What Caladrius Biosciences Inc (NASDAQ:CLBS) does is really very simple, at least in concept. It isolates tregs from a blood sample, propagates them, and reintroduces them into the patient, restoring cell balance. Caladrius’s studies have already shown that reintroduced propagated tregs stay around in the blood for a year and perhaps even longer. Even more importantly, in one key study on adolescents aged 5 to 18 with type I diabetes, 2 out of 22 patients achieved insulin independence, essentially a functional cure of the disease. 6 more achieved remission, defined as a daily insulin dose of less than 0.5 units per kilogram. So there is early evidence that the treatment can work, and Caladrius is moving forward to an exciting Phase II.
The Phase II trial began in March of last year, and has already enrolled 111 patients with recent onset type I diabetes. In fact, the California Institute for Regenerative Medicine (CIRM) has just announced a $12.2M grant for the development of this type I diabetes drug. Much of this sum will be used to cover the remaining expenses of the trial. An interim analysis on 50% of enrolled patients is tentatively scheduled for the end of the year, which will be very big for the stock either way. The trial is expected to be fully completed by late next year, at which point Caladrius will be looking for a development partner to take the candidate to the next step.
The treatment, for now dubbed CLBS03, enjoys both fast track and orphan designations, a dual advantage and the first type I diabetes candidate ever to enjoy fast track status. This alone shows the FDA’s particular interest in seeing CLBS03 continue its clinical development.
What’s important to keep in mind here is that CLBS03 does not specifically target type I diabetes patients. It is only being tested on these patients to the exclusion of other autoimmune disease sufferers for the purposes of clinical development. Whichever autoimmune disorder CLBS03 may be applied to in the future, the treatment will be exactly the same in terms of procedure. This means that if interim phase II results at the end of this year show a good level of efficacy, it would show that CLBS03 could potentially be applied to almost any autoimmune disorder, and hopefully with similar results.
The point here is not that Caladrius Biosciences Inc (NASDAQ:CLBS) has the cure for all autoimmune diseases ever in its sites. That would be overstating the case. But from a shareholder perspective, Caladrius is unique in that its flagship treatment is designed not to counter only a single disease, but rather an entire class of diseases. This gives stakeholders a unique advantage, especially with interim data coming up around the end of the year.
Normally, positive Phase II data in a trial like this would show evidence of efficacy in a single disease. That’s big enough news, assuming the market is big. But in the case of Caladrius, it’s much more than that. If CLBS03 works with type I diabetes, which earlier trials have already shown is possible, then it could just as well work with other autoimmune diseases as well. This, of course, Caladrius will have to prove in separate trials, but it is a potential that investors don’t normally see in biotech.
It makes Caladrius Biosciences Inc (NASDAQ:CLBS) a compelling pick as 2017 progresses, and a biotech play that Market Exclusive will keep a very close eye on in the coming months.
Note: This article is written by David Rich and originally published at Market Exclusive.
