On December 9, 2017 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly targeted and immuno-oncology drugs for the treatment of cancer, reported preliminary clinical data from an ongoing Phase 1b trial of its investigational Bruton’s Tyrosine Kinase (BTK) inhibitor zanubrutinib (BGB-3111) in patients with non-Hodgkin’s lymphoma (NHL) in an oral presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, GA (Press release, BeiGene, DEC 9, 2017, View Source;p=RssLanding&cat=news&id=2321942 [SID1234522476]). The preliminary data included patients with aggressive NHL subtypes such as diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL) as well as indolent NHL subtypes such as follicular lymphoma (FL) and marginal zone lymphoma (MZL). The Phase 1b data suggest that zanubrutinib was generally well-tolerated and had anti-tumor activity across these NHL subtypes.

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"In this Phase 1b trial, zanubrutinib was well-tolerated across multiple NHL subtypes, with very low rates of toxicity-related treatment discontinuation in both indolent and aggressive disease settings. These preliminary data also indicate that zanubrutinib’s complete and sustained BTK occupancy translates into high response rates in NHL subtypes beyond Waldenström’s macroglobulinemia and Chronic lymphocytic leukemia, for which data have previously been reported," commented Constantine Tam, MD, Disease Group Lead for Low Grade Lymphoma and Chronic Lymphocytic Leukemia at Peter MacCallum Cancer Centre, Director of Haematology at St. Vincent’s Hospital, Australia, and lead author of the presentation.

"Building upon the promising Phase 1b data we have presented for zanubrutinib, we are pleased to report results from additional NHL subtypes enrolled in our Phase 1b trial. We continue to pursue broad development of zanubrutinib with ongoing pivotal trials in a range of NHL subtypes both globally and in China," commented Jane Huang, MD, Chief Medical Officer, Hematology at BeiGene.

Summary of Results from the Ongoing Phase 1b Trial

The multi-center, open-label Phase 1b trial of zanubrutinib in patients with B-cell malignancies is being conducted in Australia, New Zealand, South Korea, the United States, and Europe, and consists of a dose-escalation phase and a dose-expansion phase in disease-specific cohorts. The ongoing dose-expansion component is testing doses of 160 mg twice daily (BID) or 320 mg once a day (QD). As of September 15, 2017, the date of the most recent data cutoff, 99 patients with NHL subtypes other than chronic lymphocytic leukemia/small lymphocytic lymphoma and Waldenström’s macroglobulinemia were enrolled in the study, including 34 patients in the indolent lymphoma cohort, which consisted of 24 patients with FL and 10 patients with MZL, and 65 patients in the aggressive lymphoma cohort, which consisted of 27 patients with DLBCL and 38 patients with MCL. The median follow-up time was 5.6 months (0.3-22.3 months) and 5.1 months (0.1-31.9) for indolent and aggressive lymphoma, respectively.

At the time of data cutoff, the most frequent adverse events (AEs) (occurring in ≥15% of patients) of any attribution among 34 patients with indolent lymphoma were petechiae/purpura/contusion (24%), upper respiratory tract infection (URTI) (21%), nausea (18%) and pyrexia (15%). The most frequently reported grade 3 or greater AEs (occurring in ≥5% of patients) of any attribution were anemia (9%), neutropenia (9%), urinary tract infection (6%), and abdominal pain (6%). Serious AEs were reported in 11 patients (32%). Of those, four patients had serious AEs that were considered possibly related to zanubrutinib, including one case each of nausea, urinary tract infection, diarrhea, and creatinine increase.

The most frequent AEs (occurring in ≥15% of patients) of any attribution among 65 patients with aggressive lymphoma were petechiae/purpura/contusion (25%), diarrhea (23%), constipation (22%), fatigue (18%), URTI (18%), anemia (17%), cough (15%), pyrexia (15%), and thrombocytopenia (15%). The most frequently reported grade 3 or greater AEs (occurring in ≥5% of patients) of any attribution were anemia (11%), neutropenia (9%), thrombocytopenia (9%), and pneumonia (6%). Serious AEs were reported in 26 patients (40%). Of those, three patients had serious AEs that were considered possibly related to zanubrutinib, including one case each of peripheral edema and joint effusion (occurring in the same patient), pneumonia, and pneumonitis.

