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JEFFERIES GLOBAL HEALTHCARE CONFERENCEJune 4, 2019
2Confidential
Every story starts with inspiration. Our founder discovered his when his mother passed away of a Subarachnoid Hemorrhage (SAH).
From that moment, he refocused his expertise, passion and unique abilities to ensure no one else has to lose a loved one from something he could potentially treat.
Through our specialty life sciences company, we seek to bring novel drug delivery technologies to improve outcomes in rare diseases.
To ensure that her memory lives on, and that we always remain patient-centric, he named the company for her.
Meet Grace…
Our Story
3Confidential
Agenda
Company overview 4
Products overview 8
Appendix 23
Page
4Confidential
Experienced management team
• CEO, Cedar Pharma (fka Citron) and Aster Pharma (fka Lucid) and Exec. Committee Member of Rising Pharma, all owned by Aceto; Founder of Citron and Lucid, sold to Aceto in 2016; currently on Boards of Aceto and Cronus Pharma (animal health)
• Co-founded Celon Labs., Sequoia Capital-funded specialty pharma (Oncology and Critical Care) in India providing 80+ branded generic pharmaceuticals
• Founded Gen-Source RX, leading national distributor of generic pharmaceuticals acquired by Cardinal in 2014 and Casper Pharma, specialty brand and injectable pharma company
• BS, Pharmacy, HKE College of Pharmacy, India
• Co-founder and President, Insys Therapeutics and principal inventor of SUBSYS®, sublingual fentanyl spray for the treatment of breakthrough cancer pain (peak annual revenues > $250M)
• Previously, senior positions at Unimed Pharmaceuticals (Solvay, now Abbvie) with key roles in product development and obtaining FDA approval for ANDROGEL® (peak annual revenues >$1.5B) and in formulating and executing strategy that resulted in down-scheduling of controlled prescription drug MARINOL® by US DEA
• BSc, Fergusson College; MSc, University of Poona; PhD, University of Kentucky
George Kottayil, PhD CEO & Co-Founder
• National Practice Leader, Willis Towers Watson managing services addressing employer-sponsored health centers; previously, Partner at Mercer developing programs to improve access, quality and cost-effectiveness of care delivery
• Prior experience includes senior leadership roles at EJ Financial Services, Thomas Weisel Partners, Jefferies & Co., and Glaxo SmithKline
• Independent Director of Ascension Ventures• AB, Harvard University; MD, Boston University School of Medicine; MBA, The Wharton School
Bruce Hochstadt, MDCOO
• VP Sales and Marketing, Archi-Tech Systems, working with specialty, orphan, and small molecule manufacturers; recruited, deployed, and managed the salesforce including end-to-end sales process, consultative-selling model, and financial forecasts
• Led go-to-market strategy for new product launches, marketing plan and pricing model• Prior leadership experience at Cardinal Health, IMS Health, Rosenbluth, and D&B • BA, Computer Science & Math, Augustana College; MBA, The Wharton School
Prashant Kohli, MBAVP, Commercial Operations
Vimal Kavuru, RPhChairman & Co-Founder
5Confidential
Independent Directors
• Dr. Haseltine is Chairman & President of ACCESS Health International, Inc. and Chairman of the Haseltine Foundation for Science and the Arts. He is the Founder of Human Genome Sciences and served as its Chairman and CEO for twelve years. He is also the founder of several other successful biotechnology companies. Eight pharmaceutical products from companies that he started are currently approved by US and international regulatory agencies.
• Dr. Haseltine is a Founder and CEO of Demetrix, Inc., a biotechnology company specializing in pain and anxiety medications, and a Founder and Director of X-VAX, which is developing a novel Herpes Simplex vaccine. He was a professor at Harvard Medical School and Harvard School of Public Health, where he was the Founder and Chair of the Division of Biochemical Pharmacology and the Division of Human Retrovirology. He is well known for his seminal work on cancer, HIV/AIDS and genomics, and has authored more than 200 manuscripts published in peer reviewed journals.
• Dr. Haseltine is a lifetime member of the New York Academy of Sciences, a Trustee of the New York Academy of Medicine, and an honorary trustee of the Brookings Institution.
