Arrowhead Hosts Investor & Analyst R&D Day to Introduce TRiM™ Platform and Lead RNAi-based Drug Candidates

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Arrowhead Hosts Investor &...

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR), is hosting an investor &

analyst R&D day in New York today to introduce its proprietary Targeted

RNAi Molecule (TRiM^TM) platform and review its pipeline of

RNAi therapeutic candidates.

Chris Anzalone, Ph.D., president and chief executive officer of

Arrowhead Pharmaceuticals, said: “We are excited to introduce our new

TRiM^TM platform and the first development candidates to be

advanced: ARO-HBV, targeting chronic hepatitis B infection; ARO-AAT,

targeting alpha-1 antitrypsin deficiency liver disease; and, ARO-APOC3

and our newest candidate ARO-ANG3, targeting hypertriglyceridemia. TRiM^TM

is characterized by its ligand-mediated delivery strategy, structural

simplicity, and trigger selection technology, which is an important part

of TRiM^TM that enables us to rapidly develop what we believe

are optimal RNAi therapies. It also allows tissue-specific targeting and

to that end we are announcing today that we have seen some very exciting

results using TRiM^TM technology to target genes and diseases

in the lung. This opens up a host of new opportunities for disease

targets previously not accessible to RNAi therapeutics and Arrowhead is

thrilled to be leading the industry in these efforts.”

The R&D day will feature presentations by key opinion leaders, Jeffrey

Teckman, M.D. (St. Louis University School of Medicine), Stephen

Locarnini, M.D., Ph.D. (Victorian Infectious Diseases Reference

Laboratory), and Ira Goldberg, M.D. (NYU Langone Medical Center), as

well as Arrowhead management.

A live and archived webcast of the event can be accessed on the Events

and Presentations page on the Investors section of the Arrowhead

website at http://ir.arrowheadpharma.com/events.cfm.

Agenda and Approximate Times for Discussion Topics

12:00-12:45 Lunch
1:00-1:05 Welcome and Introductions – Vince

Anzalone, CFA
1:05-1:35 TRiM^TM Platform and Pipeline

Review – Chris Anzalone, Ph.D.
1:35-1:55 Hepatitis B – Stephen

Locarnini, M.D., Ph.D.
1:55-2:05 ARO-HBV – Bruce Given, M.D.
2:05-2:25

Alpha-1 Antitrypsin Deficiency Liver Disease – Jeffrey Teckman, M.D.
2:25-2:35

ARO-AAT – Bruce Given, M.D.
2:35-2:55 Hypertriglyceridemia – Ira

Goldberg, M.D.
2:55-3:05 ARO-APOC3 and ARO-ANG3 – Bruce Given, M.D.
3:05-3:15

Concluding Remarks – Chris Anzalone, Ph.D.
3:15-3:35 Q & A – Panel

Select R&D Day Highlights

Targeted RNAi Molecule Platform (TRiM^TM)

Arrowhead’s Targeted RNAi Molecule platform, or TRiM^TM,

utilizes ligand-mediated delivery and is designed to enable multi-tissue

targeting, while being structurally simple. Active targeting has been

core to Arrowhead’s development strategy and the TRiM^TM

platform builds on more than a decade of work on actively targeted drug

delivery vehicles. Arrowhead scientists now have the ability to

progressively “TRiM” away extraneous features and chemistries and retain

optimal pharmacologic activity.

New drug candidates utilizing this technology can achieve high levels of

target gene knockdown, without an active endosomal escape component, as

was required with prior technology platforms.

The TRiM^TM platform represents an evolution in RNAi

therapeutics from biologic complexity to small molecule precision and

execution. The TRiM^TM platform comprises the following

components optimized, as needed, for each drug candidate: high affinity

targeting ligands; various linker chemistries; structures that enhance

pharmacokinetics; and highly potent RNAi triggers with sequence specific

stabilization chemistries. The platform offers several competitive

advantages, including:

Simplified manufacturing at reduced cost
Multiple routes of administration (subcutaneous, intravenous, and

inhaled)
Faster time to clinical candidates
Wide safety margins
Promise of taking RNAi to tissues beyond the liver

