Q3 2022 Rhythm Pharmaceuticals Inc Earnings Call
Yeah.
The conference will begin shortly.
As Johan during Q&A, you can dial star one one.
[music].
Good day, and thank you for standing by and work with each of the rhythm Pharmaceuticals third quarter 2022 earnings conference call. At this time, all participants are in a listen only mode.
The speaker's presentation, there will be a question and answer session to ask a question during the session goes fresh start one one on your telephone.
Today's conference is being recorded I went out and the conference over to your Speaker today, Dave Connolly Investor Relations and corporate communications at Rhythm Pharmaceuticals. Please go ahead.
Thank you.
Good morning, everybody I'm, Dave Conway.
IR and corporate communications here at rhythm for those of you participating via conference call.
Our slides can be accessed in control by going to the investors section.
On the investors page of our website at IR job rhythm TX Dot Com. This morning, we issued a press release that provides our third quarter financial results and business update which is available on our website and as listed on slide two here today with me in Boston for the conference call are David Meeker Chair, President and Chief Executive Officer Jennifer.
<unk> Executive Vice President Executive Vice President head of North America Hunter Smith, our Chief Financial Officer, and John <unk> Executive Vice President and head of international is underlying joining us from France.
Slide three I'll remind you. This call contains remarks concerning the future expectations plans and prospects, which constitute forward looking statements actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in our most recent annual or quarterly report on file with the SEC.
In addition, any forward looking statements represent our views as only of today and should not be relied upon as representing our views as of any subsequent dates we specifically disclaim any obligation to update such statements with that I'll turn the call over to David who will begin on slide five.
Thank you, Dave and good morning, everyone and thank you for joining our third quarter earnings call.
So we had a really strong quarter.
Last week, we presented the full phase III hypothalamic obesity dataset and we will review that data briefly on today's call, but the real focus of today is on the progress we are making with our commercial launches in both the U S and Europe .
Cvs is a really good rare disease opportunity both in terms of the number of patients potentially needing treatment in the syndromic nature of the disease, which facilitates patient identification funding.
The fundamental question has been whether we can get this system to work.
We felt good about the early signals in August and we feel even better about.
Today's.
On slide five CBS approval in June we have received 120 plus scripts 80, plus physicians have written prescriptions 40, plus patients have been approved for reimbursement Jennifer will go into some depth on all of these.
Country specific launches are happening in Europe and of course, we received EC authorization for Bbs, which was followed in record time of the approval for the early access program in France, Jan will provide the international update.
In clinical development, we continue to progress our multiple programs with the opportunity for significant label expansion.
Slide six.
So we had a strong presence with UBC Society meeting with 11 presentations, both our presence at costs and improve meeting, which we hosted in Berlin are good examples of how this should work in addition to being intensely focused on understanding and meeting the individual patient's needs. We want to lead with the science, we continue to learn more about.
The genetics the diseases, we're treating and the impact of step Monotype and rare diseases on the first therapy becomes available there is an acceleration in both the awareness of the disease and disease understanding we want to be leaders in that effort.
At <unk>, we presented data showing the sustained effect on BMI reduction in patients with Pompe <unk> deficiencies and body composition data showing that BMI reduction is largely due to the loss of fat mass with relative presentation.
Our genetic testing program continues to support the observation that approximately 5% of patients absolutely positive for gene where in sebree is either approved or the patient would be eligible for the phase III M&A trials.
Slide seven.
So now let me briefly review our acquired Hyperkalemia Weekly program.
Although the estimated patient population of five to 10000 patients may be similar or somewhat larger than the Bbs population. The fundamental differences. If these patients are identified are ongoing engagement with the healthcare system and for the most part we believe they are being actively followed by endocrinologists primary call point for our current commercial efforts. This is a transformational.
Opportunity for them.
So on slide eight near the results for all <unk> patients, which we announced last week 16 of 18 met the primary endpoint decreasing their BMI by 5% or more at 16 weeks 14 to 18 decrease their BMI by 10% or more at some point during the 16 week trial mean, BMI decrease was 14, 5%.
You can see on the next slide in addition to the magnitude of the BMI decrease we think the consistency of the response is quite remarkable.
So this slide nine.
