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Edited Transcript of BCRX earnings conference call or presentation 6-Aug-19 12:30pm GMT

Q2 2019 BioCryst Pharmaceuticals Inc Earnings Call

BIRMINGHAM Aug 13, 2019 (Thomson StreetEvents) -- Edited Transcript of BioCryst Pharmaceuticals Inc earnings conference call or presentation Tuesday, August 6, 2019 at 12:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* John D. Bluth

BioCryst Pharmaceuticals, Inc. - SVP of IR & Corporate Communications

* Jon P. Stonehouse

BioCryst Pharmaceuticals, Inc. - CEO, President & Director

* Megan Sniecinski

BioCryst Pharmaceuticals, Inc. - Chief Business Officer

* Thomas R. Staab

BioCryst Pharmaceuticals, Inc. - CFO, Principal Accounting Officer, Senior VP & Treasurer

* William P. Sheridan

BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP

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Conference Call Participants

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* Daniel G. Wolle

JP Morgan Chase & Co, Research Division - Analyst

* Gilbert Roland Kinsey

RBC Capital Markets, LLC, Research Division - Senior Associate

* Liisa Ann Bayko

JMP Securities LLC, Research Division - MD and Senior Research Analyst

* Peter B. Kim

Barclays Bank PLC, Research Division - Analyst

* Serge D. Belanger

Needham & Company, LLC, Research Division - Senior Analyst

* Swapnil Malekar

Jefferies LLC, Research Division - Analyst

* Tazeen Ahmad

BofA Merrill Lynch, Research Division - VP

* Tyler Martin Van Buren

Piper Jaffray Companies, Research Division - Principal & Senior Biotech Analyst

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Presentation

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Operator [1]

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Good day, ladies and gentlemen, and welcome to the BioCryst's Second Quarter 2019 Earnings Call. (Operator Instructions) As a reminder, today's conference is being recorded. I would now like to turn the call over to John Bluth at BioCryst. Sir, you may begin.

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John D. Bluth, BioCryst Pharmaceuticals, Inc. - SVP of IR & Corporate Communications [2]

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Thank you, Sydney. Good morning and welcome to BioCryst Second Quarter 2019 Corporate Update and Financial Results Conference Call. Today's press release is available on our website. Participating with me today are CEO, Jon Stonehouse; Chief Medical Officer, Dr. Bill Sheridan; CFO, Tom Staab; and Chief Business Officer, Megan Sniecinski. Following our remarks, we will answer your questions.

Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited and forward-looking financial information as well as the company's future performance and/or achievements. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results, performance or achievements to be materially different from any future results or performance expressed or implied in this presentation. You should not place undue reliance on these forward-looking statements. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website.

I'd now like to turn the call over to Jon Stonehouse.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [3]

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Thank you, John, and thank you all for joining us this morning. We're excited to update you on our progress since announcing our positive Phase III results for our HAE prophylaxis program and share some important advancements in our pipeline.

Since May, we've been engaging directly with treating physicians and HAE patients to gather important insights to build out our commercialization strategy. The demand and excitement they are expressing for our once-daily oral prophylactic therapy, BCX7353 has been resounding and consistent. 10 days ago, we had the privilege of attending the HAEA Patient Summit in Atlanta, which gathered over 1,000 attendees from the HAE community. Over the course of the summit weekend, we had one-on-one interactions with hundreds of patients. So what did we learn? Many patients are doing better preventing or treating their attacks, but they want more. Patients are tired of sticking themselves with needles. During the conference, patients were pulling BioCryst team members aside to remind us, they're waiting for our oral therapy, and we need to go fast. Some are experiencing challenges with access. It causes stress and anxiety for these patients, which is a known trigger for HAE attacks. Access is an important topic we are in the process of planning for and we will be prepared when we launch, so that we are able to knock down as many of these barriers as we can. There is a strong desire for our products profile and the benefits it can provide. An oral drug that reduces attacks and brings the convenience and a lifestyle patients want. Patients told us they're excited. Their wait for a once-daily oral prophylactic therapy is nearly over, and they want our product.

So what have we concluded? We have an amazing drug, and they want it approved. This is a direct quote from a mother of a teenager in our clinical trial. They want more than they have now with injectables. Being controlled is not enough for many. While there are new treatment options available to them, patients tell us they are still having breakthrough attacks. No drug is perfect. With the finish line now in sight, the excitement building among patients is amazing. The enthusiasm for 7353 is not confined to the U.S. Representatives from outside the U.S. attended as well, and their response was the same. No real prophylactic market exists outside the U.S., and they want to build it with 7353, an oral therapy, in Europe, Japan, Latin America and across Asia Pacific.

