Recent Advances in Drug Development Across Three Key Therapeutic Areas: Immune Targeting, Oncology Combination Therapies, and Metabolic Disorders

(Source: ACROBiosystems Official)

Source: ACROBiosystems Official

Introduction

Recently, the global pharmaceutical field has witnessed several significant advancements. Johnson & Johnson's oralIL-23Inhibitor Successfully Defeats Deucravacitinib in Phase III Head-to-Head Study for Psoriasis; Roche Announces $3.5 Billion Acquisition of 89bio, Entering the MAFLD Track; Regeneron Reports "Near-Complete Suppression" in Phase III Clinical Data for Rare Disease FOP. Meanwhile, Multiple New Drugs Produced in China Gain Clinical Approval or Submit for Marketing Authorization in Oncology, Autoimmune Diseases, and Metabolic Disorders.

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Autoimmune Diseases

Oral Formulations and Precision Medicine Advance Together

GENRIX BIOTargeting Skin Immunity, Anti-IL-15 Monoclonal Antibody Approved for Clinical Trials

On September 16, GR2301 Injection, independently developed by GENRIX BIO, received clinical trial approval from the National Medical Products Administration. This is the first GR2301 Injection produced in China.IL-15Targeted Biologics: No Similar Antibody Drugs Approved Worldwide, Expected to Become the World's First IL-15 Targeted Drug for Vitiligo. Traditional vitiligo treatments (such as phototherapy and topical steroids) have limited efficacy and are prone to recurrence. The core challenge lies in the persistent presence of tissue-resident memory T cells, with IL-15 being crucial for their survival. GR2301 can block the IL-15-related pathway, reducing recurrence at its root and offering potential advantages such as long-term remission and higher safety. In the future, it may be expanded to other autoimmune diseases. Additionally, IL-15 targeted drugs like IBI3013 from Innovent Bio and innovative vitiligo therapies such as Ruxolitinib cream are also advancing.

Johnson & Johnson Oral IL-23 InhibitorPhase III Clinical Trial Defeats Star Drug, Aiming at Psoriasis Market

On September 17, Johnson & Johnson announced its first-in-class oralIL-23RPositive Results from Two Phase III Studies (ICONIC-ADVANCE 1 and 2) of Targeted Peptide Icotrokinab. In a head-to-head study for the treatment of moderate to severe plaque psoriasis, the drug not only showed significantly superior skin clearance rates (PASI 90 and IGA 0/1) compared to the placebo group at week 16 but also outperformed the current oral standard medication, deucravacitinib (Sotyktu®), at both weeks 16 and 24. Icotrokinab demonstrated good safety and is expected to provide patients with a new convenient oral treatment option.

Novartis' Complement Inhibitor Iptacopan Achieves Another Approval in ChinaNew Indication for IgA Nephropathy

On September 18, Novartis announced that its oral complement factor B inhibitor, Iptacopan, received approval from the National Medical Products Administration (NMPA) for an additional indication to treat adult patients with primary IgA nephropathy who are at risk of rapid progression. As an oral drug that precisely targets the alternative pathway (AP) of the complement system, Iptacopan alleviates renal inflammation and damage by inhibiting excessive activation of AP through upstream mechanisms, offering a novel treatment option with the first non-immunosuppressive mechanism for IgA nephropathy. Results from Phase III clinical trials showed that Iptacopan significantly reduced proteinuria levels by 38.3% in patients while demonstrating good safety, potentially providing a new oral treatment option for IgA nephropathy patients in China.

Mechanism of Action of Ipkapane

Oncology Treatment Field

Combined Therapy and Novel Mechanisms Advance Together

Hengrui MedicineIL-15 Superagonist or Breakthrough Therapy Designation, Tackling Bladder Cancer

On September 17, SHR-1501 Injection, a Class 1 new drug independently developed by Hengrui Medicine, received breakthrough therapy designation from the National Medical Products Administration (NMPA). The drug is intended to be used in combination with Bacillus Calmette-Guérin (BCG) for the treatment of patients with non-muscle-invasive bladder cancer who do not respond to BCG. These patients have historically faced the dilemma of bladder removal, while the mechanism of action of SHR-1501 lies in stimulating immune cell proliferation and enhancing immune response, which is expected to change this treatment landscape. Phase 1/2 study data presented at the American Society of Clinical Oncology (ASCO) conference in May this year showed that among evaluable patients, the complete response rates at 3 months and 6 months after receiving SHR-1501 combined with BCG treatment were as high as 90.9%. Currently, the drug is still further exploring its potential applications as a monotherapy or in combination with other immunotherapeutic agents, offering hope to provide better treatment options for bladder cancer patients.

Results of the Phase 1/2 Study of SHR-1501

Lepu BiotechEGFR ADC Combination Therapy Receives Breakthrough Treatment Designation, Targeting Refractory Nasopharyngeal Carcinoma

On September 19, the CDE website announced that MRG003, an EGFR-targeted ADC drug developed by Lepu Biopharma, is proposed to be granted Breakthrough Therapy Designation for the treatment of patients who have previously received platinum-based therapy and PD-1/PD-L1 inhibitors, in combination with Putulizumab (PD-1 antibody).PD-L1Recurrent or metastatic nasopharyngeal carcinoma that has failed inhibitor therapy. MRG003 is currently the globally leading EGFR ADC drug in development and has previously received Breakthrough Therapy Designation and Priority Review status from the CDE, as well as Orphan Drug Designation, Fast Track Designation, and Breakthrough Therapy Designation from the FDA. In September 2024, Leap Therapeutics submitted the first marketing application for the drug's use in nasopharyngeal carcinoma, which was voluntarily withdrawn to supplement additional data. It was resubmitted in March this year and has now been included in the priority review process for monotherapy treatment of specific recurrent/metastatic nasopharyngeal carcinoma patients.

Mechanism of Action of MRG003

Metabolism and Rare Diseases

Blockbuster Deals and Disruptive Therapies Emerge

Regeneron Announces "Near-Complete Suppression" Data, FOP Therapy Application Imminent

On September 17, Regeneron announced its targetedActivin AThe monoclonal antibody Garetosmab (REGN2477) successfully achieved the primary endpoint in the Phase III study for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Data showed that doses of 3 mg/kg and 10 mg/kg reduced the number of new heterotopic ossification (HO) lesions by 94% and 90%, respectively, and decreased the total volume of new HO lesions by more than 99%, achieving nearly complete inhibition. Regeneron plans to submit a marketing application for this drug for FOP indication to the U.S. FDA by the end of 2025 and expects to file applications with other global regulatory agencies in 2026. If approved, Garetosmab will become the first effective therapy for this fatal rare disease.

Mechanism of Action of REGN2477

New Choice for Weight Loss and Blood Sugar Reduction:ZHIFEI BIOCA111 Injection Enters Clinical Stage

On September 17, CA111 Injection developed by Chenan Bio, a subsidiary of ZHIFLY BIO, was approved to enter the clinical trial stage. The product isGIPR/GLP-1RDual-target agonist for long-term weight management in adults. Its dual mechanism of action produces a synergistic effect, with the potential for superior performance in terms of efficacy and safety. Compared to single-target drugs, CA111 injection reduces adverse reactions during treatment due to the complementary effects of the dual agonist. Currently, the only GIP/GLP-1 dual receptor agonist approved for marketing in China is Eli Lilly's tirzepatide injection, with no domestically produced equivalent available yet. CA111 injection from Chenan Bio is a Class 1 innovative chemical drug that has obtained effective invention patents related to molecular structure and application. It primarily targets high-demand areas such as diabetes and weight control, showing strong market development potential.