• Dr. Hormats, Vice Chairman of Kissinger Associates, Inc., has been a senior official in the administrations of five American presidents, a top Wall Street executive and a major business leader working with companies throughout the world. His most recent government position was Under Secretary of State for Economic Growth, Energy and the Environment, which he assumed in 2009.
• Following key roles on the National Security Council staff, Dr. Hormats served as Deputy Assistant Secretary of State for Economic and Business Affairs, Deputy US Trade Representative with the rank of Ambassador and Assistant Secretary of State for Economic and Business Affairs.
• In 1982, Dr. Hormats joined Goldman Sachs where he ultimately became Vice Chairman, Goldman Sachs (International). Over his 25-year plus career at the firm, he played major roles on a variety of international economic and financial matters, including privatizations of foreign state enterprises in countries such as China, Singapore, Britain, Germany and Mexico.
Dr. Robert Hormats
Dr. William Haseltine
6Confidential
Transforming proven compounds for rare and orphan diseases through novel formulation and delivery technologies
• Novel formulation and delivery for established products
• Well-known and trusted by physicians, regulators and hospitals
• Proven safety and efficacy profile• Pursuing expedited and de-risked
505(b)(2) regulatory pathway • Three product candidates scheduled for Phase 2 / 3 in 2019-20
• Two additional promising assets in early-stage, advancing to clinic
• Multiple commercialization options being pursued to maximize value
• Secured IP for all including proprietary microparticle, bio-adhesive film, and spray technologies
• Leadership team with strong track record of bench-to-bedside success
UNIQUE VALUE PROPOSITION:
Trusted compounds reinvented
FILLING THE GAP:
Addressing critical unmet medical need
WHERE WE ARE: Developing
multiple late-stage CNS assets
• Exists within rare and orphan disease markets
• Each asset carries an orphan designation
• Provides enhanced patient outcomes and convenience
• Key Opinion Leaders (KOL) –validate strong potential for rapid adoption
7Confidential
Overview of pipeline
Approved Orphan Designation
Indication Pre-clinical Phase 1 Phase 2 Phase 3Product Candidate
Subarachnoid Hemorrhage (SAH) GTX-104 Pivotal 2H’2019 $320mm
Postherpetic Neuralgia (PHN) GTX-101 Phase 1 2H’2019 / Phase 2 1H’2020
Ataxia Telangiectasia (A-T) GTX-102 Phase 3 1H’2020
Trigeminal Neuralgia (TGN) GTX-201
Aortic Aneurysm GTX-103
$1.6bn
$150mm
Market Size
Orphan Designation Will Be Sought
8Confidential
Near-term clinical milestones
Accomplished milestonesFuture milestones
GTX-104 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4
Phase I studies
FDA meeting
Pivotal PK study
Physician-IND
NDA submission
Approval / LaunchGTX-101
Non-Clinical studies
FDA Pre-IND meeting
Device engineering / human factor study
IND submission
Phase I study (SAD/MAD)
Phase II study
Phase III efficacy study
Phase III safety study
NDA submission
Approval / LaunchGTX-102
IND submission
Phase 1 BA study
Phase III confirmatory efficacay and safety NDA submission
Approval / Launch
20232017 2018 2019 2020 2021 2022
2023
2017 2018 2019 2020 2021 2022 2023
2017 2018 2019 2020 2021 2022
Today
Assumptions
Funding is secured by May/Jun 2019
GTX-104
Clinical Study for PK bridging
GTX-101
Non-Clinical
xyz
Phase II
8 weeks
100 patients
50 sites
Phase III efficacy
12 weeks
300 subjects
100 sites worldwide
Phase III safety
6 months
500 subjects
100 sites worldwide
GTX-102
Phase III efficacy and safety
3 months efficacy study
50 subjects
1 site
GTX-101 Phase 2 then 3
2017201820192020202120222023
GTX-104Q1Q2Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4Q1Q2Q3Q4
Phase I studies
FDA meeting
Pivotal PK study
Physician-IND
NDA submission
Approval / Launch
GTX-1012017201820192020202120222023
Non-Clinical studies
FDA Pre-IND meeting
Device engineering / human factor study
IND submission
Phase I study (SAD/MAD)
Phase II study
Phase III efficacy study
Phase III safety study
NDA submission
Approval / Launch
