Item 1. Description of Business.
Corporate History and Intercorporate Relationships
The Company was incorporated under the laws of the State of
Nevada on October 5, 2004. Until recently the Company was a software development
company and its plan was to commercialize an enterprise information portal and
related software applications. It was in the early marketing stages of its
software application and infrastructure build out, and has not as yet generated
any revenue. Through the merger of its wholly-owned subsidiary, Icon Acquisition
Corporation (Merger Sub), with and into American Xeno Inc. (AXI) described
below, the Company has undertaken a new strategic and business direction. The
Company is now a bio-technology research and development company that holds an
exclusive commercial license from Massachusetts General Hospital (MGH) derived
from over 15 years of development in the field of xenotransplantation by
Novartis Pharmaceuticals (Novartis) and its associated research entities.
Xenotransplantation is defined as the transplantation of organs, cells and
tissues from one species to another. At this time, the Company has no employees
and no material business operations. AXI is a bio-technology research and
development company incorporated in Nevada on February 8, 2006 and following the
Merger (as defined below) became a wholly-owned subsidiary of the Company.
On May 3, 2007, an agreement and plan of merger (the
Agreement) was executed with AXI, Merger Sub, and the stockholders of AXI, and
on May 8, 2007 Merger Sub was merged with and into AXI and every two issued and
outstanding shares of common stock of AXI were converted into one share of
common stock of the Company (the Merger) resulting in an aggregate of
29,875,000 shares of common stock being issued. Pursuant to the Merger the
Company also cancelled 12,500,000 shares of common stock previously held by a
founding shareholder. As a result of the Merger, AXI became a wholly-owned
subsidiary of the Company and a change of control of the Company occurred as the
AXI stockholders acquired approximately 74% of the issued and outstanding shares
of common stock following the closing of the Merger.
As a part of the Merger and as set forth in the Agreement, the
Company intends to complete a private placement (the Private Placement) of up
to 1,500,000 units (Units) of the Company at a price of $1.00 per Unit to
certain eligible investors by December 31, 2007. Each Unit will consist of one
share of common stock and one common stock purchase warrant of the Company. Each
whole purchase warrant will entitle the holder for two years from the date of
issuance of the Units to acquire one additional share of common stock of the
Company at an exercise price of $1.00.
For U.S. federal income tax purposes, the Merger was effected
as a qualified reorganization under the provisions of Section 368(a) of the
United States Internal Revenue Code of 1986, as amended. For accounting
purposes, the Merger was treated as a reverse merger with AXI being the
accounting acquirer and the go-forward financial statements reflect AXIs
history from its inception on February 8, 2006. The fiscal year-end of AXI was
changed to June 30 from December 31; consequently the financial statements for
the current period presented are for the six months ended June 30, 2007 and the
comparative figures presented are for the period from inception on February 8,
2006 to December 31, 2006.
On August 13, 2007 the Company changed its name from Icon
Development, Inc. (ICON) to Xeno Transplants Corporation.
General Overview
Following the Merger, the Company has undertaken a new
strategic and business direction. The Company intends to develop therapeutic
applications of organs and cells derived from genetically engineered pigs as a
transplant alternative to limited human donor sources. The transplantation of
organs, cells and tissues from one species to another is defined as
xenotransplantation.
Xenotransplantation is intended to address the problems arising
from the limited supply of available human cells, tissues and organs for
transplantation by developing technologies to permit the transplantation of
cells, tissues and organs from other species, such as swine, into humans. There
is a critical shortage of sources for transplantation worldwide.
5
Critical Issues Governing the Application of
Xenotransplantation
A need exists for the creation of pig organs which can match
the size needed for replacing human organs. In order to address this issue, a
pig herd has been created of inbred miniature swine that develop organs which
are human in size. It is thus possible to match the pig donor and human
recipient organ sizes, which is expected to be an important factor for success
in transplantation.
It is also desirable to create a pig herd with uniformity to
facilitate production, quality assurance and quality control of donor cells and
organs. The genetic profile of the planned breeding nucleus to be selected from
the current herd should ensure uniformity of the pigs in the herd, which could
become a production lot, rather than each animal being a unique production
lot. Uniformity also facilitates genetic engineering of the herd.
Safety concerns caused by Porcine Endogenous Retrovirus
(PERV) must be addressed. As discussed in the regulatory progress section,
xenotransplantation guidelines have been created by the FDA and clinical trials
have been opened and completed under these guidelines. Beyond the existing
guidelines, miniature swine have been identified by the Company that are
believed to be incapable of infecting human cells, based on laboratory studies,
with PERV. Their subtype of PERV has been shown not to recombine with human
endogenous retroviral gene segments.
