Zanidatamab

MSKCC advanced cancer treatments in 2024 include new surgical techniques, vaccines, drugs enhancing radiation, and diagnostic tools. The FDA approved 11 drugs based on MSK's pivotal clinical trials, covering new uses for existing drugs, effective drug combinations, engineered cell therapies, and novel targeted therapies. Key developments include a KRAS vaccine for pancreatic and colorectal cancer, combination drug-radiation treatment for basal cell carcinoma, noninvasive E-nose for lung cancer detection, improved transplant success with partially matched donors, exercise impact on prostate cancer biomarkers, new imaging for lung and prostate cancers, TIL therapy for non-small cell lung cancer, robotic neurosurgery for spinal tumors, avoiding surgery for HPV throat cancer with innovative radiation, imlunestrant for ER+, HER2- advanced breast cancer, mRNA vaccine for pancreatic cancer, and immunotherapy for rectal cancer. FDA approvals included tepotinib for METex14 lung cancer, CAR T treatment for mantle cell lymphoma, selpercatinib for RET-linked thyroid cancer, repotrectinib for NTRK fusion cancers, adagrasib with cetuximab for KRAS-G12C colorectal cancer, afamitresgene autoleucel for synovial sarcoma, vorasidenib for IDH glioma, inavolisib with fulvestrant and palbociclib for breast cancer, revumenib for KMT2A leukemia, zanidatamab for HER2 biliary tract cancer, and zenocutuzumab for NRG1 fusion pancreatic and lung cancers.

10 biotech companies to watch in 2025: Anavex Life Sciences (blarcamesine for neurodegenerative diseases), Candid Therapeutics (T cell engager antibodies for autoimmune diseases), Enterprise Therapeutics (ETD001 for cystic fibrosis), Jazz Pharmaceuticals (Ziihera for biliary tract cancer), Life Biosciences (ER-100 for optic neuropathies), Ovid Therapeutics (OV888 for cerebral cavernous malformation), Skye Bioscience (nimacimab for obesity), TG Therapeutics (Briumvi for multiple sclerosis), Wave Life Sciences (WVE-006 for alpha-1 antitrypsin deficiency), and Quotient Therapeutics (somatic genomics platform for drug discovery).

The FDA accelerated approval of zanidatamab-hrii (Ziihera) for previously treated HER2-positive biliary tract cancer fills a treatment gap. The phase 2b HERIZON-BTC-01 trial supported the approval with a 52% objective response rate and median duration of response of 14.9 months. Zanidatamab, a novel bispecific HER2-targeted antibody, offers a new treatment option for this difficult-to-treat disease, with ongoing evaluations in frontline therapy.

Jazz Pharmaceuticals received FDA approval for Ziihera, a HER2-targeted therapy for HER2+ biliary tract carcinoma, with a list price of $35,500 per cycle. Piper Sandler reaffirmed its Overweight rating and $163 price target, highlighting growth in the oncology segment. Jazz's strong financial health supports portfolio expansion, with anticipation for 2025 data on Ziihera in first-line HER2+ gastroesophageal adenocarcinoma.

Zanidatamab-hrii (Ziihera), a HER2-targeting bispecific antibody, is now a category 2A treatment option in NCCN guidelines for biliary tract cancer (BTC). FDA accelerated approval was based on HERIZON-BTC-01 trial results showing a 52% ORR and 14.9 months DOR. The trial enrolled 62 HER2+ BTC patients previously treated with gemcitabine. Updated results at 2024 ASCO showed median OS of 15.5 months, with higher rates in HER2 IHC 3+ patients. Zanidatamab inhibits tumor growth through multiple mechanisms and is the first chemotherapy-free option for BTC. Common AEs included diarrhea, infusion-related reactions, abdominal pain, and fatigue, with manageable safety profile. Ongoing trials aim to validate benefits and explore zanidatamab in other HER2-expressing cancers.

November 2024 saw numerous FDA approvals, designations, and clinical trial advancements in oncology, including revumenib for KMT2A-rearranged acute leukemia, obe-cel for relapsed/refractory B-cell ALL, and fast track designations for ALE.P02 in Claudin-1-positive tumors. Other highlights include orphan drug designations for LBL-024 in neuroendocrine cancer and elraglusib in Ewing sarcoma, and a new drug application for sunvozertinib in EGFR-mutant NSCLC.

Jazz Pharmaceuticals (JAZZ) posted Q3 top-and-bottom-line beats, projecting 2024 earnings above expectations, driven by sales growth from Xywav and Epidiolex. The company received accelerated approval for Ziihera, a treatment for HER2-positive biliary tract cancer, and anticipates phase 3 PFS results on Zanidatamab. JAZZ trades at a discount to peers, making it attractive for value investors.

The FDA approved PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody as a companion diagnostic for zanidatamab-hrii to assess HER2+ status in biliary tract cancer patients, identifying those eligible for zanidatamab treatment. This is the first FDA-approved companion diagnostic for HER2+ BTC, potentially improving clinical outcomes.

The FDA approved the PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody test to assess HER2-positive status in biliary tract cancer patients, identifying those eligible for zanidatamab-hrii treatment. Zanidatamab received accelerated approval for previously treated, unresectable or metastatic HER2-positive biliary tract cancer. The phase 2b HERIZON-BTC-01 trial supported the approval, showing a 52% ORR and 14.9-month median DOR in HER2 IHC 3+ patients. Long-term follow-up data maintained cORR and DCR, with a median OS of 15.5 months. Safety data indicated common TRAEs like diarrhea and infusion-related reactions, with no deaths attributed to zanidatamab.

Jazz Pharmaceuticals' zanidatamab (Ziihera) receives US FDA accelerated approval for HER2-positive biliary tract cancer, marking the first dual HER2-targeted bispecific antibody for this indication. Based on HERIZON-BTC-01 trial data, Ziihera shows a 52% objective response rate and 14.9-month median duration of response, positioning it as an effective treatment option. The approval underscores the growing role of bispecific antibodies in cancer therapy, with sales of such antibodies projected to exceed $40 billion by 2029.