Clinical Catch-Up: June 29-July 3

Clinical Research_Compressed

Heading into the 4th of July holiday in the U.S., there was still a fair amount of clinical trial news last week. Here’s a look.

COVID-19-Related

Inovio released positive interim data from its Phase I clinical trial of INO-4800, its experimental vaccine against COVID-19. In the interim data in 40 healthy volunteers, 94% of the recipients demonstrated an overall immune response at Week 6 after two doses of the vaccine. At Week 8, the vaccine appeared to be both safe and well-tolerated with no serious adverse events. All of the adverse events were mild, grade 1 in severity. Most were redness at the injection site. In addition, in preclinical studies in animals, the vaccine provided full protection against the virus replicating in the lungs of mice that were vaccinated, then dosed with the virus.

Pfizer and BioNTech announced preliminary data from the most advanced of four of its vaccine candidates against SARS-CoV-2, the virus causing COVID-19. The BNT162 program is studying at least four vaccines, each having a different combination of mRNA format and target antigen. The preliminary data is for BNT1621b1, which encodes an optimized SARS-CoV-2 receptor binding domain (RBD) antigen.

In the 24 people who received the 10-microgram and 30-microgram doses, they all showed elevated levels of RBD-binding (receptor-binding domain) IgG concentrations of 4,813 units/ml and 27,872 units/ml at day 28, seven days after receiving the immunization. The companies say those concentrations are 8- and 46.3-times the GMC (geometric mean concentration) of 602 units/ml in a panel of 38 sera taken from patients who contracted COVID-19.

Abivax treated the first patient in its Phase IIb/III trial of ABX464 in COVID-19 patients at the University Hospital Center in Nice, France. ABX464 has a molecular action that upregulates a micro-RNA, miR-124, that acts as a brake on inflammation. It down-regulates multiple chemo- and cytokines involved in the COVID-19 cytokine storm.

DURECT Corporation initiated recruitment for its Phase II trial of DUR-928 in hospitalized COVID-19 patients with acute liver or kidney injury. The trial will enroll about 80 patients. DUR-928 is a first-in-class small molecule, new chemical entity and an endogenous epigenetic regulator. It is in Phase I and II development for three indications, including alcoholic hepatitis, acute liver or kidney injury, and chronic metabolic diseases such as NASH.

Biophytis received the go-ahead from the FDA to initiate a Phase II/III trial of sarconeos in COVID-19 patients with related respiratory failure. Sarconeos is an oral small molecule in Phase IIb trials in the US and Europe for sarcopenia and is being developed for Duchenne Muscular Dystrophy (DMD). It stimulates biological resilience via activation of the MAS receptor, with the belief that it improves muscle function, preserves strength, mobility and respiratory capacity.

Non-COVID-19-Related

Audentes Therapeutics reported two deaths in its gene therapy clinical trial. The therapy, AT132, is for X-linked myotubular myopathy. Audentes sent a letter to patient groups on May 6, indicating that a patient receiving a high dose of AT132 died from sepsis. Two other patients receiving the high dose also experienced serious side effects. Six weeks later, on June 23, the company sent out a second letter indicating one of the two patients had suffered from progressive liver dysfunction. Ultimately that patient died from a bacterial infection and sepsis.

Aileron Therapeutics enrolled the first patient in the Phase Ib schedule optimization part of its ongoing Phase Ib/II clinical trial. The trial is evaluating ALRN-6924’s potential to protect patients against chemotherapy-induced toxicities. The patients in this trial have p53-mutated extensive disease small cell lung cancer (SCLC) and are being treated with second-line topotecan after administration of ALRN-6924.

Aspira Women’s Health enrolled the first patient in a national trial of benign pelvic mass management. It will enroll more than 1,000 women for assessing ovarian cancer risk in women with an adnexal mass and a high probability for developing the disease. They will be assessed by a newly developed, multi-biomarker, proprietary algorithm based on AWH’s experience and specimen bank and leveraging FDA-cleared products for ovarian cancer risk, OVA1 and OVERA.

TFF Pharmaceuticals initiated its second clinical trial with Tacrolimus Inhalation Power. The Phase I single ascending dose and multiple ascending dose study will evaluate tacrolimus, an immunosuppressive agent for the prophylactic treatment of lung transplant rejection.

