ARWR continues to make rapid, nearly error- and delay-free, progression of their pipeline and has done
so without significant dilution to shareholders. Recently secured orphan designations and positive (safety
and activity, including duration) initial human trial data of APOC3 and ANG3 sets the stage for a
potentially accelerated and an unambiguous regulatory pathway for both candidates. Orphan status, combined with the recently announced positive topline Ph1 data, sets the stage for what could be further
acceleration of these programs through mid/late-stage development. In fact, additional clinical data, announced in early February 2020, provides further support of the potential effectiveness and safety and
should bolster the eventuality of beginning pivotal U.S. studies for both candidates.
The Janssen/J&J collaboration has already shown to be a win for the company (and shareholders), providing over $200M of dilutive-free funds in less than 12 months and facilitating the speedy
development pace of ARO-HBV (JNJ-3989). We think it is reasonable to assume that the clean safety
(to-date) profile and JNJ-3989 having already moved into a large, potentially curatively-powered Ph2b
(triple combo) study, is bolstering Janssen s confidence in both the potential of the therapy (and,
perhaps, the TRiM platform as a whole) and their working relationship with ARWR. Having now pulled
the trigger on the first option to collaborate with ARWR on (up to three) additional RNAi therapeutics is,
we think, another indication of the potential upside that Janssen sees in ARWR s technology and
capabilities.
ARO-AAT also continues to make rapid development progress and Fast Track status, granted in June
2019, could help accelerate that pace. Encouragingly, SEQUOIA, a Ph2/3 trial which was just
announced earlier this year and could serve as a pivotal U.S. registrational study (which would make it
the first U.S. pivotal study of ARWR s TRiM platform), appears to be moving quickly. IDE approval came
in April 2019, multiple sites continue to enroll and are dosing patients and new sites are expected to
come online. ARWR appears intent on leveraging the Fast Track status, noting that they have sped up
clinical site activity including getting additional locations operational and enrolling. U.S. sites were the
first to be operational and ARWR has since opened additional sites in both North America and Europe.
With APOC3, ANG3 and AAT all possibly entering pivotal studies in the near-term and JNJ-3989 now in
what could prove to be a curative study (for HBV, which could draw massive appeal), we could soon
have a lot more information to gauge the potential commercializability (and value) of Arrowhead s
development portfolio. This, by extension, also bolsters the potential likelihood of significant value inflection events/announcements (over the same timeframes).
We also remain highly encouraged by ARWR s seeming determination to continue to deepen their
development pipeline. With another three CTA filings anticipated, the company could soon have eight
TRiM clinical programs ongoing. ARO-HSD, targeting HSD17B13 for potential indications in alcohol and
non-alcohol related liver diseases, is the most recent addition and which ARWR hopes to have in clinical
studies next year.
The rapid and largely error and delay-free progress of the existing clinical pipeline, coupled with the
company continuing to deliver on its guidance related to increasing the number of high-potential TRiM
candidates under development, has moved our targeted market capitalization to $5.9B, or ~$58/share.