Cara Therapeutics(US:CARA)2021-05-09 16:00PART I – FINANCIAL INFORMATION Item 1. Financial Statements (Unaudited) Presents unaudited condensed financial statements for Q1 2021, showing a net loss of $23.3 million and total assets decreasing to $250.2 million Condensed Balance Sheet Highlights (Amounts in thousands) | Metric | March 31, 2021 | December 31, 2020 | | :----------------------------------- | :------------- | :---------------- | | Total Assets | $250,228 | $271,157 | | Total Current Assets | $188,351 | $195,065 | | Cash and Cash Equivalents | $22,519 | $31,683 | | Marketable Securities (Current) | $149,063 | $149,242 | | Total Liabilities | $19,769 | $22,156 | | Total Stockholders' Equity | $230,459 | $249,001 | Condensed Statements of Comprehensive Loss Highlights (Amounts in thousands) | Metric | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | | :----------------------------------- | :-------------------------------- | :-------------------------------- | | Total Revenue | $1,935 | $8,093 | | Research and Development Expenses | $19,131 | $33,536 | | General and Administrative Expenses | $6,365 | $4,558 | | Operating Loss | $(23,561) | $(30,001) | | Net Loss | $(23,301) | $(28,922) | | Net Loss per Share (Basic and Diluted) | $(0.47) | $(0.62) | Condensed Statements of Cash Flows Highlights (Amounts in thousands) | Metric | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | | :----------------------------------- | :-------------------------------- | :-------------------------------- | | Net Cash Used in Operating Activities | $(23,721) | $(38,303) | | Net Cash Provided by Investing Activities | $13,869 | $36,959 | | Net Cash Provided by Financing Activities | $688 | $75 | | Net Decrease in Cash, Cash Equivalents and Restricted Cash | $(9,164) | $(1,269) | | Cash, Cash Equivalents and Restricted Cash at End of Period | $22,927 | $17,444 | Notes to Condensed Financial Statements Provides detailed notes on accounting policies, marketable securities, collaboration agreements, and revenue recognition for the unaudited financial statements - Cara Therapeutics is a clinical-stage biopharmaceutical corporation focused on developing and commercializing new chemical entities designed to alleviate pruritus by selectively targeting peripheral kappa opioid receptors20 - As of March 31, 2021, the Company had raised approximately $519.6 million from equity financing and earned $203.8 million under license agreements21 - The Company adopted ASU 2019-12 on January 1, 2021, which did not have a material effect on its financial statements due to a full valuation allowance33 1. Business Cara Therapeutics is a clinical-stage biopharmaceutical company focused on developing new chemical entities for pruritus - Cara Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing new chemical entities targeting peripheral kappa opioid receptors to alleviate pruritus20 - The company has incurred substantial net losses and negative cash flows from operating activities since inception, with net losses of $23.3 million and $28.9 million for the three months ended March 31, 2021 and 2020, respectively22 2. Basis of Presentation Details the preparation of unaudited interim financial statements in accordance with SEC rules and GAAP, including ASU 2019-12 adoption - The unaudited interim condensed financial statements are prepared in accordance with SEC rules and GAAP, reflecting all necessary adjustments for fair presentation24 - The Company adopted ASU 2019-12 on January 1, 2021, which simplifies income tax accounting, but it did not materially affect financial results due to a full valuation allowance3133 3. Available-for-Sale Marketable Securities Describes the composition and fair value of available-for-sale marketable securities, including realized gains from sales - Available-for-sale marketable securities consist of debt securities from U.S. Treasury, government-sponsored entities, investment grade institutions, and municipal bonds34 Available-for-Sale Marketable Securities (Amounts in thousands) | Type of Security | Amortized Cost (Mar 31, 2021) | Fair Value (Mar 31, 2021) | Amortized Cost (Dec 31, 2020) | Fair Value (Dec 31, 2020) | | :----------------------------------- | :---------------------------- | :------------------------ | :---------------------------- | :------------------------ | | U.S. Treasury securities | $15,164 | $15,167 | $20,710 | $20,750 | | U.S. government agency obligations | $22,099 | $22,097 | $22,125 | $22,128 | | Corporate bonds | $41,674 | $41,652 | $49,080 | $49,118 | | Commercial paper | $115,179 | $115,187 | $116,139 | $116,127 | | Municipal