Q2 2021 Global Blood Therapeutics Inc Earnings Call
[music].
Greetings and welcome to 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, if anyone should 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 Sir.
Thank you and welcome to <unk> conference call to discuss the company's financial results for the second quarter 2021 and to provide a business update.
I'm, Steven <unk> <unk> head of communications and Investor Relations joining me on the call are Dr. Ted Love, our President and CEO will provide an update on our progress in the second quarter.
Jeff Farrow, our Chief Financial Officer will review our financial results.
David Johnson or DJ our Chief commercial officer will give an update on the ox bridal launch.
Dr. Kim Smith, Whitley, our executive Vice President and head of R&D will discuss our pipeline.
Ted will then give a few closing remarks from the catalyst for <unk> future.
Earlier. This afternoon, we issued a press release announcing gbt's progress and financial results for the second quarter ended June 32021.
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 of our SEC filings and press releases can be obtained from the investors page of our company website at GBT Dot com.
Forward looking statements made on this call are only as of the time. They are made you should not place undue reliance on such statements future events or simply the passage of time may cause our beliefs to change and we disclaim any obligation to update any forward looking statements other than as required by law.
That I will turn the call over to Ted.
Thank you Steven and good afternoon, everyone.
In the second quarter GBT made solid progress on our mission to transform the care of sickle cell disease and improve the lives of patients around the world.
The fundamentals of the ox for out of U S launch remains strong.
And GBT is setting itself up for long term success by advancing our regulatory strategies and our robust pipeline.
I want to highlight that just recently, we announced the initiation of 2 pivotal phase III clinical trials for <unk>.
P Selectin inhibitor for.
For visa Occlusive crisis in S. E D that we think will be best in class.
We also initiated our phase 1 trial of <unk> 601 in sickle cell patients.
We remain on track with our goal of delivering proof of concept data for 601 by the end of the year.
We're very excited by these developments and our potential to bring further therapeutic innovation to sickle cell disease.
Now turning talks Friday.
We delivered approximately 925, new prescriptions in the quarter.
Consistent with the range, we've seen since the onset of the COVID-19 pandemic.
While we anticipated this resolves we are not satisfied with it.
Looking forward, we are encouraged by several key factors.
First the net number of ex credit patients continues to increase each quarter.
This is a key driver of delivering on our second quarter revenue expectations.
Second experience in the real world setting.
Tenuous to validate the role of ox Friday as potential foundational therapy for sickle cell disease.
A growing body of real world evidence demonstrates improvements in overall clinical status of patients and of key lab values.
And third our latest market research demonstrates high levels of satisfaction, among prescribers and patients who have tried ex Friday.
In addition, we are receiving positive feedback on our recently launched educational and marketing programs.
And we have several exciting initiatives planned for the rest of the year, including a groundbreaking direct to consumer advertising campaign.
In the second quarter. The COVID-19 environment began to show signs of gradual improvement for sickle cell patients.
As overall vaccination rates increase and restrictions begin to lift in many geographies.
For sickle cell patients sentiment towards the vaccine is improving.
However, black Americans still have the lowest rates of vaccination.
As we all know more recently the Delta Varian has begun to drive an increase in COVID-19 cases, and a return of restrictive guidelines.
COVID-19 continues to be a headwind for GBT and the sickle cell community along with a longstanding health inequalities experienced by our patients.
Too often sickle cell disease is characterized as a disease of pain.
When it is in fact, a disease of multi organ failure and premature death.
Patients are often told they are going well if they arent experiencing pain crises that we know these patients are suffering premature death.
2 hemolysis.
This is a meaningful opportunity to create change and we are not standing still.
We are focused on creating a greater sense of urgency among hcp's treating people with FCB.
Along with other significant gaps in the care of those living with this disease.
A few highlights of our actions include.
We are continuing to educate the health care community on the urgent needs of patients with sickle cell disease.
We formed a sickle cell disease health equity Council.
With advocacy leaders and other prominent members of the community.
This group is working to prioritize actions on systemic issues that impact access to care.
In addition, our apps XL Grant program provided approximately $450000 in funding this year to community based organizations and institutions for sustainable programs that improve access to high quality care for sickle cell patients.
<unk>.
And in September we will cohost, the 10th annual sickle cell disease Therapeutics conference for <unk>.
With the sickle cell disease Association of America, bringing together leaders in the community to discuss the latest advances in future trends in S. E T.
All of these efforts and more are designed to improve health equity for patients with sickle cell disease, including helping them access innovative medicines like <unk> Friday.
With that I will turn the call over to Jeff to review, our second quarter results.
Thank you Ted.
Total net revenue from sales of <unk> was $47.6 million for the second quarter of 2021 and.
An increase of $16.1 million for 51% year over year on a sequential basis second quarter revenue increased by 22% from the first quarter.
The sequential growth was driven by the continued increase in the net number of patients on ex brighter income.
Leading demand from existing and new patients.
Days of inventory on hand in the second quarter was relatively flat, although absolute levels of inventory increased reflecting the growing occupied a patient base.
