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Myovant Sciences Initiates Phase 3 Clinical Trial of Relugolix in Men with Advanced Prostate Cancer

On March 1, 2017 Myovant Sciences (NYSE: MYOV) reported it has initiated a Phase 3 clinical trial, HERO, to evaluate the safety and efficacy of relugolix in treating men with advanced prostate cancer (Press release, Myovant Sciences, MAR 1, 2017, http://investors.myovant.com/news-releases/2017/03-01-2017-214455142 [SID1234517969]). Relugolix is an oral, once-daily, small molecule, gonadotropin-releasing hormone (GnRH) receptor antagonist that lowers testosterone by inhibiting pituitary release of luteinizing hormone and follicle-stimulating hormone.

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"Lowering testosterone levels is oftentimes essential for men with advanced prostate cancer," said Neal Shore, MD, Director of the Carolina Urologic Research Center, Myrtle Beach, SC. "The current therapies used to lower testosterone require injections and the most commonly used GnRH agonists initially raise testosterone and may cause a clinical flare of symptoms. A once-daily oral GnRH antagonist, which both decreases testosterone within days of initiation and has the potential to allow a rapid return of testosterone when discontinued, could offer an advantage to men with advanced prostate cancer, particularly those initiating androgen deprivation therapy and those considering intermittent therapy."

"Our hope is that relugolix will be able to provide the therapeutic benefits of direct testosterone suppression with a GnRH antagonist coupled with the convenience of a once-a-day pill," said Dr. Lynn Seely, MD, President & Chief Executive Officer of Myovant Sciences, Inc. "This international clinical trial designed to gain approval in the United States, Europe, and Asia will be the critical test to achieve that objective."

About the HERO Study
The HERO study is a randomized, open-label, parallel-group Phase 3 international clinical trial to evaluate the safety and efficacy of relugolix in men with androgen-sensitive advanced prostate cancer who require at least one year of continuous androgen deprivation therapy. Approximately 1,125 patients will be enrolled in this study in North and South America, Europe, and the Asia-Pacific region.

Patients enrolled in the study will be randomized 2:1 to receive oral relugolix 120 mg once daily or leuprolide acetate 3-month depot injection, respectively.

The primary efficacy outcome of the study will be the ability of relugolix to achieve and maintain serum testosterone suppression to castrate levels (≤ 50 ng/dL [1.7 nmol/L]) for 48 weeks in patients with androgen-sensitive advanced prostate cancer.

About Advanced Prostate Cancer
Prostate cancer is the second most prevalent form of cancer in men and the second leading cause of death due to cancer in men in the United States. According to the National Cancer Institute, approximately 2.9 million men are currently living with prostate cancer in the United States, and approximately 180,000 men are newly diagnosed in the United States each year. Treatment for advanced prostate cancer typically involves treatment with androgen deprivation therapy, which reduces testosterone to very low levels, commonly referred to as castration levels. GnRH agonists, such as leuprolide depot, or slow-release, injections, are the current standard of care for medical castration, causing long-term desensitization and down-regulation of the GnRH-axis. GnRH agonists may be associated with mechanism-of-action limitations, including the potentially detrimental initial rise in testosterone levels that can exacerbate clinical symptoms, which is known as clinical or hormonal flare.

About Relugolix
Relugolix is an oral, once-daily small molecule that acts as a GnRH receptor antagonist and has been evaluated in over 1,300 study participants in Phase 1 and multiple large controlled Phase 2 clinical trials. In these trials, relugolix has been shown to be generally well tolerated and to suppress testosterone levels in men and estrogen and progesterone levels in women. Common side effects are consistent with the lowering of testosterone in men and estrogen and progesterone in women.

USA PATENT GRANTS FOR IMP321 IN CANCER

On March 1, 2017 Prima BioMed Ltd (ASX: PRR; NASDAQ: PBMD) ("Prima", the "Company") reported the granting of patent number 9,579,382 entitled "Use of Recombinant LAG-3 or the Derivatives thereof for Eliciting Monocyte Immune Response" by the United States Patent Office (Filing, 6-K, Prima Biomed, MAR 1, 2017, View Source [SID1234517965]).

