Release Date: October 31, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Alnylam Pharmaceuticals Inc (ALNY, Financial) achieved 34% year-over-year growth in global net product revenue, generating $420 million across its four marketed products.
- The TTR franchise showed strong performance with $309 million in global net product revenues, marking a 34% increase compared to the third quarter of 2023.
- The company made significant progress with its TTR franchise, including sharing additional results from the HELIOS-B study and filing regulatory submissions in the US and EU.
- Alnylam Pharmaceuticals Inc (ALNY) initiated a Phase 1 study of ALN-HTT02 in Huntington's disease and announced encouraging initial results from the multi-dose portion of the Phase 1 study of mivelsiran in early onset Alzheimer's disease.
- The company ended the quarter with $2.8 billion in cash, cash equivalents, and marketable securities, reflecting improved operating performance.
Negative Points
- International TTR franchise growth decreased by 9% compared to the second quarter of 2024, driven by a gross to net adjustment in Portugal and large orders in partner markets in Q2.
- Net revenue from collaborations decreased by $370 million compared to the previous year, primarily due to the recognition of a $310 million upfront payment from Roche in Q3 2023.
- Non-GAAP R&D expenses increased by 12% compared to the same period last year, driven by increased costs associated with preclinical activities and clinical studies.
- Non-GAAP SG&A expenses increased by 19% compared to the same period in 2023, primarily due to higher marketing costs and preparations for the potential launch of AMVUTTRA for cardiomyopathy.
- The company decided to stop clinical development of ALN-KHK, an investigational RNAi therapeutic targeting ketohexokinase for type 2 diabetes mellitus, to optimize resource allocation.
Q & A Highlights
Q: Can you elaborate on the work done to improve RNAi delivery to adipose tissue and muscle, and what makes your approach advantageous?
A: Pushkal Garg, Chief Medical Officer, explained that Alnylam is excited about RNAi therapeutics' emerging profile, which allows for sustained knockdown of disease-causing proteins with infrequent administration. The company is guided by human genetics in selecting diseases to pursue. Adipose and muscle are new tissues of interest, and more updates will be provided at an R&D Day early next year.
Q: Regarding ALN-HTT02, what are you looking for in Phase 1 data, and how does it compare to other studies?
A: Pushkal Garg highlighted that ALN-HTT02 is Alnylam's third CNS program, showing promising preclinical results in nonhuman primates. The program targets an upstream portion of the Huntington gene, potentially offering unique efficacy. The Phase 1 study will assess safety, tolerability, and changes in mutant Huntington levels, with active start-up in the UK, Canada, and the US.
Q: How has the entry of a second silencer affected the polyneuropathy market and your trajectory?
A: Tolga Tanguler, Chief Commercial Officer, noted that despite new competition, Alnylam's products have shown robust growth, driven by quarterly administration, patient retention, and rapid knockdown capabilities. The company has established clear guidelines to ensure compliance and is seeing strong demand in the polyneuropathy market.
Q: What are your thoughts on payer discussions ahead of potential label expansion for AMVUTTRA?
A: Tolga Tanguler stated that payer discussions focus on the value proposition driven by clinical outcomes. The HELIOS-B results are compelling, and Alnylam's patient access philosophy includes value-based agreements and appropriate rebates. More details will be shared closer to the PDUFA date.
Q: How should we think about SG&A spending levels for the upcoming launches of vutrisiran in ATTR-CM?
A: Jeff Poulton, Chief Financial Officer, indicated that SG&A spending will see double-digit growth as Alnylam prepares for the US and EU launches. The company has made significant progress in hiring and preparing for the launch, with more guidance to be provided in February.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
