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Latest Phase 1/2 Clinical Trial News

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Exelixis Announces Encouraging Results from Phase 1b/2 STELLAR-002 Trial Evaluating Zanzalintinib in Combination with Immune Checkpoint Inhibitors in Advanced Kidney Cancer at ASCO 2025

May 22
Last Trade: 42.39 -0.47 -1.10

Zanzalintinib in combination with nivolumab demonstrated an objective response rate of 63% and a disease control rate of 90%  Additional results from dose-finding cohorts will also be presented  ALAMEDA, Calif. / May 22, 2025 / Business Wire / Exelixis, Inc. (Nasdaq: EXEL) today announced results from an expansion cohort of the phase 1b/2 STELLAR-002 trial evaluating zanzalintinib in combination with either nivolumab...Read more


Verastem Oncology Announces Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in Frontline Metastatic Pancreatic Ductal Adenocarcinoma

May 22
Last Trade: 8.31 0.83 11.10

Selected recommended Phase 2 dose: Dose level 1 demonstrated an ORR of 83% (10/12) in frontline metastatic pancreatic ductal adenocarcinoma Plans for registrational Phase 3 study underway in frontline metastatic pancreatic ductal adenocarcinoma Company will host an R&D investor webcast on Monday, June 2 at 11:00 am CDT to review the updated RAMP 205 data as well as updated data on VS-7375 presented at ASCO BOSTON / May 22, 2025 /...Read more


Immix Biopharma Announces Positive Results for NXC-201 at ASCO Oral Presentation, Enabling Pathway to Best-in-Class Therapy for relapsed/refractory AL Amyloidosis

May 22
Last Trade: 1.99 -0.42 -17.43

Immix Biopharma today reported ASCO abstract results from its U.S. multi-center NEXICART-2 Phase 1/2 clinical trial of NXC-201 demonstrating strong efficacy and favorable safety. NXC-201 demonstrated a complete response (CR) rate of 71% (5/7 patients)  No relapses recorded to-date; no safety signals identified  Updated results with a later data cutoff, including endpoint analysis, will be presented at ASCO by lead...Read more


Tango Therapeutics Announces First Patient Dosed in TNG456 Phase 1/2 Trial in Patients With MTAP-deleted Glioblastomas and Other Solid Tumors

May 21
Last Trade: 1.84 -0.03 -1.34

BOSTON, May 21, 2025 (GLOBE NEWSWIRE) -- Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to discovering the next generation of precision cancer medicines, today announced that the first patient has been dosed in the TNG456 Phase 1/2 trial in patients with MTAP-deleted solid tumors, with a focus on glioblastoma (GBM). TNG456 is a next-generation, brain-penetrant, MTA-cooperative PRMT5...Read more


Prime Medicine Announces Breakthrough Clinical Data Showing Rapid Restoration of DHR Positivity After Single Infusion of PM359, an Investigational Prime Editor for Chronic Granulomatous Disease

May 19
Last Trade: 1.29 0.06 4.88

First ever clinical data supporting safety and efficacy of Prime Editing in humans  Initial data from first patient dosed in Phase 1/2 trial finds single dose of PM359 led to 58% DHR positivity by Day 15 and 66% by Day 30, well above levels believed to be potentially curative  Rapid engraftment observed in both neutrophils and platelets  Encouraging safety profile; no serious adverse events related to...Read more


Armata Pharmaceuticals Announces Positive Topline Data from the Phase 1b/2a diSArm Study of Intravenously Administered AP-SA02 in Complicated Staphylococcus aureus Bacteremia

May 19
Last Trade: 1.89 0.02 1.07

All primary endpoints for safety, tolerability, and clinical response in the intent-to-treat population met AP-SA02 arm significantly improved clinical outcomes and prevented relapse compared to best available antibiotic therapy No treatment-related serious adverse events were observed with repetitive intravenous dosing LOS ANGELES, May 19, 2025 /PRNewswire/ -- Armata Pharmaceuticals, Inc. (NYSE American: ARMP) ("Armata" or the...Read more


Artiva Biotherapeutics Announces Longer-term Phase 1/2 Data Demonstrating Prolonged Durability for AlloNK® in Combination with Rituximab in Patients with B-cell-Non-Hodgkin Lymphoma at the ASGCT 28th Annual Meeting

May 13
Last Trade: 1.95 -0.03 -1.52

64% (9/14) complete response rate with AlloNK + rituximab in heavily pretreated patients that were naïve to prior CAR-T cell therapy, in line with approved auto-CAR-T therapies in aggressive B-NHL Median duration of response for AlloNK + rituximab not yet reached and is at least 19.4 months, in line with approved auto-CAR-T therapies in aggressive B-NHL AlloNK’s activity and well-tolerated safety profile in aggressive B-NHL patients...Read more


Aptose Biosciences Provides Clinical Update for the Tuspetinib-based Triple Drug Frontline Therapy in Newly Diagnosed AML Patients from the Phase 1/2 TUSCANY Trial

May 5
Last Trade: 1.71 0.00 0.00

Aptose is developing TUS+VEN+AZA as a one-of-a-kind safe and mutation agnostic frontline triple drug therapy for newly diagnosed AML patients First two dose cohorts of TUS+VEN+AZA triplet demonstrate safety, complete remissions, and MRD negativity across patients with diverse mutations, including TP53-mutated/CK AML and FLT3-wildtype AML patients SAN DIEGO and TORONTO, May 05, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc....Read more


Eupraxia Pharmaceuticals Announces Sustained Positive Treatment Outcomes in Patients with Eosinophilic Esophagitis (EoE) After Nine Months of Receiving EP-104GI

May 5
Last Trade: 5.26 0.10 1.94

Eupraxia continues to observe positive treatment outcomes in its ongoing Phase 1b/2a trial, including for the first time clinical responses measured 9 months after dosing with EP-104GI At 9 months active drug continued to be released into the esophagus in patients at a rate similar to what was seen at the 3- and 6-month time points. This is an unprecedented result with an injectable delivery system in patients with EoE Also, at 9...Read more


Recursion: Preliminary Phase 1b/2 Data for REC-4881 in Familial Adenomatous Polyposis (FAP) Demonstrates Reduced Polyp Burden

May 4
Last Trade: 4.08 -0.06 -1.45

In the Phase 2 open-label study, REC-4881 (4 mg QD) led to a median 43% reduction (n=6 patients) in polyp burden at the Week 13 assessment at time of data cutoff. Five of six patients (83%) experienced reductions in polyp burden ranging from 31% to 82%, however, one patient showed a substantial increase from baseline. At Week 13, 50% of patients (3 out of 6) achieved ≥1-point improvement in Spigelman stage, a measure of upper GI...Read more


