Pharming « Terug naar discussie overzicht

quote:

't zal maar gebeuren schreef op 20 september 2018 14:19:

plus 17% vandaag ,neemt niet weg dat mijn vertrouwen in een stabiele koers tot het nulpunt is gedaald na de laatste 3 dagen.

quote:

antop schreef op 20 september 2018 14:35:

lekker hoor ff heel druk geweest buitenland...nu gisterenavond gewoon hetzelfde vertrouwen in de Vries!Pharming is kleine speler met super product...vinden de grote niet leuk he!Maar wij hebben geduld en TRUST.

quote:

antop schreef op 20 september 2018 14:22:

er is een koers voorspelling boven de €1,00 ik denk dat dit zeker mogelijk is...als we door de 1,01 knallen we door..mag ook deze week zijn. Als iedereen weer TRUST heeft...blijven lekker zitten op deze spotgoedkope aandelen. zie het maar als een kans voor diegene die hoog instapte ze gehouden hebben is het een zeer mooi middel moment voor de koers geweest!

quote:

JaneJungle schreef op 20 september 2018 14:28:

Geweldig hoe alles zomaar omslaat. Geweldig dat koers weer terug is 0,90-1,00€. Kan naar 1,20€.
Wil niets zeggen kan ook makkelijk naar 0,65€

quote:

't zal maar gebeuren schreef op 20 september 2018 14:19:

plus 17% vandaag ,neemt niet weg dat mijn vertrouwen in een stabiele koers tot het nulpunt is gedaald na de laatste 3 dagen.

quote:

Beast98 schreef op 20 september 2018 14:39:

[...]
dan ben je niet de enige! Heeft niets met beleggen te maken dit aandeel, maar meer met een loterij. Ik wacht op een uitschieter naar boven, dan aftikken op 1,00 en nooit weer.

quote:

Maragon schreef op 20 september 2018 14:41:

[...]

degene die hoog instapte en ze gehouden heeft (niet hebben), staat nog steeds op dik verlies.

quote:

jurpsy schreef op 20 september 2018 14:47:

OPPENHEIMER.

Stock Rating:
OUTPERFORM
12-18 mo. Price Target €2.50
PHARM - NXT AM €0.97

Pharming Group N.V.
CRL Delays Prophylaxis Approval; Long-Term Story
Remains Intact
SUMMARY
Pharming (PHARM) announced the receipt of a Complete Response Letter (CRL)
regarding its sBLA for Ruconest use in prophylaxis for hereditary angioedema (HAE,
currently marketed in acute), ahead of the 9/21 PDUFA. The FDA requested a Phase
3 study be conducted before it will reevaluate Ruconest in prophylaxis; we expect
a 12-24-month delay. While this outcome is surprising, we believe today's selloff
(down ~20%) is overdone. There is little impact to medium-term sales in our model
(prophylaxis had constituted only 3% of 2021E revenues), and we believe the longterm
story remains intact. We push out prophylaxis sales and conservatively lower
acute sales estimates as Shire's Takhzyro (lanadelumab) launch and Cinryze supply
stabilization could slow Ruconest penetration. We lower our PT to €2.50 (from €3.00).
KEY POINTS
¦ The sBLA was filed on the basis of two Phase 2 studies in prophylaxis (one open
label and one randomized, double-blind, placebo-controlled, total n=56) and an
end-of-Phase 2 meeting. Given the supportive feedback from the FDA, a highprofile
Lancet publication and the strength of the Phase 2 data, both the company
and the Street had expected an approval (80% probability).

¦ We await further clarity once PHARM has discussed the CRL and potential
resolutions with the FDA. One scenario would be running the Phase 3 study
with next-generation formulations of Ruconest (subQ, intradermal) already in
development rather than taking the current IV formulation forward. We expect a
Phase 3 study of that nature could initiate mid-/2H19 (12- or 26-week duration).

¦ With prophylaxis approval delayed, investors will be focused on the sales
trajectory for Ruconest and quarterly execution in 2H18-19. We reduce our 2019E
and onward prophylaxis numbers and expect very minor off-label usage ($1M to
$5M per year). With a clean lanadelumab label and stabilized Cinryze production
constraints on plasma-derived C1C1 esterase inhibitor, we reduce our 2019Eonward
acute Ruconest sales estimates.

¦ PHARM's pipeline for Ruconest in indications outside of HAE is also increasingly
important. The Phase 2 IST for cardiac-induced nephropathy (CIN) will report data
in 4Q18 (go/no-go for company-sponsored Phase 2 in 1H19), and a Phase 1/2
study for pre-eclampsia could initiate as soon as YE18. Finally, a Phase 2 study
in cardiac protection is planned for 2H19.

