MyPEAK-1 Protocol Amendments Agreed Upon with FDA; Tenaya Implementing Changes with Sites SOUTH SAN FRANCISCO, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced it has received official notification from the U.S....Read more
TN-401 was Well Tolerated at 3E13 vg/kg dose Robust Transduction and Demonstrated Increases in PKP2 Protein Levels in First Two Patients at Week 8 Clinically Meaningful Reductions in Arrhythmia Burden Observed in First Two Patients with More Than Six Months of Follow-Up Tenaya Management to Host a Webcast Conference Call Thursday, December 11 at 5:00 pm ET to Review Preliminary Results SOUTH SAN FRANCISCO, Calif., Dec. 11,...Read more
MDNA11 demonstrates durable anti-tumor activity in phase-2 eligible expansion cohorts, enriched for immune checkpoint resistant melanoma, MSS endometrial cancer, MSI-H and TMB-H cancers, in each case exceeding objective response rate (ORR) benchmarks in these difficult to treat populations In the monotherapy expansion cohorts, irrespective of tumor type, patients treated with MDNA11, as the next treatment following progression on...Read more
Two-year epcoritamab monotherapy data demonstrate high complete response and encouraging survival rates in patients with Richter transformation (RT), highlighting its potential as a treatment option for those unsuitable for chemotherapy Additional early data show promising efficacy of epcoritamab combination regimens in patients with RT Results underscore the potential of epcoritamab as a versatile therapy for a broad range of B-cell...Read more
ARO-MAPT utilizes the Targeted RNAi Molecule (TRiM™) platform designed for subcutaneous administration and systemic delivery to the CNS by crossing the blood-brain-barrier Study initiation further highlights Arrowhead’s innovation and leadership in the delivery of siRNA PASADENA, Calif. / Dec 08, 2025 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1/2a...Read more
REC-4881 (4 mg QD) achieved rapid clinical activity, with 75% of evaluable patients showing reductions in total polyp burden and a 43% median reduction after 12 weeks of treatment (n=12) After 12 weeks off therapy (week 25 of the study), 82% of evaluable patients (9 of 11) maintained a durable reduction in total polyp burden, with a 53% median reduction observed from baseline Natural history analysis showed that 87% of untreated FAP...Read more
Registrational Expansion Cohort (REC) met primary endpoint, demonstrating statistically significant increase in dystrophin to 5.46% at 6 months (muscle content-adjusted; p<0.0001), replicating the same 7-fold change from baseline previously observed at the registrational dose Functional improvement was observed across multiple clinical endpoints at 6 months in REC; lung function was preserved at 6 months New positive...Read more
All three dose groups (50 mg, 100 mg and 200 mg) showed impressive monotherapy efficacy, with VGPR+ (very good partial response or better) of ≥70% despite limited follow-up; evidence shows that these responses are expected to deepen over time Across all dose groups, 95% (19 of 20 patients) of all evaluable VGPR+ patients achieved minimal residual disease negative status Data featured in an ASH oral presentation; LINKER-MM4 is the...Read more
Poster outlines clinical benefit with acceptable safety profile across 9 tested dosing regimens Accelerated step-up dosing and higher dosing frequency is feasible and results in increased exposure Six of eight responders presented with low bone marrow blast counts at baseline, supporting further investigation in this patient population most likely to benefit from MP0533 ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., Dec. 07,...Read more
Updated Data from 31 Adult and Adolescent SCD Patients Treated with risto-cel (Formerly BEAM-101) Show Mean Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to <40%, and Resolution of Anemia Durable for up to 20 Months Patients Required a Median of One Cell Collection Cycle and Experienced Rapid Neutrophil and Platelet Engraftment Safety Profile Remained Consistent with Busulfan Conditioning, Autologous...Read more
