- Initiation of a Phase 2 study, ALTITUDE-AD, to investigate ACU193 for the treatment of early Alzheimer’s disease expected in the first half of 2024, following positive FDA interaction in
October 2023
- Announced global collaboration and license agreement with
Halozyme for development of a subcutaneous formulation of ACU193- Initiation of a Phase 1 study to support a subcutaneous dosing option of ACU193 expected in mid-2024
- Announced a credit facility of up to
$50 million from K2 HealthVentures, to provide capital to support subcutaneous clinical work and general corporate purposes
- CSF biomarker data observed in the Phase 1 INTERCEPT-AD trial support pharmacology of ACU193 and role of amyloid beta oligomers in Alzheimer’s disease
- Cash, cash equivalents and marketable securities of
$282.7 million as ofSept. 30, 2023 , expected to support current clinical and operational activities into the second half of 2026
- Company to host conference call and webcast today at
8:00 a.m. ET
“My excitement for Acumen’s future is centered on the strength of our ACU193 development strategy for the treatment of early Alzheimer’s disease, and the deep expertise of our team to execute on that strategy. We continue to make significant operational, regulatory and strategic progress. Following our positive Phase 1 data in the third quarter, we recently had a positive interaction with the FDA and plan to initiate a Phase 2 study in the first half of 2024. On a strategic front, I am excited about our recent collaboration and licensing agreement with
Recent Highlights and Anticipated Milestones
- In
October 2023 , the Company presented additional detail from first-in-human Phase 1 study of ACU193 for early AD at the Clinical Trials on Alzheimer’s Disease (CTAD) conference.- Announced dose selection of 50 mg/kg and 35 mg/kg every 4 weeks for ACU193 treatment arms in upcoming placebo-controlled Phase 2 trial based on significant target engagement of AβOs approaching maximal effect.
- Presented data exploring target engagement modeling of ACU193 to inform dose selection, plus further analyses of dose-related amyloid plaque reduction and clinical characteristics of ARIA-E, confirming proof-of-mechanism for ACU193.
- In
October 2023 , the Company met with the FDA to discuss the next clinical trial in the development program for ACU193.- The agency indicated they are aligned in principle with the Phase 2/3 study design.
- In the first half of 2024, the Company expects to initiate a Phase 2 study, ALTITUDE-AD, as the next phase of development for ACU193.
- Today, the Company announced encouraging results from INTERCEPT-AD Phase 1 cerebrospinal fluid (CSF) biomarker data, further supporting the pharmacologic activity of ACU193.
- Observed dose dependent trend in the multiple ascending dose cohorts on CSF levels of p-tau181, total tau, neurogranin and Aβ 42/40 ratio.
- P-tau181 (p=0.049) and neurogranin (p=0.037) showed statistically significant improvement at 60 mg/kg Q4W as compared to the placebo group after three administrations of ACU193.
- Nominally significant correlation between target engagement of AβOs and change in neurogranin observed across all doses.
- Trend between target engagement of AβOs and change in p-tau181 observed across all doses.
- Plasma biomarker analysis in progress.
“We have observed a dose-dependent trend toward drug effect of ACU193 on CSF levels of p-tau181, total tau, neurogranin and the Aβ 42/40 ratio after only three administrations. The fact that we are observing changes in CSF biomarkers is highly supportive of ACU193’s downstream pharmacology as determined in our Phase 1 study,” said
Corporate Updates
- In
November 2023 , announced a global collaboration and licensing agreement withHalozyme for the development of a subcutaneous formulation of ACU193, to potentially offer additional flexibility and convenience for patients and caregivers.- Expect to initiate a Phase 1 trial investigating a subcutaneous dosing option of ACU193 in mid-2024.
- Today, announced a credit facility for up to
$50 million provided by K2 HealthVentures, for capital to support subcutaneous clinical work as well as general corporate purposes.
