On January 8, 2016 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, reported its 2016 business outlook (Filing, 8-K, Advaxis, JAN 11, 2016, View Source [SID:1234508767]). The outlook is intended to provide Advaxis investors with an overview of anticipated events and key milestones for the coming year.

Advaxis made significant strides in its clinical development programs in 2015. Positive data was presented at the 2015 American Gynecological & Obstetrical Society Annual Meeting from a Phase 2 study of lead compound axalimogene filolisbac in metastatic cervical cancer patients (GOG 0265). Axalimogene filolisbac exceeded historical survival rates in a refractory and heterogeneous patient population and, on the basis of this data, allowed the study to move forward into stage two. Advaxis commenced dosing of a Phase 1/2 trial of axalimogene filolisbac in combination with MedImmune’s durvalumab (anti-PD-L1 immune checkpoint inhibitor) for metastatic cervical cancer and head and neck cancer. The Company also began dosing of a Phase 1/2 trial of ADXS-PSA in combination with Merck’s Keytruda (pembrolizumab; anti-PD-1 immune checkpoint inhibitor) for metastatic prostate cancer. A third product candidate, ADXS-HER2, targeting HER2 positive cancers, moved into the clinic and commenced dosing.

Advaxis made significant progress in its business operations, including raising more than $100M in capital. Presented with the challenge of a clinical hold on all of its programs, Advaxis worked closely with the U.S. Food and Drug Administration (FDA) to have the hold lifted in a timely manner.

2016 OPERATIONAL MILESTONES

Advaxis anticipates the following operational milestones in 2016:

Clinical Operations

Axalimogene Filolisbac

● Finish the Special Protocol Assessment (SPA) process and initiate enrollment in a Phase 3 trial for the treatment of high-risk, locally advanced cervical cancer (AIM2CERV) in mid-2016.

● Complete enrollment of stage 2 of GOG 0265 study in patients with metastatic cervical cancer in 2016.

● Initiate Phase 2 study in combination with Incyte’s epacadostat (IDO-1 inhibitor) for the treatment of early stage cervical cancer in the first half of 2016.

● Commence enrollment of Phase 2 trial in metastatic anal cancer (FAWCETT) in the first half of 2016.

● Complete enrollment in the combination Phase 1/2 study with MedImmune’s durvalumab (anti-PD-L1 immune checkpoint inhibitor) for the treatment of HPV-associated head and neck and cervical cancer in late 2016.

● Following the successful completion of cohort 1 of the combination, which occurred in 2015, fully enroll cohort 2 (with a higher dose of durvalumab).

● Following completion of cohort 2, a dose determination will be made and enrollment in the expansion phase of the study will commence in the first half of 2016, with completion of enrollment by the end of 2016.

● Commence and complete enrollment of the Phase 2 dose-escalation study in recurrent cervical cancer (at 1×1010 cfu) in the first half of 2016.

● Present data from a Phase 1/2 window of opportunity trial in HPV-positive head and neck cancer in the first half of 2016 at a major medical meeting.

● Commence enrollment of Phase 2 study in patients with HPV-positive, non-squamous, non-small cell lung cancer following first-line induction chemotherapy via partner in the first half of 2016.

● Commence investigator initiated studies, including for other HPV-associated cancers (e.g., penile and vaginal cancers).

ADXS-PSA

● Complete enrollment of Phase 1/2 study in combination with Merck’s Keytruda (pembrolizumab), an anti-PD-1 immune checkpoint inhibitor, for the treatment of advanced, metastatic castrate-resistant prostate cancer.

● Following the successful completion of dose escalation cohorts 1 and 2, which occurred in 2015, fully enroll cohort 3 (highest dose at 1×1010) in the first half of 2016.

● Once a dose determination is made, enrollment in the Part B combination arm with ADXS-PSA and Keytruda will commence in the first half of 2016, with completion of enrollment by the end of 2016.

ADXS-HER2

● Complete enrollment of Phase 1b dose-escalation study of ADXS-HER2 in patients with HER2-driven malignancies in the first half of 2016.

● Fully enroll Part A, which is designed to establish safe dosing levels in preparation for the expansion phase of the study in HER2-driven malignancies.

● Once a dose is selected from Part A, enrollment in a Part B expansion phase will commence in the second half of 2016.

● Following completion of Part A in the Phase 1b dose escalating study, commence collaboration and cooperative group trial with the Children’s Oncology Group in osteosarcoma.

● Initiate commercialization via Aratana Therapeutics (NASDAQ:PETX) (using the name AT-014) for the treatment of canine osteosarcoma following approval from the U.S. Department of Agriculture (USDA).

Expanding Pipeline

ADXS-NEO

● MINE (My Immunotherapy Neo-Epitopes), a collaboration with Memorial Sloan Kettering Cancer Center, will progress toward the filing of an IND in 2016. MINE uses Advaxis’s Lm Technology to develop neoepitope immunotherapies based on an individual patient’s tumor.

