An orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases (orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States. In 2003, the leading orphan drug by worldwide sales revenue was Amgen's Erythropeietin (Epogen®), with sales of $2.4bn.
Geert Kersten, CEO of CEL-SCI said, "Receiving orphan-drug designation for Multikine is yet another major achievement for CEL-SCI, following the go-ahead from the FDA earlier this year for the Company's Phase III study in advanced primary head and neck cancer patients and the subsequent raising of substantial funds in April, 2007. We are putting in place all of the pieces required to make this company very successful."
(Amex: CVM) closed friday's trading at 81 cents per share.
Further Reading: A letter to shareholders:
Dear Fellow Shareholders:
So far 2007 has been an excellent year for CEL-SCI and its cancer product Multikine®. In January we received the US FDA go-ahead for the Phase III clinical trial with Multikine, a huge achievement for any drug, yet an even bigger achievement for a completely new type of drug such as our Multikine. We followed this up with a $15 million commitment to finance the Multikine manufacturing facility in Baltimore, Maryland and another $15 million in equity financing in April. In June we announced that we received orphan drug status from the FDA for Multikine. This designation brings many substantial benefits to the Company. I believe that the opportunity ahead for CEL-SCI is so compelling that I have continued to purchase CEL-SCI stock in the open market.
In talking to investors it has become clear to me that few investors understand the enormous impact that our Multikine will have on the treatment of cancer if we can confirm in our Phase III study the impressive survival results we have seen in our Phase II trial. I believe that a background summary on Multikine is helpful for that reason.
Background Summary on Multikine:
-- The science: The science behind Multikine is based on the premise that
a healthy immune system can cure cancer. When the immune system is
imbalanced or weakened, diseases like cancer occur. Balancing and
strengthening the immune system is thought to be an effective strategy
in fighting cancer.
-- What is Multikine? Multikine is a mixture of naturally occurring
cytokines, substances that regulate the immune system. The patented
Multikine mixture is representative of the mixture of cytokines
produced by a healthy immune system. CEL-SCI scientists have published
in the highly regarded "Journal of Clinical Oncology" that many
different cytokines working together are needed to put in motion the
comprehensive immune response needed to fight cancer. Most other
immunotherapies being developed are based upon the use of only one
cytokine.
-- How is it used? Multikine is the first cancer immunotherapy being
developed as a first line treatment. This means it is administered
prior to any other cancer therapy (e.g. surgery, radiation and/or
chemotherapy) because that is the time when the immune system can still
be fully activated. Once the patient has had surgery or has received
radiation and/or chemotherapy, the immune system is severely weakened
and less able to mount an effective immune response. We believe that
most of the failures of immunotherapy drugs up to now have been the
result of giving immunotherapy after the patient's immune system was
damaged by other cancer therapies.
-- How does it work? Multikine works in two ways:
1. It signals the immune system to mount an effective immune response.
Multikine changes the type of immune cell that attacks the tumor
from a CD-8 cell to a CD-4 cell. This is important because tumors
are able to defend themselves against the normal CD-8 cell attack,
but appear to be unable to defend themselves against the CD-4 cell
attack. The CD-4 cell breaks what is known as "tumor tolerance,"
thereby allowing the immune system to see and attack the tumor.
2. Multikine has been shown to render cancer cells much more
susceptible to radiation and chemotherapy treatments, thereby making
these treatments much more effective.
The combination of both of these is very important because Multikine
will fight the tumor on two fronts, without toxicity.
-- Results: In a Phase II clinical trial, Multikine was shown to increase
overall survival by 33% 3.5 years after the first treatment.
-- Its effect on recurrence of cancer: Chemotherapy, surgery and
radiation do not always succeed in killing all the cancer cells. The
persistence of cancer cells is responsible for cancer recurrence.
Multikine is injected around the tumor and into the local lymph nodes
because those areas are the most likely places for the cancer to recur
if any of the cancer cells survive. Multikine uses the immune system
to kill those cancer cells before they cause tumor recurrence.
-- Safety: Multikine has been shown to be safe and non-toxic in multiple
studies. This was not surprising because Multikine consists of
naturally derived substances given in very small dosages, just as the
body does itself every day.
-- Potentially useful for multiple tumors: Despite the fact that the
Multikine treatment given to each patient is the same (i.e., not
patient specific), the immune response induced by Multikine is
completely targeted to the patient's own tumor. This means that
Multikine may be useful against many different tumors. Multikine has
already shown activity in small studies in cervical dysplasia/neoplasia
and prostate cancer. One of its mechanisms of action suggests that it
should also be developed as an enhancement of radiation and
chemotherapy.
-- Clinical Status: Multikine is currently cleared for a Phase III
clinical trial in the US and Canada in advanced primary head and neck
cancer patients. These are recently diagnosed, not yet treated
patients. Head and neck cancer is an aggressive cancer that accounts
for about 500,000 - 600,000 new annual cases of cancer world-wide.
Advanced primary head and neck cancer patients typically have a 50%
chance of survival three years following the first treatment.
Treatments for newly diagnosed head and neck cancer have not
significantly improved over the last 50 years and therefore constitute
a clear unmet medical need.
The Phase III study, which is designed to prove that Multikine works,
is expected to enroll about 800 advanced primary head and neck cancer
patients worldwide. In its last Phase II clinical trial Multikine
increased overall survival by 33%. The Phase III study will need to
show a 10% increase in overall survival to be successful.
-- Market size: The current Phase III clinical trial pits Multikine plus
the standard of care treatments against the standard of care treatments
alone. A win for the Multikine treatment group would mean that every
patient with advanced primary head and neck cancer is supposed to get
Multikine. This would translate into a market potential of $15
billion.
We believe that Multikine will ultimately be added to the cancer treatments of many solid tumors. This it will have a huge impact on the treatment of cancer because curing more patients with the first cancer treatment will significantly decrease the number of cancer deaths. We thank you for being part of this dream.
As shareholders we truly value your participation in and support of our company. In an effort to improve our image, which has not yet caught up to where the company is today, we are currently redeveloping our corporate identity and redesigning our website. You are our most important advocates and we want your voice to be heard. We would like to collect some personal letters from you that describe why you support CEL-SCI and Multikine, and what your hopes are for Multikine. Our hope is to publish these letters on our website. Please mail these letters to Gavin de Windt, Investor Relations Manager, CEL-SCI Corporation, 8229 Boone Boulevard, Suite 802, Vienna, VA 22182, USA or send it by e-mail to gdewindt@cel-sci.com .
