On September 12, 2016 Horizon Pharma plc (NASDAQ:HZNP) and Raptor Pharmaceutical Corp. (NASDAQ:RPTP) reported the companies have entered into a definitive agreement under which Horizon Pharma will acquire all of the issued and outstanding shares of Raptor Pharmaceutical Corp. common stock for $9.00 per share in cash, for an implied fully diluted equity value of approximately $800 million (Press release, Horizon Pharma, SEP 12, 2016, View Source [SID:SID1234515096]). The transaction is expected to close in the fourth quarter of 2016.

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"The proposed acquisition of Raptor furthers our commitment to helping people with rare diseases and is a significant step in advancing our strategy to expand our rare disease business," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "Along with the potential for accelerated revenue growth, the addition of Raptor strengthens our U.S. orphan business and provides a platform to expand our orphan business in Europe and other key international markets. We look forward to working with new patient communities and building on the success of the Raptor team."

Strategic and financial benefits of the transaction:

Strengthens Horizon’s focus on rare diseases and provides expansion into Europe and other international markets.
Adds PROCYSBI delayed-release capsules and QUINSAIR (aerosolized form of levofloxacin) global rights, with PROCYSBI having strong patent protection through 2034.
Diversifies revenue with 11 medicines across three business units: orphan, rheumatology and primary care.
Bolsters rare disease revenue, which in the first half of 2016 on a pro-forma basis was 45 percent of total Horizon Pharma revenue.
Expected to be accretive to adjusted EBITDA in 2017.
"This transaction will deliver significant and immediate value to our shareholders through a compelling all-cash premium and provide ongoing value to our patients, their families and the physicians who treat them," said Julie Anne Smith, president and chief executive officer, Raptor Pharmaceutical Corp. "On behalf of the Board and management team, I extend our deepest gratitude to everyone at Raptor for their unrelenting commitment to advancing the development of our medicines and their tireless work with the patients we serve."

PROCYSBI is the first cystine-depleting agent given every 12 hours that is approved in the United States for the treatment of nephropathic cystinosis (NC), a rare metabolic disorder, in adults and children 2 years of age and older. PROCYSBI received European Commission approval as an orphan medicinal product in September 2013 for the treatment of proven NC. According to estimates, NC prevalence is as high as 1 in 100,000 live births. There are believed to be approximately 550 NC patients in the United States and 2,000 worldwide.

QUINSAIR is a proprietary inhaled formulation of levofloxacin, approved in the European Union and Canada for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis. Cystic fibrosis is a rare, life-threatening, genetic disease affecting an estimated 21,000 adults in Europe and Canada. QUINSAIR is not approved in the United States.

Raptor’s previously disclosed total net sales guidance for full-year 2016 is $125 million to $135 million, which includes both PROCYSBI and QUINSAIR. Horizon will provide additional detail regarding its guidance for full year 2017 net sales and adjusted EBITDA in the first quarter 2017.

Transaction Terms and Approvals
The acquisition is structured as an all cash tender offer for all the issued and outstanding shares of Raptor common stock at a price of $9.00 per share followed by a merger in which each remaining untendered share of Raptor common stock would be converted into the $9.00 per share cash consideration paid in the tender offer. The transaction, which has been unanimously approved by the boards of directors of both companies, is subject to the satisfaction of customary closing conditions and regulatory approvals, including antitrust approval in the United States.

Financing
Horizon intends to finance the transaction through $675 million of external debt along with cash on hand. The company has put in place fully committed financing with BofA Merrill Lynch, JPMorgan Chase Bank, N.A., Jefferies Finance LLC, and Cowen Structured Holdings, an affiliate of Cowen and Co. LLC. As of June 30, 2016, the company had $424.5 million of cash and cash equivalents on its balance sheet.

Advisors
MTS Health Partners L.P. and Citigroup Global Markets Inc. are co-lead financial advisors to Horizon Pharma in the transaction. BofA Merrill Lynch, J.P. Morgan, Jefferies LLC and Cowen and Company, LLC are financial advisors to Horizon Pharma in the transaction. Horizon Pharma’s legal advisors are Cooley LLP and McCann FitzGerald.