The method of use claims granted for this patent will provide protection for the treatment of cancer where a plurality of doses of IMP321 is used to generate a monocyte mediated response. Broad claims covering dosage and route of administration have also been granted. As part of the Company’s intellectual property strategy, further applications have been filed in the US seeking additional protection for the IMP321 product itself and for use in combination with other reagents. Patent expiry is expected to be 6 July 2029 after a patent term adjustment of 276 days.

Bristol-Myers Squibb Expands Focus on Precision Medicine with Investment and Planned Collaboration with GRAIL on Blood-Based Cancer Screening

On March 1, 2017 Bristol-Myers Squibb Company (NYSE: BMY) reported its equity investment and plans for a research collaboration with GRAIL Inc., a life sciences company whose mission is to detect cancer early when it potentially can be cured (Press release, Bristol-Myers Squibb, MAR 1, 2017, View Source [SID1234517958]). By combining the power of high intensity cancer DNA sequencing, leading edge computer science and large clinical studies into a diagnostic platform, GRAIL aims to develop highly sensitive blood tests that detect cancer in its early stages to enable earlier intervention with targeted therapies.

As an investor, Bristol-Myers Squibb will gain early access to GRAIL’s comprehensive clinical trial databases that may serve as a rich resource for understanding tumor genomics. Additionally, Bristol-Myers Squibb and GRAIL have agreed to principal terms of a research collaboration that would enable Bristol-Myers Squibb to examine its clinical data using GRAIL’s analytic tools to inform research and development decisions, guide strategies to advance point of care companion diagnostics and potentially improve selection, care and management of patients through more targeted treatments.

"A key enabler of our Immuno-Oncology strategy is to leverage precision medicine to speed the selection of the most effective combinations of therapies for patients," said Francis Cuss, MB BChir, FRCP, chief scientific officer, Bristol-Myers Squibb. "GRAIL’s future innovation potential is significant. Liquid biopsies hold the potential to support much earlier intervention and better define individual patient characteristics that may enhance treatment decisions."

Bristol-Myers Squibb has a diverse early portfolio of Immuno-Oncology mechanisms it’s evaluating across a broad range of tumors and modalities, with 10 investigational I-O molecules in the clinic and ongoing registrational trials in 14 tumor types.

Puma Biotechnology Provides Update on Review of Marketing Authorisation Application for PB272

On March 1, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that based on its recent meeting with the Rapporteur, Co-Rapporteur and review team members, as well as the European Medicines Agency (EMA), the Company plans to modify the summary of product characteristics (SmPC), sometimes referred to as the European product label, in its Marketing Authorisation Application (MAA) to restrict the intended population to patients initiating neratinib treatment within one year after completion of adjuvant trastuzumab therapy (Press release, Puma Biotechnology, MAR 1, 2017, View Source [SID1234517957]). The proposed SmPC will continue to include both hormone receptor positive and hormone receptor negative patients.

Puma recently conducted a meeting with the Rapporteur, Co-rapporteur and members of the review team as well as EMA to discuss the responses to the 120-day list of questions received in connection with the Company’s MAA for neratinib that was submitted in the summer of 2016. The initially proposed indication was for the "extended adjuvant treatment of adult patients with early-stage HER2-overexpressed/amplified breast cancer who have received prior adjuvant trastuzumab based therapy." During this meeting it was discussed that neratinib would likely be sequenced immediately after adjuvant trastuzumab and more benefit was observed in the subgroup of patients who received neratinib within 1 year from prior trastuzumab completion when compared with those patients receiving neratinib after 1 year from the completion of prior trastuzumab treatment. In addition, data from the pivotal adjuvant trastuzumab trials suggest that patients are at higher risk of recurrence closer to completion of adjuvant trastuzumab, and the risk of recurrence may decrease over time.

