Investor Presentation April 21, 2017 - Seeking Alpha€¦ · 2017-07-14 · Investor Presentation...
Transcript of Investor Presentation April 21, 2017 - Seeking Alpha€¦ · 2017-07-14 · Investor Presentation...
Investor Presentation
April 21, 2017
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Notice Regarding Forward-Looking StatementsThis presentation and the accompanying oral commentary (if any) contain both historical and forward-looking statements which are based on current expectations, estimates and projections. Statements that are not historical facts are forward-looking statements and typically are identified by words like “may,” “believe,” “anticipate,” “could,” “should,” “estimate,” “expect,” “intend,” “plan,” “project,” “will,” “forecast,” “approximately,” “budget,” “pro forma”, or the negative of these terms or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. All statements other than statements of historical facts contained in this presentation and the accompanying oral commentary (if any) are forward-looking statements, and include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the development of Trevyent and our At Home Patient Analgesia product candidates, the regulatory pathways for potential marketing approval of our product candidates, our intellectual property position, the market opportunities for our product candidates and our cash position.
Forward-looking statements involve known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Risks and uncertainties include, but are not limited to, the potential benefits of orphan drug designation, the company's ability to advance its development-stage product candidates, including Trevyent, statements about the potential benefits of our development-stage product candidates and our PatchPump technology, and statements about our ability to obtain and maintain regulatory approval of our development-stage product candidates. We discuss many of these risks in greater detail under the heading "Risk Factors" contained in our Annual Report on Form 10-K for the year ending on December 31, 2016 filed with the Securities and Exchange Commission on March 29, 2017. Forward-looking statements are not guarantees of future performance and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this presentation and the accompanying oral commentary (if any). Any forward-looking statements that we make in this presentation and the accompanying oral commentary (if any) speak only as of the date of this presentation. The “Risk Factors” section of our Form 10-K speaks only as of the date therefore. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise.
Investment Highlights A Late Stage, Pre NDA, Specialty Pharmaceutical Company
Lead Drug Candidate - Trevyent® for Pulmonary Arterial Hypertension (PAH)• Expected NDA submission Q2 2017• U.S. launch expected in H1 2018, specific timing depending on priority or normal NDA review • Expected Orphan Exclusivity; potential 7 years market exclusivity• Expected MAA submission in Europe second half 2017• Parenteral prostacyclins; high revenue, high margin drug products ~$175,000 per patient, per yr• Potential >$4.0 billion addressable market opportunity in U.S. alone
Early Stage Pipeline; At-Home Patient Analgesia (AHPA) drug product candidates• SMT – 201, Ketorolac, a non opioid, potent NSAID for post surgical pain in the home setting• SMT – 301, Bupivacaine, local anesthetic for post surgical pain in the home setting
Financial Snapshot and Key Investors• Key institutional investors – Federated, Deerfield, OrbiMed, Adage.• Cash and cash equivalents of $23.2 million as of December 31, 2016. • Cash on hand, plus potential $10.7 million second tranche closing of the 2016 PIPE, in Q3 2017,
provides cash runway into Q4 2018
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Key Trevyent Near Term Milestones
Note: Estimated regulatory, clinical and commercial milestones for our drug product candidates • *Subject to Priority Review of Trevyent NDA by FDA • ** Subject to normal Review of Trevyent NDA by FDA
Key Milestones
File MAA (EU) - Cardiome
NDA Approval
2017
Launch U.S.
2018
Launch EU - Cardiome
File NDA (U.S.)
*
*
**
**
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Our Primary Focus – Pulmonary Arterial Hypertension
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Progressive, life-changing and life-threatening disease
Orphan indication with ~30,000 patients diagnosed in the U.S.
Fewer than 200 treatment centers in the U.S.
Remodulin (treprostinil - United Therapeutics) the leading prostacyclin therapy• Revenue of ~$602 million in 2016• Annual average cost of Remodulin is ~$175,000 per patient• We believe there are only ~3,000 patients on Remodulin therapy
Change in pulmonary vasculature with disease progression leading to right sided heart failure
Treprostinil Therapies For PAH – Relative Efficacy
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Oral1,2
6 minute walk test data from 9 clinical studies Improvement from baseline in meters (M) walked
Inhaled3
Parenteral4 (SC or IV Remodulin)
10 – 23 M
18 – 31 M
80 – 125 M
1; Tapson et al., (2012); Jing et al., (2013); Tapson et al., (2013) 2; Chin, (2015) 3; McLaughlin et al.,(2010). Benza et al., (2011) 4; Tapson et al (2006). Hiremath et al (2010). Benza et al., (2013)
-7 M
Significantly higher efficacy for parenteral therapies in PAH patients
When is Parenteral Treprostinil Used to Treat PAH?
