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Telix Pharmaceuticals Ltd — Investor Presentation 2021
May 24, 2021
31324_rns_2021-05-24_8dc99933-ecab-403c-8ff6-8f35d3a07e90.pdf
Investor Presentation
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Precision Oncology
See it. Treat it.
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New Hope in a
Before After
Rare Disease
Post TLX66
TRALA Trial of TLX66
Pre TLX66 (complete bone
( [90] Y-besilesomab) in SALA marrow aplasia)
– Shareholder Update
25 May 2021
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25 May 2021
Disclaimer
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The information contained in this presentation is not intended to be an offer for subscription, invitation or recommendation with respect to shares of Telix Pharmaceuticals Limited (“Telix”) in any jurisdiction, including the United States. No representation or warranty, express or implied, is made in relation to the accuracy or completeness of the information contained in this document or opinions expressed in the course of this presentation. The information contained in this presentation is subject to change without notification.
This presentation may contain forward-looking statements which can be identified by the use of words such as “may”, “should”, “will”, “expect”, “anticipate”, “believe”, “estimate”, “intend”, “scheduled” or “continue” or similar expressions. Any forward-looking statements contained in this presentation are subject to significant risks, uncertainties, assumptions, contingencies and other factors (many of which are outside the control of, and unknown to Telix, and its directors, officers, employees, agents or associates), which may cause the actual results or performance to be materially different from any future result so performed, expressed or implied by such forward-looking statements.
There can be no assurance or guarantee that actual outcomes will not differ materially from these statements. The data and results pertaining to clinical subjects used in this presentation are illustrative of medical conditions and outcomes associated with potential applications of Telix’s product pipeline. Actual results from clinical trials may vary from those shown. None of the products or potential products described in this presentation have received a marketing authorization in any jurisdiction.
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Telix Pharmaceuticals Limited (ASX: TLX)
Executive Summary – New Hope in a Rare Disease
Systemic Amyloid Light-Chain Amyloidosis (SALA): A rare disease with few treatment options
TLX66 ([90] Y-besilesomab) is a potential therapeutic platform for SALA
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Plasma cells in the bone marrow produce abnormal protein called ‘amyloid’ which accumulates in the organs, causing them to fail.
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Potential for significantly reduced toxicity and improved tolerability compared with existing chemotherapeutic conditioning approaches.
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Incidence rate of ~12 per 1,000,000 population per annum, estimated prevalence of 30,000 to 45,000 patients in the United States and the European Union.[(1)]
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Successful engrafting of patients without the use of aggressive (in terms of morbidity/mortality) therapeutic approaches such as chemotherapy/melphalan.
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~USD $600M addressable market for US/EU5.
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Potential for significantly reduced hospitalisation time and favourable health economic profile.
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Current standard of care for transplant conditioning uses toxic chemotherapeutic bone marrow conditioning regimens, with significant morbidity and mortality.[(2)]
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Opportunity in older patients and those with other morbidities, advanced disease or complications of their disease.
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A novel CD38 monoclonal antibody, daratumumab has potential as an initial therapy for patients but is not curative or suitable for all patient populations.
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Potential to expand TLX66 into conditioning in other stem cell transplantation (SCT) areas.
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Pediatric use already in clinical trials.
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Encouraging data to take forward in this rare disease
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Develop pivotal registration study design in collaboration with the Amyloid community of patients and physicians.
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Extend orphan indication in US (EU orphan designation is for “treatment in haematopoietic stem cell transplantation”).
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Present development plans to FDA and EMA.
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Expand into other areas of SCT , including reduced intensity conditioning and other rare diseases.
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Independent academic Pediatric Phase II study of TLX66 at leading London Children's hospital awaiting ethics approval.
Notes: 1. Quock TP et al. Blood Advances. 2018.
- Hasib Sidiqi M et al. Journal of Clinical Oncology. 2018
Telix Pharmaceuticals Limited (ASX: TLX)
What is SALA?
Systemic Amyloid Light-Chain Amyloidosis
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B-cell disorder localized to the bone marrow.
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Rare disease with with an significant impact on survival.[(1)]
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Amyloid accumulation from faulty plasma cells leads to multiple organ failure.
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Prevalence of 30,000 to 45,000 patients in the United States and the European Union[(2)] , ~USD $600M addressable market for US/EU5.
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Current standard of care comprises induction therapy (cyclophosphamide, bortezomib, dexamethasone) plus high dose melphalan bone marrow conditioning (BMC), followed by HSCT[(3)] :
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This is only accessible to 10-15% of patients being able to tolerate treatment and has high treatment-related mortality even with careful patient selection.
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If a complete response is achieved, patients can live 10 yrs+.
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Daratumumab is showing potential to improve outcomes but is not curative or suitable for all patient populations.
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Role for SCT will be in recurrence / no responders / ineligible / advanced disease.
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Existing condition regimens have serious life-threatening side effects due to high dose melphalan: Mucositis, sepsis, nausea, vomiting, diarrhoea causing extended in-patient care.
