Latest news with #clinicaltrial
Yahoo
2 hours ago
- Health
- Yahoo
Qu Biologics Completes Enrolment of a Unique Study Testing a Novel Immunomodulator to Restore Immune Function in the Elderly
VANCOUVER, British Columbia, July 23, 2025 (GLOBE NEWSWIRE) -- Qu Biologics Inc., a clinical stage biopharmaceutical company developing Site Specific Immunomodulators (SSIs), a novel immunomodulation platform designed to restore innate immune function, is excited to report that they have completed enrolment of all 72 participants in the RESILIENCE Study, an innovative randomized placebo-controlled Phase 2 clinical trial evaluating the ability of Qu's first-in-class immunomodulator, QBKPN SSI, to restore innate immunity in people 65 years of age and older. QBKPN SSI is designed to restore and enhance innate immune function, the body's first line of defense against infections and other diseases such as cancer. As we age, our innate immune function declines, leaving us vulnerable to not only infectious threats, as was seen during the COVID-19 pandemic, but also to the development of chronic inflammatory diseases, metabolic disease, and cancer. The RESILIENCE Study is assessing if QBKPN treatment can restore innate immune function in the elderly, with important implications for improving response to immunological threats, including cancer and other chronic diseases, and improving all-cause mortality. Dr. Hal Gunn, CEO of Qu Biologics, stated, 'Completing enrolment of a clinical study is an exciting milestone, particularly in this case, since there are no current treatments that prevent the decline in immune function that occurs with aging. This age-related immune decline is the largest unmet need in healthcare since it increases the risk of pre-mature aging, infections, inflammatory diseases, and cancer. Positive results from our RESILIENCE Study would have very important implications for extending healthy longevity.' Immunologist Dr. Shirin Kalyan, Qu's VP of Scientific Innovation, stated, 'We have previously demonstrated in proof-of-concept studies that QBKPN treatment can reverse immune dysregulation that occurs with aging, inflammatory diseases, and cancer to improve health outcomes. Qu's SSIs are designed, not just to treat disease, but to restore health and clear important underlying causes of disease – a fundamentally different way of thinking about the treatment of disease and what medicines could be.' In advance of the results from the RESILENCE Study, Qu Biologics is completing a USD $2M bridge round and is reaching out to Pharma partners for co-development. Interested parties are welcome to contact Dr. Hal Gunn, CEO, at For more information about Qu Biologics and the science behind SSIs, please visit About Qu BiologicsQu Biologics is a clinical stage biotechnology company developing Site Specific Immunomodulators (SSI), a novel class of immunomodulators designed to restore innate immune function in targeted organs to reverse the immune dysregulation underlying many important diseases including cancer, chronic inflammatory diseases, metabolic disease, and infection. Qu Biologics has completed four Phase 2 studies in lung cancer, Crohn's disease, and ulcerative colitis. Two additional Phase 2 randomized placebo-controlled studies, in late-stage colon cancer and immunosenescence, are underway. Backed by a prestigious group of scientific advisors and board members, Qu Biologics is led by a management team that includes co-founder and CEO Dr. Hal Gunn, a physician and expert on the body's immune response to chronic disease; Chief Medical Officer - Oncology Dr. Simon Sutcliffe, former CEO of the BC Cancer Agency and a distinguished clinician, scientist and leader in cancer control in Canada and internationally; and Chief Medical Officer – Infectious Disease Dr. Ted Steiner, a clinician-scientist specializing in immune responses to infections and Head of the University of British Columbia Division of Infectious Diseases. For more information, please visit For more information regarding this press release, contact: Hal Gunn, MDCEOQu Biologics 604.734.1450 Email: media@ Qu Biologics Inc. cautions you that statements included in this press release that are not a description of historical facts may be forward-looking statements. Forward-looking statements are only predictions based upon current expectations and involve known and unknown risks and uncertainties. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of release of the relevant information, unless explicitly stated otherwise. Actual results, performance or achievement could differ materially from those expressed in, or implied by, Qu Biologics' forward-looking statements due to the risks and uncertainties inherent in Qu Biologics' business including, without limitation, statements about: the progress and timing of its clinical trials; difficulties or delays in development, testing, obtaining regulatory approval, producing and marketing its products; unexpected adverse side effects or inadequate therapeutic efficacy of its products that could delay or prevent product development or commercialization; the scope and validity of patent protection for its products; competition from other pharmaceutical or biotechnology companies; and its ability to obtain additional financing to support its operations. Qu Biologics does not assume any obligation to update any forward-looking statements except as required by law.
