Biomarin Update - Many Orphan Drugs On Target for Approval or Phase 3 Study


Chief financial officer of Biomarin, Dan Spiegelman, recently spoke at the UBS Global Healthcare Conference. Even though Biomarin has 4 orphan drugs currently on the market that bring in approximately $500 million in revenue (Naglazyme® (galsulfase) for MPS VIAldurazyme® (laronidase) for MPS IKuvan® (sapropterin dihydrochloride) Tablets for PKU, and Firdapse (amifampridine) for LEMS), Mr. Spiegelman focused most of his presentation on products they have in development, with a focus on those submitted for approval or about to enter phase 2-3 clinical trials.


Drug Submitted for FDA Approval

Vimizim (GALNS) to Treat Morquio Syndrome

Morquio syndrome (MPS-IVA) is an inherited lysosomal storage disorder in which patients are missing the enzyme N-acetyl-galactosamine-6-sulfatase. The net result is an accumulation of glycosaminoglycans throughout the body and they have features common to many MPS patients (course facial features, abnormal bone development, short stature) as well as a plethora of other problems as gycosaminoglycans accumulate in various organs.

Biomarin’s Vimizim is an enzyme replacement therapy for patients with Morquio Syndrome.  According to Mr. Spiegelman, there are an estimated 3000 patients with Morquio syndrome worldwide and the biggest challenge is locating those patients. To date, Biomarin has identified 1300 patients.

The results from their recent pivotal trial showed Vimizim to achieve its primary endpoint (6 minute walk test) and Biomarin has filed a BLA in the U.S. and a market authorization application in the EU. They anticipate potential FDA approval of the drug in the 4th quarter of this year with additional international submissions in 2013 through 2015.

 Ancillary studies are currently underway to examine the efficacy of Vimizim in patients under 5 yrs and in patients with limited ambulation. A cardiovascular study is also underway. All three studies are expected to be completed mid 2013.


Drugs Expected to Be in Phase 2/3 or Phase 3 by End of 2013

Peg-Pal for Phenylketonuria

Phenylketonuria (PKU) is an inherited metabolic disorder caused by a deficiency in the enzyme phenylalanine hydroxylase (PAH).  There are approximately 50,000 people with PKU worldwide. Patients with PKU build up toxic levels of the amino acid phenylalanine or "Phe" that can lead to numerous mental and psychological problems.  Currently, Biomarin has one product, Kuvan, to treat patients with PKU. Unfortunately, some patients do not respond to Kuvan and Biomarin is hoping that their second drug - Peg-Pal (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’) will be an alternative for those patients. PEG-PAL is an enzyme substitute therapy for patients with PKU.

Mr. Spiegelman noted the company had a positive meeting with the FDA following the completion of their phase 2 study and they plan to begin a phase 3 study in the 2nd quarter of 2013. There is a possibility that the FDA will provide an accelerated approval of the drug based on sustained reduction in Phe levels but Mr. Spiegelman also indicated that the FDA may require demonstration of neurocognitive improvement with the drug. The open label phase 3 trial will include psychiatric and executive function endpoints in addition to an evaluation of blood Phe levels.

Since the company already markets Kuvan, a commercial infrastructure is already in place for Peg-Pal.

BMN-701 for Pompe Disease

Pompe disease is a glycogen storage disorder that destroys heart, lung, and skeletal muscle function.  Treatment options (Myozyme) are available for only a small percentage of patients (early onset). Biomarin’s BMN-701 is being tested on late onset Pompe patients. The company has completed a phase 1/2 trial that showed improvements in the 6-minute walk test and pulmonary function tests. The company plans to begin a phase 2/3 study by the end of 2013.

Mr. Spielgelmann noted that the improvement in pulmonary functions is of great interest to many clinicians and they plan to have pulmonary/respiratory function as the primary endpoint for the trial.

BMN-673 for Solid Tumor / BRCA Breast Cancer

Biomarin is also examining the safety and efficacy of BMN-607 in a number of oncology patient groups. BMN-673 is a PARP inhibitor and the company plans to present data at the ASCO meeting next month that will highlight their phase 1/2 program in patients with BRCA breast cancer, BRCA ovarian cancer, Ewings sarcoma and small cell lung cancer.  Mr. Spiegelman noted that the company plans to finalize the design of a phase 3 study in BRCA mutant breast cancer after successful end-of-phase 2 meeting with the FDA. They hope to begin that study by the end of this year.

In addition to the above drugs, Biomarin has a large pipeline of drugs in development, included a drug for Batten disease, Achondroplasia, and Hemophilia A.

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