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Edited Transcript of MNTA earnings conference call or presentation 7-May-20 12:30pm GMT

Q1 2020 Momenta Pharmaceuticals Inc Earnings Call

CAMBRIDGE Jun 2, 2020 (Thomson StreetEvents) -- Edited Transcript of Momenta Pharmaceuticals Inc earnings conference call or presentation Thursday, May 7, 2020 at 12:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Anthony M. Manning

Momenta Pharmaceuticals, Inc. - Chief Scientific Officer

* Craig A. Wheeler

Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director

* Patricia L. Eisenhaur

Momenta Pharmaceuticals, Inc. - VP of IR & Communications

* Santiago Arroyo

Momenta Pharmaceuticals, Inc. - Chief Medical Officer

* Young T. Kwon

Momenta Pharmaceuticals, Inc. - Chief Financial & Business Officer

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Conference Call Participants

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* Anna Gennadiy Vorobyeva-Schiano

Goldman Sachs Group Inc., Research Division - Research Analyst

* Derek Christian Archila

Stifel, Nicolaus & Company, Incorporated, Research Division - Director & Senior Analyst

* Douglas Dylan Tsao

H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst

* Eric William Joseph

JP Morgan Chase & Co, Research Division - VP & Senior Analyst

* Julian Reed Harrison

BTIG, LLC, Research Division - VP & Biotechnology Analyst

* Stacy Ku

Cowen and Company, LLC, Research Division - Equity Research Associate

* Yanan Zhu

Wells Fargo Securities, LLC, Research Division - Associate Analyst

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Presentation

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Operator [1]

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Good morning. My name is Jake, and I'll be your conference operator today. At this time, I would like to welcome everyone to the Momenta Pharmaceuticals First Quarter 2020 Earnings Teleconference Call. (Operator Instructions)

At this time, I would like to turn the call over to our first host, Patty Eisenhaur. Ma'am, the floor is yours.

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Patricia L. Eisenhaur, Momenta Pharmaceuticals, Inc. - VP of IR & Communications [2]

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Thank you, Jake, and good morning, everyone, and thank you for joining us today for Momenta's conference call to discuss financial results and operational highlights for the first quarter of 2020. Today's call is being webcast and will be available for replay on the Investors section of our website at momentapharma.com.

Joining me on the call with prepared remarks are Craig Wheeler, President and Chief Executive Officer; and Young Kwon, our Chief Financial and Business Officer. Also available for the Q&A portion of the call are Santiago Arroyo, our Chief Medical Officer; and Tony Manning, our Chief Scientific Officer. Following our remarks, we will open the call to questions.

Before we begin, I'd like to mention that our call will contain forward-looking statements about our financial outlook, business plans, objectives and other future events and developments, including statements about the timing of regulatory filings, regulatory approvals and launches of our product candidates and products; the market potential and reception of our product and product candidates; potential competition and revenues for our product candidates; product development strategies, goals and timelines; development of our product candidates, including design, timing and goals of clinical trials and availability and timing and announcement of data and results; the use, efficacy, safety, potency, dosing, 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 studies; 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 the COVID-19 pandemic on the timing, enrollment or results of our clinical trials, operating expenses, business and the supply of our manufactured drug materials, 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 annual report on Form 10-K 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 statements made on today's call.

On the call, we will also discuss first quarter 2020 non-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.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [3]

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Thank you, Patty, and good morning, everyone. In these challenging times, I hope everyone is staying safe and healthy. Our thoughts go out to all who are affected by this COVID-19 pandemic. Momenta, like all companies, has had to adapt our operations to the crisis in many ways. As we announced last month, we have implemented a set of measures to adjust to the crisis with 3 primary objectives: first, to protect the health and safety of our Momenta community and of the patients and health care workers involved with our clinical studies; second, to preserve the integrity of our ongoing trials and to ensure our enrolled patients continue to have access to the investigational drugs they need; and third, to ensure the continuity of our business as we navigate uncertain times. These measures remain in effect. We also outlined last month the impacts expected for our ongoing and planned studies, which I will update you on during today's call.

Following my update, Young Kwon, our Chief Financial and Business Officer, will discuss our first quarter 2020 financial results, after which I'll close with some final comments, and then we'll open the call to your questions.

At Momenta, as most of you who are familiar with us know, we are focused on developing novel biologics to treat rare autoimmune-related diseases. Our core capabilities include a deep understanding of immune biology, coupled with our long-term expertise in antibody engineering.

We currently have 3 molecules in the clinic, each targeting different aspects of Fc biology. Based on animal and clinical data to date, we believe all these candidates possess best-in-class properties and franchise potential with applicability across a range of immune-mediated diseases.

Behind these candidates, we are researching a series of novel agents utilizing drug discovery platforms derived from our existing clinical programs. These include our Fc Multimer and sialylation technology platforms, which will continue to fuel the pipeline as our lead programs advance towards the market.

