PROTHENA CORP PUBLIC LTD CO
For the period ended Sep 30, 2024. Filed Nov 12, 2024.
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Sep 30, 2024 · Filed Nov 12, 2024
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Item 1. FINANCIAL STATEMENTS
Condensed Consolidated Balance Sheets (unaudited)
(in thousands, except share and per share data)
| September 30, | December 31, | |
|---|---|---|
| 2024 | 2023 | |
| Assets | ||
| Current assets: | ||
| Cash and cash equivalents | $519,262 | $618,830 |
| Accounts receivable | — | 5,159 |
| Prepaid expenses and other current assets | 15,722 | 13,941 |
| Restricted cash, current | — | 1,352 |
| Total current assets | 534,984 | 639,282 |
| Non-current assets: | ||
| Property and equipment, net | 3,254 | 3,836 |
| Operating lease right-of-use assets | 11,400 | 12,162 |
| Deferred tax assets | 41,002 | 33,893 |
| Restricted cash, non-current | 860 | 860 |
| Other non-current assets | 3,754 | 6,349 |
| Total non-current assets | 60,270 | 57,100 |
| Total assets | $595,254 | $696,382 |
| Liabilities and Shareholders’ Equity | ||
| Current liabilities: | ||
| Accounts payable | $10,330 | $25,391 |
| Accrued research and development | 10,340 | 14,724 |
| Deferred revenue, current | 8,832 | — |
| Lease liability, current | 2,613 | 1,114 |
| Other current liabilities | 14,307 | 15,662 |
| Total current liabilities | 46,422 | 56,891 |
| Non-current liabilities: | ||
| Deferred revenue, non-current | 5,589 | 67,405 |
| Lease liability, non-current | 8,881 | 10,721 |
| Total non-current liabilities | 14,470 | 78,126 |
| Total liabilities | 60,892 | 135,017 |
| Commitments and contingencies (Note 6) | ||
| Shareholders’ equity: | ||
| Euro deferred shares, €22 nominal value: | — | — |
| Ordinary shares, $0.01 par value: | 538 | 537 |
| Issued and outstanding shares — 53,808,732 and 53,682,117 at September 30, 2024 and December 31, 2023, respectively | ||
| Additional paid-in capital | 1,578,209 | 1,540,859 |
| Accumulated deficit | (1,044,385) | (980,031) |
| Total shareholders’ equity | 534,362 | 561,365 |
| Total liabilities and shareholders’ equity | $595,254 | $696,382 |
Condensed Consolidated Statements of Operations
(in thousands, except per share data)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Collaboration revenue | $970 | $84,866 | $132,984 | $91,004 |
| Revenue from license and intellectual property | — | — | 50 | 50 |
| Total revenue | 970 | 84,866 | 133,034 | 91,054 |
| Operating expenses: | ||||
| Research and development | 50,723 | 57,913 | 172,347 | 158,680 |
| General and administrative | 16,760 | 16,645 | 50,351 | 44,895 |
| Total operating expenses | 67,483 | 74,558 | 222,698 | 203,575 |
| Income (loss) from operations | (66,513) | 10,308 | (89,664) | (112,521) |
| Other income (expense): | ||||
| Interest income | 6,743 | 8,522 | 20,429 | 22,912 |
| Other expense, net | (66) | (15) | (194) | (253) |
| Total other income, net | 6,677 | 8,507 | 20,235 | 22,659 |
| Income (loss) before income taxes | (59,836) | 18,815 | (69,429) | (89,862) |
| Benefit from income taxes | (835) | (3,092) | (5,075) | (10,310) |
| Net income (loss) | $(59,001) | $21,907 | $(64,354) | $(79,552) |
| Basic net income (loss) per ordinary share | $(1.10) | $0.41 | $(1.20) | $(1.50) |
| Diluted net income (loss) per ordinary share | $(1.10) | $0.38 | $(1.20) | $(1.50) |
| Shares used to compute basic net income (loss) per share | 53,790 | 53,559 | 53,757 | 53,064 |
| Shares used to compute diluted net income (loss) per share | 53,790 | 58,004 | 53,757 | 53,064 |
Condensed Consolidated Statements of Cash Flows
(in thousands)
| Nine Months Ended September 30, | ||
|---|---|---|
| 2024 | 2023 | |
| Operating activities | ||
| Net loss | $(64,354) | $(79,552) |
| Adjustments to reconcile net loss to cash used in operating activities: | ||
| Depreciation | 672 | 652 |
| Share-based compensation | 35,444 | 29,834 |
| Deferred income taxes | (7,109) | (11,712) |
| Reduction in the carrying amount of right-of-use assets | 2,000 | 5,221 |
| Changes in operating assets and liabilities: | ||
| Accounts receivable | 5,159 | (5,159) |
| Prepaid and other assets | 768 | (6,824) |
| Accounts payable, accruals and other liabilities | (20,289) | 18,496 |
| Deferred revenue | (52,984) | (29,014) |
| Operating lease liabilities | (1,585) | (4,810) |
| Net cash used in operating activities | (102,278) | (82,868) |
| Investing activities | ||
| Purchases of property and equipment | (255) | (1,261) |
| Net cash used in investing activities | (255) | (1,261) |
| Financing activities | ||
| Proceeds from issuance of ordinary shares in public offering, net | — | 20,689 |
| Proceeds from issuance of ordinary shares in at-the-market offering, net | (295) | 2,959 |
| Proceeds from issuance of ordinary shares upon exercise of stock options | 1,908 | 20,972 |
| Net cash provided by financing activities | 1,613 | 44,620 |
| Net decrease in cash, cash equivalents and restricted cash | (100,920) | (39,509) |
| Cash, cash equivalents and restricted cash, beginning of the year | 621,042 | 712,618 |
| Cash, cash equivalents and restricted cash, end of the period | $520,122 | $673,109 |
| Supplemental disclosures of cash flow information | ||
| Cash paid for income taxes | $2,412 | $384 |
| Supplemental disclosures of non-cash investing and financing activities | ||
| Acquisition of property and equipment included in accounts payable and accrued liabilities | $71 | $93 |
| Right-of-use assets obtained in exchange for lease obligations | $217 | $3,810 |
| Reclassification of prepaid lease payments to right-of-use assets upon lease commencement | $— | $7,522 |
| At-the-market offering costs included in accounts payable and accrued liabilities | $— | $8 |
The following table provides a reconciliation of cash, cash equivalents and restricted cash reported within the statement of financial position that sum to the total of the same such amounts shown in the Condensed Consolidated Statements of Cash Flows.
| Nine Months Ended September 30, | ||
|---|---|---|
| 2024 | 2023 | |
| Cash and cash equivalents | $519,262 | $670,897 |
| Restricted cash, current | — | 1,352 |
| Restricted cash, non-current | 860 | 860 |
| Total cash, cash equivalents and restricted cash, end of the period | $520,122 | $673,109 |
Condensed Consolidated Statements of Shareholders' Equity
(in thousands, except share data)
| Ordinary Shares · Additional Paid-in Capital · Accumulated Deficit · Total Shareholders' Equity | |||||||
|---|---|---|---|---|---|---|---|
| Shares · Amount | |||||||
| Balances at December 31, 2023 | 53,682,117 | $537 | $1,540,859 | $(980,031) | $561,365 | ||
| Share-based compensation | 12,383 | 12,383 | |||||
| Issuance of ordinary shares upon exercise of stock options | 38,338 | — | 890 | 890 | |||
| Net loss | (72,239) | (72,239) | |||||
| Balances at March 31, 2024 | 53,720,455 | 537 | 1,554,132 | (1,052,270) | 502,399 | ||
| Share-based compensation | 12,041 | 12,041 | |||||
| Issuance of ordinary shares upon exercise of stock options | 51,390 | 1 | 590 | 591 | |||
| Net income | 66,886 | 66,886 | |||||
| Balances at June 30, 2024 | 53,771,845 | 538 | 1,566,763 | (985,384) | 581,917 | ||
| Share-based compensation | 11,020 | 11,020 | |||||
| Issuance of ordinary shares upon exercise of stock options | 35,887 | — | 426 | 426 | |||
| Issuance of ordinary shares upon vesting of restricted stock units | 1,000 | — | — | — | |||
| Net income | (59,001) | (59,001) | |||||
| Balances at September 30, 2024 | 53,808,732 | $538 | $1,578,209 | $(1,044,385) | $534,362 | ||
| Ordinary Shares · AdditionalPaid-inCapital · AccumulatedDeficit · TotalShareholders' Equity | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| Shares · Amount | |||||||||
| Balances at December 31, 2022 | 52,103,608 | $521 | $1,454,524 | $(833,003) | $622,042 | ||||
| Share-based compensation | 8,790 | 8,790 | |||||||
| Issuance of ordinary shares upon exercise of stock options | 179,474 | 2 | 2,538 | 2,540 | |||||
| Issuance of ordinary shares from the underwriters partial exercise of their 30-day option to purchase additional shares as part of the December 2022 public offering, net of issuance costs of $1.4 million | 395,096 | 4 | 20,897 | 20,901 | |||||
| Net loss | (46,864) | (46,864) | |||||||
| Balances at March 31, 2023 | 52,678,178 | 527 | 1,486,749 | (879,867) | 607,409 | ||||
| Share-based compensation | 10,100 | 10,100 | |||||||
| Issuance of ordinary shares upon exercise of stock options | 772,928 | 8 | 15,651 | 15,659 | |||||
| Adjustment related to December 2022 public offering | 2 | 2 | |||||||
| Issuance of ordinary shares under the at-the-market offering program, net of issuance costs of $119 thousand | 42,361 | — | 3,104 | 3,104 | |||||
| Net loss | (54,595) | (54,595) | |||||||
| Balances at June 30, 2023 | 53,493,467 | 535 | 1,515,606 | (934,462) | 581,679 | ||||
| Ordinary Shares · AdditionalPaid-inCapital · AccumulatedDeficit · TotalShareholders' Equity | |||||||
|---|---|---|---|---|---|---|---|
| Shares · Amount | |||||||
| Share-based compensation | 10,944 | 10,944 | |||||
| Adjustment related to the at-the-market offering program | (14) | (14) | |||||
| Issuance of ordinary shares upon exercise of stock options | 145,148 | 1 | 2,710 | 2,711 | |||
| Net income | 21,907 | 21,907 | |||||
| Balances at September 30, 2023 | 53,638,615 | $536 | $1,529,246 | $(912,555) | $617,227 | ||
Notes to the Condensed Consolidated Financial Statements
- Organization
Description of Business
Prothena Corporation plc (“Prothena” or the “Company”) is a late-stage clinical biotechnology company with expertise in protein dysregulation and a pipeline of investigational therapeutics with the potential to change the course of devastating neurodegenerative and rare peripheral amyloid diseases.
