THRIVE-2 exceeded its enrollment target due to patient demand; 188 patients enrolled with approximately 40% from US sites THRIVE topline readout in patients with active TED on track for September 2024 THRIVE-2 topline readout on track for year-end 2024 WALTHAM, Mass. / Jul 25, 2024 / Business Wire / Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company focused on discovering and developing potential...Read more
SUNNYVALE, Calif., July 25, 2024 (GLOBE NEWSWIRE) -- BioCardia, Inc. [Nasdaq: BCDA], a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, announced today that the confirmatory Phase 3 trial of its autologous CardiAMP cell therapy product candidate for patients with ischemic heart failure of reduced ejection fraction (HFrEF) has commenced enrollment in the United...Read more
Giroctocogene fitelparvovec study meets primary and key secondary objectives of superiority compared to prophylaxis NEW YORK / Jul 24, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced positive topline results from the Phase 3 AFFINE study (NCT04370054) evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe hemophilia A. The AFFINE study...Read more
PURPOSE 1 Data Showed Zero Infections and 100% Efficacy and Superiority of Lenacapavir to Background HIV Incidence and Daily Truvada® for PrEP If Approved, Lenacapavir Would be the First and Only Twice-Yearly PrEP Choice and Could Address Critical Gaps in Uptake and Adherence for Individuals Who Need or Want PrEP Gilead Commits to Prioritizing Swift Access and Enabling Efficient Paths for Regulatory Approval of Lenacapavir...Read more
Study enrolled 268 patients, exceeding target enrollment. Largest interventional study ever to be conducted in pulmonary sarcoidosis. Topline data are expected in the third quarter of 2025. SAN DIEGO, July 22, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: ATYR), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today...Read more
NEW YORK, July 21, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced today that the confirmatory Phase 3 trial of its allogeneic, immunoselected, and industrially manufactured stromal cell product rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease of less than five years duration...Read more
Robotic HIFU therapy continues to maintain an excellent safety profile, confirming positive safety data from prior Phase 1 and 2 studies in patients with deep infiltrating endometriosis At three months post procedure, the study’s primary endpoint of reduced acute pelvic pain in the HIFU treatment arm compared to the Sham treatment arm was not met Significant improvements were observed across primary and secondary outcome measures...Read more
SPACE pediatric Phase 3 study met its primary end point for efficacy with a significantly greater reduction in Monthly Migraine Days compared to placebo Efficacy is consistent with AJOVY (fremanezumab) pivotal Phase 3 and Real-World Evidence studies in adults with no new emergent safety signals observed Full data to be presented at a medical meeting later in 2024 TEL AVIV, Israel / Jul 18, 2024 / Business Wire / Teva Pharmaceutical...Read more
Two-year Phase III data presented at ASRS 2024 show Susvimo’s potential as an alternative to eye injections to treat diabetic macular edema (DME) and diabetic retinopathy (DR) Safety data were consistent with the known safety profile for Susvimo in people with DME and DR Additionally, the FDA has accepted the filing application for Susvimo in DME and DR based on one-year Pagoda and Pavilion study data Susvimo is a...Read more
Topline results are expected in Q3 2025 ADELAIDE, Australia and CAMBRIDGE, Mass., July 18, 2024 (GLOBE NEWSWIRE) -- Bionomics Limited (Nasdaq: BNOX) (Bionomics or Company), a biotechnology company developing novel, potential first-in-class, allosteric ion channel modulators to treat patients suffering from serious central nervous system (CNS) disorders with high unmet medical need, today announced the initiation of patient...Read more
STAR-1 topline results expected in first quarter of 2025 About 30 million acne patients seek treatment in the U.S. each year DMT310, if approved, could be the first once-weekly topical product for the treatment of acne SAN DIEGO, CA / ACCESSWIRE / July 17, 2024 / Dermata Therapeutics, Inc. (Nasdaq:DRMA)(Nasdaq:DRMAW) ("Dermata" or the "Company"), a late-stage biotechnology company focusing on the treatment of...Read more
