On March 18, 2019 CSI Laboratories, a nationally renowned laboratory specializing in comprehensive cancer diagnostics, reported that it has added the newly approved VENTANA PD-L1 (SP142) Assay as a companion diagnostic test for use in selecting triple-negative breast cancer (TNBC) patients for TENCENTRIQ (atezolizumab) (Press release, CSI Laboratories, MAR 18, 2019, View Source [SID1234534427]).

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TENCENTRIQ was granted accelerated approval for use in combination with paclitaxel protein-bound for adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 on March 8, 2019. The indication is approved under accelerated approval based on progression-free survival and continued approval may be contingent on verification and description of clinical benefit in confirmatory trials, according to the FDA.

CSI is one of the first reference laboratories to offer the new companion test. The new Assay is used to determine PD-L1 stained tumor-infiltrating immune cells of any intensity covering ≥ 1% of the tumor area.

"We are pleased to add this companion test to our diagnostic testing services, which will enable health care teams to treat TNBC patients with newer therapeutic options," said Ron Ghafary, founder and CEO of CSI Laboratories. "We continue to focus on providing a superior client experience, exceptional and accurate lab results, and quick turnaround to help our clients provide personalized cancer care to their patients."

For more information, please visit csilaboratories.com or contact Eric Diersen 678-248-8000.

On March 18, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a leading clinical-stage biopharmaceutical company developing novel cell and gene therapies for serious diseases, reported fourth quarter and full year 2018 financial results, and provided business highlights (Press release, Abeona Therapeutics, MAR 18, 2019, View Source [SID1234534483]). The Company will host a conference call on Tuesday, March 19 at 10:00 a.m. ET to discuss fourth quarter and full year results, and to provide business highlights. Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (International) or via webcast at View Source

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"The diligent work conducted over the past nine months at our GMP manufacturing facility puts us on track to initiate the pivotal VITAL study evaluating EB-101, our gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa, in mid-2019. We will produce EB-101 at our Cleveland facility, which is an important milestone for Abeona. We are also advancing our manufacturing capabilities to support our AAV gene therapy programs and expect to be at scale for GMP production in the second half of this year," said João Siffert, M.D., Chief Executive Officer. "On the lysosomal storage disease programs, we have stepped up efforts to accelerate patient enrollment in the MPS III programs and recently implemented the protocols to enroll younger, higher functioning patients."

"We believe that these important steps have positioned Abeona for success in 2019 as we focus on advancing our clinical programs and developing our pipeline utilizing novel AAV capsids," added Dr. Siffert.

Fourth Quarter and Full Year Summary Financial Results:

Cash, cash equivalents and marketable securities as of December 31, 2018 were $85.0 million, compared to $112.2 million as of September 30, 2018. The decrease in cash of $27.2 million was driven primarily by the net cash used for operating activities of $17.0 million and cash used for the acquisition of the REGENXBIO license of $10 million.

Revenues were $0.5 million for the fourth quarter of 2018 compared with $0.2 million for the fourth quarter of 2017. The increased quarterly revenues resulted from the recognition of Foundation grants that were announced during the fourth quarter of 2017.

Net loss was $0.36 per share for the fourth quarter of 2018, compared to $0.19 per share in the comparable period in 2017. For the twelve months ended December 31, 2018, net loss was $1.19 per share compared to $0.66 per share in the same period in 2017.

Fourth Quarter and Recent Highlights:

·December 6, 2018: Provided lead program updates and unveiled data from AIM AAV vector platform in cystic fibrosis and retinal diseases at R&D Day

·January 8, 2019: Appointed Christine Silverstein as Chief Financial Officer and Ed Carr as Chief Accounting Officer

·February 5-6, 2019: Presented new supportive data for novel gene therapies, including new proof-of-concept data for the AIM vector platform at WORLDSymposium

·February 11, 2019: Appointed João Siffert, M.D. as Chief Executive Officer

"As a fully-integrated organization, Abeona is on the forefront of cell and gene therapy thanks to in-house manufacturing facilities, the AIM AAV vector platform, and two programs in the clinic that have exclusive license to the AAV9 vector," said Steven H. Rouhandeh, Chairman of the Board and Executive Chairman. "Under João’s leadership, the Company is focused on maximizing these end-to-end capabilities as it prepares for important near-term milestones and beyond."

On March 18, 2019 Cellesce and Hubrecht Organoid Technology (HUB) have reported a licence agreement for the expansion of organoids using Cellesce’s own bioprocessing technology and HUB Organoid Technology for the scale up of breast organoids (Press release, Cellesce, MAR 18, 2019, View Source [SID1234553993]).

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Cellesce and HUB have an ongoing agreement for the expansion of organoids using HUB Technology. In a new project, funded by Innovate UK, Cellesce will expand breast cancer organoids using a combination of HUB Organoid Technology and Cellesce’s own bioprocessing technology. The project aims to confirm that breast cancer organoids remain genetically and phenotypically stable and continue to faithfully recapitulate the characteristics of the tumours from which they were originally derived. The project will provide support for the scaling of organoid expansion to generate the quantities of organoids required by commercial and academic researchers in drug discovery programmes.

Cellesce Chief Executive, Dr Mark Treherne, commented: "By partnering with HUB Cellesce will be in a position to provide an integrated drug discovery solution. This will be a comprehensive package that supplies organoids at scale which will increase the impact that organoid models are having on the cancer research community."

