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Pharming Augustus 2019

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dive till the bone schreef op 14 aug 2019 om 16:58:

is dit een strohalm voor Pharming om in zee te gaan met Novartis , zien ze al hun eigen trails de mist in gaan ,en pakken ze deze met bijde handen aan om de toko te redden,wie zal het zeggen!!!!

Dit vind ik nou een ongenuanceerde opmerking. Voor wat betreft AKI/CIN is al bewezen dat het werkt. Vandaar binnenkort ook de start van fase 2B onderzoek.

Ik ben blij met je als korte termijn TA-specialist... maar ben het qua meningen over de business niet vaak met je eens.
lucas D

BassieNL schreef op 14 aug 2019 om 16:40:

@lucas... er is tijdens de call meer verteld dan in het persbericht staat!

Daar heb je geen houvast aan, er moet dan ook iemand van Novartis aanwezig geweest zijn.
Dan nog is het niet door alle aandeelhouders controleerbaar. Of er moet een opname van zijn, en de opname zal geplaatst moeten worden op de Pharming site.

Karel slimpie schreef op 14 aug 2019 om 16:44:


Je heb het dus niet begrepen wat er bedoeld werd.

Daar heb ik het toch niet over, bij deze RODE beurzen USA nu MIN 554, daalt ook Pharming, of dacht jij dat we UP zouden gaan... En als het tegenzit, dalen we nog meer...

Financiële markten slaan alarm, recessie nadert....
Karel slimpie

Eurowin schreef op 14 aug 2019 om 17:04:


Daar heb ik het toch niet over, bij deze RODE beurzen USA nu MIN 554, daalt ook Pharming, of dacht jij dat we UP zouden gaan... En als het tegenzit, dalen we nog meer...

Financiële markten slaan alarm, recessie nadert....

Nee, ik doelde op die azijnzeikers

Karel slimpie schreef op 14 aug 2019 om 17:07:


Nee, ik doelde op die azijnzeikers

Ja, oké daar heb je gelijk in, is niets mis met Pharming, alleen je treft nu een uiermatige slechte beurs, alles daalt..Gr
dive till the bone
Zo zie je maar weer wat voorkennis allemaal doet met piet particulier, en waarom LT gaan als je nog geen week vooruit kan kijken.
Pharming: Creating New Generation Plasma-Free Medicines For Unmet Medical Needs
Aug. 14, 2019 10:41 AM ET | About: Pharming Group N.V. (PHGUF)

Pharming's Q2/19 results beat expectations for a second quarter in a row. Turnover is confirming that earlier perceived advantages for competition in the HAE marketplace were misguided.

Huge expansion potential for rhC1INH beyond HAE around the Ruconest franchise through its pipeline opportunities with pre-eclampsia, acute kidney injury, and ex-Ruconest investments in Pompe and Fabry diseases.

Since the successful buyback of US distribution rights from Valeant, Pharming has built a highly sophisticated rare disease sales organization across the US.

The company is now poised to add new products to its portfolio through acquisitions and license agreements, which would boost its revenues significantly.

Manufacturing investments are underway to meet the bullish outlook. A second production facility is awaiting regulatory validation in Q1/20 and will more than double existing capacity.

Editor's note: Seeking Alpha is proud to welcome The Moneytree as a new contributor. It's easy to become a Seeking Alpha contributor and earn money for your best investment ideas. Active contributors also get free access to SA Essential. Click here to find out more »

Pharming (OTCPK:PHGUF) - is a Dutch, Leiden-based company listed on Euronext Amsterdam under the ticker 'PHARM':

Pharming is specialty pharmaceutical company developing innovative products for the safe, effective treatment of rare diseases and unmet medical needs. Pharming’s lead product, Ruconest ® is a recombinant human C1 esterase inhibitor approved for the treatment of acute HAE attacks in patients in Europe, the US, Israel, Colombia and South Korea. The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.

Pharming’s technology platform includes a unique, GMP-compliant, validated process for the production of pure recombinant human proteins that has proven capable of producing industrial quantities of high quality recombinant human proteins in a more economical and less immunogenetic way compared with current cell-line or plasma fractionation-based methods.

