Clinical Trial News 20-Nov-2025

FDA Drug Approvals 

• Hyrnuo (sevabertinib) : Kinase inhibitor approved 19-Nov-2025 for the treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy. NDA #219947.  Bayer Healthcare Pharms  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=219972

• Komzifti (ziftomenib): Priority approval granted 13-Nov-2025 to treat adults with relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 mutation who have no satisfactory alternative treatment options.  NDA #220305. Kura Oncology, Inc. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=220305

• Redemplo (plozasiran): Approved 18-Nov-2025 for familial chylomicronemia syndrome. RNA interference therapeutic to reduce triglycerides. NDA #219947. Arrowhead Pharmaceuticals, Inc. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=219947/

• Kygevvi (doxecitine and doxribtimine) - Approved 03-Nov-2025. for treatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset on or before 12 years.  NDA #219792. UCB Inc. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=219792

• Daratumumab and hyaluronidase-fihj: Approved 06-Nov-2025 for high-risk smoldering multiple myeloma

M&A Deals

Major Acquisitions:

• Merck acquires Cidara Therapeutics: $9.2 billion (announced November 14). CD388, a long-acting antiviral for influenza prevention currently in Phase 3. First participant dosed September 2025, targeting 6,000 enrollment FDA. CD388 provided 76% protection from symptomatic influenza over 24 weeks in mid-stage trial. Not a vaccine, works regardless of immune status. Received FDA Breakthrough Designation in October 2025. Expected close Q1 2026. https://www.merck.com/news/

• Johnson & Johnson acquires Halda Therapeutics: $3.05 billion in cash (announced November 17). HLD-0915 oral therapy for metastatic castration-resistant prostate cancer using proprietary RIPTAC platform. First-in-class RIPTAC (Regulated Induced Proximity Targeting Chimera) designed to overcome treatment resistance. Phase 1/2 data showed 59% achieved PSA50 response in heavily pretreated patient. J&J's second major deal this year after $14.6B Intra-Cellular acquisition. https://www.jnj.com/media-center/press-releases/

• Johnson & Johnson acquires Intra-Cellular Therapies: $14.6 billion, acquiring Caplyta for schizophrenia and bipolar depression (Source)

• Novartis acquires Avidity Biosciences: Approximately $12 billion acquisition for RNA-based therapies

• Merck & Co. acquires Verona Pharma - $10 billion for Ohtuvayre (ensifentrine), a COPD treatment approved in June 2024.  https://www.merck.com/news

• Thermo Fisher acquires Clario - Up to $9.4 billion for clinical-trial endpoint and data-analytics capabilities

• Sanofi acquires Blueprint Medicines - $9.5 billion for rare disease portfolio including Ayvakit/Ayvakyt

• Endo and Mallinckrodt merger: $6.7 billion specialty pharma deal scheduled to close in 2025

• Novo Nordisk acquires Akero Therapeutics - Up to $5.2 billion (October 9) for efruxifermin, a fibroblast growth factor 21 analog for compensated cirrhosis

• Merck KGaA acquires SpringWorks Therapeutics: $3.9 billion for rare cancer therapies including Ogsiveo for desmoid tumors

• AztraZeneca acquires eSoBiotec: AstraZeneca closed near $1 billion acquisition of eSoBiotec for next-generation cell therapy manufacturing. Novartis' $12 billion Avidity deal signals confidence in RNA-based precision delivery

• Pfizer acquires Metsera: Pfizer sealed $10 billion acquisition of Metsera after bidding war with Novo Nordisk. Eli Lilly inked $1.3 million deal with Superliminal for AI/ML-generated obesity medications. These acquisitions highlight the continued growth of the obesity and GLP-1 space.

Clinical Trial Setbacks

• Sarepta Therapeutics - Duchenne muscular dystrophy confirmatory trials failed (November 4). Long-delayed trials for two drugs showed no patient benefit. Stock and outlook downgraded.

