Q3 2020 BioCryst Pharmaceuticals Inc Earnings Call
[music].
At this time all participants are in a listen only mode.
Operator: At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to your speaker today, Jon Bluth from BioCryst. Thank you, and please go ahead. Thanks, Samantha.
After the speaker presentation, there will be a question and answer session to.
Unknown Executive: Good morning, and welcome to BioCryst's third quarter 2020 Corporate Update and Financial Results conference call. Today's press release is available on our website. Participating with me today are CEO Jon Stonehouse, CFO Anthony Doyle, Chief Medical Officer Dr. Bill Sheridan, Chief Business Officer Megan Snizinski, and Chief Commercial Officer Charlie Geyer. Following our remarks, we'll 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.
Unknown Executive: 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. Thanks, Jon.
Jon P. Stonehouse: Good morning, and thank you all for joining us. We are 28 days from our PDUFA day with Orladeo, and we are ready to launch. Charlie and Megan will review the details around our readiness. But suffice it to say, we've been getting ready for this day for many years, and it's so exciting to be less than a month away from bringing our oral once-daily medicine to HAE patients who've been waiting. In addition, our partner in Japan, Torii Pharmaceuticals, is ready to launch. Late last week, we were informed of a recommendation for approval by a PMDA review committee, and we expect an approval decision by the Japanese Ministry of Health in December. While the market is much different from the U.S., this is also very exciting, as Orladeo will be the first prophylactic treatment approved for HAA patients in Japan.
Joining us we are 28 days from our PDUFA date, with Orlando and we are ready to launch Charlie and Megan will review the details around our readiness, but suffice it to say we've been getting ready for this day for many years and it's so exciting to be less than a month away from bringing our oral once daily metal.
And to HIV patients who've been waiting.
In addition, our partner in Japan, Tory Pharmaceuticals is ready to launch late last week, we were informed of a recommendation for approval by a PMDA Review Committee and we expect an approval decision by the Japanese Ministry of Health in December.
While the market is much different from the US is also very exciting as orla Dale will be the first prophylactic treatment approved for HIV patients in Japan.
In Europe, we were excited to announce last week that that the MH IRA has granted us a positive scientific opinion under their early access to medicines scheme in the UK.
Jon P. Stonehouse: In Europe, we were excited to announce last week that the MHRA has granted us a positive scientific opinion under their Early Access to Medicine scheme in the UK. This allows HAE patients and physicians in the UK to gain experience with Orladeo prior to approval. We expect European approval early next year, and launch preparations by our team in Europe are well underway.
This allows ha patients and physicians in the UK to gain experience with Orlando prior to approval.
We expect to European approval early next year and launch preparations by our team in Europe are well underway.
Our plan is to make orla Dale available to patients around the world to that end, we started planning for the next wave of countries to gain approval and bring this oral once a day medicine to patients.
Jon P. Stonehouse: Our plan is to make Orladeo available to patients around the world. To that end, we've started planning for the next wave of countries to gain approval and bring this oral once-a-day medicine to patients. Our company is transforming.
Our company is transforming having a product that we believe can generate over $500 million in global peak sales about to enter the market.
Jon P. Stonehouse: Having a product that we believe can generate over $500 million in global peak sales about to enter the market, and a pipeline right behind it with our oral factor D inhibitor for multiple complement-mediated diseases, enables us to create sustainable value for years to come. We have many major milestones in the coming months, but perhaps the most important is the U.S. approval of Orladeo. I will share more details on how we are prepared for a successful launch. I'll now turn the call over to Charlie and Megan. Thanks, Jon.
And a pipeline right behind it with our oral factor Xa inhibitor for multiple complement mediated diseases enables us to create sustainable value for years to come.
We have many major milestones in the coming months, but perhaps the most important is the us approval of Orlando.
To share more details on how we are prepared for a successful launch I'll now turn the call over to Charlie and Megan.
Thanks, John.
The Orlando PDUFA date is exactly four weeks from today and we are ready to launch.
Charlie: The Orladeo PDUFA date is exactly four weeks from today, and we are ready to launch. We're ready because we started investing in this launch years ago. We invested early because we knew that a deep understanding of the attitudes, demographics, and access needs of our customers would be critical to success. We also invested early to build an experienced team that is ready to execute our plan. I'll describe each of these key investments in more detail.
We are ready because we started investing in this launch years ago.
We invested early because we knew that a deep understanding of the attitudes demographics and access needs of our customers would be critical to success.
We also invested early to build an experienced team that is ready to execute our plan.
Ill describe each of these key investments in more detail.
First we invested in understanding our customers.
Charlie: First, we invested in understanding our customers. When we did market research to understand HAE patient and prescriber attitudes and behaviors, we didn't cut corners. We conducted very large physician and patient surveys to gain a clear and unbiased view of how our future customers will respond to Orladeo. When we surveyed 175 HA treating physicians, for example, that's an example of an audience that treats 1,300 patients, over 10% of all HA patients in the U.S. Here's what that comprehensive research tells us.
When we did market research to understand ha patient and prescriber attitudes and behaviors, we didnt cut corners.
We conducted very large physician and patient surveys to gain a clear and unbiased view of how our future customers will respond to Orlando.
When we surveyed 175 HIAA treating physicians for example, that's a sample that treats 1300 patients over 10% of all HCV patients in the us.
Here's what that comprehensive research tells us.
Patients are grateful for the injectable treatments that exists because they remember having nothing they cope with their injections, but they want something more.
