Latest news with #NaV1.8
Yahoo
07-08-2025
- Business
- Yahoo
7 Reasons Why Vertex Pharmaceuticals Is a No-Brainer Stock to Buy on the Dip
Key Points Vertex Pharmaceuticals stock sold off on two pipeline disappointments. The biotech company continues to grow with promising new drugs on the horizon. Vertex's price looks quite attractive, relative to its growth prospects. 10 stocks we like better than Vertex Pharmaceuticals › Vertex Pharmaceuticals (NASDAQ: VRTX) reported its second-quarter results after the market closed on Monday. Its shares plunged more than 17% in early trading on Tuesday. Were Vertex's Q2 results horrible? Not at all. Instead, investors reacted negatively after the big biotech company announced two disappointing developments with its pipeline. Such steep sell-offs can sometimes present great buying opportunities. I think that's the case with Vertex. Here are seven reasons why Vertex is a no-brainer stock to buy on the dip. 1. The bad news wasn't as horrible as the sell-off indicated One of Vertex's pipeline disappointments was that VX-993 didn't meet the primary endpoint in a phase 2 study evaluating the NaV1.8 pain signal inhibitor in treating acute pain following bunionectomy surgery. The other bad news was that the U.S. Food and Drug Administration (FDA) doesn't see a path for Vertex to obtain a broad label in peripheral neuropathic pain (PNP) for suzetrigine at this point. Vertex won't move forward with VX-993 as monotherapy in treating acute pain now. However, it's not the end of the world for a phase 2 program to flop. The company already markets Journavx (suzetregine) in treating acute pain. Also, Vertex isn't giving up on getting a broad PNP label for suzetrigine. It's regrouping, though, and prioritizing diabetic peripheral neuropathy (DPN) as its first PNP indication. The company plans to quickly initiate a second DPN phase 3 study. But Vertex will also work with the FDA to expand the DPN indication to add other neuropathic pain conditions and hopefully find a way to win a broad PNP label. 2. Journavx appears to be on track to become a megablockbuster Meanwhile, Journavx's commercial launch is humming along. Vertex CEO Reshma Kewalramani said in the Q2 earnings call that "formulary coverage is going really well," and "frankly, faster than I would have expected." She mentioned that the company's phase 4 confirmatory study data looks "really good, not only in terms of pain control, but also in terms of reducing opioid use." Chief Commercial Officer Duncan McKechnie revealed that Vertex is cranking up its marketing for Journavx and adding field support in response to the strong contracting and formula progress that has been achieved. He said the company is "receiving incredibly positive feedback from physicians and patients on how well Journavx is working for them clinically." According to McKechnie, Vertex remains highly confident that the pain drug will be another multibillion-dollar franchise for the company. 3. Vertex continues to grow robustly Almost lost in the shadow of the pipeline disappointments was the fact that Vertex continues to grow robustly. The company's revenue jumped 12% year over year in Q2 to $2.96 billion. It posted adjusted profits of $1.2 billion, a huge improvement from the $3.3 billion loss in the prior-year period (which was due to the Alpine acquisition). 4. Vertex's CF dominance is more secure than ever Vertex's dominance in cystic fibrosis (CF) appears to be more secure than ever. New CF drug Alyftrek is gaining momentum in the marketplace, especially with patients who haven't begun treatment with CFTR modulators or previously discontinued use of one of Vertex's other CF therapies. Alyftrek isn't just Vertex's best CF therapy yet. It should also be the company's most profitable CF drug because of a lower royalty burden, and its patents run through 2039. In addition, Vertex plans to restart the phase 1 dosing of VX-522 after a temporary pause. This messenger RNA therapy holds the potential to treat the 5,000 or so CF patients who can't benefit from Vertex's existing drugs. 5. Two new drugs could be on the way soon Vertex could soon add two new drugs with huge potential to its lineup. The company expects to file for regulatory approvals of zimislecel in treating severe type 1 diabetes in 2026. If all goes well with an interim analysis of a phase 3 study evaluating povetacicept in treating IgA nephropathy, Vertex could also file for accelerated approval of the drug in the first half of next year. 6. Trump administration policies aren't a problem for the company Some drugmakers could be hit hard by the Trump administration's tariffs on pharmaceutical imports and its plans to implement most-favored-nation (MFN) drug pricing. But not Vertex. CFO Charlie Wagner said in the Q2 call, "We expect an immaterial cost impact from tariffs in 2025 based on what we know today due to our significant U.S. presence and our geographically diverse supply chain." As for MFN, Kewalramani confirmed that Vertex didn't receive a letter that President Trump sent to multiple drugmakers demanding that they offer their medications to Medicare, Medicaid, and private insurers in the U.S. at the same low prices they charge outside the U.S. 7. The price is right Finally, the price is