The Drug Development Landscape: Trends and Predictions for 2024

Hello and a warm welcome to the latest edition of Conversations in Drug Development, brought to you by the team at Boyds. This podcast is for our fellow community of scientists and clinicians working in the wonderful world of cell and gene therapy and drug development. Thank you for tuning in and we hope you enjoy the conversation.

Hello and welcome back to another installment of Conversations in Drug Development. My name is Harriet Edwards, and I'm one of the regulatory team here at Boyds and your podcast host for today's episode. I am delighted to be joined by Professor Alan Boyd on our podcast today, founder and CEO of Boyds. Alan, welcome. Hopefully this is the first of many appearances on the podcast. Thank you very much, Harriet, and it's a pleasure to be here today and I'm looking forward to

our conversation. Absolutely. And I think it would be remiss if we didn't have the first episode of 2024 looking at all of the things we can expect to come of this year, but also notable trends and highlights from 2023 that might inform us of things to look out for in the drug development space this year. So we're going to kick it off with

a focus on advanced therapies and cell and gene therapies. We know they're in the news a lot at the moment, and I think we'd be remiss if we didn't talk about one of the key highlights of or lowlights, actually, of 2023. And something that continues to be a big topic in drug development. And that's, of course, the financial landscape. So I don't know if there's anything that immediately springs to mind, Alan.

But there was a general downturn last year, and we're not sure whether that's going to continue into this year. If you got any thoughts on that. Well, it's a very important point that you raise, Harriet, because of course, developing medicines and devices is very expensive. And without the right sort of finances and investment, we can't do anything. Now. It's interesting during Covid, clearly industry and the pharmaceutical industry played a very big part in particularly

getting the vaccines there. And so life science companies around the world became the favorite of the stock market and investors. And in 2021, something like $50 billion were invested in life science companies. Wow. That is probably the most money that's ever been put in in one single year in terms of investment. And the following year that dropped down to about 25 billion, which is still huge. Yes, it is,

but in 2023 it halved again. Wow. And I've seen figures from standard and poor where, you know, up till about September, only about, I don't know, something like 15 billion had gone in which was a big reduction. And most of that was in the United States. And that has been a sort of a general increase over maybe the last decade up to the point of the pandemic, like you said, of investment in pharma and

particularly in the biotech space. Do you think it's just we've got used to such a huge amount of investment and things are now normalizing after Covid, or is there something else going on? Well, as you know, I've been in the industry a long time, well over 30 years. So I've seen downturns and recessions. This is probably the fourth one I've experienced during my career. So it is cyclical. Now you ask me, how long is it going to last?

Well, if I could give you a precise date, um, I wouldn't be sitting here now, if you know what I mean. But it has been difficult, particularly during 2023, particularly for seed funding. This, you know, the money that goes into the very early stages of drug development and companies. So that's been a real limit. And in fact, even in our consultancy we've seen clients though they they want us to do work, just haven't got the money.

Now fortunately though, there was there was a big conference in San Francisco last week, the JP Morgan conference. And coming out of that, it's where anybody who's anybody in the pharma industry goes to. Clearly the financial situation is improving. And that's the method it's starting to and in fact, in America, they're even talking now about flotations on the New York Stock Exchange of life science companies, which is a great stimulus then, because once that gets going,

there's extra money there. People make money and reinvest it. So hopefully we're going to see an improvement in 20 some positive news. And I guess we keep our fingers crossed for that.

I think it's notable to to mention that at the back half of last year and particularly the last quarter, there were some positive acquisitions, some positive releases of funding into the the gene therapy space, particularly with I think there was one notable acquisition by AstraZeneca, somebody that we've all heard of, um, purchasing a company called Gray Cell, which was a cell therapy company, autologous and allogeneic developer for a massive $1.2 billion.

So the money is there. I think if, um, if people are willing to put it into something that's potentially a little bit more risky, like the cell therapy development that we see, but maybe it's it's just a risk versus reward thing at the moment. Yeah, I think it is. And the other thing to think of, because of the downturn, the valuation of companies has actually dropped. And so big Pharma can move in and buy them basically at a reduced rate from where they were a year ago. Wow. And we've seen.

Quite a lot of that too now, which is good actually. I mean, companies being taken over. But actually, if you're a major stockholder in the in the small company, you perhaps have not raised it as much money as you thought you might have done. So now, if you are a venture capitalist or somebody with lots of money in your pockets, it's a good time to snap up some bargains in the biotech space. That's right. It's good to go out shopping,

basically. Absolutely. January sales. You heard that here first. If if anybody's got a spare few million or even billion to to spend. But I think there is some competition and that's worth mentioning where big pharma and innovative medicines haven't really had the competition before. There is now competition from things like big tech and AI that sort of swaying the market to invest there as well. So that's maybe a factor with the the slight downturn that we've seen. Yes, certainly.

