On February 28, 2019 Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, reported fourth quarter and full year 2018 financial results and recent business highlights (Press release, Sangamo Therapeutics, FEB 28, 2019, View Source [SID1234533806]).

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Recent Highlights

ST-920 IND acceptance: The U.S. Food and Drug Administration (FDA) accepted the Investigational New Drug application (IND) for ST-920, a gene therapy candidate being evaluated for the treatment of adults with Fabry disease. Sangamo expects to initiate the Phase 1/2 clinical trial evaluating ST-920 later this year.

WORLDSymposium presentations: At the WORLDSymposium earlier in February, Sangamo presented interim results from the Phase 1/2 CHAMPIONS and EMPOWERS studies evaluating SB-913 and SB-318, zinc finger nuclease (ZFN) in vivo genome editing product candidates for the treatment of Mucopolysaccharidosis Type II (MPS II) and MPS I, respectively. Sangamo believes data from these two studies provide complementary evidence supportive of a favorable safety profile and of the activity of the ZFN in vivo genome editing approach used in both SB-913 and SB-318.

EVP of R&D appointment: Sangamo appointed Adrian Woolfson, BM BCh, PhD, as Executive Vice President of Research and Development.

Completion of acquisition of TxCell: In the fourth quarter of 2018, Sangamo completed the acquisition of TxCell, SA. The acquisition positions Sangamo as a leader in the development of CAR-Tregs, which the Company plans to evaluate for solid organ transplant rejection and for autoimmune diseases.

"The enrollment of patients last year into our five active clinical trials has laid the foundation for a steady flow of data readouts in 2019," said Sandy Macrae, CEO of Sangamo. "In the remainder of the year, we anticipate providing important data that will help us understand the potential clinical benefit of our MPS I and MPS II programs, as well as clinical results and analyses from three clinical hematology assets for hemophilia A, hemophilia B and beta thalassemia. We also expect four additional programs to progress into clinical development, including our wholly owned gene therapy for Fabry disease and CAR-Treg therapy for the prevention of solid organ transplant rejection, as well as partnered programs in sickle cell disease and oncology being developed in collaboration with Sanofi and Kite, respectively. I’m excited for what lies ahead as we continue to push forward the development of our genomic medicines."

Anticipated Milestones in 2019

In Vivo Genome Editing

SB-913: Nine patients are enrolled in the CHAMPIONS Study evaluating SB-913 for MPS II, including three patients who recently entered the study in the expanded high-dose cohort. In 2019, Sangamo expects to report longer-term safety and biochemical measurements, as well as analyses of liver biopsies and enzyme replacement therapy withdrawal experience.

SB-318: Three patients are enrolled in the EMPOWERS Study evaluating SB-318 for MPS I. In 2019, Sangamo expects to report longer-term safety and biochemical measurements, as well as analyses of liver biopsies and enzyme replacement therapy withdrawal experience.

SB-FIX: One patient is currently enrolled in the FIXtendz Study evaluating SB-FIX for hemophilia B. Sangamo expects to enroll a second subject in the study and later in 2019 to report data including safety and factor expression levels.

Second-generation reagents for in vivo genome editing platform: Sangamo has developed second-generation albumin locus ZFN constructs for potential use in the ongoing in vivo genome editing development programs. The Company plans to initiate a clinical trial this year using these second-generation ZFNs that should enable a Phase 3 decision for the MPS II program in 2020.

Gene Therapy

SB-525: Eight patients are enrolled in the Alta Study evaluating SB-525 gene therapy for hemophilia A, being developed in collaboration with Pfizer. In 2019, Sangamo expects to report data including safety and factor expression levels, as well as information regarding factor replacement use and bleeding events.

ST-920: In 2019, Sangamo anticipates activating sites for the Phase 1/2 clinical trial evaluating ST-920 gene therapy for the treatment of Fabry disease.

Ex Vivo Cell Therapy

ST-400 and BIVV003: ST-400 and BIVV003 are gene-edited cell therapies being developed in collaboration with Sanofi for the treatment of beta thalassemia and sickle cell disease. Sangamo has enrolled two patients in the Thales Study evaluating ST-400 for the treatment of beta thalassemia and in 2019 expects to report initial safety and efficacy data including levels of fetal hemoglobin and total hemoglobin. BIVV003 is being evaluated for the treatment of sickle cell disease in the Phase 1/2 PRECIZN-1 trial run by Sanofi.

TX200: In 2019, Sangamo anticipates filing a clinical trial application in Europe for TX200, an autologous CAR-Treg cell therapy for the prevention of solid organ transplant rejection. The Company expects to activate clinical sites by year-end.