Source: Pharming Group N.V.; Website

The backdrop
Pharming has been around for some time, and experienced quite a bumpy ride during its long existence. Back in the 90s, the company made worldwide news with the breeding of Herman the Bull, the very first transgenic bovine. Facing public scrutiny over its then novel and controversial genetic engineering, it wasn't until 2010 and a few near bankruptcies later that its first commercial product Ruconest for acute HAE attacks was approved by the European Medicine Agency (EMA). In July 2014, the FDA also approved the medicine.

Today, Pharming uses rabbits as a source to produce Recombinant human proteins. Produced in the milk of transgenic rabbits it offers several advantages over traditional methods of harvesting human proteins from human blood.

Recombinant proteins carry a much lower risk of disease transmission due to their method of production than plasma-derived proteins do. In addition, as the expression goes ‘breeding like rabbits’, the production capacity is sheer limitless!

The Opportunity
Today, Pharming is generating positive cash flow, forecasting increased quarterly revenues and finds itself in a position where it can finance - and has already started to develop - new medicines for unmet medical needs.

Pharming's first choice of medicine to develop was a so-called "orphan drug," due to the pricing model for such medicines: Extremely high-priced, compensating for the fact that a rare disease affects a very small percentage of the world's population. However, these same Human recombinant proteins can also be used to treat other diseases that result from a patient's own inability to naturally produce some essential proteins. Diseases with much broader patient populations that currently have unmet medical needs. A recipe for blockbuster market potential!

HAE Market Dynamics
The HAE marketplace is very dynamic, and Pharming faced a setback in 2018, receiving an FDA response letter, requiring additional clinical data for approval of their leading product Ruconest in the application of prophylaxis treatment of HAE patients.

Shortly thereafter, Pharming’s biggest competitor Takeda (NYSE:TAK) (formerly Shire), market leader in the production of medicines for HAE, got FDA approval for a new, non-plasma based prophylaxis HAE medicine branded Takhyzro (formerly called Lanamedub). This medicine is produced in Chinese hamster ovary (CHO) cells by recombinant DNA. A smart move, for its other, plasma-based HAE prophylaxis medicine called Cinryze, is becoming increasingly challenging and expensive to produce. As all this was happening, another competitor, BioCryst (BCRX) is developing a promising oral drug, also for prophylaxis treatment with the prospect of offering a more convenient way of drug-administration to HAE patients.

Many voices could be heard saying that the introduction of Takhyzro and BioCryst's new pill would mean the end of a significant market for acute HAE treatments, and that Pharming's business case for its acute HAE medicine would collapse after losing the prospect of entering the prophylactic market on a short term.

Now, one year on, the surfacing data on these competitor’s medicines are painting a very different picture.

Takeda’s new medicine Takhyzro, although quite well received, is not as effective as the company projected, and Ruconest is now profiting from a higher than expected breakthrough rate of HAE attacks of patients who are on its new prophylaxis treatment. This is opening up a whole new market for Pharming’s medicine, as the go-to medicine for HAE breakthrough attacks; an opportunity that was not anticipated. Reportedly, HAE breakthrough attacks may reach as high as 50% to 60%!

And as for the oral drug developed by BioCryst, the Phase III clinical trial data endpoints released earlier this year are so poor, that the company lost more than half of its market value overnight (from $7.39 down to $3.49 on publication date 21st May '19). Although effective to a certain extent, the question remains if the FDA will approve the drug in an environment where much better working medicines are already available. And in the event it would be approved, reality shows that the introduction of such a weak prophylaxis treatment could actually increase Pharming's sales potential for breakthrough attacks!

As patients and physicians appear to increasingly seek out Ruconest as the most effective acute treatment for HAE attacks, there is a consistent growth in the number of patients in the US. A recently published independent study in Germany shows that Pharming’s medicine Ruconest is consistently best in class. This while the market may have expected it to go downhill after the introduction of the prophylactic agent of Takeda.