right to buy Vertex on the dip. The stock's price-to-earnings-to-growth (PEG) ratio, which is based on five-year earnings growth projections of analysts surveyed by LSEG, is a super-low 0.58. I don't think Vertex's pipeline disappointments will change the fact that this biotech stock remains attractively valued with its growth prospects factored into the equation. Do the experts think Vertex Pharmaceuticals is a buy right now? The Motley Fool's expert analyst team, drawing on years of investing experience and deep analysis of thousands of stocks, leverages our proprietary Moneyball AI investing database to uncover top opportunities. They've just revealed their to buy now — did Vertex Pharmaceuticals make the list? When our Stock Advisor analyst team has a stock recommendation, it can pay to listen. After all, Stock Advisor's total average return is up 1,039% vs. just 181% for the S&P — that is beating the market by 858.19%!* Imagine if you were a Stock Advisor member when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $631,505!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,103,313!* The 10 stocks that made the cut could produce monster returns in the coming years. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of August 4, 2025 Keith Speights has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. 7 Reasons Why Vertex Pharmaceuticals Is a No-Brainer Stock to Buy on the Dip was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
05-08-2025
- Business
- Yahoo
VRTX: Vertex Shares Tank After Pain Drug Flop in Key Trial
Aug 5 - Vertex Pharmaceuticals (NASDAQ:VRTX) shares dropped nearly 17% on Tuesday after its experimental pain medication, VX-993, didn't hit the mark in a key Phase 2 trial. The drug aimed to treat acute post-surgical pain, but results showed no statistically significant improvement over placebo (p=0.119), even though the highest dose group showed a better average pain score (74.5 vs. 50.2). The company confirmed it won't move forward with VX-993 as a standalone treatment, citing no clear advantage over existing NaV1.8 inhibitors. Warning! GuruFocus has detected 6 Warning Signs with VRTX. Despite the clinical setback, Vertex delivered a solid Q2 performance. The company reaffirmed its full-year revenue guidance, which gave investors a bit of reassurance. However, the market reaction largely focused on the pipeline disappointment, as investors had high hopes for the non-opioid pain therapy space, an area with major unmet demand. The Pursuit of NaV1.8 The NaV1.8 target continues to be a competitive priority of a number of biotechnology companies; the failure of VX-993 highlights the possibility that Vertex may now have to reconsider its game plan regarding its pain pipeline or consider combination strategies. This article first appeared on GuruFocus.
Boston Globe
04-08-2025
- Business
- Boston Globe
Vertex next-generation pain candidate fails Phase 2 trial
'We do not plan to advance VX-993 as monotherapy in acute pain, because we do not expect that it will be superior to our [existing] NaV1.8 inhibitors,' said CEO Reshma Kewalramani during a Monday afternoon earnings call with investors, using a scientific shorthand for the class of drugs. She noted that the company will continue a trial testing the drug in patients with diabetes who have chronic nerve pain. It was one of multiple setbacks the company disclosed for its pain franchise Monday, as it announced its second-quarter earnings. Advertisement Vertex also announced that longtime chief scientist David Altshuler was retiring next year, to be replaced by head of global research Mark Bunnage. Altshuler, an accomplished academic geneticist, took the post in 2015 and presided over the launch of its most important Advertisement Although Journavx was On Monday, though, the company disclosed that the the Food and Drug Administration 'indicated they do not see a path' for Vertex to obtain a broad approval for Journavx in 'peripheral neuropathic pain' — i.e., chronic pain caused by nerve damage or dysfunction. Instead the company will have to obtain approval in individual subsets of chronic pain. Accordingly, Vertex said it will cancel a planned Phase 3 trial in lumbosacral radiculopathy, also known as sciatica, which the company said affects about 4 million people. Journavx had already failed to outperform placebo in a Phase 2 sciatica trial. Instead it will launch a second Phase 3 trial in diabetic peripheral neuropathy, which affects about 2 million people, in the hope of first getting approval in that indication. At the same time, Vertex announced better-than-expected sales for Journavx in acute pain, with more than 110,000 prescriptions filled. Although the pill was hailed as a potential public health breakthrough for providing a non-addictive alternative to opioids, analysts questioned how widely they would be prescribed, given the availability of cheap alternatives. On Monday, Vertex said it earned $12 million from Journavx, compared to consensus Wall Street projections of $6.7 million. Advertisement Vertex also said it has now treated 29 patients with Casgevy, its gene editing therapy for sickle cell disease, including 16 in the last quarter. Casgevy, which has been slow to launch, earned $30.4 million from second-quarter sales.
