Initiated Phase 2 clinical trial MALVERN, Pa., July 25, 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 dosing is complete in the third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a novel modifier gene therapy candidate being...Read more
Data indicates that SON-080 was well-tolerated at both doses, with no evidence of a pro-inflammatory cytokine response Pain and quality of life survey results suggest the potential for rapid improvement of peripheral neuropathy symptoms and post-dosing durability with both doses, compared to placebo controls Sonnet intends to seek a partnership to support initiation of a Phase 2 clinical trial of SON-080 in Diabetic Peripheral...Read more
ION582 showed robust and consistent benefit in communication, cognition and motor function in a broad patient population evaluated with a comprehensive set of assessment tools that collect input from parents and clinicians 97% of patients in the medium and high dose groups saw improvement in overall Angelman syndrome symptoms as measured by the SAS-CGI-C Improvements on the Bayley-4 in cognition, communication and motor function...Read more
ROCHESTER, N.Y., July 17, 2024 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating Alzheimer’s disease (AD) and cancer through the inhibition of Semaphorin 4D (SEMA4D), today announced that it will present topline data for its randomized, double-blind, phase 1b/2 SIGNAL-AD study of pepinemab treatment for Alzheimer’s disease at the Alzheimer’s...Read more
SAN DIEGO, July 15, 2024 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that enrollment has been completed and dosing initiated for the sixth dose cohort of its Phase 1/2 study of ONCT-534 for the treatment of patients with metastatic castration-resistant prostate cancer who are relapsed or refractory to...Read more
Achieved mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline >10% reduction in LVMI at 12 months in 75% of participants with elevated LVMI at baseline Sustained and consistent improvements in other key measures of cardiac status, including left ventricular wall thickness and troponin I, in majority of participants at 12...Read more
The post-dose average values for Total Pharyngeal Residue (i.e., the amount of solid food or liquid material remaining in the pharynx after the first swallow) remained meaningfully reduced at the 180-day post-dose assessment following the administration of the low dose of gene therapy BB-301 as compared to the pre-dose average values recorded for Subject 1 during the OPMD Natural History Study Critically, for three of the four food...Read more
GenFleet’s IND in China was cleared in June 2024; this is the first discovery program from the collaboration Verastem announced in 2023 to advance into human clinical trials Verastem plans to initiate development studies outside of China after evaluating initial dose escalation data from the Phase 1 study in China BOSTON / Jul 12, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to...Read more
Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control Achieved statistically significant lowering of CSF neurofilament light protein (NfL) compared to baseline at...Read more
Lead site Memorial Sloan Kettering Cancer Center (MSKCC) Timing of milestone in-line with mid-2024 guidance Data from ex-US clinical trial reported at ASGCT 2024 showed a 92% overall response rate LOS ANGELES, July 08, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”, “IMMX”) (Nasdaq: IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune...Read more
MULHOUSE, France, and SUNNYVALE, Calif., July 08, 2024 (GLOBE NEWSWIRE) -- CellProthera, a private company specializing in cell-based therapies for repairing ischemic tissues, and BioCardia, Inc. [Nasdaq: BCDA], a developer of cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, today announce success from a collaborative Phase II trial of ProtheraCytes for the treatment of acute myocardial...Read more
EMERYVILLE, Calif., June 27, 2024 /PRNewswire/ -- Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, today announced that the first participant has been dosed in a Phase 1/2 clinical trial evaluating the safety, tolerability, and immunogenicity of Z-1018, the company's investigational vaccine candidate being developed for the prevention of...Read more
