On August 7, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a focus on Myelodysplastic Syndromes (MDS), reported that the Company will release its second quarter financial results on August 14, 2019, before the market opens (Press release, Onconova, AUG 7, 2019, View Source [SID1234538269]). The Company will host a conference call on August 14, 2019, at 9 a.m. Eastern Time to discuss these results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S. or (210) 229-8823 internationally and using conference ID 9150777. The call will also be webcast live. Please click here to access the webcast on the Investor Relations page of the Company’s website. A replay will be available for 90 days.

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About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

On August 7, 2019 INmune Bio, Inc. (NASDAQ: INMB), an immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported positive preliminary data today during a presentation at Cambridge Healthtech Institute’s 7th Annual Immuno-Oncology Summit in Boston (Press release, INmune Bio, AUG 7, 2019, View Source [SID1234538303]). INB03 is being developed as part of combination immunotherapy to potentially reverse resistance to treatment.

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The Phase I trial is an open-label, dose-escalation trial in patients with advanced solid tumors. Patients received INB03, a novel, second-generation soluble TNF (tumor necrosis factor) inhibitor that works by a dominant-negative technology. Today, positive preliminary data from the first two cohorts were released. These data will be followed by a final report later this year as the company advances the program into a Phase II study.

"The goal of the Phase I study is to determine, in order of priority, the safety of INB03 in cancer patients, the dose of INB03 to take into the Phase II trials in cancer, and evidence of a biologic effect of INB03," said RJ Tesi, MD, Chief Executive Officer and Chief Medical Officer of INmune Bio. "All of these goals have been met. Using data from this trial, we have begun planning a Phase II trial using INB03 as part of combination immunotherapy in patients with cancer."

To date, 11 of 12 patients have been enrolled in one of three dosing cohorts of INB03 (0.3, 1.0 and 3.0 mg/kg). Participants were a mix of patients with advanced solid tumors that have progressed after multiple previous lines of therapy. INB03 was given once a week by subcutaneous injection. Safety, INB03 pharmacokinetics and inflammatory biomarkers were followed in all patients.

Preliminary data from patients treated in the first two cohorts are available. Patients included two males and four females, and the average age was 63-years-old. Patients in the study had prostate cancer, ovarian cancer, colon cancer (two), cholangiocarcinoma and lung cancer, with an average of three previous lines of therapy (range: two to four). INB03 was given for a median of 74 days (range: 12 to 119 days). No drug-related serious adverse events have been reported, and INB03 was well tolerated. Discontinuation of INB03 was due to tumor progression in all patients. INB03 trough drug levels were obtained before each INB03 dose. The target INB03 trough level was reached in three of three patients in the 1.0 mg/kg group. The inflammatory cytokine IL6, a biomarker of soluble TNF function, decreased by more than 50% in half of the patients, suggesting a pharmacodynamic effect of INB03.

About INB03

INB03, a novel dominant-negative TNF inhibitor, is a selective second-generation TNF inhibitor that neutralizes soluble TNF (sTNF) without blocking the function of trans-membrane TNF or TNF receptors. In animal studies, INB03 neutralization of sTNF alters the immunobiology of the tumor microenvironment (TME) to improve tumor killing by decreasing populations of cells in the TME that protect the tumor from the patient’s immune system and immunotherapy – myeloid derived suppressor cells, T regulatory cells and tumor activated macrophages. The unique mechanism of action suggests INB03 may have safety and efficacy advantages over currently approved non-selective TNF inhibitors in the treatment of cancer and other diseases.

On August 7, 2019 Biologics by McKesson, an independent specialty pharmacy for oncology and other complex therapeutic areas, reported that it was selected by Daiichi Sankyo, Inc. as the exclusive specialty pharmacy provider for TURALIO (pexidartinib) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery (Press release, McKesson, AUG 7, 2019, View Source [SID1234538337]).

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TURALIO, a first-in-class oral tyrosine kinase inhibitor for TGCT, was approved by the FDA on August 2, 2019. Surgery is the primary mode of treatment for TGCT associated with severe morbidity or functional limitations; however, some patients have disease that is not amenable to resection. For these patients, treatment options are limited because there are no approved systemic therapies for the disease. In February 2019, the FDA accepted, with Priority Review, a New Drug Application (NDA) for TURALIO based on results of the phase 3 ENLIVEN study, the first placebo-controlled study of a systemic investigational therapy in patients with TGCT.

TURALIO is approved with a Boxed Warning for the risk of serious and potentially fatal liver injury. Hepatotoxicity with ductopenia and cholestasis has occurred in patients treated with TURALIO. Across 768 patients who received TURALIO in clinical trials, there were two irreversible cases of cholestatic liver injury. One patient died with advanced cancer and ongoing liver toxicity, and one patient required a liver transplant. The mechanism of cholestatic hepatotoxicity is unknown, and its occurrence cannot be predicted. It is unknown whether liver injury occurs in the absence of increased transaminases.