Associated Press
4 hours ago
- Business
- Associated Press
Lantern Pharma Reports Complete Response in Heavily Pre-Treated Lymphoma Patient with LP-284 in Phase 1 Clinical Trial
DALLAS--(BUSINESS WIRE)--Jul 23, 2025-- Lantern Pharma Inc. (NASDAQ: LTRN), a clinical-stage oncology company leveraging its proprietary RADR® artificial intelligence (AI) platform to systematically transform drug discovery paradigms, today announces that a heavily pretreated patient with aggressive Grade 3 non-germinal center B-cell diffuse large B-cell lymphoma (DLBCL) achieved a complete metabolic response in the ongoing Phase 1 clinical trial of LP-284. This represents the first complete response observed with LP-284 and displays profound clinical activity in one of the most therapeutically challenging hematologic cancers. The RADR®-driven, AI approach enables Lantern to rapidly determine mechanisms of action of any cancer focused molecule, identify biomarker-driven subpopulations and optimize combination strategies for the clinical setting. The 41-year-old patient had previously failed three aggressive treatment regimens over 18 months over the last 15 months. These included: standard R-CHOP/Pola-R-CHP chemotherapy, CAR-T cell therapy (liso-cel), and CD3xCD20 bispecific antibody therapy (glofitamab). Following enrollment in April 2025, the patient achieved complete metabolic response with non-avid lesions after completing just two 28-day cycles of LP-284 administered on days one, eight, and 15. This remarkable clinical outcome supports LP-284's synthetic lethal mechanism and is an initial step towards a potential new paradigm for treating refractory aggressive lymphomas and B-cell malignancies. Paradigm-Shifting Observation of Complete Response in Therapeutically Exhausted Patient 'This extraordinary clinical observation represents a transformative moment for our computationally guided, therapeutic development paradigm,' said Panna Sharma, President and CEO of Lantern Pharma. 'Our RADR® platform's systematic analysis of molecular vulnerabilities enabled us to rapidly advance LP-284 from computational concept to a clinical milestone in a patient in under three years at approximately $3 million — demonstrating how AI-driven precision has the promise to fundamentally reshape pharmaceutical innovation. This complete metabolic response in a patient who had exhausted all conventional therapeutic options strongly supports our strategic thesis that computational approaches can unlock previously inaccessible therapeutic opportunities.' LP-284, a computationally optimized next-generation acylfulvene, was systematically developed with guidance from Lantern's RADR® platform to exploit synthetic lethal interactions in cancer cells harboring specific DNA damage repair deficiencies. The compound represents a fundamentally different therapeutic strategy with the potential to preferentially target malignant cells while preserving healthy tissue function — a precision approach aimed at delivering transformative clinical benefit for patients who have failed multiple prior treatment modalities. Potential to Address a Critical Therapeutic Void in Refractory DLBCL This patient's clinical journey exemplifies the devastating trajectory of aggressive DLBCL in the refractory setting. Despite achieving an initial complete metabolic response with CAR-T therapy (liso-cel) at day 30, the patient experienced disease progression by day 90. Subsequent treatment with the CD3xCD20 bispecific antibody glofitamab resulted in progressive disease with multifocal new lesions. At study entry on the LP-284-Phase 1 trial in April 2025, the patient presented with extensive multifocal bony lesions across thoracic and lumbar spine locations and hips — representing an extremely challenging clinical scenario. The achievement of complete metabolic response with non-avid lesions following just two cycles of LP-284 therapy represents a potential future paradigm-shifting alternative for treating therapeutically exhausted DLBCL patients. This encouraging outcome underscores LP-284's potential to address critical gaps in the therapeutic armamentarium for patients who have failed both conventional and cutting-edge immunotherapeutic approaches. Advancing Strategic Clinical Development and Global Expansion The complete metabolic response achievement positions LP-284 for accelerated development pathways and strengthens Lantern's strategic