カプリコア・セラピューティクス（CAPR）2026年第2四半期決算説明会：デラモセルのBLA申請経路とFDA最新情報

主要ポイント

• FDA（米食品医薬品局）の諮問委員会は、デュシェンヌ型筋ジストロフィー（DMD）患者の心筋症治療におけるデラモセル（deramocel）の有効性を裏付けるデータに関して、賛成3、反対9で否決しました。
• カプリコール（Capricor）は、24ヶ月間のHOPE-3オープンレーベル継続投与試験データおよび追加解析を含めたデラモセルの新薬承認申請（BLA）の補正を行い、上肢骨格筋機能に焦点を当てた適応症の明確化を目指す計画です。FDAは、補正の受領後に内容を審査し、現在の8月22日のPDUFA目標日を延期する意向を示しました。
• HOPE-3試験の主要評価項目は統計的有意性を維持しました。デラモセルはPUL 2.0において上肢の病勢進行を抑制し、平均差4.55%、p値は0.029でした。
• 2026年6月30日時点の現金、現金同等物および有価証券の残高は総額2億3,790万ドルとなりました。第2四半期の純損失は4,070万ドル（1株当たり0.70ドル）に拡大しました。
• 第2四半期の営業費用は、DMDプログラムを支援する治験、規制関連、製造および商業化への投資増加により、前年同期の2,770万ドルから4,290万ドルに増加しました。
• カプリコールは、規制上の見通しが明確になるまでの間、一部の商業化準備費用の支出を抑制し、デラモセルに関連しないパイプラインの開発を一時停止しています。

主要財務データ

事業および業績の動向

デラモセルの規制手続き・承認経路

FDA諮問委員会による不承認勧告（反対投票）は、DMDにおける心筋症に関する限定的な質問に対するものでした。経営陣は、心筋症がHOPE-3試験の主要な副次評価項目であった一方、同試験は上肢骨格筋機能を主要評価項目として設計・検証されたものであることを強調しました。

FDAとの協議を受け、カプリコールは24ヶ月間のオープンレーベル継続試験データおよび既存データセットの追加解析を含むBLA補正申請書を提出する予定です。提案する適応症は、HOPE-3で測定された上肢骨格筋の評価項目に焦点を当てることになります。

主要評価項目では、PUL 2.0においてデラモセル群に有利な4.55%の平均差が示され、p値は0.029でした。カプリコールは、これが絶対値で約1.2ポイントの差に相当すると述べています。

同社はこれまでに3つの治験を通じて、200名以上のDMD患者に対し約1,300回の静脈内投与を実施しました。80名以上の患者がオープンレーベル継続投与研究に参加しており、一部の患者は5年以上にわたり継続投与を受けています。

HOPE-3の統計データ更新

ピアレビューならびにFDAおよび「ランセット（The Lancet）」誌との協議の際、カプリコールは左室駆出率の評価項目に使用された統計モデルの問題点を特定しました。事前指定されたモデルのもとでは、全患者における治療差は、以前報告された2.4パーセンテージポイント（p値0.04）から、1.8パーセンテージポイント（p値0.09）に変更となりました。

経営陣は、HOPE-3の主要評価項目には影響がないと説明しました。事前指定された心筋症サブグループにおいても、治療差は2.8パーセンテージポイント、p値は0.02で変更ありませんでした。検証階層において左室駆出率より下位にある評価項目は、治療効果自体は不変であるものの、現在は名目上有意であると定義づけられています。

製造および商業化

サンディエゴにあるカプリコールの自社GMP製造施設は稼働状態にあり、デラモセルが承認された場合の初期商業ローンチに対応できる体制を整えています。同施設の2階部分の拡張工事は継続中であり、経営陣は2027年に拡張スペースの完全なバリデーション（適格性評価）およびFDA承認の取得を目指しています。

商業化に向けた準備活動は、規制上の見通しが明確になるまでペースを落として継続されています。マイケル・ムーア（Michael Moore）氏が最高商業責任者（CCO）としてカプリコールに参画し、同社のマーケットアクセス部門の指導陣とともに製品投入に向けた組織構築を進めています。

