UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
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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).
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Item 2.02 Results of Operations and Financial Condition.
On November 8, 2021, Eliem Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the quarter ended September 30, 2021. A copy of the press release is attached hereto as Exhibit 99.1.
The information in this Item and the exhibit attached hereto are being furnished and shall not be deemed “filed” for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall they be deemed incorporated by reference into any filing under the Exchange Act or the Securities Act of 1933, as amended, whether filed before or after the date hereof and regardless of any general incorporation language in such filing.
Item 7.01 Regulation FD Disclosure.
A copy of a slide presentation that the Company will use at investor conferences and presentations is attached to this Current Report as Exhibit 99.2 and is incorporated herein solely for purposes of this Item 7.01 disclosure.
The information in this Item 7.01 (including Exhibit 99.2) is being furnished, not filed, pursuant to Regulation FD. Accordingly, the information in this Item 7.01 will not be incorporated by reference into any registration statement filed by the Company under the Securities Act of 1933, as amended, unless specifically identified therein as being incorporated therein by reference. The furnishing of the information in this Item 7.01 is not intended to, and does not, constitute a determination or admission by the Company that this information is material or complete, or that investors should consider this information before making an investment decision with respect to any security of the Company.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
Exhibit Number |
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Description |
99.1 |
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Press release of Eliem Therapeutics, Inc., dated November 8, 2021 |
99.2 |
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104 |
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Cover Page Interactive Data File (embedded within the Inline XBRL document) |
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1
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 thereunto duly authorized.
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Eliem Therapeutics, Inc. |
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Date: November 8, 2021 |
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By: |
/s/ Robert W. Azelby |
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Robert W. Azelby |
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Chief Executive Officer |
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Exhibit 99.1
Eliem Therapeutics Reports Third Quarter Financial and Business Highlights
Advanced ETX-155 clinical development program, with the first subject successfully screened in epilepsy proof-of-concept trial and significant progress made toward the initiation of major depressive disorder (MDD) and perimenopausal depression (PMD) clinical trials
Continued to enroll ETX-810’s two Phase 2a chronic pain clinical trials, with topline data anticipated in the first half of 2022
On track to progress Kv7.2/3 channel opener program into Investigational New Drug (IND)-enabling studies in the first half of 2022
SEATTLE and CAMBRIDGE, UK, --(BUSINESS WIRE) – November 8, 2021 – Eliem Therapeutics, Inc. (Nasdaq: ELYM), a clinical-stage biotechnology company focused on developing novel therapies for neuronal excitability disorders to address unmet needs in chronic pain, psychiatry, epilepsy and other disorders of the peripheral and central nervous systems, today reported financial results and business highlights for the quarter ended September 30, 2021.
“Our clinical execution is progressing well,” said Bob Azelby, Eliem’s president and chief executive officer. “For ETX-155, we are excited to report that we have completed our Phase 1 studies, we have successfully screened our first patient in our photosensitive epilepsy (PSE) proof-of-concept clinical trial and we continue to progress clinical development activities for the launch of our phase 2a trials evaluating ETX-155 in patients with MDD and PMD. As we look to expand our clinical pipeline, we are increasingly excited about the potential of our Kv7.2/3 channel opener program as a clinically validated mechanistic approach to treat diseases such as epilepsy, pain and MDD, and we remain on track to progress the program into IND-enabling studies in the first half of 2022.”
Third Quarter 2021 Highlights and Recent Developments
Completed 14-day repeat dose Phase 1 study demonstrating ETX-155 was well tolerated with an approximate 40-hour half-life supporting once-daily dosing. The 14-day, repeat dose, Phase 1 study evaluated the pharmacokinetic profile and safety of ETX-155 in 20 healthy human subjects, evaluating 60 mg ETX-155 (n=15) or placebo (n=5) dosed daily in the evening for 14 days. Results demonstrated ETX-155 reached steady state concentration at Day 8 and had an approximate 40-hour half-life, confirming ETX-155’s desirable profile for a once-daily dosing regimen. The study also confirmed that ETX-155 was generally well tolerated with no severe or serious adverse events, or discontinuations. All treatment-emergent adverse events (TEAEs), including central nervous system (CNS) adverse events, were mild/moderate and transient. In particular, all somnolence adverse events were mild and the incidence was comparable in the ETX-155 and placebo groups. Notably, somnolence events were sporadic, and no subject who reported somnolence in either the ETX-155 or placebo arms reported it more than once during the dosing or follow-up period. In addition, there was no clinically meaningful
difference compared to placebo in sleep quality or next morning state of arousal, as measured by the Leeds Sleep Evaluation Questionnaire. The tolerability and safety findings of this study were consistent with those of the previous 7-day repeat dose and single ascending dose Phase 1 study. Collectively, the Company’s Phase 1 studies have demonstrated that ETX-155 has differentiated pharmacokinetic properties, including no clinically meaningful food effect and an approximate 40-hour half-life to enable a once-daily dosing regimen.
Screened the first subject in ETX-155 photosensitive epilepsy clinical trial: The Company anticipates dosing the first subject in the single-arm proof-of-concept Phase 1b PSE trial by the end of 2021. Precedent literature demonstrates that activity in single-dose PSE trials can be a reliable predictor of anticonvulsant activity in various forms of epilepsy, such as focal onset seizure.