At the time of data cutoff, 26 patients with indolent lymphoma including 17 patients with FL and nine patients with MZL were evaluable for efficacy. In patients with FL, the overall response rate (ORR) was 41% with complete responses (CRs) in 18% and partial responses (PRs) in 24% of patients. Stable disease (SD) was observed in 41% of patients. Progressive disease (PD) was observed in one patient. In patients with MZL, the ORR was 78% with no CR, and PRs in 78% of patients. SD was observed in 22% of patients. No PD was observed.

Fifty-eight patients with aggressive lymphoma including 26 patients with DLBCL and 32 patients with MCL were evaluable for efficacy. In patients with DLBCL, the ORR was 31% with CRs in 15% and PRs in 15% of patients. In patients with MCL, the ORR was 88% with CRs in 25% and PRs in 63% of patients.

About Zanubrutinib

Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of BTK that has demonstrated higher selectivity against BTK than ibrutinib (a BTK inhibitor currently approved by the U.S. Food and Drug Administration and the European Medicines Agency) based on biochemical assays, higher exposure than ibrutinib based on their respective Phase 1 experience in separate studies, and sustained 24-hour BTK occupancy in both the peripheral blood and lymph node compartments.

On December 8, 2017 Pacira Pharmaceuticals, Inc. (NASDAQ:PCRX) reported that the company is scheduled to present at the 2017 BMO Capital Markets Prescriptions for Success Healthcare Conference at 11:30 AM ET on Thursday, December 14, 2017 in New York City (Press release, Pacira Pharmaceuticals, DEC 8, 2017, View Source;p=RssLanding&cat=news&id=2321829 [SID1234522467]).

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A live audio webcast of the Pacira presentation can be accessed by visiting the "Investors & Media" section of the company’s website at investor.pacira.com. A replay of the webcast will be archived on the Pacira website for two weeks following the presentation date.

On December 8, 2017 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported new results from two studies with EndoPredict are being featured at the 2017 San Antonio Breast Cancer Symposium (SABCS) in San Antonio, Texas (Press release, Myriad Genetics, DEC 8, 2017, View Source [SID1234522466]). EndoPredict is a second-generation prognostic gene expression test for patients with breast cancer.

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"We are excited to present new data on our EndoPredict test which demonstrates our ongoing commitment to collaborate with leading academic research centers and advance personalized medicine for patients with breast cancer," said Ralf Kronenwett, M.D., Ph.D., director of International Medical Affairs, Myriad Genetics. "Importantly, these new studies add to the expanding body of evidence demonstrating how EndoPredict can be used to predict both disease recurrence as well as response to therapy."
The data are highlighted below and abstracts are available at: View Source Follow Myriad on Twitter via @MyriadGenetics and stay informed about symposium news and updates by using the hashtag #SABCS17.

EndoPredict Podium Presentation
Title: The EndoPredict score predicts residual cancer burden to neoadjuvant chemotherapy and to neuroendocrine therapy in HR+/HER2- breast cancer patients from ABCSG34.
Presenter: Peter Dubsky, M.D., Medical University of Vienna, Austria and the Breast Center St. Anna Klinik, Lucerne.
Date: Friday, Dec. 8, 2017, 3:15—5:00 p.m.
Location: Podium, GS6-04

