Sorrento Therapeutics Autologous Anti-CEA CAR-T Cell Therapy for Liver Metastases Demonstrates Therapeutic Activity in Stage IV Pancreas Cancer in a Phase 1b HITM-SURE Trial (NCT02850536)

On March 5, 2018 Sorrento Therapeutics, Inc. (NASDAQ:SRNE) ("Sorrento"), cellular therapy focused subsidiary, TNK Therapeutics, Inc. ("TNK"), and Surefire Medical, Inc., reported initial results from the Hepatic ImmunoTherapy for Metastases-Surefire, or HITM-SURE (NCT02850536), a Phase 1b single arm trial testing its autologous anti-CEA CAR-T cells administered regionally by hepatic artery infusion (HAI) via pressure directed microvalve infusion (MVI) technology (Surefire Infusion System, Surefire Medical, Inc., Westminster, Colorado) in heavily pre-treated patients with refractory CEA-positive liver metastases (LM) (Press release, Sorrento Therapeutics, MAR 5, 2018, View Source [SID1234532251]). This Phase 1b trial follows the HITM (NCT01373047) and HITM-SIR (NCT02416466) Phase 1 studies we believe demonstrated the safety and biological activity of the anti-CEA CAR-T administered with hepatic artery infusions (HAI) alone or with selective internal radiotherapy.

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The data of HITM-SURE were provided by Dr. Steven Katz, the Principal Investigator of the Study and Associate Professor of Surgery at The Roger Williams Medical Center (CharterCare Health Partners and Prospect Medical Holdings). The study is also open at Colorado University and funded in part by the Colorado Office for Economic Development and International Trade.

In total, three patients have completed the ongoing HITM-SURE protocol, two with stage IV pancreatic cancer and one with colorectal cancer (LM). All patients presented with unresectable, chemotherapy refractory CEA+ liver metastases. Patients received three HAI of anti-CEA CAR-T cells (1e10 cells per dose) along with low dose IL-2 infusion (50,000 IU/kg/day, Proleukin, Prometheus). CAR-T HAI were administered via a Surefire MVI technology. The primary objective of the study was to establish the safety of the CAR-T HAI with the pressure directed MVI device. Secondary objectives included response assessed by modified RECIST (mRECIST), immune-related response criteria (irRC), and tumor marker kinetics. Reduction in post-treatment serum CEA was noted in all patients (average change 19 ng/mL, range 3.1-39 ng/mL). Two patients have progressive disease, with a pancreatic cancer patient alive at 7 months and a colorectal cancer patient alive at 4.8 months. A patient with stage IV pancreas adenocarcinoma has no evidence of liver metastases 11 months on PET scan following three CAR-T HAIs. In the phase III MPACT study, treatment of stage IV pancreas adenocarcinoma patients with gemcitabine plus albumin-bound paclitaxel resulted in a median overall survival time of 8.7 months. It will be of interest to determine if the results from upcoming phase 2 liver metastasis HITM studies will confirm the encouraging results from our small number of patients.

The initial findings from the currently enrolling HITM-SURE trial follows the results of two other trials. In one of the previous trials, a patient survived 51 months following 3 anti-CEA CAR-T HAIs and a patient from another trial is alive 25 months after treatment.

Dr. Katz noted, "In 15 patients in the Phase 1 and 1b studies, our CAR-T hepatic artery infusion method has resulted in highly selective delivery of CAR-T to liver tumors, with avoidance of severe cytokine release syndrome and neurotoxicity. We have observed encouraging clinical outcomes in heavily pre-treated patients. Future trials will test our novel delivery strategies for pancreatic and peritoneal tumors, in addition to novel combinatorial approaches to reverse organ-specific immunosuppressive pathways. We have developed a pipeline and delivery methods specifically tailored to address barriers to effective solid tumor CAR-T therapy, including the use of Surefire’s pressure-directed microvalve infusion technology to help overcome the high interstitial pressure of these tumors. The combination of CAR-T cells and this novel delivery mechanism are powerful tool for enhancing solid tumor uptake of CAR-T cells. Regional delivery of CAR-T cells has promise to be an important component of a multifaceted approach for advanced solid tumor patients."