Advanced study start-up activities for ETX-155 Phase 2a clinical trials in MDD and PMD: The Company anticipates dosing the first subject in each of these studies in early 2022, assuming regulatory approval of its IND application.
Program Updates and Anticipated Milestones
ETX-810 in chronic pain: ETX-810 is a novel new chemical entity prodrug of the bioactive lipid palmitoylethanolamide that is currently being evaluated in two Phase 2a clinical trials in subjects with diabetic peripheral neuropathic pain (DPNP) and lumbosacral radicular pain (LSRP), commonly referred to as sciatica.
ETX-155 in depression and epilepsy: ETX-155 is a novel GABAA receptor positive allosteric modulator (PAM) that Eliem plans to evaluate in subjects with MDD, PMD and focal onset seizure (FOS).
Kv7.2/3 channel opener program: The Company’s preclinical program targets the Kv7.2/3 potassium channel that has been shown to control neuronal excitability, with clinical validation in pain and epilepsy. The program remains on track to progress to IND-enabling studies in the first half of 2022.
Anxiolytic for generalized anxiety disorder (GAD): The Company is also in early preclinical development of a novel, rapid-acting, non-sedating, non-addictive anxiolytic for the potential treatment of GAD, based on a clinically validated mechanism. The Company plans to continue the preclinical development of this program in 2022.
Third Quarter 2021 Financial Results
About Eliem Therapeutics, Inc.
Eliem Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing novel therapies for neuronal excitability disorders to address unmet needs in chronic pain, psychiatry, epilepsy and other disorders of the peripheral and central nervous systems. These disorders often occur when neurons are overly excited or inhibited, leading to an imbalance, and our focus is on restoring homeostasis. We are developing a pipeline of clinically differentiated product candidates focused on validated mechanisms of action with broad therapeutic potential to deliver improved therapeutics for patients with these disorders. Eliem channels its experience, energy, and passion for improving patients’ quality of life to fuel our efforts to develop life-changing novel therapies. At its core, the Eliem team is motivated by the promise of helping patients live happier, more fulfilling lives.
https://eliemtx.com/
Forward-Looking Statements
This press release contains forward-looking statements, including, without limitation, statements relating to: the continued development and clinical and therapeutic potential ETX-155 and ETX-810; Eliem’s plans to initiate clinical trials of ETX-155 and the timing thereof; anticipated data readouts of ETX-810 and ETX-155 and the timing thereof; the progression of the Kv7.2/3 and next-generation anxiolytic preclinical programs; the expectation that Eliem’s current cash, cash equivalents and marketable securities will fund operations through late 2023; and Eliem’s commitment to developing therapies targeting debilitating disorders. Words such as “on track,” “advance,” “progress,” “toward,” “continue,” “excited,” “potential,” “expand,” “anticipate,” “milestones,” “expect,” “demonstrates,” “intended,” “plans,” “runway,” “initiate,” “support,” “enable,” or other similar expressions, identify forward-looking statements, but the absence of these words does not necessarily mean that a statement is not forward-looking. In addition, any statements that refer to expectations, projections or other characterizations of future events or circumstances are forward-looking statements. The forward-looking statements in this press release are based upon Eliem's current plans, assumptions, beliefs, expectations, estimates and projections, and involve substantial risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in the forward-looking statements due to these risks and uncertainties as well as other factors, which include, without limitation: the clinical, therapeutic and commercial value of ETX-810, ETX-155 and Eliem’s preclinical
programs; risks related to the potential failure of ETX-810 and ETX-155 to demonstrate safety and efficacy in clinical testing; Eliem’s ability to initiate and conduct clinical trials and studies of ETX-810 and ETX-155 sufficient to achieve a positive completion; the availability of data at the expected times; Eliem's ability to obtain and protect intellectual property rights, and operate without infringing on the intellectual property rights of others; the uncertain timing and level of expenses associated with Eliem's preclinical and clinical development activities; the sufficiency of Eliem's capital and other resources; risks and uncertainties related to Eliem's compliance with applicable legal and regulatory requirements; market competition; changes in economic and business conditions; impacts on Eliem’s business due to health pandemics or other contagious outbreaks, such as the current COVID-19 pandemic; and other factors discussed under the caption "Risk Factors" in Eliem's Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2021. This filing, when available, is available on the SEC's website at www.sec.gov. Additional information will also be set forth in Eliem's other reports and filings it will make with the SEC from time to time. The forward-looking statements made in this press release speak only as of the date of this press release. Eliem expressly disclaims any duty, obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Eliem's expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.
Investors
Chris Brinzey
ICR Westwicke
chris.brinzey@westwicke.com
339-970-2843
Media
Marites Coulter
Verge Scientific
Mcoulter@vergescientific.com
415.819.2214
Eliem Therapeutics, Inc.
