# Praxis Precision Medicines, Inc.: Form 10-Q for the period ended Sep 30, 2023

- Company: [Praxis Precision Medicines, Inc.](/companies/prax.md) (CIK 1689548)
- Form: 10-Q
- Filed: 2023-11-07
- Accepted by EDGAR: Nov 7, 2023, 07:37 ET
- Period: 2023-09-30
- Accession: 0001689548-23-000159
- Inline XBRL: yes
- Main document: https://www.sec.gov/Archives/edgar/data/1689548/000168954823000159/prax-20230930.htm
- All documents and exhibits: https://www.sec.gov/Archives/edgar/data/1689548/000168954823000159/

## Figures reported in this filing

Standardized XBRL line items for the period ended 2023-09-30.

| Line item | Period | Value | XBRL concept |
| --- | --- | ---: | --- |
| Revenue | 3 months, 2023-07-01 to 2023-09-30 | $468.0k | us-gaap:Revenues |
| Revenue | 9 months, 2023-01-01 to 2023-09-30 | $1.9m | us-gaap:Revenues |
| Research and development | 3 months, 2023-07-01 to 2023-09-30 | $17.3m | us-gaap:ResearchAndDevelopmentExpense |
| Research and development | 9 months, 2023-01-01 to 2023-09-30 | $68.4m | us-gaap:ResearchAndDevelopmentExpense |
| Operating income | 3 months, 2023-07-01 to 2023-09-30 | −$25.5m | us-gaap:OperatingIncomeLoss |
| Operating income | 9 months, 2023-01-01 to 2023-09-30 | −$98.6m | us-gaap:OperatingIncomeLoss |
| Net income | 3 months, 2023-07-01 to 2023-09-30 | −$24.6m | us-gaap:NetIncomeLoss |
| Net income | 9 months, 2023-01-01 to 2023-09-30 | −$96.4m | us-gaap:NetIncomeLoss |
| Diluted EPS | 3 months, 2023-07-01 to 2023-09-30 | −$0.18 | us-gaap:EarningsPerShareDiluted |
| Diluted EPS | 9 months, 2023-01-01 to 2023-09-30 | −$1.12 | us-gaap:EarningsPerShareDiluted |
| Basic EPS | 3 months, 2023-07-01 to 2023-09-30 | −$0.18 | us-gaap:EarningsPerShareBasic |
| Basic EPS | 9 months, 2023-01-01 to 2023-09-30 | −$1.12 | us-gaap:EarningsPerShareBasic |
| Diluted shares (weighted) | 3 months, 2023-07-01 to 2023-09-30 | 135,591,429 shares | us-gaap:WeightedAverageNumberOfDilutedSharesOutstanding |
| Diluted shares (weighted) | 9 months, 2023-01-01 to 2023-09-30 | 86,447,174 shares | us-gaap:WeightedAverageNumberOfDilutedSharesOutstanding |
| Operating cash flow | 9 months, 2023-01-01 to 2023-09-30 | −$87.3m | us-gaap:NetCashProvidedByUsedInOperatingActivities |
| Capital expenditure | 9 months, 2023-01-01 to 2023-09-30 | $0 | us-gaap:PaymentsToAcquirePropertyPlantAndEquipment |
| Cash and equivalents | at 2023-09-30 | $101.1m | us-gaap:CashAndCashEquivalentsAtCarryingValue |
| Current assets | at 2023-09-30 | $103.3m | us-gaap:AssetsCurrent |
| Total assets | at 2023-09-30 | $106.7m | us-gaap:Assets |
| Current liabilities | at 2023-09-30 | $16.8m | us-gaap:LiabilitiesCurrent |
| Total liabilities | at 2023-09-30 | $20.0m | us-gaap:Liabilities |
| Shareholders' equity | at 2023-09-30 | $86.8m | us-gaap:StockholdersEquity |

## Management's discussion and analysis

As filed, 39,964 characters.

*You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K, which was filed with the Securities and Exchange Commission (the "SEC") on February 7, 2023. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Risk Factors” sections of our Annual Report on Form 10-K for the year ended December 31, 2022, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 and this Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.*

### Overview

We are a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for central nervous system, or CNS, disorders characterized by neuronal excitation-inhibition imbalance. Normal brain function requires a delicate balance of excitation and inhibition in neuronal circuits, which, when dysregulated, can lead to abnormal function and both rare and more prevalent neurological disorders. We are applying genetic insights to the discovery and development of therapies for neurological disorders through two proprietary platforms, using our understanding of shared biological targets and circuits in the brain. Each platform currently has multiple programs, with significant potential for additional program and indication expansion:

