Q2 2021 Mirum Pharmaceuticals Inc Earnings Call
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800 clients and over 370 employees across the business here.
And with both of these numbers of increasing weekly and we start leading the growth.
So as the color to the size of our opportunity in 2020, 1 state and local government and the U S is expected to spend just under 120 billion and IP.
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Thank you all for your patience, the mayor and Pharmaceuticals, Inc. Second quarter, 2021 and business update will begin shortly.
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Hello, everyone and welcome to the Mayor and Pharmaceuticals, Inc. Second quarter 2021 business update my name is <unk> and I will be your coordinator today.
You will have the opportunity to ask a question. After the presentation. If you would like to ask the question you might say by pressing star followed by 1 on your telephone keypad I will now hand, you over to you of hoist and Kennedy Chief Financial Officer again, and please go ahead.
Sure.
Good afternoon, everyone I'd like to welcome you to mirror and Pharmaceuticals second quarter 2021 conference call.
And I'm joined today by our President and CEO, Chris <unk> and our chief.
Chief operating officer, Peter average.
And with this afternoon mirrored issued a news release announcing the company's results for the quarter ended June 32021.
Copies of the news release, and our SEC filings can be found and the investor sections of our website.
Before we begin I'd like to remind you that during the course of this conference call, we will be making certain forward looking statements of Batman and that program and based on management's current expectations.
Including statements regarding the business development programs and strategies prospects commercialization and market opportunities.
The forecast and guidance and <unk>.
And the FDA interactions for announcements on sales.
And of the business methods.
These statements are subject to numerous risks and uncertainties and actual results could differ materially from the results anticipated by the statements.
Investors should read the risk factors set forth in men in the 10-Q for the quarter ended June 32021, and any subsequent reports filed with the SEC.
All statements are made as of today oversupply of 2021 and are based on information currently available to us and they are.
And does not undertake any obligation to update publicly any forward looking statements whether as a result of the new information future events or otherwise except as required by law.
Without guidance I'd like to critical of the crispy Chris.
Hey, good morning, and good afternoon.
Thanks, everyone. Thanks for bearing with the cyclical lift from what we can.
Kicked off.
Without the new per data September 29th from neuro looks about our focus is squarely on the realization of the upcoming potential wash.
Our Johnson, the lead and genetic cause of the pediatric liver transplant.
Any of the is driven by the severe pruritus and simple math.
Burden of disease.
The 8 indications of our waiting for new treatment options.
We estimate the al Jolson, and alone, maybe and more than $500 million opportunity.
And we are launch revenue now have of several of the chain and financial resources for successful commercialization, all while expanding our pipeline of potentially Registrational studies and other rare liver diseases.
<unk> is focused on advancing life changing medicines.
Today, we will recap and provide context around some of our most exciting recent steps as we continue to realize our vision of being the leading rare liver disease company.
Okay.
I will cover of regulatory and pipeline updates as well as the additional value and continue to build within the clinical programs.
Peter will share updates on commercialization and Dan will summarize our financial highlights.
With our fifth year to date from Merrill Lynch of that and call static pruritus associated with LNG options on the next month.
Our team is ready to launch upon the potential approval.
Our regulatory review remains on track and we've been able to accommodate all inspections to date.
<unk> has continued to indicate no plans for an advisory committee.
And if approved and the US narrow list of that would serve as the first treatment approved for patients with al Jolson growth.
And the iconic study, which served as a pivotal study.
The significant improvements of <unk> were observed with more than 80% of participants achieving a 1 point reduction and it's true.
We are 6 years of follow up data and see the durable responses and trial participants across the extensions of all 3 randomized studies of Merrill Lynch and eligible syndrome.
And of approval and subsequent commercial launch with marks an important milestone for us at <unk> and <unk>.
More critically and important milestone for patients living with this terrible disease.
We remain committed to further building on the body of evidence supporting nervous about the potential and eligible syndrome.
And here, we presented integrated the safety analysis of Merrell looks about and patients with eligible households on them at the equal Congress.
These data and resulted from 5 years of narrow looks about treatment across 86 patients and continues to reinforce our understanding of the drug safety and tolerability profile in patients with <unk> syndrome.
Merrill Lynch of that was well tolerated and these patients from more than 5 years with the most common treatment emergent adverse events reported the diarrhea and abdominal pain.
And iconic dire.
Diarrhea incidents was at similar rates between the Merrill Lynch and placebo during the placebo controlled portion of the study.
Also and again presented Merrill Lynch about transplant free survival data for progressive familial intrahepatic cholestasis by 2 of the piece of true at the World Congress of Paediatric, Gastroenterology, Hepatology and nutrition annual meeting.
