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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): December 04, 2025

 

 

MapLight Therapeutics, Inc.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware

333-290400

83-2163243

(State or Other Jurisdiction
of Incorporation)

(Commission File Number)

(IRS Employer
Identification No.)

 

 

 

 

 

800 Chesapeake Drive

 

Redwood City, California

 

94063

(Address of Principal Executive Offices)

 

(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: 617 984-6300

 

N/A

(Former Name or Former Address, if Changed Since Last Report)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:


Title of each class

 

Trading
Symbol(s)

 


Name of each exchange on which registered

Voting Common Stock, $0.0001 par value per share

 

MPLT

 

Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

 


Item 2.02 Results of Operations and Financial Condition.On December 4, 2025, MapLight Therapeutics, Inc. (the "Company") issued a press release announcing its financial results for the third quarter ended September 30, 2025. A copy of this press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.In accordance with General Instruction B.2. of Form 8-K, the information in this Item 2.02, and Exhibit 99.1 hereto, shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference in any of the Company’s filings under the Securities Act of 1933, as amended (the "Securities Act"), or the Exchange Act, whether made before or after the date hereof, regardless of any incorporation language in such a filing, except as expressly set forth by specific reference in such a filing.Item 7.01 Regulation FD Disclosure.On December 4, 2025, the Company updated its corporate presentation for use in meetings with investors, analysts and others. The presentation is available on the Company’s website and a copy is furnished as Exhibit 99.2 to this Current Report on Form 8-K.In accordance with General Instruction B.2. of Form 8-K, the information in this Item 7.01, and Exhibit 99.2 hereto, shall not be deemed "filed" for purposes of Section 18 of the Exchange Act or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference in any of the Company’s filings under the Securities Act or the Exchange Act, whether made before or after the date hereof, regardless of any incorporation language in such a filing, except as expressly set forth by specific reference in such a filing.Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

 

Exhibit

Number

Description

99.1

 

Press Release, dated December 4, 2025

99.2

 

Corporate Presentation, dated December 4, 2025

104

 

Cover Page Interactive Data File (formatted as inline XBRL).

 

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

 

 

MapLight Therapeutics, Inc.

 

 

 

 

Date:

December 4, 2025

By:

/s/ Christopher Kroeger

 

 

 

Christopher A. Kroeger, M.D.
Chief Executive Officer

 


EX-99.1 2 mplt-ex99_1.htm EX-99.1 EX-99.1

Exhibit 99.1

MapLight Therapeutics Reports Third Quarter Financial Results and
Highlights Corporate Progress

Topline results from Phase 2 ZEPHYR trial of ML-007C-MA for schizophrenia expected in the second half of 2026
Topline results from Phase 2 VISTA trial of ML-007C-MA for Alzheimer’s disease psychosis expected in the second half of 2027
Raised $296.5 million in gross proceeds from initial public offering and concurrent private placement completed in October 2025
Cash, cash equivalents and short-term investments sufficient to fund operations through 2027

 

SAN FRANCISCO and BOSTON, Dec. 4, 2025 (GLOBE NEWSWIRE) -- MapLight Therapeutics, Inc. (Nasdaq: MPLT), a clinical-stage biopharmaceutical company focused on improving the lives of patients suffering from debilitating central nervous system disorders, today reported financial results for the third quarter of 2025 and highlighted recent progress and upcoming milestones.

“2025 was a year of exceptional execution and acceleration for MapLight,” said Chris Kroeger, Chief Executive Officer of MapLight Therapeutics. “In the last year, we have made noteworthy progress across our entire product candidate portfolio, including the initiation of the Phase 2 ZEPHYR and VISTA studies for ML-007C-MA, completion of enrollment in our Phase 2 IRIS study of ML-004 and advancement of our preclinical programs. With a strengthened balance sheet following our recent public offering, we are in a strong financial position to continue this momentum to advance our broad and diversified pipeline of potentially best-in-class therapies for CNS disorders.”

Business Highlights and Upcoming Milestones

The Company continued to make significant progress across its pipeline of novel drug candidates with potential to address a breadth of debilitating CNS disorders.

ML-007C-MA (M1/M4 Muscarinic Agonist) for the Treatment of Schizophrenia and Alzheimer’s Disease Psychosis (ADP):

 

o
Topline results from Phase 2 ZEPHYR trial for schizophrenia expected in the second half of 2026. ZEPHYR is a randomized, double-blind, placebo-controlled, three-arm trial evaluating once- and twice-daily doses of ML-007C-MA for the treatment of hospitalized adult participants with schizophrenia experiencing an acute exacerbation of psychosis. The Company expects to enroll 300 participants in the trial, and the primary endpoint is the change from baseline in Positive and Negative Syndrome Scale (PANSS) total score at Week 5.

o
Topline results from Phase 2 VISTA trial for ADP expected in the second half of 2027. VISTA is a randomized, double-blind, placebo-controlled, two-arm trial evaluating twice-daily doses of ML-007C-MA for the treatment of hallucinations and delusions associated with ADP. The Company expects to enroll 300 participants in the trial, and the primary endpoint is the change from baseline in the Neuropsychiatric Inventory - Clinician Hallucinations and Delusions (NPI-C H+D) score at Week 7.
 
