Pompe the never ending story???
Q2 2019
"We also continued our investment in development of new pipeline programs such as Pompe disease, which you heard and also Fabry disease behind that."
Q2 2021
"In the middle, you see the developing continuous clinical development for g rhC1-Inhibitor for additional large unmet medical indications, and the transgenic manufacturing technology where we are developing next-generation protein replacement therapies. And there we have, of course, the early stage compound alpha-glucosidase for Pompe's disease development.
Q3 2021
"Simon Scholes
I’ve got 2 or 3. I mean, first of all, I was wondering if you could give us a timing on submission of the IND application in Pompe? Secondly, I was wondering -- I mean, I’ve noticed over that a number of gene therapy-based approaches are now in trials on Pompe. And I was just wondering if you could comment on how your own approach stacks up against those? And thirdly, just on the marketing costs. I think year-on-year, you’ve had a $7.7 million increase. I was just wondering if you could split out how much of that increase relates to leniolisib and other items and how much relates to RUCONEST?
Sijmen de Vries
Yes. Shall I answer -- the first one first then, Simon, about Pompe. Pompe’s disease, we’ve taken -- of course, our product -- our development is hampered significantly at this point in time by unavailability of materials necessary for manufacturing. That is a problem that we have in the pharmaceutical supply chains widely, and we are no exception for that, unfortunately.
Secondly, so I can’t give you a proper date for the IND. With regards to the uncontinued medical -- unmet medical need. We’ve just done a deep dive into this indication. And we concluded that the unmet medical need is there -- is still there, and we expect it also to continue to be there. Also, because gene therapy might have some additional hurdles in such a lysosomal storage disease as Pompe compared to many of the other diseases.
So the answer is we continue to be interested in Pompe. We’re significantly delayed because of the COVID issues with manufacturing, and we have no data at the moment for an IND, therefore, as these supply chain disruptions are widely spread and COVID is not going back and not retreating, in fact. So it’s difficult to say at the moment. That’s my first question."
Q1 2022
"Simon Scholes
Yes, hello. Thanks for taking my questions. I've just got one. I was just wondering when we might get the next piece of news flow on the Pompe program?
Sijmen de Vries
That's a good question, Simon. I would say that later on in the year, we will definitely. But it will be more towards the end of the year, we will definitely give some more updates on the Pompe program. But again, it is not one of the main value drivers, as you could appreciate, because it's in preclinical, right? That's -- and in this comp of course, that's why."
Q2 2022
"And on the right-hand side, Pompe, we continue to work on a next-generation alpha-glucosidase therapy from our own transgenic platform for the treatment of Pompe, and we're currently engaged in some preclinical studies where we will seek for differentiating features versus the current standard of care that is on the market for Pompe. And as soon as the results will become available, we will update the market on this.
Q3 2022
"Pompe's still a significant unmet medical need. And we believe that our platform may have the potential to have differentiating features versus the existing alpha-glucosidase enzyme replacement therapies. And that is why we are continuing to search for that. And if we find these, we will start developing this compound going forward."
Q4 2022
We are continuing the preclinical investigation of a next-generation alpha-glucosidase therapy for the treatment of Pompe disease, and are currently evaluating potential differentiating features of our product candidate in these preclinical studies. We expect to update the market on our findings in the second quarter of 2023.