Johnson & Johnson (JNJ) Earnings Call Transcript & Summary

Hi. I'm Jim List, Global Head of the Cardiovascular, Metabolism and Retinal team at Janssen. I have the privilege of sharing how we're improving the lives of millions of people impacted by eye diseases. Today, I'll highlight new data from the Phase I/II clinical trial of the gene therapy we're developing with MeiraGTx to treat X-linked retinitis pigmentosa, a rare inherited retinal disease. I'll also touch on why we're focused in this space and our overall strategy. But first, please take a moment to read our cautionary note on forward-looking statements contained in this webcast. Janssen is the pharmaceutical arm of Johnson & Johnson, and we are proud to be part of the broadest and most diversified health care company. Johnson & Johnson has long-standing leadership in both consumer and surgical devices through J&J Vision. Janssen's entry into eye disease only extends our commitment to support patients living with diseases of the eye. At Janssen, patient needs set the priorities. We have a legacy of entering spaces with innovations that transform lives. From young children to those young at heart, our newest commitment to eye disease is no exception. We believe Janssen's deep experience and Johnson & Johnson's broad ophthalmic footprint makes us well positioned to deliver on our mission, which is to restore and preserve vision, both for the millions of patients affected by common eye conditions and for those with rare inherited retinal diseases. Let's take a moment to look at the enormous and wide-reaching impact that eye disease can have. More than 350 million people globally have some form of visual impairment or blindness associated with the retinal disorder, and the economic burden to health care systems is even more staggering. That's why we've taken on developing transformational therapies for eye disease and are rapidly amassing new capabilities and partnerships such as retaining some of the world's great retina talent, advancing our AAV manufacturing technology, entering into strategic collaborations and forming critical partnerships with leading eye institutions. And we now have a dedicated discovery site in San Francisco to advance our early gene therapy programs as well as to discover new targets for more common eye diseases. We've made excellent progress, and we're far from finished. Here's a look at our expanding portfolio since we first established this focus area in late 2018. Our lead gene therapy candidate, AAV-RPGR, in partnership with MeiraGTx, is being studied to treat X-linked retinitis pigmentosa or XLRP. It's designed to counteract the loss of retinal cells resulting from mutation in the RPGR gene. This mutation is responsible for most cases of XLRP. Not only did this receive Fast Track designation from FDA and orphan designations from FDA and the EMA, but in late February, EMA granted both PRIME and ATMP status based on data from the ongoing Phase I/II trial. These are critical designations that accelerate the review time lines and bring us one step closer to delivering a transformational therapy. I'll speak more about our new RPGR program in just a minute. The other 2 IRD assets you see here target mutations in the genes CNGB3 and CNGA3, both of which cause achromatopsia. And lastly, our newest program is in partnership with the U.K.-based biotech, Exonate, to develop an eye drop for more common eye diseases such as diabetic retinopathy and age-related macular degeneration. Again, we're making great progress, and we're excited to now share findings from our lead RPGR asset. XLRP is an inherited retinal disease impacting males. It's typically diagnosed in children and young adults and begins in adolescence with night blindness and loss of peripheral vision and as seen here, is a relentless progressive constriction of the field of vision that eventually leads to blindness. And there are no approved treatments, which is our motivation, what inspires us to keep discovering and developing innovations. Turning now to the study. The eye is an ideal target organ for gene therapy because of its size, immune privilege and accessibility. Our approach with RPGR is to replace the effective gene, thereby halting or even reversing the pathology of the disease. We did this with subretinal application targeting the central retina using multiple retinotomies to cover the maximum amount of viable tissue. The design of the trial is composed of 3 parts: a dose-escalation phase in adults, a dose-confirmation phase in children and lastly, a dose-expansion phase. The interim data I'll review today are from the dose-escalation phase. The primary endpoint was safety and tolerability. Secondary endpoints assessed retinal sensitivity, visual function, functional vision and quality of life measurements. What I'll focus on today is the first of these, retinal sensitivity. This was assessed at baseline and again at 3, 6, 9 and 12 months. We used 2 modalities, full-field static perimetry and microperimetry as well as 3 different metrics that are seen here. Data from the dose-escalation phase suggests that our vector has an acceptable safety profile. Half of the AEs that occurred were ocular in nature, related to surgery, and were transient and resolved without intervention. There were 2 serious AEs observed noted here, no dose-limiting effects. And while we observed inflammation in 2 of 3 patients in the high-dose cohort, they were effectively treated with corticosteroids. Based on our interim results from this ongoing trial, the low dose demonstrated a significant improvement in field-of-vision, and the intermediate dose demonstrated a significant improvement in both, mean retinal sensitivity and field-of-vision. As you can see, the high dose did not show improvements likely related to the inflammation observed in 2 of the 3 patients at that dose. Based on these findings, we selected both the low and intermediate dose for our expansion cohort. If we look at all eyes in terms of central retinal sensitivity with the low and intermediate doses, we see improvement in the treated eyes, shown in red, with improvement in central retinal sensitivity; and a decline in the untreated eye, shown in blue, with a significant difference between the treated and the untreated eyes. These findings give valuable insights in the potential of our AAV-RPGR gene therapy not just to preserve, but to improve vision for people living with XLRP. We're very encouraged by these interim data, and we look forward to future readouts in the Phase I/II program and the advancement of our clinical program into Phase III. In conclusion, we're extremely proud of the progress we've made with our portfolio, and we'll continue to apply learnings from the Phase I/II program as we advance it forward, build and strengthen our capabilities in eye diseases by attracting world-class talent, expertise and partnerships and lastly, live into our team's mission to bring new transformational solutions to patients. Thank you.