11 unchanged sentences
Below is a summary of our recent significant business developments:
−Removed: As of December 31, 2020, a $35.0 million term loan was outstanding in accordance with the Second Amended and Restated Loan and Security Agreement (the “2018 Amended Facility”) between us and Hercules.
−Removed: On January 29, 2021, we and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”).
−Removed: Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”) increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million.
−Removed: On January 29, 2021, we drew down $35.0 million of the Tranche B.
−Removed: We may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021.
−Removed: Advances under Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum.
−Removed: The principal balance and all accrued but unpaid interest on advances under Tranche B is due on June 1, 2023, which date may be extended by us by up to two twelve-month periods.
−Removed: Advances under the 2021 Amended Facility may not be prepaid until July 29, 2021, following which we may prepay all such advances without charge.
−Removed: In addition to Tranche B, the 2021 Amended Facility also extends the interest only payment period of the previously funded $35.0 million term loan from January 1, 2022 to June l, 2023.
−Removed: End of term charges in respect of advances under the 2021 Amended Facility range from 1.65% to 6.85%, depending on the maturity date.
−Removed: The 2021 Amended Facility extended the funding of our operations until the second half of 2022.
−Removed: On March 1, 2021, we entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.
−Removed: We will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement.
−Removed: In March 2021, we issued 0.9 million ordinary shares at a weighted average price of $33.42 per share, with net proceeds of $27.6 million, after deducting underwriting discounts and net of offering expenses.
−Removed: This further extended the funding of our operations until the end of 2022.
+Added: Acquisition of Corlieve Therapeutics
+Added: On June 21, 2021, we entered into a share and purchase agreement (“SPA”) to acquire all outstanding shares of Corlieve Therapeutics S.A.S (“Corlieve”).
+Added: Corlieve’s lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (“TLE”).
+Added: TLE affects approximately 1.3 million people in the U.S.
+Added: and Europe alone, of which approximately 800,000 patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies.
+Added: Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life.
+Added: The lead program will be known as AMT-260.
+Added: The transaction is subject to customary closing conditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, des Finances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq.
+Added: of the French Code Monétaire et Financier.
+Added: Currently, the transaction is anticipated to be completed early in the third quarter of 2021.
+Added: Upon closing of the Corlieve Transaction we expect to pay EUR 46.3 million in cash to acquire all outstanding ordinary shares of Corlieve.
+Added: In accordance with the SPA we owe up to EUR 43.7 million in development milestones through Phase I/II and EUR 160.0 million in milestones associated with Phase III development and the approvals of AMT-260 in the U.S and European Union.
+Added: At our election we can pay up to 25% of such milestone payments in our ordinary shares.
+Added: The Corlieve transaction is expected to be accounted for as a business combination, which requires that assets acquired and liabilities assumed be recognized at their fair value as of the acquisition date.
+Added: Corlieve has an established license and collaboration agreement with REGENXBIO Inc.
+Added: that includes an exclusive license to AAV9 for the specific genetic target of AMT-260.
+Added: Under the license and collaboration agreement, REGENXBIO Inc.
+Added: is eligible to receive milestone payments and royalties on net sales of AMT-260.
CSL Behring commercialization and license agreement
−Removed: On June 24, 2020, uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (as amended, the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”) pursuant to which CSL Behring will receive exclusive global rights to etranacogene dezaparvovec, our investigational gene therapy for patients with hemophilia B (the “Product”).
−Removed: Under the terms of the CSL Behring Agreement, we are entitled to receive a $450.0 million upfront cash payment upon the closing of the CSL Behring Agreement and will be eligible to receive up to $1.6 billion in additional payments based on regulatory and commercial milestones.
−Removed: The CSL Behring agreement also provides that we will be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds.
−Removed: Pursuant to the CSL Behring Agreement, we will be responsible for the completion of the HOPE-B clinical trial, manufacturing process validation, and the manufacturing supply of the Product until such time that these capabilities may be transferred to CSL Behring or its designated contract manufacturing organization.
−Removed: Concurrently with the execution of the CSL Behring Agreement, we and CSL Behring entered into a development and commercial supply agreement, pursuant to which, among other things, we will supply the Product to CSL Behring at an agreed-upon price.
−Removed: Clinical development and regulatory activities performed by us pursuant to the CSL Behring Agreement will be reimbursed by CSL Behring.
−Removed: CSL Behring will be responsible for global regulatory submissions and commercialization requirements for the Product.
−Removed: The effectiveness of the transaction contemplated by the CSL Behring Agreement was contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom.
−Removed: As of March 31, 2021, the transaction had been cleared by the Australian and United Kingdom antitrust authorities and on May 6, 2021, the CSL Behring Agreement became fully effective after the expiration of the waiting period under the HSR Act on May 5, 2021.
−Removed: Under the CSL Behring Agreement, the upfront payment of $450.0 million was paid to us on May 7, 2021.
−Removed: We are also eligible to receive up to more than $300 million in regulatory and first commercial sale milestones, $1.3 billion in additional commercial milestones, and tiered double-digit royalties of up to a low-twenties percentage of net product sales arising from the collaboration.
−Removed: We contractually owe a single-digit percentage of revenue from the collaboration to our licensors.
−Removed: We expect to utilize a material portion of our net operating loss carryforwards in the Netherlands during 2021 as a result of closing the transaction.
−Removed: The upfront payment, net of payments owed to licensors and other parties, is expected to extend the funding of our operations into the second half of 2024 (assuming a full repayment of funds borrowed from Hercules Growth Capital, Inc.
−Removed: under our term loan facilities by 2023).
−Removed: The receipt of the near-term milestones would further expand the funding of our operations.
−Removed: However, we expect to continue to incur losses and to generate negative cash flows beyond 2021, the fiscal year in which we closed the transaction.
+Added: On June 24, 2020, (“Signing”), uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (as amended, the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”) pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, our investigational gene therapy for patients with hemophilia B (the “Product”).
+Added: The effectiveness of the transaction contemplated by the CSL Behring Agreement was contingent on the completion of review under the antitrust laws in the United States, Australia, and the United Kingdom.
+Added: The transaction had previously been cleared by the Australian and United Kingdom antitrust authorities and became fully effective on May 6, 2021, after the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 expired on May 5, 2021.
+Added: As from May 6, 2021, CSL Behring will be responsible for the development and commercialization of the Product.
+Added: We agreed to complete the validation of the current manufacturing process as well as to complete the development and validation of a next generation manufacturing process.
+Added: We will be entitled to receive a development milestone payment if we complete these activities in accordance with an agreed development plan and timeline.
+Added: CSL Behring will also be reimbursing us for our services to facilitate the completion of the HOPE-B clinical trial.
+Added: On Signing, we and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we will supply the Product to CSL Behring at an agreed-upon price commensurate with the stand-alone selling price (“SSP”).
+Added: We will be responsible to supply the Product until such time that these capabilities may be transferred to CSL Behring or its designated contract manufacturing organization.
+Added: We recorded $462.4 million, including a $450.0 million upfront cash payment following the closing of the CSL Behring Agreement as license revenue in the three and six months ended June 30, 2021.
+Added: We are eligible to receive more than $0.3 billion in regulatory, development, and first commercial sale milestones, $1.3 billion in additional commercial milestones, and tiered double-digit royalties of up to a low-twenties percentage of net product sales arising from the collaboration.
+Added: Upon closing, we contractually owed to our licensors $16.7 million of the upfront payment received from CSL Behring.
+Added: The payments we received significantly increased our cash and cash equivalents position to $677.3 million as of June 30, 2021.
+Added: This is expected to fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules Growth Capital, Inc.
+Added: under our term loan facilities by 2023, as well as payments we expected to make in relation to our acquisition of Corlieve).
+Added: We expect that the achievement of the $0.3 billion regulatory, first commercial sales and development milestones would further extend our runway by 12 to 18 months.
Hemophilia B program – Etranacogene dezaparvovec (AMT-061)
−Removed: Etranacogene dezaparvovec is our lead gene therapy candidate and includes an AAV serotype 5 (together “AAV-5”) vector incorporating the functional human Factor IX (“FIX”) Padua variant.
+Added: Etranacogene dezaparvovec is our lead gene therapy candidate and includes an AAV serotype 5 (“AAV-5”) vector incorporating the functional human Factor IX (“FIX”) Padua variant.
We are currently conducting a pivotal study in patients with severe and moderately-severe hemophilia B.
−Removed: In December 2020, we announced top-line data from the HOPE-B trial.
−Removed: The 26-week follow-up date from the trial showed that FIX activity in the 54 patients increased after dosing from ≤ 2% to a mean of 37.2% at 26 weeks, meeting a first primary endpoint of the HOPE-B trial.
