On March 30, 2017 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported that the U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) voted unanimously (11 to 0) that the benefit-risk of rituximab/hyaluronidase for subcutaneous (under the skin) injection was favorable for the treatment of certain blood cancers, which include: previously untreated follicular lymphoma, previously untreated diffuse large B-cell lymphoma (DLBCL), relapsed or refractory low grade or follicular lymphoma, and previously untreated and relapsed or refractory chronic lymphocytic leukemia (CLL) (Press release, Genentech, MAR 29, 2017, View Source [SID1234518307]). This new co-formulation includes the same monoclonal antibody as intravenous Rituxan (rituximab) and hyaluronidase, a molecule that helps to deliver medicine under the skin. The FDA is expected to make a decision on approval by June 26, 2017.

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"Subcutaneous rituximab can be administered in five to seven minutes compared to an hour and a half or more for intravenous Rituxan," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "The significant reduction in administration time could especially benefit people with blood cancer who may receive years of treatment, and we are pleased the committee unanimously supported this new co-formulation."

This co-formulation has been available in the European Union since 2014 where it is known as the subcutaneous (SC) formulation of MabThera (rituximab) and is approved in approximately 50 other countries worldwide. In the United States, Rituxan is currently approved as an intravenous formulation for the treatment of people with previously untreated follicular lymphoma, previously untreated DLBCL, relapsed or refractory low grade or follicular lymphoma, and previously untreated and relapsed or refractory CLL. Intravenous Rituxan will continue to be available to patients if subcutaneous rituximab is approved. The committee’s vote does not affect intravenous Rituxan’s approved uses in the United States or in other countries.

The ODAC recommendation was based on a review of results from a clinical development program comprising five studies that together represented more than 2,000 people across key blood cancers for which intravenous Rituxan is approved. Because the monoclonal antibody in subcutaneous rituximab and intravenous Rituxan is the same, it was possible to utilize clinical studies that linked the safety and efficacy profile of the subcutaneous co-formulation to the well-established profile of intravenous Rituxan. The studies demonstrated that subcutaneous administration of the co-formulation resulted in non-inferior levels of rituximab in the blood (pharmacokinetics) and consistent clinical efficacy and safety outcomes compared to intravenous Rituxan. The clinical program also assessed patient preferences for subcutaneous rituximab and healthcare provider opinions. The studies were the following:

SparkThera (NCT00930514): Phase Ib maintenance study in previously untreated or relapsed follicular lymphoma
SABRINA (NCT01200758): Phase III induction and maintenance study in previously untreated follicular lymphoma
SAWYER (NCT01292603): Phase Ib study in previously untreated CLL
MabEase (NCT01649856): Phase III study in previously untreated DLBCL
PrefMab (NCT01724021): Phase III patient preference study in previously untreated follicular lymphoma and DLBCL
Data from these studies were included in the company’s Biologics License Application for subcutaneous rituximab submitted to the FDA.

About Rituxan

Rituxan is a monoclonal antibody designed to attach to CD20, a protein found on certain types of B-cells. It is thought to work by attacking targeted cells together with the body’s immune system. Rituxan was discovered by Biogen and is part of a collaboration between Genentech and Biogen in the United States. Since its initial approval in 1997, Rituxan has been used to treat more than 4.4 million people with blood cancers.

About Subcutaneous Rituximab

Subcutaneous rituximab is investigational in the United States. It is a co-formulation of the same monoclonal antibody as intravenous Rituxan and recombinant human hyaluronidase (rHuPH20), a technology licensed from Halozyme Therapeutics, Inc. Hyaluronidase is an FDA-approved molecule that facilitates the delivery of a relatively large volume of medicine under the skin. The subcutaneous co-formulation can be administered in five to seven minutes, compared to 1.5 to four hours for intravenous Rituxan.

(Filing, Q4/Annual, Aptose Biosciences, 2016, MAR 29, 2017, View Source [SID1234518340])

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On March 29, 2017 Delcath Systems, Inc. (NASDAQ:DCTH), an interventional oncology Company focused on the treatment of primary and metastatic liver cancers, reported financial results for the 12 months ended December 31, 2016 (Press release, Delcath Systems, MAR 29, 2017, View Source;p=RssLanding&cat=news&id=2257229 [SID1234518333]).

