Financial Data and Key Metrics Changes - The company reported robust treatment response and long-term durability beyond 15 months from a single injection of ADVM-022, with zero rescue injections in patients from Cohort 1 [11][45] - There was a substantial reduction in annualized anti-VEGF injection frequency, with a 100% reduction in the high dose cohort and an 87% reduction in the low dose cohorts [43][45] Business Line Data and Key Metrics Changes - ADVM-022 continues to show a favorable safety profile across all four cohorts, with low-grade inflammation that is manageable with topical steroid eye drops [32][45] - The OPTIC trial has completed enrollment, and the company plans to present additional data from Cohorts 1 to 4 by the end of the year [12][45] Market Data and Key Metrics Changes - The company is preparing to advance ADVM-022 for two significant ocular disease indications: wet AMD and DME, which represent a large global market opportunity [12][46] - The ongoing INFINITY trial for diabetic macular edema is designed to demonstrate superior disease control compared to a single aflibercept injection, addressing a major cause of blindness in working adults [46][47] Company Strategy and Development Direction - The company aims to revolutionize the treatment paradigm for ocular diseases with ADVM-022, focusing on a one-and-done approach to reduce treatment burden for patients [10][11] - Plans are in place to scale up manufacturing capabilities from 200 liters to 1,000 liters to support future commercial product launch [13][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of ADVM-022 to change the disease course for patients with neovascular AMD and highlighted the importance of continuous delivery in the context of the COVID-19 pandemic [11][16] - The leadership team is focused on expanding business operations and capabilities, including clinical, regulatory, and manufacturing functions, to prepare for a pivotal trial and potential commercial launch [14][15] Other Important Information - The company has experienced limited impact from COVID-19 on its operations and ongoing clinical trials, successfully completing enrollment in Cohort 4 of the OPTIC trial [16] - The management team emphasized the importance of patient-centric approaches and the emotional impact of treatment on patients and their families [49][50] Q&A Session Summary Question: Efficacy and Safety of ADVM-022 - The management noted that improvements in anatomy and central retinal thickness have been observed, indicating potential efficacy of continuous delivery [52][53] - Concerns regarding posterior inflammation were addressed, with management expressing confidence in the safety profile of ADVM-022 compared to other treatments [54][56] Question: Management of Topical Steroids - Management discussed the experiences of patients when tapering off topical steroids, noting that some patients may experience a rebound effect, but most have been able to discontinue with minimal issues [59][60][64] Question: Inflammation Concerns - The management acknowledged the importance of monitoring inflammation levels and emphasized that the majority of patients have experienced manageable inflammation that responds well to topical steroids [67][68]

Financial Performance - As of June 30, 2020, the company had an accumulated deficit of $437.1 million and expects to incur substantial expenses and increasing losses in the foreseeable future[42] - The net loss for the six months ended June 30, 2020, was $52.1 million, an increase of $22.7 million compared to a net loss of $29.4 million for the same period in 2019[49] - The accumulated deficit as of June 30, 2020, was $437.1 million, indicating ongoing financial challenges since inception[53] - Net cash used in operating activities for the six months ended June 30, 2020, was $30.5 million, compared to $24.8 million for the same period in 2019[57] - Net cash provided by financing activities for the six months ended June 30, 2020, was $151.4 million, primarily from the sale of common stock[59] Revenue Generation - The company has not generated any revenue from product sales to date, relying instead on research, collaboration, and license arrangements with strategic partners[44] Cash Position - The company had $280.1 million in cash, cash equivalents, and short-term investments as of June 30, 2020, which is expected to fund operations into 2022[43] - As of June 30, 2020, the company had $280.1 million in cash, cash equivalents, and short-term investments, up from $166.0 million as of December 31, 2019[53] Research and Development - Research and development expenses increased by $10.2 million to $19.2 million for Q2 2020 compared to Q2 2019, primarily due to increased production costs and personnel-related expenses[50] - The company expects research and development expenses