COLLABORATION AGREEMENTS | 4. COLLABORATION AGREEMENTS Net revenues from collaborators (in thousands) consist of the following: Three Months Ended June 30, Six Months Ended June 30, Description 2019 2018 2019 2018 Sanofi Genzyme $ 4,383 $ 23,077 $ 8,500 $ 41,930 Vir Biotechnology (Vir) 1,091 6,113 2,019 7,356 The Medicines Company (MDCO) — 662 1,745 1,957 Regeneron 700 — 700 — Other 309 55 522 563 Total net revenues from collaborators $ 6,483 $ 29,907 $ 13,486 $ 51,806 The following table presents the balance of our receivables and contract liabilities related to our collaboration agreements at June 30, 2019 and December 31, 2018, in thousands: At June 30, 2019 At December 31, 2018 Receivables included in “Accounts receivable, net” $ 13,691 $ 5,625 Contract liabilities included in “Deferred revenue” 403,033 3,954 During the three and six months ended June 30, 2019, we recognized the following revenue as a result of the change in the contract liability balances related to our collaboration agreements, in thousands: Revenue recognized in the period from: Three Months Ended June 30, 2019 Six Months Ended June 30, 2019 Amounts included in contract liability at the beginning of the period $ 1,091 $ 2,019 In order to determine revenue recognized in the period from contract liabilities, we first allocate revenue to the individual contract liability balance outstanding at the beginning of the period until the revenue exceeds that balance. If additional consideration is received on those contracts in subsequent periods, we assume all revenue recognized in the reporting period first applies to the beginning contract liability as opposed to a portion applying to the new consideration for the period. The following tables provide the research and development expenses incurred by type that are directly attributable to our collaboration agreements by our collaboration partners for the periods indicated, in thousands: Three Months Ended June 30, 2019 2018 Sanofi Genzyme MDCO Vir Regeneron Sanofi Genzyme MDCO Vir Regeneron Research and development Clinical trial and manufacturing $ 2,945 $ 65 $ 248 $ 515 $ 17,572 $ — $ 5,497 $ — External services 81 — 12 51 1,834 — 6,151 — Other 34 10 211 425 241 — 292 — Total research and development expenses $ 3,060 $ 75 $ 471 $ 991 $ 19,647 $ — $ 11,940 $ — Six Months Ended June 30, 2019 2018 Sanofi Genzyme MDCO Vir Regeneron Sanofi Genzyme MDCO Vir Regeneron Research and development Clinical trial and manufacturing $ 7,771 $ 1,677 $ 542 $ 515 $ 28,095 $ 641 $ 6,051 $ — External services 216 10 248 51 4,507 — 6,351 — Other 93 60 340 425 750 — 980 — Total research and development expenses $ 8,080 $ 1,747 $ 1,130 $ 991 $ 33,352 $ 641 $ 13,382 $ — The research and development expenses incurred for each agreement listed in the tables above consist of costs incurred for external development and manufacturing services for which we are reimbursed, licensing payments made to the counterparty to such agreement and costs directly attributable to Sanofi Genzyme transition services. In addition, these expenses include a reasonable estimate of compensation and related costs as billed to our counterparties. As part of our revenue recognition policy adopted on January 1, 2018, the costs in the above table are considered as an input in our determination of transaction price when they relate to consideration received for the delivery of goods or services. For the three and six months ended June 30, 2019 and 2018, we did not incur material selling, general and administrative expenses related to our collaboration agreements. Regeneron Collaboration On April 8, 2019, we entered into a global, strategic collaboration with Regeneron Pharmaceuticals, Inc., or Regeneron, to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic targets expressed in the eye and central nervous system, or CNS, in addition to a select number of targets expressed in the liver, which we refer to as the Regeneron Collaboration. The Regeneron Collaboration is governed by a Master Agreement, referred to as the Regeneron Master Agreement, which became effective on May 21, 2019, or the Effective Date, upon closing of the Stock Purchase Agreement dated April 8, 2019, referred to as the Regeneron SPA, and issuance of 4,444,445 shares of our newly issued common stock to Regeneron for aggregate cash consideration of $400.0 million, or $90.00 per share. Please read Footnote 10 for a full discussion regarding the Regeneron SPA. Under the terms of the Regeneron Collaboration, we will work exclusively with Regeneron to discover RNAi therapeutics for eye and CNS diseases for an initial five-year research period, which we refer to as the Initial Research Term. Regeneron has an option to extend the Initial Research Term (referred to as the Research Term Extension Period, and together with the Initial Research Term, the Research Term) for up to an additional two years, for a research term extension fee of up to $400.0 million. The Regeneron Collaboration also covers a select number of RNAi