ADXS-TNBC

● Preclinical work will be finalized for ADXS-TNBC, a multiple-antigen construct for Triple Negative Breast Cancer (TNBC) with the goal of filing an IND in 2016.

Enhanced Manufacturing Capabilities

● Advaxis will continue to enhance its internal process/analytical development, quality systems, manufacturing and quality control infrastructure with the goal of accelerating and advancing its pipeline. The company plans to broaden its capabilities through the completion of a new pilot plant, GMP manufacturing facility, research and development labs, and quality control labs. In addition, Advaxis plans to undertake several technology transfers with its partners and install new innovative technologies to reduce lead times and to improve the overall supply chain.

Business Development

● Advaxis will continue to pursue partnerships for its cancer immunotherapy platform to enable additional research in combination with other cancer therapies and novel immunotherapies.

● Advaxis also continues to explore options for retaining a Latin American partner for axalimogene filolisbac to collaborate on co-development and registration in this important region.

SECOND HALF 2015 REVIEW

Since issuing its first half 2015 business update in June, Advaxis achieved several regulatory, clinical, business and operational milestones during the second half of 2015.

Clinical Milestones

Axalimogene Filolisbac

● August 20: Advaxis and MedImmune commenced enrollment in Phase 1/2 trial. First patient was enrolled in a Phase 1/2 study of axalimogene filolisbac in combination with MedImmune’s investigational anti-PD-L1 immune checkpoint inhibitor, durvalumab (MEDI4736), for the treatment of patients with advanced, recurrent or refractory HPV-associated cervical cancer and HPV-associated head and neck cancer.

● September 14: U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development awarded a three-year $1.1 million grant to Baylor College of Medicine for an ongoing Phase 2 trial in HPV-associated oropharynx (throat) cancer.

● September 17: Data from stage 1 of Phase 2 Study (GOG-0265) of ADXS-HPV were presented at 2015 American Gynecological & Obstetrical Society Annual Meeting. In patients with persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRMCC), who have progressed on at least one prior line of systemic therapy, treatment with axalimogene filolisbac resulted in a 38.5 percent 12-month overall survival rate in 26 patients.

● November 9: Poster at the Society for Immunotherapy Cancer annual meeting showed that axalimogene filolisbac may be safely administered with prophylactic antibiotics up to 1 x 1×1010 cfu in patients with PRMCC, a tenfold increase from prior studies.

● December 14: Advaxis receives Orphan Drug Status from the European Medicines Agency for axalimogene filolisbac for the treatment of anal cancer.

ADXS-PSA

● August 5: Movember Foundation and Prostate Cancer Foundation each awarded grants of $1 million to two research projects in metastatic, treatment-resistant prostate cancer.

ADXS-HER2

● September 28: First patient treated in Phase 1b dose-escalation study of Advaxis’s ADXS-HER2 in HER2-expressing solid tumors. The study is the first in-human study of the Lm Technology immunotherapy product for HER2 expressing cancers.

● December 1: Company receives Orphan Drug Status from the European Medicines Agency for axalimogene filolisbac for the treatment of anal cancer.

Business & Operations

Advaxis achieved the following operational milestones in the second half of 2015:

● The company significantly expanded its IP portfolio of new products including ADXS-NEO (neoepitopes) and PSA-2.0. In addition, Advaxis down-scaled its manufacturing process to enable rapid turn-around of ADXS-NEO for clinical use.

● Over the course of 2015, Advaxis expanded its leadership team, deepening its medical, clinical operations, manufacturing and regulatory affairs functions:

● Mayo Pujols, Vice President, Manufacturing

● Thomas W. Hare, Vice President, Clinical Operations

● Bob Ashworth, Vice President, Regulatory Affairs

● June 29: Daniel J. O’Connor received the 2015 Ernst & Young New Jersey Entrepreneur of the Year award.

● July 21: Advaxis received two patents from the United States Patent and Trademark Office (USPTO) of patents with composition of matter claims for ADXS-PSA and methods of use claims for ADXS-HER2.

● August 13: Advaxis appointed Tom Ridge, first Secretary of Homeland Security and 43rd Governor of Pennsylvania, to its board of directors.

● August 26: Advaxis announced a $25M licensing agreement with Knight Therapeutics to help commercialize its three lead drug candidates in Canada.

● September 10: Advaxis was the inaugural recipient of the Medical Visionary Angel Award from the Farrah Fawcett Foundation.

● October 27: Launched Research Collaboration with Memorial Sloan Kettering Cancer Center to develop neo-epitope immunotherapies based on an individual patient’s tumor (MINE).

● November 17: Received $1.6M Tax Credit from New Jersey Technology Business Tax Program.

