Clinical Catch-Up: April 12-16

Clinical Trial Patient and Doctor_Compressed

It was a particularly busy week for clinical trial news, in part because of several big medical conferences. Here’s a look.


AstraZeneca announced that its Farxiga (dapagliflozin) failed to hit the endpoints in its Phase III DARE-19 study in hospitalized COVID-19 patients at serious risk of developing complications. It also failed to assist hospitalized patients with recovery. The drug is a first-in-class, oral, once-daily SGLT2 inhibitor approved by the FDA to decrease the risk of hospitalization for heart failure in type 2 diabetes and with cardiovascular disease or multiple cardiovascular risk factors.

Regeneron Pharmaceuticals announced the results from its Phase III trial of recently infected asymptomatic COVID-19 patients for its REGEN-COV (casirivimab with imdevimab) antibody cocktail. The therapy decreased the overall risk of the patients progressing to symptomatic COVID-19 by 31%, which was the primary endpoint of the trial. It also decreased the overall risk of progression by 76% after the third day. In addition, REGEN-COV decreased the duration of symptoms and significantly reduced viral levels.

Regeneron also reported results from a second trial of REGEN-COV, this trial evaluating the combination therapy’s ability to decrease the risk and burden of COVID-19 infection among household contacts of SARS-CoV-2 infected individuals. This was jointly run with the National Institute of Allergy and Infectious Diseases (NIAID). The trial hit both primary and key secondary endpoints, demonstrating the antibody cocktail decreased the risk of symptomatic infections by 81% in people who were not infected when they began the trial. On average, people receiving the antibody therapy who had symptomatic infection resolved their symptoms in one week, compared to three weeks with placebo.

Rigel Pharmaceuticals announced positive topline results from its Phase II trial of fostamatinib for hospitalized patients with COVID-19. The drug is an oral spleen tyrosine kinase (SYK) inhibitor.

Romark announced initial results from its Phase III trial of NT-300 (nitazoxanide extended-release tablets, 300 mg) for mild or moderate COVID-19. A median time to sustained response was similar for patients receiving NT-300 compared with placebo, but in mild disease, sustained response was decreased by 3.1 days. It also hit a secondary endpoint, demonstrating an 85% reduction in progression to severe disease. Nitazoxanide was originally developed to treat intestinal protozoan infections caused by Cryptosporidium parvum and Giardia lamblia. It also has broad-spectrum antiviral activity.

Inovio reported interim data from its Phase I study of its COVID-19 vaccine, saying it induced a robust T-cell response against multiple variants, both the Brazil and UK variants. The data came from clinical samples from the study. The vaccine, INO-4800, is made up of an optimized DNA plasmid and delivered directly into cells via a proprietary smart device.

Merck indicated it was moving its molnupiravir into the Phase III part of the Phase II/III MOVE-OUT study in outpatient COVID-19 patients based on a planned interim analysis from the Part II part. However, the Phase III portion in hospitalized patients will not proceed because the data suggests the drug is unlikely to demonstrate a clinical benefit in that patient population. Molnupiravir is an oral antiviral candidate.

The I-SPY COVID Trial, sponsored by Quantum Leap Healthcare Collaborative (QLHC) announced that, in addition to the razuprotafib and apremilast arms, the icatibant portion of the trial has concluded. Icatibant, which is marketed by Takeda as Firazyr for hereditary angioedema (HAE), was unlikely to impact critically ill COVID-19 patients. It was chosen because it is an antagonist of the bradykinin type 2 receptor, which may be involved in respiratory impairment in COVID-19. The I-SPY COVID Trial is a Phase II study in critically ill COVID-19 patients receiving high flow oxygen or mechanical ventilation.


Bristol Myers Squibb’s CheckMate-816 trial of Opdivo (nivolumab) plus chemotherapy significantly improved pathologic complete response (pCR) in resectable stage Ib to IIIa non-small cell lung cancer (NSCLC). The study showed 24% of patients receiving Opdivo and chemotherapy achieved pCR, compared to 2.2% of chemotherapy alone.

Bayer presented data from the Phase III CHRONOS-3 trial of IV Aliqopa (copanlisib) and rituximab in relapsed indolent non-Hodgkin’s Lymphoma (iNHL). It demonstrated significant improvement in PFS, with an ORR of 80.8%.

ImCheck presented data from an ongoing Phase I/II trial of ICT01 in r/r solid and hematologic cancers with no remaining standard of care. ICT01 is an anti-Buryrophilin 3A (BTN3A) monoclonal antibody. The drug induced a broad anti-tumor response in the tumor microenvironment.