−Removed: No correlation between pre-existing neutralizing antibodies and FIX activity was found in patients with neutralizing antibody titers up to 678.2, a range expected to include more than 95% of the general population;
−Removed: one patient with a neutralizing antibody titer of 3,212.3 did not show an increase in FIX activity.
−Removed: Less than 1% of the general population is expected to have neutralizing antibody titers of greater than 3,000.
−Removed: During the 26-week period after dosing, 15 patients (28%) reported a total of 21 bleeding events, representing a reduction of 83% compared to the 123 bleeding events reported by 38 patients (70%) during the observational lead-in phase of the trial.
−Removed: Total bleeds include any bleeding event reported after the treatment of etranacogene dezaparvovec, including spontaneous, traumatic, and those associated with unrelated medical procedures, whether or not FIX treatment was required.
−Removed: Of the total bleeding events reported during the 26-week period after dosing, only three were classified as spontaneous bleeds requiring treatment, representing a reduction of 92% compared to the 37 such bleeding events reported during the observational lead-in phase.
−Removed: Mean annualized usage of FIX replacement therapy, a secondary endpoint in the clinical trial, declined by 96% during the 26-week period after dosing compared to the observational lead-in phase.
−Removed: Etranacogene dezaparvovec was generally well-tolerated.
−Removed: As of the November 2020 cut-off date, most adverse events were classified as mild (81.5%).
−Removed: The most common events included transaminase elevation treated with steroids per protocol (9 patients;
−Removed: 17%), infusion-related reactions (7 patients;
−Removed: 13%), headache (7 patients;
−Removed: 13%) and influenza-like symptoms (7 patients;
−Removed: Liver enzyme elevations resolved with a tapering course of corticosteroids and FIX activity remained in the mild range in the steroid treated patients.
−Removed: No relationship between safety and neutralizing antibody titers was observed.
−Removed: Based on interactions with the U.S.
−Removed: Food and Drug Administration (“FDA”) and the European Medicines Agency (“EMA”), we plan to incorporate FIX activity and bleeding rates at 52 weeks as additional co-primary endpoints in the study.
−Removed: On December 21, 2020, our clinical trials of etranacogene dezaparvovec, including our HOPE-B trial were placed on clinical hold by the FDA.
−Removed: The clinical hold was initiated following the submission of a safety report in mid-December relating to a possibly related serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma (“HCC”), a form of liver cancer, in one patient in the HOPE-B trial that was treated with etranacogene dezaparvovec in October 2019.
−Removed: The patient has multiple risk factors associated with HCC, including a twenty-five-year history of hepatitis C, hepatitis B, evidence of non-alcoholic fatty liver disease and advanced age.
−Removed: Chronic infections with hepatitis B and C have been associated with approximately 80% of HCC cases.
−Removed: No other cases of HCC have been reported in our clinical trials conducted in more than 67 patients in hemophilia B, with some patients dosed more than 5 years ago.
−Removed: On March 26, 2021, we submitted the results of a comprehensive investigation into the case of HCC to the FDA.
−Removed: The investigation found that it is highly unlikely the HCC was caused by etranacogene dezaparvovec.
−Removed: Multiple analyses conducted by an independent laboratory and reviewed by leading external experts in the field show that AAV vector integration in the patient’s tissue sample was extremely rare and accounted for 0.027% of the cells in the sample.
−Removed: The integration events were distributed randomly across the genome, and there was no evidence of clonal expansion or any dominant integration event.
−Removed: Additionally, whole genome sequencing of the tumor confirmed that the patient had several genetic mutations that are characteristic of HCC and are independent of vector integration.
−Removed: Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.
−Removed: On April 23, 2021, the FDA informed us that the clinical hold on our hemophilia B gene therapy program is removed after determining that we had satisfactorily addressed all issues identified.
+Added: On June 22, 2021, we presented new data from the HOPE-B pivotal study related to the 52-week period post-infusion.
+Added: Data showed that participants continued to demonstrate durable, sustained increases in FIX activity at 52-weeks post-infusion with a mean FIX activity of 41.5 percent of normal, as measured by a one-stage APTT-based clotting assay, compared to a mean FIX activity of 39.0 percent of normal at 26-weeks of follow-up.
+Added: During the 52-week period, a single dose of etranacogene dezaparvovec significantly reduced the annualized rate of bleeding requiring treatment by 80 percent from a prospectively collected 3.39 at baseline to 0.68 bleeding episodes per year (p-value <0.0001).
+Added: The annualized rate of spontaneous bleeding requiring treatment was also significantly reduced by 85 percent from a prospectively collected 1.16 at baseline to 0.18 bleeds per year during the 52-week period (p-value <0.0001).
+Added: Usage of FIX replacement therapy (IU/year and infusions/year) in all patients declined 96 percent during the 52-week period, with 52 of 54 patients (96 percent) successfully discontinuing their prophylactic infusions.
+Added: As previously announced, of the two non-responders, one patient only received a partial dose (less than 10 percent of the dosage) due to an infusion reaction and a second patient had an unusually high pre-existing NAb titer of 3,212, which is expected in less than 1 percent of the general population.
+Added: We expect all patients to complete their 78-week follow-up visits by the end of the third quarter of 2021, and we expect a BLA to be submitted to the FDA and a MAA to be submitted to EMA in the first quarter of 2022.
+Added: On December 21, 2020, our clinical trials of etranacogene dezaparvovec had been placed on clinical hold by the FDA following a preliminary diagnosis of hepatocellular carcinoma (“HCC”), a form of liver cancer, in one patient in the HOPE-B trial who was treated with etranacogene dezaparvovec.
+Added: On March 26, 2021, we submitted to the FDA the results of a comprehensive investigation that found it is highly unlikely the HCC was caused by etranacogene dezaparvovec.
+Added: On April 23, 2021, the FDA informed us that the clinical hold on our hemophilia B gene therapy program was removed after determining that we had satisfactorily addressed all issues identified.
Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the FDA and access to the current priority medicines (“PRIME”) initiative by the EMA.
3 unchanged sentences
AMT-130 has received orphan drug and Fast Track designations from the FDA and Orphan Medicinal Product Designation from the EMA.
−Removed: In June 2020, we announced the completion of the first two patient procedures in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease.
−Removed: In October 2020 we completed the third and fourth patients’ procedures.
−Removed: In February 2021, the independent Data Safety Monitoring Board (“DSMB”) overseeing the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease met.
−Removed: No significant safety concerns were noted to prevent further dosing.
−Removed: On April 5, 2021 we announced that we completed the procedures for the fifth to tenth patients completing the treatment of the first cohort.
−Removed: On April 5, 2021 we also announced the initiation of a Phase 1b/II clinical trial in Europe.
+Added: On April 5, 2021 we announced the completion of enrollment of all ten patients in the first dose cohort of our Phase Ib/II clinical trial of AMT-130.
+Added: On May 27, 2021, we announced that the study’s independent Data Safety Monitoring Board (“DSMB”) recommended we continue patient dosing after reviewing safety data from all 10 patients in the first dose cohort.
+Added: On June 16, 2021, we announced that the first two patient procedures were completed in the second, higher-dose cohort, which will include 16 patients.
+Added: The Phase I/II study is a double-blinded, randomized, and controlled clinical trial being conducted in the United States.
+Added: To date, seven patients have been treated with AMT-130 across both dose cohorts, and five patients have received an imitation surgical procedure that will serve as a control.
+Added: On April 5, 2021 we also announced the initiation of a Phase Ib/II clinical trial in Europe.
The planned Phase Ib/II study is expected to begin enrolling patients in the second half of 2021.
2 unchanged sentences
study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible.
−Removed: COVID-19 measures
+Added: Research and Development Day
+Added: On June 22, 2021, we held a research and development day to outline our vision and strategy of leveraging our AAV engine to deliver gene therapies within our key focus areas.
+Added: We provided updates on our Fabry (AMT-191) and SCA3 (AMT-150) programs and announced additional new product candidates, including AMT-210 for Parkinson’s disease, AMT-240 for Alzheimer’s disease, and AMT-161 for Amyotrophic Lateral Sclerosis (“ALS”).
+Added: These new programs are key components of our plan to expand our clinical pipeline.
+Added: We also announced our plans to construct a second cGMP manufacturing facility located in Amsterdam, The Netherlands.
+Added: Covid pandemic
The coronavirus disease (“Covid”) caused by the severe acute respiratory syndrome coronavirus 2 (“Sars-CoV 2 virus”) was characterized as a pandemic by the World Health Organization (“WHO”) on March 11, 2020.
−Removed: During late 2020 various, potentially more infectious, variants of the Sars-CoV 2 virus causing COVID-19 were identified.
−Removed: Starting March 2020, we implemented measures to address the impact of COVID-19 on our business.