This is an exciting time for our company. Despite the challenges of COVID-19, we are pleased to reconfirm that we are on track for key catalysts coming up in the next few months, including proof-of-concept results from our 2 lead programs, both expected by the end of the third quarter. Let me give you some details on these programs.

I'll start today with nipocalimab, our FcRn candidate, which will be the first of these programs to read out. By now, I'm sure you're familiar with the rationale for targeting FcRn, so I'll skip the biology and focus on the program. FcRn is a competitive field. Ultimately, we believe success will come down to the attributes of the molecule, its efficacy, its safety and its dosing and the development strategy used, indications, presentations and populations that, combined, could create a best-in-class profile versus other competing agents. We believe nipocalimab is well positioned to capture this opportunity.

Data from existing therapies, such as IVIg and plasmapheresis, have shown that there is a correlation between the lowering of autoantibodies, or IgGs, and enhanced efficacy. We believe the same correlation will hold for FcRn therapies. In our Phase I study, nipocalimab was shown to be the most potent agent for lowering IgGs with a maximum reduction of around 85%, following a single 60-milligram per kilogram dose. To date, nipocalimab has shown a strong predictable dose response with good tolerability and the ability to achieve full receptor occupancy with a single dose. Our Phase I data also has shown that we can maintain a significant IgG reduction from baseline for over 1 month with 1 infusion.

Our multi-dose Phase I cohorts have demonstrated that we can maintain 100% receptor occupancy and maximally lowered IgGs for sustained periods. And our infusion study has shown that we can safely and tolerably administer our 30-milligram per kilogram dose to patients in as few as 7.5 minutes. Taken together, these attributes point to a potential best-in-class profile.

We are developing nipocalimab to capture multi-disease franchise opportunities in 2 areas of medicine: autoimmune disease and fetomaternal disorders. We are developing both infusion and subcu options for patients. This strategy, supported by the clinical data we have to date and that we expect to generate from our ongoing trials, will support a broad label for this program. Our ultimate goal is to become the preferred agent for physicians to use across populations and medical specialties.

In autoimmune disease, IgG has been shown to drive the pathology of many rare diseases treated by different classes of specialists: neurology, hematology, dermatology, nephrology, et cetera. Each of which treats more than 1 disease for which this drug could be effective. Currently, we are evaluating nipocalimab in 2 autoimmune indications: myasthenia gravis, a neurology disorder; and warm autoimmune hemolytic anemia, a hematological disorder. We would expect to expand to other disease and specialty areas in the future as we continue to explore opportunities for nipocalimab.

The first of our autoimmune studies to read out will be Vivacity-MG, our 60-patient, Phase II clinical study in myasthenia gravis. This is a 5-arm blinded trial, with treatment in 4 arms and a placebo arm. This trial is designed to allow us to understand the performance of the drug across the full range of possible doses and to provide a large safety database, which should allow us to run a smaller, more focused Phase III trial in MG. As you know, we expect to release data from this trial soon. So I want to take a moment today to highlight its design and discuss the information we expect we'll be able to extract from the interim analysis once we see the data.

The 16 weeks' trial trials 5 arms consist of a placebo arm, a 5 mg per kg monthly arm, a 30 mg per kg monthly arm, a 60 mg per kg every 2-week arm and a 60 mg per kg single dose. Patients are dosed for 8 weeks and then followed for an additional 8 weeks. The unique design of this trial will give us information in numerous areas. First, it will give us information on how lower IgG levels may correlate to better efficacy indicators in MG. We anticipate that some of the doses, based on our Phase I data, will drive lower IgG levels than competing agents have explored in MG. Second, it will provide an extensive data set to understand tolerability and safety of nipocalimab at various multiple dose levels. Third, it will give us information on dosing interval, possibly allowing dosing once every 4 to 8 weeks. If we can demonstrate that dosing with these intervals is efficacious, I would support nipocalimab's best-in-class dosing potential. Fourth, if IgG reduction is shown to relate to efficacy, it will help us to optimize the dose we carry forward into our Phase III program and provide the possibility to individualize treatment in patients with MG through clinically meaningful dosing information to physicians. And finally, it will give us the information we need to develop an optimally dosed subcu regimen for the product. This will allow us to bridge the subcu and IV doses, and have both formulations ready in time for our MG launch.

This Phase II trial achieved target enrollment in February 2020. And while the COVID-19 crisis has created some challenges, we remain on track to report top line data from this study. We plan to provide results of our interim analysis in the late second quarter or third quarter of this year.

As of today, all patients have completed their 8-week dosing period, and we are now in the process of data source verification and working to lock the database to allow us to conduct our interim analysis.

To be clear, this look will contain data from patients during the 8-week active dosing phase of this protocol and allow assessment of the 8-week primary efficacy end point, which is Activities of Daily Living, or ADL. So it will be a robust view of the data, which we expect to inform our Phase III planning and the subsequent regulatory interactions. The final data will be available later in the year once all patients finished their 8-week observational period, all data source verified and all final queries resolved.