So on the waterfall plot last week, we focused on the purple box highlighting the five patients who had not decrease their BMI by 10% or more at 16 weeks to remind you patients were either in patients in that box, we're either on track to lose 10% or more when they discontinue the drug due to aes at the first two patients on the left hand side of the box.
Did lose 10%, but regained when the dose was decreased to allow a slower repatriation.
That patient is now again lost approximately 10% as the dose has been increased or they were noncompliant the last patient on the right.
Sure there was only one patient who was compliant and failed to reach 5% at 16 weeks. This was Dr. Ross patient, who as we showed was having rapid weight gain which continued through the dose escalation period, and then reversed course once a therapeutic dose was reached and since that time has been slowly but steadily losing more weight.
Slide 10.
13 of 14 patients reaching their first visit in the long term extension to 2009 weeks on therapy had a mean BMI decrease of 21, 1% in the five patients who have reached their second visit decreased their BMI by 26, 7% and.
In summary, we see this data is highly supportive of a strong consistent and durable effect and this heterogeneous population of patients with acquired <unk>.
Slide 11, we had a positive type C meeting with the FDA for our program and acquired Hypothalamic obesity. We're pleased to have received breakthrough designation and to have reached alignment on the phase III trial design, which is diagrammed here.
We will enroll 120 patients randomized two to one to treatment or placebo with an eight week dose titration period, followed by 52 weeks at their therapeutic dose the primary endpoint will be the change in BMI at 60 weeks.
On slide 12.
Here, we summarize our multiple ongoing clinical programs and we look forward to updating you further on those efforts in 2023.
With that I will turn the call over to Jennifer.
Thank you David.
We continue to execute on our <unk>.
Personal lines in Bbs and we are very pleased by the reception across the Bbs can mandate.
Payers and of course.
Today, we plan to share some metrics that our cumulative.
Timber 30 at the close of the third quarter.
To remind you we received <unk> approval on June 16th with just two weeks remaining in the second quarter.
Our last earnings call, we shared some initial metrics on the Bbs, which covered the first week through July .
Those numbers will be included in the metrics. We report today. So let's go ahead and start on slide 14.
This slide outlines the journey that we're on with Pacer and progression.
Early on in this journey, we are educating physicians and patients to recognize Jim to get to an accurate Bbs diagnosis.
Once the diagnosis is made we engage physicians and patients and a dialogue on the impact of it is EBIT now outlining hyperphagia and early onset obesity that result from impaired <unk> pathway signaling.
This opens the opportunity to further educate on.
Our next Jeffrey as the only approved therapy that targets the root cause by restoring the sequel are signalling.
All of this is aimed at driving.
Great.
Once a decision to treat with Jefferies may we helped secure insurance coverage for patients with a first question transition their market and provide support.
With benefits.
Hey.
Next slide.
We are very pleased with our first full quarter results from DBS.
Numbers that cumulative from launch through September 30th.
We have received over 120 <unk> prescriptions for Bbs patients.
We are pleased that these are coming from more than 80 unique prescribing physicians.
Ross the nation.
And importantly.
120, plus prescription we have already received a profile for reimbursement for more than 40 update.
It is still early in the launch for Bbs and rare disease launches that are difficult to predict or even project from here.
However, we are pleased with the first full quarter results at the system, we are helping to support for patient diagnosis and identification customer engagement education around treatment and early interactions with payers is working.
King.
Let's move to the next slide I can go into a little more detail on fluids prescribing.
Of the 80, plus prescribers the number one specialty accounting for 46% of prescribers in pediatric and adult endocrinologist, which is not surprising.
Pediatricians, a second accounting for 24% of prescribers.
Interestingly, 24% of these prescribers are near to read them and we're not called on by our territory managers prior to the receipt of prescription.
This speaks to the success of our non personal promotion efforts targeting both healthcare providers and patients.
Along with our ongoing engagement with the Bbs patient Association and community.
Next slide.
We continue to be encouraged by the payer mix of prescriptions received to date as well as the early coverage decision.
Here, we break down the payer mix on the first question.
48 <unk>.
Prescriptions are covered by commercial plans.
42% by Medicaid.
A total of 7%, our Medicare and 3% are federal plan.
Additionally, with more than 40 approvals for reimbursement.