This study was designed to show the predictive value of the EndoPredict (EP) 12-gene molecular score for tumor response to neoadjuvant chemotherapy and neoendocrine therapy. The study included biopsies from 217 women with HR+ breast cancer. Of these, 134 patients were assigned to receive neoadjuvant chemotherapy according to aggressive clinico-pathologic tumor features. The remaining 83 patients were clinically identified as having luminal A types of breast cancer and were assigned to receive neoendocrine treatment. The primary endpoint was residual cancer burden RCB0/I (i.e., good tumor response) vs. RCB II/III (i.e., poor tumor response) at time of surgery.
In the neoadjuvant chemotherapy group, 125 patients had high EP scores and nine had a low EP score. The results show that 26.4 percent of those with a high score showed a good tumor response (RCB0/I) to neoadjuvant chemotherapy, while all patients with a low score showed only a poor tumor response (Table 1). In the "luminal A" group receiving neoendocrine therapy, 39 patients had a high EP score and 44 had a low EP score. The results show that 27.3 percent of those with a low EndoPredict score and 7.7 percent with a high score achieved excellent tumor response (RCB0/I) to neoendocrine therapy (Table 1).

Table. 1 EndoPredict
Low Score EndoPredict
High Score
p-Value
Response to
Neoadjuvant
Chemotherapy 0.0 % 26.4 % p=0.0001
Response to
Endocrine Therapy 27.3 % 7.7 % P=0.015

"This exciting study is evidence that women with a high EP score responded better to neoadjuvant chemotherapy than those with a low score, while those with a low EndoPredict score responded better to neoadjuvant endocrine therapy," said Peter Dubsky, M.D., principal investigator, speaking on behalf of the Austrian Breast and Colorectal Cancer Study Group (ABCSG). "These findings are relevant to better patient selection for biomarker driven studies in the neoadjuvant setting."

EndoPredict Poster Presentation
Title: The role of EndoPredict in invasive lobular carcinoma.
Presenter: Ivana Sestak, Ph.D., Centre for Cancer Prevention, Wolfson Institute of Preventive Medicine, Queen Mary University of London.
Date: Thursday, Dec. 7, 2017, 5:00—7:00 p.m.
Location: Poster, P3-08-01

This study evaluated the role of EndoPredict molecular-clinical score (EPclin) for the prediction of distant recurrence in women diagnosed with invasive lobular carcinoma (ILC) compared to those with invasive ductal carcinoma (IDC). The study included 928 women with E R+/HER2- breast cancer: 141 had ILC, 710 had IDC and 77 were mixed type.
This result shows that EndoPredict provided significant power for predicting distant recurrence in patients with both ILC (EPclin: LR-X2=5.8) and IDC (EPclin: LR-X2=13.8). Women with ILC who had a high EPclin score were at seven times increased risk of 10-year distant recurrence with endocrine therapy only than patients with low EPclin score. In comparison, women with IDC who had a high EPclin score were at five times increased risk of 10-year recurrence than patients with low EPclin score. Importantly, there was a similar 10-year distant recurrence risk in patients with a low EPclin score (~6 percent), which suggests that chemotherapy is not indicated in these patients with a low risk score regardless of tumor type.
"Our results show that EndoPredict provided highly significant prognostic information and risk stratification in women with invasive lobular carcinoma," said Ivana Sestak, Ph.D., principal investigator, Centre for Cancer Prevention, Wolfson Institute of Preventive Medicine, Queen Mary University of London. "Importantly, the 10-year risk of distant recurrence in the EndoPredict low-risk groups was similar between ILC and IDC, suggesting that chemotherapy is not indicated for these patients, irrespective of tumor type."

About EndoPredict
EndoPredict is a second-generation, multigene prognostic test for patients diagnosed with breast cancer. The test provides physicians with information to devise personalized treatment plans for their patients. EndoPredict has been validated in approximately 4,000 patients with node-negative and node-positive cancer and has been used clinically in more than 20,000 patients. In contrast to first-generation multigene prognostic tests, EndoPredict detects the likelihood of late metastases (i.e., metastasis formation after more than five years) and, therefore, can guide treatment decisions regarding the need for chemotherapy, as well as extended anti-hormonal therapy. Accordingly, therapy decisions backed by EndoPredict confer a high level of diagnostic safety. For more information, please visit: www.endopredict.com.