"We are pleased to be able to dispense this first systemic therapy for TGCT to patients," said Ann Steagall, director of Clinical Policy, Biologics by McKesson. "It offers patients a treatment where surgery may not be an option. We’re excited to support TGCT patients as well as distribute another therapy in our growing portfolio of complex care. Due to the risk of serious and potentially fatal hepatotoxicity, TURALIO is only available through a Risk Evaluation and Mitigation Strategy (REMS) program. As a leading provider of risk assessment and care planning, Biologics is proud to help ensure that patients taking TURALIO get the level of care they need and deserve."

Biologics is committed to and recognized for its high level of customer service as well as its innovative, high-touch and multidisciplinary patient-centric approach. Each team includes a pharmacist with in-depth knowledge of therapies, an experienced nurse and a financial counselor who is familiar with various financial assistance programs and organizations that help patients. Each patient speaks to a PharmD prior to dispense and has access to them 24 hours a day, seven days a week. This highly-skilled care team works together to develop individualized care plans that address each patient’s unique clinical, financial and emotional needs and streamlines communication back to the treating provider, enabling high-quality care, differentiated outcomes and improved adherence. In addition, the Biologics team works closely with payers to ensure patients can access the specialty medications they need.

Clinicians may submit prescriptions to Biologics via phone (800.850.4306), fax (800.823.4506) or eScribe. For electronic prescribing systems, clinicians may search for Biologics within their EMR system

On August 7, 2019 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases and extend healthy lifespan, reported its financial results for the second quarter ended June 30, 2019 (Press release, CohBar, AUG 7, 2019, View Source [SID1234538366]).

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"During the quarter, we were pleased to resume the dosing of subjects in the Phase 1a/1b clinical trial of our lead candidate CB4211 for NASH and obesity, while continuing to progress our evaluation of additional novel peptides targeting fibrotic diseases, cancer, and type 2 diabetes," said Steven Engle, Chief Executive Officer. "We believe that CB4211 is the first of a number of candidates that our technology platform will identify for advancement into the clinic. Our seminar in June also provided strong validation from world class researchers and CohBar scientists of the potential of mitochondrial based therapeutics, as well as CohBar’s position as a leader in this emerging field. It’s an exciting time to be at CohBar."

Business Highlights

●Resumed CB4211 clinical trial: Dosing of subjects for the company’s Phase 1a/1b clinical trial of CB4211 resumed in June, and has continued in the current quarter for its lead MBT candidate for the potential treatment of nonalcoholic steatohepatitis (NASH) and obesity. The trial design is available at www.clinicaltrials.gov.

●Advanced new potential disease targets: During the second quarter, the company advanced its evaluation and optimization of novel analogs of mitochondrial-derived peptides for the potential treatment of fibrotic diseases. Preclinical studies provided preliminary evidence of anti-fibrotic activity in cell-based and animal models of idiopathic pulmonary fibrosis. Fibrotic disease is a major unmet medical need contributing to approximately one-third of deaths worldwide. The company also advanced its evaluation of novel analogs for the potential treatment of cancer, where in vitro data demonstrated the potential for CohBar peptides to enhance the killing of cancer cells by human immune cells. The market for cancer immunotherapy is projected to grow significantly to over $100 billion by 2023.

●Presented a novel scientific discovery at the American Diabetes Association annual conference: In June, CohBar presented its recent discovery of a mechanism of action of a family of novel peptides with positive effects on glucose tolerance in animal models of type 2 diabetes. The studies showed novel peptide analogs of CB5064 are effective in diet induced obese (DIO) mice, a widely used model of type 2 diabetes. Additional studies also demonstrated that CB5064 interacts with the apelin receptor, a key cell surface receptor involved in the regulation of glucose utilization, fluid homeostasis and cardiovascular function. Click link to ADA poster.

●Appointed new CEO and expanded Board of Directors: In May, Steven Engle was appointed as the company’s Chief Executive Officer. Mr. Engle has over two decades of executive leadership experience with public biotech companies developing breakthrough products in metabolic, autoimmune, oncologic and infectious disease areas. In April, the company expanded its Board with the addition of David Greenwood, who brings more than 30 years of financial and operational experience in biotechnology and investment banking.

●Hosted Mitochondria Based Therapeutics Seminar with key opinion leaders: In May, the company hosted a seminar, entitled "Mitochondria, a Source for Novel Therapeutics," which featured CohBar’s founders, Dr. Pinchas Cohen and Dr. David Sinclair, and CohBar’s Chief Science Officer, Dr. Ken Cundy, discussing the role of mitochondria in health and aging, and mitochondrial-derived peptides (MDPs) as a novel source of potential therapeutics for a host of major age-related diseases. A recording of the webcast is available at www.cohbar.com or by clicking on this link.

