Q2 2020 Momenta Pharmaceuticals Inc Earnings Call

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Good morning, everyone and welcome to the momentum pharmaceutical second quarter of 2020 earnings conference call. All participants will be in a listen-only mode. You need assistance placing all-conference Special by pressing the star key followed by zero after today's presentation. There will be an opportunity to ask questions to ask a question. You may press star and then one withdraw your question press star into it also know today's event is being recorded.

At this time, I'd like to turn the conference call over to Patti eisenhauer vice president of investor relations and corporate Communications, ma'am, please go ahead. Thank you, Jamie and good morning everyone else. Thank you for joining us today for mementos conference call to discuss Financial results and operational highlights for the second quarter of 2020 today's call is being webcast and will be available for replay on the investigation section of our website momentous Farm of joining me on the call. We prepared remarks are Craig wheeler president and chief executive officer and Jung Kwan our chief financial and business officer also available for the Q&A portion of the call or Santiago Arroyo our chief medical officer and Tony Manny our chief scientific officer following our remarks. We were both in the call for questions.

Before we begin I'd like to mention that our call will contain forward-looking statements about our financial Outlook business plans and objectives and other future events and developments including statements about the timing of regulatory filings and meetings for clinical development and marketing approvals the market potential and reception of our products and product candidates potential competition and revenues for our product candidates product issues and strategy schools and Timeline Design timing and goals of clinical trials and availability timing and announcement of data and results. They use efficacy safety potency posting tolerability convenience and Commercial potential of our product candidates, including their potential as best-in-class agents hypotheses regarding certain effects of our product candidates in clinical study and non-gaap operating expense guidance, including our anticipated collaborative revenues and restructuring charges. These statements are subject to risks and uncertainties that may cause actual results.

To differ materially from those projected these risks and uncertainties include the risk of the impact of covid-19 pandemic on the timing enrollment or results of our clinical trials operating expenses and the supply of our manufactured drug materials the final and quality control verification of interim data and related Alice. He's those described in the slide entitled cautionary note regarding forward-looking statements included in the presentation accompanying this call and Under The Heading risk factors in our most recent quarterly report on form 10-q filed with the Securities and Exchange Commission as well as other documents that we may file from time to time with the SEC any forward-looking statements speak only as of today's date and we assume no obligation to update any forward-looking statement made on today's call.

On the call we may also.

Discuss first quarter 2029 gaap operating expense. Please see the presentation accompanying the call for further information and Reconciliation of this measure with that. I will now turn the call over to Craig.

Thank you Patty and good morning everyone. I hope you're all doing well during these challenging times momentum continues to prioritize the health and safety of our staff health care workers and patients as we persist through this ongoing covid-19 pandemic while we continue to implement the necessary measures. We have made progress in advancing our portfolio as I'll touch on in a moment. I'm following my update Yong Kwon our chief financial and business officer will discuss our second quarter 2020 Financial results after which I'll closes the final comments and then we'll open the call to your questions.

As most of you know, what mental has three novel drug candidates in the clinic for autoimmune and rare disease. Each of our molecules has been designed to possess best-in-class properties and franchise potential with applicability across a wide range of immune-mediated diseases. Our research pipeline is also advancing as momentum continues to leverage our platform Technologies to build our pipeline for the future.

This past quarter has been an exciting one for our company. We released top-line data from an interim analysis of our Phase 2 of of acid EMG study which demonstrated proof-of-concept for nipple KO Babin my senior gravis, and we recently completed the part B of our for part phase two trial of m254 in ITP. We are now wrapping up our final data analysis Part B, and look forward to updating you later in the third quarter.

Let me now dive into the details of these programs and further updates across the portfolio.

Begin today with Nicola map are fcrn candidate and a brief review of the top-line interim date of of vivacity mg.

We designed to offer a best-in-class profile with potentially Superior safety efficacy and convenience versus standard of care agents in m g as well as other agents in the same class. Our primary goals provided facet EMG were to develop a full understanding of the relationship between IGG levels and efficacy as measured by a d l score.

To explore the dosing and efficacy advantages that Network Alabama's maximal IGG lowering long dosing intervals and Rapid infusion characteristics might deliver.

And to further demonstrate the strong safety profile of this molecule.

Our results demonstrated proof-of-concept and mg provided a blueprint for subcutaneous formulation and provided a strong dosing correlation that lays the groundwork to pursue nipple calendar in other applications.

the data

Represented in June that's all of our objectives achieved a rapid and significant lowering of IGG autoantibodies which translated into durable reductions in the mg activities of daily living scores across all doses tested specifically the trial met its primary efficacy endpoint showing a highly statistical significant and linear correlation between IGG lowering and durable MGA reductions with a P value of less than point zero zero zero one.

