January 27, 2015
By Mark Terry, BioSpace.com Breaking News Staff
San Rafael, Calif.-based BioMarin Pharmaceutical Inc. ’s approach to clinical trials for rare-disease drugs has the industry talking.
On Jan. 12, 2015, BioMarin announced interim results from its Phase 1/2 pivotal trial for BMN 190 (cerliponase alfa) for treatment of patients with late infantile CLN2 disease, a form of Batten disease. Of the 24 patients enrolled in the study, only three are in the United States. The majority of the children involved in the study receive their weekly injections at the University Hamburg-Eppendorf in Germany or in Rome at the Bambino Gesù Children’s Hospital.
The company’s President and CEO Jean-Jacques Bienaimé, indicates that Europe allows for more flexibility in setting up clinical trials. This flexibility allows for lower costs.
CLN2, or infantile neuronal ceroid lipofuscinosis, is a form of Batten disease. Only about 400 to 600 patients are diagnosed worldwide. The disease is a fatal, inherited disorder of the nervous system that primarily effects young children. Symptoms first appear between the ages of 5 and 10, primarily vision problems or seizures. Most die by the late teens or early twenties.
On Nov. 24, 2014, BioMarin acquired ProSensa Holding N.V. for about $840 million. As part of the agreement, BioMarin gained worldwide rights to several orphan-drug candidates. Of primary interest is Prosena for the treatment of Duchenne muscular dystrophy and other genetic disorders.
At least part of what is making BioMarin’s approach so interesting to investors and analysts is the possibility that if BMN-190’s trial results continue to be as effective as they have been, U.S. and European regulators might grant early approval, rather than wait for a full year of data from the 24 patients involved in the trial. Of course, it’s always possible the regulatory agencies will require more data and more patients.
“The interim data represents an important step on a journey to develop a treatment for CLN2 disorder that may be able to slow the course of this fatal disease,” said Angela Schulz, with the Department of Pediatrics at the University Medical Center Hamburg-Eppendorf in a statement. “We appreciate the commitment of the children and their families who are participating in this study.”
The primary end point of the clinical trial is a standardized mobility and language score that uses a CLN2-specific rating scale. It measures performance of mobility and language on a scale of 0 to 6 for each function. In the typical course of the disease, patients lose one point every six months. Over a course of two to four years, they generally lose most language and mobility function. In the clinical data so far, patients treated for more than six months and up to 15 months, six of the nine patients showed no net change in their scale score. The remaining three only showed a one-point decline.
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