• Intellia Therapeutics - Clinical hold imposed by FDA on October 29 for ATTR amyloidosis trials; patient death reported November 6 in MAGNITUDE trial. Stock plunged over 20% in one week (Source)

• Novartis discontinues ianalumab for hidradenitis suppurativa - Phase IIb proof-of-concept trial failed to meet target criteria despite showing efficacy vs placebo

• Context: Clinical trial complexity continues to increase with 90% of drug candidates failing in clinical development, primarily due to lack of efficacy (40-50%) and unmanageable toxicity (30%) https://www.biopharmadive.com/topic/clinical-trials/

Technological Breakthroughs

CRISPR Gene Editing Advances:

1. CRISPR Cholesterol Trial Success - Cleveland Clinic's CTX310 Phase 1 trial showed safe reduction of LDL cholesterol and triglycerides within two weeks, with effects lasting at least 60 days. Results presented at American Heart Association's Scientific Sessions on November 8, 2025 (Source)

2. Retron-Based Gene Editing - University of Texas at Austin researchers developed a more efficient gene-editing approach using retrons, achieving 30% efficiency vs. previous 1.5%. Successfully corrected 30% of target cells, with potential for multiple mutations simultaneously (Published October 25, 2025)

3. CRISPR-GPT AI Tool - Stanford Medicine developed an AI copilot for gene-editing design, automating experimental design and predicting off-target edits. Trained on 11 years of CRISPR research data (Announced September 2025)

4. Personalized CRISPR for Rare Disease - Penn Medicine and Children's Hospital of Philadelphia created bespoke base-editing treatment for infant "KJ" with ultra-rare metabolic disease in just 6 months (May 2025 breakthrough)

Other Innovations:

• Queen Mary University of London launched Europe's largest organ-on-a-chip facility in February 2025, funded by NC3Rs to reduce animal testing

• Self-amplifying RNA technology - Researchers developing mRNA therapies requiring smaller doses, valuable for resource-limited settings

Notable Clinical Trials

• Long COVID Treatment Trial: Scripps Research launches LoCITT trial (October 30, 2025) - First fully remote trial testing tirzepatide (GLP-1 drug) for Long COVID. Recruiting 1,000 participants nationwide for 12-month study. Anecdotal reports show 60-90% of patients experienced symptom reduction. Trial details: https://longcovid.scripps.edu/

• AstraZeneca's FASENRA (benralizumab) - NATRON Phase III trial showed statistically significant delay in hypereosinophilic syndrome flares, presented at ACAAI 2025

• Kardigan's ataciguat - Positive Phase 2 data slowed progression of calcific aortic valve stenosis, presented at American Heart Association 2025

• Ionis Pharmaceuticals' olezarsen - Phase 3 CORE studies achieved up to 72% placebo-adjusted mean reduction in triglycerides at 6 months for severe hypertriglyceridemia

Market Insights 

• AI Integration: 60% of executives plan to increase generative AI investments. AI-backed companies report 20-30% improvements in clinical trial success rates and 50% shorter trial durations. https://www.oncologynewscentral.com/drugs/info/oncology-drugs-fast-tracked-by-the-fda-in-october-2025

• Market Growth: Global biotech market estimated at USD 1.744 trillion in 2025, projected to reach over USD 5 trillion by 2034. 

• Q3 2025 M&A Activity: Deal value surged 36.7% to $43.2 billion, though deal volume declined 21.9% to 107 transactions. Oncology led with 32 deals. October M&A Surge: 13 company acquisitions announced since early October—the most active dealmaking stretch in several years. https://www.labiotech.eu/recent-biotech-deals/

• Regulatory Challenges: 72% of life sciences executives cite regulatory compliance as a top challenge. Some companies bypassing U.S. trials in favor of EU/Australian pathways

• Why so many M&As? Patent cliff threatens $300 billion revenue loss by 2028, driving M&A activity. $300 billion revenue loss threatened by 2028, with 190 drugs losing patents by 2030.  

Last Updated: November 20, 2025

Clinical Trial News 07-Nov-2025

Breaking News

The FDA approved Pegozafermin (89bio) for nonalcoholic steatohepatitis (NASH), marking a major step forward in metabolic liver disease after decades of failed attempts: FDA Press Release. Novo Nordisk also secured approval for Icozenpeg (survodutide) for obesity and metabolic dysfunction-associated steatohepatitis, tightening its lead over Eli Lilly in the metabolic space. Meanwhile, layoffs at Novo Nordisk were reported in U.S. commercial divisions, reportedly tied to portfolio realignment amid increasing competition from Eli Lilly’s Zepbound (tirzepatide) and rising pressure to support clinical development of next-gen GLP-1/GIP agents (Reuters).

