BURLINGTON, Mass.--(BUSINESS WIRE)--Dyax Corp. (NASDAQ:DYAX) today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for the investigation of DX-2930 for hereditary angioedema (HAE). Dyax is developing DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKal), as a subcutaneous injection for prevention of HAE attacks.
Breakthrough Therapy designation is intended to expedite the development and review of potential new medicines with early signal of clinical benefit in serious or life-threatening conditions and helps ensure patients have access to them as soon as possible. Breakthrough Therapy designation is considered when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy. The benefits of Breakthrough Therapy designation include organizational commitment involving the FDA’s senior managers and with more intensive guidance. Breakthrough Therapy designation does not change the standards for approval.
The designation is supported by the interim results of Dyax’s Phase 1b clinical trial of DX-2930 in HAE patients. The Phase 1b study met all objectives assessing safety, tolerability and pharmacokinetics of multiple subcutaneous administrations of DX-2930. Additionally, in a pre-specified proof-of-concept efficacy analysis, DX-2930 demonstrated statistically significant reductions in attack rate compared to placebo.
“Receipt of Breakthrough Therapy designation is a key milestone for the DX-2930 development program," said Burt Adelman, M.D., Executive Vice President of Research and Development and Chief Medical Officer at Dyax. “We look forward to taking full advantage of the opportunities that Breakthrough Therapy designation allows in order to maximize the possibility of a rapid path to approval.”
About DX-2930
DX-2930 is a novel, fully human monoclonal
antibody inhibitor of pKal which is currently being developed as a
subcutaneous injection for the prevention of HAE attacks. Uncontrolled
pKal activity leads to excessive generation of bradykinin, a vasodilator
thought to be responsible for the localized swelling, inflammation and
pain characteristically associated with HAE.
About HAE
HAE is a rare acute inflammatory condition
characterized by episodes of severe, often painful swelling affecting
the extremities, gastrointestinal tract, genitalia, and larynx. HAE is
caused by low or dysfunctional levels of C1 esterase inhibitor (C1-INH),
a naturally occurring molecule that inhibits plasma kallikrein, a key
mediator of inflammation, and other serine proteases in the blood. HAE
is estimated to affect up to 1 in 50,000 individuals. Learn more at www.HAEHope.com.
About Dyax
Dyax is a biopharmaceutical company focused on
the development and commercialization of novel biotherapeutics for unmet
medical needs. The Company is developing DX-2930,
a fully human monoclonal antibody, for the prevention of HAE attacks. In
March 2015, the Company reported positive safety, pharmacokinetic,
biomarker and proof-of-concept efficacy results from its Phase
1b clinical trial of DX-2930 in HAE patients. Additionally, Dyax
markets KALBITOR®
(ecallantide) for the treatment of acute attacks of HAE in patients
12 years of age and older.
Both DX-2930 and KALBITOR were identified using Dyax's proprietary phage display technology. Dyax has broadly licensed this technology under its Licensing and Funded Research Portfolio (LFRP). The current portfolio includes one FDA approved product, Eli Lilly and Company’s CYRAMZA® (ramucirumab), for which Dyax receives royalties, and multiple product candidates in various stages of clinical development for which the Company is eligible to receive future milestones and royalties.
For additional information about Dyax, please visit www.dyax.com.
For additional information about KALBITOR, including full prescribing information, please visit www.KALBITOR.com.
Disclaimer
The press release contains forward-looking
statements, including statements regarding the prospects for therapeutic
benefits and treatment advantages of an investigational product,
DX-2930, being developed for HAE. Statements that are not historical
facts are based on Dyax’s current expectations, beliefs, assumptions,
estimates, forecasts and projections about the industry and markets in
which Dyax competes. The statements contained in this press release are
not guarantees of future performance and involve certain risks,
uncertainties and assumptions that are difficult to predict. Therefore,
actual outcomes and results may differ materially from what is expressed
in such forward-looking statements. There are many factors that could
cause actual results to differ materially from those in these
forward-looking statements. These factors include the following: the
results from our Phase 1b study may not be predictive of the results or
success of future clinical trials that will be required to permit
application for regulatory approval of DX-2930; even if DX-2930
progresses through clinical trials and gains regulatory approval, it may
not gain market acceptance; others may develop technologies or products
superior to DX-2930 or that reach the market before DX-2930; Dyax is
dependent on the expertise, effort, priorities and contractual
obligations of third parties in the manufacture, quality control,
storage and clinical development of DX-2930; the costs of prosecuting,
maintaining, defending and enforcing our patents and other intellectual
property rights; the overall condition of the financial markets; and a
variety of other risks common to our industry; changing requirements and
costs associated with Dyax's planned research and development
activities; competition from new and existing treatments for HAE; the
uncertainty of patent and intellectual property protection; and other
risk factors described or referred to in Item 1A, “Risk Factors” in
Dyax’s most recent Annual Report on Form 10-K and other periodic reports
filed with the Securities and Exchange Commission. Dyax cautions
investors not to place undue reliance on the forward-looking statements
contained in this release. These statements speak only as of the date of
this release, and Dyax undertakes no obligations to update or revise
these statements, except as may be required by law.
Dyax, the Dyax logo and KALBITOR are registered trademarks of Dyax Corp.
CYRAMZA
is a trademark owned by or licensed to Eli Lilly and Company, its
subsidiaries, or affiliates.