This is always been a hypothesis and bodes very well for our drug candidate. Has it demonstrated the maximum IGG reduction possible about 85%

I won't go through all of the data here, but as a reminder nipple demonstrated efficacy across all doses tested with pooled data showing a durable response of 51.9% versus 15.4% placebo. That's a 36.5% improvement with a statistical P value of 017.

Our highest two doses 30 minutes per gig monthly and 64 gig every two weeks showed average Improvement of 3.9 points on the scale at the two-month primary endpoint measurement.

And it was also well tolerated with no infusion reactions and no withdrawal from trial due to Adverse Events.

Yep, that continues to show a best-in-class profile, which we expect will help position it if approved as the treatment of choice in mg. We observed efficacious once-monthly. I be dosing with a benign same profile and based on our early modeling. We believe what we've learned about. The dosing relationship should enable us to deliver a strong efficacy with a relatively low-volume Sub-Q options. We're currently compiling the full dataset which includes data from the eight-week follow-up. And expect to present these results at a medical conference later this year.

Furthermore we are preparing for an end-of-phase to meeting with Regulators before the end of the year which together with these results will inform the design of our phase 3 trial which were talking to initiate in the first quarter of 2021.

As you know, we have two other ongoing Trials of Macallan up. The first is Unity our Global multicenter Phase 2 clinical study of nipple in hemolytic disease of the newborn.

This is a major differentiator for us as we have the only company pursuing fcr inhibition in fetal maternal disorders in the clinic.

We believe in Apple Maps ability to maintain full receptor occupancy total blockage of pathogenic Auto and antibodies from Crossing through the placenta from the mother to the fetus.

We are continuing to treat patients already enrolled and due to the life-threatening nature of this study continues to enroll new patients at sites where they can be safely accommodated currently our European sites are enrolling patients and our us sites are expected to reopen later this year.

We are about halfway through and almond and we remain on track for proof-of-concept data from this study in 2021.

If successful this proof-of-concept study has the potential to transform the treatment landscape in HD FN importantly, you should also validate our approach in fetal tissue disorders more broadly. If we demonstrate that we can safely block antibody transfer across the placenta to the fetus. We expect these results would be duplicated in studies with any pathogen wage or autoantibody.

Moreover this data could open the door to reproductive freedom for the 30% of patients who are females of child-bearing age and took his population could help make our drug candidate the agent of choice for prescribing Physicians.

This is a Cornerstone of our strategy to build into the leading fcrn agent in the class.

Of note. We recently reported that the usfda has granted both rare pediatric disease and oral drug designations for nipple h d f n

Shows the agency's recognition of the significant unmet need in this population and offers momentum various benefits, including extended patent, exclusivity and eligibility for a priority Belcher.

Our third ongoing study of nipples tap the energy study is an Adaptive phase two or three clinical study and hemolytic anemia.

We have the most advanced fcr on candidate in the syndication. We continue to activate one of those sites globally, but due to the covid-19 pandemic patient enrollment remains suspended long. We anticipate reopening patient enrollment in the fourth quarter and have amended the study protocol to provide patients the flexibility to have some of their study visits at home in order to best protect our stuff just happens and Healthcare staff.

Topline data from this study is expected around the end of 2022.

Are successful Phase 2 study and the Broad Safety database that we are developing across our trials provide a strong foundation for us to confidently Advance our broader strategy for this molecule.

Our initial indications mg hemolytic anemia and hdfn were selected to demonstrate proof-of-concept in smaller patient populations or biomarkers are clear.

In the future, we plan to explore a broader landscape with nipple Callum at our goal is to add a new indication every 12 months or so.

We will look too.

Broaden our autoimmune franchise by expanding into the Visas in different therapeutic categories such as Dermatology or Rheumatology and potentially explore larger indications, which fall outside of the rare disease categories. We also plan to expand indications in the Maternal Fetal space.

Now I'll talk about em to 5 for our hypothyroid waited IVIG candidate.

M254 is a novel agent manufactured from IVIG. We enzymatic remodify IVIG to change the structure of the Glock hands on you and I G G by extending the effing like hands and terminating them with psychedelic acids.

Our goal and developing em 2544 ITP is to establish a high-potency agent that could reduce dosing and infusion times versus IVIG while maintaining i d i g s l i see we are currently conducting a multi-part phase 1/2 clinical trial in idiopathic thrombocytopenic purpura or ITP wrapping up our data review for Part B & Hammer dated enrollment of patients in part C.

As a reminder Part, B as a signal seeking study composed of a single sending dose cohort of ITP patients each followed by a standard 1000 dose of IVIG wage. Its intended to provide an initial look at safety and efficacy and to Aid Us in dough selection for a larger cohort in part C.

Part C is a crossover design which believe we have the two most promising doses from part be in a larger cohort of patients.

