Latest news with #VertexPharmaceuticals
Globe and Mail
14 hours ago
- Business
- Globe and Mail
Cystic Fibrosis Pipeline Outlook 2025: Insights into Drug Development and Leading 50+ Companies
DelveInsight's, ' Cystic fibrosis Pipeline Insight, 2025 ' report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Cystic fibrosis pipeline landscape. It covers the Cystic Fibrosis pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Cystic Fibrosis pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Stay ahead with the latest insights! Download DelveInsight's comprehensive Cystic Fibrosis Pipeline Report to explore emerging therapies, key players, and future treatment landscapes @ Cystic Fibrosis Pipeline Outlook Report Key Takeaways from the Cystic Fibrosis Pipeline Report In July 2025, Enterprise Therapeutics Ltd announced a study is the first to give ETD001 to people with CF. The study will be run in two parts. Part A will assess if ETD001 is safe to give to people with CF, and Part B will assess if ETD001 improves lung function. The study drug is taken twice a day, in Part A it is taken for 7 days and in Part B for 28 days. In Part B there will be a separate period where dummy medicine is given for 28 days so the treatments can be compared. In July 2025, Vertex Pharmaceuticals Incorporated conducted a study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. DelveInsight's Cystic Fibrosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for Cystic Fibrosis treatment. The leading Cystic Fibrosis Companies such as Verona Pharma, Armata Pharmaceuticals, 4D Molecular Therapeutics, Spirovant Sciences, Vertex Pharmaceuticals, Krystal Biotech, Porosome Therapeutics, SpliSense, Enterprise Therapeutics Ltd and others. Promising Cystic fibrosis Pipeline Therapies such as Tigerase®, Pulmozyme®, danazol, Brensocatib, SPL84, ETD001, ELX/TEZ/IVA, BI 3720931, JBT-101 (lenabasum) and others. Discover how the Cystic Fibrosis treatment paradigm is evolving. Access DelveInsight's in-depth Cystic Fibrosis Pipeline Analysis for a closer look at promising breakthroughs @ Cystic Fibrosis Clinical Trials and Studies Cystic Fibrosis Emerging Drugs Profile VX-121: Vertex Pharmaceuticals VX-121 and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the CFTR protein. VX-561 (deutivacaftor) is a potentiator designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. The triple combination of VX-121/tezacaftor/VX-561 is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene. It is currently being investigated in Phase III stage of development. Brensocatib: AstraZeneca Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. The drug is currently being evaluated in Phase II clinical trial to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug. ELX-02: Eloxx Pharmaceuticals ELX-02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. Structurally, ELX-02 is an aminoglycoside analogue that induces read-through of nonsense mutations through interaction with the ribosome, resulting in the production of full-length functional proteins. The therapy was designated an orphan drug in the US, an orphan medicinal product in Europe, and given fast track designation in the US. ELX-02 is being tested in CF patients carrying least one G542X mutation in two parallel open-label Phase II clinical trials. S-1226: SolAeroMed Inc. S1226 is SolAeroMed's lead therapy. S1226 is formulated to rapidly reopen constricted, mucus plugged airways, and should increase the effectiveness of respiratory drug delivery. The S1226 formulation consists of aerosolized carbon dioxide (CO2) and nebulized perflubron; which is delivered into the lung. The delivery of this formulation results in an immediate relaxant effect on the patient's constricted airways, supported by a lowering of surface tension in inflamed areas (resulting in enhanced bronchial dilation) and possible clearing of mucus plugs of blocked airways. SolAeroMed is currently conducrting a phase II clinical trial in cystic fibrosis. The Cystic Fibrosis Pipeline Report Provides Insights into The report provides detailed insights about companies that are developing therapies for the treatment of Cystic Fibrosis with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Cystic Fibrosis Treatment. Cystic Fibrosis Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Cystic Fibrosis Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Cystic Fibrosis market Explore groundbreaking therapies and clinical trials in the Cystic Fibrosis Pipeline. Access DelveInsight's detailed report now! @ New Cystic Fibrosis Drugs Cystic Fibrosis Companies Verona Pharma, Armata Pharmaceuticals, 4D Molecular Therapeutics, Spirovant Sciences, Vertex Pharmaceuticals, Krystal Biotech, Porosome Therapeutics, SpliSense, Enterprise Therapeutics Ltd and others. Cystic fibrosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Intravenous Subcutaneous Oral Intramuscular Cystic Fibrosis Products have been categorized under various Molecule types such as Monoclonal antibody Small molecule Peptide Download DelveInsight's latest report to gain strategic insights into upcoming therapies and key developments @ Cystic Fibrosis Market Drivers and Barriers, and Future Perspectives Scope of the Cystic Fibrosis Pipeline Report Coverage- Global Cystic Fibrosis Companies- Verona Pharma, Armata Pharmaceuticals, 4D Molecular Therapeutics, Spirovant Sciences, Vertex Pharmaceuticals, Krystal