COMPANY RESEARCH AND ANALYSIS REPORT

SymBio Pharmaceuticals Limited
4582
JASDAQ Growth Market

20-Apr.-2020
FISCO Ltd. Analyst

Yuzuru SatoFISCO Ltd.
http://www.fisco.co.jp

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/■ Index■Summary---------------------------------------------------------------------------------------------------------------------------------------------------------------------------- 01
1. Steady progress being made for becoming profitable in FY12/21. ..............................................................

01

2. Trends in the other development pipeline....................................................................................................

02

3. Results trends..............................................................................................................................................

02

4. Mid-Range Plan..................................... ......................................................................................................

03■Company profile---------------------------------------------------------------------------------------------------------------------------------------------------------- 04
1. History................................................... ......................................................................................................

04

2. Trends in the development pipeline. . ............................................................................................................

07■Results trends--------------------------------------------------------------------------------------------------------------------------------------------------------------- 15
1. FY12/19 results............................................................................................................................................

15

2. Outlook for FY12/20. . ............................. ......................................................................................................

16

3. Financial condition................................. ......................................................................................................

17■Mid-Range Plan----------------------------------------------------------------------------------------------------------------------------------------------------------- 19
1. Mid-Range Plan..................................... ......................................................................................................

19

2. Assumptions behind Performance Targets. . .................................................................................................

19

3. Sales growth potential.................................................................................................................................

20

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/█ Summary
Seeking to become a global specialty pharmaceutical company
Symbio Pharmaceuticals Limited <4582> (hereafter, also “the Company”) is a bio-venture that is advancing
developments in the fields of oncology, hematology, and rare diseases where there are few patients, but highmedical needs. The main drugs in the development pipeline are TREAKISYM®, whose indications as a treatment for
malignant lymphoma are expanding, and rigosertib, which is being developed for myelodysplastic syndrome (MDS).
In September 2019, the antiviral drug brincidofovir (hereafter, “BCV”) was added to the development pipeline. The
Company entered into a global licensing agreement for BCV, and the Company will develop the drug not only inJapan but overseas as well, as the Company seeks to become a global specialty pharmaceutical company.
1. Steady progress being made for becoming profitable in FY12/21
The Company has three key strategies to become profitable in FY12/21: building its own sales platform for
TREAKISYM®, launching sales of a high-added-value liquid formulation (ready-to-dilute) (“RTD”) for TREAKISYM®,
and expanding the indications for TREAKISYM®. Steady progress is being made on all three strategies. First, theCompany’s marketing agreement with Eisai Co., Ltd. <4523> for TREAKISYM® expires in December 2020, andthe Company is building a system that will allow it to quickly conduct sales after the agreement expires. By the endof Q2 FY12/20, the Company will complete building its own supply chain function, including sales representatives
along with a distribution and logistics system. Second, the Company submitted an NDA for TREAKISYM® RTD
liquid formulation in September 2019, and expects to commence sales of the RTD liquid formulation in Q1 FY12/21
if everything proceeds smoothly, and to switch from the existing lyophilized powder formulation type in one year.
Building its own sales platform and switching to the RTD formulation will significantly improve the profitability of
TREAKISYM®. In terms of expanded indications for TREAKISYM®, the Company plans to submit an application
for relapsed and refractory diffuse large B-cell lymphoma (DLBCL) in Q2 FY12/20, and by launching sales inapproximately Q3 FY12/21, sales of TREAKISYM® will likely increase further due to the growth in the number ofpatients receiving the drug. If these strategies progress smoothly, the Company expects net sales of ¥9,008mn andoperating profit of ¥1,031mn in FY12/21.

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Summary

2. Trends in the other development pipeline
For rigosertib (intravenous formulation), U.S. licensor Onconova Therapeutics, Inc. (“Onconova” “ONTX”) is conducting a global Phase III clinical trial, and top-line results (primary endpoints) are expected to be released in H2
FY2020. Based on the results of the trial, the Company is planning to apply for approval in Japan at the same time asin the U.S. and Europe, and is aiming to commence sales in 2023. Also, BCV, incensed-in in September 2019 fromChimerix Inc. (“Chimerix”) (U.S.), stands out for its ability to drastically suppress the proliferation of DNA viruses andfor its superior safety profile. BCV is expected to be developed into an effective treatment against a wide spectrumof infectious diseases caused by DNA viruses. As the first step, the Company aims to begin clinical trials in Japantargeting viral hemorrhagic cystitis (vHC)* occurring after allogeneic hematopoietic stem cell transplantation in H2
FY12/20. In addition, the Company plans to pursue business roll-out overseas, including a possible partnershipstrategy, that takes the regional characteristics of the targeted diseases into consideration. BCV is expected to beeffective for treating viral infectious diseases following organ transplants, and the Company’s target markets include
Europe, the U.S, and the Asian region including China as key markets, so this drug has tremendous potential. The
Company will decide its development strategy in overseas markets by Q3 FY12/20. The Company has acquired
manufacturing rights for BCV, and is currently in the process of selecting a manufacturing contractor ahead ofclinical trials.
*	Viral hemorrhagic cystitis (vHC): vHC is one type of viral infectious disease that frequently occurs following hematopoietic
stem cell transplantation. In vHC, hemorrhagic cystitis is caused by the proliferation of BK virus or adenovirus. In Japan,
the incidence ratio of vHC for allogeneic stem cell transplantation is between 8.6% and 24.0%. There are also reports thatthe incidence ratio of vHC for cord blood stem cell transplantation is even higher. It is generally refractory, with patients
showing primary symptoms such as frequent urination, abdominal pain, micturition pain and hematuria. In mild cases ofvHC, patients often experience no symptoms. However, in severe cases, disseminated infection can be lethal. There arealso reported cases where mortality occurs from renal impairment associated with adenovirus infection. Transplantation
with unrelated donors including cord blood, of which there is a high proportion in Japan, is a potent risk factor. Since
there are no approved drug therapies or definitive treatment in Japan, some physicians privately import cidofovir (CDV)
and administer it to their patients. However, CDV has a strong nephrotoxicity with only limited efficacy, so an effective andsafe drug therapy is eagerly awaited.

3. Results trends
In terms of results for FY12/19, the Company reported net sales of ¥2,837mn, down 26.0% year-on-year (YoY)
and an operating loss of ¥4,301mn (compared with a loss of ¥2,656mn in the previous year). The main reason
for the decrease in net sales was that sales of TREAKISYM® were halted temporarily due to quality issues at the
supplier. On the cost front, selling, general and administrative (SG&A) expenses rose 34.9% YoY to ¥5,166mn due
to the booking of upfront payments for the licensing of BCV and costs associated with building the Company’s
own sales platform. This in turn led to the larger operating loss. For FY12/20, the Company is projecting net salesof ¥3,404mn (+20.0 YoY) and an operating loss of ¥5,090mn. Although a recovery in net sales is expected dueto the calming down of the quality issue, both costs associated with building its own sales platform and researchand development (R&D) expenses are expected to increase, so the Company is forecasting that an operating loss
will continue. The Company initiated arbitration against The Medicines Company in the U.S. with the International
Chamber of Commerce (ICC), seeking damages of $82mn based on The Medicines Company’s repudiation of thelicensing agreement. The outcome of the arbitration is expected to be announced between March and June 2020,
but this is not factored into the forecasted results.