Centerview Partners LLC and Leerink Partners LLC are financial advisors to Raptor Pharmaceutical Corp. in the transaction. Raptor Pharmaceutical Corp.’s legal advisor is Latham & Watkins LLP.

On September 12, 2016 CirclePharma, Inc., reported that it will apply its computational design and synthetic chemistry platform to design and create a physical screening library of novel macrocyclicpeptides (Press release, Pfizer, SEP 12, 2016, View Source [SID1234525198]). Once completed, the library is initially expected to comprise several hundred macrocycles that will be designed to potentially disrupt bioactive conformations commonly found in protein-protein interactions known to drive disease processes, and will deploy backbone scaffolds screened in silica for intrinsic cell permeability characteristics. In addition, the design of the library will permit the simple creation of derivative libraries tailored to specific features of a therapeutic target class.

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PfizerInc.(NYSE:PFE) has entered into an agreement with Circle under which Pfizer will provide support for the library build, and Circle has granted Pfizer non-exclusive rights to screen the library against certaintargets. The rights granted to Pfizer exclude specified targets for which Circle has reserved exclusive rights to screen the library.

"This physical library will complement Circle’s target-specific computational design toolkit," said David J. Earp, J.D., Ph.D., Circle’s President and CEO. "We expect to use the library for our internal pipeline discovery work, and we will make it available to all of our collaboration partners in drug discovery."

About Macrocyclic Peptides

Macrocyclic peptides have the potential to provide access to the large proportion of therapeutic targets (estimated at up to 80%) that are considered undruggable with conventional small molecule or biologic modalities. Inparticular, there is great interest in developing macrocycles to modulate protein-protein interactions, which play a role in almost all disease conditions, including cancer, fibrosis, inflammation and infection. However, the development of macrocyclic therapeutics has been limited to this point by the need for a greater understanding of how to design macrocycles with appropriate pharm acokinetics, cell permeability and oral bioavailability. As a result, most clinical-stage macro cyclic peptide drugs address extracellular protein targets because of the challenge of identifying cell permeable macrocycles. The ability to design potent macrocycles with intrinsic permeability is expected to give access to a large number of important therapeutic targets that have been out of reach to this point.

On September 12, 2016 Cellectar Biosciences, Inc. (Nasdaq: CLRB) ("the company"), an oncology-focused biotechnology company, reported that its lead therapeutic compound, CLR 131, currently in a Phase 1 clinical trial for multiple myeloma and preparing for a Phase 2 study in multiple myeloma and other hematologic malignancies, will be evaluated by the University of Wisconsin in combination with external beam radiation as a potential combination treatment for head and neck cancers (squamous cell carcinoma) (Press release, Cellectar Biosciences, SEP 12, 2016, View Source [SID:SID1234515111]). The research will be conducted as part of a Specialized Program of Research Excellence (SPORE) grant, awarded to the University of Wisconsin by the National Cancer Institute.

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"The rigorous peer review that SPORE grants undergo provides further validation of the therapeutic benefits that CLR 131 could provide in both hematological and solid tumor malignancies. While we remain focused on advancing CLR 131 as a therapy for hematologic malignancies, we look forward to seeing the outcomes of the University’s research," said Jim Caruso, president and CEO of Cellectar Biosciences. "We are grateful for our long-standing relationship with the University of Wisconsin and congratulate them, and in particular, Dr. Paul Harari, chair of human oncology, who oversaw the SPORE grant application."

Earlier this year, Cellectar received a SBIR Fast Track award for CLR 131 from the NCI to conduct a Phase 2 clinical study in hematological malignancies. Additionally, Cellectar also received a patent for CLR 131 in combination with external beam radiation for a wide variety of cancers, including head and neck.

"We are excited to apply this promising new approach, which will allow us to simultaneously treat tumors from within using CLR 131 and from outside using external beam radiation," said Paul Harari, MD, FASTRO, Jack Fowler Professor and chairman, department of human oncology, University of Wisconsin School of Medicine and Public Health. "This combination may provide a powerful attack method against challenging solid tumors where radiation plays a central treatment role."

