A little dramatic Neo. You really think waiting 2 months is a life or death decision only indicates you are a moron when it comes to the practice of medicine. You seem to be a rather worried short that when Drisa is put through the ringer by Adcom, you know practicing physicians, Drisa will get hammered. Go luck with your position knowing jack about medicine. Your a tool...hahahaha!
Louie, I think everyone knows your feelings regarding SRPT's phase II data. No need to go there again. Yet The FDA's letter to then RNA was less than encouraging with the data that now BMRN used to submit. With Ed Kaye at the helm SRPT has other provided the data The FDA suggested or is the process of doing so. Conversely BMRN has not done so. When it comes to side affects there is no comparison. As a clinician I do have the advantage of talking to other clinicians around the country. I can tell you with all confidence that they are not impressed with Drisa data. So I stand by my statement that Drisa will not find a friendly Adcom.
I know many of you believe Drisa will get FDA approval. I guess I am a holdout on that feeling. I am probably naïve that the in the end the FDA is on the up on up. How could they ever approve a drug that has never achieved SS in any trial and is wrought with serious adverse effects. BMRN/RNA chose to ignore the FDA's strong suggestion that further studies should be done. I believe Adcom will not be so kind to Drisa. After all most of these clinicians are on the front line taking care of the boys with DMD. Should it be approved I would fall into the camp of waiting until Etep is on the market. My colleagues feel the same as me for what it's worth.
Thig, as you know, peer reviewed journals frown upon any sort of publicity prior to publication. The NEJM flat out tells you in their instructions for submission. Actually I kind of like that SRPT research goes through the peer review process. It makes in nearly impossible for the shorts to take on prestigious journals. Now this is not to say every advance in DMD needs to be published but if a advance was a super one of kind advance then I would prefer to see it published. Additionally, the FDA slurps this kind of publication right up.
Man, you make it hard to have a reasonable conversation with you. You really need to get out more Louie. The above is a list of applications that this backbone can be adapted for. Funny this came from a top draw research report written by PhD, MD's, analysts, and more. These men and women have stellar reputations and are names you would recognize. Seriously you need to collaborate more. You don't have all the answers my friend. Try opening your mind more. Here is more of the table of contents:
List of Tables 6
List of Figures 6
Sarepta Therapeutics, Inc. Snapshot 7
Sarepta Therapeutics, Inc. Overview 7
Key Information 7
Key Facts 7
Sarepta Therapeutics, Inc. - Research and Development Overview 8
Key Therapeutic Areas 8
Sarepta Therapeutics, Inc. - Pipeline Review 11
Pipeline Products by Stage of Development 11
Pipeline Products - Monotherapy 12
Sarepta Therapeutics, Inc. - Pipeline Products Glance 13
Sarepta Therapeutics, Inc. - Late Stage Pipeline Products 13
Phase III Products/Combination Treatment Modalities 13
Sarepta Therapeutics, Inc. - Clinical Stage Pipeline Products 14
Phase I Products/Combination Treatment Modalities 14
Sarepta Therapeutics, Inc. - Early Stage Pipeline Products 15
IND/CTA Filed Products/Combination Treatment Modalities 15
Preclinical Products/Combination Treatment Modalities 16
Discovery Products/Combination Treatment Modalities 17
Sarepta Therapeutics, Inc. - Drug Profiles 18
Product Description 18
Mechanism of Action 18
R&D Progress 18
Product Description 21
Mechanism of Action 21
R&D Progress 21
Product Description 22
Mechanism of Action 22
R&D Progress 22
Product Description 23
Mechanism of Action 23
R&D Progress 23
Product Description 25
Mechanism of Action 25
R&D Progress 25
Antisense Oligonucleotide to Inhibit AcpP for Burkholderia Cepacia Infections 26
Product Description 26
Mechanism of Action 26
R&D Progress 26
Antisense Oligonucleotide to Inhibit Myostatin for DM
There you go again. Louie I was calling you out. Geez. I was just thinking long term. Not tomorrow. What I making up is not garbage. I came across this today have a peek. I'm not the only one thinking down the road. Do you think the wright Brothers had a dream?
Antisense Oligonucleotide for Acinetobacter baumannii Infections 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Antisense Oligonucleotide for Drug Resistant Neisseria Gonorrhoeae Infections 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Antisense Oligonucleotide for Drug Resistant Pseudomonas Aeruginosa Infections 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Antisense Oligonucleotide for Methicillin Resistant Staphylococcus Aureus Infections 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Antisense Oligonucleotide for Rare Disease 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Antisense Oligonucleotide to Activate Lamin A for Progeria 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Antisense Oligonucleotide to Inhibit NDM-1 for Klebsiella Pneumoniae Infections 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Antisense Oligonucleotide to Inhibit TLRs for Systemic Lupus Erythematosus and Graft Versus Host Disease 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Antisense Oligonucleotides for Infectious Diseases 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Bacterial PPMO 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Product Description 43
Mechanism of Action 43
R&D Progress 43
Product Description 44
Mechanism of Action 44
R&D Progress 44
Viral PMO-X 45
Product Description 45
Mechanism of Action 45
Read the chapter headlines Louie!
