On March 14, 2016 ProNAi Therapeutics, Inc. (NASDAQ: DNAI), a clinical-stage oncology company advancing novel therapeutics for patients with cancer and hematological diseases, reported that its oncology drug candidate PNT2258 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of diffuse large B-cell lymphoma (DLBCL) (Press release, ProNAi Therapeutics, MAR 14, 2016, View Source [SID:1234509844]). This is the second orphan drug designation obtained by ProNAi for PNT2258 for the treatment of DLBCL, following a similar grant by the European Commission in August 2015.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Achieving this regulatory milestone for PNT2258 is an important advancement in our registration-oriented development plan for this cancer drug in DLBCL, a disease for which there are limited treatment options, particularly in patients who relapse or do not respond to front-line therapies." said Dr. Nick Glover, President and CEO of ProNAi.

Orphan drug designation is typically granted for novel drugs or biologics that are intended to treat rare medical diseases or conditions that affect less than 200,000 people in the United States. The designation qualifies the sponsor for certain incentives including seven years of market exclusivity after a drug’s approval, tax credits for clinical research costs, and certain application fee waivers.

ProNAi has also previously received European Commission Orphan Drug Designation for PNT2258 for the treatment of patients with DLBCL. In addition to a 10-year period of market exclusivity in the EU following marketing authorization, receiving orphan drug designation provides other incentives for companies, including scientific advice and protocol assistance during the product’s development phase.

About PNT2258 and DLBCL
ProNAi is actively enrolling patients in "Wolverine", a Phase 2 trial evaluating PNT2258 for the treatment of relapsed or refractory DLBCL and in "Brighton", a Phase 2 trial evaluating PNT2258 for the treatment of Richter’s transformation.

PNT2258 is designed to target cancers that overexpress BCL2, an important and validated oncogene known to be dysregulated in many types of cancer. BCL2 overexpression is thought to be a key driver of DLBCL, an aggressive form of cancer that is the most prevalent form of Non-Hodgkin lymphoma (NHL), comprising approximately 30% of the annual NHL diagnoses in the United States according to the Leukemia & Lymphoma Society (2013).

DLBCL affects mostly middle aged and older adults and is aggressive but potentially curable. First-line treatment of intensive combination chemotherapy involving rituximab may cure approximately 67% of patients. If this fails, second-line treatment is typically platinum-based chemotherapy along with continued rituximab. In the event that a response is achieved with second-line treatment, patients may be given a hematopoietic stem cell transplant. If second-line treatment or the transplant fails, patients are left with few options and little hope of a curative therapy. The median survival for third-line DLBCL patients is less than a year.

On March 14, 2016 WEST LAFAYETTE, Ind. A company from the Purdue Startup Class of 2014 whose innovation could help researchers and oncologists see faster than ever which drug therapies will benefit cancer patients and to what extent, reported that it has received funding from the National Institutes of Health (Press release, Purdue Research Foundation, MAR 14, 2016, View Source [SID:1234509561]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

KinaSense has received a one-year SBIR Phase l grant from the National Cancer Institute worth $203,120, with $50,000 in matching funds from the Indiana Economic Development Corporation and Elevate Ventures. The company’s technology is based on Purdue University intellectual property.

KinaSense’s technology measures the effects of cancer drugs that inhibit growth signals from a kinase, an enzyme in a cancer cell that causes the cell to grow. The technology takes information about what a particular kinase looks for in a substrate, or the protein it acts upon. It narrows the information to a shortlist of traits, which are used to design a molecular probe that reports whether or not the drug is blocking the target kinase’s action inside of the cell.

Steve Ouellette, co-founder and chief technology officer at KinaSense, said the grant will help the company begin laboratory operations.

"The SBIR funds allow us to begin working toward developing prototype tests that can be used in pre-clinical drug discovery to identify new treatments for cancer patients. Specifically, the tests we develop will be used to characterize inhibitors for a class of drug targets called receptor tyrosine kinases," he said. "The grant will also allow us benefits like having access to special programs offered by the NIH/NCI, such as I-Corps, which will help KinaSense mature as a company through specialized business development training."

Ouellette said the award is a major validation of the company’s vision and its technology.

"A lot of uncertainty was endured over the past year and a half developing research strategy, gathering support for the project from potential partners and performing due diligence on our business model," he said. "This grant is a rewarding culmination of that effort, and one of many major milestones toward realizing KinaSense’s mission to help save lives in the battle against cancer.

"I am infinitely grateful to all those who have assisted, especially the Purdue Foundry, our scientific advisers, Laurie Parker and Andrew Lipchik, and our early supporters, Horizon BioAdvance and the Elevate Purdue Foundry Fund."

Ouellette worked on the technology as a doctoral student when it was developed by Andrew Lipchik in the laboratory of Laurie L. Parker, then an assistant professor in Purdue’s College of Pharmacy. Parker and Lipchik co-founded KinaSense with Ouellette, and serve on its scientific advisory board.