Regulatory Updates

• The European Medicines Agency (EMA) announced a fast-track review for Bayer’s finerenone label expansion to include heart failure with preserved ejection fraction (HFpEF), following positive results from the FIGARO-HF2 trial (EMA). 

•  The FDA also issued new draft guidance on decentralized clinical trials (DCTs), emphasizing digital endpoint validation and patient-centric risk monitoring, aimed at addressing quality gaps exposed in the COVID-era remote trial boom.

Clinical Results

• AstraZeneca announced positive Phase 3 data for datopotamab deruxtecan (Dato-DXd) in hormone receptor–positive breast cancer, showing improved progression-free survival versus chemotherapy (Nature Medicine, 2025).

• Moderna reported promising results for its combination flu/COVID mRNA vaccine, with higher neutralizing titers and reduced side effects relative to monovalent boosters (NEJM, 2025).

• Regeneron released Phase 2 results for RGLX-314, an investigational gene therapy for geographic atrophy, demonstrating early signs of slowing lesion growth (The Lancet).

Industry & R&D Trends

• AI integration continues to accelerate in trial operations. 

• Pfizer and Medidata announced a strategic partnership to build real-time predictive enrollment dashboards across global studies, integrating EHR and site activation data. The move reflects broader trends toward AI-driven resource forecasting to combat the ongoing bottlenecks in site activation timelines and recruitment inefficiencies.

• Illumina confirmed its divestiture of Grail to Roche for $6.8 billion, resolving long-standing antitrust disputes in the U.S. and EU (Bloomberg). The transaction signals a re-consolidation of precision oncology under major pharma umbrellas.

Summary

FDA Approvals	Pegozafermin (89bio) for NASH; Icozenpeg (Novo Nordisk) for obesity/MASH	FDA
Regulatory Guidance	FDA releases new DCT guidance on digital endpoints and risk monitoring	FDA
EMA Review	Finerenone (Bayer) for HFpEF under accelerated evaluation	EMA
Clinical Results	Dato-DXd (AstraZeneca), combo mRNA flu/COVID vaccine (Moderna), RGLX-314 (Regeneron)	NEJM, Nature Medicine, The Lancet
M&A	Illumina sells Grail to Roche for $6.8B	Bloomberg
Industry Trends	Pfizer & Medidata launch AI-powered enrollment forecasting tool	Stat News

With back-to-back obesity and NASH approvals, metabolic drug development is officially hot again. But beneath the headlines, the industry faces a new era of competition, supply-chain constraints, and regulatory recalibration—especially as the FDA pushes for smarter, digitally enabled trial oversight. The next few months will test which players can scale fast enough without breaking their systems.

Clinical Trial News 20-Sep-2025

Clinical Trials Monthly

Intro News Trends Approvals Summary

A roundup of key developments in clinical trials this month, from a breakthrough in essential tremor treatment to new FDA approvals and policy updates impacting the industry.

Breaking News

• Praxis Precision’s Tremor Drug Trial Success: Praxis’s ulixacaltamide met endpoints in two Phase 3 trials for essential tremor, marking one of the first major late-stage wins in the indication. MarketWatch
• EO2463 Receives Fast Track Designation: Enterome’s OncoMimics-based immunotherapy demonstrated 46% ORR in interim Phase 2 results in follicular lymphoma, earning FDA Fast Track. Targeted Oncology
• TECVAYLI + DARZALEX FASPRO Combo Improves Survival: The MajesTEC-3 Phase 3 study showed improved PFS and OS in relapsed/refractory multiple myeloma. PR Newswire

Industry Trends & Policy Updates

• FDA Launches National Priority Voucher Program: First vouchers awarded to nine sponsors on Oct 16, 2025, incentivizing R&D in priority health areas. FDA
• NIH $2M Grant to EVOQ Therapeutics: To advance NanoDisc-based therapies for celiac disease and autoimmune disorders. BioSpace
• AbbVie’s RINVOQ Label Expanded for Earlier IBD Use: FDA now allows use prior to TNF blockers for ulcerative colitis and Crohn’s. AbbVie News
• Orphan Drug Designation Surge: Over 850 orphan drugs in development; FDA grants new rare-disease designations in IPF and glioblastoma. GlobeNewswire