We intend to hold a conference call before the end of this quarter to discuss the data from part be early data. We shared in January from partly showed clinically meaningful platelet responses in Approximately 80% of patients in line with a response rates and it all doses tested from 43 to 250 make per gig. We also noted that there was some variability in performance versus i v a g off early data.

Based on this initial data set. We made the decision to expand Part B to explore a lower dose cohorts and to add patients to each of the previously tested cohorts to better understand if we could detect a dose-response in this highly variable population.

As a reminder the primary endpoint for this trial is platelet levels over 50000 per microliter and arise from Baseline of at least $20,000 per microliter off the data continues to show good efficacy as well as variability in performance versus IVIG.

This drove our decision to advance depart see with its randomized crossover design and larger ten patient cohorts for each of two doses.

Parts he will help us better understand those performance as well as provide data on the possible effects from the order of dosing.

From a commercial standpoint ITP is an acute indication where most patients are hospitalized and speed of onset and a high response rate are important for clinicians. Once patients are stabilized. They would burn typically transition to other therapies such as people Agonist for maintenance.

We're also beginning to plan for a phase two study and chronic inflammatory demyelinating polyneuropathy or cidp pending the results from part C of our face to ITP. Trial. Our goal is to initiate a study in 2021.

Next I'd like to share an update on an exploration of the potential benefit of neck. You have to help fight code on RMG call and June. We disclosed that we were researching that you parented a novel drug candidate. We had previously tested and pancreatic cancer to evaluate if it could block covid-19 virus by binding to the spike protein wage recent academic Publications have reported a demonstrated therapeutic rationale for Heparin based drugs potentially delivering strong antiviral activity in addition to the established anticoagulant anti-inflammatory properties of heparins.

in the laboratory assays Heparin was reported two to four times more potency and blocking the ability of SARS to infect respiratory cells when compared with Gilead disappear, if our drug shows similar benefits to other efforts being tested in the clinic, we believe it could have multiple advantages including

1st. Nicky parent was designed to be a low-molecular-weight agent that retains many of the protein binding properties of Heparin without the anticoagulant binding sequence the low molecular wage provide many advantages including reliable Sub-Q and potentially a nebulized delivered to the lung if nebulized drug is needed.

Second one of the big drawbacks of most have friends is that they are anti-coagulants so they cannot be dosed at high levels as they will cause bleeding.

Our agent is a non anticoagulant Heparin derivative designed to be used in high doses for cancer patients without causing excessive anticoagulation.

Sir, our agent already has a full toxicology package GMP manufacturing supply-chain for drug substance and Drug product and demonstrated safety and Phase 1 and 2 months clinical trials. So it could be Advanced faster than other new agents being tested to date. We have confirmed the ability of neck. You care enough to bind the SARS to Cove to spike protein, which is the protein used by the coronavirus to enter in effect cells. We have now begun cell-based assays with test viruses to see if thank you parents can block viral infection of the cell.

We have seen a strong ability to block infections using our SV as a test virus and now Advanced to testing the drug with coronavirus has we're excited with the progress to date and we'll keep you updated.

Now touch briefly On Em 2:30 and m710 are to partner programs for m230 our third clinical candidate our collaboration partner CSL has been approved by m a r a to initiate a phase one study evaluating the safety and tolerability of a subcutaneous form of em to Thirty in healthy volunteers.

We've noted previously a Sub-Q formulation provides meaningful advantages including self and home Administration for autoimmune patients many of whom require chronic life-long treatments of their conditions.

CSL expects to begin this study before the end of this year CSL also formally terminated the IV formulation of them to 30.

Our other partner program is I'm 710 a biosimilar to Aaliyah Mile and expects to complete phase three enrollment by year-end and 250 2021.

Frequently we continue to advance him to sixty seven through ind-enabling studies. This is our first program to utilize our body technology, which uses are multiple choice to enhance and antibodies FC dependent and compliment affect your functions to effectively deplete target cells.

M 267 Fabric and binds to cd38 a Target on plasmacyte responsible for generating Auto antibodies and and protein.

Preclinical data suggest this candidate has the potential to the best-in-class therapeutic for management of plasmacyte mediated diseases such as multiple. Myeloma amyloidosis and rare Auto antibody mediated diseases.

We recently demonstrated that m267 has high bioavailability and activity following the subcutaneous Administration and we plan to introduce a Sub-Q version early in the development program long. We expect to submit an IND in 2021 and we are confident this will provide significant opportunities another high-value program with franchise potential.

With that, I'll now turn the call over too young to review our second quarter 2020 Financial results Young.

Thanks very much, Greg and good morning, everyone with regard to the company's financials while we have had to adjust some of our clinical plans in 2020 due to the covid-19 situation. We continue to believe we should have sufficient cash to fund operations through at least the third quarter of 2021.

