THE ORPHAN ONCOLOGY INNOVATOR - TopoTarget presentation - 21 05 2014.pdfTHE ORPHAN ONCOLOGY...

Creation of

THE ORPHAN ONCOLOGY INNOVATOR

A global leader in orphan oncology May 21st, 2014

2 – Investor Presentation – May 21, 2014

Forward-looking statements

This presentation contains certain forward-looking statements relating to the business of Topotarget and/or BioAlliance Pharma, including with respect to the progress, timing and completion of research, development and clinical trials for product candidates, the ability to manufacture, market, commercialize and achieve market acceptance for product candidates, the ability to protect intellectual property and operate the business without infringing on the intellectual property rights of others, estimates for future performance and estimates regarding anticipated operating losses, future revenues, capital requirements, and needs for additional financing. In addition, even if the actual results or development of Topotarget and/or BioAlliance Pharma are consistent with the forward-looking statements contained in this presentation, those results or developments of Topotarget and/or BioAlliance Pharma may not be indicative of their results or development in the future. In some cases, you can identify forward-looking statements by words such as "could," "should," "may," "expects," "anticipates," "believes," "intends," "estimates," "aims," "targets," or similar words. These forward-looking statements are based largely on the current expectations of Topotarget or BioAlliance Pharma as of the date of this presentation and are subject to a number of known and unknown risks and uncertainties and other factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievement expressed or implied by these forward-looking statements. In particular, the expectations of Topotarget and/or BioAlliance Pharma could be affected by, among other things, uncertainties involved in the development and manufacture of vaccines, unexpected clinical trial results, unexpected regulatory actions or delays, competition in general, currency fluctuations, the impact of the global and European credit crisis, and the ability to obtain or maintain patent or other proprietary intellectual property protection. In light of these risks and uncertainties, there can be no assurance that the forward-looking statements made during this presentation will in fact be realized. Topotarget or BioAlliance Pharma are providing such information as of the date of this presentation, and disclaim any intention or obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Important information

This presentation and the information contained in it do not constitute an offer to buy, sell or subscribe for any shares in Topotarget and/or BioAlliance Pharma in any country.

This communication is not a prospectus for the purpose of directive 2003/71/EC as amended (together with any applicable implementing measures in any member State, the “Prospectus Directive”). A prospectus to be prepared pursuant to the Prospectus Directive is intended to be published, which, when published, can be obtained from Topotarget and BioAlliance Pharma with certain limitations. Investors should not purchase or otherwise acquire any securities referred to in this communication except on the basis of information contained in a prospectus. Any decision to participate in the merger should be based on the full documentation to be published ahead of the general meetings.

The distribution, publication or release of this presentation may be prohibited or restricted by the laws or regulations applicable in certain countries. Persons who are physically present in those countries and in countries where the presentation is distributed, published, or released must comply with local restrictions. This announcement does not contain or constitute an offer of, or the solicitation of an offer to buy or subscribe for, securities to any person in the United States of America (the “United States”) or in any jurisdiction to whom or in which such offer or solicitation is unlawful. The securities referred to herein may not be offered or sold in the United States absent registration under the U.S. Securities Act of 1933, as amended (the “Securities Act”) or another exemption from, or in a transaction not subject to, the registration requirements of the Securities Act. The offer and sale of the securities referred to herein has not been and will not be registered under the Securities Act. The merging companies are European companies. Information distributed in connection with the proposed merger and the related shareholder vote is subject to European disclosure requirements that are different from those of the United States. Financial statements and information may be prepared according to accounting standards which may not be comparable to those used generally by companies in the United States. It may be difficult for you to enforce your rights and any claim you may have arising under the U.S. federal securities laws in respect of the merger, since the companies have their registered offices outside the United States. You may not be able to sue the companies or their officers or directors in a European court for violations of the U.S. securities laws. It may also be difficult to compel the companies and their affiliates to subject themselves to a U.S. court’s judgment.

