On March 15, 2016 Diffusion Pharmaceuticals Inc. (OTCQX: DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer, reported the online publication of a reviewed article on RES-529 ahead of print in Anti-Cancer Drugs (Press release, Diffusion Pharmaceuticals, MAR 15, 2016, View Source [SID:1234509556]).

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Diffusion Pharmaceuticals recently acquired RES-529, a novel PI3K/AKT/mTOR pathway inhibitor that targets both mTORC1 and mTORC2 through mTOR complex dissociation, from RestorGenex Corporation at the closing of its reverse merger on January 8, 2016. RES-529 is being developed for oncology and ophthalmology with a primary focus in the treatment of glioblastoma multiforme (GBM). Orphan drug designation has been granted by the FDA for this indication.

The article entitled "RES-529: a PI3K/AKT/mTOR pathway inhibitor that dissociates the mTORC1 and mTORC2 complexes," reviews the potential of RES-529 to inhibit tumorigenesis in glioblastoma, prostate cancer, and breast cancer. RES-529 treatment has been shown to be effective in preclinical models of glioblastoma as well as synergistic with radiation therapy in these models. In preclinical models of prostate cancer, RES-529 has been shown to be synergistic with all three common modalities of treatment: radiation therapy, chemotherapy, and hormonal therapy. The article was authored by Mark Weinberg, MD, MBA, who was previously Senior VP of Clinical Development at RestorGenex.

David Kalergis, Chairman and Chief Executive Officer of Diffusion Pharmaceuticals, said, "The acquisition of RES-529 has strengthened our oncology pipeline. Concurrent with our primary focus of advancing our lead candidate, trans sodium crocetinate (TSC), into a Phase III clinical program in GBM and a Phase II/III trial in pancreatic cancer, we are also assessing the future opportunities and plans for RES-529."

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.

On March 15, 2016 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported fourth quarter and year-end 2015 financial results and provided an update on its drug development programs (Press release, Loxo Oncology, MAR 15, 2016, View Source [SID:1234509558]). Loxo Oncology will not be conducting a conference call in conjunction with this earnings release.

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"We ended 2015 with great momentum behind LOXO-101 and our pipeline programs," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "We are excited to announce that updated LOXO-101 adult Phase 1 data have been selected for an oral plenary session at AACR (Free AACR Whitepaper) in April. This presentation will provide further follow-up on the first three enrolled patients with TRK fusions that responded to LOXO-101, and will provide initial response assessment and follow-up on the next three enrolled patients with TRK fusions. We are also pleased to announce that we have developed a next-generation selective TRK inhibitor, LOXO-195, in collaboration with our partner Array BioPharma. Last year, we supported academic research to anticipate potential mechanisms of acquired resistance to TRK inhibition and we have continued to follow published literature reports that corroborate and add to those results. Should a patient progress on LOXO-101 or another compound, we hope LOXO-195 will be able to induce a new response in the patient and functionally extend time on therapy."

Dr. Bilenker continued, "We are fortunate that our financing in November allows us to fund operations well into 2018, which we believe will provide ample runway for potentially meaningful clinical data events for LOXO-101, LOXO-195, our RET program and other possible Array collaboration projects."

