What changed in this filing

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Item 1A. Risk Factors

2020-07-30 compared with 2020-04-29 · 38 added, 13 removed, 686 unchanged (7% of the section changed)

6 unchanged sentences

This could cause the value of our securities to decline, and you may lose all or part of your investment.

Risks Related to the CSL Behring Collaboration and License Transaction

In June 2020, uniQure biopharma BV, our wholly-owned subsidiary, entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”) providing CSL Behring exclusive global rights to etranacogene dezaparvovec, our investigational gene therapy for patients with hemophilia B.

We and CSL Behring may be unable to close the transaction, and any delay in completing the transaction could diminish the anticipated benefits of the transaction or result in increased costs.

Failure to close the transaction could adversely impact the market price of our shares as well as our business and operating results, cash flows and results of operations.

The closing of the transaction is contingent on completion of the successful reviews under antitrust laws in the United States, Australia, and the United Kingdom, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended.

We cannot make any assurances that the transaction will be closed on the timeline currently contemplated or at all, or that, as part of the regulatory review process, that additional conditions or terms may be required.

While we and CSL Behring intend to pursue vigorously all required governmental clearances, the requirement to receive these clearances before the closing of the transaction could delay the transaction or result in an inability to complete the transaction.

Any delay in the completion of the transaction could diminish anticipated benefits of the transaction, including realization of expected benefits of partnering, result in additional transaction costs, loss of revenue or other effects associated with uncertainty about the transaction.

Any such delay could also delay the timelines associated with our commercialization of etranacogene dezaparvovec, including the filing of a biologics licensing application with the FDA, and such delays could cause us to bring etranacogene dezaparvovec to market after a competitive product in the United States, Europe or in other markets.

We will need to fund the investments into the development and preparation of the commercial launch of etranacogene dezaparvovec for as long as the regulatory reviews continue.

The completions of these reviews could require significant time and/or might result in modifications or even denial of the transactions.

These factors could adversely impact the cash flows and results that we are able to generate in relation to etranacogene dezaparvovec.

To the extent that the market price of the ordinary shares of our common stock reflects positive market assumptions that the transaction will close or that the transaction is advantageous to us, the price of such shares may decline if the transaction does not close for any reason or in a timely manner.

The announcement and pendency of the transaction with CSL Behring could adversely affect our business and operations.

Whether the transaction is ultimately closed or not, its announcement and pendency could have a number of negative effects on our current business, including potentially disrupting our regular operations, diverting the attention of our workforce and management team, or increasing workforce turnover.

The completion of the transaction, including, for example, efforts to obtain regulatory clearances, may require significant time and attention from our management and divert attention from the day-to-day operations of our business.

Any uncertainty over the ability of us and CSL Behring to complete the transaction could make it more difficult for us to retain certain key employees or attract new talent or to pursue business strategies.

Parties with which we have business relationships related to etranacogene dezaparvovec, either contractual or operational, may experience uncertainty as to the future or desirability of such relationships and may delay or defer certain business decisions, seek alternative relationships with third parties or seek to alter their present business relationships with us.

Parties with whom we otherwise may have sought to establish business relationships may be reluctant to enter into agreements with us.

Risks Related to the Current COVID-19 Pandemic

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In addition, the COVID-19 pandemic has affected and may continue to affect the operations of the FDA, EMA and other health authorities, which could result in delays of reviews and approvals, including with respect to our product candidates.

As evidenced by the postponement of procedures for two patients in our Phase I/II clinical study of AMT-130, the evolving COVID-19 pandemic has impacted the pace of enrollment in our clinical trials.

As evidenced by the postponement of procedures for two patients in our Phase I/II clinical study of AMT-130, the evolving COVID-19 pandemic has impacted the pace of enrollment and procedures in our clinical trials, as well as caused challenges in scheduling follow-up visits and managing other aspects of our clinical trials.

We may be affected by similar delays as patients may avoid or may not be able to travel to healthcare facilities and physicians’ offices unless due to a health emergency and clinical trial staff can no longer get to the clinic.

