On August 9, 2016 CANCER RESEARCH TECHNOLOGY (CRT) – the commercial arm of Cancer Research UK – the University of Birmingham and its commercial arm, Alta Innovations (Alta), reported an agreement to work together to develop and commercialise the university’s cancer research (Press release, Cancer Research Technology, SEP 9, 2016, View Source [SID1234523183]).

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The two-year agreement draws on the strengths of each organisation to accelerate commercialisation of the University’s world-leading research in cancer into diagnostics, treatments and technologies that could one day benefit patients.

Alta will use its expertise in working closely with world-leading scientists at the University to identify the most promising cancer research opportunities and CRT will use its expertise in commercialising cancer research and its vast network across the pharmaceutical and biotechnology industry to identify and translate promising research at the university.

CRT will work together with Alta and the University of Birmingham’s academics to identify promising cancer opportunities, including the significant clinical expertise available and the university’s leading research in areas such as immunology and genomics.

Financial benefits that come from the work will be shared between CRT and the University of Birmingham. All financial returns for CRT are invested into Cancer Research UK’s life-saving research and all returns to the University are for the benefit of the educational charity.

Professor David Adams, head of the college of medical and dental sciences at the University of Birmingham, said: "Bringing Alta and CRT together in this way means we are giving our world-class cancer researchers access to a team of people with the right skills to give their ideas the fastest route to having an impact on patients’ lives."

Dr James Wilkie, CEO of Alta Innovations, said: "This agreement is built on a long standing working relationship. Our job is to ensure that the world-leading cancer research done here gets translated into better patient outcomes as fast as possible.

"Combining the skills that Alta and CRT have built up over many years will allow our researchers to accelerate the development of their world-leading ideas and patients will benefit from these being brought to market more quickly."

Dr Phil L’Huillier, CRT’s director of business management, said: "We’ve already seen some great successes from our work with Alta Innovations, such as the formation of Revitope Oncology in 2014, an exciting new immunotherapy company. So we’re delighted to be working with the University of Birmingham and Alta Innovations to help develop and commercialise pioneering research that could lead to new treatments to patients faster.

"Developing relationships of this kind draws on the strengths and expertise of both organisations. We hope to develop more relationships like this with other top UK universities in the future to accelerate research and bring new hope to patients."

On August 9, 2016 Anthera Pharmaceuticals, Inc. (Nasdaq:ANTH) reported a business update and reported financial results for the second quarter ended June 30, 2016 (Filing, Q2, Anthera, 2016, AUG 9, 2016, View Source [SID:1234514412]).

Recent Developments and Business Highlights:

Sollpura (liprotamase) – Exocrine Pancreatic Insufficiency ("EPI")

o Phase 3 SOLUTION Clinical Study Enrollment Target of 126 Patients Met

We met the enrollment target in our Phase 3 SOLUTION clinical study evaluating the efficacy and safety of the capsule formulation of Sollpura to treat exocrine pancreatic insufficiency in patients with cystic fibrosis in early August. We expect to report topline efficacy data in the fourth quarter of 2016. For more information on the SOLUTION clinical study, please visit View Source study/.

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o Initiated SIMPLICITY Clinical Study with an Enrollment Target of 46 Patients

We began dosing patients in the SIMPLICITY clinical study which is evaluating the efficacy and safety of Sollpura supplied as a powder for oral solution. In this study, Sollpura is packaged in a convenient, easy-to-administer packet. The soluble dose form of all three digestive enzymes is mixed with water or apple juice. After an initial cohort of patients older than seven is treated for one week, this study will allow for administration of Sollpura powder for oral solution to pediatric patients ranging in age from 28 days to seven years. For more information on the study, please visit View Source

o Manufacturing Progressing to Support SIMPLICITY Study and Commercial Readiness

We successfully manufactured and released two sachet dosage strengths with a newly developed dry powder formulation for oral solution, enabling the initiation of the SIMPLICITY clinical study. We made progress with manufacturing technical transfer of all three enzymes to our contract manufacturers site. We completed demonstration batch manufacturing at commercial launch scale for lipase. Additionally, proof-of-concept was demonstrated for a high dose Lipase unit capsule – a further step towards reducing capsule burden in patients with EPI.