New Announcements

Updated LOXO-101 Phase 1 Data at AACR (Free AACR Whitepaper): Loxo Oncology announced today that updated clinical data from the ongoing LOXO-101 Phase 1 trial were accepted for oral plenary session presentation at the 2016 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in New Orleans, taking place April 16-20, 2016. This presentation will be an update to data presented at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting in November 2015, with a focus on enrolled patients with TRK fusions. The abstract title will be posted online at 4:30 p.m. ET on Wednesday, March 16.
Next-Generation Selective TRK Inhibitor: Acquired resistance to targeted therapies has proven to be an important component of long-term cancer care and targeted therapy drug development. In anticipation of potential resistance to LOXO-101, and in light of recent published literature regarding emerging mechanisms of resistance to TRK inhibition, Loxo Oncology today unveiled its next-generation selective TRK inhibitor, LOXO-195, which is capable of addressing potential mechanisms of acquired resistance that may emerge in patients receiving LOXO-101 or multikinase inhibitors with anti-TRK activity. With the LOXO-195 program, Loxo Oncology has an opportunity to strengthen its leadership position in the field of TRK inhibition and clinically extend the duration of disease control for patients with TRK-driven cancers. Loxo Oncology will begin IND-enabling activities in 2016 that should enable initiation of a LOXO-195 Phase 1 study in 2017.
2015 Financial Results and 2016 Cash Guidance. As of December 31, 2015, Loxo Oncology had cash, cash equivalents and investments of $153.9 million. The company expects a cash burn of $48 to $52 million in 2016. The company believes that based upon its current operating plan, its existing capital resources will be sufficient to fund its anticipated operations well into 2018.

Recent Highlights

LOXO-101 Program

LOXO-101 Phase 1 Pediatric Trial Initiated: In December, Loxo Oncology enrolled the first patient in the LOXO-101 pediatric Phase 1 trial, a multicenter, open-label trial in pediatric patients with advanced solid or primary CNS tumors.
LOXO-101 Phase 1 Data Presented at AACR (Free AACR Whitepaper)-NCI-EORTC: Results from the Phase 1 study of LOXO-101 were reported in a late-breaking oral presentation at the 27th AACR (Free AACR Whitepaper)-NCI-EORTC Symposium on Molecular Targets and Cancer Therapeutics in November. As of the October 20, 2015 cutoff date, six patients with cancers harboring TRK fusions had been enrolled and treated. Three of the six patients with TRK fusion cancers had been on study long enough for their first efficacy assessment, and all three had achieved an objective response at the first response assessment, as defined by standard RECIST criteria. See the full data here.
LOXO-101 Phase 2 Basket Trial Initiated: In October, Loxo Oncology enrolled the first patient in the LOXO-101 Phase 2 trial, a global, multi-center, single-arm, open-label basket trial in adult patients with solid tumors that harbor a TRK fusion.
LOXO-101 Selected for NCI-MATCH Trial: In October, the independent committee of the National Cancer Institute-Molecular Analysis for Therapy Choice (NCI-MATCH) clinical trial chose LOXO-101 as the sole, dedicated treatment arm for patients with TRK gene fusions. Loxo Oncology anticipates that the NCI will activate the TRK fusion arm of this study later in 2016.

Pipeline: RET and FGFR Pre-clinical Programs

Data on RET and FGFR Programs Presented at AACR (Free AACR Whitepaper)-NCI-EORTC: Loxo Oncology presented two preclinical posters at AACR (Free AACR Whitepaper)-NCI-EORTC containing the first publicly disclosed data for its Rearranged during Transfection (RET) and Fibroblast Growth Factor Receptor (FGFR) programs showing potential best-in-class selectivity and target coverage.
RET Clinical Trial Initiation: Consistent with prior guidance, the company plans to initiate a Phase 1 study of its RET inhibitor in late 2016 or early 2017.

Corporate Activities

Strengthened Balance Sheet: Loxo Oncology raised $76.2 million in gross proceeds in an equity financing in November 2015. Loxo Oncology’s cash, cash equivalents and marketable securities are expected to be sufficient to fund operations well into 2018.

Fourth Quarter and Year-End 2015 Financial Results

Cash, cash equivalents and investments totaled $153.9 million as of December 31, 2015, compared to $112.9 million as of December 31, 2014. The increase was attributable to $71.3 million in net proceeds from our November 2015 equity financing offset by $30.3 million in cash burn.

Research and development expenses were $9.8 million for the fourth quarter of 2015 compared to $4.6 million in the fourth quarter of 2014. The increase was primarily attributable to expanded Phase 1 and Phase 2 clinical development activities for LOXO-101 and additional full-time equivalents and other support dedicated to discovery, preclinical, and manufacturing activities at Array BioPharma or third-party vendors.