Perspective Therapeutics Announces First Patient Dosed with PSV359 in a Phase 1/2a Study in Patients with FAP-α Positive Solid Tumors

April 29
Last Trade: 2.42 -0.09 -3.59

SEATTLE, April 29, 2025 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. ("Perspective" or the "Company") (NYSE AMERICAN: CATX), a radiopharmaceutical company pioneering advanced treatments for cancers throughout the body, announced today that the first patient was treated with [212Pb]PSV359 in a Phase 1/2a dose-finding trial to determine safety and preliminary anti-tumor activity of the radiopharmaceutical [212Pb]PSV359 in patients...Read more


ALX Oncology Announces Encouraging Final Results from Phase 1 Trial Evaluating Evorpacept in Combination with Standard-of-Care Treatment in Patients with B-cell Non-Hodgkin Lymphoma

April 25
Last Trade: 0.46 -0.01 -3.11

Data to be presented at AACR 2025 Annual Meeting suggest the combination of ALX Oncology’s investigational CD47-blocker, evorpacept, plus rituximab and lenalidomide (R2) was well-tolerated and demonstrated promising anti-tumor activity Combination generated complete responses (CR) in 83% of patients with indolent relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) comparing favorably to 34% historical CR rate with R2 alone...Read more


BriaCell Therapeutics Confirms 100% Resolution of Lung Metastasis with Bria-OTS™

April 24
Last Trade: 3.04 -0.37 -10.82

(IMAGES BELOW) Complete resolution of lung metastasis confirmed at 4 month follow-up in hormone receptor positive (HR+) breast cancer patient Treatment well-tolerated and patient remains on study with stable disease elsewhere Sustained clinical response supports Bria-OTS personalized, off-the-shelf immunotherapy approach in Phase 1/2a metastatic breast cancer study PHILADELPHIA and VANCOUVER, British Columbia, April 24, 2025 (GLOBE...Read more


Clearmind Medicine Completes the Initiation of its Alcohol Use Disorder Phase I/IIa Clinical Trial at all Current Clinical Sites

April 23
Last Trade: 0.97 -0.04 -3.50

The Company is now preparing for the enrollment of the first patient to receive Clearmind’s innovative treatment as part of the trial Vancouver, Canada, April 23, 2025 (GLOBE NEWSWIRE) -- Clearmind Medicine Inc. (Nasdaq: CMND), (FSE: CWY0) (“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, today...Read more


Enlivex Therapeutics Announces Completion of Enrollment In The Phase II stage Of Its Phase I/II Trial Evaluating Allocetra In Patients With Moderate To Severe Knee Osteoarthritis

April 21
Last Trade: 0.98 0.01 1.34

Nes-Ziona, Israel, April 21, 2025 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced that the Company completed enrollment of all patients in the Phase II stage of its randomized, controlled, blinded Phase I/II trial of Allocetra™ in patients with moderate to severe knee osteoarthritis. Overall, 133 patients were randomized and...Read more


Clearmind Medicine Launches First U.S. Clinical Site for its FDA-Approved Clinical Trial to Combat Alcoholism

April 16
Last Trade: 0.97 -0.04 -3.50

Vancouver, Canada, April 16, 2025 (GLOBE NEWSWIRE) -- Clearmind Medicine Inc. (Nasdaq: CMND), (FSE: CWY0) (“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, recently announced it has initiated its Phase I/IIa clinical trial at its first U.S. clinical site, the Johns Hopkins University School of...Read more


BioCardia Announces Positive DSMB Review of CardiALLO Allogeneic Cell Therapy for Heart Failure Phase 1/2 Clinical Trial

April 15
Last Trade: 1.96 0.06 3.16

Independent Data Safety Monitoring Board (DSMB) recommends that the study proceeds as designed based on 30-day data safety assessment of participants treated in low dosage group. The company plans to progress the trial to enrollment of 39 participants in the United States. SUNNYVALE, Calif., April 15, 2025 (GLOBE NEWSWIRE) -- BioCardia, Inc. [Nasdaq: BCDA], a global leader in cellular and cell-derived therapeutics for the...Read more


Perspective Therapeutics Announces First Patient Dosed with [212Pb]VMT01 Monotherapy at 1.5 mCi in a Phase 1/2a Study of MC1R-Positive Metastatic Melanoma

April 11
Last Trade: 2.42 -0.09 -3.59

SEATTLE, April 11, 2025 (GLOBE NEWSWIRE) -- Perspective, Therapeutics, Inc. ("Perspective" or the "Company") (NYSE AMERICAN: CATX), a radiopharmaceutical company pioneering advanced treatments for cancers throughout the body, announced today that the first patient was dosed in a new cohort of a Phase 1/2a trial evaluating the safety of [212Pb]VMT01, a targeted alpha-particle therapy (TAT), as monotherapy in patients with histologically...Read more


Clearmind Medicine Announces First U.S. Clinical Site Initiation for CMND-100 Clinical Trial in Patients with Alcohol Use Disorder

April 10
Last Trade: 0.97 -0.04 -3.50

Vancouver, Canada, April 10, 2025 (GLOBE NEWSWIRE) -- Clearmind Medicine Inc. (Nasdaq: CMND), (FSE: CWY0) (“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 today that it has initiated its Phase I/IIa clinical trial at its first U.S. clinical site, the Johns Hopkins University School of...Read more


IDEAYA Biosciences Announces Phase 1/2 Expansion for IDE397 and Trodelvy® Combination in MTAP-Deletion Urothelial Cancer

April 10
Last Trade: 18.82 0.14 0.75

Initiated Phase 1/2 study expansion for IDE397, IDEAYA's MAT2A inhibitor, in combination with Trodelvy®, Gilead's Trop-2 directed ADC, in MTAP-deletion urothelial cancer based on preliminary safety and clinical efficacy data MTAP-deletion prevalence in urothelial cancer is estimated to be approximately 26% SOUTH SAN FRANCISCO, Calif., April 10, 2025 /PRNewswire/ -- IDEAYA Biosciences, Inc. (Nasdaq:IDYA), a precision medicine...Read more


Lexeo Therapeutics Announces Positive Interim Phase 1/2 Data for LX2006 in Friedreich Ataxia Cardiomyopathy Supporting Advancement to Registrational Study

April 7
Last Trade: 2.82 -0.26 -8.44

Participants with abnormal left ventricular mass index (LVMI) at baseline achieved 25% mean reduction in LVMI by 12 months or sooner Clinically meaningful improvements in majority of participants across cardiac biomarkers and functional measures All SUNRISE-FA participants achieved meaningful increases in frataxin expression at 3-months post treatment; 115% average cardiac frataxin expression increase in high dose cohort,...Read more


Beam Therapeutics Presents Additional Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress

April 5
Last Trade: 16.83 -0.41 -2.38

New Data Demonstrate Proportion of Corrected M-AAT Reached a Mean of 91% of Total AAT in Circulation at Day 28 Following BEAM-302 Treatment in 60 mg Cohort (n=3) Mean Decrease of 79% in Mutant Z-AAT Observed at Day 28 in 60 mg Cohort (n=3) Fourth Cohort Evaluating 75 mg of BEAM-302 Initiated, with Updated Data from Part A of the Phase 1/2 Trial Expected to be Presented at a Medical Conference in Second Half of 2025 CAMBRIDGE,...Read more


Sonnet’s SON-1010 Demonstrates a Strong Safety Profile in Combination with Atezolizumab for Treatment of Platinum-Resistant Ovarian Cancer, Including a Partial Response at the Highest Dose

April 4
Last Trade: 1.12 0.00 0.00

Topline safety data in SB221 study suggest clinical benefit of SON-1010 in combination with atezolizumab (Tecentriq®) The maximum tolerated dose (MTD) of SON-1010 was set at 1200 ng/kg in combination with atezolizumab in patients with platinum-resistant ovarian cancer (PROC), without dose-limiting toxicity or evidence of cytokine release syndrome at any dose level Stable disease (SD) at four months post-initiation of dosing was seen...Read more


Sernova Biotherapeutics Provides Update on Phase 1/2 Clinical Trial of Cell Pouch™ Bio-Hybrid Organ for Treatment of Type 1 Diabetes

April 1
Last Trade: 0.20 -0.02 -7.14

DSMB supports advancement for Sernova’s clinical trial Study is on track to meet key clinical trial endpoints LONDON, Ontario and BOSTON, April 01, 2025 (GLOBE NEWSWIRE) -- Sernova Biotherapeutics, (TSX: SVA) (OTCQB: SEOVF) (FSE/XETRA: PSH), a leading regenerative medicine company focused on developing it’s Cell Pouch Bio-hybrid Organ as a functional cure for type 1 diabetes (T1D), today provided an update on its ongoing clinical...Read more


Avidity Biosciences Completes Enrollment in Biomarker Cohort in Phase 1/2 FORTITUDE™ Trial for Delpacibart Braxlosiran (del-brax) in People Living with Facioscapulohumeral Muscular Dystrophy

March 31
Last Trade: 30.58 -0.23 -0.75

Del-brax FORTITUDE biomarker cohort designed for potential accelerated approval; plan to share regulatory update in Q2 2025 Regulatory alignment on global Phase 3 del-brax trial design and study initiation anticipated in Q2 2025 Plan to present topline data from FORTITUDE dose escalation cohorts in Q2 2025 On track to be first globally approved drug for FSHD SAN DIEGO, March 31, 2025 /PRNewswire/ -- Avidity Biosciences, Inc....Read more


Sensei Biotherapeutics Reports Favorable Preliminary Dose Expansion Data for Solnerstotug in PD-(L)1 Resistant Tumors

March 27
Last Trade: 0.30 -0.0035 -1.17

Initial clinical activity in a PD-(L)1 resistant population, with an ORR almost three times higher than historical PD-(L)1 rechallenge response rates, with data still maturing One durable complete response in a Merkel Cell Carcinoma (MCC) patient and two partial responses (PR), including one in a second MCC patient and one in a microsatellite instability-high (MSI-H) Colorectal Cancer (CRC) patient, all of whom were previously treated...Read more


Genelux and Newsoara Announce Positive Preliminary Phase 1b/2 Data of Olvi-Vec in Advanced Small-Cell Lung Cancer

March 25
Last Trade: 2.58 0.09 3.61

Systemic administration of Olvi-Vec in the initial dose escalation cohorts achieved a 71% disease control rate (5/7) with two partial responders. All participants with disease control experienced a reduction in all target lesions, with one participant achieving a tumor reduction of approximately 79%. Additionally, three participants, including one individual with three prior lines of treatment, achieved stable disease at lower dose...Read more


Aptevo Therapeutics RAINIER Trial Data Update: Two Additional AML Patients Achieve Remission Within 30 Days of Treatment

March 20
Last Trade: 0.27 -0.03 -8.67

Across two trials, 9 of 10 frontline AML patients achieved remission when treated with mipletamig in combination with the standard of care Triplet Combination with mipletamig continues to outperform doublet combination benchmark No Cytokine Release Syndrome (CRS) has been observed in the RAINIER trial to date Cohort 2 enrollment nears completion SEATTLE, WA / ACCESS Newswire / March 20, 2025 / Aptevo Therapeutics ("Aptevo")...Read more


REGENXBIO Reports Positive Biomarker Data From Affinity Duchenne® Trial Of RGX-202 Gene Therapy

March 19
Last Trade: 9.71 -0.15 -1.52

Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages Patient aged 3 years at dosing had expression level at 122.3% compared to control With a differentiated novel construct and proactive short course immune modulation regimen, RGX-202 continues to demonstrate encouraging safety profile with no SAEs or AESIs Phase III portion of AFFINITY DUCHENNE®...Read more


Benitec Biopharma Reports Positive Interim Clinical Results for Three Subjects Treated with BB-301 in Phase 1b/2a Study to be Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

March 19
Last Trade: 13.19 -0.40 -2.94

Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 1 Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 2, with Subject 2 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden Clinically Significant Improvements in...Read more


Indaptus Therapeutics Initiates Phase 1 Combination Study of Decoy20 with PD-1 Checkpoint Inhibitor Tislelizumab

March 18
Last Trade: 0.41 0.03 7.54

NEW YORK, March 18, 2025 (GLOBE NEWSWIRE) -- Indaptus Therapeutics, Inc. (Nasdaq: INDP), a clinical-stage biotechnology company dedicated to developing novel treatments for cancer and viral infections, today announced that it has advanced to a new expansion arm of its Phase 1b/2 clinical trial of Decoy20. This expansion will evaluate the combination of Decoy20 with BeiGene’s PD-1 checkpoint inhibitor, tislelizumab, with a focus on...Read more


Clearmind Medicine Announces Initiation of First in Human Clinical Trial with CMND-100 in Alcohol Use Disorder Patients

March 18
Last Trade: 0.97 -0.04 -3.50

Study represents the first clinical application of CMND-100, the Company’s proprietary drug platform Vancouver, Canada, March 18, 2025 (GLOBE NEWSWIRE) -- Clearmind Medicine Inc. (Nasdaq: CMND), (FSE: CWY0) (“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, announces today that it initiated its...Read more


Genmab: Investigational Rinatabart Sesutecan (Rina-S®) Continues to Show Encouraging Antitumor Activity in Patients with Advanced Ovarian Cancer

March 17
Last Trade: 20.81 -0.22 -1.05

Results from Phase 2 RAINFOLTM-01 trial (B1 cohort) showed that with a median on-study follow-up of 48 weeks, Rina-S 120 mg/m2 led to a confirmed objective response rate (ORR) of 55.6% and median duration of response (mDOR) was not reached Phase 2 RAINFOLTM-01 and Phase 3 RAINFOLTM-02 trials evaluating the safety and efficacy of Rina-S at 120 mg/m2 in patients with platinum resistant ovarian cancer (PROC) are actively...Read more


Avidity Biosciences Announces Positive Topline Del-zota Data Demonstrating Consistent, Statistically Significant Improvements in Dystrophin, Exon Skipping and Creatine Kinase in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44...