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Highlights for the fourth quarter of 2016 and recent weeks include:

Fourth quarter 2016 revenue increased 54.0% to $0.7 million and full year 2016 revenue increased 18% to $2.0 million;
Fully established national reimbursement coverage in Germany under ZE system;
Significantly expanded the number of clinical sites for the Company’s global Phase 3 clinical trial for patients with hepatic dominant ocular melanoma (the FOCUS Trial);
Announced a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA) for the design of a pivotal trial of Melphalan/HDS to treat patients with intrahepatic cholangiocarcinoma (ICC);
The American Journal of Clinical Oncology published a single-center retrospective review finding that the Company’s investigational percutaneous hepatic perfusion (PHP) with Melphalan/HDS offered promising results with a doubling of overall survival and significantly longer progression-free survival (PFS) and hepatic progression-free survival (HPFS) compared with other targeted therapies; and
Favorable data from two institutions were presented at the Regional Cancer Therapies Symposium and showed strong tumor response and overall survival with the Company’s investigational PHP therapy in patients with ocular melanoma that metastasized to the liver.
"Fiscal year 2016 was devoted to the advancement of our global FOCUS Trial in ocular melanoma liver metastases as well as other important clinical initiatives for our Melphalan/HDS as a treatment for primary and metastatic liver cancers, while at the same time we continued to facilitate the commercial availability of CHEMOSAT in Europe," said Jennifer K. Simpson, Ph.D., MSN, CRNP, President and Chief Executive Office of Delcath.

"Our FOCUS Trial was initiated in January 2016 under an SPA with the FDA to evaluate Melphalan/HDS as a treatment for ocular melanoma that has metastasized to the liver. During the year we activated more than 20 leading U.S. and European cancer centers as participating clinical sites in this study. We plan to have approximately 40 sites activated by the end of summer 2017.

"In addition, we recently announced a new SPA with the FDA for the initiation of a pivotal trial for the use of Melphalan/HDS in patients with ICC. This new trial will enroll approximately 295 ICC patients at approximately 40 clinical sites in the U.S. and Europe, with the primary endpoint of overall survival and secondary and exploratory endpoints including safety, progression-free survival, overall response rate and quality-of-life measures. We’ve designed this trial to be cost effective and intend to pursue it in a financially prudent manner. Given the sequential nature of the trial design, our investment in this study will be modest in 2017 as the Melphalan/HDS segment of the study will not occur until late in the year.

"In Europe, we continued to grow revenue and focus our efforts on obtaining favorable reimbursement in key markets. We believe our ZE national reimbursement in Germany, along with the continued presentation and publication of data supporting the use of CHEMOSAT by leading clinical experts validates our access efforts in other markets across Europe.

"During the year we also secured committed financing through a securities purchase agreement with an institutional investor to issue $35 million of senior convertible notes and common stock purchase warrants. Assuming all conditions are satisfied, we expect the quarterly releases of capital throughout 2017 will fund our clinical development plan through the end of the year, while also supporting our commercial activities in Europe.

"The commercial and clinical progress made throughout 2016 has been steady and we look forward to expanding access to our potentially life-saving PHP therapy for patients around the world afflicted with primary and metastatic liver cancer," concluded Dr. Simpson.

2016 Financial Results

Total revenue for 2016 of $2.0 million increased 18% from $1.7 million for 2015. Selling, general and administrative expenses for 2016 decreased to $9.4 million from $10.0 million in 2015. For 2016, research and development expenses increased to $8.4 million from $6.5 million in 2015. Total operating expenses for 2016 were $17.9 million compared with $16.5 million for 2015.

The Company reported a net loss for 2016 of $18.0 million or $10.59 per share based on 1.7 million weighted average common shares outstanding, compared with a net loss for 2015 of $14.7 million or $14.56 per share based on 1.0 million weighted average common shares outstanding. The increase is primarily due to a $14.3 million increase in interest expense primarily related to the amortization of debt discounts, a non-cash item, and a $1.4 million increase in operating expenses primarily related to increased investment in clinical trial initiatives. This was offset by a $12.2 million change in the fair value of the warrant liability, a non-cash item, and a $0.2 million improvement in gross profit due to higher sales.

Balance Sheet Highlights

As of December 31, 2016, Delcath had cash and cash equivalents of $4.4 million, compared with $12.6 million as of December 31, 2015. During 2016 the Company used $14.2 million of cash to fund operating activities. Delcath believes it has sufficient capital and access to committed capital to fund its operating activities through the first quarter of 2018.

(Filing, Annual, Rosetta Genomics, 2016, MAR 29, 2017, View Source [SID1234518386])

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On March 28, 2017 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company pioneering the field of regenerative medicine, reported financial and operational results for the fourth quarter and year ended December 31, 2016 as well as recent corporate progress (Press release, BioTime, MAR 28, 2017, View Source;p=RssLanding&cat=news&id=2257051 [SID1234518294]).