Based on this meeting, Puma will be revising the proposed SmPC in its MAA for neratinib to restrict the intended population to those patients initiating neratinib treatment within one year after completion of adjuvant trastuzumab therapy. The Committee for Medicinal Products for Human Use (CHMP) is continuing to review Puma’s MAA and has not yet made a final decision to recommend approval of the drug for the updated or any other indication and there is no guarantee when, if ever, the MAA will be approved. The tables below, based on the interim 5 year analysis announced in July 2016, show results for the invasive disease free survival (DFS) of the initially proposed intent to treat population and the intent to treat population (both hormone receptor positive and hormone receptor negative) in the subgroup of patients who initiated neratinib treatment within one year after completion of adjuvant trastuzumab therapy.

DFS for Initially Proposed Intent to Treat (ITT) Population
2-Year DFS 3-Year DFS 4-Year DFS 5-Year Interim DFS
Neratinib 94.3% 92.5% 91.4% 90.4%
Placebo 91.9% 90.3% 89.2% 87.9%
Absolute invasive DFS Difference 2.4% 2.2% 2.2% 2.5%

DFS for Intent to Treat (ITT) Population in Patients Initiating Neratinib Treatment Less Than One Year After the Completion of Adjuvant Trastuzumab
2-Year DFS 3-Year DFS 4-Year DFS 5-Year Interim DFS
Neratinib 93.8% 92.0% 90.8% 89.9%
Placebo 91.0% 89.5% 88.3% 86.8%
Absolute invasive DFS Difference 2.8% 2.5% 2.5% 3.1%

Puma plans to file a Current Report on Form 8-K with the Securities and Exchange Commission containing the updated Kaplan-Meier curves for the subgroup of patients randomized within one year after completion of adjuvant trastuzumab therapy for: (i) the intent to treat population; (ii) the subgroup of patients with centrally confirmed HER2 positive disease; (iii) the hormone receptor positive subgroup of patients and (iv) the hormone receptor negative subgroup of patients.

About Puma Biotechnology

PDL BioPharma Announces Fourth Quarter and Year End 2016 Financial Results

On March 1, 2017 PDL BioPharma, Inc. (PDL or the Company) (NASDAQ: PDLI) reported financial results for the fourth quarter and year ended December 31, 2016 including(Press release, PDL BioPharma, MAR 1, 2017, View Source [SID1234517953]) .

Total revenues of $66.5 million and $244.3 million for the three and twelve months ended December 31, 2016, respectively.

GAAP diluted EPS of ($0.06) and $0.39 for the three and twelve months ended December 31, 2016, respectively.
GAAP net loss attributable to PDL’s shareholders of $10.3 million and net income of $63.6 million for the three and twelve months ended December 31, 2016, respectively.

Non-GAAP net loss attributable to PDL’s shareholders of $8.6 million and net income of $108.1 million for the three and twelve months ended December 31, 2016, respectively. A full reconciliation of all components of the GAAP to non-GAAP financial results can be found in Table 4 at the end of the release.

The loss attributable to the three months ended December 31, 2016 was a result of a $51.1 million impairment charge relating to our Direct Flow Medical note receivable investment.

"2016 was a transformational year for PDL; one in which we took advantage of opportunities in the specialty pharma space as another tool to increase shareholder value," said John P. McLaughlin, president and chief executive officer of PDL. "As we look to 2017, we will focus our efforts on Noden product commercialization, along with acquiring additional specialty pharma assets, to drive value creation for PDL and our shareholders."

