Q4 2019 Earnings Call
Ladies and gentlemen, thank you for standing by and welcome to argue next 40 years when do they didn't play not sure without <unk> fourth quarter business update conference call.
This time, all participants are in listen only mode to follow the presentation, we ask that cannot be keep us flights as directed by <unk> next nine each man there will be a question answer session to follow.
Like the introduce Beth Delgiacco, Vice President Investor Relations with remarks. Thank you. Please go ahead.
Thank you operator, a press release with our fourth quarter business update.
For 2019 financial results was issued earlier today and can be found on the news and events section of our website.
Before we begin I'd like to remind you on slide two that forward looking statements may be presented during this call. These may include statements about our future expectations clinical development regulatory timelines the potential success of our product candidate financial projections and upcoming milestones.
Actual results may differ materially from those indicated by these statements.
Our Genesis is not under any obligation to update statements regarding the future or to conform those statements in relation to actual results unless required by law.
I'm joined on the call today by Tim that Howard merits CEO of our genetics Keith was C O an air Custodies CFO.
On slide three is our agenda, Tim will start with the progress we have made towards achieving our Gen X 2021 division and executing on our broad pipeline Keith will talk through our phase three adapt trial design and provide an update on commercial launch preparation for escort take them out and generalized Miocene they brought it.
Eric will then provide financial results for the full year before turning the call back to 10 for closing remarks.
Then moved to the question and answer portion of the call.
I'll now turn the call over to Tim.
Thank you Beth and welcome everybody.
Throughout 2019, we made meaningful progress towards achieving our our Gen X 2021 vision to become a fully integrated immunology company.
This progress was across all key areas some of the business research and development commercial and corporate this is shown on slide four.
We have trials ongoing that I've got to come up in Fort indications and we'll announce it 50 indication this year.
Multiple trials are up and running and Robert Johnson collaborations in failure CMO settings, and now in high risk Mds.
Our genex from 17 will enter the clinic this quarter.
And we will be unveiling our Gen X one of my team later this year.
Olive Garden view rose funneled into our commercial franchise in neuromuscular disorders, and hematology oncology.
We recently received fast track designation from the F.D.A. for I've got to pick them up in mice teams are grievous.
With the potential for expedited review, we're doing all the necessary work to be ready for launch put off of our first drug next year.
Part of this preparation is growing our team, which we're doing globally and governance, Boston and Tokyo.
Oh, good commercial each have been hires and we are building out our fuel team, which keeps will discuss.
And importantly, following our financing in November wherever you raised over $550 million, we have to capital to support this growth and expansion.
Slide five.
Today, we want to talk about innovation, which is core to our strategy.
I think how we build and advance our pipeline from discovery to development to commercial.
We build innovation into every step of our value chain.
First innovation across our discovery efforts.
How about R&D efforts are foundational to our growth strategy as we advance when our our jenny's Twentytwenty want vision.
Through our enough defects just broke them or are you P.
We work collaboratively with leading researchers to seek out you know what's your breakthroughs typically in the form of novel targets and disease biology.
Each of our current pipeline candidates has an immunology breakthrough behind it including our lead product.
I think I'm on slide six.
Most of its part any of US yet on biology suddenly awards in building I've got to pick them up and equipping the RG one FC freckmann with uptick mutations to increase its affinity for the targets and facilitate in natural binding and into several recycling Pat.
By building innovation into the core of the molecule not only does it provide us a solid funding to state.
But it has led to unique clinical characteristics across.
Efficacy safety.
And the ability to dose in multiple formulations.
On slide seven.
She was up to some apples already created through our IP based on the funding from Aiden Ochsenbine that Cdseventy is integral to leukemic stem cell biology in a CMO.
The depth of phase one data will be produced led to a global collaboration with Johnson and the broad development plan for the candidate in ammo and high risk Mds.
Two trials under the collaboration are enrolling patients, including the pivotal phase two culminated trial evaluating cues of choosing luck with Asia in newly diagnosed CMO patients and the phase won't be evaluating various combinations of Q so to sum up between at the Clecs and is a sided in in.
Newly diagnosed ammo.
We expect more tries to start in the first type of 2020, including at high risk Mds trial evaluating queues are to sum up and Asia and the trial in Japan in Mds and demo.
We expect to have a data update in 2020 from this program.
It was approximately a one year ago that we entered into the collaboration with Johnson and we're very happy with our decision to.
Promised you have to sum up to a lot on called your peers, but all of a decision in choosing Johnson.
We have developed a communicative and collaborative relationship in building out the global development plan.
And we can say to data for your own tracking on budget without initial plan.
Our apartment assets on slide eight also stem from the RFP and provides the opportunity for non dilutive income.
These include our genic, some 14 and objects from 16, which led to the creation of I come up and Staten respectively.
Our genic swamp 12, which we licensed to Leo for dermatology indications and Docgenix 115, which we licensed to Abbvie for immuno oncology.
We reaffirmed our commitment to R&D lashes innovating to new pipeline assets from the IP.
Our gentex from 17 sockets complement components sheet too and objects from 18 targets collecting done.
We will start a phase one dose finding trial of our Genex from 17 in healthy volunteers before the end of the first quarter.
Without Jennings from 18, we are undergoing lead optimization work.
Now onto hobby innovate across I would have got thinking about clinical developments on slide nine.