March 17
Last Trade: 30.58 -0.23 -0.75

On track for year end 2025 BLA submission for accelerated approval of 5 mg/kg every six weeks of del-zota in DMD44 Consistent favorable safety and tolerability across del-zota dose cohorts  Plan to present functional data in fourth quarter of 2025 Investor and analyst webcast event today at 8:00 a.m. ET SAN DIEGO, March 17, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company...Read more


Perspective Therapeutics Announces First Patient Dosed with [212Pb]VMT01 in Combination with Nivolumab in a Phase 1/2a Study of MC1R-Positive Metastatic Melanoma

March 17
Last Trade: 2.42 -0.09 -3.59

SEATTLE, March 17, 2025 (GLOBE NEWSWIRE) -- Perspective, Therapeutics, Inc. ("Perspective" or the "Company") (NYSE AMERICAN: CATX), a radiopharmaceutical company that is pioneering advanced treatment applications for cancers throughout the body, announced today that the first patient was dosed in a new cohort of a Phase 1/2a trial evaluating the safety of [212Pb]VMT01, a targeted alpha-particle therapy (TAT), in patients with...Read more


Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months

March 16
Last Trade: 11.69 -0.21 -1.76

Continued favorable safety profile for DYNE-251  DELIVER Registrational Expansion Cohort is fully enrolled; data from this cohort planned for late 2025  Potential for Biologics License Application submission for U.S. accelerated approval in early 2026  WALTHAM, Mass., March 16, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming...Read more


TransCode Therapeutics Announces Safety Review Committee Approval to Open Fourth Cohort in Phase I/II Clinical Trial

March 13
Last Trade: 6.97 -0.65 -8.53

Clinical trial Safety Review Committee (SRC) approved opening of Cohort 4 based on favorable safety data from the three patients comprising Cohort 3 No significant safety or dose limiting toxicities reported in Cohorts 1, 2, or 3 Patients currently being evaluated for Cohort 4 eligibility PK and PD data from patients in Cohort 1 and Cohort 2 consistent with preclinical results and results from Phase 0 clinical trial BOSTON, March...Read more


Arrowhead Pharmaceuticals Announces Topline Results from Part 2 of Phase 1/2 Study of ARO-C3 in Patients with IgA Nephropathy

March 10
Last Trade: 15.13 -0.26 -1.69

ARO-C3 achieved deep and sustained reductions in alternative pathway complement activity and proteinuria Mean sustained reductions in C3 of ≥87%, AH50 of ≥76%, Wieslab AP of ≥89% through week 24 Mean reduction in spot urine protein-to-creatinine ratio (UPCR) of 41% by week 24 PASADENA, Calif. / Mar 10, 2025 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced topline results from Part 2 of a Phase 1/2...Read more


Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation

March 10
Last Trade: 16.83 -0.41 -2.38

Single Dose of BEAM-302 Led to Durable, Dose-dependent Increases in Total and Functional Alpha-1 Antitrypsin (AAT), Production of Corrected M-AAT, and Decreases in Mutant Z-AAT in Circulation Across Initial Three Dose Levels Third Dose Level of BEAM-302 (60 mg, N=3) Achieved Mean Total AAT of 12.4µM at Day 28, Exceeding Protective Therapeutic Threshold, and Reduced Mutant Z-AAT up to 78% Initial Safety Findings Demonstrated BEAM-302...Read more


Eupraxia Pharmaceuticals Announces Positive Data from RESOLVE Phase 1b/2a Trial of EP-104GI for Treatment of Eosinophilic Esophagitis

February 25
Last Trade: 5.26 0.10 1.94

Histological scores and symptom scores continue to improve as EP-104GI dose, and area of esophageal coverage, increase Cohort 6 showed the greatest symptom relief scores ("SDI") of all cohorts to date at 12 weeks Cohort 6 had the greatest magnitude and percentage change in tissue health scores ("EoEHSS") of any cohort to date at 12 weeks Cohort 6 saw the greatest reduction in Peak Eosinophil Count ("PEC") of any cohort to...Read more


Regeneron Pharmaceuticals: Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial

February 24
Last Trade: 588.34 -8.51 -1.43

As presented at ARO, 10 of 11 children with at least one post-treatment assessment showed notable improvements in hearing Speech and development progress followed dramatic improvements in hearing in first child treated in the trial TARRYTOWN, N.Y., Feb. 24, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial...Read more


Calidi Biotherapeutics Announces Commencement of Recruitment for Multiple Dose CLD-101 Trial in Patients with Newly Diagnosed High Grade Glioma at Northwestern University Hospital

February 24
Last Trade: 0.41 -0.007 -1.69

SAN DIEGO, Feb. 24, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics Inc. (NYSE American: CLDI) (“Calidi”), a clinical-stage biotechnology company developing a new generation of targeted antitumor virotherapies, today announced that Northwestern University/Northwestern Memorial Hospital is starting recruitment for the CLD-101 clinical trial of its immunotherapy product. Calidi Biotherapeutics has completed the shipment of the first batch...Read more


Estrella Immunopharma Completes First Dose Cohort in STARLIGHT-1 Trial and Receives Approval to Initiate Higher Dose Cohort

February 21
Last Trade: 1.04 0.04 4.00

EMERYVILLE, Calif. / Feb 21, 2025 / Business Wire / Estrella Immunopharma, Inc. (NASDAQ: ESLA, ESLAW) (“Estrella” or the “Company”), a clinical stage biopharmaceutical company developing CD19-targeted ARTEMIS® T-cell therapies to treat cancer and autoimmune diseases, today announced the successful completion of the first dose cohort in its ongoing STARLIGHT-1 Phase I/II clinical trial. Following a review of safety and efficacy data, the...Read more


Aptose Biosciences Announces Positive Clinical Safety Review Committee (CSRC) Approval to Dose Escalate in Phase 1/2 Tuscany Trial of Frontline Triple Drug Therapy with Tuspetinib Amid Complete Responses and Favorable Safety in First Cohort

