Why Spinraza Is The Only Exciting Thing About Ionis Pharmaceuticals

The U.S. Food and Drug Administration approved in late December Ionis Pharmaceuticals Inc's IONS therapy for the treatment of spinal muscular atrophy called Spinraza.

In a research report on Friday, Jefferies' Eun Yang suggested that Spinraza is Ionis' only exciting product in its pipeline. Accordingly, the analyst maintained an Underperform rating on Ionis' stock with a $17 price target.

Here is what the analyst has to say on Ionis' other products in its pipeline.

Volanesorsen

Volanesorsen is an antisense drug in development for two rare metabolic disorders: FCS and FPL.

The analyst sees limited market potential for the therapy at $130 million which is similar to Juxtapid with approximately $79 million in the first nine months of 2016.

GCGR-Rx

IONIS-GCGRRx is an antisense drug designed to reduce the production of glucagon receptors, or GCGR, to treat patients with type 2 diabetes

The analyst questioned what therapeutics merits the therapy offers patients in the already crowded market.

FXI-Rx

FXIRx is an antisense drug designed to reduce the production of Factor XI. Factor XI is a clotting factor produced in the liver that's an important component of the coagulation pathway.

The analyst noted that recent studies found that the therapy showed no clinically meaningful reduction in platelets nor any reported tx-SAEs

DMPK-2.5Rx

IONIS-DMPK-2.5Rx is an antisense drug designed to reduce the toxic dystrophia myotonica-protein kinase, or DMPK, RNA to treat patients with Myotonic Dystrophy Type 1 (DM1).

The analyst pointed out that the program has been discontinued due to inadequate potency in muscle tissue. Ionis and its partner Biogen Inc BIIB will now develop a muscle-targeting LICA alternative.