Vor Biopharma Inc. Common Stock Wells Fargo 21st Annual Healthcare Conference

Well, thank you, Sadia, and thank you for having us. We are excited to be here, and it has just been just over a year since Vor 2.0 has rearranged itself the way we have, and we couldn't be more excited this morning. We have got the announcements that you spoke about, which speaks to a few things. It speaks to the execution that we have been able to generate very rapidly. It speaks to the KOL excitement about this new modality and this new agent, and speaks to the really great people we have been able to bring on board with Vor Bio. We are happy to be here, and Jeremy is going to walk through the exciting news that we have today.

Thank you. Great. Thanks. I will echo Dallan's thanks for the invitation and just take us through some of the events of the last week.

As Dallan mentioned, Vor 2.0 has only been in existence for about a year, about 9 months as an independent clinical development company. During that time, we have taken over and really rapidly executed on our upstream MG study, our phase III study of telitacicept in myasthenia gravis. We are proud to say that study has completed enrollment. That sets the clock for our 24-week outcome to come in the spring. We are super excited about that imminent event. Even in the meantime, we are doubling down on our confidence on telitacicept in this space. We have announced the plan to start our ocular myasthenia gravis study.

Ocular myasthenia gravis is a really important area because it is often a precursor to generalized myasthenia gravis, so it really opens up that space in the totality of the MG market, and for patients who have the spectrum of MG from ocular to generalized. We also would be presumably the third, only the third agent in MG and the first upstream mechanism. The other are FcRns. Efgartigimod has completed their study, and we believe that RYSTIGGO from UCB is conducting a study as well. However, they are both the same mechanism, FcRns, for which we think we have a significant advantage, especially as we move upstream in an earlier disease population where we hope to be able to show some degree of disease-modifying effect, where we prevent the progression from ocular MG to generalized MG. This is an outcome measure that has not yet been demonstrated.

We're still figuring out exactly how to show that, but in the meantime, we're confident we can show a significant benefit for patients with ocular MG in reduction of their symptoms and their morbidity. Finally, we are excited about the American Association of Neuromuscular & Electrodiagnostic Medicine conference, which is upcoming. This is probably the largest neuromuscular conference, certainly in the U.S., and at that, we have an oral abstract, which will look at post-talk data from our China MG study. That China MG study has been published, has been presented both the 24 and 48-week data with significant efficacy, a magnitude of which is relatively unprecedented in the MG space. What we're really going to be focusing in on here is both the depth, breadth, and durability of that response.

We're also looking now at minimal symptom expression, also known as MSE, and we're under embargo, but I'll just say that it's really important to how we look at MSE. Any point in time, having one day of MSE is probably not what patients want. I think what patients want is to be able to have minimal symptom expression for the duration of their therapeutic window. So what we'll be looking at here is the durability of MSE. So we're excited to present that data. We think it's important for patients and important to really demonstrate the efficacy of telitacicept in MG.

It'll start in the first half of 2027.

Okay. We're still working through the build.

We wanted to really double down on our confidence and get it ahead of the MG readout.

Sure. I think there's two things that will push the placebo higher. One is the baseline disease activity. So the patients in China had slightly higher baseline disease activity than has been seen in other global studies. The other is the duration of disease. Many of the patients had a shorter disease duration. Just the fact that none of them had seen any other advanced therapies. They hadn't yet been exposed to biologics such as FcRn or complement inhibitors. They just weren't available in China at the time. I think that that might do that. The other thing is, a lot of patients with other treatment options pushes things down the road. In any immunologic disease, and probably any disease, what we see is that as we get to a later line population, we get an increase in the placebo rate.

That's been true of global studies in MG across the board. So we think we'll probably look more like the representative placebo effects we've seen in recent global MG studies, somewhere in the 2-3 range. Now, that could compress your delta for your MG-ADL, but there's also a strong anticipation that we'll see an increase in the active arm. If we look at the China data we've seen, there's only a few examples, but in the few examples we've seen of data that went from China to global, what we do see is a lower placebo in China and a higher placebo when we go global. But the delta tends to stay relatively intact because we also see an increase in the active arm as we go globally, probably because the placebo effect is affecting both the placebo arm and the active arm.