UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 OR 15(d)
of The Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
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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).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Item 2.02 Results of Operations and Financial Condition.
On March 11, 2022, Clene Inc. (the “Company”) issued a press release announcing its full year operating and financial results for its year ended December 31, 2021. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K (the “Current Report”) and is incorporated herein by reference.
The information furnished in this Item 2.02, including Exhibit 99.1, shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”), as amended, or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference into any filing made by the Company under the Exchange Act or the Securities Act of 1933 (the “Securities Act”), as amended, regardless of any general incorporation language in any such filings, except as shall be expressly set forth by specific reference in such a filing.
Item 7.01 Regulation FD Disclosure.
In connection with the March 11, 2022 press release announcing the Company’s full year operating and financial results for its year ended December 31, 2021, the Company released an updated corporate presentation (the “Corporate Presentation”) on its website, www.clene.com. A copy of the Corporate Presentation is furnished as Exhibit 99.2 to this Current Report and is incorporated herein by reference. The Company plans to use its website to disseminate future updates to the Corporate Presentation and may not file or furnish a Current Report on Form 8-K alerting investors if the Corporate Presentation is updated.
The information furnished in this Item 7.01, including Exhibit 99.2, shall not be deemed to be “filed” for purposes of Section 18 of the Exchange Act, as amended, or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference into any filing made by the Company under the Exchange Act or the Securities Act, regardless of any general incorporation language in any such filings, except as shall be expressly set forth by specific reference in such a filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
Exhibit |
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Exhibit Description |
99.1 |
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99.2 |
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104 |
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Cover Page Interactive Data File (formatted as Inline XBRL). |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, hereunto duly authorized.
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CLENE INC. |
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Date: March 11, 2022 |
By: |
/s/ Robert Etherington |
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Robert Etherington |
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President and Chief Executive Officer |
2
Exhibit 99.1
Clene Reports Full Year 2021 Financial Results and Recent Operating Highlights
SALT LAKE CITY, March 11, 2022 -- Clene Inc. (Nasdaq: CLNN) along with its subsidiaries “Clene” and its wholly owned subsidiary Clene Nanomedicine Inc., a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease, today reported its full year 2021 operating and financial results, as well as an overview of fourth quarter 2021 and recent operating highlights.
“We exited 2021 with significant momentum, having made substantial clinical advancement across our portfolio of first-in-class nanotherapeutics,” said Rob Etherington, President and CEO of Clene. “This progress now has Clene positioned to achieve multiple clinical milestones in 2022, highlighted by the upcoming results from the HEALEY ALS Platform Trial. Positive results for CNM-Au8® in this study would be transformative for Clene, and more importantly, for people living with ALS.”
Fourth Quarter 2021 and Recent Operating Highlights
CNM-Au8®, a gold nanocrystal suspension, for the treatment of amyotrophic lateral sclerosis (ALS)
CNM-Au8® for the treatment of multiple sclerosis (MS)
CNM-ZnAg™ for the treatment of COVID-19
Corporate Updates
Full Year 2021 Financial Results
Clene’s cash and restricted cash totaled $50.3 million as of December 31, 2021, compared to $59.3 million as of December 31, 2020. Clene expects that its resources as of December 31, 2021, will be sufficient to fund its operations into the second quarter of 2023.
Research and development expenses were $28.4 million for the year ended December 31, 2021, compared to $15.2 million for the same period in 2020. The year-over-year increase was primarily related to the development of CNM-Au8, rent expense for the newly-leased facility in Elkton, Maryland, and personnel and stock-based compensation due to increased headcount, partially offset by decreased manufacturing and materials expense.
General and administrative expenses were $22.0 million for the year ended December 31, 2021, compared to $5.2 million for the same period in 2020. The year-over-year increase was primarily attributable to costs related to being a public company and fees for professional services, technology services, and pre-commercialization activities for CNM-Au8, and personnel and stock-based compensation due to increased headcount.
