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Kelun-Biotech's Next-Generation RET Inhibitor A400/EP0031 Receives NDA Acceptance in China for NSCLC Treatment

Targeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncologyTargeted TherapyOncology
  • Sichuan Kelun-Biotech announced that China's NMPA accepted its New Drug Application for A400/EP0031, a next-generation selective RET inhibitor for treating RET-fusion positive non-small cell lung cancer.
  • The acceptance is based on positive Phase 2 results from the KL400-I/II-01 study, which demonstrated favorable efficacy in both treatment-naïve and pretreated NSCLC patients, including those with brain metastases.
  • A400/EP0031 previously received FDA Fast Track designation in March 2024 and is being developed globally through a partnership with Ellipses Pharma for markets outside Greater China.
  • The drug addresses an unmet medical need for RET-fusion positive NSCLC patients, who represent 1.4% to 2.5% of Chinese NSCLC cases and derive limited benefit from conventional treatments.

A Study of KL590586 in Patients With Advanced Solid Tumors

NCT05265091Active, Not RecruitingPhase 1
Sichuan Kelun Pharmaceutical Research Institute Co., Ltd.
Posted 6/1/2021

A Study of EP0031 in Patients With Advanced RET-altered Malignancies

NCT05443126RecruitingPhase 1
Ellipses Pharma
Posted 9/30/2022

Nuclide Therapeutics Secures £5M to Advance ALDH1A1-Targeted Radiotheranostics for Therapy-Resistant Cancers

Targeted TherapyOncology
  • King's College London spin-out Nuclide Therapeutics has closed a £5 million investment round from Marathon Beteiligungs AG to advance radiotheranostic treatments for therapy-resistant cancers.
  • The funding will support development of ALDH1A1-targeted radiotheranostics, radioactive drugs designed to identify and eliminate cancer cells that have evaded conventional therapies.
  • The company's pipeline has demonstrated exceptional preclinical data in lung and ovarian cancers, with plans to progress lead candidates into first-in-human trials.
  • The milestone-based investment will enable expansion into additional cancer indications and acceleration of other assets in the discovery pipeline.

Precision Biologics Reports Promising Efficacy Data for Novel Ovarian Cancer ADC PB-vcMMAE-5

Monoclonal AntibodyTargeted Therapy
  • Precision Biologics' novel antibody-drug conjugate PB-vcMMAE-5 demonstrated significant anti-tumor activity in ovarian cancer models, achieving up to 92.51% cell killing in vitro and robust tumor volume reduction at higher doses.
  • The ADC targets truncated core 2 O-glycans specifically expressed by cancer cells using the improved monoclonal antibody PB-223, which shows 4-fold stronger binding affinity compared to its predecessor NEO-102.
  • Safety studies in mice showed the treatment was well-tolerated with no signs of distress or significant organ toxicity, supporting its potential for clinical development in ovarian and other cancers expressing core 2 O-glycans.

Advanced Liver Cancer Pipeline Shows Robust Growth with 50+ Companies Developing Novel Therapies

Targeted TherapyCAR-T
  • DelveInsight's 2025 pipeline report reveals over 50 active companies developing 52+ advanced liver cancer therapies, indicating strong industry commitment to addressing this challenging malignancy.
  • Recent regulatory milestones include Tempest Therapeutics receiving FDA fast track and orphan drug designations for amezalpat, while Bayer initiated Phase I trials for targeted alpha radiopharmaceutical BAY 3547926.
  • The pipeline features diverse therapeutic approaches including immunotherapy combinations, targeted therapies, and novel mechanisms like STAT3 inhibitors and CAR-T cell therapies.
  • Advanced liver cancer affects patients with disease spread beyond the liver, requiring systemic treatments focused on slowing progression and maintaining quality of life with median survival often limited to months.

Brepocitinib Achieves First-Ever Positive Phase 3 Results in Dermatomyositis, Setting New Treatment Standard

Rare DiseaseTargeted Therapy
  • Roivant and Priovant's brepocitinib demonstrated statistically significant improvement over placebo in the Phase 3 VALOR study, achieving a mean Total Improvement Score of 46.5 versus 31.2 for placebo (p=0.0006) at 52 weeks.
  • The dual TYK2/JAK1 inhibitor showed rapid onset of action with separation from placebo as early as week 4, while enabling 62% of patients to reduce steroid use to ≤2.5 mg/day compared to 34% on placebo.
  • This represents the first positive 52-week placebo-controlled trial and first successful registrational study for a targeted therapy in dermatomyositis, addressing a critical unmet need in this debilitating autoimmune disease.
  • An NDA filing is planned for the first half of 2026, positioning brepocitinib to potentially become the first approved targeted therapy for dermatomyositis patients.

