AbGenomics International (Private)

Initiating Coverage
April 4, 2016
AbGenomics International (Private)
Initiation Report
LifeSci Investment Abstract
AbGenomics (private) is a biopharmaceutical company developing antibody therapeutics
for inflammatory diseases and cancer. The Company’s lead candidate is AbGn-168H
(neihulizumab), a targeted therapy for the treatment of acute graft-vs-host disease (aGvHD).
AbGn-168H is currently being evaluated in a Phase Ib trial in steroid refractory aGvHD,
with results expected in the second half of 2016. Results from a proof-of-concept Phase II
trial in psoriatic arthritis (PsA) demonstrated that 40% of subjects treated with AbGn-168H
achieved a 20% reduction in symptoms at week 12. The Company’s preclinical oncology
product candidates are next generation antibody drug conjugates (ADC) for solid tumors.
Analysts
Jerry Isaacson, Ph.D. (AC)
(646) 597-6991
jisaacson@lifescicapital.com
Key Points of Discussion
■
■
Targeted T-cell Regulatory Agent with Broad Applications in Autoimmune and Inflammatory Diseases. AbGenomics is
developing AbGn-168H (neihulizumab) for the treatment T-cell mediated inflammatory conditions including steroid refractory acute
graft-versus-host disease (sr-aGvHD). Many inflammatory disorders share a common disease mechanism that centers on the abnormal
and chronic activation of T-cells. AbGn-168H is a monoclonal antibody that activates P-selectin glycoprotein ligand-1 (anti-PSGL-1/
CD162) and preferentially kills the T-cells that cause inflammation through the natural mechanism of activation-induced cell death.
This activity has the potential to reduce or eliminate tissue damaged associated with inflammation without the need for agents that
cause broad immunosuppression. Since the therapy is designed to eliminate, and not simply inhibit the pathogenic T-cells that underlie
these inflammatory conditions, treatment with AbGn-168H could result in durable and long-term responses.
AbGn-168H for the treatment of sr-aGvHD. sr-aGvHD is a potentially life threatening complication of allogeneic hematopoietic
cell transplantation (alloHCT). The condition arises when transplanted donor T-cells begin attacking the patient’s tissues, causing
inflammation and organ damage. First-line treatment for aGvHD is steroids, however only about 40% of patients achieve a durable
response with this therapy. sr-aGvHD remains a major cause of alloHCT-related mortality, with overall mortality estimated to be as high
as 95% by year three. Approximately 70% of sr-aGvHD-related deaths result from ongoing and uncontrolled aGvHD, and 11-30% are
due to severe opportunistic infections. There is a high unmet medical need for a treatment such as AbGn-168H that has the potential
to be an effective and safe treatment for sr-aGvHD.
■
Encouraging Responses in First Two sr-aGvHD Patients Treated with AbGn-168H. An open-label, dose-escalation Phase Ib
trial with AbGn-168H in sr-aGvHD patients is being conducted at Stanford University Medical School’s transplant center, and is
expected to enroll 12 patients. Subjects will receive AbGn-168H once a week for 4 weeks following sr-aGvHD diagnosis. The first
patient will receive 6 mg/kg of AbGn-168H, and subsequent patients will either be dose-escalated or de-escalated depending on the
safety and tolerability of AbGn-168H treatment. The primary endpoint of the trial is the dose-limited toxicity as assessed by:
◦ Grade 3-5 adverse events considered at least possibly-related to AbGn-168H occurring after the first dose and within 30 days after
the fourth dose.
◦ Grade 3-5 cytokine release syndrome or acute infusion reaction within 24 hours after AbGn-168H infusion.
◦ Grade 4 neutropenia considered at least possibly related to AbGn-168H, lasting more than 14 days.
◦ Grade 5 all-cause mortality with 7 days of infusion.
Two patients with sr-aGvHD have been treated with AbGn-168H. The patients were initially administered 6 mg/kg of AbGn-168H at
the start of therapy. A reduction from Grade II disease to Grade 0 was achieved in one of the patients after the first week of treatment.
The GvHD remained under controlled and the subject was subsequently released from the hospital. The second patient treated with
For analyst certification and disclosures please see page 28
Page 1

April 4, 2016
AbGn-168H experienced a reduction from Grade IV disease to Grade II by week 4. These early results are encouraging, and suggest
that AbGn-168H has the potential to be a safe and effective treatment for sr-aGvHD. Full results from this trial are expected in the
second half of 2016.
■ Planned Phase II study for AbGn-168H in sr-aGvHD. AbGenomics plans to initiate a randomized, open-label, Phase II trial for
AbGn-168H in patients with sr-aGvHD. The study will take place at 40 centers across the US, including Stanford Medical Center, The
Fred Hutchinson Cancer Center, MD Anderson Cancer Center, and Memorial Sloan Kettering Cancer Center. At least 90 subjects will
be randomized to receive AbGn-168H or best supportive care. The primary endpoint is overall response at Day 28, which is defined a
complete response (CR) or partial response (PR). Second endpoints include overall response rate at Day 90, as well as overall survival
at Day 90 and Day 180. The Company plans to initiate the study in the second half of 2016.
■ Large Market Opportunity for AbGn-168H in sr-aGvHD. The number of alloHCTs has grown steadily since 1980. There were
roughly 27,000 transplants performed worldwide in 2010, and 7,500 in the US in 2012. Approximately 60% of patients who undergo
alloHCT are expected to develop aGvHD, indicating that there are approximately 4,500 cases in the US and 16,200 cases worldwide
each year. Around 60% of aGvHD will be refractory to steroid treatments, meaning that there are about 2,700 patients in the US and
9,700 patients worldwide each year that would be eligible for treatment with AbGn-168H. Assuming a peak market penetration of
70% by 2023, later in this report we estimate that worldwide revenue for AbGn-168H could exceed $900 million.
■ AbGn-168H Demonstrates Proof-of-Concept in Psoriatic Arthritis. AbGenomics completed an open-label Phase II trial with
AbGn-168H in 19 patients with PsA. Subjects received AbGn-168H at weeks 0, 1, 2, 4, 6, 8, and 10. The primary endpoint is the
percentage of patients who achieve a 20% reduction in PsA symptoms according to the American College of Rheumatology (ACR20)
scale at 12 weeks. Secondary endpoints include ACR50 and ACR70 at weeks 12 and 24, indicating a 50% and 70% reduction in
symptoms, respectively. Results indicated a potential benefit of AbGn-168H. Eight of 15 patients who completed seven doses achieved
ACR20 or better at week 12.
■ Second Generation AbGn-168H for PsA and Crohn’s Disease. AbGenomics has generated a second generation AbGn-168H
called AbGn-168H2, which provides superior preclinical activity over the first generation antibody. AbGn-168H2 recognizes the same
epitope on PSGL-1 as AbGn-168H but has up to a 10-fold increase in potency in an ex-vivo delayed-type hypersensitivity (DTH)
assay. AbGenomics intends to develop AbGn-168H2 for the treatment of PsA and Crohn’s disease.
■ Next Generation Antibody Drug Conjugates for the Treatment of Solid Tumors. AbGenomics is developing two antibody drug
conjugates (ADCs) for the treatment of gastric, pancreatic, colorectal, and breast cancers. ADCs are monoclonal antibodies that are
conjugated to cytotoxic drugs by chemical linkers. They bind to tumor specific antigens on the cell surface and are internalized by
receptor-mediated endocytosis. The combination of targeted antibodies with cancer killing drugs improves drug delivery to tumors,
and potentially eliminates or reduces off target toxicity associated with traditional chemotherapeutic agents. Once inside the cell, the
ADC complex degrades, releasing a cancer-killing drug, which triggers cell death. AbGenomics expects to file an IND for its lead
oncology product candidate AbGn-107 in the first half of 2016 and expects to initiate a Phase I trial soon thereafter.
Expected Upcoming Milestones
■ H2 2016 – Completion of the Phase Ib trial with AbGn-168H in sr-aGvHD.
■ H2 2016 – Initiate Phase II trial for AbGn-168H in sr-aGvHD.
■ H2 2016 – Initiate Phase I study with AbGn-107 in colorectal, pancreatic and stomach cancers.
Page 2

