On August 5, 2015 Fate Therapeutics, Inc. (NASDAQ: FATE), a biopharmaceutical company engaged in the development of programmed cellular immunotherapeutics for the treatment of severe, life-threatening diseases, reported its financial results for the second quarter ended June 30, 2015, and recent corporate and clinical highlights (Filing, 8-K, Fate Therapeutics, AUG 5, 2015, View Source [SID:1234507034]).

"The interim data from our Phase 2 PUMA study indicate that the therapeutic value proposition of PROHEMA may include the prevention of severe life-threatening infections across a broad spectrum of bacterial, fungal and viral pathogens through a one-time administration at the time of hematopoietic cell transplantation (HCT). This would alleviate a significant cause of morbidity and mortality in patients undergoing HCT and also reduce the need for costly anti-infective treatment regimens following HCT," said Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. "During the second half of 2015, we look forward to sharing additional clinical data from our adult and pediatric clinical development initiatives for PROHEMA. Additionally, we are aggressively moving to extend the promising observations from our PROHEMA clinical experience to other cell sources used in HCT including mobilized peripheral blood, where severe infections and graft-versus-host disease represent significant unmet medical needs. We remain on track to file an IND by the end of 2015 for PROTMUNE, a programmed mobilized peripheral blood immunotherapeutic designed to enhance the therapeutic properties of donor T cells."

Recent Corporate & Clinical Highlights

· Encouraging Immunoprotection Data from Ongoing PUMA Study Observed. On May 6, 2015, the Company reported interim data from an initial 30 subjects in its ongoing Phase 2 PUMA study of PROHEMA, a programmed hematopoietic cell-based immunotherapeutic derived from umbilical cord blood. Immunocompromised subjects administered PROHEMA had a 54% reduction in the rate of infection-related adverse events, including severe bacterial, fungal and viral infections following HCT. Specifically, subjects administered PROHEMA experienced 0.7 total events per subject (13 total events in 18 subjects) as compared to 1.6 total events per subject in the control cohort (19 total events in 12 subjects). Importantly, the event rate for cytomegalovirus (CMV) infection was 0.0 in subjects administered PROHEMA (0 total events in 18 subjects) as compared to 0.3 in the control cohort (4 total events in 12 subjects); and the event rate for bacterial infections was 0.3 in subjects administered PROHEMA (6 total events in 18 subjects) as compared to 0.6 in the control cohort (7 total events in 12 subjects). The ongoing PUMA study, which is designed to investigate several key measures of hematopoietic reconstitution and immunoprotection in adult subjects undergoing double umbilical cord blood transplantation, is expected to enroll approximately 60 total subjects, randomized at a ratio of 2:1.

· First Subject Treated in Pediatric PROVIDE Study Assessing CNS Cellular Replacement Potential of PROHEMA. The first subject in the Company’s Phase 1b PROVIDE study, an open-label clinical study of PROHEMA designed to enroll 12 pediatric subjects undergoing single umbilical cord blood transplantation for the treatment of inherited metabolic disorders, has been treated. The study’s design includes serial neuro-imaging and neuro-cognitive assessments to explore the potential of programmed hematopoietic cells to durably reconstitute in the brain and deliver enzymes which are otherwise missing to the central nervous system. Multiple inherited metabolic disorders, including lysosomal and peroxisomal storage diseases such as Hurler and Hunter syndromes, Krabbe disease and other leukodystrophies, qualify for treatment under the PROVIDE study.

· IND for Programmed Mobilized Peripheral Blood Immunotherapeutic to be Filed in 2H15. The Company expects to file an Investigational New Drug application in the second half of 2015 to conduct a first-in-human clinical assessment of PROTMUNE, a programmed hematopoietic cell-based immunotherapeutic derived from mobilized peripheral blood. In preclinical models of acute graft-versus-host disease (GvHD), host mice that received programmed mobilized peripheral blood cells from a mismatched donor showed a statistically-significant (p<0.0001) reduction in GvHD score and a statistically-significant (p=0.0005) improvement in survival, as compared to mice that received vehicle treated cells. The Company plans to evaluate the potential of PROTMUNE to reduce life-threatening complications of allogeneic HCT, such as acute GvHD, in adult subjects undergoing mobilized peripheral blood HCT.