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Summary

4. Mid-Range Plan
As performance targets in its Mid-Range Plan, for FY12/22 the Company has set a net sales target of ¥10,816mn
and an operating profit target of ¥1,482mn, as the Company has set a target of sustainably securing an operating
profit margin of at least 10%. In order to achieve these targets, in addition to becoming profitable in FY12/21,
the Company plans to work on business development for BCV both in Japan and overseas, and on in-housemanufacturing of BCV in order to realize high profitability and high quality.
Key Points
In FY12/19, net sales declined due to quality issues with TREAKISYM®, and both R&D expenses and costs tobuild the Company’s own sales system increased• Targeting a turn to profitability in FY12/21 and continuous operating profit margin of at least 10%.
• Sales growth potential will further increase if global development of BCV progresses•

R e s ul ts t rends
Net sales (left)（¥mn）

Operating profit (right)

（¥mn）

5,000

-1,000
3,835

4,000
3,000

3,444

3,404

-2,1272,837-3,000-2,6562,368-2,0002,000-4,000-3,947-4,301

1,000-5,000-5,0900-6,000FY12/16

FY12/17

FY12/18

FY12/19

FY12/20 E

Source: Prepared by FISCO from the Company's financial results

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/█ Company profile
A bio-venture that conducts developments from the clinical-trials
stage, targeting the fields of oncology, hematology, and rarediseases
1. History
SymBio Pharmaceuticals is a bio-venture founded by the current Representative Director and President Chief
Executive Officer Fuminori Yoshida in March 2005. For its business strategy, its basic policy is to conduct drug
discovery and development for Underserved Therapeutic Areas in which development has not been progressed
due to the small numbers of patients. One of its features has a business model that aims to achieve highly efficientand rapid drug discovery within the areas targeting oncology, hematology, and rare diseases, which are fields withhigh medical needs, by licensing-in development candidates for which POC* for humans has been obtained, andconducting development from the clinical trials stage.
*	POC (Proof of Concept): when the usefulness and efficacy of a new drug candidate compound is recognized following its
administration to animals or humans during research and development.

The development candidate licensed-in first was the anti-cancer agent bendamustine hydrochloride (hereafter,
Bendamustine hydrochloride; product name in Japan, TREAKISYM®) indicated for malignant lymphoma that wasdeveloped by Astellas Pharma GmbH (Germany), for which the Company concluded an exclusive development andmarketing rights agreement for Japan in December 2005. With the development code SyB L-0501, the Company
began the phase I clinical trial in 2006 for indications for relapsed and refractory low-grade non-Hodgkin’s lymphoma (NHL) and mantle cell lymphoma (MCL)*, and in 2010, it acquired manufacturing and marketing approval.
It progressed licensing activities during this time, and in 2007, it expanded the target areas for the exclusive
development and marketing rights in China, South Korea, Taiwan, and Singapore. Then with Eisai as its marketing
partner, it concluded licensing agreements for Japan in 2008 and for South Korea and Singapore in 2009. Due toEisai’s change in business strategy, the Company has decided to dissolve the licensing agreements with Eisai on
December 9, 2020. For this reason, it plans to shift to its own sales system in Japan from 2021 onward. At present,
the Company is making preparations to establish sales and distribution systems.
TREAKISYM®, whose sales were launched in Japan in December 2010, continued to be subsequently developedin order to expand its indications, and in 2016, it acquired approval for indications for chronic lymphocytic leukemia(CLL) and untreated (first line of treatment) low-grade NHL/MCL, and its sales are growing. Also, in Asia, its sales
began in Singapore in 2010, in South Korea in 2011, and in Taiwan in 2012. For Taiwan, in 2008, the Company
concluded a licensing agreement with InnoPharmax Inc. (Taiwan), which is conducting sales through the Company.

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited

20-Apr.-20204582 JASDAQ Growth Market

https://www.symbiopharma.com/ir_e/

Company profile

Also, the second drug licensed-in was rigosertib (development code, SyB L-1101 (intravenous formulation)/
SyB C-1101 (oral formulation)), which is a development candidate from Onconova Therapeutics, Inc. (hereafter,
Onconova) indicated for myelodysplastic syndrome (MDS)*1 for which the Company concluded an exclusive
development and marketing rights agreement in 2011 for Japan and South Korea. Currently also, its development
is being progressed. Further, in 2017 it concluded an exclusive development and marketing rights agreement forJapan with Eagle Pharmaceuticals, Inc. <EGRX> (U.S.) for the TREAKISYM® liquid formulation, ready-to-dilute(RTD) formulation / rapid infusion (RI) formulation (development code, SyB L-1701/SyB L-1702)* 2, and in the sameway, its development is being progressed.
*1	MDS is a disease in which the patient cannot produce normal blood cells due to abnormalities in the hematopoietic
stem cells in the bone marrow, causing a decrease in normal blood cells and symptoms such as anemia, infection andhemorrhage. It is also known to transition to become acute myeloid leukemia. The condition of the bone marrow is
examined, the leukemia transition period is determined, and it is classified into four stages, such as according to thelength of the period. The high-risk type has a 25% leukemia transition period of 0.2 of a year, and the 50% survival period
median value is 0.4 of a year. There are approximately 11,000 patients in Japan. The only treatment of the root cause
is hematopoietic stem cell transplantation. In chemical therapy, azacitidine is used as the drug of first choice. In Japan,
Vidaza® of Nippon Shinyaku Co., Ltd.’s Vidaza® <4516> is on the market, and it has annual sales on a scale of ¥15 to¥16bn on a drug-price basis.
*2	Currently, TREAKISYM®, which has been approved in Japan, is a lyophilized powder formulation, which means it must
be dissolved at the medical site when it is used. As this task is unnecessary for the liquid formulation, it greatly reduces
the workload placed on healthcare workers. Also, the difference between the RTD formulation and the RI formulation is
the intravenous injection time. The RTD formulation takes the same time, 60 minutes, as existing products, but the time
for the RI formulation is as short as 10 minutes, so the burden on the patient is greatly reduced.

Moreover, in September 2019, the Company concluded an exclusive global license agreement with Chimerix thatgives the Company the rights to develop, manufacture, and commercialize BCV in all viral diseases excluding small
pox. BCV will be the third drug to be licensed-in and developed by the Company. The main features of BCV are thatit has higher anti-viral activity and a superior safety profile in comparison with cidofovir (CDV: unapproved in Japan).
Accordingly, BCV is expected to be an effective treatment against various infection diseases caused by DNA viruses.
As the first step, the Company plans to conduct development in Japan for the indications of viral hemorrhagic cystitis(vHC), conditions that can occur after allogeneic hematopoietic stem cell transplantation.
Technology licensing-in agreements
Name

Rigosertib sodium

Brincidofovir

SyB L-0501 (Lyophilized
powder formulation)
SyB C-0501 (Oralformulation)

SyB L-0501 (Lyophilized
powder formulation)
SyB C-0501 (Oralformulation)

SyB L-1701 (RTD
formulation)
SyB L-1702 (RI formulation)

SyB L-1101 (Intravenous
formulation)
SyB C-1101 (Oralformulation)

SyB V-1901 (Intravenous
formulation)

Astellas Pharma(Germany)

Astellas Deutschland(Germany)

Eagle Pharmaceuticals,Inc. (U.S.)

