Subgroup Analysis from Phase 3 Clinical Trial Supports Efficacy of Maribavir Over Conventional Therapies in Transplant Recipients With Cytomegalovirus Infection (Refractory, With or Without Resistance)

• More Than Three Times as Many Transplant Recipients With Confirmed Resistant Cytomegalovirus (CMV) Infection at Baseline Receiving the Investigational Drug Maribavir Achieved CMV Viremia Clearance Compared to Conventional Antiviral Therapies, Building on Previously Presented Results Supporting the Efficacy of Maribavir
  
• Transplant Recipients Receiving Maribavir Experienced Lower Rates of Treatment-Related Neutropenia and Acute Kidney Injury Compared to Conventional Antiviral Therapies
  
• Takeda Continues to Investigate Maribavir for the First-Line Treatment of CMV in Hematopoietic Cell Transplant Recipients in an Ongoing Phase 3 Clinical Trial

OSAKA, Japan, March 15, 2021 –Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) announced the results from a subgroup analysis of the Phase 3 TAK-620-303 (SOLSTICE) trial, for the investigational drug TAK-620 (maribavir), which supported the efficacy results from the overall randomized population. More than three times as many (62.8%; 76/121) transplant recipients with confirmed genotypic resistant CMV infection at baseline treated with maribavir achieved confirmed CMV viremia clearance at Study Week 8 (end of treatment phase) compared to those treated with conventional antiviral therapies (20.3%, 14/69) (investigator assigned treatment; IAT consists of one or a combination of ganciclovir, valganciclovir, foscarnet or cidofovir) (adjusted difference [95% CI]: 44.1% [31.3, 56.9]).¹

Findings from the overall trial population showed the study met its primary endpoint, demonstrating that maribavir was superior to conventional antiviral therapies in CMV viremia clearance at Study Week 8. Specifically, 55.7% (131/235) of transplant recipients with refractory, with or without resistance (R/R), CMV infection/disease treated with maribavir achieved confirmed CMV viremia clearance as compared to 23.9% (28/117) of those on conventional antiviral therapies (adjusted difference [95% CI]: 32.8%, [22.8, 42.7]; p<0.001).^1*†‡

“Transplant recipients with CMV infections resistant to conventional antiviral therapies are some of the hardest to treat. Current treatment options are limited, and hematologist-oncologists have to engage in a careful balance of viral clearance and side effect management,” said Dr. Rafael Duarte, Hospital Universitario Puerta de Hierro, Madrid. “We believe these data are important as they build on previously presented results supporting the potential of maribavir, which, if approved, could transform the management of CMV in these patients."

Transplant recipients receiving maribavir exhibited lower incidence of treatment-related toxicities common with conventional antiviral therapies. Those receiving maribavir experienced lower rates of treatment-related neutropenia vs. valganciclovir/ganciclovir (1.7% [4/234] vs. 25% [14/56]) and acute kidney injury vs. foscarnet (1.7% [4/234] vs. 19.1% [9/47]). Incidence of any treatment-emergent adverse events (TEAEs) was 97.4% (228/234) for maribavir and 91.4% (106/116) for the conventional therapy group.¹ The most common TEAEs in the maribavir group were dysgeusia (35.9%, 84/234), nausea (8.5%, 20/234) and vomiting (7.7%, 18/234).² Incidence of TEAEs leading to study drug discontinuation was 13.2% (31/234) in the maribavir group and 31.9% (37/116) in the conventional therapy group.¹ Two treatment-related serious TEAEs led to death (1 patient per treatment group).¹

“Current CMV management is associated with difficult tradeoffs, including management of toxicities and viremia clearance,” said Obi Umeh, MD, Vice President and Maribavir Global Program Leader, Takeda. “As we continue our research of maribavir, an oral antiviral compound, across patient populations, we are committed to addressing this unmet need so physicians potentially have an additional treatment option.”