We believe we're evaluating the broadest SGN antagonist pipeline among our competitors with trials ongoing m. cheap I T P P feed and CDP.
We expect to have up to five phase three trials ongoing this year and to announce it 50 indication.
Last month, we chefs phase two proof.
Concept NPD.
This was our third proof of concept success with Evercore take them up.
Validating our beachhead strategy and our conviction that I've got thinking about 3% it true pipeline products.
Slide 10.
We took a unique and effective approach with our phase two trial in TV.
Single viably evaluating the potential if I've got taken out across those monotherapy versus scramble therapy and concomitant use of corticosteroids.
With this adaptive trial design.
We were able to dialed in on the best dose combination for outages traditional trial.
The key words to considered in understanding our data on slide 11, our speed.
And consistency.
Around speed.
78% of patients achieved rapid disease control you. The median time to disease control for both mono and combination therapy or 14 to 15 days.
That completes clinical remission was observed in 70% of patients receiving an optimized dosing regimen, including weekly or bi weekly doses of 25, snicker kick up tick them out and corticosteroid doses that are sub optimal for these patients.
We saw completed clinical remissions, starting as early as two weeks and out to 10 weeks.
This is very fast for Patrick as patients.
We are hypothesize here, there's a biological rationale to the synergy in speed, which is at the corticosteroids into skin up regulate the synthesis of that doesn't Mclean auto antigens.
The data we saw from this phase two trials have been consistent with what we know about of cutting them up to date.
Yes, so a favorable tolerability profile as determined by an independent safety monitoring Committee.
This is what we have seen in phase one in healthy volunteers.
And also in Mg and IP phase two trials.
There was a consistently cleese correlation between knockdown in Gees, and the clinical benefit as measured by an improvement in the PDR score.
That's helpful in both regardless and fallacious and newly diagnosed and relaxing.
We are excited to present the full data sets.
Society for investigative dermatology annual meeting in May and to launch a registrational trial in the second half of Twentytwenty.
Slide 12.
Shifting gears to our innovative RTP program in which we believe volume it both fiber 10 make brick kick Ivy I've got to come up and our two ml subcu maintenance I've got to come up.
Just you would approach will maximize our ability to reach more patients and will provide optionality for our market access team.
We will be running two phase two trials, enabling registration with Abbott IP products and one face the incorporating the SC maintenance products.
We initiated the advanced trial evaluating about 150 primary IP patients dosed with doesn't make per kick Ivy I've got to come out.
For both induction and maintenance of platelet counts.
Primary endpoint for this trial will be is sustained platelet count response.
More than 50000, platelets, but Michael either blobs, Judy weeks 19 to 24 of the trial.
We plan to initiate a small ivy confirmatory trial called advanced too in the first half of Twentytwenty in about 50 property IP patients dosed with 10 make book kick Ivy I've got picked them up.
The primary endpoint for this trial given its smaller size will be around a cumulative duration of platelet count response.
And we will stop the advance upped your trial in the second half of Twentytwenty, that's been evaluate unmik per kick Ivy have got taking a lot for induction of platelet response, and the two amount subcu I've got to come up from maintenance.
We will share more on the details of this trial as we get closer to it starts.
Slide 13, finally, our fourth I've got thinking about indication CDP.
The second indication into neuromuscular franchise.
The phase two at he's trial is innovative its potential to seamlessly expand into either Registrational trial should we see an encouraging results on a go decision following analysis of the first 30 patients.
We also recognize some of the development hurdles with a hit Virginia's disease like CDP and added in key focus during initial stages to identify the best patients possible.
First we will be certainly have patients with active CDP and second.
We will be certain we have patients with disease driven by auto antibodies as shown by the supposed to have got they come up.
The final beaten which we innovate with the idea is that this will be the first trial that we will take forward our subcu enhance I've got taking about product at the onset enabling patients with this chronic progressive disease to heaven at home easy to administers fast injection.
I'm now going to turn the call over to our COO, Keith Woods to talk about that and that will commercial preparation Keith.
Thank you good morning, everyone.
Tim discussed we try to be both innovative and patient focused in our trial designs shown across the ATP PB and CDP programs. This is most evident in our phase three adapt designed for MG. This trial took into account our phase two data, but also the perspective of our key stakeholders.
Yes, we announced last month that our adapt phase three trial is now fully enrolled and we will report topline data mid year.
With data approaching and our recently granted fast track designation, we have been ramping up our commercial preparation effort and fine tuning some of our launch considerations.
We built adapt based on the positive data generated by our phase two trial, but also talking to patients physicians and incorporating their preferences into the design.
Our phase two data should fast onset of action with responses occurring after the first week clinically meaningful responses in 83% to patients and perhaps the most surprising we saw sustained responses of at least six weeks in 75% of the patients the 11 week trial.
Ended with the majority of responders still in response, so we were never never able to measure the full durability of the drug.
We took these data to the patients.
And we hear appreciation for the potential extend an extended period of clinical benefit between treatment cycles.
This can provide them freedom from the clinic, where they can go multiple weeks without doctors' visits, possibly go on vacation and not worry about therapy.
With adapt we set out to replicate the phase two data in a larger set of patients and to further validate efficacy and safety, but we also wanted to measure the range of durability that patients experience from the drug and potential for retreat.
