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Galectin Therapeutics Reports Q2 2019 Financial Results and Provides Business Update

On August 9, 2019 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results and provided a business update for the three months ended June 30, 2019 (Press release, Galectin Therapeutics, AUG 9, 2019, View Source [SID1234538519]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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Harold H. Shlevin, Ph.D., President and Chief Executive Officer of Galectin Therapeutics, said, "Over the past several months we have made significant progress advancing our proprietary compound, belapectin (GR-MD-02) for a Phase 3 trial. After having raised approximately $44.9 million in our Rights Offering and selecting a leading global CRO with deep experience in our therapeutic area and operations in over 60 countries as our clinical research organization (CRO), we recently submitted our Phase 3 clinical trial protocols to the FDA for comment along with a draft Phase 4 synopsis and statistical analysis plan. In addition, we announced that Dr. Naga Chalasani and Dr. Stephen Harrison, key NASH opinion leaders, who dedicated considerable effort in the design of the study, have been designated as co-primary investigators in the Phase 3 NASH-RX clinical trial. Consequently, we believe all of the elements to commence this important study are solidly in place. Belapectin is the first drug to show positive results in a clinical trial in patients with compensated NASH cirrhosis without esophageal varices, and it is with great optimism that we anticipate a fourth quarter launch of this very important trial."

The NASH-RX trial is designed as an international, multicenter, randomized, placebo-controlled, double-blind, parallel-group, Phase 3 study with approximately 500 patients at up to 128 sites in 11 countries in North America, Europe, Asia, and Australia. The study is designed to evaluate the safety and efficacy of two doses of belapectin for the treatment of compensated non-alcoholic steatohepatitis (NASH) cirrhosis with clinical evidence of clinically significant portal hypertension without esophageal varices. Enrollment is expected to commence in the fourth quarter of 2019 with an estimated 12-14 months to achieve full enrollment. The treatment period for Phase 3 is two years, and topline data readout is expected around the end of 2022.

"With the continued support of the medical, patient, and investment communities we are excited to be advancing this new drug toward treating the millions of people globally with NASH cirrhosis."

Richard E. Uihlein, Chairman of the Board, added, "The success of the Rights Offering, the support of Drs. Chalasani and Harrison, execution of a start-up agreement with our CRO to accelerate time to patient enrollment, and the commitment and dedication of our employees provide Galectin with a world-class team that can ensure the upcoming clinical trials are rigorously administered. We are all looking forward to the launch of this trial in the fourth quarter and the data it will provide about the important role belapectin may play in helping those suffering from NASH cirrhosis."

Summary of Key Development Programs and Updates

Submitted our Phase 3 clinical trial protocol for using belapectin (GR-MD-02) for the treatment of compensated NASH cirrhosis in patients without esophageal varices for assessment by the FDA. Also, submitted to the FDA our complete clinical development plan, a draft Phase 4 synopsis, and statistical analysis plan among other documents in response to FDA’s questions from our February 2019 meeting.

Selected a leading global CRO with deep experience in NASH cirrhosis as our partner in the planned Phase 3 NASH-RX clinical trial and executed a start-up agreement.

Welcomed Drs. Harrison and Chalasani as Co-Principal Investigators, both of whom have been actively involved in the design of these upcoming trials and believe that this study could further the understanding of NASH and the role Galectin-3 inhibition may play in the treatment of this growing epidemic.

Raised $44.9 million in the Rights Offering and $2.5 million from a common stock warrant exercise by our chairman, Richard E. Uihlein. The Rights Offering, priced at $4.28 per share, resulted in the issuance of approximately 10.5 million shares of the Company’s common stock and stock purchase warrants for approximately 2.6 million shares at $7.00 per share which expire seven years after issuance. As a result, the Company now has approximately 56.6 million shares of common stock issued and outstanding.

Scientific Presentations and Conferences

Eliezer Zomer, Vice President, will present at the 3rd Annual Anti-Fibrotic Drug Development Summit (AFDD) on November 19, 2019, in Cambridge, Massachusetts. Dr. Zomer’s presentation titled "Therapeutic Integrin Inhibition," will discuss the next generation of Galectin-3 inhibitors, as well as the discovery of functional allosteric inhibitors as part of efforts of Galectin Sciences LLC, our majority owned subsidiary.