April 4, 2016
Table of Contents
Company Description ........................................................................................................................................................ 4!
AbGn-168H – A Targeted T-cell Regulatory Agent for Inflammatory Diseases .................................................... 4!
Acute Graft-Versus-Host Disease ................................................................................................................................... 8!
Causes and Pathogenesis .............................................................................................................................................. 8!
Symptoms, Diagnosis and Staging .............................................................................................................................. 9!
Treatment for aGvHD ............................................................................................................................................... 10!
Steroid-Refractory aGvHD – Market Information ................................................................................................ 12!
Steroid Refractory Acute GvHD – Clinical Data Discussion ................................................................................... 15!
Phase Ib Trial with AbGn-168H in Steroid Refractory aGvHD ......................................................................... 15!
Planned Phase II study for AbGn-168H in sr-aGvHD ........................................................................................ 16!
Proof-of-Concept Clinical Data for AbGn-168H in Psoriatic Arthritis .................................................................. 16!
Phase II Study in PsA ................................................................................................................................................. 16!
Proof-of-Concept Clinical Data for AbGn-168H in Plaque Psoriasis ..................................................................... 17!
Phase IIa Trial .............................................................................................................................................................. 18!
Phase II Trial ................................................................................................................................................................ 19!
Other Drugs in Development for Steroid Refractory aGvHD ................................................................................. 20!
Competitive Landscape ................................................................................................................................................... 20!
Antibody Drug Conjugates for the Treatment of Cancer .......................................................................................... 21!
Intellectual Property ......................................................................................................................................................... 24!
Management Team ........................................................................................................................................................... 24!
Risk to an Investment ...................................................................................................................................................... 27!
Analyst Certification ......................................................................................................................................................... 28!
Disclosures ......................................................................................................................................................................... 28!
Page 3

April 4, 2016
Company Description
AbGenomics is a clinical-stage biotechnology company with a pipeline of therapeutic programs for inflammatory
diseases and cancer. The Company’s lead product candidate AbGn-168H (neihulizumab) is being developed for the
treatment of steroid refractory acute graft-versus-host disease (sr-aGvHD). AbGn-168H is currently being evaluated
in a Phase Ib study in sr-aGvHD at Stanford University, with results expected in second half of 2016. AbGenomics
also recently completed a Phase II trial evaluating AbGn-168H in patients with psoriatic arthritis (PsA). The
Company’s preclinical oncology program with next generation antibody drug conjugates (ADC) is targeting solid
tumors. AbGenomics’ developmental pipeline is shown in Figure 1.
Figure 1. AbGenomics’ Developmental Pipeline
Source: LifeSci Capital
AbGn-168H – A Targeted T-cell Regulatory Agent for Inflammatory Diseases
AbGenomics is developing AbGn-168H (neihulizumab) for the treatment of inflammatory conditions including
steroid-refractory acute graft-versus-host disease (sr-aGvHD). Many inflammatory disorders share a common
disease mechanism that centers on the abnormal and chronic activation of T-cells. AbGn-168H is a monoclonal
antibody that binds to the cell surface protein P-selectin glycoprotein ligand-1 (anti-PSGL-1/CD162), and selectively
kills the late-stage activated T-cells that cause severe inflammation. This targeted activity has the potential to reduce
or eliminate tissue damage associated with inflammation without compromising immune system function. Since the
therapy is designed to eliminate the uncontrolled pathogenic T-cells that underlie inflammatory conditions,
treatment with AbGn-168H could result in durable and long-term responses.
Background. PSGL-1 is expressed on the surface of hematopoietic stem cells (HSC), monocytes, neutrophils, and
T cells, and has a dual role in regulating adhesion and immune responses. 1, 2 Studies have shown that PSGL-1 binds
1 McEver, R.P. et al., 1997. Role of PSGL-1 Binding to Selectins in Leukocyte Recruitment. Journal of Clinical Investigation, 100(3),
pp485-492.
Page 4

April 4, 2016
to the selectin family of adhesion molecules, including E-selectin, L-selectin, and P-selectin. These molecules are
expressed on the surface of endothelial cells, and help anchor PSGL-1-expressing cells near sites of tissue
inflammation. Figure 2 shows that the PGSL-1-selectin interaction enables immune cells to attach to the surface of
endothelial cells in blood vessels. Once bound, immune cells transmigrate from the blood into the target tissue,
where they can respond to the inflammation.
Figure 2. PSGL-1 Anchors Immune Cells at Sites of Inflammation
Source: McEver et al., 1997
In addition to regulating adhesion, PSGL-1 molecules may also modulate the activity of T-cells. 3, 4 Studies have
found that T-cells deficient in PSGL-1 show enhanced proliferation, and PSGL-1 deficient animals exhibit
exacerbated colitis and develop a severe form of autoimmune encephalomyelitis. 5,6 Results from these studies
indicate that PSGL-1 acts to dampen T-cell responses, and suggests that agents activating PSGL-1 may be a
potential treatment for T-cell-mediated inflammatory conditions.
Mechanism of Action. AbGn-168H is a humanized monoclonal antibody designed to preferentially eliminate the
T-cells that underlie inflammatory conditions. 7,8 It binds and activates PSGL-1 on late-stage activated T-cells and
2 Levesque, J.P. et al., 1999. PSGL-1-mediated adhesion of human hematopoietic progenitors to P-selectin results in
suppression of hematopoiesis. Immunity, 11(3), pp369-378.
3 Matsumoto, M. et al., 2009. P-Selectin Glycoprotein Ligand-1 Negatively Regulates T-Cell Immune Responses. Journal of
Immunology, 183(11), pp7204-7211.
4 Angiari, S. et al., 2013. Regulatory T Cells Suppress the Late Phase of the Immune Response in Lymph Nodes through P-
Selectin Glycoprotein Ligand-1. Journal of Immunology, 191, pp5489-550.
5 Ostanin, D.V. et al., 2007. T cell-associated CD18 but not CD62L, ICAM-1, or PSGL-1 is required for the induction of
chronic colitis. American Journal of Physiology and Gastrointestinal Liver Physiology, 292(6), pp1706-1714.
6 Frias-Perez, A. et al., 2014. Development of an Autoimmune Syndrome Affecting the Skin and Internal Organs in P-selectin
Glycoprotein Ligand 1 Leukocyte Receptor–Deficient Mice. Arthritis and Rheumatology, 66(11), pp3178-3189.
7 Chen, S. et al., 2004. Cross-linking of P-selectin glycoprotein ligand-1 induces death of activated T cells. Blood, 104, pp3233-
3242.
Page 5

April 4, 2016
induces programmed cell death through the natural mechanism of activation-induced cell death. This death pathway
involves the translocation of the apoptosis-inducing factor (AIF) from the mitochondria to the nucleus, and the
release of cytochrome c. 9 Importantly, AbGn-168H does not impact the activity of resting or memory T-cells, B-
cells, or other immune cells. This feature differentiates AbGn-168H from most other treatments for inflammatory
conditions, including steroids, immunosuppressants, and anti-TNF-α biologics.
The broad immunosuppressive activity of these agents leaves patients susceptible to serious opportunistic infections,
and may lead to an increased risk for certain cancers. AbGn-168H is designed to selectively kill the uncontrolled
pathogenic T-cells that underlie inflammatory diseases without completely compromising immune function. Since
the therapy is designed to eliminate, and not simply inhibit the pathogenic T-cells, treatment with AbGn-168H may
result in durable and long-term responses. These features, combined with its potentially superior safety profile, make
AbGn-168H an attractive product candidate for the treatment of aGvHD, PsA, early onset of type-I diabetes,
Crohn’s disease, and inflammatory bowel disease.
Preclinical Data for AbGn-168H. AbGenomics’ completed a preclinical study with a surrogate hamster antimouse
PSGL-1 antibody (TAB4) in a mouse model of aGvHD. 10 Severe combined immunodeficiency (SCID)
Balb/c mice were transplanted with bone marrow and spleen cells from a C57BL/6 mouse. This resulted in a robust
and lethal graft-vs-host response within weeks of transplantation. Immediately following transplant, 14 SCID mice
received anti-mouse PSGL-1 (TAB4) and 10 received control hamster immunoglobulin (hamster Ig). Figure 3
shows that all animals infused with control Ig died from aGvHD by day 35. In contrast, more than 50% of animals
treated with TAB4 survived through day 50, and 30% of these mice were still alive through day 100.
Figure 3. Survival Percentage of Mice Treated with AbGn-168H Following Cell Transplant
Source: Huang, C.C. et al., 2005
8 Huang, C. et al., 2005. A novel apoptosis-inducing anti-PSGL-1 antibody for T cell-mediated diseases. European Journal of
Immunology, 35, pp2239-2249.
9 Chen, S. et al., 2004. Cross-linking of P-selectin glycoprotein ligand-1 induces death of activated T cells. Blood, 104, pp3233-
3242.
10 Huang, C. et al., 2005. A novel apoptosis-inducing anti-PSGL-1 antibody for T cell-mediated diseases. European Journal of
Immunology, 35, pp2239-2249.
Page 6