Charlie: Patients are grateful for the injectable treatments that exist because they remember not having them. They cope with their injections, but they want something more. Patients want more because the burdens of injectable treatment, such as preparation time, difficulties in administration, and needle fatigue, all limit their freedom and detract from their ability to live normal lives. Patients and physicians have consistently told us that they see targeted oral therapy as the next big thing and the natural evolution of HAE therapy. This explains why half the patients we surveyed who say they are very satisfied with their injectable therapy still want to switch to Orladea.
Patients want more because the burdens of injectable treatment such as preparation time difficulties in administration and needle fatigue, all limit their freedom and detract from their ability to live normal lives.
Patients and physicians told us consistently that they see a targeted oral therapy as the next big thing and the natural evolution of HIV therapy.
This explains why half the patients we surveyed who say they are very satisfied on their injectable therapies still want to switch to Orlando.
We've seen this movement already as 50% of the patients enrolling in apex asks in the US are switching from treating with tax IRO, Hey, Garda or sunrise.
Charlie: We've seen this movement already as 50% of the patients enrolling in APEX-S in the U.S. are switching from treating with Taxiro, Hegarta, or SINRAC. Megan will tell you more about how these clear patient preferences, new data on the burden of treatment, and the opportunity for an oral once-daily option are all starting to change how physicians think about HAE treatment. As part of our market preparation, we also invested in understanding the universe of healthcare providers who treat HAE. In rare diseases, there are no off-the-shelf solutions for customer targeting. You can't get traditional pharmacy prescriptions, so over the past several years, we layered on over 10 data sources to build and refine our HAE treater database. We use this data to structure our sales team, and that team started making profiling visits to their future customers in July.
Megan will tell you more about how these clear patient preferences, new data on the burden of treatment and the opportunity for an oral once daily option are all starting to change how physicians think about HIV treatment.
As part of our market preparation, we also invested in understanding the universe of healthcare providers, who treat HPP.
In rare diseases, there are no off the shelf solutions for customer targeting you can't get traditional pharmacy prescription data.
So over the past several years, we layered over 10 data sources to build and refine our HIV treater database.
We use these data to structure, our sales team and that team started making profiling visits to their future customers in July.
What we hear back from the sales team is that our list is on target.
Charlie: What we hear back from the sales team is that our list is on target, and that H.A. treaters are engaging. And with less travel right now, our reps have more time to go even deeper into their target list with customer outreach.
And that ha treaters are engaging with them.
And with less travel right now our reps have more time to go even deeper into their target list with customer outreach.
We also invested in understanding how payers will cover Orlando and how we will help customers through the access process.
Charlie: We also invested in understanding how payers will cover Orla Deo and how we would help customers through the access process. A year ago, we did qualitative interviews with 16 payers representing over 100 million covered lives, and since then, we completed additional quantitative work with 56 payers that represent over 200 million covered lines. Payor feedback has been consistent.
Charlie: They recognize the value of an oral therapy for HA patients, but they don't want to pay a premium for it. They tell us they will cover Orladeo if it costs them what they pay for injectable therapies, like Higarda and Taxiro, products that are priced at $500,000 to $600,000 per year. Equally important is how we invested to smooth the Orladeo access process itself. We set out to build a best-in-industry patient services program by taking great care to understand customer experiences, both positive and negative. There are several ways we will deliver on our goal, but I'd like to highlight two important components. The first is that every healthcare practice and their patients will have a dedicated care coordinator. We heard repeatedly from patients and providers about their frustration with being transferred from person to person during the access process.
Charlie: We also heard about the value of long-term relationships between patient service programs and their patients. Our dedicated coordinators are ready to streamline the access process, coordinate product shipments to patients, and build ongoing relationships with both patients and health care providers. The second component is that we are closely linking Orladeo patient services with our field teams to support patient access. Our field market access team has already built relationships with reimbursement coordinators in the top 200 HAE treating practices, and our team is ready to assist with prior authorizations, payer education, and other steps to speed patient access to Orladea. And finally, we invested in people. We've built a U.S. commercial team that knows how to execute.
Shipments to patients and build ongoing relationships with both patients and healthcare providers.
The second component is that we are closely linking Orlando patient services with our field teams to support patient access.
Our field market access team has already built relationships with reimbursement coordinators in the top 200 ha trading practices and.
And our team is ready to assist with prior authorizations payer education and other steps to speed patient access to Orlando.
And finally, we invested in people.
Charlie: Our commercial leadership team has a track record of rare disease launch success in oncology, gene therapy, as well as previous HAE launches. They hired a sales team that averages 20 years of industry experience, including nearly 10 years in rare diseases. Each one of them joined BioCryst because they understand the value that Orlodeo will bring to patients. They have a passion for launching products, and they want to be part of the growth and transformation of BioCryst. Our investments are about to pay off because the team is ready.
Charlie: We're ready to launch the day Orladeo is approved. We're ready because we've invested in understanding this market. We're ready because we know that HEA patients have been waiting years for a targeted oral therapy. We are ready because we don't want patients to wait any longer. Now, I'll turn the call over to Megan to describe her medical team's preparation. Thank you, Charlie.
Megan: Like the commercial theme, medical affairs is launch ready and eagerly awaiting our PDUFA date. Our preparation and execution to date have focused on two key priorities. First, gaining insights into today's HAE treatment paradigm and how this is evolving. And second, generating and publishing important data that supports the clinical benefits and potential of Orladeo. Let me turn first to the insights from our HCP interaction. Physicians are recognizing that patients still have unmet needs today despite the advancements in prophytherapy over the last several years. It's not enough to just reduce attacks. Patients now want to significantly reduce or eliminate the interference treatment has on their lives. With multiple profile alternatives available, each with its distinct profile, we're hearing clearly from HCPs that shared decision-making has become even more important in HAE.
