Tag Archives: request

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.

Google Releases Payments App, Mounts Pressure On Apple, PayPal

Google announced a pilot  app program Wednesday that aims to make it more seamless to use its Android Pay payments app. The new app, called Hands Free, will remove the need to use a smartphone at checkout, when shopping at merchants participating in the pilot — which for now is limited to a southern portion of the San Francisco Bay Area. With Hands Free, shoppers at checkout tell cashiers that they want to “pay with Google.” Checkout computers, equipped with the Android Pay app, then let cashiers call up a photo that the shopper already has uploaded to the app, to verify the shopper’s identity, and then complete the transaction. In select stores, Google is testing the use of in-store cameras to automatically confirm a shopper’s identity, based on the uploaded picture. Google is a subsidiary of Alphabet ( GOOGL ). The announcement signals Google’s growing interest in the crowded payments sectors . It’s a complex and competitive arena, with companies in the mix including  Apple ( AAPL ) with Apple Pay;  PayPal ( PYPL ) which recently split off from eBay ( EBAY ); and recent  IPO  Square ( SQ ). If Hands Free indeed signals that Google plans to aggressively tackle online payments, it could spell trouble for PayPal, according to Alex Rampell, a general partner at the noted venture capital firm Andreessen Horowitz. PayPal executives stick to their message that the San Jose, Calif.-based company is well-positioned, in part because unlike Google or Apple, it’s platform agnostic. For example, PayPal processes many of the transactions conducted over Apple Pay. On Monday, San Francisco-based Square announced that it was adding the ability to store money on its Square Cash app, adding a feature already offered by PayPal and PayPal subsidiary Venmo. Venmo and Square Cash are peer-to-peer payments apps that are popular with millennials, which use them for things such as sharing the cost of a cab fare or meal. At least one analyst says  Venmo stacks up well against rivals such as Google and Apple. Amazon.com ( AMZN ) is also in the payments business — and that’s the reason, according to industry watchers, that Amazon shoppers will not be able to pay with PayPal at any time in the near future. When PayPal spun off from eBay, executives said at the time that one advantage was the new business opportunities it couldn’t get while attached to eBay.