AVEO Oncology Reports Full Year 2015 Financial Results and Provides Business Update

On March 15, 2016 AVEO Oncology (NASDAQ:AVEO) reported financial results for the full year ended December 31, 2015 and provided a business update (Press release, AVEO, MAR 15, 2016, View Source;p=RssLanding&cat=news&id=2148504 [SID:1234509532]).

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"Over the course of 2015, AVEO has streamlined its organization and taken a fresh strategic direction to create increased shareholder value. In 2016, we are squarely focused on furthering the execution of this strategy," said Michael Bailey, president and chief executive officer. "We remain focused on retaining rights to develop our three oncology-focused clinical programs in North America, while seeing our pipeline advanced by partners in the lab, clinic and through the regulatory process in geographies or disease areas outside of our strategic focus." Mr. Bailey concluded: "We are evaluating all options for funding the clinical and regulatory development of tivozanib in North America, including TIVO-3, the Company’s Phase 3 U.S. pivotal study of tivozanib in the third line treatment of patients with renal cell cancer, and a PD1 combination study. Subject to the outcome of our settlement discussions with the SEC, the Company could be in a position to begin patient enrollment in the TIVO-3 Study in the second quarter of 2016."

Recent Updates

Exclusive Licensing Agreement for Tivozanib in Europe with EUSA Pharma and Submission of a Marketing Authorization Application for Tivozanib in Renal Cell Carcinoma. In February 2016, AVEO and EUSA Pharma announced that EUSA Pharma has submitted a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for tivozanib as a first line treatment for renal cell carcinoma (RCC). In December 2015, AVEO and EUSA Pharma announced an exclusive license agreement in which AVEO granted EUSA Pharma European rights to tivozanib for the treatment of advanced RCC. The agreement also includes a number of additional territories outside North America, including South America and South Africa, and potential additional indications. Under the terms of the agreement, AVEO received an upfront research and development funding payment of $2.5 million, and is eligible for up to $394 million in potential payments and milestones, assuming successful achievement of specified development, regulatory and commercialization objectives, as well as a tiered royalty ranging from a low double-digit up to mid-twenty percent on net sales of tivozanib in the agreement’s territories. A percentage of milestone and royalty payments received by AVEO are due to Kyowa Hakko Kirin as a sublicensing fee.

Exclusive Licensing Agreement for Tivozanib in Russia and other territories with Pharmstandard Group and Acceptance of Registration Dossier for Tivozanib in RCC by the Ministry of Health of the Russian Federation. In February 2016, AVEO announced that a registration dossier seeking to obtain marketing authorization of tivozanib as a first line treatment of advanced RCC has been accepted by the Ministry of Health of the Russian Federation. The dossier was submitted in December 2015 by Pharmstandard Group. In August 2015, AVEO licensed Pharmstandard rights to the development, manufacture and commercialization of tivozanib in the territories of Russia, Ukraine and the Commonwealth of Independent States, for all indications other than non-oncologic diseases or conditions of the eye. AVEO is eligible to receive up to $7.5 million in connection with the first marketing authorization of tivozanib in Russia. AVEO is also eligible to receive a high single-digit royalty on net sales, if any, in the above mentioned territories. A percentage of any milestone and royalty payments received by AVEO are due to Kyowa Hakko Kirin as a sublicensing fee.

Settlement discussions with the Securities and Exchange Commission. AVEO previously announced that the staff (the "SEC Staff") of the Securities and Exchange Commission (the "SEC") and AVEO have entered into discussions for the settlement of potential claims that the SEC may bring against the Company asserting that the Company previously violated federal securities laws by omitting to disclose to investors a recommendation made to the Company by the U.S. Food and Drug Administration in May 2012 that the Company conduct an additional clinical trial with respect to tivozanib. The Company’s settlement discussions with the SEC have continued, and the Company has accrued an estimated settlement liability, for accounting purposes, of $4.0 million in its financial statements as of December 31, 2015. There can be no assurance, however, that a settlement will be achieved with the SEC, or that any settlement we enter into with the SEC will be within the estimated settlement liability accrued.