The vast majority of remaining prescription R&D initial benefits verification prior authorization or first appeal stages and their teams continue to work to secure reimbursement. This process typically takes between one to three months.
Well on average commercial plans, Dr. Rajiv new indications four to six months post approval.
We are pleased to see the first commercial policies came in with updated bvs language aligned with our FDA approved label.
On Medicaid typical review periods for rare disease drugs vary by state a.
A few states with will review, a new indication within six months.
Others can take eight to 12 plus months.
Within <unk>, we're starting to see some states issued favorable policy.
There are also some states that are excluding coverage of a separate study in obesity exclusion. However, even in these states. We do see positive reimbursement on a case by case basis or an appeals process.
For those who are unable to gain reimbursement at this time, including our Medicare patients and we have exhausted all levels of appeal, we have a patient assistance program for <unk>.
For these patients we continue to pursue options for coverage. We remain confident we will see more patients reimbursed on therapy due to separate coverage policies that are developed for through the appeals process.
Moving onto the patient characteristic and the next slide.
These numbers include the 20 patients that we have transitioned from clinical trials.
We see the age range of patients for whom we have received prescriptions.
Children, and adolescence account for almost 60% of prescriptions and adults 18 and older account for more than 40%.
More than 90% of Bbs patients with prescriptions have consented to participate in our patient support service program rhythm in June enabling us to help patients achieve access prepare them to initiate and separate treatment and ensure ongoing continuous therapy.
As these patients engage with our engine support team, we hear positive feedback on their experience on therapy and with the support provided.
Next slide.
As you see from the quote.
<unk> highlighted that her daughter has more energy is less hungry and had lost 10 pounds.
Another sleeping better at night smiling more during the day.
Separately, a wonderful changed in one boy after being honest Jeffrey was due to a new found confidence.
And lastly, here, we see how and into and patient education manager, we call them <unk> was able to set expectations to educate one family on the potential side effects have been separate so they were able to manage through the titration stage to then be able to experience the benefits from decreased Panther.
And food stealing behaviors with noticeable changes that school.
We are so pleased to hear that <unk> is making a positive difference to these patients and families.
Next slide.
We have had a strong start on our Bbs launch and there are still more opportunity for growth as we continue to identify additional physicians with diagnosed patients.
We have segmented patients with Bbs into four main buckets.
First there are patients under the care of physicians already known to rhythm that we continue to engage and educate.
Second there are already diagnosed patients under the care of new physicians that we discovered through the work of our field teams.
Third there are patients to physicians may suspect of having Bbs, where the availability of genetic testing can help inform their diagnosis.
And for US the largest group, which is applying our efforts to help undiagnosed patients find that answer in their journey to diagnosis.
For each segment, we have leveraged our multichannel approach to supplement the efforts of our field team to educate a broader group of health care providers and patients. So they didn't get to quicker diagnosis and are aware of the availability of a separate as a targeted treatment option.
With that let me hand, it over to Jan to provide an update on the progress in the international region.
Thank you Jennifer and good morning, everyone.
It's been again, a very dynamic quarter for the international region.
Slide 22, as a quick reminder, that Europe is an important market for Sui.
To the European where these centers of excellence and Mod Vince genetic testing capabilities, mostly due to many patients with pharmacy and <unk> deficiencies and many patients with <unk> syndrome already identified.
On the CPC is Q1 of <unk> deficiency estimated European prevalence is.
600 2500 patients.
We have identified about 100 patients already diagnosed and received in Q.
<unk> syndrome is a European prevalence is estimated to be approximately 500 patients and we know that there are more than 1500 patients who are diagnosed and receiving you for <unk>.
Slide 23.
Regarding the European launch for <unk> Q1 of <unk>, we have made tremendous progress in securing access in Europe a.
A few weeks ago, we launched <unk> in the UK and as you can see on the left of the slide we do enjoy the support from leading nobility express like polka SASSA. Therefore.
Keith from Cambridge U K rail.
Widely recognized as one of the most influential leaders and genetic obesity and with twitchy devote their first prescriptions.
Now we are able to deliver <unk> to patients in the U K in France and in Germany. We are on track to launch very soon.
The Netherlands also Israeli by at the end of this year and things within the in Argentina in 2023.
Slide 24.
