Top Line Results from CLEAR MIND Alzheimer’s disease Trial Affirm Safety Profile of Lomecel-B™ and Provide Efficacy Signal; Full Study Data Remains on Track to Share in Coming Weeks
Long-term Survival Data from ELPIS 1 Trial to be Presented at the 2023 Scientific Sessions of the
ELPIS II Phase 2 Trial of Lomecel-B™ in HLHS Continues Enrollment; Completion of Study Enrollment Anticipated in 2024
Company to Hold Conference Call and Webcast Today,
“During our third quarter and in recent weeks, we continued to advance our Lomecel-B™ clinical programs, highlighted by postive top-line results from our CLEAR MIND clinical trial of Lomecel-B™ in the treatment of mild Alzheimer’s disease and acceptance of ELPIS I long-term survival data for presentation at the
“As we announced in October, the results from our CLEAR MIND trial provide important validation of both the safety and therapeutic potential of Lomecel-B™ in the treatment of mild Alzheimer’s disease. In addition to achieving the study’s safety endpoints, the study provided statistically significant evidence of slowing disease progression as compared to placebo. We believe these results provide a robust foundation for additional clinical programs with Lomecel-B™ in Alzheimer’s disease as well as other indications, and look forward to reporting the full study data from this trial in the coming weeks once the remaining data is received and analyzed.
“We are also pleased to see our poster, which details the five-year long-term survival data from our ELPIS I trial in hypoplastic left heart syndrome (HLHS), accepted for presentation at the upcoming
Recent Highlights
Postive Topline Data Announced from CLEAR MIND Trial
- In
October 2023 ,Longeveron announced topline results from the CLEAR MIND trial— a Phase 2a trial of Lomecel-B™ for the treatment of mild Alzheimer’s disease (AD). CLEAR MIND is a 48 patient, 4-arm, parallel design, randomized (1:1:1:1) clinical trial of Lomecel-B™ intended to evaluate the safety of a single and multiple infusions of two different dose levels compared to placebo in patients with mild AD. - The primary endpoint of safety was met based on statistical and medical assessment. There was one Serious Adverse Event (SAE) reported on each Lomecel-B™ treatment group and none on placebo. Each SAE was reviewed and assessed by the Data and Safety Monitoring Board (DSMB) with no safety issues raised.
- The secondary endpoint of change from baseline to week 39 in CADS demonstrated positive results at the prespecified statistical level of p<0.1, 2-tailed; statistically significant improvement at Week 39 in CADS was observed for the Lomecel-B™ 25 x 106 cells (25M) x 1 dose (p=0.091) versus placebo and for the pooled Lomecel-B™ Groups (25M x 1 dose, 25M x 4 doses, 100 x 106 cells (100M) x 4 doses) (p=0.099).
- Lomecel-B™ at the 25M x 1 dose demonstrated statistically significant slowing of disease progression in left hippocampal volume (p=0.015) relative to placebo; ADCS-ADL and left hippocampal volume at Week 39 were statistically significant for the pooled Lomecel-B™ treatment groups (25M x 1 dose, 25M x 4 doses, 100M x 4 doses) relative to placebo (p=0.047) and (p=0.038), respectively; other doses demonstrated numerical slowing and prevention of disease worsening relative to placebo in CADS, ADAS-cog13, CDR-SB, ADCS-ADL and left hippocampal volume at Week 39.
- Complete study data remains on track to be shared in the coming weeks once the full data is received and analyzed.
ELPIS 1 Long-term Survival Data Accepted for Presentation at AHA 2023
- An abstract highlighting long-term survival data from the ELPIS 1 trial of Lomecel-B™ for patients with hypoplastic left heart syndrome (HLHS) has been accepted for presentation as a poster at the upcoming annual meeting of the AHA.
- Previously announced long-term survival data from ELPIS I Trial of Lomecel-B™ for HLHS showed:
- Children in the ELPIS I trial had 100% survival up to five years of age after receiving Lomecel-B™ compared to approximate 20% mortality rate observed from historical control data.
- Data reinforce potential survival benefit of Lomecel-B™ for patients in this indication.
Enrollment continues in the Company’s Phase 2 study evaluating Lomecel-B™ in patients with Aging-Related Frailty in
- The Phase 2 clinical trial is a 3-arm, parallel design, randomized (1:1:1), placebo-controlled, double-blind study of two different dose levels of Lomecel-B™. The trial is expected to enroll 45 patients and has a primary objective of evaluating the safety of Lomecel-B™ as a treatment for Aging-related Frailty.
- The Phase 2 trial is being conducted in partnership with the
National Center for Geriatrics & Gerontology (NCGG;Nagoya ) andJuntendo University Hospital (Tokyo ).
