On May 14, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ:MBIO), a Fortress Biotech (NASDAQ:FBIO) Company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (CAR-T) technology, reported financial results for the first quarter ended March 31, 2018 (Press release, Mustang Bio, MAY 14, 2018, View Source [SID1234526588]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "In the first quarter of 2018, Mustang continued to execute on our strategy of developing a portfolio of differentiated CAR-T therapies for patients with aggressive forms of cancer. We are pleased to report significant progress on the build-out of our proprietary CAR-T cell processing facility at UMass Medicine Science Park in Worcester, Mass., which is on track to be fully operational and ready to process cells by the end of the year. We are also transitioning two preclinical CAR-T programs at City of Hope into the clinic in 2018, and plan to file our first Investigational New Drug Application during the fourth quarter. In March, we were delighted to announce the promotion of Sadik Kassim, Ph.D., to Chief Scientific Officer, and Knut Niss, Ph.D., to Chief Technology Officer, and look forward to continuing to work together to innovate in cell processing and to explore opportunities to leverage best-in-class science to strengthen our CAR-T pipeline. To this end, we will expand our internal research capabilities and plan to hire a team of scientists that will be fully dedicated to preclinical and translational research efforts."

Financial Results:

As of March 31, 2018, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $55.3 million, compared to $61.5 million as of December 31, 2017, a decrease of $6.2 million for the quarter.
Research and development expenses were $4.3 million for the first quarter of 2018, compared to $0.7 million for the first quarter of 2017. Non-cash, stock-based compensation expenses included in research and development were $1.5 million for first quarter of 2018, compared to $0 million for the first quarter of 2017.
Research and development expenses from license acquisitions totaled $0.1 million for the first quarter of 2018, compared to $0.6 million for the first quarter of 2017.
General and administrative expenses were $2.1 million for the first quarter of 2018, compared to $2.0 million for the first quarter of 2017. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.5 million for the first quarter of 2018, compared to $1.2 million for the first quarter of 2017.
Net loss attributable to common stockholders was $6.3 million, or $0.24 per share, for the first quarter of 2018, compared to $3.2 million, or $0.14 per share, for the first quarter of 2017.

On May 14, 2018 Molecular Templates, Inc. (Nasdaq:MTEM) ("Molecular"), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the first quarter of 2018 (Press release, Molecular Templates, MAY 14, 2018, View Source [SID1234526587]). As of March 31, 2018, cash and cash equivalents totaled $49.3 million. Molecular’s current cash balance is expected to fund operations into late 2019.

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"We are very pleased with the clinical results we have generated to date for MT-3724, which we expect to enter Phase II studies in relapsed/refractory DLBCL patients in the second half of 2018," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "In the next twelve months, we expect our clinical pipeline to expand significantly as we file INDs for MT-4019 as well as our ETBs targeting HER2 and PD-L1."

Company Highlights and Upcoming Milestones

Corporate

At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April 2018, preclinical data were presented for Molecular’s ETBs targeting PD-L1 (which incorporates Molecular’s Antigen Seeding Technology – a differentiated immune-oncology approach) and HER2.
On March 2, 2018, Molecular closed a $10 million debt facility with Perceptive Advisors. The proceeds were used to repay an existing debt facility with Silicon Valley Bank and will support Molecular’s build out of its GMP manufacturing facility, to support Molecular’s own pipeline as well as partnerships. The first tranche of $5 million was received in 1Q18 and the second tranche of $5 million is due to be received in 3Q18.
MT-3724

MT-3724 (an ETB targeting CD20) is in an ongoing Phase Ib expansion study intended to better define the single agent overall response rate in heavily pre-treated diffuse large B-cell lymphoma (DLBCL) patients with additional updates expected in 2Q18.
Molecular also expects to initiate Phase II combination studies with MT-3724 in earlier lines of DLBCL in 2H18.
MT-4019

