Intellitrials: August 2025

Breaking News

Cancer vanishes in a small trial: A next‑generation, Fc‑optimized CD40 agonist antibody, injected directly into tumors, triggered impressive responses in a Phase 1 trial involving 12 patients with metastatic cancers (including melanoma, renal cell carcinoma, and breast cancer). Six patients saw tumor shrinkage even in sites that weren't injected, and two experienced complete remission—solid proof that smarter immunotherapy may be here. No major side effects were reported. The trial now aims to expand into bladder, prostate, and glioblastoma cancers. https://www.statnews.com/2025/08/14/antibody-drug-tumor-disappearance-cd40-trial/
AI in clinical trial enrollment - progress, but challenges remain: A study centered on whether an AI model could predict and flag “trial‑ready” cancer patients for clinical studies. While the AI identified those needing treatment changes effectively, it did not boost actual trial enrollment, underscoring the sticky, multifaceted barriers to participation. https://rethinkingclinicaltrials.org/news/grand-rounds-august-1-2025-clinical-trial-notifications-triggered-by-artificial-intelligence-detected-cancer-progression-kenneth-kehl/

AI as the biotech backbone
AI and machine learning are now woven into the fabric of biotech—from accelerating drug discovery to optimizing trial design and discovering new targets. https://www.sciencedirect.com/science/article/pii/S2095177925000656
RNA therapeutics continue expanding
Riding the momentum of mRNA vaccines, RNA-based therapies are gaining traction across rare diseases, oncology, and autoimmune fields.
Patient‑centric clinical trials
Decentralized models, wearables, and analytics are reshaping trial design to reduce patient burden, boost data quality, and catch safety signals earlier.
Precision medicine & gene editing move forward
Tailored treatments based on patient genetics, especially via advanced gene-editing tools, are steadily transitioning from research to clinical reality.

Papzimeos (Precigen): the first-ever drug for recurrent respiratory papillomatosis (RRP) was approved on August 15, 2025, based on trials showing a majority of patients avoided surgery for at least 12 months—a big leap for this rare HPV-driven condition. https://investors.precigen.com/news-releases/news-release-details/precigen-announces-full-fda-approval-papzimeos-zopapogene
Brinsupri (brensocatib, Insmed):
Approved August 12, 2025, this oral pill is the first and only FDA‑approved therapy for non‑cystic fibrosis bronchiectasis, delivering hope to ~350,000–500,000 U.S. patients with chronic lung symptoms. https://investor.insmed.com/2025-08-12-FDA-Approves-BRINSUPRI-TM-brensocatib-as-the-First-and-Only-Treatment-for-Non-Cystic-Fibrosis-Bronchiectasis,-a-Serious,-Chronic-Lung-Disease
Hernexeos (zongertinib, Boehringer Ingelheim):
Received accelerated approval on August 8, 2025, to treat unresectable or metastatic non‑squamous NSCLC with specific HER2 mutations. https://www.boehringer-ingelheim.com/human-health/cancer/lung-cancer/fda-approval-hernexeos-first-oral-treatment-her2-mutant-advanced-nsclc
Modeyso (dordaviprone, Jazz Pharmaceuticals):
Greenlit on August 6, 2025, to treat diffuse midline glioma (H3 K27M mutation)—a devastating brain tumor most common in children and young adults. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025

Eli Lilly & Superluminal Medicines: Signed a hefty $1.3 billion pact to leverage Superluminal’s AI‑powered platform to discover cardiometabolic and obesity drugs. Lilly gains exclusive development rights; a lead candidate targeting melanocortin 4 receptor enters human trials next year. https://www.reuters.com/business/healthcare-pharmaceuticals/lilly-signs-up-13-billion-deal-with-superluminal-develop-obesity-medicines-2025-08-14/

Schrödinger halts CDC7 inhibitor program:
After two patients died in a Phase 1 leukemia trial, Schrödinger discontinued its SGR‑2921 (CDC7 inhibitor), sending stock tumbling. https://www.fiercebiotech.com/biotech/schrodinger-abandons-cdc7-inhibitor-after-therapy-linked-2-patient-deaths
Vedanta Biosciences shutters VE202 after Phase 2 flop:
Its live‑bacteria cocktail failed in ulcerative colitis (no significant response); the company laid off ~20% of its workforce and pivoted to its Phase 3 C. difficile program (VE303). https://www.fiercebiotech.com/biotech/vedantas-live-bacteria-cocktail-fails-reduce-ulcerative-colitis-severity-phase-2-trial

Bacteria smuggling viruses into tumors: Scientists engineered bacteria to stealthily transport viruses into tumors—evading immune detection and delivering multi-pronged assault on cancer cells.

Generative AI designs new antibiotics: Using generative AI, researchers created candidates effective against drug‑resistant gonorrhea and MRSA. https://www.reuters.com/business/healthcare-pharmaceuticals/lilly-signs-up-13-billion-deal-with-superluminal-medicines-develop-obesity-2025-08-14/

Precision gene‑editing tools and more from China: A powerful, stealthy CRISPR‑like tool has been developed that edits large DNA segments accurately and without leaving traceable footprints. And China continues to power ahead in the biotech industry. https://www.nytimes.com/2025/08/17/opinion/china-biotech.html?smid=url-share

Breakthroughs	CD40 antibody induces remissions in metastatic tumors
AI	Enhancing trial design but not yet boosting enrollment
Regs Approvals	Papzimeos, Brinsupri, Hernexeos, Modeyso all greenlit
Big Deals	Lilly invests $1.3B in AI drug discovery with Superluminal
Setbacks	Schrödinger & Vedanta hit R&D roadblocks