Clinical Trial News

Rigel Pharmaceuticals announced the first patient enrolled in a Phase 1b/2 triplet therapy trial of REZLIDHIA (olutasidenib) combined with decitabine and venetoclax for mIDH1 AML, conducted by MD Anderson. The trial aims to determine safety, tolerability, and complete remission rates in newly diagnosed and relapsed/refractory patients.

Verastem, Inc. (VSTM) develops cancer treatments targeting the RAS/MAPK pathway, focusing on pancreatic, lung, colorectal, and low-grade serous ovarian cancers. Its leading therapy combines Avutometinib and Defactinib, with a rolling NDA submission expected to finalize by 2H2024 for recurrent LGSOC. VSTM also explores additional indications for metastatic pancreatic cancer and mKRAS G12C NSCLC. The company's tight cash runway makes it a high-risk, high-reward investment, but its current valuation and pathway to approval suggest a bullish outlook.

FDA grants orphan drug designation to certepetide for cholangiocarcinoma. Certepetide is being evaluated in phase 2 BOLSTER trial with SOC therapies. It enhances drug delivery and tumor microenvironment modulation. BOLSTER trial includes first- and second-line treatments with certepetide. Certepetide also has ODD for malignant glioma, osteosarcoma, and pancreatic cancer.

UltraDx Bio's UD-X™ Single-Molecule Analyzer received China's first clinical registration approval, revolutionizing early screening for Alzheimer's Disease. The analyzer, listed on the National Medical Products Administration website, detects trace protein biomarkers at the fg/ml scale, addressing precision detection needs in clinical diagnostics.

FDA approved seven new therapies in August, including a competitor to Dupixent for prurigo nodularis, Gilead’s PPARδ agonist, and targeted cancer therapies.

Pyrotinib plus metronomic vinorelbine showed safety and efficacy in HER2+ advanced breast cancer post-trastuzumab, with median PFS of 13.5 months and ORR of 38.9%. The study highlights the need for alternative therapies due to economic constraints on ADCs. Subgroup analysis revealed better PFS in patients with lung metastases and primary trastuzumab resistance. No new AEs were reported, and the regimen warrants further study.

Bispecific antibody drug conjugates (BsADCs) represent a cutting-edge advancement in cancer therapy, addressing tumor resistance and heterogeneity with dual-targeting mechanisms. Over 60 BsADCs are currently in clinical trials, offering targeted, multi-faceted treatment with reduced side effects. Recent technological advancements have enabled efficient production, expanding their potential applications beyond cancer to autoimmune and infectious diseases.

Scientists at La Trobe University discovered WD34, an antibody-like molecule that binds to AMA1, inhibiting malaria parasite infection. WD34 targets a conserved region in AMA1, making it effective against multiple malaria species, offering hope for a broad-spectrum vaccine. Pre-clinical trials show promising results.

Vanda Pharmaceuticals' stock rose 1.8% since April, lagging the S&P 500's 7% gain. Fanapt, approved for bipolar 1 disorder, faces market challenges due to lack of differentiation from generic antipsychotics. Upcoming catalysts include FDA decisions on tradipitant for gastroparesis and motion sickness, and a Phase I study for a CFTR inhibitor to treat cholera. Vanda's cash position is strong, but its product pipeline is weak, with potential revenue declines from Hetlioz and Fanapt due to generic competition. The company rejected takeover bids, emphasizing the need for strategic cash management.

Vorasidenib, a dual inhibitor of IDH1 and IDH2 mutations, offers a breakthrough in treating IDH-mutated, grade two glioma, significantly improving progression-free survival and crossing the blood-brain barrier effectively.