Mode
Text Size
Log in / Sign up

Phase I Clinical Trials

Latest Phase I trial results across all specialties

Filtering: Phase I Clear all
50 pipeline articles
Single 6 mg erdafitinib dose shows similar pharmacokinetics in mild or moderate hepatic impairment versus healthy controls.
Gastroenterology Phase I
Single 6 mg erdafitinib dose shows similar pharmacokinetics in mild or moderate hepatic impairment versus healthy controls. Single dose of erdafitinib showed no dose changes needed for mild or moderate liver issues
This Phase 1 open-label single-dose study evaluated pharmacokinetics of erdafitinib in 26 participants with hepatic impairment versus health…
A single dose of erdafitinib worked well in people with mild or moderate liver problems, showing no need for dose adjustments.
LP-184 Alone and With Spironolactone in Recurrent Glioblastoma: Phase 1a Results
Oncology Phase I
LP-184 Alone and With Spironolactone in Recurrent Glioblastoma: Phase 1a Results New drug combo may help brain cancer patients who have run out of options
This Phase 1a trial in 63 patients (16 with glioblastoma) found LP-184 was well tolerated but with more transaminitis and thrombocytopenia i…
A new drug combination could help brain cancer patients by making their current medicine work much harder inside the tumor.
MCH1 Receptor Antagonist RGH-706 Shows Weight Loss in Preclinical and Early Clinical Study
Diabetes & Endocrinology Phase I
MCH1 Receptor Antagonist RGH-706 Shows Weight Loss in Preclinical and Early Clinical Study New Drug Targets Hunger Brain Signals to Help Shed Pounds
A preclinical and phase I/II study evaluated the MCH1 receptor antagonist RGH-706 for obesity and Prader-Willi Syndrome. In diet-induced obe…
Scientists found a new medicine that may help people lose weight by turning off hunger signals.
Donor-derived anti-CD33 CAR T-cell therapy showed 20% overall response in 15 adults with relapsed AML/MDS.
Oncology Phase I
Donor-derived anti-CD33 CAR T-cell therapy showed 20% overall response in 15 adults with relapsed AML/MDS. Donor-Derived Cells Show Promise Against Leukemia Relapse
This Phase 1/2 study enrolled 15 adults with relapsed or MRD-positive CD33+ AML/MDS after allogeneic hematopoietic cell transplantation. Don…
A new treatment using donor immune cells shows early signs of fighting leukemia after transplant.
RADAR pipeline identifies 20 high-confidence retired antigen candidates from 4,664 initial candidates.
Genetics & Precision Medicine Phase I
RADAR pipeline identifies 20 high-confidence retired antigen candidates from 4,664 initial candidates. Computers Find Safe Targets for New Cancer Vaccines
This Phase 1 computational pipeline development study utilized an in silico analysis of 4,664 initial candidates derived from GTEx. The RADA…
Imagine a protein that your body ignores when you are healthy but attacks when cells turn cancerous.
Etalanetug reduces CSF tau biomarkers in dominantly inherited Alzheimer's disease in phase 1b/2 study.
Neurology Phase I
Etalanetug reduces CSF tau biomarkers in dominantly inherited Alzheimer's disease in phase 1b/2 study. Can a new drug slow Alzheimer's toxic tau protein in the brain?
This open-label phase 1b/2 study in 8 participants with mild-to-moderate cognitive impairment due to dominantly inherited Alzheimer's diseas…
An early study shows an experimental drug lowers two key tau proteins in spinal fluid for people with a rare inherited form of Alzheimer's.
Camonsertib plus gemcitabine shows preliminary activity in advanced solid tumors with DDR alterations
Oncology Phase I
Camonsertib plus gemcitabine shows preliminary activity in advanced solid tumors with DDR alterations New Drug Combo Helps Some Solid Tumors Grow Slower
This Phase Ib trial evaluated camonsertib plus gemcitabine in 76 patients with advanced solid tumors harboring DDR gene alterations. Tumor r…
This combination might offer a new hope for patients whose tumors have stopped responding to standard treatments.