MT-4019 (an ETB candidate designed to target CD38-expressing myeloma cancer cells) is progressing through IND enabling studies.
Takeda and Molecular are evaluating CD38 ETBs and could potentially select a drug candidate for development by the end of 3Q18. If the two companies do not select a joint candidate for development, Molecular anticipates filing an IND application for MT-4019 in 3Q18 and initiating a Phase I clinical trial in 2H18.
Research

Molecular expects to file an IND application for an ETB targeting HER2 in 4Q18.
Molecular expects to file an IND application for an ETB targeting PD-L1 (with antigen seeding) in 1Q19.
Several other ETB candidates are in pre-clinical development, targeting both solid and hematological cancers.
Takeda Multi-Target Collaboration

In December 2017, Takeda selected two targets for further research using Molecular’s ETBs. This triggered $4 million in milestone payments, which were paid by Takeda in 2Q18.
Financial Results

The net loss attributable to common shareholders for the first quarter was $8.7 million, or $0.32 per basic and diluted share. This compares with a net loss attributable to common shareholders of $1.6 million, or $7.56 per basic and diluted share for the same period in 2017.

Revenues for the first quarter of 2018 were $0.5 million, compared to $1.9 million for the same period in 2017. Revenues for the first quarter of 2018 and 2017 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas, and revenues from collaborative research and development agreements. Total research and development expenses for the first quarter of 2018 were $6.7 million, compared with $1.1 million for the same period in 2017. Total general and administrative expenses for the first quarter of 2018 were $2.9 million, compared with $1.8 million for the same period in 2017.

On May 14, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, reported new scientific advisors in immuno-oncology and autoimmunity (Press release, Intellia Therapeutics, MAY 14, 2018, View Source [SID1234526584]). The advisors hail from prestigious international institutions and collectively have both scientific and clinical expertise in cell therapies in these areas.

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"The advisors we’ve assembled include researchers and physicians who are luminaries in their fields," said Intellia President and Chief Executive Officer John Leonard, M.D. "Pursuing both in vivo and ex vivo pipelines, Intellia has a broad spectrum approach to genome editing in a variety of therapeutic applications using our CRISPR/Cas9 technology. We look forward to working alongside these experts and benefiting from their deep experience and insight, to drive development of our wholly owned ex vivo programs in immuno-oncology and autoimmunity."

The following scientific leaders and clinicians are Intellia’s advisors on ex vivo cell therapy in immuno-oncology:

Evren Alici, M.D., Ph.D., assistant professor and group leader, hematology, Karolinska Institutet, Sweden

Chiara Bonini, M.D., Ph.D., full professor, Università Vita-Salute San Raffaele; deputy director, Division of Immunology, Transplantation and Infectious Diseases, Ospedale San Raffaele; and head, Experimental Hematology Unit, Ospedale San Raffaele, Italy

Daniel DeAngelo, M.D., Ph.D., associate professor of medicine, Harvard Medical School, and director, clinical and translational research, adult leukemia, Dana Farber Cancer Institute, United States

Saar Gill, M.D., Ph.D., assistant professor of medicine, Center for Cellular Immunotherapies, University of Pennsylvania, United States

Johanna Olweus, M.D., Ph.D., full professor and head, Department of Cancer Immunology, University of Oslo, and director, K.G. Jebsen Center for Cancer Immunotherapy, Norway

E. John Wherry, Ph.D., Richard and Barbara Schiffrin President’s Distinguished Professor of Microbiology, and director, Institute for Immunology, University of Pennsylvania, United States

Juan Carlos Zúñiga-Pflücker, Ph.D., professor and chair, Department of Immunology, University of Toronto, and senior scientist, Sunnybrook Research Institute, Canada
These experts are Intellia’s advisors on ex vivo cell therapy in autoimmunity:

Laurence Turka, M.D., chief scientific officer, Rheos Medicines; deputy director, Immune Tolerance Network; and professor of surgery and medicine (part-time), Massachusetts General Hospital and Harvard Medical School, United States

Kathryn Wood, D.Phil., F.Med.Sci., emeritus professor of immunology, Nuffield Department of Surgical Sciences, University of Oxford, United Kingdom

On May 14, 2018 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE American: IMUC) reported financial results for the first quarter ended March 31, 2018 (Press release, ImmunoCellular Therapeutics, MAY 14, 2018, View Source [SID1234526583]).

