Median progression free survival (mPFS) of 7.4 months in the ZFA expansion cohort evaluating zotatifin in combination with fulvestrant and abemaciclib in heavily pretreated patients Zotatifin generally well tolerated as dose escalation continues; Currently enrolling at 0.28 mg/kg in the ZF doublet evaluating zotatifin combined with fulvestrant and at 0.1 mg/kg in the ZFA triplet SOLANA BEACH, Calif. and REDWOOD CITY, Calif.,...Read more
71% objective response rate, with 46% achieving a complete response or better after 11 months of median follow-up Data to be submitted to regulatory authorities, with Biologics License Application on track to be submitted to the FDA this year Regeneron to host virtual investor event to discuss results alongside updates across its hematology portfolio on Thursday, December 14 at 8:30 a.m. ET TARRYTOWN, N.Y., Dec. 07, 2023 (GLOBE...Read more
RP1 in combination with cemiplimab demonstrated clinically meaningful improvements in complete response rate and duration of response compared to cemiplimab in the CERPASS clinical trial, but did not meet either of the two primary endpoints Positive data update for full 140 patients in the IGNYTE clinical trial cohort of RP1 in anti-PD1 failed melanoma reinforces durable benefit; biologics license application (BLA) submission planned...Read more
Confirmed partial responses without dose-limiting toxicities during dose escalation along with new preclinical data support the opportunity to further enhance efficacy through a novel split daily dosing regimen at higher dose levels of Nana-val Enrollment is underway for the sixth dose cohort of the Phase 1b dose escalation portion of the study with plans to initiate a recommended Phase 2 dose-optimization cohort as part of the study’s...Read more
REDWOOD CITY, Calif., Nov. 30, 2023 (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), as well as lower to intermediate risk myelodysplastic syndromes (LR-MDS) and novel stem cell transplant...Read more
HAYWARD, Calif., Nov. 30, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or the “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced the first subject has been dosed in the BB-301 Phase 1b/2a Clinical Treatment Study. BB-301 is the Company’s...Read more
First patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin On track for pivotal dose determination and initiation of pivotal program in 2024 ROCKVILLE, Md., Nov. 29, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the first patient received RGX-202 at dose level 2 in the Phase I/II...Read more
BX004 showed clinically meaningful improvement in pulmonary function as measured in FEV11 and Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain in a predefined subgroup of patients with reduced lung function2 Treatment with BX004 also achieved P. aeruginosa culture conversion to negative at end of 10 days of treatment in 3 patients (14%) versus none for placebo3 In a prespecified subgroup of patients on background use...Read more
Cohort 9 Following completion of the 21-day observation period at 3.4 mg/kg (Cohort 9), no dose limiting toxicities (DLTs) or serious adverse events (SAEs) were observed Two patients in Cohort 9 experienced rapid PSA reduction at three weeks post-treatment following the first ARX517 dose Cohort 8 The cohort (2.88 mg/kg) is now fully enrolled with 20 patients Recommended phase 2 dose expected by early next year SAN DIEGO,...Read more
SEATTLE, Nov. 16, 2023 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. (NYSE AMERICAN: CATX), today announced the completion of cohort 1 dosing in the Phase 1/2a clinical trial of [212Pb]VMT-α-NET in patients with unresectable or metastatic somatostatin receptor type 2 (SSTR2)-expressing neuroendocrine tumors (NETs). This milestone follows the recent completion of the first dose escalation cohort for Perspective’s melanoma therapeutic...Read more
MALVERN, Pa., Nov. 10, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the first patient has been dosed in its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease, a...Read more
VAX-31, a 31-Valent Pneumococcal Conjugate Vaccine (PCV) Candidate, is the Broadest-Spectrum PCV to Enter the Clinic Topline Safety, Tolerability and Immunogenicity Data Expected in Second Half of 2024 VAX-31 is Designed to Provide Coverage for Approximately 95% of Invasive Pneumococcal Disease Circulating in the U.S. Adult Population SAN CARLOS, Calif., Nov. 09, 2023 (GLOBE NEWSWIRE) Vaxcyte, Inc. (Nasdaq: PCVX), a vaccine...Read more
