Recording Available: Insights Forum | Thursday, December 5, 2024

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Foundation Fighting Blindness
Insights Forum Transcript
December 5, 2024

Chris Adams, Vice President, Marketing & Communications:

Hello everyone, and thank you for joining today’s Insights Forum. I am Chris Adams, the Vice President of Marketing and Communications here at the Foundation. Before we get started, I would like to briefly review a few details for today’s call. Currently, all participants’ lines are muted and without video. Please be aware that the controls are at the bottom of the Zoom interface. This control bar may collapse when it’s not in use, so if you prefer to prevent the controls from auto-hiding, you can use the following keyboard shortcuts to toggle the Always Show Meeting Controls options. If you’re using Windows commands, press the Alt key. If you’re on Mac, press Command and Backslash at the same time.

Today’s presentation is being recorded and is available with closed captioning. To activate the closed captioning, please select the ellipsis or three dots located at the bottom right of the Zoom window. Then select Captions or Show Captions. Please note that on today’s call, our speakers do have their videos live. However, all their comments will be provided verbally, and there are no slides. Throughout the call, you will be able to ask questions via the Q&A feature and the Chat features, both at the bottom of the Zoom window. We will address these questions toward the end of the call. If we do not get to your question live, we will follow up with you over the next week, so please make sure to include your name with your question. You can also submit questions 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:

Well, thank you so much, Chris, and good morning everyone. Thank you for joining us today. We are very excited to provide so many updates on our progress here at the Foundation Fighting Blindness and in the broader ophthalmic and inherited retinal disease community.

For today’s call, our agenda will include first, Peter Ginsberg, our Chief Operating Officer, who will start us off recapping several recent announcements from companies working to develop treatments for inherited retinal diseases. He will also review the final financial numbers from our fiscal year 2024 performance and provide an update on our fiscal year 2025 performance through September. As a reminder, our fiscal year goes from July to June, so we’re just through the first quarter of our fiscal year 2025.

Following Peter’s remarks, we’re very pleased to have Dr. Amy Laster on the call today to summarize our recently announced annual science funding awards. I’m also very excited to share that last month, Dr. Laster was named as the Foundation’s interim Chief Scientific Officer. Amy has been a valuable member of our team for 16 years, and we’re super excited about the impact that she will have in her new role.

Next, Jeff Klaas, our Chief Strategy and Innovation Officer, will highlight some of the innovative initiatives that we’re launching to strengthen our connections to the blind and low vision community globally and to fuel the funding for the growth of our future research investments. Then after Jeff, I will come back on the call to provide several strategic updates, including a snapshot of our next five-year strategic plan for the RD Fund, our venture philanthropy fund.

As a very special feature for today’s call, we are welcoming back my predecessor as CEO here at the Foundation and our good friend Dr. Ben Yerxa, who will highlight the recent strategic transaction that occurred last month with Opus Genetics. It’s really great to have Ben back here as a guest on the call. Many of you know it was actually Ben’s vision more than five years ago to create the Insights Forum as a means to provide Foundation and industry updates on a regular basis to all members of our community. So we’re excited to hear updates from Ben a little later in the call.

Then following Ben’s remarks, we will open the call as we always do for questions and answers, and in addition to our speakers that I just mentioned, we’ll be joined for the Q&A session by Chris Adams, who you heard just a moment ago, our Vice President of Marketing and Communications, Dr. Todd Durham, our Senior Vice President of Clinical and Outcomes Research, and Dr. Rusty Kelly, who’s the Managing Director of the RD Fund and really leads all aspects of the fund. They’ll be joining us for the Q&A session a little later in the call. With that said, let’s get things started. I’m going to turn the call over to our Chief Operating Officer, Peter Ginsberg.

Peter Ginsberg, Chief Operating Officer:

Thank you Jason, and good morning everyone. This is Peter Ginsberg, and I’m pleased to report on a number of corporate and clinical advances today. The recently announced merger of our RD Fund portfolio company Opus Genetics with Ocuphire Pharma is a key milestone as it results in substantial funding to drive forward Opus’ inherited retinal disease gene therapy pipeline. It also represents the first RD Fund portfolio company to enter the public markets. The combined company is named Opus Genetics and is traded on the NASDAQ Exchange under the highly appropriate ticker symbol IRD, which stands for Inherited Retinal Disease.

Opus’ most advanced program is a gene therapy for Leber Congenital Amaurosis 5, or LCA5, which causes significant vision loss at a young age. The company plans to enroll pediatric LCA5 patients in the trial during the first quarter of 2025. Additionally, Opus is developing early stage IRD gene therapies for people with mutations in the following genes: BEST1, RHO, RDH12, MERTK, NMAT1, and CNGB1.

Another development related to one of our RD Fund portfolio companies, Atsena Therapeutics and Nippon Shinyaku have entered into a license agreement for the commercialization of an emerging gene therapy for Leber Congenital Amaurosis 1 or LCA1, which is caused by mutations in the gene GUCY2D. Under the terms of the agreement, Nippon Shinyaku will receive exclusive rights to commercialize this gene therapy in the U.S. and Japan, and Atsena will retain commercial rights for the rest of the world. Importantly, a Phase 3 global clinical trial of the Atsena gene therapy is planned.

