プロタリックス・バイオセラピューティクス（PLX）2026年第2四半期決算説明会：エルファブリオが売上成長を牽引

主要なポイント

• プロタリックス・バイオセラピューティクス（Protalix BioTherapeutics）が発表した2026年第2四半期の売上高は1,990万ドルとなり、キエジー（Chiesi）向けの売上増を主因に、前年同期の1,570万ドルから26.8%増加しました。
• 第2四半期の純利益は前年同期の16万4,000ドル（希薄化後1株あたり0.00ドル）から380万ドル（基本的および希薄化後1株あたり0.05ドル）に増加しました。
• 上半期の売上高は、エルファブリオ（Elfabrio）の成長と2026年第1四半期に計上されたキエジーからのマイルストーン収益2,500万ドルに支えられ、前年同期の2,580万ドルに対して5,360万ドルに達しました。
• 2026年6月30日時点の現金、現金同等物および短期銀行預金は合計4,070万ドルでした。プロタリックスには未償還の負債やワラントはありません。
• 経営陣は2026年通期の売上高見通しを再確認した一方で、パートナーからの注文や出荷のタイミングによって四半期ごとの変動が生じる可能性があると警告しました。
• 「PRX-115」のRELEASE第2相試験は計画通り進行しています。経営陣は2026年末までに被験者登録が完了し、2027年後半にトップライン結果が得られると見込んでいます。

主要財務データ

2026年上半期の売上高は5,360万ドルで、2025年上半期の2,580万ドルから増加しました。製品売上高は前年同期の2,540万ドルに対し、合計2,720万ドルとなりました。

事業・業績の動向

エルファブリオは、プロタリックスとキエジーとの提携を通じて主要な商業的成長ドライバーであり続けました。経営陣によると、第2四半期の購入実績には基礎的な事業成長とキエジーによる在庫管理の両方が反映されています。そのため同社は、四半期単独の業績よりも通期の業績を見る方がより有益であると考えています。

経営陣は、欧州でエルファブリオが4週間に1回の投与スケジュールで最近承認されたことが、商業的ポジショニングを強化すると述べています。同社は、世界のファブリー病市場が2031年までに約32億ドル規模になると予測されていることに触れ、エルファブリオがその市場の15%〜20%を獲得できる可能性があると信じていると述べました。

コントロール不全の痛風を対象としたプロタリックスの持続性ウリカーゼ候補化合物「PRX-115」は、RELEASE第2相複数回投与試験において順調に進展しています。経営陣は、計画されていた治験施設のほぼすべてが開始されており、残る施設は最大でも1カ所程度であると示しました。

経営陣の見通し（ガイダンス）

プロタリックスは2026年通期の売上高ガイダンスを維持しました（ただし、決算説明会では具体的なガイダンスレンジは明かされませんでした）。経営陣は、堅調な売上動向とエルファブリオの継続的な拡大が見通しを後押ししていると述べています。

同社は、順調な進捗を前提として、RELEASE試験の被験者登録が2026年末までに完了し、2027年後半にトップライン結果が出ると見込んでいます。

RELEASE試験の進展や追加の非臨床・臨床プログラムの推進に伴い、研究開発費は増加する見込みです。また、経営陣は新研究開発法に基づく助成金が今後も継続的に利用可能であると見込んでいます。

リスクと注目点

• パートナーの注文時期、出荷スケジュール、在庫管理の影響により、売上高が四半期ごとに変動する可能性があります。
• PRX-115の第3相用法の選定は、有効性、免疫原性、抗薬物抗体、点滴静注関連反応など、第2相試験の全結果に基づいて決定されます。
• 経営陣は、推奨される用法としてメトトレキサート非併用での4週間隔投与を優先するか、メトトレキサート併用での8週間隔投与を優先するかを判断するのは時期尚早だと述べました。
• 2026年末を目指す被験者登録の完了目標は、試験の進捗状況に左右されます。
• 臨床活動の活発化に伴い、時間の経過とともに研究開発費が増加することが予想されます。

アナリスト質疑応答のハイライト

アナリストらは、プロタリックスが第3相試験に向けたPRX-115の用法をどのように選定するかに注目しました。経営陣は、決定には奏効率や投与頻度以上の要素が考慮されると述べました。免疫原性、抗薬物抗体、点滴静注関連反応、さらにはメトトレキサートの投与を回避できるかどうかも判断に影響を与えます。

