We are a clinical-stage biopharmaceutical company developing novel therapies for the treatment of schizophrenia, bipolar depression, and other neuropsychiatric diseases. We are building a pipeline that leverages the broad therapeutic potential of our lead product candidate, LB-102, which we believe has the potential to be the first benzamide antipsychotic drug approved for neuropsychiatric disorders in the United States. LB-102 is a new chemical entity and a methylated derivative of amisulpride, a second-generation antipsychotic drug approved in over 50 countries, not including the United States, because the development and regulatory requirements of the U.S. Food and Drug Administration, or FDA, for amisulpride were incompatible with patent coverage on the drug. Amisulpride is a generic drug that has been extensively used in clinical practice following its initial approval in France in the 1980s, generating at least two million monthly prescriptions in 2023 in a subset of 16 continental European countries. Among these European prescriptions for amisulpride, our data suggest that approximately 60% are for schizophrenia and schizoaffective disorders, approximately 20% are for mood disorders, approximately 14% are for anxiety, and the remainder are for a variety of other indications. We developed LB-102 in order to address the limitations of amisulpride with a differentiated therapeutic profile and strong intellectual property protection. We believe LB-102’s mechanism of action, data from our recently completed Phase 2 trial of LB-102 in acute schizophrenia, and the legacy of clinical experience with amisulpride support the continued development of LB-102 in schizophrenia and bipolar depression. If successful in treating bipolar depression, we may also develop LB-102 in the treatment of major depressive disorder, or MDD. In the future, we may also develop LB-102 for the treatment of other neuropsychiatric disorders, including schizophrenia with predominantly negative symptoms, Alzheimer’s disease-related agitation and psychosis, manic episodes associated with bipolar disorder, and cognitive impairment associated with schizophrenia, or CIAS. We believe that LB-102, if approved, can become a mainstay of psychiatric practice by offering a potentially attractive alternative to branded and generic therapeutics for the treatment of schizophrenia, bipolar depression, and other neuropsychiatric diseases. Our product candidate, LB-102, is a Phase 3-ready oral, small molecule for the treatment of acute schizophrenia, defined as a sudden and severe episode of psychotic symptoms, characterized by hallucinations, delusions, and other positive symptoms. Schizophrenia is a chronic, severe, complex, and debilitating psychiatric disorder that affects approximately 1% of the U.S. population and is a leading cause of disability. Symptoms are divided into three categories: (i) psychotic or positive symptoms, which include delusions, hallucinations, thought disorder, and movement disorder; (ii) negative symptoms, which include lack of motivation, interest, or enjoyment in daily activities, withdrawal from social life, and difficulty showing emotions; and (iii) cognitive symptoms, which encompass problems with attention, concentration, and memory. The disease is associated with increased mortality, with approximately 5% of schizophrenia patients dying by suicide, and average overall life expectancy decreasing by as much as 29 years compared to the general population. There is currently no cure for schizophrenia, which means the disease must be managed with life-long therapy, increasing the importance of therapies that can improve compliance rates and dosing challenges. In January 2025, we announced positive data from our four-week placebo-controlled, double-blinded, Phase 2 trial in the United States, which assessed the safety and efficacy of LB-102 in patients with acute schizophrenia. Results from the trial demonstrated (i) statistically significant clinical activity at all LB-102 doses tested; (ii) a significant average change in overall symptoms (effect size); (iii) a potentially class-leading tolerability profile among D2/D3 antagonists and partial agonists; and (iv) a potentially differentiated impact on cognition as measured by CogState Computerized Schizophrenia Battery of Tests. The trial achieved its primary endpoint of change in the Positive and Negative Syndrome Scale, or PANSS, a 30-item scale that measures the severity of schizophrenia symptoms, from baseline to Week 4. A statistically significant decrease in symptoms was observed for all three dose cohorts (50 mg, 75 mg, and 100 mg) compared to placebo. Additionally, our Phase 2 trial data showed a statistically significant impact on negative symptoms versus placebo at the 50 mg dose even though the inclusion criteria enriched for patients experiencing predominantly positive symptoms of schizophrenia. An exploratory post-hoc analysis of our Phase 2 data on the treatment effect in patients with negative symptoms at baseline (i.e., those patients with a PANSS Negative Subscore greater than or equal to 24) yielded similar results with a statistically significant impact on negative symptoms versus placebo at the 50 mg dose. LB-102 was generally well tolerated in the clinical trial, with adverse events being mostly transient and mild to moderate in severity. If replicated in our planned Phase 3 trial, we believe this tolerability profile has the potential to be class-leading among D2/D3 antagonists and partial agonists specifically with respect to the rate of sedation and extrapyramidal symptoms, or EPS, a group of movement disorders including involuntary movements, muscle stiffness, and tremors, that, together with sedation, are quite burdensome to patients and can result in discontinuation of treatments. The impact of LB-102 on cognitive function was also evaluated as an exploratory endpoint in this trial. After four weeks of treatment with LB-102, a robust, dose-dependent, and significant treatment effect size was identified in a post-hoc analysis in the completer population for all doses of LB-102 compared with placebo. We designed our Phase 2 acute schizophrenia trial to be potentially registrational by including a large sample size (n=359), robust statistical analyses, as well as numerous sensitivity analyses. Based on positive end-of-Phase 2 feedback from the FDA, as well as historical precedent, we believe that our Phase 2 acute schizophrenia trial may serve as one of the two pivotal trials required for approval of a new drug application, or NDA, in the United States. As a result, we believe there is a viable path to approval of LB-102 in the United States for the treatment of schizophrenia with a single, six-week Phase 3 trial alongside other planned NDA-enabling studies. However, there is no guarantee that our Phase 2 trial