Q3 2021 Global Blood Therapeutics Inc Earnings Call
Greetings and welcome to the global Blood Therapeutics Conference call. At this time all participants are in a listen only mode. A brief question and answer session will follow the prepared remarks should anyone require operator assistance. During the conference. Please press star zero on your telephone.
Keypad as a reminder, this conference is being recorded I would now like to turn the call over to Steven. Please go ahead.
Thank you and welcome to Gbt's conference call to discuss the company's financial results for the third quarter 2021 and to provide a business update I'm, Steve <unk> head of communications and Investor Relations.
With me today on the call are Dr. Ted Love, our President and CEO.
Jeff Farrow Chief Financial Officer.
David Johnson or D J, Chief commercial officer.
And Dr. Kevin Smith, Whitely, Executive Vice President and head of R&D.
During today's call Ted will provide an update on our progress in the third quarter.
Jeff will review our financial results D. J will give an update on the ox prior to launch.
Ken will discuss our pipeline and then Ted will give a few closing remarks before opening up the call for questions.
Earlier this afternoon.
We issued a press release announcing gbt's business progress and financial results for the third quarter ended September 32021. In addition, this morning, we issued a press release announcing six data presentations at the upcoming Ash annual meeting in December.
We will also hold an R&D event for investors and analysts on November 11th.
Before we begin I would like to remind you that certain statements. We make on this call that are not historical facts may be forward looking statements that are subject to risks and uncertainties.
Information concerning factors that could cause actual results to differ materially from those expressed or implied by such forward. Looking statements are contained in our SEC filings, including but not limited to our most recent quarterly report on Form 10-Q, as well as in today's press release copies.
Copies of our SEC filings and press releases can be obtained from the investors page of our company website at TVT Dot com.
The forward looking statements made on this call are only as of the time. They are made and you should not place undue reliance on such statements.
Future events or simply the passage of time may cause our beliefs to change.
We disclaim any obligation to update any forward looking statements other than that as required by law.
That I will turn the call over to Ted.
Thank you Steven and good afternoon, everyone.
<unk> continues to make solid progress on our mission to transform the care of sickle cell disease and improve the lives of patients around the world.
We are well position for long term success, both with our Friday, which has several upcoming catalysts for particularly for growth and our robust pipeline, which we believe can have further improve clinical care and F C D and expand the market.
Turning to the quarter, we delivered approximately 850, new prescriptions in Q3 a.
A decrease compared to the second quarter.
This reflects headwinds from the spike in new COVID-19 cases in the U S in August and September.
Which were at their highest level since early January.
In many states.
As widely reported in the news.
ICU capacity was strain.
Many sickle cell health care providers were diverted to address the urgent needs.
Sadly in early October Covid deaths in 2021 surpassed the number of deaths in 2020, despite the availability of vaccines this year.
While the surgeries and the pandemic infection rates.
Especially in areas, where many sickle cell patients live did not subside in Q3, we remain very optimistic for the future given the tremendous unmet need in this disease.
We also continued to refine and expand our commercial efforts to drive Ox Friday awareness education and access.
I'll provide a strong fundamental.
And long term potential give us confidence heading into 2022.
Let's consider the following.
The net number of patients taking a sporadic continues to increase each quarter.
Which contributed to us delivering on our third quarter revenue expectations.
The growing body of rollout real world evidence, including new data covering nearly 2700 patients.
That will be presented at ash validates the role of box spreader as potential foundational therapy for sickle cell disease.
Our market research demonstrates high levels of satisfaction, among prescribers and patients who have tried ox Friday.
We also have received positive feedback and results from our educational and marketing programs, including our groundbreaking direct to consumer advertising campaign.
And we have near term opportunities to significantly expand the number of patients eligible for ox Brad.
Importantly, we have an FDA action date of December 25th.
On our approval application to expand <unk> Friday.
Label to include patients as young as four years old along with the new pediatric formulation.
In Europe the market authorization application is on track for a potential approval in the first half of 2022.
We were in position to further build on these positive fundamentals and near term catalyst as the pandemic subsides and health care visits returned to normal.
We believe these factors will gradually contribute to new prescription growth in 2022.
With potentially more robust growth in the second half of the year.
In addition, we believe this timing will coincide with achieving broad payer coverage for children ages four to 11.
Each will help fuel our growth.
We are also advancing our pipeline and we will have exciting updates at ash.
Frank lack of map, we will have new phase one data to present that we believe supports the best in class potential.
For GBT six are one we will present the first data from our phase one study in both healthy volunteers and sickle cell patients.
