The license agreement with Chiesi for the global rights (ex. North America) of Raxone in LHON marks Santhera’s exit from neuro-ophthalmology. The proceeds will be reinvested in the current pipeline to bridge a funding gap until the 2020 turning point with the anticipated EU conditional approval of Puldysa (80% filing success rate) in DMD and the readout of the vamorolone pivotal “VISION-DMD” trial (35% phase IIb success rate) in DMD. The peak sales potential of both DMD drugs are 26 times higher than the peak sales potential of Raxone in LHON (ex. North America). Given the relatively high success probability of Puldysa EU conditional approval, we believe the sale of the Raxone LHON rights to Chiesi should pay out nicely. Santhera remains significantly undervalued compared to other DMD players.

 

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Key catalysts include:

  1. POL6014 phase I MAD trial results (H2 2019)
  2. Puldysa EU conditional approval (mid 2020)
  3. Vamorolone “VISION-DMD” pivotal trial results (mid 2020)