UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
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TABLE OF CONTENTS
PTC Therapeutics, Inc.
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4 | |
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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations | 42 |
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Item 3. Quantitative and Qualitative Disclosures About Market Risk | 63 |
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63 | |
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64 | |
64 | |
65 | |
66 |
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FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
The forward-looking statements in this Quarterly Report on Form 10-Q include, among other things, statements about:
| ● | our ability to negotiate, secure and maintain adequate pricing, coverage and reimbursement terms and processes on a timely basis, or at all, with third-party payors for our products or product candidates that we commercialize or may commercialize in the future; |
| ● | our ability to maintain our conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD, in the European Economic Area, or EEA, and our marketing authorizations in other jurisdictions in which Translarna has been approved, including whether the European Medicines Agency, or EMA, determines in the re-examination process of the Committee for Medicinal Products for Human Use’s negative opinion that the benefit-risk balance of Translarna authorization supports renewal of such authorization; |
| ● | our ability to utilize results from Study 041 to support a renewal of the conditional marketing authorization for Translarna for the treatment of nmDMD in the EEA and to support a marketing approval for Translarna for the treatment of nmDMD in the United States; |
| ● | expectations with respect to our ability to commercialize UpstazaTM (eladocagene exuparvovec) for the treatment of Aromatic L-Amino Acid Decarboxylase, or AADC deficiency, in the EEA, any potential regulatory submissions and potential approvals for our product candidates, our manufacturing capabilities and the potential financial impact and benefits of our leased biologics manufacturing facility and the potential achievement of development, regulatory and sales milestones and contingent payments that we may be obligated to make; |
| ● | the anticipated period of market exclusivity for Emflaza® (deflazacort) for the treatment of Duchenne muscular dystrophy in the United States under the Orphan Drug Act of 1983; |
| ● | our expectations with respect to the commercial status of Evrysdi® (risdiplam) and our program directed against spinal muscular atrophy in collaboration with F. Hoffmann La Roche Ltd and Hoffmann La Roche Inc. and the Spinal Muscular Atrophy Foundation and our estimates regarding future revenues from sales-based royalty payments or the achievement of milestones in that program; |
| ● | our expectations and the potential financial impact and benefits related to our Collaboration and License Agreement with a subsidiary of Ionis Pharmaceuticals, Inc. including with respect to the timing of regulatory approval of Tegsedi® (inotersen) and WaylivraTM (volanesorsen) in countries in which we are licensed to commercialize them, the commercialization of Tegsedi and Waylivra, and our expectations with respect to royalty payments by us based on our potential achievement of certain net sales thresholds; |
| ● | the timing and scope of our commercialization of our products and product candidates; |
| ● | our estimates regarding the potential market opportunity for our products or product candidates, including the size of eligible patient populations and our ability to identify such patients; |
| ● | our ability to obtain additional and maintain existing reimbursed named patient and cohort early access programs for our products on adequate terms, or at all; |
1
| ● | our estimates regarding expenses, future revenues, third-party discounts and rebates, capital requirements and needs for additional financing, including our ability to maintain the level of our expenses consistent with our internal budgets and forecasts and to secure additional funds on favorable terms or at all; |
| ● | the timing and conduct of our ongoing, planned and potential future clinical trials and studies in our splicing, metabolic, Bio-e and oncology programs as well as studies in our products for maintaining authorizations, label extensions and additional indications, including the timing of initiation, enrollment and completion of the trials and the period during which the results of the trials will become available; |
| ● | our ability to realize the anticipated benefits of our acquisitions or other strategic transactions, including the possibility that the expected impact of benefits from the acquisitions or strategic transactions will not be realized or will not be realized within the expected time period, significant transaction costs, the integration of operations and employees into our business, our ability to obtain marketing approval of our product candidates we acquired from the acquisitions or other strategic transactions and unknown liabilities; |
| ● | the rate and degree of market acceptance and clinical utility of any of our products or product candidates; |
| ● | the ability and willingness of patients and healthcare professionals to access our products and product candidates through alternative means if pricing and reimbursement negotiations in the applicable territory do not have a positive outcome; |
| ● | the timing of, and our ability to obtain additional marketing authorizations for our products and product candidates; |
| ● | the ability of our products and our product candidates to meet existing or future regulatory standards; |
| ● | our ability to complete Study 041, a multicenter, randomized, double-blind, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, according to the protocol agreed with the EMA; |
| ● | the potential receipt of revenues from future sales of our products or product candidates; |
