Sangamo Therapeutics and Pfizer Announce Collaboration for Hemophilia A Gene Therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. announced this week, an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter.

“Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A,” said Mikael Dolsten, MD, PhD, President of Worldwide Research and Development at Pfizer. “Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing. We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.”

“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Dr. Sandy Macrae, Sangamo’s Chief Executive Officer. “We believe Pfizer’s end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases.”

Under the terms of the collaboration agreement, Sangamo will receive a $70 million upfront payment from Pfizer. Sangamo will be responsible for conducting the SB-525 Phase 1/2 clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products, if any. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.

Gene therapy is a potentially transformational technology for patients, focused on highly specialized, one-time, treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient’s cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.

Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A. SB-525 is comprised of a rAAV vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has cleared initiation of human clinical trials for SB-525, which also has been granted orphan drug designation. Sangamo is on track this quarter to start a Phase 1/2 clinical trial to evaluate safety and to measure blood levels of Factor VIII protein and other efficacy endpoints.

 

Liver Disease Risk Increased by Type 2 Diabetes, Study Finds

People with type 2 diabetes are at greater risk of serious liver disease than those without the condition, research has shown.

Researchers warn that hospital admissions and deaths caused by liver disease are likely to rise if cases of type 2 diabetes continue to increase at current rates.

The team examined cases of liver diseases among people with diabetes from anonymised, securely linked hospital records and death records in Scotland over a ten year period.

They found that most cases of liver disease in people with type 2 diabetes are not alcohol-related but caused by a build-up of fat within liver cells – a condition known as non-alcoholic fatty liver disease or NAFLD.

NAFLD is commonly linked to obesity, which is also a risk factor for type 2 diabetes. Most people can avoid getting these conditions by following a healthy diet and taking regular exercise.

The research team – led by the Universities of Edinburgh and Southampton – found that men with type 2 diabetes are three times more likely to suffer from NAFLD than men without diabetes.

There are fewer cases of type 2 diabetes and liver disease amongst women but having type 2 diabetes increases the risk of NAFLD by five times, the study found.

People with NAFLD are more susceptible to the effects of alcohol on the liver and should avoid drinking to avoid further complications, the researchers say.

Treatment options for NAFLD – which increases the risk of life-threatening complications such as cirrhosis and liver cancer – are limited.

The study involved researchers from the Scottish and Southampton Diabetes and Liver Disease Group. It is published in the Journal of Hepatology and was funded by the Scottish Government through the Scottish Diabetes Group.

Professor Sarah Wild, of the University of Edinburgh’s Usher Institute for Population Health Sciences, said: “Preventing non-alcoholic fatty liver disease by avoiding unhealthy lifestyles in both people with and without diabetes is important because it is difficult to treat the complications of this condition.”

Professor Chris Byrne of the University of Southampton and University Hospital Southampton’s, NIHR Biomedical Research Centre said: “We have shown for the first time that type 2 diabetes is an important novel risk factor that increases numbers of hospital admissions and deaths, in people with all common chronic liver diseases. Further research is now needed to determine whether all patients with type 2 diabetes should be screened for common chronic liver diseases.”