−Removed: We mandated a work-from-home policy for all non-essential employees at our Amsterdam and Lexington facilities when the pandemic began.
−Removed: We implemented a series of protocols governing the operations of our Lexington facility to comply with the requirements of the various orders and guidance from the Commonwealth of Massachusetts and other related orders, guidance, laws, and regulations.
+Added: Since then, various, potentially more infectious, variants of the Sars-CoV 2 virus causing Covid have been identified.
+Added: Throughout the pandemic, we have been implementing measures to address the impact of Covid on our business.
+Added: We have implemented a series of protocols governing the operations of our Lexington facility to comply with the requirements of the various orders and guidance from the Commonwealth of Massachusetts and other related orders, guidance, laws, and regulations.
We continue to monitor local government rules and recommendations and office protocols will be aligned with these rules and recommendations.
−Removed: To align with the Dutch government’s measures, we implemented a mandatory work-from-home policy in Amsterdam.
−Removed: Employees based in Amsterdam who cannot perform their duties outside of our Amsterdam facility will continue to work at our Amsterdam facility.
−Removed: We adapted to operate our laboratories at our Amsterdam site to comply with social distancing rules and to ensure the health and wellbeing of our employees under the current circumstances.
−Removed: All other employees in Amsterdam will work from home through at least the end of August 2021, partly in conjunction with the ongoing expansion of our laboratory space.
−Removed: As a biopharma research and development company, we were deemed to provide essential services under the “stay at home” advisory that was issued by the Governor of Massachusetts on March 23, 2020 and we therefore have maintained our manufacturing operations at our Lexington site.
−Removed: To ensure adequate social distancing in our Lexington facility, our COVID-19 protocols generally have limited occupancy to numbers below those allowed by the Massachusetts COVID-19 guidelines.
−Removed: In our Lexington facility, we currently have implemented an occupancy limitation of approximately 25%.
−Removed: Our employees that cannot perform their duties outside of our Lexington facility continue to work at our Lexington facility.
−Removed: All other employees are required to work remotely to the extent possible through at least the end of the second quarter of 2021.
−Removed: Our actual occupancy at the Lexington facility has been less than approximately 25% of our permitted occupancy during all phases of the Massachusetts reopening plan.
−Removed: We have also implemented a mandatory COVID-19 PCR testing protocol effective February 11, 2021 that requires employees to have tested negative for COVID-19 prior to entering the Lexington facility.
−Removed: We have adapted our ongoing clinical research activities based on the directions and flexibility provided by the “FDA Guidance on Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic” issued on March 18, 2020 and updated throughout the pandemic to minimize any risk, disruption, or delay in either patient dosing or follow-up visits.
−Removed: These procedures occurred after a postponement that resulted from the COVID-19 pandemic and the associated states of emergency declarations in the United States.
−Removed: The broader implications of COVID-19 on our results of operations and overall financial performance remain uncertain.
−Removed: The COVID-19 pandemic and its adverse effects have become more prevalent in the locations where we, and our third-party business partners conduct business.
−Removed: While we have experienced disruptions in our operations as a result of COVID-19, we are adapting to the current environment to minimize the effect to our business.
+Added: Accordingly, we had mandated a work-from-home policy since March 2020 for all non-essential employees at our Amsterdam and Lexington facilities and had implemented additional protocols for our essential employees.
+Added: Effective May 29, 2021, Massachusetts has lifted all industry restrictions, with the exceptions of remaining face-covering requirements for all public and private transportation systems and facilities housing vulnerable populations, and capacity has been increased to 100% for all industries.
+Added: Furthermore, the state of emergency was lifted on June 15, 2021.
+Added: Implementation of adequate cleaning and hygiene protocols are still encouraged.
+Added: We have implemented a mandatory Covid PCR testing protocol in our Lexington facility effective February 11, 2021 that requires employees to have tested negative for Covid prior to entering the facility.
+Added: Also in our Lexington facility, allowed occupancy has been increased to 100% in line with the permitted capacity guidelines and all employees are allowed to work at the Lexington facility subject to social distancing protocols, and a daily health screening and compliance with our Covid PCR testing policy for unvaccinated employees.
+Added: All employees that are not essential employees, however, remain able to work remotely until December 31, 2021, and we have been operating significantly below 100% capacity.
+Added: At least until August 1, 2021, for purposes of monitoring Covid surges due to relaxation of requirements, no employee or contractor may enter the Lexington facility unless they are either:
+Added: (i) fully vaccinated;
+Added: or (ii) have taken an approved test for Covid and obtained a negative result.
+Added: On June 26, 2021, the Dutch government announced updated measures.
+Added: Since July 1, we allow for an occupancy rate of 50% at our Amsterdam site as well as the limited presence of visitors.
+Added: We continue to operate our laboratories at our Amsterdam site to comply with social distancing rules and to ensure the health and well-being of our employees under the current circumstances.
+Added: Employees that can continue to work from home are encouraged to do so through at least August 2021, partly in conjunction with the ongoing expansion of our laboratory space.
+Added: The broader implications of Covid, including the implications from the various variants, on our results of operations and overall financial performance remain uncertain.
+Added: We have experienced increased lead times in the delivery of equipment and disposables that we use to manufacture materials for our various programs.
+Added: Currently, these have not materially impacted our development timelines and we continue to adapt to the current environment to minimize the effect to our business.
However, we may experience more pronounced disruptions in our operations in the future.
+Added: As of December 31, 2020, a $35.0 million term loan was outstanding in accordance with the Second Amended and Restated Loan and Security Agreement (the “2018 Amended Facility”) between us and Hercules Capital, Inc.
+Added: (“Hercules”).
+Added: On January 29, 2021, we and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”).
+Added: Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”) increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million.
+Added: On January 29, 2021, we drew down $35.0 million of the Tranche B.
+Added: We may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021.
+Added: Advances under Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum.
+Added: The principal balance and all accrued but unpaid interest on advances under Tranche B is due on June 1, 2023, which date may be extended by us by up to two twelve-month periods.
+Added: Advances under the 2021 Amended Facility may not be prepaid until July 29, 2021, following which we may prepay all such advances without charge.
+Added: In addition to Tranche B, the 2021 Amended Facility also extends the interest only payment period of the previously funded $35.0 million term loan from January 1, 2022 to June l, 2023.
+Added: End of term charges in respect of advances under the 2021 Amended Facility range from 1.65% to 6.85%, depending on the maturity date.
+Added: On March 1, 2021, we entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.
+Added: We will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement.
+Added: In March and April of 2021, we issued 921,730 ordinary shares at a weighted average price of $33.52 per share, with net proceeds of $29.6 million, after deducting underwriting discounts and net of offering expenses.
In February 2021 we commenced the expansion of our Amsterdam site to build additional laboratories to support the expansions of our research and development activities as well the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.
In May 2021, we entered into a sublease agreement to let an additional approximately 1,080 square meters of office space to accommodate the hiring of additional full-time employees.
−Removed: The lease expires in October 2028 and includes a break option until October 2023.
+Added: The lease expires in October 2028 and includes an option to break the lease on October 31, 2023.
+Added: On May 17, 2021, Pierre Caloz was appointed as Chief Operating Officer.
+Added: Caloz oversees all manufacturing operations, global CMC development and innovation, supply chain, and facilities.
+Added: On June 15, 2021, Christian Klemt was appointed as Chief Financial Officer.
+Added: Klemt was our Chief Accounting Officer from August 2017 to June 2021, and he will continue to serve as general manager of our Amsterdam site.
+Added: Matthew Kapusta, who has been our Chief Executive Officer since December 2016 and had been our Chief Financial Officer from January 2015 to June 2021, will continue to serve as our Chief Executive Officer.
+Added: In connection with his transition to Chief Financial Officer, Mr.
+Added: Klemt will also serve as our Principal Financial Officer.
+Added: On June 16, 2021, our shareholders voted to approve the reappointment of Mr.
+Added: David Meek and Ms.
+Added: Paula Soteropoulos as non-executive directors of the Board of Directors.
+Added: Meek has been appointed chairman of the Board.
+Added: Philip Astley-Sparke did not stand for reappointment and retired from the Board on June 16, 2021.
+Added: Intellectual Property
+Added: On May 11, 2021, Pfizer, Inc.
+Added: filed three petitions seeking Inter Partes Review of U.S.
+Added: 9,982,248 (the “’248 Patent”) and 10,465,180 (the “’180 Patent” and together with the ‘248 Patent, the “Patents”).
+Added: The petitions collectively seek to invalidate all claims of the Patents, which relate to the expression of factor IX protein using a modified “Padua” polypeptide having a leucine in position 338.
+Added: We are in the process of responding to the petitions.
