BREAKING NEWS! There may soon be another treatment for PI patients!
The company ADMA Biologics, Inc. has developed a new treatment option, RI-002, for patients who are immune-compromised. It’s currently in the regulatory review/approval stage; they just recently submitted their Biologics License Application to the FDA.
This new drug could hold the potential to provide another treatment option for immune-compromised patients and improve their lives. Keep up to date with Patient Worthy for more information as this drug continues through the process of gaining FDA approval.
Here is the official press release:
company that develops, manufactures and intends to commercialize specialty plasma‐based biologics
for the primary immunodeficiency (PI) population and the treatment and prevention of certain
infectious diseases, announced that it has submitted its Biologics License Application (BLA) to the United States Food and Drug Administration (FDA), seeking marketing authorization for RI‐002.RI‐002 demonstrated positive results in a Phase III study in patients with PI, meeting its primary
endpoint of no serious bacterial infections (SBI). These results, included in the submission, more than
meet the requirement specified by the FDA guidance of ≤ 1 SBI per patient‐year.“The BLA submission for RI‐002 is a significant milestone for our growing company,” stated Adam
Grossman, President and CEO of ADMA Biologics. “This submission, combined with our recent
announcement of obtaining patent protection for RI‐002, places ADMA in a rare position as a company
poised to transition from development to commercial stage. We believe that RI‐002, if approved by the
FDA, has great potential to provide meaningful clinical improvement for patients living with PI, as well as
offers clinicians a much needed option for their immune compromised patients. Under PDUFA V, the
BLA filing fee is $2.3 million. The FDA reviewed ADMA’s small business waiver application and it was
approved.”
PI is a class of inherited genetic disorders that causes an individual to have a deficient or absent immune
system due to either a lack of necessary antibodies or a failure of these antibodies to function properly,
affecting approximately 250,000 people in the United States.
The FDA has a 60‐day review period to determine whether ADMA’s BLA submission for RI‐002 is
complete and acceptable for filing.
About RI‐002
ADMA’s lead product candidate, RI‐002, is a specialty plasma‐derived, polyclonal, intravenous immune
globulin (IGIV) derived from human plasma containing naturally occurring polyclonal antibodies (e.g.,
Streptococcus pneumoniae, H. influenza type B, cytomegalovirus (CMV), measles, tetanus, etc.) as well
as standardized, high levels of antibodies to respiratory syncytial virus (RSV). On June 30, 2015, ADMA
announced that it had received a notice of allowance for a U.S. patent pertaining to RI‐002 entitled
“Compositions and Methods for the Treatment of Immunodeficiency.” ADMA is pursuing an indication
for the use of this specialty IGIV product for treatment of patients diagnosed with primary immune
deficiency disease (PIDD). Polyclonal antibodies are the primary active component of
IGIV/immuneglobulin products. Polyclonal antibodies are proteins that are used by the body’s immune
system to neutralize microbes, such as bacteria and viruses. Data review indicates that the polyclonal
antibodies present in RI‐002 support its ability to prevent infections in immune‐compromised patients.
ADMA’s analysis demonstrated that the Phase III trial met the primary endpoint with no serious
bacterial infections (SBI) reported. These results more than meet the requirement specified by the FDA
guidance of ≤ 1 SBI per patient‐year. A BLA was submitted to the FDA on July 31, 2015.
About ADMA Biologics, Inc.
ADMA is a late‐stage biopharmaceutical company that develops, manufactures and intends to market
specialty plasma‐based biologics for the treatment and prevention of PI and certain infectious diseases.
ADMA’s mission is to develop and commercialize plasma‐derived, human immune globulins targeted to
niche patient populations for the treatment and prevention of certain infectious diseases. The target
patient populations include immune‐compromised individuals who suffer from an underlying immune
deficiency disease, or who may be immune‐compromised for medical reasons. ADMA’s lead product
candidate, RI‐002, for which a notice of allowance for a U.S. patent was granted pertaining to RI‐002
entitled “Compositions and Methods for the Treatment of Immunodeficiency,” has completed a Phase III
clinical trial in patients with primary immune deficiency disease (PIDD) and has met the primary
endpoint. A BLA for RI‐002 was submitted to the FDA on July 31, 2015. For more information, please
visit the company’s website at www.admabiologics.com.
