Altimmune gets FDA clearance for trial of intranasal immune modulator for treatment of early *redacted*

In preclinical studies sponsored by the National Institute of Allergy and Infectious Diseases, intranasal administration of RD-Ad5 vectors modulated the innate immune response to lethal challenge with a respiratory virus in mice and protected them from death. The immunomodulatory effects resulted in significantly decreased cellular inflammation and lower concentrations of IL-6 and other inflammatory cytokines in the lungs of treated animals compared to controls. Excessive production of inflammatory cytokines like IL-6 has been associated with the lung pathology and death in *redacted*. The protective effects were independent of any specific immunity or vaccine effects against the challenge virus. These protective effects were only observed with intranasal administration of RD-Ad5, and intramuscular administration provided no survival benefit. 

The company believes that treatment with T-*redacted* administered as a single intranasal dose to patients with an early onset of symptoms and recent diagnosis of *redacted* may prevent the progression to severe lung inflammation and thereby decrease the development of severe *redacted* and the need for hospitalization. The FDA has agreed that the Company may use its existing lot of RD-Ad5-based NasoVAX influenza vaccine for the planned T-*redacted* clinical trial allowing the company to immediately initiate the study.

Myovant submits NDA for relugolix combination tablet for the treatment of women with uterine fibroids

The NDA submission in uterine fibroids is supported by positive results from the Phase 3 LIBERTY program, which included two multinational replicate studies and an open-label extension study through one year. The NDA is the third regulatory application ant has submitted this year, following a Marketing Authorization Application to the European Medicines Agency in uterine fibroids and an NDA in advanced prostate cancer. ant is also advancing the Phase 3 SPIRIT program, evaluating relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) in women with pain associated with endometriosis, with data from a second Phase 3 study expected this quarter. SPIRIT 2, the first of the two Phase 3 studies of once-daily relugolix combination therapy in women with pain associated with endometriosis, met its co-primary efficacy endpoints and six key secondary endpoints. In addition, relugolix combination therapy was generally well-tolerated including minimal bone mineral density loss over 24 weeks. 

“An estimated five million women in the U.S. suffer from symptoms of uterine fibroids, which may include heavy menstrual bleeding, pain, and anemia – yet effective non-invasive treatment options are very limited,” said Lynn Seely, M.D., chief executive officer of ant Sciences. “If approved, we hope to redefine care for these women with relugolix combination tablet, a potential new treatment that demonstrated a predictable and clinically-meaningful reduction in menstrual blood loss while maintaining bone heh in the Phase 3 LIBERTY program.”


CTI BioPharma enrolls 1st patient in PRE-VENT Ph 3 trial

PRE-VENT is a randomized, double-blind, placebo-controlled multicenter study, which will compare pacritinib plus standard of care versus placebo and standard of care in 358 hospitalized patients with severe *redacted*, including patients with and without cancer. The primary endpoint of the trial will assess the proportion of patients who progress to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or die by Day 28.

“Initiation of patient enrollment in the PRE-VENT Phase 3 trial is an important step for CTI as we work towards providing a new therapeutic option for patients with severe *redacted*,” said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. “As a multi-kinase inhibitor, pactrinib has the potential to reduce the effects of the cytokine storm that occurs with the *redacted* infection, an inflammatory response that frequently leads to respiratory failure and need for mechanical ventilation.”

Cytokine storm is a pathological immune reaction that can be triggered by viral infection and can lead to serious complications, including acute respiratory distress syndrome (ARDS). Multiple inflammatory cytokines are upregulated in patients with severe *redacted*, including IL-1 and IL-6, and some patients have evidence of over-active macrophage activation. As a JAK2/IRAK-1 inhibitor, pacritinib may ameliorate the effects of cytokine storm via inhibition of IL-6 and IL-1 signaling. Furthermore, as a CSF-1R inhibitor, pacritinib may mitigate effects of macrophage activation syndrome. Of particular importance in this indication, pacritinib has not been associated with increased risk in infections in prior randomized studies, likely because it does not have inhibitory effects on JAK1.