"It is gratifying to observe that local infusion of CAR-T cells is very well tolerated and active in treating solid tumor metastases in the liver," said Dr. Jerome Zeldis, Sorrento Chief Medical Officer and President of TNK Therapeutics. "Based on these exciting data, we are now working on strategies to enhance the anti-solid tumor activity while lessening the complications typical of CAR-T therapy. In addition, we are planning on performing combination therapy studies using our CAR-T programs, including anti-CEA CAR-T, together with other Sorrento assets, such as our immuno-oncology checkpoint antibodies as well as Seprehvir, our clinical-stage oncolytic virus. The combination studies of anti-CEA CAR-T and Seprehvir should initiate the second half of 2018."

20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]

Qiagen has filed a 20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 20-F, , 2018, MAR 5, 2018, View Source [SID1234524431]).

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BeiGene to Present at Upcoming Investor Conferences

On March 5, 2018 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 that the company will present at two upcoming investor conferences (Press release, BeiGene, MAR 5, 2018, View Sourcephoenix.zhtml?c=254246&" target="_blank" title="View Sourcephoenix.zhtml?c=254246&" rel="nofollow">View Source;p=RssLanding&cat=news&id=2336089 [SID1234524365])

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The Cowen and Company 38th Annual Health Care Conference in Boston on Monday, March 12, at 2:10 p.m. (ET); and
The Barclays Global Healthcare Conference 2018 in Miami on Wednesday, March 14, at 4:20 p.m. (ET).
Live webcasts can be accessed from the investors section of BeiGene’s website at View Source and archived replays will be available for 90 days following each event.

BioTime to Announce Fourth Quarter and Fiscal 2017 Results on March 15, 2018

On March 5, 2018 BioTime, Inc. (NYSE American: BTX), a late stage clinical biotechnology company focused on developing and commercializing products addressing degenerative diseases, reported that it will release fourth quarter and fiscal 2017 financial and operating results on Thursday, March 15, 2018, after the close of the U.S. financial markets (Press release, BioTime, MAR 5, 2018, View Source;p=RssLanding&cat=news&id=2336117 [SID1234524386]). The Company will host a conference call and webcast on Thursday, March 15, 2018, at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss the results and recent corporate developments.

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The conference call dial-in number in the U.S./Canada is 1-866-888-8633. For international participants outside the U.S./Canada, the dial-in number is 1-636-812-6629. For all callers, please refer to Conference ID number 5878807. The live webcast can be accessed on the "Events & Presentations" page of the "Investors & Media" section on the company’s website.

A replay of the conference call will be available for seven business days beginning about two hours after the conclusion of the live call, by calling toll-free from U.S./Canada: 1-855-859-2056; international callers dial 1-404-537-3406. Use the Conference ID number 5878807. Additionally, the archived webcast will be available on the "Events & Presentations" page of the "Investors & Media" section on the company’s website.

G1 Therapeutics Announces Positive Trilaciclib Phase 2a Topline Data Showing Robust Myelopreservation Benefits in Patients with Small Cell Lung Cancer

On March 5, 2018 G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, reported positive topline data from its Phase 2a trial evaluating trilaciclib in patients undergoing chemotherapy for first-line small cell lung cancer (SCLC) (Press release, G1 Therapeutics, MAR 5, 2018, View Source [SID1234524392]). Trilaciclib is a potential first-in-class short-acting CDK4/6 inhibitor in development to preserve hematopoietic stem cells and enhance immune system function (myelopreservation) during chemotherapy.

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"The data from this trial showed clear evidence that trilaciclib preserved bone marrow and immune system function from the damaging effects of chemotherapy," said Raj Malik, M.D., Chief Medical Officer and Senior Vice President, R&D. "Moreover, the myelopreservation effects demonstrated by trilaciclib improved patient outcomes. Chemotherapy continues to be a cornerstone of cancer treatment, and trilaciclib has the potential to benefit many of these patients."

Trial Design

This double-blind, placebo-controlled trial enrolled participants with a confirmed diagnosis of extensive-stage SCLC. The trial randomized 77 treatment-naïve participants in a 1:1 ratio, and 75 received trilaciclib or placebo administered intravenously prior to each dose of standard-of-care etoposide and carboplatin (EP) chemotherapy. Participants in both arms of the trial were able to receive standard supportive care as recommended by the trial investigator. Growth factors, including granulocyte colony-stimulating factor (G-CSF) and erythropoietin, and transfusion support were available to all participants. The statistical analysis plan prospectively defined several clinically-relevant hematologic endpoints.

Key Trial Findings

Data from this signal-generating Phase 2a trial demonstrated that trilaciclib reduced clinically relevant consequences of chemotherapy-induced myelosuppression versus placebo. Trilaciclib was well tolerated, with no Grade 3/4 trilaciclib-related treatment emergent adverse events (TEAEs) reported. Baseline demographics and disease characteristics were generally well balanced between the two arms. Key hematological results are shown in the table below.