NSファルマとの紛争およびパイプラインの優先順位

カプリコールは仮処分申請を権利放棄なしで取り下げ、NSファルマとの契約上の紛争について仲裁手続きを通じて解決を図る計画です。経営陣は仲裁が2026年秋に開始されると見込んでおり、引き続き米国での契約解除を求めています。

デラモセルに直接関連しないパイプラインプログラムの開発は保留されています。カプリコールは欧州および日本での規制当局との協議を開始しており、より若年のDMD患者やベッカー型筋ジストロフィーを対象とした潜在的な治験の実施は、米国での規制手続きの進展次第となります。

経営陣の見通し

経営陣は、予定されているBLA補正の提出受領後、FDAが現在の8月22日のPDUFA目標日を延期すると予測しています。同社は提出の時期を最終調整しています。

カプリコールは商業化関連支出のペース調整を継続しており、2026年の残りの期間における資本配分の柔軟性を維持していると述べています。同社の製造拡張計画については、規制手続き次第ではあるものの、引き続き2027年中の完全なバリデーションとFDA承認の獲得を目指しています。

リスクおよび注視事項

• デラモセルのBLAは引き続きFDAの審査下にあり、諮問委員会は提案された心筋症の適応症を裏付ける証拠に対して反対票を投じました。
• 予定されているBLA補正の提出により規制手続きのスケジュールが延長され、改定後のPDUFA完了目標日は提出およびFDAの審査状況に依存します。
• 左室駆出率評価項目の改定解析において、全対象患者集団におけるp値は0.09となりました。
• FDAによるバイオリサーチ・モニタリング（BIMO）査察の結果、1件の指摘事項を含む「Form 483」が交付されました。カプリコールはすでに回答書を提出しており、フィードバックを待っている状態です。
• NSファルマとの契約紛争は未解決のままであり、仲裁手続きへ移行する見込みです。
• 商業化への支出、パイプラインのスケジュール、拡張計画は、デラモセルに関する規制上の見通しがより明確になるかどうかに引き続き依存しています。

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 afternoon ladies and gentlemen and welcome to the Capricor Therapeutics Second Quarter 2026 Conference Call. [Operator Instructions] The call is being recorded on Thursday, August 13, 2026. And I would now like to turn the conference over to CFO, AJ Bergmann, for the forward-looking statement. Please go ahead.

Anthony Bergmann

Thank you very much. Before we begin, I'd like to remind you that any statements made during today's call that are not historical are considered to be forward-looking statements. Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the risk factors section of our company's most recent annual report on Form 10-K. And our most recent quarterly reports on Form 10-Q, as well as other reports filed with the SEC, any forward-looking statements may represent our views as of today, August 13, 2026. An audio replay of the call will be available on our website following its completion. With that, I will turn the call over to Linda Marbán, CEO.

Linda Marbán

Good afternoon everyone and thank you for joining Capricor's second quarter 2026 earnings call. Our BLA for deramocel remains under review with the FDA with a current PDUFA target action date of August 22. Because that review is ongoing, there is a limit to what I can say about our interactions with the agency, but wanted to provide an update across 3 main topics: our regulatory status, pathway for deramocel, our commercial and manufacturing readiness, and our dispute with NS Pharma. I will then briefly address our pipeline programs before turning it back to AJ.

On July 29, 2026, the FDA convened the Cellular, Tissue, and Gene Therapies Advisory Committee to review our BLA. The committee was presented with a single voting question. Does the available evidence provide substantial evidence of effectiveness of deramocel for the treatment of cardiomyopathy in patients with DMD? The vote was 3 in favor, 9 against, with 0 abstentions.

That is not the outcome we had planned for, and we are, of course, disappointed, but we remain committed to working with the FDA on the next steps for this program. Our priority is, and always has been, to get deramocel to those who need it most.

I would like to provide some color in our perspective about why we continue to believe in the potential of deramocel in DMD patients. First, the indication we originally requested in the BLA going back to 2024 was the treatment of cardiomyopathy in DMD. Therefore, the focus of the FDA and the advisory committee was on whether deramocel should be approved to treat cardiomyopathy. However, the measurement of deramocel's effect on cardiomyopathy was a key secondary endpoint rather than the primary endpoint of the HOPE-3 study. And it measured change in ejection fraction across the full DMD population, rather than in patients with established cardiomyopathy, the population the proposed indication addresses.

