Zevra Therapeutics Inc (ZVRA) 2024 Q2 法說會逐字稿

Hello, and thank you for joining the Zevra Therapeutics' Q2 2024 financial results And corporate highlights call.

您好，感謝您參加 Zevra Therapeutics 的 2024 年第二季財務業績和公司亮點電話會議。

Today's call is being recorded and will be made available on the company's website following the conclusion of the call.

今天的電話會議正在錄音，電話會議結束後將在公司網站上發布。

With that, I will now turn the call over to Nichol Ochsner, Vice President of Investor Relations and Corporate Communications for Zevra Therapeutics.

現在，我將把電話轉交給 Zevra Therapeutics 投資者關係和企業傳播副總裁 Nichol Ochsner。

Good afternoon, and thank you for joining us today to review Zevra Therapeutics progress in the second quarter of 2024, outlining our clinical advances, operational achievements and financial results.

下午好，感謝您今天與我們一起回顧 Zevra Therapeutics 在 2024 年第二季度的進展，概述我們的臨床進展、營運成就和財務業績。

Before we get started, let me take a moment to provide some important information.

在我們開始之前，讓我花點時間提供一些重要資訊。

I encourage you to access the news release, which was published this afternoon and is available in the Investors section of the Zevra website.

我鼓勵您閱讀今天下午發布的新聞稿，該新聞稿可在 Zevra 網站的投資者部分找到。

As we begin our call, it's important to highlight that certain information covered in today's discussions will include forward-looking statements.

當我們開始通話時，需要強調的是，今天討論中涉及的某些資訊將包含前瞻性陳述。

We caution listeners that actual results could differ materially from these stated or implied by these forward-looking statements due to risks and uncertainties associated with the company's business.

我們提醒聽眾，由於與公司業務相關的風險和不確定性，實際結果可能與這些前瞻性陳述中明示或暗示的結果有重大差異。

Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other significant factors that may lead to actual results differing materially from those projections made.

前瞻性陳述並非承諾或保證，本質上會受到風險、不確定性和其他重大因素的影響，這些因素可能導致實際結果與預測有重大差異。

These forward-looking statements are qualified by the cautionary statements contained in the Risk Factors section in our most recent quarterly report on Form 10-Q and our other filings with the SEC annual report on Form 10-K.

這些前瞻性陳述受到我們最近的 10-Q 表季度報告的風險因素部分以及我們向 SEC 提交的 10-K 表年度報告的其他文件中風險因素部分所包含的警示性陳述的限制。

I'm pleased to welcome Zevra's management team members participating in today's call.

我很高興歡迎 Zevra 的管理團隊成員參加今天的電話會議。

I'm joined by Neil McFarlane, President and Chief Executive Officer; LaDuane Clifton, our Chief Financial Officer; Josh Schafer, our Chief Commercial Officer and Executive Vice President of Business Development; and Adrian Quartel, our Chief Medical Officer.

總裁兼執行長尼爾麥克法蘭 (Neil McFarlane) 也加入了我的行列。 LaDuane Clifton，我們的財務長； Josh Schafer，我們的首席商務長兼業務開發執行副總裁；以及我們的首席醫療官 Adrian Quartel。

Now I'll turn the call over to Neil.

現在我將把電話轉給尼爾。

Thank you, Nichol, and thank you all for making the time to join us today.

謝謝你，尼科爾，也謝謝大家今天抽出時間來加入我們。

During the second quarter, we made steady progress executing on our strategic objectives, preparing for the advisory committee meeting and the potential launch of arimoclomol, driving the launch of OLPRUVA and advancing KP1077 for sleep disorders.

第二季度，我們在執行策略目標方面取得了穩步進展，為諮詢委員會會議和阿莫洛莫的潛在上市做準備，推動了 OLPRUVA 的上市，並推進了治療睡眠障礙的 KP1077。

These objectives are important building blocks for our long-term strategic plan to build a sustainable rare disease company with reliable cash flows.

這些目標是我們長期策略計畫的重要組成部分，旨在建立一家具有可靠現金流的可持續罕見疾病公司。

An important element of executing against our strategy is to build and maintain our position of financial strength.

執行我們策略的一個重要因素是建立和維持我們的財務實力地位。

During the first half of 2024, we made investor outreach a primary objective, highlighting the opportunities and catalysts for value creation that we have as a company.

2024 年上半年，我們將投資者外展作為主要目標，強調我們作為一家公司所擁有的創造價值的機會和催化劑。

There has been a significant interest and growing momentum as we continue to execute our objectives.

隨著我們繼續執行我們的目標，人們產生了濃厚的興趣並且勢頭不斷增長。

Last week, following the favorable outcome of the FDA Advisory Committee meeting focused on arimoclomol, we undertook a modestly sized underwritten public offering to capture that momentum and build on our base of investors as we lean into the potential of our near-term catalysts.

上週，在FDA 諮詢委員會會議重點討論阿莫洛莫取得良好成果後，我們進行了規模適中的承銷公開發行，以抓住這一勢頭，並在我們挖掘近期催化劑潛力的同時，鞏固我們的投資者基礎。

With this funding, we have added net proceeds of approximately $64.5 million to our balance sheet, bringing our pro forma June 30, 2024, cash, cash equivalents and investments to $113.8 million.

透過這筆資金，我們的資產負債表中增加了約 6,450 萬美元的淨收益，使我們預計 2024 年 6 月 30 日的現金、現金等價物和投資達到 1.138 億美元。

This was the right time to demonstrate our confidence and take this important step to prepare for success.

現在是展現我們信心並邁出這一重要步驟為成功做好準備的正確時機。

The proceeds will extend our cash runway and bolster our flexibility in executing both near- and long-term objectives, including full preparation for the potential launch of arimoclomol and the flexibility to accelerate our clinical pipeline.