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On January 11, 2016 SciClone Pharmaceuticals, Inc. (NASDAQ: SCLN) reported its preliminary 2015 revenue results, and that it has achieved its revenue expectations for 2015 (Filing, 8-K, SciClone Pharmaceuticals, JAN 11, 2016, View Source [SID:1234508766]).

For 2015, the Company expects 2015 sales revenues of between $157 and $158 million, which are at the high end of the guidance of $153 million to $158 million, and which represent an approximate 16% increase of its core business revenue over 2014.

Friedhelm Blobel, PhD, SciClone’s Chief Executive Officer commented: "We are very pleased with the continued growth of our core business in 2015, led by ZADAXIN which continues to be a major growth driver for our company. In 2016, we anticipate continued growth of ZADAXIN, as well as increased momentum in our oncology product portfolio. The commercial launch of DC Bead for liver cancer was an exciting 2015 achievement, and we look forward to building its sales ramp in 2016. The China pharmaceuticals market continues to represent significant growth opportunities for SciClone. We have an excellent reputation for high quality products and commercial execution, and strong industry relationships with partners and suppliers. We intend to seek additional in-licensing opportunities to expand our product portfolio and drive near- and long-term growth. We believe that our commitment to compliance is an important market differentiator, and supports our standing as a partner-of-choice in the China pharma market."

SciClone expects to report its complete fourth quarter and full-year 2015 financial results in March 2016, and plans to provide full-year 2016 financial guidance at that time. In November 2015, in conjunction with reporting its third quarter 2015 financial results, SciClone revised upwards its 2015 full-year non-GAAP earnings per share (EPS) guidance to between $0.93 and $0.97, up from its original guidance of between $0.73 and $0.77, and expects to achieve that target.

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On January 11, 2016 Varian Medical Systems (NYSE: VAR) has reported its ProBeam proton therapy system is the first such system to receive Saudi FDA medical devices marketing authorization (Press release, Varian Medical Systems, JAN 11, 2016, View Source [SID:1234508759]).

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"We are pleased to have the opportunity to make life-saving proton therapy treatments available to cancer patients in this region," said Moataz Karmalawy, general manager of Varian’s particle therapy business.

Proton therapy makes it possible to treat certain types of cancer more precisely and with potentially fewer side effects than with conventional radiation therapy. With proton therapy, the risk of damage to healthy tissues is reduced. The method can be applied for many of the most common types of cancer and offers advantages when treating tumors close to radiosensitive tissues. In pediatric patients the risk of developing a new, radiation-induced cancer later in life can be reduced.

Varian’s ProBeam technology is being used to treat patients at the Scripps Proton Therapy Center in San Diego, the Rinecker Proton Therapy Center in Munich, and at the Paul Scherrer Institute in Switzerland. Varian also has contracts for system installations at 13 other sites around the world.

On January 11, 2016 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported its business priorities and strategic outlook for 2016 (Press release, TESARO, JAN 11, 2016, View Source [SID:1234508758]).

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"2015 was an extraordinary year for TESARO, as we obtained FDA approval for and launched our first product, VARUBI, in the United States, and we look forward to bringing VARUBI to additional patients in 2016," said Lonnie Moulder, CEO of TESARO. "We are entering 2016 in a strong position and anticipate the achievement of multiple milestones with our pipeline, including the availability of the first pivotal data from our niraparib clinical programs and the initiation of clinical trials for our immuno-oncology antibody candidates. We are confident that we have a significant opportunity to build meaningful shareholder value as we continue to execute on our mission of providing transformative therapies to people bravely facing cancer."

VARUBI (Rolapitant)

VARUBI is a selective and competitive antagonist of human substance P/neurokinin-1 (NK-1) receptors. TESARO launched VARUBI in late November of 2015, following FDA approval for use in combination with other antiemetic agents in adults, for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy.

TESARO anticipates achieving the following milestones related to VARUBI in 2016:

Continue to execute on the VARUBI commercial launch in the United States;
Submit the NDA for intravenous (IV) rolapitant in Q1; and
Submit the oral rolapitant Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in Q2.

Niraparib Franchise

Niraparib is a potent, oral PARP inhibitor that is currently being evaluated in three ongoing pivotal trials. TESARO is building a robust niraparib franchise by assessing activity across multiple tumor types and by evaluating several potential combinations of niraparib with other therapeutics. The ongoing development program for niraparib includes a Phase 3 trial in patients with ovarian cancer (the NOVA trial), a registrational Phase 2 treatment trial in patients with ovarian cancer (the QUADRA trial), and a Phase 3 trial for the treatment of patients with BRCA-positive breast cancer (the BRAVO trial). In addition, a Phase 3 trial of niraparib in first-line ovarian cancer (PRIMA) and a Phase 1/2 clinical trial designed to evaluate the combination of niraparib plus KEYTRUDA (pembrolizumab) in patients with triple-negative breast cancer or ovarian cancer are slated to begin during the first quarter. Several collaborator-sponsored studies are also underway, including combination trials of niraparib plus enzalutamide, bevacizumab, and temozolomide, in patients with prostate cancer, ovarian cancer, and Ewing’s sarcoma, respectively.