Phase 1 trial of oral ASTX727 plus talazoparib in previously treated triple-negative metastatic breast cancer patients.
Oncology Phase I
Phase 1 trial of oral ASTX727 plus talazoparib in previously treated triple-negative metastatic breast cancer patients. New Drug Combo Stops Breast Cancer Growth
This Phase 1 study enrolled 34 evaluable patients with previously treated triple-negative, hormone-resistant, or HER2-negative metastatic br…
This new combination may help some patients keep their cancer stable for months when other options have run out.
Posdinemab shows linear pharmacokinetics and predicted tau reduction in Phase 1 AD study
Neurology Phase I
Posdinemab shows linear pharmacokinetics and predicted tau reduction in Phase 1 AD study New Alzheimer's Drug Could Silence Brain's Toxic Tangles
This Phase 1 mechanistic population pharmacokinetic-pharmacodynamic model evaluated posdinemab in 69 healthy adults and participants with Al…
A new Alzheimer's drug could silence toxic brain tangles before memory loss worsens, offering hope for millions of families facing early-sta…
Lirafugratinib sequencing with futibatinib shows feasibility in FGFR2-driven cancers based on early-phase trial data
Oncology Phase I
Lirafugratinib sequencing with futibatinib shows feasibility in FGFR2-driven cancers based on early-phase trial data Can a new cancer drug work better if you switch to another drug after it stops?
This Phase I/II preprint trial evaluated lirafugratinib in 30 patients with FGFR2-driven cancers, including intrahepatic cholangiocarcinoma.…
When a cancer drug stops working, switching to a different one might help because the two medicines attack the disease in slightly different…
HS-20093 shows response in lung cancers in Phase 1a/b trial
Oncology Phase I
HS-20093 shows response in lung cancers in Phase 1a/b trial New Drug Targets Deadly Lung Cancer Cells
A Phase 1a/b trial in 306 patients with previously treated advanced solid tumors found HS-20093, a B7-H3-targeted antibody-drug conjugate, h…
A new drug attacks a specific protein on lung cancer cells, helping more than half of patients with an aggressive type while remaining safe …
Early study of CDP1 plus chemotherapy showed promising activity and manageable safety in advanced penile cancer.
Oncology Phase I
Early study of CDP1 plus chemotherapy showed promising activity and manageable safety in advanced penile cancer. New Drug Combo Shrinks Advanced Penile Cancer Tumors
This prospective phase 1 study evaluated the safety and antitumor activity of CDP1 monotherapy or in combination with standard chemotherapy …
A new drug combo shrinks advanced penile cancer tumors, offering fresh hope for patients with rare, hard-to-treat disease.
Pirtobrutinib shows activity in relapsed and refractory marginal zone lymphoma patients in a Phase 1/2 trial.
Oncology Phase I
Pirtobrutinib shows activity in relapsed and refractory marginal zone lymphoma patients in a Phase 1/2 trial. Early trial shows pirtobrutinib helped some patients with relapsed marginal zone lymphoma.
This Phase 1/2 trial evaluated pirtobrutinib in 36 patients with relapsed and refractory marginal zone lymphoma. The investigator-assessed o…
A new drug helped more than half of patients with relapsed lymphoma shrink their tumors, offering hope for those who have run out of other t…
Phase 1b ABBV-368 combinations in recurrent/metastatic HNSCC show 14.3% overall response rate
Oncology Phase I
Phase 1b ABBV-368 combinations in recurrent/metastatic HNSCC show 14.3% overall response rate Early study of ABBV-368 plus other drugs showed limited tumor response in head and neck cancer patients.
This phase 1b, multicenter, open-label study enrolled 30 adults with recurrent or metastatic head and neck squamous cell carcinoma across th…
Early trials of ABBV-368 combined with other drugs showed limited tumor shrinkage in head and neck cancer patients despite a generally safe …
Izalontamab brengitecan shows activity in pretreated NSCLC with non-classical actionable genomic alterations
Oncology Phase I
Izalontamab brengitecan shows activity in pretreated NSCLC with non-classical actionable genomic alterations For lung cancer patients who have run out of options, a new drug mix shows promise but needs more time.