Gross to net was flat from the first quarter at approximately 15%.
This reflects an increase in the mix of $3.40, B sales offset by a decrease in patient co pay support following the reset of commercial insurance out of pocket deductibles in the first quarter.
We are closely tracking new COVID-19 case rates being driven by the Delta Varian, particularly in geographies that include the most patients with sickle cell disease.
Given the ongoing headwind, we want to provide specific expectations.
For the third quarter, we expect revenue of $52 million to $54 million, which anticipates relatively flat new prescriptions gross to net and inventory dynamics and.
And reflects the current uncertainty around COVID-19.
As the impact of 2 major holidays in the third quarter.
Looking forward, we continue to anticipate that if the pandemic gradually improves we will see a corresponding improvement in new prescriptions and revenue growth.
Now turning to expenses.
Cost of sales for the second quarter was 748000 as compared with 377000 for the second 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 manufacturing costs related to <unk> sales were incurred prior to FDA approval and thus were recorded as R&D expense.
R&D expense for the second quarter of 2021 was $52 million compared with 34 million for the same period in 2020.
The increase in R&D expense in the second quarter was primarily due to costs related to the advancement of our preclinical programs, along with ox Friday and o'clock am at clinical programs.
We continue to anticipate a sequential increase in R&D expense in the third quarter.
As we begin enrollment in the 2 phase III studies for <unk>.
Which will trigger a $5 million milestone payment by GBT.
We also expect an incremental increase in the fourth quarter driven by the clock from that program. The advancement of GBP 601 studies and our other expected pipeline related activities.
SG&A for the second quarter of 2021 was $61 million compared with 49 million for the same period in 2020 day.
The increase in SG&A expense was primarily due to increased employee related costs, including noncash stock compensation and other professional and consulting services associated with the commercialization efforts for <unk>.
We anticipate a stepwise increase in SG&A expense in the third and fourth quarters, driven by the rollout of <unk> per item materials, including our DTC advertising, our measured expansion into Europe, and the initiation of multiple investigator sponsored studies.
Net loss for the second quarter was $70 million compared to 53 million for the same period in 2020.
Basic and diluted net loss per share for the second quarter was $1.12 per share compared with 86 per share for the same period in 2020.
We continued to be well positioned with a strong balance sheet with cash cash equivalents and marketable securities of 437 million at quarter end.
<unk> with $561 million at December 31, 2020.
And with that I will now turn the call over to D. J.
Thank you, Jeff and good afternoon, everyone.
We delivered another quarter of solid progress and are excited about our recent launched and upcoming initiatives.
As I've done in prior quarters, I will provide an update around the 3 key metrics that will give you further insight into our progress. These metrics are new prescriptions for <unk> rider, which informs underlying patient demand.
For a health care providers prescribing ex brighter, which captures the progress we are making in adoption.
And payer coverage, which speaks to the access environment for Ox Friday.
First new prescriptions.
Were approximately 925, new prescriptions for <unk> during the quarter consistent with the range, we have seen since the onset of the pandemic.
While it appears that in person patient visits improved somewhat in Q2 overall visits inclusive of in person that telemedicine remain below pre pandemic levels and consistent with past the past several quarters.
Over time, we believe there is a significant potential for occupied a new prescription growth, particularly from deeper penetration with existing prescribers for.
For example from among our existing prescribers, we are around 30% penetrated into their patient population.
This compares to our top 50 occupied of prescribers, who have an average penetration of 75% derma.
Demonstrating ample opportunity for growth.
As a result, the priority of our team is continuing to engage with our top targets in the second quarter. We reached 75% of these health care providers, including via a growing number of in person interactions.
As Ted mentioned.
We launched a targeted direct to consumer marketing campaign at the end of July.
The centerpiece is a new TV commercial that features actual ox for Ida patients and their families. It highlights our spread of messages and serves as an empowering call to action for patients to engage with their health care providers.
The commercial will also be available on our Friday dot com and on social media.
This groundbreaking commercial is the first of its kind in sickle cell disease, and we believe it will have a positive impact on patients' awareness and adoption and help prompt patients to ask for <unk> by name.
We are also closely monitoring additional factors that influenced prescriptions with respect to COVID-19, vaccinations data from the CDC and Kaiser family Foundation highlight that the black population is vaccinated at a lower rate than Hispanic white or Asian populations.
Specific to those with sickle cell or market research from around 100 patients in May June showed 43% already received a dose and 17% plan to as soon as possible. This is a significant increase from the February March timeframe, which showed 10% dose and 31% planning to be.
For the remaining 40%.
Vaccine hesitancy remains prominent due to fears around safety and side effects as well as waiting for longer term data.
Turning to other initiatives, we are focused on providing support to help patients start and stay on therapy.
To improve the rate and speed of new prescription conversions. We are now partnering with cover my Meds, which provides an integrated platform for health care providers to submit prior authorization forms electronically to payers.
We believe this will be easier and more efficient for health care providers.
Ex Friday adherence, which includes both compliance and persistence with stable and well within the range of our analogs during the quarter. In addition, a significant number of patients that discontinued therapy are restarting on ox Friday.
