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use | Investor Presentation |
ersonal | Investor Presentation | 31 January 2022 |
Gary Phillips CEO
developing breakthrough treatments for fibrosis and inflammation
Forward looking statement
onlyThis document contains forward-looking statements, including statements concerning Pharmaxis' future financial position, plans, and the potential of its products and product candidates, which are based on information and assumptions available to Pharmaxis as of the date of this document. Actual results, performance or achievements could be
usesignificantly different from those expressed in, or implied by, these forward-looking statements. All statements, other than statements of historical facts, are forward-looking statements.
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These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward- looking statements as a result of new information, future events or otherwise.
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Executive Summary
• Pharmaxis is a clinical stage drug development company targeting fibrosis and
cancer indications with first in class or best in class small molecule drugs in markets of high value
• Pharmaxis is the global leader in fibrosis driven by lysyl oxidase enzymes having
only | invested in a multi year research program leveraged with extensive external |
scientific collaborations | |
• Pharmaxis has 4 studies planned for 2022 that will lead to near term value | |
use | opportunities |
• Lead asset PXS-5505 is in a multinational phase 2 trial - a breakthrough clinical program | |
with disease modifying potential in Myelofibrosis | |
• IND approval to commence US investigator led phase 2 trial in liver cancer with PXS- | |
5505 as first line treatment added to existing chemotherapy. | |
• Topical drug PXS-6302 is in a phase 1c trial in patients with potential to improve | |
function and appearance of established scars with a study in burns patients to follow | |
later this year. | |
• Specific corporate strategy to deliver non-dilutive cash and cost savings from | |
commercial stage mannitol business | |
• Pharmaxis is in a strong position to fund its focused clinical program | |
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Clinical stage medicines
Targeting fibrosis
and cancer
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Pharmaxis is the global leader in lysyl oxidase chemistry and biology
Multi year research program leveraged with extensive scientific collaborations worldwide has delivered 2 drugs in the clinic
Lysyl oxidases are the final stage in fibrosis
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Tissue stiffening due to increases in collagen and number of cross-links is preventable through lysyl oxidase inhibition and at the heart of a true anti-fibrotic therapy
- PXS-5505
- Oral dosage form - one capsule twice a day
- Patent 2018
- Strong pre clinical evidence in models of fibrosis and cancer
- INDs approved for myelofibrosis and hepatocellular carcinoma
- Potential in multiple cancer indications
- Phase 1 data demonstrates a safe, well tolerated drug that gives >90% inhibition of LOX enzymes
- PXS-6302
- Topical dosage form - one application per day
- Patent 2019
- Strong pre clinical evidence in models of skin fibrosis and scarring
- Potential in prevention of scar formation and modification of existing scars
- Phase 1 data demonstrates a safe, well tolerated drug that gives full inhibition of LOX enzymes in the skin with minimal systemic exposure
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Myelofibrosis background
A rare type of bone marrow cancer that disrupts your body's normal production of blood cells
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KEY FACTS
Affects 15 in 1m people worldwide
5 Years Median survival
Age of onset 50 - 80
11% transformation to leukemia
Primary Myelofibrosis is caused by a build up of scar tissue (fibrosis) in bone marrow reducing the production of blood cells:
- Reduced red blood cells can cause extreme tiredness (fatigue) or shortness of breath
- Reduced white blood cells can lead to an increased number of infections
- Reduced platelets can promote bleeding and/or bruising
- Spleen increases blood cell production and becomes enlarged
- Other common symptoms include fever, night sweats, and bone pain
Standard of Care; JAK inhibition
- Symptomatic relief plus some limited survival improvement. 75% discontinuation at 5 years
- Median overall survival is 14 - 16 months after discontinuation
Commercial Opportunity
- Current standard of care; revenue ~US$1b per annum
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Pharmaxis Limited published this content on 31 January 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 31 January 2022 06:40:56 UTC.