Q1 2020 Earnings Call
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Ladies and gentlemen, today's conference is scheduled to begin shortly please continue to stand by and thank you for your patience again your conference call will kick in on the Tammy.
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Good morning, and welcome to ideals first quarter Twentytwenty conference call. At this time all participants are in everything only mode. There will be a question and answer session and please be advised that this call is being recorded at Agios request I would now like to turn the call over too.
Only many director of Investor Relations.
Thank you operator, good morning, everyone and welcome to our juices first quarter 2020 conference call you can access slides for today's call by going to the investor.
Section of our website at <unk> Dot Com with me on the call today any prepared remarks are dr., Jackie Farrell, our Chief Executive Officer, Dr., Chris Allen, Our Chief Medical Officer, there and well our senior Vice President of U.S. commercial global marketing and Andrew Hirsch, Our Chief Financial Officer, and head of corporate development Dr.
Bruce Par I, Chief Scientific officer will join for QNX before we get started I would like to remind everyone that some of the statement to make 'em. Paul will include forward looking statement.
Actual events or results could differ materially from those expressed or implied by any forward looking statement as a result various risk.
Uncertainties and other factors, including those set forth in their risk factor section of our most recent form 10-K filed with the FCC and any other phones that we may make with the SIFI with that I will turn the call over to Jackie.
Thanks, Holly good morning, everyone and thank you for joining our first quarter 2020 results call.
At the beginning of year, we laid out ambitious plans for 2020.
Along with the rest of the World. We couldn't know what challenges would lay ahead related to the impact of the co bid 19 pandemic.
The past few weeks have stretched each of us personally and professionally as we adapt to the necessary constraints of physical distancing and realize more than ever how our actions could impact the people around us.
We wish continued good physical and emotional helps to all of you are patients the systems health care professionals are all geos team members and humanity at large.
There was no modern playbook for a crisis of this type put it all geos, we quickly realigned our priorities around the health of our employees and their families our communities and the patients we serve.
To start in February we activated our organizational resiliency team and clinical operations response team to assess the evolving Corona virus outbreak and put plans in place to address its impact on every facet of our business.
On March 10th we made the decision for most of our employees, including lab and field personnel to work from home to reduce the likelihood of exposure or transmission of the virus to others a policy that remains in effect.
Our supply chain strategy has ensured uninterrupted supply of our commercial and clinical medicines and or patient assistance programs. My all Geos has been a constant resource to help new and existing Tim. So go patients remain on therapy, including those that have lost insurers do the unemployment.
With the help of our dedicated employees, we've been actively supporting our communities in healthcare workers, including organizing a donation of RG else's personal protective equipment supply into local hospitals, raising more than $22000 for the greater Boston food banks and encouraging blood donation.
The team work and resilience I've witnessed in these last few weeks has truly been remarkable and gives me hope that we will come out of these challenging times, even stronger than we were before.
Turning to the impact on our business, we've taken a fresh look at our priorities and made important resource allocation decisions to support execution against the most critical objectives for our business.
During this period of disruption it uncertainty we're focused on delivering on our top objectives.
Including driving tip, so performance enrolling our late stage email and glioma studies and advancing middle pivot across multiple disease areas, all while conserving cash and increasing our financial flexibility.
As a result of our prioritization efforts, we've extended our forecasted cash runway by six months through June of 2022.
Andrew will further discuss the decisions we've made later in the call.
As I mentioned earlier, we created a clinical trial task force in February to ensure the safety of patients on our clinical studies and to implement plans to keep as many patients on study as possible.
We've been managing this on a patient by patient basis, consistent with the FDA guidance.
Chris will walk you through the impact each program and trial as well as the measures our team have taken to address pandemic related challenges.
On the commercial side, we saw strong picked so well performance to start the year and an acceleration of revenues during the month of March into April, which reinforces our confidence in our 2020 product revenue guidance. There on will discuss the trends we saw in Q1 in more detail.
[noise] through these efforts we remain on track to achieve our Adrs 2025 strategic vision.
Before I wrap up I want to think every I'll just employee for their incredible work over these last few weeks youre determination in the face of adversity has quite literally say blogs and I could not be proud or to be on your routine.
Let me turn it over to you.
Thanks Jackie.
