Evidence due to arrive
Two short lists: readouts about to drop, and compounds just entering human testing. The complement to Rankings — rankings is the current consensus; this page is what could change it, and roughly when. For what would matter most if it worked, on no timetable at all, see Frontier.
15 entries across 2 sections.
Specific trial or regulatory events expected in roughly the next 6 months that would meaningfully change how we score the compound.
8 entries
Stage / status: Investigational; CagriSema NDA filed with FDA Dec 18, 2025 (decision expected ~Oct 2026)
REDEFINE 1 (Garvey NEJM 2025): CagriSema 2.4mg/2.4mg achieved -20.4% body weight at 68 weeks vs -3.0% placebo (n=3417, no T2D). Notably below the ~25% projected from Phase 2 — Novo stock fell at the December 2024 readout. Cagrilintide alone -11.5%. REDEFINE 2 (T2D, n=1206): -13.7% vs -3.4% placebo at 68 weeks. REDEFINE…
Stage / status: Investigational (pelacarsen Lp(a)HORIZON CVOT **completed 16 Jul 2026** — topline awaited; olpasiran OCEAN(a) Outcomes ongoing)
Until 2025 Lp(a) was a non-modifiable risk factor — the entire class is a major shift. Network meta-analyses (Wu Pharmacol Res 2026; Hu Front Cardiovasc Med 2026) confirm olpasiran most potent. Muvalaplin is uniquely oral and would be the first non-injectable in the class. Concomitant PCSK9i further enhances LDL reduct…
Stage / status: Investigational (NewAmsterdam/Menarini; EMA marketing-authorization applications accepted for obicetrapib and obicetrapib/ezetimibe in primary hypercholesterolaemia, EU decision expected 2H2026; PREVAIL CVOT readout expected 2026-2027)
Revival of CETP inhibition after the torcetrapib (BP/mortality) and dalcetrapib (futile) failures. Obicetrapib's hydrophilic structure avoids torcetrapib's off-target effects. BROADWAY trial: -32% LDL on top of max statin. Triple combo (obicetrapib + ezetimibe + statin) ranks highest in 2026 network meta for LDL/ApoB r…
Stage / status: Rx (olezarsen approved US/EU for FCS as Tryngolza); severe HTG under FDA review
Olezarsen (Ionis/Sobi) approved in US and EU for familial chylomicronemia syndrome. sNDA for severe hypertriglyceridemia under FDA Priority Review, PDUFA June 30 2026: pooled CORE/CORE2 Phase 3 showed triglycerides -66% and acute pancreatitis events -85%. Plozasiran (Arrowhead) siRNA in Phase 3 (SHASTA) for the same po…
Stage / status: Investigational (Phase 3; FDA submission guided to Q1 2027)
Eli Lilly TRIUMPH-1 (May 21, 2026 topline): first Phase 3 readout in obesity, n=2,339 adults with obesity + ≥1 comorbidity (HTN/dyslipidemia/OSA/OA) but no diabetes, randomized 1:1:1:1 to retatrutide 4/9/12 mg or placebo SC weekly, 80-week primary with 104-week extension. Mean weight loss: 19.0% / 25.9% / 28.3% vs 2.2%…
Stage / status: Investigational (Phase 3)
Boehringer Ingelheim/Zealand Pharma. SYNCHRONIZE-1 (no T2D) Phase 3 full results published in NEJM (Jun 2026, ADA Scientific Sessions): -16.6% mean body weight at 76 weeks vs -3.2% placebo (efficacy estimand), 85% achieving >=5% loss, loss driven predominantly by fat mass. SYNCHRONIZE-MASLD (Nature Medicine, Jun 8 2026…
Stage / status: Phase 1 in healthy volunteers, Australia — first participant dosed December 2025. Not registered on ClinicalTrials.gov, so status surfaces through company channels rather than the registry · Retro Biosciences
An oral small molecule meant to restore lysosomal function — the disposal end of autophagy, which is one of the few aging mechanisms where the biology is well enough understood to drug directly. Retro is the best-funded of the private longevity companies and this is its first asset in humans, so the readout doubles as a test of whether the whole partial-reprogramming-and-autophagy investment thesis produces anything clinical. The indication is Alzheimer's-related biology rather than aging itself, because that is what a regulator will accept as an endpoint.
Next milestone: First human data. The company has guided to 'early data around August 2026' since June and that window is now open with nothing published, so this is overdue rather than pending. A Phase 1 in healthy volunteers can only report safety, tolerability and biomarkers — there is no efficacy endpoint to hit.