ML-004 (5-HT1B/1D Agonist) for the Treatment of Autism Spectrum Disorder (ASD): The Company has completed enrollment in the IRIS Phase 2 study, with topline results expected in the second half of 2026. The IRIS study is a randomized, double-blind, placebo-controlled two-arm trial evaluating once-daily ML-004 for the improvement of core social communication deficits, with change in irritability symptoms as a key secondary endpoint. The trial randomized approximately 160 adult and adolescent participants.
 
ML-021 (M4 Antagonist) for the Treatment of Motor Deficits in Parkinson’s Disease: The Company expects to complete IND-enabling studies for ML-021 in the second half of 2026.
 
ML-009 (GPR52 Positive Allosteric Modulator) for the Treatment of Hyperactivity, Impulsivity and Agitation-Related Disorders: The Company has nominated a development candidate for advancement to IND-enabling studies.

 

Completed $296.5 Million Initial Public Offering and Private Placement: In October 2025, the Company completed its initial public offering (IPO) and concurrent private placement and sold 17,439,207 shares of common stock in aggregate (which included the full exercise of the underwriters’ option to purchase additional shares) at a price of $17.00 per share. The net proceeds to the Company were $269.8 million after deducting underwriting discounts and commissions, placement agent fees, and estimated offering expenses.

 

Third Quarter 2025 Financial Results

The Company ended the quarter with $227.2 million in cash, cash equivalents and short-term investments. The Company expects that its current cash, cash equivalents and short-term investments, together with the net proceeds from the IPO and concurrent private placement, will be sufficient to fund operations through 2027.
Research and development expenses were $27.1 million for the third quarter of 2025, compared to $16.8 million for the third quarter of 2024. The increase primarily reflects increases in clinical trial expenses, employee-related expenses and formulation and CMC expenses that were partially offset by decreases in preclinical program expenses.

General and administrative expenses were $4.4 million for the third quarter of 2025, compared to $4.1 million for the third quarter of 2024. The increase was primarily due to increases in legal and consulting fees that were partially offset by lower employee-related expenses.
Net loss was $29.4 million for the third quarter of 2025, compared to $19.0 million for the third quarter of 2024.

About MapLight Therapeutics

MapLight Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients suffering from debilitating central nervous system disorders. The company was founded by globally recognized leaders in psychiatry and neuroscience research to address the lack of circuit-specific pharmacotherapies available for patients.

For more information, please visit www.maplightrx.com.

 

Forward Looking Statements

Certain statements in this press release may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, the Company’s expectations regarding plans for and potential benefits of its current and future product candidates and programs, plans for its current and future clinical trials, plans for clinical trial design, the anticipated timing of the initiation of and results from the Company’s clinical trials and the Company’s cash, cash equivalents and short-term investments funding its operations through 2027. Words such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “develop,” “plan” or the negative of these terms, and similar expressions, are intended to identify forward-looking statements. While the Company believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to the company on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in the Company’s filings with the U.S. Securities and Exchange Commission (SEC)), many of which are beyond the Company’s control and subject to change. Actual results could be materially different. Risks and uncertainties include: global macroeconomic conditions and related volatility; expectations regarding the initiation, progress, and expected results of the Company’s preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; the risk that results obtained in any clinical trials to date may not be indicative of results obtained in ongoing or future trials; the timing or likelihood of regulatory filings and approvals; expectations regarding our ability to fund our current operations and to secure sufficient additional capital, when required, to fund product development or future commercialization efforts; and other risks and uncertainties identified in the Company’s Quarterly Report on Form 10-Q filed with the SEC on December 4, 2025, and subsequent disclosure documents the Company may file with the SEC. The Company claims the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. The Company expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law.


 

MapLight Therapeutics, Inc.

Condensed Consolidated Statements of Operations
(Unaudited)

(in thousands, except share and per share amounts)

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2025

 

 

2024

 

 

2025

 

 

2024

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

27,094

 

 

$

16,814

 

 

$

73,726

 

 

$

47,810

 

General and administrative

 

 

4,404

 

 

 

4,080

 

 

 

11,980

 

 

 

12,359

 

Total operating expenses

 

 

31,498

 

 

 

20,894

 

 

 

85,706

 

 

 

60,169

 

Loss from operations

 

 

(31,498

)

 

 

(20,894

)

 

 

(85,706

)

 

 

(60,169

)

Other income (expense), net:

 

 

 

 

 

 

 

 

 

 

 

 

Interest income

 

 

1,549

 

 

 

1,081

 

 

 

3,048

 

 

 

3,605

 

Loss from equity method investment

 

 

 

 

 

 

 

 

 

 

 

(986

)

Other income, net

 

 

531

 

 

 

797

 

 

 

1,054

 

 

 

1,199

 

Total other income, net

 

 

2,080

 

 

 

1,878

 

 

 

4,102

 

 

 

3,818

 

Net loss

 

$

(29,418

)

 

$

(19,016

)

 

$

(81,604

)

 

$

(56,351

)

Net loss per share attributable to common stockholders - basic and diluted

 

$

(37.18

)

 

$

(25.08

)

 

$

(105.70

)

 

$

(77.37

)

