PROTARA THERAPEUTICS CORPORATE PRESENTATION March 2022 FORWARD LOOKING STATEMENTS Statements contained in this presentation regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "designed," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other wo ...

Clinical Development - TARA-002 is currently in a Phase 1 clinical trial for non-muscle invasive bladder cancer (NMIBC), with approximately 30,000 high-grade NMIBC cases in the U.S. annually considered suitable for treatment [29][39]. - IV Choline Chloride has received Orphan Drug Designation and Fast Track Designation from the FDA for intestinal failure associated liver disease (IFALD), with a Phase 1 pharmacokinetic trial and a Phase 3 clinical trial planned [24][31]. - The company plans to align with the FDA on a development plan for TARA-002 and initiate a clinical trial in LM patients [60]. - The company has initiated a clinical development plan that includes a Phase 1 pharmacokinetic study and a Phase 3 clinical trial as part of its overall clinical program necessary for registration [82]. - The company is currently focused on the ongoing Phase 1 clinical trial of TARA-002 in NMIBC, which may be slower or costlier than anticipated [204]. - The regulatory approval process for TARA-002 may be more expensive and time-consuming than anticipated, potentially impacting financial performance [208]. - The FDA has granted fast track designation to IV Choline Chloride for the treatment of IFALD, but this may not guarantee a faster development or approval process [214]. Product Development and Pipeline - TARA-002 is developed from the same master cell bank as OK-432, utilizing a proprietary manufacturing process compliant with current Good Manufacturing Practices (cGMP) [35][42]. - The company aims to explore the use of TARA-002 in combination with other therapies and expand its pipeline while maintaining a disciplined investment approach [32]. - TARA-002 is being developed for the treatment of lymphatic malformations (LMs), which occur in approximately 1 in every 4,000 live births in the U.S., translating to about 1,400-1,800 cases annually [44]. - The company plans to seek approval for TARA-002 in Europe and other regions, in addition to the U.S. [37]. - The company has manufactured sufficient amounts of cGMP drug substance and drug product to initiate planned clinical trials, with scale-up for commercial demand ready to commence [83]. - The company has decided to cease further development of the product candidate Vonapanitase at this time [84]. Financial Performance and Projections - The company has not generated any revenue from product sales and does not expect to do so in the near term, necessitating additional capital to finance ongoing and future clinical trials [26]. - Protara Therapeutics has never generated any revenues and has a limited operating history, making it difficult to evaluate its business success and future viability [193]. - The company expects to incur significant operating losses for the foreseeable future as it continues research and development activities, including ongoing clinical trials for TARA-002 and IV Choline Chloride [194]. - Protara may need to raise additional financing in the future, which could be difficult to obtain on favorable terms, potentially diluting stockholder ownership [201]. - The company anticipates that continued pressure from the Affordable Care Act will lead to reduced reimbursement rates, potentially harming revenue and profitability [176]. Regulatory Environment - The FDA requires an IND submission before initiating clinical trials, which becomes effective 30 days after receipt unless safety concerns arise [128]. - The FDA mandates ongoing compliance with quality control and assurance regulations post-approval, including periodic inspections and reporting of adverse events [143]. - The FDA may withdraw approval if compliance with regulatory standards is not maintained or if new safety issues arise post-marketing [145]. - Orphan Drug Designation can be granted for drugs treating rare diseases affecting fewer than 200,000 individuals in the U.S., providing benefits like tax credits and a waiver of application fees [139]. - The complexities of biological product structures and manufacturing processes present significant hurdles for the abbreviated approval pathway, which the FDA is still working to clarify [148]. Market and Competition - The company faces competition from various pharmaceutical and biotechnology companies in the commercialization of its products [122][125]. - The ability to establish and enforce intellectual property rights for TARA-002 and IV Choline Chloride is crucial for commercial success [1]. - The company may face challenges in obtaining favorable labeling for TARA-002 and IV Choline Chloride, impacting commercialization efforts [1]. Impact of COVID-19 - The COVID-19 pandemic has caused delays in clinical trials and non-clinical studies, negatively impacting the company's operations and financial condition [185]. - The company has implemented work-from-home policies and modified operations in response to COVID-19, affecting productivity and timelines [184]. - The COVID-19 pandemic has negatively impacted Protara's clinical development plans, potentially causing delays in patient enrollment and resource availability at clinical trial sites [206]. Compliance and Legal Considerations - The company is subject to numerous federal and state regulations, which may incur significant compliance costs [181]. - The federal Anti-Kickback Statute prohibits remuneration to induce purchases of items reimbursable under federal healthcare programs, with broad interpretations of "remuneration" that could impact compliance [152]. - Compliance with various federal and state healthcare laws is costly, and violations could lead to significant penalties, including civil and criminal penalties, and exclusion from government programs [164].