We have received positive feedback and are on our recently launched initiatives to support adherence and are optimistic that they will lead to further improvement.
These initiatives include our getting started guidance and brochures, new patient kits GBT source dot com website, and our transition to a 100% high touch model for patients accessing our patient hub GBT source.
Our market research also continues to support the strong fundamentals of ox writer.
Nearly all patients report that they experienced some form of symptom improvement and are likely to recommend it to others.
And for the majority of health care providers, we're aware of Ox Friday.
Already our plan to prescribe it in the future.
Looking at ex Friday use we continue to see a broad range of characteristics, such as 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.
Care provider penetration.
During the quarter total interactions with health care providers remained steady compared to the first quarter with the percentage of in person interactions incrementally increasing each month, but still substantially lower than pre pandemic levels.
In addition, hospitals and institutions remain cautious around in person visits.
Against this backdrop, we added about 140 prescribers in the quarter, bringing our total prescribers to around 1700 since launch.
When we look at the breakdown of writers, we continue to see prescriptions being written by both specialists and non specialist, which we believe is a positive trend for the long term trajectory of the launch.
Turning to payer coverage.
In 2020, we achieve 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.
<unk> been pleased to see expanded utilization of our copay card program in the first half of 2021.
After streamlining access for patients that worked through our specialty pharma partners.
As a result, we have been helping more patients than ever before.
And a large majority of our patients.
Across all payer types have a co pay of $10 or less.
Having achieved almost 100% coverage in Medicaid.
We are now focused on making it easier for physicians to prescribe and patients to receive ox Friday.
For example.
Similar to our success in Texas, Michigan has just added ox for Ida to the common formulary, which all managed Medicaid must follow this provides for open access to ex Friday to label as of August 1st.
This was an important win that improves our ability to get patients on therapy and speaks to the growing confidence in our Friday.
In summary, we are making good progress on our strategic initiatives and have a focused plan to drive adoption. We continue to receive excellent feedback in the field and are eager to expand access and get more patients on ox Friday.
And with that I will now turn the call over to Kim to talk about the exciting developments in our pipeline.
Thank you D J and good afternoon, everyone.
I wanted to pick up on a point Ted made earlier and provide my perspective as a clinician with more than 30 years of experience in this disease.
It's well known that sickle cell patients haven't had reliable access to high quality care and their health outcomes have suffered as a result.
Also know sickle cell patients have chronic anemia, and hemolysis, which lead to long term organ damage and eventually debt.
We have the tools and knowledge to transform the treatment of this disease.
However theory real gaps for me.
As 1 of the authors of the National strategic plan on sickle cell disease published last year at the National Academy of Sciences Engineering, and Medicine I can tell you that the needs in the care of sickle cell disease are profound and not insurmountable.
I am optimistic.
There is good momentum in part fueled by innovation like ex Brighthouse.
In addition to educating health professionals on treating sickle cell disease and its underlying cause.
1 of the key drivers is to gather and publish more real world evidence.
A very important step we took this past quarter was the initiation of 2 large multicenter registry retro and prospect.
These studies will enable a deeper understanding of ex brightest long term efficacy and safety and provide additional real world evidence that we believe will support the use of ex brighter from sickle cell disease patient.
Retro is a retrospective registry collecting real world outcomes and up to 300 adults and adolescence at approximately 10 U S sites.
It will capture clinical outcome measures health resource utilization data and laboratory measures based on medical records, 1 year pre and post the initiation of <unk> therapy.
Initial data from the first 20 retro patients were presented at the <unk> meeting in June.
The results were in line with the Phase III <unk> study.
It's 50% of patients achieving an increase in hemoglobin of greater than 1 gram per deciliter.
We anticipate presenting updated results from the retro registry at the Ash meeting in December with full results sometime in 2022.
Prospect or other registry is prospective complete enrolling up to 750 patients at approximately 25 U S sites.
It will capture the same measures that bright trial, but for.
For a period spanning from 1 year pre and after 5 years post the initiation of ex spread of therapy.
Anticipate results from the prospective registry will begin to be presented in 2022.
We also continue to see additional real world evidence generated by the health care provider community.
At the <unk> meeting Dr. Allen Anderson presented an update to the data. He first presented at ESMO, showing 77 patients treated with ex Friday that overall hemoglobin levels increased by an average 2 grams per deciliter with corresponding improvements in markers of hemolysis exceeding results.
Other hope study.
Turning to the pipeline for very encourage by our progress and we remain on track with our lead programs.
From Clacker map R. P. Selectin inhibitor for reduction of ESC has the potential to be dose quarterly as opposed to monthly dosing with the current option.
This would be a meaningful improvement for patients and is in line with typical sickle cell disease practice schedule of quarterly check in.
We initiated 2 phase III studies collectively named thrive.
1 aims to reduce poc's over 48 week treatment period.
A second study aims to reduce 90 day POC readmission following an initial vse hospital hospitalization, which.
Which tragically occurring at around 50% of patients.
