Clinical Research Focus. 56th Edition

Taiho and Cullinan’s Zipalertinib Delivers Positive Phase 3 Results in Lung Cancer

Taiho Oncology and Cullinan Therapeutics’ zipalertinib plus chemotherapy met the primary endpoint in the Phase 3 REZILIENT3 trial, significantly improving progression-free survival in previously untreated patients with EGFR exon 20 insertion-positive NSCLC. The results could support a new first-line targeted treatment option for this hard-to-treat form of lung cancer.

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Big Pharma Is Betting Billions on Vaccines. Washington Is Making That Bet Harder to Win.

Pharma investment in vaccines is accelerating, but shifting US regulatory expectations are making long-term clinical development harder to predict. What does this mean for sponsors designing vaccine programs today? Read our latest analysis on why building flexibility into global development strategies is becoming increasingly important.

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FDA Approves First-Ever Gene Therapy for Glycogen Storage
Disease Type Ia

The FDA has granted accelerated approval to Genglycos, the first approved treatment for glycogen storage disease type Ia (GSDIa) in patients aged 8 and older. The one-time gene therapy targets the underlying cause of this rare genetic disease, marking an important milestone for patients who have historically relied on strict, lifelong dietary management.

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The Next GLP-1 Moment? Why Big Pharma Is Investing in Depression

Depression is emerging as one of pharma’s biggest new investment opportunities, with major companies betting on next-generation treatments, including psychedelic therapies. But success will depend on more than promising science. Trial design, patient selection, endpoints, experienced sites, and consistent execution may ultimately determine which therapies make it to market.

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Human Brain Organoids Survive for Nearly Six Years, Opening New Possibilities for Neuroscience Research

NIH-funded researchers have sustained human brain organoids for nearly six years and found that they mature according to a lifelike developmental timeline, including features of postnatal brain development. The breakthrough could enable scientists to study later stages of human brain development in the lab and create new opportunities for research into neurodevelopmental disorders such as autism.

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Merck and Moderna’s Personalized mRNA Cancer Therapy Succeeds in Phase 3

Merck and Moderna have reported a landmark Phase 3 win for their personalized mRNA cancer therapy, with intismeran plus KEYTRUDA significantly improving recurrence-free and distant metastasis-free survival in patients with resected melanoma. This marks the first positive Phase 3 result for an individualized neoantigen therapy and an mRNA-based cancer treatment, potentially opening a new chapter in personalized oncology. 

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FDA Places Clinical Hold on REGENXBIO’s RGX-121 Gene Therapy Trial

The FDA has placed a clinical hold on REGENXBIO’s RGX-121 gene therapy program for Hunter syndrome, adding a new challenge to an already complicated regulatory path. Earlier this year, the FDA declined to approve RGX-121, citing concerns about whether the study design and available evidence were sufficient to demonstrate effectiveness, a decision we covered in our previous article. Now, the program faces a different issue: asymptomatic spine MRI findings have been identified in five participants who received the therapy approximately three to six years ago, prompting the FDA to halt the trial. The case highlights how gene therapy programs can face regulatory challenges at different stages, from questions about efficacy evidence to safety signals emerging years after treatment.

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Amylyx’s GLP-1 Antagonist Cuts Hypoglycemic Events by 55% in Phase 3

Amylyx’s first-in-class GLP-1 receptor antagonist avexitide met its primary endpoint in the Phase 3 LUCIDITY trial, reducing Level 2 and Level 3 hypoglycemic events by 55% in patients with post-bariatric hypoglycemia. With all secondary endpoints met and an NDA planned by year-end, avexitide could become the first FDA-approved treatment for this serious metabolic condition.

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