Go through a clinical update.
I will share with you the status of our study based on our best estimate of the impact we see today Corona virus pandemic.
As a partner and collaborator more and more than a dozen clinical trials.
Oh focuses on supporting slides investigators that patients enrolled in our studies. They all deal with the strain of Cobiz 19.
The Jackie mentioned, a clinical operations respond to me daily.
The evolving situation mitigate disruption that's the viability of patient by patient basis.
I'll start with me right.
Why we pay Iraq.
Currently being evaluated holiday on his decision to follow senior sickle cell disease.
<unk> first quarter. This year, we achieved several important milestones for our most advanced program PK deficiency.
Well, we have the potential to provide modifying therapy for this movie.
In March we pick up was issued a positive opinion on its application for orphan drug designation.
He agency for the treatment of adult because decisions.
EMA orphan drug designation provides us with a number of benefits, including 10 years of market exclusivity.
In addition, we completed enrollment to be phase three study maybe to that PK deficiency patients who are not regularly transfusions.
Total wiimote 80 patients.
And the activate T study and PK deficiency patients who are regularly transfusion, we enrolled 27.
Both studies our focus now is to do everything we can to ensure patients on study Abaxis to study drug did you catch or clinical data, regardless of what patients ability to get to their medical center.
This includes home bid that telemedicine approach you use of local laboratory.
Courier shipments.
To our knowledge as it was all these efforts no patients on study this study drug to date.
As the core period at both studies come through and then later this year.
We do anticipate challenges to our ability to access twice a day to.
Particularly those areas most affected by dependent.
Given this.
No we anticipate topline data both studies sometime between me and 20 twice in the middle of 2021.
Based on our current assumptions potential approval of mid to put that in PK deficiency is now expected to occur in 20 to 22.
We hope to further melody timeline once we have a better understanding a one participating sites or will allow for monitoring.
Access will affect our ability to achieve database lock, what's the last patient visit occur.
Moving to the phase two study admitted Tibet and Dallas Senior we completed enrollment in this study earlier this year with 20 patients above on initial target enrollment of 17.
As you May recall, we establish proof of concept Dallas anemia in December based on interim data from this trial demonstrating that treatment.
Due to hemoglobin response of greater than or equal to one grams per deciliter and 78 evaluable patients.
Data from 13 efficacy of valuable patients, including those with data and I'll start with Cvs I've been accepted for presentation at the European Hematology Association Congress in June.
Which is being held using virtual format. This year.
As we disclosed previously.
So we like Chen of the nationally.
The two to help submitted the data from the proof of concept study admitted tobacco sickle cell disease for presentation at E.
Although the data were accepted into the new virtual format.
Yeah, <unk> decided not to present the data and the submitted for presentation at the Ash annual meeting in December.
We are still on track to make a decision on proof of concept permitted to that in sickle cell disease by mid Twentytwenty.
The goal of the studies to evaluate safety Tolerability pharmacokinetics, and pharmacodynamics escalating oil doses of middle.
In addition to the five milligram 20 milligram 50 milligram doses evaluated original protocol.
Dr. Chan recently amended study the dose patients up to 100 milligrams admitted to that twice daily for two weeks.
Dr. Chan plans enroll up to 25 patients in this study, though enrollment is currently pause in light of the cold and Mike pandemic.
Beyond mid to pivot we have developed a next generation PK, our activator AG 946 in March we received clearance from the FDA <unk> nine for six I M. D application and we expect to initiate phase one healthy volunteer study mid 20 Twond.
The phase one study will evaluate 89 forces in approximately 32 healthy volunteers in a single eight ascending dose cohort followed by a multiple ascending dose cohort.
The goal is to evaluate safety Tolerability pharmacokinetics, and pharmacodynamics of 89.6 potentially therapeutic dose.
In addition, we've included the option to enroll further multi ascending dose cohorts in adults with sickle cell disease.
Safety and Tolerability profile supports continued goes.
I'll now moved towards Whitney Hematology programs, where we're focused on geographic indication expansion for our IDH one inhibitor to it so.
To start you. We have received a day 180 was outstanding issues for our team Somo filing in relapsed and refractory AML.
And at this time, we still anticipate receiving a CHF opinion by the ended the year.