Study to evaluate safety and efficacy of novel anti-PRLR antibody; interim data anticipated in the second half of 2026 Trial design leverages operational synergies to enable accelerated registrational trials for androgenetic alopecia (AGA) and Phase 2 for endometriosis KOL seminar scheduled for December 11 to discuss new human ex vivo data, clinical trial design, and market opportunity in AGA VANCOUVER, Wash. and NEW YORK, Dec. 04,...Read more
Initiation of trial marks milestone for Crinetics’ novel nonpeptide drug conjugate platform SAN DIEGO, Dec. 03, 2025 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced the first patient has been dosed in the Phase 1/2 study evaluating CRN09682 in patients with metastatic or locally advanced somatostatin receptor type 2 (SST2)-positive neuroendocrine tumors and other SST2-expressing solid tumors....Read more
Vancouver, Canada, Dec. 02, 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 neuroplastogen-derived therapeutics to solve major under-treated health problems, today announced another key milestone in its Phase I/IIa clinical trial for the treatment of Alcohol Use Disorder (AUD): the first participant...Read more
Vancouver, Canada, Dec. 01, 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 neuroplastogen-derived therapeutics to solve major under-treated health problems, recently announced three transformative milestones in its FDA-approved Phase I/IIa clinical trial for CMND-100—the Company's proprietary,...Read more
First patient recruited at leading Israeli site accelerating enrollment momentum across global study of CMND-100 Vancouver, Canada, Nov. 25, 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 neuroplastogen-derived therapeutics to solve major under-treated health problems, today announced that Tel...Read more
Vancouver, Canada, Nov. 24, 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 neuroplastogen-derived therapeutics to solve major under-treated health problems, today announced that its independent Data and Safety Monitoring Board (DSMB) has completed a scheduled interim review of the Company’s ongoing...Read more
CAMBRIDGE, Mass. / Nov 24, 2025 / Business Wire / Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 (formerly ARO-DM1), an investigational small interfering RNA (siRNA) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) of the study are...Read more
Week 16 follow-up data, coupled with blinded exit interviews conducted up to 364 days after the study period, confirm durable and wide-ranging upper and lower-body improvements compared to placebo Participant-reported outcomes highlight substantial improvements in bladder control and muscle spasticity compared to placebo Statistically significant reduction of hyperactive reticulospinal signaling in upper and lower limbs (via...Read more
Nes-Ziona, Israel, Nov. 24, 2025 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. ( Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced positive six-month efficacy data from the Phase IIa stage of its randomized, multi-country Phase I/II Allocetra™ trial (ENX-CL-05-001) in patients with moderate to severe knee osteoarthritis (OA). The six-month follow-up has now been completed for...Read more
HOUSTON, Nov. 20, 2025 (GLOBE NEWSWIRE) -- FibroBiologics, Inc. (Nasdaq: FBLG) (“FibroBiologics”), a clinical-stage biotechnology company with 270+ patents issued and pending with a focus on the development of therapeutics and potential cures for chronic diseases using fibroblasts and fibroblast-derived materials, today announced that it has received both public and private Human Research Ethics Committee (HREC) approvals in Australia for...Read more
Prestigious Hadassah Medical Center Joins Yale, Johns Hopkins, Tel Aviv Sourasky and IMCA, in Evaluating the Company’s proprietary non-hallucinogenic MEAI-based oral drug candidate Vancouver, Canada, Nov. 20, 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 neuroplastogen-derived therapeutics to...Read more
New Clinical Trial Site Added for Clinical Studies of Reqorsa® Gene Therapy to Treat Lung Cancer Acclaim-1 and Acclaim-3 Clinical Trials Supported by FDA Fast Track Designation AUSTIN, Texas, Nov. 19, 2025 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the Company has...Read more
Vancouver, Canada, Nov. 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 neuroplastogen-derived therapeutics to solve major under-treated health problems, today announced positive top-line results from the first cohort of its Phase I/IIa clinical trial evaluating CMND-100, the Company’s proprietary...Read more
