Dimension Therapeutics, Inc. - FORM 8-K - EX-99.1

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Exhibit 99.1

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Dimension Therapeutics, Inc.

Quality of Science. Quality of Life.

August 2016

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Forward Looking Statements

These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. For example, all statements we make regarding the initiation, timing, progress and results of our preclinical and clinical studies and our research and development programs, our ability to advance product candidates into, and successfully complete, clinical studies, the timing or likelihood of regulatory filings and approvals, our ability to develop manufacturing processes and engage third parties to manufacture our product candidates for late-stage clinical use or at commercial scale, the success of strategic partnerships, if any, and our expected cash, cash equivalents and marketable securities at year end are forward looking. All forward?looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. These statements are also subject to a number of material risks and uncertainties that are described under the caption “Risk Factors” in Dimension Therapeutics’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in

Dimension Therapeutics’ additional filings with the Securities and Exchange Commission. Any forward?looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

The information contained in this presentation is not an offer to sell or the solicitation of an offer to buy the

Company’s common stock or any other securities of the Company.

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Dimension Therapeutics

A leader in Robust scientific gene therapy platform

discovering & Manufactured using mammalian process

developing new Deep expertise in liver biology

therapeutics for Unique focus on inherited metabolic diseases (IMDs)

people living with

devastating rare Highly experienced team; extensive track record in

diseases rare diseases

associated with

the liver Broad product pipeline addressing diseases with high

unmet medical need

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Clade E AAV-Based Gene Therapy Product Platform

Enhanced liver affinity; 16-110x greater liver targeting than other AAVs

Clade E Family of • Durable expression achieved in 5-10% hepatocytes

AAV Capsids • Well-established safety profile for use in human gene therapy

~25% of the population may be ineligible due to neutralizing antibodies

Proprietary • Mammalian cell-based system to leverage AAV biology

Industrialized • Scalable HeLa platform to optimize yield and quality

Manufacturing

Downstream process consistent with biologic and antibody standards

Approach

Source: Gao et al. 2004 J Virol; Fagiuoli et al. 2013 J Hepat

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Robust AAV Capsid and Transgene IP Estate

Capsid Intellectual Property

Transgene Intellectual

Exclusive, worldwide license to certain REGENXBIO Property

AAV vector technology spanning various serotypes

in up to 8 specified disease indications 1RGNX in-license

includes patent

+application directed to

Exclusive Disease Indications codon-optimized

2013 License 2015 Option & Licenseversion of the OTC gene

Hemophilia A* 2Citrullinemia Type 1

Hemophilia B* 2PKU2Exclusive WW

1 OTC Deficiency 2Wilson Diseasecollaboration and

license agreement with

GSDIa Option 1**UPENN

*License excludes AAV8 for hemophilia A or B; **Option to license rights to REGENXBIO AAV technology for

any 1 additional disease, subject to availability (diseases not licensed in whole or in part to a 3rd party)

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The Liver is a Central Organ for Rare Diseases

>400 Described Rare Monogenic Diseases Associated with the Liver

Subset Believe Well-Suited to Gene Therapy

Hemophilia Urea Cycle Disorders

Well-understood disease biology

Restoration to 5-10% of normal is

clinically meaningful

Storage Disorders Highly predictive preclinical modelsAminoacidopathies

Well-described biomarkers

Potential orphan drug designation

Source: Fagiuoli et al. 2013 J Hepat; carolguze.com/text/442-11-clinical_genetics.shtml; DMTX analyses; surgery.usc.edu/hepatobiliary/liversurgery.html

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Broad Product Pipeline Addressing Diseases with Significant Medical Need

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Pipeline Set to Deliver Major Development Milestones in Next 18 Months