Biotech, Porosome Therapeutics, SpliSense, Enterprise Therapeutics Ltd and others. Cystic fibrosis Pipeline Therapies- Tigerase®, Pulmozyme®, danazol, Brensocatib, SPL84, ETD001, ELX/TEZ/IVA, BI 3720931, JBT-101 (lenabasum) and others. Cystic Fibrosis Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Cystic Fibrosis Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Which companies are leading the race in Cystic Fibrosis drug development? Find out in DelveInsight's exclusive Cystic Fibrosis Pipeline Report—access it now! @ Cystic Fibrosis Emerging Drugs and Major Companies Table of Content Introduction Executive Summary Cystic Fibrosis: Overview Pipeline Therapeutics Therapeutic Assessment Cystic Fibrosis – DelveInsight's Analytical Perspective Late Stage Products (Phase III) Drug name: Company Name Drug profiles in the detailed report….. Mid-Stage Products (Phase II) AP-PA02: Armata Pharmaceuticals Drug profiles in the detailed report….. Early Stage Products (Phase I) VX-522: Vertex Pharmaceuticals Drug profiles in the detailed report….. Preclinical and Discovery Stage Products SPL23: SpliSense Drug profiles in the detailed report….. Inactive Products Cystic Fibrosis -Key Companies Cystic Fibrosis -Key Products Cystic Fibrosis - Unmet Needs Cystic Fibrosis - Market Drivers and Barriers Cystic Fibrosis - Future Perspectives and Conclusion Cystic Fibrosis -Analyst Views Cystic Fibrosis- Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 09650213330 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
Malaysian Reserve
21 hours ago
- Health
- Malaysian Reserve
Vertex Announces Marketing Authorization in Canada for ALYFTREK, a Once-Daily Next-Generation CFTR Modulator for the Treatment of Cystic Fibrosis
– In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV1 and superior in reducing sweat chloride compared to TRIKAFTA – – Approximately 3,800 people in Canada are now eligible for ALYFTREK, with up to 60 people potentially eligible for a medicine that treats the underlying cause of their disease for the first time – TORONTO, July 22, 2025 /CNW/ – Vertex Pharmaceuticals today announced that Health Canada has granted Marketing Authorization for PrALYFTREKTM (vanzacaftor/tezacaftor/deutivacaftor), a new triple combination therapy for patients living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. ALYFTREK brings a potentially transformative therapy to up to 60 people living with CF in Canada who were not previously eligible for a CFTR modulator. As the first once-daily CFTR modulator, ALYFTREK may also provide a new treatment option and flexibility for approximately 3,800 people given the need to take CFTR modulators with fat-containing food. 'ALYFTREK represents the next generation of CFTR modulator treatment and is a testament to our long-standing goal to address the underlying cause of cystic fibrosis, treat more people living with CF, and bring more people to normal levels of CFTR function,' said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. 'With once-daily dosing, eligibility in 113 additional mutations, and the potential to lower sweat chloride levels even further, ALYFTREK brings us one step closer to achieving this goal.' This approval is based on a comprehensive Phase 3 pivotal program, including more than 1,000 patients across more than 20 countries and more than 200 sites. The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV1 compared to TRIKAFTA) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to TRIKAFTA). In the Phase 3 study of children with CF ages 6-11 years, ALYFTREK demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV1 and absolute change from baseline in SwCl, demonstrate the benefit of ALYFTREK in this age group. ALYFTREK was generally well tolerated across all studies. 'For Canadian patients and families, the approval of ALYFTREK represents significant progress towards improved care,' said Dr. Bradley Quon, Medical and Research Director of the Adult Cystic Fibrosis Program at St. Paul's Hospital, and Associate Professor of Medicine in the Faculty of Medicine at the University of British Columbia. 'Lower levels of sweat chloride combined with the convenience of a once-a-day treatment provides a new option that has the potential to both improve CFTR function and reduce treatment burden.' ALYFTREK is currently approved in the U.S., UK and European Union and is under regulatory review in Switzerland, Australia and New Zealand. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Today Vertex CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. Sweat chloride, which measures CFTR function, is used to diagnose CF. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease. About ALYFTREKTM (vanzacaftor/tezacaftor/deutivacaftor) In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane. ALYFTREK is approved in the U.S., UK, and Canada for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. ALYFTREK is approved in the European Union for the treatment of CF in patients ages 6 years and older who have at least one non-class I mutation in the CFTR gene. Boxed Warning Elevated transaminases have been observed in some patients treated with ALYFTREK. Cases of liver failure leading to transplantation and death have been reported in patients with and without a history of liver disease taking a fixed dose combination drug containing elexacaftor, tezacaftor, and ivacaftor, which contains one same (tezacaftor) and one similar (ivacaftor) active ingredient as ALYFTREK. Liver injury has primarily been reported within the first 6 months following initiation of elexacaftor/tezacaftor/ivacaftor. See full ALYFTREK Product Monograph for further details. 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 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 Michael Siauw and Dr. Bradley Quon, and statements regarding the anticipated eligible patient population in Canada and expectations for the ALYFTREK regulatory submissions in Switzerland, Australia and New Zealand. 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 development 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) Vertex Pharmaceuticals IncorporatedInvestors:InvestorInfo@ Media: mediainfo@ +1 647-790-1600