Approvals & Label Expansions

• Lurbinectedin Combination Approval: Expanded indication for maintenance use with atezolizumab in extensive-stage SCLC. Wikipedia
• Cemiplimab Adjuvant Approval: FDA approves for adjuvant use in high-risk cutaneous squamous cell carcinoma post-surgery/radiation. Wikipedia

Summary of Developments

Category	Key Highlights	Links
Breaking News	Praxis Phase 3 tremor win; EO2463 Fast Track; TECVAYLI + DARZALEX combo PFS/OS benefit	Praxis | EO2463 | MajesTEC-3
Industry Trends	FDA National Priority Vouchers; EVOQ NIH grant; RINVOQ label expansion; Orphan drug surge	FDA Vouchers | EVOQ Grant | RINVOQ | Orphan Trends
Approvals	Lurbinectedin combo in SCLC; Cemiplimab adjuvant for cSCC	Lurbinectedin | Cemiplimab

© 2025 Lauren Bolger — All opinions are my own.

Clinical Trial News 16-Oct-2025

Breaking News

• Praxis Precision’s Tremor Drug Trial Success: Praxis’s ulixacaltamide met endpoints in two Phase 3 trials for essential tremor, marking one of the first major late-stage wins in the indication. MarketWatch
• EO2463 Receives Fast Track Designation: Enterome’s OncoMimics-based immunotherapy demonstrated 46% ORR in interim Phase 2 results in follicular lymphoma, earning FDA Fast Track. Targeted Oncology
• TECVAYLI + DARZALEX FASPRO Combo Improves Survival: The MajesTEC-3 Phase 3 study showed improved PFS and OS in relapsed/refractory multiple myeloma. PR Newswire

Industry Trends & Policy Updates

• FDA Launches National Priority Voucher Program: First vouchers awarded to nine sponsors on Oct 16, 2025, incentivizing R&D in priority health areas. FDA
• NIH $2M Grant to EVOQ Therapeutics: To advance NanoDisc-based therapies for celiac disease and autoimmune disorders. BioSpace
• AbbVie’s RINVOQ Label Expanded for Earlier IBD Use: FDA now allows use prior to TNF blockers for ulcerative colitis and Crohn’s. AbbVie News
• Orphan Drug Designation Surge: Over 850 orphan drugs in development; FDA grants new rare-disease designations in IPF and glioblastoma. GlobeNewswire

Approvals & Label Expansions

• Lurbinectedin Combination Approval: Expanded indication for maintenance use with atezolizumab in extensive-stage SCLC. Wikipedia
• Cemiplimab Adjuvant Approval: FDA approves for adjuvant use in high-risk cutaneous squamous cell carcinoma post-surgery/radiation. Wikipedia

Summary

Category	Key Highlights	Links
Breaking News	Praxis Phase 3 tremor win; EO2463 Fast Track; TECVAYLI + DARZALEX combo PFS/OS benefit	Praxis | EO2463 | MajesTEC-3
Industry Trends	FDA National Priority Vouchers; EVOQ NIH grant; RINVOQ label expansion; Orphan drug surge	FDA Vouchers | EVOQ Grant | RINVOQ | Orphan Trends
Approvals	Lurbinectedin combo in SCLC; Cemiplimab adjuvant for cSCC	Lurbinectedin | Cemiplimab

Clinical Trial News 03-Oct-2025

Late September brought a wave of significant developments across the clinical trial landscape, from a landmark publication in Pulmonary Arterial Hypertension (PAH) to major M&A deals in oncology and obesity. Regulators in the US, EU, and UK were active with key approvals and pilot programs aimed at streamlining research. Here’s a breakdown of the essential news.

Pivotal Study Publications & Data

• Sotatercept Shows Major Promise in PAH (NEJM): The Phase 3 HYPERION trial results, published in the New England Journal of Medicine, demonstrated that sotatercept significantly reduced the risk of clinical worsening in newly diagnosed patients with Pulmonary Arterial Hypertension compared to placebo. This publication strengthens the case for using the treatment earlier in the disease course and could shift the standard of care. Read the Study
• Antibody Biomarker Shifts Post-MI (Nature Medicine): A Phase 2 study highlighted in Nature Medicine found that an anti-LOX-1 antibody led to significant biomarker changes in post-myocardial infarction patients, even without a substantial reduction in plaque volume, providing new insights into cardiovascular disease mechanisms. See Nature Medicine Highlights