This global commercial opportunity is one of the many reasons we're happy to add Megan Sniecinski, our Chief Business Officer, to our leadership team. In her leadership role at PTC Therapeutics, Megan helped drive significant global revenue for multiple products through a mix of their own commercialization and partnerships. And she will add tremendous value to BioCryst as we evaluate and then execute a similar strategy.

Our patient and physician interactions, including our ongoing market research, are aligning. Overall, 7353 represents a significant market opportunity, and we are actively preparing for the launch next year.

So what's next? We're in the process of completing patient, physician and payer market research to refine our go-to-market strategy. Bill is making great strides in filling out his medical affairs team, that's holding regional advisory boards to build physician relationships and gather their feedback much more broadly than we've been able to do in the past. We are also developing the U.S. and ex U.S. commercial launch and resource plan, and are filling critical roles to support a successful launch. We look forward to sharing more on our commercial launch strategy with you this fall.

Let me now move to our oral Factor D program, which is another exciting priority for us. We are just months away from reporting clinical data from our Phase I trial for our oral Factor D inhibitor, BCX9930, which represents an even larger commercial opportunity for our company. The current market for complement-mediated disease therapy is $4 billion, double that of HAE and could double again as treatments and development target more indications. The well-established development path and widely available and accepted biomarkers enable rapid advancement of 9930 and allow us to show meaningful clinical differences in a small number of patients over a short period of time. The time line to show progress is very attractive with this program. Bill will go into more detail here, but the takeaway is that you get to a much faster answer to understand if you have a drug, and we will get an important answer soon when we report out the PK and PD results of the Phase I.

Following our Phase I readout, we will move to a proof-of-concept trial in a small number of PNH patients. This patient data will provide further proof and set us on a path to bring another valuable oral drug to many patients with rare disease.

And finally, another important piece to the puzzle is the capital to fund all of this. The good news is we have roughly $100 million on the balance sheet and a number of different options to bring in more capital. And I'd like to turn the call over to Tom to discuss this in more detail. Tom?

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Thomas R. Staab, BioCryst Pharmaceuticals, Inc. - CFO, Principal Accounting Officer, Senior VP & Treasurer [4]

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Thank you, John. With the upcoming commercial launch of 7353 and the predictive clinical data coming from our oral Factor D program, we are evaluating the best approach to fund these opportunities and have a range of options to consider. These options cover both equity and debt approaches as well as global business development opportunities. We are pleased that we have multiple alternatives to support our progress. As you may recall earlier this year, we extended our cash runway and enhanced our financial flexibility by closing a $100 million secured credit facility. Based on our successful APeX-2 trial, we currently have the ability to draw $30 million of non-dilutive capital at our option. That would be additive to our current cash balance, as well as an additional $20 million that would be available to us following FDA approval of 7353.

I am thrilled to have Megan's experience on the team to help us evaluate the global market opportunities for 7353 in order to assess where we will want to commercialize it ourselves and where we want to explore partnerships that provide non-dilutive capital.

In order to gauge our future capital requirements to support all of our programs, we need to complete our launch planning and understand BCX9930 Phase I data. We can then pair that information with our commercial investment, clinical development and partnership plans to develop the right financing mix. We look forward to sharing more details on our capital plans with you in the fall.

Our detailed second quarter 2019 financial results can be found in the press release we issued this morning, but there are a few items I need to highlight. We incurred a noncash charge of $2 million in the second quarter of 2019 associated with realizing compensation expense on 2 performance-based tranches of stock options. Although a cashless event and outside our guidance ranges, it is important to note there will be a continuing charge of approximately $3.5 million as these options continue to vest throughout the remainder of the year. And thus, these continuing charges will increase our loss per share in the third and fourth quarters of 2019 beyond our normal run rate. This charge has no impact on our cash utilization or our operating expense guidance for future quarters and the year.