Again, the money that has flowed in 2023 has made a large amount of it has been into AI companies. So if you had AI in your title, you, you know, stood a better chance of getting investments, let's put it that way. And we could talk about financials for a long time, I'm sure. And everybody has an opinion there. But since you mentioned I, I think it brings us nicely on to maybe the next hot topic or a trend to watch out for in 2024.

And that's novel technology. So I mean, there's there's many novel technologies we can talk about, but I think we first have to mention that 2023, it wasn't all bad news. There might not have been as much money in the the system for drug developers, but it was a landmark year for novel technologies, having our first ever gene edited technology approved, and that was by the UK regulator, the MHRA.

So back in November last year, we had an approval for a Crispr edited technology to treat sickle cell disease, and that followed suit with FDA approving later as well, and even other global territories now following suit as well. So really positive and and maybe something that will open the floodgates for 2024 for these type of technologies. Well,

you've raised a few points there. I mean, first of all, it was great that the MHRA was the first regulatory agency to approve it. And if there are any benefits from Brexit, then doing this sort of thing by the MHRA, Brexit's allowed them to do that to be different, to look at things in a slightly different way and be on the front foot and get these new products approved. So that's certainly happened. And and of course, the FDA themselves approved six new cell and gene

therapy products last year, which. Is huge increase. Yes it is. I mean, from the year before we had a period, I think 2122 when very few got approved. And so that's moving forward. This year, the FDA are saying they're expecting to approve 17 selling gene therapy products, which if they do, it's absolutely amazing. And if I think back, you know, when I started, you know, people often say I'm a pioneer in gene therapy. Perhaps I am.

But, you know, establishing one of the first companies in the early late 90s, early 2000 to think that 20, 25 years later, we now have 34 selling gene therapy products approved by the FDA. And, you know, a similar number here in Europe. So it really has mushroomed, and it's great to see it. When I started out in the late 90s, people said I was crazy. Nobody had ever done it. Why are you doing this after having a huge job in in Zeneca? But perhaps, you know,

I wasn't that crazy after all. And it's really satisfying now to see see what we've achieved actually with these treatments for patients. Oh absolutely. And it makes us very proud, I think, to to be part of the boys team and know that you have that legacy behind you. And to see all of these sort of gene therapies that we have the privilege of working on,

some of them come to fruition. It's exciting for us as a as a team, but also the wider sort of altruistic properties of looking at how well cell and gene therapy could actually do for patients in need. There is the ability to actually cure diseases now, and we have those products approved on the market to cure something rather than just to treat symptoms. It's it's a wonderful time to be part of the drug development space. Yeah. No, I mean it's very exciting.

I've been qualified a long time now, and most of my professional career treating patients and then developing medicines was all about trying to stop symptoms happening. But with gene therapy, actually, and I don't particularly like the word cure, but that's actually what we're doing. We're actually changing and attacking the root cause of the disease. Whereas all the other things that we treat, we all take medicines, don't we? At times we're just treating symptoms, not the root cause.

So that's what we're getting at. And and it's having us, you know, when you see particularly some of the children that have now been. Treated with things like her for the SMN disease, the inherited blindness. This is a life changing treatments not only for the, you know, the patient, but also for the families. Oh, massively. And actually, that's a really nice way to put it, Alan. I'm going to take that moving forward as treating the root

cause rather than a cure. I think that's a really great way to to describe it. And you're right, with these gene therapies that we're seeing, they are treating some really awful diseases. And a lot of them are specifically targeting neurodegenerative diseases. But in children. And to be able to offer that to the the patient population that currently don't have any options is potentially life changing. So hopefully much more of that

in 2024. Yes. So it's interesting if you look at, I've mentioned the number of selling gene therapy products that are approved. If we just take the gene therapy side, two thirds of all the gene therapy studies and that have been conducted across the world, and there are now well over 4000 of them, two thirds of those have focused on oncology. Wow. And cancer treatments. Yet up till now, we've only got two approved gene therapy products to treat cancer. And do you think there's a

reason for that? Is it that the problem with development or is it post development where costs potentially become prohibitive? I think I think what it is when we're treating genetic mutations, that's a very specific target. However, when we treat cancer patients, we're actually using the gene therapy itself to deliver a particular protein, which is then either attacking or supporting the cancer that's there or supporting

the immune response to that cancer. So it's I ought to think, particularly when I'm teaching, when we're using gene therapy to treat cancer, this is really the gene therapy is a sophisticated way of delivering a protein. And I don't think people recognize that. And of course, then we have to make sure we've got the right amount of protein there. And is it, you know, how it's it's acting. And of course, that will be tried to be eliminated

by the immune system as well. I can sort of explain why things have not happened yet, but the work goes on. And do you think there's a potential for in in 2024? And I think beyond this, this will continue the fight against cancer and the use of gene therapies to treat any oncology indication, whether that's for pediatric, specifically a rare disease or something more widespread. There is undoubtedly a focus from the pharmaceutical industry to target oncology, isn't there?

