ATL-104

Globally there are over 4 million new cases of cancer each year. Mucositis is especially severe in patients being treated for head and neck cancer, and in patients undergoing bone marrow transplantation. The potential sales for a successful mucositis product could be in excess of US$500 million per annum.

ATL-104 is being developed by Alizyme as an orally administered cancer supportive care product for the treatment of mucositis of the mouth and gastrointestinal tract arising during cancer treatment. It resulted from a research collaboration with the Rowett Research Institute in Scotland. Alizyme subsequently acquired the rights to ATL-104 and has applied it to the treatment of mucositis.

Mucositis associated with cancer treatments.

Mucositis is characterised by severe ulceration, bleeding and pain in the mouth and gastrointestinal tract, caused by damage to the cells that line these tissues by cancer chemotherapy and radiotherapy. The severity of the mucositis is influenced by the intensity of the cancer therapy and by the individual drugs used to treat particular cancer types. It can be a dose limiting side effect of cancer treatment and can require the use of powerful pain relieving medications such as opiates, parenteral nutrition (feeding via intravenous infusion) and antibiotic therapy to control associated infection and can be potentially life threatening.

Globally there are over 4 million new cases of cancer each year. Mucositis is especially severe in patients being treated for head and neck cancer, and in patients undergoing bone marrow transplantation. ATL-104 is a growth factor and is being developed for the treatment of oral and gastrointestinal mucositis. There is only one approved competitor growth factor product, Biovitrum’s Kepivance^® (palifermin), which is administered by injection and is limited to use in haematological malignancies. Thus there is a considerable commercial opportunity for a product such as ATL-104 that can be easily administered orally as part of out-patient cancer care across a broad range of cancer types, including solid tumours. The potential sales for a successful mucositis product could be in excess of US$500 million per annum.

ATL-104 has a unique product profile, being a growth factor formulated as a swallowable mouthwash, providing ease and convenience of administration and enabling local delivery of treatment for oral and gastrointestinal mucositis with no significant absorption into the body. As such, ATL-104 acts topically rather than systemically.

To address mucositis, growth factor treatments act by stimulating the growth of the cells lining the mouth and intestine. Kepivance^®, being administered intravenously, is delivered systemically. Since ATL-104 is designed to act topically, it does not have the safety considerations associated with systemically delivered treatments and therefore has the potential to be applied in the prevention of mucositis occurring during therapy for a broad range of cancer types, including solid tumours.

In Phase I clinical studies involving over 60 healthy volunteers, ATL-104 was shown to be safe and well tolerated.

In a Phase IIa ‘proof of concept’ clinical trial involving over 60 patients with lymphoma and myeloma, ATL-104 was shown to be safe and well tolerated and demonstrated a significant reduction in the duration of severe mucositis.

ATL-104 has successfully completed a Phase IIa ‘proof of concept’ clinical trial in patients with lymphoma and myeloma.

When financial resources permit, the preparation for a further Phase II study, this time in head and neck cancer, and the transfer of the recombinant protein to a large scale manufacturer in advance of Phase III development and potential commercial supply will continue.