April 4, 2016
To determine the durability of response, the skin, liver, and intestines of mice that had long-term survival over one
year were histopathologically examined. Results showed no signs of residual aGvHD, indicating a durable and long
term response. Treated mice had normal donor T-cell populations, demonstrating that TAB-4 selectively eliminated
only activated T-cells. Results from this study indicate that AbGn-168H has the potential to treat patients with
aGvHD, and other condition that involve acute or chronic T-cell mediated inflammation.
AbGn-168H Safety Profile. AbGn-168H has been studied in three clinical trials and more than 150 subjects.
AbGn-168H was generally safe and well tolerated. The most common adverse events associated with the drug have
been mild fatigue and headache.
Preclinical Data for AbGn-168H2. AbGenomics has generated a second generation AbGn-168H called AbGn-
168H2, which provides superior activity over the first generation antibody. AbGn-168H2 recognizes the same
epitope on PSGL-1 as AbGn-168H, and has a similar safety profile in preclinical models. Figure 4 below shows that
AbGn-168H2 has up to a 10-fold increase in potency compared to AbGn-168H in an ex-vivo delayed-type
hypersensitivity (DTH) assay. These data indicate that the second generation antibody may be a bio-better version of
AbGn-168H. AbGenomics intends to develop AbGn-168H2 as a treatment for PsA, early onset of type-I diabetes,
Crohn’s disease, and inflammatory bowel disease.
!
Figure 4. Performance of AbGn-168H and AbGn-168H2 in a Delayed-Type Hypersensitivity Assay
!
Source: Company Presentation
Page 7

April 4, 2016
Acute Graft-Versus-Host Disease
Acute graft-versus-host disease (aGvHD) is a potentially life threatening complication of donor-derived
hematopoietic cell after an allogeneic bone marrow transplant (alloHCT). alloHCT is used to treat blood cancers and
anemia, and involves the reconstitution of a patient’s immune system with cells from a healthy donor. aGvHD arises
when transplanted donor white blood cells called T lymphocytes (T-cells) begin attacking the patient’s tissues,
causing inflammation and organ damage. According to the Center for International Blood and Marrow Transplant
Research (CIBMTR), approximately 7,500 patients undergo alloHCT in the US each year 11 and approximately 60%
are expected to develop aGvHD. 12 First-line treatment for aGvHD is steroids, however less than 50% of patients
will achieve durable responses with this therapy. 13 Steroid refractory aGvHD (sr-aGvHD) remains a major cause of
alloHCT-related morbidity, and overall mortality estimates are as high as 95%. 14,15,16 Approximately 70% of aGvHDrelated
deaths result from ongoing and uncontrolled aGvHD, and 11-30% are due to severe opportunistic
infections. 17 There is a high unmet medical need for a treatment such as AbGn-168H that protects against a graft-vshost
reaction while maintaining robust immune function that is capable of combating infections. Since the therapy is
designed to eliminate, and not simply inhibit the pathogenic T-cells that cause aGvHD, treatment with AbGn-168H
could result in durable and long-term responses. These features, combined with its potential superior safety profile,
make AbGn-168H an appealing product candidate for the treatment of aGvHD.
Causes and Pathogenesis
aGvHD occurs following an alloHCT when donor T-cells attack the patient’s organ systems. The immune attack
causes severe and potentially life-threatening organ damage. The condition progresses in three phases, 18 and we
discuss each phase in detail below.
! Damage Phase – Patients receiving an alloHCT are treated with a conditioning regimen, which is
chemotherapy with or without radiotherapy. The treatment kills cancer cells and creates physical space in the
bone marrow to accommodate the transplanted donor cells. However, chemotherapeutic agents are toxic to all
cells within the body, so these conditioning regimes also damage healthy tissues and organs. The damage leads
to the systemic release of inflammatory cytokines including as TNF-α and IL-1, which increase the expression
of human leukocyte antigen (HLA) proteins and adhesion molecules on the patient’s antigen-presenting cells
(APCs).
11 CIBMTR,
12 Jagasia, M. et al. 2012. Risk factors for acute GvHD and survival after hematopoietic cell transplantation. Blood, 119(1),
pp296-307.
13 Arai, S. et al., 2002. Poor outcome in steroid refractory graft-versus-host disease with antithymocyte globulin treatment. Biology
of Bone and Marrow Transplantation, 8, pp155-160.
14 Bolanos-Meade, J. et al., 2001. Outcome of 21 patients undergoing unrelated bone marrow transplant for hematologic
malignancies at a single institution. Blood, 98, 382B.
15 Arai, S. et al., 2000. Management of graft-versus-host disease. Blood Reviews, 14, pp190-204.
16 Westin, J.R., et al. 2011. Steroid-Refractory Acute GvHD: Predictors and Outcomes. Advances in Hematology, 2011, pp1-8
17 Gratwohl, A. et al., 2005. Cause of death after allogeneic hematopoietic stem cell transplantation (HSCT) in early leukemia: an
EBMT analysis of lethal infectious complications and changes over calendar time. Bone Marrow Transplantation. 36(9), pp757-769.
18 Ball, L. and Egeler, R. 2008. Acute GVHD: pathogenesis and classification. Bone Marrow Transplantation, 41(2), S58-S64.
Page 8

April 4, 2016
! T-cell Activation Phase – After treatment with chemotherapy, donor bone marrow cells, including T-cells, are
transplanted into the patients. These cells enter into the patient’s circulation and will interact with APCs. If the
donor cells and patient tissues do not express identical HLA proteins on their cell surfaces, the donor T-cells
will recognize the patient’s tissues as foreign, become activated and launch the graft-versus-host reaction.
! Effector Phase – The exact mechanism of organ damage is not known but the activated donor T cells circulate
throughout the patient’s body and tend to target the patient’s skin, liver, and gut cells. Activated T cells secrete
cytokines that recruit additional immune cells such as natural killer cells and large granular monocytes, which
also kill healthy cells and contribute to overall organ damage.
Symptoms, Diagnosis and Staging
There is no definitive test for aGvHD but a positive diagnosis can be made based on patient symptoms. 19 Symptoms
of aGvHD typically occur within 100 days post-alloHCT, and most commonly involve the skin, liver, and
gastrointestinal tract. Usually the first sign of disease is a skin rash on the neck, ears, shoulders, palms, or feet. The
rash can be followed by upper and lower gastrointestinal track symptoms, which include bloody diarrhea, abdominal
pain, nausea, and vomiting. These features together with elevated levels of the liver biomarker bilirubin suggest a
graft-versus-host reaction, and would lead clinicians to suspect aGvHD. A biopsy of the involved organs can be
used to confirm disease and rule out other non-aGvHD complications.
The Keystone aGvHD Clinical Grade Scale was established in 1994 and is widely used in the clinic today to
characterize the severity of aGvHD. 20 In this system, the severity of disease corresponds directly to the degree of
organ involvement. The top half of Figure 5 shows the four stages of disease for each of the three most commonly
affected organs – the skin, liver, and gastrointestinal track. Organ staging is combined with the patient’s
performance to produce an overall grade, which helps clinicians determine prognosis and select appropriate
treatment options. Patients with a Grade II-IV moderate-to-severe aGvHD have a significantly higher mortality rate
than those with milder Grade I disease. Estimated five-year survival rates of patients with Grade III and Grade IV
disease are 25% and 5%, respectively. 21
19 Firoz, B.F. et al., 2006. Role of skin biopsy to confirm suspected acute graft-vs-host disease: results of decision analysis.
Archives of Dermatology, 142(2), pp175.
20 Przepioka, D. et al., 1995. 1994 Consensus Conference on Acute GvHD Grading. Bone Marrow Transplantation, 15, pp825-864.
21 Cahn, J.Y. et al., 2005. Prospective evaluation of 2 acute graft-versus-host (GVHD) grading systems: a joint Société Française
de Greffe de Moëlle et Thérapie Cellulaire (SFGM-TC), Dana Farber Cancer Institute (DFCI), and International Bone Marrow
Transplant Registry (IBMTR) prospective study. Blood, 106(4), p1495.
Page 9