· Strategic Research Collaboration Formed with Juno Therapeutics to Program CAR T and TCR Immunotherapeutics. On May 4, 2015, the Company entered into a research collaboration and license agreement with Juno to identify and apply small molecule modulators to program genetically-engineered chimeric antigen receptor (CAR) T-cell and T-cell receptor (TCR) immunotherapeutics. Under the collaboration, Juno paid the Company an upfront fee of $5.0 million, purchased one million shares of the Company’s common stock at $8.00 per share, and agreed to fund all of the Company’s collaboration activities during the four-year research term. For each product developed by Juno that incorporates modulators identified through the collaboration, the Company is eligible to receive clinical, regulatory and commercial milestones, plus royalties on net sales.

· Natural Killer Cell-based "Off-the-Shelf" Cancer Immunotherapeutic Programs Launched in Collaboration with University of Minnesota. In July 2015, the Company entered into a collaboration with the University of Minnesota to clinically translate two distinct NK cell-based cancer immunotherapeutic programs, both of which seek to overcome key limitations of adoptive T-cell immunotherapy including the requirement to isolate and engineer cells for each individual patient. In the first program the Company intends to accelerate the development of an "adaptive" NK cell phenotype, which has been shown to have an epigenetic profile similar to that of cytotoxic T lymphocytes and to exhibit long-term persistence in vivo. The second program leverages the Company’s proprietary induced pluripotent stem cell (iPSC) technology, which has the potential to enable the efficient and precise engineering of single pluripotent cells and the large-scale clonal expansion of such cells, in the development of iPSC-derived NK cell-based targeted cancer immunotherapeutics.

Financial Results

· Cash Position: Cash and cash equivalents as of June 30, 2015 were $81.2 million, compared to $49.1 million as of December 31, 2014. The increase is primarily driven by net proceeds from the Company’s public offering of common stock in May 2015 and cash generated from entering into the strategic research collaboration with Juno, offset by cash used to fund operating activities.

· Total Revenue: Revenue was $0.3 million for the second quarter of 2015, which was derived from the Company’s strategic research collaboration with Juno.

· Total Operating Expenses: Total operating expenses were $7.5 million for the second quarter of 2015, compared to $6.0 million for the second quarter of 2014. Operating expenses for the second quarter of 2015 includes $0.7 million of stock compensation expense, compared to $0.4 million for the second quarter of 2014.

· R&D Expenses: Research and development expenses were $4.9 million for the second quarter of 2015, compared to $4.0 million for the second quarter of 2014. The increase in R&D expenses is primarily related to an increase in personnel expense, including stock-based compensation expense, resulting from additional headcount to support the clinical development of PROHEMA and the preclinical evaluation of the Company’s other product candidates.

· G&A Expenses: General and administrative expenses were $2.7 million for the second quarter of 2015, compared to $2.1 million during the second quarter of 2014. The increase in G&A expenses is primarily related to an increase in personnel expense, including stock-based compensation expense, and an increase in intellectual property expenses.

· Common Shares Outstanding: Common shares outstanding as of June 30, 2015 were 28.6 million compared to 20.6 million in December 31, 2014. Common shares outstanding increased primarily as a result of the 6.9 million shares of the Company’s common stock issued pursuant to the May 2015 financing, and the 1.0 million shares of the Company’s common stock issued and sold to Juno pursuant to the strategic research collaboration.

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On August 5, 2015 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company focused on the discovery, development, and commercialization of targeted cancer therapies, reported financial results for the second quarter 2015 ended June 30, 2015 and provided an update on its pipeline (Press release, Loxo Oncology, AUG 5, 2015, View Source [SID:1234507028]).

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"Over the last several months, Loxo has delivered important advances against our pipeline priorities," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "Last week, we achieved an important milestone with the peer-reviewed publication of the first clinical response to LOXO-101, our tropomyosin receptor kinase (TRK) inhibitor. This important early result validates the potential of Loxo’s drug development approach – that selective, purpose-built medicines in genetically well-defined populations can show dramatic and early evidence that justifies continued and rapid drug development. Today we are excited to announce plans for a Phase 2 trial for this program, which accelerate development timelines ahead of previous plans. In addition, we have made significant progress with our preclinical pipeline, allowing us to prioritize our Rearranged during Transfection (RET) and our isoform-sparing Fibroblast Growth Factor Receptor (FGFR) programs, positioning them as our likely next IND filings."