As detailed on our presentation Merrell looks about as good of patients who achieved 75% serum bile acid reductions.
Were shown to have 100% 5 year native liver survival.
The <unk> data are under review with the European Medicines Agency.
Building on that our phase III March Chief ex study continues to advance well with greater than 65 patients randomized the pubic subtypes 123, and 4 across 2 cohorts.
As a reminder March piece of evaluate a higher dose of Merrill Lynch and has been shown to drive greater buy off the curve.
We plan to extend enrollment and.
Top line data and the second quarter of 2022.
Further brother and broadening our efforts and pediatric liver disease, our phase 2 of the embark study.
And the merits of that and biliary atresia is progressing nicely as well and we and types of anticipate topline data in 2023.
And adult liver disease.
We are excited about advancing our clinical programs per <unk>, which we believe is another potentially transformative treatment for several of the cole of <unk> liver diseases.
Earlier this year, we launched our face to the this dose study for adults with primary sclerosing cholangitis and the face of the Johan the study that will evaluate the bullets about and intrahepatic cholestasis of pregnancy.
The phase III study in primary biliary cholangitis is targeted for later this year.
Yes.
And we've incorporated regulatory feedback and should the design of the sports about studies all of which are potentially registrational.
We expect interim analyses for the Vista and the hottest studies to occur next year.
And before heading on to Peter to discuss launch readiness I want to touch on <unk> commitments to ensuring broad global access to Merrill Lynch of about <unk> syndrome patients.
To facilitate the international reach of Merrill Lynch about we recently entered into and exclusive licensing agreement with <unk> pharma to develop and commercialize <unk> and South Korea.
This builds on our partnership with Cambridge, Pharmaceuticals, and greater China, which we announced on April.
Both of <unk> pharma, and Cambridge are leaders and rare disease and share our passion to advance life changing medicines.
And we will pursue expedited approval pathways from <unk> for hours Youll syndrome based on our U S filing package.
We see the high interest level of our partners as Testament to the potential of Merrill Lynch of about 4 pediatric cholestasis patients.
On that note I will turn it over to Peter who will provide an overview of the launch readiness activities Peter.
Thanks, Chris.
With our Paducah date of September 29th just over a month away. Our team is ready to deliver of Merrill Lynch about to the allergy of simple community should it receive FDA approval.
And the last quarter, we've made significant progress and our pre commercialization efforts. Our strategy has centered on 3 areas the use of awareness and patient finding patients.
Patient services and market access.
Starting with disease awareness and patient finding our field sales team has hired fully trained and has been and the field for more than 2 months.
Connected with all of our key 125 accounts, including and depth personalized discussions with the majority the.
The team is providing disease awareness education on ours, you'll syndrome, and including recognizing will impact and severity of pruritus.
We are gaining valuable account profiling and price from the discussions.
<unk> and working with health care professionals to identify patients with ALS Youll syndrome.
Additionally, as part of our patient identification efforts mirror and participate in and industry collaboration genetic testing program, which provides free genetic testing and identifies mutations and drag 1 and notch true which caused the LNG of central.
Together with what we've learned and our field discussions as well as the genetic testing program, our clinical research and expanded access programs also support our estimates, but there are 2000 to 2500 children with the allergy of syndrome and the United States.
Continue to work with healthcare professionals to identify patients as we approach the potential launch of Merrill expense.
Turning to patient services, Miriam access plus or map, which is <unk> program to provide end to end support for allergy of syndrome patients and caregivers and initiating merrell looks about upon potential approval is ready to go from.
The map team has been staffed and trained full operational readiness will occur in the coming days in order to start preparing EAP patients to transition to commercial product upon the potential FDA approval.
Finally regarding market access the payer team has been very busy we've conducted 53 engagements within our top accounts and the team has been educating Medicaid and commercial payers on the burden of biology of syndrome and in order to prepare them to conduct the timely and evidenced based reviews and ultimately.
Access decisions upon potential approval.
We've invested early on those fronts. The burden of this disease is hard to overstate and the broader community is learning about this dynamic the cost out of pruritus is a leading indication for transplant and surgery and our jewel simple overall.
Overall pay of have recognized the devastating impact of ours, you'll syndrome, and the high clinical impact of Merrell looks at that.
We remain passionate about and complement and nervous about and the profound the impact we expect at the half on the hours Europe's into the community.
And I am thrilled about our progress to date and look forward the sharing more about our ongoing efforts with our with the look and update after our produce the date.
Mark.
I will now turn the call over to Ian to share of financials and.
Thanks Peter.
And we look at our course and financials is available in the press release distributed earlier this afternoon and in the form 10-Q.
