What changed in this filing

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Item 1. Financial Statements

2026-08-06 compared with 2026-05-07 · 202 added, 79 removed, 420 unchanged (40% of the section changed)

3 unchanged sentences

(In thousands, except share amounts)

March 31, 2026 December 31, 2025

June 30, 2026 December 31, 2025

Current assets:

1 unchanged sentence

Short-term marketable securities 508,941 662,553

Accounts receivable, net 3,927 —

Inventory 4,139 —

Prepaid expenses and other current assets 35,390 32,779

19 unchanged sentences

Convertible preferred stock, $ 0.01 par value;

40,000,000 shares authorized as of March 31, 2026 and December 31, 2025;

0 shares issued and outstanding as of March 31, 2026 and December 31, 2025

40,000,000 shares authorized as of June 30, 2026 and December 31, 2025;

0 shares issued and outstanding as of June 30, 2026 and December 31, 2025

Common stock, $ 0.01 par value;

400,000,000 shares authorized as of March 31, 2026 and December 31, 2025;

158,656,318 shares and 156,182,177 shares issued and outstanding as of March 31, 2026 and December 31, 2025, respectively

400,000,000 shares authorized as of June 30, 2026 and December 31, 2025;

159,755,526 shares and 156,182,177 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively

Additional paid-in capital 3,142,747 3,062,715

7 unchanged sentences

(In thousands, except share and per share amounts)

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

2026 2025 2026 2025

Product revenue, net $ 3,604 $ — $ 3,604 $ —

Total revenue 3,604 — 3,604 —

Operating expenses:

Cost of goods sold 93 — 93 —

Research and development 97,019 102,696 200,865 218,923

$ 103,846 $ 116,227

General and administration 33,511 29,353

Selling, general and administrative 36,283 32,267 69,794 61,620

Intangible asset amortization 750 — 750 —

Total operating expenses 134,145 134,963 271,502 280,543

2 unchanged sentences

Net loss $ ( 127,553 ) $ ( 124,119 ) $ ( 256,000 ) $ ( 257,089 )

Other comprehensive income (loss):

Net unrealized gain (loss) on marketable securities, net of tax ( 1,364 ) 17

Other comprehensive loss:

Net unrealized loss on marketable securities, net of tax ( 1,287 ) ( 1,098 ) ( 2,651 ) ( 1,081 )

Comprehensive loss $ ( 128,840 ) $ ( 125,217 ) $ ( 258,651 ) $ ( 258,170 )

9 unchanged sentences

Balance at December 31, 2025 156,182,177 $ 1,888 $ 3,062,715 $ 682 $ ( 2,051,524 ) $ 1,013,761

Issuance of common stock, net of issuance cost of $ 45

746,468 8 12,358 — — 12,366

Issuances under equity incentive plans 1,484,925 14 21,686 — — 21,700

2 unchanged sentences

Net loss — — — — ( 256,000 ) ( 256,000 )

Other comprehensive income

— — — 17 — 17

Other comprehensive loss — — — ( 2,651 ) — ( 2,651 )

June 30, 2026 159,755,526 $ 1,924 $ 3,142,747 $ ( 1,969 ) $ ( 2,307,524 ) $ 835,178

Balance at March 31, 2026 158,656,318 $ 1,913 $ 3,104,838 $ ( 682 ) $ ( 2,179,971 ) $ 926,098

Balance at December 31, 2025 156,182,177 $ 1,888 $ 3,062,715 $ 682 $ ( 2,051,524 ) $ 1,013,761

Issuance of common stock, net of issuance cost of $ 45

746,468 8 12,358 — — 12,366

Issuances under equity incentive plans 948,205 9 14,996 — — 15,005

4 unchanged sentences

— — — ( 1,287 ) — ( 1,287 )

June 30, 2026 159,755,526 $ 1,924 $ 3,142,747 $ ( 1,969 ) $ ( 2,307,524 ) $ 835,178

Balance at December 31, 2024 144,220,986 $ 1,768 $ 2,764,880 $ 2,020 $ ( 1,538,984 ) $ 1,229,684

Issuances under equity incentive plans 455,335 5 4,647 — — 4,652

Vesting of restricted stock units 1,009,121 10 ( 10 ) — — —

Stock-based compensation — — 50,887 — — 50,887

Net loss — — — — ( 257,089 ) ( 257,089 )

Other comprehensive loss — — — ( 1,081 ) — ( 1,081 )

Balance at June 30, 2025 145,685,442 $ 1,783 $ 2,820,404 $ 939 $ ( 1,796,073 ) $ 1,027,053

Balance at March 31, 2025 145,251,258 $ 1,778 $ 2,790,825 $ 2,037 $ ( 1,671,954 ) $ 1,122,686

Issuances under equity incentive plans

341,543 4 4,053 — — 4,057

Vesting of restricted stock units 92,641 1 ( 1 ) — — —

Stock-based compensation

— — 25,527 — — 25,527

Net loss — — — — ( 124,119 ) ( 124,119 )

Other comprehensive loss — — — ( 1,098 ) — ( 1,098 )

Balance at June 30, 2025 145,685,442 $ 1,783 $ 2,820,404 $ 939 $ ( 1,796,073 ) $ 1,027,053

See accompanying notes to unaudited condensed consolidated financial statements.