Pelareorep-atezolizumab combination data exceeds historical control trials with a 37.5% objective response rate, including a complete response SAN DIEGO and CALGARY, Alberta, Nov. 9, 2023 /CNW/ -- Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC), a clinical-stage immunotherapeutics company focused on oncology, today announced the presentation of positive, interim results from the Phase 1/2 GOBLET study evaluating the...Read more
SEATTLE, Nov. 08, 2023 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. (“Perspective” or “the Company”) (NYSE AMERICAN: CATX), announced today that the first patient was dosed at Washington University in St Louis in the Company’s Phase 1/2a trial evaluating the safety and efficacy of [212Pb]VMT-α-NET, a targeted alpha-particle therapy (TAT), in patients with unresectable or metastatic somatostatin receptor type 2 (SSTR2)...Read more
MDNA11 continues to demonstrate encouraging single-agent activity from the dose escalation and evaluation portion of the ABILITY-1 Study including deep ongoing partial responses with 100% reduction of target lesions in one pancreatic and 70% reduction of target lesion in one melanoma cancer patient MDNA11 also showed durable stable disease in 3 melanoma patients for at least 5 months to 18 months with concomitant shrinkage of tumor...Read more
23andMe presented data from the now completed dose escalation phase, and pharmacokinetic / pharmacodynamic (PK/PD) cohorts at the Society for Immunotherapy of Cancer Annual Meeting 2023 Dosing with 23ME-00610 monotherapy in 28 patients with advanced solid tumors up to 1400 mg every three weeks (Q3W) showed tolerability consistent with expectation for on-target effects Peripheral pharmacodynamic data demonstrate that 23ME-00610...Read more
Topline Phase I/II First-in-Human Results Presented at AAO 2023 Significant improvement in visual field, in concordance with trended improvements in visual acuity and functional vision Functional MRI demonstrates increased brain activity in visual cortex Novel small molecule photoswitch is safe and tolerable Company presentation available on IR website (ir.kiorapharma.com) Encinitas, California--(Newsfile Corp. - November 4,...Read more
WOBURN, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel portfolio of oncolytic immunotherapies, today announced updated 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. The data were presented...Read more
Initial evidence of dose-dependent disease control rate with 50% disease control rate at higher doses (≥2.8 mg/kg) and 31% disease control rate across all dose levels in a range of solid tumor types, including cold tumors Treatment-related adverse events primarily Grade 1-2 at doses up to 4 mg/kg administered once every three weeks in outpatient setting, with no reported signs or symptoms of vascular leak syndrome Two patients...Read more
Clinical dose escalation data for SNS-101 monotherapy show well tolerated safety profile, potentially best-in-class pharmacokinetics, and encouraging cytokine release profile across multiple dose cohorts First VISTA-blocking antibody administered at a dose anticipated to be therapeutically relevant without eliciting dose-limiting toxicity Monotherapy data from Phase 1/2 study to be presented in a late-breaking poster presentation at...Read more
Brian Van Tine, M.D., of Washington University School of Medicine, shares data from the ongoing phase 1/2 KB-0742 clinical study that clearly corresponds with the findings from the pre-clinical studies at leading international sarcoma meeting KB-0742 demonstrated on-mechanism, single agent anti-tumor activity and a manageable safety profile in heavily pre-treated patients with transcriptionally addicted solid...Read more
Initiated single non-human primate (NHP) safety study evaluating intravenous 4D-310 combined with rituximab/sirolimus (R/S) immunosuppressive regimen Amended INGLAXA protocol to minimize risk of atypical hemolytic uremic syndrome (aHUS) associated with intravenous (IV) AAV dosing, including addition of R/S immunosuppressive regimen 4D-310 combines a novel, targeted next generation AAV vector (C102) and GLA transgene for a single low...Read more
All six patients in the first cohort (Cohort A) were successfully implanted with the 8-channel Cell Pouch System with post-transplant follow-up periods ranging from 6 months to 3.5 years; 5 of 6 patients in Cohort A discontinued insulin therapy (insulin independent) following islet transplantation into the Cell Pouch and modest islet top-up via portal vein. All 6 patients achieved HbA1c values in the non-diabetic range (<6.5%); In...Read more