With 2 quarters under our belt, I also wanted to call your attention to cash utilization. We ended the second quarter with approximately $98 million in cash. Our operating cash use for the first half of 2019 was $53 million and is annualizing at the lower end of our projected range of $105 million to $130 million. Additionally, we continue to expect our 2019 operating expenses will be comfortably in the previously guided range of $120 million to $145 million. As we consider the best options for raising additional capital, it is very rewarding to see the strong patient and physician enthusiasm for 7353 and the rapid advancement of our oral Factor D inhibitor program.

Now I'd like to turn the call over to Bill.

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [5]

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Thanks, Tom. Like John, I attended the HAE Association Patient summit in Atlanta, and it was wonderful to see the engagement of patients and caregivers with our team and the tremendous encouragement that they gave to all of us in launching our oral once-a-day kallikrein inhibitor. They came up to our booth or found us in the halls to tell us just how much this treatment option means to them and their families. After years of IV and subcutaneous injections, many patients, even patients who are well controlled on these therapies, told us that an oral drug is a dream come true. Since we announced the APeX-2 data and first shared it with our clinical trial investigators in the United States, Canada and Europe, the response to the data has been very positive with growing momentum. In the last few months, our medical team has conducted a series of regional advisory boards comprised of U.S. allergists and immunologists who treat patients with HAE. Universally, these physicians have responded enthusiastically to the efficacy, safety profile and approvability of 7353 based on the APeX-2 results and tell us that the patient experience on study with 50% of patients experiencing at least a 70% reduction in attack frequency with a once-daily oral drug, will absolutely translate into patients using this medicine.

Lastly, I also had the opportunity to visit with staff of an experienced HAE clinical trial site that has been a major contributor to trials of every new treatment introduced for HAE and was told this drug will be life-changing for the HAE community.

So I'm very happy to report that all the activities needed to wrap up the 48-week dosing and analyses in APeX-2 and APeX-S are on track, and we are very confident in our Q4 target to submit the U.S. NDA and Q1 2020 target to submit both the European MAA and JNDA in Japan.

Moving on to our oral Factor D inhibitor program for complement-mediated diseases, I'm very pleased to say that we have also had strong support from U.S. and international expert physicians for progressing 9930 in the clinic as fast as possible because of the medical need across multiple different disease indications, where the alternative pathway of complement is the dominant bad actor, because they've been hoping for a Factor D inhibitor and especially an oral complement inhibitor drug. The nonclinical profile of 9930 is extremely promising. The key findings were absolutely dose proportional exposure over a big range, human equivalent doses of the normal dose level of 5 grams per day, prompt and sustained suppression of the alternative pathway of complement after oral dosing at a fraction of the normal dose in non-human primates. What these findings mean is that we should have a very wide margin of safe dosing in the clinic to achieve our therapeutic goals in complement inhibition.

The Phase I clinical trial in healthy subjects is off to a great start and is progressing rapidly. We look forward to sharing the safety, tolerability, PK and PD results of this study in the fourth quarter. As we've seen with other programs in the field, Phase I healthy subject studies with complement inhibitors provide vastly more information than the usual Phase I clinical trial, because we can measure suppression of this pathway in healthy subjects using alternative pathway biomarkers that are predictive of clinical effects and because this greatly facilitates dose selection for trials in the target diseases.

Also very important and unlike many other disease areas, there are commercially available standardized assays like the [Wessler ALP assay] that enable comparison across programs. Progression of 9930 from Discovery project to non-clinical development to Phase I in the clinic has gone very fast, and we have closed most of the gap with the oral Factor D inhibitor competition. We look forward to moving quickly to clinical proof-of-concept in patients with PNH in 2020. That will be very exciting as we can easily measure multiple biomarkers like LDH reticulocytes, bilirubin that are both predictive of clinical benefit and very quick to obtain after brief durations of dosing. You can look forward to seeing data on 9930 PNH subjects in 2020.

Now I'd like to turn the call over to Megan.

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Megan Sniecinski, BioCryst Pharmaceuticals, Inc. - Chief Business Officer [6]

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Thanks, Bill. For all the reasons John, Tom and Bill have laid out, I'm really excited to be here at this pivotal stage for BioCryst. It's a privilege to be part of a company that's fully dedicated to bringing life-changing oral treatments to patients with rare diseases.

Looking at what's ahead, I'm eager to bring my experience and energy to building the capabilities and infrastructure we need to commercialize 7353 and to advance our pipeline with the ultimate goal to create value for our patients. In this role, I have accountability for business development and corporate strategy, supply chain and program management. Right now my focus is on execution across 3 key areas: launch readiness, advancing our development programs and exploring BD opportunities that fit with our strategy.

