Amicus Therapeutics (FOLD) is purchasing a portfolio of ten gene therapies for $100 million and the promise to pay up to $352 million if the treatments are approved and hit undisclosed sales milestones. The deal arrives just a month after the approval of
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, Amicus’ first drug.
“This is really the next step in putting us in a place where I always hoped Amicus could be,” says John Crowley, Amicus’ chairman and chief executive. But there’s more to this story than most business deals. Both Crowley and one of the executives at the company Amicus is purchasing have children with an extremely rare disease.
Amicus has continued to be a company surrounded by excitement. Crowley’s quest, at a previous company, to create a treatment for his two children, Megan and Patrick, was documented in a number of Wall Street Journal stories, a book, and a movie starring Harrison Ford.
At that time, Amicus’ drug, Galafold, for another extremely rare disease called Fabry disease, was facing questions from the FDA even as it was approved in Europe, Canada, Australia, and Japan to treat patients as long as their illness was stemming from specific mutations. The treatment isn’t cheap however as Galafold costs $315,000 per patient per year in the U.S., which is actually a normal price for a drug for an ultrarare disease.
The Galafold approval allowed Crowley to go forward to identify new assets. He identified them in Celenex, the company Amicus is buying. Its story closely mimics Crowley’s own. Celenex was created by a Hollywood producer, Gordon Gray, whose two children had an awful and rare disease called Batten’s disease, which results in profound and lethal neurological decline.
“John, I’m very aware of your family’s journey,” Gray said the first time they talked. “Twelve years ago I was one of the producers considering bidding on your life rights.”
Crowley says Gray, who had produced films including Secretariat and Invincible, exhibited “superhuman effort” to create a drug company. “He put all of that aside to find the science that could save his daughters.” He found the technology he was looking for in gene therapies created at Nationwide Children’s Hospital Center for Gene Therapy that use a virus, called adeno-associated virus, to insert genetic code into cells. The result was a ten-patient study that tested a gene therapy against the CLN6 version of Batten that Gray’s girls have. The girls were among those 10 patients.
Gray and Crowley first connected over the summer. After a long conversation, Crowley brought a team to Ohio State. After a full day, Crowley asked Gordon and his partner if they could return with him to New Jersey. They flew there and went to Crowley’s house, where Meghan was getting ready to go back to Notre Dame. They spent the next day together at Amicus’ headquarters in Cranbury, New Jersey. Across the negotiating table, Crowley says he made a promise.
2 Biotech Stocks Turning Heads After Key Developments This Quarter
There are very few sectors in the stock market that have consistently produced winning trades. You might be able to count such sectors on one hand. Biotech is certainly among those sectors due to the emergence of highly innovative companies. These firms tend to create new avenues of creation to address unmet medical needs.
There are plenty of promising companies in the sector but it is necessary for an investor to do research thoroughly. It makes finding the best biotech stocks a bit easier. Though no one’s guaranteed a win, sifting out bad stocks is the key. One must watch the market closely to come across promising biotech stocks. Here is a look at two biotech stocks that are worth watching.
Biotech Stocks To Watch: Moleculin Biotech (MBRX)
The first biotech stock to consider is that of Moleculin Biotech Inc (NASDAQ:MBRX), which made some gains on Wednesday on the back of positive interim results for one of its products. The company, which is involved in manufacturing medicines meant for extremely resistant tumors, released data with regards to its acute myeloid leukaemia medicine annamycin.
Moleculin revealed highly positive interim data from the Phase ½ clinical study of the medicine and naturally, it has resulted in a lot of excitement in the market with regards to the stock. The stock rose by as much as 2% on the back of the news in yesterday’s trading session. It is one of the key biotech stocks to keep an eye on over the coming days.