EMBARQ-CSU1 and EMBARQ-CSU 2 will enroll more than 1800 patients suffering from CSU Studies include both biologic naïve and experienced patients HAMPTON, N.J., July 16, 2024 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (NASDAQ:CLDX) announced today the initiation of its global Phase 3 program, consisting of two Phase 3 trials (EMBARQ-CSU1 and EMBARQ-CSU2) designed to establish the efficacy and safety of barzolvolimab in...Read more
Over 100,000 patients in the United States have IPF, with two approved therapies available that only slow the course of disease progression Top line data expected in the second half of 2025 SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C. / Jul 10, 2024 / Business Wire / United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, announced full enrollment of the TETON 2 study evaluating the use of Tyvaso®...Read more
Testing the efficacy and safety of our lead product candidate, INT230-6, as Monotherapy Compared with Systemic U.S. Standard-of-Care Chemotherapy SHELTON, Conn., July 9, 2024 /PRNewswire/ -- Intensity Therapeutics, Inc. ("Intensity" or "the Company") (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumorally injected cancer therapies...Read more
NAARDEN, The Netherlands and MIAMI, July 08, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (“CVD”) with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today...Read more
WATERTOWN, Mass. and GOSSELIES, Belgium, July 08, 2024 (GLOBE NEWSWIRE) -- iTeos Therapeutics, Inc. (Nasdaq: ITOS) (“iTeos”), a clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of immuno-oncology therapeutics for patients, today announced the dosing of the first patient in GALAXIES Lung-301, a global, randomized, double-blind Phase 3 registrational clinical trial of belrestotug +...Read more
SOMERSET, N.J., July 02, 2024 (GLOBE NEWSWIRE) -- Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, announced today positive overall survival results from CARTITUDE-4, an ongoing, global randomized, open-label Phase 3 study evaluating the efficacy and safety of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide,...Read more
CARVYKTI ® is the first and only BCMA-targeted CAR-T cell therapy approved by the U.S. FDA for the treatment of patients with multiple myeloma who have had at least one prior line of therapy RARITAN, N.J., July 2, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) announced today positive results from a prespecified second interim analysis of the Phase 3 CARTITUDE-4 study evaluating CARVYKTI® (ciltacabtagene autoleucel;...Read more
116 patients enrolled June represents a new record breaking month for patient enrollment TORONTO, July 02, 2024 (GLOBE NEWSWIRE) -- Spectral Medical Inc. (“Spectral” or the “Company”) (TSX: EDT), a late-stage theranostic company advancing therapeutic options for sepsis and septic shock, today provided an update on the Company’s Tigris trial, a Phase 3 follow-on study evaluating the use of Polymyxin B Hemoperfusion (“PMX”) in a...Read more
First-and-only FcRn blocker to demonstrate superiority in activities of daily living (MG-ADLa) over placebo when added to standard of care over 24 weeks in antibody positive patients: anti-AChR+, anti-MuSK+, anti-LRP4+ HELSINKI, June 28, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced positive results from the nipocalimab Phase 3 Vivacity-MG3 study in patients with generalized myasthenia gravis (gMG)....Read more
PRINCETON, N.J., June 27, 2024 (GLOBE NEWSWIRE) -- Acasti Pharma Inc. (Nasdaq: ACST) (Acasti or the Company), a late-stage, biopharma company advancing GTX-104, its novel injectable formulation of nimodipine that addresses high unmet medical needs for a rare disease, aneurysmal subarachnoid hemorrhage (aSAH), today announced that the Company’s pivotal Phase 3 STRIVE-ON safety trial (the STRIVE-ON trial–NCT05995405) has exceeded the 50%...Read more
LOS ALTOS, Calif., June 26, 2024 (GLOBE NEWSWIRE) -- RenovoRx, Inc. (“RenovoRx” or the “Company”) (Nasdaq: RNXT), a clinical-stage biopharmaceutical company developing novel precision oncology therapies based on a local drug-delivery platform, announced today that the University of Nebraska Medical Center (UNMC) is now enrolling patients with Locally Advanced Pancreatic Cancer (LAPC) in the Company’s ongoing pivotal Phase III TIGeR-PaC...Read more
TEL AVIV, Israel, June 26, 2024 (GLOBE NEWSWIRE) -- PainReform Ltd. (Nasdaq: PRFX) ("PainReform" or the "Company"), a clinical-stage specialty pharmaceutical company focused on the reformulation of established therapeutics, today announced a major milestone on the way to registration - the successful completion of patient enrollment in its Phase 3 clinical trial for PRF-110, a novel analgesic drug candidate designed for the treatment of...Read more