The 52-week symptom data reported for Cohort 6 patients is consistent with the long-term durability data previously reported from Cohort 5; Cohorts 5 & 6 are the only groups to reach 52 weeks thus far. In Cohort 6, a durable clinical symptom response was observed 52 weeks after a single administration of EP-104GI. All 3 patients in Cohort 6 maintained a clinical benefit and 2 out of 3 patients remain in clinical remission 52 weeks...Read more
Multidomain, durable gains with continued skill acquisition over time All 8 participants showed functional gains across spectrum of disease severity 35 total developmental milestones/skills acquired across 8 participants NGN-401 at the registrational dose continues to be generally well-tolerated, with no evidence of HLH Neurogene management to discuss results during Stifel 2025 Healthcare Conference webcast today at 4:40 p.m....Read more
Milestone advances MEAI-based therapy targeting a global Alcohol Use Disorder treatment market projected to surpass $20 billion by 2032 Vancouver, Canada, Nov. 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,...Read more
48-week data show a 64% decrease from baseline in proteinuria in IgA nephropathy, 82% decrease from baseline in proteinuria in primary membranous nephropathy, and stabilization of estimated glomerular filtration rate across both diseases Vertex on track to initiate rolling submission of Biologics License Application for potential accelerated approval to the U.S. Food and Drug Administration this year; full enrollment completed for Phase...Read more
Deep, stable and durable reductions in kallikrein observed Among 32 patients who received a 50 mg dose of lonvo-z as of data cutoff: 31 (97%) were attack-free and long-term prophylaxis (LTP)-free 24 (75%) were attack-free and LTP-free for at least seven months (up to 32 months) Among the 11 patients who originally received a 50 mg dose in Phase 2, 10 were attack-free and LTP-free Continue to observe a well-tolerated...Read more
MyPEAK-1 Data Presented During Late-Breaking Session at AHA Scientific Sessions 2025 with Simultaneous Publication in Cardiovascular Research TN-201 Has Been Generally Well Tolerated at Both Doses Longer-term Follow Up of Cohort 1 Patients Showed Consistent, Deeper, and Durable Improvement in Measures of Hypertrophy Initial Cohort 2 Data Demonstrated Early Dose Responsive Increases in TN-201 Transduction and MyBP-C Protein...Read more
Final ALPHA-STAR Phase 1b/2 Results from the Full-Enrollment Population (n=29) Remain Highly Consistent with Prior Results from the Target-Enrollment Population (n=16), Demonstrating Strong Efficacy, Favorable Safety, and Potential for Infrequent Dosing Phase 3 ALPHA-ORBIT Trial for Navenibart is Progressing as Planned with Topline Results Anticipated in Early 2027; First Patient Now Enrolled in the ORBIT-EXPANSE Long-Term...Read more
Single trial to support future BLA submission for NGN-401 in patients ages ≥ 3 years Rapid trial execution underway with 12 of 13 clinical sites initiated Expect to complete trial enrollment in three to six months Neurogene management to present Phase 1/2 interim data update at Stifel Healthcare Conference on November 12 NEW YORK / Nov 06, 2025 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to...Read more
4D-150 demonstrated consistent and durable benefit across all three patient cohorts as evidenced by maintenance of visual acuity, control of retinal anatomy and reduction of treatment burden at all time points with up to 2 years of follow-up Strong dose response in favor of Phase 3 dose (3E10 vg/eye) continues to be demonstrated 4D-150 continues to be well tolerated with no new safety or intraocular inflammation findings, consistent...Read more
EMERYVILLE, Calif. / Nov 03, 2025 / Business Wire / Estrella Immunopharma, Inc. (NASDAQ: ESLA) (“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 the successful completion of the second dose cohort in Phase I portion of its STARLIGHT-1 Phase I/II clinical trial of EB103, a CD19-redirected ARTEMIS®...Read more
Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301 BB-301 has also been granted Orphan Drug Designation from both FDA and EMA All six patients enrolled into Cohort 1 met the formal statistical criteria for response to BB-301, representing a 100% response rate Following the...Read more