Subject to the results of these trials.
Plan to file 2 independent regulatory submission for separate indications.
For GBP 600 line, our next generation hemoglobin polymerization inhibitor, we completed a healthy volunteer study and have expanded into treating people with sickle cell disease to assess the safety Tolerability and PK P. D. At 601 over a 14 week period.
Our plan is to present data from these phase 1 study at the Ash meeting in December.
If the data shows hemoglobin modification in the 30% plus range similar to the preclinical data. We believe 6 sales line could be a best in class therapy.
And what's really exciting for patients is that this level of efficacy has potential to eliminate all symptoms and long term organ damage, providing a functional cure in a once daily pill.
Outside of our 2 lead programs, we continue to advance our in house and in licensed preclinical programs with a robust pipeline. We believe we have great potential to transform the treatment of sickle cell disease with multiple complementary treatment options.
Turning now to our other R&D related initiatives in the United States, we submitted our regulatory applications seeking to expand the <unk> private label to include children age 4 to 11.
These include a supplemental NDA for the expanded label a separate NDA for our new pediatric formulation.
Our F&B a included data from the Phase Iia Hope Kids, 1 study, which was presented in an oral presentation at the <unk> meeting in June.
Data from 45 children with consistent with the hope study.
No new adverse safety signals for detecting.
We are building additional clinician experience with ex Friday in the U S. A.
A pediatric early access protocol launched in January which provides ex prior to 2 children age 4 to 11 years old.
Based on strong interest we are upsizing the protocol from 50 to up to 150 patients.
As I said earlier it is vital that we treat sickle cell disease patients more proactively and.
I think the potential expansion of ex Friday to children as young as for years old is a great opportunity to address this need.
From the provider perspective, many pediatric practitioners myself included are eager to have another treatment option, particularly if it has the potential to mitigate red blood cell signaling and destruction, which can modify the course of their disease and alleviate future serious and life threatening complications.
Analog suggest adherence in this age group will likely be high similar.
Similar to the adoption of Hydroxyurea, we believe that robust adoption among pediatric health care providers could be an important accelerator with adult providers.
Turning to Europe, we remain on schedule with the Emas review of a marketing application, which we continue to believe it could be approved in the first half of 2022.
In advance of potential approval, we are building momentum with early access available in France, Germany, and other countries and we received a promising innovative medicine designation for ex brighter in the United Kingdom.
Outside of Europe. We are also working to make <unk> available in the Middle East and Latin America.
Altogether our label in global expansion plans are intended to give us the opportunity beach more than 350000 sickle cell patients around the world over the next several years.
And as we make progress against this goal we will continue to explore strategies to bring our therapies to patients and limited resource geographies, such as Africa and India.
And with that I will.
Turn it back over to Ted.
Thank you Kim.
I'm really excited about the development we've outlined today.
And our opportunity to reach more patients and.
In closing GBT continues its leadership in sickle cell disease, and it's well positioned for long term success.
We have a series of important catalyst catalyst Inc.
Dissipated over the next 12 months.
For example, <unk>.
Tim outlined we expect our momentum will be augmented with 2 potential approvals in the first half of 2022.
In the U S. Among children as young as for years old and in Europe in patients over 12 years of age.
Our pipeline is robust and advancing.
We look forward to reported early results on GBP 601 later this year.
And we're excited to move forward within Clacker Mab as a potential best in class option.
As we go forward Gpt's commitment to supporting the sickle cell community is stronger than ever as we strive to improve the lives of our patients.
Our team is unified in this effort.
And I want to thank employees for their passion and dedication, which is essential to achieving our goal.
Transforming sickle cell disease into a well managed condition.
With that we'd like to open the call for questions.
Operator.
Thank you and he would like to ask a question. Please press star 1 on your telephone keypad, a confirmation tone will indicate your line is in the question queue. You May press star 2 if he would like to remove your question from the queue and for participants using speaker equipment. It may be necessary to pick up your handset before pressing the star.
He is.
Our first question is from Mark Breidenbach with Oppenheimer. Please proceed.
Hey, guys congrats on the quarter and thanks for taking my questions.
These might be both kind of directed Kim.
I was hoping you could help us understand a little bit.
Why there is the need for both the <unk> NDA and the NDA for the pediatric label expansions to what degree are these redundant applications and who would be served by the EMEA versus the us NDA.
And the second question is just on the sizing of.
And quite a few lab trials.
Thinking back to the sustain trial of Crystal isn't.
I believe that was only just under 200 patients and it sounds like you're targeting larger trials.
For for it and collect them.
240 patients for 1 and then I'll take 280 patients for the other can you tell us anything about your powering assumptions and should we be reading into the differences in trial sizes is maybe an indication of expectations about the relative efficacy of those 2 antibodies.
Thanks for taking the questions.
Thanks, Mark and Tim feel free to add on Afghan, but I'll take a stab at the 2 questions and feel free to add on so mark for good to hear your voice.
Thanks for your question.
The reason that there is an F N D. A is because the clinical information is actually an update to the approved.
