Insmed Incorporated · NASDAQ:INSM
Insmed — Technical Presentation (February 2021)
23 slides from Insmed's technical presentation, Feb 19, 2021
Slide 1: TPIP Phase 1 Topline Results
TPIP Phase 1 Topline Results February 19, 2021
Slide 2: Forward-looking statements
This presentation contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncert
Slide 3: Powered By Purpose
Our commitment to transforming the lives of people with rare diseases drives what we do. Powered By Purpose 3
Slide 4: TPIP: Potentially Developing a Superior Treatment Paradigm
Treprostinil Palmitil Inhalation Powder (TPIP)Potentially Developing a Superior Treatment Paradigm Prostanoids have long been considered the “gold standard” therapy for PAH; however, this class of drugs has also been plagued by challengesTreprostinil palmitil has demonstrated substantial promise for
Slide 5: Treprostinil Palmitil: Harnessing the Full Potential of the Prostanoid Pathway
Treprostinil PalmitilHarnessing the Full Potential of the Prostanoid Pathway TPIP could address the shortcomings of existing prostanoid therapies by potentially:Providing prolonged, localized pulmonary vasodilationOffering improved tolerability, with fewer prostanoid-related side effectsEnabling hig
Slide 6: TPIP: A Potentially Differentiated Prostanoid
TPIP* is a dry powder formulation of treprostinil palmitil, a prodrug of the prostacyclin vasodilator treprostinilTreprostinil palmitil was formerly referred to as INS1009Once delivered to the lung, treprostinil palmitil is hydrolyzed by endogenous esterases to active treprostinilThe inhalation devi
Slide 7: September 30, 2020 R&D Day
September 30, 2020 R&D DAY 7
Slide 8: Prolonged Lung Residence Translated to Sustained Efficacy with Low Systemic Concentrations
IN PRE-CLINICAL RAT STUDIES WITH TREPROSTINIL PALMITIL INHALED SOLUTION (TPIS), Prolonged lung residence Treprostinil Treprostinil palmitil Treprostinil Treprostinil palmitil Sustained hemodynamic effect* Substantially lower peak plasma concentrations F.G. Leifer et al. (2018) Drug Res 68: 605-614 T
Slide 9: Treprostinil Palmitil Exhibited Local Vasodilatory Activity in the Lung
Treprostinil Palmitil Exhibited Local Vasodilatory Activity in the Lung, Beyond that which can be Attributed to the Circulating Treprostinil LOCAL PHARMACODYNAMIC EFFECT: DOG Hypoxemia Model RAT Thromboxane Mimetic Model 9
Slide 10: TPIP at High Dose Showed Superior Effect in Sugen-Hypoxia Rat Model for PAH
Parameter value = (Value – Value of Normal) / (Value of Vehicle – Value of Normal) Fulton Index = weight ration Right Ventricle / (Left Ventricle + Septum)Pulmonary Pressure mean Pulmonary Arterial PressureObliteration % of non-obliterated vesselsWall thickness Small vessel wall thicknessMusculariza
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Slide 12: INS1009-102 Study Design
Multiple Dose Panel 1 225 µg QD×7 days 6 subjects Placebo QD×7 days 2 subjects Single Dose Panel 1 112.5 µg 6 subjects 225 µg 6 subjects 450 µg 6 subjects Single Dose Panel 2 675 µg 6 subjects Placebo 2 subjects Multiple Dose Panel 2 112.5 µg QD ×4 days 6 subjects 225 µg QD ×3 days Matching Placebo
Slide 13: SAD Cohorts: Treatment Emergent Adverse Events
SAD Cohorts: Treatment Emergent Adverse Events (TEAEs)TEAEs were Consistent with Inhaled Prostanoid and Mostly Mild 13 * Moderate TEAEs: 225 µg – 1 subject with nausea and vomiting; 675 µg – 2 subjects with hypotension, 1 subject with chest discomfort, low oxygen saturation and dyspneaIncludes all A
Slide 14: MAD Cohorts: Subject Disposition, TEAEs
14 * Moderate TEAEs: 225 µg – 1 subject with chest discomfort (subject discontinued day 2), 1 subject with throat irritation (day 2-7), 1 subject with syncope (day 1)Includes all AEs that occurred in more than two subjects, as well as others of interest MAD Cohorts: Subject Disposition, TEAEsLower T
Slide 15: INS1009-102 Study Plasma Treprostinil Concentrations
15 INS1009-102 Study Plasma Treprostinil Concentrations Dose proportional Cmax and AUC
Slide 16: TPIP showed substantially lower Cmax and longer half-life
16 TPIP showed substantially lower Cmax and longer half-lifeSupports the potential for improved tolerability, efficacy and convenience TPIP from Study INS1009-102, Tyvaso from Han Tyvaso from Han D, et al. Eur Respir J 2016 48: PA2403
Slide 17: TPIP showed substantially lower Cmax and longer half-life
17 TPIP showed substantially lower Cmax and longer half-lifeSupports the potential for improved tolerability, efficacy and convenience TPIP from Study INS1009-102, Tyvaso from Han Tyvaso from Han D, et al. Eur Respir J 2016 48: PA2403
Slide 18: Key Takeaways from TPIP Phase 1 Study
Key Takeaways from TPIP Phase 1 study Safety profile was generally well tolerated, AEs were mild and consistent with inhaled prostanoid 2 1 3 4 5 TPIP showed substantially lower Cmax and longer half-life than that of Tyvaso PK supports development of TPIP with once daily dosing Tolerability was impr
Slide 19: Inhaled Treprostinil in Severe Pulmonary Hypertension
Inhaled Eee nee Treprostinil Pulmonary Vascular Disease Favorable Effects of Inhaled Treprostinil in Severe Pulmonary Hypertension Results From Randomized Controlled Pilot Studies ewe Ene MD Fah ‘MD, Markus Kobstal, MD,* Fredich Grimminger, MD, PuD,* Lewis J. Rubin, MD,f Weener Seeger, MD, Horst Osc
Slide 20: Three Pillars of Value Creation
ARIKAYCE® (Amikacin Liposome Inhalation Suspension)*Refractory NTM: M. avium complex (MAC)Front Line Label Expansion PRECLINICAL PHASE 1 PHASE 2 PHASE 3 APPROVED * In the U.S., as a condition of accelerated approval, Insmed is conducting an additional clinical study to support full approval. Full ap
Slide 21: Comparison of Prostacyclin Analogs/Agonists
Comparison of Prostacyclin Analogs/Agonists Approved Therapies vs. TPIP (investigational product candidate) Route of administration IV orSubcutaneous Inhaled (nebulized) Oral Oral Inhaled (dry powder) Dosing frequency Continuous 4x per day 2x or 3x per day 2x per day Once daily Dose-limiting side ef
Slide 22: Market Opportunity
22 Market Opportunity Innovate Grow Expand Current PAH Landscape New PH Opportunities Opportunities Beyond PH Prevalence of PH[1] WHO Group 1[2] Prevalence of PH ILD[4] & PH COPD[5] in WHO Group 3[2] Prevalence of Chronic Fibrosing ILD[2] [in U.S. as of 2019] [in U.S. as of 2019] [in U.S. as of 2019
Slide 23: Thank You
Thank You 23