Latest news with #NeurizonTherapeutics
The Australian
11 hours ago
- Health
- The Australian
Neurizon's NUZ-001 shows promise in Huntington's disease
Neurizon's NUZ-001 and active metabolite NUZ-001 Sulfone show strong neuroprotective effects in a zebrafish Huntington's model Results show potential of drug to counteract early neurodegenerative damage caused by disease Neurizon plans to initiate additional validation studies in mammalian models of Huntington's Special Report: Clinical-stage biotech Neurizon Therapeutics has reached a milestone in development of its lead drug candidate NUZ-001 to treat Huntington's disease. Neurizon Therapeutics (ASX:NUZ), which is dedicated to advancing treatments for neurodegenerative diseases, has rolled out new preclinical data demonstrating significant neuroprotective effects of NUZ-001 and its active metabolite NUZ-001 Sulfone, in a zebrafish model of Huntington's disease. Huntington's disease is a rare, inherited neurodegenerative disorder that causes progressive degeneration of motor function, cognition and mental health. The disease affects between 2.7 and 4.8 per 100,000 people globally with no cure and no disease-modifying treatment. The treatments available only manage symptoms. In the Huntington's disease model, targeted mRNA knockdown of the Htt (huntingtin) protein triggered hallmark disease characteristics, including: Increased cell death Morphological malformations (smaller eyes and swollen hindbrains) Impaired haemoglobin production and Reduced expression of brain-derived neurotrophic factor (BDNF), a critical biomarker of neuronal function and survival. mRNA knockdown is a lab technique that reduces the activity of a specific gene, in this case the HTT gene, which produces the protein involved in Huntington's disease. Treatment with either NUZ-001 or NUZ-001 Sulfone after Htt knockdown: Prevented developmental and morphological abnormalities Attenuated neuronal cell death Restored the delayed production of haemoglobin; and Rescued BDNF expression. Neurizon said the results provided evidence of NUZ-001 and NUZ-001 Sulfone's potential to counteract early neurodegenerative damage. Study details For the preclinical study, wild-type zebrafish embryos were raised in standard conditions. Morpholino antisense oligonucleotides (MOs) targeting Htt mRNA were then injected into one-cell stage embryos to decrease Htt expression. NUZ-001 or NUZ-001 Sulfone at 1 and 10 μM concentrations were added to the embryonic media six hours post-fertilisation to evaluate the protective effects on Htt knockdown-induced deficits. At two days post-fertilisation changes in morphology (eye size and hindbrain swelling), neuronal cell death (apoptosis), haemoglobin levels, and the BDNF expression levels were analysed. Source: Neurizon Therapeutics Knockdown of Htt (Htt MO) resulted in smaller eyes and swollen hindbrain ventricles in zebrafish embryos. Neurizon said partial rescue of eye size and full reversal of hindbrain swelling were observed with 10 μM NUZ-001 and NUZ-001 Sulfone (Figure 2b). Source: Neurizon Therapeutics Other key findings of the study include: Neuronal cell death was significantly higher in the Htt knockdown group, while treatment with 1 μM and 10 μM NUZ-001, and 10 μM NUZ-001 Sulfone, significantly reduced apoptosis Haemoglobin levels were significantly decreased in the Htt knockdown group but partially restored by both concentrations of NUZ-001 and NUZ-001 Sulfone; and Expression of BDNF transcripts was significantly rescued with 10 μM NUZ-001 and 10 μM NUZ-001 Sulfone. Watch: Last patient completes treatment in OLE study NUZ-001 showing promise NUZ-001 is currently in clinical development for the most common form of motor neurone disease (MND) called amyotrophic lateral sclerosis (ALS), where it has shown: Preclinical efficacy in enhancing proteostasis Reducing pathological protein aggregation; and Preserving neuronal function. The company said new findings in the Huntington model further underscore NUZ-001's potential as a platform therapy targeting core cellular stress and clearance mechanisms common to multiple neurodegenerative diseases. Neurizon plans to advance additional preclinical studies in mammalian models of Huntington's disease as part of its broader strategy to expand NUZ-001's therapeutic applications to other progressive neurological disorders with high unmet need. 'These results mark another important milestone in the realisation of the potential for NUZ-001 to treat a range of neurodegenerative diseases,' CEO and managing director Dr Michael Thurn said. 'Huntington's disease is a devastating, rare genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to a range of symptoms including uncontrolled movements, cognitive decline, and emotional disturbances. 'These exciting results demonstrate NUZ-001 has consistent neuroprotective effects beyond amyotrophic lateral sclerosis (ALS), strengthening our conviction in NUZ-001's potential as a disease-modifying platform therapy across a range of neurodegenerative conditions.' This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing. This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