First, for launch readiness, these activities include finalizing our launch plans based on the physician, patient and market insights John mentioned earlier. Actively building out our supply and distribution channel, both in the U.S. and ex U.S., to be well positioned to support the strong patient demand we anticipate. And concentrating our effort on the key resources and investments we need to support a highly successful launch.

The regulatory submissions are also the team's top priority, and we remain on track for the FDA submission in the fourth quarter and the EMA and Japanese submissions in the first quarter of 2020. Knowing everyday matters to the patients we serve, we will be fully focused on ways to rapidly advance our HAE programs as well as our early-stage assets like 9930.

Lastly on the BD front, as Tom noted, we're actively exploring potential partnering opportunities to maximize the value of our assets. For example, the accelerated regulatory path with 7353 SAKIGAKE Designation in Japan presents a real opportunity to consider a local partner, one who has the capabilities and strengths to bring our product to a market which currently has no approved prophylactic treatments.

I too was overwhelmed by the reception BioCryst received at the recent HAEA Patient Summit. This is a highly engaged, aligned and driven patient organization, and I'm very excited to be here and to partner with this amazing community to bring our product to patients in need.

Now let me turn it back over to Jon.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [7]

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Thanks, Megan. Let me wrap up with this. In my 12 years at BioCryst, the company has never been in a stronger position. We are hearing extraordinary customer demand for BCX7353. Next year, we will launch the first oral prophy HAE treatment and a proof-of-concept data on a potential blockbuster oral Factor D inhibitor is coming soon. And finally, we have the financial flexibility to get the capital we need to get there. That concludes our prepared remarks.

Operator, we're now ready to open it up for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Jessica Fye with JPMorgan.

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Daniel G. Wolle, JP Morgan Chase & Co, Research Division - Analyst [2]

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This is Daniel for Jessica. A couple of questions here. First, have you met the FDA to discuss the results of APeX-2? Second, given that APeX-S is an open-label trial, can you speak at a high level to whether the safety in that study is consistent with or meaningfully different from what you have described in APeX-2? And last, with the Phase III acute study now postponed to 2020 for initiation, is it possible that it goes on the back burner for a period of time to preserve cash and focus on the filing for a prophylactic?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [3]

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Daniel, I'll take the first couple. With regard to regulatory interactions on the NDA plan. We've had our pre-NDA meeting that went fine. So all of the elements that we need to submit for the NDA are on track in the fourth quarter including the rate-limiting elements, which are the long-term safety from APeX-S and APeX-2. On that front, there's no news really. We haven't learned anything new that we haven't seen previously in the Phase II and the Phase III program. As I've mentioned on previous calls, we have an independent data monitoring committee that meets regularly to review cumulative data, and they have not suggested any changes to the protocols. So that's ongoing, and we look forward to completing that work later this year.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [4]

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And then with regard to your question about HAE priorities, the prophy program and the filing and the approval is absolutely the #1 priority. You heard Megan talk about her -- one of her main priorities and focus is launch and execution, successful execution of the launch. So that takes the resources, that takes the focus. That is the priority. The acute program has been delayed a bit due to continued regulatory interactions and figuring through the CMC pieces to get to our Phase III. And as you said, we plan to get that started sometime in 2020.

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Operator [5]

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And our next question comes from the line of Maury Raycroft with Jefferies.

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Swapnil Malekar, Jefferies LLC, Research Division - Analyst [6]

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This is Swapnil on for Maury. So just one for 7353. So do you like, anticipate any unique risks with approval process, especially due to available of such strong potent prophylactic therapies out there? And then I have a follow-up.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [7]

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I didn't completely catch your question. I think it was a market competition question, but could you just -- could you restate it?

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Swapnil Malekar, Jefferies LLC, Research Division - Analyst [8]

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Yes. So do you like, think there are, like, any unique risks with the approval process for this 7353, especially given availability of potent prophylactic therapies?

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [9]

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None. So, no, we don't anticipate any unique regulatory assessments with regard to competition. It's all about this drug in front of the regulators and its safety and efficacy profile and the accumulated database that we have in Phase I, Phase II and Phase III studies. So, no, I don't think competition will play into that at all.