In frontline and now we are focused on enrollment to global frontline combination phase three studies, which will allow us to reach the largest number of AML patients with an IDH one church.
First as the agile phase three study it took so in combination with they decided he.
And the second is the cooperative group hold on one system study, some civil and I'd thought in combination with standard induction and consolidation chemotherapy.
As a well tolerated oral oncology therapy physicians continue to refer patients to both study in patients have been able to access Tim so including home shipments of necessary.
However, like startup activity has slowed and enrollment interruptions have occurred some sites to the pandemic.
Yeah, John trial, which we originally projected to complete enrollment by the end this year.
Now expected to complete in Twentytwenty, one as a result of Q1 delayed site Activations in China in enrollments interruptions across the globe.
In recent weeks some Chinese sites have been able to open and we expect to provide more precise timing on enrollment completion as countries move away from the crisis and returned to pre pandemic work environments.
The majority of clinical trial sites for the hope on 150 study or in Europe.
And enrollment at sites and heavily impacted countries has slowed.
We're still relatively early in the accrual process and since it is a fairly long study, we maybe able to make up some time along the way.
We will provide an update on expected enrollment completion once all sites activated we have a real and accrual rates.
Beyond and now we recently reopened them I went to slapstick syndrome or phase one study so what's the goal generating the data necessary to pursue potential U.S. regulatory filings.
Since we are early in the process for reopening this study sites by the activities have experienced delays and enrollment completion is now expected in 2021.
Finally, I'll turn the AG 66 hour D. H D. H inhibitor currently being evaluated in a phase one dose escalation study in advance lymphoma.
At the beginning of the year. We plan is to make a go no go decision on further development of 86 me say by the end of Twentytwenty.
As a result for the prioritization exercise the Jackie highlighted and very limited enrollment study a total of 11 patients enrolled over the course of 10 months. We have made the decision to stop in house development.
At this time, we plan to look for a partner to invest the program.
I'll now moved to solid tumors, where we are advancing pivotal study IDH inhibitors in cholangio carcinoma, and low grade glioma and evaluating 80, 270, I'm not too ace inhibitor in combination with taxing and pancreatic and lung cancers.
Last year, we recorded the positive readout of the clarity phase three study to Somo and previously treated IDH one.
Cholangio carcinoma, showing that treatment with himself will reduce the risk of disease progression or death by 63%.
In mid 2020, we expect to have mature overall survival data from this study to support the potential supplemental NDA filing.
Based on the current state of the Cobot 19 outbreak and the impact. It has had on trial site resources, we anticipate challenges in collecting BLS data from site and executing the data cleaning process.
Given the yes, indeed submission that no one can do occur sometime between the end of Twentytwenty and the middle of 2021.
The Indigo trial is our phase three study for side, there are being brain penetrant IDH inhibitor Ludwig we know initiated in December.
We're currently experiencing some start up in enrollment delays as a result of dependable.
Given the early stage of this study and this along timeline, we will likely be able to make up some time along the way.
We'll provide an update on expected enrollment completion once all sites have been activated and we have a better view on accrual rates.
In the meantime, we look forward to sharing updated data from the phase one study of fore sight and non enhancing the low Greg.
ASCO in late May.
Lastly, enrollment in AG 270 phase one dose escalation combination arms is ongoing but has slowed as some site as resources have been shifted due to the coping 19 pandemic.
We still expect to be able to make it go no go decision on this program no later than 2022.
To close I'd like to thank our clinical operations response team as well as all of our safety medical regulatory technical operations color, we're going above and beyond the call of duty. These last few months.
The implications of this crisis apart from over in the Swiss their hard work and leadership that we're able to stay in corn support our clinical trial partners and make decisions in the best interest patients.
With that I'll turn it over to Darrin to discuss our first quarter commercial performance.
Thanks, Chris.
I'm pleased to show that we delivered strong performance during first quarter recording $23 billion of net sales of tip silver, 60% increase from the fourth quarter 2019.
Growth was driven largely by increases in new scripts, and refills and both the newly diagnosed and relapse and refractory in all segments.
Our market research shows that took civil containing regimens are considered standard of care for newly diagnosed I'd. Each one mutation positive patients in eligible for more intensive treatment and first relapse or refractory patients. This is further reflected in our measurement of patient share in both the newly diagnosed enforce we love settings.