On Slide 15, you will see the adapt design.
I'd ask you to focus on the Blue box around the first eight weeks of the trial.
This is the time period in which we measure the primary endpoint.
This is the portion of the trial that focuses on registration.
Here's what we did within that first eight weeks, which is essentially a replay of the phase two.
Patients will receive one treatment cycle up for weekly doses of 10 milligram per kilogram IB ETF guarded commodity.
When a patient achieved at least a two point reduction on the Mg HDL for at least four consecutive weeks. They are classified as a responder.
The primary endpoint is the percent responders at any point during this initial eight weeks compared to placebo, notably only they ask steel Colin receptor positive patients will be considered in the primary endpoint analysis.
The rest of the trial is upside.
On the left hand side of the slide you will see the upside in the enrollment criteria that extends beyond the primary endpoint population. We included patients who are as steel coleen receptor negative in the trial. So the patients with auto antibodies against MUSC L. RP for or aggregate components of the neuromuscular junctions we.
Cap these patients at 20%, but wanted to clue include them in that study because they're so often left out of clinical development.
There is also an upside and running a 26 week trial, rather than stopping it at the eight week registration point.
We wanted to keep the trial blinded and placebo controlled up to 26 weeks. So that we could measured the full durability of the effect after one treatment cycle and assess the potential for re treatment.
Having data in hand, we will be one piece of deposit.
Of how f. guarded demod integrate into the current EMG treatment paradigm, but we also want insight from the patient.
On what their current treatment paradigm really means for quality of life.
This is one of the ways, we intend to innovate and how we approach commercialization.
In the 26 week study patients are eligible for another treatment cycle as defined per patient protocol throughout the primary study patients can receive a minimum of one cycle.
So they respond to after the first treatment cycle and do not ever lose the clinically meaningful benefit or are they can receive as many as three cycles. We will learn it patients who responded during the first eight weeks, we'll respond again with Retreatment and we will learn if patients who not too.
We do not respond during the first eight weeks do risk on after a second cycle. All of this is important for the label.
We also included a 52 week open label extension trial, where Retreatment criteria remain the same but patients can start to taper off standard of care to stimulate more of a real.
World set.
Following the 26, we primary trial and the 52 week open label extension, we will have a clear view of the median number of cycles that will typically be needed in a year and this will be very powerful for our commercial team.
With adapt we believe we have set ourselves up well not only for registration, but for launching a differentiated product into the empty space. It is this type of innovation consideration that has defined how our gentex has operated to date.
If we look at slide 16, AMG is an auto immune disease in which each patient experiences the did.
These differently, we ultimately aim to treat the individualized nature of the disease with a page patient tailored dosing regimen and with this type of trial, we will be able to do so and enabling a shift away from the balancing act between symptoms and side effects that physicians and patients currently face with the standard.
Of care with Immunosuppressants.
Ed Garding them out as a first in class FC antagonists and could also be the first to market and Mg Green space can be a significant advantage, but it makes it makes it critical to have a scientific engagement with physicians on targeting fcr and and reducing auto antibodies is a rational approach and treating mg.
The role of the auto antibody is threefold in Mg They block the neuromuscular junction they internalized receptors here or there are fewer available signals for transmission and they were accrued complement.
By addressing the fundamental driver of the disease. The autoantibody. We believe we can reach a broader group of MGP patients. We also here from the patients and physicians.
That with long standing therapies, like corticosteroids broad immunosuppressants and ideology the side effects can sometimes be worse than the symptoms of the disease. Our hope is to offer efficacious therapy with clean Tolerability profile that also allows the patient some flexibility and some time away from the clinic given.
And.
A tailored dosing regimen.
We have launched a real world evidence initiative, if we have a look at slide 17 called my real World.
MG. This is the first of its kind study and LNG, we will enroll 2000 patients. These patients have not necessarily experienced f. garner demod butter patients who live everyday with the burden of suffering from MG. We've worked very closely with global patient advocacy organizations and physicians to.
Drive awareness of my real World Mg. This is a disease that needs awareness patients need a voice and they need more treatment options.
From the study, we hope to better understand patient perspectives on diagnosis treatment symptoms and the underlying economic humanistic burden of disease.
With favorable debt.
From a debt and my real World Mg, we'd be in a strong and inform position when we engaged with both payers.
And regulatory bodies about the value of Florida, not brings to patients and the healthcare system.
My real World AMG is just one aspect of our proactive approach.
And our ship to commercial organization, we started year, having heard all key commercial functions as shown on slide 18.
The commercial leadership team in place and doing the work to build out our commercial organization, including Us and Japan, We have hired medical research liaisons and key.
Agents and have also started to build out our thought leader liaison team all to be out in the field engaging with EMG physicians, we will start the hiring of Salesforce. Following phase three data and we aim to have the team in place approximately six months ahead of launch.
Our global supply chain is also on track in preparation for launch we have manufacturing capabilities with lonza that enable us to be both flexible and scope and scalable and finally, while we're embracing a speed to market approach with our I'd product, we continue to listen to that subset of patients who may.
A subcutaneous option, we look at slide 19, advancing our Subcu ENHANZE depth guard to come on.
Product on the heels Fivea corporate priority, we plan to engage it's a corporate priority and we plan to engage with the FDA.
