Glecaprevir/Pibrentasvir

WATERTOWN, Mass.--( BUSINESS WIRE )-- Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for virology and immunology indications, today announced the company will hold a conference call and webcast on Monday, December 9 at 8:30 a.m. ET to share topline results from RSVPEDs, a first-in-pediatrics Phase 2 study evaluating zelicapavir in hospitalized and non-hospitalized children aged 28 days to 36 months with respiratory syncytial virus (RSV). Conference Call and Webcast Information The live webcast on Monday, December 9 at 8:30 a.m. ET can be accessed at " Events & Presentations " in the investors section of Enanta’s website. To participate by phone, please register for the call here . It is recommended that participants register a minimum of 15 minutes before the call. Once registered, participants will receive an email with the dial-in information. The archived webcast will be available on Enanta’s website for approximately 30 days following the event. About Zelicapavir Zelicapavir, Enanta’s lead N-protein inhibitor, is being developed for the treatment of RSV infection, and has been granted Fast Track designation by the U.S. Food and Drug Administration. Zelicapavir is a nanomolar inhibitor of both RSV-A and RSV-B activity. Zelicapavir is differentiated from RSV fusion inhibitors as the N-protein inhibitor targets the virus’ replication machinery and has demonstrated a high barrier to resistance in vitro. In preclinical studies, zelicapavir maintained antiviral potency across all clinical isolates tested and was active against viral variants resistant to other mechanisms. Zelicapavir demonstrated a favorable safety, pharmacokinetic and drug-drug interaction profile in an extensive Phase 1 program. In a Phase 2 challenge study, zelicapavir achieved highly statistically significant (p<0.001) reductions in RSV viral load and clinical symptoms compared to placebo and was safe and well-tolerated, with infrequent adverse events. Zelicapavir is currently being evaluated in RSVHR, a Phase 2b study in the elderly and/or those with congestive heart failure, chronic obstructive pulmonary disease (COPD) or asthma. About Respiratory Syncytial Virus RSV is the most common cause of bronchiolitis (inflammation of the small airways in the lung) and pneumonia in children under one year of age in the United States and a significant cause of respiratory illness in older adults and immunocompromised individuals. 1 According to the Centers for Disease Control and Prevention, virtually all children in the United States get an RSV infection by the time they are two years old and one to two out of every 100 children younger than six months of age with an RSV infection may need to be hospitalized. 2 Globally, there are an estimated 33 million cases of RSV annually in children less than five years of age, with about 3 million hospitalized and up to approximately 100,000 dying each year from complications associated with the infection. 3 RSV represents a significant health threat for adults older than 65 years of age, with an estimated 177,000 hospitalizations and 14,000 deaths associated with RSV infections annually in the United States. 4 About Enanta Pharmaceuticals, Inc. Enanta is using its robust, chemistry-driven approach and drug discovery capabilities to become a leader in the discovery and development of small molecule drugs with an emphasis on indications in virology and immunology. Enanta’s clinical programs are currently focused on respiratory syncytial virus (RSV) and its earlier-stage immunology pipeline aims to develop treatments for inflammatory diseases by targeting key drivers of the type 2 immune response, including KIT and STAT6 inhibition. Glecaprevir, a protease inhibitor discovered by Enanta, is part of one of the leading treatment regimens for curing chronic hepatitis c virus (HCV) infection and is sold by AbbVie in numerous countries under the tradenames MAVYRET ® (U.S.) and MAVIRET ® (ex-U.S.) (glecaprevir/pibrentasvir). A portion of Enanta’s royalties from HCV products developed under its collaboration with AbbVie contribute ongoing funding to Enanta’s operations. Please visit www.enanta.com for more information. Forward Looking Statements Disclaimer This press release contains forward-looking statements, including with respect to the prospects for further development and advancement of zelicapavir for the treatment of RSV. Statements that are not historical facts are based on management’s current expectations, estimates, forecasts and projections about