| Company announcement | Orphazyme A/S |
| No. 1/2017 | Ole Maaløes Vej 3 |
| DK-2200 Copenhagen N | |
| www.orphazyme.com (http://www.orphazyme.com/) | |
| Company Registration No. 32266355 | |
| 24 October 2017 |
NOT FOR RELEASE OR DISTRIBUTION OR PUBLICATION IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN OR INTO THE UNITED STATES, AUSTRALIA, CANADA OR JAPAN
This announcement does not constitute an offering circular and nothing herein contains an offering of securities. No one should purchase or subscribe for any securities in Orphazyme A/S ("Orphazyme" or the "Company") except on the basis of information in any offering circular published by the Company in connection with the potential offering and admission of such securities to trading and official listing on Nasdaq Copenhagen A/S ("Nasdaq Copenhagen"). Copies of any such offering circular will, following publication, be available at the Company's registered office and, subject to certain exceptions, through the website of the Company.
Orphazyme announces intention to launch an Initial Public Offering
Orphazyme, a Danish biotech company with a late stage orphan drug pipeline, announces its intention to launch an Initial Public Offering ("IPO" or "Offering") of its shares and to apply for admission to trading and official listing on Nasdaq Copenhagen.
The intended IPO is expected to consist of an issue of new shares to raise gross proceeds of approximately DKK 600 million to support the Company's strategy by primarily funding on-going and planned clinical trials within four orphan diseases. The total offer size will be announced in connection with the publication of a prospectus by the Company.
Orphazyme is currently owned by Novo Holdings A/S, Coöperative Aescap Venture I U.A., Sunstone Life Science Ventures Fund II K/S, Orpha Pooling B.V. (a joint venture between LSP V Coöperatieve U.A. and ALS Invest 2 B.V.), certain funds managed by Idinvest and Kurma Biofund II and other minority shareholders including members of senior management and employees. Prior to publication of the prospectus, the Company's existing share classes will be merged as part of a reorganisation of the current capital structure. The exact ownership stakes of the current shareholders after the reorganisation of the capital structure will depend on the final offer price in the IPO. None of the current shareholders have expressed an intention to sell shares in connection with the IPO and will continue as shareholders in Orphazyme in addition to undertaking lock-up obligations in connection with the IPO
Anders Hinsby, CEO of Orphazyme, said:
"Since the foundation in 2009, we have been able to establish a Danish biotech company with a product candidate for the treatment of four severe orphan diseases. We have an extensive news flow coming up and expect to have completed three phase II/III trials by the end of 2020 with the first potential marketing authorisation in 2020. We have, for a longer period of time, had many meetings with domestic and international investors and we are very pleased with the feedback received."
Georges Gemayel, Chairman of Orphazyme, said:
"Today's announcement marks an important step in Orphazyme's strategic journey. We look forward to inviting new shareholders to take part in the future ownership and to join us in our journey to assist patients with a significant unmet medical need."
About Orphazyme
Orphazyme is a Danish biotech company with a late stage orphan drug pipeline, developing new treatment options for orphan protein misfolding diseases. It was founded in 2009 based on early scientific discovery in heat shock proteins ("HSPs"). Since inception, the Company has translated the scientific discovery into a late stage clinical development programme.
The Company focuses on severe and mostly fatal diseases with a high unmet need, and with a particularly strong commitment to neuromuscular diseases and a group of severe genetic diseases called lysosomal storage diseases. The Company plans to pursue development of its lead candidate through to registration in Europe and the United States after which launch and commercialisation is expected to be undertaken by the Company.
The lead candidate arimoclomol is in development as a potential treatment for four orphan diseases; two neuromuscular diseases, sporadic Inclusion Body Myositis ("sIBM") and Amyotrophic Lateral Sclerosis ("ALS"), and two lysosomal storage diseases, Niemann Pick type C ("NPC") and Gaucher disease. Across phase I trials and phase II trials in sIBM and SOD1-ALS (a subtype of ALS), arimoclomol has been well tolerated with no significant safety risks identified to date. The phase II trials have shown consistent trends of treatment benefit. In addition, the clinical trials in NPC and Gaucher disease are pursuant to phase I trials in healthy volunteers and pre-clinical studies showing benefit of arimoclomol.
Arimoclomol stimulates the body's own production of heat shock proteins in cells experiencing stress or toxicity. HSPs form the core of the cells' protein rescue system and guard against the toxicity arising from misfolded proteins by supporting correct folding of mutated proteins and the cell's recycling system.
The Company is headquartered in Copenhagen and has 28 employees.