Hey, this year to talk about what the pet forward will look like.
Before I turn the call over.
Here to Eric I want to say, what an exciting time deceased projects as we transition into a global.
Pharma company with our phase three data mid year, the filing of our BLE by the ended the year and a plan 2021 launch of our first commercial product, which would be the first launch of Fcr and antagonists with that I'd like to turn the call over to air for for a review of our financial results Eric.
Yeah.
Thank you Keith.
So we will now on slide 20 on slide 20 goes off.
Yes.
Results, which I'll detail.
Today's press release.
These findings.
Total operating income reached 82.6 million euros folder you.
Number 2019 I.
An increase of 53.4 million euros over the same period in 2000.
The increase is related to the actual recognition of the payments.
Okay.
So these fees.
Operation, Louisiana.
So to the recognition of the milestone payments.
From.
All the advancement of ABDC 151 into first in human clinical trial.
R&D expenses for the full year 2019, with 197.7 million euros.
To 83.6 million euros in 2018.
Selling general and administrative expenses with 64.6 million euros.
2019 to 27.5 million euros hold the same period in 2018.
The increases in R&D SGN expense.
2019.
Driven by the pull this made within our late stage pipeline, including higher consulting and period expenses.
Costs on manufacturing expenses and the recruitment of additional im pleased to support ongoing activities.
We expect.
<unk> expenses to increase year on year as we saw the advanced all pipeline.
For future commercialization.
For the full year 2019 financial income.
<unk> point 4 million euros compared to 3.7 million euros for the same period in 2018.
Exchange gains totaled 6.1 million euros in 2019 compared to 12.3 million euros in 2018.
The total net loss for the year.
This understood.
2019 was 163 million euros compared to 66.6 million euros in 2000.
We ended 2019 was 1.3 billion euros cash cash equivalents clearance I understood I said.
Okay up to 565 million euros in 2018.
This significant increase can be attributed to the closing of global collaboration and license agreement with Janssen in January 2019, resulting in the 300 million dollar mice.
Payments.
And $200 million.
Listeners.
And also from global offering grows in November 2019, resulting in the 479 million euros.
[music].
I'll now turn the call back to team.
Thank you Eric Slide 20 of them.
Our Genesis portfolio forget an exciting use led by our first pivotal data sets and expected to regulate the submission in energy and the advancement of our robust pipeline.
We expect to be running up to five phase three trials and seven earlier stage clinical trials this year.
All of which will continue to feed into our commercial franchises.
I would like to take this opportunity to think our dedicated and talented team the patients and physicians who participated in our studies and view our loyal shareholders without the support of each of these stakeholders, we would not be able to realize the promise of our development programs to bring new therapies to pay.
Patients with limited treatment options.
Thank you for your time today.
I'll now turn the call over to the operator for your questions.
Thank you ladies and gentlemen, we will now be next question answer session.
Next question. Please press star one.
For your name to the amount.
I mean with your question.
And for participants.
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Once again star one.
Thank you.
Our first question comes from the line.
From Bank of America. Please go ahead.
Hi, good morning, Thanks for taking my questions.
Tim maybe a quick one for you about some recent news.
Regarding our competitor it was announced that the.
Body program.
Is not being pursued if it's also something thats related mechanistically too.
Program and I was wondering what your initial thoughts were about.
That.
Decision and what it may or May not mean for you in terms of confidence for Europe, CRM program and general and then I have a follow up thanks.
Thank you it does the and thank you for being with US today and thank you for the question. So this this just highlights that not all S. Sedans are made equal something we have been calling out from the beginning.
Thank you do see between the different molecules, a clear differentiation, along the efficacy safety and convenience angle.
We are moving forward with our Subcu products.
Both India the P clinical trial and into out as the interaction 40 Mg indication.
And maybe Tim can you just give us a little bit of color on.
When in midyear I know people have been asking events.
Answer, but when you say mid year for data is that something that you can give us a little bit more granularity on.
No we can all but I think I think midyear is the best possible guidance, we can give you today.
Okay. Thanks.
Thank you.
Thank you. Your next question comes from the line is Christopher Marai from Nomura. Please.
Please ask your conference.
Hey, good morning, and thank you for taking the questions.
So first I wanted to touch upon some of the patient stratification criteria in that phase three adapt trial for Mg and the some of our Dod checks have had suggested that the beat EMG trial over tux imagine.
These patient sales due to higher seasonal rate and that may have been.
Contributed to by higher levels or.
Different levels I guess the baseline.
Steroids usage. So I was wondering if you could comment on that in the phase three trials, we have a follow up thank you.
Sure Chris its key thanks for the question.
We did strata stratify the phase three trial not only from a geographic perspective, but also from the estill coleen receptor positive to the estill cooling receptor negative and finally, we did.
Stratify based on the background concomitant therapies I just want to call out to you that in the phase II.
Where we did not stratify.
The concomitant therapy that all patients were on was for a minimum of six to 12 months and in fact, the median time for receiving I Estes was for greater than three years.
Although there was an imbalance the impact of the background therapies in the phase two did not have a result on the outcome as three of our top responders in the F. Guard ECCMID arm were one that had not previously experienced high STS kind of what happens when you have small numbers enter trial, but ultimately we have stratified for it in phase three.
Right.
Okay, and then with respect to the Steri doses at those Ben.