You can find both on the Investor section of our corporate website at near and pharma Dot Com.
And I'll provide an overview of what we believe free of the highlights from the second quarter of 2021, all of which position us well to support the launch of <unk> and 2 geographies and advance our development pipeline for both <unk> and the looks about in their respective indications.
<unk> remains well funded and of the close of the second quarter ended June 32021, we had cash cash equivalents and investments of $238.8 million.
And also as a reminder, we have access of up to a further $85 million from Oberland capital. This figure comprises $35 million upon approval of our NDA of <unk> about the pruritus and <unk> syndrome.
And a further $50 million for mutually agreed upon and licensing for acquisition opportunities.
Additionally, we anticipate receiving a priority review voucher or PIV and connection with our expected FDA approval of <unk> of that later this year. The may provide a fair the source of funds.
With respect to operating expenses for the quarter ended June 32021, G&A expenses were $13.4 million.
The amount reflects increased activity around launch readiness, including market research disease state awareness activities and <unk>.
Personnel costs to support our planned launches.
Second the R&D expenses were $35 million compared to $18.6 million for the second quarter of 2020.
The increase share was primarily driven by an upfront payment and funding associated with the the vet gene therapy programs and with the costs associated with the initiation of <unk> clinical studies.
As Chris alluded to part of our strategy is to accelerate commercial launch and patient access globally through partnerships outside of our core U S and the EU territories.
On April the 2020 to 'twenty, 1 we announced the licensee agreement with Cambridge Pharmaceuticals.
Under the terms of disagreements and exchange for and $11 million upfront payment R&D funding and up to a further $109 million with future regulatory and commercial milestones and Cambridge has obtained the exclusive rights to develop and commercialize <unk> of that within the greater China regions for allergy of syndrome perfect.
And Barry Atresia.
We can expect double digit tiered royalties based on future product net sales.
Additionally, subsequent in the quarter and we entered into an exclusive licensing agreement with <unk> pharma.
And the terms of disagreement GC, Panama has obtained the exclusive right to develop and commercialize <unk> of that within the South Korea, the allergy of syndrome PK comparability of accretion.
We received of $5 million upfront payment and are entitled to further research and development of funding and up to $23 million to the achievement of future regulatory and commercial now looks about milestones with double digit tiered royalties based on product net sales.
So as a result of the strategic investments and experienced team and focused rare disease business model. We continue to have a robust balance sheet.
Access to approximately $400 million and.
Capital comprised of our cash position the potential <unk> monetization and the strategic partnerships, we expect to be able to sustain our operations for the next 3 years.
With that I'll turn the corner of its Chris the final comments Chris.
Thanks, Dan and thanks, again, everyone and joined the call today.
To close from continued to pave the way and the treatment of rare liver diseases.
And the potential FDA approval for the first ever medicine, KOL static pruritus and household syndrome, and our other clinical programs continuing to progress across a wide range of indications.
We are well on our way to realizing the company's vision set forth 2.5 years ago.
The briefly recap of our upcoming milestones are produced the date of September 29, primarily for that for the treatment of close out of credits and patients with <unk> syndrome.
Upon potential approval U S commercial launch will follow.
And we estimate this to be of greater than $500 million opportunity.
We have upcoming key data updates on the pediatric program with long term of liver related outcomes analysis of the Merrill Lynch of that treated patients with al Jolson and targeting upcoming medical Congresses the presentation.
And the topline data of the March <unk> Phase III study of Maryland, <unk> and Q2 next year.
Potential international regulatory events ahead include the <unk> MAA under review with the Eni subs.
Subsequent filings per hours Youll syndrome, and Europe.
And potential for partner market approvals.
Finally, our Registrational program for the likes of that and adult Cholestasis has taken shape with interim analysis of next year of the Opana and Vista studies, and intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis.
Lastly, initiation of the phase <unk> study in primary biliary cholangitis still to come this year.
We take pride and the meaningful scientific advances, we have achieved so far and our ongoing engagements with the rare liver disease patient community as we continue to execute and maintain a strong cash position.
We are confident and our ability to achieve the milestones we have set premiere and look forward to the potentially transformative impact Merrill Lynch of the anvil looks about could have on the lives of patients and their families.
Thank you again for joining us.
Operator.
Please now open the lines of questions.
Thank you Chris if you would like to ask a question. Please press star followed by 1 on your telephone keypad now.
And your mind. Please press star followed by 2 to come from your quest when comparing to ask your questions. Please ensure that youll find it on and you should likely.
Our first question is from money from the heart of.
And of SDB Leerink.
