ALN-AAT02 / Alnylam, Novo Nordisk - LARVOL DELTA

Dicerna Announces Third Quarter 2020 Financial Results and Provides a Business Update (Businesswire) - "Anticipated Upcoming Milestones: DCR-A1AT/ALN-AAT02: Program selection for advancement into Phase 2 in the first quarter 2021."

Clinical • Alpha-1 Antitrypsin Deficiency

Alnylam Pharmaceuticals Inc (ALNY) Q3 2020 Earnings Call Transcript (The Motley Fool) - "And obviously have advanced a molecule alpha one ALN AAT-02 into development. Now earlier this year, we decided to partner that program together with Dicerna, who had their own independent effort as well. And so the combined effort between Alnylam and Dicerna now really is positioned to advance quite nicely and is in -- currently going to be -- is in a Phase I/II study, we'll be going into a Phase II/III in due course."

Trial status • Alpha-1 Antitrypsin Deficiency

A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease (clinicaltrials.gov) - P1/2; N=32; Terminated; Sponsor: Alnylam Pharmaceuticals; N=96 ➔ 32; Trial completion date: Jun 2021 ➔ Jun 2020; Active, not recruiting ➔ Terminated; Trial primary completion date: Jun 2021 ➔ Jun 2020; The sponsor decided not to proceed with Part B for business reasons.

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • AAT

Alnylam submits Clinical Trial Authorization (CTA) application for ALN-AAT02, an investigational RNAi therapeutic for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (alpha-1 liver disease) (Businesswire) - P1/2, N=NA; "Alnylam Pharmaceuticals...announced today that it has submitted an Clinical Trial Authorization (CTA) application to the Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study of ALN-AAT02...for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease)....'we look forward to evaluating the safety, pharmacodynamics, and clinical activity of this molecule in a Phase 1/2 study in healthy volunteers and adults with alpha-1 liver disease, which we expect to initiate by year-end 2018.'"

European regulatory • Trial initiation date • Alpha-1 Antitrypsin Deficiency • Genetic Disorders

Blackstone and Alnylam enter $2bn drug development deal (Pharmaceutical-technology.com) - "Blackstone has agreed to provide up to $2bn to accelerate the development of Alnylam Pharmaceuticals’ RNA interference (RNAi) drugs across various debilitating diseases....Last week, Alnylam partnered with Dicerna Pharmaceuticals to develop and commercialise RNAi therapies for alpha-1 antitrypsin (A1AT) deficiency-associated liver disease."

Financing • Alpha-1 Antitrypsin Deficiency • Genetic Disorders

Alnylam and Dicerna form RNAi therapeutics collaboration on alpha-1 antitrypsin deficiency-associated liver disease and complete cross-license agreement for primary hyperoxaluria programs (Businesswire) - "Alnylam Pharmaceuticals, Inc....and Dicerna Pharmaceuticals, Inc....announced today the formation of a development and commercialization collaboration on investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease (alpha-1 liver disease)....Under the agreement, Dicerna assumes responsibility for both ALN-AAT02 and DCR-A1AT at its cost, and may progress one or both of these investigational medicines through clinical development....At the completion of Phase 3, Alnylam has the no-cost opportunity to opt-in to commercialize the selected candidate in countries outside the U.S...."

Licensing / partnership

Alnylam announces 2020 product and pipeline goals and provides updates at R&D Day (Businesswire) - "Announces Initial Positive Clinical Results with ALN-AAT02...Initial results demonstrate an improved hepatic safety profile for ALN-AAT02 – an ESC+ GalNAc conjugate – compared with ALN-AAT01, with robust knockdown of alpha-1 antitrypsin and favorable tolerability with no subjects showing clinically significant elevations in liver alanine transaminase levels."

P1/2 data

ALN-AAT02: Initial data from P1/2 trial (NCT03767829) for alpha-1 antitrypsin deficiency liver disease in late 2019 (Alnylam) - Corporate Presentation

P1/2 data

A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease (clinicaltrials.gov) - P1/2; N=96; Active, not recruiting; Sponsor: Alnylam Pharmaceuticals; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

A 2-Part Safety and Tolerability Study of ALN-AAT02 in Healthy Volunteers and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease (clinicaltrialsregister.eu) - P1/2; N=96; Ongoing; Sponsor: Alnylam Pharmaceuticals, Inc.

Clinical • New P1/2 trial

ALN-AAT02: Initial data from P1/2 trial (NCT03767829) for alpha-1 antitrypsin deficiency liver disease in late 2019 (Alnylam) - Corporate Presentation

P1/2 data

Alnylam Pharmaceuticals reports fourth quarter and full year 2018 financial results and highlights recent period activity (Businesswire) - "Alnylam announces today that it has initiated a Phase 1 study of ALN-AAT02, with initial results expected in 2019."

P1 data • Trial status

A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease (clinicaltrials.gov) - P1/2; N=96; Recruiting; Sponsor: Alnylam Pharmaceuticals; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open