This phase 1 study evaluated izalontamab brengitecan (iza-bren) in 83 patients with locally advanced or metastatic NSCLC harboring actionabl…
Lung cancer patients with limited options saw tumors shrink in 40% of cases and disease controlled in 86% using a new drug mix with manageab…
TQB2618 plus penpulimab shows 52% response in PD-1 refractory Hodgkin lymphoma
Oncology Phase I
TQB2618 plus penpulimab shows 52% response in PD-1 refractory Hodgkin lymphoma A New Drug Duo Revives Hope for Hodgkin Lymphoma After Immunotherapy Fails
A phase Ib single-arm trial in 21 Chinese patients with PD-1/PD-L1 inhibitor-refractory classic Hodgkin lymphoma found TQB2618 plus penpulim…
A new two-drug combination revived hope for Hodgkin lymphoma patients who failed prior immunotherapy, achieving a 52% response rate in early…
Setidegrasib shows antitumor activity in advanced NSCLC and pancreatic cancer with p.G12D variants.
Oncology Phase I
Setidegrasib shows antitumor activity in advanced NSCLC and pancreatic cancer with p.G12D variants. A Drug That Shreds a Famous Cancer Target
This Phase 1 study evaluated setidegrasib in 203 patients with previously treated advanced solid tumors harboring p.G12D variants. The drug …
A first-of-its-kind drug called setidegrasib shrank lung and pancreatic tumors in patients with a cancer mutation that has frustrated drugma…
Single-dose fultagliptin benzoate pharmacokinetics and safety in patients with mild to moderate renal insufficiency versus healthy volunteers
Diabetes & Endocrinology Phase I
Single-dose fultagliptin benzoate pharmacokinetics and safety in patients with mild to moderate renal insufficiency versus healthy volunteers A New Diabetes Pill That Stays Longer When Kidneys Slow
This Phase I, non-randomized, open-label, parallel-control trial evaluated the pharmacokinetics, pharmacodynamics, and safety of a single or…
A new diabetes pill builds up in the body when kidneys slow, meaning some patients with mild or moderate kidney issues may need half the usu…
Phase I trial of regorafenib plus ipilimumab and nivolumab shows 27.6% response in MSS colorectal cancer
Gastroenterology Phase I
Phase I trial of regorafenib plus ipilimumab and nivolumab shows 27.6% response in MSS colorectal cancer Who Really Responds to a Stubborn Colon Cancer Combo?
A phase I trial in 29 patients with microsatellite-stable metastatic colorectal cancer tested the combination of regorafenib, ipilimumab, an…
A three-drug combo helped some patients with stubborn colon cancer, and immune markers now predict who will benefit most.
Phase 1 trial of zamtocabtagene autoleucel shows 75% response in relapsed/refractory B-cell NHL
Oncology Phase I
Phase 1 trial of zamtocabtagene autoleucel shows 75% response in relapsed/refractory B-cell NHL Can a new cell therapy help lymphoma patients who have run out of options?
A Phase 1 clinical trial in 12 patients with relapsed or refractory B-cell non-Hodgkin lymphoma evaluated zamtocabtagene autoleucel at two d…
Five of 12 lymphoma patients treated with a new cell therapy achieved complete remission with no cancer return after five years and no sever…
Ga-PSMA-11 PET/CT shows high sensitivity for local prostate cancer but lower for lymph nodes in phase 1/2 trial.
