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Dimension Therapeutics, Inc. has announced its decision to stop development of DTX101, a gene therapy product intended to treat moderate/severe to severe hemophilia B.
The decision was based on “disappointing” results from the phase 1/2 study of DTX101, according to Annalisa Jenkins, MBBS, chief executive officer of Dimension.
“[O]ur phase 1/2, open-label clinical study did not demonstrate an ability to achieve a minimum target product profile for continued development or future commercialization,” Dr Jenkins said.
Dimension previously reported results from this 6-patient study in February.
At that time, the data showed that DTX101 could increase factor IX (FIX) expression in adults with hemophilia B, allowing 3 of them to forgo prophylactic and on-demand treatment.
However, 5 of the 6 patients in the trial had alanine aminotransferase elevations, and 1 patient had a grade 4 adverse event as a result.
Dimension said it plans to present full results from this study, including results from ongoing immune and biomarker analyses, at a future scientific conference.
“[D]imension remains committed to the hemophilia community through continued investment in the company’s ongoing [investigational new drug]-enabling activities for DTX201 for hemophilia A, in collaboration with Bayer, and the follow-up of the 6 patients dosed with DTX101 in the phase 1/2 clinical trial through an extension study that will monitor all patients for a total of 5 years,” Dr Jenkins noted.
DTX101 was designed to deliver stable expression of FIX in patients with hemophilia B. It is a non-replicating, recombinant adeno-associated viral vector, AAVrh10, with a codon-optimized FIX gene expressing wild-type FIX protein. 
Dimension Therapeutics, Inc. has announced its decision to stop development of DTX101, a gene therapy product intended to treat moderate/severe to severe hemophilia B.
The decision was based on “disappointing” results from the phase 1/2 study of DTX101, according to Annalisa Jenkins, MBBS, chief executive officer of Dimension.
“[O]ur phase 1/2, open-label clinical study did not demonstrate an ability to achieve a minimum target product profile for continued development or future commercialization,” Dr Jenkins said.
Dimension previously reported results from this 6-patient study in February.
At that time, the data showed that DTX101 could increase factor IX (FIX) expression in adults with hemophilia B, allowing 3 of them to forgo prophylactic and on-demand treatment.
However, 5 of the 6 patients in the trial had alanine aminotransferase elevations, and 1 patient had a grade 4 adverse event as a result.
Dimension said it plans to present full results from this study, including results from ongoing immune and biomarker analyses, at a future scientific conference.
“[D]imension remains committed to the hemophilia community through continued investment in the company’s ongoing [investigational new drug]-enabling activities for DTX201 for hemophilia A, in collaboration with Bayer, and the follow-up of the 6 patients dosed with DTX101 in the phase 1/2 clinical trial through an extension study that will monitor all patients for a total of 5 years,” Dr Jenkins noted.
DTX101 was designed to deliver stable expression of FIX in patients with hemophilia B. It is a non-replicating, recombinant adeno-associated viral vector, AAVrh10, with a codon-optimized FIX gene expressing wild-type FIX protein.
Dimension Therapeutics, Inc. has announced its decision to stop development of DTX101, a gene therapy product intended to treat moderate/severe to severe hemophilia B.
The decision was based on “disappointing” results from the phase 1/2 study of DTX101, according to Annalisa Jenkins, MBBS, chief executive officer of Dimension.
“[O]ur phase 1/2, open-label clinical study did not demonstrate an ability to achieve a minimum target product profile for continued development or future commercialization,” Dr Jenkins said.
Dimension previously reported results from this 6-patient study in February.
At that time, the data showed that DTX101 could increase factor IX (FIX) expression in adults with hemophilia B, allowing 3 of them to forgo prophylactic and on-demand treatment.
However, 5 of the 6 patients in the trial had alanine aminotransferase elevations, and 1 patient had a grade 4 adverse event as a result.
Dimension said it plans to present full results from this study, including results from ongoing immune and biomarker analyses, at a future scientific conference.
“[D]imension remains committed to the hemophilia community through continued investment in the company’s ongoing [investigational new drug]-enabling activities for DTX201 for hemophilia A, in collaboration with Bayer, and the follow-up of the 6 patients dosed with DTX101 in the phase 1/2 clinical trial through an extension study that will monitor all patients for a total of 5 years,” Dr Jenkins noted.
DTX101 was designed to deliver stable expression of FIX in patients with hemophilia B. It is a non-replicating, recombinant adeno-associated viral vector, AAVrh10, with a codon-optimized FIX gene expressing wild-type FIX protein.