I want to invite my co-hosts, Peter, Jeff, Rusty, Todd, for everyone come off mute and we'll go ahead and start the Q&A session now. One thing just in setting this up, which actually speaks to the state of our field. Many, many of the questions that came in, I would argue more than ever before in an Insights Forum were about the status of a specific program within a specific company. And if you think about our mission and where we were even just a few years ago, that was a rarity because there were so few companies that had clinically staged or disclosed programs in our space. That's different today. Today there are many companies that are working on treatments and cures in our space, which is exciting. Of course, that brings with it a unique challenge, because sometimes there's information that's confidential and can't be discussed publicly.

But to that end, I want to pose to Rusty, and we've spoke about Rusty quite a few times on this call already, but Rusty leads the RD Fund as the Managing Director, and he's got his finger on the pulse on much of what's happening across not only the portfolio of the RD Fund, but really across the IRD landscape. And so Rusty, I just want to pose to you just a few specific company updates, just what's available publicly, because of course we can't disclose confidential information, but the latest from what's known publicly at Nanoscope, Endogena, Ray Therapeutics, jCyte, there were questions about each of these companies. And so I figured I would just throw it to you to speak what's available publicly across all of them.

Dr. Rusty Kelley, Managing Director of the Retinal Degeneration Fund

Thank you, Jason. Rusty Kelley here, I think it is Jason, Amy and Karen have articulated very nicely is that the Foundation is experiencing a new five-year strategic plan that does include renewal strategies. Of course, we've been paying attention and interacting with many, if not all, of the companies that are going after renewal strategies, including optogenetics, cell-based therapeutics and/or pharmaceutical approaches to activating stem cell approaches to photoreceptor regeneration. You mentioned Endogena, Ray Therapeutics, Nanoscope. Just to be clear, the RD Fund has not participated in the financing of these companies. And it's also important, just to reiterate Jason's point that we are investing in for-profit companies, and we certainly have the responsibility as careful investors not to disclose information that's not in the public domain, and nor do we want to interpret data not to mislead anyone with active trials.

So Endogena, this is a company that's investigating a pharmaceutical approach for potentially activating endogenous or native stem cells to differentiate into functional photoreceptors for the treatment of RP and GA, geographic atrophy. This company is currently engaging the regulators on an end of Phase 1/2 trial for RP, and I believe they may have a trial for Stargardt disease as well. I've noticed just in the last couple of minutes online that there isn't much publicly disclosed information on this company. So, we'll just have to wait for this company to tell the public what it's doing as it engages regulators for approval to move to the next step. But we do know that they've reported safe outcomes from their first in human trial.

Ray Therapeutics and Nanoscope, these are both optogenetic companies developing therapeutics for RP and GA as well, and Stargardt disease. In the case of Nanoscope and optogenetics, I know we use a lot of technical terms in this field, but optogenetics uses gene therapy to express light sensing genes in the retina. Ray Therapeutics is at the stage of development that's evaluating non-clinical efficacy and safety and models of disease before they apply to the FDA or other international regulators for the clearance to begin a first in human trial. So stay tuned for those key regulatory steps for getting into the clinic.

And then Nanoscope, they are at the end of a Phase 2/3 trial, and they're also engaging the regulators on next steps. And I'm certain it's their hopes to seek registration, but this remains to be determined. And Ray Therapeutics, Nanoscope, there are many other optogenetic companies out there that are promising. But most of them are early stage and or early clinical, so we'll just have to wait for this field to play out.

Jason Menzo, Chief Executive Officer:

Great. Well, thank you very much, Rusty. And just to preview for the audience, in one of the future Insights forums, I'm certain we'll have Rusty as a guest speaker, really talking about the next five years with the RD Fund. As I mentioned in my remarks, the work that Rusty and his colleagues on the RD Fund board and staff have been putting into developing the next five-year plan for the fund has been immense. And I think that the audience will really appreciate hearing from your voice what the view is over the next five years. So, that'll be a future Insights Forum.

I want to come to you, Amy, next. Actually, one of the first questions that was emailed from Paul Levenson was sending on behalf of his deaf blind wife, who I assume is affected with a form of Usher syndrome. And I thought it would be good, Amy, for you to just give a state of play on the landscape around potential treatments for Usher syndrome, recognizing there's various subtypes. And then also, some of the narrative in his question was around prosthetics or other forms of technology, which obviously is now in scope for our portfolio. I have another question for you about Stargardt immediately after, but why don't you start with Usher syndrome?

Dr. Amy Laster, Senior Vice President of Science Strategy and Awards:

Thank you, Jason. Again, this is Amy Laster. So, there are both pre-clinical and clinical studies that are exploring different therapeutic strategies for Usher syndrome and RP related to visual impairment and Usher syndrome. These include gene editing strategies, gene therapy, those are more pre-clinical, as Jason just mentioned, restorative therapies like optogenetics. Rusty just talked about those as well. And as we begin to really focus on what are therapies for late-stage disease, retinal prosthetics or visual prosthetics are encompassing of that. And so, this may again have benefit for patients, even those with Usher syndrome and RP due to Usher syndrome, to have benefit when they're in late stage of disease from these types of visual prosthetics. This is something that we're talking to experts globally about things that are already in play and things that are coming on the horizon, so please stay tuned.

