On April 1, 2019 Cytori Therapeutics (NASDAQ: CYTX) ("Cytori" or the "Company") reported its fourth quarter and year-end 2018 financial results and provided updates on corporate activities (Press release, Cytori Therapeutics, APR 1, 2019, View Source [SID1234534864]). Also announced was a transaction to divest certain cell therapy assets to Lorem Vascular of Melbourne, Australia yielding $4MM in non-dilutive funding to the Company .

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Fourth quarter 2018 net loss was $2.2 million, or $0.16 per share. Operating cash burn for the fourth quarter of 2018 was approximately $2.5 million. Cytori ended the year with approximately $5.3 million of cash and cash equivalents.

"This transaction sale accomplishes a number of important objectives for the company," said Dr. Marc Hedrick, Cytori President & Chief Executive Officer. "Most critically it allows us to further increase the focus on our clinical stage oncology pipeline while bringing in non-dilutive capital. We also are able to maintain our most valuable cell therapy assets, including Japan that has a forthcoming trial readout in our ADRESU trial."

Our lead clinical stage asset, Doxorubicin Hydrochloride Cytori, formerly called ATI-0918, is an important potential therapy for Breast and Ovarian Cancer, Multiple Myeloma and Kaposi’s Sarcoma. Our current development program is focused in Europe where we believe there is a potential market opportunity of $120 million annually. In Q1 2019, Cytori submitted a letter of intent to file a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Doxorubicin Hydrochloride Cytori. Doxorubicin Hydrochloride Cytori is being developed as a generic version of Janssen’s Caelyx pegylated liposomal doxorubicin. The Company continues to evaluate potential development and commercialization partnering opportunities for Doxorubicin Hydrochloride Cytori with a focus on Europe and China. European approval and launch of Doxorubicin Hydrochloride Cytori is projected to be in late 2020.

Our second clinical stage oncology focused asset is ATI-1123, a phase II ready, patented, albumin-stabilized pegylated liposomal docetaxel. In 2018, the Company received an orphan drug designation from the U.S. FDA for the indication of small cell lung cancer and is pursuing a 505(b)(2) new drug application (NDA) pathway in the U.S. which may offer an accelerated clinical timeline and lower development cost. The Company is exploring near term development strategies and intends to advance this program aggressively in 2019.

Cytori’s ADRESU pivotal urinary incontinence trial using Cytori Cell Therapy has completed enrollment and anticipates data read out in the second quarter of 2019. If the data is positive, Cytori intends to seek expedited approval and reimbursement for the Japanese market for this indication. In Q1 2019, Cytori received approval from the United States Food & Drug Administration to expand the enrollment criteria for its RELIEF clinical trial of intravenously delivered Cytori Cell Therapy for patients with severe burn injuries.

Q4 2018 and Full Year 2018 Financial Performance

Q4 2018 and full year operating cash burn was $2.5 million and $12.0 million, compared to $4.2 million and $18.1 million for the same periods in 2017, respectively.
Q4 2018 and full year product revenues were $0.4 million and $2.7 million, compared to $0.7 million and $2.7 million for the same periods in 2017, respectively.
Q4 2018 and full year contract revenues were $0.7 million and $3.0 million, compared to $0.9 million and $3.7 million for the same periods in 2017, respectively.
Cash and debt principal balances at December 31, 2018 were approximately $5.3 million and $13.0 million, respectively.
Q4 2018 adjusted net loss was $2.8 million or $0.20 per share, compared to a net loss of $4.3 million or $1.00 per share for the same period in 2017. The adjusted net loss excludes a non-cash beneficial conversion feature (a non gaap measure) related to the issuance of our Series C convertible preferred shares in the third quarter of 2018 of $2.5 million, as well as a credit of $0.6 million related to a change in fair value of warrant liability (a non gaap measure). Q4 2018 net loss allocable to common stockholders was $2.2 million, or $0.16 per share.
Full year 2018 adjusted net loss was $14.9 million or $1.71 per share, compared to $21.0 million or $6.48 per share for the same period in 2017. The adjusted net loss excludes a non-cash beneficial conversion feature (a non gaap measure) related to the issuance of our Series C convertible preferred shares in the third quarter of 2018 of $2.5 million, as well as a credit of $2.2 million related to a change in fair value of warrant liability (a non gaap measure). Full year 2018 net loss allocable to common stockholders was $15.1 million, or $1.74 per share.
Selected Key Anticipated Milestones:

Doxorubicin Hydrochloride Cytori: File Market Authorization Application to the European Medicines Agency in late 2019 or early 2020.
ATI-1123: Clarify the FDA 505(b)(2) pathway applicability and announce clinical development plan in mid 2019.
Cell Therapy Japan: Report ADRESU urinary incontinence pivotal clinical trial results in Q2 2019.
Management Conference Call Webcast

Cytori will host a management conference call at 5:30 p.m. Eastern Time today to further discuss its progress. The webcast will be available live and by replay two hours after the call and may be accessed under "Webcasts" in the Investor Relations section of Cytori’s website. If you are unable to access the webcast, you may dial in to the call at +1.877.402.3914, Conference ID: 8766078.

