bno article

This woman may just cure cancer

MARIA MOSCARITOLO From: AdelaideNow
March 27, 2012 12:00AM

DEBORAH Rathjen's academic rigour, combined with business nous, has put Bionomics on track to reach global medical markets.

A serendipitous job offer from a former mentor changed Ms Rathjen's career trajectory, taking her from the lab to the boardroom and leadership of one of Australia's most notable young biotechnology companies.

Bionomics has enjoyed one of its most successful years, culminating in its recent licensing of its key anti-anxiety drug compound to a US pharmaceutical company for $345 million and its entry this month into the S&P All Ordinaries Index of Australia's top 500 listed companies.

It is expanding clinical trials for its anti-tumour drug, and has taken a first step towards developing a memory and anti-brain inflammation drug for Alzhemier's sufferers which, all going to plan, it hopes to have in phase I trials next year.

While its product pipeline has been well defined from the start, Dr Rathjen's path to Bionomics was an unforseen one.

The Flinders University alumnus views her career as one of calculated risks which have paid off, starting with her decision to leave her job as an immunologist to take up a job offer from a former mentor at CSIRO (where she did her PhD) for a biotech he had just started, Peptech.

"It was as simple as that. I became involved in biotech just because I got a phone call from a mentor (Geoff Grigg) who said he had just the job for me, and it turned out for me to be a dream job," she said.

It was 1988. She went to work as a research scientist, heading a team of 10 people, co-inventing drugs now used to treat rheumatoid arthritis and Crohn's disease (and licensed to Johnson & Johnson).

"It really opened my eyes to the fact you could be doing groundbreaking, leading-edge science in a biotech company. And we had no biotech industry in Australia at that point ... so it was great to be involved at what was essentially the beginnings."

(Peptech later merged with EvoGenix to become Arana Therapeutics, which was acquired by biopharmaceutical firm Cephalon International in 2009.)

That experience gave her grounding in the business skills which have proved invaluable at Bionomics: analysis of competitor therapies, program positioning, developing and defending patents.

She eventually became Pepetch's manager of business development and licensing, taking the lead on licensing agreements of peptides including one used in AIDS treatment and one to treat wrinkles.

In 2000, while she was residing temporarily in Adelaide to head a program Peptech was running in collaboration with the Women's and Children's Hospital, another call came out of the blue, this time from a recruiter looking for a CEO for the newly formed Bionomics.

It meant a permanent homecoming for the Broken Hill-born, Adelaide-schooled scientist.

"At Peptech I was really fortunate in that I was given opportunities to learn new skills, to learn about the business of biotechnology."

Built on her previous experiences, Bionomics has a very defined business strategy.

She says a key to its notable success in just over a decade is targeting therapies that have relatively few competitors but significant markets, such as its BNC210 compound, which treats anxiety.

"There's just a handful of drugs in development for anxiety; it's an area where pharmaceutical companies have really missed the boat in recent years in terms of investment in discovery and development of new treatments, which is really surprising given the burden of anxiety and depression in our community," says Dr Rathjen, who this month returned from presenting at an investor conference overseas.

"It's been left to small companies like Bionomics to undertake the initial discovery and the initial stages of development of new drugs."

Its propriety technology has been deployed on compounds targeting memory impairment in Alzheimer's patients and even those suffering schizophrenia and ADHD (its Alpha 7 program, for which it recently filed an international patent application for a possible compound, BNC1881), multiple sclerosis (Kv1.3 ion channel blocker, for which it has a licensing deal with Merck Serono) and other autoimmune diseases such as rheumatoid arthritis, and cancer (BNC105, which shuts down tumour blood vessels).

Identifying a new drug candidate takes discipline from the outset, she says, not luck. "We identify an area that we want to make our own, we identify the sort of compound that we're hunting for and we devise what we call a target product profile so we think about the end market, what does that drug need to do that other drugs that are used to treat that condition are not doing, how can we make a more effective, more potent sort of drug but at the same time doesn't have any side-effects," she said.

The team which had been working on BNC210 is now engrossed in the Alzheimers investigation.

The scarcity of deep venture cap funding for early stage discoveries has forced the company, like other young Australian biotechs, to be efficient and ruthless about its R&D investments.

"We've had to be pretty smart because, here in Australia, biotech companies can't necessarily access the kind of funds to do the early stage discovery and development of new drugs ... so I think it has actually made us be more innovative in the programs we'd consider," Dr Rathjen said.

She says being a small company has its advantages: it's "nimble" and decisions are not bogged in bureaucracy, and the team is more motivated because it has more ownership of the company's direction, and successes.

With BNC210, it was the $345 million licensing deal with Ironwood.

It has already received a $3 million payment.

Dr Rathjen said the deal represented a world-class return on investment on the compound, which had its first patent application filed in 2006, but would not specify that R&D outlay.

The licensing deal means it can pour more resources into its anti-cancer compound BNC105, she said.

It is going through a large phase II renal cancer trial in the US and Bionomics is awaiting approval for a first trial of the drug with ovarian cancer sufferers.

With sufficient phase II trial data, it hopes to find a large commercial partner able to undertake further trials and market such a cancer drug.

While the company reported a $3.46 million loss in the six months to December, it had $17.8 million in the bank, "and that's more money than Bionomics has ever had in its history", that it would pour into the Alpha 7 program and BNC105.

The company's ultimate aim would be to grow to a stage where it can fully fund the development of its drug discoveries and take them to market without licensing them to outside parties. For this, "we'd probably need to grow ten-fold from where we are today in all areas," Dr Rathjen said.

"Our ambition is to be the next CSL for Australia."

But it would remain a South Australian company, she said, and act as a "magnet" to further grow the state's bioscience industry.

---------------------------------
http://www.adelaidenow.com.au/business/sa-business-journal/she-may-just-cure-cancer/story-e6fredel-1226310374146