Clinical catch-up: Covaxin, Celyad Hold, the next step in CRISPR

The biopharmaceutical industry ended February and began March with a lot of clinical trial news. We’ll take a look.

Related to COVID-19

Ocugen reported that the FDA had lifted a clinical hold on its investigational new drug (IND) application for Covaxin, its COVID-19 vaccine. Indian company Bharat Biotech developed the vaccine. The lift will allow Ocugen to conduct a Phase II/III trial supporting its Biologics License Application (BLA) for the vaccine.

Adamis Pharmaceuticals announcement that due to the acceleration of patient enrollment in Tempol’s Phase II/III trial for COVID-19, the Data Safety Monitoring Board has determined that the study can proceed. No clinical or safety issues were observed. Data from the first 50 participants will be reviewed again in March. Tempol has demonstrated strong and broad in vitro anti-cytokine activity and anti-inflammatory properties.

RedHill Biopharma has announced positive results from phase II/III trial of RHB-107 (upamostat) in symptomatic COVID-19 patients not hospitalized. The trial is designed to evaluate the drug for symptomatic COVID-19 patients in the early stages of the disease. The trial was conducted in the United States and South Africa, and during this study period it primarily treated patients infected with the Delta variant. The data demonstrated a 100% reduction in COVID-19-related hospitalizations compared to 15% of hospitalizations in the placebo-controlled group.

Not related to COVID-19

Celyad Oncology announced that he was voluntarily suspending a clinical trial he was conducting with Merck after reporting the deaths of two patients. The Phase Ib CYAD-101-002 (KEYNOTE-B79) trial is evaluating Celyad’s CYAD-101 administered concurrently with FOLFOX chemotherapy followed by Merck’s anti-PD-1 checkpoint inhibitor Keytruda (pembrolizumab) in patients with refractory metastatic colorectal cancer. CYAD-101 is an allogeneic NKG2D CAR T therapy based on the TCR inhibitor molecule (TIM). Celyad has been notified of two deaths with similar pulmonary symptoms. The FDA formalized March 2.

GlaxoSmithKlineafter suspending three clinical trials of its respiratory syncytial virus (RSV) vaccine in pregnant women, has decided to stop registration and vaccination. The company said it was conducting analyzes of the safety data accumulated to date in the three trials, NCT04605159 (GRACE), NCT04980391 and NCT05229068.

Transcenta Holdings dose the first patient in China for its Phase IIa study of TST001 in combination with cisplatin and gemcitabine for the first-line treatment of patients with treatment-naïve locally advanced or metastatic systemic biliary tract cancer. The drug is a Claudin18.2 monoclonal antibody.

Evelo Biosciences announced post-treatment follow-up data (Part B) from its Phase II study of EDP1815 in mild and moderate psoriasis. The drug is an oral biologic in development for inflammatory diseases including psoriasis, atopic dermatitis and COVID-19. The data suggests lasting and deeper clinical responses.

NervGen Pharma completed dosing for five subjects in the first cohort of its multiple escalating dose portion of the Phase I study of NVG-291. NVT-291 is a close analog of NVG-291-R, which is an intracellular signaling peptide. NVG-291 is being developed for Alzheimer’s disease, multiple sclerosis and spinal cord injury.

Humacyte released five-year data from a phase II study of its human acellular vessel (HAV) for arteriovenous (AV) access in hemodialysis. The study reported long-term sustainable use and continued clinical function of HAV in patients with end-stage renal disease undergoing hemodialysis.

LivaNova implanted the first patient in its OSPREY clinical trial of its LivaNova aura6000 system, an implantable hypoglossal neurostimulator to treat adults with moderate to severe obstructive sleep apnea. The device is designed to maintain muscle tone in the tongue and upper airway to reduce or eliminate airway obstruction and resulting sleep apnea.

Therapeutic Intellia and Announcement from Regeneron Pharmaceuticals that the phase I study of NTLA-2001 demonstrated an average reduction in serum TTR of 93% at the highest dose on day 28 in patients with transthyretin amyloidosis (ATTR). The therapy is a CRISPR-based genome editing therapy. Intelia too announcement he had given the first patient NTLA-5001, his ex vivo candidate CRISPR-Cas9 for acute myeloid leukemia. NTLA-5001 is an autologous T-cell receptor (TCR)-T cell therapy that targets Wilms tumor antigen (WT1).

DURECT Corporation dosed the first patient in Europe in its Phase IIb AHFIRM study of larsucosterol in patients with severe alcohol-associated hepatitis (AH). Larsucosterol binds and inhibits the activity of DNA methyltransferases (DNMTs), an epigenetic enzyme elevated and associated with the hypermethylation observed in patients with HA.

NewAmsterdam Pharma dosed the first patient in its Phase III PREVAIL study of obicetrapib in patients with a history of atherosclerotic cardiovascular disease who do not have adequate control of their low-density lipoprotein cholesterol (ODO-c) despite receiving maximally tolerated lipid modifying treatments. The drug is a new cholesterol ester transfer protein (CETP) inhibitor.

