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Last Updated: January 20, 2025

Investigational Drug Information for SEP-363856


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What is the drug development status for SEP-363856?

SEP-363856 is an investigational drug.

There have been 26 clinical trials for SEP-363856. The most recent clinical trial was a Phase 2 trial, which was initiated on September 11th 2019.

The most common disease conditions in clinical trials are Schizophrenia, Disease, and Narcolepsy. The leading clinical trial sponsors are Sunovion, Sumitomo Dainippon Pharma Co., Ltd., and Sumitomo Pharma Co., Ltd.

There is one US patent protecting this investigational drug and sixty-six international patents.

Recent Clinical Trials for SEP-363856
TitleSponsorPhase
A Clinical Study to Learn if SEP-363856 Has Physical Dependence in Adults With SchizophreniaSunovionPhase 1
An Extension Study to a Clinical Study That Will Continue to Evalute the Effectiveness and Safety of SEP-363856 in People With Schizophrenia That Switch to SEP-363856 From Their From Their Current Antisychotic MedicationSunovionPhase 3
A Clinical Study That Will Meaure How Well SEP-363856 Works and How Safe it is in Adults With Generalized Anixety DisorderSunovionPhase 2/Phase 3

See all SEP-363856 clinical trials

Clinical Trial Summary for SEP-363856

Top disease conditions for SEP-363856
trials024681012141618202224SchizophreniaDiseaseNarcolepsyCataplexy[disabled in preview]
Top clinical trial sponsors for SEP-363856
trials024681012141618202224SunovionSumitomo Dainippon Pharma Co., Ltd.Sumitomo Pharma Co., Ltd.[disabled in preview]

See all SEP-363856 clinical trials

US Patents for SEP-363856

DrugnamePatent NumberPatent TitlePatent AssigneeEstimated Expiration
SEP-363856 ⤷  Subscribe Multicyclic compounds and methods of use thereof Sunovion Pharmaceuticals Inc. (Marlborough, MA) PGI Drug Discovery LLC. (Tarrytown, NY) ⤷  Subscribe
>Drugname>Patent Number>Patent Title>Patent Assignee>Estimated Expiration
Showing 1 to 1 of 1 entries

International Patents for SEP-363856

DrugnameCountryDocument NumberEstimated ExpirationRelated US Patent
SEP-363856 Australia AU2010325925 2029-12-04 ⤷  Subscribe
SEP-363856 Australia AU2016200448 2029-12-04 ⤷  Subscribe
SEP-363856 Australia AU2017248551 2029-12-04 ⤷  Subscribe
SEP-363856 Brazil BR112012013431 2029-12-04 ⤷  Subscribe
SEP-363856 Brazil BR122021013836 2029-12-04 ⤷  Subscribe
SEP-363856 Canada CA2781716 2029-12-04 ⤷  Subscribe
>Drugname>Country>Document Number>Estimated Expiration>Related US Patent
Showing 1 to 6 of 6 entries

SEP-363856: A Revolutionary Drug Candidate for Schizophrenia Treatment

Introduction

Schizophrenia, a complex and debilitating mental health disorder, has seen limited advancements in treatment options since the introduction of antipsychotic medications in the 1950s. However, a new drug candidate, SEP-363856, is poised to change this landscape with its novel mechanism of action and promising clinical results.

Discovery and Mechanism of Action

SEP-363856 was discovered through a collaborative effort between Sunovion and PsychoGenics, utilizing a mechanism-independent approach with the in vivo phenotypic SmartCube® platform and associated artificial intelligence algorithms. Unlike traditional antipsychotics that rely on dopamine D2 receptor blockade, SEP-363856 does not bind to D2 or serotonin 2A (5-HT2A) receptors. Instead, it activates trace amine-associated receptor 1 (TAAR1) and serotonin 1A (5-HT1A) receptors, offering a unique and potentially side-effect-reduced treatment option[2][4].

Preclinical and Clinical Trials

Preclinical studies have shown that SEP-363856 demonstrates broad efficacy in rodent models related to schizophrenia, including phencyclidine (PCP)-induced hyperactivity, prepulse inhibition, and PCP-induced deficits in social interaction. These studies highlighted its favorable pharmacokinetic properties and the absence of catalepsy, a common side effect of traditional antipsychotics[1].

Clinical trials, particularly the pivotal Phase 2 study (SEP361-201), have been highly encouraging. This randomized, placebo-controlled, double-blind study showed that patients with acute exacerbation of schizophrenia treated with SEP-363856 experienced statistically significant and clinically meaningful improvements in the Positive and Negative Syndrome Scale (PANSS) total score and the Clinical Global Impression Scale – Severity (CGI-S) compared to placebo[2][4].

