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Collaborative Arrangements and Licensing Agreements
 6 Months Ended
Jun. 30, 2024
Collaborative Arrangements and Licensing Agreements [Abstract]  
Collaborative Arrangements and Licensing Agreements
5.  Collaborative Arrangements and Licensing Agreements


Below, we have included our AstraZeneca, Biogen, Otsuka and Roche collaborations, which are the collaborations with substantive changes during 2024 from those included in Part IV, Item 15, Note 4, Collaborative Arrangements and Licensing Agreements, of our audited financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2023.


AstraZeneca


We have two collaborations with AstraZeneca, one focused on the joint development and commercialization of WAINUA and one focused on the treatment of cardiovascular, renal and metabolic diseases. From inception through June 30, 2024, we have received more than $920 million from these collaborations.


Over the term of our WAINUA collaboration, we are eligible to receive up to $3.6 billion, which is comprised of a $200 million upfront payment, up to $485 million in development and approval milestone payments and up to $2.9 billion in sales milestone payments. The agreement includes territory-specific development, commercial and medical affairs cost-sharing provisions. In addition, we are eligible to receive up to mid-20 percent royalties for sales in the U.S. and tiered royalties up to the high teens for sales outside the U.S.


In January 2024, we and AstraZeneca launched WAINUA in the U.S. for the treatment of adults with ATTRv-PN. As a result, we began earning royalties from WAINUA sales, which we recognize as commercial revenue in our condensed consolidated statements of operations. We will achieve the next payment of up to $30 million upon regulatory approval of WAINUA for ATTRv-PN in the European Union, or EU, under this collaboration.


During the three and six months ended June 30, 2024 and 2023, we earned the following revenue from our relationship with AstraZeneca (in thousands, except percentage amounts):

 
Three Months Ended
June 30,
    
Six Months Ended
June 30,
  
   
2024
    
2023
    
2024
    
2023
  
Revenue from our relationship with AstraZeneca
  
$
15,960
    
$
59,501
    
$
27,645
    
$
83,926
  
Percentage of total revenue
    
7
%
    
32
%
    
8
%
    
26
%


Our condensed consolidated balance sheet at June 30, 2024 included deferred contract revenue of $1.9 million from our relationship with AstraZeneca. We did not have any deferred contract revenue from our relationship with AstraZeneca at December 31, 2023.


Biogen


We have several strategic collaborations with Biogen focused on using antisense technology to advance the treatment of neurological disorders. We developed and licensed to Biogen SPINRAZA, our approved medicine to treat people with spinal muscular atrophy, or SMA. QALSODY, our medicine to treat patients with superoxide dismutase 1 amyotrophic lateral sclerosis, or SOD1-ALS, received accelerated approval from the U.S. Food and Drug Administration, or FDA, in April 2023 and marketing authorization under exceptional circumstances from the European Medicines Agency, or EMA, in May 2024. In addition, we and Biogen are currently developing numerous other investigational medicines to treat neurodegenerative diseases, including medicines in development to treat people with amyotrophic lateral sclerosis, or ALS, SMA, Alzheimer’s disease, or AD, and Parkinson’s disease, or PD. In addition to these medicines, our collaborations with Biogen include a substantial research pipeline that addresses a broad range of neurological diseases. From inception through June 30, 2024, we have received more than $3.9 billion in payments from our Biogen collaborations, including payments to purchase our stock.


Under our 2013 strategic neurology collaboration, we earned a $20 million milestone payment from Biogen when the EMA approved Biogen’s Marketing Authorization Application, or MAA, filing of QALSODY in the second quarter of 2024. We recognized this milestone payment as R&D revenue in full in the second quarter of 2024 because we did not have any remaining performance obligations related to the milestone payment. We will achieve the next milestone payment for QALSODY of $10 million if the Ministry of Health, Labour and Welfare of Japan approves Biogen’s Japanese New Drug Application filing of QALSODY.


In the second quarter of 2024, Biogen's option to license ION582, an investigational antisense medicine for the potential treatment of Angelman Syndrome, expired unexercised. As a result, we recognized $30 million of R&D revenue from previously deferred milestone payments related to the ION582 study because we did not have any remaining performance obligations. We will achieve the next milestone payment of $25 million if Biogen advances IONIS-MAPTRx into Phase 3 development under our 2012 neurology collaboration.


