On May 25, 2016 DelMar Pharmaceuticals, Inc. (OTCQX: DMPID) ("DelMar" and the "Company"), a company focused on developing and commercializing proven cancer therapies in new orphan drug indications, reported the successful completion of an End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) (Press release, DelMar Pharmaceuticals, MAY 25, 2016, View Source [SID:1234512771]). Schedule your 30 min Free 1stOncology Demo! "Our End-of-Phase 2 meeting was an important milestone for the Company as we advance the clinical development of VAL-083," stated Jeffrey Bacha, chairman and CEO of DelMar Pharmaceuticals. "We are pleased with the FDA’s guidance on our Phase 3 trial design in patients with glioblastoma who have failed prior therapy with temozolomide and bevacizumab (Avastin), and we look forward to finalizing the trial design and initiating the study as quickly as possible."
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DelMar’s advanced development program will feature a single randomized Phase 3 study measuring survival outcomes compared to a "physicians’ choice" control, which, if successful, would serve as the basis for a New Drug Application (NDA) submission for VAL-083. The control arm will consist of a limited number of salvage chemotherapies currently utilized in the treatment of Avastin-failed GBM. The final pivotal trial design will be confirmed with the FDA following further discussions with the Company’s clinical advisors.
The FDA confirmed that it will consider the totality of data available, including data obtained from DelMar’s other planned clinical trials in related GBM populations, when assessing the NDA. The FDA also noted that DelMar can rely on prior US National Cancer Institutes (NCI) studies and historical literature to support non-clinical data required for an NDA filing and that DelMar will have the option to file under a 505(b)(2) strategy, which allows a sponsor to rely on already established safety and efficacy data in support an NDA.
"There is a major unmet medical need in refractory glioblastoma," said Mr. Bacha. "The encouraging data from our clinical trials to date, combined with historical data from prior clinical trials from the NCI, suggest that VAL-083 has the potential to improve therapeutic outcomes for GBM patients who currently have no viable treatment options. We are now one step closer to achieving that goal."
About VAL-083
VAL-083 is a "first-in-class," small-molecule chemotherapeutic. In more than 40 Phase I and II clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated clinical activity against a range of cancers including lung, brain, cervical, ovarian tumors and leukemia both as a single-agent and in combination with other treatments. VAL-083 is approved in China for the treatment of chronic myelogenous leukemia (CML) and lung cancer, and has received orphan drug designation in Europe and the U.S. for the treatment of malignant gliomas. DelMar recently announced that the USFDA’s Office of Orphan Products had also granted an orphan designation to VAL-083 for the treatment of medulloblastoma.
DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by the expression of MGMT, a DNA repair enzyme that is implicated in chemotherapy resistance and poor outcomes in GBM patients following standard front-line treatment with Temodar (temozolomide).
DelMar conducted a Phase I/II clinical trial in GBM patients whose tumors have progressed following standard treatment with temozolomide, radiotherapy, bevacizumab and a range of salvage therapies. Patients were enrolled at five clinical centers in the United States: Mayo Clinic (Rochester, MN); UCSF (San Francisco, CA) and three centers associated with the Sarah Cannon Cancer Research Institute (Nashville, TN, Sarasota, FL and Denver, CO) (clinicaltrials.gov identifier: NCT01478178). DelMar announced the completion of enrollment in a Phase II expansion cohort in September, 2015.
About Glioblastoma Multiforme (GBM)
Glioblastoma multiforme (GBM) is the most common and most malignant form of brain cancer. Approximately 15,000 people are diagnosed with GBM each year in the U.S., with similar incidence in Europe. Standard of care is surgery, followed by either radiation therapy, or radiation therapy combined with temozolomide. Approximately 60 percent of GBM patients treated with temozolomide experience tumor progression within one year. More than half of glioblastoma patients will fail the currently approved therapies and face a very poor prognosis.
Alkermes Announces Initiation of Phase 1 Clinical Study of Immuno-Oncology Drug Candidate ALKS 4230
On May 25, 2016 Alkermes plc (NASDAQ: ALKS) reported the initiation of a phase 1 clinical study of the company’s immuno-oncology drug candidate, ALKS 4230 (formerly referred to as RDB 1450), a novel Selective Effector Cell Activator (SECA) protein designed for targeted interleukin-2 (IL-2) receptor activation (Press release, Alkermes, MAY 25, 2016, View Source [SID:1234512770]). Schedule your 30 min Free 1stOncology Demo! The multi-center phase 1 study is designed to evaluate the safety, tolerability and immunological-pharmacodynamic effects of ALKS 4230 in the treatment of patients with solid tumors.
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The phase 1 study will be conducted in two stages: a dose-escalation stage followed by a dose-expansion stage. In the first stage of the study, ALKS 4230 will be administered as an intermittent intravenous infusion with ascending doses in patients with solid tumors who are refractory or intolerant to established therapies. This first stage of the study is designed to determine a maximum tolerated dose, and to identify the optimal dose range of ALKS 4230 based on measures of immunological-pharmacodynamic effects. Following the identification of the optimal dose range of ALKS 4230 in the first stage of the study, the dose-expansion stage of the study will evaluate ALKS 4230 in patients with selected solid tumor types. Initial results from the first stage of the phase 1 study are expected in 2017.
"ALKS 4230 is a unique immuno-oncology candidate that is designed to harness the IL-2 mechanism in a selective way that enhances tumor-killing immune cells, so that a patient’s own immune system can be activated in order to fight cancer more effectively," said Elliot Ehrich, M.D., Chief Medical Officer of Alkermes. "We have designed this initial clinical study of ALKS 4230 to be highly informative and to position us for phase 2 studies, including those that may include ALKS 4230 in combination with other immuno-oncology therapies. We are excited about the start of the clinical program and the potential for ALKS 4230 to make an impact for patients with cancer."
About ALKS 4230 and the SECA Immuno-Oncology Program
ALKS 4230 is a novel selective effector cell activator (SECA) protein designed to preferentially bind and signal through the intermediate affinity interleukin-2 (IL-2) receptor complex, thereby selectively activating and increasing the number of immunostimulatory tumor-killing immune cells while avoiding the expansion of immunosuppressive cells that interfere with anti-tumor response. The selectivity of ALKS 4230 is designed to leverage the proven anti-tumor effects while overcoming limitations of existing IL-2 therapy which activates both immunostimulatory and immunosuppressive receptors.
SECA proteins are engineered using Alkermes’ proprietary PICASSOTM circular permutation technology leveraging human protein biology to achieve their unique mechanism of action.
Transgenomic Reports First Quarter 2016 Financial Results
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.
Interim Report for the Nine Months Ended March 31, 2016
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
Ignyta Announces RXDX-105 Phase 1 Data Presentation at the 2016 ASCO Annual Meeting
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