Financial Overview
Key components of our results of operations include the following:
−Removed: Three months ended March 31,
+Added: Three months ended June 30,
+Added: Six months ended June 30,
(in thousands)
+Added: (in thousands)
Total revenues
+Added: Cost of contract revenue
Research and development expenses
Selling, general and administrative expenses
−Removed: As of March 31, 2021, and December 31, 2020, we had cash and cash equivalents of $260.8 million and $244.9 million, respectively.
−Removed: We had a net loss of $41.6 million in the three months ended March 31, 2021, compared to $28.0 million for the same period in 2020.
−Removed: As of March 31, 2021, and December 31, 2020, we had accumulated deficits of $826.3 million and $784.7 million, respectively.
−Removed: Our losses will be materially impacted by the amount of license revenue that we will recognize in accordance with ASC 606 as a result of the closing of the transaction contemplated under the CSL Behring Agreement, which became fully effective on May 6, 2021.
+Added: Net income / (loss)
+Added: As of June 30, 2021, and December 31, 2020, we had cash and cash equivalents of $677.3 million and $244.9 million, respectively.
+Added: We had a net income of $399.5 million and $357.9 million in the three and six months ended June 30, 2021, compared to a net loss of $42.6 million and $70.6 million for the same periods in 2020.
+Added: As of June 30, 2021, and December 31, 2020, we had accumulated deficits of $426.8 million and $784.7 million, respectively.
+Added: We recorded a net income in the three and six months ended June 30, 2021 as a result of the closing of the CSL Behring transaction on May 6, 2021.
We anticipate that our expenses will increase substantially as we:
1 unchanged sentence
● Advance multiple research programs related to gene therapy candidates targeting liver-directed and central nervous system (“CNS”) diseases;
+Added: ● 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;
● Continue to expand, enhance and optimize our technology platform, including our manufacturing capabilities, next-generation viral vectors and promoters, and other enabling technologies;
−Removed: ● Continue to expand our employee base to support research and development, as well as general and administrative functions;
● Maintain, expand, and protect our intellectual property portfolio, including in-licensing additional intellectual property rights from third parties.
−Removed: ● Build out our commercial and medical affairs infrastructure and seek marketing approval for any product candidates.
See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
6 unchanged sentences
In making estimates and judgments, management employs critical accounting policies.
−Removed: During the three months ended March 31, 2021, there were no material changes to our critical accounting policies as reported in our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the SEC on March 1, 2021 .
+Added: During the six months ended June 30, 2021, there were no material changes to our critical accounting policies as reported in our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the SEC on March 1, 2021 , with the exception of determining the accounting treatment in accordance with ASC 606 related to the CSL Behring Agreement as discussed below.
We believe that the assumptions, judgments, and estimates related to the treatment of the CSL Behring Agreement, the amended BMS CLA, share-based payments, corporate income taxes related to valuation allowance, and accounting for operating leases under ASC 842 to be our critical accounting policies.
−Removed: The preparation of our consolidated financial statements for the three-month period ended March 31, 2021, required us to analyze the accounting treatment of the CSL Behring Agreement.
−Removed: The effectiveness of the transaction contemplated by the CSL Behring Agreement was contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom.
−Removed: The review processes in Australia and the United Kingdom were completed prior to January 6, 2021.
−Removed: As of March 31, 2021, regulatory approval in the United Sates had not occurred.
−Removed: On May 5, 2021, the waiting period under the HSR Act expired and the transaction became fully effective on May 6, 2021.
−Removed: As of March 31, 2021, we concluded that we had no enforceable right to receive the $450.0 million upfront payment, in accordance with the CSL Behring Agreement as payment was contingent upon the successful completion of reviews under the HSR Act and the reviews were not completed by March 31, 2021.
−Removed: Therefore, we determined we would not recognize any revenue in relation to the upfront payment, the regulatory and sale milestone payments, or the royalties (together “CSL Behring License Revenue”) in accordance with ASC 606 during the three-month period ended March 31, 2021.
−Removed: We recognize deferred tax assets to the extent that we determine that these assets are more likely than not to be realized.
−Removed: We determined that recognition of the CSL Behring License Revenue in 2021 will consume a material portion of our net operating loss carryforwards in the Netherlands.
−Removed: However, we expect to continue to record operating losses in the years thereafter.
−Removed: Accordingly, we expect to continue to record a full valuation allowance in relation to our net operating loss carryforwards in the Netherlands as of the end of the current fiscal year.
−Removed: We determined the impact which recognition of CSL Behring License Revenue is estimated to have, with regards to the expected effective tax rate of 0% that was applied with respect to our Dutch operations as of March 31, 2021, is immaterial.
−Removed: Accordingly, we continued to record a full valuation allowance as of March 31, 2021, in relation to our net operating loss carryforwards in the Netherlands.
−Removed: We recognize collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that are reimbursable by BMS under the BMS CLA (as defined below) and the amended BMS CLA as well as other related agreements.
−Removed: Collaboration Revenue related to these contracted services is recognized when performance obligations are satisfied.
−Removed: We recognized license revenues associated with the amortization of the non-refundable upfront payment and target designation fees we received from BMS in 2015.
−Removed: We evaluated our outstanding performance obligation following the amendment of the BMS CLA on December 1, 2020 and determined that our remaining performance obligation is immaterial.
+Added: The preparation of our consolidated financial statements for the three- and six- month period ended June 30, 2021, required us to analyze the accounting treatment of the CSL Behring Agreement that we entered into on June 24, 2020 (“Signing”).
+Added: The effectiveness of the transaction had been contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom.
+Added: The transaction had previously been cleared by the Australian and United Kingdom antitrust authorities and became fully effective on May 6, 2021 (“Closing”), after the waiting period under the HSR Act expired on May 5, 2021.
+Added: We identified two material performance obligations related to the CSL Behring Agreement:
+Added: (i) Sale of the exclusive global rights to the Product (“License”);
+Added: (ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).
+Added: We continued to develop the Product between Signing and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” and together with the License the “License Sale”).
+Added: We determined that the fixed upfront payment of $450.0 million and the $12.4 million that we received in relation to the Additional Covenants should be allocated to the License Sale.
+Added: In addition, we concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well.
+Added: We determined that the sale of the License was completed on May 6, 2021, when we transferred the License and CSL Behring assumed full responsibility for the development and commercialization of the Product.
+Added: At Closing we evaluated the amount of potential payments and the likelihood that the payments will be received.
+Added: We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.
+Added: Since we cannot control the achievement of regulatory and first commercial sales milestones, we concluded that the potential payments are constrained as of Closing.
+Added: We determined that we would recognize revenue related to these payments, only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter.
+Added: We will include payments related to sales milestones in the transaction price when their achievement becomes probable, and we will include royalties on the sale of Product once these have been earned.
+Added: We recognized $462.4 million of revenues related to the License Sale in the three and six months ended June 30, 2021
+Added: We determined that the variable milestone payment related to Manufacturing Development Services should be allocated to the Manufacturing Development performance obligation.
+Added: We concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin.
+Added: The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan.
+Added: The variable consideration will be reduced if we do not complete the development by pre-agreed dates.
+Added: We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.
+Added: Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes.
+Added: Since we cannot control the timing or outcome of any regulatory decisions, we concluded that we would recognize revenue related to this payment when it becomes probable that the milestone has been achieved.
+Added: We did not recognize any revenue related to Manufacturing Development during the three and six months ended June 30, 2021.
+Added: We recognized $0.4 million of collaboration revenue in the three and six months ended June 30, 2021, compared to nil in the same periods in the prior year.
+Added: We generate such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring.
+Added: CSL Behring may request additional development services or to request us to support the transfer of manufacturing to a party designated by CSL Behring.
+Added: These collaboration services will be reimbursed at the pre-agreed full-time-employee rate (“FTE-rate”).
+Added: We concluded that these rights at Closing do not represent material rights.
+Added: We also analyzed the impact of Closing on our net deferred tax asset in the Netherlands.
+Added: Prior to Closing we had determined that the potential Closing would represent insufficient positive evidence to overcome the significant negative evidence of having been loss-making during the past three years.
+Added: Accordingly, we had therefore recorded a full valuation allowance related to our net deferred tax assets.
+Added: We determined that in accordance with Dutch tax law the upfront payment as well as payments related to the Additional Covenants, to the extend such activities were conducted in the 12 months ended December 31, 2020, can more likely than not be included in the Dutch tax return for the 12-month period ended December 31, 2020.
+Added: Including this income in our 2020 tax position will consume substantially all of our Dutch net operating losses for the years 2011 to 2018.
+Added: We treated the adjustment to our 2020 filing position resulting in the release of the valuation allowance related to our Dutch net operating losses for the years 2011 to 2018 as a discrete event in the three months ended June 30, 2021.
+Added: We allocated part of the release to the tax benefit of share issuance cost incurred in 2014, 2015, 2017 and 2018.