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A more tolerable SCT condition regimen has potential to increase the number of patients successfully treated with this standard.
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Organ failure,
death
Dysfunctional Free antibody Amyloid fibrils,
plasma cells light chain accumulate in organs
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Notes: 1. See: https://onlinelibrary.wiley.com/doi/full/10.1002/ajh.23828
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Quock TP et al. Blood Advances. 2018.
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Venner C, et al. Cyclophosphamide, bortezomib, and dexamethasone therapy in AL amyloidosis is associated with high clonal response rates and prolonged progression-free survival. Blood. (2012) 119 (19): 4387–4390.
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Telix Pharmaceuticals Limited (ASX: TLX)
Unmet Medical Need for HSCT Transplantation
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The number of transplants, and indications for transplant continue to increase year on year
Use of hematopoietic stem cell transplantation (HSCT) is growing[(1)] in malignant hematological conditions and expanding in non-hematological malignancies as well as rare / immune-mediated diseases.
Conditioning with chemotherapy is associated with high levels of toxicity, and excludes patients who could not tolerate chemotherapy, for example those with rare complex diseases.
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Total number of HSCTs performed in the
U.S. and reported to CIBMTR [(1)]
25,000
20,000
15,000
2013 2014 2015 2016 2017
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Notes: 1. Average annual growth is >20% according to the Center for International Blood and Marrow Transplant Research (CIBMTR).
Telix Pharmaceuticals Limited (ASX: TLX)
TLX66 + HSCT is a Potential Therapeutic Platform for SALA
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90Y-anti-CD66-MTR is potentially an ideal BMC agent for HSCT
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Compared with existing
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Significantly reduced toxicity chemotherapeutic
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• Improved tolerability approaches[(1, 2)]
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Potential for outpatient administration:
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ü Reduced hospitalisation time
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Favourable health economic profile due to:
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ü Reduced hospitalisation requirements ü Reduced side effects from chemotherapy
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ü Lower requirement for supportive care of the patient
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More broadly applicable in:
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ü Older patients
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ü Patients with other morbidities (e.g. poor renal function)
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Potential to extend in multiple other conditioning regimens including pediatric use
Indications for Hematopoietic Stem Cell Transplant (US, 2018)[1]
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Notes: 1. Orchard KH, et al. Targeted radiotherapy in the conditioning prior to haematopoietic stem cell transplantation: results of a Phase I trial using an yttrium90-labelled anti-CD66 murine monoclonal antibody demonstrating consistently high BM uptake. Biol Blood Marrow Transplant . 2006;12:10–11.
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Fasslrinner F, et al. Radioimmunotherapy in Combination with Reduced-Intensity Conditioning for Allogeneic Hematopoietic Cell Transplantation in Patients with Advanced Multiple Myeloma. Biol Blood Marrow Transplant. 26 (2020) 691-697.
Telix Pharmaceuticals Limited (ASX: TLX)
The TRALA Study
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Ta R AL A rgeted adiotherapy for myloidosis
TRALA Treatment Protocol – Overview
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Phase I/IIa dose escalation of targeted radiotherapy alone for stem cell transplant conditioning in SALA.
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Key eligibility:
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Diagnosis of new or recurrent systemic AL-amyloidosis.
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Measurable clonal plasma cell dyscrasia.
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Amyloid related organ dysfunction or organ syndrome.
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Estimated life expectancy of at least 6 months.
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Primary outcome – tolerability.
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Secondary outcomes include disease response.
Treatment Regime
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Telix Pharmaceuticals Limited (ASX: TLX)
The TRALA Study – Phase I Results
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A well-tolerated treatment successfully engrafting patients without chemotherapy
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Nine patients with AL amyloidosis received TLX66 as the sole bone marrow conditioning agent prior to undergoing autologous HSCT.
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ALL completed the trial.
Example Treatment Response Profile Patient lives disease free for more than a year
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ALL were successfully engrafted.
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Disease response as measured by fall in clonal free light chains (FLC) seen in seven out of the nine patients.
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Two of nine demonstrated a durable complete response (CR) within the first 100 days post-transplant.
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Reduction in the measurable malignant plasma cell was seen in six of eight evaluable patients.
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ALL patients remain alive with a reduced need for further Amyloid therapy.
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TLX66 demonstrated a favourable safety profile and was very well tolerated in all patients – especially in comparison to chemotherapybased regimens.
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TLX66
100
90
80
70
60
50
40
30
20
10
0
Date of treatment
31/03/1630/04/1631/05/1630/06/1631/07/1631/08/1630/09/1631/10/1630/11/1631/12/1631/01/1728/02/17
lambda
FLC mg/L
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Telix Pharmaceuticals Limited (ASX: TLX)
Next Steps
Extension of orphan indication in US
Develop pivotal registration study design in collaboration with the Amyloid community of patients and physicians
Present development plans to FDA and EMA in 2H 2021
Expand into other areas of SCT Independent Academic Pediatric Phase II study planned in Q3 2021
Telix Pharmaceuticals Limited (ASX: TLX)
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telixpharma.com
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