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Vertex Pharmaceuticals Incorporated (VRTX)

NasdaqGS - NasdaqGS Real-time price. Currency in USD
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205.06+1.25 (+0.61%)
At close: 04:00PM EST
205.61 +0.55 (+0.27%)
Pre-market: 04:26AM EST
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  • J
    A couple of more positive announcements about clinical trials in progress treating additional patients with SCD Beta thal DM Type 1 or pain on top success just reported in AMKD will help the stock break out again after current consolidation. Patience will be greatly rewarded especially after the story to be told at JPM health care conference next month about the 3 different drugs entering pivotal late stage clinical trials next year with submissions for regulatory approval likely a year from now for CTX001 VX121 and VX561 with tezacaftor and possibly VX 147. Add positive results in the phase 2 clinical trials for treating pain and the stock will be hitting all time highs next year.
  • K
    Keith S
    Possible new medical breakthrough gives hope for people with Type 1 diabetes
  • d
    Vertex Pharmaceuticals price target raised to $265 from $250 at RBC Capital - RBC Capital analyst Brian Abrahams raise
    Vertex Pharmaceuticals price target raised to $265 from $250 at RBC Capital -
    RBC Capital analyst Brian Abrahams raised the firm's price target on Vertex
    Pharmaceuticals to $265 from $250 and keeps an Outperform rating on the
    shares. The analyst is updating his model to reflect more de-risked
    prospects for the company's VX-147 kidney program following its data release
    yesterday. Abrahams adds that while Vertex shares saw some upside on the
    news, the stock still "does not nearly reflect" the fundamental fair value of
    the company's CF franchise and pipeline.
  • G
    December 1, 2021 07:30 AM EST R&D
    Battling a lineup of skeptics, Vertex claims another early clinical win — this time in kidney disease
    Jason Mast
    Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

    The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

    Protein urine levels, or proteinuria, is a widely accepted biomarker for kidney function and a potential predictor for a patient’s long-term risk of going into kidney failure.

    “As a first result, I think it’s terrific,” said Jonathan Himmelfarb, director of the Kidney Research Institute at the University of Washington, who was not involved in the study. “If it’s sustainable and it reflects improvement in an injury process in the kidneys, it is potentially very significant.”

    Vertex sees the study as a proof of concept for its efforts to tackle a larger set of kidney diseases driven by the gene APOL1. Variants of the gene, which plays a role in the immune system, can help boost protection against parasitic infections, including human African trypanosomiasis, also known as African sleeping sickness.

    But patients who carry one or especially two variants are also at a higher risk of developing kidney disease. Vertex cites figures that 50% of African Americans carry at least one variant and 13% carry two. It’s one of several factors — alongside access to healthcare and other social determinants of health — that account for the wide disparity in kidney disease between white and Black Americans.

    Vertex’s molecule, VX-147, is designed to inhibit APOL1 and slow kidney injury.

    “It’s a very important target from a public health perspective,” Himmelfarb said.

    The data, while early, will likely come as welcome news for analysts, many of whom have remained skeptical of the company’s ability to repeat the success of its cystic fibrosis franchise. In a note Tuesday, Jefferies analyst Michael Yee placed the bar for success at a 15% reduction in proteinuria, but “the higher the better.”

    The 47.6% reduction came from 13 patients, with Vertex excluding 3 patients who it said did not comply with trial protocols. There were no serious adverse events, the company said. The most common side effects were headache, back pain and nausea, which all occurred in over 15% of patients.

    It’s the second successful readout for Vertex this fall after landmark results on a potential cure for type 1 diabetes came in October, albeit for just one patient.

    Vertex now plans to move VX-174 into a larger pivotal trial for multiple types of APOL1-mediated kidney diseases. The company did not disclose details for the study, but the FDA has said that, for certain kidney diseases, the agency will grant accelerated approval based on reduction of proteinuria, providing a clear path to market if early results bear out in a larger study.

