Biomarker cohort for delpacibart braxlosiran (del-brax), the first potential therapy to target DUX4, is measuring changes in DUX4 regulated biomarkers; del-brax 2 mg/kg will be administered every six weeks Enrollment in the del-brax biomarker cohort expected to be completed in 1H 2025; on track to initiate del-brax functional cohort in 1H 2025 In previously reported initial data, del-brax 2 mg/kg every six weeks showed unprecedented...Read more
Dose-dependent increase in neuroprotective APOE2 expression in all participants with ongoing durability at 12 months Consistent reductions across CSF tau biomarkers and tau PET in majority of participants LX1001 well tolerated across all dose cohorts with no reports of amyloid-related imaging abnormalities (ARIA) Company to host webcast today at 7:00 AM ET NEW YORK, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc....Read more
Newly analyzed biomarker data from the initial dose-escalation Phase 1 AD trial found that troculeucel reduces GFAP, NfL, p-tau181, GDF-15, and LTBP2 levels, which are detectable up to 10 years before dementia symptoms appear according to recent reports. This suggests a potential role for the use of troculeucel in delaying or preventing dementia onset in asymptomatic individuals with detectable biomarkers. Preliminary analysis from the...Read more
Encouraging preliminary anti-tumor activity observed in heavily pre-treated population At ≥ 90 mg QD, 6 out of 11 (54.5%) patients with FGFR3+ mUC achieved a confirmed partial response (PR), with 100% disease control rate and sustained duration of activity Positive safety results across all QD doses, with infrequent FGFR2/FGFR1-associated toxicities Conference call scheduled for October 25th, 2024, at 8AM EDT CARLSBAD, Calif.,...Read more
SARASOTA, Fla. and MIAMI, Oct. 24, 2024 (GLOBE NEWSWIRE) -- NAYA Biosciences (“NAYA”) (NASDAQ: NAYA), a life science portfolio company dedicated to bringing breakthrough treatments to patients in oncology, autoimmune diseases, and fertility, today announced an update regarding its clinic trial plans. The Company received regulatory approval from the Israeli Ministry of Health in July 2024 and subsequent institutional review board...Read more
ROCHESTER, N.Y., Oct. 24, 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 promising new efficacy and safety data for its randomized, double-blind, phase 1b/2 SIGNAL-AD study of pepinemab treatment for Alzheimer’s disease at...Read more
Deep attack rate reductions achieved in both dose levels tested; a single 50 mg dose resulted in a mean monthly attack rate reduction of 77% and 81% compared to placebo during weeks 1-16 and 5-16, respectively Eight of 11 patients in the 50 mg arm were completely attack free following a one-time infusion through the latest follow-up; data support NTLA-2002’s potential to be a functional cure for hereditary angioedema (HAE) NTLA-2002...Read more
Continued, progressive improvement in motor function and achievement of motor milestones at 12-months post-treatment represents an important and statistically significant change, in contrast to the disease course observed in BridgeBio’s ongoing CANinform natural history comparator study Significant and sustained reductions in N-acetylaspartate (NAA) levels in urine, cerebrospinal fluid (CSF), and brain in all participants who received...Read more
Enrollment on track in registrational Phase 2 portion of PYNNACLE clinical trial evaluating rezatapopt as monotherapy in patients with TP53 Y220C and KRAS wild-type advanced solid tumors; more than 75% of sites activated across the U.S., Europe, and Asia-Pacific; interim analysis expected by mid-2025 Dose-limiting toxicities observed in rezatapopt and Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) combination arm of Phase 1b...Read more
SAN DIEGO, Oct. 22, 2024 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT) (the “Company”) today announced updated data from its Phase 1/2 Study of ONCT-534 for the treatment of patients with relapsed or refractory metastatic Castration-Resistant Prostate Cancer (mCRPC). Based on initial pharmacokinetic results, two additional dosing cohorts with twice daily (BID) oral dosing of ONCT-534 had been incorporated in the Phase...Read more
NEW YORK, Oct. 22, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease, today announced that interim data from the ongoing Phase 1/2 trial (NCT03634007) of LX1001 have been selected as a late-breaking oral presentation at the Clinical Trials on Alzheimer’s...Read more