"HUB Organoid Technology will benefit from Cellesce’s innovative technology to expand large quantities of organoids such as breast cancer organoids." added Dr Rob Vries, Managing Director of the HUB.

On March 18, 2019 Cytovation AS, a privately held biotech company, reported that the first patients have been treated in a Phase I clinical study with CyPep-1, a novel therapeutic agent being developed as a new topical therapy for HPV-induced warts (Press release, Cytovation, MAR 18, 2019, View Source [SID1234561559]). The initiation of this clinical trial marks the start of the clinical development of CyPep-1 in dermatology and oncology applications, with a Phase I trial of CyPep-1 in malignant tumors planned to start in the second half of 2019.

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First patients treated in a Phase I study with CyPep-1 as an investigational therapy for benign, HPV-induced cutaneous tumors (warts).
Novel targeted lytic mechanism offers broad application for multiple indications.
Further clinical trial in malignant tumors to start in 2H 2019.
CyPep-1 has been designed and developed by Cytovation. It is a first-in-class lytic agent with broad application across benign and malignant tumors as a result of its novel mechanism of action. CyPep- 1 selectively targets tumor cells, forming pores that destabilise and rupture the membrane to kill the cell and release neoantigens into the microenvironment and circulation, while leaving healthy cells intact. The release of neoantigens enables cytotoxic CD8 positive T-cells to mount a systemic immune response offering the possibility of long-lasting immunity against the tumor and, in the case of warts, HPV infection. For more information about the mode of action, click here.

Cytovation has formulated CyPep-1 as a cream for the topical treatment of HPV-induced warts, a large medical need for which there are currently no approved drug therapies. The Company has also developed CyPep-1 as a solution for intra-tumoral injection, as a single agent or in combination with checkpoint inhibitors.

Cytovation’s Chief Scientific Officer, Lars Prestegarden, said: "We are very pleased to achieve this first clinical milestone with CyPep-1 and to begin its formal clinical development. We have designed CyPep-1 to exploit key differences between healthy and tumor cells and have seen strong evidence of its efficacy in preclinical tumor models. We are excited to see if these results can be translated to humans both with this new trial in warts and the future trial in cancer. Both trials are expected to start during 2019 and we are looking forward to reporting first findings later in the year."

The clinical trial with CyPep-1 is a randomized, placebo-controlled, double-blind Phase I study. The study is being conducted at the Centre for Human Drug Research (Leiden, the Netherlands) and will enrol 58 patients with cutaneous warts. Results are expected in the late 2019. (ClinicalTrials.gov Identifier: NCT03846648).

On March 18, 2019 Puma Biotechnology, Inc. (Nasdaq: PBYI) reported that its licensing partner Specialised Therapeutics Asia (STA) has received marketing authorization from Australia’s Therapeutic Goods Administration (TGA) to commercialize NERLYNX (neratinib) in Australia for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer following adjuvant trastuzumab based therapy (Press release, Puma Biotechnology, MAR 18, 2019, View Source [SID1234534408]). STA has submitted regulatory applications to gain approval to introduce NERLYNX in Singapore. Further applications are planned by STA in other countries in South East Asia, including Malaysia, Brunei, Vietnam and Thailand.

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TGA approval was based on the Phase III ExteNET trial, a multicenter, randomized, double-blind, placebo-controlled trial of neratinib following adjuvant trastuzumab treatment. Women (n=2,840) with early-stage HER2-positive breast cancer and within two years of completing adjuvant trastuzumab were randomized to receive either neratinib (n=1420) or placebo (n=1420) for one year.

The results of the ExteNET trial demonstrated that after two years of follow-up, invasive disease-free survival (iDFS) was 94.2% in patients treated with neratinib compared with 91.9% in those receiving placebo (HR 0.66; 95% CI: 0.49, 0.90, p=0.008).

The most common adverse reactions (>5%) were diarrhea, nausea, abdominal pain, fatigue, vomiting, rash, stomatitis, decreased appetite, muscle spasms, dyspepsia, AST or ALT increase, nail disorder, dry skin, abdominal distention, weight loss, and urinary tract infection. The most common adverse reaction leading to discontinuation was diarrhea, which was observed in 16.8% of neratinib-treated patients. Hepatotoxicity or increases in liver transaminases led to drug discontinuation in 1.7% of neratinib-treated patients.

"TGA approval marks the first time Australian women are being presented with an opportunity for extended-adjuvant therapy that will reduce the risk of disease recurrence in patients who would otherwise have had a relapse," said Carlo Montagner, Chief Executive Officer of Specialised Therapeutics. "We are pleased to be at the forefront of this new treatment paradigm and look forward to changing outcomes for these women and their families."

Puma Biotechnology’s CEO and President, Alan H. Auerbach, added, "Reducing the risk of disease recurrence remains a need for patients, despite advances in the treatment of early stage HER2-positive breast cancer. We are pleased that our partner STA will be bringing this new medicine to patients throughout Australia and would like to express our appreciation to the patients, caregivers and physicians who contributed to the neratinib clinical development program and, more specifically, the ExteNET trial. We are committed to continuing to expand NERLYNX accessibility to patients around the world."

About HER2-Positive Breast Cancer

Approximately 20 to 25 percent of breast cancer tumors over-express the HER2 protein. HER2-positive breast cancer is often more aggressive than other types of breast cancer, increasing the risk of disease progression and death. Although research has shown that trastuzumab can reduce the risk of early stage HER2-positive breast cancer returning after surgery, up to 25% of patients treated with trastuzumab experience recurrence.