invest in what you know. I have 2 kids with C.F. and buying this stock just continues to make sense. application to wider spread diseases will blow this up over next 5 to 10 years!
Apparently the FTC does not understand that Concert's CTP-656 is a modified version of VRTX patented Kalydeco. Concert will not be able to market that product by itself without hag to pay a very very heavy royalty to VRTX which makes the product uncompetitive and as expensive as Kalydeco
VX 787 to treat influenza A close to FDA approval based on announcement from JNJ today. Will likely be a big seller worldwide since it has significant improvements compared to tamiflu (shows benefit even if started after 48 hrs from onset of symptoms with good safety profile). Milestone and licensing/royalty payments from JNJ to VRTX likely next year if not sooner.
After hours, news of FDA approval for kalydeco to treat all 23 residual function CF mutations ages 2 and older. Revenue guidance for 2017 raised by company anticipating 900 additional patients to start treatment this year in the US, and news that 5 additional 'splice' mutations may get approval for ivacaftor monotherapy soon representing another potential 500 patients in the US to get treatment soon. Tezacafor NDA submission and approval by FDA likely in the second half of this year, and phase 1 and 2 triple combo clinical trial data expected in the 3rd quarter along with VX 371 data in treating 508dd with Orkambi and patients with PCD. Stock holding current price range despite market turmoil today, and likely to test prior all time highs as FDA approvals and positive clinical trial data get reported through the summer and early fall.
Ivacaftor has been added in PCD trial with VX 371. Also University of Alabama has started a small 40 patient trial of Ivacaftor in COPD in collaboration with NHBLI and VRTX. It is worth to mention that Polycystic kidney disease is also a "ciliopathy" and a simple Ivacaftor could improve patient out come and could delay the onset of renal failure.
Vertex Pharmaceuticals stock price target raised to $132 from $128 at Leerink MARKETWATCH – 6:54 AM ET 04/28/2017
".........funnel most of our revenue, or OpEx anyway, into R&D and new diseases. And that's exactly the kinds of disease we're looking for whether it's internal investments or external investments for what's after CF. And I think as you know, David Altshuler joined us several years ago. He's spent a lot of time, I would say, tailoring our portfolio of internal research to diseases like that. And there are diseases like that we've talked about like sickle cell disease, Alpha-1 antitrypsin disease, adrenoleukodystrophy. "
Let's hope they are serious about that.
Earnings were good. No reaction on the board!!
AXSM (MC $85 M)(Cash $50 M) 5x BIG Phase 3 in various indications targeting large Markets with first interim results in Q3 2017 = 2000% POTENTIAL..UNKNOWN LOW FLOAT GEM !!!
Undiscovered and massive undervalued Biotech Stock with lots of Big News on the way.This Stock is brutally undervalued with a Market cap of just $85 million and $50 million in cash .Founder and Ceo is the larget shareholder holding over 7 million shares (30%) more than 50% of O/S is owned by Insider and Institutions which is a great sign .
This undiscovered stock could be the next 10 bagger gem if just one of their 5 ongoing Phase 3 programs is successful .GL
Market-Cap: $85 Million Cash: $50.6 Million(cash runway into the first quarter of 2019.) Price:$3.70
Shares Out: 23 Million
Anticipated Near-Term Clinical Milestones
Clinical Trial Initiations: -- Phase 2/3 clinical trial of AXS-05 in AD agitation (2Q 2017)
Clinical Trial Readouts:
-- Phase 3 COAST-1 trial of AXS-02 in knee OA associated with BMLs, interim analysis (3Q 2017)
-- Phase 3 CREATE-1 trial of AXS-02 in CRPS, interim efficacy analysis (4Q 2017)
-- Phase 3 STRIDE-1 trial of AXS-05 in TRD, top-line data (1Q 2018)
From dough on Twitter. COWEN Names with the best potential to report Q1 results in excess of consensus include $ALXN $CELG $EXEL $ICPT $IRWD $KERX $PCRX $VRTX
A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi
Resource links provided by NLM:
Genetics Home Reference related topics: cystic fibrosis MedlinePlus related topics: Cystic Fibrosis Genetic and Rare Diseases Information Center resources: Cystic Fibrosis U.S. FDA Resources
Further study details as provided by Vertex Pharmaceuticals Incorporated:
Primary Outcome Measures:
Safety & Tolerability as measured by number of subjects who experience AEs, and clinically significant changes in ECG parameters, vital signs, Laboratory tests, Spirometry, and ophthalmologic tests. [ Time Frame: from baseline up to 28 days post last administration of study drug, up to 16 Weeks ] Safety & Tolerability as measured by percentage of subjects who experience AEs, and clinically significant changes in ECG parameters, vital signs, Laboratory tests, Spirometry, and ophthalmologic tests. [ Time Frame: from baseline up to 28 days post last administration of study drug, up to 16 Weeks ] Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: from study baseline at Day 28 in each Treatment Period ]
Secondary Outcome Measures:
PK parameter estimation of VX-371 trough concentration (Ctrough) [ Time Frame: from study baseline at Day 28 in each Treatment Period ]
Estimated Enrollment: 150 Study Start Date: February 2016 Estimated Study Completion Date: June 2017 Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Not a very complimentary report. Remember the CF franchise started with no input from Leiden. He just piggy banked on CF momentum to make himself very very rich over the years while the stock tanked from over $140+ to low $70.
"Vertex CEO's compensation, stock value fell in 2016"
Research uncovers potential new treatment to treat and stop progression of cystic fibrosis
Researchers from the George Washington University (GW), the University of Perugia, and the University of Rome have discovered a potential new drug to treat and stop the progression of cystic fibrosis. Thymosin α1 (Tα1) is a novel therapeutic single m
Going down from here..
SPRING CLINICAL TRIAL for VX 210 and Acute cervical SCI: I found this late stage drug to be a big mover for VRTX in coming months. This is a hidden gem that no one understands well. VX 210 phase I/ 2A data is very intriguing. 36% patient converted to ASIA C or D from ASIA A is a very meaningful clinical result after 12 month of follow up. I understand this was not a placebo control trial but this signal is very strong. Acute SCI costs over 1 million per patient per year and if this drug can show similar results in the spring clinical trial, it will be a mega-block buster. VX 803 results in ovarian cancer and lymphoma will be coming out in next few weeks.
Harry Boxer has posted his Charts of the Day video on VRTX at TheTechTrader site noting: Vertex Pharmaceuticals Incorporated (VRTX) had big drug news a few days ago, and it went from under 90 to 112. The last two days it has been consolidating. On Tuesday, it popped from 107 to 112.69, pulled back and closed up 4.84, or 4.5%, to 112.29, on 2.8 million shares traded. Solid volume. If it gets through Tuesday’s high, then we may very well see it run up to the 130-5 zone.
No new news today and it is up around $4. i wonder if there may be a takeover in the works?
Barron's piece has the following passage: Should investors be jumping into Vertex now? Jefferies analyst Brian Abrahams and team see more upside: Based on our updates, we are increasing our tgt to $126 and expect VRTX to continue to appreciate into first triple data. Model updates are described above and we increase our terminal growth rate based on distancing from competitors. We expect appreciation into POC 3x-combo data 2H17, which we view as much less binary given multiple shots on goal. We also believe that this de-risking makes this story increasingly attractive to investors and potential acquirers.