Cision Canada
a day ago
- Health
- Cision Canada
Vertex Announces Marketing Authorization in Canada for ALYFTREK, a Once-Daily Next-Generation CFTR Modulator for the Treatment of Cystic Fibrosis
- Approximately 3,800 people in Canada are now eligible for ALYFTREK, with up to 60 people potentially eligible for a medicine that treats the underlying cause of their disease for the first time - TORONTO, July 22, 2025 /CNW/ - Vertex Pharmaceuticals today announced that Health Canada has granted Marketing Authorization for Pr ALYFTREK TM (vanzacaftor/tezacaftor/deutivacaftor), a new triple combination therapy for patients living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. ALYFTREK brings a potentially transformative therapy to up to 60 people living with CF in Canada who were not previously eligible for a CFTR modulator. As the first once-daily CFTR modulator, ALYFTREK may also provide a new treatment option and flexibility for approximately 3,800 people given the need to take CFTR modulators with fat-containing food. "ALYFTREK represents the next generation of CFTR modulator treatment and is a testament to our long-standing goal to address the underlying cause of cystic fibrosis, treat more people living with CF, and bring more people to normal levels of CFTR function," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "With once-daily dosing, eligibility in 113 additional mutations, and the potential to lower sweat chloride levels even further, ALYFTREK brings us one step closer to achieving this goal." This approval is based on a comprehensive Phase 3 pivotal program, including more than 1,000 patients across more than 20 countries and more than 200 sites. The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV 1 compared to TRIKAFTA) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to TRIKAFTA). In the Phase 3 study of children with CF ages 6-11 years, ALYFTREK demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV 1 and absolute change from baseline in SwCl, demonstrate the benefit of ALYFTREK in this age group. ALYFTREK was generally well tolerated across all studies. "For Canadian patients and families, the approval of ALYFTREK represents significant progress towards improved care," said Dr. Bradley Quon, Medical and Research Director of the Adult Cystic Fibrosis Program at St. Paul's Hospital, and Associate Professor of Medicine in the Faculty of Medicine at the University of British Columbia. "Lower levels of sweat chloride combined with the convenience of a once-a-day treatment provides a new option that has the potential to both improve CFTR function and reduce treatment burden." ALYFTREK is currently approved in the U.S., UK and European Union and is under regulatory review in Switzerland, Australia and New Zealand. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Today Vertex CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. Sweat chloride, which measures CFTR function, is used to diagnose CF. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease. About ALYFTREK TM (vanzacaftor/tezacaftor/deutivacaftor) In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane. ALYFTREK is approved in the U.S., UK, and Canada for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. ALYFTREK is approved in the European Union for the treatment of CF in patients ages 6 years and older who have at least one non-class I mutation in the CFTR gene. Boxed Warning Elevated transaminases have been observed in some patients treated with ALYFTREK. Cases of liver failure leading to transplantation and death have been reported in patients with and without a history of liver disease taking a fixed dose combination drug containing elexacaftor, tezacaftor, and ivacaftor, which contains one same (tezacaftor) and one similar (ivacaftor) active ingredient as ALYFTREK. Liver injury has primarily been reported within the first 6 months following initiation of elexacaftor/tezacaftor/ivacaftor. See full ALYFTREK Product Monograph for further details. 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 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 Michael Siauw and Dr. Bradley Quon, and statements regarding the anticipated eligible patient population in Canada and expectations for the ALYFTREK regulatory submissions in Switzerland, Australia and New Zealand. 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 development 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) Vertex Pharmaceuticals Incorporated Investors: [email protected] Media: [email protected] or Canada: +1 647-790-1600 SOURCE Vertex Pharmaceuticals (Canada) Inc.