所得將擴大我們的現金跑道，並增強我們執行近期和長期目標的靈活性，包括為可能推出的阿莫洛莫做好充分準備，以及加快我們的臨床管道的靈活性。

Now I'll share a summary of our key second quarter accomplishments and outline why we remain optimistic in our ability to deliver on our strategic plan.

現在，我將分享我們第二季主要成就的摘要，並概述為什麼我們對實現策略計畫的能力保持樂觀。

Let's start with arimoclomol.

讓我們從阿莫洛莫開始。

Our product candidate for Niemann Pick Disease Type C or NPC.

我們針對 C 型尼曼皮克氏症或 NPC 的候選產品。

On August 2, the Genetic Metabolic Diseases Advisory Committee, or GMDC, voted favorably that arimoclomol is effective in the treatment of NPC.

8 月 2 日，遺傳代謝疾病諮詢委員會 (GMDC) 投票贊成阿莫洛莫對治療鼻咽癌有效。

While the vote is not binding, we believe this is an important factor as the FDA completes its review.

雖然投票不具約束力，但我們認為這是 FDA 完成審查的重要因素。

Our PDUFA date is September 21, which is fast approaching.

我們的 PDUFA 日期是 9 月 21 日，很快就到了。

We received the first round of labeling comments last Friday and are working closely with the FDA.

我們上週五收到了第一輪標籤意見，並與 FDA 密切合作。

If approved, arimoclomol would be the first drug in the US indicated for the treatment of NPC, and it would be eligible for a priority review voucher.

如果獲得批准，阿莫洛莫將成為美國第一個用於治療鼻咽癌的藥物，並將有資格獲得優先審查券。

As a reminder, there are roughly 900 people in the US with NPC, of which approximately a third are diagnosed and treated.

提醒一下，美國大約有 900 名鼻咽癌患者，其中約三分之一被診斷和治療。

More than 70 of those patients are currently enrolled in our US Expanded Access Program, or EAP.

其中 70 多名患者目前已加入我們的美國擴展准入計劃 (EAP)。

As the FDA review continues, we will maintain the US EAP to ensure access for patients until commercial supply is available.

隨著 FDA 審查的繼續，我們將維持美國 EAP，以確保患者能夠使用，直到商業供應可用。

In addition, subsequent to US approval, we will seek regulatory approval in Europe, where an additional 70 to 80 patients are enrolled in our global EAP program.

此外，在美國獲得批准後，我們將在歐洲尋求監管部門的批准，屆時將有 70 至 80 名患者加入我們的全球 EAP 計畫。

We are actively preparing for the commercial launch of arimoclomol.

我們正在積極準備阿莫洛莫的商業上市。

Our commercial infrastructure was built to optimize the strategic fit between OLPRUVA and arimoclomol.

我們的商業基礎設施旨在優化 OLPRUVA 和阿莫洛莫之間的策略契合度。

Both products address genetic metabolic diseases with multidisciplinary treatment teams that are often co-located within the same centers of excellence, allowing us to reach the majority of prescribers with a targeted commercial infrastructure.

這兩種產品都透過多學科治療團隊來治療遺傳代謝疾病，這些治療團隊通常位於同一卓越中心，使我們能夠透過有針對性的商業基礎設施接觸到大多數處方者。

Our rare disease specialists and medical science liaisons, who are currently promoting and educating on OLPRUVA and Urea Cycle Disorders, or UCDs, are also engaging with prescribers when appropriate to raise awareness for NPC.

我們的罕見疾病專家和醫學聯絡員目前正在宣傳和教育 OLPRUVA 和尿素循環障礙 (UCD)，並在適當的時候與處方醫生合作，以提高對 NPC 的認識。

Further, our market access team has initiated pre-approval clinical discussions with payers regarding arimoclomol as a potential treatment for NPC.

此外，我們的市場准入團隊已開始與付款人就阿莫洛莫作為鼻咽癌的潛在治療方法進行預先批准臨床討論。

These prelaunch activities will help ensure patients have access once available.

這些啟動前活動將有助於確保患者一旦可用即可使用。

Now turning to OLPRUVA where we continue to make process with the commercial launch initiated at the end of January 2024.

現在轉向 OLPRUVA，我們繼續進行流程，並於 2024 年 1 月底啟動商業發布。

Over the last few quarters, our assumption on the limited awareness of OLPRUVA has been confirmed.

在過去的幾個季度中，我們對 OLPRUVA 認知度有限的假設得到了證實。

However, we've made progress with healthcare providers to increase awareness levels and identify that we have more work to do to increase patient awareness.

然而，我們與醫療保健提供者在提高意識水平方面取得了進展，並認識到我們還有更多工作要做，以提高患者的意識。

As a reminder, there are 1,100 UCD patients in the US of which more than 800 are receiving treatment.

提醒一下，美國有 1,100 名 UCD 患者，其中 800 多人正在接受治療。

The prescribing community has identified a significant number of these patients who can benefit from OLPRUVA.

處方界已確定大量患者可以從 OLPRUVA 中受益。

While we are encouraged by their response, the number of patient enrollments is not yet where we would like it to be.

雖然我們對他們的反應感到鼓舞，但患者登記數量尚未達到我們希望的水平。

During the second quarter, we had nine new patient enrollments, which we define as a prescription for a patient who's on our Quick Start program or one who is receiving a paid dispense.

在第二季度，我們登記了 9 名新患者，我們將其定義為參加我們的快速啟動計畫的患者或正在接受付費配藥的患者的處方。

We have been working to build awareness amongst clinicians who treat UCD and to ensure market access for patients.

我們一直致力於提高治療 UCD 的臨床醫生的認識，並確保患者的市場准入。

The team has done an outstanding job of engaging HCPs at target centers of excellence and at medical conferences to build brand awareness.

