RNA - Competitive Landscape, 2022

This “RNA- Competitive landscape, 2022” report provides comprehensive insights about 85+ companies and 100+ drugs in RNA Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

RNA: Understanding

RNA: Overview

RNA interference (RNAi) is one of the pathways, collectively named RNA silencing pathways that employ small RNAs as guides for sequence-specific silencing. RNAi was discovered in C. elegans and defined as sequence-specific mRNA degradation induced by long double-stranded RNA (dsRNA). The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all human cells.

RNA interference (RNAi) or Post-Transcriptional Gene Silencing (PTGS) is a conserved biological response to double-stranded RNA that mediates resistance to both endogenous parasitic and exogenous pathogenic nucleic acids, and regulates the expression of protein-coding genes. This natural mechanism for sequence-specific gene silencing promises to revolutionize experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas.

Report Highlights

• In April 2022, Alnylam Pharmaceuticals, Inc.,the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022 under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review
• In November 2021, Arrowhead Pharmaceuticals Inc. announced that its collaborator, Janssen Pharmaceuticals, Inc., (Janssen) one of the Janssen Pharmaceutical Companies of Johnson & Johnson, has disclosed its collaboration with Arrowhead on investigational compound JNJ-75220795, which is in a Phase I clinical study
• In November 2021, Arrowhead Pharmaceuticals announced that it entered into an exclusive license agreement with GSK under which GSK will develop and commercialize ARO-HSD.
• In March 2020, Silence Therapeutics announced a strategic collaboration with AstraZeneca to discover, develop and commercialize small interfering RNA (siRNA) therapeutics for the treatment of cardiovascular, renal, metabolic and respiratory diseases. The collaboration draws on Silence's extensive experience as a leader in the discovery, development, and delivery of siRNA therapeutics together with AstraZeneca's industry-leading expertise in disease biology and target identification with the aim of developing first-in-class and differentiated therapeutics to address significant unmet need.
• In October 2019, Dicerna Pharmaceuticals announced a research collaboration and licensing agreement with Roche to develop novel therapies for the treatment of chronic hepatitis B virus. The collaboration will focus on worldwide development and commercialization of DCR-HBVS, Dicerna's investigational therapy in Phase I clinical development. The collaboration also includes the discovery and development of therapies targeting multiple additional human and viral genes associated with HBV infection using the technology platforms of both companies
• In July 2019, Arrowhead Pharmaceuticals announced US FDA granted orphan drug designation to ARO-ANG3 for the treatment of HoFH

Company Profiles & their Late-stage Drug Profiles

1. Company Overview: Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam is executing on its "Alnylam P5x25" strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

Product Description: Vutrisiran

Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hATTR and wild-type ATTR (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections.

2. Company Overview: Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. Arrowhead is focused on developing innovative drugs for diseases with a genetic basis, characterized by the overproduction of one or more proteins.

Product Description: ARO-APOC3

ARO-APOC3 is a subcutaneously administered RNAi therapeutic designed to reduce the production of Apolipoprotein C-III (Apoc-III), a component of triglyceride-rich lipoproteins (TRLs) including VLDL and chylomicrons, and is a key regulator of triglyceride metabolism. The company believes that knocking down the hepatic production of Apoc-III may result in reduced VLDL synthesis and assembly, enhanced breakdown of TRLs, and better clearance of VLDL and chylomicron remnants. It is currently being developed as a potential treatment for patients with severe hypertriglyceridemia (SHTG), familial chylomicronemia syndrome (FCS), and mixed dyslipidemia. Arrowhead's investigational RNA interference (RNAi) therapeutic is currently under phase III trial for FCS and phase II for mixed dyslipidemia and SHTG. Furthermore, Arrowhead has successfully completed the phase I trial for hypertriglyceridemia and FCS.

3. Company Overview: Eli Lily and Company

Lilly was founded in 1876 by Colonel Eli Lilly, a man committed to creating high-quality medicines that met real needs in an era of unreliable elixirs peddled by questionable characters. Company conduct business consistent with all applicable laws and are honest in dealings with customers, employees, shareholders, partners, suppliers, competitors and the community.

Product Description: LY3561774

LY3561774, is the first clinical-stage candidate to emerge from Dicerna's collaboration with Lilly. The successful IND filing for LY3561774 marked an important milestone. Currently, the drug is in Phase II stage of development for the treatment of Dyslipidemia

4. Company Overview: Dicerna Pharmaceuticals

Dicerna Pharmaceuticals, Inc (subsidiary of Nova Nordisk) is a biopharmaceutical company using ribonucleic acid (RNA) interference (RNAi) to develop medicines that silence genes that cause disease. The Company's proprietary GalXC™ technology is being applied to develop potent, selective, and safe RNAi therapies for treatment of rare diseases, chronic liver diseases, cardiovascular diseases, neurodegenerative diseases, pain, and viral infectious disease. Dicerna aims to treat disease by addressing the underlying causes of illness with capabilities that extend beyond the liver to address a broad range of diseases, focusing on target genes where connections between gene and disease are well understood and documented.

