Vertex Pharmaceuticals

[Brahim_av]

Vertex Pharmaceuticals is een Amerikaans biofarmaceutisch bedrijf gevestigd in Boston, Massachusetts.
Het bedrijf werd in 1989 opgericht door Joshua S. Boger.
(NASDAQ:VRTX)

Het was een van de eerste biotechbedrijven die een expliciete strategie van rationeel medicijnontwerp hanteerde in plaats van combinatorische chemie. Het bedrijf heeft hoofdkantoren in South Boston, Massachusetts, en drie onderzoeksfaciliteiten, in San Diego, Californië, en Milton Park, in de buurt van Oxford, Engeland.

VRTX houdt zich bezig met het ontdekken, ontwikkelen, produceren en op de markt brengen van geneesmiddelen met kleine moleculen voor patiënten met ernstige ziekten.
Het bedrijf richt zich op de ontwikkeling en commercialisering van therapieën voor de behandeling van cystische fibrose, infectieziekten, waaronder virale infecties zoals griep en bacteriële infecties, auto-immuunziekten zoals reumatoïde artritis, kanker, inflammatoire darmaandoeningen en neurologische aandoeningen, waaronder pijn en multiple sclerose.

Vertex Pharmaceuticals Incorporated is huidig wereldmarktleider in de behandeling van Cystic fibrosis (CF) (of taaislijmziekte).
Cystic fibrosis (CF) is een ernstige, aangeboren ziekte. Kenmerkend voor CF is dat de slijmvliezen taai slijm afscheiden.
Dit veroorzaakt verstoppingen in met name de luchtwegen, de darmen, de alvleesklier en de lever.

Vertex is focused on discovering, developing and producing innovative medicines so people with serious diseases can lead better lives.
"Our scientists don’t see the impossible as an obstacle; they see it as a good place to start."

De leidende spelers op deze markt zijn onder meer Genentech, Inc., Novartis AG, Gilead Sciences Inc., Vertex Pharmaceuticals Incorporated, AbbVie Inc., GlaxoSmithKline Plc.,
Johnson & Johnson, Allergan plc, Pharmaxis Ltd. en Mylan N.V.

vertex-logo_2.png

Hoewel Vertex het enige bedrijf is dat momenteel medicijnen heeft in de CFTR-modulatorgeneesmiddelklasse, vallen andere spelers op met producten die nu op de markt zijn
en die tot andere medicijnklassen behoren. Naast het mucolytische Bronchitol (mannitol) in de Verenigde Staten en het PERT-product Pertzye (pancrelipase) in de Verenigde Staten,
bezit Chiesi twee antibiotica voor inhalatie: Bramitob/Bethkis (tobramycine) in de Verenigde Staten en vijf grote Europese markten (5EU )
(Frankrijk, Duitsland, Italië, Spanje en het Verenigd Koninkrijk) en Quinsair (levofloxacine) in de 5EU.
Twee andere belangrijke bedrijven zijn Viatris (voorheen Mylan), eigenaar van de meest populaire inhalatie-antibiotica, en Nestle HealthScience, eigenaar van twee PERT-producten.
Zenpep (pancrelipase) en Viokace zijn de PERT-producten (pancrelipase).
Gilead, Teva, Genentech, AbbVie, Vivus, Horizon Therapeutics en Pharmaxis zijn de andere farmaceutische bedrijven die momenteel medicijnen op de markt hebben die worden gebruikt
voor de behandeling van cystische fibrose.

[Brahim_av]

Vertex’s main area of focus is cystic fibrosis (CF). With its four CF medicines, Vertex is treating the majority of the 83,000 patients living with CF in the United States, Europe, Canada and Australia.

Vertex’s CF sales rose22% in the first half of 2022 primarily driven by higher sales of Trikafta (marketed as Kaftrio in Europe). Trikafta sales in 2022 are being driven by strong uptakes in international markets as well additional patients starting treatment with Trikafta, most notably pediatric patients (6-11 years of age) in the United States.

In the second half of the year, Vertex expects continued robust sales of Kaftrio/Trikafta in international markets following new reimbursements recently secured in France, Spain and Italy.

Meanwhile, Vertex is evaluating its CF medicines in younger patient populations and aims to have small molecule treatments for approximately 90% of people with CF. Additionally, Vertex is pursuing genetic therapies to address the remaining 10% of CF patients, including an mRNA approach in partnership with Moderna MRNA.