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All of our product candidates are in research or development.

We have not generated any significant revenues from the sale of products and do not expect to generate any revenue before 2022.

We have not generated any significant revenues from the sale of products or manufacturing of our product for a licensee and do not expect to generate any such revenue before 2022.

Our lead product candidates, etranacogene dezaparvovec (also known as AMT-061) and AMT-130, and any of our other potential product candidates will require extensive preclinical and/or clinical testing and regulatory approval prior to commercial use.

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In addition, there is the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biological activity of the genetic material or other components of products used to carry the genetic material.

As of March 31, 2020, a total of three patients reported serious adverse events related to the treatment of AMT-060, our first generation hemophilia B gene therapy, in our Phase I/II trial, including one patient with a short, self-limiting fever in the first 24 hours after treatment and two patients with mild, asymptomatic elevations in liver transaminases.

As of June 30, 2020, a total of three patients reported serious adverse events related to the treatment of AMT-060, our first generation hemophilia B gene therapy, in our Phase I/II trial, including one patient with a short, self-limiting fever in the first 24 hours after treatment and two patients with mild, asymptomatic elevations in liver transaminases.

Additionally, one patient in our ongoing Phase IIb study of etranacogene dezaparvovec underwent hip surgery due to a pre-existing condition and was treated perioperatively with short-acting factor replacement.

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Additionally, we may not be able to scale up some or all of our manufacturing processes that may results in delays in regulatory approvals or otherwise adversely affect our ability to manufacture sufficient amounts of our products.

Many factors common to the manufacturing of most biologics and drugs could also cause production interruptions, including raw materials shortages, raw material failures, growth media failures, equipment malfunctions, facility contamination, labor problems, natural disasters, disruption in utility services, terrorist activities, or cases of force majeur and acts of god (including the effects of the COVID-19 pandemic) beyond our control.

Many factors common to the manufacturing of most biologics and drugs could also cause production interruptions, including raw materials shortages, raw material failures, growth media failures, equipment malfunctions, facility contamination, labor problems, natural disasters, disruption in utility services, terrorist activities, or cases of force majeure and acts of god (including the effects of the COVID-19 pandemic) beyond our control.

We also may encounter problems in hiring and retaining the experienced specialized personnel needed to operate our manufacturing process, which could result in delays in our production or difficulties in maintaining compliance with applicable regulatory requirements.

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In the event of an accident or failure to comply with environmental, occupational health and safety and export control laws and regulations, we could be held liable for damages that result, and any such liability could exceed our assets and resources, and could result in material harm to our business, financial condition and results of operations.

Our resources might be adversely affected if we are unable to meet our product supply needs and obligations.

To meet our expected future production needs, we will need to complete the validation of our existing manufacturing processes as well as to develop larger scale manufacturing processes.

We might be unable to successfully complete the validation or development to sufficiently meet our future production needs.

As a result, we may need to dedicate more of our resources to complete the validation or development, which could adversely impact our ability to develop our other proprietary programs, to meet our production needs, to conserve our cash, or to receive financial payments pursuant to our agreement with third parties, including with CSL Behring in return for supplying etranacogene dezaparvovec following regulatory approval.

Risks Related to Regulatory Approval of Our Products

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The success of our product candidates will depend on many factors, including:

● closing and successful execution of our transaction with CSL Behring for the commercialization of etranacogene dezaparvovec;

● successful completion of preclinical studies and clinical trials, and other work required by regulators;

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We have incurred significant losses to date, expect to incur losses over the next several years and may never achieve or maintain profitability.

We had a net loss of $28.0 million in the three months ended March 31, 2020, $124.2 million in the full year 2019 and $83.3 million in the full year 2018.

As of March 31, 2020, we had an accumulated deficit of $687.7 million.

We had a net loss of $70.6 million in the six months ended June 30, 2020, $124.2 million in the full year 2019 and $83.3 million in the full year 2018.

As of June 30, 2020, we had an accumulated deficit of $730.3 million.

