Summary of Syntara Investor Webinar Company Overview - **Company**: Syntara - **Product**: Amsulostat - **Indication**: Myelofibrosis Key Points from the Webinar FDA Guidance and Clinical Development - Syntara received FDA guidance on the clinical development of amsulostat, which was more conservative than expected, suggesting a phased approach rather than a direct transition to a pivotal study [3][6][11] - The FDA's concerns centered around the lack of control data in the ongoing phase two study, which complicates the assessment of safety and efficacy [7][11] - The proposed new pathway involves conducting a phase two b study with a control group, followed by a phase three study, which is expected to be more efficient and potentially smaller in size [8][9][10][11] - The estimated cost for the phase two b study is around $25 million, significantly lower than the previously estimated $80 million for the phase three study [20][21] Financial Position and Funding - Syntara's cash position is projected to last until the first half of 2027, extended due to the postponement of phase three activities [14][44] - The company plans to explore two funding avenues: partnering the drug and raising capital [12][22][26] - Previous successful partnerships for drugs in similar stages indicate a strong market appetite for amsulostat, despite the shift to a phase two b study [24][36] Clinical Trial Design and Patient Recruitment - The phase two b study will involve approximately 90 patients, with a focus on understanding the baseline characteristics of suboptimal patients [29][30] - The endpoints for the phase two b study will include symptom score and spleen volume [33] - The FDA has emphasized the need for clear definitions of suboptimal patients, which will be refined during the trial design process [35][46] Pipeline and Future Studies - Syntara has multiple ongoing studies, including those for myelodysplastic syndrome and skin scarring, which are funded through non-dilutive cash sources [16][39] - Results from these studies are expected to provide additional safety and efficacy data that could support the FDA review process for amsulostat [16][17] Market Reaction and Company Outlook - The market reaction to the FDA guidance was negative, reflecting disappointment over the slower development pathway [56][57] - Despite the delay, the company maintains that the clinical development pathway remains solid and recognizes the potential value of amsulostat for patients with myelofibrosis [57] Conclusion - Syntara is positioned to navigate the revised clinical development pathway for amsulostat while leveraging its other assets to maintain a robust pipeline and financial stability [57][58]