Fueled by its deep scientific expertise built over decades of research, the Company is advancing a pipeline of therapeutic candidates for a number of indications and novel targets for which its ability to integrate scientific insights around neurological dysfunction and the biology of misfolded proteins can be leveraged. The Company’s wholly-owned programs include birtamimab for the potential treatment of AL amyloidosis, and a portfolio of programs for the potential treatment of Alzheimer’s disease including PRX012, which targets amyloid beta (Aβ), and PRX123, a novel dual Aβ-tau vaccine. The Company’s partnered programs include prasinezumab for the potential treatment of Parkinson’s disease and other related synucleinopathies that targets alpha-synuclein in collaboration with Roche. In addition, we have partnered BMS-986446 (formerly PRX005) for the potential treatment of Alzheimer’s disease that targets tau and PRX019 for the potential treatment of neurodegenerative diseases with an undisclosed target in two separate license agreements with Bristol Myers Squibb (BMS). The Company is also entitled to certain potential milestone payments pursuant to the Company’s share purchase agreement with Novo Nordisk pertaining to the Company’s ATTR amyloidosis business (inclusive of coramitug, formerly PRX004).
The Company was formed on September 26, 2012, under the laws of Ireland and re-registered as an Irish public limited company on October 25, 2012. The Company's ordinary shares began trading on The Nasdaq Global Market under the symbol “PRTA” on December 21, 2012, and currently trade on The Nasdaq Global Select Market.
Liquidity and Business Risks
As of September 30, 2024, the Company had an accumulated deficit of $1.0 billion and cash and cash equivalents of $519.3 million.
Based on the Company's business plans, management believes that the Company’s cash and cash equivalents at September 30, 2024, are sufficient to meet its obligations for at least the next twelve months. To operate beyond such period, or if the Company elects to increase its spending on research and development programs significantly above current long-term plans or enters into potential licenses and/or other acquisitions of complementary technologies, products or companies, the Company may need additional capital. The Company expects to continue to finance future cash needs that exceed its cash from operating activities primarily through its current cash and cash equivalents, payments pursuant to its agreements with Roche, BMS, and Novo Nordisk, and, to the extent necessary, through proceeds from public or private equity or debt financings, loans and other collaborative agreements with corporate partners or other arrangements.
- Summary of Significant Accounting Policies
Basis of Preparation and Presentation of Financial Information
These accompanying Condensed Consolidated Financial Statements have been prepared in accordance with the accounting principles generally accepted in the U.S. (“GAAP”) and with the instructions for Form 10-Q and Regulation S-X statements. Accordingly, they do not include all of the information and notes required for complete financial statements. These Condensed Consolidated Financial Statements should be read in conjunction with the Consolidated Financial Statements and Notes thereto contained in the Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (the “SEC”) on February 22, 2024 (the “2023 Form 10-K”). These unaudited interim Condensed Consolidated Financial Statements are presented in U.S. dollars, which is the functional currency of the Company and its consolidated subsidiaries. These Condensed Consolidated Financial Statements include the accounts of the Company and its consolidated subsidiaries. All intercompany balances and transactions have been eliminated in consolidation.
Unaudited Interim Financial Information
The accompanying Condensed Consolidated Financial Statements and related disclosures are unaudited, have been prepared on the same basis as the annual consolidated financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair presentation of the results of operations for the periods presented. The year-end condensed consolidated balance sheet data was derived from audited financial statements, however certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted. The condensed consolidated results of operations for any interim period are not necessarily indicative of the results to be expected for the full year or for any other future year or interim period.
Use of Estimates
The preparation of the Condensed Consolidated Financial Statements in conformity with U.S. GAAP requires the Company to make judgments, estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses, and related disclosures. On an ongoing basis, management evaluates its estimates, including critical accounting policies or estimates relatedto revenue recognition and research and development expenses. The Company bases its estimates on historical experience and on various other market specific and other relevant assumptions that management believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Because of the uncertainties inherent in such estimates, actual results may differ materially from these estimates.
Significant Accounting Policies
There were no significant changes to the accounting policies during the nine months ended September 30, 2024, from the significant accounting policies described in Note 2 of the Notes to Consolidated Financial Statements in the 2023 Form 10-K.
Concentration of Risks and Other Risks and Uncertainties
Financial instruments that potentially subject the Company to concentration of credit risk consist of cash and cash equivalents. The Company places its cash equivalents with high credit quality financial institutions and, by policy, limits the amount of credit exposure with any one financial institution. Deposits held with banks have exceeded, and will continue to exceed, federally insured limits. The Company is exposed to credit risk in the event of a default by the financial institutions holding its cash and cash equivalents. The Company has not experienced any losses on its deposits of cash and cash equivalents and its credit risk exposure is up to the extent recorded on the Company's Condensed Consolidated Balance Sheet.
The Company’s business is primarily conducted in U.S. dollars except for its agreements with contract manufacturers for drug supplies which are primarily denominated in Euros. The Company recorded a loss on foreign currency exchange rate differences of approximately$194,000 and$253,000 during the nine months ended September 30, 2024, and 2023, respectively. If the Company increases its business activities that require the use of foreign currencies, it may be exposed to losses if the Euro and other such currencies continue to strengthen against the U.S. dollar.
As of September 30, 2024, and December 31, 2023, approximately$3.3 million and$3.8 million, respectively, of the Company’s property and equipment, net was held in the U.S. anda nominal amount was in Ireland.
The Company is subject to a number of risks similar to other late-stage clinical biotechnology companies, including, but not limited to, the need to obtain adequate additional funding, possible failure of current or future preclinical testing or clinical trials, its reliance on third parties to conduct its clinical trials, the need to obtain regulatory and marketing approvals for its drug candidates, competitors developing new technological innovations, the need to successfully commercialize and gain market acceptance of the Company’s drug candidates, its right to develop and commercialize its drug candidates pursuant to the terms and conditions of the licenses granted to the Company, protection of proprietary technology, and the need to secure and maintain adequate clinical trial management, manufacturing, packaging, labeling, storage, testing, and distribution arrangements with third parties. The Company also relies on third-party consultants to assist in managing these third parties and assist with its clinical trial operations and manufacturing. If the Company does not successfully commercialize or partner any of its drug candidates, it will be unable to generate product revenue or achieve profitability. Further, the Company is also subject to broad market risks and uncertainties resulting from recent events, such as inflation, rising interest rates, and recession risks, as well as supply chain and labor shortages.
Segment
The Company operates inone segment. The Company’s chief operating decision maker (the “CODM”), its chief executive officer, manages the Company’s operations and evaluates the Company’s financial performance on a consolidated basis for purposes of allocating resources.
Recent Accounting Pronouncements
On March 6, 2024, the SEC issued final rule, “The Enhancement and Standardization of Climate-Related Disclosures for Investors”, which requires registrants to disclose material climate-related risks, including descriptions of board oversight and risk management activities, the material impacts of these risks on a registrants strategy, business model and outlook and any material climate-related targets or goals. The rule requires these climate-related information to be disclosed in registration statements and annual reports. Registrants will also need to quantify certain effects of severe weather events and other natural conditions in a note to their audited financial statements. In addition, accelerated and large accelerated filers will need to disclose Scope 1 and Scope 2 greenhouse gas (GHG) emissions, if material, which will be subject to third-party assurance. The Company will be required to comply with the rule in fiscal year beginning January 1, 2025 for all disclosures other than the compliance with quantitative and qualitative disclosure requirements of material expenditures and material impacts on financial estimates that directly result from (1) activities to mitigate or adapt to the climate-related risks, (2) targets or goals and (3) transition plans will be required beginning fiscal year 2026. The Company’s other compliance dates are the following: 1) Scope 1 and Scope 2 GHG emissions - fiscal year beginning January 1, 2026; Limited assurance - fiscal year beginning January 1, 2029; Reasonable assurance - fiscal year beginning January 1, 2033; and Electronic tagging - fiscal year beginning January 1, 2026. The Company is currently evaluating the impact of the new standard on its consolidated financial statements and related disclosures. On April 4, 2024, the Securities and Exchange Commission (SEC) voluntarily stayed implementation of its recently adopted Climate Disclosure Rules.