As with all trials during this COVID-19 crisis, we do expect minor gaps in our data set due to the inability of patients to physically meet with their doctors. We have also moved to remote monitoring of sites when necessary and possible, which means not all customary on-site verifications will occur and the time needed to source verify the data is necessarily longer. This is why we cannot be more specific on exact timing today. Once we have the data and finalize the trial, our next task will be to get our Phase III MG trial up and running. Our team is working very hard to adapt our plans to include innovative approaches like remote physician visits, home infusions and systematic remote monitoring to determine if we can implement them in our Phase III protocol. As we are all seeing changes of this type are being supported by both EMEA (sic) [EMA] and FD (sic) [FDA] regulators, it is our hope that these changes will allow us to start Phase III MG trial in early 2021.

For the Energy study, our adaptive Phase II/III clinical study of nipocalimab in hemolytic anemia, we continue to activate clinical sites in both the United States and the European Union. As we announced in April, we have temporarily suspended patient enrollment and are actively working to amend the study protocol to facilitate remote study conduct where possible. It is our intent, if possible, to accelerate the restart of the study. As a reminder, this is a large adaptive Phase II/III study with over 100 patients. Notably, nipocalimab is the lead FcRn inhibitor targeting this indication for which we have been granted Fast Track and Orphan Drug Designations by the FDA.

Shifting from autoimmune to fetomaternal medicine, I'll now highlight progress for our Unity study. This is a global, multicenter Phase II clinical study of nipocalimab in hemolytic disease of the fetus and newborn. It is the first clinical application of FcRn inhibition in fetomaternal disorders, and our antibodies' ability to maintain full receptor occupancy should enable total blockage of pathogenic auto and alloantibodies from passing through the placenta from the mother to the fetus. We continue treating patients currently enrolled and due to the life-threatening nature of HDFN, this study continues to enroll new patients at sites where they can be safely accommodated. However, due to COVID, certain sites are reallocating resources to the pandemic and are not enrolling new patients at the present time.

We believe proof-of-concept in this indication could not only transfer -- transform the treatment landscape in HDFN, but would also validate our approach in fetomaternal disorders more broadly. Moreover, we believe our ability to block the transfer of an antibody across the placenta shouldn't change depending on the antibody. Therefore, this agent could also block mom's autoantibodies from crossing the placenta and attacking the fetus, which could possibly open therapeutic options for women of childbearing age with autoimmune disease who wish to become pregnant.

This is another pillar in our broad label strategy for nipocalimab to become the preferred agent for physicians to use across populations and medical specialties. We are hopeful we can avoid lengthy enrollment delays in this trial, so we can get this drug to the women who need it as quickly as possible. Once we better understand the COVID delays, we will update you on our expected time line to trial completion.

We are working to build a winning FcRn franchise with nipocalimab and believe there is enormous commercial opportunity here. I look forward to updating you on the results of our interim analysis from the Vivacity-MG trial in the near future.

Now I'll move on to M254, our hypersialylated IgG program. As a reminder, M254 is a novel agent manufactured from IVIg. We enzymatically modify IVIg to change the structure of the glycans on constituent IgG by extending the Fc glycans and terminating them with sialic acids. Our goal in developing M254 was to establish a high-potency agent that could reduce dosing and infusion times versus IVIg while maintaining IVIg's efficacy. IVIg is a difficult-to-use agent, requiring long, sometimes multi-day administrations of very high volumes that give most patients difficult-to-manage adverse events. Despite this, it generates approximately $6 billion a year in autoimmune sales globally. If our agent delivers on its promise, it could have significant benefits over IVIg in terms of safety and the patient convenience and require less plasma to produce, thereby expanding the market opportunity.

We are currently conducting a multi-part Phase I/II clinical trial in idiopathic thrombocytopenic purpura, or ITP. We are in Part B of this 4-part trial, which is composed of a single ascending dose cohort of ITP patients, each followed by the standard 1,000 mg per kg dose of IVIg. Early data we shared in January from Part B showed a dose response similar to IVIg's. Based on the data we shared in January, we made the decision to expand Part B to explore 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 end point for efficacy is achieving platelet levels of over 50,000 per microliter and a rise from baseline of at least 20,000 per microliter.

While enrollment has slowed due to the impact of COVID-19, we continue to recruit patients at sites that remain open. Due to the slower enrollment, we now expect to complete results from Part B sometime in the third quarter of 2020. Part C and D will be delayed as we need results from Part B, but we do expect to get Part C underway this year. We look forward to sharing complete Part B results with you and are targeting to do so in the third quarter.

Notably, these Phase II delays will also delay the start of the M254 trial in chronic inflammatory demyelinating polyneuropathy, or CIDP. Parts C and D will respectively evaluate single and multiple doses of M254 in a crossover design to establish the sensitivity to which product it's administered first and provide data on M254 in a multi-dose setting. This data will be important as we plan for CIDP, which is a chronic indication. We now expect to initiate the CIDP study in 2021.