Clene reported a net loss of $9.7 million, or $0.16 per share, for the year ended December 31, 2021, compared to a net loss of $19.3 million, or $1.10 per share, for the same period in 2020. Included in net loss for the year ended December 31, 2021, was an unrealized gain from the change in fair value of contingent earn-out liabilities of $37.5 million, compared to $14.1 million in the prior year period.
About Clene
Clene is a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative disease by targeting energetic failure, an underlying cause of many neurological diseases. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit www.clene.com or follow us on Twitter, LinkedIn and Facebook.
About CNM-Au8®
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8® is a federally registered trademark of Clene Nanomedicine, Inc.
About CNM-ZnAg
CNM-ZnAg, a proprietary zinc-silver ionic solution, has demonstrated broad antiviral and antimicrobial activity.
Forward-Looking Statements
This press release contains “forward-looking statements” which are intended to be covered by the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Clene’s actual results may differ from its expectations, estimates and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as “expect,” “estimate,” “project,” “budget,” “forecast,” “anticipate,” “intend,” “plan,” “may,” “will,” “could,” “should,” “believes,” “predicts,”
“potential,” “might” and “continues,” and similar expressions are intended to identify such forward-looking statements. These forward-looking statements involve significant known and unknown risks and uncertainties, many of which are beyond Clene’s control and could cause actual results to differ materially and adversely from expected results. Factors that may cause such differences include Clene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; Clene’s ability to achieve commercial success for its marketed products and drug candidates, if approved; Clene’s ability to obtain and maintain protection of intellectual property for its technology and drugs; Clene’s reliance on third parties to conduct drug development, manufacturing and other services; Clene’s limited operating history and its ability to obtain additional funding for operations and to complete the licensing or development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on Clene’s clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in Clene’s Annual Report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Clene’s subsequent filings with the U.S. Securities and Exchange Commission. Clene undertakes no obligation to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based, subject to applicable law. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.
Media Contact |
Investor Contact |
Erica Fiorini, Ph.D., or David Schull |
John Woolford |
Russo Partners, LLC |
Managing Director, Westwicke |
Erica.fiorini@russopartnersllc.com |
clene@westwicke.com |
David.schull@russopartnersllc.com |
+1-443-213-0506 |
+1-212-845-4253 |
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Source: Clene Inc.
CLENE INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
(Audited)
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Year Ended December 31, |
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2021 |
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2020 |
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Revenue: |
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Product revenue |
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$ |
570 |
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$ |
176 |
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Royalty revenue |
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153 |
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30 |
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Total revenue |
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723 |
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206 |
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Operating expenses: |
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Cost of revenue |
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289 |
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65 |
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Research and development |
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28,416 |
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15,204 |
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General and administrative |
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21,996 |
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5,151 |
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Total operating expenses |
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50,701 |
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20,420 |
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Loss from operations |
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(49,978 |
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(20,214 |
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Other income (expense), net: |
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Interest expense |
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(870 |
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(950 |
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Gain on extinguishment of notes payable |
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648 |
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— |
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Loss on extinguishment of convertibles notes payable |
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— |
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(540 |
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Gain on termination of lease |
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— |
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51 |
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Change in fair value of preferred stock warrant liability |
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— |
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(14,615 |
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Change in fair value of common stock warrant liability |
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983 |
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— |
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Change in fair value of derivative liability |
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— |
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29 |
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Change in fair value of Clene Nanomedicine contingent earn-out |
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33,953 |
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12,659 |
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Change in fair value of Initial Stockholders contingent earn-out |
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3,589 |
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1,465 |
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Australia research and development credit |
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1,519 |
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3,210 |
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Other income (expense), net |
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(12 |
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34 |
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Total other income (expense), net |
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39,810 |
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1,343 |
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Net loss before income taxes |
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(10,168 |
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(18,871 |
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Income tax benefit (expense) |
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428 |
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(406 |
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Net loss |
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(9,740 |
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(19,277 |
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Other comprehensive income (loss): |
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Foreign currency translation adjustments |
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(92 |
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284 |
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Total other comprehensive income (loss) |
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(92 |
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284 |
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Comprehensive loss |
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$ |
(9,832 |
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$ |
(18,993 |
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Net loss per share-- basic and diluted |
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$ |
(0.16 |
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$ |
(1.10 |
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Weighted average common shares used to compute basic and diluted net loss |
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61,558,455 |
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17,503,992 |
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CLENE INC.
CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share amounts)
(Audited)
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December 31, |
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2021 |
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2020 |
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ASSETS |
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Current assets: |
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Cash |
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$ |
50,288 |
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$ |
59,275 |
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Accounts receivable |
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49 |
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21 |
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Inventory |
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41 |
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191 |
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Prepaid expenses and other current assets |
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4,205 |
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3,502 |
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Total current assets |
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54,583 |
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62,989 |
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Restricted cash |
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58 |
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— |
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Right-of-use assets |
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3,250 |
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1,029 |
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Property and equipment, net |
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5,172 |
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4,225 |
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TOTAL ASSETS |
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$ |
63,063 |
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$ |
68,243 |
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LIABILITIES AND STOCKHOLDERS’ EQUITY |
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Current liabilities: |
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Accounts payable |
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$ |
1,923 |
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$ |
1,124 |
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Accrued liabilities |
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3,610 |
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3,960 |
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Income tax payable |
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— |
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164 |
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Deferred revenue from related parties |
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— |
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112 |
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Operating lease obligations, current portion |
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347 |
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194 |
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Finance lease obligations, current portion |
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146 |
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190 |
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Clene Nanomedicine contingent earn-out, current portion |
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— |
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5,924 |
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Total current liabilities |
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6,026 |
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11,668 |
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Operating lease obligations, net of current portion |
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4,370 |
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1,785 |
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Finance lease obligations, net of current portion |
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97 |
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205 |
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Notes payable |
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14,484 |
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1,949 |
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Convertible notes payable |
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4,598 |
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— |
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Deferred income tax |
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— |
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260 |
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Common stock warrant liability |
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474 |
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— |
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Clene Nanomedicine contingent earn-out, net of current portion |
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18,100 |
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46,129 |
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Initial Stockholders contingent earn-out |
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2,317 |
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5,906 |
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TOTAL LIABILITIES |
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50,466 |
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67,902 |
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Commitments and contingencies |
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Stockholders’ equity: |
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Common stock, $0.0001 par value: 150,000,000 and 100,000,000 shares authorized at December 31, 2021 and December 31, 2020, respectively; 62,312,097 and 59,526,171 shares issued and outstanding at December 31, 2021 and December 31, 2020, respectively |
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6 |
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6 |
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Additional paid-in capital |
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175,659 |
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153,571 |
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Accumulated deficit |
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(163,301 |
) |
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(153,561 |
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Accumulated other comprehensive income |
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233 |
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|
325 |
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TOTAL STOCKHOLDERS’ EQUITY |
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12,597 |
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|
341 |
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TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY |
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$ |
63,063 |
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$ |
68,243 |
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Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Clene's actual results may differ from its expectations, estimates, and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Words such as "expect," "estimate," "project," "budget," "forecast," "anticipate," "intend," "plan," "may," "will," "could," "should," "believes," "predicts," "potential," "might" and "continues," and similar expressions are intended to identify such forward-looking statements. These forward-looking statements involve significant known and unknown risks and uncertainties, many of which are beyond Clene’s control and could cause actual results to differ materially and adversely from expected results. Factors that may cause such differences include Clene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; Clene’s ability to achieve commercial success for its marketed products and drug candidates, if approved; Clene’s ability to obtain and maintain protection of intellectual property for its technology and drugs; Clene’s reliance on third parties to conduct drug development, manufacturing and other services; Clene’s limited operating history and its ability to obtain additional funding for operations and to complete the licensing or development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on Clene’s clinical development, commercial and other operations, as well as those risks more fully discussed in the section the section entitled “Risk Factors” in Clene’s recently filed Annual Report on Form 10-K (filed March 11, 2022), as well as discussions of potential risks, uncertainties, and other important factors in Clene’s subsequent filings with the U.S. Securities and Exchange Commission. Clene undertakes no obligation to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based, subject to applicable law. All information in this presentation is as of the date of presented or the date made publicly available. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this presentation.
