Hypertrophic Cardiomyopathy-Pipeline Intelligence, 2019

1. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019

2. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Introduction CmaxInsight’s, “Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019”, report provides comprehensive insights about pipeline drugs across this Indication. A key objective of the report is to establish the understanding for all the pipeline drugs that fall under Hypertrophic Cardiomyopathy. Highlights and Scope of the Report • Establish a comprehensive understanding of key competitor information, analysis, and insights to improve R&D strategies • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage • Discover in licensing and out licensing strategies by identifying potential partners with progressing projects for Hypertrophic Cardiomyopathy to enhance and expand business potential and scope • Plan mergers and acquisitions successfully by identifying major players with the most promising pipeline therapeutics in the target demographic • Our extensive in-depth analysis on therapy portfolio support the client in decision- making process regarding their therapeutic portfolio by identifying the reason behind the dormant or discontinued drugs • Develop strategic initiatives by understanding the focus areas of leading companies. • Assess challenges and opportunities that influence Hypertrophic Cardiomyopathy R&D The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by CmaxInsight’s team of industry experts. Secondary sources information and data has been collected from various printable and non- printable sources like search engines, News websites, Government Websites, Trade Journals, White papers, Magazines, Trade associations, Books, Industry Portals, Industry Associations and access to available databases. Please note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated mechanism of action. 2 ©CmaxInsight|This report is a licensed product and is not to be photocopied

3. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Executive Summary Xxxxxxx Key findings from the report include the following: Xxxxxxxx 3 ©CmaxInsight|This report is a licensed product and is not to be photocopied

4. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Executive Summary Snapshot Hypertrophic Cardiomyopathy Snapshot Most Promising Emerging Drug Candidate Companies Active Companies Leading Firms Xxxxx Pipeline Activity 28% 72% 28% 6 5 6 11 6 7 1 Phase I Phase II Phase III IND Discovery Pre-Clinical Leading firms Most Prominent Target Xxxxx Xxxxx Key Market Drivers Key Market Barriers Xxx Xxxxx Key Pipeline Developments Pre-Clinical Xxx Phase-I Xxx Phase-II Xxx Phase-III Xxx 4 ©CmaxInsight|This report is a licensed product and is not to be photocopied

5. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Table of Contents Introduction .................................................................................................................... 1 Executive Summary .......................................................................................................... 3 Executive Summary Snapshot ........................................................................................... 4 Disease Overview ............................................................................................................. 8 Epidemiology.......................................................................................................................... 8 Causes .................................................................................................................................... 8 Symptoms .............................................................................................................................. 8 Pathophysiology ..................................................................................................................... 8 Diagnosis ................................................................................................................................ 8 Treatment .............................................................................................................................. 8 Competitive Landscape ..................................................................................................... 9 Comparative Analysis ............................................................................................................. 9 Pipeline Therapeutics ...................................................................................................... 10 Late Stage Products (Filed & Phase III) ............................................................................. 11 Comparative Analysis ........................................................................................................... 11 Mavacamten (MYK-461): MyoKardia ............................................................................... 12 Product Description ............................................................................................................. 12 Research and Development ................................................................................................. 12 Product Development Activities .......................................................................................... 15 Key Development Milestone ............................................................................................... 15 Mid Stage Products (Phase II) .......................................................................................... 17 Comparative Analysis ........................................................................................................... 17 Drug name: Company name ............................................................................................ 18 Product Description ............................................................................................................. 18 Research and Development ................................................................................................. 18 Product Development Activities .......................................................................................... 18 Key Development Milestone ............................................................................................... 18 Early Stage Products (Phase I) .......................................................................................... 20 Comparative Analysis ........................................................................................................... 20 Drug name: Company name ............................................................................................ 21 Product Description ............................................................................................................. 21 Research and Development ................................................................................................. 21 Product Development Activities .......................................................................................... 21 Key Development Milestone ............................................................................................... 21 Pre-clinical and Discovery Products .................................................................................. 23 Comparative Analysis ........................................................................................................... 23 Drug name: Company name ............................................................................................ 24 Product Description ............................................................................................................. 24 Research and Development ................................................................................................. 24 5 ©CmaxInsight|This report is a licensed product and is not to be photocopied

6. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Product Development Activities .......................................................................................... 24 Key Development Milestone ............................................................................................... 24 Therapeutic Assessment .................................................................................................. 26 Assessment by Route of Administration.............................................................................. 26 Assessment by Stage and Route of Administration ............................................................. 27 Assessment by Molecule Type ............................................................................................. 28 Assessment by Stage and Molecule Type ............................................................................ 29 Assessment by Target .......................................................................................................... 30 Assessment by Stage and Target ......................................................................................... 31 Dormant and Discontinued Products ............................................................................... 32 Comparative Analysis ........................................................................................................... 32 Drug name: Company name ............................................................................................ 33 Product Description ............................................................................................................. 33 Research and Development ................................................................................................. 33 Product Development Activities .......................................................................................... 33 Reason for Dormancy / Discontinuation ............................................................................. 33 Companies involved in Therapeutics Development .......................................................... 34 Appendix ......................................................................................................................... 35 Report Methodology ............................................................................................................ 35 Report Price .................................................................................................................... 36 About CmaxInsight .......................................................................................................... 37 Contact Us ....................................................................................................................... 37 Disclaimer ....................................................................................................................... 37 6 ©CmaxInsight|This report is a licensed product and is not to be photocopied

7. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 List of Tables Table 1: Total Products for Hypertrophic Cardiomyopathy (HCM) ........................................... 9 Table 2: Late Stage Products (Filed & Phase III) ...................................................................... 11 Table 3: Mid Stage Products (Phase II) .................................................................................... 17 Table 4: Early Stage Products (Phase I) .................................................................................... 20 Table 5: Pre-clinical and Discovery Stage Products ................................................................. 23 Table 6: Assessment by Route of Administration .................................................................... 26 Table 7: Assessment by Stage and Route of Administration ................................................... 27 Table 8: Assessment by Molecule Type ................................................................................... 28 Table 9: Assessment by Stage and Molecule Type .................................................................. 29 Table 10: Assessment by Target .............................................................................................. 30 Table 11: Assessment by Stage and Target ............................................................................. 31 Table 12: Dormant Products .................................................................................................... 32 Table 13: Discontinued Products ............................................................................................. 32 Table 14: MyoKardia, Overview, 2019 ..................................................................................... 34 Table 15: Company 2, Overview, 2019 .................................................................................... 34 List of Figures Figure 1: Number of Products under Development for Hypertrophic Cardiomyopathy (HCM) ................................................................................................................................................ 9 Figure 2: Late Stage Products (Filed & Phase III) ................................................................. 11 Figure 3: Mid Stage Products (Phase II) ............................................................................... 17 Figure 4: Early Stage Products (Phase I) ............................................................................... 20 Figure 5: Assessment by Route of Administration ............................................................... 26 Figure 6: Assessment by Stage and Route of Administration .............................................. 27 Figure 7: Assessment by Molecule Type .............................................................................. 28 Figure 8: Assessment by Stage and Molecule Type ............................................................. 29 Figure 9: Assessment by Target ........................................................................................... 30 Figure 10: Assessment by Stage and Target ........................................................................ 31 Figure 11: Dormant and Discontinued Products.................................................................. 32 7 ©CmaxInsight|This report is a licensed product and is not to be photocopied

8. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Disease Overview Xxxxx Epidemiology Xxxxx Causes Xxxxx Symptoms Xxxxxx Pathophysiology Xxxx Diagnosis Xxxx Treatment Xxxxx 8 ©CmaxInsight|This report is a licensed product and is not to be photocopied

9. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Competitive Landscape Comparative Analysis Figure 1: Number of Products under Development for Hypertrophic Cardiomyopathy (HCM) 10 9 Filed Number of Products 8 Phase III Phase II 6 5 Phase I Pre-Clinical 4 3 3 Discovery 2 2 2 Dormant 2 1 Discontinued 0 Stage of Development Source: CmaxInsight Table 1: Total Products for Hypertrophic Cardiomyopathy (HCM) Stage of Development Filed Phase III Phase II Phase I Pre-Clinical Discovery Dormant Discontinued Number of Products Source: CmaxInsight 9 ©CmaxInsight|This report is a licensed product and is not to be photocopied

10. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Pipeline Therapeutics 1 Avadel Pharmaceuticals 1 Arena Pharmaceuticals 1 Filed Balance Therapeutics 1 Phase III Evotec AG Phase II 1 GlaxoSmithKline Phase I 1 Heptares Therapeutics Pre-clinical 1 2 1 Jazz Pharmaceuticals Discovery 1 1 Johnson & Johnson Dormant 1 NLS Pharma Discontinued Company Name 1 Ono Pharmaceutical 1 OptiNose 1 Pfizer 1 Reset Therapeutics 1 Roche 1 Sanofi 1 Seelos Therapeutics 1 Suven Life Sciences 1 Taisho Pharmaceutical 1 Takeda 1 Theranexus 0 1 2 3 4 5 Number of Products 10 ©CmaxInsight|This report is a licensed product and is not to be photocopied

11. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Late Stage Products (Filed & Phase III) Comparative Analysis Figure 2: Late Stage Products (Filed & Phase III) Total Active Products 9 10 8 Number of Products 6 Filed Products 2 4 Phase III Products 3 2 0 Stage of Development Source: CmaxInsight Table 2:Late Stage Products (Filed & Phase III) Drug name Company Phase MoA RoA Molecule type Source: CmaxInsight 11 ©CmaxInsight|This report is a licensed product and is not to be photocopied

12. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Mavacamten (MYK-461): MyoKardia Product Description Xxx Research and Development Clinical Studies Clinical Trials Description Intervention (Company) NCT No. Start date 1 ⁰ Phase Status Indication Completion III Recruiting Mavacamten (MyoKardia) Symptomatic obstructive HCM EXPLORER-HCM May 2018 Jun 2020 II/III Enrolling by invitation Mavacamten HCM NCT03723655 (Long-term safety extension Study of MAVERICK-HCM or EXPLORER-HCM) Oct 2018 Jun 2022 II Active, not recruiting Mavacamten Symptomatic obstructive HCM PIONEER-OLE (Extension Study of PIONEER) Apr 2018 Feb 2021 II Recruiting Mavacamten Symptomatic Non-Obstructive HCM and Preserved Left Ventricular Ejection Fraction MAVERICK-HCM Mar 2018 Jun 2019 II Completed Mavacamten Symptomatic HCM and Left Ventricular Outflow Tract Obstruction PIONEER-HCM Aug 2016 Nov 2017 I Completed Mavacamten Healthy Volunteers NCT02356289 Jan 2015 Jul 2015 I Completed Mavacamten HCM NCT02329184 Dec 2014 Mar 2016 I Completed Mavacamten Healthy Volunteers NCT02480296 Aug 2015 Jul 2016 Source: CmaxInsight 12 ©CmaxInsight|This report is a licensed product and is not to be photocopied

13. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Clinical Trial Description EXPLORER-HCM: Study Design & Details Phase III Start date May 2018 1 ⁰ CompletionJun 2020 Trial ID EXPLORER- HCM Sponsor MyoKardia Study Design Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: Multicenter, international, double-blind study Primary: Secondary: EXPLORER-HCM Results Efficacy Safety Interim Results Source Source: CmaxInsight NCT03723655: Study Design & Details Phase Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: Results Efficacy Safety Interim Results Source Source: CmaxInsight NCT03723655: Study Design & Details Phase Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: Results Efficacy 13 ©CmaxInsight|This report is a licensed product and is not to be photocopied

14. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Safety Interim Results Source Source: CmaxInsight PIONEER-OLE: Study Design & Details Phase Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: Results Efficacy Safety Interim Results Source Source: CmaxInsight MAVERICK-HCM: Study Design & Details Phase Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: Results Efficacy Safety Interim Results Source Source: CmaxInsight PIONEER-HCM: Study Design & Details Phase Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: 14 ©CmaxInsight|This report is a licensed product and is not to be photocopied

15. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Results Efficacy Safety Interim Results Source Source: CmaxInsight Product Development Activities Designation Deals Patent Technology Funding & financing Key Development Milestone 15 ©CmaxInsight|This report is a licensed product and is not to be photocopied

16. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Mavacamten General Description Drug Name Generic Name Alias Name Company Product Type Therapy Area Indications Highest Stage of Development for HCM Designation Licensed From Licensing Type Development Partner Institutional Partner Route of Administration Target Mechanism of Action Technology Molecule Type Drug Origin Chemical Name Chemical Formula Molecular Weight CAS Registration No. ATC Classification Source: CmaxInsight 16 ©CmaxInsight|This report is a licensed product and is not to be photocopied

17. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Mid Stage Products (Phase II) Comparative Analysis Figure 3: Mid Stage Products (Phase II) 10 Total Active Products 9 8 Number of Products 6 Phase II Products 2 4 2 0 Stage of Development Source: CmaxInsight Table 3:Mid Stage Products (Phase II) Drug name Company Phase MoA RoA Molecule type Source: CmaxInsight 17 ©CmaxInsight|This report is a licensed product and is not to be photocopied

18. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Drug name: Company name Product Description Xxxxx Research and Development Clinical Studies Clinical Trials Description Intervention (Company) NCT No. Start date 1 ⁰ Phase Status Indication Completion Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Source: CmaxInsight Clinical Trial Description Trial Name: Study Design & Details Phase II Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: Results Efficacy Safety Interim Results Source Source: CmaxInsight Product Development Activities Designation Deals Patent Technology Funding & financing Key Development Milestone Xxxx 18 ©CmaxInsight|This report is a licensed product and is not to be photocopied

19. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Drug Name General Description Drug Name Generic Name Alias Name Company Product Type Therapy Area Indications Highest Stage of Development for HCM Designation Licensed From Licensing Type Development Partner Institutional Partner Route of Administration Target Mechanism of Action Technology Molecule Type Drug Origin Chemical Name Chemical Formula Molecular Weight CAS Registration No. ATC Classification Source: CmaxInsight 19 ©CmaxInsight|This report is a licensed product and is not to be photocopied

20. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Early Stage Products (Phase I) Comparative Analysis Figure 4: Early Stage Products (Phase I) 10 Total Active Products 9 8 Number of Products 6 Phase I Products 2 4 2 0 Stage of Development Source: CmaxInsight Table 4:Early Stage Products (Phase I) Drug name Company Phase MoA RoA Molecule type Source: CmaxInsight 20 ©CmaxInsight|This report is a licensed product and is not to be photocopied

21. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Drug name: Company name Product Description Xxxxx Research and Development Clinical Studies Clinical Trials Description Intervention (Company) NCT No. Start date 1 ⁰ Phase Status Indication Completion Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Source: CmaxInsight Trial Name: Study Design & Details Phase II Start date Sponsor Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: 1 ⁰ Completion Trial ID Study Design Primary: Secondary: Results Efficacy Safety Interim Results Source Source: CmaxInsight Product Development Activities Designation Deals Patent Technology Funding & financing Key Development Milestone Xxxx Drug Name 21 ©CmaxInsight|This report is a licensed product and is not to be photocopied

22. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 General Description Drug Name Generic Name Alias Name Company Product Type Therapy Area Indications Highest Stage of Development for HCM Designation Licensed From Licensing Type Development Partner Institutional Partner Route of Administration Target Mechanism of Action Technology Molecule Type Drug Origin Chemical Name Chemical Formula Molecular Weight CAS Registration No. ATC Classification Source: CmaxInsight 22 ©CmaxInsight|This report is a licensed product and is not to be photocopied

23. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Pre-clinical and Discovery Products Comparative Analysis Figure 6: Pre-clinical and Discovery Stage Products Total Active Products 9 10 8 Number of Products 6 Pre-Clinical Products 2 4 Discovery Products 2 2 0 Stage of Development Source: CmaxInsight Table 5:Pre-clinical and Discovery Stage Products Drug name Company Phase MoA RoA Molecule type Source: CmaxInsight 23 ©CmaxInsight|This report is a licensed product and is not to be photocopied

24. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Drug name: Company name Product Description Xxxxxx Research and Development Pre-clinical Studies Xxxxxx Product Development Activities Designation Deals Patent Technology Funding & financing Key Development Milestone Xxxx 24 ©CmaxInsight|This report is a licensed product and is not to be photocopied

25. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Drug Name General Description Drug Name Generic Name Alias Name Company Product Type Therapy Area Indications Highest Stage of Development for HCM Designation Licensed From Licensing Type Development Partner Institutional Partner Route of Administration Target Mechanism of Action Technology Molecule Type Drug Origin Chemical Name Chemical Formula Molecular Weight CAS Registration No. ATC Classification Source: CmaxInsight 25 ©CmaxInsight|This report is a licensed product and is not to be photocopied

26. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Therapeutic Assessment Assessment by Route of Administration Figure 5:Assessment by Route of Administration 2 1 Oral Intravenous 1 Intranasal Unspecified 12 Source: CmaxInsight Table 6: Assessment by Route of Administration Route of Administration Oral Intravenous Intranasal Unspecified Number of Products 26 ©CmaxInsight|This report is a licensed product and is not to be photocopied

27. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Assessment by Stage and Route of Administration Figure 6:Assessment by Stage and Route of Administration 1 1 Discovery 1 1 1 Pre-clinical Oral 2 1 Phase I Intravenous 3 Phase II Intranasal 4 Unspecified Phase III 1 Filed 0 1 2 3 4 5 No. of Products Source: CmaxInsight Table 7: Assessment by Stage and Route of Administration Route of Administration Stage of Development Filed Phase III Phase II Phase I Pre-clinical Discovery Phase I Pre-clinical Pre- clinical Discovery Number of Products Oral Intravenous Intranasal Unspecified Source: CmaxInsight 27 ©CmaxInsight|This report is a licensed product and is not to be photocopied

28. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Assessment by Molecule Type Figure 7:Assessment by Molecule Type 1 Small Molecule 3 Biologics 2 Peptide Unspecified 2 Source: CmaxInsight Table 8: Assessment by Molecule Type Molecule Type Small Molecule Biologic Peptide Unspecified Number of Products Source: CmaxInsight 28 ©CmaxInsight|This report is a licensed product and is not to be photocopied

29. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Assessment by Stage and Molecule Type Figure 8:Assessment by Stage and Molecule Type 1 1 Discovery 1 1 1 Pre-clinical Small Molecule 2 1 Phase I Biologic 3 Phase II Peptide 4 Unspecified Phase III 1 Filed 0 1 2 3 4 5 No. of Products Source: CmaxInsight Table 9: Assessment by Stage and Molecule Type Molecule Type Stage of Development Filed Phase III Phase II Phase I Pre-clinical Discovery Phase I Pre-clinical Pre- clinical Discovery Number of Products Small Molecule Biologic Peptide Unspecified Source: CmaxInsight 29 ©CmaxInsight|This report is a licensed product and is not to be photocopied

30. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Assessment by Target Figure 9:Assessment by Target 1 Target 1 3 Target 2 2 Target 3 Target 4 2 Source: CmaxInsight Table 10: Assessment by Target Target Target 1 Target2 Target 3 Target 4 Number of Products Source: CmaxInsight 30 ©CmaxInsight|This report is a licensed product and is not to be photocopied

31. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Assessment by Stage and Target Figure 10:Assessment by Stage and Target 1 1 Discovery 1 1 1 Pre-clinical Target 1 2 1 Phase I Target 2 3 Phase II Target 3 4 Target 4 Phase III 1 Filed 0 1 2 3 4 5 No. of Products Source: CmaxInsight Table 11: Assessment by Stage and Target Target Stage of Development Filed Phase III Phase II Phase I Pre-clinical Discovery Phase I Pre-clinical Pre- clinical Discovery Number of Products Target 1 Target2 Target 3 Target 4 31 ©CmaxInsight|This report is a licensed product and is not to be photocopied

32. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Dormant and Discontinued Products Comparative Analysis Figure 11: Dormant and Discontinued Products Total Active Products 9 10 8 Number of Products 6 Dormant Products 2 4 Discontinued Products 2 2 0 Stage of Development Source: CmaxInsight Table 12: Dormant Products Drug Name Company Stage of Dormancy Source: CmaxInsight Table 13: Discontinued Products Drug Name Company Stage of Discontinuation Source: CmaxInsight 32 ©CmaxInsight|This report is a licensed product and is not to be photocopied

33. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Drug name: Company name Product Description Xxxxx Research and Development Clinical Studies Clinical Trials Description Intervention (Company) NCT No. Start date 1 ⁰ Phase Status Indication Completion Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Xxxxx Source: CmaxInsight Product Development Activities Xxxx Reason for Dormancy / Discontinuation Xxxx 33 ©CmaxInsight|This report is a licensed product and is not to be photocopied

34. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Companies involved in Therapeutics Development Table 14: MyoKardia, Overview, 2019 Company Description Entity Industry Parent Company Headquarter Country Market Capitalization Net Revenue Net Income Website Source: CmaxInsight MyoKardia Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Table 15: Company 2, Overview, 2019 Company Entity Industry Parent Company Headquarter Country Market Capitalization Net Revenue Net Income Website Source: CmaxInsight Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx Xxxx 34 ©CmaxInsight|This report is a licensed product and is not to be photocopied

35. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Appendix Report Methodology CmaxInsight is a premier source for pharmaceutical, life science, medical devices and biotechnology sector analysis, delivering exclusive, trusted commercial insight into industry performance through its proprietary platform. CmaxInsight’s reports are continuously updated and revised. The following research methodology is followed for all databases and reports. Coverage The objective of updating CmaxInsight coverage is to ensure that it represents the most up to date vision of the industry possible. The CmaxInsight is a fully-integrated solution for comprehensive intelligence on various pharmaceutical products, both in-market and in pipeline, across the globe. Secondary Research The secondary research on internal and external sources is being carried out to source qualitative and quantitative information relating to each market. The secondary research sources that are typically referred to include, but are not limited to: •Company websites, annual reports, financial reports, broker reports, investor presentations and SEC Filings. •Industry trade journals and other literature. •Internal and external proprietary databases. •National government documents, statistical databases and market reports. •News articles, press releases and web-casts specific to the companies operating in the market. Primary Research CmaxInsight conducts primary research by interacting with the healthcare professionals, doctors, industry’s experts and industry participants and commentators in order to validate its data and analysis. The discussion provides first-hand information on the market size, market trends, growth trends, competitive landscape, future outlook, for example. •It helps in validating and strengthening the secondary research findings. •It further develops the Analysis Team’s expertise and market understanding. 35 ©CmaxInsight|This report is a licensed product and is not to be photocopied

36. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 Report Price Report Name Single User License USD 800 Corporate License USD 2000 Hypertrophic Cardiomyopathy: Pipeline Intelligence- 2019 36 ©CmaxInsight|This report is a licensed product and is not to be photocopied

37. Hypertrophic Cardiomyopathy (HCM): Pipeline Intelligence, 2019 About CmaxInsight CmaxInsight is a global market research and consulting firm that specialized in providing high- value customized business research, intelligence reports, and databases across pharmaceutical, life science, and healthcare domain. Our aim is to maximize insight with intelligence which assists the clients in decision making and to address new challenges in a fast-paced healthcare environment. We empower healthcare organization at every stage to make the process rapid, better and accurate. We help our clients in bridging the gaps in their business through screening, prioritization, in-depth commercial assessment, independent evaluation of opportunities which facilitating decision-making; identify potential market opportunities, competitor assessments, and business environment assessment. The pharmaceuticals market is in its growth stage and we are uniquely positioned to successfully endeavour the dimensions of the fast growing market by providing cutting-edge market and pipeline information, to our clients. Contact Us If you have any queries about this report or would like further information, please contact us at the below given email address. Email: sales@cmaxinsight.com Disclaimer All Rights Reserved. No part of this publication may be reproduced, stored in a retrieval system or transmitted in any form by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior permission of the publisher, CmaxInsight. The facts of this report were believed to be correct at the time of publication but cannot be guaranteed. Please note that the findings, conclusions and recommendations that CmaxInsight delivers will be based on information gathered in good faith from both primary and secondary sources, whose accuracy we were not always in a position to guarantee. As such CmaxInsight can accept no liability whatever for actions taken based on any information that may subsequently prove to be incorrect. 37 ©CmaxInsight|This report is a licensed product and is not to be photocopied