On September 12, 2016 PRA Health Sciences, Inc. (NASDAQ: PRAH) and Takeda Pharmaceutical Company Limited (TSE: 4502) reported that the companies have entered into a new partnership agreement under which PRA Health Sciences (PRA) will serve as Takeda’s primary strategic partner to deliver on the company’s pipeline and marketed products clinical development and post-approval needs (Press release, Takeda, SEP 12, 2016, View Source [SID:SID1234515124]).

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"This partnership is a fundamental part of Takeda’s R&D transformation and represents a truly innovative approach to clinical development, unprecedented in our industry," said Andy Plump, M.D., Ph.D., Director, Chief Medical and Scientific Officer at Takeda. "PRA has a reputation for providing tailored sourcing solutions. We believe PRA will be an ideal partner as we focus, deliver and advance our current and future pipeline."

The innovative partnership provides a flexible operating model that combines operational expertise, transferred from Takeda to PRA, with PRA’s wide range of global capabilities. This model is aimed to improve operating efficiencies, drive globalization and reduce fixed infrastructure costs. This flexible approach will facilitate the development of new medicines by focusing resources, teams and activities where needed, an important requirement for an industry-leading, highly agile R&D organization focused on meeting patient needs.

"This partnership is an exciting opportunity for PRA and represents a significant milestone and transformational business opportunity for us," said PRA Chief Executive Officer Colin Shannon. "This is the first time that a pharmaceutical company and a clinical research organization (CRO) have come together to create such a comprehensive business and operational strategy. This partnership model redefines collaboration and is the first of its kind in the CRO industry."

The partnership will enable PRA to utilize its internal resources and expertise to manage an entire pipeline of studies for Takeda, across Phases I-IV and provide Regulatory, Pharmacovigilance and other operational services for both development and marketed product portfolios. The transformation is expected to result in approximately 300 Takeda employees supporting drug development and marketed products to be given the opportunity to transition to PRA in the United States and Europe, subject to appropriate information and consultation with works councils, unions, and employee representatives. Discussion regarding Japan employees is ongoing between Takeda and PRA.

On September 12, 2016 Circle Pharma, Inc., reported that it will apply its computational design and synthetic chemistry platform to design and create a physical screening library of novel macrocyclicpeptides (Press release, Circle Pharma, SEP 12, 2016, View Source [SID1234635670]). Once completed, the library is initially expected to comprise several hundred macrocycles that will be designed to potentially disrupt bioactive conformations commonly found in protein-protein interactions known to drive disease processes, and will deploy backbone scaffolds screened in silica for intrinsic cell permeability characteristics. In addition, the design of the library will permit the simple creation of derivative libraries tailored to specific features of a therapeutic target class.

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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PfizerInc.(NYSE:PFE) has entered into an agreement with Circle under which Pfizer will provide support for the library build, and Circle has granted Pfizer non-exclusive rights to screen the library against certaintargets. The rights granted to Pfizer exclude specified targets for which Circle has reserved exclusive rights to screen the library.

"This physical library will complement Circle’s target-specific computational design toolkit," said David J. Earp, J.D., Ph.D., Circle’s President and CEO. "We expect to use the library for our internal pipeline discovery work, and we will make it available to all of our collaboration partners in drug discovery."

About Macrocyclic Peptides

Macrocyclic peptides have the potential to provide access to the large proportion of therapeutic targets (estimated at up to 80%) that are considered undruggable with conventional small molecule or biologic modalities. Inparticular, there is great interest in developing macrocycles to modulate protein-protein interactions, which play a role in almost all disease conditions, including cancer, fibrosis, inflammation and infection. However, the development of macrocyclic therapeutics has been limited to this point by the need for a greater understanding of how to design macrocycles with appropriate pharm acokinetics, cell permeability and oral bioavailability. As a result, most clinical-stage macro cyclic peptide drugs address extracellular protein targets because of the challenge of identifying cell permeable macrocycles. The ability to design potent macrocycles with intrinsic permeability is expected to give access to a large number of important therapeutic targets that have been out of reach to this point.