position in the competitive hematologic oncology landscape. This clinical milestone provides important confirmation of the company's systematic approach to therapeutic development and creates additional opportunities for international partnership expansion and collaborative research initiatives. Lantern's ongoing Phase 1 dose-escalation study (NCT06132503) is designed to evaluate LP-284's safety profile, optimal dosing parameters, and preliminary efficacy signals across multiple aggressive lymphoma subtypes. The trial's systematic design enables precise evaluation of LP-284's therapeutic potential across genetically defined patient populations, with a current focus on aggressive NHL subtypes including mantle cell lymphoma and high-grade B-cell lymphomas. LP-284's multiple FDA Orphan Drug Designations position the compound for potential expedited regulatory pathways and enhanced market exclusivity frameworks. Transforming Global Oncology Through Computational Precision This clinical development provides important confirmation of the transformative potential of Lantern's RADR® platform, which leverages over 200 billion oncology-focused data points and 200+ machine learning algorithms to assist in systematically identifying and optimizing therapeutic opportunities. The platform's computational efficiency enabled Lantern to rapidly advance the LP-284's program into the clinic in under three years at a cost of approximately $3 million — representing striking development efficiency compared to traditional pharmaceutical paradigms. The complete metabolic response achievement positions LP-284 to seek a future role within a global blood cancer market focused on B-cell cancer that is estimated at $4 billion annually, with DLBCL representing the largest aggressive lymphoma subtype affecting approximately 200,000 patients globally each year. The critical unmet need in refractory/relapsed settings represents a substantial commercial opportunity for innovative therapeutic approaches that can deliver meaningful clinical benefit to therapeutically exhausted patient populations. Next Steps and Future Development Lantern plans to continue enrollment in the Phase 1 trial while closely monitoring the responding patient and other potential future patients for durability of response and efficacy signals. The company anticipates providing additional clinical updates as the trial progresses and more patients reach evaluable timepoints. The complete response achievement positions LP-284 for potential accelerated development pathways and creates opportunities for strategic partnerships as Lantern advances its synthetic lethal portfolio toward later-stage clinical trials. About LP-284 LP-284 is an investigational next-generation acylfulvene designed to exploit synthetic lethal interactions in cancer cells with DNA damage repair deficiencies. Developed with guidance from Lantern's AI platform RADR®, LP-284 represents a novel therapeutic approach with potential to address critical gaps in the treatment of relapsed or refractory non-Hodgkin's lymphoma and other hematologic malignancies. The compound is currently being evaluated in a Phase 1 clinical trial (NCT06132503) to determine its safety profile, optimal dosing, and potential activity in patients with aggressive NHL subtypes who have failed standard therapies. LP-284 has received multiple Orphan Drug Designations from the U.S. Food and Drug Administration, including designations for mantle cell lymphoma and high-grade B-cell lymphomas, recognizing its potential to address significant unmet medical needs in rare cancer populations. About Lantern Pharma Lantern Pharma (NASDAQ: LTRN) is an AI-driven biotechnology company focused on accelerating and optimizing the discovery, development, and commercialization of cancer therapies. Its proprietary RADR® platform leverages artificial intelligence and machine learning with the aim of uncovering novel therapeutic opportunities, accelerating drug development timelines, and improving patient outcomes. The company's approach combines advanced computational biology with traditional pharmaceutical development to transform how cancer drugs are discovered and developed. For more information, visit: - Website: ( ) - LinkedIn: - X: @lanternpharma ( Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements include, among other things, statements relating to: future events or our future financial