Even overgenomen van Techspec, het raport Oppenheimer...
Oppenheimer heeft zijn koersdoel aangepast naar aanleiding van het uitstel door de FDA voor profylactisch waar om aanvullende data wordt gevraagd. Zie hier de inhoud van het op 20 september aangepaste rapport Oppenheimer;
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.