LogicBio Therapeutics Reports Third Quarter 2022 Financial Results and Provides Business Updates

Third Quarter 2022 Financial Results:

Three Months Ended September 30, 2022, and 2021

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's proprietary manufacturing process, mAAVRx™, aims to overcome one of the current limitations of AAV manufacturing by improving yields and product quality. The company is based in Lexington, MA. For more information, visit www.logicbio.com, which does not form a part of this release.

About LB-001

LB-001 is an investigational, first-in-class, single-administration, genome editing therapy for early intervention in methylmalonic acidemia (MMA) using LogicBio®'s proprietary GeneRide® drug development platform. GeneRide technology utilizes a natural DNA repair process called homologous recombination designed to enable precise editing of the genome without the need for exogenous nucleases and promoters that have been 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 intravenously 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.

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 the potential of the GeneRide® platform and the closing of the proposed merger with Alexion. The terms "believe," "look forward," "future," "intend," "designed," "potential," "suggests," "plans," "expects" 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 risk that existing preclinical and/or clinical data may not be predictive of the results of ongoing or later preclinical and/or clinical results; the risk that we may not be successful in efforts to leverage our technologies for business development or otherwise; 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, including the actual time it takes to demonstrate clinical efficacy; the competitive landscape; changes in the economic and financial conditions of LogicBio; and our ability to consummate the proposed merger with Alexion, including our ability to satisfy the conditions to the proposed merger. 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, 2021 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.

LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Operations

(In thousands, except share and per share data)

(Unaudited)

Three Months Ended September 30,

Nine Months Ended September 30,

2022

2021

2022

2021

REVENUE

Collaboration and service revenue

$ 2,717

$ 2,120

$ 8,732

$ 3,383

Total revenue

2,717

2,120

8,732

3,383

OPERATING EXPENSES

Research and development

5,051

7,806

15,524

21,482

General and administrative

3,410

4,257

10,293

12,081

Total operating expenses

8,461

12,063

25,817

33,563

LOSS FROM OPERATIONS

(5,744)

(9,943)

(17,085)

(30,180)

OTHER INCOME (EXPENSE):

Interest income

147

3

197

13

Interest expense

(176)

(270)

(591)

(824)

Other (expense) income, net

(2)

(3)

(2)

(3)

Total other expense, net

(31)

(270)

(396)

(814)

Loss before income taxes

(5,775)

(10,213)

(17,481)

(30,994)

Income tax benefit

—

28

—

28

Net loss

$ (5,775)

$ (10,185)

$ (17,481)

$ (30,966)

Net loss per share—basic and diluted

$ (0.18)

$ (0.31)

$ (0.53)

$ (0.96)

Weighted-average common stock outstanding—basic and diluted

32,962,733

32,443,960

32,962,221

32,181,912

LogicBio Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(In thousands)

As of

September 30, 2022

December 31, 2021

(Unaudited)

Cash and cash equivalents

$ 30,780

$ 53,480

Other assets

7,821

9,290

TOTAL ASSETS

$ 38,601

$ 62,770

Total liabilities

$ 22,862

$ 32,043

Stockholders' equity

15,739

30,727

TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY

$ 38,601

$ 62,770

Investor Contact:
Stephen Jasper
Gilmartin Group
(858) 525-2047
stephen@gilmartinir.com

Media Contacts:
Adam Daley
Berry & Company Public Relations
W:212-253-8881
C: 614-580-2048
adaley@berrypr.com

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SOURCE LogicBio Therapeutics, Inc.