In addition to these acquisitions and licensing transactions, there have been several companies advancing therapeutic candidates for a range of retinal diseases. BlueRock Therapeutics received authorization from the US FDA to launch a phase I/II clinical trial for its photoreceptor cell therapy for people with IRD such as retinitis pigmentosa and cone-rod dystrophy. This cell therapy was licensed from Fujifilm and Opsis Therapeutics, which is a company co-founded by Dr. David Gamm, who is a world-renowned retinal cell therapy pioneer at the University of Wisconsin and who also recently received significant funding from the Foundation for the development of retinal and photoreceptor cell therapies derived from induced pluripotent stem cells. Also, I should note that BlueRock is an important Outreach partner of the Foundation and is also a supporter of the Foundation’s natural history studies, as announced earlier this year.

Also, Kiora Pharmaceuticals has received authorization in Australia to launch a Phase 2wo trial for its photo switch, which is a small molecule that’s designed to restore some vision to people with advanced retinal diseases. The Phase 2 trial will enroll 36 patients with ultra-low vision or no light perception from advanced RP, and that trial is expected to begin in 2025. Early funding enabling this advancement was through the Foundation’s translational research program and a Gund Harrington Scholar Award to Dr. Richard Kramer from UC Berkeley for his research of photo switches for restoring vision. Kiora has a strategic partnership with Thea for the development and commercialization of this therapeutic.

Additionally, the Usher 3 Initiative, which is a non-profit dedicated to treatments for Usher 3, launched a Phase 1 clinical trial in Perth, Australia to evaluate the safety and tolerability of an emerging oral drug known as BF-844. This trial is the first for an Usher 3 syndrome treatment, and the Foundation Fighting Blindness funded early studies to prepare for this trial through our translational research program. Also, the Usher 3 Initiative and the Foundation recently announced a partnership to launch an USH3 natural history cohort within our UNI-RARE natural history study.

Finally, Avantgarde Bio recently dosed the first patient in its Phase ½ retinal gene therapy clinical trial for people with Usher Syndrome type 1B, which is caused by mutations in the gene MYO7A. The trial is expected to enroll 15 patients aged 18 to 50 in Naples, Italy with two additional sites for the Phase ½ trial in the UK to be announced. I got to say it’s really inspiring to track all of these developments across the world for inherited retinal diseases and dry AMD, and we look forward to the data that will be generated from this clinical testing.

Now moving on to our financial update. We completed our fiscal 2024 on June 30th, and our audited financial statements are now available on our website in the About Us section under financial reporting. For fiscal 2024, our unrestricted net fundraising revenue was $38.6 million, and operating expenses were $20 million, yielding a net fundraising surplus of $18.6 million, which significantly exceeded our expectations, thanks to a special $5 million campaign gift. The combination of that surplus with funds previously committed means that the Foundation was able to invest more than $30 million in retinal disease research and education in our fiscal 2024.

For the current year, which is fiscal 2025, we’re targeting $36.4 million in unrestricted revenue against $22.9 million in operating expenses for a $13.5 million net fundraising surplus. For the first quarter of fiscal 2025 that ended in September, our results were on track with our budgeted plans for the quarter.

This is a very busy time of year for the Foundation for fundraising, and as part of preparing for this important giving season, we continue to enhance ways for you to connect with the Foundation on a more personal level, including for those who prefer to reach out by phone. We recently have added a well-trained internal customer service team that’s available to take calls and personally help connect community members to available resources.

I’m now pleased to turn the call over to Dr. Amy Laster. As Jason mentioned, Amy is our interim Chief Scientific Officer, and Amy’s going to provide a research update. Amy.

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

Thank you, Peter. Today I’m pleased to share with you that the Foundation just announced 35 new research grants totaling $19.8 million in our fiscal year 2024. This investment, which is the largest ever for the Foundation in a single fiscal year really underscores our commitment to advancing treatments and potential cures for retinal degenerative diseases that affects millions worldwide.

The Foundation’s rigorous review process, which is led by our Scientific Advisory Board consisting of over 60 top scientists and clinicians from around the globe, really ensures that each funded project has the potential to deliver transformative insights and advancements in retinal disease treatment. This year’s grants support a diverse portfolio now of 100 research initiatives across 85 laboratories and clinical research institutions globally, driving progress in treatment approaches from gene agnostic therapies to those targeting specific genetic causes such as retinitis pigmentosa, Usher Syndrome, Leber Congenital Amaurosis, Stargardt’s disease, and dry age-related macular degeneration.

Within this year’s funding, the Foundation has renamed the Translational Research Acceleration Program to the David Brint Translational Research Program in honor of David Brint who served as the Foundation’s board chair from 2016 to 2024. Under his leadership, we expanded our clinical development efforts, including the 2018 launch of the RD Fund. In our fiscal year 2024, nearly $9 million was awarded for new grants for the David Brint Translational Research Program, supporting eight projects developing treatments inclusive of gene agnostic approaches for retinitis pigmentosa, Leber Congenital Amaurosis and Usher syndrome.

I want to highlight just a few of the new programs from this banner funding year, and I encourage you to visit our website to learn about all 35 projects. First, to our clinical studies portfolio through our clinical innovation awards we received the strongest pool of applicants to date. Each of this year’s recipients have projects to develop better or reliable methods to evaluate disease progression and to determine a therapeutic effect in a clinical trial. This aligns with our current strategic priority to drive research that establishes clinically meaningful IRD endpoints. Additionally, aligned with our goal to fund research to create disease models for IRDs and dry age-related macular degeneration that will propel potential treatments from the research phase to the clinic, we’re funding multiple programs to develop or characterize non-rodent models of disease for EYS, Usher 2C, Crb1, and Choroideremia.

Finally, toward our strategic goal for talent recruitment and development of the next generation of research scientists that’s focused on retinal diseases, we awarded funding to seven clinicians who are at early or mid-stage of their research career development. Their projects collectively address both basic science and therapy development for IRDs and dry AMD.