Yahoo
a day ago
- Business
- Yahoo
Vertex Pharmaceuticals' Quarterly Earnings Preview: What You Need to Know
Valued at a market cap of $117.7 billion, Vertex Pharmaceuticals Incorporated (VRTX) is a biotechnology company that discovers, develops, and commercializes innovative therapies for severe diseases. While the Boston, Massachusetts-based company is best known for its groundbreaking treatments for cystic fibrosis, it is also advancing a robust pipeline targeting conditions such as sickle cell disease, beta thalassemia, type 1 diabetes, alpha-1 antitrypsin deficiency, and muscular dystrophies. It is scheduled to announce its fiscal Q2 earnings for 2025 after the market closes on Monday, Aug. 4. Before this event, analysts project this healthcare giant to report a profit of $3.74 per share, up 128.2% from a loss of $13.27 per share in the year-ago quarter. The company has surpassed Wall Street's bottom-line estimates in two of the last four quarters, while missing on two other occasions. Its earnings of $3.55 per share in the previous quarter fell short of the consensus estimates by 3.8%. More News from Barchart Opendoor Stock Is Surging Higher in a Frenzied Retail Rally. How Should You Play OPEN Shares Here? This Penny Stock Wants to Become the MicroStrategy of Dogecoin Robinhood Stock Stumbles as S&P 500 Inclusion Is Once Again Off the Table for HOOD Tired of missing midday reversals? The FREE Barchart Brief newsletter keeps you in the know. Sign up now! For the full year, analysts expect VRTX to report EPS of $15.67, up significantly from a loss of $1.35 per share in fiscal 2024. Its EPS is expected to further grow 12.4% year-over-year to $17.61 in fiscal 2026. VRTX has lagged behind the S&P 500 Index's ($SPX) 14.5% uptick over the past 52 weeks, with its shares down 6.7% over the same time frame. However, it has outpaced the Health Care Select Sector SPDR Fund's (XLV) 11.4% drop over the same period. On May 5, VRTX delivered weaker-than-expected Q1 results, prompting its share price to tumble 10% in the following trading session. The company's revenue grew 3% year-over-year to $2.8 billion, but missed the consensus estimates by 1.8% due to a decline in revenue from its international markets, primarily driven by headwind in Russia, where Vertex is experiencing a violation of its intellectual property rights. Moreover, due to higher operating expenses, its adjusted EPS of $4.06 fell 14.7% from the year-ago quarter, falling short of analyst estimates by 3.8%. Wall Street analysts are moderately optimistic about VRTX's stock, with an overall "Moderate Buy" rating. Among 32 analysts covering the stock, 16 recommend "Strong Buy," one indicates a "Moderate Buy,' 14 suggest "Hold,' and one advises a 'Strong Sell' rating. The mean price target for VRTX is $514.73, implying a 12.3% potential upside from the current levels. On the date of publication, Neharika Jain did not have (either directly or indirectly) positions in any of the securities mentioned in this article. All information and data in this article is solely for informational purposes. This article was originally published on
Globe and Mail
a day ago
- Business
- Globe and Mail
3 Healthcare Stocks to Buy Before They Announce Game-Changing Clinical Trial Results
Key Points Eli Lilly, Summit Therapeutics, and Vertex Pharmaceuticals all have important ongoing clinical trials. Shares of these drugmakers could jump if the data from the studies is positive. All three companies have attractive prospects beyond that. 10 stocks we like better than Eli Lilly › Few things can jolt a pharmaceutical company's shares like positive results from highly anticipated clinical trials. Even so, long-term investors should have a balanced approach to potentially positive data readouts. Putting aside the fact that it's impossible to predict the outcome of a clinical trial beforehand, even when the results are up to Wall Street's standards, it's hardly worth investing in the company unless there are other good reasons to think it will perform well over long periods. That's why you may want to take a closer look at Eli Lilly (NYSE: LLY), Summit Therapeutics (NASDAQ: SMMT), and Vertex Pharmaceuticals (NASDAQ: VRTX). All three drugmakers should release results from key clinical trials within the next 18 months, and all three have strong prospects for the next five years at least. 