FDA approve Ipsen's Onivyde for the first-line treatment of metastatic pancreatic adenocarcinoma

• Statistically significant improvement in OS and PFS for the NALIRIFOX arm over the Gem+NabP arm

• Median OS was 11.1 months in the NALIRIFOX arm and 9.2 months in the Gem+NabP arm [HR] 0.84, p-value 0.0403)

• Median PFS was 7.4 months in the NALIRIFOX arm and 5.6 months in the Gem+NabP arm HR 0.70]; p-value 0.0001

FDA Grants Full Approval for BALVERSA to Treat Locally Advanced or Metastatic Bladder Cancer with Select Genetic Alterations

• Statistically significant improvements in OS, PFS, and ORR were demonstrated for erdafitinib compared with chemotherapy. Median OS was 12.1 months (95% CI: 10.3, 16.4) for patients who received erdafitinib and 7.8 months (95% CI: 6.5, 11.1) for those who received chemotherapy (hazard ratio [HR] 0.64 [95% CI: 0.47, 0.88]; p-value=0.0050).

• Converts and modifies the indication of the 2019 Accelerated Approval, converting to traditional approval in 57 months.

FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma

• First FDA approval of a therapy intended to reduce the risk of relapse in pediatric patients with HRNB

• Efficacy was evaluated in an externally controlled trial comparing outcomes from Study 3b (investigational arm) and Study ANBL0032 (clinical trial-derived external control arm)

• EFS HR was 0.48 (95% CI: 0.27, 0.85) and OS HR was 0.32 (95% CI: 0.15, 0.70)

European Commission Grant Conditional MA to Pfizer’s ELREXFIO® for Relapsed and Refractory Multiple Myeloma

• CMA based on clinically meaningful response rates, duration of response, and safety from the Phase 2 MagnetisMM-3 trial

• ORR 61.0% (75/123); 35.0% ≥complete response. Fifty responders switched to biweekly dosing, and 40 (80.0%) improved or maintained their response for ≥6 months

• With median follow-up of 14.7 months, median duration of response, PFS & OS (secondary endpoints) have not been reached

FDA grants accelerated approval to pirtobrutinib for 3L CLL/SLL

• Approval based on BRUIN (NCT03740529], an open-label, single-arm, multicohort trial (N=108)

• ORR was 72% (95% CI: 63, 80), DOR was 12.2 months (95% CI: 9.3, 14.7). All responses were partial responses

FDA approves repotrectinib for ROS1-positive non-small cell lung cancer

➤ First FDA approval that includes patients with ROS1-positive NSCLC who have previously received a ROS1 tyrosine kinase inhibitor (TKI), in addition to patients who are TKI naïve

➤ Traditional approval was granted on one single-arm study, utilizing high and durable responses for approval - 79% ORR in the ROS1 TKI naïve group, median DOR was 34.1 months; 38% ORR in those receiving prior treatment with a ROS1 inhibitor, median DOR 14.8 months

Truqap (capivasertib) plus Faslodex approved in the US for patients with advanced HR-positive breast cancer

➤ First-in-class AKT inhibitor has potential to reshape treatment for breast cancer patients with specific biomarker alterations (PIK3CA, AKT1 or PTEN)

➤ Truqap+Faslodex reduced the risk of disease progression or death by 50% vs.Faslodex alone in patients with tumours harbouring PI3K/AKT pathway biomarker alterations (HR 0.50, 95% confidence interval 0.38-0.65; p=<0.001; median PFS 7.3 versus 3.1 months)

BeiGene Receives European Commission Approval for BRUKINSA® (zanubrutinib) for the Treatment of Relapsed or Refractory Follicular Lymphoma

• BRUKINSA is the first and only BTK inhibitor approved for follicular lymphoma in the European Union

• ORR was 69.0% in the BRUKINSA plus obinutuzumab arm versus 45.8% in the obinutuzumab arm (P = 0.0012), with a median follow-up of approximately 20 months

• Responses were durable with an 18-month DOR of 69.3% in the BRUKINSA combination arm

• Median PFS was 28.0 months vs. 10.4 months for obinutuzumab alone (HR: 0.50 [95% CI: 0.33, 0.75]; P = 0.0007)