Today’s Research Reports on Stocks to Watch: Ultragenyx Pharmaceutical and BioMarin Pharmaceutical

NEW YORK, NY / ACCESSWIRE / May 29, 2018 / Ultragenyx Pharma shares jumped after announcing its clinical trial results for Crysvita® ( burosumab), while BioMarin Pharma earned approval for its Palynziq™ ( pegvaliase- pqpz) Injection.

RDI Initiates Coverage on:

Ultragenyx Pharmaceutical Inc.
https://rdinvesting.com/news/?ticker=RARE

BioMarin Pharmaceutical Inc.
https://rdinvesting.com/news/?ticker=BMRN

Shares of Ultragenyx Pharmaceutical increased 4.36 percent to close at 67.31 on Friday, following its announcement of pediatric Phase 2 clinical trial of Crysvita® ( burosumab) results for the treatment of X-linked hypophosphatemia (XLH) in children aged 5 to 12 years. XLH is a rare, chronic progressive musculoskeletal disorder that affects children and adults. Crysvita is the first treatment to target the underlying pathophysiology of XLH – excess production of fibroblast growth factor 23 (FGF23), a hormone that regulates phosphate excretion and active vitamin D production by the kidney. According to Tom Carpenter, M.D., the lead study investigator, Director of the Yale Center for X-Linked Hypophosphatemia, and Professor of Pediatric Endocrinology at Yale University School of Medicine “This study was the natural extension of recent discoveries related to disease mechanisms in XLH, and in particular FGF23’s role as a mediator of renal phosphate metabolism.” “The therapeutic approach taken, to directly inhibit FGF23’s renal actions, demonstrates the potential advantages to therapy of metabolic disease when targeting a central disease mechanism. Sustained improvements in serum phosphorus resulted, with corresponding improvement in skeletal abnormalities. The approach should prove to be an important advance in the therapy of XLH,” he added.

Access RDI’s Ultragenyx Pharmaceutical Inc. Research Report at:
https://rdinvesting.com/news/?ticker=RARE

On Friday, the BioMarin Pharmaceutical’s stocks gained 4.77 percent to close at 92.49, following its announcement of approval from the U.S. Food and Drug Administration (FDA) for Palynziq™ ( pegvaliase- pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. The symptom of PKU is inability to break down Phe, an amino acid that is found in all forms of protein, if left untreated, high levels of Phe may toxicate the brain and might lead to serious neurological and neuropsychiatric-related problems, impacting the way a person thinks, feels, and acts. “BioMarin is thrilled to be able to offer this important new therapy to adults with PKU who are unable to control their Phe levels with existing options. The approval of Palynziq is the culmination of more than a decade of perseverance by BioMarin employees dedicated to bringing treatments to PKU adult patients,” said Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin.

Access RDI’s BioMarin Pharmaceutical Inc. Research Report at:
https://rdinvesting.com/news/?ticker=BMRN

Our Actionable Research on Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) can be downloaded free of charge at Research Driven Investing.

Research Driven Investing

We are committed to providing relevant and actionable information for the self-directed investor. Our research is reputed for being a leader in trusted, in-depth analysis vital for informed strategic trading decisions. The nimble investor can leverage our analysis and collective expertise to execute a disciplined approach to stock selection.

RDInvesting has not been compensated; directly or indirectly; for producing or publishing this document.

Disclaimer: This article is written by an independent contributor of RDInvesting.com and Nadia Noorani, a CFA® charter holder, has provided necessary guidance in preparing the document templates. RDInvesting.com is neither a registered broker dealer nor a registered investment advisor. For more information please read our full disclaimer at www.rdinvesting.com/disclaimer.

CONTACT

For any questions, inquiries, or comments reach out to us directly at:

Address:

Research Driven Investing, Unit #901 511 Avenue of the Americas, New York, NY, 10011

Email:

contact@rdinvesting.com

CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.

SOURCE: RDInvesting.com

ReleaseID: 501047