0001558370-21-009963 | 10-Q | XBRL Viewer | Rhythm Pharmaceuticals INC

Document and Entity Information - shares	6 Months Ended
Document and Entity Information - shares	Jun. 30, 2021	Jul. 26, 2021
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Transition Report	false
Document Period End Date	Jun. 30, 2021
Entity File Number	001-38223
Entity Registrant Name	RHYTHM PHARMACEUTICALS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	46-2159271
Entity Address, Address Line One	222 Berkeley Street
Entity Address, Address Line Two	12th Floor
Entity Address, City or Town	Boston
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02116
City Area Code	857
Local Phone Number	264-4280
Title of 12(b) Security	Common Stock, $0.001 par value per share
Trading Symbol	RYTM
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		50,227,489
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2021
Document Fiscal Period Focus	Q2
Entity Central Index Key	0001649904
Amendment Flag	false

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Current assets:
Cash and cash equivalents	$ 69,339	$ 100,854
Short-term Investments	298,815	71,938
Prepaid expenses and other current assets	10,300	8,876
Total current assets	378,454	181,668
Property and equipment, net	3,051	3,195
Right-of-use asset	1,671	1,807
Intangible assets, net	4,886
Restricted cash	328	403
Total assets	388,390	187,073
Current liabilities:
Accounts payable	5,009	4,900
Accrued expenses and other current liabilities	11,880	12,559
Lease liability	570	535
Total current liabilities	17,459	17,994
Long-term liabilities:
Deferred tax liability	16,984
Lease liability	2,258	2,551
Total liabilities	36,701	20,545
Commitments and contingencies (Notes 5)
Stockholders' equity:
Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at June 30, 2021 and December 31, 2020
Common stock, $0.001 par value: 120,000,000 shares authorized; 50,226,739 and 44,235,903 shares issued and outstanding June 30, 2021 and December 31, 2020, respectively	50	44
Additional paid-in capital	802,584	625,762
Accumulated other comprehensive income	21	49
Accumulated deficit	(450,966)	(459,327)
Total stockholders' equity	351,689	166,528
Total liabilities and stockholders' equity	$ 388,390	$ 187,073

CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Jun. 30, 2021	Dec. 31, 2020
CONSOLIDATED BALANCE SHEETS
Preferred stock par value per share	$ 0.001	$ 0.001
Preferred stock, authorized	10,000,000	10,000,000
Preferred stock, issued	0	0
Preferred stock, outstanding	0	0
Common stock, par value per share	$ 0.001	$ 0.001
Common stock, authorized	120,000,000	120,000,000
Common stock, issued	50,226,739	44,235,903
Common stock, outstanding	50,226,739	44,235,903

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
Product revenue, net	$ 274		$ 309
Operating expenses:
Cost of sales	137		141
Research and development	25,104	$ 22,997	45,015	$ 45,501
Selling, general, and administrative	15,465	8,921	29,983	21,717
Total costs and expenses	40,706	31,918	75,139	67,218
Loss from operations	(40,432)	(31,918)	(74,830)	(67,218)
Other income (expense):
Other income			100,000
Interest income, net	21	801	175	1,937
Total other income, net	21	801	100,175	1,937
Income (loss) before taxes	(40,411)	(31,117)	25,345	(65,281)
Provision for (benefit from) income taxes	(5,022)		16,984
Net income (loss)	$ (35,389)	$ (31,117)	$ 8,361	$ (65,281)
Net income (loss) per share, Basic (in dollars per share)	$ (0.70)	$ (0.71)	$ 0.17	$ (1.48)
Net income (loss) per share, Diluted (in dollars per share)	$ (0.70)	$ (0.71)	$ 0.17	$ (1.48)
Weighted average shares, Basic (in shares)	50,209,484	44,098,860	48,931,127	44,074,352
Weighted average shares, Diluted (in shares)	50,209,484	44,098,860	49,644,704	44,074,352
Other comprehensive income (loss):
Net income (loss)	$ (35,389)	$ (31,117)	$ 8,361	$ (65,281)
Unrealized (loss) gain on marketable securities	79	567	(28)	630
Comprehensive income (loss)	$ (35,310)	$ (30,550)	$ 8,333	$ (64,651)