團隊在吸引 HCP 參與目標卓越中心和醫學會議以建立品牌知名度方面做得非常出色。

The team has successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients, which is remarkable since access to HCPs has become more challenging across the industry.

團隊成功地吸引了大多數診斷和治療 UCD 患者的臨床醫生和思想領袖，這一點非常了不起，因為整個產業獲得 HCP 變得越來越困難。

Additionally, our managed care team continues to engage with government and commercial payers to ensure broad access for patients.

此外，我們的管理式醫療團隊繼續與政府和商業付款人合作，以確保患者獲得廣泛的服務。

We have increased OLPRUVA coverage to 75% of covered lives with improved formulary status across healthcare plans and have established comprehensive patient services programs designed to assist with the reimbursement hurdles experienced by the rare disease community.

我們已將 OLPRUVA 覆蓋率提高到受保人的 75%，改善了整個醫療保健計劃的處方狀態，並建立了全面的患者服務計劃，旨在幫助解決罕見疾病群體所遇到的報銷障礙。

As I mentioned earlier, there are always opportunities for refinement during launch, and we are implementing changes to improve patient engagement.

正如我之前提到的，在發布過程中總是有改進的機會，我們正在實施變革以提高患者的參與度。

One key change was our transition to Orsini as our specialty pharmacy partner, who is a leader in pharmacy solutions for rare diseases.

一個關鍵的變化是我們轉向 Orsini 作為我們的專業藥房合作夥伴，Orsini 是罕見疾病藥房解決方案的領導者。

This transition completed in mid-June, including a rebalance of our OLPRUVA inventory in the channel, which impacted our net sales revenue in the quarter.

這項過渡於 6 月中旬完成，包括對通路中的 OLPRUVA 庫存進行重新平衡，這影響了我們本季的淨銷售收入。

LaDuane will provide more details later in the call.

拉杜安將在稍後的電話會議中提供更多細節。

We believe these enhancements to our commercial infrastructure will further support the OLPRUVA launch and will have a positive impact on our commercialization efforts for arimoclomol with limited incremental cost.

我們相信，這些對我們商業基礎設施的增強將進一步支持 OLPRUVA 的上市，並將以有限的增量成本對我們的阿莫洛莫商業化工作產生積極影響。

Now I'd like to turn your attention to KP1077, our clinical candidate for the treatment of idiopathic hypersomnia, or IH, a rare chronic sleep disorder.

現在我想請您關注 KP1077，這是我們用於治療特發性睡眠過度（IH）（一種罕見的慢性睡眠障礙）的臨床候選藥物。

IH is characterized by excessive daytime sleepiness and difficulty waking, also known as sleep inertia.

IH 的特徵是白天過度嗜睡和難以醒來，也稱為睡眠慣性。

This disease impacts approximately 37,000 people in the US As you may recall KP1077 is comprised of serdexmethylphenidate or SDX, which was designed to steadily release the methylphenidate, its active ingredient.

這種疾病影響了美國約 37,000 人。

Its unique pharmacokinetic profile allows for flexible dosing to overcome these primary IH symptoms and ensures patients receive the optimal drug concentration during waking hours.

其獨特的藥物動力學特徵允許靈活的劑量來克服這些主要的 IH 症狀，並確保患者在清醒時接受最佳的藥物濃度。

SDX is currently designated as a schedule for controlled substance by the US Drug Enforcement Administration due to demonstrated lower risk or abuse potential.

由於已證明 SDX 風險或濫用潛力較低，目前已被美國緝毒局指定為受管制物質表。

At the SLEEP 2024 meeting in June, we presented the pharmacokinetics of SDX when administered in the morning and at night.

在 6 月的 SLEEP 2024 會議上，我們介紹了早上和晚上服用 SDX 的藥物動力學。

The clinical data showed peak exposure occurs the morning after a nighttime dose when the patient needs it most to manage sleep inertia.

臨床數據顯示，夜間服藥後的早晨出現暴露高峰，此時患者最需要它來控制睡眠慣性。

We also reported positive results from our Phase II clinical study of KP1077 in patients with IH.

我們也報告了 KP1077 在 IH 患者的 II 期臨床研究的正面結果。

In this proof-of-concept study, KP1077 was well tolerated at all dose levels, including the notably high dose of 320 milligrams daily.

在這項概念驗證研究中，KP1077 在所有劑量水平下均具有良好的耐受性，包括每日 320 毫克的極高劑量。

Adverse events throughout the study were mild similar to other methylphenidate products and did not lead to early discontinuation.

整個研究中的不良事件與其他哌醋甲酯產品相似，輕微，且不會導致提前停藥。

KP1077 showed clinically meaningful benefits in change from baseline at the end of seven weeks of treatment against secondary and exploratory endpoints, which included change in the Epworth Sleepiness Scale, the IH Severity Scale, the Sleep Inertia Visual Analog Scale and a relatively new scale to assess the symptoms and severity of brain fog.

KP1077 在治療七週結束時相對於次要和探索性終點的基線變化顯示出具有臨床意義的益處，其中包括Epworth 嗜睡量表、IH 嚴重程度量表、睡眠惰性視覺模擬量表和相對較新的評估量表的變化腦霧的症狀和嚴重程度。

We are encouraged by these results showing that KP1077 is well tolerated and demonstrates clinically meaningful benefits.

我們對這些結果感到鼓舞，這些結果表明 KP1077 具有良好的耐受性並表現出具有臨床意義的益處。

Importantly, the study successfully fulfilled the objectives of informing the design of a pivotal efficacy trial.

重要的是，該研究成功實現了為關鍵療效試驗的設計提供資訊的目標。

We consulted with key opinion leaders, payers and patient advocates knowledgeable in the rare sleep space to help interpret these results and have submitted a briefing book to the FDA for an end of Phase II meeting at the end of the third quarter.

