The study analyzed that the Neurofibromatosis therapeutics pipeline comprises of approximately 25 therapeutic candidates in different stages of development.
Majority of the pipeline drug candidates are being developed to be administered by oral route. Celldex Therapeutics Inc. and Merck Co. Inc. are in the process of developing a monoclonal antibody drug candidate, CDX0158, and Pembrolizumab respectively for the treatment of NF. Lixte Biotechnology Holdings Inc. is in the process of developing two drug candidates, in the Pre-Clinical stage of development for the treatment of neurofibromatosis (NF). In September 2016, the USFDA granted Orphan Drug Designation to BXCL101, a drug candidate of BioXcel Therapeutics Inc. for the treatment of NF type 2.
Unmet medical needs driving the neurofibromatosis therapeutics pipeline
Since there is no treatment available for NF, the growing medical needs of the people suffering from the disease are driving its therapeutics pipeline growth. High rate of recurrence/progression of the disease and poor long-term benefit of surgery can be viewed as a limitation of this treatment option.
Positive clinical trial results propelling the growth of the pipeline therapeutics of the Neurofibromatosis
The drugs being developed by the different pharmaceutical companies demonstrated positive clinical trial results which in-turn increasing their further development. For instance, In June 2014, Array BioPharma Inc. announced positive results of Phase I/II, combination of binimetinib and LEE011. Selumetinib being developed by the AstraZeneca plc. And it met primary endpoint as confirmed partial responses (tumor volume decreases from baseline of =20%) in 17 of the 24 children (71%) in Phase I study.
Some of the key players developing drugs for the treatment of neurofibromatosis include Novartis AG, AstraZeneca Plc, Pfizer Inc., GlaxoSmithKline plc, Merck & Co. Inc, BioXcel Therapeutics, Inc.
Key Topics Covered:
1. Research Background 1.1 Research Objectives 1.2 Definition 1.3 Research Scope 1.3.1 Pipeline Analysis by Phase 1.3.2 Pipeline Analysis by Molecule Type 1.3.3 Pipeline Analysis by Route of Administration 1.4 Key Stakeholders
2. Research Methodology 2.1 Secondary Research 2.2 Primary Research 2.2.1 Breakdown of Primary Research Respondents 126.96.36.199 by industry participant 188.8.131.52 by company type
3. Executive Summary
4. Pipeline Outlook 4.1 Disease Overview 4.1.1 Types of Neurofibromatosis 4.2 Causes and Symptoms 4.3 Diagnosis 4.3.1 NF1 Diagnosis 4.3.2 NF2 Diagnosis 4.4 Prognosis 4.5 Prevention 4.6 Treatment 4.7 Epidemiology 4.8 Key Drivers 4.9 Key Barriers 4.9.1 Overlapping of NF with Many RAS-Driven Disorders 4.10 Neurofibromatosis Therapeutics Pipeline Analysis 4.10.1 Pipeline Analysis by Phase 4.10.2 Pipeline Analysis by Molecule Type 4.10.3 Pipeline Analysis by Route of Administration 4.10.4 Pipeline Analysis by Company
5. Neurofibromatosis (NF) Therapeutics Pipeline Analysis by Phase (2018) 5.1 Phase II 5.1.1 Selumetinib 184.108.40.206 Clinical trial 220.127.116.11 Clinical trial results 18.104.22.168 Strategic development 5.1.2 Everolimus 22.214.171.124 Clinical trial 126.96.36.199 Clinical trial results 5.1.3 Lapatinib 5.2 Phase I/II 5.3 Phase 0 5.3.1 AR42 188.8.131.52 Clinical trials 184.108.40.206 Strategic development 5.4 Pre-Clinical 5.4.1 LB201 220.127.116.11 Strategic development 5.4.2 LB205 5.5 Discovery 5.5.1 Small Molecule to Inhibit Hsp90-Alpha and TRAP1 for NF Type 2 18.104.22.168 Strategic development
6. Clinical Trials Review 6.1 Clinical Trials by Region 6.2 Clinical Trials by Trial Status