Q2 2021 Orphazyme A/S Earnings Call

Yeah.

[music].

Welcome to our conference call. Please continue to standby.

Standby your conference will begin shortly.

[music].

Yes.

Yeah.

Good day, ladies and gentlemen, and thank you for standby and welcome to all of US I first half 'twenty 'twenty Wanda Sports Conference call. At this time all participants are in listen only mode. After just because the presentation. There will be a question and answer session to ask a question during the session.

[laughter] deprived who started one earth telephone.

My Love. This conference is being recorded now I would like be trying to call over to Amy Cornbread Investor relation of Arthur said. Please proceed.

Thank you Laurel and good afternoon, everyone and thank you for joining us today.

On the call with me some offers on South, Florida, Chief Executive Officer, Andrew <unk>, Chief Financial Officer, I promised lately.

Medical Officer.

The slides for this call will be available for download on the Investor Relations section of our website.

Please note that the Q&A will take place at the end of the presentation via conference call.

Before we begin I would like to remind you that this call will contain forward looking statements concerning or behind the future expectations plans prospects.

He and performance, which constitute forward looking statements for the purposes of the Safe Harbor provisions under the private Securities Litigation Reform Act of French 95.

Actual results may differ materially from those indicated by these forward looking statements.

Various important factors, including those discussed in our filings with the SEC.

In addition, any forward looking statements represent our views only as of the date of this webcast and should not be relied upon as representing our views as of any subsequent date.

Specifically disclaim any obligations to update such statements.

Now I'll turn the call over to Chris I'll pick up.

Thank you. Thank you Amy welcome everyone and thank you for joining us.

The last few months have been a profound and incentive payouts for offline.

On June 18th we reported received a complete response letter or CRA. All four of them will come also niemann pick disease type C from the U S FDA.

She's very disappointing news forced us to make a series of difficult decisions regarding our business and our people.

And I would like to take this opportunity to thank everyone. In particular, the popping in remaining employees at all of them and the N. P. C patient community for their continued support.

We made those hard decision based on one fact and one conviction.

That is as we sit here today.

The unmet need for patients with MPC remains very high.

The fact is there is still no approved therapy in the U S. For these rare genetic progressively debilitating and often fatal neurodegenerative disease.

And our conviction is and remains the <unk> could provide significant therapeutic benefit for mtc patients alone or in combination with maybe let's start in Europe and in the U S.

This conviction is supported by the strength of the already amongst the most data.

Indeed, our am Okumura has demonstrated statistically significant and clinically meaningful effects on the disease progression in N. P. C. It has shown durable response and it has demonstrated a good safety profile up to 36 months in the open label extension study.

On August 21st of this year the results of our people tell or two study were published in the journal of inherited metabolic disease.

We're very pleased with this publication and it provides validation of our program through a robust peer reviewed scientific process.

Our early access program in the U S, Germany, and France is still underway and we will continue to recruit new patients. We now have more than 100 patients in early access program evenly split between Europe, and the U S and we expect to prolong the early access program until around locomotive becomes commercially available in these markets.

Which is very important to the patient community.

Over the last few months as we've been working diligently to ensure a successful path forward to approval. We are focused only on NPC and have discontinued development of RM optimal for Atlas and IBM and are in the process of discontinuing our trial and negotiate.

EMEA is a review of our immuno mode in NPC is still underway in Europe, and we expect a <unk> opinion by the end of this year with potential marketing authorization in the beginning of next year.

And in the U S. We plan to request a type a meeting with the FDA in the coming weeks.

In summary, we've gone through a lot of changes in the first half of this year, we acted swiftly and decisively we restructured our business to conserve capital maintaining our guidance announced on June 18th in order to focus on our two registration priorities and are focused on leveraging our existing position to secure registration.

From Oklahoma.

With that I'll turn the call over to <unk> to review the numbers in more detail and then I'll wrap up the call and dose.

Thank you Christophe for the first half of the year, Australia, Japan for potential launch of a multimodal NNPC with the receipt of this euro we are taking many steps to conserve capital.