Receipt of $3.5 Million AV-380 Inventory Reimbursement Payment from Novartis. AVEO previously announced that Novartis exercised its right under its license agreement for AV-380, AVEO’s first-in-class, potent, humanized inhibitory antibody targeting growth differentiation factor 15 (GDF15), to acquire AVEO’s inventory of clinical quality drug substance. This reimbursement payment of approximately $3.5 million was received in the first quarter of 2016. Novartis acquired an exclusive, worldwide license for the development and commercialization of AV-380 and related antibodies in August 2015. Under terms of the agreement, AVEO received an upfront payment of $15 million and the reimbursement payment of approximately $3.5 million, and is eligible to receive clinical, regulatory and sales-based milestone payments totaling $308 million, assuming successful advancement of the product. AVEO will also be eligible to receive tiered royalties on product sales ranging from high single digits to a low double-digit.
Full Year 2015 Financial Highlights

AVEO ended 2015 with $34.1 million in cash and investments.

Total collaboration revenue for 2015 was approximately $19.0 million compared with $18.1 million for 2014. The increase was primarily due to the recognition of $18.5 million of revenue associated with the receipt of a $15.0 million upfront payment for our license of AV-380 to Novartis and Novartis’ subsequent purchase of clinical material for $3.5 million. These amounts were partially offset by a decrease of $3.6 million of revenue from Astellas following the termination of our collaboration agreement in 2014 and a decrease of $14.3 million of revenue recognized from our arrangement with Biogen due to the one-time recognition of previously deferred revenue following an amendment to our agreement in 2014.

Research and development (R&D) expense for 2015 was $12.9 million compared with $38.3 million for 2014. The decrease in R&D expense was primarily due to a reduction in personnel-related, IT, and facilities expenses following AVEO’s January 2015 strategic restructuring as well as a decrease in outsourced services costs primarily related to the completion of the manufacture of AV-380 material in 2014; and a decrease in medical affairs and external clinical trial costs associated with the decreased number of active patients enrolled in our clinical trials.

General and administrative (G&A) expenses for 2015 were $14.2 million compared to $18.6 million for 2014. The decrease is primarily the result of a decrease in personnel-related, facilities, IT, insurance and other infrastructure costs following the Company’s January 2015 strategic restructuring as well as a decrease in external legal costs associated with various ongoing legal matters. These amounts were partially offset by $4.0 million in expense incurred in 2015 related to the accrual of an estimated settlement liability, for accounting purposes, related to the potential SEC claims and an increase in depreciation expense due to the acceleration of depreciation in connection with the termination of our lease agreement of 650 East Kendall Street in September 2014.

Restructuring and lease exit expense for 2015 was $4.4 million compared with $11.7 million for 2014. The expenses incurred during 2015 relate to costs associated with elimination of our research function and the associated reductions in headcount as part of our January 2015 restructuring. The expenses incurred during 2014 relate to costs associated with partially vacating and subsequently terminating the agreement for our leased space at 650 East Kendall Street, which occurred in September 2014.
Net loss for 2015 was $15.0 million, or a loss of $0.27 per basic and diluted share compared with net loss of $52.7 million or a loss of $1.01 per basic and diluted share for 2014.
Updated Financial Guidance

AVEO believe that its cash resources would allow the Company to fund its current operations into the fourth quarter of 2017. This estimate does not include the payment of potential licensing milestones or the uncommitted costs of conducting any contemplated clinical trials, and assumes no milestone payments from AVEO’s partners, no additional funding from new partnership agreements, no equity financings, no debt financings or accelerated repayment thereof and no further sales of equity under the Company’s ATM. This estimate also does not include any amount AVEO may agree to pay in excess of the estimated settlement liability, for accounting purposes, that the Company has established with respect to a potential settlement of claims with the SEC, as described in the Company’s 2015 annual report filed on Form 10-K.

28th Annual ROTH Conference

In lieu of a financial results conference call, AVEO will be presenting a corporate update at the 28th Annual ROTH Conference tomorrow, March 16, 2016, at 9:00 a.m. Pacific Time. A live webcast of the presentation can be accessed by visiting the investors section of the company’s website at www.aveooncology.com. A replay of the webcast will be archived for 30 days following the presentation date.

SRI International Awarded $19.8M to Develop Cancer Preventive Vaccines

On March 15, 2016 SRI International announced that it has been awarded a contract of up to $19.8 million from the US National Cancer Institute (NCI) PREVENT Cancer Preclinical Drug Development Programme to support the development of potential cancer preventive agents or vaccines (Press release, SRI International, MAR 15, 2016, View Source [SID:1234509931]).

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SRI Biosciences, a division of SRI International, will conduct preclinical studies to assess the efficacy of specific compounds or vaccines for preventing invasive-cancer development. Researchers will also identify biomarkers to help quantify the effectiveness of the experimental compounds and vaccines.