Labels. So that's an F N b a day.
<unk>, new formulation has to be on.
New NDA on its own so that's why we have an NDA and that from day 1 is for the clinical.
New NDA as for the new formulation for the pediatric formulation.
With regard to the trial size, we don't tend to give out the details.
How our studies are powered but we do stress that we generally have a significant power in our studies, we tend to if anything overpower studies rather than underpowered them.
And Youre right. Our study is a little bit larger I believe the <unk> study was designed to be.
Jeff just north of 200 subjects.
Did have dropout sales. So you may be looking at the final numbers being around 200 patients I believe they did enroll more than I don't remember the exact numbers, but.
But our study is likely 240 patients.
Our steady.
For hospital readmission.
Actually a different endpoint is powered around hospital readmission, which we expect to control rate to be around 50%.
And again, we are well powered.
To look at.
Even let's say a 30% reduction in a 50% rate, but we don't talk too much about the details but bottom.
Bottom line is we are we tend to be heavily power from that study.
Kevin feel free to add to that if you if you'd like.
No Ted that was great. Thank you.
Alright, thank you.
Our next question is from at the a young with Cantor. Please proceed.
For taking my question.
Nice quarter.
I guess 1 is just you know in light of what's happened is delta variant and all the dynamics of vaccinations in underserved populations like how confident are you that.
You can achieve a potential inflection in new scripts I know, it's something that you had talked about.
Kind of line I know it sounds like in the third quarter, it's probably will be flat, but I just wanted to kind of see if I could get some more granularity on the fourth quarter and then my other question is just.
For the DTC campaign like what have you gleaned from now all of that and what makes you want to do it now versus maybe initially upfront. Thank you.
Great. Thanks for late to get to hear your voice.
Jay you may want to talk more specifically, but I would say debt.
With regard to an inflection you're right.
We have been hoping that restrictions we're going to liberalize.
Liberalize.
And infections were going to.
Greatly diminished Unfortunately, as you point out with the Delta variant, we've kind of been heading in the wrong direction.
Profoundly in the wrong direction and some of the states, where we have a lot of patients.
But the truth is we can't control the Covid.
Environment, we can control what we're doing so we continue to push on all of the things that we can push on.
Both with our our organization as well as our educational materials, our patient support.
We are very excited about the DTC. The reason that we couldn't do that initially is because everything that we do has to be FDA approved.
It takes some time to get.
Commercial's Aruba D J.
Please feel free to add more about some other things that youre doing particularly the DCC.
Yes, Thanks, Ted and thanks for leap yet that's exactly right DTC campaign is always something that we had in our plan.
We started with a very holistic approach that included websites that we've had there've been updated with the new campaign is well social media and digital advertising and then always part of that campaign was then to add a more robust.
Commercial as well, but it does take time to produce those it does take time to get it through the FDA and we wanted to educate the market a bit on ox Friday before we before we started that commercial so everything is kind of lined up for us now.
And Ted right I think I think this headwind that's kind of not unique to JBT, but is unique to the industry.
This pandemic, it's what youre doing about it to set yourself up for long term success going forward and we're doing a lot. We're doing a lot on the adherence campaigns for doing a lot with cover my meds to help physicians a process and start patients more efficiently and now of course, we're activating and educating the patients directly. So we think all of these things help us up our help us.
Out for that that future growth that we that we fully expect.
Okay, great. Thank you.
Yeah.
Our next question is from Andrew Berens with CEB Leerink. Please proceed.
Hi, Thanks, Let me also add my congratulations on the strong sales this quarter guys.
Thank you, we can get to share with them.
And getting some anecdotal feedback from physicians, who are using the drug in patients. Thus fail hydroxyurea irrespective of the hemoglobin level. So I was wondering if you know what percentage of patients getting the drug for getting it for reasons other than to increase hemoglobin and.
Then.
Also what percentage of patients could I ask spread are also getting a dock.
And then lastly, I might have missed it during the prepared comments, but did you update the persistence rate are you seeing any increase in patients that returned a therapy after stopping.
For our DJ will definitely want to add to some of those but I would say to 1 of your questions.
Yes, there are physicians Pat start patients on a Friday for reasons other than hemoglobin for example pay.
Patients can have.
Relatively higher hemoglobin, but have leg ulcers.
Net are very difficult to have I know anecdotally.
There have been.
Some encouraging findings along that.
The label for Ox Friday actually doesn't have a hemoglobin.
Hemoglobin indication because fundamentally what the drug is intended to do is to treat the underlying base of your disease and per prevent that from advanced including your anemia. So in theory, we probably will want to see the future of this therapy being indicated.
<unk> indicated for children, when they're very young before their hemoglobin dropped but you know they are going to drop and prevent them from dropping that that's really where we want to go long term, but I would say anecdotally probably 1 of them are common reasons to begin it now even with a relatively higher hemoglobin.
He is to treat things like light colors, but also maintain health in patients that are going doing well because we know in the future. They are unlikely to continue to do well for <unk>.
You did.
Yeah, just to just a couple of additional comments.
