As we kick off a fresh week in the biotech space, here are two companies that look set to move on recent updates, and what to look out for with each.
Omeros Corporation (NASDAQ:OMER)
First up, Omeros. Towards the end of last week, Omeros announced the first patient dosing in its now ongoing phase 2 trial for its lead candidate OMS 721. The trial is just one of a host of ongoing trials for the drug, with a phase 3 in atypical hemolytic uremic syndrome (aHUS) and a phase 2 in thrombotic microangiopathies (TMAs) Being the two most advanced as things stand. The drug in question is what’s called a mannan-binding lectin-associated serine protease-2 (MASP-2) inhibitor. It’s an interesting area of biotechnology, and one that is limited to research by Omeros only. This limitation derives from the fact that researchers working for the company discovered the MASP2 protein, and first identified its MOA.
So how does it work? In our bodies we’ve got what’s called the complement system. It’s a part of the immune system responsible for initiating an immune response to trauma and infection. In some instances, however, mutations in MASP-2 cause an unjustified or overt response, and this leads to a number of complement system related immune disorders, including the aforementioned aHUS, TMAs and the indication Omeros is targeting with the just announced face to dosing – renal disease. MASP-2 inhibitors, of which OMS-721 is one, limit the impact of these overt responses, and so, in theory, can be used to counter complement system related immune disorders.
It’s not a massive trial, with only 16 patients slated for investigation and split across certain types of renal disease, but with a 12 week dosing period, Omeros expects to apply for breakthrough designation, and in turn, accelerated approval.
So what are we looking for going forward? The company, as mentioned, expects dosing to take 12 weeks, and beyond that, we expect topline from the trial sometime during the third quarter this year. If the drug demonstrates any level of efficacy, a phase 3 should follow quickly – likely before the close of 2016.
With the ongoing phase 2, the newly initiated phase 2, and the late stage phase 3 all running concurrently, the next 6 to 8 months should be pretty news heavy for Omeros, and so we expect some volatility going forward. Potential upside catalysts are plentiful, but the same applies for downside shocks if OMS-721 proves ineffective across any of its target indications. One to watch, and a potentially rewarding small-scale speculative allocation as part of a wider portfolio ahead of near-term milestones.
MediciNova, Inc. (NASDAQ:MNOV)
Next, MediciNova. MediciNova is a California-based biotech, with a small molecule therapeutics focus. The company just reported interim data from one of its lead trials, and volume spiked on the announcement.
The data relates to MN-166, a clinical stage candidates targeting and the amyotrophic lateral sclerosis (ALS). This condition got some pretty widespread press couple of years ago as part of the ice bucket challenge, so some reading this might already be familiar with what it entails. For those that are not, however, it’s a degenerative condition that’s results from an inability of nerves to communicate mobility instructions to particular muscles in the body. Over time, it leads to restrictive movement, and eventually, paralysis. MN-166 is already available and approved in Asia as a post stroke treatment, but one of its side effects is the attenuation of what are called activated glia cells. Non-activated glia cells are responsible for the lining and insulation that surrounds nerve cells. Activated glia cells, on the other hand, result in the sort of issues associated with ALS and other neurodegenerative conditions, and through that attenuation, MediciNova is hoping MN-166 can become an effective treatment not just for post stroke victims, but also for a host of other conditions, with ALS top of the list.
So what are we looking for with this one going forward? Well, the data showed that the drug is somewhat effective, but the trial is relatively small, with just 25 subjects completing the six-month treatment period. As such, chances are the company will need to run an extension trial before it can submit an NDA to the FDA. Having said this, it’s a pretty rare condition, and the only currently available treatment (standard of care) has relatively limited efficacy – Riluzole. With this in mind, if MediciNova can demonstrate safety and tolerability (which shouldn’t be a problem based on the already approved nature of the drug) it will go a long way towards any potential approval.
Going forward, therefore, we are looking to full topline to gauge chances of approval. The trial is expected to close out early next year. One to keep an eye on.