Clinical Catch-Up: November 22-26

Clinical Trial_Compressed

Thanksgiving week was marked by numerous clinical trial announcements. Here’s a look.


NRx Pharmaceuticals announced an initial samples of Phase II vaccinated patients in order to assess the potential effectiveness of the BriLife vaccine against the Delta variant of the SARS-CoV-2 virus. Blood from an initial sample of 11 participants demonstrated effective neutralizing antibodies against the original wild-type Wuhan strain and was evaluated for antibodies to Delta. Of 11 tested, 10 could neutralize Delta.

Ocugen reported the U.S. Food and Drug Administration (FDA) had issued a clinical hold on its Investigational New Drug application (IND) to evaluate its COVID-19 vaccine, BBV152 (Covaxin). The vaccine is an investigational vaccine in the U.S., developed by Bharat Biotech in collaboration with the Indian Council of Medical Research (ICMR) – National Institute of Virology (NIV). It has emergency use authorization in 17 countries. Ocugen and Bharat Biotech are co-developing the vaccine in the U.S. and Canada.

Clover Biopharmaceuticals initiated a Phase II study of its COVID-19 booster vaccines. The candidate, SCB-2019, will be evaluated as a heterologous booster in people previously vaccinated with their CoronaVac or the AstraZeneca/FIocruz-Oxford COVID-19 vaccine.


Transgene and NEC Corporation announced preliminary immunogenicity and clinical data on TG4050 and the first six patients treated. The drug is an individualized neoantigen cancer vaccine being tested in ovarian cancer and head and neck cancer. The drug was well tolerated and was associated with the first signs of clinical activity in a Phase I study.

Lixte Biotechnology resumed enrolling patients into its pharmacologic study of its LB-100 in patients where surgical removal of their cancer is indicated. It was suspended in 2020 due to the COVID-19 pandemic. LB-100 is a first-in-class PP2A inhibitor.

Achieve Life Sciences announced the completion of the fifth and final Data Safety Monitoring Committee (DSMC) review of its Phase III ORCA-2 trial of cytisinicline for smoking cessation. There were no concerns regarding the study and the DSMC found study compliance was excellence and was progressing well despite COVID-19. The drug is a plant-based alkaloid that binds to the nicotinic acetylcholine receptor.

Intellia Therapeutics received approval for the amendment to its ongoing Phase I study in the UK of NTLA-2001. It will now include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The original patient population was ATTR amyloidosis patients with polyneuropathy (ATTRv-PN). NTLA-2001 is an in vivo CRISPR/Cas9-based genome editing candidate designed to inactivate the TTR gene in liver cells to prevent the production of TTR protein.

Affimed announced interim clinical data from the investigator-initiated Phase I/II trial at The University of Texas MD Anderson Cancer Center of cbNK cells pre-complexed with the company’s innate cell engager AFM13. There were no serious adverse events and there was a 100% objective response rate (ORR) with a 42% complete response rate (CRR) in 12 patients after the first of two planned cycles at the recommended Phase Ii dose.

Apollomics dosed the first patient in the Phase III trial of APL-106 (uproleselan injection) for adults with relapsed or refractory acute myeloid leukemia (AML) in China. The company licensed the Greater China rights for the drug from GlycoMimetics. The drug is designed to block E-selection, an adhesion molecule on cells in the bone marrow, preventing binding with blood cancer cells.

Diffusion Pharmaceuticals dosed the first patients in its Altitude Trial of trans sodium crocetinate in healthy volunteers subjected to incremental levels of physical exertion while exposed to hypoxic and hypobaric conditions, or “simulated altitude.” The idea is to determine the effectiveness of the drug in improving oxygen delivery to the blood and tissues during exercise under hypoxic conditions. Hypoxia is a serious complication of many conditions, including hypoxic solid tumors.