Financial Results

For the three months ended June 30, 2019, the Company reported a net loss applicable to common stockholders of $3.1 million, or $0.06 per share, compared to a net loss applicable to common stockholders of $4.1 million, or $0.11 per share, for the three months ended June 30, 2018. The decrease was primarily due to lower preclinical, clinical and non-cash stock-based compensation expenses in the current period compared to the prior year period.

Research and development expense for the three months ended June 30, 2019, was $1.5 million compared with $1.5 million for the three months ended June 30, 2018. There was an increase of about $0.6 million in clinical development expenses which was offset by a similar amount of decrease in non-cash stock-based compensation expense. General and administrative expense for the three months ended June 30, 2019, were $1.5 million, compared to $2.3 million for the three months ended June 30, 2018, primarily due to a decrease in non-cash stock-based compensation expenses.

As of June 30, 2019, the Company had $52.0 million of cash and cash equivalents. The Company also has a $10 million unsecured line of credit, under which no borrowings have been made to date, and potential additional capital under its At the Market common stock issuance agreement. The Company believes there is sufficient cash, including availability of the line of credit, to fund currently planned operations at least through December 31, 2020. The Company expects that it will require more cash to fund operations after December 31, 2020 and believes it will be able to obtain additional financing as needed. The currently planned operations include estimated costs related to a planned Phase 3 clinical trial through December 31, 2020. While the costs of the trial and general overhead during the Phase 3 trial are expected to be approximately $100 million, the costs and timing of such trial is not yet completely finalized.

Epizyme Reports Business Progress and Second Quarter 2019 Results

On August 9, 2019 Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported second quarter 2019 financial results (Press release, Epizyme, AUG 9, 2019, View Source [SID1234538518]).

"Over the course of the first half of 2019, we made tremendous progress across all aspects of Epizyme’s business, and the second half of the year holds even greater opportunities as we transition to a commercial-stage company and work to achieve our Vision 2020," said Robert Bazemore, president and chief executive officer of Epizyme. "Our vision is to bring tazemetostat to both epithelioid sarcoma and follicular lymphoma patients, while also expanding its development in the future to additional tumor types and combinations, to advance research efforts to bring EZM8266 and other early programs into the clinic, and to further enhance our leadership in the field of epigenetic drug discovery and development. The milestones we have achieved so far this year, along with the anticipated clinical and regulatory catalysts over the next several months, position this to be our most significant year yet. We look forward to continuing our progress in an effort to impact the lives of as many people as we can."

Leadership Team Expansion

Epizyme recently appointed Paolo Tombesi as chief financial officer to further support the company’s transition to a commercial-stage organization. Mr. Tombesi brings over 30 years of extensive financial and accounting expertise to Epizyme, most recently serving as the chief financial officer at Insmed, Inc.

Tazemetostat for Epithelioid Sarcoma (ES)

NDA Submission Accepted for Priority Review: The U.S. Food and Drug Administration (FDA) has accepted for filing Epizyme’s New Drug Application (NDA) for accelerated approval of tazemetostat, its lead investigational agent. The company has proposed an indication for the treatment of patients with metastatic or locally advanced epithelioid sarcoma who are not eligible for curative surgery. The FDA granted Priority Review for the NDA and has set a Prescription Drug User Fee Act (PDUFA) target action date of January 23, 2020. The submission is based primarily upon data from the 62 patient ES cohort in the company’s ongoing Phase 2 trial of tazemetostat, which were recently presented at ASCO (Free ASCO Whitepaper), and the safety and tolerability data generated across the tazemetostat development program.

Confirmatory Program Anticipated to Begin in 2H 2019: Epizyme has aligned on the design of its confirmatory trial to support full approval of tazemetostat for ES. The company plans to conduct a 1:1 randomized, controlled clinical trial in the front-line treatment setting comparing tazemetostat in combination with doxorubicin, a commonly used systemic treatment in this setting, versus placebo plus doxorubicin in approximately 150 patients. The primary efficacy endpoint will be progression-free survival, and secondary efficacy endpoints will include overall survival, disease control rate, overall response rate and duration of response. The safety run-in portion of the study is expected to begin in the second half of 2019.

Tazemetostat for Follicular Lymphoma (FL)

Planned NDA Submission for All-Comer FL on Track for Fourth Quarter: Epizyme is preparing to submit an NDA for accelerated approval of tazemetostat for the treatment of adult patients with relapsed or refractory FL, regardless of their EZH2 mutational status, who have received at least two prior systemic therapies. The company expects to submit the NDA for this indication in the fourth quarter of 2019.