Blisibimod – Systemic Lupus Erythematosus ("SLE")

o Topline Data from Phase 3 CHABLIS-SC1 Clinical Study

We continue to collect and prepare final data from the Phase 3 CHABLIS-SC1 clinical study as the final patients continue treatment in the study. The last patient in the study received their final study dose on July 28th. As described in the protocol, patients are followed for eight weeks after their last dose at which time the final safety data is collected. Due to timing of this final visit, the company expects topline efficacy and safety data will be available prior to the annual American College of Rheumatology Annual Meeting in November. Topline data from the CHABLIS-SC1 will include the primary endpoint evaluation, a six-point reduction in the Systemic Lupus Erythematosus Responder Index (SRI-6) as well as safety and tolerability data from the study. For more information on the CHABLIS-SC1 study, please visit View Source

o Phase 3 CHABLIS 7.5 Clinical Study Initiated

CHABLIS 7.5, Anthera’s second Phase 3 clinical study successfully enrolled its first patient. This study will evaluate the efficacy and safety of blisibimod in patients who, despite corticosteroid use, continue to have clinically-active lupus (SLE) and the presence of anti-double-stranded DNA and low complement which are of known serological markers of lupus. For more information about the CHABLIS 7.5 study, visit View Source

Blisibimod – IgA Nephropathy

o Positive Trends Reported on Phase 2 BRIGHT-SC Clinical Study

In June 2016, interim data from the BRIGHT-SC study, which enrolled 57 patients, demonstrated a positive trend in lower proteinuria in blisibimod versus placebo treated patients. While the numerical reduction in proteinuria in blisibimod versus placebo treated patients at week 24 in the BRIGHT-SC study did not meet the predefined primary endpoint of complete or partial response, longer-term data from the study demonstrated an increasingly large separation in proteinuria favoring the blisibimod treated arm compared to placebo. Additionally, secondary biomarker data from the study, including changes in total B cell counts and changes in immunoglobulins IgA, IgG, and IgM, were highly consistent with previous studies with blisibimod and demonstrated marked reduction after 8 weeks on study. As a result of the increasing proteinuria effect after 24 weeks of dosing, and the demonstration of blisibimod’s effect on immunological markers relevant to IgA nephropathy including reductions of B cells, and immunoglobulins including IgA, IgG and IgM, we elected to continue the study until the last subject enrolled completes 48 weeks of evaluation. For more information about the BRIGHT-SC study, visit View Source

Summary of Financial Results

· Cash Position. We ended the second quarter of 2016 with cash and cash equivalents totaling $28.5 million, compared to $47.0 million as of December 31, 2015. The decrease in cash was mainly attributable to research, development and operating expenses during the six months ended June 30, 2016.
· R&D Expense. Research and development expenses for the three and six months ended June 30, 2016 totaled $12.0 million and $21.6 million, respectively, compared to $8.5 million and $14.5 million for the corresponding periods in 2015. The increase is mainly attributable to higher clinical development expenses resulting from the acceleration of patient enrollment in the SOLUTION clinical study, the initiation of the SIMPLICITY clinical study, manufacturing scale-up costs associated with Sollpura, and the initiation of the CHABLIS-7.5 clinical study in severe lupus patients.
· G&A Expense. General and administrative expenses for the three and six months ended June 30, 2016 totaled $2.6 million and $4.8 million, respectively, compared to $1.7 million and $3.6 million for the corresponding periods in 2015. The increase is primarily due to higher non-cash stock-based compensation expense of $0.6 million and $0.9 million, respectively, recognized during the three and six months ended June 30, 2016.
· Research Award. A research award, granted to us in March 2015 by the Cystic Fibrosis Foundation Therapeutics, Inc. and recorded as an offset to operating expense, totaled $261,000 for the three and six months ended June 30, 2016. The amount of research award recognized represents the value prescribed to the milestones that we achieved under the award agreement during the current period. There were no research award amounts recorded during the comparative period in 2015.
· Net Loss. Net loss for the three and six months ended June 30, 2016 was $14.3 million, or $0.35 per basic and diluted share and $26.1 million, or $0.64 per basic and diluted share, respectively, compared to $8.9 million, or $0.25 per basic and diluted share and $16.6 million, or $0.52 per basic and diluted share for the corresponding periods in 2015.

On August 9, 2016 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company"), a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology, endocrinology and women’s health, reported financial and operating results for the second quarter ended June 30, 2016.

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Commenting on recent key developments, David A. Dodd, President and Chief Executive Officer of the Company, stated, "After the end of Q2, we concluded two important out-license agreements for Zoptrex, confirming the market’s interest in our lead oncology compound, Zoptrex (zoptarelin doxorubicin). Zoptrex is a novel synthetic peptide carrier linked to doxorubicin as a New Chemical Entity (NCE). Based on recent information regarding the survival of patients in the Phase 3 clinical trial of Zoptrex, we expect to complete the trial by year-end. If the results of the trial warrant doing so, we intend to file a new drug application for Zoptrex in the first half of 2017."