GTX-1022017201820192020202120222023
IND submission
Phase 1 BA study
Phase III confirmatory efficacay and safety
NDA submission
Approval / Launch
9Confidential
Agenda
Company overview 4
Products overview 8
Appendix 23
Page
10Confidential
Overview of Subarachnoid Hemorrhage (SAH)GTX-104
GTX-101GTX-102
Sources: National Inpatient Sample (NIS), Healthcare Cost and Utilization Project (HCUP), Agency for Healthcare Research and Quality; UpToDate; Fletcher Spaght primary research
Medicare37%
Medicaid15%
Pvt Ins37%
Uninsured / Other11%
SAH Payers
Devastating neurological condition• 10-15% die before reaching hospital• 20% of admitted patients die in the hospital
One of the most expensive acute conditions to treat• $220,000 average per patient hospital charge
34kaSAH
85% 15%
• Caused by ruptured brain aneurysm
• Associated with vasospasm that leads to delayed cerebral ischemia
• Typically results from vascular malformation or arterial dissection
• Vasospasm less of an issue
6knaSAH
40k (non-traumatic) SAH patients treated annually in US
*Specifications Manual for Joint Commission National Quality Measures (Nimodipine Treatment Administered performance measure)
Nimodipine (FDA approval - 1988) improves neurological outcomes in SAH• Use and timing of nimodipine administration tracked by the Joint Commission*• Only available in oral capsule and liquid solution in US• IV formulation available ex-US has limited utility due to high alcohol content
GTX-104
GTX-104
11Confidential
Novel nanoparticle formulation for IV infusion
Benefits: Novel nanoparticle technology facilitates aqueous formulation of insoluble nimodipine and enables a safe, continuous standard peripheral IV infusion
• Better manage hypotension• 100% bioavailability
• Low intra-subject variability• No drug-drug interactions / food effects
10 – 15 nm
Drug loaded Micelle
Nimodipine
Surfactant Monomers
Unmet Need as oral nimodipine has several limitations:• Dose-limiting side-effects
(hypotension)• Difficult to administer to
unconscious patients or those with impaired swallowing
• Low bioavailability (high first-pass metabolism)
• Narrow administration window as food effects lower bioavailability significantly
• Concomitant use with CYP3A inhibitors is contraindicated
GTX-104 Solution:
GTX-101GTX-102
GTX-104
12Confidential
GTX-104’s superior value proposition
Robust pharmacoeconomic model will help fine-tune pricing
+100% bioavailable+Control hypotension+No food effects +No DDI- Drug intake
- Medication error- Nursing burden- Rescue therapy- ICU / length of stay+JC compliance*+Economic impact
+Safer +Outcomes+Convenient+Faster recovery- Disease burden
Pricing sensitivity study is planned
* Nimodipine administration in SAH patients is a key Joint Commission (JC) quality measure for hospitals with stroke certification
Clinical Value Patient Value Hospital Value
GTX-101GTX-102
GTX-104
13Confidential
Total US addressable markets of ~$325M
Sources: National Inpatient Sample (NIS), Healthcare Cost and Utilization Project (HCUP), Agency for Healthcare Research and Quality; UpToDate; Fletcher Spaght primary research; IMS
Experts at leading stroke centers suggest strong use case for at least 60% of total SAH inpatient days
34kannual inpatient
admissions
$322M
aSAH
6kannual inpatient
admissionsnaSAH
460kPatientdays
$700/day
12 daysaverage
LOS
9 daysaverage
LOS
GTX-101GTX-102
GTX-104
14Confidential
Commercial strategy to deploy direct salesforce
Source: Based on average # of SAH patients treated annually at comprehensive stroke centers interviewed by Fletcher Spaght, Inc.; HCUP National (Nationwide) Inpatient Sample, 2011
Highly concentrated hospital base treating majority of SAH patients
Launch focus on 176 stroke centers managing ~45% of SAH patients
Location of Certified Comprehensive Stroke Centers
90 110 170 425 410
1,395
>100 50-100 25-49 10-24 5-9
15Confidential
Overview of Postherpetic Neuralgia (PHN)GTX-101
PHN clinically defined as pain persisting 3 months after onset of rash• Many clinicians do not wait and instead start treating pain as
soon as the rash clears
• Leading cause of debilitating pain in elderly