+Added: This resulted in an increase of additional paid- in capital as well as the recognition of deferred tax expenses of $3.0 million during the three and six months ended June 30, 2021.
+Added: Based on changes to Dutch tax law enacted in June 2021 our remaining net operating tax loss related to 2019 can be carried forward indefinitely.
+Added: We continue to expect incurring tax losses for the foreseeable future including 2021.
+Added: As such, we retain our full valuation allowance related to the remaining net deferred asset as of June 30, 2021.
+Added: We recognize revenue when the customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services.
+Added: To determine revenue recognition for arrangements that we determine are within the scope of ASC 606, we perform the following five steps:
+Added: (i) identify the contract with the customer;
+Added: (ii) identify the performance obligations in the contract;
+Added: (iii) determine the transaction price;
+Added: (iv) allocate the transaction price to the performance obligations in the contract;
+Added: and (v) recognize revenue when (or as) we satisfy a performance obligation.
+Added: We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer.
+Added: At contract inception, once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations and assess whether each promised good or service is distinct.
+Added: We then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.
+Added: We estimate the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract.
+Added: The consideration may include fixed consideration or variable consideration.
+Added: At inception of each arrangement that includes variable consideration, we evaluate the amount of potential payments and the likelihood that the payments will be received.
+Added: We utilize either the most likely amount method or expected amount method to estimate the amount expected to be received based on which method best predicts the amount expected to be received.
+Added: The amount of variable consideration included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur in a future period.
+Added: We allocate the transaction price based on the estimated SSP of each of the performance obligations.
+Added: Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts that we expect to receive for the satisfaction of each performance obligation.
+Added: We recognize revenue when or as control of the performance obligation transfers to the customer.
+Added: For performance obligations which consist of licenses and other promises, we utilize judgement to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time.
+Added: Following the Closing of the CSL Behring agreement, we recognize license revenue related to the License Sale of the global rights to the Product.
+Added: We evaluated that our performance obligation related to the License Sale was satisfied on Closing and recognized $462.4 million in license revenue related to a $450.0 million upfront payment and $12.4 million of payments in relation to Additional Covenants.
+Added: We will recognize additional license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of Product have been earned.
+Added: We recognize collaboration revenues associated with Manufacturing Development.
+Added: We will recognize collaboration revenue related to a contractual development milestone when it becomes probable the milestone will be achieved.
+Added: We recognize collaboration revenues associated with Development Services that will be reimbursed by CSL Behring relating to clinical development activities.
+Added: These services are provided by our employees.
+Added: Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.
+Added: In May 2015, we and Bristol-Myers Squibb (“BMS”) entered into a collaboration and license agreement and various related agreements with BMS (“BMS CLA”).
+Added: We have recognized license revenues associated with the amortization of the non-refundable upfront payment and target designation fees we received from BMS in accordance with the BMS CLA.
+Added: We evaluated our outstanding performance obligation following the amendment of the BMS CLA on December 1, 2020 (“amended BMS CLA”) and determined that our remaining performance obligation related to license revenues is immaterial.
We updated our measure of progress accordingly and amortized the remaining balance of unrecognized revenue as of December 1, 2020.
1 unchanged sentence
We will recognize revenue from these payments when earned or as sales occur.
+Added: We recognize collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that are reimbursable by BMS under the BMS CLA and the amended BMS CLA as well as other related agreements.
+Added: Collaboration revenue related to these contracted services is recognized when performance obligations are satisfied.
+Added: Cost of Contract Revenues
+Added: We expense contract fulfillment costs associated with license revenue recognized under the CSL Behring Agreement as costs of contract revenues.
Research and development expenses
6 unchanged sentences
● Costs associated with our research activities for our next-generation vector and promoter platform;
+Added: ● Costs associated with the rendering of collaboration services as well as the continued development of Product between Signing and Closing;
● Facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies.
7 unchanged sentences
● the timing of regulatory approvals;
−Removed: ● our ability to agree to ongoing development budgets with collaborators who share the costs of our development programs.
+Added: ● our ability to agree to ongoing development budgets with collaborators who reimburse the costs of our development programs.
A change in the outcome of any of these variables with respect to our product candidates that we may develop, including as a result of the Covid pandemic, could mean a significant change in the expenses and timing associated with the development of such product candidate.
2 unchanged sentences
We incur expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.
−Removed: Our selling costs include employee expenses as well as professional fees related to the preparation of a commercial launch of etranacogene dezaparvovec.
+Added: Our selling costs include employee expenses, as well as professional fees related to the preparation of a commercial launch of etranacogene dezaparvovec and advisory fees related to obtaining the CSL Behring Agreement.
Other items, net
−Removed: Our other income primarily consists of payments to subsidize our research and development efforts as well as income from the subleasing of our Amsterdam facility.
+Added: Our other income primarily consists of payments received to subsidize our research and development efforts, income from the subleasing of our Amsterdam facility, the recognition of the equity stake received in VectorY B.V., as well as an employee retention credit under the U.S.
+Added: Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”).
Our other expense primarily consists of expenses we incur in relation to our subleasing income.
Results of Operations
−Removed: Comparison of the three months ended March 31, 2021 and 2020
−Removed: The following table presents a comparison of our results of operations for the three months ended March 31, 2021 and 2020.
−Removed: Three months ended March 31,
+Added: Comparison of the three months ended June 30, 2021 and 2020
+Added: The following table presents a comparison of our results of operations for the three months ended June 30, 2021 and 2020.
+Added: Three months ended June 30,
(in thousands)
1 unchanged sentence
Operating expenses:
+Added: Cost of contract revenues
Research and development expenses
2 unchanged sentences
Other expense
−Removed: Loss from operations
+Added: Income / (loss) from operations
Other non-operating items, net
−Removed: Net loss before income tax expense
+Added: Net income / (loss) before income tax expense
Income tax expense
−Removed: Our revenue for the three months ended March 31, 2021 and 2020 was as follows:
−Removed: Three months ended March 31,
+Added: Net income / (loss)
+Added: Our revenue for the three months ended June 30, 2021 and 2020 was as follows:
+Added: Three months ended June 30,
(in thousands)
2 unchanged sentences
Total revenues
−Removed: We recognized $0.0 million License Revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the period ended March 31, 2020.
−Removed: We did not recognize any License Revenue from the December 1, 2020 amended BMS CLA for the period ended March 31, 2021.
−Removed: We recognized $0.5 million Collaboration Revenue in the three months ended March 31, 2021, compared to $0.1 million for the same period in 2020.
+Added: We recognized $462.4 million in license revenue related to the License Sale on Closing of the CSL Behring Agreement in May 2021.
+Added: We did not recognize any such license revenue related to the CSL Behring Agreement in the three months ended June 30, 2020.
+Added: We recognized $1.5 million in license revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the three months ended June 30, 2020.
+Added: We did not recognize any license revenue under the amended BMS CLA during the three months ended June 30, 2021.
+Added: We recognized $1.5 million collaboration revenue in the three months ended June 30, 2021, compared to $0.0 million for the same period in 2020.
+Added: Since May 6, 2021, we recognize collaboration revenue related to services we provide in accordance with the CSL Behring Agreement.
+Added: Our collaboration revenue increased further as a result of increased services provided to BMS following our December 2020 amendment of the BMS CLA.
+Added: Cost of contract revenues
+Added: We recognized $23.2 million in cost of contract revenues for the three months ended June 30, 2021, compared to nil the same period in 2020.
+Added: We recorded $16.7 million of expenses related to payments owed to our licensors on Closing as well as $6.5 million of other external expenses incurred to fulfill the License Sale to CSL Behring.
Research and development expenses
−Removed: Research and development expenses for the three months ended March 31, 2021 were $32.7 million, compared to $26.0 million for the same period in 2020.
+Added: Research and development expenses for the three months ended June 30, 2021 were $32.7 million, compared to $28.4 million for the same period in 2020.
Other research and development expenses are separately classified in the table below.
These are not allocated as they are deployed across multiple projects under development.
−Removed: Three months ended March 31,
+Added: Three months ended June 30,
(in thousands)
11 unchanged sentences
Etranacogene dezaparvovec (AMT-060/061)
−Removed: In the three months ended March 31, 2021 and 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial.
−Removed: Following the completion of patient enrollment into our HOPE-B trial we also started incurring costs related to preparation of a Biologics License Application (“BLA”) and marketing authorization application (“MAA”) and for commercialization of etranacogene dezaparvovec.
+Added: In the three months ended June 30, 2021 and 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial.
We enrolled patients into a six-month lead in phase between January 2018 and September 2019 and dosed a total of 54 patients between January 2019 and March 2020.
−Removed: Our expenses related to etranacogene dezaparvovec were largely unaffected by the COVID-19 pandemic as we completed enrollment prior to the lockdowns in those countries that we enroll patients.
