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This medical breakthrough has its origins in Singapore

Dr Foo Fatt Kah
Dr Foo Fatt Kah • 4 min read
This medical breakthrough has its origins in Singapore
Vonjo, used to treat myelofibrosis, an uncommon form of bone marrow cancer, can trace its origins to Singapore’s biotech efforts nearly two decades ago / Photo: CTI Biopharma website
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The shareholders of CTI Biopharma Inc (CTI) are celebrating. CTI, a Seattle-based biopharmaceutical company whose shares are listed on the US Nasdaq capital market, last month announced that it was being acquired by a large European healthcare company, Swedish Orphan Biovitrium AB (SOBI), in an all-cash transaction. The equity value of CTI at SOBI’s offer price of US$9.10 ($12) per share was US$1.7 billion. CTI specialises in the development and commercialisation of drug therapies for blood cancers.

A one-product company, CTI’s sole commercially approved drug, Vonjo (pacritinib), treats myelofibrosis, an uncommon form of bone marrow cancer leading to a severe lack of blood cells and is consequently associated with poor patient survival outcomes.

Myelofibrosis is linked to the faulty functioning of an enzyme known as Janus Associated Kinase 2 (JAK2), which can arise from certain gene mutations. The JAK family of proteins is a central component in certain cell signalling pathways needed for normal cell growth and healthy immune response. Vonjo, in a class of drugs known as JAK2 inhibitors, asserts its therapeutic benefit by modulating the action of JAK2 in a highly selective way.

In September 2020, CTI submitted a new drug application (NDA) to the US Food and Drug Administration (FDA) for accelerated approval for Vonjo soon after the company obtained very valuable clinical trial data in a group of myelofibrosis patients who had severely low platelet cell counts, or thrombocytopenia.

No drug could address this important unmet clinical need, and these unfortunate patients faced limited therapeutic options and a grim outlook. The FDA duly processed the NDA expeditiously, and Vonjo was approved in February 2022 as the first drug therapy to address the specific needs of cytopenic myelofibrosis patients.

Made in Singapore

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Some analysts have since forecast that Vonjo will approach peak sales in the region of US$1 billion annually, making it a potential “blockbuster” product, defined by analysts as drugs achieving at least US$1 billion in annual sales.

It should not go unnoticed that the origins of this breakthrough medication are founded in Singapore. The drug hails from the early research pipeline of the city-state’s first-ever biotech company, S*Bio, which was formed in the year 2000 as a three-way joint venture between the Singapore Economic Development Board Investments and the US pharmaceutical company Chiron.

S*Bio focused on researching and developing novel small-molecule drugs for cancer treatment. Vonjo, then known as SB1518, was amongst the first anti-cancer molecules developed by S*Bio.

See also: Health care, not 'sick care'

Following a landmark Series B financing of US$26 million co-led by Bio*One Capital and Aravis Ventures in 2008, S*Bio could progress its two lead drug candidates, one of which was SB1518, through their early clinical trial programmes.

Other major international institutional investors were attracted to participate in S*Bio’s funding rounds, such as Novartis Bioventures, Mitsui Ventures, Zurcher Kantonalbank and Lacuna Apo Biotech Fund.

As it turned out, the clinical development path for Vonjo was lengthy and complicated, but CTI eventually picked up the worldwide rights from S*Bio in an asset purchase in 2012. The deal involved an upfront payment to S*Bio and continued payment tranches over time based on the achievement of certain commercial and regulatory milestones.

S*Bio would also enjoy certain future royalties on worldwide Vonjo net sales. This asset deal was structured in such a way as to allow S*Bio’s continued participation in the long-term success of Vonjo. The rest, one could say, is history.

Singapore has long recognised the importance of fostering a strong knowledge-based economy as part of its long-term national development strategy. It has invested heavily in developing world-class life science infrastructure integrated into an ecosystem, attracting significant scientific talent and leading international investors.

Amidst all the jubilant merry-making at CTI, we should not forget to celebrate Singapore’s notable involvement and the early actions taken by S*Bio’s management and board — all of which have played a pivotal role in this success story.

Foo Fatt Kah, a medical doctor, is the co-founder and managing director of Luminor Capital. He was formerly Asian venture partner for Aravis Ventures based in Singapore. He was also a limited partner in Aravis Venture I and is a limited partner in Aravis Biotech II

Editors' note: The article has been amended to address an inaccuracy on the joint venture partners involved

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