Condensed Consolidated Balance Sheets
(In thousands, except share and per share amounts)
(unaudited)
Assets |
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September 30, 2021 |
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December 31, 2020 |
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Current assets: |
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Cash and cash equivalents |
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$ |
62,819 |
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$ |
20,487 |
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Short-term marketable securities |
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83,199 |
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— |
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Prepaid expenses and other current assets |
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12,614 |
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1,511 |
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Total current assets |
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$ |
158,632 |
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$ |
21,998 |
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Long-term marketable securities |
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23,619 |
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— |
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Other long-term assets |
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— |
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2,633 |
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Total assets |
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$ |
182,251 |
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$ |
24,631 |
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Liabilities, Redeemable Convertible Preferred Stock, and |
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Current liabilities: |
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Accounts payable |
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2,579 |
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1,086 |
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Accounts payable, related party |
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— |
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207 |
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Accrued expenses |
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2,979 |
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1,219 |
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Accrued expenses, related party |
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32 |
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— |
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Redeemable convertible preferred stock tranche liability |
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— |
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551 |
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Total current liabilities |
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$ |
5,590 |
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$ |
3,063 |
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Total liabilities |
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$ |
5,590 |
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$ |
3,063 |
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Commitments and contingencies |
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Redeemable convertible preferred stock, $0.0001 par value, |
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— |
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46,551 |
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Stockholders’ equity (deficit): |
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Common stock, $0.0001 par value per share, 250,000,000 and |
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3 |
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1 |
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Additional paid-in capital |
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241,747 |
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3,152 |
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Accumulated other comprehensive income |
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(18 |
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— |
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Accumulated deficit |
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(65,071 |
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(28,136 |
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Total stockholders’ equity (deficit) |
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$ |
176,661 |
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$ |
(24,983 |
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Total liabilities, redeemable convertible preferred stock, |
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$ |
182,251 |
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$ |
24,631 |
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Eliem Therapeutics, Inc.
Condensed Consolidated Statements of Operations
(In thousands, except share and per share amounts)
(unaudited)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2021 |
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2020 |
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2021 |
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2020 |
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Operating expenses: |
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Research and development |
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$ |
5,704 |
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$ |
1,930 |
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$ |
15,455 |
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$ |
4,644 |
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Research and development, related party |
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285 |
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17 |
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988 |
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286 |
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General and administrative |
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3,394 |
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312 |
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8,526 |
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888 |
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Total operating expenses |
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9,383 |
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2,259 |
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24,969 |
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5,818 |
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Loss from operations |
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(9,383 |
) |
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(2,259 |
) |
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(24,969 |
) |
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(5,818 |
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Other income (expense): |
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Change in fair value of redeemable convertible preferred stock tranche liability |
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— |
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— |
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(11,718 |
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— |
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Foreign currency gain (loss) |
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(252 |
) |
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1 |
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(268 |
) |
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13 |
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Other income, net |
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20 |
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— |
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20 |
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— |
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Total other income (expense) |
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(232 |
) |
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1 |
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(11,966 |
) |
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13 |
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Net loss |
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$ |
(9,615 |
) |
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$ |
(2,258 |
) |
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$ |
(36,935 |
) |
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$ |
(5,805 |
) |
Accretion of redeemable convertible preferred stock to redemption value and cumulative preferred stock dividends |
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(1,322 |
) |
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(461 |
) |
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(4,548 |
) |
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(1,352 |
) |
Net loss attributable to common stockholders |
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$ |
(10,937 |
) |
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$ |
(2,719 |
) |
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$ |
(41,483 |
) |
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$ |
(7,157 |
) |
Net loss per share attributable to common stockholders, basic and diluted |
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$ |
(0.70 |
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$ |
(1.46 |
) |
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$ |
(5.49 |
) |
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$ |
(3.85 |
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Weighted-average number of shares outstanding used to compute net loss per share attributable to common stockholders, basic and diluted |
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15,585,611 |
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1,863,860 |
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7,554,300 |
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1,859,713 |
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Clinical Stage Neurology Company Focused on Neuronal Excitability Disorders Corporate Presentation | November 2021
Forward-Looking Statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this presentation, including statements regarding our future financial condition, results of operations, business strategy and plans, and objectives of management for future operations, as well as statements regarding industry trends, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially,” “predict,” “should,” “will” or the negative of these terms or other similar expressions. We have based these forward-looking statements largely on our current expectations and projections about future events and trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, among other things, risks related to: the success, cost and timing of our product development activities and clinical trials; our expectations about the timing of achieving regulatory approval and the cost of our development programs; our ability to obtain funding for our operations, including funding necessary to complete further development and commercialization of our product candidates; the impact of the COVID-19 pandemic on our operations; the commercialization of our product candidates, if approved; our plans to research, develop and commercialize our product candidates; our plans to develop additional product candidates; our ability to obtain, maintain, expand, protect and enforce our intellectual property rights; our ability to operate our business without infringing, misappropriating or otherwise violating the intellectual property rights of third parties; our ability to attract collaborators with development, regulatory and commercialization expertise; future agreements with third parties in connection with the commercialization of our product candidates; the size and growth potential of the markets for our product candidates and our ability to serve those markets; the rate and degree of market acceptance of our product candidates; regulatory developments in the United States and foreign countries; our ability to contract with third-party suppliers and manufacturers and their ability to perform adequately; the success of competing products that are or may become available; and our ability to attract and retain key scientific or management personnel. These risks are not exhaustive. New risk factors emerge from time to time and it is not possible for our management to predict all risk factors, 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 from those contained in, or implied by, any forward-looking statements. More information about the risks and uncertainties faced by Eliem is contained under the caption “Risk Factors” set forth in Eliem’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, which is available on the SEC’s website at www.sec.gov, and in other subsequent reports and filings Eliem will make with the SEC from time to time. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. Except as required by law, we undertake no obligation to update publicly any forward-looking statements for any reason after the date of this presentation. Certain information contained in this presentation relates to, or is based on, studies, publications, surveys and other data obtained from third-party sources and the Company's own internal estimates and research. While we believe these third-party sources to be reliable as of the date of this presentation, we have not independently verified, and make no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, all of the market data included in this presentation involves a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of such assumptions. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. These and other factors could cause results to differ materially from those expressed in the estimates made by the independent parties and by us. Finally, while we believe our own internal research is reliable, such research has not been verified by any independent source. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this presentation, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and investors are cautioned not to unduly rely upon these statements.
