On July 13, 2020 Tyme Technologies, Inc. (NASDAQ: TYME), an emerging biotechnology company developing cancer metabolism-based therapies (CMBTs), reported preliminary financial and operating results for its first fiscal quarter ended June 30, 2020 (Press release, TYME, JUL 13, 2020, View Source [SID1234561836]). During first quarter fiscal 2021, the Company presented a growing body of preclinical and clinical data at major international medical meetings; continued enrolling patients in multiple studies including, second and third-line pancreatic cancer trials and the HopES Sarcoma Phase II trial; advanced planning for clinical trials in metastatic breast cancer and hematological cancers; and continued ongoing preclinical and mechanism data studies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As of June 30, 2020, the Company had approximately $21.3 million in cash and cash equivalents compared to $26.7 million as of March 31, 2020.

TYME’s operational cash burn rate for the first quarter of fiscal year 2021 was $6.7 million compared to $5.9 million for the fourth quarter of fiscal year 2020 and $6.2 million for the first quarter of fiscal 2020. The burn rate was below our previous projections and predominantly reflected costs associated with our ongoing TYME-88-Panc Phase II trial, as well as the launch of the pivotal phase of our TYME-88-Panc trial to evaluate SM-88 as a potential treatment for patients with third-line pancreatic cancer. Based on active clinical trials and other business developments, TYME continues to anticipate that its quarterly cash usage, or "cash burn rate", will average between $7.0 to 8.0 million per quarter for fiscal year 2021.

Anticipated Upcoming Key Events

TYME currently expects the following key events in calendar year 2020:

Continue to advance enrollment in TYME-88-Panc pivotal study
Continue to advance enrollment in the HopES Sarcoma Phase II Trial
Continue to advance enrollment in PanCAN’s Precision PromiseSM adaptive randomized Phase II/III registration-intent trial in patients with pancreatic cancer using oral SM-88 in second-line monotherapy
Publish final data for SM-88 Phase II prostate study
Initiate plans for SM-88 clinical programs into other tumor types potentially including metastatic breast, recurrent prostate and/or hematological cancers
Provide guidance on PanCAN’s Precision Promise SM adaptive Phase II/III trial evaluating SM-88 in patients with first-line pancreatic cancer in combination with gemcitabine (Gemzar ) and nab-paclitaxel (Abraxane )
Present and/or publish final data from Part 1 of TYME-88-Panc study
Continue proof-of-concept and IND-enabling activities for TYME-18
Summary of Recent Developments

TYME Presents New Preclinical Data Supporting SM-88 Mechanism of Action at AACR (Free AACR Whitepaper) 2020

TYME announced new preclinical findings that describe the unique mechanism of action of its lead cancer metabolism-based candidate, SM-88 (racemetyrosine). The data were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2020 Virtual Meeting from June 22 to June 24, 2020. Key highlights are as follows:

Preclinical data demonstrated statistically significant tumor reduction in SM-88 treated arms versus controls
Builds on confirmed tumor responses achieved in human clinical studies across 15 different cancer types
Multiple monotherapy effects of SM-88 were shown, including increased oxidative stress, disruption of autophagy, and tumor immunomodulation
Preclinical results were demonstrated across multiple tumor types including, pancreatic, colon, breast, and ovarian
Reduction of immunosuppressive cells in the tumor microenvironment supports exploring combination with immuno-oncology treatments
Oral SM-88 is designed to selectively disrupt cancer’s metabolic process and cause cancer cell death; SM-88 is currently in a pivotal trial for third-line pancreatic cancer
TYME-18 Demonstrates Anti-Cancer Effect in New Preclinical Data Presented at AACR (Free AACR Whitepaper) 2020

TYME announced new preclinical findings that described the unique anti-cancer effects of its cancer metabolism-based pipeline candidate, TYME-18. The data were presented at the AACR (Free AACR Whitepaper) 2020 Virtual Meeting from June 22 to June 24, 2020. Key highlights are as follows:

