Just listened to the call and nothing major to announce there.
Things in general good, enrollment has been good for Balance, but frustrating for Bright.
Compared to the January company presentation which showed:
Balance at 70% enrollment = 70%*78 = 55 patients = 23 remaining
Bright at 95% enrollment = 95%*30 = 28.5 (29) patients = 1 remaining
It is now clear that since then (in the past 4 months) Balance has recruited 12 patients which is pretty good (11 now remaining), while Bright is still waiting on that final 1 patient. I guess from the speed of enrollment one might be able to infer that in general Patients that are on Fabryzyme and replagal are patients with faster degradation of eGFR, which is why it was/is easier to recruit them into the Bridge and Balance trials, than it is to find patients for the Bright trial.
- Balance Inclusion Criteria: Linear negative slope of eGFR over approximately 1 year of ≥ 2 mL/min/1.73 m²/year
- Bright Exclusion Criteria: Linear negative slope of eGFR of ≥ 2 mL/min/1.73 m2 based on at least 4 serum creatinine values over approximately 2 years (including the value obtained at the screening visit)
Meaning, while timelines are different, essentially, Balance can only recruit those with eGFR deterioration equal to or greater than 2ml eGFR points per year, while oppositely, Bright can only recruit those with less than 2ml deterioration in eGFR across a longer timeline of 2 years.
So (while I understand this is a horrid way of looking at it) providing an inferred insight that Fabryzyme and Replagal are both not doing great for patients, and hopefully meaning easier for PRX-102 to show promise.
Though in terms of provision to the FDA for Bright Interim data the final Bright patient recruitment wouldn't be particularly valuable, I hope they can recruit the last Bright patient soon as would be good to have the timing for full Bright data read-out set, and hopefully start mitigating Bright clinical trial costs somewhat.
We now understand that taliglucerase has about 130 patients in Brazil, representing about 20% market share in Brazil, which is pretty decent from physician acceptance standpoint considering PLX isn't doing any promotion over there. Though shipment remuneration was a low 1.4 million to Pfizer and 2.2 million to Brazil in Q1 every little helps.
Still deciding on OPRX-106, and I do hope they wait till at least post 2Q FDA meeting on AA before coming to any conclusions on that. And personally, in general hopefully await new milestone from Chiesi before going it alone. For OPRX-106 they do not appear limited by any patent related timelines really as any competitor would not only need to produce their version of anti-TNF alpha, but would have to copy the entire plant based expression system to be able to compete on the oral delivery method. So given there is no specific time constraint on OPRX-106, then waiting till hoped for AA wouldn't harm the company and also would mitigate potential fund raising to us.
Again OPRX-106 patient type is quite readily available and the timeline of such a study would be relatively short, so once the finances are more in the clear, then doing the OPRX research shouldn't be a big loss of time for them or us.