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Common stock	Additional Paid-in Capital	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit	Total
Beginning balance at Dec. 31, 2019	$ 44	$ 606,307		$ (325,331)	$ 281,020
Beginning balance (in shares) at Dec. 31, 2019	43,996,753
Increase (Decrease) in Stockholders' Equity
Stock compensation expense		5,475			5,475
Issuance of common stock in connection with ESPP		324			324
Issuance of common stock in connection with ESPP (in shares)	18,673
Issuance of common stock in connection with exercise of stock options		383			383
Issuance of common stock in connection with exercise of stock options (in shares)	72,964
Unrealized gain on marketable securities			$ 63		63
Net income (loss)				(34,164)	(34,164)
Ending balance at Mar. 31, 2020	$ 44	612,489	63	(359,495)	253,101
Ending balance (in shares) at Mar. 31, 2020	44,088,390
Beginning balance at Dec. 31, 2019	$ 44	606,307		(325,331)	281,020
Beginning balance (in shares) at Dec. 31, 2019	43,996,753
Increase (Decrease) in Stockholders' Equity
Net income (loss)					(65,281)
Ending balance at Jun. 30, 2020	$ 44	615,691	630	(390,612)	225,753
Ending balance (in shares) at Jun. 30, 2020	44,115,612
Beginning balance at Mar. 31, 2020	$ 44	612,489	63	(359,495)	253,101
Beginning balance (in shares) at Mar. 31, 2020	44,088,390
Increase (Decrease) in Stockholders' Equity
Stock compensation expense		3,028			3,028
Issuance of common stock in connection with exercise of stock options		174			174
Issuance of common stock in connection with exercise of stock options (in shares)	27,222
Unrealized gain on marketable securities			567		567
Net income (loss)				(31,117)	(31,117)
Ending balance at Jun. 30, 2020	$ 44	615,691	630	(390,612)	225,753
Ending balance (in shares) at Jun. 30, 2020	44,115,612
Beginning balance at Dec. 31, 2020	$ 44	625,762	49	(459,327)	166,528
Beginning balance (in shares) at Dec. 31, 2020	44,235,903
Increase (Decrease) in Stockholders' Equity
Stock compensation expense		5,191			5,191
Issuance of common stock in connection with ESPP		388			388
Issuance of common stock in connection with ESPP (in shares)	17,000
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units		3,466			3,466
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units (in shares)	198,855
Issuance of common stock upon completion of public offering, net of offering costs	$ 6	161,725			161,731
Issuance of common stock upon completion of public offering, net of offering costs (in shares)	5,750,000
Change in unrealized gain (loss) on marketable securities			(107)		(107)
Net income (loss)				43,750	43,750
Ending balance at Mar. 31, 2021	$ 50	796,532	(58)	(415,577)	380,947
Ending balance (in shares) at Mar. 31, 2021	50,201,758
Beginning balance at Dec. 31, 2020	$ 44	625,762	49	(459,327)	166,528
Beginning balance (in shares) at Dec. 31, 2020	44,235,903
Increase (Decrease) in Stockholders' Equity
Net income (loss)					8,361
Ending balance at Jun. 30, 2021	$ 50	802,584	21	(450,966)	351,689
Ending balance (in shares) at Jun. 30, 2021	50,226,739
Beginning balance at Mar. 31, 2021	$ 50	796,532	(58)	(415,577)	380,947
Beginning balance (in shares) at Mar. 31, 2021	50,201,758
Increase (Decrease) in Stockholders' Equity
Stock compensation expense		5,669			5,669
Issuance of common stock in connection with exercise of stock options		383			383
Issuance of common stock in connection with exercise of stock options (in shares)	24,981
Change in unrealized gain (loss) on marketable securities			79		79
Net income (loss)				(35,389)	(35,389)
Ending balance at Jun. 30, 2021	$ 50	$ 802,584	$ 21	$ (450,966)	$ 351,689
Ending balance (in shares) at Jun. 30, 2021	50,226,739

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020
Operating activities
Net income (loss)	$ 8,361	$ (65,281)
Adjustments to reconcile net loss to cash used in operating activities:
Stock-based compensation expense	10,860	8,503
Gain on sale of priority review voucher	(100,000)
Deferred tax provision	16,984
Depreciation and amortization	518	340
Non-cash rent expense	(122)	(114)
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	(1,374)	(352)
Accounts payable, accrued expenses and other current liabilities	(598)	(8,923)
Net cash used in operating activities	(65,371)	(65,827)
Investing activities
Purchases of short-term investments	(362,469)	(43,413)
Maturities of short-term investments	135,542	105,147
Proceeds from sale of priority review voucher	100,000
Milestone obligation under license agreement	(5,000)
Purchases of property and equipment	(260)
Net cash (used in) provided by investing activities	(132,187)	61,734
Financing activities
Net proceeds from issuance of common stock	161,731
Proceeds from the exercise of stock options	3,849	566
Proceeds from issuance of common stock from ESPP	388	324
Net cash provided by financing activities	165,968	890
Net decrease in cash, cash equivalents and restricted cash	(31,590)	(3,203)
Cash, cash equivalents and restricted cash at beginning of period	101,257	62,697
Cash, cash equivalents and restricted cash at end of period	$ 69,667	$ 59,494

Nature of Business

6 Months Ended

Jun. 30, 2021

Nature of Business

Nature of Business

Rhythm Pharmaceuticals, Inc.

Notes to Unaudited Condensed Consolidated Financial Statements

(In thousands, except share and per share information)

1. Nature of Business

Rhythm Pharmaceuticals, Inc. (the “Company” or “we”) is a commercial-stage biopharmaceutical company focused on changing the paradigm for the treatment of rare genetic diseases of obesity, which are characterized by early-onset, severe obesity and an insatiable hunger or hyperphagia. Our lead product candidate is IMCIVREE^® (setmelanotide), a potent melanocortin-4 receptor, or MC4R, agonist for the treatment of rare genetic diseases of obesity. We believe IMCIVREE, for which we have exclusive worldwide rights, has the potential to restore dysfunctional MC4R signaling due to impaired MC4R pathway function. MC4R pathway deficiencies result in the disruption of satiety signals and energy homeostasis in the body, which, in turn, leads to intense feelings of hunger and to obesity. IMCIVREE has been approved by the U.S. Food and Drug Administration, or FDA, for chronic weight management in adult and pediatric patients six years of age and older with obesity due to proopiomelanocortin, or POMC, proprotein convertase subtilisin/kexin type 1, or PCSK1, or leptin receptor, or LEPR, deficiency confirmed by genetic testing. IMCIVREE also has been approved by the European Commission for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE is now commercially available in the United States, and we are pursuing an international strategy to establish access and reimbursement for IMCIVREE in the European Union, or EU.

The Company is a Delaware corporation organized in February 2013 under the name Rhythm Metabolic, Inc., and as of October 2015, under the name Rhythm Pharmaceuticals, Inc.

The Company’s continued development efforts are focused on obesity related to several single gene-related, or monogenic, MC4R pathway deficiencies: Bardet-Biedl and Alstrom syndromes; obesity due to a genetic variant in one of the two alleles of the POMC, PCSK1 or LEPR gene, or heterozygous POMC, PCSK1 or LEPR obesity (collectively HETs); obesity due to steroid receptor coactivator 1, or SRC1, deficiency; obesity due to SH2B adapter protein 1, or SH2B1, deficiency; hypothalamic obesity; and MC4R deficiency obesity. In addition, we have expanded our development program to explore setmelotide’s potential efficacy in patients with severe obesity which may be due to variants in an additional 31 genes that are related to the MC4R pathway. There are additional diseases being studied as part of investigator-initiated protocols. Currently, there are no effective or approved treatments for these MC4R pathway-related diseases. The Company believes that the MC4R pathway is a compelling target for treating these genetic diseases because of its critical role in regulating appetite and weight by promoting satiety and weight control, and that peptide therapeutics are uniquely suited for activating this target.