therapeutic programs designed to target genes expressed in the liver, including our previously-announced collaboration with Regeneron to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis. We retain broad global rights to all of our other unpartnered liver-directed clinical and preclinical pipeline programs. The Regeneron Collaboration is governed by a joint steering committee that is comprised of an equal number of representatives from each party. Regeneron will lead development and commercialization for all programs targeting eye diseases (subject to limited exceptions), entitling us to certain potential milestone and royalty payments pursuant to the terms of a license agreement , the form of which has been agreed upon by the parties . We and Regeneron will alternate leadership on CNS and liver programs, with the lead party retaining global development and commercial responsibility. For CNS and liver programs, both we and Regeneron will have the option at lead candidate selection to enter into a co-co collaboration agreement , the form of which has been agreed upon by the parties, whereby both companies will share equally all costs of, and profits from, all development and commercialization activities under the program . If the non-lead party elects to not enter into a c o- c o c ollaboration a greement with respect to a given CNS or liver program, we and Regeneron will enter into a l icense a greement with respect to such program and the lead party will be the “Licensee” for the purposes of the l icense a greement. If the lead party for a CNS or liver program elects to not enter into the c o - c o c ollaboration a greement , then we and Regeneron will enter into a l icense a greement with respect to such program and leadership of the program will transfer to the other party and the former non-lead party will be the “Licensee” for the purposes of the l icense a greement. In addition, we and Regeneron have agreed to evaluate anti-C5 antibody-siRNA combinations for C5 complement-mediated diseases including evaluating the combination of Regeneron’s pozelimab (REGN3918), currently in Phase 1 development, and cemdisiran, currently in Phase 2 development. We retain control of cemdisiran monotherapy development, and Regeneron will lead combination product development. We and Regeneron will negotiate and enter into a co-co collaboration agreement to equally share costs and potential future profits on the monotherapy program, and a license agreement for cemdisiran to be used in a combination product pursuant to which Regeneron will be solely responsible for all development and commercialization costs and we will receive low double-digit royalties and commercial milestones of up to $325.0 million on any potential combination product sales, in each case in accordance with the terms set forth in the agreed-upon binding term sheet attached to the Regeneron Master Agreement, combined for accounting purposes and treated as a single agreement. In connection with the Regeneron Master Agreement, Regeneron made an upfront payment of $400.0 million Regeneron has the right to terminate the Regeneron Master Agreement for convenience upon ninety days’ notice. The termination of the Regeneron Master Agreement does not affect the term of any license agreement or co-co collaboration agreement then in effect. In addition, either party may terminate the Regeneron Master Agreement for a material breach by, or insolvency of, the other party. Unless earlier terminated pursuant to its terms, the Regeneron Master Agreement will remain in effect with respect to each program until (a) such program becomes a terminated program or (b) the parties enter into a license agreement or co-co collaboration agreement with respect to such program. The Regeneron Master Agreement includes various representations, warranties, covenants, dispute escalation and resolution mechanisms, indemnities and other provisions customary for transactions of this nature. For any license agreement subsequently entered into, the licensee will generally be responsible for its own costs and expenses incurred in connection with the development and commercialization of the collaboration products. The licensee will pay to the licensor certain development and/or commercialization milestone payments totaling up to $150.0 million for each collaboration product. In addition, following the first commercial sale of the applicable collaboration product under a license agreement, the licensee is required to make certain tiered royalty payments, ranging from low double-digits up to 20%, to the licensor based on the aggregate annual net sales of the collaboration product, subject to customary reductions. For any co-co collaboration agreement subsequently entered into, we and Regeneron will share equally all costs of, and profits from, development and commercialization activities. In the event that a party exercises its opt-out right, the lead party will be responsible for all costs and expenses incurred in connection with the