April 4, 2016
Figure 5. Keystone Clinical Grade Scale
Stage Skin Liver GI Tract
1
Rash 500 ml/day
2
Rash ≥25-50% of body
surface area
Bilirubin 3.1-6 mg/dL
Diarrhea >1000 ml/day
3
Rash on > 50% of body
surface area
Bilirubin 6.1-15 mg/dL
Diarrhea >1,500 ml/day
4
Generalized erythroderma
with bullous formation
Bilirubin >15 mg/dL
Diarrhea >1,500 ml/day
or severe abdominal pain
Grade
I Stage 1-2 None None
II Stage 3 or Stage 1 or Stage 1
III N/A Stage 2-3 or Stage 2-4
IV Stage 4 or Stage 4 N/A
Source: Przepiorka, D. et al., 1995
It is worth noting that patients with the same Grade but different patterns of organ involvement often have
significantly different outcomes. For example, a patient with Stage 4 skin aGvHD would be expected to have a
much more favorable outcome than a patient with Stage 4 liver aGvHD, although both have overall Grade IV
disease.
Treatment for aGvHD
Prophylactic treatments are used to prevent aGvHD but approximately 60% of alloHCT patients will still develop
disease. There is not an agreed upon prophylactic therapy, however most treatments combine methotrexate (MTX)
with a calcineurin inhibitor (CNI) such as tacrolimus or cyclosporine A. When prophylaxis fails, clinicians are
presented with few treatment options to reverse disease progression. Below we discuss the therapies for Grade I,
Grades II-IV, and steroid refractory disease. We also note the potential benefits of mild aGvHD on cancer
eradication and highlight the secondary risks associated with all treatments options.
Treatment for Grade I aGvHD. Patients with Grade I disease present with rash over ≤ 50% of their body, and
show no signs of liver or gastrointestinal involvement. First-line treatment for this form of the disease usually
Page 10

April 4, 2016
involves topical steroids. For the approximately 60-75% of patients with Grade I who become refractory to steroid
treatment, a topical formulation of the CNI tacrolimus has been used as a second-line therapy. 22
Treatment for Grades II-IV aGvHD. First-line treatment for Grades II-IV aGvHD is off label use of steroids, 23
which have been shown to be superior to immunosuppressants including cyclosporine A and anti-thymocyte
globulin (ATG). 24,25 Initial treatment is usually systemic glucocorticoids, such as methylprednisolone. Approximately
25%-40% of patients receiving methylprednisolone will have a complete response, defined as the complete
resolution of aGvHD symptoms in all affected organs. 26, 27 It is estimated that 40-50% of Grades II-IV patients will
experience improvement in symptoms, defined as resolution of skin rashes, and a reduction in gastrointestinal and
liver symptoms. 28, 29 We note that response rates decrease as the severity of disease increases, with grade IV patients
having the lowest overall response rate. Patients who do not respond to steroids seven days following treatment are
diagnosed with sr-aGvHD.
sr-aGvHD Treatments. There are no approved therapies for sr-aGvHD, highlighting a significant unmet medical
need for these patients. Current treatment options include a variety of immunosuppressive agents and experimental
therapies. The most widely used agents for sr-aGvHD include antithymocyte globulin (ATG) and TNF-α inhibitors.
Approximately 20-50% of patients respond to ATG therapy, 30 however less than 20% achieve durable responses.
ATG treatment can cause severe side effects, including acute febrile reactions, hypotension, thrombocytopenia, and,
in rare instances, the development of post-transplantation lymphoproliferative disorders, 31 so there is a need for
safer and more durable treatment options.
Other immunosuppressants available for the treatment of sr-aGvHD include TNF-α inhibitors, as well as anti-IL-2
receptor–, anti-IL6 receptor–, anti-CD20–, and anti-CD52–targeted therapies. These agents have not been
rigorously tested in clinical trials, making it very hard to determine efficacy. There is also a safety risk with these
therapies, since they broadly suppress immune function and leave patients susceptible to opportunistic bacterial and
viral infections. Unlike the therapies we highlighted in this section, AbGn-168H may eventually be a treatment that
is both effective and safe for sr-aGvHD patients.
22 Antin, J. et al., 2004. Novel approaches to the therapy of steroid-resistant acute graft-versus-host disease. Biology of Blood and
Marrow Transplantation, 10(10), pp655-668.
23 Martin, P.J. et al., 2012. First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the
American Society of Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation. 18(8), pp1150-1163.
24 Martin, P.J. et al., 1991. A retrospective analysis of therapy for acute graft-versus-host disease: secondary treatment. Blood, 77,
pp1821- 1828.
25 Martin, P.J. et al., 1990. A retrospective analysis of therapy for acute graft-versus-host disease: initial treatment. Blood, 76,
pp1464-1472.
26 Hings, I.M. et al., 1994. Treatment of moderate and severe acute GVHD after allogeneic bone marrow transplantation.
Transplantation, 58(4), pp437.
27 Lee, S.J. et al., 2004. Effect of up-front daclizumab when combined with steroids for the treatment of acute graft-versus-host
disease: results of a randomized trial. Blood, 104(5), pp1559.
28 Chao, N. et al., 2015. Treatment of acute graft-versus-host disease. Wolters Kluwer.
29 Deeg, J., 2007. How I treat refractory acute GVHD. Blood. 109(10), pp4119-4126.
30 Doney, K. et al., 1985. A randomized trial of antihuman thymocyte globulin versus murine monoclonal antihuman T-cell
antibodies as immunosuppressive therapy for aplastic anemia. Experimental Hematology. 13(6), pp520-524.
31 Curtis, R.E. et al., 1999. Risk of lymphoproliferative disorders after bone marrow transplantation: a multi-institutional study.
Blood, 94(7), pp2208-2216.
Page 11

April 4, 2016
One important potential advantage of AbGn-168H over other therapies including ATG, TNF-α inhibitors, and
biologics is that AbGn-168H is a targeted T-cell regulatory agent that does not induce broad immunosuppression. It
has the potential to treat sr-aGvHD without increasing the risk of serious infection. Since the therapy is designed to
eliminate, and not simply inhibit the pathogenic T-cells, treatment with AbGn-168H could result in durable and
long-term responses. These features, combined with its potentially superior safety profile compared to ATG, TNFα
inhibitors and the biologics, make AbGn-168H an attractive product candidate for the treatment of aGvHD and
PsA.
!
Risks Associated with Current aGvHD Treatment. The majority of treatment options for aGvHD include
immunosuppressants. These drugs uniformly suppress the patient’s immune system, which makes them more
susceptible to severe infections and infection-related deaths. Antimicrobial and antiviral prophylaxis treatments are
administered to manage infections and mitigate infections, however these therapies are not always effective. A large
retrospective study compared infection rates following alloHCT in 14,403 patients between 1980-2001. 32 Results
showed that 11% (597/5,377) of deaths were due to infections. The proportion of deaths associated with each
infectious agent is shown in Figure 6. The median time of death due to infection following alloHCT was
determined to be 3 months, with a range of 0 months to 158 months. These data highlight the need for better
treatment options that retain general immune system function while selectively blocking graft-vs-host reactions.
AbGn-168H may eventually fill this role by providing a treatment that is both effective and safe for sr-aGvHD.
Figure 6. Lethal Infections Following alloHCT
Infectious Agent
Proportion of Total Deaths
Bacteria 36% (217/597)
Virus 31% (183/597)
Fungi 28% (166/597)
Parasite 5% (32/597)
Source: LifeSci Capital
Steroid-Refractory aGvHD – Market Information
The number of alloHCTs has grown steadily since 1980. There were roughly 27,078 transplants performed
worldwide in 2010, and 7,554 in the US in 2012. 33,34 Figure 7 shows the number of alloHCTs in the US by
indication in 2012. The vast majority of alloHCT are performed in patients with blood cancers.
32 Gratwohl, A. et al., 2005. Cause of death after allogeneic hematopoietic stem cell transplantation (HSCT) in early leukemia: an
EBMT analysis of lethal infectious complications and changes over calendar time. Bone Marrow Transplantation. 36(9), pp757-769.
33CIBMTR. 2014. Current uses and outcomes of hematopoietic stem cell transplantation, 2014 summary slides.
34 http://www.wbmt.org/fileadmin/pdf/01_General/Task_force_Slide_set_2010_July2015-ATT_C2_HB.pdf
Page 12