2 unchanged sentences

(In thousands)

Three Months Ended

Six Months Ended

Operating activities

7 unchanged sentences

Right-of-use asset amortization for finance lease

Non-cash interest expense related to revenue participation right liability

Other non-cash items

Changes in operating assets and liabilities:

Accounts receivable, net ( 3,927 ) —

Inventory ( 4,139 ) —

Prepaid expenses and other current assets ( 2,613 ) ( 3,650 )

8 unchanged sentences

Purchases of property and equipment ( 3,271 ) ( 9,356 )

Net cash provided by investing activities 94,460 18,748

Cash paid to acquire finite-lived intangible asset ( 28,800 ) —

Net cash provided by (used in) investing activities ( 9,618 ) 176,683

Financing activities

4 unchanged sentences

Net cash provided by (used in) financing activities 234,028 ( 2,139 )

Net increase (decrease) in cash, cash equivalents and restricted cash 182,300 ( 116,482 )

Net decrease in cash, cash equivalents and restricted cash ( 3,796 ) ( 32,222 )

Cash, cash equivalents and restricted cash at beginning of period 208,432 176,535

19 unchanged sentences

These interim financial results are not necessarily indicative of results expected for the full fiscal year or for any subsequent interim period.

During the three months ended March 31, 2026 the Company added significant accounting policies related to intangible assets and the liability related to the revenue participation right.

During the six months ended June 30, 2026 the Company added significant accounting policies related to product revenue, accounts receivable, inventory, cost of goods sold, intangible assets and the liability related to the revenue participation right.

Aside from these new accounting policies, there were no material changes to the Company's significant accounting and financial reporting policies from those reflected in the 2025 Annual Report on Form 10-K.

11 unchanged sentences

Concentration of Credit Risk and Other Risks and Uncertainties

Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash, cash equivalents, and marketable securities.

Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash, cash equivalents, marketable securities, and accounts receivable.

Substantially all of the Company’s cash and cash equivalents are deposited in accounts with financial institutions that management believes are of high credit quality.

4 unchanged sentences

The Company is exposed to credit risk in the event of a default by the financial institutions holding its cash, cash equivalents and marketable securities and issuers of marketable securities to the extent recorded on the Condensed Consolidated Balance Sheets.

As of March 31, 2026 and December 31, 2025, the Company had no off-balance sheet concentrations of credit risk.

As of June 30, 2026 and December 31, 2025, the Company had no off-balance sheet concentrations of credit risk.

The Company’s accounts receivable are primarily related to product sales.

For the three and six months ended June 30, 2026, one customer accounted for all product revenues.

As of June 30, 2026, one customer accounted for all accounts receivable related to product sales.

The Company monitors the creditworthiness of its customers and collaboration partners and has not experienced significant credit losses to date.

The Company is subject to a number of risks similar to other early commercial-stage biopharmaceutical companies, including, but not limited to, the risk that its approved product, AVLAYAH TM (tividenofusp alfa-eknm), may not achieve expected levels of market acceptance or commercial success;

19 unchanged sentences

Impairments, if any, are based on the excess of the carrying amount over the recoverable amount of the asset.

There were no impairments during the three months ended March 31, 2026 and 2025.

There were no impairments during the six months ended June 30, 2026 and 2025.

Cash, Cash Equivalents and Restricted Cash

1 unchanged sentence

Cash equivalents are reported at fair value.

Cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Statements of Cash Flows is composed of cash and cash equivalents reported in the Condensed Consolidated Balance Sheets and restricted cash of $ 3.1 million as of March 31, 2026 and December 31, 2025, which is included within other non-current assets in the Condensed Consolidated Balance Sheets.

Cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Statements of Cash Flows is composed of cash and cash equivalents reported in the Condensed Consolidated Balance Sheets and restricted cash of $ 3.1 million as of June 30, 2026 and December 31, 2025, which is included within other non-current assets in the Condensed Consolidated Balance Sheets.

Restricted cash relates to letters of credit supporting the Company’s headquarters building lease and a letter of credit supporting the Company’s credit card program.

10 unchanged sentences

Accounts Receivable

Accounts receivable are included within prepaid expenses and other current assets on the Condensed Consolidated Balance Sheets.

The accounts receivable balance represents amounts receivable from the Company's collaboration partners net of an allowance for credit losses, if required.

Accounts receivable represents amounts arising from product sales and is recorded net of allowances for credit losses, if required.

The Company estimates an allowance for credit losses by considering factors such as credit quality, the age of the accounts receivable balances, and current economic conditions that may affect a customer’s ability to pay.

The Company's payment terms are generally less than 90 days from the invoice date.