Regarding <unk> syndrome in Europe , we are beginning to deliver <unk> to patients with Bbs, we're looking for precision medicines that can significantly reduce hunger and body weight.
We received the final authorization from the European Commission in September as we announced although this summer we achieved a very specific and rail reimbursed early access program called Q1, Prince even before the <unk> recommendation and the EMEA approval and just 32 days following the FDA approval.
Did you say that a record time.
One of those four reimbursed early access to innovative therapies when a positive risk benefit balance you recognize in the windows of therapeutic alternatives are available.
Approximately 700 patients with <unk>.
Syndrome, diagnosing trends there was a clear unmet medical needs.
In Germany, we have engaged a gain with the joined favorable committee the GVA with very positive interaction so far as.
As we did for <unk> given the rebound we are seeking an exemption from the lifestyle Greg's reimbursements exclusion list and we are looking forward to launching <unk> in Germany in the first half of 2023.
In the meantime, we're also engaging with and scale with the receipt in the United Kingdom, Italy, Spain, and the Netherlands.
Slide 25.
But not least.
We did so both last fall's.
International meeting in the past we related obesity called improved 2022. It was 1 billion and we are very pleased with the turnout and the level of engagement and discussion.
It was led by Professor Peter Kiernan Commerciality in the lean was a chilled limiting and focus so much more of each of the units.
Seton Medical center as co chair.
Nearly one of these physician and scientist to share ideas and best practices on how to address unmet needs in the diagnoses and treatments of people with.
CVR audiences of EQT and how to integrate the genetic tests and therapeutics as part of routine patient care.
It was truly focused on science and evidence based care practices for in Q4, but we view.
No doubt.
The field to move for a lot of and we are already planning for the next improvement.
Andrew.
Thank you so much.
Turning to slide 27.
We closed the quarter very well capitalized with $348 million in cash on hand.
$14 million in additional proceeds from the green shoe from our offering pro forma that would put cash on hand of $362 million.
Including the partial exercise of the Green shoe, we raised $140 million and our public offering in September and importantly, we successfully expanded and deepened our investor base anchored by some of the most sophisticated long term investors in biotechnology.
In addition, following <unk> marketing authorization for <unk> Sebree in Bbs, we received the additional $37 5 million from healthcare royalty partners during the quarter as per the terms of the revenue interest financing agreement concluded in June .
<unk> investment demonstrates.
Demonstrates confidence in him sebree for Bbs and our potential to generate strong revenue growth in this indication we remain eligible to receive an additional $25 million upon the achievement of certain sales milestones in 2023.
Together these transactions provide rhythm with significant financial resources to execute on our global development and commercialization strategies and extend our runway into 25 2025.
On slide 28.
Third quarter represented a strong start to our Bbs launch with more than 120 scripts written since Paducah in June .
Revenues of $4 3 million represent an early start to the sales growth, we expect Bbs as the patient access process takes approximately one to three months following the receipt of a patient start form from a physician.
In addition, given the additional the initial dose titration schedule. The first one month shipment of sebree to patients once approved.
<unk> fewer vials of drug than the second and subsequent shipments finish.
The initial product shipment has approximately 55% to 70% of the volume.
For the typical at those shipments beginning in month, two and continuing on from there. This lower per patient shipment volume is reflected in revenues during the first quarter of Bbs fails.
Cost of goods sold for the third quarter was $496000 approximately 12% of product revenue. This.
This was primarily driven by the amortization of milestones previously paid.
As well as 5% royalty due to epson under our original licensing agreement. These two factors represent over 86% of total Cogs.
R&D expenses for the quarter were $21 million compared to $27 5 million for the third quarter last year.
There was also a decrease in R&D from $31 1 million in the <unk>.
Second quarter of this year.
The year over year quarterly reduction was the result of a $5 $2 million decrease in clinical trial costs and a $1 3 million dollar decrease in CMC costs decreased clinical trial costs were due to study design amendments to our phase II DAYBREAK study as well as reduced activity or completion from the face from the Bbs phase III.
The phase II basket, Q Tc and renal studies. These decreases were partially offset by increasing increased spending on our M&A open label extension and weekly formulation switch in de Novo studies.
Quarter over quarter decrease was largely related to the same factors.
Turning sequencing expenses decreased by $1 2 million.
I'm, sorry increased by $1 2 million in the quarter.
