E N A N T A Pharmaceuticals CREATING SMALL MOLECULE DRUGS FOR VIRAL INFECTIONS AND LIVER DISEASES Corporate Presentation June 22, 2021 Forward Looking Statements Disclaimer This presentation contains forward-looking statements concerning our business, operations and financial performance and condition, as well as our plans, objectives and expectations for our research and development programs, our business and the industry in which we operate. Any statements contained herein that are not statements of histo ...

Financial Data and Key Metrics Changes - For Q2 2021, total revenue was $20.1 million, a decrease from $27.6 million in the same period of 2020, primarily due to lower royalty revenue from AbbVie's MAVYRET product sales [31][32] - Royalty revenue was based on 50% of MAVYRET sales at a royalty rate of 10%, with AbbVie's global MAVYRET product sales reported at $415 million [31][32] - Net loss for the quarter was $22 million, or a loss of $1.09 per diluted common share, compared to a net loss of $6 million, or a loss of $0.30 per diluted common share, for the same period in 2020 [36] Business Line Data and Key Metrics Changes - The company has three active clinical programs in virology and liver diseases, conducting seven clinical trials, including studies for hepatitis B, respiratory syncytial virus (RSV), and non-alcoholic steatohepatitis (NASH) [6][7][27] - Preliminary data from the Phase 1b study of EDP-514 in chronic HBV patients showed it was safe and well-tolerated, with a mean reduction of 1 log in HBV RNA compared to 0.3 log reduction in placebo [10][13] Market Data and Key Metrics Changes - The ongoing studies for EDP-938 in RSV are crucial as RSV is expected to reemerge globally after COVID-19 mitigation measures subside, with trial sites established in multiple regions [22][23] - The company is preparing for the initiation of clinical trials for EDP-721, an oral HBV RNA destabilizer, expected to start in mid-2021 [19][29] Company Strategy and Development Direction - The company aims to develop an all-oral functional cure for chronic HBV infection by combining EDP-514 and EDP-721, targeting multiple mechanisms to enhance treatment efficacy [16][18][66] - The strategy includes exploring combination therapies with external agents, such as siRNA or immunotherapy, to enhance the chances of achieving a functional cure for HBV [62][66] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming catalysts across the pipeline, including preliminary results from the Phase 1b study of EDP-514 in viremic HBV patients and the initiation of EDP-721 trials [5][29] - The impact of COVID-19 on clinical trial recruitment and timelines was acknowledged, particularly for the NASH program, with expectations for interim analysis data to be reported in Q3 2021 [27][51] Other Important Information - The company ended the quarter with approximately $400 million in cash and marketable securities, providing a strong financial position to support ongoing and future clinical trials [36] - The company is also advancing its discovery initiatives for SARS-CoV-2, focusing on developing oral protease and polymerase inhibitors [26][71] Q&A Session Summary Question: Can you provide more granularity on RNA declines and the 800 mg dose optimization for EDP-514? - Management noted high trough levels at both 200 mg and 400 mg doses, with a focus on safety and tolerability rather than virology in the current study [38][40][41] Question: What are the latest views on the NASH program and potential paths forward? - Management highlighted the ongoing studies for EDP-297 and EDP-305, with expectations for data in mid-2021 and a focus on identifying effective doses for combination therapies [47][52] Question: Were any serological tests done at week 2 for EDP-514? - Management clarified that the study was not designed to assess viral loads at that time, but the data will be compiled for future analysis [54][57] Question: Can you discuss the safety profile of EDP-514 and the onset of adverse events? - Management indicated that adverse events were mild and scattered throughout the study, with no concerning patterns observed [63] Question: How does the company view the potential for protease inhibitors versus polymerase inhibitors for COVID-19? - Management expressed confidence in both targets, emphasizing the importance of targeting conserved regions to minimize resistance [78][79] Question: What are the regulatory considerations for the RSV program? - Management stated that discussions with the FDA regarding pivotal trial designs and endpoints will occur after finalizing the Phase 2b program [89]

