On March 29, 2018 Institut Curie and Freenome reported a strategic collaboration to evaluate Freenome’s artificial intelligence (AI) genomics platform as a novel tool to predict patient response to immuno-oncology therapies by observing changes in circulating cell-free biomarkers (Press release, Institut Curie, 29 29, 2018, View Source [SID1234525067]).

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!

In recent years so-called liquid biopsies have established the possibility of detecting circulating fragments of tumor DNA (ctDNA) and circulating tumor cells (CTCs) in the blood of patients with cancer. Institut Curie has been a pioneer in the field since the late 90’s with the first program on disseminated tumor cell (DTC) detection in the bone marrow of patients with early breast cancer. Since then, Institut Curie’s Circulating Tumor Biomarkers Laboratory has developed numerous innovative ctDNA techniques (ddPCR and NGS-based techniques).

Meanwhile, Freenome’s breakthrough use of machine learning looks beyond tumor DNA and processes the full range of cell-free (cf) biomarkers in the blood. By assembling an ever-expanding library of cell-free disease signatures, Freenome is developing a range of non-invasive blood tests for the early detection of cancer and early prediction of response to various oncology therapies. As a first step in this collaboration with Institut Curie, Freenome will analyze samples from the Analysis of Circulating Tumor Markers in the Blood (ALCINA) Trial, an umbrella trial allowing assessment of different circulating biomarkers and their correlation with clinical and pathological characteristics pertaining to response to PD-1 inhibitors alone and in combination with other therapies.

"Our machine learning scientists and molecular biologists are evaluating the cell-free genome – given 60-80%1,2,3 of cfDNA comes from immune cells – and other analytes. These provide a more complete picture of the dynamic interaction between the tumor and its environment," said Blandine Merino, VP of Business Development at Freenome. "Our approach incorporates a variety of biological signals, such as genomic, proteomic and epigenetic changes, providing new insights into possible mechanisms of resistance and guiding treatment selection for patients."

There is currently a high unmet need for biomarkers with high sensitivity and specificity for response prediction in immuno-oncology. The analysis of these data could open the way to new targets in precision oncology and improve therapeutic decision-making, particularly with the limitations associated with current approaches such as PD-L1 expression testing and assessing tumor mutational burden. Freenome’s tests would help to identify patients who are more likely to respond to PD-1 inhibitors; currently, approximately 80% of patients with advanced non-small-cell lung cancer, for example, do not respond to PD-1 inhibitors.4,5

According to Pr. François-Clement Bidard, Principal Investigator at Institut Curie, "Freenome is developing a novel approach which could revolutionize the way we analyze cell-free biomarkers for patients with cancer treated with immunotherapy; this approach complements a pipeline of innovative research projects that is ongoing at Institut Curie Circulating Tumor Biomarkers Laboratory."

Amaury Martin, Head of Institut Curie Technology Transfer and Industrial Partnerships Office and Director of the Institut Carnot Curie Cancer, added, "This agreement is one of the first at the Institut Curie with a company specializing in both cell free biomarkers and applying machine learning to large data sets. The Institut Curie MC21 strategic plan has identified innovation around liquid biopsy and big data as a major axis of medical-scientific research. With the arrival of a Data Director, Institut Curie, through its Carnot Institute, is determined to take a leading position in this key area for personalized medicine in oncology. The technologies developed by Freenome will fully benefit our patients and its access within the Institute will speed up future collaborations for developing and validating predictive tests."

About Freenome

Freenome is an AI genomics company on a mission to empower everyone with the tools they need to prevent, detect, and treat their diseases. By applying advanced machine learning techniques to recent breakthroughs in genomic science, Freenome is developing noninvasive blood tests to detect early-stage cancer and make treatments more effective. The company has raised $78 million from investors such as Andreessen Horowitz, Google Ventures, Polaris Partners, and Founders Fund.

For more information: View Source

On March 29, 2018 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results for the year ended December 31, 2017 (Press release, Galectin Therapeutics, MAR 29, 2018, View Source [SID1234525800]). These results are included in the Company’s Annual Report on Form 10-K, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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!