Lead formulations evaluated in the Phase 1/2 study demonstrated robust immune responses to influenza A, influenza B, and SARS-CoV-2 strains Safety profile of the mRNA-based combination vaccine candidates consistent with the companies’ COVID-19 vaccine The companies plan to start a pivotal Phase 3 trial in the coming months NEW YORK & MAINZ, GERMANY / Oct 26, 2023 / Business Wire / Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq:...Read more
Global Phase 1/2 CHORD trial investigating otoferlin gene therapy (DB-OTO) represents Regeneron’s first auditory program and is currently enrolling patients DB-OTO is part of a growing pipeline of genetic medicines for hearing loss and other therapeutic areas that are being advanced by the company TARRYTOWN, N.Y., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced preliminary, positive...Read more
BNT211 combines two innovative approaches in one regimen with first-in-class potential: an autologous CAR-T cell therapy targeting the oncofetal antigen Claudin-6 (CLDN6) and a CLDN6-encoding CAR-T cell amplifying RNA vaccine (“CARVac”) Data presented at ESMO Congress 2023 demonstrates that the application of CARVac increases the persistence of the adoptively transferred autologous CAR-T cells BNT211 continues to show encouraging...Read more
37% ORR and 14.9 months median DOR in 78 evaluable NRG1+ NSCLC patients 42% ORR and 9.1 months median DOR in 33 evaluable NRG1+ PDAC patients Sufficient clinical data expected in 1H24 to support potential BLA submissions UTRECHT, The Netherlands and CAMBRIDGE, Mass., Oct. 23, 2023 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS) (“Merus”, “the Company”, “we”, or “our”), a clinical-stage oncology company developing innovative,...Read more
Data presented at the European Society for Medical Oncology from the Phase 1/2 trial with bavdegalutamide showed 11.1 months radiographic progression free survival in mCRPC patients with tumors harboring AR 878/875 mutations Interim data from the Phase 1/2 trial of Arvinas’ second PROTAC AR degrader, ARV-766, showed robust efficacy in a broader mCRPC patient population with a tolerability profile well-suited to both early- and...Read more
Confirmed response rate 35% (11/31), across all tumor types and patient cohorts treated with 1 mg priming dose, including three confirmed, complete responses 32% (6/19) confirmed response rate in patients with small cell lung cancer (SCLC), including one confirmed complete response 42% (5/12) confirmed response rate in other neuroendocrine tumor types, including two confirmed complete responses Generally well tolerated;...Read more
The MRD trial will evaluate the effect of this treatment combination on minimal residual disease. LAWRENCEVILLE, N.J., Oct. 18, 2023 (GLOBE NEWSWIRE) -- IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage drug-development company focused on developing non-viral DNA-mediated immunotherapy and next-generation vaccines, announces that the first patient in a Phase 1/2 clinical trial evaluating IMUNON’s IMNN-001 in combination with bevacizumab...Read more
BDC-3042, a novel Dectin-2 agonistic antibody that stimulates the innate immune system, is being evaluated in a Phase 1/2 dose-escalation and expansion study in patients with a broad range of solid tumors REDWOOD CITY, Calif., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Bolt Biotherapeutics (Nasdaq: BOLT), a clinical-stage biopharmaceutical company developing novel immunotherapies for the treatment of cancer, today announced that the first...Read more
Optimal levels of degradation of GSPT1 in peripheral blood mononuclear cells and tumors observed at all doses, consistent with preclinical studies Tumor size reductions observed in patients with biomarker-positive tumors Safety profile supports further clinical development of MRT-2359 Conference call and webcast at 8:00 a.m. ET today BOSTON, Oct. 17, 2023 (GLOBE NEWSWIRE) -- Monte Rosa Therapeutics, a clinical-stage biotechnology...Read more
The Combination Arm (Part B) of the Phase 1/2 Clinical Trial Builds Upon the Initial Safety, Tolerability and Pharmacokinetic Data of KVA12123 in the Monotherapy Arm (Part A) Initial Combination Therapy Clinical Data Anticipated in Q2 2024 SEATTLE, Oct. 17, 2023 (GLOBE NEWSWIRE) -- Kineta, Inc. (Nasdaq: KA), a clinical-stage biotechnology company focused on the development of novel immunotherapies in oncology that address cancer...Read more
