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Heplisav-B: which tox species, and why?

hepatitis B vaccine (recombinant), adjuvanted · TLR9 (CpG 1018 adjuvant)
Species
Mouse, rat and cynomolgus monkey (repeat-dose); rabbit and baboon escalation studies
“The CpG 1018 adjuvant alone was evaluated in 3 genotoxicity studies, 2 safety escalation studies (rabbit and baboons) and 3 repeated toxicology studies (mice, rats, and cynomolgus monkeys).”
✓ VerifiedFDA summary basis for approval, p. 15Source ↗
Why that species
Adjuvanted vaccine immunogenic in mice, rats and baboons despite rodent-primate differences in TLR9 expression
“These studies demonstrated that despite some known differences in TLR9 expression pattern between rodent and primate hematopoietic cells, the 1018 ISS adjuvanted vaccine is immunogenic in all three animal species. This provides support to the choice of mice, rat and monkeys (baboons and cynomolgus monkeys) for the safety studies.”
✓ VerifiedEMA assessment report, p. 21Source ↗
Surrogate molecule
No surrogate; the clinical vaccine (HBsAg + CpG 1018) and CpG 1018 alone were tested
“Repeat-dose toxicity and reproductive and developmental toxicity studies were performed with HBsAg plus CpG 1018 and single-dose, repeat-dose and genotoxicity studies were performed with the CpG 1018 adjuvant alone.”
✓ VerifiedFDA summary basis for approval, p. 14Source ↗
Species: general tox
Mouse, rat and cynomolgus monkey
“The CpG 1018 adjuvant alone was evaluated in 3 genotoxicity studies, 2 safety escalation studies (rabbit and baboons) and 3 repeated toxicology studies (mice, rats, and cynomolgus monkeys).”
✓ VerifiedFDA summary basis for approval, p. 15Source ↗
Species: DART
Rat (fertility, embryo-fetal and pre/postnatal development in a single combined study)
“HBsAg plus CpG 1018 was also tested in rats in a reproductive and developmental toxicity study which assessed the potential effects on mating behavior, fertility, gestation, embryo-fetal development, parturition, lactation and maternal behavior”
✓ VerifiedFDA summary basis for approval, p. 15Source ↗
Species: carcinogenicity
Not conducted
“No studies assessing the carcinogenicity of Heplisav B or 1018 ISS adjuvant have been performed. This is acceptable for this type of product in line with EMAs guidelines for vaccines and adjuvants and compliant with ICH S1A, considering that the exposure to this vaccine will be sporadic.”
✓ VerifiedEMA assessment report, p. 31Source ↗
Surrogate used in
No surrogate used; vaccine and CpG adjuvant alone tested directly
“Repeat-dose toxicity and reproductive and developmental toxicity studies were performed with HBsAg plus CpG 1018 and single-dose, repeat-dose and genotoxicity studies were performed with the CpG 1018 adjuvant alone.”
✓ VerifiedFDA summary basis for approval, p. 14Source ↗

Same question, other oligonucleotides

Amondys 45

casimersen · DMD exon 45 (PMO)
Adult male mouse and cynomolgus monkey; juvenile male rat

Amvuttra

vutrisiran · TTR (siRNA)
Rat and cynomolgus monkey (monkey only pharmacologically active; rat for off-target/class tox); rabbit EFD

Cyfendus

anthrax vaccine adsorbed, adjuvanted · TLR9 (CpG 7909 adjuvant)
Rat only

Dawnzera

donidalorsen · Prekallikrein / KLKB1 (ASO)
Mouse (CD-1) and cynomolgus monkey

Defitelio

defibrotide · Fragmented DNA oligonucleotides
Rat and dog

Exondys 51

eteplirsen · DMD exon 51 (PMO)
Cynomolgus monkey and juvenile rat (eteplirsen); mdx/C57BL mouse incl. mouse surrogate

Givlaari

givosiran · ALAS1 (siRNA)
Rat and cynomolgus monkey (both pharmacologically active); rabbit inactive (EFD only)

Izervay

avacincaptad pegol · C5 (aptamer)
Rabbit and dog (IVT ocular studies); rat and cynomolgus monkey (7-day IV); monkey the only pharmacologically relevant species

Kynamro

mipomersen · APOB (ASO)
Mouse, rat and cynomolgus monkey (clinical ASO plus rodent surrogates)

Leqvio

inclisiran · PCSK9 (siRNA)
Rat and cynomolgus monkey (both pharmacologically active); rabbit for EFD

Macugen

pegaptanib · VEGF-165 (aptamer)
Rabbit and dog (pivotal IVT); rat IV for systemic; supportive monkey, mouse

Onpattro

patisiran · TTR (siRNA)
Rat and cynomolgus monkey (monkey pharmacologically active; rat off-target/LNP)

Oxlumo

lumasiran · HAO1 (siRNA)
Rat and cynomolgus monkey (both pharmacologically relevant); rabbit for EFD

Qalsody

tofersen · SOD1 (ASO)
Cynomolgus monkey (IT, relevant species), CD-1 mouse (SC) and rat single-dose IT

Qfitlia

fitusiran · Antithrombin / SERPINC1 (siRNA)
Healthy rat and cynomolgus monkey (GLP); hemophilia A mouse, hemophilia A dog and induced-HA monkey models

Redemplo

plozasiran · APOC3 (siRNA)
Rat (off-target toxicity) and cynomolgus monkey (pharmacologically relevant)

Rivfloza

nedosiran · LDHA (siRNA)
Cynomolgus monkey (relevant) plus mouse (nedosiran inactive; off-target assessment)

Rytelo

imetelstat · Telomerase (oligo)
Mouse and cynomolgus monkey (general tox); mouse and rabbit (EFD)

Spinraza

nusinersen · SMN2 (ASO)
Juvenile cynomolgus monkey (IT) and CD-1 mouse (SC, systemic effects)

Tegsedi

inotersen · TTR (ASO)
Mouse, rat and cynomolgus monkey; monkey the only pharmacologically relevant species

Tryngolza

olezarsen · APOC3 (ASO)
Mouse (with surrogate) and cynomolgus monkey

Viltepso

viltolarsen · DMD exon 53 (PMO)
Male CD-1 mouse and male cynomolgus monkey (plus juvenile male mouse)

Vitravene

fomivirsen · CMV IE2 mRNA (ASO)
Rabbit and monkey

Vyondys 53

golodirsen · DMD exon 53 (PMO)
Mouse, cynomolgus monkey (adult males) and juvenile rat

Wainua

eplontersen · TTR (ASO)
Mouse and cynomolgus monkey (monkey the pharmacologically relevant species)

Waylivra

volanesorsen · APOC3 (ASO)
Mouse, rat and cynomolgus monkey (rabbit for embryo-fetal); monkey most relevant

Zanvastro

zilganersen · GFAP (ASO)
Mouse (SC and ICV, juvenile) and juvenile monkey (intrathecal); mouse surrogate ASO arm

Summary across all 28 oligonucleotides →