"In 2017 we achieve a major milestone in establishing GR-MD-02 as the first drug to show positive results in a clinical trial in patients with compensated NASH cirrhosis without esophageal varices," said Peter G. Traber, M.D., president, chief executive officer and chief medical officer of Galectin Therapeutics. "We believe this is the first randomized clinical trial of any drug to demonstrate clinically meaningful positive effects, including reducing portal hypertension, facilitating an improvement in liver cell death (a key component of NASH), and reducing the development of new esophageal varices, in this important group of patients. The drug has also always proven to be safe and well tolerated.

"In May 2018, we will be meeting with the Food and Drug Administration to present the results of our NASH-CX clinical trial. We hope to come to an agreement with the FDA on a plan for a Phase 3 clinical trial. In addition, we have submitted an application for Breakthrough Therapy Designation for GR-MD-02. We are also encouraged by the recent findings of a Phase 1 clinical trial of GR-MD-02 used in combination with pembrolizumab (KEYTRUDA) on melanoma patients, which may represent another path forward for GR-MD-02 into the significant market for evolving new cancer therapies. The positive results of the various trials conducted over the course of the past year have demonstrated that GR-MD-02 has potential in a variety of applications, from NASH to Immunotherapy and Skin Disease. In addition, through our strong intellectual property program, we are protecting the value of our asset and opening additional opportunities on a global basis. The management team at Galectin is dedicated to advancing our trials to unlock the full value of our potential."

Expected Upcoming Milestones

The Company will be meeting with the FDA in early May 2018 to present the results of its NASH-CX clinical trial. The purpose of the meeting is to seek agreement on a plan for a Phase 3 clinical trial

The Company has filed a request with the Food and Drug Administration to grant GR-MD-02 Breakthrough Therapy Designation as therapy for patients with NASH Cirrhosis without esophageal varices.

The Company has been selected to make an oral presentation at the International Liver Meeting in April 2018 in Paris. The presentation, which will be made by Dr. Naga Chalasani, one of the principal NASH-CX clinical trial investigators, is entitled, "A multicenter, randomized, double-blind, PLB-controlled trial of Galectin-3 inhibitor (GR-MD-02) in patients with NASH cirrhosis and portal hypertension."

The Company continues to remain in ongoing discussions with a number of pharmaceutical companies about potential partnerships.
Summary of Key Development Programs and Updates

The Company, in partnership with Providence Cancer Institute, presented preclinical and early clinical data from an investigator-initiated Phase 1 clinical trial of GR-MD-02 used in combination with pembrolizumab (KEYTRUDA). According to one of the principal investigators at the Providence Cancer Institute, the objective response rate of five out of eight patients (62.5%) in this trial with advanced melanoma, including two complete responses, is very encouraging and compares favorably with the known response rates with pembrolizumab alone (ORR of ~ 33%).

The company has begun enrolling cohort 3 (GR-MD-02 8 mg/kg), of the pembrolizumab, combination immunotherapy clinical trial, which will include at least 10 patients with melanoma, to provide a larger group of patients to evaluate. It is hoped additional data can be reported in mid-2018 when we anticipate a decision on progressing to phase 2.

The Company announced it has received two new patents in China and two new patents in Japan for the Company’s lead compound, GR-MD-02.

The Company announced it entered into a $10 million unsecured line of credit facility with stockholder and new director Richard E. Uihlein in December 2017.

Dr. Peter G. Traber, M.D., Chaired the Anti-Fibrotic Drug Development Summit 2017, which is dedicated to the translation of fibrotic mechanisms into clinically effective therapeutics.
Financial Results

For the year ended December 31, 2017, the Company reported a net loss applicable to common stockholders of $17.5 or $0.49 per share, compared to a net loss applicable to common stockholders of $22.4 million, or $0.76 per share, for 2016. The decrease is largely due to lower preclinical, clinical, legal, and stock-based compensation expenses.

Research and development expense for 2017 was $11.7 million, compared with $15.3 million, for 2016. The decrease primarily relates to a reduction in costs for the NASH-CX Phase 2 clinical trial as it winds down and lower preclinical costs.

General and administrative expense for 2017 was $4.5 million, compared to $6.2 million, for 2016, primarily due to a decrease in legal and stock-based compensation expenses.

