Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence-2019

1. Facioscapulohumeral Muscular Dystrophy (FSHD): Intelligence, 2019 Pipeline Published on: April 2019

2. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Introduction CmaxInsight’s, “Facioscapulohumeral Muscular Dystrophy (FSHD): 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 Facioscapulohumeral Muscular Dystrophy. 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 Facioscapulohumeral Muscular Dystrophy 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 Facioscapulohumeral Muscular Dystrophy R&D Secondary resources such as Company Websites, Annual Reports, Financial Reports, Analyst Reports, Investor Presentations, Press Releases, web-casts Industry Trade Journals, Pubmed, NCBI, Oxford and other literature and in-house analysis by CmaxInsight’s team of industry experts are referred for preparing the report. 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. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Insights Pipeline Drugs Therapeutic Assessment Disease Overview Insights Leading Company Analysis Clinical Trial Landscape Mergers, Acquisitions & Collaboration Deals 3 ©CmaxInsight|This report is a licensed product and is not to be photocopied

4. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Executive Summary Snapshot Facioscapulohumeral Muscular Dystrophy Snapshot Pipeline at a Glance 1 2 2 2 Phase I Phase III Phase II Pre-Clinical & Discovery Leading Companies & their Most Prominent Targets •Target •Target Company 1 Company 2 Company 3 Company 4 •Target •Target Market Drivers Market Barriers 4 ©CmaxInsight|This report is a licensed product and is not to be photocopied

5. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Table of Contents Introduction ................................................................................................................................ 1 Insights ........................................................................................................................................ 3 Executive Summary Snapshot ....................................................................................................... 4 Disease Overview ........................................................................................................................ 9 Subdivisions of Facioscapulohumeral Muscular Dystrophy ............................................................... 9 Causes ................................................................................................................................................. 9 Symptoms ........................................................................................................................................... 9 Pathophysiology ................................................................................................................................. 9 Diagnosis ............................................................................................................................................. 9 Treatment ......................................................................................................................................... 10 Epidemiology .................................................................................................................................... 10 FSHD Research and Development ................................................................................................ 11 Competitive Landscape ............................................................................................................... 12 Comparative Analysis ....................................................................................................................... 12 Pipeline Therapeutics ................................................................................................................. 13 Mid Stage Products (Phase II) ...................................................................................................... 14 Comparative Analysis ....................................................................................................................... 14 ACE-083: Acceleron Pharma ........................................................................................................ 15 Description ........................................................................................................................................ 15 Product Profile .................................................................................................................................. 15 Research and Development ............................................................................................................. 16 Clinical Trial Landscape ..................................................................................................................... 16 Product Development Activities ....................................................................................................... 17 Key Development Milestone ............................................................................................................ 18 Early Stage Products (Phase I)...................................................................................................... 19 Comparative Analysis ....................................................................................................................... 19 Drug name: Company name ........................................................................................................ 20 Product Description .......................................................................................................................... 20 Research and Development ............................................................................................................. 20 Product Development Activities ....................................................................................................... 20 Key Development Milestone ............................................................................................................ 20 Pre-clinical and Discovery Products ............................................................................................. 22 5 ©CmaxInsight|This report is a licensed product and is not to be photocopied

6. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Comparative Analysis ....................................................................................................................... 22 Drug name: Company name ........................................................................................................ 23 Product Description .......................................................................................................................... 23 Research and Development ............................................................................................................. 23 Product Development Activities ....................................................................................................... 23 Key Development Milestone ............................................................................................................ 23 Featured Clinical Evidence for FSHD ............................................................................................. 25 Universities Working on FSHD ..................................................................................................... 26 Patient Support and Advocacy Resources .................................................................................... 27 Friends of FSH Research .............................................................................................................. 27 FSH Society ................................................................................................................................. 27 Muscular Dystrophy Association ................................................................................................. 27 Muscular Dystrophy Canada ........................................................................................................ 27 Muscular Dystrophy UK ............................................................................................................... 27 National Organization for Rare Disorders (NORD) ........................................................................ 27 Therapeutic Assessment ............................................................................................................. 28 Assessment by Route of Administration .......................................................................................... 28 Assessment by Stage and Route of Administration ......................................................................... 29 Assessment by Molecule Type ......................................................................................................... 30 Assessment by Stage and Molecule Type ......................................................................................... 31 Assessment by Target ....................................................................................................................... 32 Assessment by Stage and Target ...................................................................................................... 33 Dormant and Discontinued Products ........................................................................................... 34 Comparative Analysis ....................................................................................................................... 34 Drug name: Company name ........................................................................................................ 35 Product Description .......................................................................................................................... 35 Research and Development ............................................................................................................. 35 Product Development Activities ....................................................................................................... 35 Reason for Dormancy / Discontinuation .......................................................................................... 35 Leading Companies Analysis ........................................................................................................ 36 Company Description ....................................................................................................................... 36 Company Profile ............................................................................................................................... 36 6 ©CmaxInsight|This report is a licensed product and is not to be photocopied

7. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Company, Total Revenue .................................................................................................................. 37 Revenue by Business Segments ........................................................................................................ 38 Geography-Wise Revenue ................................................................................................................ 38 Report Price ................................................................................................................................ 39 About CmaxInsight ...................................................................................................................... 39 Contact Us .................................................................................................................................. 39 Disclaimer ................................................................................................................................... 39 7 ©CmaxInsight|This report is a licensed product and is not to be photocopied

8. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 List of Tables Table 1: Total Products for Facioscapulohumeral Muscular Dystrophy .................................. 12 Table 3: Mid Stage Products (Phase II) .................................................................................... 14 Table 4: Early Stage Products (Phase I) .................................................................................... 19 Table 5: Pre-clinical and Discovery Stage Products ................................................................. 22 Table 6: List of featured Clinical Evidence for Facioscapulohumeral Muscular Dystrophy (FSHD) ...................................................................................................................................... 25 Table 7: List of Universities working on Facioscapulohumeral Muscular Dystrophy (FSHD) .. 26 Table 8: List of Universities working on Facioscapulohumeral Muscular Dystrophy .............. 27 Table 9: Assessment by Route of Administration .................................................................... 28 Table 10: Assessment by Stage and Route of Administration ................................................. 29 Table 11: Assessment by Molecule Type ................................................................................. 30 Table 12: Assessment by Stage and Molecule Type ................................................................ 31 Table 13: Assessment by Target .............................................................................................. 32 Table 14: Assessment by Stage and Target ............................................................................. 33 Table 15: Dormant Products .................................................................................................... 34 Table 16: Discontinued Products ............................................................................................. 34 Table 13: Acceleron Pharma Profile, 2018 .............................................................................. 36 List of Figures Figure 1: Number of Products under Development for Facioscapulohumeral Muscular Dystrophy ............................................................................................................................. 12 Figure 3: Mid Stage Products (Phase II) ............................................................................... 14 Figure 4: Early Stage Products (Phase I) ............................................................................... 19 Figure 5: Assessment by Route of Administration ............................................................... 28 Figure 6: Assessment by Stage and Route of Administration .............................................. 29 Figure 7: Assessment by Molecule Type .............................................................................. 30 Figure 8: Assessment by Stage and Molecule Type ............................................................. 31 Figure 9: Assessment by Target ........................................................................................... 32 Figure 10: Assessment by Stage and Target ........................................................................ 33 Figure 11: Dormant and Discontinued Products.................................................................. 34 Figure 11: Company, Total Revenue .................................................................................... 37 Figure 11: Company, Total Revenue .................................................................................... 38 Figure 11: Company, Total Revenue .................................................................................... 38 8 ©CmaxInsight|This report is a licensed product and is not to be photocopied

9. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Disease Overview Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder characterized by muscle weakness and wasting (atrophy). The disorder gets its name from muscles that are affected in the face (facio), around the shoulder blades (scapulo), and in the upper arms (humeral). Subdivisions of Facioscapulohumeral Muscular Dystrophy Facioscapulohumeral muscular dystrophy 1 Facioscapulohumeral muscular dystrophy 2 Causes FSHD1 is caused by abnormal expression of the DUX4 gene, which is located in the D4Z4 region of chromosome 4 Clinical variability From generation to generation in a singleFamily Between different families with the same genetic profile Frequent side to side asymmetry Symptoms Symptoms usually appear before age 20, but can begin in infancy or later in adulthood Severity of the condition varies widely and some people remain asymptomatic Many of these patients have trouble walking and running, and develop upper arm muscle weakness, ultimately limiting their ability to perform daily tasks, such as eating or getting dressed Descending pattern of muscle involvement First affecting the face, shoulder, and arms Distal lower extremity (e.g., foot drop) Thigh and pelvic girdle Characterized by specific (focal) muscle involvement Significant impairment ~20% will require a wheelchair ~20% on work disability due to the disease Pathophysiology Xxxx Diagnosis Diagnosis is based on characteristic features and genetic testing 9 ©CmaxInsight|This report is a licensed product and is not to be photocopied

10. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Treatment There are no FDA approved therapies for FSHD Bracing devices Lower leg braces (AFO’s), back supports, walking devices, etc Multi-disciplinary approach – Physical / Occupational Therapy (PT/OT) Addressing target muscle weakness Pain Respiratory function Hearing / vision Surgical (scapular fixation) Unmet Needs Slowly progressing muscle weakness diseases No approved pharmacology Therapies Foot drop (ankle weakness) affects a majority of patients leading to multiple challenges Upper arm weakness (biceps) is reported in a majority of FSHD patients Epidemiology FSHD appears to affect males and females in relatively equal numbers FSHD has an estimated prevalence of 1 in 20,000 people in the United states The estimated prevalence is between four and ten per 100,000 people About 95 percent of all cases are FSHD1; the remaining 5 percent are FSHD2 10 ©CmaxInsight|This report is a licensed product and is not to be photocopied

11. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 FSHD Research and Development Immunomodulation targeting T-cells RNA interference Antisense oligonucleotides (ASO) CRISPR Stem cells 11 ©CmaxInsight|This report is a licensed product and is not to be photocopied

12. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Competitive Landscape Comparative Analysis Figure 1: Number of Products under Development for Facioscapulohumeral Muscular Dystrophy 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 Facioscapulohumeral Muscular Dystrophy Stage of Development Filed Phase III Phase II Phase I Pre-Clinical Discovery Dormant Discontinued Number of Products Source: CmaxInsight 12 ©CmaxInsight|This report is a licensed product and is not to be photocopied

13. Facioscapulohumeral Muscular Dystrophy: 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 13 ©CmaxInsight|This report is a licensed product and is not to be photocopied

14. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Mid Stage Products (Phase II) Comparative Analysis Figure 2: 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 2:Mid Stage Products (Phase II) Drug name ACE-083 Acceleron Pharma Company Phase II MoA RoA Molecule type Source: CmaxInsight 14 ©CmaxInsight|This report is a licensed product and is not to be photocopied

15. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 ACE-083: Acceleron Pharma Description ACE 083, a recombinant fusion protein consisting of a modified form of human follistatin linked to the human immunoglobulin G2 Fc domain, is being developed by Acceleron Pharma. It is in Phase II stage of development for the treatment of facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease. It is a locally active agent that may be useful for diseases of focal muscle loss such as muscular dystrophies, such as facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease (CMT). Product Profile Product Profile of ACE-083 Drug Name ACE-083 Generic Name NA Alias Name ACE083, ACE 083 Company Acceleron Pharma Product Type Mono Therapy Area Neuromuscular Facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease (Phase II) Indications Highest Stage of Development Phase II Orphan Drug Designation (FDA), Fast Track Designation (FDA) Intramuscular (IM) TGF-beta superfamily protein Designation Route of Administration Target Mechanism of Action TGF-beta superfamily protein inhibitors Technology NA Molecule Type Recombinant fusion protein Source: CmaxInsight 15 ©CmaxInsight|This report is a licensed product and is not to be photocopied

16. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Research and Development Clinical Studies Clinical Trials Description Intervention (Company) NCT No. Start date 1 ⁰ Phase Status Indication Completion II Active, not recruiting ACE-083 (Acceleron Pharma) FSHD NCT02927080 Nov 2016 Mar 2020 I Completed ACE-083 (Acceleron Pharma) Musculoskeletal Diseases NCT02257489 Sep 2014 Mar 2016 Source: CmaxInsight Clinical Trial Landscape NCT02927080: Study Design & Details Phase II Start date Nov 2016 Sponsor Acceleron Pharma 1⁰ Completion Mar 2020 Study is divided in two parts: Part 1: Open-label, dose-escalation Part 2: Randomized, double-blind, and placebo controlled Study Adult (18 Years and older) with FSHD N=58 NA Part 1: ACE-083 150 mg OR 200 mg OR 200 mg IM for once every 3 weeks for up to 5 doses Part 2: ACE-083 up to 250 mg IM vs. PBO Placebo Primary:Safety and tolerability from Day 1 to Day 141 Secondary: Estimation of systemic exposure to ACE-083 following local intramuscular administration from Day 1 to Day 141 Primary: Target muscle volume increase (via MRI) Key Secondary Endpoints: Functional walk and upper limb tests along with patient reported outcomes Part 1: Identify dose to be used in Part 2 Part 2: Increases in muscle volume, strength and function Trial ID NCT02927080 Study Design Population Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: Analysis for Part-1 Endpoints Analysis for Part-2 Aim Results Part-1 of Phase II Mean total muscle volume increases (14% to 20%) Reduction in absolute fat fraction (overall muscle quality improvement) No serious adverse events, a majority of adverse events were injection-site related and grades 1-2 16 ©CmaxInsight|This report is a licensed product and is not to be photocopied

17. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 One patient with related grade 3 non-serious adverse event of “lower leg intramuscular swelling” Acceleron Pharma Corporate Presentation 2019, Acceleron Pharma Corporate Presentation 2018 Source Source: CmaxInsight NCT02257489: Study Design & Details Phase I Start date Sep 2014 Sponsor Acceleron Pharma Population 1 ⁰ CompletionMar 2016 Randomized, Single Group Assignment, Double-Blind, Placebo-Controlled, Dose-Ranging Study Healthy Postmenopausal Women N=58 NA ACE-083 50 mg OR 100 mg OR 150 mg OR 200 mg IM Placebo Primary:Safety and tolerability from Day 1 to Day 92 Secondary: Pharmacokinetics and pharmacodynamics from Day 1 to Day 92 Trial ID NCT02257489 Study Design Prior Rx Study Drug/Regimen Comparator/Regimen Endpoints: Results Safety Fifty-eight postmenopausal women were enrolled, 42 ACE-083 and 16 Placebo. No serious adverse events (AE), dose-limiting toxicities, or discontinuations resulting from AEs occurred. ACE-083 treatment generated dose-dependent mean total muscle volume increases of up to 14.5% in the rectus femoris (RF) and 8.9% in the tibialis anterior (TA) three weeks after the last dose NCBI Source Source: CmaxInsight Product Development Activities Conference Presentation: 27 Feb, 2019: Preliminary results from Part 1 of the Phase II trial evaluating ACE-083 in patients with FSHD, were presented at the 2018 World Muscle Society (WMS) Annual Meeting in October 2018 27 September, 2018: Acceleron announced presentations on ACE-083 from Part 1 of ongoing Phase II trial in patients with facioscapulohumeral muscular dystrophy (FSHD) at the 23rd International Annual Congress of the World Muscle Society 26 April, 2018: Acceleron announced preliminary results from the ongoing Phase II trial of ACE-083 in FSHD), presented at the American Academy of Neurology (AAN) 70th Annual Meeting in Los Angeles, California on April 26, 2018 17 ©CmaxInsight|This report is a licensed product and is not to be photocopied

18. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Designation: 12 July, 2018: Acceleron Pharma received FDA Orphan Drug Designation for ACE-083 in Facioscapulohumeral Muscular Dystrophy 01 May, 2018: Acceleron received FDA Fast Track Designation for ACE-083 in Facioscapulohumeral Muscular Dystrophy (FSHD) Results Announcement: 08 January, 2018: Acceleron Pharma announced preliminary results from Part 1 of the ACE- 083 Phase II trial in patients with FSHD Key Development Milestone Mar 2019: Part 2 of the Phase II FSHD trial has completed patient enrollment, with preliminary topline results expected in the second half of 2019 18 ©CmaxInsight|This report is a licensed product and is not to be photocopied

19. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Early Stage Products (Phase I) Comparative Analysis Figure 3: 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 3:Early Stage Products (Phase I) Drug name Company Phase MoA RoA Molecule type Source: CmaxInsight 19 ©CmaxInsight|This report is a licensed product and is not to be photocopied

20. Facioscapulohumeral Muscular Dystrophy: 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 Source: CmaxInsight Trial Name: 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 Product Development Activities Designation Deals Patent Technology Funding & financing Key Development Milestone Xxxx 20 ©CmaxInsight|This report is a licensed product and is not to be photocopied

21. Facioscapulohumeral Muscular Dystrophy: 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 21 ©CmaxInsight|This report is a licensed product and is not to be photocopied

22. Facioscapulohumeral Muscular Dystrophy: 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 4:Pre-clinical and Discovery Stage Products Drug name Company Phase MoA RoA Molecule type Source: CmaxInsight 22 ©CmaxInsight|This report is a licensed product and is not to be photocopied

23. Facioscapulohumeral Muscular Dystrophy: 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 23 ©CmaxInsight|This report is a licensed product and is not to be photocopied

24. Facioscapulohumeral Muscular Dystrophy: 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 24 ©CmaxInsight|This report is a licensed product and is not to be photocopied

25. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Featured Clinical Evidence for FSHD Product-related evidence Pipeline candidates Non-product related Evidence Table 5: List of featured Clinical Evidence for Facioscapulohumeral Muscular Dystrophy (FSHD) Drug name / Issue Title Source: CmaxInsight Source: CmaxInsight Journal Date Note: This section provides ‘FSHD-Feature Clinical Evidence’ for last 5 years Inclusion Criteria Rules • • • • • • • • • • Anything about pipeline drugs Pre-clinical studies of therapies Biomarkers Pathogenesis Epidemiology Risk factors Patient segmentation Disease progression Diagnosis Treatment 25 ©CmaxInsight|This report is a licensed product and is not to be photocopied

26. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Universities Working on FSHD Table 6: List of Universities working on Facioscapulohumeral Muscular Dystrophy (FSHD) Drug name / Issue Source: CmaxInsight Research Focus Source Date Note: Only includes data for last 5 years* 26 ©CmaxInsight|This report is a licensed product and is not to be photocopied

27. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Patient Support and Advocacy Resources Table 7: List of Universities working on Facioscapulohumeral Muscular Dystrophy Association Name Friends of FSH Research FSH Society Muscular Dystrophy Association Muscular Dystrophy Canada Muscular Dystrophy UK National Organization for Rare Disorders (NORD) Source: CmaxInsight Research Focus Country Source 27 ©CmaxInsight|This report is a licensed product and is not to be photocopied

28. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Therapeutic Assessment Assessment by Route of Administration Figure 4:Assessment by Route of Administration 2 1 Oral Intravenous 1 Intranasal Unspecified 12 Source: CmaxInsight Table 8: Assessment by Route of Administration Route of Administration Oral Intravenous Intranasal Unspecified Number of Products 28 ©CmaxInsight|This report is a licensed product and is not to be photocopied

29. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Assessment by Stage and Route of Administration Figure 5: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 9: 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 29 ©CmaxInsight|This report is a licensed product and is not to be photocopied

30. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Assessment by Molecule Type Figure 6:Assessment by Molecule Type 1 Small Molecule 3 Biologics 2 Peptide Unspecified 2 Source: CmaxInsight Table 10: Assessment by Molecule Type Molecule Type Small Molecule Biologic Peptide Unspecified Number of Products Source: CmaxInsight 30 ©CmaxInsight|This report is a licensed product and is not to be photocopied

31. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Assessment by Stage and Molecule Type Figure 7: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 11: 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 31 ©CmaxInsight|This report is a licensed product and is not to be photocopied

32. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Assessment by Target Figure 8:Assessment by Target 1 Target 1 3 Target 2 2 Target 3 Target 4 2 Source: CmaxInsight Table 12: Assessment by Target Target Target 1 Target2 Target 3 Target 4 Number of Products Source: CmaxInsight 32 ©CmaxInsight|This report is a licensed product and is not to be photocopied

33. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Assessment by Stage and Target Figure 9: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 13: 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 33 ©CmaxInsight|This report is a licensed product and is not to be photocopied

34. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Dormant and Discontinued Products Comparative Analysis Figure 10: 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 14: Dormant Products Drug Name Company Stage of Dormancy Source: CmaxInsight Table 15: Discontinued Products Drug Name Company Stage of Discontinuation Source: CmaxInsight 34 ©CmaxInsight|This report is a licensed product and is not to be photocopied

35. Facioscapulohumeral Muscular Dystrophy: 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 35 ©CmaxInsight|This report is a licensed product and is not to be photocopied

36. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Leading Companies Analysis Acceleron Pharma: Company Description Acceleron Pharma is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair. Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its Phase II pulmonary program with sotatercept in pulmonary arterial hypertension. Company Profile Table 16: Acceleron Pharma Profile, 2018 Company Name Entity Industry Headquarter Year Founded Business Segments Acceleron Pharma Inc. Public Biopharmaceutical Cambridge, Massachusetts, United States 2003 Hematologic, neuromuscular, and pulmonary diseases $14 Million Net Revenue (Year ended 31 Dec, 2018) Net Income (Year ended 31 Dec, 2018) CEO Website $5.5 Million Habib Dable http://acceleronpharma.com Source: CmaxInsight 36 ©CmaxInsight|This report is a licensed product and is not to be photocopied

37. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Company, Total Revenue XXXX Figure 11: Company, Total Revenue Total Revenue (2014-2017) 120 25.00% 100 20.00% Revenue In USD Million 80 15.00% 60 10.00% 40 5.00% 20 0 0.00% 2014 2015 2016 2017 Total Revenue AGR % Source: CmaxInsight 37 ©CmaxInsight|This report is a licensed product and is not to be photocopied

38. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Revenue by Business Segments XXXX Figure 12: Company, Total Revenue SEGMENT WISE REVENUE (2017) Source: CmaxInsight Geography-Wise Revenue XXXX Figure 13: Company, Total Revenue REGION WISE REVENUE (2017) Other Japan Oceania Middle East Southeast Asia North America Source: CmaxInsight 38 ©CmaxInsight|This report is a licensed product and is not to be photocopied

39. Facioscapulohumeral Muscular Dystrophy: Pipeline Intelligence, 2019 Report Price Report Name Single User License USD 800 Corporate License USD 2000 Facioscapulohumeral Muscular Dystrophy: 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. 39 ©CmaxInsight|This report is a licensed product and is not to be photocopied