CAMBRIDGE, Mass., June 26, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the first patient was treated with BEAM-302, an investigational in vivo base editing medicine designed to precisely correct the underlying cause of severe alpha-1 antitrypsin deficiency (AATD), that is currently being evaluated in a Phase 1/2...Read more
Statistically significant, potent, and durable allele-selective silencing: 46% mean reduction in CSF mutant huntingtin (mHTT) protein compared to placebo, preservation of wild-type huntingtin (wtHTT) protein, and generally safe and well-tolerated profile achieved in 30 mg multidose cohort Statistically significant correlation between mHTT lowering and slowing of caudate atrophy - an imaging biomarker predictive of clinical...Read more
First patient dosed with AllocetraTM in thumb osteoarthritis, a degenerative, debilitating and progressive disease that affects millions of people Thumb osteoarthritis currently has no FDA-approved therapy and no effective long-term treatments Ness-Ziona, Israel, June 24, 2024 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced...Read more
Company to begin enrollment of patients aged 1-3 years Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design Based on recent commercial landscape, confirmed accelerated approval pathway remains available given ongoing unmet need and RGX-202 differentiated design Remains on track to...Read more
All 12 patients who received the full dose of VX-880 as a single infusion demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90 All patients achieved ADA-recommended target HbA1c levels <7.0% and >70% time-in-range (70-180 mg/dL), and 11 of 12 patients reduced or eliminated use of exogenous insulin 3 patients with at least 12 months of follow-up, and therefore evaluable, met the primary endpoint...Read more
Established Medium Dose as Safe and Tolerable Dose in Current OCU410ST Clinical Trial DSMB Determination to Proceed with High Dose Cohort Dosing MALVERN, Pa., June 21, 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 Data and Safety Monitoring Board (DSMB)...Read more
SOLANA BEACH, Calif., June 20, 2024 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine, today congratulates its partner Aspen Neuroscience, Inc. on use of the ClearPoint Neuro Navigation System to transplant dopaminergic neuron precursor cells (DANPCs) for all enrolled patients with Parkinson’s Disease (PD)...Read more
21 months median OS in amezalpat arm vs. 15 in control arm 50% (20/40) of patients on amezalpat arm remain in survival follow up 0.65 hazard ratio, maintained since 0.59 observed in primary analysis 10 months earlier Early and persistent separation of survival curves OS is the primary regulatory endpoint for first-line HCC Company to host webcast conference call today at 8:30am ET BRISBANE, Calif., June 20, 2024...Read more
ELE-101 is a patent-protected, synthetic formulation of psilocin, designed to offer the therapeutic benefits of psilocybin in a more consistent, controllable, and shorter treatment paradigm of approximately two hours. The Phase 2a part of the study will evaluate the safety, tolerability, subjective effects, and efficacy of a single intravenous (IV) dose of ELE-101 in 6-12 participants with Major Depressive Disorder (MDD). Results are...Read more
AUSTIN, Texas, June 18, 2024 (GLOBE NEWSWIRE) -- XBiotech (NASDAQ: XBIT) announced today data from its Phase 1/Phase 2 randomized, double-blind, placebo-controlled multi-center study for advanced pancreatic cancer. Known as 1-BETTER, the study examined Natrunix (anti-interleukin-1alpha) antibody in combination with an established chemotherapy regimen (ONIVYDE (ON) + 5-Fluorouracil (5FU) + Leucovorin (LV), a regimen that is already...Read more
High-dose NGN-401 has been well-tolerated, and low-dose NGN-401 continues to show a favorable safety profile Interim safety data presented at the International Rett Syndrome Foundation (IRSF) ASCEND Summit NEW YORK / Jun 18, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced...Read more
Durable improvements across consistent clinical domains in both adult and pediatric patients, including motor skills, communication/socialization, autonomic function, seizures, and an encouraging safety profile seen across adult (up to 52 weeks) and pediatric (up to 22 weeks) patients with different genetic mutation severity Longer-term data from both adult patients showed sustained and new improvements across multiple efficacy...Read more
