The excitement at Jerusalem-based Gamida Cell, a maker of cell and immune therapy technologies, is palpable.

The biotechnology company has started enrolling patients for a last-stage clinical trial for a drug it believes will help increase the success of bone marrow transplants in blood cancer patients, and help them better withstand the ordeal of the lifesaving procedure.

The patients are being enrolled in the US, Spain, The Netherlands and Singapore. Should the results of the trial, as hoped, be positive, that would lead to the launch of a commercially available product in 2020, Gamida Cells CEO Yael Margolin said in an interview with The Times of Israel.

We are at an exciting transition point, and moving from being a research and development firm, based in Israel, to an international commercial firm, said Margolin who has headed the company for the past 12 years in her sun-drenched office at the biotech firms headquarters in Jerusalem. We need to prepare to commercialize the product. We are now looking at various sites in Israel for a new manufacturing facility and looking to employ some 100 people. These workers will be added to the 40 already employed in Jerusalem.

Gamida Cells CEO, Dr. Yael Margolin (Courtesy)

Preliminary clinical data has already revealed that the risk of their leading product for blood cancers, NiCord, not meeting its targets in the Phase 3 trial, is low, added Margolin.

The drug has already received a breakthrough therapy designation by the US Food and Drug Administration (FDA). The designation is given to a drug that is meant to treat a serious or life-threatening condition, and where preliminary clinical evidence indicates that it may demonstrate a substantial improvement on at least one clinically significant target (endpoint) over other available therapies. The designation also entitles the company to get more and closer FDA guidance to help bring the treatment faster to patients.

The combination of the low clinical risk based on the previous trial results and the lower regulatory risk, because the drug is being developed in close collaboration with the FDA, has spurred the company into a flurry of activity that is aimed at scaling up its production facilities to get ready for the day NiCord hits the markets.

The company said last month it raised $40 million from investors including Novartis, which is already a major shareholder in the firm. The funds will support the ongoing Phase 3 stage for NiCord. The company also announced, on July 20, that it received a $3.5-million grant from the Israeli government that will support the further development of NiCord and other drugs that the company is developing, including therapies for sickle cell disease and for blood and solid cancers. Gamida has also appointed a new chief medical officer, Ronit Simantov, who will be based in the US.

The first market for our drug will be the US, Margolin said.

The Gamida Cell lab in Jerusalem where umbilical cord blood is stored in tanks, July 16, 2017. (Shoshanna Solomon/Times of Israel)

NiCord, which would be the first drug developed by Gamida to hit the market if the trial goes well is believed to increase the chances of a successful bone marrow transplantation process for patients who do not have a rapidly available, fully matched, bone marrow donor.

Today some high-risk blood cancers cannot be cured unless patients undergo a bone marrow graft. For that purpose, a perfect 100-percent match needs to be found, a process that in the US takes an average of three to four months, if the patient is lucky. Sometimes, no match is found.

There are 70,000 patients a year globally with blood cancers who need a bone marrow transplant, Margolin said. It is a rare condition. But for that transplant, you need a donor with full tissue matching. As many as 50% dont get to the transplant phase, because they havent found a matching donor in time.

Umbilical cord blood collected from newborn babies contains stem cells, which can be used to treat diseases. Today cord-blood banks around the world store the cord blood. It great advantage is that because it is so young, there is no need for a full tissue matching.

The big advantage with umbilical blood is that you dont need full tissue matching; a partial match is enough, Margolin continued. Most patients generally find at least one unit of cord blood that partially matches them.

Stem cells in a bag in Gamida Cells Jerusalem lab, July 16, 2017 (Shoshanna Solomon/Times of Israel)

The problem is that the quantity of cells in each unit is not huge, and it is the number of stem cells in the cord blood that is critical to the success of transplantation.

Our idea is to leverage the advantages of the cord blood and overcome the limitations of the cell number by applying our own platform technology, called NAM Technology, added Margolin. This technology allows us to take one unit of umbilical cord blood and expand the number of stem cells within it and enhance their performance.

Gamida Cell selects the stem cells from the unit and puts them in a culture together with a molecule called Nicotinamide (NAM) a form of Vitamin B3 and adds other ingredients. This culture, to which the firm holds intellectual property rights, increases the number of stem cells, and enhances their functionality, Margolin said.

The cells are then harvested from the culture, frozen in a small blood-bag in a final formulation that is ready for infusion, and then shipped to hospitals via couriers. Doctors thaw the product by the bedside of the patients and infuse the fluid into them.

From start to finish, our process takes three weeks, Margolin said. The average search for a bone marrow match takes three to four months.

The clinical trial underway is enrolling patients aged 16 years and older.

An earlier trial of the drug showed that patients transplanted with NiCord showed a more rapid engraftment the amount of time needed for the development of a minimal amount of white blood cells, or neutrophils, in the blood. That minimum amount indicates the patient is now less vulnerable to infections and bleeding following the transplant, and is an indication of success.

In the pilot phase clinical trials, the median time to neutrophil engraftment with NiCord was 11 days, compared to three to four weeks in patients who received standard umbilical cord blood. The results in a study conducted at Duke University also showed a lower rate of infection 22% vs 54%; and a lower duration of hospitalization compared to standard umbilical cord engraftment, Margolin said.

Now the company is enrolling patients for its larger, Phase 3 multi-national, randomized controlled registration study. And in February it said it had already transplanted its first patient, as part of the trial.

We hope to publish positive topline data from the Phase3 study in the first half of 2019 and launch the product on the market in 2020, she said.

Metal barrel with a frozen bag of umbilical cord stem cells ready for delivery from Gamida Cells Jerusalem lab, July 16, 2017. (Shoshanna Solomon/Times of Israel)

A metal barrel within which was a frozen bag of umbilical cord stem cells was waiting to be picked by a courier in the lobby of the Gamida Cell offices, ready to be thawed and injected into a patient somewhere around the world.

We have a sophisticated infrastructure that coordinates everything between the cord bank blood and our manufacturing site and the hospital where the patient is to be treated, Margolin continued. This infrastructure is 100% robust, but we plan to scale this up toward commercialization.

The $40 million in funds the company raised last month is expected to last until late 2019. After that, she added, all options are on the table: an IPO, or teaming up with a strategic partner, are both possibilities for the future.

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Cell therapy firm in flurry of activity as hope nears for bone marrow patients - The Times of Israel