ジェロン（GERN）2026年第3四半期決算説明会：RYTELOの成長と費用ガイダンスの引き下げ

ジェロン（Geron Corporation、NASDAQ: GERN）は、2026年第3四半期の決算でRYTELO（ライテロ）の成長が持続したと発表する一方、通期の営業費用見通しを引き下げました。また、独立委員会が主要評価項目の達成は困難と判断したことを受け、第III相IMPACT MF試験の中止を決定しました。

主要なポイント

• 2026年第3四半期の製品売上高（純額）は6,010万ドルに達し、前年同期の4,720万ドルから前年同期比27%増、前四半期比5%増となりました。
• RYTELOの需要は前四半期比で10%増加し、4四半期連続で需要増を記録しました。
• 処方施設数は当四半期中に8%増加し、RYTELOの採用実績は発売以来1,700施設以上に拡大しました。この成長は主に地域医療機関が牽引しています。
• ジェロンは、2026年のRYTELO製品売上高（純額）ガイダンスである2億2,000万〜2億4,000万ドルを再確認し、結果はそのレンジの中限から上限になると見込んでいます。
• 通期の営業費用ガイダンスは、従来の2億3,000万〜2億4,000万ドルから2億2,000万〜2億3,000万ドルへ引き下げられました。
• 第III相IMPACT MF試験は、全生存期間の主要評価項目達成が困難と判断されたため中止されました。経営陣によると、イメテルスタットの既知の安全性プロファイルを変更するような新たな安全性シグナルは確認されませんでした。

主要財務データ

経営陣はまた、年初からの累計営業費用が前年同期比で9%減少したと述べました。

事業および業績の動向

RYTELOの商業的な勢いは、ジェロンの2026年第3四半期決算説明会における中心的な焦点であり続けました。需要は前四半期比で10%増加し、処方施設数は8%増加しました。経営陣はこの拡大の多くを地域医療機関（がん専門クリニック）によるものとし、これを長期的な普及の鍵となる源泉と位置付けています。

ジェロンは、二次治療の低リスク骨髄異形成症候群におけるRYTELOの臨床的有効性を強調し、投与量や血球減少症の管理について血液がん専門医への啓発活動を行うことで、対面・非対面でのターゲットを絞った働きかけを通じたさらなる普及を支援する方針です。

同社はまた、欧州市場での段階的な商業展開を開始しました。ドイツでは2026年末までに販売が開始される見込みで、フランスが2027年に続く予定です。経営陣は、この段階的モデルにより価格統制を維持し、初期のインフラ投資を制限し、潜在的なパートナーシップに対する柔軟性を保持することを目的としていると述べています。

ジェロンは、欧州における機会がRYTELOの希少生薬（オーファン・ドラッグ）指定、単一適応症のプロファイル、および主に欧州で実施されたIMerge試験による治験責任医師の経験によって支えられていると指摘しました。また、経営陣は、低メチル化製剤が米国ほど欧州に定着していないことも挙げました。

経営陣の見通し（ガイダンス）

ジェロンは、2026年のRYTELO製品売上高（純額）ガイダンス（2億2,000万〜2億4,000万ドル）を維持しました。経営陣は前四半期比での継続的な成長に基づき、売上高がレンジの中限から上限で着地すると見込んでいます。

同社は、2026年の通期営業費用合計ガイダンスを2億2,000万〜2億3,000万ドルに引き下げました（従来の見通しは2億3,000万〜2億4,000万ドル）。この改定は、RYTELO、製造活動、市販後の臨床コミットメントへの投資を維持しつつ、人件費の削減と継続的な事業規律を反映したものです。

2026年の残りの期間について、ジェロンはGross-to-Net控除率が20%台前半から半ばの範囲になると見込んでいます。経営陣は、試験の終了作業があるため、2026年中に骨髄繊維症プログラム関連の支出が大幅に削減されるとは予想していません。

2026年以降を見据え、経営陣は欧州戦略の進展や市販後臨床コミットメントの進展に伴い、費用が増加する可能性があると述べました。

リスクと注視すべきポイント

独立データモニタリング委員会は、JAK阻害薬治療後の再発または難治性の骨髄繊維症において、イメテルスタットが主要評価項目である全生存期間を達成する可能性が低いことから、第III相IMPACT MF試験の中止を勧告しました。

