Catabasis Pharmaceuticals Inc (NASDAQ:CATB) is up on heavy volume heading into the close of this week, after the company announced a partnership with billion-dollar pharmaceutical company Sarepta Therapeutics Inc (NASDAQ:SRPT). It has been a pretty busy quarter for deals and mergers in the biotech space, and a number of double digit percentage point gains have been locked in for companies and their shareholders. Longer-term, however, only some of these partnerships will come to successful fruition. Is the Catabasis, Sarepta partnership one of them?
Let’s take a look.
This deal is rooted in a condition called Duchenne’s muscular dystrophy (DMD). It’s a disease rooted in a genetic mutation, for which there is currently no cure (and very little by way of symptomatic treatment). It’s pretty rare, but this doesn’t mean there isn’t a market potential for a drug entering the space. Ethical questions aside, the rarity of diseases such as DMD means companies are able to justify high pricing for any commercialized products, by way of citing this pricing as incentive to allocate resources to what might otherwise be a non-profitable venture.
Back to DMD specifically, it affects anywhere between one in every 3,500 to 5,000 males worldwide, and it is universally fatal. Sufferers generally die by the age of 30, after a number of years of drawn out degeneration. In other words, there’s a massive unmet need – if not quantitatively, definitiely medically.
So what will the latest collaboration mean for the space?
It is a pretty interesting approach. DMD is caused by a faulty version of a protein called dystrophin, which comes about as the result of a mutation in a gene also called dystrophin. Dystrophin is required for a bunch of different functions, but primarily is related to structure of muscles and connective tissues. The lack of functional dystrophin in DMD patients translates to the symptoms and gradual degeneration. There are two leading treatment spaces right now, one of which focuses on something called exon skipping, and another that focuses on protein inhibition.
Exon skipping is a modification of the transcription process at RNA level that instructs the gene to skip out a particular exon, and in doing so, causes the gene to produce an incomplete, but still functional version of the dystrophin protein. Sarepta’s Eteplirsen just picked up approval for DMD in the US, and is a treatment based on this concept. That’s what Sarepta is bringing to the table.
Catabasis is bringing its own candidate, CAT-1004, an NF-kB inhibition drug called Edasalonexent, to the table, and the two companies are going to attempt to demonstrate that both drugs used as a combination therapy are more affected than when used as a monotherapy. By way of a brief explanation of why this might prove a valid thesis, NF-kB inhibition Targets the NF-kB protein, a protein that when activated (as it is when dystrophin levels are weak in DMD patients) causes inflammation and suppresses muscle regeneration. The last thing a DMD patients needs is muscle regeneration issues, so basically the combination therapy will work on one hand to produce a semi-functional form of dystrophin, while on the other hand working to promote muscle regeneration and suppress inflammation (read: fibrosis) via NF-kB inhibition.
From a scientific perspective, it looks like a promising approach. Of course, many of these sorts of combination therapies fail when scrutiny is applied, but both groups have demonstrated efficacy in their respective populations, so there isn’t any obvious reason why a combination shouldn’t build on this data. The only thing we can think of that might hinder a combined treatment is bio availability, but chances are this will be the subject of an early-stage problem resolution as part of the partnership.
The bottom line here is this: Sarepta is a big name in the space, and there are a number of juniors it might have chosen to work with. Due diligence in place, it chose Catabasis, validating the company’s science (as well as its approach) in the process. For us this makes the latter well worth watching.
Subscribe below and we’ll make sure you stay updated as data starts to roll out of the preclinical models on this one. It’s going to be an interesting journey, so don’t miss out on the catalysts!
Disclosure: We have no position in CATB or SRPT and have not been compensated for this article.