As these awards indicate, there is a diverse range of research that’s taking place in both academic settings and in the clinic, and really much of the recent progress that we’re talking about on this call today has grown from the research funded directly by the Foundation Fighting Blindness. This is where our actions translate into impact.

I’d like to highlight one other exciting announcement that we made this week. The Advanced Research Projects Agency for Health, also known as ARPA-H is an agency within the US Department of Health and Human Service. They’ve awarded the University of Colorado and their medical campus along with the Foundation and six other research groups up to $46 million to support research and innovation in vision-restoring human whole eye transplantation.

This federal program called THEA, Total Human Eye-allotransplantation Advancement, has been established by the ARPA-H to further develop a combination of medical, therapeutic and surgical technologies to advance whole eye transplantation as a cure for blindness in people with ocular-based disease or damage, including inherited retinal diseases.

The Foundation in our role has assembled a multidisciplinary team of renowned scientists, researchers, and physicians at multiple research institutions. This consortium, which is led by the University of Colorado Anschutz and includes Johns Hopkins University, University of Wisconsin-Madison, Indiana University School of Medicine, University of Southern California, Cedars-Sinai Medical Center, and the National Eye Institute.

The Foundation will run strategic point for the consortium and serve the central function of ensuring progress maintains alignment with the program’s requirements and mission, with the Foundation’s Senior Director of our Preclinical Translational Research program, Dr. Chad Jackson, overseeing the six-year project.

With that, I’d like to turn the program over to our Chief Strategy and Innovation Officer, Jeff Klaas.

Jeff Klaas, Chief Strategy and Innovation Officer:

Thank you, Amy, and good morning everyone. This is Jeff Klaas speaking. As Amy and Peter have described, the breadth and the strength of our expanding research portfolio is inspiring. It’s a testament to the important commitment of our donors, our volunteers, researchers, and other key stakeholders. Our Strategy and Innovation team here at the Foundation has many compelling activities and initiatives underway to expand awareness of our mission, cultivate stronger relationships with you, our valued global community members, and forge strategic partnerships to explore novel avenues for fundraising.

You can be a part of these efforts. Just this week we deployed our Giving Tuesday campaign, part of the Global Generosity Movement, unleashing the power of radical generosity. The good news is that you still have time to participate and amplify your donation. Every dollar you give to the Foundation will be matched with additional funds committed by Foundation donors, and this is an amazing opportunity to maximize your impact and come together to advance the life-changing research ending blinding diseases.

In October, we also celebrated Blindness Awareness Month. Throughout the month, we encouraged community members to come together to share their personal experiences with blindness and visual impairments through our social media campaign “Share Your Vision.” Our goal this year was to reach 3 million individuals and to encourage them to share their stories using the hashtag #ShareYourVision. I’m pleased to report that we exceeded our target, reaching 3.7 million people through this campaign. It was truly inspiring to feature the many unique and inspiring stories shared by individuals around the world.

In addition to increasing community outreach interactions, we’ve also expanded ways to support the Foundation’s mission by diversifying our giving platforms. As part of our commitment to embracing new technologies, we’re making it easier for donors to contribute through donor-advised funds, stock donations, and cryptocurrency. To efficiently offer these options, we formed a partnership with a Giving Block, a leading organization that equips nonprofits with innovative fundraising tools and techniques. We’ve streamlined our donation process with a simple widget that provides donors with a seamless way to support our mission. Additionally, this expansion helps us reach new donors around the world who share our mission of finding treatments and cures for blinding diseases.

In addition to accepting donations of stock from donor-advised funds, we’re also embracing the future of philanthropy by accepting cryptocurrency donations. Donors can now make contributions through FightingBlindness.org using a wide array of digital currencies such as Bitcoin.

In addition to encouraging direct contributions, one of the other primary ways that we leverage the power of community network is through local fundraising events. We’re halfway through our fiscal year and we’ve completed nearly 30 events across the United States, including VisionWalks, Night for Sight dining events, and Scramble for Sight golf events.

We’re also continuing our growth globally. Over the past two months, we’ve hosted Vision Seminars in Paris and London with more international activities planned for 2025. If you’re interested in being an international ambassador, expanding our global reach, programmatic fundraising and mission work, we are looking for volunteers, so please reach out to us at info@FightingBlindness.org or go to our website and connect with our team to learn more about this exciting opportunity.

There’s one final special event this weekend that I would like to highlight and encourage you to attend. We’re hosting a virtual National Chapter Webinar this Saturday, September 7th at 12:30 pm Eastern to provide a more detailed update on the latest advancements in clinical trials for blinding diseases. Our experts will share the most recent research findings and clinical trial updates, and then we’ll have a patient perspective panel to highlight the significant impact these trials can have on the fight against blinding diseases. Whether you’re a patient, a caregiver, or a healthcare professional, this webinar will offer valuable information, so please join us on Saturday and encourage your friends and family interested in eye health research to participate.

The key message here is to get involved. You can participate in our in-person or virtual events, use our newly adopted formats to volunteer, engage in our growing community properties or donate via new efficient platforms. All of these expanded engagement opportunities are crucial in advancing research into treatments and cures for retinal diseases.

Thanks to the passion and hard work of our community, we are poised to move more therapies into clinical trials and across the finish line.

I’m now pleased to turn the call over to our CEO, Jason Menzo.

Jason Menzo, Chief Executive Officer:

Thank you, Jeff, and as everyone who’s on this call can hopefully tell, there is significant momentum across the board advancing our mission. It really is an exciting time with so much progress. Every time we do this Insights Forum, I get re-energized and re-enthused. It’s terrific to see all the questions that are already coming in and all the chat, and just as a community, it’s wonderful just to hear these advances and know that collectively we’re moving the mission forward.

