Capricor Therapeutics (CAPR) Q2 2026 Earnings Call: Deramocel-BLA-Pfad und FDA-Update

Wichtigste Erkenntnisse

• Der Beratungsausschuss der FDA stimmte mit 3 Ja-Stimmen und 9 Nein-Stimmen gegen die Auffassung, dass die Nachweise die Wirksamkeit von Deramocel bei der Behandlung von Kardiomyopathie bei Patienten mit Duchenne-Muskeldystrophie (DMD) belegen.
• Capricor plant, den BLA-Antrag für Deramocel um 24-Monats-Daten aus der offenen Verlängerungsstudie von HOPE-3 sowie um zusätzliche Analysen zu ergänzen. Angestrebt wird eine präzisierte Indikation mit Schwerpunkt auf der Skelettmuskelfunktion der oberen Extremitäten. Die FDA deutete an, die Ergänzung nach Eingang zu prüfen und das derzeitige PDUFA-Zieldatum vom 22. August zu verlängern.
• Der primäre Endpunkt von HOPE-3 blieb statistisch signifikant. Deramocel verlangsamte das Fortschreiten der Erkrankung der oberen Extremitäten gemäß PUL 2.0 mit einer mittleren Differenz von 4,55 % und einem p-Wert von 0,029.
• Die Liquiden Mittel, Zahlungsmitteläquivalente und veräußerbaren Wertpapiere beliefen sich zum 30. Juni 2026 auf insgesamt 237,9 Millionen US-Dollar. Der Nettoverlust im zweiten Quartal weitete sich auf 40,7 Millionen US-Dollar bzw. 0,70 US-Dollar je Aktie aus.
• Die Betriebsausgaben im zweiten Quartal stiegen von 27,7 Millionen US-Dollar auf 42,9 Millionen US-Dollar, getrieben durch Investitionen in klinische Studien, regulatorische Belange, Herstellung und Kommerzialisierung zur Unterstützung des DMD-Programms.
• Capricor verlangsamt bestimmte Ausgaben für die Vorbereitung der Kommerzialisierung und hat die Arbeiten an der Pipeline ohne Bezug zu Deramocel vorübergehend ausgesetzt, bis eine größere regulatorische Klarheit besteht.

Wichtigste Finanzdaten

Geschäftsentwicklung und operative Leistung

Regulatorischer Pfad für Deramocel

Das negative Votum des FDA-Beratungsausschusses betraf eine eng gefasste Fragestellung zur Kardiomyopathie bei DMD. Das Management betonte, dass Kardiomyopathie ein wichtiger sekundärer Endpunkt in HOPE-3 war, während die Studie primär auf die Skelettmuskelfunktion der oberen Extremitäten als primären Endpunkt ausgelegt und konzipiert war.

Nach Gesprächen mit der FDA plant Capricor die Einreichung einer BLA-Ergänzung, die 24-Monats-Daten aus der offenen Verlängerungsstudie und weitere Analysen des bestehenden Datensatzes enthält. Die vorgeschlagene Indikation würde sich auf den in HOPE-3 gemessenen Endpunkt der Skelettmuskulatur der oberen Extremitäten konzentrieren.

Der primäre Endpunkt zeigte bei PUL 2.0 eine mittlere Differenz von 4,55 % zugunsten von Deramocel mit einem p-Wert von 0,029. Laut Capricor entsprach dies einer absoluten Differenz von etwa 1,2 Punkten.

Das Unternehmen hat in drei klinischen Studien rund 1.300 intravenöse Infusionen an mehr als 200 DMD-Patienten verabreicht. Mehr als 80 Patienten nehmen an offenen Verlängerungsstudien teil, von denen einige seit über fünf Jahren kontinuierlich Infusionen erhalten.

Statistisches Update zu HOPE-3

Während des Peer-Review-Prozesses und der Gespräche mit der FDA sowie „The Lancet“ identifizierte Capricor ein Problem mit dem statistischen Modell, das für den Endpunkt der linksventrikulären Ejektionsfraktion verwendet wurde. Unter dem vorab festgelegten Modell änderte sich der Behandlungsunterschied für alle Patienten von den zuvor berichteten 2,4 Prozentpunkten mit einem p-Wert von 0,04 auf 1,8 Prozentpunkte mit einem p-Wert von 0,09.