By contrast, HOPE-3 was actually designed with a skeletal functional primary endpoint and with power to assess efficacy in upper limb function. [ In pre, ] the advisory committee was not asked to vote on whether they believe the data on the HOPE-3 primary efficacy endpoint could support approval of the product, nor whether the overall benefit-risk profile of deramocel was favorable. We continue to believe that the data on the primary, as well as multiple other endpoints, support a finding of effectiveness on these measures.

It is worth noting that in a separate discussion on upper limb function during the ADCOM, the committee's feedback was directionally supportive of the clinical evidence for the primary endpoint in upper limb function. The discussion was substantive and the full record is public for anyone who wants to review it.

Now, this brings me to an update that I am very pleased to share. We are continuing to work closely with FDA on a potential path forward for deramocel focused on an upper limb skeletal muscle indication reflected in the primary efficacy endpoint of HOPE-3.

To that end, following discussions with the agency, subsequent to our Advisory Committee meeting, we plan to submit an amendment to our BLA that includes the 24-month open-label extension data from the HOPE-3 study, along with additional analyses on the existing data package, in order to support a refined indication focused on the primary endpoint. The FDA has indicated it is willing to review this amendment and upon receipt to extend the PDUFA action date accordingly. We are finalizing the timing of that submission and will provide an update as appropriate.

We appreciate the FDA's engagement throughout this process and its shared commitment to addressing the major unmet need for Duchenne muscular dystrophy.

Now there were 2 other developments in the review this quarter. In July, we were proud to report that the results of the HOPE-3 clinical trial were published in The Lancet following extensive and independent peer review. The first publication of the full Phase 3 dataset, an important milestone for this program and for the field.

The publication highlights the efficacy of deramocel and the supplement highlights the mechanism of action as well as the individual patient-level data. There's a lot of information available publicly, and we are confident that this highly regarded publication will help support continued progress for our deramocel program.

In connection with that peer review and as part of our dialogue with the FDA and The Lancet, we identified an issue with the statistical model in the clinical study report and reverted back to the statistical analysis plan version 3.0 put in place prior to unblinding. That model, the one underlying our top-line release, included an interaction term combining 2 independent variables, age and baseline, which were part of the pre-specified plan.

The only endpoint directly impacted was left ventricular ejection fraction in all patients, the key secondary endpoint of the HOPE-3 study. At top line, we reported a 2.4 percentage point treatment difference with a p-value of 0.04. As published in The Lancet under the pre-specified model, the measure of left ventricular ejection fraction in all patients was a 1.8 percentage point treatment difference with a p-value of 0.09. We took the most conservative approach available to us in the publication and in follow-up interactions with FDA.

Nothing else changed in the data or its analysis. We remind you in the pre-specified cardiomyopathy subgroup, the result was unchanged at p equals 0.02 with a 2.8 percentage point treatment difference. The endpoints below left ventricular ejection fraction in the testing hierarchy are characterized now as nominally significant with treatment effect unchanged.

Now, let me be clear that the HOPE-3 primary endpoint was unaffected and is significant both statistically and clinically. Deramocel demonstrated a statistically significant slowing of upper limb disease progression as measured by PUL 2.0 with a mean difference of 4.55% in favor of deramocel with a p-value of 0.029, which corresponds to a 1.2 point absolute change in [ total full point of ]. We believe the efficacy and safety data supporting the potential for deramocel is strong.

We have administered approximately 1,300 intravenous infusions across our clinical program to over 200 patients with DMD in 3 separate clinical trials. More than 80 patients are in our collective open-label extension studies, with some receiving continuous infusions for more than 5 years, and the long-term safety profile is consistent and well-characterized.

The open public hearing part of the advisory committee included testimony from patients, families and clinicians living with Duchenne muscular dystrophy. We were grateful that their experience is part of the record, and we look forward to continuing with the FDA on a path forward for deramocel.

Also in July, as part of the review process, the FDA conducted a bioresearch monitoring inspection, or BIMO, and issued a Form 483 citing 1 observation. We have submitted our responses and are currently awaiting feedback.

Second, let me talk a little bit about our commercial readiness and manufacturing. We are continuing our commercial readiness activities, but at a slower pace until we have further regulatory clarity. And although the scope and timing of some of them may change, depending on the outcome of the review, we are controlling our cash against this.