Sort of normalized or are they being controlled and any manner and then finally with respect to have.
The company expects to release the data should we expect to conference call. A PR and then just top line data would follow up presentations and publications or more or.
Or detailed updates from the company.
When the when the data hits. Thank you.
Yes, all I can say is that a.
Steroid dose is comparable to what we've seen in phase two.
And we will be bringing the data fourth we'll do topline first and then more in depth release at a later time.
Thank you.
Thank you. Your next question comes from the line.
From Guggenheim. Please ask your question.
Hi, guys. This is Derek on free cash.
Congrats on the quarter and thanks for taking our questions maybe just had a couple here.
How much treatment burden as a weekly hospital visits I mean, and then sort of what are you trying to achieve with subcu and enhance subcu in terms of the injection frequency.
Yes, so first of all I guess I would clarify Derek that it's not necessarily a hospital visits to have this four week period of of injections that can be done via home infusion as well, but ultimately when we spoke with the stakeholders and offered up different.
And they really preferred to maximize the durable benefit that these four doses allowed as compared to just being on a chronic therapy on a fixed schedule every other week or so so.
The.
Convenience that this allows might not be during that 22 days, but it's the benefit that exists afterward your second.
Question was in regard to Subcu first of all the Halozyme enabled subcu, we've already shared that we're utilizing NRC ATP trial and that we will be taking that forward into EMG. So that we can provide more out optionality not only for patients and for health care professionals, but also for our market access team.
I think you're asking maybe the difference between the Halozyme enabled subcu and the Subcu that we'll be using an ITC and largest patterns of how much the injection frequency could get if you could you give the what's the added duration you could get at with the Halo.
Theoretically yes at this time, we don't know the theoretical answer we did shared data at this CDP meeting in December where the possibility of going to every other week is something we're looking at.
All right.
That's very helpful. Thank you and congrats again on a good 2019.
Thank you Eric.
Thank you. Your next question comes from the line it's Derek.
Stifel. Please ask your question.
Hey, good morning, guys and thanks for taking the question I just have a few around the adapt study.
And in energy. So first off can you just give us a sense of what you'll put out with a topline in terms of the re dosing and also around the 26 week data in the phase three and then the second question would be.
Can you just remind us on the the dosing criteria in phase three so again, what sort of symptoms do patients need to actually experienced required that additional dosing and then I have one follow up.
Okay, Great Eric I'll go ahead and take this one as well.
You can expect to see with the topline data readout is all patients will have completed the 26 week trial and will show data from the primary trial will be sharing data along efficacy safety and convenience for efficacy can we replicate the phase two results safety does it look consistent with.
All of our other if current ECCMID studies and for convenience what can we accomplished.
With this patient Taylor dosing.
You asked why the 26 week trial when you have a primary endpoint at week, eight, but having a placebo controlled blinded study owner Retreatment is absolutely crucial for our payer conversations we wanted to arm the commercial.
Team with data, while keeping the primary endpoint analysis as close to phase you as possible and essentially we feel that this de risk the trial and your last question with the criteria for Retreatment, which patients needed to lose their clinically meaningful response, which was defined as a two point change from baseline LDL. This was the only way that we could.
Truly measure the full duration of the impact of FBR to come up.
Okay and then just my follow up was you mentioned on the and the in your comments around how many are I guess, how often maybe mg patients go into the office a year.
Do you have figure for that in terms of how often they are going into see their physicians and.
I guess, whether it be for dosing immunosuppressants or whatever.
No, but like just like how often are they generally seeing their physician to manage their their condition.
Yes, Eric we got data, but it's quite variable because as you know the EMG patient population, there's quite a range. So.
You are some they're seeing their physician quite frequent others.
Maybe less severe they're not seeing them as many times per year.
Hi, Thanks, guys.
Thank you.
Thank you. Your next question comes from the line of James Gordon.
Morgan. Please go ahead.
Hello, and thanks for taking the questions James Gordon from JP Morgan I actually I brought in the question asked a question on potential competitor. So the hyper sialylated data that we saw recently and I was just early data the interested in getting towards the few aspects of that data. So.
Just wanted to give me an adult billing.
It's a lot more convenient and convention I mean, it all Berlin and so you don't need to inject as much into someone could that mean, a switch from conventional amenable built into it as chairman therapy will be less compelling.
Do you think it could mean, hoping as differently so less as a acute therapy, most a chronic therapy and just the extent to which we can lead to date they've already presented.
TP to any other indications.
But to some extent that we're reading from one indication to another coming to that two of hyper sialylated or do we need to be quite careful and any thoughts on the everyday to be very interesting. Thanks.
Thank you James Thank you for joining us on the call today, So I think you call it out.
Early data I think you need to wait for more data what we have seen on the outside.
Is it kind of more efficient and IP edgy on the program basis.
But it still has the g. So the way I VIP is actually utilize Tonight GP is as you correctly save for the acute use for the rescue use that is not the use which we envisage fort I've got to come up where we think we will position to drug for chronic use and actually our key competitive that is most likely going to be at TPG to separate agonists.
If you had a second question.
Okay.
Your next question comes from the line, Jason Butler from JMP. Please ask your question.
Hi, Thanks for taking my questions I have to the first one just on 117 can you maybe just point to any PD markers are biomarkers that you're going to be focused on in this first trial and then second question on PV.