Statistically Significant Improvement in Percent Predicted Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO) Versus Placebo at Week 24 (Primary Endpoint) and Week 48 (Secondary Endpoint) Statistically Significant Improvement in St. George’s Respiratory Questionnaire (SGRQ) Total Score at Week 24 (Secondary Endpoint) 97% of Patients Completed Double-Blind Treatment Through Week 48 with No Trial Drug Related Adverse...Read more
Bagsværd, Denmark, 26 June 2024 – Novo Nordisk (NYSE: NVO) today announced that the CLARION-CKD phase 3 trial failed to meet its primary endpoint and that it will recognise an impairment loss of around DKK 5.7 billion related to the intangible asset ocedurenone in the second quarter of 2024. Novo Nordisk acquired ocedurenone from KBP Biosciences PTE., Ltd. in 2023. The phase 3 lead indication trial CLARION-CKD, which investigated...Read more
ANX005-Treated Patients Demonstrated Faster and More Complete Recovery from Week 1 through Week 26 on Primary and Multiple Pre-Specified Endpoints Two and a Half Times More ANX005-Treated Patients Returned to a Normal / Pre-Disease State of Health Over Placebo on GBS-DS by Week 26, Increasing Over Time ANX005 Beneficial Impact Larger in Patients with North American and European Baseline Characteristics Across Key Measures of...Read more
First immunotherapy regimen before and after surgery to extend survival in bladder cancer WILMINGTON, Del. / Jun 25, 2024 / Business Wire / Positive high-level results from the NIAGARA Phase III trial showed AstraZeneca’s IMFINZI® (durvalumab) in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of event-free survival (EFS) and the key secondary endpoint...Read more
SOUTH SAN FRANCISCO, Calif. / Jun 25, 2024 / Business Wire / Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, today announced that the first patient has been dosed in the company’s Phase 3 NEAT (Neurologic Effects of EryDex on Subjects with A-T) clinical trial to evaluate the...Read more
Achieved 28% and 33% Reduction in Composite of All-Cause Mortality and Recurrent Cardiovascular Events in the Overall and Monotherapy Populations, Respectively Reduced All-Cause Mortality by 36% and 35% in the Overall and Monotherapy Populations, Respectively, in a Pre-Specified Secondary Endpoint Demonstrated Clinically Significant Benefits on 6-Minute Walk Test, Kansas City Cardiomyopathy Questionnaire and NYHA Class – Key Measures of...Read more
PRESERVE 2 Did Not Achieve Statistical Significance in the Primary Endpoint of Overall Survival (OS) in the Intent-to-Treat (ITT) Population The Company Will Focus its Efforts on the Global Extensive-Stage Small Cell Lung Cancer (ES-SCLC) Market G1 is Sufficiently Funded to Achieve Anticipated Company Profitability in the Second Half of 2025 \Management to Host Webcast and Conference Call Today at 8:30 AM ET RESEARCH TRIANGLE...Read more
FRONTIER2 data presented at ISTH 2024 showed that up to 95% of people, who had no prior prophylaxis treatment, experienced zero treated bleeds while on Mim8 Bagsværd, Denmark, 23 June 2024 – Novo Nordisk announced results from the phase 3 FRONTIER2 trial of 254 adults and adolescents aged 12 years and over with haemophilia A, with and without inhibitors. The trial assessed both once-weekly and once-monthly prophylactic...Read more
Study proves inhaled insulin is as effective as usual care (primarily automated insulin delivery pumps or multiple daily injections) for adults living with T1D meeting the primary endpoint Patients utilizing inhaled insulin reached target A1c (less than 7%) 30% of the time compared to 17% with usual care and 24% had time-in-range (TIR) above 70% with no increased hypoglycemia compared with 13% with usual care More than 50% of subjects...Read more
In the primary endpoint, tirzepatide reduced moderate-to-severe OSA severity by up to 62.8% (about 30 fewer events per hour) In a key secondary endpoint from two clinical studies, 43.0% and 51.5% of participants taking tirzepatide at the highest dose reached the criteria for disease resolution as defined by apnea-hypopnea index and Epworth Sleepiness Scale measures Lilly submitted tirzepatide for the treatment of...Read more
First Phase 3 HIV Prevention Trial Ever to Show Zero Infections Independent Data Monitoring Committee Recommended That Gilead Stop the Blinded Phase of the PURPOSE 1 Trial at Interim Analysis and Offer Open-Label Lenacapavir to All Participants FOSTER CITY, Calif. / Jun 20, 2024 / Business Wire / Gilead Sciences, Inc. (Nasdaq: GILD) today announced topline results from an interim analysis of its pivotal, Phase 3 PURPOSE 1...Read more
