Cyclerion Therapeutics, Inc. · NASDAQ:CYCN
Cyclerion Therapeutics — J.P. Morgan Healthcare Conference 2020
16 slides from Cyclerion Therapeutics's conference presentation, Jan 13, 2020
Slide 1: J.P. Morgan Healthcare Conference
J.P. Morgan Healthcare Conference January 13, 2020 Peter Hecht, CEO
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements. Any statements contained in this presentation that are not historical facts may be deemed to be forward looking statements. Words such as “anticipate,” “believe,” “potential,” “expect,” “may,” “will,” “should,” “could,” “pl
Slide 3: Priorities for 2020
Priorities for 2020 1 2 3 DN praliciguat partnering: out-license discussions based on promising phase 2 SCD olinciguat phase 2 completion: potential for raising standard four therapeutic domains of care across CNS IW-6463 advancement: advancement from successful phase 1 in healthy volunteers to ongo
Slide 4: Praliciguat in diabetic nephropathy: out-license discussions based on promising phase 2
1 Data Praliciguat in diabetic nephropathy (DN): out-license discussions based on promising phase 2 Out-license discussions underway support further development • UACR reductions on top of standard of care •20%1 placebo-adjusted (p=0.03032) • 24%1 absolute change from baseline • • • reductions in bl
Slide 5: Phase 2 showed promising improvement in UACR
Phase 2 showed promising improvement in UACR 20 % UACR change from baseline LS mean (90% CI) change UACR in (mg/g) (mITT)1 0 -20 -40 (90 % CIs by dose & timepoint) 0 1 4 8 12 weeks of treatment 1. mITT: modified intent-to-treat population n=133 excludes data from site 00A where data inconsistencies
Slide 6: Olinciguat in sickle cell disease: potential for raising standard of care
2 Olinciguat in sickle cell disease (SCD): potential for raising standard of care across four therapeutic domains Dynamic mechanism - alone or complementing approved treatments Topline phase 2 results mid-2020 • • upstream + downstream pharmacological intervention impact potential in four clinical d
Slide 7: Olinciguat: upstream and downstream intervention in SCD
Olinciguat: upstream and downstream intervention in SCD Upstream • increased HbF leads to reduced proportion of sickled RBCs1 Increased hemolysis leads to reduced nitric oxide state sGC restores deficient nitric oxide signaling Downstream • • improved blood flow decreased vascular inflammation & cel
Slide 8: Potential to raise standard of care across four therapeutic domains
Potential to raise standard of care across four therapeutic domains improve daily symptoms reduce painful crises (VOC) • newly approved therapies each target a single clinical domain… …olinciguat has the potential to improve four daily symptoms and end-organ protection remain unaddressed, decreasing
Slide 9: Olinciguat phase 2 trial designed to support rapid advancement
DESIGN •double blind •global sites •4 dose levels •up to 88 patients aged 16 – 70 •12-week treatment in all SCD genotypes OBJECTIVES Olinciguat phase 2 trial designed to support rapid advancement assess safety and tolerability confirm PK profile in SCD patients development of the CYCN fit-for-purpos
Slide 10: IW-6463 in CNS: advancing development for serious neurodegenerative diseases
3 IW-6463 in CNS: advancing development for treatment of serious neurodegenerative diseases Ph 1 showed safety, target Additional clinical studies in 2020 to accelerate and de-risk program engagement, CNS exposure Preclinical evidence: stimulation of nitric oxide-cGMP pathway improves determinants o
Slide 11: IW-6463 potential to restore nitric oxide-cGMP signaling
IW-6463 potential to restore Nitric oxide insufficiency leads to nitric oxide-cGMP signaling Improved brain health • • • • • neuroinflammation and neurodegeneration impaired neurovascular blood flow decreased inflammation increase blood flow neuroprotection and enhanced cognition Figure adapted from
Slide 12: Positive phase 1 IW-6463 results support further development
Positive phase 1 IW-6463 results support further development Newly released—completed December 2019 Phase 1 study design Insights • CNS drug presence at levels expected to be pharmacologically active QD dosing, linear and predictable PK may be taken with or without food evidence of target engagement
Slide 13: Clinical direction: accelerate and de-risk into high value CNS indications
Clinical direction: accelerate and de-risk into high value 2019 CNS indications 2020 2021 translational pharmacology study in elderly subjects safety, tolerability, PK, biomarkers: CBF, cGMP completed December initiated January 2020 additional clinical studies in neurodegenerative disorders 2019 ong
Slide 14: Cyclerion 2020
Cyclerion 2020 • • catalysts across programs partnering clinical trials ~$102M cash1 and reduced our priorities into Q2 2021 burn support Team, talent and intensity to deliver 1. Preliminary, unaudited unrestricted cash, cash equivalents and restricted cash balance as of Dec 31, 2019 14
Slide 15: Priorities for 2020
Priorities for 2020 1 2 3 DN praliciguat partnering: out-license discussions based on promising phase 2 SCD olinciguat phase 2 completion: potential for raising standard four therapeutic domains of care across CNS IW-6463 advancement: advancement from successful phase 1 in healthy volunteers to ongo
Slide 16: J.P. Morgan Healthcare Conference
J.P. Morgan Healthcare Conference January 13, 2020 Peter Hecht, CEO
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