On MARCH 1, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the fourth quarter and year ended December 31, 2016 (Press release, Puma Biotechnology, MAR 1, 2017, View Source [SID1234517983]).

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Unless otherwise stated, all comparisons are for the fourth quarter and full year 2016 compared to the fourth quarter and full year 2015.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $72.7 million, or $2.04 per share, for the fourth quarter of 2016, compared to a net loss of $61.7 million, or $1.90 per share, for the fourth quarter of 2015. Net loss applicable to common stock for the full year 2016 was $276.0 million, or $8.29 per share, compared to $239.3 million, or $7.45 per share, for the full year 2015.

Non-GAAP adjusted net loss was $43.4 million, or $1.22 per share, for the fourth quarter of 2016, compared to non-GAAP adjusted net loss of $40.0 million, or $1.23 per share, for the fourth quarter of 2015. Non-GAAP adjusted net loss for the full year 2016 was $158.8 million, or $4.77 per share, compared to $144.3 million, or $4.49 per share, for the full year 2015. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the fourth quarter of 2016 was $41.0 million. Net cash used in operating activities for the full year 2016 was $141.7 million. At December 31, 2016, Puma had cash and cash equivalents of $194.5 million and marketable securities of $35.0 million, compared to cash and cash equivalents of $31.6 million and marketable securities of $184.3 million at December 31, 2015. The Company’s balance of cash, cash equivalents and marketable securities at year-end 2016 includes the net proceeds of approximately $162 million received from the Company’s public offering in October 2016.

"We made significant progress with the neratinib clinical program during the fourth quarter," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "We presented additional results of several ongoing studies at the 2016 San Antonio Breast Cancer Symposium, including our Phase II CONTROL trial of PB272 in the extended adjuvant treatment of HER2-positive early stage breast cancer; a biomarker analysis of the NSABP FB-7 Phase II trial for the neoadjuvant treatment of HER2-positive locally advanced breast cancer; and the Phase II SUMMIT trial of PB272 for ERBB2 (HER2) mutant, HER2 non-amplified metastatic breast cancer. We also initiated a Managed Access Program for PB272 (neratinib) outside the United States, providing physicians and patients access to PB272 when there are limited or no other therapeutic options available."

Mr. Auerbach added, "During 2017, we anticipate the following key milestones with neratinib: (i) reporting additional data in the second quarter of 2017 from the Phase II CONTROL trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer using antidiarrheal prophylaxis with loperamide and other agents (budesonide, colestipol); (ii) reporting interim Phase I/II data in the second quarter of 2017 from the NSABP FB-10 trial of neratinib plus Kadcyla (T-DM1) in HER2-positive metastatic breast cancer (MBC); (iii) reporting data in the second quarter of 2017 from the Phase II SUMMIT basket trial of neratinib in patients with HER2 non-amplified solid tumors that have a HER2 mutation; (iv) reporting data from the Phase III trial in third-line HER2-positive MBC patients in the first half of 2017; (v) reporting data in the second quarter of 2017 from the TBCRC-022 Phase II trial of neratinib plus capecitabine in HER2-positive MBC patients with brain metastases; (vi) reporting Phase II data in the second quarter of 2017 from the SUMMIT basket trial of neratinib in HER2-negative breast cancer patients with HER2 mutations; (vii) reporting final 5-year disease free survival (DFS) data during the second half of 2017 from the ExteNET Phase III trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer; and (viii) announcing regulatory decisions in the United States and European Union on neratinib for the extended adjuvant treatment of patients with HER2-positive early stage breast cancer in the third quarter of 2017."

Operating Expenses

Operating expenses were $72.9 million for the fourth quarter of 2016, compared to $62.1 million for the fourth quarter of 2015. Operating expenses for the full year 2016 were $276.6 million compared to $240.3 million for the full year 2015.

General and Administrative Expenses:

General and administrative expenses were $16.5 million for the fourth quarter of 2016, compared to $9.6 million for the fourth quarter of 2015. General and administrative expenses for the full year 2016 were $53.8 million compared to $31.8 million for the full year 2015. The increase of approximately $22.0 million during the full year 2016 compared to the same period in 2015 resulted primarily from increases of approximately $9.4 million in stock-based compensation, $2.8 million in payroll and related costs, $7.1 million in professional fees and expenses and $2.2 million in facility and equipment costs. These increases reflect higher legal and compliance expenses, as well as overall corporate growth.

Research and Development Expenses:

Research and development expenses were $56.4 million for the fourth quarter of 2016, compared to $52.5 million for the fourth quarter of 2015. Research and development expenses for the full year 2016 were $222.8 million, compared to $208.5 million for the full year 2015. The increase of approximately $14.3 million during the full year 2016 compared to the same period in 2015 resulted primarily from increases of approximately $12.8 million in stock-based compensation, $6.2 million for internal clinical development, regulatory affairs and quality assurance and internal chemical manufacturing expenses and $2.0 million in consultants and contractors related expenses, offset by a $6.8 million decrease in clinical trial expenses.

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.

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]).

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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.

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.

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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.

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.

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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.

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