Pardon my French, but these preclinical results are bloody...

Pardon my French, but these preclinical results are bloody fantastic news, even if the market doesn’t have a clue.

Sure, it will be said that these are only preclinical results, but the excellent preclinical models used by PYC provide very good insight, very early on, into whether PYC-001 is likely to be safe and effective in humans with the rare, inherited eye disease, ADOA1 (see slide below). In addition, these results have potential relevance to other eye diseases in which OPA1 insufficiency plays a key role.



One of the major factors that turns investors off early-stage biotechs is the very high level of risk that comes with drug development. As has been oft repeated, the overall probability of a preclinical drug making it through to market is less than 10%. An added deterrent for investors is the average 9 or so years it takes for those 10% of drugs to get to the finishing line.

PYC’s approach challenges these norms, radically boosting the likelihood of success and significantly accelerating speed to market.

As can be seen in the table below, the likelihood of the average preclinical drug achieving marketing approval is just 7.9%. Post Phase 2 success, the likelihood of marketing approval rises to just over 50%. A coin flip.



Using genetically validated targets dramatically increases a drug’s likelihood of success, as seen in the following two tables. For example, Alnylam Pharmaceuticals has demonstrated that the likelihood of success of its drugs (from pre-clinical stage to marketing approval) is 64.3%. When compared with the table above, this gives a drug just entering clinical development  a higher chance of success than the average post-Phase 2 stage drug.





PYC has not only increased the likelihood of success for its two lead drugs, it has also increased their potential speed to market.

The rare, inherited eye diseases that PYC has chosen for its two lead indications have the possibility of advancing from preclinical to approval in just 5-6 years, compared with 9 years for the average drug.

The estimated value of the combined markets of the two lead indications is $3bn.

Finally, because PYC’s drugs are significantly derisked much earlier in the drug development timeline and PYC is using its platform to progress one new drug candidate each year, expect increasing pharma interest in licensing or potential acquisition.