bonds | $11,663 | $11,688 | $11,680 | $11,684 | | Total | $205,779 | $205,791 | $219,734 | $219,807 | - During the three months ended March 31, 2021, the Company sold available-for-sale debt securities with a fair value of $8,029 thousand, resulting in realized gains of $39 thousand47 4. Accumulated Other Comprehensive Income (Loss) Presents changes in accumulated other comprehensive income (loss), including reclassified realized gains on security sales Changes in Accumulated Other Comprehensive Income (Loss) (Amounts in thousands) | Metric | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | | :----------------------------------- | :-------------------------------- | :-------------------------------- | | Balance, December 31 / 2019 | $73 | $170 | | Other comprehensive loss before reclassifications | $(22) | $(238) | | Amount reclassified from accumulated other comprehensive income | $(39) | — | | Net current period other comprehensive loss | $(61) | $(238) | | Balance, March 31 | $12 | $(68) | - Realized gains on sales of securities of $39 thousand were reclassified from accumulated other comprehensive income (loss) into net loss for the three months ended March 31, 202150 5. Fair Value Measurements Outlines the fair value hierarchy for financial instruments, with most assets categorized as Level 2 - The Company classifies financial instruments into a fair value hierarchy (Level 1, 2, 3) based on the observability of inputs5455 - Most financial assets, including U.S. Treasury securities, government agency obligations, corporate bonds, commercial paper, and municipal bonds, are categorized as Level 2, with fair values estimated using third-party pricing services566061 6. Restricted Cash Details the $408 thousand in restricted cash held as collateral for the Stamford office lease - The Company maintains $408 thousand in restricted cash as collateral for a standby letter of credit related to its Stamford office lease, consistent across March 31, 2021, and December 31, 2020626365 7. Prepaid expenses Summarizes the composition of prepaid expenses, including R&D clinical costs and insurance Prepaid Expenses (Amounts in thousands) | Category | March 31, 2021 | December 31, 2020 | | :----------------------------------- | :------------- | :---------------- | | Prepaid R&D clinical costs | $10,557 | $11,286 | | Prepaid insurance | $2,007 | $223 | | Other prepaid costs | $454 | $567 | | Total Prepaid Expenses | $13,018 | $12,076 | 8. Accounts Payable and Accrued Expenses Provides a breakdown of accounts payable and accrued expenses, including research projects and compensation Accounts Payable and Accrued Expenses (Amounts in thousands) | Category | March 31, 2021 | December 31, 2020 | | :----------------------------------- | :------------- | :---------------- | | Accounts payable | $4,765 | $4,893 | | Accrued research projects | $7,190 | $6,194 | | Accrued compensation and benefits | $2,336 | $4,955 | | Accrued professional fees and other | $589 | $839 | | Total | $14,880 | $16,881 | 9. Stockholders' Equity Reports on the vesting and settlement of performance-based and time-based restricted stock units in Q1 2021 - In February and March 2021, 76,750 performance-based restricted stock units and 32,669 time-based restricted stock units vested and were settled in common stock6869 10. Collaboration and Licensing Agreements Details key collaboration and licensing agreements with Vifor, VFMCRP, Maruishi, and CKDP for CR845/difelikefalin - Vifor Agreement (October 2020) granted Vifor exclusive U.S. license for CR845/difelikefalin injection for pruritus in hemodialysis and peritoneal dialysis patients, with $100 million upfront, $50 million from common stock purchase, eligibility for $50 million additional common stock investment upon U.S. regulatory approval, up to $240 million in sales-based milestones, and a 60% net profit-sharing arrangement (excluding FMCNA sales)72737475 - VFMCRP Agreement (May 2018) granted VFMCRP exclusive license for CR845/difelikefalin injection worldwide (excluding U.S., Japan, South Korea), receiving $50 million upfront and $20 million from common stock purchase, with eligibility for up to $470 million in regulatory and commercial milestones and tiered double-digit royalties on net sales, and a profit-sharing arrangement with VFMCRP for FMCNA dialysis clinics in the U.S.818283 - Maruishi Agreement (April 2013) granted Maruishi exclusive license for CR845/difelikefalin in Japan for acute pain and uremic pruritus, with eligibility for clinical/regulatory milestones (up to $10.5 million), a one-time sales milestone, sub-license fees, and