Yahoo
11-05-2025
- Business
- Yahoo
We Think Neurizon Therapeutics (ASX:NUZ) Needs To Drive Business Growth Carefully
We can readily understand why investors are attracted to unprofitable companies. For example, biotech and mining exploration companies often lose money for years before finding success with a new treatment or mineral discovery. But while the successes are well known, investors should not ignore the very many unprofitable companies that simply burn through all their cash and collapse. So, the natural question for Neurizon Therapeutics (ASX:NUZ) shareholders is whether they should be concerned by its rate of cash burn. In this report, we will consider the company's annual negative free cash flow, henceforth referring to it as the 'cash burn'. We'll start by comparing its cash burn with its cash reserves in order to calculate its cash runway. Our free stock report includes 6 warning signs investors should be aware of before investing in Neurizon Therapeutics. Read for free now. You can calculate a company's cash runway by dividing the amount of cash it has by the rate at which it is spending that cash. As at December 2024, Neurizon Therapeutics had cash of AU$10m and no debt. In the last year, its cash burn was AU$10m. So it had a cash runway of approximately 12 months from December 2024. That's quite a short cash runway, indicating the company must either reduce its annual cash burn or replenish its cash. You can see how its cash balance has changed over time in the image below. View our latest analysis for Neurizon Therapeutics Although Neurizon Therapeutics reported revenue of AU$1.5m last year, it didn't actually have any revenue from operations. To us, that makes it a pre-revenue company, so we'll look to its cash burn trajectory as an assessment of its cash burn situation. Remarkably, it actually increased its cash burn by 567% in the last year. With that kind of spending growth its cash runway will shorten quickly, as it simultaneously uses its cash while increasing the burn rate. Clearly, however, the crucial factor is whether the company will grow its business going forward. So you might want to take a peek at how much the company is expected to grow in the next few years. Given its cash burn trajectory, Neurizon Therapeutics shareholders should already be thinking about how easy it might be for it to raise further cash in the future. Generally speaking, a listed business can raise new cash through issuing shares or taking on debt. One of the main advantages held by publicly listed companies is that they can sell shares to investors to raise cash and fund growth. By comparing a company's annual cash burn to its total market capitalisation, we can estimate roughly how many shares it would have to issue in order to run the company for another year (at the same burn rate). Neurizon Therapeutics has a market capitalisation of AU$69m and burnt through AU$10m last year, which is 15% of the company's market value. Given that situation, it's fair to say the company wouldn't have much trouble raising more cash for growth, but shareholders would be somewhat diluted. On this analysis of Neurizon Therapeutics' cash burn, we think its cash burn relative to its market cap was reassuring, while its increasing cash burn has us a bit worried. Looking at the factors mentioned in this short report, we do think that its cash burn is a bit risky, and it does make us slightly nervous about the stock. On another note, Neurizon Therapeutics has 6 warning signs (and 3 which don't sit too well with us) we think you should know about. Of course, you might find a fantastic investment by looking elsewhere. So take a peek at this free list of companies with significant insider holdings, and this list of stocks growth stocks (according to analyst forecasts) Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