development and commercialization of the collaboration products under the applicable co-co collaboration agreement, subject to continued sharing of costs through defined points. If a party exercises its opt-out right, following the first commercial sale of the applicable collaboration product under a co-co collaboration agreement, the lead party is required to make certain tiered royalty payments, ranging from low double-digits up to 20%, to the other party based on the aggregate annual net sales of the collaboration product and the timing of the exercise of the opt-out right, subject to customary reductions and a reduction for opt-out transition costs. Due to the uncertainty of pharmaceutical development and the high historical failure rates generally associated with drug development, we may not receive any milestone or royalty payments from Regeneron under the Regeneron Master Agreement or any future license agreement, or under any co-co collaboration agreement in the event we exercise our opt-out right. Our obligations under the Regeneron Collaboration include: (i) a research license and research services, collectively referred to as the Research Services Obligation; (ii) a worldwide license to cemdisiran for combination therapies, and manufacturing and supply, and development service obligations through completion of Phase 2 clinical trials, collectively referred to as the C5 License Obligation; and (iii) development, manufacturing and commercialization activities for cemdisiran monotherapies, referred to as the C5 Co-Co Obligation. The research license is not distinct from the research services primarily as a result of Regeneron being unable to benefit on its own or with other resources reasonably available, as the license is providing access to specialized expertise, particularly as it relates to RNAi technology that is not available in the marketplace. Similarly, t he worldwide license to cemdisiran for combination therapies is not distinct from the manufacturing and supply , and development service obligations, as Regeneron cannot benefit on its own from the value of the license without receipt of supply. Separately, the cemdisiran monotherapy co-co collaboration agreement is under the scope of ASC 808 as we and Regeneron are both active participants in the development, manufacturing and commercialization activities and are exposed to significant risks and rewards that are dependent on commercial success of the activities of the arrangement. The development, manufacturing and commercialization activities are a combined unit of account under the scope of ASC 808 and are not deliverables under ASC 606. As of the Effective Date, the total transaction price was determined to be $521.6 million, comprised of the $400.0 million upfront payment and $121.6 million of variable consideration related to research, development, manufacturing and supply activities. We utilized the expected value method to determine the amount of reimbursement for these activities. We determined that any variable consideration related to sales-based royalties and milestones related to the worldwide license to cemdisiran for combination therapies is deemed to be constrained and therefore has been excluded from the transaction price. In addition, we are eligible to receive future milestones upon the achievement of certain criteria during early clinical development for the eye and CNS programs. We are also eligible to receive royalties on future commercial sales for certain ocular, CNS or liver targets, if any; however, these amounts are excluded from variable consideration under the combined Regeneron agreements as we are only eligible to receive such amounts if, after a drug candidate is identified, the form of license agreement is subsequently executed resulting in a license that is granted to Regeneron. Any such subsequently granted license would represent a separate transaction under ASC 606. We will re-evaluate the transaction price at the end of each reporting period. There was no change in the transaction price from the Effective Date to June 30, 2019. The transaction price was allocated to the obligations based on the relative estimated standalone selling prices of each obligation, over which management has applied significant judgment. We developed the estimated standalone selling price for the licenses included in the Research Services Obligation and the C5 License Obligation primarily based on the probability-weighted present value of expected future cash flows associated with each license related to each specific program. In developing such estimate, we also considered applicable market conditions and relevant entity-specific factors, including those factors contemplated in negotiating the agreement, the probability of success and the time needed to commercialize a product candidate pursuant to the associated license. We developed the estimated standalone selling price for the services and/or manufacturing and supply included in each of the obligations, as