我們諮詢了對罕見睡眠空間有豐富知識的關鍵意見領袖、付款人和患者權益倡導者，以幫助解釋這些結果，並已向FDA 提交了一份簡報，以便在第三季度末召開第二階段會議。

With only one FDA-approved treatment, there remains a large unmet need for therapies to address the symptoms of IH.

由於只有一種 FDA 批准的治療方法，針對 IH 症狀的治療方法仍有大量未滿足的需求。

We are conducting market research on the Phase II data to better understand KP1077's differentiated profile, position in the treatment landscape and to inform our business case.

我們正在對 II 期數據進行市場研究，以更好地了解 KP1077 的差異化概況、在治療領域的地位，並為我們的業務案例提供資訊。

Finally, we've made progress with Celiprolol, our product candidate for the treatment of vascular Ehler-Danlos Syndrome or VEDS, which impairs COL3A1 connective tissue and leads to vascular and hollow organ ruptures.

最後，我們在塞利洛爾（Celiprolol）方面取得了進展，這是我們治療血管埃勒-當洛斯綜合徵（VEDS）的候選產品，該綜合徵會損害COL3A1 結締組織並導致血管和中空器官破裂。

Celiprolol's mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation.

塞利洛爾的作用機制旨在透過血管擴張和平滑肌鬆弛來減少動脈壁內膠原纖維的機械應力。

Celiprolol is the primary treatment option in various EU countries and we believe it could address the significant unmet need in the US as there are no approved treatments for the 7,500 patients with VEDS.

塞利洛爾是多個歐盟國家的主要治療選擇，我們相信它可以解決美國未滿足的重大需求，因為目前尚無針對 7,500 名 VEDS 患者的批准治療方法。

Celiprolol has received both orphan drug and breakthrough therapy designations from the FDA.

塞利洛爾已獲得 FDA 的孤兒藥和突破性治療指定。

During the second quarter, we restarted recruitment of the Celiprolol Phase III trial, also known as the DiSCOVER trial.

第二季度，我們重新啟動了 Celiprolol III 期試驗（也稱為 DiSCOVER 試驗）的招募。

This decentralized event-driven trial is being conducted under a special protocol assessment.

這項分散的事件驅動試驗是在特殊方案評估下進行的。

We are encouraged by the significant interest among patients to enroll in this trial which has exceeded our expectations, underscored the unmet need within the VEDS community and preserves the value of the program while we conduct our portfolio review.

我們對患者參加這項試驗的濃厚興趣感到鼓舞，這超出了我們的預期，強調了 VEDS 社區內未滿足的需求，並在我們進行投資組合審查時保留了該計劃的價值。

As part of the strategic planning initiative kicked off in January, we continue to assess the value of each of our programs.

作為一月份啟動的策略規劃計劃的一部分，我們將繼續評估每個計劃的價值。

Our intent is to fully understand the unmet needs of the rare disease patient community within a potential market and then develop a solid clinical and business case for how Zevra can develop therapies to address those needs.

我們的目的是充分了解潛在市場中罕見疾病患者群體未滿足的需求，然後開發可靠的臨床和商業案例，說明 Zevra 如何開發療法來滿足這些需求。

Looking ahead, we have three key priorities.

展望未來，我們有三個關鍵優先事項。

First, to receive approval and successfully launch arimoclomol by leveraging the infrastructure built for OLPRUVA; Second, to drive the launch of OLPRUVA; and third, to discuss the design for a pivotal study evaluating the efficacy of KP1077 in patients with IH in our end of Phase II meeting at the end of Q3.

首先，利用為 OLPRUVA 建造的基礎設施獲得批准並成功推出 arimoclomol；二是推動OLPRUVA的上市；第三，在第三季末的 II 期會議結束時討論一項關鍵研究的設計，該研究評估 KP1077 對 IH 患者的療效。

We remain focused on execution to deliver a strong second half of 2024 and are well positioned financially to execute against those objectives.

我們仍然專注於執行力，以在 2024 年下半年實現強勁成長，並在財務上處於有利地位來實現這些目標。

Now LaDuane will provide an update on our financial results.

現在，拉杜安將提供我們財務業績的最新資訊。

Thank you, Neil, and good afternoon.

謝謝你，尼爾，下午好。

As we begin, I encourage you to refer to our quarterly report on Form 10-Q, which we intend to file later today for more detailed information.

在我們開始之前，我鼓勵您參考我們的 10-Q 表格季度報告，我們打算在今天稍後提交該報告以獲取更多詳細資訊。

We have made meaningful progress during the second quarter, and our financial results also reflect discipline in our capital allocation to drive towards success in reaching our strategic objectives.

我們在第二季度取得了有意義的進展，我們的財務表現也反映出我們在資本配置方面的紀律，以推動成功實現我們的策略目標。

Our second-quarter results included net revenue of $4.4 million, which includes $3.1 million in net reimbursements from the French EAP for arimoclomol and $1.3 million of royalties and other reimbursements under the AZSTARYS license.

我們第二季的業績包括 440 萬美元的淨收入，其中包括法國 EAP 對阿莫洛莫的淨報銷額為 310 萬美元，以及 AZSTARYS 許可下的特許權使用費和其他報銷額為 130 萬美元。

For our commercial product, OLPRUVA, we recognize commercial product revenue when shipments are received by our specialty pharmacy.

對於我們的商業產品 OLPRUVA，我們在我們的專業藥局收到出貨時確認商業產品收入。

And as we previously announced, we transitioned to a new specialty pharmacy during the quarter, which required us to ship new product and recognize returns from the prior specialty pharmacy, which offset revenue for the period.

正如我們之前宣布的，我們在本季度過渡到新的專業藥房，這要求我們運送新產品並確認先前的專業藥房的回報，這抵消了該期間的收入。

The result was de minimis revenue recognized during Q2.

結果是第二季度確認了微量收入。

We believe this transition will lead to improved patient services as enrollments grow.