We believe these 601 data will be position to provide proof of concept for this program and started to fulfill the tremendous potential we see for GBT. So one to provide a functional cure and appeal for sickle cell disease.
All of this activity demonstrates gpt's commitment to this community.
And in September we launched the GBT Foundation.
A nonprofit charitable organization that will fund programs that support the sickle cell disease community and beyond through education empowerment access and health equity.
Also in September GBT co hosted the 10th annual sickle cell disease Therapeutics conference.
Which brings together community leaders to discuss the latest advances in future trends in FCB.
Importantly, there were several sessions on COVID-19, vaccines, including education on how they work and people sharing their experiences getting the vaccine.
This is a critical part of our work and we will continue to support the sickle cell community.
With that I will turn the call over to Jeff to review, our third quarter results.
Thank you Ted totaled.
Total net revenue from sales of Ox Friday was $52 1 million third quarter of 2021, consistent with the guidance range we provided.
Third quarter revenue increased by $15 2 million or 41% year over year.
On a sequential basis third quarter revenue increased by nine 5% from the second quarter.
This sequential growth was driven by the continued increase in the net number of patients on last Friday, including.
Including demand from existing and new patients and reflected lower new prescriptions in the quarter a trend that we believe could continue into the fourth quarter.
Days of inventory on hand in the third quarter was relatively flat, although absolute levels of inventory increased reflecting the growing arthritis patient base.
Gross to net was approximately 14% a slight decrease from the second quarter, reflecting less Medicaid and co pay deductions, partially offset by higher 340 <unk> sales.
Like most in our industry, we saw lower than anticipated new prescriptions in the third quarter, primarily driven by the surge in Covid cases from the Delta variant.
We believe this headwind will continue in the fourth quarter.
And we remain cautious on the overall COVID-19 environment, given who we are heading into the winter months and there continues to be lower rates of vaccination among black Americans as compared to the overall population.
Sales are also typically impacts in the fourth quarter by major holidays.
Including significantly lower visits to Hcp's as office is shut down in the second half of December.
Given these factors for the fourth quarter, we expect revenue of approximately $54 million to $56 million.
This range assumes an incremental increase in gross to net.
Flat inventory dynamics compared to the third quarter.
While in the prior year, we saw an increase in inventory levels in the fourth quarter, we don't have enough history to predict trend with certainty.
Looking forward, we continue to anticipate as the pandemic gradually improves and as we gain more traction with our commercial efforts, we will see a corresponding improvement in new prescriptions and revenue growth.
Now turning to expenses.
Cost of sales for the third quarter was 830000 as compared with 513000 for the third quarter of 2020 and consistent on a gross margin basis year over year.
Cost of sales was low in both years as the majority of the manufacturing costs related docks bridal sales were incurred prior to FDA approval industrial were recorded as an R&D expense.
R&D expense for the third quarter of 2021 was $51 million compared with $40 million for the same period in 2000.
The increase in R&D expense in the third quarter was primarily due to costs related to the advancement of our preclinical programs.
As well as our GBP 601 phase one study and two o'clock I Mab phase III studies.
We continue to anticipate an incremental increase in R&D expense in the fourth quarter driven by the <unk> studies, which includes the $5 million milestone payment by GBT.
And the advancement of GBP 601, and other pipeline activities.
SG&A for the third quarter of 2021 was $68 million compared with 55 million for the same period in 2020.
The increase in SG&A expense was primarily due to increased employee related costs, including noncash stock compensation and supporting.
The commercialization of <unk>, including the rollout of new materials, and our direct to consumer advertising.
Other factors driving this increase were our measured expansion into Europe, and the initiation of multiple ox Friday investigator sponsored studies.
We anticipate another stepwise increase in SG&A expense in the fourth quarter driven by these same factors as well as launch readiness activities in preparation for a potential pediatric approval.
Net loss for the third quarter was $71 million compared to $60 million for the same period in 2020.
Basic and diluted net loss per share for the third quarter was $1 13 per share compared with 97 per share for the same period in 2020.
We continued to be well positioned with a strong balance sheet with cash cash equivalents in marketable securities of $417 million at quarter end compared with $561 million at December 31, 2020.
This includes the opportunistic addition of approximately $44 million during the quarter from net equity proceeds from our at the market or ATM financing.
And with that I will now turn the call over to D. G.
Thank you, Jeff and good afternoon, everyone.
I will provide an update on three key metrics that will give you further insight into our progress. These metrics are new prescriptions for <unk>, brighter, which informs underlying patient demand.