Enanta’s business and the industry in which it operates and management’s beliefs and assumptions. The statements contained in this release are not guarantees of future performance and involve certain risks, uncertainties and assumptions, which are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. Important factors and risks that may affect actual results include: the development risks of early stage discovery efforts in the disease areas in Enanta’s research and development pipeline, such as RSV; the impact of development, regulatory and marketing efforts of others with respect to competitive treatments for RSV; Enanta’s limited clinical development experience; Enanta’s need to attract and retain senior management and key scientific personnel; Enanta’s need to obtain and maintain patent protection for its product candidates and avoid potential infringement of the intellectual property rights of others; and other risk factors described or referred to in “Risk Factors” in Enanta’s most recent Annual Report on Form 10-K for the fiscal year ended September 30, 2024 and other periodic reports filed more recently with the Securities and Exchange Commission. Enanta cautions investors not to place undue reliance on the forward-looking statements contained in this release. All forward-looking statements contained in this release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Enanta undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Centers for Disease Control & Prevention – Respiratory Syncytial Virus Last accessed: December 2024. Centers for Disease Control & Prevention – RSV in Infants and Young Children Last accessed: December 2024. Shi, Ting et al. “ Global, regional, and national disease burden estimates of acute lower respiratory infections due to respiratory syncytial virus in young children in 2015: a systematic review and modelling study .” Lancet (London, England) vol. 390,10098 (2017): 946-958. doi:10.1016/S0140-6736(17)30938-8 Falsey, Ann R et al. “ Respiratory syncytial virus infection in elderly and high-risk adults. ” The New England Journal of Medicine vol. 352,17 (2005): 1749-59. doi:10.1056/NEJMoa043951

WATERTOWN, Mass.--( BUSINESS WIRE )-- Enanta Pharmaceuticals, Inc. (NASDAQ:ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for virology and immunology indications, today reported financial results for its fiscal fourth quarter and year-ended September 30, 2024. “Our fiscal fourth quarter was an exciting time for Enanta as we announced positive data from a Phase 2a human challenge study of EDP-323, our RSV L-inhibitor. We believe these results are among the strongest ever reported for an antiviral in an RSV challenge study, and significantly unlock further promise of our RSV program, in addition to advancing our leadership role in the RSV treatment landscape,” said Jay R. Luly, Ph.D., President and Chief Executive Officer at Enanta Pharmaceuticals. “With results from our RSVPEDs study of zelicapavir, our RSV N-inhibitor, expected in December, we could potentially have two of the leading clinical candidates for the treatment of RSV with different mechanisms of action, providing us with important optionality. The results of these studies will guide our decisions as we work to develop first-in-disease and best-in-class treatments for patients suffering from RSV.” Dr. Luly added, “We also made notable progress in advancing and expanding our immunology portfolio with the nomination of EPS-1421 as our lead development candidate for our KIT inhibition program. We are excited about the potential for potent and selective, oral, small molecule inhibitors for the treatment of chronic spontaneous urticaria and possibly other mast cell driven diseases. We are also pleased to introduce our second discovery stage program to develop oral STAT6 inhibitors for the treatment of type 2 immune driven diseases, with an initial focus on the treatment of atopic dermatitis, and future expansion opportunities in asthma and other indications. With multiple ongoing programs in both virology and immunology, we are committed to advancing our pipeline to help patients and to create value for shareholders.” Fiscal Fourth Quarter and Year-Ended September 30, 2024 Financial Results Total revenue was $14.6 million for the three months ended September 30, 2024, which consisted of royalty revenue derived from worldwide net sales of AbbVie’s hepatitis C virus (HCV) regimen MAVYRET ® /MAVIRET ® , compared to royalty revenue of $18.9 million for the three months ended September 30, 2023. For the