CAMBRIDGE, MA / ACCESS Newswire / November 3, 2025 / Moderna, Inc. (NASDAQ:MRNA) today announced that the first patient has been dosed in a Phase 1/2 study of mRNA-2808, the Company's investigational mRNA-based T-cell engager (TCE) for participants with relapsed or refractory multiple myeloma (RRMM). The first dose was administered at SCRI Oncology Partners in Nashville, Tennessee, in collaboration with Sarah Cannon Research Institute...Read more
Milestone advances MEAI-based therapy targeting a global Alcohol Use Disorder treatment market projected to surpass $20 billion by 2032 Vancouver, Canada, Oct. 30, 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
First patient dosed in Phase 1/2a study with novel immune rejuvenator, a trispecific antibody-fusion protein Two MDX2004 abstracts accepted for poster presentation at SITC Annual Meeting 2025 WESTON, Mass., Oct. 28, 2025 (GLOBE NEWSWIRE) -- ModeX Therapeutics Inc., an OPKO Health company (NASDAQ: OPK), today announced the initiation and recent dosing of the first patient in a Phase 1/2a clinical trial (NCT07110584) with...Read more
Pelareorep plus atezolizumab achieves 30% ORR in second-line or later (≥2L) squamous cell anal carcinoma (SCAC) relative to 13.8% for the FDA-approved 2L treatment The median duration of response was 15.5 months compared to 9.5 months for the current standard of care Company expects to discuss single-arm accelerated approval study with FDA in Q1 2026 SAN DIEGO, Calif., Oct. 28, 2025 /CNW/ -- Oncolytics Biotech® Inc. (NASDAQ:...Read more
KYV-101 resulted in a profound reduction in disease-associated autoantibodies and impact on disease activity in patients with difficult-to-treat rheumatoid arthritis (RA) KYV-101 continues to demonstrate a well-tolerated profile, consistent with observations from 100 patients treated with KYV-101 to date1 Emerging IIT data in RA reinforce broad potential for KYV-101 in rheumatology indications EMERYVILLE, Calif., Oct. 25, 2025...Read more
First two monotherapy dose levels (400 mg QD and 600 mg QD) cleared, with no dose-limiting toxicities reported Promising anti-tumor activity observed in patients with various solid tumors, including advanced pancreatic ductal adenocarcinoma No nausea, vomiting, or diarrhea greater than Grade 1 was observed Enrollment initiated for VS-7375 in combination with cetuximab in patients with advanced KRAS G12D mutant solid tumors, including...Read more
Atlantic Health, a leading provider of health care based in New Jersey, to conduct the investigator-initiated Phase 1B/2 study Preclinical studies indicate Annamycin targets key factors in pancreatic cancer HOUSTON, Oct. 23, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and...Read more
2L MTAP-del pancreatic cancer median progression free survival (mPFS) 7.2 months with an objective response rate (ORR) of 25% Combination studies with RAS(ON) inhibitors ongoing, data anticipated 2026 49% ORR and mPFS 9.1 months in histology agnostic cohort of multiple late line, difficult to treat cancers provide further evidence of strong activity Company to hold conference call today at 8:30am ET – BOSTON,...Read more
Vancouver, Canada, Oct. 21, 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 announced the enrollment of the last patient for the first cohort of its Phase I/IIa clinical trial evaluating CMND-100, the Company’s...Read more
GFH375 resulted in a 41% ORR for patients with heavily pre-treated pancreatic ductal adenocarcinoma (68% had received 2 or more prior lines of therapy) at the 600 mg daily dose At month four, the OS rate was 92.2%; median OS has not been reached 91.5% of patients experienced a reduction in tumors and a disease control rate of 96.7%; nearly half the patients evaluated remain on treatment Low discontinuation rate due to adverse events of...Read more
Responses were rapid and durable, and tumor shrinkage was observed in 82 percent of patients New findings from this investigational study build on the strength of RYBREVANT® (amivantamab-vmjw) in non-small cell lung cancer and broadens its potential across additional solid tumors RARITAN, N.J., Oct. 19, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) today announced promising new results from the Phase 1b/2 OrigAMI-4 study...Read more