The Company evaluates the creditworthiness of each counterparty on a regular basis.

As of June 30, 2026, the credit profile of the customer was deemed to be in good standing and, as such, an allowance for credit losses was not recorded.

Revenue Recognition

The Company recognizes product revenue in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”), Topic 606, Revenue from Contracts with Customers (“ASC 606”).

In March 2026, the U.S.

Food and Drug Administration granted accelerated approval of AVLAYAH™ (tividenofusp alfa-eknm), and the Company commenced commercial sales in the United States during the three months ended June 30, 2026.

AVLAYAH is the Company’s only commercial product, and all product revenue to date has been generated in the United States.

Under ASC 606, the Company is required to complete the following five steps:

(i) identify the contract(s) with a customer;

(ii) identify the performance obligations in the contract;

(iii) determine the transaction price, including variable consideration, if any;

(iv) allocate the transaction price to the performance obligations in the contract;

and (v) recognize revenue when (or as) the entity satisfies a performance obligation.

The Company sells AVLAYAH to a single specialty distributor ("Distributor”) in the US, which is the Company's sole customer, who delivers to (i) hospitals where patients are administered the medication in a healthcare setting and (ii) to a specialty pharmacy (“SP”) provider where patients are administered by a healthcare professional in either a healthcare professional or home setting.

The Company’s distribution agreements with the Distributor, together with each accepted purchase order, is accounted for as a contract with a single performance obligation — the delivery of AVLAYAH to the Distributor.

Revenue is recognized when the Company satisfies a performance obligation by transferring control of the promised goods to the customer.

Revenue is measured as the amount of consideration the Company expects to receive in exchange for transferring AVLAYAH.

The Company sells AVLAYAH at a Wholesale Acquisition Cost (“WAC”) and records product revenue at the net selling price (the transaction price) — WAC reduced by variable consideration and by consideration payable to the Distributor and to other parties in the distribution channel that does not represent payment for a distinct good or service.

The Company estimates variable consideration using the most-likely-amount method and includes it in the transaction price only to the extent it is probable that a significant reversal of cumulative revenue recognized will not occur when the related uncertainty is resolved.

Estimates are reassessed each reporting period, with any change recognized in net product revenue in the period of change.

The components of variable consideration are:

• Distributor service fees — fees payable to the Distributor for distribution.

These services are not distinct from the sale of AVLAYAH and the related fees, calculated as a contractual percentage of the WAC of product sold, are recorded as a reduction of product revenue when the related sales are recognized.

• Government rebates — amounts owed under the Medicaid Drug Rebate Program and supplemental state Medicaid agreements, refunds under the Medicare Part B discarded-drug provisions, and rebates to the U.S.

Department of Veterans Affairs and the Department of Defense (including under the TRICARE program).

The Company estimates these amounts using statutory and contractual rebate

rates and probability-weighted assumptions for the expected payer and channel mix, recorded as a reduction of product revenue with a corresponding current liability.

• Product returns — the product may be returned only in the limited circumstances specified in the distribution agreement, principally product that is damaged or within a defined period of its expiration date.

The Company records a reserve for estimated returns as a reduction of product revenue with a corresponding refund liability.

• Other fees and incentives — amounts payable to a specialty pharmacy that dispenses AVLAYAH purchased through the Distributor and co-pay assistance program for eligible patients with commercial insurance in the U.S.

The co-pay assistance programs assist commercially insured patients and are intended to reduce each participating patient’s portion of the financial responsibility of the purchase price up to a specified dollar amount of assistance.

The Company records funds paid under co-pay assistance program for each patient as a reduction of revenue.

Variable consideration payable to the Distributor, and to parties against which the Distributor has a contractual right of off-set — Distributor service fees, third-party-logistics fees, and government chargebacks processed through the Distributor is presented as a reduction of accounts receivable.

Variable consideration payable to parties other than the Distributor is presented within other accrued costs and current liabilities.

Inventory is stated at the lower of cost or net realizable value, with cost determined on a first-in, first-out ("FIFO") basis.

Inventory costs consist primarily of raw materials, third-party contract manufacturing organization ("CMO") costs, and other direct production costs, including allocable overhead, related to our commercial product AVLAYAH.

Inventory is valued using standard costing methodology, which approximates actual cost.

Prior to FDA approval, all costs incurred in connection with the manufacture of AVLAYAH were recorded as research and development expenses in the consolidated statements of operations in the period incurred.

As of June 30, 2026, we had $ 12.6 million of expensed pre-launch inventory, which we expect to be sold through by January 2027.

The Company expenses costs associated with the manufacture of product candidates prior to regulatory approval.

Inventory consists of the Company’s currently approved product, including raw materials and work-in-process.

The Company began capitalizing inventory costs associated with AVLAYAH upon receiving U.S.

Food and Drug Administration ("FDA") accelerated approval in March 2026, at which time the inventory was determined to have probable future economic benefit.

Additionally, inventory used in research and development activities is expensed to research and development at the time of such designation.

