EP Vantage Interview – Noxxon's transformation entering pivotal phase

Noxxon Pharma’s transition from a technology platform based company to one developing its own product portfolio is reaching a pivotal phase. With contract research style deals with the likes of Pfizer, Roche and Eli Lilly having come and gone, the private German biotech now has three internally-derived and unpartnered candidates in the clinic.

Early-stage trial results over the next 12 months could provide Noxxon with that all-important proof-of-principle data, which Iain Buchanan, chief executive, hopes will spark some serious partnership discussions: “The burden of proof in today’s market falls a great deal on the companies. We’ve had to invest in our pipeline to generate data that will persuade people that this is a valid approach.” That approach, based on developing chemically synthesised RNA oligonucleotides, presents another challenge with Mr Buchanan keen to distance Noxxon’s technology from the somewhat stuttering field of RNAi and antisense based therapeutics.

Mirror image

Established in 1997, Noxxon has developed a technology platform which produces so-called ‘spiegelmers’, essentially mirror images of RNA molecules. These compounds are comparable to proteins in structure, bind to molecular targets in a similar manner to antibodies, yet are chemically synthesised and therefore do not require complex biological production processes.

The potential in the technology received validation of sorts through a number of deals struck with big pharma companies. Pfizer in 2006, Roche in 2007 and Eli Lilly in 2008 all signed broad research collaborations with Noxxon, but these were contract research in nature, with the German company tasked with applying its spiegelmer technology to identify candidates against specific targets provided by its partners.

However, all these partnerships fizzled out, prompting Noxxon’s switch to developing its own spiegelmer candidates and initiating its first clinical trial in June 2009 with NOX-E36, which remains its most advanced product.

NOX-E36 – which targets monocyte chemotactic protein-1 (MCP-1, also known as CCL2), a key pro-inflammatory chemokine – is currently undergoing a phase Ib trial in diabetic nephropathy. Results should be available by the end of the year, with the aim of starting a phase II trial in the first quarter of 2012 which should then read out by the third quarter.

Noxxon’s has two other phase I candidates. NOX-A12 also entered the clinic in 2009 and holds potential to treat a range of cancers, but initially is being developed for haematological tumours such as chronic lymphocytic leukaemia and multiple myeloma. A phase I trial of NOX-H94 started last week, with the intention to treat anaemia of chronic disease by targeting the peptide hormone hepcidin. Phase II trials of both these compounds should also start early next year.

Proof in the pudding

The results from these trials with all three candidates will go a long way to providing greater validation of Noxxon’s technology, which is likely to have a significant bearing on partnership discussions.

Having completed a €35m ($44m) series D financing round last year, the company is reasonably placed to fulfil its short-term commitments to developing its three clinical candidates across multiple indications. “Our cash balance is certainly sufficient to take us through to the first quarter of 2013,” says Mr Buchanan, a prediction which excludes any potential cash flow from partnerships.

As for those potential partners, Mr Buchanan is naturally keen to collaborate with companies which offer clinical trial expertise in the therapeutic areas that match Noxxon’s candidates. “We’re at an interesting stage and certainly getting some preliminary interest from some of the people who have been tracking us.”

Given Noxxon’s relative maturity, some longer-term investors might be hoping positive trial data could lead to an exit in the form of a trade sale or IPO, although the latter remains a long shot according to Mr Buchanan: “Never say never, but I don’t spend a great deal of my time worrying about preparing a prospectus for a public offering.”

And although the company has been around for almost 15 years, Noxxon underwent a major re-financing in 2007 through a series C, which brought in the likes of TVM Capital, Sofinnova Partners and Edmond de Rothschild Investment Partners, followed by NGN Capital who led the series D last year. As such, investor pressure to exit will have been eased somewhat.

Antibody like

Aside from the challenges of generating positive clinical data, in terms of attracting broad partnership interest it seems the company has a bit of re-branding to do with regard to how it positions its technology, which has some similarities to both antibody and RNAi products.

Given the disappointment of the RNAi and antisense field so far to meet lofty expectations (Vantage Point - Clinical progress required to reignite RNAi field, May 20, 2011), Mr Buchanan is nailing Noxxon’s flag firmly to the antibody mast.

“There is healthy scepticism around sRNA and antisense molecules, that there has not been the dramatic success that some people had anticipated many years ago when investments were being made. People falsely put Noxxon in the same bucket as Alnylam Pharmaceuticals or Silence Therapeutics, but we’re not, we’re far more in the same (antibody-alternative) space as the likes of F-Star, Ablynx and Bicycle Therapeutics.”

By positioning its spiegelmers more as chemical antibodies, Noxxon will be hoping to mimic the relative success so far of MorphoSys rather than suffer the setbacks Alnylam has experienced. Both companies are also attempting to make that tough transition from technology platform to internal product development.

How successful Noxxon will be in making its transformation depends heavily on the ability of its three clinical candidates to deliver safe and effective data.

“Whichever way you cut it, 2012 will certainly be a year of transition for the company,” says Mr Buchanan. “We will have clinical results which will hopefully enhance the value of the company. What the company does thereafter, and how it manages that through partnerships in the future, we’ll have to see.”

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