Ionis absorbs Glaxo rare disease cull

From Ionis Pharmaceuticals’s perspective, Glaxosmithkline’s decision to opt out of its collaboration on inotersen and Ionis-FB-LRX is not the worst outcome that could befall it. After all, Glaxo had paid $35m seven years ago to secure rights for up to six Ionis antisense assets, and along the way kicked in more than $50m in collaboration revenue for a clinical programme that required fewer than 200 patients to be filing ready in TTR amyloidosis.

True, it could have used Glaxo’s commercial know-how to launch inotersen should it get approval from the US FDA and the EU, but the California-based group says it is now pursuing partners to commercialise the inotersen, following the model it used in creating a marketing subsidiary called Akcea Therapeutics.

Change in focus

As with the Akcea asset volanesorsen, inotersen treats a rare disease and as such would not require a huge sales and marketing force to reach specialists.

Leerink analyst Paul Matteis also cautioned that Glaxo’s withdrawal could stimulate increased concern over inotersen’s safety profile, especially a well-documented thrombocytopenia side effect. Meanwhile, Stifel analyst Stephen Willey declared it a “value creating event”, suggesting that the group would be able to find a new partner easily – he cited Pfizer because of its own amyloidosis products.

Mid- and late-stage Ionis collaborations
Project Licensee Forecast collaboration revenue, 2022
Marketed Spinraza Biogen 394
Kynamro Kastle Therapeutics
Phase III Alicaforsen Atlantic Pharmaceuticals -
Plazomicin Achaogen -
IONIS-FXIRx Bayer 39
Phase II IONIS-HTTRx Roche (option) 16
BIIB067 Biogen (option) 3
IONIS-STAT3-2.5Rx AstraZeneca 2
AKCEA-APO(a)-LRx Novartis -
IONIS-HBV-LRx GlaxoSmithKline (option) -
IONIS-HBVRx GlaxoSmithKline (option) -
Source: EvaluatePharma

As for Ionis-FB-LRX, a focus on dry age-related macular degeneration might require a bigger commercial partner, but as an early phase II candidate this is less of an immediate concern.

Ionis shares dipped more than 4% in the pre-market following the announcement, but now are trading less than 1% below Thursday’s close. Glaxo is maintaining collaborations on the hepatitis B projects Ionis-HBVRx and Ionis-HBV-LRx.

Who's next

Glaxo recently announced that it was trimming its own in-house pipeline, so termination of outside collaborations is logical (Glaxo gets out the axe, July 26, 2017).

In announcing second-quarter results, Glaxo's chief executive, Emma Walmsley, had said the UK big pharma had “completed a strategic review of our rare disease business and for the assets currently in our pipeline intend to secure opportunities to further development and generation of financial returns outside of GSK to give them the very best possible chance of successful clinical development”.

Other rare disease assets that could be subject to externalization include an AL amyloidosis asset called GSK2398852 and the CD34-positive stem cell therapy GSK2696275 for Wiskott-Aldrich syndrome. Another antisense collaboration, with Idera Pharmaceuticals, could also be in the spotlight – this particular partnership focused on renal diseases – not to mention work being done with Oxford Biomedica’s Lentivector platform in rare diseases.

Ionis might not take too much damage, but other partners without late-phase or commercial-stage assets may not be so lucky if Glaxo keeps looking at opportunities to trim its pipeline.

To contact the writer of this story email Jonathan Gardner in Virginia at or follow @ByJonGardner on Twitter

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