Onconova Therapeutics,
Inc. (U.S.)

Chimerix Inc. (U.S.)

Date agreement
was concluded /agreement period

December 2005 /Whichever is longer; the10-year period from thefirst product sales, or themarket-exclusive period
in JapanMarch 2007 / Whichever
longer; the 10-year period
from the first productsales or the marketexclusive period

September 2017 /Whichever longer; theproduct-patent period
or the market-exclusive
periodJuly 2011 / Whichever
longer; the 10-year period
from the first productsales (7 years in South
Korea), the marketexclusive period, or thepatent-validity period, ineach country

September 2019

Content of themain agreements

Exclusive development
and marketing rights inJapanExclusive development
rights and marketingrights in China (including
Hong Kong), Taiwan,
South Korea, andSingaporeExclusive development
rights and marketingrights in Japan

Exclusive development
rights and marketingrights in Japan and South
Korea

Exclusive global license
agreement concerning
the rights to develop,
manufacture, andcommercialize BCV in
all DNA virus indications
excluding smallpox

Development code

Licensing-in
partner

TREAKISYM®

Source: Prepared by FISCO from the Company's securities report and news releaseWe encourage readers to review our complete legal statement on “Disclaimer” page.

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

Technology licensing-out agreements
SyB L-0501 (Lyophilized powder formulation)
Licensing-out
partner

InnoPharmax Inc. (Taiwan)

Eisai Co., Ltd. (Japan)

Cephalon, Inc. (U.S.)

Date agreement
was concluded /agreement period

March 2008 / 10 years from thefirst product sales in Taiwan

From August 2008 to December
2020

From May 2009 to December
2020

March 2009 / 10 years from thefirst product sales in China

Content of the main
agreements

Exclusive development rights
and marketing rights in Taiwan

Joint development rights andexclusive marketing rights inJapanExclusive development rights
and marketing rights in South
Korea and Singapore

Exclusive development rights
and marketing rights in China(including Hong Kong)

Source: Prepared by FISCO from the Company’s securities report

History
Date
March 2005
December 2005

Summary
Established SymBio Pharmaceuticals Limited at Minato-ku, Tokyo
Concluded a license agreement with Astellas Pharma GmbH (Germany) to acquire exclusive development and marketing rights inJapan for anti-cancer agent Bendamustine Hydrochloride

March 2006

Obtained manufacturer's license (packaging, labeling and storage) from Tokyo Metropolitan Government

March 2007

Concluded a license agreement with Astellas Deutschland GmbH (Germany) to acquire development and marketing rights in
China, Taiwan, South Korea and Singapore for anti-cancer agent SyB L-0501

August 2008

Concluded a license agreement with Eisai Co., Ltd. to grant co-development and marketing rights in Japan for anti-cancer agent
SyB L-0501

March 2009

Concluded sublicense agreement with Cephalon, Inc. (U.S.) to grant development and marketing rights in China for anti-cancer
agent SyB L-0501

May 2009

Concluded a license agreement with Eisai to grant co-development and marketing rights in South Korea and Singapore for anticancer agent SyB L-0501

September 2010

Launched SYMBENDA® (generic name: bendamustine hydrochloride) in Singapore for the treatment of low-grade non-Hodgkin’s
lymphoma and chronic lymphocytic leukemia

October 2010

Acquired manufacturing and marketing approval of anti-cancer agent for malignant lymphoma TREAKISYM® in Japan (launched
in December 2010)

July 2011

Concluded a license agreement with Onconova Therapeutics, Inc. for anti-cancer agents SyB L-1101/SyB C-1101

October 2011

Launched SYMBENDA® (generic name: bendamustine hydrochloride) in South Korea for the treatment of chronic lymphocytic
leukemia and multiple myeloma

October 2011

Listed on Osaka Securities Exchange JASDAQ Growth Market

February 2012

Launched INNOMUSTINE® in Taiwan for the treatment of low-grade non-Hodgkin's lymphoma and chronic lymphocytic leukemia

October 2015

Concluded a licensing agreement with The Medicines Company (U.S.) to acquire exclusive development and marketing rights inJapan for post-operative, self-administered pain-management medication, SyB P-1501 (the agreement ended in November 2017)

May 2016

Established SymBio Pharma USA, Inc. at Menlo Park, California, USA

August 2016

Acquired manufacturing and marketing approval of anti-cancer agent for malignant lymphoma TREAKISYM® in Japan for the
additional indication of chronic lymphocytic leukemia

December 2016

Acquired manufacturing and marketing approval of anti-cancer agent for malignant lymphoma TREAKISYM® in Japan for the
additional indication of first-line treatment of low-grade non-Hodgkin's lymphoma and mantle cell lymphoma

September 2017Concluded a license agreement with Eagle Pharmaceuticals, Inc. to acquire development and marketing rights in Japan forbendamustine liquid formulations (RTD formulation and RI formulation)*RTD: Ready-to-dilute, RI: Rapid Infusion

October 2017

Filed for arbitration for damages against The Medicines Company (U.S.) due to the non-fulfillment of the licensing agreement

July 2018
September 2019

TREAKISYM® was newly listed as the standard treatment for malignant lymphoma in the 2018 edition of the Japan Society of
Hematology’s Guidelines for the Treatment of Hematopoietic Tumors,
Concluded an exclusive global license agreement with Chimerix (U.S.) concerning the rights to develop, manufacture, andcommercialize the antiviral drug, brincidofovir (excluding smallpox)

Source: Prepared by FISCO from the Company’s securities report and website

We encourage readers to review our complete legal statement on “Disclaimer” page.

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited

20-Apr.-20204582 JASDAQ Growth Market

https://www.symbiopharma.com/ir_e/

Company profile

Profit margin is expected to increase with the expansion ofindications for TREAKISYM® as the standard treatment for
malignant lymphoma and progress in the shift to the RTD liquid
formulation from 2021 onward
2. Trends in the development pipeline(1) TREAKISYM® (generic name: bendamustine hydrochloride)
TREAKISYM® is an anti-cancer agent for malignant lymphoma. Malignant lymphoma is a disease in which
lymphocytes, which are a type of white blood cell, undergo canceration (tumorification) and lumps (masses) cangrow in lymph nodes and organs other than lymph nodes (such as the stomach, intestines, thyroid, spinal cord,lung, liver, skin, and eyes) distributed throughout the body. It is said to be the most common of the blood cancers,
with approximately 10 out of every 100,000 people contracting it each year in Japan. Malignant lymphoma is
mainly divided into Hodgkin's lymphoma (HL) and non-Hodgkin's lymphoma (NHL), with about 90% of cases inJapan being NHL. It is classified into low-grade, medium-grade, and high-grade according to the progression
rate of the symptoms, and there are various disease types.
Types of non-Hodgkin’s lymphoma
Type according to grade

Non-Hodgkin's lymphoma type (disease type)Low grade: Indolent lymphoma(progresses yearly)

Follicular lymphoma (grade 1, 2), MALT lymphoma, lymphoplasmacytic lymphoma
Mycosis fungoides, Sezary syndrome, chronic lymphocytic leukemia / small lymphocytic lymphoma, etc.