Vertex and Moderna plan to begin clinical development of their CFTR mRNA therapy in 2022/2023.

While Vertex’s main focus is on the development and strengthening of its CF franchise, the company also has a rapidly advancing mid- to late-stage pipeline in six additional diseases beyond CF like acute pain, sickle cell disease, beta-thalassemia, APOL1-mediated kidney diseases (AMKD) and cell therapy for type I diabetes. Many of these candidates represent multibillion-dollar opportunities. Programs in five disease areas are entering or progressing through late-stage clinical development. Multiple late-stage projects established proof-of-concept when data were released this year.

Vertex is co-developing a gene-editing treatment, exa-cel (formerly CTX001) in partnership with CRISPR Therapeutics CRSP, in two devastating diseases — sickle cell disease and thalassemia. Phase I/II studies of exa-cel in adult transfusion-dependent b-thalassemia in Europe and sickle cell disease in the United States are ongoing. Vertex and CRISPR Therapeutics plan to file regulatory applications for exa-cel for both indications in Europe and the United Kingdom in the fourth quarter of 2022 while discussions with the FDA are ongoing. Vertex expects exa-cel to be its next commercial launch.

In April, Vertex increased its investment in collaboration with CRISPR Therapeutics. Per the amended deal, Vertex will lead the global development and future commercialization of CTX001.

Inaxaplin (formerly VX-147) is being developed in a single pivotal phase II/III study in patients with APOL1-mediated kidney disease, or AMKD with two APOL1 mutations and proteinuric kidney disease. Enrollment is ongoing, with more than 30 sites active in the United States. In June 2022, the FDA granted Breakthrough Therapy Designation (BTD) to inaxaplin for treating APOL1-mediated focal segmental glomerulosclerosis, or FSGS based on phase II proof-of-concept data, which showed a 47.6% reduction in proteinuria.

VX-548, a novel first-in-class, non-opioid NaV1.8 inhibitor, is being evaluated in two phase II acute pain studies, one following bunionectomy surgery and the other following abdominoplasty surgery. In March 2022, Vertex announced positive data from the two phase II studies on VX-548. The studies demonstrated statistically significant and clinically meaningful relief of pain and established proof of concept for VX-548. Based on this data, the company plans to advance VX-548 into pivotal phase III development for acute pain in the fourth quarter of 2022.

A phase I/II study is ongoing on VX-880, Vertex’s stem cell-derived fully differentiated islet cell replacement therapy for the treatment of type I diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia. The program was placed on a clinical hold by the FDA in May, based on insufficient information for dose escalation, data available from the two patients treated at half dose demonstrated proof-of-concept. In July, the FDA lifted the clinical hold and the study resumed enrollment in the United States. Three patients have been treated in the phase I/II study to date.

Conclusion
Vertex’s dependence on just the CF franchise for commercial revenues is a concern. It needs a growth opportunity outside CF. Vertex’s non-CF programs carry significant risk, which is a concern.

Nonetheless, a consistent rise in CF sales, the rapid progress of non-CF pipeline candidates, minimal competition in its core CF franchise and regular business development should keep the stock afloat going forward.

[Brahim_av]

5 Augustus 2022

Vertex Pharma Raises 2022 product revenue forecast to $8.6 bln-$8.8 bln
from $8.4 bln-$8.6 bln previously, citing overseas demand for its blockbuster drug Trikafta, which treats cystic fibrosis (CF)

Reports Q2 adj. EPS of $3.60, beating estimates of $3.48 – Refinitiv data

As many as 7 brokerages, including Barclays, raise PT on VRTX
"While investors may see commercial performance of CF as
unexciting, that's the point; we're attracted to the company's
steady market expansion" - BMO

[Brahim_av]

2022-08-23 16_29_12-Vertex Pharmaceuticals Incorporated Common Stock (VRTX) Analyst Research _ Nasda.png

[Brahim_av]

https://www.vrtx.com/our-science/pipeline/

2022-08-30 11_36_26-Vertex Pharmaceuticals _ Our Science _ R&D Pipeline.png

2022-08-30 11_38_13-Vertex Pharmaceuticals _ Our Science _ R&D Pipeline.png

[Brahim_av]

