Q2 2016 Outlook Report - Extract

2 / April 2016 © Informa UK Ltd 2016 (Unauthorized photocopying prohibited.)

April 2016 / 3© Informa UK Ltd 2016 (Unauthorized photocopying prohibited.)
Summary
Our Early 2016 Outlook Report highlighted several high-impact catalysts. The results of these
catalysts can be found on Page 5 of this report. Biomedtracker Likelihood of Approval (LOA)
opinions successfully predicted 78% of catalyst outcomes that occurred in Early 2016.
In this report, we cover catalysts from 21 drugs, devices and diagnostics expected to occur
in Q2 2016. We have also included a list of Large Impact catalysts in the drug and device/
diagnostic areas through Q2 2016 from our sister product, CatalystTracker. The catalyst
calendars are provided in Excel by downloading the supplemental material at the top of this
page. In addition, each drug’s likelihood of Phase/PDUFA review success and overall Likelihood
of Approval (LOA) given their particular phase, drug class, and disease group are provided.
About the Author
Biomedtracker is an independent research service that offers proprietary clinical assessments
and patient-based revenue forecasts of developmental drugs within a comprehensive and
intuitive drug information database. Clients from the pharmaceutical, biotech, and investment
industries rely on Biomedtracker for its insight on the likelihood of approval, commercial
potential, and future data and regulatory catalysts for drugs within the competitive landscape
of every important disease and indication. Over the last several years, Biomedtracker has
become the leader in providing objective information alongside evidence based clinical
assessments and investment research on pipeline drugs worldwide. For more information on
getting direct access to Biomedtracker, please email BioMedSupport@sagientresearch.com.
Meddevicetracker is an all new medtech intelligence platform that provides clients with
real-time data and analysis on medical devices and diagnostics. From the people behind
Biomedtracker, comes an event-driven research service for the medical device and diagnostic
marketplace. For access to Meddevicetracker please contact your sales representative or email
MDTSupport@sagientresearch.com.

Disclaimer
Copyright © 2016 Sagient Research
This report is published by Sagient Research (the Publisher). This report contains information
from reputable sources and although reasonable efforts have been made to publish accurate
information, you assume sole responsibility for the selection, suitability and use of this report
and acknowledge that the Publisher makes no warranties (either express or implied) as to, nor
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contained herein. The Publisher wishes to make it clear that any views or opinions expressed in
this report by individual authors or contributors are their personal views and opinions and do
not necessarily reflect the views/opinions of the Publisher.

Contents
Early 2016 Outlook Catalyst Results.............................................................................................................6
Drugs....................................................................................................................................................................8
NurOwn for Amyotrophic Lateral Sclerosis (ALS) (BCLI)..........................................................................8
LEE011 for Breast Cancer (NVS).....................................................................................................................9
Victoza for Diabetes Mellitus, Type II (NVO)............................................................................................ 11
Semaglutide for Diabetes Mellitus, Type II (NVO).................................................................................. 11
Sofosbuvir/Velpatasvir FDC for Hepatitis C (HCV) (Antiviral) (GILD)................................................... 12
Tivantinib for Hepatocellular (Liver) Cancer (HCC) (ARQL)................................................................... 13
Austedo for Huntington’s Disease (TEVA)................................................................................................ 14
ZS-9 for Hyperkalemia (AZN)....................................................................................................................... 15
Epidiolex for Lennox-Gastaut Syndrome (GW)....................................................................................... 16
Eteplirsen for Muscular Dystrophy (SRPT)................................................................................................ 17
Rociletinib for Non-Small Cell Lung Cancer (NSCLC) (CLVS).................................................................. 18
Niraparib for Ovarian Cancer (TSRO).......................................................................................................... 19
Polyvalent Antigen-KLH Conjugate Vaccine for Ovarian Cancer (MBVX).......................................... 20
Aldoxorubicin for Sarcoma (CYTR)............................................................................................................. 21
Devices & Diagnostics................................................................................................................................... 22
Fantom Bioresorable Scaffold for Coronary Artery Disease (REVA).................................................... 22
DIAM Spinal Stablization System for Disc and Spine Repair (MDT)..................................................... 23
Cartiva SCI for Osteoarthritis (Cartiva)...................................................................................................... 24
AMPLATZER PFO Occluder for Patent Foramen Ovale Repair (STJ)..................................................... 25
Q2 2016 Large Impact Drug/Device Catalyst Calendar........................................................................ 26