MALVERN, Pa., June 20, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the first patient has been dosed in its Phase 3 liMeliGhT clinical trial for OCU400—a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP). “Each clinical...Read more
TREMFYA® Phase 3 Crohn's disease study achieves all primary and secondary endpoints SPRING HOUSE, Pa., June 20, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced positive topline results from the pivotal Phase 3 GRAVITI investigational study of TREMFYA® (guselkumab) subcutaneous (SC) induction therapy in adult patients with moderately to severely active Crohn's disease.1 The study met both co-primary endpoints,...Read more
Favorable Findings from the Longest Public Clinical Study in CIDP Support HYQVIA as an Effective Long-Term Treatment Option for Maintaining Stable Disease Course in CIDP ADVANCE Clinical Program Reflects Takeda’s Commitment to Continued Research on the Role of Immunoglobulin (IG) Therapy for Patients with Rare Neuroimmunological Disorders OSAKA, Japan & CAMBRIDGE, Mass. / Jun 18, 2024 / Business Wire / Takeda (TSE:4502/NYSE:TAK)...Read more
Naporafenib is a potential first-in-class and best-in-class pan-RAF inhibitor for multiple RAS/MAPK pathway-driven tumors and has been dosed in over 500 patients to date Favorable mOS and mPFS demonstrated in pooled analysis of Phase 1b and Phase 2 trials in NRASm melanoma Randomized Stage 1 readout for naporafenib plus trametinib vs. trametinib monotherapy expected in 2025 SAN DIEGO, June 18, 2024 (GLOBE NEWSWIRE) -- Erasca, Inc....Read more
In Study 502, lumateperone 42 mg achieved statistically significant and clinically meaningful results in both the primary and the key secondary endpoints Lumateperone 42 mg met the primary endpoint of change from baseline at Week 6 on the Montgomery-Åsberg Depression Rating Scale (MADRS) total score versus placebo (4.5 point reduction v. placebo; p<0.0001; Cohen’s d effect size (ES)=0.56) Lumateperone 42 mg also met the key...Read more
BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line results from the interim analysis expected in 2025 Recent Type C interactions with U.S. Food and Drug Administration (FDA) focused on the validated glycosylated alpha-dystroglycan (αDG) bioassay and our interim analysis plans reinforce BridgeBio's belief that there is potential to...Read more
The Phase 3 PRISM-MEL-301 trial will study the efficacy and safety of brenetafusp (IMC-F106C; PRAME-A02) in combination with nivolumab versus nivolumab or nivolumab + relatlimab in first-line advanced or metastatic cutaneous melanoma First registrational Phase 3 trial investigating PRAME-targeted therapy (OXFORDSHIRE, England & CONSHOHOCKEN, Penn. & ROCKVILLE, Md., US, 18 June 2024) Immunocore Holdings plc (Nasdaq: IMCR)...Read more
SKYLINE Study in Dravet Syndrome Narrowly Missed its Primary Endpoint of Reduction in Convulsive Seizure Frequency and Showed Clinically Meaningful and Nominally Significant Effects in Multiple Key Secondary Efficacy Endpoints SKYWAY Study in Lennox-Gastaut Syndrome Missed its Primary Endpoint of Reduction in Major Motor Drop Seizures Soticlestat Showed a Consistent and Favorable Safety and Tolerability Profile in Both Studies Takeda...Read more
The Independent Data Monitoring Committee (IDMC) Recommends Continuation of Phase 3 REGAL Trial Without Any Modifications No Safety or Futility Concerns Were Raised Based on the Efficacy and Safety Assessment of All REGAL Patients Interim Analysis Anticipated by Q4 2024 NEW YORK, June 17, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage...Read more
WATERTOWN, Mass. and GOSSELIES, Belgium, June 17, 2024 (GLOBE NEWSWIRE) -- iTeos Therapeutics, Inc. (Nasdaq: ITOS) (“iTeos”), a clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of immuno-oncology therapeutics for patients, and its development partner GSK, have initiated the first, global Phase 3 registration study of belrestotug + dostarlimab doublet versus placebo + pembrolizumab in...Read more
RAISE trial met its first co-primary endpoint demonstrating rapid cessation of status epilepticus in a highly refractory patient population The trial failed to achieve statistical significance on the second co-primary endpoint of the proportion of patients not progressing to IV anesthesia Marinus will continue to prioritize the ZTALMY® franchise and expansion opportunities in highly refractory, chronic epilepsies with Phase 3 TrustTSC...Read more
First and only BTK inhibitor to demonstrate favorable overall survival trend vs. standard-of-care chemoimmunotherapy in this setting WILMINGTON, Del. / Jun 16, 2024 / Business Wire / Positive results from the ECHO Phase III trial showed AstraZeneca’s CALQUENCE® (acalabrutinib) in combination with bendamustine and rituximab demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS)...Read more
