$CAPR Total Catalyst Map

What is already known. HOPE-3 randomized 106 patients. Capricor reports a 54% slowing of upper-limb decline and p=0.029 in its published analysis. These are company-reported findings, not an FDA endorsement. In its July briefing, the FDA concluded that the original prespecified analysis did not meet the primary endpoint and questioned later statistical-plan changes, missing-data assumptions and potential unblinding.

What could change. The question is the reliability and clinical meaning of the treatment effect under an acceptable analysis. Capricor’s Q2 update also corrected the LVEF result to p=0.09; it cannot be described as a statistically significant cardiac endpoint. Results lower in the testing hierarchy require caution.

Risks and limits. Different analysis populations and methods produce different p-values. The company’s positive description and the FDA’s critical assessment must remain separately attributed. The July review predates the August major amendment.

Primary evidence: Capricor · July 29 HOPE-3 publication announcement · SEC · Q2 2026 Form 10-Q, filed August 14 · FDA · July 29 briefing document, especially pp. 8–9, 30–47 and 52 · Capricor · March 12 MDA presentation update

What is already known. The committee voted 3 in favor and 9 against, with no abstentions, on substantial evidence of effectiveness for cardiomyopathy in DMD. This was an adverse advisory vote on that question. Discussion of upper-limb function was not a separate favorable approval vote.

What could change. The FDA considers the panel’s advice but makes the final decision. Additional evidence and a revised proposed indication can change the review questions without erasing the concerns already documented.

Risks and limits. A new panel must not be invented as an upcoming event. The existing vote is neither an approval nor the final action on the amended application. No date for another AdCom is confirmed in the sources reviewed.

Primary evidence: Capricor · July 30 advisory committee results · FDA · July 29 meeting materials · FDA · July 29 briefing document, especially pp. 8–9, 30–47 and 52

What is already known. The September 17 announcement describes 24-month outcomes already included in the major amendment. Of 106 randomized patients, 82 reached that time point: 40 originally assigned to deramiocel and 42 to placebo. These counts describe follow-up, not a newly randomized trial.

What could change. Durability, consistency across analyses and clinically interpretable effect sizes could strengthen or weaken confidence in the amended case. Publication allows readers to assess the underlying methods beyond an announcement.

Risks and limits. After crossover, the extension does not retain a concurrent randomized placebo comparison. Attrition, selection, external natural-history matching and missing data matter. Scheduled presentations are not verified results, and their titles do not establish efficacy.

Primary evidence: Capricor · September 17 WMS announcement · SEC · August 24, Exhibit 99.1 · ClinicalTrials.gov · HOPE-3, NCT05126758

What is already known. The program concerns engineered muscle-targeting extracellular vesicles. Capricor identifies both presentations as preclinical. They are distinct from deramiocel, the DMD cell-therapy candidate under FDA review.

What could change. Evidence of delivery, biological activity and repeat-dosing feasibility could inform future development choices. A credible next step would require a funded development plan and, eventually, regulatory clearance for human studies.

Risks and limits. Preclinical delivery is not demonstrated patient benefit. Poster acceptance does not establish an IND filing, a clinical start or full resumption of previously paused internal programs. No such dates have been verified.

Primary evidence: Capricor · September 17 WMS announcement · SEC · Q2 2026 Form 10-Q, filed August 14

What is already known. The target moved from August 22 to November 22 after the FDA accepted a major amendment. The submission includes 24-month HOPE-3 extension analyses and a refined proposed indication focused on upper-limb function. Acceptance for review does not mean agreement with the evidence or the proposed label.

What could change. Approval would create a legal route to commercialization within the actual label. A complete response or a further procedural development would require a new assessment of the remaining work, timing and funding. Label breadth and practical treatment requirements matter alongside the headline decision.

Risks and limits. Efficacy, statistical robustness, safety and inspection issues remain material. The PDUFA date is a target, not a guarantee of approval or a guaranteed publication time. No approval probability is assigned here.

Primary evidence: Capricor · August 24 PDUFA extension · SEC · August 24, Exhibit 99.1 · Capricor · September 17 WMS announcement · FDA · July 29 briefing document, especially pp. 8–9, 30–47 and 52

What is already known. The Q2 10-Q reports a July 2026 Bioresearch Monitoring inspection and one Form 483 observation concerning procedures, documentation, vendor oversight and audit-report timing. Capricor had submitted its response and was awaiting feedback.