And that's access and you know the ability to participate in Medicaid program to open up the early next year and and I think the the other point is on the commercial side. There's a there's a lot of different plans out there kind of of heterogeneity of of of practices are on coverage decisions and how those are made and windows and made including some of the the local have N B C block.
For any new medicine, and and and especially the medicine off the bat. So I think the the headline point is the Medicaid will be probably a quarter delay and on the commercial side it'll be heterogeneous the variable the ice.
On a on great I appreciate that kind of thing.
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The staff.
Our next question is from the afternoon or Haney as type of contact Gaffney and your line is open and please go ahead.
Can I team can I get to my checking my questions and number of them for you and maybe the first place the times would be.
How should be thinking about what are the key tech items for parents, we recently received from [noise] Hi.
Hi, Thank detailed probably another I bought inhibitor approved for the treatment of <unk>.
Do you think that pricing could be competitive between P thick and <unk>.
And on the second question is.
What is the likelihood of potentially being able to have the sixth here and then free survival analysis to be part of the label and then I have 1 more call up.
And thanks for the questions I'll touch on the <unk>.
Couple of those points, and then kind of a Peter and talk a little bit about what we do on the patient side to support the access but you know from the from.
Of the addressable market of out of the 2025 and 2500 patients and the us.
Could become eligible right away within the first year of launch.
And then the second question I have is on the timing of March <unk>.
Are you able to comment on.
And how what percentage of total enrollment has been achieved at that point.
And thank you so much for taking from my long question.
And thanks for the question as always.
And I'll start with the finished and then hand it back over the.
Peter to talk about some of the.
Accounting dynamics and patient volume.
On March <unk>.
So as <unk> said in the prepared remarks, we're over 65 patients randomized that study and can go up to 90.
But is not needed to go up to 90, and so we are looking at the balance across the piece ex subtypes.
Trying to hit.
The targeted distributions that were getting quite close to.
Kind of some of the color I can give on the dynamic there.
And of over 65.
And getting close to the completion of <unk> speak to the account and patient funding dynamics.
Sure sure happy to provide a little bit of color there.
And so as I mentioned, our field team has been out there of profiling and those 125 accounts for over 2 months now and.
And certainly finding patients and.
Ultimately were kind of confident and the estimates we've put out there in terms of total number of patients based on those interactions among other data points I think your specific question was about.
How many of them would be kind of initial candidates.
I think.
The.
On the what we see and this is probably not uncommon and any genetic disease.
The phenotypic variability and the presentation and probably of approximately half of the patients with ALS Youll syndrome have pretty severe disease and are really kind of refractory to the available off label therapies that are out there today. So I would kind of think of that as an initial kind of place with the probably a logical place to start.
I think in terms of the.
Utilization, but ultimately building out over time.
We're confident that merrell looks back to address the full population.
Thank you.
Thank you Anthony.
Our next question is from Ed Arce of H C. Wainwright your.
Your line is open. Please proceed with your question.
Christopher the 2 we're also very active looking and uhm markets with partners I pick the deep.
The talked about and are prepared remarks, South Korea, and China, where there's an opportunity for expedited Ah regulatory pathways based on the the U S potential approval as well as the other do you have the fees were it's possible to reach those markets on it and and a relatively attract the timeline and also.
Favorable market access. So we're you know of Europe is certainly part of the international expansion plan, but we think there's a lot of opportunity even outside of the <unk>.
[noise], Okay, and sort of your personal details.
Perhaps still on T sick for the faithfully and must study data that's coming in the second quarter of next year.
Can you guys have any progress with the F. D. A specifically regarding what specific needs of looking for for peace and approval and the U S or are we and.
Holding pattern and until we see the data.
Yeah. Thanks for the the question. So the March P..6 study was extensively and discuss with FDA. So we said we worked everything into that study to address what they're looking for.
So of course, you have to get the results, but we feel confident that the dose of narrow it to that and that study you should see improvements on the activity levels that we saw and the face too and to go study.
And looking forward to that read out and the second quarter.
And Ah sounds good cast 1 last question this 1 for E and.
We went over the major components off and you.
The us launch pad on on of June syndrome can you tell us really rough ballpark cause of each major component and so account and marketing and the patient access programs.
Yeah can I go on.
And the haven't given the specific kind of guidance around the kind of bright downstairs, you think about the the numbers of people and activities and go over the whole of the whole that'll facing expenses. It's it's of fatty it's very small number considering did you think about sales force shake ample we've got.
10, the ex cause of the Chi manages <unk> that 5 people across the U S. So very very compact and efficient model instead of the of the the the commercialization of spam from that perspective.
Okay that sounds scary. Thanks, again for taking the questions and we look forward to look at the Pentagon September.