• 60% of PHN patients have pain that persists for over a year
5 years, 20%
Duration of Pain in PHN patients
2-5 years, 15%
1-2 years, 25%
250k new 250k persistent
500k patients with PHN
Sources: Fletcher Spaght primary research; CDC; peer reviewed journal articles
• Wide anatomic distribution
• Scalp, face, and neck often involved
• Spanning joints and skin folds
~1M casesof shingles annually (US)
GTX-101
GTX-101GTX-102
GTX-104
16Confidential
PHN standard of care has limitations
• Unmet Need as lidocaine patches are suboptimal• 40% of patients experience insufficient pain relief • Optimal analgesic effect could take up to 2 weeks• Patches difficult to use, fall off, and unsightly • Skin sensitivity and irritation
• Gabapentin and opioid abuse has continued to proliferate
Treatment of PHN most often consists of gabapentin and lidocaine patches
Antidepressantsamitriptyline, nortriptyline, desipramine, duloxetine
Topical anesthetic 5% Lidocaine patch, ZT Lido 1.8%
Generic gabapentin
First Line Second Line Third Line
Opioids / Interventional
Branded AnticonvulsantsLyrica®, Gralise®, Horizant®
>90% of PHN patients are managed by PCPsRefractory cases referred to neurologists or pain specialists
GTX-101GTX-102
GTX-104
>90%
17Confidential
Innovative spray delivery of bupivacaine
• Metered-dose of bupivacaine spray forms a thin bioadhesive topical film– Touch-free, non-greasy application– Convenient, portable 30mL plastic bottles
• Biphasic delivery mechanism enables rapid onset and continuous pain relief for up to 8 hours– No skin sensitivity reported in Phase 1 study
Mechanism of bioadhesive film formation
Mechanism of film formation
GTX-101 solution:
GTX-101GTX-102
GTX-104
18Confidential
Non-opioid, more effective solution with expanded use case
• Rapid onset of action and extended relief
• Easy to use (no peeling and cutting patches to size)
• Ability to precisely cover the affected area
• No skin sensitivity or irritation
Easier, more convenient application
GTX-101GTX-102
GTX-104
• More potent analgesic and opioid-sparing
• Well understood and efficacious
• Longer acting vs. lidocaine
• Strong KOL support for bupivacaine
Bupivacaine, an ideal topical analgesic
• Convenience will improve compliance and expand use
• Burgeoning market for non-PHN indications
• Enthusiasm across specialties
Expanded use for PHN and other indications
19Confidential
Total US addressable market of ~$1.5b
Sources: Fletcher Spaght primary research and analysis; IMS
Positioned to displace and expand the lidocaine patch market
30M doses
$1.6bPHN 130%
100M doses 120%Other Pain
39M“doses”
Current lidocaine patch market
Anticipated use relative to lidocaine patch
120M“doses”
$10/dose
Comparable to Lidoderm®price/patch
GTX-101 “dose” = single lidocaine patch• Diabetic neuropathic pain • Fibromyalgia • Focal/peripheral neuropathy• Complex regional pain syndrome• Spine-related pain• Intercostal pain
GTX-101GTX-102
GTX-104
20Confidential
Overview of Ataxia Telangiectasia (A-T)GTX-102
Unmet Need as no effective therapeutic options currently available • No drugs approved for A-T•
21Confidential
Novel oromucosal betamethasone formulation
Novel formulation of betamethasone intended to significantly improve neurological symptoms of ataxia telangiectasia• Clinical benefits: ~30% decrease in ataxia symptoms* observed on day 31 in an earlier clinical study of oral
betamethasone• Ease of administration: 1-3x 140µL applied onto tongue using metered dose spray
– dosing: 0.25, 0.5 or 1.0 mg per spray
Placebo Betamethasone Efficacy
Day -1 Day 31 Day -1 Day 31 Δ 95% CI for the median P value
Total Score* 46(14-69)
41.5 (26-68)
50 (20-68)
33(19-55)
-13 (-28 to 14)
-19 to -5.5 0.01
Source: Zannolli R, et al. A randomized trial of oral betamethasone to reduce ataxia symptoms in ataxia telangiectasia. Mov Disord. 2012;27:1312–6. Note: Data are medians (ranges); P values calculated using Wilcoxon rank sum test
Clinical data for betamethasone oral solution in A-T patients(Scores for ataxia symptoms assessed by ICARS)
*Includes assessments of posture and gait disturbance and kinetic, speech and oculomotor functions
GTX-101GTX-102
