Juno Therapeutics vies for cancer treatment lead (Source:FT)December 17, 2014 3:52 pm Juno Therapeutics vies for cancer treatment lead Andrew Ward, Pharmaceuticals Correspondent When Juno Therapeutics lists on Nasdaq on Friday, it will be one of the biggest events in a big year for the buoyant US biotech sector. The Seattle-based company has attracted attention because one of its early backers was Jeff Bezos, founder of Amazon. But increasingly it is Juno’s science that has been making headlines as it vies for leadership of one of the hottest areas of drug development. Juno is pitted against rivals including Novartis, the big Swiss pharmaceuticals group, in a new field of cancer medicine called cellular immunotherapy. This involves extracting blood from patients and re-engineering the disease-fighting T-cells in a way that enhances their ability to destroy tumours. Once reinfused, these modified blood cells proliferate into a cancer-hunting army that eliminates the disease and provides lasting protection against its return. That, at least, is the impression created by successful early trials of these so-called Car-T therapies, named after the genetically engineered chimeric antigen receptors (Cars) which help the T-cells recognise a specific protein associated with cancer. There is still much to prove. The trials have so far only involved small numbers of patients with certain types of haematological cancer. But the results have been strong enough to suggest cellular immunotherapy has the potential to transform the way some cancers are treated. Novartis revealed data this month showing that, of 39 children with deadly acute lymphocytic leukaemia, 92 per cent were completely cleared after treatment with the company’s CTL019 therapy, often called Cart-19. Some suffered relapse, but 70 per cent were still cancer-free after six months. This has encouraged a surge of investment in smaller companies working on similar products including Juno, which is expected to raise up to $212m this week on top of the $300m in private financing attracted since April. Another contender, Kite Pharma of California, raised $106m in a June IPO and has since seen its share price soar by 80 per cent. Industry executives say Car-T marks a coming-of-age for the wider category of cell and gene therapies which scientists have been working on for decades, but which are only now beginning to near commercial fruition. Optimism about this blossoming science has helped US biotech companies raise $4bn in 59 IPOs so far in 2014, with the Nasdaq biotech index up almost 30 per cent. Big pharma is also placing bets. Pfizer, Johnson & Johnson and GlaxoSmithKline have all invested in cellular immunotherapy this year. Cellular immunotherapy is part of a broader wave of groundbreaking cancer treatments that harness the body’s immune system to target tumours. Whereas other companies, such as Merck and Bristol-Myers Squibb, are developing drugs that aim to stimulate the immune system from within the body, Car-T therapies are tailored for individual patients in a laboratory. This is a complex and expensive process but has the potential to produce more potent treatments. Cart-19 was granted breakthrough therapy designation by the US Food and Drug Administration in July, opening the way for fast-track regulatory review. Novartis plans to file for approval in 2016 and has predicted “multibillion-dollars in peak sales”. Others are more cautious. Richard Vosser, analyst at JPMorgan, has noted concerns over potential long-term toxic side effects. The treatment relies on the modified T-cells recognising tumours and leaving healthy tissue alone — but this can be risky. It is also unclear whether the therapy can be expanded beyond blood cancers into more prevalent solid tumours. “Cell and gene therapy are going to be a pillar of medicine and it’s just a question of who has the courage to make the leap,” says Usman Azam, head of Novartis’s cell and gene therapies unit. A reminder of potential perils ahead came last month when Dendreon, an early trailblazer in cellular immunotherapy, filed for bankruptcy facing $620m of debts. Revenues from its Provenge prostate cancer treatment were once forecast to top $4bn, but have so far reached just $300m, held back by a cumbersome production process and insufficient clinical benefit to justify the $93,000 price tag. Mr Azam says there are lessons to be learnt from Dendreon’s experience. “They had the courage to take the first step but the science moved on and that shows how important it is to move quickly.”