As you can see from this slide in June we began working on our corporate restructuring, we anticipate an approximate 60% cost reduction in total operating expenses for the restructuring while being able to retain our core capabilities to support our medical regulatory and pre commercial activities will.

We will continue to incur some expenses relating to our restructuring program in the second half, but we anticipate a significant reduction in our operating expenses by the end of 2021.

Do you see the financial figures for the first half <unk> recorded net revenues of $15.0 million Danish kroner in the first half of 2021, the revenue relates to the sales of our multiple in France as part of the ongoing <unk> early access program will continue to record revenues in this region on an ongoing base.

Mrs.

Our operating expenses increased to 479 million Danish kroner in the first half of 'twenty, one compared to prior year due to converge Association planning biomarker mode.

R&D expenses were 265 million Danish kroner in the first six months of this year, an increase of 98 million Danish kroner compared to the same period of the prior year.

This increase was largely due to the expenses relating to the Yale is ahead of the trial readouts as well as cost per production of inventory.

General and administrative expenses were 240 million Danish kroner in the first six months of 'twenty, one an increase of 136 million Danish kroner compared to the same period in 2020.

The increase was primarily due to the buildup of our commercial organization, including loans preparations ahead of the potential approved for a modicum of MPC and personnel and support functions to enable our business growth.

Overall, our net loss was 251 Kronos in the first half of 2020 compared to a loss of 464 million Danish kroner in the first half of 2021.

We ended the first half of 2021.

With 334 million Danish kroner in cash compared to 727 billion Danish kroner as at the end of December 2020.

As you see here on slide nine the operating loss operating expense and cash flow guidance. We provided on June 18th remains the same.

For 'twenty, one we anticipate net revenues of between 30% and 40 million Danish kroner relating to unlock more sales from the ongoing EAP in France.

And while the net revenues a positive to our balance sheet, we will need additional capital to advance our multiple towards patients. So we're exploring options to secure funds in the second half of this year.

With that I'll hand over the call to Christophe.

Thanks on us so as we look ahead into the second half of the year. We are focused on executing on our two major priorities EMA approval NFS fall in the U S flooring, Oklahoma.

Our strategy is clear and focused we've taken hard decisions. We've completed the restructuring and we are assessing financing options to secure additional capital for commercialization.

In the next six months and in 2022.

We have several people sort of milestones that could create significant value for offline.

First in Europe. The review of <unk> is ongoing and we do continue to anticipate an opinion from the CHP in Q4 of 2021.

And we will continue to record revenues and book sales.

Pre commercial activities to gain reimbursement in priority EU markets ahead of potential approval our ongoing.

We also expect EMA and UK image Ari approval decisions in the first half of 2022.

In the U S. We will request a type a meeting in the next few weeks to align our regulatory path forward with the FDA.

And despite the headwinds over the last few months.

We do remain committed to our mission to improve the life of MPC patient by delivering on our therapy or in market as quickly as we can.

We look forward to keeping you updated on our progress in the months ahead, now I would like to Hana.

To handle the call back to the moderator for the Q&A session L'oreal bucket too.

Thank you Sir and once again, if you would like to ask question Jess Breakfast bar, one and if you wanted to cancel it just spreads well hashed E. Once again. Please press it's far one for a question.

Yeah.

And sorry. Your first question comes from the line of Vincent Andrews from Guggenheim. Please go ahead. Your line is open.

Hi, Good morning, guys. This is Eddie on for yacht and thanks for taking my question. So your focus has been a remarkable mall can you talk about any of the work youre doing on the BD front to sort of partner with other programs are sort of bring in other assets and so youre, how youre thinking about BD over the next six to 12 months. Thank you.

Thank you Jean Christophe here, so as I said at the moment, we are solely focused on delivering on our two registration milestones one in Europe and one in the U S. A.

And once we have more clarity on this we can now.

What options, we have but that's really the focus of our.