Overall response rate of 90% in cohort with durable responses observed; one patient remains in complete remission at 31 months All patients were heavily pretreated/refractory to BTK inhibitors, and only one patient has started new anti-WM treatment after MB-106 Outpatient administration was allowed and found to be feasible Currently no FDA-approved CAR-T treatments for WM Data presented at the European Hematology Association 2024...Read more
Key Highlights Enrolled patients with severe knee osteoarthritis who were indicated for knee-replacement surgery received a single local injection of AllocetraTM into the knee as a “last resort” Three-month data readout showed a significant reduction in pain and a favorable safety profile Pain reduction: patients reported an average pain reduction of 64% from baseline Complete Pain Resolution: 33% of patients reported complete...Read more
At 14-months median follow-up in the pivotal trial, 50% of patients achieved a complete response or better and a 71% overall response rate, as presented in an EHA oral presentation and simultaneously published in the Journal of Clinical Oncology Also presented at EHA, a retrospective study of patient outcomes that compared investigational linvoseltamab to real-world standard-of-care treatment in clinical practice TARRYTOWN, N.Y.,...Read more
89% of CRc's were subjects with "poor prognosis" cytogenetics and/or mutations Current median durability of response (mDOR) of CRc's = 6 months and increasing (n=9) In 2nd line subjects (n=10) Annamycin in combination with Cytarabine (AnnAraC) achieved an estimated median overall survival (mOS) of 6 months and increasing plus 5 CR's (50%) and 6 CRc's (60%) Recruitment increased to 22 subjects and CRc's in all subjects...Read more
Data continue to support revumenib's potential to enhance current standard of care agents 96% CRc (23 of 24 pts) observed in BEAT AML trial exploring revumenib in combination with venetoclax/azacitidine in newly diagnosed mNPM1 or KMT2Ar AML 52% CRc (14 of 27 pts) observed in AUGMENT-102 trial of revumenib in combination with fludarabine-cytarabine in acute leukemia patients with R/R mNPM1, NUP98r or KMT2Ar WALTHAM, Mass., June 14,...Read more
All patients treated in the RUBY trial are free of vaso-occlusive events post-renizgamglogene autogedtemcel (reni-cel) infusion Patients had early normalization of total hemoglobin with a mean within the normal range at >14 g/dL and rapid and sustained improvements in fetal hemoglobin well above levels of >40%. Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan...Read more
All patients treated in the EdiTHAL trial maintained hemoglobin levels above the transfusion threshold and are transfusion-free post-renizgamglogene autogedtemcel (reni-cel) infusion Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant EHA EdiTHAL poster presentation on Friday, June 14 at 6 p.m. CEST/Noon...Read more
No CRS, ICANS, infections or serious adverse events observed in either of the first two patients through data cut-off of May 28, 2024 CABA-201 exhibited anticipated profile of CAR T cell expansion and contraction with complete B cell depletion observed in both patients by day 15 post-infusion Improvements in both patients’ specific disease measures, consistent with academic experience of a similar 4-1BB CD19-CAR T, suggest emerging...Read more
Phase 1b/2a open-label trial in the U.S. of certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers BASKING RIDGE, N.J., June 13, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, announced...Read more
Avidity plans to accelerate the initiation of registrational cohorts in FORTITUDE™ trial Delpacibart braxlosiran (AOC 1020), the first investigational therapy to target the underlying cause of FSHD, provided greater than 50% mean reduction across multiple DUX4 regulated gene panels (2 mg/kg at four months) Trends of functional improvement including muscle strength, reachable workspace, and positive trends in patient and clinician...Read more
Bexicaserin achieved an overall median seizure reduction of 56.1% in countable motor seizures over an approximate 6-month treatment period; participants randomized to the PACIFIC placebo group achieved a median seizure reduction of 57.3% Favorable safety and tolerability results observed 100% of participants who completed the PACIFIC Study entered the OLE End of Phase 2 Meeting scheduled for this summer LA JOLLA, Calif. / Jun 10,...Read more