FDA Breakthrough Therapy Designation

The FDA granted SEP-363856 Breakthrough Therapy Designation based on the positive results from the Phase 2 studies. This designation underscores the potential of SEP-363856 as a novel treatment for schizophrenia, a condition that has seen few major advances in treatment since the advent of antipsychotic pharmacotherapy in the 1950s[2].

Safety Profile

The safety analysis of SEP-363856 presented at the 2021 American Psychiatric Association Virtual Annual Meeting highlighted a differentiated side effect profile compared to currently approved atypical antipsychotics. The cumulative rate and patterns of adverse events were distinctly different for SEP-363856, suggesting a more favorable safety profile[3].

Impact on Negative Symptoms

SEP-363856 has also shown promise in addressing the negative symptoms of schizophrenia, which are often resistant to treatment with current antipsychotics. Short-term and long-term treatment with SEP-363856 resulted in improvements in the Brief Negative Symptom Scale (BNSS) total score and BNSS factor scores, indicating its potential to address a significant unmet need in schizophrenia treatment[3].

Market Projection

Given its unique mechanism of action, favorable safety profile, and positive clinical results, SEP-363856 is poised to make a significant impact in the market for schizophrenia treatments. Here are a few key projections:

Market Need

Schizophrenia affects approximately 2.4 million people in the U.S., and the current treatment options often come with substantial side effects and limited efficacy in treating negative and cognitive symptoms. SEP-363856 addresses this unmet need, offering a new class of medicines that could improve the quality of life and functional skills of patients[2].

Competitive Advantage

SEP-363856's non-D2 mechanism of action sets it apart from traditional antipsychotics, potentially reducing side effects such as weight gain, metabolic issues, and extrapyramidal symptoms. This could make it a preferred option for both patients and healthcare providers[1][2].

Regulatory Pathway

With the FDA Breakthrough Therapy Designation, SEP-363856 is on an accelerated regulatory pathway. This designation is expected to expedite the development and review process, potentially leading to earlier market approval[2].

Global Development

Sunovion has exclusive rights to develop and commercialize SEP-363856 globally. The drug is being studied in a global development program for schizophrenia and is also under consideration for other indications, such as Parkinson’s disease psychosis[4].

Conclusion

SEP-363856 represents a groundbreaking advancement in the treatment of schizophrenia, offering a novel mechanism of action that avoids the limitations and side effects associated with traditional antipsychotics. With its favorable safety profile, positive clinical results, and regulatory advantages, SEP-363856 is well-positioned to transform the treatment landscape for schizophrenia and potentially other neuropsychiatric disorders.

Key Takeaways

  • Unique Mechanism of Action: SEP-363856 activates TAAR1 and 5-HT1A receptors, distinct from traditional D2 receptor blockade.
  • Positive Clinical Results: Statistically significant improvements in PANSS and CGI-S scores in clinical trials.
  • FDA Breakthrough Therapy Designation: Accelerated regulatory pathway due to its potential to address an unmet medical need.
  • Favorable Safety Profile: Differentiated side effect profile compared to current antipsychotics.
  • Global Development: Being studied globally for schizophrenia and other potential indications.

FAQs

What is the mechanism of action of SEP-363856?

SEP-363856 activates trace amine-associated receptor 1 (TAAR1) and serotonin 1A (5-HT1A) receptors, unlike traditional antipsychotics that block dopamine D2 receptors[1][2].

Has SEP-363856 shown efficacy in clinical trials?

Yes, SEP-363856 has demonstrated statistically significant and clinically meaningful improvements in the Positive and Negative Syndrome Scale (PANSS) total score and the Clinical Global Impression Scale – Severity (CGI-S) in clinical trials[2][4].

What is the FDA's stance on SEP-363856?

The FDA has granted SEP-363856 Breakthrough Therapy Designation, indicating its potential to address an unmet medical need and expediting its development and review process[2].

How does the safety profile of SEP-363856 compare to current antipsychotics?

SEP-363856 has a differentiated side effect profile compared to currently approved atypical antipsychotics, with a lower cumulative rate and different patterns of adverse events[3].

What are the potential market implications of SEP-363856?

SEP-363856 could significantly impact the market for schizophrenia treatments by offering a new class of medicines with a favorable safety profile and efficacy in addressing negative symptoms, potentially improving the quality of life for patients[2][3].

Sources

  1. PubMed: SEP-363856, a Novel Psychotropic Agent with a Unique, Non-D2 Mechanism of Action.
  2. Sunovion and PsychoGenics: SEP-363856 Has Received FDA Breakthrough Therapy Designation for the Treatment of People with Schizophrenia.
  3. Psychiatric Times: Late-Stage Pipeline Candidate Looks Promising for the Treatment of Schizophrenia.
  4. Drug Discovery News: SEP-363856 successful against schizophrenia.

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