- ***Cerebrum™***, our small molecule platform, utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies
- ***Solidus™***, our antisense oligonucleotide, or ASO, platform, is an efficient, targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology

Our platforms utilize a deliberate, pragmatic and patient-guided approach, leveraging a suite of translational tools, including novel transgenic and predictive translational animal models and electrophysiology markers, to enable an efficient path to proof-of-concept in patients. Through this approach, we have established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy. For our most advanced product candidate under the Cerebrum™ platform, ulixacaltamide (also known as PRAX-944), topline results from the Phase 2b Essential1 clinical trial in essential tremor, or ET, were announced in the first quarter of 2023. We initiated our Phase 3 studies in ET in the fourth quarter of 2023, with enrollment expected to complete in the first half of 2024 and topline results expected in the second half of 2024. We initiated the PRAX-562 Phase 2 EMBOLD study in the first quarter of 2023 and expect to announce topline results in the first half of 2024. We also announced positive results from our PRAX-628 Phase 1 study in May 2023. In June 2023, we initiated a Phase 2 proof of concept study evaluating PRAX-628 in patients with a Photo Paroxysmal Response, or PPR, with results anticipated in the fourth quarter of 2023. Upon completion of the PPR study, we plan to initiate a Phase 2 study in focal epilepsy in the first half of 2024. For our most advanced product candidate under the Solidus™ platform, elsunersen (also known as PRAX-222), we completed dosing in the first cohort of the EMBRAVE study and announced preliminary data from the first dose cohort in October 2023.

We were incorporated in 2015 and commenced operations in 2016. Since inception, we have devoted substantially all of our resources to developing our preclinical and clinical product candidates, building our intellectual property, or IP, portfolio, business planning, raising capital and providing general and administrative support for these operations. We employ a “virtual” research and development model, relying heavily upon external consultants, collaborators, contract development and manufacturing organizations and contract research organizations, or CROs, to conduct our preclinical and clinical activities. Since inception, we have financed our operations primarily with proceeds from the sale and issuance of equity securities.

We are a development stage company and we have not generated any revenue from product sales, and do not expect to do so for several years, if at all. All of our product candidates are still in preclinical and clinical development. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our product candidates, if approved. We have incurred recurring operating losses since inception, including a net loss of $96.4 million for the nine months ended September 30, 2023. As of September 30, 2023, we had an accumulated deficit of $627.0 million. We expect to incur significant expenses and operating losses for the foreseeable future as we expand our research and development activities. In addition, our losses from operations may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities. We anticipate that our expenses will be maintained or increased in connection with our ongoing activities, as we:

- advance our lead product candidate, ulixacaltamide, to a late stage clinical trial for the treatment of ET;
- advance our PRAX-562 product candidate in the EMBOLD clinical trial;
- advance our PRAX-222 product candidate in the EMBRAVE clinical trial;
- advance our PRAX-628 product candidate;
- advance our preclinical candidates to clinical trials;
- further invest in our pipeline;
- further invest in our manufacturing capabilities;
- seek regulatory approval for our product candidates;
- maintain, expand, protect and defend our IP portfolio;
- acquire or in-license technology;
- establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval; and
- when needed, increase our headcount to support our development efforts and any future commercialization efforts.

As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions. We may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses or when or if we will be able to achieve or maintain profitability. Even if we are able to generate product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

As of September 30, 2023, we had cash and cash equivalents of $101.1 million. We believe that our cash and cash equivalents as of September 30, 2023 will enable us to fund our operating expenses and capital expenditures into the first quarter of 2025. We have based this analysis on our current financial needs and ongoing research and development plans, which are limited to advancing product candidates through, but not beyond, their current clinical trials. We have based our assessment on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect. See “—Liquidity and Capital Resources.”

### Financial Operations Overview

### Revenue

We have not generated any revenue from the sale of products since inception and do not expect to generate any revenue from the sale of products for several years, if at all. As discussed in Note 7 to our condensed consolidated financial statements, we entered into an Option and License Agreement, or the Collaboration Agreement, with UCB Biopharma SRL, or UCB, in December 2022. We recognized $0.5 million and $1.9 million of collaboration revenue from the Collaboration Agreement during the three and nine months ended September 30, 2023, respectively.

### Operating Expenses

#### Research and Development Expenses

The nature of our business and primary focus of our activities generate a significant amount of research and development costs. Research and development expenses represent costs incurred by us for the following:

- costs to develop our IP portfolio;
- discovery efforts leading to development candidates;
- clinical development costs for our product candidates; and
- costs to develop our manufacturing technology and infrastructure.