Urology Phase I
Ga-PSMA-11 PET/CT shows high sensitivity for local prostate cancer but lower for lymph nodes in phase 1/2 trial. Imaging test shows promise for detecting prostate cancer spread in early trial
A prospective phase 1/2 single-arm trial in 173 high-risk prostate cancer patients found Ga-PSMA-11 PET/CT had 0.971 sensitivity for local d…
A new imaging test found prostate cancer in lymph nodes for 56% of patients, leading doctors to cancel surgery for 13% and adjust radiation …
CD19/BCMA CAR-T cells show early signal in six patients with refractory generalized myasthenia gravis
Oncology Phase I
CD19/BCMA CAR-T cells show early signal in six patients with refractory generalized myasthenia gravis A One-Time Treatment Lets Patients with Severe Muscle Disease Stop Their Steroids
A Phase 1 study of 6 patients with refractory generalized myasthenia gravis found autologous CD19/BCMA CAR-T cells without prior lymphodeple…
A one-time infusion of engineered immune cells allowed patients with a debilitating muscle-weakening disease to stop steroids and live sympt…
Phase Ib/II trial of nintedanib plus nivolumab in pretreated NSCLC adenocarcinoma shows 25% 6-month PFS rate
Pulmonology & Critical Care Phase I
Phase Ib/II trial of nintedanib plus nivolumab in pretreated NSCLC adenocarcinoma shows 25% 6-month PFS rate New Drug Combo Helps Some Lung Cancer Patients Live Longer
A single-arm, open-label phase Ib/II trial in 52 patients with pretreated stage IIIB/IV NSCLC adenocarcinoma found a 6-month progression-fre…
A new drug pair safely extends life for some advanced lung cancer patients who previously tried immunotherapy or have high PD-L1 markers.
Phase 1/2 trial shows RT + bevacizumab + olaptesed pegol extends PFS and OS in unmethylated GBM
Oncology Phase I
Phase 1/2 trial shows RT + bevacizumab + olaptesed pegol extends PFS and OS in unmethylated GBM New Drug Combo Slows Brain Cancer Growth
A Phase 1/2 expansion arm in six patients with newly-diagnosed, unmethylated GBM found that adding bevacizumab to radiotherapy and olaptesed…
Adding a second drug to stop blood vessel growth gives patients with brain cancer more time before the disease returns.
Phase 1 trial of off-the-shelf NK cell therapy shows no DLTs or responses in relapsed/refractory AML
Oncology Phase I
Phase 1 trial of off-the-shelf NK cell therapy shows no DLTs or responses in relapsed/refractory AML A Safer, Ready-to-Use Cell Therapy Takes Aim at Tough Leukemia
A single-arm, open-label, dose-escalation Phase 1 trial enrolled 6 adults with relapsed/refractory AML who received SAR445419, an off-the-sh…
A new, ready-to-use cell therapy proved safe in a first human trial for aggressive leukemia, offering hope for patients who have run out of …
Mitoxantrone liposome plus CMOP shows high response rates in untreated peripheral T-cell lymphoma patients.
Oncology Phase I
Mitoxantrone liposome plus CMOP shows high response rates in untreated peripheral T-cell lymphoma patients. New Drug Combo Helps Most Patients With Hard-to-Treat Lymphoma
This phase 1b multicenter clinical trial evaluated mitoxantrone hydrochloride liposome combined with cyclophosphamide, vincristine, and pred…
A new drug combo wrapped in fat bubbles helped 89% of patients with hard-to-treat lymphoma shrink tumors without causing deaths.
Phase 1 trial shows feasibility of 3D CRT plus SBRT boost for metastatic spinal cord compression
Palliative Care Phase I
Phase 1 trial shows feasibility of 3D CRT plus SBRT boost for metastatic spinal cord compression Adding a second radiation boost helps spinal cord patients
A Phase 1 clinical trial in 13 patients with metastatic epidural spinal cord compression (MESCC) found the combined 3D CRT and SBRT boost re…
A second, highly targeted radiation treatment helps people with spinal cord compression move better and feel less pain without causing new n…
Reni-cel gene therapy shows transfusion independence in small phase 1-2 trial for β-thalassemia
Genetics & Precision Medicine Phase I
Reni-cel gene therapy shows transfusion independence in small phase 1-2 trial for β-thalassemia New Gene Edit Stops Blood Transfusions
A single-group phase 1-2 trial in 9 adults with transfusion-dependent β-thalassemia found all participants achieved neutrophil engraftment a…
A new gene edit reactivates natural red blood cell production, potentially ending the need for lifelong transfusions in adults with severe b…
Renizgamglogene autotemcel infusion showed engraftment and hemoglobin increases in severe sickle cell disease patients.
