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LICENSE AND COLLABORATION AGREEMENTS
9 Months Ended
Sep. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
LICENSE AND COLLABORATION AGREEMENTS

3. LICENSE AND COLLABORATION AGREEMENTS

F. Hoffman-La Roche Ltd.

For the three and nine months ended September 30, 2023, the Company recognized $22.5 million and $66.8 million, respectively, of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”) related to the amortization of the single, combined performance obligation, with no such activity for the corresponding periods ended September 30, 2024. Under the Roche Agreement, the Company has granted Roche options to acquire ex-U.S. rights to certain future Duchenne development programs (the “Options”) in exchange for separate option exercise payments, milestone and royalty considerations, and cost-sharing provisions. These Options were accounted for as material rights related to individual programs and recorded in deferred revenue at the inception of the Roche Agreement. The value assigned to the material rights is included in deferred revenue until such rights expire or are exercised. On February 12, 2024, Roche declined to exercise its optional rights related to one external, early stage development program, which resulted in the immediate recognition of $48.0 million of collaboration revenue for the nine months ended September 30, 2024. As of September 30, 2024 and December 31, 2023, the Company had total deferred revenue of $452.0 million and $487.4 million, primarily related to the separate material rights for the Options associated with the Roche Agreement, of which $127.0 million and $50.4 million were classified as current, respectively.

The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three and nine months ended September 30, 2024, costs reimbursable by Roche and reflected as a reduction to operating expenses were $61.5 million and $101.1 million, respectively. For the three and nine months ended September 30, 2023, costs reimbursable by Roche and reflected as a reduction to operating expenses were $34.9 million and $83.4 million, respectively. As of September 30, 2024 and December 31, 2023, there were $66.4 million and $29.8 million, respectively, of collaboration and other receivables included in other current assets on the unaudited condensed consolidated balance sheets.

In accordance with the Roche Agreement, the Company agreed to enter into a supply agreement with Roche in order to supply them with clinical and commercial batches of ELEVIDYS (the “Supply Agreement”). While the Supply Agreement is in the process of being negotiated, the Company has delivered batches of commercial ELEVIDYS supply to Roche that were agreed upon on a purchase order-by-purchase order basis. Contract manufacturing revenue and royalty revenue are included in collaboration and other revenue in the accompanying unaudited condensed consolidated statements of comprehensive income (loss). The following table summarizes certain Roche activity for the three and nine months ended September 30, 2024:

 

 

For the Three Months Ended
September 30, 2024

 

 

For the Nine Months Ended
September 30, 2024

 

 

(in thousands)

 

Contract manufacturing revenue

$

27,891

 

 

$

33,698

 

Royalty revenue

$

9,510

 

 

$

12,066

 

Cost of sales (inventory costs related to products sold to Roche)

$

13,725

 

 

$

15,379

 

 

There was no similar activity for the same periods of 2023.

Research and Option Agreements

The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $34.8 million in research milestone payments. Under these agreements, there are $116.8 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and royalty payments based on the net sales of the developed products upon commercialization. For the nine months ended September 30, 2023, the Company recognized $7.5 million of up-front payments related to an exercised option as research and development expense, with no similar activity for the three months ended September 30, 2023. For the three and nine months ended September 30, 2023, the Company recognized $3.8 million of research milestone expense as research and development expense. During the three and nine months ended September 30, 2024, the Company did not exercise any options, nor did any additional research milestone payments become probable of occurring.

Milestone Obligations

The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of September 30, 2024, the Company may be obligated to make up to $2.7 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within research and option agreements entered into by the Company as of September 30, 2024, which are discussed above. For the three and nine months ended September 30, 2023, the Company recognized up-front, development milestone and other expenses of $3.8 million and $11.9 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of comprehensive income (loss), with no similar activity for the three and nine months ended September 30, 2024.