SG&A expenses for the third quarter were $21 9 million compared to $17 5 million in the same quarter last year were down slightly from $42 3 million in the second quarter of this year.
The year over year increase was primarily due to PBS commercial launch in the U S as well as increases in head count for our U S and international commercial organizations.
Our share count as of September 30 was $55 7 million basic and diluted shares an increase of $5 5 million shares versus September 32021, primarily reflecting the impact of our recent share offerings loss per share was <unk> 79.
Versus <unk> 70 in Q3 of 'twenty one.
As I indicated on the prior slide following our September offering and receive the second tranche from the HCR capped royalty financing agreement.
We closed this quarter with 3300 $48 million in cash on hand, and subsequently received an additional $14 million from the shoe.
That gives us approximately $3 million to $162 million.
Which.
Which will be sufficient to fund planned rhythm observation operations into 2025 with that I'll turn the call back over to David.
Thank you Hunter.
Hope what you've heard today is that this is a really good quarter, but we feel great about it.
Moving forward, we remain laser focused on executing against our strategic priorities for 2022, and 2023 that is continued execution in our U S. DBS launch and internationally continuing to work the market access for both pumps <unk> deficiency, and now Bbs and continued execution on our clinical development programs not only in the transfer.
Formative opportunity we've talked about now twice on hypothalamic obesity, but also continued progress in our ongoing M&A DAYBREAK pediatric and weekly trials and we look forward to our next update with that we'll open it up for Q&A.
Yes.
As a reminder.
To ask a question you May press Star one one on your telephone.
Please standby, we compile the Q&A roster.
Our first question comes from the line of Phillip Naidoo from Cowen Your line is open.
Good morning, Congrats on the progress and thanks for taking our questions. A couple of commercial questions for us from US first can you speak a bit more about the <unk> program. What is the team at <unk> due to get the patients on therapy stay compliant with therapy, and how does <unk> help with.
The physicians with the appeals process.
The first question and then the second question you mentioned the average time from script to reimbursements one to three months.
Is that likely to decrease as the launch progresses, where do you think ultimately that time.
Thanks.
Okay.
So a little bit about churn.
We don't hear was.
Program that was debated quite customized for the needs of the customer so far.
So everything here starts with that pace.
Back when we actually receive that patients with pad our patient education manage our path then reaches out to the patients.
For introductions and also too Jeff.
Educate Jeff in terms of what the process will look like moving forward.
Similarly for any new per Scriber, there is a joint meeting with the pen.
And our territory manager to once again outline expectations moving forward.
So at the prior authorizations dates which is required for every script that we received.
The intent service execute benefit Verifications, and then really works to understand that specific pairs exact prior authorization requirements that needs to be filled out at this stage I will say that some of our patients.
Have complicated insurance background I mean, we had a patient that had three different insurers, where we need to figure out who was the primary insurer and then what's the process from there. So there's a lot of work done upfront to understand that.
The mansion.
<unk> initiated the prior authorization on behalf of this position through cover my Meds.
Which then pushes all the relevant information to the doctor and and as accounts.
The ACP does need to complete all required documentation.
But one thing that we continue to do is we learn from the experiences that we have for example.
What we saw.
Found out quickly.
Dave.
These denials, where either because of incomplete information or because.
They outline Bbs not to be.
No.
<unk> or approved for the indication.
We recommend just in terms of the first stage of apparel that is supplemented with a medical a letter of medical necessity as well as an FDA approval just to once again minimize the upfront denials.
So upon that Michelle we then follow up in terms of the payer decision every two days and keep everybody.
Updated on the status.
<unk> denied we go through the same process just in terms of the appeals process to exactly understand what documents need to be submitted to two et cetera.
So.
Following reimbursement approval then the ensign team schedule of shipments to the patients and the injection training.
That's up appropriate expectations in terms of what it should be like on drug.
And once the patient initiated therapy. There is very regular touch base just to answer any questions make sure that the patients being titrated appropriately.
So there is a very tight.
No connection, especially in the first 30 days just to answer any questions. So overall once again, we just really tried to provide a very personalized experience for both the patients as well.
Hey.
Yeah.
Onto the next question, which was relating to the average time from script to reimbursement of protocol.
I'll just give one example, just to highlight what is.