First gaining insights into todays HCV treatment paradigm and how this is evolving and second generating and publishing important data, which supports the clinical benefits and potential for Orlando.
Let me turn first to the insights from our HCP interactions.
Physicians are recognizing patients still have unmet needs today, despite the advancements in prophy therapy over the last several years.
Megan: It's not possible for physicians to know which therapy is best for patients because the decision isn't just one dimension. Instead, physicians must partner with their patients to understand each individual's needs, personal goals, and preferences when it comes to treatment. And the way many physicians we speak with see it, Orladeo helps accelerate this shift, whether it's from our market research or what we've heard from physicians across hundreds of interactions. We know patients have been wanting an oral option and are even actively asking their HCP about it. Patients want to decrease their burden, even if they're doing well on their current treatment, and Orla Deo represents an ideal choice for this unmet need. It offers the chance to not only reduce the tax but also reduce the burden of treatment.
Megan: It's a convenient, more discreet, effective therapy with no needle trauma or prep, ultimately helping patients reach the goal of leading a more normal life. We're focused on educating physicians about the disease burden that remains, and the research data we share with them resonates with them. Our work continues to focus on driving this awareness and providing tools to physicians to help them navigate the shared decision-making process with their patients. Patients will also push for this. There are highly engaged communities that advocate often and consistently.
And providing tools to a physician to help them navigate the shared decision making process with their patients.
Patients also will push for this.
There are a highly engaged communities that advocates often and consistently the patience desire for an oral treatment will amplify the need for physicians to engage in the share dialogue.
Megan: The patient's desire for an oral treatment will amplify the need for physicians to engage in this shared dialogue. The other strategic priority for our launch is generating and publishing important clinical data. These publications will serve as critical resources for both our medical and sales team in the launch phase. Last month, Jackie, the leading journal for the Allergy and Immunology Academy, published the pivotal trial results from our APEX 2 study. The authors reinforce that Orlodeo is an effective, targeted oral prophytherapy and represents a major step in allowing patients to live a normal life. We also have a strong presence at the upcoming College Congress on November 13 through the 15th.
The other strategic priority for launches generating in publishing important clinical data. These publications will serve as critical resources for both our medical and sales team in the launch phase.
Last month, Jackie believing at journal for the allergy and Immunology Academy publish the pivotal trial results from our apex to study.
The authors reinforced that Orla day wasn't effective targeted oral profi therapy and represents a major step in allowing patients to live a normal life.
We also have a strong presence at the upcoming College Congress on November 13th through the 15th we have six accepted abstract including a distinguished oral presentation for 48 week clinical data.
Megan: We have six accepted abstracts, including a distinguished oral presentation for our 48-week clinical data. The new analyses show Orladeo's continued treatment benefits on clinical outcomes, including attack rate reduction and improvements in quality of life and patient satisfaction scores. In addition, for the first time at a scientific forum, we will present the findings from our Comprehensive Burden of Treatment Survey with patients, caregivers, and HCPs. Our research shows how today's prophylactic treatment impacts patients' and caregivers' lives and reveals the opportunities we have with Orladeo to reduce this burden. The medical team has been out virtually engaging with the physician community.
The new analyses show Orlando's continued treatment benefit on clinical outcomes, including a tax rate reduction and improvements in quality of life and patient satisfaction scores.
Megan: Our clinical data is responding, and the exchanges are helping to advance the shift in the HAE treatment paradigm I spoke of earlier. As we look ahead at the launch during COVID, physicians tell us they're still seeing their patients either in in-office visits or remotely via telemedicine. Given Orladeo's profile, the pandemic doesn't create any barriers to initiating treatment for our oral therapy. This is particularly important should COVID restrictions significantly limit in-person care visits. Plus, as Charlie explained, we're able to ship directly to patients, no trips to the pharmacy are required, and no refrigeration challenges occur. And unlike injectable options, there's no patient training requirement on how to administer an oral once-daily capsule.
Megan: Lastly, in terms of readiness, we're prepared from a supply perspective with ample drug product and inventory. We are ready for the final packaging activities, with a plan to have finished packs available for shipment as soon as possible after approval. As we transform into a global commercial company, we're equally excited by the pipeline we have behind Orladeo. I'll hand the call over to Bill for more on our clinical progress and upcoming data readout. Thanks, Megan.
We're equally excited by the pipeline, we have behind Orlando I'll hand, the call over to bill for more on our clinical progress and upcoming data Readouts.
Thanks Megan.
The multiple of dialer is exciting for patients exciting for physicians and exciting for bikers.
Bill: The launch of Olodeo is exciting for patients, exciting for physicians, and exciting for BioCryst. Oral drug, psoriasis is really mass. At BioCryst, we discover, develop, and commercialize oral medicines for rare diseases. And all day, I was at the front of the train with a pipeline of homegrown investigational new drugs right behind me. Lolodeo was discovered by our research team in Birmingham in October of 2013.
Oral drugs for rare diseases really matter.
Bill: And now, just five years from its first phase one study, HAE patients may be less than a month away from the oral, once-daily medicine they have been waiting for. We are applying the lessons from the successful Olidio development program to the other programs in our pipeline. Next in line behind Orladeo is BCX9930, our oral factor D inhibitor for complement-mediated diseases. In September, we reported outstanding clinical data with BCX9930 monotherapy in treatment naive PNH patients receiving up to 400 mg BID. We saw rapid and dose-dependent reductions in key biomarkers, including LDH, and increased hemoglobin levels in all PNH patients in the trial. Increased hemoglobin levels were maintained without transfusion.