Large Impact Catalysts from the Biomedtracker Early 2016 Outlook Report
Occurred Date Lead Company Product Market
2/10/2016 AbbVie Elagolix Obstetrics/ Gynecology
3/28/2016 Alder ALD403 Neurology
2/22/2016 Amgen Romosozumab Endocrine
3/14/2016 Celator Vyxeos Oncology
2/9/2016 Celltrion Inflectra Autoimmune/Immunology
3/17/2016 CymaBay MBX-8025 Cardiovascular
3/22/2016 Eli Lilly Taltz Autoimmune/Immunology
2/16/2016 Flexion Zilretta Rheumatology
3/1/2016 Gilead Odefsey Infectious Disease
3/29/2016 Newron Zadago Neurology
3/30/2016 Opko Rayaldee Endocrine
3/31/2016 PharmaMar Aplidin Oncology
3/24/2016 Portola Betrixaban Hematology
2/26/2016 Roche Gazyva Oncology
1No Previous LOA adjustment

Catalyst Did LOA Predict
Outcome
1-Day Perf 1-Week Perf 1-Month Perf
Phase III Solstice - Top-Line Results Yes -2.43% 2.86% 5.14%
Phase IIb Chronic Migraines - Top-Line
Results
Yes 42.67% 43.83% N/A
Phase III FRAME (PMO) - Top Line Data Yes -1.91% -5.22% 0.33%
Phase III 301 - OS Data Yes 484.31% 534.64% N/A
FDA Advisory Panel Meeting N/A1 N/A N/A N/A
Phase II HoFH - Top-Line Results N/A1 -23.53% -19.41% N/A
PDUFA for BLA - First Review Yes 1.31% 2.06% N/A
Phase III - vs. TCA - Topline Data Yes 22.64% -1.70% -21.89%
PDUFA for NDA - First Review N/A1 1.78% 1.62% 7.87%
PDUFA For NDA - First Review No N/A N/A N/A
PDUFA for NDA - First Review No -6.14% N/A N/A
Phase III ADMYRE - Top-Line Results N/A1 N/A N/A N/A
Phase III APEX - Top-Line Data N/A1 -6.21% -6.80% N/A
PDUFA for sBLA - Rituximab-Refractory Yes 0.49% 0.49% -3.35%

NurOwn for Amyotrophic Lateral Sclerosis (ALS) (BCLI)
Drug Company Partner(s) Indication(s) Date Range Expected
Catalyst(s)
NurOwn BrainStorm
Cell
Therapeutics
Inc.
Amyotrophic
Lateral
Sclerosis
4/01/2016-
06/30/2016
Phase II
BCT-001-
US - Topline
Results
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
II Neurology Biologic 30.77% 16.67% Above
BrainStorm Cell Therapeutics (BCLI) expects top-line results from their Phase II BCT-001-US
study of NurOwn for the treatment of amyotrophic lateral sclerosis (ALS) in the second quarter
of 2016. NurOwn is BCLI’s only therapy in clinical development and positive results from this
study would have a significant impact on their ability to further expand their development
into other CNS disease such as Parkinson’s and Hungtington’s Disease. Currently the only U.S.
approved therapy in ALS is riluzole.
The NurOwn technology functions by processing adult human mesenchymal stem cells that
are present in the bone marrow and are capable of self-renewal and differentiation into many
cell types. The mesenchymal stem cell which secrete neurotrophic factors (MSC-NTF) are then
implanted back into the patient.
In context to this data readout, the Company has reported mixed but encouraging results
from their single-arm, Phase IIa MSC-NTF-002 clinical study of 12 patients in Israel. For the
trial’s secondary endpoint, the study demonstrated that at the six months post-treatment,
forced vital capacity declined from -5.1% per months pre-treatment to -1.2% per month
post-treatment (p=0.036) while ALS Functioning Rating Score-Revised declined from -1.2
points per month pre-treatment to -0.6 points per month post treatment (p=0.052). Thus,
because the Phase II BCT-001-US study also includes ALS Functional Rating Score Scale slopes
as its secondary endpoint, topline results at the second quarter of 2016 will further elucidate
NurOwn’s efficacy in amyotrophic lateral sclerosis in a larger, placebo-controlled trial consisting
of 48 patients.
Drugs