Program Updates

Loxo provided the following updates on its development programs:

LOXO-101: the only potent, oral, selective inhibitor of the TRK family of proteins

Clinical Validation for LOXO-101 Published in Cancer Discovery

The research brief published on July 27, 2015 in Cancer Discovery provides early clinical validation of LOXO-101 against TRK fusion cancer.

The publication, authored in collaboration with Foundation Medicine, Inc. and the Doebele Research Lab at the University of Colorado, includes the case study of the first TRK fusion patient treated in the Phase 1 trial of LOXO-101. A woman with advanced soft tissue sarcoma widely metastatic to the lungs enrolled on study and experienced substantial tumor regression, with imaging studies after one month demonstrating a partial response (PR) as defined by standard RECIST 1.1 criteria. The patient’s shortness of breath rapidly resolved and she was able to discontinue her supplemental oxygen and resume activities of daily living. As discussed in the publication, with four months of treatment, additional CT scans demonstrated almost complete tumor disappearance of the largest tumors. After four months of dosing, the patient did not have any adverse events that were attributed to LOXO-101.

The publication also describes novel assays for assessing LOXO-101’s impact on TRK signaling, patient-derived TRK fusion models in vitro and in vivo which illustrate LOXO-101’s TRK inhibition, and the first-ever description of the molecular epidemiology of TRK fusions in soft tissue sarcoma.

LOXO-101 Phase 2 Trial to Open in the Second Half of 2015

Based on evidence from the Phase 1 trial, including drug exposures that exceeded expectations, Loxo plans to initiate a Phase 2 trial in the second half of this year, earlier than previously anticipated. The trial will be a multicenter, international Phase 2 open label study in adult cancer patients whose tumors harbor TRK fusions. Loxo will provide additional details on the timing, size, and design of the Phase 2 trial after the first clinical site is open.
Loxo believes that a wide variety of tumor types harbor TRK fusions, and plans to work creatively with clinical partners to identify patients with these genetic alterations. Loxo will provide additional details on these plans after the first clinical site of the Phase 2 trial is open.

LOXO-101 Phase 1 Update

In the ongoing Phase 1 study, LOXO-101 continues to consistently achieve systemic drug exposures anticipated to inhibit TRK signaling by over 90%.
Loxo anticipates presenting additional data from the Phase 1 study at a leading medical meeting likely by the first half of 2016.
An update on the future direction of the ongoing Phase 1 trial will be provided in conjunction with the start of the Phase 2 trial.

Pre-clinical Programs

Prioritization of RET and FGFR Programs

Loxo’s collaboration with Array BioPharma continues to generate compelling and differentiated chemical matter. Loxo has used its multi-target, prioritize-by-success collaboration structure to focus on RET and FGFR.
Activating fusions and mutations in RET have been identified across a range of cancer histologies. Loxo is designing a highly specific RET inhibitor that optimizes on-target potency for RET fusions, activating mutations, and anticipated resistance mutations.
Activating fusions and mutations in FGFR3 and FGFR2 have also been identified across a range of cancer histologies. However, most small molecule FGFR inhibitors are functionally equipotent against isoforms FGFR1, FGFR2 and FGFR3, and are associated with metabolic and systemic toxicities that limit dose, duration of therapy and target engagement. Loxo is designing an FGFR1-sparing inhibitor that has the potential to avoid many of the side effects that have been endemic to the FGFR class.

Consistent with prior guidance, Loxo has submitted preclinical data abstracts for presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) meeting in November 2015 in Boston, MA. These data will provide evidence of some of the clinically-differentiating features of these programs. Loxo will provide IND guidance for one of these programs by the end of 2015.

Loxo has elected not to file an IND in the first half of 2016 on either of the two unnamed preclinical programs discussed in previous announcements. These programs have identified lead candidates, but still require additional target validation to merit clinical investigation, in the opinion of the Loxo scientific advisory board and management.