In sharp contrast to those expectations, Takeda's medicine for acute HAE attacks - Firazyr - (Ruconest's main competition) has suffered a major decline of more than 60% (page 9 of Takeda Q1/19 report published on 31st of July) over the last couple of months.
Deel 2:

Generating Cash
The trend break in the company's struggles happened when Ruconest was approved, but took flight when Pharming succeeded in buying back US distribution rights from Valeant at the end of 2016.

In the 2018 annual report, published on March 28, we can find that Pharming achieved a net profit of €25.0 million in 2018, which corresponded to a net margin of 19 percent. (In 2017, Pharming suffered a loss of €76 million). Turnover from product sales rose to €134.3 million, which was 51 percent more than the €88.7 million from a year earlier.

On July 25, the company reported on the first 6 months of 2019. Now well into 2019, Ruconest sales exceed expectations once more. Total Ruconest revenues of €42.5 million in Q2/19 were again significantly higher than analyst expectations (consensus estimates were app. €38.8 million). The second quarter’s turnover beating Q1/19 by more than 20%!

Another noteworthy fact is that the Q2/19 gross margin at 87% was higher than for any quarter since 2014.

Financial highlights:

The Company made record revenues in the first half year, an increase of 31% to €77.9 million, from €59.5 million for the same period last year

US net product sales increased 33% year-on-year to €75.0 million (H1 2018: €56.3 million), and 21% quarter on quarter to €40.9 million from €33.7 million in the first quarter of 2019, reflecting strong growth despite a more competitive marketplace.

Net profit increased by 60% to €13.6 million, compared to €8.5 million for H1 2018

Positive cash-flows during the quarter were driven by strong revenue, generating almost €10 million above the cash required for operating costs

The equity position improved from €61.8 million at the end of December 2018 to €77.5 million at the end of the first half of 2019

Other financial liabilities, which refers to the contingent consideration for the milestones, reflects the payment of the first successful sales performance milestone in March 2019 and the revised probability and timing for paying the last milestone. The next milestone will appear in current liabilities once it is certain of being incurred, which is expected to happen later this year.

Source, Pharming Group N.V; Pharming Q2/19 report

While a range of other products are approved for the treatment of acute and prophylaxis HAE, and others are trying to get into the market, Pharming’s challenge to compete with the large pharmaceutical companies in the US is still very real. In spite of more competition, the increase in Pharming's sales is driven by strong US performance, and is confirming the data that Pharming is gaining market share.

Source Pharming Group N.V : Online webinar H1/19 (in Dutch)

Above numbers tell their own story, independent of market opinions and I believe the unlimited production capabilities for Ruconest, coupled with a clean safety profile and increasing diagnosis rates, will enable the product to achieve more than 300 million euros in 2020 sales.

Pharming is also working on new forms of user-friendly Ruconest administration for HAE, aiming to enter the HAE prophylaxis market with a new competitive edge. As a response to the FDA's request, a Phase III study with this new application form is expected to start sometime during 2020/2021.

Market potential beyond the HAE market
In 2018, two significant steps forward in clinical development for additional indications for rhC1INH (including Ruconest) outside of HAE were made.

The first was the positive outcome with clinically and statistically significant results from a proof of concept study of Ruconest in contrast-induced nephropathy. Based on this study, A Phase I/II Trial of Ruconest with acute kidney injury patients undergoing percutaneous coronary intervention (PCI) accompanied by contrast-enhanced examinations is set to begin later this year:

PCI is a part of the procedure used to treat narrowed coronary arteries in coronary heart disease. A catheter is used to visualize the blood vessels with x-ray imaging. After this, a coronary angioplasty can be performed in which a deflated balloon is moved into the obstructed artery and inflated to relieve the narrowing. Devices such as stents are then used to keep the artery open. These procedures both require higher volumes of contrast medium and are more likely to lead to thromboembolic events which trigger the complement system and culminate in reperfusion injury to the kidney (tissue damage caused when blood supply returns to tissue after a period of ischemia or lack of oxygen).

Here, the target U.S. patient population at ca. 0.5 million is many times the size of the HAE patient population!

