Grumpy-XL schreef op 19 november 2021 07:22:
Daniel Levine: Well, what’s not about leniolisib from studies that have been performed to date?
Anurag Relan: So, Novartis conducted a two-part phase 2/3 study, using leniolisib. Part 1 was an open label dose escalation study to assess the safety, tolerability, PKPD, in six patients with APDS and those results have been published in the journal Blood. Those results were then used to determine the optimal dose in the double-blind placebo-controlled part of the study, Part 2, with an additional approximately 30 patients. This study, which is still wrapping up, is designed to assess the safety as well as efficacy of the drug. The primary endpoints of the study are looking at changes in the B-cell profile specifically, trying to determine how many naïve, out of total B cells, are developing, and secondly to look at lymphoproliferation. So, looking at the size of the lymph nodes and how those change over time, under either placebo or with the drug. Part 2 of the study completed enrollment in June of this year and we expect that the results from that study will be available early in 2022. In addition, there is an ongoing open-label extension study to allow patients who were in that part to study to roll over to receive leniolisib on a longer-term basis to collect additional safety and efficacy data.
Daniel Levine: What’s the path forward? What would it take to get to a regulatory filing for approval?
Anurag Relan: The goal is first to review the data that we receive in the beginning of 2022 and if all goes well, to file both with FDA and EMA in 2022, and hopefully even have a regulatory approval, before the end of 2022.
Daniel Levine: Pharming’s not alone in looking at PI3K inhibitors to treat immune disorders. Are you looking at all beyond APDS?
Anurag Relan: We are and we’re going to look at this very carefully in a scientifically driven manner, really looking at what the unmet needs are in various conditions. I think our focus right now is around APDS and getting the results analyzed and discussing the path forward with FDA and EMA, as well as other regulatory agencies across the world to see if a leniolisib would be a suitable treatment for APDS, but we are definitely going to be looking at other immune disorders where there may be an opportunity to use a drug such as the leniolisib.
Daniel Levine: And how does leniolisib figure into Pharming’s broader pipeline?
Anurag Relan: Pharming has been focused on the field of immunology for quite a number of years, decades. In fact, we have, one product on the market called Ruconest. It’s a recombinant human C1 inhibitor for the treatment of acute attacks of hereditary angioedema, which is another rare disease involving the complement and contact cascades of the immune system. In addition to that, we have a new partnership with Orchard Therapeutics to develop a gene therapy for HAE. Really the work that we’re doing now with APDS, and leniolisib is an extension and focus of our work in the field of immunology. So, I think the work with leniolisib fits in quite nicely with this pipeline.
Daniel Levine: Anurag Relan, chief medical officer of Pharming. Anurag, thanks for your time today.
Anurag Relan: Really appreciate the time Dan and speaking to you about APDS. Thank you.