April 4, 2016
Figure 7. Hematopoietic Stem Cell Transplants by Indication in 2012
Source: Center for International Blood & Marrow Transplant Research
Figure 8 shows that there were roughly 7,500 alloHCTs performed in the US in 2012. 35,36 Approximately 60% of
patients receiving alloHCT are expected to develop aGvHD, 37 indicating that that are approximately 4,500 cases in
the US each year. 60% of these cases will be refractory to steroid treatments, meaning that there are approximately
2,700 patients with steroid refractory aGvHD in the US each year that would be eligible for treatment with AbGn-
168H.
Figure 8. AbGn-168H’s US Addressable Patient Population in 2015
Percent of
Patients
Number of
Patients
Allogeneic HCTs 7,500
% of patients with aGvHD 60% 4,500
% of steroid refractory patients 60% 2,700
Patients with steroid refractory aGvHD 2,700
Source: LifeSci Capital
Using the same approach as above, we highlight the potential worldwide target population for AbGn-168H. Figure
9 shows that were roughly 27,000 alloHCT in 2012. 38,39 We estimate that there are roughly 9,700 cases of sr-aGvHD
each year that would be eligible for treatment with AbGn-168H
35 CIBMTR. 2014. Current uses and outcomes of hematopoietic stem cell transplantation, 2014 summary slides.
36 http://www.wbmt.org/fileadmin/pdf/01_General/Task_force_Slide_set_2010_July2015-ATT_C2_HB.pdf
37 Won Choi, S. & Reddy, P. 2014. Current and emerging strategies for the prevention of graft versus host disease. Nature
Reviews Clinical Oncology, 11(9), pp536-547.
38 CIBMTR. 2014. Current uses and outcomes of hematopoietic stem cell transplantation, 2014 summary slides.
39 http://www.wbmt.org/fileadmin/pdf/01_General/Task_force_Slide_set_2010_July2015-ATT_C2_HB.pdf
Page 13

April 4, 2016
Figure 9. AbGn-168H’s Worldwide Addressable Patient Population in 2015
Percent of
Patients
Number of
Patients
Allogeneic HCTs 27,000
% of patients with aGvHD 60% 16,200
% of steroid refractory patients 60% 9,700
Patients with steroid refractory aGvHD 9,700
Source: LifeSci Capital
AbGn-168H’s Market Potential. We conducted a scenario analysis to estimate the US market potential for AbGn-
168H in sr-aGvHD. The analysis is based on several assumptions listed below:
! Addressable Population – We used similar assumptions as detailed in Figure 8 above to calculate the
addressable population of patients who may be candidates for AbGn-168H. There were approximately
2,700 affected individuals in the US with steroid refractory aGvHD in 2012.
! Market Penetration – The market penetration for an approved therapy in an indication with such a poor
prognosis is likely to be high, and we estimate a peak penetration range of 70% by 2023. The transplant
market is also highly concentrated, with the top 15 centers performing approximately 40% of all AML
transplants. This makes commercialization straightforward and increases the chances for high penetration.
! Pricing – We are assuming a price per year of $200,000, which is in line with the current price of approved
therapies for orphan diseases.
Figure 10 shows potential US sales of AbGn-168H based on the assumptions listed above. We estimate product
launch in 2019 at an initial market penetration of 30%, and assume peak market penetration of 70% by 2023. This
scenario yields estimated peak sales of $380 million. Given the lack of safe and effective therapies for sr-aGvHD, it
may be possible for AbGn-168H to capture greater than 70% of the market.
Figure 10. Scenario Analysis of US Market Potential for AbGn-168H
2019 2020 2021 2022 2023
Market Penetration 30% 40% 50% 60% 70%
Treated Patients 816 1,088 1,360 1,631 1,903
Price Per Patient $200,000
Yearly Sales $162 M $218 M $274 M $326 M $380 M
Source: LifeSci Capital
Figure 11 shows potential worldwide sales of AbGn-168H based on the assumptions listed above. We estimate
product launch in major markets in 2019 at an initial market penetration of 10%, and assume peak market
penetration of 50% by 2023. This scenario yields estimated peak sales of $970 million. Given the lack of safe and
effective therapies for sr-aGvHD, it may be possible for AbGn-168H to capture greater than 50% of the market.
Page 14

April 4, 2016
Figure 11. Scenario Analysis of Worldwide Market Potential for AbGn-168H
2019 2020 2021 2022 2023
Market Penetration 10% 20% 30% 40% 50%
Treated Patients 970 1,940 2,910 3,880 4,850
Price Per Patient $200,000
Yearly Sales $194 M $388 M $582 M $776 M $970 M
Source: LifeSci Capital
Steroid Refractory Acute GvHD – Clinical Data Discussion
AbGenomics is conducting a Phase Ib trial with AbGn-168H in patients with sr-aGvHD. In this section, we provide
the details of the study, and highlight the preliminary results. At the time of this report, 2 patients have received
AbGn-168H and both had reductions in the severity of aGvHD. These early encouraging results suggest that
AbGn-168H has the potential to be a safe and effective treatment for patients with sr-aGvHD. Following the
completion of the Phase Ib study, the Company plans to initiate a randomized, open-label Phase II study in 2016.
Phase Ib Trial with AbGn-168H in Steroid Refractory aGvHD
Trial Design. AbGenomics is conducting an open-label, dose-escalation Phase Ib trial with AbGn-168H in patients
with steroid refractory aGvHD. 40 This trial is being conducted at Stanford University Medical School’s transplant
center, and will enroll 12 patients. Subjects will be placed in one of four dosing cohorts, and will receive AbGn-
168H once a week for 4 weeks following sr-aGvHD diagnosis. The first patient will receive 6 mg/kg of AbGn-
168H, and subsequent patients will either be dose-escalated or de-escalated depending on the safety and tolerability
of AbGn-168H treatment. All patients will remain on the standard of care, which are steroids with or without
immunosuppressants. The primary endpoint of the trial is the dose-limited toxicity as assessed by:
! Grade 3-5 adverse events considered at least possibly-related to AbGn-168H occurring after the
first dose of AbGn-168H and within 30 days after the 4 th dose.
! Grade 3-5 cytokine release syndrome or acute infusion reaction within 24 hours after AbGn-168H
infusion.
! Grade 4 neutropenia considered at least possibly related to AbGn-168H, lasting more than 14 days.
! Grade 5 all-cause mortality with 7 days of infusion.
The secondary endpoints designed to assess efficacy are:
! The response to therapy at day 28 following treatment.
! The response to therapy at 3 months after diagnosis of aGvHD, as measured by the aGvHD
grading scale as defined in the protocol.
40 https://clinicaltrials.gov/ct2/show/NCT02436460
Page 15

April 4, 2016
The scale measures the grade of aGvHD based on skin, liver, and gastrointestinal track symptoms and is discussed
in the Symptoms, Diagnosis and Staging section of this report.
Preliminary Trial Data. As of the writing of this report, two patients with sr-aGvHD have been treated with
AbGn-168H. The patients were initially administered 6 mg/kg of AbGn-168H at the start of therapy. A reduction
from Grade II disease to Grade 0 was achieved in one of the patients after the first week of treatment. Dosing was
de-escalated to 3 mg/kg at week 3 after the patient experienced septic shock. Importantly, the study investigator
indicated that the sepsis was not AbGn-168H-related, and was likely due to the immunosuppressant that the patient
was receiving as part of the standard of care. The GvHD remained under controlled and the subject was
subsequently released from the hospital. The second patient treated with AbGn-168H experienced a reduction from
Grade IV disease to Grade II by week 4. These results are preliminary, and support the possibility of AbGn-168H as
a safe and effective therapy for the treatment of sr-aGvHD.
Planned Phase II study for AbGn-168H in sr-aGvHD
Trial Design. AbGenomics plans to initiate a randomized, open-label, Phase II trial for AbGn-168H in patients
with sr-aGvHD. The study will take place at 40 centers across the US, including Stanford Medical Center, The Fred
Hutchinson Cancer Center, MD Anderson Cancer Center, and Memorial Sloan Kettering Cancer Center. At least 90
subjects will be randomized to receive AbGn-168H or best supportive care. The primary endpoint is overall
response at Day 28, which is defined a complete response (CR) or partial response (PR). Secondary endpoints
include overall response rate at Day 90, as well as overall survival at Day 90 and Day 180. The Company plans to
initiate the study in the second half of 2016.
Proof-of-Concept Clinical Data for AbGn-168H in Psoriatic Arthritis
AbGenomics has completed enrollment in an open-label Phase II trial with AbGn-168H in patients with psoriatic
arthritis (PsA). In this section, we provide the details of the study, and highlight the results. Upon the completion of
the study, the Company does not intend to further develop AbGn-168H in this indication. Instead AbGenomics will
develop its second generation anti-PSGL-1/CD162 AbGn-168H2 for this indication.!
Phase II Study in PsA
Phase II Study Design. AbGenomics completed an open-label Phase II trial with AbGn-168H in patients with
PsA. 41 20 subjects received AbGn-168H at weeks 0, 1, 2, 4, 6, 8, and 10. The primary endpoint is the percentage of
patients that achieve a 20% reduction in PsA symptoms according to the American College of Rheumatology
(ACR20) at 12 weeks. Secondary endpoints include ACR20, ACR50 and ACR70 at different time points.
Trial Results. Results for 8 of the 15 patients that completed all seven doses of treatment are discussed below.
Figure 12 shows data from the 15 patients that completed all seven treatments. Week 12 results indicate that 53.3%
of patients achieved ACR20 or better. 40% of patients in the intent to population achieved an ACR20 at week 12.
41 https://clinicaltrials.gov/ct2/show/NCT02267642
Page 16