ジェロンは治験データをアンブラインドして分析し、今後の学会で結果を発表する予定です。経営陣はイメテルスタットの既知のプロファイルを変更するような安全性の所見は報告されていないとしていますが、有効性評価の不成立により、骨髄繊維症における開発機会の1つが失われることになります。

欧州での商業化も段階的に進められます。製品の提供可能性、保険償還の進捗、支出は市場ごとに異なり、同社は引き続きパートナーシップの選択肢を検討しています。

アナリスト質疑応答のハイライト

将来の支出：経営陣は資本配分における規律を維持すると述べつつも、欧州での商業化の進展や市販後試験の進捗に伴い支出が増加すると見込んでいます。IMPACT MF試験の終了費用により、2026年中に大きなコスト削減効果が得られるとは予想されていません。

RYTELOの成長軌道：経営陣は具体的な普及転換点（インフレクション・ポイント）を示しませんでした。代わりに、地域医療への浸透拡大と数千人の追加対象患者群に支えられ、前四半期比で着実な成長が続くと見込んでいます。

事業開発戦略：ジェロンは既存の米国商業組織を活用できるアセットを模索しています。経営陣は地域医療に強みを持つ血液・がん領域の製品を主なターゲットとして挙げており、ピーク時の売上高が数億ドル規模に達する可能性のあるアセットが適していると述べました。同社は自らの優先アプローチを、選別的な「ストリング・オブ・パールズ（真珠のネックレス）」戦略と表現しました。

欧州での機会：経営陣は、欧州における二次治療の対象患者数が、米国で挙げられた約8,000人と同等以上であると試算しています。ドイツが最初の市場として選ばれたのは、実行可能な保険償還パスと製品の革新性を反映できる価格設定が見込めるためです。

決算説明会の全トランスクリプト

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

Hello, and welcome to the Geron Corporation Third Quarter 2026 Earnings Call. [Operator Instructions] Please be advised that today's conference is being recorded.

I would now like to turn the call over to Dawn Schottlandt, SVP, Investor Relations and Corporate Affairs. You may begin.

Dawn Schottlandt

Good morning, everyone. Welcome to the Geron Corporation Third Quarter 2026 Earnings Conference Call. Before we begin, please note that during the course of presentation and question-and-answer session, we will be making forward-looking statements regarding future events, performance, plans, expectations and other projections, including those related to our 2026 financial guidance, our current RYTELO commercialization strategy and related opportunities in the U.S. and the EU, the therapy potential of RYTELO and other anticipated clinical and commercial events and related time lines, the sufficiency of our financial resources and other statements that are not historical fact, which, of course, involve risks and uncertainties that could cause actual events performance and results to differ materially from those contained in these forward-looking statements. Therefore, I refer you to the risks and uncertainties described in today's earnings release and other the heading Risk Factors in Geron's most recent periodic report filed with the SEC, which identify important factors that could cause actual results to differ materially from those contained in these forward-looking statements and future updates to Geron's risks and uncertainties, disclosures, including in its upcoming quarterly report on Form 10-Q.

Geron undertakes no duty or obligation to update its forward-looking statements.

Joining me on today's call are several members of Geron's management team; Harout Semerjian, Chief Executive Officer; Ahmed ElNawawi, our Chief Commercial Officer.

With that, I'll turn the call over to Harout to discuss Geron's progress in the third quarter.

Harout Semerjian

Thank you, Dawn, and good morning, everyone. We continue to execute our strategy to build a leading hematology company. We're delivering consistent retail demand growth, reinforcing our confidence in the low-risk MDS U.S. commercial opportunity and RYTELO's growth trajectory over the next several years. We also initiated a gated commercial launch in targeted European markets that gives us the optionality to scale up RYTELO's availability through partnerships.

Last week, we announced the discontinuation of the IMPACT MF trial evaluating imetelstat in relapsed and refractory myelofibrosis following a recommendation by the independent DMC. We believe this is the right course of action for patients and for Geron.