No not a focus of our promotional efforts, we continue to observe that approximately half of to kill reducing boots settings as in combination most frequently with them Hypomethylating agent.
Furthermore, This group is supported by continued expansion of the unique prescriber base by 25% from Q4.
Well, we did see the typical Q1 seasonal increase in patient assistance, resulting from changes of benefit design of the started the year. The magnitude of increase was less than we expected as underlying took civil demand grew substantially during the quarter.
Our patient assistance program I'll juice continues to operate uninterrupted into most of the coupon 19 pandemic as we support patients through these challenging times.
As Jackie mentioned in response to the grilling concerns related to the pandemic. Our fuel team has been working from home since mid March we've experienced success continuing to engage customers with tools, enabling remote interruptions.
The urgency to treat patients with and though is high and professional oncology guidelines paper effective all options with well tolerated safety profiles as health care professionals Russell with the needs of patients recruited my team.
Given these dynamics types of as efficacy safety and clinical benefit profiles position as well as a preferred option for treating I each one positive patients.
These factors coupled with sustained momentum in physician demand through April give us confidence in our full year 2020, less trips over net sales guidance of $105 million to $115 million.
We continue to monitor market trends carefully given the ongoing impacts of the pandemic.
I want to thank exceptional team at all Geos for rising to the challenges posed by cobot my team and continuing to deliver for patients are customers and the business.
I will now turn to Andrew to discuss our first quarter financials.
Thanks Darren.
Our first quarter results can be found in the press release be issued this morning, which I'll summarize.
More detail will be included in our 10-Q filing later today.
I'll start by discussing a quarter and then describe some of the cash conservation actions, we've taken that have been able to increase from way.
Total revenue for the first quarter was $87 million, which consisted of $23 million that net sales of tip salvo.
$61 million of collaboration revenue and $3 million, if I'd for royalty revenue.
The year over year increase in revenue was primarily driven by a 42 million dollar increase in collaboration revenue.
As a result of BMS is notification that they would not be extending the 2016 meal research collaboration.
And that they won't be selecting a program for continued development and it often right.
We recognize the vast majority of the deferred revenue on the balance sheet during the quarter.
Going forward if people recognize the remain during the second quarter <unk> exploration and the research terms in mid May.
After that collaboration revenue will mostly consist of reimbursement for activities were conducting on BMS is this path for commercialization and development of I depot and reimbursement under our see signed agreement.
Quarter over quarter revenue growth was also driven by an increase of $40 million of net sales of himself, though that Darren described earlier.
Gross to net was largely consistent with the first quarter of 29 team, but we ended the first quarter with channel inventory levels lower than needed in December both in absolute units and weeks of demand.
Cost of sales for the quarter was $533000.
Turning to operating expenses R&D for the first quarter was $91 million, a decrease of $4 million compared to the first quarter of 2019.
The year over year, decreasing R&D was largely driven by the recording of milestones for the initiation of the hope on 150 phase three study in the first quarter of 29 team.
In winding down at the tip several clarity phase three study and the phase one here to logic malignancies study.
Selling general and administrative expenses were $39 million for the first quarter, representing a 7 million dollar increase over first quarter 2019, driven by increased headcount to support our U.S. operations and the initial catered infrastructure build any you.
We ended the quarter with cash cash equivalents in marketable securities of $613 million.
As Jack you laid out to the getting into call. You recently made important resource allocation decisions to support the execution of our critical business objectives, while conserving cash and increasing our financial flexibility.
As part of this exercise we conducted a critical review of our spend across the business to capture reduced spending levels that will naturally up her can be impacted the global pandemic such as travel in clinical trials bag.
In addition, we made decision to delay or reduced spend a lower priority activities.
These include stopping development individually sixthree sex parking Selectra research programs, including our programming theatrics attack deal.
Delaying the start of longer term clinical studies that are required but not time sensitive for our main clinical programs.
Limiting hiring except for a select group roles that will help us build critical capability.
Well, it significantly reducing our contract workforce and delaying I 10 facilities infrastructure project.
It's important to note that we made these decisions given the uncertainty the current global situation with injected into the financial markets and not out of any liquidity concerns.