我們相信，隨著註冊人數的增加，這種轉變將導致患者服務的改善。

Additionally, Cost of goods sold was inflated during Q2 due to recognition of a $3.2 million obsolescence reserve against OLPRUVA inventory, which is nearing expiration.

此外，由於 OLPRUVA 庫存已接近到期，確認了 320 萬美元的過時準備金，因此第二季的銷售成本有所上漲。

This excess inventory was ordered prior to our acquisition of Acer and the previous delay in the products launch impacted the rate of usage, leading to the need for this reserve to be recognized in the quarter.

這些多餘的庫存是在我們收購宏碁之前訂購的，先前產品發布的延遲影響了使用率，導致需要在本季度確認該儲備。

Our R&D expenses for the second quarter were $10.5 million, which is a slight decrease compared to the first quarter of 2024 and primarily due to the completion of the KP1077 Phase II trial.

我們第二季的研發費用為1050萬美元，與2024年第一季相比略有下降，主要是由於KP1077第二期試驗的完成。

Selling, general, and administrative expenses were $12.6 million during second quarter, reflecting our commercial team being in place for the entire quarter and actively engaged in activities to build awareness and provide patient services related to OLPRUVA.

第二季的銷售、一般和管理費用為 1,260 萬美元，反映出我們的商業團隊在整個季度都在到位，並積極參與了提高 OLPRUVA 意識和提供病患服務的活動。

Net loss for Q2 2024 was $19.9 million or $0.48 per basic and diluted share which reflects an increase driven by our investments in our commercial infrastructure.

2024 年第二季的淨虧損為 1,990 萬美元，或每股基本股和稀釋股 0.48 美元，這反映了我們對商業基礎設施投資推動的成長。

At the beginning of the quarter, we announced the refinancing of our existing debt with a new $100 million credit facility from which we took an initial draw of $60 million.

在本季初，我們宣布透過一項新的 1 億美元信貸安排對現有債務進行再融資，我們從中首次提取了 6,000 萬美元。

A second tranche of up to $20 million is available at our discretion until October 5, 2025.

我們可酌情在 2025 年 10 月 5 日之前提供第二筆最多 2,000 萬美元的資金。

And a third tranche of up to $20 million will become available upon approval of arimoclomol, in each case, subject to certain terms and conditions.

在阿莫洛莫獲得批准後，將根據特定條款和條件提供第三筆高達 2000 萬美元的資金。

As of June 30, 2024, total cash, cash equivalents and investments were $49.3 million, which was a decrease of $3.4 million compared to March 31.

截至2024年6月30日，現金、現金等價物及投資總額為4,930萬美元，較3月31日減少340萬美元。

Use of cash during the period was $17.4 million, offset by net proceeds of $14 million from our initial draw from our credit facility.

在此期間使用的現金為 1,740 萬美元，被我們最初從信貸額度中提取的 1,400 萬美元淨收益所抵消。

Total long-term debt was $58.3 million as of June 30, 2024.

截至 2024 年 6 月 30 日，長期債務總額為 5,830 萬美元。

As Neil mentioned earlier, we successfully completed a modestly sized underwritten public offering, which brought $64.5 million in net proceeds to Zevra, along with attracting a cadre of institutional investors well known in the biotech industry as long-term supporters of innovation and solid execution.

正如尼爾之前提到的，我們成功完成了規模適中的承銷公開發行，為Zevra 帶來了6450 萬美元的淨收益，同時吸引了一群在生物技術行業眾所周知的創新和穩健執行的長期支持者的機構投資人。

We issued approximately 10.6 million shares at a price of $6.50 per share.

我們以每股 6.50 美元的價格發行了約 1,060 萬股股票。

This offering was significantly oversubscribed, which testifies to the strong sentiment around the potential of the several upcoming value creation opportunities for Zevra.

此次發行獲得了大幅超額認購，這證明了人們對 Zevra 即將到來的幾個價​​值創造機會的潛力的強烈情緒。

Combining the net proceeds from this offering with our existing resources, pro forma June 30, 2024, cash, cash equivalents and investments is $113.8 million.

將本次發行的淨收益與我們現有的資源結合，預計到 2024 年 6 月 30 日，現金、現金等價物和投資為 1.138 億美元。

Our cash runway now extends into Q1 2027.

我們的現金跑道現已延伸至 2027 年第一季。

Pro forma common and fully diluted shares outstanding were 52.6 million and 67.9 million, respectively.

備考普通股和完全稀釋後已發行股分別為 5,260 萬股和 6,790 萬股。

No warrants were issued in connection with this offering.

沒有發行與本次發行相關的認股權證。

It is important to note that our cash run rate guidance is based on our current operating plan, available cash, cash equivalents and investments including the additional cash received through our recently completed secondary offering and are subject to continued compliance with our debt covenants.

值得注意的是，我們的現金運行率指引是基於我們目前的營運計畫、可用現金、現金等價物和投資，包括透過我們最近完成的二次發行收到的額外現金，並且需要繼續遵守我們的債務契約。

Our forecast includes commercial revenue from the sales of OLPRUVA, reimbursements from the French EAP for arimoclomol and ongoing royalties under the AZSTARYS license agreement.

我們的預測包括 OLPRUVA 銷售的商業收入、法國 EAP 對阿莫洛莫的報銷以及 AZSTARYS 許可協議下持續的特許權使用費。

It does not include commercial revenue from sales of arimoclomol nor the sale of the PRV, which would follow FDA approval.

它不包括阿莫洛莫銷售或 PRV 銷售的商業收入，這將在 FDA 批准後進行。

We are optimistic about the outlook and our focus is to create long-term value for shareholders through disciplined execution against our plan in support of our mission to become a leading rare disease company.

我們對前景持樂觀態度，我們的重點是透過嚴格執行我們的計劃，為股東創造長期價值，以支持我們成為領先的罕見疾病公司的使命。

Now we will turn the call over to the operator for questions.

