On May 24, 2016 Sandoz, a Novartis division and the global leader in biosimilars, reported that the European Medicines Agency has accepted their Marketing Authorization Application for a biosimilar to Roche’s EU-licensed MabThera (rituximab) (Press release, Novartis, MAY 24, 2016, View Source [SID:1234512719]). Rituximab is a monoclonal antibody that is used to treat non-Hodgkin’s lymphoma, which includes follicular lymphoma and diffuse large B-cell lymphoma, chronic lymphocytic leukemia and autoimmune diseases such as rheumatoid arthritis. Sandoz is seeking approval for the same indications as the reference product.

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"Patients with hematologic or blood cancers and rheumatoid arthritis, as well as their doctors, often have few treatment options and have long relied on rituximab as a vital part of their treatment," said Richard Francis, Division Head and CEO of Sandoz. "If approved, we believe our biosimilar rituximab will help broaden access to this important therapy and liberate healthcare resources that can be used to fund other innovative medicines."

Sandoz believes that the totality of data in its submission will demonstrate that our biosimilar rituximab has essentially the same biological substance as, and the final drug product is highly similar to, the reference product. In addition to analytical, functional and pre-clinical data, the submission includes data from two pivotal confirmatory safety, PK/PD and efficacy studies that involved 629 follicular lymphoma and 173 rheumatoid arthritis patients.

Sandoz is committed to increasing patient access to high-quality, life-enhancing biosimilars. It is the pioneer and global leader in biosimilars and currently markets three biosimilars. Biosimilar rituximab is part of Sandoz’ growing oncology and immunology portfolios. The oncology portfolio includes two marketed products (filgrastim and epoetin-alfa) and biosimilar candidate pegfilgrastim, which is under regulatory review in the US and EU. Today’s announcement marks the sixth of 10 regulatory filings that the company plans to submit over a three-year period (2015-2017). As a division of the Novartis Group, Sandoz is well-positioned to lead the biosimilars industry based on its experience and capabilities in development, manufacturing and commercialization.

On May 24, 2016 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported an update on its ongoing OVATION study, a Phase Ib dose escalating clinical trial combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery (Press release, Celsion, MAY 24, 2016, View Source [SID:1234512740]). GEN-1 is an IL-12 DNA plasmid vector formulated as a nanoparticle in a non-viral delivery system to cause the sustained local production and secretion of the Interleukin-12 (IL-12) protein loco-regionally to the tumor site.

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The Company announced that an abstract detailing the trial design of the OVATION Study has been accepted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which will take place from June 3-7 in Chicago. The abstract, entitled "Phase 1 study of the safety and biological activity of intraperitoneal IL-12 plasmid formulated with PEG-PEI-cholesterol lipopolymer administered in combination with standard neoadjuvant chemotherapy (NAC) in patients with newly diagnosed ovarian cancer," will be presented in a poster session on Monday, June 6th from 1:00 PM to 4:00 PM.

The Company also announced today that it has completed enrollment of the second cohort of the OVATION Study and expects to report clinical data from that cohort mid-year. Celsion has previously reported highly encouraging data from the first cohort of the OVATION Study. In the first three patients dosed, GEN-1 plus standard chemotherapy produced excellent results, with no dose limiting toxicities and highly promising efficacy signals leading to successful surgical outcomes as summarized below:

Of the three patients treated in the first cohort, two patients demonstrated stable disease (SD) and one patient demonstrated a complete response (CR), as measured by RECIST criteria.
All patients had successful resections of their tumors, with two patients having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed, and one patient with Stage IV ovarian cancer having an optimal R1 resection.
One patient demonstrated a pathological complete response (pCR). pCRs are typically seen in less than 7% of patients receiving neoadjuvant chemotherapy followed by surgical resection, and have been associated with a median overall survival (OS) of 72 months, which is more than three years longer than those who do not experience a pCR.
All patients experienced a dramatic > 96% drop in their CA-125 protein levels as of their most recent study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells. A 50% reduction in CA-125 levels is considered meaningful. All patients’ CA-125 levels were below the standard cutoff level of 35 U/mL.
Celsion also expects to report translational data from the first two cohorts of the OVATION Study early in the third quarter of 2016. The translational data will provide further insight into GEN-1’s mechanism of action through the evaluation of dose-related changes in the tumor and peritoneal immune cell population, as well as through the peritoneal cytokine levels. Moreover, immune cell density, a common indicator of survival in ovarian cancer patients, will be explored to further demonstrate the therapy’s efficacy in these patients.

"We continue to successfully execute our OVATION Study in newly diagnosed ovarian cancer patients, and are pleased to report that our second cohort is now fully enrolled," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "The translational and clinical data seen to date underscore the potential efficacy and safety profile of GEN-1, and we look forward to learning more about the utility of our gene-based immunotherapy approach as our data set matures."