Weighted-average common stock outstanding - basic and diluted

 

 

791,127

 

 

 

758,204

 

 

 

772,031

 

 

 

728,343

 

 

Select Condensed Consolidated Balance Sheet Data
(Unaudited)

(in thousands)

 

 

September 30,

 

 

December 31,

 

 

 

2025

 

 

2024

 

Cash, cash equivalents and short-term investments

 

$

227,168

 

 

$

108,795

 

Total assets

 

 

257,222

 

 

 

136,916

 

Total current liabilities

 

 

18,562

 

 

 

15,920

 

Total liabilities

 

 

23,706

 

 

 

21,721

 

Total stockholder's deficit

 

 

(274,177

)

 

 

(193,628

)

 

For investor inquiries: investors@maplightrx.com

For media inquiries: media@maplightrx.com


EX-99.2 3 mplt-ex99_2.htm EX-99.2

Slide 1

Corporate Presentation December 4, 2025 Exhibit 99.2 


Slide 2

This presentation and any accompanying oral commentary have been prepared by MapLight Therapeutics, Inc. (“MapLight”, “we,” “us,” “our,” the “Company”, or similar terms) for informational purposes only and not for any other purpose. This presentation contains trademarks, service marks, trade names and copyrights of MapLight and other companies which are the property of their respective owners. This presentation discusses product candidates that are under pre-clinical and clinical study, and which have not yet been approved for marketing by the U.S. Food and Drug Administration. No representation is made as to the safety or efficacy of these product candidates for the uses for which they are being studied. Statements contained in this presentation and the accompanying oral commentary, other than statements of historical facts, may be forward-looking statements, including, but not limited to: statements about our expectations regarding the potential benefits, efficacy and safety of our product candidates and platform; our expectations with regard to the design and results of our research and development programs, preclinical studies, and clinical trials; our preclinical, clinical, and regulatory development plans for our product candidates; our expectations with regard to our ability to discover, develop, license, or acquire additional product candidates and advance such product candidates into, and successfully complete, preclinical studies and clinical trials; the potential patient populations for our product candidates and any future product candidates; and our business strategy. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expects,” “plans,” “anticipates,” “could,” “intends,” “targets,” “projects,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negative of these terms or other similar expressions. These statements involve substantial known and unknown risks, uncertainties and other factors that may cause our actual results, timing of results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include risks and uncertainties that are described in the “Risk Factors” section of our Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (“SEC”) on December 4, 2025 and other filings we make with the SEC from time to time. These documents are available under the “SEC Filings” page of the “Investors” section of our website at www.maplightrx.com. New risks emerge from time to time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially and adversely from those anticipated or implied in the forward-looking statements. We may not actually achieve the plans, intentions, or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. The forward-looking statements in this presentation represent our views as of the date of this presentation. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. Except as required by law, neither we nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements in this presentation and the accompanying oral commentary. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this presentation. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. These data involve a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. Safe Harbor and Forward-Looking Statements


Slide 3

Innovation-Driven Company Globally Recognized Scientific Founders Established in 2018 to >100 Employees Broad & Diversified Product Pipeline Differentiated M1/M4 Muscarinic Agonist Multiple Product Candidates + Discovery Platform Strong Financial Position ~$497M in Pro Forma Cash (as of 9/30)(1) Cash Runway Through 2027 Chris Kroeger, MD CEO and Founder Vish Setia Chief Financial Officer Erin Foff, MD, PhD Chief Medical Officer 3 Corporate Snapshot James Lillie, PhD Chief Scientific Officer Anatol Kreitzer, PhD Chief Discovery Officer Kristopher Hanson General Counsel Jonathan GillisChief Administrative & Accounting Officer Pro forma cash based on cash, cash equivalents and short-term investments balance of ~$227.2M (as of September 30, 2025) and net proceeds from IPO (including exercise of overallotment option) and concurrent private placement of ~$269.8M after deducting underwriting discounts and commissions, placement agent fees and offering expenses.


Slide 4

Employ Medicinal Chemistry, Pharmacokinetics and Formulation Expertise to Optimize Drug Properties Our Rational Drug Discovery Approach Seeks to Address the Lack of Neural Circuit-Specific Pharmacotherapies Explore Causal Link Between Neural Circuit Activity and Disease Symptoms Identify Potential Druggable Targets Within These Disease-Relevant Neural Circuits Validate Expression, Selectivity and Location of Potential Targets in Brain Tissue


Slide 5

Our Pipeline of Product Candidates GPR = G-protein-coupled receptor. PAC = peripherally acting anti-cholinergic. PAM = positive allosteric modulator. Leveraging Our Versatile Circuit-Based Discovery Platform for Ongoing Pipeline Expansion Program Circuit Indications Preclinical Phase 1 Phase 2 Phase 3 Anticipated Milestones ML-007C-MA M1/M4 agonist co-formulated with PAC Direct and Indirect Pathways Schizophrenia Topline results in 2H 2026 Alzheimer’s Disease Psychosis Topline results in 2H 2027 ML-004 5-HT1B/1D agonist Dorsal Raphe to Nucleus Accumbens Autism Spectrum Disorder Sociability/Irritability Topline results in 2H 2026 ML-021 M4 antagonist Direct Pathway Parkinson’s Disease IND-enabling studies complete in 2H 2026 ML-009 GPR52 PAM Indirect Pathway Hyperactivity/Impulsivity Preclinical candidate nominated ZEPHYR VISTA IRIS Potential in other indications being explored


