On October 26, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported the acceptance by the China National Medical Products Administration (NMPA) of a new drug application (NDA) for zanubrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) (Press release, BeiGene, OCT 24, 2018, View Source;p=RssLanding&cat=news&id=2373150 [SID1234530153]). Zanubrutinib was discovered in BeiGene’s research facilities in Beijing, China, and is being developed globally by BeiGene as a monotherapy and in combination with other therapies to treat various hematologic malignancies. In August, the NMPA accepted BeiGene’s first NDA for zanubrutinib for the treatment of patients with R/R mantle cell lymphoma (MCL).

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"Our team has made three NDA submissions in China this year, including two for zanubrutinib and one for tislelizumab, our investigational anti-PD-1 antibody. We are hopeful that these submissions, if approved, could further transform BeiGene as well as bring important new treatment options to cancer patients," commented John Oyler, co-founder, CEO and Chairman of BeiGene.

"We are delighted that the submission for zanubrutinib in patients with relapsed/refractory CLL/SLL was accepted by the NMPA in China, and we are excited to announce the top-line pivotal data for zanubrutinib in these patients, which demonstrated a high overall response rate of 80 percent despite a relatively short follow-up. These results in China are also consistent with the data from our global studies," said Dr. Xiaobin Wu, General Manager of China and President of BeiGene, Ltd.

The NDA is supported by an extensive clinical, non-clinical and chemistry, manufacturing and control (CMC) data package, including the results from a 91-patient single-arm pivotal Phase 2 study in Chinese patients with R/R CLL/SLL treated with zanubrutinib, dosed at 160 mg orally twice daily. An independent review of response data from this study, with a data cut-off of June 15, 2018 and a median follow-up of 9.1 months, showed an overall response rate (ORR) of 80 percent, inclusive of complete response (2%), partial response (39%), and partial response with lymphocytosis (40%). The median duration of response has not been reached, as a majority of the responders remain in a response. The safety profile was consistent with previously reported clinical data for zanubrutinib. Updated data with additional follow-up of the patients in the study will be submitted to the NMPA as an additional document of the NDA and are planned to be presented at an upcoming medical conference.

Zanubrutinib was recently granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Waldenström macroglobulinemia (WM). BeiGene plans to submit an NDA to the FDA for zanubrutinib as a potential treatment for patients with WM in the first half of 2019 based on results from a global Phase 1 study.

Zanubrutinib is being evaluated in a broad global registration program, including a fully enrolled Phase 3 clinical trial in patients with WM, comparing zanubrutinib with ibrutinib, the currently approved BTK inhibitor for WM. Zanubrutinib is also being studied in comparison to bendamustine/ rituximab in a Phase 3 clinical trial in patients with previously untreated CLL/SLL, as well as in a pivotal randomized Phase 2 trial in combination with GAZYVA (obinutuzumab) in patients with R/R follicular lymphoma. In China, besides the pivotal Phase 2 trials in R/R MCL and R/R CLL/SLL, BeiGene has completed enrollment in a pivotal Phase 2 clinical trial in patients with WM. BeiGene also plans to initiate a Phase 3 trial comparing zanubrutinib to ibrutinib in patients with R/R CLL/ SLL.

About Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma
Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are forms of non-Hodgkin lymphoma, a type of blood cancer, that arise from B lymphocytes. CLL and SLL are essentially the same disease, with the only difference being the location where the cancer primarily occurs.i When most of the cancer cells are located in the bloodstream and the bone marrow, the disease is referred to as CLL, although the lymph nodes and spleen are often involved. When the cancer cells are located mostly in the lymph nodes, the disease is called SLL.ii

About Zanubrutinib
Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of Bruton’s tyrosine kinase (BTK) that is currently being evaluated in a broad pivotal clinical program globally and in China as a monotherapy and in combination with other therapies to treat various B cell malignancies.