The Company evaluates its inventory on a periodic basis for obsolescence, slow-moving, excess, or otherwise unsalable items, and records a write-down to net realizable value when the carrying value is in excess of net realizable value and there are no alternative future uses for the inventory, or if inventory quantities exceed expected future demand prior to expiration.

If the Company determines an adjustment is required to the carrying value of inventory, those adjustments are recognized as Cost of Goods Sold in the Consolidated Statements of Operations and Comprehensive Loss.

Inventory that is not expected to be consumed beyond our normal operating cycle is classified as non-current inventory and included within other non-current assets in the balance sheet.

Cost of Goods Sold

Cost of Goods Sold consists primarily of direct and indirect costs related to the manufacture of AVLAYAH for commercial sale, including third-party manufacturing costs, raw material and component costs, packaging services, freight, and storage costs.

Prior to the FDA approval of AVLAYAH in March 2026, costs incurred for the manufacture of AVLAYAH were recorded as research and development expenses and therefore will not be included in cost of sales.

As a result, the cost of goods sold related to AVLAYAH will initially reflect a lower average per unit cost of materials, as previously expensed inventory is utilized and sold to customers.

Intangible Assets

18 unchanged sentences

The resulting non-cash interest expense is recognized in Interest and other income, net in the Condensed Consolidated Statements of Operations and Comprehensive Loss.

The liability related to the revenue participation right and related interest expense are based on current estimates of the amount and timing of projected royalty payments, which are based on estimates of future AVLAYAH net sales.

of future AVLAYAH net sales.

The Company periodically assesses the forecasted net sales and to the extent the amount or timing of estimated royalty payments are materially different than previous estimates, the Company will account for any such change by adjusting the liability related to the sale of future revenues and prospectively recognizing the related non-cash interest expense.

6 unchanged sentences

Diluted net loss per share is the same as basic net loss per share, since the effects of potentially dilutive securities are antidilutive given the net loss for each period presented.

The weighted-average common shares outstanding as of March 31, 2026 include the pre-funded warrants to purchase shares of common stock that were issued in connection with the February 2024 private placement and December 2025 public offering, as discussed further in Note 10.

The weighted-average common shares outstanding as of June 30, 2026 include the pre-funded warrants to purchase shares of common stock that were issued in connection with the February 2024 private placement and December 2025 public offering, as discussed further in Note 11.

Recently Issued Accounting Pronouncements

8 unchanged sentences

Assets and liabilities measured at fair value at each balance sheet date are as follows (in thousands):

March 31, 2026

June 30, 2026

Level 1 Level 2 Level 3 Total

22 unchanged sentences

The pricing services utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly.

The Company has not transferred any assets or liabilities between the fair value measurement levels for the three months ended March 31, 2026 or for the year ended December 31, 2025.

The Company has not transferred any assets or liabilities between the fair value measurement levels for the six months ended June 30, 2026 or for the year ended December 31, 2025.

Marketable Securities

All marketable securities were considered available-for-sale at March 31, 2026 and December 31, 2025.

All marketable securities were considered available-for-sale at June 30, 2026 and December 31, 2025.

On a recurring basis, the Company records its marketable securities at fair value using Level 1 or Level 2 inputs as discussed in Note 2, "Fair Value Measurements".

The amortized cost, gross unrealized holding gains or losses, and fair value of the Company’s marketable securities by major security type at each balance sheet date are summarized in the tables below (in thousands):

March 31, 2026

June 30, 2026

Amortized Cost Unrealized Holding Gains Unrealized Holding Losses Aggregate Fair Value

31 unchanged sentences

$ 759,841 $ 1,054 $ ( 20 ) $ 760,875

As of March 31, 2026 and December 31, 2025, some of the Company's marketable securities were in an unrealized loss position.

The Company has no t recognized an allowance for credit losses as of March 31, 2026 or December 31, 2025.

As of June 30, 2026 and December 31, 2025, some of the Company's marketable securities were in an unrealized loss position.

The Company has no t recognized an allowance for credit losses as of June 30, 2026 or December 31, 2025.

The Company determined that it had the ability and intent to hold all marketable securities that have been in a continuous loss position until maturity or recovery.

2 unchanged sentences

All marketable securities with unrealized losses as of each balance sheet date have been in a loss position for less than twelve months or the loss is not material.

As of March 31, 2026, all of the Company’s marketable securities have an effective maturity of less than two years .

As of June 30, 2026, all of the Company’s marketable securities have an effective maturity of less than two years .

Inventory consisted of the following (in thousands):

June 30, 2026 December 31, 2025

Raw materials $ — $ —

Work in process 4,139 —

Finished goods — —

Total inventory $ 4,139 $ —

No adjustments to the carrying value of inventory were recorded during the three and six months ended June 30, 2026 and 2025, respectively.

All inventory as of June 30, 2026 is classified as current.

Intangible Asset

3 unchanged sentences

Food and Drug Administration’s accelerated approval of AVLAYAH, a $ 36.0 million milestone payment became due under this arrangement.