Medium grade: Aggressive lymphoma(progresses monthly)

Follicular lymphoma (grade 3), mantle cell lymphoma, diffuse large B-cell lymphoma
Peripheral T cell lymphoma, extranodal NK / T cell lymphoma, adult T cell leukemia / lymphoma (chronictype), etc.

High grade: Highly aggressive
lymphoma (progresses weekly)

Burkitt's lymphoma, acute lymphocytic leukemia / lymphoblastic lymphoma
Adult T-cell leukemia / lymphoma (acute type, lymphoma type), etc.

Source: Prepared by FISCO from National Cancer Center Hospital materials

Among these, currently the Company has acquired marketing approval for indications for relapsed and refractory
low-grade non-Hodgkin’s lymphoma (NHL), mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL),
and untreated (first line of treatment) low-grade NHL/MCL. In particular, in 2016 its use in this field started tospread following the acquisition of marketing approval for untreated low-grade NHL/MCL, and then in July 2018,
in its treatment guidelines, the Japan Society of Hematology (JSH) recommended TREAKISYM® and Rituximab®
combination therapy (BR therapy) as the standard treatment, and it is becoming established as the standard
treatment in both name and reality. In the field of untreated (first line of treatment) low-grade NHL, previously thestandard treatment was R-CHOP therapy*, but on looking at the market penetration rates, after Q4 FY12/17(October to December 2017), BR therapy had overtaken it, and as of Q2 FY12/19 (April to June), BR therapy had
a 55% share of the market as a whole. In light of the high efficacy of BR therapy, the Company aims to increase
the market penetration rate up to 64% in 2020 in the untreated area at the end of 2020.
*	R-CHOP therapy: a multi-drug combination therapy combining Rituximab® and 4 other drugs

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

BR therapy It is superior to R-CHOP for both efficacy and safety

Source: From the Company’s results briefing material

Treatments for untreated (first line of treatment), low-grade NHL patients

Source: From the Company's results briefing material

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

The Company is currently progressing five drugs in the development pipeline. Of these, in the phase III clinical
trial for the expansion of the indication of the existing lyophilized powder formulation TREAKISYM® for relapsed
and refractory DLBCL, the observation periods of all test subjects were completed in September 2019. On
November 5, 2019, the Company announced favorable results, with the overall response rate, which was theprimary endpoint of the trial, exceeding the expected response rate. Currently, the Company is preparing to submit
a new drug application (NDA) during Q2 FY12/20. If steady progress is made, it is expected that approval could
be granted and sales commence in Q3 FY12/21. If relapsed and refractory DLBCL is approved as an indication,
the potential market size for TREAKISYM® will grow to more than double the size of the existing market. Thisis because the number of patients for all three areas covered by existing indications is currently a little less than
10,000 but the number of relapsed and refractory DLBCL patients will increase by 1.5 times if the new indication isapproved. Patient advocacy groups and relevant academic societies have also filed petitions urging the authorities
to make BR therapy available as early as possible. As soon as sales begin, TREAKISYM® is expected to rapidly
penetrate the market in the field of relapsed and refractory DLBCL.
In September 2019, the Company applied for marketing approval for the RTD formulation, which is the
TREAKISYM® liquid formulation. If steady progress is made, the Company expects to obtain approval in Q4
FY12/20 and to launch sales in Q1 FY12/21. The Company plans to switch 95% of the product from the current
lyophilized powder formulation to the RTD formulation by the end of 2021, and from the beginning of 2022 theCompany will aim to quickly achieve a 100% switch. Also, the Company started clinical trials of the RI formulationin November 2018 with the main aim of confirming its safety (planned number of cases, 36), and is aiming tocomplete clinical trials in H1 FY12/20, and launch sales in H1 FY12/22. The indications for the RTD/RI formulations
include all those for which TREAKISYM® has already been approved as well as relapsed and refractory DLBCL.
Teva Pharmaceuctical Industries Ltd. (U.S.) has already commercialized the RTD/RI formulations as BENDEKA®
on the U.S. market. With the acquisition of a 97% share of the bendamustine market as of 2017, it appears thatmost patients have already switched to the liquid formulation. In addition to reducing the burden on healthcare
professionals by eliminating the need to dissolve the drug, the administration time for the RI formulation is
only 10 minutes (compared to 60 minutes for the existing products and RTD formulation), so the burden on
patients is also greatly reduced. Therefore, there are strong calls for its early marketing approval in Japan. As the
exclusive sales period for the existing, lyophilized powder formulation type in Japan ends in 2020, generics may
be developed for it. But if the RTD/RI formulations are launched, there will be major differences in terms of theirfunctions, so this would effectively extend the exclusive marketing period until 2031. If the RTD/RI formulations
are launched, the drug prices will be the same level as the previous products, but the supplier will be changed
to Eagle Pharmaceuticals, so at FISCO we think it is highly likely that the profit margin will improve compared tothe existing products.

We encourage readers to review our complete legal statement on “Disclaimer” page.

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COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

For the treatment of malignant lymphoma, TREAKISYM® obtained approval for use as a pretreatment agent for the
chimeric antigen receptor T-cell (CAR-T) therapy (KYMRIAH® intravenous transfusion), which obtained NHI pricelisting in May 2019. TREAKISYM® is also currently used as a drug in combination therapy in multiple clinical trials.
Most recently, in February 2020, it was announced that the primary endpoints have been achieved in a domestic
Phase II clinical trial (Polatuzumab vedotin + BR combination therapy) being conducted by Chugai PharmaceuticalCo., Ltd. <4519> to treat relapsed and refractory DLBCL patients. This therapy received marketing approval in
the U.S. in June 2019 and in Europe in January 2020, so there is a high likelihood that it will receive approval in
Japan as well. Whether to treat relapsed and refractory DLBCL patients with BR therapy or Polatuzumab vedotin+ BR therapy will be determined by doctors based on a patient’s condition, but in both cases TREAKISYM®
will be used. These developments signal that TREAKISYM® now has an even more solid position as a standard
treatment for the future, and at FISCO we believe that it will result in an expansion of the market for TREAKISYM®.
*	Marketed by Novartis Pharma (Switzerland). The areas of indication are CD19 positive recurrent/refractory B-cell acutelymphoblastic leukemia and DLBCL (there are multiple other requirements for administration), and in Japan it is believedthat there are just under 250 patients per year who could be administered this drug.