twelve months ended September 30, 2024, total revenue was $67.6 million compared to $79.2 million for the same period in 2023. The decrease in the quarter and in year-over-year revenue is due to a decline in AbbVie’s sales of MAVYRET ® /MAVIRET ® . A portion (54.5%) of Enanta’s ongoing royalty revenue from AbbVie’s net sales of MAVYRET ® /MAVIRET ® is paid to OMERS, one of Canada’s largest defined benefit pension plans, pursuant to a royalty sale transaction affecting royalties earned after June 2023. For financial reporting purposes, the transaction was treated as debt, with the upfront purchase payment of $200.0 million recorded as a liability. Each quarter, Enanta records 100% of the royalty earned as revenue and then amortizes the debt liability proportionally as 54.5% of the cash royalty payments are paid to OMERS through June 30, 2032, subject to a cap of 1.42 times the purchase payment, after which point 100% of the cash royalty payments will be retained by Enanta. Interest expense was $2.6 million for the three months ended September 30, 2024 and $10.9 million for the twelve months ended September 30, 2024. This compares to interest expense of $3.2 million for the three months ended September 30, 2023 and $5.1 million for the twelve months ended September 30, 2023. Research and development expenses were $30.8 million for the three months ended September 30, 2024, compared to $36.2 million for the three months ended September 30, 2023. For the twelve months ended September 30, 2024, research and development expenses were $131.5 million compared to $163.5 million for the same period in 2023. The decrease in the quarter and in year-over-year research and development expenses is primarily due to a decrease in costs associated with Enanta’s COVID-19 program as the company announced previously that plans to pursue any further COVID-19 efforts would be in the context of collaborations. These decreases were partially offset by increased costs associated with Enanta’s immunology programs. General and administrative expenses totaled $13.7 million for the three months ended September 30, 2024, compared to $13.8 million for the three months ended September 30, 2023. For the twelve months ended September 30, 2024, general and administrative expenses were $57.9 million compared to $52.9 million in 2023. The increase in year-over-year general and administrative expenses was due to an increase in legal fees related to the company’s patent infringement suit against Pfizer. Interest and investment income, net, totaled $3.2 million for the three months ended September 30, 2024, compared to $4.7 million for the three months ended September 30, 2023. The decrease was due to lower cash and investment balances year-over-year. For the twelve months ended September 30, 2024, interest and investment income, net, totaled $14.8 million compared to $11.4 million in 2023. The increase was due to an increase in average invested cash due to receipt of $200.0 million from OMERS in April 2023 as well as changes in interest rates year-over-year. Enanta recorded an income tax benefit of $0.4 million for the three months ended September 30, 2024, compared to an income tax benefit of $1.4 million for the three months ended September 30, 2023. Enanta recorded an income tax benefit of $1.7 million for the twelve months ended September 30, 2024, compared to an income tax expense of $2.8 million for the twelve months ended September 30, 2023. The income tax benefit during 2024 was due to interest earned on a pending $28.7 million federal income tax refund. Despite recording a loss before taxes during the twelve months ended September 30, 2023, Enanta recorded tax expense driven by the receipt of the $200.0 million from OMERS, which is treated as income for Federal and State income tax purposes. Net loss for the three months ended September 30, 2024, was $28.8 million, or a loss of $1.36 per diluted common share, compared to a net loss of $28.1 million, or a loss of $1.33 per diluted common share, for the corresponding period in 2023. For the twelve months ended September 30, 2024, net loss was $116.0 million, or a loss of $5.48 per diluted common share, compared to a net loss of $133.8 million, or loss of $6.38 per diluted common share for the corresponding period in 2023. Enanta’s cash, cash equivalents and marketable securities totaled $248.2 million at September 30, 2024. Enanta expects that its current cash, cash equivalents and short-term marketable securities, as well as its continuing retained portion of royalty