American Association for Cancer Research (AACR) Special Conference: Tumor Immunology and Immunotherapy: New immunology data showed ability of INO-3107 to induce an antigen-specific T cell response against HPV-6 and HPV-11 and drive recruitment of T cells into airway tissues and papilloma International Society of Vaccines Conference: Full safety and efficacy data from Phase 1/2 clinical study in patients with Recurrent Respiratory...Read more
Independent Data Safety and Monitoring Board Endorsed Dose Escalation and Broadening of Eligibility Criteria; Cohort 2 Now Enrolling Initial Data from Cohort 1 to be Reported in December 2024 Highlights Recently Presented Insights on Pediatric MYBPC3-associated HCM Disease Burden SOUTH SAN FRANCISCO, Calif., Oct. 17, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with...Read more
LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and...Read more
14 of 15 participants enrolled achieved a clinical response 10 of 12 participants in the 120mg cohort achieved a complete response No serious adverse events; no grade 3 or higher adverse events reported Initial data from BEACON study expected week of January 6th, 2025, including 180mg Q8W cohort Company to host conference call and webinar today at 8:00 a.m. EDT REDWOOD CITY, Calif., Oct. 14, 2024 (GLOBE NEWSWIRE) -- Jasper...Read more
Subject 1 and Subject 2 experienced durable, clinically meaningful improvements in swallowing at 9-months and 6-months post-BB-301 treatment, respectively, with Subject 2 achieving a Sydney Swallow Questionnaire Score Representative of Clinically Normal Swallowing Management plans to host a conference call on October 14 at 8:30 am EDT to discuss the interim results, details below HAYWARD, Calif., Oct. 12, 2024 (GLOBE NEWSWIRE) --...Read more
[212Pb]VMT01 was observed to be safe, and no dose-limiting toxicities were observed at the two doses tested (3.0 and 5.0 mCi) Prolonged progression-free survival and tumor response were observed in heavily pretreated patients who received the low (3.0 mCi) dose of [212Pb]VMT01, consistent with preclinical findings Trial is progressing with testing [212Pb]VMT01 at a lower (1.5mCi) dose to further elucidate the optimal dose for...Read more
ROCKVILLE, Md., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics, Inc. (NYSE American: TOVX), (“Theriva” or the “Company”), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced a positive outcome from the Data and Safety Monitoring Committee (DSMC) review of results from the second Cohort of its Phase 1b/2a randomized, double-blinded,...Read more
Now dosing at dose expansion level of 450 million NXC-201 CAR+T cells. First cohort at 150 million CAR+T cells already successfully completed Each of these two doses have produced complete responses in prior clinical studies Lead site Memorial Sloan Kettering Cancer Center (MSKCC); 4 disclosed clinical sites so far NXC-201 is the only one-time CAR-T treatment option being studied for relapsed/refractory AL Amyloidosis in U.S....Read more
Rhenium (186Re) Obisbemeda delivered by convection enhanced delivery (CED) continues to show safety, response, and potential efficacy Mean Phase 2 absorbed dose was 300 Gy and 89% of patients exceeded the minimal dose threshold of 100 Gy ReSPECT-GBM Phase 1/2 trial has expanded to two new sites at leading U.S. academic medical centers in New York and Upper Midwest AUSTIN, Texas, Oct. 01, 2024 (GLOBE NEWSWIRE) -- Plus Therapeutics,...Read more
LYON, France, October 1, 2024 - EDAP TMS SA (Nasdaq: EDAP), the global leader in robotic energy-based therapies, today announced that the first patients have been treated in a Phase I/II study evaluating the Company’s proprietary Focal One® robotic high-intensity focused ultrasound (HIFU) technology for the treatment of benign prostatic hyperplasia (BPH). “We’ve been offering targeted HIFU ablation to prostate cancer patients for more...Read more
Target enrollment in the chronic cohort close to completion Vancouver, British Columbia--(Newsfile Corp. - September 30, 2024) - NervGen Pharma Corp. (TSXV: NGEN) (OTCQB: NGENF), a clinical-stage biotech company dedicated to developing innovative solutions for the treatment of nervous system damage, today announced that target enrollment of the chronic cohort in its Phase 1b/2a proof-of-concept, double-blind, randomized...Read more
EMERYVILLE, Calif. / Sep 27, 2024 / Business Wire / Estrella Immunopharma, Inc. (NASDAQ: ESLA, ESLAW) (“Estrella” or the “Company”), a clinical stage biopharmaceutical company developing CD19 and CD22-targeted ARTEMIS® T-cell therapies to treat cancer and autoimmune diseases, today announced that the first patient enrolled in its STARLIGHT-1 Phase I/II clinical trial has achieved a complete response (CR) one month after receiving an...Read more