In the third quarter, we continued to see increased prescribing from community practices leading to a quarter-over-quarter demand growth of 10% and net revenue growth of 5% or $60.1 million. This represents a 27% increase in net revenue year-over-year. This performance keeps us on track to achieve the mid- to high end of our full year 2026 net revenue guidance range of $220 million to $240 million and reinforces our confidence that RYTELO has a long growth runway in the U.S.

This year, we started executing our ex-U.S. go-to-market commercial strategy, focused on addressing unmet patient needs and broadening access to RYTELO while prioritizing U.S. price integrity. Recent developments, including the exclusion of orphan drugs like RYTELO from the CMS globe model gives us the opportunity to serve more patients globally. We initiated a gated commercial launch to make RYTELO available in targeted European markets, beginning with Germany, where RYTELO is expected to be commercially available by the end of this year. In Europe, our approach enables us to retain strategic control of pricing without having to establish full infrastructure in each market or commit to a large upfront investment. This hybrid model also allows us to remain flexible and open to partnership opportunities.

To support our global reach, we recently increased our engagement and presentations at multiple international medical meetings, and we're encouraged by the interest from hematologists in key markets. We will continue to explore opportunities in other geographies where we believe we can help address the significant unmet need of low-risk MDS patients. As we continue to strengthen our operations, another area of focus for us is financial discipline where our investments are intentional and targeted. Our operating expenses were down 9% year-to-date compared to the prior year reflecting our continued focus on operational efficiency. This discipline is enabling us to update our full year 2026 total operating expense guidance range from $220 million to $230 million. With $339 million of cash on hand, we have a strong balance sheet to fund our current operations which gives us flexibility to pursue business development opportunities.

With that, I'll turn it over to ElNawawi to provide more detail on RYTELO's commercial performance and our execution.

Ahmed ElNawawi

Thank you, Harout. We are pleased to report our fourth consecutive quarter of demand growth for RYTELO, reinforcing the need for this drug and its potential long-term value as a preferred treatment and second-line low-risk MDS. In the third quarter, we achieved a 10% demand growth for RYTELO compared to the second quarter of this year, an 8% increase in prescribing accounts, predominantly from community practices expanding our footprint to more than 1,700 accounts since launch. With first and second-line patient starts on a rolling 12-month basis at 36%.

We believe that RYTELO has the potential to deliver consistent quarter-over-quarter growth for years to come, driven in large part by continued adoption in the community setting where there are still thousands of patients that can benefit from RYTELO. Our focus is on accelerating that adoption through targeted personal and nonpersonal engagements that reinforce RYTELO's differentiated clinical efficacy in second line low-risk MDS patients. while continuously educating hemoncs on dosing and management of cytopenias.

RYTELO's unique mode of action as a first-in-class telomeres inhibitor. It's broad label and position in NCCN guideline provide a strong foundation to drive durable long-term growth.

Turning to our efforts in Europe. We've taken a gated operational approach where we drive regulatory engagement and local reimbursement process to ensure RTYELO reaches the appropriate patients at a price that reflects unique value. Our model allows us to broaden RYTELO's access with a staged upfront investment while preserving flexibility for the future. We have already initiated our launch in Europe and plan to make RYTELO commercially available in Germany by the end of the year with trends to follow in 2027.

We are encouraged by the interest we're seeing across markets, supported by investigated experience with imetelstat and the significant unmet need in low-risk MDS. I now turn it over to Joe to discuss our medical and scientific engagement efforts.

Joseph Eid

Thanks, ElNawawi. Last week, we announced our decision to discontinue the Phase III IMPACT MF trial evaluating imetelstat and JAK inhibitor relapsed/refractory myelofibrosis. This decision was based on the recommendation from the independent data monitoring committee informing us that the trial was unlikely to meet its primary endpoint of overall survival. Importantly, there were no safety signals observed in the trial that changed the known safety profile of imetelstat. We are committed to unblinding the data, conducting a detailed analysis of this rich data set and sharing findings at an upcoming medical meeting that may help inform future research. We are deeply grateful to the patients and families as well as the investigators and clinical teams whose dedication made this research possible.