On December 7, 2017 Sandoz, a division of Novartis and the global leader in biosimilars, reported data demonstrating the pharmacokinetics (PK), pharmacodynamics (PD), safety and immunogenicity of proposed biosimilar pegfilgrastim as compared to the reference biologic, Neulasta*[1] (Press release, Novartis, DEC 7, 2017, View Source [SID1234522450]). The Phase I study, conducted in healthy volunteers, confirmed that Sandoz biosimilar pegfilgrastim matches the reference biologic in terms of PK, PD, safety and immunogenicity profiles[1]. The data were presented during the 2017 San Antonio Breast Cancer Symposium.

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"At Sandoz, we are committed to developing high-quality biosimilar and generic medicines that provide the oncology community with treatment options to help manage their patients," said Mark Levick, MD PhD, Global Head of Development, Biopharmaceuticals, Sandoz, "And it starts with following the science. These findings add to the totality of evidence supporting our proposed biosimilar pegfilgrastim."

Sandoz biosimilar pegfilgrastim is currently under review by the European Medicines Agency (EMA) for use in the same indication as the reference biologic. Pegfilgrastim is a long-acting formulation of filgrastim (granulocyte colony-stimulating factor [G-CSF])[2].

About the study
Study participants were randomized to receive a single 6 mg subcutaneous injection of biosimilar pegfilgrastim or reference medicine on Day 0[1]. After dosing, study participants underwent a 4-week assessment period followed by a 8-week washout period before crossing over to receive the other medicine, and were assessed for a further 4-weeks[1].

The results demonstrated that Sandoz proposed biosimilar pegfilgrastim matched the reference medicine in the PK and PD comparisons as primary endpoints, in terms of absolute neutrophil count (maximum effect attributed to study medication) (95% CI: [0.97, 1.02]) and maximum serum concentration of study medication after administration (90% CI: [1.03, 1.19])[1]. Secondary endpoints of safety and immunogenicity were comparable between both groups[1].

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved generic or biosimilar products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Neither can there be any guarantee that, if approved, such generic or biosimilar products will be approved for all indications included in the reference product’s label. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; the particular prescribing preferences of physicians and patients; competition in general, including potential approval of additional generic or biosimilar versions of such products; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; litigation outcomes, including intellectual property disputes or other legal efforts to prevent or limit Sandoz from selling its products; general economic and industry conditions, including the effects of the persistently weak economic and financial environment in many countries; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

On December 8, 2017 Personalis, Inc., a leading provider of advanced genomic sequencing and analytics to support the development of personalized cancer vaccines and other next-generation cancer immunotherapies, reported that the company’s Chief Scientific Officer (CSO), Dr. Richard Chen, will be speaking at the upcoming Neoantigen Summit, being held in Boston, MA from November 14-16, 2017 (Press release, Personalis, DEC 8, 2017, View Source [SID1234522469]).

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The presentation entitled, "From Sample to Neoantigens for Vaccines: Key Challenges and Solutions" will cover topics including:

cfDNA Neoantigens: dealing with tumor heterogeneity
Improving neoantigen identification
Elaborating tumor microenvironment (TME), immuno-modulators and vaccine response biomarkers
Overcoming poor sample quality and quantity for NGS sequencing
Addressing sequencing coverage gaps that can harbor neoantigens
Validation and regulatory issues on the way to commercialization
Central to this presentation will be Personalis’ ACE ImmunoID Platform. The platform enables the comprehensive characterization of a tumor’s immuno-genomics including neoantigens, the tumor microenvironment, HLA, immuno-modulators and mechanisms of tumor escape to aid rational vaccine design.

"We are continuing to innovate to help our customers build safe, effective, and scalable personalized cancer vaccines. In this talk we will discuss how our ACE ImmunoID platform has been designed to overcome key challenges in personalized cancer vaccine development process starting from a tumor sample to neoantigen identification and rational vaccine design," said Dr. Richard Chen, CSO at Personalis.

Personalis will also be exhibiting during the Neoantigen Summit. Representatives will be available to answer questions about the company’s neoantigen identification and immuno-genomics capabilities.