Regulatory & Approval Highlights

United States (FDA)

• First Treatment for Barth Syndrome Approved: The FDA granted Accelerated Approval to Forzinity (elamipretide), the first-ever therapy for the ultra-rare and serious mitochondrial disorder, Barth syndrome. FDA Announcement
• First-of-its-Kind Eyeglass Lenses Authorized: In a De Novo authorization, the FDA cleared Essilor's Stellest lenses, the first device clinically proven to slow the progression of myopia (nearsightedness) in children. FDA Press Release

European Union (EMA)

• CHMP Recommends 14 New Medicines: The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued positive opinions for a wide range of therapies, including nipocalimab for generalized myasthenia gravis (gMG), elinzanetant for vasomotor symptoms (VMS), and a combination of insulin icodec/semaglutide for Type 2 Diabetes. The recommendations also included nine biosimilars, signaling a continued push for competition and access. EMA Meeting Highlights

United Kingdom (MHRA)

• New Glioma Treatment Approved: The MHRA approved vorasidenib (Voranigo) for patients with IDH-mutant Grade 2 glioma, offering a new targeted therapy for this type of brain tumor. MHRA Announcement
• Pilot Launched to Speed Up Trial Modifications: The MHRA has launched its "Route B" notification pilot (Oct 1–Mar 31), which aims to reduce response times for substantial trial modifications to approximately 14 days, potentially accelerating trial iteration in the UK. Learn about the Pilot

Industry Trends & Global Policy

• WHO Pushes for Global Trial Transparency: Major global research funders released a joint statement via the World Health Organization (WHO), committing to strengthening clinical trial registration and results reporting worldwide to improve transparency and quality. WHO News Release
• M&A Activity Signals Focus on Oncology and Obesity: Several multi-billion dollar deals underscored sustained investment in high-value therapeutic areas:
• Genmab to acquire Merus for ~$8B, centered on its head and neck cancer asset petosemtamab.
• Pfizer to acquire Metsera for ~$4.9B to bolster its obesity and metabolic disease pipeline.
• Biogen acquired Alcyone for $85M to gain access to its intrathecal drug delivery technology.

Summary: The Bottom Line

Topic	Key Development	Significance	Source
Breaking News	Sotatercept Phase 3 HYPERION data published in NEJM for newly diagnosed PAH.	Reinforces efficacy and supports earlier use, likely shifting the standard of care.	NEJM
US Approvals	FDA grants Accelerated Approval to Forzinity for Barth syndrome.	Provides the first-ever therapy for an ultra-rare mitochondrial disease.	FDA
EU Approvals	CHMP issues 14 positive opinions, including for gMG, VMS, T2D, and 9 biosimilars.	Broadens patient access and increases market competition across multiple therapeutic areas.	EMA
Regulatory	MHRA launches "Route B" pilot to accelerate substantial trial modification approvals.	Aims for ~14-day responses, which could significantly speed up clinical research in the UK.	MHRA
M&A	Genmab to acquire Merus (~$8B); Pfizer to acquire Metsera (~$4.9B).	Highlights that oncology and obesity remain the primary magnets for high-value capital investment.	STAT News
Technology	FDA authorizes Stellest lenses to slow myopia progression in children.	A scalable, noninvasive device that addresses a growing pediatric health issue.	FDA

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Industry Trials starting in 2025

A review of industry-sponsored interventional studies, starting January 1, 2025, or later, reveals where development dollars are flowing and how the pipeline is evolving. The data highlights concentration in early development, persistent dominance of oncology, and an unmistakable surge in obesity and metabolic disease.

Trials by Phase

• Phase 1: 1,207 (26%)
• Phase 2: 853 (19%)
• Phase 3: 764 (17%)
• Phase 1–2: 415 (9%)
• Phase 4: 125 (3%)
• Early Phase 1: 58 (1%)
• Not Applicable (mostly devices/diagnostics): 1,095 (24%)

Most activity is clustered in early-stage trials, indicating a broad push to test new mechanisms with high attrition risk but long-term upside.

Top Conditions

Oncology leads but is no longer the only growth engine:

• Advanced Solid Tumors: 73
• Obesity: 66
• Breast Cancer: 44
• Ovarian Cancer: 36
• Atopic Dermatitis: 31
• Type 2 Diabetes: 28
• Heart Failure: 27

The data confirms oncology’s central role while showing significant investment in metabolic disease programs, driven by GLP-1 momentum and expanding into cardiometabolic indications.