OVATION Study Design

The Phase Ib trial will evaluate weekly intraperitoneal dosing of GEN-1 in combination with neoadjuvant chemotherapy, the standard of care for patients newly diagnosed with ovarian cancer. Concurrently with neoadjuvant chemotherapy, enrolled patients will receive escalating weekly doses of GEN-1, from levels beginning at 36mg/m², to 47mg/m², 61mg/m², 79mg/m², and up to 103mg/m² weekly for 8 treatments in total, with interval debulking surgery to follow. The regimen will primarily be evaluated for its safety and tolerability.

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy in patients with peritoneally metastasized ovarian cancer, and recently completed a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer.

On May 24, 2016 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported the publication of an abstract for a poster session at the 2016 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, Illinois (Press release, Ignyta, MAY 24, 2016, View Source [SID:1234512753]). The abstract relates to the results of the Phase 1 clinical trial of RXDX-105, the company’s orally available, small molecule multikinase inhibitor with potent activity against such targets as RET and BRAF. The poster session will be held on Sunday, June 5, 2016.

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"We are honored that the ASCO (Free ASCO Whitepaper) Scientific Program Committee has selected the abstract describing exciting data from our Phase 1 clinical trial of RXDX-105 for a poster presentation," said Pratik Multani, M.D., Chief Medical Officer of Ignyta. "We are looking forward to sharing updated data from this clinical trial in this prestigious scientific forum."

Details of the presentation are as follows:

Title:
A phase 1 dose escalation study of RXDX-105, an oral RET and BRAF inhibitor, in patients with advanced solid tumors. (Abstract number 2574, Poster number 274)
Date/time: Sunday, June 5, 2016, 8:00 AM – 11:30 AM, Central time

On May 24, 2016 Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) reported its Interim Report for the nine months ended March 31, 2016. The report includes the financial results and a review of operations for the period (Press release, Benitec Biopharma, MAY 24, 2016, View Source [SID:1234512755]).

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Summary of the key points from the Interim Report:
• The net loss for the nine months was $A18.5 million and included research and development spending of AU$11.1 million. Benitec’s current assets at March 31, 2016 were A$26.5 million.

• In March 2016 Benitec announced encouraging results of its recent in vivo efficacy study of BBHB-331. Key findings indicate that a single BB-HB-331 treatment in the PhoenixBio mouse model can result in suppression of hepatitis B (HBV). These results demonstrate the potential utility of an approach that combines RNAi with gene therapy to treat HBV, and the Company intends to advance the HBV program towards the clinic.

• On February 26, 2016 Benitec announced that it would wind-down its hepatitis C program and terminate the program upon completion of patients in Cohort 4 in its Phase I/IIa clinical trial for TT-034.

• In December 2015, Benitec announced positive in vitro data demonstrating the efficacy of BBHB-331 and supporting the progression of BB-HB-331 into in vivo preclinical testing. The data was presented at the HEPDART 2015 conference in the US in December 2015.

• In August 2015, Benitec completed a NASDAQ listing raising A$18.8 million (US$13.8 million) before costs.

• In July 2015, Benitec announced it acquired full rights to its preclinical DNA-directed RNA interference based hepatitis B therapeutic program for $2.5 million. The program was previously a joint development collaboration between Benitec and Biomics.

• Benitec anticipates completing in vivo preclinical proof of concept studies for age-related macular degeneration (‘AMD’) and oculopharyngeal muscular dystrophy (‘OPMD’) by the end of calendar year 2016

On May 24, 2016 Transgenomic, Inc. (NASDAQ: TBIO) reported financial results for the first quarter ended March 31, 2016, and provided a business update (Press release, Transgenomic, MAY 24, 2016, View Source [SID:1234512757]).

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Business Update

During the first quarter of 2016 Transgenomic (TBIO) continued to work to realign its core activities around the commercialization of ICE COLD-PCR (ICP), an innovative technology that enables the use of DNA liquid biopsies for better, safer and less costly diagnosis and treatment of many diseases. Broader commercialization of ICP is expected to provide a foundation for expansion of the Company’s licensing and partnering strategy in order to maximize the value of this broadly enabling technology.

As part of its strategic focus on broadly commercializing ICP, TBIO is exiting lower growth legacy businesses, a process that is now largely complete. TBIO conducted a strategic review of the Patient Testing Business Unit in the first quarter of 2016 resulting in a decision to suspend testing at its CLIA laboratory in New Haven, Connecticut, and consolidate all remaining CLIA activities in the Company’s laboratory in Omaha, Nebraska. Divestiture options for these legacy Patient Testing assets are being pursued. Suspension of these Patient Testing activities and closure of the New Haven lab have significantly reduced expenses, resulting in savings of over $1 million a month.