BOCA RATON, Fla., Sept. 26, 2024 (GLOBE NEWSWIRE) -- INmune Bio Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology and inflammation company, continues to advance its Natural Killer (NK) cell therapy, INKmune™, in a Phase I/II trial (the “CaRe PC” trial) for men with metastatic Castration-Resistant Prostate Cancer (mCRPC). The Company is pleased to announce initial results from the first patient cohort in the...Read more
Preliminary finding of stable disease in two out of three patients at 6 months in the first subject cohort Combination of Ampligen and Imfinzi continues to be generally well-tolerated with no severe adverse events or dose-limiting toxicities OCALA, Fla., Sept. 19, 2024 (GLOBE NEWSWIRE) -- AIM ImmunoTech Inc. (NYSE American: AIM) (“AIM”) today announced positive preliminary data from the Phase 1b/2 study (“DURIPANC”) evaluating...Read more
4D-150 demonstrated robust and durable clinical activity across all wet age-related macular degeneration (wet AMD) patient populations based on longest available follow-up data In broad population (Phase 2b), 70% injection-free through 52 weeks In severe population (Phase 1/2a), 83% overall reduction in annualized injections through 52 weeks 4D-150 continues to be safe and well tolerated with intraocular inflammation (IOI)...Read more
GAITHERSBURG, Md., Sept. 18, 2024 (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, announces the Company will present further data from BiomX’s Phase 1b/2a study of BX004 for the treatment of cystic fibrosis patients with chronic Pseudomonas aeruginosa pulmonary...Read more
Confirmed partial responses (PRs) doubled to four since ASCO GI in MSS CRC patients without liver and peritoneal metastases for overall response rate (ORR) of 24% (4/17) with ADG126 10 mg/kg every three weeks (Q3W) in combination with pembrolizumab Median progression-free survival of 8.5 months observed in patients without liver and peritoneal metastases at the ADG126 10 mg/kg Q3W dose At the 10 mg/kg dose level, 12-month...Read more
Treatment with rinatabart sesutecan (Rina-S) showed encouraging response rate in heavily pretreated patients with ovarian cancer in dose expansion cohort Responses with Rina-S were observed across FRα expression levels Phase 3 trial will further evaluate the safety and efficacy of Rina-S at 120 mg/m2 in patients with advanced ovarian cancer COPENHAGEN, Denmark / Sep 15, 2024 / Business Wire / Genmab A/S (Nasdaq: GMAB) announced today...Read more
Median duration of response reaches 7.4 months with combination treatment in patients with aggressive form of disease New results show potential of RYBREVANT® beyond lung cancer BARCELONA, Sept. 14, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) today announced new data from the Phase 1b/2 OrigAMI-1 study, which showed RYBREVANT® (amivantamab-vmjw) combined with chemotherapy (mFOLFOX6 [FOLFOX] or FOLFIRI)...Read more
Updated monotherapy data for Nectin-4 targeting zelenectide pevedotin in metastatic urothelial cancer (mUC) showed a promising 45% overall response rate (ORR), 11.1 months median duration of response and a generally well-tolerated safety profile EphA2-targeting BT5528 demonstrated an emerging differentiated safety profile and antitumor activity in patients with advanced solid tumors, including a 45% ORR in mUC 6.5 mg/m2 every two weeks...Read more
Combination of masofaniten plus enzalutamide continues to be well tolerated with durable reductions in PSA in patients with mCRPC Phase 2 dose expansion currently underway at the RP2CDs of masofaniten 600 mg BID in combination with enzalutamide 160 mg QD Across all dosing cohorts, 88% of patients achieved PSA90, 69% of patients achieved PSA90 in less than 90 days, and 63% of patients achieved PSA <0.2ng/mL. After 15.2 months of...Read more
Anti-tumor clinical responses were observed in 29% (4/14) of efficacy-evaluable patients with PROC treated with TransCon IL-2 β/γ in combination with chemotherapy Initial data suggest clinical activity in heavily pre-treated PROC patients and that TransCon IL-2 β/γ in combination with chemotherapy was generally well-tolerated COPENHAGEN, Denmark, Sept. 13, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today...Read more
Two of the first three patients in the Phase 1 cohort of the Phase 1/2a clinical trial, treated at the highest dose of 6 billion cells per treatment, were found to have improved CDR-SB cognitive scores, resulting in a clinical upgrade from moderate AD to mild AD after only three months on therapy. First patient dosed in the Phase 2 cohort of the trial, continuing at the highest dose of 6 billion cells per treatment. With the...Read more