What could change. Acceptance of the response could remove one review uncertainty. A demand for additional remediation or evidence could add work and time. The BIMO observation concerns clinical-study oversight and should not be confused with a manufacturing-site inspection.

Risks and limits. The company’s view that the issue does not compromise data integrity is its own assessment. A submitted response is not a verified FDA clearance. Absence of a new public update cannot be treated as resolution.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · FDA · July 29 briefing document, especially pp. 8–9, 30–47 and 52

What is already known. The Q2 filing describes an externally supported phase 1 vaccine study. Preliminary findings were generally well tolerated but showed limited neutralizing-antibody responses. Final results were still expected. The broader pause in internal programs does not itself prove that this externally funded study stopped.

What could change. The balance of safety and immune responses could determine whether the platform merits additional investment or a subsequent study. A potential NIH-funded phase 2 is conditional on requirements being met; it is not a confirmed award or trial launch.

Risks and limits. Preliminary tolerability is not vaccine efficacy. A strong T-cell narrative cannot substitute for an actual final dataset. The forecast can move with the external study’s progress, and no registry identifier for this program was independently verified in this review.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · Capricor · August 13 corporate update

What is already known. Cash, equivalents and marketable securities totaled $237.9 million at June 30. Operating cash use was $60.7 million for the first six months. Q2 net loss was $40.7 million; this is a different measure from operating cash use. The latest filing supports operations for at least twelve months from issuance, not a newly confirmed runway through late 2027.

What could change. Fresh cash balances, manufacturing commitments, legal spending and any launch budget will show how much flexibility remains after the FDA action. Revenue quality matters if commercialization begins.

Risks and limits. A reporting window inferred from the normal quarterly cycle is not issuer guidance or a scheduled earnings call. Do not project runway by mechanically dividing cash by one quarter’s loss, or count uncertain milestones as cash received.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · Capricor · August 13 corporate update · Capricor · May 12 Q1 results · Capricor · official investor calendar

What is already known. Capricor’s May 7 action seeks rescission of the U.S. distribution agreement. Its allegations concern pricing and launch readiness; they are not adjudicated facts. The August 7 update described the court allowing the requested arbitration route. The company’s earlier sequencing assumptions preceded the move of the FDA target to November.

What could change. An outcome could alter who distributes deramiocel, how proceeds are shared and who funds launch execution. A settlement could remove uncertainty while retaining contractual compromises.

Risks and limits. The filing of a claim does not terminate the agreement. Capricor cannot be described as already free of the U.S. arrangement. Legal timing, damages and commercial terms are unresolved. The separate Japanese agreement must not be treated as automatically canceled.

Primary evidence: Capricor · May 7 legal action, updated August 7 · SEC · Q2 2026 Form 10-Q, filed August 14 · Capricor · August 13 corporate update

What is already known. The Q2 filing reports no additional ATM sales from January 1 through its August 14 filing date. It gives 58,138,989 common shares outstanding at August 13. Existing financing mechanisms and equity instruments remain relevant, but unused capacity is not cash in the bank.

What could change. New capital can extend development or launch capacity while changing existing shareholders’ percentage ownership. Terms, issue price, instrument type and actual proceeds are more informative than the existence of a shelf or ATM alone.

Risks and limits. The June cash snapshot is not the current balance. Regulatory delay, legal costs and manufacturing investment may change funding needs. No offering, size, price or completion date is forecast here. Share counts after the last verified filing may differ.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14

What is already known. Capricor states that deramiocel may qualify for a priority review voucher under the rare pediatric disease framework. The designation and the possibility of qualification are not a voucher already owned or sold.

What could change. If awarded and later monetized, a voucher could provide non-product cash. Award, transfer terms and cash receipt would be separate observable events.

Risks and limits. Qualification remains conditional. No value, purchaser, sale probability or timing is assumed in the cash snapshot or runway. This item is supported by the company’s SEC disclosure; an FDA award has not been verified.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14

What is already known. The lease permits either party to terminate if deramiocel has not received FDA approval on terms acceptable to Capricor by December 31. Termination requires notice within the contractual period; it is not automatic on the calendar date.

What could change. The outcome can affect facilities, future commitments and the manufacturing plan. A revised agreement or exercise of the right would need to be evaluated on its actual terms.

Risks and limits. This is a commercial contract condition, not a second FDA deadline or evidence that approval will occur. Lease accounting and operating cash payments are different measures. No automatic shutdown or lease cancellation is assumed.