Encouraging preliminary activity observed across diverse set of advanced ALK-positive solid tumors Global enrollment ongoing for adult and adolescent patients with advanced ALK-positive solid tumors beyond NSCLC in a Phase 2 cohort of the ALKOVE-1 trial CAMBRIDGE, Mass., Oct. 18, 2025 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted...Read more
SOLANA BEACH, CALIFORNIA / ACCESS Newswire / October 7, 2025 / 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 announced results from a Phase I-II clinical study at Skåne University Hospital in Lund, Sweden evaluating the ClearPoint Prism Neuro Laser Therapy System's safety, feasibility, and efficacy. The study cohort...Read more
Robust improvement demonstrated across diverse set of clinical measures Patient-reported outcomes support clinical meaningfulness of improvements in function and strength Meaningful improvements in overall disease burden reported by both patients and physicians WALTHAM, Mass., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people...Read more
SEATTLE, Oct. 02, 2025 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. ("Perspective" or the "Company") (NYSE AMERICAN: CATX), a radiopharmaceutical company that is pioneering advanced treatments for cancers throughout the body, today announced that the first patient was treated with [212Pb]PSV359 in a second cohort of the Company’s Phase 1/2a dose-finding trial to determine safety and preliminary anti-tumor activity of the...Read more
Clinical data was reported for the first time in patients receiving an 8 mg dose per injection (Cohort 9 of the dose escalation portion of RESOLVE), the highest dose planned in this trial. Patients in Cohort 9 experienced the largest improvements in tissue health outcomes and eosinophil reduction observed to date. RESOLVE Safety Committee and members of the Eupraxia Clinical Advisory Board endorsed using the 8 mg dose per injection as...Read more
Results to be presented at the AACR Special Conference in Cancer Research: Advances in Pancreatic Cancer Research—Emerging Science Driving Transformative Solutions BASKING RIDGE, N.J., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases,...Read more
Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity score-matched external control High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures Mean cerebrospinal fluid...Read more
On track for first FDA Biologics License Application (BLA) approved cell therapy in orphan indication relapsed/refractory AL Amyloidosis – Los Angeles, CA, Sept. 18, 2025 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or ”IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, today announced that 50% enrollment milestone has already...Read more
No dose-limiting toxicities or cytokine release syndrome observed in RAINIER to date; mipletamig shows consistently favorable safety and tolerability Trial progressing efficiently, Cohort 4 open for enrollment SEATTLE, WA / ACCESS Newswire / September 16, 2025 / Aptevo Therapeutics ("Aptevo" or "the Company") (NASDAQ:APVO), a clinical-stage biotechnology company developing novel bispecific and trispecific immuno-oncology...Read more
SAN DIEGO, Sept. 16, 2025 /CNW/ -- Oncolytics Biotech® Inc. (Nasdaq: ONCY) ("Oncolytics" or the "Company"), a clinical-stage immunotherapy company developing pelareorep, today announced updates on enrollment progress, U.S. site expansion, and expected data readouts from the ongoing GOBLET trial evaluating pelareorep in gastrointestinal cancers. The study is supported in part by a grant from the Pancreatic Cancer Action Network...Read more
First patient dosed with [212Pb]VMT01 3.0 mCi in combination with nivolumab [212Pb]VMT01 3.0 mCi single agent dose Cohort re-opened SEATTLE, Sept. 15, 2025 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. ("Perspective" or the "Company") (NYSE AMERICAN: CATX), a radiopharmaceutical company that is pioneering advanced treatments for cancers throughout the body, announced today that the first patient was dosed in a new cohort...Read more
In preclinical studies presented today at the RNA Leaders USA Congress 2025, investigational ARO-MAPT demonstrated potent and long-lasting MAPT mRNA and Tau protein suppression with uniform distribution throughout the CNS after subcutaneous administration in non-human primates PASADENA, Calif. / Sep 10, 2025 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed a request for regulatory...Read more
Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and 10-Meter Walk/Run Test (10mWRT) at approximately one year Unprecedented rapid reduction in creatine kinase (CK) to near normal levels maintained over 16 months of follow-up and 25% increase of normal in dystrophin production, reflecting...Read more
Overall response rate (ORR) of 57% (4/7; 3cPR+1uPR) in patients treated with a combination of 30 mg IDE397 plus 7.5mg/kg Trodelvy® (Dose level 2); ORR of 33% (3/9; 3cPR) at 15 mg IDE397 plus 10mg/kg Trodelvy® (Dose level 1) Manageable safety profile at both expansion doses, consistent with known adverse events observed with each agent alone, with no treatment related serious adverse events observed at the IDE397 30mg and Trodelvy® 7.5...Read more
Rolling NDA submission for zidesamtinib in TKI pre-treated advanced ROS1-positive NSCLC initiated under FDA's RTOR program, with completion on track for the third quarter of 2025 CAMBRIDGE, Mass., Sept. 7, 2025 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today presented pivotal...Read more
All patients in the first cohort achieved between 27- to 208-fold increases in α-Gal A activity relative to mean normal level All patients in first cohort discontinued enzyme replacement therapy Preliminary data show AMT-191 has a manageable safety profile at the highest dose Updated clinical results expected in the first half of 2026 LEXINGTON, Mass. and AMSTERDAM, Sept. 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a...Read more
SOUTH SAN FRANCISCO, Calif., Sept. 4, 2025 /PRNewswire/ -- IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a leading precision medicine oncology company, today announced they have enrolled their first patient with non-small cell lung cancer (NSCLC) in the ongoing Phase 1/2 combination trial of IDE397, a potential first-in-class, small molecule adenosyltransferase 2a (MAT2a) inhibitor, and Trodelvy® (sacituzumab govitecan-hziy), a Trop2-directed...Read more
Achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses of WVE-006 (11.9 µM total AAT, 7.2 µM M-AAT) First-ever demonstration of therapeutically restored physiological serum AAT production in a Pi*ZZ individual during a non-drug related acute phase response (20.6 µM total AAT, 10.3 µM M-AAT) Single dose of 400 mg achieved 12.8 µM total...Read more
At 12 months, 2/3rds of Cohort 5 patients (48mg dose, 4mg per site) remained in clinical remission after their treatment with EP-104GI All Cohorts followed to 9 months have maintained clinically meaningful improvements in tissue health as measured by EoE Histological Scoring System (“EoEHSS”) No Serious Adverse Events (“SAE”) or any events of oral or gastrointestinal candidiasis have been reported to date in the entire...Read more
Median Overall Survival (OS) of 15 months for subjects with complete remission (n=8) Median OS of 2nd Line efficacy evaluable population of 12 months (n=9) Median OS of Intent to Treat population (1L-7L) of 9 months (n=22) OS demonstrated by Annamycin in the MB-106 trial is significantly above industry expectations for relapsed AML (4-6 months) Clinical study report anticipated in Q1 2026 Company continues to execute Part A of the...Read more
BRANFORD, Conn., Aug. 27, 2025 /PRNewswire/ -- Azitra, Inc. ("Azitra") (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced the dosing of the first patient in its Phase 1/2 clinical trial of ATR04-484, a topically applied live biotherapeutic product candidate designed to treat EGFR inhibitor ("EGFRi")-associated rash. Azitra has...Read more
At all doses and formulations evaluated in Part 1 of the trial, Z-1018 was well-tolerated and demonstrated a favorable tolerability profile, including lower solicited local and systemic post-injection reactions, versus Shingrix Z-1018 demonstrated robust immune responses in all dose arms, including a 100% humoral vaccine response rate at the dose selected for advancement, with comparable immunogenicity to Shingrix Dynavax selects the...Read more
ENX-CL-05-001 Trial: 3-months topline data In the overall modified intention-to-treat (mITT) population, improvements across all efficacy and secondary endpoints, including 24% reduction in knee pain and 26% improvement in knee function, were observed in the AllocetraTM treatment arm vs placebo; moreover, 72% reduction in knee pain and 95% improvement in knee function were observed for age-related primary osteoarthritis patients...Read more