To look like as we move forward.
One where in the states.
Of launch where there aren't a lot of policies outlined for them separate for the Bbs indication.
As time progresses, we hope that there are policies that are put in place that will make the whole reimbursement process quicker just in terms of our peripheral.
So as we're working within this.
Rates of lines.
One example, I will outline is for example, one pair it took.
A bit of steps, including going to the appeals process to get the first scripts approved.
However, subsequent scripts.
Scripts, they came to that specific payer words approved within days.
Even for specific parents.
Some time for the initial script to get approved but we hope as we move forward. Similarly, the time to scratch your approval well Peter.
Thank you. Thank you Phil next thank you that's very helpful.
Thank you one moment for our next question.
Our next question comes from the line of Dae Gon from Stifel. Your line is open.
Hey, Good morning Hope you guys can hear me and thanks for taking my questions and congrats on the progress from me as well.
One on <unk> and one commercial question for me.
With regards to the phase III <unk> trial design that you outlined a couple of days ago.
Mentioned, it's about 120 patients randomized placebo to <unk> and I guess 99, 5% powering as it stands.
Can you maybe walk us through the thought process behind going after 120 patients that 99, 5% is extremely derisking, but just wanted to get your thoughts on.
Why you've sort of landed at that $1 20 number and then commercial question.
Also 120 prescriptions, where more than 120 prescriptions can you talk about how much overlap. There is with the 350 that you had talked about previously identified diagnosed and maybe as an extension how does that compare with the cribbs registry. Thanks, so much.
Yes, certainly.
The ACO question person central second so.
The 120 patients going into the.
The FDA meetings, we renew and you all knew that given the efficacy we had seen in the phase two the number of patients required to prove efficacy was likely to be relatively small.
120 patients is way more than that not surprisingly so that was really a discussion around safety.
This is a new indication and that was just in the negotiated if you will agreed upon the number of patients that would provide a robust.
Evaluation of safety in this new indication, but it was not driven by efficacy.
Thanks, Jonathan.
Relating to the 120 scripts.
Say that one piece.
Just in terms of.
The outline that almost a quarter of the scripts were coming from HCP is that were not known to them. Prior to your first question. So.
One piece to.
To the.
Our progress just in terms of the teams continue to make around diagnosing additional.
Physicians with Bbs patients it speaks to the very.
Collaborative relationship that we have with the Bbs patient Association and just in terms of helping to inform.
The availability of defray it also speaks to the.
So I believe relationship.
Know that we have with the folks.
And health care providers over at Marsh scale.
In terms of the exact overlap of the scripts.
<unk>.
<unk> that are in the Cribbs registry, we don't have that information in terms of understanding.
The.
Physicians with specific patients that are part of the cribbs registry itself.
I'll say that just in general though the teams are just continuing to expand the opportunity around educating the physicians that we have.
That already have babies.
Bbs patients and once again.
Really trying to expand the opportunity just in terms of additional patients.
That get diagnosed.
Great. Thanks, so much for the color guys.
Thank you Dan.
Great.
Next question.
One moment for our next question.
Okay.
Okay.
Next question comes from the line of Corn Jenkins from Goldman Sachs. Your line is open.
Good morning, everyone and congrats on the quarter.
Sure. The 4500 patients is the more appropriate Tam from sebree in bvs, which is compared to the 2500 you've shared previously so what are the some of the key items since the lines that give you confidence in the expanded addressable market.
Yes, maybe I'll lead off and then Jennifer can supplement. So this is math that we just did.
The original epidemiology.
Rare diseases as we know is tends to be soft just because theres not a lot of publications and data to inform that as we've gotten much deeper into this community. We have been able to triangulate different points of information to get to a comprehensive the number. We believe is larger I E. The 4500, plus kind of opportunity one of the major ones is just <unk>.
European opportunity, where can be used France.
As John said, the number of diagnosed a bbs patients in France was 700 easily the number of patients with Bbs is probably to ex that or more but if you just use a thousand patients is the number for France, and then correct for the U S population brands are about $70 million in the U S of course of $3 30.
Thats, the kind of triangulation and the other thing that is beginning to give us some what's really interesting and Jennifer highlighted. This is the rule that testing will play we're extremely early in terms of that but it's a clinical diagnosis, but blood test genetic tests are part of a clinical diagnosis may inform and so in a prior world instead of having to wait for a patient to develop five or six.
