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.

On May 14, 2018 ERYTECH Pharma (Euronext: ERYP – Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported its financial results for the quarter ended March 31, 2018 (Press release, ERYtech Pharma, MAY 14, 2018, View Source [SID1234526581]).

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"Following our successful Nasdaq listing at the end of last year, the first quarter of this year has been focused on the execution of the plan to advance our pipeline into solid tumor indications," said Gil Beyen, chief executive officer at ERYTECH. "Set up activities are on track for the launch of a pivotal Phase 3 trial in second line pancreatic cancer and a Phase 2 trial in first line triple-negative breast cancer. We are expanding our teams and increasing our manufacturing capacity in Europe and the U.S. Furthermore, we will meet with the FDA to discuss our ALL program and await the CHMP’s feedback on our MAA submission for relapsed and refractory ALL. We look forward to providing updates later this year."

Business Highlights

Feedback was obtained from the Commission for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA) on the design of the proposed Phase 3 trial with eryaspase in second line pancreatic cancer, confirming earlier feedback by the U.S. Food and Drug Administration (FDA). The proposed Phase 3 trial will evaluate eryaspase in combination with standard chemotherapy, compared to standard chemotherapy alone, in approximately 500 patients in the United States and Europe. The primary endpoint will be overall survival (OS). An interim analysis is foreseen when approximately two-thirds of events have occurred. Set up activities for this pivotal Phase 3 clinical trial are ongoing. Enrollment of the first patient is expected in the third quarter of 2018.
Metastatic triple-negative breast cancer (TNBC) was selected as the next solid tumor indication for the development of eryaspase. TNBC is an aggressive and metabolically active form of breast cancer for which limited treatment options are available. The planned proof of concept Phase 2 trial will evaluate eryaspase in combination with standard chemotherapy, compared to standard chemotherapy alone, in approximately 60 previously untreated patients in Europe and the United States. An interim analysis is foreseen. The primary endpoint will be objective response rate. Set up activities are ongoing and start of patient enrollment is expected in the third quarter of 2018.
ERYTECH is planning to expand the clinical development of eryaspase to first-line pancreatic cancer, as well as to other solid tumor indications. Program updates are expected later in 2018 and early 2019.
Full results from the U.S. Phase 1 trial evaluating eryaspase in combination with chemotherapy for the treatment of first line adult ALL were presented at the annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The data showed eryaspase was well tolerated. Based on the pharmaco-kinetic data and the safety findings, the recommended dose for further clinical development was determined to be 100 U/kg. A meeting with the FDA to discuss the next steps in ALL in the United States is upcoming, based on which we expect to be able to provide feedback during the third quarter of 2018.
Additionally, the Company also presented pre-clinical data on the combination of eryaspase and erymethionase, methionine-gamma-lyase encapsulated in red blood cells. The data in this study suggested that this combination could be promising in vitro and in vivo in a gastric cancer model with tumor growth inhibition in-vivo and decreased tumor cell viability in vitro.
Earlier today, the Company announced the expansion of its executive management team with the addition of Alex Dusek as VP of Commercial Strategy, to lay the groundwork for commercial success and ensure commercial product preparedness, primarily in the U.S. He brings over 25 years of experience including commercial strategic roles at Argos Therapeutics, Bayer, and United Therapeutics.
Financial Highlights

Net loss for the three-month period ended March 31, 2018 was €11.7 million, compared to €6.5 million in the same period of 2017. The €5.2 million increase was primarily attributable to:
An increase in R&D expenses by €1.9 million, related to the Company’s intensified clinical and regulatory activities, as well as the additional staffing for preclinical research and clinical development.
An increase in G&A expenses by €0.8 million, resulting from continued infrastructure developments in line with the Company’s growth.
The accounting of a €2.5 million financial loss, as the Company’s cash position denominated in euros was impacted by the negative currency exchange variation in the period of the U.S. dollar against the euro.
As of March 31, 2018, ERYTECH had cash and cash equivalents totaling €171.8 million, compared with €185.5 million as of December 31, 2017. The €13.8 million decrease in total cash and cash equivalents in the three-month period comprised a total net cash utilization of €11.2 million for operating, investing and financing activities, and a €2.6 million negative foreign exchange impact on the Company’s cash position denominated in U.S. dollars. This financial accounting loss had no real cash impact, as the U.S. dollar position is kept in that currency for U.S. dollar disbursements.
Key Upcoming Milestones Expected in 2018

Meeting with the FDA to discuss next steps in ALL
Launch of a pivotal Phase 3 clinical trial in second-line pancreatic cancer in Europe and the United States
Launch of a Phase 2 proof-of-concept clinical trial in TNBC
CHMP opinion on MAA resubmission for GRASPA in R/R ALL
Initiation of a Phase 2 proof-of-concept clinical trial in first-line pancreatic cancer
Initiation of Phase 1 clinical trial with erymethionase
First Quarter Results 2018 Conference Call Details

As a reminder, ERYTECH management will hold a conference call and webcast on Tuesday, May 15th, 2018 at 02:30pm CET / 08:30am EDT to discuss business highlights and financial results for the first quarter of 2018. Gil Beyen, Chairman and CEO, Eric Soyer, CFO and COO and Iman El-Hariry, CMO will deliver a brief presentation, followed by a Q&A session.

The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 3498017#

USA/Canada: +1 833 818 6807

United-Kingdom: +44 2031070289

Switzerland: +41 445802606

Germany: +49 6922224728

France: +33 176748988

Belgium: +32 24003547

Sweden: +46 856619361

Finland: +358 972519310

Netherlands: +31 207075547

Spain: +34 914142503

The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 800 585 8367, Conference ID: 3498017#

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

2018 Financial Calendar:

General Assembly Meeting of Shareholders: Friday, June 22, 2018 at 10:00am CET in Paris
Quarterly financial updates:
Business Update and Financial Highlights for the 2nd quarter and first-half of 2018: September 17, 2018 (after U.S. market close), followed by a conference call and webcast on September 18, 2018 (2:30pm CET/8:30am ET)
Business Update and Financial Highlights for the 3rd quarter of 2018: November 12, 2018 (after U.S. market close), followed by a conference call and webcast on November 13, 2018 (2:30pm CET/8:30am ET)
Upcoming Investor Conferences:

BioEquity Europe 2018, May 16, Ghent
Gilbert Dupont Annual Healthcare Conference, May 29, Paris
Jefferies 2018 Global Healthcare Conference, June 5-6, New York
Journée Valeurs Moyennes SFAF, June 20, Paris
JMP Life Sciences Conference, June 20, New York
European Midcap Event – Spring, June 26-27, Paris