Yahoo
04-08-2025
- Business
- Yahoo
Vertex Announces Results from Phase 2 Study of VX-993 for the Treatment of Acute Pain
– Treatment with the selective NaV1.8 pain signal inhibitor VX-993 after bunionectomy surgery did not meet the primary endpoint – – Treatment with VX-993 was generally safe and well tolerated, with safety profile similar to placebo arm – BOSTON, August 04, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced topline results from its recently completed Phase 2, randomized, double-blind, placebo-controlled dose-ranging study evaluating the safety and efficacy of its investigational selective NaV1.8 pain signal inhibitor, VX-993, in treating acute pain after bunionectomy surgery. Treatment with VX-993 did not result in a statistically significant improvement on the primary endpoint of the time-weighted Sum of the Pain Intensity Difference from 0 to 48 hours (SPID48) compared to placebo. VX-993 was generally safe and well tolerated. Most adverse events (AEs) were mild to moderate, and there were no serious adverse events (SAEs) related to VX-993. Based on these results, Vertex will not progress VX-993 into pivotal development as monotherapy in acute pain. "This proof-of-concept study was powered to test whether VX-993 would result in higher clinical efficacy than previously demonstrated with the NaV1.8 pathway," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "Based on these results, as well as the totality of preclinical data and results from our previous bunionectomy clinical studies, VX-993 is not expected to be superior to our existing NaV1.8 inhibitors and therefore we will not be advancing it as monotherapy in acute pain." Primary Efficacy Outcomes Chart Treatment Groups Placebo n = 71 High-dose VX-993 (180 mg first dose/90 mg every 12 hours) n = 71 Mid-dose VX-993 (70 mg first dose/35 mg every 12 hours) n = 77 Low-dose VX-993 (10 mg first dose/5 mg every 12 hours) n = 73 Hydrocodone bitartrate /acetaminophen reference arm (5 mg/325 mg every 6 hours) n = 75 Mean SPID48 50.2 74.5 71.5 54.0 94.4 Mean SPID48 difference from placebo(95% CI) P value vs. placebo -- 24.3 (-6.3, 54.9) 0.1190 21.2 (-8.7, 51.2) 0.1643 3.7 (-26.7,34.1) 0.8094 44.2 (14.0, 74.4) 0.0043 367 patients were enrolled in the studyAll p-values are based on individual comparisons to placeboCI: confidence interval Safety Results VX-993 was generally safe and well tolerated at all doses studied in the trial. The overall incidence of adverse events on VX-993 was similar to placebo. The majority of the AEs were mild or moderate in severity. There were no SAEs related to VX-993 in the study. No patients treated with VX-993 discontinued study drug due to AEs. The most common AEs (incidence >5% in either combined VX-993, hydrocodone bitartrate/acetaminophen (HB/APAP) or placebo group, respectively) were nausea (4.1%, 14.7%, 11.3%), headache (2.7%, 6.7%, 1.4%), dizziness (1.4%, 5.3%, 1.4%) and vomiting (1.4%, 5.3%, 2.8%). Adverse events were generally consistent with the post-surgical setting. About the VX-993 Phase 2 Acute Pain Study The Phase 2 study was a randomized, double-blind, placebo-controlled, dose-ranging study that evaluated three different doses of VX-993 administered orally in 367 patients with acute pain following bunionectomy surgery. The study also included a hydrocodone bitartrate/acetaminophen (HB/APAP) reference arm. The primary endpoint was the time-weighted Sum of the Pain Intensity Difference (SPID) over the first 48 hours of treatment, as recorded on the 11-point Numeric Pain Rating Scale (NPRS), compared to placebo. The study was designed to test whether greater NaV1.8 inhibition with VX-993 would translate to higher efficacy than what has already been demonstrated with other NaV1.8 inhibitors. The study was powered accordingly to demonstrate a treatment effect higher than previously achieved. Patients were randomized to 5 treatment arms: VX-993 high dose — 180 mg first dose and 90 mg every 12 hours (at 12, 24 and 36 hours after the first dose), VX-993 mid dose — 70 mg first dose and 35 mg every 12 hours (at 12, 24 and 36 hours after the first dose), or VX-993 low dose — 10 mg first dose and 5 mg every 12 hours (at 12, 24 and 36 hours after the first dose), the reference arm of HB/APAP 5 mg/325 mg administered orally every 6 hours over 42 hours, or placebo. Patients reported their pain intensity on the NPRS at each scheduled time point through 48 hours. The first dose of study drug was administered on the day of surgery, approximately 3 hours post-operatively on average. In order to maximize pain severity, a popliteal block was not used in this study. VX-993 is investigational and has not been approved by health authorities globally. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Carmen Bozic, M.D., in this press release, and statements regarding Vertex's expectations for VX-993 as a treatment for acute pain, and the company's plans not to progress VX-993 as a monotherapy in acute pain. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's clinical programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@ Media: mediainfo@ orInternational: +44 20 3204 5275orU.S.: 617-341-6992 Sign in to access your portfolio