CAMBRIDGE, Mass. & MONTREAL / Jun 10, 2024 / Business Wire / Repare Therapeutics Inc. (“Repare” or the “Company”) (Nasdaq: RPTX), a leading clinical-stage precision oncology company, today announced the first patient has been dosed in the Company’s camonsertib monotherapy non-small cell lung cancer (NSCLC) expansion of the TRESR clinical trial. “Camonsertib has demonstrated a promising signal of prolonged progression free survival in...Read more
Company expects to lock database in June and remains on track to report key outcomes later in July. Pepinemab targets astrocyte reactivity and neuroinflammation, believed to be key drivers of neurodegeneration. ROCHESTER, N.Y., June 06, 2024 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the...Read more
IDMC recommendation to advance based on favorable safety profile observed in Phase 1a portion of trial WALTHAM, Mass., June 6, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it has advanced into the Phase 1b portion of its Phase 1/2 proof-of-concept trial of revumenib, the Company's highly...Read more
REDWOOD CITY, Calif., June 06, 2024 (GLOBE NEWSWIRE) -- Biomea Fusion, Inc. (“Biomea” or the “Company”) (Nasdaq: BMEA), announced that the Company has received notice from the U.S. Food and Drug Administration (FDA) that a full clinical hold has been placed on Biomea’s ongoing Phase I/II clinical trials of the Company’s investigational covalent menin inhibitor BMF-219 in type 2 and type 1 diabetes (COVALENT-111 and COVALENT-112),...Read more
100% of Participants (n=6) With CAH Maintained Androstenedione (A4) Below the Upper Limit of Normal at all Time Points on Atumelnant (80 mg) CAH Participants Achieved More Than a 90% Reduction of A4 and 97% Reduction of 17-OHP on Atumelnant (80 mg), Beginning at Two Weeks and Sustained Through 12 Weeks In the ADCS Trial, Atumelnant (80 mg) Normalized 24-hr Urinary Free Cortisol Levels for 100% of Participants (n=5) During Treatment...Read more
After More Than Four Years of Follow-up in the Pivotal ZUMA-3 Study, Median Overall Survival (OS) was 26 Months and the OS Rate was 40% at 48 Months Survival Benefit was Seen Regardless of Age, Prior Treatment or Subsequent Allogeneic Stem Cell Transplant Status Data Being Presented at the 2024 American Society of Clinical Oncology Annual Meeting SANTA MONICA, Calif. / Jun 03, 2024 / Business Wire...Read more
Phase 1/2 pivotal study met the primary safety and efficacy endpoints 51% (18 out of 35) of patients achieved Complete Response, requiring no surgeries after treatment with PRGN-2012; complete responses have been durable beyond 12 months with median duration of follow up of 20 months as of data cutoff 86% of patients (30 out of 35) had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year...Read more
Preliminary analysis of data from the EPCORE™ NHL-2 study demonstrates patients with previously untreated follicular lymphoma (FL) who received epcoritamab in combination with rituximab-lenalidomide (R2) experienced a 95% overall response rate (ORR) Results from the optimization cohort of the EPCORE™ NHL- 1 study show that mitigation strategies led to a clinically significant reduction in rate of cytokine release syndrome (CRS) and...Read more
Data demonstrated promising monotherapy activity with olomorasib across a range of KRAS G12C-mutant solid tumors, including non-small cell lung cancer, and a tolerability profile in combination with pembrolizumab that is well-suited to first-line lung cancer development INDIANAPOLIS, June 1, 2024 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) today announced updated data from the Phase 1/2 clinical trial evaluating olomorasib as...Read more
Established Medium Dose as Safe and Tolerable Dose in Current OCU410 Clinical Trial DSMB Recommends Continuing with High-Dose Cohort Dosing with Concurrent Phase 2 Dosing MALVERN, Pa., May 31, 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 a positive outcome of...Read more
SAN DIEGO, May 30, 2024 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that enrollment has been completed and dosing initiated for the fifth dose cohort of its Phase 1/2 study of ONCT-534 for the treatment of patients with metastatic castration-resistant prostate cancer who are relapsed or refractory to...Read more