Financial Results for Quarter Ended
- Revenues, Cost of Revenues and Gross Profit: Revenues for each of the three months ended
September 30, 2023 and 2022 were approximately$0.2 million and$0.3 million , respectively. Grant revenue for the three months endedSeptember 30, 2023 and 2022 was$0 and$0.1 million , respectively. The decrease of$0.1 million , or 100%, was primarily due to a reduction in grant funds available due in part to the completion of the grant-funded clinical trials. Clinical trial revenue, which is derived from the Bahamas Registry Trial, for the three-month periods endedSeptember 30, 2023 and 2022 was$0.2 million . Clinical trial revenue for the three months endedSeptember 30, 2023 decreased by less than$0.1 million , or 29%, compared to the same period in 2022 as a result of a decrease in participant demand. - Related cost of revenues was approximately
$0.1 million and$0.2 million for the three months endedSeptember 30, 2023 and 2022, respectively. The decrease of$0.1 million , or 45%, was primarily due to the decrease in the revenues earned from the Bahamas Registry Trial. This resulted in a gross profit of approximately$0.1 million for both of the three month-periods endedSeptember 30, 2023 and 2022. - General and Administrative Expense: General and administrative expenses for the three months ended
September 30, 2023 increased to approximately$3.1 million , compared to$2.1 million for the same period in 2022. The increase of approximately$1.0 million , or 49%, was primarily related to increases of$0.3 million in compensation and benefit expenses,$0.4 million of expenses related to legal and professional fees and$0.4 million of expenses related to the subscription rights offering. These increases were partially offset by a decrease of$0.1 million in stock-based compensation expense. - Research and Development Expenses: Research and development expenses for the three months ended
September 30, 2023 decreased to approximately$1.8 million , from approximately$3.0 million for the same period in 2022. The decrease of$1.1 million , or 38%, was primarily due to a decrease of$1.0 million in research and development expenses driven by lower spend on clinical trials and supplies. - Selling and Marketing Expenses: Selling and marketing expenses for the three months ended
September 30, 2023 and 2022 were approximately$0.3 million and$0.2 million , respectively. Selling and marketing expenses consist primarily of investor and public relations expenses. - Other Income (Expense): Other income for the three months ended
September 30, 2023 was$0.1 million , which consisted of interest income. Other expense for the three months endedSeptember 30, 2022 was less than$0.1 million . - Net Loss: Net loss was approximately
$5.1 million and$5.2 million for the three-month periods endedSeptember 30, 2023 and 2022, respectively. - Cash and short-term investments: As of
September 30, 2023 , the Company had cash and cash equivalents of$2.0 million , marketable securities of$2.0 million and working capital of approximately$1.7 million . As ofDecember 31, 2022 , cash and cash equivalents was$10.5 million , marketable securities were$9.2 million and working capital was approximately$15.4 million . - Subsequent Events: On
October 13, 2023 the Company closed a registered direct equity offering priced at-the-market under Nasdaq rules and concurrent private placement, with gross proceeds of approximately$4 million , before deducting the placement agent’s fees and other offering expenses payable byLongeveron .Longeveron currently intends to use the net proceeds from the offering to fund the ongoing clinical and regulatory development of Lomecel-B™ and for capital expenditures, working capital and general corporate purposes. - Certain reclassifications have been made to prior year financial statements to conform to classifications used in the current year. These reclassifications had no impact on net loss, shareholders’ equity or cash flows as previously reported.
- Financial Outlook: Based on the Company’s current operating plan and financial resources, we believe that our existing cash and short-term investments will be sufficient to cover expenses and capital requirements into the first quarter of 2024.
