For anyone who has been following Lupuzor with interest, the topline results from the phase 3 trial were released today, and they weren't good. Despite 68.8% of subjects on Lupuzor seeing benefits, 59.2% of those on the placebo saw benefits as well, which kept the results from reaching the significance needed for the primary endpoint. Essentially, the placebo effect (which has been increasing over recent years) means that patients will not be accessing this drug any time soon. Which is a real shame, as the trial had zero serious adverse side effects, which is an incredible safety profile for a systemic autoimmune treatment.
Had this trial been successful, Lupuzor would have been approved for lupus patients, making it available as an off-label treatment for others. Lupuzor's parent company, ImmuPharma, had indicated that they were exploring Sjogren's Syndrome trials if this trial had been successful.
There may be future for Lupuzor if ImmuPharma can get additional funding from the big pharmaceutical companies, but that will be many more years from now, at best.
Well that's disappointing. I've been following the trials with hope. Lupuzor seemed promising.
Hmmm, the Fat Lady isn't exactly singing on this one from what I gather (see bold text below). But yes, more time and data will be necessary before commercial licensing and FDA approval comes about. I think ImmunPharma realized the trend vs. Placebo + Standard of care (steroids) prior to reporting the top-line Phase-III results for Lupazor (of course they did...they are good at their jobs) and realized the need for a separate, redesigned Phase-III trial, that can distinctly demonstrate their desired end-points.
http://www.proactiveinvestors.co.uk/companies/news/195172/immupharma-drug-needs-further-analysis-it-could-still-have-a-role-in-treating-lupus-city-analyst-195172.html
WATCH: Drug needs further analysis, City analyst
The effectiveness among the 153 that got to the end of the course of treatment was 68.8% (versus 59.2% for the placebo).
Importantly, in patients who had a certain type of lupus biomarker called anti-dsDNA autoantibodies, Lupuzor worked well when compared with the placebo. Added to that, 7.6% of this group went into full remission, while none did in the placebo group.
The safety profile remained good with no adverse effects
Chairman Tim McCarthy said: ?Lupuzor has demonstrated, in this study, a superior response rate over placebo and its exceptional safety, giving it, we believe, a compelling product profile.
?Whilst we are disappointed at the high response in the placebo plus standard of care group that resulted in statistical significance not being reached between the two treatment groups, we believe Lupuzor has the potential to bring a much needed safe treatment to the millions of Lupus sufferers around the world.
?We look forward to providing our shareholders with further updates in due course."
Further investigation merited
ImmuPharma said it believes the top line results ?provide evidence for the continued investigation into the development and commercialization of Lupuzor?. It thinks the drug has the potential to offer patients and physicians a much needed effective and safe treatment for lupus.
Following requests from both investigators and patients, ImmuPharma has begun a further study. This will allow all patients who took part in the phase III trial to receive Lupuzor plus the ?standard of care? medication for six months in what?s called an open-label scheme.
The results will be gathered independent of the pivotal trial just completed.
The plan is to look at the full dataset before making a decision, but the company said there is demand for a new lupus treatment.
Expressions of interest
It also said there had been ?expressions of interest? in the Lupuzor programme and the phase III study.
?ImmuPharma is in ongoing discussions with a number of larger pharmaceutical companies,? it added.
?The results of this study will now be shared with those potential commercial partners. There can be no certainty as to the outcome or timing of these discussions.?
Disappointing news indeed. :(
The results do raise interesting questions regarding the strength of the placebo effect
on autoimmune diseases in general.
Normally, "placebo effect" only accounts for about 33%+ of perceived improvements in studies.
"I believe therefore I am"? Not so sure...
The fact that only those receiving the actual medication (Lupuzor) went into remission says something about the limitations of the placebo effect.
Tim McCarthy, ImmuPharma chairman, gave an interview following the results. You can view the video yourself (https://vimeo.com/265176047) (it's about 7 minutes), but the summary is that he feels that Lupuzor still has a lot of promise and he is confident in its efficacy and safety. He notes that several pharma partners have expressed 'very strong interest'. He also states that ImmuPharma is still in a strong cash position after raising $10 million.
"We shouldn't forget Lupuzor and I do want to emphasize the fact that Lupuzor is still there and we will continue to push it forward... as far as I'm concerned, we still have an extremely attractive drug."
I'd be interested in seeing if the "benefits" seen in both controls were subjective or objective. Was there improvement in symptoms, or were the benefits in improved lab data (which, as most of us know, doesn't necessarily mean you feel better).
Dealing with Autoimmune Disease for research can be very tricky because of the flare and remit mature of most of them anyway.