CLENE | Leadership Transforming the treatment of neurodegenerative disorders by restoring and protecting neurological function
CLENE | Overview December 31, 2021 Cash and restricted cash on hand (audited): $50.3M ALS Registration Trial Topline data in 2H 20222 >300 patient years of CNM-Au8 clinical exposure CNM-Au8® a gold nanocrystal suspension, in development as the first cellular energetic catalyst to remyelinate1 & protect neurological function Manufacturing expansion in progress, preparing for possible commercialization in 2023 Strong IP: 150+ patents on Clean-Surface- Nanocrystal technology (CSN®) platform Data on File, Clene Nanomedicine, Inc. 1Robinson et al. Sci Rep. 2020 Feb 11;10(1):1936.2https://clinicaltrials.gov/ct2/show/NCT04414345.
CLENE | Pipeline
Fu, H., et al; Nature Neuroscience (2018) 21: 1350-1358. Zhu et al. Proc Natl Acad Sci USA . 2015 Mar 3;112(9):2876-81. Rone et al. J Neurosci. 2016 Apr 27;36(17):4698-707. Neurons With High Energetic Demand Are At Increased Risk For Neurodegenerative Disease ~0.5% NAD+/NADH unit decline per decade (~0.13 mV units per year by 31P-MRS Imaging) Brain Energy Potential Declines With Normal Aging Closed squares = averaged data by age group: 21–26 yrs, 33–36 yrs, and 59–68 yrs old; Open squares= individual subject values PARKINSON’S DISEASE Dopaminergic Neurons AMYOTROPHIC LATERAL SCLEROSIS Motor Neurons HUNTINGTON’S DISEASE Medium Spiny Neurons FRONTOTEMPORAL DEMENTIA Spindle Neurons Specific Neuronal Populations Are Vulnerable to Energetic Failure MULTIPLE SCLEROSIS Axonal Degeneration
Vertices, Edges, & Facets Key to Catalytic Activity CNM-Au8 Catalytically Active Nanocrystal Suspension Clean Surfaced, Highly Faceted Shape Enhances Catalytic Activity CNM-Au8 Nanocrystal CNM-Au8® | Catalytically-Active Nanocrystals Intersection of Physics and Biology Electron Sharing Drives Catalytic Activity 60 mL per bottle (once daily)
CNM-Au8 Nanocrystal Mechanistic Effects Improved Energy Production and Utilization Promotes Neuroprotection and Remyelination CNM-Au8® | Improves Energy Production to Promote Neuroprotection and Remyelination
CNM-Au8® | Preclinical Evidence for Energetic Improvement Therapeutic Activity Across Remyelination + Neuroprotection Models Data on File, Clene Nanomedicine, Inc. Robinson et al. Sci Rep. 2020 Feb 11;10(1):1936. MN Neuroprotection ALS SOD1, In Vitro Forebrain Neuroprotection C9ORF72 In Vitro Alzheimer’s Disease Induced Neurons In Vitro
CNM-Au8® | Significant Global Opportunity Source: Lancet Neurol. 2018 Nov;17(11):939-953; ~6.1M patients globally, data as of 2016.. PARKINSON’S DISEASE ~6.1M pts globally; $6B projected by 20263 2ND most common neurodegenerative disorder; only symptomatic treatments Source: Lancet Neurol. 2018 Dec;17(12):1083-1097. MND includes amyotrophic lateral sclerosis, spinal muscular atrophy, hereditary spastic paraplegia, primary lateral sclerosis, progressive muscular atrophy, and pseudobulbar palsy MOTOR NEURON DISEASE (ALS, Other Orphan Disorders) ALS sales >$1B globally by 20291. Current drugs are largely ineffective, mostly generic Source: Lancet Neurol. 2019 Mar;18(3):269-285; ~2.2.M patients globally, data as of 2016 MULTIPLE SCLEROSIS ~2.2M pts globally; $23B market2 Only approved treatments are immunomodulators 1 Clarivate, DRG, ALS 2020. 2 Westad et al. 2017, doi:10.1038/nrd.2017.107;. 3 Parkinson’s Market Data Forecast, April 2021.
