U.S. Markets closed

BioMarin Begins Pediatric Study on Achondroplasia Candidate

Zacks Equity Research
1 / 1

NantHealth, Inc. (NH) Reports Q3 Loss, Misses Revenue Estimates

NantHealth, Inc. (NH) delivered earnings and revenue surprises of 23.08% and -10.83%, respectively, for the quarter ended September 2018. Do the numbers hold clues to what lies ahead for the stock?

BioMarin Pharmaceutical Inc BMRN stated that it has dosed the first participant in a phase II study, evaluating its pipeline candidate, vosoritide, in pediatric patients with achondroplasia, a genetic disorder causing dwarfism in infants and toddlers.

The phase II placebo-controlled study (n=70) on vosoritide will be conducted on children with achondroplasia in the age bracket of 0-5 years. The primary objective of the study will be to evaluate safety, tolerability and effect of vosoritide on height Z-scores.

We would like to remind investors that vosoritide is already being assessed in a phase III trial on children aged five to 11 years with achondroplasia. Top-line data from this study are expected to be announced in the second half of 2019, followed by an open-label extension analysis with the lower dose (15 µg/kg/day). Vosoritide has an Orphan Drug status in both the United States and the EU for achondroplasia.

Earlier in May, BioMarin received an FDA nod for its pipeline candidate, pegvaliase, to be marketed by the trade name of Palynziq injection, developed to reduce blood phenylalanine (Phe) levels in Phenylketonuria (PKU) patients.

BioMarin already has a PKU drug in its portfolio called Kuvan. Revenues of the same rose 7% to $99.1 million in the first quarter of 2018. Kuvan is a key growth driver of BioMarin’s revenues. Per settlement with Dr Reddy’s RDY and Par Pharmaceuticals, a subsidiary of Endo International plc ENDP, Kuvan generics are expected to enter the U.S. market from October 2020 onward.

Shares of BioMarin have inched up 0.9% year to date against the industry’s decline of 6.9%.

 

BioMarin currently carries a Zacks Rank #2 (Buy). Another top-ranked stock in the biomedical sector is CRISPR Therapeutics AG CRSP, sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here

CRISPR Therapeutics’ loss per share estimates has been narrowed by 7.8% for 2018 and 9% for 2019 in the last 60 days. The stock has skyrocketed 151.5% year to date.

Wall Street’s Next Amazon

Zacks EVP Kevin Matras believes this familiar stock has only just begun its climb to become one of the greatest investments of all time. It’s a once-in-a-generation opportunity to invest in pure genius.

Click for details >>


Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
 
BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report
 
Endo International plc (ENDP) : Free Stock Analysis Report
 
Dr. Reddy's Laboratories Ltd (RDY) : Free Stock Analysis Report
 
CRISPR THERAPTC (CRSP) : Free Stock Analysis Report
 
To read this article on Zacks.com click here.
 
Zacks Investment Research