Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company focused on developing novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases [3] - The company has a late-stage pipeline that includes 11 clinical programs, primarily centered around two compounds: MN-166 (ibudilast) and MN-001 (tipelukast) [3] - MN-166 (ibudilast) is currently in Phase 3 trials for amyotrophic lateral sclerosis (ALS) and degenerative cervical myelopathy (DCM), and is Phase 3-ready for progressive multiple sclerosis (MS) [3] Clinical Trials - An abstract regarding the Phase 2b/3 COMBAT clinical trial of MN-166 (ibudilast) in ALS patients has been selected for a poster presentation at the 36th International Symposium on ALS/MND scheduled for December 5-7, 2025 [1][2] - The presentation will cover trial updates and baseline characteristics, indicating ongoing progress in the clinical development of MN-166 [2] Product Information - MN-166 (ibudilast) is a small molecule that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, and is being developed for various neurodegenerative diseases, including ALS, progressive MS, and DCM [2] - The compound is also under investigation for other conditions such as glioblastoma, Long COVID, chemotherapy-induced peripheral neuropathy (CIPN), and substance use disorder [2]

Business Highlights - Roivant completed a $1.5 billion share repurchase program in June 2025, repurchasing approximately 149 million shares at an average price of $10.09, reducing the share count by over 15%[23] - The company expanded its pipeline by initiating 6 potentially registrational studies and 3 proof-of-concept (POC) studies[23] - Roivant increased shareholder exposure to clinical and litigation catalysts over the next 36 months[23] Brepocitinib Program - Topline data from the Phase 3 VALOR study of brepocitinib in dermatomyositis (DM) is expected in the second half of 2025[20, 27] - The VALOR study enrolled 241 adults with active DM, with 38% from the US, 32% from the EU, and 30% from the rest of the world[35] - Approximately 40% of subjects taking oral corticosteroids (OCS) at baseline in the VALOR study were able to eliminate OCS entirely by the end of the study[45] LNP Litigation - Summary judgment phase is ongoing in the US Moderna case, with a jury trial scheduled for March 2026[18, 27, 52] - Ongoing progress is expected in the Pfizer/BioNTech case following the Markman hearing, with a decision potentially in 2025[18, 27, 52] Financial Update - Research and development (R&D) expense was $153 million, with an adjusted R&D expense of $141 million (non-GAAP)[58] - General and administrative (G&A) expense was $134 million, with an adjusted G&A expense of $63 million (non-GAAP)[58] - Roivant had $4.5 billion in cash, cash equivalents, restricted cash, and marketable securities as of June 30, 2025[58]

Summary of Clinical Trial Pipeline Tracker Industry Overview - **Industry**: Biopharma - **Region**: North America - **Industry View**: In-Line for Biotechnology, Attractive for North America [6][6] Key Changes in Clinical Trials New Trials Initiated 1. **Acadia**: Initiated a Phase 3 trial (NCT07095465) of ACP-204 for Lewy Body Dementia Psychosis [8] 2. **BeOne Medicines**: Initiated a Phase 2 trial (NCT07100938) for BGB-45035 versus placebo in adults with moderate to severe active rheumatoid arthritis [8] 3. **Bristol-Myers Squibb**: - Initiated a Phase 2/3 trial (NCT07100080) of Izalontamab Brengitecan (BMS-986507) versus Platinum-Pemetrexed for EGFR-mutated non-small cell lung cancer after failure of EGFR TKI therapy [8] - Initiated a Phase 1 trial (NCT07105059) of Teclistamab and Mezigdomide in people with multiple myeloma [8] 4. **Jazz Pharmaceuticals**: Initiated a Phase 2 trial (NCT07102381) of Zanidatamab in combination with chemotherapy for HER2-positive breast cancer [8] 5. **Neurocrine**: Initiated a Phase 3 trial (NCT07105098) for NBI-1117568 in inpatient adults with schizophrenia [8] Trials Currently Recruiting 1. **Abbvie**: Recruiting in a Phase 2 study (NCT07023289) to assess adverse events in adults receiving Telisotuzumab Adizutecan for colorectal cancer [9] 2. **Biohaven**: Recruiting in a Phase 1 study (NCT07054684) of BHV-1400 in IgA nephropathy [9] 3. **Bristol-Myers Squibb**: - Recruiting in a Phase 2 study (NCT06762769) of Isatuximab and Iberdomide for high-risk smouldering myeloma [9] - Recruiting in multiple other studies for various conditions [9][17] Suspended Trials - No specific trials were reported as suspended during this period [3][19] Terminated Trials - No specific trials were reported as terminated during this period [4][21] Other Important Information - The report includes a comprehensive list of trials, their stages, and identifiers for tracking purposes [18][22] - The document emphasizes the potential conflicts of interest due to Morgan Stanley's business relationships with the companies covered [6][6] Conclusion The biopharma industry in North America is actively progressing with numerous new clinical trials, particularly in oncology and neurodegenerative diseases. The ongoing recruitment efforts indicate a robust pipeline, while the industry outlook remains positive.

