Aptorum Group Limited (APM) Earnings Call Transcript & Summary
April 23, 2022
Earnings Call Speaker Segments
Craig Brelsford
attendeeDarren, welcome back. Great to see you as always.
Darren Lui
executiveHi, Craig. Nice to be here.
Craig Brelsford
attendeeAptorum Group is a pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs. For viewers new to the story, Darren, let's start with an overview of your current development pipeline.
Darren Lui
executiveSure. So Aptorum Group, we are a NASDAQ-listed clinical-stage biopharmaceutical company. We focus on a number of unmet medical needs, including in areas of infectious disease, oncology, autoimmune disease, as well as women's health. Some of our key pipeline right now, we have 2 drug candidates, which currently have completed the Phase I clinical trials and will be shortly embarking on the Phase II clinical trials for proof-of-concept, which here, in this case, include 2 drugs, which is the ALS-4 drug against a very deadly bacterial disease called MRSA, as well as a second drug, which is targeting a highly unmet oncology disease or neuroblastoma, both of which have now since completed our Phase I clinical trials. We also have our third product, which is what we call RPIDD technology, which is a liquid biopsy-based technology combined with genetic sequencing approach. We provide diagnosis of infectious disease inside the patient's body. It is a very exciting project. It's a collaboration with a Singapore research institute called A*Star. And this product is currently also under human clinical validation as well, and we'll be looking to commence pre-commercialization steps preparation over the course of beginning of 2023. And lastly, we also have an asset, which is in women's health space, which is actually targeting women's menopause. This particular supplement is going to be commercialized quite shortly in parts of Asia as well as major cities in Europe, and we'll be looking to start the distribution process subject to registration in the United States, hopefully, later this year as well for this particular women's health menopause supplement. We also recently announced a very exciting oncology and also autoimmune platform as well, which are actually targeting latest generation of cancer mutations, which also are highly unmet disease areas, in order to boost our pipeline.
Craig Brelsford
attendeeThank you, Darren, for that overview of your pipeline. I want to turn to the first or second one that you mentioned just now. SACT-1, that is the repurposed small molecule compound for the treatment of patients with neuroblastoma. In January, Aptorum announced that the United States Food and Drug Administration had granted orphan drug designation to SACT-1. Talk about that and tell us why SACT-1 is so promising a drug with which to fight neuroblastoma.
Darren Lui
executiveSure. For those who don't know what neuroblastoma, it's actually an orphan cancer. So it's actually a rare form of cancer, which are predominantly affecting children below 12 years old. It's basically cancer that's grown along the nerve tissues, and it could be anywhere in your body. Unfortunately, neuroblastoma patients have a very low survival rate. The 5-year survival rate is on average about 40%. But 6 out of 10 children do not make it beyond the 5 years. And it is a disease that is highly focused by the FDA and highly encouraged for development by the U.S. FDA. And within that, if you follow us, we have our own drug discovery unit, where in the last number of years, we have identified, through our 2,600 drug library, we've identified one particular candidate, which was previously an FDA-approved drug that appears to be effective and efficacious against neuroblastoma disease. As a result of that, we have selected that particular candidate to push forward with its new formulation work. We have been granted recently a patent for the neuroblastoma indication using the drug. And also on top of that, this drug has now recently completed a Phase I clinical trial in the United States and it is now ready to proceed on to what we call the Phase Ib/IIa clinical trials subject to the respective IND application being cleared. And as you rightly said, we have recently been granted by the U.S. FDA what we call the orphan drug designation and effectually recognizing that this particular drug by the FDA is actually available to benefit from the orphan drug review process, which usually involves a more rapid process in the review. And also it provides a 7-year marketing exclusivity to the extender once the drug has been approved for marketing later on when they complete this clinical trials as well.
Craig Brelsford
attendeeDarren, in January, Aptorum announced the launch of its oncology and autoimmune discovery and development platform. This platform targets unmet mutations and novel biomarkers. Please tell our audience how the platform could help address the unmet medical needs of patients suffering from non-small cell lung cancer and possibly other types of cancer.