On November 27, 2023, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update 2023-07 ("ASU 2023-07"), Segment Reporting- Improvements to Reportable Segment Disclosures, which requires public entities to provide disclosures on significant segment expenses that are regularly provided to the CODM and included within each reported measure of segment profit or loss and other segment items on an annual and interim basis. The guidance also requires public entities to provide all disclosures about reportable segment’s profit or loss and assets in interim periods that are currently required annually. Public entities with a single reportable segment have to provide all disclosures required by Accounting Standards Codification (“ASC”) 280, Segment Reporting including the significant segment expense disclosures. The guidance is applied retrospectively to all periods presented in financial statements and is effective for fiscal years beginning after December 15, 2023, and for interim periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of adopting the update on the Company's disclosures.
In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures, which requires public business entities to disclose a tabular reconciliation using both percentages and amounts, broken out into specific categories with certain reconciling items at or above 5% of the expected tax further broken out by nature and/or jurisdiction. The guidance also requires all entities to disclose income taxes paid, net of refunds, disaggregated by federal (national), state and foreign taxes for annual periods and to disaggregate the information by jurisdiction based on a quantitative threshold. All entities are required to apply the guidance prospectively, with the option to apply it retrospectively. The guidance will be effective for the Company’s annual period ending December 31, 2025. Early adoption is permitted. The Company is currently evaluating the impact of the new standard on its income tax disclosures.
- Fair Value Measurements
The Company measures certain financial assets and liabilities at fair value on a recurring basis, including cash equivalents. Fair value is an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or a liability. A three-tier fair value hierarchy is established as a basis for considering such assumptions and for inputs used in the valuation methodologies in measuring fair value:
Level 1 — inputs are quoted prices (unadjusted) for identical assets or liabilities in active markets.
Level 2 — inputs are other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly.
Level 3 — inputs are unobservable inputs that are supported by little or no market activities, which would require the Company to develop its own assumptions.
The fair value hierarchy also requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. The carrying amounts of certain financial instruments, such as cash equivalents, prepaid expenses and other current assets, accounts receivable, accounts payable and accrued liabilities, approximate fair value due to their short-term nature.
Based on the fair value hierarchy, the Company classifies its cash equivalents within Level 1. This is because the Company values its cash equivalents using quoted market prices. The Company’s Level 1 securities consisted of $499.2 million and $589.9 million in money market funds included in cash and cash equivalents at September 30, 2024, and December 31, 2023, respectively.
- Composition of Certain Balance Sheet Items
Prepaid Expenses and Other Current Assets
Prepaid and other current assets consisted of the following (in thousands):
| September 30, | December 31, | |
|---|---|---|
| 2024 | 2023 | |
| Prepaid R&D expenses | $13,120 | $10,998 |
| Prepaid G&A expenses | 1,537 | 803 |
| Other | 1,065 | 2,140 |
| Prepaid expenses and other current assets | $15,722 | $13,941 |
Property and Equipment, net
Property and equipment, net consisted of the following (in thousands):
| September 30, | December 31, | |
|---|---|---|
| 2024 | 2023 | |
| Machinery and equipment | $9,137 | $9,019 |
| Purchased computer software | 2,204 | 2,232 |
| 11,341 | 11,251 | |
| Less: accumulated depreciation and amortization | (8,087) | (7,415) |
| Property and equipment, net | $3,254 | $3,836 |
Depreciation expense was $0.2 million and $0.7 million for the three and nine months ended September 30, 2024, respectively, compared to $0.2 million and $0.7 million for the three and nine months ended September 30, 2023, respectively.
Other Current Liabilities
Other current liabilities consisted of the following (in thousands):
| September 30, | December 31, | |
|---|---|---|
| 2024 | 2023 | |
| Payroll and related expenses | $11,869 | $13,245 |
| Professional services | 999 | 288 |
| Other | 1,439 | 2,129 |
| Other current liabilities | $14,307 | $15,662 |
- Net Income (Loss) Per Ordinary Share
Net income (loss) per ordinary share was determined as follows (in thousands, except per share amounts):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Numerator: | ||||
| Net income (loss) | $(59,001) | $21,907 | $(64,354) | $(79,552) |
| Denominator: | ||||
| Weighted-average ordinary shares outstanding used in per share calculations - basic | 53,790 | 53,559 | 53,757 | 53,064 |
| Weighted-average ordinary shares outstanding used in per share calculations - diluted | 53,790 | 58,004 | 53,757 | 53,064 |
| Net income (loss) per share: | ||||
| Basic net income (loss) per ordinary share | $(1.10) | $0.41 | $(1.20) | $(1.50) |
| Diluted net income (loss) per ordinary share | $(1.10) | $0.38 | $(1.20) | $(1.50) |
Potentially issuable ordinary shares were not used in computing diluted net loss per ordinary share as their effect would be anti-dilutive due to the loss recorded during the three and nine months ended September 30, 2024 and nine months ended September 30, 2023, and therefore diluted net loss per share is equal to basic net loss per share.
The equivalent ordinary shares not included in diluted net income (loss) per share because their effect would be anti-dilutive are as follows (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Stock options to purchase ordinary shares | 11,297 | 2,110 | 11,297 | 9,939 |
| Restricted Stock Units (RSU) | 24 | 27 | 24 | 27 |
| Total | 11,321 | 2,137 | 11,321 | 9,966 |
- Commitments and Contingencies
Lease Commitments
As of September 30, 2024, the Company currently has four leases relating to its facilities in the United States and Dublin, Ireland.
South San Francisco Facility
The Company had a noncancelable operating sublease (the “SSF Lease”) covering 128,751 square feet of office and laboratory space in South San Francisco, California, U.S. (the “SSF Facility”), which expired on December 31, 2023.
Total operating lease cost wasnil andnil for the three and nine months ended September 30, 2024, respectively, and $1.6 million and $4.8 million for the three and nine months ended September 30, 2023, respectively. Total cash paid against the operating lease liability wasnil andnil for the three and nine months ended September 30, 2024, respectively, and $1.6 million and $4.9 million for the three and nine months ended September 30, 2023, respectively. The Company obtained a standby letter of credit which could be drawn down by the sublandlord in the event the Company failed to fully and faithfully perform all of its obligations under the SSF Lease and to compensate the sublandlord for all losses and damages the sublandlord may have suffered as a result of the occurrence of any default on the part of Company not cured within the applicable cure period. This standby letter of credit was collateralized by a certificate of deposit of the same amount which was classified as restricted cash as of December 31, 2023. The remaining standby letter of credit amount of $1.4 million was released to the Company in May 2024.
Sub-Sublease of South San Francisco Facility
The Company had a Sub-Sublease Agreement (the “Sub-Sublease”) with Assembly Biosciences, Inc. covering approximately 46,641 square feet of office and laboratory space of the SSF Facility. The Sub-Sublease expired on December 15, 2023, in connection with the expiration of the SSF Lease. The Sub-Sublease was considered an operating lease under ASC 842. For thethree and nine months ended September 30, 2024, the Company recordednil andnil, respectively, and$0.7 million and $2.2 million, for the three and nine months ended September 30, 2023, respectively, of sub-lease rental income as an offset to its operating expenses.
Dublin
In June 2021, the Company entered into a lease agreement for office space in Dublin, Ireland, which commenced in August 2021 and had an initial term ofone year. In addition, the Company entered into a lease agreement for additional office space in Dublin, Ireland, which commenced in August 2023 and had an initial term ofone year. Both leases have an automatic renewal clause, pursuant to which each agreement will be extended automatically for successive periods equal to their current terms, unless each agreement is cancelled by the Company. In April 2024, the Company renewed both leases, each for anotherone year term with termination dates in July 2025.
Brisbane Facility
On October 28, 2022, the Company entered into a noncancelable operating sublease (the "Brisbane Sublease") to sublease approximately 31,157 square feet of office and laboratory space located in Brisbane, California (the “Brisbane Facility”) with Arcus Biosciences, Inc., (the "Sublandlord"). The Brisbane Sublease became effective on October 28,2022. The Brisbane Sublease provides that the Company's obligation to pay rent commenced on July 1, 2023, which is subject to abatement for the first six months following such date, with the exception of the seventh rent payment that was due upon execution of the Brisbane Sublease. The Company is obligated to make lease payments totaling approximately $14.9 million over the lease term, which expires on September 30, 2028, unless terminated earlier. The Brisbane Sublease further provides that the Company is obligated to pay the Sublandlord certain costs, including taxes and operating expenses. The Company has the option to extend the sublease by providing written notice at least nine months prior to the expiration of the sublease term. As of September 30, 2024, the Brisbane Sublease has a remaining lease term of 4.0 years.
