The current example from GlycoMimetics shows the difficulties with AML RR studies. Their phase 3 trial for AML RR failed on the primary endpoint. Although median survival of 13.1 months (400% longer than other studies) was one of the longest survival times ever achieved, this failed to reach statistical significance because the placebo arm suddenly and inexplicably also achieved one of the longest survival times ever, 12.3 months. On Monday, the public evaluation of all available data took place and there was a surprise. While the AML population R for relapse did not show any success, the other population of AML R for primary refractory achieved an incredible increase in survival time from 10 months in the placebo arm, to a peak of 40 months, with an average of over 30 months in the treatment arm. Nobody can currently explain why, although independent scientists from the Alliance for Oncology, the NCI and the FDA were involved in the evaluation. AML RR remains one of the riskiest investments for investors. Unless there are tangible successes, the share price will remain at a low level.
I've also been invested in biotech for a while. My experience is that preclinical companies often have people at the top, but the brilliant scientists are bad CEOs. In particular, there is often a lack of communication. This is how it is felt by many. I agree with that point. On the other hand, there is little to report in such companies. In the small group of employees, little usually changes, there are no scientific changes that would be relevant for a long time and during clinical studies the company is as blind and deaf to the data as we are. The CEO is often expected to present a possible cooperation candidate or takeover candidate every week. But that is completely unrealistic. It is also unrealistic to expect this to be announced publicly, even if there were non-binding discussions. Such discussions should often remain secret and this would also be critical from the perspective of the stock exchange regulator. Publicly fantasizing about takeovers every time that don't happen could also be interpreted as market manipulation. To receive such an accusation as a company would be fatal in every respect for such a small biotech company. The CEO is doing a balancing act between the investors and how he can continue to finance the company without sales and profits. I understand the criticism and can understand it to a certain extent, but I don't share it to the same extent because there is nothing for me to report. However, I would like to be corrected as I have not been involved with this company for long. I cannot judge what communication errors or promises have been made by the company over the last 6 years.
Can anybody help me further? Hemogenyx submitted an application for approval of a phase 1 study to the FDA last year and gave the green light to start after the problems were resolved. Does anyone have the application or a link where it can be found? This contains important information about the planned course of the study, patient size and duration.
I would like to answer the questions as to why I "only" assume a market capitalization of 500-800 million, assuming phase 3 is positive. My opinion is that it takes time and money to get approval. Difficulties regularly arise in studies, particularly with AML. In addition, it is a gene therapy that has never been tested in AML. It is not known what additional requirements the FDA and the EMA will place on the studies. All of this increases the time factor but above all the cost factor. A normal AML phase 3 study recruits an average of 300-400 patients, and treatment and follow-up are carried out as planned for at least 3 years. Costs of 100 million are still calculated conservatively. Currently 1.3 billion shares have been issued. I expect a reverse split and a subsequent capital increase. That will be a very big dilution. At the end of all things, production must be guaranteed in terms of quality and quantity before an application for approval is made, because that is part of the application. Precisely because this is a potentially groundbreaking new therapy for AML, there are currently so many unknowns that you cannot take into account but should take into account as a possible investor. The various capital increases that will probably be necessary, together with the expected costs that could amount to several hundred million, I realistically see a market capitalization of initially half a billion until approval. Nobody knows if it helps or who it helps. The problem with AML is precisely the different genetic variants. A variety of gene mutations regularly occur in the same AML patient, which has always made treatment so difficult. Whether even CAR T cells will have difficulties there or perhaps only a certain population, no one knows. Yes, if it really is a therapy that changes everything for the majority of patients, then we are talking about potential sales of over 4 billion per year. But I don't count on that when making my investment decision! If that were the case, then the value would not be foreseeable at the moment. I once saw a pharmaceutical company go from $0.5 to $70. But anyone who sets this as their goal will certainly lose a lot of money on biotech investments. The first milestone will be to avoid directly killing the first patient with the treatment. It sounds macabre but that is the reality of AML. I would be happy to take any positive results that come with me. If a possible partner is found who finances part or all of the whole party, then I'll rethink everything!
They have chosen AML as one of the most difficult cancers to treat and in turn one of the most difficult populations within AML, RR. I don't know whether the high-risk group of tsmAML is included, who have a few weeks of life left after diagnosis. Hardly anything has changed in AML treatment in 50 years, and for the most part nothing has changed in 30 years. Only for some subpopulations with certain gene mutations are there special treatments with slightly better survival times. All of this makes it a high-risk investment; over 90% of AML Phase 3 trials over the last 30 years have failed at their primary endpoint. However, if it represents a groundbreaking new therapy for AML, the treatment would quickly become a billion-dollar business. We are currently in phase 1. I estimate from personal experience at other AML companies that even with problem-free studies up to phase 3, success cannot be expected before 2030. Therefore, I see this as one of my earliest (clinical phase status) and riskiest investments. This will either be a total loss or a 3,000% gain.
Hello everyone, I'm new here and newly invested. I just wanted to note that the FDA has a special process for CAR T cell therapy studies. Patients in phase 1 and phase 2 may not be treated simultaneously. This means that patient 1 receives the drug and then you have to wait a certain time (days/weeks) until patient 2 receives the drug, etc. This will extend the study duration compared to "normal" clinical studies.