Aeterna Zentaris Inc. and Yakult Honsha Co., Ltd. announced the signing of an exclusive development, commercialization and licensing agreement for the development, registration and marketing of perifosine, Aeterna Zentaris’ lead anti-cancer compound, for Japan. Perifosine, a novel oral PI3K/Akt inhibitor, is currently in two phase 3 programmes for the treatment of colorectal cancer and multiple myeloma in the United States and Europe.

Under the terms of the agreement, Aeterna Zentaris will receive an initial 6 million Euro (US$ 8.3 M) upfront payment and will be entitled to receive additional up to 44 million Euro (US$ 60.9 M) payments upon achieving certain pre-established milestones including clinical and regulatory events in Japan. Furthermore, Aeterna Zentaris will be supplying perifosine to Yakult Honsha on a cost-plus-basis and be entitled to receive double-digit royalties on future net sales of perifosine in Japan. Yakult Honsha will be responsible for the development, registration and commercialization in Japan.

“We are delighted to have a partner such as Yakult Honsha who has a proven track record in oncology and particularly in colon cancer, one of the main indications being targeted with perifosine. This partnership marks another important milestone in our quest to bring perifosine to market worldwide which could provide cancer patients with a novel treatment option. Furthermore, perifosine could generate significant long-term revenue for Aeterna Zentaris while building value for our shareholders,” said Juergen Engel, PhD, president and CEO of Aeterna Zentaris, and continued, “We have now out-licensed perifosine rights to Keryx Biopharmaceuticals for North America, to Handok for Korea and to Yakult Honsha for Japan, while Aeterna Zentaris retains all rights for the rest of the world. In order to ensure a worldwide timely development of perifosine, Aeterna Zentaris and all its partners have agreed to free access to all data.”

Shigeyoshi Sakamoto, head, pharmaceutical division/managing director, member of the board of Yakult Honsha, said, “We are delighted to have added perifosine to our pipeline in oncology which we have positioned as our key therapeutic domain. We look forward to pursuing development and commercialization of perifosine in Japan, by appropriately and efficiently utilizing the data of ongoing phase III trials in the United States and Europe for patients with colorectal cancer and multiple myeloma. This partnership will enable us to further strengthen our initiative in the field of colorectal cancer and to embark on new fields including multiple myeloma.”

Perifosine is a novel, oral anticancer treatment that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway. The product works by interfering with membranes of cancer cells thereby inhibiting Akt signalling which then affects cell death, growth, differentiation and survival. Perifosine, in combination with chemotherapeutic agents, is currently being studied for the treatment of colorectal cancer, multiple myeloma and other cancers, and is the most advanced anticancer compound of its class in late-stage development. Perifosine as monotherapy, is being explored in other indications. The US Food & Drug Administration (FDA) has granted perifosine orphan-drug designation in multiple myeloma and neuroblastoma, and Fast Track designations in both refractory advanced colorectal cancer and multiple myeloma.

Additionally, an agreement was reached with the FDA to conduct the phase III trials in both of these indications under a Special Protocol Assessment. Perifosine has also been granted orphan medicinal product designation from the European Medicines Agency (EMA) in multiple myeloma. Furthermore, perifosine has received positive Scientific Advice from the EMA for both the advanced colorectal cancer and multiple myeloma programmes, with ongoing phase III trials for these indications expected to be sufficient for registration in Europe.

Yakult Honsha is a leading Japanese company focused on the development and marketing of pharmaceuticals, foods, beverages, and cosmetics.

Aeterna Zentaris is a late-stage oncology drug development company currently investigating potential treatments for various cancers including colorectal, ovarian, endometrial cancer and multiple myeloma.