The Brisbane Sublease is considered an operating lease and the accounting lease commencement date was on July 31, 2023 when the Company gained control over the Brisbane Facility. The Company recorded a right-of-use asset of approximately $11.4 million and lease liability of approximately $3.6 million relating to the Brisbane Sublease on the lease commencement date. The discount rate used to determine the lease liability was 5.76%. The initial measurement of the right-of-use asset for the Brisbane Sublease includes the tenant improvement added by the Company wherein the lessor was deemed the accounting owner.
The Company was entitled to an improvement allowance of up to$9.3 million, to be used for costs incurred by the Company to construct certain improvements to the Brisbane Facility and to prepare for the Company's occupancy of the Brisbane Facility. As of September 30, 2024, all of the $9.3 million improvement allowance has been received from the Sublandlord and the Company is obligated to fund construction costs incurred in excess of the improvement allowance.
Total operating lease cost for the Brisbane Sublease was$0.8 million and $2.4 million for thethree and nine months ended September 30, 2024, respectively, and$0.5 million and $0.5 millionfor three and nine months ended September 30, 2023, respectively. Total cash paid against the operating lease liability was $0.7 million and $2.0 million for thethree and nine months ended September 30, 2024, respectively, and$0.2 million and $0.2 millionfor three and nine months ended September 30, 2023, respectively.
In conjunction with the Brisbane Sublease, the Company obtained a standby letter of credit in the initial amount of $0.9 million, which may be drawn down by the Sublandlord in the event the Company fails to fully and faithfully perform all of its obligations under the Brisbane Sublease and to compensate the Sublandlord for all losses and damages the Sublandlord may suffer as a result of the occurrence of any default on the part of the Company not cured within the applicable cure period. As of September 30, 2024, none of the standby letter of credit amount of $0.9 million has been used.
The following table sets out a maturity analysis of payments under the Company’s operating leases, including a reconciliation to the lease liabilities recognized in the Condensed Consolidated Balance Sheets as of September 30, 2024 (in thousands):
| Year Ended December 31, | Operating Leases |
|---|---|
| 2024 (3 months) | 808 |
| 2025 | 3,188 |
| 2026 | 3,158 |
| 2027 | 3,269 |
| 2028 | 2,523 |
| Thereafter | — |
| Total | $12,946 |
| Less: Present value adjustment | (1,452) |
| Lease liability | $11,494 |
Indemnity Obligations
The Company has entered into indemnification agreements with its current and former directors and officers and certain key employees. These agreements contain provisions that may require the Company, among other things, to indemnify such persons against certain liabilities that may arise because of their status or service and advance their expenses incurred as a result of any indemnifiable proceedings brought against them. The obligations of the Company pursuant to the indemnification agreements continue during such time as the indemnified person serves the Company and continues thereafter until such time as a claim can be brought. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is unlimited; however, the Company has a director and officer liability insurance policy that limits its exposure and enables the Company to recover a portion of any future amounts paid. As a result of its insurance policy coverage, the Company believes the estimated fair value of these indemnification agreements is minimal. Accordingly, the Company had no liabilities recorded for these agreements as of September 30, 2024, and December 31, 2023.
Other Commitments
In the normal course of business, the Company enters into various firm purchase commitments primarily related to research and development activities. As of September 30, 2024, the Company had non-cancelable purchase commitments to suppliers for $8.0 million of which $2.4 million is included in current liabilities, and contractual obligations under license agreements of $0.3 million of whichnil is included in current liabilities. The following is a summary of the Company's non-cancelable purchase commitments and contractual obligations as of September 30, 2024 (in thousands):
| Total | 2024 | 2025 | 2026 | 2027 | 2028 | Thereafter | |
|---|---|---|---|---|---|---|---|
| Purchase Obligations (1) | $8,036 | $7,664 | $269 | $103 | $— | $— | $— |
| Contractual obligations under license agreements | 323 | 49 | 64 | 60 | 60 | 45 | 45 |
| Total | $8,359 | $7,713 | $333 | $163 | $60 | $45 | $45 |
(1) Purchase obligations consist of non-cancelable purchase commitments to suppliers and contract research organizations.
- Significant Agreements
Roche License Agreement
In December 2013, the Company through its wholly owned subsidiary Prothena Biosciences Limited and Prothena Biosciences Inc entered into a License, Development, and Commercialization Agreement (the “License Agreement”) with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. (together, “Roche”) to develop and commercialize certain antibodies that target α-synuclein, including prasinezumab, which are referred to collectively as “Licensed Products.” Upon the effectiveness of the License Agreement in January 2014, the Company granted to Roche an exclusive, worldwide license to develop, make, have made, use, sell, offer to sell, import and export the Licensed Products. The Company retained certain rights to conduct development of the Licensed Products and an option to co-promote prasinezumabin the U.S. During the term of the License Agreement, the Company and Roche will work exclusively with each other to research and develop antibody products targeting alpha-synuclein (or α-synuclein) potentially including incorporation of Roche’s proprietary Brain Shuttle™ technology to potentially increase delivery of therapeutic antibodies to the brain. The License Agreement provided for Roche making an upfront payment to the Company of $30.0 million, which was received in February 2014; making a clinical milestone payment of $15.0 million upon initiation of the Phase 1 clinical trial for prasinezumab, which was received in May 2014; making a clinical milestone payment of $30.0 million upon dosing of the first patient in the Phase 2 clinical trial for prasinezumab, which was achieved in June 2017; and making a clinical milestone payment of $60.0 million upon dosing of the first patient in the global Phase 2b PADOVA study for prasinezumab, which was achieved in May 2021.
For prasinezumab, Roche is obligated to pay:
up to $290.0 million upon the achievement of development, regulatory, and various first commercial sales milestones;
up to $155.0 million upon achievement of U.S. commercial sales milestones;
up to $175.0 million upon achievement of ex-U.S. commercial sales milestones; and
tiered, high single-digit to high double-digit royalties in the teens based on U.S. and ex-U.S. annual net sales, subject to certain adjustments, with respect to the applicable Licensed Product.
Roche bore 100% of the cost of conducting the research collaboration under the License Agreement during the research term, which expired December 31, 2017. In May 2021, the Company exercised its rights under the terms of License Agreement to receive potential U.S. commercial sales milestone and royalties, in lieu of a U.S. profit and loss share for prasinezumab in Parkinson’s disease. Thus, in the U.S., through May 28, 2021, the parties shared all development costs, all of which were allocated 70% to Roche and 30% to the Company, for prasinezumab in the Parkinson’s disease indication. If the Company opts in to participate in co-development and co-funding for any other Licensed Products and/or indications, the parties will share all development and commercialization costs, as well as profits, all of which will be allocated 70% to Roche and 30% to the Company.
The Company initiated a Phase 1 clinical trial for prasinezumab in 2014. Following the Phase 1 clinical trial, Roche became primarily responsible for developing, obtaining and maintaining regulatory approval for and commercializing Licensed Products. Roche also became responsible for the clinical and commercial manufacture and supply of Licensed Products.
In addition, the Company has an option under the License Agreement to co-promote prasinezumab in the U.S. in the Parkinson’s disease indication. If the Company exercises such option, it may also elect to co-promote additional Licensed Products in the U.S. approved for Parkinson’s disease. Outside the U.S., Roche will have responsibility for developing and commercializing the Licensed Products. Roche bears all costs that are specifically related to obtaining or maintaining regulatory approval outside the U.S. and will pay the Company a variable royalty based on annual net sales of the Licensed Products outside the U.S.
The License Agreement continues on a country-by-country basis until the expiration of all payment obligations under the License Agreement. The License Agreement may also be terminated (i) by Roche at will after the first anniversary of the effective date of the License Agreement, either in its entirety or on a Licensed Product-by-Licensed Product basis, upon 90 days’ prior written notice to the Company prior to first commercial sale and 180 days’ prior written notice to Prothena after first commercial sale, (ii) by either party, either in its entirety or on a Licensed Product-by-Licensed Product or region-by-region basis, upon written notice in connection with a material breach uncured 90 days after initial written notice, and (iii) by either party, in its entirety, upon insolvency of the other party. The License Agreement may be terminated by either party on a patent-by-patent and country-by-country basis if the other party challenges a given patent in a given country. The Company’s rights to co-develop Licensed Products under the License Agreement will terminate if the Company commences certain studies for certain types of competitive products. The Company’s rights to co-promote Licensed Products under the License Agreement will terminate if the Company commences a Phase 3 study for such competitive products.
The License Agreement cannot be assigned by either party without the prior written consent of the other party, except to an affiliate of such party or in the event of a merger or acquisition of such party, subject to certain conditions. The License Agreement also includes customary provisions regarding, among other things, confidentiality, intellectual property ownership, patent prosecution, enforcement and defense, representations and warranties, indemnification, insurance, and arbitration and dispute resolution.
