JAZZ PHARMACEUTICALS FRANCE

Researchers at Icahn School of Medicine identified lurbinectedin's mechanism, activating STING-IFN signaling to boost immune response against small-cell lung cancer, enhancing PD-L1 blockade therapy efficacy.

Jazz Pharmaceuticals' ZIIHERA® approved for HER2-positive biliary tract cancer; Merus's BIZENGRI® approved for NRG1+ pancreatic adenocarcinoma and NSCLC; AstraZeneca's IMFINZI® approved for limited-stage small cell lung cancer and under Priority Review for muscle-invasive bladder cancer.

Jazz Pharmaceuticals announced CEO Bruce Cozadd's retirement plan; he will stay on until a successor is found within a year. Cozadd co-founded Jazz in 2003 and led it through significant growth, including the $7 billion acquisition of GW Pharmaceuticals. The company remains on track to meet its 2024 revenue guidance of $4 billion to $4.1 billion, with a focus on Xywav and Epidiolex. A special committee of independent directors will lead the search for Cozadd's replacement, considering both internal and external candidates.

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 global Acute Myeloid Leukemia Therapeutics Market is projected to grow at a CAGR of 13.4% during 2024-2032, driven by advances in targeted therapies and immunotherapies.

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.

The Justice Department's Consumer Protection Branch, with over 120 attorneys and staff, works to protect Americans' health, safety, and economic security by enforcing laws related to products like tobacco, food, medical devices, pharmaceuticals, and dietary supplements. The branch collaborates with various agencies, including the FDA, to hold accountable those who threaten public safety through unlawful practices, such as distributing tainted food, selling adulterated medical products, or falsifying clinical trial data. Recent actions include prosecuting opioid manufacturers and enforcing regulations on e-cigarettes to protect youth. The branch also defends FDA's medical and scientific judgments in court, ensuring that the agency's expertise is respected.

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.

NCCN adds Jazz Pharmaceuticals' Ziihera as a category 2A treatment for biliary tract cancers (BTC). Ziihera, a dual HER2-targeted bispecific antibody, received FDA accelerated approval for previously treated, HER2-positive BTC. The approval was based on a 41.3% confirmed objective response rate in a Phase IIb trial, with a median overall survival of 15.5 months. Ziihera offers a chemotherapy-free option for BTC patients with limited treatment options.

Ziihera, the first FDA-approved dual HER2-targeted bispecific antibody for HER2+ (IHC 3+) BTC, is now available in the U.S. It is recommended by the NCCN Guidelines as a category 2A treatment option for BTC, based on a 52% objective response rate and median duration of response of 14.9 months from the HERIZON-BTC-01 trial. Continued approval may depend on verification of clinical benefit in a confirmatory trial.