The Company is subject to risks and uncertainties common to commercial-stage companies in the biotechnology industry, including but not limited to risks associated with the commercialization of approved products, completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Commercialization of approved products will require significant resources and in order to market IMCIVREE, the Company must continue to build its sales, marketing, managerial and other non-technical capabilities or make arrangements with third parties to perform these services. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities.

Liquidity

The Company has incurred operating losses and negative cash flows from operations since inception. As of June 30, 2021, the Company had an accumulated deficit of $450,966. The Company has primarily funded these losses

through the proceeds from the sales of common and preferred stock, asset sales as well as capital contributions received from the former parent company, Rhythm Holdings LLC. To date, the Company has minimal product revenue and management expects operating losses to continue for the foreseeable future. The Company has devoted substantially all of its resources to its drug development efforts, comprising of research and development, manufacturing, conducting clinical trials for its product candidates, protecting its intellectual property, pre-commercialization activities and general and administrative functions relating to these operations. The future success of the Company is dependent on its ability to develop its product candidates and ultimately upon its ability to attain profitable operations.

In February 2021, the Company completed the sale of a Rare Pediatric Disease Priority Review Voucher, or PRV, that it received in connection with the approval of IMCIVREE for $100,000. As the PRV did not have a carrying value, the gain recognized within Other income (loss) was equal to the gross proceeds received, with costs related to the sale of the voucher recorded within selling, general and administrative expenses.

At June 30, 2021, the Company had $368,154 of cash and cash equivalents and short-term investments on hand. In the future, the Company will be dependent on obtaining funding from third parties, such as proceeds from the issuance of debt, sale of equity, product sales and funded research and development programs to maintain the Company's operations and meet the Company's obligations. There is no guarantee that additional equity or other financings will be available to the Company on acceptable terms, or at all. If the Company fails to obtain additional funding when needed, the Company would be forced to scale back, terminate its operations or seek to merge with or be acquired by another company. Management believes that the Company's existing cash resources will be sufficient to fund the Company's operations into at least the second half of 2023.

Summary of Significant Accounting Policies

6 Months Ended

Jun. 30, 2021

Summary of Significant Accounting Policies

Summary of Significant Accounting Policies

2. Summary of Significant Accounting Policies

Basis of Presentation

The Company's unaudited condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States, or GAAP, and the applicable rules and regulations of the Securities and Exchange Commission, or SEC, regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates, or ASU, of the Financial Accounting Standards Board, or FASB. As permitted under these rules, certain footnotes or other financial information that are normally required by GAAP have been condensed or omitted.

The accompanying interim balance sheet as of June 30, 2021, the statements of operations and comprehensive income (loss) for the three and six months ended June 30, 2021 and 2020, the statements of stockholders equity for the three and six months ended June 30, 2021 and 2020 and the statements of cash flows for the six months ended June 30, 2021 and 2020 and the related footnote disclosures are unaudited. In management's opinion, the unaudited interim financial statements have been prepared on the same basis as the audited financial statements as of and for the year ended December 31, 2020 and include all adjustments, which are all normal recurring adjustments, necessary for the fair presentation of the interim financial statements. The results for the six months ended June 30, 2021 are not necessarily indicative of the results expected for the full fiscal year, any other interim periods, or any future year or period.

The accompanying unaudited condensed consolidated financial statements reflect the application of certain significant accounting policies as described below and elsewhere in these notes to the unaudited condensed consolidated financial statements. As of June 30, 2021, there have been no material changes in the Company's significant accounting policies from those that were disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020.

Certain amounts totaling $63 in the consolidated statement of stockholders’ equity for the three months ended March 31, 2020, related to unrealized gains on marketable securities, have been reclassified from additional paid-in capital to accumulated other comprehensive income to conform to the current period presentation. This reclassification had no impact on the previously reported results of operations or cash flows for the three months ended March 31, 2020.

Risks and Uncertainties

There are many uncertainties regarding the COVID-19 pandemic, and the Company is closely monitoring the impact of the pandemic on all aspects of its business, including how the pandemic will impact its patients, employees, suppliers, vendors, business partners and distribution channels. While the pandemic did not materially affect the Company's financial results and business operations for the six months ended June 30, 2021, the Company is unable to predict the impact that COVID-19 will have on its financial position and operating results in future periods due to numerous uncertainties. The Company will continue to assess the evolving impact of the COVID-19 pandemic and will make adjustments to its operations as necessary.

Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements if these results differ from historical experience, or other assumptions do not turn out to be substantially accurate, even if such assumptions are reasonable when made. Significant estimates relied upon in preparing these financial statements include accruals related to research and development expenses, assumptions used to record stock-based compensation expense and the valuation allowance on the Company's deferred tax assets. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ materially from those estimates.

Principles of Consolidation

The consolidated financial statements include the accounts of Rhythm Pharmaceuticals, Inc. and its wholly-owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation.

Off-Balance Sheet Risk and Concentrations of Credit Risk

Financial instruments, which potentially subject the Company to significant concentration of credit risk, consist primarily of cash and cash equivalents and short-term investments, which are maintained at two federally insured financial institutions. The deposits held at these two institutions are in excess of federally insured limits. The Company has not experienced any losses in such accounts and management believes that the Company is not exposed to significant credit risk due to the financial position of the depository institutions in which those deposits are held. The Company has no off-balance sheet risk, such as foreign exchange contracts, option contracts, or other foreign hedging arrangements.

Segment Information

Operating segments are defined as components of an entity about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company currently operates in one business segment, which is the development and commercialization of therapies for patients with rare diseases. A single management team that reports to the Chief Executive Officer comprehensively manages the entire business. The Company does not operate separate lines of business with respect to its product or product candidates. Accordingly, the Company has one reportable segment.

Revenue Recognition

The Company recognizes revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers, or ASC 606. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services.