Research demonstrating the efficacy of life supporting pig
organs and cells in primates is necessary before human clinical trials can be
conducted. Research evidence has been demonstrated for donor specific
immunological tolerance to organs from cloned miniature swine whose expression
of alpha galactose (the major target of rejection) has been knocked out. This
combination of technologies, tolerance and pig genetic engineering, has resulted
in survival of life supporting pig kidneys in primates for up to 83 days,
without evidence of rejection. In addition, tolerance is now being demonstrated
in patients receiving allogeneic organ transplants.
Chronology of Development
During the 1980s, development began on a herd of miniature
swine inbred for specific genes. Research was conducted demonstrating
transplantation tolerance between mice and rat tissues. In 1991, work to create
a more fully inbred miniature swine herd was started. In 1996, tolerance was
demonstrated for pig skin transplanted into mice. During 1999, tolerance
protocols were further refined for pig tissue transplanted into mice and a
research team demonstrated that there was no detectable PERV transmission in
patients that had received living pig tissue and who could be traced. In 2000,
tolerance was refined in transplants between mismatched pig models. During 2004,
replication competent human tropic PERV were shown to be absent from the germ
line of certain inbred miniature swine, and resulting mouse models with
humanized bone marrow demonstrated that PERV was not transmitted into human
cells. In 2005, evidence of tolerance was demonstrated in life supporting pig
kidneys transplanted into baboons.
The Companys Intellectual Property
Massachusetts General Hospital
On May 16, 2006 (the Effective Date), AXI signed an exclusive
license agreement with MGH to have the exclusive right to commercially develop,
manufacture, distribute and use products and processes for public use with
regard to xenotransplantation. The MGH license includes 38 issued U.S. patents
and 51 issued international patents, with multiple patent applications.
Technologies covered by AXIs exclusive commercial licensee from MGH include the
following:
- Exclusive rights to the
commercial use of a herd of proprietary in-bred miniature swine for
xenotransplantation. The AXI pig herd has been in-bred over decades in a
controlled scientific environment in order to ensure greater animal uniformity,
which facilitates consistent transplant quality control.
- The Company has identified
proprietary miniature swine which are believed to be incapable of infecting
human cells, based on laboratory studies. This may be an important factor in
ensuring transplantation safety.
6
- AXIs exclusively licensed
patent rights include the methods and composition for targeting and eliminating
a major cause of rejection of xenotransplants.
- The Company believes it will be
in a strong position in the field of tolerance whereby a patients immune system
can be re-educated to more closely recognize transplanted foreign cells, tissues
and organs as self, reducing the requirement for chronic systemic
immunosuppressive drugs.
In order to maintain its license in good standing, the Company
must pay to MGH:
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1)
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$25,000 within 30 days of the Effective Date (paid).
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2)
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$175,000 within 18 months of the Effective Date or when
the Company secures $5,000,000 in financings, whichever is earlier.
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3)
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Reimbursement for any costs associated with the preparation,
filing, prosecution and maintenance of all patent rights.
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4)
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An annual license fee of $5,000 for the first four years,
and $20,000 for every year thereafter (this fee can be waived if the Company
spends a minimum of $100,000 per year for other hospital supported patent
projects).
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5)
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$1,125,000 per product that is successfully brought
to commercial production. Payments that add up to the $1,125,000 will
be due upon each successful stage to obtain commercial production.
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6)
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A royalty of 6% of net sales (as defined in the agreement).
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The Company is obligated to perform the following from the
Effective Date of the agreement:
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1)
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Raise $500,000 by March 31, 2007 (completed).
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2)
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Raise an additional $5,000,000 within 18 months of the
Effective Date.
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3)
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Commit $100,000 per year for two years for support of
animal research within two years of the Effective Date.
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4)
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Complete various phases, studies, and applications as set
forth in the agreement.
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The term of the agreement is in effect until such time as all
obligations have been met by the Company.
Infigen, Inc. Patents
On January 31, 2007, AXI also acquired a patent assignment from
Infigen, Inc. consisting of two issued U.S. and seven issued international
patents and multiple patent applications relating to producing pigs by cloning,
including the methods used in producing the Companys genetically engineered
pigs. In these miniature swine a specific porcine gene, which is a major cause
of xenotransplantation rejection, has been knocked out. The genetic
engineering uses somatic cell nuclear transfer (SCNT), including oocyte
maturation, oocyte/donor cell fusion, oocyte activation and embryo transfer.