The number of health care providers, prescribing Okta, Friday, which captures the progress we are making in adoption.
And payer coverage, which speaks of the access environment <unk> Friday.
First new prescriptions, there were approximately 850, new prescriptions for <unk> during the quarter, reflecting progress with our recently launched initiatives offset primarily by the headwinds from the Spike in COVID-19 cases in August and September driven by the spread of the Delta variant.
Unfortunately, many of the regions that had the highest growth in the Delta way also have the highest numbers of sickle cell disease patients <unk>.
<unk> made many southern states, such as Texas and Georgia.
This resulted in fewer interactions between health care providers in SCD patients and fewer engagements between health care providers and our field teams. In addition, we saw that some SCD clinicians, particularly in the southern states were pulled away from routine patient care in order to treat COVID-19 patients.
All of this contributed to fewer new prescriptions in the quarter.
As we monitor the Covid environment and think about recovery. We believe the key leading indicators that are predictive of a return to growth or the improvement in industry wide new to brand prescriptions and S. C. D patient visits beginning to return to historical levels.
In order to improve our ability to drive new prescriptions.
We have a variety of strategies to drive ox rider awareness adoption and adherence we regularly evaluate the size and structure of our commercial operations to optimize our engagements and in fact in October we reorganized some of our commercial function into two business units.
We believe this change will enhance communication alignment and synergies and ultimately accelerate growth in the number of patients on a Friday.
I remain confident in our team's strategies and tactics and believe they will be positive growth drivers going forward.
Our targeted patient campaign launched at the end of July and features actual occupied a patient from their families. They clearly highlights ox bright and messages and serves as an empowering call to action for patients to engage with their health care providers, the commercial which broke ground as the first of its kind in sickle cell disease, well exceeded its targeted audience reach.
In the initial month of the campaign.
While it is still early to measure pull through prescriptions, we are seeing encouraging signs that more patients are seeking information about ox writeup.
The campaign is running on multiple channels, including connected TV social media in print and can be viewed on ox Friday dotcom.
To complement our DTC campaign, we continue to focus on education.
We believe having a fundamental understanding of the root cause of SCD is critical in understanding the value of box rider and initiating patients on therapy.
In the fourth quarter, we are activating a specialized sickle cell clinical team throw an established partner to do virtual education on SCD pathophysiology with our targeted Hcp's. They will also set up branded engagements for our sickle cell therapeutic specialists. This team is actively being trained and will be deployed starting December one and throw out too.
<unk> thousand 22.
We are also educating <unk> on the importance of improving oxygen delivery, reducing hemolysis and educating patients on the potential impact of low hemoglobin.
In Q3, we also successfully conducted branded ox brightest speaker education events with patients. We believe having trained HCP experts, providing education directly to patients is another important way to inform patients about their disease and ox brighter.
It is our goal that is branded and unbranded awareness increases the large portion of SCD patients that remain untreated.
With any therapy engage with their hep's and initiate ox brighter therapy.
In the third quarter, we continued to make progress improving new prescription conversions.
New offerings like cover my Meds, which make the prior authorization process easier and more efficient for health care providers are helping us improve the rate and speed of taking new prescriptions from enrollment to patients starting on therapy during the quarter at Friday adherence, which includes compliance and persistence was stable and within the range of our analog.
In addition, a significant number of patients that discontinued therapy are restarting on ox Friday.
To further support adherence in the fourth quarter, we plan to launch two new features for GBT source solutions, our patient hub.
First we are improving our email communications with newsletters time to a patient journey with GBT source to provide information on how to access and use the hub.
And ongoing support provided by our nurse adherence program.
Second we are optimizing our communications with patients by deploying mobile messaging with a focus on increasing engagements and reducing discontinuation.
Our market research also continues to support the strong fundamentals of Ark Friday.
Nearly all patients report that they experienced some form of symptom improvement and are likely to recommend it to others and nearly all health care providers, who are aware of Ark Friday have already prescribed it or plan to in the future.
That said the research also shows that COVID-19 continues to negatively impact patients' ability to maintain their appointments and complete lab work to monitor their disease.
Looking at our Friday use we continue to see broad range of characteristics, such as a baseline hemoglobin and boc burden, suggesting that prescribers are increasingly recognizing the importance of addressing polymerization and long term health.
Which leads me to my second metric health care provider penetration during the quarter total interactions with health care providers remain below pre pandemic levels hospitals and institutions in states like Texas, Louisiana, and Florida were restrictive or cautious about in person visits.
