Vor Biopharma Inc. Common Stock Citigroup’s Biopharma Back to School Summit 2026

Sure. Well, thank you, Geoff, and glad to be here and to tell you about our progress. Does it work? Yeah. I am here with my team.

They will pipe in and answer questions with me. Vor is a company focusing on B-cell-mediated autoimmune diseases. It has in-licensed our main asset a year ago around June 2025. It is called Telitacicept. It is a BAFF-APRIL inhibitor, which has the ability to tackle the upstream of the B-cell lineage. There is a big unmet need with that, and it basically modulates the B-cell development, production, and survival and inhibits the production of pathogenic autoantibodies. It has been approved in six indications in China. That is very important. There is a wealth of data out there in several autoimmune diseases, and we have chosen in the West, in the U.S., and elsewhere to develop Teli in myasthenia gravis and Sjögren's disease to start with. Since we licensed the asset a year ago, we have made tremendous progress.

You probably saw the announcement yesterday that we completed the enrollment of our phase III trial in myasthenia gravis. It is a huge achievement for a company of our kind, especially we took over a trial which was not doing very well at the time, and it is now doing very well. Jeremy will talk about it further. We believe that we have a fantastic opportunity in MG. We are always hearing from the physicians that there is a big unmet critical need there. They need therapies with a deeper clinical efficacy, but even more so, a durable clinical efficacy. That is what lacking with the current therapies. We believe Telitacicept has everything here.

As a matter of fact, I'll draw your attention to AANEM in a couple of weeks, where we'll present very compelling and exciting data on an endpoint, which is probably the come to Jesus endpoint in MG, which is called minimal symptom expression, which is basically achieving less than 1 in MG ADL for patients, basically asymptomatics. There we will show that we have a deep but even more important durable effect on patients achieving MSE. That's for MG, but we also saw that we announced yesterday that we have the intention to start ocular myasthenia gravis phase III in the first half of 2027. We believe it's a perfect adjacency. It's logical. It's not busy.

There are only two products approved, and there is also a high unmet medical need, and we can help patients earlier in their disease journey and modify the disease because we act on the upstream and help those patients. We can achieve more than $1 billion in this indication. MG is our beachhead indication, multi-billion dollar potential, now complemented by OMG. Again, shows our confidence in our ability to execute in our results as well with this second myasthenia gravis indication. I'll finish by saying that we also have Sjögren's disease. We have a phase III. We started de novo, I think, late last year. Jeremy? March, actually. So March. We are enrolling very well, above expectations. Shows the two things again, our ability to execute, but also the interest from the space in a white space.

Yeah. There is no product approved.

We have the potential of achieving multi-billion dollars in this indication. We'll talk about it more. It's probably a market which at start should be more than $10 billion, but could be more because all these large indications and tabletop number AD, there is always a potential to treat patients better and earlier.

Sure. That is probably a great question for Jeremy.

First, we will start differentiating BAFF-APRIL from APRIL. I think IgAN has taught us that in IgAN specifically, there is not a lot added from BAFF to APRIL. Sibeprenlimab, povetacicept, Atacicept, Telitacicept all have roughly similar levels of Gd-IgA1 reduction, proteinuria reduction across when dosed appropriately for IgAN. That led to the argument that we are not sure what BAFF is adding. That is the exception, not the rule. In a disease where you are targeting the autoantigen, which is Gd-IgA1, they are produced by mucosal plasma cells. APRIL inhibition is very effective at reducing mucosal plasma cell generation of Gd-IgA1. Therefore, APRIL alone tends to be similarly effective to BAFF-APRIL in IgAN. That is not the case for any of the other autoimmune diseases we are going to talk about, where you have to address the autoreactive B cell that is making the pathogenic antibody, not the pathogenic antigen.

Even once we get there is differentiation within BAFF-APRIL inhibitors, some of which have a higher potency for BAFF, some of which have a higher potency for APRIL. Think of it like the bispecific antibody. If you do not get the stoichiometry correct, you will end up either overdosing or underdosing in different disease indications with the wrong molecule. Telitacicept specifically has a 2-to-1 BAFF to APRIL mechanism. It is the most TACI-like native TACI. That may be lucky, may be by design, but the 2-to-1 gives the opportunity to dose to APRIL and overdose on BAFF, which is absolutely acceptable and safe. BENLYSTA taught us, belimumab, that 1 mg per kg, 10 mg per kg, relatively similar efficacy, maybe a little improvement, but no increased safety liability when you 10X the dose of BAFF.

APRIL going from even 80 to 240 in the povetacicept phase II studies of IgAN showed that there was clearly a dose-dependent increase in the incidence of hypogammaglobulinemia. At the end of the day, the goal has to be dosing to APRIL, because APRIL is the one that you need to maximize without going over, and if you do that with a drug that is APRIL heavy, you will underdose BAFF. Whereas with a drug like Telitacicept, by dosing to APRIL, in some situations, we may slightly over target BAFF, but that is acceptable. In other places, in other patients with different heterogeneous drivers, hitting that level of BAFF is critical to modulating that upstream mechanism of B-cell inhibition.