BioMarin & Prosensa Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock

BioMarin Pharmaceutical Inc. and Prosensa Holding N.V. recently announced they have entered into a definitive agreement in which BioMarin will offer to purchase all of the outstanding ordinary shares of Prosensa for $17.75 per share, for a total up front consideration of approximately $680 million. In addition, two approximately $80-million contingent milestones are payable for the approval of drisapersen in the US no later than May 15, 2016, and Europe no later than February 15, 2017, respectively.

“BioMarin is dedicated to the rare disease community, and the acquisition of Prosensa fits strategically with our mission of delivering therapies that address serious unmet medical needs,” said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. “We are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition.”

Mr. Bienaimé continued, “We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible. Further, if we are successful in advancing drisapersen to early regulatory approvals, we believe this transaction would be accretive to operating and GAAP profitability in 2017.”

Pat Furlong, President and Founder of Parent Project Muscular Dystrophy, said “BioMarin has a successful track record of developing new therapies for people with devastating disorders and for effectively collaborating with health authorities and patient communities. We look forward to working with BioMarin to bring new treatments to boys with Duchenne and other forms of muscular dystrophy.”

Under the terms of the definitive agreement, BioMarin will offer to acquire all of Prosensa’s issued and outstanding ordinary shares and all ordinary share equivalents in an all cash transaction for $17.75 per share for an upfront purchase price of approximately $680 million. Prosensa shareholders may also receive two regulatory milestone payments of approximately $80 million for receiving approval in the US no later than May 15, 2016, and in Europe no later than February 15, 2017, respectively. In addition, within 5 business days of signing the purchase agreement BioMarin will purchase from Prosensa a $50 million convertible note. If the transaction fails to close for any reason, the note will automatically convert into 4,395,914 shares of Prosensa’s stock.

The transaction is expected to be accounted for as a business combination. BioMarin will maintain operations at Prosensa’s headquarters, based in Leiden, The Netherlands and integrate Prosensa personnel from that office.

The acquisition will provide BioMarin with worldwide rights to multiple orphan-drug candidates, including drisapersen, which is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration. Prosensa’s pipeline is comprised of several potential products that leverage their proprietary RNA-modulating technology platform for the treatment of various genotypes of Duchenne muscular dystrophy and other genetic disorders.
Hans Schikan, Chief Executive Officer of Prosensa, added “BioMarin has established itself as a leader in rare diseases, characterized by strong management, thorough execution, and a resounding commitment to patients in developing and commercializing treatments in which there is a high unmet medical need. This transaction will enhance Prosensa’s mission by bringing innovative therapies to patients across the world as quickly and efficiently as possible. The deal also creates shareholder value by positioning Prosensa’s strong portfolio of orphan drug candidates for future success with a prominent rare disease company that has the experience and dedication to bring drisapersen and our follow-on compounds to the hands of patients who desperately need them.”

BioMarin will effect the transaction primarily through a tender offer for all of the issued and outstanding Prosensa ordinary shares (the Offer) and expect to close in the first quarter of 2015. The commencement of the Offer will be subject to having obtained workers council advice, and the consummation of the Offer is subject to the satisfaction of customary closing conditions for a transaction of this nature, including the tender of at least 80% of the issued and outstanding Prosensa ordinary shares and the receipt of regulatory clearance. Following completion of the Offer, the Supervisory Board of Prosensa will consist of five individuals designated by BioMarin and two individuals who currently serve on the Supervisory Board of Prosensa, who will act as independent directors. The two independent directors will, in accordance with Dutch practice, act as independent supervisory directors to protect the interest of any minority shareholders until BioMarin utilizes certain available reorganization structures available under Dutch law to acquire full ownership of Prosensa’s outstanding shares and/or its business. An Extraordinary General Meeting will be convened in connection with the Offer and to adopt, among other things, certain resolutions relating to the reorganization of Prosensa.

Synthetic oligonucleotides can bind to specific regions of pre-mRNA to induce exon skipping or exon inclusion, reduce mutated toxic RNA or protein, remove specific protein domains, or block protein expression. Exon skipping can restore the proper reading frame to the dystrophin mRNA, allowing shorter but still functional dystrophin protein to be produced, potentially maintaining or improving muscle function in people with DMD.

Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. Drisapersen has been administered to over 300 patients in seven clinical studies. It is generally well tolerated, with an adverse event (AE) profile consistent with this class of molecule. Drisapersen is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration.

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (about 20,000 new cases each year). DMD is caused by a mutation in the gene that encodes for dystrophin, a protein that is important in connecting the cytoskeleton of muscle fibers to the extracellular matrix. Its deficiency in DMD leads to progressive muscle weakness, loss of ambulation in early adolescence, and typically death due to pulmonary or cardiac insufficiency in the late twenties. Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures. There is currently no cure for DMD.

Prosensa N.V. is a biotechnology company based in Leiden, The Netherlands, which discovers and develops RNA-modulating therapeutics for the treatment of genetic disorders. The primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including DMD, myotonic dystrophy and Huntington’s disease.

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. For additional information, visit www.BMRN.com.