Momentous announcement from GSK marks key inflection point in Professor Andrew Wilks’ 20-year journey.
GlaxoSmithKline’s Ojjaara (momelotinib), a JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor with origins in Australia, has received approval from US regulators for the treatment of myelofibrosis.
The treatment was developed as candidate CYT387 at Cytopia, an Australian biotech company founded by Professor Andrew Wilks in 1999 and headed by Prof Wilks, Dr Chris Burns, and Andrew Macdonald.
Prof Wilks and his team at the Ludwig Institute for Cancer Research discovered and developed a suite of patents around JAK1 and JAK2, which were licensed by Cytopia and taken through preclinical and early clinical development.
Myelofibrosis, rare a bone cancer, can lead to severely low blood counts, including anaemia and thrombocytopaenia; constitutional symptoms such as fatigue, night sweats, and bone pain; and splenomegaly. To date, Ojjaara it is the only approved medicine for both newly diagnosed and previously treated myelofibrosis patients with anemia that addresses the key manifestations of the disease.
While initially approved as a treatment for myelofibrosis, a media release said the molecule has the potential to gain additional approvals for treating a number of inflammatory diseases.
In 2009, the then ASX-listed Cytopia was acquired by Toronto-based YM Biosciences for $AU14 million; in 2012, Gilead Sciences acquired YM Biosciences, whose lead drug was the Melbourne-developed CYT387, for $US510 million; in 2018 Gilead was reported to have sold the drug to Vancouver’s Sierra Oncology for $US3 million up-front; and last year Sierra sold CYT387 to GlaxoSmithKline for $US1.9 billion ($A2.9 billion), making it the highest acquisition amount paid for a drug invented in Australia.
AusBiotech CEO Lorraine Chiroiu says, “This approval is a huge moment for all of those involved in the development of momelotinib, and Australia’s biotech community, as it showcases Australia’s world-class research capabilities and pioneering talent in translating breakthrough discoveries into much-needed therapies that benefit patients’ lives. It is rewarding to see more than 20 years of development, all of which started in a Melbourne laboratory, lead to the approval of a therapy to treat patients with myelofibrosis.”
Prof Wilks is now managing director of SYNthesis Bioventures, a fund focused on early-stage therapeutics, while Dr Burns is CEO of Amplia Therapeutics, a biotech company developing small molecule inhibitors to treat cancer and fibrotic diseases.
