Clinical Development Weekly Digest – August 2

Artificial Intelligence in Clinical Development

Breaking AI Healthcare News

Headline	Description
OpenAI GPT-5 Launch	On Aug 7, 2025, OpenAI launched GPT-5, transforming AI for drug discovery and clinical dev. [Ask your company's IT: can we run this thing on the trial data, or do legal's eyelids start twitching?]
AI Scribes Decrease Physician Burnout	Randomized trial (Nov 2024–Jan 2025): Nabla AI scribes cut time-in-note by 9.5%, improved Mini-Z burnout scores across 14 specialties.
Fauna Brain™ AI Platform	Multi-agent system scores drug targets in 2.5 minutes, for ≈$0.01/target; rapidly automates pharma R&D workflows.

Clinical Applications / Regulatory Developments

Nature Analysis: AI models in healthcare are often evaluated against flawed benchmarks, calling for more rigorous standards.
Ethical AI Distribution: Study protocol underway to ensure equitable vaccine distribution using AI in LMICs.

Gene Therapy Clinical Milestones

Regulatory & Trial Tracker

Therapy	Indication	Status / Date	Main Outcome
Zevaskyn (beremagene geperpavec-svdt)	Recessive Dystrophic EB	FDA approved Apr 2025	Wound healing, pain reduction, sustained Type VII collagen (≥2yrs).
Kebilidi (eladocagene exuparvovec-tneq)	AADC Deficiency	FDA approved Nov 2024	Motor function and quality-of-life improvement for brain-delivered gene therapy.
CAR-T (CIBMTR study)	NHL / ALL (Brazil)	Analysis 2020–2025	OS 73% (NHL), 79% (ALL), median 200-day follow-up.
Neurogene NGN-401	Rett syndrome	Phase 1/2; FDA registrational Ongoing	Fast enrollment, CGI-I & developmental milestones as endpoints.

Radioligand Theranostics Revolution

Major FDA Expansion

Therapy	New Indication	Pivotal Trial	Key Results
Pluvicto (Lu 177 vipivotide tetraxetan)	PSMA+ mCRPC (Pre-chemotherapy)	PSMAfore	ORR: 50.7% vs 14.9% rPFS: 9.3 vs 5.6 months HR: 0.41; p<0.0001

Novel Theranostic Developments

Dual-Targeting FAP + αvβ3: First-in-human trial: 88.9% had tumor shrinkage/disease stabilization (n=9).
Terbium-161 [161Tb]Tb-PSMA-I&T: Higher tumor dose, better index than lutetium-177. If you’re still shouting “Where’s my alpha?” at the scanner, here’s your hopeful headline.
Lu-177 PSMA-617 Re-treatment: Mayo data: median OS 14.5mo, ≥50% PSA decline in 45% pts, median PFS 9mo.

This Week in Perspective

AI platforms now reduce drug target identification from weeks to minutes, gene therapy is being democratized (“Baby KJ” N-of-1 models anyone?), and radioligand therapy is shifting from salvage to first-line.

Meanwhile, the FDA’s Pluvicto expansion essentially validates what you quietly told the tumor board: "Waiting for last-line to give the good stuff is so 2017." If your IR team’s coffee consumption tripled this week, you’re living the paradigm shift.

Workflow Tip: For Pluvicto trials, review PSMA-PET imaging standardization protocols—interpretation nuances are evolving fast. Coordination with nuclear medicine is officially “not optional.”
Clinical Advice: Genes and AI are nice, but remember: proper patient selection is still the art, and no, GPT-5 can’t yet join your MDT.