With all the political turmoil in the United States and across the Western world of late, it can be easy to lose track of less sensationalist politics, like the passing of bills that will, in time, prove quite consequential.
Amid the news-monopolizing US election and its aftermath, the 21st Century Cures Act passed both the House and the Senate this month, and the implications could be far-reaching. Some biotech companies should get more funding, shortened approval timelines and less stringent endpoint requirements. Patients should get easier access to potentially life-saving treatments quicker than under the previous legislation. For others though, it’s not so great. The FDA, for example, will pick up a $500 million boost to cover the increased workload associated with the Act, but this will go nowhere near the amount that the agency actually needs to solve some of its long-standing problems like staffing and facilities.
Biotech investors fall into the former category.
With shortened and less stringent development pathways, catalysts in the space should come thick and fast, and there’s likely going to be plenty of potential upside on offer on data driven gains. Here’s a look at three companies set to benefit, and why.
Medtronic PLC (NYSE:MDT)
Let’s kick things off with Medtronic. This one’s rooted in changes to the approval process for new medical devices. When it comes to devices (really, this could be said for the wider industry approval process) the FDA is seen to be stuck in the past, and the bringing of the agency up to date is the core argument for the passing of Cures. In other words, the industry has moved forward, but until now, the regulatory body hasn’t. Some of the key medical device provisions of the bill, and their regulatory implications, include an expedited review program, an expanded scope of devices eligible for HDE, an increased reliance on real world evidence (i.e. patient experience, as opposed to just numbers); a streamlined combination product review process; and the reclassification of certain software products and fringe devices to remove the necessity for an approval application.
Each one of these, especially the breakthrough program and the widened HDE scope, should benefit Medtronic going forward. The company has a range of pipeline assets that would fall under the medical device classification, and in turn, stand to benefit from the provisions outlined in Cures.
Egalet Corp (NASDAQ:EGLT)
Moving on, let’s look at Egalet. Where Medtronic stands to gain from the elements of Cures that relate to medical device assessment, Egalet stands to benefit from elements of the bill that address the opioid abuse epidemic in the US. The bill will see more than $1 billion funneled into the opioid abuse space going forward, much of which will go towards kitting out treatment centers, but a large portion of which will go towards the development of abuse deterrent assets – assets just like those Egalet is developing right now. The company juts picked up a patent covering the abuse deterrent properties of its oxycodone tablets, marketed as OXAYDO. OXAYDO picked up FDA approval as an abuse-deterrent immediate release oxycodone, the first of its type, back in September 2015. It’s got a decision pending with the FDA concerning an abuse deterrent ER morphine called ARYMO currently, and a host of early stage assets seeking to pick up an abuse deterrent label as they mature through Egalet’s pipeline. With the Cures Act bringing opioid abuse front and center, companies with pipelines dedicated to the niche are going to draw a lot of speculative attention, and of those that fall in to this category, Egalet is leading the pack.
Caladrius Biosciences Inc (NASDAQ:CLBS)
Finally, Caladrius. We’ve done devices, and we’ve done opioid abuse, now let’s move on to another major area of medicines development that Cures aims to bolster: regenerative medicine. Specifically, stem cell treatments. The stem cell space has had a pretty tough time to date, with ethical and political roadblocks often standing in the way of its advance – especially in the US. The promise of the technology that underpins it is undeniable, however, and even against the industry headwinds, a number of companies in the US have pushed forward in the space.
One of these is Caladrius. The company operates a cell therapy manufacturing facility through a subsidiary called PCT. Basically, PCT prepares stem cell therapies for other entities, which the latter use to conduct trials during an asset’s early stages of development. It’s a type of CMO, specifically working with stem cells in the development asset space. As such, the more research and development activity that exists in the stem cell space, the more trials will be undertaken, and the more companies will need stem cell CMO’s like PCT to prepare their development batches for them.
The Cures Act has brought into play a system whereby stem cell therapies won’t need to be put to the test in large, phase III studies (the studies that they needed to undergo previously) before getting approval. Instead, a company can apply for approval based on early stage safety tests, and then monitor efficacy post approval as part of a follow-up study. It’s one of the most controversial elements of Cures – there will no doubt be some therapies that get approved based on safety data and later turn out not to work – but for companies like Caladrius, this doesn’t really matter; at least not from its PCT CMO perspective, defraying the risks that Cures entails for other stem cell companies proper.