Aurinia pharmaceuticals ($AUPH) has a likely winner in voclosporin

Aurinia Pharmaceuticals is a company putting all of their eggs into one basket. In this case, that basket is called “Voclosporin,” an immunosuppressant that hopes to treat lupus, a somewhat common – and nasty – autoimmune disease. Specifically, AUPH is hoping to treat a kidney-related complication of lupus called “lupus nephritis,” a malady that more than half of lupus patients suffer.

In the phase 2 trial, adding voclosporin to the current standard of care appeared to beat out the standard of care alone in treating lupus nephritis (LN).

Very Recent Phase 2 Trial Showed Promise

The phase 2 trial tested two doses of voclosporin (a low dose arm and a high dose arm) against the standard of care alone. The endpoints were chosen well and were actually rather stringent – the primary endpoint was complete remission of LN at the 24-week mark, and secondary endpoints were partial remission, time to response, and remission rates at the 48 week mark. As you can see in the table below, the low dose of voclosporin met the primary endpoint, with significantly more patients reaching complete remission by week 24. Voclosporin met all the secondary endpoints as well – and by week 48, significantly more patients on the high dose of voclosporin had also reached complete remission.

Significantly more patients on both doses achieved partial remission, as well – and, ultimately, the trial met every secondary endpoint.

Taken from Parikh et al., 2017

At first glance, the safety data may appear daunting. The low dose of voclosporin, in particular, had a huge number of patients die (10 out of 89). However, there are several mitigating factors. First, the high dose of voclosporin had a much lower percentage of deaths, which suggests that the drug itself was not entirely to blame – previous trials have found that voclosporin has a very dose-dependent response, so if the drug were particularly dangerous we would expect to see more deaths in the high-dose arm. Second, the sponsors and investigators of the study determined that all of the deaths were unrelated to voclosporin. Third, most of the deaths occurred within the first two months of the study, and appeared to be typical causes of death for patients with severe lupus.

As you can see below, overall, safety data from this study tracked well with safety data from the studies of other immunosuppressants.

Taken from Parikh et al., 2017

I will note, however, that there were a few things I noticed that were a little disquieting. First, AUPH has tested voclosporin on a few other immune-related diseases – with some good effect, actually – but haven’t brought any of those formulations to market. I’m not certain why (though I admit, I haven’t looked all that hard). Second, as I noted earlier, voclosporin appears to have a strong dose-dependent effect. Why, I wonder, did the lower dose see a greater and more rapid treatment effect? Given that the lower dose group also had more patients pass away, was there perhaps some issue with randomization?

Conclusion: Worth a Chance

Because volcosporin is the only drug in AUPH’s pipeline, the success or failure of the eventual phase 3 trial will be a remarkable binary event. When the final phase 2 data for volcosporin was released a few months ago, AUPH’s stock price increased more than 200%. If the phase 3 trial is a success, I’d expect an even larger price movement.

I think that AUPH have set themselves up well for the eventual phase 3 trial of voclosporin. Unfortunately, the estimated completion date for the phase 3 trial is currently December 2019 – so we have a long time to wait, which increases the odds of something weird happening, like AUPH running out of money.

I wouldn’t put a big chunk of your portfolio towards this one, but I plan on investing at least a little bit of mine in it.

Prediction: Phase 3 Success

I would take a pretty small stake, but given the data and the potential gain, I think it’s worth putting some money towards this one.

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