The study met its primary endpoint of overall survival with a 41% reduction in the risk of death in people with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) treated with Columvi plus chemotherapy This Columvi combination could provide a much-needed off-the-shelf treatment option for people with transplant-ineligible R/R DLBCL Data were featured in the congress Press Briefing and presented today in the Plenary...Read more
ENERGIZE is the First Study to Demonstrate Efficacy of an Oral Treatment for Non-Transfusion-Dependent Alpha- and Beta-Thalassemia Additional ENERGIZE Poster Presentation Highlights Improvements in Fatigue and Exercise Capacity in Patients Treated with Mitapivat Compared to Placebo Agios to Webcast Virtual Investor Event on June 16, 2024 at 10 a.m. Eastern Time or 4 p.m. Central European Summer Time CAMBRIDGE, Mass., June 15,...Read more
Kaplan-Meier Estimate of Duration of Response at 12 Months in Patients Who Achieved a Complete Response at Three Months was 82.3% (95% CI, 75.9%, 87.1%) Side Effect Profile Consistent with Previous Clinical Trials of UGN-102 UroGen will Host a Virtual Event Today at 11:00 AM Eastern Time PRINCETON, N.J. / Jun 13, 2024 / Business Wire / UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing...Read more
mRNA-1283 met its primary vaccine efficacy endpoint in a Phase 3 trial, demonstrating non-inferior vaccine efficacy against COVID-19 compared to Spikevax® in participants 12 years of age and older Higher efficacy was observed in mRNA-1283 compared to Spikevax in adults 18 years of age and older CAMBRIDGE, MA / ACCESSWIRE / June 13, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced that its Phase 3 trial of mRNA-1283, an...Read more
Clinical Trial Results and Potential New Drug Application Resubmission Expected in the Second Half of 2024 LEXINGTON, Mass. / Jun 13, 2024 / Business Wire / Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated and metabolic diseases, today announced the completion of enrollment in a Phase 3 dry eye chamber clinical trial...Read more
NEW YORK / Jun 12, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced that CIFFREO, a Phase 3 global, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD) did not meet its primary endpoint of improvement in motor function among boys 4 to 7 years of age treated with...Read more
SOUTH SAN FRANCISCO, Calif., June 11, 2024 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, today announced initiation of the SYNCHRONY Outcomes study, a Phase 3 trial evaluating the efficacy and safety of efruxifermin (EFX) in patients with compensated cirrhosis,...Read more
Domvanalimab is the only anti-TIGIT antibody in Phase 3 for upper gastrointestinal (GI) adenocarcinomas with the potential to be first-to-market for this patient population HAYWARD, Calif. / Jun 10, 2024 / Business Wire / Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, today announced the completion...Read more
mRNA-1083 met its primary endpoints, eliciting higher immune responses against influenza virus and SARS-CoV-2 than licensed flu and COVID vaccines in adults 50 years and older, including an enhanced influenza vaccine in adults 65 years and older CAMBRIDGE, MA / ACCESSWIRE / June 10, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced that its Phase 3 trial of mRNA-1083, an investigational combination vaccine against influenza and...Read more
Data from Longest and Largest Hemophilia Gene Therapy Study Show Durable and Sustained Bleed Control and Factor VIII Expression Maintained Four Years Post-ROCTAVIAN Infusion Additional Data Show Meaningful Impact of ROCTAVIAN on Health-Related Quality of Life (HRQoL) SAN RAFAEL, Calif., June 7, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that new data supporting the long-term safety and...Read more
Topline Readout Expected in Q4 2024 NASHVILLE, Tenn. / Jun 06, 2024 / Business Wire / Melt Pharmaceuticals, Inc. (“Melt”), a clinical-stage pharmaceutical company developing novel approaches for procedural sedation, today announced that the first patient has been dosed in its Phase 3 program evaluating the safety and efficacy of its lead product candidate, MELT-300, a non‑IV, non-opioid tablet that combines fixed doses of midazolam...Read more
ABBV-383 is a B-cell maturation antigen (BCMA) bispecific antibody T-cell engager being evaluated in relapsed/refractory multiple myeloma (r/r MM) The CERVINO Phase 3 trial will evaluate the efficacy, safety, and tolerability of ABBV-383 monotherapy compared with standard available therapies (SATs) in patients with r/r MM who have received at least two lines of prior therapy NORTH CHICAGO, Ill., June 5, 2024 /PRNewswire/ --...Read more