This is often referred to as pig cloning. SCNT is presently the only known
method for introducing complex and predictably regulated genetic modifications
into the pig genome. The Infigen patents cover key technologies with respect to
pig cloning and have been demonstrated to be enabling. The Company believes that
these patents represent one of the most efficient processes currently available
for performing genetic modifications in pigs.
Business Relationships
The Company also has an agreement with Minitube of America,
Inc. (Minitube) whereby Minitube will assist the Company in developing its
intellectual property. The term of the agreement is for six months, ending July
2007, and the Company is obligated to pay $166,000 for setting up costs and
consulting fees related to the agreement, and reimburse Minitube for any
additional expenses they may occur, including sampling fees. Minitube agreed to
accept 68,000 units, consisting of one share of common stock of the Company and
one-half of one warrant to purchase one share of common stock at a price of
$2.00 exercisable for 18 months, at a deemed price of $1.00 per unit in payment
of $68,000 of the total amount.
Minitube has demonstrated leadership in developing and engineering
innovative technologies for bovine, porcine, canine and equine reproduction.
They have expertise applicable in optimizing the breeding and husbandry of the
miniature swine herd for use in pre-clinical and clinical studies and later
meeting commercialization requirements. In a collaborative program under the
contract Minitube will evaluate and optimize environmental conditions, pig nutrition,
sow farrowing and nursery systems. The breeding facility will also be renovated
to accommodate ergonomic pig handling, segregation of boars and sows, and the
development of a custom miniature swine mounting phantom for semen collection.
The boars will be trained for semen collection and an andrology lab will be
established to evaluate semen samples for artificial insemination. An estrus
detection system and stud-sow artificial insemination management will be established.
Pregnant sow management will be established, including farrowing preparations,
sow lactation, evaluating milk supplements, baby pig weaning and husbandry.
7
Acquisition Letter of Intent
On September 20, 2007 the Company entered into a Letter of
Intent (the LOI) to acquire CrossCart, Inc. (CrossCart), a privately held
California corporation.
CrossCart has developed and patented a technology intended to
make animal tissues usable for human applications. The process has been tested
in primate models and human studies and clinical trial of an anterior cruciate
ligament (ACL) replacement device has received FDA approval.
CrossCart is expecting to roll out the ACL device in Europe
over the next one to two years, and subsequently intends to complete the U.S.
pivotal trial and develop further applications such as soft tissue patches and
bone dowels and screws.
The Companys technology potentially provides the opportunity
for xenotransplantation of multiple types of organs and cells, including whole
organ liver and kidney transplants, and islet cell transplants, from the same
proprietary genetically modified donor animal, GalT-KO miniature pigs.
CrossCart's technology is potentially applicable to a variety of medical devices
including orthopedics, ligaments, cartilage and bone; cardiovascular materials,
including heart valves and vascular grafts; and cosmetics, including soft tissue
patches and injectable collagen.
Management of the Company believes that that the acquisition of
CrossCart has the potential to significantly improve the business opportunities
of both companies through the following key factors:
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the potential to significantly accelerate the Companys product
commercialization timetable;
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the possibility of expanding the Companys potential markets and
diversifying revenue streams; and
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it could create strong synergies in the Companys research and development
programs and administrative cost savings.
The terms of the LOI are summarized as follows:
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the terms of the LOI are subject to negotiation and execution of a
definitive acquisition agreement which shall be approved by the respective
boards of directors of each corporation and by the stockholders of CrossCart;
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all of the stock of CrossCart, including common stock, preferred stock,
and stock issuable on conversion of debt, shall be exchanged for 22 million
shares of common stock of the Company. CrossCart will become a wholly-owned
subsidiary of the Company;
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certain current stockholders of the Company and CrossCart will negotiate
the terms of a voting trust whereby all their combined stock will be voted
together for any transaction or event involving a material acquisition or
disposal of assets, a change in control, a change in the majority of
directors, or a change in senior management;
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the CrossCart stockholders will have the right to nominate two directors
to the board of the Company;
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the Company shall advance $250,000 to CrossCart for operating capital, on
or before October 30, 2007, in the form of a non-refundable deposit. In the
event that the acquisition does not close, the $250,000 will be converted to
an investment in CrossCart by the Company on the same terms as the next
financing obtained by CrossCart;
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a further contribution of $2.25 million in operating capital will be
provided by the Company to CrossCart on or before the closing date of the
acquisition; and
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closing is set to occur on or before November 30, 2007.
