Strategic Partners A/S

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00:40 Thank you, Loral and good afternoon, everyone. And thank you for joining us today.

On the call with me from Orphazyme are Christophe Bourdon, Chief Executive Officer; Anders Vadsholt, Chief Financial Officer, and Thomas Blaettler, Chief 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 and A will take place at the end of the presentation via a conference call. 01:04 Before we begin, I would like to remind you that this call contain forward looking statements concerning future expectations plans, prospects, public strategy and performance, which constitute forward looking statements for the purposes of the Safe Harbor Provisions, under the Private Securities Litigation Reform Act of nineteen ninety five.

01:23 Actual results may differ materially from those indicated by these forward statements as a result of very 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.

We specifically disclaim any obligations to update such statements. 1:45 Now, I'll turn the call over to Christophe.

Christophe?

01:49 Thank you. Thank you, Amy.

Welcome, everyone, and thank you for joining us. The last few months have been a profound and intensive period for Orphazyme.

As you know, on June eighteen, we reported receipt of a Complete Response Letter of CRL for arimoclomol for Niemann-Pick disease type C from the U.S. FDA.

This 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 Orphazyme and the NPC patient community for their continued support.

02:28 We made those hard decisions based on one facts and one conviction. Fact is, as we sit here today, the unmet need for patients with NPC remains very high.

Fact is, there is still no approved therapy in the U.S. for this rare, genetic, debilitating and often fatal neurodegenerative disease.

And our conviction is and remains that could provide significant therapeutic benefits for NPC patients alone or in combination with miglustat in Europe and in the U.S. This conviction is supported by the strength of the arimoclomol data.

03:08 Indeed arimoclomol has demonstrated statistically significant and clinically meaningful effects on the progression in NPC. It has shown the response and has demonstrated a good safety profile up to thirty six months in the open-label extension study.

03:25 On August twenty one of this year, the results of our Pivotal 002 Study were published in the journal of inherited disease. We're very pleased with this publication, it provides validation of our program through a robust peer-reviewed scientific process.

03:43 Our Early Access Program in the U.S., Germany, and France is still on way, and we will continue to recruit new patients. We now have more than one hundred patients in our Early Access Program evenly split between Europe and the U.S.

And we expect to prolong the Early Access Program until arimoclomol becomes commercially available in these markets, which is very important to the patient community. 04:06 Over the last few months, we've been working diligently to ensure a successful path forward to approval.

We are focused only on NPC and have discontinued development of arimoclomol for ALS and IBM and are in the process of discontinuing our trial in . 04:25 EMA’s review of arimoclomol in NPC is still on the way in Europe and we expect a CHMP 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. 04:43 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 eighteen in order to focus on the two registration priorities and are focused on leveraging our existing position to secure registration for arimoclomol. 05:04 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.

Anders?

5:12 Thank you, Christophe. For the first half of the year, our strategy was to pay for potential launch of arimoclomol in NPC with the receipt of the CRP, we have taken many steps to conserve capital.

As you can see from this slide, in June, we began working on our coverage restructuring, we anticipate an approximate sixty percent 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. 05:41 We'll 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 twenty twenty one.

(ph) financial figures for the first half, over some recorded, net revenues of thirteen point two million Danish kroner in the first half of twenty twenty one. The revenue relates to the sales of arimoclomol in France as part of the ongoing remunerated early access program will continue to record revenues in this region on an ongoing basis.

06:14 Our operating expenses increased to four hundred and seventy nine million Danish kroner in the first half of twenty one compared to the prior year due to association planning for arimoclomol. 06:24 R&D expenses were two sixty five million Danish kroner in the first six months of this year, an increase of ninety eight million Danish kroner compared to the same period of the prior year.

This increase was largely due to the expenses relating to the ALS and IBM ahead of the trial readouts, as well as cost for the production of inventory. 06:44 General and administrative expenses were two forty million billion Danish kroner in the first six months of twenty one, an increase of (ph) million Danish kroner compared to the same period in twenty twenty.

The increase was primarily due to the build of our commercial organization, including the loans preparations ahead of the potential approved for arimoclomol NPC and personnel and support functions to enable our business growth. 07:08 Overall, our net loss was two fifty one kroner in the first half of twenty twenty compared to a loss of four hundred and fifty four million Danish kroner in the first half of twenty twenty one.

We ended the first half of twenty twenty one with three thirty four million Danish kroner in cash, compared to seven twenty seven million Danish kroner at the end of December twenty twenty. 07:34 As you see here on slide nine, the operating loss, operating expense, and cash flow guidance we provided on June eighteen remains the same.

For twenty one, we anticipate net revenues of between thirty and forty million Danish kroner relating to arimoclomol sales from the ongoing EAP in France. 07:52 And while the net revenues are positive to our balance sheet, we'll need additional capital to advance arimoclomol towards patients to exploring options to secure funds in the second half of this year.

08:03 With that, I'll will hand over the call to Christophe.

08:07 Thanks, Anders. So, as we look ahead into the second half of the year, we are focused on executing on two major priorities.

EMA approval and our path forward in the U.S. for arimoclomol.

Our strategy is clear and focused. We've taken hard decisions.

We've completed the restructuring and we are accessing financing options to secure additional capital for commercialization. 08:34 In the next six months and in twenty twenty two, we have several pivotal and milestones that could create significant value for alpha line.

First in Europe, the review of arimoclomol is ongoing and we do continue to anticipate an opinion from the CHMP in Q4 of twenty twenty one. And we will continue to record revenues and book sales.

08:57 Our pre-commercial activities to gain reimbursement in priority EU markets ahead of potential approval ongoing. We also expect EMA and UK MHRA approval decisions in the first half of twenty twenty two.

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 NPC patients by delivering (ph) therapy arimoclomol as quickly as we can.

09:31 We look forward to keeping you updated on our progress in the months ahead. Now, we like to hand the call back to the moderator for the Q and A session.

Loral, back at you.

10:12 Hi, good morning guys. This is (ph) on for Yatin.

Thanks for taking my question. So, your focus has been arimoclomol, can you talk about any of the work you're doing on the BD front to sort of partner with other programs or sort of bring in other assets?

So, how you're thinking about BD over the next six to twelve months? Thank you.

10:30 Thank you. This is Christophe here.

So, as I said, you know, at the moment, we are solely focused on delivering on our two registration milestones one 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 that's really the focus of us at the moment.

10:54 Thank you.

11:21 Perfect. And then with that, we close the call.

Thank you very much. Have a good day everyone.

Bye, bye.