TYME-18 leverages unique metabolism of cancer to create an intra-tumoral treatment for inoperable cancers
Preclinical animal studies demonstrated TYME-18’s potential to dramatically shrink and/or resolve tumors
TYME-18 treatment resulted in a 91.6% (11/12) complete resolution of established tumors within two weeks
Novel mechanism of action contributes to a strong anti-cancer effect while minimizing systemic and local toxicity
Preclinical studies are ongoing with a goal of identifying an IND-enabling program
TYME Announced Health Economic Research Study Presented at ISPOR and Published in the Journal Value in Health Demonstrated Reduction in Total Cost of Care with Increased Use of New Medicines for Treatment of Patients with Pancreatic Cancer

The results of a health economic outcomes study demonstrated that the therapeutic benefit of increasing the use of novel medicines is so great that it is driving a decrease in the actual total cost of healthcare. The supporting data from the study were presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Virtual Meeting held from May 18 to May 20 and published in the Society’s journal, Value in Health.

The study looked at treatment inflation-adjusted expenses per patient for pancreatic cancer care between 2009 and 2016 and found that for every additional $1 spent on medicines for pancreatic cancer in 2016, there was a reduction in non-drug spending of $8-$9.

About SM-88

SM-88 is an oral investigational modified proprietary tyrosine derivative that is believed to interrupt the metabolic processes of cancer cells by breaking down the cells’ key defenses and leading to cell death through oxidative stress and exposure to the body’s natural immune system. Clinical trial data have shown that SM-88 has demonstrated encouraging tumor responses across 15 different cancers, including pancreatic, lung, breast, prostate and sarcoma cancers with minimal serious grade 3 or higher adverse events. SM-88 is an investigational therapy that is not approved for any indication in any disease.

About TYME-18

TYME-18 is composed of a proprietary surfactant delivery agent with a specific sulfonic acid component1. It is designed for intra-tumoral administration of difficult to treat tumors and leverages the acidic tumor microenvironment and signaling pathways to kill cancer cells. TYME-18 is distinct in composition, but like SM-88, aims to leverage susceptibilities of a cancer that are related to its altered metabolism. Initial preclinical data for TYME-18 in animal tumor models demonstrate rapid and complete tumor regression, with no reported local or systemic toxicities. TYME-18 continues to be studied as a potential therapy for difficult to treat tumors that may not be eligible for surgical or other interventions.

About TYME-88-Panc Pivotal Trial

The TYME-88-Panc pivotal trial applies the latest advances in the field of cancer metabolism by evaluating the efficacy and safety of an oral investigational compound that targets the metabolic mechanisms of the disease at its source. A prospective, open label pivotal trial in metastatic pancreatic cancer for patients who have failed two lines of any prior systemic therapy. The trial is designed to evaluate the safety and efficacy of SM-88 used with MPS (methoxsalen, phenytoin and sirolimus) in advanced pancreatic cancer and will measure multiple endpoints, including overall survival, progression free survival, relevant biomarkers, quality of life, safety, and overall response rate.

On July 13, 2020 Gilead Sciences, Inc. (Nasdaq: GILD) and Arcus Biosciences, Inc. (NYSE: RCUS), an oncology-focused biopharmaceutical company working to create best-in-class cancer therapeutics, reported the closing of their option and co-development and co-commercialization partnership agreement signed on May 27, 2020 (Press release, Gilead Sciences, JUL 13, 2020, View Source [SID1234561835]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The closing occurred following the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Under the terms of the agreements, the closing of this transaction triggered a payment of $175 million by Gilead to Arcus. In addition, Gilead made an equity investment in Arcus of approximately $200 million by purchasing shares at a price of $33.54 per share. As a result of this investment and Gilead’s participation in Arcus’s follow-on offering on May 28, 2020, Gilead now owns nearly 8.2 million shares of common stock of Arcus, representing approximately 13 percent of Arcus’s outstanding shares.

