On March 28, 2017 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results for the year ended December 31, 2016 (Filing, Q4/Annual, Galectin Therapeutics, 2016, MAR 28, 2017, View Source [SID1234518377]). 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.
Galectin Therapeutics management will host a conference call at 9:00 a.m. Eastern time March 28, 2017 to discuss this press release.
To access the conference call dial 844-899-6544 and provide the operator with Pin Number 89964111.
Galectin also invites all interested parties to listen to its conference call via webcast at View Source The webcast will also be available on the investor relations portion of the Company’s website at View Source The webcast will be archived on the Company’s website within two hours of the live call. The webcast will be available on the Company’s website at www.galectintherapeutics.com for 90 days.

Page 1 of 7
Management Commentary
"Galectin Therapeutics achieved a number of significant milestones in the development of our lead compound, GR-MD-02, during 2016," said Peter G. Traber, M.D., president, chief executive officer and chief medical officer of Galectin Therapeutics. "Furthermore, we’ve recently completed equity financings that have generated sufficient funding to cover currently planned expenditures through 2017.
"While non-alcoholic steatohepatitis (NASH) cirrhosis remains GR-MD-02’s primary disease target, a number of additional trials have provided encouraging early results that provide additional insight on the clinical effect of GR-MD-02. In total, what we have seen in our early trials in moderate-to-severe plaque psoriasis and severe atopic dermatitis have consistently shown that GR-MD-02 demonstrates clinically significant, biological activity in humans.
"NASH cirrhosis represents a large unmet medical need with no currently approved therapies, and we are very pleased with our progress in the NASH-CX trial. A drug that can halt progression of, or reverse existing fibrosis, in NASH cirrhosis patients would be a breakthrough therapeutic intervention that may prevent complications, alleviate the need for liver transplant, and even prevent death.
"Our Phase 2b NASH-CX clinical trial enrollment exceeded its target and, to date, 71 patients have completed all 52 weeks of infusions with GR-MD-02, and 155 patients have completed 26 weeks of infusions. More than 3,400 infusions (or 85% of the maximum infusions in the trial) have been administered, with no drug-related serious adverse reactions and a dropout rate that is below the rate included in the trial design. The top-line data readout of the NASH-CX trial remains on track for early December 2017.

"The Providence Cancer Center is continuing two investigator-initiated Phase 1 clinical trials of GR-MD-02 used in combination with approved immunotherapies, Yervoy and Keytruda in patients with advanced melanoma, oral/head and neck cancer (OHN) and non-small cell lung cancer (NSCLC). There have been no safety concerns in either of these studies. Of the five patients with advanced melanoma who underwent combination therapy with GR-MD-02 (2 mg/kg) and Keytruda, one had an impressive partial response, heading towards a complete response, and one had a mixed response.

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!

"Galectin Therapeutics also announced positive results in studies of GR-MD-02 for patients with serious skin diseases. This an encouraging new indication for GR-MD-02 and one of the previously mentioned demonstrations that the drug is biologically active in humans.

"A Phase 2, exploratory study of GR-MD-02 in patients with severe plaque psoriasis showed all five patients enrolled had significant clinical improvement (mean of 52% improvement) as measured by an objective measurement, the PASI (Psoriasis Area and Severity Index). After receiving 8 mg/kg doses of GR-MD-02 for up to 24 weeks, the fifth patient, who also had the most severe baseline disease, had an 82% PASI improvement approximately one month following the full thirteen infusions (24 weeks).

"In an investigator-initiated protocol, GR-MD-02 was also used to treat three adult patients with severe atopic dermatitis, each of whom had been recalcitrant to multiple therapies over many years. All three patients have had a marked clinical effect with near resolution of pruritus, or itching, and regression of skin lesions. Two patients achieved a 64% and 74% reduction in Eczema Area and Severity Index (EASI), after only 6 weeks and 3 drug infusions. These findings are believed to demonstrate a clinically significant effect of this novel investigational drug in this patient population."

"In summary, we believe that Galectin is in a solid position from clinical, financial and leadership perspectives," Dr. Traber concluded.

