The ID Pipeline: FDA Activity From the Week of May 5, 2019

Here is a look at infectious disease-related US Food and Drug Administration (FDA) news from the week of May 5, 2019.

Moderna Publishes Phase 1 Results on mRNA Vaccines Against 2 Potential Pandemic Influenza Strains

On Friday, May 10, 2019, Moderna, Inc. announced the publication of phase 1 results from 2 studies evaluating mRNA vaccines against H10N8 and H7N9 influenza viruses.

In findings published in the journal Vaccine, the studies demonstrated that mRNA vaccines were well-tolerated and elicited robust immune responses.

“Both seasonal and pandemic influenzas are serious public health problems, and there is a clear need for effective vaccines that can be quickly developed and deployed. Production of current flu vaccines takes significant time and requires virus or antigen production in cell-culture or eggs and in dedicated facilities,” Mike Watson, senior vice president of vaccine partnerships and health impact at Moderna and a study co-author, said in a press release. “These Phase 1 data highlight the potential of Moderna’s mRNA platform to demonstrate similar or better immunogenicity than existing vaccines, which can be rapidly produced in a multi-use facility.”

The full press release is available here.

BioCryst Pharmaceutics Unveils Phase 1 Results of IV Galidesivir for Marburg Disease and Yellow Fever

On Thursday, May 9, 2019, BioCryst Pharmaceuticals, Inc. announced the completion of a phase 1 clinical trial of intravenous (IV) galidesivir for the treatment of Marburg disease and Yellow Fever.

Galidesivir was generally safe and well tolerated in the placebo-controlled trial, which evaluated the safety, tolerability, and pharmacokinetics of escalating doses of the drug in 4 cohorts: 5 mg/kg, 10 mg/kg, 15 mg/kg, and 20 mg/kg.

“These results for the IV route of administration of galidesivir, which build on a previous successful Phase 1 trial for the IM route, support its continued development for serious and life-threatening infections from RNA viruses. We appreciate the government’s continued financial support for and collaboration on the program as we advance galidesivir into a trial in patients with Yellow Fever in Brazil during the upcoming Yellow Fever season,” Dr. William Sheridan, chief medical officer of BioCryst, said in a press release.

The galidesivir development program is substantially funded with federal funds from the National Institute of Allergy and Infectious Diseases and by the Biomedical Advanced Research and Development Authority.

The full press release is available here.

Merck Announces Phase 2 Trial Results for Investigational 15-Valent Pneumococcal Conjugate Vaccine in Infants

On Wednesday, May 8, 2019, Merck announced phase 2 trial results for an investigational 15-valent pneumococcal conjugate vaccine (V114) for infants.

According to a company statement, V114 met its primary endpoint by demonstrating noninferiority for the 13 serotypes contained in both vaccines. V114 also induced an immune response in infants for two additional disease-causing serotypes, 22F and 33F, which are not contained in PCV13.

“Children under the age of two are at increased risk for pneumococcal infection, which in some cases may lead to serious illnesses like pneumococcal pneumonia,” Dr. David Greenberg, study investigator and physician in the Pediatric Infectious Disease Unit of Soroka University Medical Center in Beer-Sheva, Israel, said in a press release. “These phase 2 data evaluating V114 in infants are encouraging and mark important progress to helping expand protection against pneumococcal disease for this vulnerable patient population.”

The full press release is available here.

Nabriva Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Lefamulin

On Wednesday, May 8, 2019, Nabriva Therapeutics submitted a marketing authorization application to the European Medicines Agency for both the oral and intravenous (IV) formulations of lefamulin.

Lefamulin is a potentially first-in-class, semi-synthetic pleuromutilin antibiotic for the treatment of community-acquired pneumonia (CAP) in adults 18 years of age and older. The application is supported by 2 phase 3 clinical trials, LEAP 1 and LEAP 2.

“The MAA filing for lefamulin represents another major milestone for Nabriva as we continue to make significant progress toward our commitment of addressing the urgent and unmet medical need for novel anti-infective agents for patients with serious infections,” Dr. Jennifer Schranz, chief medical officer of Nabriva Therapeutics, said in a press release. “The spread of bacterial resistance and the growing recognition of certain adverse effects associated with guideline concordant treatment options has significantly complicated selection of empiric antibiotic therapy for patients with CAP. Effective alternative monotherapy options with a novel mechanism of action are urgently needed to address this growing unmet medical need.”

In February, the FDA accepted the New Drug Applications for both the oral and intravenous (IV) formulations of lefamulin for the treatment of CABP. The Prescription Drug User Fee Act goal date for the completion of the FDA’s review is August 19, 2019.

The full press release is available here.

RedHill Biopharma Submits NDA for H pylori Infection Treatment

On Tuesday, May 7, 2019, RedHill Biopharma Ltd. submitted a New Drug Application to the FDA for Talicia^® (RHB-105) for the treatment of H pylori infection.

In 2 positive US-based phase 3 studies, Talicia met the primary endpoint of superiority over historical standard-of-care eradication rate of 70%, demonstrating 89.4% efficacy in eradicating H pylori infection, and also demonstrated 84% eradication of H pylori infection with Talicia vs. 58% in the active comparator arm (p<0.0001).

“Talicia demonstrated robust results in its clinical studies for H pylori infection, a common and increasingly resistant and difficult to treat pathogen,” Ira Kalfus, MD, RedHill’s medical director, said in a press release. “Importantly, in our clinical study, no H pylori resistance to rifabutin, one of the key ingredients in Talicia, was identified pre and post treatment. We believe Talicia has the potential to become the new first-line, standard-of-care therapy for H pylori infection and would like to thank the patients, investigators and clinical support staff who were involved in the clinical studies of this important potential new therapy.”

The full press release is available here.

Leronlimab (PRO 140) Monotherapy Trial Reaches Ultimate Goal for HIV Suppression

On Monday, May 6, 2019, CytoDyn Inc. announced that its monotherapy trial with leronlimab (PRO 140) has produced promising results that support the design of a pivotal phase 3 monotherapy trial.

New data show that a 525 mg dose achieves 95% response rate after the first 10 weeks of monotherapy with leronlimab when used as a single agent for maintenance of HIV viral load suppression. More than 110 patients have reached nearly 1 year of suppressed viral load with monotherapy on an original dose of 350 mg or higher dosages.

The half-life of leronlimab is now confirmed at approximately 10 days versus the previous understanding of approximately 3 days.

“We are excited to now have this new clinical data that enables us to more precisely design the protocol for our planned pivotal monotherapy trial with leronlimab (PRO140),” Nader Pourhassan, PhD, CytoDyn president and CEO, said in a press release. “If successful, the pivotal monotherapy trial with leronlimab (PRO140) will hopefully generate the high-quality clinical data needed to underpin a successful potential label expansion, should the FDA approve our first BLA for leronlimab (PRO140) as a combination therapy.”

The FDA has granted a “Fast Track” designation to leronlimab (PRO 140) as a combination therapy with HAART for HIV-infected patients.

The full press release is available here.