Laut Management war der primäre Endpunkt von HOPE-3 davon nicht betroffen. In der vorab festgelegten Kardiomyopathie-Subgruppe blieb das Ergebnis ebenfalls unverändert bei einem Behandlungsunterschied von 2,8 Prozentpunkten und einem p-Wert von 0,02. Endpunkte, die in der Testhierarchie unter der linksventrikulären Ejektionsfraktion liegen, werden nun als nominell signifikant eingestuft, wobei ihre Behandlungseffekte unverändert sind.

Herstellung und Kommerzialisierung

Capricors eigene GMP-Herstellungsanlage in San Diego ist betriebsbereit und darauf ausgelegt, bei einer Zulassung von Deramocel eine erste kommerzielle Markteinführung zu unterstützen. Der Ausbau der zweiten Etage der Anlage wird fortgesetzt, wobei das Management eine vollständige Validierung und FDA-Zulassung der erweiterten Flächen im Jahr 2027 anstrebt.

Aktivitäten zur Vorbereitung der Kommerzialisierung werden bis zur regulatorischen Klärung in einem langsameren Tempo fortgeführt. Michael Moore ist als Chief Commercial Officer zu Capricor gestoßen und baut gemeinsam mit der Führungsebene für den Marktzugang die Organisation für die Markteinführung auf.

Streitigkeit mit NS Pharma und Pipeline-Prioritäten

Capricor hat seinen Antrag auf einstweilige Verfügung ohne Präjudiz zurückgezogen und plant, den vertraglichen Streit mit NS Pharma im Wege eines Schiedsverfahrens beizulegen. Das Management geht davon aus, dass das Schiedsverfahren im Herbst 2026 beginnen wird, und strebt weiterhin die Aufhebung der US-Vereinbarung an.

Arbeiten an Pipeline-Programmen, die nicht direkt mit Deramocel in Zusammenhang stehen, sind vorübergehend ausgesetzt. Capricor hat regulatorische Gespräche in Europa und Japan aufgenommen, während potenzielle Studien mit jüngeren DMD-Patienten sowie zur Becker-Muskeldystrophie vom Fortschritt des US-Zulassungsverfahrens abhängen werden.

Ausblick des Managements

Das Management geht davon aus, dass die FDA das derzeitige PDUFA-Zieldatum vom 22. August nach Eingang der geplanten BLA-Ergänzung verlängern wird. Das Unternehmen schließt derzeit die Zeitplanung für die Einreichung ab.

Capricor steuert die Ausgaben für die Kommerzialisierung weiterhin verhalten und gab an, sich Flexibilität bei der Kapitalallokation für den Rest des Jahres 2026 vorzubehalten. Der Ausbau der Herstellungskapazitäten zielt weiterhin auf eine vollständige Validierung und FDA-Zulassung im Jahr 2027 ab, vorbehaltlich des regulatorischen Verfahrens.

Risiken und Beobachtungspunkte

• Der BLA-Antrag für Deramocel wird weiterhin von der FDA geprüft, und der Beratungsausschuss stimmte gegen die Nachweise zur Unterstützung der vorgeschlagenen Kardiomyopathie-Indikation.
• Die geplante BLA-Ergänzung wird den zeitlichen Rahmen der Zulassung verlängern, wobei das überarbeitete PDUFA-Datum von der Einreichung und der Prüfung durch die FDA abhängt.
• Die überarbeitete Analyse des Endpunkts der linksventrikulären Ejektionsfraktion ergab in der Gesamtstudienpopulation einen p-Wert von 0,09.
• Eine Inspektion der FDA zur Überwachung der biologischen Forschung (Bioresearch Monitoring) führte zu einem Formular 483 mit einer Beanstandung. Capricor hat seine Stellungnahme eingereicht und wartet auf Rückmeldung.
• Der Vertragsstreit mit NS Pharma bleibt ungelöst und wird voraussichtlich in ein Schiedsverfahren münden.
• Ausgaben für die Kommerzialisierung, der Zeitplan der Pipeline und die Expansionspläne hängen weiterhin von einer größeren regulatorischen Klarheit für Deramocel ab.

Vollständiges Transkript der Videokonferenz zu den Quartalszahlen

Vollständiges Transkript der Telefonkonferenz

Ausführungen des Managements

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.