Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA), a leading
developer of investigational ribonucleic acid interference (RNAi)
therapeutics, today announced that late-breaking data from the
PHYOX Phase 1 trial of DCR-PHXC for the treatment of primary
hyperoxaluria (PH) will be presented at the American Society of
Nephrology (ASN) Annual Kidney Week 2018, being held October 23-28
in San Diego. The poster presentation will include clinical data
from the ongoing PHYOX Phase 1 study in patients with primary
hyperoxaluria type 1 and type 2 (PH1 and PH2). PH is a family of
severe, rare, inherited disorders of the liver that often result in
kidney failure.
Details for the poster presentation are as follows:
- Session Title: Late-Breaking
Clinical Trials Posters [LB-PO]
- Poster Title: PHYOX: A Safety
and Tolerability Study of DCR-PHXC in Primary Hyperoxaluria Types 1
and 2
- Poster Number: TH-PO1167
- Date/Time: Thursday, October 25,
2018, 10:00 a.m. - 12:00 p.m. PT
- Presenter: Dr. Bernd Hoppe,
M.D., PHYOX investigator and head of the Division of Pediatric
Nephrology in the Department of Pediatrics at the University of
Bonn, Germany
The Company recently reported initial proof-of-concept data from
the PHYOX Phase 1 trial demonstrating significant and sustained
reduction in urinary oxalate levels following single-dose
administration in adults with PH1 and PH2.
For information on ASN Kidney Week 2018, visit
https://www.asn-online.org/education/kidneyweek/.
About DCR-PHXC
DCR-PHXC is an investigational drug in development for the
treatment of all forms of primary hyperoxaluria (PH), and the most
advanced product candidate utilizing Dicerna's GalXCTM technology.
GalXC is a proprietary platform invented by Dicerna scientists to
discover and develop next-generation RNAi-based therapies designed
to silence disease-driving genes in the liver. In animal models of
PH, DCR-PHXC selectively silences lactase dehydrogenase (LDHA) in
the liver, blocking the excess production of oxalate, a hallmark of
the disease. In preclinical studies of DCR-PHXC, the compound was
well tolerated with no adverse effects in the liver. Studies have
shown that people who are completely deficient in LDHA show no
liver dysfunction and can lead normal lives. LDHA deficiency in the
liver might be beneficial for patients with PH, as the LDHA enzyme
is implicated in the abnormal production of oxalate in PH, which in
turn is responsible for the severe damage to kidneys and other
organ systems in patients with PH.
About Primary Hyperoxaluria (PH)
Primary hyperoxaluria (PH) is a family of severe, rare, genetic
liver disorders characterized by overproduction of oxalate, a
natural chemical in the body that is normally eliminated as waste
through the kidneys. In patients with PH, the kidneys are unable to
eliminate the large amount of oxalate that is produced, and the
accumulation of oxalate can result in severe damage to the kidneys
and other organs. Currently, there are no approved therapies for
the treatment of PH.
There are three known types of PH, each of which results from a
mutation in a specific gene, as well as PH for which the molecular
basis remains unknown, often referred to as idiopathic PH (IPH) or
"no mutation detected" (NMD) PH. The known PH mutations cause a
decrease in the activity of a specific enzyme in the liver,
triggering an increase in oxalate production. In each case the
decreased enzyme activity changes the balance of intermediary
metabolites, resulting in overproduction of oxalate. The three
genetically known types of PH are: 1,2
- PH1, which is caused by a mutation in
the AGXT gene, causing a deficiency of the enzyme
alanine:glyoxylate-aminotransferase (AGT)
- PH2, which is caused by a mutation in
the GRHPR gene, causing a deficiency of the enzyme
glyoxylate/hydroxypyruvate reductase (GR/HPR)
- PH3, which is caused by a mutation in
the HOGA1 gene, causing a deficiency of the enzyme
4-hydroxy-2-oxoglutarate aldolase (HOGA)
Patients with severe PH often undergo both liver and kidney
transplants, which are major surgical procedures, and subsequently
must take immunosuppressant drugs for the rest of their lives.
Patients with decreased renal function may also experience
oxalosis, which involves a build-up of oxalate in other organs such
as the bone, skin, heart, and retina, possibly causing other
concomitant, debilitating complications.