Data Show a Statistically Significant 87% Reduction in IgG4-RD Flares, With Primary and All Key Secondary Endpoints Met First Randomized, Placebo-Controlled Trial to Demonstrate Benefit in IgG4-RD THOUSAND OAKS, Calif., June 5, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced positive topline results from its randomized, double-blind, multicenter, placebo-controlled Phase 3 clinical trial (NCT04540497) evaluating the...Read more
Namodenoson granted Orphan Drug and Fast track status from the FDA RAMAT GAN, Israel / Jun 05, 2024 / Business Wire / Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced an update on the status of its oncological lead drug candidate, Namodenoson in the treatment of advanced liver...Read more
Data Demonstrates Sustained Safety and Efficacy in Treating Signs and Symptoms of Dry Eye Disease NASHVILLE, Tenn. / Jun 05, 2024 / Business Wire / Harrow (Nasdaq: HROW), a leading North American eyecare pharmaceutical company, is pleased to announce results from its ESSENCE‑2 open-label extension (OLE) clinical study for VEVYE® (cyclosporine ophthalmic solution) 0.1%, the first and only cyclosporine to treat the signs and symptoms of...Read more
Positive Results from Two-year Interim Analysis Includes Participants from Phase 3 RESPONSE Study and are Highly Consistent with One-year Interim Analysis Reduction in Patient-Reported Pruritus (Itching) was Rapid and Durable in Participants with Moderate to Severe Symptoms Subset Analysis of Participants with Compensated Cirrhosis Demonstrated Clinically Meaningful Improvements in Markers of Cholestasis and Liver...Read more
Late-breaking data to be presented at the 2024 American Society of Clinical Oncology (ASCO®) Annual Meeting Dual immunotherapy combination of Opdivo plus Yervoy demonstrated improved survival benefit compared to lenvatinib or sorafenib in this patient population Objective response rate (ORR) for Opdivo plus Yervoy was more than double that for lenvatinib or sorafenib, with median duration of response of 30 months PRINCETON, N.J. /...Read more
Company exploring path forward for uproleselan in multiple AML settings based on observed efficacy results, including clinically meaningful results in primary refractory AML, and significant unmet patient need Uproleselan demonstrated a clinically meaningful improvement in median overall survival (mOS) for patients with primary refractory AML; mOS was 31.2 months for the uproleselan arm compared to 10.1 months for the placebo arm in this...Read more
Single Infusion of ANX005 30 mg/kg Met Primary Endpoint, Delivering a Highly Statistically Significant and Clinically Meaningful 2.4-fold Improvement in GBS-DS vs. Placebo at Week 8, p=0.0058 ANX005 Demonstrated Early and Sustained Improvements in Key Secondary Endpoints Including Muscle Strength, Nerve Damage and Ventilation ANX005 Displayed Rapid Target Engagement and was Generally Well-Tolerated Across Doses Real-World Evidence...Read more
First Scientific Presentation of PATHFNDR-2 Acromegaly Safety and Efficacy Data Following Positive Topline Announcement, Showing Rapid and Sustained IGF-1 Responses in People Treated with Paltusotine New PATHFNDR-1 Patient-Reported Outcomes Analysis Showed Paltusotine Lessened Day-to-Day Breakthrough Symptom Exacerbations versus Prior Treatment with Standard of Care Injections, as Assessed by Acromegaly Symptom Diary Long-Term Data...Read more
Three-year follow-up results from TRANSFORM show ongoing event-free survival and durable responses with Breyanzi compared to standard of care Results from a subgroup analysis from mantle cell lymphoma cohort of TRANSCEND NHL 001 show Breyanzi demonstrated consistent clinical benefit regardless of number of prior lines of therapy, supporting use of Breyanzi in earlier lines of treatment Data from bridging therapy subgroup analysis of...Read more
Plozasiran achieved statistically significant median reductions in triglycerides up to 80% and mean reductions in APOC3 up to 94% at month 10 Plozasiran achieved a statistically significant reduction in incidence of acute pancreatitis versus placebo Plozasiran is the company’s first investigational RNAi-based therapy to show clinical efficacy in a Phase 3 study Arrowhead plans to highlight recent data for its cardiometabolic pipeline...Read more
