Congratulations first for a great medical achievement today.
Q-1. What is the life span of RBC with HbAT87Q?
Q-2. What happens if a patient who got lenti-B gene therapy, who now has good range hemoglobin, donates his blood to someone with B-thal or sickle cell or say to a normal person with acute blood loss anemia after trauma?
I think BLUE is going to be a biotech giant of over 100 B in coming years.
A positive Ib result could shot up the stock. We are talking about pancreatic, ovarian, glioblastoma multiforme, NSCL cancers and other ALT dependent tumors.
Immune therapy, PDL-1 and other kinase drugs improve survival by 3 months - 9 months and not truly meaningful survival drugs and are approved for advanced disease. VX 970 if works and has acceptable safety profile, will be used with first line topoisomerase 1 inhibitors and I consider this drug as " VRTX Avastin."
If I am buying VRTX at this level, it is because of VX 970. If drug was ineffective, it would never have re-entered the clinical trial and VRTX was running trials in Oxford for a while. I think safety is more of question than proof of concept. Preclinical models on this drug precursors like VX 821 and VX 822 are worth to read about. VRTX has huge patent protection portfolio for ART inhibitors. US government has patents for monoclonal antibodies for this target, so it is very valid drug target. Only AZN has a drug at present for this target.
551D/ 508 shows 661 improves FEV1 by 4.5% over Ivacaftor proving Iva is not enough and correctors have a role. Also as I posted earlier Phase 2 Delta 508 F of IVC lost improvement in the follow up period from week 16-40 published in Chest. Sep 2012, Vol 142. No 3. Page 718-724. 120 patient cohort is not a small group in the study.
It may seems to be negative to many but on the second thought if VX 771 can work independently as a potentiator for Homozygous and given 3% boost in lung function, which seems to be approvable to CF experts, then it will do the same for heterozygous i.e 3% improvement alone or with VX 661. So we have a drug Ivacaftor own by VRTX that will work all across CF, that is 70, 000 patients. CHECK MADE! Win win, no matter what?
10% royalities for VX Ivacaftor, Orkambi and VX 661 were sold by CF foundation for 3.7 billion in Nov 2014. Guess what simple math tells me ? 37 B only for CF franchise. VX 661 is positive in 2 phase II trial consistenly and its failure chances are very low after Orkami almost approval.
VX 970 ( ART inhibitor) is another blockbuster ready for intial read out. VX 787 is progressing nicely and Cethrin should start phase II b in July 2015. I can almost bet you that VX 745 will score big in Alzheimer Dementia and John Alum will sell his virtual company EIP biopharm and Alzheimer patents to VRTX.
I am expecting huge run from here. VRTX is a long term buy and shorting it is equal to losing your luck. Institutions owns it any way and they will do their math and initial manipulations to get cheap retail shares.
Congratulations to all my long friends including thirdme ( smarty), Dr. harveysmith, gladpick, verity and last but not least my positive approach friend qdelfan.
VRTX is next DNA ( Genentech)
no worries, it is secondary future offering authorization approval. Either they are planning to acquire a company or looking to raise big capital.
Orkambi should fly through the meeting tomorrow. VX 770 phase II trial was negative in patient with homozygous F 508 del. Trial had 112 patient in VX 770 group and 28 in placebo. At week 16 VX 770 group FEV-1 was 1.7 % better than placebo but follow up 16-40 week this effect was not sustained. This was published in Chest 2012, a peer review Journal.
Study 103 is now extended to 05/2016. Study was fully enrolled with 500 patients in 03/2015 and had stopped recruiting new patients then. My best assessment is that Primary end point has been met ( 2 week assessment #$%$ day combo of Oseltamivir and VX787) and now JNJ/ VRTX are extending this study for secondary end point i.e safety until next flu season to start phase III trial.
Dr. Jacqueline French NYU presented slides from AES 2013 on VX 765 and each arm has 10 patients only at various dosage for resistant epilepsy ( non conclusive but positive response at 600 mg and 1200 mg tid dosage). I know inflammation causes seizure and seizure causes inflammation. All SZ drugs cause drowsiness but VX 765 doesn't. I rest my case.
Craig Venture has very well said " it is not about what genes you got, it is about what you do with it".
This drug needs a proper proof of concept in resistant epilepsy at 600 mg and 900 mg dose. I think neurology academia is looking towards VRTX. Preclinical data is too strong to ignore that this drug has a strong role in epilepsy. Studies were discontinued prematurely after 40 patients in phase II b due to cost issues and pipe line prioritization ( Hep C disaster). This transformational medicine and first in class. My hope is this will lead to a anoxic brain injury drug in a cardiac arrest patient, an unmet and sad clinical scenario, where inflammation from anoxia causes cell death followed by brain edema and further cuts off brain circulation.
EIP pharmaceutical has started couple of phase II studies of VX 745 in AD including a dynamic quantitative PET scan study measuring amyloid plaque. This is going on under the analysts radar.
INCY gained 6 B from Baricitinib for RA and VX 509 is dead despite of a positive phase II B study in RA two years ago.
unfortunately they don't believe in science. same guy brought VRTX to $66 last year after CF conference in Europe. Bipolar disorder.
VRTX is not for sale. It carries a value way higher than $180. Cethrin data for Acute SCI is pointing in positive direction for C4-C7 injury. VX 787 phase II b will be out any day and it should be positive ( protease inhibitors carries a long success story in HIV and Hep C) to move into phase III next winter. VX 970( Avastin of VRTX) preliminary results 06/2015. Dr. David Altshuler is going to bring lot of innovative and transformational work in VRTX in coming years.
same people who are willing to pay for 6" of improvement. Have you every watch " Cialis" ad before the nightly news? Something is good about the word " six".
EIP pharma has licensed this drug from VRTX and they are starting a phase 2 a trial in VU medical CTR in Amsterdam. Does any one remembers or knows VRTX deal with EIP? Drug basis for AD look very attractive.
I think Cethrin is very exciting for acute SCI. C5 level is the most common after MVA and data in 40 patient study published in Journal of Neurotrauma with C4-C7 is very interesting. Combining Cethrin with Acorda or NVS drug will not be a bad idea.