On October 24, 2018 The Phase III ARAMIS (Androgen Receptor inhibiting Agent for MetastatIc-free Survival) trial that investigated darolutamide in men with non-metastatic castration-resistant prostate cancer (nmCRPC), met its primary endpoint (Press release, Bayer, OCT 24, 2018, View Source [SID1234530182]). Darolutamide significantly extended metastasis-free survival (MFS) compared to placebo. The safety profile and the tolerability of darolutamide observed in the ARAMIS trial were consistent with previously published data on darolutamide. ARAMIS is a randomized, multi-center, double-blind, placebo-controlled trial in patients with nmCRPC. Darolutamide is an investigational, oral androgen receptor (AR) antagonist developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

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"Despite recent advances in nmCRPC, there remains a high unmet need for additional treatment options that delay the time to metastases," said Scott Fields, MD, senior vice president and head of Oncology Development at Bayer’s Pharmaceutical Division. "We are encouraged by the results of the ARAMIS trial and look forward to presenting the full data at an upcoming scientific meeting."

Bayer plans to discuss the data from the ARAMIS trial with health authorities regarding the submission of a new drug application. Bayer has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for darolutamide in men with nmCRPC.

About the ARAMIS Trial
The ARAMIS trial is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial evaluating the safety and efficacy of oral darolutamide in patients with nmCRPC who are currently being treated with androgen deprivation therapy (ADT) and are at risk for developing metastatic disease. More than 1,500 patients were randomized in a 2:1 ratio to receive 600 mg of darolutamide twice a day or matching placebo.

The primary endpoint of this trial is metastasis-free survival (MFS), defined as time between randomization and evidence of metastasis or death from any cause. The secondary objectives of this trial are overall survival (OS), time to first symptomatic skeletal event (SSE), time to initiation of first cytotoxic chemotherapy, time to pain progression and characterization of the safety and tolerability of darolutamide.

About Castration-Resistant Prostate Cancer (CRPC)
Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2018, an estimated 1.2 million men will be diagnosed with prostate cancer, and about 358,000 will die from the disease worldwide. Prostate cancer is the fifth leading cause of death from cancer in men.

Prostate cancer results from the abnormal proliferation of cells within the prostate gland, which is part of a man’s reproductive system. It mainly affects men over the age of 50, and the risk increases with age.

Treatment options range from surgery to radiation treatment to therapy using hormone-receptor antagonists, i.e. substances that stop the formation of testosterone or prevent its effect at the target location. However, in nearly all cases, the cancer will become resistant to conventional hormone therapy1.

CRPC is an advanced form of the disease where the cancer keeps progressing even when the amount of testosterone is reduced to very low levels in the body. The field of treatment options for castration-resistant patients is evolving rapidly, but until recently, there have been no approved treatment options for CRPC patients who have rising Prostate-Specific Antigen (PSA) levels during ADT and no detectable metastases. In men with progressive nmCRPC, a short PSA doubling time has been consistently associated with reduced time to first metastasis and death2.

About Darolutamide
Darolutamide is an investigational, non-steroidal androgen receptor antagonist with a chemical structure that binds to the receptor and exhibits antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells. Darolutamide has also completed Phase 1/2 studies in patients with mCRPC. A Phase 3 study in metastatic hormone-sensitive prostate cancer (ARASENS) is ongoing. Information about these trials can be found at www.clinicaltrials.gov.

Darolutamide is not approved by the U.S. FDA, the European Medicines Agency or any other health authority.

On October 24, 2018 Melinta Therapeutics, Inc., (NASDAQ: MLNT), a commercial-stage company discovering, developing and commercializing novel antibiotics to treat serious bacterial infections, reported that its board of directors has appointed John H. Johnson as interim chief executive officer (CEO), effective immediately (Press release, Cempra, OCT 24, 2018, View Source [SID1234530248]). Mr. Johnson, a director of Melinta, succeeds Dan Wechsler, who is stepping down from his role as president, CEO and director to pursue other opportunities. The Board and Mr. Wechsler mutually agreed that now is the right time to transition leadership of the Company.

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"John is an accomplished biopharmaceutical industry leader with more than 20 years of direct expertise in the antibiotics space, and we are pleased that he is leading Melinta during this pivotal time. Under John’s leadership, we are confident that sales of commercial products, Baxdela (delafloxacin), Vabomere (meropenem and vaborbactam), Orbactiv (oritavancin), and Minocin (minocycline) for Injection, will continue to accelerate, and that he will focus on strengthening the financial position of Melinta by optimizing the integrations of the infectious disease business of The Medicines Company and Cempra," said Kevin Ferro, chairman of Melinta Therapeutics.