may serve as one of the two pivotal trials required for FDA approval, and in such case, we may be required to conduct an additional pivotal trial in acute schizophrenia. The adequacy of our Phase 2 trial to support registration will be a matter of review by the FDA at the time of NDA submission and will depend on the totality of the data included in our submission, including the results of our planned Phase 3 trial. We are planning to initiate a six-week Phase 3 trial of LB-102 in acute schizophrenia patients in the first quarter of 2026, which we believe, if positive, could be sufficient to support a regulatory application for approval in the United States along with our completed Phase 2 trial and other planned NDA-enabling studies. The Phase 3 trial is anticipated to be a three-arm, inpatient, double-blinded, placebo-controlled, oral once-daily dose of LB-102 in patients with acute schizophrenia, with a six-week treatment duration. We plan to study the effects of 50 mg LB-102 or 100 mg LB-102 versus placebo in this trial, and patients will be randomized in a 1:1:1 ratio across the three arms of the trial. The sample size will be approximately 400 patients, and we plan to conduct this trial at approximately 25 sites entirely in the United States. The primary endpoint of the trial is anticipated to be change from baseline in PANSS at Day 42. We expect to disclose topline data from this Phase 3 trial in the second half of 2027 and, if positive, meet with the FDA in the first quarter of 2028 to discuss the potential for submission of an NDA. In addition to our clinical development program in schizophrenia, we plan to leverage our expertise in neuropsychiatry and the unique mechanism of action of LB-102 to develop our product candidate in other indications, starting with bipolar depression. Most people living with bipolar depression experience dramatic shifts in mood, energy, and behavior, alternating between manic and depressive states. It is estimated that 2.8%, or approximately seven million Americans, experience bipolar disorder in a year, and approximately 40 million people live with bipolar disorder worldwide. Our initial Phase 2 trial will explore the utility of LB-102 in controlling the depressive symptoms of the disease. We plan to initiate this potentially registrational Phase 2 trial in bipolar depression in the first quarter of 2026, with topline data expected in the first quarter of 2028. We believe LB-102’s strong antagonism of the D2, D3, and 5HT7 receptors makes it well suited for treating bipolar depression, providing potential to control psychosis and mania through its effects on D2 and potential for antidepressive and pro-cognitive effects through its antagonism of 5HT7 and D3. Our Phase 2 trial of LB-102 in acute schizophrenia demonstrated strong antipsychotic activity and suggests opportunities for potential differentiation in bipolar depression given the observed tolerability profile (low rates of EPS, sedation, and gastrointestinal side effects) and positive impact on cognition. Amisulpride is approved for the treatment of dysthymia, a form of depression, in certain countries outside of the United States and has been shown to be as effective as certain approved agents for MDD and dysthymia. We believe that results in dysthymia and MDD provide strong scientific and clinical rationale for development of LB-102 in the treatment of depressive episodes associated with bipolar disorder or bipolar depression because episodes of major depression, whether unipolar (as in MDD) or bipolar (as in bipolar depression), are typically characterized by a similar imbalance in the neurotransmitters serotonin, noradrenaline, and dopamine, regardless of the underlying pathophysiology of the disease. There is wide use of amisulpride in bipolar disorder with approximately 3.4% of at least two million monthly prescriptions written for this indication in a select group of European countries including Germany, France, Italy, Spain, and several others. A non-racemic form of amisulpride also showed antidepressant activity in two independent third-party, placebo-controlled bipolar depression trials with an approximately 17- to 18-point reduction in Montgomery–Åsberg Depression Rating Scale, or MADRS, from baseline observed across these studies. Additionally, among the four antipsychotics approved for schizophrenia and MDD or treatment resistant depression that were also studied in late-stage bipolar depression trials (quetiapine, cariprazine, aripiprazole, and olanzapine), three out of four, or 75%, generated positive data for the treatment of bipolar depression. Our planned Phase 2 trial for bipolar depression will utilize a fixed-flexible dose of LB-102. This trial design allows us to evaluate two doses of LB-102 in the trial, thereby increasing the chances for a patient to derive clinical benefit from treatment with LB-102, while retaining the advantages of a two-arm trial, which is known to mitigate the risk of a high placebo rate. Additionally, flexible dose trials typically have better signal detection than fixed dose trials for depression, as flexible dose trials lower the magnitude of symptom reduction with placebo. We believe LB-102 has the potential to provide improved tolerability and clinical activity in bipolar depression compared to currently available treatments worldwide, which are associated with troubling adverse events and insufficient efficacy for certain symptoms, including cognitive impairment associated with the disease. We are also developing a long-acting injectable, or LAI, formulation of LB-102, which may improve compliance, a common issue in patients with schizophrenia and bipolar disorder. We believe an effective LAI form of LB-102 has the potential to benefit patients worldwide, as relatively few approved agents are available as long-acting formulations and there are no benzamide class LAIs currently available or in development worldwide. The American Psychiatric Association recommends injectable formulations in circumstances where doing so will improve adherence, decrease mortality, reduce hospitalization risk, and decrease treatment discontinuation rates. We have commenced LAI formulation development and expect to continue these efforts in 2026. The U.S. market for branded antipsychotic drugs was approximately $12 billion as of 2024. Despite the widespread use of generic antipsychotic drugs, several of these branded drugs each generate U.S. sales in excess of $1 billion annually. Additionally, while available therapeutics to treat schizophrenia and bipolar depression demonstrate clinical benefit, a significant unmet need remains for a treatment that balances tolerability with clinically meaningful efficacy for the chronic management of symptoms related to both psychosis and mood disorders. Our principal executive office is located in New York, NY.
