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 Advaxis, Inc. (NASDAQ:ADXS), a clinical stage biotechnology company developing cancer immunotherapies, reported that it has welcomed New Jersey Governor Christopher J. Christie and BioNJ President and Chief Executive Officer Debbie Hart on Sept. 9 to celebrate the completion of its new state-of-the-art manufacturing facility and laboratory with a ribbon-cutting event before close to 200 people (Press release, Advaxis, SEP 12, 2016, View Source [SID:SID1234515095]).

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A photo accompanying this release is available at View Source

Advaxis built out the 30,000-square-foot expansion at its College Road East headquarters to include a clean-room manufacturing and laboratory facility to develop and manufacture clinical-grade products to support Advaxis’ clinical trials and future commercialization of its immuno-oncology therapeutics.

"I’m proud Advaxis is increasing its footprint and investment in the Garden State and specifically the Princeton Corridor, which in the past several years has grown as a beacon for world-renowned biotech and life sciences accomplishments," Governor Christie said. "For the benefit of New Jerseyans, Advaxis is emerging as a leading innovator in the biotech industry, rapidly developing state-of-the-art cancer treatment for patients in need, creating great private-sector jobs and attracting private investments from global companies who want to be a part of its budding successes."

Advaxis is conducting several clinical trials evaluating its proprietary Lm Technology using bioengineered live attenuated Listeria monocytogenes (Lm) bacteria to activate the immune system to attack cancer cells. In addition, the company announced a global collaboration agreement with Amgen for its My Immunotherapy Neo-Epitopes or MINE program for the development of ADXS-NEO, which will enter phase 1 trials in 2017.

"The Advaxis team believed in this technology and believed that it could help patients in need even when the company was struggling a few years ago, but just this year, the company has reported two complete responses in patients who have received AXAL in clinical trials, meaning the cancer in these two patients has disappeared," said Ms. Hart during the event. "Advaxis is a true New Jersey success story."

AXAL, or axalimogene filolisbac, is being tested in a Phase 3 clinical study called AIM2CERV to evaluate safety and efficacy in patients with high-risk, locally advanced cervical cancer. The U.S. Food and Drug Administration (FDA) has granted AXAL orphan drug designation as well as Fast Track designation for adjuvant therapy and a Special Protocol Assessment for the Phase 3 AIM2CERV trial. The trial is set to dose its first patient this year.

"Governor Christie’s administration has worked to help life science companies like Advaxis and many others succeed here in New Jersey, and his leadership in this field has benefited our state with jobs and economic development, and, more importantly, has facilitated Advaxis’ opportunity to develop its immunotherapies with the goal of helping cancer patients around the world for years to come," said Daniel J. O’Connor, president and CEO of Advaxis.

On September 12, 2016 IntelGenx Corp., (TSXV: IGX) (OTCQX: IGXT), reported that they have entered into a licensing, development and supply agreement with Chemo Group ("Chemo") granting Chemo the exclusive license to commercialize two generic products for the USA market and one product on a worldwide basis (Filing, 8-K, IntelGenx, SEP 12, 2016, View Source [SID:SID1234515092]).

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The Chemo Group is a privately held global pharmaceutical company with over 5,000 employees and operations in over 40 countries and revenues over $1.2 billion annually.

Under the terms of the agreement, Chemo has obtained certain exclusive rights to market and sell IntelGenx’ products in exchange for upfront and milestone payments, together with a share of the profits of commercialization. Chemo also has a right of first refusal to obtain the exclusive commercialisation rights for two of the products to include any country outside the USA.

"We are pleased to have established a partnership with a significant global pharmaceutical company," commented Dr. Horst Zerbe, President and CEO of IntelGenx. "This is the first step into a long term strategic alliance. We see the completion of this deal as further validation of our oral delivery platforms and our ability to attract high quality partnerships. This partnership is the beginning of what we believe will lead to further product agreements in expanding our global reach of our innovative product pipeline."

On September 12, 2016 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing new therapeutics and molecular diagnostics that target the underlying mechanisms of cancer, reported that it has submitted a formal response to the U.S. Food and Drug Administration (FDA) regarding the previously announced clinical hold of Aptose’s Phase 1b clinical trial of APTO-253 in patients with hematologic cancers (Press release, Aptose Biosciences, SEP 12, 2016, View Source;p=RssLanding&cat=news&id=2201082 [SID:SID1234515091]). Aptose provided responses to all of the questions cited in the clinical hold letter issued by the FDA.

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"This submission represents months of disciplined labor to resolve a manufacturing matter related to APTO-253 that arose during our Phase 1b Trial in patients with AML and high-risk MDS," commented Dr. William G. Rice, Chairman, President and Chief Executive Officer. "Although the FDA will make the ultimate decision whether our clinical trial may resume, all of their questions have been addressed."

During a Phase 1b clinical trial with APTO-253, a clinical site experienced stoppage of the infusion pump during an IV infusion caused by back pressure as a result of clogging of the in-line filter. The Company determined the root cause was a chemistry-based issue with the molecule, and the Company is now working with a drug product that does not cause filter clogging or pump stoppage during mock infusion studies performed to confirm the acceptability of the updated product through the clinical infusion procedures. Such improvements to the APTO-253 manufacturing process required to address the filter clogging event will be incorporated into a Chemistry, Manufacturing and Control (CMC) amendment to our Investigational New Drug application.

On September 12, 2016 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported that results from its on-going Phase 1b/2 trial of aldoxorubicin in combination with ifosfamide/mesna in patients with advanced sarcomas will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress being held in Copenhagen, Denmark, from October 7-11, 2016 (Press release, CytRx, SEP 12, 2016, View Source;p=RssLanding&cat=news&id=2201143 [SID:SID1234515090]).

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"Presenting the results from this trial at the ESMO (Free ESMO Whitepaper) Congress underscores the continued anti-tumor activity of aldoxorubicin across a variety of treatment settings for patients with advanced sarcomas," said Sant Chawla, M.D., F.R.A.C.P., the trial’s principal investigator and Director of the Sarcoma Oncology Center in Santa Monica, California. "The data show that aldoxorubicin can successfully be combined with ifosfamide and mesna, which are regularly given with doxorubicin to patients with first-line soft tissue sarcomas. Based on encouraging results, the trial was expanded to 50 patients and continues to enroll."

The Phase 1b/2 clinical trial has enrolled 38 patients to date with locally advanced, unresectable, and/or metastatic soft tissue sarcoma, intermediate-grade or high-grade chondrosarcoma or osteosarcoma. In the dose escalation phase, patients received either 170mg/m2 or 250mg/m2 of aldoxorubicin in combination with up to a 14-day continuous infusion of ifosfamide (1g/m2/day) plus mesna over a 28-day cycle. The expansion phase will enroll patients at the 250mg/m2 dose of aldoxorubicin and will allow for patients that had received prior chemotherapy to be included. The primary endpoint of the study is safety, and secondary endpoints include overall response rates and progression-free survival.

About Aldoxorubicin

Aldoxorubicin is a rationally-engineered cytotoxic which combines doxorubicin, a widely used chemotherapeutic agent, with a novel linker molecule that binds directly and specifically to circulating albumin, the most abundant protein in the bloodstream. Protein-hungry tumors concentrate albumin, which facilitates the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. Typically, doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 450 mg/m2. Using this acid-sensitive linker technology, aldoxorubicin delivers greater doses of doxorubicin (3 ½ to 4 times). To date, there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2,000 mg/m2. Aldoxorubicin is the first-ever single agent to show superiority over doxorubicin in a randomized clinical trial in first-line STS.