On October 25, 2016 Onxeo S.A. (Euronext Paris, Nasdaq Copenhagen: ONXEO), an innovative company specialized in the development of orphan oncology therapeutics, reported its consolidated financials for the period ending September 30, 2016 and provided an update on major milestones achieved during the third quarter of 2016 (Press release, Onxeo, OCT 25, 2016, View Source [SID1234516029]).

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"The third quarter of 2016 was particularly eventful and productive for Onxeo. We made remarkable progress in terms of advancing our three key portfolio products as well as on the business development front. We announced results from two important preclinical studies, the first of which reinforces the rationale for developing Livatag as a potential new therapeutic option for HCC. Regarding the Livatag "ReLive" study, we are well on track to finalize the recruitment of patients in the near term, allowing the release of preliminary results in mid-2017 as planned. Data from another preclinical study confirmed the potential benefits of using AsiDNATM in combination with PARP inhibitors such as olaparib. This summer, we signed an exclusive licensing agreement with Pint Pharma for Beleodaq in South America, further expanding our product’s commercial potential. Lastly, our successful capital increase executed at the end of the third quarter has enabled us to increase our cash runway and strengthen our institutional shareholder base, including a number of US-based, specialized investors. We are well-equipped to address the opportunities expected in the coming months, as we work to deliver innovative therapeutic options that patients critically need, while creating value for our shareholders," commented Judith Greciet, CEO of Onxeo.

Continued advancement on key assets

Comprehensive preclinical and clinical work strengthening the products’ potential
Onxeo has progressed in the development of its key compound, Livatag (doxorubicin nanoformulation in Phase III trial for treatment of hepatocellular carcinoma). With more than 90% of the patients randomized as of September 30, 2016, the company is on track to deliver the preliminary results of the ReLive Phase III clinical study in mid-2017, in line with its development plan.

In an effort to expand the application of Livatag into other indications, Onxeo has also announced the first outcomes of its Livatag preclinical program, demonstrating enhanced efficacy effect in combination with immunotherapy, which validates its broader strategy for the product. Data from two in vivo studies have also confirmed the increased exposure and preferential affinity for the liver, supporting Onxeo’s current ReLive Phase III study rationale.

Onxeo has also made significant progress on Beleodaq, its pan-HDAC inhibitor already approved for PTCL (peripheral T-cell lymphoma). The company has started an initiative to develop an oral formulation of the compound, which would give a clear competitive advantage as well as expand the product’s potential application to indications for which such an oral formulation is appropriate. This development is on track, with prototypes designed and improved bioavailibity shown in an animal pharmacokinetic study.

Moreover, the company recently signed a promising new collaboration with the Royal College of Surgeons in Ireland (RCSI) for a research program on Beleodaq conjugate molecules, to improve product lifetime and stability properties, ultimately aiming to generate new patent opportunities.

Active preparation for the clinical development of first-in-class product AsiDNATM
Since the AsiDNATM acquisition, the Company has undertaken significant efforts to optimize the manufacturing process in terms of cost and duration, and is on track to manufacture its first clinical batch by the end of 2016, allowing for the initiation of a Phase I trial planned for 2017, after appropriate regulatory toxicologic assay.

The Company’s first objective is to show AsiDNATM activity when administered via the IV route, which would dramatically expand the potential of this compound. In parallel, preclinical research demonstrating the synergistic effect of Onxeo’s signal-interfering DNA product candidate in combination with various PARP (PolyADP-Ribose Polymerase) inhibitors has been published, confirming the interest of AsiDNATM compared to PARP inhibitors alone and the interest of the combination of these two DNA repair inhibitors.

Solid progress in business development and intellectual property

In the third quarter, Onxeo has strengthened its AsiDNATM intellectual property portfolio in the US with a new patent valid until 2031, confirming the innovative nature of the science behind its signal-interfering DNA product.

