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October 2017 / 1
Biomedtracker Q4 2017 Outlook Report
Q4 2017 Q4 2017
OUTLOOK REPORT
October 2017 / 2
Biomedtracker Q4 2017 Outlook Report
Summary In this report, we cover catalysts from 21 drugs expected to occur in Q4 2017. For each drug, the likelihood of Phase/PDUFA review success and overall Likelihood of Approval (LOA) given their particular phase, drug class, and disease group are provided. The results of the catalysts highlighted in our Q3 2017 Outlook Report can be found on Page 4. At the end of this report, we have included a list of Large Impact catalysts through Q4 2017. The catalyst list is also provided in Excel by downloading the supplemental material at the top of this page. Like our report? Have any questions or feedback? Please let us know at [email protected].
About the Author Biomedtracker is an independent research service that offers proprietary clinical assessments 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 recent 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 [email protected].
Disclaimer Copyright © 2017 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 accepts liability for, the accuracy or fitness for a particular purpose of the information or advice 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.
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Biomedtracker Q4 2017 Outlook Report
Contents Outcomes of Biomedtracker's Large Impact Catalysts from the Q3 2017 Outlook Report ........................... 4
Tirasemtiv for Amyotrophic Lateral Sclerosis (ALS) (CYTK) ............................................................................ 6
Auryxia for Anemia Due to Chronic Renal Failure, Dialysis-Independent (KERX) ........................................... 7
Benralizumab for Asthma (AZN) .................................................................................................................... 7
Simponi Aria (IV) for Axial Spondyloarthritis (JNJ) ......................................................................................... 9
Simponi Aria (IV) for Psoriatic Arthritis (PA) (JNJ) .......................................................................................... 9
Adcetris for Cutaneous T-Cell Lymphoma (CTCL) – NHL (SGEN) ................................................................. 10
Letermovir for Cytomegalovirus (CMV) Infection (Antiviral) (MRK) ............................................................ 11
Intepirdine for Dementia (AXON) ................................................................................................................ 12
Dengvaxia for Dengue Fever - Vaccines and Treatments (SNY) ................................................................... 13
Axicabtagene Ciloleucel for Diffuse Large B-Cell Lymphoma (DLBCL) - NHL (KITE) ..................................... 14
Emicizumab for Hemophilia A (RHHBF) ....................................................................................................... 15
Heplisav for Hepatitis B Prevention (Vaccines, Antiviral) (DVAX) ................................................................ 16
Tenapanor for Irritable Bowel Syndrome (IBS) (ARDX) ................................................................................ 17
Firdapse for Lambert-Eaton Myasthenic Syndrome (LEMS) (CPRX) ............................................................ 18
Luxturna for Leber's Congenital Amaurosis (Ophthalmology) (ONCE) ........................................................ 19
Dsuvia for Moderate to Severe Pain (ACRX) ................................................................................................ 20
UX003 for Mucopolysaccharidosis VII (MPS VII; Sly Syndrome) (RARE) ...................................................... 20
GR-MD-02 for Non-Alcoholic Steatohepatitis (NASH) (GALT) ...................................................................... 21
Macrilen for Short Stature / Growth Hormone Deficiency (AEZS) ............................................................... 22
Iclaprim (IV) for Skin and Skin-Structure Infections (Antibacterial) (MTFB) ................................................. 23
RBP-6000 for Substance Use Disorder (Indivior) ......................................................................................... 24
Alicaforsen for Ulcerative Colitis (UC) (Atlantic) .......................................................................................... 25
Q4 2017 Large Impact Drug Catalysts……………..…..………………..…………………………………………………………………26
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Biomedtracker Q4 2017 Outlook Report
Outcomes of Biomedtracker's Large Impact Catalysts from the Q3 2017 Outlook Report Occurred
Date Lead Company Product Market Catalyst Did LOA Predict
Outcome
LOA Before Outcome
LOA After Outcome
7/7/2017 Emmaus Endari Hematology PDUFA for NDA - First Review Yes 89%
(5% Above Avg.) 100%
(Approved)
7/11/2017 Pfizer Mylotarg Oncology FDA Advisory Panel Meeting No 77%
(5% Below Avg.) 89%
(7% Above Avg.)
7/12/2017 Novartis Kymriah Oncology FDA Advisory Panel Meeting Yes 93%
(11% Above Avg.) 95%
(13% Above Avg.)
7/17/2017 Paratek Omadacycline Infectious Disease
Phase III Oral-only - Top-line Results Yes 67%
(6% Above Avg.) 72%
(11% Above Avg.)