One of the ways that we’re doing that is by having a very specific and intentional strategic plan. Last quarter on this call, we highlighted the Foundation’s new Five-year Science Strategic Plan, which is the most aggressive and robust funding plan we’ve ever undertaken as an organization, and as a reminder, we’re 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.

Another important strategic initiative that our team has been working on in parallel is the development of the next Five-year Strategic Plan for the RD Fund, and as many of you know, because we talk about it on the Insights Forum pretty regularly, the RD Fund is the venture arm of the Foundation and it’s led by Dr. Rusty Kelly, who will be joining the call in a few minutes for the Q&A.

The RD Fund was established in 2018 to be a natural complement to the work of the Foundation, driving promising treatments from the lab into and through the clinic. The RD Fund operates as a venture philanthropy model, which essentially means that it’s advancing mission-related investments in companies with projects that are nearing clinical testing, and all the financial returns from those investments come back to the Foundation of the RD Fund to recycle and reinvest to further the mission.

The RD Fund is the first and only fund designed to accelerate therapeutics specifically for inherited retinal diseases and dry age-related macular degeneration, including its advanced form GA or Geographic Atrophy. And in its first six years, the RD Fund has advanced our mission with over 30 regulatory and clinical milestones achieved by portfolio companies that we’ve invested in.

In addition to delivering these impressive clinical returns, the strategies for achieving sustainability for the fund over the next five years are really to emphasize financial returns coupled with a meaningful fundraising campaign we anticipate launching at the beginning of calendar year 2025.

The current portfolio for the fund consists of several first and best in class programs, diversified across indication, time of intervention and modality. In the next five years, the fund aims to bolster inherited retinal disease, dry AMD and GA coverage within the portfolio by expanding our existing portfolio, while also further diversifying the portfolio with a focus on gene agnostic approaches, including cell-based strategies and a particular emphasis on interventions for late stage vision loss restoration, including for the first time ever, the addition of visual prosthetics within the purview of the RD Fund.

The plan that we’re talking about today was developed under the leadership of Dr. Rusty Kelly and Dr. Alicia Kimball, who are the two driving forces behind the fund from a staff perspective. It specifically sets forth four main strategic imperatives to guide the fund’s activity and focus. Number one, to actively support and expand our existing portfolio by leveraging the extensive resources of the Foundation to strengthen the companies that we’ve invested in and help them navigate challenging financing conditions. Number two, to increase portfolio diversification by broadening the investment scope to emphasize strategies aimed at later stage disease while expanding the dry AMD and GA segment of our portfolio. Number three, to develop a flexible investment strategy where in the near term, we’re going to prioritize financial return that generally follows larger market indications in clinically staged programs. And number four, to achieve sustainability for the fund by delivering financial returns from prior investments back to the fund to fuel future investments while still supporting small catalytic investments for transformational programs. As we look to the future, the RD Fund will become an even more important factor in driving research for more treatments and cures for inherited retinal disease and dry AMD.

As Peter mentioned earlier in this call, a company that was actually conceived and launched by the RD Fund, Opus Genetics, just themselves completed a transformational merger. Opus launched in September of 2021 as a patient-focused gene therapy company targeting inherited retinal diseases. The initial seed funding to launch the company was led by the RD Fund and included investments from the Manning Family Foundation and Bios Partners. The company was focused on advancing the pioneering work of its scientific founders, Doctors Jean Bennett and Junwei Sun from the University of Pennsylvania and Dr. Eric Pierce from Harvard and Mass Eye and Ear. In the years that have followed since the launch of Opus, Opus has had an incredible run, building a pipeline of early stage assets and including a treatment for LCA5, which is currently in a Phase ½ clinical trial.

As I mentioned before, Dr. Ben Yerxa served as the CEO here at the Foundation Fighting Blindness for five years before moving to take on the CEO role of Opus Genetics and served as CEO of Opus Genetics prior to and up until the acquisition that just took place. Now he serves as the president of the new Opus Genetics. Prior to joining the Foundation, Dr. Yerxa served as president and co-founder of Envisia Therapeutics, another company focused on developing novel ocular sustained delivery therapies. He’s held founding and executive positions in several other ophthalmology-based organizations, focusing on the discovery and development of investigational new drugs, Phase 3 clinical programs, new drug applications, and importantly, drug approvals. He holds 60 US patents, which is only about 60 more than I have, which is pretty good. Ben, it is just so great to have you back here on the call today and for you to share some of the exciting developments that are happening at Opus Genetics. I’m going to turn the call over to you, Dr. Ben Yerxa.

Dr. Ben Yerxa, President, Opus Genetics

Great, thanks, Jason. It’s great to be back here with the community, and let me tell you a little bit more about Opus. The mission for Opus has been really clear from the beginning and it really hasn’t changed. We saw a gap in the IRD gene therapy space where there was great science, often funded by FFB’s grants program, but was not moving into clinical trials despite having clinical candidates at leading institutions that were ready for full development. The gap occurred for a variety of reasons. Some assets were isolated as single programs within big universities like Penn and Harvard, and others have been licensed by bigger companies and their priorities just shifted. Regardless, there were more than half a dozen of these orphaned orphan assets that needed a framework to move them ahead. So the Opus mission really evolved into solving this problem by developing a basket of IRD gene therapy assets into and through the clinic in a financially sustainable way.

The mission, by the way, became a very contrarian MO because of our timing. The company was incepted in early 2021 during the biggest biotech boom I’ve essentially ever seen in 30 years. And once we finalized the initial financing and licensed the first few assets, our mission of accumulating and developing many assets at once started to hit strong headwinds. We had deliberately only secured startup funding and intended to raise a much larger round once the assets were secured and a full-time management team was in place.