Part I [Business Overview](index=6&type=section&id=Item%201.%20Business) Enanta Pharmaceuticals develops small molecule drugs for viral infections and liver diseases, funding its R&D pipeline primarily through royalties from AbbVie's HCV treatment - The company's primary focus is on discovering and developing **small molecule drugs** for **viral infections** (RSV, HBV, SARS-CoV-2, hMPV) and **liver diseases** (NASH)[20](index=20&type=chunk) - Funding for wholly-owned R&D programs is supported by **royalties** from the collaboration with AbbVie on the HCV treatment MAVYRET/MAVIRET[20](index=20&type=chunk) Financial Metrics (as of FY 2020) | Financial Metric | Value (as of FY 2020) | | :--- | :--- | | Cash, Cash Equivalents & Marketable Securities | $419 million | | Fiscal 2020 Royalty Revenue | $122.5 million | Enanta's Research and Development Pipeline (as of September 30, 2020) | Disease Area | Product Candidate / Class | Discovery/Preclinical | Phase 1 | Phase 2 | Phase 3 | Marketed | Status | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | **HCV** | glecaprevir (Protease Inhibitor) | | | | | ✓ | Marketed by AbbVie in MAVYRET/MAVIRET | | **RSV** | EDP-938 (N-Protein Inhibitor) | | | ✓ | | | Phase 2b RSVP study ongoing | | **NASH** | EDP-305 (FXR Agonist) | | | ✓ | | | Phase 2b ARGON-2 study ongoing | | **HBV** | EDP-514 (Core Inhibitor) | | ✓ | | | | Two Phase 1b studies ongoing | | **NASH** | EDP-297 (FXR Agonist Follow-on) | | ✓ | | | | Phase 1 study ongoing | | **hMPV** | Non-Fusion Inhibitor | ✓ | | | | | Discovery | | **COVID-19** | SARS-CoV-2 Inhibitor | ✓ | | | | | Discovery | [Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including heavy reliance on AbbVie's HCV drug sales, clinical development uncertainties, intense competition, and potential impacts from the COVID-19 pandemic - Financial prospects are **heavily dependent on AbbVie's success** in selling MAVYRET/MAVIRET for HCV, with sales potentially impacted by reduced diagnoses and treatment rates due to the COVID-19 pandemic[101](index=101&type=chunk) - The company faces **substantial competition** in all its target disease areas (HCV, RSV, HBV, NASH, hMPV, SARS-CoV-2), with many competitors having more advanced product candidates and greater resources[104](index=104&type=chunk)[107](index=107&type=chunk) - Clinical drug development is a **lengthy, expensive, and uncertain process**, and the company's pipeline candidates have not yet advanced beyond Phase 2 trials, with potential for delays or failures[121](index=121&type=chunk) - The ongoing COVID-19 pandemic has **impacted business operations and clinical trials**, causing delays in recruitment for studies like the ARGON-2 (NASH) and RSVP (RSV) trials[108](index=108&type=chunk)[110](index=110&type=chunk) - The company **relies on third-party manufacturers**, including some in China, for clinical drug supplies, with potential disruptions from trade wars, political unrest, or pandemics adversely affecting operations[145](index=145&type=chunk)[148](index=148&type=chunk) [Properties](index=54&type=section&id=Item%202.%20Properties) Enanta leases approximately 67,000 square feet of office and laboratory space for its corporate headquarters in Watertown, Massachusetts - The company leases a total of approximately **67,000 square feet** of office and laboratory space in Watertown, Massachusetts, across two separate lease agreements[191](index=191&type=chunk) [Legal Proceedings](index=54&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings as of the report date - The company is **not currently involved in any material legal proceedings**[192](index=192&type=chunk) Part II [Market for Common Equity and Related Matters](index=55&type=section&id=Item%205.