Research and development expenses were $25.6 million for the year ended December 31, 2015, compared to $14.5 million for the year ended December 31, 2014. The increase was primarily due to expanded Phase 1 and Phase 2 clinical development activities for LOXO-101 and additional full-time equivalents and other support dedicated to discovery, preclinical, and manufacturing activities at Array BioPharma or third-party vendors. The company also recognized R&D-related stock-based compensation expense of $3.3 million during the year ended December 31, 2015 compared to $2.0 million for the year ended December 31, 2014.

General and administrative expenses were $3.2 million for the fourth quarter of 2015 compared to $2.6 million in the fourth quarter of 2014. The increase was primarily due to additional full-time equivalents, increased compensation costs and increased costs associated with operating as a public company.

General and administrative expenses were $10.5 million for the year ended December 31, 2015, compared to $6.2 million for the year ended December 31, 2014. The increase was primarily due to additional full-time equivalents, increased compensation costs and increased costs associated with operating as a public company. The company also recognized G&A-related stock-based compensation expense of $2.8 million during the year ended December 31, 2015 compared to $1.0 million for the year ended December 31, 2014.

Net loss attributable to common stockholders was $12.9 million for the fourth quarter of 2015, compared to $7.1 million for the fourth quarter of 2014. Net loss attributable to common stockholders was $35.9 million for the year ended December 31, 2015, compared to $20.7 million for the year ended December 31, 2014.

About LOXO-101

LOXO-101 is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In an ongoing Phase 1 clinical trial, LOXO-101 has demonstrated encouraging preliminary efficacy. LOXO-101 is also being evaluated in a global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions, and a Phase 1 dose escalation trial in pediatric cancer patients. For additional information about both the LOXO-101 clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123.

On March 15, 2016 BIND Therapeutics, Inc. (NASDAQ: BIND), a biotechnology company developing targeted and programmable therapeutics called ACCURINS, reported financial results for the fourth quarter and full year 2015 (Press release, BIND Therapeutics, MAR 15, 2016, View Source [SID:1234509533]). Additionally, the Company announced a shift in its research and discovery strategy to focus on the development of innovative medicines, primarily in cancer. Moving forward, BIND plans to focus on the development of therapeutics that leverage the ability of ACCURINS to incorporate novel combinations of targeting ligands and unique payloads including oligonucleotides and potent kinase inhibitors, creating synergistic properties in a single particle.

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"Our new strategy is to build a pipeline of innovative medicines that address challenges that small molecule chemistry or antibody engineering have not been able to overcome," said Andrew Hirsch, president and chief executive officer, BIND Therapeutics. "Historically, nanoparticle-based therapies have used previously approved medicines as therapeutic payloads to provide equivalent efficacy with improved safety. Our new strategy leverages the unique attributes of ACCURINS, specifically their ability to precisely target cells and tissues with ligand-mediated binding while containing high concentrations of novel therapeutic payloads. We believe this approach will lead to innovative medicines with the potential to achieve therapeutic outcomes unachievable through existing therapeutic modalities."

ACCURINS possess several advantageous properties. The surface of ACCURINS can be functionalized with ligands that can achieve tissue localization, cellular internalization or biological activity, thereby increasing the number of therapeutic strategies available. In addition, the modular nature of ACCURINS enables the functionalization of more than one type of ligand on the surface. ACCURINS are also able to incorporate novel therapeutic payloads with diverse physical and chemical properties, including oligonucleotides and potent kinase inhibitors. The release rate of Accurin payloads can also be controlled to optimize the amount of drug getting to the target tissue over the optimal amount of time.