PART I – FINANCIAL INFORMATION [Item 1. Condensed Consolidated Financial Statements](index=7&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements) The company reported a net loss of $37.0 million for the nine months ended Sep 30, 2021, with total assets decreasing to $180.7 million [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to $180.7 million as of September 30, 2021, driven by a reduction in cash and cash equivalents Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2021 (unaudited) | Dec 31, 2020 | | :--- | :--- | :--- | | Cash and cash equivalents | $40,742 | $168,598 | | Marketable debt securities | $97,668 | $0 | | **Total Assets** | **$180,697** | **$203,157** | | Total Liabilities | $10,291 | $3,914 | | **Total Stockholders' Equity** | **$170,406** | **$199,243** | | **Total Liabilities and Stockholders' Equity** | **$180,697** | **$203,157** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Net loss for the nine months ended September 30, 2021, increased to $37.0 million due to higher operating expenses Statement of Operations Summary (in thousands) | Metric | Q3 2021 | Q3 2020 | Nine Months 2021 | Nine Months 2020 | | :--- | :--- | :--- | :--- | :--- | | Research & development | $4,093 | $2,796 | $17,020 | $8,330 | | General & administrative | $6,737 | $5,266 | $20,182 | $17,157 | | **Total operating expenses** | **$10,830** | **$8,062** | **$37,202** | **$25,487** | | **Net loss** | **($10,777)** | **($7,970)** | **($37,024)** | **($25,170)** | | Net loss per share | ($0.96) | ($1.26) | ($3.30) | ($4.26) | [Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operations was $27.6 million for the first nine months of 2021, contributing to a $127.9 million decrease in cash Cash Flow Summary for the Nine Months Ended September 30 (in thousands) | Cash Flow Activity | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | ($27,595) | ($16,641) | | Net cash (used in)/provided by investing activities | ($100,083) | $3,346 | | Net cash (used in)/provided by financing activities | ($228) | $178,686 | | **Net (decrease)/increase in cash** | **($127,906)** | **$165,391** | | **Cash, cash equivalents, and restricted cash - end of period** | **$41,487** | **$165,955** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=14&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail the company's clinical-stage status, accounting policies, and confirmation of sufficient liquidity for the next 12 months - The company is a clinical-stage biopharmaceutical company focused on advancing therapies for cancer and rare diseases, with key programs including TARA-002 (for LMs and NMIBC) and IV Choline Chloride (for IFALD)[28](index=28&type=chunk) - Management believes that current financial resources are **sufficient to satisfy liquidity needs for at least twelve months** from the report's issuance date[31](index=31&type=chunk) Stock-Based Compensation Expense (in thousands) | Expense Category | Q3 2021 | Q3 2020 | Nine Months 2021 | Nine Months 2020 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $392 | $158 | $1,114 | $568 | | General and administrative | $2,324 | $2,623 | $7,340 | $6,839 | | **Total** | **$2,716** | **$2,781** | **$8,454** | **$7,407** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=28&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses its clinical portfolio strategy, rising operating losses, and sufficient liquidity for the next 12 months [Overview and Pipeline](index=28&type=section&id=Overview%20and%20Pipeline) Protara is a clinical-stage biopharmaceutical company focused on cancer and rare diseases with key programs TARA-002 and IV Choline Chloride - The company's portfolio includes two main development programs: **TARA-002** for non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs), and **IV Choline Chloride** for intestinal failure associated liver disease (IFALD)[108](index=108&type=chunk)[113](index=113&type=chunk) - The FDA cleared the Investigational New Drug (IND) application for TARA-002 in NMIBC in October 2021, and the company plans to start a **Phase 1 trial by year-end**[110](index=110&type=chunk) - TARA-002 has received **Rare Pediatric Disease designation** from the FDA for the treatment of LMs[111](index=111&type=chunk) [Results of Operations](index=31&type=section&id=Results%20of%20Operations) Operating loss for the first nine months of 2021 grew to $37.2 million, driven by increased R&D and G&A expenses Comparison of Results of Operations (in thousands) | Metric | Nine Months 2021 | Nine Months 2020 | Change | | :--- | :--- | :--- | :--- | | Research and development | $17,020 | $8,330 | $8,690 | | General and administrative | $20,182 | $17,157 | $3,025 | | **Operating loss** | **($37,202)** | **($25,487)** | **($11,715)** | | **Net Loss** | **($37,024)** | **($25,170)** | **($11,854)** | [Liquidity and Capital Resources](index=33&type=section&id=Liquidity%20and%20Capital%20Resources) The company holds $138.4 million in cash and securities, deemed sufficient to fund operations for at least the next twelve months - As of September 30, 2021, the company had **$138.4 million in cash, cash equivalents, and marketable debt securities**[135](index=135&type=chunk) - The company believes its current financial resources are **sufficient to satisfy its estimated liquidity needs for at least twelve months** from the issuance of the financial statements[137](index=137&type=chunk) - Net cash used in operating activities increased to **$27.6 million** for the nine months ended Sep 30, 2021, from $16.6 million in the same period of 2020, driven by a higher net loss[140](index=140&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company indicates it has no material exposure to market risk requiring disclosure - The company states this item is **"Not applicable"**[145](index=145&type=chunk) [Item 4. Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2021 - Management concluded that as of September 30, 2021, the company's disclosure controls and procedures were **effective at the reasonable assurance level**[147](index=147&type=chunk) - There were **no changes in internal control over financial reporting** during the third quarter of 2021 that have materially affected, or are reasonably likely to materially affect, internal controls[149](index=149&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=36&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently party to any material legal proceedings - The company is **not currently a party to any legal proceedings** that are likely to have a material adverse effect on its business[152](index=152&type=chunk) [Item 1A. Risk Factors](index=36&type=section&id=Item%201A.%20Risk%20Factors) Key risks include limited operating history, dependence on key products, future financing needs, and reliance on third parties [Risks Related to Financial Condition](index=36&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition) The company faces significant financial risks from its limited operating history, lack of revenue, and need for additional capital - The company has a **very limited operating history**, has never generated revenue, and expects to incur significant future losses[154](index=154&type=chunk)[155](index=155&type=chunk) - Future operations depend on **raising additional financing**, which may not be available on favorable terms or at all, with the COVID-19 pandemic potentially limiting access to capital[160](index=160&type=chunk) [Risks Related to Drug/Biologics Development](index=39&type=section&id=Risks%20Related%20to%20Drug/Biologics%20Development) Success is highly dependent on the uncertain clinical development and regulatory approval of its main product candidates - The company's business is primarily dependent on the successful clinical development, regulatory approval, and commercialization of **TARA-002 and IV Choline Chloride**[165](index=165&type=chunk) - As a company, Protara has **never conducted a clinical trial or submitted a BLA/NDA**, which could lead to unforeseen delays and costs[169](index=169&type=chunk) - The development of TARA-002 for lymphatic malformations (LMs) is difficult to predict in terms of timing and cost because there are **no existing FDA-approved therapies for LMs**[170](index=170&type=chunk) [Other Risks Related to Our Business](index=45&type=section&id=Other%20Risks%20Related%20to%20Our%20Business) Business risks include intense competition, reliance on third-party manufacturing, and evolving healthcare regulations - The company faces **significant competition** from established pharmaceutical companies with greater financial and operational resources[186](index=186&type=chunk) - The company currently has **limited marketing capabilities and no sales organization**, and must build these functions or rely on third parties to commercialize any approved products[196](index=196&type=chunk) - Protara **relies completely on third-party contractors** for the supply, manufacturing, and distribution of its clinical drug supplies, which includes sole-source suppliers[229](index=229&type=chunk) - The business is subject to stringent and changing data privacy laws like **GDPR and CCPA**, and a security breach could lead to significant penalties and operational disruption[255](index=255&type=chunk)[256](index=256&type=chunk)[259](index=259&type=chunk) [Risks Related to Intellectual Property Rights](index=68&type=section&id=Risks%20Related%20to%20Intellectual%20Property%20Rights) The company's competitive position relies on its ability to obtain, maintain, and enforce its intellectual property rights - The company's success depends on its ability to **obtain and maintain patent protection**, which is an expensive and uncertain process[266](index=266&type=chunk)[267](index=267&type=chunk) - Protecting intellectual property rights globally is difficult, as laws and enforcement mechanisms in foreign countries, particularly developing ones, **may not be as robust as in the U.S.