Product Description: DCR-HBVS

DCR-HBVS is an investigational GalXC RNAi therapy in development with Roche for the treatment of chronic hepatitis B virus (HBV) infection Chronic HBV increases the risk of developing liver failure, liver cancer, or cirrhosis - a condition that permanently scars the liver. DCR-HBVS has the potential to contribute to the ultimate goal of achieving a functional cure in patients with chronic HBV and is currently being studied by Roche as part of Phase II clinical trial in combination with other treatment regimens to induce functional cures in patients. Moreover, it is also studied in phase I for chronic hepatitis B.

DCR-HBVS is designed to knock down selectively viral hepatitis B surface antigen (HBsAg) messenger RNA (mRNA) and protein expression in liver hepatocytes. HBsAg is required for the HBV virus lifecycle and is a marker used to determine the extent of chronic HBV viral activity. Preclinical data have demonstrated a greater than 99.9% reduction in circulating HBsAg in mouse models of HBV infection.

5. Company Overview: Vir Biotechnology

VIR-2218 (ALN-HBV02) is a subcutaneously administered, investigational RNAi therapeutic targeting the hepatitis B virus (HBV) genome for the treatment of chronic HBV infection by Alnylam collaborators at Vir Biotechnology. ALN-HBV02 is designed to inhibit expression of all HBV proteins, including hepatitis B surface antigen (HBsAg). ALN-HBV02 utilizes Enhanced Stabilization Chemistry-Plus (ESC+)-GalNAc delivery platform. Vir presented results from the Phase II study of ALN-HBV02, showing a positive safety profile and reduction of hepatitis B surface antigen. The data demonstrated that BRII-835 (VIR-2218), an investigational small interfering ribonucleic acid (siRNA) that mediates RNA interference (RNAi), results in dose-dependent reduction in hepatitis B surface antigen (HBsAg) in Chinese patients with chronic HBV infection who received two doses of BRII-835 (VIR-2218). Brii Bio and Vir Biotechnology have agreed to commence clinical development efforts in China for VIR-2218.

Product Description: VIR-2218

VIR-2218 (ALN-HBV02) is a subcutaneously administered, investigational RNAi therapeutic targeting the hepatitis B virus (HBV) genome for the treatment of chronic HBV infection by Alnylam collaborators at Vir Biotechnology. ALN-HBV02 is designed to inhibit expression of all HBV proteins, including hepatitis B surface antigen (HBsAg). ALN-HBV02 utilizes Enhanced Stabilization Chemistry-Plus (ESC+)-GalNAc delivery platform. Vir presented results from the Phase II study of ALN-HBV02, showing a positive safety profile and reduction of hepatitis B surface antigen. The data demonstrated that BRII-835 (VIR-2218), an investigational small interfering ribonucleic acid (siRNA) that mediates RNA interference (RNAi), results in dose-dependent reduction in hepatitis B surface antigen (HBsAg) in Chinese patients with chronic HBV infection who received two doses of BRII-835 (VIR-2218). Brii Bio and Vir Biotechnology have agreed to commence clinical development efforts in China for VIR-2218

Future of Competitive Landscape of RNA is estimated to be very strong. Key emerging drugs include Alnylam Pharmaceuticals, Vutrisiran and many other.

RNA Analytical Perspective

In-depth RNA analysis: Assessment of Products

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition - deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

RNA clinical assessment of products

The report comprises of comparative clinical assessment of products by development stage, product type, and route of administration, molecule type.

RNA Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing RNA drugs?
• How many RNA drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of RNA?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the RNA therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for RNA and their status?
• What are the key designations that have been granted to the emerging and approved drugs?

Key Players

• Alnylam Pharmaceuticals
• Suzhou Ribo Life Science
• Arrowhead Pharmaceuticals
• Sylentis
• Vir Biotechnology
• Arbutus Biopharma
• Silenseed
• OliX Pharmaceuticals
• Bio-Path Holdings
• Janssen Pharmaceuticals
• Eli Lilly and Company
• Dicerna Pharmaceuticals
• Amgen
• Sirnaomics
• Silence Therapeutics
• Sylentis
• Silence Therapeutics
• Suzhou Ribo Life Science
• Phio Pharmaceuticals
• Amylon Therapeutics
• Aro Biotherapeutics
• DTx Pharma
• BONAC Corporation

Key Products

• Vutrisiran
• SR061
• ARO-APOC3
• DCR-PHXC
• SYL-1001
• Fitusiran
• Zilebesiran
• VIR-2218
• AB 729
• Antisense K-ras RNA gene therapy
• OLX 101A
• Lumasiran
• Prexigebersen-A
• JNJ-3989
• LY 3561774
• DCR-HBVS
• Olpasiran
• STP705
• ARO-HSD
• ARO-MUC5AC
• RBD1016
• ANGPTL3 siRNA
• SLN124
• JNJ-75220795
• LY3819469
• ARO-HIF2
• SYL 1801
• STP707
• DRC-AUD
• SLN360
• SR043
• SR044
• PH 894
• PH 762PF-06650833
• Polyoxidonium
• Remimazolam
• OP-101
• Rayaldee
• OT-101
• ADM03820
• OLT 1177
• NVXCoV 2373
• DFV890
• Crizanlizumab
• MAS-825
• NBT-NM108
• Ambrisentan
• Sargramostim
• Gam-COVID-Vac
• mRNA-1283
• MP1032
• Molnupiravir
• M5049
• Covifenz
• INOmax
• MW33
• RNA convalescent plasma
• LB1148
• Mavrilimumab

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