Slide 6

ML-007C-MA Lead Asset in Development for Schizophrenia and ADP


Slide 7

Muscarinic Receptor Agonism is the First Novel MoA Approved for Treatment of Schizophrenia in Decades AE = adverse events; AD = Alzheimer’s disease; ADP = Alzheimer’s disease psychosis. Represents 2022 TRx estimates as per Symphony Health. Ringeisen H, et al. Mental and Substance Use Disorders Prevalence Study: Findings report. RTI International; 2023. Ismail et al, Nat Rev Neurol. PMC March 2022. Ropacki SA et al, Am. J. Psychiatry 162 (2005). Represents global sales estimates based on third-party market research sources, accessed November 2025. Sub-Optimal Current Standard of Care Primarily rely on D2 dopamine receptor blockade Primarily treat positive symptoms Boxed warning and risk of serious motor and metabolic AEs 64 million antipsychotics Rx in US (1) Suboptimal outcomes and low compliance rates Multiple drugs achieved >$5B peak sales, with notable uptake for late entrants with modest differentiation Modulate ACh without direct D2 dopamine receptor blockade Efficacy across positive and negative symptoms No boxed warning or existing antipsychotics’ class warnings & precautions Schizophrenia and ADP affect 5M+ people in the US (2)(3) Potential for treatment of psychosis, cognitive and motor symptoms in multiple other conditions Emergence of Novel Muscarinic Class Global Sales for Antipsychotics and Muscarinic Class Projected to Exceed $20B by 2032 (4)


Slide 8

Activity at M1 andM4 receptors Agonist / antagonistperipheral exposure mismatch Lack of M1 receptor activity Endogenous ACh required for activity for PAMs Positive & negative symptom improvement Signal of cognitive benefit Elevated rates of cholinergic AEs BID / TID dosing, with extended titration Fasting requirements Hepatic / renal warnings Lower rates of cholinergic AEs Potential for more convenient dosing, shorter titration and no food effects Efficacy in large RCTs No mechanistic rationale for cognitive benefit Cholinergic neuron degeneration in AD (PAMs) M4 Only Approaches Agonist / PAM Cobenfy (KarXT) Dual M1/M4 Agonist + Peripheral Antagonist (1)(2)(3) Differences exist among study designs, and caution should be exercised when comparing data across trials. ACh = acetylcholine. AE = adverse events. AD = Alzheimer’s disease. BID = twice daily; RCT = randomized clinical trials; TID = three times a day. Cobenfy FDA prescribing information. Horan et al, poster at ECNP Congress, Oct 2023. Pooled results from EMERGENT-2/3 trials in patients with cognitive impairment at baseline. Based on publicly disclosed study design of ongoing ADEPT-1/2 trials in ADP. Despite Recent Advances Within the Muscarinic Class, Significant Room for Further Improvement


Slide 9

Safety and Tolerability Improved Ease of Use Improvement Across Key Symptom Domains Broad IP Portfolio Expected to Provide Coverage Into 2040s (2) ML-007C-MA Novel M1/M4 Agonist + Fesoterodine(1) 9 ML-007C-MA: Our Novel M1/M4 Muscarinic Agonist Potential Areas of Differentiation Strong Activation of Both M1 & M4 Receptors Synchronized Agonist / Antagonist Exposure Rational and Deliberate Clinical Strategy Fesoterodine is indicated for the treatment of overactive bladder in adult patients with symptoms of urge urinary incontinence, urgency, and frequency. Intellectual property portfolio coverage based on latest-to-expire issued patents and provisional applications covering composition of matter for ML-007, treatment and method of use. Significant Need for a Safer and More Convenient Treatment Option With Robust M1/M4 Activation


Slide 10

Amphetamine-Induced Hyperlocomotion PCP-Induced Hyperlocomotion Conditioned Avoidance Response (2) ~13x ~9x ~10x Xanomeline ML-007 Relative Peak Intrinsic Activity (1) Xanomeline ML-007 Protein Binding Oral Bioavailability Peripheral Tissue Accumulation Extensive (incl. liver) Low Stronger Relative Potency Demonstrated in Multiple In Vivo Studies Represents ratios of ED50 for xanomeline vs. ML-007 in head-to-head preclinical models ED50 = median effective dose; PCP = phencyclidine. Represents data normalized to responses of control agonist, oxotremorine, in human GTPγS M1 and M4 in vitro assays. Model demonstrated that xanomeline at 10mg/kg demonstrated comparable effects to ML-007 at 1mg/kg. Favorable Physical & Chemical Properties Robust M1/M4 Receptor Activation M1 M4 ML-007 is a Novel Muscarinic Agonist That Has Exhibited Activity at Both M1 and M4 Receptors