Yes, so a lot of our credit patients start and believe it or not in sickle cell disease, such a devastating disease are actually treatment naive they've never been on treatment.
Which is kind of amazing.
So we get patients that have been on previous therapies or failed hydroxyurea or couldn't tolerate it as well as patients that we literally have not been on any treatment at all interestingly a little over half of our patients are on ox Friday alone, but that means that a little under half of our patients are actually in a combination.
<unk> regimen and that includes Hydroxyurea very commonly as our data would support but also.
<unk> as well.
And so it fluctuates quarter to quarter, but approximately in the low teens low double digits are on our combination with <unk> and we expect that to go up over time as people get more and more experience with the newer products.
I will say that persistency rate.
What we talked about is that our overall adherence rate has not.
Fluctuated Q2 has been consistent with other quarters and as well within that range of of analogs that we look at so nothing to report there and then I think you asked a little bit about restarts and restarts continue to happen that's been a really nice phenomenon.
<unk> said in previous quarters about 20% of patients discontinue therapy, ultimately restart and we have seen that trend continue in Q2 as well.
Okay. Thanks for the color I appreciate it congrats again.
Thank you.
Our next question is from Elmer Perez with Roth Capital Partners. Please proceed.
Yes, good afternoon, everyone.
What I'd like to ask.
From the team is that we observed debt do you have beaten.
And that for you every quarter since launch do we have a an estimation of hump, but what is the discrepancy between the number of patients treated.
With the 2 drugs.
Hi, Omar.
It's a good question, it's a little bit hard for us to estimate obviously, because we don't have access to Novartis is data and it's also a little bit complicated because when patients.
Again on a debt on our back for you how they actually get a loading dose. So there are 2 doses on the front end, so it's a little bit hard for us to estimate accurately.
As you know I personally am very excited that both drugs do very well and patients get access to.
These cutting edge therapy. So I think their launch is going well certainly doing compared to Covid I think we're doing well as well, but looking at their numbers and our numbers, it's not something that we put a lot of attention on.
Okay.
Also state Ted that they are approved in 40 for different countries.
How do you compare in that regard.
We're approved in 1 country.
It's a pretty it's a pretty easy comparison.
We are as you know however.
Not a company that has an infrastructure of the magnitude of Novartis.
The truth is the biggest market.
Are the United States.
Europe, and we've already filed in Europe.
There is a significant market in the GCC and I think you know we have distributorship relationships, there already and Latin America, and we're working on there so the debt the likely market we are.
Either already filed in.
Or moving towards application and may not be 40, and the yen, but our numbers will be increasing significantly, particularly around the size of the market.
You still get them bid 1 country versus for the 44.
And 1 last question is.
Maybe Jeff if you could help me out here. So it appears that in this quarter.
For your new patients than in the previous 2 or 3 quarters.
Yet the revenue is much higher.
Is it reconcilable by the fact that you have.
Building stack of existing patients and or the appearance is improving.
For the drug or discontinuation rates are declining.
Yeah, No. That's a great question Elmer it really is to the to the middle point that you highlighted there. It's the growing base of patients that continue to build over time and as a launch continues the bulk of the prescriptions and ultimately the bulk of the revenues comes from those patients and so that's where we're seeing a lot.
Of that growth come from patients that initiated maybe in Q1, where we only had 1 or 2 models, we get the benefit of free models in the second quarter. So that's typically how that runs.
Okay. Okay. Thank you so much.
<unk>.
Our next question is from Ritu <unk> with Cowen and company. Please proceed.
Good afternoon, guys. Thanks for taking the question.
Wanted to drill down a little further on various patient starts.
D J.
What was the motivation for these patients to restart have you been able for us on why they quit on the first place.
And my follow up was about the EHR Anderson dataset I'm for 1 that generated higher.
Hemoglobin increases for the phase III data.
What was it about.
That patient profile.
That may have driven the increase in hemoglobin.
Response there.
So DJ why don't you start and then maybe Kim you can follow up on Alan's data.
Sure Yeah, Yeah Ritu the great question on the restart so we did do some surveys with patients who discontinued and restarted ox brighter.
And so the reasons or perhaps what you might expect.
Some patients stopped because of early side effects.
And they decided that they wanted to give it another chance mainly because theres been a lot more education.
Really focused on making sure physicians and patients understand that if you get an early gi upset or or headache debt. There's things that can be done right theres dose adjustments that are made and so patients are.
Oftentimes reporting bad debt they are eager to restart under the under a protocol that may help them get through and avoid those side effects. So that's been that's been great to hear there've been just other life factors that people report things like.
Hey, I move Hey, I started on the new job for I lost my job.
And things like that which are other reasons patients stopped but didn't necessarily want to stay stop and always felt that they would come back talks Friday and so they've now had a chance to stabilize and restart their therapy. So those are the main things we hear back cash.
Jim.
And for the Allen Anderson study, yes that increase in hemoglobin and the proportion of individuals' that achieved 1 gram per deciliter in higher rate was quite impressive and I think that there may be a couple of factors at play given that at 77 days.