As a result, we now expect that our Q1 ending cash balance in addition to expected product revenue in royalties, excluding anticipated program specific milestone payments will now fund our current operating plan through June 2022.
With that operator, please open the line for question.
Thank you and I, sorry, mindshare, ladies and gentlemen, if you have a question at this time just press Star then one on your telephone keypad to withdraw your question press the pound or hash King. Please standby, while we compiled they can Iraq.
And our this question is from tighter than year end with Piper Sandler. Please go ahead.
Hey, guys. Good morning, great to hear but the increase cash runway. Thanks for taking the question I guess, a middle pivot in sickle cell would you states you know the decision on proof of concept remains on track for mid 2020 could you and I understand the data will be presented at ash.
It will we see any data when you make that decision at mid year and if so what will we see and specifically what do you need to see to make a decision in terms of proof concept or moving forward.
Chris do you want take that one.
Sure. Good morning, everybody, it's Chris found in here and.
The what you should anticipate is a topline data sharing similar to what we did with Dallas can be a data at ash at the end of last year, where we presented some efficacy data.
On on the primary endpoint of that study, which would increase in hemoglobin.
And I made some high level statements around safety I have some of you who attended our Investor event will also note that we did provide some individual patient data.
So I can't tell you precisely what will be in that announcement today, because we're going to be looking at data.
And pulling all of that together in terms of what we need to see for proof of concept that is to move too.
The next stage of development from an efficacy standpoint, there are two read outs that we're interested in one of them. It addresses the issue of the alright.
Hemodialysis and India.
And the second is related to.
Findings that we would see from the drug that could have an effect and upsilon and data occlusive crisis, So really looking at oxygen dissociation curve cyclin curves.
Looking at HCP to three DPG happening in red cells and patient you're on different doses of treatment and then of course looking out over the course of their treatment to see what changes we might see in hemoglobin as well as endpoints that would be addressing an effective retro polices sort of chronic and ongoing demolished.
It's a lot of different data that will be looking at and.
Looking forward to being able to provide so our view of that data in the middle every year.
And just as a quick follow up could you give anymore color on your decision to increase that goes to 100 milligrams and should we expect to see a meaningful number of patients on the EUR 100 mic dose at year end with some of the challenges you're experiencing in the near term enrollment.
Hard to answer I'm part two of your question at this point.
Everyone's.
Yes, I'm, hoping that.
With HM.
Measures that have been taken do it addresses pandemic they will continue to be.
And that the NIH itself.
Being that they will be able to begin to enroll patients.
They feel like they've got control on went over your Superpay think are important for.
For doing that.
With regards to 100 milligram be I'd does.
In in part of a tiny deficiency, we treated.
Those patients starting.
I agree with 300 milligram be I'd, which was the initial dose in our phase to drive PK study.
And in our activate trials now I raised our pivotal studies. We go from five to 50 milligram Metallocene me a trial, we start study to Doug.
During our data from Dallas senior and some other aspects. They now decided they wanted to study.
100 milligrams of trial.
Right and we're very appreciative of that because that will give us a greater dose ranging ics.
Experience in terms of looking at all those other endpoints I was talking about.
Great.
That's very helpful. Thanks for taking the questions.
You're welcome.
Thank you.
Our next question.
From annually.
Rolla with JP Morgan.
Hey, guys. Thanks, so much for taking the question.
On the finance side and extending the cash runway. So what's the assumption here for sort of enrollment across studies picking up in the infrastructure.
We're building continuing and it depends.
Demick is prolonged.
What are the push pull levers on the cash runway guidance, maybe being it extended potentially thanks so much.
So.
So its Jackie high on upon our Anders probably going to want to jump in on this but I'll just start at a very high level, there and I think Andrew said this in his prepared remarks there.
Some expenses that we would expect.
Because of the delight.
It's too.
Hi, a little bit AD and a very.
Minimum so I don't know Andrew if you want to add anything yet.
Sure, Yes, I think the actions that I talked about in terms of things that were delaying or pushing off those are through that runway period.
And so.
As you probably well aware, it's a pretty dynamic situation and so you network.
We're poised to react as the situation changes in so.
I think.
There are potentially other levers as we see business developments unfold.
Sure.
Throughout the period, but it just it's too hard to predict what those are going to be right. Now. So we felt comfortable that these are the right decisions to make today.