Hematology Phase I
Renizgamglogene autotemcel infusion showed engraftment and hemoglobin increases in severe sickle cell disease patients. One Gene Edit Stopped Painful Sickle Cell Crises in Most Patients
This single-group, open-label, multicenter Phase 1-2 trial enrolled 28 patients with severe sickle cell disease aged 12 to 50 years. Followi…
A single infusion of CRISPR-edited stem cells eliminated painful sickle cell crises in 27 out of 28 patients by switching the body back to p…
Anti-BCMA CAR T-cell therapy bb21217 with bb007 showed 69.4% response in relapsed multiple myeloma.
Oncology Phase I
Anti-BCMA CAR T-cell therapy bb21217 with bb007 showed 69.4% response in relapsed multiple myeloma. Memory Cells Make CAR T Last Longer
This Phase I study evaluated anti-B-cell maturation antigen CAR T-cell therapy bb21217 manufactured with the phosphoinositide 3-kinase inhib…
CAR T cells trained to act like long-term guards may keep multiple myeloma away for years instead of burning out quickly after initial treat…
Linvoseltamab shows improved outcomes versus real-world standard of care in relapsed/refractory multiple myeloma
Oncology Phase I
Linvoseltamab shows improved outcomes versus real-world standard of care in relapsed/refractory multiple myeloma New Hope for Myeloma Patients After Three Failed Treatments
A Phase 1/2 study with an external control arm compared linvoseltamab to real-world standard of care in 105 triple-class exposed or refracto…
Patients with multiple myeloma who failed three prior treatments show higher response rates and longer survival with linvoseltamab compared …
Pivekimab sunirine shows activity in frontline and relapsed Blastic plasmacytoid dendritic cell neoplasm.
Dermatology Phase I
Pivekimab sunirine shows activity in frontline and relapsed Blastic plasmacytoid dendritic cell neoplasm. A New Drug Shows Striking Promise for a Rare and Aggressive Blood Cancer
This phase I/II single-arm trial evaluated pivekimab sunirine in adults with Blastic plasmacytoid dendritic cell neoplasm (BPDCN). In the pr…
For a cancer with very few options, this therapy is changing the outlook for patients and creating a clear path to potentially curative stem…
First-in-human study finds VS-01 generally well tolerated in decompensated cirrhosis with ascites
Gastroenterology Phase I
First-in-human study finds VS-01 generally well tolerated in decompensated cirrhosis with ascites Early safety study of VS-01 infusion in 12 patients with advanced liver disease
This phase 1b, open-label, non-randomized study in 12 patients with decompensated cirrhosis, ascites, and covert hepatic encephalopathy foun…
A new liver treatment called VS-01 was safe for patients with advanced disease, causing mostly mild stomach issues in an early study of 12 p…
Phase Ib trial: Mam-A DNA vaccine plus endocrine therapy induces T cells in ER+ breast cancer
OB/GYN & Women's Health Phase I
Phase Ib trial: Mam-A DNA vaccine plus endocrine therapy induces T cells in ER+ breast cancer Can a breast cancer vaccine help the immune system target tumors?
A phase Ib clinical trial in 21 patients with estrogen receptor-positive breast cancer prior to surgery found that adding Mammaglobin-A DNA …
A breast cancer vaccine triggered specific immune cells to attack tumors in early trial patients, showing promising biological activity befo…
Pilot nationwide carrier screening in Singapore identifies 0.9% of couples at increased risk
Genetics & Precision Medicine Phase I
Pilot nationwide carrier screening in Singapore identifies 0.9% of couples at increased risk Singapore pilot program screens couples for genetic disease risk, finds 0.9% at higher risk
A pilot implementation study of a nationwide reproductive carrier screening program in Singapore, using a 112-gene panel, found 0.9% of 456 …
Singapore's new genetic screening program found 0.9% of couples carry risks for recessive diseases, sparking interest and support for expand…
Novel TWISTARE MRI technique shows potential for faster quantitative T2 brain mapping in preclinical validation
Radiology & Imaging Phase I
Novel TWISTARE MRI technique shows potential for faster quantitative T2 brain mapping in preclinical validation New MRI technique shows promise for faster, precise brain tissue mapping in lab tests
A preclinical validation study of the TWISTARE MRI technique for quantitative T2 and B1 mapping at 7T demonstrated high precision in phantom…
A new MRI technique called TWISTARE could cut scan time in half while keeping brain tissue maps just as precise as current methods.