Slide 11

210/3 mg BID Dose (2) 330/6 mg QD Dose (2) ML-007 PAC Ratio Dose notation refers to the co-formulated combination of ML‑007 and PAC. For example, 210/3 mg indicates 210 mg of ML‑007 and 3 mg of peripherally acting anti-cholinergic (PAC) . BID = twice daily; CV = coefficient of variation; QD = once daily. Data points show geometric mean observed data from individual study; lines represent model-simulated values. Observations after 7 days of maintenance dosing. PAC concentration of active metabolite 5-HMT. Calculated using last maintenance Day 7 dose data. Low Variability Observed at Target Doses – CV of ~30% (3) Steady State Plasma Concentrations From Study 013 (1) Close Matching of Plasma Exposures for ML-007 & PAC to Offset Peripheral Cholinergic Activity ML-007 or PAC Plasma Concentration (ng/mL) ML-007C-MA Concentration Ratio ML-007 or PAC Plasma Concentration (ng/mL) ML-007C-MA Concentration Ratio


Slide 12

Four Phase 1 Studies Completed 270 Subjects Enrolled – Healthy Adults and Elderly 1,500+ Doses of ‘007 Administered ADP = Alzheimer’s disease psychosis. BID = twice daily; MTD = maximum tolerated dose; PAC = peripherally acting anti-cholinergic. QD = once daily; TEAE = treatment-emergent adverse event. N = 58 Dose dependent target engagement with ML-007 Single agent maximum tolerated dose (MTD) achieved Pairing with PAC blocked peripheral cholinergic effects N = 106 Wide range of ML-007 to PAC ratios evaluated across 13 cohorts Identified optimal ratios to balance pro- and anti-cholinergic peripheral effects N = 24 Close matching of ML-007 and PAC peripheral exposures Low rates of TEAEs observed that were mild and transient in nature Exposure profile supported potential BID / QD dosing N = 82 First study that evaluated co-formulated bi-layer tablet Four cohort design, included two cohorts with multi-day dosing paradigm Evaluated food effects, titration and dosing in healthy adults and healthy elderly Finalized study design of Phase 2 trials in schizophrenia & ADP Co-Administered Extended Release Co-Formulated Extended Release Immediate Release Immediate Release Study 001 Study 011 Study 012 Study 013 Rational and Deliberate Clinical Development Strategy to Establish a Differentiated Profile


Slide 13

ML-007C-MA Key Areas of Potential Differentiation


Slide 14

Safety and Tolerability KarXT: Elevated Rates of Pro and Anticholinergic AEs TEAEs reported in 67-89% of subjects in Phase 1 healthy volunteer trial at doses similar to the clinically efficacious doses, with moderate TEAEs in 17-33% of subjects (1) Phase 2 and 3 trials in schizophrenia patients reported similar rates and types of AEs (2) 52-week OLE studies in schizophrenia reported discontinuation rates of ~47-77% (3) Early real-world evidence suggest tolerability challenges (e.g., lower doses, longer titrations, incidence of nausea / vomiting) (4) ML-007C-MA: Precision Matching of Agonist / Antagonist Synchronizing peripheral exposures of components designed to offer improved tolerability Rational and deliberate clinical development approach to identify optimal ratios of components Favorable safety / tolerability profile observed in healthy adults and elderly subjects to date Differences exist among study designs, and caution should be exercised when comparing data across trials. ADP = Alzheimer’s disease psychosis; AE = adverse event; OLE = open label extension; TEAE = treatment-emergent adverse event; TID = three times a day. Brannan et al, poster at ASCP 2019. Phase 1 trial with multiple ascending doses in healthy volunteers. AE rates shown across cohorts. Brannan et al, posters at NEI Congress, Nov 2023. Pooled results from EMERGENT trials. Based on FDA NDA Review for Cobenfy. Represents interim data submitted as part of 120-day update. Kutz et al, poster at Psych Congress 2025. Areas of Potential Differentiation


Slide 15

Placebo Combined (N=8) 165/3 mg BID (N=6) 210/3 mg BID (N=6) 270/6 mg QD (N=6) 330/6 mg QD (N=6) Subjects With Any TEAE (1) 2 (25%) 6 (100%) 3 (50%) 3 (50%) 4 (67%) Cholinergic Classification – Subjects (%) Procholinergic 0 (0%) 4 (67%) 3 (50%) 2 (33%) 4 (67%) Anticholinergic 1 (13%) 3 (50%) 0 (0%) 1 (17%) 0 (0%) Other 1 (13%) 5 (83%) 3 (50%) 3 (50%) 3 (50%) % of TEAEs by Severity Mild 100% 100% 95% 93% 86% Moderate 0% 0% 5% 7% 14% Severe 0% 0% 0% 0% 0% Study 013: Safety and TolerabilityHealthy Adults (Non-Elderly) – All Doses Evaluated BID = twice daily; ECG = electrocardiogram; QD = once daily; TEAE = treatment-emergent adverse event. AEs include events occurring after administration of target dose until 24 hours after last dose. Procedural AEs are excluded. Reported as N (%). Phase 2 Target Doses for Schizophrenia 1-4 dose titration, followed by dosing at target doses for 7 days Generally favorable safety and tolerability observed, with mostly mild and transient TEAEs No serious or severe TEAEs Low rates of anticholinergic TEAEs at target doses No clinically meaningful changes in mean blood pressure or ECG parameters observed No liver-related findings observed