Sonnet's lead product, SON-1010 (IL12-FHAB), is being evaluated in combination with atezolizumab (Tecentriq®) in patients with advanced platinum-resistant ovarian cancer (PROC) (SB221) Topline safety, cytokine, and efficacy data suggest a strong potential for clinical benefit using the current maximum dose of SON-1010 A second patient with PROC in the E6 combination cohort recently had a confirmed PR, so 2 out of 3 total patients had...Read more
BOCA RATON, FL, Aug. 04, 2025 (GLOBE NEWSWIRE) -- INmune Bio Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology and inflammation company, is pleased to report that its Phase I/II trial (the “CaRe PC” trial) of INKmune™ for men with metastatic castration-resistant prostate cancer (mCRPC) has met its primary and secondary endpoints and is now closed to further enrollment. INKmune™ was well tolerated at all three dose...Read more
Compelling new preliminary data consistent with previously reported preclinical findings demonstrating certepetide’s potential ability to enhance the effectiveness of immunotherapy BASKING RIDGE, N.J. and SUBIACO, Australia, July 17, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid...Read more
[IMAGES BELOW] Complete resolution maintained at 6 months in first patient treated with BriaCell’s Bria-OTS in Phase 1/2a study No treatment limited toxicities observed Patient remains on study with stable disease elsewhere PHILADELPHIA and VANCOUVER, British Columbia, July 09, 2025 (GLOBE NEWSWIRE) -- BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW, BCTXZ) (TSX: BCT) (“BriaCell” or the “Company”), a clinical-stage biotechnology...Read more
Eye drop administration of KB801 designed to enable sustained expression of NGF in the front of the eye Investor call and webcast to be held July 9 at 8:30 am ET to discuss program and trial design PITTSBURGH, July 09, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the first patient has been dosed in its Phase 1/2 clinical trial (“EMERALD-1”), a 2:1 randomized, double-masked,...Read more
In April 2025 the Sixth and Final Subject of Cohort 1 was Safely Treated with the Low Dose of BB-301 in the Phase 1b/2a Clinical Treatment Study (NCT06185673) Independent Data Safety Monitoring Board Review Has Been Completed for All Six Subjects Enrolled into Cohort 1, and the Data Safety Monitoring Board Recommended Continuation of Subject Enrollment for the Phase 1b/2a Clinical Treatment Study Following the Positive Data Safety...Read more
National Footprint of NEXICART-2 Trial Sites Expanded On track for first Biologics License Application (BLA) approved cell therapy in unaddressed orphan indication NEXICART-2 interim results were presented at ASCO 2025 LOS ANGELES, CA, July 07, 2025 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or ”IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL...Read more
8 of 9 participants (89%) treated in the 240mg and 360mg single dose cohorts achieved a complete response 8 of 11 participants (73%) enrolled in the 180mg Q8W open label extension study achieved a complete response at 12 weeks Results from the 240mg Q8W and the 240mg followed by 180mg Q8W cohorts appear to be confounded by issues with one drug product lot ETESIAN study in asthma to be stopped due to same drug product lot...Read more
Vancouver, Canada, June 30, 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 announced a historic milestone: the first participant has been dosed with CMND-100, its proprietary MEAI-based oral drug candidate, in its...Read more
BOSTON, June 27, 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 Phase 1/2 trial of TNG462 and Revolution Medicines’ daraxonrasib (RAS(ON) multi-selective inhibitor) or zoldonrasib (RAS(ON) G12D-selective inhibitor) in patients with MTAP-deleted...Read more
Attractive safety profile demonstrated across all dosing cohorts and routes of administration (IV, SC, and IM); all reported adverse events (AEs) deemed unrelated or classified as mild to moderate and largely related to injection site and infusion reactions with no serious or severe adverse events observed Following a single dose, serum concentrations of VYD2311 remained high at six months with an observed half-life of the IM dose...Read more
