– Patients living with AHP suffer from chronic
debilitating symptoms such as severe abdominal pain and often seek
care from gastroenterologists –
– Alnylam to leverage Ironwood’s leading
capabilities in GI to help raise AHP awareness and bring givosiran
(if approved) to gastroenterologists and other healthcare
practitioners in the U.S. –
Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), the leading RNAi
therapeutics company and Ironwood Pharmaceuticals, Inc. (NASDAQ:
IRWD), a GI healthcare company, announced today a U.S. GI disease
education and promotional agreement for Alnylam’s givosiran, an
investigational RNAi therapeutic targeting aminolevulinic acid
synthase 1 (ALAS1) for the potential treatment of AHP. Givosiran
has received Priority Review designation and Breakthrough Therapy
Designation from the U.S. FDA, as well as Orphan Drug Designation
in the U.S. The Prescription Drug User Fee Act (PDUFA) date for
givosiran is set for February 4, 2020.
Under the terms of the agreement, Ironwood will provide AHP
disease education to gastroenterologists and other healthcare
practitioners that Ironwood currently calls on for LINZESS®
(linaclotide). If approved by the U.S. FDA, Ironwood clinical sales
specialists will then begin givosiran promotional efforts,
augmenting Alnylam’s broader commercialization activities.
“AHP is a rare disease with chronic, debilitating, and sometimes
life-threatening attacks. Nearly all patients living with AHP
consult at least one, if not several, gastroenterologists due to
the recurring abdominal pain associated with their disease and are
often misdiagnosed due to minimal disease awareness and a limited
number of treatment options,” said Barry Greene, President of
Alnylam. “As Alnylam prepares for the potential launch and
commercialization of givosiran around the world, leveraging
Ironwood’s U.S. GI commercial expertise and depth of relationships
within the GI community represents a significant opportunity to
expand medical education and diagnosis for patients with AHP.”
“We are excited about the opportunity to partner with Alnylam to
increase awareness among healthcare providers about the devastating
effects of AHP and, if approved, to then discuss givosiran as a
potential new treatment option for patients suffering from this
severe, potentially life-threatening disease,” said Tom McCourt,
President of Ironwood.
The non-exclusive agreement covers an approximately three-year
term. Ironwood will collaborate with Alnylam on AHP disease
education designed to help ensure AHP patients are accurately
diagnosed and to support access to treatment with givosiran once
prescribed (assuming approval). Ironwood will receive fixed
payments and royalties in the mid-teens percent on net sales
generated from prescriptions or referrals from certain physicians
related to Ironwood’s promotional efforts. Alnylam will maintain
responsibility for all other aspects of givosiran, and retains all
global development and commercialization rights.
About Acute Hepatic Porphyria
Acute hepatic porphyria (AHP) refers to a family of rare,
genetic diseases characterized by potentially life-threatening
attacks and for some patients chronic debilitating symptoms that
negatively impact daily functioning and quality of life. AHP is
comprised of four subtypes, each resulting from a genetic defect
leading to deficiency in one of the enzymes of the heme
biosynthesis pathway in the liver: acute intermittent porphyria
(AIP), hereditary coproporphyria (HCP), variegate porphyria (VP),
and ALAD-deficiency porphyria (ADP). These defects cause the
accumulation of neurotoxic heme intermediates aminolevulinic acid
(ALA) and porphobilinogen (PBG), with ALA believed to be the
primary neurotoxic intermediate responsible for causing both
attacks and ongoing symptoms between attacks. Common symptoms of
AHP include severe, diffuse abdominal pain, weakness, nausea, and
fatigue. The nonspecific nature of AHP signs and symptoms can often
lead to misdiagnoses of other more common conditions such as
irritable bowel syndrome, appendicitis, fibromyalgia, and
endometriosis, and consequently, patients afflicted by AHP often
remain without a proper diagnosis for up to 15 years. In addition,
long-term complications of AHP and its treatment can include
chronic neuropathic pain, hypertension, chronic kidney disease and
liver disease, including iron overload, fibrosis, cirrhosis and
hepatocellular carcinoma. Currently, there are no treatments
approved to prevent debilitating attacks or to treat the chronic
manifestations of the disease.
About Givosiran
Givosiran is an investigational, subcutaneously administered
RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1)
in development for the treatment of acute hepatic porphyria (AHP).
Monthly administration of givosiran has the potential to
significantly lower induced liver ALAS1 levels in a sustained
manner and thereby decrease neurotoxic heme intermediates,
aminolevulinic acid (ALA) and porphobilinogen (PBG), towards normal
levels. By reducing accumulation of these intermediates, givosiran
has the potential to prevent or reduce the occurrence of severe and
life-threatening attacks, control chronic symptoms, and decrease
the burden of the disease. Givosiran utilizes Alnylam’s Enhanced
Stabilization Chemistry ESC-GalNAc conjugate technology, which
enables subcutaneous dosing with increased potency and durability
and a wide therapeutic index. The safety and efficacy of givosiran
were evaluated in the ENVISION Phase 3 trial with positive results;
these results have not been evaluated by the FDA, the EMA or any
other health authority.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading 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 genetic, cardio-metabolic, hepatic infectious, and central
nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning
science, RNAi therapeutics represent a powerful, clinically
validated approach for the treatment of a wide range of severe and
debilitating diseases. Founded in 2002, Alnylam is delivering on a
bold vision to turn scientific possibility into reality, with a
robust discovery platform. Alnylam’s first commercial RNAi
therapeutic is ONPATTRO® (patisiran), approved in the U.S., EU,
Canada, and Japan. Alnylam has a deep pipeline of investigational
medicines, including five product candidates that are in late-stage
development. Looking forward, Alnylam will continue to execute on
its "Alnylam 2020" strategy of building a multi-product,
commercial-stage biopharmaceutical company with a sustainable
pipeline of RNAi-based medicines to address the needs of patients
who have limited or inadequate treatment options. Alnylam employs
over 1,200 people worldwide and is headquartered in Cambridge, MA.
