Q&A: Newly-minted CSL chief executive Paul McKenzie and chief medical officer Bill Mezzanotte

29 Mar 2023
endpts.com
Phase 3Executive ChangeVaccineClinical ResultGene Therapy
Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault. With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript. Endpoints: You took over this month as CEO from long-time CSL veteran Paul Perreault — in what ways are you going to move the company into new areas, and what have you learned from what Mr. Perreault as he led CSL over the last decade? McKenzie : It’s a real privilege to be able to be given this opportunity to work with this fantastic management team. That dedication and promise we have to patients and from my first interaction with CSL, that was critically important. From Paul, that passion, he knows some of the patients in hemophilia by first name, and that’s something that won’t change. What I also learned was that as the organization grows larger, is to really focus. It’s easy to do lots of things, and harder to do a few things very well. As a management team we spent a lot of time, as we came and dealt with Covid, on focus and making sure everyone in the organization understands where we need to be and where we need to put our efforts. That doesn’t mean ideas aren’t welcome, but not every idea can be prosecuted on a given day. Right now we’re focused on our journey to sustainable and profitable growth and that requires us to reestablish firmly our leadership in the plasma market, requires us to continue to drive our R&D portfolio and interest and desire in differentiating. It’s not like we have a big change in strategy. As a management team we have great continuity; we’re firmly rooted in our CSL 2030 strategy, and a year or two out, we’ll start working on our 2035 and 2040. Endpoints : You recently won approval for the world’s first gene therapy for hemophilia B — Hemgenix. How competitive is this space going to be in the near term? And when you look at the data Pfizer released in December, is there a concern that this market isn’t big enough for two $3 million-plus gene therapies? McKenzie : What we know is our data and our data look fantastic. We’re an AAV5 platform out of the partnership with uniQure, and continue to see good durability. We set a high bar. AAV5 is unique in our ability to dose even though people may have antibodies prior to AAV5, which is very differentiated from the AAV8 or AAV9 historic platforms. We’re very bullish in our ability to make a real difference for patients that want to have a solution other than the standard of care, [CSL’s] Idelvion. Mezzanotte : Every area we’re in has healthy competition and Pfizer has great scientists, but we’re quite happy with the molecule we have, the data we’ve generated and of course the commercial experience we have. Endpoints : Can you say anything about the uptake or interest in Hemgenix since its approval? Mezzanotte : There’s substantial interest and we’ve already had physicians in the US write prescriptions. However, this is gene therapy, not a pill, so there’s work with the payers to ensure reimbursement and that the sites are going through the process right so the dose is administered correctly. Obviously you don’t want to mess up the dosage of an expensive therapy. We expect very soon to be able to report that patients have been dosed. But I can tell you around the world there’s a lot of interest. I was just in Japan, and I was interviewed there about Hemgenix. Endpoints : How have payers reacted to the list price of $3.5 million, and has it been a barrier to access? McKenzie : In the US, the ICER report really looked at the overall burden of disease and showed this to be a very effective treatment. In the US, we’ve had good rapport, and good discussions relative to reimbursement. Not a lot of differentiation of the model. More of, “Hey, is it the right patient, can they be successful?” And in the other parts of the world, there’ll be different business models that likely will have to be implemented to ensure this is a durable product for patients. We’ll listen and be attentive to what the different countries we need, but I think in the US, the discussions have been in the right direction and firmly based on the ICER data. Mezzanotte : I was just at an R&D meeting and the general sentiment was we want to figure out a way to pay for these therapies. These are the future. I think they’re playing with a variety of models on how to do that, but I sat in on some meetings with payers and there really isn’t pushback on the drug, and there are questions on pricing, but we’re having good discussions and because of our history in hemophilia, we have relationships with them. Endpoints : Looking at your Phase III pipeline, what assets are you most excited about and going to be seeing readouts in the nearest future? McKenzie : I shook up my colleagues by saying at our investor day that we had a number in there that I thought could be standard of care in the future if they have the data we hope. Obviously the number one coming out is garadacimab, an anti-FXIIa monoclonal antibody, which showed great Phase III efficacy and tolerability data, and we’ll be filing that this year as a potential treatment of hereditary angioedema (HAE) by subcutaneous administration. We’re still excited about CSL112, our apoA-I product for patients who have suffered a heart attack; those data will be coming out next year. And one we haven’t talked about as much but I remain excited about, and we’ll be seeing data at some point this year or maybe early next, is [CSL964,] an alpha-1 antitrypsin, which is being tested in both prevention and treatment of graft versus host disease. From the Vifor side, very soon, by the first half of this year we should have data on SNF472, which actually came from a little company in Spain. This is a quite impactful drug if it works. Mezzanotte : On flu, aQIVc (Adjuvanted Cell Culture Influenza Vaccine) takes the best of our cell technology, Flucelvax, with our adjuvant [MF59] to bring the next standard of care in flu. That will start in Phase III clinical trials in this Northern Hemisphere campaign and we really think that will be the next bar that people will compare to non-egg-based adjuvanted product offering, which continues our differentiation journey. And then beyond that is the differentiation built around self-amplifying mRNA.
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