"I am pleased to serve as interim CEO of Melinta and will continue to work closely with the board of directors, executive management and the broader team to further advance the Company’s mission to provide life-saving therapeutic solutions that address the evolving global threat of bacterial infections and antibiotic resistance. This is an exciting time for Melinta and I look forward to contributing to the continued growth and future success of the Company by delivering anti-infective solutions to patients," said John H. Johnson, interim chief executive officer and director of Melinta Therapeutics.

Mr. Ferro added, "On behalf of the board of directors, we would like to thank Dan for his contributions to the Company and we wish him well in his future endeavors."

John H. Johnson has more than 30 years of biopharmaceutical industry, executive leadership and commercial experience at leading global organizations, including Johnson & Johnson, Eli Lilly & Company, ImClone and Pfizer, Inc. In addition to Melinta, Mr. Johnson currently serves on the boards of Aveo Oncology, Histogenics Corporation, Portola Pharmaceuticals, Inc., and is chairman of Strongbridge Biopharma plc. Mr. Johnson previously served as a director at Cempra and Sucampo. He also previously served as president and chief executive officer of Dendreon Corporation from February 2012, became chairman in July 2013, and served as chairman until June 2014 and president and chief executive officer until August 2014. Prior to this role, Mr. Johnson served as president of Eli Lilly & Company’s Global Oncology Unit following the company’s 2008 acquisition of ImClone Systems Incorporated, where he served as chief executive officer and on ImClone’s board. Prior to ImClone, Mr. Johnson served as the company group chairman of biopharmaceuticals within Johnson & Johnson, where he was responsible for biotechnology, immunology and oncology commercial business units. Prior to that role, he held several executive

positions at Johnson & Johnson, Parkstone Medical Information Systems, Inc., Ortho-McNeil Pharmaceutical Corporation and Pfizer Inc. While at Ortho-McNeil, Mr. Johnson was responsible for the company’s anti-infectives portfolio. During his career, Mr. Johnson also served as a member of the board of directors of Pharmaceutical Research and Manufacturers of America (PhRMA), the Health Section Governing Board of Biotechnology Industry Organizations (BIO), and BioNJ.

On October 24, 2018 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on using the body’s immune system to fight cancer, reported that a Pre-Submission (Pre-Sub) meeting with the US FDA has been scheduled on Monday, December 17, 2018 (Press release, Anixa Biosciences, OCT 24, 2018, View Source [SID1234530499]). The meeting is to discuss the proposed preclinical and clinical performance testing plan required to support a pre-market application, and to determine the appropriate regulatory pathway for the Cchek prostate cancer confirmation test.

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The purpose of a Pre-Sub meeting is to permit a dialogue between the FDA and a diagnostic developer to facilitate a better understanding of the regulatory path enabling commercialization. With the Pre-Sub request, Anixa presented to the FDA a clinical trial plan and a group of questions on which it would like comment and clarification. After the meeting, and receipt of official minutes from the meeting, Anixa expects to have a better understanding of the regulatory pathway and anticipated timeline for commercialization.

"We are pleased that we were able to get our meeting with the FDA scheduled before the end of the year. We are looking forward to a productive discussion with the FDA that will help clarify the commercialization pathway for Cchek as a prostate cancer diagnostic test. I want to make clear that this new dialogue with the FDA is independent of and in addition to the dialogue we have already started regarding our CAR-T therapy. Both programs and corresponding interactions with the FDA will move forward independently at their own pace," stated Dr. Amit Kumar, President and CEO of Anixa Biosciences. "For Anixa Biosciences, this is an exciting time as we now have two technologies and products, regarding which we are working with regulators."

On October 23, 2018 Alkermes plc (Nasdaq: ALKS) reported financial results for the third quarter of 2018 (Press release, Alkermes, OCT 23, 2018, View Source [SID1234530060]).