Second Quarter 2015 Financial Results

As of June 30, 2015 Loxo had aggregate cash, cash equivalents and investments of $101.8 million, compared to $112.9 million as of December 31, 2014.

The Company continues to expect cash burn of $30-$33 million in 2015, and based on the current operating plan, the Company believes existing capital resources will be sufficient to fund anticipated operations into 2017.

Research and development expenses were $5.7 million for the second quarter 2015 compared to $2.7 million for the second quarter 2014. The increase was primarily due to expanded clinical development activities for LOXO-101 and additional full-time equivalents and other support dedicated to discovery, preclinical, and manufacturing activities at Array BioPharma. The Company also recognized R&D-related stock-based compensation expense of $0.9 million during the second quarter of 2015 compared to $0.3 million for the second quarter of 2014.

Research and development expenses were $9.5 million for the six months ended June 30, 2015 compared to $4.8 million for the six months ended June 30, 2014. The increase was primarily due to expanded clinical development activities for LOXO-101 and additional full-time equivalents and other support dedicated to discovery, preclinical, and manufacturing activities at Array BioPharma. The Company also recognized R&D-related stock-based compensation expense of $1.4 million during the six months ended June 30, 2015 compared to $0.3 million for the six months ended June 30, 2014.

General and administrative expenses were $2.4 million for the second quarter 2015 compared to $1.2 million for the second quarter 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 $0.8 million during the second quarter of 2015 compared to $0.2 million for the second quarter of 2014.

General and administrative expenses were $4.8 million for the six months ended June 30, 2015 compared to $2.0 million for the six months ended June 30, 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 $1.3 million during the six months ended June 30, 2015 compared to $0.2 million for the six months ended June 30, 2014.

Net loss attributable to common shareholders was $8.1 million and $14.2 million for the three and six months ended June 30, 2015, respectively, compared to $3.9 million and $6.8 million for the three and six months ended June 30, 2014, respectively.

On August 5, 2015 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported financial results for the three and six months ended June 30, 2015 (Press release, Delcath Systems, AUG 5, 2015, View Source;p=RssLanding&cat=news&id=2075948 [SID:1234507026]).

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Highlights of the second quarter and recent weeks included:

Quarterly product revenue of $0.5 million, an increase of nearly 85.7% compared with the second quarter of 2014

Total operating expenses decreased by 35.1% to $4.0 million from $6.1 million in the second quarter of 2014

Enrollment opened for an intrahepatic cholangiocarcinoma (ICC) cohort of the Company’s global Phase 2 trial of Melphalan/HDS in hepatocellular carcinoma

Positive data regarding the Delcath Hepatic CHEMOSAT Delivery System highlighted at ASCO (Free ASCO Whitepaper) 2015

Exceeded 200 commercial treatments performed with CHEMOSAT since the product was launched in Europe

Multiple abstracts accepted for presentation at upcoming major medical conferences, including CIRSE, ESMO (Free ESMO Whitepaper), and EADO
Benefits of CHEMOSAT therapy to liver cancer patients affirmed by Key Opinion Leaders at European forum
Jennifer Simpson, Ph.D., MSN, CRNP named President and CEO

Granted U.S. Orphan Drug Designation for Melphalan/HDS for the treatment of cholangiocarcinoma from the U.S. Food and Drug Administration (FDA)

"We made considerable progress toward achieving our key milestones for 2015 as evidenced by continued revenue growth, increasing procedure volumes and advancement of our clinical development program," said Dr. Simpson. "We once again achieved record quarterly product revenue with year-over-year and sequential increases that have us already meeting our full-year 2014 sales on a local currency basis. This growth underscores our commercial progress in key European markets and highlights continued market adoption of CHEMOSAT.

"The opening of enrollment of the ICC cohort of our Phase 2 HCC study was a meaningful milestone as we believe our Melphalan/HDS treatment may offer significant clinical benefit for ICC patients who face limited treatment options. A positive efficacy signal may provide a regulatory path to a U.S. registration trial in this indication, and consolidated safety data from the HCC and ICC cohorts will offer valuable information for us to provide to the FDA. Our recent receipt of orphan drug designation from the FDA for melphalan for the treatment of cholangiocarcinoma, which includes ICC, highlights the medical need for viable treatments in this indication.

