Clinical Catch-Up: September 20-24

Clinical Trial Concept_Compressed

There was still some carryover from the European Society of Medical Oncology (ESMO) Congress 2021, but there was plenty of other clinical trial news last week. Here’s a look.


Gritstone bio dosed the first volunteer in its Phase I trial of GRT-R910 in subjects 60 years of age or older. GRT-R910 is a self-amplifying mRNA (SAM) second-generation SARS-CoV-2 vaccine to boost and expand the immunogenicity of first-generation COVID-19 vaccines.

AiCuris Anti-infective Cures started a pilot study with immunomodulator AIC649 for asymptomatic or mildly symptomatic COVID-19 patients. The study will determine if the treatment can prevent more severe symptoms, including respiratory failure that may develop with progression of the disease. AIC649 is an inactivated parapoxvirus.

Pfizer and BioNTech presented interim data from a Phase II/III study of their COVID-19 vaccine in children five to 11 years of age. The data had a favorable safety profile and “robust” antibody responses in the children who received two 10 microgram doses at 21 days apart. This was similar to a study at higher doses, 30 micrograms, in the 16 to 25-year-old group.

Johnson & Johnson announced new data, both real-world evidence and Phase III data, supporting that its one-shot COVID-19 vaccine had strong and long-lasting protection. The booster shot was 94% effective when given two months after the first dose in a U.S. study. The antibody levels were four to six times higher with the booster compared to the single shot alone. The study also found that a booster shot six months after the first shot might be even more protective, creating a twelvefold higher antibody level four weeks after the booster, regardless of age.

pHOXBIO announced results from its Phase II/III study of its novel prophylactic nasal spray in SARS-CoV-2, the virus that causes COVID-19. In the high-risk healthcare staff given pHOXWELL compared to placebo, there were 63% fewer infections. pHOXWELL is a combination of natural virucides and Vita Raphael, a proprietary solution, that is designed to prevent viral infection for coronaviruses, influenzas and rhinoviruses.

BetterLife Pharma announced that its wholly owned subsidiary, Altum Pharmaceuticals, and Pontificia Universidad Catolica de Chile, enrolled the first patient in the Phase I portion of the Phase I/II trial of AP-003 in COVID-19. The drug is an inhaled interferon alpha-2b product.

Corvus Pharmaceuticals announced results from its Phase III trial of mupadolimab for COVID-19. The data suggested improvement in the primary and key secondary endpoints compared to placebo. Mupadolimab is a humanized monoclonal antibody against CD73 with a proposed dual mechanism of activating B cells to generate immune responses to viruses and tumor antigens, as well as inhibiting production of immunosuppressive adenosine in the tumor micro-environment.

Enlivex Therapeutics initiated dosing in its Phase IIb study of Allocetra in severe and critical COVID-19 patients with ARDS. Allocetra is being developed as a universal off-the-shelf cell therapy to reprogram macrophages into their homeostatic state.

Gilead Sciences reported positive data from a Phase III trial of a three-day course of Veklury (remdesivir) for intravenous use for non-hospitalized COVID-19 patients at high risk for disease progression. In the study, the antiviral drug demonstrated a statistically significant 87% reduction in risk of COVID-19 related hospitalization or all-cause death by Day 28 compared to placebo. It also showed that there was an 81% decrease in risk for medical visits for COVID-19 or all-cause death by Day 28. In particular, the drug appears to be most effective when used early in the disease.


AstraZeneca and Daiichi Sankyo presented data on their Enhertu in previously treated patients with HER2-mutant (HER2m) unresectable and/or metastatic non-squamous non-small cell lung cancer (NSCLC). The results were from the HER2-mutant cohort of the pivotal DESTINY-Lung01 Phase II trial. Enhertu is a HER2-directed antibody drug conjugate.

The primary results demonstrated a confirmed objective response rate (ORR) of 54.9% in patients receiving Enhertu. One (1.1%) complete response (CR) and 49 (53.8%) partial responses (PR) were identified. A confirmed disease control rate (DCR) of 92.3% was observed with a decrease in tumor size in most patients.

The two companies also shared data from a Phase III trial of Enhertu showing superior progression free survival to Genentech’s Kadcycla (trastuzumab emtansine) for HER2-positive unresectable and/or metastatic breast cancer in patients who were previously treated with trastuzumab and ataxane. This was from the Phase III DESTINY-Breast03 study. It provided a 72% reduction in the risk of disease progression or death compared to Kadcycla.

AstraZeneca also presented data from the COAST Phase II study demonstrating oleclumab or monalizumab in combination with Imfinzi (durvalumab) improved PFS and ORR compared to Imfinzi alone in unresectable, Stage III NSCLC patients who had not progressed after concurrent chemoradiation therapy (CRT). Oleclumab is an anti-CD73 monoclonal antibody. Monalizumab is an anti-NKG2A monoclonal antibody. Imfinzi is an anti-PD-L1 checkpoint inhibitor.