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"Our solid results in the quarter were in-line with expectations, driven by the growth of our proprietary commercial products, the continued strength of our royalty and manufacturing business, and the important investments we are making in our late-stage pipeline and commercial organization," commented James Frates, Chief Financial Officer of Alkermes. "Our diverse business is financially strong and we are well positioned to execute on our strategy to drive value and long-term growth. Based on our outlook for the remainder of the year, today we are raising our financial expectations for 2018, primarily driven by upside from AMPYRA revenues."

Quarter Ended Sept. 30, 2018 Financial Highlights

Total revenues for the quarter were $248.7 million. This compared to $217.4 million for the same period in the prior year, representing an increase of 14%. Proprietary product net sales for VIVITROL and ARISTADA were $116.0 million for the quarter, reflecting a 24% increase compared to the same period in the prior year.
Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $34.4 million for the quarter, or a basic and diluted GAAP net loss per share of $0.22. This compared to GAAP net loss of $36.3 million, or a basic and diluted GAAP net loss per share of $0.24, for the same period in the prior year.
Non-GAAP net income was $11.6 million for the quarter, or a non-GAAP basic and diluted earnings per share of $0.07. This compared to non-GAAP net income of $4.2 million, or a non-GAAP basic and diluted earnings per share of $0.03, for the same period in the prior year.
"The third quarter was highlighted by the launch of ARISTADA INITIO1, the newest addition to the ARISTADA product family. This new offering is resonating with healthcare providers and the early trends are encouraging. ARISTADA INITIO further differentiates ARISTADA in the market and provides an opportunity to address unmet patient need," stated Jim Robinson, President and Chief Operating Officer of Alkermes. "We are also making important strides with VIVITROL as the product continues to grow and as policymakers continue to activate in their response to the opioid crisis. We look forward to providing updates on our progress."

Quarter Ended Sept. 30, 2018 Financial Results

Revenues

Net sales of VIVITROL were $79.9 million, compared to $69.2 million for the same period in the prior year, representing an increase of approximately 15%.
Net sales of ARISTADA were $36.1 million, compared to $24.5 million for the same period in the prior year, representing an increase of approximately 48%.
Manufacturing and royalty revenues from RISPERDAL CONSTA, INVEGA SUSTENNA/XEPLION and INVEGA TRINZA/TREVICTA were $77.2 million, compared to $79.4 million for the same period in the prior year, reflecting the timing of RISPERDAL CONSTA manufacturing shipments.
Manufacturing and royalty revenues from AMPYRA/FAMPYRA2 were $20.3 million, compared to $24.5 million for the same period in the prior year.
Research and development revenues were $16.3 million, of which $15.7 million related to the collaboration with Biogen for BIIB098, or diroximel fumarate.
Costs and Expenses

Operating expenses were $285.9 million, compared to $255.7 million for the same period in the prior year, primarily reflecting increased investment in the commercialization of VIVITROL and ARISTADA.
"Against the backdrop of the highly-anticipated upcoming regulatory interactions for ALKS 5461 for the adjunctive treatment of major depressive disorder and the ALKS 3831 ENLIGHTEN-2 pivotal study data in schizophrenia, we continue to make important progress across our other pipeline assets. BIIB098 for multiple sclerosis is on track for NDA submission by year-end and ALKS 4230, our immuno-oncology program, is gaining momentum, highlighted by the recent initiation of combination therapy evaluation," said Richard Pops, Chief Executive Officer of Alkermes. "Our results this quarter demonstrate the strong and resilient company we have carefully built over the years, with important medicines driving an expected topline in excess of $1 billion and a diverse development portfolio of late-stage product candidates, each with the potential to impact the practice of medicine and change the growth trajectory of the company. As we head into the fourth quarter, the business is well positioned for growth and the opportunities ahead."

Recent Events:

ARISTADA

Completed enrollment of six-month phase 3b study evaluating ARISTADA INITIO plus the ARISTADA two-month dose and INVEGA SUSTENNA in patients experiencing an acute exacerbation of schizophrenia
ALKS 4230

Initiated clinical evaluation of ALKS 4230 in combination with PD-1 inhibitor pembrolizumab
Submitted new clinical protocol for subcutaneous dosing phase 1 study to the ALKS 4230 Investigational New Drug (IND) application
Upcoming Milestones:

The following outlines the company’s expected upcoming milestones.