As of December 31, 2017, the Company had $3.1 million of cash and cash equivalents. In December 2017, the Company entered into a $10 million line of credit and received $4.5 million in proceeds in January 2018 from common stock warrant exercises. The Company believes it has sufficient cash, including availability under the line of credit, to fund currently planned operations and research and development activities through at least March 31, 2019.

Positive Results in NASH Cirrhosis

GR-MD-02, a proprietary polysaccharide pharmaceutical preparation that inhibits galectin proteins, recently completed a Phase 2b clinical trial (NASH-CX). There were statistically significant and clinically relevant positive effects of GR-MD-02 on HVPG and other parameters in patients with NASH cirrhosis without esophageal varices following one year of therapy. Patients without esophageal varices comprise about 50 percent of the total population of patients

Page 3 of 6

with NASH cirrhosis, and is estimated to be 2.5 million people in the United States. This group of patients is readily diagnosed by endoscopy which is already part of the standard of care for patients with suspected NASH. The drug was well tolerated during this one-year trial. The Company believes that this is the first randomized clinical trial of any drug to demonstrate clinically meaningful positive effects in this important group of patients. Full details of Galectin’s NASH-CX trial can be found in a supplemental slide deck of our corporate presentation on the home page of our website.

Encouraging Results in Cancer Immunotherapy

GR-MD-02 also showed encouraging Phase 1b results in combination with pembrolizumab (KEYTRUDA) to treat advanced melanoma. In the first two cohorts of this study, five out of eight patients (62.5 percent) with advanced melanoma had objective responses, with two complete and three partial responses, which compares favorably with the known response rates with pembrolizumab alone (~33 percent). The study continues with additional results expected in Summer 2018. Full details on Galectin’s combination cancer immunotherapy program can be found in a supplemental slide deck to our corporate presentation on the home page of our website.

On March 29, 2018 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 that data from its Phase 1 study of prexigebersen (BP1001) as a treatment for haematological malignancies was published in the online edition of The Lancet Haematology in an article titled, "Liposomal Grb2 antisense oligodeoxynucleotide (BP1001) in patients with refractory or relapsed haematological malignancies: a single-centre, open-label, dose-escalation, phase 1/1b trial (Press release, Bio-Path Holdings, MAR 29, 2018, View Source [SID1234525801])."

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 single-center, open-label, dose-escalation phase 1/1b trial enrolled and treated 39 subjects (aged ≥18 years) with refractory or relapsed hematologic malignancies at MD Anderson Cancer Center in Houston. The objectives of this study were to establish the toxicity and tolerance of escalating doses of BP1001 monotherapy in patients with refractory or relapsed leukemia, to assess the maximum tolerated dose of BP1001, and to determine the optimal biologically active dose of BP1001, defined as a 50% reduction in Grb2 expression in circulating leukemia cells. The study also assessed the in-vivo pharmacokinetics of BP1001 and tumor response.

The study employed a 3+3 dose escalation strategy, with at least three patients enrolled at each dose level. BP1001 was administered intravenously, twice weekly for 28 days with a starting dose in cohort 1 of 5 mg/m², cohort 2 (10 mg/m²), cohort 3 (20 mg/m²), cohort 4 (40 mg/m²), cohort 5 (60 mg/m²), or cohort 6 (90 mg/m²). Following completion of monotherapy, the safety and toxicity of BP1001 (60 or 90 mg/m²) in combination with 20 mg low-dose cytarabine (twice-daily subcutaneous injections) was evaluated in a phase 1b study in patients with refractory or relapsed acute myeloid leukemia (i.e., those patients who were refractory to at least one previous therapy regimen and no more than one previous salvage regimen).

The study results showed that BP1001 was well tolerated, with early evidence of anti-leukaemic activity in combination with low-dose cytarabine. To further explore this anti-leukaemic activity, BP1001 in combination with low-dose cytarabine combination is being studied in an ongoing phase 2 clinical trial in patients with previously untreated acute myeloid leukaemia who are ineligible for intensive induction therapy.

In addition to the publication of the study results, the article was selected for commentary by Xavier Thomas, MD and Etienne Paubelle, MD, PhD, Department of Hematology, Lyon-Sud Hospital, Pierre Bénite, France. In their view, "Much improvement is still needed in the treatment of these high-risk patients. Because of their specific mechanism of action and their good tolerance, liposome-incorporated antisense oligodeoxynucleotides might be an effective new therapeutic strategy."