I'll now touch on our earliest clinical program, M230. This program is partnered with CSL, which is currently running a Phase I clinical program to evaluate the safety and tolerability of M230 in healthy volunteers. Because many autoimmune conditions are chronic and require lifelong treatment, CSL is planning to introduce a subcutaneous formulation into the Phase I program in the second half of 2020, subject to any COVID-related delays. A subcutaneous formulation will offer additional convenience benefits to patients, including self and home administration.

Finally, in January, we nominated M267, a SIFbody development candidate targeting CD38, as an early development candidate. Preclinical data suggest M267 has the potential to be a best-in-class therapeutic for the management of plasmacyte-mediated diseases such as multiple myeloma, AL amyloidosis and rare autoantibody-mediated diseases. So we believe this program has significant opportunity to be another high-value program with franchise potential. We have initiated IND-enabling studies for this program, and notably, COVID has not slowed down our research team, and this program is on track for the clinic in 2021.

As I hope you can tell from this update, while we are seeing delays with COVID, our company is operating at full capacity, and we are doing everything we can to minimize timing delays for our programs.

With that, I'll now turn the call over to Young to review our first quarter 2020 financial results. Young?

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Young T. Kwon, Momenta Pharmaceuticals, Inc. - Chief Financial & Business Officer [4]

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Thanks very much, Craig, and good morning, everyone. With regard to the company's financials, I would like to start by mentioning that while we have had to adjust some of our clinical plans in 2020 due to the COVID-19 situation, we believe we have sufficient capital to fund operations through at least the third quarter of 2021.

In the first quarter of 2020, we reported a net loss of $39.6 million compared to a net loss of $44.8 million for the same quarter of last year. The decrease was primarily due to lower G&A costs offset by increased manufacturing and clinical development expenses.

Product revenue for the first quarter totaled $8.7 million compared with $2.4 million for the same period in 2019. The increase was primarily due to higher net sales of Glatopa by our partner Sandoz driven by volume increases.

R&D revenue for the first quarter totaled $0.2 million compared to $1.8 million for the same period in 2019. The decrease was primarily due to lower reimbursement revenue for Glatopa expenses and lower revenue recognized from Mylan's upfront payments associated with the biosimilar collaboration.

First quarter total GAAP operating expenses were $49.6 million compared to $52.2 million for the same period in 2019. First quarter R&D expense increased to $34.2 million compared to $28 million in the same period in 2019. This was primarily due to an increase in manufacturing and clinical trial costs for nipocalimab and M254 and increased spending on M710. First quarter G&A expense decreased to $14.6 million compared to $24.2 million in the same quarter period in 2019. This was primarily due to lower depreciation and rent costs associated with the modification of the Bent Street lease and lower legal costs.

For the first quarter of 2020, our non-GAAP operating expense was $44.7 million. As a reminder, our non-GAAP operating expense is defined as total operating expenses less stock-based compensation, less restructuring costs and less collaborative reimbursement revenues. Finally, we ended the first quarter with $487.9 million in cash, cash equivalents and marketable securities compared to $545.1 million at the start of the year.

Turning now to our guidance for 2020. I due to lower clinical trial enrollment trends as a result of the COVID-19 pandemic, we expect our full year non-GAAP operating expenses will be lower than $220 million to $240 million as previously guided for 2020. We anticipate providing an update on expected non-GAAP operating expense for 2020 with our second quarter 2020 financial results.

With that, I'll turn the call back over to Craig for closing remarks.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [5]

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Thanks, Young. As you've heard, we remain in a strong corporate position with cash to fund operations through the third quarter of 2021 and 2 near-term proof-of-concept readouts in the coming months.

Overall, I'm very pleased with the progress our team is making during this difficult period. They have put patients first and are showing their mettle as they work to adjust to very difficult working conditions to keep our programs moving. It is my hope, due to the Momenta team's determination, we will emerge from COVID crisis with an intact and advanced portfolio in an even stronger organization. I look forward to keeping you all updated on our progress, stay safe and help where you can as we work to end the COVID crisis and deal with its aftermath.

Thank you again for joining us. And I'll turn the call back over to Patty to get the Q&A underway.

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Patricia L. Eisenhaur, Momenta Pharmaceuticals, Inc. - VP of IR & Communications [6]

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Thank you. And Jake, you can open up the Q&A.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Derek Archila with Stifel.

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Derek Christian Archila, Stifel, Nicolaus & Company, Incorporated, Research Division - Director & Senior Analyst [2]

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Great. Congrats on all the progress. So just a couple from us. Maybe first, Craig, you had a competitor yesterday talk about transitioning their program from IV to subcu, so I just wanted to kind of get a sense of how you guys are thinking about IV and the subcu presentation and maybe some of the advantages of having both.

And then maybe a question for Tony in terms of like what -- how are you thinking about the depth of response that you could see with nipocalimab in MG and how that potentially could translate to durability? And should we be thinking about the differences between a competitive binder like efgartigimod versus a full blocking monoclonal like nipocalimab or some of the other fcRns out there?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [3]

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Derek, Thanks. I'll take the first one as you suggested, then I'll let Tony address the second one, and Santiago can feel free to add there if he likes.