Firmonertinib plus anlotinib shows preliminary efficacy in advanced EGFR-mutant NSCLC.
Pulmonology & Critical Care Phase I
Firmonertinib plus anlotinib shows preliminary efficacy in advanced EGFR-mutant NSCLC. Early trial shows firmonertinib plus anlotinib helps some advanced lung cancer patients
This single-arm, phase I/II trial evaluated firmonertinib combined with anlotinib in patients with advanced non-small cell lung cancer (NSCL…
Combining firmonertinib and anlotinib helped 64% of advanced lung cancer patients shrink tumors and keep disease controlled for over a year …
Subcutaneous amivantamab shows shorter administration time and fewer infusion reactions in advanced solid tumors
Oncology Phase I
Subcutaneous amivantamab shows shorter administration time and fewer infusion reactions in advanced solid tumors Could a simple injection replace hours-long cancer infusions?
A phase Ib clinical trial in 158 patients with advanced solid malignancies evaluated subcutaneous amivantamab administration. Compared to hi…
A new injection method for advanced cancer treatment cuts administration time to under 10 minutes and reduces severe side effects compared t…
Phase I study finds amrubicin-cisplatin with radiotherapy feasible in limited-stage SCLC
Pulmonology & Critical Care Phase I
Phase I study finds amrubicin-cisplatin with radiotherapy feasible in limited-stage SCLC Small study tests new drug combination with radiation for limited-stage small cell lung cancer
A Phase I dose-finding trial in 9 treatment-naïve patients with limited-stage small cell lung cancer established amrubicin 25 mg/m² plus cis…
A new drug combination with radiation showed a 100% response rate and 64.8% five-year survival in early-stage small cell lung cancer patient…
Phase 1 study finds molnupiravir 800 mg well-tolerated with no NHC accumulation in healthy Chinese adults
Infectious Disease Phase I
Phase 1 study finds molnupiravir 800 mg well-tolerated with no NHC accumulation in healthy Chinese adults How does a COVID-19 pill behave in the body? A small study in healthy men offers clues.
An open-label, fixed-sequence Phase 1 study in 16 healthy male Chinese adults evaluated the pharmacokinetics and safety of oral molnupiravir…
Understanding how a drug moves through the body is a crucial first step. A small, early study in healthy Chinese men found the active part o…
SMART radiation shows 90% 4-year local control in small oligometastatic abdominopelvic cohort
Urology Phase I
SMART radiation shows 90% 4-year local control in small oligometastatic abdominopelvic cohort Can targeted radiation safely treat small, recurring cancer spots in the abdomen?
A single-arm Phase 1/2 trial in 10 patients with solid tumor abdominopelvic oligometastases found Stereotactic MR-Guided Adaptive Radiation …
Targeted radiation safely controlled small recurring cancer spots in the abdomen for 90% of patients with only mild side effects after four …
Fulzerasib plus cetuximab shows 69% response in first-line KRAS-mutated NSCLC
Oncology Phase I
Fulzerasib plus cetuximab shows 69% response in first-line KRAS-mutated NSCLC Can a new drug combination shrink lung tumors in people with a specific KRAS mutation?
A single-arm phase 1b/2 trial in 47 treatment-naive patients with KRAS-mutated NSCLC found a confirmed objective response rate of 69% (90% C…
A new two-drug combination shrank tumors in nearly 70% of people with advanced lung cancer driven by a specific genetic mutation.
Lu-PSMA-617 plus pembrolizumab shows PSA response in mCRPC phase 1b/2 trial
Oncology Phase I
Lu-PSMA-617 plus pembrolizumab shows PSA response in mCRPC phase 1b/2 trial Can a new drug combo help men with advanced prostate cancer?
