Porgaviximab

Inmazeb is the world’s first treatment for Ebola. Regeneron announced that the U.S. Food and Drug Administration approved Inmazeb, a three-antibody cocktail to treat Ebola infections in adults and children. Inmazeb is the world’s first treatment for Ebola. Although Regeneron Pharmaceuticals has been in the news lately for its antibody cocktail against COVID-19 that was used to treat President Trump, it’s not the only arrow in the company’s quiver. Today it announced that the U.S. Food and Drug Administration (FDA) approved Inmazeb, a three-antibody cocktail to treat Ebola infections in adults and children. It has also been approved for newborns of mothers who have tested positive for Ebola. Inmazeb is made up of three monoclonal antibodies: atoltivimab, maftivimab and odesivimab-ebgn. The cocktail was engineered using the company’s VelocImmune platform and associated VelociSuite technologies. The antibodies have similar structure, but bind to different, non-overlapping antigens on Zaire ebolavirus glycoprotein. They neutralize the virus by blocking its ability to invade patients’ bodies and by recruiting other immune cells to target infected cells. Ebola is rare and deadly and typically causes outbreaks in sub-Saharan Africa, although there were 11 people treated in the U.S. during the 2014 to 2016 epidemic. There is currently an ongoing outbreak in the Democratic Republic of the Congo (DRC). The disease spreads through contact with blood, body fluids and tissues of animals, and through contact with infected people, as well as through sexual contact with someone sick with the disease, as well as after recovery from the disease. The early signs of the disease include fever, aches and pains, severe headache, and abdominal pain, weakness, diarrhea and vomiting, then moves onto hemorrhaging, bleeding or bruising. The average case fatality rate of Ebola virus disease (EVD) is about 50%. In 2019, the FDA approved an Ebola vaccine, Merck’s Ervebo. “We are incredibly proud that the FDA has approved Inmazeb, which is also known as REGN-EB3,” said George D. Yancopoulos, president and chief scientific officer of Regeneron. “This is the first time the FDA has approved a treatment specifically for Ebola, which has caused a number of deadly outbreaks. Decades of investment in our VelociSuite rapid response technologies, the dedication of world-class scientists, and the courageous contributions of healthcare providers and patients, together with remarkable cooperation between leading international health organizations and govenrments, have led to this important moment.” As part of a deal announced in July, Regeneron will produce a specific number of Inmazeb doses over six years to the Biomedical Advanced Research and Development Authority (BARDA). In response to the 2018 outbreak in the DRC, Regeneron worked with the World Health Organization (WHO), FDA and other global health organizations to provide Inmazeb under a compassionate use protocol and include it in the four-arm PALM (Pamoja TuLinde Maisha) Trial. With BARDA, Regeneron plans to continue to offer the therapy for free in response to outbreaks in the DRC through the MNEURI protocol for compassionate use. “Since 2015, BARDA has partnered with Regeneron to develop a life-saving treatment for Ebola Zaire,” said Gary Disbrow, acting director of BARDA. “The Food and Drug Administration’s approval of Inmazeb shows the power of public private partnerships to bring forward these critical treatments and improve global public health. BARDA is continuing our collaboration with Regeneron on other life-threatening diseases such as MERS and COVID-19, and we look forward to continued success.” The therapy’s safety and efficacy were demonstrated in the 681-patient PALM Trial, which was initiated in 2018 in the DRC. The trial was jointly sponsored by the WHO, the National Institutes of Health (NIH) and the Institut National de Recherche Biomedical (INRB) in the DRC. The trial was halted early after a pre-specified interim analysis showed Inmazeb was superior to Gilead’s remdesivir, currently the only approved therapy for COVID-19, and Mapp Biopharmaceutical’s ZMAPP.