tiered low double-digit to low twenties royalties on net sales, and earned a $2.0 million milestone in January 2021 for Phase 3 trial initiation8889253 - CKDP Agreement (April 2012) granted CKDP exclusive license for CR845/difelikefalin in South Korea, with eligibility for development/regulatory milestones (up to $3.8 million), sub-license fees, and tiered high single-digit to high teens royalties on net sales9192255 11. Revenue Recognition Explains revenue recognition policies for upfront and milestone payments from collaboration agreements under ASC Topic 606 - Revenue is recognized in accordance with ASC Topic 606. Upfront payments and milestone payments are recognized, with future milestone and royalty payments considered variable consideration and constrained at inception due to uncertainty93111113 - For the Vifor Agreement, the entire transaction price of $111.6 million was recognized as license and milestone fees revenue in 2020 upon license grant103114 - For the VFMCRP Agreement, the $55.4 million transaction price was recognized as revenue as R&D services were performed, completed by December 31, 2020104115127 - For the Maruishi Agreement, $9.6 million was allocated to the license (recognized immediately) and $5.7 million to R&D services (recognized over time until July 2015). In January 2021, a milestone event resulted in $1.2 million license and milestone fees revenue and $0.7 million collaborative revenue120132 12. Net Loss Per Share Presents net loss per share calculations, excluding potentially dilutive shares due to net losses Net Loss Per Share (Basic and Diluted) | Metric | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | | :----------------------------------- | :-------------------------------- | :-------------------------------- | | Net loss | $(23,301) | $(28,922) | | Weighted-average common shares outstanding | 49,917,990 | 46,724,951 | | Net loss per share | $(0.47) | $(0.62) | - Potentially dilutive shares (stock options and restricted stock units) were excluded from diluted net loss per share calculations for both periods due to the Company's net losses, making their inclusion anti-dilutive138141143 13. Stock-Based Compensation Outlines stock plans, RSU grants to executives, and a breakdown of stock-based compensation expenses - The Company has two stock plans: the 2019 Inducement Plan for new employees and the 2014 Equity Incentive Plan for various equity compensation awards144145 - In March 2021, 176,000 performance-based and 100,000 time-based restricted stock units were granted to executive officers. Compensation expense for performance-based RSUs is recognized upon achievement of performance targets149150 Stock-Based Compensation Expense (Amounts in thousands) | Category | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | | :----------------------------------- | :-------------------------------- | :-------------------------------- | | Research and development (Stock Options) | $1,590 | $1,609 | | General and administrative (Stock Options) | $1,014 | $1,070 | | Total Stock Option Expense | $2,604 | $2,679 | | R&D (RSUs) | $568 | $14 | | G&A (RSUs) | $822 | $153 | | Total RSU Expense | $1,390 | $167 | 14. Income Taxes Discusses pre-tax losses and the full tax valuation allowance, with no income tax benefit recognized for Q1 2021 - The Company recognized pre-tax losses of $23.3 million and $29.0 million for the three months ended March 31, 2021 and 2020, respectively164 - A full tax valuation allowance was recognized against deferred tax assets. No benefit from income taxes was recognized for Q1 2021 because the Company's 2020 revenue exceeded $70 million, making it ineligible for the Connecticut R&D Tax Credit Exchange Program164165 15. Commitments and Contingencies Details contractual obligations including the Enteris License Agreement, manufacturing agreements, and operating leases - Enteris License Agreement (August 2019) granted non-exclusive license for Peptelligence® technology for oral delivery of peptide APIs, with $8.0 million upfront paid ($4.0 million cash, $4.0 million common stock), obligations for milestone payments and low-single digit royalties, and an expected $10.0 million milestone payment in Q2 2021171172173260 - Manufacturing Agreement (July 2019): Master Manufacturing Services Agreement (MSA) with Patheon UK Limited and Patheon Manufacturing Services LLC for commercial supplies of CR845/difelikefalin injection174175 - Leases: Operating lease for Stamford office space (Stamford Lease, December 2015, amended June 2020). Lease expense recognized on a straight-line basis. Operating lease cost