First at the moment.

Thank you.

Once again, if you would like to ask a question just France is star one if you want to withdraw with Joe spreads to Husky.

Yeah.

No more question at this time back to you Chris.

Right.

Perfect and then with that we close the call. Thank you very much.

Good day, everyone Bye bye.

This concludes our conference for today. Thank you for participating you may now all disconnect.

Okay.

[music].

Yes.

[music].

[music].

[music].

[music].

Good afternoon, everyone and thank you for joining us today.

I would make them off is on South, Florida, Chief Executive Officer, Andrew <unk>, Chief Financial Officer.

Lately Chief Medical Officer for.

The slides for this call will be available for download on the Investor Relations section of our website.

Please note that the Q&A will take place at the end of the presentation via conference call.

Before we begin I would like to remind you that this call will contain forward looking statements concerning <unk> future expectations plans prospects.

And performance, which constitute forward looking statements for the purposes of the Safe Harbor provision under the private Securities Litigation Reform Act of French 95.

Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in our filings with the SEC.

In addition, any forward looking statements represent our views only as of the date of this webcast and should not be relied upon as representing our views as of any subsequent date.

Specifically disclaim any obligations to update such statement now I'll turn the call over to Craig Dahl.

Yeah.

Thank you. Thank you Amy welcome everyone and thank you for joining us.

The last few months I've been a profound and incentive payout for offline.

As you know on June 18th we reported received a complete response letter or CRA, all Florida come over for Niemann pick disease type C from the U S FDA.

It's very disappointing news forced us to make a series of difficult decisions regarding our business and our people.

And I would like to take this opportunity to thank everyone in particular, the departing and remaining employees at all for Lyme and the N P C patient community for their continued support.

We made those hard decision based on one fact and one conviction.

That is as we sit here today.

The unmet need for patients with MPC remains very high.

The fact is there is still no approved therapy in the U S. For these rare genetic progressively debilitating and often fatal neurodegenerative disease.

And our conviction is and remains that our EMA will come all could provide significant therapeutic benefits for anticipation alone or in combination with maybe the start in Europe and in the U S.

This conviction is supported by the strength of the IR inbox animal data.

Indeed, our EMA come all has demonstrated statistically significant and clinically meaningful effects on the disease progression in N. P. C. It has shown durable response and has demonstrated a good safety profile up to 36 months in the open label extension study.

On August 21st of this year the results of our people tell Oh two study were published in the journal of in our inherited metabolic disease.

We're very pleased with this publication and it provides validation of our program through a robust peer reviewed scientific process.

Our early access program in the U S, Germany, and France is still underway and we will continue to recruit new patients. We now have more than 100 patients in our early access program evenly split between Europe, and the U S and we expect to prolong the early access program until our remote come out it becomes commercially available in these markets.

Which is very important to the patient community.

Although the last few months as we've been working diligently to ensure a successful path forward to approval. We are focused only on MPC and I've. Just continued development of our AML camel foil us and IBM and are in the process of discontinuing our trial and negotiate.

EMEA is a review of our in Oklahoma and MPC is still underway in Europe, and we expect our kitchen to your opinion by the end of this year with potential marketing authorization at the beginning of next year.

And in the U S. We plan to request a type a meeting with the FTA in the coming weeks.

In summary, we've gone through a lot of changes in the first half of this year, we exited swiftly and decisively we restructured our business to conserve capital and maintaining our guidance announced on June 18th in order to focus on our two registration priorities and are focused on leveraging our existing position to secure registration.

Foreign Muslim.

With that I'll turn the call over to Anders to review the numbers in more detail and then I'll wrap up the call and us.

Thank you Christophe for the first half of the year, Australia was painful potential launch of our multiple NNPC with the receipt of this year, while we are taking many steps to conserve capital.

As you can see from this slide in June we began working on our corporate restructuring, we anticipate an approximate 60% cost reduction in total operating expenses for the restructuring while being able to retain our core capabilities to support our medical regulatory and pre commercial activities will.