On the competition, yes, we do see people looking at subcu and IV. I think our view has not changed from -- into the start of our program. We believe that both presentations are going to be important here. Subcu, because of the wide range of diseases and patient preferences, is going to be preferred by some patients. And we, therefore, have been for quite some time actually working on a very -- what we think is going to be a very good, strong, potentially an industry-leading subcu presentation. However, we made the determination that until we understood the best way to dose, we didn't want to actually introduce subcu because we felt that using our infusion was a better way to understand the drug and then bridging to the right subcu doses. And so we'll have it ready for launch, and we think it's an important component of the offering for these products because of the nature of the diseases.

That being said, we think IV will be, and will be continued to be, used in a large portion of the population as well. These patients are seen quite frequently and, depending on the disease, some more than others in their physician offices. And what we've seen so far is that with our agent at least, we can have very rapid and safe infusions. So if you think about the ability to do an infusion, once per month or maybe once for every 2 months, if we're fortunate with the data from our Phase II trial versus what are going to be painful and relatively high-volume self-injections, I think a fair number of patients will continue to want the infusions. And so we actually think both presentations are going to be a part of the playbook for the leading agents in the indications.

So with that, I'll turn it over to Tony, and he can talk a little bit about the comparative mechanisms here.

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Anthony M. Manning, Momenta Pharmaceuticals, Inc. - Chief Scientific Officer [4]

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Yes, Derek, thanks for the question. The durability of the clinical response will, we believe, be related to the kinetics of the IgG reduction. And clearly, nipocalimab has some great advantages in being able to drive down and maintain the low IgG levels. But actually, I think for the sort of clinical relevance of all of that, I think I'd probably pass this over to Santiago to describe a little more his thinking about how the kinetics of IgG reduction relate to the durability of the clinical response. Santiago, do you want to take it from here?

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [5]

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Sure. There has been suggestions that the IgG response is -- can actually drive the efficacy. There is suggestions in the literature from the plasmapheresis and IVIg literature, and there is suggestion also from some of the competitors' data. So we are truly very interested in understanding that relationship between IgG and efficacy, and that's how we power our study. If that were to be true, hopefully, we'll be able to understand much better how to individualize the dose for the patient.

I wanted also to add on Craig's comments on subcutaneous dosing that it's very easy to get early on pin down on a subcutaneous dose without full understanding of dosing and dosing interval, and we've been trying to avoid that as much as we have done. And you will see that that's why we have developed our subcutaneous formulation, but we haven't pushed it forward into the clinic until we have full understanding of dosing. I think that there is a lot of possibilities with the subcutaneous dosing. And again, the critical piece will be how low could be the dose that we could inject on a weekly or biweekly basis or even longer. So this study results would probably allow us to understand that piece and then we'll push forward the subcutaneous formulation along with the IV.

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Operator [6]

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Our next question comes from Eric Joseph.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - VP & Senior Analyst [7]

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I guess just a couple on 254 for me. Looking to the data in ITP that you plan to present in the third quarter, just hoping to level set expectations in terms of patient numbers and the total number of cohorts that we should expect. Should we expect, I guess, data from the lower dose cohort that you guys have initiated? Would you expect to have visibility on a dose response or a minimally effective dose when Part B reads out?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [8]

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Sure. Thanks for the question. I'll start and then if Santiago wants to add anything to it, I welcome that.

So the decision that we made to add lower dose cohorts was because we actually saw efficacy down to our lowest dose, and the decision to expand the number of patients in a cohort was because we saw wide variability in how patients respond. And therefore, we were unsure with a low number of patients in each cohort, if we could actually effectively see a dose response. And so we needed to see more patients in a cohort. And to support that, we actually shared with you in January the variability that IVIg has always seen in this population. And so what you will see is more patients per cohort and then lower cohorts.

We haven't specifically talked about numbers because we have given the clinical team a fair amount of flexibility to add doses to be able to understand that dose response as well as to add patients to understand what's necessary for being able to evaluate a dose response. So we haven't really given any specific guidance, but you can expect that there will be significantly more patients that we will bring forward. And we're in the middle of that now.

Santiago, do you want to add anything to that?

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [9]

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No, nothing else. That's clear. Thank you.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [10]

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Okay. Thank you.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - VP & Senior Analyst [11]

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Okay. That's helpful. Maybe just one follow-up. I know that one of the motivations to be able to explore lower doses is to allow for subcu formulation potential with 254. Do you have a sense at this point sort of what quantities of drug would be amenable to a subcu formulation? Are you potentially in that range already at 60 milligrams? Or do you guys necessarily need to see efficacy on lower doses to enable that subcu optionality?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [12]

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Yes. Thanks. Well, the answer to that question is a little tricky because as you look at the IVIg class, they have dramatically expanded what we think about as subcu. So they're giving massive dose of subcu and some of the IVIg presentations now. Our view is we would like to make a patient-friendly subcu, if we go to subcu. And so that's why we're thinking about the lower doses that we can go to. But also, there's a lot of other benefits besides subcu and going lower in terms of amount of plasma needed, cost of goods, all of those types of things. So lower is better in every dimension if we can get there.