You mentioned the mechanistic.
Potential synergy with steroids have you generated any data to support this synergy in preclinical models. Thanks.
Jason Thank you for joining us. Thank you very much so PPD markets sort of Cumberland look alike on 17, and or pretty obvious I think we would be looking.
And things like freesheet too.
Could also looked at Marcus for CTX evaluation, So we will be catheter model.
Putting these sit in Marcus this one of the allegan aspects of a complement inhibitors.
It allowed us to basically optimize the dosing regimen in phase two healthy volunteers.
The.
Second question remind me most relating to walk.
TV and just have you any is going to the preclinical data supporting the synergy.
So that is limited should they have done that academic groups and I think.
These are public public data in the literature, but actually it's variables shown that's corticosteroids up regulate the synthesis of this mclean Walmart and as Mclean CD all to averages NPV and does the only actually to explain the rapid onset of action also corticosteroids in in a disease.
Like TV.
And we believe that is.
A hypothesis she is that the mode of action of adapting and loved community Synergizes with this model of action if you take away the toxic pressure.
Some of the ultra antibody going after those modine warm and smuggling three and you apparently the synthesis that could explain why this is such a fast closing of the lesions.
Okay, great. Thanks for taking the questions.
Thank you.
Thank you. Your next question comes from the line with Matthew Harrison from Morgan Stanley. Please ask your question.
Hi, Thank you Mrs. Max on for Matthew Harrison can you confirm or can you provide an update on enrollments for the CDP trial have you confirmed auto antibody involvement in any patients yet.
Thank you for taking the question.
Thank you for joining us on the call today. So no we have not been public on any update in the said the trial and I think.
Like update you can expect from the company is and when we approach to 30 patient go local.
Okay. Thank you.
Thank you.
Thank you. Your next question comes from the line of questioning why are you from Wolfe Research. Please ask your question.
Hey, guys, so given that higher AMG trials kind of use that mgd I'll drop as your primary endpoint, what's the FDA thinking on adapt if it hits on the primary responder analysis, but misses on MD 80 drops and have you gotten feedback from any callouts on the clinical meaningfulness of that endpoint and also on.
As a body just got acts and then momenta disclose on their earnings side yesterday that they'll have a weekly subcu dose based on our colleagues that means that momentum is going to have maybe a five make for keurig, maybe at most headache for keurig on their weekly arm and we don't really see momentous potency drop until 15 at 30. So is it fair.
To say you guys should be the only fcr and that can have a biweekly subcu going forward. Thanks.
Yes. Thank you for this question so from from an apartment endpoints point of view.
Yes sure.
Has been testing and above the endpoint with and both the FDA and the PMT. So the delta of two points on an LTL score is indeed widely accepted thus clinically meaningful.
And the four consecutive visits requiring the top of two points or more.
Actually it hasn't volume from the PMTA I cannot comment on how the FDA able to react to the data in case, we would be missing on this end points.
Yes, maybe escorting on some of the over and bonds I think it's premature to talk about.
Subcu dosing.
I think of hitting the lump all the heavy if so everybody is using the word subcu dosing, but you need to make a difference between injection and infusion I think all as shown on thank goodness, and which do not have access to the halozyme technology would either have to refer to multiple subcu.
Injections or a bigger volumes sub June fusion I think momenta is not an exception to this so to answer your question, Yes, I think we'd likely the only back to here within every other week subcu injection.
The thousands milligram per kilogram data hold true.
Thanks, so much.
Thank you.
Your next question comes from the line of Seattle slots from Credit Suisse. Please ask income.
Hi, guys. Thanks for taking the question just quick follow up on the bringing strategy sporting has formulation. So I know you're going to engage with the FDA. During this year, but curious if it could outlined some of the scenarios and what could that imply from.
Hi, My perspective on getting subcutaneous in as far in relation to Jim GE stations. Following the launch thanks.
Hi, This is Keith.
Thanks for the question.
Scenarios that could be in play and what we actively look forward to discussing with the FDA is because such we since we have such a perfect biomarker.
With GE.
Looking at PK PD in healthy volunteers would be our first in preferred option to discuss with them since we can prove.
Noninferiority situation NPD.
The other case scenario could be that we need to do PK PD in EMG patients.
And finally, I would say that the.
Situation that would be not our preferred would be having to run a full phase three we do know that the FDA has gotten much more comfortable with the enhanced technology, but we're not going to guestimate until we've had that meeting, but thats pretty much your three scenarios.
Fair enough. Thanks.
Thank you. Your next question comes from the line of year when were aware from Cowen. Please ask your comes.
Great. Thanks for taking a couple of questions. Let me sit on line one isn't adapt the other one is through the to them. So.
So just in the depth.
Your Oragenics isn't the lead obviously it is also the first company that's doing this individualized so to tailor dosing regimen.
The company's like momentum I may be mute event or testing more of a fixed dosing scheme. So now that you're going to heaven Haynes and sub Q. I mean, you you'll have sort of the ability to to go to market with this individualized dosing regimen, but would you consider also doing a fixed dosing regimen if need be give.
And that you're going to have a subcu just in case, they a more chronic dosing would would boost efficacy and then of another follow on.
Hey, this is Tim speaking so.