Financial Results
For the year ended December 31, 2016, the Company reported a net loss applicable to common stockholders of $22.4 million, or $0.76 per share, compared with a net loss applicable to common stockholders of $21.1 million, or $0.88 per share, for 2015. The increase is largely due to higher research and development expenses primarily related to the Phase 2 clinical program.
Research and development expense for 2016 was $15.3 million, compared with $13.1 million for 2015. The increase primarily relates to costs for the Phase 2 clinical trials begun in 2015, partially offset by lower preclinical costs.
General and administrative expense for 2016 was $6.2 million, compared with $7.0 million for 2015, primarily due to a decrease in stock based compensation.
As of December 31, 2016, the Company had $15.4 million of non-restricted cash and cash equivalents. In January and February 2017, the Company raised a total of $1.5 million in net proceeds from issuance of common stock. The Company believes it has sufficient cash to fund currently planned operations and research and development activities through December 31, 2017.

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]).

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!

Know more, wherever you are:
Latest on Asterias Biotherapeutics’ Cancer Pipeline, book your free 1stOncology demo here.

"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.

On March 28, 2017 Medical Prognosis Institute A/S (MPI.ST) (Denmark and Phoenix, AZ, USA) reported that Oncology Venture a spinout from MPI and 2-BBB Medicines BV have entered into an exclusive global license agreement on 2-BBB’s Phase 2 lead product 2B3-101 – now called 2X-111 (Press release, 2X Oncology, MAR 28, 2017, View Source [SID1234526107]). 2X-111 has demonstrated clinical activity in a phase 2 study in metastatic Breast Cancer patients and in patients with Glioblastoma (primary brain cancer) both hard to treat cancers with a huge unmet medical need. 2X-111 will be combined with its Drug Response Predictor (DRP) as a companion diagnostic in DRP(TM) focused Phase 2 trials for selected, high-likelihood responder patients. The drug will be developed as a 2X Oncology Inc. pipeline drug. 2X Oncology Inc. is a spinout of Oncology Venture developing Precision medicine for Women’s cancers. MPI has a 10% ownership in 2X Oncology.

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 active anticancer drug in 2X-111, doxorubicin, is an almost identical molecule to epirubicin, for which it was recently announced that the DRP(TM) biomarker from MPI can, with certainty, identify those patients who benefit from treatment with the drug. A retrospective-prospective validation (i.e. the highest validation level) has been achieved for the epirubicin DRP(TM), and 2X-111 therefore has a similar risk reduced profile (like LiPlaCis in the Oncology Venture pipeline).

Under the terms of the agreement, Oncology Venture will be responsible for the development and commercialization of 2X-111 in oncology. Oncology Venture will, through 2X Oncology, fund and execute the mutually agreed clinical development plan, which includes an initial phase 2 clinical study in metastatic Breast Cancer through screening of approximately 250 patients in Denmark and potentially other countries with its DRP biomarker as a predictive/companion diagnostic. MPI will on behalf of OV handle the screening.

"It is great to see OV’s ability to identify proven active drugs to in-license and develop using MPI’s technology. I believe that the cutting-edge science and compelling clinical data behind the drug (now called 2X-111) in combination with our unique Drug Response Predictor (DRP(TM)) biomarker from MPI technology gives an exceptional risk reduced opportunity to develop effective treatments for hard to treat cancers," says Adjunct Professor Peter Buhl Jensen, M.D., CEO of MPI. "MPI has ownership of 10% of spinouts from Oncology Venture and development of drugs through OV is considered as an important validation of the technology as well as an important potential income stream," Peter Buhl Jensen further commented.