Performance Obligations
As of September 30, 2024, and December 31, 2023, there wereno remaining performance obligations under the License Agreement since the obligations related to research and development activities were only for the Phase 1 clinical trial and the remaining obligations were delivered or performed.
Milestone Accounting
Under the License Agreement, the Company is eligible to receive certain milestone payments upon the achievement of development, regulatory and various first commercial sales milestones. Milestone payments are evaluated under ASC Topic 606. Factors considered in this determination included scientific and regulatory risk that must be overcome to achieve each milestone, the level of effort and investment required to achieve the milestone, and the monetary value attributed to the milestone. Accordingly, the Company estimates payments in the transaction price based on the most likely approach, which considers the single most likely amount in a range of possible amounts related to the achievement of these milestones. Additionally, milestone payments are included in the transaction price only when the Company can conclude it is probable that a significant revenue reversal will not occur in future periods when the milestone is achieved.
The Company excludes the milestone payments and royalties in the initial transaction price calculation because such payments are considered to be variable considerations with constraint. Such milestone payments and royalties will be recognized as revenue once the Company can conclude it is probable that a significant revenue reversal will not occur in future periods.
The clinical and regulatory milestones under the License Agreement after the point at which the Company could opt out are considered to be variable considerations with constraint due to the fact that active participation in the development activities that generate the milestones is not required under the License Agreement, and the Company can opt out of these activities. There are no refunds or claw-back provisions and the milestones are uncertain of occurrence even after the Company has opted out. Based on this determination, these milestones will be recognized when the Company can conclude it is probable that a significant revenue reversal will not occur in future periods.
Collaboration Agreement with Bristol Myers Squibb
On March 20, 2018, the Company, through its wholly owned subsidiary Prothena Biosciences Limited (“PBL”), entered into a Master Collaboration Agreement (the “Collaboration Agreement”) with Celgene Switzerland LLC (“Celgene”), a subsidiary of Celgene Corporation (which was acquired by Bristol Myers Squibb (“BMS”) in November 2019), pursuant to which Prothena granted to Celgene a right to elect in its sole discretion to exclusively license rights both in the U.S. (the “US Rights”) and on a global basis (the “Global Rights”), with respect to the Company’s programs to develop and commercialize antibodies targeting tau, TDP-43 and an undisclosed target (the “Collaboration Targets”).
The Collaboration Agreement provided for Celgene making an upfront payment to the Company of $100.0 million, plus future potential license exercise payments and regulatory and commercial milestones for each program under the Collaboration Agreement, as well as royalties on net sales of any resulting marketed products. In connection with the Collaboration Agreement, the Company and Celgene entered into a Share Subscription Agreement on March 20, 2018, under which Celgene subscribed to 1,174,536 of the Company’s ordinary shares for a price of $42.57 per share, for a total of approximately $50.0 million.
BMS US and Global Rights and Licenses
On a program-by-program basis, beginning on the effective date of the Collaboration Agreement and ending on the date that the IND Option term expires for such program (which generally occurs sixty days after the date on which the Company delivers to BMS the first complete data package for an IND that was filed for a lead candidate from the relevant program), BMS may elect in its sole discretion to exercise its US Rights to receive an exclusive license to develop, manufacture and commercialize antibodies targeting the applicable Collaboration Target in the U.S. (the “US License”). If BMS exercises its US Rights for a collaboration program, it is obligated to pay the Company an exercise fee of approximately $80.0 million per program. Thereafter, following the first to occur of (a) completion by the Company, in its discretion and at its cost, of Phase 1 clinical trials for such program or (b) BMS’ election to assume responsibility to complete such Phase 1 clinical trials (at its cost), BMS would have the sole right to develop, manufacture and commercialize antibody products targeting the relevant Collaboration Target for such program (the “Collaboration Products”) in the U.S.
On a program-by-program basis, following completion of a Phase 1 clinical trial for a collaboration program for which BMS has previously exercised its US Rights, BMS may elect in its sole discretion to exercise its Global Rights with respect to such collaboration program to receive a worldwide, exclusive license to develop, manufacture and commercialize antibodies targeting the applicable Collaboration Target (the “Global License”). If BMS exercises its Global Rights, BMS would be obligated to pay the Company an additional exercise fee of $55.0 million for such collaboration program. The Global Rights would then replace the US Rights for that collaboration program, and BMS would have decision making authority over developing, obtaining and maintaining regulatory approval for, manufacturing and commercializing the Collaboration Products worldwide.
After BMS’ exercise of Global Rights for a collaboration program, the Company is eligible to receive up to $562.5 million in regulatory and commercial milestones per program. Following an exercise by BMS of either US Rights or Global Rights for such collaboration program, the Company will also be eligible to receive tiered royalties on net sales of Collaboration Products ranging from high single digit to high teen percentages, on a weighted average basis depending on the achievement of certain net sales thresholds. Such exercise fees, milestones and royalty payments are subject to certain reductions as specified in the Collaboration Agreement, the agreement for US Rights and the agreement for Global Rights.
BMS will continue to pay royalties on a Collaboration Product-by-Collaboration Product and country-by-country basis, until the latest of (i) expiration of certain patents covering the Collaboration Product, (ii) expiration of all regulatory exclusivity for the Collaboration Product, and (iii) an agreed period of time after the first commercial sale of the Collaboration Product in the applicable country (the “Royalty Term”).
Term
The term of the Collaboration Agreement expired on May 24, 2024.
The term of any US License or Global License would continue on a Licensed Product-by-Licensed Product and country-by-country basis until the expiration of all Royalty Terms under such agreement.
Performance Obligations
The Company assessed the Collaboration Agreement and concluded that it represented a contract with a customer within the scope of ASC 606. Per ASC 606, a performance obligation is defined as a promise to transfer a good or service or a series of distinct goods or services. At inception of the Collaboration Agreement, the Company is not obligated to transfer any US License or Global License to BMS unless BMS exercises its US Rights or Global Rights, respectively, and the Company is not obligated to perform development activities under the development plan during preclinical and Phase 1 clinical trials including the regulatory filing of the IND.
The discovery, preclinical and clinical development activities performed by the Company are to be performed at the Company’s discretion and are not promised goods or services and therefore are not considered performance obligations under ASC 606, unless and until the Company agrees to perform the Phase 1 clinical trials (after the IND option exercise) that are determined to be performance obligations at the time the option is exercised. Per the terms of the Collaboration Agreement, the Company may conduct discovery activities to characterize, identify and generate antibodies to become collaboration candidates that target such Collaboration Target, and thereafter may pre-clinically develop collaboration candidates to identify lead candidates that target such Collaboration Target and file an IND with the U.S. Food and Drug Administration (the “FDA”) for a Phase 1 clinical trial for such lead candidates. In the event the Company agrees to be involved in a Phase 1 clinical trial, the Company will further evaluate whether any such promise represents a performance obligation at the time the option is exercised. If it is concluded that the Company has obligated itself to an additional performance obligation besides the license granted at IND option exercise, then the effects of the changes in the arrangement will be evaluated under the modification guidance of ASC 606.
The Company is not obligated to perform manufacturing activities. Per the terms of the Collaboration Agreement, to the extent that the Company, at its discretion, conducts a program, the Company shall be responsible for the manufacture of collaboration candidates and collaboration products for use in such program, as well as the associated costs. Delivery of manufactured compound (clinical product supply) is not deemed a performance obligation under ASC 606 as the Company is not obligated to transfer supply of collaboration product to BMS unless BMS exercises its right to participate in the Phase 1 development.
Compensation for the Company’s provision of inventory supply, to the extent requested by BMS would be paid to the Company by BMS at a reasonable stand-alone selling price for such supply. Given that (i) there is substantial uncertainty about the development of the programs, (ii) the pricing for the inventory is at its standalone selling price and (iii) the manufacturing services require the entity to transfer additional goods or services that are incremental to the goods and services provided prior to the resolution of the contingency, the Company’s supply of product is not a material right. Therefore, the inventory supply is not considered a performance obligation unless and until, requested by BMS.
In addition to the grant of the US License after BMS exercises its US Rights for a program, BMS would be entitled to receive certain ancillary development services from the Company, such as technology transfer assistance, regulatory support, safety data reporting activities and transition supply, if requested by BMS.
In addition to the grant of the Global License after BMS exercises the Global Rights for a program, BMS is entitled to receive certain ancillary development services from the Company, such as ongoing clinical trial support upon request by BMS, transition supply, if requested by BMS, and regulatory support for coordination of pharmacovigilance matters.
The Company evaluated the potential obligations to transfer the US Licenses and Global Licenses and performance of the ancillary development services subsequent to exercise of the US Rights and Global Rights, if the options are exercised by BMS, under ASC 606-10-55-42 and 55-43 to determine whether the US Rights or the Global Rights provided BMS a “material right” and concluded that BMS’ options to exercise its US Rights and Global Rights represented “material rights” to BMS that it would not have received without entering into the Collaboration Agreement.