"Our internal discovery efforts leverage the modular nature of ACCURINS, allowing us to efficiently engineer ideal combinations of tumor-directed targeting ligands and new classes of therapeutic payloads," said Jonathan Yingling, Ph.D., chief scientific officer of BIND. "When encapsulating oligonucleotide-based payloads, ACCURINS have the potential to protect against enzymatic degradation and clearance in the plasma, target tissues beyond the liver, and concentrate them in target cells through ligand-mediated binding that results in tumor cell death. By incorporating small molecule kinase inhibitors, we believe ACCURINS can control the biological activity of tumor-protective immune cells and reverse the immunosuppressed tumor microenvironment. Following optimization of lead product candidates and completion of preclinical studies, we anticipate initiation of clinical testing for one or more of our proprietary innovative product candidates as early as 2018."

BIND remains committed to its ongoing collaborations and clinical-stage programs. The Company’s collaborations with AstraZeneca and Pfizer are intended to enable greater inhibition of important cellular pathways with our collaborators’ proprietary kinase inhibitors. Additionally, the Company expects to communicate topline data from the ongoing iNSITE 1 and 2 trials with BIND-014 in squamous cell non-small cell lung cancer (NSCLC), advanced cervical cancer and head and neck cancer in April 2016. As previously announced, further development of BIND-014 is contingent upon data from these trials.

Anticipated 2016 Milestones:

Report topline data on the full 40 patients in iNSITE 1 squamous NSCLC trial with BIND-014

Report Stage 1 data from iNSITE 2 trial with BIND-014 in advanced cervical and head and neck cancers

Report in vivo proof-of-concept (POC) data for targeting tumor associated macrophages

Identify first ACCURINS-based immuno-oncology product concept

Report initial in vivo POC data for discovery programs, with preclinical pharmacokinetic and efficacy data expected in second half of 2017

In vivo POC data for targeting guanylate cyclase-C (GC-C) receptors expressed on tumors, specifically GI malignancies

Further in vivo POC data in delivering single and double stranded RNA fragments to target cells and achieving target knock-down

Demonstrate in vitro and in vivo POC for achieving endosomal escape with double stranded RNA

2015 Business and Pipeline Highlights:

Continued preclinical work, and generated promising initial data, on Accurin versions of anti-infective and oligonucleotide-based therapies

Continued preclinical work related to targeting of tumor-associated macrophages

Strengthened scientific leadership and corporate governance with appointment of Jonathan Yingling, Ph.D., as chief scientific officer for BIND, and appointment of Arthur Tzianabos, Ph.D., to Board of Directors

Completed preclinical development for AZD2811, an Accurin containing AstraZeneca’s Aurora B Kinase inhibitor, for which AstraZeneca initiated a phase 1 clinical study in the fourth quarter of 2015

Advanced collaboration with Pfizer following its exercise of its option to obtain an exclusive license to develop and commercialize an Accurin drug candidate

Achieved $4.0 million milestone with AstraZeneca for dosing first patient in Accurin AZD2811 phase 1 clinical trial

Received $2.5 million option exercise fee from Pfizer to acquire an exclusive license for the development and commercialization of first compound covered by the agreement

Recognized $14.3 million in 2015 in revenue from milestones, option exercises and reimbursable expenses related to our collaborations with AstraZeneca, Pfizer and Roche

Enrolled patients in phase 2 iNSITE 1 and iNSITE 2 trials allowing the Company to report clinical data in April 2016

Completed build out of dedicated manufacturing space, through an arrangement with a large contract manufacturing organization, that is capable of producing ACCURINS at the double-digit kilogram scale

Fourth Quarter and Full Year 2015 Financial Results

Fourth quarter and full year 2015 revenue was $6.4 million and $15.4 million, compared to $3.0 million and $10.4 million for the same periods in 2014, respectively. The increase in revenue for the fourth quarter and full year 2015 was primarily due to the achievement of the $4 million milestone upon dosing of the first patient in Accurin AZD2811 phase 1 clinical trial.