About MPI’s multiple biomarker called Drug Response Predictor – DRP(TM)
MPI’s DRP(TM) is a tool for developing tumor-derived genetic signatures to predict which cancer patients are high likely to respond to a given anti-cancer product. The DRP(TM) has been tested in 37 trials, where 29 trials showed that drug-specific DRP(TM) Biomarkers could predict which patients responded well to the treatment. The DRP(TM) platform has amongst others been externally validated and published in collaboration with leading statisticians at the MD Anderson Cancer Center. The DRP(TM) method can be used to design the Clinical Development Plan, i.e. to select which indications are relevant for a given anti-cancer drug. In addition to this, the individual genetic patterns of patients can be analyzed as part of a screening procedure for a clinical trial to ensure inclusion of patients with a high likelihood of response to the drug. DRP(TM) builds on comparison between sensitive and resistant human cancer cell lines, including genomic information from cell lines combined with clinical tumor biology and clinical correlates in a systems biology network. The DRP(TM) is a Big Data tool based on messenger RNA.
The DRP(TM) platform can be used in all cancer types, and has been patented for more than 70 anti-cancer drugs in the US.

More about tumor biopsies
The pathologist evaluates biopsies to see if it is cancer. At the operational theatre, the surgeon places the biopsy in formalin and submits the specimen to the pathology department. Here a technician embeds the tumor biopsy in a paraffin block to allow the cutting of very fine slices of the specimen. The slices are put on glass slides and stained with various dyes and finally evaluated by the pathologist. The tumor in formalin and paraffin are stored. The biopsy block belongs to the patient and follows her or him if she or he changes hospital. In Scandinavia, hospitals are mostly public and patients usually stick to their local hospital. The patient and her or his clinical data can be followed over years as well as CT scans and blood tests. Biopsy blocks can be reclaimed for tests.
In MPIs and Oncology Ventures LiPlaCis study more than 1,100 patients have agreed to allow the two companies to attain material from the paraffin embedded biopsies and to measure the gene expression on the material and relate it to treatment outcome. Gene expression is a measurement of the activity of the genes. Some are very active some are not active. There are 20,000 (twenty thousand) genes in normal tissue and in cancer and modern gene expression technology allows the measurement all the genes in one step on a single chip. MPI uses the commercially available Affymetrix chip for this purpose. MPI has developed and validated a link between gene expression and the efficacy of a range of anticancer drugs. This press release informs about a validation of the method by use of biopsy material and clinical data on four anticancer agents used in daily practice for breast cancer treatment. The companies have worked together with the Danish Cooperative Group for Breast Cancer and breast cancer experts on 10 hospitals to attain the data. The study encompasses data from more than 800 patients with metastatic Breast Cancer receiving almost 2800 treatment lines or an average of 3.5 treatments per patient. Biopsies were from the original diagnosis of Breast Cancer i.e. at a time where the patient was evaluated as curable and most of the patients at then received so-called adjuvant therapy with chemotherapy, irradiation and antihormone therapy after surgery. On average the biopsies were 10 years old.

On March 28, 2017 Numab Therapeutics AG ("Numab") reported a research and option agreement with Ono Pharmaceutical Co., Ltd. ("Ono") for the identification of a multi-specific antibody candidate for development in immuno-oncology (Press release, Numab, MAR 28, 2017, View Source [SID1234525266]).

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 extremely pleased to start our collaboration with Ono, a company with a track record of scientific excellence and innovation, and that most recently made its mark by launching a new era in cancer treatment with the development of the first anti-PD-1 antibody, Nivolumab/Opdivo."

Tweet this
Under the agreement, Ono will obtain an option to acquire intellectual property rights to, and exclusive rights to develop and commercialize, the selected lead compound to be generated through this collaboration, which will exploit one of Ono’s novel therapeutic approaches in immuno-oncology. In exchange Numab will receive research funding and up to CHF 258 million in upfront and milestone payments plus tiered single to double digit royalties on sales.

Oliver Middendorp co-CEO of Numab, said: "We are extremely pleased to start our collaboration with Ono, a company with a track record of scientific excellence and innovation, and that most recently made its mark by launching a new era in cancer treatment with the development of the first anti-PD-1 antibody, Nivolumab/Opdivo."

Hiromu Habashita, Corporate Officer, and Executive Director of Discovery & Research of Ono, said: "We highly value the capabilities of Numab’s multi-specific antibody platform. It is robust, and optimized to reproducibly yield product candidates with outstanding binding properties. We believe it is ideally suited to fuel our immuno-oncology drug discovery and consequently a multi-specific antibody to be generated through this collaboration will provide hope to sufferers of cancer."