In the second quarter, we reported a net loss of fifty-seven million compared to a net loss of $140 million for the same quarter last year.

A decrease was primarily due to lower G&A costs offset by increased manufacturing and clinical trial development costs.

The reduction in net loss also reflects a 42 point nine billion dollar manufacturing charge. We took in the second quarter of 2019.

Product revenue for the second quarter total 6.6 million compared with 3.3 million for the same. In 2019. The increase was primarily due to home net sales of l'tovah R&D revenue for the second quarter was less than zero point 1 million compared to one point eight million for the same period in 2019 bought the decrease was primarily due to lower reimbursement revenue for expenses and lower Revenue recognized from Islands upfront payment associated with a similar collaboration.

Second-quarter total gaap operating expenses 464 million compared to 121.8 million for the same period in 2019.

Second-quarter Rd expense increase the 38.8 million compared to thirty two point 1 million in the same period in 2019.

This was primarily due to an increase in manufacturing and clinical trial costs for m254 and an increase in share-based compensation expense also in part by lower lease costs.

Second-quarter GNA expense decreased to twenty five point three million compared to forty six point six million in the same period in two thousand and nineteen. This was primarily due to a payment of 21 million in June of 2019 reflecting the company's portion of a settlement payment lower legal fees and lower depreciation at rent costs due to the modification to the bench Street lease in 2019 partially offset by an increase in share-based compensation expense.

For the second quarter of 2020 our non-gaap operating expense was 44.6 Million as a reminder. Our non-gaap operating expense is defined as total operating expenses, stock-based compensation restructuring costs and collaborative reimbursement revenues.

Finally, we ended the second quarter with four hundred fifty point six million in cash cash equivalents and marketable securities compared to 545.1 million months of the year.

Turning now to our guidance for 2020 due to decreased clinical trial enrollment Trends as a result of the covid-19 pandemic. We expect our full-year wage. Gap operating expenses will be lower than the range of 220 to 240 million has previously guided for 20 20 and will now be in the range of 200 to 220 million.

Sorry, I was on mute. Thanks young as you can. See we remain in a strong corporate position and despite the challenging backdrop covid-19 you to make meaningful progress across our portfolio in particular with our first proof-of-concept data for nipple Kalam app.

We look forward to reporting data from our part B of the phase 1/2 study of em to 5 for an ITP later this quarter along with the complete results from a vested EMG later this year.

I look forward to.

Keeping you updated on all of our progress and thank you again for joining us. Now. Turn the call back to the operator Q&A underway.

Ladies and gentlemen at this time. We will begin the question-and-answer session to ask a question. You may press star and then one using a touch-tone telephone to withdraw your questions. You may press star in two months. If you are using a speakerphone, we do ask that you please pick up the handset before pressing the numbers to ensure the best sound quality us again, that is star and then one task or question.

And our first question today comes from Derek is Sheila from stifel, please go ahead with your question.

Hey, good morning guys and congrats on the progress just a couple of questions on m254 then one on so just on empty 54 first. So you noted initiated Parts in a trial. Just wanted to see if you could provide any color on the high and low dose you might have brought forward there. Um the second one being can you just remind us on empty 54 in terms of the patent coverage you have their home. We saw some recent patent filings that included some of the earlier data from earlier this year for the 43 big party. So just wanted to get your inner thoughts there and then lastly on nipple county map. Um, when did we could get an update on the Sub-Q development strategy for that molecule. Thanks.

Sure, and I think I've mastered my mute button there. So thanks. Yeah on part C. I think you can take confidence that they we have taken forward doses in part be home. And I I was mentioning that we continue to see consistent results. I'm not going to talk about the specific Doses and details because it's kind of we want to make sure we have all the data analysis wrapped up and then talking about the whole trial but we did see continued to see in a good results. And so that gave us the confidence to advance into Parts see in terms of the patent coverage as we said before we have patents and we'll continue to build our patent wage state. We also have a fair amount of know-how in what's a very complex manufacturing process to be able to titrate this enzymatic reaction properly. So so we feel we have, you know, pretty good pretty good coverage. I really don't have all the details in front of me of all the different patterns and and how they're working. But yeah, we'll we'll continue to build that patent to state but also make sure we keep that uh, you know Trade Secrets as tight as we can on some of the manufacturing process wage.

Terms of Sub-Q, you know, we we probably will talk about it when we put it into the clinic. I mean, we don't want too much competitively but we're looking at a whole series of things that I think about what that program and once we now are are Doses and they are bridge study. That's probably when we'll talk about it. We have done the formulation and we know we can formulate quite high concentrations, you know, just as often as shown in our modeling indicates that we should be able to get quite sufficient volumes up cues, but we're we're going to hold off on that until we specifically have the bridge trial designed and then we'll talk about all the details with you guys. Got it. Great. Thanks, Greg and congrats again on the progress here Hector.

and our next question comes from Robin Curtis from

Yes, please. Go ahead with your question.