Positive Updated Data from Ongoing Phase 2 Trial: At ICML, Epizyme presented data from the Phase 2 study of tazemetostat, as of a June 7, 2019 data cut off, showing that patients with an EZH2 mutation (n=43) had a 77% overall response rate (ORR) and a median duration of response (DOR) of 8.3 months. Patients with wild-type EZH2 (n=53) had a mature 34% ORR and 13-month DOR. Notably, favorable safety and tolerability were observed, with treatment-related adverse events resulting in only 5% of patients discontinuing treatment due to an adverse event. Enrollment is completed and the study is ongoing.

Confirmatory Program Anticipated to Begin in 2H 2019: To support full approval, Epizyme is planning to conduct a confirmatory program with an adaptive study evaluating the combination of tazemetostat with the chemo-free treatment regimen "R2" (Revlimid plus Rituxan) in the second line or later treatment setting for FL patients, both with and without EZH2 activating mutations. The final design is subject to alignment with FDA, and the company anticipates initiating the safety run-in portion in the second half of 2019.

FL Expansion Plans Established: Multiple additional clinical studies are anticipated to begin in the second half of 2019, and are designed to evaluate tazemetostat in earlier lines of FL treatment and in new combination regimens, including:

a combination study of tazemetostat with rituximab in patients with relapsed and/or refractory FL, and

a combination study of tazemetostat with R-CHOP in front-line patients in collaboration with the Lymphoma Study Association.

Tazemetostat for Additional Solid Tumors

Epizyme is planning several proof-of-concept clinical trials to begin in the second half of 2019 intended to explore the potential of new tazemetostat combinations in multiple solid tumor cancers, including:

a combination study of tazemetostat with the standards of care in patients with castration-resistant prostate cancer, and

a combination study of tazemetostat with a PARP inhibitor in patients with platinum-resistant solid tumors, such as small-cell lung cancer, triple-negative breast cancer and ovarian cancer.

EZM8266 for Sickle Cell Disease

Epizyme anticipates beginning clinical development of EZM8266, a novel, first-in-class G9a inhibitor, with a Phase 1 clinical trial in the second half of 2019.

Second Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $331.0 million as of June 30, 2019, as compared to $215.6 million as of June 30, 2018.

Revenue: Collaboration revenue for the second quarter of 2019 was $5.9 million, compared to $12.0 million for the second quarter of 2018. The collaboration revenue recognized in the second quarter of 2019 relates to revenue earned as part of the company’s collaboration with Boehringer Ingelheim. In the second quarter of 2018, Epizyme recognized a $12.0 million milestone under its agreement with GlaxoSmithKline.

R&D Expenses: Research and development (R&D) expenses were $40.9 million for the second quarter of 2019, compared to $31.3 million for the second quarter of 2018. The increase is primarily due to a $10.0 million development milestone paid to Eisai in connection with the submission of the NDA in the second quarter of 2019

G&A Expenses: General and administrative (G&A) expenses were $15.7 million for the second quarter of 2019, compared to $10.9 million for the second quarter of 2018. The increase is due primarily to increased pre-commercialization activities and staffing, as well as increased personnel related expenses.

Net Loss Attributed to Common Stockholders: Net loss attributable to common stockholders was $48.5 million, or $0.53 per share, for the second quarter of 2019, compared to $29.1 million, or $0.42 per share, for the second quarter of 2018.

Financial Guidance

Based on its current operating plan, Epizyme continues to expect its cash runway to extend into the first quarter of 2021.

The company will not hold a conference call in conjunction with these results.

Tagrisso significantly improves overall survival in the Phase III FLAURA trial for 1st-line EGFR-mutated non-small cell lung cancer

On August 9, 2019 AstraZeneca reported positive overall survival (OS) results from the Phase III FLAURA trial, a randomised, double-blinded, multi-centre trial of Tagrisso (osimertinib) in previously-untreated patients with locally-advanced or metastatic non-small cell lung cancer (NSCLC) whose tumours have epidermal growth factor receptor (EGFR) mutations (Press release, AstraZeneca, AUG 9, 2019, View Source [SID1234538494]).

