LogicBio Therapeutics, Inc. (NASDAQ:LOGC), a clinical-stage genetic medicine company, today provided updates around its SUNRISE Phase 1/2 clinical trial and development pipeline, and announced the appointment of Susan R. Kahn to its Board of Directors.
SUNRISE Phase 1/2 Clinical Trial Update
SUNRISE is a first-in-human, open-label, multi-center, Phase 1/2 clinical trial designed to assess the safety and tolerability of an intravenous infusion of LB-001, the company’s investigational, single-administration genome editing therapy, in pediatric patients with methylmalonic acidemia (MMA). SUNRISE is designed to enroll up to eight patients across two age groups (six months to two years old, and three to twelve years old) and evaluate LB-001 at two dose levels (5 x 1013 vg/kg and 1 x 1014 vg/kg). In October, LogicBio announced early data from the SUNRISE trial demonstrating the first-ever in vivo genome editing in children. These early data showed measurable levels of albumin-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression. In November, the company disclosed that the third patient dosed in SUNRISE, who received 5 x 1013 vg/kg of LB-001 and was in the lower age group, experienced a serious adverse event (SAE). This SAE has been completely resolved and all laboratory parameters that were abnormal at the time the SAE was observed have returned to normal. The SUNRISE trial is continuing in accordance with the protocol and additional safety measures implemented following the occurrence of the SAE. The company plans to report additional interim data from the SUNRISE trial in the second quarter of 2022.
“We believe reporting interim data from SUNRISE in the second quarter of 2022 instead of by year-end 2021 will allow us to amass a more robust and meaningful data set,” said Fred Chereau, president and chief executive officer of LogicBio. “Safety is our top priority, and we are pleased that the patient who experienced the SAE is now fully recovered. We expect to dose two additional patients in the near term, one patient in the older age group at the higher dose, and another patient in the lower age group at the lower dose.”
Nomination of New GeneRide Development Candidate for the Treatment of Hereditary Tyrosinemia Type 1
In accordance with its previously announced guidance, LogicBio also announced the nomination of a new development candidate, LB-401, for the treatment of hereditary tyrosinemia type 1 (HT1). This development candidate is based on the company’s GeneRide genome editing platform. HT1 is a rare, genetic disorder characterized by elevated blood levels of the amino acid tyrosine. This condition is caused by a shortage of the enzyme fumarylacetoacetate hydrolase (FAH), one of the enzymes required for the multi-step process that breaks down tyrosine.
In preclinical studies presented at the 2021 ESGCT Annual Meeting (ESGCT) in October, HT1 models with acute liver damage showed that GeneRide-edited hepatocytes substantially repopulated the entire liver within four weeks post-administration, replacing the diseased hepatocytes with corrected hepatocytes. HT1 mice that received the GeneRide-FAH vector were no longer reliant on the current standard of care for the disease, and demonstrated restored normal body growth, liver function, and undetectable succinyl acetone levels.
“We are excited to explore the potential of our GeneRide platform in HT1, a devastating rare disease that can present within the first months of patients’ lives,” said Mariana Nacht, Ph.D., chief scientific officer of LogicBio. “The preclinical data presented at ESGCT for this indication validate our confidence in leveraging the selective advantage observed in our GeneRide platform to deliver corrected hepatocytes that drive improved disease markers.”
Susan R. Kahn Appointed to Board of Directors
The company also announced today the appointment of Susan R. Kahn to its Board of Directors. From September 2007 to November 2021, Ms. Kahn was the executive director of the National Tay-Sachs & Allied Diseases Association (NTSAD), a highly regarded patient advocacy group for children and adults affected by rare genetic diseases. Previously, she was at Genzyme Genetics, where she led initiatives to develop and execute new business opportunities, acquisition and partnering strategies, new product and technology assessments, and technology licensing. Before that, Ms. Kahn worked at Chiron Diagnostics in roles of increasing responsibility, including roles in business development and finance. She earned an A.B. in applied mathematics-economics from Brown University and an M.B.A. from the Tuck School of Business at Dartmouth.
“Sue is a leading advocate for those impacted by rare genetic diseases. Her passion for these patients and their families aligns perfectly with our mission at LogicBio,” said Mr. Chereau. “I am thrilled to welcome Sue to our Board and am excited to leverage her expertise and thought leadership in the advocacy space as we continue to advance our mission to deliver the hope of genetic medicine to people impacted by devastating, early onset diseases.”
“I am excited to join the LogicBio Board at what I believe is a pivotal time for the company,” said Ms. Kahn. “Having spent the last fourteen years supporting patients and families affected by rare genetic diseases, I look forward to helping LogicBio deliver much-needed treatment options to patients.”
About LogicBio Therapeutics
LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The company’s genome editing platform, GeneRide™, is a new approach to precise gene insertion harnessing a cell’s natural DNA repair process potentially leading to durable therapeutic protein expression levels. The company’s gene delivery platform, sAAVy™, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit www.logicbio.com, which does not form a part of this release.