Bill: BCX9930 has been safe and well tolerated at all doses in the trial. No drug-related serious adverse events have been reported. Seven PNH patients naive to C5 inhibitor treatment are currently receiving BCX9930 in this trial, with four patients beyond 12 weeks of therapy, including two with more than 32 weeks. And all seven treatment-naive patients are continuing to benefit from BCX9930 treatment. Our goal with this dose-ranging study is to determine the optimal dose of BCX9930 to apply across an advanced development program that includes multiple complement-mediated diseases. We now plan to complete the trial by enrolling up to eight additional subjects, including up to six subjects with inadequate response to C5 inhibitors. The overall total, including the previously enrolled patients naive to C5 inhibitors, will be up to 16 patients dosed with BCX9930 up to 500 mg twice a day.
At 930 in this trial.
<unk> for patients beyond 12 weeks of therapy, including two with more than 32 weeks.
And all citizens treatment naive patients are continuing to benefit from 90 930 treatment.
Alcohol with this post ranging study is to determine the optimal dose B 690, 930 to apply across an advanced development program that includes multiple compliment many other diseases.
I mean, they have planned to complete the trial on rolling up to eight additional subjects, including up to six subjects with inadequate response to see five inhibitors.
The overall total including the previously enrolled patients naive to see five inhibitors will be up to 16 patients those to be <unk> 90, 930 up to 500 milligrams twice a day.
We plan to report comprehensive clinical data for both treatment naive patient and inadequate responders to see far treatments once enrollment and dose ranging Ah completed.
Bill: We plan to report comprehensive clinical data for both treatment-naive patients and inadequate responders to C5 treatments once enrollment and dose ranging are completed. Our trial sites for the inadequate responder patients are now open in Europe, but the resurgence of COVID and renewed lockdowns in many European countries have impacted the start-up phase and enrolment. Given these impacts and our enrolment goals, we expect to complete and report the completed dose-ranging study in P and H, including inadequate responders, in the first quarter.
Bill: We've had productive interactions with regulators on the program to discuss next steps in this indication, including study designs for the advanced development of BCX9930 and PNH, which I plan to start next year. You may recall that the FDA has granted both fast-track status and Orson drug designation for BCX9930 and PNH. In 2021, we expect to be conducting advanced development trials in PNH, as well as exploring BCX9930 in new indications. Three big reasons why BCX9930 is so exciting. For one, we couldn't be more pleased with the clinical data for 99.30 we have seen thus far. 2. It represents a pipeline for a molecule with many indications.
Bill: 3, it is coming right behind all of that. As you'll now hear from Anthony, we are fully resourcing our continued clinical progress with VCX9930 and delivering a successful all-day award. Thanks, Bill.
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Thanks Bill.
The PDUFA date for Orlando is fast approaching we will continue to invest in this transformational event for the company and in the coming quarters. We look forward to discussing the revenue we generate from this next evolution in the AG space, which will transform our financial position.
Anthony J. Doyle: The BDUFA date for Orladeo is fast approaching. We'll continue to invest in this transformational event for the company, and in the coming quarters, we look forward to discussing the revenue we generate from this next evolution in the HEE space, which will transform our financial position. While we invest in this major catalyst, we also continue to work with Bill and the clinical team to invest in BCX9930 and the development cycle pushing that program forward across multiple complement-mediated diseases. You can find our detailed financials in today's earnings press release, and I'd like to call your attention to a few items. We ended Q3 with $149 million in cash. Our operating expenses, not including non-cash stock compensation, for the quarter were $46 million, and were $127 million through the third quarter.
While we invest in this major catalysts. We also continue to work with Bill and the clinical team to invest in BCS 90, 930, and the development cycle pushing that program forward across multiple complement mediated diseases you.
You can find a detailed financials in today's earnings press release, and I'd like to call your attention to a few items.
Anthony J. Doyle: Based on our ongoing investments in the Orladeo launch and the 9930 program, guidance previously provided for net operating cash usage and operating expenses remains unchanged. Cash runway also remains unchanged, with cash on hand through Q2 of next year. With the imminent inclusion of revenue from Orladeo adding to our balance sheet, we continue to evaluate additional sources of capital, including royalty under debt financing, partnerships for 99.30, and other financing options. We believe that the options available to us provide us with outstanding financial flexibility. These are exciting and transformational times at BioCryst. The tremendous work by the team has gotten us to this point, where we have so many upcoming catalysts. Our continued investment in the development of BCX9930 and the launch of Orladeo is indicative of both the strong position that we're in and our confidence that these investments support the company in delivering on its strategy and providing value to our shareholders. Thanks, Anthony.
Is completed enrollment and we are waiting for the virology data typically legs in these studies as it needs to be processed and analyzed at a central lab.
We remain confident we will report data later this quarter.
Part one is the dose ranging part of the study the.
The primary endpoint of safety we.
We will also collect data on secondary endpoints, including clinical outcomes and Barology.
In recent conversations with our major funding partner of the program NAIAD, we understand that this data is of gaining item for the program.
Jon P. Stonehouse: In addition to our commercial transformation with Orladeo and the exciting clinical progress with 9930, we are also completing the Phase I trial of BCX9250, our oral drug for FOP, and Part I of a clinical trial in COVID-19 patients with our broad-spectrum antiviral Galidese. Part 1 of the GALADESIVIR study has completed enrollment, and we are waiting for the virology data that typically lags in these studies as it needs to be processed and analyzed at a central lab. We remain confident we will report data later this quarter. Part one is the dose-ranging part of the study, and the primary endpoint is safety. We will also collect data on secondary endpoints, including clinical outcomes and virology. In recent conversations with our major funding partner of the program, NIAID, we understand that this data is a gating item for the program. Well, the study's not powered to show efficacy, but some evidence of clinical and or virologic activity is important for the program to continue to move forward. So we look forward to reporting the data later this quarter. That's it for our update.