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Anthony J. Gringeri, PhD, President and Chief Executive Officer commented: "In the first quarter, we continued to make progress in advancing our Stem-to-T-Cell program. In April we announced that we had been able to verify successful transfer of the selected T cell receptor genetic material into human hematopoietic stem cells. This milestone represents the next important step in validating the Stem-to-T-Cell approach, and is a key component of the proof-of-concept work for this technology which lays the groundwork for undertaking planning for preclinical testing. From a corporate perspective, we are continuing to work with Ladenburg Thalmann & Co. Inc. as our strategic financial advisor to assist in the review of our business and assets and the exploration of strategic opportunities for enhancing stockholder value, including the potential sale or merger of the Company."

First Quarter 2018 Financial Results

For the quarter ended March 31, 2018, ImmunoCellular incurred a net loss of $1.0 million, or $0.02 per basic and diluted share, compared to a net loss of $5.9 million or $1.67 per basic and diluted share, for the quarter ended March 31, 2017. The decrease in the net loss is primarily due to the suspension of the ICT-107 phase 3 trial in June of 2017 and reductions in the Company’s other research and development programs along with reductions in general and administrative expenses.

ImmunoCellular also reported $1.6 million of cash used in operations during the most recent quarter compared to $6.1 million in the same period in 2017. The Company continues to seek favorable payment terms with its creditors and reduced its current liabilities by almost $900,000 during the quarter. No warrants were exercised in the most recent quarter; accordingly, there were no financing proceeds. There are approximately $930,000 of warrants that remain outstanding from the July 2017 financing. These warrants currently have an exercise price of $0.35 and expire in July 2018. As of March 31, 2018, the Company had approximately $5 million of cash and 41.9 million shares of common stock outstanding.

In light of previous recent updates on its research program, ImmunoCellular is not holding a conference call to discuss first quarter 2018 financial results at this time. The Company plans to provide relevant updates at an appropriate time in the future.

On May 14, 2018 Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, reported it is proceeding with plans for a Phase 3 clinical trial program with its galectin-3 inhibitor GR-MD-02 in NASH cirrhosis, incorporating advice and guidance obtained in a meeting with the US Food and Drug Administration (FDA) (Press release, Galectin Therapeutics, MAY 14, 2018, View Source [SID1234526582]).

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The target population of the Phase 3 clinical trial will be patients with NASH cirrhosis without esophageal varices. The primary endpoint will be chosen from two endpoints that the FDA agreed may be acceptable: The change in hepatic venous pressure gradient (HVPG), which is a measure of liver blood pressure, or the progression to esophageal varices. Both primary endpoints may be considered surrogate endpoints for clinical outcomes in the target population with NASH cirrhosis. Details of the phase 3 clinical trial design, including projected timings and costs, will be announced once the planning phase has been completed and the company has a final clinical trial protocol that is acceptable to the FDA.

"Planning for a Phase 3 development program represents a significant milestone for Galectin Therapeutics and a pathway forward for the development of GR-MD-02 as a potentially important therapy in patients with NASH cirrhosis," said Dr. Peter G. Traber, M.D., CEO and CMO of Galectin Therapeutics. "The basis for advancing to Phase 3 is the positive effects of GR-MD-02 on HVPG and the possible prevention or postponement of development of esophageal varices in the Phase 2 NASH-CX trial, which we believe is the first large, randomized clinical trial of any drug to demonstrate a clinically meaningful improvement in these patients. The potential choice between two primary endpoints for Phase 3 trials provides enhanced flexibility in designing the strongest trial to replicate the efficacy demonstrated in the Phase 2 NASH-CX trial. Additionally, the clinical trial design discussed with the FDA provides for interim analysis which may provide confirmation of Phase 2 results and enhanced confidence for the ultimate results of the Phase 3 trial.