Patient treatment continues in the Phase 3 NOVA trial, and based upon the most recently observed event rate, TESARO continues to expect data to be available in the second quarter of 2016. Enrollment of the QUADRA trial of niraparib for the treatment of patients with ovarian cancer who have received three or more prior lines of chemotherapy continues, and initial response rate data from this trial is anticipated to become available in the second quarter. A niraparib NDA submission is planned for the second half of 2016.

TESARO anticipates completing the following milestones related to niraparib in 2016:

Initiate patient enrollment in the Phase 3 clinical trial of niraparib in first-line ovarian cancer (PRIMA) in Q1;
Initiate patient enrollment in the niraparib/KEYTRUDA (pembrolizumab) combination trial in Q1;
Report data from the Phase 3 NOVA trial and from the QUADRA trial of niraparib in Q2;
Submit the NDA for niraparib in 2H; and
Continue to enroll the Phase 3 BRAVO trial of niraparib in breast cancer patients with germline BRCA mutations throughout 2016.

Immuno-Oncology Portfolio

TESARO’s antibody drug candidates targeting PD-1, TIM-3, and LAG-3 continue to advance, and the Investigational New Drug (IND) application for TSR-042, our anti-PD-1 antibody candidate, has been submitted to the U.S. Food and Drug Administration (FDA).

TESARO anticipates completing the following milestones related to its immuno-oncology portfolio in 2016:

Initiate a Phase 1 clinical trial of TSR-042 in Q1;
Submit the IND for TSR-022 (anti-TIM-3 antibody) in Q2;
Select a clinical candidate targeting LAG-3 in 1H 2016;
Identify a dose and schedule for TSR-042 by YE 2016; and
Select bispecific clinical candidates targeting PD-1/TIM-3 and PD-1/LAG-3 in 2016.

Year-End 2015 Cash Position

TESARO ended 2015 with approximately $230 million in cash and cash equivalents (unaudited).

About VARUBI

VARUBI is a substance P/neurokinin-1 (NK-1) receptor antagonist indicated in combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy. VARUBI is contraindicated in patients receiving thioridazine, a CYP2D6 substrate. The inhibitory effect of a single dose of VARUBI on CYP2D6 lasts at least seven days and may last longer. Avoid use of pimozide; monitor for adverse events if concomitant use with other CYP2D6 substrates with a narrow therapeutic index cannot be avoided. Please see full prescribing information, including additional important safety information, available at www.varubirx.com.

An intravenous formulation of rolapitant is also being developed. TESARO licensed exclusive rights for the development, manufacture, commercialization, and distribution of VARUBI (rolapitant) from OPKO Health, Inc.

On January 11, 2016 Spectrum Pharmaceuticals, Inc. (NasdaqGS:SPPI), a biotechnology company with fully integrated commercial and drug development operations and a primary focus in Hematology and Oncology and Servier Canada Inc., an affiliate of Servier a research-oriented pharmaceutical company which is pioneering new therapies primarily for cancer and cardiovascular diseases, reported the signing of a strategic partnership (Press release, Spectrum Pharmaceuticals, JAN 11, 2016, View Source [SID:1234508757]). As part of this partnership, Spectrum will grant the exclusive rights to develop and commercialize in Canada a franchise of four Spectrum hemato-oncology drugs: ZEVALIN (ibritumomab tiuxetan) Injection for intravenous use, Folotyn(pralatrexate injection), Beleodaq (belinostat) for Injection and Marqibo (vinCRIStine sulfate LIPOSOME injection) for intravenous infusion. Spectrum will receive $6 million in upfront payments, plus development milestone payments and royalties based on net product sales.

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"We are pleased to announce this strategic partnership with Servier, a leading company in Canada," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "This deal allows us to continue to focus on our core priorities including SPI-2012, Poziotinib, Apaziquone and Evomela. This year we plan to initiate two key trials with drugs that target blockbuster markets and we are looking forward to hearing from the FDA on two of our NDA submissions. We believe that our pipeline has never been as strong as it is today, and we continue to focus on executing on our key priorities."

"This strategic partnership between Spectrum Pharmaceuticals and Servier Canada will contribute to consolidate our global strategy in Oncology. Our ambition is to become a benchmark player in this field," said Frédéric Sesini, Executive Vice-President International Operations of Groupe Servier. "We are fully committed in providing Canadian patients with innovative treatment options. With this key partnership, Servier Canada Oncology will start operating and will work to make these treatments available soon," underlined Frederic Fasano, Chief Executive Officer of Servier Canada Inc.