Mr. Dodd continued his commentary with an update on the development of Macrilen (macimorelin), "We are pleased to announce that we should complete enrollment in our confirmatory Phase 3 study of Macrilen for the evaluation of adult growth hormone deficiency by the end of August. As a result, we are very confident that the study of Macrilen will be concluded in 2016. If our expectations for completion of the confirmatory Phase 3 study are realized and if the top-line results indicate that the product attained the primary endpoint of the Phase 3 study, we expect to file an NDA for Macrilen during the first half of 2017. Since the regulatory review period for the Macrilen confirmatory study is six months, we could begin commercializing the product late in 2017."

Second Quarter 2016 Financial Highlights

R&D costs were $3.7 million for the three-month period ended June 30, 2016 and $7.4 million for the six-month period then ended, compared to $4.5 million and $8.9 million, respectively, for the three-month and six-month periods ended June 30, 2015. The decrease for the three-month and six-month periods ended June 30, 2016, as compared to the same period in 2015, is mainly attributable to lower comparative third-party costs. Third-party costs attributable to Zoptrex decreased considerably during the three-month and six-month periods ended June 30, 2016, as compared to the same periods in 2015, mainly due to the fact that dosing of patients in the ZoptEC trial was completed in February 2016. This is consistent with our expectations as we are approaching the end of the clinical trials. In addition, during 2015, we started the confirmatory Phase 3 clinical trial of Macrilen, which explains the increase in costs for this product candidate. The overall decrease in R&D costs is also explained by lower employee compensation and benefits costs, lower facilities rent and maintenance as well as lower other costs. A substantial portion of this decrease is due to the realization of cost savings in connection with our effort to streamline our R&D activities and to increase our commercial operations and flexibility by reducing our R&D staff, which was started in 2014, and for which a provision was recorded in the third quarter of 2014.

G&A expenses were $1.9 million for the three-month period ended June 30, 2016, and $3.8 million for the six-month period then ended, compared to $2.0 million and $5.4 million, respectively, for the three-month and six-month periods ended June 30, 2015. The comparative decrease for the six-month period is mainly attributable to the recording, in the prior year quarter, of certain transaction costs allocated to warrants in connection with the completion of the March 2015 Offering.

Selling expenses were $1.7 million for the three-month period ended June 30, 2016 and $3.4 million for the six-month period then ended, essentially unchanged as compared to the three-month and six-month periods ended June 30, 2015. The selling expenses for the three- and six-month periods ended June 30, 2016 and 2015 represent the costs of our contracted sales force related to the co-promotion activities as well as our internal sales management team. Those activities were launched during the fourth quarter of 2014.

Net loss for the three-month and six-month periods ended June 30, 2016 were $7.0 million and $10.7 million, respectively, or $0.71 and $1.08, respectively, both per basic and diluted share. During the same three-month and six-month periods in 2015, our net loss was $15.1 million and $24.8 million, respectively, or $13.65 and $27.22, respectively, per basic and diluted share for the same period in 2015. The decrease in net loss for the three-month and six- month periods ended June 30, 2016, as compared to the same periods in 2015, is due largely to lower operating expenses and higher comparative net finance income.

Cash and cash equivalents were approximately $26.2 million as at June 30, 2016, compared to approximately $33.0 million as at March 31, 2016.

On August 9, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN) reported financial results for its fiscal fourth-quarter 2016, provided an update on recent business highlights and provided fiscal first-quarter 2017 and fiscal year 2017 financial guidance (Press release, Myriad Genetics, AUG 9, 2016, View Source [SID:1234514554]).

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"Fourth-quarter performance met our expectations, and we continue to make substantial progress on advancing our product portfolio and growing Myriad into a larger, more diversified personalized medicine company," said Mark C. Capone, president and CEO of Myriad. "Myriad is on track to deliver on its five year strategic goals. We continue to be the worldwide leader in hereditary cancer testing, and our portfolio diversification efforts are accelerating with substantive volume growth and reimbursement for new commercial products, pivotal validations for our pipeline products, the acquisitions of Assurex Health and Sividon, and significant international expansion. Given the multibillion dollar market opportunity for our portfolio, we remain confident that we can deliver better health outcomes for patients and significant long-term value for shareholders."