April 4, 2016
Five patients did not complete the study and were not included in the analysis. Five responders were previously
treated with either TNF-α or anti-IL-17, suggesting there is a potential niche for AbGn-168H in this patient
population. We note that a more potent T-cell regulatory agent such as the second generation AbGn-168H2 may
provide even more benefit to patients with PsA.
Figure 12. Response Rates with AbGn-168H in PsA
Figure 13 compares the response rates for AbGn-168H with brodalumab, ustekinumab and secukinumab. The
percent response at week 12 in the intent to treat population is comparable to the Phase II results for the other
biologics listed.
Figure 13. Response Rates with AbGn-168H in PsA
Week 12
AbGn-168H
(% ITT)
Brodalumab
(% ITT)
Ustekinumab
(% ITT)
Secukinumab
(% ITT)
ACR20 40% 39% 42% 39%
ACR50 30% 14% 25% na
ACR70 10% 5% 11% na
Source: Company Presentation
Proof-of-Concept Clinical Data for AbGn-168H in Plaque Psoriasis
AbGenomics has completed two Phase II trials with AbGn-168H in patients with moderate-to-severe plaque
psoriasis. The Company does not have plans to development AbGn-168H for this indication, so the results
discussed below should be viewed as proof-of-concept data. There were no serious adverse events recorded in over
150 treated patients. A reduction in the Psoriasis Activity Score Index (PASI) was observed in the treatment group,
however the studies did not meet the primary endpoints. Below we describe the design and results of both trials.
Page 17

April 4, 2016
Phase IIa Trial
Trial Design. AbGenomics completed a randomized, double-blind, placebo-controlled Phase IIa trial with AbGn-
168H in patients with moderate to severe plaque psoriasis. 42 The objectives of the study were to investigate efficacy,
safety, tolerability, and pharmacokinetics (PK) of multiple doses of AbGn-168H. Fifty-four patients were
randomized 1:1:1 to receive AbGn-168H 0.5 mg/kg, AbGn-168H 3 mg/kg, or placebo. Patients were administered
AbGn-168H once a week for 6 weeks. The primary endpoint was PASI75 at week 12. PASI75 is the most
commonly used endpoint for psoriasis trials, and indicates a 75% reduction in plaque psoriasis symptoms.
Trial Results. Seventeen patients in the AbGn-168H 0.5 mg/kg group, 17 patients in the AbGn-168H 0.3 mg/kg
group, as well as 16 patients in the placebo group were analyzed for efficacy (Per Protocol Set). The primary
endpoint of the study was not met, however a greater percentage of patients receiving AbGn-168H achieved
PASI50 compared to the patients receiving placebo. PASI50 refers to a 50% reduction in plaque psoriasis
symptoms. Figure 14 shows that 35.3% of patients in the AbGn-168H 3 mg/kg cohort had achieved PASI50 at
week 12 compared to 18.8% in the placebo group. The difference remained at week 16.
Figure 14. PASI50 after Treatment with AbGn-168H or Placebo
Source: Company Presentation
Safety. Nineteen patients in the AbGn-168H 0.5 mg/kg group, 18 patients in the AbGn-168H 0.3 mg/kg group, as
well as 17 patients in the placebo group were analyzed for safety (Treatment Set). The mean study drug compliance
was 86% in the 0.5 mg/kg group, 93% in the 3 mg/kg group, and 99% in the placebo group. There were treatment
emergent adverse events in 68.4% of the AbGn-168H 0.5 mg/kg group, 61.1% of the AbGn-168H 3 mg/kg group,
and 23.5% of the placebo group. The majority of these events were mild or moderate. 15.8% (3/19) of patients in
the AbGn-168H 0.5 mg/kg group, 38.9% (7/18) in the AbGn-168H 3 mg/kg group developed low titer anti-drug
42 https://clinicaltrials.gov/ct2/show/NCT02223039
Page 18

April 4, 2016
antibodies (≤625) sometime during the study.
Phase II Trial
Trial Design. AbGenomics completed a randomized, double-blind, placebo-controlled Phase II trial with AbGn-
168H in patients with moderate to severe plaque psoriasis. 43 Patients were randomized 1:1:1 to receive placebo,
AbGn-168H 6 mg/kg, or AbGn-168H 9 mg/kg. Patients were administered of AbGn-168H on weeks 0, 1, 4, and 5.
The trial enrolled a total of 50 subjects and 48 completed treatment. The primary efficacy endpoint was the
proportion of patients achieving PASI75 at week 10. This study was completed in 2015.
Trial Results. Seventeen patients in the AbGn-168H 6 mg/kg group, 16 patients in the AbGn-168H 9 mg/kg
group, as well as 15 patients in the placebo group were included in efficacy analysis (Per Protocol Set). The primary
endpoint of the study was not met, however a greater percentage of patients receiving AbGn-168H achieved
PASI50 compared placebo at several time points. PASI50 refers to a 50% reduction in plaque psoriasis symptoms.
Analysis of median PASI score, median percent PASI change from baseline, DLQI, and pain VAS further suggested
clinical efficacy of AbGn-168H as a therapy for psoriasis and possibly psoriatic arthritis. Figure 15 shows that
43.8% of patients in the AbGn-168H (AbGn-168H) 9 mg/kg group achieved PASI50 at week 8 compared to 13.3%
for placebo (p=0.0013).
Figure 15. PASI50 After Treatment with AbGn-168H or Placebo
Source: Company Presentation
Safety. 58.8% of the 6 mg/kg group, 56.3% of the 9 mg/kg group, and 23.5% of the placebo group reported a
treatment-emergent AE. The most common AEs in the treatment group were headache, fatigue, vomiting, upper
43 https://clinicaltrials.gov/ct2/show/NCT01855880
Page 19

April 4, 2016
respiratory tract infection, and sinus congestion. 23.5% (4/17) of patients in the 6 mg/kg group, and 31.2% (5/16)
of patients in the 9 mg/kg group developed anti-drug antibodies sometime during the study. The first sign of ADA
occurred at week 12. The ADA titer was generally low (≤3125), most of them became negative at End of Study
(week 20), with the exception of 1 subject in the 9 mg/kg group who had an ADA titer of 15265 at Week 20.
Other Drugs in Development for Steroid Refractory aGvHD
We have identified three late stage products in development for steroid refractory aGvHD, and they are listed in
Figure 16. Jazz Pharmaceutical (NasdaqGS: JAZZ) has completed a Phase III trial for Leukotac (inolimomab) in
Europe, and expects to begin analyzing the data in the second half of 2015. Adienne Pharmaceutical (private)
announced promising Phase II data with Begedina (begelomab) for the treatment of steroid refractory aGvHD, and
expects to initiate a US Phase III trial during the first half of 2016. Mesoblast’s (ASX: MSB) Prochymal (remestemcel-
L) is currently being tested in a confirmatory US Phase III trial, and results are expected in mid 2016. 44 Prochymal has
received approval in Japan, and conditional approval in Canada and New Zealand for the treatment of aGvHD in
children. Commercial launch of Prochymal is expected following approval in a major market.
Figure 16. Late Stage Product Candidates for Steroid Refractory aGvHD
Company Drug Mechanism Development Phase
Jazz Pharmaceuticals
(NasdaqGS: JAZZ)
Leukotac (inolimomab)
Interleukin-2 receptor
antagonist
Phase III in Europe
Adienne Pharmaceuticals (private)
Begedina (begelomab)
Dipeptidyl peptidase-4
(CD26)
antagonist
Planned Phase III
Mesoblast (ASX: MSB)
Prochymal
(remestemcel-L)
Cell therapy designed
to secrete antiinflammatory
cytokines
Phase III
Source: LifeSci Capital
Competitive Landscape
Although an alloHCT can be curative for patients with hematopoietic malignancies and diseases, the 5-year overall
survival following the procedure is only roughly 52%, 45 with one of the major causes of mortality following alloHCT
being GvHD. There are several promising agents being studied as prophylactic treatments for aGvHD such as
Velcade (bortezomib) combined with tacrolimus, Zolina (vorinostat), and Vidaza (azacitidine). 46,47,48 These treatments
44 https://clinicaltrials.gov/ct2/show/NCT02336230
45 O’Meara, A. et al., 2014. Forty years of hematopoietic stem cell transplantation: a review of the Basel experience. Swiss Medical
Weekly, 144, w13928.
46 https://clinicaltrials.gov/ct2/show/NCT00369226
Page 20