The company was also actively engaged in key operational and business development initiatives and achieved an important business development milestone, signing an exclusive license agreement with Pint Pharma for the commercialization of Beleodaq (belinostat) for PTCL in seven major South American countries.

Q3 revenue growth

Revenues for the third quarter of 2016 amounted to €1.23 million compared to €1.1 million in the third quarter of 2015 (+8%).

– €0.8 million of recurring revenues corresponding to product supplies to commercial partners and royalties on partners’ sales

– €0.4 million of non-recurring revenues, relating to the recognition under IFRS of upfront payments on certain licensing agreements

Over the first 9 months of the year, total revenues stood at €3.1 million, out of which €2.6 million were recurring revenues vs. €2.0 million in 2015 (+30%).

Long-term visibility reinforced with a successful €12.5 million capital increase

In early October, Onxeo successfully completed a capital increase of 5,434,783 new ordinary shares, raising gross proceeds of €12.5 million in a Private Placement. This capital increase strengthens and diversifies Onxeo’s shareholder base with the addition of prominent US-based healthcare institutional investors.

Proceeds from the capital increase, received on October 5, add to the €22.4 million consolidated cash balance at the end of September 2016, which extends Onxeo’s cash runway until Q2 2018. This capital will allow the company to pursue and accelerate the ongoing development of its pipeline assets, including the AsiDNATM and Livatag programs, as well as advance key preclinical programs, such as the combination therapy studies for AsiDNATM, Livatag, and Beleodaq.

Key near- and mid-term milestones

Livatag:
– Preclinical combination plan

– Next DSMB for Phase III trial: Q4 2016

– Preliminary Phase III trial results: expected mid-2017

AsiDNATM:
– Phase I initiation (monotherapy systemic) now expected in 2017, based on current CMC progress

Beleodaq:
– New oral formulation validated, ready to enter clinic: Q3 2017

– Preclinical combination study results: end of 2016 and onwards

– 1st-line PTCL Phase III initiation: end of 2016

On October 25, 2016 Baxter International Inc. (NYSE:BAX) reported results for the third quarter of 2016, and increased its earnings per share outlook for full-year 2016. Baxter’s third quarter worldwide sales totaled $2.6 billion, an increase of 3 percent on a reported basis and 4 percent on a constant currency basis as compared to the prior-year period (Press release, Baxter, OCT 25, 2016, View Source [SID1234515982]).

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"We are pleased with the continued strength across our core franchises as well as our improved financial performance, both of which are reflected in our third quarter results," said José (Joe) E. Almeida. "Given this progress, we are intensifying our focus on portfolio management and innovation to accelerate our efforts to introduce new products and therapies."

Financial Results

During the quarter, Baxter reported income from continuing operations of $127 million, or $0.23 per diluted share, on a GAAP (Generally Accepted Accounting Principles) basis. These results included net after-tax special items totaling $184 million, primarily related to business optimization initiatives, intangible asset amortization, debt extinguishment and Baxalta related spin-off costs.

On an adjusted basis, excluding special items, Baxter’s third quarter income from continuing operations totaled $311 million, or $0.56 per diluted share, exceeding the company’s previously-issued guidance of $0.43 to $0.45 per diluted share.

Baxter’s worldwide sales totaled $2.6 billion in the third quarter, an increase of 3 percent on a reported basis and 4 percent on a constant currency basis as compared to the prior-year period. Sales within the U.S. were $1.1 billion, advancing 6 percent, while international sales totaled $1.5 billion, representing a 1 percent increase on a reported basis, and an increase of 3 percent on a constant currency basis. Adjusting for the impact of foreign exchange, generic competition for cyclophosphamide, and 2015 PROTOPAM orders, Baxter’s sales increased 12 percent in the U.S. and rose 6 percent globally in the third quarter.

