March 1, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SAN RAFAEL, Calif. – Patients and investors are waiting with bated breath for BioMarin Pharmaceuticals to release late-stage results of its Batten disease therapy trial on Wednesday.
If the trial results are positive, The Street reports the company is prepared to move to file for regulatory approval by the U.S. Food and Drug Administration as well as the European Medicines Agency.
BioMarin is looking for 2016 and 2017 to be banner years as Jean-Jacques Bienaimé, BioMarin’s chief executive officer, expects the company to finally turn a profit based on its rare disease treatments. Positive results from the Batten disease therapy, as well as another late stage drug for phenylketonuria, a genetic disorder, would certainly help the company meet that goal. The drug, pegvaliase, would treat the buildup of the amino acid phenylalanine in patients diagnosed with phenylketonuria.
Company stock has fallen about 45 percent over the past year, but is currently seeing slight upward momentum in anticipation of positive trial results. BioMarin stock is currently trading at $83.53 per share, up from its opening price of $82.50 per share.
The company got off to a rough start in January when the FDA rejected BioMarin’s application for Kyndrisa (drisapersen), for the treatment of Duchenne muscular dystrophy. BioMarin is examining the FDA’s complete response letter to see how best to move forward with that drug. The drug is also up for approval in Europe, where there are approximately 5,000 patients eligible for the drug. The European regulatory body is expected to make a ruling on Kyndrisa this summer. There was some reprieve for BioMarin as the FDA also rejected rival drugmaker Sarepta Therapeutics ’ DMD treatment. Both drugs utilize “exon skipping” technology, which allows the damaged cells’ repair mechanisms to fix the specific genetic mutation that affects about 13 percent of individuals with the disease.
In addition to the Batten disease and phenylketonuria treatments, BioMarin is pushing a mid-stage drug for reveglucosidase alfa, a treatment for late-onset Pompe disease. During the annual J.P. Morgan Healthcare Conference in January, BioMarin announced positive results for the Phase II trial. Reveglucosidase alfa is a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA). The drug replaces the enzyme (GAA) that prevents the glycogen build up that causes Pompe disease. Interim results show patients improved respiratory muscle improvements, which the company said may indicate a possible halt in decline or improvement in lung capacity and endurance.
BioMarin also has a number of other treatments for rare diseases in its pipeline, including Vimizim, its enzyme replacement therapy for patients with Morquio A Syndrome patients, experimental treatments for dwarfism and a gene therapy for hemophilia A.
In support of its rare disease treatments, on Monday BioMarin launched BioMarin RareConnections to provide “personalized assistance to patients in the United States, who have been prescribed or are considering a BioMarin therapy for Morquio A, MPS VI and phenylketonuria (PKU), and their caregivers.”
“Patients and their families battling rare conditions face uncommon challenges. As a leader in the community, it is our responsibility to provide unparalleled support to help ensure our patients, who are eligible for BioMarin approved therapies, have access to, and are connected with, the exceptional care they deserve,” Jeff Ajer, BioMarin’s chief commercial officer, said in a statement.