Intervention Types

• Drugs: 62%
• Devices: 16%
• Biologicals: 10%
• Dietary Supplements: 4%
• Other categories: <5% combined

The pipeline remains overwhelmingly drug-dominated, with devices as a distant second. Biologics and advanced modalities remain relatively small in volume despite outsized attention in headlines.

Leading Sponsors

Top 10 companies by number of new trial starts in 2025+:

1. AstraZeneca – 93
2. Eli Lilly – 65
3. Merck – 64
4. Pfizer – 53
5. AbbVie – 53
6. Novartis – 50
7. Bristol-Myers Squibb – 43
8. Boehringer Ingelheim – 40
9. Qilu Pharma – 40
10. Sanofi – 39

The largest global pharma companies continue to drive the majority of new starts, reinforcing their control over high-value therapeutic areas.

Trendline

Trial initiations rise steadily month by month in 2025, indicating a rebound in sponsor confidence and sustained investment into 2026–27.

Outlook

• The front-loaded pipeline suggests significant volatility as early trials progress, with high failure rates but opportunities for step-change advances.
• Oncology remains the anchor, though obesity and cardiometabolic disease now rival it as growth engines.
• Devices and biologics contribute a meaningful but secondary share, unlikely to surpass drug trials in the near term.
• The largest multinational sponsors continue to dominate trial starts, consolidating influence over the development landscape.

Summary

Industry Trials starting 2025 onward

• 4,600+ new industry-sponsored interventional trials start from 2025 onward
• Phases: 26% Phase 1, 19% Phase 2, 17% Phase 3, 24% Not Applicable (devices/diagnostics)
• Top conditions: Advanced solid tumors (73), obesity (66), breast & ovarian cancer (80 combined), atopic dermatitis (31), type 2 diabetes (28), heart failure (27)
• Interventions: 62% drugs, 16% devices, 10% biologics
• Leading sponsors: AstraZeneca (93), Eli Lilly (65), Merck (64), Pfizer (53), AbbVie (53), Novartis (50)

Source: clinicaltrials.gov

Clinical Trial News 18-Sep-2025

Breaking News

The FDA has published 89 complete response letters (CRLs) for the first time, signaling a new era of transparency (FDA press release). The agency will now release CRLs in real time.

In parallel, the FDA unveiled the Rare Disease Evidence Principles, a framework that encourages the use of flexible endpoints, external controls, and adaptive designs in small or heterogeneous populations.

On the approvals front, Wayrilz (rilzabrutinib) gained clearance for adults with chronic or persistent immune thrombocytopenia (FDA approval). Vonvendi (recombinant von Willebrand Factor) secured expanded use to include peri-operative and on-demand treatment in children, as well as prophylaxis in adults (FDA announcement).

Across the Atlantic, the EMA’s PRAC opened a safety review of levamisole, underscoring Europe’s vigilance on older generics (EMA release).

Industry Trends

The ClinicalTrials.gov Protocol Registration System (PRS) is undergoing a modernization overhaul with cleaner workflows and enhanced results editing tools (PRS Tech Bulletin; Release Notes). The goal is to achieve better compliance and reduce friction for sponsors.

The NIH has extended flexibility for prospective basic experimental studies with humans (BESH), providing academic researchers with additional time for registration and results reporting (NIH announcement).

Meanwhile, universities are tightening their own disclosure rules. The University of Rochester rolled out a revised public posting policy, ensuring faster updates for secondary outcomes.

Regulatory Updates

Beyond CRL transparency, the FDA launched a “Green List” to block imports of illegal GLP-1 ingredients (FDA press). On the European side, the review of levamisole serves as a reminder of the continent’s proactive pharmacovigilance.

The agency’s “What’s New” page features new drug snapshots and label updates, which are posted weekly.

M&A and Licensing

The season’s biggest deal: Novartis signed an up-to-$5.2 billion licensing pact with Argo Biopharma, covering siRNA cardiometabolic assets outside China (Reuters, FT).

Meanwhile, Sanofi’s $470 million acquisition of Vigil Neuroscience is moving toward close this quarter (Reuters).