to continue increasing as it invests in advancing its gene therapy product candidate ADVM-022 and other research programs[50] Administrative Expenses - General and administrative expenses rose by $3.5 million to $10.6 million for Q2 2020 compared to Q2 2019, driven by higher personnel costs and facility-related expenses[51] - The company anticipates that general and administrative expenses will rise in future periods to support its gene therapy programs and organizational growth[51] Clinical Trials and Product Development - The OPTIC trial for ADVM-022 has shown long-term durability beyond 15 months from a single IVT injection with zero anti-VEGF rescue injections in the high-dose cohort[35] - The INFINITY Phase 2 trial has been initiated to evaluate ADVM-022 in patients with diabetic macular edema, with plans to enroll approximately 33 patients[35] - The company is scaling up manufacturing processes from 200L to 1000L to support the future commercial launch of ADVM-022[35] - The FDA granted Fast Track designation for ADVM-022 for the treatment of wet age-related macular degeneration[35] Manufacturing and Operations - The company does not have operational clinical or commercial manufacturing facilities, relying on third-party contractors for clinical manufacturing[43] - The company has licensed the right to use AAV.7m8 to GenSight Biologics for their product candidate GS030, currently in clinical development[37] - The company has experienced limited impact from the COVID-19 pandemic on its operations and clinical trials to date[39] Internal Controls and Governance - As of June 30, 2020, the company's disclosure controls and procedures were deemed effective by the Chief Executive Officer and Chief Financial Officer[61] - There were no changes in internal control over financial reporting during the three months ended June 30, 2020 that materially affected internal controls[61] - The management acknowledges that disclosure controls and procedures cannot prevent all errors and fraud, providing only reasonable assurance[62] - The design of the control system must consider resource constraints and the cost-benefit ratio of controls[62] - The company recognizes the inherent limitations in all control systems, which may not detect all control issues or instances of fraud[62] Funding and Financial Strategy - The company plans to seek additional funding through equity or debt financings to support its development and commercialization efforts[54]

Financial Data and Key Metrics Changes - The company ended Q1 2020 with $297 million in cash, cash equivalents, and marketable securities, which is expected to finance operations until 2022 [11] - The financial results for the first quarter of 2020 were reported alongside the announcement of the new INFINITY trial for ADVM-022 [5] Business Line Data and Key Metrics Changes - The ongoing OPTIC Phase I trial for ADVM-022 has shown promising data, demonstrating long-term durability beyond one year with zero rescue injections in patients treated in Cohort 1 [9][14] - The INFINITY Phase 2 trial for diabetic macular edema (DME) is now open for patient enrollment, with the FDA having approved the IND for diabetic retinopathy [10][15] Market Data and Key Metrics Changes - Approximately 30 million people in the U.S. are impacted by diabetes, with about 1.5 to 2 million patients likely suffering from DME, representing a significant market opportunity [15][42] - The current standard-of-care therapy for DME requires frequent anti-VEGF intravitreal injections, which presents a challenge for patient compliance [16] Company Strategy and Development Direction - The company aims to position itself as a leader in gene therapy, focusing on developing a pipeline of novel gene therapies beyond ADVM-022 [10] - The INFINITY trial is designed to demonstrate superior control of disease activity following a single injection of ADVM-022 compared to aflibercept [17] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the impact of COVID-19 on operations but emphasized the commitment to advancing clinical trials and maintaining patient safety [7][12] - The company remains focused on developing transformative one-time treatments for patients with wet AMD and DME [20] Other Important Information - The collaboration with Regeneron has been terminated, allowing the company to explore new indications and targets [77] - The company has implemented remote study monitoring and precautionary measures at study sites due to COVID-19 [7][12] Q&A Session Summary Question: Can you clarify the DME trial design and the criteria for rescue injections? - Management confirmed that the criteria for rescue injections in the INFINITY trial are stringent, with expectations that more than half of aflibercept patients would require rescue by week 16 [24][34] Question: What are the implications