The drive to exit legacy businesses has had major effects on the Company’s operations and financial results. The Genetic Assays and Platforms and Patient Testing businesses have been classified as discontinued operations. Information presented for current and prior quarter periods in the financial statements has been modified to reflect them as discontinued operations.

Paul Kinnon, Transgenomic President and Chief Executive Officer, commented, "We are now nearing completion of our plan to exit our low growth, unprofitable legacy businesses, thereby releasing management to focus solely on commercialization and adoption of our ICP technology. As stated in our conference call last month, we are optimistic that the foundation being developed for ICP will begin to bear fruit over the remainder of 2016. The recent release of additional compelling concordance data is an essential element in providing potential partners and customers with the validation they need to demonstrate that ICP can deliver on its promise to enable and simplify the detection of genetic alterations, which is central to implementation of personalized and precision medicine. We believe that the ICP platform has the potential to make TBIO a leader in the rapidly emerging liquid biopsy market, and we have now turned our full attention to further development of our ICE COLD-PCR technology. Having alternatives to tissue-based biopsies is expected to fundamentally change how we diagnose and treat cancer and other disorders. The terrific results from our concordance study have re-affirmed our excitement about the near and long-term potential for this broadly-enabling technology, and we look forward to continued progress in the months ahead."

First Quarter Financial Results from Continuing Operations

Net sales for the first quarter of 2016 were $0.2 million as compared with $0.7 million for the same period in 2015. The $0.5 million decrease reflects phasing of contracts in the first quarter due to client sample availability issues, a situation the Company expects to be transitory.

Gross profit was a negative $0.3 million, compared with gross profit of $0.3 million for the same period in 2015. The negative gross profit in the first quarter of 2016 is due to the lower revenues noted above coupled with a substantial one-time milestone expense associated with product commercialization. A significant portion of Cost of Goods Sold is fixed costs associated with the operation of Transgenomic’s laboratory. The Company anticipates that gross profit percentages will increase as revenues from ICP-based products and services rise.

Operating expenses were $2.0 million during the first quarter of 2016 as compared to $2.3 million in the first quarter of 2015. The $0.3 million decrease in operating expenses was due to lower professional fees in the first quarter of 2016 as compared to the same period in 2015.

The net loss from continuing operations for the first quarter of 2016 was $2.1 million or $0.10 per share, compared with a net loss of $2.3 million or $0.28 per share for the first quarter of 2015. Modified EBITDA, which is a non-GAAP measure that Transgenomic views as an appropriate and sound measure of the Company’s results, was a loss of $2.1 million for the first quarter of 2016, compared to a loss of $1.8 million for the same period in 2015. A reconciliation of Net Loss to Modified EBITDA is presented below.

Cash and cash equivalents were $0.2 million at March 31, 2016, compared with $0.4 million at December 31, 2015. As previously announced, during the first quarter of 2016, the Company completed a financing that raised approximately $2.0 million in net proceeds.

Recent Highlights

Released New Study Showing 100% Concordance between ICE COLD-PCR (ICP) Liquid Biopsies and Conventional Tissue Biopsy Results; ICP Also Identified More Tumor Mutations than Conventional Methods
The concordance study confirmed that ICP-enriched testing identified all mutations detected by standard tissue biopsy PCR. Notably, ICP also identified additional mutations missed by conventional tissue biopsy. The study confirmed that ICP’s ultra-high sensitivity enables use of plasma-based liquid biopsies for cancer mutation detection, replacing costly and invasive tissue biopsies and enabling ongoing patient monitoring.
Launched First Rapid Turnaround Breast Cancer Analysis Panel at 2016 AACR (Free AACR Whitepaper) Annual Meeting
The liquid biopsy test uses Multiplexed ICE COLD-PCR to detect actionable tumor mutations in genes relevant to treatment decisions with high sensitivity. Notably, results are available in 7-10 days, in contrast to turnaround times of up to four weeks for other testing methods.
Announced Data Presentation Confirming Utility of ICP Liquid Biopsy Technology at AACR (Free AACR Whitepaper)
Includes first systematic data confirming concordance of ICP liquid and tissue biopsy results
Launched 1st Commercially Available Assay for Ultra Low Level Detection of EGFR C797S Mutations for Lung Cancer, New MX-ICP Liquid Biopsy Tests for Detection of Colorectal and Melanoma Tumor Mutations, and New MX-ICP Panels for Liquid Biopsy Detection of RAS and PIK3CA Tumor Mutations
Launched multiple important new ICE COLD-PCR liquid biopsy cancer tests.