Zanidatamab

FDA reviews 5 drugs with PDUFA dates in Nov 2024: DFD-29 for rosacea, eladocagene exuparvovec for AADC deficiency, inavolisib for PIK3CA-mutated HR-positive breast cancer, acoramidis for transthyretin amyloid cardiomyopathy, and zanidatamab for HER2-amplified biliary tract cancer.

Jazz Pharmaceuticals (JAZZ) is a global biopharmaceutical company focusing on sleep, hematology/oncology, and pain, with notable products like Xyrem, Xywav, Zepzelca, Rylaze, and Epidiolex. In 2023, it achieved $3.8 billion in revenue, completed a Phase 3 trial for Zepzelca, and advanced zanidatamab development. With $1.6 billion in cash and $5.8 billion in debt, Jazz continues to innovate through R&D and strategic acquisitions, partnering with BeiGene and Life Science Cares.

DelveInsight's 'Breast Cancer Pipeline Insight 2024' highlights a robust pipeline with 100+ companies developing 120+ drugs, driven by rising incidence, tech advancements, and increased awareness. Key companies and drugs include Ambrx, CSPC ZhongQi, Merus, and ARX788, DP303c, SHR-A1811, among others. FDA fast-track designations and breakthrough therapy designations for drugs like 9MW2821 and inavolisib underscore significant progress.

Zanidatamab (ZW25) plus mFOLFOX6-2 with or without bevacizumab showed 83.3% and 100% confirmed overall response rates, respectively, in a phase 2 study for HER2-positive mCRC, with manageable safety profile and no treatment-related deaths.

ALX Oncology Holdings Inc. develops Evorpacept [ALX148], a first-in-class CD47 inhibitor with an inactive Fc domain reducing side effects. ASPEN-06 Phase 2 trials combining Evorpacept with trastuzumab, ramucirumab, and paclitaxel (TRP) showed favorable results in HER2-positive gastric and gastroesophageal junction (GEJ) cancer. Evorpacept has FDA Fast Track designations for several indications, making the shares potentially bullish.

Jazz Pharmaceuticals to present five abstracts at ESMO Congress 2024, including new data on zanidatamab for HER2-positive mGEA and mCRC, and Zepzelca for relapsed SCLC.

Jazz Pharmaceuticals completes BLA submission for zanidatamab, a HER2-targeted bispecific antibody, seeking accelerated approval for treatment of previously-treated HER2-positive metastatic biliary tract cancer. If approved, zanidatamab would be the first HER2-targeted treatment for this indication in the U.S. The BLA includes data from the Phase 2b HERIZON-BTC-01 trial showing a confirmed objective response rate of 41.3% in HER2-positive BTC patients, with a manageable safety profile.