Disclaimer


Creating Onxeo a Global Leader in Orphan Oncology

French innovative company specializing in the

development of drugs within orphan oncology

diseases

Danish biopharmaceutical company within orphan

oncology diseases

Vision: To be a global leader and pioneer in oncology, with a focus on orphan diseases, and to make the difference Mission:We are determined to develop innovative medicines to provide patients with hope and significantly improve their lives


2 companies dedicated to

orphan oncology

Complementary pipelines with

late-stage products

Experienced management

teams

Dedicated to the development of innovative therapies for orphan oncology diseases with significant unmet medical needs

Highlights of Transaction

Endorsed by the main shareholders of both companies

A global leader in orphan oncology

5 – Investor Presentation – May 21, 2014 5

Objectives of Transaction

Provide improved treatment options for patients and increased value creation for shareholders

1. Build Onxeo, a global leader in oncology focused on orphan diseases with significant unmet medical needs

2. Supported by a highly complementary pipeline of late-stage products with several significant value-creating events anticipated in both the short and medium term

3. Benefiting from operational efficiencies, combined knowledge-sharing, and a diversified revenue stream driving growth

4. Resulting in a broadened shareholders base with increased market capitalization and increased attractiveness to specialized investors

5. Under the leadership of an experienced management team and supervised by a highly skilled international Board of Directors with proven track records


Transaction Summary

• Creation of Onxeo through the merger of BioAlliance Pharma and Topotarget • Topotarget shareholders will receive 2 new BioAlliance Pharma shares for every 27 Topotarget

shares • Implying approximately 1/3 ownership for Topotarget and approximately 2/3 ownership for

BioAlliance Pharma • BioAlliance Pharma is listed on Euronext Paris and application will be made for a dual listing on

NASDAQ OMX Copenhagen

Governance

• Judith Greciet to be appointed CEO of Onxeo • Patrick Langlois to be appointed Chairman of the Board of Onxeo • Bo Jesper Hansen, current Chairman of Topotarget, and Per Samuelsson of HealthCap funds,

current Board Member of Topotarget, are expected to join the Board of Directors of Onxeo

Approvals and Expected Timing

• Definitive merger agreement unanimously approved by both BioAlliance Pharma’s and Topotarget’s Boards of Directors on May 21, 2014

• Merger to be approved by both Topotarget’s and BioAlliance Pharma’s shareholders at EGMs planned on June 27 and June 30, 2014, respectively

• Support and voting agreements from the largest shareholders Financière de la Montagne, Idinvest Partners, HBM Healthcare Investments and HealthCap funds

• Completion of the merger expected July/August 2014

Definitive Terms of the Merger


7

Orphan Oncology: Strategic Positioning in a High Value Market

Orphan oncology : A fast growing market

Rare Diseases: high unmet medical needs

• 7,000 rare or orphan diseases have been identified

• Treatments only exist for less than 5%

• Orphan oncology indications make up the largest and fastest growing group of diseases

Access to market facilitated by authorities

• More rapid development pathways

• Shorter review / approval cycles

• Favourable reimbursement environment for differentiated products

• Commercial exclusivities (US: 7 y. / EU: 10 y.)

$45.5Bn $80.6Bn

2013 2018

CAGR=12.1%

Source: EvaluatePharma


Overview of Topotarget 1

Business overview

• Established in 2000, listed on NASDAQ OMX Copenhagen

• Focused on late-stage clinical development in orphan oncology

• Cash Q1 FY14: DKK 109.7 million

• Upon NDA acceptance to file, recently received $10 million cash and 1 million Spectrum Pharmaceuticals (NASDAQ: SPPI) shares in Q1 2014

Strong expertise and innovation

• Lean and cost-efficient organization: 12 employees with strong clinical and regulatory experience

• Lead compound belinostat (HDAC inhibitor) tested in various indications

NDA under review for BeleodaqTM in PTCL in the USA

Potential new orphan oncology indications

• Strong partnership with Spectrum Pharmaceuticals (NASDAQ: SPPI)

