Would the FDA grant Orphan Drug status based on the December readout, when a new readout is already imminent, or would this foreshadow what is to come in April? Surely the situation is being updated both at Faron and the FDA, even though no announcements have been delivered to investors.
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It would feel very peculiar if the April reading were a disappointment after this. However, one would think this would calm down the commotion caused by that lawsuit. You certainly wonât get bored with this stock.
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Here is an excerpt from the press release, translated by the translator:
"Key Highlights
- Orphan Drug Designation further strengthens the beksmarilimab program by providing clinical development and commercialization benefits
- Faron is on track to report front-line and HMA-failed (r/r) MDS top-line efficacy results in April 2025"
Juho has stated in interviews that due to the fast track, information and questions are constantly being fed to the FDA. Now, from my Faron perspective, it seems the results continue to be strong!
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With what data can any statuses be granted to the first line? Surely almost nothing is ready yet?
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Indeed, for Orphan designation, Phase 1 or 2 results are not required. Although it can be obtained with them too. Test tube experiments or animal studies and the mechanism of action are sufficient, along with the fact that the disease is rare and good treatments are not available. Why Orphan was granted for AML already in August 2023, but not for MDS, is unknown. Was it perhaps only sought for AML at that time? The Phase 1 efficacy results then, however, guided the focus to MDS.
The processing of a status application takes months, and the application is made using a specific form, so it is unlikely that the application would contain different information than we have. Rolling submission of data via Fast Track means that information for the drugâs marketing authorization can be submitted in parts, rather than as a single lump sum, as without FT, only when the entire package is assembled. The idea that new results would have influenced MDS Orphan is understandable, but the ASH results from December at the latest would have already supported it. If the FDA indeed wanted to see evidence of efficacy in MDS after the modest AML results. If and if.
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Does anyone know if someone who has received bex treatment has ever written here? It just starts to feel strange if there are going to be half a thousand recipients, and no one has even heard about it from anyone. Or do they have to sign a non-disclosure agreement?
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Let me throw a counter-question: what added value would it bring if such a person wrote here on an INVESTMENT forum? Do we suspect that those authority-monitored studies are scams, and there arenât really any tested individuals? Or would we get a confirmation of bexâs efficacy from an individual experiential expert that cannot be published in official research results?
I personally canât think of what added value someone who has personally experienced bex could bring to this forum. Instead, I suspect that these messages also reached thousands of eyes unnecessarily.
Letâs focus on whatâs essential? Although I thought this challenge from @Clark_kent was quite relevant, and it could have led to a good discussion:
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Well, in my opinion, it could even bring added value if patients themselves came to tell about their experiences with treatments, and what condition they are in currently. Itâs a crazy challenge, but I understand your frustration.
I remember from years ago Markku already said that the fruit was starting to be ripe; itâs probably already rotten after all these years of ripening 
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Hardly even two hundred patients have received bexmarilimab so far. Furthermore, the patients come from different countries, such as the United States and Finland.
But as someone already stated, patients certainly wonât come to an investment forum in the middle of a clinical trial to tell how theyâre doing. Oh dear, what a question.
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US attention:
https://www.onclive.com/view/fda-grants-orphan-drug-designation-to-bexmarilimab-for-myelodysplastic-syndromes
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Faron hired a Medical Director (for solid tumor projects?)
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Exactly.
And I strongly believe that patients would have come forward publicly to report if the things told, e.g., about side effects/or their absence, were false.
I suppose the test subjects also sign some agreement where it would likely be mentioned that it is the company and authorities who handle the communication.
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NĂ€köjÀÀn olisi parempi tehdĂ€ lihavuutta âhoitaviaâ kuin tappavia tauteja hoitavia lÀÀkkeitĂ€ 
âUnder the terms of the agreement, Zealand Pharma will receive upfront cash payments of USD 1.65 billion, including USD 1.4 billion due upon closing and USD 250 million over the first two anniversaries of the collaboration. Zealand Pharma is also eligible for development milestones of USD 1.2 billion primarily linked to initiation of Phase 3 trials with petrelintide monotherapy and sales-based milestones of USD 2.4 billion, for a total consideration to Zealand Pharma of up to USD 5.3 billion.â
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Apparently, Nightingale Health is aiming for the main list. Has this been discussed regarding Faron? Evidently, there could be advantages if international investors are sought.
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Hopefully not yet. Only when sales start bringing in money. In this sense, Nightingale is slightly ahead of Faron, and it might be a timely move for them.
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I started wondering, if Faronâs Bexmaril drugâs Phase 2 results are excellent, which scenario would be more likely: a partnership agreement or an acquisition offer made by a big pharmaceutical giant.
I considered that a partnership agreement might be more likely because large pharmaceutical companies often enter into partnership agreements when promising drugs are involved. In partnership agreements, they can leverage another companyâs research and development without full ownership. In such a case, Faron could receive funding and support for the drugâs continued development, while the large pharmaceutical company receives a share of the potential profits. The advantage of a partnership agreement is that it allows for the use of broader resources and larger marketing networks, without the company needing to completely give up its ownership.
An acquisition offer is also possible, of course, but I would assume it to be less likely than a partnership agreement. The drugâs ultimate market potential is not yet entirely clear, and even if the Phase 2 results are excellent, commercializing the drug still requires significant investments. Large pharmaceutical companies could be interested in making an acquisition offer, but in such a case, they would need to carefully assess the drugâs commercialization risk and potential.
If the results were truly promising and the drugâs market potential was high in their assessments, then an acquisition offer could emerge.
What do you think, would Faron be willing to sell itself?
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For the past five years, it has been discussed daily here that this will probably happen. The companyâs management and investors have discussed it.
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Certainly, if a sufficiently hefty offer comes to the table. Itâs unlikely, however, that such an offer would be received at this stage. Partnering has been mentioned repeatedly in Juhoâs speeches, and if the spring results are sufficient, that is the most likely scenario. Then towards fixed assets and thereby added value for the company, at which point a jackpot offer for the entire company might become relevant.
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Big pharma generally aims to minimize risk. In that case, a partnership agreement is, in my opinion, more likely at this stage. The timing of this partnership deal is not yet certain, and this too could be delayed further*. An acquisition offer is, in my opinion, more likely only at the stage when Bex receives marketing authorization for blood cancers and demonstrates efficacy in phase two trials for combination therapies of solid tumors.
\* Herantisâ CEO stated in the Q4 webcast that agreeing on these partnership deals takes about 4-6 months, so if there isnât already an agreement awaiting signature, finalizing the deal could well be delayed by another half a year from the next reading.
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Partnership deal. Faronâs strategy is to partner in blood cancer and, with that revenue stream, continue BEX Phase 2 in solid tumors independently.
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