7/17/2017 Puma Nerlynx Oncology PDUFA for NDA - First Review Yes 90%
(8% Above Avg.) 100%
(Approved)
7/27/2017 AstraZeneca Imfinzi Oncology Phase III MYSTIC - Topline Results No 42%
(7% Above Avg.) 32%
(3% Below Avg.)
8/7/2017 Valeant Vyzulta Ophthalmology PDUFA for NDA - Second Review N/A 90%
(11% Above Avg.) 90%
(11% Above Avg.)
8/24/2017 Adamas Gocovri Neurology PDUFA for NDA - First Review Yes 95%
(12% Above Avg.) 100%
(Approved)
8/30/2017 Teva Austedo Neurology PDUFA for sNDA - First Review Yes 91%
(8% Above Avg.) 100%
(Approved)
9/8/2017 Roche Lampalizumab Ophthalmology Phase III Spectri - Top-Line Results No 55%
(4% Above Avg.) 43%
(8% Below Avg.)
9/11/2017 AbbVie Upadacitinib Autoimmune/ Immunology
Phase III SELECT-BEYOND - Top-Line Results
Yes 67%
(8% Above Avg.) 68%
(9% Above Avg.)
9/12/2017 SAGE Brexanolone Neurology Phase III STATUS - Top-Line Results No 52%
(Same As Avg.) 0%
(Suspended)
9/13/2017 Amicus Zorblisa Metabolic Phase III SD-005 - Top-Line Results No 64%
(2% Above Avg.) 0%
(Suspended)
9/18/2017 OptiNose Xhance Allergy PDUFA for NDA - First Review Yes 91%
(2% Above Avg.) 100%
(Approved)
9/20/2017 Alnylam Patisiran Metabolic Phase III APOLLO - Top-Line Results No 61%
(1% Below Avg.) 76%
(14% Above Avg.)
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Biomedtracker Q4 2017 Outlook Report
9/22/2017 Johnson & Johnson Sirukumab Autoimmune/ Immunology
PDUFA for BLA - First Review Yes 74% (13% Below Avg.)
41% (18% Below Avg.)
9/26/2017 Axovant Intepirdine Neurology Phase III MINDSET - Top-Line
Results No
54% (2% Above Avg.)
0% (Suspended)
9/28/2017 PTC Translarna Metabolic FDA Advisory Panel Meeting Yes 78% (11% Below Avg.)
77% (12% Above Avg.)
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Biomedtracker Q4 2017 Outlook Report
Tirasemtiv for Amyotrophic Lateral Sclerosis (ALS) (CYTK)
Drug Company Partner(s) Indication(s) Date Range Expected Catalyst(s)
Tirasemtiv Cytokinetics, Inc. N/A Amyotrophic Lateral Sclerosis
12/08/2017 - 12/10/2017
Phase III VITALITY ALS - Top-Line
Results
Phase Disease Group Drug Class Group/Class Phase Success
Group/Class LOA (PTS)
BMT LOA Opinion
III Neurology NME 43.28% 35.68% Below Tirasemtiv, in development by Cytokinetics, is a fast skeletal muscle troponin activator being developed as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) and certain other debilitating diseases and conditions associated with muscle weakness and fatigue or neuromuscular dysfunction. Tirasemtiv has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Medicines Agency. To date, Cytokinetics has conducted the Phase IIb BENEFIT-ALS clinical trial to evaluate the safety, tolerability and efficacy of tirasemtiv in patients with ALS. The study did not achieve its primary efficacy endpoint of mean change from baseline in the ALS Functional Rating Scale -Revised (ALSFRS-R) total score obtained at Visits 6 and 7 (tirasemtiv versus placebo) while secondary endpoints evaluating measures of respiratory performance and other measures of skeletal muscle function and fatigability produced mixed results. This outcome proved to be devastating for Cytokinetics, especially after the failure of its entire oncology pipeline in 2013 as well as poor Phase IIb results for its heart failure drug, omecamtiv mecarbil (under partnership with Amgen). Currently, Tirasemtiv is being re-evaluated in another Phase III study, VITALITY-ALS, which completed enrollment in August 2016. The trial is designed to assess its effects versus placebo on measures of respiratory function and muscle strength in people with ALS using an increased dosage compared to BENEFIT-ALS. Early safety data suggested an increase in dizziness associated with higher doses, so the increased dose could prove problematic. Results from VITALITY-ALS are expected in the fourth quarter of 2017, preferably at the International Symposium on ALS/MND on December 8-10, 2017. Tirasemtiv appears to be the best hope for the Company’s relatively small pipeline, and the upcoming results will prove pivotal for the outlook of Tirasemtiv and Cytokinetics.
