Bio-technology investors are all too familiar with PDUFA dates and what they can mean for companies that have a pending approval. Many factors influence the FDA’s decision when considering a drug candidate for approval. In this case, there are some factors they weigh heavily in favor of A.P. Pharma, Inc., (Public, NASDAQ:APPA).
TapeBeat.Com, the news and research portal for the serious investor is proud to present information regarding A.P. Pharma, Inc., (Public, NASDAQ:APPA) a specialty pharmaceutical company based in Redwood City, California. Its current pipeline utilizes a group of polymeric compounds dubbed Biochronomer as its drug delivery mechanism with a number of advantages over current delivery means. A.P. Pharma’s website claims the product is easy to manufacture, is inert to acid sensitive compounds such as nucleic acids and proteins (most biological systems function under slightly acidic conditions), has flexible delivery times and mechanisms and can take different forms ranging from glassy crystalline to injectable semi-solids. Probably the most important advantage of the Biochronomer system is that the actives (drugs) can be incorporated into a drug matrix by simple physical mixing means that allow injectable gels, microspheres, coatings and strands to be created depending on the necessary application. Each of these different forms is capable of controlling site and speed of the active ingredient delivery without the use of active and sometimes questionable liquids such as solvents in the finished drug product.
A relevant point from above is that the polymer (drug delivery system) is inert, just like other polymers such as PTFE (Teflon) and other plastics utilized in medicine for temporary (IV tubes) and permanent (hip and joint replacement) applications utilized in biochemical settings every day. Working on the assumption that these polymers are virtually inactive chemically in a biological system (our bodies) and the drugs they carry are released at a pre-determined and acceptable rate; the safety concerns of the product should be minimal.
On March 18th, 2010 A.P. Pharma expects to hear from the FDA about its PDUFA (Prescription Drug User Fee Act) for APF530, an injectable antiemetic for chemotherapy-induced nausea and vomiting (CINV). According to the website en.wikipedia.org, there are two types of CINV. Acute onset CINV occurs within the first day following chemotherapy treatment and delayed onset CINV occurs more than one day after treatment and may persist for several days. As anybody who has experienced uncontrollable vomiting and nausea for an extended time can testify, this greatly diminishes one’s quality of life. It can affect a cancer patient’s decision on delaying and duration of chemotherapy treatment.
Currently there is only one drug approved for both acute and delayed onset CINV via serotonin 5-HT3 antagonists, Palonosetron. Serotonin 5-HT3 antagonists work by blocking the effects of a chemical called serotonin, which is produced in the brain and stomach. They prevent and relieve nausea and vomiting caused by chemotherapy. APF530 works by utilizing its delivery system and the 5-HT3 receptor antagonist, Granisetron, as its active ingredient. Granisetron was approved by the FDA for the treatment of acute onset CINV in 1994. The patent on it expired in 2007, so it is freely available for use in APF530.
An advantage of utilizing a previously-approved active ingredient is that the NDA can be submitted under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act in which the application can rely upon the FDA's previous safety and efficacy profile for APF530's active ingredient. The 505(b)(2) approval route has been widely utilized since its implementation in September 2004. The number of approvals using this process is increasing annually and is much cheaper and quicker than the route of proving the entire efficacy and safety profile of a new form of a drug. According to the FDA’s website and http://www.contractpharma.com/articles/2010/03/trends-in-505b2-approvals, in 2009 35 drugs were approved via this route. This was a dramatic increase from earlier in the provision’s lifespan. For example, from 1996 to 2001 there was only an average of about 4 drugs approved annually via the 505(b)(2) route!! Pharmaceutical companies are beginning to see the merits in utilizing this pathway as a quicker and cheaper means to getting some of their qualified drugs to market. More information on the 505(b)(2) pathway to approval can be found on the FDA website, http://www.fda.gov.
According to phase III data presented by A.P. Pharma on September 2008 on 1395 chemotherapy patients, APF530 was shown to be equally as effective as (statistically non-inferior to) Palonosetron in the prevention of both acute onset and delayed onset CINV. The information available arguably increases the likelihood of approval for APF530 for at least the acute if not both the acute and delayed onset CINV. Doing so would give oncologists around the U.S. another weapon in their arsenal against cancer. This should benefit both the patients’ quality of life and tolerance for the only weapon against certain types of cancers, chemotherapy. For A.P. Pharma, this would be long term win and a great stride forward in further development of its Biochronomer drug delivery system.
Disclosure: Mr. Ramey is long APPA