Slide 16

Study 013: Safety and TolerabilityHealthy Adults (Non-Elderly) – At Target Doses in Ongoing Phase 2 Schizophrenia Trial BID = twice daily; QD = once daily; TEAE = treatment-emergent adverse event. AEs include events occurring after administration of target dose until 24 hours after last dose. Procedural AEs are excluded. Reported as N (%). Placebo arm reported three AEs, including two episodes of headache. One 330/6 mg participant discontinued; after mild AEs and one moderate AE (dyspepsia), the investigator supported continuation, but the participant withdrew consent. Placebo Combined (N = 8) (2) 210/3 mg BID (N = 6) 330/6 mg QD (N = 6) (3) Subjects With Any TEAE (1) 2 (25%) 3 (50%) 4 (67%) Mild 2 (25%) 3 (50%) 4 (67%) Moderate (3) 0 (0%) 1 (17%) 2 (33%) Severe 0 0 0 Most Common TEAEs (>1 Subject in any Dose Group) Chills 0 0 2 (33%) Hyperhidrosis 0 1 (17%) 2 (33%) Nausea 0 3 (50%) 3 (50%) Dizziness 0 3 (50%) 2 (33%) Dyspepsia 0 0 2 (33%) At the target doses for Phase 2 schizophrenia trial: Most TEAEs (~91%) were mild and transient in nature Low rates of moderate TEAEs –nausea (N=1), dyspepsia (N=2) No severe or serious TEAEs No episodes of anticholinergic TEAEs No episodes of vomiting or constipation


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KarXT: Translation of Tolerability Profile From Healthy Volunteers to Schizophrenia Patients Types of AEs and % of moderate AE shows high translatability from healthy subjects to schizophrenia patients TEAEs in Schizophrenia Trials (% of Subjects) – Represents Highest Severity (2) TEAEs in Healthy Adults (% of Subjects) – Represents Highest Severity (1) Sources: “Xanomeline plus trospium: A novel strategy to enhance pro-muscarinic efficacy and mitigate peripheral side effects” poster presented at ASCP 2019. Based on FDA NDA Review for Cobenfy. Mild Moderate Mild Moderate Severe Generally consistent or better tolerability profile in schizophrenia patients than healthy volunteers based on % of subjects with observed AEs 17


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Study 013: Safety and TolerabilityHealthy Elderly – All Doses Evaluated Placebo Combined (N=7) (2) 165/3 mg BID (with 2d titration) (N=6) 210/3 mg BID (with 2-7d titration) (N=11) (3) 330/6 mg QD(with 7d titration) (N=6) (4) Subjects with Any TEAE (1) 5 (71%) 4 (67%) 8 (73%) 6 (100%) Cholinergic Classification – Subjects (%) Procholinergic 1 (14%) 2 (33%) 6 (55%) 6 (100%) Anticholinergic 1 (14%) 1 (17%) 4 (36%) 0 Other 3 (43%) 3 (50%) 8 (73%) 6 (100%) % of TEAEs by Severity Mild 100% 100% 93% 72% Moderate (3) 0% 0% 7% 28% Severe 0% 0% 0% 0% Phase 2 Target Dose for ADP ADP = Alzheimer’s disease psychosis; BID = twice daily; ECG = electrocardiogram; QD = once daily; TEAE = treatment-emergent adverse event. AEs include events occurring after administration of target dose until 24 hours after last dose. Procedural AEs are excluded. Reported as N (%). One participant (placebo) discontinued from the study due to an AE during titration; one participant (placebo) had an AE during maintenance dosing that led to study drug withdrawal. Two participants had their dose reduced to 165/3 mg BID after experiencing AEs; one additional participant was discontinued from the study for non-compliance with clinic rules. Four participants had AEs that led to withdrawal of study drug and one additional participant had their dose reduced to 270/6 mg QD due to AEs. 2-7 day titration period, followed by dosing at target doses for 7 days Generally favorable safety and tolerability with BID dosing, with mostly mild and transient TEAEs Highest dose given QD was not well tolerated – potential to explore lower QD doses Low rates of anticholinergic TEAEs No clinically meaningful changes in mean blood pressure or ECG parameters observed No liver-related findings observed


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Study 013: Safety and TolerabilityHealthy Elderly – At Target Dose in Ongoing Phase 2 Alzheimer’s Disease Psychosis Trial ADP = Alzheimer’s disease psychosis; BID = twice daily; TEAE = treatment-emergent adverse event. AEs include events occurring after administration of target dose until 24 hours after last dose. Procedural AEs are excluded. Reported as N (%). Two participants discontinued study drug (placebo) due to AEs. Two participants on 210/3 mg BID dose reduced to 165/3 mg BID after experiencing AEs. Placebo CombinedN = 7 (2) 210/3 mg BID  (with 2-7d titration) N = 11 (2) Subjects with Any TEAE (1) 5 (71%) 8 (73%) Mild 5 (71%) 8 (73%) Moderate 0 2 (18%) Severe 0 0 Most Common TEAEs (>2 Subjects) Hyperhidrosis 0 3 (27%) Nausea 0 3 (27%) Dizziness 1 (14%) 3 (27%) Headache 0 4 (36%) Tremor 0 3 (27%) At the target dose for Phase 2 ADP trial: Most TEAEs (~93%) were mild and transient in nature Low rates of moderate TEAEs No severe or serious TEAEs No episodes of urinary retention Limited episodes of vomiting or constipation