Good attempts. Thank you so much for taking my question and I appreciate it just to follow up a little bit on the 250 for when you when you push forward. Are you are you confident in your cost of goods that that it will be affordable for you to manufacture the process and then regarding FDR end when you look at like your Doses and as the data came through with the idea with the idg lowering, how are you thinking about what the profile might look like? What would be the ideal profile that you'd like to take forward for the ivy as far as those frequency? And how many different doses would you like to offer. Thank you. Sure. Thanks. Yeah for yeah, we are quite confident in the cost of goods, you know as I you know again, I'm trying not to talk to about too much of the details of it. But we told you may we continue to see uh, you know results similar to what we saw before and if you recall we were seeing strong results across all of our our those go towards um, so we we continue to feel that you know, we are well with

An effective cost of goods for the doses that we're looking at taking forward and and so we're not not too concerned about that side of things on fcrn. We're still working through the dosing models. We remember we have we have the second set of data that the eight-week extension that will give us a lot more information on on how about the longer-term doses lasted and so we're still working through all of that data as well as we have to have conversations with the FDA. Obviously. We'd like long dosing intervals. Uh, and we also want to make sure that we have the evidence that we can actually enhance efficacy. Uh, so we're we're looking at both boxes of that and as I think Santiago talked about in one of our prior calls, you know the ability to actually treat with with low volumes and then be able to increase doses for some patients also as a real nice thing that we can do with a rep scrape. So we haven't got it all nailed down yet. But you know, if I say long long dosing interval saf saf dosing as well as high potencies, obviously what we're looking for in our our pastry

And just a follow-up just for clarification cuz you roughly what timing at what time do you think you'll have Clarity from the FDA on Sub-Q and Clarity on the dosing schedule you think that would be by the end of the year or a little bit longer. Do you have a sense of when that would occur? Yeah. Our intent is to meet with the FDA ready to start these trials in the first quarter of next year Santiago. Do you offer a comment on any of the any of the uh timing? Yeah. We we are aiming to meet with the FDA on the forequarter of the year and and so at that time off more clarity about the tours and those intervals and if we do we have to if we do have that meeting that will mean that we can start the trial rate up in the first quarter. Yep. Yep. Yep. I'm great. Thank you guys.

Our next question comes from Stacey Q from Calendar, please go ahead with your question.

For taking my questions and congratulations on the progress. Um, so to follow up on an earlier question. So when could we expect initiation of party for m254 could occur somewhat in parallel to Parts see and then you commented on the manufacturing process. What would be the potential manufacturing or scale up getting factors for broad commercialization? I guess that's another way have we passed the gating factors that might have stalled other high-profile lady programs. Thank you. Sure. Thanks for the question. You know, the first is on Part D. I think you were asking about about our trial which is the the the multiple-dose extension. We we will make a decision on that. Once we execute the data from part c as you recall, we also are planning to start cidp trial which will be multiple housing. And so we may we may ultimately be able to use the cidp trial for the multiple doses not have to do the part the extension but we haven't made a final decision on that and it really is going to depend. I mean, we're we're obviously anxious. Yep.

Good morning. Thank you.

To see the broader numbers in the cross over data from part C just to kind of better understand this molecule and I think that will answer a lot of questions for us in terms of how we would handle part part D versus how we were thinking about what we can get out of a face off the ITP trial. The second quest part of your question was really around manufacturing and I think it's a strong. Yes that we have overcome the technical hurdles Thursday. We it's basically a uh, a scale up of volume at this point in time. We have very good control over the enzymatic reactions and we can make very reliable and reproducible products suck multiple scales. And so we feel quite confident about the technology side of this in terms of being able to maintain control over it and as we scale up and the larger larger scale we get to cost of goods gets better wages are so we we feel at least technically on the process. We are in good shape.

Thank you. Thank you.

And our next question comes from Alexander Duncan from Piper Sandler, please you know with your question.

Hey, good morning. Thanks for the question Santiago. And Tony. Could you address how you approach clinical development for the nipple m254 franchises from a scientific and clinical perspective. It appears that both agents could have advocacy potential across numerous indications. And in many cases overlapping indications due to the precedent of IVIG usage so early in your view their certain profiles for an indication that makes it a better fit for an info or 2.4 and could we see combination therapy development and select indications in the future. Thank you. I'm sure let me start and then I'll turn it over to the guys to technical guys to answer your question fully but you know, we we understand that there may be places where these drugs actually could both be effective and indication or potentially be either an indication. And so that's you know, we're acutely aware of that. We do, uh, you know, look at our initial indications 4rf CRM agent in terms of places, which we know that they are primarily antibody driven.

