Clinical Trial News 05-Jan-2026

Clinical Trial News: Year-End 2025 Recap & January 2026 Kickoff

FDA Drug Approvals

• Nereus (tradipitant): Approved 30-Dec-2025 for the prevention of vomiting associated with motion sickness in adults. Vanda Pharmaceuticals. FDA Link

• Yartemlea (narsoplimab-wuug): Approved 24-Dec-2025 as the first treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Omeros Corporation. FDA Link

• Aqvesme (mitapivat): Approved 23-Dec-2025 for the treatment of anemia in adults with alpha- or beta-thalassemia. Agios Pharmaceuticals. FDA Link

• Myqorzo (aficamten): Approved 19-Dec-2025 for symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Includes a Boxed Warning for heart failure risk. Cytokinetics, Inc. FDA Link

• Fesilty (fibrinogen, human-chmt): Approved 19-Dec-2025 for acute bleeding in patients with congenital fibrinogen deficiency. Octapharma. FDA Link

• Rybrevant Faspro (amivantamab and hyaluronidase-lpuj): Subcutaneous formulation approved 18-Dec-2025 for EGFR-mutated non-small cell lung cancer (NSCLC). Johnson & Johnson. FDA Link

• Exdensur (depemokimab-ulaa): Approved 16-Dec-2025 as the first ultra-long-acting IL-5 antagonist for severe eosinophilic asthma (administered twice yearly). GSK. FDA Link

• Nuzolvence (zoliflodacin): Approved 12-Dec-2025 for uncomplicated urogenital gonorrhea. First-in-class oral antibiotic for MDR strains. Innoviva. FDA Link

• Lerochol (lerodalcibep-liga): Approved 12-Dec-2025 to reduce LDL-C in adults with hypercholesterolemia. LIB Therapeutics. FDA Link

• Uplizna (inebilizumab-cdon): Label expansion approved 11-Dec-2025 for generalized Myasthenia Gravis (gMG) in AChR+ and MuSK+ patients. Amgen. FDA Link

• Waskyra (etuvetidigene autotemcel): Approved 09-Dec-2025 as the first gene therapy for Wiskott-Aldrich Syndrome. Rocket Pharmaceuticals. FDA Link

• Breyanzi (lisocabtagene maraleucel): Approved 04-Dec-2025 as the first CAR T-cell therapy for relapsed/refractory marginal zone lymphoma (MZL). Bristol Myers Squibb. FDA Link

M&A Deals

• Pfizer acquires Metsera: $10 billion (Closed Dec 2025). Pfizer beat out Novo Nordisk in a bidding war to secure Metsera’s GLP-1/GIP obesity pipeline. Source

• Merck acquires Cidara Therapeutics: $9.2 billion (Announced Dec 2025). Focus on CD388, a universal long-acting antiviral for influenza prevention. Source

• Thermo Fisher acquires Clario: $9.4 billion (Announced Dec 2025). Strategic move to integrate high-end clinical-trial endpoint data and analytics. Source

• J&J acquires Intra-Cellular Therapies: $14.6 billion (Completed). The largest biopharma deal of the year, securing Caplyta for neuropsychiatric indications. Source

• Novartis acquires Avidity Biosciences: $12 billion (Closed Dec 2025). Expands Novartis’ reach into RNA-based therapies for rare neuromuscular diseases. Source

Clinical Trial Setbacks

• Sanofi (tolebrutinib): Discontinued development for Primary Progressive Multiple Sclerosis (PPMS) after Phase 3 PERSEUS trial failed to meet the primary endpoint of delaying disability progression (Dec 15, 2025). Source

• Kamada Pharmaceuticals: Terminated Phase 3 trial for Inhaled AAT (Alpha-1 Antitrypsin) in December following a futility analysis. Source

• NIH Funding Freeze: In Dec 2025, federal budget constraints led to a pause in roughly 3.5% of all NIH-funded active trials, affecting approximately 74,000 enrolled patients. Source

Technological Breakthroughs

• FDA Qualifies First AI Drug Development Tool (DDT): On Dec 8, 2025, the FDA officially qualified an AI-driven tool for patient stratification in MASH (formerly NASH) trials, marking a regulatory milestone for generative AI in trial design. Source

• Edge AI for Patient Monitoring: New data from Jan 2026 suggests "Edge AI" wearables (processing data on-device) reduced data latency in decentralized trials by 40%, significantly improving remote patient safety monitoring. Source

Market Insights

• The 2026 "Patent Cliff": Major expiries for blockbusters like Keytruda and Darzalex are projected to trigger a $230 billion revenue loss by 2030, fueling the current surge in aggressive, late-stage M&A. Source

• Oncology Shift: While oncology still leads M&A volume, deal values in Neuroscience and Metabolic (Obesity/MASH) diseases have surpassed oncology for the first time in five years as of Q4 2025. Source

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Summary Table: Recent Major FDA Actions

Brand Name	Active Ingredient	Company	Indication	Approval Date
Nereus	Tradipitant	Vanda	Motion Sickness (Vomiting)	30-Dec-2025
Yartemlea	Narsoplimab	Omeros	HSCT-TMA	24-Dec-2025
Aqvesme	Mitapivat	Agios	Thalassemia Anemia	23-Dec-2025
Myqorzo	Aficamten	Cytokinetics	Obstructive HCM	19-Dec-2025
Exdensur	Depemokimab	GSK	Severe Eosinophilic Asthma	16-Dec-2025
Nuzolvence	Zoliflodacin	Innoviva	Gonorrhea	12-Dec-2025
Lerochol	Lerodalcibep	LIB Therapeutics	Hypercholesterolemia	12-Dec-2025
Waskyra	Etuvetidigene	Rocket Pharma	Wiskott-Aldrich Syndrome	09-Dec-2025
Breyanzi	Liso-cel	BMS	Marginal Zone Lymphoma	04-Dec-2025

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. FDA Link

• 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. FDA Link

• Redemplo (plozasiran): Approved 18-Nov-2025 for familial chylomicronemia syndrome. RNA interference therapeutic to reduce triglycerides. NDA #219947. Arrowhead Pharmaceuticals, Inc. FDA Link

• 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. FDA Link

M&A Deals

• 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.  

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.