of the trial design for future registrational studies? - Management indicated that the trial design is robust and aims to show superiority over current standard care, with potential for creative approaches in future studies [29][66] Question: How does the company plan to address the dosing for DME patients? - The same two doses used in wet AMD trials will be applied, with flexibility to adapt based on interim analysis results [66] Question: What is the expected timeline for data from the INFINITY trial? - Management stated that data will be shared once patients are enrolled and treated, with further guidance on timelines to follow [69] Question: Can you discuss the inflammation kinetics observed in the OPTIC trial? - Inflammation is expected to peak early post-injection, with steroid eye drops effectively managing it [72][74] Question: What are the next steps following the OPTIC trial? - The company plans to analyze data from Cohort 4 before determining the design of future studies [48][68]

Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME) Market Overview - Over 30 million people in the U S are living with diabetes, all at risk of DR[6] - DR affects approximately one in three (33%) adults with diabetes[6] - DME affects approximately 5% of people with diabetes[7] - The global sales for approved anti-VEGF therapies is $11 billion[6] - Standard-of-care therapy for DME is anti-VEGF intravitreal injections, which require frequent and long-term administration[6] ADVM-022 Gene Therapy - ADVM-022 is a one-time intravitreal injection for durable expression of therapeutic protein[7] - It produces codon-optimized aflibercept for efficient VEGF and PlGF inhibition[7,8] INFINITY Phase 2 Trial - The INFINITY trial is a Phase 2 clinical trial of ADVM-022 in Diabetic Macular Edema (DME)[2,9,10] - The trial will enroll approximately 33 patients and is designed to demonstrate superior disease control compared to a single aflibercept injection[10] - The primary objective is to assess the time to worsening of DME disease activity[9]

Financial Performance - As of March 31, 2020, the company had an accumulated deficit of $407.9 million and expects to incur substantial expenses and increasing losses from operations in the foreseeable future[42] - The company reported total operating expenses of $23.791 million for Q1 2020, an increase of $8.084 million compared to $15.707 million in Q1 2019[48] - The net loss for Q1 2020 was $22.906 million, compared to a net loss of $14.489 million in Q1 2019, representing an increase in loss of $8.417 million[48] - The company has not generated any revenue from product sales to date, relying instead on research, collaboration, and license arrangements[43] - Net cash used in operating activities was $13.6 million for the three months ended March 31, 2020, compared to $13.2 million for the same period in 2019[56] - The company has not generated positive cash flow or net income from operations since inception[52] Research and Development - Research and development expenses for Q1 2020 were $14.751 million, up from $10.131 million in Q1 2019, reflecting an increase of $4.620 million[48] - The company expects research and development expenses to increase in future periods as it continues to invest in advancing its gene therapy product candidate ADVM-022[49] - The OPTIC trial for ADVM-022 has shown long-term durability beyond 1 year with zero rescue injections in Cohort 1[34] - The INFINITY Phase 2 trial will enroll approximately 33 patients to evaluate ADVM-022 in patients with diabetic macular edema (DME)[36] Cash and Investments - The company had $297.1 million in cash, cash equivalents, and short-term investments as of March 31, 2020, sufficient to fund operations into 2022[42] - As of March 31, 2020, the company had an accumulated deficit of $407.9 million and $297.1 million in cash, cash equivalents, and short-term investments, up from $166.0 million as of December 31, 2019[52] - Net cash provided by financing activities for the three months ended March 31, 2020, was $148.6 million, primarily from the sale of common stock[58] - The company sold 10,925,000 shares of common stock for $140.9 million in net proceeds in February 2020[53] Administrative Expenses - General and administrative expenses rose to $9.040 million in Q1 2020 from $5.576 million in Q1 2019, an increase of $3.464 million[48] - General and administrative expense rose by $3.5 million to $9.0 million for the three months ended March 31, 2020, from $5.6 million in the same period of 2019[50] - General and administrative expenses are anticipated to rise in future periods to support the advancement of gene therapy programs[50] Future Funding - Future funding will be required for development, commercialization, and internal research programs, with potential reliance on equity or debt financings[53]