performance; the potential advantages of our RADR ® platform in identifying drug candidates and patient populations that are likely to respond to a drug candidate; our strategic plans to advance the development of our drug candidates and antibody drug conjugate (ADC) development program; estimates regarding the development timing for our drug candidates and ADC development program; expectations and estimates regarding clinical trial timing and patient enrollment; our research and development efforts of our internal drug discovery programs and the utilization of our RADR ® platform to streamline the drug development process; our intention to leverage artificial intelligence, machine learning and genomic data to streamline and transform the pace, risk and cost of oncology drug discovery and development and to identify patient populations that would likely respond to a drug candidate; estimates regarding patient populations, potential markets and potential market sizes; sales estimates for our drug candidates and our plans to discover and develop drug candidates and to maximize their commercial potential by advancing such drug candidates ourselves or in collaboration with others. Any statements that are not statements of historical fact (including, without limitation, statements that use words such as 'anticipate,' 'believe,' 'contemplate,' 'could,' 'estimate,' 'expect,' 'intend,' 'seek,' 'may,' 'might,' 'plan,' 'potential,' 'predict,' 'project,' 'target,' 'model,' 'objective,' 'aim,' 'upcoming,' 'should,' 'will,' 'would,' or the negative of these words or other similar expressions) should be considered forward-looking statements. There are a number of important factors that could cause our actual results to differ materially from those indicated by the forward-looking statements, such as (i) the risk that we may not be able to secure sufficient future funding when needed and as required to advance and support our existing and planned clinical trials and operations, (ii) the risk that observations in preclinical studies and early or preliminary observations in clinical studies do not ensure that later observations, studies and development will be consistent or successful, (iii) the risk that our research and the research of our collaborators may not be successful, (iv) the risk that we may not be successful in licensing potential candidates or in completing potential partnerships and collaborations, (v) the risk that none of our product candidates has received FDA marketing approval, and we may not be able to successfully initiate, conduct, or conclude clinical testing for or obtain marketing approval for our product candidates, (vi) the risk that no drug product based on our proprietary RADR ® AI platform has received FDA marketing approval or otherwise been incorporated into a commercial product, and (vii) those other factors set forth in the Risk Factors section in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission on March 27, 2025. You may access our Annual Report on Form 10-K for the year ended December 31, 2024, under the investor SEC filings tab of our website at ( ) or on the SEC's website at ( ). Given these risks and uncertainties, we can give no assurances that our forward-looking statements will prove to be accurate, or that any other results or events projected or contemplated by our forward-looking statements will in fact occur, and we caution investors not to place undue reliance on these statements. All forward-looking statements in this press release represent our judgment as of the date hereof, and, except as otherwise required by law, we disclaim any obligation to update any forward-looking statements to conform the statement to actual results or changes in our expectations. View source version on CONTACT: Investor Contact Investor Relations [email protected] +1-972-277-1136 KEYWORD: UNITED STATES NORTH AMERICA TEXAS INDUSTRY KEYWORD: RESEARCH CLINICAL TRIALS HEALTH TECHNOLOGY BIOTECHNOLOGY HEALTH PHARMACEUTICAL OTHER SCIENCE SCIENCE ONCOLOGY SOURCE: Lantern Pharma Inc. Copyright Business Wire 2025. PUB: 07/23/2025 07:56 AM/DISC: 07/23/2025 07:56 AM
Yahoo
5 hours ago
- Business
- Yahoo
BD Announces First Pharma-Sponsored Clinical Trial Using BD Libertas™ Wearable Injector Technology for Biologic Drugs
