On September 24, 2015 shares of Arrowhead were up as much as 20% after announcing positive phase 2a trial results for its Hepatitis B trial. Investors thought the results were good which is why the stock ended the day on the positive side. In addition to obtaining positive phase 2a Hep B trial results in humans, the company released pre-clinical results for Hep B in Chimpanzees. The human testing was a single dose of ARC-520 together with a NUC known as entecavir, on the other hand the Chimpanzee trial combined both ARC-520 and NUCs — like nucleoside inhibitors used as standard of care for Hepatitis B patients.
The human patients who were HBV e-antigen positive took a combo of 4 mg/kg of ARC-520 — single dose — together with a NUC such as entecavir. These patients that took this combo achieved a 92% gene knockdown of the Hep B virus. The maximum gene knockdown observed with this combination ended up being 98%.
As mentioned above four chimpanzees who were HBV e-antigen positive showed a 99% gene knockdown by taking between 6 to 11 monthly doses of ARC-520. The downside though is that the chimpanzees who were HBV e-antigen negative displayed only an 81% gene knockdown. Thus far the gene knockdown numbers are good but now we will have to wait to see if the FDA will allow for multi-dose trials of HBV in humans.
To date though ARC-520 is safe and tolerable for all the patients that have taken it. Of the 84 or more patients who have received doses of ARC-520 there have been no serious adverse events. If the FDA allows for multi-dosing then it is highly possible for Arrowhead to possibly eventually obtain a functional cure for the Hep B virus.
One company probably eyeing the development of Arrowhead’s HBV candidate ARC-520 is probably Gilead Sciences (NASDAQ:GILD). This is because Gilead bought a company known as Pharmasset back in 2011 for approximately $11 billion. Gilead did so for the purpose of getting its food in the door in the Hepatitis C space. Gilead has since been taking market control in the Hepatitis C space. Now it could possibly look to capture an even bigger market, bigger than Hepatitis C, the Hepatitis B market. If Gilead does want to add Arrowhead into its arsenal it will probably have to offer up a substantial offer to management to justify a possible buyout.
Heron Therapeutics (NASDAQ:HRTX)
On September 22, 2015 shares of Heron Therapeutics soared as high as 22% in after-hours trading after announcing positive phase 2 results for post-operative pain. The phase 2 trial used a drug entity known as HTX-011. This drug molecule uses Heron’s delivery technology known as the Biochronomer drug delivery technology. The HTX-011 is a local anesthetic drug in combination with an anti-inflammatory molecule known as meloxicam.
The phase 2 trial that Heron ran recruited up to a total of 64 patients that were split into different dosing groups. One group of patients received 200 mg or 400 mg of bupivacaine combined with meloxicam. The other group of patients received a placebo compound instead to compare it to the live drug. The primary endpoint of the trial was looking for the pain intensity score known as the Summed Pain Intensity — SPI — score.
With the SPI patients are measured between the drug and placebo group to determine the amount of post-operative pain experienced after a surgery. It was all good for Heron Therapeutics as the primary endpoint was achieved with great ease. This is because Heron’s drug combination together with HTX-011 displayed an SPI score of 38.5 which is way lower than the placebo SPI score of 124.2. This means that HTX-011 displayed a 69% decrease in pain compared to its placebo counterpart.
Passing on the primary endpoint is what any biotechnology company could hope for, but the company’s drug was even able to pass all secondary endpoint as well. One thing to note though is that this trial tested for post-operative pain for patients undergoing bunionectimies, but this type of pain reduction using HTX-011 could also be applied to other post-operative pain as well. This means that this drug could obtain market share for multiple opportunities in the pain sector.
Vertex Pharmaceuticals (NASDAQ:VRTX)
On September 25, 2015 Vertex Pharmaceuticals announced that the European Union Committee For Medicinal Products for Human Use — CHMP — issued a recommendation of a Marketing Authorization of Orkambi for the treatment of Cystic Fibrosis patients that are age 12 and older that have two copies of the 508del mutation. Orkabmi is a Cystic Fibrosis drug combining lumacaftor and ivacaftor — also known as KALYDECO. KALYDECO was the company’s first approved drug to treat patients with Cystic Fibrosis.
The European Union even felt it necessary to expand approval for KALYDECO to patients that are between the ages of 2 to 5 with Cystic Fibrosis. On top of that the European Union also wanted to approve KALYDECO who have these nine gating mutations listed below. These mutations include:
One thing to note though is that this does not mean that these compounds are approved for use in Europe already. This now means that the European Union itself has three months now to decide whether or not Orkambi and KALYDECO should be approved for these indications listed above. Typically though the European Union follows the recommendation of the CHMP just like the FDA typically follows the recommendation of an FDA advisory committee. With that in mind then it should be no problem for the European Union to approve Orkambi and KALYDECO.
[Image Courtesy of the European Journal of Molecular Biology]