Acumen Pharmaceuticals(US:ABOS)2022-03-27 16:00markdown Part I [Business](index=7&type=section&id=Item%201.%20Business) Acumen Pharmaceuticals is a clinical-stage biopharmaceutical company developing ACU193 for Alzheimer's disease [Our Product Candidate and Strategy](index=9&type=section&id=Our%20Product%20Candidate%20and%20Strategy) ACU193 is a humanized monoclonal antibody targeting AßOs for early AD, with a strategy to advance clinical trials - ACU193 is designed to selectively target AßOs with **over 500-fold greater selectivity** for AßOs over Aß monomers and limited binding to amyloid plaques, which may lead to a better safety profile, particularly a lower rate of ARIA[23](index=23&type=chunk)[49](index=49&type=chunk) - The company's strategy includes rapidly advancing ACU193, evaluating it in combination with other drugs (e.g., those targeting tau or inflammation), exploring its use in other indications like Down Syndrome-associated dementia, and expanding the portfolio[28](index=28&type=chunk) [Clinical Development Plan](index=29&type=section&id=Clinical%20Development%20Plan) The INTERCEPT-AD Phase 1 trial evaluates ACU193's safety and pharmacokinetics in early AD patients INTERCEPT-AD Trial Design | Part | Type | Participants | Dosing Cohorts | | :--- | :--- | :--- | :--- | | Part A | Single Ascending Dose (SAD) | 32 | Single IV doses of 2, 10, 25, or 60 mg/kg vs. placebo. | | Part B | Multiple Ascending Dose (MAD) | 30 | Three doses of 10 mg/kg Q4W, 60 mg/kg Q4W, or 60 mg/kg Q2W vs. placebo. | - Due to COVID-19 related delays in site activation and enrollment, the company is expanding the number of trial sites and now anticipates reporting topline data in the **first half of 2023**[113](index=113&type=chunk) [Competition](index=32&type=section&id=Competition) Acumen faces significant competition from approved and late-stage Alzheimer's disease therapies - ACU193 will compete with Biogen's approved drug Aduhelm and late-stage candidates from major pharmaceutical companies including Eisai (lecenamab), Eli Lilly (donanemab), and Roche (gantenerumab), all of which target amyloid plaques and are anticipated to complete Phase 3 studies in 2022-2023[121](index=121&type=chunk) - Numerous other companies are developing therapies with Aß, AßO, and amyloid plaque-related targets, as well as therapies targeting other underlying causes of AD[122](index=122&type=chunk) [Government Regulation](index=34&type=section&id=Government%20Regulation) The company's operations are subject to extensive FDA regulations for drug development, approval, and compliance - The development and approval of biologics like ACU193 is a lengthy process requiring submission of an Investigational New Drug (IND) application before clinical trials and a Biologics License Application (BLA) for marketing approval[139](index=139&type=chunk)[149](index=149&type=chunk) - The FDA offers expedited programs such as Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval for drugs treating serious conditions, which could potentially apply to ACU193[155](index=155&type=chunk)[161](index=161&type=chunk) - Upon approval, a biologic product like ACU193 is granted **12 years of data exclusivity** in the U.S. under the BPCIA, protecting it from biosimilar competition during that period[172](index=172&type=chunk) - The company's sole product candidate, ACU193, is a humanized monoclonal antibody that selectively targets soluble amyloid-beta oligomers (AßOs), a key underlying cause of Alzheimer's disease[12](index=12&type=chunk) - A Phase 1 clinical trial, INTERCEPT-AD, was initiated in the second quarter of 2021 to evaluate the safety, tolerability, and proof of mechanism of ACU193 in early AD patients. Topline data is anticipated in the **first half of 2023**[15](index=15&type=chunk)[16](index=16&type=chunk) - Acumen holds an exclusive, perpetual, irrevocable, royalty-free, worldwide license from Merck for the research, development, and commercialization of ACU193, which originated from a collaboration between 2003 and 2011[53](index=53&type=chunk)[125](index=125&type=chunk) - The company's intellectual property portfolio for ACU193, licensed from Merck, includes one issued U.S. patent and 18 issued foreign patents, which are projected to expire in **July 2031**[129](index=129&type=chunk) [Risk Factors](index=45&type=page&id=Item%201A.%20Risk%20Factors) The company faces substantial financial, development, and operational risks, including dependence on ACU193 and funding needs - The company is a clinical-stage entity with a limited operating history, no approved products, no revenue, and a net loss of **$100.6 million in 2021**. It will require substantial additional funding to continue operations[194](index=194&type=chunk)[195](index=195&type=chunk)[200](index=200&type=chunk) - The company is substantially dependent on the success of its sole product candidate, ACU193, and has concentrated its efforts in the high-risk field of Alzheimer's disease drug development, which has seen very limited success[205](index=205&type=chunk)[208](index=208&type=chunk) - The COVID-19 pandemic has caused delays in clinical site activation and patient enrollment for the INTERCEPT-AD trial and may continue to adversely affect