Tobias that that runway through through June of 2022.
And then you know we're closely monitoring the situation and we'll be able to kind of react if need be.
And just another quick one real quick.
So for activate and activate T. Great I think you said that.
No patients have missed the dose yet.
But what what sort of measures and strategy you have.
Haven't place to sort of mitigate any potential last follow up data over the course of the next next few months. Thanks, so much.
Okay.
Well, we've got to our team is now.
Our focus on staying in touch with investigators.
For for not just start PK, our program, but for all of our trials as much as possible.
With our CRM partners in terms of.
Tim.
Dissipating when patients are coming up two important study.
After key efficacy data key safety data and and get to keep getting drug to patients as we've been able to do.
I think there will be certainly the potential for us to have to evaluate how we would address.
Missing data, depending on how much it is and which data you're talking about whether you do it three centric very sensitivity analyses.
That could be discussed with the authorities et cetera.
But at this point the overall goal for us and bastards weaken Hal.
We've been pretty effective at keeping patients on treatment and getting the requisite.
Data that we need to collect I think one of the biggest impact you heard from us on our prepared remarks in terms of the pandemic.
Our aspects around.
Accrual.
And then actually getting in the sites to extract data out.
So that's about I think andrew's chronic enter dynamic situation. It is.
Constantly evolving some places are getting better.
Smoke is starting to clear a little bit places, where we're still just working on a day by day patient by patient site by site basis.
Okay and thanks for taking your question.
Thank you. Our next question comes from Peter Lawson with Barclays.
Hi, Thanks for taking my questions just just wanted to very revenues.
If you provide any more detail around potentially off label use you're seeing increased off label usage with it.
Scripts again longer and any changes in inventory Greg. Thank you.
Darren.
As David Yes, Darren here.
So have you talked about it we limited insight into into utilization.
In expanded expanded indication in terms of disease settings.
We are aware they will have antidotal use.
Beyond CML Michael NGL.
Glioma.
I can give you exact exact number for at this point.
In terms I think the other part of your question was about inventory.
Yes, correct, yes, three yes the.
I think Miguel.
I can shift you Andrew or it can take it.
Sure enough I'm happy to answer it.
Yes, so I think thats really just because of the shipping patterns right, but the quarter ended on Monday, and Tuesday about Monday, Tuesday, and typically legacy PC shipments and so we don't recognize revenue until so thats received so it's really just related to the vagaries of timing and shipping patterns.
Great. Thank you and then just on that.
Sorry, Im sorry, I was just as having many one thing to note is that because I think it was sort of embedded in your question.
In or Hypomethylating agents in.
And I amount. So I mean deadline is were worth noting we haven't seen an impact from the pandemic in terms of the general dynamics of how the direct continues.
Maybe just averaged just over into notice is that the key driver the drivers of growth in the in the quarter was a was a significant uptake gun and ammo.
Thats been the main driver.
Yes, good. Thank you thanks Kurt.
Just on the 20 sites the reopening.
To that takes a come back a line when do you think therapy.
Back fully on learning and is that kind of a good modeling thanks for your.
In the us.
When do we think the Chinese sites will be coming back online.
Well it looks like that.
Happening now and then I think it really one of the key factors for us.
Is to look at screening rates, because you've got a screen.
100 patients.
Slide 10 potentially.
We eligible.
Yeah.
AML patients remembered equity IC and eligible as well so thats, probably the strongest predictor for us to start to get some insight in terms of how this is ramping back up and what the impact is on the overall accrual timeline I can't emphasize that now because.
Yeah.
I think big impacts for for AML trials has that AML patients are relatively higher urgency of treatment in the oncology spectrum, where just about everybody.
Is pretty urgent.
But if you think about it really busy hospital that is just overwhelmed.
With patients with cold in dealing with that.
Enrolling a patient clinical trial versus.
Giving them standard care, maybe a decision you just have to make out pragmatism.
Great. Thanks, much very much appreciate it.
Okay.
Thank you our next question from Chris Shibutani with Cowen. Please go ahead.
Thank you and and good morning, with Miller pivots add in Dallas the opportunity.