And and we know we're potentiating more of an anti-inflammatory activity on on hyperstimulated. So that kind of points Us in different directions, but on the longer-term development piece how I would ask maybe Tony to come into law in his thoughts and then

And then Santiago on the development strategy.

Yes, maybe just a just a begin this you know, autoimmune diseases are chronic are lifelong are generally treated like a combination of therapies that evolved over time. So I think as we've thought about how our our particular molecules could fit into the treatment algorithm. We've had a very very broad is very high on make medical need patients will need different therapies at different times. And so, you know, whereas we primarily have been sitting our molecules where we think the uh, the mechanism of of action presence of autoantibodies for instance, um is appropriate. I think we have a much broader views that says overtime one could sneeze and agents being used together in a treatment algorithm used in many many similar indications, but many many many different ones. So I think we have a very very broad view of

The potential for these two franchise agents. Um, but I think Santiago has a you know has a much more realistic view about how in the short-to-medium term we will be developing these agents and so long. I think I'll I'll pass it to him to be able to provide you a bit more color on that.

Yeah, thank you for the question. I mean m254 little differentiated assets and two by four right now the strategy that we have for the Middle East basically to be assisted Russia on a five-year in Ultra Moon disorders. And obviously the first indication will be see ADP and ITT the second indication the 2 indicators that we are walk-in with with Depot, We have any static key for the autoimmune disorders for the aluminum disorders. They are immune disorders represented the Maternal Fetal franchise. We are starting with hcfa with early-onset. We will progress to a broader population and to order immune disorder and they are many of those in the afternoon area. We are working right now in a two large franchise's the new dollar g with my starting with my dog.

And mythology starting with vulnerability in India and scrape was mentioning. We are thinking about a third indication that we would like to develop and again, you know therapy in which we could propose the use of of Nepal kalyana.

Thank you guys.

And our next question comes from Eric Joseph from JPMorgan, please go ahead with your question.

Hey guys. Thanks for taking my questions. Just a commercial a question for 250 for it seemed like the distribution of IVIG is a little more regulated than other biologics being plasma-derived and and in some regions kind of classified as blood and so to the extent that that's the case would you expect similar regulation for 204? Correct me if I'm wrong there and um and what if any manufacturing, um, uh considerations, uh are unique here if say you were to pursue globalization, um, sorry Global commercialization independently, um, are there would there be opportunities to centralize manufacture them to buy for next Thursday? Sure. Thanks. Thanks Eric. And that's a question which isn't fully answered yet. But what we have done is demonstrated that we can we can get equivalent to 5 for using different sources of IB.

So from a technical active.

City of the drug perspective. It looks pretty good. You know the the the way to think about how we look at IVIG is it's a raw material. So that's the raw material that we buy and then we usually use that in time section modify the structure of that to make m254. So as a raw material it's going to have to meet all the specs whatever we buy of IVIG and in the market for this in and so there are wage as well as Global players and i b i g and you know, the so the regulatory Pathways going to have to be defined by us because things are the first set of used as a raw material. And so that with us on The Regulators back to us, you know, depends upon exactly how we take this asset forward. If we partner this program, you know, then then we actually are working with, you know, the companies that whatever company has their their Global supply chain and taxes on our own then we would actually be working in sourcing more likely if we did that from a smaller Regional Regional or smaller Global player. And so there's a lot yet to be told in that story though the data log.

Website typically shows that we can actually make the same effect of drug from multiple sources of different ideologies. And and as a raw material, we're kind of a little bit in Uncharted Territory, but we feel pretty good about fact that we can guarantee it activity of our compound. So we'll we'll learn a lot more as we go forward.

Okay, great. Thanks to get the question. Thanks.

Our next question comes from Greg Savana van from Goldman Sachs, please go ahead with your question.

Yeah, good morning. Thanks for taking my questions congrats on the progress. Just a few if I could I just wanted to ask about the

Hello.

Once again, he would like to ask. Hello. I can't hear you.

Can anybody hear that question?

Craig fella, okay.

operator

Once again, if you would like to ask a question, please press star and then one Greg if you want to press star and one will join me back in.

Craig's line has been rejoined.

Great, how are you can continue with your question.

Hey, can you hear me now? Sorry about that. We got you on the progress and take my questions. If I could just ask about phase three and mg. I realize you don't have your dosing down just yet. But are there any details you can share in terms of you know, perhaps what your primary endpoint would be the inclusion exclusion criteria or sample size. Just want to know how similar you're planning on doing that study relative. Uh, you know, the other phase 3 trial that's out there and mg wage and then just second question has to do with next indications for McCallum. I know you talked about Dermatology potentially and off and so is this strategy to go into indications where others are already playing, um, in terms of um, you know additional indications for the other Ser ends or or wage

There would be an opportunity to think differentially, um about where to go in those other areas. Thanks.