"The discovery and development of cancer preventative agents is an area that is underserved, primarily because the length of required clinical trials can be resource-prohibitive for many companies. Our work in biomarker discovery may provide validated surrogate endpoints that can help to shorten clinical trials in cancer prevention," said Lidia Sambucetti, Ph.D., senior director of Cancer Biology, Centre for Discovery Technologies, SRI Biosciences, and principal investigator for the NCI contract. "The PREVENT programme is an opportunity to identify and advance novel strategies for cancer prevention."

SRI Biosciences will conduct detailed preclinical pharmacological studies

Under the three-year contract, SRI Biosciences will conduct detailed preclinical pharmacological studies to determine the efficacy of experimental agents, as well as test biomarkers that may parallel the effectiveness of response to these agents.

Under the PREVENT programme, for efficacy and biomarker testing, two NCI task orders in the area of cancer prevention have already been awarded to SRI Biosciences: The first to develop a mesothelin-based vaccine against ovarian cancer; and another to develop novel models for testing preventative agents against ovarian cancer.

For the ovarian cancer programme, SRI Biosciences optimised a vaccine strategy designed to mount both antibody-based and cellular immunity against mesothelin tumour antigen. SRI researchers are currently testing whether the vaccine can help prevent ovarian cancer. In addition, SRI generated encouraging data supporting the development of a new model that will be used to test experimental drugs for ovarian cancer prevention.

Oncurious NV and BioInvent Partner with US Research Consortium to accelerate Phase I/IIa clinical development of TB-403

On March 15, 2016 Oncurious NV, an emerging oncology company focused on the development of innovative orphan drugs for the treatment of pediatric tumors, reported that it has signed a partnership with the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), a collaboration of 25 US academic medical centers, teaching hospitals and other entities, with the purpose of facilitating and conducting collaborative research activities and investigations of new treatments for neuroblastoma, medulloblastoma and other pediatric cancers (Press release, Oncurious, MAR 15, 2016, View Source [SID:1234509563]). NMTRC is headquartered at Helen DeVos Children’s Hospital in Grand Rapids, MI, USA.

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The partnership is a last step in the preparative work by Oncurious and BioInvent International AB before initiating a Phase I/IIa clinical program with TB-403 for the treatment of medulloblastoma, neuroblastoma and Ewings sarcoma, all rare, life-threatening pediatric cancers.
BioInvent International AB is the clinical development partner for the planned TB-403 clinical trial. Final preparations to enable opening the study for enrollment are ongoing.
TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF). PlGF is expressed in several types of cancer, including medulloblastoma, neuroblastoma and Ewings sarcoma. High expression of the PlGF receptor neuropilin 1 has been shown to correlate with poor overall survival.

A paper in Cell in February 2013 (Cell, 152, 1065-76, 2013), highlighted for the first time that PlGF plays a vital role in the brain and that its expression is required for the growth and spread of medulloblastoma. The paper was based on pre-clinical research conducted by Prof Rakesh Jain from the Massachusetts General Hospital at Harvard (Boston) and the team of Prof Peter Carmeliet from VIB/ KU Leuven.

Patrik De Haes, MD, Executive Chairman of Oncurious nv comments "This agreement with NMTRC will give Oncurious and BioInvent International access to a significant number of centers with considerable expertise in treating children with medulloblastoma. The broad NMTRC clinical network will be a major positive as we set out to develop TB-403 as a novel improved treatment for children with medulloblastoma, neuroblastoma and Ewings sarcoma."

Michael Oredsson, CEO of BioInvent comments: "The collaboration with NMTRC and its investigator network brings a wealth of experience in the paediatric oncology field into this project, and takes us one important step closer to potentially providing a new, targeted treatment option for a group of patients with a tremendous unmet medical need."

FDA Grants Spectrum Pharmaceuticals Approval of EVOMELA™ (melphalan) for Injection

On March 15, 2016 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported that the U.S. Food and Drug Administration (FDA) has granted approval of EVOMELA for use in two indications: 1) use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation (ASCT) in patients with multiple myeloma (MM), and 2) for the palliative treatment of patients with MM for whom oral therapy is not appropriate (Press release, Spectrum Pharmaceuticals, MAR 15, 2016, View Source [SID:1234509559]). This is the first product to be FDA-approved for the high-dose conditioning indication in MM.

