LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today reported financial results for the second quarter ended June 30, 2021 and highlighted recent corporate milestones.
| LEXINGTON, Mass., Aug. 9, 2021 /PRNewswire/ -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today reported financial results for the second quarter ended June 30, 2021 and highlighted recent corporate milestones. “LogicBio made significant progress in the first half of the year. Most importantly, we dosed the first patient in our groundbreaking Phase 1/2 SUNRISE trial, which we believe represented the first in vivo gene editing therapy delivered systemically to a pediatric patient,” said Frederic Chereau, president and chief executive officer of LogicBio Therapeutics. “We also entered into collaborations with Daiichi Sankyo and CANbridge Pharmaceuticals, which represented a validation of the potential of our GeneRide™ and sAAVy™ platforms. The second quarter also saw us strengthen our leadership team with key executive appointments. I am confident this progress positions us well to expand our platforms and pipeline. We continue to look forward to providing an update on SUNRISE enrollment, highlighting dose escalation and age de-escalation, expected later in the year, as well as interim data from the SUNRISE trial, expected by year-end.” Anticipated Milestones for 2021: Ph 1/2 SUNRISE Trial for LB-001 in Pediatric Patients with MMA
Pipeline
Recent Business Highlights: First patient dosed in Phase 1/2 SUNRISE clinical trial for LB-001 for the treatment of MMA in pediatric patients
Entered into collaborations with Daiichi Sankyo and CANbridge Pharmaceuticals
Preclinical data presented at ASGCT
Strengthened leadership team with key appointments
Second Quarter 2021 Financial Results:
About the SUNRISE Trial The SUNRISE trial is an open-label, multi-center, Phase 1/2 clinical trial designed to assess the safety and tolerability of a single intravenous infusion of LB-001 in pediatric patients with methylmalonic acidemia (MMA) characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. On June 2, 2021, the Company announced that the first patient was dosed. Seven leading centers in the United States and one in Saudi Arabia are expected to participate in the trial. With the aim of evaluating LB-001 at an early age, before irreversible damage has occurred, the SUNRISE trial is initially enrolling 3-12 year old patients with the potential to include infants as young as 6 months old after meeting certain safety parameters and biomarker detection indicating genome integration and protein expression. The SUNRISE trial will enroll up to eight patients and evaluate a single administration of LB-001 at two dose levels. About LB-001 LB-001 is an investigational, first-in-class, single-administration, gene editing therapy for early intervention in methylmalonic acidemia (MMA) using the GeneRide™ platform. GeneRide technology utilizes a natural DNA repair process called homologous recombination that enables precise editing of the genome without the need for exogenous nucleases and exogenous promoters that are associated with an increased risk of immune response and cancer. LB-001 is designed to non-disruptively insert a corrective copy of the methylmalonyl-CoA mutase (MMUT) gene into the albumin locus to drive lifelong therapeutic levels of MMUT expression in the liver, the main site of MMUT expression and activity. LB-001 is delivered to hepatocytes via liver-targeted, engineered recombinant adeno-associated virus vector (rAAV-LK03). Preclinical studies found that LB-001 was safe and demonstrated transduction of hepatocytes, site-specific genomic integration, and transgene expression. LB-001–corrected hepatocytes in a mouse model of MMA demonstrated preferential survival and expansion (selective advantage), thus contributing to a progressive increase in hepatic MMUT expression over time. LB-001 resulted in improved growth, metabolic stability, and survival in MMA mice. The U.S. Food and Drug Administration (FDA) granted fast track designation, rare pediatric disease designation and orphan drug designation for LB-001 for the treatment of MMA. In addition, the European Medicines Agency (EMA) granted orphan drug designation for LB-001 for the treatment of MMA. About Methylmalonic Acidemia (MMA) Methylmalonic acidemia (MMA) is a rare and life-threatening genetic disorder affecting approximately 1 in 50,000 newborns in the United States. In the most common form of MMA, a mutation in a gene called methylmalonyl-CoA mutase (MMUT) prevents the body from properly processing certain fats and proteins. As a result, toxic metabolites accumulate in the liver, in muscle tissue and in the brain. Symptoms include vomiting, lethargy, seizures, developmental delays and organ damage. There is no approved medical therapy addressing the underlying cause of the disease. To manage the symptoms, patients go on a severely restrictive, low-protein, high-calorie diet, often through a feeding tube. Even with aggressive management, these patients often experience life-threatening metabolic crises that can cause permanent neurocognitive damage. Because of the need for early intervention, newborns are screened for MMA in every state in the United States. About LogicBio Therapeutics LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The Company’s gene 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. Forward-Looking Statements Statements in this press release regarding LogicBio’s strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited to statements regarding dosing representing the first in vivo gene editing therapy delivered systemically to a pediatric patient; the expected timing of providing an update on enrollment, dose escalation and age de-escalation in the SUNRISE trial; the expected timing of announcing interim clinical data in the SUNRISE trial; being well-positioned to continue to expand our platforms and pipeline; our anticipated milestones and the timing thereof; enrolling patients as young as 6 months old in the SUNRISE trial; the potential benefits of LB-001; and the sites expected to participate in the SUNRISE trial. The terms “anticipate,” “believe,” “confident,” “expect,” “look forward,” “on track,” “positions us,” “potential,” “will” and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the potential direct or indirect impact of the COVID-19 pandemic on our business, operations, and the markets and communities in which we and our partners, collaborators and vendors operate; the risk that existing preclinical data may not be predictive of the results of ongoing or later clinical trials; the risks that clinical trials may not be successful or may be discontinued or delayed for any reason; manufacturing and process development risks, including delays relating to continuously improving our manufacturing processes; risks associated with management and key personnel changes and transitional periods; the actual funding required to develop and commercialize product candidates, including for safety, tolerability, enrollment, manufacturing or economic reasons; the timing and content of decisions made by regulatory authorities; the actual time it takes to initiate and complete preclinical and clinical studies; the competitive landscape; changes in the economic and financial conditions of LogicBio; and LogicBio’s ability to obtain, maintain and enforce patent and other intellectual property protection for LB-001 and any other product candidates. Other risks and uncertainties include those identified under the heading “Risk Factors” in LogicBio’s Annual Report on Form 10-K for the year ended December 31, 2020 and other filings that LogicBio may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and LogicBio does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Investor Contact: Laurence Watts Media Contacts: Adam Daley Jenna Urban
SOURCE LogicBio Therapeutics, Inc. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Company Codes: NASDAQ-NMS:LOGC | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
View original content:
