Enanta Pharmaceuticals is dedicated to developing innovative therapeutics through advanced chemistry and rigorous scientific research. The company has made significant strides in treating chronic hepatitis C virus (HCV) infections, having successfully developed combination therapies that have cured over a million patients. Enanta's pipeline includes a range of antiviral and immunology programs targeting diseases such as hepatitis B virus (HBV), respiratory syncytial virus (RSV), and COVID-19. Their approach emphasizes the creation of highly differentiated oral small molecules that address high unmet medical needs, showcasing their commitment to scientific excellence and impactful patient care.

In the field of immunology, Enanta is focusing on developing potent and selective oral inhibitors targeting key pathways involved in inflammatory diseases. Their initial programs include targeting the receptor tyrosine kinase KIT, which plays a crucial role in mast cell activation and survival, and the transcription factor STAT6, which is involved in type 2 immune responses. These programs aim to provide effective treatments for conditions like chronic spontaneous urticaria and atopic dermatitis, addressing significant gaps in current therapeutic options. Enanta's innovative approach not only seeks to alleviate symptoms but also aims to tackle the underlying causes of these diseases, thereby improving the quality of life for patients.

United States

Enanta Pharmaceuticals is using its robust chemistry-driven approach and drug discovery capabilities to become a leader in the discovery and development of small molecule drugs for viral infections and liver diseases. Enanta’s research and development efforts are focused on the following disease targets: respiratory syncytial virus (RSV), non-alcoholic steatohepatitis (NASH), hepatitis B virus (HBV), human metapneumovirus (hMPV) and SARS-CoV-2.

Infectious Diseases

Liver Diseases

Lung Diseases

Enanta Pharmaceuticals

Achromatopsia

Genetic disorder

Hepatitis C

Mavyret is a combination of 2 direct-acting antiviral agents, with distinct mechanisms of action. Glecaprevir is an inhibitor of the HCV NS3/4A protease, which is necessary for the proteolytic cleavage of the HCV encoded polyprotein and for viral replication. Pibrentasvir is an inhibitor of HCV NS5A, which is essential for viral RNA replication and virion assembly.

MAVYRET (Glecaprevir)

Respiratory syncytial virus

Respiratory virus

Viral infection

EDP-938, Enanta’s lead N-protein inhibitor, is being developed for the treatment of RSV infection. Enanta believes EDP-938 is differentiated from fusion inhibitors currently in development by others for RSV because this N-protein inhibitor targets the virus’ replication machinery and has demonstrated high barriers to resistance against the virus in vitro. EDP-938 has also been shown to reduce viral load below the level of detection in vivo. Additionally, it is possible that N-protein inhibitors may be effective treatments at later stages of infection.

Zelicapavir (EDP-938)

COVID-19

EDP-235 potently and selectively inhibits SARS-CoV-2 replication in &nbsp;multiple cellular models, including primary human airway epithelial &nbsp;cells, with an EC90 of 33nM. EDP-235 retained activity against protease &nbsp;enzymes from currently circulating SARS-CoV-2 variants. Importantly, &nbsp;EDP-235 has excellent lung distribution preclinically and demonstrates &nbsp;properties supportive of once daily oral dosing. Furthermore, EDP-235 &nbsp;has shown activity against other coronaviruses, providing the &nbsp;opportunity to potentially treat other infections that may emerge in the &nbsp;future.

EDP-235

EDP-323, a novel, oral direct-acting antiviral selectively targeting the RSV L-protein, which is essential for RSV replication. EDP-323 has shown sub-nanomolar potency against RSV-A and RSV-B&nbsp;in vitro&nbsp;and is not expected to have cross resistance to other classes of inhibitors. EDP-323 could be used alone or in combination with other agents, such as EDP-938, to potentially broaden the treatment window or addressable patient populations.

EDP-323 

Non-alcoholic steatohepatitis

EDP-297 is a potent FXR agonist and Enanta’s follow-on FXR agonist &nbsp;candidate being developed for the treatment of NASH. EDP-297 represents a &nbsp;class of FXR agonists that has been designed to take advantage of &nbsp;increased binding interactions with the receptor. Preclinical findings &nbsp;of EDP-297 demonstrate potent anti-fibrotic, anti-inflammatory and &nbsp;hepatoprotective effects. EDP-297 has demonstrated preclinical &nbsp;potency greater than that published on any FXR agonist in clinical &nbsp;development today. Further, in preclinical models EDP-297 has been shown &nbsp;to be targeted to tissues important for efficacy, namely liver and &nbsp;intestine, versus plasma and skin.
<a href="https://www.enanta.com/investors/news-releases/press-release/2020/Enanta-Pharmaceuticals-Initiates-Phase-1-Clinical-Study-of-EDP-297-its-Highly-Potent-and-Targeted-Follow-On-Farnesoid-X-Receptor-Agonist-for-the-Treatment-of-Non-Alcoholic-Steatohepatitis/default.aspx">Source</a>

ENP-297 (FXR inhibitor)

EDP-305 is a &nbsp;potent Farnesoid X receptor (FXR) agonist and Enanta’s lead product &nbsp;candidate being developed primarily for the treatment of NASH. FXR is a &nbsp;nuclear receptor and a main regulator of bile acid levels in the liver &nbsp;and small intestine. It responds to bile acids by regulating gene &nbsp;transcription of key enzymes and transporters, many of which play &nbsp;important roles in lipid metabolism, insulin resistance, inflammation, &nbsp;and fibrosis. EDP-305 represents a class of FXR agonists that has been &nbsp;designed to take advantage of increased binding interactions with the &nbsp;receptor. This non-bile acid class contains steroid and non-steroid &nbsp;components and does not contain the carboxylic acid group normally &nbsp;present in other classes of FXR agonists and natural bile acids that can &nbsp;lead to the formation of taurine and glycine conjugates.
<a href="https://www.enanta.com/investors/news-releases/press-release/2020/Enanta-Announces-Results-of-INTREPID-Study-of-EDP-305-for-the-Treatment-of-Primary-Biliary-Cholangitis/default.aspx">Source</a>

EDP-305 (FXR Agonist)

Hepatitis B

EDP-514, a &nbsp;novel class II hepatitis B virus (HBV) core inhibitor, is Enanta’s lead &nbsp;core inhibitor candidate. Core inhibitors, also known as capsid assembly &nbsp;modulators or core protein allosteric modulators, are a novel class of &nbsp;replication inhibitors that have been shown to act at multiple steps in &nbsp;the HBV lifecycle. Preclinical data demonstrate that EDP-514 is a potent &nbsp;inhibitor of HBV replication and prevents the denovo formation of new cccDNA in primary human hepatocytes when given early during HBV infection. In vitro data &nbsp;also show that EDP-514 is pan-genotypic, and that combinations of &nbsp;EDP-514 with nucleoside reverse-transcriptase inhibitors (NRTIs, current &nbsp;anti-viral therapies for HBV) or a class I core inhibitor result in &nbsp;additive to synergistic antiviral effects. ln vivo models of EDP-514 demonstrate excellent efficacy with &gt;4-log viral load reduction in HBV-infected PXB mice.
<a href="https://www.enanta.com/investors/news-releases/press-release/2019/Enanta-Pharmaceuticals-Initiates-Phase-1-Clinical-Study-of-EDP-514-its-Lead-Core-Inhibitor-for-the-Treatment-of-Hepatitis-B-Virus-HBV/default.aspx">Source</a>

Enanta Pharmaceuticals

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