Are there any shortfalls of production at small scale?
The only thing that doesn't become easier at small scale is certain aspects of compliance, such as batch records. If you were
making Herceptin or Rituxan in a 12,000-L bioreactor, you would have a large batch record; it's the same size batch record
if you're only making 12 L for a patient lot: the size of the bioreactor has come down, but you still have the same compliance
How much protein does each batch or lot yield? How does this quantity relate to the dosage needed for the patient? Does the
patient receive dosing over an extended period?
The process was identified as being sufficient to produce enough product to treat a patient for at least three years. Patients
with indolent non Hodgkin's lymphoma are faced with eventual disease progression in most cases. FavId is designed to extend
the time to progression for patients with follicular lymphoma, one of the most common types of lymphoma. Each patient receives
six initial monthly subcutaneous injections of FavId along with four daily subcutaneous injections of Luekine (GM-CSF), a
cytokine intended to enhance the immune system response to FavId. If the patient's disease is stable or in remission, then
he or she moves into maintenance therapy and receives bi-monthly injections for one year and then quarterly injections until
Is this type of production economically feasible? How does your cost of goods compare with those for mass-produced biologics?
It really is a completely different business model than mass-produced biologics, and until a few years ago, it was not economically
feasible. Today, by using our proprietary processes including the use of baculovirus and insect cell cultures, we have rapid
turnaround time for the product, and "time is money." Moreover, the availability of affordable disposable materials and equipment
has helped to make this new approach to medicine practical and affordable.
How will personalized medicine affect patient care?
We have found that there is great variability within one type of cancer: there are many types of breast cancer, colon cancer,
etc. This is because there is variability in the types of mutations leading to cancer; we need personalized therapy to address
the specific tumor. It used to take too long to sequence the DNA and generate the protein. It was too expensive and time consuming.
We have completed over 300 lots for our Phase 3 trial; we have an impressive data base before going commercial. That's rare
for the biotech industry, which often has only 5–10 lots at commercial scale when they apply for a license, because each lot
is so expensive.
This means we're sitting on 300+ cell culture runs, purification runs, and aseptic fills. If you look at final product testing,
which requires 10–20 tests, and multiply that by 300, there is a huge amount of quality control testing data available.
How long do you think you will get approval?
Assuming positive data from our ongoing registration trial, we anticipate approval in early 2009.
Is it challenging to get approval (of product or process) because your clinical trial patient numbers are so small per lot?
We have a process that is shown to be reproducible for the wide variety of patient tumors we receive. Our historical data
should demonstrate a very robust and licensable product. The clinical data will speak for itself; a safe and effective product
should get rapid approval. Once approved, we anticipate rapid market penetration.
How long do you think personalized medicine will become a routine, or even a central part of production and a business model
in the industry?
The use of personalized treatments is already occurring and the use of personalized products is in its infancy, but I expect
in 10 years we will see this as a significant sector of the biotech industry.