While the studies not power to show efficacy, some evidence of clinical and or by real logic activity is important for the program to continue to move forward. So.
So we look forward to reporting up the data later this quarter.
Jon P. Stonehouse: As I stated at the beginning, our company is changing. The transformation to a commercial stage company with an exciting pipeline creates a very bright future for patients and for shareholders. With multiple approvals, data updates, and revenue coming soon, you will have the opportunity to see this transformation for yourself, and we look forward to updating you along the way. That's it for our prepared remarks. We will now open it up for your questions. And, ladies and gentlemen, as a reminder, if you would like to ask an audio question, you will need to press star 1 on your telephone. To withdraw your question, press the pound.
Operator: Please stand by while we compile the Q&A. Your first question comes from the line of Jessica Fye with J.P. Morgan. Good morning, this is Daniel from. Thanks for taking our question. A couple of questions here, starting with Orladeo. Is it possible for you to set expectations on the cadence for volume uptake and early part of launch? Is there potential for early access programs in the U.S. and U.K. to drive a bolus of patients who might start out the gate or will be more? Yeah, thanks for the question, Daniel. We're not going to give guidance.
Press release, there is a statement, saying that you have plans to add to take new patients.
When you take these patients are you starting them at 400 milligram or you're an up dosing them to 500 or are you comfortable with the safety profile to start dosing them at 500 milligram dose.
Bill you want to take that one.
Sure Daniel Thanks for the question I'm very comfortable with the safety profile at all doses tested up to and including 500 milligrams twice a day.
Jon P. Stonehouse: This is our first launch. We're in a COVID environment. You know, you're right that there are a lot of patients on the drug currently, and that could affect the uptake, but we're just not going to give guidance at this time. We're really confident that this drug has the potential to be north of a half a billion dollars in global peak sales. And, and so, and as Charlie and Meg have said, we're ready. And switching to 9930, what doses are the seven treatment-naive patients currently on? And in the press release, there are statements saying that we have plans to take new patients. When you take these patients, are you starting them at 400 milligrams and updosing them to 500? Or are you comfortable with the safety profile to start dosing them at 500? Bill, do you want to take that one?
The goal of the study is to have.
Have adequate information across a broad dose range southern where they can select the best dose to take forward into controlled studies best development. So our but that's the point of the study.
Bill: Sure, Daniel, thanks for the question. We're comfortable with the safety profile of all the doses we've tested, up to and including 500 milligrams twice a day. The goal of the study is to have adequate information across a broad dose range, so then we can select the best dose to take forward into controlled studies in advanced development. That's the point of the study.
Bill: We'll give an update on exactly what doses and how many subjects at each dose once we complete the study. I think we gave a good, pretty thorough, update in September, so I don't want to go over that again today, but yes, the answer to your question is we'll be testing doses up to and including 500 milligrams twice a dose. Okay, thanks. And one last...
Jon P. Stonehouse: Given the recent setbacks with competitors in the FOP setting, can you tell us a little bit more about 9250 and how it differentiates from the competitors? So, Bill, I'll start, and then maybe you can follow up. It's a recurring lesson that we learn in this space of rare diseases and the importance of an oral drug. We saw this in HAE with companies that were ahead of us and then sold out, companies behind us that couldn't come up with once-a-day drugs, and now we see the same thing, as you mentioned in FOP. So, that's why we continue to press forward in investing in 9250. It's an L2 inhibitor, so it acts directly on the target that affects the disease, but I'll let Bill describe it a bit further. Sure, so as Jon just mentioned, this disease is caused by a mutation in a kinase in the, which happens to be a transmembrane protein with the kinase domain inside the cell.
First in human study.
Study has progressed well and we.
Look forward to reporting the results in the last quarter this year.
You know I think that the medical need for patients with proper dysplasia assist against progressive is absolutely no unless it's a terrible disease and the struggles with.
With that pellet Parroting program and they act as an eye anti body program there.
Really clearly demonstrate that that medical need is just as strong and so I think that going after.
Going off to the mutated protein directly and resetting the activity is that upregulated, it's kind of nice.
Still looks to us to be a really promising approach and you know we have goodness moves forward.
Bill: So, in our non-clinical research, we showed that oral dosing with BCX9250 could achieve effects inside the cell in animal experiments where we measure heterotopic ossification, which is a hallmark of this disease. So that was the signal we needed to invest in our first-in-human study. That study has progressed well, and we look forward to reporting the results in the last quarter of this year. So, you know, I think that the medical need for patients with fibrodysplasia ossificans progressiva is absolutely enormous.
Thank you very much.
You're welcome.
Your next question comes from the line of Kara Bancroft with Piper Sandler.
Hi, guys. Good morning, I was hoping maybe you could provide a little more detail on the number of sales reps that you have and in terms of manufacturing the the packets, how many pills should be given per pack and do you have a plan for sampling.
So Charlie you want to us yes.
Yes, So hey, Terry this is Charlie thanks for the question.
Bill: It's a terrible disease, and, you know, the struggles with the palovarotene program and the active in A antibody program really clearly demonstrate that that medical need is just as strong. And so I think that going after the mutated protein directly and resetting the activity of that upregulated kinase still looks to us to be a really promising approach, and, you know, we hope it moves forward. Thank you very much. You're welcome. Your next question comes from the line between Tara Bancroft and Piper Sandler.