Third Quarter 2023 Financial Results
- Cash Balance. As of
September 30, 2023 , cash, cash equivalents and marketable securities totaled$282.7 million , compared to cash, cash equivalents and marketable securities of$172.2 million as ofJune 30, 2023 . This increase is due to the net proceeds from the Company’s public offering of approximately$122 million onJuly 21, 2023 . Altogether, this runway is expected to be sufficient to support current clinical and operational activities into the second half of 2026. - Research and Development (R&D) Expenses. R&D expenses were
$11.2 million for the three-month period endedSeptember 30, 2023 , compared to$8.3 million for the three-month period endedSeptember 30, 2022 . The increase in R&D expenses was primarily due to increased costs related to materials, consulting, personnel and other costs.
- General and Administrative (G&A) Expenses. G&A expenses were
$4.9 million for the three-month period endedSeptember 30, 2023 , compared to$3.1 million for the three-month period endedSeptember 30, 2022 . The increase in G&A expenses was primarily due to increased costs related to personnel, consulting and legal/patent services.
- Loss from Operations. Losses from operations were
$16.0 million for the three-month period endedSeptember 30, 2023 , compared to$11.4 million for the three-month period endedSeptember 30, 2022 . This increase was due to the increased R&D and G&A expenses over the prior year period.
- Net Loss. Net loss was
$13.0 million for the three-month period endedSeptember 30, 2023 , compared to$10.7 million for the three-month period endedSeptember 30, 2022 .
Conference Call Details
To participate in the live conference call, please register using this link. After registration, you will be informed of the dial-in numbers including PIN. Please register at least one day in advance.
The webcast audio will be available via this link.
An archived version of the webcast will be available for at least 30 days in the Investors section of the Company's website at www.acumenpharm.com.
About ACU193
ACU193 is a humanized monoclonal antibody (mAb) discovered and developed based on its selectivity for soluble AβOs, which
About INTERCEPT-AD
INTERCEPT-AD was a Phase 1,
About
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Any statement describing Acumen’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Words such as “believes,” “expects,” “anticipates,” “could,” “should,” “would,” “seeks,” “aims,” “plans,” “potential,” “will,” “milestone” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements include statements concerning Acumen’s business, and Acumen’s ability to achieve its strategic and financial goals, including its projected use of cash, cash equivalents and marketable securities and the expected sufficiency of its cash resources into the second half of 2026, the therapeutic potential of Acumen’s product candidate, ACU193, including against other antibodies, the anticipated timeline for initiating a Phase 2 clinical trial of ACU193 and a Phase 1 trial to support a subcutaneous dosing option of ACU 193, and the expected use of proceeds from a credit facility. These statements are based upon the current beliefs and expectations of
CONTACTS:
Investors:
abraun@acumenpharm.com
Media: AcumenPR@westwicke.com
Condensed Balance Sheets (in thousands, except share and per share data) | |||||||
(unaudited) | |||||||
ASSETS | |||||||
Current assets | |||||||
Cash and cash equivalents | $ | 94,917 | $ | 130,101 | |||
Marketable securities, short-term | 120,517 | 47,504 | |||||
Prepaid expenses and other current assets | 3,164 | 2,724 | |||||
Total current assets | 218,598 | 180,329 | |||||
Marketable securities, long-term | 67,270 | 15,837 | |||||
Restricted cash | 189 | — | |||||
Property and equipment, net | 123 | 165 | |||||
Right-of-use asset | 2 | 105 | |||||
Other assets | 189 | 151 | |||||
Total assets | $ | 286,371 | $ | 196,587 | |||
LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||||
Current liabilities | |||||||
Accounts payable | $ | 1,362 | $ | 1,640 | |||