Fourth quarter and full year 2015 research and development (R&D) expenses were $11.1 million and $37.3 million compared to $8.0 million and $28.9 million for the same periods in 2014, respectively. The increase in R&D expenses for the fourth quarter of 2015 compared to the fourth quarter of 2014 was primarily due to higher clinical development expenses of BIND-014 and reimbursable manufacturing expenses related to Accurin AZD2811. The increase in R&D expenses for the full year 2015 compared to the full year 2014 was primarily due to headcount growth to support the development of BIND’s internal programs and our collaborations, which led to an increase in salaries and benefits, higher reimbursable manufacturing expenses related to Accurin AZD2811 and increased clinical development expenses of BIND-014.

Fourth quarter and full year 2015 general and administrative expenses were $4.6 million and $17.6 million, respectively, compared to $4.2 million and $15.1 million for the fourth quarter and full year 2014, respectively.

Fourth quarter net loss was $7.6 million, or $0.37 per basic and diluted share, compared to a net loss of $8.5 million, or $0.51 per basic and diluted share for the fourth quarter of 2014. 2015 net loss was $36.6 million, or $1.81 per basic and diluted share, compared to a net loss of $32.5 million, or $1.97 per basic and diluted share for 2014.

Cash, cash equivalents and short-term investments were $36.9 million as of December 31, 2015. The Company continues to expect its cash, cash equivalents and short-term investments will fund anticipated operations into the fourth quarter of 2016.

On March 14, 2016 Celator Pharmaceuticals, Inc. (Nasdaq: CPXX) reported positive results from the Phase 3 trial of VYXEOS (cytarabine: daunorubicin) Liposome for Injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukemia (AML) compared to the standard of care regimen of cytarabine and daunorubicin known as 7+3 (Press release, Celator Pharmaceuticals, MAR 14, 2016, View Source [SID:1234509534]). The trial met its primary endpoint demonstrating a statistically significant improvement in overall survival. Data will be submitted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting.

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The median overall survival for patients treated with VYXEOS in the study was 9.56 months compared to 5.95 months for patients receiving 7+3, representing a 3.61 month improvement in favor of VYXEOS. The hazard ratio (HR) was 0.69 (p=0.005) which represents a 31 percent reduction in the risk of death versus 7+3. The percentage of patients alive 12 months after randomization was 41.5% on the VYXEOS arm compared to 27.6% on the 7+3 arm. The percentage of patients alive 24 months after randomization was 31.1% on the VYXEOS arm compared to 12.3% on the 7+3 arm.

"The overall survival advantage seen with CPX-351 compared to 7+3, along with a superior response rate and no increase in serious toxicity indicates that we’ll likely have a new standard of care for treating older patients with secondary AML," said Jeffrey E. Lancet, M.D., senior member and chief of the Leukemia/Myelodysplasia Program at Moffitt Cancer Center and the principal investigator for the study. "This represents a major step forward for a very difficult-to-treat patient population."

VYXEOS also demonstrated a statistically significant improvement in induction response rate (CR+CRi of 47.7% versus 33.3%; p=0.016) and this significance was maintained for the analysis of CR alone (CR of 37.3% versus 25.6%, p=0.040).

Sixty-day all-cause mortality was 13.7% versus 21.2%, in favor of patients treated with VYXEOS.

No substantial difference in Grade 3 or higher adverse events was observed between VYXEOS and 7+3. In the intent-to-treat population, Grade 3 or higher, hematologic adverse events were similar for overall infections, febrile neutropenia, and bleeding events. In the intent-to-treat population, Grade 3 or higher, non-hematologic adverse events were similar across all organ systems, including cardiac, gastrointestinal, general systems, metabolic disorders, musculoskeletal, nervous system, respiratory, skin and renal.

"These findings confirm that VYXEOS provides the first opportunity we’ve had in decades to extend survival for patients with high-risk AML," added Gail Roboz, M.D., Professor of Medicine and Director of the Leukemia Program at the Weill Medical College of Cornell University and the New York-Presbyterian Hospital in New York. "Also, more patients in remission means more who are eligible for potentially curative therapy."

Based on these results the company expects to submit a New Drug Application (NDA) for VYXEOS with the U.S. Food and Drug Administration (FDA) later this year and submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the first quarter of 2017.