REDWOOD CITY, Calif., May 30, 2024 (GLOBE NEWSWIRE) -- Biomea Fusion, Inc. (“Biomea”) (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing oral covalent small molecules to treat and improve the lives of patients with metabolic diseases and genetically defined cancers, today announced the completion of enrollment of the first three dose expansion arms of COVALENT-111, with a total of over 260...Read more
BHV-1510 is a novel trophoblast cell surface antigen-2 (Trop-2) directed antibody drug conjugate (ADC) that has demonstrated a highly differentiated preclinical monotherapy efficacy profile, the potential for broader therapeutic margin than other Trop-2 ADCs currently in development, and synergistic affects when combined with anti-PD1 therapy Biohaven also entered into a clinical supply agreement with Regeneron to study the combination...Read more
ATRN-119 is the first and only macrocyclic ATR inhibitor in the clinic, with best in class potential On track to complete dose escalation in ABOYA-119 clinical trial and potentially generate initial human efficacy data in 2H 2024 Through the first 5 cohorts, ATRN-119 has been found to be safe and well tolerated with no dose limiting toxicities (DLTs) observed DOYLESTOWN, Pa., May 28, 2024 (GLOBE NEWSWIRE) -- Aprea Therapeutics,...Read more
FG-3246 and enzalutamide combination therapy led to a median radiographic progression free survival (rPFS) of 10.2 months in biomarker unselected patients FG-3246 demonstrated an acceptable safety profile; adverse events consistent with those observed in other antibody drug conjugate therapies with a MMAE payload Recommended Phase 2 dose of FG-3246 was established at 2.1 mg/kg every 3 weeks in combination with enzalutamide given at...Read more
Eight of nine patients responding to the treatment, plus the encouraging safety data observed in the RESOLVE Phase 1b/2a trial of EP-104GI, provided the catalyst for the trial expansion Early low-dose cohorts showing meaningful improvement in patient symptoms and biological evidence of disease Trial expansion allows more patients to be treated at higher doses and followed for up to 52 weeks, up from 24 weeks VICTORIA, BC, May 23,...Read more
83% (5/6) of patients achieved a confirmed partial response in cohort 1, the most mature dose level; one dose-limiting toxicity was observed, however, the dose level was subsequently cleared after additional patients were enrolled Follow up of patients in the additional dose and schedule cohorts is ongoing to determine the recommended Phase 2 dose for study expansion Poster presentation on Saturday, June 1, 2024 at the ASCO Annual...Read more
Trial's first and second cohorts maintaining signs of potential efficacy out to 24 weeks Data from the second cohort showed improvements in eosinophil counts and histological scores out to 12 weeks suggesting a dose response over the first cohort Data from the trial's third cohort suggest improvements in patient-reported symptoms and histological outcomes at 12 weeks EP-104GI showed an encouraging safety profile in all three...Read more
In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose Dependent 27% Mean Splicing Correction Across All Patients in the 5.4 mg/kg Cohort at 3 Months DYNE-101 Showed Improvement in Myotonia, Muscle Strength, and Timed Function Tests and in DM1-ACTIVc and MDHI Patient Reported Outcomes In Phase 1/2 DELIVER Trial, DYNE-251 Showed 3.2% Mean Unadjusted (7.6% Mean Muscle Adjusted) Dystrophin Expression at 6 Months in 10 mg/kg...Read more
MIAMI, May 16, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced the completion of the last patient’s final visit in the Company’s Phase 1b/2a clinical trial of AJ201 for the treatment of spinal and bulbar muscular atrophy (“SBMA”), also...Read more
Development of BIIB105, an investigational antisense oligonucleotide for amyotrophic lateral sclerosis (ALS), will be discontinued based on data from the Phase 1/2 ALSpire study Biogen and Ionis continue their long-standing commitment to developing therapies for ALS given the devastating impact of this progressive, fatal neurodegenerative condition CARLSBAD, Calif. and CAMBRIDGE, Mass., May 16, 2024 /PRNewswire/ -- Ionis...Read more
Development of BIIB105, an investigational antisense oligonucleotide for amyotrophic lateral sclerosis (ALS), will be discontinued based on data from the Phase 1/2 ALSpire study Biogen and Ionis continue their long-standing commitment to developing therapies for ALS given the devastating impact of this progressive, fatal neurodegenerative condition CAMBRIDGE, Mass. and CARLSBAD, Calif., May 16, 2024 (GLOBE NEWSWIRE) -- Biogen Inc....Read more