FRANKLIN LAKES, N.J., July 23, 2025 /PRNewswire/ -- BD (Becton, Dickinson and Company) (NYSE: BDX), a leading global medical technology company, announces the first pharma-sponsored combination product clinical trial using the BD Libertas™ Wearable Injector for subcutaneous delivery of complex biologics. The selection of BD Libertas™ Wearable Injector for this pharma-sponsored trial follows successful outcomes from more than 50 BD-conducted pre-clinical and clinical studies, including a device clinical study demonstrating excellent performance with 100% of study participants stating they would likely use the BD Libertas™ Wearable Injector if prescribedi,ii. The pharma-sponsored combination product clinical trial represents a significant advancement in accelerating innovation in drug-device combination products that provide greater flexibility for patients, including potential conversion from infused medications that require patients to travel to a hospital or clinic to more convenient patient care in various settings, including self-injection at home. "This trial demonstrates BD's commitment to helping pharma companies by advancing large-volume injection science, ensuring therapies are accessible and patient friendly by offering more efficient and convenient options for biologics," said Patrick Jeukenne, worldwide president of BD Pharmaceutical Systems. "BD's enhanced testing capabilities acquired through ZebraSci and the proven capabilities of the BD Libertas™ Wearable Injector technology further position BD as an innovative leader in drug delivery." The BD Libertas™ Wearable Injector is an innovative, prefilled, patient ready-to-use drug delivery systemiii designed to enable delivery of complex biologics via subcutaneous injection. The biologics market is expected to grow to more than $670 billioniv by 2030 and for pharmaceutical companies developing these complex drugs, the BD Libertas™ Wearable Injector offers a customizable, patient-centric solution. The BD Libertas™ Wearable Injector: Supports delivery of high-viscosity biologics (up to 50 centipoise), enabling a wide range of subcutaneous therapies Is offered in 2 to 5 mL and 5 to 10 mL configurations providing flexibility for diverse therapeutic requirements Features a fully mechanical, patient ready-to-use design with a simple "peel, stick and click" mechanism, requiring no end-user filling or assemblyiii BD's ongoing validations of fill-finish and final assembly processes with multiple Contract Manufacturing Organizations (CMOs) enable the company to support pharmaceutical partners from development through commercial-scale production. For more information about how BD Libertas™ Wearable Injector is enabling biologic therapy delivery, visit About BD Libertas™ Wearable InjectorBD Libertas™ Wearable Injector is a product in development; some statements are forward looking and are subject to a variety of risks and uncertainties. BD Libertas™ Wearable Injector is a device component intended for drug-device combination products and not subject to FDA 510(k) clearance or separate EU CE mark certification. About BD BD is one of the largest global medical technology companies in the world and is advancing the world of health by improving medical discovery, diagnostics and the delivery of care. The company supports the heroes on the frontlines of health care by developing innovative technology, services and solutions that help advance both clinical therapy for patients and clinical process for health care providers. BD and its more than 70,000 employees have a passion and commitment to help enhance the safety and efficiency of clinicians' care delivery process, enable laboratory scientists to accurately detect disease and advance researchers' capabilities to develop the next generation of diagnostics and therapeutics. BD has a presence in virtually every country and partners with organizations around the world to address some of the most challenging global health issues. By working in close collaboration with customers, BD can help enhance outcomes, lower costs, increase efficiencies, improve safety and expand access to health care. For more information on BD, please visit or connect with us on LinkedIn at X (formerly Twitter) @BDandCo or Instagram @becton_dickinson. Contacts: Media: Investors: Fallon McLoughlin Adam Reiffe BD Corporate Communications BD Investor Relations 201.258.0361 201.847.6927 i Early feasibility clinical study of investigational BD Libertas™ Wearable Injector (WI) evaluated 5ml, non- Newtonian ~8cP subcutaneous placebo