• Highly skilled international Board of Directors and management


1 Topotarget’s Belinostat under FDA review for PTCL

Beleodaq® (Belinostat)

• An efficacious treatment addressing high unmet need in PTCL

• Positive Phase II trial showing efficacy and favorable safety profile

• Under Priority Review by FDA with expected response date early August 2014

• Long-term IP, until 2021 with possible term extension to 2026

• Represents a significant commercial opportunity

Peripheral T-cell lymphoma – PTCL Licensing Agreement

• A subtype of non-Hodgkin’s lymphoma (NHL) which affects T-cells

• Estimated global incidence of 12,000/year

• No approved treatments in EU in 2nd-line refractory/relapsed

• Only conditionally approved products available in the US in 2nd line

• Potential deal value $320 million

• Double-digit royalties + clinical, regulatory, and sales milestones

• Would receive $25 million upon FDA approval

• Spectrum exclusive territory: North America, India and a first right of negotiation for China

• Co-development of new indications with cost sharing Spectrum 70%/Topotarget 30%

• All product supply chain managed by Spectrum


Business overview

• Established in 1997 in France, listed on Euronext Paris since 2005

• Develops innovative treatments addressing high unmet needs in Orphan/Oncology diseases

• 2 advanced clinical programs and 2 supportive care registered/marketed products

• Cash FY13: €11.3 million

Overview BioAlliance Pharma 1

Strong expertise and innovation

• 50 employees (70% R&D) with full-range of expertise from preclinical to registration & business development

• 2 technological platforms (Lauriad® and Transdrug™)

• Fully proven expertise with 2 successful NDAs/ MAs

• Extensive IP portfolio

• Highly experienced management team and a highly skilled international Board of Directors


1 BioAlliance Pharma’s Pipeline Addressing High Unmet Medical Needs

Hepatocelullar Carcinoma (HCC)

• A nanoformulated doxorubicine, innovative therapy targeting survival in HCC

• International Phase III trial ongoing: 100+ patients already enrolled out of 400

• Fast track designation in US

• Worldwide intellectual protection until 2032

• Orphan status in both Europe and US

• Significant commercial opportunity with potential sales up to €800 million

• Most prevalent form of primary liver cancer

• 2rd cause of cancer-related deaths

• Highly treatment-resistant cancer

• Only one product approved in the market

LIVATAG®

Mechanism of action of Livatag®


1 BioAlliance Pharma’s Pipeline Addressing High Unmet Medical Needs

Oral mucositis

• A mucoadhesive anti-inflammatory tablet to prevent severe oral mucositis in head & neck cancer patients

• International Phase II trial completed in May 2014, results expected in 4Q14

• Strong IP protection in EU, Japan & US until 2029

• Orphan status in Europe, Fast Track designation in the US

• Significant commercial opportunity with potential sales up to €200 million to €400 million

• Side effects of cancer treatments – Radiotherapy

• Fairly frequent in head & neck cancers and frequently severe (65%)

• Leads to hospitalization and interruption of treatment

• No preventive treatment in head & neck cancer patients

VALIDIVE®

Severe mucositis

Validive® tablet


• Flexible and focused organization, operations in Paris and Copenhagen

• Reinforced news flow

• Highly complementary pipeline with 3 advanced programs (registration, Phase 3 & Phase 2)

• 3 technological platforms with multiple potential applications

• Proven track-record with 2 and soon 3 international approvals

• Commercial strategy: direct commercialization in Europe and partnering elsewhere

• Fully-aligned growth strategies based on orphan oncology pipelines with mid-to late-stage programs and potentially top selling products

• Consistent licensing/marketing strategies

• Highly experienced professionals

• Listed companies with long term shareholders and large retail base

BioAlliance Pharma And Topotarget: Strongly Complementary Companies 2


Product Ph 1 Ph 2 Ph 3 Registration Launch Milestones

Belinostat (PTCL)

PDUFA* (US) date Aug 2014

Combo BelCHOP (PTCL)

Livatag (HCC 2nd Line)