    The company estimates 100,000 people in the US and Europe are currently living with kidney disease potentially addressable by their molecule.
  • C
    This stock is undervalued on just the existing CF franchise alone.
    With the promising data coming out of the diabetes type 1 and kidney disease studies this stock a must own stock.
    The prudent course of action would be to load up here and hold for the next few years.
  • R
    Great company getting into a new winning strategy the correct way. Look for a new high in 2022.
  • j
    Tide is turning. Next up positive news on pain
  • T
    Great potential - expect big leg up at next earnings
  • D
    For a 200 dollar stock to simply move 0.6% on such a Big Up day, is a bit depressing.
  • K
    My neighbor in ELYRIA, OHIO is the guy cured of his diabetes. It will be 5-10 years away before this is mainstream, so not an investment for me, I'm to old. I'm buying this stock in my trust to leave for my children.
  • C
    The time has come to buy as much as you can afford!
    This stock is an absolute giveaway at these prices!
    The upside potential is tremendous!
  • t
    Hopefully this will push us in an uptrend again.

    i do realy want to know who sold. Hedge funds and management bought. is it realy just retail investors that just sold their position
  • D
    The Kidney disease drug is Great news, but the Diabetes type 1 drug is AWESOME news.!!!
  • S
    Sunil Kololgi
    Anybody saw the nyt article ?
  • T
    What to expect in 6 years?
    1) There is no competition for the CF drugs for the next 2.5 years. Trikafta can stop lung function decline or can improve the lung function. For any competition to claim the same efficacy it will take at least 4.5 years (2.5+2 years of testing). The revenue and earnings from CF drugs will be at least 8B and $15 per share, respectively, in the coming year.
    2) There are 170K patients suffering from SCD and TDT, and there are 25K people with SCD in the US alone. CTX001 offers functional cures for these patients, and the registration filing would occur toward the end of the next year. For this indication there is $BLUE to compete for the same market. The cost for the treatment would be about $1.5MM. The COO estimated that a three-fold increase in benefitable patient number would occur if milder conditioning regimen became available. He is assuming about 6K SCD patients would be treated with the current conditioning. This means that a minimum 10 B for the revenue which can be realized over about ten years. The average annual rev. would be at least 1B.
    3) Another multibillion-dollar opportunity awaits us with the success of VX-147, which treats APOL1 dependent FSGS. There is no competition for treating the underlying cause of the disease. There are 100K patients with FSGS in the US and EU. I project the revenue from VX-147 could rival that from CF drugs. This means about 8 B per year a few years from the launch. It may take 6 years.
    4) Another multibillion-dollar market opportunity resides with the pain drug, VX-548 which is in Phase 2. Acute pain alone could generate 2B.
    5) VX-880 is dosed to treat Type-1 diabetes. A quarter of 60K T1D patients is already taking immune-suppressive drug. COO estimated that this is a 5B opportunity. It will be known how long this cell transplant will last.
    6) Status of AATD and DMD treatment will be known.
    7) To sum up, in several years (~6 years) Vertex could more than double the current revenue, and the market cap could reach 200B ($20B X 10). For rapidly growing company 10 times the revenue for computing the market cap is not unreasonable.
  • V
    Almost $204 now
  • J
    Successful people don't become that way overnight. What most people see at a glance wealth, a great career, purpose is the result of hard work and hustle over time. I pray that anyone reads this will be successful in life
  • B
    The New York Times article certainly gave this stock a boost, but I think we'll need another catalyst to keep the momentum going.
    I hit a paywall, so I googled the headline and came up with a lot more useful information than reading the NYT article.
    Although the NYT article itself has already been reprinted by various other news outlets, there are other stories offering relevant commentary on the subject, including similar "big news" from another biotech back in 2011, which actually was following another line of PREVENTATIVE treatment for the early staged of type 1 diabetes.
    In a nutshell, that previous announcement turned out to be a big disappointment, I suspect because the initial big announcement led people to believe a cure for longstanding cases of type 1 diabetes was discovered, which was not ever the case.
    In any case, there is some deep rooted skepticism surrounding the idea a cure will be found, and in this case, we are only looking at anecdotal evidence, as compelling as it may seem, and actual approval, pending a full series of trials could still be five years out at this point.
  • D
    Fantastic News about the Type 1 diabetes study/trial. Can't wait for the formal press release and peer reviewed article to come. This could be HUGE for vertex.
  • D
    Just Wait For The Peer Reviewed Article, About The Type 1 Diabetes Approach.!!