I think it will depend a little bit on the update me I call out that and our colleagues into space like Momenta and immune event or still in phase two so I think thats still testing some of the basic characteristics of the molecules and west is still need to craft. The fish. These strategies, we really crafted the face these tenants.
On the strength of our molecule.
I don't think we should extrapolate between molecules by the way.
But I think the phase two data for that will be telling.
You're absolutely right next to the people that we have a second shot on goal that the subcu product, which is giving us in principle more dosing optionality. So that the data speak and then we will adjust to that.
Okay, and then question of culminated in because it to some of which is a program that Jay when those very serious about.
The culminates study potentially I think you're mentioning it potentially could be pivotals 150 patients.
In patients who are not candidates for intensive chemotherapy or in the Eva AML patients.
The study with as it will be days, but the study does not have a control.
And from what I can tell the primary CR in the second there is a CR OID MRV at least what some clin trials. It Doesnt look look PFS in survival or soda secondaries. So I guess my question is are you looking at PFS and survival and then what would make that into a pivotal study just given that venclexta.
The fruit I mean I know there's.
Lot of enthusiasm for the drug and were very enthusiastic to agenda is moving very quickly, but I just want to understand this phase two trial design. Thank you.
Thank you for the question and to be honest I don't know the company NASA to the question because it will hinge on the data. So you're absolutely right that is no control are now for the and Venetoclax.
Five days a combination trial the phase two study based somebody's. They file there was also no control arm and actually in today's world, It's getting increasingly difficult when just enroll patients in a and five days arm as a control.
And whether this trial would qualify for legislation trial will actually depend on the strength of the data. So I believed that our bottling Jensen called the study registration directed.
That means that that is a believed that if the data would be strong at least that has a conversation to be hope with the FDA.
Similar to the conversation they had all the that the classified days a combination data.
Does that make sense.
Yes, It does and then pre clinically if theres synergies between because of tourism Abbott as another clocks and maybe we should just can discuss is there any overlapping toxicity. Thank you.
Yes, Im glad you asked the question because this is this is the excitement behind the combination trial, we'll be testing queues in combination with venetoclax with and without by data.
The Ash post the switch we presented last December.
Basically highlights the preclinical work demonstrating a synergy between queues and Venetoclax, which is even stronger than the synergy, which we already document that's for cues of it by data.
So that has a tremendous amount of excitement around these data because we think that the future of ammo is going to be combination therapy, where on the one hand, you push button as high this postpaid as possible CR.
And you're trying to maximize the job.
Correlation of your response, we estimate maintain our controlling Mississippi and that's I think let's use of doosan up could be such a strong combination partners because with this unique mode of action. It synergizes with standard of cash, but it's also set up the which due to its favorable safety and tolerability profile could be the backbone of low.
With them therapy.
Thank you.
Thank you. Your next question comes from the line of Greg.
From Goldman Sachs. Please ask your question.
Okay. Thank you thank for taking my questions and congrats on all the progress in 2019.
I also want to add.
I guess I want applaud you for them my real World Mg trial.
And maybe the question is.
This is for Eric.
Im effort for Keith.
What specifically can you gain from that study.
That would.
Ill add to the commercial story for effort to Jamaat I just wanted to be more granular and how we should be looking at that study.
And then maybe a question for Eric as it relates to.
2020, and financial guidance I would expect a significant step up in SGN and the second half as you kind of hopefully buildout of commercial sales force filing positive phase three data and EMG, but how should we be thinking about.
Perhaps R&D throughout.
2020, thank you.
Great great. Thanks, I'll start and handed over to Eric.
The reason why my real World Mg is import.
Britain.
It's really for us to build on the value proposition of up Kartik Amod right now theres not a lot of research that goes into the impact of what AMG is doing to a patient and their life what.
Does it mean for the amount of treatment they were seeing the hospitalizations the burden of having the disease on not only exam, but sometimes the caregiver that they have the payer doesn't know much about this so if we documented in a real and then apply that to a real economic model, we're going to be able to a different.
Hey, I forgot my treat patients from what historically taking place in these patients. This should set us up very well as we go to build value dossiers to be utilized across the globe. The second thing is it's very important to us today.
I, just say where patient focused organization, but to be a patient focused organization and this is also a commitment to their space. It's a commitment to the EMG patients and we want to give the patients of voice because the data will be available to the patient advocacy organizations.
Eric you want to answer the second question.
Absolutely. So thank you for the question.
So, yes, we expect to significantly increase.
On rates Twentytwenty, we did not give any guidance.
You should look for as Ginny expenses.
What we have done these beat my still approached.
So it means that we're going to wait until we have the results of all lined up study to accelerate on the recruitment of good commercial team we have already.
Could shift and key positions.
The real acceleration is going to be up until we have the results from adapt and regarding on.
Consider to seeing.
You should look at 2019, we had on the once these two trials.
In.
2020, we are planning to hop up to five.
History trials, so I mean, a significant increase so this is going to do reflect TWC in R&D expenses and the civil element to consider is that.
Yeltsin collaboration so we expect to close to two significant.
Also.
You will see a lot of clinical trials talking into its twitchy.
Hi, Thank you very much.
Thank you.
Thank you. Your next question comes from the line.
From Barclays. Please.
Yes.
Hi, I was wondering if you just comment on and Youre thinking about sort of a holistic real world.