現在我們將把電話轉給接線員詢問問題。

(Operator Instructions) Louise Chen, Cantor.

（操作員說明）Louise Chen，Cantor。

Congratulations on all the progress this quarter.

恭喜本季取得的所有進展。

So I had a few for you.

所以我幫你準備了一些。

First question was how you think about OLPRUVA sales in the second half of '24, and then are you -- do you have any thoughts on initial thoughts on pricing for arimoclomol, if it gets approved?

第一個問題是您如何看待 OLPRUVA 在 24 年下半年的銷售情況，然後您對 arimoclomol 定價的初步想法（如果它獲得批准）有什麼想法嗎？

And then last question is just on arimoclomol, the patent protection and marketing exclusivity.

最後一個問題是關於阿莫洛莫的專利保護和行銷獨佔性。

Thanks, Louise.

謝謝，路易絲。

Why don't I start with the last question, and I'll tee up the pricing, and then I'll hand it off to Josh to talk a little bit about arimoclomol and as well as OLPRUVA.

我為什麼不從最後一個問題開始，我會確定價格，然後我會將其交給 Josh 來談談阿莫洛莫和 OLPRUVA。

In regards to the patent protection and marketing exclusivity for arimoclomol, we rely on orphan drug exclusivity for up to seven years.

關於阿莫洛莫的專利保護和行銷獨佔權，我們依賴孤兒藥獨佔權長達七年。

And then in regards to pricing for arimoclomol, it's a little early for us to be talking about pricing as we're just now in the labeling discussions.

然後，關於阿莫洛莫的定價，我們現在談論定價還為時過早，因為我們剛剛正在進行標籤討論。

But I think it's -- I want to make it clear to everybody that it is our goal to make arimoclomol as widely available as possible as we can.

但我認為，我想向所有人明確表示，我們的目標是盡可能廣泛地提供阿莫洛莫。

And with that, I'll hand it off to Josh to talk a little bit about what we've been doing around understanding the market for arimoclomol around what we could do for pricing as well as OLPRUVA sales in the second half of 2024.

接下來，我將把它交給 Josh，談談我們在了解阿莫洛莫市場方面所做的一些工作，以及我們在定價以及 2024 年下半年 OLPRUVA 銷售方面可以做的事情。

Josh?

喬許？

Yes.

是的。

With regards to arimoclomol pricing, we have conducted pretty extensive market research with payers, and we've gone out and we've tested the clinical profile and really pressured them around responding to the value proposition and the clinical value of arimoclomol for patients with NPC.

關於阿莫洛莫的定價，我們與付款人進行了相當廣泛的市場研究，我們已經出去測試了臨床概況，並真正向他們施加壓力，要求他們回應阿莫洛莫對鼻咽癌患者的價值主張和臨床價值。

I have to say that it's widely been viewed as what could potentially be foundational therapy, if approved by payers.

我不得不說，如果得到付款人的批准，它被廣泛視為可能成為基礎療法。

But of course, the final label will ultimately influence the final price and how payers view that.

但當然，最終的標籤最終將影響最終價格以及付款人的看法。

But we're working very closely with them, and we're putting in place patient services and other resources to make sure that arimoclomol will be as widely available to patients as possible.

但我們正在與他們密切合作，我們正在落實患者服務和其他資源，以確保盡可能廣泛地為患者提供阿莫洛莫。

And then with regards to OLPRUVA in the second half.

然後是下半場的 OLPRUVA。

And I'd like to just remind everyone that the first half was really built or was really focused on building awareness where there was very little awareness within the prescribing community for OLPRUVA.

我想提醒大家的是，上半年真正建立或真正專注於提高意識，而處方界對 OLPRUVA 的認識很少。

We're very happy with the progress the team made in terms of getting in front of prescribers and payers and really building that awareness across that prescribing community, and we have more than 75% covered lives for patients with UCD.

我們對團隊在處方者和付款人面前的進展感到非常高興，並真正在整個處方社區中建立了這種意識，並且我們為 UCD 患者覆蓋了超過 75% 的生命。

We've also put in place some other resources and tactics, including a free trial program and a demonstration program to be able to allow the physicians and prescribers to see how OLPRUVA works.

我們還部署了一些其他資源和策略，包括免費試用計劃和演示計劃，以便能夠讓醫生和處方者了解 OLPRUVA 的工作原理。

And we've made a change to our specialty pharmacy.

我們對我們的專業藥房進行了更改。

All of this, I think, bodes well for the second half.

我認為，所有這些都是下半年的好兆頭。

And as we really begin to focus on building more awareness within the patient community.

當我們真正開始專注於在患者群體中提高意識。

We're looking forward to seeing increased enrollments.

我們期待看到入學人數的增加。

Tim Lugo, William Blair.

提姆·盧戈，威廉·布萊爾。

Congratulations on all the progress, obviously, a transitional quarter.

恭喜所有的進展，顯然，這是一個過渡季度。

I guess after the AdCom, it seems like combination use with miglustat is going to be maybe occurring a little bit more than I maybe had expected.

我想在 AdCom 之後，與 miglustat 的聯合使用似乎會比我預期的要多一些。

I'd just love to hear your thoughts around combination use in the real world. once approved?

我很想聽聽您對現實世界中組合使用的想法。一旦批准？

And how does that fit into your pricing thoughts?

這如何符合您的定價想法？

And then maybe also some initial kind of feedback you've heard from the patient activity groups post AdCom?

然後也許還有一些您從 AdCom 後患者活動小組聽到的初步回饋？

Thanks, Tim.

謝謝，蒂姆。

I think -- let me handle your first question, and then I might actually ask Josh to talk a little bit about the advocacy engagement and/or Adrian.