Turning to low-risk MDS. This quarter, we presented at leading hematology meetings, both global and regional further communicating the growing body of evidence supporting RYTELO's differentiated profile, including evidence of disease-modifying activity. Just as importantly, these meetings created meaningful opportunities to engage directly with the medical community exchange insights and deepen scientific dialogue around the evolving treatment of low-risk MDS.

At SOHO, we continue to build strong momentum through impactful data presentations, sponsored dedication and broad power engagement powered by a coordinated full team approach. Our low-risk MDS real-world evidence poster showed meaningful hemoglobin improvement with imetelstat despite short treatment duration and later line news in U.S. community oncology practice in the immediate post-launch period. This marked the second real-world evidence presentation this year adding to the growing body of evidence that reinforces findings from our Phase III IMERSE trial and the safety, efficacy and tolerability profile of RYTELO.

Additional data presented at the 88th Annual Meeting of the Japanese Society of Hematology and the 2026 Annual Meeting of the German, Austrian and Swiss Societies for hematology and medical oncology further highlighted the durable clinical outcomes observed within imetelstat and expanded the growing body of evidence supporting its differentiated profile. Across the presentations, Phase III IMerge findings and real-world experience reinforce the durability and clinical relevance of treatment outcomes, including findings suggesting that treatment emergent cytopenia may reflect an on-target effect associated with meaningful clinical outcomes, such as hemoglobin improvement and transfusion independence in low-risk MDS.

Importantly, the presentations also demonstrated growing scientific interest and investigator experience with imetelstat outside the U.S. as we continue to deepen engagement with the global hematology community. Beyond congress presentations, we continue to strengthen the scientific foundation of RYTELO through 2 manuscripts published in peer-reviewed journals, further expanding the body of evidence supporting imetelstat's role and low-risk MDS and its potential to address significant unmet need.

Looking ahead, the 68th ASH Annual Meeting in December will provide another opportunity to build on this body of evidence with new data across low-risk MDS and other hematological diseases that will further inform our understanding of RYTELO's clinical potential. Our 6 accepted abstracts at ASH reinforce the clinical and biological evidence of RYTELO and we look forward to sharing additional details later today. Our progress has been recognized beyond the medical community. We are honored to have been nominated for the pre Gallium USA Award for best product for orphan or rare diseases. This recognition represents decades of pioneering work in telomerase inhibition and most importantly, a sustained commitment to addressing significant unmet need for people living with low-risk MDS. It is a meaningful acknowledgment of the science behind RYTELO and the potential to translate that science into better outcomes for patients.

I'll now hand it over to Michelle to walk through the financials.

Michelle Robertson

Thank you, Joe, and good morning, everyone. For more detailed results from the third quarter, please refer to the press release we issued this morning, which is available on our website. Our third quarter financial results highlight the progress we are making on our commercial strategy and operational excellence. We are in a strong financial position with a balance sheet that enables targeted and disciplined investments into further growth opportunities, including business development.

In the third quarter, total net product revenue for the 3 months ended September 30, 2026, was $60.1 million compared to $47.2 million in Q3 of 2025. Gross to net deductions decreased to 19.3% for the 3 months ended September 30, 2026 compared to 21.6% for the same period in 2025. For the remainder of 2026, we continue to expect gross to net to be in the low to mid-20s. Research and development expenses for the 3 months ended September 30, 2026, were $12.7 million compared to $21.1 million in expenses for the same period in 2025. The decrease in research and development expenses was a result of lower headcount costs from the workforce reduction in December 2025 and lower clinical chemistry manufacturing and control costs. For the remainder of 2026, we expect continued investment in CMC and our clinical post-marketing commitments, offset by lower employee costs.

Due to wind-down activities, we do not expect any significant decrease in spending on our MF program in 2026. Selling, general and administrative expenses for the 3 months ended September 30, 2026, were $35.7 million compared to $39 million for the same period in 2025. This decrease was primarily due to lower general and administrative personnel-related expenses as a result of the workforce reduction in December 2025. Total operating expenses, excluding COGS, for the 3 months ended September 30, 2026 were $48.3 million compared to $60.1 million for the same period in 2025. As of September 30, 2026, we had approximately $339 million in cash, cash equivalents, restricted cash and marketable securities compared to $327 million as of June 30, 2026. We are committed to maintaining our financial discipline and are well positioned to fund growth from our current operations.