36ミリグラムのPRX-115固定用量に関して、経営陣は第1相の薬物動態および薬力学データに基づいて決定されたと述べました。進行中の複数回投与第2相試験により、各治療群における抗薬物抗体反応のより明確な像が得られると期待されています。

エルファブリオの購入パターンについて、経営陣は第2四半期の好調な受注水準について、各市場におけるキエジーの在庫計画や製造上の要件が一因であると説明しました。四半期ごとの受注は単独で評価すべきではないと改めて強調しました。

また、経営陣はコントロール不全の痛風市場における既存のトップ企業の売上高が2025年に13億ドルに達し、16億ドルに向けて推移していることに言及し、これがPRX-115にとって好材料であると説明しました。

決算説明会文字起こし全文

決算説明会の完全なトランスクリプト

経営陣による説明

Operator

Good morning, ladies and gentlemen, and welcome to the Protalix BioTherapeutics Second Quarter 2026 Financial and Business Results Conference Call. As a reminder, this conference is being recorded. I will now turn the conference over to your host, Mr. Mike Moyer of LifeSci Advisors, Investor Relations for Protalix. Please go ahead.

Mike Moyer

Thank you, operator, and welcome to the Protalix BioTherapeutics Q2 2026 Financial Results and Business Update Conference Call. With me today are Dror Bashan, President and CEO; and Gilad Mamlok, Senior Vice President and Chief Financial Officer. Press release announcing the financial results and corporate updates was issued this morning and is available now on the Protalix website. Please take a moment to read the disclaimer about forward-looking statements in the press release. The earnings release and this teleconference include forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual results to differ materially from the statements made. Factors that could cause actual results to differ are described in the disclaimer and in Protalix' filings with the U.S. Securities and Exchange Commission. I will now turn the call over to Mr. Bashan. Dror?

Dror Bashan

Thank you, Mike, and thank you, everyone, for joining this morning. Our results today reflect the strength of our profitable commercial partnerships, and we remain confident in our outlook for this year. We entered the second half of 2026 with Elfabrio sales through Chiesi continuing to drive growth of our business. Gilad will walk through the financial details, but the key driver in the first half was continued growth in Elfabrio's revenues, together with the previously reported $25 million Chiesi milestone payment recognized in the first quarter of this year. Elfabrio sales through Chiesi continue to drive revenues from selling, and this performance reflects further global penetration of Elfabrio and continued growth. This continued growth keeps us on track with our full year 2026 revenue guidance with the global Fabry market projected to reach approximately $3.2 billion by 2031, Elfabrio is positioned to capture 15% to 20% of this market, supported by our partnership with Chiesi, which is strengthened by the recent approval of the once every 4 weeks dosing regimen in Europe. We believe our revenue mix, particularly the continued expansion of Elfabrio position us well for sustained long-term value creation and [ profitability ].

On the clinical side, PRX-115 continues to advance as planned. PRX-115 is designed as a long-acting differentiated uricase, and we believe it has the potential to meaningfully improve quality of life for patients with uncontrolled gout, which could be a significant inflection point and revenue driver for Protalix. There is a significant unmet need in this population, and we expect top line results from our RELEASE study in the second half of 2027. Beyond PRX-115, our strategy remains centered on rare renal diseases, where we believe our capabilities and platform offer a clear advantage, and we remain focused on execution across our partnerships and pipeline, and we believe our business model limits downside risks while preserving meaningful upside as we advance our clinical programs. With that, I will turn the call over to Gilad for a detailed review of our financial results and outlook. Gilad, please.