On July 13, 2020 Paige, a global leader in computational pathology, reported it received an additional $15M from Goldman Sachs Merchant Banking Division, totaling $20M from the firm (Press release, Paige AI, JUL 13, 2020, View Source [SID1234561834]). The funding will be added to previously announced Series B financing, bringing the total round to $70M, including an additional investment from Healthcare Venture Partners of $5M on top of their previous $10M investment. The funding brings the Company’s total capital raised to over $95M. Paige will use this new capital to further accelerate its global leadership position in transforming pathology workflows in the field of cancer, while working closely with biopharma companies to create custom diagnostic and clinical trial solutions to improve patient care.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We appreciate the continued recognition and support we’ve received from Goldman Sachs as we gain traction and prove early results in the clinical and biopharma space," said Leo Grady, Ph.D., CEO of Paige. "This new funding will help ensure that the Paige Platform and our advanced computational pathology products will drive the next generation of pathology and improve cancer care globally."

A recent company milestone for Paige include naming David Castelblanco, Managing Director at Goldman Sachs, as a new member to Paige’s Board of Directors.

"We have been very impressed with the Company and its pace of development. We are excited to increase our commitment to support Leo, Thomas and the Paige team’s transformative work with artificial intelligence and machine learning in the cancer field," said David Castelblanco.

Paige also released multiple ASCO (Free ASCO Whitepaper) and Modern Pathology publications showing the value of the Paige offering. Recently, Paige was also recognized by Frost and Sullivan’s 2020 Enabling Technology Leadership Award in the North American AI-based digital pathology industry.

"We initially invested in Paige recognizing the potential of their products to add significant value to the industry and impact the future of cancer care," said Jeffrey C. Lightcap, Senior Managing Director of Healthcare Venture Partners. "After seeing Paige make tremendous progress in such a short period, we added to our investment to further accelerate their growth."

ABOUT GOLDMAN SACHS MERCHANT BANKING

Founded in 1869, The Goldman Sachs Group, Inc. is a leading global investment banking, securities and investment management firm. Goldman Sachs Merchant Banking Division (MBD) is the primary center for the firm’s long-term principal investing activity. MBD is one of the leading private capital investors in the world with investments across private equity, growth equity, infrastructure, private debt, and real estate.

On July 13, 2020 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed the Container Closure Integrity (CCI) test that is required by the U.S. Food and Drug Administration (FDA) for its CypCaps product and has passed the FDA-required test. This is the final FDA-required test on the product that will be used in the company’s planned clinical trial in locally advanced, inoperable pancreatic cancer (Press release, PharmaCyte Biotech, JUL 13, 2020, View Source [SID1234561833]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of the completed test, "It is with great enthusiasm that we announce our Cell-in-a-Box encapsulated cell product CypCaps has passed the CCI test. This test is a component of the 24-month stability study. As with the test results we announced earlier this month, the CCI test is part of the ongoing study to determine the shelf life of the CypCaps final product that the FDA requires for all medical products. The data from the CCI test will be included in our IND. All future longer-term shelf life analyses, such as the next CCI test at the one-year post-production time period, will be reported to the FDA but is not required for PharmaCyte to submit its Investigational New Drug application (IND)."

As explained previously, regulatory agencies around the world, including the FDA, require a shelf-life determination for all medical products. Living products, like cell therapies such as CypCaps, as well as live vaccines etc., are particularly sensitive and more prone to inactivation over time, so it is especially important to determine the shelf-life for PharmaCyte’s clinical trial product.

The FDA specifically required a CCI test be run on PharmaCyte’s clinical trial product and that the data from the CCI test be included in PharmaCyte’s IND.

More can be read about the specifics of the CCI test in PharmaCyte’s June 15, 2020 press release: View Source

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On July 13, 2020 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that Zepzelca (lurbinectedin) was added by the National Comprehensive Cancer Network (NCCN) to the Clinical Practice Guidelines in Oncology (NCCN Guidelines) for small cell lung cancer (SCLC) on July 7, 2020 (Press release, Jazz Pharmaceuticals, JUL 13, 2020, View Source [SID1234561832]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The U.S. Food and Drug Administration (FDA) approved Zepzelca under accelerated approval on June 15, 2020 for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy.1 The approval was based on the data from the open-label, single-arm monotherapy trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

The NCCN Guidelines for SCLC now include lurbinectedin as a recommended regimen for both patients who relapse six months and less after prior systemic therapy and for patients who relapse more than six months after prior systemic therapy. For patients who relapse six months and less, lurbinectedin is a preferred regimen.

