Intravenous Busulfan-Based Myeloablative Conditioning Regimens Prior to Hematopoietic Cell Transplantation for Hematologic Malignancies.

Busulfan (Bu)-containing regimens are commonly used in myeloablative regimens prior to allogeneic hematopoietic cell transplantation (HCT). Yet, there is considerable variability on how Bu is administered related to frequency (four times a day, Q6 or daily, Q24) and the combination with other chemotherapeutic agents (cyclophosphamide, Cy or fludarabine, Flu). A prospective cohort study of recipients of Bu-based conditioning according to contemporary practices was used to compare different approaches in using Bu (BuCy Q6 N=495; BuFlu Q24 N=331; BuCy Q24 N=96; BuFlu Q6 N=91) in patients with myeloid malignancies from 2009 to 2011. BuFlu Q24 recipients were more likely to be older and tended to have worse performance status and higher comorbid burden. The cumulative incidence of hepatic veno-occlusive disease (p=0.4), idiopathic pneumonia (p=0.5) and seizures (p=0.5) did not differ across groups. One-year HCT related mortality ranged from 12% to 16% (P=0.8), three-year relapse incidences ranged from 32% to 36% (p=0.8) and three-year overall survival ranged from 51% to 58% (p=0.2) across groups. This study demonstrates that the use of intravenous Bu Q6 or Q24 or accompanied by Cy or Flu have similar outcomes in the myeloablative setting for treatment of myeloid malignancies.
Copyright © 2016 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

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Myriad to Present Three New Studies at the AUA Annual Meeting

On May 06, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported that results from three studies will be featured at the American Urological Association annual meeting, which will take place May 6-10 in San Diego, Calif (Press release, Myriad Genetics, MAY 6, 2016, View Source [SID:1234512009]). Poster discussions include new data for the Prolaris test in patients with low-risk prostate cancer, as well as investigational molecular diagnostic tests for renal and bladder cancer.

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"Prolaris is the leading prognostic genetic test for patients with prostate cancer and is the only such test that predicts the 10-year risk of real oncologic outcomes including death, metastases and recurrence. The evidence in favor of genetic testing is expanding, and we’re excited to present a new analysis at AUA that further confirms the strong prognostic power of Prolaris in men with low-risk localized prostate cancer," said Michael Brawer, senior vice president of Medical Affairs, Myriad Genetic Laboratories. "We also are presenting new data for our investigational renal and bladder cancer tests, which further underscore Myriad’s commitment to developing pioneering molecular diagnostic tests for other urologic diseases."

Results of the studies to be presented are described below and abstracts are now available at: www.aua2016.org/abstracts/. Follow Myriad on Twitter via @MyriadGenetics to stay informed about news and updates from the Company.

Highlighted Presentations

Title: The CCP score provides significant prognostic information in Gleason score <6 patients.
Date: Friday, May 6, 2016: 8:00—10:00 a.m. PT.
Location: Poster MP2.
Presenter: Jay Bishoff, M.D., Intermountain Urological Institute.
This meta-analysis of five studies evaluated the ability of the Prolaris test (CCP score) to predict oncologic outcomes (i.e., recurrence or death) in 440 patients with low-risk localized prostate cancer, which was defined as a Gleason score of 6 or less. The results showed that the Prolaris test is a significant predictor of oncologic outcomes in patients with low-risk disease (HR 1.5; p<0.009). Prolaris also was a better independent predictor of outcomes than traditional clinical features as measured by CAPRA (Cancer of the Prostate Risk Assessment; HR 1.27; p<0.03). When the Prolaris and CAPRA scores were assessed together, the combined clinical risk (CCR) score provided even greater predictive power (HR 1.83; p<0.0014). In this study, Prolaris was a strong predictor of the 10-year risk of oncologic outcomes in patients with localized prostate cancer and a Gleason score of 6 or less.