Financial Data and Key Metrics Changes - Q2 Rytello net revenues were $49 million, representing an increase of approximately 24% over the first quarter [5][29] - As of June 30, 2025, cash and marketable securities were approximately $433 million, up from $503 million as of December 31, 2024 [29] - Research and development expenses for Q2 were $22 million, down from $31 million for the same period in 2024, primarily due to lower clinical trial costs [30] Business Line Data and Key Metrics Changes - Demand growth in Q2 was 17% higher compared to Q1, with approximately 1,000 sites of care utilizing Rytello, an increase of about 400 new sites since the beginning of the year [13][14] - Approximately two-thirds of accounts that previously ordered Rytello have reordered in Q2 [14] - Payer access improved, with approximately 90% of US covered lives now under favorable Rytello medical coverage policies, up from 85% in Q1 [16] Market Data and Key Metrics Changes - The company is focused on increasing brand awareness among US hematologists treating lower-risk MDS patients, with positive feedback on educational efforts [7][23] - The company plans to expand its commercial sales force and customer-facing roles by over 20% to enhance KOL support and advocacy [8][20] Company Strategy and Development Direction - The primary strategy remains the successful commercialization of Rytello in the US, with a focus on increasing brand awareness and physician education [5][10] - The company is preparing for the launch of Rytello in select EU markets next year, maintaining financial discipline in investments for this launch [10][22] - The appointment of Harout Semerjian as the new President and CEO is expected to drive shareholder value and enhance the company's strategic direction [4][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of commercial strategies and the potential for continued growth, particularly in the US market [11][54] - The company is optimistic about the enrollment momentum in the Phase III IMPACT MF trial, expecting completion by year-end [11][25] Other Important Information - The company has doubled the size of its medical affairs team to enhance community awareness and KOL advocacy [23] - The gross-to-net percentage remained in the mid-teens from Q1 to Q2, consistent with previous guidance [29] Q&A Session Summary Question: How many active patients are now receiving Rytello? - The company estimates that there are at least 600 active patients based on the number of accounts that have prescribed Rytello [36] Question: How sustainable is the 17% quarter-over-quarter demand growth? - The company focuses on business drivers such as new patient starts and duration of treatment, with positive trends observed in physician prescribing behavior [42][44] Question: What was the biggest issue that held back the launch in Q4 and Q1? - Management identified the need to increase awareness, ensure prescribing comfort, and engage KOLs as key strategies that have been pivoted since March [52][53] Question: Any qualitative insights on early Q3 demand trends? - The company sees conscious optimism in demand trends based on internal sales data and claims data [62] Question: Any anecdotes about the type of physicians currently prescribing Rytello in earlier lines? - The company noted that earlier line use is seen among physicians who have had patient success in later lines of therapy [71] Question: What is the EU commercialization strategy? - The company is focused on securing the highest possible reimbursable rate and is engaged with potential partners for commercialization in the EU [81][84]

Core Viewpoint - Contineum Therapeutics reported a wider GAAP net loss per share of $(0.62) for Q2 2025, missing the estimate of $(0.44), while continuing to advance its clinical pipeline with significant increases in research and development expenses [1][2][5] Financial Performance - GAAP EPS for Q2 2025 was $(0.62), a 59.0% decrease from $(0.39) in Q2 2024 [2] - Research and Development Expense rose to $14.1 million, a 78.5% increase from $7.9 million in Q2 2024 [2] - General and Administrative Expense increased to $3.8 million, up 26.7% from $3.0 million in Q2 2024 [2] - The company ended the quarter with a cash position of $175.5 million, sufficient to fund operations through 2027 [10] Business Overview - Contineum focuses on developing small molecule therapies for diseases with high unmet medical needs, particularly through its main drug candidates PIPE-791 and PIPE-307 [3][4] - PIPE-791 is being studied for idiopathic pulmonary fibrosis (IPF), progressive multiple sclerosis (PrMS), and chronic pain, while PIPE-307 targets depression and relapsing-remitting multiple sclerosis (RRMS) [3][4] Operational Highlights - The company advanced PIPE-791 in multiple indications and completed chronic toxicity studies, with plans for a global Phase 2 trial for IPF later in 2025 [7] - Patient dosing for PIPE-791 in a Phase 1b trial for chronic pain began in March 2025, with results expected in the first half of 2026 [7] - PIPE-307 is currently in the VISTA Phase 2 proof-of-concept trial for RRMS, with top-line data expected in Q4 2025 [8] Strategic Focus - Contineum has prioritized its resources on PIPE-791 for IPF, postponing trials for PIPE-791 in PrMS and first-in-human studies for CTX-343 [9] - The company maintains strategic partnerships, particularly with Johnson & Johnson for PIPE-307, to support its development efforts [4][8] Future Outlook - Management did not provide explicit guidance for the next quarter or fiscal 2025 but indicated that the strong cash position supports planned development activities [12] - Investors will monitor the timing and outcomes of clinical milestones for PIPE-791 and PIPE-307, as well as enrollment trends and potential partnership developments [13]