PH occurs in an estimated 1 in 120,000 live births around the
world.3 The estimated genetic prevalence of PH1 is 1 in
151,887 births, which implies more than 5,000 patients in the
United States and European Union have the disease.3 The
estimated genetic prevalence of PH2 is 1 in 310,055 and that of PH3
is 1 in 135,866.3 The median age at the first appearance of PH1
symptoms is 5.8 years.4 The median age at diagnosis of PH1 is
between 4.2 and 11.5 years, depending on whether nephrocalcinosis
(calcification in the renal parenchyma, the functional part of the
kidney) is present.5 Fifty percent of patients with PH1 reach
end-stage renal disease (ESRD) by their mid-30s.2
About Dicerna Pharmaceuticals, Inc.
Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company
focused on the discovery and development of innovative,
subcutaneously delivered RNAi-based therapeutics for the treatment
of diseases involving the liver, including rare diseases, viral
infectious diseases, chronic liver diseases, and cardiovascular
diseases. Dicerna is leveraging its proprietary GalXC™ RNAi
technology platform to build a broad pipeline in these core
therapeutic areas, focusing on target genes where connections
between target gene and diseases are well understood and
documented. Dicerna intends to discover, develop and commercialize
novel therapeutics either on its own or in collaboration with
pharmaceutical partners. For more information, please visit
www.dicerna.com.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements. Such
forward-looking statements are subject to risks and uncertainties
that could cause actual results to differ materially from those
expressed or implied in such statements. Examples of
forward-looking statements include, among others, statements we
make regarding: (i) the therapeutic and commercial potential of the
GalXC™ platform, including DCR-PHXC; (ii) research and development
plans related to GalXC,™ including DCR-PHXC; and (iii) the
potential of our technology and drug candidates in our research and
development pipeline. The process by which an early stage platform
such as GalXC (including DCR-PHXC, our lead product candidate)
could potentially lead to an approved product is long and subject
to highly significant risks. In general, most earlier stage drug
candidates do not ultimately become approved drugs. Applicable
risks and uncertainties include those relating to Dicerna's
clinical and preclinical research and others identified under the
heading "Risk Factors" included in the Company's filings with the
Securities and Exchanges Commission (SEC). These risks and
uncertainties include, among others, the cost, timing and results
of preclinical studies and clinical trials and other development
activities; the unpredictability of the duration and results of
regulatory review of New Drug Applications and Investigational
NDAs; market acceptance for approved products and innovative
therapeutic treatments; competition; the possible impairment of,
inability to obtain and costs of obtaining intellectual property
rights; and possible safety or efficacy concerns, general business,
financial and accounting risks and litigation. The forward-looking
statements contained in this press release reflect Dicerna's
current views with respect to future events, and Dicerna does not
undertake and specifically disclaims any obligation to update any
forward-looking statements.
References
1.
Oxalosis & Hyperoxaluria Foundation.
Overview of hyperoxaluria. 2017. Available at:
https://ohf.org/overview/. Accessed July 6, 2017.
2.
Rare Kidney Stone Consortium. Primary
hyperoxaluria. 2010. Available at:
http://www.rarekidneystones.org/hyperoxaluria/physicians.html.
Accessed July 6, 2017.
3. Hopp, K, Cogal, A, Bergstralh, E, et al. Phenotype-genotype
correlations and estimated carrier frequencies of primary
hyperoxaluria. Journal of the American Society of Nephrology 2015;
26(10):2559-2570. 4. van der Hoeven SM, van Woerden CS, Groothoff
JW. Primary hyperoxaluria type 1, a too often missed diagnosis and
potentially treatable cause of end-stage renal disease in adults:
results of the Dutch cohort. Nephrology, Dialysis, Transplantation
2012; 27(10):3855-3862. 5. Tang X, Bergstrath EJ, Mehta RA, Vrtiska
TJ, Milliner DS, Lieske JC. Nephrocalcinosis is a risk factor for
kidney failure in primary hyperoxaluria. Kidney International 2015;
87:623-631.
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version on businesswire.com: https://www.businesswire.com/news/home/20181005005423/en/
Investors:Rx Communications GroupPaula Schwartz,
917-322-2216pschwartz@rxir.comorMedia:SmithSolveAlex Van
Rees, 973-442-1555 ext. 111alex.vanrees@smithsolve.com
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