| ● | our sales, marketing and distribution capabilities and strategy, including the ability of our third-party manufacturers to manufacture and deliver our products and product candidates in clinically and commercially sufficient quantities and the ability of distributors to process orders in a timely manner and satisfy their other obligations to us; |
| ● | our ability to establish and maintain arrangements for the manufacture of our products and product candidates that are sufficient to meet clinical trial and commercial launch requirements; |
| ● | the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; |
| ● | our ability to complete any post-marketing requirements imposed by regulatory agencies with respect to our products; |
| ● | our expectations with respect to the potential financial impact and benefits of our leased biologics manufacturing facility and our ability to satisfy our obligations under the terms of the lease agreement for such facility; |
| ● | our ability to satisfy our obligations under the indenture governing our 1.50% convertible senior notes due September 15, 2026; |
| ● | our regulatory submissions, including with respect to timing and outcome of regulatory review; |
| ● | our plans to advance our earlier stage programs and pursue research and development of other product candidates, including our splicing, metabolic, Bio-e and oncology programs; |
2
| ● | our expectations with respect to the COVID-19 pandemic and related response measures and their effects on our business, operations, clinical trials, potential regulatory submissions and approvals, our collaborators, contract research organizations, suppliers and manufacturers; |
| ● | whether we may pursue business development opportunities, including potential collaborations, alliances, and acquisition or licensing of assets and our ability to successfully develop or commercialize any assets to which we may gain rights pursuant to such business development opportunities; |
| ● | the potential advantages of our products and any product candidate; |
| ● | our intellectual property position; |
| ● | the impact of government laws and regulations; |
| ● | the impact of litigation that has been or may be brought against us or of litigation that we are pursuing against others; and |
| ● | our competitive position. |
We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. We have included important factors in the cautionary statements included in this Quarterly Report on Form 10-Q, particularly in Part II, Item 1A. Risk Factors as well as in Part I, Item 1A. Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2022, that we believe could cause actual results or events to differ materially from the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make.
You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the year ended December 31, 2022 completely and with the understanding that our actual future results may be materially different from what we expect. We do not assume any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by applicable law.
In this Quarterly Report on Form 10-Q, unless otherwise stated or the context otherwise requires, references to “PTC,” “PTC Therapeutics,” “the Company,” “we,” “us,” “our,” and similar references refer to PTC Therapeutics, Inc. and, where appropriate, its subsidiaries. The trademarks, trade names and service marks appearing in this Quarterly Report on Form 10-Q are the property of their respective owners.
All website addresses given in this Quarterly Report on Form 10-Q are for information only and are not intended to be an active link or to incorporate any website information into this document.
3
PART I—FINANCIAL INFORMATION
Item 1. Financial Statements.
PTC Therapeutics, Inc.
Consolidated Balance Sheets (unaudited)
In thousands (except shares)
September 30, | December 31, | ||||||
| 2023 |
| 2022 | ||||
Assets | |||||||
Current assets: |
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Cash and cash equivalents | $ | | $ | | |||
Marketable securities |
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Trade and royalty receivables, net |
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Inventory, net |
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Prepaid expenses and other current assets |
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Total current assets |
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Fixed assets, net |
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Intangible assets, net |
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Goodwill |
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Operating lease ROU assets | | | |||||
Deposits and other assets |
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Total assets | $ | | $ | | |||
Liabilities and stockholders’ deficit |
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Current liabilities: |
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Accounts payable and accrued expenses | $ | | $ | | |||
Deferred revenue |
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Operating lease liabilities- current | | | |||||
Finance lease liabilities- current | | | |||||
Liability for sale of future royalties- current | | | |||||
Total current liabilities |
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Long-term debt |
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Contingent consideration payable |
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Deferred tax liability |
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Operating lease liabilities- noncurrent | | | |||||
Finance lease liabilities- noncurrent | | | |||||
Liability for sale of future royalties- noncurrent | | | |||||
Other long-term liabilities | | | |||||
Total liabilities |
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Stockholders’ deficit: |
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Common stock, $ |
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Additional paid-in capital |
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Accumulated other comprehensive income |
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Accumulated deficit |
| ( |
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Total stockholders’ deficit |
| ( |
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Total liabilities and stockholders’ deficit | $ | | $ | | |||
See accompanying unaudited notes.
4
PTC Therapeutics, Inc.