Advancing 7 Program Portfolio

Hem B available data*•OTC initial data

File OTC IND•Hem B additional data

Value • Initiate OTC ph I/II clinical trial•File GSDIa IND

GSDIa US orphan designation•GSDIa EU orphan designation

OTC EU orphan designation •Hem A IND enabling studies

Expanded IMD portfolio •Citrullinemia type 1, PKU,

Wilson Disease development

candidates

201612-18 Months

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Inherited Metabolic Disease Programs

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Ornithine Transcarbamylase (OTC) Deficiency

Disease Patient Population

X-linked, urea cycle disorder (UCD)~10,000 patients WW

Genetic defect in ammonia~80% late onset

detoxification

Adverse cognitive & neurological•Most common UCD, ~55%

effects, coma, death

Treatments limited, non-curative•Severe, early onset in males

•Late onset in males & females

Sources: National Urea Cycles Disorder Foundation; NORD; Batshaw et al. Mol Gen Metab 2014; Tuchman et al 2008; Enns N Engl J Med 2007; Foschi et al. World J Gastro ESPS Manuscript no: 13209; carolguze.com/text/442-11-clinical_genetics.shtml; Dimension Therapeutics unpublished data

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DTX301: Replace OTC to Address Toxic Hyperammonemia

Well understood disease biology; Well-described Path to HPOC

highly predictive preclinical models Biomarkers Defined

• Serum ammonia

Durable Expression of Functional OTC in spf ash OTC Mouse • Urinary orotic acid • Granted orphan drug

designation (US/EU)

• Excess carbamoyl • hPOC serum ammonia

phosphate converted

to orotic acid • Adult males and at-risk

• Increased serum females

ammonia; neurotoxic

Source: Dimension Therapeutics unpublished data

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Glycogen Storage Disease Type Ia (GSDIa)

Disease Patient Population

Autosomal recessive, inborn error of~6,000 patients WW

glucose metabolism Dx at birth

Deficient glucose-6-phosphatase

(G6Pase)

Reduced QOL, long term risks•Most common GSD, ~25%

Strict diet and frequent feedings of•No approved drug therapies

uncooked starch or Glycosade®

Sources: Weinstein et al. Hum Gen Ther 2010; Lee et al. Hepatology 2012; Chou et al Nat Rev Endo 2010; Boers et al. Orphanet Journal of Rare Diseases 2014; www.curegsd.org; The Children’s Fund for GSD Research

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DTX401: Replace G6Pase to Address Life-Threatening Hypoglycemia

Well understood disease biology; Well-described Path to HPOC

highly predictive preclinical models Biomarkers Defined

• Single dose AAV8 gene therapy increases G6Pase • Blood glucose

• Corresponding reduction in hepatic glycogen • Glycogen stores • hPOC time to

hypoglycemia

Improved G6Pase Activity in Canines

• Glycogen build up in • Adult males and

liver females

• Inability to release

glucose, raises risk of

hypoglycemia

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Broad Inherited Metabolic Disease Portfolio

Well understood disease biology;Well-describedProduct

highly predictive preclinical modelsBiomarkersOpportunity

Wilson • Autosomal recessive copper disorder•WW prevalence >50K

Disease • Deficiency in copper transporter P-• Copperpatients

type ATPase

Autosomal recessive disorder•~50K prevalent patients

PKU • Phenylalanine

Deficiency in phenylalanine hydrolasedeveloped world

Autosomal recessive UCD

Citrullinemia •14% of all UCDs

Deficiency in argininosuccinate• Ammonia

Type 1 •WW prevalence ~2K

synthetase

Sources: Barends MGM 2014; Batshaw MGM 2014; Citrullinemia Type I NCBI Bookshelf; Engel et al Hum Mut 2009; Foschi World J Gastro 2015; Marti?n-Herna?ndez Orph J Rare Dis 2014; NORD; Ruder Ped Neuro 2014; Ruegger J Inherit Metab Dis 2014; Summar Acta Paediatr. 2008; Summar Crit Care Clin 2005; Tuchman et al Mol Genet Metab 2008; Berry Genet Med 2013; Hanley J Genet Disor Genet Rep 2013; Vockley Gen Med 2014; Zerjav Orph J Rare Dis 2015; Beinhardt Clin Gast Hepat 2014; Bull Nat Gen 1993; EASL Prac Guide; Gomes Ann Hum Bio 2015; Kaler Nat Rev Neurol 2011; Roberts & Schilsky Hepat 2008; Schilsky Biochimie 2009; WD Pathogenesis Semin Liver Dis 2000