"We are delighted to have these very favorable data published in The Lancet Haematology and to have the formal commentary of Drs. Thomas and Paubelle, two recognized international hematologists," noted Peter H. Nielsen, Chief Executive Officer of Bio-Path. "We continue to enroll our Phase 2 study of prexigebersen in patients with untreated acute myeloid leukemia and these anti-leukemic data are especially encouraging."

On March 29, 2018 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage immunotherapeutics company, reported financial results for the year ended December 31, 2017 (Press release, Altimmune, MAR 29, 2018, View Source [SID1234525052]).

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!

Corporate Highlights

Enrolled first two cohorts of government funded Phase 1 trial of NasoShield, an intranasal vaccine against anthrax infection

Positive data in two Phase 2 clinical programs:

Announced positive proof-of-concept Phase 2 flu vaccine trial results with our NasoVAX vaccine

Announced positive pre-clinical data from the Company’s SparVax-L trial comparing SparVax-L and BioThrax against anthrax infection

Extended its IP protection of NasoShield in the U.S. with a Notice of Allowance from the U.S. Patent Office

Elected Mitchel Sayare, Ph.D., as Chairman of its Board of Directors

Raised approximately $30 million in financing, including through a Series B preferred offering, cash acquired in connection with the reverse merger with PharmAthene and a pre-merger private placement with existing investors, providing cash into the first quarter of 2019
"We have had a very data-rich few weeks with results being reported from our NasoVAX, HepTcell, and SparVax-L programs and moving forward on enrollment in our Phase 1 trial of NasoShield," said William J. Enright, Chief Executive Officer of Altimmune. "We are very excited by the positive results from our NasoVAX trial and look forward to continuing to advance that program. NasoVAX is a very different type of flu vaccine that has tremendous potential as an effective, easy-to-use vaccine that potentially provides better protection than current vaccines. We are also excited by the results on our SparVax-L trial and look forward to moving that program forward once we secure additional government funding. We continue to evaluate our HepTcell results will update investors on our next steps as we better understand those results."

Mr. Enright continued, "operationally, we are pleased with our progress. In 2017 we closed the reverse merger with PharmAthene, allowing us to leverage our resources and create a focused immunotherapeutics company. We strengthened our scientific team with the promotion of Dr. Sybil Tasker to Chief Medical Officer in early 2017. Additionally, in January 2018, Mitchel Sayare, Ph.D. was elected as Chairman of our Board of Directors bringing in-depth biotechnology experience as the former CEO of Immunogen. We anticipate continuing to build on our momentum in 2018 as we move forward with our NasoVAX, SparVax-L and NasoShield programs."

LOGO

Financial Results for the Year Ended December 31, 2017

Revenue for the year ended December 31, 2017 was $10.7 million compared to $3.2 million for 2016. The increase was due to $5.7 million increase in revenue from our contract with BARDA and $1.8 million revenue from the NIAID contract we assumed from our merger with PharmAthene in May 2017.

Research and development expenses were $18.4 million for the year ended December 31, 2017 compared to $7.2 million for 2016. The increase in research and development expenses was primarily the result of increases relating to NasoShield, NasoVAX, HepTcell, and SparVax-L clinical and preclinical trial costs, partially offset by $0.5 million reduced spending on the Oncosyn program. Research and development expenses for the year ended December 31, 2016 did not include PharmAthene or costs incurred under the NIAID contract.

General and administrative expenses were $8.5 million for the year ended December 31, 2017, compared to $7.1 million for 2016. The increase was the combined result of increased professional fees related to the merger with PharmAthene and costs incurred by us as a public company, including insurance costs and stock compensation expense, offset by $2.4 million of costs related to our initial public offering incurred in 2016 that did not recur in 2017.

We determined that our goodwill was impaired and a non-cash goodwill impairment charge of $35.9 million was recorded during the year ended December 31, 2017 which was classified as a component of operating expenses. The non-cash charge resulted from our goodwill assessment based on our market capitalization plus an implied control premium relative to the carrying value of our net assets. The non-cash charge has no effect on our current cash balance or operating cash flows.