By business, Hospital Products sales of $1.6 billion in the third quarter increased 2 percent on a reported basis and 3 percent on a constant currency basis. Adjusting for the impact of foreign exchange, cyclophosphamide and PROTOPAM, Hospital Products sales advanced 7 percent from the prior-year period. Hospital Products performance in the quarter benefited from strong sales across the portfolio, driven by increased demand and favorable pricing in the U.S. for the company’s IV solutions, nutritional therapies, pharmacy injectables and IV access administration sets. Strength internationally in the company’s hospital pharmacy compounding services and cytotoxic contract manufacturing business also contributed to growth in the quarter.

Baxter’s Renal sales totaled $977 million in the third quarter, representing a 4 percent increase on a reported basis, and a 6 percent increase on a constant currency basis. Growth was driven by strong sales of peritoneal dialysis products as well as increased demand globally for continuous renal replacement therapies. Contributing to growth in the quarter were the recent launches of Baxter’s new Automated Peritoneal Dialysis (APD) systems, AMIA in the U.S. and HOMECHOICE CLARIA outside the U.S. Both of these systems utilize Baxter’s SHARESOURCE Connectivity Platform, which is the first and only two-way, remote patient management system for home dialysis therapy globally.

Financial Outlook

Based on the company’s strong performance in the first three quarters of the year, Baxter is raising its financial outlook for full-year 2016 including sales growth of approximately 2 percent on a reported basis or 4 percent on a constant currency basis, and earnings from continuing operations, before special items, of $1.88 to $1.91 per diluted share for the full year. Previous guidance called for reported sales of 1 to 2 percent (or 3 to 4 percent constant currency) and earnings of $1.69 to $1.74 per diluted share.

For the fourth quarter, the company expects sales growth of approximately 2 percent on both a reported and constant currency basis. Baxter expects earnings from continuing operations, before special items, of $0.49 to $0.52 per diluted share for the fourth quarter of 2016.

The earnings per share guidance for the fourth quarter and full-year 2016 excludes $0.05 and $0.22, respectively, per diluted share of intangible asset amortization expense; an estimated $0.01 and $0.07, respectively, per diluted share of Baxalta separation-related expense activities; an estimated $0.07 and $0.51, respectively, per diluted share of business optimization charges; and $7.84 per diluted share of asset impairment, debt extinguishment loss, product related reserve adjustments, and Baxalta retained stake gains for full-year 2016. These estimates are based on information reasonably available at the time of this release and future events or new information may result in different actual results. Reconciling for the inclusion of these items results in GAAP earnings of $0.36 to $0.39 per diluted share for the fourth quarter of 2016, and $8.92 to $8.95 per diluted share for full-year 2016.

A webcast of Baxter’s third quarter conference call for investors can be accessed live from a link on the company’s website at www.baxter.com beginning at 7:30 a.m. CDT on October 25, 2016.
Please see www.baxter.com for more information regarding this and future investor events and webcasts.

Baxter provides a broad portfolio of essential renal and hospital products, including home, acute and in-center dialysis; sterile IV solutions; infusion systems and devices; parenteral nutrition; biosurgery products and anesthetics; and pharmacy automation, software and services. The company’s global footprint and the critical nature of its products and services play a key role in expanding access to healthcare in emerging and developed countries. Baxter’s employees worldwide are building upon the company’s rich heritage of medical breakthroughs to advance the next generation of healthcare innovations that enable patient care.

A Single Arm, Open-Label, Multi-Centre, Phase I/II Study Evaluating the Safety and Clinical Activity of AUTO2, a CAR T Cell Treatment Targeting BCMA and TACI, in Patients with Relapsed or Refractory Multiple Myeloma

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On October 25, 2016 Array BioPharma (NASDAQ: ARRY) and Pierre Fabre reported that results from the Phase 3 COLUMBUS trial of binimetinib and encorafenib in BRAF-mutant melanoma will be presented at the 2016 Society for Melanoma Research (SMR) Annual Congress in Boston, Massachusetts on November 9 (Press release, Array BioPharma, OCT 25, 2016, View Source;p=RssLanding&cat=news&id=2215774 [SID1234515994]). Findings from COLUMBUS evaluating the combination of encorafenib plus binimetinib ("combination") in patients with unresectable or metastatic BRAF-mutant melanoma will be presented as an oral, late-breaking abstract. Two data analyses from the NEMO Phase 3 trial will also be presented, which evaluated binimetinib in patients with NRAS-mutant melanoma.