Product Revenue, Net

Subsequent to its regulatory approval in the U.S. on November 25, 2020, the Company began to sell IMCIVREE in the U.S. in March, 2021. The product is distributed through an exclusive third-party logistics, or 3PL, distribution agent that does not take title to the product. Once the product is delivered to the Company’s exclusive specialty pharmacy provider, our sole customer in the U.S., the customer (or “wholesaler”) takes title to the product. The wholesaler then distributes the product to health care providers and patients. In our exclusive distribution agreement with the 3PL company, the Company acts as principal because we retain control of the product. The Company generally does not offer returns of product sold to the customer.

Revenue from product sales are recognized when the customer obtains control of our product, which occurs at a point in time, upon transfer of title to the customer because at that point in time we have no ongoing obligations to the customer. There are no other performance obligations besides the sale of product. We classify payments to our customer or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses in our consolidated statements of operations. Otherwise, payments to a customer or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. Taxes collected from the customer relating to product sales and remitted to governmental authorities are excluded from revenue. Because our payment terms are generally forty-five days, we conclude there is not a significant financing component because the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred since the expected amortization period of the asset that we would have recognized is one year or less.

Reserves for Variable Consideration

Revenues from product sales are recorded at the net sales price, or the transaction price, which includes estimates of variable consideration for which reserves are established and which result from discounts, returns, chargebacks, rebates, co-pay assistance and other allowances that are offered within contracts between us and our customer, health care providers and other indirect customers relating to the sale of IMCIVREE. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known.

The following are the components of variable consideration related to product revenue:

Chargebacks: The Company estimates obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers and patients at prices lower than the list prices charged to our customer. The government and other entities charge us for the difference between what they pay for the product and the selling price to our customer. The Company records reserves for these chargebacks related to product sold to our customer during the reporting period, as well as our estimate of product that remains in the distribution channel at the end of the reporting period that we expect will be sold to qualified healthcare providers and patients in future periods.

Government rebates: The Company is subject to discount obligations under government programs, including Medicaid programs, Medicare and Tricare in the United States. We estimate Medicaid, Medicare and Tricare rebates based upon a range of possible outcomes that are probability-weighted for the estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses on our consolidated balance sheet. For Medicare, we also

estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program. On a quarterly basis, we update our estimates and record any adjustments in the period that we identify the adjustments.

Trade discounts and allowances: The Company provides customary invoice discounts on IMCIVREE sales to our U.S. customer for prompt payment that are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive and pay for various distribution services from our customer in the distribution channel. For services that are either not distinct from the sale of our product or for which we cannot reasonably estimate the fair value, such fees are classified as a reduction of product revenue.

Product Returns: Our customer has limited return rights related to the product’s damage or defect. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. Based on the distribution model for IMCIVREE and the price of IMCIVREE, the Company believes there will be minimal returns.

Other incentives: Other incentives include co-payment assistance the Company provides to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. The estimate is recorded as a reduction of revenue in the same period the related revenue is recognized.

During the three and six months ended June 30, 2021, we recorded product revenue, net, of $274 and $309. The table that summarizes balances and activity in each of the product revenue allowance and reserve categories has not been included for the three and six months ended June 30, 2021 due to the immateriality of the revenue recognized during the period.

Cost of Product Sales

Prior to receiving approval from the FDA in November 2020 to sell IMCIVREE in the United States, the Company expensed all costs incurred related to the manufacture of IMCIVREE as research and development expense because of the inherent risks associated with the development of a drug candidate, the uncertainty about the regulatory approval process and the lack of history for the Company of regulatory approval of drug candidates. Subsequent to receiving FDA approval in November 2020, the Company has capitalized a nominal amount of inventory related costs that were incurred subsequent to FDA approval. At June 30, 2021, the Company had $28 of inventory recorded as a component of other current assets on the condensed consolidated balance sheet.

Cost of product sales will consist of manufacturing costs, transportation and freight, amortization of capitalized intangibles, royalty payments and indirect overhead costs associated with the manufacturing and distribution of IMCIVREE. Cost of product sales may also include period costs related to certain manufacturing services and inventory adjustment charges. The Company is currently evaluating the impact of this previously expensed inventory on the future cost of product sales.

Accounts Receivable, Net

In general, accounts receivable consists of amounts due from customers, net of customer allowances for cash discounts and chargebacks. The Company's contracts with customers have standard payment terms that generally require payment within 45 days. The Company analyzes accounts that are past due for collectability, and periodically evaluates the creditworthiness of its customers. As of June 30, 2021, we determined an allowance for doubtful accounts was not required based upon our review of contractual payment terms and individual customer circumstances.

Intangible Assets, Net

Definite-lived intangible assets related to capitalized milestones under license agreements are amortized on a straight-line basis over their remaining useful lives, which are estimated to be the remaining patent life. If our estimate of

the product’s useful life is shorter than the remaining patent life, then a shorter period is used. Amortization expense is recorded as a component of cost of sales on the consolidated statements of operations and comprehensive income (loss).

Intangible assets are evaluated for impairment at least annually in the fourth quarter or more frequently if impairment indicators exist. Events that could result in an impairment, or trigger an interim impairment assessment, include the decision to discontinue the development of a drug, the receipt of additional clinical or nonclinical data regarding our drug candidate or a potentially competitive drug candidate, changes in the clinical development program for a drug candidate, or new information regarding potential sales for the drug. In connection with any impairment assessment, the fair value of the intangible assets as of the date of assessment is compared to the carrying value of the intangible asset. Impairment losses are recognized if the carrying value of an intangible asset is both not recoverable and exceeds its fair value.

Fair Value Measurements

Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. Financial assets and liabilities carried at fair value are classified and disclosed in one of the following three categories:

Level 1 — Quoted market prices in active markets for identical assets or liabilities.

Level 2 — Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities.

Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities.

The Company’s cash equivalents and marketable securities at June 30, 2021 and December 31, 2020 were carried at fair value, determined according to the fair value hierarchy. See Note 4 for further discussion.

