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BCRX JPM January 13th Webcast

BCRX JP Morgan Health Care Webcast

Biocryst had a webcast call at JP Morgan’s conference on January 13th. Below is a transcript of the call.

At an HAE patient summit, after that presentation, my colleagues from Biocryst and I were swarmed by the participants, and our tiny little table outside outside the ballroom.

For me, it culminated in a conversation with a patient who made a point to find me. And tell me her story. She tells me she recently lost her son to a laryngeal HAE attack. She shares her son’s passion for life, but also shares how his life was limited because of the burden of treatment. She says he always asked Mom, why isn’t there a pill for HAE. And she looks me dead in the eye, and says, It’s too late for our son, but not for others go faster, patients are waiting. This is when I realized this isn’t some small convenience improvement. This is big.

I got back to my office, I typed it out and taped it on my computer screen as a daily reminder, and a reminder of who we were working for. To my surprise, this quote has truly taken on a life of its own and become a driving force in our company. And I guess I shouldn’t be surprised. It’s not a slogan. It’s a quote. And it has real meaning for our employees. And the result is we’re going fast. Seven years from discovery to FDA approval when it takes on average 10 is evidence, we’ve been going fast. But now we’re going faster. And this is big. We are often running to make sure that patients who want are the day Oh, in the US get access to it. And while we’re only a month in and the launch is going well. We look forward to giving you updates on our launch progress throughout the year.

Next up patients in Japan where we expect to get approval this month. This is really fast as we will be the first and only prophylactic treatment available to patients in Japan. Think about it. Our partner Tory pharmaceuticals is able to offer patients something more than treating attacks in a once daily world capsule.

This is big.

And then, in early q2, we expect approval for patients in Europe. Again, a different market where most patients treat attacks rather than take medicine to prevent. Our European team is ready to launch to offer patients a once daily oral treatment option for preventing attacks. This is three simultaneous launches of a product. With global peak sales potential of north of a half a billion dollars is definitely big. This alone could be enough. But there’s more to the story.

We took advantage of all that we learned through our ha experience and are applying it to other rare diseases to go fast for more patients. Our pipeline is full of what we see as an entire molecule, or entire pipeline and one molecule would be cx 9930, our oral faculty inhibitor to complement mediated diseases. There is strong scientific evidence why factor D plays a role in a number of complement mediated diseases. And we have recent evidence that 9930 works with the proof of concept data we presented last year in PNAS patients. Now our goal is to go fast for these patients by dancing 9932, pivotal studies and P and H and proof of concept and other indications this year.

We also have early evidence that this is big and complement mediated diseases to with market research in teenage patients telling us the same story we heard with HAE patients. They want an oral treatment. And it’s understandable given the burden of injectable therapy for all patients suffering from rare diseases. But we have the opportunity to do even more for these patients by helping teenage patients who have extravascular hemolysis and are not adequately controlled with their current injectable therapy or patients with nephritis diseases that have no treatment at all.

Advancing an oral factor D inhibitor that’s big.

And behind 9930 we have bc x 9254 FLP. This is a horrible disease with no approved treatments. This is a disease that affects children. And the only sign that they have it early in life is their big toes point going inward. Then when they reach the toddler stage, they begin to have soft tissue inflammation that turns into bone. And these adorable children start to have bone growth through their bodies, and joint freezing that can make them wheelchair bound by the time they reach their teens. It would be extraordinary to bring these patients to treatment. And even more extraordinary, if it’s oral. This is a really tough target. And there it was once a very crowded and competitive space. But the need for therapy still exists. And we have evidence from our phase one that we may have a drug. And while there is much more to do, we need to keep going because these patients are waiting.

I shared with you our strategy, and evidence that we’re changing. Now let’s take a look at how we got here. How this transformation came about. It’s much more than a strategy of oral drugs for patients with rare disease. As I said at the beginning, what makes it extraordinary is turning the strategy into reality.

It starts with our drug discovery team, a team that builds these enzyme blockers, one atom at a time. So you not only have a potent inhibitor, but it’s also specific to the target. These targets are part of large enzyme families like proteases and kinases, getting them to bind to the target and not to others in the family. Now that’s extraordinary.Next, it takes a seasoned regulatory and development team that strives to do it better than the rest. A team that can design studies investigators contribute to and believe in a team that finds patients to enroll, which is always a challenge in rare disease studies. By taking care with investigators, their staff, and patient advocacy groups, were becoming the sponsor of choice. Because we are viewed differently. A team that has demonstrated that they can enroll pivotal studies and file in major territory simultaneously. And doing it with high quality and the need to go fast. That’s extraordinary. And finally, you need capital. Yes, we’ll be generating revenue from sales in Orlando this year. And that’s very exciting. But we also completed a financing that provides the capital to focus on executing our plan, and not the next milestone to do the next point is having investors like royalty pharma, in theory, providing access to $325 million in capital, we believe is evidence that this is a very good investment. And even more evidence that this isI’m not one to adequately or accurately predict the future. But I hope you agree that our company is transforming. And this is big.