It's still a small sample size, even though it's a nice sample size for a single center, we know the Doctor Amazon does a great job at each of total is.
Young people with sickle cell disease before they start ups brighter.
It may be more inclined to discuss whatever side effects. They may have going forward and managing the side effects more proactively and so there might be.
Increase in adherence in that correct.
The other thing is that.
His.
Individuals that were also.
Unstable doses of Hydroxyurea was at a higher proportion going in and that May have had a small effect as well.
Got it and a very quick housekeeping follow up question, given the importance of being.
The sickle cell population in London, and that were post Brexit how should we be thinking about approval in the U K is it still cover EMEA or is there a separate MH or a path we have to pursue now I have no idea for anymore.
Yeah. It's a good question Ritu, yes, there is a relationship in place between the EMA.
And the U K debt will essentially have the approval would be driven off of the EMEA work. So that that will continue I don't know the timeline for how long that will continue to be the b to b to plan, but that's the.
Temporary planning will fall into that time window.
Yeah.
Our next question is from Tim then Hu with Wells Fargo Advisors. Please proceed.
Hi, good afternoon, thanks for taking my questions.
I have 2.2 on the OXXO bright at launch and 1.
601.
For the launch.
Could you talk about the distributions for the new patients in the second quarter in terms of how they did.
Distribute over the course of the quarter.
And whether there are more patients in the beginning or it's more evenly distributed.
And could you provide a little bit color.
The July launch trend.
My second question on the <unk>.
With regard to vaccination and what can we expect.
In relationship to launch.
You mentioned, 40% of patients the kind of a at least partially.
Vaccinated for that rate is although not very high but I think it's enough to probably make.
The difference in the launch trajectory.
Although for those patients are fully.
Vaccinated.
The next quarter or so.
Do you think there are other.
<unk> items in addition to vaccination status that might impair.
Impact to whether they are willing to inc.
Wrapped with.
Your doctor and get a being treated with any new drug.
I have a follow up politics for 1.
Okay did you I think quite a few questions. There for you sure Yeah, no I'm happy to answer those so regarding the distribution of new patients over the course of the quarter. It was pretty even if it was pretty consistent throughout the quarter.
A month there July obviously, we have we are not reporting here on Q3.
Just to remind you July.
1 of those holidays of the 2 holidays in the quarter 1 of the major ones is is in July and so as with all the launches I've ever worked on fourth of July week is always a down week for volume and we saw that same dynamic.
Around the holiday so that gives you a little color on July vaccination expectations and impact on launch so yeah. It absolutely helps that we saw a significant increase vaccination rates in the sickle cell population. We're super excited about that and we think that that does and will translate into patients selling more confident to go in that.
That said.
All of the reports going on right now around the Delta variant being infectious to people with vaccinations is gonna be a concern for our patients right. They are still not going to want to be infected so because of the CDC guidelines around the impact it can have on sickle cell patients in particular, so we're monitoring this very closely to determine.
How are patients are feeling about it we have seen masking requirements go up over the last 24 to 48 hours in key markets a third of the new infections I just read today are in 2 of our major States, Florida, and Texas together make up a third of all new infections in the country right now those are the number 2 in <unk>.
<unk> 3 largest sickle cell states in the country. So these are the things we are monitoring very closely.
I said earlier, we have a lot of initiatives that are rolling out have already rolled out in the first half for this year, but even more in the second half with the commercial activating patients empowering patients and then also with physicians cover my meds and other tactics that we think will have a very positive impact as patients become vaccinated, but we're going to have to mom.
For this delta virus closely and I think better understand the impact it will have on our patients.
Right, Yeah got it that makes a lot of sense.
6 O..1 since you have now completed the healthy volunteer portion of this call.
Pete.
It's.
I'm wondering.
From that study.
You have a sense of the hemoglobin modification.
That you could achieve.
And also what is that.
What is for the sickle cell.
Disease patient part what is the dosing strategy.
<unk>.
Relationship, 2% hemoglobin modification are you trying to achieve.
For some future that is even higher than 30% when you design the doses. Thank you.
Okay.
Tim do you want to take that.
Yes, so I think that the first part of or actually I'll answer the last part first so when we are looking at a healthy volunteer study and the information that comes out of the healthy Volunteer study, we'll have good safety data.
And when you look at the 6 up to 6 individuals with sickle cell disease will have good PK PD data as well as some of the red cell parameters that we hope to share with you at the Ash meeting later this year I think that it's really too early to see.
Hey, what we would expect about exact dosing, but we're hoping that these preliminary studies will provide more information for that.
Yeah.
And then the other thing I can add is that the early human volunteer data have encouraged us that the dose to get to these target levels for modification of 30 plus percent.
It looks like it may be in the 100 day 200 milligrams per day in the sickle cell patients. So obviously, we need to document that and as Kim said that the day exact data that we want to present at ash, but the early data from from.
From a human volunteers as supportive for that.
Our next question is from Jason <unk> with Bank of America. Please proceed.
Hi, Thanks for taking the question. This is Perry on the line for Jason.