Title: A study to evaluate the prognostic and predictive utility of CCP and HRD assays and genetic sequencing in patients undergoing neoadjuvant chemotherapy in bladder cancer.
Date: Sunday, May 8, 2016: 1:00—3:00 p.m. PT.
Location: Poster MP49.
Presenter: Hristos Kaimakliotis, M.D., Indiana University.
This exploratory study evaluated three molecular assays to determine if they were able to predict response to neoadjuvant chemotherapy with cisplatin in patients with urothelial bladder cancer (UBC). The assays included 1) a cell cycle progression score, 2) the homologous recombination deficiency (HRD) score, and 3) genetic sequencing of a set of 80 genes associated with UBC. The results showed that RB1 mutations were associated with response to cisplatin neoadjuvant chemotherapy, and the predictive ability was improved by the addition of either the CCP or HRD scores. Additionally, HRD could be used to predict risk of disease recurrence in patients after neoadjuvant chemotherapy followed by cystectomy. If validated, these tests may help identify chemo-responsive patients.

Title: Prognostic utility of a multi-gene signature (the cell cycle proliferation score) in patients with renal cell carcinoma (RCC) after radical nephrectomy.
Date: Monday, May 9, 2016: 3:30—5:30 p.m. PT.
Location: Poster MP78.
Presenter: Adam Feldman, M.D., Massachusetts General Hospital.
The objective of this study was to assess the ability of the Myriad myPlan Renal Cancer cell cycle progression test to predict long-term oncologic outcomes in patients with surgically-resected renal cell carcinoma (RCC). Outcomes were defined as disease recurrence (local or metastatic) or disease-specific survival (DSS). Patient data were censored at five-years of follow-up. In the training cohort (N= 305), the myPlan Renal Cancer test was a significant prognostic predictor for recurrence (HR: 1.74; p = 0.02) and DSS (HR: 2.59; p< 0.001) after adjusting for clinical variables. The validation cohort (N=262) demonstrated a consistent and significant prediction of recurrence and DSS, with the strongest association being for DSS (HR: 2.2; p < 0.001) after adjusting for clinical variables. Based on these data, the myPlan Renal Cancer test appears to be a significant and independent predictor of key long-term oncologic outcomes in patients who have undergone nephrectomy for RCC, providing prognostic information beyond what is available from clinical parameters. Additional studies are underway to evaluate the utility of the score when derived from diagnostic biopsy.

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.

About myChoice HRD
Myriad’s myChoice HRD is the first homologous recombination deficiency test that can detect when a tumor has lost the ability to repair double-stranded DNA breaks, resulting in increased susceptibility to DNA-damaging drugs such as platinum drugs or PARP inhibitors. High myChoice HRD scores reflective of DNA repair deficiencies are prevalent in all breast cancer subtypes, ovarian and most other major cancers. In previously published data, Myriad showed that the myChoice HRD test predicted drug response to platinum therapy in certain patients with triple-negative breast and ovarian cancers. It is estimated that 1.8 million people in the United States and Europe who are diagnosed with cancers annually may be candidates for treatment with DNA-damaging agents. For more information visit: www.myriad.com.

About Myriad myPlan Renal Cancer
Myriad myPlan Renal Cancer is a molecular prognostic test that measures the expression levels of cell cycle progression genes to provide an accurate assessment of cancer aggressiveness in patients with renal cell carcinoma. For more information visit: View Source

8-K – Current report

On May 6, 2016 Mast Therapeutics, Inc. (NYSE MKT: MSTX), a biopharmaceutical company developing novel, clinical-stage therapies for sickle cell disease and heart failure, reported financial results for the first quarter ended March 31, 2016 (Filing, Q1, Mast Therapeutics, 2012, MAY 6, 2016, View Source [SID:1234512073]).