Meaningful antitumor activity observed in recurrent/metastatic HRAS mutant HNSCC, including CR Data validate activity of farnesyltransferase inhibitors and support rapid advancement of KO-2806 into combinations with targeted therapies in large solid tumor indications Results to be presented in late-breaking oral session at 2023 ESMO Congress on Saturday SAN DIEGO, Oct. 17, 2023 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq:...Read more
SEATTLE, Oct. 16, 2023 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. (“Perspective” or “the Company”) (NYSE AMERICAN: CATX), today announced the completion of recruitment for the first patient cohort in its Phase 1/2a dose escalation study of 212Pb-VMT01 (clinicaltrials.gov identifier NCT05655312), its targeted alpha-particle therapy, in development for the treatment of MC1R-positive metastatic melanoma. “We are encouraged by...Read more
Avutometinib + Sotorasib Combination Demonstrated Preliminary Efficacy with Confirmed Responses in both KRAS G12C Inhibitor Resistant and Naïve Patients No New Safety Signals Observed in Combination; Most Treatment-Related Adverse Events Mild to Moderate Enrollment of Patients Naïve to or Previously Treated with a KRAS G12C Inhibitor Ongoing in Expansion Phase BOSTON / Oct 14, 2023 / Business Wire / Verastem Oncology (Nasdaq: VSTM)...Read more
LOS ANGELES / Oct 14, 2023 / Business Wire / Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, announced the poster presentation of a Phase I/II trial of alisertib plus pembrolizumab for the treatment of patients with Rb-deficient head and neck squamous cell carcinoma (Clinicaltrials.gov identifier NCT04555837) at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston,...Read more
Data demonstrate preclinical potent T cell activation and differentiated mechanism of action and enhanced clinical efficacy irrespective of PD-L1 status when administered in combination with chemotherapy Toripalimab demonstrated twelve-fold higher binding affinity to PD-1 compared to pembrolizumab In vitro studies show significantly higher activation of T cells compared to pembrolizumab in multiple assay systems REDWOOD CITY,...Read more
Demonstrated on-mechanism, single agent anti-tumor activity in heavily pre-treated patients with transcriptionally addicted solid tumors Showed manageable safety profile, with no grade 3/4 neutropenia, dose proportional exposure, dose-dependent target engagement, and 24-hour plasma half-life Dose escalation continues; dosing of patients at the 80 mg dose level is ongoing Enrollment ongoing in dose expansion phase in...Read more
NEW YORK, Oct. 11, 2023 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that its partner GenFleet Therapeutics (Shanghai), Inc. has dosed the first patient in a Phase Ib/II trial evaluating SLS009 (GFH009) in relapsed/refractory Peripheral...Read more
First study of patients with metastatic prostate cancer to evaluate standard-of-care chemotherapy (docetaxel) combined with an antibody drug conjugate (PDS0301). Decrease in prostate specific antigen (PSA) levels was seen in all patients at all three tested doses of PDS0301. The combination was well-tolerated at all tested dose levels. Data to be presented by National Cancer Institute as an oral presentation at Cytokines...Read more
Preliminary data from the first cohort demonstrates that EP-104GI was well tolerated with no drug-related adverse events reported VICTORIA, BC, Oct. 11, 2023 /CNW/ - Eupraxia Pharmaceuticals Inc. ("Eupraxia" or the "Company") (TSX: EPRX), a Phase 2 clinical-stage biotechnology company with an innovative drug delivery technology, today announced the initiation of the second cohort for its Phase 1b/2a clinical trial in eosinophilic...Read more
New data package reveals improvements in multiple categories for TPST-1120 combined with atezolizumab + bevacizumab versus standard of care atezolizumab + bevacizumab in a global Phase 1b/2 study 30% confirmed ORR achieved in TPST-1120 arm compared to 13.3% for atezolizumab + bevacizumab in the control arm, a substantial increase specific to the TPST-1120 arm compared to the previous data cut of 17.5% versus 10.3% in the control...Read more
Company reaches next clinical trial milestone for the intramuscular treatment of Chronic Lower Back Pain with the first novel allogenic cellular therapy in the United States PHOENIX, Oct. 10, 2023 /PRNewswire/ -- Creative Medical Technology Holdings, Inc. ("Creative Medical Technology" or the "Company") (NASDAQ: CELZ), a biotechnology company focused on a regenerative approach to immunotherapy, endocrinology, urology, gynecology, and...Read more
