Larimar Therapeutics Reports Fourth Quarter and Full Year 2022 Operating and Financial Results
- First cohort of Larimar’s Phase 2 dose exploration trial of CTI-1601 in participants with Friedreich’s ataxia (FA) is fully enrolled and proceeding as planned
Larimar expects to provide an update on the next steps of the Phase 2 trial in Q2 2023, after the FDA and independent data monitoring committee review data from the first cohort and provide feedback to the Company- Top-line safety, pharmacokinetic, and pharmacodynamic (e.g., frataxin level) data from both of the Phase 2 trial’s planned cohorts expected in 2H 2023
- Cash and investments of $118.4 million at December 31, 2022 provides projected cash runway into 2H 2024
“Our recent progress provides a strong foundation for growth as we work to develop CTI-1601 as the first FA therapy designed to potentially address frataxin deficiency, which is the underlying cause of the disease,” said
2022 and Subsequent Highlights
Larimar recently completed enrollment in the 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in participants with FA. The trial was cleared to begin enrollment in this first cohort inSeptember 2022 , when theU.S. Food and Drug Administration (FDA) lifted the full clinical hold previously placed on the CTI-1601 program and imposed a partial hold. The initiation of additional cohorts in the Phase 2 trial and/or the initiation of other clinical trials of CTI-1601 are contingent on a review of the Phase 2 trial’s 25 mg cohort data by its independent data monitoring committee (IDMC) and the FDA. Larimar expects to provide an update on the next steps for the Phase 2 trial in Q2 2023, after it receives feedback from the FDA and IDMC on their review of data from the trial’s 25 mg cohort. Top-line safety, pharmacokinetic, and pharmacodynamic (e.g., frataxin level) data from both cohorts of the Phase 2 trial are expected in 2H 2023.- In
September 2022 ,Larimar raised net proceeds of approximately$75.2 million through an underwritten offering of common stock, withDeerfield Management and other notable life science investors participating in the offering. - In
October 2022 ,Larimar announced the issuance ofU.S. Patent No. 11,459,363, which provides composition of matter protection for CTI-1601 into at leastJuly 2040 . - In
February 2023 ,Larimar strengthened its leadership team with the appointment ofGopi Shankar , PhD, MBA, FAAPS, to the newly created position of Chief Development Officer. In this role,Dr. Shankar is responsible for the strategic development of Larimar’s clinical and R&D programs.Dr. Shankar joinedLarimar with more than 20 years of experience leading the development of novel biologics, most recently serving as Vice President and Global Head, Biologics Development Sciences atJanssen Research & Development (a pharmaceutical company ofJohnson & Johnson, Inc. ).
Fourth Quarter and Full Year 2022 Financial Results
As of
The Company reported a net loss for the fourth quarter of 2022 of
Research and development expenses for the fourth quarter of 2022 were
General and administrative expenses for the fourth quarter of 2022 were
For the full year 2022, the Company reported a net loss of
Research and development expenses for the full year 2022 were
General and administrative expenses for the full year 2022 were
About
Forward-Looking Statements
This press release contains forward-looking statements that are based on Larimar’s management’s 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 Larimar’s expectations regarding its ability to resolve the partial clinical hold imposed by the FDA related to CTI-1601, Larimar’s ability to develop and commercialize CTI-1601 and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its CTI-1601 clinical development plan and other matters regarding Larimar’s business strategies, ability to raise capital, 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, Larimar’s ability to successfully engage with the FDA and satisfactorily respond to requests from the FDA for further information and data regarding the CTI-1601 clinical trial including the FDA review of data from cohort one from the Phase 2 dose exploration trial and FDA‘s agreement to escalate the dosing in cohort two, the timing and outcomes of Larimar’s interactions with the FDA concerning the partial clinical hold, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including CTI-1601 clinical milestones; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of CTI-1601 may not be predictive of the results or success of later clinical trials, and assessments; the ongoing impact of the COVID-19 pandemic on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale CTI-1601’s manufacturing process; Larimar’s ability to obtain regulatory approvals for CTI-1601 and future product candidates; Larimar’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s 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 Larimar’s management’s views only 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:
Joyce Allaire
LifeSci Advisors
jallaire@lifesciadvisors.com
(212) 915-2569
Company Contact:
Michael Celano
Chief Financial Officer
mcelano@larimartx.com
(484) 414-2715
| Consolidated Balance Sheet (unaudited) |
||||||||
| 2022 | 2021 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 26,825 | $ | 70,097 | ||||
| Marketable securities | 91,603 | — | ||||||
| Prepaid expenses and other current assets | 2,311 | 2,107 | ||||||
| Total current assets | 120,739 | 72,204 | ||||||
| Property and equipment, net | 831 | 1,049 | ||||||
| Operating lease right-of-use assets | 2,858 | 3,406 | ||||||
| Restricted cash | 1,339 | 1,339 | ||||||
| Other assets | 638 | 669 | ||||||
| Total assets | $ | 126,405 | $ | 78,667 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,686 | $ | 1,660 | ||||
| Accrued expenses | 8,408 | 6,592 | ||||||
| Operating lease liabilities, current | 611 | 594 | ||||||
| Total current liabilities | 10,705 | 8,846 | ||||||
| Operating lease liabilities | 4,797 | 5,408 | ||||||
| Total liabilities | 15,502 | 14,254 | ||||||
| Commitments and contingencies (See Note 8) | ||||||||
| Stockholders’ equity: | ||||||||
| Preferred stock; as of outstanding as of |
— | — | ||||||
| Common stock, authorized as of 43,269,200 and 17,710,450 shares issued and outstanding as of |
43 | 18 | ||||||
| Additional paid-in capital | 262,496 | 180,645 | ||||||
| Accumulated deficit | (151,605 | ) | (116,250 | ) | ||||
| Accumulated other comprehensive loss | (31 | ) | — | |||||
| Total stockholders’ equity | 110,903 | 64,413 | ||||||
| Total liabilities and stockholders’ equity | $ | 126,405 | $ | 78,667 | ||||
| Consolidated Statements of Operations | ||||||||||||||||
| (In thousands, except share and per share data) (unaudited) |
||||||||||||||||
| Three Months Ended |
Year Ended |
|||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 7,218 | $ | 6,292 | $ | 24,250 | $ | 38,396 | ||||||||
| General and administrative | 3,221 | 2,794 | 12,276 | 12,069 | ||||||||||||
| Total operating expenses | 10,439 | 9,086 | 36,526 | 50,465 | ||||||||||||
| Loss from operations | (10,439 | ) | (9,086 | ) | (36,526 | ) | (50,465 | ) | ||||||||
| Other income (expense), net | 1,014 | (48 | ) | 1,171 | (171 | ) | ||||||||||
| Net loss | $ | (9,425 | ) | $ | (9,134 | ) | $ | (35,355 | ) | $ | (50,636 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.21 | ) | $ | (0.50 | ) | $ | (1.37 | ) | $ | (2.95 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 43,897,603 | 18,338,853 | 25,761,394 | 17,164,284 | ||||||||||||
Source: Larimar Therapeutics