In a single-arm phase 1b/2 trial of 37 patients with mCRPC, Lu-PSMA-617 plus pembrolizumab achieved a 50% PSA decline in 28 participants (76…
A new drug combo helped 28 of 37 men with advanced prostate cancer cut their PSA levels in half while keeping side effects mostly mild to mo…
Ifinatamab deruxtecan shows antitumor activity in advanced treatment-refractory solid tumors in phase 1/2 trial
Oncology Phase I
Ifinatamab deruxtecan shows antitumor activity in advanced treatment-refractory solid tumors in phase 1/2 trial Early trial shows experimental drug shrinks tumors in some patients with advanced cancers
This first-in-human phase 1/2 trial of 97 patients with advanced treatment-refractory solid tumors found a confirmed objective response rate…
An early trial of a new experimental drug shrank tumors in 34% of patients with advanced cancers that had stopped responding to standard tre…
Phase 1b trial finds tafasitamab combinations manageable in Japanese B-cell NHL patients
Hematology Phase I
Phase 1b trial finds tafasitamab combinations manageable in Japanese B-cell NHL patients Early study tests tafasitamab alone and in combinations for Japanese lymphoma patients
A phase 1b trial in 24 Japanese patients with relapsed/refractory or untreated B-cell NHL assessed tafasitamab alone or in combination. All …
Japanese lymphoma patients received new drug tafasitamab alone or with others, showing manageable side effects like low blood counts and nau…
Phase 1 trial of WVT078 with/without WHG626 shows preliminary activity in relapsed/refractory multiple myeloma
Oncology Phase I
Phase 1 trial of WVT078 with/without WHG626 shows preliminary activity in relapsed/refractory multiple myeloma Can a new combination therapy help when multiple myeloma returns?
A phase 1 dose-escalation trial in 56 patients with relapsed/refractory multiple myeloma assessed the BCMA×CD3 bispecific antibody WVT078 al…
Adding a second drug to a new antibody therapy nearly doubled response rates in early trials for patients with returning multiple myeloma.
Modeling study predicts human pharmacokinetics for TAK-500, an anti-CCR2 antibody-drug conjugate
Oncology Phase I
Modeling study predicts human pharmacokinetics for TAK-500, an anti-CCR2 antibody-drug conjugate Early modeling study predicts how a new cancer drug candidate might behave in humans
A preclinical and clinical modeling study used cynomolgus monkey data and human parental antibody data to simulate human pharmacokinetics fo…
A computer model predicts that a new cancer drug stays in the body longer as the dose increases, helping scientists plan safe first-in-human…
Prime editing therapy shows early neutrophil activity in two patients with p47-deficient CGD
Genetics & Precision Medicine Phase I
Prime editing therapy shows early neutrophil activity in two patients with p47-deficient CGD Early trial shows gene editing therapy may restore immune function in two patients with rare disease
A Phase 1 trial in two participants with p47-deficient chronic granulomatous disease (CGD) found that autologous CD34+ cell therapy using pr…
Two patients with a rare immune disorder regained the ability to fight infections after receiving a new gene editing therapy that fixed thei…
AAV9 gene therapy shows biochemical improvement, stable motor scores in GM1 gangliosidosis phase 1-2 trial
Pediatrics Phase I
AAV9 gene therapy shows biochemical improvement, stable motor scores in GM1 gangliosidosis phase 1-2 trial Can gene therapy slow a rare, fatal childhood brain disease? Early trial shows some signs it might.
In a phase 1-2 trial of nine children with type II GM1 gangliosidosis, a single IV infusion of AAV9 encoding β-galactosidase increased CSF β…
A rare fatal childhood brain disease showed signs of slowing in an early trial where gene therapy reduced toxic buildup and stabilized some …
FMT + Nivolumab Shows 20% ORR in 10 ICI-Refractory GI Cancer Patients
Oncology Phase I
FMT + Nivolumab Shows 20% ORR in 10 ICI-Refractory GI Cancer Patients Could a gut bacteria transplant help when immunotherapy stops working for stomach cancer?
A phase I study in 10 patients with anti-PD-(L)1-refractory GI cancers found the combination of fecal microbiota transplantation (FMT) and n…
When stomach cancer resists standard immunotherapy, a gut bacteria transplant combined with treatment helped tumors shrink in two patients a…