Preliminary Data from PTI-428 + Orkambi 28 Day Clinical Study on Track for Q4 2017
Initial Data from PTI-801 + Orkambi 14 Day Clinical Trial Anticipated in Q4 2017
Initial Data from PTI-808 SAD and MAD HV 7 day Study Anticipated in Q4 2017
Initial Data from PTI-808 + PTI-801 + PTI-428 coadministration HV 7 day Study Anticipated in Q4 2017
Dual Combination CF Clinical Study of PTI-801 + PTI-808 Expected to Initiate in Q4 2017
Preliminary Data from PTI-428 + Kalydeco 14 Day Clinical Study Anticipated in Q1 2018
Triple Combination CF Study of PTI-428 + PTI-801 + PTI-808 Planned to Initiate in 1H 2018
CAMBRIDGE, Mass., Nov. 14, 2017 (GLOBE NEWSWIRE) — Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced third quarter 2017 results and provided an update on the Company’s product candidates.
“Next generation CF therapies based on combined use of CFTR modulators are fundamental to raising the bar of disease targeting treatment. The team at Proteostasis has worked determinedly to deliver three truly novel, differentiated CFTR modulators to the clinic, generating data on safety, tolerability, and PK of their combined use,” said Meenu Chhabra, President and CEO of Proteostasis Therapeutics. “Our portfolio of CFTR modulators includes PTI-801, a new generation corrector, PTI-428, a novel class of CFTR modulator termed amplifier, and PTI-808, a novel potentiator. Our CFTR modulators have shown synergy in vitro with other known and investigational CFTR modulators, creating a multiplicity of possible development pathways, including add-on therapies to marketed CFTR modulators and proprietary double and triple combinations. Notably, in vitro data in patient cells showed that the combination of PTI-801 and PTI-808 restored CFTR protein activity to levels beyond those observed in cells from healthy carriers. Given the potential therapeutic benefit of multiple combination options and our commitment to next generation modulators, our combination development plan includes a PTI-801 and PTI-808 doublet and a PTI-808, PTI-801, and PTI-428 triplet. This effort enables us to both diversify our combination strategy and understand the value potential of each component within our portfolio. We look forward to initiating the dual combination study in CF patients in the fourth quarter of 2017, and a triple combination study in first half of 2018.”
PTI-428 – Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Amplifier
Proteostasis is conducting a 28-day Phase 2 clinical trial for PTI-428, its CFTR amplifier, which is dosing CF patients who receive PTI-428 or placebo for 28 days in addition to Orkambi® as their background therapy. The Company has completed enrollment and expects preliminary data from this cohort in the fourth quarter of 2017. The Company is also conducting a 14 day study of patients on Kalydeco® to receive PTI-428 or placebo for 14 days. The study has commenced dosing and Proteostasis currently intends to report preliminary data in the first quarter of 2018.
PTI-801 – New Generation CFTR Corrector; PTI-808 – Novel CFTR Potentiator
PTI-801 is a new generation CFTR corrector that was internally discovered in a high-throughput screen based on its demonstrated in vitro enhancement of CFTR function and synergy with CFTR amplifiers. The Company is conducting a 14-day Phase 1 study of PTI-801 and is currently enrolling and dosing CF patients on Orkambi® background therapy. Proteostasis expects to report initial data from this study in the fourth quarter of 2017.
Proteostasis is also conducting a Phase 1 study of PTI-808, its CFTR potentiator. Proteostasis expects to report initial safety, tolerability and PK data in healthy volunteers in the fourth quarter of 2017.
PTI Combination studies in Healthy Volunteer and CF Patients
Proteostasis is now enrolling healthy volunteers in safety, tolerability and PK studies in dual and triple combinations of its proprietary CFTR modulators, with preliminary data expected in the fourth quarter of 2017. The first combination study in CF patients will investigate the coadministration of PTI-801 and PTI-808 and is planned to start in the fourth quarter of 2017, to be followed by the initiation of a triple combination study of PTI-808, PTI-801, and PTI-428 in the first half of 2018.
Third Quarter 2017 Financial Results
Three Months Ended September 30, 2017
Revenue was $1.6 million for the three months ended September 30, 2017, compared to $1.7 million for the three months ended September 30, 2016. The decrease of $0.1 million is the result of a reduction of $0.7 million of revenue recognized under the Company’s collaboration with Biogen, which terminated in the fourth quarter of 2016. This decrease was partially offset by an increase of $0.6 million of revenue recognized under the Company’s agreement with Astellas in the three months ended September 30, 2017, which is primarily a result of payments under the agreement being recognized as revenue over the research term, with a cumulative catch-up for the elapsed portion of the research term.
Research and development expenses were $12.9 million for the three months ended September 30, 2017, compared to $9.2 million for the three months ended September 30, 2016. The increase of $3.7 million was primarily due to an increase of $1.4 million in activities supporting our CF program as we continue to advance our candidates in the clinic. During the three months ended September 30, 2017, costs incurred on PTI-801 and PTI-808 accounted for a combined increase of $5.4 million, or $2.7 million each, largely related to increased manufacturing, preclinical and clinical trial expenses as we transition to Phase 1 clinical trials. The increase was partially offset by a $3.3 million decrease in spending on PTI-428 as compared to the same period last year. Additionally, there was an increase of $1.2 million in personnel-related costs during the three months ended September 30, 2017, as compared to September 30, 2016, including $0.3 million in employee stock-based compensation expense, primarily driven by an overall increase in headcount to support the development of our CF product pipeline.
General and administrative expenses were $2.7 million for the three months ended September 30, 2017, compared to $3.3 million for the three months ended September 30, 2016. The decrease of $0.6 million in general and administrative expenses was primarily due to a decrease of $0.4 million in professional fees and a $0.4 million decrease in facility costs, partially offset by an increase of $0.3 million in personnel-related costs, including $0.2 million of employee stock-based compensation.