Recently, across ChinaThe field of spherical polypeptides and oligonucleotides (TIDES) welcomes a seriesEnterExpand.AusperBioAimed at treating chronic hepatitis B to achieve functional cureAntisense Oligonucleotide (ASO) TherapyAHB-137 Granted Approval to Initiate Phase 3 Clinical Trial in China.Avidity BiosciencesDelpacibart zotadirsen (del-zota), an investigational therapy for the treatment of Duchenne Muscular Dystrophy (DMD), has been granted Breakthrough Therapy Designation by the U.S. FDA. The company plansSubmit for marketing application to FDA as planned by the end of 2025.Ionis PharmaceuticalsPositive Results from Phase 3 Clinical Trial of Donidalorsen, an Investigational ASO Therapy for the Treatment of Hereditary Angioedema (HAE), Poised for Potential FDA Approval Soon.ThisThis article will provide a brief introduction to some of the important advancements, only.For readers' reference.

AHB-137: Approved to Initiate Phase 3 Clinical Trial in China

AusperBio Announces that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China has Approved the Initiation of AHB-137Phase 3 Clinical TrialAHB–137 is an ASO therapy designed to treat chronic hepatitis B with the aim of achieving functional cure.This therapy precisely matches the RNA of the hepatitis B virus, recruits enzymes in liver cells to destroy the viral RNA, thereby halting the replication of the hepatitis B virus and the production of viral proteins.This Phase 3 clinical trial is a randomized, double-blind, multicenter study conducted in China, aimed at evaluating the efficacy of AHB-137 at a dose of 300 mg over a 24-week treatment period in patients receiving stable nucleos(t)ide analog therapy.TheHepatitis B e antigen (Efficacy and Safety of HBeAg-Negative Chronic Hepatitis B Patients.

According to a previous press release from AusperBio,AHB-137 treatment canHepatitis B Surface Antigen (HBsAg)Rapid decline and induction of higher HBsAg seroconversion.AHB-137 Demonstrates Exciting Efficacy Data and Favorable Safety Data in Phase 2b Clinical Trials, Further Confirming the Clinical Benefits Observed in Phase 2a Trials. The Approval of This Phase 3 Clinical Trial Marks a Significant Milestone.AusperBioA crucial step towards achieving the clinical cure goal for chronic hepatitis B.

Delpacibart zotadirsen (del-zota): Granted Breakthrough Therapy Designation by the FDA

Avidity Biosciences announced that its investigational therapy del-zota for the treatment of DMD has been granted Breakthrough Therapy Designation by the U.S. FDA.For the treatment of Duchenne Muscular Dystrophy (DMD) amenable to exon 44 skipping.The therapy is currently undergoing the Phase 2 EXPLORE44 open-label extension trial (EXPLORE44-OLE).Avidity expects to submit a Biologics License Application (BLA) to the FDA by the end of 2025 as planned.

Previously published study results showed that del-zota at doses of 5 mg/kg and 10 mg/kg both demonstrated strong skeletal muscle delivery capabilities, with oligonucleotide levels in skeletal muscle reaching 200 nM.Exon 44 skipping efficiency increased significantly by 40%. Dystrophin production increased significantly, averaging 25% of normal levels, with a maximum reach of 58% of normal levels. Creatine kinase levels decreased to near-normal levels, reducing by more than 80% compared to baseline.

Del-zota consists of a monoclonal antibody targeting type 1 transferrin receptor (TfR1) conjugated with an oligonucleotide that promotes exon 44 skipping in the precursor mRNA encoding dystrophin, with TfR1 being highly expressed in muscle tissue.This therapy produces a protein that functions almost identically to normal dystrophin by altering the mRNA splicing process. Monoclonal antibodies targeting TfR1 can enhance muscle-specific delivery of oligonucleotides by binding to receptors on the surface of muscle cells.