For more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at @Alnylam or
on LinkedIn.
About Ironwood Pharmaceuticals
Ironwood Pharmaceuticals (Nasdaq: IRWD) is a GI-focused
healthcare company dedicated to creating medicines that make a
difference for patients living with GI diseases. We discovered,
developed and are commercializing linaclotide, the U.S. branded
prescription market leader for adults with irritable bowel syndrome
with constipation (IBS-C) or chronic idiopathic constipation
(CIC).
We are also advancing two late-stage, first-in-category GI
product candidates: IW-3718 is a gastric retentive formulation of a
bile acid sequestrant being developed for the potential treatment
of persistent gastroesophageal reflux disease, and MD-7246 is a
delayed-release formulation of linaclotide that is being evaluated
as an oral, intestinal, non-opioid, pain-relieving agent for
patients suffering from abdominal pain associated with IBS with
diarrhea.
Ironwood was founded in 1998 and is headquartered in Cambridge,
Mass. For more information, please visit our website at
www.ironwoodpharma.com or www.twitter.com/ironwoodpharma;
information that may be important to investors will be routinely
posted in both these locations.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
Alnylam's views with respect to the potential benefits of
givosiran, the expected timing of the completion of regulatory
reviews of the NDA submitted for givosiran, the anticipated
benefits of the agreement entered into with Ironwood and the
potential payments to Ironwood under such agreement, and
expectations regarding its “Alnylam 2020” guidance for the
advancement and commercialization of RNAi therapeutics, constitute
forward-looking statements for the purposes of the safe harbor
provisions under The Private Securities Litigation Reform Act of
1995. Actual results and future plans may differ materially from
those indicated by these forward-looking statements as a result of
various important risks, uncertainties and other factors,
including, without limitation, Alnylam's ability to discover and
develop novel drug candidates and delivery approaches, successfully
demonstrate the efficacy and safety of its product candidates, the
pre-clinical and clinical results for its product candidates, which
may not be replicated or continue to occur in other subjects or in
additional studies or otherwise support further development of
product candidates for a specified indication or at all, actions or
advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials
or result in the need for additional pre-clinical and/or clinical
testing, delays, interruptions or failures in the manufacture and
supply of its product candidates, obtaining, maintaining and
protecting intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in continuing to establish a commercial and
ex-United States infrastructure, successfully launching, marketing
and selling its approved products globally, Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future,
competition from others using technology similar to Alnylam's and
others developing products for similar uses, Alnylam's ability to
manage its growth and operating expenses, obtain additional funding
to support its business activities, and establish and maintain
strategic business alliances and new business initiatives,
Alnylam's dependence on third parties for development, manufacture
and distribution of products, the outcome of litigation, the risk
of government investigations, and unexpected expenditures, as well
as those risks more fully discussed in the “Risk Factors” filed
with Alnylam's most recent Annual Quarterly Report on Form 10-QK
filed with the Securities and Exchange Commission (SEC) and in
other filings that Alnylam makes with the SEC. In addition, any
forward-looking statements represent Alnylam's views only as of
today and should not be relied upon as representing its views as of
any subsequent date. Alnylam explicitly disclaims any obligation,
except to the extent required by law, to update any forward-looking
statements.
Ironwood Forward Looking Statements
This press release contains forward-looking statements.
Investors are cautioned not to place undue reliance on these
forward-looking statements including statements about the
anticipated benefits of the disease education and promotional
agreement entered into with Alnylam, the potential payments to
Ironwood under such agreement, the effect of Ironwood’s disease
education activities and promotional efforts (if approved) on
Alnylam’s givosiran, and the development, launch, commercial
availability and commercial potential of linaclotide and Ironwood’s
other product candidates. Each forward‐looking statement is subject
to risks and uncertainties that could cause actual results to
differ materially from those expressed or implied in such
statement. Applicable risks and uncertainties include, but are not
limited to, the risk that Ironwood does not realize the benefits of
the disease education and promotional agreement entered into with
Alnylam, the effectiveness of Ironwood’s development and
commercialization efforts, the risk that Ironwood’s clinical
programs and studies may not progress or develop as anticipated,
including that studies are delayed or discontinued for any reason
such as safety, tolerability, enrollment, manufacturing, economic
or other reasons, the risks that findings from Ironwood’s completed
studies may not be replicated in later studies, the efficacy,
safety and tolerability of linaclotide and Ironwood’s other product
candidates and the risks listed under the heading “Risk Factors”
and elsewhere in Ironwood’s Quarterly Report on Form 10-Q for the
quarter ended June 30, 2019, and in Ironwood’s subsequent SEC
filings. These forward-looking statements speak only as of the date
of this press release, and Ironwood undertakes no obligation to
update these forward-looking statements.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190813005261/en/
Christine Regan Lindenboom Alnylam Investors and Media
617-682-4340 Josh Brodsky Alnylam Investor Relations 617-551-8276
Meredith Kaya Ironwood Investors and Media 617-374-5082
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