The costs above comprise the following categories:

- personnel-related expenses, including salaries, benefits and stock-based compensation expense;
- expenses incurred under agreements with third parties, such as consultants, investigative sites and CROs, that conduct our preclinical and clinical studies and in-licensing arrangements;
- costs incurred to maintain compliance with regulatory requirements;
- costs incurred with third-party contract development and manufacturing organizations to acquire, develop and manufacture materials for preclinical and clinical studies; and
- depreciation, amortization and other direct and allocated expenses, including rent and other operating costs, such as information technology, incurred as a result of our research and development activities.

We expense research and development costs as incurred. We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors and our clinical investigative sites. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our condensed consolidated balance sheets as prepaid expenses or accrued expenses. Non-refundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized, even when there is no alternative future use for the research and development. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

As a company operating in a virtual environment, a significant portion of our research and development costs have been external costs. We track direct external research and development expenses to specific platforms and product candidates upon commencement. Due to the number of ongoing studies and our ability to use resources across platforms, indirect or shared operating costs incurred for our research and development platforms, such as personnel, facility costs and certain consulting costs, are not recorded or maintained on a platform-specific basis.

The following table reflects our research and development expenses, including direct expenses summarized by platform and indirect or shared operating costs recognized as research and development expenses during each period presented (in thousands):

|  | Three Months Ended September 30, 2023 | 2022 | Nine Months Ended September 30, 2023 | 2022 |
| --- | ---: | ---: | ---: | ---: |
| Cerebrum™ | $5,832 | $16,293 | $23,857 | $76,720 |
| Solidus™ | 2,806 | 3,655 | 16,330 | 13,883 |
| Personnel-related (including stock-based compensation) | 6,840 | 8,714 | 21,744 | 30,590 |
| Other indirect research and development expenses | 1,782 | 1,777 | 6,447 | 5,518 |
| Total research and development expenses | $17,260 | $30,439 | $68,378 | $126,711 |

Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will be maintained or increase in the foreseeable future as we advance our product candidates through the development phase, and as we continue to discover and develop additional product candidates, build manufacturing capabilities and expand into additional therapeutic areas.

At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from sales or licensing of our product candidates. This is due to the numerous risks and uncertainties associated with drug development, including the uncertainty of:

- our ability to add and retain key research and development personnel;
- the timing and progress of preclinical and clinical development activities;
- the number and scope of preclinical and clinical programs we decide to pursue;
- our ability to successfully complete clinical trials with safety, tolerability and efficacy profiles that are satisfactory to the FDA or any comparable foreign regulatory authority;
- our ability to successfully develop, obtain regulatory approval for, and then successfully commercialize, our product candidates;
- our successful enrollment in and completion of clinical trials;
- the costs associated with the development of any additional product candidates we identify in-house or acquire through collaborations;
- our ability to discover, develop and utilize biomarkers to demonstrate target engagement, pathway engagement and the impact on disease progression of our product candidates;
- our ability to establish and maintain agreements with third-party manufacturers for clinical supply for our clinical trials and commercial manufacturing, if our product candidates are approved;
- the terms and timing of any collaboration, license or other arrangement, including the terms and timing of any milestone payments thereunder;
- our ability to obtain and maintain patent, trade secret and other IP protection and regulatory exclusivity for our product candidates, if approved;
- our receipt of marketing approvals from applicable regulatory authorities;
- our ability to commercialize products, if approved, whether alone or in collaboration with others; and
- the continued acceptable safety profiles of our product candidates.

A change in any of these variables with respect to the development of any of our product candidates would significantly change the costs, timing and viability associated with the development of that product candidate. For example, if the FDA or another regulatory authority were to delay our planned start of clinical trials or require us to conduct clinical trials or other testing beyond those that we currently expect, or if we experience significant delays in enrollment in any of our planned clinical trials, we could be required to expend significant additional financial resources and time to complete our clinical development activities. We may never obtain regulatory approval for any of our product candidates. Drug commercialization will take several years and require significant development costs.

#### General and Administrative Expense

General and administrative expenses consist primarily of personnel-related costs, including salaries, benefits and stock-based compensation, for personnel in our executive, finance, legal, commercial and administrative functions. General and administrative expenses also include legal fees relating to corporate matters; professional fees for accounting, auditing, tax and administrative consulting services; commercial-related costs to support market assessments and scenario planning; insurance costs; administrative travel expenses; and facility-related expenses, which include direct depreciation costs and allocated expenses for office rent and other operating costs, such as information technology. Costs to secure and defend our IP are expensed as incurred and are classified as general and administrative expenses. These costs relate to the operation of the business and are unrelated to the research and development function or any individual platform or product candidate.

