Deck Review with Regenxbio
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Cover slide to convey the company’s value proposition - focused on AAV gene therapies.
Regenxbio lays out their goals to cure disease, not just treat it. What’s important here is the three main parts being equally laid out. This shows shareholders that the company values both their platform business and pipeline equally. I would value the platform a little more, but most companies like Regenxbio lead off with only their internal pipeline and see licenses as periphery activities. A great addition would be a focus on their value prop - something like expression or natural AAVs that would get at the heart of their focus on pipeline, protein, and product.
The next section is focused on their internal pipeline highlighting how the team sees the future of Regenxbio. The slide is pretty vanilla.
Like all good drug companies, they focus on their flagship program - an AAV therapy for wet AMD. They describe the problem (leaky blood vessels in the eye) and how large it is (~2M patients) along with the vector (AAV8) and delivery cargo (anti-VEGF Fab). However, the company doesn’t mention Eylea or other wet AMD medicines that are already approved. Regenxbio alludes to issues around delivery of drugs like Eylea, but doesn’t go too deep on this slide or the presentation in general about how competitive their RGX-314 program will really be amongst clinicians.
An important point for any gene therapy is delivery whether it’s a transgene or a CRISPR protein. A major reason why Regenxbio focused on wet AMD and opthamology in general is that delivering something to the eye is a lot easier than delivering something to the brain. A point the company could make is make clear that their delivery method (sub-retinal) is more invasive than the method current wet AMD medicines use (intravitreal). That would help understand the reasoning behind Regenxbio’s delivery method - broader coverage across the retina and higher protein expression.
This is a really clear slide laying out how their trial is designed. It’s a phase I/II so the focus is on safety; the pivotal trial will come later. A really important aspect of any trial is the inclusion/exclusion criteria. A lot of companies play games with this early on to get decent data across 10 patients that ends up failing for 100s of patients in a later trial. You’re seeing these games right now with some of the COVID-19 vaccines in phase I development. Regenxbio is focusing on patients that have the highest prevalence of wet aMD and ones that are likely to respond to an anti-VEGF therapy.
Pretty simple slide on the design of their phase I. They’re just trying to show safety and get a sense on dosing - the optimal and maximum.
Then they show the results they got for the phase I. They met their primary endpoint and showed safety so far.
This slide shows how dosage is connected to the protein expression levels of the therapeutic cargo. This is a pretty obvious experiment for any company wanting to show safety and get a sense on how to dose their drug for the next trial.
This slide shows how increasing doses of their gene therapy reduce the number of injections. This is going to be an important experiment and data set to argue for clinicians to switch over from something like Eylea. Regenxbio would need to show efficacy in a pivotal trial to have a high enough value proposition.
This slide alludes to potential efficacy of RGX-314 without needing another injection. Their BCVA data is interesting; however, it’s over a small patient population and needs to be expanded in the next trial. The current efficacy data is in line with other wet AMD results, and maybe a little below it. The Phase IIb trial will show more.
This slide shows the patient outcomes before and after being dosed with RGX-314. This is a really powerful slide to convey the value prop of their medicine versus the standard of care - less dosing and invasive delivery procedures.
Another slide on efficacy for patients that need several injections. The data needs a jew more months to show clear results.
Efficacy results for patients that don’t need a new injection. This data also needs a few more months to develop. The presentation goes through similar slides for Regenxbio’s work on diabetic retinopathy and other indications.
The next section of the presentation goes into the platform. This slide gives an overview of Regenxbio’s core technology focused on AAV7-10 and natural or close-to-natural variants. The slide shows the title of two NEJM papers that were pivotal for Regenxbio’s success. The company lays our 4 key features that sum up the value prop of the NAV platform to partners.
This impressive slide shows the adoption of the NAV platform by other companies. This was part of the company’s masterplan to focus on licensing then add internal development as the platform was validated by others.
For every gene and cell therapy company, manufacturing is top-of-mind. Although they don’t mention their collaboration with WuXi AppTec, Regenxbio is conveying the scale of their infrastructure to have the capacity to commercialize their own gene therapies.
Team and Milestones
Pretty generic team slide every public company has. Rarely does a founder still lead a biotech company after more than a decade.
The upper half of this slide is more geared toward investment banks on Wall Street - what’s the quarterly guidance that may or may not be productive but is a feature of being public. The bottom half is very useful as it lays out the company’s important milestone for their internal products. Milestones around their platform business would be useful to add.
The deck does a good job at quickly describing the problems around AAV gene therapies, wet AMD, and beyond and how Regenxbio is positioned as a business to solve them. More work can be done to convey Regenxbio’s competitive advantages and highlight the core value of their NAV platform.
Life science companies are multi-phasic and change according to what experiments work and don’t work. Sometimes the current leadership can’t change to lead the company in the next phase. What’s really impressive about Regenxbio beyond their licensing business is that the co-founder is still CEO.
Follow up questions for the founder and the team:
What “next-generation” techniques are being used to burnish the NAV platform? Would a company like 4D or AskBio be acquisition targets?
What feedback is the company getting from clinicians about Eylea versus RGX-314? How about for payors on the price of the gene therapy versus anti-VEGF therapies that will become generic soon?
Any guidance on pre-clinical work on metabolic diseases and CNS?
What are the key milestones for the licensing business? Is the core metric number of new programs, maximum royalty rates, or something else?