Arcutis Biotherapeutics initiated a pivotal Phase III trial of roflumilast cream (ARQ-152) for mild-to-moderate atopic dermatitis in patients between the ages of 2 and 5 years of age. Roflumilast cream is a once daily topical formulation of roflumilast, a potent and selective phosphodiesterase type 4 inhibitor (PDE4).

Instil Bio presented clinical data from its study of ITIL-168 in a compassionate use program for metastatic melanoma. Of the 21 patients, 14 achieved an OR, four achieved a CR. ITIL-168 is an autologous cell therapy made from tumor infiltrating lymphocytes.

Oncternal Therapeutics presented a poster at AACR describing its Phase Ib trial of cirmtuzumab and paclitaxel in locally advanced/unresectable or metastatic HER2-negative breast cancer. Of the 15 intent-to-treat patients as of April 10, eight had a best response of PR, one of which remained durable for 52 weeks, and four had stable disease. Cirmtuzumab is a potential first-in-class monoclonal antibody targeting ROR1 (Receptor tyrosine kinase-like orphan receptor 1).

Oncurious announced encouraging data from its Phase I dose escalation trial of TB-403 in pediatric patients with r/r medulloblastoma (MB). TB-403 is a humanized monoclonal antibody against PIGF, which is expressed in several types of cancers.

SAGE Therapeutics and Biogen reported topline results from the Phase II KINETIC Study of SAGE-324 in people with essential tremor. The trial hit its primary endpoint of a statically significant reduction from baseline compared to placebo in The Essential Tremor Rating Assessment Scale (TETRAS) Performance Subscale Item 4 upper limb tremor score on Day 29. SAGE-324 is an oral neuroactive steroid (NAS) GABAA receptor positive allosteric modulator (PAM).

BeiGene presented interim analysis of its RATIONALE 303 Trial of tislelizumab in second- or third-line NSCLC. The drug continued to demonstrate meaningful survival benefits in this setting. Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody designed to minimize binding to FcyR on macrophages.

Lyra Therapeutics presented data from its Phase II LANTERN trial of LYR-210 for chronic rhinosinusitis. LYR-210 is designed to be administered in-office and delivers a sustained release therapeutic for up to six months at difficult-to-access nasal sites of inflammation as an alternative to surgery.

Myovant Sciences and Pfizer dosed the first patient in the Phase III SERENE trial evaluating the contraceptive efficacy of relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) in healthy women 18 to 35 years of age at risk for pregnancy. It will enroll 900 sexually active, healthy women with presumed normal fertility.

HOOKIPA Pharma announced positive preliminary Phase I immunogenicity data for HB-201 and HB-202 to treat Human Papillomavirus 16-positive cancers. HB-201 is a single-vector therapy and HB-201 and HB-202 are an alternating two-vector therapy for advanced metastatic HPV16+ cancers.

Synlogic presented data from the Phase I trial of SYNB1618 for Phenylketonuria (PKU). SYNB1618 is an oral Synthetic Biotic therapy designed to break down phenylalanine in the GI tract. The data indicated the drug was well tolerated and metabolically active.

BioSig Technologies completed enrollment in the PURE EP 2.0 clinical trial. The PURE EP System was used in all types of arrhythmia cases, including atrial fibrillation, ventricular tachycardia, and atrial flutter. PURE EP System is a computerized system intended for acquiring, measuring, recording and storing of electrocardiographic and intracardiac signals.

NKMax and Merck KGaA, Darmstadt, Germany, expanded their collaboration to include a Phase I/IIa trial of SNK-1 in combination with either gemcitabine/carboplatin or gemcitabine/carboplatin plus cetuximab (Erbitux) in locally advanced or metastatic NSCLC that has progressed after previous TKI therapy. SNK01 is an autologous natural killer cell therapy.

Bausch & Lomb and Novaliq GmbH announced statistically significant topline data from their Phase III GOBI trial of NOV03 (perfluorohexyloctane) as a first-in-class eye drop for dry eye disease (DED) associated with meibomian gland dysfunction (MGD). NOV03 is an investigational, proprietary, water-free and preservative-free solution based on EyeSol technology from Novaliq GmbH.

Peptilogics completed a Phase I trial of PLG0206 for prosthetic joint infections (PJI). The drug showed a positive safety profile and favorable pharmacokinetics. PLG0206 is a broad-spectrum anti-infective.

