Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) has announced the initiation of a phase-1/2 clinical trial of ALN-GO1. The drug is targeted at the treatment of Hyperoxaluria type-1. The trial is expected to begin with the administering of the drug in healthy volunteers, followed by PH1 patients. The drug already holds the Orphan Drug designation, from both the FDA and the EMA. Data from these trials is expected to be released during the later part of 2016.

It is estimated that a total of 5,000 patients are affected by PH1 worldwide, making it an ultra-rare orphan disease. The disease is usually diagnosed when the patients are in their childhood, but can rapidly progress, resulting in kidney failure and organ damage. PH1 is actually an in-born error of metabolism, leading to excessive production of oxalate.

However, due to a lack of treatment options, patients often need to undergo frequent renal dialysis or combined liver/kidney transplant. ALN-GO1 targets glycolate oxidase, in genes, resulting in starvation of the oxalate production pathway. Alnylam expects to enroll 40 healthy volunteers and 20 PH1 patients, for the trial. The company has stated that the study is aimed at evaluating the safety and tolerability of the drug in single and multiple subcutaneous doses.

A recent filing by Eastern Capital, to the SEC, has revealed that the hedge-fund holds a stake of 3.8 million shares, 4.5%, in ALNY. The move indicates that the fund sees potential in the company and expects to improve significantly over the next few months. Other large shareholders include Portfolio Services LTD (4.5%) and Kenneth B. Dart (4.5%). Furthermore, Alnylam is expected to present at the Barclays Global Healthcare Conference, due to be held on March 15, in Miami. A webcast of the presentation, as well as the company’s presentation at the Cowen and Company 36th Annual Healthcare conference, would be available on the company website.

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) completed the March 9 session, with a gain of 4.5%, in its share value, to reach a close at $60.12.