On April 1, 2019 Sierra Oncology, Inc. (Nasdaq: SRRA), a clinical stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that preclinical data for SRA737+LDG, its Chk1 inhibitor plus non-cytotoxic low dose gemcitabine (LDG), in combination with immunotherapy, are being reported today in a late-breaking oral presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 being held in Atlanta, Georgia (Press release, Sierra Oncology, APR 1, 2019, View Source [SID1234534861]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Given the limited response rates for anti-PD-L1 drugs in patients with small cell lung cancer (SCLC), better treatment options are sorely needed. Our data, showing durable tumor regressions when combining SRA737+LDG with anti-PD-L1 in a mouse model, warrant further clinical studies to investigate the potential of this approach as a strategy to improve outcomes in our patients," said senior researcher Lauren Averett Byers, M.D., Associate Professor of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center, Houston, Texas.

In the study, SRA737+LDG demonstrated significant anti-tumor activity when combined with anti-PD-L1 in a mouse model of SCLC, resulting in durable tumor regressions. These results were accompanied by an induction of anti-tumor cytotoxic T-cells and M1 macrophages and a corresponding reduction in several pro-tumorigenic cell types in the tumors of drug-treated animals.

"The unique replication stress-inducing properties of SRA737 as potentiated by non-cytotoxic low dose gemcitabine have been previously demonstrated to result in intrinsic anti-tumor activity in multiple preclinical models. These striking new data provide evidence for SRA737+LDG’s activation of innate immune signaling and the establishment of an anti-tumor immune microenvironment that synergizes with immune checkpoint inhibitors," said Dr. Christian Hassig, Chief Scientific Officer, Sierra Oncology. "These findings provide a mechanistic basis for the growing body of evidence highlighting a synergistic interplay between replication stress and anti-tumor immune responses and afford a compelling rationale for evaluating SRA737+LDG with immunotherapy in the clinic."

SRA737 AACR (Free AACR Whitepaper) Late-Breaking Oral Presentation:
Date/Time: Monday, April 1st from 3:00 to 5:00 pm ET
Session: Minisymposium: Late-Breaking Research 2
Title: Combination treatment of the CHK1 inhibitor, SRA737, and low dose gemcitabine demonstrates profound synergy with anti-PD-L1 inducing durable tumor regressions and modulating the immune microenvironment in small cell lung cancer
Authors: Triparna Sen, Carminia M. Della Corte, Snezana Milutinovic, Lixia Diao, Robert J Cardnell, Ryan J. Hansen, Bryan Strouse, Michael P. Hedrick, Christian Hassig, Jing Wang, Lauren A Byers.
Location: Georgia World Congress Center, Room B404

Summary slides from this presentation will be made available on the company’s website at www.sierraoncology.com

About SRA737+LDG
SRA737 is a potent, highly selective, orally bioavailable small molecule inhibitor of Checkpoint kinase 1 (Chk1), a key regulator of cell cycle progression and the DNA Damage Response (DDR). SRA737+LDG is a novel drug combination, where non-cytotoxic low dose gemcitabine acts as a potent inducer of replication stress that potentiates SRA737’s anti-tumor activity.

Phase 1/2 clinical trials of SRA737 as monotherapy (NCT02797964) and in combination with low dose gemcitabine (NCT02797977) in multiple solid tumors (ovarian, prostate, non-small cell lung, squamous (head & neck, anal), colorectal, small cell lung, sarcoma, cervical, anogenital) are ongoing. Sierra has also prepared for a potential clinical study of SRA737 in combination with a PARP inhibitor.

Sierra Oncology retains the global commercialization rights to SRA737.

On April 1, 2019 Vaccibody AS and Nektar Therapeutics (Nasdaq: NKTR) reported the presentation of new preclinical data for VB10.NEO, a personalized neoantigen cancer vaccine, combined with bempegaldesleukin (NKTR-214 or bempeg), a CD122-preferential IL-2 pathway agonist (Press release, Vaccibody, APR 1, 2019, View Source [SID1234534860]). These data were presented today in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to present these novel preclinical data that show combining bempeg with VB10.NEO synergize to increase both the breadth and the depth of the neoantigen-specific immune response. These unique and non-overlapping mechanisms produced an expansion of the VB10.NEO elicited neoantigen-specific T cells and demonstrated enhanced anti-tumor efficacy in mice. We look forward to evaluating this novel immuno-oncology combination in a clinical study in patients with advanced or metastatic squamous cell carcinoma of head and neck later this year," said Agnete B. Fredriksen, Ph.D., Vaccibody’s President and Chief Scientific Officer.