SAN DIEGO, Oct. 05, 2023 (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 Phase 1/2 dose escalation/dose expansion study of ONCT-534, the company’s dual-action androgen receptor inhibitor. “The dosing of the first patient with ONCT-534 is an important milestone for...Read more
CARLSBAD, Calif. / Oct 05, 2023 / Business Wire / Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that the results of imaging analyses demonstrating rapid improvement in outer retinal structure from patients enrolled in a Phase 1/2a clinical study of RG6501 (OpRegen) (ClinicalTrials.gov Identifier:...Read more
mRNA-1083 showed strong immunogenicity against influenza and COVID-19, with an acceptable reactogenicity and safety profile, compared to licensed standalone vaccines Company to begin Phase 3 trial of mRNA-1083 in adults 50 years and above CAMBRIDGE, MA / ACCESSWIRE / October 4, 2023 / Moderna, Inc. (NASDAQ:MRNA) today announced positive interim results from the Phase 1/2 trial of mRNA-1083, an investigational combination vaccine...Read more
Robust increases of up to 700-fold over baseline in plasma GCase enzyme activity in first two patients treated with FLT201 Normalization of leukocyte GCase in both patients demonstrates cellular uptake from plasma FLT201 has been well tolerated, with no serious adverse events Company to host conference call today at 8 a.m. ET LONDON, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today...Read more
Company remains on track to report Part 2 results from the study in November 2023 Part 1 data of the Phase 1b/2a study accepted for oral presentation at upcoming IDWeek 2023 CAMBRIDGE, Mass. and NESS ZIONA, Israel, Oct. 04, 2023 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria,...Read more
All patients treated with VX-880 in Parts A and B have follow-up data beyond Day 90 and have demonstrated islet cell engraftment and glucose-responsive insulin production All patients showed improvement across all measures of glucose control, including decreases in HbA1c, increases in blood glucose time-in-range, and reduction or elimination of insulin use The two patients with at least 1 year of follow-up met the criteria for the...Read more
Anti-tumor activity noted in the highest dose cohort of NT219 in combination with cetuximab; 2 out of 4 SCCHN patients at the highest dose cohort demonstrated a confirmed partial response NT219 exposure at the highest dose level reaching human equivalent dose for efficacy based on animal models Target engagement was confirmed in patients’ tissue biopsies Phase 2 study of NT219 in combination with cetuximab in 2L SCCHN, is...Read more
Cleared First Three Monotherapy Cohorts with No Dose Limiting Toxicity and No Consistent Pattern of Adverse Events at any Dose Level >90% VISTA Receptor Occupancy Observed in the 30 mg Dosing Cohort VISTA Blocking KVA12123 Engineered to Provide Strong Single Agent Anti-Tumor Activity While Minimizing Cytokine Related Adverse Events Clinical Trial is Advancing to Higher Monotherapy Dose Levels and in Combination with Pembrolizumab...Read more
100% (9/9) overall response rate and 67% (6/9) complete response rate (MRD 10-5) observed in heavily pre-treated patients with daratumumab relapsed/refractory AL Amyloidosis as of the September 20, 2023 data cutoff 100% (4/4) overall response rate and 75% (3/4) complete response rate observed in t(11;14) relapsed/refractory AL Amyloidosis Best responder duration of response was 19.2 months with response ongoing; median follow-up of...Read more
Trial met its primary endpoint with a CR/CRh rate of 23% at interim analysis of the pooled KMT2Ar AML and ALL cohorts (p-value = 0.0036); an additional 14% of patients proceeded to transplant without achieving CR/CRh 65% (32/49) overall response rate in KMT2Ar AML; CR/CRh rate of 24.5% 50% (7/14) of transplanted patients have initiated, and 21% (3/14) of transplanted patients could initiate revumenib as post-transplant...Read more
Intent-to-treat population shows 9-month OS improvement over control arm Subgroup of patients treated with IMNN-001 + PARPi shows meaningful PFS and OS trend compared with control arm Continued follow-up is indicated to confirm initial observations LAWRENCEVILLE, N.J., Sept. 28, 2023 (GLOBE NEWSWIRE) -- IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage biotechnology company focused on developing DNA-mediated immunotherapies and...Read more