The carrying amounts reflected in the consolidated balance sheets for accounts payable and accrued expenses approximate their fair values due to their short-term maturities at June 30, 2021 and December 31, 2020, respectively.

Net Income (Loss) Per Share

Basic net income (loss) per share is computed by dividing the net income (loss) by the weighted-average number of common shares outstanding during the period, without consideration of potential dilutive securities. Diluted net income (loss) per common share is computed by adjusting the weighted-average shares outstanding for the potential dilutive effects of common stock equivalents outstanding during the period calculated in accordance with the treasury stock method. For purposes of the diluted net income (loss) per share calculation, 640,318 stock options and 73,259 restricted stock units were considered to be common stock equivalents for the three and six months ended June 30, 2021. For the three and six months ended June 30, 2020, the common stock equivalents have been excluded from the calculation of diluted net income (loss) per share, as their effect would be anti-dilutive for the period presented due to the net losses incurred for such periods.

The following table includes the potential common shares, presented based on amounts outstanding at each period end, that were excluded from the computation of diluted net loss per share due to their anti-dilutive effect, for the periods indicated:


	Three Months Ended				Six Months Ended
	June 30,				June 30,
	2021		2020		2021		2020
Stock options		5,151,118		4,239,682		4,691,373		4,239,682
Restricted stock units		185,176		204,662		133,286		204,662
Potential common shares		5,336,294		4,444,344		4,824,659		4,444,344

Subsequent Events

The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required.

Application of New or Revised Accounting Standards

From time to time, new accounting pronouncements are issued by the FASB and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its financial position or results of operations upon adoption.

In June 2016, the FASB issued ASU 2016-13, Financial Instruments-Credit Losses-Measurement of Credit Losses on Financial Instruments, which has been subsequently amended by ASU No. 2018-19, ASU No. 2019-04, ASU No. 2019-05, ASU No. 2019-10, ASU No. 2019-11 and ASU No. 2020-03, or ASU 2016-13. The provisions of ASU 2016-13 modify the impairment model to utilize an expected loss methodology in place of the currently used incurred loss methodology and require a consideration of a broader range of reasonable and supportable information to inform credit loss estimates. Since the Company ceased to be an emerging growth company as of December 31, 2020, the Company adopted the standard during the fourth quarter of 2020 and applied the modified retrospective method of adoption to the Company’s financial statements as of January 1, 2020. Based on the composition of the investment portfolio as of the adoption date, the adoption of this standard did not have a material impact on the Company’s financial position, results of operations and cash flows for the year ended December 31 2020 and no adjustment was required to be recorded to the opening retained earnings balance as of January 1, 2020.

In December 2019, the FASB issued ASU 2019-12, Income Taxes-Simplifying the Accounting for Income Taxes, or ASU 2019-12. ASU 2019-12 eliminates certain exceptions related to the approach for intraperiod tax allocation, the methodology for calculating income taxes in an interim period and the recognition of deferred tax liabilities for outside basis differences. The new guidance also simplifies aspects of the accounting for franchise taxes and enacted changes in tax laws or rates and clarifies the accounting for transactions that result in a step-up in the tax basis of goodwill. The standard is effective for annual periods beginning after December 15, 2020 and interim periods within, with early adoption permitted. Adoption of the standard requires certain changes to be made prospectively, with some changes to be made retrospectively. We have adopted ASU 2019-12 as of January 1, 2021 and the adoption of this standard did not have a material impact on the Company’s financial position, results of operations and cash flows.

Accrued Expenses

6 Months Ended

Jun. 30, 2021

Accrued Expenses

Accrued Expenses

3. Accrued Expenses

Accrued expenses consisted of the following:


	June 30,		December 31,
	2021		2020
Research and development costs	$	5,663	$	5,815
Professional fees		1,688		648
Payroll related		4,188		5,916
Other		341		180
Accrued expenses	$	11,880	$	12,559

Fair Value of Financial Assets

6 Months Ended

Jun. 30, 2021

Fair Value of Financial Assets

Fair Value of Financial Assets

4. Fair Value of Financial Assets

As of June 30, 2021 and December 31, 2020, the carrying amount of cash and cash equivalents and short-term investments was $368,154 and $172,792, respectively, which approximates fair value. Cash and cash equivalents and short-term investments includes investments in U.S. treasury securities and money market funds that invest in U.S. government securities that are valued using quoted market prices. Accordingly, money market funds and government funds are categorized as Level 1. The financial assets valued based on Level 2 inputs consist of corporate debt securities and commercial paper, which consist of investments in highly-rated investment-grade corporations.

The following tables present information about the Company's financial assets measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values:


	Fair value Measurements as of
	June 30, 2021 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash Equivalents:
Commercial Paper	$	—	$	14,998	$	—	$	14,998
Money Market Funds		45,941		—		—		45,941
Marketable Securities:
Corporate Debt Securities and Commercial Paper		—		298,815		—		298,815
Total	$	45,941	$	313,813	$	—	$	359,754


	Fair value Measurements as of
	December 31, 2020 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash Equivalents:
Corporate Debt Securities and Commercial Paper	$	—	$	36,242	$	—	$	36,242
Money Market Funds		63,182		—		—		63,182
Marketable Securities:
Corporate Debt Securities and Commercial Paper		—		71,938		—		71,938
Total	$	63,182	$	108,180	$	—	$	171,362

Marketable Securities

The following tables summarize the Company's marketable securities:


	June 30, 2021
			Gross		Gross
	Amortized		Unrealized		Unrealized		Fair
	Cost		Gains		Losses		Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	298,794	$	25	$	(4)	$	298,815
	$	298,794	$	25	$	(4)	$	298,815


	December 31, 2020
			Gross		Gross
	Amortized		Unrealized		Unrealized		Fair
	Cost		Gains		Losses		Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	71,895	$	43	$	—	$	71,938
	$	71,895	$	43	$	—	$	71,938

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

Right of Use Asset and Lease Liability

6 Months Ended

Jun. 30, 2021

Right Of Use Asset and Lease Liability

Right Of Use Asset and Lease Liability

5. Right Of Use Asset and Lease Liability

The Company has a material operating lease for its head office facility and other immaterial operating leases for certain equipment. The Company’s office lease has a remaining lease term of 4.0 years. The Company measured the lease liability associated with the office lease using a discount rate of 10% at inception. The Company estimated the incremental borrowing rate for the leased asset based on a range of comparable interest rates the Company would incur to borrow an amount equal to the lease payments on a collateralized basis over a similar term in a similar economic environment. As of June 30, 2021, the Company has not entered into any lease arrangements classified as a finance lease.