Abundant, richly vascularized and functioning islets observed throughout all chambers of Sernova’s Cell Pouch more than 5 years after islet transplantation Histological data confirmation of healthy beta, alpha and delta cells secreting insulin, glucagon, and somatostatin in all Cell Pouch Chambers Sernova’s Cell Pouch safely contains its therapeutic cells and provides full retrievability using conventional instruments and...Read more
Consistent improvement in patient reported outcomes with 10 of 11 evaluable patients in the first four cohorts experiencing a reduction in symptom (SDI1) scores at 12 weeks The fourth cohort showed the greatest percentage change in histology (EoEHSS2) scores of any cohort to date The RESOLVE Phase 1b/2a trial is progressing as anticipated with no serious adverse events reported in all four fully-dosed cohorts VICTORIA, BC,...Read more
Increase in endogenous cortisol production achieved in all patients in higher dose cohorts of BBP-631, a result seen for the first time ever in CAH patients The gene therapy was well tolerated with no treatment-related serious adverse events (SAEs) reported Despite novel scientific advancements achieved with this program, the data do not warrant additional capital investment at this time and the gene therapy budget is being...Read more
Topline results expected in 2Q 2025 CAMBRIDGE, Mass. / Sep 10, 2024 / Business Wire / Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today announced it has completed enrollment in the Phase 2 EMBRAZE trial designed to show...Read more
Rapid-fire Oral Presentation to Highlight Efficacy, Safety, and Quality of Life Outcomes LOS ANGELES, Sept. 10, 2024 (GLOBE NEWSWIRE) -- ACELYRIN, INC. (Nasdaq: SLRN), a late-stage clinical biopharma company focused on accelerating the development and delivery of transformative medicines in immunology, today announced that positive data from its Phase 1/2 clinical trial of lonigutamab (an anti-insulin-like growth factor 1 receptor,...Read more
New data showed improvements in cognition and behavior during the first year of treatment with additional increases demonstrated as treatment continued Clinical effects observed across the Phase 1/2a and open-label extension studies (OLEs) of zorevunersen are a first in the treatment of Dravet syndrome and support plans for the Company’s Phase 3 registrational study Zorevunersen generally well-tolerated across the...Read more
Elraglusib Demonstrated Anti-Tumor Activity with Two Ongoing Durable Complete Responses and ~62% Disease Control Rate in First 8 Patients with Relapsed/Refractory Ewing and Ewing-related Sarcomas Enrollment Ongoing with Topline Data Anticipated in 1H 2025 CHICAGO and FORT WORTH, Texas, Sept. 09, 2024 (GLOBE NEWSWIRE) – Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage...Read more
Updated Phase 1 data from ALKOVE-1 and ARROS-1 clinical trials to be presented at the ESMO Congress 2024 Durable activity of NVL-655 and zidesamtinib in heavily pre-treated patient populations supports ongoing Phase 2 investigation in earlier lines of treatment Company plans to host a conference call on September 14, 2024 at 8:30 a.m. ET/2:30 p.m. CEST following oral presentations at ESMO CAMBRIDGE, Mass., Sept. 9, 2024...Read more
Bexicaserin achieved an overall median seizure reduction of 57.7% in countable motor seizures over an approximate 9-month treatment period Favorable safety and tolerability results continue to be observed Data to be presented at the 15th European Epilepsy Congress in Rome, Italy Full 12-month OLE dataset expected early next year LA JOLLA, Calif. / Sep 08, 2024 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a...Read more
Trem-cel + Mylotarg demonstrated engraftment, shielding, broadened therapeutic window, and patient benefit VCAR33ALLO demonstrates encouraging biomarker data at lowest dose New asset VADC45 with significant potential opportunities across oncology, gene therapy, and autoimmune disorders CAMBRIDGE, Mass., Sept. 05, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today announced...Read more
First trial in Rigel's multi-year strategic development alliance with MD Anderson Phase 1b/2 trial of decitabine and venetoclax in combination with Rigel's targeted mIDH1 inhibitor REZLIDHIA for patients with mIDH1 AML SOUTH SAN FRANCISCO, Calif., Sept. 5, 2024 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today announced the first...Read more
SOUTH SAN FRANCISCO, Calif., Sept. 04, 2024 (GLOBE NEWSWIRE) -- ALX Oncology Holdings Inc., (“ALX Oncology” or “the Company”) (Nasdaq: ALXO), an immuno-oncology company developing therapies that block the CD47 immune checkpoint pathway, announced the first patients have been dosed in an arm of the randomized UMBRELLA phase 1/2 clinical study partnered with Sanofi that is evaluating evorpacept in combination with SARCLISA®...Read more