Financial Data and Key Metrics Changes - The company reported a strong financial position with over $300 million in cash, following a public follow-on offering that raised approximately $141 million, expected to fund operations into 2022 [10][11] - Specific financial metrics for Q4 2019 were not detailed in the provided content, but the overall financial health appears robust [10] Business Line Data and Key Metrics Changes - The lead gene therapy candidate, ADVM-022, is advancing with significant clinical progress, particularly in the OPTIC trial for wet AMD, with promising data from Cohorts 1 and 2 showing robust efficacy and a favorable safety profile [8][14][15] - The company plans to explore a second indication for diabetic retinopathy (DR) with an IND submission expected in the first half of 2020 and a Phase 1/2 trial beginning in the second half of the year [8][17] Market Data and Key Metrics Changes - The diabetic retinopathy market is highlighted as a large and underserved area, with an estimated 8 million people affected in the U.S., but only 2 million diagnosed and 1 million treated [17][18] - The prevalence of diabetic retinopathy is expected to increase alongside the growing prevalence of diabetes, indicating a significant unmet need for effective therapies [18] Company Strategy and Development Direction - The company is focused on delivering transformative therapies for patients with serious ocular and rare diseases, positioning itself as a leader in gene therapy [7][9] - The strategy includes advancing the clinical development of ADVM-022 and expanding the pipeline of novel gene therapies using their industry-leading AAV platform [9] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the fluid situation regarding the coronavirus and its potential impact on operations, but reported no specific impacts on the OPTIC trial to date [6][12] - The management expressed excitement about the potential of ADVM-022 as a one-time treatment for wet AMD and DR, emphasizing the commitment to advancing these programs [11][18] Other Important Information - The company has moved to a new headquarters in Redwood City to support growth plans and expand core capabilities [9] - Angela Thedinga was appointed as Chief Technology Officer to lead the manufacturing strategy as the company advances toward later-stage clinical trials [10] Q&A Session Summary Question: Enrollment status for Cohort 4 and potential read-throughs for diabetic retinopathy - Management confirmed active screening for Cohort 4 with strong interest from investigators, indicating rapid enrollment is expected [21][22] - Regarding diabetic retinopathy, management discussed potential read-throughs from AMD data, emphasizing safety and efficacy considerations [23][24] Question: Safety signals and implications for intravitreal therapy - Management highlighted the differences in inflammation types between ADVM-022 and other therapies, noting that ADVM-022 has shown manageable low-grade inflammation [30][33] Question: Status of steroid tapering in cohorts - Management provided insights into the tapering of steroids used in previous cohorts, indicating that inflammation has been low-grade and manageable [48][51]

[PART I](index=3&type=section&id=PART%20I) [Business](index=3&type=section&id=Item%201%20Business) Adverum Biotechnologies is a clinical-stage gene therapy company developing ADVM-022 for ocular diseases and leveraging a proprietary AAV vector platform - Adverum's lead product candidate, **ADVM-022**, is a gene therapy for wet AMD and diabetic retinopathy, administered as a single intravitreal injection using an AAV.7m8 vector[7](index=7&type=chunk) - The company is conducting the **OPTIC trial**, a Phase 1 study of ADVM-022 in wet AMD patients, which has received **FDA Fast Track designation**[7](index=7&type=chunk) - Adverum plans to file an **IND for ADVM-022 in diabetic retinopathy** in the first half of 2020 and initiate a Phase 1/2 trial in the second half of 2020[7](index=7&type=chunk) - The company's manufacturing process utilizes the **Baculovirus Expression Vector System (BEVS)** for high-yield, scalable AAV vector production up to **2000-liter scale**[38](index=38&type=chunk) - In August 2019, development of **ADVM-043 for Alpha-1 Antitrypsin deficiency was discontinued** due to a lack of clinically meaningful protein expression in a Phase 1/2 trial[33](index=33&type=chunk) [Our Product Candidates](index=6&type=section&id=Our%20Product%20Candidates) Adverum's product pipeline includes lead programs for wet AMD and diabetic retinopathy, a rare disease candidate, and a partnered program Product Pipeline Status | Gene Therapy Candidate | Indication | Preclinical | Phase 1 | Phase 2 | Phase 3 | | :--- | :--- | :--- | :--- | :--- | :--- | | **Lead Programs** | | | | | | | ADVM-022 | Wet