DYNE-251: Granted Breakthrough Therapy Designation by the U.S. FDA

Dyne Therapeutics AnnouncesThe U.S. FDA has granted the investigational therapy DYNE-251Breakthrough Therapy Designation, for DMD patients suitable for exon 51 skipping therapy.The designation was granted based on data from the ongoing DELIVER clinical trial. Dyne has completed enrollment of 32 patients in the registrational expansion cohort of the DELIVER trial, with data from this cohort expected to be released by the end of 2025.The company is expected to submit a Biologics License Application (BLA) for accelerated approval in the United States in early 2026.

DYNE-251 is composed of a phosphorodiamidate morpholino oligonucleotide (PMO) conjugated with an antibody fragment (Fab) targeting transferrin receptor 1 (TfR1).TfR1 is highly expressed in muscle tissue. This therapy aims to achieve targeted delivery to muscle tissue, promoting exon skipping both inside and outside the nucleus, thereby enabling muscle cells to produce a truncated but functional dystrophin protein, with the goal of halting or reversing disease progression. DYNE-251 has previously been granted Fast Track and Orphan Drug designations by the FDA, as well as Rare Pediatric Disease designation.

Sanofi Nearly $400 Million to Boost Potential "First-in-Class" Therapy Development

Arrowhead Pharmaceuticals announced that its subsidiary Visirna has signed an agreement with Sanofi. According to the terms of the agreement, Sanofi will obtain exclusive rights to develop and commercialize the investigational therapy plozasiran (VSA001) in Greater China. Plozasiran is a potential "first-in-class" treatment.siRNATherapy, aimed at inhibiting the production of apolipoprotein C-III (APOC3), for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG).According to the agreement, Sanofi will pay Visirna a prepayment of 130 million US dollars and up to 265 million US dollars in the future based on regulatory milestones.

Visirna has successfully completed the Phase 3 clinical trial of plozasiran in Chinese FCS patients, achieving the primary efficacy endpoint and all key secondary endpoints. Previously announcedTest ResultsDisplay,Plozasiran reduced patients' median triglyceride levels by up to 89% and median APOC3 levels by up to 93% at the 10th month, showing statistically significant differences compared to the placebo group.Visirna submitted a new drug marketing application to the China National Medical Products Administration (NMPA) in January 2025, which has been officially accepted. This collaboration will further advance the clinical application process of plozasiran in the Greater China region, potentially offering a new treatment option for patients with hypertriglyceridemia.

Semaglutide: Phase 3a Clinical Trial Data Released

Recently,JAMA Internal MedicineA study conducted in Japan and Korea was published in a journal, utilizingSemaglutide TreatmentOverweight or Obese Patients in East AsiaPhase 3a Clinical TrialsPositive Results. The studyA total of 201 adult patients were enrolled, with a BMI ≥27.0 and two or more related complications, or a BMI ≥35.0 and one or more related complications, among whom 25.4% had comorbid type 2 diabetes.Participants were randomly assigned to receive once-daily oral semaglutide 50 mg or placebo, combined with lifestyle intervention, for a duration of 68 weeks.

The research results show that,Once-daily oral semaglutide 50 mg led to an average weight loss of 14.3% in East Asian patients who were overweight or obese (with or without type 2 diabetes), significantly outperforming the placebo group. Additionally, cardiometabolic risk factors improved, and the treatment demonstrated a favorable safety profile.This discovery brings new treatment hope to East Asian populations, especially patients with type 2 diabetes.