Tag Archives: technology

BioMarin Stock Up As Drug Slows Progress Of Deadly Rare Disease

Shares of rare-disease specialist BioMarin Pharmaceutical ( BMRN ) rose Thursday after the company reported late Wednesday that its drug for Batten disease was on track for approval after a successful trial. BioMarin presented data from its pivotal study of 24 children taking its drug cerliponase alfa for the rare genetic condition, which causes most of its sufferers to lose the ability to walk and talk by the age of six and to die before they reach puberty. Though at least 20 genes have been associated with Batten disease, cerliponase alfa targets patients with a CLN2 mutation, who BioMarin estimates number 1,200 to 1,600 in its commercial territories. BioMarin said patients taking the drug showed 80% less than the expected rate of decline in function for the untreated population, and that the treatment was generally safe and well-tolerated. MRI measurement also showed a slower rate of brain deterioration. The company said it plans to submit the drug for approval with the FDA and the European Medicines Agency around midyear. Cerliponase alfa has already been designated an orphan drug, which means it will be protected from competition in the near term if it is approved. “Whether FDA will approve this is not clear, but stabilization is a major treatment effect in these very sick children, and they’ve had ‘very fulsome dialogue about requirements to support registration’ with agencies,” wrote RBC Capital Markets analyst Michael Yee in a research note. “They haven’t communicated specifically what the numerical benchmark is to get approved, but agencies are aware of the natural-history evidence.” Yee estimates peak annual sales of at least $250 million for the drug, while Evercore ISI analyst Mark Schoenebaum reckons the opportunity at double that number. BioMarin stock was up nearly 2% in late-morning trading on the stock market today , near 89. The stock has been recovering since hitting an 18-month low on Feb. 10, as it suffered both from the market sell-off and the FDA’s rejection of its muscular dystrophy drug Kyndrisa in January. Other MD drug candidates haven’t been faring much better: The FDA put off making a decision on Sarepta Therapeutics ‘ ( SRPT ) eteplirsen until May after issuing a scathing assessment of the drug in its briefing documents, while PTC Therapeutics ( PTCT ) said Monday that the FDA had refused to receive its application for ataluren, despite its having already been approved in Europe.

5 Weird Things That Have Been Listed On eBay

On eBay ( EBAY ), you can buy one year’s worth of work — to be completed in little more than four months. While that listing may be a joke — it appears not to be — over the years eBay has garnered fame and infamy for the sometimes weird items and services people have listed on the popular e-tail website. And last week was no different. On Feb. 25, a prankster listed Egyptian President Abdel Fattah al-Sissi on the site after the president reportedly said, “By God, if it were possible for me to be sold, I would sell myself,” in an address on state television . His condition was listed as “slightly used” and the seller would not accept returns. Bidding surpassed $100,000 before eBay pulled the listing. Unlike many of eBay’s e-commerce rivals such as Amazon.com ( AMZN ), Wal-Mart ( WMT ) and even PayPal ( PYPL ), the San Jose-based company’s platform is set up in such a way that bizarre items are almost inevitable (though it is worth noting that you can buy some pretty weird stuff on Amazon as well). In that spirit, here’s a look at some of my favorite odd listings that have appeared on eBay: 1. As noted above, this one is currently live on the site . Listed is “one year of work equivalent to 2,000 hours of specialized labor over 4.25 months.” For $150,000 a “loyal, dedicated and tireless contractor” will labor 16 hours a day, seven days a week with no break or lunch — for projects like painting, light construction work and “bamboo plant propagation.” EBay spokeswoman Penny Bruce told IBD the listing is indeed in compliance with the company’s policies. 2. Another odd auction — again, completely legit — was the World’s Largest BBQ Pit offered by a Texas man for the princely sum of $350,000. The 76-foot-long truck trailer has 24 barbecue pit doors, a walk-in cooler, beer taps and a “place for stereo equipment” and TV. The truck cab was an extra $50,000. It sold. 3. In 2004, a man tried to sell an F-18 warplane via eBay for $1 million. Ultimately, the fighter jet did not sell — it used to be a Blue Angel-owned aircraft — but it did attract the attention of the FBI, which told the seller that the plane had to remain in the U.S. 4. The last of our favorite weird items sold via eBay is a letter that Nobel Prize winning physicist Albert Einstein sent to philosopher Eric Gutkind shortly before Einstein’s death. The letter references philosophical  and theological themes, and expresses Einstein’s belief that God does not exist. It sold for … $3 million.