If the ongoing drama and wild stock movements with Sarepta Therapeutics (NASDAQ: SRPT) and its back and forth with the U.S. Food and Drug Administration (FDA) has proven one thing, it’s that holding a stock on the brink of a major FDA approval or rejection is no easy task.
One of the reasons for the extreme volatility is the vanishingly small amount of data and number of patients involved in its controversial muscular dystrophy trial. This type of volatility can be avoided somewhat by focusing on companies that have just successfully completed larger Phase 2 trials instead.
A successful and relatively large Phase 2 trial often provides critical backing for a pivotal trial that is often lacking in more volatile companies like Sarepta . They also tend to be overlooked compared to pivotal trials, allowing shrewd investors to get in before the crowd. Here are three biotechs that just recently announced successful Phase 2 trial data for drugs with very big addressable markets.
Teva Pharmaceutical Industries Ltd. (NYSE: TEVA) just announced top-line results from one of its leading studies in a chronic migraine indication. The drug in question, TEV-1845, is part of a family of drugs called calcitonin gene-related peptide monoclonal antibodies, and this family has been hailed as a big step forward in the migraine space for the past five years. As yet, the FDA is still to approve a candidate in this class, and Teva is now closer to being the first.
The trial was a Phase 2b double-blind, 261-patient study designed to pitch both a low and a high dose of TEV-1845 against placebo across a three-month regimen. Primary endpoints and secondary endpoints were a reduction in the number of headache hours and headache days.
The trial hit on both counts, reducing both headache days and hours from a baseline measure at both dose levels. A pivotal trial is expected to kick off before the close of the fourth quarter, and we could see a New Drug Application (NDA) during the equivalent period in 2017, if all runs smoothly.
This one is another Phase 2b, this time for schizophrenia. The drug is a 5-HT2A receptor antagonist called MIN-101 and developed by Minerva Neurosciences Inc. (NASDAQ: NERV). The 5-HT2A receptor is currently one of the primary targets in psychotherapy drug development. Research has shown that too many of these types of receptors are closely linked to depression and schizophrenia. This hypothesis derived from investigating the brains of suicide victims.
The receptor itself is linked to serotonin production, which suggests that overproduction of serotonin may lead to psychosis. MIN-101 is designed to inhibit the receptor, and in turn, reduce serotonin levels.
Minerva just released data from the trial, which was designed to test the efficacy and safety of the drug in patients with symptoms of schizophrenia. The primary endpoint was a statistically significant improvement in these symptoms over placebo, and it came in as met when measured using an industry standard scale called the PANSS three factors negative symptoms subscale. Secondary endpoints measured the drug’s impact versus placebo using other scales, including the Brief Negative Symptoms Scale total score, Clinical Global Impression of Severity and the Clinical Global Impression of Improvement, among others. All came in as met.
This one is carrying through into an extension before moving into pivotal trials, so the next major milestone is the release of the continuation data, set for before the end of the year. A pivotal trial should commence come in the first quarter of 2017.
Oramed Pharmaceuticals Inc. (NASDAQ: ORMP) is going after one of the holy grails of the biotech/health care space: oral insulin. The company just reported top-line results from a Phase 2b as well, designed to gauge the safety and efficacy of its lead oral insulin candidate, the ORMD-0801 capsule.
The study measured its efficacy against a primary endpoint of a reduction of night-time glucose, as measured by way of a weighted mean. In 180 patients across a 28-day period, the drug demonstrated a mean reduction of night-time glucose to the tune of 6.47% between the active arm of the trial and the control placebo arm. Tolerability proved no real issue, with no serious adverse events reported in any patients — rare in a trial of this size.
Oral insulin has been elusive to biotech companies big and small to date, primarily due to the difficulty of getting the insulin to the liver without it being absorbed and digested on its way through the gastrointestinal tract. Oramed has designed ORMD-0801 to resist premature breakdown, and the trials to date look as though its design may be effective.
We should get some more detailed data across the next few weeks, and while a pivotal Phase 3 trial has not yet been announced, chances are it will be before the close of 2016.