Additional Relevant Details Following is a schedule of the Company’s presentations at NACFC, including links to the abstracts:Title: Phase 1 Initial Results Evaluating Safety, Tolerability, PK and Biomarker Data Using PTI-428, a Novel CFTR Modulator, in Patients with Cystic Fibrosis
Time/Date: Saturday, Oct. 29, 10:30-11:50 a.m. ET
Description: Preliminary data will be presented from the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 trials of PTI-428 – a novel genotype-agnostic amplifier – including safety, tolerability, pharmacokinetics (PK) and biomarker evaluation.Title: A Novel Modulator of CFTR Chloride Ion Mobility with a Distinct Mutation-Specific Profile to Existing CFTR Modulators
Time/Date: Thursday, Oct. 27, 11:15 a.m.-1:45 p.m.
Description: Proteostasis Therapeutics scientists will report a new class of CFTR modulators with characteristics distinct from known potentiators, correctors and amplifiers, which may represent a novel mechanism for improving mutant CFTR function. Investigation of these modulators shows that on F508del- CFTR, they work cooperatively with known correctors and amplifiers, but do not synergize with known potentiators. In CFTR-mediated chloride transport assays, these compounds demonstrate a mutation-dependent efficacy that is distinct from that of known CFTR potentiators.Title: Novel CFTR Modulator Combination of Amplifier, Corrector and Potentiator Provides Advantages Over Two Corrector-Based Combinations
Time/Date: Thursday, Oct. 27, 11:15 a.m.-1:45 p.m.
Description: Building on the foundation of the novel amplifier class of CFTR modulator, Proteostasis Therapeutics is developing a novel triple-combination therapy using CFTR amplifiers, correctors and potentiators. In human bronchial epithelial (HBE) cells electrophysiology measurements, the triple combination therapy restores the activity of mutant F508del-CFTR protein to near-normal CFTR activity. In addition, the amplifier-based triple combination provides potential advantages through its ability to modulate non-F508del CFTR and by overcoming substrate limitations for correctors and potentiators. Based on the data generated in the HBE cells, the combined use of these three molecules has the potential to restore mutant CFTR function in CF patients homozygous for the F508del mutation to clinically beneficial levels.