AMD | | ■ | | | | ADVM-022 | Diabetic Retinopathy | ■ | | | | | **Rare Disease** | | | | | | | ADVM-053 (Early Dev) | Hereditary Angioedema | ■ | | | | | **Partnered Program** | | | | | | | GS030 (GenSight) | Retinitis Pigmentosa | | ■ | | | OPTIC Trial Cohort Details | Cohort | Patient Count (n) | Dose (vg/eye) | Prophylactic Steroid | | :--- | :--- | :--- | :--- | | Cohort 1 | 6 | 6 x 10^11 | 14-day oral taper | | Cohort 2 | 6 | 2 x 10^11 | 14-day oral taper | | Cohort 3 | 9 | 2 x 10^11 | 6-week topical taper | | Cohort 4 | 9 | 6 x 10^11 | 6-week topical taper | - As of February 2020, in the OPTIC trial, **6 of 6 patients in high-dose cohort 1 remained rescue injection-free** at a median follow-up of **50 weeks**, while **4 of 6 patients in lower-dose cohort 2 were rescue injection-free** at **24 weeks**[22](index=22&type=chunk) - **ADVM-022** has been **well-tolerated** with no drug-related serious adverse events or dose-limiting toxicities, and low-grade ocular inflammation was responsive to steroid eye drops[22](index=22&type=chunk) - In May 2019, the **FDA lifted a clinical hold on the ADVM-022 IND**, allowing dose escalation up to **2 x 10^12 vg/eye**, though a partial clinical hold remains for **6 x 10^12 vg/eye** doses pending CMC resolution[32](index=32&type=chunk) [Competition](index=13&type=section&id=Competition) The company faces significant competition from established anti-VEGF therapies and emerging next-generation treatments for ocular diseases - **ADVM-022** will compete with established anti-VEGF therapies from major pharmaceutical companies[40](index=40&type=chunk) - Key competitive categories for wet AMD and diabetic retinopathy include biosimilar anti-VEGFs, combination therapies, next-generation anti-VEGFs, and long-acting delivery systems or gene therapies like **RGX-314**[40](index=40&type=chunk) [License and Collaboration Agreements](index=14&type=section&id=License%20and%20Collaboration%20Agreements) The company manages various agreements, including a Regeneron collaboration expiring in 2020, an exclusive license for AAV.7m8, and an ongoing dispute with Virovek - The research collaboration with **Regeneron**, initiated in May 2014, is anticipated to expire on **May 1, 2020**, as Regeneron did not exercise its extension option[41](index=41&type=chunk) - The company holds an **exclusive worldwide license** from the University of California for intellectual property related to the **AAV.7m8 capsid** used in **ADVM-022**[42](index=42&type=chunk) - A dispute exists with **Virovek, Inc.** regarding their notice of intent to terminate a non-exclusive license for AAV manufacturing technology, which Adverum disputes[49](index=49&type=chunk) [Intellectual Property](index=16&type=section&id=Intellectual%20Property) The company protects its technology through over 40 issued patents and 150 pending applications, covering AAV-based delivery and the AAV.7m8 vector - The company owns or licenses over **40 issued patents** and **150 pending patent applications** in the U.S. and foreign jurisdictions[50](index=50&type=chunk) - Company-owned patent families cover AAV-based delivery of anti-VEGF proteins for eye diseases, with patents expected to expire between **2033 and 2039**[51](index=51&type=chunk) - A key exclusively licensed patent family covers the novel **AAV.7m8 vector**, with patents expected to expire in **2032**[54](index=54&type=chunk) [Risk Factors](index=24&type=section&id=Item%201A%20Risk%20Factors) The company faces substantial financial, development, clinical trial, third-party reliance, commercialization, and intellectual property risks, including a history of losses and dependence on ADVM-022 success - **Financial Risk:** The company has a history of significant operating losses and will require additional funding beyond existing cash, which is projected to fund operations into **2022**[79](index=79&type=chunk)[80](index=80&type=chunk)[81](index=81&type=chunk) - **Development Risk:** The business substantially depends on the success of **ADVM-022**, whose IND remains on partial clinical hold for **6 x 10^12 vg/eye** dosing pending resolution of FDA CMC requirements[83](index=83&type=chunk) - **Clinical Trial Risk:** Preliminary and interim data from trials like **OPTIC** may change, and ocular inflammation is a known side effect of **ADVM-022** requiring management[88](index=88&type=chunk)[92](index=92&type=chunk) - **Third-Party Reliance Risk:** Reliance on third parties for preclinical testing, clinical trials, and manufacturing introduces risks of delays, non-compliance, and supply disruptions[94](index=94&type=chunk)[95](index=95&type=chunk)[96](index=96&type=chunk) - **Commercialization Risk:** Significant uncertainties exist regarding market acceptance, pricing, and reimbursement for a single-administration gene therapy, alongside