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Thomas R. Staab, BioCryst Pharmaceuticals, Inc. - CFO, Principal Accounting Officer, Senior VP & Treasurer [10]

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Yes. And I'll point you to -- I can't remember if it was 2018 or '17, 2018 I guess, the FDA had their patient meeting with the HAE Patient Association, and it was crystal clear from the audience response when they were asked a question, what's your number? Or what's your top priorities with regard to new therapies, and convenience was #1. So they've heard loud and clear that patients want more convenient therapies.

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Swapnil Malekar, Jefferies LLC, Research Division - Analyst [11]

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Okay. And I have one follow-up question on other programs. So for FOP molecule, which is especially targeting ALK2, do you see any like off-target preclinical signals, especially because of closely related targets such as TGF B [tower] active and type 1 receptors?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [12]

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Thanks for the question. So the FOP program is on track to enter the clinic in coming months this year. As you may recall, just to manage regulatory and clinical resources earlier in the year, we made the decision to prioritize 9930. So that's going great. FOP is on track. All kinase inhibitors are multi-kinase inhibitors as, if you're familiar with the field, that's been very interesting to see how that's evolved. There's no such thing as a mono kinase inhibitor. So at some dose level, all kinase inhibitors will have off-target effects. We're very comfortable with the nonclinical profile of the drug, supporting entry into the clinic. And I don't anticipate that, that will be an issue. Obviously, all of our Phase I studies are done very carefully with single ascending dose and multiple ascending dose components. And the Phase I trial for this particular drug will be conducted in healthy subjects.

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Operator [13]

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Our next question comes from Brian Abrahams with RBC Capital Markets.

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Gilbert Roland Kinsey, RBC Capital Markets, LLC, Research Division - Senior Associate [14]

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This is Bert on for Brian. Just wanted to get kind of an update on your current thoughts on the 7353 pricing in terms of what you may be able to charge compared to the competitors in this space to capture share and/or to be tried in earlier lines.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [15]

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Yes. So we're still conducting the payer research and doing the pricing analysis. The key, and I've said this before, is to be able to price it at a point where you knock down the barriers, but you don't leave a lot of money on the table. And we haven't gotten the data to make those decisions at this point in time. But what I will tell you is with our competitors having drugs of upwards of $600,000 per patient per year, we have a lot of flexibility and a lot of room, especially with a small molecule cost of goods. So we'll know more about that later. The key here, and I think this is really important, is with some of the early market research that we're getting showing that the majority of patients want to use an oral medicine with our profile, the feedback we got from hundreds of patients at the HAE Patient Summit, there is a significant opportunity with this drug.

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Operator [16]

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Our next question comes from Liisa Bayko with JMP Securities.

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Liisa Ann Bayko, JMP Securities LLC, Research Division - MD and Senior Research Analyst [17]

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Can you maybe illuminate us as to when we might see a presentation of the data and a little bit more on quality of life and those kind of metrics? And I guess, how important is that latter point?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [18]

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Sure. Hello Liisa, it's Bill. We're working hard on producing materials to present at scientific meetings and also publication planning for the main manuscript for APeX-2. It's always a bit hard to predict when those things are coming out, but our objective is, of course, to support our launch with a high-quality publication in a great journal. And I think the data's terrific, so we feel pretty confident that we can do that. We've got complete engagement from the principal investigator and the other key investigators on the study to help us get those publications out. The upcoming meetings of the College Allergy meeting later this year and [CWID II] early next year and then [ACAAI], we'll be trying to have materials at all of those meetings.

On the last point, with regard to other elements. I think that one of the interesting sets of conversations or a theme of conversation at the Patient Summit was also over the long term, what happens and improvement in patient well-being after months of therapy, and that's probably true for all of these drugs. So I think that story is still evolving, and it'll be interesting to see what type of quality of life data we get out of our long-term safety study as well as APeX-2.

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Liisa Ann Bayko, JMP Securities LLC, Research Division - MD and Senior Research Analyst [19]

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Okay, great. And then for the Factor D program, can you maybe comment on recent data from the competitive landscape? I know there were some data released recently from Achillion. And then how you're -- how you anticipate your content might compare or contrast? What are the key differences, even from a kind of chemistry perspective, just to help us understand. Where do you see kind of the niche after what you've seen and what we've seen so far with Achillion's program?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [20]

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Sure. We have a lot of respect, of course, for all of our competition. The data presented, I think is encouraging for the field and indicates that they are making some progress on their programs. I think it's very difficult at this stage to give you a direct answer in terms of head-to-head comparisons. We'll have more information that everybody will be able to assess once we have our safety PK and PD profile from the Phase I. Just in general, it's worth pointing out that the serine protease field and serine protease inhibitors are difficult targets. That's why there are only 2 companies that we know of, most likely, actually entering the space of oral Factor D inhibitors. We're really thrilled with our preclinical data and we're closing the gap on our competition. So given the market size, I'm not worried that there's more than 1 player.

