Forum Topics PYC PYC Announcement today

Pinned straw:

Added 2 months ago

PYC announced today their fourth drug candidate for Phelan McDermid Syndrome (PMS): “..... has been able to restore the missing SHANK3 protein that causes PMS in neurons derived from a PMS patient.”

PMS being the same genetic condition NEU is going after with their NNZ2591 molecule. The approaches are very different with PYC using a RNA drug.

PYC have been able to grow the neurons from an affected and two unaffected individuals in the lab, apply the PYC drug and have had a positive response.

This is of course a positive however a very early step in the development of this drug.   Essentially what was observed was the positive results for the “organ in a dish” trial as we have seen for the 3 other drugs they have under development.

However what will cause investors to take notice, is if PYC have further success in their dose escalation clinical trials for their most advanced drug for the treatment of Retinitis Pigmentosa type 11. This represents the frontier of testing of their suite of drugs in humans.   We will find out between now and Christmas. 

2 months ago

@Scoonie it is certainly good to keep an eye on these developments (speaking as a $NEU holder).

It is worth noting that from the start of Phase 1 Clinical Trials until the approval of an NDA (using the US FDA as the reference) the time frame is typically 6-7 years. For rare conditions (like PMS), the timeframe for drug development is typically 3 to 4 years longer, because of the challenges involved in patient recruitment. That is certainly what $NEU has experienced with its treatments to date.

So, we are likely around a decade away from seeing these treatments on the market, in the success case. Lots of water reamins to pass under the bridge.

From an investment perspective, this is a good reminder to be careful in modelling terminal values. Drugs eventually face revenue decline, whether from generic competition post patent expiry, or from substitution from future advanced treatments. So, a sustainable pharma company has to have a strong R&D process and portfolio, and the capability to reinvest a proportion of its cashflows into creating future growth, organically or inorganically. Big pharma does a combination of both.

From my perspective, this isn't something to be too concerned about at this stage in valuing a company like $NEU, as multiples are ultimately supported by M&A - major companies are always on the lookout for good drugs to buy in their areas of theray focus and the multiples can be very attractive. But it will definitely become a major consideration once a company like $NEU has all its products launched (3-4 years time). You then have to ask "what's next" and "how long is the runway" ahead?