Thursday was a big day for two companies, in two very different ways, in the biotech space. News out of the Horizon Pharma PLC (NASDAQ:HZNP) camp initiated a major selloff in the company, while an FDA announcement regarding Regeneron Pharmaceuticals Inc (NASDAQ:REGN) didn’t have an equivalent impact to the upside, but it did set the company up for some considerable strength if things run smoothly going forward.
Here’s our take on both events.
So, starting with Horizon Pharma PLC (NASDAQ:HZNP), the company just announced that it will be discontinuing its lead Friedreich’s ataxia study. For those not familiar with this condition, it’s an inherited disease rooted in the nervous system. Basically, it slowly, but continuously, damages the nervous system of the sufferer and – progressively – leads to symptoms such as gait disturbance, scoliosis, heart disease and diabetes. It doesn’t affect the brain, but this doesn’t make it any less severe from a debilitation perspective, and there’s currently no cure. Vitamin treatment is essentially all a patient can do to try and slow the progression of the disease.
Horizon was trying to fill this unmet need with a drug called Actimmune.
The drug is already approved for use in patients with chronic granulomatous disease and severe, malignant osteopetrosis, and Horizon was looking to expand on this batch of indications the FA target. The drug was in a phase III study, and early data looked positive.
In the trial, which compared Actimmune with placebo in 96 patients, across a 26 week dosing period, the drug failed to improve over control across a host of primary and secondary endpoints, and as such, is scrapped in this indication.
It’s a blow for the FA community, of course, and Horizon Pharma PLC (NASDAQ:HZNP) and its shareholders, but with no discernible clinical benefit on offer, there’s not much else the company could do. Management says it will now pick apart the data drawn from the trial in an attempt to try and salvage any worthwhile information for future trials (assumption, both for it with Actimmune in other indications and for other companies in an FA indication) but that’s all the worth the company is going to get for now.
Moving on, let’s shift gear, and look at Regeneron Pharmaceuticals Inc (NASDAQ:REGN).
This company submitted a registration application in Europe to the EMA earlier this year (the equivalent of an NDA to the FDA in the US) for a drug called dupilumab. It’s an IL-4/IL-13 inhibitor, and Regeneron Pharmaceuticals Inc (NASDAQ:REGN) developed it in collaboration with French drug maker Sanofi SA (ADR) (NYSE:SNY) as part of a moderate-to-severe atopic dermatitis indication. The data hat underpins the submission is excellent, and it’s widely considered a shoo in for approval. Readers will probably be aware that the drug is also with the FDA as we speak, and is set for PDUFA at the end of March.