Just listened to the latest quarterly call:
Key things:
1. Over 110 of the total 130 total patients recruited across all trials. Currently still expecting to finalise enrollment this year.
2. Based on the relatively high number of patients recruited, expecting to apply for Expedited Review in the form of Accelerated Approval for products that meet an unmet need from the FDA. Accelerated approval is also being entertained as 2 of the current treatments (Fabryzyme & Galafold) were approved this way via surrogate markers, but for 102 uses clear eGRF slope changes. Galafold was approved based on reduction of GL-3 in kidney cells vs Placebo, and Fabryzyme approved based on another biomarker - the amount of lysosomal storage based on kidney biopsies. However eGFR is now taken as the gold standard for assessing Fabry treatment efficacy, and for which again in the latest Bridge data we see quite astounding results in the reversal of the degrading slope.
3. Call appears to confirm again that after 102, OPRX-106 is the key product from continued development, (110 taking the back burner). While they say they are in discussions with potential partners on 106, they also state that they may wish to go with running their own large phase IIb trial, based on the positive feedback from the physicians, and the ability to negotiate a better position for themselves with larger bedrock of data. While yes this will ultimately mean more spending from PLX I agree with them on this for growing greater shareholder return on investment in the long-run.
4. They have 42 million in cash at the moment, which is expected to take them to at least filing of the Phase III results and dependent on attaining Accelerated Approval may take them to full approval. This one would imagine would be something like data read out end 2019-early 2020 with approval in mid-late 2020.
Costs are expected to go down as the 102 trials taper off. Cost for a new potential beginning of OPRX-106 looks like thy would be deferred as they state that this decision to go alone or with a partner will depend on discussion with FDA on 102 review process and timelines. This would be good in terms of making sure the cash they have does get them to the above mentioend 102 finish lines.
Then they would receive significant cash milestones from Chiesi and then push with the Phase IIb OPRX-106 trial, which I would expect to be faster to run, both for recruitment and for actual trial duration (note the recent Phase IIa 106 trial of 20 patients took 5 months to complete, and Topline data 1 months later) and hopefully overall not as expensive as for Fabry.
Cost basis is also expected to be supported by the recognition of R&D support payment from Chiesi. However the 25 Million upfront from the new US deal while received will not be recognised until commercial production begins (same as with the 25 million from the ex-US deal).
5. No other new expected partnerships to be announced in 2018.
6. Lower sales of taliglucerase to Brazil and Pfizer, but with a shipment ready to go out to Brazil which should be thus recognised in the next quarter.
All in all looking positive, but I can see that investors will be waiting to see full data and also will be wary of continued potential expenses with the potential costs of 106 Phase IIb being covered only by PLX. And thus hoping to get in later for cheaper. Thus keeping the valuation depressed compared to where 'it should be'.
AI
