New drugs take too long to get to market because of clinical trial bottlenecks. Two cancer doctors built AI-enabled tech to speed up the process. https://t.co/XO01YCVQ41 https://t.co/XO01YCVQ41 ...

How does removing the recommendation though increase choice, increase the patients ability to come to a decision with doctors since so many insurance plans and programs depend on ASIP and CDC recommendations in order to provide the the nocost lowcost vaccines. Isn't this removing choice effectively from patients. Now the promise of the Secretary Kennedy is to uh let people know that vaccines will be available if people want them. you will be able to get a vaccine if you want them.So, that's a decision betwe ...

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.

Ultragenyx Pharmaceutical Inc. RARE and Mereo BioPharma Group plc MREO stocks are plunging on Thursday. What Happened? The companies on Wednesday announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis around the end of the year, consistent with the original plan. OI, also known as brittle bone disease, is a group of genetic disorders that ...

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 Solution - AI-enabled technology aims to accelerate the clinical trial process [1] - Two cancer doctors developed the AI solution [1]

Rural Healthcare Challenges - Rural cancer patients face later diagnoses, increased uninsurance/underinsurance, and worse outcomes due to their location [8] - The "rural cancer gap" shows widening mortality rate disparities between rural and urban areas since 1999 [8] - Nearly 20% of Americans live in rural areas, but only 3% of oncologists practice there [9] - Kansas rural provider to patient ratio exceeds 500 to 1, compared to urban areas closer to 80 to 1 [12] - 140 hospitals have closed in the past 10 years, with a projection of 600 more at imminent risk [13] - Cancer care accounts for 30-40% of hospital revenue nationwide, but is increasingly diverted to urban centers [13][14] - Hospitals are closing at a rate of two per month [15] - Only 2% of clinical trials are offered in rural areas, creating "clinical trial deserts" [15] Proposed Solutions - Expand Medicaid, support rural providers with loan forgiveness and flexible training, and decentralize clinical trials via mobile units and telemedicine [17] - Change the narrative around rural cancer patients and value them [18] - Implement technology innovation, such as hub and spoke models and empowering community navigators with technology [19] - The rural oncology home model partners patients with local providers and leverages telehealth to access specialized oncologists [20]

Core Insights - NeOnc Technologies Holdings, Inc. has signed a non-binding term sheet with Quazar Investment to establish a new investment and clinical platform in the MENA region [1][2] - The partnership aims to leverage the UAE's clinical trial infrastructure to advance NeOnc's late-stage drug candidates [3][7] Investment and Structure - The term sheet outlines the formation of NuroMENA Holdings Ltd, which will oversee the establishment of NuroCure, an Abu Dhabi-based subsidiary responsible for clinical trials [2][5] - A proposed equity investment of $50 million is contingent upon NeOnc meeting specific conditions within 120 days [4][8] Clinical Development - NuroCure will initiate clinical trials for NEO100 and NEO212, targeting aggressive brain cancers such as Diffuse Intrinsic Pontine Glioma and glioblastoma multiforme [7][9] - The partnership is positioned to enhance NeOnc's clinical programs through collaboration with Cleveland Clinic Abu Dhabi [3][10] Financial Strategy - Quazar will lead a capital formation round of up to $50 million, with 70% allocated for acquiring NeOnc common stock and 30% for clinical trial and infrastructure development [8][10] - The investment strategy includes executing a Sub-License Agreement for NEO100 and NEO212, facilitating clinical efforts in the region [5][11] Company Background - NeOnc Technologies is focused on developing therapeutics for central nervous system cancers, with a robust patent portfolio extending to 2038 [11] - The company's drug candidates, NEO100 and NEO212, are currently in Phase II clinical trials under FDA Fast-Track status [11]

Seralutinib Partnership with Chiesi - Chiesi will provide a $160 million immediate development reimbursement to Gossamer[13] - Gossamer and Chiesi will split US profits 50/50, with mid-to-high teens royalties to Gossamer ex-US[13] - Regulatory milestones could reach up to $146 million, and sales milestones up to $180 million[13] - R&D costs will be split 50/50 worldwide, with Gossamer leading global development and US commercialization of PAH & PH-ILD[13] - Gossamer has a pro forma cash position of approximately $396 million[20, 38] Seralutinib in PAH - Seralutinib is in an ongoing registrational Phase 3 trial for PAH, with topline results expected in Q4 2025[13, 24, 26] - The PROSERA Phase 3 study is a double-blind, placebo-controlled trial with 175 patients per arm[26] - The US has approximately 30,000 to 50,000 PAH patients, with a 5-year survival rate of 57%[13, 22, 23, 35] Seralutinib in PH-ILD - The US has approximately 60,000 to 100,000 PH-ILD patients[13, 29, 35] - The median 5-year survival for PH-ILD patients is 23%[13, 35] - A Phase 3 study in PH-ILD is expected to begin in mid-2025[13, 20] - Only one therapy is approved for PH-ILD in the US[29, 30, 35]

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]