Highly experienced management team Clinical and preclinical pipeline based on clinically validated mechanisms of action Two clinically differentiated lead product candidates with top-line data readouts across five indications over next 24 months ~$170M* cash runway to late 2023 allows for top line data readouts and advancement of preclinical assets Rethinking treatment for nervous system disorders * Cash, cash equivalents and marketable securities as of September 30, 2021
Powered by Successful and Talented Executives from Pioneering Organizations General Management, Commercial & Corporate Development Research & Development Valerie Morisset, PhD EVP R&D and Chief Scientific Officer Joanne Palmer, PhD Chief Development Officer Amy Chappell, MD Chief Medical Officer Robert Azelby, MBA Chief Executive Officer Erin Lavelle Chief Operating Officer & Chief Financial Officer James Bucher, JD EVP and General Counsel Deep expertise in neuroscience research, clinical development and commercialization Cymbalta, Lamictal, Neurontin, Lyrica, Trobalt, Vyepti, Vixotrigine Leadership experience in both large pharma and small biotech Large: Amgen, GSK, Novartis, Biogen, Lilly Small: Alder, Juno, Convergence, Exelixis Highly skilled in public/private capital raising and corporate development with successful exits Exits: Alder, Convergence, Juno, Immunomedics, Cascadian
Eliem is Developing Novel Therapies With Multiple Opportunities to Address Interrelated Diseases Approaching interrelated disease states with multiple MOAs Innovating within clinically validated mechanisms of action Multiple “pipeline-in-a-product” opportunities ETX-810 ETX-155 Kv7 Chronic Pain Epilepsy ETX-155 Kv7 Depression / Anxiety ETX-155 Kv7 Next-gen anxiolytic
Eliem Pipeline: Four Programs with Clinically Validated MOAs and Multiple Upcoming Clinical Catalysts Product Candidate (Mechanism) Lead indications Preclinical Phase 1 Phase 2 Anticipated Clinical Milestones ETX-810 (PEA prodrug) Diabetic peripheral neuropathic pain Topline Phase 2a data (1H 2022) Lumbosacral radicular pain (sciatica) Topline Phase 2a data (1H 2022) ETX-155 (GABAA receptor PAM) Major depressive disorder (MDD) Topline Phase 2a data (1H 2023) Perimenopausal depression (PMD) Topline Phase 2a data (1H 2023) Focal onset seizure (FOS) Phase 1b photosensitive epilepsy data (1H 2022) Kv7 Program (Kv7.2/3 channel opener) Pain, Epilepsy, Depression Next Gen Anxiolytic (2,3-benzo) Generalized anxiety disorder (GAD) PEA: palmitoylethanolamide GABAA PAM: GABAA receptor positive allosteric modulator
ETX-810 Anticipated Milestones Diabetic Peripheral Neuropathic Pain (DPNP) Phase 2a Data 1H 2022 Lumbosacral Radicular Pain (LSRP) Phase 2a Data 1H 2022
Chronic Pain: Large Commercial Opportunity with High Unmet Need ETX-810 Current Treatment Paradigm ETX-810 has opportunity to be preferred 2nd line monotherapy or used in combination <50% of patients achieve ≥50% reduction in pain significant residual pain Significant tolerability issues (e.g., dizziness, nausea, somnolence, weight gain) Poor compliance / frequent switching Abuse liabilities (e.g., opioids) Novel MoAs polypharmacy/ combination therapy Unmet Need Est. Chronic Pain US+EU Treated Patients ~$20b Rx market (2018) Antidepressants (e.g., Cymbalta) Anticonvulsants (e.g., Lyrica) NSAIDs Opioids Other >36m >47m 3rd line 2nd line 1st line Sources: Decision Resources Group (DRG), Neuropathic Pain Landscape and Forecast (June 2020); DRG Current Treatment: Physician Insights, Neuropathic pain (October 2019) Decision Resources Group (DRG), Chronic Pain Landscape and Forecast (March 2021); DRG Current Treatment: Physician Insights, Chronic Pain (July 2019)
ETX-810: Prodrug of PEA (palmitoylethanolamide), an Endogenous Bioactive Lipid Acting as a Master Regulator of Neuroinflammation and Pain Signaling ETX-810 Petrosino and Di Marzo, Br J Pharmacol. 2017 174:1349 ETX-810 is being developed to restore PEA levels to reduce persistent neuroinflammation and pain signaling in chronic pain PEA is a master regulator of neuroinflammation and pain signaling with a pleiotropic mechanism1 Reduced PEA levels hypothesized in chronic pain pathology1 Inhibition of inflammatory mediator release from mast cells/monocytes/macrophages Agonism of PPAR-alpha inhibition of pro-inflammatory gene expression Agonism of GPR55 action on microglia activation and phagocytic activity Entourage effect via FAAH inhibition increase endocannabinoid levels (AEA, 2-AG, OEA) ETX-810