Seven Real-World Studies Demonstrate NEFECON's Efficacy in IgA Nephropathy Treatment Across Diverse Patient Populations

Real World EvidenceDrug ApprovalTargeted Therapy
  • Seven new real-world evidence studies from leading Chinese hospitals will be presented at the 18th International IgA Nephropathy Symposium, demonstrating NEFECON's efficacy and safety across diverse patient populations including pediatric cases and those with renal insufficiency.
  • The studies validate NEFECON's position as the world's first etiological treatment for IgA nephropathy, showing significant proteinuria reduction and renal function preservation with extended treatment beyond 9 months.
  • NEFECON has achieved regulatory approval from major agencies including FDA, EMA, and NMPA, and was included in China's National Reimbursement Drug List in November 2024, reinforcing its role as first-line cornerstone therapy for IgAN patients.

FORE Biotherapeutics Reports Durable Clinical Benefit with Plixorafenib in BRAF-Altered Thyroid Cancers

Targeted TherapyOncology
  • FORE Biotherapeutics presented Phase 1/2a data showing plixorafenib achieved a median progression-free survival of 64 months and 85.7% clinical benefit rate in MAPK inhibitor-naïve BRAF V600-mutated papillary thyroid cancer patients.
  • The novel BRAF inhibitor demonstrated durable disease control in both papillary and anaplastic thyroid cancers, with four patients remaining on treatment for over five years without requiring combination with MEK inhibitors.
  • The company is advancing the registrational FORTE Phase 2 basket trial evaluating plixorafenib across multiple BRAF-altered tumor types, including thyroid cancers where BRAF alterations occur in 45-60% of cases.

A Study to Assess the Efficacy and Safety of FORE8394 in Participants With Cancer Harboring BRAF Alterations

NCT05503797RecruitingPhase 2
Fore Biotherapeutics
Posted 2/21/2023

Rybrevant Plus Lazcluze Combination Demonstrates Superior Overall Survival in First-Line EGFR-Mutated NSCLC

Targeted Therapy
  • Rybrevant (amivantamab-vmjw) plus Lazcluze (lazertinib) showed statistically significant and clinically meaningful overall survival improvement in previously untreated locally advanced or metastatic NSCLC patients.
  • The phase III Mariposa study demonstrated efficacy in patients with EGFR exon 19 deletions or L858R substitution mutations.
  • Tagrisso (osimertinib) combined with pemetrexed and platinum-based chemotherapy also showed significant OS improvement compared to Tagrisso monotherapy in first-line treatment.
  • These results represent important advances in targeting EGFR-mutated non-small cell lung cancer with combination therapeutic approaches.

Lutetium Lu 177 Dotatate Shows Promise in Metastatic Bronchopulmonary Neuroendocrine Tumors

Real World EvidenceDrug ApprovalTargeted Therapy
  • Lutetium Lu 177 dotatate demonstrated antitumor activity in patients with metastatic bronchopulmonary neuroendocrine tumors, achieving partial responses in 17% of patients in a real-world study.
  • The median progression-free survival was 10.8 months across all patients, with patients having higher Krenning scores showing better outcomes at 14.7 months.
  • The treatment was well tolerated with no grade 3 or higher adverse events, with the most common side effects being fatigue, nausea, and abdominal pain.

Mednovo Doses First Patient in Phase III Trial of Lutetium-177 Radiotherapy for Neuroendocrine Tumors

Targeted Therapy
  • Mednovo Group successfully dosed the first patient in its Phase III clinical trial of Lutetium [177Lu] Oxodotreotide Injection for gastroenteropancreatic neuroendocrine tumors on August 22, 2025.
  • The targeted radiotherapeutic drug works by binding to somatostatin receptors and represents a pivotal advancement in treating GEP-NETs with near-commercial production standards.
  • C-Ray Therapeutics served as the exclusive manufacturing partner, utilizing China's first fully automated radiopharmaceutical production line built to global cGMP standards.
  • The Phase III milestone demonstrates significant progress in radiopharmaceutical development, with C-Ray supporting over 50 CRDMO projects across the full drug development lifecycle.

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