Source Pharming Group N.V; Press release Oct.7, 2018

In 2018, there was another positive conclusion of a full clinical program for investigation of Ruconest as a treatment for early-stage pre-eclampsia. Following up on that study, Pharming received approval of the Dutch investigating center's ethics committee.

June 2019, Pharming announces it is commencing a clinical study of the effects of its recombinant human C1 esterase inhibitor (rhC1INH), RUCONEST, in patients with pre-eclampsia:

Pre-eclampsia (PE) is a life-threatening multisystem condition in pregnancies leading to increased maternal and neonatal mortality and morbidity. Fifty thousand maternal deaths during pregnancy per year around the world are recorded for patients who proceed to eclampsia, while many more are caused by long-term irreversible damage to organs caused by PE. Treatments include termination of pregnancy or premature birth. Even if born safely, over half of these newborns will suffer from growth restrictions, learning difficulties or moderate to severe disabilities.

Premature delivery is presently the only solution for PE, and there are no approved therapies. This is not a viable option for early PE (from week 20 of pregnancy). The main goal of therapy is therefore to prolong a safe pregnancy for PE patients as long as possible to improve the chances of a good outcome from delivery.

Almost 2.5 million cases of PE are reported annually, with rates running at between 1% and 17% of all pregnancies in various communities and countries,

These percentages present another huge market potential!

Source Pharming Group N.V; Press release June 11, 2019

The pipeline candidates for pre-eclampsia and acute kidney injury are expected to produce readouts in 2020.

Over the past year, Pharming has continued to make progress on their human recombinant ?-glucosidase for the treatment of Pompe disease, for which manufacturing is being up-scaled to provide material for studies and to produce clinical trial material. Another indication that is currently being pursued is Fabry disease.

Additional studies, although not initiated by Pharming itself are underway:

Tissue Damage after Hypoxic Event: Delayed Graft Function (new Investigator initiated study)
Shock response after trauma: Hypovolemic Shock – ongoing preclinical research projects with the US Army and US Air Force
Deel 3:

Source Pharming Group N.V: Investor Presentation - June 21, 2018

The above-mentioned indications are actively developed at the time of writing, but beyond these many more are there to be pursued in the future. Many diseases have to do with the complement system, a major part of our immune system, responsible for certain immune-mediated inflammation reactions.

Read more about the complement system and Pharming's Pipeline on their website

Source Pharming Group N.V: Investor Presentation - June 21, 2018

Securing the company's future
Investments in manufacturing are underway to meet the bullish outlook. A new production facility is awaiting regulatory validation in 1Q20. This facility more than doubles existing capacity, while a larger unit (adding 60% capacity) will be under construction soon. The facilities meet not only growing HAE demand but also pipeline expansion.

Pharming has implemented a Business Development unit and is actively seeking to add new products to its rare-disease portfolio through acquisitions and license agreements, which would significantly boost its revenues. Since the successful buyback of US distribution rights from Valeant, Pharming has built a sophisticated rare disease sales organization across the US, reportedly employing some of the best sales people in the HAE market space. A very promising setup to bring such newly acquired products or distribution rights to market. News on new acquisitions or deals are expected to be announced during the remainder of this year.

On August 13th, 2019 Pharming announces the acquisition of an exclusive license to CDZ173, a late stage drug for the treatment of APDS:

Pharming Group N.V. announces it has entered into a development collaboration and license agreement with Novartis to develop and commercialize CDZ173, a small molecule phosphoinositide 3-kinase delta (PI3K?) inhibitor being developed by Novartis to treat patients with Activated Phosphoinositide 3-kinase Delta Syndrome (“APDS”)

Source Pharming Group N.V: Press release Aug.13,2019

The study is currently recruiting for a Phase II/III study that can be submitted to the FDA and EMA for registration immediately upon completion.

The drug named Leniolisib, offers Pharming an excellent opportunity to better utilize its sales channel with a similar market potential as Ruconest. APDS occurs in 1 or 2 people out of a million. The most important market is the US, with a patient population of around 300 to 600 people. The condition was discovered in 2013 and as diagnosis may improve over time, this number may grow like it does with HAE.