injections in 52 healthy adult subjects for functionality, tissue effects, subject tolerability (100mm Visual Analog Scale (VAS)) and acceptability (questionnaires with 5-point Likert or yes /no responses). Tissue effects were measured from WI removal post-injection through 24 hours with calipers (wheals) or graded on a 5-point scale (erythema, bleeding) from 0-none to 4-severe, significant, respectively. Where tissue effects were observed, the majority (>50%) were resolved within 60 minutes and all within 24 hours. Subject pain (100mm VAS) peaked mid-injection (mean 9.1mm, SD 13.4) and rapidly resolved within 30 minutes (mean 0 .4mm, SD 2.6). Subjects found acceptable (Likert agree + strongly agree or yes responses) their peak pain (≥90.2%), injection site appearance (≥92.2% ).ii Woodley, W. D. et al. Clinical Evaluation of an Investigational 5ml Wearable Injector in Healthy Human Subjects. Clin Transl Sci. 2021 May;14(3):859-869. Pharma filled and assemblediv Mordor Intelligence (2025). Biologics Market Size - Industry Report on Share, Growth Trends & Forecasts Analysis (2025 - 2030). View original content to download multimedia: SOURCE BD (Becton, Dickinson and Company) Sign in to access your portfolio
Yahoo
15 hours ago
- Business
- Yahoo
XORTX Completes Non-Brokered Private Placement of Units
Not For Distribution to United States News Wire Services or for Dissemination in the United States CALGARY, Alberta, July 22, 2025 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ('XORTX' or the 'Company') (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat gout and progressive kidney disease, is pleased to announce the closing of its previously announced non-brokered private placement of units ('Units'), whereby it issued 1,267,123 Units at a price of US$0.73 per Unit for aggregate gross proceeds of US$925,000 pursuant to the listed issuer financing exemption under Part 5A of National Instrument 45-106 – Prospectus Exemptions (the 'Offering'). Under the Offering, each Unit consisted of one common share in the capital of the Company ('Common Share') and one common share purchase warrant ('Warrant'). Each Warrant entitles the holder thereof to purchase one additional Common Share at a price of US$1.20 for a period of sixty (60) months following the date of issuance provided, however, that if the closing price of the Common Shares on the Nasdaq is greater than US$2.00 for ten (10) or more consecutive trading days, the Warrants will be accelerated and will expire on the 30th business day following the date of such notice. Closing of the Offering was approved by the TSX Venture Exchange ('TSXV'), and the securities issued under the Offering will not be subject to a four-month and one-day statutory hold period. The Company intends to use the proceeds of the Offering as more specifically described in the Offering Document and for gout programs, general corporate and working capital purposes. In connection with the Offering, the Company paid an aggregate of $12,264 in finder's fees and issued, in aggregate, 16,800 finder's warrants. The securities have not and will not be registered under the U.S. Securities Act of 1933, as amended (the 'U.S. Securities Act'), or any applicable state securities laws and may not be offered or sold to, or for the account or benefit of, persons in the United States or 'U.S. persons,' as such term is defined in Regulation S promulgated under the U.S. Securities Act, absent registration or an exemption from such registration requirements. This news release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of the securities in any jurisdiction in which such offer, solicitation, or sale would be unlawful. The issuance of 8,191 Units to an insider of the Company constitutes a 'related party transaction' as such term is defined in Multilateral Instrument 61-101 – Protection of Minority Securityholders in Special Transactions ('MI 61-101'). Pursuant to Sections 5.5(a) and 5.7(1)(a) of MI 61-101, the Company relied on exemptions from the formal valuation and minority shareholder approval requirements, respectively, as neither the fair market value of the Units nor the consideration for such Units, insofar as it involves the insider, exceeds 25 percent of the Company's market capitalization. In other news, the non-brokered private placement that was announced on May 19, 2025 will not proceed. About XORTX Therapeutics Inc. XORTX is a