Donidalorsen: Phase 3 Clinical Trial Results Announced

Ionis Pharmaceuticals announced positive results from the Phase 3 OASISplus study of its investigational ASO therapy, donidalorsen, for the treatment of hereditary angioedema.As an investigational ASO therapy targeting prekallikrein (PKK), donidalorsen aims to prevent acute HAE attacks by inhibiting the production of PKK.Donidalorsen is currently under review by the U.S. FDA as a potential "first-in-class" prophylactic treatment for HAE, with a PDUFA date set for August 21.

In the study, patients transitioned from their previous treatment to donidalorsen according to a predefined standardized protocol, with no increase in disease attacks observed during the process. After 16 weeks of treatment, the average HAE attack frequency was further significantly reduced compared to their prior treatment.The Angioedema Control Test (AECT) score also showed that the proportion of patients reporting good disease control increased from 67% to 93% after switching medications. Regardless of prior treatment used, there was a general improvement in patients' quality of life assessments.

Icotrokinra (JNJ-2113): Submission of NDA to the U.S. FDA

Johnson & Johnson and Protagonist Therapeutics Announce Submission to U.S. FDANew Drug Application (NDA), seeking approval for its investigational once-daily oral peptide therapy icotrokinra (JNJ-2113) for the treatment of moderate to severe plaque psoriasis (PsO) in adults and pediatric patients aged 12 years and older.Icotrokinra is a potential "first-in-class" targeted oral peptide that can selectively block the IL-23 receptor (IL-23R).IL-23 plays a key role in the pathogenic T-cell activation in moderate to severe plaque PsO and is fundamental to the inflammatory response in PsO, other dermatological conditions, rheumatic diseases, and IL-23-mediated gastrointestinal disorders.Icotrokinra can bind to IL-23R with single-digit picomolar affinity and potently inhibit IL-23 signaling in human T cells with selectivity.

This application includes data from four pivotal Phase 3 clinical trials, all part of the ICONIC clinical development program, including ICONIC-LEAD, ICONIC-TOTAL, and ICONIC-ADVANCE 1 and ICONIC-ADVANCE 2.Icotrokinra successfully achieved all primary and co-primary endpoints in the aforementioned Phase 3 study, in patients aged 12 years and older with moderate to severe...PsOSignificant skin symptom clearance and good safety were demonstrated in both adult and pediatric patients.

Semaglutide: Approved for New Indications in China

Novo Nordisk announced that NMPA has officiallyApprovalSemaglutide Injection Receives New Indication for Chronic Kidney Disease (CKD).This is a glucagon-like peptide-1 (GLP-1) receptor agonist, which has been approved to reduce the risk of sustained decline in estimated glomerular filtration rate (eGFR), end-stage renal disease, and cardiovascular death in adult patients with type 2 diabetes mellitus accompanied by chronic kidney disease.

Semaglutide injection has previously been approved in China for adult patients with type 2 diabetes (T2DM) whose blood glucose levels are not adequately controlled by diet, exercise, metformin, and/or sulfonylurea drugs. It is also indicated for reducing the risk of major adverse cardiovascular events in T2DM patients with cardiovascular disease. Additionally, it demonstrated potential renal protective effects in the cardiovascular outcomes trial SUSTAIN 6.Can reduce the risk of renal composite events by 36% in T2DM patients with cardiovascular disease or at high risk.

YKYY029 Injection: Approved for Clinical Trials in China

YKYY029 Injection Receives Approval in ChinaClinical Trial Implied Permission, intended for the development of hypertension treatment. This is independently developed by Yuekang Pharmaceutical's subsidiary, Yuekang Innovation, and Hangzhou Tianlong.AGTGeneTargetedSmall interfering RNA (siRNA) drugs, with an entirely new sequence.

YKYY029 Injection, as an siRNA drug, is unique in that it is conjugated with the GalNAc delivery system developed by Yuekang Pharmaceutical, enabling efficient liver-targeted delivery.This product utilizes the interference and silencing mechanism of siRNA to target and silence at the source.AGTmRNA, effectively blocking the synthesis of AGT protein.AGT protein is the upstream protein of the renin-angiotensin-aldosterone system (RAAS). Inhibiting its expression will fundamentally suppress the blood pressure-raising effects of the RAAS system, thereby reducing blood pressure.

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