Stage / status: Phase 2 QUELL-CV, recruiting (NCT07656727) — first participant dosed June 2026. Phase 1 reported April 2026 · BioAge Labs
A brain-penetrant, orally available NLRP3 inflammasome inhibitor, aimed at the inflammatory arm of aging rather than at weight or lipids directly. NLRP3 is the clearest druggable node in 'inflammaging' — the low-grade chronic inflammation that tracks with almost every age-related disease — and this is one of the first times that target has reached Phase 2 in people selected for cardiovascular risk rather than for an autoimmune diagnosis. Phase 1 was unusually striking: median hsCRP fell roughly 85% at 60 mg once daily over 21 days and 83-86% at 120 mg over 14 days, with no serious adverse events. hsCRP is an inflammation marker, not an outcome, which is exactly what Phase 2 exists to test.
Next milestone: Topline from the Phase 2 QUELL-CV dose-ranging trial (n=160 estimated, 12 weeks, placebo-controlled, obesity plus cardiovascular risk factors), guided to the second half of 2026 with registry completion listed December 2026. The question is whether an ~85% drop in an inflammation marker produces anything a patient would notice.
Genuinely new interventions with little human data yet — first-in-human trials, novel mechanisms, preclinical-to-Phase-1. Listed here because their evidence base is about to exist, not because we judge them promising. Provisional scores; ones to watch, not recommendations.
7 entries
Stage / status: Investigational (Phase 2a planning); no off-label or compounding pathway
AlzeCure Pharma (Sweden) lead clinical asset. Novel mechanism — small-molecule pan-Trk allosteric kinase potentiator. Mechanistically distinct from anti-amyloid mAbs and cholinesterase inhibitors — directly relevant given the Cochrane 2026 finding that the entire anti-amyloid class produces only trivial cognitive benef…
Stage / status: Investigational (repurposing; ARPA-H PROSPR-funded)
Transposon Therapeutics. ARPA-H PROSPR award (up to $22M) funds a ~200-participant 48-week trial in healthy adults aged 60-65 testing whether suppressing LINE-1 retrotransposons improves mobility, cognition and vitality (the Gorbunova/Sedivy inflammaging hypothesis). Existing safety from PSP/ALS-FTD Phase 2 trials de-r…
Stage / status: Investigational (Phase 1; first patient dosed Jun 9 2026)
Life Biosciences (co-founded by David Sinclair). First partial-reprogramming therapy dosed in a human: FDA cleared the IND Jan 2026 and the first Phase 1 patient was dosed Jun 9 2026 — believed to be the world's first in-human test of partial epigenetic reprogramming. Phase 1 (NCT07290244) in non-arteritic anterior isc…
Stage / status: Investigational (Phase 1 SAD/MAD)
Lysoway Therapeutics. First TRPML1 agonist in the clinic, first participant dosed May 2026 in a Phase 1 SAD/MAD study (Melbourne) in healthy volunteers, developed for Alzheimer's/Parkinson's. A clean safety and target-engagement readout would be early human proof that pharmacologically boosting autophagy works. No publ…
Stage / status: Investigational (Phase 1b/2; not approved)
Verve Therapeutics (Lilly). Heart-2 Phase 1b (NEJM 2026, n=35): a single infusion reduced PCSK9 up to 88% and LDL-C up to 62% (max -69%), durable to 18 months, no serious adverse events. First-in-human in-vivo base editing for hypercholesterolemia; Phase 2 planned late 2026. Frontier modality, durability/safety still e…
Stage / status: Research/clinical setting
Conboy lab 2020 showed neutral saline + albumin exchange had rejuvenating effects similar to young plasma — pointing to a dilution mechanism, not 'youth factors.' FDA 2019 warned against Ambrosia-style young-plasma transfusions for anti-aging. New human data: a single-blinded, placebo-controlled RCT (Fuentealba/Kiprov,…
Stage / status: Investigational (EU Phase 1 complete; US Phase 1b/2a in actinic keratosis ongoing — NCT07340697, completion listed 2026)
The senolytic idea finally reaching human skin with a randomized, vehicle-controlled design. Rubedo reported preliminary Phase 1 results in March 2026 from a single-centre, ascending-dose, randomized, double-blind, vehicle-controlled EU trial across three populations — plaque psoriasis, atopic dermatitis, and photo-age…