Can you just a reminder, whether or not we're going to see any information in the mid may abstracts versus the presentation and other 13 patients that you'll have there how many will be a valuable with all the same markers in particular for hemoglobin, just a little bit more insight into what you'll be able to present. Thanks.
Well, we will have.
Some data in the abstract and that we'll certainly give us give you an opportunity to understand a little bit about that certainly we'll have more information in the.
In the presentation.
And what we plan on doing is presenting data for patients who have.
Safety data for all patients who had at.
Ladies one dose of drug.
And then we'll present efficacy data in patients who made it out 12 weeks. So when we add ash we spoke to.
Eight patients since then weve enrolled a total of 20.
And then our data cut.
Well, then dictate and then.
The duration of follow up is how many patients get in above with both how from Cowen Femia It won't be the complete dataset.
And so then we'll follow up with that but we would anticipate we have a.
A sizable portion of those patients that you'll be able to get a good center, both efficacy and safety.
Got it and then just to confirm with the sickle cell opportunity I believe there were plans for 25 patients to be enroll was that actually its.
Even if the NIH group has decided not to do the virtual presentation within moments on target.
So so the NIH study, Chris has the opportunity to.
Accrue up to 25 patients.
And I think what they're going to ultimately there they're sample size.
Is likely to be somewhere between properly probably between 15 and 25. They can evaluate 15 invaluable in software. They can go way up to 25, I think with them.
If we believe we maybe able to learn and those kinds of results.
I know guidance on when we.
We'll be able to.
To provide the results yet because that trial.
Has an open yet it's being affected by the pandemic and.
We're not going to ready to disclose the sample size on the that would depend on what we saw on healthy volunteer.
Part of the study.
Great. Thank you for all the Permian updates.
Yes.
Thank you.
Our next question comes from Alan.
Yeah young with Cantor Fitzgerald.
Hey, guys. Thanks for taking my question and hope everybody doing well over there.
Maybe a couple from me one.
Can you just talk a little bit advanced.
And then the third question is.
Around these add to activate they seemed like at least my assumption I wanted to them.
Smaller one maybe what perhaps coming earlier than the bigger one and I. Just wonder is there any sequencing in color that you can provide.
In light of the update guidance today. Thanks.
Hi, early gets Jackie so just on the in Bristol I'd.
Question data continues to be strongly partnered with.
BMS Celgene under the 2010 agreement and we continue to co promote our deferred together in the U.S. thing things are going very well with that partnership as they have for almost a decade.
We.
Next question should the.
Oh I can take up the the second question Jackie.
So regarding regarding.
The medical Alex.
Mentioned in my in my remarks earlier on a number of.
Of treat recommendations have been issued over the last month I'm trying to provide guidance to the community about how to handle the treatment of AML patients.
As the institutions practices wrestle with.
Covered by GE.
Those those treatment guidance guideline.
Yes.
Favor the use of world treatments were possible in order to minimize patient visits to the correct.
Acquisitions.
Transplants infusions all that.
The the.
So overall orals of.
Favored.
In both the new we'd like those patients as well as in the relapsed refractory patient setting.
I think.
Activating yes, yes, yes.
Yes.
So really it's Chris here that those the too.
Trials, while they may have some overlapping sites. There's there are also being run at some sites independently sounds like of only activate.
Hi, good only activate T and some sites have both.
So it is weak I can't provide you any guidance around sequencing in terms of how.
Kobin may or may not.
Impact.
Sort of our ability to get in there get data out et cetera. Our original guidance is that we would provide topline data at the end of the year for both trials answer and as I stated in my prepared remarks coal.
Hi, we continue ladies and gents.
Ken Asbury lighter and in terms of time, we ask that you. Please limit your questions to one.
Before we proceed.
Limit your questions to one our next question is from Michael Smith with Guggenheim.
Hey, guys. Thanks for taking my questions I just had one on.
AG 946 I notice.
Thats a backup molecules so to speak was designed to have.
Aroma taken inhibition relative to mid apparel led by almost just wondering if there any other corrector sdks that might be a different taking a potent.
See our selectivity or anything that might.
Hi, guys improved characteristics, even further flows mcewen, how you think what positioning that longer term.
Both in English.
We now proceeds and ours nine nine in PKD.
And then the other question was really on tips. So no.
And we heard from other companies that does thing a little bit of warehousing in some cases of for cancer drugs.