¦ We expect continued negativity and pressure on the shares as noise around
competition, a strong Takhyzro label/launch, and the prophylaxis disappointment
flush out. However, long-term investors should note the impressive Ruconest US
sales growth in acute treatment, multitude of pipeline catalysts, and prudent OPEX
spend (potentially break-even in 2018). We stay bullish.

quote:

jurpsy schreef op 20 september 2018 14:43:

STIFEL.

BUY
TARGET PRICE CHANGE

September 20, 2018 EU Pharmaceuticals
Pharming Group N.V.
PHARM – AEX; PHAR.AS

No prophylaxis approval but sell-off overdone
Summary
Pharming has received a complete response letter (CRL) from the FDA to expand the use
of Ruconest for the prophylaxis treatment of HAE, beyond the existing use in the acute
setting. We are surprised by the FDA's decision and request for a further clinical trial,
given the relative strength of the clinical data compared to Cinryze as one of the approved
drugs for prophylaxis use. We can only speculate that the more recent approvals for
Haegarda and Takhzyro have moved the FDA's goalposts on what data are required.
While approval for Ruconest in the prophylaxis setting was not a game-changer given
the focus on Ruconest as the best option for acute, rational, on demand treatment, we
did expect a boost to sales growth in 2019 from renewed sales force promotion and have
therefore pushed back our sales contribution from prophylaxis use to 2021. This lowers
our 2022 sales forecast from $425m to $350m, and therefore our TP is reduced to €1.45.
We believe the recent sell-off has been overdone and therefore reiterate our Buy rating.

Key Points

Surprising FDA rejection, Ruconest clearly works and is safe. Pharming has
previously reported, presented and published (in The Lancet) highly positive Phase II
clinical data with the use of Ruconest for the prophylaxis treatment of HAE. A onceweekly
and twice-weekly IV regimen resulted in significant reductions in HAE attacks
vs placebo, with what we also regard was a greater response rate (>50% reduction in
attacks) compared to Cinryze. The data demonstrate the product clearly works in the
prophylaxis setting, has been on the market since 2014 as an acute treatment, has an
established/acceptable safety profile, and anecdotally has been used as a prophylaxis
option, particularly during the recent shortages of Cinryze. The FDA's decision to ask for
another clinical trial, just to confirm a treatment response already observed, is bizarre in
our view. Our only conclusion is that the FDA has taken a wider market view in light of
approvals for Haegarda and Takhzyro as new prophylaxis treatments and sees less need
to sanction an accelerated approval (i.e. on Phase II data).

Options for next steps. We understand that Pharming needs to have a follow-up meeting
with the FDA to understand what clinical data the regulator requires in order to seek
prophylaxis approval. We see little merit in proceeding with the current IV regimen for the
next clinical trial, instead waiting for more convenient formulations (SC, IM, transdermal
patch), for a more appropriate offering in the prophylaxis setting. We therefore have
pushed back potential sales contribution from use in the prophylaxis setting until 2021,
which lowers our 2022 sales estimate to $350m from $425m.

Even with shifting dynamics, significant acute opportunity remains. We do expect
the overall HAE market to shift to more convenient prophylaxis products, such as
Haegarda (twice-weekly, SC), Takhzyro (once every 2 weeks, SC) and potentially daily
oral tablets (e.g. BioCryst's BCX7353 in Phase III), with a 40:60 proph:acute ratio today
to a 75:25 split in favour of proph by 2022. However, that still leaves a significant market
opportunity, we estimate at around $700m, and assuming Ruconest becomes available in
more convenient formulations, we believe Ruconest will have the best profile in the acute
setting, as a standard regimen but also to treat breakthrough attacks which will inevitably
occur in patients on prophylactic options. A head-to-head trial of Ruconest vs Firazyr due
by the end of 2018 will also be important given the likely genericisation of Firazyr by July
2019. Ultimately, we also expect the likes of Takyzyro to help to expand the market for
HAE treatment, as Shire anticipates, rather than displacing existing treatments, i.e. more
market expansion than market rotation.

Sell-off overdone. At the core of Pharming is a validated and differentiated technology
platform that has so far delivered Ruconest and has the potential to deliver further
candidates, such as for Pompe disease, due to enter the clinic by year-end. In our view
the recent sell-off presents an attractive buying opportunity given overblown concerns
over the headwinds to Ruconest.

quote:

Super trio schreef op 20 september 2018 14:42:

[...]vertrouwen komt te voet en gaat te paard. Denk dat het nu wel goed zit met het vertrouwen. Hoop dat de directie van Pharming hun lesje hebben geleerd er moet meer en beter worden gecommuniceerd met de aandeelhouders.