February 20
Last Trade: 1.71 0.00 0.00

TUS+VEN+AZA triplet achieves complete responses (CRs) in difficult-to-treat TP53-mutated/CK AML and FLT3-wildtype AML patients, including a measurable residual disease (MRD) negative remission Dosing of initial 40 mg cohort complete; no prolonged myelosuppression or dose-limiting toxicities No dose reductions to the standard-of-care components of the regimen (AZA/VEN) in first cohort CSRC endorses escalation to 80 mg dosing,...Read more


IMUNON Announces Translational Data from Phase 1/2 OVATION 2 Study of IMNN-001 in Advanced Ovarian Cancer

February 19
Last Trade: 1.25 0.83 200.84

Data reinforce dose-dependent mechanism with IMNN-001 100mg/m2 dose associated with 20% increase in IL-12 levels compared to 79mg/m2 dose Results from OVATION 2 Study continue to validate TheraPlas® technology, demonstrating DNA-mediated production of key anti-cancer immune cytokines following treatment IMNN-001 continues to show favorable safety profile, with no reports of serious immune-related adverse events LAWRENCEVILLE, N.J.,...Read more


Cabaletta Bio Announces Updated Clinical Data Demonstrating Deepening Clinical Responses across Multiple Indications with Rese-cel at February Scientific Meetings

February 18
Last Trade: 1.75 -0.03 -1.69

Clinical efficacy continued to deepen over time with three SLE patients in DORIS remission, the first LN patient achieving complete renal response, and the first dermatomyositis patient maintaining a major TIS improvement; each of these patients discontinued all immunosuppressants and are off steroids as of the latest follow-up  Safety profile continues to suggest favorable risk-benefit in the first 10 patients dosed; 90% of...Read more


Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003

February 18
Last Trade: 2.81 -0.08 -2.77

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial  Average microdystrophin expression of 110% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation, best-in-class Duchenne muscular dystrophy gene therapy candidate  Encouraging early signals of potential cardiac benefit...Read more


BioCardia Reports Completion of Low Dose Cohort Enrollment for CardiALLO Phase I/II Clinical Trial of BCDA-03 Allogeneic Mesenchymal Stem Cells to Treat Ischemic Heart Failure of Reduced Ejection Fraction (HFrEF)

February 13
Last Trade: 1.96 0.06 3.16

Focus on Patients with Elevated Markers of Heart Stress and Inflammation Procedural enhancement using FDA Approved Morph DNA steerable guide for therapeutic delivery SUNNYVALE, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- BioCardia, Inc. [Nasdaq: BCDA], a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, announced today completion of enrollment and dosing in the low...Read more


Aptose Biosciences’ Frontline Triple Drug Therapy with Tuspetinib Achieves Notable Responses in Newly Diagnosed AML Patients in the Phase 1/2 TUSCANY Trial

February 12
Last Trade: 1.71 0.00 0.00

TUS+VEN+AZA triplet achieves Cycle 1 complete remission (CR) in TP53-mutated/CK AML TUS+VEN+AZA triplet achieves Cycle 1 complete remissions in FLT3-wildtype AML patients TUS+VEN+AZA triplet shows favorable safety with no alteration of VEN and AZA dosing PK levels of TUS in the triplet remain equivalent to levels as TUS or TUS+VEN therapy SAN DIEGO and TORONTO, Feb. 12, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or...Read more


INmune Bio Opens Phase II in High Dose Cohort of INKmune™ Trial in Prostate Cancer

February 12
Last Trade: 7.30 -0.20 -2.67

Medical review of Phase 1 cohorts demonstrated no safety issues Data to be presented at the 10th Annual Innate Killer Summit, March 3-5 BOCA RATON, Fla, Feb. 12, 2025 (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...Read more


Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa

February 6
Last Trade: 13.42 -0.24 -1.76

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued improvement in hearing, cognition and adaptive behavior Long-term safety data with median follow-up of two years, and out to more than four years, demonstrate that tividenofusp alfa was generally well tolerated Regulatory submission for accelerated approval is planned for early 2025; U.S. launch...Read more


Sangamo Therapeutics Announces Updated Phase 1/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit, Improvements in Kidney Function and Favorable Safety Profile

February 6
Last Trade: 0.47 -0.02 -4.35

Sustained benefit demonstrated with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for nearly four years for the longest treated patient as of the data cutoff date Positive mean estimated glomerular filtration rate (eGFR) slope observed in the 23 patients who had reached at least one-year follow-up, indicating notable improvements in renal function All 18 patients who began study on enzyme replacement therapy...Read more


Nervgen Pharma Completes Dosing of First Subject in Subacute Cohort of its Phase 1b/2a Clinical Trial of NVG-291 in Spinal Cord Injury

February 6
Last Trade: 5.10 -0.30 -5.56

Vancouver, British Columbia--(Newsfile Corp. - February 6, 2025) - NervGen Pharma Corp. (TSXV: NGEN) (OTCQB: NGENF), a clinical-stage biotech company dedicated to developing neurorestorative therapeutics, today announced that the first subject has been enrolled and dosed in the subacute cohort of its Phase 1b/2a proof-of-concept, double-blind, randomized placebo-controlled clinical trial (NCT05965700) evaluating its lead candidate,...Read more


Pyxis Oncology Initiates New PYX-201 Combination Trial and Initiates Cohort Expansions of Ongoing Monotherapy Trial

February 4
Last Trade: 1.23 0.03 2.50

Initiation of Phase 1/2 combination trial with PYX-201, an extracellular ADC targeting Extradomain-B Fibronectin (EDB+FN) and Merck’s KEYTRUDA® (pembrolizumab) in multiple solid tumors Phase 1 monotherapy trial expanded to include cohorts in recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC) Ongoing commitment to innovative cancer therapies for patients with limited treatment options BOSTON, Feb. 04, 2025 (GLOBE...Read more


Salarius Pharmaceuticals Announces Patient Enrollment to Resume in Investigator-initiated Phase 1/2 Clinical Trial Using Seclidemstat with Azacitidine to Treat Hematologic Cancers

February 3
Last Trade: 0.77 -0.0066 -0.85

HOUSTON, Feb. 03, 2025 (GLOBE NEWSWIRE) -- Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX) announces that patient enrollment will resume in the investigator-initiated Phase 1/2 clinical trial evaluating seclidemstat in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML). The trial is being conducted at the University of Texas MD Anderson Cancer Center (MDACC) and is...Read more


BriaCell Therapeutics Announces Resolution of Lung Metastasis in First Patient treated with Bria-OTS™