"We continue to advance our plans for a global pivotal Phase 3 clinical trial in ocular melanoma (OM) that has metastasized to the liver with overall survival as the primary endpoint. We hope to initiate this trial by the end of 2015.

"The positive feedback from the three-day Key Opinion Leader Forum we convened with leading clinicians from across Europe who are using CHEMOSAT was very encouraging. We look forward to these favorable findings being published in a white paper in a peer-reviewed journal.

"We are pleased with the meaningful progress made this quarter. With the additional resources secured through our recent public offering, we are well positioned to achieve several critical milestones during the second half of 2015," concluded Dr. Simpson.

Second Quarter Financial Results

Total revenue for the second quarter of 2015 of $0.5 million increased 85.7% from $0.3 million for the second quarter of 2014. Selling, general and administrative expenses during the second quarter of 2015 were $2.5 million, a decrease of $2.1 million or 45.6% from $4.6 million for the same period in 2014.

Total operating expenses for the second quarter of 2015 decreased by 35.1% to $4.0 million from $6.1 million for the same period in 2014. This decrease reflects a reduction in severance and compensation-related expenses following significant workforce restructurings throughout 2014 and into 2015, as well as a reduction in facility expenses following sub-leases of its 810 Seventh Ave location in 2014.

The Company recorded a net loss for the second quarter of 2015 of $3.7 million, a decrease of $0.9 million or 19.6%, compared with a net loss of $4.6 million for the same period in 2014. This decrease is primarily due to a $2.1 million reduction in operating expenses, a $0.1 million improvement in gross profit and a $1.3 million change in the fair value of the warrant liability, a non-cash item.

First Half Financial Result

Total revenue for the first half of 2015 of $0.9 million increased 62.2% from $0.6 million for the first half of 2014. Selling, general and administrative expenses during the first half of 2015 were $5.5 million, a decrease of $2.9 million or 34.1% from $8.4 million for the same period in 2014.

Total operating expenses for the first half of 2015 decreased by 29.9% to $8.0 million from $11.4 million for the same period in 2014. This decrease reflects a reduction in severance and compensation-related expenses following significant workforce restructurings throughout 2014 and into 2015, as well as a reduction in facility expenses following sub-leases of its 810 Seventh Ave location in 2014.

The Company recorded a net loss for the first half of 2015 of $7.2 million, a decrease of $2.7 million or 27.2% compared with a net loss of $9.9 million for the same period in 2014. This decrease is primarily due to a $3.4 million reduction in operating expenses, a $0.2 million improvement in gross profit and a $0.9 million change in the fair value of the warrant liability, a non-cash item.

Balance Sheet Highlights

Cash and cash equivalents as of June 30, 2015 were $14.1 million, compared with $20.5 million as of December 31, 2014. During the first six months of 2015, net cash used in operating activities was $9.0 million.

On August 5, 2015 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, reported two research projects involving the company’s Lm Technology immunotherapy candidate ADXS-PSA, which is being developed for prostate cancer, have been selected as 2015 Movember Foundation-PCF Challenge Awards, sponsored by the Movember Foundation and the Prostate Cancer Foundation (PCF) (Press release, Advaxis, AUG 5, 2015, View Source [SID:1234507025]). Grants amounting to $1 million each have been awarded to two ADXS-PSA teams conducting innovative large-scale research projects concerning metastatic, treatment-resistant prostate cancer, an advanced form of the disease with often-lethal outcomes.

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The Movember Foundation-PCF Challenge Awards support cross-disciplinary teams of investigators conducting pioneering research to address critical, unmet medical needs for prostate cancer patients. These highly competitive awards make large investments in multi-year projects that may fall outside the parameters of traditional funding organizations. Each proposal submitted to PCF was subjected to a rigorous peer review process that assessed the scientific merit of the project as well as its potential impact for patients. To date, the Movember Foundation has donated approximately $39 million to PCF to support innovative prostate cancer research, funding 28 research awards in the U.S., Canada and Great Britain.

Both ADXS-PSA projects awarded involve principal investigator Adam P. Dicker, M.D., Ph.D., Chair of the Department of Radiation Oncology at the Sidney Kimmel Cancer Center at Thomas Jefferson University, and will evaluate progression-free survival (PFS) as the primary endpoint and look at deep sequencing of T cell receptors to follow expansion of specific T cell clones.

