investor presentationgalmedpharma.investorroom.com/...+march+2016+... · 2015: a year of execution...
TRANSCRIPT
Investor PresentationMarch 2016
This presentation contains forward-looking statements about our expectations, beliefs or intentions regarding, among other things, our productdevelopment efforts, business, financial condition, results of operations, strategies or prospects. In addition, from time to time, we or ourrepresentatives have made or may make forward-looking statements, orally or in writing. Forward-looking statements can be identified by the use offorward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of thesewords or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. These forward-lookingstatements may be included in, but are not limited to, this presentation, various filings made by us with the SEC, press releases or oral statementsmade by or with the approval of one of our authorized executive officers. Forward-looking statements relate to anticipated or expected events,activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, thesestatements are inherently subject to risks and uncertainties that could cause our actual results to differ materially from any future results expressed orimplied by the forward-looking statements. Many factors could cause our actual activities or results to differ materially from the activities and resultsanticipated in forward-looking statements, including, but not limited to, the factors summarized below.
These factors include, but are not limited to, the following: FDA approval of, or other regulatory action with respect, to AramcholTM; the commerciallaunch and future sales of aramchol or any other future products or product candidates; our ability to achieve favorable pricing for aramchol; ourexpectations regarding the commercial market of NASH in patients who also suffer from obesity and insulin resistance; third-party payorreimbursement for aramchol; our estimates regarding anticipated capital requirements and our needs for additional financing; patient market size andmarket adoption of aramchol by physicians and patients; the timing, cost or other aspects of the commercial launch of aramchol; the timing and costof Phase IIb and Phase III trials for aramchol or whether such trials will be conducted at all; completion and receiving favorable results of Phase IIb andPhase III trials for aramchol; the development and approval of the use of AramcholTM for additional indications or in combination therapy; and ourexpectations regarding licensing, acquisitions and strategic operations.
These statements are only current predictions and are subject to known and unknown risks, uncertainties and other factors that may cause our or ourindustry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-lookingstatements. Given these uncertainties, you should not rely upon forward-looking statements as predictions of future events.
All forward-looking statements attributable to us or persons acting on our behalf included in, but not limited to, this presentation speak only as of thedate hereof and are expressly qualified in their entirety by the foregoing. We undertake no obligations to update or revise forward-looking statementsto reflect events or circumstances that arise after the date made or to reflect the occurrence of unanticipated events. In evaluating forward-lookingstatements, you should consider these risks and uncertainties.
Forward Looking Statement
AramcholTM addresses a significant, and growing unmet need in the U.S., EU & RoW – Non-alcoholic steatohepatitis (NASH) and other liver-related diseases
Focused Strategy, Broad Vision
First in a new class of drug candidates with proof-of-concept as demonstrated in Phase I & IIa clinical trials; no serious or drug-related adverse events observed
Novel Technology
Completed pre-clinical and 4 clinical trials (Phase Ia/Ib, PK Food effect & Phase IIa … on time and under budget)
Strong Track Record of Execution
~10% population in U.S. & EU-5 nations has NASH; prevalence expected to rise in parallel with obesity and diabetes. No approved drugs; adding incremental, significant shots-on-goal
Significant Market Opportunity
The Galmed Story
1
GLMD trades significantly below comparable companies. Exceptionally modest Enterprise Value
Compelling Valuation
2015: A Year of Execution & Clinical Progress
A Year Ago Today
Regulatory Pathway
Clinical Trials Initiated
Clinical Infrastructure (Countries / Sites)
Additional PoC Clinical Trials In Advanced Formation
Share Price
X
0
0/0
0
$9.29
2
2
10/~55
3
$5.18
22
Macro Environment:
Galmed:
Screened Subjects for ARREST Study
0 >400
AramcholTM
First-in-Class
Potentially disease modifying
Novel; Strong Intellectual Property portfolio
Synthetic conjugate of ARAchidic acid (fatty), and CHOLic acid (bile)
No serious or drug-related adverse events observed to date
Orally administered
3
How is AramcholTM Unique?