Primary evidence: SEC · July 14 Form 8-K, lease agreement · SEC · Q2 2026 Form 10-Q, filed August 14

What is already known. Capricor has described preparations for a potential launch while moderating investment pending regulatory clarity. The FDA decision and the U.S. distribution dispute are separate dependencies. The existence of a facility does not establish a launch date.

What could change. Approval would shift the questions toward eligible patients, payer access, supply reliability, net economics and treatment delivery. Actual sales and cash collection would be stronger evidence of execution than a launch-intent announcement.

Risks and limits. Label restrictions, reimbursement, infusion logistics, manufacturing release and partner disputes can delay or limit uptake. No assumed price, eligible population, sales curve or market share is used in this map.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · Capricor · August 13 corporate update · Capricor · August 24 PDUFA extension

What is already known. NCT05126758 and NCT04428476 describe continuing follow-up. Their estimated study-completion entries are administrative study milestones. HOPE-3’s randomized primary analysis has already occurred; the registry date does not create a second future pivotal readout.

What could change. Longer follow-up may inform durability, safety and interpretation of the evidence. A new release would matter according to its design, completeness and relevance to the eventual regulatory question.

Risks and limits. Estimated completion dates can change. Completion does not guarantee immediate publication, and an open-label extension cannot be treated as a new placebo-controlled trial. No separate March 2027 results release has been verified.

Primary evidence: ClinicalTrials.gov · HOPE-3, NCT05126758 · ClinicalTrials.gov · HOPE-2 OLE, NCT04428476 · Capricor · September 17 WMS announcement

What is already known. The company distinguishes its initial manufacturing capability from a later expansion. The Q2 update places second-floor validation and inspection in 2027. This is a development target, not completed clearance or a booked inspection appointment.

What could change. Additional compliant capacity could support demand if deramiocel is approved and adopted. Until then, the balance between readiness and cash preservation remains important.

Risks and limits. Validation delays, process consistency, inspection findings and capital requirements can change the schedule. More physical space does not automatically translate into releasable doses or revenue. The 2027 target is kept outside specific quarters because the source does not allocate one.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · Capricor · August 13 corporate update

What is already known. The Japanese Nippon Shinyaku arrangement is separate from the U.S. dispute. The Q2 filing says the European term sheet expired and no definitive European distribution agreement had been executed as of June 30. Regulatory engagement outside the U.S. does not itself establish an application or decision date.

What could change. Regional rights, cost sharing and milestone eligibility could broaden development and financing options. A signed agreement would need assessment of obligations and conditional payments, not just headline potential.

Risks and limits. Do not carry an expired term sheet forward as an active commercial partnership. U.S. approval would not automatically authorize Japan or Europe. No unearned milestone is included in liquidity or runway.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · Capricor · May 12 Q1 results

What is already known. Deramiocel is CAP-1002. Capricor discusses younger DMD populations and Becker muscular dystrophy as potential extensions, subject to the lead program’s path. Historical CAP-1002 work in COVID-19, cardiac and pulmonary settings does not establish a current catalyst: INSPIRE, HFpEF and ALPHA are listed as completed, while ALLSTAR is terminated.

What could change. A formal new program would define population, endpoint, budget and development requirements. It could diversify the pipeline, but would also add execution and financing demands.

Risks and limits. Older studies and scientific rationale are not active late-stage programs. No near-term efficacy result is inferred from a historical registry record. The 2026–2027 horizon here is monitoring only, with no verified new-indication timetable.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14 · ClinicalTrials.gov · INSPIRE, NCT04623671 · ClinicalTrials.gov · ALLSTAR, NCT01458405 · ClinicalTrials.gov · HFpEF, NCT02941705 · ClinicalTrials.gov · ALPHA, NCT03145298

What is already known. The 10-Q reports a Mesoblast patent action involving deramiocel, alleging infringement of three patents and seeking remedies including damages and injunctive relief. These are allegations and requested remedies, not a finding of infringement or an issued injunction.

What could change. A substantive outcome could affect legal costs, licensing economics or operating freedom. This risk is separate from the Nippon Shinyaku distribution dispute and from FDA review.

Risks and limits. No merits outcome, damages amount, restriction on current activity or resolution date is inferred. The current description relies on one authoritative company SEC filing; a later court docket outcome was not independently verified.

Primary evidence: SEC · Q2 2026 Form 10-Q, filed August 14