我想——讓我來處理你的第一個問題，然後我可能會請喬許談談倡導參與和/或阿德里安。

I think one of the things that's really important is you get what you study, when it comes to labeling discussion, and we studied arimoclomol versus placebo with underlying routine care.

我認為真正重要的事情之一是你得到你所研究的內容，當談到標籤討論時，我們研究了阿莫洛莫與安慰劑的基礎常規護理。

And those patients could have been stratified to miglustat or some other kind of primary treatment that the patient had a routine care, I think, was standard.

這些患者可能會被分層接受米格司他或其他某種主要治療，我認為患者接受的常規護理是標準的。

And our discussions and the AdCom, we -- and I think now as we're driving into our labeling discussions, you usually get what you studied.

我們和 AdCom 的討論，我認為，現在當我們深入標籤討論時，您通常會得到您所研究的內容。

And for us, that's arimoclomol versus placebo.

對我們來說，這就是阿莫洛莫與安慰劑的對比。

That's the outcome that we're looking for.

這就是我們正在尋找的結果。

So I can't talk more about the combination therapy at this point in the game because we're in the process of round 1.

所以我現在不能多談論遊戲中的聯合治療，因為我們正處於第一輪的過程中。

But I do think that there's precedent around study -- you get what you study.

但我確實認為學習是有先例的——你學什麼就會得到什麼。

In regards to the initial feedback from the patient advocacy organizations, let me ask Josh to talk a little bit about where we're seeing some excitement from the advocacy organizations today.

關於患者倡導組織的初步回饋，讓我請喬許談談我們今天在倡導組織中看到的一些令人興奮的地方。

Yes.

是的。

I think if anyone tuned into the advisory committee meeting 10 days or so ago, you would have seen overwhelming support for arimoclomol from prescribers, caregivers and patients.

我認為，如果有人在 10 天左右之前收聽諮詢委員會會議，您會看到處方醫生、護理人員和患者對阿莫洛莫的壓倒性支持。

And that really is a reflection of the benefits that many patients have received from arimoclomol through our Expanded Access program and open-label program.

這確實反映了許多患者透過我們的擴展獲取計劃和開放標籤計劃從阿莫洛莫中獲得的益處。

And as we move forward, we expect that support to really continue.

隨著我們的前進，我們期望這種支持能夠真正持續下去。

I can also say that as we've gone out and we've talked with payers and physicians and done some market research and tested the profile many of them really see arimoclomol as the first approved drug for Niemann Pick really is the foundation for therapy for these patients.

我還可以說，當我們出去時，我們與付款人和醫生進行了交談，做了一些市場研究並測試了概況，他們中的許多人確實認為阿莫洛莫是尼曼皮克第一個批准的藥物，確實是治療的基礎這些病人。

Operator?

操作員？

(Operator Instructions) Oren Livnat, H.C. Wainwright.

（操作員說明）Oren Livnat, H.C.溫賴特。

Let me add my congrats to the outcome of the AdCom.

讓我對 AdCom 的成果表示祝賀。

Just so I'm clear, you mentioned you've entered the first round of labeling comments with the discussions with the FDA.

我很清楚，您提到您已進入第一輪標籤評論並與 FDA 進行討論。

Can you say if you've actually had any explicit additional information requests or anything new you've had to provide in this process?

您能否說一下您是否確實有任何明確的附加資訊請求或在此過程中必須提供的任何新內容？

And I have follow-ups.

我還有後續行動。

So the answer to your question is, we have gotten our first round of labeling negotiations.

所以你問題的答案是，我們已經進行了第一輪標籤談判。

And I think your second question was in regard into any specific information requests.

我認為你的第二個問題是關於任何具體的資訊請求。

We have received additional information requests, as you can imagine throughout this process as you continue to get them.

我們收到了額外的資訊請求，就像您在整個過程中不斷收到的資訊請求一樣。

In regards to new data to be provided, we have not had any request to add new data more so than what has been shared specifically with the advisory committee.

關於要提供的新數據，除了專門與諮詢委員會共享的數據外，我們沒有收到任何添加新數據的請求。

All right

好的

--

--

Sorry, with the FDA, not the advisory committee.

抱歉，是 FDA，不是諮詢委員會。

With the FDA.

與FDA。

Okay.

好的。

Perfect.

完美的。

Assuming you're approved in late September, do you have any expectations for timing for product availability, given the significant overlap with OLPRUVA.

假設您在 9 月下旬獲得批准，考慮到與 OLPRUVA 的重大重疊，您對產品上市時間有任何期望嗎？

I assume that will be relatively quickly, but I'm curious if you need to set up any different reimbursement support infrastructure given this is a new indication unlike UCD.

我認為這會相對較快，但我很好奇您是否需要建立任何不同的報銷支援基礎設施，因為這是與 UCD 不同的新適應症。

And in the NPC community and centers of excellence, I guess I'm going already asked about the feedback post AdCom, but I'm just curious if there's any -- you didn't sense that there's any particular preparation or expectations there post AdCom?

在 NPC 社區和卓越中心，我想我已經詢問了 AdCom 後的反饋，但我只是好奇是否有任何反饋 - 你沒有感覺到 AdCom 後有任何特殊的準備或期望？

Are you hearing about warehousing of patients or any proactive outreach maybe to patients and families in anticipation of potential approval?

您是否聽說過有關患者入庫的消息，或者是否有任何針對患者和家屬的主動外展活動，以期獲得潛在的批准？

Let me start by talking a little bit about the potential for our PDUFA at the end of September.

首先讓我談談 9 月底 PDUFA 的潛力。

We are on track in order to be able to have product in the channel within the customary timeframe, let's call it, 8 to 12 weeks, maybe even smaller than that 8 to 10 weeks post launch, which is fairly standard.

我們正在走上正軌，以便能夠在通常的時間範圍內（我們稱之為 8 到 12 週，甚至可能比發布後的 8 到 10 週更短）在渠道中提供產品，這是相當標準的。

Let me turn it over to Josh to talk a little bit about new infrastructure and/or the needs in the NPC community post the advisory committee.

