Investor Presentaiton
For personal use only
New hope in a rare disease
Progressing development of TLX66 in bone marrow conditioning
SALA1 is a rare disease with a poor prognosis (median survival ~11 months if untreated)
TELIX
PHARMACEUTICALS
•
Plasma cells in the bone marrow produce abnormal protein called 'amyloid' which accumulates
in the organs and causes them to fail
•
Prevalence of ~30,000 to 45,000 (US + EU combined) patients, ~US$600M² TAM in US and 'EU5'
Faulty
plasma cells
•
Current standard of care comprises induction therapy (cyclophosphamide, bortezomib,
dexamethasone) plus high dose melphalan BMC³, followed by HSCT4,
5
V
Free antibody
•
A novel monoclonal antibody, daratumumab has potential as an initial therapy for patients but is
not curative or suitable for all patient populations
light chain
C C
TRALA study: Phase I trial of 90Y-besilesomab (TLX66) in SALA
.
Primary endpoint: Safety and toxicity of 90Y-besilesomab as the sole BMC regimen for
autologous HSCT in patients with SALA
Amyloid
protein
accumulates
in organs
•
Study complete, preliminary data (9 pts) demonstrated 100% engraftment and high PR/CR
rate (5/2) survival data. Regulator consultation in progress for next phase of development
1. Systemic amyloid light chain amyloidosis.
Management estimate based on reported incidence x estimated unit
dosing price, based on prevalence of 12M persons per year (Monique
Minnema and Stefan Schönland, The EBMT Handbook: Hematopoietic Stem Cell
Transplantation and Cellular Therapies, 2019)
Telix Pharmaceuticals Limited (ASX: TLX)
1. Bone marrow conditioning.
2. Hematopoietic stem cell transplant.
3. Venner C, et al. Blood. (2012) 119 (19): 4387-4390.
4. https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-002231-18/GB
Organ failure, death
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