But in terms of what's effective for a subcu, even at 60 milligram, if you compare it to the high volumes that are being given today with IVIg subcu, it would be much improved. But we'd love to get into the kind of range like we're thinking about with the -- for example, in the FcRn agents, which would make a very convenient subcu. But it's too early to tell if we can get there yet. But subcu in IVIg is a really interesting -- it's different than every other space because of the way some of the IVIg suppliers have introduced subcu products.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - VP & Senior Analyst [13]

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Got it. And maybe just one last question on nipocalimab. I guess when it comes to Phase III planning in MG, is the idea to advance a single-dose regimen here? Or is it possible you might move kind of multiple regimens depending on how the Vivacity data looks?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [14]

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Yes. Santiago, why don't you take that one?

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [15]

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Yes. We haven't really started to design the Phase III program because in part, it truly rests on the results of the Phase II. So when we have these results, which will be very soon, we'll be able to start to put together a Phase III program. And as Craig was saying, we are aiming to start that Phase III program very soon, hopefully, at the end of the year or beginning of next year.

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Operator [16]

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Next question comes from Graig with Goldman Sachs.

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Anna Gennadiy Vorobyeva-Schiano, Goldman Sachs Group Inc., Research Division - Research Analyst [17]

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This is Anna on for Graig Suvannavejh. Just a few questions on M254. Can you comment on the general variability observed in patient responses to classic IVIg? And what's the read-through to M254? Is there anything we can look for that's unique to specific autoimmune indications that could mediate the patient response variability?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [18]

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Sure. Maybe, Tony, you can talk a little bit about some of the data we've seen in IVIg. And then Santiago, you can talk about -- a little bit about how we're thinking about the dosing there?

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Anthony M. Manning, Momenta Pharmaceuticals, Inc. - Chief Scientific Officer [19]

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Yes. Well, so Anna, we have looked at all of the available Phase III results for 3 IVIg approvals, and it's -- and we've shared that with you guys at JPMorgan. And no, there is a lot of variation by individual in the response to IVIg administration. I think there's really not a strong understanding as to what drives those -- the variable response. But it's clear that IVIg is very effective at boosting patient platelet numbers above some of those critical levels that you would need to maintain their health status. And so that's why we've really looked at the clinical response and the rates of clinical response. And I think there, what we see is quite a consistent story that M254 can boost platelet levels, achieve those clinical responses pretty consistently as we've shared with you. So I think there's not a good understanding of the variability of response to IVIg. And so I think that's one of the reasons why we've chosen to expand the patient numbers in our Part B and get additional information on the clinical response.

I should hand it over to Santiago, really, to talk more about the impact of that variation on the clinical responses.

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [20]

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Yes. I mean from the beginning because we analyzed previous data, data from other companies, it was clear that the variability was going to be an issue to deal with in the trial. That's why we designed the trial in 2 parts. I mean in part -- several parts, but the critical parts are Part B and Part C. Part B is a dose-escalating part in which we try to get an understanding of what are the doses that may work. And Part C is an expansion where we'll focus in one or 2 doses in which we'd really want to include many more patients to be able to understand fully and with precision that, that dose is the right dose to carry over to Phase III.

So we are now in Part B, and what we've done is beef up a little bit the number of patients. We have a lot of flexibility in our study design to do that. And we are exploring different doses, from very low doses to moderate doses, to try to understand that. What is clear is that our results are showing that the concept has proven, basically, low doses of M254 leads to a responder rate that is similar to the responder rate that has been seen with IVIg. So that's where we are, and that's a good place to be. And now again, with more patients in Part B and then with the expansion of Part C, it would be clarified which dose or doses should we take to Phase III.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [21]

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Yes. And guys, I just going to add one -- I'm going to add -- sorry, I'm going to add one quick point on this is that one of the things we talked about is -- in prior conversations is the implications of this variability and how we think about the product. And we are increasingly thinking that rather than kind of chasing a highly variable population that we may want to think about our product more as a typical new drug going after this, and when you look at like all the other agents that are going after ITP, they're looking at over 50,000, 20,000, which is the basic end points, and then that's it, right? So -- and part of the challenge is to how -- why do we have to expand to really understand the relative performance versus IVIg. And so we're thinking about all of those things as we go forward, and it may be better to kind of just go straight at the different indications rather than to try to do comparisons. And the reason is IVIg is very poorly studied. And so it's almost like we're studying IVIg at the same time as we're studying our program. So...

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Operator [22]

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Our next question comes from Stacy Ku, Cowen.

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Stacy Ku, Cowen and Company, LLC, Research Division - Equity Research Associate [23]

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I have a few. First, on 254, I'd just like to clarify the commentary on the lower dose cohort. What are the lower doses? And I guess asked another way, are you decreasing the doses until you no longer see the same efficacy as IVIg? And I have a few follow-ups.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [24]

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Sure. I'll answer first is that we haven't disclosed the cohorts, but Santiago, why don't you talk a little bit about the goal of looking at the lower cohorts?