Sure, let me let me let me you know in the first one and send it over to Santiago. We'll come back and do the second one indications. And the first thing I'll say is you can be assured that we're not going to put drug holidays into our phase three study rollback, uh for 4 mg but Santiago, do you want to comment a little bit on the design? Yeah. I mean, I think it will be premature to talk about the the time because we are working through it right now. I'm working through the results. I mean, we recently lock the the main database so it would be premature. Obviously we'll use as primary endpoint will change from Baseline ideal. There are several ways that that can be defined and I would prefer not to go over it right now because we we actually haven't make us our mind and similarly with the exclusion inclusion criteria. I seen at some point we'll be able to discuss about that right now. We are still working through it.

Yeah, thanks. And and you know, I I will just add you know that we view this as a chronic dosing and so you can expect us to use a standard repeat dosing strategy as opposed to dosage and then take patients off. That's that's kind of our view of the right way to do this and this and disease your second question was on indications. Uh, and you know, the first I I'll say is that we already are doing uh, a differentiation were first name and email were the only folks in fetal maternal at this point, but I'll let young provided just a little more color in terms of how you thinking young also is a chief business officer has the commercial aspects of the company. So I'll ask him took it a little bit in terms of how you thinking of it indications prospectively.

Sure. Hey, Greg young here, you know certainly as we think about additional indication opportunities beyond the ones that were in you know, we're looking at you know diseases that are rare which it has been the, you know, the strategy we've had all the day but also indications that maybe other specialty autoimmune disorders outside the rear space and I would say that you know by and large, you know, certainly long as are pure companies generate clinical data in indications that we're not in we think that we have a great opportunity to pursue those if we chose because we believe we have the best club in here. So certainly the D risking in those indications would be you know, something that would be in our favor. However, you know, I guess all things being equal and I think we have to this is nice either or answer because I think we can do both but I personally I think that the opportunity to kind of break ground and some of these other indications, um, you know allows us to be, you know, obviously have a personal life.

Potential and that's something which we are looking at, you know quite quite a bit as well. So I think we can do both if we only had to pick one personally. I pick the differentiated a new indication take thank you. I just have a quick follow-up on 254. I just want to go back to I think in your prepared comments Craig mentioned about you continue to see variability in the data. Can you just maybe comment a little bit more whether that's around a particular dose or or if there are multiple doses where you see the variability know it's it's across the sand and you know, it's he said that earlier too. That's when we talked about the early data. This is a highly variable disease both inter and intra patience. And so we're we're trying to see if we can turn that we're seeing Visa v i b i g but also it's evident that you know there when we start looking at the biology what we're doing we're we're dramatically enhancing at least at least one aspect of what ibid does but but higher wage

Things are going to have different effects. And so we're still trying to sort all that out. You know, the I think I commented it one of the earlier meetings that if you look at our our efficacy just you know blindly, it looks pretty good writing when you start looking at it and come back and you're saying why are some up some down and that kind of stuff and that's what I mean by variability.

So we'll we'll be able to you know, prevent paint all that picture for you when we talk to you about it. Once we get all our data analysis done. So I guess the bottom line is you could continue to be confident that we're hitting them with the trial and that the comparison diabetology is puzzling at some points. I would say so

All right. Thank you very much. Yeah. Thank you.

Our next question comes from Douglas Castle from HC Wainwright, please go ahead with your question.

Hi, good morning. Thank you very much for taking the questions either Craig. Maybe is the starting point or you know, just on the HD. I think you should have characterized as sort of like a proof-of-concept. Although I think in the past you sort of indicated you think it could be a registration all study just curious in terms of you know, the thoughts in terms of the sizing of the study of fifteen patients. I mean with consideration of sort of maybe increasing the size a little bit and sort of really putting yourself on a track to to to to file with it, especially given the fact that your typical that you know, you could result in mg. Certainly, you know would would perhaps give us all more confidence in terms of the outcome for the for the for the molecule.

Question, you know the the the challenges of where we are and feed them Eternal is that we're in a very rare. We're we're in severe early-onset. So it's a it's a small population which is a nice opportunity for us. We take a while to enroll the trial and so I I think how we think about the and how we might broaden it out would really depend on that how strong the results are, you know, the the possibility in a in a population the smallest seeing an early approval exists and uh because we see strong advocacy and we continue to hope that we will continue to see strong ethics. They would this trial and this syndication. It's something that has a lot of weight because it's it's basically life-changing and life-saving but it's fairly right. We you know, the FDA would never really do something like that and make a decision like that.