Study Achieved the Primary Endpoint; Mitapivat Demonstrated a Statistically Significant Transfusion Reduction Response Compared to Placebo Statistical Significance Achieved for All Key Secondary Endpoints Evaluating Additional Measures of Reduction of Transfusion Burden Compared to Placebo ENERGIZE-T is the First Phase 3 Study to Demonstrate Efficacy of an Oral, Disease-Modifying Treatment for Transfusion-Dependent Alpha-...Read more
57% of patients treated with IMFINZI were alive at three years in ADRIATIC Phase III trial WILMINGTON, Del. / Jun 02, 2024 / Business Wire / Positive results from the ADRIATIC Phase III trial showed AstraZeneca’s IMFINZI® (durvalumab) demonstrated statistically significant and clinically meaningful improvements in the dual primary endpoints of overall survival (OS) and progression-free survival (PFS) compared to placebo for patients...Read more
Four-year Analysis Conducted by GHSG Reported ADCETRIS + ECADD Combination Improved Progression-Free Survival, Showing Superior Efficacy and Tolerable Safety Profile in Patients with Newly Diagnosed Stage IIb/III/IV Classical Hodgkin Lymphoma vs eBEACOPP, a Current Standard of Care in This Setting in Europe The HD21 Study Adds to the Body of Evidence Supporting ADCETRIS as a Backbone Agent in the Treatment of Specific Lymphomas Results...Read more
Detailed data from Phase 3 ECHELON-3 study demonstrate investigational ADCETRIS regimen reduced risk of death by 37 percent compared to chemotherapy alone, resulting in median overall survival of 13.8 months versus 8.5 months Third Phase 3 trial in third type of lymphoma to show improvement in overall survival with an ADCETRIS-containing regimen NEW YORK / Jun 01, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced detailed...Read more
First presentation of data from Phase 3 KRYSTAL-12 study showed statistically significant and clinically meaningful improvement in progression-free survival with KRAZATI compared to standard of care chemotherapy Late-breaking data featured in an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting PRINCETON, N.J. / Jun 01, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced...Read more
An unprecedented 60% of patients remain alive without disease progression after five years Updated results show continued 81% reduction in risk of progression or death and 94% reduction in progression of brain metastases compared to XALKORI® NEW YORK / May 31, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced longer-term follow-up results from the Phase 3 CROWN trial evaluating LORBRENA® (lorlatinib, a third-generation...Read more
Donidalorsen delivered significant and sustained reductions in HAE attacks, with high levels of disease control and improvement in quality of life measures with monthly or every two-month dosing; continued attack rate reduction over time In first-of-its-kind prospective analysis, patients switching from prior prophylactic treatment to donidalorsen experienced further reductions in mean monthly HAE attack rates from...Read more
Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today at 5:00 p.m. ET NOVATO, Calif., May 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced positive topline results from the Phase 3 GlucoGene study (NCT05139316) evaluating DTX401, an investigational...Read more
Unprecedented: Ivonescimab Is the First Drug to Achieve Clinically Meaningful Benefit over Pembrolizumab in Randomized Phase III Clinical Trial in NSCLC Monotherapy Ivonescimab Achieved Clinically Meaningful PFS Benefit in HARMONi-2 Trial Conducted by Akeso PFS Improvement Was Observed Broadly in Patients Across Subgroups, including PD-L1 Low and PD-L1 High Expressing Tumors, Squamous and Non-Squamous Histologies Full Data Set to be...Read more
TransportNPC™ is the most comprehensive ongoing controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes for the treatment of Niemann-Pick Disease Type C1 (NPC1) Topline data from the 48-week interim analysis of 104 enrolled patients is anticipated for H1 2025 If 48-week data demonstrate significance, submission of New Drug Application (NDA) to the Food and Drug Administration (FDA) and Marketing...Read more
PHILADELPHIA and VANCOUVER, British Columbia, May 30, 2024 (GLOBE NEWSWIRE) -- BriaCell Therapeutics Corp. (NASDAQ: BCTX, BCTXW) (TSX: BCT) (“BriaCell” or the “Company”), a clinical-stage biotechnology company that develops novel immunotherapies to transform cancer care, announces initiation of a first-in-human, Phase 1/2 study evaluating safety and efficacy of Bria-OTS™, BriaCell’s personalized off-the-shelf next generation...Read more