About KinaSense

KinaSense is an early-stage biotechnology company based in West Lafayette, Indiana. Our mission is to help save lives in the battle against cancer. We develop novel tests for identifying new therapies and directing their use in the clinic. In doing so, we strive to be on the forefront of precision medicine for personalized cancer care.

On March 14, 2016 SRI International reported that it has been awarded a contract worth up to US$19.8m from the National Cancer Institute (NCI) in the US to support the development of potential preventative cancer agents or vaccines (Press release, SRI International, MAR 14, 2016, View Source [SID:1234509560]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the three-year contract, which is part of the PREVENT Cancer Preclinical Drug Development Programme, the Menlo Park, CA-based firm’s SRI Biosciences will conduct preclinical studies to assess the efficacy of specific compounds or vaccines for preventing invasive-cancer development.

Researchers will also identify biomarkers to help quantify the effectiveness of the experimental compounds and vaccines.

The PREVENT Cancer Drug Development Program is an NCI-supported pipeline to bring new cancer preventing interventions and biomarkers through preclinical development towards clinical trials.

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

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

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

On March 14, 2016 Janssen-Cilag International NV reported that data from a post-hoc analysis of the Phase 3 COU-AA-302 trial showed that ZYTIGA (abiraterone acetate) plus prednisone provided an 11.8 months overall survival (OS) benefit (53.6 months vs 41.8 months; HR = 0.61 [95% CI, 0.43-0.87]; p = 0.0055), compared to an active control of placebo plus prednisone, in men with early and less aggressive chemotherapy-naïve metastatic castration-resistant prostate cancer (mCRPC) (Press release, Johnson & Johnson, MAR 14, 2016, View Source [SID:1234509557]).1

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Data from the post-hoc analysis, presented today at the European Association of Urology (EAU) 2016 Congress in Munich, Germany, demonstrated almost triple the OS benefit previously shown (4.4 months) in the final analysis of the COU-AA-302 trial (34.7 months ZYTIGA plus prednisone vs 30.3 months placebo plus prednisone; HR = 0.81 [95% CI, 0.70-0.93]; p = 0.0033). The final analysis was originally presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2014 Congress and included a broader range of men with asymptomatic or mildly symptomatic chemotherapy-naïve mCRPC.2

The post-hoc analysis divided patients into two groups to identify which group experienced a greater treatment benefit. The patients in Group 1 were in an earlier, less advanced and less symptomatic stage of the disease (which was defined as having a Brief Pain Inventory [BPI] Short Form score of 0-1, prostate-specific antigen [PSA] below 80 ng/ml and a Gleason score [GS] of below 8). Those in Group 2 were in a later, more advanced and more symptomatic stage of the disease (defined as a having a BPI of 2 or over and/or PSA of 80 ng/ml or above, and/or a GS of 8 or more). The analysis revealed that patients in both groups experienced an OS benefit when treated with ZYTIGA plus prednisone, compared to placebo plus prednisone (Group 1: 11.8 months; HR = 0.61 [95% CI, 0.43-0.87]; p = 0.0055) (Group 2: 2.8 months; HR = 0.84 [95% CI, 0.72-0.99]; p = 0.0321).1

"Post-hoc analyses such as this are very important in helping us to identify the patients who could benefit most from therapies such as novel hormone agents, and at what stage of a patient’s disease they could be most effective." said Professor Kurt Miller, Department of Urology, Chariteì Berlin, Berlin, Germany. "As men with prostate cancer are living longer, quality of life is an increasingly important factor for them and their families. It is therefore encouraging to see that when used earlier, patients can stay on ZYTIGA for longer and delay the need for additional, more invasive treatments," he continued.

In addition to OS benefit, the post-hoc analysis data also revealed that both groups showed improvement in disease progression, cancer-related pain and treatment duration when treated with ZYTIGA plus prednisone, compared to placebo plus prednisone:

Time to chemotherapy use was increased by 12.7 months in Group 1 and 8.8 months in Group 2
Group 1: 37.0 months vs 24.3 months; HR = 0.64 [95% CI, 0.46-0.89]; p = 0.0073
Group 2: 23.3 months vs 14.5 months; HR = 0.71 [95% CI, 0.60-0.85]; p = 0.0001

There was an improvement in median time to opiate use for cancer-related pain in both groups
Group 1: not reached vs 41.0 months; HR = 0.69 [95% CI, 0.48-0.99]; p = 0.0409
Group 2: 30.5 months vs 19.3 months; HR = 0.70 [95% CI, 0.59-0.84]; p = 0.0001

Median time on treatment almost doubled in both groups
Group 1: 20.4 months vs 11.2 months; HR = 0.41 [95% CI, 0.31-0.54]; p < 0.0001
Group 2: 12.3 months vs 7.2 months; HR = 0.54 [95% CI, 0.46-0.62]; p < 0.0001

Jane Griffiths, Company Group Chairman, Janssen Europe, the Middle East and Africa (EMEA) said: "Janssen is proud that this study continues to deliver valuable insights as to how best to treat different stages of advanced prostate cancer. We hope that this additional analysis will help healthcare professionals to define the most effective treatment pathway for individual patients. We remain committed to continuing our research in this area with the aim of helping to improve outcomes for men affected by this disease now and in the future."

