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Re: DewDiligence post# 154152

Friday, 05/17/2013 6:54:54 PM

Friday, May 17, 2013 6:54:54 PM

Post# of 252175
PRTA - 4Q12 CC notes (3/28/13)

Doing a bit more DD on PRTA (ELN spin-out) given what I think is cheap valuation (sub-$200M), big cash balance/no debt, and interesting pipeline so a few notes on their prior 4Q12 CC:

1. Lead drug, NEOD001, targeting AL and AA amyloidosis (orphan condition) is in Phase 1. There are an estimated 15,000 patients in U.S. and Europe with AL amyloidosis and 8,000 with AA amyloidosis. The effective dose in pre-clinical studies for this drug was 4mg/kg and management noted how monoclonal antibody pre-clinical dose data generally translated over pretty well into the clinic so that's the level they expect to see activity in humans. Management expects this dose level and potential activity will come during the MAD part of Phase 1 in 2014.

2. PRTA's second drug is NEOD002, an antibody which targets alpha-synuclein for treatment of Parkinson's disease. PRTA expects to file IND and initiate P1 trials for this drug in 2014.

3. PRTA's third drug is MCAM, an antibody which targets a cell adhesion molecule for inflammatory disease and metastatic cancer. PRTA expects to file IND and initiate P1 trials for this drug in 2015.

4. PRTA's existing cash will see them through P2 data for NEOD001 and P1 data for both NEOD002 and MCAM (i.e., at least well into 2015).

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