−Removed: In addition, we continue to incur costs for the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec.
+Added: We also incur costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec.
+Added: During 2020 and up to the Closing of the CSL Behring Agreement we also incurred costs related to the preparation of a Biologics License Application (“BLA”) and marketing authorization application (“MAA”) and for commercialization of the Product.
+Added: After the Closing, CSL Behring will be responsible for the clinical and regulatory development and commercialization of the Product.
+Added: Direct research and development expenses related to clinical development incurred between Closing and June 30, 2021 are presented net of reimbursements due from CSL Behring.
Huntington disease (AMT-130)
−Removed: In the three months ended March 31, 2021 and March 31, 2020, our external costs for the development of Huntington’s disease were primarily related to the execution of our Phase I/II clinical trial.
+Added: In the three months ended June 30, 2021, and June 30, 2020, our external costs for the development of Huntington’s disease were primarily related to the execution of our Phase I/II clinical trial in the United States.
+Added: In addition, our external costs in the three months ended June 30, 2021, included expenses related to the preparation of our Phase I/II clinical trial in Europe that we announced in April 2021.
Preclinical programs & platform development
−Removed: In the three months ended March 31, 2021, we incurred $2.3 million of costs primarily related to our preclinical activities primarily associated with product candidates, SCA3 (AMT-150) and Fabry disease (AMT-190), as well as various other research programs and technology innovation projects, compared to costs of $1.5 million in the same period in 2020, which included costs related to our product candidate for Hemophilia A (AMT-180) that was subsequently deprioritized in June 2020.
+Added: In the three months ended June 30, 2021, we incurred $2.4 million of costs primarily related to our preclinical activities primarily associated with product candidates, SCA3 (AMT-150) and Fabry disease (AMT-190 and AMT-191), as well as various other research programs and technology innovation projects, compared to costs of $1.8 million in the same period in 2020, which also included costs related to our product candidate for Hemophilia A (AMT-180) that was deprioritized in June 2020.
Other research & development expenses
−Removed: ● We incurred $11.6 million in personnel and contractor related expenses in the three months ended March 31, 2021, compared to $9.3 million for the same period in 2020.
−Removed: Our costs during the three months ended March 31, 2021 increased by $2.3 million as a result of the recruitment of personnel to support the development of our product candidates;
−Removed: ● We incurred $2.7 million in share-based compensation expenses in the three months ended March 31, 2021, compared to $2.4 million for the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel;
−Removed: ● We incurred $4.6 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended March 31, 2021, compared to $4.0 million in the same period in 2020;
−Removed: ● We incurred $2.5 million in other costs in the three months ended March 31, 2021, compared to $0.8 million for the same period in 2020 related to miscellaneous other costs we incur as a result of expanding our organization.
+Added: ● We incurred $13.1 million in personnel and contractor related expenses in the three months ended June 30, 2021, compared to $10.0 million for the same period in 2020.
+Added: Our costs during the three months ended June 30, 2021 increased by $3.1 million as a result of the recruitment of personnel to support the development of our product candidates;
+Added: ● We incurred $4.4 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended June 30, 2021, compared to $4.2 million in the same period in 2020;
+Added: ● We incurred $3.3 million in share-based compensation expenses in the three months ended June 30, 2021, compared to $2.9 million for the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel.
Selling, general and administrative expenses
−Removed: Selling, general and administrative expenses for the three months ended March 31, 2021 were $12.4 million, compared to $9.1 million for the same period in 2020.
−Removed: ● We incurred $3.9 million in personnel and contractor related expenses in the three months ended March 31, 2021, compared to $3.2 million in the same period in 2020.
−Removed: The increase in the three months ended March 31, 2021, relates to the recruitment of personnel;
−Removed: ● We incurred $3.1 million in share-based compensation expenses in the three months ended March 31, 2021, compared to $1.9 million in the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel;
−Removed: ● We incurred $2.9 million in professional fees in the three months ended March 31, 2021, compared to $1.2 million in the same period in 2020.
+Added: Selling, general and administrative expenses for the three months ended June 30, 2021 were $17.3 million, compared to $11.5 million for the same period in 2020.
+Added: ● We incurred $4.5 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the three months ended June 30, 2021, compared to nil in the same period in 2020;
+Added: ● We incurred $4.2 million in personnel and contractor related expenses in the three months ended June 30, 2021, compared to $2.9 million in the same period in 2020.
+Added: The increase in the three months ended June 30, 2021, relates to the recruitment of personnel;
+Added: ● We incurred $3.7 million in share-based compensation expenses in the three months ended June 30, 2021, compared to $2.8 million in the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel;
+Added: ● We incurred $2.0 million in professional fees in the three months ended June 30, 2021, compared to $3.0 million in the same period in 2020.
We regularly incur accounting, audit and legal fees associated with operating as a public company.
−Removed: Additionally, in the three months ended March 31, 2021, we incurred professional fees in relation to our licensing transaction with CSL Behring.
Other items, net
−Removed: We recognized $0.0 million in income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the three months ended March 31, 2021, compared $0.2 million for the same period in 2020.
+Added: We recognized $3.0 million in other income in relation to the equity stake received in VectorY B.V.
+Added: in conjunction with the Settlement Agreement in the three months ended June 30, 2021, compared to no such income for the same period in 2020.
+Added: We recognized $1.3 million in other income of employee retention credit under the U.S.
+Added: CARES Act in the three months ended June 30, 2021, compared to no such income for the same period in 2020.
+Added: We recognized $3.0 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the three months ended June 30, 2021, compared to $0.3 million for the same period in 2020.
Other non-operating items, net
6 unchanged sentences
As of the same date, we recognized a derivative financial liability related to a contingent payment due to BMS upon the consummation of a change of control transaction (“CoC-payment”) as described elsewhere in the Quarterly Report on Form 10-Q .
−Removed: Our other non-operating items, net, for the three months ended March 31, 2021 and 2020 were as follows:
−Removed: Three months ended March 31,
+Added: Our other non-operating items, net, for the three months ended June 30, 2021 and 2020 were as follows:
+Added: Three months ended June 30,
(in thousands)
1 unchanged sentence
Interest expense - Hercules long-term debt
−Removed: Foreign currency gains, net
+Added: Foreign currency gains / (losses), net
Other non-operating gains
−Removed: Total other non-operating income, net
−Removed: We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $4.6 million during the three months ended March 31, 2021, compared to a net gain of $4.6 million during the same period in 2020.
−Removed: In the three months ended March 31, 2021, we recognized income of nil as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $2.0 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.
−Removed: We recognized $0.2 million of deferred tax expense for the period ended March 31, 2021 and nil in the same period ended 2020.
+Added: Total other non-operating income / (loss), net
+Added: We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $6.6 million during the three months ended June 30, 2021, compared to a net loss of $3.6 million during the same period in 2020.
+Added: In the three months ended June 30, 2021, we recognized income of nil within other non-operating gains as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $0.2 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.
+Added: Income tax expense
+Added: We recognized $3.3 million of deferred tax expense in the three months ended June 30, 2021, and nil in the same period ended 2020.
+Added: Comparison of the six months ended June 30, 2021 and 2020
+Added: The following table presents a comparison of the six months ended June 30, 2021 and 2020.
+Added: Six months ended June 30,
+Added: (in thousands)
+Added: Total revenues
+Added: Operating expenses:
+Added: Cost of contract revenues
+Added: Research and development expenses
+Added: Selling, general and administrative expenses
+Added: Total operating expenses
+Added: Other expense
+Added: Income / (loss) from operations
+Added: Non-operating items, net
+Added: Income / (loss) before income tax expense
+Added: Income tax expense
+Added: Net income / (loss)
+Added: Our revenue for the six months ended June 30, 2021 and 2020 was as follows:
+Added: Six months ended June 30,
+Added: (in thousands)
+Added: License revenue
+Added: Collaboration revenue
+Added: Total revenues
+Added: We recognized $462.4 million license revenue related to the License Sale on Closing of the CSL Behring Agreement in May 2021.
+Added: We did not recognize any such license revenue related to the CSL Behring Agreement in the six months ended June 30, 2020.
+Added: We recognized $1.6 million license revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the six months ended June 30, 2020.
+Added: We did not recognize any license revenue under the amended BMS CLA during the six months ended June 30, 2021.
+Added: We recognized $1.9 million collaboration revenue in the six months ended June 30, 2021, compared to $0.1 million for the same period in 2020.
+Added: The increase in collaboration revenue primarily relates to the increased activities under the amended BMS CLA and the CSL Behring Agreement.
+Added: Cost of contract revenues
+Added: We recognized $23.2 million in cost of contract revenues for the six months ended June 30, 2021 compared to nil the same period in 2020.