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"I am very proud to announce that Spectrum has been able to bring another new cancer drug to the market," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "This represents the commercialization of our sixth Hematology/Oncology product in the U.S. Our EVOMELA formulation does not contain propylene glycol and is reconstituted and admixed with normal saline. This new formulation also uses Captisol technology, which allows the admixture solution to be stable for 4 hours at room temperature in addition to the 1 hour following reconstitution and has been used in several other FDA-approved products. We are very excited about the approval of EVOMELA as this fits seamlessly into our existing commercial infrastructure. In addition to our five other currently marketed products, we believe revenues from EVOMELA will help us expeditiously develop the potential blockbusters that we have in our late-stage pipeline."

"The approval of EVOMELA marks the first new formulation of melphalan approved by the FDA, since its initial approval in 1964," said Dr. Parameswaran Hari, Armand J. Quick/William F. Stapp Professor of Hematology at the Medical College of Wisconsin, Director of the Adult Blood and Marrow Transplant Program at Froedtert Hospital and the Section Head of Hematologic Malignancies and Transplantation, in the Division of Hematology and Oncology in the Department of Medicine. "Melphalan is extensively used in the treatment of multiple myeloma and is the main drug in conditioning therapy pre-transplant. EVOMELA’s new formulation does not contain propylene glycol and is stable for 4 hours at room temperature in addition to the 1 hour following reconstitution."

Spectrum Pharmaceuticals gained global development and commercialization rights to EVOMELA from Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) in March 2013. Spectrum assumed responsibility for completing the pivotal Phase 2 clinical trial, and was responsible for filing the NDA. Under the license agreement, Ligand received a license fee and is eligible to receive milestone payments, as well as royalties following potential commercialization.

About Multiple Myeloma

Multiple Myeloma is a systemic malignancy of plasma cells that accumulate in the bone marrow, usually associated with monoclonal antibody secretion, and results in bone marrow failure and bone destruction. It is the second most common hematologic disease with nearly 30,000 new cases projected in the US in 2016 and over 11,000 deaths annually (American Cancer Society Stats, 2016). The rate of ASCT for patients with MM is growing by approximately 3.3% annually.

Melphalan is the most commonly used IV agent for high-dose conditioning for patients undergoing ASCT for MM. The current IV melphalan market is approximately $100 million annually, with predominant use in ASCT; EVOMELA is the only intravenous melphalan product that is approved for use in the high-dose conditioning indication.

About EVOMELA

EVOMELA was approved by FDA based on its bioequivalence to the standard melphalan formulation (Alkeran) in a Phase 2 clinical study (Aljitawi et al, Bone Marrow Transplant, 2014) via the 505(b)(2) regulatory pathway. EVOMELA has been granted Orphan Drug Designation by the FDA for its use as a high-dose conditioning regimen for patients with MM undergoing ASCT.

EVOMELA’s new melphalan formulation does not contain propylene glycol. The use of the Captisol technology to reformulate also contributes to the 4-hour admixture stability of EVOMELA at room temperature. This is in addition to the 1 hour stability of reconstituted EVOMELA drug product at room temperature and 24 hour stability at refrigerated temperature (5°C).

Please see the Important Safety Information below and the full prescribing information, including BOXED WARNINGS, for EVOMELA at www.evomela.com.

Important Safety Information

WARNING: SEVERE BONE MARROW SUPPRESSION, HYPERSENSITIVITY, and LEUKEMOGENICITY

Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) melphalan to oral melphalan have shown more myelosuppression with the IV formulation. Monitor hematologic laboratory parameters.

Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation of melphalan. Discontinue treatment with EVOMELA for serious hypersensitivity reactions.

Melphalan produces chromosomal aberrations in vitro and in vivo. EVOMELA should be considered potentially leukemogenic in humans.

Contraindications

History of serious allergic reaction to melphalan.

Warnings and Precautions

Nausea, vomiting, diarrhea or oral mucositis may occur. Provide supportive care using antiemetic and antidiarrheal medications as needed.

Hepatic disorders ranging from abnormal liver function tests to clinical manifestations such as hepatitis and jaundice have been reported after treatment with melphalan. Hepatic veno-occlusive disease has also been reported. Monitor liver chemistries.

EVOMELA can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to avoid pregnancy during and after treatment with EVOMELA. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, advise the patient of potential risk to the fetus.

Melphalan-based chemotherapy regimens have been reported to cause suppression of ovarian function in premenopausal women, resulting in persistent amenorrhea in approximately 9% of patients. Reversible or irreversible testicular suppression has also been reported.