Clinical Validation of PEA: Compelling Body of Evidence Highlighting PEA’s Activity and Tolerability in Chronic Pain ETX-810 Paladini et al, Pain Physician, 2016, 19:11-24 Artukoglu et al, Pain Physician, 2017, 20:353-362 From Cymbalta (duloxetine) meta-analyses in chronic pain: Osani et al, Korean J Intern Med, 2019;34(5):966-973; Weng et al, Osteoarthritis Cartilage, 2020;28(6):721-734; Enomoto et al, J Pain Res, 2017;10:1357-1368; Ney et al, Pain Medicine, 2013;14:706-719 From Lyrica (pregabalin) meta-analyses in chronic pain: Onakpoya et al. BMJ Open, 2019;9(1):e023600; Parsons et al. Curr Med Res Opin, 2016;32(5):929-37; Zhang et al. Acta Anaesthesiol Scand, 2015;59(2):147-59; Ney et al, Pain Medicine, 2013;14:706-719 Consistent, clinically meaningful reductions in pain Benign tolerability profile Activity across a broad range of chronic pain conditions PEA in Chronic Pain >2,500 patients treated with PEA in >35 published clinical studies of PEA >1500 patients studied in 15 RCTs ~900 patients treated with PEA Meta-Analyses of PEA Chronic Pain Clinical Studies Reference Key Conclusions Paladini 20161 (12 studies) 81% achieved “mild pain” by day 60 (compared to 41% in control) Artukoglu 20172 (8 RCTs) 2-point pain score reduction* vs control *Mean pain score delta vs placebo for benchmark chronic pain drugs: Cymbalta - 0.8 to 1.23 Lyrica - 0.5 to 1.14
Clinical Validation of PEA: Two Large Placebo-Controlled Studies Demonstrate Clinical Activity and Dose-Dependent Response ETX-810 Guida et al, DOLOR, 2010; 25:35-42 Post-hoc analysis of Guida low back pain study by Cruccu et al, CNS & Neurological Disorders - Drug Targets, 2019; 18:491-495 Steels et al, Inflammopharmacology, 2019;27:475-485 VAS: Visual Analog Scale WOMAC: Western Ontario and McMaster Universities Osteoarthritis Index NRS: Numerical Rating Scale (segmented numeric version of VAS scale) Steels 20193 – Knee osteoarthritis N=111, PEA monotherapy 300 mg / 600 mg vs placebo, 8-week BID dosing Guida 20101 – Low back pain / sciatica N=636, PEA monotherapy 300 mg / 600 mg vs placebo, 21-day BID dosing Pain Score Reduction WOMAC total score Placebo 300 mg PEA 600 mg PEA * Anova p<0.001 * * -0.9 Δ vs placebo -3 Δ vs placebo ** 600 mg p=0.001 * 300 mg p=0.037 ** * Placebo PEA 300 mg PEA 600 mg Statistically significant reduction in pain vs placebo at d21 600 mg significantly better than 300 mg 82% of 600 mg group had ≥50% reduction in pain2 Higher neuropathic pain correlated with higher efficacy2 Significant reduction in WOMAC total score (pain, stiffness, and function) vs placebo at Wk 8, with dose-dependent response Statistically significant reduction in NRS pain vs placebo at Wk 8 (-2.1 pain reduction vs placebo at 600mg, data not shown)
ETX-810: Opportunity to Be a First-in-Class PEA Prescription Therapeutic for Chronic Pain ETX-810 Prodrug Program Rationale Program Goals Clear dose response in PEA RCTs Opportunity to enhance exposure Develop new chemical entity (NCE) through prodrug approach Optimize PK of bioactive PEA Maximize probability of clinical trial success Bring regulated PEA product to market supported by robust RCTs Rapid oral absorption and conversion to biologically active PEA through series of enzymatic hydrolysis steps Favorable pharmacokinetics – increased half-life and dose-dependent increase in exposure Strong preclinical activity and dose-dependent effect in models of inflammatory pain and neuropathic pain New IP generated with patent protections to 2037 Ester prodrug moiety Hydrolysis in tissue & serum Active PEA Protected PEA Absorption ETX-810
ETX-810 Has an Improved PK Profile and 3X Higher Exposure Compared to Dietary Supplement PEA ETX-810 Prodrug pharmacokinetics results in higher PEA concentration over time and 3x exposure improvement PEA concentration over time PEA exposure 600mg PEA Dietary supplement 1000 mg ETX-810 (= 490 mg PEA) 3x higher exposure* Longer half-life of bioactive PEA Significantly higher area under the curve *Up to ~6x higher exposure on daily basis with 1000 mg BID dosing 1000 mg BID dosing expected to yield 6x higher PEA exposure