Well, the post-COVID hangover was kind of brutal, and 2022 was a terrible year for biotech funding, especially for early-stage companies like Opus. Then 2023 was even worse, and it became clear that it was going to be a game of survival. So Opus basically hunkered down while also being opportunistic, getting some more assets and remaining true to its mission. We were able to use the resources we had and some additional capital from our investors, mostly led by the Foundation’s RD Fund, to get our first program, a gene therapy for LCA5 into the clinic. This was an important milestone because as a gene therapy company, once you’re in the clinic, you’re in a different category, which opens up investment opportunities from more venture capital firms.

We got really nice initial clinical results, but in late 2023 investors were still skittish and just didn’t step forward with enough conviction. Fortunately, our lead investigator at Penn, Dr. Tomas Aleman, got some podium time at the Annual Academy of Ophthalmology meeting, and we started getting noticed by strategic partners, such as bigger biotechs and even big pharma. The issue for Opus was that these kinds of conversations take a long time, and we were still pretty early for a deal like that.

Meanwhile, we kept getting more and more good data from the trial, and then we ended up having a conversation with Ocuphire about a business combination. The deal with Ocuphire ended up being a really good fit because Ocuphire had substantial cash and an R&D team with expertise in retinal therapeutics and IRDs, including the CEO, Dr. George McGrath, and Opus had a pipeline of seven assets, one that had reached clinical proof of concept, one employee, yours truly, and limited cash to move the company forward.

In fact, Ocuphire had so much conviction in Opus as the acquirer that they changed their name to ours and adopted our mission and brand. They agreed to bring me on as President and added three Opus directors to their expanded board, including Dr. Jean Bennett, Dr. Adrian Graves, and myself. Now, Opus Genetics is the only public pure-play IRD gene therapy company with a NASDAQ ticker symbol, IRD. Anyone can buy the stock. I mean, how cool is that? Remember, FFB and the RD Fund created this from scratch three years ago.

Moving forward, we’ve got a full pipeline to develop with seven gene therapy assets at various stages of development and all of which stand on their own scientific merits. Immediate priority goes to those programs that can generate near-term clinical results, which are the LCA5 and BEST1 programs. For LCA5, we expect to have preliminary data from pediatric subjects in the third quarter of 2025 and preliminary results from the BEST1 trial in the fourth quarter of 2025. The other programs are moving along, pending additional funding from either grants or project-based funding from nonprofits like FFB and others.

Our business model allows us to partner with advocacy groups to fund programs in early development to de-risk them prior to entering the clinic. These programs include gene therapies for IRDs caused by mutations in the genes RDH12, RHO, NMAT1, MERTK, and CNGB1. If we do this right, Opus will become an engine that cranks out novel gene therapies for IRDs on a recurring basis. To be clear, this is still challenging novel work that requires intense focus to navigate all the twists and turns that occur during full development, but we are committed to figuring this all out.

2025 is going to be a big year for Opus, and we have four clinical data readouts. In addition to the LCA5 and BEST1 gene therapy data, we also have two Phase 3 clinical readouts for the Phentolamine program that has a commercial partner. These data points are for dim light disturbance in the first quarter of 2025 and for presbyopia in the first half of 2025. If we’re successful with these trials, it could lead to meaningful commercial milestones and royalties in the future. It is a great lineup of potential catalysts for investors and the patient community to track our progress.

So with that, I’ll turn the program back over to Jason and look forward to taking any questions related to Opus and our pipeline.

Jason Menzo, Chief Executive Officer:

That’s awesome. Thank you so much, Ben, and of course, congratulations on all the progress that you’ve achieved. It’s got to feel pretty good not to be the only employee anymore, so two thumbs up for that. We’re just so thrilled at what has transpired with Opus since the very beginning and where it is today, primed obviously for even brighter days ahead.

Before we open the call for questions, I just wanted to follow up on one of Amy’s comments specifically about the renaming of our Translational Research Acceleration program in honor of our recently retired board chair, David Brint. Renaming this program honors David’s relentless dedication to accelerating research and expanding the Foundation’s resources to pursue treatments for retinal diseases. We’re so grateful for David’s many years of contributions and are extremely pleased to keep his impact top of mind through this awards program. David continues to serve on our board. He’s still exceptionally involved with the organization, but we felt it was a perfect opportunity to recognize his impact by renaming this important program in his honor.

With that said, we are now ready to open the call and take your questions and comments. It’s about 20 minutes before noon, so we’ll have about 20 minutes for calls here on the East Coast. It’s 11:40. So I’m going to ask Chris to go ahead and please provide instructions for how you can ask questions.

Chris Adams:

Thanks, Jason. There are several methods that you can use to ask the questions. You can submit them through the Q&A or the Chat function at the bottom of the Zoom screen, and please make sure to include your name so that we can follow up afterward. As Jason said, you can also send an email to info@fightingblindness.org, and we will follow up with you in the next week.

Jason Menzo, Chief Executive Officer:

Very good. Thank you so much, Chris. I invite our panelists to please come on camera, turn your mute button off so we can hear you, and we’ll go ahead and get started. We’re in a unique position, and I think one of the themes from this call is the building momentum and how different the mission feels maybe today than it has even in recent history. To really exemplify that, the first question that came in is about a program that is actually on the cusp of interactions with the FDA to seek FDA approval. There’s actually two programs that are really close in two different areas of our mission, so I’m going to ask Dr. Todd Durham to provide an update on both Nanoscope, which some folks have chatted in, and then also Alkeus. Perhaps Todd, if you could briefly describe the strategy of what Alkeus’ treatment is aiming to do, because there were some questions about vitamin A and Stargardt, so maybe we can cover that whole basket in one topic here.