revenue, will continue to be sufficient to meet the anticipated cash requirements of its existing business and development programs into fiscal 2027. Virology RSV Enanta is progressing multiple clinical programs comprising a robust antiviral portfolio aimed at treating populations at high-risk for serious outcomes from RSV infection. This includes zelicapavir, Enanta’s lead, oral N-protein inhibitor, and EDP-323, its oral L-protein inhibitor, both of which received Fast Track designation from the U.S. Food and Drug Administration (FDA). Zelicapavir is being evaluated in two Phase 2 clinical trials in high-risk pediatric and adult populations. Enrollment is complete in RSVPEDs, a first-in-pediatrics Phase 2, randomized, double-blind, placebo-controlled study of zelicapavir in hospitalized and non-hospitalized RSV patients that are 28 days to three years of age. The company is on track to report topline data in December 2024. RSVHR is a Phase 2b, randomized, double-blind, placebo-controlled study of zelicapavir in adults with RSV infection who are at high risk of complications, including age over 65 years and/or those with congestive heart failure, chronic obstructive pulmonary disease or asthma. Enrollment in RSVHR is progressing, and the company is targeting enrollment completion in the current Northern Hemisphere RSV season. Enanta’s second clinical RSV candidate, EDP-323, is a novel oral, direct-acting antiviral selectively targeting the RSV L-protein. In September 2024, Enanta announced positive topline results for EDP-323 in a Phase 2a challenge study of healthy adults infected with RSV. Treatment with EDP-323 achieved statistically significant (p=<0.0001) reductions in both viral load and clinical symptoms compared to placebo. Overall, EDP-323 was generally well-tolerated and demonstrated a favorable safety profile that was comparable to placebo over 5 days of dosing through Day 28 of follow-up. There were no serious adverse events and no discontinuations of EDP-323. With these positive results the company has a potential second approach for treating RSV that may offer a best-in-disease opportunity. Pending RSVPEDs data results, Enanta will provide next steps for EDP-323 and its RSV program. Immunology Today, Enanta announced the expansion of its immunology portfolio which is focused on designing and developing highly potent and selective, oral small molecule inhibitors for the treatment of inflammatory diseases, by targeting key drivers of the type 2 immune response. KIT Inhibitor EPS-1421: Enanta nominated EPS-1421 as its lead development candidate. EPS-1421 is a novel, potent and selective oral inhibitor of KIT, designed to treat chronic spontaneous urticaria and potentially other indications by depleting mast cells, thereby addressing a primary driver of these diseases. EPS-1421 inhibits KIT with nanomolar potency in both binding and cellular assays and is highly selective for KIT versus other kinases. Further, EPS-1421 has demonstrated good in vitro and in vivo ADME properties preclinically. The company expects to conduct scale-up activities and IND enabling studies in 2025. STAT6 Inhibitors: The company’s second discovery program is aimed at developing oral STAT6 inhibitors for the treatment of type 2 immune driven diseases and will initially focus on atopic dermatitis and potentially other indications by blocking the IL-4/IL-13 signaling pathway, thereby addressing a primary driver of these diseases. Currently, Enanta is advancing novel, potent and selective oral inhibitors of STAT6. The company’s prototype inhibitors demonstrate potent activity and high selectivity for STAT6 over other STATs in both biochemical and cellular assays. Enanta continues to evaluate multiple compounds in preclinical studies and expects to conduct lead optimization activities for this program in 2025. Corporate Enanta will not be holding a conference call with today’s fiscal fourth quarter and year-end update. The company will provide its next update with the release of the RSVPEDs study results, expected in December 2024. About Enanta Pharmaceuticals, Inc. Enanta is using its robust, chemistry-driven approach and drug discovery capabilities to become a leader in the discovery and development of small molecule drugs with an emphasis on indications in virology and immunology. Enanta’s clinical programs are currently focused on respiratory syncytial virus (RSV) and its earlier-stage immunology pipeline aims to develop treatments for inflammatory diseases by targeting key drivers of the type 2 immune