BioCardia resumed cases in its CardiAMP Heart Failure Trial since being paused due to COVID-19. The trial is evaluating CardiAMP cell therapy, an autologous bone marrow-derived mononuclear cell formulation to stimulate the body’s natural healing response in treating heart failure which develops after a heart attack.

Iterum Therapeutics announced topline results from its SURE 1 clinical trial for uncomplicated urinary tract infections (uUTI). Sulopenem is a novel anti-infective. There were two independent primary endpoints. First, in a patient population with baseline pathogens resistant to quinolones, the drug demonstrated superiority to ciprofloxacin. In the second population of patients with organisms susceptible to quinolones, sulopenem was not non-inferior to ciprofloxacin and did not hit the related primary endpoint.

Aptose Biosciences got the go-ahead from the FDA to initiate a Phase Ia/b clinical trial of CG-806 in acute myeloid leukemia. CG-806 is an oral FLT3/BTK inhibitor. It is currently in a Phase I dose escalation trial in patients with B-cell malignancies, including chronic lymphocytic leukemia and non-Hodgkin’s lymphomas (NHL), who have failed or are intolerant to current therapies.

Exelixis initiated the CONTACT-02 Phase III trial of Cabometyx (cabozantinib) in combination with Roche’s Tecentriq (atezolizumab) in metastatic castration-resistant prostate cancer (CRPC) who have been previously treated with one novel hormonal therapy. It is part of a clinical trial collaboration between the two companies that includes two additional Phase III pivotal trials, CONTACT-01 in metastatic non-small cell lung cancer and the planned CONTACT-03 trial in metastatic renal cell carcinoma.

CVRx published results from the BeAT-HF Phase III trial in the Journal of the American College of Cardiology. BAROSTIM NEO demonstrated clinically significant improvements in patient-centered symptomatic endpoints compared to the control group. BAROSTIM NEO sends electrical pulses to baroreceptors in the wall of the carotid artery.

Rubius Therapeutics completed dosing of the first dose-escalation cohort in its Phase I/II trial of RTX-240. RTX-240 is an allogeneic cellular therapy for patients with relapsed/refractory or locally advanced solid tumors.

AOBiome initiated a Phase IIb trial in pruritus (itch) associated with atopic dermatitis by B244 in patients with eczema (atopic dermatitis). B244 is a first-in-class, topical formulation of beneficial ammonia oxidizing bacteria formulated as a topical spray used twice daily for 28 days.

Altimmune dosed the first patient in its Phase Ib trial of NasoShield, a single dose intranasal anthrax vaccine candidate. The trial expects to enroll 42 healthy subjects who will receive NasoShield or placebo and be followed for six months.

Akero Therapeutics presented results from a 16-week analysis of secondary and exploratory endpoints in its Phase IIa BALANCED clinical trial of efruxifermin (EFX) in nonalcoholic steatohepatitis (NASH). Of the 40 patients who responded who had end-of-treatment biopsies, 48% showed at least a one-stage improvement in fibrosis (scarring) without worsening of NAFLD activity score (NAS) and 28% demonstrated at least a two-stage improvement in fibrosis. Also, 48% of responders hit NASH resolution without the fibrosis getting worse. There were also improved glycemic control and dyslipidemia (high triglyceride, cholesterol or fat phospholipids), in addition to weight loss, across all levels of dosages. The treatment was generally well tolerated.

Impel NeuroPharma published results from its SNAP 101 Phase I study of INP105 in healthy adults. INP105 is a powder formulation of olanzapine targeting the upper nasal cavity using the company’s propellant-enabled POD technology. Olanzapine is the most-used treatment for acute agitation but is currently only an intramuscular injection.

Helsinn Group and MEI Pharma announced they were discontinuing their Phase III trial of pracinostat with azacytidine in Acute Myeloid Leukemia (AML). The decision was made after an interim futility analysis by an Independent Data Monitoring Committee determined the trial was unlikely to meet the primary endpoint of overall survival (OS) compared to the control group. There were no safety issues reported.

Pracinostat is an oral histone deacetylase inhibitor. Azacytidine is a chemotherapy drug marketed under the brand name Vidaza by Celgene, a Bristol Myers Squibb company.

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