Imagine a vanilla powder, mixed with water and taken once a day, which can significantly slow the progression of Alzheimer’s disease (AD). While this pitch sounds straight from a late-night infomercial, it’s actually the authentic promise of Accera’s medical food Axona, based on well documented research about the brain function in AD patients and the results of controlled clinical trials.
From a regulatory standpoint, medical foods sit in a place between ethical drugs and nutritional supplements. Like drugs, medical foods are regulated by the FDA but face different standards. For approval, medical foods must be shown to address a clinical nutritional deficiency and be generally recognized as safe. In the case of Axona that deficiency lies in the reduced ability of neurons to process glucose, the most abundant energy source, in the brains of patients with AD. Without treatment this deficiency effectively results in the neurons starving to death. Axona, which will be available by prescription only, works by providing these neurons with an alternative fuel source, specifically ketones.
Accera, which plans to launch Axona in the first quarter of 2009, conducted several trials to show the efficacy of Axona (called Ketasyn, or AC-1202, during trials). In a phase 2b trial, Axona was given to patients with AD in addition to other Alzheimer’s disease medications they may have been taking such as Aricept or Exelon. During the first double blind phase of this study, for patients who were ApoE4(-) (ApoE is a known genetic mutation related to AD), taking Axona resulted in a greater than 5 point improvement compared to taking placebo in ADAS-cog scores, a common measure of cognitive function. Although the effects were not as dramatic, data also suggested Axona had a disease-stabilizing effect on ApoE4(+) patients, who represent about 50% of AD patients. Accera also conducted a trial focusing on Age-Associated Memory Impairment (the natural decline in memory that may be a precursor to AD) demonstrating clinically meaningful improvements in several measures related to memory. These results point to a possibility of using Axona earlier in treatments as the inability to process glucose is known to happen years, if not decades, before the development of AD.
While Accera does not possess a composition of matter patent for Axona, the company does possess several other patents most significantly related to formulation. According to Accera President and CEO Steve Orndorff, without Accera’s proprietary formulation taking doses of Axona at therapeutic levels would generate significant gastrointestinal side effects. The company will initially offer the product as a powder to be mixed with four ounces of water, but will likely offer a pre-mixed liquid in the future.
As a substantially different treatment approach, Axona benefits in entering a market where the traditional approaches to treatment have shown relatively limited efficacy. This novel approach also means that some physicians may not fully understand the reasons for prescribing the treatment. Orndorff acknowledged the “need for some physician education,” but thinks Axona’s story is fairly clear given that reduced glucose processing of neurons is “a physiological hallmark of [AD]” that is well documented in scientific literature. Another challenge for Axona is that medical foods are excluded from reimbursement under Medicare Part D. While this policy will leave some patients to pay out-of-pocket, Accera is pursuing coverage by private payers and state Medicaid programs. With the large and growing AD population (estimated at 5M in the US in 2007 by the Alzheimer’s Association), Orndorff thinks annual sales of $400 to $500M are achievable.
Having just closed a $35M financing round led by Inventages Venture Capital, Accera plans to use the proceeds of this round for all the activities needed to launch Axona. Part of this plan includes hiring a sales force to target the primary care physicians, internists and neurologists who treat the majority of AD patients. Accera is looking to add 35 sales reps in major metropolitan areas. Beyond Axona, Accera has small molecule drugs in development in the pre-clinical stage for conditions including Parkinson’s and traumatic brain injury. And with the cash flow offered by the imminent launch of Axona, Orndorff expects Accera to look to “strategically in-license products either in the development stage or to give the sales force another product in the bag”.
Orndorff notes that Accera is not a medical food company, but saw Axona as an opportunity to both get a product quickly to market and offer another weapon in the arsenal against AD. While a food-based approach may be considered non-traditional – especially for a neurological condition in a drug-focused world — trials have shown significant results for Axona. What remains to be seen in 2009 is how the broader community of physicians, patients and their caregivers responds to this novel approach.