"While the company believes the results of the Phase 2 NASH-CX trial may represent a breakthrough for patients with NASH cirrhosis and believes that the results meet the FDA requirements for Breakthrough Therapy designation, the FDA has not granted breakthrough designation at this time based on the Agency’s current assessment that additional confirmatory data are needed to identify the level of change in HVPG that is reasonably likely to predict clinical outcomes. Although we disagree with FDA’s decision not to grant Breakthrough Therapy designation at this time, we understand their position because our NASH-CX trial is to our knowledge indeed the first randomized clinical trial of any drug to demonstrate a clinically meaningful improvement in HVPG in NASH cirrhosis patients. Importantly, the Phase 3 program will not be impeded by the lack of Breakthrough Designation at this time. The program continues to benefit from Fast Track designation which provides many of the same advantages as Breakthrough Therapy designation, including potential for accelerated approval and priority review."

About NASH Cirrhosis

NASH cirrhosis is the final stage in the progression of non-alcoholic steatohepatitis (NASH), a disease of the liver which affects millions of people in the U.S. and worldwide. The liver cell death and inflammation seen in NASH eventually causes progressive scarring of the liver, which eventually can result in liver cirrhosis. While the early stages of NASH can be treated by changes in lifestyle, such as losing weight and exercising, once the disease progresses to NASH cirrhosis there is no treatment available short of a liver transplant. Of the total number of individuals in the world felt to presently have NASH, it is predicted that NASH cirrhosis will eventually kill 20 million of those people.

One of the results of NASH cirrhosis is an increase in blood pressure in the portal vein that brings blood and nutrients from the digestive tract through the liver and then out to the rest of the body. As the scarring effect of cirrhosis on the liver progresses, blood flow through the liver becomes more difficult, increasing the blood pressure in the portal vein, creating varying degrees of portal hypertension. Eventually, this increase in blood pressure causes the veins connected to the liver to dilate and form esophageal varices, which are dilated veins that divert blood through the esophagus, bypassing flow through the liver. These dilated veins in the esophagus are prone to bleeding, which is a major cause of morbidity and mortality in patients with NASH cirrhosis. About half of the patients with well compensated NASH cirrhosis do not have varices and identification of these patients is determined by endoscopy which is included in the standard of care for all patients with cirrhosis.

About the NASH-CX Trial

The NASH-CX trial was a randomized, double-blind, placebo-controlled Phase 2b clinical trial which enrolled 162 NASH cirrhosis patients; NASH-cirrhosis was confirmed both by liver biopsy and by confirmation of an elevated hepatic venous pressure gradient (HVPG). Enrolled patients received either 2 mg/kg or 8 mg/kg of GR-MD-02 or placebo every other week for 52 weeks, for a total of 26 doses. The aim of the NASH-CX clinical trial was to evaluate the safety and efficacy of GR-MD-02 in patients with well-compensated NASH cirrhosis. The primary study endpoint was a reduction in HVPG. Patients treated with GR-MD-02 were evaluated to determine the change in HVPG as compared to patients treated with placebo. Secondary end-points include NASH fibrosis stage and percent of fibrotic tissue based on liver biopsy and other non-invasive measures (see: www.clinicaltrials.gov for further details).

About GR-MD-02

GR-MD-02 is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of fatty liver disease and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. The drug binds to galectin-3 proteins and disrupts its function. Preclinical data in animals have shown that GR-MD-02 has robust treatment effects in reversing liver fibrosis/cirrhosis and reducing portal hypertension in cirrhosis.