KemPharm CEO says positioned for ‘continued growth’

KemPharm announced that Richard Pascoe, CEO of KemPharm, has issued a Letter to Shareholders. The letter read in part, "This is a very exciting time in the growth of KemPharm and our strategic transformation into a leading rare disease company. The numerous accomplishments in 2022 and prior have set the stage for us to achieve multiple milestones during 2023 as we advance our arimoclomol and KP1077 programs towards key inflection points that have the potential to position us for success in fulfilling our mission to become a commercially-focused rare disease company. Since the beginning of the year, we have made several enhancements to the leadership team that are squarely focused on enhancing shareholder value as we seek to secure regulatory approval for our pipeline assets, build top-tier commercial capabilities, and enhance our pipeline through targeted business development transactions. With the senior team firmly in place and focused on achieving our key objectives, I am confident that we will deliver value in 2023 and beyond…Our goals as a company, and as a leadership team, are two-fold. First, we seek to improve the lives of patients by developing novel therapeutics for rare diseases where there are no treatments, or where current treatment options are insufficient. Second, we must drive value for our shareholders. I believe these are two sides of the same coin. If we are successful in these endeavors, which I firmly believe we will be, and we do so by carefully managing our resources, then we will have delivered on our mission to provide value to both patients and shareholders…For 2023, KemPharm is focused on four key areas for arimoclomol: The resubmission of the NDA to the FDA targeted to be filed as early as the third quarter of 2023; Supporting the continuation of the EAPs in the various countries where it has been available; Identifying a regulatory path forward with the European Medicines Agency; and Building the commercial infrastructure needed to support an ultra-rare disease product…As announced in December 2022, KemPharm initiated the Phase 2 clinical trial investigating KP1077 as a treatment for idiopathic hypersomnia. This was a significant clinical milestone for KemPharm and provides a springboard for several value creating events during the year, including: Interim Phase 2 KP1077 IH efficacy and safety data as early as the third quarter of 2023 and full Phase 2 data by year end 2023; and Potential to advance KP1077 directly into phase 3 studies in narcolepsy as early as prior to year-end 2023 based on previous Phase 1 studies of serdexmethylphenidate, the sole pharmaceutical agent in KP1077, and learnings from the ongoing Phase 2 study in IH…We believe KemPharm is stronger today than it has been at any point since its inception. We possess a diverse product pipeline led by arimoclomol and KP1077, and supported by the ongoing commercialization of AZSTARYS by our partner, Corium, Inc. Supporting our strategic and pipeline development efforts is a strong financial foundation, bolstered by $102.9 million in cash, cash equivalents and long-term investments as of December 31, 2022. Unlike many development-stage biopharmaceutical companies, we believe our balance sheet provides the ability to pursue our strategic and product development objectives while also seeking external business development opportunities aligned with our strategic pivot to a fully integrated commercial company. Given this, and based on our current operating forecast, we expect available capital will allow us to pursue our development plans and extend our cash runway into 2026. In closing, we believe the numerous milestone opportunities anticipated for 2023 and beyond will position KemPharm for continued growth as we focus on bringing much-needed therapies to patients with rare diseases."