applicable, primarily based on the nature of the services to be performed and/or goods to be manufactured and estimates of the associated costs. The estimated standalone selling price of the C5 Co-Co- Obligation was developed by estimating the present value of expected future cash flows that Regeneron is entitled to receive. We allocated the transaction price to each unit of account based on the applicable accounting guidance as follows: (i) $178.5 million to the Research Services Obligation; (ii) $93.5 million to the C5 License Obligation; and (iii) $249.6 million to the C5 Co-Co Obligation. We measure proportional performance over time using an input method based on cost incurred relative to the total estimated costs for each of the identified obligations, on a quarterly basis, by determining the proportion of effort incurred as a percentage of total effort we expect to expend. This ratio is applied to the transaction price allocated to each obligation. Management has applied significant judgment in the process of developing our estimates. Any changes to these estimates will be recognized in the period in which they change as a cumulative catch up. As of June 30, 2019, the aggregate amount of the transaction price allocated to the remaining Research Services Obligation and C5 License Obligation that was unsatisfied is $271.3 million, which is expected to be recognized through the term of the Regeneron Collaboration as the services are performed. This amount excludes the transaction price allocated to the remaining C5 Co-Co Obligation accounted for under ASC 808. We had deferred revenue of $401.8 million as of June 30, 2019, which is classified as current or non-current in the condensed consolidated balance sheets based on the period the services are expected to be performed. Sanofi Genzyme Collaboration Collaboration Amendment On April 8, 2019, we and Sanofi Genzyme entered into an amendment to our 2014 Sanofi Genzyme collaboration, which we refer to as the Collaboration Amendment. Under the Collaboration Amendment, we and Sanofi Genzyme agreed to conclude the research and option phase under our collaboration agreement. In connection and simultaneously with entering into the Collaboration Amendment, we and Sanofi Genzyme also entered into the Amended and Restated ALN-AT3 Global License Terms with respect to ALN-AT3 (fitusiran) and certain back-up products, which we refer to as the A&R AT3 License Terms. The A&R AT3 License Terms amend and restate the ALN-AT3 Global License Terms entered into by us and Sanofi Genzyme in January 2018 to modify certain of the business terms. The material collaboration terms for fitusiran, as previously announced, will continue unchanged. In connection with entering into the Collaboration Amendment and the A&R AT3 License Terms , we agreed to advance, at our cost, a selected investigational asset in an undisclosed rare genetic disease through the end of IND-enabling studies. Following completion of such studies, we will transition, at our cost, such asset to Sanofi Genzyme. Thereafter, Sanofi Genzyme will fund all potential future development and commercialization costs for such asset. If this asset is developed and approved, we will be eligible to receive tiered double-digit royalties on global net sales. No changes were made to our Exclusive License Agreement with Sanofi Genzyme, referred to as the Exclusive TTR License, pursuant to which we have global rights for the development and commercialization of ONPATTRO, together with vutrisiran and all back-up products, which remains in full force and effect. Amended and Restated Investor Agreement In connection with the Collaboration Amendment, we and Sanofi Genzyme also entered into an Amended and Restated Investor Agreement, referred to as the A&R Investor Agreement, which amended and restated the Investor Agreement entered into by us and Sanofi Genzyme in February 2014, referred to as the Original Investor Agreement. Pursuant to the A&R Investor Agreement, Sanofi Genzyme was released from the lock-up restrictions under the Original Investor Agreement and was permitted to sell shares of our common stock in transactions approved by us or in fully bought block sale transactions satisfying the conditions set forth in the A&R Investor Agreement. As of January 17, 2019, Sanofi Genzyme owned 10,554,134 shares of our common stock and as of May 3, 2019, Sanofi Genzyme reported owning no shares of our common stock. Under the A&R Investor Agreement, until the earlier of (i) the fifth anniversary of the expiration of the last to expire royalty term or the earlier termination of the collaboration agreement, as amended by the Collaboration Amendment, and (ii) the date after December 31, 2021 on which the beneficial ownership of Sanofi Genzyme and its affiliates no longer represents at least 5% of the outstanding shares of common stock, Sanofi Genzyme and its affiliates will be bound by certain “standstill” provisions, including an agreement not to propose or support a proposal to acquire us. |