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COLUMBUS TRIAL DATA

Data from the Phase 3 study will be featured as an oral presentation during the late-breaking abstract session on Wednesday, November 9 from 10:00 – 10:15 AM ET:

Abstract 2617508: Results of COLUMBUS Part 1: A Phase 3 Trial of Encorafenib (ENCO) Plus Binimetinib (BINI) Versus Vemurafenib (VEM) or ENCO in BRAF-Mutant Melanoma
Presenter: Keith T. Flaherty, M.D., Director of the Termeer Center for Targeted Therapy, Massachusetts General Hospital and Professor of Medicine, Harvard Medical School, Boston, Massachusetts
As reported in late September, 577 patients were randomized 1:1:1 to receive the combination of encorafenib plus binimetinib, encorafenib alone, or vemurafenib alone. In the analysis of the primary endpoint, the median PFS for patients treated with the combination of encorafenib plus binimetinib ("combination") was 14.9 months versus 7.3 months for patients treated with vemurafenib; HR (0.54), [95% CI 0.41-0.71], p<0.001. Analysis of a secondary endpoint comparing the PFS of patients treated with combination to patients treated with encorafenib showed a median of 14.9 months versus 9.6 months with HR (0.75), [95% CI 0.56-1.00], p=0.051, which did not reach statistical significance. The combination was generally well-tolerated and reported adverse events were overall consistent with previous combination encorafenib plus binimetinib clinical trial results in BRAF-mutant melanoma patients.

ARRAY BIOPHARMA INVESTOR RECEPTION

Array will be hosting an investor reception immediately following the conclusion of SMR. The event will be held on Wednesday, November 9 from 1 – 3 pm ET in Boston and will include an encore presentation of the COLUMBUS results by Dr. Flaherty at 1 pm ET. The public is welcome to participate in the presentation through a webcast (live and replay): View Source

For questions regarding the reception, please contact Melissa Green/ConferenceSource at 303-325-8800 or [email protected].

About Metastatic Melanoma
Melanoma is the fifth most common cancer among men and the seventh most common cancer among women in the United States, with more than 76,000 new cases and over 10,000 deaths from the disease expected in 2016. Novel therapies that target the RAS/RAF/MEK/ERK pathway have a strong scientific rationale for activity in advanced melanoma, as up to 50 percent of patients with metastatic melanoma have activating BRAF mutations, the most common gene mutation in this patient population. Activating NRAS mutations are present in up to 20 percent of patients with metastatic melanoma, and is a poor prognostic indicator for these patients.

About Binimetinib & Encorafenib
MEK and BRAF are key protein kinases in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Research has shown this pathway regulates several key cellular activities including proliferation, differentiation, survival and angiogenesis. Inappropriate activation of proteins in this pathway has been shown to occur in many cancers, such as melanoma, colorectal and thyroid cancers. Binimetinib is a late-stage small molecule MEK inhibitor and encorafenib is a late-stage small molecule BRAF inhibitor, both of which target key enzymes in this pathway.

Binimetinib and encorafenib are being studied in clinical trials in advanced cancer patients, including the recently initiated Phase 3 BEACON CRC trial that is evaluating encorafenib in combination with cetuximab with or without binimetinib in patients with BRAF V600E-mutant colorectal cancer. Array submitted a New Drug Application (NDA) for binimetinib in NRAS-mutant melanoma to the FDA at the end of June 2016. The FDA accepted the NDA with a target action date under the Prescription Drug User Fee Act (PDUFA) of June 30, 2017. Array also expects to submit an NDA for binimetinib and encorafenib in BRAF-mutant melanoma to the FDA in 2017.