"I look forward to having the opportunity to explore the potential of ADXS-PSA as immunotherapy in locally advanced and metastatic prostate cancer in two clinical trials," said Dr. Dicker. "These trials will help further the scientific understanding on the effects of targeting the prostate-specific antigen associated with prostate cancer."

The following ADXS-PSA proposals were selected to receive funding:

The first proposal, "CARAVAN (Checkpoint-Radiation-Vaccine Neoadjuvant) trial for metastatic prostate cancer," is led by researchers at the Sidney Kimmel Cancer Center at Thomas Jefferson University (Adam P. Dicker, M.D., Ph.D.) and the University of California, San Francisco (UCSF) Helen Diller Family Comprehensive Cancer Center (Lawrence Fong, M.D.). This is a Phase 1/2 study investigating intraprostatic anti-CTLA-4 and PD-1 blockade, plus radiation and ADXS-PSA in metastatic prostate cancer (N= ~42 +/-, its variable).

The second proposal, "Altering the natural history of metastatic prostate cancer using stereotactic ablative radiotherapy and immune stimulation," is led by researchers at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center (Phuoc T. Tran, M.D., Ph.D. and Ashley Ross, M.D., Ph.D.) and Sidney Kimmel Cancer Center at Thomas Jefferson University (Adam P. Dicker, M.D., Ph.D.). This is a Phase 1/2 study that combines stereotactic ablative radiation to prostate cancer metastases, followed by treatment with ADXS-PSA (N= ~36).

"We are tremendously honored to have ADXS-PSA recognized and supported in this way by the 2015 Movember Foundation-PCF Challenge Awards," said David J. Mauro, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Advaxis. "This funding will bring about the research necessary to potentially bring ADXS-PSA immunotherapy to people with metastatic prostate cancer, a disease that is expected to take the lives of 27,540 men in 2015."

Advaxis recently announced that enrollment has initiated in the Phase 1/2 KEYNOTE-046 clinical trial evaluating the combination of ADXS-PSA and Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with previously treated, metastatic castration-resistant prostate cancer (mCRPC). KEYNOTE-046 is the first-in-human study of ADXS-PSA and the second study initiated to evaluate the use of KEYTRUDA in the treatment of advanced prostate cancer. Data from preclinical studies suggest that Advaxis’s Lm Technology immunotherapies in combination with a PD-1 antibody may lead to an enhanced anti-tumor immune response.

About the Prostate Cancer Foundation

The Prostate Cancer Foundation (PCF) is the world’s leading philanthropic organization funding and accelerating prostate cancer research. Founded in 1993, PCF has raised more than $615 million and provided funding to more than 2,000 research programs at nearly 200 cancer centers and universities. The PCF global research enterprise now extends to 19 countries. PCF advocates for greater awareness of prostate cancer and more efficient investment of governmental research funds for transformational cancer research. Its efforts have helped produce a 20-fold increase in government funding for prostate cancer. For more information, click here.

About the Movember Foundation

The Movember Foundation is a global charity raising funds and awareness for men’s health. These funds deliver breakthrough research and support services to allow men to live longer, healthier, happier lives. Since 2003, millions have joined the men’s health movement, raising more than $650 million and funding over 1,000 programs through impact investments, focusing on prostate cancer, testicular cancer and poor mental health.

The Foundation runs awareness and fundraising activities year-round, with the annual Movember campaign in November being globally recognized for its fun and innovative approach to raising money and getting men to take action for their health. During Movember, we challenge men to grow a moustache or to make a commitment to get active and MOVE, both of which are about real action for health and are done to spark conversation and raise vital funds and awareness. The Foundation’s vision is to have an everlasting impact on men’s health.

Movember is fully accredited by the Better Business Bureau, and for the past three years, has been named a Top 100 best NGO by The Global Journal. For more information please visit Movember.com. Movember is a registered 501(c)(3) charity.