We anticipate that our general and administrative expenses may increase in the future as we increase our headcount, when needed, to support the expected growth in our research and development activities and the potential commercialization of our product candidates. We also expect to incur additional IP-related expenses as we file patent applications to protect innovations arising from our research and development activities.

### Other Income

#### Other Income, Net

Other income, net consists of interest income from our cash, cash equivalents and marketable securities and amortization of investment premiums and discounts.

### Income Taxes

Since our inception, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or for our earned research and development tax credits due to our uncertainty of realizing a benefit from those items. Income taxes are determined at the applicable tax rates adjusted for non-deductible expenses, research and development tax credits and other permanent differences. Our income tax provision may be significantly affected by changes to our estimates. There was no income tax provision recognized for the three and nine months ended September 30, 2023 and 2022.

### Results of Operations

### Comparison of the Three Months Ended September 30, 2023 and 2022

The following table summarizes our condensed consolidated statements of operations for each period presented (in thousands):

|  | Three Months Ended September 30, 2023 | 2022 | Change |
| --- | ---: | ---: | ---: |
| Collaboration revenue | $468 | $— | $468 |
| Operating expenses: |  |  |  |
| Research and development | 17,260 | 30,439 | (13,179) |
| General and administrative | 8,724 | 13,851 | (5,127) |
| Total operating expenses | 25,984 | 44,290 | (18,306) |
| Loss from operations | (25,516) | (44,290) | 18,774 |
| Other income: |  |  |  |
| Other income, net | 884 | 345 | 539 |
| Total other income | 884 | 345 | 539 |
| Net loss | $(24,632) | $(43,945) | $19,313 |

#### Collaboration Revenue

The $0.5 million increase in collaboration revenue is associated with the revenue recorded as research services are provided and costs are incurred under the Collaboration Agreement with UCB that was executed in December 2022.

#### Research and Development Expense

The following table summarizes our research and development expenses for each period presented, along with the changes in those items (in thousands):

|  | Three Months Ended September 30, 2023 | 2022 | Change |
| --- | ---: | ---: | ---: |
| Cerebrum™ | $5,832 | $16,293 | $(10,461) |
| Solidus™ | 2,806 | 3,655 | (849) |
| Personnel-related (including stock-based compensation) | 6,840 | 8,714 | (1,874) |
| Other indirect research and development expenses | 1,782 | 1,777 | 5 |
| Total research and development expenses | $17,260 | $30,439 | $(13,179) |

The $13.2 million decrease in research and development expenses was primarily attributable to the following:

- $10.5 million decrease in expense related to our Cerebrum™ platform, driven primarily by:
- $4.2 million decrease in spend for our ulixacaltamide program, primarily due to prior year Phase 1 spending and decreased activity related to our Essential1 study, partially offset by costs related to Essential3 study start up activities;
- $2.9 million decrease in clinical-related spend for our PRAX-114 program due to our strategic realignment in the second quarter of 2022;
- $1.4 million decrease in spend for our PRAX-562 program, primarily related to prior year manufacturing spend and Phase 1 trial spend, partially offset by current quarter costs related to the EMBOLD Phase 2 clinical trial;
- $1.4 million decrease in activities for our earlier stage assets; and
- $0.6 million decrease in clinical-related spend for our PRAX-628 Phase 1 clinical trial.
- $0.8 million decrease in expense related to our Solidus™ platform, primarily related to prior year PRAX-222 toxicology spend and clinical study spend, partially offset by current quarter spend for our EMBRAVE Phase 2 clinical trial; and
- $1.9 million decrease in personnel-related costs due to decreased headcount.

#### General and Administrative Expense

The $5.1 million decrease in general and administrative expenses was primarily attributable to the following:

- $2.5 million decrease in personnel costs due to decreased headcount;
- $1.9 million decrease in consulting expenses and professional fees, and
- $0.7 million decrease in other general and administrative expenses.

#### Other Income

Other income for the three months ended September 30, 2023 was comprised of interest income on our cash and cash equivalents. Other income for the three months ended September 30, 2022 was comprised of interest income on our cash, cash equivalents and marketable securities and investment premium and discount amortization.

### Comparison of the Nine Months Ended September 30, 2023 and 2022

The following table summarizes our condensed consolidated statements of operations for each period presented (in thousands):

|  | Nine Months Ended September 30, 2023 | 2022 | Change |
| --- | ---: | ---: | ---: |
| Collaboration revenue | $1,932 | $— | $1,932 |
| Operating expenses: |  |  |  |
| Research and development | 68,378 | 126,711 | (58,333) |
| General and administrative | 32,121 | 46,822 | (14,701) |
| Total operating expenses | 100,499 | 173,533 | (73,034) |
| Loss from operations | (98,567) | (173,533) | 74,966 |
| Other income: |  |  |  |
| Other income, net | 2,168 | 677 | 1,491 |
| Total other income | 2,168 | 677 | 1,491 |
| Net loss | $(96,399) | $(172,856) | $76,457 |

#### Collaboration Revenue

The $1.9 million increase in collaboration revenue is associated with the revenue recorded as research services are provided and costs are incurred under the Collaboration Agreement with UCB that was executed in December 2022.

