Soligenix Inc (SNGX)

[jellybean]

Dr -- have listened to the conference call and looked at the available publications (except the entire Blood Jan 23 because I refuse to pay $25 for a publication that will soon be available for free).

Here are my thoughts, fwiw:

Primary endpoint missed, but there is a scientific explanation for that miss and the trend was in the right direction.
Secondary endpoints all impressive, especially the reduction in death.
Trials were small and few but consistent.

GVHD is a side effect of bone marrow transplants which is a curative therapy for many diseases ranging from cancer to Wiskott-Aldrich.

One last comment... the FDA considers trial size with respect to the proportion of the prospective patient population. dor has enrolled 200 patients out of a potential yearly pool of 10,000. 1 out of 50 is a pretty good representation.

A bone marrow transplant does not progress to death in the majority of patients and so any therapy that reduces the risk of death associated with the transplant is impressive.

The use of steroids for GVHD is a well known proven treatment; this is an improvement in SOC.

Comparing orbec to provenge seems unreasonable. Although at a big picture level there are some similarities (small patient numbers, missed primary endpoints, overall survival benefit) there are some glaring differences.

1. Survival as a secondary endpoint was prospectively designed into the trial and so there are no questions regarding type1 errors.
2. Provenge PROLONGS life by a mean of 4.3 mths (go read the statistical review and you will see that this is the number the FDA stats guy presents its 23.2 vs 18.9 for the combined trial datahttp://www.fda.gov/ohrms/dockets/ac/07/briefing/2007-4291B1_03a.htm), while orbec PREVENTS deaths. That is not a subtle difference.
3. The ph2 and ph3 trial results were consistent and should if combine enhance the efficacy numbers. One of the comments made by the FDA statistical reviewer for Provenge was that The Guidance for Industry--Providing Clinical Evidence of Effectiveness for Human Drugs and Biological Products (see ref: [1]) explicitly says “With regard to quantity, it has been FDA’s position that Congress generally intended to require at least two adequate and well-controlled studies, each convincing on its own, to establish effectiveness.” It implies that positive results from at least two adequate and well-controlled trials are, in general, required for licensure application. Two positive trials not only substantially reduce the chance of making a false claim, but also assure that a result could be more robust, replicated in different settings, and more representative to the targeted population.


This drug is already on the market, and therefore, a known entity and available.

What would be the result of approving this NDA:

1. deaths will be prevented

What would be the result of not approving this NDA:

1. unnecessary patient deaths
2. some risks as docs may try to use the drug off-label which is not safe

At the risk of encouraging the Dendronauts, one comment. The Provenge trials were not powered or designed to look at overall survival, but the orbec trial was....is a substantial difference. And, while David Miller may not be able to come up with a reason for what could happen to improve overall survival between the treatment arms, that may only be because there is still so much about cancer that is unknown.