Enter most of our all of your data are in and so we're a little garden saying this is going to happen or not. It's going to be breakthrough. But but all the results so far and certainly the month the awarding of a pediatric and breakthrough from the FDA. I think gives us I mean a pediatric and and orphan for us gives us real confidence at the idea of paying good attention here, right? So with that story is going to probably told in the first half of next year once we actually get more of the patients through and really have a data set that we can talk about with the FDA.

It it helps. I know you sort of indicated the past that enrollment had been a little affected by covid-19.

instead of came

Up in terms of supply of ipig and whether you know, you're sort of strategy in obviously have multiple different sort of options as that program advances in terms of whether you partner with somebody and so forth. That's what point. Do you think you'll need to want to make a firmer commitment or sort of defined your strategy in terms of how you're going to support Supply IVIG Your Life cereal? Yeah. We're we're already working at that issue hard obviously with positive data, we've had conversations people paying attention to us. And so we'll we'll probably make that decision within the next six to twelve months, you know, which we negotiate a partnership for this. There's a whole bunch of considerations one is supply of ideology. And the other is just cost of goods, you know, if you put it into an existing IDF supply chain, it's probably a cheaper product and making it with finished IVIG. And so there's lots of from the cost of goods perspective. So there's there's lots of considerations and we're actively pursuing them and and we'll probably have that figured out in the next six to twelve months.

Okay, and is that something that would take place before you went into pivotal trials with with with the molecule? Not necessarily? Okay. So we we we haven't made a final decision that but thought it might be a good thing if we could could nail that strategy first and we have some time when with the part see ongoing. Okay, great. Thank you so much. Sure. Thanks.

And ladies and gentlemen, our final question today comes from from Wells Fargo, please go ahead with your question.

I thanks for taking my question then congratulations on the progress. So first on the M25 or study just want to get a sense of how many patients get you enrolled in part D and is the remaining item before data release really just the follow-up of like one month follow-up after IVIG treatment in perhaps the small remaining number of patients. Yeah. So thanks for the question. We didn't give you the specific numbers. We had told you were going to add add cohorts and that and look at different times different cohorts and we'll give all of that when we do the release but we did say earlier that we were wrapping up enrollment. So yeah, we are now, you know kind of getting all that last eight in from the from the 1-month follow-up and and make sure we get the analysis properly understood and validated and then we will call for everybody to understand it better.

Got it. And then on Parts have you talked about the size of the trial and also with regard to the variability on the platelet count endpoint with the size of the portion of the trial part C be sufficient to address that variability. Also probably will the trial be sorry blinded under understand the volume is different. So maybe it's difficult to provide. But it would there be blinding for the perceived sure. I'm going to I'm going to turn that over to Santiago to just talk about what you can't talk about about that parsee Santiago.

And how are you may be muted?

I'm sorry, I was muted so the party is not blinded is a randomized study which we will cross over those of 254 better soon. As one gram of IVIG. I will have to cross over cohorts with in that part of the study each cohort will have approximately fifteen patients. We are aiming to have that. Over the next several months.

I see. So if you look at the variability and do a statistical analysis with the Temptations per cohort be kind of a powered sufficiently to address variability it will it will be power sufficiently in the setting of a face to a study to understand. What would be that whatever. Yes.

Got it. Thank you. And on the hdf and sighed sorry in terms of the hdf and pediatric wage designation orphan drug designation has FDA seen clinical data from the unity study and has those any data factored into the destination. The designations sure is a rare disease, you know, we are are infrequent engagement with the FDA around this study. And so obviously they are aware of where we are as a business owner, you know, someday, I'll give you want to talk a little bit about how how you work with the FDA in these rare diseases.

We actually working very closely with the FDA. I mean, obviously this indication is high-risk indication. It's a very delicate indication in which we are working with the patients that are pregnant and and we actually have had you know several meetings and interactions with the APA along the way.

Got it, very helpful and lastly for the subcutaneous strategy for Network map is a Sub-Q injection. The only option you're considering or could there be a sub infusion version being considered? Thanks sure. And and I I would say that, you know, depending on the dose of those are feasible. Our goal is to have a simple injection maybe with an auto-injector that could they patients could continue to use at home. We may be able to you know, extend extension lines. If we even further if we use some kind of Sub-Q injection device or patch but we we are looking at all that with our with our our delivery team, but we haven't made any final decision on that yet.

Got it, very helpful. Thank you so much, sir. Thank you.

And ladies and gentlemen with that we'll end today's question-and-answer session. I'd like to turn the conference call back over to Craig wheeler for any closing remarks. Okay. Thank you guys. I know this is a tough one. We're working from our living rooms, but I do appreciate everybody. Obviously. We're working really hard to make sure we can keep everything on track in the company and I look forward to updating you in the coming quarter and at the next turn off thank you, bye-bye. Ladies and gentlemen that does conclude today's conference call. We do. Thank you for attending you may not disconnect your lines.