The Company has decided to abort development of TREAKISYM® oral formulation (development code: SyB
C-0501) for progressive solid tumors and systemic lupus erythematosus (SLE). This decision was made because
the results of trials did not exceed expectations, and, in order to best utilize limited management resources, theCompany made the decision to prioritize the domestic and overseas development of BCV, for which the Company
has newly acquired a license as a part of its business strategy.
*	An autoimmune disease in which the patient’s own immune system mistakenly attacks normal cells. It is designated as an
intractable disease because it causes inflammation and tissue damage in various organs throughout the body. There areapproximately 60,000 to 100,000 patients in Japan.

TREAKISYM®
Drug

Indication

Progress

r/r low Low-grade NHL/MCL

Approved October 2010

CLL

Approved August 2016

1st line Low-grade NHL/MCL

Approved December 2016r/r DLBCL

P3 completed and achieved the primary endpoint, scheduled toapply for marketing approval in Q2 FY12/20

SyB L-1701 (RTD liquid
formulation)

All indications

Applied for marketing approval in September 2019, targeting launchin Q1 FY12/21

SyB L-1702 (RI liquid
formulation)

All indications

Currently undergoing clinical trials, complete trials in the first half of
FY12/20, targeting launch in the first half of FY12/22

SyB L-0501(FD lyophilized
powder formulation)

Source: Prepared by FISCO from the Company's results briefing material and website(2) Rigosertib (intravenous formulation/oral formulation)
Rigosertib is an anti-cancer agent that has a unique multi-kinase inhibitory action (which causes cancer cells todie by inhibiting the multiple kinases involved in cancer cell proliferation, invasion and metastasis). Its development
is being progressed indicated for high-risk myelodysplastic syndrome.

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10 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

For the current development situation, a global Phase III clinical trial is being conducted by licensor Onconova for
the intravenous formulation indicated for relapsed and refractory high-risk MDS (final target 360 patients), and asof November 2019 more than 90% of the target number of patients had been registered. In Japan, the Company’s
assigned area, the Company has registered subjects for 48 of the target number of 50 cases as of the end of
December 2019. In H1 FY12/20, Onconova plans to announce the top-line results (primary endpoints). Based on
the results of the trial, the Company is planning to apply for approval in Japan at the same time as in Europe andthe U.S. The aim is to submit the application for marketing approval in 2021, and receive approval in Q4 FY12/22.
The potential market size is seen as being roughly the same as Vidaza®, at ¥15 to ¥16bn.
For the oral formulation, the Phase I clinical trial was completed for the single drug indicated for relapsed andrefractory high-risk MDS in June 2019, while in the future, the plan is to switch to development for its joint use
with azacitidine. However, it appears that development is now being given lower priority for the time being, as
Onconova’s business strategy is to first aim for marketing approval for an intravenous formulation for high-risk
MDS, and also because a physician-led phase I trial targeting progressive KRAS* positive NSCLC (non-small celllung cancer) as a new indication is going to start in 2020. Onconova has confirmed a tumor suppressive effect ina KRAS-positive non-small cell carcinoma animal model by using an immune checkpoint inhibitor and rigosertib
in combination, and is expected to proceed with clinical development.
*	KRAS is a cancer gene. The KRAS gene usually inhibits the replication of cells, but when a mutation of the KRAS geneoccurs, a signal for cells to replicate is continually sent out, which causes cancerous cells to actively replicate. Patientswith mutated KRAS genes have been identified in a considerable percentage of patients with certain forms of cancer,
including colorectal cancer and lung cancer.

Rigosertib
Drug
SyB
L-1101(Intravenous
formulation)

SyB C-1101 (Oralformulation)

Indication

Progress

Relapsed and refractory high-risk MDS

P3 global clinical trials ongoing
Onconova plans to announce top-line data in H1 FY2020
If satisfactory, plans to apply for approval in Japan at the same time as in the
U.S. and Europe

Relapsed and refractory high-risk MDS
single drug

P1 completedUntreated high-risk MDS (AZA
combination use)

Under preparation

Untreated high-risk MDS (AZA
combination use)

P2/3 global clinical trial under preparation at Onconova
If it starts, the Company plans to participate

Source: Prepared by FISCO from the Company's results briefing material and website(3) Brincidofovir (BCV) (Intravenous formulation/Oral formulation)a) Overview of Brincidofovir and licensing agreement
Brincidofovir (BCV) is a lipid conjugate of cidofovir (CDV), which is known as a treatment of cytomegalovirus(CMV) retinitis. BCV is an antiviral drug candidate that has higher anti-viral activity and a superior safety profile incomparison with CDV. Lipid conjugatation allows for more efficient uptake of BCV into cells than CDV alone. Onceinside target cells, the lipid chain is cleaved, releasing CDV, which is then converted to its active form of cidofovir
diphosphate (CDV-PP), which fulfills the role of inhibiting viral DNA replication. For this reason, data showing that
BCV has a much higher anti-viral replication effect than CDV and other anti-viral drugs have been obtained fromin vivo tests and other studies. In terms of the safety profile, CDV has the side effect risk of strong nephrotoxicity,
including the risk of renal dysfunction, through the accumulation of CDV in renal tubular epithelial cells. However,
because the lipid conjugation of BCV brings no accumulation of CDV in renal tubular epithelial cells, BCV has theoutstanding feature of reducing the risk of nephrotoxicity associated with CDV. CDV has been granted fast track
designation by the U.S. FDA for the prevention of cytomegalovirus and the treatment of adenovirus and smallpox,
while in Europe the EMA has granted orphan drug designation for the same viruses.

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11 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

How BCV works

Source: Reprinted from the Company’s website

Chimerix had been developing an oral formulation of BCV, but it had discontinued development because it did
not obtain favorable results in Phase III clinical trials, including some side effects. Subsequently, Chimerix had
been looking for a partner to whom it could license out BCV in order to concentrate its management resourcesin the anti-cancer agent field, while the Company was searching for new drug agents to license in. The timing
was right for both companies, and they concluded a licensing agreement for the manufacture, marketing anddevelopment (excluding smallpox) of BCV. The main factors behind the Company’s decision included the factthat BCV is used for rare diseases and underserved therapeutic areas, which matched the Company’s targets
for development, and because, like TREAKISYM®, the indicated diseases are in the hematologic disease field,so there would be no need for additional sales representatives, and thus there would be significant synergies interms of marketing efficiency.
Chimerix has suspended development of an oral formulation, but the Company sees the reason for this as
being that the absorption rate of the drug from the gastrointestinal tract was low. The Company believes that an
intravenous formulation could have the same effect as an oral formulation at just 10% of the dosage, so it thinks
there is a higher probability of success. This agreement covers both the intravenous formulation and the oral
formulation, as the Company believes that going forward, there is a possibility that it can resolve the issues with theoral formulation through new improvements to the drug. Of the viral infectious diseases, the reason why smallpox
alone is excluded from the agreement is that the U.S. government needs to maintain its ability to manufacture andstockpile a smallpox treatment independently within the country as a measure to counter bioterrorism.
What is notable in this agreement is that it is a global license, and also that it covers manufacturing rights. This drug
will be used to treat viral infectious diseases that occur following hematopoietic stem cell transplantation or organtransplants. The organ transplant market is large not only in the U.S. and Europe, but also in Asia, where there issignificant growth potential. TREAKISYM® has been marketed in South Korea, Taiwan, and Singapore throughsales partners. However, sales volumes were small and there was only a negligible impact on the Company’sbusiness performance. If the overseas rollout of BCV is successful, the Company will consider growth as a global
company.