#### Research and Development Expense

The following table summarizes our research and development expenses for each period presented, along with the changes in those items (in thousands):

|  | Nine Months Ended September 30, 2023 | 2022 | Change |
| --- | ---: | ---: | ---: |
| Cerebrum™ | $23,857 | $76,720 | $(52,863) |
| Solidus™ | 16,330 | 13,883 | 2,447 |
| Personnel-related (including stock-based compensation) | 21,744 | 30,590 | (8,846) |
| Other indirect research and development expenses | 6,447 | 5,518 | 929 |
| Total research and development expenses | $68,378 | $126,711 | $(58,333) |

The $58.3 million decrease in research and development expenses was primarily attributable to the following:

- $52.9 million decrease in expense related to our Cerebrum™ platform, driven primarily by:
- $29.0 million decrease in clinical-related spend for our PRAX-114 program due to our strategic realignment in the second quarter of 2022;
- $11.6 million decrease in spend for our PRAX-562 program primarily related to prior year manufacturing spend and Phase 1 trial spending, partially offset by costs for our EMBOLD Phase 2 clinical trial in 2023;
- $9.1 million decrease in spend for our ulixacaltamide program, primarily due to prior year Phase 2a and Phase 1 trial spend, prior year manufacturing costs and decreased spend in the current year related to our Essential1 trial, partially offset by startup costs related to our Essential3 study;
- $5.4 million decrease in activities for our earlier stage assets due to prioritization of our clinical-stage programs; and
- $2.2 million increase in clinical-related spend for our PRAX-628 program driven by our Phase 1 clinical trial and our Phase 2 PPR clinical trial.
- $8.8 million decrease in personnel-related costs due to decreased headcount;
- $2.4 million increase in expense related to our Solidus™ platform, primarily driven by costs associated with the initiation of the PRAX-222 EMBRAVE study in 2023 and associated milestone payment earned by Ionis Pharmaceuticals, Inc., or Ionis, partially offset by manufacturing expenses incurred in the prior year and a $2.0 million license fee earned by Ionis in 2022; and
- $0.9 million increase in indirect expenses, none of which were individually significant.

#### General and Administrative Expense

The $14.7 million decrease in general and administrative expenses was primarily attributable to the following:

- $6.8 million decrease in professional fees and consulting expenses;
- $5.6 million decrease in personnel-related costs due to decreased headcount; and
- $2.3 million decrease in other general and administrative expenses.

#### Other Income

Other income for the nine months ended September 30, 2023 and 2022 was comprised of interest income on our cash, cash equivalents and marketable securities and investment premium and discount amortization.

### Liquidity and Capital Resources

### Sources of Liquidity

Since our inception, we have incurred significant losses in each period. We have not yet commercialized any of our product candidates, which are in various phases of preclinical and clinical development, and we do not expect to generate revenue from sales of any products for several years, if at all.

To date, we have financed our operations primarily with proceeds from the issuance of redeemable convertible preferred stock and from the sale of common stock through an initial public offering, follow-on public offerings and at-the-market offerings under our shelf registration statement. From inception through September 30, 2023, we have raised $613.6 million in aggregate cash proceeds from such transactions, net of issuance costs. As of September 30, 2023, we had cash and cash equivalents of $101.1 million.

In November 2021, we entered into an Open Market Sale Agreement, or the Sales Agreement, with Jefferies LLC, or Jefferies, to provide for the offering, issuance and sale of up to an aggregate amount of $125.0 million of common stock from time to time in at-the-market offerings for which Jefferies acted as sales agent. We terminated the Sales Agreement in June 2023. During the nine months ended September 30, 2023, we issued and sold 14,291,937 shares under the sales agreement for aggregate net proceeds of $24.1 million. We issued and sold a total of 18,279,207 shares under the Sales Agreement for aggregate net proceeds of $40.7 million after deducting commissions and offering expenses payable by us.

On June 21, 2023, we completed a public offering of: (i) an aggregate of 64,449,690 shares of common stock at a public offering price of $0.95 per share, including the underwriters' full exercise of their option to purchase 9,299,690 additional shares of common stock, and (ii) pre-funded warrants to purchase 7,050,000 shares of common stock at a public offering price of $0.9499 per share. The purchase prices per share for each pre-funded warrant represents the per share offering price for the common stock, less the $0.0001 per share exercise price for each underlying share. Total net proceeds generated from the offering were approximately $63.4 million, after deducting underwriting discounts, commissions and other offering expenses payable by us.