So what’s next? This year, the successful launch of our first oral drug for patients with a rare disease and advance our pipeline into multiple indications is what’s being the evidence of this will emerge over the course of the year. So until then, consider this. We invested years ago in drug supply and months ago in hiring the sales force to go fast. And the evidence is we were calling on customers less than 24 hours after approval and started shipping product less than two weeks later. That’s fast. This is not an ordinary time. And this is not an ordinary company. But at the end of the day, we are making extraordinary efforts to create the ordinary and deliver it to those who never thought it was possible. We look forward to updating you on our continued progress of approvals launches, and advancing our pipeline to the next big oral therapy for patients suffering from rare diseases. And our goal is not to only be able to say this is big, but this is bigger.

Thank you.


I’d like to invite everybody back for the q&a portion and Jess.

Great. Thanks, john. And just while the rest of the team is coming back online here, just to remind folks that if you want to ask a question on the portal, the ask question button on your screen, and that’ll send them to me.

But maybe first question, you know, I know it’s super early in the launch of Orlando. But how is this progressing relative to your expectations? And how has the engagement with positions been so far? You know, Charlie, you want to take yours off news? And incident one? Yeah. Hey, thanks, Jess. So as you heard john, say, we made investments to get out of the gates quickly, we were promoting the morning of December 4, and we’ve been really pleased with the excitement and enthusiasm we’re seeing both from physicians and from patients. So we’ve had numerous national promotional events for physicians and patients that have been really well attended. And, you know, we’re just we’re pleased with how things are going so far.

Maybe sticking with the launch? How did your thinking and plans for the launch evolved with the endemic and how you plan to efficiently market the product in a virtual setting?

Yeah, so that’s, that’s something we’ve been planning for, all the way along in 2020. You know, we, we figured we’d be launching into a COVID environment. And so we got plenty of practice up front of john mentioned, we had our Salesforce come in early last summer. And so many of them already had experience promoting in a COVID environment before they came to biocryst. And then we’re out there, working with customers, in a virtual sense, ever since then, in some places in the country, we’re able to reach people in person and others, it’s virtual. But the excitement around the world really opens real doors and virtual doors. And so we’re getting access. And we’re, we’re, we’re pleased with the access that we’re getting the conversations we’re having. Just like, I think I’d add, there’s been quite a bit of pent up demand, I think, for an oral drug in this space, and that, that doesn’t get affected by COVID. Right. People have been waiting for years for this drug. And so you know, patients are reaching out to their doctors, doctors are reaching out to their patients. And so we’re pleased with how things are progressing at this early stage.

Okay. Also, what caused the shift in timing for the Orlando approval decision in Japan to move into January from December? Megan, you want to take them?

Sure, john, thanks. I just flew in December in Japan, the government had adopted a decision to implement a drug repricing and an off year and in 2021, and that’ll be April of this year. So associated with that decision, they essentially shifted the approval that would have happened in December into the January timeframe. But I would I would know Orla dado did your an important step with a review panel, which essentially recommended or Ladell for approval in Japan to the MHL W. And that occurred back in November.

Okay, got it.

Let’s take a couple questions from the portal here. Thanks for sending those in.

How are you planning to reach other markets outside of the US and Europe? I think we think we know how you’re planning to proceed in Japan. But maybe you can elaborate on that one.

Up, Charlie. So the plan is that we’ve got three filings right now, one for the US, one for Europe and one for Japan. And so our intent is to take that around the world, and to be launching it, you know, we’re patients around the world. And the prioritization will be filtered by these criteria. And you know, the first one is, we’re not going to generate any new data. So being able to take the data that we have, and hopefully one of the filings that we have largely one of the filings that we have. The second is, you know, is there a market there? And you know, what, what effects does it have on pricing? And then third is, how do we do it? Is it clear that we could use a distributor or a partner or is it a geography that we could do on our own? And so again, plan is, you know, because every country matters here, right, as we continue to layer on more and more and more, and so we’re really excited. And Charlie’s team is already on their way.

On the first wave, host Europe.

Okay, great.