About orphan drugs
Orphan drugs denote pharmaceutical products that target rare, often severe diseases affecting a small number of people. The diseases are in general serious, debilitating and often fatal if not treated.
Following the Orphan Drug Act in the United States in 1983, implementation of orphan drug regimes have taken place in other key markets as well, for example Japan in 1993 and the EU in 2000. The Orphan Drug Act was set up to financially incentivise the development of pharmaceutical products for severe diseases affecting so few individuals that it would not be considered profitable otherwise.
Developing treatments for orphan diseases present a series of advantages for the drug developers during the development and marketing phase, including regulatory support incentives, likely less competition and high unmet medical need, higher clinical development success rate, generally lower development and commercialisation costs as patient groups are small, potential market exclusivity and usually higher pricing of products.
Project Pipeline
Orphazyme has potential registration studies in several indications ongoing or in the planning. The first indication for arimoclomol is expected to be the treatment of NPC. The results of the already initiated clinical trial in NPC are expected in Q3 2018 with potential marketing authorisation in H1 2020. In Q3 2017 a sIBM phase II/III clinical trial was initiated. Additionally, Orphazyme expects to have initiated clinical studies in the remaining indications, ALS and Gaucher disease, by the end of 2018. Further, the Company has developed expertise to assess new leads and have identified a number of new molecular entities ("NMEs") which may generate clinical candidates.
The figure below provides an overview of the expected news flow until 2020, subject to completion of the Offering in order to fund the clinical trials currently ongoing and in planning.
Strategy
Orphazyme's strategy is to develop treatments for orphan diseases with protein misfolding where it can apply its specialised know-how in HSPs. Important elements of Orphazyme's strategy are the following:
- Advance the development of arimoclomol for the treatment of sIBM, ALS, NPC and Gaucher disease by completion of clinical development programmes
- Design the commercialisation and go-to-market strategy for arimoclomol
- Develop new molecular entities for other protein misfolding diseases based on current technology platform
Orphazyme's key strengths
Late stage project pipeline with several potential registration studies ongoing or in planning
Orphazyme is developing arimoclomol in four indications. Orphazyme has fully recruited a phase II/III trial in NPC (trial results expected Q3 2018), initiated a phase II/III trial in sIBM in August 2017 and plans to start a phase II/III trial in ALS in H2 2018. In addition, a phase II proof-of-concept trial in Gaucher disease will be initiated in Q2 2018[1]. If positive, all three phase II/III trials are intended to form the basis for a single study filing in each respective indication. The Company expects to have completed all three phase II/III trials by the end of 2020 with the first potential marketing authorisation in 2020.
Careful choice of target indications, based on insight into the function of HSPs and optimisation for clinical success by focusing on diseases with well-defined patient populations, disease pathology and/or genetics
Orphazyme aims to enhance development success rates by choosing target patient populations with well-defined pathology and/or genetics that are expected to respond more uniformly to treatment and by using clinical and biochemical parameters to select and stratify patients for the clinical trials. A tightly defined patient population increases the likelihood of robust signal detection in a clinical trial and to optimise chances of clinical success. Orphazyme therefore specifically pursues rare disease indications instead of more common diseases.
Technology platform shows potential with clinical proof-of-concept1
Orphazyme's lead program, arimoclomol, has been studied in a number of pre-clinical experiments allowing it to be characterised in a variety of different disease models and pathologies, many of which are published in leading peer-reviewed scientific journals including Nature Medicine and Science Translational Medicine. The ability of arimoclomol to mobilise HSPs and provide benefit have been validated across several protein misfolding diseases including relevant animal models for NPC, ALS and sIBM. Moreover, the benefit of arimoclomol has been established and characterised in cells from patients diagnosed with NPC and Gaucher disease across a range of mutations.
Technology protection with active patenting strategy aimed at maintaining Orphazyme's technology position
Since its founding, Orphazyme has strategically and actively pursued patent protection of its inventions. Orphazyme now holds the rights to a comprehensive patent portfolio and continues to actively pursue further patent protection and exclusivity opportunities. Orphazyme focuses on protecting small molecule inducers of HSPs, including the main candidate arimoclomol, as well as new molecular entities.
Experienced management team with relevant and complementary skill set
Orphazyme's senior management team has experience from the biotech and pharmaceutical industries with a broad range of skills to succeed in the industry, including experience with research and development, finance and intellectual property rights. The Company's CEO and CSO have been with the Company since its inception.
Bank Syndicate
Carnegie and Danske Bank are acting as Joint Global Coordinators and Joint Bookrunners, and Oddo BHF SCA is acting as Co-Lead Manager for the proposed IPO.