### Cash Flows

The following table provides information regarding our cash flows for each period presented (in thousands):

| Net cash (used in) provided by: | Nine Months Ended September 30, 2023 | 2022 |
| --- | ---: | ---: |
| Operating activities | $(87,275) | $(156,237) |
| Investing activities | 39,000 | 74,295 |
| Financing activities | 87,745 | 5,078 |
| Net increase (decrease) in cash, cash equivalents and restricted cash | $39,470 | $(76,864) |

#### Operating Activities

Our cash flows from operating activities are greatly influenced by our use of cash for operating expenses and working capital requirements to support our business. We have historically experienced negative cash flows from operating activities as we have invested in developing our portfolio, drug discovery efforts and related infrastructure. The cash used in operating activities resulted primarily from our net losses adjusted for non-cash charges and changes in operating assets and liabilities, which are primarily the result of increased expenses and timing of vendor payments.

During the nine months ended September 30, 2023, net cash used in operating activities of $87.3 million was primarily due to our $96.4 million net loss and $11.0 million in changes in operating assets and liabilities primarily related to decreases in accrued expenses and accounts payable, partially offset by $20.1 million of non-cash charges primarily related to stock-based compensation.

During the nine months ended September 30, 2022, net cash used in operating activities of $156.2 million was primarily due to our $172.9 million net loss and $7.3 million in changes in operating assets and liabilities primarily related to a decrease in accrued expenses, partially offset by $23.9 million of non-cash charges primarily related to stock-based compensation.

#### Investing Activities

During the nine months ended September 30, 2023, net cash provided by investing activities of $39.0 million was primarily related to maturities of marketable securities.

During the nine months ended September 30, 2022, net cash provided by investing activities of $74.3 million was primarily related to maturities of marketable securities, partially offset by purchases of marketable securities.

#### Financing Activities

During the nine months ended September 30, 2023, net cash provided by financing activities of $87.7 million consisted primarily of net proceeds from at-the-market offerings and our June 2023 follow-on public offering.

During the nine months ended September 30, 2022, net cash provided by financing activities of $5.1 million consisted of net proceeds from at-the-market offerings of $4.3 million and proceeds from purchases of common stock under our employee stock purchase plan and from the exercise of stock options, partially offset by the payment of issuance costs for our at-the-market offerings and the payment of taxes related to the vesting of restricted stock units.

### Plan of Operation and Future Funding Requirements

We expect our expenses to increase substantially in connection with our ongoing research and development activities, particularly as we advance the preclinical activities and clinical trials of our product candidates. As a result, we expect to incur substantial operating losses and negative operating cash flows for the foreseeable future. We anticipate that our expenses will increase substantially if and as we:

- advance the clinical development of our clinical-stage product candidates within our Cerebrum™ and Solidus™ platforms;
- advance the development of any additional product candidates;
- conduct research and continue preclinical development of potential product candidates;
- make strategic investments in manufacturing capabilities;
- maintain our IP portfolio and opportunistically acquire complementary IP;
- seek to obtain regulatory approvals for our product candidates;
- potentially establish a sales, marketing and distribution infrastructure and scale-up manufacturing capabilities to commercialize any products for which we may obtain regulatory approval;
- when needed, add clinical, scientific, operational, financial and management information systems and personnel, including personnel to support our product development and potential future commercialization efforts and to support our operations as a public company; and
- experience any delays or encounter any issues with any of the above, including but not limited to failed studies, complex results, safety issues or other regulatory challenges.

We are unable to estimate the exact amount of our working capital requirements, but based on our current operating plan, we believe that our cash and cash equivalents as of September 30, 2023 will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2025. However, we have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

Because of the numerous risks and uncertainties associated with product development and potential collaborations with third parties for the development of our product candidates, we may incorrectly estimate the timing and amounts of increased capital outlays and operating expenses associated with completing the research and development of our product candidates. Our funding requirements and timing and amount of our operating expenditures will depend on many factors, including, but not limited to:

- the scope, progress, results and costs of preclinical studies and clinical trials for our platforms and product candidates;
- the number and characteristics of product candidates and technologies that we develop or may in-license;
- the costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval;
- the costs necessary to obtain regulatory approvals, if any, for products in the United States and other jurisdictions, and the costs of post-marketing studies that could be required by regulatory authorities in jurisdictions where approval is obtained;
- the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our IP rights and defending any IP-related claims;
- the continuation of our existing licensing arrangements and entry into new collaborations and licensing arrangements;
- the costs we incur in maintaining business operations;
- the costs associated with being a public company;
- the revenue, if any, received from commercial sales of any product candidates for which we receive marketing approval;
- the effect of competing technological and market developments; and
- the extent to which we acquire or invest in businesses, products and technologies, including entering into licensing or collaboration arrangements for product candidates, although we currently have no commitments or agreements to complete any such acquisitions or investments in businesses.