**[272](index=272&type=chunk) - The company may be **sued for infringing third-party intellectual property rights**, which could result in costly litigation and prevent or delay the commercialization of its products[277](index=277&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=74&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports no unregistered sales of equity securities during the period - **None reported**[293](index=293&type=chunk) [Item 3. Defaults Upon Senior Securities](index=74&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reports no defaults upon senior securities - **None reported**[294](index=294&type=chunk) [Item 4. Mine Safety Disclosures](index=74&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company's business - **None reported**[295](index=295&type=chunk) [Item 5. Other Information](index=74&type=section&id=Item%205.%20Other%20Information) The company reports no other information for this item - **None reported**[296](index=296&type=chunk) [Item 6. Exhibits](index=74&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the report, including corporate governance documents and officer certifications - The Exhibit Index lists all documents filed as part of the Form 10-Q, including corporate governance documents, officer certifications, and XBRL data files[297](index=297&type=chunk)[299](index=299&type=chunk)

Financial Performance - The total operating expenses for Q2 2021 were $12.8 million, up from $7.3 million in Q2 2020, resulting in an operating loss of $12.8 million compared to a loss of $7.3 million in the prior year [126]. - The net loss for Q2 2021 was approximately $12.8 million, an increase of $5.6 million compared to a net loss of $7.1 million in Q2 2020 [126]. - Net loss for the six months ended June 30, 2021, was approximately $26.2 million, compared to a net loss of $17.2 million for the same period in 2020, representing an increase of $9.0 million [134]. - The company incurred net losses of approximately $12.8 million and $7.1 million for the three months ended June 30, 2021, and 2020, respectively [134]. Expenses - Research and development expenses for Q2 2021 were approximately $5.9 million, an increase of $3.4 million compared to Q2 2020, primarily due to manufacturing activities for TARA-002 [126]. - General and administrative expenses for Q2 2021 were approximately $6.9 million, reflecting an increase of $2.1 million compared to Q2 2020, driven by higher stock-based compensation and new corporate headquarters expenses [127]. - Research and development expenses for the six months ended June 30, 2021, were approximately $12.9 million, an increase of $7.4 million compared to the same period in 2020 [130]. - General and administrative expenses for the six months ended June 30, 2021, were approximately $13.4 million, reflecting an increase of $1.6 million from the prior year [131]. Cash Position - The company had approximately $145.0 million in cash, cash equivalents, and marketable debt securities as of June 30, 2021 [116]. - The company's cash, cash equivalents, and marketable debt securities as of June 30, 2021, were $145.0 million, down from $168.6 million as of December 31, 2020 [134]. - Cash flows used in operating activities for the six months ended June 30, 2021, were approximately $21.4 million, an increase of $8.5 million compared to $12.9 million in 2020 [139]. - Net cash used in investing activities was $100.2 million for the six months ended June 30, 2021, compared to net cash provided of $3.5 million in 2020, a change of $103.7 million [140]. Future Outlook - The company does not expect to generate revenues prior to 2022, if ever, and will need to raise additional capital to finance ongoing and future clinical trials [113]. - As of June 30, 2021, the company had an accumulated deficit of approximately $73.0 million and expected to continue incurring significant operating losses for the next few years [114]. - The company believes its current financial resources are sufficient to meet liquidity needs for at least twelve months from the issuance of the financial statements [136]. - The company faces uncertainty in raising additional capital due to global economic conditions and the impact of the COVID-19 pandemic [137]. Clinical Development - TARA-002 is in later stage development for lymphatic malformations (LMs) and non-muscle invasive bladder cancer (NMIBC), with approximately 65,000 NMIBC diagnoses annually in the U.S. [107][113]. - The FDA granted Rare Pediatric Disease designation for TARA-002 for the treatment of LMs, with a significant dataset from a Phase 2 study involving over 500 patients secured [109]. - The company anticipates that the COVID-19 pandemic may negatively impact clinical trials and overall business operations due to resource limitations at trial sites [119].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36694 | Protara Therapeutics, Inc. | | --- | | (Exact name of registrant as specified in its charter) | (State or other jurisdict ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 Form 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 Or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36694 PROTARA THERAPEUTICS, INC. (Exact Name of Registrant as Specified in Its Charter) (State or Other Jurisdiction of Incorporatio ...

ARA PRO THERA S Corporate Presentation March 2021 . Chloride for IFALO replacement therefy for int Forward Looking Statements Statements contained in this presentation regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "designed," "estimates," "anticipates," "expects," "plans," "intends," "may," "c ...