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"The first quarter of 2016 was a productive one for Mast. Not only did we complete enrollment in our Phase 3 EPIC study of vepoloxamer in sickle cell disease, but also we announced positive data from a Phase 2a study of AIR001 in patients with heart failure with preserved ejection fraction conducted at Mayo Clinic, and the selection of AIR001 for a double-blind, placebo-controlled Phase 2 study in approximately 100 patients with HFpEF to be conducted at premier U.S. clinical centers that make up the HFN," stated Brian M. Culley, Chief Executive Officer.

"With 388 patients, the EPIC study was the largest placebo-controlled study in sickle cell disease ever concluded and should provide many insights into the activity of vepoloxamer in this indication. Importantly, vepoloxamer has the potential to become the first and only approved therapy for shortening the duration of a sickle cell vaso-occlusive crisis and we are working diligently toward generating top-line results, which we expect to announce this quarter," continued Mr. Culley. "Meanwhile, we are advancing our two heart failure programs. Our 150-patient Phase 2 study of vepoloxamer in chronic heart failure is ongoing, with ten study sites now open, and the HFN’s 100-patient Phase 2 study of AIR001 in HFpEF is expected to begin in the third quarter of 2016."

First Quarter 2016 Operating Results

The Company’s net loss for the first quarter of 2016 was $11.2 million, or $0.06 per share (basic and diluted), compared to a net loss of $9.6 million, or $0.06 per share (basic and diluted), for the same period in 2015.

Research and development expenses for the first quarter of 2016 were $7.9 million, an increase of $1.9 million, or 30%, compared to $6.0 million for the same period in 2015. The increase was due mainly to increases of $0.9 million in external nonclinical study fees and expenses, $0.5 million in external clinical study fees and expenses, and $0.3 million in personnel expenses.

The increase in external nonclinical study fees and expenses was due primarily to increased costs related to preparation for a new drug application for vepoloxamer ($0.5 million) and research-related manufacturing for vepoloxamer ($0.5 million), offset by a decrease in research-related manufacturing for AIR001 ($0.1 million). The increase in external clinical study fees and expenses was due primarily to increased costs related to the Phase 2 study of vepoloxamer in heart failure ($0.5 million) and the EPIC study ($0.3 million), offset by a decrease

related to discontinuation of a Phase 2 study of vepoloxamer in acute limb ischemia, which the Company began to wind-down in the third quarter of 2015 ($0.3 million).

Selling, general and administrative (SG&A) expenses for the first quarter of 2016 were $2.8 million, a decrease of $0.8 million, or 21%, compared to $3.6 million for the same period in 2015. SG&A expenses for the first quarter of 2015 included $0.4 million of severance expenses and $0.3 million of share-based compensation resulting from the termination of employment of the Company’s former president and chief operating officer in February 2015 and the acceleration of stock option vesting pursuant to the terms of his option agreements.

Interest expense for the first quarter of 2016 was $0.5 million, which was related to the Company’s debt facility. There was no interest expense for the first quarter of 2015.

Spectrum Pharmaceuticals Highlights an Oral Presentation on Apaziquone at the 2016 Annual Meeting of the American Urological Association Education and Research Inc. (AUA) in San Diego, California, May 6-10, 2016

On May 6, 2016 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported presentations of clinical data for apaziquone to be presented at an oral presentation and a moderated poster session at the American Urological Association Education and Research Inc., being held in San Diego, California, from May 6-10, 2016 (Press release, Spectrum Pharmaceuticals, MAY 6, 2016, View Source [SID:1234512013]).

