On May 13, 2017 Dendreon reported findings from a new analysis of its PROCEED registry, which followed men with metastatic castrate-resistant (hormone-refractory) prostate cancer (mCRPC) treated with PROVENGE (sipuleucel-T) in a real-world treatment setting (Press release, Dendreon, MAY 13, 2017, View Source [SID1234519101]). The analysis found that African-American patients demonstrated an additional median OS benefit of 9.3 months compared with Caucasian patients (37.3 months vs 28.0 months, respectively).i Among the group of patients below the median prostate specific antigen (PSA) levels at the time of PROVENGE treatment, African-American patients demonstrated an additional OS benefit of nearly two additional years (20.9 months) compared with Caucasian patients (54.3 months vs. 33.4 months, respectively).i

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"These new PROCEED registry data suggest that patients with asymptomatic or minimally symptomatic mCRPC may benefit the most with early use of PROVENGE and provide a rationale for immunotherapy as an early treatment strategy in sequencing algorithms for mCRPC"
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These compelling results were presented today in an oral podium presentation at the 112th American Urological Association (AUA) Annual Meeting in Boston by lead author A. Oliver Sartor, M.D., the Laborde Professor of Cancer Research in the Departments of Medicine and Urology at Tulane University School of Medicine. PROVENGE is the first and only U.S. Food and Drug Administration (FDA)-approved autologous cellular immunotherapy on the market.

"These new findings are very encouraging given that African-American men with prostate cancer have a mortality rate more than twice as high as Caucasian men and historically have presented with aggressive disease and have had worse outcomes in both real-world settings and controlled clinical trials," said Dr. Sartor. "The fact that we saw an even greater benefit in African-American patients within the lower PSA quartile ranges is also important and provides further evidence that PROVENGE should be used as early as possible within its labeled indication."

The PROCEED registry enrolled more than 1,900 patients with mCRPC who received PROVENGE between 2011 and 2013 in everyday treatment settings.i Of these, approximately 12 percent were African-American.i The analysis presented at the AUA meeting compared OS in a subset of African-American patients (n=210) and Caucasian patients (n=420) matched by baseline PSA.i

The analysis showed that the median OS was significantly greater in the African-American patients than in the Caucasian patients (37.3 months vs 28 months, p<0.001).i African-American patients also had better outcomes than Caucasian patients when OS was assessed based on the median PSA level (26.8 ng/mL) and by PSA quartiles.i Among those with a PSA level below the median, the OS was 54.3 months for African-American patients vs. 33.4 months for Caucasian patients – a difference of 20.9 months (p<0.001).i A multivariate analysis found that African-American race was an independent baseline predictor of improved OS (p<0.001) following treatment with PROVENGE.

The findings from the PROCEED analysis regarding the full population are consistent with an analysis of the Phase 3 IMPACT registration trial of PROVENGE published in Urology in 2013. In that analysis, a lower baseline PSA level was associated with a greater overall survival benefit with PROVENGE. Among patients with a baseline PSA ≤22.1 ng/mL, the median OS was 41.3 months for those treated with PROVENGE vs. 28.3 months for those in the control arm – an improvement of 13 months.ii

"These new PROCEED registry data suggest that patients with asymptomatic or minimally symptomatic mCRPC may benefit the most with early use of PROVENGE and provide a rationale for immunotherapy as an early treatment strategy in sequencing algorithms for mCRPC," said James Caggiano, president of Dendreon. "We are pleased to be able to provide this new clinical data about how metastatic prostate cancer patients respond to and benefit from PROVENGE in everyday clinical practice. It should be useful to urologists and oncologists in supporting treatment decisions for their patients, especially their African-American patients, who typically are more likely to be diagnosed with advanced disease and to have higher mortality."

About Prostate Cancer in African-American Men

Prostate cancer is the most frequently occurring non-cutaneous cancer among men in the United States and is second only to lung cancer among the leading causes of cancer-related deaths.iii

Prostate cancer is also the most commonly diagnosed cancer in African-American men, representing 31 percent of all cancers.iv It is estimated that in 2016, one in six African-American men were diagnosed with prostate cancer – an estimated 29,530 new cases – and one in 23 had a lifetime probability of dying from their disease.v The incidence of prostate cancer is 60 percent higher among African-American than Caucasian men, and the mortality rate is more than twice as high,vi which prostate cancer incidence patterns from population modeling suggest is likely due to a higher incidence of preclinical disease and higher risk of progression to metastatic disease before clinical diagnosis among African-American men compared with the general population.vii Based on this modeling, African-American men are more likely to be diagnosed with prostate cancer at a younger age and a higher stage and to have their disease progress after treatment compared with Caucasian men.vi The lifetime probability of an African-American man dying of prostate cancer is almost double that of a Caucasian man (4.4 percent vs. 2.4 percent).iv