8
Research and Development
As the Company is in the early stages of its development it has
not incurred any research and development expenses to date. Its total
expenditures have related to administrative costs and costs associated with the
acquisition, maintenance and development of intellectual property.
Markets
Transplantation Market Overview
Worldwide, there is a large and increasing shortage of donor
tissues and organs for critically ill patients. In the US alone, the current
waiting list for an organ transplant is over 90,000 patients.
i
In
addition to patients on the waiting list, many more patients could benefit from
transplants but are deemed ineligible because of their condition and the extreme
shortage of donor tissues and organs. Thus, the large and growing number of
patients on the waiting list for an organ transplant falls far short of the true
need.
Not only are human donor organs in scare supply, the quality of
donor organs has declined as demand has increased. While there was a sufficient
supply of pristine organs from healthy young adults (generally head trauma
victims) in the early days of organ transplantation, more and more so-called
marginal or extended criteria organs are now being transplanted from older
donors with chronic conditions and infections. There is no government agency
that sets standards for organ acceptability for transplant. The US Department of
Health and Human Services contracts with the United Network for Organ Sharing
(UNOS) to handle transplant system logistics, and distribution based on
medical need. However, decisions about whether organs are usable are made by
individual surgeons, and there is a high degree of variability in this
decision-making process.
The market change through availability of genetically
engineered porcine xenografts is potentially transformational, in that it
addresses the central dilemma of transplantation -- that people must die or
undergo traumatic procedures in order for patients with end-stage organ disease
to survive. The economic benefits also could be substantial.
Xenotransplantation provides the possibility of becoming a
viable alternative for transplant patients, as a widely available, life-saving,
cost-effective treatment that could also offer improved quality of life.
______________________________________
i
Organ
Procurement and Transplantation Network, data as of July 20, 2007, United
Network for Organ Sharing
9
Liver Transplantation
Liver transplantation, including xenotransplants for temporary
support and bridge indications as well as destination therapy, could provide a
potentially large market opportunity for the Company. Liver transplantation
currently offers the only definitive life-saving therapy for patients with
end-stage liver disease (ESLD) or fulminant hepatic failure. Allogeneic liver
transplantation is highly successful, with an overall one-year patient survival
rate of approximately 87% for patients with primary liver
transplants.
i
Unfortunately, according to UNOS, more than 16,000
individuals in the US alone await donor liver transplantation
ii
,
while only about 6,000 cadaveric and 300 living donor transplants have been
performed annually in recent years.
iii
The gap between supply and
demand is increasing yearly by 10 to 15%
iv
, and up to 15% of patients
in need of a transplant die each year before a suitable organ can be
identified.
v
Moreover, the large number of patients on the waiting
list for transplantation has been kept artificially low due to stringent
criteria that exclude many patients. Innovative surgical techniques, greater
reliance on living donors for liver segment donations for pediatric patients,
cadaveric split liver transplantation for pediatric patients, and use of
extended criteria donors, have been adopted to address the critical donor
shortage. Despite these efforts, the demand for liver transplants continues to
greatly surpass availability. Living donor liver transplantation currently
provides about 5% of graft needs, and research indicates that this figure is not
expected to increase significantly in the future.
vi
For adult
patients, xenotransplantation may provide the only realistic solution. Moreover,
the number of patients eligible for liver transplantation would increase
significantly if the organ shortage were resolved.
ESLD caused by hepatitis C virus (HCV) is the leading
indication for liver transplantation, accounting for about 60% of transplant
recipients.
vii
The prevalence of ESLD in the US is approximately 5%
(15 million patients).
viii
In the US alone, about 4.1 million people
are infected with HCV, and 70 to 80% of these individuals are expected to
develop chronic liver disease.
ix
ESLD patients typically experience
recurrent disease early after transplantation since effective antiviral agents
for HCV are still not available. Because of the poor results after
retransplantation in ESLD patients and the scarcity of donor organs, repeat
transplantation is seldom indicated. This situation could be substantially
eliminated with xenotransplantation from GalT-XO miniature pigs, particularly
since there is evidence that human HCV does not infect the porcine liver; as
such, xenotransplantation could become the method of choice for these
patients.