We will continue to incur some expenses related to our restructuring program in the second half, but we anticipate a significant reduction in our operating expenses by the end of 2021.

You see the financial figures for the first half <unk> recorded net revenues of $15.0 million Danish kroner in the first half of 2021, the revenue relates to the sales of our muscle in France as part of the ongoing Numerate early access program will continue to record revenues in this region on an ongoing.

<unk>.

Our operating expenses increased to 479 million Danish kroner in the first half of 'twenty, one compared to the prior year due to come on association planning fly them off mall.

R&D expenses were 265 million Danish kroner in the first six months of this year, an increase of 98 million Danish kroner compared to the same period of the prior year.

This increase was largely due to the expenses relating to the AOS and IBM ahead of the trial Readouts as well as cost for the production of inventory.

General and administrative expenses were 240 million Danish kroner in the first six months of 'twenty, one an increase of 136 million Danish kroner as compared to the same period in 2020.

The increase was primarily due to the buildup of our commercial organization, including loans preparations ahead of the potential proof for our ultimate MPC and personnel and support functions to enable our business growth.

Overall, our net loss was 251 <unk> in the first half of 2020 compared to a loss of 464 million Danish kroner in the first half of 2021.

We ended the first half of 2021.

With 334 million Danish kroner in cash compared to 727 million Danish kroner as at the end of December 2020.

As you see here on slide nine the operating loss operating expense and cash flow guidance. We provided on June 18th remains the same.

'twenty, one we anticipate net revenues of between 30% and 40 million Danish kroner is leading to a much more sales from the ongoing EAP in France.

And while the net revenues a positive to our balance sheet will need additional capital to advance <unk> towards patients. So we are exploring options to secure funds in the second half of this year.

With that I'll hand over the call to Christophe.

Thanks on us so as we look ahead into the second half of the year. We are focused on executing on our two major priorities EMA approval <unk> in the U S flooring optimal.

Our strategy is clear and focused we've taken a hard decisions. We've completed the restructuring and we are assessing financing options to secure additional capital for commercialization.

In the next six months and in 2022.

We have several pivotal milestones that could create significant value for <unk>.

First in Europe. The review of <unk> is ongoing and we do continue to anticipate an opinion from the see HMP in Q4 of 2021.

And we will continue to record revenues and book sales.

Our pre commercial activities to gain reimbursement in priority EU markets head of potential approval are ongoing we also expect EMA and UK image Ari approval decisions in the first half of 2022.

In the U S. We will request a type a meeting in the next few weeks to align our regulatory path forward with the FDA.

And despite the headwinds over the last few months.

We do remain committed to our mission to improve the life of mtc patients by delivering another therapy are removed.

As quickly as we can.

We look forward to keeping you updated on our progress in the months ahead.

Now I would like to and then the handle the call back to the moderator for the Q&A session Laura bucket too.

Thank you Sir and once again, if you would like to ask question Jess Breakfast bar, one and if you want to cancel it just spreads well hashed E. Once again. Please press it's far one for a question.

Yeah.

Yeah.

And sorry. Your first question comes from the line of the essence in Nash.

Probably Guggenheim. Please go ahead your line is open.

Hi, Good morning, guys. This is Eddie on for yacht and thanks for taking my question. So your focus has been our remarks, one more can you talk about any of the work youre doing on the BD front or partner with other programs are sort of bring in other assets and so youre, how youre thinking about BD over the next six to 12 months. Thank you.

Thank you Jean Christophe here, so as I said at the moment, we are solely focused on delivering on our two registration.

Also <unk> won in Europe, and one in the U S.

And once we have more clarity on this we're going to assess what options, we have but thats really the focus of our.

First at the moment.

Thank you.

Okay.

Once again, if you would like to ask a question just breakfast by one and if you want to withdraw with Joe's spreads for Husky.

No more question at this time back to you Chris.

Right.

Perfect and then with that we close the call. Thank you very much.

Good day, everyone Bye bye.