response, including KIT and STAT6 inhibition. Glecaprevir, a protease inhibitor discovered by Enanta, is part of one of the leading treatment regimens for curing chronic hepatitis c virus (HCV) infection and is sold by AbbVie in numerous countries under the tradenames MAVYRET ® (U.S.) and MAVIRET ® (ex-U.S.) (glecaprevir/pibrentasvir). A portion of Enanta’s royalties from HCV products developed under its collaboration with AbbVie contribute ongoing funding to Enanta’s operations. Please visit www.enanta.com for more information. Forward Looking Statements This press release contains forward-looking statements, including statements with respect to the prospects for advancement of Enanta’s clinical programs in RSV and its preclinical programs targeting KIT and STAT6 inhibition. Statements that are not historical facts are based on management’s current expectations, estimates, forecasts and projections about Enanta’s business and the industry in which it operates and management’s beliefs and assumptions. The statements contained in this release are not guarantees of future performance and involve certain risks, uncertainties and assumptions, which are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. Important factors and risks that may affect actual results include: the impact of development, regulatory and marketing efforts of others with respect to vaccines and competitive treatments for RSV; the discovery and development risks of Enanta’s programs in virology and immunology; Enanta’s lack of clinical development experience; Enanta’s need to attract and retain senior management and key research and development personnel; Enanta’s need to obtain and maintain patent protection for its product candidates and avoid potential infringement of the intellectual property rights of others; and other risk factors described or referred to in “Risk Factors” in Enanta’s Form 10-K for the fiscal year-ended September 30, 2023, and any other periodic reports filed more recently with the Securities and Exchange Commission. Enanta cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this release, and Enanta undertakes no obligation to update or revise these statements, except as may be required by law. Tables to Follow ENANTA PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS UNAUDITED (in thousands, except per share amounts) Three Months Ended Twelve Months Ended September 30, September 30, 2024 2023 2024 2023 Revenue $ 14,607 $ 18,932 $ 67,635 $ 79,204 Operating expenses Research and development 30,778 36,167 131,476 163,524 General and administrative 13,683 13,795 57,850 52,887 Total operating expenses 44,461 49,962 189,326 216,411 Loss from operations (29,854 ) (31,030 ) (121,691 ) (137,207 ) Interest expense (2,581 ) (3,151 ) (10,940 ) (5,148 ) Interest and investment income, net 3,249 4,664 14,843 11,360 Loss before income taxes (29,186 ) (29,517 ) (117,788 ) (130,995 ) Income tax benefit (expense) 363 1,410 1,743 (2,821 ) Net loss $ (28,823 ) $ (28,107 ) $ (116,045 ) $ (133,816 ) Net loss per share Basic $ (1.36 ) $ (1.33 ) $ (5.48 ) $ (6.38 ) Diluted $ (1.36 ) $ (1.33 ) $ (5.48 ) $ (6.38 ) Weighted average common shares outstanding Basic 21,190 21,057 21,157 20,969 Diluted 21,190 21,057 21,157 20,969 ENANTA PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS UNAUDITED (in thousands) September 30, September 30, 2024 2023 Assets Current assets Cash and cash equivalents $ 37,233 $ 85,388 Short-term marketable securities 210,953 284,522 Accounts receivable 6,646 8,614 Prepaid expenses and other current assets 12,413 13,263 Income tax receivable 31,999 31,004 Short-term restricted cash 608 — Total current assets 299,852 422,791 Property and equipment, net 32,688 11,919 Operating lease, right-of-use assets 40,658 22,794 Long-term restricted cash 3,360 3,968 Other long-term assets 94 803 Total assets $ 376,652 $ 462,275 Liabilities and Stockholders' Equity Current liabilities Accounts payable $ 8,002 $ 4,097 Accrued expenses and other current liabilities 13,547 18,339 Liability related to the sale of future royalties 34,462 35,076 Operating lease liabilities 1,524 5,275 Total current liabilities 57,535 62,787 Liability related to the sale of future royalties, net of current portion 134,779 159,429 Operating lease liabilities, net of current portion 53,943 21,238 Series 1 nonconvertible preferred stock 1,350 1,423 Other long-term liabilities 231 663 Total liabilities 247,838 245,540 Total stockholders' equity 128,814 216,735 Total liabilities and stockholders' equity $ 376,652 $ 462,275