Initiating Registrational Cohorts with Update on Path to Registration by Year-End 2024 Virtual Investor Event Today at 8:00 a.m. ET WALTHAM, Mass., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced new clinical data from its ongoing...Read more
New Sites Expand Opportunities for Patient Access and Enrollment Clinical Trial Sites Added Include Cleveland Clinic, UC Davis, and Sutter Health Lead site Memorial Sloan Kettering Cancer Center (MSKCC) Data from ex-U.S. clinical trial reported at ASGCT 2024 showed a 92% overall response rate LOS ANGELES, CA, Aug. 28, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”, “IMMX”) (Nasdaq: IMMX), a...Read more
MALVERN, Pa., Aug. 28, 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 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. Stargardt...Read more
Effective immediately, clinical sites in the VISTA-101 Phase 1/ Phase 2 clinical trial can resume screening of patients for enrollment in the clinical study 30 patients have been enrolled in the clinical trial to date, including a monotherapy arm with KVA12123 and a combination arm utilizing KVA12123 together with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) Kineta anticipates the trial to be fully enrolled by the end of 2024...Read more
LEXINGTON, Mass. and AMSTERDAM, Aug. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease, a rare, inherited genetic disease. The Phase I/IIa study is a multi-center, open-label trial being conducted...Read more
Demonstrates Prolonged Progression Free Survival of Two Patients in Acclaim-1 and Partial Remission from Maintenance Therapy in Acclaim-3 Re-focuses Oncology Clinical Development Program and Advances Work to Establish Biomarkers to Enrich Clinical Trial Patient Populations AUSTIN, Texas, Aug. 14, 2024 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on...Read more
ROCHESTER, N.Y., Aug. 14, 2024 (GLOBE NEWSWIRE) -- Vaccinex (Nasdaq: VCNX) today provided an update on new clinical findings from its SIGNAL-AD Phase 1b/2 trial of pepinemab antibody in Alzheimer’s disease. Our Company recently announced positive results of the phase 1b/2 study of its lead product, pepinemab, in early stages of Alzheimer’s disease (AD). The purpose of this report is to share additional data related to cognitive effects...Read more
Aptevo's lead candidate APVO436 (mipletamig) to be evaluated in combination with standard of care venetoclax and azacitidine Company anticipates new data set to bolster existing compelling data reported to date, including efficacy outcomes more than double the benchmarks* Outcomes also expected to identify recommended Phase 2 dose APVO436 renamed as mipletamig: Aptevo adopts new generic name for future use SEATTLE, WA / ACCESSWIRE...Read more
Delpacibart zotadirsen (AOC 1044) delivered unsurpassed muscle concentrations of PMO of 200 nM after three doses of 5 mg/kg Statistically significant 37% increase in exon 44 skipping and up to 66% skipping with delpacibart zotadirsen 5 mg/kg at four months Statistically significant increase of 25% of normal in dystrophin production and restored total dystrophin up to 54% of normal with delpacibart zotadirsen 5 mg/kg at four...Read more
The QUILT 502 trial is testing ImmunityBio’s N-803 (ANKTIVA®) in combination with the AdHER2DC investigational vaccine for endometrial cancer, a gynecological cancer with lower survival rates and limited effective post-second-line treatment. The AdHER2DC vaccine targets the HER2 protein, which is elevated in 30% of endometrial cancer. ANKTIVA, recently approved for BCG-unresponsive non-muscle invasive bladder cancer carcinoma in situ,...Read more
Robust microdystrophin expression observed in new data from pivotal dose Patients aged 5.8 and 8.5 years at dosing had expression levels at 77.2% and 46.5% of control, respectively Consistent high expression of microdystrophin across treated patients in all age groups continues to support plans for accelerated approval Expects to initiate pivotal trial in Q4 2024 ROCKVILLE, Md., Aug. 1, 2024 /PRNewswire/ -- REGENXBIO...Read more
Results affirm expectation of a novel mechanism of action targeting astrocyte activation to delay progression of Alzheimer’s disease from early stage Mild Cognitive Impairment Findings echo previous phase 2 results in Huntington’s disease based on similarities to neuroimmune pathology in Alzheimer’s ROCHESTER, N.Y., July 31, 2024 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering...Read more
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
Terns Pharmaceuticals is a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity. Terns’ pipeline contains three clinical stage development programs including GLP-1 receptor...
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