example of a patient in the study on placebo · 2019. 7. 30. · cerebral edema, sometimes referred...

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Remedy Pharmaceuticals is a privately-held, phase 3 ready, acute care pharmaceutical company. We are focused on bringing life saving hospital-based treatments to people afflicted by central nervous system-related edema. Our lead product, CIRARA™, is a high affinity inhibitor of Sur1-Trpm4 channels, which are upregulated in ischemic and traumatic events. CIRARA is designed to close these channels and thus prevent or reduce edema in a variety of CNS-related indications. In a randomized, double-blind phase 2 study in large hemispheric stroke patients, CIRARA reduced mortality as well as improved functional outcomes, based on modified Rankin Scale (mRS) scores. Addressing the unmet need of CNS-related edema presents a multi-billion dollar opportunity Cerebral edema, sometimes referred to as brain swelling, is the presence of exces- sive fluid in the brain. Cerebral edema can result from brain trauma or from non-trau- matic causes, such as a stroke. When injury occurs, the blood brain barrier may break down, allowing fluid to accumulate in the brain’s extracellular space. The brain is especially susceptible to injury from edema because it is located within a confined space and cannot expand. If untreated, cerebral edema can cause death. Take stroke for example. Some 78% of ischemic stroke victims who die in the first week, die as a result of edema – brain swelling. And yet, no therapy has been developed to prevent this edema, until now. INTRODUCING CIRARA Sur1-Trpm4 channels are upregulated following ischemia and open due to ATP depletion. This results in sodium influx, which is followed by water that leads to cell swelling, damage and death. The resulting endothelial cell dysfunction results in blood brain barrier disruption, which allows fluids to cross into the brain, i.e., brain swelling. CIRARA is a potent blocker of Sur1-Trpm4 channels which prevents or reduces brain swelling through this mechanism. CIRARA is an IV formulation that can be administered bedside or in an ambulance. CIRARA utilizes the company’s patented Measured Phase Dosing (MPD™) regimen to enable optimal drug concentrations. Worldwide, CNS-related edema represents a $25 billion market opportunity. BROAD IP PORTFOLIO 16 patents issued from 5 patent families. The IP portfolio covers methods of use, formulations, manufacturing processes, and proprietary dosing, both domestically and international. 78% WWW.REMEDYPHARMACEUTICALS.COM RESEARCH PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 BRAIN EDEMA IN LIVER FAILURE POST SURGICAL EDEMA PERITUMORAL EDEMA POST CARDIAC ARREST INTRAVENTRICULAR HEMORRHAGE SUBARACHNOID HEMORRHAGE INTRACEREBRAL HEMORRHAGE MODERATE/SEVERE TRAUMATIC BRAIN INJURY SPINAL CORD INJURY ROBUST CNS EDEMA PIPELINE MODERATE/SEVERE STROKE EXAMPLE OF A PATIENT IN THE STUDY ON PLACEBO 1 6.8 Hours 72 Hours Lesion grows 6.8 Hours 72 Hours MLS occurs NOTE LESION GROWTH, MIDLINE SHIFT 3.9 Hours EXAMPLE OF A PATIENT IN THE STUDY ON CIRARA 2 72 Hours No lesion growth 72 Hours No MSL NOTE NEAR ABSENCE OF LESION GROWTH OR MIDLINE SHIFT 1 57-year old male enrolled in GAMES-RP 2 50-year old male enrolled in GAMES-RP 3.9 Hours NOTE: CIRARA is an investigational drug and is not approved for use.

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Page 1: EXAMPLE OF A PATIENT IN THE STUDY ON PLACEBO · 2019. 7. 30. · Cerebral edema, sometimes referred to as brain swelling, is the presence of exces-sive fluid in the brain. Cerebral

Remedy Pharmaceuticals is a privately-held, phase 3 ready, acute care pharmaceutical company. We are focused on bringing life saving hospital-based treatments to people afflicted by central nervous system-related edema.

Our lead product, CIRARA™, is a high affinity inhibitor of Sur1-Trpm4 channels, which are upregulated in ischemic and traumatic events. CIRARA is designed to close these channels and thus prevent or reduce edema in a variety of CNS-related indications.

In a randomized, double-blind phase 2 study in large hemispheric stroke patients, CIRARA reduced mortality as well as improved functional outcomes, based on modified Rankin Scale (mRS) scores.

Addressing the unmet need of CNS-related edema presents a multi-billion dollar opportunityCerebral edema, sometimes referred to as brain swelling, is the presence of exces-sive fluid in the brain. Cerebral edema can result from brain trauma or from non-trau-matic causes, such as a stroke.

When injury occurs, the blood brain barrier may break down, allowing fluid to accumulate in the brain’s extracellular space. The brain is especially susceptible to injury from edema because it is located within a confined space and cannot expand. If untreated, cerebral edema can cause death.

Take stroke for example. Some 78% of ischemic stroke victims who die in the first week, die as a result of edema – brain swelling. And yet, no therapy has been developed to prevent this edema, until now.

INTRODUCING CIRARASur1-Trpm4 channels are upregulated following ischemia and open due to ATP depletion. This results in sodium influx, which is followed by water that leads to cell swelling, damage and death. The resulting endothelial cell dysfunction results in blood brain barrier disruption, which allows fluids to cross into the brain, i.e., brain swelling.