BRIEF-Vertex And CRISPR Therapeutics Announce Global Exa-Cel
Regulatory Submissions For Sickle Cell Disease And Beta Thalassemia In 2022
13:52 (27-09) - Bron: Reuters

Sept 27 (Reuters) - Vertex Pharmaceuticals Inc, CRISPR Therapeutics AG :

* VERTEX AND CRISPR THERAPEUTICS ANNOUNCE GLOBAL EXA-CEL REGULATORY SUBMISSIONS
FOR SICKLE CELL DISEASE AND BETA THALASSEMIA IN 2022

* VERTEX PHARMACEUTICALS INC - EMA AND MHRA SUBMISSIONS ARE ON TRACK FOR Q4 2022

* VERTEX- EXA-CEL TO BE SUBMITTED TO U.S. FDA FOR ROLLING REVIEW BEGINNING
IN NOVEMBER, WITH COMPLETION OF U.S. SUBMISSION PACKAGE IN Q1 2023

[Brahim_av]

11 oktober 2022
Drugmaker provides update for its drugs, VX-634 and
VX-864, targeting a type of genetic disease
VRTX began human trial for VX-634, and expects to begin mid-stage study for VX-864 in the next few weeks ** Co is developing VX-634 and VX-864 as possible treatments for alpha-1 antitrypsin (AAT) deficiency, an inherited deficiency of the AAT protein which raises risk of several serious diseases ** Co adds that additional AAT-correcting molecules will enter clinical stage in 2023 ** Jefferies says VRTX has the potential to address both the pulmonary and hepatic issues of AAT compared to competitors who address either solely the pulmonary or hepatic aspect ** BMO Capital says "appreciate potential" in VX-864 mid-stage trial for long-term or cumulative benefit, but awaits additional clarity on rationale due to lack of efficacy ** Including session moves, VRTX up 35% YTD

[Brahim_av]

19:38 (28-10) - Bron: Reuters (vertaald vanuit het Engels)
** Aandelen van Vertex Pharmaceuticals Inc stijgen met 6,8% naar $ 307,61, handelend boven de 200-daagse
voortschrijdende gemiddelde prijs van $ 269,13
** VRTX rapporteerde in het derde kwartaal een stijging van 17,6% in de omzet, geholpen door sterke verkoop van zijn medicijn om cystic fibrose te behandelen (CF), een soort erfelijke aandoening van de longen en
spijsverteringssysteem
IN EEN EIGEN LIGA
** BMO Capital Markets ("outperform"; PT: $355) zegt het denkt "de concurrentiedreiging die uitgaat
van AbbVie is in het beste geval een gok"
** De makelaar voegt eraan toe dat "de enige echte concurrent van een Vertex product in CF is een nieuw Vertex-product"
** Baird ("neutral", PT: $ 280) zegt dat VRTX goed gepositioneerd is voor toekomstige fusies en overnames met
contant geld en equivalenten die in de nabije toekomst de grens van $10 miljard zullen overschrijden.
** Wells Fargo ("overweight"; PT: $ 335) zegt de combinatie van VRTX's CF-franchise en voortschrijdende pijplijn
"haalt waarschijnlijk het schoonste groeiverhaal in large-cap biotech"
** UBS ("kopen"; PT: $ 342) zegt dat de verkoopprognose voor 2022 is verhoogd maar lijkt nog steeds conservatief
gezien de ondergrens van het vooruitzicht een daling op kwartaalbasis zou impliceren.

[Brahim_av]

Sinds ik dit bedrijf heb gespot en op dit forum deel zie ik voorlopig enkel positief nieuws.
Er zit nog veel potentieel in!
Zeker de moeite waard voor long term investors!

Vertex: Promising Prospects Amid Stretched Valuation
Jul. 04, 2023 11:10 PM ETVertex Pharmaceuticals Incorporated (VRTX)7 Comments
Stephen Ayers profile picture
Stephen Ayers
4.45K Followers
Summary
Vertex Pharmaceuticals saw a 13% increase in Q1 2023 product revenue to $2.37 billion,
primarily due to the robust uptake of its cystic fibrosis treatments.
The company's net income fell due to higher expenses.
The FDA will conduct a priority review for Vertex and CRISPR Therapeutics' gene-edited therapy
for severe sickle cell disease by December 2023, and a standard review for transfusion-dependent
beta-thalassemia by March 2024.
Vertex advanced VX-548 to a pivotal, Phase 3 trial for the treatment of acute pain.
VX-548 has the potential to be a game-changer for pain due to its non-opioid properties.
Despite a high valuation, Vertex's strong growth, profitability, and pipeline prospects make it a
long-term "Buy" for any diverse biotechnology-focused portfolio.