GTX-104
GTX-102
22Confidential
Total addressable market$150M
Serviceable addressable market is about 60% of patients or 1500 per year presenting a strong use case per KOL interviews
Total US addressable market of ~$150M
*Estimated A-T incidence 1 in 20,000-100,000 live births; median A-T patient lifespan 25 years (“Ataxia-telangiectasia.” Opal and Bonilla. UpToDate, 2017)
~2,500Prevalence (US
Patient Population)*
$60,000Price / Year
60%
High penetration of this serviceable market can be expected with most US patients managed at A-T Clinical Center (Johns Hopkins)
23Confidential
Grace Therapeutics – Summary
Rare and orphan disease company with CNS focus and strong global IP estate
Leverage innovative drug delivery and proprietary formulation technologies
De-risked, 505(b)(2) regulatory pathway utilizing approved molecules
Robust pipeline with three lead assets on cusp of late-stage development
Proven management team with track record of bench-to-bedside success
24Confidential
Agenda
Company overview 2
Products overview 8
Appendix 23
Page
25Confidential
Strong intellectual propertyUS Publication # Publication Date Abstract
20170296485 Oct 19, 2017 A polymeric bio-adhesive film forming topical spray formulation providing a modified, pulsatile (e.g., biphasic) release of the active agent(s) once the solvent evaporates and the film sets, e.g., on human skin is disclosed. In certain embodiments, the active agent is bupivacaine hydrochloride.20170296484 Oct 19, 2017
GTX-101: Topical Film-Forming Spray (Postherpetic Neuralgia)
US Publication # Publication Date Abstract
10092553 Oct 9, 2018 A nimodipine injection concentrate and diluted formulation comprises nimodipine (base or salt), an effective amount of a hydrophilic surfactant, and a pharmaceutically acceptable carrier for injection which is an aqueous solution, an organic solvent, an oil, or a cyclodextrin, such that the nimodipine is substantially contained in a concentrated injection solution, suspension, emulsion or complex as a micelle or a colloidal particle or an inclusion complex and the formulation is stable and clear. In certain embodiments, the hydrophilic surfactant is polysorbate 80.
10092557 Oct 9, 2018
20180325882 Nov 15, 2018
20180325886 Nov 15, 2018
US Publication # Publication Date Abstract
20170196889 Jul 13, 2017
A stable oral spray formulation comprising a glucocorticoid together with other excipients is disclosed. In preferred embodiments, the spray formulation is used to treat neurological disorders such as ataxia by being sprayed in an effective dose into the mouth of a patient.
Approved orphan designation
GTX-104: Stable Nimodipine Parenteral Formulation (Subarachnoid Hemorrhage)
Approved orphan designation
GTX-102: Betamethasone Oral Spray Formulation and Method of Use (Ataxia Telangiectasia)
Approved orphan designation
GTX-201: Therapeutic Composition of Intranasal Lidocaine (Trigeminal Neuralgia)
US Application # Filling Date Abstract
62/781969 Dec 19, 2018The present invention discloses a (preferably) preservative free intranasal composition in multi-dose or bi-dose or unit-dose spray container to treat the pain associated to tri-geminal neuralgia, facial neuropathic pain, facial cancer induced neuropathic pain and migraine pain.
Slide Number 1Slide Number 2AgendaExperienced management teamIndependent Directors Transforming proven compounds for rare and orphan diseases through novel formulation and delivery technologiesOverview of pipelineNear-term clinical milestonesAgendaOverview of Subarachnoid Hemorrhage (SAH)�GTX-104Novel nanoparticle formulation for IV infusionGTX-104’s superior value propositionTotal US addressable markets of ~$325MCommercial strategy to deploy direct salesforceOverview of Postherpetic Neuralgia (PHN)�GTX-101PHN standard of care has limitationsInnovative spray delivery of bupivacaineNon-opioid, more effective solution with expanded use caseTotal US addressable market of ~$1.5bOverview of Ataxia Telangiectasia (A-T)�GTX-102Novel oromucosal betamethasone formulationTotal US addressable market of ~$150MGrace Therapeutics – Summary AgendaStrong intellectual property