Drug Development and Designation - Kintara Therapeutics received Orphan Drug Designation from the FDA for VAL-083 for the treatment of DIPG, a rare childhood brain cancer [82]. - VAL-083 has been designated as an orphan drug by both the FDA and EMA for the treatment of gliomas, including GBM [87]. - Kintara is evaluating the potential combination of VAL-083 with PARP inhibitors for ovarian cancer, for which it has also received orphan drug designation [86]. - VAL-083 has demonstrated activity against various tumor types in prior Phase 1 and Phase 2 clinical studies sponsored by the NCI, leveraging data from over 40 studies [83]. - The GBM AGILE Study employs a cost-efficient, adaptive study design, allowing multiple therapies to be evaluated simultaneously [91]. - Kintara anticipates topline results from the GBM AGILE Study for VAL-083 around the end of Q4 2023 [83]. - The GBM AGILE Study has screened over 1,300 patients, with enrollment rates 3 to 4 times greater than traditional GBM studies [90]. - VAL-083 has shown activity against MGMT-unmethylated GBM cells resistant to TMZ and nitrosoureas, and retains high activity in p53 mutated NSCLC, ovarian cancer, and medulloblastoma [99]. - In clinical studies, REM-001 Therapy achieved a complete response in approximately 80% of evaluable tumor sites in CMBC patients [101]. - VAL-083 and REM-001 have received Fast Track Designations from the FDA for their respective indications [106]. Financial Performance and Strategy - As of December 31, 2022, cash and cash equivalents were 11.780 million on June 30, 2022 [113]. - For the three months ended December 31, 2022, the net loss was 5.893 million for the same period in 2021 [115]. - Research and development expenses for the six months ended December 31, 2022, were 7.695 million in 2021 [116]. - Total assets decreased from 9.917 million on December 31, 2022 [113]. - Research and development expenses decreased to 3,902 for the same period in 2021, a reduction of 47% [121]. - General and administrative expenses were 1,993 for the same period in 2021, a decrease of 28% [121]. - Net loss for the three months ended December 31, 2022, was 5,893 for the same period in 2021 [121]. - For the six months ended December 31, 2022, research and development expenses decreased to 7,695 in the same period in 2021, a decline of 32% [126]. - General and administrative expenses for the six months ended December 31, 2022, were 4,171 in the same period in 2021, a decrease of 30% [130]. - Net cash used in operating activities decreased to 10,177 for the same period in 2021, a reduction of 16% [132]. - The company reported an accumulated deficit of 4,874 as of December 31, 2022 [136]. - The company paused the REM-001 program to conserve cash for the development of VAL-083, indicating a strategic shift in resource allocation [124]. - Management is pursuing various financing alternatives to fund operations, including issuing new equity and entering strategic partnerships [137]. - The company has not generated any revenues to date and does not expect to achieve revenues until product candidates are commercialized or partnered [136]. - The company expects to finance future cash needs primarily through public or private equity and debt offerings, and/or strategic collaborations [139]. - If additional funding is not secured, the company may have to delay, reduce, or eliminate clinical trials or research and development programs [139]. Clinical Program Updates - The company paused the REM-001 program to conserve cash, expecting to save approximately $3.0 million through calendar 2023 [82]. - Avastin received full approval in the US, Canada, Australia, and Japan for recurrent GBM, but has a "black-box warning" for severe side effects [97]. - The median survival for GBM patients whose tumors progress after Avastin treatment is less than five months [98]. Accounting and Expense Recognition - The company recognizes compensation costs from stock-based awards over the service period based on fair value measurements [142]. - For the six months ended December 31, 2022, the company utilized the Black-Scholes model to estimate grant-date fair value for stock option awards [142]. - The company estimates expenses for clinical trials based on contracts with vendors and adjusts clinical expense recognition if actual results differ from estimates [143]. - There were no material adjustments to prior period estimates of accrued expenses for clinical trials for the six months ended December 31, 2022, and 2021 [143]. - The company does not have any off-balance sheet arrangements [144].
TuHURA Biosciences, Inc.(HURA) - 2023 Q2 - Quarterly Report