[Brahim_av]

https://seekingalpha.com/article/4615177-vertex-promising-prospects-amid-stretched-valuation?mailingid=31995339&messageid=must_reads&serial=31995339.2186875&utm_campaign=Must%2BReads%2Brecurring%2B2023-07-05&utm_content=seeking_alpha&utm_medium=email&utm_source=seeking_alpha&utm_term=must_reads

Introduction
Vertex Pharmaceuticals (NASDAQ:VRTX) is a global biotech leader, dedicated to developing transformative treatments for serious diseases. With four approved drugs, Vertex has established a significant footprint in the cystic fibrosis [CF] market, providing treatments to the majority of the approximately 88,000 CF patients across North America, Europe, and Australia. Further, it continues to evolve its CF portfolio through label expansions, new medicines, and expanded reimbursements. The company's promising pipeline extends beyond CF to diseases like sickle cell, beta thalassemia, various pain conditions, APOL1-mediated kidney disease, type 1 diabetes, and alpha-1 antitrypsin deficiency. It's also exploring earlier-stage programs in muscular dystrophies. Through relentless scientific innovation and strategic investments, Vertex is focused on enhancing patient health with medicines targeting causal human biology, aiming to provide transformative and sustainable clinical and commercial success.

Recent developments: The FDA has accepted CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals' Biologics License Applications for exa-cel, a gene-edited therapy for blood disorders. The agency will conduct a priority review for its use in severe sickle cell disease by December 8, 2023, and a standard review for transfusion-dependent beta-thalassemia by March 30, 2024. Additionally, Vertex Pharmaceuticals is partnering with Lonza to construct a new manufacturing facility, dedicated to producing its islet cell therapies for type 1 diabetes. Vertex's T1D therapies, VX-880 and VX-264, are currently in phase 1/2 trials.

This article presents a brief review of the company's latest financial results, stock evaluation, forward-looking projections, and includes an investment suggestion.

Q1 2023 Earnings
Let's first start with the company's most recent earnings report. In Q1 2023, Vertex Pharmaceuticals saw a 13% increase in product revenue to $2.37 billion, primarily due to robust uptake of Trikafta/Kaftrio internationally and continued performance in the U.S. Domestic revenue grew by 3% to $1.4 billion, while revenue outside the U.S. rose by 33% to $971 million. However, expenses like R&D, Acquired IPR&D, and SG&A also increased to $1.3 billion and $1.2 billion respectively, due to higher acquired IPR&D expenses and support costs for advancing programs and global therapy launches. GAAP and Non-GAAP net income fell by 8% and 12% respectively, due to these higher expenses, despite strong revenue growth and increased interest income. As of March 31, 2023, Vertex had $11.5 billion in cash and marketable securities, a rise from $10.9 billion at the end of 2022, driven by revenue growth and operating cash flow. The company reiterated its 2023 financial guidance, forecasting CF product revenue of $9.55 to $9.7 billion.

VRTX Stock Assessment
Despite Vertex Pharmaceuticals' seemingly high valuation with a forward P/E of 24.09 and a price-to-book ratio of 6.28, the company exhibits robust growth, with a YoY revenue increase of 15.86% and strong three-year earnings growth. Vertex's profitability is also commendable, featuring high margins (gross profit margin of 59.06% and net income margin of 35.40%) indicating operational efficiency. The stock has outperformed the S&P 500 over multiple periods, demonstrating positive momentum.

Chart
Data by YCharts
While the high valuation and a modest debt burden of $789.2 million warrant caution, the company's strong growth, profitability, and positive momentum, supplemented by a substantial cash reserve of $10.41 billion, may make it an attractive consideration for investors.

