Investor Presentation March 2016 Forward Looking Statement This - - PowerPoint PPT Presentation
Investor Presentation March 2016 Forward Looking Statement This - - PowerPoint PPT Presentation
Investor Presentation March 2016 Forward Looking Statement This presentation contains forward-looking statements about our expectations, beliefs or intentions regarding, among other things, our product development efforts, business, financial
This presentation contains forward-looking statements about our expectations, beliefs or intentions regarding, among other things, our product development efforts, business, financial condition, results of operations, strategies or prospects. In addition, from time to time, we or our representatives have made or may make forward-looking statements, orally or in writing. Forward-looking statements can be identified by the use of forward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. These forward-looking statements may be included in, but are not limited to, this presentation, various filings made by us with the SEC, press releases or oral statements made 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, these statements are inherently subject to risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward-looking statements. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated 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 commercial launch and future sales of aramchol or any other future products or product candidates; our ability to achieve favorable pricing for aramchol; our expectations regarding the commercial market of NASH in patients who also suffer from obesity and insulin resistance; third-party payor reimbursement for aramchol; our estimates regarding anticipated capital requirements and our needs for additional financing; patient market size and market adoption of aramchol by physicians and patients; the timing, cost or other aspects of the commercial launch of aramchol; the timing and cost
- f 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 and
Phase III trials for aramchol; the development and approval of the use of AramcholTM for additional indications or in combination therapy; and our expectations 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 our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking
- statements. 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 the date hereof and are expressly qualified in their entirety by the foregoing. We undertake no obligations to update or revise forward-looking statements to reflect events or circumstances that arise after the date made or to reflect the occurrence of unanticipated events. In evaluating forward-looking statements, 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
- f 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 $9.29 2 2 10/~55 3 $5.18
2
2
Macro Environment: Galmed:
Screened Subjects for ARREST Study
>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
- bserved 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) Demonstrated ability 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
- f NASH to life-
threatening liver disease through resolution of NASH as measured by disappearance of ballooning (biopsy)
- Treat the underlying
condition, 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
- f ballooning
(biopsy) Metabolic indices, showed trends of improvement Significant improvement 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 6 30 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
- f 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
Chronic Toxicology (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
- f 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
- f 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 IIb Ongoing Phase III < PoC (noteworthy)
NASH
80% of Market
Fibrosis
20% of Market
(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 accumulation In patients with HIV, liver disease is among the leading causes of death Nearly half of the HIV infected patients without viral hepatitis that undergo evaluation for unexplained liver test abnormalities are found to have NAFLD The 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 few The first step in extrapolating additional commercial value with
- ur existing assets
Signifies 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
Biologic / Peptide Small Molecule
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
Ongoing In Submission or <6 Months
- 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.com for more information
Thank you!
Josh Blacher, CFO 16 Tiomkin Street, Tel Aviv 6578317 Israel T: +1.646.780.7605 | M: +972.52.770.2655 josh@galmedpharma.com