Financial Data and Key Metrics Changes - The net loss attributable to common stockholders for Q2 2025 was approximately $24.5 million, compared to approximately $9.7 million for the same period in 2024, indicating a significant increase in losses [33] - Research and development expenses totaled approximately $5.8 million for Q2 2025, down from approximately $7.1 million in Q2 2024 [33] - General and administrative expenses were approximately $2.3 million for Q2 2025, compared to approximately $2.8 million for the same period in 2024 [33] - The impairment of acquired in-process research and development intangible assets was $16.5 million, compared to zero during the same period in 2024 [34] - As of June 30, 2025, the company had cash and cash equivalents of approximately $33.4 million, which is expected to fund operations into 2026 [34] Business Line Data and Key Metrics Changes - The phase two MINDFUL trial for EXPAREL confirmed that Alzheimer's patients with two or more biomarkers of inflammation are optimal candidates, with an estimated 40% to 60% of all Alzheimer's cases fitting this profile [7][8] - In the key subgroup of patients with high inflammation, the trial observed an effect size of 0.27 on the primary endpoint EMAC and 0.23 on a key secondary behavioral endpoint, the neuropsychiatric inventory [15][16] - The company has decided not to pursue treatment-resistant depression at this time and is reevaluating opportunities for EXPAREL beyond Alzheimer's disease [10][11] Market Data and Key Metrics Changes - The company anticipates filing for approval of Cordstrom in both the UK and US by mid-2026, with expectations that it will qualify for a Priority Review Voucher if approved in the US [12] - The company is exploring shorter pathways to market for EXPAREL, including opportunities in rare diseases to maximize impact and accelerate patient access [9] Company Strategy and Development Direction - The company is focused on securing strategic partnerships to advance the EXPAREL program, which requires substantial investment in manufacturing and clinical trials [8][9] - The company aims to prepare for an end of phase two meeting with the FDA to align on the design of the phase three trial for EXPAREL [21] - The company is committed to exploring strategic partnerships to fuel growth and complete trials, while also ensuring the development of Cordstrom and INKmune [36] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of EXPAREL as a first-in-class drug for Alzheimer's disease, emphasizing its unique approach to addressing neuroinflammation [7][8] - The management highlighted the importance of the recent phase two trial results and the potential for strategic partnerships to support the program's advancement [22] - Management acknowledged the challenges of the current operating environment but remains optimistic about the company's robust pipeline and potential value creation [36] Other Important Information - The company plans to publish the trial results in a peer-reviewed journal and prepare a briefing book for the FDA meeting, expected to occur before the end of the year [35] - The company is actively exploring additional indications for Cordstrom beyond recessive dystrophic epidermolysis bullosa [12][26] Q&A Session Summary Question: Confirmation of end of Phase two meeting date with FDA - The specific meeting date has not been set yet, and the briefing book is being prepared [41] Question: Atmosphere at AAIC and feedback from thought leaders - Feedback was positive, with strong interest in the neuropsychiatric inventory and the absence of amyloid-related imaging abnormalities [42][44] Question: Strategic partnerships for EXPAREL - A partnership is not expected until after the end of phase two meeting with the FDA, as partners will want to see the publication and FDA feedback first [46][48] Question: Insights from ongoing statistical analysis for Cordstrom - The statistical analysis plan is expected to yield improved data for submission, and the open-label trial is planned for early 2027 [70][72]

XPro Program for Alzheimer's Disease - XPro1595 is designed to selectively inhibit soluble TNF to treat neurologic disease, with Phase 1b study demonstrating safety and dose-dependent reduction in inflammatory cytokines in cerebrospinal fluid (CSF)[34] - A Phase 2 study of XPro1595 in early Alzheimer's patients with biomarkers of inflammation used an enriched population (n=100) with amyloid and ≥ 2 biomarkers of inflammation, showing a beneficial signal across multiple measures[42, 57] - In the Safety Analysis Set (SAF) population (n=206) for the Phase 2 study, 51% were female, and 95.1% were white[39] - In the mITT population (n=200), 75% were Amyloid-beta (Aβ) positive[39] - The most common Treatment Emergent Adverse Event (TEAE) in the XPro1595 group was Injection Site Reaction (ISR), occurring in 52.5% of patients[54] - The company plans to validate the enriched population in a fully powered trial and anticipates an end-of-Phase 2 meeting with the FDA in Q4 2025[56, 59] CORDStrom Program for RDEB - CORDStrom is an investigational disease-modifying treatment for recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic disease affecting an estimated 4000 people in the US, UK, and EU, representing a > $1B peak sales opportunity[64] - A Phase 2 trial of CORDStrom in 30 pediatric patients with RDEB showed beneficial effects with respect to Itch Man Scale, iscorEB clinician score, skin score, and QOL, with no CORDStrom-related serious adverse events reported[66] - The company plans to compile and file a BLA in the US & MAA in UK/EU in 1H 2026[77] INKmune Program for Cancer - INKmune is an off-the-shelf NK cell therapy candidate designed to convert patient's resting NK cells into cancer-killing memory-like NK cells[78] - An INKmune® mCRPC Phase I/II Trial is ongoing, with safety endpoints met and evidence of in-vivo NK cell activation and regression of some tumor lesions by PSMA-PET[80]