Eleven patients enrolled overall in trial to date, with enrollment progressing faster than anticipated Monotherapy arm has advanced to a fourth cohort, with initial monotherapy PK and safety data expected in the fourth quarter of 2023 and topline monotherapy data expected in 2024 New guidance for initial combination PK and safety data expected in the first quarter of 2024, with preliminary anti-tumor activity data expected...Read more
SAN DIEGO and SUZHOU, China and SHANGHAI, China, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Gracell Biotechnologies Inc. ("Gracell" or the "Company", NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing innovative and highly efficacious cell therapies for the treatment of cancer and autoimmune disease, today announced that the first patient has been dosed in a Phase 1b/2 clinical trial evaluating its...Read more
Data from ongoing trial suggest clinical benefit for ENPP1 Deficiency, including improvement in key biomarkers, patient reported outcomes (PROs) and functional outcomes Improvement in the Global Impression of Change (GIC) observed in all three dose cohorts in ABCC6 Deficiency (PXE) trial INZ-701 was generally well tolerated and exhibited a favorable safety and immunogenicity profile in both trials Company to host...Read more
All 8 patients treated with OTX-2002 in initial two cohorts achieved highly specific on-target genomic engagement, intended epigenetic state change and robust downregulation in expression of c-MYC, a historically ‘undruggable’ target First-known clinical observation of pre-transcriptional gene modulation using a programmable epigenomic mRNA candidate Clinical proof-of-platform established; potential applicability across a broad range...Read more
Initiation of the Phase 2a portion follows DRC review of positive safety and tolerability data from recently completed Phase 1b portion Study being conducted in partnership with the U.S. Department of Defense LOS ANGELES, Sept. 26, 2023 /PRNewswire/ -- Armata Pharmaceuticals, Inc. (NYSE American: ARMP) ("Armata" or the "Company"), a biotechnology company focused on pathogen-specific bacteriophage therapeutics for...Read more
Available clinical data from the two adult patients dosed with TSHA-102 in the first cohort (low dose) to be discussed during upcoming quarterly earnings call following Independent Data Monitoring Committee (IDMC) review Dosing of third adult patient and completion of enrollment in the low-dose cohort expected in the fourth quarter of 2023 Dosing of first pediatric Rett syndrome patient expected in the first quarter of 2024 DALLAS,...Read more
Initiation of Clinical Study at Allegheny Health Network Seeks to Provide Treatment Options to Pancreatic Cancer Patients WESTON, Fla. and PITTSBURGH, Sept. 26, 2023 /PRNewswire/ -- Cantex Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed, and Allegheny Health Network...Read more
Chronic spinal cord injury cohort results expected in mid-2024 Subacute spinal cord injury cohort results expected in late 2024/early 2025 Vancouver, British Columbia--(Newsfile Corp. - September 25, 2023) - NervGen Pharma Corp. (TSXV: NGEN) (OTCQX: NGENF), a clinical stage biotech company dedicated to developing innovative solutions for the treatment of nervous system damage, today announced the first subject has been dosed in the...Read more
Data readouts expected before year end Previously reported preliminary efficacy data on its Phase 1B/2 clinical trial with Annamycin in monotherapy treatment of STS lung mets with >60% of the subjects exhibiting stable disease after two cycles of treatment Annamycin has Fast Track Status and Orphan Drug Designation from FDA for the treatment of soft tissue sarcoma HOUSTON, Sept. 21, 2023 /PRNewswire/ -- Moleculin Biotech, Inc.,...Read more
NEW YORK, Sept. 20, 2023 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that dosing is now underway in its Phase 1b/2 ADVANCED-2 trial evaluating intravesical instillation of TARA-002, the Company’s investigational cell-based therapy, for the treatment of high-grade non-muscle invasive bladder...Read more
Late-breaking data presented at 9th ESWI Influenza Conference in Valencia SAN DIEGO / Sep 19, 2023 / Business Wire / Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, today announced the results of a phase 1/2 study showing that...Read more
BioNTech is initiating a Phase 1/2 clinical trial of the mRNA-based mpox vaccine program, BNT166 The Coalition for Epidemic Preparedness Innovations (CEPI) commits funding of up to $90 million for the development of vaccine candidates Data generated by this partnership will contribute to CEPI’s 100 Days Mission, a global effort to accelerate the development of well-tolerated and effective vaccines against future viral threats with...Read more
