Jazz Pharmaceuticals (JAZZ) investor relations material

Oncology pipeline and zanidatamab developments

• Zanidatamab, a biparatopic HER2 antibody, shows synergy with immune checkpoint inhibitors and has demonstrated statistically significant overall survival benefits in both triplet and doublet regimens for gastric and biliary tract cancers.

• Ongoing pivotal trials target frontline biliary tract cancer, late-line breast cancer, and a pan-tumor basket trial aiming for a tumor-agnostic label, with promising activity in colorectal and non-small cell lung cancers.

• Breakthrough designation was achieved for colorectal cancer, and discussions with the FDA are ongoing for rapid approval pathways in additional indications.

• Commercial strategy focuses on replacing trastuzumab in first-line gastric cancer, promoting triplet regimens, and expanding the addressable patient population through education and market growth.

• Future breast cancer plans include moving zanidatamab into earlier lines and combining with novel HER2 TKIs, with collaborations underway and a focus on de-escalation strategies in curative settings.

Epilepsy and neuroscience portfolio

• EPIDIOLEX remains a key asset with exclusivity into the late 2030s, supported by new clinical trials in focal onset seizures, juvenile myoclonic epilepsy, and expanded studies in LGS and other DEEs.

• Efforts are underway to broaden the EPIDIOLEX label to cover all DEEs, with ongoing discussions with the FDA and new evidence generation studies.

• The Actio acquisition adds a targeted therapy for rare epilepsy caused by KCNT1 mutations, with promising preclinical and clinical data and a focus on personalized medicine.

• Pipeline includes a phase I Kv7 opener for absence epilepsy and a next-generation orexin 2 agonist for sleep disorders, with IND clearance and healthy volunteer studies planned.

Commercial and strategic outlook

• Oncology and epilepsy are expected to remain core pillars, with rare disease expertise driving future pipeline growth and strategic acquisitions.

• Market expansion is anticipated through increased HER2 therapy utilization, new indications, and tumor-agnostic approvals.

• The company aims for $3–5 billion in peak sales for zanidatamab, leveraging its differentiated mechanism and broad development strategy.

• Sleep-wake franchise durability is supported by Xywav’s low sodium profile and dosing flexibility, while orexin agonists are positioned as complementary rather than replacement therapies.

• The rare disease strategy is expected to shape the pipeline through 2030, building on proven capabilities in oncology, epilepsy, and sleep disorders.