VB10.NEO is designed to specifically activate a patient’s immune system to tumor-specific antigens, called neoantigens. Bempeg is designed to expand and proliferate tumor antigen-specific T cells in the tumor microenvironment. Addition of bempeg to VB10.NEO is intended to drive maximal expansion of vaccine-induced neoantigen-specific T cells for the treatment of cancer.

"Personalized T cell vaccines could play a critical and central role in cancer immunotherapy," said Jonathan Zalevsky, Ph.D., Chief Scientific Officer at Nektar. "These preclinical data highlight the potential of combining a personalized cancer vaccine with a T cell proliferator to induce maximal expansion of vaccine-induced T cell clones and durable responses and specific anti-tumor immunity. We are highly encouraged by these results and look forward to testing this unique approach to personalized cancer treatment in patients with squamous cell carcinoma of the head and neck."

Details of the poster presentation at AACR (Free AACR Whitepaper) are as follows and will be available for download at the time of presentation at View Source and View Source

Abstract #2256
Title: "Combination of neoantigen DNA plasmid vaccine VB10.NEO and NKTR-214, a CD122-biased immunostimulatory cytokine, induces strong neoantigen-specific T cell responses and sustained tumor regression in pre-clinical models"
Session: Clinical Research – Combination Therapies 1
Session Data and Time: Monday, April 1, 2019, 1:00 p.m. – 5:00 p.m. Eastern Time

Combination of VB10.NEO and NKTR-214 synergizes to elicit greater breadth and depth of neoantigen-specific T cell responses than each individual treatment.
The synergistic effect was observed in both CD4 and CD8 T cells, and most pronounced on CD8 T cell responses, further supporting the combination’s potential to induce strong immunogenic CD8+ T cell responses.
VB10.NEO in combination with NKTR-214 and anti-PD-1 induce rapid, complete and durable tumor regression of small tumors and long-lasting stabilization of large tumors supporting the rationale for examining the combination clinically.
In September 2018, Nektar and Vaccibody entered into a clinical collaboration to evaluate bempegaldesleukin in combination with VB10.NEO. A pilot study evaluating the combination in patients with squamous cell carcinoma of the head and neck is planned to begin mid-2019.

About VB10.NEO
VB10.NEO, is a proprietary therapeutic DNA vaccine which uses the patient’s own neoantigens for the personalized treatment of cancer patients. A phase I/IIa neoantigen clinical trial is currently enrolling patients with locally advanced or metastatic melanoma, non-small cell lung carcinoma, clear renal cell carcinoma as well as urothelial cancer or squamous cell carcinoma of the head and neck.

About bempegaldesleukin (NKTR-214)
Bempegaldesleukin is an investigational, first-in-class, CD122-preferential IL-2 pathway agonist designed to provide rapid activation and proliferation of cancer-killing immune cells, known as CD8+ effector T cells and natural killer (NK) cells, without over activating the immune system. Bempegaldesleukin stimulates these cancer-killing immune cells in the body by targeting CD122 receptors found on the surface of these immune cells. CD122, which is also known as the Interleukin-2 receptor beta subunit, is a key signaling receptor that is known to increase proliferation of these effector T cells.1 In clinical and preclinical studies, treatment with bempegaldesleukin resulted in expansion of these cells and mobilization into the tumor micro-environment.2,3 Bempegaldesleukin has an antibody-like dosing regimen similar to the existing checkpoint inhibitor class of approved medicines.

On April 1, 2019 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical stage targeted oncology company, reported that it will present at the H.C. Wainwright & Co. Global Life Sciences Conference in London, England on Monday, April 8th at 11:30am BST/6:30am ET/3:30am PT. Christopher C. LeMasters, Chief Business Officer, will provide a corporate update at the conference (Press release, Mirati, APR 1, 2019, Mirati Therapeutics To Present At The H.C. Wainwright & Co. Global Life Sciences Conference [SID1234534859]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentations will be webcast and made available through the "Investors" section of www.mirati.com, and replays will be made available for 90 days following the events.

On April 1, 2019 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will host a conference call on Tuesday, April 2nd at 8:00 a.m. Eastern Time to provide a clinical development update (Press release, Sangamo Therapeutics, APR 1, 2019, View Source [SID1234534858]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The conference call will be webcast live and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 6063108. For those unable to listen in at the designated time, a conference call replay will be available for one week following the call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 6063108.