SOUTH SAN FRANCISCO, Calif., Sept. 15, 2023 (GLOBE NEWSWIRE) -- Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immuno-oncology company developing novel T cell engagers, today announced dosing of the first patients with small cell lung cancer (SCLC) in an ongoing Phase 1/2 trial of HPN328, a DLL3 targeting TriTAC®, in combination with atezolizumab (Tecentriq®). Harpoon previously entered a Master Clinical Supply Agreement with...Read more
Favorable safety and tolerability profile of OCU400 investigational drug product in RP and LCA subjects to date Clinical study update suggests continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or...Read more
Post-hoc analysis from Phase 1b/2a study in pulmonary sarcoidosis presented at the European Respiratory Society (ERS) International Congress 2023. 7.7% of patients in the 3.0 and 5.0 mg/kg efzofitimod group relapsed following steroid taper, compared to 54.4% in the placebo and efzofitimod 1.0 mg/kg group (p=0.017). Rate of change for forced vital capacity (FVC) was significantly improved for the 3.0 and 5.0 mg/kg efzofitimod group...Read more
CAMBRIDGE, Mass., Sept. 05, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that in August, the first patient was treated with BEAM-201, a quadruplex-edited allogeneic CAR-T cell investigational therapy. BEAM-201 is being evaluated in a Phase 1/2 clinical study for the treatment of relapsed/refractory T-cell acute...Read more
AUSTIN, Texas, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, today announced treatment of the first patient in Part B (Cohort 4) of the ReSPECT-LM Phase 1/2a dose escalation clinical trial of rhenium (186Re) obisbemeda for the treatment of...Read more
Alignment with US Food and Drug Administration on a Recommended Phase 2 Dose for NVL-520 of 100 mg daily CAMBRIDGE, Mass., Sept. 5, 2023 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the initiation of the Phase 2 portion of ARROS-1, its Phase 1/2 clinical trial of...Read more
SEATTLE / Aug 30, 2023 / Business Wire / Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, announced the successful initiation of the second IgA nephropathy (IgAN) dose cohort in RUBY-3, a phase 1b/2a study in autoimmune glomerulonephritis. Safety Monitoring Committee (SMC) review has determined that repeat...Read more
Additional biomarker data up to two years of treatment continue to demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers Additional safety data up to two years of treatment continue to demonstrate that DNL310 is generally well tolerated Previously reported data showed robust and statistically significant reduction of NfL, a marker of neuronal...Read more
Enrolled 52 participants with Developmental and Epileptic Encephalopathies (DEEs) into the PACIFIC Study Participants have a broad range of DEEs including Lennox-Gastaut syndrome, Dravet syndrome, SCN2A-related epilepsies, CDKL5 deficiency disorder, among others PACIFIC topline results remain on track for around year-end 2023 LA JOLLA, Calif. / Aug 23, 2023 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a...Read more
BOSTON, MA / ACCESSWIRE / August 17, 2023 / Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company focused on novel biotherapeutics through its proprietary Anticalin® technology platform for respiratory diseases and cancer, today announced that the Company has achieved an undisclosed milestone payment from Boston Pharmaceuticals. The milestone is based on dosing the first patient in a Boston...Read more
Repotrectinib continued to demonstrate high response rates and durable responses, including robust intracranial responses, in patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer who were TKI-naïve or previously treated with one TKI and no chemotherapy Median duration of response and progression-free survival to be disclosed for the first time in the pooled Phase 1/2 population alongside updated results...Read more
Initial data show clinical responses from four of four indolent lymphoma patients, including complete response in follicular lymphoma patient previously treated with CD19 CAR-T cell therapy Aligns with ongoing results from investigator-sponsored trial at Fred Hutch that show ongoing complete remission for more than three years Update on data presented at 5th iwCAR-T, Scottsdale, AZ Safety Review Committee unanimously approved...Read more