operations[246](index=246&type=chunk)[247](index=247&type=chunk) - The company relies on contract manufacturing organizations (CMOs) for the supply of ACU193 and contract research organizations (CROs) to conduct its clinical trials, creating dependence on third-party performance[296](index=296&type=chunk)[302](index=302&type=chunk) - Intellectual property for ACU193 is licensed from Merck, and the primary patents are predicted to expire in **2031**, making the company reliant on maintaining this license and potential regulatory exclusivity[326](index=326&type=chunk)[342](index=342&type=chunk)[348](index=348&type=chunk) [Unresolved Staff Comments](index=106&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments) The company reports no unresolved staff comments from the SEC - There are no unresolved staff comments[493](index=493&type=chunk) [Properties](index=106&type=section&id=Item%202.%20Properties) The company leases corporate headquarters in Charlottesville, VA, and additional office space in Carmel, IN - The company's corporate headquarters are in a leased office in Charlottesville, VA, with the lease expiring in **December 2022**. It also leases office space in Carmel, IN, with a lease expiring in **August 2023**[494](index=494&type=chunk) [Legal Proceedings](index=106&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings - The company is not subject to any material legal proceedings[495](index=495&type=chunk) [Mine Safety Disclosures](index=106&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[496](index=496&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=107&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's common stock trades on Nasdaq under 'ABOS', with IPO proceeds used for business development and no dividends paid - The company's common stock is listed on The Nasdaq Global Select Market under the symbol '**ABOS**'[498](index=498&type=chunk) - The company raised net proceeds of **$168.6 million** from its Initial Public Offering, which closed in **July 2021**[501](index=501&type=chunk) - No cash dividends have ever been paid, and the company does not intend to pay any in the foreseeable future[499](index=499&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=108&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) The company reported a significant net loss in 2021 due to non-cash charges and increased operating expenses, with IPO proceeds providing liquidity through 2025 [Results of Operations](index=111&type=section&id=Results%20of%20Operations) The company's net loss significantly increased in 2021 due to a non-cash charge and higher operating expenses Results of Operations (in thousands) | | Year Ended December 31, | Change | | :--- | :--- | :--- | | | **2021** | **2020** | **$** | | **Grant and other revenue** | **$ —** | **$ 1,436** | **$ (1,436)** | | **Operating expenses** | | | | | Research and development | 12,305 | 7,997 | 4,308 | | General and administrative | 7,279 | 1,351 | 5,928 | | **Total operating expenses** | **19,584** | **9,348** | **10,236** | | **Loss from operations** | **(19,584)** | **(7,912)** | **(11,672)** | | **Total other income (expense)** | **(81,022)** | **587** | **(81,609)** | | **Net loss** | **(100,606)** | **(7,325)** | **(93,281)** | - Research and development expenses increased by **$4.3 million**, from **$8.0 million** in 2020 to **$12.3 million** in 2021, primarily due to increased costs for personnel, materials, and CRO services related to the initiation of the clinical trial[530](index=530&type=chunk) - General and administrative expenses increased by **$5.9 million**, from **$1.4 million** in 2020 to **$7.3 million** in 2021, mainly due to higher personnel, insurance, accounting, and legal expenses following the transition to a public company[531](index=531&type=chunk) [Liquidity and Capital Resources](index=113&type=section&id=Liquidity%20and%20Capital%20Resources) The company's liquidity from its 2021 IPO is projected to fund operations through 2025, with future funding from equity, debt, or collaborations - As of December 31, 2021, the company had cash and cash equivalents of **$122.2 million** and marketable securities of **$103.7 million**[536](index=536&type=chunk) - The company received net proceeds of **$168.6 million** from its IPO in **July 2021**[535](index=535&type=chunk) - Based on the current operating plan, existing cash and marketable securities are expected to fund operations at least through **2025**[544](index=544&type=chunk) [Critical Accounting Policies, Significant Judgments and Use of Estimates](index=115&type=section&id=Critical%20Accounting%20Policies%2C%20Significant%20Judgments%20and%20Use%20of%20Estimates) Critical accounting policies involve significant judgments and estimates for stock-based compensation, pre-IPO valuations, and accrued R&D expenses - Stock-based compensation is calculated using the Black-Scholes model, with key subjective inputs including expected volatility (based on a peer group) and expected term (using the 'simplified method')[551](index=551&type=chunk) - Prior to the IPO, the fair value of common stock was determined using complex valuation methods such as the Option Pricing Method (OPM) and a hybrid of the OPM and Probability-Weighted