About PROVENGE (sipuleucel-T)

PROVENGE (sipuleucel-T) is an autologous cellular immunotherapy indicated for the treatment of asymptomatic or minimally symptomatic metastatic castrate-resistant (hormone refractory) prostate cancer.

IMPORTANT SAFETY INFORMATION

Acute Infusion Reactions: Acute infusion reactions (reported within 1 day of infusion) may occur and include nausea, vomiting, fatigue, fever, rigor or chills, respiratory events (dyspnea, hypoxia, and bronchospasm), syncope, hypotension, hypertension, and tachycardia.

Thromboembolic Events: Thromboembolic events, including deep venous thrombosis and pulmonary embolism, can occur following infusion of PROVENGE. The clinical significance and causal relationship are uncertain. Most patients had multiple risk factors for these events. PROVENGE should be used with caution in patients with risk factors for thromboembolic events.

Vascular Disorders: Cerebrovascular events (hemorrhagic/ischemic strokes and transient ischemic attacks) and cardiovascular disorders (myocardial infarctions) have been reported following infusion of PROVENGE. The clinical significance and causal relationship are uncertain. Most patients had multiple risk factors for these events.

Handling Precautions: PROVENGE is not tested for transmissible infectious diseases.

Concomitant Chemotherapy or Immunosuppressive Therapy: Chemotherapy or immunosuppressive agents (such as systemic corticosteroids) given concurrently with the leukapheresis procedure or PROVENGE has not been studied. Concurrent use of immune-suppressive agents may alter the efficacy and/or safety of PROVENGE.

Adverse Reactions: The most common adverse reactions reported in clinical trials (≥ 15% of patients receiving PROVENGE) were chills, fatigue, fever, back pain, nausea, joint ache, and headache.

For full Prescribing Information, please visit View Source

On May 12, 2017 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported new data demonstrating the utility of the Prolaris test to accurately predict the 10-year risk of metastases in men treated for prostate cancer (Press release, Myriad Genetics, MAY 12, 2017, View Source [SID1234519100]). The data are being presented tomorrow at the American Urological Association (AUA) 2017 Annual Meeting in Boston, Mass.

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This study was conducted in collaboration with Stephen Bardot, M.D., and colleagues at Ochsner Clinic in New Orleans, Louisiana. A total of 767 men with localized prostate cancer were evaluated using the Prolaris test plus CAPRA (i.e., clinical features) to predict the risk of metastatic disease up to 10 years following diagnosis. Approximately 40 percent of the patients in the study were African Americans (AA). Among all 767 patients 39 men, or 5.1 percent, developed metastases and among the 646 men who received definitive therapy (e.g., surgery, radiation, radiation and hormones) 28 men, or 4.3 percent, developed metastatic disease.

The results showed that the Prolaris test was a significant predictor of metastatic disease with a nearly 3-fold increased risk for each one-unit increase on the Prolaris test score (Hazard Ratio per unit score = 2.76; P = 2.8×10-11). Importantly, there was no difference in predictive performance between races (p=0.20) or treatment groups (p=0.09). When combined with CAPRA the Prolaris test was highly predictive of metastatic disease (HR for combined clinical risk (CCR) = 3.86; p= 2.8×10-23). Contrary to expectations, this large study found no evidence that AA men have more aggressive prostate cancer than non-AA men after accounting for all molecular and clinical information.

"Our study confirmed that the Prolaris test significantly predicts which men are likely to develop metastatic disease, regardless of race, risk group or treatment approach," said Dr. Bardot. "This study also included a large group of AA men which have historically been underrepresented in clinical outcomes studies. This study demonstrated that Prolaris provided more accurate precision in providing prognosis in African American and non-African Americans equally."

The findings from the current study are consistent with the findings of an earlier study that demonstrated the ability of the Prolaris test in predicting cancer progression, as measured by both biochemical recurrence and metastatic disease, after radical prostatectomy. That prior study found that patients with a high Prolaris test score had a six-fold higher risk of developing metastases compared to low risk patients.