February 3
Last Trade: 3.04 -0.37 -10.82

Lung metastasis (from metastatic breast cancer) resolved - after only 2 months (4 doses) of treatment with Bria-OTS™ monotherapy No toxicity related to the treatment; Patient remains on study Unprecedented result in first patient dosed supports Bria-OTS™ personalized immunotherapy approach Ongoing Phase 1/2a dose escalation study to evaluate Bria-OTS™, BriaCell's personalized off-the-shelf immunotherapy in metastatic breast cancer...Read more


uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease

February 3
Last Trade: 15.16 -0.07 -0.46

Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment  Company expects to initiate enrollment of second dose cohort in the first quarter of 2025  LEXINGTON, Mass. and AMSTERDAM, Feb. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today...Read more


Invivyd Announces Positive Phase 1/2 Clinical Data for VYD2311, a Monoclonal Antibody Designed to be a Superior Alternative to COVID-19 Vaccination for the Broad Population

February 3
Last Trade: 0.75 -0.01 -1.64

Recruitment completed and all doses administered for VYD2311 ongoing Phase 1/2 clinical trial (40 subjects) evaluating 3 routes of administration Phase 1/2 clinical data for VYD2311 to date are positive for both safety and pharmacokinetics, and are supported by antiviral activity data from Invivyd's standard virologic assessments Pooled, blinded adverse events (AEs) to date are mild or moderate and thus far deemed unrelated to study...Read more


uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS

January 30
Last Trade: 15.16 -0.07 -0.46

Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort  Company Expects to Initiate Enrollment of Second Dose Cohort in the First Quarter of 2025  LEXINGTON, Mass. and AMSTERDAM, Jan. 30, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs,...Read more


INmune Bio Expands INKmune Trial in Prostate Cancer to Veterans

January 28
Last Trade: 7.30 -0.20 -2.67

First patient treated in Phase II portion of clinical trial in metastatic prostate cancer at the West Los Angeles Veterans Administration Hospital Boca Raton, Florida, Jan. 28, 2025 (GLOBE NEWSWIRE) --  INmune Bio, Inc. (NASDAQ: INMB) (the “Company”), a clinical-stage inflammation and immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, has an ongoing Phase...Read more


Adagene Announces Updated Data from Phase 1b/2 Study of Muzastotug in Combination with KEYTRUDA® (pembrolizumab) in Colorectal Cancer at ASCO Gastrointestinal Cancers Symposium

January 27
Last Trade: 1.82 0.29 18.95

Muzastotug (ADG126), an Anti-CTLA-4 SAFEbody® in Combination with pembrolizumab showed 33% overall response rate in microsatellite stable colorectal cancer Four confirmed partial responses out of twelve patients with 20 mg/kg loading dose followed by 10 mg/kg Q3W + pembrolizumab No Grade 4/5 treatment related adverse events were observed and no discontinuations occurred to date SAN DIEGO and SUZHOU, China, Jan. 27, 2025 (GLOBE...Read more


Exelixis Announces Encouraging Results from Phase 1b/2 STELLAR-001 Trial Evaluating Zanzalintinib Alone or in Combination with an Immune Checkpoint Inhibitor in Metastatic Colorectal Cancer at ASCO GI 2025

January 25
Last Trade: 42.39 -0.47 -1.10

In a subgroup analysis of patients without liver metastases, adding atezolizumab to zanzalintinib led to enhanced progression-free survival and overall survival ALAMEDA, Calif. / Jan 25, 2025 / Business Wire / Exelixis, Inc. (Nasdaq: EXEL) today announced results from an expansion cohort of the phase 1b/2 STELLAR-001 trial evaluating zanzalintinib alone or in combination with atezolizumab (Tecentriq®) in patients with...Read more


Novo Nordisk successfully completes phase 1b/2a trial with subcutaneous amycretin in people with overweight or obesity

January 24
Last Trade: 67.35 -0.82 -1.20

Bagsværd, Denmark, 24 January 2025 – Novo Nordisk today announced topline results from a phase 1b/2a clinical trial with amycretin, a unimolecular GLP-1 and amylin receptor agonist intended for once weekly subcutaneous administration. The trial investigated the safety, tolerability, pharmacokinetics, and proof-of-concept after once-weekly subcutaneous administrations of amycretin in 125 people with overweight or obesity. The trial was...Read more


Perspective Therapeutics Continues to Pursue Dose Escalation of [212Pb]VMT-α-NET in its Ongoing Phase 1/2a Clinical Trial Based on Updated Interim Data Presented at the 2025 ASCO Gastrointestinal Cancers Symposium

January 24
Last Trade: 2.42 -0.09 -3.59

Updated interim results with an additional 10 weeks of follow-up on the nine patients in Cohorts 1 (2.5 mCi) and Cohort 2 (5.0 mCi) of the ongoing Phase 1/2a study support continuation of dose-finding for [212Pb]VMT-α-NET [212Pb]VMT-α-NET continued to have a favorable safety profile, with no dose-limiting toxicities observed at the two doses tested Three of seven patients in Cohort 2 experienced investigator-assessed objective...Read more


Lisata Therapeutics and WARPNINE Announce Encouraging Preliminary Results from the Phase 1b/2a iLSTA Trial Evaluating Certepetide in Locally Advanced Non-Resectable Pancreatic Ductal Adenocarcinoma

January 23
Last Trade: 2.70 0.19 7.57

Data corroborate previously reported preclinical data demonstrating certepetide’s ability to enhance the effectiveness of immunotherapy Preliminary results to be presented at the 2025 ASCO Gastrointestinal Cancers Symposium BASKING RIDGE, N.J. and SUBIACO, Australia, Jan. 23, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative...Read more


Century Therapeutics Announces Investigator-Initiated Phase 1/2 Trial of its iPSC-derived iNK Cell Therapy CNTY-101 in B-cell Mediated Autoimmune Diseases Sponsored by Internationally Renowned Key Opinion Leaders

January 21
Last Trade: 0.56 -0.02 -2.65

Investigator-initiated trial (IIT) to be led by pioneers of cell therapy in B-cell mediated autoimmune disease, the Schett/Mackensen group at Friedrich-Alexander University Erlangen-Nürnberg  Trial to evaluate and enhance clinical experience with CNTY-101 in patients with systemic lupus erythematosus, lupus nephritis, idiopathic inflammatory myopathy, and diffuse cutaneous systemic sclerosis  PHILADELPHIA, Jan. 21, 2025...Read more


Regulatory Approval Clears Path for Oncolytics Biotech to Advance Promising Pancreatic Cancer Treatment, Following a Review of Safety Data

January 15
Last Trade: 0.43 0.03 6.99

New pancreatic cancer cohort moves forward with full enrollment of 30 patients in Stage 1 SAN DIEGO and CALGARY, AB, Jan. 15, 2025 /CNW/ -- Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, today announced that Germany's medical regulatory body, the Paul-Ehrlich-Institute (PEI), has approved the continuation of patient enrollment into Cohort 5 of the...Read more