Accrued clinical trial expenses | 1,566 | 2,717 | |||||
Accrued expenses and other current liabilities | 3,168 | 3,350 | |||||
Operating lease liability | 2 | 105 | |||||
Total current liabilities | 6,098 | 7,812 | |||||
Total liabilities | 6,098 | 7,812 | |||||
Commitments and contingencies | |||||||
Stockholders’ equity | |||||||
Preferred stock, | — | — | |||||
Common stock, | 6 | 4 | |||||
Additional paid-in capital | 487,077 | 359,949 | |||||
Accumulated deficit | (206,301 | ) | (170,427 | ) | |||
Accumulated other comprehensive loss | (509 | ) | (751 | ) | |||
Total stockholders’ equity | 280,273 | 188,775 | |||||
Total liabilities and stockholders’ equity | $ | 286,371 | $ | 196,587 |
Condensed Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (unaudited) | |||||||||||||||
Three Months Ended | Nine Months Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Operating expenses | |||||||||||||||
Research and development | $ | 11,179 | $ | 8,309 | $ | 29,025 | $ | 21,615 | |||||||
General and administrative | 4,860 | 3,062 | 13,627 | 9,374 | |||||||||||
Total operating expenses | 16,039 | 11,371 | 42,652 | 30,989 | |||||||||||
Loss from operations | (16,039 | ) | (11,371 | ) | (42,652 | ) | (30,989 | ) | |||||||
Other income (expense) | |||||||||||||||
Interest income, net | 3,124 | 663 | 6,840 | 1,000 | |||||||||||
Other expense, net | (42 | ) | (2 | ) | (62 | ) | (1 | ) | |||||||
Total other income | 3,082 | 661 | 6,778 | 999 | |||||||||||
Net loss | (12,957 | ) | (10,710 | ) | (35,874 | ) | (29,990 | ) | |||||||
Other comprehensive gain (loss) | |||||||||||||||
Unrealized gain (loss) on marketable securities | 137 | — | 242 | (734 | ) | ||||||||||
Comprehensive loss | $ | (12,820 | ) | $ | (10,710 | ) | $ | (35,632 | ) | $ | (30,724 | ) | |||
Net loss per common share, basic and diluted | $ | (0.24 | ) | $ | (0.26 | ) | $ | (0.79 | ) | $ | (0.74 | ) | |||
Weighted-average shares outstanding, basic and diluted | 54,229,630 | 40,502,860 | 45,474,953 | 40,491,181 |
Condensed Statements of Cash Flows (in thousands) (unaudited) | |||||||
Nine Months Ended | |||||||
2023 | 2022 | ||||||
Cash flows from operating activities | |||||||
Net loss | $ | (35,874 | ) | $ | (29,990 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||
Depreciation | 42 | 20 | |||||
Stock-based compensation expense | 4,511 | 2,173 | |||||
Amortization of premiums and accretion of discounts on marketable securities, net | (1,344 | ) | 575 | ||||
Amortization of right-of-use asset | 103 | 100 | |||||
Changes in operating assets and liabilities: | |||||||
Prepaid expenses and other current assets | (436 | ) | 2,058 | ||||
Other assets | (38 | ) | (78 | ) | |||
Accounts payable | (278 | ) | 996 | ||||
Accrued clinical trial expenses | (1,151 | ) | 1,358 | ||||
Operating lease liability | (103 | ) | (100 | ) | |||
Accrued expenses and other current liabilities | (182 | ) | (1,062 | ) | |||
Net cash used in operating activities | (34,750 | ) | (23,950 | ) | |||
Cash flows from investing activities | |||||||
Purchases of marketable securities | (178,857 | ) | (12,129 | ) | |||
Proceeds from maturities and sales of marketable securities | 55,997 | 71,860 | |||||
Proceeds from sale of property and equipment | 3 | — | |||||
Purchases of property and equipment | (7 | ) | (126 | ) | |||
Net cash provided by (used in) investing activities | (122,864 | ) | 59,605 | ||||
Cash flows from financing activities | |||||||
Proceeds from issuance of common stock, net of issuance costs | 122,294 | — | |||||
Proceeds from exercise of stock options | 325 | 19 | |||||
Payments for deferred offering costs | — | (296 | ) | ||||
Net cash provided by (used in) financing activities | 122,619 | (277 | ) | ||||
Net change in cash and cash equivalents and restricted cash | (34,995 | ) | 35,378 | ||||
Cash and cash equivalents and restricted cash at the beginning of the period | 130,101 | 122,162 | |||||
Cash and cash equivalents and restricted cash at the end of the period | $ | 95,106 | $ | 157,540 |
Source:
2023 GlobeNewswire, Inc., source