ION582 demonstrated consistent improvements across multiple functional domains in Angelman syndrome patients ION582 was safe and well tolerated at all dose levels Ionis plans to move ION582 into pivotal trial Detailed ION582 data to be presented at upcoming medical meeting CARLSBAD, Calif., May 16, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a...Read more
MALVERN, Pa., May 15, 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 dosing is complete in the second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease as a...Read more
92% (12/13) overall response rate (ORR) for relapsed/refractory AL Amyloidosis patients enrolled in NEXICART-1: 12 out of 12 patients not exposed to prior BCMA-targeted bispecific responded to NXC-201 (100% ORR), of which 9 out of 12 were complete responders (75% CRs) 1 patient with prior exposure to BCMA-targeted bispecific treatment did not respond Best responder duration of response was 28.0 months with response ongoing as...Read more
Preliminary data detailed in an ASGCT oral presentation include results for one of the youngest children in the world to receive a gene therapy for genetic deafness Ongoing Phase 1/2 CHORD trial is currently enrolling infants and children in the U.S., UK and Spain TARRYTOWN, N.Y., May 08, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the investigational gene therapy DB-OTO improved...Read more
Previously released ABACUS-1 clinical trial results reported KIO-301, a small molecule photoswitch, is safe, tolerable, and improved vision in patients with late-stage retinitis pigmentosa. Additional analysis of functional MRI data demonstrated a statistically significant increase in neural activity over baseline within the brain's visual processing center. The increase in observed brain activity was time-dependent and...Read more
CAMBRIDGE, Mass., April 30, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced the approval of its first clinical trial application (CTA) for its RestorAATion-2 clinical trial of WVE-006, the company’s first-in-class RNA editing oligonucleotide, which is being developed for the treatment...Read more
OCALA, Fla., April 29, 2024 (GLOBE NEWSWIRE) -- AIM ImmunoTech Inc. (NYSE American: AIM) (“AIM” or the “Company”) today announced that it has taken an essential step forward in testing the combination of AIM’s Ampligen® (rintatolimod) and AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor Imfinzi® (durvalumab) in the treatment of late-stage pancreatic cancer (the “DURIPANC” study). See: ClinicalTrials.gov...Read more
Boca Raton, Florida, April 29, 2024 (GLOBE NEWSWIRE) -- INmune Bio, Inc. (NASDAQ: INMB) (the “Company”), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, continues to advance its Natural Killer (NK) cell therapy, INKmune™, in a Phase I/II trial for men with metastatic castration-resistant prostate cancer (mCRPC). The Company is pleased to...Read more
PASADENA, Calif. / Apr 24, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) announced today that it has dosed the first subjects in a Phase 1/2a clinical trial (NCT06209177) of ARO-CFB, the company’s investigational RNA interference (RNAi) therapeutic, in up to 66 healthy volunteers and patients with complement mediated kidney disease. James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at...Read more
Phase 1 part of combined Phase 1/2 study initiated as part of pandemic preparedness against highly pathogenic avian influenza (H5N1) virus, considered to be potential future pandemic threat Study will assess monovalent vaccine candidate, encoding an influenza A H5-antigen using proprietary second-generation mRNA backbone Avian influenza is latest program progressing to clinical trials under broad infectious disease collaboration...Read more
Nes-Ziona, Israel, April 22, 2024 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced that the first two patients have been dosed in the Company’s multi-country, randomized, controlled Phase I/II trial evaluating AllocetraTM in up to 160 patients with moderate to severe knee osteoarthritis. The Phase I/II multi-center trial is...Read more
MALVERN, Pa., April 19, 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 dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an...Read more