CNM-Au8® | Neuroprotection & Remyelination Data on File, Clene Nanomedicine, Inc. Phase 2 and Phase 3 Clinical Trials CNM-Au8 treatment effect on ALS disease progression Ongoing Established brain target engagement & safety Planned: Demonstrate Disease Modification Concluded enrollment due to COVID Established brain target engagement & safety Key Findings Disease Clinical Trials Q4 2021 Q3 2021 Q1 2022 Q2 2022 Q3 2022+ Establish target engagement & safety in non-active, progressive MS population
CNM-Au8® | Safety Summary Over 300 Years of Subject Exposure Without Any Safety Signals Patient Exposure Across PD, MS, & ALS Long-term dosing experience up to 125 weeks All Animal Toxicology Studies Resulted in No-Adverse Effect Level (NOAEL) Findings Clean Toxicology Findings Multiple species up to 9-months treatment Up to maximum feasible dosing without any toxicology findings related to CNM-Au8 Assessed as Predominantly Mild-to-Moderate Severity and Transient Well Tolerated Adverse Event (AE) Profile No related CNM-Au8 AEs leading to discontinuation of treatment No SAEs related to CNM-Au8 considered severe, life-threatening, or resulting in death Data on File, Clene Nanomedicine, Inc.
Difference in brain NAD+ and NADH fraction at Week 12 (End of Treatment) Difference in bioenergetic metabolites (e.g., ATP, NAD) concentration at Week 12 – 16 Difference in brain membrane markers (PE, PC, etc.) at Week 12 – 16 Change in Brain Bioenergetic Potential (NAD+/NADH) vs. Baseline 1 2 CNM-Au8 Effects on Brain Energetic Metabolites A Phase 2, Open Label, Sequential Group, Investigator Blinded Study of Magnetic Resonance Spectroscopy (31P-MRS) to Assess the Effects of CNM-Au8 for the Bioenergetic Improvement of Impaired Neuronal Redox State (REPAIR) N = Up to 15 per dosing cohort (7.5, 15, 30, or 60 mg) Phase 2 Exploratory Stable Relapsing MS Early Parkinson’s Disease Clinic Visit Safety Clinic Visit Safety Clinic Visit Primary Endpoint 31P-MRS Scan Clinic Visit Follow-up 31P-MRS Scan Baseline Visit 31P-MRS Scan + 4-6 weeks Data on File, Clene Nanomedicine, Inc. Non-Active Progressive MS (Underway) Non-active, progressive MS patients is more severe than relapsing MS., and a high unmet need for disease modifying therapeutic options Day 0
CNM-Au8 Improves Brain Energy Metabolism Increases NAD+/NADH Ratio in MS & PD Phase 2 Results 1° Endpoint 2° Endpoint NAD is an essential molecule responsible for cellular energy production Exploratory (ATP Normalization) Glanzman et al. 2021. International Parkinson and Movement Disorder Society (MDS) Virtual Congress 2021. September 17 – 22, 2021 . Data on File, Clene Nanomedicine, Inc.
(January 2020); FPFV 16-Jan-2020; 45 of 42 enrolled (Nov-2020) Phase 2 1 Study was powered for MUNIX primary endpoint Neurophysiology MUNIX1 Pulmonary Function Forced Vital Capacity Function & QoL ALSFRS-R, ALSSQOL-SF Disease Progression & Survival Vucic et al. BMJ Open. 2021 Jan 11;11(1):e041479. Data on File, Clene Nanomedicine, Inc.
| Pioneered Use of MUNIX Biomarker Primary Endpoint: Spinal Cord Lower Motor Neuron Protection Muscle Fibers Spinal Cord Motor Neuron Bulbar Onset ALS (Brainstem) Limb Onset ALS (Spinal Cord) Primary Endpoint: Spinal Cord Lower Motor Neuron Motor Unit Index (MUNIX) Sum Biceps brachii Tibialis Anterior Abductor Pollicis Brevis Abductor Digiti Minimi + + + Motor Units MUNIX biomarker estimates the number of functioning lower motor neurons serving specific muscles Vucic et al. BMJ Open. 2021 Jan 11;11(1):e041479.
