Segment Information

3 Months Ended

Mar. 31, 2025

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, Early Discovery and Discontinued Programs, as well as categorized general and administrative expenses. The research and development programs are considered significant by the Company. The CODM assesses 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 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, 2025 and 2024, 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,
	2025		2024
Program specific expenses:
Rett syndrome	$	4,524	$	2,019
Batten disease	845		1,190
Early Discovery	1,287		1,466
Unallocated internal expenses:
Personnel-related	5,404		4,718
Stock-based compensation	1,570		564
Manufacturing	3,107		2,979
Other (a)	1,023		605
Total research and development expenses	$	17,760	$	13,541
General and administrative specific expenses:
Personnel-related	$	2,484	$	2,101
Stock-based compensation	2,478		480
Professional and consultant fees	1,277		1,502
Office-related	632		604
Other (b)	1,288		551
Total general and administrative expenses	$	8,159	$	5,238
Other income (expense)(c)	3,272		1,858
Net loss	$	(22,647)	$	(16,921)
(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 (expense) included in net loss includes interest income, interest expense, other income and other expense.