1Q26 Update. Commercial Details the Focus of the Call. 4th Year OLE Data Continue to Be Supportive. P3 Data in Mid-27. NDA Rolling 1Q27.

研报英文原文证据摘录

1Q26 Update. Commercial Details the Focus of the Call. 4th Year OLE Data Continue to Be Supportive. P3 Data in Mid-27. NDA Rolling 1Q27.

■ Take 002: the first patient has been dosed in OSPREY, a Phase 1, dose-escalating, open-label study of STK-002 in patients

with autosomal dominant optic atrophy (ADOA), marking the formal clinical start for the company’s second TANGO-based

program. STK-002, an antisense oligonucleotide designed to upregulate OPA1 from the wild-type allele (good mRNA already

made in the cell at about 50% of normal levels), has orphan drug designation and is positioned as a potential first disease-

modifying therapy for ADOA, a progressive, irreversible optic neuropathy with no approved treatments. Management has

indicated that decisions from OSPREY will be guided primarily by safety, tolerability, and exposure data across dose levels,

with secondary assessments of visual function, ocular structure, and quality of life important for dose selection. Importantly,

management emphasized that early improvements in visual function could be particularly meaningful—potentially de-risking

the program and shortening the duration of future studies. In addition, ADOA is a disease of low energy production andBIOTECHNOLOGY reasonable validated assays exist to directly monitor energy levels in the eye for very early reads on POC. Based on the totality

of data, management will assess whether STK-002's profile supports advancement to chronic dosing and larger studies.

■ Mechanistically straightforward. Mechanistically, STK-002 is designed to correct OPA1 haploinsufficiency (one good, one

bad gene) through Stoke’s TANGO platform to remove the toxic exon from non-productive OPA1 transcripts - regeneratingEQUITY productive mRNA and increasing protein expression.