KITE-037: Kite, a Gilead Company, has announced the intention to file an IND for KITE-037, an allogeneic anti-CD19 CAR-T cell therapy, in the latter half of 2019.

Fourth Quarter 2018 Financial Results

For the fourth quarter ended December 31, 2018, Sangamo reported a consolidated net loss of $18.7 million, or $0.18 per share, compared to a net loss of $13.1 million, or $0.15 per share, for the same period in 2017. As of December 31, 2018, the Company had cash, cash equivalents, marketable securities and interest receivable of $400.5 million.

Revenues for the fourth quarter ended December 31, 2018 were $26.8 million, compared to $13.1 million for the same period in 2017. The increase came primarily from approximately $11.5 million in revenues related to the collaboration agreements with Pfizer for hemophilia A, and $9.0 million in revenues related to the collaboration with Kite, a Gilead Company, which included reimbursement of $2.7 million in research services. Fourth quarter 2018 revenues were primarily generated from Sangamo’s collaboration agreements with Pfizer, Kite, Sanofi, and Dow Agrosciences.

Total operating expenses for the fourth quarter ended December 31, 2018 were $47.6 million, compared to $26.8 million for the same period in 2017. Research and development expenses were $33.3 million for the fourth quarter of 2018, compared to $19.4 million for the same period in 2017. The increase was primarily due to manufacturing and clinical trial expenses related to the progress of the Company’s clinical development programs. General and administrative expenses were $14.4 million for the fourth quarter of 2018, compared to $7.5 million for the same period in 2017. This increase was primarily due to increased headcount in general support of growth for the Company’s pipeline and clinical programs.

Full Year 2018 Results

For the year ended December 31, 2018, the consolidated net loss was $68.3 million, or $0.70 per share, compared to a consolidated net loss of $54.6 million, or $0.70 per share, for the year ended December 31, 2017. Revenues were $84.5 million for the year ended December 31, 2018, compared to $36.6 million for the same period in 2017. The increase in revenues was primarily related to our collaboration and license agreements with Kite and Pfizer. Total operating expenses were $161.6 million for the year ended December 31, 2018, compared to $92.9 million for the same period in 2017. The increase in operating expenses was primarily related to overall Company growth and manufacturing and R&D expenses related to the advancement of Sangamo’s therapeutic pipeline.

Financial Guidance for 2019

Operating Expense: Sangamo expects operating expense of $210 to 220 million for the year ending December 31, 2019.

Cash and Investments: Sangamo projects that current cash, cash equivalents, marketable securities and interest receivable position should last at least two years.

Conference Call

Sangamo will host a conference call today, February 28, 2019, at 5:00 p.m. Eastern Time, which will be open to the public. The call will also 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 6875578. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on February 28, 2019 to 11:59 p.m. ET on March 7, 2019. 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 6875578.

On February 28, 2019 Sensei Biotherapeutics, Inc., a clinical-stage biopharmaceutical company developing precision immuno-oncology therapies, reported that it will present novel preclinical data on SNS-723, a first-in-class CAR-T cell therapy and additional Phase 1 data on the long-term effects of SNS-301, a first-in-class cancer immunotherapy, both targeting a novel tumor specific embryonic antigen, human aspartate β-hydroxylase (ASPH), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta, Georgia March 29 – April 3, 2019 (Press release, Sensei Biotherapeutics, FEB 28, 2019, View Source [SID1234533832]).

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Poster Session Details:

Poster Title: CAR-T cell therapies targeting aspartyl β-hydroxylase (ASPH)
Session Date and Time: Monday, April 1: 1:00-5:00pm ET
Session Title: Adoptive Cell Therapy 2
Location: Exhibit Hall B, Poster Section 22, Poster Board 5
Abstract Number: 2306

Poster Title: Long-term immunogenicity results from a first-in-human study evaluating the anti-ASPH cancer vaccine, SNS-301
Session Date and Time: Monday April 1: 8:00am – 12:00pm ET
Session Title: Cancer Vaccines and Intratumoral Immunomodulation
Location: Exhibit Hall B, Poster Section 22, Poster Board 9
Abstract Number: 1454

About SNS-723

SNS-723 is a first-in-class CAR-T cell therapy that is currently in preclinical development targeting human aspartate β-hydroxylase (ASPH), a cell surface enzyme that is normally expressed during fetal development. The recognition domain of the CAR is the scFv portion of a high affinity anti-ASPH mAb. SNS-723 is designed to overcome one of the major hurdles in T-cell therapy, the identification of antigens that permit effective targeting of tumors in the absence of non-tolerable and/or off-target toxicities to essential tissues and organs. Experiments to further characterize ASPH-targeted CAR-T cells are ongoing with the goal of moving these promising therapeutics into clinic.