The Company’s corporate headquarters is located in Boston, Massachusetts. This facility houses the Company’s research, clinical, regulatory, commercial and administrative personnel. The Company’s lease agreement commenced May 2019 and has a term of six years with a five-year renewal option to extend the lease. The Company has not included the five-year renewal option to extend the lease in its measurement of the ROU asset or lease liability.

The following table presents the maturities of the Company’s operating lease liability related to office space as of June 30, 2021, all of which is under a non-cancellable operating lease:


	Operating Lease
Remainder of 2021	$	404
2022		818
2023		834
2024		851
2025		502
Thereafter		—
Total operating lease payments		3,409
Less: imputed interest		581
Total operating lease liability	$	2,828

Intangible Assets, Net

6 Months Ended

Jun. 30, 2021

Intangible Assets, Net

Intangible Assets, Net

6. Intangible Assets, Net

As of June 30, 2021, the Company’s finite-lived intangible assets, which totaled $4,886, resulted from the capitalization of certain milestone payments made to Ipsen Pharma, S.A.S., or Ipsen, in accordance with the terms of the

Company’s license agreement with Ipsen, in connection with the Company’s first commercial sale of IMCIVREE in the U.S. in March 2021.


2021	$	228
2022		454
2023		455
2024		455
2025		455
Thereafter		2,839
Total	$	4,886

The Company began amortizing its finite-lived intangible assets in April 2021 over an 11 year period based on IMCIVREE’s expected patent exclusivity period. Amortization expense totaled $114 for the three and six months ended June 30, 2021. Amortization expense will be included in cost of sales on the consolidated statements of operations and comprehensive loss.

Income Taxes

6 Months Ended

Jun. 30, 2021

Income Taxes

Income Taxes

7. Income Taxes

The Company recorded a tax ( benefit) of ($5,022) for the three month period ended June 30, 2021. The Company recorded a tax provision of $16,984 for the six month period ended June 30, 2021. The sale of the PRV resulted in a tax provision of $22,006 recorded during the three month period ended March 31, 2021, which will be offset by ordinary losses generated by the Company over the remainder of current year. The Company expects to have sufficient tax losses in the current year to offset the income from the sale and thus no current year liability is expected. The Company expects to maintain a full valuation allowance against its net deferred tax assets for the year.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References + Details

Common Stock

6 Months Ended

Jun. 30, 2021

Common Stock

Common Stock

8. Common Stock

On February 9, 2021 the Company completed a public offering of 5,750,000 shares of common stock at an offering price of $30.00 per share, which included the exercise in full by the underwriters of their option to purchase up to 750,000 additional shares of common stock. The Company received $161,731 in net proceeds after deducting underwriting discounts, commissions and offering expenses.

During the three months ended March 31, 2020, the Company entered into a separation agreement with its former Chief Executive Officer, Keith Gottesdiener, M.D. The Company modified certain equity awards held by Dr. Gottesdiener. The modification included the continuation of vesting of stock options through the end of December 31, 2020 and an extension of the post-termination exercise period for vested options from 90 days to up to two years. In connection with this modification, the Company recorded an incremental compensation charge of $2,811 during the six months ended June 30, 2020.

As of June 30, 2021, an aggregate of 10,014,129 shares of common stock were reserved for future issuance under the Company’s stock plans, including outstanding stock options and restricted stock units that have been issued of 6,277,702 shares of common stock and 983,993 shares are available for future grants under the Company’s 2017 Employee Stock Purchase Plan.

Related-Party Transactions

6 Months Ended

Jun. 30, 2021

Related-Party Transactions

Related-Party Transactions

9. Related-Party Transactions

Expenses paid directly to consultants and vendors considered to be related parties amounted to $487, $916, $1,097, and $1,776 for the three and six months ended June 30, 2021 and 2020, respectively. Outstanding payments due to these related parties as of June 30, 2021 and December 31, 2020 were $0 and $187, respectively, and were included within accounts payable on the balance sheet.

Summary of Significant Accounting Policies (Policies)

6 Months Ended

Jun. 30, 2021

Summary of Significant Accounting Policies

Basis of Presentation

Risks and Uncertainties

Use of Estimates

Principles of Consolidation

Off-Balance Sheet Risk and Concentrations of Credit Risk

Segment Information

Revenue Recognition

Product Revenue, Net

Reserves for Variable Consideration

The following are the components of variable consideration related to product revenue:

Accounts Receivable, Net

Intangible Assets, Net

Fair Value Measurements

Level 1 — Quoted market prices in active markets for identical assets or liabilities.

Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities.

