Same data, different conclusions. Well, this is how it is. Sometimes one wonders if the FDA is playing favorites. However, Pharming seems to be able to proceed properly in the drug approval processes of different countries.
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An interesting company that Pohjolan_Eka has commendably brought to light.
As a basis for an investment decision, I’m happy to start with a worst-case scenario. Here, I will initially only examine Ruconest and the share of Joenja that has received approval, what is already in hand or likely to come.
According to the information I found, Ruconest’s patent expires this year. The new drugs that have entered the market for the initial treatment of HAE attacks will inevitably reduce its sales. As CK has stated, it will continue to have a role in the arsenal of emergency polyclinics.
Will the expected increase in Joenja’s APDS sales be enough to compensate for Ruconest’s sales decline?
The number of APDS patients in potentially involved OECD countries is roughly 1000 people with a prevalence of 1/million. If Joenja treatment penetration is conservatively estimated at 50%, annual sales would be in the order of 200 MUSD. Joenja’s patent in the US expires in 2032, and an extension application has been submitted. In Japan, it expires in 2038, if I recall correctly. Uncertainties remain regarding obtaining approval, for example, from the EMA.
I will not dwell further on ongoing studies to expand Joenja’s indications for use. There may well be potential, but I am also somewhat skeptical about Joenja’s possibilities regarding somewhat vague immunodeficiency syndromes, which can be multi-etiological (cf. Diamyd and only a specific haplotype). Mitochondrial disease and its drug treatment are interesting, but much still needs to be confirmed.
According to this year’s estimate, revenue is in the order of 400 MUSD, of which research expenses are 100M, sales expenses and administration 220M. If we proceed with a bad scenario, and the new studies do not yield results and are terminated, and there is still enough left over from 300 MUSD in sales to justify the current valuation.
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Regarding Ruconest, it’s important to remember that it is a biologic. In the case of biosimilars, competition is not quite as aggressive because development is more expensive and the approval process is a bit more demanding. In such cases, there shouldn’t be any “dumping price” competitors entering the market.
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The prevalence of APDS without redefinition is estimated to be 1.5 per million people. In Japan, there are slightly over 7 million children in that age range, which would make the number of patients just over ten. However, since it is an autoimmune disease that negatively affects lifespan, one could expect a higher incidence in child cohorts (because some adults have already died), so we are probably talking about low tens. This number of patients will, of course, expand as more evidence of Joenja’s benefits and efficacy becomes available for VUS/PID/CVID cases. The greatest positive, in my opinion, was that the drug was approved at all, which gives hope for FDA approval for American children without expensive new additional studies.
This is actually the most interesting aspect of the company from an investor’s perspective. In a bad scenario, the valuation is justified, and in a good scenario, the company is flagrantly undervalued. I love these kinds of ‘bets’ where you can win a lot of money if the company succeeds, but also the risks for failure are limited, as safeguards quickly provide a supportive floor.
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Positive statement from the European EMA on Joenja:
Pharming Group N.V. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adult and pediatric patients 12 years of age and older.
- If approved, Joenja® (leniolisib) would become the first approved treatment in the European Union for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency
- Decision based on Phase II/III clinical data demonstrating statistically significant impact on measures of immune dysregulation and immunodeficiency
- Final European Commission decision expected in Q2 2026
Investors went wild over the positive news and the stock price deservedly rose… wait, it’s not even rising 
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There’s no data, but my gut feeling is that the final decision will 90% follow the board’s stance. Maybe this was already priced in 
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A press release is expected next week regarding the FDA’s requirements for what the approval of Joenja’s label expansion in the US will entail and the timeline Pharming intends to follow. Personally, I see a reasonable risk of negative news here, even though the data was accepted in Japan.
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A breakthrough here could reflect negatively on Pharming’s stock.
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Additionally, regarding Joenja, no news is bad news. The FDA has already held a hearing regarding the negative decision, and if only cosmetic changes were required, the next steps would surely have been announced by now. Due to the silence, I expect negative news in the coming weeks, detailing required additional studies and a delay in the drug’s pediatric expansion for the US market.
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So has it finally happened—has revenue growth ground to a halt? The guidance has remained unchanged for now:
At least Joenja is growing at a good pace, and it seems that the U.S. FDA application was successfully resubmitted without issues. The rascals didn’t even release a bulletin about it.
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A strong reaction in the stock market, a 25% drop today. I see it as a panic reaction. Joenja’s rising sales within 1-2 years and Ruconest’s even declining sales justify the current share price, even if this year’s and next year’s trials end up being a disappointment.
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