Our first biotechnology mover of note this week is New Jersey-based The Medicines Company (NASDAQ:MDCO). On Tuesday, the company announced that the Food and Drug Administration (FDA) has approved one of its lead drug development candidates, an asset called Vabomere, for adults with complicated urinary tract infections (cUTI), including a type of kidney infection, pyelonephritis, caused by specific bacteria.
The drug itself is a combination of two different types of drugs, one of which is an already very well-established antibacterial and another of which is The Medicines Company’s attempt to bring something new to the table; and a successful attempt at that.
The former is called Meropenem, marketed under the tradename Merrem, and initially having been developed and sold by biotech giant AstraZeneca plc (ADR) (NYSE:AZN). It’s now generic, however, and has a place on the World Health Organization’s List of Essential Medicines (which are the most effective and safe medicines needed in a health system).
The letter, the second component of Vabomere, is a bit more interesting. It’s called vaborbactam and it’s proprietary to The Medicines Company. When antibiotics are used too much, bacteria become resistant to them. This resistance is rooted in the production of what are called Beta lactase enzymes, produced by the bacteria, which degrade the unthreatening question. Vaborbactam is what’s called a non-β-lactam β-lactamase inhibitor. It was initially discovered by Rempex Pharmaceuticals, a subsidiary of The Medicines Company, but has been pushed through to commercialization by the latter. Basically, it inhibits the enzyme that is responsible for the degradation of the antibiotic and, in doing so, is able to restore the potency of an antibiotic that might otherwise have lost potency (or in other words, become useless through antibiotic resistance).
This is a pretty big deal for The Medicines Company, as it serves as proof of concept for using this method to restore potency across a wide range of gram-negative bacterial infections (and, specifically, the antibiotics used to treat them) going forward.
Ahead of the bell on Wednesday, markets are yet to respond to the news. The Medicines Company picked up a couple of percentage points during standard trading on Tuesday and has gained around 6% during after-hours trading, but we expect the company will appreciate further into this coming Wednesday session as and when standard participation gets underway.
The second mover of note today is Acorda Therapeutics, Inc. (NASDAQ:ACOR). On Tuesday, we learned that the FDA has issued what it calls a Refusal to File (RTF) to the company as a response to Acorda’s New Drug Application (NDA) for an asset called Inbrija.
This one is a Parkinson’s disease drug and it is designed to help patients overcome the so-called OFF periods that are associated with levodopa treatment. Levodopa is the standard of care treatment in the space, but it only works for certain periods of the day. The periods for which it works are called ON, and for those that it doesn’t, OFF. The latter periods are associated with some pretty nasty symptoms, primarily rooted in dyskinesia, which is a sort of involuntary movement type condition.
Inbrija isn’t anything new from an active compound perspective – it is just a reformulated version of the above-mentioned Levodopa. It is a new take on the drug, however, with the administration method being inhalation. The idea is that the standard administration Levodopa (generally intravenous or subcutaneous) could deal with the major symptoms of the disease, while the inhalable alternative could be used as a sort of maintenance type, top-up administration version of the treatment.
Data from the phase 3 trial that supported the application looked relatively strong, but the agency in the US clearly isn’t happy with what has been presented. We don’t have too much information as to what has gone wrong, but we do know that the company has noted two reasons why the FDA issued the RTF. Taken directly from the PR announcing the development:
“First, the date when the manufacturing site would be ready for inspection, and, second, a question regarding the submission of the drug master production record. FDA also requested additional information at resubmission, which was not part of the basis for the RTF.”
Acorda is down 25% on the news and there is a good chance we will see a continued depreciation early session on Wednesday and at least until we get some degree of insight into how the company plans to deal with the setback (and most importantly, what it’s going to cost).