The Janssen Pharmaceutical Companies of Johnson & Johnson presented new analysis from the CHRYSALIS study of Rybrevant (amivantamab-vmjw) monotherapy and a combination with Lazertinib in advanced NSCLC patients with epidermal growth factor receptor (EGFR) mutations who progressed after Osimertinib. The data demonstrated that patients receiving the combination therapy had higher activity and longer duration of response (DOR), even in patients who were found to be resistant to third-generation EGFR tyrosine kinase inhibitors (TKIs).

Regeneron Pharmaceuticals and Sanofi presented positive Phase III data from a combination treatment of Libtayo (cemiplimab) with a physician’s choice of platinum-double chemotherapy in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) irrespective of histology and across all PD-L1 expression levels. The combination was compared to chemotherapy alone. The study hit the primary overall survival (OS) endpoint and all key secondary endpoints.

Novartis presented positive results from the Phase III MONALEESA-2 trial of Kisqali (ribociclib) in combination with letrozole in HER2 breast cancer. It hit the mark in OS after five years in postmenopausal women with HER+HER2- advanced or metastatic breast cancer with no previous systemic treatment for advanced disease.

RhoVac completed patient recruitment of the Phase IIb BRaVac trial of RV001 in prostate cancer with a biochemical recurrence after curative intent therapy. RV001 is an immuno-oncologic drug that is presented to the immune system as an antigen, stimulating T-cells to identify and destroy cells that carry the protein.

Mirati Therapeutics presented data from the Phase I/II KRYSTAL-1 study of adagrasib alone and with cetuximab in non-small cell lung cancer.  The data showed that in the evaluable patients who received adagrasib, there was a disease control rate of 87%, and in enrolled patients, median PFS was 5.6 months.

Bayer initiated the FIND-CKD Phase III trial of Kerendia (finerenone) in addition to guideline-directed therapy on the progression of chronic kidney disease (CKD) in nondiabetic CKD. The primary objective is superiority of finerenone over placebo in delaying progression of CKD, based on mean rate of change in kidney function over time from baseline to month 32.

Daiichi Sankyo Company and Sara Cannon Research Institute announced new first-in-human data from DS-77300 in advanced solid tumors. The drug is a B7-H3 directed DXd antibody drug conjugate (ADC). They presented the dose escalation part of the Phase I/II trial at ESMO 2021. 

Genentech presented new data from the Phase III Impower-010 study, reinforcing the significant disease-free survival (DFS) benefit offered by Tecentriq (atezolizumab) for Stage II-IIIA NSCLC whose tumors express PD-L1. The drug decreased the risk of disease recurrence or death (DFS) by 34% compared to best supportive care.

OSE Immunotherapeutics announced positive final data from its Phase III trial of Tedopi in HLA-A2 positive advanced NSCLC after immune checkpoint inhibitor failure. Tedopi is a neoepitope-based cancer vaccine.

BeyondSpring Pharmaceuticals presented the final intention-to-treat dataset from its DUBLIN-3 Phase III trial of plinabulin in combination with docetaxel versus docetaxel alone for second or third-line NSCLC with EGFR wild type. Plinabulin is a selective immunomodulating microtubule-binding agent (SIMBA).

Veru updated clinical data from the positive Phase Ib/II trial of sabizabulin (VERU-111) in 80 men with metastatic castration resistant prostate cancer who have progressed on at least one novel androgen receptor targeting agent. Sabizabulin is a new oral chemical entity from a novel class of drugs that target unique binding sites on microtubules.

Fulcrum Therapeutics presented multiple posters and presentations on losmapimod for facioscapulohumeral muscular dystrophy (FSHD). The results from the Phase IIb ReDUX4 trial demonstrated clinically relevant benefit across multiple structural, functional and patient reported endpoints.

Hutchmed (China) Limited initiated a Japan registration-enabling bridging study for surufatinib in advanced neuroendocrine tumors (NETs). Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with VEGFR and FGFR.

Seagen and Genmab presented interim data from two cohorts of the Phase Ib/II innovaTV 205 multi-cohort trial of tisotumab vedotin in recurrent or metastatic cervical cancer. Initial data demonstrated encouraging and durable anti-tumor activity as first-line therapy, with a 55% ORR in advanced cervical cancer patients who had not received previous systemic therapy; and with tisotumab vedotin in combination with pembrolizumab in advanced cervical cancer patients who experienced disease progression after 1-2 lines of prior systemic therapy, with a 38% ORR.