Business Wire
04-08-2025
- Business
- Business Wire
Vertex Announces Results from Phase 2 Study of VX-993 for the Treatment of Acute Pain
BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced topline results from its recently completed Phase 2, randomized, double-blind, placebo-controlled dose-ranging study evaluating the safety and efficacy of its investigational selective NaV1.8 pain signal inhibitor, VX-993, in treating acute pain after bunionectomy surgery. Treatment with VX-993 did not result in a statistically significant improvement on the primary endpoint of the time-weighted Sum of the Pain Intensity Difference from 0 to 48 hours (SPID48) compared to placebo. VX-993 was generally safe and well tolerated. Most adverse events (AEs) were mild to moderate, and there were no serious adverse events (SAEs) related to VX-993. Based on these results, Vertex will not progress VX-993 into pivotal development as monotherapy in acute pain. 'This proof-of-concept study was powered to test whether VX-993 would result in higher clinical efficacy than previously demonstrated with the NaV1.8 pathway,' said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. 'Based on these results, as well as the totality of preclinical data and results from our previous bunionectomy clinical studies, VX-993 is not expected to be superior to our existing NaV1.8 inhibitors and therefore we will not be advancing it as monotherapy in acute pain.' Primary Efficacy Outcomes Chart 367 patients were enrolled in the study All p-values are based on individual comparisons to placebo CI: confidence interval Safety Results VX-993 was generally safe and well tolerated at all doses studied in the trial. The overall incidence of adverse events on VX-993 was similar to placebo. The majority of the AEs were mild or moderate in severity. There were no SAEs related to VX-993 in the study. No patients treated with VX-993 discontinued study drug due to AEs. The most common AEs (incidence >5% in either combined VX-993, hydrocodone bitartrate/acetaminophen (HB/APAP) or placebo group, respectively) were nausea (4.1%, 14.7%, 11.3%), headache (2.7%, 6.7%, 1.4%), dizziness (1.4%, 5.3%, 1.4%) and vomiting (1.4%, 5.3%, 2.8%). Adverse events were generally consistent with the post-surgical setting. About the VX-993 Phase 2 Acute Pain Study The Phase 2 study was a randomized, double-blind, placebo-controlled, dose-ranging study that evaluated three different doses of VX-993 administered orally in 367 patients with acute pain following bunionectomy surgery. The study also included a hydrocodone bitartrate/acetaminophen (HB/APAP) reference arm. The primary endpoint was the time-weighted Sum of the Pain Intensity Difference (SPID) over the first 48 hours of treatment, as recorded on the 11-point Numeric Pain Rating Scale (NPRS), compared to placebo. The study was designed to test whether greater NaV1.8 inhibition with VX-993 would translate to higher efficacy than what has already been demonstrated with other NaV1.8 inhibitors. The study was powered accordingly to demonstrate a treatment effect higher than previously achieved. Patients were randomized to 5 treatment arms: VX-993 high dose — 180 mg first dose and 90 mg every 12 hours (at 12, 24 and 36 hours after the first dose), VX-993 mid dose — 70 mg first dose and 35 mg every 12 hours (at 12, 24 and 36 hours after the first dose), or VX-993 low dose — 10 mg first dose and 5 mg every 12 hours (at 12, 24 and 36 hours after the first dose), the reference arm of HB/APAP 5 mg/325 mg administered orally every 6 hours over 42 hours, or placebo. Patients reported their pain intensity on the NPRS at each scheduled time point through 48 hours. The first dose of study drug was administered on the day of surgery, approximately 3 hours post-operatively on average. In order to maximize pain severity, a popliteal block was not used in this study. VX-993 is investigational and has not been approved by health authorities globally. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Carmen Bozic, M.D., in this press release, and statements regarding Vertex's expectations for VX-993 as a treatment for acute pain, and the company's plans not to progress VX-993 as a monotherapy in acute pain. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's clinical programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading 'Risk Factors' in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)