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [25]

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Yes, the goal is to determine what is the minimal efficacious dose. So we'll go as low as is needed to understand that piece.

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Stacy Ku, Cowen and Company, LLC, Research Division - Equity Research Associate [26]

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Okay. That's helpful. And then as we wait for those Phase II studies in ITP, as you look at these 3 IVIg approvals, what are the different options for the potential Phase III design? Will the approach be more for acute or chronic dosing? Or could you get a faster acute approval and then pursue the chronic indication? So any details on what you guys are thinking would be appreciated.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [27]

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Sure. Young, do you want to talk a little bit about how we're thinking about things commercially there?

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Young T. Kwon, Momenta Pharmaceuticals, Inc. - Chief Financial & Business Officer [28]

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Certainly. I mean, Stacy, for ITP, the market for the IVIg is fairly small. IVIg is used in a portion of patients primarily as a rescue therapy, and then patients often go on to oral agents or other agents for the diseases that don't necessarily have the same kind of infusion time that IVIg has. And so we do believe that there is an opportunity in the -- that initial segment for M254. Ultimately, the size of that segment will depend on whether we would be able to expand the market from how it's used today as a rescue therapy to something more chronic. As a chronic therapy, M254 would be going up against the other agents such as TPO agonists, and we'd have to evaluate the commercial opportunity and potential in that regard. And that's still, I would say, an open question regarding the -- both the, I'd say, clinical strategy as well as the commercial opportunity there.

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Stacy Ku, Cowen and Company, LLC, Research Division - Equity Research Associate [29]

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Okay. That's really helpful. And I guess last question about 281, to the 281. Could you provide more details about the extension study? On ClinicalTrials.gov, it looks like the trial has been temporarily suspended. So just wondering are those MG patients that have already rolled over allowed to continue? And how are they being monitored?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [30]

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Sure. Santiago, I will turn that to you.

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [31]

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Yes. It's really a sad story here with COVID. We felt that we were losing sight of many patients in the open-label study because they couldn't be visited. So we decided to pause that study. The patients have continued to be monitored -- with their physicians and remote monitoring most of it. And it depends on the country, right? We have many patients in Spain, for example, which was a difficult situation. Our plan, however, is to be able to offer an open-label extension study to all of those patients once we resume the program probably early next year, so that all of the patients that have contributed to the Phase II proof-of-concept can benefit from that long open-label extension study. So that's the current situation. And fortunately, it hasn't been terrible in all of the places just because this COVID has affected very -- in a very different way, different countries and even different regions in the countries.

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Operator [32]

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We have a question from Douglas Tsao with H.C. Wainwright.

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Douglas Dylan Tsao, H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst [33]

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Hello, can you hear me?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [34]

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Yes, we can.

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Douglas Dylan Tsao, H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst [35]

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Just given the progress you've made with nipocalimab as well as 254 as well as sort of the evolution and the sort of -- or developments in the autoimmune space, just curious just your thinking around M230, and I understand that that's a partner program, but sort of -- perhaps sort of your interest in sort of maintaining at the 50% sort of profit share level has changed just given the fact that, that program is sort of moving ahead, a little slower than maybe we originally hoped.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [36]

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Sure. Thanks, Doug. I think right now, we kind of look at it as an option, right? The -- once we see the data, when we finally see it, then we'll have a good sense of the performance of that program and then we can make an informed decision. So it's a pretty low expense for us right now to keep that option open. And then when we see a data set, which we would see from CSL before the dramatic expansion of costs as they go into multiple indications, we can make that call. So it's kind of an option play for us, I guess, is the best way I would say we're thinking about it right now.

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Douglas Dylan Tsao, H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst [37]

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Okay. And then as a follow-up on the last question. For 254, how do you think sort of the 2 sort of approaches of sort of a chronic versus sort of a more acute application of 254? And how does that affect your sort of value proposition and what you necessarily need to do in your clinical studies?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [38]

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Well, I think, Doug, obviously, the big opportunities for this program, just like they are for IVIg, are in the chronic dosing. That's why you look at CIDP, it's such a very large indication for IVIg, and we think for this agent as well. So that's kind of the holy grail of where we ultimately want to get to. And as Young said, we're thinking a little bit about is there faster paths that we can get to with an acute approval and those types of things. But even with the IVIgs, acute approvals do not give them broad use in the chronic side. So we're thinking about this more as a novel agent that we actually generate the multi-dose data in Parts C and D and then go into a CIDP trial in its own right, a Phase II/III kind of CIDP program, so we'd really understand and can characterize this drug as a best-in-class and try to develop in a broader market opportunity.

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Operator [39]

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We have a question from Yanan Zhu, Wells Fargo.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [40]

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So first off on nipocalimab, is there a possibility to have a data readout in late second quarter? I know it's a highly fluid situation and not to say there is any certainty, but I just want to understand the earliest possible data readout timing.