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Improved Ease of Use KarXT: Dosing Requirements to Manage Tolerability Profile (1)(2) Dosing: BID in schizophrenia. TID in ADP Titration: 3-8 days in schizophrenia. 5 weeks in ADP Fasting: required in both schizophrenia and ADP (elderly patients) ML-007C-MA: Ease of Use for Patients and Care Providers Dosing: QD & BID in schizophrenia and BID in ADP Titration: minimal titration (1 dose for schizophrenia, 1 week for ADP) Fasting: not required; expect dosing in normal fed state Differences exist among study designs, and caution should be exercised when comparing data across trials. ADP = Alzheimer’s disease psychosis; BID = twice daily; QD = once daily; TID = three times a day. Based on publicly disclosed study designs for EMERGENT-1/2/3 trials in schizophrenia. Based on publicly disclosed study designs for ADEPT-1/2/4/5 trials in ADP. Areas of Potential Differentiation


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21 Study 013: Planned Phase 2 Doses Achieved CSF Exposure At Or Above Target Levels AIH = amphetamine-induced hyperlocomotion; BID =twice daily; CSF = cerebrospinal fluid; ED50 = median effective dose; PCP = phencyclidine-induced hyperlocomotion. QD = once daily. ML-007 lines represents modeled estimates for CSF exposure based on CSF sampling conducted during Study 013. ML-007 was tested in multiple animal models. CSF exposures were correlated with pharmacodynamic effects to define a target efficacious range of 14 to 27 ng/mL, based on the AIH and PCP models, respectively. 210/3 mg BID Dose 330/6 mg QD Dose Modeled Steady State CSF Concentrations From Study 013 (1) ML-007 CSF Concentration (ng/mL) ML-007 CSF Concentration (ng/mL) ML-007 Cavg ~120 ng/mL ML-007 Cavg ~140 ng/mL ED50 PCP (27 ng/mL) 2 ED50 AIH (14 ng/mL) 2 ED50 PCP (27 ng/mL) 2 ED50 AIH (14 ng/mL) 2


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Improvement Across Key Symptom Domains KarXT: Cognitive Benefit Trend in Exploratory Analyses (1) Xanomeline showed clinically meaningful cognitive benefit in Phase 2 AD trial (2) ML-007C-MA: Potential Improvement Across Symptom Domains ML-007 has demonstrated robust M1/M4 activity (vs. xanomeline) in vitro and in vivo Both M1 and M4 receptors play significant roles in reduction of positive and negative symptoms in preclinical models of schizophrenia ML-007’s pro-cognitive potential demonstrated in a mouse model of Alzheimer’s disease Differences exist among study designs, and caution should be exercised when comparing data across trials. ACh = acetylcholine; AD = Alzheimer’s disease; ADP = Alzheimer’s disease psychosis; PAM = positive allosteric modulator. Horan et al, poster at ECNP Congress, Oct 2023. Pooled results from EMERGENT-2/3 trials in patients with cognitive impairment at baseline. Veroff AE et al, PubMed 9876958, Dec 1998. Woolf NJ. et al, Neurobiology Learn Mem, Nov 1996 M4 Specific Approaches: No Mechanistic Rationale for Cognitive Benefit Lack of M1 activity may impact broader efficacy as reported in recent competitor updates PAM approach requires endogenous ACh – reduced in neurodegenerative diseases like ADP (3) Areas of Potential Differentiation


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23 ML-007 Dose Response Xanomeline Dose Response AIH = Amphetamine-induced hyperlocomotion. M1KO = M1 knock-out. M4KO = M4 knock-out. WT = wild type. M1 and M4 Muscarinic Receptors Play a Significant and Complementary Role in Reduction of Symptoms ML-007 Demonstrated Stronger Agonism at Both M1 and M4 Receptors Relative to Xanomeline Head-to-Head Pharmacodynamic Efficacy in AIH Models


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ns. ns. ns. * Vehicle ML-007 Xanomeline * ‘Familiar’ ‘Novel’ 24 Spatial Memory Social Memory * Significantly different from vehicle (p<0.05). ITI = inter-trial interval. Source: Comparison of 3 mg/kg xanomeline with 0.3 mg/kg ML- 007 in age-matched (10-13 months old) TG2576 cohorts. N=14-16 per arm, 10 min pretreatment time. ML-007 Showed an Improvement in Memory in a Transgenic Mouse Model of Alzheimer’s Disease 5 min 5 min 30 min ITI Familiar Novel 10 min Habituation (Hab) i.p. drug injection 10 min Social Memory (SM) 10 min Social Interaction (SI) i.p. drug injection


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ML-007C-MA Clinical Development


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26 Establish Efficacy / Safety in Key Indications Explore and Pursue Additional Indications Parallel development plans in schizophrenia and ADP Phase 2 studies designed to be adequate and well controlled Data-driven clinical design and execution strategy designed to mitigate placebo response Prioritization of key registration-enabling activities Broad potential in multiple indications, including: Cognition in Alzheimer’s disease Psychoses in other neurodegenerative conditions Bipolar disorder Dyskinesias Autism spectrum disorder Our Development Strategy for ML-007C-MA