The amount was capitalized as an intangible asset and will be amortized on a straight-line basis over its estimated useful life.

The amount is included in accounts payable in the Condensed Consolidated Balance Sheet as of March 31, 2026, and is expected to be fully paid by Q3 2026.

The amount was capitalized as an intangible asset and is amortized on a straight-line basis over its estimated useful life.

The Company paid $ 28.8 million of the milestone payment during the three months ended June 30, 2026, and the remaining $ 7.2 million is included in accounts payable in the Condensed Consolidated Balance Sheet as of June 30, 2026.

The Company recognized amortization expense of $ 0.7 million for the three and six months ended June 30, 2026.

As of June 30, 2026, the accumulated amortization expense was $ 0.7 million, and the carrying value of the intangible asset was $ 35.3 million.

Future annual amortization expense for the unamortized intangible assets is as follows (in thousands):

Years Ending December 31:

2026 (six months) $ 1,500

Thereafter 21,750

Total $ 35,250

In addition to the U.S.

1 unchanged sentence

Research and Development Funding Collaboration Agreement

In January 2024, the Company entered into a Collaboration and Development Funding Agreement with an unrelated third party, which obligates third party to provide up to $ 75.0 million of funding and collaborate with the Company to conduct a global Phase 2a study of BIIB122/DNL151 in patients with Parkinson’s disease and confirmed pathogenic variants of LRRK2.

In January 2024, the Company entered into a Collaboration and Development Funding Agreement with an unrelated third party, which obligates the third party to provide up to $ 75.0 million of funding and collaborate with the Company to conduct a global Phase 2a study of DNL151 in patients with Parkinson’s disease and confirmed pathogenic variants of LRRK2.

This arrangement is further described in Note 4, " Acquisition, License Agreement and Research and Development Funding Collaboration Agreement", to the consolidated financial statements in the 2025 Annual Report on Form 10-K.

Under this arrangement, the Company recognized an offset to research and development expenses of $ 6.0 million and $ 5.1 million in the Condensed Consolidated Statements of Operations and Comprehensive Loss for the three months ended March 31, 2026 and 2025, respectively.

The Company recorded current deferred research and development funding liabilities of $ 15.1 million and $ 21.1 million on the Condensed Consolidated Balance Sheet as of March 31, 2026 and December 31, 2025, respectively.

Under this arrangement, the Company received $ 12.5 million during the three months ended June 30, 2026, resulting in cumulative payments received of $ 62.5 million as of June 30, 2026.

Payments received are recorded as a deferred research and development funding liability and recognized as an offset to research and development expenses as the underlying research and development costs are incurred.

The Company recognized an offset to research and development expenses of $ 7.6 million and $ 3.8 million for the three months ended June 30, 2026 and 2025, respectively, and $ 13.6 million and $ 8.8 million for the six months ended June 30, 2026 and 2025, respectively, in the Condensed Consolidated Statements of Operations and Comprehensive Loss.

The Company recorded current deferred research and development funding liabilities of $ 20.0 million and $ 21.1 million on the Condensed Consolidated Balance Sheets as of June 30, 2026 and December 31, 2025, respectively.

Collaboration Agreements

2 unchanged sentences

The details of the Biogen Collaboration Agreement, the August 2023 amendment and July 2024 side letter to the ROFN and Option Agreement, and the payments the Company has received, and is entitled to receive, are further described in Note 5, "Collaboration Agreements", to the consolidated financial statements in the 2025 Annual Report on Form 10-K.

The Company has no remaining performance obligations under the Biogen Collaboration Agreement, and therefore no contract liability remained on the Condensed Consolidated Balance Sheets as of March 31, 2026 or December 31, 2025 .

As of March 31, 2026, the Company had no t recorded milestone revenue or product sales under the Biogen Collaboration Agreement.

In May 2026, the Company and Biogen announced topline results from the Phase 2b LUMA study of BIIB122/DNL151 in individuals with early-stage Parkinson’s disease.

The LUMA study did not meet its primary or secondary endpoints.

Based on these results, the Company and Biogen discontinued further development of BIIB122 in idiopathic Parkinson's disease.

The Company continues to independently conduct the Phase 2a BEACON study evaluating the small molecule inhibitor in carriers of a pathogenic LRRK2 variant.

As of June 30, 2026, the Company had no t recorded milestone revenue or product sales under the Biogen Collaboration Agreement.

In October 2018, the Company entered into a Collaboration and License Agreement ("Sanofi Collaboration Agreement") with Genzyme Corporation, a wholly-owned subsidiary of Sanofi S.A.

The details of the Sanofi Collaboration Agreement, the February 2025 side letter, and the payments the Company has received, and is entitled to receive, are further described in Note 5, "Collaboration Agreements", to the consolidated financial statements in the Company's 2025 Annual Report on Form 10-K.

