Sarepta Therapeutics (NASDAQ:SRPT)
On Monday June 29, 2015 Sarepta had announced that it had completed its rolling submission of its New Drug Application — NDA — to the FDA. A rolling submission is an FDA policy whereby biotech companies can submit their marketing approval application in small parts on a rolling basis instead of doing it all at once in the end of the cycle. This NDA submission for approval is for a drug known as Eteplirsen which is an exon-skipping RNA drug produced by Sarepta Therapeutics.
Eteplirsen treats a rare disease known as Duchenne Muscular Dystrophy — DMD — whereby young boys lose the ability to create proper dystrophin levels to keep muscle movement in the body. Eteplirsen is a drug that is able to restore the body into being able to produce dystrophin levels once again thus in turn restoring proper muscle function for these boys with DMD. Sarepta stated that it had completed its final submission package to the FDA on June 26, 2015.
Now Sarepta must await for a PDUFA date — meaning the date where the FDA decides upon approval for the Eteplirsen drug in patients with DMD. One thing to note though is that the company stated that they also put a request in their final submission packet to the FDA. Sarepta requested to the FDA for the ability to receive an FDA rule known as “Priority Review”. Priority review allows the FDA to review a drug at a much quicker time frame instead of waiting 10 months later. If granted Priority Review that would allow Eteplirsen to be reviewed by 6 months instead of 10 months.
Celgene (NASDAQ:CELG) & Juno Therapeutics (NASDAQ:JUNO)
On Monday June 29, 2015 shares of Juno Therapeutics were up 38% in after-hours trade after announcing a 10-year partnership agreement with Celgene. This collaboration will involve both companies coming together to develop therapies in two distinct areas. These areas include immunotherapies against many types of cancers and auto-immune diseases. For the consideration of this partnership Juno will be receiving $1 billion dollars.
About $150 million of the $1 billion will be payed as upfront cash, and the rest will be Celgene buying 9.1 million shares of Juno at $93 per share. The good news though is that the investment doesn’t stop there because over time Celgene can elect to buy more shares of Juno if it chooses to do so bringing up its ownership stake much higher.
The way it will work is that Juno has the ability to commercialize its immunotherapy products in the U.S. while Celgene gets commercialization rights in all other territories. Juno will also receive royalty sales from the other territories that Celgene will be responsible for. All this is great news for Juno but it gets even better, because Juno will have the option to co-develop and co-promote certain Celgene products pending company authorization.
Applied Genetics Technology (NASDAQ:AGTC)
On Thursday July 2, 2015 shares of Applied Genetics Technology were up 22% after the company announced a partnership deal with Biogen to produce gene-based therapies for eye diseases. More specifically the total collaboration deal is worth more than $1 billion dollars. Biogen is making an initial investment of $124 million, of which $34 million is being put in as an equity investment by Biogen along with pre-clinical research funding for AGTC.
Since Biogen will technically own the drugs AGTC will be able to only receive single-digit to mid-teen royalty percentages from sales upon marketing approval for all the eye products in AGTC’s pipeline. Biogen investing in AGTC marks the huge demand for these gene therapy companies which attempt to correct faulty genes by replacing them with working genes. This in turn is theoretically expected to reverse/slow progression of the afflicted disease.
AGTC has a gene therapy platform known as the Adeno-associated Virus — AAV — gene therapy platform. Biogen will also be able to leverage AGTC’s technology for use in six different gene targets. Of which AGTC will get to choose three of the six gene targets that Biogen must go after. This deal was a good deal for AGTC because it didn’t have a lot of cash and now it won’t need to dilute to take its company to the next stages of clinical development.
Vertex Pharmaceuticals (NASDAQ:VRTX)
On July 2, 2015 Vertex announced that it had received FDA approval for its second Cystic Fibrosis drug known as Orkambi. Orkambi is a combination drug that combines a previously approved Cysitic Fibrosis drug known as Kalydeco together with a new one from Vertex known as lumacaftor. Cystic Fibrosis is a rare lung disease in which sticky mucus builds up in the lungs leading to lung damage and other lung problems.
Orkambi was approved for Cystic Fibrosis patients with the F508del gene mutation. There are about 8,500 Cystic Fibrosis patients that have the F508del mutation, and Orkambi was shown to help these patients. Orkambi was able to improve lung function in these Cystic Fibrosis patients by 2% in a late-stage clinical trial. For Now Vertex is targeting patients with this mutation that are age 12 and older.
Further testing will need to be done in Cystic Fibrosis patients that have this F508del gene mutation that are under the age of 12 before it can be approved for this age group. This approval is huge because many analysts predict that Orkambi could potentially obtain $5 billion in peak sales. The problem though is that like all other drugs that treat rare diseases the price tag is once again pretty hefty. That is because treatment with Orkambi will cost $259,000 per year per patient. For now insurance companies will pay up these high premiums costs for these drugs that treat rare diseases, but nobody really knows for how long.