Merck announced final OS data from the Phase III KEYNOTE-355 trial of Keytruda (pembrolizumab) in combination with chemotherapy for first-line treatment of metastatic triple-negative breast cancer. The combination demonstrated a statistically significant and clinically meaningful improvement in OS.

Byondis presented positive data from its pivotal Phase III TULIP trial of its ADC trastuzumab duocarmazine compared to physician’s choice treatment in pretreated HER2-positive unresectable locally advanced or metastatic breast cancer. The trial hit the primary endpoint of PFS, demonstrating a statistically significant improvement over PC-7 months for SYD985 compared to 4.9 for physician’s choice.

Clearside Biomedical completed dosing in Cohort 2 of the OASIS Phase I/IIa trial of CLS-AX (axitinib injectable suspension) in patients with neovascular age-related macular degeneration (wet AMD).  CLX-AX is a proprietary suspension of axitinib for suprachoroidal injection. Axitinib is a tyrosine kinase inhibitor (TKI).

Hutchmed initiated the SURTORI-01 Phase III study of surufatinib (Sulanda, in China) in combination with toripalimab compared to FOLFIRI for patients with advanced neuroendocrine carcinoma (NEC) who have progression of disease or intolerable toxicity after previous first-line chemotherapy. Toripalimab is marketed as Tuoyi in China by Shanghai Junshi Biosciences Co. Surufatinib is an oral angio-immune kinase inhibitor that selectively inhibitors the tyrosine kinase activity associated with VEGR and FGFR.

Accutar Biotechnology received the go-ahead from the FDA for its Phase I trial of AC0682 in estrogen receptor (ER)-positive breast cancers. AC0682 is an orally bioavailable, chimeric degrader molecule that targets and degrades ER alpha protein.

Rhythm Pharmaceuticals presented new data and analyses from a Phase II and Phase III trial of setmelanotide. The Phase III trial was new subgroup data from the 52-week study in Bardet-Biedl syndrome (BBS), demonstrated that the drug achieved statistically significant weight loss and hunger reduction. The complete topline data from the exploratory Phase II Basket Trial showed the drug hit clinically meaningful weight loss or BMI-Z reduction in 30% of study participants with obesity due to variants of the SRC1 gene.

Cellectar Biosciences completed part A of a safety and tolerability study of iopofosine I-131 in combination with external beam radiation (EBRT) in relapsed or refractory head and neck cancer. The investigator-initiated trial is being conducted by the University of Wisconsin.

Erasca dosed the first patient in HERKULES-3, a Phase Ib/II master protocol clinical study of ERAS-007 in combination with various agents in gastrointestinal cancer. The initial focus is on advanced colorectal cancer. ERAS-007 is a potential best-in-class ERK1/2 inhibitor.

Bayer initiated the FIND-CKD Phase III trial of Kerendia (finerenone) in addition to guideline-directed therapy, on the progression of chronic kidney disease (CKD) in patients with nondiabetic CKD. Kerendia is approved for use to reduce the risk of sustained eGFR decline, end-stage kidney disease, cardiovascular death, nonfatal myocardial infarction, and hospitalization for heart failure in adults with CKD associated with type 2 diabetes.

TG Therapeutics published results from the integrated safety analysis of Unoniq (umbralisib) for r/r lymphoid malignancies. The drug is a PI3k-delta and CK1-epsilon inhibitor. The drug is commercially available for r/r marginal zone lymphoma and follicular lymphoma.

TFF Pharmaceuticals announced topline results of its Phase I trial of Tacrolimus Inhalation Powder. The study demonstrated a promising safety profile and therapeutic drug levels at low doses. 

AstraZeneca and Merck reported positive data from the Phase III PROpel study of Lynparza (olaparib) in combination with abiraterone and prednisone as first-line treatment for metastatic castration-resistant prostate cancer (mCRPC) with or without homologous recombination repair (HRR) gene mutations. Abiraterone is Johnson & Johnson’s Zytiga. Lynparza is a PARP inhibitor. The Independent Data Monitoring Committee determined at a planned interim analysis that the study had hit the primary endpoint of rPFS in mCRPC patients who had not received previous treatment in the first-line setting, including NHAs or chemotherapy. The study also demonstrated a trend towards improved OS, but that data is not ready yet and it will continue to evaluate OS as a key secondary endpoint.

Capricor Therapeutics announced positive final data from the HOPE-2 Phase II trial of CAP-1002 in advanced Duchenne muscular dystrophy (DMD). CAP-1002 is the company’s cell-based therapy whose mechanism of action is immunomodulatory, anti-fibrotic and demonstrated to regenerate skeletal and cardiac muscle cells.

vTv Therapeutics announced results of a multiple ascending dose study of HPP737 for psoriasis. The drug is an oral phosphodiesterase type 4 inhibitor. 

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