Stock of the Day

May 27, 2020

BioMarin Pharmaceutical (BMRN)

$55.27
-$0.78 (-1.4%)
Market Cap: $10.67B

About BioMarin Pharmaceutical

BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc. The company was incorporated in 1996 and is headquartered in San Rafael, California.

Today's Trend

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is under pressure despite positive long-term clinical data. Investors appear to be treating the Pompe disease update as confirmation of an existing product profile rather than a major new catalyst, while the company’s valuation and expectations may be limiting the immediate upside.

  • BioMarin reported five-year Phase 3 data for POMBILITI® (cipaglucosidase alfa-atga) plus OPFOLDA® (miglustat) in adults with late-onset Pompe disease. Patients maintained motor function, including walking ability, while respiratory function remained relatively stable; no new safety signals were identified. The results support the combination’s durability and may strengthen its long-term commercial position. BioMarin Announces Five-Year Phase 3 Data for POMBILITI + OPFOLDA
  • The long-term evidence is particularly relevant for BioMarin because sustained motor and lung benefits could support physician confidence, patient retention and continued adoption of POMBILITI plus OPFOLDA in the rare-disease market. BioMarin Reports Five-Year Pompe Disease Data Showing Durable Motor Function
  • BioMarin also partnered with Profluent to use artificial intelligence in designing novel medicines. The deal could expand future discovery capabilities, but it is an early-stage development and has limited near-term earnings impact. Profluent and BioMarin Partner on AI-Designed Medicines
  • A newly granted global license to Ascendis Therapeutics is expected to provide BioMarin with royalty revenue, offering a potentially useful source of recurring, asset-light income. BioMarin Grants Ascendis Global License
  • Shares declined following the Pompe data announcement, suggesting investors may have been looking for a more significant surprise or immediate financial upgrade. The reaction also reflects the risk that strong clinical results are already partly reflected in expectations, particularly with BioMarin trading at a high earnings multiple. BioMarin Adds Five-Year Evidence for POMBILITI + OPFOLDA
← Previous Stock of the DayNext Stock of the Day →

Recent News