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LICENSE AND COLLABORATION AGREEMENTS | 12 Months Ended
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Dec. 31, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract] |
LICENSE AND COLLABORATION AGREEMENTS | LICENSE AND COLLABORATION AGREEMENTS In October 2019, the Company entered into a worldwide, exclusive licensing agreement with a third party for tralesinidase alfa (formerly referred to as BMN 250), an investigational enzyme replacement therapy to treat Sanfilippo Syndrome Type B. In consideration, the Company received an upfront payment of $3.0 million, a minority 15% equity ownership interest in the licensee, and is entitled to receive royalties on net sales of tralesinidase alfa and milestone payments if certain development, regulatory and sales milestones are met by the licensee. Subsequently, the third party licensee raised additional funding and issued the Company incremental shares to maintain its 15% minority interest. As of December 31, 2022, the balance of the equity investment included in Other Assets on the Company’s Consolidated Balance Sheets was $12.6 million, which was fully impaired in 2023 based on new developments that lead the Company to conclude that factors existed indicating this non-marketable strategic investment was no longer realizable. The loss on the equity investment due to impairment was recorded to Other Income (Expense), Net on the Company’s Consolidated Statements of Operations. In July 2017, the Company executed a license agreement with Sarepta Therapeutics (Sarepta) that provides Sarepta with global exclusive rights to the Company’s Duchenne muscular dystrophy (DMD) patent estate for EXONDYS 51 and all future exon-skipping products. Under the license agreement, Sarepta pays the Company royalties and may pay the Company certain milestone payments for exons 51, 45, 53 and possibly other exon-skipping products. In 2021, the Company and Sarepta amended the license agreement to, among other things, make the license co-exclusive at a future date and reduce future royalty rates. On October 1, 2015, the Company entered into an agreement with Ares Trading S.A. (Merck Serono) under which the Company acquired all global rights to KUVAN and PALYNZIQ from Merck Serono, with the exception of KUVAN in Japan. Previously, the Company had exclusive rights to KUVAN in the U.S. and Canada and PALYNZIQ in the U.S. and Japan. Pursuant to the amended and restated KUVAN Agreement, if future sales milestones were met, the Company was obligated to pay Merck Serono up to a maximum of €60.0 million, all of which were met and paid as of December 31, 2023. Pursuant to the Pegvaliase Agreement, the Company also paid Merck Serono €125.0 million in cash when the PALYNZIQ development milestones were achieved. In October 2012, the Company licensed to Catalyst Pharmaceutical Partners, Inc. (Catalyst) the North American rights to develop and market FIRDAPSE, the Company's former commercial product for the treatment of Lambert-Eaton myasthenic syndrome. In exchange for the North American rights to FIRDAPSE, commencing in the first quarter of 2019 the Company receives royalties of 7% to 10% on net product sales of FIRDAPSE in North America. The Company is engaged in R&D collaborations with various other entities. These provide for sponsorship of R&D by the Company and may also provide for exclusive royalty-bearing intellectual property licenses or rights of first negotiation regarding licenses to intellectual property development under the collaborations. Typically, these agreements can be terminated for cause by either party upon written notice. In 2020, the Company entered into a research and collaboration agreement with a third party and received a convertible note, which was recorded in Other Assets on the Company's Consolidated Balance Sheets. In 2023, the Company recorded a $11.9 million impairment loss on the convertible note as it was deemed unrecoverable based on new information. The impairment loss was recorded to Other Income (Expense), Net on the Company's Consolidated Statements of Operations.

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