For additional information, please contact
Orphazyme
Anders Hinsby, CEO +45 31 44 31 39
Impact Partners
Michael Steen-Knudsen +45 25 17 18 15
Important notice
This announcement is not an offer to sell or a solicitation of any offer to buy any securities issued by Orphazyme A/S ("Orphazyme" or the "Company") in any jurisdiction where such offer or sale would be unlawful and the announcement and the information contained herein are not for distribution or release, directly or indirectly, in or into such jurisdictions, including but not limited to, the United States, Australia, Canada and Japan.
This document does not constitute, or form part of, an offer to sell, or a solicitation of an offer to purchase, any securities in the United States. The securities referred to herein have not been and will not be registered under the U.S. Securities Act of 1933, as amended (the "Securities Act") and may not be offered or sold within the United States absent registration or to an exemption from, or in a transaction not subject to, the registration requirements of the Securities Act. There is no intention to register any securities referred to herein in the United States or to make any offering of the securities in the United States.
In any member state of the European Economic Area ("EEA Member State"), other than Denmark, that has implemented Directive 2003/71/EC as amended (together with any applicable implementing measures in any member State, the "Prospectus Directive"), this communication is only addressed to and is only directed at investors in that EEA Member State who fulfil the criteria for exemption from the obligation to publish an offering memorandum, including qualified investors, within the meaning of the Prospectus Directive as implemented in each such EEA Member State.
This announcement is only being distributed to and is only directed at (i) persons who are outside the United Kingdom or (ii) to investment professionals falling within Article 19(5) of the U.K. Financial Services and Markets Act 2000 (Financial Promotion) Order 2005 (the "Order") or (iii) high net worth entities falling within Article 49(2)(a) - (d) of the Order (the persons described in (i) through (iii) above together being referred to as "relevant persons"). The securities are only available to, and any invitation, offer or agreement to subscribe, purchase or otherwise acquire such securities will be engaged in only with, relevant persons. Any person who is not a relevant person should not act or rely on this document or any of its contents.
This announcement does not constitute an offering memorandum and nothing herein contains an offering of securities. No one should purchase or subscribe for any securities in the Company, except on the basis of information in the offering memorandum published by the Company in connection with the potential offering and admission of such securities to trading and official listing on Nasdaq Copenhagen A/S. Copies of the offering memorandum will, following publication be available from the Company's registered office and, subject to certain exceptions, on the website of the Company.
Carnegie Investment Bank, filial af Carnegie Investment Bank AB (publ), Sverige and Danske Bank A/S (together the "Joint Global Coordinators and Joint Bookrunners") and Oddo BHF SCA ("Co-Lead Manager" and together with the Joint Global Coordinators and Joint Bookrunners, the "Managers") and their affiliates are acting exclusively for the Company and no-one else in connection with the potential offering. They will not regard any other person as their respective clients in relation to the potential offering and will not be responsible to anyone other than the Company for providing the protections afforded to their respective clients, nor for providing advice in relation to the potential offering, the contents of this announcement or any transaction, arrangement or other matter referred to herein.
In connection with the potential offering, the Managers and any of their affiliates, acting as investors for their own accounts, may subscribe for or purchase shares and in that capacity may retain, purchase, sell, offer to sell or otherwise deal for their own accounts in such shares and other securities of the Company or related instruments in connection with the potential offering or otherwise. Accordingly, references in the offering memorandum, if published to the shares being issued, offered, subscribed, acquired, placed or otherwise dealt in should be read as including any issue or offer to, or subscription, acquisition, placing or dealing by, such Managers and any of their affiliates acting as investors for their own accounts. The Managers do not intend to disclose the extent of any such investment or transactions otherwise than in accordance with any legal or regulatory obligations to do so.
Matters discussed in this announcement may constitute forward-looking statements. Forward-looking statements are statements that are not historical facts and that can be identified by words such as "believe", "expect", "anticipate", "intends", "estimate", "will", "may", "continue", "should", and similar expressions. The forward-looking statements in this announcement are based upon various assumptions, many of which are based, in turn, upon further assumptions. Although the Company believes that these assumptions were reasonable when made, these assumptions are inherently subject to significant known and unknown risks, uncertainties, contingencies and other important factors that are difficult or impossible to predict and are beyond its control. Such risks, uncertainties, contingencies and other important factors could cause actual events to differ materially from the expectations expressed or implied in this release by such forward-looking statements. The information, opinions and forward-looking statements contained in this announcement speak only as at its date, and are subject to change without notice.
[1] The Company considers "proof-of-concept" to be benefit across efficacy endpoints and assessments as compiled evidence that constitutes proof-of-concept, also in absence of statistical significance
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Orphazyme ApS via Globenewswire