Sorry, I guess I, just got distracted by a number of the number of sales reps in number of sales sorry, yeah, sorry, as we've said before we haven't just for competitive reasons. We haven't wanted to disclose exact number but what we've been saying is we know our competitors to be in the range of 30 to 50.
And the all the Salesforce dot the targeting information I described in our remarks I told us that that was the right size will fit right in that range of 30 to 50 U.S. sales representatives.
Megan you want to take the packaging question.
Charlie: Hi guys, good morning. I was hoping maybe you could provide a little more detail on the number of sales reps that you have, and in terms of manufacturing the packets, how many pills should be given per pack, and do you have a plan for the samples, Charlie? You want to... Yes, so, Tara, this is Charlie.
Charlie: Thanks for the question. Sorry, I just got distracted by the... The number of sales reps? The number of sales reps, yes, sorry. As we've said before, we haven't, just for competitive reasons, we haven't wanted to disclose the exact number, but what we've been saying is we know our competitors to be in the range of 30 to 50, and all the Salesforce, the targeting information I described in our remarks told us that that was the right size, so we'll fit right in that range of 30 to 50 U.S. sales reps. And, Megan, you want to take the packaging question?
Megan: Sure, Jon. Hi Tara. This is Megan.
Jon P. Stonehouse: So we haven't sort of disclosed formally the product presentation, but as you can imagine, for an oral once-daily treatment, the product packaging will certainly support the ease of a patient taking one capsule once a day. And Tara, what was the third part? I thought there was a third part to your question. Yeah, yeah. I was actually wondering if you guys plan on, Yeah, in this space, there's things called quick start that, you know, allow a patient to get access quickly while they're going through the reimbursement process. I hate to use the word sampling because that has connotations of mass market products where reps bring boxes of samples into a doctor's office. That is not the case here,
Jon P. Stonehouse: But the quick start program is absolutely a part of the offering. You know, a big piece of our strategy is when a doctor makes a decision to write a script so that that patient gets the product as quickly as possible. And so, you know, through the hub services Charlie talked about and all the other things that he described in preparation, we want to do that as well or better than anybody in this space. Thank you. You're welcome. Your next question comes from the line of Gina Wang with Barclays. Hi there, this is David on behalf of Gina.
Programs I was just novartis tattered be there in.
In the refractory patients how do you see Dennis visitors Mexican of action and also drug profile differentiated from the program.
Bill you want to take that.
Sure. Thanks for the question second D is a very attractive targets assist catalytic Amazon and the alternative parkland complement very proximal so blocking <unk> kind of way you too <unk>.
Block Opsonization.
And resolve the extravascular hemolysis.
Bill: Thanks for taking our questions. So one question on 9A30. Given the good long-term durability observed for the competitor programs, which is Novartis Factor B inhibitor in refractory patients, how do you see 9A30's mechanism of action and also drug profile differentiate from the program? Bill, do you want to take that?
That occurs in patients being treated with say five inhibitors as well as blackie blocking all of the downstream six and treating the introduction homolysis at the same time. So approximal complimented edition in general is very attractive the number of indications here is enormous and the future use.
Bill: Thanks for the question. Factor D is a very attractive target. It's the first catalytic enzyme in the alternative pathway of complement, and very proximal, so blocking Factor D can allow you to block opsonization and resolve the extravascular hemolysis that occurs in patients being treated with C5 inhibitors, as well as block all of the downstream effects and treat the intravascular hemolysis at the same time. Proximal complement inhibition, in general, is very attractive.
<unk>.
This class of medicines is going to be very important across the Friday indications dermatology indications hematology indication.
So there's room for more than one player that's for sure.
Bill: The number of indications here is enormous, and the future uses. This class of medicines is going to be very important across. Nephritis Indications, Rheumatology Indications, Hematology Indications, The other programs are at an early stage, too, and as we were just discussing with FOP a few minutes ago, things can happen. So, you know, you never know what's going to happen to those other programs as we progress. We're very excited about the profile we're seeing with 9930. It has superb dose proportional exposure in pharmacokinetics.
Bill: It has crystal clear and consistent and robust suppression of the alternative pathway in pharmacodynamic assays and incredibly impressive clinical results in our dose-ranging study in P and H patients, so we couldn't be more happy. I think that we really look forward to getting our advanced development program up and running after we complete our first study. Great. Thanks for hanging out.
Jon P. Stonehouse: Your next question comes from Jon Wolleben with JMP Security. Hi, good morning, congratulations on the progress, and thanks for taking the questions. I have one for each of the most advanced programs, and I guess I'll start with Orladeo.
Megan: Can you remind us of the benefits of the Segagaki designation and if any of those change if the U.S. approval comes before the approval in Japan? So Megan, you want to, you want to tackle that one? Sure, Jon. Hi Jon.
Megan: So, the benefit that we receive from the Sagagaki designation is the accelerated review schedule. So, again, with us being in the late stages, we've certainly benefited from that advantage. The other benefit comes with respect to the pricing discussions and negotiations. There would be premiums within the final completed price if you, with the Sagagaki designation.
Megan: So, again, that's another benefit. And I think your last question was with respect to any change if U.S. approval comes first. The spirit of the designation is actually to incent companies to bring innovative medicines to Japan first, and we've achieved that by submitting the JNDA ahead of the file being accepted with the FDA. So, again, we feel really confident in where we are and look forward to the Ministry of Health approval decision next month. Yeah, and I think the other thing I'd say, Jon, is, like I said in the prepared remarks, a completely different market.
We expect that with you know a real convenient and therapy. That's highly effective that it's also going to increase the diagnosis of HIV patients in Japan. So we're super excited about the launch with Tori.
