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| We also announced positive initial results from the Phase 1 study of ALN-KHK with robust target engagement and encouraging safety that supported continued development as a novel treatment for Type 2 diabetes |
| We believe all of this puts us on track with our Alnylam P5x25 goals, making Alnylam a top-tier biotech developing and commercializing transformative medicines for patients around the world with rare and prevalent diseases driven by a high-yielding pipeline, the first and/or best-in-class product candidates from our organic product engine, all while delivering exceptional financial results |
| The early clinical data from this program continue to be very encouraging |
| Zilebesiran also made exciting progress for hypertension with encouraging results in the KARDIA-1 Phase 2 study |
| And I just want to kind of underscore that we’re really confident on the potential effects of [indiscernible] outcomes and the [indiscernible] was already well designed and executed to deliver these results |
| Assuming positive data and regulatory approval, we believe that vutrisiran will be well positioned to serve patients with ATTR cardiomyopathy, addressing unmet needs with the potential for a highly competitive market-leading profile, including a unique mechanism of action that works upstream of protein production and enables rapid knockdown of TTR, an impactful clinical profile with the potential to reduce mortality and CV hospitalizations and helping exorable decline in functional capacity and quality of life, an attractive quarterly dosing schedule that aligns with physician visits, support strong adherence and provides the flexibility of in-office or at-home dose administration, and favorable payer dynamics, where coverage under Medicare Part B is expected to result in the majority of patients having zero out-of-pocket costs and where we also expect payers to favor monotherapies for the next several years |
| We think we have ample power for those, and we think those are great endpoints based on what we learned from APOLLO-B to really demonstrate what we seem to see – be seeing is in terms of differentiated profile |
| Today, we are announcing enhancements to the HELIOS-B statistical plan to optimize the study for success and a strong and competitive label |
| Q4 was another strong quarter for our commercial portfolio, with both our TTR franchise and our Ultra-Rare franchise, delivering growth in excess of 30% compared with Q4 2022 |
| The quarter-over-quarter growth was driven by the following: demand growth driven by AMVUTTRA’s performance, including recent launches in Spain and Italy and continued strong ONPATTRO performance in markets, where AMVUTTRA is not yet available, an improvement in gross to net deductions in the quarter following price reductions in Q3, primarily in Germany that were highlighted on our Q3 call |
| We are confident that the study updates I’ve just reviewed, refinements to endpoints and extension of the blinded study period further enhance the power of the study and our confidence that HELIOS-B will deliver a positive result |
| This remarkable and unique pace of innovation puts us in a great position to have a robust, self-sustainable pipeline that can deliver meaningful impact to patients across multiple disease areas |
| So I think the changes we’ve made today, I’m glad you agree why and a 3-month addition to further increase the robustness of the encouraging data we already see from APOLLO and APOLLO-B, I think just further consolidates that we anticipate a positive study, and we’re confident about that |
| Based on our assumptions, as informed by APOLLO-B and other studies as well as the conduct and execution of HELIOS-B, we remain confident about HELIOS-B and its ability to deliver a positive result |
| That represents another tailwind that supports the success in the powering of the study |
| hATTR polyneuropathy patients are now on AMVUTTRA, a testament to its clearly differentiated market-leading profile, including rapid knockdown, reversal of polyneuropathy manifestations, well-established safety profile with thousands of patient years of experience, along with only 4 times a year administration |
| I mean, as Pushkal indicated, these analytical enhancements obviously makes us very confident and HELIOS-B visibility to show headed benefit on vutrisiran on top of TAP as well as obviously demonstrating the value of vutrisiran in a pure placebo |
| This includes the robust product growth for our four wholly earned commercial medicines, delivering $1.24 billion in global net product revenues and hitting the remarkable milestone of over 5,000 patients now on an Alnylam commercial RNAi therapeutic |
| Total net product revenues grew 32% in the fourth quarter versus prior year or 30% at a constant exchange rate as we continue to steadily increase the number of patients on each of our therapies |
| Our non-GAAP operating loss for 2023 was $60 million, representing a nearly $500 million improvement compared with 2022 primarily driven by strong top line results, both in product sales as well as revenue from collaborations, as I previously highlighted |
| In summary, AMVUTTRA with its market-leading profile is now available and reimbursed in all major markets across the U.S., Canada, Europe and Japan, driving robust patient and revenue growth in 2023 and we believe we are well-positioned for future growth |
| GIVLAARI and OXLUMO delivered $92 million in combined product sales during the fourth quarter, representing an 11% increase compared with the third quarter and a solid 30% growth compared with the fourth quarter of 2022 |
| As I have said, we are really pleased with the execution |
| So, with the profile that Tolga has described, we think we are in a really good position to drive broad commercial uptick of AMVUTTRA and obviously assuming positive results from HELIOS-B and approval |
| Beyond three months, we actually see good separation even in the monotherapy group even on outcomes and recurrent CV events specifically |
| And accordingly, as we shared at AHA last November, we were encouraged to see a favorable trend in mortality that separated beginning as early as 9 months and continuing to expand over 24 months |
| For OXLUMO, well positioned as the market-leading therapy in PH1, we delivered a robust 14% increase in product sales compared with the third quarter, which was driven by the following: a 9% increase in the U.S |
| And as we’ve looked also in the combination group over time, what we saw was favorable effects on outcomes there as well |
| In conclusion, we are very pleased with the results in the fourth quarter with both our TTR and Ultra-Rare franchises, which delivered strong growth in patients on therapy during the quarter as well as robust year-over-year growth in revenues with both franchises delivering 30% or greater growth versus the fourth quarter of 2022 |
| We are well positioned as we enter 2024 to continue delivering our medicines to more patients in need around the world |
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| Gross margin on product sales was 78% for the full year, representing a 6% decrease compared with 2022, primarily due to increased excess and obsolete charges due to canceling manufacturing commitments and the impairment of ONPATTRO inventory that had been manufactured for future demand associated with the ATTR cardiomyopathy indication for patisiran for which we did not receive regulatory approval in addition to a higher royalty rate payable on net sales of AMVUTTRA |
| Moreover, when we look at those data, actually, the lack of apparent separation was due to a few early events that happened in the patisiran arm in the first three months |
| So, Gena, in terms of your questions, the – as we have said, our TAP drop-in rates are lower than we expected |
| And if they would look at month 18, tafamidis was faring worse than placebo for mortality |
| And so we don’t think that we have, in any way, made it harder |
| So, if that doesn’t hit, are you then in like a more difficult position than you were before the stats plan change? Thank you |
| So, I think getting too over-indexed on one endpoint that went in the wrong direction, you have to be careful about that |
| In fact, the tafamidis rates at baseline were 40%, substantially less than the 50% we had assumed when powering the study |
| And we think these refinements to the analysis plan will only optimize the study for success in getting a strong and competitive label |
| If we were panicked, you would have seen those things some time ago |
| Obviously, there is always benefit risk |
| Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent periodic report on file with the SEC |
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