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

Okay, thanks Jason. Everyone, this is Todd Durham. Recently, Nanoscope Therapeutics announced on their website in a press release their plan to submit a biologic licensing application to the Food and Drug Administration for their optogenetic therapy MCO-10. They are planning this application for the first quarter of the calendar year 2025, and just as a reminder, this is an optogenetic therapy intended for use in individuals with advanced RP and according to the company’s website, their studies have shown improvements in functional vision, visual acuity, and sight discrimination. So we are obviously very excited about this advance. We wish them all the best, and they’ve been to the Foundation for updates on that program.

Secondly, Alkeus Pharmaceuticals has had a number of interactions with the FDA, and they recently announced that they got FDA rare pediatric disease designation and fast track designation amongst others that they’ve obtained along the way. These designations really recognize the unmet medical need for Stargardt disease, which is the target of Alkeus’ product, Gildeuretinol, ALK-OO1, which is being studied currently in four clinical trials. So far, these results are very encouraging, seeming to slow the progression of lesions in the retina in those studies.

The company also announced their plans to look at the top line data from one of their more recent studies, the TEASE-2 trial, in 2025. So we’re obviously very excited about advances in Stargardt for obvious reasons that many people are affected with it. It’s the most prevalent of our IRDs. And Amy, I don’t know if you want to comment on the therapeutic strategy for ALK-OO1 for this therapy or not.

I think that’ll do it for now, Jason.

Jason Menzo, Chief Executive Officer:

All right, thanks Todd. Actually, I’m going to come to Amy next. Amy, a couple of questions chatted in, and hopefully our attendees don’t mind. I try to take questions that are similar and put them together just so that way we can answer as much of the spirit behind the question as we can.

So a couple of questions related to RP specifically. We use this term all the time on these calls about gene agnostic approaches, so approaches to treating the disease that are not specifically targeting a particular gene or a particular mutation. The first question is in cases like that, so a clinical trial for a gene agnostic approach, does the trial design still require genetic testing in a specific gene identified? That’s sort of question one. Question two is perhaps even just to give a broad swath of what’s happening in the gene agnostic space. We’ll talk about Nacuity in a minute, but perhaps some of the other gene agnostic strategies, Amy?

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

Thank you. Again, this is Amy Laster. So generally with gene agnostic approaches, it is just as Jason described. It may be a treatment that will benefit individuals without specifically knowing the mutation that is causing the disease. In a clinical trial design, however, it can be designed two ways. One, it could be a clinical trial that’s designed that everyone, let’s say, with retinitis pigmentosa would be enrolled, if they have a clinical diagnosis of retinitis pigmentosa.

There are also instances where the sponsor may decide that everyone with retinitis pigmentosa, but specifically with mutations in PDA6 beta will be enrolled in the clinical trial. That does not mean that it is not a gene agnostic approach, but it simply means that it gives the sponsor a bit more control over how the treatment is affecting patients. So oftentimes as the trial progresses, they may include additional individuals with mutations for a specific disease, and then ultimately upon approval, there could be an opening of that particular gene agnostic approach for a larger swath of individuals than those that were targeted in the clinical trial. It’s really dependent on how the trial is designed and how the sponsor feels it’s best going to demonstrate not only safety of the particular treatment but also that it is effective.

In general, there are several different approaches for retinitis pigmentosa. Now I’m kind of shifting a little bit to retinitis pigmentosa. It could be genetic technology approaches such as we’ve heard gene therapies or things that are gene editing or RNA editing. These genetic technologies generally could benefit individuals with IRDs, in which case knowing the genetic mutation is important for those particular strategies. There are small molecule strategies also, and that’s just generally your drug, and generally the drugs, you don’t necessarily need in some cases to know the gene mutation because they’re basically disrupting the pathway that has gone bad in disease.

So as Jason mentioned, Nacuity have a program that we’ll talk about a little bit later for those. And then there are some cell-based strategy approaches as well or kind of late-stage disease approaches. Peter mentioned earlier about the BlueRock sponsoring of Phase ½ clinical trials, which is using photoreceptors to be transplanted into a degenerating retina, and that is gene agnostic in the sense that it’s not that you need to know the gene mutation that’s causing a disease. That particular trial is ongoing.

Jason Menzo, Chief Executive Officer:

Thank you so much, Amy. We’ll come back to you in just a minute. We often in this call get specific questions almost like patient by patient, individual by individual. “My daughter’s specifically affected by this particular gene, this particular mutation. Can you advise me on what we should do?” I tend to like to take those types of questions offline. So if we don’t get to your question and it’s really specific, there’s an intentional reason. We try to use this opportunity to talk in as broad a case as possible, so that way the majority of folks can benefit from the question and answer. However, any question that you chat in, we have and we will follow up one-on-one offline. We do after every call. If we don’t get to your question, that’s the reason why sometimes and that’ll be the plan. Don’t be discouraged if we don’t specifically address every question here on the call.

That said, broadly, there were a couple questions on specific genes where there are some really promising updates that we can share. I’m going to invite Rusty Kelly, who’s the Managing Director of the RD Fund to provide updates on two programs that are under the management of a company called Thea. So an update on CEP290 and USH2A, and then a portfolio company in the RD Fund, Nacuity, which we’ve spent a few minutes talking about here today. Rusty, perhaps you can give updates on those three programs.