| Evidence for Motor Neuron Protection Primary Endpoint (MUNIX %, LS Mean Change) All Randomized All Placebo Limited Rate of MUNIX Decline in Bulbar Onset Insufficient Spinal Cord Lower Motor Neuron Progression in Early Bulbar Trial Participants RESCUE-ALS: A Phase 2, randomized, double-blind, placebo-controlled study of CNM-Au8 to slow disease progression in ALS. MNDA Virtual Symposium, 2021 Data on File, Clene Nanomedicine, Inc. +45%
| Significant Impact on ALSFRS-R Decline Exploratory (ALSFRS-R Responder Analysis, < 6-point decline) All Randomized All Bulbar All Limb Sensitivity RESCUE-ALS: A Phase 2, randomized, double-blind, placebo-controlled study of CNM-Au8 to slow disease progression in ALS. MNDA Virtual Symposium, 2021 Data on File, Clene Nanomedicine, Inc.
| Significant Quality of Life Improvement Exploratory (ALS Specific QOL-SF) All Randomized RESCUE-ALS: A Phase 2, randomized, double-blind, placebo-controlled study of CNM-Au8 to slow disease progression in ALS. MNDA Virtual Symposium, 2021 Data on File, Clene Nanomedicine, Inc.
| Significant Impact on ALS Disease Progression Exploratory Endpoint (Disease Progression) 1ALS Disease Progression defined as: Death, or Tracheostomy, or Non-invasive ventilation, or Gastrostomy tube All Bulbar All Limb Sensitivity RESCUE-ALS: A Phase 2, randomized, double-blind, placebo-controlled study of CNM-Au8 to slow disease progression in ALS. MNDA Virtual Symposium, 2021 Data on File, Clene Nanomedicine, Inc. All Randomized
| Joint Rank: Survival & ALSFRS-R Scoring Berry et al. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Apr;14(3):162-8. ALSFRS-R Decline Survival CAFS All Randomized Exploratory Endpoint Pre-specified (Combined Assessment of Survival and Function [CAFS]) Score participants based on relative function or time of death
| Impact on Joint Rank Score to Wk36 Post Hoc (Combined Assessment of (i) Survival, (ii) King’s Clinical Stage 4, (iii) ALSFRS-R) Data on File, Clene Nanomedicine, Inc. ALSFRS-R Decline Survival Non-Invasive Ventilation Gastrostomy Tube King’s Clinical Stage 4 By Average of Summated Scores
| Potential Impact on Survival Exploratory Endpoint (Observed Survival vs. Median Predicted) ENCALS Predicted Median Survival Observed Survival Data on File, Clene Nanomedicine, Inc.