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12 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

Also, the quality issue for TREAKISYM® that arose in FY12/19 impacted the decision to have the agreement
include manufacturing rights. The Company understands the importance of controlling manufacturing on its own
and building a system to suppress business risks to the maximum extent possible. The Company is currently inthe process of selecting a manufacturing contractor, and this will be possible as long as the partner company has
the technology to manufacture a highly-active anti-viral drug. It is expected that a decision will be made soon.
In conjunction with the conclusion of the licensing agreement, the Company paid developer Chimerix an upfront
payment of US$5mn (approximately ¥540mn) in Q3 FY12/19, and will pay future milestones of up to US$180mn(approximately ¥19.4bn), as well as a double-digit royalty on net sales of brincidofovir products.
b) Future development plans
As a future development plan, the Company is planning to first develop an intravenous formulation to treat viralhemorrhagic cystitis following hematopoietic stem cell transplantation in Japan. The Company is aiming to start aclinical trial in H2 FY12/20, and is targeting the launch of sales in 2025. Chimerix has already completed a Phase
I clinical trial, so it appears the Company wants to start from a Phase II clinical trial citing the data from Chimerix’s
Phase I clinical trial. In addition, the Company is considering making use of the SAKIGAKE system, which can
reduce the reviewing time needed for drug approval.
In Japan, there are roughly 3,700 hematopoietic stem cell transplants (allogeneic) in each year, and it is reported
that the incidence rate for viral hemorrhagic cystitis (vHC) is between 8.6% and 24% (even higher for cord blood
stem cell transplantation) for these transplants. Currently, there are no effective approved drugs, and in somecases, physicians are individually importing non-approved CDV, but these have side effects, so a safe treatment isdesirable. By advancing clinical development in Japan before anywhere else in the world, the Company is looking
to lay the foundation for a global business roll-out. The Company will decide its development plan regarding its
overseas development strategy by Q3 FY12/20. On a global basis, there are approximately 35,000 hematopoietic
stem cell transplants (allogeneic) performed each year, and when organ transplants are included, the number ofprocedures exceeds 100,000. Thus, the Company will be developing a treatment for a variety of viral infectious
diseases that occur following these transplant operations. As for overseas business expansion, the Company
plans to implement a partnership strategy that harnesses the regional characteristics of the targeted diseases.
Future BCV development

Source: From the Company's results briefing material

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13 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

In addition, the novel coronavirus pneumonia, which has continued to spread worldwide since February 2020, is
an RNA virus infectious disease, and therefore basically falls outside the protective scope of BCV, which inhibits
DNA viruses. However, despite its mechanism of action being unknown, it has been found effective as a treatment
for Ebola hemorrhagic fever, also an RNA virus infectious disease. Therefore, the Company plans to contact
Chimerix and have a reverification performed.
BCV’s first development and commercialization target
Disease field

Indication

Hematopoietic Viral
stem cell
hemorrhagictransplants
cystitis (vHC)

Current status

Problems• With no approved drug therapies in Japan, some physicians privately import cidofovir (CDV)• Bladder perfusion isand administer it to their patients.
the only treatment(The standard treatment method is to administer 5mg/kg weekly for two weeks, thenfor symptoms.• subsequently administer the same dosage once every two weeks as maintenance therapy)
No definitive
treatments• Bladder perfusion is the only treatment for symptoms.
• Patients suffer from urinary problems and pain, and disseminated infections can be lethal.

Source: Prepared by FISCO based on the Company’s materials

Estimated number of estimated allogeneic hematopoietic stem cell transplants and kidney plants(Number of cases, people)
Region (population)

U.S. (320 million)

EU (550 million)

Japan (130 million)

Hematopoietic stem celltransplants (allogeneic)

8,700

16,400

3,700

Other
6,454

Total
35,254

Kidney transplants

19,86020,0001,648

39,05280,560Liver transplants

7,800

7,400

438

10,06225,700

Other transplants

5,940

4,500

124

1,276

11,840Source: Prepared by FISCO, based on the Company’s materials prepared according to Chimerix’s materials (January 2019)

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14 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile█ Results trends
Decline in revenue in FY12/19 due to TREAKISYM® quality issue,
increase in R&D expenses and costs to build the Company’s own
sales system
1. FY12/19 results
In FY12/19, net sales decreased 26.0% YoY to ¥2,837mn, the operating loss was ¥4,301mn (compared to a loss of¥2,656mn in the previous year), the ordinary loss was ¥4,376mn (compared to a loss of ¥2,748mn in the previousyear) and the loss was ¥4,376mn (compared to a loss of ¥2,752mn in the previous year). The main reason for the
lower net sales was that the Company temporarily halted imports and sales of TREAKISYM® products that were
imported from Astellas Deutschland GmbH, a subsidiary of Astellas Pharma Inc. <4503> because problems such as
contaminants and appearance defects were found in these products. The Company procures TREAKISYM® products from suppliers with factories in Belgium and Germany. It looks like there were problems with the manufacturing
process and quality control at the factory in Germany, which had been the Company’s supplier. However, there werethen also problems with products supplied by a factory in Belgium, and this resulted in net sales falling well short ofthe previous year’s results as well as the Company’s forecasts. With these quality issues, the Company is in ongoingtalks with Astellas Deutschland GmbH, including demanding corrective measures, to have the company fulfill its
obligation as a stable supplier, but it appears that the quality issue has not been completely resolved. It appears
that the size of the end market for TREAKISYM® in 2019 on an NHI drug price basis was roughly flat year-on-year
at approximately ¥8,400mn. While shipments from the Company declined, it appears that inventories held by sales
partner Eisai and its pharmaceuticals wholesalers were sold.
On the cost front, R&D expenses rose 33.2% YoY to ¥2,441mn. The aforementioned upfront payment for BCV
accounted for ¥540mn of these expenses, while the rest were related to clinical trials for TREAKISYM® andrigosertib. Other SG&A expenses increased 36.5% YoY to ¥2,724mn. This increase was mainly due to higher
costs recorded to build the Company’s own sales platform for TREAKISYM®. During Q4 FY12/19, the Company
recruited additional TREAKISYM® regional sales managers and sales representatives, formed business allianceswith pharmaceutical wholesalers, and moved to a system with two logistics centers (eastern Japan and westernJapan), as well as bolstered information systems, with the aim of enhancing its distribution and logistics capabilities.
FY12/19 results(¥mn)
FY12/18
Results

FY12/19
YoY (%)