Net Income (Loss) Per Share


	Three Months Ended				Six Months Ended
	June 30,				June 30,
	2021		2020		2021		2020
Stock options		5,151,118		4,239,682		4,691,373		4,239,682
Restricted stock units		185,176		204,662		133,286		204,662
Potential common shares		5,336,294		4,444,344		4,824,659		4,444,344

Subsequent Events

Application of New or Revised Accounting Standards

Summary of Significant Accounting Policies (Tables)

6 Months Ended

Jun. 30, 2021

Summary of Significant Accounting Policies

Schedule of potential common shares, presented based on amounts outstanding at each period end, excluded from computation of diluted net loss per share attributable to common stockholders


	Three Months Ended				Six Months Ended
	June 30,				June 30,
	2021		2020		2021		2020
Stock options		5,151,118		4,239,682		4,691,373		4,239,682
Restricted stock units		185,176		204,662		133,286		204,662
Potential common shares		5,336,294		4,444,344		4,824,659		4,444,344

Accrued Expenses (Tables)

6 Months Ended

Jun. 30, 2021

Accrued Expenses

Schedule of accrued expenses


	June 30,		December 31,
	2021		2020
Research and development costs	$	5,663	$	5,815
Professional fees		1,688		648
Payroll related		4,188		5,916
Other		341		180
Accrued expenses	$	11,880	$	12,559

Fair Value of Financial Assets (Tables)

6 Months Ended

Jun. 30, 2021

Fair Value of Financial Assets

Schedule of financial assets and liabilities measured at fair value on a recurring basis


	Fair value Measurements as of
	June 30, 2021 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash Equivalents:
Commercial Paper	$	—	$	14,998	$	—	$	14,998
Money Market Funds		45,941		—		—		45,941
Marketable Securities:
Corporate Debt Securities and Commercial Paper		—		298,815		—		298,815
Total	$	45,941	$	313,813	$	—	$	359,754


	Fair value Measurements as of
	December 31, 2020 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash Equivalents:
Corporate Debt Securities and Commercial Paper	$	—	$	36,242	$	—	$	36,242
Money Market Funds		63,182		—		—		63,182
Marketable Securities:
Corporate Debt Securities and Commercial Paper		—		71,938		—		71,938
Total	$	63,182	$	108,180	$	—	$	171,362

Schedule of marketable securities


	June 30, 2021
			Gross		Gross
	Amortized		Unrealized		Unrealized		Fair
	Cost		Gains		Losses		Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	298,794	$	25	$	(4)	$	298,815
	$	298,794	$	25	$	(4)	$	298,815


	December 31, 2020
			Gross		Gross
	Amortized		Unrealized		Unrealized		Fair
	Cost		Gains		Losses		Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	71,895	$	43	$	—	$	71,938
	$	71,895	$	43	$	—	$	71,938

Right of Use Asset and Lease Liability (Tables)

6 Months Ended

Jun. 30, 2021

Right Of Use Asset and Lease Liability

Schedule of operating lease maturities


	Operating Lease
Remainder of 2021	$	404
2022		818
2023		834
2024		851
2025		502
Thereafter		—
Total operating lease payments		3,409
Less: imputed interest		581
Total operating lease liability	$	2,828

Intangible Assets, Net (Tables)

6 Months Ended

Jun. 30, 2021

Intangible Assets, Net

Schedule of finite-lived intangible assets


2021	$	228
2022		454
2023		455
2024		455
2025		455
Thereafter		2,839
Total	$	4,886

Nature of Business (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended	6 Months Ended
Nature of Business (Details) - USD ($) $ in Thousands	Feb. 28, 2021	Jun. 30, 2021	Jun. 30, 2021	Dec. 31, 2020
Nature of Business
Accumulated deficit		$ (450,966)	$ (450,966)	$ (459,327)
Proceeds From Sale Of Priority Review Voucher	$ 100,000		100,000
Product revenue, net		274	309
Carrying amount of cash and cash equivalents and short term investments		$ 368,154	$ 368,154	$ 172,792

- Definition

Amount, including tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value-added and excise.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 41
-Subparagraph (a)
-URI http://asc.fasb.org/extlink&oid=120311839&loc=d3e9038-108599

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 5
-URI http://asc.fasb.org/extlink&oid=121604090&loc=SL49130545-203045

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (b)
-URI http://asc.fasb.org/extlink&oid=120311839&loc=d3e8933-108599

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (a)
-URI http://asc.fasb.org/extlink&oid=120311839&loc=d3e8933-108599

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 30
-Subparagraph (a)
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
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-URI http://asc.fasb.org/extlink&oid=120311839&loc=d3e8736-108599

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
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-Section 50
-Paragraph 4
-Subparagraph (a)
-URI http://asc.fasb.org/extlink&oid=121604090&loc=SL49130543-203045

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 42
-URI http://asc.fasb.org/extlink&oid=120311839&loc=d3e9054-108599

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 40
-URI http://asc.fasb.org/extlink&oid=120311839&loc=d3e9031-108599

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 924
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 11.L)
-URI http://asc.fasb.org/extlink&oid=6472922&loc=d3e499488-122856

+ Details

Summary of Significant Accounting Policies (Details) $ in Thousands	3 Months Ended			6 Months Ended
	Jun. 30, 2021 USD ($)	Jun. 30, 2020 USD ($)	Mar. 31, 2020 USD ($)	Jun. 30, 2021 USD ($) item segment
Summary of Significant Accounting Policies
Unrealized gain on marketable securities		$ 567	$ 63
Off Balance Sheet Risk and Concentrations of Credit Risk
Number of federally insured financial institutions \| item				2
Segment Information
Number of operating segments \| segment				1
Number of reporting segments \| segment				1
Revenue Recognition
Payment terms				45 days
Product revenue, net	$ 274			$ 309
Accounts receivable standard payment terms				45 days
Other current assets
Inventory
Inventory	$ 28			$ 28

Summary of Significant Accounting Policies - Shares Excluded For EPS (Details) - shares	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Shares excluded from the computation of diluted net loss per share
Common stock equivalents excluded from the computation of diluted net loss per share due to their anti-dilutive effect (in shares)	50,209,484	44,098,860	49,644,704	44,074,352
Shares excluded from the computation of diluted net loss per share	5,336,294	4,444,344	4,824,659	4,444,344
Stock options
Shares excluded from the computation of diluted net loss per share
Common stock equivalents excluded from the computation of diluted net loss per share due to their anti-dilutive effect (in shares)	640,318		640,318
Shares excluded from the computation of diluted net loss per share	5,151,118	4,239,682	4,691,373	4,239,682
Restricted stock units
Shares excluded from the computation of diluted net loss per share
Common stock equivalents excluded from the computation of diluted net loss per share due to their anti-dilutive effect (in shares)	73,259		73,259
Shares excluded from the computation of diluted net loss per share	185,176	204,662	133,286	204,662