Adverse Reactions

The most common adverse reactions observed in at least 50% of patients with multiple myeloma treated with EVOMELA were neutrophil count decreased (100%), white blood cell count decreased (100%), lymphocyte count decreased (98%), platelet count decreased (98%), diarrhea (93%), nausea (90%), fatigue (77%), hypokalemia (74%), anemia (66%), and vomiting (64%).
In a single-arm clinical study, twelve (20%) patients with multiple myeloma who received EVOMELA conditioning for ASCT experienced a treatment emergent serious adverse reaction. The most common serious adverse reactions ( > 1 patient, 1.6%) were pyrexia, hematochezia, febrile neutropenia, and renal failure.

In a randomized clinical trial studying the palliative treatment of patients with multiple myeloma, severe myelotoxicity (WBC ≤1,000 and/or platelets ≤25,000) was more common in the IV melphalan arm (28%) than in the oral melphalan arm (11%).
Drug Interactions

No formal drug interaction studies have been conducted. When nalidixic acid and IV melphalan are given simultaneously, the incidence of severe hemorrhagic necrotic enterocolitis has been reported to increase in pediatric patients.
Use in Specific Populations

It is not known whether melphalan is present in human milk. Because many drugs are excreted in human milk and because of the potential for serious adverse reactions in nursing infants from melphalan, breastfeeding is not recommended during treatment with EVOMELA.

Advise females of reproductive potential to avoid pregnancy, which may include the use of effective contraception methods, during and after treatment with EVOMELA.

For Palliative Treatment, consider dose reduction for patients with renal impairment receiving EVOMELA.
About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled six FDA-approved products, including Onyx Pharmaceuticals’ Kyprolis, Baxter International’s Nexterone and Merck’s NOXAFIL IV. There are also more than 30 Captisol-enabled products currently in clinical development.

FDA Grants Second Orphan Designation For VAL-083

On March 15, 2016 DelMar Pharmaceuticals, Inc. (OTCQX: DMPI) ("DelMar" and the "Company"), reported that the FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation for its lead product candidate, VAL-083, in the treatment of medulloblastoma (Press release, DelMar Pharmaceuticals, MAR 15, 2016, View Source [SID:1234509555]). The investigational drug candidate previously received an orphan designation for glioblastoma in the United States and in Europe.

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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.

"We are pleased to achieve this important regulatory milestone and to continue a collaborative relationship with the FDA and the OOPD as we continue to expand the development of VAL-083," commented Jeffrey Bacha, chairman and CEO of DelMar. "Orphan designation is a major step toward expediting this promising therapy to an additional patient population with few treatment options."

DelMar has been conducting clinical trials with VAL-083 as a potential new treatment for glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer. In September 2015, DelMar announced completion of enrollment in a Phase II clinical trial in refractory GBM. The Company anticipates top-line overall survival data from this trial in the first half of 2016.

Through its research, DelMar has also been exploring the unique cytotoxic mechanism of VAL-083 in order to identify additional indications where VAL-083 may address modern unmet medical needs in the treatment of cancer. In November 2015, DelMar presented new pre-clinical data in a poster entitled, "Dianhydrogalactitol (VAL-083) Offers Potential Therapeutic Alternatives in the Treatment of Pediatric Brain Tumors," at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Advances in Pediatric Research: From Mechanisms and Models to Treatment and Survivorship Conference.

Medulloblastoma is the most common malignant pediatric brain tumor, accounting for 15-30% of all childhood intracranial neoplasms. Although multidisciplinary treatment has improved the 5-year survival rates in children significantly, the prognosis for certain subtypes of medulloblastoma and for recurrent disease remains poor with a median overall survival of less than one (1) year.

In historical NCI-sponsored clinical studies, VAL-083 demonstrated clinical activity against medulloblastoma. In these studies VAL-083 was investigated both as a stand-alone therapy and in combination with other chemotherapeutic regimens. DelMar’s recent pre-clinical research demonstrates that VAL-083 is active against medulloblastoma cells with difficult to treat sonic hedgehog (SHH) characteristics and p53 mutations; and VAL-083 in combination with temozolomide completely inhibits self-renewal of pediatric brain cancer stem cells (CSCs).

"Taken together, we believe these data will serve as a basis for our clinical development strategy with VAL-083 in pediatric brain tumors," continued Mr. Bacha. "We plan to continue our discussions with leading clinical investigators in order to undertake the necessary steps to advance VAL-083 into clinical studies as a potential treatment for children suffering from recurrent and difficult-to-treat medulloblastoma subtypes."