Data from first adult patient dosed in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in key efficacy measures, including Clinical Global Impression – Improvement (CGI-I), Clinical Global Impression – Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), four weeks post-treatment Principal Investigator (PI) observed...Read more
First patient with MC1R-positive metastatic melanoma treated with 212Pb-VMT01 Preliminary data readout from dose escalation study expected Q4 2023 RICHLAND, Wash. & CORALVILLE, Iowa, Aug. 11, 2023 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. (“Perspective” or “the Company”) (NYSE AMERICAN: CATX), announces the first patient was dosed at the University of Wisconsin in the Company’s Phase 1/2a dose...Read more
TCER® IMA402 is the first next-generation, half-life extended TCR Bispecific targeting PRAME to enter the clinic Patient enrollment for IMA402 Phase 1/2 trial underway The trial will evaluate safety, tolerability, and anti-tumor activity of IMA402 in patients with recurrent and/or refractory solid tumors First clinical data expected in 2024 Tuebingen, Germany and Houston, Texas, August 10, 2023 – Immatics N.V. (NASDAQ:...Read more
Durable anti-cancer activity was observed during monotherapy dose escalation without dose-limiting toxicities Late-stage pancreatic cancer patient achieved 80% tumor shrinkage with complete regression observed in two of three metastatic lesions in the liver in response to single-agent MDNA11 Immune pharmacodynamic data continues to support a differentiated mechanism with preferential expansion of potent cancer-fighting immune...Read more
Recruitment of individuals with spinal cord injury (SCI) initiated at Shirley Ryan AbilityLab in Chicago Chronic SCI cohort results expected in mid-2024 and subacute SCI cohort results in late 2024/early 2025 Vancouver, British Columbia--(Newsfile Corp. - August 8, 2023) - NervGen Pharma Corp. (TSXV: NGEN) (OTCQX: NGENF), a clinical stage biotech company dedicated to developing innovative solutions for the treatment of nervous...Read more
Interim safety data up to 28 months showed Dose 1 and 2 of PBGM01 were well tolerated and had a favorable safety and immunological profile Dose 2 resulted in substantial improvements in key CSF biomarkers and was able to achieve normal levels of CSF β-Gal activity and GM1 gangliosides, similar to healthy controls Dose 2 biomarker responses demonstrated durability up to 12 months after treatment Imagine-1 study participants showed...Read more
SAN DIEGO, Aug. 03, 2023 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced the receipt of a Study May Proceed letter from the U.S. Food and Drug Administration (FDA), for a Phase 1/2 dose escalation study of ONCT-534, a novel dual-action androgen receptor inhibitor (DAARI), in patients with metastatic...Read more
First patient dosed with combination of IDE397, the IDEAYA MAT2A inhibitor, and AMG 193, the Amgen MTA-cooperative PRMT5 inhibitor, in Amgen-sponsored Phase 1/2 clinical trial Global clinical trial will evaluate IDE397 and AMG 193 combination as potential first-in-class synthetic lethality combination in MTAP-deletion patients with planned expansion in NSCLC Earlier reported unconfirmed partial response for...Read more
Independent Data Monitoring Committee recommended REVEAL Phase 1/2 trial continuation and proceeding with dosing of second patient based on encouraging initial clinical data from the first adult with Rett syndrome dosed with investigational gene therapy TSHA-102 Initial clinical update from the first patient dosed with TSHA-102 planned for forthcoming quarterly earnings call Dosing of second patient expected in the third quarter of...Read more
Dose response demonstrated in favor of highest tested dose of 3E10 vg/eye, including 100% reduction in supplemental anti-VEGF injections (4 of 4 evaluable patients injection-free) and a clinically meaningful reduction in mean central subfield thickness (CST) at 36 weeks in patient population with high anti-VEGF need Assessment of outcomes beyond 36 weeks in the 3E10 vg/eye dose cohort showed a durable reduction in supplemental anti-VEGF...Read more
MIAMI, July 27, 2023 (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 that the first patient has been dosed in the Phase 1b/2a clinical trial of AJ201 for the treatment of spinal and bulbar muscular atrophy (“SBMA”), also known as Kennedy's...Read more
Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by rare and niche allergic and immunological diseases. Our lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development...
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