+Added: We recorded $16.7 million of expenses related to payments owed to our licensors on Closing as well as $6.5 million of other external expenses incurred to fulfill the License Sale to CSL Behring.
+Added: Research and development
+Added: Research and development expenses for the six months ended June 30, 2021 were $64.8 million, compared to $54.4 million for the same period in 2020.
+Added: Other research and development expenses are separately classified in the table below.
+Added: These are not allocated as they are deployed across multiple projects under development.
+Added: Six months ended June 30,
+Added: (in thousands)
+Added: Etranacogene dezaparvovec (AMT-060/061)
+Added: Huntington's disease (AMT-130)
+Added: Programs in preclinical development and platform related expenses
+Added: Total direct research and development expenses
+Added: Employee and contractor-related expenses
+Added: Facility expenses
+Added: Share-based compensation expense
+Added: Other expenses
+Added: Total other research and development expenses
+Added: Total research and development expenses
+Added: Direct research and development expenses
+Added: Etranacogene dezaparvovec (AMT-060/061)
+Added: In the six months ended June 30, 2021 and 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial.
+Added: We enrolled patients into a six-month lead in phase between January 2018 and September 2019 and dosed a total of 54 patients between January 2019 and March 2020.
+Added: We also incur costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec.
+Added: During 2020 and up to the Closing of the CSL Behring Agreement, we also incurred costs related to the preparation of a BLA and MAA and for commercialization of the Product.
+Added: After the Closing, CSL Behring will be responsible for the clinical and regulatory development and commercialization of the Product.
+Added: Direct research and development expenses related to clinical development incurred between Closing and June 30, 2021, are presented net of reimbursements due from CSL Behring.
+Added: Huntington disease (AMT-130)
+Added: In the six months ended June 30, 2021 and June 30, 2020, our external costs for the development of Huntington’s disease were primarily related to the execution of our Phase I/II clinical trial in the United States.
+Added: In addition, our external costs in the six months ended June 30, 2021, included expenses related to the preparation of our Phase I/II clinical trial in Europe that we announced in April 2021.
+Added: Preclinical programs & platform development
+Added: In the six months ended June 30, 2021, we incurred $4.7 million of costs primarily related to our preclinical activities primarily associated with product candidates, SCA3 (AMT-150) and Fabry disease (AMT-190 and AMT-191), as well as various other research programs and technology innovation projects, compared to costs of $3.2 million in the same period in 2020, which also included costs related to our product candidate for Hemophilia A (AMT-180) that was deprioritized in June 2020.
+Added: Other research & development expenses
+Added: ● We incurred $24.7 million in personnel and contractor related expenses in the six months ended June 30, 2021, compared to $19.3 million for the same period in 2020.
+Added: Our costs during the six months ended June 30, 2021 increased by $5.4 million as a result of the recruitment of personnel to support the development of our product candidates;
+Added: ● We incurred $9.0 million in operating expenses and depreciation expenses related to our rented facilities in the six months ended June 30, 2021, compared to $8.2 million in the same period in 2020 as a result of expanding our organization;
+Added: ● We incurred $6.0 million in share-based compensation expenses in the six months ended June 30, 2021, compared to $5.3 million for the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel.
+Added: Selling, general and administrative expenses
+Added: Selling, general and administrative expenses for the six months ended June 30, 2021 were $30.3 million, compared to $20.6 million for the same period in 2020.
+Added: ● We incurred $8.1 million in personnel and contractor related expenses in the six months ended June 30, 2021, compared to $6.1 million in the same period in 2020.
+Added: The increase in the six months ended June 30, 2021, relates to the recruitment of personnel;
+Added: ● We incurred $6.8 million in share-based compensation expenses in the six months ended June 30, 2021, compared to $4.8 million in the same period in 2020 primarily driven by increase in awards granted, including those to newly recruited personnel;
+Added: ● We incurred $5.0 million in professional fees in the six months ended June 30, 2021, compared to $4.2 million in the same period in 2020.
+Added: We regularly incur accounting, audit and legal fees associated with operating as a public company;
+Added: ● We incurred $4.5 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the six months ended June 30, 2021, compared to nil in the same period in 2020.
+Added: Other items, net
+Added: We recognized $3.0 million in other income in relation to the equity stake received in VectorY B.V.
+Added: in conjunction with the Settlement Agreement in the six months ended June 30, 2021, compared to no such income for the same period in 2020.
+Added: We recognized $1.3 million in other income of employee retention credit under the U.S.
+Added: CARES Act in the six months ended June 30, 2021, compared to no such income for the same period in 2020.
+Added: We recognized $3.0 million in income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the six months ended June 30, 2021, compared $0.5 million for the same period in 2020.
+Added: Other non-operating items, net
+Added: We recognize interest income associated with our cash and cash equivalents.
+Added: We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S.
+Added: We recognize foreign exchange results related to changes in these foreign currencies.
+Added: We issued warrants to BMS in 2015.
+Added: We recognize changes in the fair value of these warrants within other non-operating income / (expense).
+Added: Following the termination of the BMS warrants on December 1, 2020, we no longer recognize changes in the fair value of these warrants within other non-operating (expense) / income.
+Added: As of the same date, we recognized a derivative financial liability related to a contingent payment due to BMS upon the consummation of a CoC-payment as described elsewhere in the Quarterly Report on Form 10-Q .
+Added: Six months ended June 30,
+Added: (in thousands)
+Added: Interest income
+Added: Interest expense
+Added: Foreign currency gains, net
+Added: Other non-operating gains, net
+Added: Total non-operating income, net
+Added: We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $11.2 million during the six months ended June 30, 2021, compared to a net gain of $1.0 million during the same period in 2020.
+Added: In the six months ended June 30, 2021, we recognized income of nil within other non-operating items as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $2.2 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.
+Added: Income tax expense
+Added: We recognized $3.5 million of deferred tax expense for the six months ended June 30, 2021 and nil in the same period ended 2020.
Financial Position, Liquidity and Capital Resources
−Removed: As of March 31, 2021, we had cash, cash equivalents and restricted cash of $263.5 million.
−Removed: We believe our cash and cash equivalents as of March 31, 2021, combined with the $100.0 million 2021 Amended Facility, will enable us to fund our operating expenses, including our debt repayment obligations, as they become due and capital expenditure requirements until the end of 2022.
−Removed: Following the receipt of the $450.0 million upfront payment, which was paid to us on May 7, 2021, we expect that our cash and cash equivalents will be sufficient to fund operations into the second half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facility by 2023).
−Removed: We are also eligible to receive up to $1.6 billion of milestones and tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds, both of which are not included in the above guidance.
−Removed: Three months ended March 31,
+Added: As of June 30, 2021, we had cash, cash equivalents and restricted cash of $680.2 million which include payments received from CSL Behring following the Closing.
+Added: This is expected to fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facility by 2023, as well as payments we expected to make in relation to our acquisition of Corlieve).
+Added: We expect that the achievement of $0.3 billion of regulatory, first commercial sale, and development milestones would further extend our runway by 12 to 18 months.
+Added: Six months ended June 30,
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period
−Removed: Net cash used in operating activities
+Added: Net cash generated from / (used in) operating activities
Net cash used in investing activities
2 unchanged sentences
Cash, cash equivalents and restricted cash at the end of period
−Removed: We have incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics (“AMT”) Holding N.V.
−Removed: We had a net loss of $41.6 million during the three months ended March 31, 2021, compared to a net loss of $28.0 million during the same period in 2020.
−Removed: As of March 31, 2021, we had an accumulated deficit of $826.3 million.
+Added: We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics (“AMT”) Holding N.V.
+Added: As a result of receiving the upfront payment upon Closing of the CSL Behring Agreement, we generated $375.2 million cash flows from operations during the six months ended June 30, 2021.
+Added: We recorded net income of $399.5 million and $357.9 during the three and six months ended June 30, 2021, compared to a net loss of $42.6 million and $70.6 million during the same period in 2020.
+Added: As of June 30, 2021, we had an accumulated deficit of $426.8 million.
Sources of liquidity
−Removed: From our first institutional venture capital financing in 2006 through March 31, 2021, we funded our operations primarily through private and public placements of equity securities and convertible and other debt securities as well as payments from our collaboration partners.
+Added: From our first institutional venture capital financing in 2006 through May 2021, we funded our operations primarily through private and public placements of equity securities and convertible and other debt securities as well as payments from our collaboration partners.
+Added: In May 2021, we received a $462.4 million cash payment due from CSL Behring upon Closing.
+Added: In addition, we could potentially receive up to $0.3 billion in regulatory, first commercial sales and development milestone payments from CSL Behring.
On March 1, 2021, we entered into a Sales Agreement with SVB Leerink with respect to an ATM offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.