1. Target the underlying CAUSE of the disease – excess fat in the liver
2. Addresses both the hepatic and metabolic parameters of NASH
3. No serious or drug-related adverse events observed to date
4
A Visible Reduction in Lipid Deposits & Ballooning (In-Vivo)
Gilat et. al., HEPATOLOGY, Vol. 38, No. 2, 2003
Fatty Liver
Fatty Liver Treated with AramcholTM
Rodents in this study were treated with AramcholTM for ten weeks
5
AramcholTM: Results-to-date in, and Future Objectives of Clinical Trials
Reduction in liver fat
Resolution of NASH (hepatocyte
ballooning)
Effect on metabolic syndrome
Safety profile
ArmacholTM
(TPP)Phase I Phase II Phase IIb Phase III
Not evaluated (goal of Phase I study is safety screening)
Demonstratedability to significantly reduce liver fat content
Confirmation of effect on reducing liver fat content
Efficacy and safety to be confirmed in pivotal Phase III trials
• Prevent progression of NASH to life-threatening liver disease through resolution of NASH as measured by disappearance of ballooning (biopsy)
• Treat the underlyingcondition, metabolic syndrome, by improving insulin resistance and other parameters of the metabolic syndrome
• High safety profile for chronic (1X daily) dosing
Trend of improvement in adiponectin, ALT HOMA and other liver-function parameters
Resolution of NASH as measured by disappearance of ballooning(biopsy)
Metabolicindices, showed trends of improvement
Significantimprovement in metabolic syndrome
No notable changes in safety parameters
No severe drug-related adverse events observed
Confirmation of clean safety profile
6
Phase IIa : Statistically Significant Reduction in Liver Fat Content
7
Clinical Gastroenterology and Hepatology 2014: The Fatty Acid–Bile Acid Conjugate Aramchol Reduces Liver Fat Content in Patients With Nonalcoholic Fatty Liver Disease; Drs. Tuvia Gilat, Maya Halpern, Ran Oren, et. al.; 58 patients completed the study.
Phase IIa: Enhanced Adiponectin Levels
8
Clinical Gastroenterology and Hepatology 2014: The Fatty Acid–Bile Acid Conjugate Aramchol Reduces Liver Fat Content in Patients With Nonalcoholic Fatty Liver Disease; Drs. Tuvia Gilat, Maya Halpern, Ran Oren, et. al.; 58 patients completed the study.
Phase IIa: Improvement in Endothelial Function
9
Clinical Gastroenterology and Hepatology 2014: The Fatty Acid–Bile Acid Conjugate Aramchol Reduces Liver Fat Content in Patients With Nonalcoholic Fatty Liver Disease; Drs. Tuvia Gilat, Maya Halpern, Ran Oren, et. al.; 58 patients completed the study.
10
Phase IIa: Marked Improvement in Liver Function (ALT)
-12.0
-10.0
-8.0
-6.0
-4.0
-2.0
0.0
Visit 2 Visit 3 Visit 4 Visit 5 Visit 630 days post
treatment
ALT
(U
/L)
Placebo (n=19)
Aramchol 100 mg/d (n = 18)
Aramchol 300 mg/d (n = 20)
Immediate relapse in ALT Improvement following conclusion
of treatment
Clinical Gastroenterology and Hepatology 2014: The Fatty Acid–Bile Acid Conjugate Aramchol Reduces Liver Fat Content in Patients With Nonalcoholic Fatty Liver Disease; Drs. Tuvia Gilat, Maya Halpern, Ran Oren, et. al.; 58 patients completed the study.