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Operator [21]

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And our following question comes from Serge Belanger with Needham & Co.

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Serge D. Belanger, Needham & Company, LLC, Research Division - Senior Analyst [22]

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First, a question on the prophylactic program. I think the last time you talked to us back in May, you talked about a potential marketing strategy that would emphasize sampling in a lower-priced product. So after the Patient Summit and some of these other HAE meetings and the feedbacks you've had with patients and physicians, is that still the marketing strategy going forward or has that evolved?

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [23]

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Yes. I think -- well, the strategy is the same that we want to position ourselves by making it easy for patients to get our drug and to move to the front of the line. I think what's different is the opportunity here is much, much, much bigger. Price discounts, I'm not going to talk about that until we get the pricing research done. But we really see the demand for this drug to be huge. And so I think the opportunity is very big.

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Serge D. Belanger, Needham & Company, LLC, Research Division - Senior Analyst [24]

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Okay. And how would you compare, I guess, the European opportunity and how that market has evolved over the last few years with some of these new products?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [25]

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Yes. So there really isn't a prophylaxis market, to be honest with you. I think that's the key difference. Pricing pressure, as we know, in any therapeutic area is harder and heavier in Europe, but our goal is to build a prophylactic market. And so as I've mentioned before, the drugs that have been successful in this space had 90% of their sales come from U.S. and 10% from abroad. I see a real opportunity to build value outside the U.S. as well. So I think it's not that the U.S. won't bring us great value, we think there's significant value in the U.S., but we think there's significant value abroad as well. And the feedback, our experience with patients and the KOLs in Europe. I mean, remember, we've done a lot of our trials in Europe, and we've gotten a lot of excitement. We were approached by people from Brazil, Europe, other parts of South America, Japan, and just a lot of enthusiasm from around the world to get access to our drug.

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Serge D. Belanger, Needham & Company, LLC, Research Division - Senior Analyst [26]

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Okay. A couple of questions for the acute HAE program. Any feedback, I guess, from your meetings, at these recent meetings about the appetite for an oral product? And then can you also talk about how extensive the additional CMC formulation work that needs to be done before you undertake the Phase III trial?

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [27]

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So in terms of the feedback, I don't think I got a single question from a patient on acute. It was all about prophylaxis, and you can understand that, that they'd much rather prevent attacks than treat attacks. So that answers your first question.

On your second question, we just -- we want to have a formulation that's in Phase III that's better than what we had in Phase II, and we're still working through that. We're confident we'll get there, but we're still working through it.

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Operator [28]

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(Operator Instructions) Our next question comes from Gena Wang with Barclays.

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Peter B. Kim, Barclays Bank PLC, Research Division - Analyst [29]

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Just 2 quick questions.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [30]

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Gena, we can't hear you.

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Peter B. Kim, Barclays Bank PLC, Research Division - Analyst [31]

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Can you hear me?

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [32]

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Yes. It's better, thank you.

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Peter B. Kim, Barclays Bank PLC, Research Division - Analyst [33]

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This is Peter for Gena. Just 2 quick ones from us. One is for 7353 program. Do you expect to have any adcom meeting? And our second question is on the CMC formulation for the ZENITH-2. Was there a request by FDA? Or was that more initiated on your end?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [34]

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Peter, it's Bill. It's always hard to handicap whether or not you're going to have an adcom, but you always need to be prepared. So we'll be prepared.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [35]

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Yes, on the formulation front, it was ours, not FDA. We just, as I said before, we want a better formulation in Phase III than Phase II and so it was ours.

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Operator [36]

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Our following question comes from Tazeen Ahmad with BofA Merrill Lynch.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [37]

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Jon, just wanted to follow up on your plans for commercial organizations. So you've got your application underway and almost ready to submit. How are you thinking about the size of the sales force that you might need? Obviously, you're a rare disease-focused company, so you won't need a large infrastructure, but have you started the interview process. Do you think that you might be open to hiring folks from competing companies? And can I also ask what your current cash runway is?

