For Immediate Release

Media Contact: Caroline Grossman

781.771.5579
caroline.grossman@gmail.com

Cequent Files its First IND with FDA: CEQ508, a tkRNAi Drug Candidate in Oncology
First Orally Delivered RNAi Investigational Drug to be Tested in Humans
CAMBRIDGE, Massachusetts (November 12, 2009) – Cequent Pharmaceuticals, a pioneer in the development of
novel products to deliver RNAi-based treatments to prevent and treat human disease, has announced that it has
filed its first IND (investigational new drug) application with the U.S. Food and Drug Administration (FDA).
CEQ508, an orally administered tkRNAi drug candidate, targets beta-catenin, a key oncogene implicated in the
formation of colonic polyps and in the progression of polyps to colorectal cancer. Cequent expects to begin the
Phase I clinical trial in the FAP (familial adenomatous polyposis) patient population during the first quarter of 2010
at the Fred Hutchinson Cancer Research Center, in Seattle, Washington, part of the Fred Hutchinson/University
of Washington Cancer Consortium.

“A first IND filing is obviously a momentous event for a young company like Cequent, and this is also a critical
milestone in the development of RNAi therapeutics in general, as our proposed Phase I trial will be the first test of
an orally administered RNA interference drug in humans,” said Cequent Chief Executive Officer Peter Parker. “In
2006, when RNAi was the subject of the Nobel Prize in Medicine, it was hailed as ‘a revolution in biology’ –
RNAi’s discovery created the opportunity to address targets that were previously difficult to treat by effectively
deactivating the specific gene or genes implicated in the progression of a disease by “interfering” with messenger
RNA. However, success in developing RNAi therapies has been elusive to date because of delivery issues – how
do you transport and deposit the molecules triggering RNAi into the target cells and diseased tissues. We believe
that our orally administered tkRNAi technology has shown great potential in solving that significant problem.”

This Phase I clinical trial, as proposed to the FDA, would be conducted to determine safety and tolerability of
CEQ508 at escalating doses in a total of 18 adult FAP patients. A key readout and secondary objective of the
proposed trial includes analysis of biomarker (beta-catenin) expression changes in the gastrointestinal tract of
patients determined from biopsy samples taken prior to taking the drug, and following a daily, 28-day dosing
regimen. The principal investigator (PI) of this clinical trial will be Gideon Steinbach, M.D., Ph.D., associate
professor of medicine at the University of Washington and the PI of a number of earlier FAP studies. The Cancer
Consortium maintains a registry of FAP patients and is also one of 40 National Cancer Institute-designated
comprehensive cancer centers nationwide.

In preclinical testing with non-human primates, tkRNAi therapeutic candidates have demonstrated potent silencing
of the beta-catenin, a protein known to accumulate and lead to the proliferation of polyps in affected patients.
During preclinical testing, CEQ508 demonstrated an encouraging safety profile when administered as a daily oral
therapeutic.

FAP is a rare inherited gastrointestinal disease that causes hundreds to thousands of precancerous polyps to
form in the colon. Approximately 35,000 people in the U.S. carry the genetic mutation inherent to the disease.
Today, without prophylactic removal of the colon, people with FAP almost inevitably develop cancer, and there is
no generally accepted pharmacological treatment available. FAP has been designated as an orphan disease
under the U.S. Orphan Drug Act, which provides various incentives for sponsors to encourage development of
products for rare diseases. Phase I studies of novel therapeutics for such rare, underserved diseases are often
allowed to enroll patients as opposed to healthy volunteers, potentially accelerating the timeline to develop
approved products.

Commenting on Cequent’s progression as a company, Mr. Parker said, “We also continue to make important
progress with our tkRNAi inflammatory bowel disease (IBD) program, for which this CEQ508 trial serves as a
regulatory and safety pathbreaker. Cequent is moving ahead with preclinical development of tkRNAi products
targeting immunological genes implicated in the pathology of IBD.”

Mr. Parker continued, “We have now entered a new phase in the company’s lifecycle; we have transitioned away
from research to focus on our clinical programs, much of which we will pursue with contract resources. As such,
we have promoted Alison Silva to the new position of vice president of drug development, overseeing all clinical
development and regulatory affairs for the company. Co-inventor of the tkRNAi technology, Johannes Fruehauf,
M.D., will support the upcoming clinical trial through a consultancy arrangement as vice president of medical
affairs.”

About Cequent Pharmaceuticals, Inc. (www.cequentpharma.com)

An early-stage biopharmaceutical company, Cequent is pioneering the development of novel therapeutics to
prevent and treat a wide range of human disorders – from inflammatory disease to cancer – based on the
company’s proprietary technology, TransKingdom RNA interference (tkRNAi). Cequent’s first products, now
entering clinical development, are orally administered drug candidates targeting colon-cancer prevention and
inflammatory bowel disease. The company designed its powerful tkRNAi technology as a therapeutic to
deactivate specific disease-causing genes safely and effectively, using non-pathogenic bacteria as an engine to
produce and deliver RNAi directly into cells. It is based on ground-breaking scientific research originating at the
Institut Pasteur (Paris, France) and at the Beth Israel Deaconess Medical Center/Harvard Medical School. A
privately held company based in Cambridge, Massachusetts, Cequent was established in 2006.

Recent financing developments

In October 2009, Cequent announced that it had initiated a Series B round of financing, seeking to raise $15
million to take the company through Phase II clinical trials with its lead drug candidate. This week, it closed on
$3.35 million in first-tranche equity financing. Participating investors included Ampersand Ventures, Gold Hill
Capital, Novartis Option Fund, Pappas Ventures, and Yasuda Enterprise Development, all of whom participated in
the company’s Series A funding in 2007. A second tranche from this group is expected to yield $3.2 million in
2010, subject to successful advancement of Cequent’s tkRNAi technology into human studies.

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