Dec 04, 2016 Press Release for Alnylam
Alnylam Reports Positive Interim Clinical Results for Fitusiran from Ongoing Phase 2 Open Label Extension Study in Patients with Hemophilia A or B without Inhibitors
Dec 04, 2016
- Once-Monthly, Subcutaneous Fitusiran Achieves Median Annualized Bleeding Rate (ABR) of 1.0, with Median Observation Period of 5.7 Months -
- Fitusiran Generally Well Tolerated with No Thromboembolic Events -
- Alnylam On Track to Initiate Phase 3 Program in Early 2017 -
- Management to Discuss New Clinical Data in Webcast Conference Call
Today,
New clinical data showed that once-monthly subcutaneous administration of fitusiran achieved consistent lowering of AT and increases in thrombin generation, resulting in a median estimated annualized bleeding rate (ABR) of 1.0 in patients with hemophilia A or B without inhibitors. In addition, fitusiran was generally well tolerated, with no thromboembolic events or laboratory evidence of pathologic clot formation through the data cut-off date.
"We are encouraged by the longer-term tolerability data and clinical
activity of fitusiran, with a median ABR of 1.0 in patients with
hemophilia A or B with up to 14 months of continuous dosing. These data,
combined with positive results we reported yesterday in hemophilia
patients with inhibitors, continue to highlight what we believe to be
the significant potential of fitusiran as a once-monthly subcutaneous
investigational medicine for prevention of bleeding in people with
hemophilia and RBD," said
The Phase 2 OLE study results as of the data cut-off date of
The data reported from the Phase 2 OLE study also includes the first
reported elective surgical procedure in a fitusiran-treated patient.
Specifically, a patient with severe hemophilia A receiving 50 mg monthly
fitusiran underwent an elective septoplasty procedure. Prior to the
surgical procedure, the patient's AT levels were 13 percent relative to
baseline. As reported by the investigator via personal communication,
the cumulative periprocedural utilization of recombinant factor VIII was
approximately 20 percent of that typically used by the investigator for
this type of surgery in a severe hemophilia A patient. Based on the
Fitusiran was generally well tolerated with the longest period of exposure of up to 14 months of continuous treatment. All adverse events (AEs) were mild or moderate in severity, with the most common AEs consisting of mild injection site reactions (ISRs) in 4 out of 16 patients (25 percent). Asymptomatic and reversible alanine aminotransferase (ALT) increases greater than 3 times the upper limit of normal (ULN), without concurrent elevations in bilirubin greater than 2 times ULN, were observed in three patients in the OLE study, all of whom have medical history of hepatitis C infection (HCV). All breakthrough bleeding events were successfully managed with replacement factor. There were no drug-related serious adverse events (SAEs), no discontinuations due to AEs in the OLE study, and no thromboembolic events or laboratory evidence of pathologic clot formation through the data cut-off date.
To view the fitusiran clinical results described in this press release, please visit www.alnylam.com/capella.
Conference Call Information
Alnylam management will discuss these clinical data in a webcast
conference call today,
About Fitusiran
Fitusiran is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia A and B and rare bleeding disorders (RBD) currently in early stage clinical development. Fitusiran is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding in patients with hemophilia and RBD. AT, also known as "antithrombin III" and "SERPINC1" is a liver-expressed plasma protein and member of the "serpin" family of proteins that acts by inactivating thrombin and other coagulation factors. AT plays a key role in normal hemostasis by helping to limit the process of fibrin clot formation. However, in hemophilia, insufficient thrombin generation results in impaired fibrin clot formation. Lowering AT in the hemophilia setting may promote the generation of sufficient levels of thrombin needed to form an effective fibrin clot and prevent bleeding. This rationale is supported by human genetic data suggesting that co-inheritance of thrombophilic mutations, including AT deficiency, may ameliorate bleeding in hemophilia. Lowering of AT is a unique and innovative strategy for restoring hemostasis in people with hemophilia. Fitusiran utilizes Alnylam's ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.
In
About RNAi
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
About
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is leading the
translation of RNAi as a new class of innovative medicines. Alnylam's
pipeline of investigational RNAi therapeutics is focused in 3 Strategic
Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of
RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic
Disease, with a pipeline of RNAi therapeutics toward genetically
validated, liver-expressed disease targets for unmet needs in
cardiovascular and metabolic diseases; and Hepatic Infectious Disease,
with a pipeline of RNAi therapeutics that address the major global
health challenges of hepatic infectious diseases. In early 2015, Alnylam
launched its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics as a whole new class of
innovative medicines. Specifically, by the end of 2020, Alnylam expects
to achieve a company profile with 3 marketed products, 10 RNAi
therapeutic clinical programs - including 4 in late stages of
development - across its 3 STArs. The company's demonstrated commitment
to RNAi therapeutics has enabled it to form major alliances with leading
companies including Ionis, Novartis, Roche, Takeda,
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 for RNAi therapeutics,
including fitusiran, its expectations regarding the timing of clinical
studies and the presentation of clinical data, including for its studies
of fitusiran, its expectations regarding its STAr pipeline growth
strategy, its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics, and its plans regarding the
pursuit of pre-clinical programs 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 our 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 establishing a commercial and ex-
The scientific information referenced in this news release relating to
fitusiran is preliminary and investigative. Fitusiran has not been
approved by the
View source version on businesswire.com: http://www.businesswire.com/news/home/20161204005038/en/
(Investors and Media)
or
(Investors)
Source:
News Provided by Acquire Media
For Media Inquiries, please contact:
Christine Akinc
Chief Corporate Communications Officer media@alnylam.com 617-682-4340
For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
MEDIA KIT
Essential assets and documents related to Alnylam