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27 Trial Type Double-blind, placebo-controlled trial Double-blind, placebo-controlled trial Location US only, multicenter (25 sites) Global, multicenter (100 sites) Participants Hospitalized adult patients with schizophrenia Sample size = 300 Subjects with psychosis associated with Alzheimer’s disease Sample size = 300 Primary Endpoint Change in PANSS score at 5 weeks vs. baseline Change in NPI-C H+D scores at 7 weeks vs. baseline Treatment Groups 1:1:1 randomization: placebo, 210/3mg BID and 330/6mg QD Option to drop down: 165/3mg BID or 270/6mg QD 1:1 randomization: placebo and 210/3 mg BID Option to drop down: 105/1.5mg BID Topline Results Expected in second half of 2026 Expected in second half of 2027 Phase 2 in Schizophrenia Phase 2 in ADP Design of Ongoing Phase 2 Studies ADP = Alzheimer’s disease psychosis; BID = twice daily; QD = once daily; PANSS = Positive and Negative Syndrome Scale; NPI-C H+D = Neuropsychiatric Inventory Clinician, Hallucinations and Delusions.


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Strategies to Mitigate Placebo Response in Ongoing Phase 2 Schizophrenia Study Limited number of sites (25) Single geography - US only Extensive vetting of sites using: Quality metrics for raters in recent acute schizophrenia studies Recent enrollment in positive acute schizophrenia studies 1:1 interviews with PIs Input from external consultants Individual site enrollment gated based on data quality from initial participants Limited number of participants (N=300) Single study with conservative enrollment metrics to avoid pushing enrollment too fast Participants required to complete a 3-step robust eligibility review process Duplicate patient exclusion process Target population must have moderate-to-severe symptoms at screening and baseline Excludes participants with >20% improvement from screening to baseline In-sourced site relationship and data quality oversight responsibilities with in-house CRA / monitoring program Robust quality monitoring during study conduct (independent central review, regular cadence of blinded data analytics) Placebo response training to all participant facing site staff Expectancy bias education to participants CRO selected based on track record in recent schizophrenia studies 28 PI = Principal investigator. Similar Strategies Employed in the Ongoing Phase 2 VISTA Study for ADP Study Conduct Site Selection Enrollment


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ML-004 Autism Spectrum Disorder (ASD)


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30 Core symptoms: Impaired social communication Restricted, repetitive thoughts and behaviors Comorbid neurobehavioral symptoms include: Irritability and aggression (in 25% of ASD patients) Hyperactivity, mood lability Affects ~1.8M children & adolescents and >5M adults in US No FDA approved treatments for core symptoms of ASD Only widely accepted intervention is long-term behavioral therapy (30-40 hours / week) Off label usage of ADHD medications, SSRIs, SNRIs, and supplements Two atypical antipsychotics approved for ASD-associated irritability Risk of serious side effects Ineffective in treating core social features Dysregulated serotonin signaling implicated in ASD pathology ML-004 – novel IR/ER oral formulation of zolmitriptan (5-HT1B/1D agonist) Zolmitriptan approved for acute treatment of migraines Safety, tolerability and pharmacokinetics evaluated in Phase 1 trials Ongoing Phase 2 trial to evaluate efficacy and safety in ASD patients ASD Overview Unmet Medical Need ML-004 Potential ML-004: Potential Treatment of Social Communication Deficits and Irritability in ASD Patients ASD = autism spectrum disorder; ADHD = attention-deficit/hyperactivity disorder; SSRI = selective serotonin reuptake inhibitors; SNRI = serotonin-norepinephrine reuptake inhibitors. Source: US CDC prevalence estimates.


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Multi-center, randomized, double-blind, placebo-controlled Randomized approximately 160 subjects with ASD Adolescent (age 12-17) and adult (age 18-45) Flexible dosing paradigm, with target maintenance dose of 48mg and 72mg Primary Endpoint: ABI, Social Communication Domain Score Change vs. baseline assessed after 12 weeks of maintenance dosing Secondary Endpoint: Change in ABC-I score vs. baseline for patients with moderate or greater irritability score at baseline Other secondary and exploratory endpoints Patients eligible to participate in 52-week OLE trial following completion of Phase 2 trial 31 ABI - Autism Behavior Inventory, ABC-I - Aberrant Behavior Checklist-Irritability; ASD = autism spectrum disorder. OLE = open label extension. ML-004: Ongoing Global Phase 2 Trial in ASD


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Potential for a well tolerated and convenient option without sacrificing strong M1/M4 activation Broad development strategy, including parallel advancement in schizophrenia and ADP Versatile and reproducible discovery platform leveraging our deep understanding of circuit biology Extensive medicinal chemistry and formulation expertise ML-004 in Phase 2 trial for treatment of sociability and / or irritability symptoms of ASD Expect to continue advancing early-stage pipeline Seasoned team with experience in drug development and commercialization Strong financial position with cash runway through key milestones Differentiated M1/M4 Muscarinic Agonist Broad and Diversified Product Pipeline 32 Circuit-Specific Discovery Strategy Established Track Record of Execution Corporate Summary