It may be that La Jolla Pharmaceutical is to be applauded for cutting its losses and ditching its two kidney disease candidates GCS-100 and LJPC-1010 after the FDA insisted on the kind of chemical composition data that would have been hard and expensive to acquire. Shareholders, though, were disappointed, sending its shares down 15% to $16.49 on Friday.
Perhaps stung by the agency’s demands, the company has opted to pour some of the money it has saved by shuttering these programmes into two antibiotics, which, as they are based on gentamycin – a known quantity as far as the FDA is concerned – pose far less regulatory risk. But the company’s immediate future now rests on its only clinical-stage product, hypotension drug LJPC-501, and regulatory is not the only type of risk.
Seeking a partner
Phase II data on GCS-100 last March were promising, and the galectin-3 inhibitor was in a phase IIb trial in chronic kidney disease when La Jolla pulled the plug (Galecto rewarded for its focus on a tangible target, November 4, 2014). According to La Jolla, the FDA wanted additional chemical characterisation of ‘100 prior to further clinical development, but the company says the product is a complex polysaccharide mixture and this level of analysis would have been prohibitively expensive.
La Jolla’s chief executive George Tidmarsh said on a conference call that the exacting nature of the FDA’s demands was unexpected, and the company had felt that it had provided normal information on product characterisation.
As LJPC-1010 is a chemically related to GCS-100 and has the same mode of action, its development has also been halted. LJPC-1010 was under development for the hot area du jour – non-alcoholic steatohepatitis, but had yet to reach the clinic.
Even if phase I trials had started on schedule next month it would have been at the back of the NASH queue. It is therefore perhaps less of a loss to La Jolla than GCS-100 (Astra first big pharma to dip toe in simmering NASH waters, April 8, 2015).
Mr Tidmarsh said that the company hoped to fund a partner for the projects, though he conceded that it was “hard to say” who would purchase their rights. The company believes in the underlying science, but the FDA is clearly less sure, and by La Jolla’s own admission a buyer would have to develop new analytical methods to obtain the chemical characterisation data requested by the agency.
Lower hurdle
The company’s two new products ought to be a much easier win when they come before the FDA – but that is likely to take a good few years as they are still pre-clinical.
It has licensed two gentamycin derivatives, which it is calling LJPC-30Sa and LJPC-30Sb, from the Indiana University Research and Technology Center. Mr Tidmarsh said the level of clinical data necessary to get these drugs approved ought to be lesser as gentamycin is a known quantity – these molecules are purified components of a product that is already approved.
30Sa and 30Sb are not just intended to treat serious bacterial infections. La Jolla intends to exploit gentamycin’s ability to disrupt gene transcription, which is its antimicrobial mechanism, to correct the mutations that lead to genetic disorders in humans such as cystic fibrosis and Duchenne muscular dystrophy.
Gentamycin has been proven to work in cystic fibrosis, La Jolla says, but the trials’ duration was limited by the antibiotic’s kidney toxicity. The company’s new compounds lack this effect, it says. Despite this Mr Tidmarsh would not say when clinical trials might be expected to start.
One clinical product
For the foreseeable future, then, the only clinical-stage drug in La Jolla’s pipeline is LJPC-501, a formulation of angiotensin II intended for catecholamine-resistant hypotension. La Jolla is currently recruiting patients in a randomised phase III study, Athos 3, with increase in blood pressure as the primary endpoint. Results are expected by the end of 2016.
And they had better be prompt – and positive – as that is when the money will run out. Analysts have not appended sales forecasts to any of La Jolla’s candidates, so it is difficult to say what the company will obtain should it reach market – or what it has lost by halting GCS-100 and LJPC-1010.
To contact the writer of this story email Elizabeth Cairns in London at [email protected] or follow @LizEPVantage on Twitter
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