JAMA Neurology published a study today by researchers with Biogen, Brown University, University College London, VU University Medical Center, Amsterdam, the Netherlands, California Pacific Medical Center in San Francisco, Bioclinica in Newark, Calif., and Cytel in Waltham, Massachusetts looking at ARIA in two Phase III trials, ENGAGE and EMERGE. The combined studies included 3,285 participants with Alzheimer’s who received one or more doses. The researchers found 425 cases in the combined aducanumab group experienced ARIA (41.3%), and ARIA-edema was identified in 362 patients, or 35.2%. Of them, 94 (26%) had symptoms, such as headache, confusion, dizziness, and nausea. They found ARIA-microhemorrhage and ARIA-superficial siderosis in 197 patients (19.1%) and 151 patients (14.7%), respectively.

The study noted that most ARIA-E events were seen early in treatment, either while attempting to find the correct dose or shortly after hitting the target dose. It notes, “These results are consistent with clinical studies of other anti-ABeta antibodies in which ARIA also primarily occurred within the first months of treatment, while the risk of ARIA-E decreased during the subsequent treatment course.”

Aptevo Therapeutics reported a clinical update for its Phase Ib Expansion study of APVO436 in acute myeloid leukemia (AML). The preliminary data included one complete remission. APVO436 is a bispecific CD3XCD123 ADAPTIR designed to redirect the immune system to destroy leukemia cells that express CD123 molecules on their surface.

Ocuphire Pharma enrolled the first patients in its Phase III MIRA-3 trial of Nyxol Eye Drops for the reversal of pharmacologically-induced mydriasis (dilation of the pupil). It now has six sites enrolling patients and 10 more sites expected soon.

Scynexis published results from its Phase III VANISH-306 trial. The data showed oral ibrexafungertp demonstrated superiority over placebo for vaginal yeast infection. It demonstrated sustained clinical effect at Day 25 follow-up with 83.9% of patients achieving a complete resolution of symptoms.

Sterna Biologicals received approval in Germany to run a Phase IIa study of SB010 in patients with moderate to severe asthma. The drug is an inhaled liquid formulation of a special type of catalytic antisense oligonucleotide.

Merck announced that it had paused its Phase II IMAGINE-DR trial of a combination of MK-8507, a non-nucleoside reverse transcriptase inhibitor, and islatravir (ISL), a nucleoside reverse transcriptase translocation inhibitor, for HIV. Then, on November 23, both Gilead Sciences and Merck, announced they were pausing a Phase II trial of islatravir and lenecapavir in HIV. They stated, “This temporary pause has been implemented out of an abundance of caution, to allow the companies to consider potential protocol adjustments to the trial in light of Merck’s announcement on November 18 regarding the decision to stop dosing in the Phase II IMAGINE-DR clinical trial evaluating the once-weekly combination of MK-8507 and islatravir.”

Kura Oncology announced the FDA had placed a partial clinical hold on its Phase Ib KOMET-001 study of KO-539 in r/r acute myeloid leukemia (AML). The study was paused to investigate a reported death. KO-539 is an oral drug that targets the menin-KMT2A/MLL protein-protein interaction.

Akcea Therapeutics announced a drug it licensed from Pfizer in 2019 hit the primary endpoint in a Phase IIb trial in patients with elevated non-HDL-C and triglycerides. The drug, vupanorsen, is an antisense therapy. Ionis Pharmaceuticals acquired Akcea in 2020. The drug achieved a statistically significant drop in non-HDL-C at all doses tested at 24 weeks.

Hutchmed and AstraZeneca initiated a Phase III SACHI trial in China of Orpathys (savolitinib) in combination with AstraZeneca’s Tagrisso (Osimertinib). Orpathys is an oral, potent, and highly selective MET tyrosine kinase inhibitor (TKI). Tagrisso is a third-generation, irreversible epidermal growth factor receptor (EGFR) TKI. They will test the combination in locally advanced or metastatic EGFR mutation-positive non-small cell lung cancer (NSCLC) with MET amplification after disease progression on EGFR inhibitor therapy.

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