We recorded an income tax benefit of $5.6 million during the year ended December 31, 2017, which reflected estimated tax refunds we expect to receive from carrying back our 2017 net operating losses to offset the 2016 federal and state income taxes paid by PharmAthene.

Net loss attributable to common stockholders for the year ended December 31, 2017 was $51.4 million compared with $11.5 million for 2016. Excluding the non-cash goodwill impairment charges, net loss attributable to common stockholders for the year ended December 31, 2017 was $15.4 million compared to $11.5 million for 2016.

Net loss per share attributable to common stockholders for the year ended December 31, 2017 was ($4.01) compared with ($1.66) for 2016. Excluding the non-cash goodwill impairment charges, net loss per share attributable to common stockholders for the year ended December 31, 2017 was ($1.21), compared to ($1.66) for 2016.

At December 31, 2017, the Company had cash, cash equivalents, and restricted cash of approximately $12.3 million, of which $3.5 million was restricted under the terms of the Series B preferred offering.

LOGO

Non-GAAP Measures

To supplement the Company’s unaudited financial statements presented in accordance with generally accepted accounting principles ("GAAP"), this press release includes a discussion of adjusted net loss attributable to common stockholders and adjusted net loss per share attributable to common stockholders, in each case adjusted for the loss due to a goodwill impairment charge. The Company believes that these non-GAAP measures, when taken into consideration with the corresponding GAAP financial measures, provide investors with meaningful comparisons of current results to prior period results by excluding items that the Company does not believe reflect its fundamental business performance. See the attached schedule for a reconciliation of net loss to adjusted net loss and loss per share to adjusted loss per share for the twelve months ended December 31, 2017 and 2016.

On March 29, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD) a clinical-stage biopharmaceutical company focused on the development of CART-cell therapies, reported that the company will present updates on its ongoing Phase I clinical trials at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held April 14–18, 2018, in Chicago (Press release, Celyad, MAR 29, 2018, View Source [SID1234532518]). Poster presentations will feature updated data from Celyad’s THINK[1] trial, the new SHRINK[2] and LINK[3] trials in metastatic colorectal cancer, as well as data from a preclinical study showing that the addition of either the CD28 or 4-1BB co-stimulatory domains to CYAD-01 construct brings no benefit in terms of in vitro activity of the receptor.

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!

Poster Presentation Details:

Poster Title: The THINK clinical trial: Preliminary evidence of clinical activity of NKG2D chimeric antigen receptor T cell therapy (CYAD-01) in acute myeloid leukemia.

Poster Number: CT129

Session Title: Phase I Trials in Progress

Session Date & Time: Tuesday, April 17, 2018, 8:00 AM – 12:00 PM CDT

Location: Mc Cormick place south, Exhibit Hall A, Poster Section 42, Poster Board 12

Download the poster

Poster Title: The SHRINK clinical trial: A phase I study assessing the safety and clinical activity of multiple doses of an NKG2D-based CAR-T therapy, CYAD-01, administered concurrently with the neoadjuvant FOLFOX treatment in patients with potentially resectable liver metastases from colorectal cancer.

Poster Number: CT123

Session Title: Phase I Trials in Progress

Session Date & Time: Tuesday, April 17, 2018, 8:00 AM – 12:00 PM CDT

Location: Mc Cormick place south, Exhibit Hall A, Poster Section 42, Poster Board 6

Download the poster

Poster Title: The LINK clinical trial: A phase I study assessing the safety and clinical activity of multiple hepatic transarterial administrations of an NKG2D-based CAR-T therapy, CYAD-01, in patients with unresectable liver metastases from colorectal cancer.

Poster Number: CT134

Session Title: Phase I Trials in Progress

Session Date & Time: Tuesday, April 17, 2018, 8:00 AM – 12:00 PM CDT

Location: Mc Cormick place south, Exhibit Hall A, Poster Section 42, Poster Board 17

Download the poster

Poster Title: NKG2D as a chimeric antigen receptor – DAP 10 provides optimal co-stimulation for NKG2D based CARs.

Session Title: Adoptive Cell Therapy 3

Poster Number: 3583

Session Date & Time: Tuesday, April 17, 2018, 8:00 AM – 12:00 PM CDT

Location: Mc Cormick place south, Exhibit Hall A, Poster Section 24, Poster Board 21