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"Asterias has achieved substantial progress over the last year, highlighted by previously announced positive interim efficacy and safety results from our ongoing SCiStar Phase 1/2a clinical trial, as well as initiation of operations at our new cGMP manufacturing facility for AST-OPC1," said Steve Cartt, President and Chief Executive Officer. "We are particularly encouraged by the results we have seen to date in the SCiStar trial, which we believe provide solid evidence of the potential for AST-OPC1 to help patients with complete paralysis recover arm, hand and finger function. Such recovery is critically important to increasing both independence and quality of life in patients who suffer complete cervical spinal cord injuries. In 2017, we look forward to completing study enrollment, engaging in discussions with the FDA to determine our optimal clinical path forward, and reporting more efficacy and safety data later in the year as it becomes available."

As of February 28, 2017, Asterias’ cash and cash equivalents totaled $19.9 million and combined with its available-for-sale securities totaled $33.5 million.

"Our cash position is significantly improved from the same time last year, which allows us to continue to advance our programs towards important development milestones," said Ryan Chavez, Chief Financial Officer. "We believe we currently have sufficient capital to fund operations through at least the first quarter of 2018."

2016 and Recent Key Achievements

Presented positive early efficacy data from the first cohort of the SCiStar study testing a dose of AST-OPC1 within the predicted efficacious dose range. In this cohort, 10 million AST-OPC1 cells were delivered to patients with neurologically complete (AIS-A 10) cervical injuries. Patients from this cohort showed improvement in upper extremity motor function at 3-months following administration of AST-OPC1. Asterias subsequently reported efficacy results for five patients from this cohort that showed additional motor function improvement at 6-months and 9-months following administration of AST-OPC1. Earlier this month, Asterias reported that the sixth, and final, patient in this cohort showed additional motor function improvement at 6-months following administration of AST-OPC1. The results suggest a meaningful and favorable difference to date in recovery of arm, hand and finger function in patients treated with the 10 million cell dose of AST-OPC1 versus the level of expected rates of spontaneous recovery shown from historical control data of a closely matched patient population.
Received FDA clearance to expand the SCiStar study to include two additional cohorts of subjects with less severe AIS-B (motor complete, sensory incomplete) injuries.
Commenced dosing of two additional cohorts in the SCiStar study: AIS-B patients treated with 10 million AST-OPC1 cells; and, following clearance by the study’s Data Monitoring Committee, AIS-A patients treated with the higher dose of 20 million AST-OPC1 cells.
Successfully completed the validation and start-up of its internal current Good Manufacturing Practices (cGMP) manufacturing facility at Asterias headquarters in Fremont, CA in preparation for late-stage clinical trials of AST-OPC1.
Reported positive long-term follow-up results from the Phase 1 clinical trial of AST-OPC1.
Received Orphan Drug Designation from FDA for AST-OPC1 for the treatment of acute spinal cord injury.
Successfully completed an End-of-Phase 2 meeting with the FDA for AST-VAC1 in Acute Myeloid Leukemia (AML).
Completed clinical protocol development and the transfer of the manufacturing processes to produce AST-VAC2 to Cancer Research UK (CRUK) as part of the company’s ongoing partnership with CRUK to conduct a Phase 1/2a study for AST-VAC2 in non-small cell lung cancer.
Financial Results

Total revenues were $1.8 million for the fourth quarter and $7.0 million for the year ended December 31, 2016. Revenues were comprised of grant income as well as royalty revenues on product sales by licensees. Research and development expenses were $7.9 million in the fourth quarter and $25.5 million in the year ended December 31, 2016, with the primary driver being expenses associated with our AST-OPC1 program. General and administrative expenses were $2.4 million in the fourth quarter and $15.5 million in the year ended December 31, 2016. Non-cash expenses related to warrants distributed to shareholders other than BioTime earlier in the year, which were included in general and administrative expenses, were $5.3 million.

Net loss was $9.3 million, or $0.20 per share, for the fourth quarter and $35.5 million, or $0.83 per share, for the year ended December 31, 2016. For the year ended December 31, 2016, net cash used in operating activities was $19.0 million and net cash provided from financing activities was $28.5 million.

As of December 31, 2016, Asterias’ cash and cash equivalents totaled $19.8 million and combined with its available-for-sale securities totaled $35.1 million.