關注市場分析:裘馨氏肌肉萎縮症 (DMD)

本報告提供裘馨氏肌肉萎縮症 (DMD)的治療藥市場相關分析，疾病概要和患者數的變化、預測，目前的主要的治療方法，法規環境和近幾年主要的事件，現在開發中臨床實驗的進展與上市計劃，主要的治療藥的市場趨勢預測 (今後10年份)，資本交易的動向等調查。

分析的要點

疾病的背景情況

治療方法

• 皮質類固醇
• 基因治療
• 維他命D供給

流行病學

已上市醫藥品

• 各國的認證情形

開發平台上的醫藥品

將產生的主要事件

法規相關的主要事件 (共6件)

授權合約、資產收購交易 (共4件)

母體專利

商機

臨床實驗形勢

• 贊助商數:各狀態
• 贊助商數:各相位
• 近幾年趨勢

相關分析

• 處方藥的資訊

附錄

This Market Spotlight report covers the Duchenne Muscular Dystrophy market, comprising key marketed and pipeline drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, probability of success, recent events and analyst opinion, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.

Key Takeaways:

Datamonitor Healthcare estimates that in 2017, there were 182,100 prevalent cases of Duchenne muscular dystrophy (DMD) in males worldwide, and forecasts that number to increase to 199,100 prevalent cases by 2026.

Approved drugs in the DMD space target glucocorticoid receptor, dystrophin gene, and RNA translation. These drugs are administered via the oral and intravenous routes.

The largest proportion of industry-sponsored drugs in active clinical development for DMD are in Phase II, with only drugs in the NDA/BLA phase.

Therapies in development for DMD focus on a wide variety of targets. The majority of pipeline drugs are administered via the intravenous or oral routes, with the remainder being intramuscular, intraarterial, and subcutaneous formulations.

High-impact upcoming events for drugs in the DMD space comprise topline Phase I/II, Phase II, Phase Iib, and Phase III trial results, an estimated PDUFA date for an NDA, and an expected option exercise decision.

The overall likelihood of approval of a Phase I DMD asset is 21.8%, and the average probability a drug advances from Phase III is 62.5%. Drugs, on average, take 8.6 years from Phase I to approval, compared to 8.9 years in the overall metabolic space.

There have been 21 licensing and asset acquisition deals involving DMD drugs during 2015-20, seven of which occurred in 2017. The largest deal during the period was the $3,520m licensing agreement between Vertex and CRISPR Therapeutics signed in 2017, as part of which the two companies will discover and develop gene editing therapies for the treatment of DMD and myotonic dystrophy type 1 (DM1).

The distribution of clinical trials across Phase I-IV indicates that the majority of trials for DMD have been in the early and midphases of development, with 71% of trials in Phase I-II, and only 29% in Phase III-IV.

The US has a substantial lead in the number of DMD clinical trials globally. The UK leads the major European markets, while Israel has the top spot in Asia.

Sarepta Therapeutics has the highest number of ongoing clinical trials for DMD, with 10 trials.

Sarepta Therapeutics leads industry sponsors with the highest number of clinical trials for DMD, followed by PTC Therapeutics.

TABLE OF CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

• Corticosteroids
• Genetic therapies
• Vitamin D supplement

EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

• SGT-001 for DMD (December 18, 2019)
• SGT-001 for DMD (November 12, 2019)
• Vyondys 53 for DMD (August 19, 2019)
• CAP-1002 for DMD (July 15, 2019)
• CPF-06939926 for DMD (June 28, 2019)
• SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
• AT702 for DMD (April 8, 2019)
• Casimersen for DMD (March 28, 2019)
• Catena for DMD (February 25, 2019)
• SGT-001 for DMD (February 7, 2019)
• Emflaza for DMD (January 7, 2019)
• CAP-1002 for DMD (December 21, 2018)
• PF-06252616 for DMD (August 30, 2018)
• Multiple Drugs for DMD (August 30, 2018)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

• For Sarepta, Another Exon-Skipping Drug
• Surprise As FDA Approves Sarepta's Duchenne Drug Vyondys 53
• Adverse Event Concerns With Solid Biosciences' Candidate DMD Gene Therapy
• Sarepta's Vyondys 53 Whiff Looks Costly As Nippon Shinyaku Submits Viltolarsen
• Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen
• PTC To Fight EMA Translarna No

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

• Sarepta Secures $1.15bn From Roche In Ex-US DMD Gene Therapy Deal
• Astellas To Pay $3bn For Gene Therapy Company Audentes
• Vertex Gene-Editing Play Includes Exonics Buy, Expanded Tie-Up With CRISPR
• Audentes Gains License To NCH's DMD, DM1 Candidates

PARENT PATENTS

REVENUE OPPORTUNITY

CLINICAL TRIAL LANDSCAPE

• Sponsors by status
• Sponsors by phase
• Recent events

BIBLIOGRAPHY

• Prescription information

APPENDIX

LIST OF FIGURES

• Figure 1: Trends in prevalent cases of DMD, 2017-26
• Figure 2: Overview of pipeline drugs for DMD in the US
• Figure 3: Pipeline drugs for DMD, by company
• Figure 4: Pipeline drugs for DMD, by drug type
• Figure 5: Pipeline drugs for DMD, by classification
• Figure 6: CAP-1002 for DMD (July 15, 2019): Phase II - HOPE-2
• Figure 7: Casimersen for DMD (March 28, 2019): Phase III - ESSENCE (Study 4045-301; Exon 45 & 53)
• Figure 8: Catena for DMD (February 25, 2019): Phase III - SYROS
• Figure 9: PF-06252616 for DMD (August 30, 2018): Phase II - PK and PD Study
• Figure 10: Key upcoming events in DMD
• Figure 11: Probability of success in the DMD pipeline
• Figure 12: Licensing and asset acquisition deals in DMD, 2015-20
• Figure 13: Parent patents in DMD
• Figure 14: Clinical trials in DMD
• Figure 15: Top 10 drugs for clinical trials in DMD
• Figure 16: Top 10 companies for clinical trials in DMD
• Figure 17: Trial locations in DMD
• Figure 18: DMD trials status
• Figure 19: DMD trials sponsors, by phase

LIST OF TABLES

• Table 1: Prevalent cases of DMD, 2017-26
• Table 2: Marketed drugs for DMD
• Table 3: Pipeline drugs for DMD in the US
• Table 4: SGT-001 for DMD (December 18, 2019)
• Table 5: SGT-001 for DMD (November 12, 2019)
• Table 6: Vyondys 53 for DMD (August 19, 2019)
• Table 7: CAP-1002 for DMD (July 15, 2019)
• Table 8: PF-06939926 for DMD (June 28, 2019)
• Table 9: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
• Table 10: AT702 for DMD (April 8, 2019)
• Table 11: Casimersen for DMD (March 28, 2019)
• Table 12: Catena for DMD (February 25, 2019)
• Table 13: SGT-001 for DMD (February 7, 2019)
• Table 14: Emflaza for DMD (January 7, 2019)
• Table 15: CAP-1002 for DMD (December 21, 2018)
• Table 16: PF-06252616 for DMD (August 30, 2018)
• Table 17: Multiple Drugs for DMD (August 30, 2018)
• Table 18: Historical global sales, by drug ($m), 2014-18
• Table 19: Forecasted global sales, by drug ($m), 2020-24