Cyclerion Therapeutics, Inc. · NASDAQ:CYCN
Cyclerion Therapeutics — Corporate Update (October 2019)
17 slides from Cyclerion Therapeutics's corporate update, Oct 30, 2019
Slide 1: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Our forward-looking statements are based on current beliefs and expectations of
Slide 2: Praliciguat DN and HFpEF phase 2 study results
Praliciguat DN and HFpEF phase 2 study results • HFpEF: terminated development • • • Missed primary endpoint with no observed improvement on HFpEF symptoms Evidence of pharmacological activity Generally well tolerated • Diabetic nephropathy: merits further investigation and discussion with potential
Slide 3: Diabetic Nephropathy (DN) Proof of Concept Study: Topline Results
Diabetic Nephropathy (DN) Proof of Concept Study: Topline Results
Slide 4: Praliciguat DN proof of concept study overview
Praliciguat DN proof of concept study overview Study Design • RCT designed to assess safety and tolerability of praliciguat and the effect on renal function in patients with DN on RAAS inhibitors Primary endpoints: Urine albumin creatinine ratio (UACR) and TEAEs Secondary endpoints: BP, HR, and meta
Slide 5: DN study results overview
DN study results overview •Treatment was associated with improvements in several vascular and metabolic parameters including blood pressure, HbA1c and LDL cholesterol levels. •Praliciguat was generally well tolerated. Most common AEs were dizziness, diarrhea and constipation. Discontinuations due to
Slide 6: DN study: change in UACR over weeks
DN study: change in UACR over 12 weeks Placebo 20 mg praliciguat 40 mg praliciguat 20 0 -20 -40 (90 % CIs by dose & timepoint) 0 1 4 Weeks of 8 Treatment 12 % Change from Baseline
Slide 7: DN study: data from one site inconsistent with overall study population
DN study: data from one site inconsistent with overall study population • During statistical validation, data from one clinical trial site (00A) were found to be inconsistent with those of the overall study population At site 00A, a significantly greater percentage of patients assigned to the pralic
Slide 8: DN study: comparison of UACR change over time with and without site 00A
DN study: comparison of UACR change over time with and site 00A ITT Population without Without Site 00A 20 20 0 0 -20 -20 -40 -40 0 1 4 8 12 0 1 4 8 12 Weeks of Treatment Weeks of Treatment Placebo 20 mg praliciguat 40 mg praliciguat 9 % Change from Baseline % Change from Baseline
Slide 9: DN study: primary analysis and week data with and without 00A site
DN study: primary analysis and week 12 data with and without 00A site * Nominal p-value; not adjusted for multiplicity 10 Outcome Variable: UACR ITT (n=156) Without Site 00A (n=133) PBO PRL Combined PBO PRL Combined Average of Weeks 8 and 12 Geometric Mean % Change (90% CI) -14.8% (-27, +0.4) -27.8%
Slide 10: Praliciguat in diabetic nephropathy: next steps
Praliciguat in diabetic nephropathy: next steps • Based on these results, we believe praliciguat warrants further investigation as a potential treatment to improve kidney function and cardiometabolic parameters in diabetic nephropathy • We intend to pursue an out-license of praliciguat for late-stag
Slide 11: DN is a common and serious complication of diabetes
DN is a common and serious complication of diabetes leading to progressive loss of kidney function Diabetic Nephropathy: • • Affects up to 40% of diabetes patients Leads to end-stage renal disease (ESRD) requiring renal replacement therapy (dialysis or kidney transplant) Survival on dialysis is wors
Slide 12: Strategic focus
Strategic focus • • • • • Praliciguat diabetic nephropathy out licensing IW-6463 CNS (phase 1) topline results expected Q4 2019 Olinciguat sickle cell disease (phase 2) topline results expected mid-2020 Preclinical programs Reduce our monthly cash expenses by 25%; expect cash on hand to cover operat
Slide 13: Conclusion: Praliciguat DN and HFpEF phase 2 study results
Conclusion: Praliciguat DN and HFpEF phase 2 study results • HFpEF: terminated development • • • Missed primary endpoint with no observed improvement on HFpEF symptoms Evidence of pharmacological activity Generally well tolerated • Diabetic nephropathy: merits further investigation and discussion wi
Slide 14: Conclusion: Praliciguat DN and HFpEF phase 2 study results
Conclusion: Praliciguat DN and HFpEF phase 2 study results * HFpEF: terminated development * Missed primary endpoint with no observed improvement on HFpEF symptoms * Evidence of pharmacological activity * Generally well tolerated * Diabetic nephropathy: merits further investigation and discussion wi
Slide 15: Capacity-HFpEF Proof of Concept Study Topline Results
Capacity-HFpEF Proof of Concept Study Topline Results
Slide 16: CAPACITY HFpEF (C1973-204) Study Design and Topline Results
CAPACITY HFpEF (C1973-204) Study Design and Topline Results Study Design • • • Designed to assess safety and tolerability of praliciguat and the effects on peak exercise capacity in patients with HFpEF (EF 40%) Primary endpoints: change in peak VO2 (CPET) and TEAEs at 12 weeks n=196 randomized 1:1 p
Slide 17: CAPACITY HFpEF (C1973-204) Study Design and Topline Results
CAPACITY HFpEF (C1973-204) Study Design and Topline Results Study Design * Designed to assess safety and tolerability of praliciguat and the effects on peak exercise capacity in patients with HFpEF (EF > 40%) + Primary endpoints: change in peak VO2 (CPET) and TEAEs at 12 weeks + n=196 randomized 2:1
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