On September 9, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that it successfully completed a pre-IND (Investigational New Drug) meeting with the U.S. Food and Drug Administration (FDA) regarding the development plan for Annamycin, including the clinical study design and dosing strategy for the initial phase 1b/2 protocol for soft tissue sarcomas with lung metastases (Press release, Moleculin, SEP 9, 2020, View Source [SID1234564802]).

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Moleculin submitted a proposed clinical protocol for FDA review entitled, "Phase 1b/2 Study of Liposomal Annamycin (Annamycin) in Subjects with Previously Treated Soft-Tissue Sarcomas with Pulmonary Metastases." The proposed study is an open-label, multicenter, single-arm, dose escalation and expansion study to evaluate single-agent Annamycin in up to 55 patients with soft tissue sarcoma (STS) with lung metastases for whom chemotherapy is considered appropriate. The primary objectives of the dose escalation phase are to evaluate the safety of Annamycin and identify the maximum tolerated dose (MTD) or the recommended Phase 2 dose (RP2D).

In summary, the FDA, among other items:

did not object to the proposed clinical study design while providing guidance on additional assessments

agreed the proposed dose escalation schedule appeared reasonable

commented regarding the consideration for including adolescents in oncology clinical trials

stated that a repeat dose toxicology study of 3 months is required before initiating a registration study

recommended an EOP1 meeting after completion of the RP2D.

"We are pleased to complete the pre-IND meeting with the FDA, and will move forward with our plans to file the IND by the end of 2020 and initiate a Phase 1b/2 trial of Annamycin for the treatment of soft tissue sarcomas metastasized to the lungs," said Wally Klemp, Chief Executive Officer of Moleculin. "We appreciate the FDA’s guidance as we endeavor to find a cure for certain cancers metastasized to the lungs."

On September 9, 2020 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for its fiscal year ended June 30, 2020 (Press release, MEI Pharma, SEP 9, 2020, View Source [SID1234564823]).

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"This past year was marked by progress on multiple fronts, particularly for zandelisib, formerly called ME-401, which is our PI3K delta inhibitor in the Phase 2 TIDAL study intended to support an accelerated approval marketing application with FDA," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "The progress on zandelisib this past year includes entering a global license, development and commercialization agreement with Kyowa Kirin Company, maturing Phase 1b study data that continues to support best-in-class potential, as well as obtaining Fast Track designation from FDA."

Dr. Gold continued: "With about $183 million in cash at the start of our fiscal year, we believe we have an operational runway at least through 2023 and are well positioned to advance our ongoing clinical programs. In particular, we look forward to completing TIDAL enrollment as we initiate additional clinical studies to fully explore zandelisib’s potential to benefit patients with B-cell malignancies while we continue our staged build-out of a commercial infrastructure to optimally capture value from the program."

Fiscal Year 2020 Zandelisib (ME-401) and Corporate Highlights

Zandelisib (formerly called ME-401), an oral, once-daily, investigational drug-candidate selective for phosphatidylinositol 3-kinase delta (PI3Kδ), for B-Cell Malignancies

The World Health Organization approved "zandelisib" as the International Nonproprietary Name, or INN, for ME-401, the Company’s investigational cancer treatment being developed as an oral, once-daily, selective PI3Kδ inhibitor for the treatment of B-cell malignancies.
In May 2020, the Company and Kyowa Kirin Co. presented updated data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program from a Phase 1b study of zandelisib (ME-401) for the treatment of B-cell malignancies. These data evaluating patients on an intermittent dosing schedule of zandelisib showed that treatment was generally well tolerated and demonstrated an 83% overall response rate in patients with relapsed or refractory follicular lymphoma (n=36). The responses were durable with no median yet reached (median follow-up of 13.2 months: range: 3.0-27.6).
In April 2020, the Company entered into a global license, development and commercialization agreement with Kyowa Kirin Co. to further develop and commercialize MEI’s zandelisib:
MEI and Kyowa Kirin will co-develop and co-promote zandelisib in the U.S.
MEI to book U.S. sales on 50-50 profit and cost sharing.
$100 million upfront cash payment to MEI.
$582.5 million in potential development, regulatory and commercial milestones
Kyowa Kirin obtains exclusive commercialization rights ex-U.S.
MEI to receive escalating tiered royalty payments starting in the teens on ex-U.S. sales.
In March 2020, the Company was granted Fast Track designation by the U.S. FDA for zandelisib for the treatment of adult patients with relapsed or refractory follicular lymphoma.
Corporate Highlights

