LB Pharmaceuticals Inc Common Stock R&D Day 2026

Well, good afternoon, and welcome to the very first LB Pharma R&D Day. I am Heather Turner, CEO, and I am delighted that you all were able to navigate through the restaurant, through the TV set and to ultimately find your way to this hidden room here in N.Y. So thank you so much for being with us here today. Throughout this presentation, of course, we will be making forward-looking statements. Before we move into the agenda, I wanted to take a moment and reflect on the mission of LB Pharma. It is the North Star that guides us in the work that we do today.

I have had the privilege of leading this company for almost exactly two years to this day, and I have really developed a deep appreciation for the devastating impact that these brain disorders like schizophrenia, bipolar disorder, major depressive disorder, can have, not just on the patients who are suffering, but also their families, their care partners, and frankly, society. These are prevalent diseases. 3 million people in the U.S. suffer from schizophrenia, 7 million suffer from bipolar disorder, and 20 million suffer from major depressive disorder. I am sure that people in this room have friends or family members who have been and experienced the dimming of their lives that these types of disorders can accomplish. I am also certain that many of us know people who have been suffering from these disorders and can see firsthand the impact that they have on people's lives.

Our mission, as it is noted here, is to boldly advance high-impact therapies that will help people with brain disorders to live their life more fully, more brightly. Our aim is to alleviate the burden that many of these individuals feel, that their families feel, that their care partners feel. We have an amazing team. You will get to meet many of them throughout the day today, and you will see that this is a group that is passionate, they are experienced, and they are convicted in the pursuit that we have towards our mission. They are also fun to be around, quite honestly. Speaking today is our new CMO, Dr. Susan Kozauer, and our Chief Commercial Officer, Kaya Pai Panandiker. Gad Soffer and Joe Miller will also be available later today to answer questions.

You will also hear from our esteemed guest, Dr. Roger McIntyre, who is a Professor of Psychiatry and a Professor of Pharmacology and Toxicology at the University of Toronto. Let us turn to the agenda. I will be kicking off the day with a discussion around LB Pharma, as well as the LB-102 opportunity. Dr. Susan Kozauer will join and talk about the LB-102 clinical data, as well as some of the observations that we saw in our phase II NOVA-1 trial, and some of the additional research that we are going to be continuing to do. Dr. McIntyre is going to come up and present for the first time preclinical data supporting the potential differentiation of LB-102, and in particular, the observations that we observed in our phase II NOVA-1 trial.

He will also talk about the reasons to believe for that low rate of EPS, as well as the LB-102 mechanism. Ms. Kaya Pai Panandiker is going to come up and she'll talk about our approach to commercialization in schizophrenia. Then I'll come back and talk about what to expect with the NOVA-2 data readout. This is a snapshot of LB Pharma today. We have three important readouts over the next three years. We have our phase III NOVA-2 schizophrenia data reading out in 2027. We have our phase II bipolar depression data reading out in 2028, and we have the adjunctive MDD data reading out in 2029. All of these clinical trials are fully funded, and we have cash that takes us beyond the second quarter of 2029. What is LB-102? LB-102 is a structural modification of amisulpride.

Amisulpride is a generic antipsychotic that is available around the world, but was never approved as an antipsychotic in the U.S. It's a selective inhibitor of D2, D3, and 5-HT7, and it has very few other off-target effects. It's considered one of the most efficacious antipsychotics with one of the highest effect sizes, and it's also considered one of the more safe and tolerable antipsychotics with one of the lowest discontinuation rates. It does have limitations. It is actually very poor at permeating the blood-brain barrier. As a result, it requires high doses. For schizophrenia, it's dosed between 400 and 800 milligrams a day, and it also requires twice-daily dosing. It is a very widely prescribed antipsychotic. It was first approved in the late 1980s and continues to be used.

In 2023, you can see here on the left that almost 2 million prescriptions were written for amisulpride for a wide variety of indications, as noted in the pie chart. It has approvals in schizophrenia, negative symptoms of schizophrenia, as well as dysthymia, which is a persistent form of depression. The founders of LB Pharma set out to build and create a molecule that could address amisulpride's limitations, and they did this through methylating amisulpride. The resultant molecule is LB-102, and it's a molecule that has improved potency. What we determined was that 50 milligrams of LB-102 is about the same as 400 milligrams of amisulpride, and this is based on the human dopamine receptor occupancy data. Both of these doses have about 70% dopamine receptor occupancy. We also enabled once-daily dosing, and this is because there is a longer residence time in the CNS for LB-102.