What do you see as the key sales opportunity for Orlando worldwide as the breakdown between the US and other regions. Surely you want to take that?

Yeah, we see the peak sales being north of $500 million worldwide. And, you know, typically us is going to be the biggest market as the most advanced and highest price. for existing therapies out there. It’s usually around 80, or 90%. But the percentage of global revenue is gonna be really meaningful, as john just described, and we look forward to taking this drug around the world. And as investors try to track this launch, when do you anticipate guiding the street on sales? And what launch metrics Do you anticipate being able to provide?

Yeah, I’ll take this one again, Charlie. So a couple of things. First off, you know, remember that December 3 was the producer day, and there was a holiday in there. And it takes some time, as Charlie is mentioned around getting reimbursement. And so the month of December, you should really expect next to nothing. The first quarter, though, will be our first full quarter sales and the way we’re thinking about, we’re not going to provide guidance. But but we will provide sales when we report out the quarter. And if the sales are very clear, and we’re performing at or above expectations, then unlikely we’ll provide more if if it needs some clarity, then we’ll add more information to help provide that clarity.

Can you talk about how quickly you expect to secure reimbursement, really just walk through your thoughts on the timing of their decisions as well as the expected payer mix?

Sure, yeah. So that’s something we’re really focused on, particularly this quarter, our market access team is out there actively engaging with payers, some of that is is just the blocking and tackling, you have to do make sure they have their NDC codes loaded, etc. But then we’re trying to make sure that they understand the value proposition and when payers look at the the proposition for roadeo. That what they’ve always told us and it continues to be true, they say, you know, this is a product that they see reversing. So we expect a lot of progress there. This quarter.

What about the potential mix? Oh, sorry, the mix. So looking at claims data in the past, it’s about 70% commercial amongst reimburse patients and you know, early days, we think the same will apply to our our paramedics.

And we go back to the portal questions here.

When is the next factor D update? And what specific data will we see how will the data be shown? below, you want to take that to

the next slide or update will be in this quarter in first quarter of 2021.

dose ranging study is fully enrolled now with 16 subjects, the majority of those have no past background of being treated with C five inhibitors. And we’re using non 3d as monotherapy. And we have some patients who have inadequate response to C five inhibitors as well. So you can expect that we’ll put out quite a bit of information from the study. haven’t decided exactly what forum yet, but in the first quarter, it will be more than a press release. It will be a detailed run through bouncing around here a little bit.

Can you maybe just remind us where you stand with Galidesivir?

Sure. So we finished part one, we put out the data, the tail end of last year. Remember, it was the dose raging part of the study and you know, it was nice to see that the drug appeared safe and generally well tolerated. And there was some activity on the the virus, the COVID virus and then we also said that there was some animal work done in in hamsters that also showed activity if gala disappear against the COVID virus. But given where we are with the pandemic, the fact that the vaccines are rolling out, and there are a number of other therapeutic options niad clear late communicated to us that they prefer that we go after a different virus in order to get this ultimately stockpiled into the SNS. And so we’re pursuing Marburg and, you know, I think the data that we got on the clinical side will be valuable in this program. And we still strongly believe that there’s no need for multiple broad spectrum antivirals to protect us for the next outbreak that occurs.

Okay, great. I’m going back to Orla, do you expect initial uptake to be driven by patients new to therapy, the switching from parental alternatives or some combination?

Surely, yeah, so it’s early days in the launch. But let me tell you what we saw in our Apex s long term safety program, because I think that’s indicative of what we expect in the marketplace. So what since we opened up Apex bass in the US, in the spring of 2019 50% of the patients enrolling have been coming off of an injectable prophy therapy, and then the other 50% are stepping up. Previously, they were on a cube only, but they’re stepping up to pro feet when they see that there’s there’s an option to do it with an oral one scale. And so that that’s consistent with all of our market research with physicians and patients. And so we expect about, you know, half coming switches from prophy, and half people stepping up to do pro p for the first time within a while.

Okay, great. Another one here, with two larger pharma companies developing factor B and vector d, how can your drug differentiate from others? Let me start building and then maybe you can provide some more color.