Array BioPharma retains exclusive rights to binimetinib and encorafenib in key markets including the U.S., Japan, Canada, Korea and Israel. Pierre Fabre will have exclusive rights to commercialize both products in all other countries, including Europe, Asia and Latin America.

On October 25, 2016 Horizon Pharma plc (NASDAQ:HZNP) a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, reported that it has completed the acquisition of Raptor Pharmaceutical Corp. (NASDAQ:RPTP) (Press release, Horizon Biopharm, OCT 25, 2016, View Source [SID1234515996]).

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"The acquisition of Raptor directly aligns with our long-term strategy and evolution into a rare disease focused company, where now more than half of our medicines are used to treat patients with rare diseases," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "The added infrastructure in Europe and other key international markets will further benefit the access to both our current and newly acquired medicines as well as position us for the potential introduction of ACTIMMUNE for Friedreich’s ataxia in Europe in future years if the results of the Phase 3 trial are positive."

Strategic rationale

Strengthens Horizon Pharma’s focus on rare diseases and provides expansion into Europe and other international markets.
Adds PROCYSBI (cysteamine bitartrate) 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.
Financial impact

Including the expected impact of the Raptor acquisition for the remainder of 2016, Horizon Pharma is raising its full-year 2016 net sales guidance on a GAAP basis, including the previously announced $65 million settlement with Express Scripts as a one-time reduction, to approximately $980 to $985 million. Horizon Pharma is raising its net sales guidance on a non-GAAP adjusted basis to approximately $1.045 to $1.050 billion excluding the $65 million settlement. The exclusion of the $65 million settlement from GAAP net sales guidance is the only adjustment reflected in Horizon Pharma’s full-year 2016 non-GAAP adjusted net sales guidance. Net sales from Raptor medicines for the last two months of 2016 are expected to add between $20 and $25 million to Horizon Pharma total net sales.
Including the expected impact of the Raptor acquisition for the remainder of 2016, Horizon Pharma is confirming its full-year 2016 adjusted EBITDA guidance of approximately $450 to $460 million.
As previously announced, Horizon Pharma expects the acquisition of Raptor to be accretive to adjusted EBITDA in 2017. Horizon Pharma will provide guidance for 2017 net sales and adjusted EBITDA in the first quarter of 2017.
Transaction details
The depositary for the tender offer has advised Horizon Pharma and Raptor that, as of the expiration of the tender offer at midnight, New York time, at the end of the day on October 24, 2016, a total of 71,590,496 shares of Raptor common stock were validly tendered and not validly withdrawn, representing approximately 83% of Raptor’s outstanding shares. In addition, the depositary advised that Notices of Guaranteed Delivery have been delivered with respect to 3,014,509 additional shares, representing approximately 3.5% of Raptor’s outstanding shares. All of the conditions to the offer have been satisfied and on October 25, 2016, Horizon Pharma and Misneach Corporation accepted for payment and will promptly pay for all shares validly tendered and not validly withdrawn prior to the expiration of the tender offer.

Following its acceptance of the tendered shares, Horizon Pharma completed its acquisition of Raptor through the merger of Misneach Corporation with and into Raptor without a vote of Raptor’s stockholders pursuant to Section 251(h) of the Delaware General Corporation Law. As a result of the merger, Raptor became an indirect wholly owned subsidiary of Horizon Pharma. In connection with the merger, all Raptor shares not validly tendered into the tender offer (other than shares owned by Raptor, Horizon Pharma, Misneach Corporation or any of their respective direct or indirect wholly owned subsidiaries and shares held by any person who was entitled to and has properly demanded statutory appraisal of his, her or its shares) have been canceled and converted into the right to receive the same $9.00 per share in cash, without interest (less any required withholding taxes) as will be paid for all shares that were validly tendered and not validly withdrawn in the tender offer. Raptor common stock will cease to be traded on the NASDAQ Global Select Market