The Company has no remaining performance obligations under the Sanofi Collaboration Agreement, and therefore no contract liability remains on the Condensed Consolidated Balance Sheets as of March 31, 2026 or December 31, 2025 .

Under the Sanofi Collaboration Agreement, the Company is eligible to receive milestone payments totaling up to approximately $ 495.0 million upon achievement of certain clinical, regulatory and sales milestone events for Peripheral Products, and variable royalties on worldwide net sales.

As of March 31, 2026, the Company had earned milestone payments of $ 100.0 million and had no t recorded any product sales under the Sanofi Collaboration Agreement.

As of June 30, 2026, the Company had earned milestone payments of $ 100.0 million and had no t recorded any product sales under the Sanofi Collaboration Agreement.

In January 2018, the Company entered into a Collaboration and Option Agreement ("Takeda Collaboration Agreement") with Takeda Pharmaceutical Company Limited ("Takeda").

3 unchanged sentences

The February 2025 ATV:TREM2 discontinuation and the PTV:PGRN Collaboration Agreement are further described in Note 5, "Collaboration Agreements", to the consolidated financial statements in the Company's 2025 Annual Report on Form 10-K.

In April 2026, Takeda notified the Company of its decision to terminate the collaboration agreement related to PTV:PGRN.

As a result, the Company will regain full rights to DNL593 and the related intellectual property portfolio.

Subsequent to the effective date of the termination of the PTV:PGRN Collaboration Agreement, there are no future milestones, cost, or profit sharing related to this agreement.

As of March 31, 2026, the Company had earned an aggregate of $ 10.0 million in option fee payments and $ 10.0 million in milestone payments from Takeda under the PTV:PGRN and ATV:TREM2 Collaboration Agreements, and had no t recorded any product sales under either agreement.

In April 2026, Takeda notified the Company of its decision to terminate the PTV:PGRN collaboration agreement in its entirety.

As a result, the Company regained full rights to DNL593 and the related intellectual property portfolio.

Subsequent to the effective date of the termination, there are no future milestones, cost, or profit sharing obligations related to this agreement.

The Company did not recognize any revenue, gain, asset or other material accounting impact in connection with the termination, other than the final cost-sharing settlement.

The final cost-sharing settlement related to the PTV:PGRN Collaboration Agreement is reflected in the cost sharing table below.

As of June 30, 2026, the Company had earned an aggregate of $ 10.0 million in option fee payments and $ 10.0 million in milestone payments from Takeda under the PTV:PGRN and ATV:TREM2 Collaboration Agreements, and had no t recorded any product sales under either agreement.

Cost Sharing Payments and Reimbursements

Cost sharing payments to collaboration partners recorded as expenses in research and development expenses in the Condensed Consolidated Statements of Operations and Comprehensive Loss, and cost sharing reimbursements from collaboration partners recorded as an offset to expense in research and development expenses in the Condensed Consolidated Statements of Operations and Comprehensive Loss are as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

2026 2025 2026 2025

Takeda Collaboration Agreement:

6 unchanged sentences

LRRK2 cost sharing payments (2)

2,024 4,229 5,887 8,880

Net cost sharing payments (reimbursements) $ 511 $ 2,780 $ 2,757 $ 5,798

_________________________________

(1) Cost sharing reimbursements of $ 1.6 million and $ 1.6 million were recorded as a receivable within prepaid expenses and other current assets on the Condensed Consolidated Balance Sheets as of March 31, 2026 and December 31, 2025, respectively.

(2) Cost sharing payments due to Biogen of $ 3.9 million and $ 2.8 million were recorded within other accrued costs and current liabilities on the Condensed Consolidated Balance Sheets as of March 31, 2026 and December 31, 2025, respectively.

(1) Cost sharing reimbursements of $ 1.5 million and $ 1.6 million were recorded as a receivable within prepaid expenses and other current assets on the Condensed Consolidated Balance Sheets as of June 30, 2026 and December 31, 2025, respectively.

(2) Cost sharing payments due to Biogen of $ 2.0 million and $ 2.8 million were recorded within other accrued costs and current liabilities on the Condensed Consolidated Balance Sheets as of June 30, 2026 and December 31, 2025, respectively.

Balance Sheet Components

1 unchanged sentence

Accrued expenses and other current liabilities consists of the following (in thousands):

March 31, 2026 December 31, 2025

June 30, 2026 December 31, 2025

Accrued compensation $ 13,424 $ 32,179

11 unchanged sentences

The Company received $ 200.0 million in gross proceeds in March 2026 triggered by the U.S.

Food and Drug and Administration’s accelerated approval of AVLAYAH.

Food and Drug Administration’s accelerated approval of AVLAYAH.

The Company is entitled to receive an additional $ 75.0 million upon approval of AVLAYAH by the European Medicines Agency ("EMA") on or before December 31, 2029.

1 unchanged sentence

The royalty obligation to Royalty Pharma will cease upon cumulative royalty payments reaching a cap of 3.0 times the total funding received, or 2.5 times the funding received if such cap is reached in respect of sales that occur on or before the end of the first quarter of 2039.