was $406 thousand for Q1 2021, up from $234 thousand in Q1 2020177181183 Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations. Management discusses financial condition, operations, product pipeline, and liquidity, highlighting KORSUVA's development and regulatory milestones - Cara Therapeutics is a clinical-stage biopharmaceutical company developing KORSUVA (CR845/difelikefalin), a first-in-class kappa opioid receptor agonist, for pruritus and pain195 - The FDA accepted the NDA for KORSUVA injection for CKD-aP in February 2021 and granted Priority Review in March 2021, with a PDUFA target action date of August 23, 2021198203 - The COVID-19 pandemic has impacted clinical trial initiation and patient enrollment for some trials, but no significant delays in clinical development or commercial timelines are currently expected200202 Overview Introduces Cara Therapeutics as a clinical-stage biopharmaceutical company developing KORSUVA for pruritus - The company is a clinical-stage biopharmaceutical company developing KORSUVA (CR845/difelikefalin), a first-in-class KOR agonist, for pruritus by selectively targeting peripheral kappa opioid receptors195 - KORSUVA injection has shown significant reductions in itch intensity and improved quality of life in hemodialysis patients with CKD-aP in Phase 3 trials196 - The FDA accepted the NDA for KORSUVA injection for CKD-aP in February 2021 and granted Priority Review in March 2021, with a PDUFA target action date of August 23, 2021198 Recent Developments Highlights the FDA's Priority Review for KORSUVA injection and the EMA MAA submission, noting COVID-19 impacts - The COVID-19 pandemic has affected clinical trial initiation and patient enrollment for some trials, including Oral KORSUVA for NP and CLD-aP, but no significant delays in clinical development or commercial timelines are currently expected200202 - The FDA accepted the NDA for KORSUVA (CR845/difelikefalin) injection for moderate-to-severe pruritus in hemodialysis patients in February 2021 and granted Priority Review in March 2021, with a PDUFA target action date of August 23, 2021203 - VFMCRP submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in March 2021, with a decision expected in Q2 2022204 Overview of Our Product Candidates Presents CR845/difelikefalin as a selective kappa opioid receptor agonist with injectable and oral forms for various pruritus indications - CR845/difelikefalin is a new chemical entity designed to selectively stimulate peripheral kappa opioid receptors, avoiding CNS-related side effects of traditional opioids205 - The product candidate pipeline includes KORSUVA injection for CKD-aP (NDA accepted, Priority Review granted) and Oral KORSUVA for NDD-CKD pruritus, Atopic Dermatitis, CLD-aP (ongoing Phase 2), and Notalgia Paresthetica (ongoing Phase 2)209 - CR845/difelikefalin is available in injectable form for hospital/dialysis settings and oral form for outpatient treatment of pruritus or chronic pain208 KORSUVA (CR845/Difelikefalin) Injection for Treatment of Chronic Kidney Disease-Associated Pruritus (CKD-aP) Details the FDA's Priority Review for KORSUVA injection for CKD-aP and positive Phase 3 trial results - NDA for KORSUVA injection for moderate-to-severe pruritus in hemodialysis patients was accepted by the FDA in February 2021 and granted Priority Review in March 2021, with a PDUFA target action date of August 23, 2021210 - KALM-2 Phase 3 trial met primary and key secondary endpoints, showing 54% of KORSUVA patients achieved a ≥3-point improvement in worst itching NRS score at week 12 (vs. 42% placebo, p=0.02)215 - KALM-1 Phase 3 trial also met primary and all secondary endpoints, with 51% of KORSUVA patients achieving a ≥3-point improvement in worst itching NRS score at week 12 (vs. 28% placebo, p=0.000019)217 Oral KORSUVA (CR845/Difelikefalin) for Treatment of Non-Dialysis Dependent (NDD) Chronic Kidney Disease-Associated Pruritus (CKD-aP) Reports positive Phase 2 results for Oral KORSUVA in NDD-CKD pruritus and plans for a Phase 3 program - Phase 2 trial of Oral KORSUVA (1.0 mg) for NDD-CKD patients achieved the primary endpoint of statistically significant reduction in weekly mean of daily worst itching NRS scores vs. placebo after 12 weeks (-4.4 KORSUVA vs. -3.3 placebo, p=0.018)219 - The FDA indicated acceptability of Stage 5 pre-dialysis CKD patients for a Phase 3 trial and potential to use existing KORSUVA injection data to support approval based on a single Phase 3 trial for Oral KORSUVA in this population222 - The company plans to initiate its Phase 3 program for Oral KORSUVA in NDD-CKD by year-end 2021222 