competition from established and developing therapies[107](index=107&type=chunk)[118](index=118&type=chunk)[112](index=112&type=chunk) - **Intellectual Property Risk:** Reliance on licensed patents and an ongoing dispute with **Virovek** over a manufacturing technology license pose risks to product development[141](index=141&type=chunk) [Unresolved Staff Comments](index=54&type=section&id=Item%201B%20Unresolved%20Staff%20Comments) The company reports no unresolved staff comments - Not applicable[176](index=176&type=chunk) [Properties](index=55&type=section&id=Item%202%20Properties) In January 2020, the company relocated its corporate headquarters to an 81,000 square foot facility in Redwood City, California, under a ten-year lease - The company's new corporate headquarters is an **81,000 square foot facility** in Redwood City, California, under a **ten-year lease** that commenced in January 2020[177](index=177&type=chunk) [Legal Proceedings](index=55&type=section&id=Item%203%20Legal%20Proceedings) The company reports no legal proceedings - Not applicable[178](index=178&type=chunk) [Mine Safety Disclosures](index=55&type=section&id=Item%204%20Mine%20Safety%20Disclosures) The company reports no mine safety disclosures - Not applicable[178](index=178&type=chunk) [PART II](index=56&type=section&id=PART%20II) [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=56&type=section&id=Item%205%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's common stock trades on Nasdaq under "ADVM", has approximately 15 record holders, and has never paid cash dividends, nor does it anticipate doing so - The company's common stock is listed on the **Nasdaq Global Market** under the ticker symbol **\"ADVM\"**[180](index=180&type=chunk) - The company has never declared or paid cash dividends and intends to retain all future earnings for business development[182](index=182&type=chunk) [Selected Financial Data](index=56&type=section&id=Item%206%20Selected%20Financial%20Data) The company is not required to provide this information as it qualifies as a smaller reporting company - The company is not required to provide this information as it qualifies as a smaller reporting company[184](index=184&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=57&type=section&id=Item%207%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) For 2019, the company reported a net loss of **$64.5 million**, an improvement from 2018, with **$166.0 million** in cash and investments, supplemented by a **$140.8 million** February 2020 offering, expected to fund operations into 2022 Results of Operations (2019 vs. 2018) (In thousands) | (In thousands) | 2019 | 2018 | Change | | :--- | :--- | :--- | :--- | | Collaboration and license revenue | $250 | $1,612 | $(1,362) | | Research and development | $40,419 | $50,133 | $(9,714) | | General and administrative | $28,376 | $24,560 | $3,816 | | Impairment of goodwill and intangible assets | $0 | $5,000 | $(5,000) | | **Operating loss** | **$(68,545)** | **$(78,081)** | **$9,536** | | **Net loss** | **$(64,486)** | **$(72,627)** | **$8,141** | - **R&D expenses decreased by $9.7 million** in 2019, primarily due to a **$7.9 million reduction** in manufacturing costs for **ADVM-022** and **ADVM-053**[209](index=209&type=chunk) - **G&A expenses increased by $3.8 million** in 2019, driven by higher consulting, professional services, and facilities costs[210](index=210&type=chunk) - As of December 31, 2019, the company had an accumulated deficit of **$385.0 million** and cash, cash equivalents, and short-term investments totaling **$166.0 million**[188](index=188&type=chunk)[215](index=215&type=chunk) - In February 2020, the company raised an additional **$140.8 million** in net proceeds from a public stock offering, with the total cash position expected to fund operations into **2022**[215](index=215&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=65&type=section&id=Item%207A%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is not required to provide this information as it qualifies as a smaller reporting company - The company is not required to provide this information as it qualifies as a smaller reporting company[221](index=221&type=chunk) [Financial Statements and Supplementary Data](index=65&type=section&id=Item%208%20Financial%20Statements%20and%20Supplementary%20Data) The consolidated financial statements for 2019 and 2018 are presented, showing 2019 total assets of **$222.7 million**, total liabilities of **$47.8 million**, and a net loss of **$64.5 million**, reflecting the adoption of Topic 842 Consolidated Balance Sheet Data (as of Dec 31) (In thousands) | (In thousands) | 2019 | 2018 | | :--- | :--- | :--- | | Cash and cash equivalents | $65,897 | $154,949 | | Short-term investments | $100,138 | $50,130 | | **Total assets** | **$222,727** | **$213,495** | | **Total