Encouraging Tolerability in Phase 1 Study With All AEs Being Mild and at Similar Rates as Placebo, With No Discontinuations ETX-810 * same subjects participated in both the 150mg fasted and fed dosing conditions Highly differentiated Phase 1 tolerability profile for a chronic pain drug Participants were dosed every 12 hrs for 6 consecutive days; a single dose was administered on day 7 All doses were administered following a meal MAD Study (n=20) Adverse Event ETX-810 1000mg BID (n=8) ETX-810 500mg BID (n=8) Placebo (n=4) Any AE 38% 38% 50% Nausea 25% 0% 25% Vomiting 0% 0% 25% Menorrhagia 0% 25% 0% Dysmenorrhea 0% 13% 0% Insomnia 0% 13% 0% Headache 13% 0% 25% Dizziness 13% 0% 0% Muscle twitching 13% 0% 0% Muscle spasms 13% 0% 0% SAD Study (n=60) Adverse Event ETX-810 (50-1200mg) (n=48*) Placebo (n=12) Any AE 29% 33% Somnolence 10% 8% Dizziness 8% 0% Headache 4% 0% Disorientation 2% 0% Euphoric mood 2% 0% Paraesthesia 2% 0% Nausea 6% 17% Diarrhoea 2% 0% Dry mouth 2% 0% Dyspepsia 0% 8% Fatigue 2% 17% Pallor 2% 0% Palpitations 2% 0% Phase 2 dose
ETX-810: Two Phase 2a Proof of Concept Studies Now Enrolling With Topline Data Expected 1H 2022 ETX-810 Implementing clinical development strategies to refine patient population and limit placebo effect Lumbosacral Radicular Pain (LSRP) Diabetic Peripheral Neuropathic Pain (DPNP) NCT04778592 NCT04688671 N = 122 R N = 162 R ETX-810 (1000mg BID) Placebo BID ETX-810 (1000mg BID) Placebo BID 4 Weeks Study + 1 Week Follow up 4 Weeks Study + 1 Week Follow up Anticipated topline data 1H 2022 Anticipated topline data 1H 2022 Objectives: Demonstrate clinically meaningful improvement in neuropathic pain Confirm safety & tolerability Primary Outcome Measure: Change from baseline to Week 4 in weekly average of the daily pain score Rated on 11-point pain intensity numerical rating scale (PI-NRS) 80% power to detect a 1-point difference from placebo
Aiming to Develop a NCE with Desired Clinical Profile to Address the Large Chronic Pain Market ETX-810 * Decision Resources Group estimated chronic pain 2028 prevalence by indication, February 2021 A novel chronic pain therapy with a desirable product profile is a multibillion dollar opportunity Commercial Opportunity Target Profile for a New Chronic Pain Treatment Non-opioid, with no abuse liability Novel mechanism of action Efficacy as monotherapy and in combination Benign side effect profile No drug-drug interactions (DDIs) DPNP LSRP (neuropathic back pain) Chronic Low Back Pain OA Pain 2028 US+EU Forecast: ~50m 2nd line or later drug-treated chronic pain patients ~10m ≥2nd line treated LSRP and DPNP patients in US+EU At ~$5K annual price, every 2% patient market share is worth ~$1b/year in LSRP and DPNP alone Fibromyalgia, RA pain, Cancer Pain, PHN Broad expansion opportunities into large chronic low back pain and OA pain indications
ETX-155 Anticipated Milestones Photosensitive Epilepsy Data Expected 1H 2022 Major Depressive Disorder Topline Phase 2a Expected 1H 2023 Perimenopausal Depression Topline Phase 2a Expected 1H 2023
ETX-155: A Differentiated Neuroactive Steroid GABAA Positive Allosteric Modulator ETX-155 Dual potent activity at synaptic and extrasynaptic GABAA receptors, with high intrinsic efficacy Well tolerated at exposure levels that have translated to clinical efficacy for other GABAA PAMs No clinically meaningful food effect Convenient once-daily dosing with ~40-hr half-life Strong IP position with patent protection to 2039 Clinical validation for MOA (GABAA PAM)
Clinical Development Focused on Depressive Disorders and Focal Onset Seizure – Large Markets With Considerable Unmet Need ETX-155 Perimenopausal Depression (PMD) Estimated annual prevalence (US+EU) MoA Rationale Unmet Needs Reduced GABA levels increased MDD severity1 Clinically validated (SAGE-217) Faster onset of action Improved tolerability/efficacy Novel MoAs Same as MDD Novel MoAs directly addressing reduced neurosteroid levels Reduced neurosteroid levels PMD symptoms Clinically validated in neurosteroid-driven PPD (SAGE-217) ~32m (~9m failed ≥1 prior therapy)2 ~8m (~2m with no history of MDD)3 GABAergic deficits epileptic state Clinically validated in orphan epilepsies (ganaxolone) Novel MoAs better seizure control Positive impact on mood as #1 comorbidity is depression4 ~2m (~0.8m with uncontrolled seizures)5 Epilepsy / Focal Onset Seizure (FOS) Major Depressive Disorder (MDD) Luscher et al, Mol Psychiatry, 2011;16(4):383-406 Decision Resources Group (DRG)– Unipolar Depression Disease Landscape and Forecast Freeman et al, JAMA Psychiatry, 2014;72(1):36-43 Kanner AM, Biol Psychiatry, 2003;54(3):388-98 DRG – Epilepsy Disease Landscape and Forecast, May 2021
