Haemophilia A - KOL Insight

Description

Altuviiio? Despite gaining European approval, experts are uncertain about the prospects for BioMarin's Roctavian - why? Could Novo Nordisk's next-generation FVIII mimetic pipeline antibody Mim8 be superior to Roche's SoC Hemlibra? KOLs critically assess the prospects of launched and pipeline therapies.

In addition to the full report, licensed users have access to the Report Highlights slide deck and the following KOL Bulletins via the Attachments area.

16 Jun 23 KOL Bulletin -- Expert opinion on the Phase III BASIS data for Pfizer's marstacimab

16 Jun 23 KOL Bulletin -- Expert perspective on Phase III ATLAS-PPX data for Alnylam and Sanofi's fitusiran

11 Jul 23 KOL Bulletin -- Leading haematologist opines on US approval of BioMarin's haemophilia A gene therapy Roctavian

Executive summary

Current and future treatment algorithm

Research objectives

Marketed therapies

Blood coagulation factor stimulant
- Key insights summary
- Hemlibra (emicizumab; Roche)
Recombinant Factor VIII therapies
- Newer long-acting recombinant Factor VIII therapies
- Altuviiio (efanesoctocog alfa; Amunix/Bioverativ [Sanofi])

Pipeline products

Non-factor replacement therapies

Gene therapy
- Key insights summary
- ASC618 (CRISPR/cas9-edited hFVIII gene therapy; ASC Therapeutics)
- Peboctocogene camaparvovec (DTX201; Bayer HealthCare/Dimension Therapeutics)
- Giroctocogene fitelparvovec (PF-07055480/SB-525; Sangamo Therapeutics/Pfizer)
- Dirloctocogene samoparvovec (SPK-8011) and SPK-8016 (Spark Therapeutics)
- TAK-754 (SHP654; Takeda)
- Roctavian (valoctocogene roxaparvovec; BioMarin Pharmaceutical)
Anti-tissue factor pathway inhibitor mAbs
- Key insights summary
- Concizumab (anti-TFPI mAb; Novo Nordisk)
- Marstacimab (anti-TFPI mAb; Pfizer)
FVIII mimetic antibody
- Key insights summary
- Mim8 (NNC0365-3769 A; Novo Nordisk)
Antithrombin III inhibitors/RNA interference
- Key insights summary
- Fitusiran (Alnylam Pharmaceuticals/Sanofi)
Activated protein C receptor modulator
- Key insights summary
- SerpinPC (Centessa Pharmaceuticals)
Other early-stage programmes
- Key insights summary
- BT200 (RNA modulator/vWF inhibitor; Band Therapeutics)
- IDO 8 (tolerogenic dendritic cell therapy; Idogen)