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New York Heart Association NYHA 1 NYHA 2 NYHA 3 NYHA 4
Patients in progressive state 20% 35% 30% 15%
Oral therapy e.g. PDE5 inhibitors, ERA’s
✓ ✓ ✓ ✓
Inhaled therapy e.g. iloprost, treprostinil
✓ ✓ Parenteral (SC / IV) treprostinil
*Estimated
~30,000 diagnosed patients in U.S.
~24,000 eligible patients*
Patients taking SC or IV treprostinil*
~3,000*
Current Treprostinil Administration*
8 8 * Illustrated examples
Infused Intravenously (IV) or Subcutaneously (SC) 24 hours a day, every day
Limitations of Treprostinil Administration
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Pumps not specifically designed for PAH or treprostinil Complex programming and error prone filling
• Infusion system complications in 28% of patients in controlled clinical studies
Subcutaneous Infusion site pain in 85% of patients • May be linked to meta-cresol preservative IV pump reservoir requires drug dilution
• Incorrect dosing possible• Non-aseptic technique can cause blood stream
infection and sepsis which may lead to death Not water resistant Back-up pump needed 24 hours a day
Prostacyclin Administration Errors – A National Survey*
Serious errors in medication administration reported by 68% of respondents The most common error types (reported by ~25%), included:
• Incorrect cassette placed in the pump• Inaccurate pump programming• Errors in drug dosing Interviews with PAH nurses; 94% reported serious errors 9 errors, all at different centers, believed to have contributed to patient death
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*Prostacyclin administration errors in PAH patients admitted to hospitals in the United States: a national survey. Kingman et al. Journal of Heart and Lung Transplantation 2010.
Survey of 97 PAH Clinicians ,18 PAH Specialty Nurses
May 2014, “Voice of the Patient” meeting with FDA & 85 PAH patients
Key discussion topics:
• The impact of PAH on daily life & currently available therapies
Summary of major themes:
• PAH is a progressive, devastating disease
• PAH negatively affects all aspects of patients’ lives
• Nearly all participants described using combination therapy
• And…
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Patients emphasized the need for medications that are effective, have convenient dosing schedules and are easy and safe to administer
Patients are Asking FDA for Better Treatment Options
12 N.B. Trevyent is a development stage drug product not approved for sale in any jurisdiction
Late Stage Drug Product for PAH
505 (b)(2) NDA submission expected Q2 2017
Expected U.S. approval late 2017 or early 2018 depending on NDA review period
Expected U.S. launch 2018
Subcutaneous or Intravenous PAH drug therapy Combines SteadyMed’s treprostinil and PatchPump infusion system
No patient filling• Prefilled with treprostinil at site of manufacture• Sterile drug product with potential for reduced blood stream infection• No drug vials, no syringes, no connectors, no diluents Simple dosing with no patient programming• Infusion rate pre-programmed at site of
manufacture• Potential to reduce patient dosing error Compact and water resistant
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Trevyent - Addressing the Limitations of Existing Therapy
N.B. Trevyent is a development stage drug product not approved for sale in any jurisdiction
Simplicity!
Connect to SC or IV cannula Trevyent automatically starts infusion
• Provides patient feedback via LED’s and audible tones
Trevyent runs for 48 hours• Provides LED and audible feedback Simply remove, dispose and replace with a new Trevyent
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Trevyent is Preservative Free
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Potential for reduced infusion site pain vs. Remodulin• More patients may use treprostinil earlier and less switches to IV therapy
Non-clinical evaluation of m-cresol preservative on infusion site pain• Continuous subcutaneous infusion for 7 days• Formulations representative of Remodulin (with m-cresol) and Trevyent (without m-cresol)
Substantially less tactile sensitivity* to ‘Preservative Free’ formulation
Mea
n P
lace
bo-C
orre
cted
Incr
ease
in T
actil
e S
ensi
tivity
Rel
ativ
e to
Bas
elin
e (g
ram
s fo
rce)
0 1 2 3 4 5 6 7
1 2 3 4 5 6 7 study day
Drug formulation without m-cresol (representative of Trevyent)
Drug formulation with m-cresol (representative of Remodulin)
* Measured by electronic Von Frey instrument
Compelling Medical Community Interest in Using Trevyent
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0
10
20
30
40
50
60
1 2 3 4 5 6 7
% P
refe
renc
e
1 = Not at all interested 7 = Would definitely want to adopt
~90% scored 6 or higher
2012 commissioned market research survey of 20 PAH practitioners;12 PAH physicians and 8 PAH specialty nurses from major PAH treatment centers
“Less intrusive”, “more convenient for the patient”, “easy to use”
“How interested would you or your patients be in switching to Trevyent?”