Oh, definitely. But I think where the real successes have come is with the Car T's and the immunotherapies. All the Car T's are aimed predominantly at at oncology diseases. And we do use ex vivo transfection now for things like the sickle cell treatments for immune deficiencies. So we've got the technology. We just have to work it out. And it's as you said we have the technology. I think people now we're starting to get experience in developing

these types of medicines. There's the regulatory framework that's starting to evolve and back up the development of such novel and individual types of medicine as well. It's not one size fits all anymore. And that's, I guess, where I look at things. I know you you look at them from a completely different perspective,

having a medical background. But there are also some barriers, even with all of that knowledge and, and access, that we have access to those medicines, once we've demonstrated safety and efficacy is a little bit tricky as well, particularly for countries that maybe are less developed than, uh, sort of the ones that we would expect to see approvals in the UK, the EU and the US, etc.. Do you think that procurement and pricing of these products, we go to

all of the effort of getting them approved and and working on them, developing them for people in need, and then we can't actually give them to people in need. Do you think that's a problem? Yes, I think it certainly is. I think at the moment I'm being

totally honest here. The selling gene therapies that are being approved are for Western societies, the wealthier nations, because they are expensive, they're expensive to develop and they're expensive to, you know, keep manufacturing and be out there. And they're not really for the masses at the moment.

And I think that's something we need to really address particularly, I mean, let's face it, you know, in places like Africa, we have trouble delivering even the basic medicines to them, let alone these, you know, these very complicated technical delivery systems that are developing. And when you think about some of these delivery systems and they're actually personalized medicines, aren't they. They're made per patient on, on many occasions there might be issues with needing.

To cry out for the the medicine to. To have it in ultra cold temperatures. And like you say, when we're looking at something like a non developed country or continent like Africa that maybe can't sustain that kind of infrastructure, that makes it a real shame that actually the population that need it aren't going to have access to it for some time. No. On the other side, we all have to start somewhere. Yes. And and I think,

you know what we're doing. If you stand back and think we're actually using DNA to treat and to say, you know, attack the root cause of a disease now. And so it's just started. We've only been going sort of 20, 25 years. Seriously. Most treatment modalities that have been developed, you know, Ace inhibitors, the antibody statins took about 20 years from the first ideas to actually making it to be a medicine. That's that's standard.

You know, you can look at all the, you know, the breakthroughs in the last 50 years. So it's early days yet for cell and gene therapy. And I think what we will do, it will evolve. The manufacturer will get cheaper and more straightforward. And so it probably won't happen in my lifetime. But you know, we'll see say, in 30, 40 years, the technologies we have now are much more widespread. But I'd like to think so. And I agree with you.

I think it's something that everybody that's working on these medicines has at the forefront of their mind. They don't want these medicines to be exclusively accessible for people that can afford it. And so I think as long as everybody is conscious of that, there is going to be a move to make things more accessible. As you said, when when the technology and the scalability is there to support lower costs, which is positive.

And I think you said before, we're in the infancy of cell and gene therapy. We've made great strides so far, and hopefully 2024 will be another landmark year of notable approvals and changes and maybe some new regulations and guidance to support such development. But we are still at the very beginning of the journey with silent gene therapy. So overall positive. Yes, certainly.

Well, I'm a believer, you know that. And I'd like to finish this topic on a positive note as well, because there was a general increase at the end of last year in phase three trials for sale and gene therapy, particularly gene therapies. I think it was a 10% increase was reported. So I think that could hopefully mean we're looking at new approvals in in 2024. And as you said, I think there's up to 17 predicted and maybe even

some more to come. That's right. So we've talked about gene therapy and genome editing as one of the novel technologies. But we can't get away from artificial intelligence either. Um, I know we talk about it a lot here. And artificial intelligence within the drug development space can

mean so many different things. Um, for those people that maybe haven't listened in, we do have a specific episode about artificial intelligence, so we talk about it in much more detail there and would encourage you to to listen in to that. But, Alan, do you have any thoughts on trends for the use of artificial intelligence within drug development within salon gene therapy for 2024?

Well, there's quite a few areas. Yeah. And these are already happening. I mean, we if you think about the identification of molecules to treat disease and drug development at that level, drug discovery already and we have used AI for a long time where we take a particular structure, we can search for which bits of that structure have previously known to cause side effects and adverse effects and things. So medicinal chemists can then modify that structure. So that's already there.