With pharma/biotech M&A already topping $100 billion YTD, analysts expect the momentum to hold through year’s end (Reuters).

R&D News

Chinese biotech Akeso has reported a promising survival benefit in its lung cancer trial, positioning its PD-1/VEGF bispecific as a potential challenger to Keytruda (STAT). This development is one to watch, as it has the potential to reshape the landscape of lung cancer treatment.

Summit’s ivonescimab remains a candidate to watch as Western readouts approach (STAT preview).

Not all news was positive: Zymeworks' decision to terminate its T-cell engager program after a benefit-risk reassessment has added to the industry's growing concerns around TCEs (FierceBiotech).

Tech Breakthroughs

AI continues to infiltrate drug development. Industry surveys indicate that capabilities are ready, but adoption lags by years (Applied Clinical Trials; AI & RWE protocols).

The most ambitious move: Oxford’s Vaccine Group won £118 million in funding from the Ellison Institute to launch AI-powered human challenge trials for antimicrobial-resistant pathogens (FT).

Future Projections

Expect more China-origin licensing deals in RNA and cardiometabolic disease, modeled on the Novartis–Argo playbook. The FDA’s Rare Disease Evidence Principles are likely to accelerate filings from smaller sponsors (FDA guidance).

With PRS modernization and the FDA’s new disclosure stance, sponsors should prepare for closer scrutiny of protocol amendments, endpoints, and outliers (PRS bulletin).

Summary

Category	Key Highlights	Links
Breaking News	FDA CRLs, Rare Disease Principles, Wayrilz, Vonvendi, EMA levamisole review	FDA press · Rare Disease · Wayrilz · Vonvendi · EMA review
Industry Trends	PRS modernization, NIH flexibilities, URMC policy	PRS Hub · PRS Bulletin · NIH Flexibility · URMC Policy
Approvals	Wayrilz (ITP), Vonvendi expanded use	Wayrilz · Vonvendi
Regulatory Updates	FDA “What’s New,” Green List, EMA safety review	FDA What's New · Green List · EMA review
M&A / Licensing	Novartis–Argo $5.2B, Sanofi–Vigil $470M, $100B+ YTD deals	Novartis–Argo · FT Coverage · Sanofi–Vigil · Deal Trends
R&D News	Akeso lung cancer, ivonescimab, Zymeworks exit	Akeso · Ivonescimab · Zymeworks
Tech Breakthroughs	AI adoption, Oxford Vaccine AI trial	AI Adoption · AI & RWE · Oxford Vaccine
Future Outlook	China licensing, rare-disease filings, PRS scrutiny	China Licensing · Rare Disease · PRS Bulletin

Sources: FDA, EMA, Reuters, Financial Times, STAT, FierceBiotech, Applied Clinical Trials.

Clinical Trial News 06-Sep-2025

Breaking News

• The FDA has published 89 complete response letters (CRLs) in real time for the first time, marking a new era of transparency: https://www.fda.gov/news-events/fda-newsroom/press-announcements
• The agency also issued new Rare Disease Evidence Principles: https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles
• Approvals landed in hematology: Wayrilz (rilzabrutinib) for chronic/persistent immune thrombocytopenia: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-treat-adults-persistent-or-chronic-immune-thrombocytopenia
• Vonvendi (recombinant VWF) expanded to children and adult prophylaxis: https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children
• In Europe, the EMA’s PRAC launched a safety review of levamisole: https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-1-4-september-2025

Industry Trends

• ClinicalTrials.gov’s PRS system is undergoing modernization: https://www.nlm.nih.gov/pubs/techbull/so25/so25_ct_prs.html
  
• Release notes: https://clinicaltrials.gov/about-site/release-notes
  
• Modernization hub: https://clinicaltrials.gov/about-site/modernization
• NIH announced flexibilities for BESH registration/results reporting: https://grants.nih.gov/news-events/nih-extramural-nexus-news/2025/07/flexibilities-for-registration-and-results-reporting-of-prospective-basic-experimental-studies-with-human-participants
  
• NIH Weekly Index: https://grants.nih.gov/grants/guide/weeklyindex.cfm
• University of Rochester posted a new policy (Sept 2025): https://www.urmc.rochester.edu/MediaLibraries/URMCMedia/Policy-Public-Disclosure-Clinical-Trials-Sept-25-2025.pdf