Darren Lui
executiveSure. So for those who don't know what non-small cell lung cancer, it's a form of a subset of lung cancer, and usually, non-small cell lung cancer is considered to be a highly unmet and highly fatal disease. And the reason for the various mutations is that cancers constantly mutate themselves, especially when it's being stimulated by therapeutic agents. So as we apply more chemotherapy or other types of treatment, typically, cancer cells are able to respond and able to evolve and develop resistance to these chemical agents as a result. So as a result, many non-small cell lung cancer patients, for example, will go through, over a couple of years, different forms of mutations, different generations of mutations of the lung cancer cells, and you'll be basically reaching towards the end of its life where the non-small cell lung cancer cells itself are resistant to basically all the available current therapeutics in the market. So as a result, the patients' mortality is extremely high if the cancer cells are able to mutate to that latest generation. So for example, as you can see in some of our presentations on our website, one of the typical mutations is called EGFR. EGFR mutation is currently now upcoming to what we call a fourth-generation mutation, which is currently a highly unmet mutation class of non-small cell lung cancer and typically entail a very high mortality rate for patients. And these are the areas where Aptorum is very excited to now announce our own drug design and drug discovery process to target these what we call highly unmet latest generation mutation of these cancer types. And we have 2 core focuses and one of them is a what we call next-generation inhibitor, which is an approach towards targeting the mutation as well as supplement it and basically increase in efficacy potentially by combination with protein degradation approach to supplement the efficacy of the next generation inhibitors. So you see that on our website, there are currently 2 focuses of Aptorum, targeting a next-generation inhibitor, potentially in combination with protein degradation approach to target these types of mutations including EGFR mutations and ALK mutations in their final -- latest generation of mutations as our next generation of clinical drugs available for these diseases.
Craig Brelsford
attendeeHey, Darren, let's go back to that pipeline. Earlier, you mentioned RPIDD. That's R-P-I-D-D, which stands for rapid pathogen diagnostics liquid biopsy technology. Recently, you announced -- Aptorum announced that Aptorum Innovations Holding Limited, your subsidiary company, had commenced the clinical validation of this molecular-based technology for the diagnosis of pathogens such as viruses, bacteria, fungi and parasites. Please describe the validation and its possible implications.
Darren Lui
executiveSure. For those who don't know about our RPIDD technology, it's basically our next generation liquid biopsy based approach towards infectious disease diagnostics. And for those who don't know what liquid biopsy is, we're basically using a small blood sample to perform genetic sequencing analysis on what we call an untargeted, unbiased basis towards identifying the type of infectious disease that are currently affecting the patient's body, which is evident through the genetic materials inside the blood sample itself. It's a very exciting project. We believe that it has a major impact to disrupt the first-line diagnostics technology that are currently available in the market right now, which is typically blood culture, which people are fully aware that they are actually very often inaccurate, ineffective, and takes a long duration to generate the results as well, because it basically involves growing the cells in a laboratory space. So we believe that actually RPIDD is a very exciting technology that could potentially replace this first line of diagnostics that are currently used in the market, and also is able to pick up a much more deep and complete data range of the potential infectious disease. And this includes both DNA, RNA, virus, bacteria and no infection inside the blood based on the genetic materials available through the blood sample.
Craig Brelsford
attendeeDarren, to briefly review, you've given us a very exciting pipeline. You've given us a product that you're already commercializing. Now in summary, let's wrap it all up into an essential value proposition. Why should investors take an interest in Aptorum Group right now?
Darren Lui
executiveI think this year is a very exciting year for Aptorum. As I said, we are already in a clinical stage of biopharmaceutical company. We have multiple shots on target, including our ALS-4 MRSA molecule program, which is about to start the Phase II clinical trials, as we said, subject to an IND clearance, as well as our SACT-1 also looking to embark on to what we call Phase Ib/IIa human clinical trials for initial readout of its efficacy in the United States, also subject to the IND clearance. So all of this is going to happen on this side of the year, in 2023, as well as our validation process in our RPIDD technology, our human clinical validation, which is ongoing at the moment, where we are yet to announce the very exciting results of this particular technology in the later part of this year once we get it ready to start the pre-commercialization stage over the course of 2023. And on top of that, we have our women's health menopause supplement, which are currently in commercialization stage and shortly will be available for distribution in major countries, including Europe and Asia, as well as hopefully later this year in the United States as well. So hopefully, we'll be able to bring these very exciting products into their respective clinical stage very, very shortly, hopefully, subject to our ongoing preclinical work of those products.
Craig Brelsford
attendeeIt's a great story, Darren. Thank you for sharing it with us today.
Darren Lui
executiveThank you. It's nice to be here.
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