Instil Bio Announces Clinical Progress in China for IMM2510/SYN-2510, a Clinical-Stage PD-L1xVEGF Bispecific Antibody

January 14
Last Trade: 21.02 -3.59 -14.59

ImmuneOnco announced dosing of first patient in its recently initiated Phase 1b/2 clinical trial of IMM2510/SYN-2510 in combination with chemotherapy in patients with advanced NSCLC in China ImmuneOnco announced initial clinical data from the 1L advanced NSCLC trial in China is expected as early as 2H 2025 Instil is targeting initiation of a potential first-line advanced NSCLC clinical trial of IMM2510/SYN-2510 combined with...Read more


Korro Bio Announces Dosing of First Participants in REWRITE Phase 1/2a Study of KRRO-110 for Alpha-1 Antitrypsin Deficiency and Provides Pipeline Update

January 13
Last Trade: 13.80 -1.01 -6.82

KRRO-110 is the first product candidate from Korro’s proprietary RNA editing OPERATM platform Interim readout from REWRITE expected in the second half of 2025 Korro to advance additional pipeline programs towards clinical development CAMBRIDGE, Mass., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on...Read more


Ocugen Announces Positive 2-Year Data Across Multiple Mutations from Phase 1/2 Clinical Trial of OCU400 —A Novel Modifier Gene Therapy for Retinitis Pigmentosa

January 13
Last Trade: 0.91 -0.0039 -0.43

100% (9/9) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes at both one and two years 100% (9/9) of treated evaluable subjects demonstrated improvement or stabilization in mobility testing, which was only performed up to one year Improvement in visual function was statistically significant (p=0.01, treated vs untreated eyes), regardless of mutation at two...Read more


Passage Bio Announces Interim Data from upliFT-D Study in FTD-GRN and Provides Business Updates

January 10
Last Trade: 0.37 0.02 6.08

PBFT02 demonstrated durable, elevated CSF PGRN levels and early evidence of reduction in plasma NfL levels, a disease progression biomarker, compared to published natural history data Evaluating Dose 2, 50% lower than Dose 1, in subsequent FTD-GRN and FTD-C9orf72 patients to allow for dose exploration and support regulatory strategy Expect to report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H...Read more


Dyne Therapeutics Reports New Clinical Data Showing Compelling Impact on Multiple Measures of Myotonic Dystrophy Type 1 (DM1); Dyne Plans to Initiate Registrational Expansion Cohort to Support Potential Submission for U.S. Accelerated Approval for DYNE-10...

January 10
Last Trade: 11.69 -0.21 -1.76

DYNE-101 in DM1: Dyne plans to initiate global Registrational Expansion Cohort of ACHIEVE trial with registrational dose of 6.8 mg/kg Q8W following study data showing splicing correction and robust and sustained functional improvements; potential to support H1 2026 submission for U.S. Accelerated Approval DYNE-251 in Exon 51 DMD: Based on recent FDA feedback, pursuing U.S. Accelerated Approval with dystrophin as surrogate endpoint;...Read more


Aptose Biosciences Announces First AML Patients Dosed with Tuspetinib Triplet Frontline Therapy in TUSCANY Trial

January 9
Last Trade: 1.71 0.00 0.00

SAN DIEGO and TORONTO, Jan. 09, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company, today announced dosing the first set of patients in the TUSCANY Phase 1/2 study with tuspetinib (TUS) in combination with venetoclax (VEN) and azacitidine (AZA) as a frontline triple drug combination (triplet) therapy for patients newly diagnosed with acute...Read more


Precision BioSciences: iECURE Reports Complete Clinical Response in First Infant Dosed with its In Vivo Gene Editing Candidate ECUR-506 in an Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency

January 9
Last Trade: 4.58 -0.06 -1.29

First in vivo gene insertion clinical trial dosing infants reports complete clinical response in the first participant at the lowest dose level (1.3 x 1013 GC/kg) of ECUR-506 from three months post exposure to the end of study (six months post exposure) as demonstrated by the removal of standard of care ammonia scavenging medicines, followed by absence of hyperammonemic crises and normalization of protein intake The OTC-HOPE Phase 1/2...Read more


Moleculin Biotech Highlights Development Progress of Annamycin, Phase 2 Data Outperforms Billion-Dollar Assets in AML, Phase 3 Data Readouts in 2025 & 2026

January 8
Last Trade: 0.94 -0.0047 -0.50

Received US Institutional Review Board (IRB) approval for pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) and engaged leading contract research organization (CRO); On track to begin dosing of Annamycin in combination with cytarabine for the treatment of R/R AML in Q1 2025 Expected timelines for recruitment updates and preliminary readouts of MIRACLE trial accelerated to 2H 2025 (n=45); 1H 2026 (n=~75-90) with potential...Read more


Jasper Therapeutics Reports Positive Data from BEACON Study of Briquilimab in Chronic Spontaneous Urticaria

January 8
Last Trade: 4.77 -0.10 -2.05

Rapid onset of deep and durable clinical responses observed across multiple dosing cohorts with a favorable safety profile Mean change in UAS7 from baseline of -26.6 observed in the 240mg single dose cohort at 8 weeks, multiple dosing regimens ≥120mg demonstrated UAS7 change of more than -25 points 100% (N=3) Complete Responses (UAS7 = 0) observed in the 240mg single dose cohort at 8 weeks and 66% maintained Well Controlled disease at...Read more


EDAP TMS Announces First Pancreatic Cancer Patient Treated with Proprietary HIFU Technology

January 8
Last Trade: 1.77 -0.03 -1.67

AUSTIN, Texas, January 8, 2025 - EDAP TMS SA (Nasdaq: EDAP), the global leader in robotic energy-based therapies, today announced that the first patient has been treated in a phase I/II PULS Trial sponsored by the Centre Léon Bérard, Lyon, France, evaluating proprietary High Intensity Focused Ultrasound (HIFU) technology for the treatment of pancreatic tumors. “The initiation of the PULS Trial represents a major milestone in efforts to...Read more


Nervgen Pharma Completes Enrollment in Chronic Cohort of its Phase 1b/2a Clinical Trial of NVG-291 in Spinal Cord Injury

January 2
Last Trade: 5.10 -0.30 -5.56

Topline data from the chronic cohort is expected in Q2 2025 Institutional Review Board (IRB) protocol amendment changes approved, and screening has been initiated for subacute cohort Vancouver, British Columbia--(Newsfile Corp. - January 2, 2025) - NervGen Pharma Corp. (TSXV: NGEN) (OTCQB: NGENF), a clinical-stage biotech company dedicated to developing neurorestorative therapeutics, today announced that it has enrolled the 20th and...Read more


Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-INHBE for the Treatment of Obesity