1

Parameter

EP (1) + placebo
Patients
N = 37 EP + trilaciclib
Patients
N = 38 %
Reduction P-
value (2)
Patients with Gr 3/4 Hematologic TEAEs

27 (73.0%) 9 (23.7%) 67.5 % <0.0001
Patients with Gr 3/4 Neutropenia

30 (81.1%) 15 (39.5%) 51.3 % 0.0002
Patients with Gr 4 Neutropenia

16 (43.2%) 2 (5.3%) 87.7 % 0.0001
Patients with Gr 4 Neutropenia in Cycle 1

13 (35.1%) 1 (2.6%) 92.6 % 0.0003
Cycles with Febrile Neutropenia

5 1 80.8 % 0.1542
Patients with Febrile Neutropenia

3 (8.1%) 1 (2.6%) 67.9 % 0.2773
Patients with G-CSF Administration

24 (64.9%) 4 (10.5%) 83.8 % <0.0001
Patients with Chemotherapy Cycle Delays

25 (67.6%) 15 (39.5%) 41.6 % 0.0170
Patients with Chemotherapy Dose Reductions

13 (35.1%) 3 (7.9%) 77.5 % 0.0033

(1) etoposide and carboplatin
(2) significance testing at two-sided alpha = 0.2 per prospectively defined analysis plan
The trilaciclib arm also showed favorable trends with reduced Grade 3 anemia, red blood cell transfusions, and Grade 3 thrombocytopenia versus placebo. There was no Grade 4 anemia or thrombocytopenia in either arm.

In addition to demonstrating myelopreservation benefits across multiple hematopoietic lineages, trilaciclib showed favorable trends versus placebo for overall response rate (ORR), duration of response (DOR) and progression free survival (PFS). The survival data are still immature.

• ORR by blinded independent central review (BICR): trilaciclib 66.7%, placebo 62.2% (p=0.6759)

• Median DOR (BICR): trilaciclib 5.7 months, placebo 4.3 months (p=0.1449)

• PFS (investigator, including clinical progression) median: trilaciclib 6.2 months, placebo 5.0 months (hazard ratio 0.6, p=0.06)
The company plans to share these data with U.S. and European regulatory authorities this year and discuss next steps for the development of trilaciclib. The company also plans to present results from this trial, including updated data from the Phase 1b portion, at a medical meeting later this year.

G1 is currently conducting two additional clinical trials of trilaciclib to assess myelopreservation in second- / third-line SCLC and first- / second- / third-line triple-negative breast cancer, with preliminary data from both trials expected in the fourth quarter of 2018. In addition to myelopreservation, trilaciclib’s effect on overall survival (OS) is being evaluated in a Phase 2a trial in first-line extensive stage SCLC as part of a combination regimen with Tecentriq / carboplatin / etoposide. Enrollment of that trial was completed last month, two quarters ahead of schedule.

"The strength of this dataset provides us with a solid foundation to advance the development of trilaciclib and its ultimate commercialization," said Mark Velleca, M.D., Ph.D., Chief Executive Officer. "As shown by our non-exclusive collaboration with Genentech, there is significant interest in combining trilaciclib with checkpoint inhibitor / chemotherapy regimens. We believe that trilaciclib has the potential to become backbone therapy for multiple chemotherapeutic regimens across a variety of cancer types, delivering significant benefits to patients and creating a substantial long-term commercial opportunity."

2

Webcast and Conference Call

The G1 management team will host a conference call and webcast at 8 a.m. EST today. The live call may be accessed by dialing 866-763-6020 (domestic) or 210-874-7713 (international) and entering the conference code: 3098523. A live and archived webcast will be available in the Investors section of G1’s website at www.g1therapeutics.com.

About Trilaciclib (G1T28)

Trilaciclib is a potential first-in-class short-acting CDK4/6 inhibitor in development to preserve hematopoietic stem cells and enhance immune system function during chemotherapy. Trilaciclib is administered intravenously prior to chemotherapy and has the potential to significantly improve treatment outcomes.

Trilaciclib is being evaluated in four randomized Phase 2 clinical trials: a trial in newly diagnosed, treatment-naive SCLC patients (NCT02499770), a trial in previously treated SCLC patients (NCT02514447), a trial in patients with triple-negative breast cancer (NCT02978716), and a trial in combination with Tecentriq and chemotherapy in SCLC patients (NCT03041311).