Alnylam Pharmaceuticals

Novartis has signed an up to $5.2 billion licensing and options deal with China-based Argo Biopharmaceutical for experimental cardiovascular drugs using RNA interference technology.

Zilebesiran demonstrated clinically meaningful reductions in office systolic blood pressure (-5.0 mmHg) at three months in patients with uncontrolled hypertension and high cardiovascular risk.

Novo Nordisk announced plans to initiate Phase 3 trials for coramitug, a potential first-in-class amyloid depleter antibody for ATTR amyloidosis with cardiomyopathy, following successful completion of a Phase 2 study.

The complement-based therapeutics field is experiencing significant growth following recent FDA approvals, including Novartis' alternative complement inhibitor for PNH and SYFOVRE approval.

Novartis has received FDA approval for Leqvio (inclisiran), a first-in-class siRNA therapy that reduces LDL cholesterol with just two injections per year.

• Scholar Rock has appointed David L. Hallal as CEO, transitioning from his role as Chairman of the Board, as the company prepares for the global launch of apitegromab for spinal muscular atrophy. • The company has bolstered its leadership team with three key appointments: Akshay Vaishnaw as President of R&D, R. Keith Woods as Chief Operating Officer, and Vikas Sinha as Chief Financial Officer. • Apitegromab, a first-in-class muscle-targeted treatment for SMA, has received FDA priority review following successful Phase 3 trials, with potential approval and launch expected later this year.

• An FDA advisory committee voted 9-3 in favor of approving Alnylam's Onpattro (patisiran) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), with a final decision expected by October 8th. • Despite FDA reviewers questioning the clinical significance of Onpattro's modest efficacy in the APOLLO-B trial, the panel was swayed by the drug's established safety profile and potential to address the underlying disease mechanism. • If approved, Onpattro would compete with Pfizer's Vyndamax/Vyndaqel (tafamidis), which generated $2.5 billion in sales last year, marking another milestone for RNA interference technology following its historic 2018 approval for ATTR polyneuropathy.

The FDA has approved Oxlumo (lumasiran), the first-ever treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare genetic disorder that causes kidney damage through excessive oxalate production.

Moderna has filed patent infringement lawsuits against Pfizer/BioNTech in multiple countries, claiming their Comirnaty vaccine infringes on mRNA technology patents that Moderna pioneered and invested billions in developing.

Alnylam's RNAi drug vutrisiran demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in ATTR cardiomyopathy patients, with efficacy increasing to 33% in patients not taking Pfizer's tafamidis.