• Investigational BKX-001 is an oral small-molecule activator of BAX, developed as a potential treatment for leukemia, lymphoma and solid tumors¹
• Ipsen obtains an exclusive license to research, develop, manufacture and commercialize novel therapeutics targeted to the BCL-2 associated protein (BAX), an effector of apoptosis

• Ipsen and BAKX Therapeutics Inc. will be jointly responsible for research and development activities and will jointly share in the profits from commercialization

Paris (France), 27 July 2021 – Ipsen (Euronext: IPN; ADR: IPSEY) and BAKX Therapeutics Inc. have signed an exclusive worldwide-collaboration agreement to research, develop, manufacture and commercialize BKX-001 as a potential treatment for leukemia, lymphoma and solid tumors.

Apoptosis is the naturally occurring process of programmed cell death. Deregulated apoptosis can lead to uncontrolled cell division and the development of a tumor.² The apoptosis cell-signaling pathway has been proven to be a target for cancer therapy with the development and approval of BCL-2 inhibitors for the treatment of certain hematological malignancies.³ BAX is a novel target in this pathway that is downstream of all anti-apoptotic proteins like BCL-2, BCL-XL, MCL-1 etc.⁴ Direct activation of BAX has several possible outcomes as an investigational cancer therapy, potentially addressing multiple tumor types and the resistance encountered while targeting only the antiapoptotic BCL-2 family proteins. BAX activation by small-molecule agonists have been shown to promote apoptosis in leukemia-cell lines and human samples, while sparing healthy cells in vitro and suppress human acute myeloid leukemia xenografts and increased host survival without toxicity in vivo.⁵

Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen, said “As part of our strategy, we continue to strengthen our pipeline and deliver exciting external-innovation opportunities. We are, therefore, delighted to partner with the expert team at BAKX Therapeutics to move BKX-001 into further preclinical development, with the goal of achieving a development candidate that can be evaluated for the potential treatment of hematological malignancies and solid tumors. Importantly, this collaboration also reflects the shared values across our two organizations and, as a result, we will be building a strong cross-company team to advance this innovation for people living with these forms of cancer, and their healthcare teams.”

Sree Kant, Founder and CEO of BAKX Therapeutics Inc. said “We are excited to partner with Ipsen on investigational BKX-001. This program is the result of pioneering work on the BAX protein driven by our scientific co-founders Loren D Walensky and Evripidis Gavathiotis. This partnership brings together Ipsen’s excellent clinical development and commercial capabilities with our industry-leading knowledge of the BAX protein and our unique computational platform. This is a collaboration of very distinct complementary capabilities that can together drive important treatment options in cancer therapy for patients, faster.”

Under the agreement, Ipsen will pay BAKX Therapeutics Inc. $14.5m upon closing, comprising an equity investment and an upfront payment, followed by up to $837.5m in milestone payments. The companies would also share equally costs and profits.