-ENDS-
NOTES TO EDITORS

About the COU-AA-302 study

COU-AA-302 is an international, randomised, double-blind, placebo controlled Phase 3 study that included 1,088 men with mCRPC who had not received prior chemotherapy and were randomised to receive ZYTIGA (abiraterone acetate) 1,000 milligrams (mg) administered orally once-daily plus prednisone 5 mg administered twice-daily or placebo plus prednisone 5 mg administered twice-daily. The co-primary endpoints of the study were rPFS and OS. Key secondary endpoints included time to opiate use, time to initiation of chemotherapy, time to Eastern Cooperative Oncology Group (ECOG) performance status deterioration and time to PSA progression. The final analysis was presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2014 Congress.

The post-hoc analysis used the final dataset for the intent-to-treat population (n = 1088), to stratify patients into Group 1 (BPI 0-1, PSA < 80 ng/ml and GS < 8) and Group 2 (BPI = 2 and/or PSA = 80 ng/ml and/or GS = 8). OS, radiographic progression-free survival (rPFS), time to CT use, time to opiate use, and time on treatment with ZYTIGA plus prednisone vs placebo plus prednisone were analysed by the Kaplan–Meier method and Cox proportional hazards regression.

About ZYTIGA (abiraterone acetate)

ZYTIGA is the only approved therapy that inhibits production of androgen, which fuels prostate cancer growth, via inhibiting the CYP17 enzyme complex present at three sources: the testes, adrenals and the tumour itself.

ZYTIGA has been approved in more than 90 countries and to date, has been prescribed to more than 269,500 men worldwide.

Indication3

In 2011, ZYTIGA in combination with prednisone/prednisolone was approved by the European Commission (EC) for the treatment of metastatic castration-resistant prostate cancer (mCRPC) in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen.

In December 2012, the EC granted an extension of the indication for ZYTIGA permitting its use, in combination with prednisone or prednisolone, for the treatment of mCRPC, in adult men who are asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated.3

Further Information3

For a full list of side effects and for further information on dosage and administration, contraindications and other precautions when using ZYTIGA, please refer to the summary of product characteristics, which is available at: View Source;mid=WC0b01ac058001d124

On March 14, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended December 31, 2015 (Filing, Q4/Annual, Stemline Therapeutics, 2015, MAR 14, 2016, View Source [SID:1234509551]).

Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "During the fourth quarter, we made significant progress across all three of our development programs. Key achievements included 1) generating and presenting data from our ongoing Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) which we have designed to support potential registration, 2) treating our first patient in the second stage of our Phase 2 program of SL-701 in advanced brain cancer, and 3) the opening of our IND for SL-801 on schedule. At the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December, we presented very strong data from our ongoing SL-401 trial in BPDCN, demonstrating a high overall response rate in BPDCN, with multiple complete responses. We are also very pleased with the pace of enrollment in BPDCN, and note that we are tracking in-line with our expectations. We look forward to sharing key clinical and regulatory updates on SL-401 throughout this year."

Dr. Bergstein continued, "We continue to evaluate SL-401 in several additional malignancies that overexpress IL-3R. Specifically, we have single agent trials enrolling AML patients with minimal residual disease and patients with advanced high-risk myeloproliferative neoplasms. We also expect our first clinical studies that combine SL-401 with other therapies will begin this year."

Dr. Bergstein concluded, "Our other pipeline candidates, SL-701 and SL-801, continue to advance. Our SL-701 trial is currently enrolling patients and we expect updates from this program in the second half of the year. The SL-801 IND is open, and we are on track to treat our first patient this quarter. With our strong cash position, we believe we have sufficient financial resources to achieve significant clinical and regulatory milestones this year and beyond."

Fourth Quarter 2015 Financial Results Review

Stemline ended the fourth quarter of 2015 with $97.5 million in cash, cash equivalents and investments, as compared to $104.0 million as of September 30, 2015, which reflects a cash burn of $6.5 million for the quarter. The company ended the fourth quarter of 2015 with 18.2 million shares outstanding.

For the fourth quarter of 2015, Stemline had a net loss of $10.2 million, or $0.58 per share, compared with a net loss of $6.9 million, or $0.53 per share, for the same period in 2014. The net loss for full year 2015 was $37.2 million, or $2.15 per share, as compared with a net loss of $28.8 million, or $2.23 per share, in the prior year.

Research and development expenses were $7.9 million for the fourth quarter of 2015, which reflects an increase of $2.8 million, or 56%, compared with $5.1 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to the ramp up in our clinical trial activities for SL-401 and SL-801.

General and administrative expenses were $2.6 million for the fourth quarter of 2015, which reflects an increase of $0.6 million, or 30%, compared with $2.0 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to an increase in compensation expense relating to administrative employees.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!