Oral KORSUVA (CR845/difelikefalin) for Treatment of Moderate-to-Severe Pruritus Associated with Atopic Dermatitis (AD) Discusses Phase 2 trial results for Oral KORSUVA in AD pruritus, noting primary endpoint not met but positive subgroup analysis - KARE Phase 2 trial for AD pruritus did not meet the primary efficacy endpoint (change from baseline in weekly mean of daily 24-hour Itch NRS score at week 12) for any dose group in the ITT population224 - A prespecified analysis showed a statistically significant change in the primary endpoint for the mild-to-moderate (BSA<10%) patient population (p=0.036, All doses vs placebo)225 - The company intends to request an End of Phase 2 Meeting with the FDA in H2 2021 and aims to initiate a Phase 3 program by year-end228 Oral KORSUVA (CR845/Difelikefalin) for Treatment of Chronic Liver Disease-Associated Pruritus Reports on the ongoing Phase 2 trial for Oral KORSUVA in CLD-associated pruritus, with top-line data expected in H2 2021 - A Phase 2 trial of Oral KORSUVA for pruritus in patients with hepatic impairment due to PBC is ongoing, with top-line data expected in the second half of 2021231 - Previous Phase 1 safety and PK trial in CLD patients showed Oral KORSUVA was generally well-tolerated with dose-proportional PK parameters in mild-to-moderate CLD232 Oral KORSUVA (CR845/difelikefalin) for Treatment of Moderate-to-Severe Pruritus Associated with Notalgia Paresthetica (NP) Announces the initiation of a Phase 2 trial for Oral KORSUVA in Notalgia Paresthetica in January 2021 - A Phase 2 randomized, double-blind, placebo-controlled trial for Oral KORSUVA in adult subjects with NP was initiated in January 2021, evaluating efficacy and safety over an 8-week treatment period233 Intravenous CR845/Difelikefalin for Treatment of Acute Postoperative Pain Reports positive Phase 2/3 study results for IV CR845 in postoperative pain and PONV, with next steps under evaluation - Adaptive Phase 2/3 study of CR845/difelikefalin in abdominal surgery patients showed statistical significance for pain relief (AUC 0-24 hours) with the 1.0 mcg/kg dose (p=0.032)235 - Both 0.5 and 1.0 mcg/kg doses of CR845 injection showed statistically significant improvements in PONV impact scores and reduced anti-emetic medication use237 - The company has completed an advisory meeting with the FDA regarding the regulatory path for PONV and is evaluating next steps238 Human Abuse Liability Trial of CR845/Difelikefalin Injection Highlights the successful completion of a Human Abuse Liability trial, showing low 'drug liking' for CR845 injection - A Human Abuse Liability (HAL) trial of CR845/difelikefalin injection successfully completed in Q4 2014, demonstrating statistically significant lower 'drug liking' scores compared to pentazocine (p <0.0001)239 - CR845/difelikefalin injection showed no 'drug liking' dose response and was not different from placebo, supporting its potential as a non-scheduled or low (Schedule V) scheduled peripheral kappa opioid239 Respiratory Safety Phase 1 Trial of CR845/Difelikefalin Injection Confirms a favorable respiratory safety profile for CR845 injection based on Phase 1 trial results - A Phase 1 trial evaluating respiratory safety of CR845/difelikefalin injection in April 2017 showed no statistically significant differences in respiratory measures between CR845 doses and placebo240241 - All treatment-emergent adverse events were mild and resolved without intervention, indicating a favorable respiratory safety profile241 Collaboration and License Agreements Summarizes key collaboration and license agreements for KORSUVA with Vifor, VFMCRP, Maruishi, and CKDP - Vifor Agreement (October 2020) granted Vifor exclusive U.S. license for KORSUVA injection in hemodialysis and peritoneal dialysis patients, receiving $100 million upfront and $50 million from common stock purchase, with eligibility for $50 million additional common stock investment upon U.S. regulatory approval, up to $240 million in sales-based milestones, and a 60% net profit-sharing arrangement (excluding FMCNA sales)242243244245246 - VFMCRP Agreement (May 2018) granted VFMCRP exclusive license for KORSUVA injection worldwide (excluding U.S., Japan, South Korea), receiving $50 million upfront and $20 million from common stock purchase, with eligibility for up to $470 million in regulatory and commercial milestones and tiered double-digit royalties on net sales, and a profit-sharing arrangement with VFMCRP for FMCNA dialysis clinics in the U.S.248249 - Maruishi Agreement (April 2013) granted Maruishi exclusive license for CR845/difelikefalin in Japan for acute