Vs. forecast

3,835

4,465

2,837

-997

-26.0%-1,627

Product sales

3,809

4,457

2,811

-998

-26.2%-1,645

Other sales

25

7

26

0

3.2%

19

Gross profit

1,172

1,466

864

-308

-26.3%-601

SG&A expenses

3,828

5,053

5,166

1,337

34.9%

113

R&D expenses

1,832

2,508

2,441

608

33.2%

-66

Other SG&A expenses

1,996

2,545

2,724

728

36.5%

179

Operating profit (loss)-2,656-3,587-4,301-1,645--714

Ordinary profit (loss)-2,748-3,612-4,376-1,627--764

Profit (loss)-2,752-3,616-4,376-1,623--760

Net sales

Initial forecast

Results

YoY

Source: Prepared by FISCO from the Company's financial results and results briefing material

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15 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

Expecting net sales to recover and expenses to continue to increase
in FY12/20
2. Outlook for FY12/20
The outlook for the FY12/20 results is for net sales to increase 20.0% YoY to ¥3,404mn, an operating loss of¥5,090mn (compared to a loss of ¥4,301mn in FY12/18), an ordinary loss of ¥5,134mn (a loss of ¥4,376mn), anda loss of ¥4,803mn (a loss of ¥4,376mn). Net sales are expected to increase YoY for the first time in two years dueto the recovery in the volume of TREAKISYM® procured.
Meanwhile, on the cost front, R&D and other SG&A expenses are expected to increase continuously. With respect
to R&D expenses, the initial payment of ¥540mn in conjunction with the BCV agreement will disappear, but theCompany is expecting to pay a milestone payment of ¥500mn based on the marketing approval of the RTD
formulation of TREAKISYM® (liquid formulation), as well as the addition of clinical trial expenses for BCV, on top of
those for TREAKISYM® and rigosertib, so the Company is forecasting a 11.9% YoY increase to ¥2,731mn.
Also, other SG&A expenses are expected to increase 28.6% YoY to ¥3,505mn, mainly due to expenses to establish
the Company’s own sales platform. The Company plans to complete the building of its sales system, logistics
system, and internal information system by Q2 FY12/20, and expects to commence its own sales immediately
following the expiration of the marketing agreement in December 2020. In terms of the salesforce, the Company
will divide the nation into six blocks, and build a locally-rooted platform with 62 people (including approximately 30
regular employees and 30 contract MRs). From April 2020 onward, the Company plans to hire 30 contract MRs
and provide them with training. Concerning internal information systems, including a system to manage orders, theCompany is currently conducting test operations with the goal of operation from H2, and is otherwise preparing to
be able to quickly transition to the new system after the agreement with Eisai expires in December 2020.
Outlook for FY12/20(¥mn)
FY12/19
Results

FY12/20
Company forecast

Change% change

Net sales

2,837

3,404

566

20.0%

Gross profit

864

1,146

281

32.5%

SG&A expenses

5,166

6,236

1,069

20.7%

R&D expenses

2,441

2,731

289

11.9%
28.6%

2,724

3,505

780

Operating profit (loss)-4,301-5,090-788-

Ordinary profit (loss)-4,376-5,134-757-

Profit (loss)-4,376-4,803-426-

Other SG&A expenses

Source: Prepared by FISCO from the Company’s financial results and results briefing material

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16 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Company profile

In October 2017, the Company filed a petition for arbitration with the International Chamber of Commerce (ICC) in
the U.S. against The Medicines Company for damages for the non-fulfillment of a licensing agreement.* The final
documents were submitted in December 2019, and the outcome will likely be announced within three to six months
if the case proceeds as usual. The claim was for US$82mn, and the decision announced by the ICC will effectively
be the final confirmation, but the impact of this has not been included in the Company’s forecasted results. Novartis
AG (Switzerland) announced in January 2020 that had made The Medicines Company a subsidiary, so there is norisk that compensation for damages will become unpayable.
*	In 2015, the Company concluded a licensing-in agreement with The Medicines Company for a self-administered, short-term
acute pain management medication. The Company, acting in the best interests of patients, temporarily suspended newpatient enrollment for the drug from April 2017 due to the concern about the continuity of The Medicines Company’sbusiness regarding the product. In October 2017, the Company filed for arbitration with the ICC seeking a payment of
US$82mn as compensation for damages for the non-fulfillment of the licensing agreement. In the arbitration, the Company
claims that The Medicines Company was not able to provide the Company with adequate assurance of its performanceof obligations under the licensing agreement in light of its decision to discontinue commercialization activities regarding
the product and withdraw from markets in the U.S. and Europe, and that such failure by The Medicines Company was amaterial breach of the licensing agreement. The Company then terminated the licensing agreement.

Issued stock acquisition rights to procure funds for businessactivities, the required procurement amount could change
depending on the outcome of the ICC arbitration
3. Financial condition
Looking at the financial condition at the end of FY12/19, total assets stood at ¥5,273mn, a decrease of ¥965mn
from the end of the previous fiscal year. The main factors behind this change were a ¥137mn increase in accounts
receivable-trade, a ¥910mn decrease in cash and deposits, and a ¥533mn decline in merchandise and finishedgoods under current assets. For merchandise and finished products, assets were previously booked at the time of
shipment from Astellas Deutschland’s factories, but due to the quality issue that arose, this process was changed
so that assets are booked after completion of an examination upon arrival. Under non-current assets, there wasa combined increase of ¥169mn in software and software in progress related to the Company’s effort to build its
own sales system.
Total liabilities stood at ¥873mn, an increase of ¥463mn from the end of the previous fiscal year. The main factor
behind this change was an increase of ¥136mn in accounts payable-other, which was partly offset by a decreaseof ¥605mn in accounts payable-trade. In addition, total net assets stood at ¥4,400mn, a decrease of ¥501mn fromthe end of the previous fiscal year. While capital stock and capital surplus totaled ¥3,798mn following the exerciseof stock acquisition rights, retained earnings decreased ¥4,376mn due to the recording of a loss. Consequently,
the equity ratio decreased from 70.1% at the end of the previous fiscal year to 71.7%.
The Company issued the 50th and 51st Stock Acquisition Rights (with Exercise Price Revision Clauses) via anallotment to EVO FUND on March 16, in order to raise funds for business activities through H1 FY12/21. Theserights represent 10 million dilutive shares, and if all of the rights are exercised, shareholder value per share would
be diluted by 37.8%. However, if the Company receives compensation for damages based on the aforementioned
arbitration with The Medicines Company, the Company can limit the number of rights exercised depending on theamount received from the arbitration. The amount of funds to be raised is approximately ¥5.4bn, and if more than acertain amount of compensation for damages are received, the Company can at that time buy back and cancel all
non-exercised stock acquisition rights. Therefore, the outcome of the arbitration is of high interest.