Looking forward
Ruconest appearing to gain market share as the go to acute treatment option, while Takeda and CSL Behring (OTCPK:CSLLY) continue to battle over the prophylactic segment of the HAE market.

Pharming expects continued revenue growth from sales of Ruconest for 2019, especially in the US and Western Europe, with better results in each quarter, compared to the comparable quarter a year earlier.

The ongoing Business Development activities are set to deliver more new products in the near future. Products that can be brought to market through its specialized sales organization, contributing to the company's revenues.

Because of previously described negative outlook on the company's future for their HAE medicine, in September 2018, stock prices fell sharply from its earlier highs. The happening changes in landscape on the HAE playing field that are now working to Pharming's advantage are not yet reflected in its current stock price. As a result, today I see a short-term opportunity to benefit from a sharp increase of the stock value in pursuit of its earlier highs.

Due to its accelerating and expanding platform, sales growth momentum, and an execution-focused management team, I believe Pharming is a very attractive investment opportunity with a potential to more than double its stock price over the mid-term.

Considering the blockbuster potentials in its pipeline, the company may well be marked as a takeover candidate once clinical data starts producing results, in which case the stock price may soar to unforeseen heights, as is no exception in the Biotech/Pharmaceutical industry. This would also be expected to happen, maybe even more so, when the company is allowed to remain independent and start to develop medicines for more 'mainstream' diseases.

The main risks to Pharming's prospects of successfully developing new indications include slower sales growth for Ruconest in the EU and the US.

2019/2020: Quarterly updates on continued US/EU Ruconest sales growth
2019/2020: Plans for Phase III prophylaxis study in HAE
2019/2020: New deals or acquisitions in the rare-disease market-space
2019/2020: Investigation of new Ruconest formulations/delivery tech
2H19: Initiate studies in acute kidney injury
2H19: Update on pre-eclampsia phase I/II safety study
2H20: Initiate Phase I for alpha-galactosidase in Pompe disease
3Q20: Initial Phase I/II data in pre-eclampsia
2020/2021 Phase III study on new forms of Ruconest administration for HAE prophylaxis
Disclosure: I am/we are long PHGUF. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

voda schreef op 14 aug 2019 om 16:40:

Wat denk je van de afwijzing van profylaxe?

Profylaxe is NIET afgewezen/afgekeurd of welke afkeurende benaming dan ook.

Er is aanvullende data nodig, dat is toch echt wat anders.

PS: Voor je het jezelf weer af vraagt zoals gisteren, nee mijn positie is nog niet veranderd. De turbo's short doen het uitmuntend.
Karel slimpie
[Modbreak IEX: Gelieve op uw taalgebruik te letten, bericht is bij dezen verwijderd.]
dive till the bone
heb vanmiddag gezegd dat ik hem weer terugkoop rond 1,10€ ,maar ik zet hem nog wat lager, of heb jij een beter idee ? Karel
1 2 3

dive till the bone schreef op 14 aug 2019 om 16:51:

Moeten ze niet nog meer blunders maken 1 2 3 anders hou je het niet vol

dat is waar, maar geldt dat niet alle beurgenoteerde bedrijven, denk eens aan solide Albert Heijn die toen der tijd door de afdeling Amerika opgelicht werd door de direktie en wat te denken van Imtech …...en zo kan ik nog wel even doorgaan, laat de Vries maar schuiven … zwarte cijfers ...

weveka schreef op 14 aug 2019 om 17:24:

moet dat nou zo hard omlaag ?

Kijk eens naar de DOW en alle Europese indexen, alles daalt als een raket en dat neemt alles mee...

De Monitor schreef op 14 aug 2019 om 17:18:


PS: Voor je het jezelf weer af vraagt zoals gisteren, nee mijn positie is nog niet veranderd. De turbo's short doen het uitmuntend.

Net als die gasten die bij roulette alles op rood zetten. 100x valt zwart en uiteindelijk komt er heus wel een keer rood. ''kijk zie je nou'' zeggen ze dan. ''ik zei toch dat rood een keer zou komen''
Dat ze een hoop verloren/misgelopen hebben laten ze maar even achterwege.
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