Appendix 4C – Q2 FY22 Quarterly Cash Flow Report
- NZ’s regulator Medsafe authorizes Phase 2 clinical trial for ATH434; more jurisdictions to follow
- New poster presentation and publications provide further evidence of potential of ATH434 to treat neurodegenerative diseases
- New US composition of matter patent secures exclusivity for a new class of compounds targeting Parkinson’s and Alzheimer’s diseases
- Active investor relations program in Australia and US
- Cash balance on 31 December 2021 of A$37M
Alterity Therapeutics Limited (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, releases its Appendix 4C Quarterly Cash Flow Report and update on company activities for the quarter ending 31st December 2021 (Q2 FY22).
The Company’s cash position on 31 December 2021 was $37M with operating cash outflows of $4M. This was consistent with company expectations and largely due to the preparation for the Phase 2 clinical trial for Alterity’s lead drug candidate ATH434 in Multiple System Atrophy (MSA), a Parkinsonian disorder with no approved therapy.
In accordance with ASX Listing Rule 4.7C, payments made to related parties and their associates included in item 6.1 of the Appendix 4C incorporates directors’ fees, consulting fees, remuneration and superannuation at commercial rates.
Alterity continues to identify new drug candidates to expand its portfolio and protect its therapeutic approach to address neurodegeneration.
On 14th December, Alterity announced that the New Zealand Medicines and Medical Devices Safety Authority (Medsafe) had authorized Alterity’s Phase 2 clinical trial for ATH434 in MSA to be recruited in that country. This is the first jurisdiction to authorize the trial with further countries to follow this year. Alterity expects to launch the trial in the first quarter of 2022.
The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients with early-stage MSA. The study will explore the effect of ATH434 treatment on imaging and protein biomarkers such as aggregating α-synuclein and excess iron, which are important contributors to MSA pathology. Clinical endpoints and other biomarkers will permit comprehensive assessment of ATH434 efficacy along with characterization of safety and pharmacokinetics. Patients will receive treatment for 12 months which will provide an opportunity to detect changes in efficacy endpoints to optimize design of a definitive Phase 3 study.
In November, Alterity gave a poster presentation at the American Autonomic Society 32nd Annual International Symposium. The poster, entitled, “Cardiovascular safety and pharmacokinetics of ATH434, a novel small molecule inhibitor of α-synuclein aggregation, in adults and older adults”, described results from the Company’s Phase 1 clinical trial conducted in healthy volunteers. In the Phase 1 trial, ATH434 was well tolerated in adult and ≥ 65-year-old volunteers and demonstrated
no cardiac adverse event signal and no clinically significant changes in blood pressure or heart rate at any dose. ATH434 also demonstrated dose dependent pharmacokinetics (PK) after single and multiple oral doses and a half-life that supports twice-daily dosing.
Alterity’s profile continues to bolster with the publication of two preclinical studies demonstrating the potential of ATH434 to treat Parkinsonian disorders. Non-motor symptoms are common in patients with Parkinsonian disorders, such as Parkinson’s disease and MSA. Parkinson’s disease patients experience gastrointestinal complications, cognitive deficits, autonomic dysfunction, and mood disturbance and these non-motor manifestations are an important source of morbidity and reduced quality of life. As published in the Journal of Parkinson’s Disease, “ATH434 Reverses Colorectal Dysfunction in the A53T Mouse Model of Parkinson’s Disease” presents results from a preclinical study investigating the effect of ATH434 on gastrointestinal complications. Alterity also announced the publication in Plos One of an in vitro study concluding that the novel mechanism of action of ATH434 provides a compelling case for its continued development as a therapeutic agent in neurodegenerative diseases associated with iron accumulation.
Activity is also increasing within the investment community in Australia and the US with Alterity’s Chief Executive Officer, David Stamler, MD, presenting at the MST Financial Lifesciences & Biotech Forum and the Benchmark Company Discovery One-On-One Investor Conference in 2021. Dr. Stamler also presented at the at the H.C. Wainwright BIOCONNECT Virtual Conference during the annual JP Morgan Healthcare Conference in January 2022.
Post the reporting period, the Company announced the United States Patent and Trademark Office granted a composition of matter patent that was issued a Notice of Allowance in August 2021. The patent covers more than 80 novel compounds and secures exclusivity for a new class of iron chaperones designed to redistribute the excess iron implicated in many neurodegenerative diseases, including Parkinson’s and Alzheimer’s diseases.
“This past quarter has been highly active as we move toward the initiation of our Phase 2 clinical trial for MSA with our profile significantly bolstered within the investment, patient, clinical and research communities,” said Dr Stamler. “It’s an exciting time for our company, but more importantly for clinicians and patients who understand intimately the devastation of the diseases we are targeting and understand the urgent need to develop new treatments that address the underlying pathology of these diseases.”
About Multiple System Atrophy
Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by a combination of symptoms that affect both the autonomic nervous system and movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability. MSA is a Parkinsonian disorder characterized by motor impairment, autonomic instability that affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within the support cells of the central nervous system and neuron loss in multiple brain regions. MSA affects approximately 15,000 individuals in the U.S., and while some of the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no cure.1
1National Institute of Health: Neurological Disorders and Stroke, Multiple System Atrophy Fact Sheet
Alterity’s lead candidate, ATH434, is the first of a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve nerve cells by restoring normal iron balance in the brain. In this way, it has excellent potential to treat Parkinson’s disease as well as various forms of atypical Parkinsonism such as Multiple System Atrophy (MSA). ATH434 has successfully completed a Phase 1 clinical trial demonstrating the agent is well tolerated, orally bioavailable, and achieved brain levels comparable to efficacious levels in animal models of MSA, with the objective of restoring function in patients with MSA and other Parkinsonian disorders.
ATH434 has been granted Orphan designation for the treatment of MSA by the U.S. FDA and the European Commission.
About Alterity Therapeutics Limited
Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company’s lead asset, ATH434, has the potential to treat various Parkinsonian disorders. Alterity also has a broad drug discovery platform generating patentable chemical compounds to intercede in disease processes. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company’s web site at www.alteritytherapeutics.com.
Authorization & Additional information
This announcement was authorised by David Stamler, CEO of Alterity Therapeutics Limited.