Larimar Therapeutics Reports Third Quarter 2023 Operating and Financial Results
- Completed full enrollment and dosing of the 50 mg cohort in Phase 2 Friedreich's ataxia (FA) dose exploration trial; based on blinded observations during the dosing period, there were no serious adverse events in either the CTI-1601 (nomlabofusp) or placebo groups.
- Top-line safety, pharmacokinetic, and frataxin data from the Phase 2 trial now expected in Q1 2024, refined from H1 2024
- Initiation of open label extension (OLE) trial with 25 mg daily dosing of nomlabofusp remains on track for Q1 2024; interim data expected in Q4 2024
- Cash, cash equivalents and marketable securities of
$95.6 millionas of September 30, 2023, provides projected cash runway into Q1 2025
“We are pleased with the execution and pace at which our nomlabofusp program is advancing. The 50 mg cohort in our Phase 2 dose exploration trial is fully enrolled and all 15 participants have completed dosing and continue in the blinded follow up period. Based on blinded observations during the dosing period, there were no serious adverse events in either the nomlabofusp or placebo groups. We expect top-line safety, pharmacokinetic, and frataxin data now in the first quarter of 2024, refined from the first half of 2024. As our next major catalyst, clinical findings from the 50 mg cohort should provide additional data to inform the dose and dose regimen for our dose exploration trial, potential registrational trial and any dosing updates to our soon to be initiated OLE trial,” said
“We continue to have ongoing interactions with global regulatory health authorities regarding manufacturing, regulatory pathways, and clinical development with a focus on initiating the pediatric clinical development program and planning our global clinical studies. The addition of Dr.
Third Quarter and Subsequent Highlights
November 2023, Larimarcompleted enrollment and dosing of the 50 mg cohort of its Phase 2 double-blind dose exploration trial evaluating CTI-1601 (nomlabofusp) for the treatment of Friedreich’s ataxia. Treatment assignment of the fully enrolled cohort of 15 participants remains blinded as they complete the follow up period. Participants were dosed daily with nomlabofusp or placebo for the first 14 days, and then every other day until Day 28. Based on blinded Phase 2 observations during the dosing period, there were no serious adverse events for either the nomlabofusp or placebo groups. Top-line Phase 2 safety, pharmacokinetic, and frataxin data from skin and buccal cells from both the 25 mg and 50 mg cohorts is now expected in the first quarter of 2024, refined from the first half of 2024. Initiation of additional U.S.clinical trials or potential further dose escalation in these trials is contingent on FDA review of Phase 2 data from the 50 mg cohort due to the partial clinical hold.
November 2023, Larimarreaffirmed guidance for initiation of the OLE trial evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver. Participants who complete treatment in the Phase 2 dose exploration trial, or who previously completed a prior clinical trial of nomlabofusp are potentially eligible for the OLE. The OLE will evaluate the safety and tolerability, pharmacokinetics, and measures of frataxin levels in peripheral tissues as well as other exploratory pharmacodynamic markers ( lipid profiles and gene expression data)following long-term subcutaneous administration of nomlabofusp. Clinical measures collected during the trial will be compared to data from a synthetic control arm derived from participants in the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) database. The OLE trial is expected to begin in Q1 2024 with interim data expected in Q4 2024.
October 2023, Larimarappointed Jeffrey W. Sherman, M.D., F.A.C.P. to the Company’s Board of Directors. Dr. Sherman, Executive Vice President, Chief Medical Officer (CMO) at Horizon Therapeutics Public Limited Company (recently acquired by Amgen), brings more than 25 years of pharmaceutical experience, specializing in regulatory and clinical strategy, and therapeutic development for rare diseases.
- As of
October 2023, "nomlabofusp" was published as the INN (International Nonproprietary Name) and USAN (United States Adopted Name) for CTI-1601.
July 2023, Larimarreceived FDA clearance to initiate both a 50 mg cohort in the Phase 2 dose exploration trial evaluating nomlabofusp for FA and an OLE trial following FDA review of unblinded safety, pharmacokinetic, and frataxin data from the Phase 2 trial’s 25 mg cohort.
Third Quarter 2023 Financial Results
The Company reported a net loss for the third quarter of 2023 of
Research and development expenses for the third quarter of 2023 were
General and administrative expenses for the third quarter of 2023 were
Other income (expense), net was
The Company reported a net loss for the 9-month period ending
Research and development expenses for the 9-month period ending
General and administrative expenses for the 9-month period ending
Other income (expense), net was
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 ability to develop and commercialize nomlabofusp (also known as CTI-1601) and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials and overall 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, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical milestones and continued interactions with the FDA regarding the partial clinical hold; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later clinical trials, and assessments; the potential impact of public health crises 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
Chief Financial Officer
|Condensed Consolidated Balance Sheet|
|Cash and cash equivalents||$||38,721||$||26,825|
|Prepaid expenses and other current assets||2,890||2,311|
|Total current assets||98,480||120,739|
|Property and equipment, net||601||831|
|Operating lease right-of-use assets||2,898||2,858|
|Liabilities and Stockholders’ Equity|
|Operating lease liabilities, current||708||611|
|Total current liabilities||6,558||10,705|
|Operating lease liabilities||4,682||4,797|
|Commitments and contingencies (See Note 8)|
authorized as of
|Additional paid-in capital||268,223||262,496|
|Accumulated other comprehensive gain (loss)||7||(31||)|
|Total stockholders’ equity||92,712||110,903|
|Total liabilities and stockholders’ equity||$||103,952||$||126,405|
|Condensed Consolidated Statements of Operations|
|(In thousands, except share and per share data)|
|Three Months Ended
||Nine Months Ended
|Research and development||$||6,585||$||5,582||$||17,022||$||17,032|
|General and administrative||3,754||2,931||10,574||9,055|
|Total operating expenses||10,339||8,513||27,596||26,087|
|Loss from operations||(10,339||)||(8,513||)||(27,596||)||(26,087||)|
|Other income, net||1,275||193||3,640||157|
|Net loss per share, basic and diluted||$||(0.21||)||$||(0.37||)||$||(0.55||)||$||(1.32||)|
|Weighted average common shares outstanding, basic and diluted||43,903,738||22,228,228||43,899,670||19,649,558|
|Other comprehensive gain (loss):|
|Unrealized gain (loss) on marketable securities||(5||)||17||38||(40||)|
|Total other comprehensive gain (loss)||(5||)||17||38||(40||)|
|Total comprehensive loss||$||(9,069||)||$||(8,303||)||$||(23,918||)||$||(25,970||)|
Source: Larimar Therapeutics