%20Market%20for%20the%20Company%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Enanta's common stock trades on NASDAQ under "ENTA", and the company has never paid cash dividends nor anticipates doing so - Common stock is traded on **The NASDAQ Global Select Market** under the symbol "**ENTA**"[198](index=198&type=chunk) Quarterly Stock Price (Fiscal Years 2020 & 2019) | Fiscal Year | Quarter | High ($) | Low ($) | | :--- | :--- | :--- | :--- | | **2020** | First | 67.88 | 57.15 | | | Second | 62.12 | 38.40 | | | Third | 58.59 | 44.90 | | | Fourth | 54.57 | 42.07 | | **2019** | First | 86.42 | 64.09 | | | Second | 106.80 | 68.67 | | | Third | 101.27 | 80.52 | | | Fourth | 89.25 | 58.02 | - The company has **never declared or paid cash dividends** and does not expect to in the foreseeable future[199](index=199&type=chunk) [Selected Consolidated Financial Data](index=57&type=section&id=Item%206.%20Selected%20Consolidated%20Financial%20Data) This section provides selected financial data for the past five fiscal years, highlighting a shift to a net loss in FY2020 despite stable total assets Selected Historical Financial Data (in thousands, except per share data) | Metric | 2020 | 2019 | 2018 | | :--- | :--- | :--- | :--- | | **Revenue** | $122,473 | $205,197 | $206,625 | | **Research and development** | $136,756 | $142,213 | $94,856 | | **Total operating expenses** | $164,112 | $168,459 | $118,297 | | **Income (loss) from operations** | $(41,639) | $36,738 | $88,328 | | **Net income (loss)** | $(36,168) | $46,383 | $71,956 | | **Diluted net income (loss) per share** | $(1.81) | $2.21 | $3.48 | | **Total assets (at year end)** | $486,132 | $489,829 | $414,227 | | **Cash, cash equivalents and marketable securities** | $419,283 | $400,249 | $325,119 | [Management's Discussion and Analysis (MD&A)](index=58&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial performance, noting a significant revenue decrease and net loss in FY2020, while maintaining strong liquidity [Results of Operations](index=63&type=section&id=Results%20of%20Operations) FY2020 revenue decreased significantly to $122.5 million due to lower AbbVie royalties, resulting in a net loss of $36.2 million despite slightly reduced R&D expenses Revenue Comparison (in thousands) | Year Ended September 30, | 2020 | 2019 | 2018 | | :--- | :--- | :--- | :--- | | Royalties | $122,473 | $205,197 | $191,625 | | Milestones | — | — | $15,000 | | **Total revenue** | **$122,473** | **$205,197** | **$206,625** | - The **$82.7 million decrease** in revenue in FY2020 was due to lower numbers of treated HCV patients as a result of the worldwide COVID-19 pandemic and competitive pricing pressures[233](index=233&type=chunk) R&D Expense by Program (in thousands) | R&D Program | 2020 | 2019 | 2018 | | :--- | :--- | :--- | :--- | | Virology | $85,856 | $75,087 | $40,047 | | Liver disease | $45,001 | $66,892 | $54,691 | | Other | $5,899 | $234 | $118 | | **Total R&D expenses** | **$136,756** | **$142,213** | **$94,856** | - R&D expense decreased by **$5.5 million** in FY2020 compared to FY2019, primarily due to the timing of clinical studies and the impact of the COVID-19 pandemic, which paused or delayed enrollment in several trials[237](index=237&type=chunk) [Liquidity and Capital Resources](index=65&type=section&id=Liquidity%20and%20Capital%20Resources) As of September 30, 2020, Enanta maintained strong liquidity with $419 million in cash and securities, despite a significant decrease in operating cash flow in FY2020 - The company's principal sources of liquidity are cash, cash equivalents, and marketable securities, totaling **$419 million** as of September 30, 2020[241](index=241&type=chunk) Summary of Cash Flows (in thousands) | Activity | 2020 | 2019 | 2018 | | :--- | :--- | :--- | :--- | | Net cash from Operating | $7,088 | $71,418 | $29,220 | | Net cash from Investing | $19,830 | $(86,664) | $(35,402) | | Net cash from Financing | $8,983 | $2,574 | $4,409 | - The **decrease in cash from operations** in FY2020 was primarily driven by a decrease in royalty payments received from AbbVie and an increase in R&D costs[241](index=241&type=chunk) [Consolidated Financial Statements and Supplementary Data](index=68&type=section&id=Item%208.