Expected Return Method (PWERM)[555](index=555&type=chunk)[556](index=556&type=chunk) - The company is an 'emerging growth company' and has elected to use the extended transition period for complying with new or revised accounting standards[563](index=563&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=119&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, Acumen is not required to provide this information - The company is not required to provide this information as it qualifies as a "smaller reporting company"[568](index=568&type=chunk) [Financial Statements and Supplementary Data](index=119&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section presents the company's audited financial statements and notes for 2021 and 2020 Key Balance Sheet Data (in thousands) | | Dec 31, 2021 | Dec 31, 2020 | | :--- | :--- | :--- | | Cash and cash equivalents | $122,162 | $43,777 | | Marketable securities | $103,694 | $— | | **Total Assets** | **$230,330** | **$44,429** | | **Total Liabilities** | **$5,147** | **$6,367** | | **Total Stockholders' Equity (Deficit)** | **$225,183** | **$(18,591)** | Key Statement of Operations Data (in thousands) | | Year Ended Dec 31, 2021 | Year Ended Dec 31, 2020 | | :--- | :--- | :--- | | Grant and other revenue | $— | $1,436 | | Research and development | $12,305 | $7,997 | | General and administrative | $7,279 | $1,351 | | **Loss from operations** | **$(19,584)** | **$(7,912)** | | **Net loss** | **$(100,606)** | **$(7,325)** | | **Net loss per share** | **$(5.02)** | **$(17.48)** | [Changes in and Disagreements With Accountants on Accounting and Financial Disclosure](index=151&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20With%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) The company reports no disagreements with its accountants on accounting principles, disclosures, or auditing procedures - None reported[702](index=702&type=chunk) [Controls and Procedures](index=151&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of December 31, 2021, remediating a prior material weakness - Disclosure controls and procedures were deemed effective as of **December 31, 2021**[704](index=704&type=chunk) - A previously identified material weakness in internal controls related to segregation of duties was remediated in the **second half of 2021**[706](index=706&type=chunk) [Other Information](index=152&type=section&id=Item%209B.%20Other%20Information) The board amended director compensation policy and appointed Kim C Drapkin to the board and as audit committee chair - On **March 24, 2022**, the board amended the non-employee director compensation policy to increase the initial stock option grant to **50,000 shares** and the annual grant to **25,000 shares**[711](index=711&type=chunk) - Kim C. Drapkin was appointed to the board of directors and as chair of the audit committee, effective **April 1, 2022**[712](index=712&type=chunk) Part III [Directors, Executive Officers and Corporate Governance](index=153&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance is incorporated by reference from the 2022 Proxy Statement - Information is incorporated by reference from the **2022 Proxy Statement**[716](index=716&type=chunk) [Executive Compensation](index=154&type=section&id=Item%2011.%20Executive%20Compensation) Information on executive and director compensation is incorporated by reference from the 2022 Proxy Statement - Information is incorporated by reference from the **2022 Proxy Statement**[717](index=717&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=154&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Information on security ownership and equity compensation plans is incorporated by reference from the 2022 Proxy Statement - Information is incorporated by reference from the **2022 Proxy Statement**[718](index=718&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=154&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) Information on related party transactions and director independence is incorporated by reference from the 2022 Proxy Statement - Information is incorporated by reference from the **2022 Proxy Statement**[719](index=719&type=chunk) [Principal Accountant Fees and Services](index=154&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) Information on principal accountant fees and services is incorporated by reference from the 2022 Proxy Statement - Information is incorporated by reference from the **2022 Proxy Statement**[720](index=720&type=chunk) Part IV [Exhibit and Financial Statement Schedules](index=154&type=section&id=Item%2015.%20Exhibit%20and%20Financial%20Statement%20Schedules) This section lists exhibits filed with the Form 10-K and notes the omission of financial statement schedules - The financial statements are included in Item 8, and all related schedules are omitted as inapplicable or otherwise included[720](index=720&type=chunk)[721](index=721&type=chunk) - A list of exhibits filed with the report is provided, including key corporate and compensatory agreements[721](index=721&type=chunk)[722](index=722&type=chunk) [Form 10–K Summary](index=156&type=section&id=Item%2016.%20Form%2010%E2%80%93K%20Summary) The company reports no Form 10-K summary - None[723](index=723&type=chunk)