In April 2020, Cheryl L. Cohen, former chief commercial officer of Medivation, Inc. and a product launch and commercialization veteran with over 25 years of service in the pharmaceutical and biotechnology industry, joined the Board of Directors.
In December 2019, the Company closed an underwritten public offering of 32,343,750 shares of its common stock for total gross proceeds of approximately $51,750,000. Additionally, during the year ended June 30, 2020, MEI sold 5,471,684 shares under the ATM Sales Agreement for net proceeds of $20.8 million.
In July 2019, Tamar Howson, M.S., MBA, a highly experienced business development executive with over 30 years of service in the pharmaceutical and biotechnology industry, joined the Board of Directors.
Fiscal Year 2020 Financial Results

As of June 30, 2020, MEI had $182.6 million in cash, cash equivalents, and short-term investments with no outstanding debt. Additionally, MEI has a receivable of $20.4 million that is expected to be received from the Japanese taxing authorities in fiscal year 2021 that was withheld from the $100 million paid by Kyowa Kirin Co. under the terms of April 2020 global license, development and commercialization agreement. The withholding was a result of the US Internal Revenue Service being closed because of the COVID pandemic, resulting in an inability to provide the necessary documentation to support an exemption from the required foreign withholding.
For the year ended June 30, 2020, cash used in operations was $45.3 million compared to $39.4 million for 2019.
Research and development expenses were $34.1 million for the year ended June 30, 2020, compared to $32.3 million for 2019. The increase was primarily related to increased development costs associated with zandelisib, as well as increased personnel costs to support our clinical trial activities.
General and administrative expenses were $16.7 million for the year ended June 30, 2020, compared to $14.6 million for 2019. The increase primarily relates to personnel costs and general corporate expenses incurred during the year ended June 30, 2020.
MEI recognized revenues of $28.9 million for the year ended June 30, 2020, compared to $4.9 million for the year ended June 30, 2019. Revenues resulted from the recognition of license revenue associated with the Kyowa Kirin license agreement as well as fees allocated to research and development activities related to the Kyowa Kirin and Helsinn license agreements.
Net loss was $46.0 million, or $0.51 per share, for the fiscal year ended June 30, 2020, compared to net loss of $16.8 million, or $0.24 per share for 2019. The Company had 111,513,689 shares of common stock outstanding as of June 30, 2020, compared with 73,544,576 shares as of June 30, 2019.
The adjusted net loss for the fiscal year ended June 30, 2020, excluding non-cash expenses related to changes in the fair value of the warrants issued in connection with the May 2018 financing (a non-GAAP measure), was $23.1 million.
Conference Call and Webcast

MEI Pharma will host a conference call with simultaneous webcast today, September 9, 2020, at 5:00 p.m. Eastern time to provide a corporate update. To access the live call, please dial (866) 939-3921 (United States) or (678) 302-3550 (International), conference ID 49919899. The conference call will also be webcast live and can be accessed at www.meipharma.com. A replay of the webcast will be available approximately one hour after the conclusion of the call.

On September 9, 2020 TScan Therapeutics, a biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies in oncology, reported plans to file two Investigational New Drug (IND) applications in their liquid tumor program in 2021 (Press release, TScan Therapeutics, SEP 9, 2020, View Source [SID1234564844]). Their first product, TSC-100, targets HA-1 and is designed to treat patients receiving hematopoietic stem cell transplant therapy with the goal of preventing relapse, a high unmet need in this setting. TScan announced selection of their lead TCR and its advancement to IND-enabling activities. Simultaneously, TScan announced selection of their second target, HA-2, with plans to file a second IND in 2021. These products are the first two TCRs in a multi-TCR program designed to provide treatment options for the majority of patients receiving stem cell therapy.