And then a follow-up on the nipocalimab side is based on the 4 doses that you are evaluating, could you model out the best dose even if that is not one that's tested, such as 30 mg per kg every 2 weeks or 60 mg every month? So if -- do you have that ability to have the robust prediction at additional doses? And then I have one in the pipeline.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [41]

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Sure. Yes. Okay. Understood. So I'll just give you an answer on the first one, and I'm going to turn it over to Santiago to talk about the design of the trial and the dosing model because you're on to exactly the point of why we designed the trial this way. But first, as I said in my prepared remarks, we may be able to release this in late second quarter or early third quarter. And it really is kind of uncertain because of the difficulties of contacting sites and making sure we do all of the stuff necessary to make sure we have a robust data set. So it's our hope we can. But the #1 priority we have is that for that 8-week dosing period, we want to make sure we have robust understanding of the data set before we actually come on and talk about it. So there's some flexibility in the team. Santiago and his team are working very hard on this, and we'll have it out as soon as possible.

Santiago, why don't you talk a little bit about our ability to do interpolate doses here?

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [42]

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Sure. So that's precisely the whole idea of our Phase II study with the complexity of the 4-dose arms and the placebo arm was precisely to be able to do that: to model the data and, at the end of the day, understand what is the best dose, what is the best dose interval and also to provide data for the subcutaneous dosing. So that's what we are doing. Actually, the study is powered on that. It's powered on the relationship between IgG reduction and ADL and QMG as efficacy measures.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [43]

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Got it. That's very helpful. On M254, so I think in the past, you've mentioned streamlining the trial after Part B. I'm wondering if Part B is positive, what are the potential ways to accelerate the program? And also maybe adding on that, for Part -- for the Part C design, I'm wondering what additional information can be gained in Part C versus the already now expanded part B.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [44]

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Sure. Sure. Thanks. So we are still making a determination in terms of exactly that we -- what we'll get out of Part B. But you can be assured that if we go into the Part C piece, as designed, it actually has more patients than we would get out of just the expansions here. So what we're just trying to do in Part B is to get the best understanding we can to pick the 2 doses to put into Part C, which will be broader. And then the other piece is that when we look at -- what I was just talking about, when we look at the use in CIDP, we have no multi-dose information yet in patients on this and so getting that multi-dose and those things would be really helpful to understand how this drug works when you dose it over multiple doses, so that we can actually best predict how to dose it properly in a chronic indication. So that's really what Part C and D are really designed to do for us.

Santiago, anything you want to add there?

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Santiago Arroyo, Momenta Pharmaceuticals, Inc. - Chief Medical Officer [45]

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No, no. Nothing else. I mean we are -- again, we are expanding Part B to get more data. We believe that probably Part C will be important to kind of get more precision on the dose, but that's a matter of looking at all the data and make a call. There is variability in the data, and thus having a larger number of patients' dataset is always important.

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Operator [46]

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Our final question comes from Julian Harrison.

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Julian Reed Harrison, BTIG, LLC, Research Division - VP & Biotechnology Analyst [47]

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On your CD8 (sic) [CD38] SIFbody, just curious if you have a sense for whether or not you'll need to show activity in daratumumab refractory population? And can that possibly be the ultimate goal for positioning? And can you remind us what the dosing will likely look like? Is subcu a possibility here?

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [48]

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Sure. Young, do you want to take that?

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Young T. Kwon, Momenta Pharmaceuticals, Inc. - Chief Financial & Business Officer [49]

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Sure. I mean, certainly, the idea behind the program is that we have engineered a molecule that has an ability, potentially has significant effector function associated with enhanced cell killing. And that's certainly what we've seen in preclinical models. The idea behind the program commercially is that we may have the potential to have a more robust CD38 agents that I think from a commercial perspective, conceptually targeting that kind of refractory population would make sense. However, we do believe that if it's successful as a more potent CD38, then we don't see a reason why we wouldn't be able to be more aggressive in treating patients who have yet been exposed to daratumumab.

So yes, at this stage, it's a bit early to tell, but we also have some initial data that we have put out in one of our presentations around the ability to actually kill cells in a patient that has a refractory -- that's refractory to daratumumab. So we think there's an opportunity there. It's still early days given the nature of the program. And we'll see how it progresses with the kind of product profiling as well as the clinical data when we get there. As it relates to the formulation, certainly, we're thinking very carefully about the potential for subcu as part of the development strategy. And certainly, we'll unfold that as the program advances.

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Operator [50]

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There are no more questions in the queue.

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Craig A. Wheeler, Momenta Pharmaceuticals, Inc. - President, CEO & Executive Director [51]

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Okay. Thank you. And let me just thank everybody again for taking the time to join us on our call here today. And everybody, stay safe, and we'll talk to you very soon with hopefully some positive trial results. Thank you very much, and have a great day.

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Operator [52]

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That now concludes the call, everyone. You may now disconnect.