On September 8, 2015 Inovio announced that it had dosed its first patient with its Universal HIV vaccine known as PennVax-GP. This trial is being run as a phase 1 trial that will assess the safety and tolerability of the vaccine in these patients with HIV. Although the phase 1 trial primarily tests for safety, there will possibly be a measure of some type of efficacy as well. For example the efficacy could test for the amount of T-cells the patient generates taking Pennvax-GP.
This is a nice easy trial for Inovio because they just need to supply the vaccine only and aren’t running the trial at all. That is because this trial is being conducted by a partner known as the HIV Vaccine Trials Network — HTVN. Because this is a phase 1 trial this vaccine will be given to four groups of healthy patients, instead of actual patients with HIV. The reason for this is because it is a initial study to determine T-cell effects observed so that the company can move on to more advanced phase 2 trials with infected patients.
The company is also choosing to give patients PennVax-GP either with or without an immune booster known as IL-12. This immune booster helps increase efficacy of the drug for patients but the company will have to modify it as best it can without increasing any type of toxicity. That is because PennVax-GP on its own, like many of Inovio’s vaccines, are made using computer genetic code –synthetic vaccines — and water.
An initial phase 1 trial that Inovio ran with its Phase 1 PennVax-GP vaccine displayed that 24 out of 27 patients, or 89% were able to display robust CD4 and CD8 immune responses. This is a positive result because patients with HIV typically aren’t able to establish an immune response to the virus on their own. As mentioned above the HTVN is in charge of the trial thus it has given research funding to Inovio of $25 million as a contract for developing a vaccine of this caliber. While still very early Inovio’s PennVax-GP vaccine looks quite promising as an HIV treatment.
Tetraphase Pharmaceuticals (NASDAQ:TTPH)
On September 8, 2015 shares of Tetraphase Pharmaceuticals tumbled 80% after the company announced that its phase 3 trial for complicated Urinary Tract Infections — cUTI — failed to meet on the primary endpoint. The phase 3 trial, known as IGNITE2, enrolled up to 908 patients who were randomized to either receive eravacycline or levofloxacin. Patients were randomized and given a certain dose of each drug.
For instance one group of patients were given intravenous administration of 1.5 mg/kg eravacycline in the first 24-hour treatment period followed by oral administration of the drug of 200 mg every 12 hours. On the other hand the other group of patients received intravenous administration of 750 mg for the first 24-hour treatment period followed by 750 mg orally every 24 hours. The company failed to meet on the primary endpoint on eravacycline being superior to levofloxacin in this patients population with cUTI.
Considering the stock tanked 80% it is obvious that investors weren’t happy with the results either. That is not to say that the company is dead though because this is one of those biotechnology companies with a great backup plan. For instance even though eravacycline failed in the cUTI indication, it has succeeded already in another. That is because eravacycline has been tested for complicated intra-abdominal infections. In this indication the company’s drug was able to meet upon the primary endpoint establishing statistical significance.This intra-abdominal infection indication isn’t as big as the cUTI indication, but still its a pretty good size market for the company to target.
Akeiba Therapeutics (NASDAQ:AKBA)
On September 9, 2015 shares of Akeiba Therapeutics skyrocketed 53% in one day after the company had announced positive phase 2 results for its Dialysis patients with Anemia due to Chronic Kidney Disease — CKD. This phase 2 trial enrolled approximately 94 patients who were taking a placebo therapy known as rESA therapy. The rESA therapy stands for recombinant erythropoiesis stimulating agent.
The phase 2 trial used the company’s drug Vadadustat — previously known as AKB-6548 to determine if patients could be switched from rESA therapy to Vadadustat and still maintain proper hemoglobin levels throughout the life of the trial which lasted for 16 weeks. Hemoglobins are necessary in people to carry oxygen from the lungs to tissues in other parts of the body. The patients in this trial have anemia due to their CKD. The point of the test is to determine if Vadadustat can maintain or increase hemoglobin levels in these patients thus being statistically significant in the trial against the placebo compound.
Patients in the trial were split into three different dosing groups and the trial ran for 16-weeks. Patients in the trial received 300 mg of Vadadustat taken once a day, 450 mg of Vadadustat taken once a day, and 450 mg of Vadadustat taken three times per week in conjunction with dialysis. Patients with CKD go three times a week for dialysis anyways and adding Vadadustat for these patients with Anemia is more convenient anyways.
The compound Vadadustat achieved efficacy in the 16-week period meeting on the primary endpoint of maintaining hemoglobin levels for these patients. Not only did the company’s drug do that in the highest dose group was able to do that in all dosing groups. To top this off the drug Vadadustat was safe for the most part with at least 13.8% of the patients achieving some serious adverse events. Although this isn’t abnormal as this patient population is used to seeing these types of adverse events taking a placebo compound. The next step for the company is to move on to a phase 3 trial that will enroll more patients and hopefully prove efficacy once again in an attempt to gain FDA approval in this indication.
[Image Courtesy of Hylok USA]