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

Thank you, Jason. This is Rusty Kelly speaking. With regard to the first two programs, CEP290, USH2A, there are multiple programs in early-stage non-clinical development for these programs, but the two programs that Jason referred to with Thea Pharmaceuticals, you may know that from recent press releases, there’s several unique genetic approaches to treating CEP290 and USH2A that were being developed by a company called ProQR. ProQR  happens to be a company that the RD Fund made a significant investment in, and Thea Pharmaceuticals announced recently the acquisition of these two programs known as sepofarsen and ultevursen, respectively.

There’s not a lot to report at the moment. As you would imagine, a company that acquires assets, there’s a transition period for developing a clinical strategy to relaunch those trials. I think the best thing that we can do is stay tuned for any news published by Thea on the future clinical development of these two programs.

With regard to Nacuity, another important RD Fund portfolio company, was formed based on the work of Peter Campochiaro at Hopkins, and this is to develop an oral antioxidant for the treatment of RP related to Usher, all Usher in fact. So there’s an ongoing Phase ½ study in Australia evaluating this oral dosage of N-acetylcysteine amide, NACA, the antioxidant in RP and is expected to conclude next summer when the company will reengage the FDA to discuss the analysis of NACA trial and next steps in its clinical development.

Jason Menzo, Chief Executive Officer:

Excellent. Thank you, Rusty. Picking up on that, Amy, I want to come to you, and actually there’s another interesting question that just got chatted in. So I’m going to surprise you with twofold, but this will be something really easy for you to describe. One, there’s another similar trial that many folks in the community have heard of NAC Attack, and perhaps you could speak a little bit to what NAC Attack is, and then we had a very specific question chatted in the BlueRock trial. “Are they inducing stem cells to be rods, cones or both?” Probably oversimplifying it. I know you can speak to that, so if you could speak to those two.

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

Okay, so with the BlueRock, they are inducing a photoreceptor, so rods and cones quickly to answer that. Thank you for that question. Good question. And this is Amy Laster speaking again. Sorry, I didn’t say that initially.

Going back to NAC Attack. So just as Rusty mentioned with the NACA, the work began out of the laboratory of Dr. Peter Campochiaro, who’s at the Johns Hopkins University, and he is the principal investigator on a current phase three clinical trial for N-acetylcysteine, NAC, lacking the A that is in NACA. It’s just N-acetylcysteine is the compound, and NACA is just a modified form of NAC that is designed to be a little bit more potent and bioavailable in the retina. So very similar compounds in terms of what they will accomplish, in terms of mechanism, but just the amount of it and availability of it is different between these two trials.

So the NAC Attack, this is a 45-month study. It’s enrolling more than 400 patients around the globe, including US and Canada, Mexico, Europe, and they’re looking to do the first assessment of the NAC groups benefit at about two years. Then those that are assigned to the placebo group of the trial, they’ll move over if there is benefit to the treatment trial. We are too anticipating outcomes from the NAC Attack as well as, excuse me, Nacuity’s NACA trial.

Jason Menzo, Chief Executive Officer:

Very good. Thank you, Amy. Want to shift. We always get a bunch of questions about our My Retina Tracker Registry and our no-cost Genetic Testing program. Oftentimes we speak about the Registry in the content of the call itself. Today, we didn’t have as much discussion about that, but I do want to give Todd an opportunity to talk a little bit about My Retina Tracker, why it’s important, how folks get genetically tested, when they should get retested if there wasn’t an identified pathogenic gene identified. Just the brief overview of those topics, please, Todd.

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

Yeah, thanks, Jason. Hello, this is Todd Durham again. My Retina Tracker Registry is a study that we sponsor and run here at the Foundation Fighting Blindness. It is intended to help us understand the genetic mutations associated with inherited retinal disease and to describe the characteristics of those individuals who have IRDs. This is also a platform that enables us to contact individuals with inherited retinal disease about research opportunities.

One of the main ways that folks become enrolled in My Retina Tracker Registry is through our sponsored genetic testing program. The typical way many of you, if you’ve already been diagnosed with inherited retinal disease, have probably already had many clinical workups, but many people, when they’re seeing their ophthalmologist for the first time, getting a clinical exam, a recommendation might be for genetic testing to confirm what the clinician sees as a preliminary diagnosis on the basis of their findings. And for that, we have our sponsored testing program, which allows us to offer at no cost to patients the genetic tests through our lab partner, Prevention Genetics, and the genetic counseling that we recommend that comes along with that test. This information then is provided back to your clinician and to you for further action. You can use this to help you follow research news and find clinical trial opportunities that might be relevant for you.

We find that in the commercial panels that are used in our program and others, that IRD patients, between 50 and 65% of them will have a clear-cut diagnosis from the genetic tests. That leaves a significant proportion of people who don’t have a clear-cut answer, and the reason for that can be varied from person to person. A lot of times it has to do with maybe we see one variant in a disease that requires two mutations, and one of them we believe is disease-causing, but maybe one we don’t have enough evidence to call it a clear-cut case. So that’s a fair number of those cases that are unclear. But there are still a significant proportion of cases remaining that aren’t so easy to detect using the methods that we use in clinical panels like complex structural variants and things that aren’t easily picked up with the technologies that are readily available.

If you are in that category of an unclear diagnosis from a genetic test, the first recommendation is to go back to your ordering provider or your genetic counselor. They can keep you up to date on the progress on the clinical panels. And in our program, if you’ve had access to a clinical panel in the last four or five years, you’re not eligible for retesting in our program. The reason why is that technology is not changing so rapidly anymore such that we’re not going to pick up something new in such a short period of time. The best recommendation is to go back to your genetic counselor or ophthalmologist and find out when is the best testing interval for retesting, and sometimes you need a totally different approach based on that conversation.