Our in-house GMP manufacturing facility in San Diego is operational and positioned to support an initial commercial launch if approved. The expansion to the second floor of that same facility continues, and our goal remains full validation and FDA approval of the expanded space estimated to be in 2027. The space is ideal for early commercialization and allows for the most flexibility as we continue to scale our CMC capacity to account for potential demand.

On the commercial side, Michael Moore joined us as our Chief Commercial Officer, bringing direct DMD and rare disease commercial experience. And he has judiciously been building out the launch organization alongside our market access leadership.

Now let me talk for a minute about our dispute with NS Pharma. The state court was scheduled to hear our motion for preliminary injunction on August 10, ahead of the FDA's expected PDUFA date. However, we determined that resolving this contractual dispute in arbitration following the agency's decision would give the parties a more complete regulatory record to work from. Therefore, we withdrew the motion without prejudice.

In terms of timeline, we estimate the arbitration process to begin this fall to address the contract dispute while continuing to pursue commercial readiness activities for deramocel. Now, let me be clear that our position on the underlying dispute has not changed. We continue to believe that the pricing structure in the U.S. agreement is fundamentally flawed in a way that would impede patient access and we continue to seek rescission. What changed is the current process by which we are pursuing a remedy. Our view of the merits of the case has not changed.

Now, very quickly turning to our pipeline, I would like to state that all pipeline work that is not directly related to deramocel is on hold right now until we have further regulatory clarity. Having said that, in terms of life cycle management of deramocel, we have initiated regulatory engagement in Europe and Japan. Our expansion plans, including those for younger DMD patients and for Becker muscular dystrophy, remain priorities, and the timing of those clinical trial initiations will be stage-gated by the timeline of our regulatory pathway for deramocel in the U.S. to treat those with Duchenne muscular dystrophy later stage.

With that, I will now turn the call over to AJ to review the financial results.

Anthony Bergmann

Thank you, Linda. As of June 30, 2026, Capricor had cash, cash equivalents and marketable securities totaling approximately $237.9 million, and there was no revenue recognized for the second quarter of 2026 or 2025.

Total operating expenses for the second quarter of 2026 were approximately $42.9 million compared to approximately $27.7 million for the second quarter of 2025. The increase was primarily driven by continued investment in clinical, regulatory and manufacturing activities as well as commercial infrastructure supporting our Duchenne program.

Net loss for the second quarter of '26 was approximately $40.7 million or $0.70 per share compared to a net loss of approximately $25.9 million or $0.57 per share for the second quarter of 2025. And for the 6 months ended June 30, 2026, our net loss was approximately $74.7 million compared to approximately $50.3 million for the same period in 2025.

As of June 30, 2026, we had an accumulated deficit of approximately $379.6 million. Our expense profile this quarter reflects investment across our 3 main areas: regulatory and clinical activities in support of our DMD program, manufacturing capacity expansion efforts, and commercial readiness activities.

As Linda noted, we are pacing certain commercial expenditures as the regulatory timeline develops and becomes more clear, and we continue to have flexibility in how we deploy capital across the remainder of the year. With that, I will turn the call back over to Linda for a closing.

Linda Marbán

Thank you, AJ. As all of you know, the last year has been one of highs and lows for Capricor. We were stunned by the [indiscernible] and pleased by the HOPE-3 data. We were encouraged by the acceptance of the HOPE-3 data for resubmission in response to the [indiscernible] and disappointed by the advisory committee's recommendation. Although we understood it based on the narrow voting question and the disparity between the indication we had previously asked for and the data from HOPE-3, which was powered to assess skeletal muscle as its primary goal.

We have previously stated this, we were reassured by the strength of our data by publication in The Lancet, and we were amazed by the outpouring of support for deramocel by the DMD community. We hear their voices as well and will continue to work tirelessly to try and get deramocel to every eligible patient based on their physician's recommendation.

We are grateful to FDA for their flexibility and for their collaborative approach. We will be submitting updated data to the FDA as soon as possible and look forward to their review.

Lastly, due to the sensitivity of our ongoing discussions with the Food and Drug Administration, we are not holding a Q&A today, and we look forward to providing updates to you as they become available. Thank you for your time. We look forward to positive updates in the future.

Operator

This concludes today's call. Thank you all for participating. You may now disconnect.