CMND-100 has the potential to provide an innovative and advanced treatment option for hundreds of millions of people around the world afflicted with Alcohol Use Disorder Vancouver, Canada, June 25, 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...Read more
Phase 1 dose-escalation study to evaluate VS-7375 in the U.S., with plans to expand as monotherapy into pancreatic cancer and non-small cell lung cancer cohorts, along with colorectal cancer in combination with cetuximab BOSTON / Jun 24, 2025 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that the...Read more
STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as primary basis of approval Isaralgagene civaparvovec showed a favorable safety and tolerability profile Sangamo intends to submit a Biologics License Application (BLA) in 2026 RICHMOND, Calif. / Jun 24, 2025 /...Read more
Aligned with FDA on NDA submission strategy for TKI pre-treated patients with advanced ROS1-positive NSCLC and participation in Real-Time Oncology Review; the company plans to initiate a rolling NDA submission in July 2025 with target completion in the third quarter of 2025 In 117 ROS1 TKI pre-treated patients, including 50% who had received ≥ 2 prior ROS1 TKIs ± chemotherapy, ORR by BICR was 44% (95% CI: 34, 53) with initial...Read more
Vancouver, Canada, June 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 announced the addition of Tel Aviv Sourasky Medical Center (TASMC), Tel Aviv, Israel, as an additional clinical site for its ongoing Phase...Read more
PBFT02 continued to demonstrate robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history Dose 2, 50% lower than Dose 1, substantially increased CSF PGRN levels at 30-days, reaching the upper limit of a healthy adult reference range Plan to amend upliFT-D protocol to include a prophylactic course of low dose anticoagulation and modify inclusion criteria to...Read more
[212Pb]VMT-α-NET Phase 1/2a study is advancing into Cohort 3 with a fixed administered dose that is up to 20% higher (6 mCi) than the dose administered to patients in Cohort 2 Dosimetry sub-study analysis presented at the Society of Nuclear Medicine & Molecular Imaging (SNMMI) 2025 Annual Meeting to advance utility of dosimetry in clinical development when considered with clinical data On track to submit further clinical updates...Read more
85% remission rate observed in evaluable frontline AML patients Mipletamig combination enables rare pathway to transplant in a previously ineligible unfit patient No dose-limiting toxicities observed among evaluable patients; safety profile remains strong Evidence mounts for mipletamig as a potentially transformational addition to standard of care in frontline AML as targeted CD123 x CD3 approach showing power and precision, limited...Read more
LYL314 demonstrated robust clinical responses, with an 88% overall response rate and a 72% complete response rate in patients treated in the third- or later-line setting (N = 25) 71% of patients with complete response remained in complete response at ≥ 6 months Manageable safety profile appropriate for outpatient administration with no Grade ≥ 3 cytokine release syndrome and low rates of Grade ≥ 3 ICANS with rapid resolution Pivotal...Read more
With up to three years of follow-up, a single dose of lonvo-z led to a 98% mean reduction in monthly HAE attack rate in all 10 patients All 10 patients were attack-free and treatment-free for a median of 23 months through the latest follow-up, demonstrating the potential of lonvo-z to become the first one-time therapy for most HAE patients Lonvo-z was well tolerated and continues to demonstrate a favorable safety profile The global...Read more
Results from the EPCORE® NHL-2 trial show investigational treatment with epcoritamab in combination with rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) led to an overall response rate (ORR) of 87 percent and a complete response (CR) rate of 65 percent in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) Data further demonstrates the potential of epcoritamab in combination with salvage...Read more
Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by rare and niche allergic and immunological diseases. Our lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development...
CLICK TO LEARN MORE