Company Molecule GABAAR Activity Pharmacokinetics Half-life Oral Bioavailability Clinical Validation (positive RCT) Synaptic Extra-synaptic Food effect Half-life Oral Bioavailability MDD PPD or PMD Epilepsy ETX-155 No ~40 hrs ~70% (tablet) 1H 2023 1H 2023 2024 SAGE-217 (zuranolone) Yes 14-18 hrs 68% (capsule) - ganaxolone Yes 2-3 hrs 10% (capsule) - - PRAX-114 Yes 12–15 hrs Not disclosed 1H 2022 TBD - ETX-155 Differentiation: Significantly Longer Half-Life, Lack of Food Effect, Favorable Bioavailability and Broad GABAAR Activity ETX-155 Sources: SAGE-217: Hoffmann et al, Clin Pharmacokinet, 2020;59(1):111-120; Hoffmann et al, ASCP 2018, poster #782; Botella et al, J Med Chem, 2017;60(18)7810-7819. PRAX-114: Praxis Precision Medicines. 2020 Form S-1 Registration Statement Ganaxolone: Hulihan et al., American Epilepsy Society Annual Meeting 2020, poster RCT: randomized, controlled clinical trial PPD: Postpartum Depression; MDD: Major Depressive Disorder; PMD: Perimenopausal Depression; PTSD: Post-Traumatic Stress Disorder; ET: Essential Tremor
Phase 1 Study in Healthy Subjects: Excellent Pharmacokinetics and Safety & Tolerability Profile with No Severe or Serious Adverse Events ETX-155 ETX-155 Phase 1 Repeat-Dose Results Favorable pharmacokinetics Steady state reached at day 8 ~40-hour half-life at steady state 60 mg evening dosing was well tolerated No SAEs or discontinuations All AEs were mild/moderate and transient CNS AE details The rate of CNS AEs were comparable in ETX-155 and placebo groups Most CNS AEs occurred at Tmax (3-4 hrs post-dose) 7 reports of somnolence out of 24 ETX-155-treated patients (no subject reported somnolence more than once during dosing period) Leeds Sleep Evaluation Questionnaire indicates no difference in next-morning alertness or disruption in sleep quality compared to placebo 7-day Repeat Dose 14-day Repeat Dose Combined ETX-155 60 mg (n=9) Placebo (n=6) ETX-155 60 mg (n=15) Placebo (n=5) ETX-155 60 mg (n=24) Placebo (n=11) n (%) n (%) n (%) n (%) n (%) n (%) ≥1 TEAE 5 (56) 3 (50) 9 (60) 4 (80) 14 (58) 7 (64) Somnolence 1 (11) 2 (33) 6 (40) 2 (40) 7 (29) 4 (36) Fatigue 0 0 4 (27) 1 (20) 4 (17) 1 (9) Headache 2 (22) 2 (33) 1 (7) 0 3 (13) 2 (18) Dizziness 1 (11) 0 2 (13) 0 3 (13) 0 Most common treatment-emergent AEs (In ≥10% of ETX-155 treated subjects across repeat dose studies)
ETX-155 Does Not Have a Clinically Meaningful Food Effect: Potential to Impact Efficacy, Safety, and Compliance ETX-155 has not been assessed in a head-to-head study against PRAX-114, SAGE-217, or ganaxolone, and the study designs and analytical methods for all four product candidates may be different. As a result, such data may not be directly comparable. ETX-155 Presence of a food effect may impact: Efficacy Exposure reduced or increased if medication not taken with food Safety and Tolerability Timing/severity of AEs associated with Cmax Compliance More strict daily routine required to maintain drug levels within the required range for efficacy and safety SAGE-2172 PRAX-1141 Ganaxolone3 NO FOOD EFFECT4 ETX-155 AUC(0-t) Cmax FOOD EFFECT Clinically meaningful higher exposure if taken with food FOOD EFFECT Clinically meaningful lower exposure if taken with food 1.25x 0.8x 1.6x 2.9x 2.6x 3.0x 0.6x Fed/Fasted Ratio Tablet, 30mg, human Suspension, 30mg, human Capsule, 30mg, human Capsule, 5 mg/kg, dog Reported Fed/Fasted Ratios for GABAA PAM class Praxis Precision Medicines, Form S-1/A, Oct 15, 2020 Hoffmann et al, Clin Pharmacokinet, 2020;59(1):111-120; Hoffmann et al, ASCP 2018, poster #782 U.S. Patent No. 9,029,355 Range of fed/fasted ratios for AUC and Cmax required to claim absence of food effect on bioavailability, per FDA Guidance For Industry, Food-Effect Bioavailability and Fed Bioequivalence Studies, December 2002