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For more information about the AUA Meeting and for a complete list of abstracts, please refer to the conference web site at View Source

Friday, May 6, 2016 1:00 PM-3:00 PM PT (Moderated Poster Session)

Abstract # Type Title First Author Location

MP13-07
Poster
Improved Efficacy of Adjuvant, Single Dose Intravesical Apaziquone by Timing post-Resection in Two Double
blind, Randomized, Placebo-Controlled Phase 3 Studies in Non-Muscle Invasive Bladder Cancer
Fred Witjes
Room 28 ABC

Saturday, May 7, 2016, 8:00 AM-10:00 AM PT (Podium Presentation)

Abstract # Type Title First Author Location

PD11-07
Podium
Integrated Results of Two Multicenter, Randomized, Placebo Controlled, Double Blind, Phase 3 Trials (SPI-611/612) of Single-Dose Intravesical Apaziquone Immediately Following Resection in Patients with Non-Muscle Invasive Bladder Cancer

6-K – Report of foreign issuer [Rules 13a-16 and 15d-16]

On May 6, 2016 Oncolytics Biotech Inc. (TSX: ONC) (OTCQX: ONCYF) (FRA: ONY) ("Oncolytics" or the "Company") reported its financial results and operational highlights for the first quarter ended March 31, 2016 (Filing, Q1, Oncolytics Biotech, 2016, MAY 6, 2016, View Source [SID:1234512055]).

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"We recently reported data from two sponsored, randomized Phase 2 studies, highlighting reduced tumor burden in ovarian cancer and improved longer term survival in pancreatic cancer," said Dr. Brad Thompson. "The data from these studies will be very useful in identifying potential indications and study designs that we could advance into later stage studies in the future."

Selected Highlights

Since January 1, 2016, selected highlights announced by the Company include:

Clinical Program

· Treatment of the first patients in a Phase Ib study of pembrolizumab (KEYTRUDA) in combination with REOLYSIN and chemotherapy in patients with advanced pancreatic adenocarcinoma, the Company’s first trial examining REOLYSIN in combination with a checkpoint inhibitor;
· Updated results from a randomized Phase 2 clinical trial of its lead product, REOLYSIN, in combination with paclitaxel in patients with ovarian cancer (GOG-0186H), where an intent-to-treat analysis, as assessed by CA-125 antigen levels, showed statistically significantly reduction in tumor burden;
· Updated results from a randomized Phase 2 clinical trial of its lead product, REOLYSIN, in combination with carboplatin and paclitaxel in patients with pancreatic cancer (NCI-8601), where an intent-to-treat analysis of overall survival on patients with confirmed treatment regimes, as assessed by the percentage of patients surviving for two years, showed a statistically significantly higher percentage of patients surviving two years in the test arm versus the control arm (p = 0.001), the crossover arm versus the control arm (p = 0.03) and the test plus crossover arms versus the control arm (p = 0.0004);
Basic Research

· Two poster presentations covering preclinical work in multiple myeloma and colorectal cancer being made by the Company’s research collaborators at the 2016 American Association of Cancer Research annual meeting;
Financial

· Entry into an "at-the-market" equity distribution agreement with Canaccord Genuity Corp. permitting Oncolytics at its sole discretion, from time to time and until March 16, 2018, to sell common shares having an aggregate offering value of up to $4.6 million; and
· At March 31, 2016 the Company reported $22.3 million in cash, cash equivalents and short-term investments. At May 5, 2016, the Company had approximately $21.4 million in cash, cash equivalents and short-term investments, which is expected to provide sufficient funds to support several small early-stage immunotherapy combination studies as well as both a run-in and a registration study in muscle-invasive bladder cancer.

ONCOLYTICS BIOTECH INC.
INTERM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION

As at March 31,
2016
$ December 31,
2015
$
Assets
Current assets
Cash and cash equivalents 20,233,408 24,016,275
Short-term investments 2,088,800 2,060,977
Accounts receivable 59,648 340,059
Prepaid expenses 229,288 506,669
Total current assets 22,611,144 26,923,980

Non-current assets
Property and equipment 411,762 459,818
Total non-current assets 411,762 459,818

Total assets 23,022,906 27,383,798

Liabilities And Shareholders’ Equity
Current Liabilities
Accounts payable and accrued liabilities 2,554,338 2,709,492
Total current liabilities 2,554,338 2,709,492