Jon P. Stonehouse: And it'll be the first prophylactic therapy for patients, and we expect that with real convenience and therapy that's highly effective, it's also going to increase the diagnosis of HAE patients in Japan. So we're super excited about the launch with TORI.
Got it that's helpful.
190, 930 with data from poor responders come in first quarter now what do you think of the minimum amount of follow up you would want to have before reporting that data.
Bill.
Thanks for the question so the.
Jon P. Stonehouse: That's helpful. In one on 99.30 with the data from C5 correspondents coming in first quarter now, what do you think is the minimum amount of follow up you'd want to have before reporting that data? Phil?
That's a huge advantage of studying patients with paroxysmal nocturnal include area. It dose ranging studies is that the hotlines that like tight dehydrogenase is sort of short.
So it's only like one to three days.
Three days at the outside so like 14 days of treatment.
Bill: Thanks for the question. The huge advantage of studying patients with paroxysmal nocturnal hemoglobinuria in dose-ranging studies is that the half life of lactate dehydrogenase is so short. So it's only like one to three days. Three days is the average outcome. So 14 days of treatment gives you a very good handle on pharmacodynamic biomarker effects. Obviously, longer durations of treatment give you a handle on safety, and we're doing both of those things. So the original idea of the study was that we could get a lot of value out of 28 days of treatment with forced titration, and I'm happy to say that has proven to be true. So I don't want to give you a minimum duration, but the original design had that initial 28 days.
Jon P. Stonehouse: That's helpful. And just the last one for me, could you provide a little more color on the conversations with NIAID as far as gating factors are concerned? Is there some level of clinical or antiviral activity? And were these conversations before or after the more recent Remdesivir publications in the New England Journal and then all the subsequent WHO publications? Yes, so the conversations happened very recently.
Jon P. Stonehouse: As you know, companies are working really hard, and so is the government in terms of bringing therapies to patients who need them. You know, there's the approval of remdesivir. There are antibodies that are being investigated, oral therapies.
Jon P. Stonehouse: And so, you know, like any program, you want to see some benefit, right, from your therapy, and that's what NIAID is looking for. I don't think there's any impossible hurdle here in terms of what they're looking for. They completely understand that the study is not powered to show efficacy, but some signal would be beneficial, and so we're really excited to see the data. Thanks for the update and congrats again on the progress. As a reminder, if you would like to ask an audio question, please press star then the number one on your telephone keypad. Your next question comes from the line of Brian Abrahams with RBC Capital Markets. Hi, this is Steve Mellon. I'm on behalf of Brian Abrahams.
I would like to ask an audio question. Please press Star then the number one on your telephone keypad.
Your next question comes from the line of Brian <unk> Abrahams with RBC capital markets.
Hi, This is Steve Welling them on for Brian Abrams. Thanks for taking my question I was just a little or no 30 program can you give us a little more color on the next steps and.
You know what might end points look like in Registrational trial, there and maybe.
Any additional indications you might pursue.
Bill you want to take that.
Sure I think that I would.
Jon P. Stonehouse: Thanks for taking my call. Just on the 99.30 program, can you give us a little more color on the next steps? What might endpoints look like in a registrational trial there, and maybe any additional indications you might receive. Bill, do you want to take that?
Bill: Sure. You know, I think that, you know, I would encourage those of you who are interested to look at the studies that others have done. That sets a precedent in the field, obviously.
Bill: Our goal is to have a broad label and monotherapy with BCX9930 to treat this disease, and we're very confident about that. And so our intent is to create an advanced development program with rigorously designed controlled studies that achieve those objectives. And you can look forward to hearing more about that once we start those studies next year. With regard to endpoints, there's a limited selection. They're all pretty obvious.
Bill: Clearly, the goals here are to fix the anemia, stop the hemolysis, fix the anemia, and prevent transfusions and eradicate the symptoms of the disease. So we measure all of those things, and in previous studies, things like reduction in transfusions, maintenance of hemoglobin, and reduction in LDH have all been used as endpoints. So, again, once we've completed all of the required steps, we've started the study, so we'll talk in more detail about the... Primary Endpoint in the Hierarchical Order of the Second. Yeah, Steve, I would say a couple more things. One, if you look at our slide deck, you'll see a slide that has the mechanism and then a number of different indications in a box on the right-hand side These are extremely attractive to us.
Inside those are extremely attractive to us and so that gives you some indication of what will consider to pursue and then the goal is next year to have.
Multiple us clinical trials advanced development studies in multiple indications next year so.
Jon P. Stonehouse: And so that gives you some indication of what we'll consider pursuing. And then the goal is next year to have multiple clinical trials, advanced development studies, and multiple indications next year. So, super excited that we have a pipeline and a molecule of 99. I think that I would just add another comment with regard to just the incredible enthusiasm that we're getting from our external collaborators who are top experts in hematology and nephrology, for example. They see that this mechanism of action and the dose finding in PNH can be translated directly into these other areas, and they see just how strong this drug is performing.
I'm Super excited that we have a pipeline in a molecule at 90 930.
Yeah, I think that I was just add another comment with regard to just the incredible enthusiasm.
Alright that we're getting from an external collaborators who topics in hematology an apology for example, they see that this mechanism of action and that does finding in pain and I just can be translated directly into these other areas and they see it just has strongest.
Drug is performing sore.
This program is going to be completely transformed it.
Bill: So this program is going to be completely transformative. Your next question comes from the line of Serge Belanger with Needham & Company. Hey, good morning.
Okay.
Your next question comes from the line of search Ellinger Needham and company.
Hi, Good morning couple of questions on overdue, John you're 28 days from the <unk>.
