v3.26.1
Segment Information
 3 Months Ended
Mar. 31, 2026
Segment Reporting [Abstract]  
Segment Information Segment Information
Operating segments are defined as components of an entity for which separate financial information is available and that is regularly reviewed by the CODMs in deciding how to allocate resources and in assessing performance. The Company’s CODMs are (i) the Chief Executive Officer and (ii) the President and Chief Financial Officer. The Company is a clinical stage biotechnology company that operates as a single operating segment and has one reportable segment. In addition to reviewing the expenses in the consolidated statement of operations, the CODMs are provided with research and development costs for Rett syndrome, Batten disease and Early Discovery, as well as categorized general and administrative expenses. The research and development programs are considered significant by the Company. The CODMs assess the financial performance of the segment and decides how to allocate resources based on net loss on a consolidated basis. The measure of segment assets is reported on the condensed consolidated balance sheets as total consolidated assets. The CODMs also assess performance of the segment according to preclinical and clinical data, the stage of development of the research and development programs, along with program specific expenses and market conditions in the pharmaceutical and biotechnology sectors.
As of March 31, 2026 and 2025, the Company did not have any significant long-lived assets located outside of the United States. Information on the segment and reconciliation to net loss is as follows (in thousands):
Three Months Ended March 31,
20262025
Program specific expenses:
Rett syndrome$11,037 $4,524 
Batten disease178 845 
Early Discovery613 1,287 
Unallocated internal expenses:
Personnel-related6,456 5,404 
Stock-based compensation1,791 1,570 
Manufacturing4,197 3,107 
Other (a)
878 1,023 
Total research and development expenses$25,150 $17,760 
General and administrative specific expenses:
Personnel-related$2,626 $2,484 
Stock-based compensation1,838 2,478 
Professional and consultant fees1,402 1,277 
Office-related610 632 
Other (b)
1,723 1,288 
Total general and administrative expenses$8,199 $8,159 
Other income(c)
2,415 3,272 
Net loss$(30,934)$(22,647)
(a) The Other expense segment items category within research and development expense is mainly comprised of: Consultant fees for programs not specified above, IT Software and network support and rent expenses.
(b) The Other expense segment items category within general and administrative expense is mainly comprised of: Insurance, IT software and network support, market research, and tax expenses.
(c) Other income included in net loss is mainly comprised of: Interest income, interest expense, other income and other expense.