We are reiterating our 2026 RYTELO net revenue guidance with the expectation we will come in at the mid- to high end of the $220 million to $240 million range, reflecting consistent quarter-over-quarter net revenue growth throughout the year. We are lowering our total operating expense guidance from $230 million to $240 million to the range of $220 million to $230 million. This reduction reflects our ability to drive continued operational excellence, while also making strategic investments into RYTELO and future growth opportunities. With $339 million in cash, we are well positioned to execute on our financial and strategic priorities through the remainder of the year.

With that, I'll turn the call back to Harout for closing remarks.

Harout Semerjian

Thanks, Michelle. RYTELO has grown for 4 consecutive quarters, placing us on track to achieve our 2026 net revenue guidance and maximize our long-term growth potential. Beyond RYTELO, our financial strength gives us the flexibility to explore business development opportunities to build a leading hematology company.

Operator, we're now ready to start the Q&A session.

Operator

[Operator Instructions] And our first question comes from Tara Bancroft of TD Cowen.

質疑応答

Tara Bancroft

So it's great to see the reduced OpEx guidance this year. It all makes sense. But I'm wondering how we should think about OpEx expectations really going forward, especially with the Phase III not factored in. If there are any savings from that? Maybe you could tell us also what you plan to put that towards in terms of future pipeline or any thoughts regarding that, that would be great.

Harout Semerjian

Yes. Thank you, Tara. Maybe that's a question Michelle can address.

Michelle Robertson

Yes. Thanks, Tara. So yes, we reduced our OpEx for 2026. What I would say about the go-forward investments, we'll continue to be very disciplined in our capital allocation. You will see some increases next year as our Europe strategy matures as well as the post-marketing commitment in clinical trials. So although the remainder of the OpEx will be steady, we will see some increases for those 2 material later.

Operator

And our next question comes from Gil Blum of Needham.

Gil Blum

Congrats on the progress here. So definitely seeing consistent growth quarter-over-quarter kind of in the low -- in the single digits. What do you think could cause a rate change or change in the rate of uptake that would be more of an acceleration? And is this something we should expect? Or should we continue to see this kind of like consistent growth over time? And I have a follow-up.

Harout Semerjian

Thank you, Gil. As we have communicated multiple quarters ago, the way we look at RYTELO's growth, if we do things well, is it will deliver consistent growth quarter-over-quarter. And we're very pleased today to announce the fourth quarter of that strategy in action where we continue every quarter to increase demand to increase sales, to increase penetration in the first-line, second-line market patient segment and to reduce OpEx. And we believe that's a winning strategy on the long term. That's kind of where we are, Gil. I know sometimes people ask us -- what is the exact inflection point. And we continue to maintain this is a growth quarter-over-quarter if we do this well. And we do believe that there are thousands of more patients who can benefit from RYTELO for many years to go to come. That's why we do see the long-term perspective of RYTELO growth are quite good. But the curve of the growth is really what we are pleased with, and we think that this is something that we can continue to grow. I don't hope that answers your question. And I think you had a follow-up.

Gil Blum

Yes. That was helpful. And just given the financial discipline here and a pretty meaningful cash position. You guys mentioned BD a couple of times. Is there a specific character of product that you're looking for? Is this likely an [indiscernible] asset? I mean, any guidelines that you can give us to what you're thinking of?

Harout Semerjian

Sure. Yes. No, I mean so if you step back and look at where we are as a company, we have a very healthy base business in RYTELO in the U.S. that's growing every quarter and we reiterated our mid- to high level of our guidance of $230 million to $240 million, right? So that's from a U.S. perspective. We're also today, very excited to share that we're surgically -- the work that has happened ex-U.S. especially in Europe over the last several months, especially on the pricing side, puts us in a very good position to actually make the RYTELO available in the commercial setting, starting with Germany before end of the year and then rolling into other countries such as France next year while maintaining optionality of partnership, which is really a win-win strategy that doesn't put us in a situation where we have to shell out a lot upfront. We do it in a very, very specific way.