Stations of the disease.
We think theres, a great opportunity for patients to be diagnosed earlier on but their initial presenting signs and symptoms complemented by by genetics, So, we'll see where that goes but.
That answer your question.
Yes. It does thank you and then what have you learned from the 25% of prescribers that you werent known to rhythm.
To the lines that might inform kind of your next year are targeting as you continue to expand this opportunity.
Good afternoon.
So I think like this in terms of the backgrounds of the prescribers.
It's interesting just in terms of.
Where they are so of the 25% I would say that it's probably a higher percentage that may come from.
Our only dock or pediatrician's versus our target.
Some of them also similar to our targets had certification with interest in terms of obesity medicine and such so I think it's a little bit of a mix in terms of the prescribers.
In terms of how they came to us it could be that they learned about <unk> had a bbs patient. It could also be because the patient themselves learned about them, so afraid and went to there.
Physician to inquire about the drug.
No.
It's clear that in terms of the organization, we have a certain level of field.
Members on ground educating but this is where we really turn on our non personal promotion effort to try to reach a broader set of physicians and patients out there to make them aware and educated in terms of the disease itself as well as the availability of truck.
And Ken maybe one thing that just in our experience in other rare diseases, what's quite remarkable but theyre not our target population primary care physicians for example, as Jennifer said, if they come to attention because they have a patient and they are willing to give it a try some of those physicians continue to grow they've taken interest and begin to take on more pace.
And truly become expert in the area. So it doesn't exclude the possibility that a primary care physician could be.
Leading treater, if you will.
Thank you.
Thank you one moment for next question.
Our next question comes from the line of Joseph Stringer from Needham Your line is open.
Hi, Good morning, Thanks for taking my questions two from US. The first one is on the you were identified and diagnosed Bbs patients previously had had.
Greater than around 300, just curious.
What percentage of those are.
Below are under the age of six.
And.
We're therefore not eligible for commercial truck and then.
Second question is.
And that you're just over a quarter into the launch and Bbs, but.
Just curious if you can provide some.
Early metrics on persistence and compliance rates now that youre getting more patients on drug.
In line with expectations so far.
Yes, great Jennifer.
Yes.
Sure.
C outline.
<unk> 350 patients.
Say that at this point, we really don't know the percentage that are under six years of age.
So that percentage I cannot outline at this point of time, but what I will say is that our focus is not of course on that.
The greater than 350, but continuing to get additional patients to a diagnosis that we have very targeted plays that are outlining to be quite fruitful Matt perspective.
And maybe one example, I can provide on that point in terms of how we get additional patients to a diagnosis.
I've mentioned in the past that there are very specific ways, we use machine learning as well as ICD 10 code as well as a follow up in terms of our Euro result, just to have very targeted follow ups from our field teams, but.
A separate example is the fact that for Bvs a lot of these patients have official issues.
Cause by retinal abnormalities that once again sort of speaking to how we deploy our teams.
There are many many more ophthalmologists out there versus retinal specialists in the U S. So our field team may focused on the retinal specialists while we.
We go out and educate the product groups.
Our non personal promotion efforts and these are proving.
Proving to be successful.
I wouldn't necessarily just focus on the greater than $3 50 number but we are once again continue to expand that opportunity.
In terms of.
Metrics around persistence and compliance on this point I will say that we.
Spent a lot of time internally as an organization just to walk through and planned for just in terms of how can we educate the patient and ACP.
You understand that.
All of this drug and really try to maintain them through the initial titration phase. So that then they could get to the appropriate therapeutic dose.
And then start to see the benefits of being on treatment. So I will say that and once again, it's very early days, but at the same time, we are quite quite happy just in terms of the ability for these patients to get through and once again start to.
Experienced the benefits that we're hearing about.
Okay.
The answer to your question Joy.
Yes.
Thanks for the color and thanks for taking my questions.
Thank you.
Our next question.
Our next question was offline of Michael Higgins from Ladenburg. Your line is open.
Okay.
Michael Higgins from Ladenburg Your line is open.
Thanks, our equity I Couldnt quite hear you there thanks, guys for taking the questions. Congrats from me as well and we continued progress impressive.
