What is Acasti and what does it do?
Acasti is a late-stage specialty pharma company focused on advancing therapies for specific rare diseases. What sets us apart is that we are taking a rather low-risk approach to rapidly and more efficiently bring drug therapies to patients. This approach consists of taking already marketed drugs - drugs that often have been around for decades - and reformulating them, which is Acasti’s core strength. One of the ways in which we do this is by chemically altering how the drug is administered - our candidate GTX-104, which consists of the drug nimodipine, is the perfect example of this idea. Nimodipine is currently only available as an oral and is very insoluble making it difficult to absorb, which results in variable blood levels; however, with GTX-104, we figured out how to change these properties so that nimodipine can be delivered via a continuous iv infusion. This is simply one example of how we re-purpose marketed compounds for orphan diseases in a way that not only improves efficacy and safety, but also convenience, which improves compliance.
We have a deep pipeline with three drugs in the clinic, and several more in a pre-clinical stage with which we are focusing on animal studies.
We will have two drugs ready to move into phase 3 in 2023, and a third drug which is a topical spray used for a pain indication for a disease called postherpetic neuralgia going into phase two.
What are the results showing so far for your other candidate, GTX-102?
GTX-102 is an oral spray form of betamethasone intended to treat the symptoms of ataxia telangiectasia, a rare genetic, neurodegenerative disorder that affects children at a very young age. Recently, we announced topline data on our primary endpoints, and it indicated that our oral spray is being rapidly absorbed in the oral mucosa and that it is achieving blood levels that are equivalent to the injectable form of betamethasone (the only form available in the U.S.). Furthermore, we also reached the target blood levels of the oral solution of betamethasone that is only available in Europe and was used in a study published by the university of Siena, a center of excellence for diagnosing and treating patients with ataxia-telangiectasia, where the oral solution saw very positive results with the drug after only 30 days of use. So, all in all, the data so far is very promising.
Acasti enjoys an Orphan Drug Designation (ODD) for some of its drugs; which ones are they and what does the 7-years US market exclusivity mean if you want to commercialize?
We have received ODD for all three of our drugs in clinical stages. This is something one requests from the FDA - then, the FDA determines whether the company is the first to pursue a particular indication with that specific form of the drug and confirms that there are no direct competitors. If the company is indeed the first, and the drug is approved, the company receives seven years of market exclusivity - which entails that the FDA will not approve any other competitive drug for that indication. This market exclusivity is for 10 years in European territory.
Are there any other policies of this sort that incentivize the development of therapies for rare diseases?
There are very important advantages available to companies when developing orphan drugs. For example, the FDA will allow you to get a tax credit of up to 50% of your R&D costs incurred in the United States. Another big advantage is that they will often waive the PDUFA fee (Prescription Drug User Fee Amendments) which is a fee of millions of dollars that a company pays when their drug is approved.
The other important advantage is that the FDA usually provides an expedited review and approval time for orphan drugs. This can reduce the time to market from roughly one year to six months. Furthermore, they are also quite helpful and supportive throughout the development process. We have found the FDA to be collaborative with companies that focus on rare diseases, being generous and rapid in delivering feedback, guidance, and making their guidance clear.
Where do you see Acasti going in the future?
Our main objective for the next three to five years is to complete development of the three drugs that we have in the pipeline and to successfully commercialize the first two ourselves. To give you an idea, our lead drug candidate GTX-104 is a drug targeted for intensive care patients who have suffered a condition called Subarachnoid hemorrhage, or bleeding on the brain caused by a burst aneurysm. There are about 50,000 patients in the US who are treated each year at about 1,400 stroke centers, of which about 400 of the largest centers treat about 70% of SAH patients. This means we should be able to easily commercialize this product by ourselves with a small sales force. We expect to launch GTX-104 in 2025, see a good sales ramp, become profitable, and reinvest our earnings in our pipeline where we have four to six very interesting candidates at a pre-clinical stage. Finally, for us it is also very important to work with our patients and their families, reaching out to them and helping them get access to these drugs through a patient support program if they cannot afford their medicines themselves.
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