Amylyx Pharmaceuticals presented long-term survival data from the CENTAUR trial of AMX0035 in patients with amyotrophic lateral sclerosis (ALS). The CENTAUR trial was a 24-week study of 137 patients with ALS. AMX0035 is designed to reduce neuronal death and dysfunction by targeting endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

Zai Lab and NovoCure updated their Phase III LUNAR trial of Tumor Treating Field (TTFields) in stage 4 NSCLC after platinum failure. Novocure was informed that the pre-specified interim analysis for the study would be accelerated based on the length of accrual and the number of events observed to date. The interim analysis included data from 210 patients. The recommendation was for the trial to continue with no evidence of increased systemic toxicity. TTFields are electric fields that disrupt cancer cell division.

Burning Rock Biotech announced that its liquid biopsy assay had a strong performance in the FDA-led Sequencing Quality Control Phase II study. Burning Rock’s assay was the most accurate assay at 25ng input, with roughly equivalent sensitivity but superior precision compared to other assays.

Moderna announced positive interim Phase I data for its mRNA-1345 vaccine for respiratory syncytial virus (RSV) and 7-month interim Phase II data from its cytomegalovirus (CMV) virus vaccine. mRNA-1345 demonstrated increased RSV neutralizing antibodies in seropositive younger adults. For the CMV vaccine candidate, the data from the Phase II study also demonstrated neutralizing antibodies, with plans to initiate a Phase III trial later this year.

NervGen Pharma received the greenlight to conduct a Phase I trial of NVG-291 in Australia. The drug is a specific and selective protein tyrosine phosphatase sigma inhibitor. It is being developed to reduce the clinical effects of nerve damage caused by trauma, such as spinal cord injury, traumatic brain injury or stroke, or neurodegenerative diseases like multiple sclerosis or Alzheimer’s disease.

Agenus dosed the first cancer patients with agenT-797 in its Phase I trial. agenT-797 is an allogeneic iNKT cell therapy. The therapy has been advancing in trials in patients with acute respiratory distress syndrome (ARDS) secondary to COVID-19, but now they are studying it in cancer. iNKT cells penetrate tissues and have showed potent tumor killing activities.

Oligos Therapeutics began dosing the first cohort of hepatitis B patients in its ongoing ALG-000184-201 trial. The study is evaluating ALG-000184, a Class II capsid assembly modulator that inhibits viral replication.

TILT Biotherapeutics hit the primary endpoint of safety in its Phase I trial with TILT-123’s first dose. TILT-123 is being developed as a monotherapy or in combination with tumor infiltrating lymphocytes (TILs). The company’s TILT technology modifies the tumor microenvironment and eliminates its ability to suppress responses to cancer. TILT-123 is a 5/3 chimeric serotype adenovirus armed with two human cytokines, TNF-alpha and IL-2.

GlaxoSmithKline halted enrollment in its Phase II Induce-3 trial and Induce-4 Phase II trial of feladilimab in cancer. Induce-3 was studying the drug with Merck’s Keytruda in patients with PD-L1 positive recurrent locally advanced or metastatic head and neck squamous cell carcinoma. Induce-4 was studying the drug alone compared to Keytruda and chemotherapy.

Genentech announced new 2-year data from Part 2 of FIREFISH, its Phase II/III trial of Evrysdi (risdiplam) in infants aged 1-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The drug continued to improve motor function between months 12 and 24, including ability to sit without support. Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier.

Illuminaire Biotechnologies and Memorial Sloan Kettering Cancer Center got the greenlight from the FDA to begin a Phase I study of Illuminare-1. The drug is a small molecule, myelin binding agent under development as a surgical adjunct to improve the visualization and delineation of critical nerve structures intra-operatively in real time. It will be conducted in patients undergoing robot-assisted radical prostatectomy.

AFYX Therapeutics presented Phase II data of Rivelin Clobetasol for the treatment of oral lichen planus (OLP), a chronic inflammatory condition that causes painful mouth ulcers. The therapy is a novel mucoadhesive patch designed to deliver clobetasol to lesions on wet tissue surfaces. The data showed promise and were easy to apply, remove, well tolerated and associated with high compliance.

Horizon Therapeutics published new pooled data from the Tepezza (teprotumumab-trbw) Phase II and III trials. The data reinforced that the drug significantly improves eye bulging and double vision for TED patients. Tepezza is a fully human monoclonal antibody and a targeted inhibitor of the insulin-like growth factor-1 receptor (IGF-1R). TED is Thyroid Eye Disease, a serious, progressive and vision-threatening rare autoimmune disease.

TG Therapeutics announced positive data from two Phase III trials, ULTIMATE I and II, of ublituximab compared to teriflunomide in relapsing multiple sclerosis (RMS). Both trials met their primary endpoints, with ublituximab showing a statistically significant decrease in annualized relapse rate (ARR) over 96 weeks. Ublituximab is a glycoengineered monoclonal antibody targeting a unique epitope on CD20-expressing B-cells.

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