Jason Menzo, Chief Executive Officer:

Great. Thank you so much, Todd. And Chris just put in the chat, the link to the My Retina Tracker Registry, which is at www.myretinatracker.org. A lot of great information there as well. Thank you, Chris.

We’ve only got about three minutes left. I want to get to two other questions. There was a question specifically chatted in about the Opus pipeline, and the question was specifically about one of the genes in the Opus pipeline, NMAT1. I know, Ben, you’re not really in a position to talk specifically about timing of each product or program specifically, but maybe generically you can speak about the timing now that Opus has funding and the transaction’s behind us. What does the priorities and the roadmap look like in terms of the pipeline and getting things cranked back up?

Dr. Ben Yerxa, President, Opus Genetics

Yeah, it’s a great question, one that we get a lot, as you can imagine. I’ve probably said this before, but when you think about how do we manage seven programs, it’s kind of like having seven children. I don’t mean that as a joke. It’s like basically we love them all the same. It’s just some are older than others, so some are getting ready to go to college and go into clinical trials. Others are just younger and just need more time but importantly need more money. So it’s really dependent on us getting some additional capital, whether it’s project specific or just general working capital to move all these things forward. We’re in active discussions to make that happen on an individual project basis. Until we secure the funding, I’m hesitant to comment on timing because it’s just uncertain until we have the actual capital for those, but we are working on it. Stay tuned and sorry it’s taking so long.

Jason Menzo, Chief Executive Officer:

Yeah, it’s probably fair to say folks who’ve been watching the Opus pipeline for the last couple of years may be thinking, ‘Well, wait, why is this date slipping on this particular program?’

Dr. Ben Yerxa, President, Opus Genetics

Why did something jump ahead? Yeah.

Dr. Ben Yerxa, President, Opus Genetics

It’s based on financing. It’s what investors will finance right now. So it’s really all about the money. The science is there, we just need the money.

Jason Menzo, Chief Executive Officer:

Thank you so much, Ben. And obviously the Foundation, the RD Fund under Rusty’s leadership, we’re working very hard to help aim to solve that problem.

Jason Menzo, Chief Executive Officer:

With Opus as a partner, it’s not lost on us how important this is. The last question I want to get to today, there’s a number of questions chatted in about whole eye transplant, and Amy spoke to the concept in this initiative from ARPA-H, where the Foundation is actually leading the strategic management of a consortium, including multiple academic institutions, up to a $46 million initiative. This is really, really huge. Obviously, it’s great for the Foundation to be aligned on such a high profile and potentially world-changing type of initiative. Peter, the question that I wanted to pose to you to wrap up the call today is why now? What’s the history with this? What has been done previously? Why is the federal government looking at this as an opportunity today?

Peter Ginsberg, Chief Operating Officer:

Thanks, Jason. This is Peter Ginsberg. As Amy highlighted, we brought together a fantastic group of institutions to bring this whole eye transplant opportunity forward and hopefully bring it from concept to bedside and actually benefit patients. That won’t be tomorrow, it won’t be next year, but we’re hopeful that within the next six years, we’ll actually be able to provide the whole eye transplant in patients in a clinical trial setting.

Why now? We worked with ARPA-H, that’s the government agency that awarded these funds, to develop this opportunity because there’s so much work going on across the world toward whole eye transplant. The problem is it’s all going on in specific silos. The group that we brought together, we bring all those different technologies together because if one technology moves forward, perhaps around connecting the optic nerve, that’s great, but if the other technologies don’t move forward within the whole eye transplant continuum, then it doesn’t really get us to where we need to get. The time was right to bring all of these performers together so that they could work in a fashion with continuity and they could share ideas and put their different technologies together for the benefit of patients that require these whole eye transplants. The time was right because the technology was there, but there had been no entity bringing those technologies together in a broad ultimate solution. We’re really excited to be a central part of this.

Frankly, as we tend to say at the Foundation, we don’t do the research, we fund the research, but within this consortium of centers doing the whole eye transplant work, the Foundation is the glue. We glue these different puzzle pieces together and hopefully provide a solution that will benefit patients in the long-term.

Jason Menzo, Chief Executive Officer:

Thank you so much. That’s a great way to end the call today. Thank you, Peter. And as always, I just want to extend my personal gratitude and thanks to all the members of the community, our board, our trustees, our chapter leaders, the volunteers, walk captains, committees for the fundraising events, I mean all the different folks, folks in London who hosted us a couple of weeks ago for a fantastic event, folks in Paris a couple of weeks before that. It is a global community, and it does take everyone in this community working together to advance this mission. On behalf of the entire Foundation team, I just want to thank all of you for your passion, hard work. We really are poised to move more therapies into clinical trials and across the finish line, and it’s only going to happen with all of us working together. So thank you so much.

We always welcome your feedback and suggestions related to this webcast or the Foundation in general. You can reach us at any time by emailing us at info@fightingblindness.org. And as always, you can learn more at our website fightingblindness.org. If you have any questions, don’t hesitate to reach out. I’m going to turn the call over to Chris to wrap things up.

Chris Adams, Vice President, Marketing & Communications:

Thanks, Jason. As Jason said, we’d like to thank everyone for joining us today for today’s call. As a reminder, there will be a transcript and recording of today’s call within the next week on our website www.fightingblindness.org. Also, be sure to follow us on social media to stay informed on the latest news and activities from the Foundation. As always, if there’s any other information you need, please reach out to us by sending an email to info@fightingblindness.org. Thank you and have a great day.