Against this backdrop, we still added about 130, new prescribers in the quarter, bringing our total prescribers to more than 1800 since launch when we look at the breakdown of writers we continue to see prescriptions being written by both specialists to non specialists, which we believe is a positive trend, but the long term trajectory of the launch.
Turning to payer coverage in 2020, we achieved broad coverage with more than 90% of covered lives having access in the United States.
This strong coverage has contributed to improved efficiency and converting new prescriptions to patients starting therapy.
We are now focused on making it easier for physicians to prescribe and patients receive ox Friday. This include streamlining the process for physicians with cover my meds working with plans to simplify patient level process for coverage and providing access to our co pay program.
Our team is also laying the groundwork for the potential pediatric label expansion.
By meeting with payers to educate and prepare them for the expansion of the approved patient population down to four years old.
This work gives us confidence that we can achieve broad coverage for the four to 11 year old age group faster than we did in the adolescent and adult population.
On a similar note I also want to highlight that our teams are well underway with launch preparations. This includes ongoing engagement with HCP targets.
That serve a large number of pediatric patients to discuss ox brightest current approved indications.
Gaining experience through the pediatric early access program.
Advancing medical and scientific education initiatives, including sharing initial data from the EAP such as the pediatric quality of life data presented at F. S C D or through our medical science liaisons and finalizing our market research and related launch strategies.
I'm confident that we will be well prepared for potential pediatric expansion and the entire team is extremely excited at the opportunity to bring out Friday to younger pediatric patients, who we believe can benefit from ox writer.
And with that Kim will now talk about the developments in our pipeline.
Thank you D J and good afternoon, everyone on today's call I will provide a preview of the data we will present at ash.
<unk> annual meeting in December along with an update on our efforts to potentially expand the label and geographic reach of <unk> right now.
We will present three posters at ash that further support the safety and efficacy of box right.
The first is an expansion of the previously reported Symphony claims analysis.
Now includes nearly 2700 sickle cell patients ages 12 and older.
The second covers durability of response and safety data for the long term use of Fox <unk> and the open label extension of the Phase III Hope study.
And the third is an update it read out from a retrospective retro registry and 10 U S sites.
As a reminder, we recently launched the retro and prospect post marketing registry to enable a deeper understanding of vacs brightest long term efficacy and safety and provide additional real world evidence that we believe will support itchy and sickle cell disease patients.
Plan to update our educational materials, reflecting this data does that our M. S. Alex can proactively share the latest real world evidence immediately after the ash meeting.
Turning to the pipeline.
Frank lack of map R. P. Selectin inhibitor for reduction of ESP, we have initiated two phase III studies collectively means drive.
One is evaluating the reduction of ESP over a 48 week treatment period and the other is evaluating the 90 day. He is C. Readmission rate following an initial POC hospitalization, which tragically occurs and around 50% of patients.
At the Ash meeting, we will present data from our phase one study and then Clacker Mad in healthy volunteers. The result of which we believe support its best in class potential as a medicine for quarterly dosing.
We believe this would be a meaningful improvement for patients compared to monthly dosing and aligns well with a typical sickle cell disease practice schedule of quarterly check ins.
For GBP 601, our next generation hemoglobin polymerization inhibitor. We are excited to present, the first data from our phase one study at the Ash meeting.
This includes data from healthy volunteers and in patients with sickle cell disease.
Both the preclinical data and the healthy volunteer data that was released today in our abstract.
Observed a linear PK and dose dependent increases in hemoglobin occupancy with 601.
The single dose healthy volunteer data exceed the corresponding right of hemoglobin occupancy achieved over a similar single dose range.
As a sickle cell patient data at Ash continued this trend and shows hemoglobin modification and the 30% plus range. We believe GBT six O one could be a potential best in class therapy.
And what's really exciting for patients is that we believe this level of efficacy has potential to eliminate all symptoms and long term organ damage and many patients providing a functional cure in a once daily pill.
As the FDA continues to review our approval applications for potential pediatric expansion. We also continue to see strong interest in our early access protocol, which we expanded from 50.
250 patients age four to 11.
We believe that beginning treatment earlier has the potential to modify the course of their disease and alleviate future serious and life threatening complications by mitigating red blood cell signaling and destruction.
Turning to Europe, we remain on schedule with their review of our marketing authorization application.
We continue to believe our MAA could be approved in the first half of 2022.
In advance of potential approval, we are building momentum with early access programs with a particular focus on France.
The United Kingdom, and Germany, which are of the highest strategic importance.
We are also working to make <unk> available in the middle East and potentially also in Latin America.
