IPO Report: uniQure (QURE)

Francis Gaskins |

uniQure (QURE) is a leader in the field of gene therapy and has developed the first and currently the only gene therapy product to receive regulatory approval in the European Union.

13 other companies are scheduled to IPO this week.  The full IPO calendar is available at IPOpremium.com

QURE scheduled a $64 million IPO with a market capitalization of $235 million at a price range midpoint of $235 for Wednesday, February 5, 2014, on the Nasdaq.

Manager, Joint managers: Jefferies, Leerink Partners, Piper Jaffray

Co-Managers: None
SEC Documents

Overview

QURE’s first product, Glybera, was approved by the European Commission in October 2012 under exceptional circumstances for the treatment of a subset of patients with lipoprotein lipase deficiency, or LPLD, a potentially life-threatening, orphan metabolic disease.

Valuation

Glossary

Cash burn rate:  when a company loses money with minimal depreciation & amortization (think biopharmas), the negative Price/Loss ratio is an indication of cash burn rate.  The higher the loss the lower the absolute (negative) ratio.  The lower the loss the higher the absolute (negative) ratio.

 

Valuation Ratios

Mrkt

Price /

Price /

Price /

Price /

% offered

annualizing Sept 9 mos

Cap (mm)

Sls

Erngs

BkVlue

TanBV

in IPO

uniQure B.V. (QURE)

$235

252.0

-6.3

4.4

13.5

27%

Conclusion
Positive,approved in European Union, shareholders not buying, high cash burn rate, see valuation below.

Business

QURE is a leader in the field of gene therapy and has developed the first and currently the only gene therapy product to receive regulatory approval in the European Union.

Gene therapy offers the prospect of long-term and potentially curative benefit to patients with genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration.

Glybera

QURE’s first product, Glybera, was approved by the European Commission in October 2012 under exceptional circumstances for the treatment of a subset of patients with lipoprotein lipase deficiency, or LPLD, a potentially life-threatening, orphan metabolic disease.

QURE expects to launch Glybera commercially in selected European countries in the first half of 2014 through its collaboration with Chiesi Farmaceutici S.p.A., or Chiesi, which QURE entered into in April 2013.

QURE retains full commercial rights to Glybera in the United States.

In August and December 2013, QURE met with the Food and Drug Administration, or FDA, to discuss the regulatory pathway for Glybera in the United States, and QURE plans to file an Investigational New Drug application, or IND, with the FDA for Glybera in the first half of 2014.

QURE is developing a pipeline of additional AAV-based gene therapies through multiple collaborations designed to accelerate the development and commercialization of these programs.

Manufacturing
QURE believes that its manufacturing facility in Amsterdam, which the European Medicines Agency, or EMA, has approved for clinical and commercial grade production, and QURE’s facility near Boston, Massachusetts, which QURE is currently building out and equipping, will enable QURE to produce Glybera and other gene therapies cost-effectively at commercial scale.

To fulfill the key conditions of the approval of Glybera by the EMA, QURE ise required to implement a patient registry prior to commercial launch and to complete an additional, post-approval clinical trial of Glybera, which QURE intends to commence in the second half of 2014.

The principal goal of these programs will be to obtain additional data regarding the safety, efficacy and clinical benefit of Glybera. QURE also believes that these programs will help QURE  to better define and target the LPLD patient population, as well as to raise awareness of LPLD and of Glybera in the clinician community.

Intellectual property
QURE’s manufacturing patent families contain issued patents in the United States, Europe and other territories, as well as numerous pending patent applications.

QURE has non-exclusively in-licensed from the NIH a patent family relating to the insect cell-based manufacturing of AAV-based vectors.

The patents in this family include two issued patents in the United States and one issued patent in Europe, as well as issued patents in other jurisdictions.

The standard 20-year term for patents in this family will expire in 2022. This patent family relates to technology used in Glybera and all of QURE’s development programs.

Competition

QURE is aware of several companies focused on developing gene therapies in various indications, including bluebird bio, Sangamo BioScience, AGTC, Oxford Biosciences, Spark Therapeutics, Audentes Therapeutics, RegenX and Asklepios, as well as several companies addressing other methods for modifying genes and regulating gene expression.

Although companies and research institutions in the gene therapy field tend to focus on particular target indications, any advances in gene therapy technology made by a competitor may be used to develop therapies competing against Glybera or one of QURE’s product candidates.

QURE may also face competition with respect to the treatment of some of the diseases that it is seeking to target with its gene therapies from protein pharmaceuticals under development at pharmaceutical and biotechnology companies such as Pfizer, Baxter, Bayer, Novo Nordisk, Genzyme, Shire, BioMarin, Biogen Idec and numerous other pharmaceutical and biotechnology firms.

5% stockholders

Entities affiliated with Forbion  35.5%

Cooperatieve Gilde Healthcare II U.A. 13.5%

Entities affiliated with Advent   6.2%

Coller International Partners V-A, L.P.  44.5%

Chiesi Farmaceutici S.p.A.  9.1%

Use of proceeds

QURE expects to net $56.4 million from its IPO. Proceeds are allocated as follows:

$15 million to complete the building out and equipping of QURE’s manufacturing facility in Lexington, Massachusetts;

$8 million to support QURE’s further clinical development of Glybera, and its application for marketing approval of Glybera and preparation for potential commercial launch in the United States;

$5 million to fund QURE’s share of the costs related to its planned Phase I/II clinical trial of AMT-060 in hemophilia B;

$11 million to advance the development of QURE’s other product candidates and research activities, including financing its collaboration with 4D Molecular Therapeutics; and

the remainder for working capital and for general corporate purposes, including the costs of operating QURE’s facilities in Amsterdam and Lexington, Massachusetts, service on its indebtedness and potentially for acquisitions or investments in other businesses, technologies or product candidates.

DISCLOSURE: The views and opinions expressed in this article are those of the authors, and do not represent the views of equities.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions. To read our full disclosure, please go to: http://www.equities.com/disclaimer

Companies

Symbol Name Price Change % Volume
QURE uniQure N.V. 5.68 -0.31 -5.18 651,060
CWYCF China Railway Constr Corp 1.28 0.00 0.00 0

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