讓我把它交給 Josh，讓他談談新的基礎設施和/或諮詢委員會後 NPC 社區的需求。

Oren, as a reminder, we built the commercial team really focused not just on OLPRUVA, but in anticipation of an approval and launch of arimoclomol.

Oren，提醒一下，我們建立的商業團隊不僅專注於 OLPRUVA，而且還期待阿莫洛莫的批准和上市。

So it is really rightsized to be able to optimize both products.

因此，能夠優化這兩種產品確實是合理的。

And we also have the benefit of having a team out there now talking to prescribers about UCD.

我們還有一個好處是，現在有一個團隊正在與處方醫生討論 UCD。

Oftentimes, these are the same physicians who are seeing Niemann Pick patients.

通常，這些醫生也會為尼曼皮克病人看診。

And so our team is out there profiling some of these physicians, understanding where the patients are.

因此，我們的團隊正在對其中一些醫生進行分析，以了解患者所在的位置。

Our team on the market access side is having conversations with payers on the clinical differentiation of arimoclomol and the need to treat.

我們市場准入方面的團隊正在與付款人就阿莫洛莫的臨床差異和治療需求進行對話。

So the team is in place.

所以團隊就位了。

We feel quite confident that it's rightsized for both products.

我們非常有信心它適合這兩種產品。

And we will be well positioned to begin commercialization as soon as drug is in the channel.

一旦藥物進入管道，我們就可以立即開始商業化。

In terms of where these patients are.

就這些患者所在的位置而言。

We continue to work very closely with the investigators in our expanded access program.

我們將繼續在擴大訪問計劃中與調查人員密切合作。

As a reminder, we've got more than 70 patients here in the US who are in that program as well as other patients were being seen by those same investigators who are eagerly awaiting the approval of arimoclomol.

提醒一下，我們在美國有 70 多名患者參與了該項目，而其他患者也正在接受同樣的研究人員的檢查，他們正在熱切地等待阿莫洛莫的批准。

And I guess just lastly, you segued into the EAP patients, is there any preparation you can do -- separate or in addition to standard market pre-commercial activities with regard to this EAP population, in particular, since you know the patients and have relationships with them directly to some extent.

我想最後，您談到了 EAP 患者，您是否可以做任何準備 - 單獨或除了針對該 EAP 人群的標準市場預商業活動之外，特別是，因為您了解患者並且已經在某種程度上與他們有直接的關係。

Is there anything you can do to expedite their transition to commercial products when this product is available as opposed to maybe what the process would be with a new patient?

當產品上市時，您可以做些什麼來加快他們向商業產品的過渡，而不是新患者的流程？

So thanks, Oren.

謝謝，奧倫。

A couple of things.

有幾件事。

One is, I think, number one, we should get on the table here so that we're going to continue to support these patients in the EAP program until we've got commercial supply and access for patients to be able to transition.

我認為，第一，我們應該在這裡討論，以便我們將繼續在 EAP 計劃中支持這些患者，直到我們獲得商業供應和患者能夠過渡的機會。

Answering your second part of the question, and we are working directly with investigators, not necessarily with patients directly because recall, the EAP program is still collecting data, and we don't have to direct to hands on with patients.

回答你問題的第二部分，我們正在直接與研究人員合作，不一定直接與患者合作，因為回想起來，EAP 計畫仍在收集數據，我們不必直接與患者接觸。

The next thing you asked about is in regard to, can you set up your EAP program to allow for more seamless transition?

您問的下一件事是，您能否設定 EAP 計劃以實現更無縫的過渡？

I think I want to make it clear.

我想我想把話說清楚。

It's our goal to be able to transition our EAP patients to commercial supply within the first year post launch, and that will allow us to be able to move on to other areas that we can collect data and real-world data and other things that can help patients move forward.

我們的目標是能夠在推出後的第一年內將我們的 EAP 患者轉變為商業供應，這將使我們能夠繼續前進到其他領域，我們可以收集數據和真實世界數據以及其他可以幫助患者前進。

So there are things that we can do, and we're working with those sites and investigators.

所以我們可以做一些事情，我們正在與這些網站和調查人員合作。

One of them is around supply.

其中之一是圍繞供應。

We may give a 90-day supply, then it may go to a 60-day supply.

我們可能會提供 90 天的供應量，然後可能會提供 60 天的供應量。

But we're doing this in a very cautious way to ensure that every patient continues to have access to the EAP and supply.

但我們正在以非常謹慎的方式這樣做，以確保每位患者繼續獲得 EAP 和供應。

I can't be more emphatic about the fact that we are here to make sure that we're taking care of these patients in the EAP until they can get transitioned.

我必須強調這樣一個事實：我們來這裡是為了確保我們在 EAP 中照顧這些患者，直到他們能夠過渡。

This does conclude the Q&A portion of today's call.

今天電話會議的問答部分到此結束。

I would now like to turn the call back over to Neil McFarlane for any additional or closing remarks.

我現在想將電話轉回尼爾·麥克法蘭，請他發表補充或結束語。

Thank you.

謝謝。

We continue to make solid advances towards achieving our mission of building a leading patient-focused rare disease therapeutics company.

我們繼續紮實推進，以實現我們的使命，建立一家領先的以患者為中心的罕見疾病治療公司。

As we look to our upcoming catalysts in the second half of 2024, our priorities are clear. and we look forward to updating you in the future.

當我們展望 2024 年下半年即將推出的催化劑時，我們的優先事項很明確。我們期待將來為您提供最新消息。

Thank you for joining us today.

感謝您今天加入我們。

This does conclude today's program.

今天的節目到此結束。

Thank you for your participation, and you may now disconnect.

感謝您的參與，您現在可以斷開連接。