Financial Highlights

Below are tables summarizing the financial results and revenue by product class for our fiscal fourth-quarter 2016 and full fiscal-year 2016:

Revenue
Fiscal Fourth-Quarter Fiscal Year
($ in millions) 2016 2015 % Change 2016 2015 % Change
Molecular diagnostic testing revenue

Hereditary cancer testing revenue $ 152.8 $ 163.8 (7 %) $ 632.3 $ 638.3 (1 %)

Vectra DA testing revenue 12.7 11.8 8 % 47.8 43.7 9 %

Prolaris testing revenue 3.5 0.7 400 % 11.3 2.1 438 %

Other testing revenue 4.8 2.5 92 % 14.3 11.4 25 %

Total molecular diagnostic testing revenue 173.8 178.8 (3 %) 705.7 695.5 2 %

Pharmaceutical and clinical service revenue 12.7 11.1 14 % 48.1 27.6 74 %

Total Revenue $ 186.5 $ 189.9 (2 %) $ 753.8 $ 723.1 4 %

Income Statement
Fiscal Fourth-Quarter Fiscal Year
($ in millions) 2016 2015 % Change 2016 2015 % Change
Total Revenue $ 186.5 $ 189.9 (2 %) $ 753.8 $ 723.1 4 %

Gross Profit 146.5 152.4 (4 %) 596.5 575.7 4 %
Gross Margin 78.6 % 80.3 % 79.1 % 79.6 %

Operating Expenses 110.8 116.2 (5 %) 429.7 441.5 (3 %)

Operating Income 35.7 36.2 (1 %) 166.8 134.2 24 %
Operating Margin 19.1 % 19.1 % 22.1 % 18.6 %

Adjusted Operating Income 39.0 48.2 (19 %) 179.5 167.3 7 %
Adjusted Operating Margin 20.9 % 25.4 % 23.8 % 23.1 %

Net Income 23.4 18.7 25 % 125.3 80.2 56 %

Diluted EPS 0.32 0.26 23 % 1.71 1.08 58 %

Adjusted EPS $ 0.36 $ 0.41 (12 %) $ 1.63 $ 1.45 12 %

Business Highlights

myRisk Hereditary Cancer
Myriad signed a new agreement with Blue Shield of California, retaining Myriad’s previous network status. Myriad ended the quarter with 65 percent of revenue under long-term contracts for its hereditary cancer business.
A new study in the New England Journal of Medicine demonstrated that in patients with advanced prostate cancer, the rate of deleterious mutations in myRisk genes was approximately 12 percent. This is consistent with other cancers that have genetic testing guidelines. Every year in the United States approximately 25,000 patients are diagnosed with advanced prostate cancer.
Myriad made the first content additions to myRisk adding three additional genes including GREMM1, POLE and POLD-1. These genes were recently added to the NCCN guidelines on genetic testing based upon their role in hereditary colon cancer.
Myriad announced the ability to customize the myRisk panel for genetics experts who are interested in ordering a subset of the myRisk genes on the full panel.

Vectra DA
Vectra DA volumes were up five percent year-over-year in the fiscal fourth-quarter with approximately 41,300 tests performed.
Myriad signed three additional private payer contracts for Vectra DA that collectively cover approximately one million lives in the United States.
At the European League Against Rheumatism (EULAR) annual meeting, Myriad presented new data showing the ability of Vectra DA to predict sustained clinical remission after discontinuation of Humira. In patients with a low Vectra DA score, 57 percent had sustained clinical remission following discontinuation of Humira. In patients with a high Vectra DA score, 60 percent experienced flare within one year of discontinuation of Humira.
In a second study presented at EULAR of 180 treatment naïve patients, the Vectra DA score was a statistically significant predictor of clinical remission with 12-month remission rates meaningfully higher in patients with a greater reduction in Vectra DA score versus those with a smaller reduction.

Prolaris
Prolaris volume increased 91 percent year-over-year and 11 percent sequentially with approximately 4,750 tests ordered.
At the American Urological Association annual meeting, Myriad presented the first study validating Prolaris exclusively in a low-risk only patient population. In 440 patients with a Gleason score of six or less, patients with a high Prolaris score had three times the rate of prostate specific mortality and eight times the rate of biochemical recurrence relative to patients with a low Prolaris score.
Myriad signed three additional private payer contracts for Prolaris that collectively cover approximately one million lives in the United States.