Extra-Hepatic Targeting

Arrowhead believes that for RNAi to reach its true potential, it must

target organs outside the liver. Arrowhead is leading this expansion

with the TRiM^TM platform that holds the promise of reaching

multiple tissues, including the lung and tumors. ARO-Lung1, the first

candidate against an undisclosed gene target in the lung, reached almost

90% target knockdown following inhaled administration in rodents. In

addition, the ARO-HIF2 candidate targeting renal cell carcinoma achieved

85% target gene knockdown in a rodent tumor model. Clinical Trial

Authorization (CTA) filings are planned in Q4 2018 and in 2019 for

ARO-Lung1 and ARO-HIF2, respectively.

ARO-AAT

ARO-AAT, Arrowhead’s second generation subcutaneously administered

clinical candidate for the treatment of alpha-1 antitrypsin deficiency

liver disease, achieved up to 92% knockdown in monkeys, thought to be

near complete suppression of hepatic production of the alpha-1

antitrypsin protein. In non-GLP rat and monkey exploratory toxicology

studies, no changes in clinical chemistries were observed and no

histopathology suggestive of organ toxicity at doses up to 300 mg/kg

(100x expected human dose). Arrowhead plans to file a CTA in Q1 2018 to

initiate clinical studies, pending completion of GLP toxicology studies.

ARO-HBV

ARO-HBV, Arrowhead’s third generation subcutaneously administered

clinical candidate for the treatment of chronic hepatitis B virus

infection, achieved up to 99.9% knockdown of hepatitis B surface antigen

(HBsAg), e-antigen (HBeAg), and HBV DNA in rodent models. In a non-GLP

rat exploratory toxicology study, no changes in clinical chemistries or

histopathology changes suggestive of organ toxicity were observed at

doses up to 300 mg/kg (75-100x expected human dose). Arrowhead plans to

file a CTA in Q2 2018 to initiate clinical studies, pending completion

of cross-reactivity and GLP toxicology studies.

ARO-APOC3 and ARO-ANG3

Arrowhead is expanding its cardiovascular disease portfolio utilizing

the TRiM™ platform. Added to existing programs, ARO-LPA and ARO-AMG1,

both partnered with Amgen; will be ARO-APOC3, targeting apolipoprotein

C-III, and ARO-ANG3, targeting angiopoietin-like protein 3 (ANGPTL3).

ARO-APOC3 and ARO-ANG3 will both be developed for the treatment of

hypertriglyceridemia. CTA filings are planned for one or both candidates

by the end of 2018.

About 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. RNA interference, or RNAi, is a

mechanism present in living cells that inhibits the expression of a

specific gene, thereby affecting the production of a specific protein.

Arrowhead’s RNAi-based therapeutics leverage this natural pathway of

gene silencing.

For more information, please visit www.arrowheadpharma.com,

or follow us on Twitter @ArrowheadPharma.

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please visit http://ir.arrowheadpharma.com/alerts.cfm.

Safe Harbor Statement under the Private Securities Litigation Reform

Act:

This news release contains forward-looking statements within the

meaning of the “safe harbor” provisions of the Private Securities

Litigation Reform Act of 1995. These statements are based upon our

current expectations and speak only as of the date hereof. Our actual

results may differ materially and adversely from those expressed in any

forward-looking statements as a result of various factors and

uncertainties, including the safety and efficacy of our product

candidates, the duration and impact of regulatory delays in our clinical

programs, our ability to finance our operations, the future success of

our scientific studies, our ability to successfully develop drug

candidates, the timing for starting and completing clinical trials,

rapid technological change in our markets, and the enforcement of our

intellectual property rights. Our most recent Annual Report on Form 10-K

and subsequent Quarterly Reports on Form 10-Q discuss some of the

important risk factors that may affect our business, results of

operations and financial condition. We assume no obligation to update or

revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

View source version on businesswire.com: http://www.businesswire.com/news/home/20170914005965/en/

Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
ir@arrowheadpharma.com
or
Investors

and Media:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578
britchie@lifesciadvisors.com
www.lifesciadvisors.com

Source: Arrowhead Pharmaceuticals, Inc.

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