Based on the strength of the data from these two outcomes studies, Myriad has added risk of metastases to the Prolaris test report for clinicians, making Prolaris the first and only genetic test to provide this endpoint as validated by two outcomes studies.

"Myriad Genetics is committed to innovation and being the leader in genetic testing for men diagnosed with prostate cancer," said Michael Brawer, M.D., senior vice president of Medical Affairs, Myriad Genetic Laboratories. "We have multiple outcome studies that show the ability of Prolaris to predict the 10-year risk of prostate cancer specific mortality, and we now have two studies that predict the risk that treatment will fail and men will end up with metastatic disease. We are excited to provide all of this relevant information in a single test report for clinicians."

Follow Myriad on Twitter via @MyriadGenetics and stay informed about symposium news and updates by using the hashtag #AUA17. Details of the presentation at AUA follow.

Featured Presentation
Title: Evaluating the Prognostic Utility of the CCP Score for Predicting Prostate Cancer Aggressiveness in African American Men
Presenter: Steven Bardot, M.D., Ochsner Medical Center
Date: Saturday, May 13, 2017: 7:00-9:00 a.m. ET.
Location: Moderated Poster MP28-19; Room 253AB.

About Prolaris
Prolaris is a novel 46-gene RNA-expression test that directly measures tumor cell growth characteristics for stratifying the risk of disease-specific mortality in patients with prostate cancer. Prolaris provides a quantitative measure of the RNA expression levels of genes involved in the progression of tumor growth. Low gene expression is associated with a low risk of disease-specific mortality in men who may be candidates for active surveillance and high gene expression is associated with a higher risk of disease-specific mortality in patients who may benefit from additional therapy. For more information visit: www.prolaris.com.

On May 12, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to larotrectinib for the "treatment of solid tumors with NTRK-fusion proteins." NTRK fusions are genetic abnormalities that occur rarely in various adult and pediatric solid tumors.

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The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

About Larotrectinib (LOXO-101)
Larotrectinib (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, larotrectinib has demonstrated encouraging preliminary efficacy. Larotrectinib is also being evaluated in the NAVIGATE global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions, and the SCOUT Phase 1/2 trial in pediatric patients, including patients with advanced cancer, TRK gene fusions and infantile fibrosarcoma. Larotrectinib has been granted Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation by the U.S. FDA. For additional information about the larotrectinib 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 or visit www.loxooncologytrials.com.

On May 12, 2017 Celsion Corporation (NASDAQ:CLSN), an oncology drug development company, reported financial results for the quarter ended March 31, 2017 and provided an update on its development programs for ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin and GEN-1, an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein (Press release, Celsion, MAY 12, 2017, View Source [SID1234519075]). The Company’s lead program is ThermoDox which is currently in Phase III development for the treatment of primary liver cancer and in Phase II development for the treatment of recurrent chest wall breast cancer. The Company’s immunotherapy program consists of GEN-1 and is currently in Phase I development for the localized treatment of ovarian cancer.

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"Celsion continues to make major progress with respect to our ongoing global, pivotal Phase III OPTIMA Study in primary liver cancer. This ground-breaking study continues to attract interest and support from the medical community, international regulatory agencies, and research organizations like the National Institutes of Health," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "Our product development efforts in immuno-oncology are equally important. We have demonstrated the potential of our GEN-1 IL-12 immunotherapy program to be an effective adjuvant, in both first and second-line ovarian cancer. Recruiting the immune system to work in combination with the standard of care in this patient population has been the goal of medical researchers worldwide. With GEN-1, we believe there is the potential for a break-through and we look forward to reporting comprehensive clinical results and translational research data from our Phase 1B OVATION Study at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2017."

Recent Developments
ThermoDox
Announced the Publication of Preclinical Results of ThermoDox for the Treatment of Bladder Cancer in the International Journal of Hyperthermia. The Company reported results from porcine in vivo studies to evaluate ThermoDox in combination with loco-regional mild hyperthermia for targeted drug delivery to the bladder wall as a potential treatment for bladder cancer. Doxorubicin accumulation and distribution within the bladder wall with ThermoDox plus mild bladder hyperthermia was achieved at concentrations nearly ten times higher than with free intravenous doxorubicin combined with mild bladder hyperthermia. The study was conducted under a Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health (NIH) to evaluate whether
ThermoDox combined with mild heating of the bladder can target drug delivery in the bladder.