Boehringer Ingelheim and Eli Lilly and company announcement results of the phase III EMPULSE trial. They found that adults hospitalized with acute heart failure were 36% more likely to experience clinical benefit over 90 days if initiated on Jardiance (empagliflozin) after stabilization and before discharge compared to placebo.

Clinical benefit reflected a composite primary endpoint including all-cause mortality, frequency of heart failure events, time to first heart failure event, and symptoms, as measured by total symptom score Kansas City Cardiomyopathy Questionnaire (KCCQ-TSS).

Therapeutic Actuate and University of Kansas Cancer Center registered the first patient in a phase II trial combining elraglusib and retifanlimab with gemcitabine/nab-paclitaxel for advanced pancreatic cancer as first-line treatment. Elraglusib is the company’s small molecule glycogen synthase kinase-3 beta inhibitor. Retifanlimab is Incyte Corporation’s intravenous PD-1 checkpoint inhibitor.

Suzhou Kintor Pharma dosed the first patient in its Phase II study of KX-826 (pyrilutamide) in the United States for male androgenetic alopecia (AGA). KX-826 is an androgen receptor antagonist.

NeuroGenesis and Hadassah Medical Center reported positive NF-L biomarker data from a phase II trial in progressive multiple sclerosis treating NG-01 cell therapy. NF-L is a protein released into the CSF by damaged neurons. The Company’s technology involves harvesting the patient’s bone marrow, then using a proprietary process, identifying a subpopulation of bone marrow cells cultured and enhanced into remyelinating biofactor cells (NG-01) that also possess neurotrophic and neuroprotective.

Project X initiated dosage in the phase I study of prosetin in healthy volunteers and people with ALS. Prosetin is a potent, orally-activated, brain-penetrating protein kinase kinase kinase (MAP4k) inhibitor that targets endoplasmic reticulum (ER) stress.

AmMax announced additional positive data from its Phase II trial of AMB-05X by intra-articular (IA) injection for the treatment of tenosynovial giant cell tumor (TGCT). AMB-05X is a monoclonal antibody against CSF1R.

Genprex dosed the first patient in the Phase I/II Acclaim-1 study of Reqorsa Immunogene Therapy in combination with Tagrisso (osimertinib) in patients with advanced non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso from AstraZeneca. Reqorsa (quaratusugene ozeplasmid) is a systemic gene therapy delivery platform. The active ingredient is the TUSC2 gene, a tumor suppressor gene.

Innocoll reported positive initial results from a phase III INN-CB-024 study of Xaracoll in patients undergoing abdominoplasty. In patients receiving the drug, a decrease in pain intensity and opioid use was observed 24 hours after surgery. The Xaracoll (bupivacaine HCL) implant is a bioabsorbable collagen implant that relieves pain by delivering bupivacaine to the surgical site.

Therapeutic Xalud dose the first patient in a Phase IIa study of XT-150 for pain related to facet joint osteoarthritis (FJOA). The drug is a locally injectable plasmid DNA gene therapy expressing IL-10v, a proprietary modified variant of human interleukin-10 (IL-10).

LEXEO Therapeutics announced positive baseline expression and biomarker data from the low-dose cohort (Cohort 1) of its Phase I/II trial of lX1001. LX1001 is an investigational AAV-based gene therapy that delivers the protective gene APOE2 into the CNS of APOE4-homozygous Alzheimer’s patients to halt or slow disease progression.

Cytokinetics registration open for cohort 4 of its REDWOOD-HCM phase II trial of Aficamten for hypertrophic cardiomyopathy. The cohort will recruit patients with non-obstructive hypertrophic cardiomyopathy. The drug is a next-generation cardiac myosin inhibitor.

Elgan Pharma announced “outstanding results” from ELGN-GI phase III trial. GLGN-GI is a patented enteral insulin formulation for the treatment of intestinal malabsorption that causes food intolerance in premature infants.

Sanofi and Astra Zeneca reported positive results from the phase III MELODY study of nirsevimab against respiratory syncytial virus (RSV). Nirsevimab is a vaccine.

NuCana stopped its Phase III study in bile duct cancer with Acelarin after an interim futility analysis. The analysis revealed a higher objective response rate (ORR) in patients with bile duct cancer who received the drug plus cisplatin, but this did not translate into an overall benefit in terms of survival.

Aravive announcement new positive Phase Ib data from the ongoing Phase Ib/II trial of batiraxcept in clear cell kidney cancer (ccRCC) and new data from a high biomarker subset. Batiraxcept is an ultra-high affinity decoy protein that binds to GAS6, the only ligand that activates AXL, which inhibits the growth of metastases and tumors, and restores sensitivity to anti-cancer drugs.

Evofem Biosciences has completed its registration in its EVOGUARD Phase III trial of EV100 for the prevention of chlamydia infection in women and the prevention of urogenital gonorrhea infection in women. EVO100 (Phexxi) is lactic acid, citric acid, potassium bitartrate, an on-demand method of contraception.

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