developing!a!novel!treatmentfor! refractory!cancer!pain! · // the amorsa approach – drug...
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Developing A Novel Treatment for
Refractory Cancer Pain
Pain that is constant, severe and disabling
and for which there is no FDA-‐approved treatment
Amorsa Therapeu;cs Inc Cancer Cluster Showcase – BIO2014
www.amorsatx.com
// Refractory Cancer Pain (“RCP”)
• Key characteristics: – Constant – Severe – Disabling – Conventional therapy doesn’t work
• Often have pain intensity >7 (on a pain scale of 0-10; 10 indicating the worst imaginable pain)
• No FDA-approved treatment for these patients
“There is no suffering worse than intractable (refractory) pain” Dr. Forest Tennant, M.D.
// The Amorsa Approach – Drug Repurposing
• “Repurposing” and “reformulating” the FDA approved anesthetic, ketamine, for RCP
• Numerous success stories using this approach • Ketamine shown to have “opioid sparing” effects • Plan to follow the 505(b)2 regulatory path • Several other potential indications for the
technology
“Repurposed drugs could be the key to R&D producHvity” (GEN 2013)
// Ketamine Extended Release Tablets (“K-ER”)
Once-‐daily applica;on
250 mg tablets (50mg API)
12-24 h window of action
A convenient, safe and effec0ve treatment for refractory cancer pain
Effec;ve
Convenient Compelling Preclinical Results
• Animal studies showing slow and steady release
• No spikes in ketamine concentration
• Extended release, once-daily tablet formulation
• Improved compliance
• Sub-anesthetic dose • Schedule III • Neuro-protective formulation
• Understood mechanism of action
• Substantial body of evidence
Safe
// Concentra;on-‐Time Plot
Combined PK data for Ketamine/Norketamine; R. Kubota et al., J. Cur. Surg., 2013
…a6er oral administra0on of ketamine solu0on in healthy volunteers
0
100
200
300
0 2 4 6 8 10 12
ng/mL
h
Dose 50 mg Cmax 280 ng/mL Tmax 1.1-‐1.6 h T1/2 1.1-‐5.3 h
Desired Efficacy/Safety Range
// In Vitro Performance - Amorsa K-ER
§ Prototypical tablets, d=9-10 mm, 200 mg total (20-30 mg API) § Multiple formulations of a “tunable” release matrix § Robust in vitro/in vivo correlations showing steady, extended
release
0
20
40
60
80
100
0 5 10 15 20 25 30
Release, %
Time, h
K-‐ER Formula8on Three Ini8al Release Profiles
KTM-‐1
KTM-‐2
KTM-‐3
// Market Opportunity
Global Therapeutics Market 2013 2019 CAGR
Cancer Pain Management +$4 Billion $7 Billion 9.1%
Refractory Cancer Pain Indication Only
~600,000 cancer patients (U.S. Only)
Peak Sales Potential for Amorsa K-ER
Worldwide: $550 Million U.S. Only: $350 Million
Other Potential Indications Including
Depression > $1 Billion
// Amorsa K-ER Development Plan
Initial Indication
Market Opportunity
Refractory Cancer Pain
~25% of Advanced
Cancer Patients
505(b)(2) Regulatory Strategy – A Rapid Approval Route
Preclinical Phase 1 Phase 2
Preclinical Studies • Safety and efficacy in animal model • Op;mize formula;on
Phase 1 Study • Healthy volunteers (n=15-‐20) • Human safety and pK profile • Drug tolerability
Phase 2 Study • Cancer pa;ents (n=150-‐200) • Placebo controlled, double blind • Efficacy & safety • Dose and dose frequency
Estimated 3 Years
// Leadership Team
Joe Blanchard, MBA
Alex Nivorozhkin, PhD
Mike Palfreyman, PhD
Mihir Kamdar, MD
Jacob Weintraub, Esq.
Chief Execu;ve Officer
Chief Opera;ng Officer
Chief Scien;fic Officer
Chief Medical Officer
IP Counsel
Aushon (COO/CBO), Altus (VP/GM), Genencor
(Director)
Inotek (Head of Chemistry), MGH (Sr. Program Manager)
Marion Merrell Dow (VP
Research), EnVivo (VP Development)
Director, MGH Cancer Pain Clinic
Palmer & Dodge, AstraZeneca
(Patent Aborney)