Following recent consultation with FDA, all patients randomized from start of TEBE-AM Phase 2/3 trial will be included in the Phase 3 intent-to-treat population Phase 3 will continue three arms: KIMMTRAK monotherapy, KIMMTRAK in combination with pembrolizumab, and control Expected to accelerate time to final Phase 3 overall survival analysis (OXFORDSHIRE, England & CONSHOHOCKEN, PA & ROCKVILLE, MD, US, 29 May 2024)...Read more
MENLO PARK, Calif., May 28, 2024 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic, and neurologic disorders by modulating the effects of the hormone cortisol, today announced that GRACE, the Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with...Read more
Phase 3 program enrolled 1,984 patients across COAST and ShORe trials Trials designed to replicate superiority in visual outcomes demonstrated in Phase 2b Topline data from both pivotal trials expected in mid-CY2025 MELBOURNE, Australia and PRINCETON, N.J., May 28, 2024 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ: OPT “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly...Read more
KEYTRUDA® (pembrolizumab) is the first and only immunotherapy-based regimen to show a statistically significant improvement in OS as pre-operative (neoadjuvant) treatment with chemotherapy and then as a single agent after surgery (adjuvant) compared to pre-operative chemotherapy in patients with high-risk early-stage TNBC New OS results build on the pathological complete response and event-free survival data previously reported from the...Read more
Phase 3 Study Achieves Primary Endpoint for Both Dosage Strengths of Brensocatib with Statistically Significant and Clinically Meaningful Reduction in Frequency of Pulmonary Exacerbations Versus Placebo Treatment with Brensocatib Also Achieves Statistical Significance on Multiple Secondary Endpoints for Both Dosage Strengths Versus Placebo Brensocatib Well-Tolerated at Both Dosage Strengths Results from ASPEN Validate DPP1 Inhibition...Read more
In the overall trial population, survival results numerically favoured AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan but did not reach statistical significance TROPION-Lung01 previously met the dual primary endpoint of progression-free survival in the overall trial population Results support applications currently under review by regulatory authorities globally including in the US and EU This announcement contains inside...Read more
In the ALIGN study, atrasentan, in addition to supportive care with a renin-angiotensin system (RAS) inhibitor, demonstrated a statistically significant 36.1% proteinuria (protein in urine) reduction vs. placebo + supportive care at 36 weeks1 Endothelin A (ETA) receptor activation contributes to elevated proteinuria in IgAN2-5; atrasentan is a potent, selective ETA receptor antagonist with potential to reduce persistent proteinuria and...Read more
Secondary endpoint data for estimated glomerular filtration rate (eGFR) showed numerical improvement over 6 months vs. placebo1; additional 6-month open-label data to be presented at a future medical meeting2,3 Fabhalta showed a favorable safety profile with no new safety signals1 C3G, an ultra-rare kidney disease caused by alternative complement pathway overactivation, progresses to kidney failure in ∼50% of patients within 10...Read more
Nearly one-half of patients on mirikizumab achieved endoscopic response at 52 weeks; most of these patients were also in clinical remission INDIANAPOLIS, May 21, 2024 /PRNewswire/ -- In Eli Lilly and Company's (NYSE: LLY) pivotal Phase 3 VIVID-1 study, patients with moderately to severely active Crohn's disease, with or without previous biologic failure, achieved statistically significant and clinically meaningful...Read more
VE303 is an orally administered, potential first-in-class live biotherapeutic product candidate being developed to prevent recurrence of Clostridioides difficile infection (CDI) BOSTON / May 21, 2024 / Business Wire / PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company, noted today that its Founded Entity, Vedanta Biosciences, a clinical-stage company that is developing...Read more
Trial achieves statistically significant primary endpoint (p=0.0006) Secondary endpoint demonstrates statistically significant overall success of Mino-Lok therapy with a greater percentage of patients retaining their catheters (p=0.0025) CRANFORD, N.J., May 21, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and...Read more
Immix Biopharma is a clinical-stage biopharmaceutical company pioneering a novel class of CAR-T cell therapies and Tissue-Specific Therapeutics targeting oncology and immuno-dysregulated diseases with >75 patients treated to-date. Our lead cell therapy asset is NXC-201...
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