April 4, 2016
have the potential to decrease the incidence of aGvHD, and improve patient outcomes. However, severe forms of
aGvHD, including those that are steroid refractory, have been and will continue to be very difficult to treat despite
these promising prophylactic options.
The 2-year overall survival for sr-aGvHD is 30%, and the 5-year overall survival for Grade IV patients is less than
5%. 49,50 AbGenomics’ AbGn-168H is one of the few treatments in development intended to treat patients with sraGvHD.
Unlike the other broad immunosuppressant treatments in development from Jazz Pharmaceuticals and
Adienne Pharmaceuticals, AbGn-168H is a targeted T-cell regulatory agent that has the potential to be a safe and an
effective treatment option, with durable, long-term efficacy.
Antibody Drug Conjugates for the Treatment of Cancer
AbGenomics is developing two antibody drug conjugates (ADCs) for the treatment of gastric, pancreatic, colorectal,
and breast cancers. ADCs are monoclonal antibodies that are conjugated to drugs by chemical linkers. They bind to
tumor specific antigens on the tumor cell surface, and are internalized by receptor-mediated endocytosis. The
combination of targeted antibodies with cancer killing drugs improves drug delivery to tumors, and potentially
eliminates or reduces off target toxicity associated with traditional chemotherapeutic agents. Once inside the cell, the
ADC complex degrades, releasing a cancer-killing drug, which triggers cell death. AbGenomics expects to file an
IND for its lead oncology product candidate AbGn-107 in the first half of 2016, and expects to initiate a Phase I
trial soon thereafter.
AbGn-107 for the Treatment of Gastric, Pancreatic, and Colorectal Cancers. AbGenomics is developing
AbGn-107 as a bio-superior treatment for patients with gastric, pancreatic, or colorectal cancers. This ADC targets
the Lewis (Le a )-like antigen, which is a glycoepitope expressed on the surface of many cancer cells. AbGn-107 is a
humanized version of a chimeric antibody AbGn-7 that was previously in clinical development. 51 Preclinical data
suggest that AbGn-107 is more potent than AbGn-7 in treating cancers expressing Le a -like glycoepitope.
AbGenomics estimates that AbGn-107 binds 50% of stomach and pancreatic tumors, and approximately 25% of
colorectal cancers.
AbGn-107 is a humanized version of a chimeric antibody AbGn-7, which was previously in clinical development. 52
AbGn-7 was evaluated in an open-label, dose-escalating Phase I trial in patients with chemo-refractory advanced
solid tumors. A total of 13 patients received treatment, and AbGn-7 1 mg/kg and AbGn-7 3 mg/kg were evaluated.
The primary endpoint of the study was safety and tolerability. 3 of 5 evaluable patients receiving AbGn-7 1 mg/kg
had stable disease, and 2 had progressive disease. 1 of 5 patients in the AbGn-7 3 mg/kg cohort completed the
study, and had stable disease. One patient in the AbGn-7 1 mg/kg cohort had a progression-free survival of 225
days, suggesting possible efficacy of AbGn-7.
47 https://clinicaltrials.gov/ct2/show/results/NCT00810602
48 Goodyear, O.C. et al., 2012. Azacitidine augments expansion of regulatory T cells after allogeneic stem cell transplantation in
patients with acute myeloid leukemia (AML). Blood, 119(14), pp3361-3369.
49 Xhaard, A. et al., 2012. Steroid-refractory acute GVHD: lack of long-term improved survival using new generation anticytokine
treatment. American Society for Blood and Marrow Transplantation, 18(3), pp406-413.
50 Jacobsohn, D.A. & Vogelsang, G.B., 2007. Acute graft versus host disease. Orphanet Journal of Rare Diseases, 2(35).
51 https://clinicaltrials.gov/ct2/show/NCT01466569
52 https://clinicaltrials.gov/ct2/show/NCT01466569
Page 21

April 4, 2016
All patients enrolled had at least one treatment emergent adverse event. The study investigator determined that
approximately one-third of these events were due to treatment with AbGn-7. The most common adverse events
were gastrointestinal disorders, infections, metabolism and nutrition disorders, and musculoskeletal and connective
tissue disorders.
Preclinical data suggest that AbGn-107 is more potent than AbGn-7 in treating cancers expressing Le a -like
glycoepitope. Figure 17 compares properties of AbGn-7 and AbGn-107.
Figure 17. Properties of AbGn-7 and AbGn-107
Property AbGn-7 AbGn-107
Format Naked antibody ADC
Origin
Chimeric antibody
Humanized version of
AbGn-7
Target Le a -like glycoepitope Le a -like glycoepitope
Mechanism of
Action
Apoptosis/ADCC*/CDC**
Specifically targets tumors
and delivers potent
anticancer agents to induce
cell death
Stage Phase I completed in 2011 IND filing in H1 2016
*ADCC = Antibody-dependent-cell-mediated cytotoxicity
**CDC = Complement-dependent cytotoxicity
Source: LifeSci Capital
AbGn-110 for the Treatment of Breast Cancer and Gastric Cancer. AbGenomics is developing AbGn-110 for
the treatment of breast and gastric cancers. AbGn-110 is a bio-superior of Roche’s (SWX: RO) Kadcyla (adotrastuzumab
emtansine [T-DM1]), which is an FDA approved therapeutic for the treatment of HER2-positive,
metastatic breast cancer. Sales of Kadcyla were approximately $577 million for the first 9 months of 2105,
highlighting the market potential for a safe and effective therapy in this setting.
Both AbGn-110 and Kadcyla work by transferring a potent cytotoxic agent to breast tumors. AbGn-110 is designed
to outperform Kadcyla by:
! Delivering a validated payload with greater potency than Kadcyla.
! Improved stability that allows for an enhanced pharmacodynamics and pharmacokinetics profile.
! Greater safety profile – Kadcyla has a boxed warning for liver toxicity, heart toxicity, and potentially death.
! Improved solubility, lowering the risk of aggregation systemically.
Page 22

April 4, 2016
Preclinical studies thus far indicate that AbGn-110 is a more potent inducer of cell death than Kadcyla. Figure 18
shows that in a human breast cancer xenograft model, AbGn-110, at both 3 mg/kg and 1 mg/kg, significantly
inhibited tumor growth over a 40 day period. The black line labeled with App-ADC shows the results from using an
already approved ADC (Kadcyla) for the treatment of metastatic HER2-positive breast cancer. AbGenomics expects
to move AbGn-110 into clinical trials in the second half of 2017.
Figure 18. Performance of AbGn-110 in a Human Breast Cancer Xenograft Model
Source: Company Presentation
Page 23

April 4, 2016
Intellectual Property
AbGenomics currently owns 14 US patents protecting the intellectual property associated with AbGn-168H and
AbGn-107. Figure 19 lists those granted US patents.
Figure 19. AbGenomics’ Patent Portfolio
Program
AbGn-168H related
AbGn-107 related
US patents
No. 7744888
No. 8298540
No. 8628775
No. 7604800
No. 8287871
No. 8361472
No. 8663641
No. 8828397
No. 8557579
No. 9089614
No. 7674605
No. 7982017
No. 8568718
No. 9193794
Source: LifeSci Capital
Management Team
Ron (R. H.) Lin, Ph.D.
Chief Executive Officer & Chairman of the Board
Dr. Lin is founder, CEO and chairman of AbGenomics International Inc. (2006 – now). He founded AbGenomics
Corp. and served as CEO and chairman from 2000 to 2006. To better position the company, Dr. Lin re-organized
the company and established a new headquarters in San Francisco Bay Area where he and his team can efficiently
collaborate with world’s top talent, and key opinion leaders. He is the key inventor of 13 granted and 9 pending
worldwide patents. Under his leadership and well-established collaborative culture, the company discovered more
than 6 promising therapeutic candidates. Prior to joining AbGenomics, Dr. Lin was founding Professor and
Director of Graduate Institute of Immunology, College of Medicine, National Taiwan University (1991 – 2000). Dr.
Lin previously also served as an Associate Editor for Journal of Biomedical Science (1998 – 2002) and a member of
the Editorial Board for Int. Immunopharmacology (2001-2007). He received his Diploma in Biotechnology, Summa
Cum Laude, and Doctorate in Immunology, Magna Cum Laude, from the University of Tübingen in Germany. He has
conducted his research works at three world renowned institutions: Max Planck Institute for Immunogenetics in
Page 24

