On August 6, 2020 Proteostasis Therapeutics, Inc. (Nasdaq: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF), reported financial results for the second quarter ended June 30, 2020 (Press release, Proteostasis Therapeutics, AUG 6, 2020, View Source [SID1234563204]).
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"The COVID-19 pandemic has highlighted the unmet needs of the CF community and reaffirmed our commitment to providing more treatment choices for people living with CF," said Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics. "Our team continues to work tirelessly to advance our mission and we anticipate announcing data from the ex vivo portion of the CHOICES development program in the fourth quarter of 2020, and plan to move into the clinical portion by year-end."
The CHOICES (Crossover trial based on Human Organoid Individual response in CF – Efficacy Study) development program is based on testing PTI drug combinations in an ex vivo study and, subsequently, in a clinical trial to assess the predictability of the organoid assay for clinical outcomes. The ex vivo portion of the study will quantify the functional response of patient-derived organoids to PTI investigational agents dirocaftor, posenacaftor and nesolicaftor. Enrollment of 502 patients with CF in the ex vivo portion of the study is now complete and the Company expects ex vivo data in the fourth quarter of 2020. The dataset will enable targeted enrollment into the clinical portion of the CHOICES trial of subjects with rare Cystic Fibrosis transmembrane conductance regulator (CFTR) mutations based on their individual ex vivo response. The clinical portion of the trial is expected to begin by the end of 2020. PTI and its HIT-CF partners are currently in the process of activating approximately 30 clinical sites in Europe to support the CHOICES execution. The results from the CHOICES clinical trial may serve as the basis for a potential Marketing Authorization Application with the European Medicines Agency (EMA) in 2021 through a novel regulatory pathway.
Recent Highlights
In June of this year, Proteostasis announced results from in vitro studies evaluating the use of PTI-129 as a potential treatment for COVID-19. PTI-129 is a pre-clinical, once-daily, oral small molecule identified through our Disease Relevant Translation (DRT) platform and originally designed to treat protein misfolding disorders involving the unfolded protein response (UPR). In in vitro studies conducted at Calibr, the drug discovery division of Scripps Research, PTI-129 demonstrated the potential to reduce viral protein production in host cells by activating the adaptive branches of the UPR pathway and reducing the levels of misfolded cellular proteins.
Second Quarter 2020 Financial Results
Proteostasis reported a net loss of approximately $8.9 million for the three months ended June 30, 2020, as compared to a net loss of $20.0 million for the same period in the prior year.
The Company recorded no revenue in the three months ended June 30, 2020 and 2019.
Research and development expenses for the three months ended June 30, 2020 were $4.6 million, as compared to $16.9 million for the same period in the prior year. The decrease in research and development expenses for the three months ended June 30, 2020 was primarily due to a decrease in clinical-related activities and related work as the CF studies progressed to data read out in late 2019.
General and administrative expenses for the three months ended June 30, 2020 were $4.4 million, as compared to $3.7 million for the same period in the prior year. The increase in general and administrative expenses for three months ended June 30, 2020 was due primarily to an increase in professional fees supporting financing activities.
Cash, cash equivalents and short-term investments totaled $48.9 million as of June 30, 2020, compared to $69.5 million as of December 31, 2019. The Company believes that its existing cash, cash equivalents and short-term investments are sufficient to fund operations into the second half of 2021. However, additional funding will be necessary to advance the Company’s proprietary combination therapy candidates through regulatory approval and into commercialization, if approved.