About Primary Immune Deficiency Disease (PI)
PIDD is a class of inherited genetic disorders that causes an individual to have a deficient or absent
immune system due to either a lack of necessary antibodies or a failure of these antibodies to function
properly. PIDD patients are more vulnerable to infections and more likely to suffer complications from
these infections. According to the World Health Organization, there are over 150 different presentations
of PIDD. As patients suffering from PIDD lack a properly functioning immune system, they typically
receive monthly, outpatient infusions of IGIV therapy. Without this exogenous antibody immune
support, these patients would be susceptible to a wide variety of infectious diseases. PIDD has an
estimated prevalence of 1:1,200 in the United States, or approximately 250,000 people.
Forward‐Looking Statements
This press release contains “forward‐looking statements” pursuant to the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995. Forward‐looking statements include, without limitation,
any statement that may predict, forecast, indicate, or imply future results, performance or achievements,
and may contain the words “estimate,” “project,” “intend,” “forecast,” “target,” “anticipate,” “plan,”
“planning,” “expect,” “believe,” “will,” “will likely,” “is likely”, “should,” “could,” “would,” “may” or, in
each case, their negative, or words or expressions of similar meaning. These forward‐looking statements include, but are not limited to, statements concerning interpretations of final data, possible
characteristics of RI‐002, acceptability of RI‐002 for any purpose by physicians patients or payers,
concurrence by FDA with our conclusions and the satisfaction by us of its guidance, the acceptability by
the FDA of the BLA, the likelihood and timing of FDA action with respect to any further filings by the
Company, results of the clinical development, continuing demonstrations of safety, comparability of
results of RI‐002 to other comparably run IVIG trials, improvements in clinical outcomes, potential of RI‐
002 to provide meaningful clinical improvement for patients living with PI, as well as to offer clinicians
with an option for their immune compromised patients, market data and incidence of infection,
regulatory processes, potential clinical trial initiations, potential investigational new product
applications, biologics license applications, expansion plans, the achievement of clinical and regulatory
milestones, commercialization efforts of the Company’s product candidate(s) and trends relating to
demand for source plasma. Forward‐looking statements are subject to many risks and uncertainties that
could cause our actual results and the timing of certain events to differ materially from any future results
expressed or implied by the forward‐looking statements, including, but not limited to, risks as to whether
final and secondary data will be accepted as encouraging, positive or will otherwise lead to an effective
or approved product, whether we will be able to demonstrate efficacy or gain necessary approvals to
market and commercialize any product, whether the FDA will accept our data, accept our submission of
the BLA, continue to recognize its previously reported guidance, grant a license, or approve RI‐002 for
marketing, whether we will meet any of our clinical or regulatory milestones, whether we will develop
any new products or expand existing ones, whether we will receive FDA approval of our new facility,
whether there may be changes in regional and worldwide supply and demand for source plasma,
whether we will be able to attract sufficient donors and operate our new facility effectively or profitably,
whether we can sell our plasma in the marketplace at prices that will lead to adequate amounts of
revenue, whether we will be able to sustain the listing of our common stock on the NASDAQ Capital
Market, whether we will meet any timing targets expressed by the Company, and other risks and
uncertainties described in our filings with the U.S. Securities and Exchange Commission, including our
most recent reports on Forms 10‐K, 10‐Q and 8‐K, and any amendments thereto. Therefore, current and prospective security holders are cautioned that there also can be no assurance that the forward‐looking
statements included in this press release will prove to be accurate. In light of the significant uncertainties
inherent to the forward‐looking statements included herein, the inclusion of such information should not
be regarded as a representation or warranty by ADMA or any other person that the objectives and plans
of ADMA will be achieved in any specified time frame, if at all. Except to the extent required by
applicable laws or rules, ADMA does not undertake any obligation to update any forward‐looking
statements or to announce revisions to any of the forward‐looking statements.
CONTACT: Brian Lenz
Vice President and Chief Financial Officer |201‐478‐5552 | www.admabiologics.com