April 4, 2016
Germany, Basel Institute for Immunology in Switzerland, and the Immunobiology Division at Howard Hughes
Medical Institute at Yale University.
Shih-Yao Lin, MD, Ph.D.
Chief Medical Officer
Prior to joining AbGenomics, Dr. Lin was a research fellow (1998-2004) at the Laboratory of Allergy and
Immunology, Department of Experimental Pathology, Beth Israel Deaconess Medical Center in Boston, USA. Dr.
Lin received his MD degree from the School of Medicine, National Taiwan University, and completed the resident
training at the Department of Laboratory Medicine, National Taiwan University Hospital. He also received his PhD
degree in Immunology from the Centre d'Immunologie de Marseille-Luminy, University of Aix-Marseille II in
France.
Jim Early
Chief Financial Officer
Jim has over thirty years of experience in finance and accounting with more than twenty years in
pharmaceuticals/life science/healthcare, and ten years in financial services and real estate development. Jim’s
pharmaceutical and life science experience includes serving on the senior team for Synageva BioPharma (now
Alexion) for three years as SVP Finance and Administration and Corporate Secretary. At Zila Pharmaceuticals Jim
held several progressive titles over five years and served primarily as Director of Finance and Acquisitions, where he
led several successful multi-million dollar acquisitions providing financial modeling, due diligence, and integration
services for each transaction. Prior to this, Jim served four years as a financial analyst for Schein Pharmaceutical,
Steris Laboratories division. Prior to his time at Synageva, Jim served three years as VP Finance and CFO for
Anesthesiologists Associated, Inc. Most recently Jim has served as an interim CFO for his own consulting firm,
Early Financial Consulting, LLC, providing strategic planning, business development and finance/accounting
services in both pharmaceutical and financial services. Jim holds an active CPA license from the state of California,
having begun his career on the audit staff of PriceWaterhouseCoopers. He has a BBA from the University of Notre
Dame and an MBA from UCLA’s Anderson School of Management. Jim has also performed volunteer services in
the US and Brazil and is conversant in Portuguese.
Leewen Lin, Ph.D., LL.M.
Senior Vice President of IP Management and Quality Assurance
Prior to AbGenomics, Dr. Lin had been Senior Staff Fellow in Center of Biologics Evaluation and Research, US
Food and Drug Administration, Director of Biotechnology Development Program of Yung-Shin Pharmaceutical,
and Vice President of BioTrust International Corporation. Dr. Lin was also a member of the Intellectual Property
Committee of Academia Sinica, and served as an advisor to the Biotechnology and Pharmaceutical Promotion
Group, Ministry of Economic Affairs, Taiwan, on legal and regulatory affairs. Dr. Lin received her Ph.D. in
Biochemistry from North Carolina State University, and obtained her postdoctoral training in Genetics and
Molecular Biology in Harvard Medical School and Massachusetts General Hospital. In addition, she enrolled in the
J.D. program in Georgetown Law School while working full time at FDA. She received her L.L.M at Soochow
University Law School, Taipei, Taiwan.
Page 25

April 4, 2016
Ruth Li, Ph.D.
Vice President of Operation
Prior to joining AbGenomics, Dr. Li was a Professor in College of Public Health of National Taiwan University. She
obtained her Ph.D. from University of Illinois at Urbana-Champaign and completed her postdoctoral fellowship in
School of Public Health at Yale University.
Hazel Cheng, Ph.D.
Vice President of Project and Alliance Management
Prior to joining AbGenomics, Dr. Cheng was Vice President of Science and Technology of Westar Nutrition Corp.,
Costa Mesa in California. Her vast range of experiences include project management, business development,
manufacture operation, quality control and regulatory compliance of pharmaceutical cGMP. She received her Ph.D.
in Immunology from National Taiwan University School of Medicine.
Cheng-Chi Chao, Ph.D.
Senior Director of Technology and Preclinical Research
Dr Chao has over 20 years of experience in biopharmaceutical industry and medical research, focusing on drug
discovery and development in inflammatory diseases and oncology. Prior to joining AbGenomics, he was a senior
scientist and project team leader in a number of top ten global pharmaceutical companies, such as Merck, Schering-
Plough/DNAX research institute and Novartis. He was responsible for directing the progress of target / lead
identification and validation, and in vivo pharmacology. He has been involved in several therapeutic protein and
antibody programs that successfully advanced from discovery to clinic in his career. Dr. Chao received his Ph.D. in
Immunology from University of Iowa School of Medicine and was a postdoctoral fellow in the Department of
Microbiology and Immunology at Stanford University. Dr. Chao also serves as a board member and was a Vice
President in 2007 for Chinese Bioscience Association, USA.
Lingzhi Sun
Controller
Lingzhi brings in more than 10 years experience in public accounting and industry. Before working in industry she
was an audit senior manager in one of the Big Four accounting firm specializing in serving multinational and global
technology clients as well as venture- backed fasting growing companies. Lingzhi has significant experience with
initial and secondary offerings and on-going SEC reporting and Sarbanes- Oxley 404 requirements. She has served
companies conducting GAAP conversions from their local GAAP to US GAAP or to IFRS. Lingzhi also gained
significant experience in system implementation and consolidation through her career in the industry. She was the
project lead for two of the accounting system implementation projects. She participated all the key decision making
processes and made significant contributions to the scoping and validation of the new accounting and reporting
systems. She has worked on many complex accounting areas such as revenue recognition (ASC 605), earning per
share calculation (ASC 260), goodwill impairment analysis (ASC 350), stock based compensation (ASC805), business
combinations (ASC805), convertible debt and derivatives accounting (ASC470 and ASC815) etc. Lingzhi is a
Certified Public Account in California and a member of the AICPA.
Page 26

April 4, 2016
Risk to an Investment
We consider an investment in AbGenomics to be a high-risk investment. AbGenomics is a developmental stage
company with no history of taking a treatment to market, and currently has no FDA approved products in its
portfolio. The Company’s products in development may fail in clinical trials or fail to be approved by the FDA or
other regulatory agencies. Furthermore, early indications of efficacy do not necessarily translate into positive latestage
results. As with any company, AbGenomics may be unable to obtain sufficient capital to fund planned
development programs. Regulatory approval to market and sell a drug does not guarantee that the drug will
penetrate the market, and sales may not meet the expectations of investors.
Page 27

April 4, 2016
Analyst Certification
The research analyst denoted by an “AC” on the cover of this report certifies (or, where multiple research analysts are primarily responsible
for this report, the research analyst denoted by an “AC” on the cover or within the document individually certifies), with respect to each
security or subject company that the research analyst covers in this research, that: (1) all of the views expressed in this report accurately
reflect his or her personal views about any and all of the subject securities or subject companies, and (2) no part of any of the research
analyst's compensation was, is, or will be directly or indirectly related to the specific recommendations or views expressed by the research
analyst(s) in this report.
DISCLOSURES
This research contains the views, opinions and recommendations of LifeSci Capital, LLC (“LSC”) research analysts. LSC (or an affiliate)
has received compensation from the subject company for producing this research report. Additionally, LSC expects to receive or intends
to seek compensation for investment banking services from the subject company in the next three months. LSC (or an affiliate) has also
provided non-investment banking securities-related services, non-securities services, and other products or services other than investment
banking services to the subject company and received compensation for such services within the past 12 months. LSC does not make
a market in the securities of the subject company.
Neither the research analyst(s), a member of the research analyst’s household, nor any individual directly involved in the preparation of
this report, has a financial interest in the securities of the subject company. Neither LSC nor any of its affiliates beneficially own 1% or
more of any class of common equity securities of the subject company.
LSC is a member of FINRA and SIPC. Information has been obtained from sources believed to be reliable but LSC or its affiliates (LifeSci
Advisors, LLC) do not warrant its completeness or accuracy except with respect to any disclosures relative to LSC and/or its affiliates and
the analyst's involvement with the company that is the subject of the research. Any pricing is as of the close of market for the securities
discussed, unless otherwise stated. Opinions and estimates constitute LSC’s judgment as of the date of this report and are subject to change
without notice. Past performance is not indicative of future results. This material is not intended as an offer or solicitation for the purchase
or sale of any financial instrument. The opinions and recommendations herein do not take into account individual client circumstances,
objectives, or needs and are not intended as recommendations of particular securities, companies, financial instruments or strategies to
particular clients. The recipient of this report must make his/her/its own independent decisions regarding any securities or financial
instruments mentioned herein. Periodic updates may be provided on companies/industries based on company specific developments or
announcements, market conditions or any other publicly available information. Additional information is available upon request.
No part of this report may be reproduced in any form without the express written permission of LSC. Copyright 2016.
Page 28