In the Gulf Cooperation Council region. We are ahead of schedule on our regulatory filings and in October we received approval for <unk> in the United Arab Emirates, Our first approval outside of the U S.
And prepare for multiple growth opportunities ahead.
While COVID-19 continues to have an impact.
Our country is making progress.
This pandemic will come to an end.
And we are seeing progress in the sickle cell disease community has more people impacted by the disease received COVID-19 vaccination.
As we move forward Gpt's commitment to supporting the sickle cell community is stronger than ever as we strive to improve the lives of our patients.
As we approach the end of the year.
I wanted to take a moment to highlight that we recently celebrated our 10 year anniversary.
I want to thank everyone. That's been involved with our success over the past decade.
We have truly made incredible progress and are grateful to be positively impacting the lives of patients.
We received a significant recognition for this impact last week with our proprietary receiving the very prestigious Prix gallium <unk> USA Award for best Biotechnology product.
Some of our original discovery team Frac spread or were on hand to receive the award in New York City.
It is a tremendous honor to receive this award.
And the result of hard work by so many.
Yes, it was bittersweet.
And shortly thereafter, we learned of the passing of Hertz and the <unk>.
A well known sickle cell warrior and talented artists who use his personal battle.
And as powerful artwork to advocate for patients and raise awareness of the disease.
We knew hartwell.
He was in our office a few days before his passing.
And presented GBT with an installation.
Beautiful and complex depiction of sickle cell disease.
As of 601.
The second is looking at single and multiple ascending doses in SCD patients I'm.
I'm going to discuss the data in a single dose healthy volunteer study and how we interpret this data.
With each single dose, we measured hemoglobin occupancy levels.
From a single dose of 601 at 50 milligrams. The main preliminary occupancy was just under 1%.
At the <unk> thousand 500 milligram dose the mean preliminary percent hemoglobin occupancy was 25%.
This exceeded the hemoglobin occupancies reported for healthy volunteers, receiving single doses of <unk> over a similar dose range.
When plotting this data and the box <unk> data together on a graph the trend line for 601 is definitely steeper than that than that which we saw with <unk>.
What does this mean for the multiple dose study.
In general with a long half life.
Half life drug additional separation would be expected to occur with multiple dose data versus the single dose data in healthy volunteers as well as patients.
Before I wrap up I also wanted to clarify that the reference to the GBP 601 abstract used to support the comparison <unk> published an error. The correct publication has the title pharmacokinetics and pharmacodynamics of <unk> GBP 440 in healthy adults and <unk>.
<unk> with sickle cell disease, which was published in the British Journal of clinical pharmacology in February of 2019.
I also wanted to mention that GBP 601 was well tolerated with the Hilli.
Healthy volunteer study and the single dose study in SCD patients.
While there was one serious AE in the healthy volunteer study. This AE was not related to the study drug overall.
And receptor occupancy.
Using <unk>.
<unk> versus 601.
And ultimately the increases in hemoglobin that you saw with <unk> versus 600 Wonder if you could just clarify that because I know in the publication there isn't a box solitaire arm.
So with <unk>, it's a little bit complicated, but only in sickle cell mice.
In sickle cell mice. Unlike every other species, we tested <unk> tour has.
Unpredictable exposure, so we can't get the Occupancies the target occupancies that we'd like so in the published studies with <unk> <unk>.
Several animals did achieve the target occupancies and we see robust responses in the towns mouse model.
But what we do see with 601 in comparison is significantly lower doses were able to achieve the.
Target Occupancies.
In addition at similar occupancy was 601, we see significantly greater efficacy because compared to <unk>.
Got it okay. Thank you.
The next question is from a cash to worry from Jefferies. Please go ahead.
Thanks, so much so if we look at the data today and healthy six one looks about three to five times more potent than a Friday and I guess the simple math of all doing is saying if it's getting dose about 10% 10 X lower than ox bright out with 102 hundred milligram dose are we really going to get a fair shot on goal in terms of hitting that 30% modification threshold and kind of the downstream effects.
In hemoglobin now you've talked about the loading dose and you've you've mentioned that it'll be applied twice and we will basically accelerate the process to getting a steady state hemoglobin modification. So a what is the loading dose and how will it be applied for 601 and do you feel that the data at ash will be a fair read on <unk> hundred one's ability to increase hemoglobin and modify.
In hemoglobin now you've talked about the loading dose and you've you've mentioned that it'll be applied twice and we will basically accelerate the process to getting a steady state hemoglobin modification. So a what is the loading dose and how will it be applied for 601 and do you feel that the data at ash will be a fair read on <unk> hundred one's ability to increase hemoglobin and modify.