About SNS-301

SNS-301 is a first-in-class cancer immunotherapy targeting human aspartate β-hydroxylase (ASPH), a cell surface enzyme that is normally expressed during fetal development. Following fetal development, the protein is no longer expressed. Expression of ASPH is uniquely upregulated in more than 20 different cancer types and promotes cancer cell growth, cell motility and invasiveness. ASPH expression levels in various tumors are inversely correlated with tumor resistance and patient survival. Through enhanced antigen presentation and other engineered immunotherapeutic features, SNS-301 is designed to overcome self- tolerance and induce robust and durable ASPH-specific humoral and cellular immune responses. SNS-301 is paired with a companion diagnostic to select antigen-positive patients and is delivered intradermally for ease of administration.

On February 28, 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 its financial and operational results for the year ended December 31, 2018 (Press release, Sierra Oncology, FEB 28, 2019, View Source [SID1234533856]).

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"In 2018, with our opportunistic acquisition of the Phase 3 asset, momelotinib, we transformed Sierra into a late-stage company developing a diverse portfolio of promising drug candidates. We are currently advancing discussions with regulators to determine the registration path for momelotinib and expect to report next steps in the first half of 2019. Our registration strategy envisions conducting one additional Phase 3 trial in second line myelofibrosis patients, a population with significant unmet medical needs, in order to recapitulate the meaningful clinical benefits observed in two previously completed Phase 3 trials," said Dr. Nick Glover, President and CEO of Sierra Oncology. "We have also enrolled a substantial number of patients into the two ongoing trials for our oral Chk1 inhibitor, SRA737, and remain on track to report clinical data from these studies in the first half of 2019. In addition, our Cdc7 inhibitor, SRA141, recently delivered compelling preclinical efficacy data and has been cleared by the FDA to begin clinical trials."

2018 Highlights:

Momelotinib:

In August 2018, we acquired momelotinib, a potent, selective and orally-bioavailable JAK1, JAK2 and ACVR1 inhibitor. Momelotinib has been investigated in two completed Phase 3 trials for the treatment of myelofibrosis and has demonstrated a potentially differentiated therapeutic profile encompassing anemia-related clinical benefits, as well as achieving substantive splenic volume reduction and constitutional symptom control.

In October 2018, we hosted a Key Opinion Leader call featuring a presentation by distinguished medical oncologist Dr. Srdan Verstovsek, MD, PhD, Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, Houston, Texas, who discussed momelotinib and its potential to address unmet medical needs in myelofibrosis.

In December 2018, we reported aggregated transfusion independence responses from more than 150 transfusion dependent myelofibrosis patients demonstrating robust and consistent response rates within and across the two completed SIMPLIFY Phase 3 clinical trials and a translational biology study in transfusion dependent patients with myelofibrosis. More than 44% of these patients became transfusion free for at least 12 weeks and nearly 50% were transfusion independent for at least 8 weeks. Data from the latter study were also presented in a poster at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in San Diego, California.

SRA737:

During 2018, we continued to enroll patients into the two ongoing trials for SRA737, our potent, highly selective, orally bioavailable small molecule inhibitor of Chk1. Supported by emerging clinical validation for Chk1 inhibition in high grade serous ovarian cancer (HGSOC), we prioritized enrollment of genetically defined HGSOC patients into the SRA737 monotherapy trial, while continuing to enroll patients into the trial’s other indications. The SRA737+LDG (low dose gemcitabine) trial which has been enrolling across four indications, was also modified to prioritize for the enrollment of genetically defined HGSOC patients. In April 2018, we presented preclinical data in a late-breaking poster at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2018 Annual Meeting in Chicago demonstrating that SRA737 is active in both poly ADP-ribose (PARP) inhibitor resistant and CCNE1 amplified HGSOC. In November 2018, we reported preclinical data in a poster presented at the 30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium in Dublin, Ireland demonstrating that SRA737 has impressive efficacy in highly aggressive models of ovarian cancer (CCNE1-amplified and MYCN-overexpressing HGSOC patient-derived xenograft models).