The cobot 19 crisis early on Im just wondering if you've seen anything.
And so we're able to take that molecule forward.
The same time 89.6 take several other.
Attributes. In addition, you had not having.
Our target effects underwhelming pace that.
That could make it better molecule.
Or or with patients.
Okay, great. Thank for taking my questions very helpful.
Thank you. Our next question is from Mohit Bansal with Citigroup.
Great. Thanks for taking my question I hope everybody staying safe.
Couple of question if I may monies are you seeing.
Any trends in terms of some doctors choosing dip so even over induction regimen, especially doing pandemic.
Asking from the point of view elderly patients who may be as it relates eligible for induction.
And then the second part of this has more to a floor strategy.
Sure. This this deal that you'll be edge.
Lead validation should we take this has signal of company now focusing more on the rare genetic diseases as the core franchise, while de prioritizing hematology for the future programs. Thank you.
Okay, let's start here I know.
I was just going to hop in real quick on the strategic question Darren and then let you answer because I think districts in the one is pretty fast sorry to interrupt you Darren.
No. The answer is is no I mean I think we.
No I don't think but I never we had already plans to make a decision on next steps related to DHL DHL over the course of.
This year and we came to that decision in a little bit earlier than the year, maybe than we had previously expected and the treatment landscape for both.
Non Hodgkin's lymphoma, where we were studying the drug as well as a male have both significantly changed overtime and based on that as compared to when we started this program and as we said just given the.
Lengthy time to enroll patients we decided that it we removed that particular.
Program out.
Out from the portfolio and try to partner and I personally think that if.
That drug as it ever sees a lot of day, it's probably going to be in a combination.
Regimen and if that's the way forward for it we'd rather do that.
Potentially with a partner.
So that's the most of that Darren.
Sorry.
And with regard to your question.
If you if you look at the treatment guidelines that referred to earlier, both those you buy ash and institutions that have been handling a disproportion number of publication.
Both will will our indicates that it's reasonable to consider lesson senses.
Treatment options for patients, who otherwise may have been eligible to receive intensive chemotherapy 77, three so so ivas startups OVO would would be one of those.
One of those reasonable options for those patients whether alone or in combination as we were seeing the community do spontaneously.
That said I have been I haven't heard of any.
I have received any anecdotes that.
Indicate that we could still has been industry within those in those treatments, yet, but it's still somewhat early days.
Very helpful. Thank you.
Yes.
[music].
Thank you I next question comes from Mark Breidenbach with Oppenheimer.
Hey, guys good morning, and thanks for putting the in.
A quick one probably for Chris on activate and activate T.
I'm wondering since we're looking at potential maybe six months delay.
Total topline data from from previous guidance.
Are we going to see any difference in the follow up times for the assessments of them quickly. These trials versus whats specified in the original protocols and specifically with regards to activate T. I'm wondering if.
This is the one that uses a transfusion independence and equipment not hemoglobin response and I'm wondering if you can comment on how we'll be able to de convolute any reductions and transfusions from drug versus patients simply avoiding going into the clinic as a result, but the pandemic or any thoughts.
You can given that would be very helpful.
Yeah, Okay. The.
First part is would we expect a delay and follow up.
And can't say for sure at this point.
But our our view of this is that there were a 20, you've got 27 patients and activate T and so each one of them is extremely important and so the.
One upside I. If there is the lack of a better term insight you know where each and every patient is as opposed to you.
10000 patient cardiology trial, where.
Did you just can't do that so as best as where we can tell them. We're trying really hard to stay on top of assessments and patients getting their visit thing and in touch with the investigators.
And then your question around it did they miss a visit because coated because they're there.
Outpatient clinic or their clinic, however, their position or group was a prioritize another direction, we will be able to capture that.
And.
And we'll have to see what that looks like for how many patients were actually affected in terms of analyzing that that data.
Okay. That's helpful. Thank you the only other than the only other thing I would add ins just goes with with some of the higher level of things, you're saying you know we and other companies now have developed case report form.
The capture.
Coping related events and then document team.
It's really imports.
And so when when the dust clears from this that'll be a key aspects in terms of describing a result, and if you look at the guidelines from the agencies day.
They provide a lot of.
Input on how you.