Identifying potential product candidates and conducting preclinical testing and clinical trials is a time consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially available for many years, if ever. Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.

Adequate additional funds may not be available to us on acceptable terms, or at all. We do not currently have any committed external source of funds. Market volatility could also adversely impact our ability to access capital as and when needed. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of holders of our common stock. Additional debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends and may require the issuance of warrants, which could potentially result in dilution to the holders of our common stock.

If we raise additional funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit or terminate our product development programs or any future commercialization efforts or grant rights to develop and market product candidates to third parties that we would otherwise prefer to develop and market ourselves.

### Critical Accounting Policies and Significant Judgments and Estimates

Our management’s discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of these condensed consolidated financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.

There have been no changes to our critical accounting policies from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations – Critical Accounting Policies and Significant Judgments and Estimates” included in our Annual Report on Form 10-K filed with the SEC on February 7, 2023, other than as disclosed in Note 2 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q.

### Recently Issued Accounting Pronouncements

We have reviewed all recently issued standards and have determined that, other than as disclosed in Note 2 to our condensed consolidated financial statements appearing elsewhere in this Quarterly Report on Form 10-Q, such standards will not have a material impact on our condensed consolidated financial statements or do not otherwise apply to our current operations.

## Market risk

As filed, 716 characters.

We are exposed to market risk related to changes in interest rates. Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates, particularly because cash, cash equivalents and marketable securities we may hold at any time may be in the form of money market funds or marketable debt securities or may be invested in U.S. Treasury and U.S. government agency obligations. However, because of the short-term nature and low risk profile of the instruments in our portfolio at any given time, an immediate change in market interest rates of 100 basis points would not have a material impact on our financial position or results of operations.

## Controls and procedures

As filed, 2,190 characters.

### Management’s Evaluation of Our Disclosure Controls and Procedures

We maintain “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the "Exchange Act") as controls and other procedures of an issuer that are designed to ensure that information required to be disclosed by the issuer in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by the issuer in the reports that it files or submits under the Exchange Act is accumulated and communicated to the issuer's management, including its principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. Our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and our management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.

Our management, with the participation of our Chief Executive Officer and Chief Financial Officer (our principal executive officer and principal financial officer, respectively), evaluated the effectiveness of our disclosure controls and procedures as of September 30, 2023. Based on the evaluation of our disclosure controls and procedures as of September 30, 2023, our Chief Executive Officer and Chief Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at the reasonable assurance level.

### Changes in Internal Control Over Financial Reporting

There were no changes in our internal control over financial reporting that occurred during the period covered by this Quarterly Report on Form 10-Q that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

## Risk factors

As filed, 9,487 characters.

The risk factors set forth below update, and should be read in conjunction with, the risk factors previously disclosed in the section entitled "Risk Factors" of our Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on February 7, 2023 and in the sections entitled "Risk Factors" of our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2023 and June 30, 2023 filed with the SEC on May 11, 2023 and August 9, 2023, respectively.

#### Risks Related to Preclinical and Clinical Development

***Interim, topline and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data.***

From time to time, we may publicly disclose preliminary or topline data from our clinical trials, which are based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a more comprehensive review of the data related to the particular study or trial. We also make assumptions, estimations, calculations and conclusions as part of our analyses of data, and we may not have received or had the opportunity to fully and carefully evaluate all data. As a result, the topline or preliminary results that we report may differ from future results of the same studies, or different conclusions or considerations may qualify such results, once additional data have been received and fully evaluated. Topline or preliminary data also remain subject to audit and verification procedures that may result in the final data being materially different from the topline or preliminary data we previously reported. As a result, topline and preliminary data should be viewed with caution until the final data are available.

From time to time, we disclose interim data from our preclinical studies and clinical trials. Interim data from clinical trials that we complete are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patient data become available. Adverse changes between interim data and final data could significantly harm our business and prospects. Further, additional disclosure of interim data by us or by our competitors in the future could result in volatility in the price of our common stock.