Gilad Mamlok

Thank you, Dror. For the second quarter of 2026, total revenues were $19.9 million compared to $15.7 million in the second quarter of 2025. For the first half of 2026, total revenues were $53.6 million compared to $25.8 million for the first half of 2025. For the second quarter of 2026, revenues from selling goods were $19.8 million compared to $15.4 million in the second quarter of 2025, an increase of $4.4 million. For the first half of 2026, revenues from selling goods were $27.2 million compared to $25.4 million for the first half of 2025, an increase of $1.8 million. The increase was driven mainly by higher sales to Chiesi. For the remainder of the results, I will report only on the second quarter, and you can refer to this morning's press release for additional year-to-date. Cost of revenues were $7.8 million compared to $5.9 million in the same period in 2025, an increase of $1.9 million. The increase was mainly attributable to higher sales volumes to Chiesi [indiscernible] and partially offset by lower sales to Pfizer. R&D expenses were $4.4 million, down from $6 million in the prior year period, a decrease of $1.6 million. The decrease was mainly driven by a $2.1 million recorded under the new R&D law as a reduction of R&D expenses. As of 2026, this grant is available for us under the R&D law on an ongoing basis. We expect to continue to incur expenses as the RELEASE study progresses and additional preclinical and clinical programs. G&A expenses were $3.1 million, up $0.5 million from the prior year period, largely attributable to higher salary and related expenses. Finance income net was $0.2 million compared to financial expenses net of $0.5 million in the second quarter of 2025. The change was mainly due to exchange rate fluctuations. Taxes on income were $1.1 million compared to $0.5 million in the second quarter of 2025, an increase of $0.6 million. This increase resulted mainly from taxes on income derived from global intangible low tax income or GILTI resulting from limitations under IRC Section 174. Net income for the quarter was $3.8 million or $0.05 per share basic and diluted compared to net income of $164,000 or $0 per share basic and diluted in the second quarter of 2025. Turning to the balance sheet, cash, cash equivalents and short-term bank deposits totaled $40.7 million as of June 30, 2026. We have no outstanding debt or warrants, providing us with substantial financial flexibility to support our continued pipeline advancements. As we have noted in prior quarters, our revenues can vary from quarter-to-quarter based on the timing of shipments and orders from our partners. We believe it is more useful to evaluate our business on a full year basis, and we remain confident in our full year 2026 guidance. With that, I will turn the call back over to Dror.

Dror Bashan

Thank you, Gilad. In closing, positive revenue trends keeps us firmly on track to meet our full year 2026 guidance. We have a strong cash position to maintain our operations and advance our clinical and preclinical assets. We are confident in the momentum behind our business and about the opportunities ahead. Now I will ask the operator to open the line for questions.

Operator

[Operator Instructions] And our first question -- our first question will come from Raghuram Selvaraju with H.C. Wainwright.

質疑応答

Unknown Analyst

This is [ Yuan Zhi ] sitting in for Ram Selvaraju. I have 2. The first is, so your presentation highlights E4W without methotrexate and the E8W with MTX as the 2 differentiated profiles within RELEASE. What totality of evidence framework will determine which regimen advances to Phase 3? And could a modest month 6 responder rate difference be outweighed by eliminating MTX or extending dosing to E8W?

Dror Bashan

I repeat the question? Actually, both. I mean if you manage to 1 of the 5 [indiscernible] without methotrexate [indiscernible], which can be a huge competitive edge, but also a lower frequency of using the drug is also a big advantage, and we potentially can have both.

Unknown Analyst

Right. Okay. So yes, I'll repeat the question. It's -- my question is what totality of evidence will determine which regimen advances to Phase 3? And could a modest month 6 responder rate difference be outweighed by eliminating the methotrexate or extending dosing to every 8 weeks?

Dror Bashan

We are running now the multiple dose study on the Phase 2. We have to see the outcome. And by the outcome, we can decide with which regimens we continue. It's difficult to tell you today. I mean, under what we have from the mechanism of action and the data from the Phase 1 and whatever we understand about the molecule and the PEGylation of the molecule and the outcomes we have so far, we think that these 4 arms, I'm not speaking about the placebo could be met, which one will be met better. We have to consider there is an immunogenicity aspect, there is an IRR aspect. So it's not just the frequency. But let's say, under the assumption immunogenicity is good or low or whatever the definition is and with a pretty low IRR. And then it's only about the frequency and/or with or without methotrexate. We have to see.

Unknown Analyst

Got it. Understood. The other question I have is relating to the 10-K, which reports approximately about 50% of Phase 1 subjects who have developed ADAs and that comes with lower incidence at higher doses. My question is, how does the combined PK, PD and immunogenicity data support selecting that fixed 36-milligram dose? And what ADA profile and RELEASE would support every 4 weeks dosing without methotrexate?

