GlobeNewswire Inc | Mar 4, 2021 07:00AM EST

March 04, 2021

-- Reported preliminary Phase 1 findings from a Single Ascending Dose (SAD) trial that suggest single subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg in Friedreich'sataxia (FA) patients -- Placebo-controlled Phase 1 trials in FA patients remain on track for topline data in Q2 2021 -- Cash and investments of $92.6 million as of December 31, 2020

BALA CYNWYD, Pa., March 04, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today reported its fourth quarter and full year 2020 operating and financial results.

2020 was a transformational year for Larimar as we entered the public market, built a strong institutional shareholder base, and reported initial clinical findings in our lead Friedreichs ataxia (FA) program, said Carole Ben-Maimon, MD, President and Chief Executive Officer ofLarimar. These accomplishments were enabled by the talent and commitment of our team, who successfully navigated the challenges of the pandemic to execute on our goals without compromising patient safety. We are thankful for their work and for the dedication of the patients who participated in our clinical trials and the Friedreichs Ataxia Research Alliance, all of whom were key components of our recent progress.

Dr. Ben-Maimon continued, Looking ahead to 2021, we believe we are poised to achieve critical milestones in the development of CTI-1601 as a frataxin replacement therapy. We recently completed dosing of the third cohort in our placebo-controlled multiple-ascending dose (MAD) trial and expect to report topline data from both this study and our placebo-controlled single- ascending dose (SAD) study in the second quarter. These data will provide key insights into CTI-1601s safety and tolerability, as well as into the frataxin levels achieved in patients administered CTI-1601 at the evaluated doses and dosing regimens. We remain on track to initiate our open-label extension, the JIVE trial, and a pediatric MAD trial in FA patients during the second half of the year, which will allow us to further advance CTI-1601s development. We believe the sustained progress of these clinical programs, combined with the continued execution of our corporate milestones, will position us to generate stakeholder value as we advance toward our goal of addressing the unmet needs of FA patients.

2020 and Subsequent Highlights

-- In May 2020, Larimar announced the completion of the reverse merger between Chondrial Therapeutics, Inc. and Zafgen, Inc. The combined, publicly traded clinical-stage biotechnology company began operating under the name Larimar Therapeutics, Inc. and its shares commenced trading on the Nasdaq Global Market on May 29, 2020, under the ticker symbol LRMR. -- In May 2020, Larimar completed a private placement of common stock and pre-funded warrants to purchase common stock for $80 million of gross proceeds before placement agent fees and expenses. -- In August 2020, the European Commission granted an orphan drug designation for CTI-1601 for the treatment of FA. This designation complements previously received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. -- In December 2020, Larimar announced the completion of dosing in its SAD clinical trial in FA patients. Preliminary data from the SAD trial suggest that single subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg. -- Larimar recently completed dosing of the third cohort of its double-blind, placebo-controlled, MAD clinical trial. Topline data from both the SAD and MAD trials are expected in Q2 2021.

Fourth Quarter and Full Year 2020 Financial Results

As of December 31, 2020, the Company had cash, cash equivalents, and marketable debt securities totaling $92.6 million.

The Company reported a net loss for the fourth quarter of 2020 of $14.2 million, or $0.89 per share, compared to a net loss of $6.1 million, or $1.00 per share, for the fourth quarter of 2019.

Research and development expenses for the fourth quarter of 2020 were $10.6 million compared to $5.4 million for the fourth quarter of 2019. The increase in research and development expenses compared to the prior year period was primarily driven by higher clinical supply manufacturing costs, an increase in clinical trial costs, an increase in personnel related costs due to headcount additions in our research and development functions and an increase in stock compensation expense associated with stock option grants made in 2020.

General and administrative expenses for the fourth quarter of 2020 were $3.8 million, compared to $0.8million for the fourth quarter of 2019. The increase in general and administrative expenses as compared to the prior year period was primarily driven by an increase in professional fees and insurance costs that are primarily due to the costs of operating as a public company, an increase in personnel related costs due to increased headcount, and an increase in stock-based compensation associated with stock option grants made in 2020.

For the full year 2020, the Company reported a net loss of $42.5 million, or $3.57 per share, compared to a net loss of $23.1 million, or $3.80 per share for the same period in 2019.

Research and development expenses for the full year 2020 were $31.4 million compared to $20.8 million for the same period in 2019. The increase in research and development expenses compared to the prior year period was primarily driven by higher clinical supply manufacturing costs, an increase in clinical trial costs, an increase in personnel related costs due to headcount additions in our research and development functions and an increase in stock compensation expense associated with stock option grants made in 2020 partially offset by a decrease in toxicology studies costs.

