Ridgeback Biotherapeutics LP said the FDA has granted orphan drug status to mAb-114, an experimental treatment for Ebola. It’s a step forward for the 4-year-old biotech focused on pediatric orphan and infectious diseases with limited or no treatment options for patients in the developing world.

Ebola fits the bill. It has no cure and is concentrated in those areas Ridgeback is most concerned with, where access to treatment is too expensive or unavailable. Others are in the race, including Gilead Sciences Inc., which is collaborating with the CDC and the U.S. Army Medical Research Institute for Infectious Diseases on GS-5734, remdesivir, a nucleotide prodrug. GS-5734 is being tested in the Democratic Republic of Congo’s current Ebola outbreak, as is mAb-114.

Miami-based Ridgeback entered a patent license agreement with the National Institute of Allergy and Infectious Diseases for intellectual property related to the monoclonal antibody in December. The company has committed “considerable capital to quickly advance mAb-114 to licensure,” CEO and co-founder Wendy Holman told BioWorld, though she added the company has not disclosed how much it has raised.

“We have completed the manufacturing technology transfer process and have begun manufacturing mAb-114 at a commercial manufacturing facility,” Holman said. “We hope to soon assist our partners in supplying mAb-114 to patients in need.”

Ridgeback is privately held and interested in partnering and sponsoring research with like-minded academic centers and inventors. Holman includes Peter Patriarca and Tom Monath on her team of experts at Ridgeback. Patriarca is a scientific advisory board member at VBI Inc. and is a principal of Immuno-Vax LLC. He is also a former medical officer in the CDC and the FDA. At the CDC he steered the agency’s global poliomyelitis eradication efforts and was a director in the Center for Biologics Evaluation and Research at the FDA. Monath is formerly chief medical officer at Hookipa Biotech AG and chief technical officer of Paxvax Inc., where he specialized in developing new vaccines. He is managing partner and chief science officer at Crozet Biopharma.

Holman recently joined the Presidential Advisory Council on HIV/AIDS and is a member of the board of trustees at the Sabin Vaccine Institute in Washington. She said she has about 20 years of focusing on “supporting and investing in life-saving and life-changing technologies.” She frames Ridgeback as “a woman-owned small business.”

Her husband, Wayne Holman, founded Ridgeback Capital Management, a separate entity from Ridgeback Biotherapeutics, in 2006. In November, Ridgeback Capital invested in Harpoon Therapeutics Inc.’s series C financing of $70 million. In March 2018, Ridgeback participated in funding Immutep Ltd., of Sydney, which raised about A$6.9 million (US$5.4 million). Before founding Ridgeback Capital, Wayne Holman was a pharmaceuticals equity research analyst for Merrill Lynch.

Ridgeback Biotherapeutics’ has no timeline to share for developing mAb-114, but early stage phase I data found the antibody safe, well-tolerated and easy to administer. The study began in May 2018 and the drug is currently being used to treat Ebola patients in the Democratic Republic of Congo.

In the study, 18 healthy adults received the drug, which binds to the core receptor binding domain of the Zaire ebolavirus surface protein to prevent the virus from infecting human cells, at the NIH’s clinical testing center in Bethesda, Md. Participants in the phase I trial received a single intravenous infusion of mAb-114, administered over approximately 30 minutes. Three participants received a 5-mg/kg dose; five participants received a 25-mg/kg dose; and 10 participants received a 50-mg/kg dose. All infusions were well-tolerated. Four participants reported mild side effects, such as discomfort, muscle or joint pain, headache, nausea, and chills in the three days following the infusion.

As investigators expected, levels of mAb-114 in the blood increased as the dosage was increased. Investigators also observed relatively uniform levels of absorption, distribution and elimination of mAb-114 among participants.

The drug is formulated as a freeze-dried powder and does not require freezer storage, according to the NIH. The powder is reconstituted using sterile water and added to saline for administration.

There is also an ongoing phase II/III trial for mAb-114 being held in the Democratic Republic of Congo.

Scientists at the NIH’s Vaccine Research Center developed mAb-114 along with scientists at the National Institute of Biomedical Research, the Institute for Research in Biomedicine and Vir Biotechnology Inc.’s subsidiary Humabs Biomed, both based in Bellinzona, Switzerland; and the U.S. Army Medical Research Institute of Infectious Diseases at Fort Detrick, Md. The Defense Advanced Research Projects Agency funded the production of mAb-114 for clinical testing.