Yeah, I think that's actually part of the expense almost.
Our next question is from Stephen feet House of Raymond James Stephen Your line of sight and please go ahead.
I guess this is Ryan dishman on <unk> C. T House Uhm wanted to ask and the decision to submit the the M. A H L. G is based on the only on the iconic sticks your event free survival data and I'm worried.
And your New addition of long-term SBA brightest data and there.
It's actually the question so the.
The MAA package that we're working towards for hours, you'll syndrome does include and expanded dataset and so.
And back to some of the early conversations with.
About how the she'll syndrome and.
About pizza, because well frankly and his programs.
If they had asked for incorporating and looking at long term outcomes and.
And and a submission.
Submission.
And so what we have been looking at with the eligible syndrome package and in addition to the iconic which serves as of a pivotal study and how we think about Europe similar to the way it is and the U S. We.
We are looking at supportive analyses on long term event free survival.
You also have the collaboration with the natural history effort looking at outcomes and the pool average yield syndrome. The study so the 3 randomized phase 2 studies and the ultimate and the follow up.
That encompass 86 children and total and the the 6 years of follow up looking at that cohort of patients against a natural history of controls and.
And evaluating event free survival and that's work that's ongoing and will be part of the.
European package.
Okay. That's helpful. And then I also wanted to ask and I can get.
He used to be embark steady.
Familiar of trees, you activate it's like in China view of the Cambridge collaboration and extra on patient studying and willing and.
China.
That's still to come so we are we're active and site startup, but they're not opening and quite yet similar.
Similar will also look at Korea per se.
And the thing with GC farmer.
Okay, and and and then finally in terms of the future market share and.
China, how how different and would you anticipate market penetration to be across the Alley G O P chicken and biliary atresia versus the the U S market.
And thanks for the questionnaire that.
The Hartford really characterize that without.
Kind of training can bridge into the conversation frankly, because there are some country specific launch dynamics debt.
As they get kicked off I don't want to get ahead of speaking for them.
The work on their lawn plans and secret thing the indications.
Okay. Thank you very much for speeches.
Okay.
Thanks for the questions.
Our next question comes from Brian's Corny, if that comes from Brian and your line of items can kind of hack.
The roll and to potentially Registrational portions of the study after the interim time points.
So the the interim analyses will and.
And the guide that goes selection and sizing of the second portion of the size.
Great. Thank you.
Ex depression.
[noise] as a reminder, should you wish to ask any size of questions. Please price stuff on it by 1 on your telephone keypad now.
Our next question is from at February of Guggenheim Partners access your line of sight and please proceed.
Great. Thanks for taking the question a couple for me just the first first you just wanted to maybe get your comments uhm any meaningful takeaways.
From the competitor Piecyk label, and consider and think about 2 and the post March on Peace day Piecyk of next year and.
As it pertains to you know no publications with regulators and secondly, sort of understand that the biliary atresia. The cause of you know on the line cause of the disease not well understood. The just wonder yeah. Your thoughts around sort of you know Derisking L. G. S piece of data sets and general could be as and kind of think about the the biliary atresia.
Read out the and 2023.6.
Yeah, and thanks for the question.
First kind of out of a recent approval and piecyk.
Which is great cause the right to see a product of fruit and it's really terrible indication and and I think from a high level of what we saw there was really on line with expectations based on what we have learned from Fda's.
Viewpoints on looking at.
The writers as.
And and for 1 point and the setting like us and.
And how it all ties into the transplant dynamics and it's just terrible component of this disease.
So instead of out of the great progress points for the space and and how we think about our program as well.
And the moving to the Bill of area Atresia point.
That is at the very different disease than ours, you'll syndrome and perfect.
Because of not only of the head originated but also just the the physical biology and.
And a post kasai setting is the.
It's a different disease.
And which is why we're approaching it and a different way with the Embarq study looking at 6 months bilirubin share my losses.
Those too.
Sarah markers being the the most and looked at and predictive prognostic markers and and looking at transport the survival and some of the recent analysis of been presented at the scientific Congresses.
And so looking to get a reed and a relatively quick on the timeframe on.
On what the potential impact of Maryland, and that is in these kind of quite sick children.
Thank you.
Thank you that Sir we currently have a nice day of the question style hundred per kind of that to cry.
Great. Thank you and thanks for during the call today and for your interest and mirror.
We're excited about the events ahead of us and patients suffering from allergy of some of them are waiting for new treatment options and we look forward to providing on update after the upcoming during the day. Thank you so much.
This concludes today's call. Thank you off the joining have a great rest of your day and you may now disconnect your lines.
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