Primary safety endpoint met; VTP-200 was generally well-tolerated, with no treatment-related grade 3 or higher adverse events (AEs) or serious AEs (SAEs). Positive trends in clearance rate for both high-risk (hr)HPV (60%, Group 2) and cervical lesions (67%,Groups 2 and 5), were observed in the groups receiving the highest ChAdOx dose. Pooled data from the five different active dose groups demonstrated no statistically significant...Read more
SAN DIEGO, April 18, 2024 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that the first patient has been dosed in the fourth cohort of its Phase 1/2 study of ONCT-534 for the treatment of patients with advanced prostate cancer who are relapsed or refractory to approved androgen receptor pathway inhibitors...Read more
First efficacy signals demonstrated for a gene therapy under development for Oculopharyngeal Muscular Dystrophy (OPMD) which affects ~15,000 patients worldwide BB-301 facilitated improvements across multiple measures of swallowing function in the first Phase 1b/2a clinical study subject as compared to pretreatment assessments conducted during the observational natural history portion of the study Virtual R&D Day being held today...Read more
Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains; improvements consistent or exceeding Dose-escalation Cohorts data at Day 170 Additional long-term data in Dose-escalation Cohorts showed increasing and sustained clinical benefit through Day 758 Company will host investor call at 8:00 a.m. ET NOVATO, Calif., April 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)...Read more
STARMAP (Study Treating Acquired Resistance: MAPK Amplifications) is a first-in-human, 3-part, Phase 1/2 study of BBI-825 as a single agent and in combination with select targeted cancer therapies BBI-825 is a novel, oral, selective ribonucleotide reductase (RNR) inhibitor and Boundless Bio’s second extrachromosomal DNA (ecDNA)-directed therapy (ecDTx) to enter clinical development Preclinically, BBI-825 has demonstrated tumor growth...Read more
Twenty patients with indolent (n=18) and aggressive (n=2) R/R B-NHL received evorpacept plus standard rituximab and lenalidomide (“R2”) Evorpacept plus R2 was well tolerated with a safety profile similar to historical R2 The combination achieved promising initial activity with a best overall response rate (“ORR”) of 94% and a complete response rate (“CRR”) of 83% in patients with indolent R/R B-NHL (R2 historical CRR benchmark is...Read more
Late-breaking data to be featured in an oral presentation at the American Association for Cancer Research (AACR) annual meeting on Monday, April 8 and highlighted as part of the official meeting press program PRINCETON, N.J. / Apr 08, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced data from the cohorts of the Phase 1/ 2 KRYSTAL-1 study evaluating KRAZATI® (adagrasib) in combination with cetuximab for the...Read more
Favorable safety and immunogenicity profile in ABCC6 Deficiency, with clinical improvements in vascular pathology, visual function and patient reported outcomes (PROs) Natural history study highlights stroke as common feature among patients with early-onset ABCC6 Deficiency Favorable safety, immunogenicity and clinical outcome data were maintained through 48 weeks in Cohorts 1-3 in ENPP1 Deficiency; Data from Cohort 4...Read more
SAN DIEGO, April 07, 2024 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel portfolio of oncolytic immunotherapies, today announced the presentation of interim results from ARTACUS, a Phase 1/2 clinical trial evaluating RP1 monotherapy for the treatment of skin cancers in patients who have had solid organ or hematopoietic cell transplants, by Michael R....Read more
Established Low Dose as Safe and Tolerable Dose in Current OCU410 Clinical Trial DSMB Approval to Proceed with Medium Dose Cohort Dosing Malvern, Pa., April 05, 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 Data and Safety Monitoring Board (DSMB) for the...Read more
Improved VELOS™ manufacturing process delivering higher cNeT doses Protocols updated to evaluate the benefit of enhanced host conditioning, with further data expected in 2H 2024 First three patients dosed in CHIRON and THETIS with enhanced host chemo-conditioning, along with IL-2 aligned to standard TIL therapy, show improved cNeT persistence and engraftment LONDON, April 04, 2024 (GLOBE NEWSWIRE) -- Achilles...Read more