Current Clinical Practice - What Percent of Your Patients Are Taking Prostacyclins?
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2% 3% 6% 16% 12% 6%
9%
20%
9%
5%
10%
17%
6% 11%
14%
16%
16% 20%
14%
5%
36% 32% 26%
16%
20% 24% 21% 10%
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
NYHA FC I
NYHA FC II
NYHA FC III
NYHA FC IV
Tyvaso
Orenitram
Ventavis
Remodulin SC
Remodulin IV
Veletri
Flolan
Prostacyclin by NYHA Segment
Physicians prefer parenteral formulations in more severe patients due to efficacy over other route of administrations and the ability to reach sufficient dosages
Market Research with physicians (N=26), nurses (N=5), and patients (N=10). May 2016.
1% 14%
33% 35%
2%
1%
3% 11%
12%
5%
6%
17%
9% 4%
4%
9%
6% 7%
5%
7%
16% 17%
10%
3%
36% 32%
19%
9% 20% 22% 17%
10%
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
NYHA FC I
NYHA FC II
NYHA FC III
NYHA FC IV
Tyvaso
Orenitram
Ventavis
Remodulin SC
Remodulin IV
Veletri
Flolan
Trevyent
Trevyent has potential to capture a large share of the prostacyclin class; majority coming from Remodulin as well as an increasing use of parenteral treprostinil i.e. Trevyent
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Prostacyclin by NYHA Segment
What Percent of Your Patients on Prostacyclins Would You Prescribe Trevyent*?
Market Research with physicians (N=26), nurses (N=5), and patients (N=10). May 2016.
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Trevyent Usability Validated with PAH Patients
2 year program of 8 Human Factors studies completed
Goal – Design Trevyent specifically for treprostinil and patients with PAH
148 subjects in U.S. and Europe
92 PAH patients, 33 PAH Physicians, 23 other
Usability of Trevyent validated
Study Complete Goal
1 ✓ Ethnographic research
2 ✓ User interface evaluation
3 ✓ User interface evaluation
4 ✓ Wearability preferences
5 ✓ Full simulated use
6 ✓ User interface evaluation
7 ✓ Full simulated use
8 ✓ Final validation
Trevyent Flow Accuracy Meets Specification
Target accuracy over 48 hrs = +/- 6% Trevyent group average accuracy over 48 Hours = +/- 0.9% Group standard deviation = 0.27%
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Bio-waiver confirmed; No clinical studies required for approval• 505(b)(2) NDA in the U.S.
• Hybrid 10(3) Application in the E.U.
Remodulin Reference Listed Drug• Safety and efficacy data to be included in Trevyent prescribing information
Example of FDA product approvals through bio-waiver • Parenteral epoprostenol reformulation (Veletri®, Actelion) for PAH
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“FDA shall waive the requirement for clinical studies if the new drug product is a parenteral injection and it contains the same active and inactive ingredients in the
same concentration as a previously approved drug.”FDA regulation 21 CFR 320.22(b)
Streamlined Regulatory Pathway; Bio-waiver Confirmed
Trevyent Granted Orphan Designation
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Concurrence from FDA that Trevyent has the potential for clinical superiority vs. parenteral, inhaled and oral treprostinil Granted based on potential for:
• Improved safety due to reduced patient error – no patient dose programming• Reduced incidence of blood stream infection – sterile product, no patient filling• Reduced infusion site pain – no m-cresol preservative
Significant Potential Benefit• 7 years PAH market exclusivity*
* Subject to NDA approval
Forging Strong Relationships with the PAH Community
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BePHenomenal.com
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Late stage drug product candidate; NDA submission expected Q2 2017
In development to address limitations of current treprostinil therapy
Potential to take share from existing treprostinil sales and offertherapy to more PAH patients
Planned U.S. launch in 2018, if approved
High revenue and high margin potential; ~$175,000 per patient per year
N.B. Trevyent is a development stage drug product not approved for sale in any jurisdiction
Post-Surgical Pain – A Significant Unmet Opportunity
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>70 million surgeries performed annually in the U.S.