At inception of the Collaboration Agreement, there were a total of six options, including US Rights and Global Rights to acquire a US License and a Global License, respectively, and rights to request certain development services (following exercise of the US Rights and Global Rights, respectively) for each of the three programs. None of which were remaining as of May 24, 2024. The deferred revenue balance as of September 30, 2024 of$14.4 million is related to the outstanding PRX019 Phase 1 Clinical Trial Obligation (“PRX019 Phase 1 Clinical Trial Obligation”). Per ASC 606, the US Rights and Global Rights are material rights and therefore are performance obligations. The goods and services underlying the options are not accounted for as separate performance obligations, but rather become performance obligations, if and when, an option is exercised.
US License Agreement for the Tau/BMS-986446 Collaboration Target
BMS exercised its US Rights for the tau/BMS-986446 (formerly PRX005) Collaboration Target and on July 30, 2021, PBL entered into a U.S. License Agreement granting BMS an exclusive license to develop, manufacture and commercialize tau Collaboration Products in the United States targeting tau (the “Tau US License Agreement”). The Company received an associated option exercise fee of $80.0 million.
The Tau US License Agreement included the following distinct performance obligations: (1) the delivery of the US License for tau/BMS-986446 Collaboration Target (“Tau US License Obligation”); and (2) the Company’s obligation to provide development activities under the development plan during any Phase 1 clinical trials (the “Tau US Development Services Obligation”). Revenue allocated to the Tau US License Obligation was recognized when the Company satisfied its obligation at a point in time, while the revenue allocated to the Tau US Development Services Obligation was recognized over time usingan input-based model. All performance obligations have been delivered.
Global License Agreement for the Tau/BMS-986446 Collaboration Target
Subsequently, BMS exercised its Global Rights for the tau/BMS-986446 Collaboration Target and on July 5, 2023, PBL entered into a Global License Agreement granting BMS an exclusive license to develop, manufacture and commercialize tau Collaboration Products globally for any and all uses or purposes with respect to any human or animal disease, disorder or condition (the “Tau Global License Agreement”). The Tau Global License Agreement supersedes and replaces the Tau US License Agreement in its entirety. The Company received an associated option exercise fee of $55.0 million in August 2023 and it is eligible to receive regulatory and sales milestones up to $562.5 million upon achievement of certain events, including regulatory approval of a tau Collaboration Product, and on BMS achieving certain annual, worldwide net sales thresholds. The Company also is eligible to receive tiered royalties on net sales of tau Collaboration Products, ranging from high single digit to high teen percentages, on a weighted average basis depending on the achievement of certain net sales thresholds.
The Company’s distinct performance obligation under the Tau Global License Agreement was limited to the delivery of the Global License for tau/BMS-986446 Collaboration Target (“Tau Global License Obligation”). Revenue allocated to the Tau Global License Obligation was recognized by the Company at the time that the license was delivered in July 2023.
Global License Agreement for the undisclosed/PRX019 Collaboration Target
On May 24, 2024, PBL entered into a Global License Agreement granting BMS an exclusive license to develop, manufacture and commercialize Collaboration Products targeting an undisclosed target (including PRX019) globally for any and all uses or purposes with respect to any human or animal disease, disorder or condition (the “PRX019 Global License Agreement”). The Company received an associated option exercise fee of $80.0 million in June 2024 and is eligible to receive further development and regulatory milestones of up to $242.5 million upon achievement of certain development and regulatory milestones, including regulatory approval, of a Collaboration Product, and up to $375.0 million upon BMS achieving certain annual, worldwide net sales thresholds. The Company also is eligible to receive tiered royalties on annual, worldwide net sales of Collaboration Products, ranging from high single digit to high teen percentages, on a weighted average basis depending on the achievement of certain net sales thresholds. Such milestones and royalty payments (i) could be reduced in the case where BMS is successful in developing a modified version of PRX019 that achieves certain specified improved metrics, and (ii) are subject to certain reductions as specified in the PRX019 Global License Agreement.
The PRX019 Global License Agreement included the following distinct performance obligations: (1) the delivery of the Global License for the undisclosed Collaboration Target (“PRX019 Global License Obligation”); and (2) the Company’s obligation to run a Phase 1 clinical trial for PRX019. Pursuant to the terms of the PRX019 Global License Agreement, BMS may elect to assume responsibility for completing such Phase 1 clinical trial (at its cost). Revenue allocated to the PRX019 Global License Obligation was recognized when the Company satisfied its obligation at a point in time, while the revenue allocated to the PRX019 Phase 1 Clinical Trial Obligation is recognized over time usingan input-based model.
Transaction Price
At inception of the Collaboration Agreement, the Company did not transfer any goods or services to BMS that were material. Accordingly, the Company concluded that the initial transaction price would be recognized as a contract liability and would be deferred until the Company transfers control of goods or services to BMS (which would be when BMS exercises the US Right or Global Right and receives control of the US License or Global License for at least one of the programs), or when the IND Option term expires if BMS had not yet exercised the US Right, or when the Phase 1 Option term expires if BMS had not yet exercised the Global Right, or at the termination of the Collaboration Agreement, whichever occurs first. At such point that the Company transfers control of goods or services to BMS, or when the option expires, the Company would recognize revenue as a continuation of the original contract. Under this approach, the Company would treat the consideration allocated to the material right as an addition to the consideration for the goods or services underlying the contract option.
At inception of the Collaboration Agreement, the Company estimated the standalone selling price for each performance obligation (i.e., the US Rights and Global Rights by program). The estimate of standalone selling price for the US Rights and Global Rights by program was based on the adjusted market assessment approach using a discounted cash flow model. The key assumptions used in the discounted cash flow model included the market opportunity for commercialization of each program in the U.S. or globally depending on the license, the probability of successfully developing and commercializing a given program target, the estimated remaining development costs for the respective program, the estimated time to commercialization of the drug for that program, and a discount rate.
The initial transaction price under the Collaboration Agreement, pursuant to ASC 606, was $110.2 million, including the $100.0 million upfront payment and $10.2 million premium on the ordinary shares purchased under the SSA. The Company allocated the initial transaction price across the US Rights and Global Rights for each program in a range of approximately $15-$25 million and $10-$18 million, respectively.
The Company did not include the option fees in the initial transaction price because such fees are contingent on the options to the US Rights and the Global Rights being exercised. Upon the exercise of the US Rights and the Global Rights for a program, the Company would have the obligation to deliver the US License and Global License and provide certain ancillary development services if requested by BMS, subsequent to its exercise of the US Rights and Global Rights, respectively, for such program. The Company would include the option fees in the transaction price at the point in time a material right is exercised and the Company transfers control of the goods and services to BMS. In addition, the Company did not include in the initial transaction price certain clinical and regulatory milestone payments since they relate to licenses for which BMS had not yet exercised its option to obtain and these variable considerations are constrained due to the likelihood of a significant revenue reversal.
Upon entering into the Tau Global License Agreement, the Company granted BMS a Global License for the tau/BMS-986446 Collaboration Target, which transferred control of such underlying Global License to BMS. Following execution of the Tau Global License Agreement, BMS paid the Company a $55.0 million option exercise fee. Under the continuation of the original contract method, the Company computed the relative sales price after the Company transferred control of the Global License for tau/BMS-986446. The Company used the original allocated consideration for the Global Right for tau/BMS-986446 of $17.9 million (computed at the inception of the contract) plus the $55.0 million option exercise fee to arrive at the total transaction price of approximately $72.9 million. Given that the Company’s distinct performance obligation under the Tau Global License Agreement was limited to the Tau Global License Obligation no further allocation was required.
Upon entering into the PRX019 Global License Agreement, the Company granted BMS a Global License for the undisclosed/PRX019 Collaboration Target, which transferred control of such underlying Global License to BMS. Following execution of the PRX019 Global License Agreement, BMS paid the Company an $80.0 million option exercise fee. As the original contract contemplated a US and Global payment for $80.0 million and $55.0 million, respectively, and a new payment structure and only one license was agreed to, accordingly, we accounted for the payment under modification accounting. The Company concluded that the modification would be accounted for on a prospective basis as a termination of the existing contract and creation of a new contract. The Company computed the relative sales price for the identified remaining performance obligations consisting of the Global License for PRX019 and the PRX019 Phase 1 Clinical Trial Obligation. The transaction price consisted of the original allocated consideration for the US Right for PRX019 of $24.9 million, and original allocated consideration for the Global Right for PRX019 of $17.4 million (both computed at the inception of the Collaboration Agreement) plus the $80.0 million option exercise fee to arrive at the total transaction price of approximately $122.4 million. This total transaction price was allocated using the relative sales price method between the PRX019 Global License Obligation and the PRX019 Phase 1 Clinical Trial Obligation.
The best estimate of selling price for the Global License for PRX019 was based on a discounted cash flow model. The key assumptions used in the discounted cash flow model used to determine the best estimate of selling price for the license included the market opportunity for commercialization of PRX019, the probability of successfully developing/commercializing PRX019, the remaining development costs for PRX019, and the estimated time to commercialization of PRX019 using a discount rate of 13%. Based on the relative selling price method, the amount that the Company allocated to the performance obligations was as follows: $106.3 million to the license to be recognized concurrent with the delivery of the license; and $16.1 million as development services for the Phase 1 clinical trial to be recognized based on input-based model over the service period.