| Well Tolerated & No Safety Signals Data on File, Clene Nanomedicine, Inc. No CNM-Au8 related serious adverse events (SAEs) No CNM-Au8 related drug discontinuations No imbalances in treatment emergent adverse event (TEAEs) by system organ class TEAEs were predominantly mild-to-moderate and transient Most common TEAEs associated with CNM-Au8 (aspiration pneumonia, n=3; nausea, n=2; abdominal discomfort, n=2) Safety Summary
Exploratory Endpoints Combined Joint Rank (Survival + ALSFRS-R) Voice pathology PRO (ALSAQ) Pharmacodynamic markers Slow Vital Capacity Hand-Held Dynamometry Survival Change in ALSFRS-R Registration Study: 24-Week Treatment Period (3:1 randomization, 120 active [30mg, 60mg]: 40 placebo) 1 2 Phase 3 Anticipated full unblinded data readout: 2H 2022
Exploratory Endpoints Treatment of Visual Pathway Deficits In Chronic Optic Neuropathy for Assessment of Remyelination in Non-Active Relapsing MS Change in Low Contrast Letter Acuity (LCLA) At Week 24 Up to 48-Week Placebo-Control 2:1 Randomization (Active: Placebo) 15mg, 30mg, Placebo (n=73 of 150) Optical Coherence Tomography (OCT) Multi-focal VEP Amplitude & Latency Full field-VEP Amplitude & Latency MRI Endpoints Visual Function (High Contrast) QOL / EDSS 1 2 Change Composite Clinical Response 9HPT / SDMT / T25FW / LCLA Phase 2 -42 to -1 24-Week Blinded Fixed Treatment Period Up to 24-Week Blinded Extension Period (Until LPLV 24Wk Visit) LPLV *Subject to ongoing COVID-19 related site research restrictions generally implemented to protect MS patients taking standard-of-care immunosuppressive therapies Anticipated top-line unblinded data: 2H 2022 Insights to inform new Phase 2/3 MS trial
LCLA Phase 2 Primary: Functional Visual Improvement LCLA Correlates with clinically meaningful deficits in QOL, EDSS and MSFC, MRI, and OCT1 MS Functional Endpoints Phase 2 Exploratory: Neuroprotection/Remyelination Endpoints The Visual System is a Window into the Brain 9-Hole Peg Test Symbol Digit Modalities Timed 25-Ft Walk Measuring MS Functional Improvement 1Balcer et la. Mult Scler. 2017 Apr;23(5):734-747. doi: 10.1177/1352458517690822.
Significant Clinical Improvement Across Blinded Study Population Primary Endpoint: LCLA (Best-Corrected) & Secondary Endpoint: (m)MSFC Phase 2 2° | (m)MSFC 1° | LCLA Glanzman et al. VISIONARY-MS: Update to a Phase 2 Clinical Trial of CNM-Au8, Catalytically Active Gold Nanocrystals Suspension, for the Treatment of Chronic Optic Neuropathy ACTRIMS, February 2022.
Proportion of participants who require COVID-19 related hospitalization, or died at Day 28 Secondary Endpoint Exploratory Endpoints Primary Endpoint Time to substantial alleviation of COVID-19 symptoms through Day 28 Number of hospital free days through Day 28 Mean change in SARS-CoV-2 viral load Change in oxygen saturation slope Change in Global Impression (severity and change)
Trade Secrets Patent Status b Patent Description Issued & Allowed Patents 150+ Pending Applications ~20 Total Patents/ Applications >170 Process And Method/Device (Clean Surface; Gold CSN) State of Matter (CNM-Au8) Method of Use (Prevent Demyelination & MoA) Method of Use (Bi-Metallic Au/Pt; Antimicrobial) Plasma Conditioning Electrode Design & Cycling Trough Flow, Temp, Pressure Concentration & Filtration Strong Intellectual Property Extensive Patent Portfolio With Protection Through 2035 a & Proprietary Trade Secrets; Plus 7-year Orphan Drug Designation a With Patent Restoration Term (assuming 5-year extension). b As of 30-Sepbember-2021. CSN® Clean surface nanocrystal (CSN) therapeutics
Clene | Proprietary Nanocrystal Manufacturing In-House ISO8 Clean Room Clinical Production in Maryland Designed to be Scalable to Commercialization Validated CMC Processes Patented Hydro-electro- Crystallization Proprietary Trade Secrets
Anticipated Timeline & Upcoming Milestones 2020 - 2024 VISIONARY-MS
Nanotherapeutics Platform Potential first-in-class nanotherapeutic with high catalytic activity to drive energy production and utilization in stressed CNS cells Applications across neurology, infectious disease, and oncology Lead Asset: CNM-Au8 for Neurorepair CNM-Au8 improves cellular energy production and utilization to promote neuroprotection and remyelination Phase 2 ALS proof-of-concept evidence of clinical meaningful benefit Phase 3 Healey ALS platform trial results expected in 2H 2022 Phase 2 VISIONARY-MS trial results expected 2H 2022 Strong Execution Capabilities Proprietary electrochemical manufacturing process produces nanotherapeutics, scalable to commercialization Strong IP, including 150+ granted patents, and trade secrets CLENE | Company Highlights
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