Recent Developments
PDL announced today that the company’s board of directors has authorized the repurchase of up to $30 million of the company’s common stock through March 2018.
As a result of ARIAD Pharmaceuticals, Inc. being acquired by Takeda Pharmaceuticals Company Limited on February 16, 2017, PDL exercised its put option with ARIAD and will be repaid an estimated $110 million, which is 1.2 times the original investment less any sums paid to date. We received $9.3 million of royalty payments through December 31, 2016. The cash repayment is expected in late March or early April of 2017.
PDL received a royalty payment for the first quarter of 2017 in the amount of $14.2 million for royalties earned on sales of Tysabri. The duration of this royalty payment is based on the sales of product manufactured prior to patent expiry, the amount of which is uncertain.
In January 2017 PDL monetized $7.0 million of certain assets of Direct Flow Medical acquired through its foreclosure.
Revenue Highlights
Total revenues of $66.5 million for the three months ended December 31, 2016 included:
Royalties from PDL’s licensees to the Queen et al. patents of $15.5 million, which consisted of royalties earned on sales of Tysabri under a license agreement;
Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $28.1 million, which consisted of the change in estimated fair value of our royalty right assets, primarily related to the Depomed, Inc., University of Michigan, ARIAD and AcelRx Pharmaceuticals, Inc.;
Interest revenue from notes receivable financings to kaléo and CareView Communications of $5.5 million; and
Product revenues of $17.5 million from sales of Tekturna and Tekturna HCT in the United States and Rasilez and Rasilez HCT in the rest of the world (collectively, the Noden Products).
Total revenues decreased by 63 percent for the three months ended December 31, 2016, when compared to the same period in 2015.
The decrease in royalties from PDL’s licensees to the Queen et al. patents is due to the expiration of the patent license agreement with Genentech, Inc.
The decrease in royalty rights – change in fair value was primarily due to the $27.8 million decrease in fair value of the University of Michigan Cerdelga royalty right asset and the decrease in fair value of the AcelRx Zalviso royalty rights asset, partially offset by an increase in the fair value of the ARIAD Pharmaceuticals, Inc. royalty right asset.
PDL received $25.3 million in net cash royalty and milestone payments from its royalty rights in the fourth quarter of 2016, compared to $34.4 million for the same period of 2015.
The decrease in interest revenues was primarily due to the early repayment of the Paradigm Spine, LLC notes receivable investment.
Product revenues were derived from sales of the Noden Products.
Total revenues decreased by 59 percent for the twelve months ended December 31, 2016, when compared to the same period in 2015.
The decrease in royalties from PDL’s licensees to the Queen et al. patents is due to the expiration of the patent license agreement with Genentech, Inc.
The decrease in royalty rights – change in fair value was primarily driven by a $36.6 million decrease in the fair value of the University of Michigan royalty rights Cerdelga asset, a $23.1 million decrease in the fair value of the Depomed royalty rights asset and a $3.0 million decrease in the fair value of the Viscogliosi Brothers, LLC royalty right asset, partially offset by a $14.8 million increase in the fair value of the ARIAD Pharmaceuticals, Inc. royalty right asset.
PDL received $72.6 million in net cash royalty payments and milestone payments from its acquired royalty rights in the twelve months ended December 31, 2016, compared to $43.4 million for the same period of 2015.
Product revenues and interest revenue variances were the same as the three months ended December 31, 2016.
Operating Expense Highlights
Operating expenses were $74.2 million for the three months ended December 31, 2016, compared to $16.5 million for the same period of 2015. The increase in operating expenses for the three months ended December 31, 2016, as compared to the same period in 2015, was primarily a result of a $51.1 million impairment charge relating to our Direct Flow Medical note receivable investment and $11.4 million in expenses related to the Noden operations.
Operating expenses were $114.9 million for the twelve months ended December 31, 2016, compared to $40.1 million for the same period of 2015. The increase in operating expenses for the twelve months ended December 31, 2016, as compared to the same period in 2015, was the result of the Direct Flow Medical impairment and $25.6 million in expenses related to the acquisition of the Noden Products and its operations.
Other Financial Highlights
PDL had cash, cash equivalents, and investments of $242.1 million at December 31, 2016, compared to $220.4 million at December 31, 2015.
Net cash provided by operating activities in the twelve months ended December 31, 2016 was $101.7 million, compared with $301.5 million in the same period in 2015.