TO ALL,
Here is the long awaited reply from Dr. Gendreau (Cypress Chief Medical Officer):
"Sorry for the delay in responding, as you note, it is a busy and
exciting time for us. I will attempt to answer as many of your
questions as I can. Please keep in mind that I can only provide
publicly available answers, as we are very careful and aware of both FDA and SEC rules in regards to disclosure of results.
Q1) The Prosorba column is a protein-A column. That being the case,
there is little specificity of the column for RA or any other autoimmune condition, correct? Therefore, is Cypress considering other conditions (Lupus, MS, myasthenia gravis, thyroid disease, Sjogren's) where the Prosorba column may play a therapeutic role? Are tests in progress? If not, why not? Has there been any off-label use for other conditions? What was the limit of the original FDA approval? Has CYPB out-licensing the technology to anyone?
A1) You are probably correct about lack of specificity. Given that the mechanism of action in RA is not well understood, it is difficult to say with conviction that we can predict results in other auto-immune diseases, but the assumption is reasonable. Our focus is on RA as it represents the major commercial opportunity. Applicability to any other indications in the future will depend on success in RA. FDA approval currently is for ITP only. No technology has been out-licensed.
Q2) I understand that CYPB has ongoing research to try and elucidate
the mechanism by which the column helps in ITP and RA. Are you looking at the bound material from the columns? I would imagine that since various classes of IgGs bind the column (through the Fc region, mostly) then perhaps electrophoresis and conventional chromatography of the bound proteins may help identify a unique characteristic in RA patients' serum. Dr. Blank had mentioned that you are studying the blood rather than the bound proteins. What types of things do you hope to find in the blood? It is known that there is an anemia of chronic disease in RA. There is also an elevated ESR and some are positive for Rheumatoid factor (approx 70%). Any leads?
A2) The scientific program is being conducted as an extramural program
looking at a variety of issues, including genetics, immunological
markers, changes in blood parameters, complement, immunoglobulins,
T-cell markers, B-cell markers, recall stimulation responses, adsorption and release characteristics of the columns and so on. This program is still in the early stages after about 18 months of effort. No public disclosures have been made as to results at this time.
Q3) Has Protein-G ever been tested?
A3) Not to my knowledge.
Q4) How does this technology differ from plasmapheresis?
A4) This is plasmapheresis with a column in the return plasma flow line to the patient.
Q5) I understand that the side-effects include: chills/fever, N/V,
pain, respiratory difficulties.....a flu-like condition. How long do
these effects last? Are the patients immune-compromised secondary to
the removal of IgG from the serum? Rate of infections?
A5) Side effects in the trial were generally mild and appeared to be present in the blinded arm with equal frequency, i.e. they didn't seem to be column dependent. We feel that the safety profile of the column will surprise people, and it will be one of the advantages of this therapy as compared to other aggressive RA treatments. Details are not yet public, but there is no evidence of any degree of immunocompromise or related infections.
Q6) How do you see this technology comparing to Immunex's Enbrel
(TNFR:Fc fusion)? Short-term vs. Long-term?
A6) We believe each will have their place. Enbrel looks like an excellent product from what we have seen. It is important to note that Enbrel and Prosorba are targeting different populations of patients, so in a sense they are not even competing for the same patients. Prosorba will be positioned for the
most severe, refractory patients, at least initially.
Q7) What is the visibility of the Prosorba column among
Rheumatologists?
A7) Needs work.
Q8) Will this be an in-house therapy? Or could this be done on an
out-patient basis? How will physicians learn about the availability of the column?
A8) This can be done out-patient (less expensive) or in-house. A marketing effort will need to be undertaken to let physicians know about this new treatment alternative-this is not at all unique to
Cypress. Our challenges after approval will be cost-effective delivery of the therapy, in a convenient and safe manner. That is actually where the primary focus of our activities will go, in addition to exploring use of the column with concomitant therapy.
Hope this helps.
R. Michael Gendreau, M.D.
Vice President & Chief Medical Officer
Cypress Bioscience, Inc.
619-452-2323 x113" |
