Welcome to this week’s edition of the Life Sciences Voice Top Five Newsletter, your go-to source for the latest breakthroughs in the life sciences industry! This time, we’re looking at phase 3 results from Pfizer’s sickle cell trial, what the FDA has to say about Novo’s manufacturing facility in Indiana, the third indication awarded to Wegovy and many more! Stay informed and inspired by the innovations driving life sciences forward.
Pfizer’s Inclacumab Fails to Meet Endpoint in Phase 3 Sickle Cell Trial
Pfizer’s sickle cell therapy candidate, inclacumab, did not meet its primary endpoint in the late-stage Thrive-131 trial, which evaluated its ability to reduce vaso-occlusive crises (VOCs) in over 240 patients. The randomized, double-blind, placebo-controlled study ran for 48 weeks but showed no meaningful reduction in VOCs compared to placebo. Inclacumab, a P-selectin inhibitor acquired through Pfizer’s $5.4 billion purchase of Global Blood Therapeutics, was generally well tolerated, with common side effects including anemia, headache, and back pain. The outcome follows earlier setbacks, including the withdrawal of Oxbryta in 2024, leaving osivelotor as Pfizer’s main remaining late-stage SCD program.
FDA Flags Manufacturing Deficiencies at Novo Nordisk’s Bloomington Facility
The U.S. Food and Drug Administration issued a Form 483 to Novo Nordisk’s Bloomington, Indiana, site after a June–July inspection, citing six manufacturing deficiencies. Observations included inadequate investigations into contamination, equipment failures, pest detection, and lapses in environmental monitoring. Inspectors noted contaminants such as cat hair and human hair in vial stoppers, as well as insufficient assessment of bacterial incursions. The FDA also flagged reliance on supplier testing of polysorbate 20 and 80 without internal verification. The Bloomington facility has a history of regulatory challenges, previously affecting approvals for Regeneron’s Eylea and delaying Moderna’s COVID-19 vaccine boosters.
FDA Grants Novo Nordisk’s Wegovy Accelerated Approval for MASH Treatment
The FDA has granted accelerated approval for Novo Nordisk’s Wegovy 2.4 mg in adults with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to severe fibrosis, excluding those with cirrhosis. Approval was based on interim results from the ongoing two-stage Essence trial, where 63% of patients achieved resolution of steatohepatitis without fibrosis progression at 72 weeks, versus 34% on placebo. Nearly 40% saw fibrosis improvement without worsening inflammation, compared to less than 25% in controls. A third achieved both outcomes, versus 16% on placebo. Final confirmatory data are expected around 2029. Wegovy now has three FDA-approved indications.
Regeneron’s Cemdisiran Meets Phase 3 Endpoint in Generalized Myasthenia Gravis Trial
Regeneron reported positive phase 3 results for cemdisiran in generalized myasthenia gravis (gMG). The study enrolled 190 adults with anti-AChR antibodies, dividing them into cemdisiran alone, cemdisiran plus pozelimab, or placebo groups. At 24 weeks, cemdisiran patients achieved a 2.3-point reduction on the MG-ADL symptom scale versus placebo, while the combination showed a 1.74-point decline; both were statistically significant, meeting the primary endpoint. Secondary measures were also successful. Cemdisiran outperformed the dual regimen, prompting Regeneron to plan a U.S. approval filing in early 2026. The therapy originated from a licensing agreement with Alnylam updated in 2024.
Dynavax Reports Positive Early-Stage Results for Experimental Shingles Vaccine
Dynavax announced early-phase results for its experimental shingles vaccine candidate, Z-1018, showing comparable immune responses to GlaxoSmithKline’s Shingrix. In a phase 1/2 trial of participants aged 50–69, Z-1018 achieved a 100% humoral response after the second dose, versus nearly 97% for Shingrix. Cellular immune responses reached almost 90% compared with Shingrix’s 93.5%. Z-1018 demonstrated lower rates of grade 2 and 3 post-injection reactions than Shingrix, with 13% local and 27.5% systemic, versus over 52% and 63%, respectively. Dynavax plans to advance the candidate into the trial’s second stage in participants aged 70 and older.
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