The Companys management currently projects a price of $50,000
per xenotransplant organ. This figure is based on the current acquisition fee of
approximately $25,000 for a human donor organ
x
, which has minimal
clinical history and a resultant risk of disease and deterioration. Human organs
are usually supplied on an emergency basis, matching the death of a donor with
the critical condition of a recipient. In contrast, the Companys GalT-KO
xenotransplant organs would be procured from inbred miniature swine raised in a
GMP-SPF barrier facility, with extensive monitoring and testing from birth to
sacrifice for a scheduled transplant. Given the sizeable number of known and
potential liver transplant recipients, the market potential for the Companys
GalT-KO porcine liver xenotransplants could be very large.
_____________________________________
i
Organ
Procurement and Transplantation Network, Liver Kaplan-Meier Graft Survival Rates
for Transplants Performed 1997-2004
ii
Organ Procurement and
Transplantation Network, data as of July 20, 2007, United Network for Organ
Sharing
iii
Organ Procurement and Transplantation Network, Liver
Transplants Performed in the US, January 1, 1988-April 30, 2007
iv
Organ Procurement and Transplantation Network, data as of July 20,
2007, United Network for Organ Sharing
v
Harper, AM, The OPTN
Waiting List, in
Clinical Transplants 2000
, JM Cecka and PI Terasaki,
eds. 2001, UCLA Immunogenetics Center: Los Angeles. p 73-84
vi
2006 US Organ Procurement and Transplantation / Scientific Registry of
Transplant Recipients Annual Report
vii
Armstrong, GL, et al.,
The past incidence of hepatitis C virus infection: implications for the future
burden of chronic liver disease in the United States. Hepatology 200. 31(3):
777-782
viii
Ibid.
ix
US Centers for Disease
Control and Prevention, 2007
x
New England Organ Bank standard
acquisition fees for the period July 1, 2004 through June 30, 2005.
10
Kidney Transplantation
In the US alone, approximately 16,000 kidney transplants are
performed annually and over 72,000 patients are on the waiting list for
transplants.
i
The vast majority of renal failure patients are treated
by dialysis, which currently is the only alternative to transplantation;
however, dialysis is more expensive and has higher rates of morbidity and
mortality than transplantation. Data from the National Kidney and Urologic
Diseases Information Clearinghouse demonstrate the large differences in survival
times between dialysis and kidney transplantation patients (see figure below).
If xenotransplants were available to increase the supply of donor organs,
treatment also could occur earlier in the progression of renal disease and
before damage occurred elsewhere in the body.
The initial focus of the Companys Kidney Transplant Program
will be on dialysis patients with high sensitization against human tissues, who
are unlikely to be offered a human kidney transplant. About 15% of the 300,000
ESRD patients on dialysis have high panel reactive antibodies (PRAs) against
human donor tissues. MGH has found low PRAs in these patients against porcine
tissues. For this subgroup of patients alone, the market potential is very
large.
Additional Transplant Programs
The Company is also evaluating other promising GalT-KO
xenotransplant product development opportunities for major unmet medical needs.
Islet Cell Transplantation
The Company is exploring technology options and potential
collaborations for developing the use of porcine pancreatic islet cells from its
GalT-KO miniature swine to treat brittle type 1 diabetics and diabetic
patients with renal failure. There are approximately 100,000 brittle type 1
diabetics in the US alone who do not respond adequately to insulin therapy.
These patients might be able to achieve an improved life expectancy if islet
cell transplants were not limited by the lack of donor cells. Islet cell
transplants have demonstrated the capability to remove short-term insulin
dependence in brittle diabetics, but with only approximately 500 pancreases
available each year in the US for islet cell extraction and transplantation, the
development of this potentially life-saving therapy is currently severely
limited to a very small minority of patients.
FDA Approval Process
Development of a therapeutic product for human use is a multi-step
process. First, in vitro and animal testing must be conducted in a manner consistent
with good laboratory practices to establish the potential safety and effectiveness
of the experimental product in a specific disease. Before human clinical trials
may begin, an investigational new drug or other application containing the preclinical
data, manufacturing and control information, and a clinical investigative plan
must be submitted to and accepted by the FDA. In addition, approval and oversight
by a institutional review board and adherence to requirements for proper informed
consent from study subjects are required.
_______________________________________
i
Organ
Procurement and Transplantation Network, data as of July 20, 2007, United
Network for Organ Sharing
11
Clinical trials typically involve three phases, although those
phases can overlap. Phase I is conducted to evaluate the safety, and if possible,
to gain early indications of effectiveness in the patient population for which
the product is intended for use. Phase II clinical trials are conducted in groups
of patients to further study safety and effectiveness. Phase III studies are
usually randomized, double blind studies testing for product safety and effectiveness
in an expanded patient population to evaluate the overall risk/benefit relationship
of the product. The Company will accrue clinical and pharmaco-economic data
to facilitate both FDA and reimbursement approval. Because of the potential
lifesaving role of Xenos products and the unfulfilled demand of significant
numbers of potential patients, the Company may receive Orphan Drug, Fast Track
and Priority Review designations by the FDA for each of its product research
and development programs. There can be no assurance, however, that the Company
will receive such designations.