"We appreciate the decision by the NCCN to quickly incorporate Zepzelca into the Clinical Practice Guidelines in Oncology as it supports our commitment to ensuring that patients with relapsed small cell lung cancer are able to access this important new treatment option," said Robert Iannone M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "Zepzelca has the potential to fill an unmet need in relapsed SCLC where treatment options have been limited."

The NCCN, a not-for-profit alliance of 30 leading U.S. cancer centers devoted to patient care, research, and education, is dedicated to improving and facilitating quality, effective, efficient, and accessible cancer care so patients can live better lives. The intent of the NCCN Guidelines is to assist in the decision-making process of individuals involved in cancer care—including physicians, nurses, pharmacists, payers, patients and their families—with the ultimate goal of improving patient care and outcomes.

About Zepzelca (lurbinectedin)
Zepzelca, also known as PM1183, is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.1

Zepzelca for injection 4 mg is a prescription medicine used to treat adults with a kind of lung cancer called small cell lung cancer that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted. Additional studies will further evaluate the benefit of Zepzelca for this use.

Important Safety Information

Before receiving ZEPZELCA, tell your healthcare provider about all of your medical conditions, including if you:

have liver or kidney problems.
are pregnant or plan to become pregnant. ZEPZELCA can harm your unborn baby.
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with ZEPZELCA.
You should use effective birth control (contraception) during treatment with and for 6 months after your final dose of ZEPZELCA.
Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with ZEPZELCA.
Males with female partners who are able to become pregnant should use effective birth control during treatment with and for 4 months after your final dose of ZEPZELCA.

are breastfeeding or plan to breastfeed. It is not known if ZEPZELCA passes into your breastmilk. Do not breastfeed during treatment with ZEPZELCA and for 2 weeks after your final dose of ZEPZELCA. Talk to your healthcare provider about the best way to feed your baby during treatment with ZEPZELCA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain other medicines may affect how ZEPZELCA works.

What should I avoid while using ZEPZELCA?

Avoid eating or drinking grapefruit, or products that contain grapefruit juice during treatment with ZEPZELCA.

ZEPZELCA can cause serious side effects, including:

Low blood cell counts. Low blood counts including low neutrophil counts (neutropenia) and low platelet counts (thrombocytopenia) are common with ZEPZELCA, and can also be severe. Some people with low white blood cell counts may get fever, or an infection throughout the body (sepsis), that can cause death. Your healthcare provider should do blood tests before you receive each treatment with ZEPZELCA to check your blood cell counts.
Tell your healthcare provider right away if you develop:
fever or any other signs of infection
unusual bruising or bleeding
tiredness
pale colored skin
Liver problems. Increased liver function tests are common with ZEPZELCA, and can also be severe. Your healthcare provider should do blood tests to check your liver function before you start and during treatment with ZEPZELCA.
Tell your healthcare provider right away if you develop symptoms of liver problems including:
loss of appetite
nausea or vomiting
pain on the right side of your stomach area (abdomen)
Your healthcare provider may temporarily stop treatment, lower your dose, or permanently stop ZEPZELCA if you develop low blood cell counts or liver problems during treatment with ZEPZELCA.

The most common side effects of ZEPZELCA include:

tiredness
low white and red blood cell counts
increased kidney function blood test (creatinine)
increased liver function blood tests
increased blood sugar (glucose)
nausea
decreased appetite
muscle and joint (musculoskeletal) pain
low level of albumin in the blood
constipation
trouble breathing
low levels of sodium and magnesium in the blood
vomiting
cough
diarrhea
These are not all of the possible side effects of ZEPZELCA.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

More information about Zepzelca, including Full Prescribing Information and Patient Information, is available here.

ZEPZELCA is a trademark of PharmaMar, S.A. used by Jazz Pharmaceuticals under license.