pharmaceutical company with three clinically advanced products in development: 1) our lead program XRx-026 program for the treatment of gout; 2) XRx-008 program for ADPKD; and 3) XRx-101 for acute kidney and other acute organ injury associated with respiratory virus infections. In addition, the Company is developing XRx-225, a pre-clinical stage program for Type 2 diabetic nephropathy. XORTX is working to advance products that target aberrant purine metabolism and xanthine oxidase to decrease or inhibit production of uric acid. At XORTX, we are dedicated to developing medications that improve the quality of life and health of individuals with gout and other important diseases. Additional information on XORTX is available at For more information, please contact: Allen Davidoff, CEO Nick Rigopulos, Director of Communications adavidoff@ nick@ +1 403 455 7727 +1 617 901 0785 Forward-Looking Statements Statements contained in this news release that are not historical facts are 'forward-looking information' or 'forward-looking statements' within the meaning of applicable Canadian securities laws. Such forward-looking statements or information are provided to inform the Company's shareholders and potential investors about management's current expectations and plans relating to the future. Readers are cautioned that reliance on such information may not be appropriate for other purposes. Any such forward-looking information may be identified by words such as 'anticipate', 'proposed', 'estimates', 'would', 'expects', 'intends', 'plans', 'may', 'will', and similar expressions, although not all forward-looking information contains these identifying words. More particularly and without limitation, the forward-looking information in this news release includes (i) expectations regarding the Company's current and future financing plans; (ii) expectations concerning the Company's plans and objectives in respect of the Offering's gross proceeds; and (iii) expectations regarding the Company's business plans and operations. Forward-looking information is based on a number of factors and assumptions that have been used to develop such information, but which may prove to be incorrect and are inherently subject to significant business, economic and competitive uncertainties, and contingencies. The material factors and assumptions used in preparing the forward-looking information contained herein include, among others, our ability to obtain additional financing; the accuracy of our estimates regarding expenses, future revenues and capital requirements; the success and timing of our preclinical studies and clinical trials; the performance of third-party manufacturers and contract research organizations; our plans to develop and commercialize our product candidates; our plans to advance research in other kidney disease applications; and our ability to obtain and maintain intellectual property protection for our product candidates. Although the Company believes that the expectations reflected in such forward-looking information are reasonable, undue reliance should not be placed on forward-looking information because the Company can give no assurance that such expectations will prove to be correct. The forward-looking information in this news release reflects the Company's current expectations, assumptions and/or beliefs based on information currently available to the Company. Any forward-looking information speaks only as of the date on which it is made and, except as may be required by applicable securities laws, the Company disclaims any intent or obligation to update any forward-looking information, whether as a result of new information, future events or results or expressly qualified by this cautionary statement. More detailed information about the risks and uncertainties affecting XORTX is contained under the heading 'Risk Factors' in XORTX's Annual Report on Form 20-F filed with the SEC, which is available on the SEC's website, (including any documents forming a part thereof or incorporated by reference therein), as well as in our reports, public disclosure documents and other filings with the securities commissions and other regulatory bodies in Canada, which are available on Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy of this release. No stock exchange, securities commission or other regulatory authority has approved or disapproved the information contained herein.