ALKS 5461

Joint meeting of the Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee to review the ALKS 5461 New Drug Application (NDA) on Nov. 1, 2018
Prescription Drug User Fee Act (PDUFA) target action date on Jan. 31, 2019
ALKS 3831

Topline results for ENLIGHTEN-2, a six-month weight study of ALKS 3831 compared to olanzapine in patients with stable schizophrenia in Q4 2018
BIIB098 (diroximel fumarate)

Planned submission of the NDA for diroximel fumarate for the treatment of multiple sclerosis in Q4 2018
ALKS 4230

Presentation of initial clinical data from ongoing dose-escalation stage of the phase 1 study at the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November 2018
Initiation of subcutaneous dosing phase 1 study in early 2019
Financial Expectations for 2018

Alkermes is updating its financial expectations for 2018 to reflect greater than expected revenues from AMPYRA. The following outlines Alkermes’ updated financial expectations for 2018.

Revenues: The company now expects total revenues to range from $1.015 billion to $1.045 billion, increased from its previous expectation of $975 million to $1.025 billion. This increase was driven by upside from AMPYRA following delayed generic competition in 2018. Included in this total revenue expectation, Alkermes continues to expect VIVITROL net sales to range from $300 million to $330 million, although closer to the lower end of this range, and ARISTADA net sales to range from $140 million to $160 million.
Cost of Goods Manufactured and Sold: The company continues to expect cost of goods manufactured and sold to range from $180 million to $190 million.
Research and Development (R&D) Expenses: The company continues to expect R&D expenses to range from $415 million to $445 million.
Selling, General and Administrative (SG&A) Expenses: The company continues to expect SG&A expenses to range from $515 million to $545 million.
Amortization of Intangible Assets: The company continues to expect amortization of intangibles to be approximately $65 million.
Net Interest Expense: The company continues to expect net interest expense to be approximately $10 million.
Income Tax Expense: The company continues to expect income tax expense of up to $10 million.
GAAP Net Loss: The company now expects GAAP net loss to range from $180 million to $210 million, or a basic and diluted loss per share of $1.16 to $1.35, based on a weighted average basic and diluted share count of approximately 155 million shares outstanding. This compares to previous expectations of GAAP net loss in the range of $210 million to $240 million, or a basic and diluted loss per share of $1.35 to $1.55, based on a weighted average basic and diluted share count of approximately 155 million shares outstanding.
Non-GAAP Net Income: The company now expects non-GAAP results to range from non-GAAP net income of $20 million to $50 million, or a non-GAAP basic earnings per share of $0.13 to $0.32 and a non-GAAP diluted earnings per share of $0.12 to $0.31, based on a weighted average basic share count of approximately 155 million shares outstanding and a weighted average diluted share count of approximately 161 million shares outstanding. This compares to previous expectations of non-GAAP net results in the range of non-GAAP net loss of $10 million to non-GAAP net income of $20 million, or a basic and diluted non-GAAP net loss per share of $0.06 to a non-GAAP basic earnings per share of $0.13 and a non-GAAP diluted earnings per share of $0.12, based on a weighted average basic share count of approximately 155 million shares outstanding and a weighted average diluted share count of approximately 161 million shares outstanding.
Share-Based Compensation: The company continues to expect share-based compensation of approximately $120 million.
Capital Expenditures: The company now expects capital expenditures to range from $65 million to $75 million, compared to a previous expectation in the range of $80 million to $90 million.
Conference Call
Alkermes will host a conference call and webcast presentation with accompanying slides at 8:30 a.m. ET (1:30 p.m. BST) on Tuesday, Oct. 23, 2018, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 888 424 8151 for U.S. callers and +1 847 585 4422 for international callers. The conference call ID number is 6037988. In addition, a replay of the conference call will be available from 11:00 a.m. ET (4:00 p.m. BST) on Tuesday, Oct. 23, 2018, through 5:00 p.m. ET (9:00 p.m. GMT) on Tuesday, Oct. 30, 2018, and may be accessed by visiting Alkermes’ website or by dialing +1 888 843 7419 for U.S. callers and +1 630 652 3042 for international callers. The replay access code is 6037988.