Big companies in rare diseases, not an advantage. These are not arms races where the more you spend, you know, the better you do. This is why we think there’s a lot of applicability to what we learned in HAE both in the launch we’re currently going through but also in the development of Orladeyo, and the filing and approval. So we’re going against Takeda and CSL and ha, those aren’t small companies. And so, you know, we’re not we’re not worried about competing in these rare disease spaces. And we think it’s a, you know, a core to our strategy. But bill, I don’t know if there’s anything else you want to say about? Sure. No, I think that targeting either affected D or effective D makes perfect sense. At the end of the day, it’s going to be the benefit, risk profile of each new medicine that gets evaluated by regulators and label as a result of all of the work we do. So the fundamental characteristics of our drug that we know that so far, I’m really pleased with it has linear and does proportional PK, psyche profiles. Excellent. We’ve learned those chronically for over 40 weeks in some of the subjects on our dose ranging study and pnh. And the enthusiasm from the principal investigators have the ability to fully recruit that study in the face of a worldwide outbreak and COVID, I think, speaks to the recognition by pnh hematologist that blocking the alternative pathway is a great idea. So very promising. He just want one other thing I would add that I think is an advantage for smaller companies is we do things differently than than big companies, the amount of attention that we give investigators, advocacy groups, the importance that we place on that at all levels in the company. I mean, Bill and I and Charlie and Megan and others travel all around the world, meeting with investigators meeting with patient groups. We haven’t been able to do that with with COVID. But we hope to get back to that when when we’re all vaccinated. But that that I really want to stress to investors that that kind of care, and approach helps you enroll studies faster, helps you build relationships that get you advocacy, and support and it makes a difference for sure.

Okay, great. Um, can you talk about the next steps for development of 9930? How many studies do you need to support a filing in teenage or would you pursue treating patients sci fi poor responders or both? And what would the endpoints look like in our registrational trial?

Design of those studies that begin points and sample sizes and so on, when we start the study, so there’s no need for us to advertise to our competitors in advance. But in general, I think that our goal is monotherapy, abroad label and superiority to see five inhibitors. And one of my goals is to make the sci fi inhibitors obsolete in plh, by introducing monotherapy with PC x 9930. You know, the precedents are there. There have been several controlled studies and plh. And we’ve had great discussions with regulators dose ranging study, we’ll determine the dose based on the evidence from that study and PK PD modeling. And then we’ll finalize the designs and get going. So I think that likely will need more than one study. But, you know, this is a rare disease studies a small, highly efficient, parents not difficult to come by. And, you know, we look forward to really get moving on their program this year. And I’d add to Bill’s comment that I made a statement in the prepared remarks beforehand, around market research we’ve done with teenage patients Charlie’s started. And the story is the exact same as what we saw on AJ, these people want something more than an injectable therapy, there’s a real burden. I mean, don’t get me wrong, they’re super grateful to Alexion for having a treatment when they had nothing. But they want more. And it’s completely understandable that an oral option would be attracted.

Okay, so if you can’t kind of provide the you know, development plan just yet. Can you lay out the timelines for future updates beyond the data, detailed data you’re talking about in the first quarter?

Sure. So beyond the first quarter, this year, starting pivotal studies in Pnh, also this year, starting a proof of concept study and selected the fractus indications. So the scientific evidence that validates dysregulation of the alternative pathway in those diseases is also very strong. So it’s very attractive. There are no approved therapies in those illnesses. medical need is huge. And they’re serious and life threatening diseases. So that is exciting, too. We had great discussions with nephrologists last year. And we are proposing what to do with regulators. And once we have the feedback, again, that’ll start so this year, you can expect to hear about a pivotal studies, mph proof of concept study in the Fridays and probably later in the year, an update on the evolving long term data coming from the dose ranging study. So we already have patience beyond 40 weeks, and later in the year, it’s going to be a really nice body of evidence, and probably will be able to withdraw the C five inhibitor, you know, as time goes by and the machine.

Yeah, I just add, I mean, think about it for a minute. We just got approval of our first oral drug for a rare disease in December, and we’re launching it and we’re about to enter into pivotal studies in the next Rare Disease The following year, that that that’s pretty impressive. That’s going fast.

Okay, great. Another question on the portal? How are you thinking about the son or Olivia? How are you thinking about the magnitude of pent up demand, relative to the pent up demand for the prior prophylactic therapies?

We see a lot of pent up demand. So it’s, you know, the it’s been great for the HA patients. And if you think back to 12 years ago, when they pretty much had nothing.

But and then there were seven injectable drugs launched since then. So that that’s great. But what you know, as john talked about earlier, what we’re all talking about here is there’s such a demand for oral therapy because with those injectables, there’s a treatment burden that’s associated with it. And it’s not just, you know, I’m afraid of needles, but there is a needle fatigue, there’s preparation, storage of the products. How do I travel with these products, all of that creates a burden in people’s lives. And an oral once daily is is what they’ve always wanted. You heard the crowd applauding in John’s audio earlier. There’s, you know, amongst people who are doing well on pro products, as well as those who’ve been afraid to step up to profi there’s pent up demand in both those places. Yeah, and just if the question is, you know, should people use the

Is injectable appropriate therapies as as markers, I wouldn’t do that because there’s differences. One difference is we don’t know how many people they transferred from clinical trials on to commercial therapy compared to what we’re doing. And the other is that they use multiple specialty pharmacies to distribute which there’s some built up of inventory, where we’re using one. So when we report out sales, you’re going to know exactly what we sold rather than any kind of inventory buildup.