Summary of Safety Data
11
Study N Summary of Safety Results
ChronicToxicology
(non-clinical)
No AEs and minimal toxicity following single doses (750 mg/kg); repeat dose studies in rats up to 6-months (1000 mg/kg) and in dogs up to 9-months (1500 mg/kg); reproductive studies in rats (1000 mg/kg) and rabbits (750 mg/kg). NOAEL determined at highest dose tested for all studies; didn’t reach MTD
Phase I 41
Single doses of AramcholTM from 30 mg to 900 mg and repeated dose of 100 mg, 300 mg were found to be safe and well tolerated in healthy male subjects
Phase IIa 57
No severe AEs during the 3-month treatment period. Mild AE in 6 patients were mild or moderate and transient, and did not differ between the placebo and treated groups
PK /Food Effect
66
All doses of AramcholTM were safe and well tolerated. No serious AEs. Most of AEs were mild and unrelated to AramcholTM and all AEs were transient and gave no indication of target organ toxicity
Design:•Multicenter, randomized, double-blind, placebo-controlled, dose
ranging study
Participants: • Biopsy-diagnosed NASH patients with obesity and insulin resistance
Doses:• Placebo (62 patients)• 400 mg (89 patients)• 600 mg (89 patients)
Treatment Plan:
• 12 months treatment (once-daily tablet) and 3 months of follow-up • Interim analysis planned on first 120 patients completing 6 months
of treatment (1H16). Top-line results expected in 2H17
Number of Subjects (Est.):
• 240 patients• ~70 sites in U.S., Europe, Latin America and Israel
Primary Endpoint:
• Statistically significant reduction in liver fat content measured by MRS
Secondary Endpoints:
• Resolution of NASH (ballooning); no worsening of fibrosis – both as measured by biopsy• Improvement of liver and metabolic markers
ARREST Study
12
NASH: Addressable Market (US + EU-5)
Adult Population (441m)
NAFLD (167m)
NASH (45m)
Diagnosed (5.5m)
Treated (1.6m)
13
Source: Deutsche Bank by 2025
NASH: Competitive Landscape
14
Phase IIbOngoing
Phase III< PoC(noteworthy)
NA
SH8
0%
of
Ma
rket
Fib
rosi
s2
0%
of
Ma
rket
(Cirrhosis; IV Formulation)
ARREST Status Update: Patients Screened
15
-
50
100
150
200
250
300
350
400
450
Europe/Israel United States Latin America
Selecting Additional Pipeline Indications
16
Strong Scientific Foundation
Moderate Competition
and Clear TPP Advantage
>$1.0B+ Commercial
Potential
ARRIVE Study
17
Design:
• Randomized, double-blinded, allocation-concealed, placebo-controlled, proof-of-concept Phase IIa clinical trial, which is an investigator-initiated study, conducted at the University of California San Diego by Professor Rohit Loomba
Participants:•Up to 50 patients with HIV-associated lipodystrophy and
nonalcoholic fatty liver disease
Doses:• Placebo (up to 25 patients)• 600 mg (up to 25 patients)
Treatment Plan:
• 12 weeks treatment (once-daily tablet) and 1 month of follow-up • Top-line results in 2H17
Primary Endpoint:
• Improvement in hepatic steatosis as measured by MRI
Secondary Endpoints:
• An improvement in total body fat, metabolic profile, and liver biochemistry
Strategic & Scientific Rationale
Scientific rationale:HIV-associated lipodystrophy refers to abnormal, abdominal visceral fat accumulationIn patients with HIV, liver disease is among the leading causes of deathNearly half of the HIV infected patients without viral hepatitis that undergo evaluation for unexplained liver test abnormalities are found to have NAFLDThe prevalence of NAFLD is higher in individuals with HIV infection than in the general population
Strategic rationale:Like NASH, there are no therapies for the treatment of HIV-associated NAFLD and clinical trials in this area have been fewThe first step in extrapolating additional commercial value with our existing assetsSignifies the beginning of our de-risking strategy through more potential applications and end markets for our product
18
HIV-Lipo & NAFLD: Addressable Market
HIV Population US + EU-5a,b,c (2.0m)
… With Lipodystrophyd (785k)
… Who Require Treatmentd (471k)
AramcholTM Market Sharee – 40% (188k)
Projected Peak Annual Salese (~$1.0B)
19
Sources: (a) CDC, (b) WHO, (c)Global Markets Direct (Lipodystrophy – Pipeline Review, H2 2015), (d) Epocrates, and (e)GLMD estimates.
