Slide 1: Study 0170 (REDWOOD) Ampreloxetine Phase 3 Results
© Theravance Biopharma (Unpublished Work). All rights reserved. THERAVANCE BIOPHARMA ® , THERAVANCE ® , the Cross/Star logo and MEDICINES THAT MAKE A DIFFERENCE ® are registered trademarks of the Theravance Biopharma group of companies (in the U.S. and certain other countries). All third - party tra
Slide 2: Forward-looking statements
Under the safe harbor provisions of the U . S . Private Securities Litigation Reform Act of 1995 , the company cautions investors that any forward - looking statements or projections made by the company are subject to risks and uncertainties that may cause actual results to differ materially from th
Slide 3: Phase 3 program overview
Phase 3 program overview 3 Study 0169: 4 weeks Randomized, double - blind, placebo controlled, parallel group Phase 3 Registrational Program Extension Study All patients who complete 0169 may enroll into 0170 All patients who complete 0170 may enroll into 0171 + de novo Study 0170*: 22 weeks Randomi
Slide 4: Study 0170 design and primary endpoint
Primary Endpoint: Worsening of OSHA#1 and PGI - S (Treatment Failure) NCT03829657 *n=154 planned; n =number of subjects enrolled in the randomized withdrawal period. OHSA, Orthostatic Hypotension Symptom Assessment; PGI - S, Patient Global Impression of Severity; RWD, randomized withdrawal design. 4
Slide 5: Pre-specified subgroup analyses: patient reported outcomes
OHDAS LS Mean Difference (95% CI) Overall (n=112) - 0.0 ( - 0.6, 0.6) MSA (n=36) - 0.8 ( - 2.1, 0.4) PD (n=58) 0.1 ( - 0.6, 0.9) PAF (n=18) 1.4 ( - 0.3, 3.0 ) Odds Ratio (95% CI) Overall (n=128 ) 0.60 (0.27, 1.29) MSA (n=40) 0.28 (0.05, 1.22) PD (n=68) 0.79 (0.26, 2.30) PAF (n=20) 0.89 (0.09, 8.41)
Slide 6: OHQ questionnaire composite scores and individual items for MSA patients
LS Mean (95% CI) OHSA Symptom composite score - 1.6 ( - 2.7, - 0.5) Dizziness - 1.5 ( - 3.2, 0.2) Vision - 1.7 ( - 3.2, - 0.3) Weakness - 0.7 ( - 2.3, 0.9) Fatigue - 1.5 ( - 3.1, 0.1) Trouble concentrating - 1.8 ( - 3.3, - 0.4) Head/neck discomfort - 2.2 ( - 3.7, - 0.7) OHDAS Daily activities compos
Slide 7: Longitudinal analysis of OHSA composite score for MSA patients
Longitudinal analysis of OHSA composite score for MSA patients 7 33 34 33 33 33 33 31 33 31 32 64 60 51 44 42 20 20 19 17 20 19 18 20 0 0 4 0 4 8 12 16 18 20 22 2 4 6 8 0169 4 - week double - blind 0170 6 - week RW period 0170 16 - week open label period Placebo, n= Ampreloxetine, n= Week OHSA Compo
Slide 8: Overall safety summary
• SAEs: 1 of 2 events was reported as study drug related • 53% TEAEs (n=105), majority mild to moderate • 8% SAEs (n=16), none reported as related to ampreloxetine • 5 deaths • 3 AEs of special interest: ‒ Supine hypertension (n=1) ‒ Myocardial infarction (n=1) ‒ Atrial arrythmia (n=1) • SAEs: 2 of
Slide 9: Overall ampreloxetine summary
Overall ampreloxetine summary ‣ Novel chemical entity, discovered and developed at Theravance Biopharma ‣ Potent and high - affinity norepinephrine reuptake inhibitor ‣ Orally bioavailable with QD dosing ‣ Mechanism of action consistent with durability of effect ‣ Patent protection until 2037 in US