Biotech Gainers On Tuesday: Amicus Therapeutics, Inc. (FOLD) And Ionis Pharmaceuticals Inc (IONS)

While wider markets turn there focus to the US elections, the biotech space is alive and well, and after a tough end to last week, a number of companies have enjoyed gains throughout the start of the week, and looks set to pick up further as the week matures.

Here are some of the week’s biggest movers so far, and a look at what’s driving the action. The companies in focus are Amicus Therapeutics, Inc. (NASDAQ:FOLD) and Ionis Pharmaceuticals Inc (NASDAQ:IONS).

First then, Amicus Therapeutics, Inc. (NASDAQ:FOLD).

This one is a pretty simple move. The company put out its latest earnings report on Monday, and alongside the report, we got an update as to the progress as to its development pipeline. In a lot of these early stage biotech companies (yes, Amicus Therapeutics, Inc. (NASDAQ:FOLD) is worth a little over $1.1 billion, but there’s a robust pipeline, speculation as to the potential of which underpins the majority of its valuation) the numbers don’t really matter when it comes to earnings, day. Instead, markets focus on the business and operational updates that nearly always accompany the numbers. This is what generally decides sentiment, and that’s very much the case here.

The company gave us an update as to its lead asset on the operational report, Galafold for patients with Fabry disease. The drug (called migalastat in the US) is approved in Europe in this target indication, but is as yet to pick up a US approval in the same. The update noted that 50 patients are currently being treated, and reimbursed for that treatment as part of an expanded access program, and that in the US, we should see a regulatory update on an optimal filing pathway by year end. This suggests we might see an approval medium term, something that would considerably expand the drug’s (and in turn, the company’s) peak sales potential.

Markets recognized this fact, and bought into the company ahead of the regulatory pathway update. The company surged 20% on release, and is up just shy of 6% at time of writing on Tuesday.

Next up, Ionis Pharmaceuticals Inc (NASDAQ:IONS). This one relates to both Ionis and its partner in the development program we are about to discuss, Biogen Inc (NASDAQ:BIIB). The two companies are working together to bring a drug to market called SPINRAZA, for the treatment of spinal muscular atrophy (SMA) in children. For those not familiar with the condition, it’s a really nasty disease that will start to show symptoms in patients as early as 6 months of age (although the specific target of this trial was patients with a slightly later onset, what’s called SMA type 2).

Biogen Inc (NASDAQ:BIIB) and Ionis Pharmaceuticals Inc (NASDAQ:IONS) are aiming to show that SINRAZA has the potential to improve motor function in these patients (loss of motor function trough muscular degradation is a key symptom), and if the two companies are succesfull, it would represent one of the only treatments (the only on label) available for patients in this condition.

The data the company put out showed that children who received SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment, and that the safety profile of the drug was sound. This is an interim analysis, but with the data coming out as positive, patients receiving the drug will now move into an open label extension. Biogen Inc (NASDAQ:BIIB) expects that it can set up for commercialization for the drug as soon as early 2017 (the company slated end 2016, but we think this is unlikely) and it’s this potential that is driving Ionis, and for that matter, Biogen, higher today.

Note: This article is written by Mark Collins and originally published at Market Exclusive.