During the three and six months ended June 30, 2024 and 2023, we earned the following revenue from our relationship with Biogen (in thousands, except percentage amounts):

 
Three Months Ended
June 30,
    
Six Months Ended
June 30,
  
   
2024
    
2023
    
2024
    
2023
  
Revenue from our relationship with Biogen
  
$
138,159
    
$
97,402
    
$
197,395
    
$
167,903
  
Percentage of total revenue
    
61
%
    
52
%
    
57
%
    
53
%


Our condensed consolidated balance sheets at June 30, 2024 and December 31, 2023 included deferred contract revenue of $241.8 million and $307.4 million, respectively, from our relationship with Biogen.


Otsuka


In 2023, we entered into an agreement with Otsuka Pharmaceutical Co., Ltd., or Otsuka, to commercialize donidalorsen in Europe. In the second quarter of 2024, we expanded the agreement to include commercialization rights for donidalorsen in the Asia-Pacific region in addition to Europe. As a result, we received a $20 million upfront payment from Otsuka. Under the amended agreement, we are eligible to receive up to $290 million, which is comprised of $85 million in upfront payments, up to $65 million in regulatory milestone payments and up to $140 million in sales milestone payments over the term of the collaboration. In addition, we are eligible to receive tiered royalties up to 30 percent on net sales. We are responsible for completing the ongoing development of donidalorsen. We retained the rights to commercialize donidalorsen in the U.S. and in the rest of the world, assuming regulatory approvals. From inception through June 30, 2024, we have received $85 million in payments from Otsuka.


We identified two performance obligations under our amended agreement for the Asia-Pacific region, comprised of our license of donidalorsen to Otsuka and R&D services for donidalorsen. We allocated the transaction price of $20 million based on the estimated stand-alone selling price of each performance obligation as follows:

●
$17.5 million for the license of donidalorsen; and
●
$2.5 million for the R&D services for donidalorsen.


In the second quarter of 2024, we recognized $17.5 million as revenue in full because Otsuka had full use of the license without any continuing involvement from us. We are recognizing revenue for our R&D services performance obligation as we perform services based on our effort to satisfy our performance obligation relative to our total effort expected to satisfy our performance obligation. We currently estimate we will satisfy our R&D services performance obligation in March 2026. We will achieve the next payment of $15 million if the EMA accepts a MAA filing for donidalorsen in the EU under this collaboration.


During the three and six months ended June 30, 2024 and 2023, we earned the following revenue from our relationship with Otsuka (in thousands, except percentage amounts):

 
Three Months Ended
June 30,
    
Six Months Ended
June 30,
  
   
2024
    
2023
    
2024
    
2023
  
Revenue from our relationship with Otsuka
  
$
18,905
    
$
—
    
$
19,691
    
$
—
  
Percentage of total revenue
    
8
%
    
0
%
    
6
%
    
0
%


Our condensed consolidated balance sheets at June 30, 2024 and December 31, 2023 included deferred contract revenue of $8.8 million and $8.5 million, respectively, from our relationship with Otsuka.


Roche


We have three collaborations with Hoffmann-La Roche Inc. and F. Hoffmann-La Roche Ltd, collectively Roche: one to develop treatments for Huntington’s disease, or HD, one to develop IONIS-FB-LRx for the treatment of immunoglobulin A, or IgA, nephropathy, or IgAN, and one to develop RNA-targeted programs for AD and HD. From inception through June 30, 2024, we have received more than $345 million in payments from our Roche collaborations.


In July 2024, Roche discontinued development of IONIS-FB-LRx for the treatment of geographic atrophy, or GA, following the completion of the Phase 2 study, which showed a favorable safety profile and target engagement, but insufficient efficacy to advance into Phase 3 development.


Over the term of the IONIS-FB-LRx collaboration for the treatment of IgAN, we are eligible to receive up to $430 million, which is comprised of a $35 million license fee, up to $25 million in development milestone payments, up to $90 million in regulatory milestone payments and up to $280 million in sales milestone payments. We will achieve the next payment of $23.5 million if Roche advances IONIS-FB-LRx for the treatment of IgAN under this collaboration.


During the three and six months ended June 30, 2024 and 2023, we earned the following revenue from our relationship with Roche (in thousands, except percentage amounts):

   
Three Months Ended
June 30,
    
Six Months Ended
June 30,
  
   
2024
    
2023
    
2024
    
2023
  
Revenue from our relationship with Roche
  
$
11,089
    
$
5,754
    
$
22,595
    
$
8,121
  
Percentage of total revenue
    
5
%
    
3
%
    
7
%
    
3
%


Our condensed consolidated balance sheets at June 30, 2024 and December 31, 2023 included deferred contract revenue of $15.0 million and $36.7 million, respectively, from our relationship with Roche.