Just a question on the.
Each for 11 age group for our expert.
And.
If this patient population is approved I guess, how should we think about the subgroup.
In terms of a ramp.
In terms of sales.
Should we should we expect it to be faster and.
In the context of.
Potential increase in here adherence in this patient population.
You said you wanted to tackle that.
Sure happy to comment on that so yeah, we expect this day.
To ramp nicely in this population, we think it's a really important.
A group of patients for a lot of reasons, namely the mechanism of action would support that you want to start on Friday as soon as possible to prevent any downstream damage. So really important for the patients tell.
This is 17000 patients in the United States that are between 4 and 11 years old. So it's a large portion of the market.
And we've been calling on these customers. So pediatric hematologists are in our target list today and many of them have used ox brighter and their kids that are 12 and older. So they have experience and now they have even longer term safety and efficacy data at the time of the expanded approval. So for all those reasons. We think we'll get ahead of the game the per.
Parents will be seeing the commercial the parents will be asking for ox bright other parents will be advocating for their children and on top of that our data shows that the younger the population the better the adherence and in this population and Kim can probably speak better than I can about it as a pediatric hematologist. The parents are involved.
For the kids take the medication. So we would expect adherence to be at the highest level in this population.
No TJ I agree with that.
The 1 thing I would add Perry this is Jeff as debt.
There's quite a bit of excitement about this opportunity as well we had set up in our EAP program about for 50 people, we've actually blown through that already and have up the the number to 150. So I think there's quite a bit of excitement both on the pediatric hematology side as well as patients.
Our next question is from Paul Choi with Goldman Sachs. Please proceed.
Hi, everyone. Good afternoon, and let me also offer my congratulations on the quarter as well too from a place for.
First.
Thank you.
First I guess with regard to.
The commercial offer supply patients.
Could you maybe just comment on if there is any sort of dynamic among identified patients who may be waiting for.
Emerging clinical trial options such as your own.
For <unk>, <unk> or 6 to 1 or potentially competing assets and are perhaps deferring.
No.
Getting onto therapy for non occupied at that I had a follow up question on 601.
Sure.
You know I I hope not I mean.
If I understood your question, Paul they would be not.
Joining.
For the ranks of patients taking <unk> right to weigh down a new therapy that might be available in and in years down the road.
That would be tantamount to having high blood pressure.
Today, and saying I'm going to wait on the new drugs it doesn't.
Body is accumulating damage.
And your risk of a premier to GAAP is only increasing so.
And I think our education campaigns are directly.
Centered around that you don't want to wait.
On existing therapy, and if you'd bought down the road you were going to get a transplant. It would actually make a lot of sense for you to protect your body each day, while you're waiting on a transplant. So.
I think our educational efforts to really go after that would not be.
Strategically was weighted approach waiting on additional therapies.
Okay. Thanks.
Thanks for that Ted and then for my follow up question with regarding to 601.
I guess now that you are actively enrolling in.
Patients sickle cell patients here can you maybe help us think through how youre thinking about the duration of exposure of that will be potentially available for these patients that you had from pension.
Great.
Wanted to ask I'm, just sort of thoughts on how duration of treatment and.
And exposure share Mike.
Sort of be framed relative to what the initial results were presented for for a rock solid for thank you very much.
Sure Paul So I can't feel free to add on to this but the nice thing about.
Our you know quite voluminous experienced with ox Friday is that we know a lot about the mechanism of hemoglobin polymerization and 6 O..1 works through the same mechanism. So we think we can extrapolate a lot. The other thing that I would emphasize.
601 is that it is likely to have a very long half life.
Weeks in fact as opposed to days.
Ex spread to compensate for that we will be giving a loading dose. So the loading dose will very quickly get patients to a higher level of modification and then the daily dose would be essentially there to replace the drug that they metabolize each day and that's why.
That would be likely a very low dose. So we think in terms of exposure.
Just based on a Friday.
The hemoglobin rise occurs quickly in the hope study it was within 2 weeks of being on therapy.
And that hemoglobin rise is essentially maintained.
As long as for patients remain on therapy. So I think we would anticipate 601 to look very similar to that but obviously.
We will be getting the data with 601 and presenting it.
For everyone's view I'd ask but I think that's what we would expect based on the mechanism and Kim please add or correct for that.
No Ted I would agree and I just want to clarify.
For parts first question I think that the.
Incentive about pairing <unk> with and clock in Nab I just wanted to make sure that we are clear that individuals who will be participating in a clock on that trial should be allowed to be on a table Joseph ex bright annealing in so I hope that that would be a disincentive.
Okay.
Good day.
Operator are you there.
Courtney.
Kim can you hear us.
I can hear you.
But I think we're live.
Okay, let's see if.
We're still trying to get out we'll try to continue to say well you know greater on maybe I should just close yes.
Okay, well I, just I will close it looks like we may have bit of a Texas there I just want to thank.
Thank you everyone for joining our call today, we hope you all stay safe and healthy and please feel free to reach out to us. If you have any additional questions. Thank you again.
Yeah.