Tagrisso showed a statistically-significant and clinically-meaningful improvement in OS, a secondary endpoint in the FLAURA Phase III trial, compared with erlotinib or gefitinib both of which were previous standard-of-care (SoC) treatments in this setting. The FLAURA trial met its primary endpoint in July 2017, showing a statistically-significant and clinically-meaningful improvement in progression-free survival (PFS), increasing the time patients lived without disease progression or death from any cause. The safety and tolerability of Tagrisso was consistent with its established profile.

José Baselga, Executive Vice President, Oncology R&D said: "Today’s positive results show that Tagrisso provides an unprecedented survival outcome versus previous standard-of-care epidermal growth factor receptor tyrosine kinase inhibitors, reaffirming Tagrisso as the 1st-line standard-of-care for EGFR-mutated metastatic non-small cell lung cancer."

AstraZeneca plans to present the OS results from the FLAURA trial at a forthcoming medical meeting.

Tagrisso is currently approved in 74 countries, including the US, Japan and the EU, for 1st-line EGFRm metastatic NSCLC.

About lung cancer

Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths, more than breast, prostate and colorectal cancers combined.1 Lung cancer is broadly split into NSCLC and small cell lung cancer (SCLC), with 80-85% classified as NSCLC.2 Approximately 10-15% of NSCLC patients in the US and Europe, and 30-40% of patients in Asia have EGFR-mutated (EGFRm) NSCLC.3-5 These patients are particularly sensitive to treatment with EGFR-tyrosine kinase inhibitors (TKI) which block the cell-signalling pathways that drive the growth of tumour cells. Approximately 25% of patients with EGFRm NSCLC have brain metastases at diagnosis, increasing to approximately 40% within two years of diagnosis.6 The presence of brain metastases often reduces median survival to less than eight months.7

About Tagrisso

Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI designed to inhibit both EGFR-sensitising and EGFR T790M-resistance mutations, with clinical activity against central nervous system metastases. Tagrisso 40mg and 80mg once-daily oral tablets have now received approval in more than 70 countries, including the US, Japan and the EU, for 1st-line EGFRm advanced NSCLC, and in more than 80 countries, including the US, Japan, China and the EU, for 2nd-line use in patients with EGFR T790M mutation-positive advanced NSCLC. Tagrisso is also being developed in the adjuvant setting (ADAURA trial), in the locally-advanced unresectable setting (LAURA), in combination with chemotherapy (FLAURA2) and with potential new medicines (SAVANNAH, ORCHARD).

About FLAURA

The FLAURA trial assessed the efficacy and safety of Tagrisso 80mg orally once daily vs. comparator EGFR-TKIs (either erlotinib [150mg orally, once daily] or gefitinib [250mg orally, once daily]) in previously-untreated patients with locally-advanced or metastatic EGFRm NSCLC. The trial was double-blinded and randomised, with 556 patients across 29 countries.

About AstraZeneca in lung cancer

AstraZeneca has a comprehensive portfolio of approved and potential new medicines in late-stage clinical development for the treatment of different forms of lung cancer spanning several stages of disease, lines of therapy and modes of action. We aim to address the unmet needs of patients with EGFR-mutated tumours as a genetic driver of disease, which occur in 10-15% of NSCLC patients in the US and EU and 30-40% of NSCLC patients in Asia, with our approved medicines Iressa (gefitinib) and Tagrisso, and ongoing Phase III trials ADAURA, LAURA, FLAURA and FLAURA2 as well as the Phase II combination trials SAVANNAH and ORCHARD.3-5

Our extensive late-stage Immuno-Oncology programme focuses on lung cancer patients without a known genetic mutation which represents up to 50% of all patients with lung cancer. Imfinzi (durvalumab), an anti-PDL1 antibody, is in development as monotherapy (Phase III trials ADJUVANT BR.31, PACIFIC-4, PACIFIC-5, and PEARL) and in combination with tremelimumab and/or chemotherapy (Phase III trials AEGEAN, PACIFIC-2, NEPTUNE, POSEIDON, ADRIATIC and CASPIAN).

About AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

InnoCure Therapeutics, Accelerating Development of Next-Generation Target Protein Degradation Mechanism New Drug

On August 8, 2019 InnoCure Therapeutics (CEO Hye-dong Yoo) reported the company is accelerating the development of new drugs using next-generation target protein degradation mechanisms (Press release, InnoCure Therapeutics, AUG 8, 2019, View Source;nid=233623 [SID1234651740]). According to the company, they are conducting research to develop treatments for rare diseases including anticancer drugs and degenerative brain diseases by utilizing next-generation new drug development technologies such as ‘PROTAC’ and ‘AUTOTAC’, artificial intelligence (AI) to shorten drug development time and increase drug efficacy, big data analysis, and molecular modeling to induce specific protein degradation mechanisms.