General and administrative expenses for the full year 2020 were $11.4 million, compared to $2.4million for the same period in 2019. The increase in general and administrative expenses as compared to the prior year period was primarily driven by an increase in professional fees and insurance costs that are primarily due to the costs of operating as a public company, an increase in personnel related costs due to increased headcount, and an increase in stock-based compensation associated with stock option grants made in 2020.

About Larimar TherapeuticsLarimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The companys lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program in the U.S. as a potential treatment for FA. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.

Forward-Looking StatementsThis press release contains forward-looking statements that are based on Larimars managements beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimars ability to develop and commercialize CTI-1601 and other planned product candidates, Larimars planned research and development efforts, and other matters regarding Larimars business strategies, use of capital, results of operations and financial position, and plans and objectives for future operations.

In some cases, you can identify forward-looking statements by the words may, will, could, would, should, expect, intend, plan, anticipate, believe, estimate, predict, project, potential, continue, ongoing or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimars product development activities, studies and clinical trials, including CTI-1601 clinical milestones; the ongoing impact of the COVID-19 pandemic on Larimars clinical trial, manufacturing, regulatory and nonclinical study timelines, ability to raise additional capital and general economic conditions; Larimars ability to optimize and scale CTI-1601s manufacturing process; Larimars ability to obtain regulatory approval for CTI-1601 and future product candidates; Larimars ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and successfully commercialize any approved product candidates; Larimars ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by the Company with the Securities and Exchange Commission (SEC), including but not limited to Larimars periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent views as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law.

Investor Contact: Company Contact:Joyce Allaire Michael CelanoLifeSci Advisors Chief Financial Officerjallaire@lifesciadvisors.com mcelano@larimartx.com(212) 915-2569 (484) 414-2715

Larimar Therapeutics, Inc.Consolidated Balance Sheet(unaudited) December31, December31, 2020 2019 Assets Current assets: Cash and cash equivalents $ 68,148 $ 1,009 Marketable debt securities 24,490 ? Prepaid expenses and other current assets 5,314 3,741 Total current assets 97,952 4,750 Property and equipment, net 1,040 274 Operating lease right-of-use assets 3,936 87 Restricted cash 1,339 ? Other assets 419 90 Total assets $ 104,686 $ 5,201 Liabilities and Stockholders? Equity (Deficit) Current liabilities: Accounts payable $ 2,634 $ 3,539 Accrued expenses 5,843 2,259 Operating lease liabilities, current 515 97 Total current liabilities 8,992 5,895 Operating lease liabilities 6,002 ? Total liabilities 14,994 5,895 Commitments and contingencies (See Note 9) Stockholders? equity: Preferred stock; $0.001 par value per share;5,000,000 shares authorizedas of December 31, 2020 and December 31, 2019; no ? ? shares issued andoutstanding as of December 31, 2020 and December31, 2019Common stock, $0.001 par value per share;115,000,000 sharesauthorized as of December 31, 2020 and December 31,2019; 15 6 15,367,730 and 6,091,250 shares issued andoutstanding as ofDecember 31, 2020 and December 31, 2019,respectivelyAdditional paid-in capital 155,290 22,432 Accumulated deficit (65,614 ) (23,132 )Accumulated other comprehensive gain 1 ? Total stockholders? equity (deficit) 89,692 (694 )Total liabilities and stockholders? equity $ 104,686 $ 5,201 (deficit)

Larimar Therapeutics, Inc.Consolidated Statements of Operations(In thousands, except share and per share data)(unaudited) Three Months Ended December 31, Year Ended December 31, 2020 2019 2020 2019 Operating expenses:Research and $ 10,563 $ 5,406 $ 31,407 $ 20,790 developmentGeneral and 3,832 751 11,397 2,424 administrativeTotaloperating 14,395 6,157 42,804 23,214 expensesLoss from (14,395 ) (6,157 ) (42,804 ) (23,214 )operationsOther income, 192 82 322 82 netNet loss $ (14,203 ) $ (6,075 ) $ (42,482 ) $ (23,132 )Net loss pershare, basic $ (0.89 ) $ (1.00 ) $ (3.57 ) $ (3.80 )and dilutedWeightedaverage commonshares 15,985,199 6,091,250 11,883,106 6,091,250 outstanding,basic anddiluted