HIV-Associated LD: Competitive Landscape
Phase II >=Phase III<=Phase I
Bio
log
ic /
Pep
tid
eSm
all
Mo
lecu
le
20
Maximize Commercial Potential of Existing Assets
21
NASH
HIV-Associated Lipodystrophy & NAFLD
Cardiovascular TBA – Phase IIa
Juvenile NAFLD/NASH – Phase I/II
Fibrotic NASH (Combination) – Phase IIa
On
go
ing
In Su
bm
ission
or <
6 M
on
ths
• IND/IIT•Funded•PoC Trials
Strong Cash Position; Clean Balance Sheet
22
(Figures in millions, unless otherwise noted) Current Share Price Appreciation:
Company Ticker Share Price
Market
Value
Enterprise
Value YTD 2016
Trailing 30
Days 2015
52-Week
High
52-Week
Low
Conatus CNAT 2.24$ 45$ 2$ -22% 22% -59% -74% 60%
Galectin GALT 1.52$ 44$ 30$ -7% 30% -53% -63% 41%
Genfit PA GNFT-FR 32.95$ 868$ 721$ -7% 11% -22% -48% 46%
Intercept ICPT 128.45$ 3,135$ 2,439$ -14% 30% -4% -59% 43%
Tobira TBRA 7.70$ 145$ 98$ -23% 12% 22% -68% 27%
Average -15% 21% -23% -62% 43%
Median -14% 22% -22% -63% 43%
MC-Weighted -13% 26% -8%
Galmed GLMD 5.18$ 57$ 32$ -32% -15% 31% -62% 22%
NASDAQ Composite COMP-USA 4,708 -6% 8% 6% -10% 12%
NASDAQ Health Care IXHC-USA 631 -16% 6% 7% -27% 11%
NASDAQ Biotechnology NBI 2,821 -20% 5% 11% -33% 12%
Russell 2000 Index RUT^ 1,094 -4% 11% -6% -16% 16%
Market data as of 3/7/2016.
Comparable Company Analysis
23
$13.50
Execution Focused; Upcoming Near- and Mid-Term Milestones
24
1Q15
Begin enrollment in Europe
First-Patient-In Phase IIa ARRIVE
Study
ARREST Study interim results on 120 patients for 6
months of treatment
Unfold the Development of
Non-Invasive Companion Diagnostic Program
2Q15 3Q15 4Q15 1Q16 2Q16
Initiate ARREST Study; begin enrollment in
Israel; Expansion Clin. Ops. in the US
Begin enrollment in US and LatAm
Initiation of Phase IIa PoC clinical trial in new indication
Business Development
Announce developments in
combination therapy for
advanced (fibrotic) NASH
Mergers & Acquisitions
Over the two years, there have been five NASH assets acquired/licensed by BigPharma – all at earlier stages of development than AramcholTM –for escalating deal values:
25
Economics
(USD in MMs)
Seller Buyer Asset Mechanism
Transaction
Structure Date
Stage of
Development Upfront Total
Lumena Shire LUM002 (Phase
I, NASH)
ASBT Inhibitor Acquisition 5/12/14 Phase I (LUM002) 260$ 600$
Galecto BMS TD-139 Galectin-3
Inhibitor
License 11/3/14 Phase I N/A 444$
Phenex Gilead Px-102 FXR Agonist Asset Acquisition 1/6/15 Phase II N/A 470$
Regulus AstraZeneca RG-125 miR-103/107
Inhibitor
License 4/7/15 Preclinical N/A 500$
Pharmaxis Boehringer
Ingelheim
PXS478A SSAO/VAP-1
Inhibitor
Asset Acquisition 5/18/15 Preclinical 31$ 840$
Current Analyst Recommendations
26
Bank Analyst Rating Price Target
SunTrust Edward Nash Buy $19
ROTH Elemer Piros, Ph.D. Buy $20
Maxim Jason Kolbert Buy $24
FBR Vernon T. Bernardino Buy $24
H.C. Wainwright Yi Chen, Ph.D. CFA Buy $21
Please visit www.galmedpharma.comfor more information
Thank you!
Josh Blacher, CFO 16 Tiomkin Street, Tel Aviv 6578317 Israel
T: +1.646.780.7605 | M: +972.52.770.2655 [email protected]