Summary of Significant Accounting Policies - Application of New or Revised Accounting Standards (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Summary of Significant Accounting Policies
Right-of-use asset	$ 1,671	$ 1,807
Operating Lease, Liability	$ 2,828

Accrued Expenses (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Accrued Expenses
Research and development costs	$ 5,663	$ 5,815
Professional fees	1,688	648
Payroll related	4,188	5,916
Other	341	180
Accrued expenses	$ 11,880	$ 12,559

Fair Value of Financial Assets (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Fair Value of Financial Assets and Liabilities
Carrying amount of cash and cash equivalents and short term investments	$ 368,154	$ 172,792
Fair value of financial assets and liabilities
Marketable Securities	298,815	71,938
Corporate Debt Securities and Commercial Paper
Fair value of financial assets and liabilities
Marketable Securities	298,815	71,938
Recurring \| Estimated fair value
Fair value of financial assets and liabilities
Fair value of assets	359,754	171,362
Recurring \| Commercial Paper \| Estimated fair value
Fair value of financial assets and liabilities
Cash Equivalents	14,998
Recurring \| Corporate Debt Securities and Commercial Paper \| Estimated fair value
Fair value of financial assets and liabilities
Cash Equivalents		36,242
Marketable Securities	298,815	71,938
Recurring \| Money Market Funds \| Estimated fair value
Fair value of financial assets and liabilities
Cash Equivalents	45,941	63,182
Recurring \| Level 1 \| Estimated fair value
Fair value of financial assets and liabilities
Fair value of assets	45,941	63,182
Recurring \| Level 1 \| Money Market Funds \| Estimated fair value
Fair value of financial assets and liabilities
Cash Equivalents	45,941	63,182
Recurring \| Level 2 \| Estimated fair value
Fair value of financial assets and liabilities
Fair value of assets	313,813	108,180
Recurring \| Level 2 \| Commercial Paper \| Estimated fair value
Fair value of financial assets and liabilities
Cash Equivalents	14,998
Recurring \| Level 2 \| Corporate Debt Securities and Commercial Paper \| Estimated fair value
Fair value of financial assets and liabilities
Cash Equivalents		36,242
Marketable Securities	$ 298,815	$ 71,938

Fair Value of Financial Assets - Marketable Securities (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Fair Value of Financial Assets and Liabilities
Amortized Cost	$ 298,794	$ 71,895
Gross Unrealized Gains	25	43
Gross Unrealized Losses	(4)
Fair Value	298,815	71,938
Corporate Debt Securities and Commercial Paper
Fair Value of Financial Assets and Liabilities
Amortized Cost	298,794	71,895
Gross Unrealized Gains	25	43
Gross Unrealized Losses	(4)
Fair Value	298,815	71,938
Recurring \| Estimated fair value \| Corporate Debt Securities and Commercial Paper
Fair Value of Financial Assets and Liabilities
Fair Value	298,815	71,938
Recurring \| Level 2 \| Estimated fair value \| Corporate Debt Securities and Commercial Paper
Fair Value of Financial Assets and Liabilities
Fair Value	$ 298,815	$ 71,938

Right of Use Asset and Lease Liability (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2021	Dec. 31, 2020
Right Of Use Asset and Lease Liability
Remaining term of operating lease (in years)	4 years
Operating lease discount rate	10.00%
Right-of-use asset	$ 1,671	$ 1,807
Operating Lease, Liability	$ 2,828
Lease term (in years)	6 years
Lease renewal term (in years)	5 years
Option to extend	true

Right of Use Asset and Lease Liability - Operating Lease Maturities (Details) $ in Thousands	Jun. 30, 2021 USD ($)
Right Of Use Asset and Lease Liability
Remainder of 2021	$ 404
2022	818
2023	834
2024	851
2025	502
Total operating lease payments	3,409
Less: imputed interest	581
Total operating lease liability	$ 2,828

Intangible Assets, Net (Details) $ in Thousands	3 Months Ended	6 Months Ended
Intangible Assets, Net (Details) $ in Thousands	Jun. 30, 2021 USD ($)	Jun. 30, 2021 USD ($)
Finite-Lived Intangible Assets, Net, Amortization Expense, Fiscal Year Maturity [Abstract]
2021	$ 228	$ 228
2022	454	454
2023	455	455
2024	455	455
2025	455	455
Thereafter	2,839	2,839
Total acquired lease intangible assets, net	4,886	$ 4,886
Amortization period		11 years
Amortization of Intangible Assets	$ 114	$ 114

Income Taxes (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
Income Taxes (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Mar. 31, 2021	Jun. 30, 2021
Income Taxes
Income taxes	$ (5,022)	$ 22,006	$ 16,984

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References + Details

Common Stock (Details) - USD ($) $ / shares in Units, $ in Thousands		3 Months Ended	6 Months Ended
	Feb. 09, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020
Common Stock
Common stock, authorized			120,000,000		120,000,000
Price per share	$ 30.00
Issuance of stock (in shares)	5,750,000
Net proceeds from issuance of common stock	$ 161,731		$ 161,731
Incremental compensation charge				$ 2,811
Maximum \| Underwriter option to purchase
Common Stock
Additional shares	750,000
Former Chief Executive Officer, Keith Gottesdiener, M.D. \| Minimum
Common Stock
Post-termination exercise period for vested options		90 days
Former Chief Executive Officer, Keith Gottesdiener, M.D. \| Maximum
Common Stock
Post-termination exercise period for vested options		2 years
2017 Employee Stock Purchase Plan
Common Stock
Common stock reserved for issuance			10,014,129
Shares available for future grant			983,993
2017 Employee Stock Purchase Plan \| Equity Option
Common Stock
Common stock reserved for issuance			6,277,702

Related-Party Transactions (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020
Accounts Payable
Related Party Transaction
Outstanding payments due to consultants	$ 0		$ 0		$ 187
Consultant
Related Party Transaction
Expenses paid to consultants	$ 487	$ 916	$ 1,097	$ 1,776,000