The latter half of 2016 is set to be a big period in the development stage biotech space, with a host of companies set to report data from high profile trials. Two that have the potential to move heavily on their respective announcements are Medgenics Inc (NYSEMKT:MDGN) and Kura Oncology Inc (NASDAQ:KURA). Here’s why, and what to look out for come release day.
First up, let’s look at MedGenics. The company just announced the kicking off of a phase II/III in its ADHD indication, investigating the efficacy of a drug called NFC-1. The drug is part of a family of experimental treatments called, and stick with us here, non-stimulant metabotropic glutamate receptor (mGluR) neuromodulators. They are a relatively new type of treatment, having only really been discovered early last decade, but their potential as neurological condition therapies is already well documented. In this instance, glutamate is used to mediate what are called slow synaptic potentials, while also modulating ion channels, and coupling to GTP binding proteins. The combination of these three effects can inhibit the neurological processes that cause a particular type of ADHD that derives from genetic disorders impacting the mGluR network. That is, at least, what MedGenics is hoping.
So where are we with the trial? As mentioned, the company just announced the first dosing in a phase II/III, desgined to test the efficacy of the drug in circa 90 patients, aged 12-17, with ADHD (the mGluR type). It’s a 25 site study, and will test the patients across an initial six-week period (standard dosing course is six weeks). After the initial six weeks, MedGenics will run a one week follow up. The trial is blinded with a placebo arm, and topline is asset for before the close of the year.
The ADHD market is set to become a $10 billion market by 2020, so any data that hints at the effectiveness of NFC-1 as an ADHD treatment, albeit in this slightly reduced target population, has the potential to inject some immediate upside into MedGenics’ market capitalization. Before the topline data hits at the end of the year, interim data is set for Q3, and this could be just as important going forward as the study completion. Watch for a change from baseline in what’s called the ADHD-rating scale Total Score, and change from baseline in the Global Improvement Scale, as an indication of efficacy.
Let’s move on to Kura Oncology. This one is a California based biotech with, as its name suggests, an oncology primary focus. The company’s lead development candidate is a drug called, Tipifarnib, and it’s targeting a range of HRAS mutant tumors, including lymphoma and T-Cell lymphoma. Trials in both of these latter indications have just kicked off, but it’s a solid tumor indication (various cancers) that we are focusing on for the purposes of this discussion. Why? Because data from a phase II in this solid tumor indication is set to hit press during the second half of 2016, and if it supports an efficacy thesis, it would be a strong indication that there will be further efficacy readouts in the other phase IIs ongoing.
So how does the drug work? In these sorts of cancers, mutations in the HRAS gene leads to excessive proliferation of cancer cells, and is the root cause of the spread and rapid replication that is the nature of tumor growth. Tipifarnib prevents a process called protein farnesylation (we don’t need to go into this in too much detail here) and in doing so essentially turns off the HRAS gene. This stops the proliferation process that causes the issues, and in theory, should be able to halt tumor growth and cancer cell replication in patients.
HRAS mutation root cancers are a massive market, and just as with the above-discussed MedGenics candidate, an approval could open up access for Kura to a billion-dollar patient population. As such, any indication from the ongoing phase IIs that the drug is effective would translate to some immediate upside for Kura. We don’t know specifically when the data will hit, but it’s set for release at some point during the latter half of this year, so we’ll keep an eye on things as they play out and update as we learn anything.