On May 15, 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 first quarter ended March 31, 2020 and provided a corporate update (Press release, Proteostasis Therapeutics, MAY 15, 2020, View Source [SID1234558185]).
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"The COVID-19 pandemic exacerbates the needs and anxieties of the CF community, and has further intensified our dedication to bringing more treatment choices to patients with CF," said Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics. "We are utilizing traditional and novel study approaches to develop our new, proprietary CFTR modulators – posenacaftor, dirocaftor and nesolicaftor – for rare and common CF mutations. In recent months, we have met with regulators in Europe to advance our goal of initiating the first large, personalized medicine study in CF: CHOICES. Employing theratyping, this study will use a laboratory assay to match our CFTR modulators to individual CF patients regardless of their CFTR genotype. This has the potential to increase access to treatment options and optimize the risk-benefit and cost-effectiveness for patients treated with CFTR modulators."
With the global spread of the ongoing COVID-19 pandemic in the first quarter of 2020, the Company implemented business continuity plans designed to address and mitigate the impact of the COVID-19 pandemic on its employees and its business, including clinical trials, supply chains and third-party providers. The Company continues to closely monitor the COVID-19 situation as it evolves its business continuity plans and response strategy.
First Quarter and Recent Highlights
In April of this year, Proteostasis announced it received a High Strategic Fit score from the Clinical Trials Network (CTN) for the HIT-CF – CHOICES protocol. The CHOICES protocol was assessed and scored by CF medical experts, experienced study coordinators, statisticians and trained patient reviewers across multiple domains and it has received the maximum rating in the domain of ‘Fit with ECFS Strategic Priorities’. The European Cystic Fibrosis Society formed this Clinical Trial Network (ECFS-CTN) to enhance clinical research across 58 participating sites in 17 countries. Participating sites conduct only those CF trials that have been reviewed and accepted after the ECFS-CTN protocol review process.
Proteostasis also announced in April that it received Scientific Advice from the Dutch Medicines Evaluation Board (MEB) on the CHOICES program for the treatment of people living with CF. The Company and the MEB discussed the development plan for PTI’s triple combination of dirocaftor, posenacaftor and nesolicaftor in the Netherlands and across Europe. The MEB expressed support for PTI’s personalized medicine approach and its goal of delivering effective medicines to patients who currently have no treatment options. The MEB also supported the expansion of the Company’s personalized medicine approach in more common genotypes, including F508del homozygous and heterozygous patients.
In February of this year, Proteostasis announced the completion of enrollment of 502 patients with CF in the HIT-CF Europe project, a research project which aims to pave the path to personalized medicine in CF. HIT-CF is leading a European-based initiative that is conducting confirmatory trials to assess the predictability of the organoid assay for clinical benefit, such as the CHOICES study (Crossover trial based on Human Organoid Individual response in CF – Efficacy Study). Proteostasis’ drug combinations will be tested first in an ex vivo study. Then, responders and non-responders will be selected for the CHOICES study. Dosing in CHOICES is expected to begin in the second half of 2020, with preliminary clinical data anticipated to be reported in early 2021. We do not anticipate any material changes in these timelines due to COVID -19, but continue to monitor its potential impact on these activities.
In January of this year, Proteostasis announced a regulatory update following the completion of a scientific advice meeting with the Medicines and Healthcare Products Regulatory Agency in the United Kingdom (MHRA) that outlined a path forward for the initiation and execution of our Phase 3 program, including establishment of a common safety database to support the safety profile of the proprietary combination, and the potential filing of a Marketing Authorization Application for posenacaftor, dirocaftor and nesolicaftor. The Company announced that it will continue to seek additional advice from other major regulatory agencies throughout 2020.
First Quarter 2020 Financial Results
Proteostasis reported a net loss of approximately $9.9 million for the three months ended March 31, 2020, as compared to a net loss of $14.4 million for the same period in the prior year.
The Company recorded no revenue in the three months ended March 31, 2020, as compared to revenue of $5 million for the same period in the prior year.
Research and development expenses for the three months ended March 31, 2020 were $6.5 million, as compared to $16.1 million for the same period in the prior year. The decrease in research and development expenses for the three months ended March 31, 2020 was primarily due to a decrease in clinical-related activities.
General and administrative expenses for the three months ended March 31, 2020 were $3.6 million, as compared to $3.9 million for the same period in the prior year. The decrease in general and administrative expenses for three months ended March 31, 2020 was due primarily to a decrease in professional fees.
Cash, cash equivalents and short-term investments totaled $57.1 million as of March 31, 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.