Shareholders’ equity
Share capital
Authorized: unlimited
Issued:
March 31, 2016 – 118,697,122
December 31, 2015 – 118,151,622 261,224,148 261,324,692
Contributed surplus 26,359,606 26,277,966
Accumulated other comprehensive income 590,919 760,978
Accumulated deficit (267,706,105) (263,689,330)
Total shareholders’ equity 20,468,568 24,674,306
Total liabilities and equity 23,022,906 27,383,798

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF LOSS AND COMPREHENSIVE LOSS

2016 2015
For the three month period ending March 31 $ $
Expenses
Research and development 2,726,129 2,425,539
Operating 1,360,412 1,182,734
Operating loss (4,086,541) (3,608,273)
Interest 69,621 56,435
Loss before income taxes (4,016,920) (3,551,838)
Income tax expense 145 —
Net loss (4,016,775) (3,551,838)
Other comprehensive income items that may be
reclassified to net loss
Translation adjustment (170,059) 225,591

Net comprehensive loss (4,186,834) (3,326,247)
Basic and diluted loss per common share (0.03) (0.04)

Weighted average number of shares (basic and diluted) 118,199,985 99,557,654

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF CHANGES IN EQUITY


Share Capital
$

Contributed
Surplus
$

Accumulated
Other
Comprehensive
Income
$

Accumulated
Deficit
$

Total
$

As at December 31, 2014 237,657,056 25,848,429 280,043 (249,966,335) 13,819,193

Net loss and comprehensive income — — 225,591 (3,551,838) (3,326,247)
Issued, pursuant to Share Purchase
Agreement 1,925,596 — — — 1,925,596
Issued, pursuant to "At the Market"
Agreement 14,636,918 — — — 14,636,918
Share based compensation — 114,970 — — 114,970
As at March 31, 2015 254,219,570 25,963,399 505,634 (253,518,173) 27,170,430

Share Capital
$

Contributed
Surplus
$

Accumulated
Other
Comprehensive
Income
$

Accumulated
Deficit
$

Total
$

As at December 31, 2015 261,324,692 26,277,966 760,978 (263,689,330) 24,674,306

Net loss and comprehensive income — — (170,059) (4,016,775) (4,186,834)
Issued, pursuant to Share Purchase
Agreement
— — — — —
Issued, pursuant to "At the Market"
Agreement (100,544) — — — (100,544)
Share based compensation — 81,640 — — 81,640
As at March 31, 2016 261,224,148 26,359,606 590,919 (267,706,105) 20,468,568

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS

2016 2015
For the three month period ending March 31 $ $

Operating Activities
Net loss for the period (4,016,775) (3,551,838)
Amortization – property and equipment 45,942 45,130
Share based compensation 81,640 114,970
Impact of unrealized foreign exchange (gains) losses 141,295 (305,156)
Net change in non-cash working capital 724,655 949,705
Cash used in operating activities (3,023,243) (2,747,189)

Investing Activities
Acquisition of property and equipment — (11,940)
Purchase of short-term investments (27,823) (29,292)
Cash used in investing activities (27,823) (41,232)

Financing Activities
Proceeds from Share Purchase Agreement — 1,925,596
Proceeds from "At the Market" equity distribution agreement (100,544) 14,636,918
Cash provided by financing activities (100,544) 16,562,514
(Decrease) increase in cash (3,151,610) 13,774,093
Cash and cash equivalents, beginning of period 24,016,275 14,152,825
Impact of foreign exchange on cash and cash equivalents (631,257) 651,105
Cash and cash equivalents, end of period 20,233,408 28,578,023

To view the Company’s Fiscal 2016 First Quarter Consolidated Financial Statements, related Notes to the Consolidated Financial Statements, and Management’s Discussion and Analysis, please see the Company’s quarterly filings, which will be available under the Company’s profile at www.sedar.com and on Oncolytics’ website at View Source