●Held Annual General Meeting and review: In June, the company held its Annual General meeting and provided a review and update on the company’s progress and plans. The slides are available on the company’s website at www.cohbar.com.

During the second quarter and more recently, Dr. Pinchas Cohen and Dr. Nir Barzilai continued to be recognized as international leaders in the study of mitochondrial science, aging and age-related diseases:

●Dr. Cohen delivered a keynote presentation on "Systems Biology of the Mitochondria in Aging" at the American Aging Association Annual Meeting in San Francisco, in May 2019; and "Addressing the Challenges of an Aging Society Through Innovation" at a panel entitled "Forum on Aging," at the G20 Meeting in Japan in June 2019. In addition, Dr. Cohen co-authored "Metabolomic profile of diet-induced obesity mice in response to humanin and small humanin-like peptide 2 treatment," published in Metabolomics, and "The mitochondrial-derived peptide MOTS-c is a regulator of plasma metabolites and enhances insulin sensitivity," published in Physiological Reports. He also authored an editorial published in The Journal of Molecular Medicine entitled "MOTS-c: an equal opportunity insulin sensitizer."

●Dr. Barzilai delivered a keynote presentation, entitled "Advancements in Genomics," at the Glenn Center Harvard Symposium in May 2019, and the keynote address at the Nutrition and Obesity Research Center (NORC) at the Harvard Annual Symposium in July 2019. In addition, Dr. Barzilai presented data at the Biology of Aging, Gordon Research Conference on Molecular, Cellular and Physiological Determinants of Lifespan and Healthspan. More recently, Dr. Barzilai co-authored three papers on "Creating the Next Generation of Translational Geroscientists," "The Development of Clinical Trials to Extend Healthy Lifespan," and a highly publicized paper in Nature on exome sequencing in type 2 diabetes.

Financial Highlights

●Cash and Investments. CohBar had cash and investments of $16.8 million as of June 30, 2019, compared to $22.2 million as of December 31, 2018. The cash burn for the quarter ended June 30, 2019, was approximately $2.9 million.

●R&D Expenses. Research and development expenses were $1.4 million in the three months ended June 30, 2019, compared to $1.8 million in the prior year quarter. The decrease was primarily due to the timing of certain preclinical and clinical costs incurred in the prior year period partially offset by an increase in costs associated with our research programs focused on the continuing development of peptides.

●G&A Expenses. General and administrative expenses were $1.5 million for the three months ended June 30, 2019, compared to $1.3 million in the prior year quarter. The increase in general and administrative expenses was primarily due to legal expenses related to the protection of our intellectual property, recruiting costs, and increased director fees.

●Net Loss. For the three months ended June 30, 2019, net loss, which included $0.8 million of non-cash expenses, was $3.1 million, or $0.07 per basic and diluted share. For the three months ended June 30, 2018, net loss, which included $0.9 million of non-cash expenses, was $3.3 million, or $0.08 per basic and diluted share.

Second Quarter Investor Call and Slide Presentation:

Date: August 7, 2019

Time: 5:00 p.m. EDT (2:00 p.m. PDT)

Conference Audio

-Dial-in U.S. and Canada: (800) 239-9838
-Dial-in International: (323) 794-2551
-Conference ID No.: 8671787

Slide Presentation

-Go to www.webex.com, click on the ‘Join’ button and enter meeting number 923 119 468 and Password CWBR, or
-Go to www.cohbar.com and click on Q2 2019 Shareholder Presentation at top of homepage.

For individuals participating in the Investor Call and Slide Presentation, please call into the conference audio and log into Webex approximately 10 minutes prior to its start.

An audio recording of the call will be available beginning at 8:00 p.m. (EDT) on August 7, 2019, through August 28, 2019. To access the recording please dial 1-844-512-2921 in the U.S. and Canada, or 1-412-317-6671 internationally, and reference Conference ID No. 8671787. The audio recording along with the slide presentation will also be available at www.cohbar.com during the same period.

About CB4211

CohBar’s lead clinical program is based on CB4211, a first-in-class mitochondria based therapeutic, that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide, which has been shown to play a significant role in the regulation of metabolism, and was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes an evaluation of biological activity relevant to NASH and obesity. NASH is a chronic and silent disease in its early stages that can progress to more serious disease stages, such as advanced fibrosis, cirrhosis, liver failure or liver cancer. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is no approved treatment for the disease.