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Hemophilia Programs

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~$6.2B WW Market for Hemophilia B and A Products

Disease Patient Population

Deficiency of blood coagulation~28,000 Hem B patients WW

factor IX (FIX) or factor VIII (FVIII) ~140,000 Hem A patients WW

Joint hemorrhages, potential for

severe disabilities; brain micro- •~68% Hem B, 78% Hem A

bleeds moderate to severe disease

Frequent, invasive, non-curative

infusions of factor replacement •$100,000-$300,000 annual costs

per patient

Sources: Riley et al. Haemophilia 2011; National Hemophilia Foundation; World Federation of Hemophilia; www.fiercepharmamarketing.com/story/biogens-alprolix-nod-just-first-tremor-hemophilia-market-shake/2014-04-02; Stonebraker et al. Haemophilia (2011), 1–4; Baker et al. Haemophilia (2012); Stonebraker et al. Haemophilia (2010), 16, 20–32; National Hemophilia Foundation; www.ptcommunity.com/news/2014-12-29-000000/hemophilia-market-likely-undergo-major-changes; Johnson and Zhou. ASH Annual Meeting Educational Program. 2011; Ponder. Haemophilia 2008; Manco-Johnson et al. NEJM 2007

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DTX101 for Hemophilia B

DTX101 Phase I/II Study Ongoing

• Vector capsid Clade E AAVrh10 • Open-Label, safety, dose finding study

• Gene cassette wild type factor IX • Bayesian adaptive design

• Liver specific enhancer-promoter • Primary endpoints safety & FIX activity

• HEK293 process for hPOC • Up to 12 males with moderate/severe to

severe Hem B (?2% FIX levels)

• TPP* targeting stable FIX activity >10%

Dosing: 1.6e12-1e13 GC/kg, single

• Reduce dependency for on demand & peripheral venous infusion

prophylactic FIX therapies

Cohort 1 dosing complete

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DTX201: Global Collaboration with Bayer

Hemophilia A

A leading gene therapy Global leader in hemophilia

company drug development and

commercialization

• $20M upfront, up to $232M in milestones

• High single-digit to low double-digit royalties not exceeding the mid-teens

• R&D expense reimbursement

• DMTX responsible for preclinical activities and Phase I/II clinical trial, funded by Bayer

• Bayer to fund subsequent trials and commercial activities at clinical POC

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DTX201: Durable FVIII Expression in NHPs

DTX201 for Hem A IND-enabling Studies Ongoing

hFVIII Expression in Non Human Primates • Seven male cynomolgus macaques

administered IV with 1.2e13 GC/kg

Sustained FVIII expression in NHPs beyond

60 weeks

3 of 5 animals retain expression greater

than 16 weeks; 2 of 7 beyond 34 weeks

Sustained RNA expression

Reduced incidence of anti-FVIII inhibitors

Sources: DMTX-PENN data (ASGCT 2016, EHA 2016)

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Dimension: A Leader in Liver Directed Gene Therapy

Advancing Product Portfolio Broad 7 program pipeline delivering multiple near-term

milestones

Focus on Inherited Metabolic Diseases Addressing severe, debilitating unmet medical need

Building Gene Therapy Product Platform Investing in scalable, quality HeLa manufacturing

Highly Experienced Team Strong R&D and manufacturing capabilities

Well-Capitalized Expected cash runway through 1Q 2018

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Dimension Therapeutics, Inc.

Quality of Science. Quality of Life.

August 2016

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