GLOBAL RESEARCH ARCHIVE
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.
Research evidence excerpt
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.
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