1. Eli Lilly In April, Eli Lilly reported positive phase 3 results for its oral GLP-1 candidate, orforglipron. While the market reacted positively to this development, the study in question focused on diabetes patients and used A1c reduction as its primary endpoint. All eyes will be on the company's ongoing late-stage studies for orforglipron in obesity; it should release data from at least one of those clinical trials within the next year. Eli Lilly's work in weight management has taken center stage in the past few years. The pharmaceutical leader could, once again, make a breakthrough by being one of the first to launch a highly effective oral GLP-1 anti-obesity drug. Since current options are administered subcutaneously, you can expect orforglipron to reach a reasonable level of success on the market -- but that's only if it performs well in phase 3 obesity studies. If it fails to do so, Lilly's shares could plunge. Even so, the stock should still be a buy. True, orforglipron would strengthen Lilly's already robust lineup. But even if it falls short of expectations in late-stage studies, the company has several other candidates in development, including retatrutide, which is also in phase 3 trials. Meanwhile, Lilly continues to generate consistent financial results. Revenue and earnings have been growing at a good clip, and that should continue for the foreseeable future. Lastly, Eli Lilly is an excellent dividend growth stock. Although results from phase 3 trials for orforglipron in obesity will be important to monitor, the stock should perform well over the long run, regardless of the outcome of these trials. 2. Summit Therapeutics Summit Therapeutics is developing ivonescimab, a cancer medicine it licensed from Akeso, a China-based biopharma. Ivonescimab is already approved in China; however, Summit needs to conduct clinical trials elsewhere to support approval in the U.S., Europe, and other regions where it holds marketing rights. At least one of these studies will be of particular interest to investors. Summit is testing its crown jewel in a late-stage trial against Merck 's Keytruda in patients with non-small cell lung cancer (NSCLC), in a study called Harmoni-3. Enrollment in the study is ongoing, and there is a good chance we'll see top-line data from it by the end of next year. Summit's stock soared when it reported that ivonescimab did better than Keytruda at reducing the risk of recurrence or death in NSCLC patients, in a study conducted in China. However, reproducing this result elsewhere could, once again, jolt the stock price. On the flip side, Summit's share price will move in the wrong direction if the results aren't what Wall Street expects. Should you buy the stock? To me, Summit Therapeutics' prospects for the next five years seem attractive. Ivonescimab is being tested across a range of different cancers, and it looks highly promising in NSCLC, which could be its most important market. Even with the possibility that it may not perform as well in studies outside of China, having already been approved by regulatory authorities in one region reduces the risk of significant clinical and regulatory setbacks. Ivonescimab looks like a potential pipeline in a drug. And if you invest in Summit Therapeutics now, you might reap the benefits of its leading candidate's potential. 3. Vertex Pharmaceuticals Vertex Pharmaceuticals is a proven innovator. The company's strategy is to develop breakthrough medicines where there's a high unmet need. One of Vertex's new targets is type 1 diabetes (T1D). There are no cures for this chronic disease, but the biotech's candidate zimislecel could be a functional cure, in the sense that it could recover patients' ability to produce their own insulin -- something that people with T1D can't do. In the phase 1/2 portion of an ongoing phase 1/2/3 clinical trial, 10 of 12 patients who received zimislecel were insulin-free after a one-year follow-up. All 12 were free of severe hypoglycemic events after 90 days. Vertex should have data from the late-stage portion of this ongoing clinical trial within the next year. Positive results will be well received. The company also aims to submit regulatory applications for this product sometime in 2026. Zimislecel should be a nice addition to Vertex's portfolio. The biotech remains the leader in the market for drugs that treat the underlying genetic causes of cystic fibrosis, a business that is still helping it drive strong revenue and earnings growth. Furthermore, Vertex has added several medicines to its portfolio over the past five years, including Journavx for acute pain and Casgevy for two rare blood-related disorders. Vertex Pharmaceuticals' pipeline also has promising programs beyond zimislecel. Well beyond the announcement of clinical-trial results for this promising T1D therapy, the stock will be in an excellent position to perform. Should you invest $1,000 in Eli Lilly right now? Before you buy stock in Eli Lilly, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Eli Lilly wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. 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