Neuroprotection Peptides

A brain-targeting tetrapeptide studied for neuroprotection and targeted drug delivery to injured brain and spinal cord tissue.

A porcine brain-derived peptide mixture approved in some countries for stroke recovery and neurodegenerative conditions.

An eight-amino-acid ADNP-derived peptide studied for microtubule stabilization, tau-related neuroprotection, and rare ADNP syndrome.

A hexapeptide analog studied for cognitive enhancement via hepatocyte growth factor pathway activation.

An approved, once-monthly, self-administered subcutaneous GalNAc-conjugated antisense oligonucleotide (ASO) from Ionis and AstraZeneca that treats transthyretin-mediated (ATTR) amyloidosis by lowering the liver's production of transthyretin (TTR). Transthyretin is a liver-made transport protein that can misfold and deposit as amyloid in nerves and the heart; eplontersen is a short synthetic strand of chemically modified DNA/RNA that binds TTR messenger RNA and directs its enzymatic (RNase H1) degradation before the protein is made, cutting circulating TTR by roughly 80%. A triantennary N-acetylgalactosamine (GalNAc) tag delivers it to liver cells, allowing a low-dose 45 mg injection just once a month via autoinjector or pre-filled syringe. Marketed as Wainua (US) and Wainzua (EU), it was FDA-approved in December 2023 for the polyneuropathy of hereditary ATTR amyloidosis (ATTRv-PN) on the strength of the NEURO-TTRansform trial, and is now approved in more than 20 countries. Eplontersen is the antisense (ASO) counterpart to the siRNA drug vutrisiran (Amvuttra): both silence TTR, and both were tested in ATTR cardiomyopathy - but where vutrisiran's HELIOS-B trial succeeded on top of a stabilizer background, eplontersen's much larger CARDIO-TTRansform cardiomyopathy trial missed its primary endpoint in July 2026, with a benefit seen only in the prespecified monotherapy subgroup. It is a GalNAc-ASO sibling of Ionis's olezarsen and pelacarsen and the second-generation successor to the earlier, non-GalNAc TTR antisense drug inotersen (Tegsedi).

An endogenous mitochondrial-derived peptide studied for neuroprotection, cellular stress resistance, and longevity, whose blood levels fall with age and are elevated in centenarians.

A naturally occurring longevity hormone (secreted alpha-klotho) being studied for cognition and brain aging; a single low-dose injection improved memory in aged monkeys, and a first-in-human Phase 1 trial in older adults is underway.

A coenzyme essential for cellular energy production, studied for anti-aging and metabolic support.

An 11-amino-acid CNTF-derived peptide studied for its ability to upregulate BDNF expression and promote neurogenesis in cognitive decline models.

A short bioregulatory peptide studied for neuroprotective effects and cognitive support in aging.

A synthetic peptide analog of tuftsin studied for anxiolytic and nootropic properties.

A GLP-1 receptor agonist FDA-approved for type 2 diabetes and chronic weight management.

A synthetic peptide derived from ACTH, studied for cognitive enhancement and neuroprotective effects.

A mitochondria-targeted peptide FDA-approved as FORZINITY (elamipretide) for Barth syndrome in September 2025 — the first FDA-approved mitochondrial-targeted therapeutic.

A twice-daily oral peptide analog of the IGF-1 tripeptide glycine-proline-glutamate (GPE) that is the first and only medicine approved to treat Rett syndrome. Marketed by Acadia Pharmaceuticals as Daybue, trofinetide does not correct the underlying MECP2 mutation; instead it works on the downstream consequences - it is thought to restore synaptic signaling, dampen neuroinflammation, and normalize overactive microglia and astrocytes, improving neurobehavioral symptoms. It won FDA approval in March 2023 for adults and children 2 years and older. In late 2025 the FDA cleared a dye- and preservative-free powder formulation (Daybue STIX, broadly available in April 2026), and in June 2026 the EMA's CHMP adopted a positive opinion recommending European authorization.

An investigational, brain-penetrant anti-amyloid antibody from Roche/Genentech that uses the proprietary 'Brainshuttle' transferrin-receptor delivery system to reach the brain far more efficiently than conventional antibodies. Trontinemab (development codes RG6102 / RO7126209) is a 2+1 bispecific molecule: it fuses the amyloid-beta-clearing antibody gantenerumab to a fragment that grabs transferrin receptor 1 (TfR1) on blood-brain-barrier cells, hitching a ride into the brain via the same receptor-mediated transport that carries iron. That shuttle lets a low intravenous dose clear amyloid plaques rapidly and deeply while triggering strikingly little of the brain swelling and micro-bleeding (ARIA) that limits approved anti-amyloid drugs. In the Phase Ib/IIa Brainshuttle AD study (NCT04639050), the 3.6 mg/kg dose removed roughly 107 centiloids of amyloid after 28 weeks, driving about 91-92% of participants below the amyloid-positivity threshold (24 centiloids) - with ARIA-E seen in fewer than 5% of participants, well below the ~13% reported for lecanemab and ~24% for donanemab. On the strength of those data, Roche launched two identical pivotal Phase 3 trials - TRONTIER 1 and TRONTIER 2 - in early symptomatic Alzheimer's disease (about 1,600 patients across 18 countries, begun in 2025), and at the 2026 Alzheimer's Association International Conference (AAIC) in London it unveiled PrevenTRON, a Phase 3 prevention trial in 1,600 cognitively unimpaired people at high risk (elevated plasma p-tau217). Trontinemab is a prescription-stage investigational biologic - it is not approved anywhere and is not a supplement or research chemical.

