Drug can prevent CMV in HSCT recipients

A new drug can prevent cytomegalovirus (CMV) in patients undergoing hematopoietic stem cell transplant (HSCT), according to a study published in The New England Journal of Medicine.

The drug, called CMX001, is an oral nucleotide analog lipid-conjugate that blocks replication of double-stranded DNA viruses.

HSCT recipients who took CMX001 after engraftment were less likely to develop CMV than HSCT patients who received placebo, researchers found.

“With current agents, between 3% and 5% of allogeneic transplant patients develop CMV disease within 6 months of transplantation, and a small number of them may die of it,” said investigator Francisco Marty, MD, of the Dana-Farber Cancer Institute in Boston.

“There clearly is a need for better treatments with fewer adverse effects. This clinical trial examined whether the disease can be prevented, rather than waiting for blood tests to show that treatment is needed.”

The phase 2 trial involved 230 HSCT recipients treated at 27 centers across the US. The patients were randomized to receive placebo or CMX001 at doses ranging from 40 mg a week to 200 mg twice a week.

Treatment began after engraftment, at about 2 to 3 weeks post-transplant, and continued for 9 to 11 weeks.

The study’s primary efficacy outcome was a “CMV event,” which was defined as CMV that affects the lung, digestive tract, or other organs, or a detectable amount of CMV in the blood at the end of treatment.

CMV events occurred in 25% (43/171) of patients who received CMX001 and 37% (22/59) of patients who received placebo. However, when CMX001 was given at the optimal dose—100 mg twice a week—only 10% of patients had a CMV event.

“The results show the effectiveness of CMX001 in preventing CMV infections in this group of patients,” Dr Marty said. “Because CMX001 is known to be active against other herpes viruses and against adenoviruses that sometimes affect transplant patients, it may be useful as a preventive or treatment agent for those infections as well.”

Most of the side effects associated with CMX001 were gastrointestinal in nature. Diarrhea was common and often serious in patients who received the drug at 200 mg twice a week.

Patients in this dose group were more likely to experience elevated alanine aminotransferase levels as well. But this was not associated with increases in levels of bilirubin or aspartate aminotransferase.

This research was funded by Chimerix, the company developing CMX001.

A new drug can prevent cytomegalovirus (CMV) in patients undergoing hematopoietic stem cell transplant (HSCT), according to a study published in The New England Journal of Medicine.

The drug, called CMX001, is an oral nucleotide analog lipid-conjugate that blocks replication of double-stranded DNA viruses.

HSCT recipients who took CMX001 after engraftment were less likely to develop CMV than HSCT patients who received placebo, researchers found.

“With current agents, between 3% and 5% of allogeneic transplant patients develop CMV disease within 6 months of transplantation, and a small number of them may die of it,” said investigator Francisco Marty, MD, of the Dana-Farber Cancer Institute in Boston.

“There clearly is a need for better treatments with fewer adverse effects. This clinical trial examined whether the disease can be prevented, rather than waiting for blood tests to show that treatment is needed.”

The phase 2 trial involved 230 HSCT recipients treated at 27 centers across the US. The patients were randomized to receive placebo or CMX001 at doses ranging from 40 mg a week to 200 mg twice a week.

Treatment began after engraftment, at about 2 to 3 weeks post-transplant, and continued for 9 to 11 weeks.

The study’s primary efficacy outcome was a “CMV event,” which was defined as CMV that affects the lung, digestive tract, or other organs, or a detectable amount of CMV in the blood at the end of treatment.

CMV events occurred in 25% (43/171) of patients who received CMX001 and 37% (22/59) of patients who received placebo. However, when CMX001 was given at the optimal dose—100 mg twice a week—only 10% of patients had a CMV event.

“The results show the effectiveness of CMX001 in preventing CMV infections in this group of patients,” Dr Marty said. “Because CMX001 is known to be active against other herpes viruses and against adenoviruses that sometimes affect transplant patients, it may be useful as a preventive or treatment agent for those infections as well.”

Most of the side effects associated with CMX001 were gastrointestinal in nature. Diarrhea was common and often serious in patients who received the drug at 200 mg twice a week.

Patients in this dose group were more likely to experience elevated alanine aminotransferase levels as well. But this was not associated with increases in levels of bilirubin or aspartate aminotransferase.

This research was funded by Chimerix, the company developing CMX001.

A new drug can prevent cytomegalovirus (CMV) in patients undergoing hematopoietic stem cell transplant (HSCT), according to a study published in The New England Journal of Medicine.

The drug, called CMX001, is an oral nucleotide analog lipid-conjugate that blocks replication of double-stranded DNA viruses.

HSCT recipients who took CMX001 after engraftment were less likely to develop CMV than HSCT patients who received placebo, researchers found.

“With current agents, between 3% and 5% of allogeneic transplant patients develop CMV disease within 6 months of transplantation, and a small number of them may die of it,” said investigator Francisco Marty, MD, of the Dana-Farber Cancer Institute in Boston.

“There clearly is a need for better treatments with fewer adverse effects. This clinical trial examined whether the disease can be prevented, rather than waiting for blood tests to show that treatment is needed.”

The phase 2 trial involved 230 HSCT recipients treated at 27 centers across the US. The patients were randomized to receive placebo or CMX001 at doses ranging from 40 mg a week to 200 mg twice a week.

Treatment began after engraftment, at about 2 to 3 weeks post-transplant, and continued for 9 to 11 weeks.

The study’s primary efficacy outcome was a “CMV event,” which was defined as CMV that affects the lung, digestive tract, or other organs, or a detectable amount of CMV in the blood at the end of treatment.

CMV events occurred in 25% (43/171) of patients who received CMX001 and 37% (22/59) of patients who received placebo. However, when CMX001 was given at the optimal dose—100 mg twice a week—only 10% of patients had a CMV event.

“The results show the effectiveness of CMX001 in preventing CMV infections in this group of patients,” Dr Marty said. “Because CMX001 is known to be active against other herpes viruses and against adenoviruses that sometimes affect transplant patients, it may be useful as a preventive or treatment agent for those infections as well.”

Most of the side effects associated with CMX001 were gastrointestinal in nature. Diarrhea was common and often serious in patients who received the drug at 200 mg twice a week.

Patients in this dose group were more likely to experience elevated alanine aminotransferase levels as well. But this was not associated with increases in levels of bilirubin or aspartate aminotransferase.

This research was funded by Chimerix, the company developing CMX001.