liabilities** | **$47,770** | **$12,328** | | **Total stockholders' equity** | **$174,957** | **$201,167** | Consolidated Statement of Operations Data (Year ended Dec 31) (In thousands) | (In thousands) | 2019 | 2018 | | :--- | :--- | :--- | | Collaboration and license revenue | $250 | $1,612 | | Total operating expenses | $68,795 | $79,693 | | **Operating loss** | **$(68,545)** | **$(78,081)** | | **Net loss** | **$(64,486)** | **$(72,627)** | | Net loss per share | $(1.01) | $(1.18) | - The company adopted the new lease accounting standard (**Topic 842**) on January 1, 2019, recording a right-of-use asset of **$23.1 million** and a lease liability of **$24.7 million**[259](index=259&type=chunk) - As of December 31, 2019, the company had federal net operating loss carryforwards of approximately **$159.5 million** and federal R&D tax credit carryforwards of **$4.6 million**, subject to ownership change limitations[295](index=295&type=chunk) [Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=85&type=section&id=Item%209%20Changes%20in%20and%20Disagreements%20with%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None[299](index=299&type=chunk) [Controls and Procedures](index=85&type=section&id=Item%209A%20Controls%20and%20Procedures) Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2019, a conclusion affirmed by Ernst & Young LLP - Management concluded that as of December 31, 2019, the company's disclosure controls and procedures were effective[300](index=300&type=chunk) - Management concluded that the company's internal control over financial reporting was effective as of December 31, 2019, based on the **COSO 2013 framework**[301](index=301&type=chunk) - The independent registered public accounting firm, **Ernst & Young LLP**, issued an unqualified opinion on the effectiveness of the company's internal control over financial reporting as of December 31, 2019[304](index=304&type=chunk) [Other Information](index=88&type=section&id=Item%209B%20Other%20Information) This section details the company's 2020 Annual Meeting of Stockholders, scheduled for June 23, 2020, including proposal deadlines - The **2020 Annual Meeting of Stockholders** is scheduled for **June 23, 2020**[310](index=310&type=chunk) [PART III](index=89&type=section&id=PART%20III) [Directors, Executive Officers and Corporate Governance](index=89&type=section&id=Item%2010%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) The information required by this item is incorporated by reference from the company's definitive proxy statement for its 2020 Annual Meeting of Stockholders - Information is incorporated by reference from the definitive proxy statement[312](index=312&type=chunk) [Executive Compensation](index=89&type=section&id=Item%2011%20Executive%20Compensation) The information required by this item is incorporated by reference from the company's definitive proxy statement for its 2020 Annual Meeting of Stockholders - Information is incorporated by reference from the definitive proxy statement[313](index=313&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=89&type=section&id=Item%2012%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) The information required by this item is incorporated by reference from the company's definitive proxy statement for its 2020 Annual Meeting of Stockholders - Information is incorporated by reference from the definitive proxy statement[314](index=314&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=89&type=section&id=Item%2013%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) The information required by this item is incorporated by reference from the company's definitive proxy statement for its 2020 Annual Meeting of Stockholders - Information is incorporated by reference from the definitive proxy statement[315](index=315&type=chunk) [Principal Accountant Fees and Services](index=89&type=section&id=Item%2014%20Principal%20Accountant%20Fees%20and%20Services) The information required by this item is incorporated by reference from the company's definitive proxy statement for its 2020 Annual Meeting of Stockholders - Information is incorporated by reference from the definitive proxy statement[316](index=316&type=chunk) [PART IV](index=89&type=section&id=PART%20IV) [Exhibits, Financial Statement Schedules](index=89&type=section&id=Item%2015%20Exhibits%2C%20Financial%20Statement%20Schedules) This section provides an index of financial statements and exhibits filed with the Form 10-K, with consolidated financial statements located in Item 8 - This section contains the index of financial statements and exhibits filed with the Form 10-K[318](index=318&type=chunk)[319](index=319&type=chunk) [Form 10-K Summary](index=91&type=section&id=Item%2016%20Form%2010-K%20Summary) The company reports no Form 10-K summary - None[322](index=322&type=chunk)