Request an invite
Atlas/Oligonucleotides/Macugen/First dose

Macugen: how was the first human dose set?

pegaptanib · VEGF-165 (aptamer)
Check my plan vs. these
First-in-human starting dose
Not stated in public reviews
NOAEL / key findings
NOAEL 3 mg/eye dog, 2 mg/eye rabbit; rat IV NOAEL 1 mg/kg/day (nephropathy, PEG vacuolated macrophages)
“In the peer-review, additional injection-related, but no drug-related findings were observed and a NOAEL of 3 mg/eye was confirmed.”
✓ VerifiedEMA assessment report, p. 10Source ↗

Same question, other oligonucleotides

Kynamro

mipomersen · APOB (ASO)
Applicant estimated safe starting dose from nonclinical results; method not specified

Zanvastro

zilganersen · GFAP (ASO)
Starting-dose rationale not stated; FDA capped clinical IT dose at 25 mg (~1/10 mouse/monkey NOAEL), later 50 mg Q12W

Amondys 45

casimersen · DMD exon 45 (PMO)
Not stated in public reviews

Amvuttra

vutrisiran · TTR (siRNA)
Not stated in public reviews

Dawnzera

donidalorsen · Prekallikrein / KLKB1 (ASO)
Not stated in public reviews

Exondys 51

eteplirsen · DMD exon 51 (PMO)
Not stated in public reviews

Givlaari

givosiran · ALAS1 (siRNA)
Not stated in public reviews

Izervay

avacincaptad pegol · C5 (aptamer)
Not stated in public reviews

Leqvio

inclisiran · PCSK9 (siRNA)
Not stated in public reviews

Onpattro

patisiran · TTR (siRNA)
Not stated in public reviews

Oxlumo

lumasiran · HAO1 (siRNA)
Not stated in public reviews

Qalsody

tofersen · SOD1 (ASO)
Not stated in public reviews

Qfitlia

fitusiran · Antithrombin / SERPINC1 (siRNA)
Not stated in public reviews

Redemplo

plozasiran · APOC3 (siRNA)
Not stated in public reviews

Rivfloza

nedosiran · LDHA (siRNA)
Not stated in public reviews

Rytelo

imetelstat · Telomerase (oligo)
Not stated in public reviews

Spinraza

nusinersen · SMN2 (ASO)
Not stated in public reviews

Tegsedi

inotersen · TTR (ASO)
Not stated in public reviews

Tryngolza

olezarsen · APOC3 (ASO)
Not stated in public reviews

Viltepso

viltolarsen · DMD exon 53 (PMO)
Not stated in public reviews

Vyondys 53

golodirsen · DMD exon 53 (PMO)
Not stated in public reviews

Wainua

eplontersen · TTR (ASO)
Not stated in public reviews

Waylivra

volanesorsen · APOC3 (ASO)
Not stated in public reviews

Summary across all 24 oligonucleotides →