Companion Diagnostics
Myriad presented data from the first prospective validation of myChoice HRD in concert with TESARO’s NOVA study. In the study, which evaluated platinum-sensitive ovarian cancer patients, myChoice HRD positive patients receiving niraparib demonstrated a 9.1 month increase in progression free survival relative to patients receiving placebo.

Sividon Diagnostics Acquisition
Myriad completed the acquisition of Sividon Diagnostics during the fiscal fourth quarter and gained United States and Chinese distribution rights to EndoPredict, a best-in-class breast cancer prognostic test. Myriad announced plans to launch EndoPredict in the United States in the second half of fiscal year 2017.
A large head-to-head study comparing EndoPredict with Oncotype Dx was recently published in the Journal of the National Cancer Institute. The study, which evaluated 928 women from the TransATAC study, showed that EndoPredict "markedly outperformed" Oncotype Dx with a prognostic power that was more than four times greater. Also, EndoPredict low-risk patients had a 10-year rate of distant metastases of 5.8 percent versus low risk Oncotype Dx patients at 10.1 percent.

Assurex Health Acquisition
In August, Myriad signed a definitive agreement to acquire Assurex Health, a personalized medicine company providing treatment decision support to healthcare providers for their patients with mental health disorders. Assurex’s lead product, GeneSight Psychotropic, evaluates 12 genes known to play a significant role in psychotropic drug response and is used in patient treatment selection. Assurex performed over 150,000 GeneSight tests in Myriad’s fiscal year 2016 and generated more than $60 million in total revenue. The acquisition is subject to the satisfaction of customary closing conditions.

International
International revenues were up 93 percent year-over-year in the fourth quarter and accounted for approximately six percent of total product revenue in the quarter.
Myriad recently signed an agreement with BMI Healthcare in the United Kingdom covering Myriad’s complete testing portfolio including EndoPredict, Prolaris, Tumor BRACAnalysis CDx and hereditary cancer testing. BMI Healthcare is the largest private hospital group comprised of 59 hospitals and clinics across the UK, servicing 1,500,000 outpatient visits per year.

Share Repurchase
During the quarter, the Company repurchased approximately 1.6 million shares, or $55 million, of common stock under our share repurchase program and ended the quarter with approximately $195 million remaining on our current share repurchase authorization.
Fiscal Year 2017 and Fiscal First-Quarter 2017 Financial Guidance
Below is a table summarizing Myriad’s fiscal year 2017 and fiscal first-quarter 2017 financial guidance:

Revenue Adjusted
Earnings Per
Share GAAP Diluted
Earnings Per
Share
Fiscal Year 2017 $740-$760 million $1.00-$1.10 $0.47 – $0.57

Fiscal First-Quarter 2017 $168-$170 million $0.25-$0.27 $0.14 – $0.16

Myriad’s fiscal year 2017 financial guidance includes the impact of the Assurex Health acquisition which Myriad expects to close at the end of the fiscal first-quarter 2017.

These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The Company will provide further details on its business outlook during its conference call today to discuss the fiscal fourth-quarter financial results and fiscal year 2017 and fiscal first-quarter 2017 financial guidance.

On August 9, 2016 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical and clinical-stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported it has agreed to extend through August of 2017 and expand its sponsored research agreement with MD Anderson Cancer Center (MD Anderson), the world’s largest cancer research facility (Press release, Moleculin, AUG 9, 2016, View Source [SID1234519562]).

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Moleculin’s Chairman and CEO, Walter Klemp, commented, "The research we have sponsored to date at MD Anderson has generated some very promising new technologies, including WP1066, a novel small molecule that is an effective stimulator of patients’ natural immune response and inducer of tumor cell death in the in vivo models of even the most difficult to treat cancers." He added, "We are now extending and expanding this agreement in light of new indications that we may be able to improve upon the WP1066 technology significantly."

Dr. Waldemar Priebe, Professor of Medicinal Chemistry at MD Anderson and Scientific Advisor to Moleculin, a shareholder and inventor of licensed technology, explained, "We are very excited about the potential for orally administered WP1066 in clinic, and we are now actively investigating expansion of clinical uses of Moleculin’s licensed therapeutics and researching their methods of administration while preserving drugability of this and similar compounds. For example, the injectable/iv administered versions of WP1066 would greatly expand its clinical applications and commercial potential. The continued research sponsored by Moleculin could make this possible."

Mr. Klemp concluded, "Moleculin’s continuing research sponsorship also facilitates access to grant funding from both public and private sources, and we are pleased that the expansion of this critical research collaboration will provide access to cutting edge research capability and potentially create more patentable discoveries."