About the KEYNOTE-046 Trial

KEYNOTE-046 is a multicenter, dose determining, open-label Phase 1/2 study designed to evaluate the safety and efficacy of ADXS-PSA as a monotherapy and in combination with KEYTRUDA in approximately 51 mCRPC patients. Part A of the study will be a dose escalating study designed to establish the maximum tolerated dose of ADXS-PSA as a monotherapy. Part B will consist of a dose escalating trial of ADXS-PSA in combination with KEYTRUDA, followed by an expansion cohort phase. The primary objective is to evaluate safety and tolerability of the two immunotherapies, with the secondary objective to evaluate anti-tumor activity and PFS. Further information about KEYNOTE-046 can be found on ClinicalTrials.gov, using Identifier NCT02325557.

About Prostate Cancer

Prostate cancer is the second most common form of cancer affecting men in the United States: an estimated one in seven men will be diagnosed with prostate cancer in his lifetime. The American Cancer Society estimates that approximately 220,800 new cases of prostate cancer will be diagnosed and about 27,540 men are expected to die of the disease in 2015.

About ADXS-PSA

ADXS-PSA is an Lm Technology immunotherapy that is designed to target the prostate-specific antigen (PSA), a protein produced exclusively by prostate cells that is associated with prostate cancer. ADXS-PSA secretes the PSA antigen, fused to the powerful immunostimulant tLLO, directly inside the antigen presenting cells that are capable of driving a cellular immune response to PSA expressing cells. This approach is also designed to inhibit the Treg and myeloid-derived suppressor cells (MDSCs) that contribute to immunologic tolerance of prostate cancer. In preclinical analysis, ADXS-PSA inhibits the immunosuppression caused by Treg and MDSC cells localized inside tumors that may promote immunologic tolerance of prostate cancer.

On August 5, 2015 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company focused on the discovery, development, and commercialization of targeted cancer therapies, reported financial results for the second quarter 2015 ended June 30, 2015 and provided an update on its pipeline (Press release, Loxo Oncology, AUG 5, 2015, View Source [SID:1234507019]).

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"Over the last several months, Loxo has delivered important advances against our pipeline priorities," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "Last week, we achieved an important milestone with the peer-reviewed publication of the first clinical response to LOXO-101, our tropomyosin receptor kinase (TRK) inhibitor. This important early result validates the potential of Loxo’s drug development approach – that selective, purpose-built medicines in genetically well-defined populations can show dramatic and early evidence that justifies continued and rapid drug development. Today we are excited to announce plans for a Phase 2 trial for this program, which accelerate development timelines ahead of previous plans. In addition, we have made significant progress with our preclinical pipeline, allowing us to prioritize our Rearranged during Transfection (RET) and our isoform-sparing Fibroblast Growth Factor Receptor (FGFR) programs, positioning them as our likely next IND filings."

Program Updates

Loxo provided the following updates on its development programs:

LOXO-101: the only potent, oral, selective inhibitor of the TRK family of proteins

Clinical Validation for LOXO-101 Published in Cancer Discovery

The research brief published on July 27, 2015 in Cancer Discovery provides early clinical validation of LOXO-101 against TRK fusion cancer.

The publication, authored in collaboration with Foundation Medicine, Inc. and the Doebele Research Lab at the University of Colorado, includes the case study of the first TRK fusion patient treated in the Phase 1 trial of LOXO-101. A woman with advanced soft tissue sarcoma widely metastatic to the lungs enrolled on study and experienced substantial tumor regression, with imaging studies after one month demonstrating a partial response (PR) as defined by standard RECIST 1.1 criteria. The patient’s shortness of breath rapidly resolved and she was able to discontinue her supplemental oxygen and resume activities of daily living. As discussed in the publication, with four months of treatment, additional CT scans demonstrated almost complete tumor disappearance of the largest tumors. After four months of dosing, the patient did not have any adverse events that were attributed to LOXO-101.

The publication also describes novel assays for assessing LOXO-101’s impact on TRK signaling, patient-derived TRK fusion models in vitro and in vivo which illustrate LOXO-101’s TRK inhibition, and the first-ever description of the molecular epidemiology of TRK fusions in soft tissue sarcoma.

