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$FSDC (thread): Merger with Gemini in January. Focused on huge GA/dAMD market (think multiples of wAMD). No current Tx, $APLS C3 first to market, with other C inhibitors to follow $ISEE $NGM. Gemini is to GA a bit like LOXO was to cancer. Precision ophthalmology -> Disruptive Tx
Basic science was covered in this terrific and comprehensive thread by @JamesEKrause ,also note exchange with @BiotechPort Pretty clear that CFH (and CFI) plays a critical role in "some" GA/dAMD patients; that "some" = 2 to 3x wAMD market (gulp) https://twitter.com/JamesEKrause/status/1344739379454107656?s=20
Or why all GA clinical trials look at responses in CFH (and CFI) variants; they are a significant % of GA pts. One way to think about GA is that CFH/CFI WT's act as modulators overseeing the complement cascade, and muts lead to increase in inflammatory components.
Instead of treating all GA pts with C3 and/or C5 inhibitors, Gemini's disruptive idea is to provide recombinant CFH (and CFI) to only those patients who have CFH (or CFI) variants. 1st product GEM103 is a recomb CFH, & POC trial is enriched for CFH variants. Readout likely in May
It's only a 3 mo endpt; typically GA trials need at least 6 mo, or 12 mo, to show a delta. But in a patient enriched population, 3 mo could be possible (else 6 mo), and a complete game-changer. No wonder FDA just fast-tracked the product. But Gemini doesn't stop there.
For those patients responding to CFH (monthly to 3-mo injections), Gemini will offer a GT option with an AAV8 IVT injection as a one-time therapy. Essentially doubling down on precision therapy. P1 trial with GEM103 was clean, no inflamm, good biomarker data.
Also have their own natural history GA study rather than rely on legacy studies for control GA rates. And they get to repeat recomb+GT strategy with CFI variants -> more market. Cool science, strong mgmt, A+ investors. Much thx to @buysidebio for the original idea and discussions