Biotech Stocks To Watch: Eyepoint Pharmaceuticals (EYPT)
The other biotech stock that has recorded impressive gains on the market today is the Eyepoint Pharmaceuticals Inc (NASDAQ:EYPT) stock. On Wednesday, the company announced an important deal with regard to dexamethasone intraocular suspension 9% (DEXYCU). Eyepoint announced that it has managed to reach an agreement with one of the biggest players in the U.S. integrated delivery systems.
[Read More] 3 Pharma Stocks To Watch In December 2019
The deal is for two years and the other party is going to offer DEXYCU in its aforesaid systems. It is a major boost for Eyepoint and it has resulted in 5% to close at $1.54 in Wednesday’s trading session.
2 Biotech Stocks to Watch As The Sector Hits New High
The biotech sector has thrown plenty of winners over the course of the last decade or so and if experts are to be believed then it is going to continued to do so for the foreseeable future. Great advancements are being made in the biotech industry and many companies are working on a highly advanced product.
In such a situation, it is imperative for an intelligent investor to look for companies worth investing in. One should watch the latest developments in the sector and track specific companies closely. Here is a look at two biotech stocks that are worth watching.
The first biotech stock to watch is that of Neoleukin Therapeutics Inc (NLTX), which has managed to gain significantly over the past two weeks. On November 13, the company released its third-quarter results and since then it has rocketed by as much as 75%. The Canadian company made a loss of $59.1 million in the quarter, which worked out to $2.26 per share.
The company is a biopharmaceutical company that is involved in immunotherapies using de novo protein design technology. The company’s lead product candidate is NL-201, which is an amalgamation of both IL-2 and IL-15 and helps in removing alpha receptor binding.
The other biotech stock that is worth watching is Verastem Inc (NASDAQ:VSTM). It made significant gains over the past week. On Monday, the company announced that it has submitted a Marketing Authorization Application for its product COPIKTRA to the European Medicines Agency.
COPIKTRA is a phosphoinositide 3-kinase. It’s meant for patients suffering from refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and relapsed or refractory follicular lymphoma (FL).
The company is seeking approval for the same in the European market. Verastem stated that it has seen significant improvement in patients who used the product. The stock rocketed by as much as 60% after the news broke and remains one of the stocks to watch this week.
2 Biotech Stocks To Watch Gaining Momentum Right Now
Biotech is a sector that has grown significantly over the past decade or so and continues to hold a lot of promise if market experts are be believed. Hence, it is no surprise that investors are almost always on the lookout for the next winners from among the hundreds of penny stocks in the market.
However, if one has to identify a promising stock, then he has to watch the market closely and do his own research quite thoroughly. Here is a look at two biotech stocks that should be watched closely by all investors.
Oramed Pharmaceuticals Hits New Highs
The first biotech stock that should be put into the watch list is that of Oramed Pharmaceuticals, Inc. (NASDAQ:ORMP). The company, which is engaged in developing oral drug delivery systems, announced highly positive results from the Phase 2b trial of its product ORMD-0801.
The product in question is an oral insulin treatment and if it is eventually approved then it would become the very first orally administered insulin product. Hence, the optimism around the product and the Oramed stock is perfectly understandable.
Over the course of the past two weeks, the stock has gained by as much as 135%. Moreover, the stock made a new 52-week high of $5.80 in Monday’s trading session.
Clearside Biomedical Jumps 70% in 2-Week
The other biotech stock that has enjoyed an impressive rally in recent times is that of Clearside Biomedical Inc (NASDAQ:CLSD). The company developed a specially designed suprachoroidal space (SCS) microinjection platform. It helps administer medicines to the eyes. One of the most important medicines in its pipeline, Xipere, treats macular edema. The disease can seriously affect a person’s eyesight.
Earlier this year, the company announced that it had decided to postpone Xipere due to issues with its contractors. However, a complete response letter from the FDA in October revealed that the regulatory body needed more information about the medicine. The information demanded did not include anything that was related to the effectiveness of the medicine. The stock has gained 70% over the past two weeks.
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