Clinical Trials and Pipeline Advancements - CAN-2409 combined with valacyclovir extended median survival to 20.6 months in NSCLC patients with disease progression despite ICI therapy, showing good safety and immune system activation [1] - The company is advancing its pipeline with ongoing trials for obesity treatments and other metabolic disorders [2] - QUILT 3.032 study showed a 71% complete response rate in BCG-unresponsive NMIBC, supporting submissions to the European Medicines Agency in Q4 2024 [3] - Certepetide, designed to enhance anti-cancer agent delivery, completed patient enrollment with results anticipated in 2025 [4] - The company plans to conduct an interim data analysis in Q1 2025 and submit findings to the FDA to discuss next steps in the clinical development program [5] - MAVERIC-2 trial will explore CardiolRx's effects in patients who stopped using IL-1 blockers, with an expected readout before the Phase 3 MAVERIC-3 trial begins [8] - The company is advancing clinical trials for VLS-01 and EMP-01 programs, with data readouts expected in the new year [6] - The Phase 2 trial of intranasal foralumab for non-active secondary progressive multiple sclerosis was expanded to prestigious US Medical Centers [12] - VLS-01, a buccal film formulation of DMT for treatment-resistant depression, is set to progress through its Phase 2 study with topline results expected around the end of the year [13] - ANKTIVA's clinical trials are expanding in NMIBC, prostate cancer, and NSCLC, with a broader market launch targeted for 2025 [14][15] - NV-387 is advancing into Phase 2 clinical trials for MPox, RSV, and influenza [16][31][32] - The ASCEND trial targeting metastatic pancreatic ductal adenocarcinoma is expected to deliver interim data in January [18] - A pediatric clinical trial for Anaphylm commenced in December 2024, following positive FDA feedback [19] - The Phase 2 trial of OK-101 for dry eye disease showed promising reductions in ocular pain and inflammation [21] - CAN-2409's Phase 3 trial in intermediate-to-high-risk localized prostate cancer met its primary endpoint, showing a statistically significant improvement in disease-free survival [25] - The SKNJCT-003 clinical study is evaluating Doxorubicin-containing microneedle array in treating BCC, with over 25% of the 60 patients already randomized [26] - VK2809 demonstrated significant reductions in liver fat, NASH resolution, and fibrosis improvement in the Phase 2b VOYAGE study [27] - EMP-01, an oral formulation of R-MDMA, is set to initiate a Phase 2 trial for social anxiety disorder, with results anticipated by the end of 2025 [30] - The Phase 2 CALLIPER trial for progressive multiple sclerosis is expected to deliver top-line data in April 2025 [45] - The Phase 2 ARCHER trial in acute myocarditis completed enrollment in September 2024, with results expected in early 2025 [39] Strategic Collaborations and Regulatory Submissions - The company entered a collaboration agreement with Swanielle to explore expanding the Phase 2 study into the Asia-Pacific region [11] - ANKTIVA's J-code will be effective in January 2025, facilitating wider patient access [15] - The company plans to submit ANKTIVA for approval in the United Kingdom and the European Union [15] - A New Drug Application for Anaphylm sublingual film will be submitted to the FDA, aiming to advance the first orally delivered epinephrine treatment for severe allergic reactions [35] - The company will expand the availability of Libervant buccal film, which has received FDA Orphan Drug Exclusivity for treating seizure clusters in pediatric patients [36] Key Therapeutic Areas and Innovations - Key trials include cancer immunotherapies, treatments for neurological conditions like multiple sclerosis and Alzheimer's, and novel approaches to metabolic diseases and chronic pain [9] - The company is advancing groundbreaking research into Alzheimer's disease, following the dosing of the first patient with moderate Alzheimer's [12] - NV-387's Phase 2 trial in MPox aims to address the high fatality rate of MPox Clade 1/1b, for which no effective treatments are currently available [31] - Vidofludimus calcium is being investigated for its neuroprotective and anti-inflammatory properties in progressive multiple sclerosis [33] - The CENDIFOX trial is evaluating the combination of certepetide with FOLFIRINOX-based therapies for pancreatic, colon, and appendiceal cancers [34] - CardiolRx is advancing into pivotal late-stage trials for recurrent pericarditis, with key milestones anticipated in 2025 [37] - The Phase 2 trial of OK-101 for neuropathic corneal pain aims to address a condition with no FDA-approved treatments [44]
2025 biotech outlook: Small-cap innovators tackling chronic and rare diseases