%20Consolidated%20Financial%20Statements%20and%20Supplementary%20Data) This section presents the audited consolidated financial statements and the independent auditor's unqualified opinion, highlighting critical audit matters related to R&D accruals - The independent auditor, PricewaterhouseCoopers LLP, issued an **unqualified opinion** on the consolidated financial statements and the effectiveness of internal control over financial reporting as of September 30, 2020[293](index=293&type=chunk) - A **critical audit matter** identified was the significant management judgment required in **estimating research and development and pharmaceutical drug manufacturing accruals**, which are based on contract terms, timelines, and progress of activities[303](index=303&type=chunk)[304](index=304&type=chunk) - The company **adopted the new lease accounting standard (Topic 842)** on October 1, 2019, resulting in the recognition of right-of-use assets of $7.2 million and lease liabilities of $8.6 million[350](index=350&type=chunk) [Controls and Procedures](index=68&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that both disclosure controls and internal control over financial reporting were effective as of September 30, 2020, with no material changes - Management concluded that the company's disclosure controls and procedures were **effective** at a reasonable assurance level as of September 30, 2020[258](index=258&type=chunk) - Based on the COSO 2013 framework, management concluded that the company's internal control over financial reporting was **effective** as of September 30, 2020[260](index=260&type=chunk) Part III [Directors, Executive Officers and Corporate Governance](index=70&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance is incorporated by reference from the company's 2021 Proxy Statement - Detailed information on directors, executive officers, and corporate governance is **incorporated by reference** from the 2021 Proxy Statement[265](index=265&type=chunk) [Executive Compensation](index=70&type=section&id=Item%2011.%20Executive%20Compensation) Details regarding executive compensation are incorporated by reference from the company's 2021 Proxy Statement - Information on executive compensation is **incorporated by reference** from the 2021 Proxy Statement[266](index=266&type=chunk) [Security Ownership and Equity Compensation Plans](index=70&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) This section details securities authorized under equity compensation plans as of September 30, 2020, with further ownership details incorporated by reference Equity Compensation Plan Information (as of September 30, 2020) | Plan Category | Securities to be issued upon exercise (a) | Weighted-average exercise price ($) (b) | Securities remaining for future issuance (c) | | :--- | :--- | :--- | :--- | | **Equity plans approved by security holders** | 3,399,000 | $47.81 | 1,407,000 | | **Equity plans not approved by security holders** | — | — | — | | **Total** | **3,399,000** | **$47.81** | **1,407,000** | Part IV [Exhibits and Financial Statement Schedules](index=72&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statement%20Schedules) This section lists all financial statements, schedules, and exhibits filed as part of the Form 10-K, including governance documents and material contracts - The financial statements are included under **Part II, Item 8** of the report[274](index=274&type=chunk) - A **list of all exhibits** filed with the report is provided, including governance documents, material contracts like the AbbVie agreement, and compensatory plans[277](index=277&type=chunk)

From Chemistry to Cures ENANTA Pharmaceuticals 1 A Phase 2 dose ranging, randomized, double-blind and placebo-controlled study of EDP-305 in subjects with primary biliary cholangitis (PBC) with or without an inadequate response to ursodeoxycholic acid (UDCA) Topline Results 06MAY2020 Forward Looking Statements Disclaimer This presentation contains forward-looking statements concerning our plans, objectives and expectations for EDP-305 and its development for NASH. Any statements contained herein that are no ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended September 30, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number 001-35839 | --- | --- | --- | |-----------------------------------------|-----------------------------------------------------------------|------------------------------------------ ...