We'll certainly talk more about this as we begin to delve more in how the Foundation can be impactful in this area. Also, it's just worth noting, and I know Todd shared this earlier in his comments, but the Foundation has partnered with different Usher organizations launching various natural history studies for the different types of Usher syndrome. Todd spoke about the Usher three initiative, but we've had natural history studies and for Usher2A, Usher1B, Usher1F. The data that's gleaned from these studies, they will inform clinical trial design, patient recruitment for clinical trials for any emerging therapies. Dr. Kelley, do you want to talk about some specific activities for Usher syndrome from the company space? I know you've already touched on some of these.

Dr. Rusty Kelley, Managing Director of the Retinal Degeneration Fund

Thanks, Amy. Happy to do it. I think Usher syndrome represents one of those cornerstones to the Foundation, as Amy nicely pointed out. We have a lot of activity with Usher, including the various funding strategies that are led by Dr. Angela Bowman at the Foundation, the natural history studies, as Amy mentioned, that Todd Durham leads, and of course, we're happy to report that the RD Fund, just to be specific to the RD Fund activities. For the sake of this question, we have two companies that are active in Usher. One of them is Atsena Therapeutics. They have a gene augmentation strategy for rescuing variants associated with the USH1B gene that is currently in IND readiness studies or these non-clinical studies, as I mentioned earlier, that allow you to approach the regulators for clearance for a first in human study.

And then we have Nacuity Pharmaceuticals. This is a clinically-staged company that's developing an antioxidant. It's a small-molecule strategy for addressing Usher related to RP. And it's currently being evaluated in a Phase 2 clinical trial in Australia with two-year data expected to be reported on next summer.

And then we also had an investment in ProQR, as Peter Ginsberg mentioned earlier, Sepul, one of our new partners with the registry is developing ultevursen, a genetic approach for targeting exon 13 of the USH2A gene. And this is a program they licensed from ProQR, as Peter mentioned. And we have a significant investment in ProQR for advancing those earlier clinical trials. So, lots of exciting clinical activity at the RD Fund.

Jason Menzo, Chief Executive Officer:

Great. Thank you, Dr. Kelly. Thank you, Dr. Laster, thank you for the questions. There's more. There's many, many more. We've got about six minutes left, so we're going to move rapidly. I want to bring Peter and Jeff, a couple of questions to both of you. Peter, first, there was some news this week from Ocugen, I'm wondering if you could speak to that. And after that, Jeff, questions around our Visions Connections program that Chris spoke of. So, I'll bring that to you in just a second. But Peter, why don't you start with the news from Ocugen this week?

Peter Ginsberg, Chief Operating Officer:

Well, I'll say first of all, the Foundation was encouraged that there are several new therapeutics in late-stage clinical development for inherited retinal diseases and dry AMD. One of those is OCU400 from Ocugen. And Ocugen announced in June that it had dosed its first patient in its Phase 3, what they're calling liMeliGhT trial for OCU400. And for that Phase 3 trial that's dosing patients right now, the plan is to test 150 RP patients, and that will be 75 participants with RHO gene mutations and 75 participants that are gene agnostic, so more broad-based RP patients. That study is ongoing right now. They announced just this week that they've expanded the study to include Canada. So, looking forward to that study continuing and seeing the results of that study.

Jason Menzo, Chief Executive Officer:

Thank you, Peter. Jeff, so question was chatted then about our new Vision Connection forums that we're going to be having in certain markets. Two parts to the question. One, maybe just highlight where folks can find information about where those are going to be and what markets we're going to be having those. And then what if you're not in one of those markets? How can you plug into that type of resource?

Jeff Klaas, Chief Strategy & Innovation Officer:

Thanks, Jason. And thank you for the question. This is Jeff Klaas, our Chief Strategy and Innovation officer. The question around Vision Connection is really around programs that are specifically focused on enriching and supporting the gathering of our community. And so what you can expect from those programs are, as Chris highlighted, educational presentations, community engagement, fundraising opportunities, and ways to really create moments of engagement and experience for those who are affected by blinding diseases. Now, one of the ways to be able to connect with us on that certainly is to go to our website, investigate some of the information, which I believe is under Vision Connection. And the second would be to register specifically. And if you don't have an opportunity to engage in a community where you have physical presence or other community members are available with an official Foundation Fighting Blindness chapter, I'd encourage you to reach out directly to us and respond a lot of the ways that we connect with our communities as we look for volunteer leaders and we can connect others in meaningful ways, which allow them to receive those benefits even in a virtual manner.