Hbf and then maybe if I can sneak this in.
Can you also comment on if you were you'd be willing to pursue an accelerated approval pathway with <unk> hundred one and if we make any details on that at the R&D day. Thank you.
In a strong position to.
Make that conclusion or not at ash, So we feel good about that.
Uhm with regard to accelerated approval.
What I've always said in drug development is get some data.
Talk to the F D a and you'll have a good discussion rather than going to the F. D. A with a bunch of hypothetical and having a terrible discussion with my career. So right. Now we are really focused on getting some data. So that we understand ox Friday and then we will go and have a <unk>.
For a discussion with the F D. A about our development strategy and then we will tell you about it but we really don't want to put the card before the horse started talking about our development strategy.
Before we know quite.
Quite frankly pretty much about the drug. So we're just trying to proceed with this in a very thoughtful way. So we can give you good feedback I I will tell you that I personally at this point I'm not leaning towards an accelerated.
Brutal strategy I'm more focused on a full approval strategy I think that may be possible. If we get that kind of efficacy profile that we anticipate we've made but again that's that that's really a statement that I'm, making on the basis of no data, but simply speculation about what the data might look like.
Understood. Thanks, so much.
The next question is from at least yeah, yeah some cancer.
Go ahead.
Hey, guys. Thanks for taking my question I Congrats on the.
Or the freak out and that's awesome.
I guess again, continuing continually around six O y and I apologize.
You know it it seems like a single does he tried to 25% it seems like with multiple there's obviously it'll be more how do you think that translates on T. V. O C reduction in and is that something that you think now is kind of more important than it was with an expire they have like the same level of importance from a regulatory standpoint.
Yeah, it's a good personal easier and gallon by Kim to follow on and add anything to this but I would say is that.
I think we've demonstrated already with <unk> bright out.
Associated with the disease. So I think all the science or actually fits together very well and we would expect.
Once we get to bigger studies.
With sticks out one if we're modifying more hemoglobin, we would expect the hemoglobin levels to be higher in the VLC reductions to likely be lower we have to prove all that and we know we need to prove out that but the science has been actually remarkably consistent.
Great. Thank you.
The next question is from Mark Breidenbach from Oppenheimer. Please go ahead.
Hey, good afternoon. Thanks for taking my question just a very quick one quickly for me.
I'm wondering if we're actually going to see multi dose hemoglobin occupancy data in sickle cell patients in the ash presentation or they're just gonna be healthy volunteers or is it just gonna be single single dose hemoglobin occupancy and then maybe I'm wondering if we can get P. J to just comment on any.
Major differences between your your pediatric launch strategy versus what you've already been doing in adults and I'm I'm, just trying to get a sense for how much of an overlap. There is in terms of target health care providers and things of that sort of thank you.
Kim do you want to talk about the ash fix out one multi Donaldson D. J would take the question about pediatric launch.
Yes, Mark Thanks for that question I think that it's our intention Kid show multiple ascending dose data in individuals with sickle cell disease in healthy volunteers.
Yeah, Mark this is D J regarding the pediatric pending.
A potential approval and launch them. So we are in full fledge launch preparation mode mode. As you would expect the good news is 97% of the patients between four and 11 years old in the United States or in geographies, where we already have sickle cell therapeutic specialist.
Hi.
The vast majority of the physicians that treat those patients are also already in our target list and being called on we are expanding our target list by about 200 sickle cell specialists at approval and those represent the incremental 200 physicians that are really only focused on younger kids and don't have anyone currently.
12, and older. So there'd be no reason to be in.
Our current target list. So it's a very small group of expansion and we've already got relationships with the vast majority of other pediatric hematologist. So we feel really good about that and leveraging that and of course, they have some experience with ox Brian over the last couple of years in the 12 and older.
But the preparation on on many fronts is the same in terms of.
Great work by our payer access teams preparing the payers, having those meetings, making sure we can get broad coverage as quick as possible our patient support services and educational initiatives that we plan to launch with are all being developed.
Mmm.
You're probably not gonna change that if.
If you have a reactionary approach so the the whole point of developing disease modifiers is to intervene.
Offend chilly when people are still normal tried to correctly anemia, and and children you may be able to do that very early and you're able to do it reliably and chauffeur.
If you intervene early.
And keep them normal as opposed to wait for their hemoglobin to go down to <unk>.