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17 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited

20-Apr.-20204582 JASDAQ Growth Market

https://www.symbiopharma.com/ir_e/

Company profile

Balance sheet and management indicators(¥mn)
End-FY12/16

End-FY12/17

End-FY12/18

End-FY12/19

Change

6,685

4,036

6,038

4,887

-1,150

5,719

2,947

4,821

3,910

-910

193

215

200

386

185

Total assets

6,878

4,252

6,239

5,273

-965

Total liabilities

1,393

1,012

1,337

873

-463

450

-

-

-

-

5,484

3,239

4,901

4,400

-501

73.5%

63.6%

70.1%

71.7%

Current assets(Cash and deposits)
Non-current assets(Interest-bearing debt)
Net assets
Management indicator
Equity ratio

Source: Prepared by FISCO from the Company's financial results

Overview on the issuance of acquisition rights by third-party allotment
50th Stock Acquisition Rights

51st Stock Acquisition Rights

Total number of stock acquisitionrights700

300

Number of dilutive shares from theissuance

700

300

Exercise price and conditions forrevising the exercise price

94% of the simple average value of the volume weighted average price on each trading day for the fiveconsecutive trading days prior to the Revision Date.
Minimum exercise price is ¥291

Expected exercise period

In principle, commitment to the exercise all Stock
Acquisition Rights within five months following
issuance (commitment to the exercise of at least
40% of issued Stock Acquisition Rights within 56
trading days)
Excluding the case in which an event triggering theextension of the commitment period occurs

In principle, commitment to the exercise of all Stock
Acquisition Rights within 46 trading days from theexercise start date specified by the Company
Excluding the case in which an event triggering an
extension of the commitment period occurs

Expected start date of exercise

March 17,2020

To be determined

Expected completion date of fullcommitment

August 21,2020

To be determined

3,829

1,641

Amount to be funded (¥mn)
Source: Prepared by FISCO from the Company's news release

Uses of the funds(¥mn)
50th Stock Acquisition
Rights51st Stock Acquisition
RightsExpected timing of expenditure

Development of in-licensed drugs

2,375

55

Creation of the Company’s own sales platform

1,431

54

March 2020 to June 2021

Investment in new in-licensing, M&A, and other

0

1,535

October 2020 to June 2021

3,806

1,644

Total

March 2020 to June 2021

Source: Prepared by FISCO from the Company's news releaseWe encourage readers to review our complete legal statement on “Disclaimer” page.

18 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/█ Mid-Range Plan
Targeting a turn to profitability in FY12/21 and to continuously havean operating profit margin of at least 10%
1. Mid-Range Plan
In February 2020, the Company announced a Mid-Range Plan that spans the three years through FY12/22. In thePlan, the Company aims to have net sales of ¥10,816mn and operating profit of ¥1,482mn in FY12/22, and tosecure an operating profit margin of at least 10% on a continual basis. In FY 12/21, the Company aims to begin
full operation of its own sales platform. Based on this, by quickly switching from the current lyophilized powder
formulation type of TREAKISYM® to the RTD formulation of TREAKISYM®, and by expanding market penetration
with the additional indication of to treat relapsed and refractory DLBCL to quickly achieve annual net sales of¥10bn on an NHI price basis, the Company hopes to achieve an operating profit in FY12/21 and thereafter realize
sustainable growth. With the addition of BCV to the development pipeline, the Company aims to achieve growth asa global specialty pharmaceutical company.
Mid-Range Plan(¥mn)
FY12/19 results

FY12/20
forecast

FY12/21targets

FY12/22
targets

Net sales

2,837

3,404

9,008

10,816

Operating profit (loss)-4,301-5,090

1,031

1,482

Ordinary profit (loss)-4,376-5,134987

1,438

Profit (loss)-4,376-4,803

1,356

1,717

Source: Prepared by FISCO from the Company's results briefing material

2. Assumptions behind Performance Targets
The majority of net sales are product sales for TREAKISYM®. The sales targets take into account recent sales
trends and the pace of market penetration. Net sales through FY12/20 are sales based on product shipment unit
prices to Eisai, but from FY12/21, sales will be based on shipment unit prices to pharmaceutical wholesalers by theCompany’s own sales platform, so net sales are expected to rapidly increase. The main factor for the increase insales in FY12/22 is the increase in demand because of the additional indication of relapsed and refractory DLBCL,
for which approval is expected in FY12/21.
The Company is expecting gross profit margin to increase from the FY12/20 forecast of 33.7% to 70–80% in
FY12/22 due to the shift to its own sales platform and progress in the switch to the RTD formulation.
The Company is forecasting SG&A expenses of ¥6,236mn in FY12/20, and forecasts SG&A expenses to remainaround the ¥6.0bn level from FY12/21 onward (excluding milestone payments). Of this, R&D expenses are estimatedbased on the Company’s plans for development plan for its existing pipeline, comprising TREAKISYM®, the rigosertib
intravenous and oral formulations, and BCV, but expenses such as initial payments on agreements related to the new
pipeline are not included. Other SG&A expenses mainly consist of expenses for sales and marketing, production
and logistics operations, business development, and management related to TREAKISYM®, and the Company
expects the main cost increases to be for personnel and associated expenses resulting from the increase in medical
representatives. The Company is planning to increase its workforce from 106 people at the end of 2019 to 152
people at the end of 2020, but is not planning any more significant increases in personnel thereafter.

We encourage readers to review our complete legal statement on “Disclaimer” page.

19 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Mid-Range Plan

Number of employees

Source: From the Company's results briefing material

From FY12/21 onward, profit exceeds ordinary profit. This is due to the fact that the Company reflected the impact
amount of the reduction of losses carried forward in conjunction with becoming profitable in tax-effect accounting.

Sales growth potential will be even larger if the global roll-out of
BCV proceeds well
3. Sales growth potential
Looking at sales growth potential, if marketing approval of TREAKISYM® for the indication of relapsed and refractory
DLBCL is obtained, the number of potential patients in Japan will roughly double all at once. Estimates of potential
sales will vary depending on what percentage is set for the market penetration rate. Excluding DLBCL, potential sales
are estimated at around ¥12bn to ¥13bn on a drug-price basis. If simply calculated, potential sales are expected toapproximately double to ¥24bn to ¥26bn by adding patients with relapsed and refractory DLBCL.
Moreover, if rigosertib is approved for an indication for untreated high-risk MDS, it can be expected to achievesales of around the same scale of azacitidine (approximately ¥15bn). The sales growth potential for both drugs
will increase from ¥8.5bn in 2018 to around ¥40bn on an NHI drug price basis, while the Company’s net sales willamount to more than ¥30bn, but the addition of BCV as a new pipeline drug has increased the growth potential
even more. As discussed above, BCV stands out for its strong anti-viral action against multiple DNA viruses, andthere are strong hopes for its development as a drug to treat viral infectious diseases following hematopoietic stem
cell transplantation and organ transplants. If its global roll-out is successful, the sales growth potential could exceed¥100bn, which makes it a drug of high interest going forward.

We encourage readers to review our complete legal statement on “Disclaimer” page.

20 21

COMPANY RESEARCH AND ANALYSIS REPORTFISCO Ltd.
http://www.fisco.co.jp

SymBio Pharmaceuticals Limited
4582 JASDAQ Growth Market

20-Apr.-2020
https://www.symbiopharma.com/ir_e/

Mid-Range Plan

Illustration of “The 2nd SymBio”

Source: From the Company's results briefing material

We encourage readers to review our complete legal statement on “Disclaimer” page.

21 21

Disclaimer
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FISCO Ltd.
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