We retained the binding profile of amisulpride, and this is important. LB-102, like amisulpride, is a selective inhibitor of D2, D3, and 5-HT7. This is important because it supports clinical development in a wide range of indications. D2 is, of course, important for treating psychosis and mania. D3 and 5-HT7 both are pathways that are related to depression, cognition, as well as anhedonia. LB-102 also has a unique mechanism that it shares with amisulpride, and that is it preferentially engages with the presynaptic autoreceptors that are implicated in mood, anhedonia, and cognition at lower doses. This is actually a part of the preclinical data that Dr. Roger McIntyre will be presenting later. This is important because what it enables is a treatment of a wide variety of symptoms through dose.

What you see with amisulpride is that at its highest doses, which are reflected here in the red, it is used to treat psychosis, schizophrenia, psychotic related disorders. At its lowest doses, which are noted here in the yellow, it is used to treat depression. So at the high doses, amisulpride is used at 400 to 800 milligrams a day. For depression, amisulpride is used between 50 and 100 milligrams a day. Emerging from the NOVA-1 clinical trial, we believe is a profile for LB-102 that supports an opportunity for it to be the branded antipsychotic of choice. We noted and we observed a compelling efficacy profile, one that was reflected by a clinically meaningful reduction in the PANSS total score, as well as a very strong, robust treatment effect. In addition, we saw an effect as early as week 1, which we know is an attractive aspect for prescribers.

We also believe we have the potential for a class-leading safety profile. This is based on the low rate of EPS that we observed, the minimal QT prolongation, and the negligible sedation. We also had very few GI side effects. In addition, there are opportunities to potentially demonstrate a benefit in two significant areas of unmet need. The first is cognition and the second is negative symptoms. With respect to negative symptoms in the NOVA-1 trial, we observed a statistically significant effect on the PANSS negative symptom subscale at the 50-milligram dose. This is consistent with that mechanism that we just talked about. With respect to cognition, we utilized Cogstate to assess a battery of tests, and we found a robust dose-dependent treatment effect on cognition. A benefit in either one of these areas of unmet need will be very attractive to prescribers.

In addition to all of that, we have a very simple-to-use profile. This is once-daily dosing. We are not expecting a food effect, no challenging drug-drug interactions, and all patients start at a therapeutic dose. There is no titration. Our goal in all of this is to really transform what is possible for people with schizophrenia. Too few people achieve long-lasting relief. 73% of patients do not experience a long-lasting quality of life. To us, this is unacceptable, and we are focused on changing that. I will now bring up Dr. Kozauer to talk through the clinical profile of LB-102, as well as why we believe we have an opportunity for meaningful differentiation. Great. Whoops. Okay. Excuse me.

That was graceful. Thank you, Heather, very much.

I will be running through the development program with you at this point. First, I want to just take a minute to tell you just a little bit about myself. I am the newbie here. I have been CMO here, at LB for 44 days. What I will tell you about that, however, is that with every day, I am just increasingly excited about the potential of LB-102 and what it can bring to our patients. I started my career a few decades ago as an inpatient psychiatrist, treating the most severely mentally ill population. Quite honestly, I wish I had a drug that had the qualities of LB-102 in my toolbox back then. As we go through this presentation, I think you'll understand why. We're right in the middle of a development program in a range of neuropsychiatric disorders.

The left side of the slide here shows our completed clinical trials. We've got a phase I single ascending and multiple ascending dose study in healthy volunteers, a phase I-B PET receptor occupancy trial, and a phase II trial, NOVA-1, in patients experiencing an acute exacerbation of schizophrenia, all complete. On the right side of the slide, we have our ongoing and planned clinical trials. We've got NOVA-2, our phase III trial in schizophrenia. Those patients, as well as some de novo patients, can roll into NOVA-3, our open label 52-week safety and tolerability study. In addition to that, moving away from schizophrenia, we also have our phase II study, ILLUMINATE-1, in bipolar depression. Finally, we have a phase II trial in adjunctive MDD, which is slated to start early next year.