Conference Call and Webcast
Management will host a conference call today at
Title | Longeveron Third Quarter 2023 Financial Results |
Date | |
Time | |
Conference Call Details | 1-877-407-0789 - 1-201-689-8562- |
Conference ID | 13742198 |
Call me™ Feature | Click Here |
Webcast | Click Here |
About
Forward-Looking Statements
Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management’s current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as “believe,” “expects,” “may,” “looks to,” “will,” “should,” “plan,” “intend,” “on condition,” “target,” “see,” “potential,” “estimates,” “preliminary,” or “anticipates” or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, the anticipated use of proceeds from the offering, as well as statements about the ability of Longeveron’s clinical trials to demonstrate safety and efficacy of the Company’s product candidates, and other positive results; the timing and focus of the Company’s ongoing and future preclinical studies and clinical trials and the reporting of data from those studies and trials; the size of the market opportunity for the Company’s product candidates, including its estimates of the number of patients who suffer from the diseases being targeted; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of the Company’s product candidates; the Company’s ability to obtain and maintain regulatory approval of its product candidates in the
Investor Contact
Tel: 617-308-4306
Email: mmoyer@lifesciadvisors.com
---tables to follow---
Condensed Statements of Operations | ||||||||||||||||
(In thousands, except per share data) | ||||||||||||||||
(Unaudited) | ||||||||||||||||
Three months ended | Nine months ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
Revenues | ||||||||||||||||
Clinical trial revenue | $ | 150 | $ | 210 | $ | 605 | $ | 860 | ||||||||
Grant revenue | - | 55 | 41 | 241 | ||||||||||||
Total revenues | 150 | 265 | 646 | 1,101 | ||||||||||||
Cost of revenues | 96 | 173 | 423 | 549 | ||||||||||||
Gross profit | 54 | 92 | 223 | 552 | ||||||||||||
Operating expenses | ||||||||||||||||
General and administrative | 3,092 | 2,074 | 8,322 | 6,481 | ||||||||||||
Research and development | 1,843 | 2,960 | 6,910 | 6,107 | ||||||||||||
Selling and marketing | 280 | 245 | 580 | 766 | ||||||||||||
Total operating expenses | 5,215 | 5,279 | 15,812 | 13,354 | ||||||||||||
Loss from operations | (5,161 | ) | (5,187 | ) | (15,589 | ) | (12,802 | ) | ||||||||
Other income and (expenses) | ||||||||||||||||
Non-operating lawsuit expense | - | - | - | (1,398 | ) | |||||||||||
Other income and (expenses), net | 55 | (57 | ) | 204 | (178 | ) | ||||||||||
Total other income and (expenses), net | 55 | (57 | ) | 204 | (1,576 | ) | ||||||||||
Net loss | $ | (5,106 | ) | $ | (5,244 | ) | $ | (15,385 | ) | $ | (14,378 | ) | ||||
Deemed dividend attributable to warrant down round feature | (798 | ) | - | (798 | ) | - | ||||||||||
Net loss attributable to common stockholders | $ | (5,904 | ) | $ | (5,244 | ) | $ | (16,183 | ) | $ | (14,378 | ) | ||||
Basic and diluted net loss per share | $ | (0.28 | ) | $ | (0.25 | ) | $ | (0.77 | ) | $ | (0.69 | ) | ||||
Basic and diluted weighted average common shares outstanding | 21,178,767 | 21,001,613 | 21,106,464 | 20,952,569 |
See accompanying notes to unaudited condensed financial statements.
Condensed Balance Sheets | |||||||||
(In thousands, except share and per share data) | |||||||||
2023 | 2022 | ||||||||
(Unaudited) | |||||||||
Assets | |||||||||
Current assets: | |||||||||
Cash and cash equivalents | $ | 1,967 | $ | 10,503 | |||||
Marketable securities | 1,966 | 9,155 | |||||||
Prepaid expenses and other current assets | 1,015 | 404 | |||||||
Accounts and grants receivable | 96 | 218 | |||||||
Total current assets | 5,044 | 20,280 | |||||||
Property and equipment, net | 2,544 | 2,949 | |||||||
Intangible assets, net | 2,539 | 2,409 | |||||||
Operating lease asset | 1,301 | 1,531 | |||||||
Other assets | 197 | 244 | |||||||
Total assets | $ | 11,625 | $ | 27,413 | |||||
Liabilities and stockholders’ equity | |||||||||
Current liabilities: | |||||||||
Accounts payable | $ | 829 | $ | 1,751 | |||||
Accrued expenses | 1,473 | 650 | |||||||
Current portion of lease liability | 586 | 564 | |||||||
Estimated lawsuit liability | - | 1,398 | |||||||
Deferred revenue | 506 | 506 | |||||||
Total current liabilities | 3,394 | 4,869 | |||||||
Long-term liabilities: | |||||||||
Lease liability | 1,599 | 2,041 | |||||||
Total long-term liabilities | 1,599 | 2,041 | |||||||
Total liabilities | 4,993 | 6,910 | |||||||
Commitments and contingencies (Note 9) | |||||||||
Stockholders’ equity: | |||||||||
Preferred stock, | - | - | |||||||
Class A common stock, | 6 | 6 | |||||||
Class B common stock, | 15 | 15 | |||||||
Additional paid-in capital | 85,976 | 83,712 | |||||||
Stock subscription receivable | (100 | ) | (100 | ) | |||||
Accumulated deficit | (78,956 | ) | (62,773 | ) | |||||
Accumulated other comprehensive loss | (309 | ) | (357 | ) | |||||
Total stockholders’ equity | 6,632 | 20,503 | |||||||
Total liabilities and stockholders’ equity | $ | 11,625 | $ | 27,413 |
See accompanying notes to unaudited condensed financial statements.
Source:
2023 GlobeNewswire, Inc., source