Aside from the 3 current competitors, I see no immediate important competition coming though the pipeline.
As far as I can see there are only 2 potentials on the horizon but neither look very promising I feel and neither look set to hit the market anytime soon, giving PRX-102 some good shielding from the only potential new agents for Fabry. The 2 are : 1. Sanofi Substrate Reduction Therapy, and 2. Sangamo Gene therapy
- First For Sanofi, they are currently in Phase III, and have 2 ongoing studies. The larger of the 2 studies, (114 expected participants), is focused on subjective endpoints of neuropathic and abdominal pain, vs placebo. eGFR isn't even an endpoint in the study. The smaller of the 2, (90 expected participants), has it's primary endpoint being heart related on Left-Ventricular Mass Index, vs Fabrazyme, Replagal & Galafold. It does have eGFR as a secondary endpoint. But it seems that's not the key item they are going for if they aim to get approval.
I believe Dr Warnock previously referred to the first trial as being heavily delayed in recruitment due to the subjective endpoint. Additionally, with their stated primary endpoints I'm not sure they would have an easy time getting approval. Regulators don't like subjective endpoints and proving a change in LVMI, I recall Dr Warnock also saying takes a lot longer to see any appreciable differences. The study obviously does reflect this at 18 months to outcome, but even then.
Additionally, with the trials being vs placebo and Fabrazayme/Replagal/Galafold, respectively, there is obviously no direct comparison vs PRX-102, which while Balance and Bridge show superiority to the ERTs (galafold is for a different amenable subset of patients), gives PRX-102 some shielding.
Also additionally, based on the study designs, I don't think Sanofi is looking to cannabilise on Fabrazyme, but would look to position Venglustat as a complementary therapy to support in pain and heart indexes. So would not be direct competition for PRX-102 anyway.
Finally on Sanofi, the latest data I can see, related to a 9 person phase 2 trial, of which Venglustat has no impact on reduction of "GL3" which is a different name for GB3 by week 26, hence failing it's primary endpoint. Seven participants continued to the extension study, where changes were said to be observed at 3 years later. Though obviously this is based on a very small number of patients.
So I think we can see that it doesn't look like a great prospect. It also failed a separate trial in autosomal dominant polycystic kidney disease (ADPKD) in 2021, which looked at reducing GL1. Then tag on the Idorsia failure in Fabry.
- Secondly, there is one remaining gene therapy programme that is ongoing via SGMO, but they are taking forever to recruit patients into their Phase 1/2 dose-ranging trial so things would appear to be something like 6-7 years away if all goes according to their own 'unlikely' plan. By unlikely I mean that SGMO has mentioned that they intend to try and save time and go straight to a Phase 3 without even finishing their extraordinarily delayed Phase 1/2, but to me that looks fanciful. Additionally they had already mentioned wanting to do this about 2 years ago after data from just 4 patients. Obviously that didn't get anywhere either. So far it has taken them 4 years to recruit just 13 of the 48 expected enrollment participants.
At the base of this post, I herein post a post I posted :) on the SGMO ST board, for which I only got one person responding to me and didn't really answer the question in any useful manner.
Thus that is my summary on current DD. PRX-102 I feel stands to gain strong market share. I believe based on the Japan Bridge study design that Chiesi and PLX are confident to get both once per 2 weeks and once per month labelling. I believe Replagal will lose out largely to PRX-102 in ex-US. I believe Fabrazyme will lose out significantly in the key US market, as they will lose their US ERT monopoly and I'm sure many patients and their physicians will be keen to try the new drug. I see no important competition coming through for the reasonable future e.g. minimum 7 years out, probably more.
SGMO post:
"Something I don't get. How are they [mentioning they are] planning to do a Phase 3 trial in Fabry, if they have only recruited 13/48 (27%) of the patients in the Phase 1/2 trial? Of which from the new [worldsymposium] presentation it would seem only the first 6 patients have been on the therapy for more than 6 months.
I also see that they were talking of planning a Phase 3 trial all the way back in November 2021 off the results of the first 4 patients in the Phase 1/2 trial. Seems a bit of a small number on both accounts to go straight to Phase 3. Anything happened to delay the previous Phase 3 [plan]?
I don't get how they would go to Phase 3, without at least completing the Phase 1/2? Or at least having something like at least 1 year data on at least half of the intended 48 patients stated for phase 1/2 enrolment.
I get that after the first 9, they go into "expansion cohorts", but still seems a very low number of patients to go straight to Phase 3.
Also I see no eGFR efficacy data which is the gold-standard of efficacy in Fabry.
Seems odd. Please feel free to let me know what I'm missing here.
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