Jason Menzo, Chief Executive Officer:

Thank you, Jeff. Listen, we're running up against the clock. I want to hit a couple really important questions. One, so many of the questions this time, but always around clinical questions. Should I be taking this supplement? Should I be doing this, should I be doing that? And want to be clear that the Foundation Fighting Blindness, while being the global leader in driving the research to provide preventions and cures for blinding retinal diseases, we aren't a clinical provider, so we don't give clinical advice or medical advice. We strongly encourage you to get in touch with an inherited retinal disease specialist, which could be a low vision ophthalmologist, could be a retinal focused ophthalmologist, and anyone who is not sure in their local market how to access the right type of specialist, contact us at info@fightingblindness.org and we can make a recommendation based on your zip code and where you are. I know that many questions have been chatted in, and that's the best way for us to be able to play a role in connecting you with a clinical expert in your local market.

Okay, couple quick hits as we wrap things up. Hi, Sherry Rogers who asked about the Registry. Todd, briefly, how does someone get into the Registry and what if someone has AMD and not an IRD, what's the role of AMD in the My Retina Tracker Registry?

Dr. Todd Durham, Senior Vice President of Clinical and Outcomes Research

If you want to enroll in the Registry, the easiest thing to do is go to myretinatrackerregistry.org. You can find a link for it on our company website. And there you fill out your information, sign the informed consent. The short answer about AMD is the Registry is primarily focused for individuals with an inherited retinal disease, but we do have, we can accommodate raising your hand to say you have either dry AMD or wet AMD or both. I also wanted to say, it'll be important to do that, if you're so willing, we do have requests for information with people with AMD, and we have even in the last year sent out several requests for information from those individuals. So you can be contacted about research opportunities if you register with AMD.

Jason Menzo, Chief Executive Officer:

Thank you, Todd. Rusty, last question. It's been chatted like six times in the last four minutes, so I want to make sure I give you a chance just at a very high level, the status of jCyte and their previously publicly announced next steps with jCyte and their next clinical trial?

Dr. Rusty Kelley, Managing Director of the Retinal Degeneration Fund

So again, just being careful what is and what isn't disclosed. I don't have all of that committed to memory, but I can tell you that jCyte is in a similar spot as Nanoscope where they are packaging up their data with a new manufacturing strategy, which is obviously one of the critical linchpins of success in advanced therapies, including cell-based therapy, is to make sure that you can appropriately manufacture a candidate product with great purity. And so, they're making great strides in that regard and engaging regulators, again, to assess the evaluation of an analysis of their Phase 2 data to see if they can move forward into a registrational trial.

Jason Menzo, Chief Executive Officer:

Thank you, Rusty. It is just a minute after noon here on the East Coast. Just some final remarks from my desk. Number one, just to reiterate the comments earlier, and as Rusty has shared, we're engaged in, I would say, nearly every company that's pursuing clinical programs or certainly within the scope of our mission. However, it doesn't mean that we're able to disclose confidential information, so puts us in a tricky spot sometimes. But everything that's publicly disclosed on anything in our space is certainly available on those companies' websites. So if it's on the company's website, then it's obviously been publicly disclosed. And another really important thing that we do is that news in our space. We have great stories on our website. We have quick alerts that happen via our podcast. Ben Shaberman does an exceptional job of interviewing leaders in our field on the Eye on the Cure Podcast, we have the clinical trial pipeline on our website that denotes the clinical status of every program in our space.

And of course, we have links to clinicaltrials.gov and things of that nature. So we're engaged in the entire field, but sometimes it's tricky because there's information that is not publicly disclosed, and so we're not permitted, honestly, from a legal standpoint in disclosing things that are not publicly disclosed by the company. With that said, what a fantastic engagement today. Several hundreds of folks that were here live through the call, which is great. Obviously, several thousand that'll listen to it on replay as soon as it gets posted immediately after the call. But it is a few minutes after 12 now, so we've reached the end of our time. Certainly we're energized by the tremendous support of the community. Encourage you, please get involved locally, if you're here in the United States connecting with a chapter. If you're in Paris, we've got our VISION seminar, Chris referenced, that's happening late this fall.

If you're in London, we've got the VISION seminar we discussed that’s happening later this fall. Certainly you can engage with us on social, online, et cetera. We always welcome feedback and suggestions related to this webcast or anything related to the foundation. And you can reach us anytime directly by sending an email to info@fightingblindness.org. And of course, a great resource is fightingblindness.org website, which changes daily. There's so much new information we post nearly every day. And of course, if you have any questions, don't hesitate to reach out to me directly. I'm going to turn it over to Chris Adams to wrap things up for the call.

Chris Adams, Vice President, Marketing & Communications:

Thanks everyone, and again, thank you for joining us for today's call. As Jason mentioned, there will be a transcript and recording available on our website, fightingblindness.org, within the next week. And for the latest information outside of our website, you can follow us on social, on Facebook, Twitter, LinkedIn, Instagram, TikTok, and Threads. If you have any questions or need any further information, please send us an email at info@fightingblindness.org. Thank you, and have a great day.