Gamida Cell Ltd., a maker of cell and immune therapy technologies, on Wednesday published the results of a key late-stage clinical trial of its flagship drug, which aims to increase the success of bone marrow transplants in blood cancer patients triggering a surge in its Nasdaq-traded shares.

Gamidas shares jumped almost 26% on the US exchange Wednesday. The shares have advanced 163% in the past 12 months, leading to a company valuation of $733 million. The firm held its initial public offering of shares on the Nasdaq in 2019 to help finance the final stage trial of the drug.

The phase three study tested the safety and efficacy of its omidubicel drug, previously called NiCord, in patients with blood-based cancers.

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The data showed that omidubicel resulted in faster blood count recovery, fewer bacterial and viral infections, and fewer days in the hospital.

Stem cells in bag in Gamida Cells Jerusalem lab, July 2017 (Shoshanna Solomon/TimesofIsrael)

All of these are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant, said Mitchell Horwitz, principal investigator and professor of medicine at the Duke Cancer Institute, in a statement released by the Jerusalem-based firm.

The data was from the international, multi-center, randomized Phase 3 study of the drug, designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared with a group of patients who received a standard umbilical cord blood transplant.

In May, the firm said that the study yielded positive results and met a major target showing that omidubicel led to a statistically significant reduction in the time needed to for patients to begin making healthy new cells after receiving stem cells, which is a key milestone in a patients recovery from a bone marrow transplant.

Omidubicel, which would be the first drug developed by Gamida to hit the market, 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.

Julian Adams, chief executive officer of Gamida Cell (Courtesy)

Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.

Today some high-risk blood cancers cannot be cured unless the patient undergoes a bone marrow graft. For that purpose, a perfect 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.

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. Its great advantage is that because it is so young, there is no need for a full tissue matching and a partial match is enough. Most patients generally find at least one unit of cord blood that partially matches them.

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.

Gamida overcomes this limitation by expanding the number of stem cells within one unit of umbilical cord blood and enhancing their performance.

The firm said Wednesday that it expects to submit a Biologics License Application to the US Food and Drug Administration for omidubicel in the second half of this year.

We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor, said Julian Adams, chief executive officer of Gamida Cell, which was founded in 1998.

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Hope grows for blood cancer patients, as cell therapy firm's key trial succeeds - The Times of Israel

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