Announced Support for the OPTIMA Study from the China FDA and Vietnam Ministry of Health. The Company discussed ThermoDox and the OPTIMA Study with regulatory agencies in two key markets, China and Vietnam. The Company met with the China Food and Drug Administration (CFDA) to review the ongoing Phase III OPTIMA Study and regulatory pathway for ThermoDox in China. CFDA was presented with the final overall survival data from the Chinese patient cohort of the HEAT study, which demonstrated a survival benefit in patients treated with ThermoDox plus optimized RFA versus optimized RFA alone. The CFDA informed the Company that if the ongoing Phase III OPTIMA Study is successful, the trial could serve as the basis for a direct regulatory filing in China without the need to file for prior approval in the U.S. or European Union which is currently required for foreign company application. This would allow the Company to accelerate its plans for a regulatory filing in China and, if approved, provide for a significantly earlier launch date in China than originally expected. The Company’s management team also met with the Ministry of Health in Vietnam and based on that meeting, it will move forward with launching additional trial sites for the OPTIMA Study in that country. The Company plans to activate 5 additional clinical trial sites in Vietnam by the second quarter of 2017. Vietnam represents a significant market for ThermoDox where HCC incidence rates are among the highest in the world.

Announced the Issuance of Two New Patents for ThermoDox. In January 2017, the Company announced the issuance of two patents which are directly applicable to the method of treating cancer using our current ThermoDox formulation. These new patents further strengthen the Company’s global patent portfolio around novel heat-sensitive liposome engineered to address a broad range of difficult-to-treat cancers.

GEN-1 Immunotherapy
Announced Continuing Positive Data from the OVATION Study in Newly Diagnosed Advanced Ovarian Cancer Patients. In January 2017, the Company announced data from the first four cohorts of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1 with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery. In the first twelve patients dosed in the OVATION Study, GEN-1 plus standard chemotherapy produced impressive results, with no dose limiting toxicities and highly promising efficacy signals in this difficult to treat cancer. The efficacy data included highly encouraging tumor response rates – 100% disease control rate (DCR) and 75% objective response rate (ORR), successful surgical resections of the eligible patients’ tumors, impressive pathological responses and dramatic, clinically meaningful drops in CA-125 protein levels. In February 2017, the Company presented two posters at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Clinical Immuno-Oncology Symposium held from February 23 – 25, 2017 in Orlando, FL. The ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium focused on the latest clinical and translational research in immuno-oncology and the implications for clinical care.

Corporate Development
Raised $6.8 Million Through Two Equity Offerings in December 2016 and February 2017. The Company completed two equity offerings of shares of common stock, or pre-funded warrants in lieu thereof, to purchase common stock with institutional healthcare and retail investors totaling $6.8 million in gross proceeds.

Financial Results
For the quarter ended March 31, 2017, Celsion reported a net loss of $5.2 million, or $0.12 per share, compared to a net loss of $5.7 million, or $0.24 per share, in the same period of 2016. Operating expenses were $4.9 million in the first quarter of 2017 compared to $5.3 million in the same period of 2016. This decrease was primarily due to lower general and administrative expenses.

Research and development (R&D) costs were relatively constant at $3.5 million and $3.4 million in the first quarters of 2017 and 2016, respectively. Clinical development costs for the Phase III OPTIMA Study were $1.6 million in the first quarter of 2017 compared to $1.0 million in the same period of 2016 due to higher patient enrollment and investigator grant expenses in the trial. R&D costs for other development programs were lower as a result of the Company’s tighter clinical development focus around the pivotal Phase III OPTIMA Study for the treatment of primary liver cancer and the clinical development program for GEN-1 IL-12 immunotherapy for the localized treatment of ovarian cancer coupled with lower costs in the first quarter of 2017 associated with the production of ThermoDox clinical supplies to support the OPTIMA Study. General and administrative expenses decreased $0.4 million, from $1.9 million in the first quarter of 2016 to $1.5 million in the first quarter of 2017. This 21% decrease in general and administrative expenses in 2017 is primarily the result of reduction in personnel costs and lower professional fees.

Net cash used in operations was $3.1 million in the first quarter of 2017 compared to $4.7 million in the same period of 2016. The Company ended the first quarter of 2017 with $4.5 million of total cash and cash equivalents. In February 2017, the Company raised $5 million in gross proceeds under a secondary public offering with various institutional and retail investors.