"The successful outcome of this Phase 3 trial represents an important advance for AML patients, their families and clinicians," said Scott Jackson, Chief Executive Officer of Celator Pharmaceuticals. "It also marks a major milestone for Celator, for VYXEOS, and for our CombiPlex platform. We offer our sincere thanks to the patients and investigators who participated in this study and we will work closely with regulatory authorities to make this new treatment available to the AML community as soon as possible."

The clinical trial was conducted in partnership with The Leukemia & Lymphoma Society (LLS) through its Therapy Acceleration Program (TAP), which has supported the clinical development of VYXEOS beginning in Phase 2.

About VYXEOS

VYXEOS (cytarabine:daunorubicin) Liposome for Injection, also known as CPX-351, is a nano-scale co-formulation of cytarabine and daunorubicin at a synergistic 5:1 molar ration. VYXEOS represents a novel approach to developing combinations of drugs in which molar ratios of two drugs with synergistic anti-tumor activity are encapsulated in a nano-scale liposome in order to maintain the desired ratio following administration. VYXEOS was granted orphan drug status by the FDA and the European Commission for the treatment of acute myeloid leukemia (AML). VYXEOS was also granted Fast Track designation for the treatment of elderly patients with secondary AML. In addition to the Phase 3 trial, Celator published results from two randomized, controlled, Phase 2 trials with VYXEOS. The first trial was conducted in newly diagnosed elderly AML patients and the second trial was conducted in patients with AML in first relapse.

Phase 3 Trial Design

The randomized, controlled, Phase 3 trial (Protocol NCT01696084), enrolled 309 patients at 39 sites in the United States and Canada, and compared VYXEOS to the conventional cytarabine and daunorubicin treatment regimen (commonly referred to as 7+3) as first-line therapy in older (60-75 years of age) patients with high-risk (secondary) AML. Patients were stratified for age (60 to 69 and 70 to 75 years of age) and AML type; treatment-related AML, AML with documented history of MDS with prior treatment with hypomethylating agent therapy, AML with documented history of MDS without prior hypomethlyating agent therapy, AML with a documented history of chronic myelomonocytic leukemia (CMMoL), and de novo AML with a karyotype characteristic of myelodysplastic syndrome (MDS).

Patients were randomized 1:1 to receive either VYXEOS or 7+3. Patients could receive one or two inductions, and responding patients could receive one or two consolidations. First induction for VYXEOS was 100u/m2; days 1, 3, and 5 by 90-minute infusion and for the control arm was cytarabine 100mg/m2/day by continuous infusion for 7 days and daunorubicin 60mg/m2 on days 1, 2, and 3 (7+3). Second induction for VYXEOS-treated patients was 100u/m2 on days 1 and 3 and the control arm was cytarabine 100mg/m2/day by continuous infusion for 5 days and daunorubicin 60mg/m2 on days 1 and 2 (5+2).

Only patients with documented CR or CRi were eligible to receive chemotherapy consolidation. Consolidation for VYXEOS-treated patients was 65u/m2 on days 1 and 3 and the control arm was cytarabine 100mg/m2/day by continuous infusion for 5 days and daunorubicin 60mg/m2 on days 1 and 2 (5+2).

About AML

Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood characterized by the uncontrolled proliferation of immature blast cells in the bone marrow. AML is generally a disease of older adults, and the median age of a patient diagnosed with AML is about 67 years. The American Cancer Society estimates that there will be 19,950 new cases of AML and 10,430 deaths from AML in the U.S. in 2016. In Europe the number of new cases is estimated to be 18,000 and in Japan the number is 5,500. The Company estimates that nearly 70 percent of AML patients are over the age of 60, and approximately 75 percent are intermediate or high risk. Furthermore, approximately half of those patients are considered suitable for intensive treatment.

Even with current treatment, overall survival for AML is poor. In patients over 60 years of age, the 5 year survival rate is less than 10%. In high-risk (secondary) AML, overall survival is lower, resulting in an acute need for new treatment options for these patients.