It was a typically busy week for clinical trials, with announcements in a wide variety of indications. Here’s a look. It was a typically busy week for clinical trials, with announcements in a wide variety of indications. Here’s a look. Array BioPharma announced the results from its Phase III BEACON CRC trial. The trial evaluated a triple combination of Braftovi (encorafenib), a BRAF inhibitor, Mektovia (binimetinib), a MEK inhibitor, and Erbitux (cetuximab), an anti-EGFR antibody, in patients with advanced BRAF V600E -mutant metastatic colorectal cancer (mCRC) after one or two lines of therapy. The bottom line is that the triple-combination almost doubled overall survival in the patient subpopulation. The patients who received the therapy, dubbed Braftovi Triplet, showed a statistically significant improvement in overall survival (OS) and objective response rate (ORR) compared to cetuximab plus irinotecan-containing regimens. Median progression-free survival (mPFS) for patients receiving Braftovi Triplet was 4.3 months compared to 1.5 months. A group of researchers at the University of Surrey , UK, conducted a clinical trial of 15 patients with non-muscle invasive bladder cancer. The patients were treated with a virus called CVA21, or ICAM-1-targeted immunotherapeutic-coxsackievirus A21. The CVA21 virus is a common cold virus. The trial was published in the journal Clinical Cancer Research . The approach was dubbed CAVATAK. Patients in the trial received CVA21 directly into their bladders by way of a catheter a week before surgery. After surgery, the researched studied tissue samples and found that the CVA21 virus only infected cancer cells, not healthy tissues. In addition, urine samples showed that the virus continued to grow and attack more cancerous cells. One patient was cancer-free one week after receiving the treatment. Sobi and Sanofi presented data from the A-LONG and ASPIRE and B-LONG and B-YOND clinical trials in hemophilia A and B, respectively. The focus was on the long-term outcomes of patients with hemophilia A and B who had switched from prophylaxis with Electa or Alprolix from on-demand treatment. Of 70 patients with hemophilia A who switched in the A-LONG/ASPIRE trials, 96% continued prophylaxis for more than six months with a median duration of 4.8 years. Of the 52 patients in B-LONG/B-YOND, 96% continued prophylaxis for more than six months with a median duration of prophylactic treatment of 3.6 years. New research from three clinical trials supports that Sarepta Therapeutics ’ Exondys 51 (eteplirsen) slows respiratory decline in Duchenne muscular dystrophy (DMD). Sarepta, along with Harvard Medical School, The Children’s Hospital of Philadelphia, Nationwide Children’s Hospital , the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center and Ohio State University published results from three trials in the Journal of Neuromuscular Diseases . These particular trials focused on respiratory function, comparing the patients receiving Exondys 51 to patients matched by age range, steroid use, and genotype from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS) global database. They studied eligible ambulatory DMD patients for at least four years, primary non-ambulatory DMD patients for two years, and an ongoing open-label multicenter trial of ambulatory DMD patients aged seven to 16 years. All three studies found significant slowing of respiratory decline in the patients receiving Exondys 51 and it was consistent across all stages of the disease studied. The U.S. Centers for Disease Control and Prevention (CDC) published research in the journal Lancet Infectious Diseases outlining the results from two experimental Ebola drugs, Gilead Sciences ’ remdesivir and Mapp Biopharmaceutical ’s ZMapp. Both successfully inhibited the growth of the virus in human cells in laboratory studies. “They’re currently being tested in a clinical trial but we need to verify and make sure that they were going to be effective,” said Laura McMullan , a CDC microbiologist who led the research, reported MSN . “Information that we knew about how well they worked was based on a different Ebola virus variant so we needed to make sure that, indeed, these compounds were going to bind and block the virus and measure that, and see if it worked as well in order for the clinical trials to proceed.” GlaxoSmithKline ’s ViiV Healthcare reported that HIV patients who shifted to Dovato (dolutegravir plus lamivudine) maintained viral suppression similar to patients on a tenofovir alafenamide fumarate (TAF-containing regimen of at least three drugs) for at least six months. The Phase III TANGO trial met its primary endpoint of non-inferiority, with no patients meeting virologic withdrawal criteria or developing treatment resistance. Biohaven Pharmaceutical Holding Company , based in New Haven, Connecticut, released positive data from its Phase III pivotal trial of rimegepant to treat migraine. The results were published in the New England Journal of Medicine (NEJM) . The patients in the trial, all adults, had at least a one-year history of migraine and two to eight migraine attacks of moderate or severe intensity per month. Of the 1,186 patients, 594 received the drug and 592 received placebo. Of those, 537 in the rimegepant group and 535 in the placebo group were evaluable for efficacy. The overall mean age of the patients was 40.6 years and 88.7% were women. In the rimegepant cohort, 19.6% were pain-free two hours after receiving the drug, while 12% were pain-free in the placebo group. In the rimegepant group, 37.6% were free from their most bothersome symptoms two hours after dosing and 25.2% were in the placebo group. Novartis , Amgen and the Banner Alzheimer’s Institute announced they were halting two pivotal Phase II/III clinical trials in the Alzheimer’s Prevention Initiative Generation Program. The trials were evaluating the BACE1 inhibitor CNP520 (umibecestat). An analysis of unblinded data during a scheduled review showed that the patients were getting worse in some of the measurements of cognitive function, the opposite of what they were hoping. The sponsors all decided that any patient benefit wasn’t worth the risk.