Personal loans, so far in Bbs.
For Julian David.
Given a lot of detail answered a lot of our questions coming in on the launch you would provide a lot of detail on the patient count curious if you can give us patient counts.
For <unk> and sebree on pumps, the Lipper PCF SKU, one also to kind of.
Help us understand the launch and Bbs kind of whats going thanks.
Yes, I think.
We're not updating those numbers that we stuck with it from the beginning I think they are informative and know the right number as we said tens of patients on treatment was our expectation we have tens of patients on treatment.
I'm not going to speak to that number specifically.
And the reason Michael basically is it doesn't inform Bbs launch there are quite different in terms of disease populations.
<unk> metrics or circumstances, if you will that allow a bbs patients to come to diagnosis and get access to treatment aside from the fact that it's just a larger population overall as well so.
So that's progressing we have like I said, the tens of patients, but the focus here is really on the Bbs launch and how that's.
The other other than the reimbursement part of it reimbursement of course.
The earlier population has been helpful. In somewhat predictive of how things are going to go for Bbs and as Jennifer said, we feel good about that.
I appreciate that one other one if I could here.
The H O trials continue.
Continuing now and given the long term extension, we have some patients of 25.
Do you know when we may see an update on those and at later time points.
Yes, I mean, we will obviously continue to follow these patients. So again I can imagine that the subsequent earnings call a little bit depends on sort of when we do data cuts on that.
<unk> data cuts.
Weekly obviously so in fact, we've tried to time them about twice a year. So bottom line is yes, we will update it will be in 2023.
Well keep you informed but we are going to be following that closely.
I appreciate it thanks guys.
Thank you.
And once again Thats star one one for questions.
One moment for next question.
Our next question will come from the line of Jeff Hung from Morgan Stanley . Your line is open.
Hi, This is conference on for Jeff. Thank you for taking our question. We have two the first one is on the de Novo study Youre starting in 2023 could you remind us what the study design and what you're hoping to see from that trial and then our second question is on the phase III and hyperbolic obesity do you mind, providing additional color on the rationale for all patients starting answer your 0.5.
<unk> one in phase two they started on one or two milligram. Thank you.
Yes, yes, so the de Novo trial is a double blind randomized controlled trial in patients predominantly with DBS. We will have a small number of patients with PPL since we'll be with pumps and left bar specific leases will also be looking at the effect of the weekly dose in that population and we'll be looking at.
Two major primary endpoints, we've pursued all along which is the change in BMI again that'll be a population that will span children and adults were getting BMI as the United <unk>.
Endpoint, if you will it allows us to at least aggregate that to a certain degree and then hyperphagia and I'll just say on the Hyperphagia point again. This is something that as we come to understand this population in disease more thoroughly.
Hi, Prophage, a component continues to be reinforced and I think it is growing in awareness on the health care provider side of the equation I think patients. Some are articulating them more of the quotes that Jennifer shared with you today. They werent Cherry picked and those are those are close to just many of those quotes and like I said, the vast majority of them speak to some level.
The improvement in their hunger and that being an important part of what others benefiting from the drug so we'll be looking at that now and that trial as well.
On the phase III trial in terms of the dosing.
Just a practical thing we've been still experimenting a little bit with different titration regimens in the pumps. The left bar, we started lower and when slower than that.
Quite a bit of Louie, we're more aggressive if you will and part of being more aggressive was to take advantage of a 14 week placebo controlled period, where we wanted to get patients to their target dose as quickly as possible and the ongoing <unk> studies a year along.
Study as you know and therefore, there's less urgency to get people to target dose and we do know that.
Going a little more slowly than a little more.
Just starting at a lower dose going a little more slowly increases the tolerability of the drug through that first four week period, which is when they are at greatest risk for any of the Gi complaints, which may cause people to struggle being on the drug so that's practical.
Yeah.
Got it thank you so much.
Thank you Catherine next question.
I'm not showing any further questions in the queue I'd like to turn the call back over to David Meeker for any closing remarks.
Okay, well, thanks, everyone again for tuning in to calls in short order here and we look very much forward to our next one fourth quarter earnings coming up.
'twenty three.
Good day.
And this concludes today's conference call. Thank you for participating you may now disconnect everyone have a great day.
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