Significant Payment Terms
The upfront payment of $100.0 million was received in April 2018, while all option fees and milestone payments are due within 30 days after the achievement of the relevant milestone by BMS or receipt by BMS of an invoice for such an amount from the Company.
The Collaboration Agreement does not have a significant financing component since a substantial amount of consideration promised by BMS to the Company is variable and the amount of such variable consideration varies based upon the occurrence or non-occurrence of future events that are not within the control of either BMS or the Company. Variable considerations related to clinical and regulatory milestone payments and option fees are constrained due to the likelihood of a significant revenue reversal.
Revenue and Expense Recognition
For the three and nine months ended September 30, 2024, collaboration revenue from BMS was $1.0 million and $133.0 million, respectively, and for the three and nine months ended September 30, 2023, $84.9 million and $91.0 million, respectively. Collaboration revenue for the three months ended September 30, 2024 was related to PRX019 Phase 1 Clinical Trial Obligation performed while collaboration revenue for the nine months ended September 30, 2024 included recognition of $107.9 million for the transfer of the PRX019 Global License and partial performance of the PRX019 Phase 1 Clinical Trial Obligation. In addition, the material rights for the US Rights and Global Rights for the TDP-43 Collaboration Target of $14.6 million and $10.4 million, respectively, expired unexercised on May 24, 2024 as a result of the expiration of the research term of the Collaboration Agreement. Accordingly, $25.0 million of deferred revenue was recognized as revenue on May 24, 2024. As of September 30, 2024, the aggregate amount of the transaction price allocated to the performance obligations that are unsatisfied was $14.4 million. The Company hadnil and $5.2 million accounts receivable from BMS at September 30, 2024, and December 31, 2023, respectively.
Deferred Revenue
The deferred revenue balance at the beginning of the quarter ended September 30, 2024 was$15.4 million. During the three months ended September 30, 2024, $1.0 million was recognized as collaboration revenue related to the PRX019 Phase 1 Clinical Trial Obligation performed. As of September 30, 2024, the total deferred revenue balance of$14.4 million relates to outstanding performance obligations related to the PRX019 Phase 1 Clinical Trial Obligation of which$8.8 million, and$5.6 million remained in current and non-current deferred revenue, respectively. The deferred revenue balance will be recognized as revenue over the remaining service period.
Milestone and Royalties Accounting
Under the Tau Global License Agreement, the Company is eligible to receive milestone payments of up to $187.5 million upon the achievement of certain specified regulatory milestones and milestone payments of up $375.0 million upon the achievement of certain specified commercial sale milestones. Under the PRX019 Global License Agreement, the Company is eligible to receive milestone payments of up to $242.5 million upon the achievement of certain specified development and regulatory milestones and milestone payments of up $375.0 million upon BMS achieving certain annual, worldwide net sales thresholds. Milestone payments are evaluated under ASC Topic 606. Factors considered in this determination included scientific and regulatory risk that must be overcome to achieve each milestone, the level of effort and investment required to achieve the milestone, and the monetary value attributed to the milestone. Accordingly, the Company estimates payments in the transaction price based on the most likely approach, which considers the single most likely amount in a range of possible amounts related to the achievement of these milestones. Additionally, milestone payments are included in the transaction price only when the Company can conclude it is probable that a significant revenue reversal will not occur in future periods.
The Company excluded the milestone payments and royalties in the initial transaction price because such payments are considered to be variable considerations with constraint. Such milestone payments and royalties will be recognized as revenue at a point in time when the Company can conclude it is probable that a significant revenue reversal will not occur in future periods.
The Company did not achieve any clinical and regulatory milestones under the Collaboration Agreement during thethree and nine months ended September 30, 2024 and 2023.
Novo Nordisk Share Purchase Agreement
On July 8, 2021, the Company together with its wholly owned subsidiary, PBL, entered into a definitive share purchase agreement with Novo Nordisk A/S and Novo Nordisk Region Europe A/S (each an unrelated party). Under the terms of such agreement, Novo Nordisk acquired PBL’s wholly-owned subsidiary, Neotope Neuroscience Limited (“NNL”) and gained full worldwide rights to the intellectual property and related rights to the Company’s ATTR amyloidosis business and pipeline. Upon consummation of the transaction, NNL ceased to be a related party of PBL. The aggregate purchase price consisted of an upfront payment of $60.0 million in cash, subject to customary purchase price adjustments.
Should Novo Nordisk achieve certain stages of development or commercialization for products or product candidates containing coramitug (formerly PRX004)or a derivative thereof in ATTR amyloidosis, PBL is entitled to receive certain milestone payments based on specified development and commercial milestones. The development and commercialization milestone payments will be discounted if the milestone events are achieved with respect to other indications. Should Novo Nordisk achieve specified thresholds of worldwide, annual net sales of the milestone products, regardless of indication, PBL will also be entitled to receive specified one-time net sales milestone payments. All milestone payments attributable to an achieved milestone will be paid to PBL, subject to Novo Nordisk’s offset right for indemnity claims or unpaid amounts in respect of any purchase price adjustment.
The upfront payment of $60.0 million was accounted for as revenue in 2021. In addition to the upfront payment, Novo Nordisk agreed to pay for certain out of pocket expenses under the Transition Services Agreement, which netted to $0.7 million after closing adjustments related to the sale of the ATTR amyloidosis business and pipeline.
Contingent Consideration/Milestone Accounting
In December 2022, the Company received a $40.0 million development milestone payment related to the continued advancement of coramitug in a Phase 2 clinical trial for the treatment of ATTR amyloidosis with cardiomyopathy. This amount was accounted for as revenue from license and intellectual property in 2022.
The Company is eligible to receive additional development and sales milestone payments from Novo Nordisk totaling up to $1.13 billion upon achievement of certain specified development and commercial sales milestones under the share purchase agreement.
The Company excluded the milestone payments in the initial transaction price because such payments are considered to be variable considerations with constraint. Such milestone payments will be recognized as revenue at a point in time when the Company can conclude it is probable that a significant revenue reversal will not occur in future periods.
Revenue Recognition
No revenue was recognized related to the transaction during thethree and nine months ended September 30, 2024 and 2023, respectively. The Company hadno accounts receivable from Novo Nordisk as of September 30, 2024 and December 31, 2023, respectively.
- Shareholders' Equity
Ordinary Shares
As of September 30, 2024, the Company had 100,000,000 ordinary shares authorized for issuance with a par value of $0.01 per ordinary share and 53,808,732 ordinary shares issued and outstanding. Each ordinary share is entitled toone vote and, on a pro rata basis, to dividends when declared and the remaining assets of the Company in the event of a winding up.
Euro Deferred Shares
As of September 30, 2024, the Company had 10,000 Euro Deferred Shares authorized for issuance with a nominal value of €22 per share. No Euro Deferred Shares are outstanding at September 30, 2024. The rights and restrictions attaching to the Euro Deferred Shares rank pari passu with the ordinary shares and are treated as a single class in all respects.
December 2022 Offering
In December 2022, the Company completed an underwritten public offering of an aggregate of 3,250,000 of its ordinary shares at a public offering price of $56.50 per ordinary share. The Company received aggregate net proceeds of approximately $172.4 million, after deducting the underwriting discount and offering costs.
In January 2023, the Company issued an additional 395,096 ordinary shares resulting from the underwriters’ partial exercise of their 30-day option to purchase up to an additional 487,500 ordinary shares of as part of the December 2022 underwritten public offering. The Company received approximately $20.9 million proceeds from the exercise, net of underwriting discount but before deducting any offering costs.
At-the-Market Offerings
In December 2021, the Company entered into an Equity Distribution Agreement (the “December 2021 Distribution Agreement”), pursuant to which the Company could issue and sell, from time to time, the Company's ordinary shares. In connection with entering into the December 2021 Distribution Agreement, on December 23, 2021, the Company filed with the SEC a prospectus supplement relating to the offer, issuance and sale of up to $250.0 million of the Company’s ordinary shares (the “December 2021 Prospectus”) pursuant to the December 2021 Distribution Agreement.
For thethree and nine months ended September 30, 2023, the Company sold and issuednil and 42,361 ordinary shares, respectively, pursuant to the December 2021 Distribution Agreement under the December 2021 Prospectus. For thenine months ended September 30, 2023, total gross proceeds were approximately$3.2 million before deducting underwriting discounts, commissions, and other offering expenses payable by the Company of$0.1 million.
The December 2021 Prospectus was no longer effective as of March 23, 2024. As of March 23, 2024, the Company had sold and issued 953,589 ordinary shares pursuant to the December 2021 Distribution Agreement under the December 2021 Prospectus for total gross proceeds of approximately$56.3 million before deducting underwriting discounts, commissions, and other offering expenses payable by the Company of$1.8 million.
In February 2024, the Company amended the Equity Distribution Agreement that it entered into in December 2021 (the “Amended Distribution Agreement”), pursuant to which the Company may issue and sell, from time to time, the Company's ordinary shares. In connection with amending the Amended Distribution Agreement, on February 22, 2024, the Company filed with the SEC a prospectus relating to the offer, issuance, and sale of up to $250.0 million of the Company’s ordinary shares (the “February 2024 Prospectus”) pursuant to the Amended Distribution Agreement. For thethree and nine months ended September 30, 2024, the Company sold and issuedno ordinary shares pursuant to the Amended Distribution Agreement under the February 2024 Prospectus.