Consolidated Statements of Operations (unaudited)
In thousands (except shares and per share amounts)
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||
| 2023 |
| 2022 |
| 2023 |
| 2022 | ||||||
Revenues: |
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Net product revenue | $ | | $ | | $ | | $ | | |||||
Collaboration revenue |
| — | |
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Royalty revenue | | | | | |||||||||
Manufacturing revenue | | — | | — | |||||||||
Total revenues |
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Operating expenses: |
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Cost of product sales, excluding amortization of acquired intangible assets |
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Amortization of acquired intangible assets |
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Research and development |
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Selling, general and administrative |
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Change in the fair value of contingent consideration |
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Intangible asset impairment | — | — | | — | |||||||||
Total operating expenses |
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Loss from operations |
| ( |
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Interest expense, net |
| ( | ( |
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Other expense, net |
| ( | ( |
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Loss before income tax benefit |
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| ( |
| ( |
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Income tax benefit |
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Net loss attributable to common stockholders | $ | ( | $ | ( | $ | ( | $ | ( | |||||
Weighted-average shares outstanding: | |||||||||||||
Basic and diluted (in shares) |
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Net loss per share—basic and diluted (in dollars per share) | $ | ( | $ | ( | $ | ( | $ | ( | |||||
See accompanying unaudited notes.
5
PTC Therapeutics, Inc.
Consolidated Statements of Comprehensive Loss (unaudited)
In thousands
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||
| 2023 |
| 2022 |
| 2023 |
| 2022 | ||||||
Net loss | $ | ( | $ | ( | $ | ( | $ | ( | |||||
Other comprehensive income (loss): |
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Unrealized gain (loss) on marketable securities, net of tax |
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Foreign currency translation gain, net of tax |
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Comprehensive loss | $ | ( | $ | ( | $ | ( | $ | ( | |||||
See accompanying unaudited notes.
6
PTC Therapeutics, Inc.
Consolidated Statements of Stockholders’ (Deficit) Equity (unaudited)
In thousands (except shares)
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| Accumulated |
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| Additional |
| other |
| Total | ||||||||||||
Three months ended September 30, 2023 | Common stock | paid-in |
| comprehensive | Accumulated | stockholders’ | |||||||||||
| Shares |
| Amount |
| capital |
| (loss) income |
| deficit |
| deficit | ||||||
Balance, June 30, 2023 | | $ | | $ | | $ | ( | $ | ( | $ | ( | ||||||
Exercise of options |
| | — | | — | — | | ||||||||||
Restricted stock vesting and issuance, net |
| | — | — | — | — | — | ||||||||||
Share-based compensation expense |
| — | — | | — | — | | ||||||||||
Net loss |
| — | — | — | — | ( | ( | ||||||||||
Comprehensive income |
| — | — | — | | — | | ||||||||||
Balance, September 30, 2023 |
| | $ | | $ | | $ | | $ | ( | $ | ( | |||||
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| Accumulated |
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| Additional |
| other |
| Total | ||||||||||||
Three months ended September 30, 2022 | Common stock | paid-in |
| comprehensive | Accumulated | stockholders’ | |||||||||||
| Shares |
| Amount |
| capital |
| income |
| deficit |
| deficit | ||||||
Balance, June 30, 2022 |
| | $ | | $ | | $ | | $ | ( | $ | ( | |||||
Exercise of options |
| | — | | — | — | | ||||||||||
Restricted stock vesting and issuance, net |
| | — | — | — | — | — | ||||||||||
Share-based compensation expense |
| — | — | | — | — | | ||||||||||
Net loss |
| — |
| — |
| — |
| — | ( |
| ( | ||||||
Comprehensive income |
| — |
| — |
| — |
| | — |
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Balance, September 30, 2022 |
| | $ | | $ | | $ | | $ | ( | $ | ( | |||||
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| Accumulated |
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| Additional |
| other |
| Total | ||||||||||||
Nine months ended September 30, 2023 | Common stock | paid-in |
| comprehensive | Accumulated | stockholders’ | |||||||||||
| Shares |
| Amount |
| capital |
| income |
| deficit |
| deficit | ||||||
Balance, December 31, 2022 |
| | $ | | $ | | $ | | $ | ( | $ | ( | |||||
Exercise of options |
| | | | — | — | | ||||||||||
Restricted stock vesting and issuance, net |
| | | — | — | — | | ||||||||||
Issuance of common stock in connection with an employee stock purchase plan |
| | — | | — | — | | ||||||||||
Issuance of common stock in connection with a milestone payable | | | | — | — | | |||||||||||
Share-based compensation expense |
| — | — | | — | — | | ||||||||||
Net loss |
| — | — | — | — | ( | ( | ||||||||||
Comprehensive income |
| — | — | — | | — | | ||||||||||
Balance, September 30, 2023 |
| | $ | | $ | | $ | | $ | ( | $ | ( | |||||
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| Accumulated |
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| Additional |
| other |
| Total | ||||||||||||
Nine months ended September 30, 2022 | Common stock | paid-in |
| comprehensive | Accumulated | stockholders’ | |||||||||||
| Shares |
| Amount |
| capital |
| (loss) income |
| deficit |
| equity (deficit) | ||||||
Balance, December 31, 2021 |
| | $ | | $ | | $ | ( | $ | ( | $ | | |||||
Exercise of options | |
| — |
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| — |
| — | | |||||||
Restricted stock vesting and issuance, net | |
| — |
| — |
| — |
| — | — | |||||||
Issuance of common stock in connection with an employee stock purchase plan |
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| — |
| — |
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Share-based compensation expense |
| — |
| — |
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| — |
| — |
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Net loss |
| — |
| — |
| — |
| — |
| ( |
| ( | |||||
Comprehensive income |
| — |
| — |
| — |
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| — |
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Balance, September 30, 2022 |
| | $ | | $ | | $ | | $ | ( | $ | ( | |||||
See accompanying unaudited notes.
