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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Array BioPharma To Present At The Cowen 38th Annual Healthcare Conference

On March 5, 2018 Array BioPharma Inc. (Nasdaq: ARRY) reported that its Chief Executive Officer, Ron Squarer, will speak at the Cowen 38th Annual Healthcare Conference in Boston (Press release, Array BioPharma, MAR 5, 2018, View Source;p=RssLanding&cat=news&id=2336279 [SID1234524384]).

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The public is welcome to participate in the conference through a webcast on the Array BioPharma website.

Event:

Cowen 38th Annual Healthcare Conference

Presenter:

Ron Squarer, Chief Executive Officer, Array BioPharma

Date:

Monday, March 12, 2018

Time:

11:20 a.m. Eastern Time

Webcast:

http://wsw.com/webcast/cowen46/arry/

Avid Bioservices to Report Financial Results for Third Quarter of Fiscal Year 2018 After Market Close on March 12, 2018

On March 5, 2018 Avid Bioservices, Inc. (NASDAQ:CDMO) (NASDAQ:CDMOP), a dedicated contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that financial results for the third quarter of fiscal year 2018 ended January 31, 2018 on March 12, 2018 after market close and will host a conference call and webcast at 1:30 PM Pacific Time (4:30 PM Eastern Time) (Press release, Avid Bioservices, MAR 5, 2018, View Source [SID1234524385]). Avid’s senior management will discuss financial results for the third quarter and review recent corporate developments.

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To listen to the live webcast, or access the archived webcast, please visit: View Source

To listen to the conference call, please dial (877) 312-5443 or (253) 237-1126 and request the Avid Bioservices call.

ArQule Reports Fourth Quarter and Full Year 2017 Financial Results

On March 5, 2018 ArQule, Inc. (NASDAQ: ARQL) reported its financial results for the fourth quarter and full year of 2017 (Press release, ArQule, MAR 5, 2018, View Source [SID1234524407]).

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For the quarter ended December 31, 2017, the Company reported a net loss of $7,760,000 or $0.09 per share, compared with net loss of $6,820,000 or $0.10 per share, for the quarter ended December 31, 2016. The Company reported a net loss of $29,203,000 or $0.39 per share, for the year ended December 31, 2017, compared with a net loss of $22,718,000 or $0.33 per share, for the year ended December 31, 2016.

At December 31, 2017, the Company had a total of approximately $48,036,000 in cash and marketable securities.

Key Highlights

ARQ 531, orally bioavailable, potent and reversible BTK inhibitor, is recruiting on schedule in the phase 1a trial. Initial phase 1a data will be presented at American Association for Cancer Research (AACR) (Free AACR Whitepaper) which will be held April 14 through April 18, 2018.
Miransertib, lead proprietary AKT inhibitor, is recruiting at three sites for Overgrowth Diseases. The phase 1/2 trial for Overgrowth Diseases evaluating patients with PROS and Proteus syndrome, age two and older in the U.S. and EU, continues to dose.
Data supporting miransertib in combination with the aromatase inhibitor, anastrozole, for the treatment of advanced endometrial cancer will be presented at AACR (Free AACR Whitepaper). Phase 1b trial conducted at Memorial Sloan Kettering presents potential clinical path forward in this hard to treat disease setting.
Derazantinib, a pan-FGFR inhibitor, continues to enroll on schedule in a registrational trial for FGFR2 fusion driven intrahepatic cholangiocarcinoma (iCCA). The recruitment of patients needed to perform the interim analysis is expected to be completed by year-end.
ArQule has granted a Roivant Sciences Ltd. subsidiary an exclusive license to develop and commercialize derazantinib in the People’s Republic of China, Hong Kong, Macau, and Taiwan. Deal terms include an upfront payment to ArQule of $3 million and an additional $2.5 million development milestone within the first year. ArQule is also eligible for regulatory and commercial milestones and royalties on future sales of derazantinib in Greater China.
2018 Goals

ARQ 531 – BTK Inhibitor

Complete phase 1a trial in refractory B-cell malignancies
Publish foundational pre-clinical paper including chemical and crystal structure
Present initial phase 1a data at AACR (Free AACR Whitepaper) and additional data later this year
Miransertib – AKT Inhibitor Rare Diseases

Initiate registrational program in Proteus syndrome
Publish NIH phase 1 trial findings and compassionate use case reports
Miransertib and ARQ 751 – AKT Inhibitors Oncology

Present data from phase 1b in oncology for miransertib
Present data from phase 1a in oncology for ARQ 751
Derazantinib – FGFR Inhibitor

Complete dosing of patients needed to conduct interim analysis in registrational trial in 2nd line iCCA
Explore opportunities in additional tumor types
"During 2017 we have worked diligently to advance all the assets in our proprietary pipeline, and we are now in the position to make 10 presentations at AACR (Free AACR Whitepaper) in April demonstrating our commitment to precision medicine," said Paolo Pucci, Chief Executive Officer of ArQule. "2018 has the prospect of being a pivotal year for ArQule as we see a complete data set from the phase 1a trial of our BTK inhibitor, ARQ 531, launch a registrational program in Proteus syndrome, and explore emerging late-stage opportunities in oncology with our AKT program."

"For ArQule the scientific highlights of 2017 were achieving, in collaboration with the NIH, clinical proof of concept with miransertib in Proteus syndrome, initiating a registrational trial with derazantinib and initiating a phase 1 trial for ARQ 531," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "Consistent with our strategy we are now preparing to launch a registrational program for Proteus syndrome, as well as continuing with our ongoing trials for our BTK inhibitor, ARQ 531, and derazantinib. In addition, the data to be presented at AACR (Free AACR Whitepaper) for miransertib in combination with anastrozole will highlight a new opportunity."

Revenues and Expenses

Revenues for the quarter ended December 31, 2017, were zero, compared with revenues of $1,187,000 for the quarter ended December 31, 2016. Revenues for the year ended December 31, 2017 were zero compared with revenues of $4,709,000 for the year ended December 31, 2016. Research and development revenue in 2016 includes revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement.

Research and development expenses in the fourth quarter of 2017 were $4,721,000, compared with $6,242,000 for the fourth quarter 2016. Fiscal 2017 research and development expenses were $19,468,000 compared with $20,042,000 for fiscal 2016.

Research and development expense decreased $1.5 million in the fourth quarter of 2017 compared to the fourth quarter of 2016 primarily due to lower outsourced clinical and product development costs of $1.0 million, professional fees of $0.3 million and labor related costs of $0.2 million. Research and development expense decreased $0.6 million in 2017 primarily due to lower labor costs.

General and administrative expenses in the fourth quarter of 2017 were $1,849,000, compared with $1,808,000 for the fourth quarter of 2016. General and administrative expenses for fiscal 2017 were $7,551,000, compared to $7,563,000 for fiscal 2016.

2018 Financial Guidance

For 2018, ArQule expects revenue to range between $3 and $4 million. Net use of cash is expected to range between $26 and $28 million for the year. Net loss is expected to range between $27 and $30 million, and net loss per share to range between $(0.30) and $(0.34) for the year. ArQule expects to end 2018 with between $23 and $25 million in cash and marketable securities.

Conference Call and Webcast

ArQule will hold its fourth quarter and full year financial results call today, March 5, 2018 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events and Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. A replay will be available two hours after the completion of the call and can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events and Presentations."