pain and uremic pruritus, receiving $15.0 million upfront, with eligibility for up to $10.5 million in clinical/regulatory milestones, a one-time sales milestone, sub-license fees, and tiered low double-digit to low twenties royalties, and earned a $2.0 million milestone in January 2021250251252253 - CKDP Agreement (April 2012) granted CKDP exclusive license for CR845/difelikefalin in South Korea, receiving $0.6 million upfront, with eligibility for up to $3.8 million in development/regulatory milestones, sub-license fees, and tiered high single-digit to high teens royalties254255 Manufacturing and License Agreements Details the Enteris License Agreement for Peptelligence® technology and the MSA with Patheon for CR845/difelikefalin manufacturing - Enteris License Agreement (August 2019) granted a non-exclusive license for Peptelligence® technology for oral delivery of peptide APIs, with $8.0 million upfront paid ($4.0 million cash, $4.0 million common stock), obligations for milestone payments and low-single digit royalties, and an expected $10.0 million milestone payment in Q2 2021257258259260 - Patheon UK Limited MSA (July 2019): Master Manufacturing Services Agreement with Patheon and Patheon Greenville for commercial supplies of CR845/difelikefalin injection at Monza, Italy and Greenville, North Carolina sites262264 Components of Operating Results Explains the components of revenue, R&D expenses, G&A expenses, and other income, including tax implications - Revenue primarily consists of upfront payments and milestone/sub-license payments from license agreements. No product sales revenue to date265 - R&D expenses are primarily for CR845/difelikefalin development, including compensation, clinical trials, CRO fees, and stock-based compensation. R&D expenses for 2021 are expected to be consistent with 2020266267270 - General and administrative expenses include salaries, stock-based compensation, facility costs, legal fees, and consulting services. Expected to be consistent with 2020, with potential increases for commercialization efforts273274276 - Other income, net, includes interest/dividend income, realized gains/losses on securities, and accretion/amortization. Benefit from income taxes relates to state R&D tax credits, but no benefit for Q1 2021 due to revenue exceeding $70 million277278 Results of Operations Compares revenue, R&D, and G&A expenses for Q1 2021 and Q1 2020, highlighting significant changes Revenue Comparison (Amounts in thousands) | Revenue Type | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | % Change | | :----------------------------------- | :-------------------------------- | :-------------------------------- | :------- | | License and milestone fees | $1,192 | $8,021 | -85% | | Collaborative revenue | $706 | — | N/A | | Clinical compound revenue | $37 | $72 | -49% | | Total Revenue | $1,935 | $8,093 | -76% | R&D Expense Comparison (Amounts in thousands) | R&D Category | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | % Change | | :----------------------------------- | :-------------------------------- | :-------------------------------- | :------- | | Direct clinical trial costs | $10,000 | $25,738 | -61% | | Consultant services | $1,170 | $1,275 | -8% | | Stock-based compensation | $2,158 | $1,623 | 33% | | Other R&D operating expenses | $5,772 | $4,873 | 18% | | Total R&D Expense | $19,131 | $33,536 | -43% | G&A Expense Comparison (Amounts in thousands) | G&A Category | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | % Change | | :----------------------------------- | :-------------------------------- | :-------------------------------- | :------- | | Professional fees and public/investor relations | $877 | $1,131 | -22% | | Stock-based compensation | $1,974 | $1,223 | 61% | | Other G&A operating expenses | $3,483 | $2,183 | 60% | | Total G&A Expense | $6,365 | $4,558 | 40% | - Other income, net, decreased by 73% to $260 thousand in Q1 2021 from $957 thousand in Q1 2020, primarily due to lower interest income and net accretion income from a lower yield on investments289 - No benefit from income taxes was recognized in Q1 2021 (vs. $122 thousand in Q1 2020) due to ineligibility for the Connecticut R&D Tax Credit Exchange Program290 Liquidity and Capital Resources Discusses the company's funding history, current cash position, and future capital needs for KORSUVA development - Since inception, the Company has raised approximately $776.4 million, primarily from equity sales ($446.3 million), convertible preferred stock/debt ($73.3 million), and license agreements ($203.8 million)292 - As of March 31, 2021, unrestricted