Conducted initial pivotal study interactions with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA); Company has clear registration path for 4D-710 for treatment of cystic fibrosis (CF) lung disease in people with CF (pwCF) who are ineligible for or cannot tolerate approved CF modulator therapies AEROW clinical trial Phase 2 Expansion Cohort dose selection and initiation expected in Q2 2024, with...Read more
Deep Cyclic Inhibitor of the MAPK pathway demonstrated strong tumor growth inhibition in preclinical studies of RAF or RAS mutant tumors, both as monotherapy and in combination Phase 1 portion of the Phase 1/2a trial designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of IMM-6-415 and establish a candidate recommended Phase 2 dose (RP2D) Initial PK, PD and safety data expected in...Read more
Phase 1/2a End of Study Data: 70mg doses demonstrated substantial and sustained reductions in convulsive seizure frequency on top of the best available anti-seizure medicines; Median reductions of 85% (n=10) at 3 months and 74% (n=9) at 6 months after last dose Open Label Extension Studies: Durable reductions in seizures and clinically meaningful improvements in multiple measures of cognition and behavior were maintained over 12...Read more
STAR-0215 Dosed Once or Twice Over 6 Months Reduced Monthly Attack Rates by 90-96%, Supporting Chronic Dosing 2 or 4 Times Per Year 92-100% Decrease in Moderate or Severe Attacks and 91-95% Reduction in Attacks Requiring Rescue Medications with STAR-0215 Very Well-Tolerated with No Serious Adverse Events and No Discontinuations Phase 3 Initiation on Track for Q1 2025, with Top-Line Results Expected by Year End...Read more
Annamycin in combination with Cytarabine (AnnAraC) achieves CRc rate of 60% (50% CR) in 2nd line AML subjects AnnAraC has potential to fill significant unmet need and expected to increase 2nd line AML CRs more than double over existing 2nd line therapies All 82 Annamycin subjects (in multiple studies) continue to show no signs of cardiotoxicity during study; Lower toxicity profile than traditional intensive therapy Annamycin is...Read more
Rapid improvements demonstrated for proptosis, clinical activity scores, and diplopia versus placebo with a favorable safety profile Phase 2b/3 trial to be initiated in the second half of 2024 Conference call to review these data to be held at 8:30 a.m. ET today LOS ANGELES, March 20, 2024 (GLOBE NEWSWIRE) -- ACELYRIN, INC. (Nasdaq: SLRN), a late-stage clinical biopharma company focused on accelerating the development and delivery...Read more
REDWOOD CITY, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) in mast cell driven diseases such as chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CIndU), today announced that the first patient has been dosed in Jasper’s Phase 1b/2a (SPOTLIGHT) clinical study of...Read more
NUV-1511 is the Company’s first drug-drug conjugate (DDC) to enter the clinic NEW YORK / Mar 14, 2024 / Business Wire / Nuvation Bio Inc. (NYSE: NUVB), a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, today announced that the first patient has been dosed in a Phase 1/2 study of NUV-1511, the Company’s first DDC to enter the clinic. “Dosing...Read more
IMM-1-104 has been well-tolerated, demonstrating the potential for a differentiated safety profile 100% suppression of acquired RAS alterations was observed in evaluable patients profiled for ctDNA and treated with IMM-1-104, supporting goal of Universal-RAS activity Target lesion regression observed in over half of patients treated with IMM-1-104 at 320mg or 240mg QD, with best individual lesion regression of -35.7% and...Read more
Vancouver, Canada, March 13, 2024 (GLOBE NEWSWIRE) -- Clearmind Medicine Inc. (Nasdaq, CSE: CMND), (FSE: CWY) (“Clearmind” or the "company"), a clinical- stage biotech company focused on discovery and development of novel psychedelic-derived therapeutics to solve major under-treated health problems, announced that it has received approval from the Israeli Ministry of Health to commence its phase I/IIa clinical trial for alcohol use...Read more
C4 Therapeutics is pioneering a new class of small-molecule drugs that selectively destroy disease-causing proteins via degradation using the innate machinery of the cell. This targeted protein degradation approach offers advantages over traditional drugs, including the potential to treat a wider range of diseases...
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