The other place we're using AI is in imaging and the reading of images with scans and X-rays and things like that. So that's already there. There are also some companies now using AI in toxicology studies. Yes. Because you'll be aware that thing that takes the time is analyzing and looking at all the slides that we take for the various organs of animals that have been exposed to potential treatments. And all of that at the moment pretty much is manual, isn't it?

So absolutely. And there's thousands and thousands of slides. But now there are ways of looking at, should we say, the slides from the experiment with slides from animals that have not been exposed to the drug. So it's an excellent way of doing that. I know it's in its infancy, but that's happening. Yeah. And the other thing that's happening as well is it's in clinical. We now can use AI to write clinical protocols.

We can use AI to look at the literature to find out, you know, what's the right endpoint to use in a clinical trial as well. So it it is there. I think one thing we have to be careful of is using some of the we say the things like chat PTC yes. Um, and using that for creating. Documents and pulling information from it. And certainly, as you know, Harriet, I've more or less banned the use of that in writing our

regulatory documents. And the importance of that is that we have to be totally accurate with our regulatory documents, and if we don't know the source of the information, then it could we could get in there. And that could create a safety problem for patients. Absolutely.

And we did touch on that in the I, um, episode actually, not just ChatGPT, but many of these platforms that are in their infancy are fantastic in terms of their, the idea of them, but at the moment they are only as good as the source data that's input into them. So the more they are used, the more data is collected, the more accurate,

the more reliable they become. But as you said, for for using things, for writing regulatory documents, for writing clinical protocols based on previous data, etc. actually, it needs to be used with extreme caution because there's only a certain amount of sources that these platforms use, and they may be missing a complete perspective because they haven't got that source data from everywhere.

So fantastic idea. I think it's something that will definitely be used moving forward in drug development. There are so many exciting areas, but it's still even more infant if possible than genome editing and all of the other novel technology so must be used with extreme caution. Yeah, no, I entirely agree with you. And of course, you know, at the moment we haven't got any defined regulations from the regulatory agencies about the use of AI. Yes.

Or rather the correct use or the right use of it. And that will come. I'm sure it will. Yeah. And we're all learning. I think that's the key, isn't it? We're all learning the regulators as well as the developers. And it's as with many of these newer technologies, it's about being collaborative and working together to make sure that that we build framework wise from a regulatory perspective is usable and pragmatic and ensuring safety at the end of the day as well. So it's a it's a tricky task for

regulators. I definitely don't envy the the other side of the fence in terms of other topics and things that we might look out for in 2024. I think we've we've covered the obvious, Alan, but is there anything else that you think is a definite trend to look out for this year? Yes. One area is antibody drug conjugates.

Now, those of you who've been around in industry for a while know we've already been there in the past, probably in the sort of 90s into But from what I'm seeing and being at conferences, talking to people and clients coming to, to to ask for help, there is a definite focus again on antibody drug conjugate product or ADCs, as they're called it sort of moved on from just connecting, say, a chemical entity to an antibody. Now people are even putting genes attached to some of these,

these proteins and things like that. So they get the antibodies. The ADC space is is kind of realizing its true potential. Then I guess absolutely. And there's definitely a resurgence. So I think it's something to watch out. And the companies that are now doing this. So yeah, again technology moves on and you know we're adding genes and other things to these antibodies, which is great. It's amazing.

It's certainly an exciting time to be part of um, of any drug development, but particularly with the innovative therapies that we see often at Boyds. It's um, it's going to be hopefully another exciting year. Alan, it has been wonderful, as always, to to speak to you, but to be able to bring you the podcast and have everybody listening, I think everyone will agree. It's been fascinating and we'll definitely look forward to having you on another

installment before we end today. Are there any other hot topics or points that you would like to to raise for people to look out for in 2024? People who know me know I'm an

optimist. Um, and I think going forward, once the money starts flowing again, I think again we're going to see growth, perhaps not in the first half of the year, but the second half. I think that's where the impact will come again and we'll all get switched on. The money will start flowing, more companies will be financed and we'll be looking at even more products coming forward, which is very exciting because after all, people often say, you know, I'm a businessman.

Well, yes, I am running a business, but I'm a doctor first. And the most important thing to me is to get new medicines out there for patients. That's why we do it. Absolutely. I couldn't agree more. Alan, it has been wonderful to have you on, and I look forward to reviewing some of these trends, maybe at the end of the year as well, to see if we were right or not. Okay. Thanks very much, Harriet.

I've really enjoyed the conversation. Thank you. Thank you for listening to Conversations in Drug Development, the podcast series brought to you by the team at boys. Don't forget to follow us on the usual podcast platforms or visit our website to ensure you don't miss out on any of our future episodes.