Six or eight or nine that's not normal and you accumulate disease, when you're when you're running around with those levels. I mean, we know already uhm that uhm, 15% somewhere in the range of 10% to 15% of kids have suffered salon ischemic strokes in the first decade of life. So.
I'm not sure.
Why we'd be content with that if we could do something about it.
Okay. Thank you very much.
Mhm.
The next question is from Sharon Lee Some curious securities. Please go ahead.
Hi, Thanks for taking our questions.
A couple of them, but do you have any idea as to what percent occupancy unique to.
To extract the most staircase benefit while avoiding potential side effects.
What do.
Do you think you will start to run into certain weird oxygen haemoglobin interaction issues potential.
Yeah, that's a great question, Jim and maybe we ultimately you have to do the clinical work right to figure this out but one of the reasons that we do all of this toxicology work before we go into humans.
Is to try to get an understanding or what are the likely.
Consequences and risk of the drugs that you can look for that when you're actually doing your clinical study. So we've done an extensive amount of work Preclinically, Iran.
And the truth is it's very well tolerated.
We actually modify 100 per cent of hemoglobin and it does not hurt animals.
So will that'd be true in humans.
A proactive rather than a reactive approach to treatment.
Is the possibility of preserving Oregon function and so the younger that we can get into age groups faster I think we'll be able to look at preservation of Oregon function and the spleen as you know is one of the.
Oregon's where we see dysfunction relatively early in life, which is why so many children need to be placed on penicillin early in life. So I agree with you that we may be able to really look at Oregon preservation and the spleen is an optimal oregon to start with.
The next question is from Gregory Renzo from RBC capital markets cause go ahead.
[noise] parameters, we look at our industrywide, new to brand Uhm metrics, which is basically new prescription drugs in the United States and if you look at the kind of industry audits on that it. It's it's well below prepandemic level still in Q3 was not was worse and Q2.
And then the other thing we look at it as a sickle cell disease specific audit that we do we look at the the claims databases for sickle cell patients and that's where we get insights into are they actually seeing their health care providers at the same rate. They were and that is still below prepandemic levels and got a little bit worse than Q3 because of the surge. So we're hopeful.
In queue for things will start to turn around we look at that those audits once once a quarter. So we'll look at it again in queue for but in Q3 that was Ah Ah clear headwind for us.
The next question is from Matthew Harrison from Morgan Stanley. Please go ahead.
I would really educate people to focus on the modification more than anything else. Now. We're also gonna be doing some other studies, which are probably more correlated with modification like the locker analysis as well and we'll proceed presenting all of that data as well.
The next question is from Monica American Janie from Evercore. Please go ahead.
Okay. Thanks.
The question I, just Don fix that one as well and the data that we saw this morning, what what was the time point that occupancy measure that just wondering on the P. K as in Dragon.
That could be the peak level of occupancy even after doctor thing and and then thinking to us what kind of the highest the rest of it.
You should expect to see and then in the non cohort at Ash and would you consider going.
200, Meg 50 occupancy levels aren't consistently above 30% that at that does.
So Brian you can take that <unk>.
The the first part we're we're talking about occupancy at the peak.
So it's just because it's a single dose what you expect with any drug that has a long half life. After multiple days of dosing the C. Max to the see men actually are very similar.
Sure the highest decile physicians and other specialists have written prescriptions for ox Friday, so they still have opportunity to grow into their second group of patients but.
But overall some of the mid tier and lower tier physicians only about a third of them have written a prescription for ox Bryan again. This speaks to access to their physicians ability to educate their or excuse me access to patients and the ability to educate patients during the pandemic. So we have great opportunity in both of those those those areas.
Pediatric hematologist.
Have only written occupied and to the extent that they have experienced in 12 and older a lot of pediatricians have a lot of kids between 411 so.
They tell us that they've got opportunity.
To write it for for those kids that they've been really holding onto so.
It's an opportunity for us soon after launch.
The next question is from Richard background from Cowen. Please go ahead.
Good evening guys. Thanks for taking the question.
I wanted to ask you about the comment that you made about the one constituting a functional cure in a pill I know you want us to focus on occupancy, but I'm also thinking about the other.
Io markers that were presented pretty early on in novato toward development.
<unk>.
For this mechanism what sort of hemoglobin change.
What's sort of Elliot.
H change bilirubin change could constitute reduced organ damage.
And other improved outcomes above and beyond just the Ocs and I'd love to ask.
Dr. Smith Whitley that question too.
Okay.
As it has with with with Ox Friday predicts the hemoglobin and other outcomes going in the direction that you'd like.
So Kim you feel free to correct me.