Ph 3 Read-out 2016

Validive

(Oral Mucositis)

Ph 2 preliminary data Q4 2014

NCI sponsored Studies Solid Tumors & Lymphomas In patients with Hepatic Dysfunction

Broad Late-Stage Orphan Oncology Portfolio O

n-g

oin

g 2

A promising pipeline with excellent mid-term perspectives: • Belinostat: beyond PTCL, 2 to 4 additional indications anticipated in hematology/solid tumors with a sales

potential > 250M€ worldwide • Livatag/ Validive: extended indications to enlarge their market value potential • * FDA decision on Belinostat


Operational efficiency

• Optimize R&D activities between teams

• Leverage key expertises between both teams

• Rationalize support services and general administration

Technologies/products • 3 internationally approved products

• HDACi • Lauriad (mucoadhesive tab.) • Transdrug (nanoformulation)

• 3 US and European licensed products

Partnership & licensing

• Strengthen further business development organization

• Leverage successful partnership with Spectrum Pharmaceuticals

• Build new partnership agreements on key programs

• Establish direct sales force in Europe

Complementary Strengths and Expertise to Create Value 3

Development

• Increase development capacity and optimize costs

• Initiate new promising indications within life cycle management

• Enhance assets’ value by enlarging development programs

• Create superior expertise in orphan oncology development


• Dual listing in Paris and Copenhagen

• Increased market capitalization

• Heightened international visibility

• Transaction supported by key shareholders of BioAlliance Pharma and Topotarget and unanimously recommended by both Boards of Directors

• Attractive to long term institutional and US and EU biotech investors

Strengthened Capital Market Proposition

Onxeo projected shareholding structure

4

Increased market capitalization

Financière de la Montagne/

Idinvest Partners 12,3% HealthCap Funds

/ HBM Healthcare

4,3%

Others 83,4%

€168m

€70m

BioAlliance Pharma Topotarget

Market data as of May 20, 2014


Highly skilled management team based in Paris with key individuals in Copenhagen

17

International Board of Directors reflecting the spirit of the merger

Experienced Management Team and Board of Directors 5

• Judith Greciet, 15 years in strategy and operations

• Executive committee with broad experience in clinical development, CMC, regulatory and quality as well as business development and finance

• Strong and committed teams with 70% dedicated to R&D

• Track record of bringing products through the FDA and EMA approvals process (Belinostat, Loramyc/Oravig, Sitavig)

• International Board of Directors chaired by Patrick Langlois (former CFO of Aventis SA), with the additions of Bo J. Hansen (Chairman of SOBI) and Per Samuelsson (HealthCap funds)

• Experienced individuals highly skilled in strategy, product development, pharma and biotech environment, external growth, M&A and financing


A winning player

• Operating in an attractive market: orphan oncology

• With a strong pipeline: 3 advanced development programs with multiple applications

• Proven expertise with accomplished teams

Bright future for additional value creation

• Significant short-term catalysts expected in 2H 2014:

Approval of belinostat in the US

Preliminary results of Validive phase II trial

• Heightened attractiveness for US and European-based investors

Bringing efficient and valuable treatments to patients with strong unmet medical needs

Onxeo, a Global Leader in Orphan Oncology


Definitive Timetable and Next Steps

May 21, 2014 Approval of definitive merger documentation by Boards of Directors, following exercise of Topotarget warrants

May 22-27, 2014

Filing of definitive merger plan with French and Danish authorities (post-exercise period of the Topotarget equity warrants)

Clearance statement (“enregistrement”) on merger prospectus (Document E) from French regulator and passport of merger prospectus to Denmark

Invitation to the Extraordinary General Meetings

June 27, 2014 Extraordinary General Meeting of Topotarget to approve merger

June 30, 2014 Extraordinary General Meeting of BioAlliance Pharma to approve merger

July/August 2014 Completion of the merger

THE ORPHAN ONCOLOGY INNOVATOR - TopoTarget presentation - 21 05 2014.pdfTHE ORPHAN ONCOLOGY...

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