Watch shares of Alnylam Pharmaceuticals

Alnylam presents Phase 3 results from ILLUMINATE-A study of Lumasiran

Alnylam Pharmaceuticals (ALNY) announced Phase 3 results from the ILLUMINATE-A study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – in development for the treatment of primary hyperoxaluria type 1.

The clinical data were presented at a late-breaking session at the European Renal Association-European Dialysis and Transplant Association International Congress being held as a virtual event on June 6-9.

Lumasiran achieved the ILLUMINATE-A primary endpoint with a 53.5% mean reduction in urinary oxalate relative to placebo and showed a 65.4% mean reduction in urinary oxalate relative to baseline.

All tested study secondary endpoints were met, including the proportion of patients achieving near-normalization or normalization of urinary oxalate, compared with zero percent in the placebo group.

Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events and with mild injection site reactions as the most common drug-related AE.

Primary hyperoxaluria (PH) constitutes a group of rare inherited disorders of the liver characterized by the overproduction of oxalate, an end-product of metabolism. High levels of oxalate are toxic because oxalate cannot be broken down by the human body and accumulates in the kidneys.

PH1 is an ultra-rare orphan disease caused by excessive oxalate production, and elevated urinary oxalate levels are associated with progression to end-stage kidney disease and other systemic complications.

Based on the ILLUMINATE-A results, Alnylam filed a New Drug Application with the U.S. Food and Drug Administration.

The FDA has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act.

In addition, the Marketing Authorisation Application for lumasiran has been submitted to and validated by the European Medicines Agency, and has received Accelerated Assessment designation.


Stifel analyst Paul Matteis believes full Alnylam’s Lumasiran data presented at ERA-EDTA reflect a relatively derisked clinical profile that’s very likely to attain regulatory approval.

Lumasiran treatment led to robust and durable reductions in urinary oxalate, which based on the well described biology in PH1 should ultimately lead to clinical benefit on renal outcomes, he contends.

The analyst notes that for the stock, lumasiran is already mostly priced-in here, though there’s some investment debate surrounding the market opportunity which is generally perceived to be ultra-orphan/small.

While he is also somewhat conservative in how he models revenues for lumasiran, there could be upside to numbers should approval/launch increase the PH1 diagnosis rate. Matteis has a Buy rating and a $159 price target on the shares.

ALNY closed at $127.59.


To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.

One Reply to “Watch shares of Alnylam Pharmaceuticals”

  1. it is understandable that LUMASIRAN decreases the oxalate production. What will happen to the already deposited oxalate ? if a patient has already renal failure with systemic oxalate deposits, can they undergo only renal transplant without the need for liver transplant with LUMASIRAN, ?

Leave a Reply

Your email address will not be published. Required fields are marked *