Following completion of clinical investigations, the preclinical and clinical data that have been accumulated, together with chemistry, manufacturing and controls specifications and information, are submitted to the FDA in a Biologics License
Application for review and approval. For medical devices a PMA must be submitted for review and approval.
Regulatory Progress in Xenotransplantation
Safety concerns in the mid 1990s slowed the progress of xenotransplantation clinical trials. However, because of its enormous potential public health benefits in combination with infectious disease risks, the Secretary of Health and Human
Services asked the FDA and Centers for Disease Control and Prevention to form a special committee to evaluate the risks posed by xenotransplantation.
The major focus was on the potential transmission of PERV. While there was no PERV transmission in previous clinical studies, there were many closed committee meetings and public hearings, which led to defined regulatory guidelines to minimize the
safety risks of xenotransplantation. The regulations include requirements for barrier facilities and their operations, which are defined so that exogenous pathogens are removed from the donor herd, and for monitoring of patients.
The special committees deliberations also uncovered some unique benefits of xenotransplantation beyond increasing the supply of donor cells, tissues and organs. These benefits include the ability for careful microbiological screening of the
animal donors from birth to sacrifice, as opposed to the limited screening of human donors before transplantation. Based on these regulatory guidelines, multiple clinical trials have been opened and completed. The regulatory consensus is that
infectious disease and patient monitoring safety concerns have been addressed in the guidelines. For efficacy of cell and organ transplants, there are clearly defined and monitored endpoints, which have been utilized in the development of existing
transplantation drugs and devices.
12
Competition
Source of porcine organs
The Company believes that its proprietary inbred miniature swine will be desirable donors for xenotransplantation because of the ability to match donor and recipient organ size and the potential to identify miniature swine donor pigs which may be
incapable of infecting human cells. The Company is unaware of any other suppliers of inbred miniature swine.
Porcine cloning
The Company considers Revivicor and the Mayo Clinic group as potential competitors in using nuclear cloning and genetic engineering of pigs for xenotransplantation.
Geron and their joint venture, stART Licensing Inc., may hold some relevant intellectual property (originally from the Roslin Institute) that they have been nonexclusively licensing to interested parties. These patents have broad generic claims to
methods of nuclear transfer in which quiescent cells are used as the nuclear transfer donor cell. Nuclear transfer cloning has also been successfully accomplished by Revivicor and the Mayo Clinic group. The Company is leveraging the prior work done
with Immerge, Infigen and University of Missouri in this area. The major target for this work by all the above groups was the production of pigs whose organs reduce rejection.
The other area of interest has been dominant transgenesis of human complement inhibitor proteins in pigs. The Company has control of the assets and intellectual property from Novartis and Imutran in this area. The Mayo group and Revivicor both have
pigs with similar but not identical proteins expressed.
Tolerance
From the recent Nature Medicine publications, the Company expects that creation of tolerance will be necessary to prevent immune responses to further develop against swine organs in primate recipients. Through the Companys license with MGH, it
believes that it has a strong position in the field of tolerance (the re-education of the patients immune system to more closely recognize foreign cells, tissues and organs as self) to potentially reduce the need for chronic
systemic immunosuppressive drugs. The Company is unaware of current competitors that are creating tolerance in primates.
Stem Cells
Stem cell research to create organs is potentially attractive but is also anticipated to be at least 5 to 10 years away from experimental clinical utility and an additional decade for commercialization. Even for creating cells and tissues, it is
unclear if human embryonic stem cells are controllable enough in their differentiation to specific cells for assuring both their safety and efficacy in transplantation. Also, if the cells are derived from a single source of stem cells and
transplanted to multiple recipients, they will require immunosuppression against rejection. If autologous cells are transplanted after nuclear transfer, the underlying autoimmunity that caused the disease (e.g. Type 1 diabetes) will still need to be
controlled against a patients autologously derived cells. The use of hematopoietic stem cells has not demonstrated consistent results to date.
In contrast to the extensive history and acceptance of porcine based products in medical applications, the creation and destruction of embryonic stem cells continue to encounter significant moral/religious opposition.
13