Louie, I understand where you are coming from. As of now SRPT's infectious disease programs are in their infancy. Yet, Ebola and Marburg and corresponding platforms at least show safety. The next logical step is efficacy. However, I am thinking more long term and different diseases that are endemic but can be fully epidemic. For example influenza or MRSA. I understand mutation is a problem but as rapidly as biotech is progressing it seems to me that SRPT's patents have great promise. So I invest now fully understanding it is early. In the meantime the exon deletions are front and center. This could give SRPT a advantage to work on other treatments that may end up being big advances we have never thought about.
Your point is right on and fully understood by players here that see long term value in the platform. It is only those like Simp and Hsimp that fail to grasp the implications of the NEJM article. In fact I added to my core position upon the news. John Moulton full understands. This man knows more about morpholinos than any of us on the board. I will listen to him as you did. Sorry Louie even you but you I am beginning to think you're not a fruitcake when you nicer and more patient.
As usual, you missed mweather's point. The article appearing in the NEJM essentially validates the PMO plus platform. This is not a distant cousin of Etep. While I have no issue on focusing on the near term catalyst...Etep it is ridiculous to think the more savy street players missed that the platform extends beyond Etep. That is good for the stock in the long term. Funny you and Simp have the same set of glasses. Namely both of you suffer from tunnel vision. Look beyond Ebola/Marburg and tell us what you see in your little brain.
Conrad, don't even try to educate simpleton Simp. He has tunnel vision obscured my who knows which drugs. He thinks this is just a Ebola or Marburg treatment. He fails to see the much bigger picture thar SRPT can essentially design drugs effective against a host of infectious agents not to mention other genetic diseases. He would prefer that SRPT stay a DMD company only. These articles don't get published in the NEJM because they are a joke. The editors and reviewers clearly get the significance.
Sure I can. What percentage of vaccines are 100% effective in preventing the intended disease? What percentage of the general population in a developed country actually receive the vaccine. Now consider Africa and the disparate populations and beliefs, the geographical isolation, and host of other factors. What percentage even get the vaccine and how many of those who receive it will be protected. Nowhere near as many as you think which means a oral or IV agent will be needed. I could go on but I doubt if you will get it. You obviously don't understand the bigger picture. If this can be done for Marburg it can be done as Dr. Mouton mentioned with a number of viral infections. SRPT is no longer just a DMD company. When they have the financial wherewithal they will be able to branch into infectious disease.
Louie, I was talking about a broad spectrum of infectious disease. Not necessarily in the next few months but the platform does have that potential given time. That is why I think this safety study is such a big deal. Portends of things to come!
Ha Thig. Do you think SRPT has enough money to get her behind a drug she likely detests given Drisa upcoming failure even with the one in hundred thousand both are approved.
Louie, look at the relationship between New Link Genetics and Merck if my memory is correct. Merck gave NLNK a large cash infusion to partner up on a Ebola vaccine. Maybe now some major pharma outfit will want to partner up with SRPT. I think this is fairly significant for SRPT in regard to acute treatment. The one trick pony is no longer valid.
John, could you explain, as a expert in this technology, what this means for other viral disease. Ie, influenza, viral encephalitis, Ebola, and a myriad of other infectious diseases. Assuming now that the backbone and platform are safe how difficult would it be to add a sequence to the backbone to allow efficacy across a broad spectrum of infectious diseases. I, for one, would be happy if you could elaborate on the above and any additional comments you feel are pertinent. Thanks!!!
I could rip you vaccine argument to bits and pieces. You make way to many assumptions medically to even be taken seriously. I won't bother. This is big ant way you want to slice it.
I would think this will help. This is a piece of news we were not expecting at this time. When you think about complete safety in 44 volunteers this should put a nail in the coffin of those that don't believe in the backbone or platform. One could even argue that it will help those in Adcom when they are made aware that Eteps backbone and AVI-7288 are either identical or very similar. I am a lousy predictor of how the market will react in the morning but I would think we get a pop.
While I agree hws, it is highly unlikely that primary care Docs will be treating these boys initially I do think SRPT should meet with those Doctors experienced in treating boys with DMD,not just about Etep,but other exons in the pipeline. After all the only treatment agent has been some form of steroid up until now.