cash, cash equivalents, and marketable securities totaled $228.3 million, expected to fund operations into 2023296312 - The Company expects to continue incurring significant expenses and operating losses as it develops KORSUVA and seeks marketing approval, with future funding needs to be met through equity/debt financings and collaboration arrangements304310311 Cash Flows Analyzes changes in net cash used in operating, investing, and financing activities for Q1 2021 versus Q1 2020 Summary of Cash Flows (Amounts in thousands) | Activity | Three Months Ended March 31, 2021 | Three Months Ended March 31, 2020 | | :----------------------------------- | :-------------------------------- | :-------------------------------- | | Net cash used in operating activities | $(23,721) | $(38,303) | | Net cash provided by investing activities | $13,869 | $36,959 | | Net cash provided by financing activities | $688 | $75 | | Net decrease in cash, cash equivalents and restricted cash | $(9,164) | $(1,269) | - Net cash used in operating activities decreased from $38.3 million in Q1 2020 to $23.7 million in Q1 2021, primarily due to a lower net loss and changes in operating assets and liabilities315316 - Net cash provided by investing activities decreased from $37.0 million in Q1 2020 to $13.9 million in Q1 2021, mainly due to changes in maturities, redemptions, and purchases of marketable securities317318 Contractual Obligations and Commitments Outlines the company's contractual obligations, including operating leases and license agreements - Contractual obligations include operating lease obligations for the Stamford office, the Enteris License Agreement, and the MSA with Patheon321 - The Company has no material non-cancelable purchase commitments with contract manufacturers or service providers322 Discussion of Critical Accounting Policies Confirms no significant changes to critical accounting policies from the prior annual report - No significant changes to critical accounting policies were made during the three months ended March 31, 2021, from those described in the Annual Report on Form 10-K for 2020328 Item 3. Quantitative and Qualitative Disclosures About Market Risk. Discusses market risks, primarily interest rate and credit quality, related to the investment portfolio, noting immaterial exposure to interest rate changes - The Company invests a majority of its cash reserves in available-for-sale marketable securities, including investment-grade debt instruments, U.S. government obligations, corporate bonds, commercial paper, and municipal bonds329 Marketable Securities Investment (Amounts in millions) | Metric | March 31, 2021 | December 31, 2020 | | :----------------------------------- | :------------- | :---------------- | | Invested in marketable securities | $205.8 | $219.8 | | Yield | 0.26% | 0.32% | | Maturities | Through January 2024 | Through December 2023 | - A hypothetical 100 basis point increase in interest rates would result in immaterial decreases in the fair values of the marketable securities portfolio334 - No charges to credit loss expense were recorded for available-for-sale securities for the three months ended March 31, 2021 and 2020335 Item 4. Controls and Procedures. Management assessed disclosure controls and procedures as effective, with no material changes to internal control over financial reporting during Q1 2021 - Disclosure controls and procedures were evaluated as effective as of March 31, 2021, providing reasonable assurance that required information is recorded, processed, summarized, and reported timely337 - No material changes in internal control over financial reporting occurred during the quarter ended March 31, 2021338 PART II – OTHER INFORMATION Item 1. Legal Proceedings The company is not currently involved in any material legal proceedings that would adversely affect its business or financial condition - The Company is not currently involved in any material arbitration, litigation, or claims that would adversely affect its business, operating results, or financial condition344 Item 1A. Risk Factors. No material changes to risk factors were reported compared to the Annual Report on Form 10-K for 2020 - No material changes in risk factors were reported compared to the Annual Report on Form 10-K for the year ended December 31, 2020345 Item 6. Exhibits Lists exhibits filed with Form 10-Q, including certifications, XBRL taxonomy extensions, and corporate governance documents - The exhibits include certifications from the CEO and CFO, Inline XBRL Taxonomy Extension documents, and corporate organizational documents356357