LEE011 for Breast Cancer (NVS)
Catalyst(s)
LEE011 Novartis AG Otsuka Breast Cancer 01/01/2016-
6/30/2016
Phase III
MONALEESA
- Top-Line
Results
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
III Oncology NME 42.48% 34.66% Average
LEE011 (ribociclib) is an orally administered cyclin-dependent kinase (CDK) 4/6 inhibitor
currently in Phase III development for the treatment of breast cancer. CDKs are involved in cell
cycle regulation. Novartis announced that they expect to see top-line Phase III data in the first
half of 2016 for LEE011 in breast cancer.
In January 2014, a randomized double-blind, placebo-controlled Phase III study
(MONALEESA-2) of LEE011 in combination with letrozole for the treatment of postmenopausal
women with hormone receptor positive (HR+), HER2 negative (HER2-), advanced breast cancer
who have received no prior therapy for advanced disease was initiated as part of the larger
MONALEESA program. This study, which enrolled 667 women by early 2015, is the first of many
trials of LEE011 in combination with other agents that Novartis expects to form the bases of
several regulatory applications over the coming years.
Novartis expects the first submission for LEE011 to occur in late 2016 or early 2017, with final
analysis based on the predefined progression free survival (PFS) data from the MONALEESA-2
trial, provided that the requisite number of events occur no later than early in the third quarter
of 2016. We look forward to the initial data from the Phase III MONALEESA-2 study in first line
HR+/HER2- postmenopausal advanced breast cancer, as this will likely pave the way for further
study and regulatory filings of additional combinations of LEE011 for the treatment of breast
cancer.

Victoza for Diabetes Mellitus, Type II (NVO)
Catalyst(s)
Victoza Novo Nordisk
A/S
Diabetes
Mellitus, Type
II
06/10/2016-
06/14/2016
Phase IIIb
– LEADER
- Updated
Results at ADA
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
Approved Endocrine Non-NME N/A N/A N/A
Semaglutide for Diabetes Mellitus, Type II (NVO)
Catalyst(s)
Semaglutide Novo Nordisk
A/S
Diabetes
Mellitus, Type
II
4/1/2016-
6/30/2016
Phase III
SUSTAIN
6 - Top-Line
Results
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
III Endocrine Non-NME 67.95% 58.56% Above
Semaglutide for Diabetes Mellitus, Type II (NVO)
Catalyst(s)
Semaglutide Novo Nordisk
A/S
Diabetes
Mellitus, Type
II
06/10/2016-
6/14/2016
Phase III -
Results at ADA
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
III Endocrine Non-NME 67.95% 58.56% Above

Victoza
Victoza is Novo Nordisk’s daily injected glucagon-like peptide-1 (GLP-1) analogue, which leads
the segment commercially. Interestingly, in March 2016, top-line results from its LEADER
trial, which was required by the FDA to demonstrate cardiovascular (CV) safety, showed a
statistically significant reduction in major adverse CV events (MACE).
Details were not released, however, but will be presented at the upcoming American Diabetes
Association (ADA) conference. It will be important to see how large the reduction in MACE was
– especially since the trial was statistically overpowered due to the need for certain safety data
– as well as whether anything else could have impacted the results, such as blood pressure
reduction.
Officials did say with the top-line release that all three components of the MACE endpoint
contributed to the benefit (CV death, myocardial infarction, and stroke). They are hoping this
impact on atherosclerotic events will differentiate it from the oral SGLT2 inhibitor Jardiance,
which showed a benefit primarily on CV death and heart failure hospitalization. Some have
postulated that, in addition to a benefit in heart failure patients, Jardiance’s effect could be
more related subclinical left ventricular dysfunction. This can be common in diabetics, though
it is not certain what type of dysfunction could be impacted by the drug (eg a large percent is
diastolic dysfunction, and another question is whether patients would need to have a degree of
dysfunction caused by a myocardial infarction to benefit).
It is not entirely clear how Victoza impacts atherosclerosis, so it will be interesting to see
if there is more information at the conference. Officials have touted the potential multiple
mechanisms that Victoza has shown preclinically, including an impact on a number of
metabolic parameters, inflammation, pro-coagulant activity, and other effects.
Semaglutide
Semaglutide is Novo Nordisk’s an injected once-weekly glucagon-like peptide-1 (GLP-1)
analogue for type 2 diabetes, intended to meet the challenge from other weekly GLP-1
agonists to Victoza.
Five of the SUSTAIN trials have reported top-line data, with signs (from indirect comparisons)
that semaglutide could be more effective than Victoza, possibly without substantially
increasing nausea, though more details are needed on other symptoms, such as vomiting. Per
a company representative, data from at least one of the trials may be at the upcoming ADA
meeting, so it will be useful to get more details on its profile.
SUSTAIN 6’s has a much larger patient population that will yield relatively more long term
results. The study involves patients with clinical or subclinical cardiovascular disease and was
initiated to demonstrate CV safety for the FDA. Top-line results are expected in the second
quarter.