The $ 200.0 million, net of transaction costs of approximately $ 0.4 million, is recorded as a non-current liability on the Company's Condensed Consolidated Balance Sheet as of March 31, 2026 based on the estimated timing of future royalty payments.

The $ 200.0 million, net of transaction costs of approximately $ 0.4 million, is recorded as a non-current liability on the Company's Condensed Consolidated Balance Sheet as of June 30, 2026 based on the estimated timing of future royalty payments.

The royalty payments to Royalty Pharma will be recorded as a reduction of the liability.

The additional $ 75.0 million contingent upon EMA approval of AVLAYAH has not been recognized as a liability as of March 31, 2026, as the receipt is contingent upon a future regulatory event.

The additional $ 75.0 million contingent upon EMA approval of AVLAYAH has not been recognized as a liability as of June 30, 2026, as the receipt is contingent upon a future regulatory event.

Upon receipt, the additional proceeds will be recorded as an increase to the liability, net of any incremental transaction costs, and the effective interest rate will be adjusted prospectively.

As of March 31, 2026, the Company's estimate of total future royalty payments resulted in an effective annual interest rate of approximately 11 %.

No royalty payments and non-cash interest expense were recognized during the three months ended March 31, 2026.

As of March 31, 2026, the carrying value of the liability approximates its estimated fair value.

As of June 30, 2026, the Company's estimate of total future royalty payments resulted in an effective annual interest rate of approximately 12 % and recorded $ 5.9 million of non-cash interest expense during the three months ended June 30, 2026.

No payments were made during the three and six months ended June 30, 2026.

As of June 30, 2026, the carrying value of the liability approximates its estimated fair value.

The Company’s projections of future royalty payments are subject to estimation uncertainty and are based on various assumptions underlying projected net product sales over the term of the agreement.

5 unchanged sentences

Both leases are further described in Note 7, "Commitments and Contingencies," to the consolidated financial statements in the Company's 2025 Annual Report on Form 10-K.

There were no changes to the terms of the leases recognized under ASC 842 during the three months ended March 31, 2026 or 2025.

There were no changes to the terms of the leases recognized under ASC 842 during the three and six months ended June 30, 2026 or 2025.

The following table summarizes lease costs recognized for the periods presented (in thousands):

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

2026 2025 2026 2025

Operating lease cost

2 unchanged sentences

Amortization of ROU assets

915 911 1,831 1,798

178 180 357 361

Variable lease cost

1,792 1,338 2,832 2,691

Total lease costs

$ 4,706 $ 4,322 $ 8,694 $ 8,636

Operating cash flows for operating leases were $ 3.0 million and $ 2.9 million for the three months ended March 31, 2026 and 2025, respectively.

Operating cash flows for operating leases were $ 3.0 million and $ 2.9 million for the three months ended June 30, 2026 and 2025, respectively, and $ 6.0 million and $ 5.8 million for the six months ended June 30, 2026 and 2025, respectively.

Indemnification

3 unchanged sentences

In the normal course of business, the Company enters into firm purchase commitments primarily related to supply of commercial product, certain research and development services, and clinical manufacturing activities.

The Company had contractual obligations of $ 109.1 million as of March 31, 2026, of which $ 91.8 million relates to supply of commercial product, and $ 44.3 million as of December 31, 2025.

The Company had contractual obligations of $ 104.7 million as of June 30, 2026, of which $ 91.9 million related to supply of commercial product, and $ 44.3 million as of December 31, 2025.

Contingencies

4 unchanged sentences

In January 2026, the underwriters of the Company’s December 2025 public offering exercised their option to purchase an additional 746,468 shares of common stock, resulting in aggregate net proceeds to the Company of approximately $ 12.4 million.

As of March 31, 2026, 28,331,779 shares of pre-funded warrants remained outstanding, and no pre-funded warrants were exercised during the three months ended March 31, 2026.

As of June 30, 2026, 28,331,779 shares of pre-funded warrants remained outstanding, and no pre-funded warrants were exercised during the six months ended June 30, 2026.

These warrants are classified as permanent equity because they are freestanding financial instruments that are immediately exercisable, do not embody an obligation for the Company to repurchase its shares and permit the holders to receive a fixed number of shares of common stock upon exercise.