Earnings Call Review
During the recent earnings call, Vertex Pharmaceuticals reported a strong start to 2023, with a 13% increase in Q1 global cystic fibrosis [CF] product revenues compared to Q1 2022. The company secured US approval for Trikafta in patients aged two to five years and completed Exa-cel U.S. rolling BLA submissions for sickle cell disease and beta thalassemia. With a promising pipeline, Vertex is focusing on multiple programs with potential for near-term launch, including exa-cel and the vanzacaftor triple in CF, and VX-548 for acute pain. Vertex also highlighted progress in their CFTR mRNA therapy, VX-522, gene-editing program, Exa-cel, pain program, VX-548, potential treatment for APOL1-mediated kidney disease, inaxaplin, and Type 1 diabetes programs. The company also updated about ongoing studies in Alpha-1 Antitrypsin Deficiency. The company cited strong finances, a broad pipeline, and a talented team as reasons for optimism for delivering for patients and shareholders in the future.

Vertex's VX-548 Shows Significant Pain Relief in Phase 2 Studies
I'd like to touch more upon VX-548 for the treatment of pain. Two Phase 2 studies conducted by Vertex Pharmaceuticals for their selective NaV1.8 inhibitor VX-548 showed significant improvement in acute pain following abdominoplasty and bunionectomy surgeries. The results suggest a rapid, statistically significant, and clinically meaningful relief in pain, as compared to placebo. The studies also included an active reference arm of the opioid hydrocodone bitartrate/acetaminophen (HB/APAP) to assess the effectiveness of VX-548. The drug showed a tolerable safety profile, with most adverse events reported as mild to moderate, and no serious adverse events related to VX-548.

Given the positive results from these Phase 2 studies, Vertex advanced VX-548 into a Phase 3 trial for acute pain (enrolling) and Phase 2 trial for neuropathic pain (enrolling).

VX-548's potential for the treatment of pain is promising. Unlike opioids such as HB/APAP, which were used as a reference in these studies, VX-548 targets a specific voltage-gated sodium channel (NaV1.8) implicated in pain signalling, potentially offering pain relief without the risk of addiction and other serious side effects often associated with opioids. Therefore, it could represent a significant advancement in pain management if it continues to demonstrate efficacy and safety in further trials.

My Analysis & Recommendation
In conclusion, I believe Vertex Pharmaceuticals presents a compelling investment opportunity. The company's commitment to innovating transformative treatments for serious diseases has driven impressive growth and profitability, and the robust pipeline across multiple disease areas shows significant potential. The company's robust financials, coupled with its strategic advancements in clinical trials, are driving the firm towards a promising future. The breadth and depth of Vertex's research and development strategy are noteworthy, with several promising programs close to market launch, including treatments for CF, sickle cell disease, beta thalassemia, and acute pain. Additionally, Vertex's success in the first quarter of 2023 highlights the strong market acceptance of its current products, underlining the strength of its business model.

Looking ahead, investors should monitor Vertex's progress on its clinical trials and the potential approval of its drugs, which could further propel growth. Keep an eye on the company's spending and its effects on profitability, given the increased R&D and SG&A expenses. Moreover, its high valuation and debt level are important factors to consider.

I am recommending a "Buy" for Vertex stock. However, given the company's high valuation metrics, the stock price may stagnate in the near term. Hence, this recommendation is tailored for long-term investors who can tolerate potential short-term downside risk. Over time, the company's robust growth prospects, profitability, strong balance sheet, and promising drug pipeline could generate substantial returns for patient investors.

Risks to Thesis
When the facts change, I change my mind.

As with any investment, there are several potential risks that could impact my "Buy" recommendation for Vertex Pharmaceuticals.

First, much of Vertex's success is linked to the continued growth and acceptance of its cystic fibrosis treatments. Any setbacks or unexpected competition in this space could negatively impact its revenue and profitability.

Second, the pharmaceutical industry is highly regulated. Any changes to health care laws, regulations, or policies could affect Vertex's business model and profitability. Delays in drug approvals, more stringent regulatory standards, or unanticipated side effects resulting in drug recalls could pose significant risks.

Third, the company's research and development projects involve substantial financial risk, and there is no guarantee of a profitable return on these investments. Any delays or failures in clinical trials could be costly and could adversely affect Vertex's stock price.

Lastly, Vertex's high valuation and significant debt level present financial risks. While the company's strong growth and profitability could support its high valuation over time, any financial or operational setbacks could lead to a stock price correction.