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Msg  152009 of 152168  at  11/20/2020 1:00:59 PM  by

2fingers


ASH 2020 non-oncology presentations

Selected non-oncology presentations at Ash 2020

Project

Company

Detail

Abstract

Etranacogene dezaparvovec

Uniqure

First data from ph3 Hope-B trial in haemophilia B

LBA-6

Giroctocogene fitelparvovec

Sangamo/Pfizer

Haemophilia A, Alta trial, 11 pts, not clear what the cutoff is

671

DTX201/BAY 2599023

Bayer/Ultragenyx

FVIII gene therapy, first-in-human trial, <22mth data

1539

CTX001

Crispr/Vertex

2 SCD subjects free of crises at 12mth & 3mth, generating total haemoglobin of ~10g/dl

4

Oxbryta

Global Blood

72-week data from pivotal Hope study in SCD

1716

FT-4202

Forma Therapeutics

Jul 2020 data cut, 3 SCD patients

679

Mitapivat

Agios

Jul 2020 data cut, 9 SCD patients

681

RP-L102

Rocket Pharmaceuticals

Fanconi anaemia gene therapy, 2 pts at Aug 2020 data cut; registrational trial under way

674

RP-L201

Rocket Pharmaceuticals

LAD-I gene therapy, 2 pts; US filing due 2022

675

 

As with many Ash presentations, up-to-date details will only be made available at the meeting itself. In haemophilia A Sangamo and Ultragenyx investors will look for updated results from studies of the gene therapies giroctocogene fitelparvovec and BAY 2599023 respectively.
 

Ash also promises a significant focus on sickle cell disease; Crispr/Vertex’s CTX001 study had already made waves when its abstract was first disclosed, and it is now apparent that it has been selected for presentation at an Ash press briefing too (Ash 2020 preview – early winners and losers, November 6, 2020).

Agios and Forma will present sickle cell data on their duelling pyruvate kinase R activators, mitapivat and FT-4202 respectively. Investors will hope for more data than each abstract’s July 2020 cutoff discloses.

And a relatively little-known gene therapy player, Rocket Pharmaceuticals, will hope to make a splash with data on two rare disease projects, RP-L102 for Fanconi anaemia and RP-L201 for leukocyte adhesion deficiency-I. A poster on the first two patients treated with the former asset had sent Rocket up 63% over the period of last year’s Ash meeting.

Though fund managers’ end-of-year portfolio rationalisations often cause a post-Ash lull some investors will remain hopeful once the meeting gets under way.

Ash 2020 will take place in virtual format on December 5-8.

(Source: Evaluate Vantage article November 20, 2020)

 


 


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