~80% of patients experience inadequate pain relief
Often used therapies include:
• Opioids
- Poor side effect profile, risk of addiction and abuse
• Potent non steroidal anti-inflammatories (NSAIDs)
- Inconvenient dose initiation (multiple IV or IM doses over 24 hours in hospital)
• Local anesthesia delivered by elastomeric pumps
Goal – Reduce the length of hospital stay and treat patients at home with effective and well tolerated therapy
At Home Patient Analgesia (AHPA) Pipeline
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SMT–201 Ketorolac AHPADrug product; combination of the NSAID ketorolac and PatchPump
Provides pain relief at the opioid level from a non-opioid
Continuous subcutaneous infusion eliminates IV/IM initiation in hospital over 24 hrs
Around-the-clock pain relief for 3-5 days in the home setting
Avoids abuse, addiction and other side effects linked to opioids
SMT-301 Bupivacaine AHPAContinuous infusion of local anesthetic directly into surgical wound
Early stage; formulation development essentially completed
Ketorolac AHPA Proof of Concept PK Study
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Comparable Bioavailability
4 Intramuscular injections
Continuous subcutaneous delivery
12 healthy volunteers,120 mg of ketorolac over 24 hours by multiple IM injection or continuous SC infusion
Cross-over PK study design
Comparable Area Under the Curve (AUC) and Maximum Concentration (Cmax) value
Proof of Concept established
Bupivacaine AHPA Proof of Concept Nerve Block Study
Study summary• Single injection at sciatic nerve in pigs; looked at local sensation and gross motor
behavior• SteadyMed bupivacaine high concentration low volume formulation vs. Marcaine
high volume low concentration• Single injection in pigs into the sciatic nerve region• Goal to determine if a small volume will provide effective anesthesia• Encouraging results; next steps = continuous infusion study
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1. PCB – controls delivery rate and dose, sensors provide visible and audible feedback to patient2. ECell – an expanding battery that acts as the ‘motor’ in the PatchPump to drive the piston3. Piston – compresses the collapsible drug container to deliver drug through a soft cannula4. Drug container – aseptically filled with sterile liquid drug at site of manufacture5. Infusion Set – delivers drug subcutaneously or intravenously
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2
3
4
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Management - Relevant Experience and Track Record
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Jonathan Rigby, MBA – President and CEO• 27+ years experience in pharma, medical device and drug delivery industry• Co-founded Zogenix, who developed a drug delivery-enabled product to U.S. launch• Held key responsibilities in the development and licensing of a launched &
development stage product for the treatment of PAH• Executed licensing of an inhaled product to treat PAH• Prior experience includes: Merck, Bristol–Myers Squibb, Phillips, Zogenix
David Nassif, J.D. – EVP and CFO• 20+ years of life sciences industry experience• Extensive experience in private equity financing, finance law, business development
and SEC compliance• Prior experience includes: Amphastar, Zogenix & Questcor
Peter D. Noymer, Ph.D. – EVP and COO• 15+ years of experience from drug delivery combination product development to
commercialization, including Adasuve®, the first FDA approved inhalable treatment for acute agitation
• Prior experience includes: Aradigm & Alexza
Management - Relevant Experience and Track Record
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Carl Hicks – VP, Patient Advocacy and Community Outreach• Known worldwide as a ambassador in the fight against pulmonary hypertension;
served over a decade as a member and chairman of the board of the Pulmonary Hypertension Association’s (PHA) board of trustees, currently is the only lifetime honorary member of the board
• Executive vice president of PHA, and has dedicated his life to the struggle following the loss of his daughter Meaghan to IPAH
Douglas Neale – VP, Commercial Operations• ~20 years experience, including the commercialization of both billion-dollar and
smaller niche products, including several 505(b)2, across multiple therapeutic categories, with leadership responsibilities ranging from distribution to reimbursement, marketing and sales
• Prior experience includes: Eisai, Cephalon, ProStraken
Robert Zwolinski, MBA – SVP Operations• 19+ years experience, including the manufacture of aseptic liquid drug filled container
closure systems• Prior experience includes: Aradigm & Mylan
Investment Highlights A Late Stage, Pre NDA, Specialty Pharmaceutical Company
Lead Drug Candidate - Trevyent® for Pulmonary Arterial Hypertension (PAH)• Expected NDA submission Q2 2017• U.S. launch expected in H1 2018, specific timing depending on priority or normal NDA review • Expected Orphan Exclusivity; potential 7 years market exclusivity• Expected MAA submission in Europe second half 2017• Parenteral prostacyclins; high revenue, high margin drug products ~$175,000 per patient, per yr• Potential >$4.0 billion addressable market opportunity in U.S. alone
Early Stage Pipeline; At-Home Patient Analgesia (AHPA) drug product candidates• SMT – 201, Ketorolac, a non opioid, potent NSAID for post surgical pain in the home setting• SMT – 301, Bupivacaine, local anesthetic for post surgical pain in the home setting
Financial Snapshot and Key Investors• Key institutional investors – Federated, Deerfield, OrbiMed, Adage.• Cash and cash equivalents of $23.2 million as of December 31, 2016. • Cash on hand, plus potential $10.7 million second tranche closing of the 2016 PIPE, in Q3 2017,
provides cash runway into Q4 2018
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www.steadymed.com President & CEO; Jonathan Rigby