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Intangible assets and goodwill | 6 Months Ended
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Jun. 30, 2025
Goodwill and Intangible Assets Disclosure [Abstract] |
Intangible assets and goodwill | 12. Intangible assets and goodwill Definite-lived intangibles Definite-lived intangible assets consisted of the following at June 30, 2025 and December 31, 2024: ​
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | ​ | Ending Balance at | ​ | ​ | ​ | ​ | ​ | Foreign | ​ | ​ | Ending Balance at
Definite-lived | ​ | ​ | December 31, | ​ | ​ | ​ | ​ | ​ | currency | ​ | ​ | June 30,
intangible assets, gross |  | ​ | 2024 |  | ​ | Additions |  | ​ | translation | ​ | ​ | 2025
Waylivra | ​ | ​ | 12,397 | ​ | ​ | 3,293 | ​ | ​ | 1,705 | ​ | ​ | 17,395
Tegsedi | ​ | ​ | 18,249 | ​ | ​ | 2,021 | ​ | ​ | 2,361 | ​ | ​ | 22,631
Kebilidi | ​ | ​ | 10,731 | ​ | ​ | — | ​ | ​ | — | ​ | ​ | 10,731
Upstaza | ​ | ​ | 106,937 | ​ | ​ | — | ​ | ​ | — | ​ | ​ | 106,937
Sephience | ​ | ​ | — | ​ | ​ | 25,000 | ​ | ​ | — | ​ | ​ | 25,000
Total definite-lived intangibles, gross | ​ | $ | 148,314 | ​ | $ | 30,314 | ​ | $ | 4,066 | ​ | $ | 182,694
​
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | ​ | Ending Balance at | ​ | ​ | ​ | ​ | ​ | Foreign | ​ | ​ | Ending Balance at
Definite-lived | ​ | ​ | December 31, | ​ | ​ | ​ | ​ | ​ | currency | ​ | ​ | June 30,
intangible assets, accumulated amortization |  | ​ | 2024 |  | ​ | Amortization |  | ​ | translation |  | ​ | 2025
Waylivra | ​ | ​ | (5,273) | ​ | ​ | (1,187) | ​ | ​ | (720) | ​ | ​ | (7,180)
Tegsedi | ​ | ​ | (5,609) | ​ | ​ | (1,735) | ​ | ​ | (789) | ​ | ​ | (8,133)
Kebilidi | ​ | ​ | (112) | ​ | ​ | (447) | ​ | ​ | — | ​ | ​ | (559)
Upstaza | ​ | ​ | (18,526) | ​ | ​ | (4,456) | ​ | ​ | — | ​ | ​ | (22,982)
Sephience | ​ | ​ | — | ​ | ​ | (34) | ​ | ​ | — | ​ | ​ | (34)
Total definite-lived intangibles, accumulated amortization | ​ | $ | (29,520) | ​ | $ | (7,859) | ​ | $ | (1,509) | ​ | $ | (38,888)
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
Total definite-lived intangibles, net | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | $ | 143,806
​ Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets. For the six months ended June 30, 2025, royalty payments of $2.0 million and $3.3 million were recorded for Tegsedi and Waylivra, respectively. As of June 30, 2025, a royalty payable of $0.9 million and $1.4 million for Tegsedi and Waylivra, respectively, was recorded on the consolidated balance sheet within accounts payable and accrued expenses. Pursuant to the Censa Merger Agreement, in June 2025, a $25.0 million milestone from the Company to the former Censa securityholders was triggered when the EC granted marketing authorization to Sephience for the treatment of children and adults living with PKU. The milestone was recorded as an intangible asset and is being amortized to cost of product sales over its expected useful life on a straight-line basis. As of June 30, 2025, the $25.0 million milestone was recorded on the consolidated balance sheet within accounts payable and accrued expenses. ​ The former Censa securityholders may also be entitled to receive other contingent payments subject to certain terms set forth in the Censa Merger Agreement related to sales of Sephience. In accordance with the guidance for an asset acquisition, the Company will record such payments when they become payable to the former Censa securityholders and increase the cost basis for the Sephience intangible asset. For the three months ended June 30, 2025 and 2024, the Company recognized amortization expense of $4.1 million and $2.9 million, respectively, related to the Upstaza/Kebilidi, Sephience, Waylivra, and Tegsedi intangible assets. For the six months ended June 30, 2025 and 2024, the Company recognized amortization expense of $7.9 million and $54.4 million, respectively, related to the Emflaza rights, Upstaza/Kebilidi, Sephience, Waylivra, and Tegsedi intangible assets. The estimated future amortization of the Upstaza/Kebilidi, Sephience, Waylivra, and Tegsedi intangible assets is expected to be as follows: ​
​ | ​ | ​ | ​
​ |  | As of June 30, 2025
2025 | ​ | $ | 9,382
2026 | ​ |  | 18,751
2027 | ​ |  | 18,751
2028 | ​ |  | 18,751
2029 and thereafter | ​ |  | 78,171
Total | ​ | $ | 143,806
​ The weighted average remaining amortization period of the definite-lived intangibles as of June 30, 2025 is 8.8 years. Goodwill As a result of the Agilis Merger on August 23, 2018, the Company recorded $82.3 million of goodwill. As of June 30, 2025, there have been no changes to the balance of goodwill since the date of the Agilis Merger. Accordingly, the goodwill balance as of June 30, 2025 is $82.3 million.

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- Definition The entire disclosure for goodwill and intangible assets. + References Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef -Topic 350 -SubTopic 30 -Name Accounting Standards Codification -Publisher FASB -URI https://asc.fasb.org/350-30/tableOfContent Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef -Topic 350 -SubTopic 20 -Name Accounting Standards Codification -Publisher FASB -URI https://asc.fasb.org/350-20/tableOfContent + Details
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