A neuropeptide with broad neuroimmune functions, studied for inflammatory conditions and nerve repair.

An approved, quarterly (once-every-three-months) subcutaneous GalNAc-conjugated small interfering RNA (siRNA) from Alnylam that treats transthyretin-mediated (ATTR) amyloidosis by silencing the TTR gene in the liver. Transthyretin is a liver-made transport protein that can misfold and pile up as amyloid deposits in nerves and, critically, the heart; vutrisiran uses RNA interference (RNAi) to degrade both mutant and wild-type TTR messenger RNA before the protein is made, lowering circulating TTR by roughly 80% and starving the amyloid of its raw material. Marketed as Amvuttra, it was first FDA-approved in June 2022 for the polyneuropathy of hereditary ATTR amyloidosis (HELIOS-A), and in March 2025 the FDA expanded the label to ATTR cardiomyopathy (ATTR-CM) of wild-type or hereditary disease - making vutrisiran the first RNAi therapeutic shown to reduce cardiovascular death and cardiovascular events, on the strength of the HELIOS-B outcomes trial. It shares the exact GalNAc-siRNA delivery platform that underlies the cardiometabolic RNAi medicine inclisiran and the investigational agents olpasiran, lepodisiran, zerlasiran, zilebesiran, zodasiran, solbinsiran and plozasiran, and it is the direct successor to Alnylam's earlier intravenous TTR siRNA patisiran (Onpattro).

An investigational next-generation RNA interference (RNAi) therapeutic from Alnylam Pharmaceuticals designed to silence the transthyretin (TTR) gene and treat transthyretin amyloidosis (ATTR) - the same disease targeted by Alnylam's approved drugs patisiran and vutrisiran (Amvuttra) and by Ionis/AstraZeneca's eplontersen (Wainua/Wainzua). Nucresiran (formerly ALN-TTRsc04) is a subcutaneous GalNAc-conjugated small interfering RNA (siRNA) built on Alnylam's newer IKARIA platform, engineered for deeper, more durable knockdown of both mutant and wild-type TTR with the potential for once- or twice-yearly dosing - a substantial step beyond the current quarterly (vutrisiran) or monthly (eplontersen) schedules. In an interim Phase 1 single-ascending-dose study in 48 healthy volunteers (presented at AHA 2024), a single dose of 300 mg or higher rapidly lowered serum TTR by more than 90% by Day 15 and by more than 96% at peak (Day 29), with more than 70% reduction still present at one year after a single 300 mg dose and low patient-to-patient variability; all doses were well tolerated with no injection-site reactions and no liver safety signals. Alnylam has moved nucresiran into a Phase 3 TRITON program: TRITON-PN (NCT07223203) in hereditary ATTR with polyneuropathy, an open-label study using vutrisiran as an active comparator, and TRITON-CM (NCT07052903), a large event-driven cardiovascular outcomes trial in ATTR cardiomyopathy dosing nucresiran 300 mg subcutaneously once every six months. Nucresiran is investigational and not approved by the FDA, EMA or any regulator; it is studied only in clinical trials and is not a supplement or research chemical.

Remternetug (development code LY3372993) is an investigational IgG1 monoclonal antibody from Eli Lilly that binds a pyroglutamate-modified form of amyloid-beta (N3pG-Aβ) found almost exclusively in the aggregated plaques of Alzheimer's disease. It is the follow-on to donanemab (Kisunla), Lilly's FDA-approved anti-amyloid antibody that hits the same target, and is designed to clear existing brain amyloid rapidly and, distinctively, to be delivered by a short subcutaneous injection that patients or care partners can give at home rather than only by intravenous infusion in a clinic. In an early-phase study three-quarters of treated participants reached the amyloid-clearance threshold within about six months, and remternetug is now in Phase 3 across symptomatic early Alzheimer's (TRAILRUNNER-ALZ 1), a large prevention program (TRAILRUNNER-ALZ 3), and inherited-Alzheimer prevention through the DIAN-TU network. It is not approved by the FDA or any regulator for any use.