LOXO-101 Phase 2 Trial to Open in the Second Half of 2015

Based on evidence from the Phase 1 trial, including drug exposures that exceeded expectations, Loxo plans to initiate a Phase 2 trial in the second half of this year, earlier than previously anticipated. The trial will be a multicenter, international Phase 2 open label study in adult cancer patients whose tumors harbor TRK fusions. Loxo will provide additional details on the timing, size, and design of the Phase 2 trial after the first clinical site is open.
Loxo believes that a wide variety of tumor types harbor TRK fusions, and plans to work creatively with clinical partners to identify patients with these genetic alterations. Loxo will provide additional details on these plans after the first clinical site of the Phase 2 trial is open.

LOXO-101 Phase 1 Update

In the ongoing Phase 1 study, LOXO-101 continues to consistently achieve systemic drug exposures anticipated to inhibit TRK signaling by over 90%.
Loxo anticipates presenting additional data from the Phase 1 study at a leading medical meeting likely by the first half of 2016.
An update on the future direction of the ongoing Phase 1 trial will be provided in conjunction with the start of the Phase 2 trial.

Pre-clinical Programs

Prioritization of RET and FGFR Programs

Loxo’s collaboration with Array BioPharma continues to generate compelling and differentiated chemical matter. Loxo has used its multi-target, prioritize-by-success collaboration structure to focus on RET and FGFR.
Activating fusions and mutations in RET have been identified across a range of cancer histologies. Loxo is designing a highly specific RET inhibitor that optimizes on-target potency for RET fusions, activating mutations, and anticipated resistance mutations.
Activating fusions and mutations in FGFR3 and FGFR2 have also been identified across a range of cancer histologies. However, most small molecule FGFR inhibitors are functionally equipotent against isoforms FGFR1, FGFR2 and FGFR3, and are associated with metabolic and systemic toxicities that limit dose, duration of therapy and target engagement. Loxo is designing an FGFR1-sparing inhibitor that has the potential to avoid many of the side effects that have been endemic to the FGFR class.
Consistent with prior guidance, Loxo has submitted preclinical data abstracts for presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) meeting in November 2015 in Boston, MA. These data will provide evidence of some of the clinically-differentiating features of these programs. Loxo will provide IND guidance for one of these programs by the end of 2015.

Loxo has elected not to file an IND in the first half of 2016 on either of the two unnamed preclinical programs discussed in previous announcements. These programs have identified lead candidates, but still require additional target validation to merit clinical investigation, in the opinion of the Loxo scientific advisory board and management.

Second Quarter 2015 Financial Results

As of June 30, 2015 Loxo had aggregate cash, cash equivalents and investments of $101.8 million, compared to $112.9 million as of December 31, 2014.

The Company continues to expect cash burn of $30-$33 million in 2015, and based on the current operating plan, the Company believes existing capital resources will be sufficient to fund anticipated operations into 2017.

Research and development expenses were $5.7 million for the second quarter 2015 compared to $2.7 million for the second quarter 2014. The increase was primarily due to expanded clinical development activities for LOXO-101 and additional full-time equivalents and other support dedicated to discovery, preclinical, and manufacturing activities at Array BioPharma. The Company also recognized R&D-related stock-based compensation expense of $0.9 million during the second quarter of 2015 compared to $0.3 million for the second quarter of 2014.

Research and development expenses were $9.5 million for the six months ended June 30, 2015 compared to $4.8 million for the six months ended June 30, 2014. The increase was primarily due to expanded clinical development activities for LOXO-101 and additional full-time equivalents and other support dedicated to discovery, preclinical, and manufacturing activities at Array BioPharma. The Company also recognized R&D-related stock-based compensation expense of $1.4 million during the six months ended June 30, 2015 compared to $0.3 million for the six months ended June 30, 2014.

General and administrative expenses were $2.4 million for the second quarter 2015 compared to $1.2 million for the second quarter 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 $0.8 million during the second quarter of 2015 compared to $0.2 million for the second quarter of 2014.

General and administrative expenses were $4.8 million for the six months ended June 30, 2015 compared to $2.0 million for the six months ended June 30, 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 $1.3 million during the six months ended June 30, 2015 compared to $0.2 million for the six months ended June 30, 2014.

Net loss attributable to common shareholders was $8.1 million and $14.2 million for the three and six months ended June 30, 2015, respectively, compared to $3.9 million and $6.8 million for the three and six months ended June 30, 2014, respectively.