Based on this, InnoCure Therapeutics was recognized for its technological prowess when it was selected for the R&D project of the Ministry of SMEs and Startups’ TIPS program in February 2019, in which Magna Investment participated as the operator.

The company said that it has secured several new substances effective in the field of rare diseases at the research institute that moved to Pangyo, Gyeonggi-do in June, and is currently pursuing IND application for clinical trials in 2021. It is also pursuing joint research with pharmaceutical and bio companies specializing in degenerative brain diseases and rare diseases in the United States.

"InnoCure is actively utilizing PROTAC and AUTOTAC, next-generation technologies that can serve as ‘game changers’ in the new drug development field," said Bae Jun-hak, Vice President of Magna Investment. "The management team, which has experience in global bio companies and a global human network, possesses the know-how in global market entry strategies."

Meanwhile, PROTAC targeted protein degradation technology is known as a drug development technology that can increase efficacy and reduce side effects with small doses. Avinas, founded in 2014 by Professor Craig Crews of Yale University, a pioneer in PROTAC technology, became the first PROTAC technology company to enter clinical trials with a drug using PROTAC technology when its prostate cancer treatment under development received clinical approval from the FDA in March 2019.

InnoCure Therapeutics CEO Yoo Hye-dong is currently conducting clinical research on a drug for degenerative brain diseases that was jointly researched with a U.S. East Coast biotech company specializing in degenerative brain diseases while working at NCE Sciences, a San Francisco-based startup in the U.S. While working at Celgene, she participated in the development of new drugs, including the development of an alternative to the blockbuster nano-oncology drug ‘Abraxane.’

In January 2018, he founded Innocure Therapeutics.

Cytovation and SMS-oncology sign agreement on conduct of phase I/II trial with CyPep-1 in solid tumors

On August 8, 2019 Cytovation AS ("Cytovation") and Specialized Medical Services-oncology BV ("SMS-oncology") reported that SMS-oncology has been selected as the CRO to conduct the phase I/II clinical trial with CyPep-1 in patients with advanced solid cancers (Press release, Cytovation, AUG 8, 2019, View Source [SID1234561561]).

In recent months, Cytovation has progressed through preclinical and toxicology studies with its lead candidate CyPep-1, a first-in-class lytic agent. Through its unique pharmacological properties, CyPep-1 selectively targets and lyses tumor cell membranes based on their altered molecular composition. This mode of action kills cancer cells, releases tumor antigens, and potentially induces a tumor-specific immune response by in-situ immunization.

Cytovation is currently initiating a first-in-human trial with intratumoral injection of CyPep-1 and aims to reach first in patient (FPI) milestone by the end of 2019. An agreement is now concluded with the oncology dedicated CRO SMS-oncology in Schiphol, the Netherlands.

The study is designed as an open-label, dose escalation phase I/II trial to evaluate the safety, efficacy and pharmacokinetics of intratumoral CyPep-1 in patients with advanced solid cancers. The multicenter trial in Europe will enroll approximately 18 patients. SMS-oncology is developing the protocol and giving guidance regarding patient inclusion criteria and investigational sites for a best-fit and seamless transition to clinic. This will be followed by full conduct of the trial, expected to commence in Q4 2019.

Mr. Kjell-Inge, CEO of Cytovation: "We are excited CyPep-1 is now progressing to the next phase of development, which is an important step for our company. We are pleased with selecting SMS-oncology as our partner in this transition to clinic. From the initial discussions, they lived up to their reputation as experts in the field of early phase and immuno-oncology trials. Preparations of the trial are moving forward in a fast pace, and we feel confident our trial will be conducted in an optimal and valuable matter with our goals is mind."

Ms. Philine van den Tol, CEO of SMS-oncology: "We are delighted to support Cytovation and look forward to jointly make the CyPep-1 trial a great success. It is exactly these type of innovative projects SMS-oncology leverages great experience in, and beyond all, is passionate
about. The enthusiasm of Cytovation is catching and our team can’t wait to see results of this promising first-in-class lytic compound."

CyPep-1, in a cream formulation for topical application, is currently being evaluated in a phase I trial in patients with HPV-induced cutaneous tumors (warts).