New, we can move to 9250 for a bogey. What were the key learnings from the recently announced phase one results? And how do you plan to advance that asset for fop?

Sure, so Jason, basics 3250 is a kinase inhibitor. And the very first thing we want to understand, you know, first in human study in healthy subjects, is the safety profile. So we’re really, really pleased with the safety profile, we start with 1950. In essence, there’s nothing that not only is there no dose related findings, but there are basically no findings that lead you to believe there’s any safety defect because drug. The second important outcome is the PK profile, and the exposure of the drug in healthy subjects. And as we increase the dose, we get mimiron dose proportional increase in exposure, that’s always a good thing to see. And finally, at the 20 milligram dose, we had exposures that were similar to the drug exposure in rodent models of abnormal bone formation, heterotopic ossification, which is the hallmark of this op. So all in all, it was a very successful experiment.

kinase inhibitors can be difficult to discover and develop. And we couldn’t be more pleased, the next steps to make more drug and conduct longer term toxicology studies. So that’ll be what your point is this year.

Yeah, I would add to what Bill said that we didn’t get many questions about FLP RL to inhibitor until we put out this data. And now that people realize, you know, we’ve got an out to inhibitor that’s got really nice pk. And so far, it looks like a really good safety and tolerability profile. And then the competitive landscape dramatically changing that we’ve got shot. And as I said, in the presentation, so horrible disease. And so to bring forward a therapy, and it’s a really tough target, as evidenced by how difficult it’s been to find a therapy that we think we got a real shot.

Can you maybe elaborate a little bit on the competitive landscape, the other assets and development for fop, and how many 250 is differentiated?

Will you want to tackle that? Sure. No, it’s been sad to see that Halliburton has had problems with premature closure of bone growth plates. So that that makes it very difficult to use that drug to the children when the target is in this disease and children.

Similarly, it’s been sad to see the activity antibody program essentially come to a halt because of this in a clinical trial, that there are other companies, kinase inhibitors, they’re all early stage, we haven’t really seen any data in any fop patients yet with any of those compounds.

So I think that, you know, this sort of teaches you that until, until there’s a successful therapy, you should keep going.

Okay, great.

For me, the last thing, just financial question. However, the recent $345 million of royalty in credit financing deals, affected your cash runway, and are those proceeds enough to get you to profitability, and are the two initial transactions totaling 75 million factored into that calculation. So you want to just happen been, the financing has been transformational to John’s point, allowing the company the opportunity to focus on delivering and executing on our strategy is a really big deal for us.

You know, in terms of what it does for our runway, so as of q3 on a pro forma basis, it puts us over $350 million in cash, based on the guidance that we’ve given previously, that would get us at eight To 2023. Now, obviously, this year, we’ll be generating revenue. And we’ll also be investing in moving quickly along from a development perspective.

So I would expect that guidance not to change right now the variables will change in terms of the revenue and the investment, but I would still expect this to get into 2023.

Our focus is not necessarily on, you know, how quickly it can be invested in these products.

And then in terms of the 75 million, so the big thing about the 75 million is that a, it’s on our option. So if we, if we need it based on revenue milestones that we need to achieve, that we can draw on it, if we don’t need it at that point, you know, we wouldn’t have to take it down.

You know, those, we factor it in again, it would be based on those variables, basis, more than anything else based on revenue and how we achieve revenue targets going forward.

But the option to have the option to drop down at our own discretion is a big deal and ensure that we have flexibility on a go forward basis.

And I’d add, it wasn’t that long ago that the only source of additional capital we had was our shares. And when we put out data investors would wait for the next financing, we’re not in that spot. Right, we’re in a very, very different spot with a number of different Well, first off a very nice balance sheet and then a number of different levers to pull. And just from operating the company puts us in a different mindset, right? It’s about focus on launching movement. As you know, one of the first questions moving into other countries successfully launching, they’re advancing the pipeline advancing, you know, fop and 9930, and it’s just different than worried about the next data of the next financing.

Great. Well, with that we are out of time. So thanks, everyone, for tuning in. And thanks to the buyer, Christine. Yeah, thanks, Jess.

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