The issuance and sale of the Company’s ordinary shares pursuant to the December 2021 Distribution Agreement and the Amended Distribution Agreement is deemed an “at-the-market” offering and is registered under the Securities Act of 1933, as amended.
- Share-Based Compensation
Equity Incentive Plans
The Company’s equity incentive plans, the 2018 Long Term Incentive Plan, as amended (the “2018 LTIP”),2020 Employment Inducement Incentive Plan, as amended (the “2020 EIIP”), and previously, the Amended and Restated 2012 Long Term Incentive Plan (the “2012 LTIP”), reserve ordinary shares for the issuance of stock options, stock appreciation rights, restricted shares, RSUs, performance bonus awards, performance share units awards, dividend equivalents and other shareor cash-based awards to eligible individuals. Options granted under each of the 2018 LTIP, 2020 EIIP, and 2012 LTIP expire no later thanten years from the date ofgrant.
In May 2024, the Company’s shareholders approved an amendment to the 2018 LTIP to increase the number of ordinary shares available for issuance under the 2018 LTIP by 2,000,000 ordinary shares. As of September 30, 2024, the number of ordinary shares authorized under the 2018 LTIP was 16,620,433. Upon adoption of the 2018 LTIP, no new awards are permitted under the 2012 LTIP.
As of September 30, 2024, the number of ordinary shares authorized under the 2020 EIIP was 1,485,000 and 186,043 ordinary shares remained available for future awards under the 2020 EIIP. The Company’s Board of Directors has adopted a series of amendments to increase the ordinary shares available for issuance under the 2020 EIIP and it reserves the right to both amend the 2020 EIIP to increase the number of ordinary shares available and make additional awards to key new hires.
The Company’s option awards generally vest over four years, while RSUs vest overtwo years. As of September 30, 2024,4,029,431 ordinary shares remained available for grant under its equity plans.
Share-based Compensation Expense
Share-based compensation expense recorded in these Condensed Consolidated Financial Statements was based on awards granted under the 2012 LTIP, the 2018 LTIP, and the 2020 EIIP. The estimated forfeiture rate as of September 30, 2024 was 7%. Changes in our estimates and assumptions relating to forfeitures may cause us to realize material changes in stock-based compensation expense in the future.
The amount of unearned share-based compensation related to unvested stock options at September 30, 2024, is$84.9 million. The weighted-average period over which this unearned share-based compensation is expected to be recognized is 2.66 years.
The following table summarizes share-based compensation expense for the periods presented (in thousands):
| Three Months Ended September 30, | Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |
| Research and development | $5,128 | $4,948 | $16,217 | $14,178 |
| General and administrative | 5,892 | 5,996 | 19,227 | 15,656 |
| Total share-based compensation expense | $11,020 | $10,944 | $35,444 | $29,834 |
The Company recognized tax benefits from share-based awards of $2.0 million and $1.9 million for the three months ended September 30, 2024 and 2023, respectively, and $6.3 million and $5.3 million for the nine months ended September 30, 2024 and 2023, respectively.
The fair value of the options granted to employees and non-employee directors during the three months ended September 30, 2023 and nine months ended September 30, 2024 and 2023 was estimated as of the grant date using the Black-Scholes option-pricing model using the key assumptions listed in the following table. There wereno stock options granted during the three months ended September 30, 2024, thus no key assumptions are shown in the table below.
| Three Months Ended September 30, · Nine Months Ended September 30, | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| 2023 · 2024 · 2023 | |||||||||
| Expected term (in years) | 4.42 | - | 4.42 | 4.60 | - | 5.56 | 4.38 | - | 5.40 |
| Expected volatility | 76.8% | - | 76.8% | 74.5% | - | 78.6% | 76.4% | - | 90.1% |
| Risk-free interest rate | 4.3% | - | 4.4% | 3.8% | - | 4.7% | 3.5% | - | 4.4% |
| Expected dividend yield | —% | —% | —% | ||||||
| Weighted average grant date fair value | $40.83 | $19.21 | $37.67 | ||||||
The fair value of employee stock options is amortized on a straight-line basis over the requisite service period for each award. Each of the inputs discussed above is subjective and generally requires management judgment to determine.
The following table summarizes the Company’s stock option activity during the nine months ended September 30, 2024:
| Options | WeightedAverageExercisePrice | WeightedAverageRemainingContractualTerm (years) | AggregateIntrinsicValue(in thousands) | |
|---|---|---|---|---|
| Outstanding at December 31, 2023 | 9,866,337 | $29.06 | 6.60 | $118,447 |
| Granted | 2,156,450 | 29.45 | ||
| Exercised | (125,615) | 15.19 | ||
| Forfeited | (470,719) | 36.14 | ||
| Expired | (129,710) | 30.92 | ||
| Outstanding at September 30, 2024 | 11,296,743 | $28.97 | 6.25 | $11,170 |
| Vested and expected to vest at September 30, 2024 | 10,968,503 | $28.74 | 6.16 | $11,169 |
| Vested at September 30, 2024 | 7,521,340 | $24.85 | 5.00 | $11,125 |
The total intrinsic value of options exercised was $0.3 million and $5.7 million during thethree months ended September 30, 2024 and 2023 respectively, and $1.3 million and $51.1 million during the nine months ended September 30, 2024 and 2023, respectively, determined as of the date of exercise.
The following table summarizes the activity and related information for RSUs during the nine months ended September 30, 2024:
| Number of Units | Weighted AverageGrant-DateFair Value | WeightedAverageRemainingContractualTerm (years) | AggregateIntrinsicValue(in thousands) | |
|---|---|---|---|---|
| Unvested at December 31, 2023 | 25,250 | $58.01 | 1.09 | $918 |
| Units Granted | — | — | ||
| Units Vested | (1,000) | 68.71 | ||
| Units Forfeited | — | — | ||
| Unvested at September 30, 2024 | 24,250 | $57.57 | 0.37 | $406 |
| Unvested and expected to vest at September 30, 2024 | 23,620 | $57.71 | 0.36 | $395 |
The fair value of RSUs was determined on the date of grant based on the market price of the Company’s ordinary shares as of that date. The fair value of the RSUs is recognized as an expense on a straight-line basis over the vesting period of each RSU. Upon the vesting of the RSUs, a portion of the shares vested are sold by the employee to satisfy employee withholding tax requirements (sell-to-cover). As of September 30, 2024, total compensation cost not yet recognized related to unvested RSUs was $0.3 million, which is expected to be recognized over a weighted-average period of 0.41 years. RSUs settle into ordinary shares upon vesting.
- Income Taxes
The major taxing jurisdictions for the Company are Ireland and the U.S. The Company recordedan income tax benefitof$0.8 million and$5.1 million for the three and nine months ended September 30, 2024, as compared to $3.1 million and$10.3 million for the three and nine months ended September 30, 2023, respectively. The provision for income taxes differs from the statutory tax rate of 12.5%applicable to Ireland primarily due to Irish net operating losses for which a tax provision benefit is not recognized, U.S. income taxed at different rates, adjustments to deferred tax assets for the deductibility of stock compensation and capitalization of research and development costs.
The Company has generally computed its interim period provision for (benefit from) income taxes by applying its forecasted effective tax rate to year-to-date earnings. However, due to a significant amount of U.S. permanent differences relative to the amount of U.S. forecasted income used in computing the effective tax rate, the effective tax rate is highly sensitive to minor fluctuations in U.S. forecasted income. As such, the Company has computed the U.S. component of the consolidated provision for (benefit from) income taxes for thethree and nine months ended September 30, 2024, using an actual year-to-date tax calculation.
The non-U.S. tax expense continues to be zero due to cumulative historic and year-to-date losses and a full valuation allowance on our deferred tax assets in our non-U.S. jurisdictions.
Deferred income taxes reflect the net tax effect of temporary differences between the carrying amount of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The Company's deferred tax assets (“DTA”) are composed primarily of its Irish subsidiaries' net operating loss carryforwards, California net operating loss carryforwards available to reduce future taxable income of the Company's U.S. subsidiaries, federal and California tax credit carryforwards, share-based compensation, capitalized R&D, and other temporary differences. The Company maintains a valuation allowance against certain U.S. federal and state and Irish deferred tax assets. Each reporting period, the Company evaluates the need for a valuation allowance on its deferred tax assets by jurisdiction.
No provision for income tax in Ireland has been recognized on undistributed earnings of the Company’s U.S. subsidiaries as the Company considers the U.S. earnings to be indefinitely reinvested.
The Company is subject to reviews and audits by the U.S. Internal Revenue Service (“IRS”), the Irish Revenue Commissioners, and other taxing authorities from time to time. The Company’s U.S. subsidiaries are currently under examination by the IRS for tax year 2021. The Company periodically reviews its uncertain tax positions. The Company’s assessment is based on many factors, including any ongoing IRS audits. For the three and nine months ended September 30, 2024, the Company’s assessment did not result in a material change in unrecognized tax benefits.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.