Editorial watch list
Two short lists: readouts about to drop, and the genuinely new science worth watching. The complement to Rankings — rankings is the current consensus; this page is what could change it.
14 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 (FDA NDA accepted, Priority Review; PDUFA July 24 2026 — children/adolescents/adults)
Otsuka's bid for a non-stimulant 'next Adderall' — it hits the same dopamine/norepinephrine targets without being a scheduled drug. Backed by four pivotal Phase 3 trials across adults, adolescents and children (positive topline 2024-2025); 2026 post-hoc analyses (n=744 adults) showed gains in self-reported executive fu…
Stage / status: Investigational (pelacarsen Lp(a)HORIZON CVOT readout expected 2026; 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 (FDA NDA accepted, Priority Review; PDUFA Q3 2026 — narcolepsy type 1)
The wakefulness frontier's biggest swing — Takeda's orexin agonist restores the exact signal narcolepsy patients lack instead of stimulating broadly like amphetamine or modafinil. Phase 3 FirstLight and RadiantLight (NT1, ~270+ patients) met all primary and secondary endpoints, with ~80% reduction in weekly cataplexy a…
Stage / status: Investigational (Phase 3)
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…
Genuinely new interventions with little human data yet — first-in-human trials, novel mechanisms, preclinical-to-Phase-1. High discovery value, provisional scores; ones to watch, not recommendations.
6 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,…