On August 7, 2019 Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) reported its second quarter 2019 financial results and highlights (Press release, Synta Pharmaceuticals, AUG 7, 2019, View Source [SID1234538288]):

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Madrigal has continued to progress on several important fronts during the second quarter. We have moved forward with our Phase 3 study of MGL-3196 (resmetirom) in patients with biopsy-proven non-alcoholic steatohepatitis (NASH) and liver fibrosis (MAESTRO-NASH)," stated Paul Friedman, M.D., Chief Executive Officer of Madrigal. "We were also fortunate to add Jim Daly to our Board of Directors. Jim’s deep commercial expertise will be invaluable as we move forward with development of resmetirom in NASH and Non-Alcoholic Fatty Liver Disease (NAFLD). In addition, in recognition of our clinical development progress and corresponding growth of the Company, we promoted Becky Taub, M.D., to President of R&D."

Becky Taub, M.D.,CMO, President, Research & Development of Madrigal, added, "Madrigal is enrolling patients in our MAESTRO-NASH Phase 3 clinical study, and although it’s early in the process, we are encouraged by the rate of site openings and patient screening numbers. We are also pleased to announce that our abstract "Effects of Resmetirom (MGL-3196) on Hepatic Fat, Lipids, Liver Enzymes and Markers of Liver Fibrosis in an Open-Label 36-Week Extension Study in NASH Patients" has been selected for oral presentation at the Liver Meeting AASLD 2019 in Boston, November 2019. A poster, "Steatosis and Fibrosis Measured as Continuous Variables on Paired, Serial Liver Biopsies in the Resmetirom (MGL-3196) 36-Week Phase 2 NASH Study" will also be presented at AASLD meeting."

Additional information about Madrigal’s Phase 3 study in patients with NASH [NCT03900429] can be obtained at www.clinicaltrials.gov.

Financial Results for the Three and Six Months Ended June 30, 2019

As of June 30, 2019, Madrigal had cash, cash equivalents and marketable securities of $466.4 million, compared to $483.7 million at December 31, 2018. Cash used in operating activities during the first six months of 2019 was $18.0 million.

Operating expenses were $22.7 million and $40.8 million for the three and six month periods ended June 30, 2019, compared to $7.8 million and $14.9 million in the comparable prior year periods.

Research and development expenses for the three and six month periods ended June 30, 2019 were $15.6 million and $28.0 million compared to $5.1 million and $10.3 million in the comparable prior year periods. The increase is primarily attributable to additional activities related to initiation of our Phase 3 clinical trial in NASH, including a payment due related to a milestone achieved under our agreement with Roche, an increase in headcount and increased non-cash stock compensation from stock option awards.

General and administrative expenses for the three and six month periods ended June 30, 2019 were $7.1 million and $12.9 million compared to $2.7 million and $4.6 million in the comparable prior year periods. The increase is due primarily to higher non-cash stock compensation expense from stock option awards.

Interest income for the three and six month periods ended June 30, 2019 was $3.0 million and $6.0 million compared to $1.2 million and $1.9 million in the comparable prior year periods. The change in interest income was due primarily to a higher average principal balance in our investment portfolio in 2019, and increased interest rates.

About resmetirom (MGL-3196)

Among its many functions in the human body, thyroid hormone, through activation of its beta receptor, plays a central role in controlling lipid metabolism, impacting a range of health parameters from levels of serum cholesterol and triglycerides to the pathological buildup of fat in the liver. Attempts to exploit this pathway for therapeutic purposes in cardio-metabolic and liver diseases have been hampered by the lack of selectivity of older compounds for the thyroid hormone receptor (THR)-ß, chemically-related toxicities and undesirable distribution in the body.

Madrigal recognized that greater selectivity for thyroid hormone receptor (THR)-ß and liver targeting might overcome these challenges and deliver the full therapeutic potential of THR-ß agonism. Madrigal believes that resmetirom is the first orally administered, small-molecule, liver- directed, truly ß-selective THR agonist.

Based on the positive Phase 2 clinical study results in patients with NASH (Phase 2 36-Week Results Press Release), Madrigal initiated a Phase 3 multinational, double-blind, randomized, placebo-controlled study of resmetirom in patients with non-alcoholic steatohepatitis (NASH) and fibrosis to resolve NASH and reduce progression to cirrhosis and/or hepatic decompensation (Phase 3 Initiation Press Release and ClinicalTrials.gov NCT03900429). Additionally, in both the NASH Phase 2 study, and a second positive Phase 2 clinical study in patients with heterozygous familial hypercholesterolemia (Phase 2 HeFH Results Press Release), significant reductions in multiple atherogenic lipids were observed. As a result, Madrigal is designing a Phase 3 study intended to treat the prevalent dyslipidemias in NAFLD and NASH patients and improve the fatty liver phenotype in this population.