I just wanted to add that it's a very difficult population of really understanding study. When you look at individuals who have gone through curative therapies.
You don't even achieve normal hemoglobin values, and sometimes even not even normal LDH values, depending on the degree of mix kind of rhythm, but you have individuals who become symptom free.
I think that this is something that we'll continue to learn more about as we studied 601 and go through the kind of clinical trials.
But even with a curative therapies, we really haven't been able to find them consistency.
With what hemoglobin and other Biomarkers are mean in relation to cure.
The next question is for me I didn't so merger from Guggenheim Partners. Please go ahead.
Yeah.
Hey, guys. Let me ask you a question.
On the upcoming approval at our upcoming could do for them from kids I think you addressed it a little bit but can you just talk about the goal from a reimbursement perspective I'll quickly reimbursement could be achieved and then you know your view in terms of uptick in kids versus adults and then correct me if I'm wrong I think this approach.
At about 17000 patients to the label didn't can you just talk about the number of exactly how much how many new patients will be added things.
That's great. Thanks for the question, Yeah, now I'll ask DJ to address those okay, yeah yacht and so from a payer perspective. The good news is it's because we've already got approval for ox brighter for the 12 and older. The majority of states will consider this a line extension and because it's in pediatric patients where they clearly recognize the high unmet need.
<unk>.
We expect the the payer discussions to go even faster than it went with the 12 and older Remember we gave guidance in New York spread original launch of one year to get broad coverage and we were able to accomplish that in in three quarters. We expect this to be a bit faster than that.
You know exact dates are I can't give you it will take a process still you know you have to introduce the product to them. It does have to go through some process to get reviewed.
Reviewed even as a as a line extension to get on their formulary.
You'd expect from repeat dosing and and do you have available to you like what what the your experience was with X brighter when you dose a single time in healthy volunteers like what hemoglobin occupancy you achieved and then how that translated into patients with multi dosing.
Hi been this is Ted Uhm, we actually did send out some information I think we may even have that on our website. So you can go onto the website and look at that comparison.
About <unk> six of Barrick.
So there was a pretty big difference.
But if you look at the individual patients you're going to see Theres a lot of overlap.
And so if you just randomly pick six patients you may not see much of a difference even though we know there's quite a big difference between 905 hundred.
Yes.
The next question is from John Newman from Canaccord. Please go ahead.
John Your line is open.
We can't hear anything similar sort of on.
The next question is from and dress or <unk> from Wedbush Securities. Please go ahead.
Good evening and thanks for taking our questions.
Before I start just a quick Oh on software.
Our condolences on the loss of your friends I'm, sorry to hear about that.
I've written a prescription for <unk>.
Know that our highest prescribers like the top 50 doctors are writing it for 75% of their patients. So we know what it looks like and now it's just about continuing the education and getting patient access to their health care providers. So that we can increase those rates.
The next question is from Yanan, Zhu from Wells Fargo Securities. Please go ahead.
Thanks for taking my questions.
<unk> you mentioned.
50%, no sequelae and global been in Kuwait individuals and in the gene therapy gene editing studies.
I think the non cyclical.
Cyclic globin level is 45% it was pretty consistent between different studies.
Question is.
Is 45%.
Kind of a.
Receptor occupancy that that can give a lot of confidence in our <unk>.
Oc free.
And the other a functional cure measures.
Is that the right way to think about it.
And related and also importantly, do you think with the current study.
Do you are you confident that.
That 45% level, it's something that is it is within the reach of the study.
Yes, it's a good question yeah.
Yeah actually.
Anti inflammatory crap.
So we wouldn't anticipate.
In a significant anti inflammatory effect that.
So we do expect that with our therapy, there will be a delay.
When initiating therapy and the resolution of inflammation and the symptoms and outcomes associated with inflammation can be one of the things about ablative therapy is it is highly immuno suppressive in fact, that's what you're doing you're completely wiping out the hematologic.
It can in Insignificantly the immune system.
When you do these food so you do get a profound and immediate anti inflammatory effect, which we will not bring.
With with our therapies of course, but overtime.
As we saw in the hope study, we would expect information to recede and Vlccs to decline.
Okay.
This concludes today's question and answer session I would like to turn the floor back over to Ted Love for closing comments.
In closing I'd, just like to thank everyone for joining the call today.
I also want to remind you that we look forward to seeing many of you at our R&D day next week as well as seeing many of you virtually in the meantime, I want you all to stay safe healthy and please feel free to reach out if there are any additional questions.
This concludes today's conference call you may disconnect. Your lines at this time. Thank you for your participation.
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