We will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement.
−Removed: In March 2021 we issued 0.9 million ordinary shares for net proceeds of $27.6 million.
+Added: In the six months ended June 30, 2021, we issued 921,730 million ordinary shares for net proceeds of $29.6 million.
On December 6, 2018, we signed the 2018 Amended Facility with Hercules that both refinanced our then-existing $20.0 million credit facility and provided us with an additional unconditional commitment of $15.0 million as well as a conditional commitment of $15 million that expired on June 30, 2020.
14 unchanged sentences
Advances under the Tranche B may not be prepaid until six-months after the Closing Date, following which we may prepay all such advances without charge.
−Removed: As of March 31, 2021, $70.0 million was outstanding under the 2018 Amended Facility and the 2021 Amended Facility (December 31, 2020:
+Added: As of June 30, 2021, $70.0 million was outstanding under the 2018 Amended Facility and the 2021 Amended Facility (December 31, 2020:
$35.0 million under the 2018 Amended Facility).
5 unchanged sentences
If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.
−Removed: Net Cash used in operating activities
−Removed: Net cash used in operating activities was $41.3 million for the three months ended March 31, 2021 and consisted of a net loss of $41.6 million adjusted for non-cash items, including depreciation and amortization expense of $1.9 million, share-based compensation expense of $5.8 million, unrealized foreign exchange gains of $5.3 million and deferred tax expense of $0.2 million.
−Removed: Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million.
−Removed: These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $8.0 million primarily related to the increase in a contract asset and receivable recorded for CSL expenses to be reimbursed as well as an increase in various prepayments for the year and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $5.8 million primarily related to an increase in contract liability for CSL expenses to be reimbursed and an increase in accruals for services provided by vendors not yet billed offset by the pay-out of employee bonuses.
−Removed: Net cash used in operating activities was $33.0 million for the three months ended March 31, 2020 and consisted of a net loss of $28.0 million adjusted for non-cash items, including depreciation and amortization expense of $1.7 million, share-based compensation expense of $4.4 million, fair value gains on derivative financial instruments of $2.0 million, unrealized foreign exchange gain of $4.8 million, and an increase in unamortized deferred revenue of $0.3 million.
+Added: Net Cash generated from / used in operating activities
+Added: Net cash generated from operating activities was $375.2 million for the six months ended June 30, 2021 and consisted of net income of $357.9 million adjusted for non-cash items, including depreciation and amortization expense of $3.7 million, share-based compensation expense of $12.8 million, unrealized foreign exchange gains of $12.3 million and other non-cash items, net, of $0.5 million.
+Added: Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $12.6 million.
+Added: These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $3.4 million primarily related to an increase in various prepayments and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $15.9 million primarily related to an increase in amounts to be paid to licensors upon the CSL Behring Closing.
+Added: Net income primarily consisted of $462.4 million license revenue recognized on Closing.
+Added: Net cash used in operating activities was $62.9 million for the six months ended June 30, 2020 and consisted of a net loss of $70.6 million adjusted for non-cash items, including depreciation and amortization expense of $3.5 million, share-based compensation expense of $10.1 million, fair value gains on derivative financial instruments of $2.2 million, unrealized foreign exchange gain of $0.9 million, and a decrease in other non-cash items, net of $1.6 million.
Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $1.3 million.
−Removed: These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets of $1.3 million primarily as a result of increase in prepayments for the year and a net decrease in accounts payable, accrued expenses and other liabilities of $3.2 million primarily related to pay-out of employee bonuses.
+Added: These changes primarily related to a net decrease in accounts receivable and accrued income, prepaid expenses, and other current assets of $1.1 million and a net decrease in accounts payable, accrued expenses and other liabilities of $2.3 million.
Net cash used in investing activities
−Removed: In the three months ended March 31, 2021, we used $3.9 million in our investing activities compared to $2.9 million for the same period in 2020.
−Removed: Three months ended March 31,
+Added: In the six months ended June 30, 2021, we used $6.2 million in our investing activities compared to $4.6 million for the same period in 2020.
+Added: Six months ended June 30,
(in thousands)
3 unchanged sentences
Total investments
−Removed: The build out of the Amsterdam site was $2.4 million for the three months ended March 31, 2021, compared to $0.4 million for the same period in 2020.
+Added: The build out of the Amsterdam site consumed $4.5 million cash during the six months ended June 30, 2021, compared to $1.8 million for the same period in 2020.
The increase is a result of the construction of additional laboratories to support the expansions of our research and development activities as well as the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.
Net cash generated from financing activities
−Removed: In the three months ended March 31, 2021, we generated $62.6 million in our financing activities compared to $1.0 million for the same period in 2020.
−Removed: Three months ended March 31,
+Added: In the six months ended June 30, 2021, we generated $65.1 million in our financing activities compared to $3.5 million for the same period in 2020.
+Added: Six months ended June 30,
(in thousands)
1 unchanged sentence
Proceeds from loan increment, net of debt issuance costs
−Removed: Proceeds from issuance of ordinary shares
−Removed: Share issuance costs from issuance of ordinary shares
+Added: Proceeds from issuance of ordinary shares, net of issuance costs
Proceeds from issuance of shares related to employee stock option and purchase plans
Net cash generated from financing activities
−Removed: We received proceeds of $28.7 million associated with our ATM offering in March 2021, offset by fees of $1.2 million.
In January 2021, we received $34.6 million net proceeds from the Hercules 2021 Amended Facility.
−Removed: During the three months ended March 31, 2021, we received $0.4 million from the exercise of options to purchase ordinary shares in relation to our share incentive plans compared to $1.0 million for the same period in 2020.
+Added: We received net proceeds of $29.6 million associated with our ATM offering in March and April 2021.
+Added: During the six months ended June 30, 2021, we received $1.0 million from the exercise of options to purchase ordinary shares in relation to our share incentive plans compared to $3.5 million for the same period in 2020.
Funding requirements
−Removed: We believe our cash and cash equivalents as of March 31, 2021, combined with the $100.0 million 2021 Amended Facility as well as the upfront payment of $450.0 million that we became entitled to receive following the closing of our transaction with CSL Behring on May 6, 2021, will enable us to fund our operating expenses, including our debt repayment obligations, as they become due and capital expenditure requirements into the second half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facility by 2023).
+Added: We believe our cash and cash equivalents as of June 30, 2021, will fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facilities by 2023, as well as payments we expected to make in relation to our acquisition of Corlieve).
+Added: We expect that the achievement of the $0.3 billion in regulatory, first commercial sales and development milestones would further extend our runway by 12 to 18 months.
Our future capital requirements will depend on many factors, including but not limited to:
● achieving the milestones and royalties as defined within the CSL Behring Agreement;
+Added: ● payments we will make in relation to the contemplated acquisition of Corlieve;
+Added: ● the scope, timing, results, and costs of our current and planned clinical trials, including those for AMT-130 in Huntington’s disease;
● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;
● the amount and timing of revenue, if any, we receive from commercial sales of any product candidates for which we, or our collaboration partner, receives marketing approval in the future;
−Removed: ● the amount and timing of revenue, if any, we receive from manufacturing products from CSL Behring.
−Removed: ● the scope, timing, results, and costs of our current and planned clinical trials, including those for AMT-130 in Huntington’s disease;
+Added: ● the amount and timing of revenue, if any, we receive from manufacturing products for CSL Behring.
● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;
9 unchanged sentences
Contractual obligations and commitments
−Removed: The table below sets forth our contractual obligations and commercial commitments as of March 31, 2021, that are expected to have an impact on liquidity and cash flows in future periods.
+Added: The table below sets forth our contractual obligations and commercial commitments as of June 30, 2021, that are expected to have an impact on liquidity and cash flows in future periods.
(in thousands)
3 unchanged sentences
We have not included these commitments on our balance sheet or in the table above because the achievement and timing of these milestones is not fixed and determinable.
−Removed: We also have obligations to make future payments that become due and payable upon the collection of the upfront payment or milestone payments from CSL Behring.
−Removed: We have not included these commitments on our balance sheet or in the table above because these payments only become due and payable after the CSL Behring Agreement became fully effective on May 6, 2021.
+Added: We also have obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring.
+Added: We have not included these commitments on our balance sheet or in the table above because the achievement and timing of these milestones is not fixed and determinable.
We enter into contracts in the normal course of business with clinical research organizations (“CROs”) for preclinical research studies and clinical trials, research supplies and other services and products for operating purposes.
1 unchanged sentence
Off-Balance Sheet Arrangements
−Removed: As of March 31, 2021, we did not have any off-balance sheet arrangements as defined in Item 303(a)(4) of Regulation S-K.
+Added: As of June 30, 2021, we did not have any off-balance sheet arrangements as defined in Item 303(a)(4) of Regulation S-K.
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.