To date, we have financed our operations primarily through the sale of equity securities and convertible debt, venture loans, through upfront payments from our collaboration partners and, to a lesser extent, subsidies and grants from governmental agencies and fees for services.

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We expect to continue to incur significant expenses and losses over the next several years, and our net losses may fluctuate significantly from quarter to quarter and year to year.

Our losses will be materially impacted by the amount of license revenue that we will recognize in accordance with ASC 606 following the potential closing of our collaboration and license agreement with CSL Behring.

We anticipate that our expenses will increase substantially as we:

● Build-out our commercial infrastructure and seek marketing approval for any product candidates (including etranacogene dezaparvovec) that successfully complete clinical trials;

● Advance the clinical development of AMT-130, our Huntington’s disease gene therapy program;

● Continue to build-out our clinical, medical and regulatory capabilities;

● Build-out our commercial and medical affairs infrastructure and seek marketing approval for any product candidates (including etranacogene dezaparvovec in case our collaboration and license agreement with CSL Behring would not close) that successfully complete clinical trials;

● Advance multiple research programs related to gene therapy candidates targeting liver-directed and CNS diseases;

● Continue to expand, enhance and optimize our technology platform, including our manufacturing capabilities, next-generation viral vectors and promoters, and other enabling technologies;

● Continue to expand our employee base to support research and development, as well as general and administrative functions;

● Acquire or in-license rights to new therapeutic targets or product candidates;

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Failure to obtain capital when needed may force us to delay, limit or terminate our product development efforts or other operations which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.

We expect to incur significant expenses in connection with our on-going activities and that we will likely need to obtain substantial additional funding in connection with our continuing operations.

We expect to incur significant expenses in connection with our on-going activities and that we will likely need to obtain substantial additional funding in connection with our continuing operations, in particular if the CSL Behring transaction would not close.

In addition, we have based our estimate of our financing requirements on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect.

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Our existing and any future indebtedness could adversely affect our ability to operate our business.

As of March 31, 2020, we had $35.0 million of outstanding principal of borrowings under the 2018 Amended Facility, which we are required to repay in monthly principal installments from January 2022 through June 2023.

As of June 30, 2020, we had $35.0 million of outstanding principal of borrowings under the 2018 Amended Facility, which we are required to repay in monthly principal installments from January 2022 through June 2023.

We could in the future incur additional debt obligations beyond our borrowings from Hercules.

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Our share price has been and may in the future be volatile.

From the start of trading of our ordinary shares on the Nasdaq Global Select Market on February 4, 2014 through April 27, 2020, the sale price of our ordinary shares ranged from a high of $82.49 to a low of $4.72.

The closing price on April 27, 2020, was $64.06 per ordinary share.

From the start of trading of our ordinary shares on the Nasdaq Global Select Market on February 4, 2014 through July 28, 2020, the sale price of our ordinary shares ranged from a high of $82.49 to a low of $4.72.

The closing price on July 28, 2020, was $39.52 per ordinary share.

The stock market in general and the market for smaller biopharmaceutical companies in particular, have experienced extreme volatility that has often been unrelated to the operating performance of particular companies.

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Our directors, executive officers and major shareholders, if they choose to act together, will continue to have a significant degree of control with respect to matters submitted to shareholders for approval.

Our directors, executive officers and major shareholders holding more than 5% of our outstanding ordinary shares, in the aggregate, beneficially own approximately 46.3% of our issued shares (including such shares to be issued in relation to exercisable options to purchase ordinary shares) as at March 31, 2020.

Our directors, executive officers and major shareholders holding more than 5% of our outstanding ordinary shares, in the aggregate, beneficially own approximately 46.0% of our issued shares (including such shares to be issued in relation to exercisable options to purchase ordinary shares) as at June 30, 2020.

As a result, if these shareholders were to choose to act together, they may be able, as a practical matter, to control many matters submitted to our shareholders for approval, as well as our management and affairs.

83 unchanged sentences

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1A. Risk Factors