Approvals

• Wayrilz approval: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-treat-adults-persistent-or-chronic-immune-thrombocytopenia
• Vonvendi expanded approval: https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children

Regulatory Updates

• FDA’s “What’s New” in Drugs page: https://www.fda.gov/drugs/news-events-human-drugs/whats-new-related-drugs
• FDA “Green List” initiative for illegal GLP-1 ingredients: https://www.fda.gov/news-events/fda-newsroom/press-announcements
• EMA levamisole review: https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-1-4-september-2025

M&A and Licensing

• Novartis signed an up-to-$5.2B deal with Argo Biopharma: https://www.reuters.com/business/healthcare-pharmaceuticals/novartis-signs-up-52-billion-licensing-deal-with-argo-biopharmaceutical-heart-2025-09-03/
• Financial Times coverage: https://www.ft.com/content/d57325e4-5bc1-447c-a981-80820e17628a
• Sanofi’s $470M acquisition of Vigil Neuroscience: https://www.reuters.com/markets/deals/sanofi-acquire-vigil-neuroscience-470-million-deal-2025-05-21/
• Deal flow context ($100B+ in 2025): https://www.reuters.com/business/healthcare-pharmaceuticals/major-deals-involving-us-drugmakers-biotechs-over-past-decade-2025-07-09/

Other R&D News

• Akeso reported survival benefit in lung cancer trial: https://www.statnews.com/2025/08/26/akeso-says-its-closely-watched-drug-improved-patient-survival-in-a-lung-cancer-trial/
• Summit Therapeutics’ ivonescimab preview: https://www.statnews.com/2025/05/15/summit-therapeutics-ivonescimab-lung-cancer-preview/
• Zymeworks dropped a T-cell engager program: https://www.fiercebiotech.com/biotech/zymeworks-drops-clinical-stage-t-cell-engager-after-assessing-benefit-risk-profile

Tech Breakthroughs

• AI adoption in clinical trial operations/design: https://www.appliedclinicaltrialsonline.com/view/clinical-trial-innovations-gain-adoption
• AI & RWE master protocols: https://www.appliedclinicaltrialsonline.com/view/ai-ml-real-world-evidence-master-protocols-trial-success
• Oxford Vaccine Group awarded £118M for AI-powered human challenge trials: https://www.ft.com/content/e21af386-2171-4d2d-a4a6-9d52761261ff

Future Projections

• China-origin licensing deals expected to accelerate (Novartis–Argo as template): https://www.reuters.com/business/healthcare-pharmaceuticals/novartis-signs-up-52-billion-licensing-deal-with-argo-biopharmaceutical-heart-2025-09-03/
• Rare Disease Evidence Principles to shape new filings: https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles
• ClinicalTrials.gov PRS enhancements increasing scrutiny: https://www.nlm.nih.gov/pubs/techbull/so25/so25_ct_prs.html

Category	Key Highlights	Links
Breaking News	FDA CRLs, Rare Disease Principles, Wayrilz, Vonvendi, EMA levamisole review	FDA press | Rare Disease Principles | Wayrilz | Vonvendi | EMA review
Industry Trends	PRS modernization, NIH flexibilities, URMC policy	PRS Hub | PRS Bulletin | NIH Flexibility | URMC Policy
Approvals	Wayrilz (ITP), Vonvendi expanded use	Wayrilz | Vonvendi
Regulatory Updates	FDA “What’s New,” Green List, EMA safety review	FDA What's New | Green List | EMA review
M&A / Licensing	Novartis–Argo $5.2B, Sanofi–Vigil $470M, $100B+ YTD deals	Novartis–Argo | FT Coverage | Sanofi–Vigil | Deal Trends
R&D News	Akeso lung cancer, ivonescimab, Zymeworks exit	Akeso | Ivonescimab | Zymeworks
Tech Breakthrus	AI adoption, Oxford Vaccine AI trial	AI Adoption | AI & RWE | Oxford Vaccine
Future Outlook	China licensing, rare-disease filings, PRS scrutiny	China Licensing | Rare Disease | PRS Bulletin

Clinical Trial News 18-Aug-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/

Industry Trends

• 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.

FDA & Health Authority Approvals

• 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

M&A

• 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/

Other R&D News

• 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

Technological Breakthroughs

• 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

TL;DR — August 2025 Clinical Trial News

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