CIRARA is a potent blocker of Sur1-Trpm4 channels which prevents or reduces brain swelling through this mechanism.

CIRARA is an IV formulation that can be administered bedside or in an ambulance.

CIRARA utilizes the company’s patented Measured Phase Dosing (MPD™) regimen to enable optimal drug concentrations.

Worldwide, CNS-related edema represents a $25 billion market opportunity.

BROAD IP PORTFOLIO16 patents issued from 5 patent families.

The IP portfolio covers methods of use, formulations, manufacturing processes, and proprietary dosing, both domestically and international.

78%

WWW.REMEDYPHARMACEUTICALS.COM

RESEARCH PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3BRAIN EDEMA IN LIVERFAILURE

POST SURGICAL EDEMA

PERITUMORAL EDEMA

POST CARDIAC ARREST

INTRAVENTRICULARHEMORRHAGE

SUBARACHNOIDHEMORRHAGE

INTRACEREBRALHEMORRHAGE

MODERATE/SEVERETRAUMATIC BRAININJURY

SPINAL CORD INJURY

ROBUST CNS EDEMA PIPELINE

MODERATE/SEVERESTROKE

EXAMPLE OF A PATIENT IN THE STUDY ON PLACEBO1

6.8 Hours 72 Hours

Lesion grows

6.8 Hours 72 Hours

MLS occurs

NOTE LESION GROWTH, MIDLINE SHIFT

3.9 Hours

EXAMPLE OF A PATIENT IN THE STUDY ON CIRARA2

72 Hours

No lesion growth

72 Hours

No MSL

NOTE NEAR ABSENCE OF LESION GROWTH OR MIDLINE SHIFT

1 57-year old male enrolled in GAMES-RP2 50-year old male enrolled in GAMES-RP

3.9 Hours

NOTE: CIRARA is an investigational drug and is not approved for use.

Page 2: EXAMPLE OF A PATIENT IN THE STUDY ON PLACEBO · 2019. 7. 30. · Cerebral edema, sometimes referred to as brain swelling, is the presence of exces-sive fluid in the brain. Cerebral

In a Phase 2 randomized, double blind, placebo-controlled study in large hemispheric stroke, CIRARA cut mortality by more than 50%, halved midline shift, and improved functional outcomes.

• 90-day mortality in the CIRARA group was reduced by 53% vs. placebo. • Midline shift, a key indicator of brain swelling, was halved in the CIRARA

group versus the placebo group.• There were no deaths in patients treated with CIRARA less than 8 hours

from onset of stroke. In contrast, half the placebo subjects treated in less than 8 hours died.

• 29% more CIRARA-treated patients had 0-4 mRS – a standard measure-ment of functional outcome – versus placebo patients.

• Using a “shift” analysis, the mRS at 90 days was improved across the board (p=0.12).

• In subjects dosed in <8 hours, 75% of CIRARA patients had 0-4 mRS, and 63% had 0-3 mRS, versus 25% for both 0-3 and 0-4 mRS in the placebo group.

• In subjects ≤ 70 years old, mortality was reduced by 64% versus placebo (12% vs. 33%) and 0-4 mRS was improved by 32% versus placebo (69% vs. 47%).

• Using a “shift” analysis, the mRS at 90 days in patients ≤70 was improved across the board (p=0.048).

• While CIRARA did not reduce the incidence of decompressive craniectomy (DC), regardless of whether CIRARA patients underwent DC or not, they had substantially improved survival rates and mRS scores versus placebo patients.

• CIRARA was well tolerated.

KEY FINDINGS FROM THE 90-DAY FOLLOW UP INCLUDE:

Multiple near-term catalysts• FDA end-of-Phase 2 meeting• Phase 3 large hemispheric stroke study design• Phase 2 Spinal Cord Injury pilot study enrollment

• Phase 2 Traumatic Brain Injury pilot study data analysis; Phase 2b design• Breakthrough Therapies/Fast Track designation applications• Orphan Drug applications for other indications

REMEDY PHARMACEUTICALS122 West 27th Street, 10th FloorNew York, NY 10001212.586.2226

Novel solution addressing major unmet medical need: CNS edemaLarge, underserved patient populationNo effective therapies exist to treat CNS edemaLead product, CIRARA, Phase 3 ready with strong Phase 2 dataLead indication, large hemispheric stroke, represents multi-billion dollar global opportunityOrphan Drug status granted for acute spinal cord injury and acute subarachnoid hemorrhage; potential orphan designations in other indications

DOWN

VERSUS PLACEBOP=0.06

90-DAY MORTALITY

REDUCED

VERSUS PLACEBOP=0.0006

72-HOUR MIDLINE SHIFT

IMPROVED

VERSUS PLACEBOP=0.23

90-DAYmRS

INVESTMENT HIGHLIGHTS: MANAGEMENT:David GeliebterExecutive ChairmanSven JacobsonChief Executive OfficerRichard SteinhartChief Financial OfficerAnn TunstallVP Regulatory & Operational AffairsThomas MacAllisterVP Research & Development, and General Counsel

NOTE: CIRARA is an investigational drug and is not approved for use.