7
PTC Therapeutics, Inc.
Consolidated Statements of Cash Flows (unaudited)
In thousands
Nine Months Ended September 30, | |||||||
| 2023 |
| 2022 | ||||
Cash flows from operating activities | |||||||
Net loss | $ | ( | $ | ( | |||
Adjustments to reconcile net loss to net cash used in operating activities: |
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Depreciation and amortization |
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Non-cash operating lease expense |
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Non-cash royalty revenue related to sale of future royalties | ( | ( | |||||
Non-cash interest expense on liability related to sale of future royalties | | | |||||
Intangible asset impairment | | — | |||||
Change in valuation of contingent consideration |
| ( | ( | ||||
Unrealized loss on ClearPoint Equity Investments |
| | | ||||
Unrealized loss on ClearPoint convertible debt security | | | |||||
Unrealized (gain) loss on marketable securities- equity investments | ( | | |||||
Realized loss on the sale of Clearpoint Equity Investment | | — | |||||
Non-cash stock consideration, milestone payment | | — | |||||
Disposal of asset | | | |||||
Deferred income taxes | ( | — | |||||
Amortization of (discounts) premiums on investments, net |
| ( | | ||||
Amortization of debt issuance costs |
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Share-based compensation expense |
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Unrealized foreign currency transaction losses, net |
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Changes in operating assets and liabilities: |
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Inventory, net |
| ( | ( | ||||
Prepaid expenses and other current assets |
| | ( | ||||
Trade and royalty receivables, net |
| ( | ( | ||||
Deposits and other assets |
| | ( | ||||
Accounts payable and accrued expenses |
| | | ||||
Other liabilities |
| ( | ( | ||||
Deferred revenue |
| ( | — | ||||
Net cash used in operating activities | $ | ( | $ | ( | |||
Cash flows from investing activities |
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Purchases of fixed assets | $ | ( | $ | ( | |||
Purchases of marketable securities- available for sale | — | ( | |||||
Purchases of marketable securities- equity investments | ( | ( | |||||
Sale and redemption of marketable securities- available for sale | | | |||||
Sale and redemption of marketable securities- equity investments | | | |||||
Sale and redemption of ClearPoint Equity Investments | | — | |||||
Acquisition of product rights and licenses | ( | ( | |||||
Net cash (used in) provided by investing activities | $ | ( | $ | | |||
Cash flows from financing activities |
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Proceeds from exercise of options | $ | | $ | | |||
Repayment of Convertible Notes | — | ( | |||||
Proceeds from employee stock purchase plan |
| | | ||||
Debt issuance costs related to senior secured term loan | ( | — | |||||
Payment of finance lease principal | ( | ( | |||||
Net cash provided by (used in) financing activities | $ | | $ | ( | |||
Effect of exchange rate changes on cash |
| | ( | ||||
Net decrease in cash and cash equivalents |
| ( |
| ( | |||
Cash and cash equivalents, and restricted cash beginning of period |
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Cash and cash equivalents, and restricted cash end of period | $ | | $ | | |||
Supplemental disclosure of cash information |
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Cash paid for interest | $ | | $ | | |||
Cash paid for income taxes | | | |||||
Supplemental disclosure of non-cash investing and financing activity |
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Unrealized gain (loss) on marketable securities, net of tax | $ | | $ | ( | |||
Right-of-use assets obtained in exchange for operating lease obligations | — | | |||||
Acquisition of product rights and licenses | | | |||||
Milestone payable | | | |||||