Taking that approach NMG on one of your Fcr on competitors has.
Talked about Fcr Indian complementary with with.
Compliment therapies and that there could be perhaps more of a portfolio like approach in treating AMG, which I know this kind of and in some of the way said that approach has taken on the wireless starting an eye TP I could just I was just wondering how your views on that.
If you think that for most Mg patience and ask our tenant regimen with would be sufficient. Thank you.
So let me take Thats question I think again it will depend on the adept phase two data.
Theoretically we would argue that the ultimate antibody is causal to the disease and one of the multiple modes of action. It has.
Involved recruitment of complements so we believe that by eliminating the ultimate antibody you don't need actually a complement blocker on top of that.
But we need to mislead that data and see how much room, they still for improvements and after we had a chance.
To see adopting with an action for sure complement book or cannot address.
The 20% so to go into separate ultra antibody negative patients, which by the way out in very high unmet need because the ultimate antibody involved is certainly not recruiting complement.
Thank you.
Thank you.
Thank you. Your next question comes from the line of it sounds like calling birds from TD Securities. Please ask your question.
Hi, first my congrats to the whole team nice progress in 2019, and looking forward to all of the Readouts coming through we not 2020. My first question. While there's two questions left the first one is actually on the ATP indication so with regard to the Subcu trial.
That you plan to initiate so the kumble IP so Q.
I fully understand then respect effect that you cannot give any details on the exact trial design, but I would like to understand.
Oh, you will actually tried to tailor made this to the patient need and the response. So how can you measure well the proper maintenance dose would be and to what this expense does this still require a hands on monitoring in a hospital setting also relating a little bit to the CP like on this question.
Earlier in the second question I would like to go back to because I presume up and Venetoclax a rational.
It's quite interesting to see the acceleration.
Going on in the hands of young some and actually the effect that you can combine it with the VH premium metric I was wondering if there is actually opens up doors to up towards other rational combinations are you involved in that work or is this fully in the hands of Yeltsin. Thank you.
Hello, Thank you for joining us on the call the answer to your IP question goes as follows the biomarker of course to monetize whether patients aren't stay in response to spaces counts.
And I think if you go to more severe PPD patients would be another that they frequently efficient that physician to has platelet count checks. So it's a relatively easy practice and by the way that is effect this which is already being used today for the current IP medication.
Concerning youre just to sum up question I think we have been saying in public that actually and subject of the.
Global development plans the Janssen is to find the test different patient subsets and ammo, but also all the logical combinations. So yes. We are actively involved in exploring combinations and you'll see more combinations coming out of the gate in the future.
Okay. Thank you.
That's it.
Thank you. Your last question comes from the line of Yamin Xu from Wells Fargo. Please ask your question.
Hi, Thanks for fitting in and congratulations on the progress last year.
So first a question regarding the adapt a trial patient population baseline characteristics.
Could you.
Broadly.
Talk about whether in terms of the disease severity and background treatment.
Or concurrent treatment, whether those are all in line with your phase two population.
I understand that the antibody or status is different but other than that.
Are those that characteristics.
Thirdly lie and particularly also.
Whether prior IBG plex patients are allowed in either of those trials. Thanks.
Thank you for the.
A question that the baseline characteristics inclusion exclusion criteria and are almost identical to devons switched redeployed into phase two trial, so expect patients to be on background medication and to see either placebo order pickup taking about being dose on top of stand of cash standard of care can involved.
The difficulty in yesterday's inhibitors, and steroids, and then ice tea slight Michael Federal Eights and is the fire, Brian Cyclo sport and on the likes.
Yes, she used to pick some up use equally as an abuse and is permits historically, but we need to respect to shows and washout period. Its before patients can come on study again identical to what we did for the phase two.
I think the slight difference between the patient population in the adapt trial and the phase two proof of concept study. It's when you look at the end ZFP classification of the patients and bit including I've also patients which are in class for b.
Which from in the medical point of view is probably not important but.
Just any sort of broad label.
Got it got it. Thank you very helpful and also Oh, sorry, if I missed this earlier, but I think that there was earlier question on re dozing criteria in adapt.
Presumably is symptom based.
And just wonder if you could talk more about how is read dosing.
The need for closing decided.
So in order to measure the full duration of benefits.
Good to have an objective measure for work.
Good morning, before you can read those thats all specified in the portal think of worsening on the L. score, which is also the score we use for the primary endpoint, but the protocol is specifying the deals and we have not really.
Disclosed those details.
I see and lastly.
10 patients receive back to back for weekly infusions at a later part of the trial after the eight week a primary analysis period.
No. They they cannot so during the 26 week study you've got the patient would have to have at least a period of four week interval between the first cycle in the second which is why Mitch mentioned in the prepared statements I think we'll see a scenario of some patients that experienced only one cycle of therapy and have this is.
Extended duration of benefit you'll see some that have time for two and a complete non responder, which is where you're asking about on the back to back or a placebo patients non response could maximize as many as three over that 26 week period.
Got it very helpful. Thank you.
Thank you.
Thank you there are no further questions at this time I'll now turn the call basketball.
This concludes the call today, we want to thank you for your time and ongoing support we look forward to an exciting year and updating you on our progress.
Thank you that this concludes our conference for today. Thank you all for participating you May August.
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