Further, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the value of the particular program, the approvability or commercialization of the particular product candidate and our company in general. In addition, the information we choose to publicly disclose regarding a particular study or clinical trial is typically selected from a more extensive amount of available information. Others may not agree with what we determine is the material or otherwise appropriate information to include in our disclosure, and any information we determine not to disclose may ultimately be deemed significant with respect to future decisions, conclusions, views, activities or otherwise regarding a particular product candidate or our business. If the interim, preliminary or topline data that we report differ from later, final or actual results, or if others, including the FDA and comparable foreign regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize our product candidates may be harmed, which could harm our business, financial condition, results of operations and prospects.

#### Risks Related to our Reverse Stock Split

***The reverse stock split may not increase the market price per share of our common stock meaningfully or at all, and any such increase may not be sustained or may be insufficient to generate increased investor interest, maintain liquidity for our stockholders, or maintain our market capitalization.***

On November 6, 2023, our stockholders approved amendments to our Amended and Restated Certificate of Incorporation to effect a reverse stock split to all of the outstanding shares of our common stock at a ratio ranging from any whole number between 1-for-5 and 1-for-25, or the reverse stock split, subject to our board of directors’ authority to elect, without further action on the part of our stockholders, whether to effect the reverse stock split and, if so, to determine the reverse stock split ratio from among the approved range. While we anticipate that the reverse stock split will result in an increase in the market price per share of our common stock, we cannot be assured that such increase will occur or, if it does, that such increase will result in any permanent or sustained increase in the market price of our common stock, which is dependent upon many factors, including our business and financial performance, general market conditions and our prospects for future success.

Even assuming an increase in the market price per share of our common stock occurs as a result of the reverse stock split, there is no guarantee that such increase will enhance the level of investment in our common stock by institutional investors or increase analyst and broker interest in our company. In addition, it is possible that the reverse stock split may adversely affect our stockholders’ liquidity by reducing the number of shares outstanding after the reverse stock split, particularly if the price of our common stock does not increase as a result of the reverse stock split, or if it does not increase in proportion to the reduction in the number of shares of our common stock outstanding immediately before the reverse stock split. The reduction in the number of outstanding shares may lead to reduced trading and a smaller number of market makers for our common stock, thus reducing our stockholders’ liquidity.

Furthermore, should the per share market price of our common stock fail to increase in proportion to the reverse stock split ratio, or should the overall value of our common stock decline, after the reverse stock split, then the value of the shares of our common stock held by our stockholders will also decrease. In some cases, the per-share stock price of companies that have effected reverse stock splits subsequently declined back to pre-reverse split levels, and accordingly, it cannot be assured that the total market value of our common stock will remain the same after the reverse stock split is effected.

Any of the foregoing would have an adverse effect on our common stock and could adversely affect our business.

***The reverse stock split may not result in more normalized trading in our common stock or decrease the market volatility of our common stock and could result in increased trading costs for our stockholders.***

Reverse stock splits can be viewed negatively by the market and it is difficult to predict how the market will respond to a reverse stock split of our common stock. Due to the smaller number of shares of our common stock that will be outstanding after the reverse stock split, trading in our common stock may decrease and, due to the decrease in trading volume, smaller trades in our common stock could result in increased volatility as compared to the trading volume and volatility of our common stock prior to the reverse stock split.

The reverse stock split will also increase the number of stockholders who own “odd lots” of less than 100 shares of our common stock. A purchase or sale of less than 100 shares (an “odd lot” transaction) may result in incrementally higher trading costs through certain brokers, particularly “full service” brokers. Therefore, those stockholders who own fewer than 100 shares of our common stock following the reverse stock split may be required to pay higher transaction costs if they sell their shares of our common stock.

***The authorized shares of common stock that will become available as a result of the reverse stock split may not be sufficient to fulfill our current and anticipated corporate purposes.***

Because the reverse stock split will decrease the number of outstanding shares of our common stock by a ratio in the range of 1-for-5 to 1-for-25 but would not effect a decrease to the number of shares of common stock that we are authorized to issue, the reverse stock split will result in a relative increase in the number of unissued shares of our common stock. Such relative increase in the number of unissued shares of our common stock may not be sufficient to fulfill our current and anticipated corporate purposes, including financing activities, public or private offerings of common stock, issuance of options and other equity awards pursuant to our incentive plans, and strategic business development transactions. If the increase in the number of unissued shares of our common stock generated by the reverse stock split proves insufficient to fulfill our corporate purposes, we may need to delay corporate activities and incur additional expenses in order to obtain stockholder approval to increase our authorized shares of common stock, or we may have to abandon such activities entirely. Accordingly, we may be prevented from acting promptly, or at all, to fulfill our corporate purposes, which could adversely affect our business and prospects.

Filed Nov 7, 2023.
Source: SEC EDGAR, the filing index for accession 0001689548-23-000159. Last ingested by Listed: Nov 5, 2026, 14:01 ET. No prices; not investment advice.