Dror Bashan

So we will -- in Phase 2, we will have the, I would say, the ADA against the enzyme. So we have, I think, a clearer picture of the different ADAs and how -- and what are the outcomes, of course, the different arms. So then we'll see where we are. Don't forget the Phase 2 is the multiple dose. Phase 1 was a single ascending dose chose 36-milligram according to one, I would say, the PK, PD, if I may say, of course, and also under the assumption that this specific, I would say, regimen could be the highest or the more, I would say, convenient and by the way, for the patients, if I may say, both once in 4 weeks without methotrexate and the once in 8 weeks with methotrexate, which call them like the highest outcomes if possible.

Operator

Our next question will come from John Vandermosten with Zacks SCR.

John Vandermosten

Great. Nice to hear from you guys. And congratulations on Elfabrio's patent term extension and the approval in South Korea. Things are going pretty good for that product. I want to start out with a question on just the trend in purchases of Elfabrio. I'm wondering, is there any benefit to having lumpy purchases because we had a nice increase this quarter. And I'm wondering, is that due perhaps to the new approvals in certain geographies? Or is there some other reason in terms of like product runs or some other reason why it makes sense to have lumpy rather than steady purchases of Elfabrio?

Gilad Mamlok

John, thank you. As we always say, one quarter, we prefer to look at the picture always year-to-date. We did have a nice quarter from Chiesi, which reflects also their growth of the business, but mainly reflects their inventory management.

John Vandermosten

Okay. So there's no -- I mean, there's no rationale behind why they wouldn't make it smooth rather than lumpy.

Dror Bashan

This is not a fair definition, I would say. They operate with a lot of sense and logic, of course, and they are responsible for what they are doing. They have their own -- they see the demand. They know how much they sit on certain amount of inventories globally or continent. And of course, their manufacturing plant vis-a-vis other programs that are marketing with a big company. So all in all, this is how it was decided and applied. And of course, to make sure we sit on enough stocks, both [ DS and DP ].

John Vandermosten

Okay. And jumping to 115, I believe you've opened up some new sites or at least on ClinicalTrials.gov, it seems like there are a number of sites listed there compared to previous -- early in the year. How is that going in terms of site activation? And kind of where are you right now? How much can you tell me? And then how many more do you think might be opening in the future so far in 2026?

Dror Bashan

We have actually maybe one site is missing. That's it. And we -- at present, we expect to finalize enrollment within the next 5 months, I mean by year-end as we planned. We'll have to see if things indeed will go this way. So far, so good, knock on wood. That's it. I think overall, except one site that maybe is missing, we are actually signed on whatever we plan to course.

John Vandermosten

And last question is on kind of a bigger picture question on Amgen. They raised price pretty significantly this year, first quarter and second quarter saw 20-plus percent price increases on Krystexxa. And I'm wondering what does that tell you about the market? Does it make it look more attractive? I'm not sure of the details. Perhaps it was a payer mix or something like that. I didn't get into that. But I'm just wondering if you have any thoughts on what that dramatic price increase means for the space.

Dror Bashan

I don't have any insights on that. And I don't know, you get it from what they say or you get it from calculating sales to estimate the number of patients or...

John Vandermosten

It was in the press release or second quarter press release, they said 23% increase in and pricing, even the volumes were down. And they didn't go into it, but I mean, my thought was there's -- the market will accept a higher price, which may make it look more attractive for you guys for 115. So I just wanted to see if that had any impact on your thoughts for this product and the pathway forward.

Gilad Mamlok

The revenues keep growing. It was $1.3 billion in 2025. We see now the trend going to $1.6 billion. So it's very encouraging for 115 as well, as we said.

Operator

And this now concludes our question-and-answer session. I would like to turn the floor back over to Dror Bashan for closing comments.

Dror Bashan

Thank you. So I just like to thank everybody that joined our call, and we look forward to report on our Q3 results as well. I think overall, we have a good strong cash position to maintain our operations and advance our clinical and preclinical assets. And again, we are confident in the momentum behind our business and about the opportunities ahead. So thank you very much.

Operator

Ladies and gentlemen, thank you for your participation. This does conclude today's teleconference. You may disconnect your lines, and have a wonderful day.