Globe and Mail
21 hours ago
- Business
- Globe and Mail
ALKS Down Despite Positive Top-Line Data From Narcolepsy Study
Alkermes ALKS announced positive top-line data from the phase II Vibrance-1 study, which evaluated its novel, investigational, and oral orexin 2 receptor agonist, alixorexton (formerly ALKS 2680), for treating patients with narcolepsy type 1 (NT1). In the Vibrance-1 study, patients were randomized to receive alixorexton (4 mg, 6 mg or 8 mg) or placebo to be taken once daily for six weeks. Data from the study showed that treatment with alixorexton across all doses led to a statistically significant, clinically meaningful and dose-dependent improvement from baseline versus placebo in wakefulness on the Maintenance of Wakefulness Test (MWT) — the study's primary endpoint. Treatment with alixorexton across all doses demonstrated statistically significant and clinically meaningful improvements from baseline in excessive daytime sleepiness versus placebo at week six on the Epworth Sleepiness Scale — a key secondary endpoint of the Vibrance-1 study. Although all three doses of alixorexton led to improvements in weekly cataplexy rates — also a key secondary endpoint of the study — only the 6 mg dose achieved statistical significance. This might have hurt investor sentiment and caused the stock to decline 8.8% yesterday. Also, treatment with once-daily alixorexton led to robust and clinically meaningful improvements in patient-reported outcomes for excessive daytime sleepiness, fatigue and cognition compared to placebo. Treatment with alixorexton across all doses was generally safe and well-tolerated. ALKS' Price Performance Shares of Alkermes have declined 7.3% so far this year compared with the industry 's decrease of 2.2%. Building on the overall success of the Vibrance-1 study, management is planning to initiate a global phase III program for alixorexton in patients with NT1. NT1 is a chronic sleep disorder causing excessive daytime sleepiness and sudden muscle weakness called cataplexy due to orexin deficiency. We note that orexin agonists like alixorexton directly target the brain's orexin system — the root cause of NT1. Unlike other available drugs that only fight sleepiness, orexin agonists may also prevent cataplexy, offering a more natural and complete treatment approach. This can make them a promising and transformative treatment in narcolepsy care. The detailed safety and efficacy data from the phase II Vibrance-1 study are expected to be presented at an upcoming scientific conference. ALKS' Other Development Activities With Alixorexton Besides NT1, alixorexton is also being studied for the treatment of narcolepsy type 2 ('NT2') and idiopathic hypersomnia ('IH'). The phase II Vibrance-2 study is evaluating the safety and efficacy of alixorexton versus placebo in adults with NT2. Enrollment in this study is expected to be completed shortly, with data from the same expected during fall. In April 2025, the company initiated the phase II Vibrance-3 study, evaluating the safety and efficacy of alixorexton in adults with idiopathic hypersomnia, a rare, chronic and neurological sleep disorder. Per management, if successfully developed and upon potential approval, alixorexton can serve an area of high unmet medical need in the treatment of NT1 and NT2 as well as IH. However, upon potential approval, alixorexton is likely to face competition from Axsome 's AXSM Sunosi (solriamfetol), which is presently marketed in the United States for the treatment of narcolepsy. Several label expansion studies on solriamfetol are also currently underway. Axsome acquired the U.S. rights for Sunosi from Jazz Pharmaceuticals JAZZ in May 2022. AXSM began selling Sunosi in the U.S. market in May 2022. JAZZ received approval for Sunosi as a treatment for narcolepsy in 2019. Jazz's other sleep disorder drugs, Xyrem and Xywav, also hold a strong market share. ALKS' Zacks Rank & Stock to Consider Alkermes currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Arvinas ARVN, sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today's Zacks #1 Rank stocks here. In the past 60 days, estimates for Arvinas' 2025 loss per share have narrowed from $1.60 to $1.51. Loss per share estimates for 2026 have narrowed from $3.28 to $2.98 during the same period. ARVN stock has plunged 60.7% year to date. Arvinas' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 82.09%. Zacks' Research Chief Names "Stock Most Likely to Double" Our team of experts has just released the 5 stocks with the greatest probability of gaining +100% or more in the coming months. Of those 5, Director of Research Sheraz Mian highlights the one stock set to climb highest. This top pick is a little-known satellite-based communications firm. Space is projected to become a trillion dollar industry, and this company's customer base is growing fast. Analysts have forecasted a major revenue breakout in 2025. Of course, all our elite picks aren't winners but this one could far surpass earlier Zacks' Stocks Set to Double like Hims & Hers Health, which shot up +209%. Free: See Our Top Stock And 4 Runners Up Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Alkermes plc (ALKS): Free Stock Analysis Report Jazz Pharmaceuticals PLC (JAZZ): Free Stock Analysis Report Axsome Therapeutics, Inc. (AXSM): Free Stock Analysis Report Arvinas, Inc. (ARVN): Free Stock Analysis Report