Now coming to BD, this also allows us -- especially now with the MF overhang behind us. Obviously, we're going to be looking into that very rich database and truly understand what are some of the trends in myelofibrosis. But given that, that's really -- it never was really part of our base strategy, at least not in the last couple of years, it was always treated as an upside. But now we're in a good situation to really pursue BD opportunities. So we have a winning team in the U.S. from a commercialization perspective. what would make the most sense is really to bring in another asset to put it in the bag of that team that's shown its ability to grow RYTELO and to get into other hematology oncology assets. As you know, 80% of this business is actually in the community. So it can really be any asset in hematology or oncology that is predominantly a community disease that as a first kind of bullseye.

And then we will keep the right to be opportunistic beyond that given that our approach of being very selective, targeted where we invest and really drive value through that has now been taking life over the last couple of -- over the last 4 quarters, I would say, and we do believe we can do that with other assets. We're not competing with a Pfizer or Novartis with needing assets with $2 billion, $3 billion peak sales, assets that have a few hundred million potential peak sales is plenty for us and then we can pursue a string of pearls approach with that over the next several years. So that's what we're excited about, not just for the base business, but also where we can go with BD. Does that help answer the question, Gil?

Gil Blum

Yes. Very helpful.

Operator

[Operator Instructions] Our next question comes from Stephen Willey of Stifel.

Stephen Willey

Maybe just a quick housekeeping question for Michelle. Any notable changes to inventory during the quarter? And then I just have a follow-up.

Michelle Robertson

No, you just remain within our 2% to 4% guidance that we saw share in the half 2 to 4 weeks.

Stephen Willey

And then just curious about how you're thinking about the opportunity here in Europe. I know that you're initially entering kind of a very limited number of markets. But can you maybe speak to any differences in how the disease might be managed there just relative to the U.S.? I guess when I look at REBLOZYL, they're generating, I think, less than 20% of their sales ex U.S. I'm sure is is partially pricing-based, but would perhaps suggest some differences in terms of how the disease is managed.

Harout Semerjian

Sure. Maybe I'll start off and then ElNawawi can supplement as well. Look, I mean, from a patient perspective, it's really the same size of number of patients that we have in the U.S., in the U.S. have said about 8,000 patients would be eligible for that second line patient population. And then as you know, European average age is even older. So it's at least that number of patients from a macro perspective overall. The reason obviously, we're choosing Germany to go with. I mean that is the country where you have a meaningful path for innovation in a healthy pricing point in line with the innovation that we can bring. ElNawawi, do you want to add on top of that additional reasons to believe?

Ahmed ElNawawi

Sure. And a couple of points maybe Steve, you referred to even BMS. They have gone through several price iterations because of their other label and multiple indications. We are a single indication drug with a outran designation, which does give us high showing, especially in Germany reimbursement perspective, plus the market is not as diversified in terms of payment options in Europe compared to the U.S., where in the U.S., we see a much higher use of HMA in second and third line, which is not reflect what the European market, so we see a bit of differences and by vote.

Harout Semerjian

Yes. And just to be also addressed, the reversal point you made, Steve. I mean that is you -- probably know that, that in most European markets, it is restricted to Rh positive. And I mean, typically larger pharma has to think about the portfolio overall, especially protecting larger products like their PD-1s. We don't have that interest, so we have a label that we can pursue especially in Rh negative patient population, Wilson designation. HMAs are not as entrenched in Europe, older population. And as you know, the clinical trial we had, IMERGE, was predominantly European. So there's significant advocacy as well from the medical community wanting to see RYTELO available for that patient.

Stephen Willey

Congratulations on achieving profitability.

Operator

Thank you. I show no further questions at this time. I'd like to turn it back to Harout Semerjian for closing remarks.

Harout Semerjian

Thank you to everyone joining our call today. We look forward to updating you on the progress in the next several quarters. Thank you.

Operator

This concludes today's conference call. Thank you for participating, and you may now disconnect.