Progressing ETX-155 in Epilepsy: Phase 1b Proof-of-Concept Trial in Photosensitive Epilepsy (PSE) to De-risk Focal Onset Seizure Study ETX-155 Study Details Rationale Design: Phase 1b, single-center, randomized, double-blind, placebo-controlled, 2-sequence crossover study Objective: Provide evidence of inhibition of PPR in subjects with PSE Dose: Single dose of 135 mg (MTD), then titrate down until loss of effect N= 6 Primary Outcome Measure: Change in PPR range vs placebo at 1, 2, 4, 6, and 8hr PSE is characterized by a photoparoxysmal response (PPR) triggered by light stimulation Single dose PSE trials are valuable in predicting efficacy in epilepsy and aiding in dose selection for later phase trials Reduction of an induced PPR EEG response in PSE has proven a reliable biomarker of anticonvulsant activity in epilepsy for most approved ASMs1 Data anticipated 1H 2022 1. Yuen and Sims, Seizure, 2014, 23:490-493
Progressing ETX-155 in Depressive Disorders: Two Phase 2a RCTs of ETX-155 in MDD and PMD, with Topline Data Anticipated in 1H 2023 ETX-155 Screening Outpatient Treatment Follow-up 28 days Week 14 days 1 2 3 4 ETX-155 60 mg tablets qd x 28 days Placebo qd x 28 days MDD Phase 2a ~80 subjects 12 US sites d1 d8 d15 d22 d29 1:1 RANDOMIZATION d43 PMD Phase 2a ~80 subjects 15 US sites Topline data from both studies anticipated 1H 2023 Primary Endpoint (both PMD and MDD studies) Change from baseline in HAMD17 over days 8, 15, 22, and 29* Key Secondary Endpoints Change from baseline in HAMD17 at day 8, 15, 22, 29, and 43* 50% response rate in HAMD17 at day 8, 15, 22, 29* HAMD17: Hamilton Depression Rating Scale * Evaluating additional HAMD measurement at Day 3.
ETX-155: Potentially Clinically Differentiated Oral Neuroactive Steroid in Markets with Significant Unmet Needs ETX-155 Improve Efficacy Leverage absence of food effect & significantly longer half-life ETX-155 Opportunities Unmet Needs Improve Tolerability Highly encouraging CNS AE rates in healthy subjects Improve Durability Leverage longer half-life and evaluate longer dosing periods (i.e., ≥28 days) Novel MoA Clinicians combine different MoAs to improve seizure control Well Tolerated Encouraging Phase 1 tolerability data when considering use as an add-on therapy Positive impact on mood Potential to provide differentiated benefit on common depression comorbidity Depressive Disorders Slow onset of efficacy (~6+ wks) High refractory rates Tolerability issues limit compliance Focal Onset Seizure 30% of patients on ASMs have uncontrolled seizures #1 co-morbidity is depression
Kv7.2/3 Program: Developing a Differentiated Kv7.2/3 Opener For Multiple Neuronal Excitability Indications Kv7 IND-enabling studies anticipated to initiate 1H 2022 Opportunity across multiple indication areas Epilepsy Chronic Pain Depression Kv7 Eliem Kv7 Program Goal Kv7 Opportunity Program Status Maintain efficacy with improved tolerability and safety Multiple lead and backup chemotypes in novel IP space Improved metabolic stability Potent at Kv7.2/3 and selective vs Kv7.1/4 Human genetic validation Strong clinical validation in pain and epilepsy (retigabine, flupirtine, XEN1101) Clear translational path to clinical efficacy Metabolic/safety liabilities with existing molecules
Multiple Catalysts and Value-Creating Milestones Across Pipeline – Existing Cash Runway Through Five Topline Data Catalysts 2H 2021 1H 2022 2H 2022 1H 2023 Ph 1b PSE Topline data Ph 2a DPNP Ph 2a LSRP Ph 2a MDD ETX-810 ETX-155 Ph 2a PMD Ph 2 FOS Preclinical Kv7 program IND-enabling studies GAD program candidate selection Ph 1 Cash runway into late 2023 Ph 1 IND-enabling studies Topline data Topline data Topline data Topline data Anticipated Catalysts and Key Milestones Financial Summary Cash, Cash Equivalents & Marketable Securities $169.6 million As of Sept 30, 2021 Q3 2021 Operating Expenses $9.4 million R&D: $6.0 million G&A: $3.4 million
Highly experienced management team Clinical and preclinical pipeline based on clinically validated mechanisms of action Two clinically differentiated lead product candidates with top-line data readouts across five indications over next 24 months ~$170M* cash runway to late 2023 allows for top line data readouts and advancement of preclinical assets Rethinking treatment for nervous system disorders * Cash, cash equivalents and marketable securities as of September 30, 2021
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