During 2018, we prepared for a potential Phase 1b/2 combination trial of SRA737 with the PARP inhibitor niraparib, in subjects with metastatic castration-resistant prostate cancer. In February 2018, we signed a supply agreement with Janssen Research & Development, LLC where they will supply niraparib for the study. We are currently evaluating the optimal timing to commence this trial within the context of our recently expanded portfolio. In April 2018, we presented supportive preclinical data at the AACR (Free AACR Whitepaper) 2018 Annual Meeting in Chicago demonstrating that SRA737 synergizes with niraparib to kill carcinoma cells via multiple cell death pathways.

In November 2018, we reported preclinical data for SRA737 combined with Immunotherapy in a poster presented at the AACR (Free AACR Whitepaper) Conference on Tumor Immunology and Immunotherapy in Miami Beach, Florida. SRA737 activated the innate immune signaling STING pathway and demonstrated significant anti-tumor activity in an immunocompetent preclinical model of small cell lung cancer (SCLC).

SRA141:

During 2018, we successfully completed the Investigational New Drug Application (IND) filing process with the U.S. Food and Drug Administration (FDA) for our novel oral Cdc7 inhibitor, SRA141, and have prepared for a potential Phase 1/2 trial of the drug candidate in patients with advanced colorectal cancer. We are currently evaluating the optimal timing to commence this trial within the context of our recently expanded portfolio.

In November 2018, we reported preclinical data for SRA141 in a poster presented at the 30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium held in Dublin, Ireland, demonstrating that SRA141 potently and selectively inhibits Cdc7, thereby interfering with DNA replication and cell cycle dynamics within tumor cells while sparing normal cells. This unique mechanism results in significant in vitro anti-proliferative activity in a broad spectrum of tumor cell lines derived from both solid and hematologic cancers and translates into robust efficacy and tumor regressions in rodent xenograft cancer models.

Year End 2018 Financial Results (all amounts reported in U.S. currency)

Research and development expenses were $41.1 million for the year ended December 31, 2018, compared to $30.2 million for the year ended December 31, 2017. The increase was primarily due to an increase of $7.2 million in clinical trial costs mainly related to SRA737, a $3.0 million upfront fee paid to Gilead for the acquisition of our lead product candidate momelotinib and a $3.0 million increase in personnel-related and allocated overhead costs, partially offset by decreases of $1.9 million in third-party manufacturing costs related to SRA737 and SRA141 and $0.4 million in research, preclinical and other support costs. Research and development expenses included non-cash stock-based compensation of $4.5 million and $4.0 million for the year ended December 31, 2018 and 2017, respectively.

General and administrative expenses were $14.3 million for the year ended December 31, 2018, compared to $12.5 million for the year ended December 31, 2017. This increase was primarily due to a $1.7 million increase in personnel-related costs, professional fees and allocated overhead and a $0.2 million increase in business development costs. General and administrative expenses included non-cash stock-based compensation of $2.3 million and $1.9 million for the years ended December 31, 2018 and 2017, respectively.

For the year ended December 31, 2018, Sierra incurred a net loss of $53.3 million, compared to a net loss of $42.0 million for the year ended December 31, 2017.

Cash and cash equivalents totaled $106.0 million as of December 31, 2018, compared to $100.3 million as of December 31, 2017. At December 31, 2018, there were 74,365,965 shares of common stock issued and outstanding, with another 10,577,941 issuable upon exercise of stock options and warrants, and a term loan of $4.9 million.

On February 28, 2019 Geron Corporation (Nasdaq: GERN) reported that it will release its fourth quarter and full year 2018 financial results after the market closes on Thursday, March 7, 2019 via press release, which will be available on the Company’s website at www.geron.com/investors (Press release, Geron, FEB 28, 2019, View Source [SID1234533872]). Geron will host a conference call to discuss the financial results as well as recent events at 4:30 p.m. ET the same day.

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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

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Participants may access the conference call live via telephone by dialing domestically +1 (877) 303-9139 or internationally +1 (760) 536-5195. The passcode is 6771719. Participants are advised to dial in at least 10 minutes prior to minimize any delay in joining the call. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

On February 28, 2019 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported an upcoming poster presentation at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place from March 29 – April 3, 2019 in Atlanta, GA (Press release, Bio-Path Holdings, FEB 28, 2019, View Source [SID1234533906]).

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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

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Details for the poster presentation are as follows:

Date: Wednesday, April 3, 2019
Presentation Time: 8:00 am – 12:00 pm Eastern Time
Location: Georgia World Congress Center, Exhibit Hall B
Session: Gene- and Vector-Based Therapy, Poster Section 12
Abstract: 4786
Title: "BP1003, A Novel Liposome-Incorporated STAT3 Antisense Oligodeoxynucleotide Inhibitor"