Since semaglutide is fairly similar in structure to Victoza, it will be interesting to see if there are
any signs of similar trends for a CV benefit, though officials may not present that much detail
unless it shows superiority. A company official acknowledged that SUSTAIN 6 study does not
have the statistical power that LEADER did and a larger trial is likely to needed to demonstrate
a CV benefit. Based on a simple statistical estimate, BMT estimates it could possibly be
significant if the risk reduction is more than around 14%.
Novo Nordisk also has an oral formulation of semaglutide in development, though some doses
have shown a somewhat high rate of vomiting.
Sofosbuvir/Velpatasvir FDC for Hepatitis C (HCV) (Antiviral) (GILD)
Catalyst(s)
Sofosbuvir/
Velpatasvir
FDC
Gilead
Sciences, Inc.
Hepatitis C
(HCV) Antiviral
6/28/2016 PDUFA for
NDA - First
Review
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
NDA Infectious
diseases
NME 87.84% 75.00% Above
Sofosbuvir/GS-5816 is a single pill combination of the prodrug Sofosbuvir and the NS5A
replication inhibitor GS-5816. Sofosbuvir/Velpatasvir FDC(SOF/VEL) was granted Breakthrough
Therapy designation as well as priority review for the New Drug Application submitted October
28, 2015. The U.S. Food and Drug Administration (FDA) has set a target action date under the
Prescription Drug User Fee Act (PDUFA) of June 28, 2016 for Gilead’s New Drug Application
(NDA) for SOF/VEL for the treatment of chronic genotype 1-6 hepatitis C virus (HCV) infection.
The NDA for SOF/VEL is supported by data from four Phase III ASTRAL trials, which evaluated
the fixed-dose combination in hepatitis C genotypes 1-6. Of the 1,035 patients treated with
SOF/VEL for 12 weeks in the ASTRAL-1, ASTRAL-2 and ASTRAL-3 studies, 1,015 (98 percent)
achieved the primary efficacy endpoint of SVR12. The ASTRAL-4 study randomized 267
patients with decompensated cirrhosis (Child-Pugh class B) to receive 12 weeks of SOF/VEL
with or without ribavirin (RBV), or 24 weeks of SOF/VEL. Those who received SOF/VEL plus RBV
for 12 weeks achieved an SVR12 rate of 94 percent, while those who received SOF/VEL for 12
weeks and 24 weeks achieved SVR12 rates of 83 percent and 86 percent, respectively. Overall,
treatment with SOF/VEL for 12 weeks was well tolerated and resulted in high SVR4 rates in
patients with genotype 1-6 HCV infection.

Due to its earlier successes in virtually all patient populations, we anticipate that the U.S. Food
and Drug Administration will grant approval to Sofosbuvir/GS-5816 on the first review.
Tivantinib for Hepatocellular (Liver) Cancer (HCC) (ARQL)
Catalyst(s)
Tivantinib ArQule, Inc. Daiichi
Sankyo,
Kyowa Hakko
Kirin
Hepatocellular
(Liver) Cancer
(HCC)
04/01/2016-
05/15/2016
Phase III
METIV -HCC
- Interim
Analysis
Phase Disease
Group
Drug Class Group/
Class Phase
Success
Group/Class
LOA (PTS)
BMT LOA
Opinion
III Oncology NME 42.11% 34.30% Above
Tivantinib (ARQ 97) is a small molecule, orally available, selective inhibitor of c-Met, a tyrosine
kinase receptor for hepatocyte growth factor that is thought to play multiple key roles in
human cancer, including cancer cell growth, survival, angiogenesis, invasion and metastasis.
Hepatocellular cancer (HCC) is the latest indication in which tivantinib is being developed.
In a Phase II study in unresectable HCC patients who had failed one prior systemic therapy
(predominantly sorafenib), MET-diagnostic high cohort patients showed significant
improvements in progression free survival (PFS) and overall survival (OS). Median PFS and OS
in the tivantinib versus placebo arms were 2.4 versus 1.5 months, and 7.2 versus 3.8 months,
respectively.
Given the large unmet need in sorafenib-refractory patients, and the increase in survival in a
clearly defined patient population, tivantinib is likely to be approved for Met-high patients in
the refractory setting if the Phase II results are confirmed. The Phase III study of tivantinib in a
larger Met-high population is thus pivotal in determining whether tivantinib will be approved in
HCC, which is one of seven pursued indications. Phase III interim results are expected early in
the second quarter of 2016.

This is a sample of the 52 page Outlook Report.
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