Replies to post #172949 on Biotech Values

[masterlongevity]

Look at soliris for an example of this strategy.


Also, most people don't remember, but rituxan was an orphan approval

[DewDiligence]

XOMA—[Gevokizumab] probably worth a 20 to 40 percent (WAG) premium once more broadly approved in general NIU.

I don’t understand what you mean by a 20-40% premium in this context—please clarify.

[GrthzGd]

Seeking orphan approvals first. Other possible reasons besides pricing:

(1) crossing the lower regulatory hurdle for an orphan may translate to a smoother regulatory approval for wider uses later on, and/or

(2) orphan markets resemble beachheads under classic startup theory. I.e., By attacking and serving the strongest, localized "pain point" first you then cheaply establish credibility, garner "evangelists" and fund the program with early, high margin revenues before attacking the wider market, where you otherwise would need to spend more marketing dollars to displace existing solutions.

Just guessing here....

[nuere]

XOMA
There are 3 IL-1 inhibitors on the market associated with the ligand/receptor signaling interaction, anakinra (Kineret; Amgen/Biovitrum), canakinumab (Ilaris; Novartis) and rilonacept (Arcalyst; Regeneron).
Anakinra and rilonacept inhibits the effect of IL-1alpha as well as those of beta.
As I understand it, diacerein/rhein prevents, the formation of IL-1beta and not the receptors.

Currently there are 3 IL-1 inhibitors on the market in the world. 2 are Mabs and approved in the US in orphan indications. But the third is diacerein, approved in the EU for treatment of Osteoarthritis. The MOA is very different and probably less clean than the Mabs - it works by preventing the creation on the IL-1 receptors (by interfering with ICE - part of the activation system for creating the receptors). The caveats to the pertinence of the diacerein data to Xoma's osteo trial are:

[iwfal]

XOMA - One more note (a setup for interpreting EOA data). Having read multiple previous failed attempts to treat EOA it is pretty clear we don't know with much confidence what "healing EOA" looks like on a scan. Multiple trials with 'positive' scan data - but null or, often, meaningfully worse patient symptoms.

With that said - what I'd look for is strong data (low p value - e.g. 0.001) in one or more of the 'typical' EOA metrics (about 10ish - but then with multiple possible subgroups like symptom responders, joints with baseline palpable swelling, ...). If they used MRI for all patients (their presentations are not irrefutably clear on this) that may to be possible since MRI has such high fidelity wrt various parameters relating to EOA.