December 23
Last Trade: 15.13 -0.26 -1.69

ARO-INHBE targets a known pathway that signals the body to store fat in adipose tissue In preclinical studies ARO-INHBE reduced body weight and fat mass with a novel mechanism of action that may better preserve lean muscle mass compared to currently approved obesity therapies PASADENA, Calif. / Dec 23, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase...Read more


Immix Biopharma Announces Positive U.S. Clinical Data From First Four Patients in NEXICART-2 U.S. Trial of sterically-optimized CAR-T NXC-201 in relapsed/refractory Light Chain (AL) Amyloidosis

December 19
Last Trade: 1.99 -0.42 -17.43

All four patients treated with NXC-201 normalized their disease markers within 30 days of dosing, of which, two are already classified as complete responders (CR), and the remaining two are bone marrow MRD negative (10-6); all patients remain in response as of the data cutoff of Nov 14, 2024 Bone marrow MRD negativity predicts future CR; company believes remaining two patients could be confirmed as CRs in the coming weeks and...Read more


Ocugen: Data and Safety Monitoring Board Reviews Interim Safety Data of Phase 2 Subjects of OCU410 ArMaDa Clinical Trial for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

December 19
Last Trade: 0.91 -0.0039 -0.43

OCU410 has a very favorable safety and tolerability profile No serious adverse events related to the study drug have been reported, such as exudation, infectious endophthalmitis, intraocular Inflammation, anterior ischemic optic neuropathy, or vasculitis MALVERN, Pa., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing...Read more


Verastem Oncology Provides a Clinical Update for RAMP 203 Trial in Advanced KRAS G12C Mutant Non-Small Cell Lung Cancer

December 18
Last Trade: 8.31 0.83 11.10

No dose-limiting toxicities were observed in the RAMP 203 first triplet combination cohort of avutometinib and LUMAKRAS™ (sotorasib) plus defactinib in patients previously treated with a G12C inhibitor BOSTON / Dec 18, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced preliminary clinical data for the triplet combination of...Read more


Immunocore announces treatment of first patient in the Phase 1/2 trial of IMC-R117C targeting PIWIL1 in advanced gastrointestinal cancers

December 17
Last Trade: 31.16 -0.05 -0.16

The Phase 1/2 trial will assess the safety and clinical activity of IMC-R117C, as a monotherapy and in combination with standards of care, in patients with advanced cancers expressing PIWIL1 First immunotherapy program to target PIWIL1, a cancer-testis antigen overexpressed in a range of cancers, including colorectal cancer (OXFORDSHIRE, England & CONSHOHOCKEN, Penn. & ROCKVILLE, Md., US, 17 December 2024) Immunocore...Read more


Tenaya Therapeutics Reports Promising Early Data from MyPEAK™-1 Phase 1b/2 Clinical Trial of TN-201 for Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy

December 17
Last Trade: 0.43 -0.0049 -1.13

TN-201 Well Tolerated at 3E13 vg/kg Dose AAV9 Capsid Demonstrated Robust Delivery of TN-201 Transgene to Heart Muscle Cells Resulting in Increasing RNA Expression and an Increase in Protein Levels Observed at One Year Circulating Biomarkers and Other Clinical Measures Mostly Remained Stable or Improved from Baseline Tenaya Management to Host a Webcast Conference Call Today at 8:00 a.m. ET SOUTH SAN FRANCISCO, Calif., Dec. 17, 2024...Read more


Aprea Therapeutics Announces Twice Daily (BID) Dosing of Patients in ABOYA-119 Clinical Trial of ATRN-119 to Potentially Optimize Clinical Outcomes and Strengthen Clinical Path Forward

December 11
Last Trade: 1.66 -0.004 -0.24

Twice daily (BID) dosing regimen expected to maximize clinical benefit for patients by optimizing the activity of Aprea’s experimental drug, ATRN-119, over a 24-hour daily cycle New regimen potentially optimizes clinical outcomes and strengthens the clinical path forward ATRN-119 is the first macrocyclic ATR inhibitor to enter clinical trials DOYLESTOWN, Pa., Dec. 11, 2024 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq:...Read more


Astria Therapeutics Announces Positive Final Results from Target Enrollment in the ALPHA-STAR Phase 1b/2 Trial of Navenibart for HAE

December 11
Last Trade: 3.92 -0.16 -3.92

Navenibart Demonstrated 6 Months of HAE Attack Prevention with 1 or 2 Doses  90-95% Mean Monthly Attack Rate Reduction Supporting Chronic Dosing 2 or 4 Times Per Year  67% Attack-Free Rate Over 6 Months in Cohorts 2 and 3  Favorable Safety and Well-Tolerated Profile  Phase 3 Initiation on Track for Q1 2025  BOSTON / Dec 11, 2024 / Business Wire / Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical...Read more


ALX Oncology Announces New Data Demonstrating Evorpacept in Combination with Zanidatamab Generates Promising Antitumor Activity in Advanced Breast Cancer

December 10
Last Trade: 0.46 -0.01 -3.11

Data from Phase 1b/2 clinical trial to be presented at 2024 San Antonio Breast Cancer Symposium (SABCS) show encouraging clinical activity in patients with heavily pretreated HER2-positive breast cancer who had received multiple HER2-targeted agents, including fam-trastuzumab deruxtecan-nxki (ENHERTU®) Combination therapy was well tolerated with a manageable safety profile consistent with prior experience with each investigational...Read more


Lisata Therapeutics Announces Completion of Enrollment in the CENDIFOX Trial

December 10
Last Trade: 2.70 0.19 7.57

BASKING RIDGE, N.J., Dec. 10, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced the successful completion of patient enrollment in all three cohorts of the Phase 1b/2a CENDIFOX trial. This investigator-initiated trial, led by Dr. Anup Kasi at The...Read more


Curis Announces Additional Data from TakeAim Leukemia Study

December 10
Last Trade: 2.00 0.01 0.50

LEXINGTON, Mass., Dec. 10, 2024 /PRNewswire/ -- Curis, Inc. ("Curis") (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, yesterday presented data from the TakeAim Leukemia study (CA-4948-102) in relapsed/refractory (R/R) Acute Myeloid Leukemia (AML) at the 66th ASH annual meeting. The additional data presented include data for 21 patients with...Read more


AC Immune Reports Interim Safety Data from Phase 1b/2 ABATE Trial of ACI-24.060 in Down syndrome

December 10
Last Trade: 1.69 -0.41 -19.52

ACI-24.060 was generally safe and well tolerated in individuals with Down syndrome with no serious adverse events related to the study drug No cases of amyloid-related imaging abnormalities observed in this study population Based upon these findings, AC Immune plans to open the high-dose cohort in ABATE in individuals with Down syndrome Lausanne, Switzerland, December 10, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage...Read more


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