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [38]

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Sure. So with regard to the commercial infrastructure, we're hiring, and we're filling jobs as we speak. So in fact, we're -- and I think it's posted, we've got a head of sales that we're currently recruiting. In terms of the size of the sales force, you rightly pointed out that these are really small sales forces in rare disease. We haven't nailed down exactly what that is until we complete the market research and have a better idea of what the segments are, what the physician population is. We've got a pretty good idea now, but we haven't completed it, and we'll share more of that in the fall.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [39]

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And then on cash runway?

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Thomas R. Staab, BioCryst Pharmaceuticals, Inc. - CFO, Principal Accounting Officer, Senior VP & Treasurer [40]

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Tazeen, it's Tom. Thanks for the question. So on cash runway, it continues to be into 2020, and like I said in my prepared remarks, we have a lot of financial flexibility. Obviously, our cash runway is largely dependent on the commercial outlay and some of the research that John mentioned as well as our plans with the 9930 program. And so it continues to be in 2020, and we'll refine the cash runway as we get more information.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [41]

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Okay. And that includes your plans for commercial structure buildout, correct?

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Thomas R. Staab, BioCryst Pharmaceuticals, Inc. - CFO, Principal Accounting Officer, Senior VP & Treasurer [42]

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Yes, it does.

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Operator [43]

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And the last question comes from Tyler Van Buren with Piper Jaffray.

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Tyler Martin Van Buren, Piper Jaffray Companies, Research Division - Principal & Senior Biotech Analyst [44]

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I had a question on 9930 and the initial data at year-end. I found it interesting that you guys mentioned that it influences financing options, given how early it is. Obviously, safety is important, but can you just -- you spoke about PK-PD suppression of the pathway and standardized assays, so could you give a little bit more specificity on what type of PK-PD measurements you'll be looking at to measure suppression of the pathway and what magnitude of suppression would be promising as you guys think about moving into PNH patients?

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William P. Sheridan, BioCryst Pharmaceuticals, Inc. - Chief Medical Officer & Senior VP [45]

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Sure. So I would frame it up as follows. And a lot of this you can get from the literature on complement and the development of other products and investigational drugs in the field of complement inhibitors. So there are some commercial standardized assays that interrogate the alternative pathway, which is -- Factor D is the critical insight into an alternative pathway. And one of those is the [Wessler BP assay]. So that's the first thing. And the reason that's important is because you can buy the kit and follow the instructions. So if everybody buys the kit and follows the instructions that makes the data a bit more interpretable. One program to the next to the next. The remaining assays are more bespoke, but they're well published. And there are a whole range of them including AP hemolysis assays, Factor D B assays and the like. And a lot of that is in the literature. So we have a comprehensive set in healthy subjects that you can measure. You might ask, how can you measure these things in healthy people, they don't have the disease. The reason is the alternative pathway is always switched on. It's always ticking over. So you can measure suppression of that in healthy people. So it's a very comprehensive set. And what we're really looking for here is very strong suppression of the alternative pathway. If we don't get very strong suppression with our drug, that will be a big surprise on the basis of -- that would be a big surprise on the basis of the data we've shown with oral dosing in the monkey. That's in our public slide decks. So we're looking forward to seeing very strong suppression of the alternative pathway. And that will be the -- that will be a key element in interpreting the Phase I.

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Operator [46]

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I'm showing no further questions at this time. I would now like to turn the call back to Jon Stonehouse for closing remarks.

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Jon P. Stonehouse, BioCryst Pharmaceuticals, Inc. - CEO, President & Director [47]

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Yes, thank you. So as I said in the prepared remarks, I don't think we've been in a stronger position at BioCryst. We're about to file and launch a drug that I can tell you, having been at the HAE Patient Summit, patients really want. The demand is extremely high. And that leads us to conclude that the opportunity here is significant. And the great news is filing the end of this year and launch next year. On top of that, we've got oral Factor D inhibitor with 9930, that's a pipeline in a molecule. As I said, the current market doubles that of HAE, and it could double from there with additional indications. And so advancing that and advancing that quickly, so that we get another great oral drug out to patients, many patients, with different rare diseases is another great opportunity. And we look forward to starting to update you on all the progress that we make this fall. So thanks again for your interest in BioCryst, and have a great day.

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Operator [48]

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Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program, and you may all disconnect. Everyone have a great day.