2 unchanged sentences

Stock Option Activity

The following table summarizes stock option activity for the three months ended March 31, 2026:

The following table summarizes stock option activity for the six months ended June 30, 2026:

Number of Options

6 unchanged sentences

( 893,471 ) 34.89

Balance at March 31, 2026 22,547,509 $ 25.77

Vested and expected to vest at March 31, 2026 22,547,509 $ 25.77

Exercisable at March 31, 2026 14,509,636 $ 29.32

Balance at June 30, 2026 21,343,159 $ 25.53

Vested and expected to vest at June 30, 2026 21,343,159 $ 25.53

Exercisable at June 30, 2026 14,065,727 $ 28.80

The estimated fair value of stock options granted to employees were calculated using the Black-Scholes option-pricing model using the following assumptions:

Three Months Ended March 31,

Six Months Ended June 30,

Expected term (in years)

6 unchanged sentences

Restricted Stock Activity

The following table summarizes restricted stock unit ("RSU") activity for the three months ended March 31, 2026:

The following table summarizes restricted stock unit ("RSU") activity for the six months ended June 30, 2026:

Number of RSU shares Weighted-Average Fair Value at Date of Grant per Share

3 unchanged sentences

Forfeited ( 563,254 ) 19.84

Unvested and expected to vest at March 31, 2026 6,876,009 $ 18.74

Unvested and expected to vest at June 30, 2026 6,604,540 $ 18.78

Stock-Based Compensation Expense

The Company’s results of operations include expenses relating to stock-based compensation as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

2026 2025 2026 2025

Research and development

2 unchanged sentences

9,462 10,199 18,955 20,222

$ 22,915 $ 25,387 $ 46,002 $ 50,547

Net Loss Per Share

The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share amounts):

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

2026 2025 2026 2025

Net loss $ ( 127,553 ) $ ( 124,119 ) $ ( 256,000 ) $ ( 257,089 )

7 unchanged sentences

Since the Company was in a loss position for all periods presented, basic net loss per share is the same as diluted net loss per share for all periods as the inclusion of all potential shares of common stock outstanding would have been anti-dilutive.

Potentially dilutive securities, including options issued and outstanding, Employee Stock Purchase Plan (“ESPP”) shares issuable, and restricted shares subject to future vesting that were not included in the diluted per share calculations for the periods presented because they would be anti-dilutive totaled approximately 30.0 million and 27.4 million shares as of March 31, 2026 and 2025, respectively.

Potentially dilutive securities, including options issued and outstanding, Employee Stock Purchase Plan (“ESPP”) shares issuable, and restricted shares subject to future vesting that were not included in the diluted per share calculations for the periods presented because they would be anti-dilutive totaled approximately 28.5 million and 28.5 million shares as of June 30, 2026 and 2025, respectively.

Non-Marketable Equity Investment

2 unchanged sentences

The Company recorded the investment in the shares of Series A Preferred Stock at $ 15.0 million which represents the fair value of the shares on the date of issuance.

There have been no subsequent observable price changes in orderly transactions for the identical or similar equity securities of Tenvie, and as such, the measurement of this investment remains unchanged, with the $ 15.0 million included within other non-current assets in the Condensed Consolidated Balance Sheets as of both March 31, 2026 and December 31, 2025.

Segment Information

1 unchanged sentence

There have been no material changes to the Company's segment structure, the basis of segmentation, or the measurement basis for segment profit or loss or segment assets, compared to those disclosed in Note 14, "Segment Information", to the consolidated financial statements in the Company's 2025 Annual Report on Form 10-K.

The following table presents selected financial information with respect to the Company’s single operating segment for the three months ended March 31, 2026 and 2025 (in thousands):

The following table presents selected financial information with respect to the Company’s single operating segment for the three and six months ended June 30, 2026 and 2025 (in thousands):

Therapeutics Segment

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

2026 2025 2026 2025

Product revenue, net $ 3,604 $ — $ 3,604 $ —

Cost of goods sold 93 — 93 —

External research and development expenses - TV programs, including cost sharing 40,419 37,594 78,883 84,052

4 unchanged sentences

Total research and development expenses 97,019 102,696 200,865 218,923

Personnel related general and administrative expenses

Personnel related selling, general and administrative expenses

22,921 20,115 45,485 38,853

Other general and administrative expenses

Other selling, general and administrative expenses

13,362 12,152 24,309 22,767

Total general and administrative expenses 33,511 29,353

Total selling, general and administrative expenses 36,283 32,267 69,794 61,620

Intangible asset amortization $ 750 $ — $ 750 $ —

Segment operating expenses 134,145 134,963 271,502 280,543

3 unchanged sentences

$ ( 127,553 ) $ ( 124,119 ) $ ( 256,000 ) $ ( 257,089 )

There is no difference between the segment net loss and total consolidated net loss for the three months ended March 31, 2026 and 2025.

There is no difference between the segment net loss and total consolidated net loss for the three and six months ended June 30, 2026 and 2025.

Subsequent Event

On June 12, 2026, the Company entered into an asset purchase agreement (the "PRV Transfer Agreement") pursuant to which the Company agreed to sell its Rare Pediatric Disease Priority Review Voucher ("PRV") for gross proceeds of $ 195.0 million in cash.

The Company received the PRV in connection with the FDA accelerated approval of AVLAYAH in March 2026.

On July 27, 2026, the Company completed the sale and received gross proceeds of $ 195.0 million pursuant to the terms of the PRV Transfer Agreement.

No proceeds or gain from the sale were recognized in the condensed consolidated financial statements as of and for the three and six months ended June 30, 2026.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1. Financial Statements