Charlie: A couple questions on Orladeo. Jon, you're 28 days from the PDUFA deadline. You're probably limited in what you can say about the ongoing review, but can you comment on whether the pre-approval manufacturing inspection has been completed? And then, a second question regarding expected payer coverage for Orlodeo. How long do you think payers will have a policy for the product, you know? Is it a six-month process? And then will prior authorizations kind of reflect what we see now with the current prophylaxis? Okay, so I'll take the first one, Serge, and then... Charlie, I'll take the second piece on the payer.
Probably limited and where you can say about the ongoing review, but can you comment on.
Whether the preapproval manufacturing.
Inspection has been completed and then second question regarding.
They're expected pair coverage for <unk>.
Uhm, what how long do you think pairs will have a policy for the product you know is it a six month process and then we'll be prior authorizations.
Reflect.
What we see now with the current prophylactic.
Excellent.
Okay. So I'll take the first one surgeon then Charlotte I'll take the second piece on the payer Yeah. I mean, we're 28 days away. So we have to be way down the path and R. As I said before our confidence level is high I'm not gonna get into the specifics we've referenced before what F. D. A has said about.
Jon P. Stonehouse: Yeah, I mean, we're 28 days away, so we have to be way down the path. And as I said before, our confidence level is high; I'm not going to get into the specifics. But we've referenced before what FDA has said about manufacturing inspections in the world of COVID. And you know, depending on where you're manufacturing, they'll either accept previous FDA inspections if there are no major issues, or they'll accept foreign country inspections if there are no issues. And so, and I think we've mentioned to you before, we have dual manufacturers for both drug substance and drug product, and three of those four are domestic, they're here in the US. So the confidence level is extremely high going into 28 days to PDUFA. And then Charlie, do you want to talk about the payer?
Manufacturing inspections in the world of Covid and they'll depending on where you are manufacturing they'll either accept previous FDA inspections, if there's no major issues or they'll accept foreign country.
Inspections I've there are no issues and so and I think we've mentioned to you before we have dual manufacturers on both drug substance and drug product and three of those for our domestic they're here in the U S. So confidence level extremely high going into 28 days to Paducah, and then Charlie you Wanna talk about the air Yeah. So.
<unk> have you heard and my my remarks, we recently finished research with.
Charlie: Yeah, so, Serge, as you heard in my remarks, we recently finished research that we did with 56 payers covering over 200 million lives. And the big point is that they expect to cover Orladeo. I mean, that's something that we've been hearing consistently.
Charlie: Now, that said, you know, with any disease, any drug launch, particularly in rare diseases, it doesn't mean all payers will cover it on the exact same time schedule. So we expect that there's going to be a mix, and our market access team is really well prepared for that. There will be a number of payers that will cover right out of the gate, and there will be others that we just have to work with to educate them a little bit more. And we've got that all in our planning. We've got a really experienced market access team who've done this before in other rare diseases and has a great track record of success. So the ultimate, the end point, though, is we're looking to get access for all patients who want and can benefit from Orladeo, and we're confident we'll get there.
Charlie: Oh, and sorry, you also asked about the prior authorization similarities. Yeah, we expect, you know, and we've said this before, the main tool that most payers use in HAE is prior authorization. They just want to make sure that patients really have HAE, and all of our work with payers says that they're going to prior authorize this drug just the same way they would do with Taxira or Higarda. There's one thing, one kind of exception to that or one opportunity we see is that a lot of patients are switching from existing prophylaxis.
Charlie: We expect a lot of those patients to switch over. So they've already been through the PA process, and we expect that that will reduce the burden of that process. Your final question comes from the line of Maurice Raycroft from... Hi, this is Swapnil Anpurmori. One question on the Galida Severe trial. Can you walk us through some of the scenario analysis of what NIAID and BARDA might look like before stockpiling Galida Severe for COVID-19? Yeah, I think jumping to stockpile is a huge leap from where we are today.
Jon P. Stonehouse: We haven't gotten into the exact details of what you have to see, but we will certainly review the data with them, and, you know, we'll get their input on, you know, what activity we see or don't see from that data, and, you know, then we'll communicate what the plan is moving forward. Okay, and then one question on 7353. So is there any status update on higher doses that you're planning to dose in order to see an improved efficacy for HAE? We don't think we need it. The 150 mg dose is an excellent dose. We see patients go from having roughly three attacks per month down to one.
It's a highly competitive profile and with a once daily oral as.
Jon P. Stonehouse: It's a highly competitive profile, and with a once-daily oral. As Charlie mentioned and Megan mentioned, patients have been waiting, and doctors are ready. So are we. Okay, thank you for taking our questions. You're welcome. I'm showing no further questions at this time.
As Charlie mentioned and Megan mentioned.
No patients have been waiting and doctors are ready. So so are we.
Okay. Thank you for taking my questions.
You're welcome.
Im showing no further questions at this time I would now like to turn the call back over to Mr. Stonehouse for any additional or closing remarks.
Jon P. Stonehouse: I would now like to turn the call back over to Mr. Stonehouse for any additional or closing remarks. So we're really excited about that. I hope that you come away from this call having listened to Charlie and Megan and that we made really smart investments early on to be ready. And I am extremely confident that this team and the things that we've done to get ready are going to position us for a successful launch.
Yes. So the next earnings call, we have we'll be talking about an approved product and how we are launching orla Dale. So we're really excited about that I hope that you come away from this call having listened to Charlie and Megan that we made really smart investments early on to be wrong.
So, the company's changing. You're going to be seeing it over the coming months, and we look forward to updating you along the way. Thanks for your interest, and have a great day. Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. Thank you for watching!