Industry Challenge - Clinical trial bottlenecks cause delays in new drug launches [1] AI Solution - AI-enabled technology aims to accelerate the clinical trial process [1] Company Focus - Two cancer doctors developed the AI solution [1]

Summary of Capricor Therapeutics (CAPR) Update Call Company Overview - **Company**: Capricor Therapeutics - **Focus**: Development of Daramyacel, a cell therapy candidate for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) Key Points Regulatory Update - Capricor received a Complete Response Letter (CRL) from the FDA regarding the Biologics License Application (BLA) for Daramyacel, indicating that the application does not meet the requirements for substantial evidence of effectiveness and requires more clinical data [4][5] - The review clock has been stopped until a complete response is submitted, and Capricor does not believe a new BLA will be required [5][41] - Capricor is preparing a written response to the CRL and has initiated engagement with the FDA for further discussions [5][10] Clinical Data and Efficacy - The BLA was supported by data from the HOPE-two trial, which demonstrated statistically significant benefits in cardiac function [7][8] - The HOPE-two open label extension study showed significant results in left ventricular ejection fraction with a p-value of 0.0079 [9] - Ongoing Phase III HOPE-three clinical trial is seen as a key opportunity to address the FDA's request for additional clinical data [10] - HOPE-three has completed a twelve-month evaluation of 104 DMD patients, with data expected in Q3 2025 [10] Manufacturing and Quality Control - Capricor is committed to resolving outstanding issues in the chemistry, manufacturing, and controls (CMC) section of the application [11] - The company believes that the issues raised by the FDA are resolvable in a timely manner [11] Industry Context - The FDA's decision is viewed as concerning, especially given the high unmet medical need in DMD and the innovative nature of the therapy [6][12] - Capricor emphasizes the importance of using long-term extension data and real-world evidence in evaluating therapies for rare diseases like DMD [12] Support from Medical Community - A group of leading DMD physician scientists submitted a letter to the FDA expressing support for the Daramyacel clinical program [13] - The letter highlighted their extensive experience in treating DMD and their belief in the efficacy and safety of Daramyacel [13] Future Outlook - Capricor remains committed to working collaboratively with the FDA and is optimistic about the potential for Daramyacel to receive approval [14][15] - The company aims to provide updates as they achieve clarity with the FDA regarding the path forward [69] Financial and Strategic Considerations - Capricor maintains its priority review status and is focused on addressing the FDA's concerns without needing a new BLA [48] - The partnership with NS Pharma remains strong, with continued commitment to commercialization in the U.S. despite the CRL setback [58] Additional Insights - The FDA's cancellation of the Public Advisory Committee meeting without explanation raised concerns about the review process [6] - Capricor's approach to statistical analysis in clinical trials has been a point of contention with the FDA, particularly regarding the use of parametric versus nonparametric models [30][36] This summary encapsulates the critical updates and insights from the Capricor Therapeutics conference call, highlighting the company's current challenges and future strategies in the context of regulatory approval for Daramyacel.

Industry Challenge - Clinical trial bottlenecks cause delays in new drug launches [1] AI Solution - AI-enabled technology aims to accelerate the clinical trial process [1] Company Focus - Two cancer doctors developed the AI solution [1]

Industry Bottleneck - Clinical trial bottlenecks cause delays in new drug launches [1] - AI-enabled technology aims to accelerate the drug development process [1] Technological Innovation - Two cancer doctors developed AI-enabled tech to address clinical trial inefficiencies [1]