Rare Disease Report

Biomarin Plans To Have a Very Good 2014

JANUARY 13, 2014
RDR Staff

Biomarin has had a very newsworthy day. Early this morning, the company announced they plan to soon begin clinical development for its gene therapy to treat hemophilia A. They also announced that the American College of Medical Genetics and Genomics ( ACMG) Practice Guidelines recommend treatment of PKU should be a life long commitment and should include pharmacotherapy. That is good news for the company given they have the only drug approved for PKU and are hoping to get second drug approved  for PKU next year.

And this afternoon, the CEO of Biomarin, Jean-Jacques Bienaime, spoke at the 32nd Annual J.P. Morgan Healthcare Conference. Highlights from that presentation are provided below. While Biomarin has 4 orphan drugs on the market that bring in approximately $530-$555 million in revenue in 2013 (Naglazyme® (galsulfase) for MPS VI, Aldurazyme® (laronidase) for MPS I, Kuvan® (sapropterin dihydrochloride) Tablets for PKU, and Firdapse® (amifampridine) for LEMS), Mr Bienaime focused most of his presentation on products they have in development, including Vimizin and Peg-Pal.

Vimizim (GALNS) for Morquio Syndrome
 Vimizim is an enzyme replacement therapy for patients with MPS IVA (Morquio Syndrome).  Morquio syndrome 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.
According to Mr Bienaime, there are an estimated 3000 patients with Morquio syndrome worldwide. To date, Biomarin has identified 1500 patients and currently has 274 in active trials.

Moving forward, Biomarin hopes to gain approval of Vimizim this year in the United States (first quarter 2014) and Europe (second quarter 2014). A PDUFA date of Feb 28th is currently on the calendar in which they expect will lead to approval. In an Advisory Board meeting late last year, the panel voted 20-1 in favor or approval.

And while a final price has not be set, Mr Biemaine stated they expect 2014 earnings from Vimizim to be in the $60-$70 million range. Not bad for a first year.

Peg-Pal for PKU
Peg-Pal (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’) is an enzyme substitute therapy for patients with phenylketonuria (PKU) which 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, many patients do not respond to Kuvan as well as needed and Biomarin is hoping that Peg-Pal may be an alternative for those patients. According to Mr Bienaime, there are approximately 10,000 adults patients with PKU in the United States but only 850 take Kuvan. The company hopes many of the remaining 9000+ patients not taking Kuvan will benefit from Peg-Pal. At present, the company has 1850 of those patients in their database. How many will benefit from Peg-Pal is not clear.
Mr Bienaime noted that they expect to complete their pivotal phase 3 studies by the end of 2014 and submit their BLA to the FDA in the first quarter 2015.

Other Drugs in Pipeline
Mr Bienaime noted several other orphan drugs currently in phase 1 or 2 trials. These included orphan drugs to treat late onset Pompe disease, achondroplasia, and late infantile CL2N Batten disease.

BMN 701 for Late Onset Pompe Disease. 
This is a lysosomal storage disorder caused be a deficiency of acid alfa glucosidase (GAA) that leads to accumulation of glycogen in muscles. There are currently 3000-6000 patients worldwide and while there are orphan drugs for this condition (Myozyme/Lumizyme), there remains an unmet need. Boimarin has completed its phase 1/2 study and plans to begin enrolling for its phase 2/3 study beginning first quarter 2014.
BMN 111 for Achondroplasia
This is a common form of dwarism.  There are 18,000 – 24,000 patients in United States and Europe with achondroplasia and the company hopes that its drug, BMN 111 will help approximately one quarter of those patients.  A phase 1 study has been completed and they plan to start a global phase 2 study first quarter 2014.
BMN 1090 for Late Infantile CLN2 Batten Disease
This is a very rare  and fatal condition due to mutations in the CLN2 gene. There are only 400-600 patients worldwide with the condition. Most die by the age of 8-12 years of age. According to Mr. Bienaime, the company has initiated a phase 1/2 study in the third quarter of 2013 and they are cautiously optimistic that this drug has potential for rapid approval due to the limited number of patients involved and the severity of the condition.

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