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"We are excited to progress this T cell therapy solution for patients receiving stem cell transplant therapy," said David Southwell, Chief Executive Officer at TScan. "TScan’s goal in both its liquid and solid tumor programs is to learn from patients who respond well to therapy to treat those who are less fortunate. This represents a significant step in the progression of TScan as a TCR therapy company. Our lead TCR, TSC-100, was discovered internally using our TCR discovery platform, R-Scan, and was significantly derisked using our proprietary genome-wide safety screening platform, T-Scan. By extending our liquid tumor program to also include HA-2, we are one step closer to providing a comprehensive solution for patients receiving stem cell therapy. We also remain on track to nominate solid tumor targets in 2021."

Leveraging its core TCR discovery platform, R-Scan, TScan identified its lead HA-1 TCR after screening over 100,000,000 T cells from HA-1-negative donors. In preclinical experiments, TSC-100 showed strong activity against HA-1-positive leukemia cells and a clean safety profile, with minimal off-target interactions or alloreactivity. TSC-100 will be used to engineer donor T cells in the context of a stem cell transplant, with the goal of preventing relapse in HA-1-positive patients with AML, MDS, or adult ALL. To expand this program to patients not expressing HA-1, TScan is developing a second TCR specific for HA-2, termed TSC-101.

"Our goal is to provide a therapeutic option for every patient," said Gavin MacBeath, Chief Scientific Officer. "Our discovery team is already identifying TCRs that will allow our products to address an even broader patient population. Expanding our repository of therapeutic TCRs also enables us to develop multiplexed TCR therapies, which better mimic natural immune responses and provide more robust treatments for heterogeneous cancers like AML, as well as a diverse range of solid tumors. Using our two core platforms, we remain on track to nominate our first solid tumor candidates in early 2021."

About HA-1

HA-1 is a well-characterized minor histocompatibility antigen that is expressed on all blood cells, including leukemia cells, but is not expressed at appreciable levels in other normal tissues. It is associated with clinical benefit by generating a ‘graft vs. leukemia’ effect in the context of hematopoietic stem cell transplants in which the patient is HA-1-positive and the donor is HA-1-negative. Over half of all patients express the HA-1 target.

About HA-2

Similar to HA-1, HA-2 is a minor histocompatibility antigen expressed specifically on blood cells and is associated with clinical benefit through a ‘graft vs. leukemia’ effect. Addition of TSC-101, an HA-2-specific TCR, will expand the pool of eligible patients for TScan’s liquid tumor program.

On September 9, 2020 Phosplatin Therapeutics, a clinical stage pharmaceutical company focused on oncology therapeutics, reported the addition to its growing team of Joseph F. O’Donnell, MD, as Interim Chief Medical Officer and Jason Summa in the newly created role of Vice President of Clinical Development (Press release, Phosplatin, SEP 9, 2020, View Source [SID1234564877]).

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"We welcome both Dr. O’Donnell and Jason to our rapidly growing clinical team. As a long-standing member of our Scientific Advisory Board from Phosplatin’s early days, Dr. O’Donnell is uniquely qualified to oversee the strategy, direction and execution of our clinical development program. Jason brings deep knowledge as a biotechnology and pharmaceutical executive with many years of clinical trial experience in a variety of solid tumors, including ten years in the prostate cancer space," said Robert Fallon, President and Chief Executive Officer of Phosplatin Therapeutics. "As Phosplatin continues to advance PT-112 to the next stage of development, having successfully completed our second and third Phase 1 clinical trials earlier this year, the clinical development expertise brought to these roles by both Dr. O’Donnell and Jason will be invaluable in our continued efforts in Phase 2 development of PT-112."

Dr. O’Donnell is a deeply experienced clinical oncologist who began his career at the National Cancer Institute, and after that had a long-standing academic career at the Geisel School of Medicine at Dartmouth, where he most recently served as the Elizabeth Decamp McInerney Professor of Medicine. He also served for many years as an Associate Dean and Senior Scholar of the C Everett Koop Institute at Dartmouth. He is on the editorial board of the Journal of Cancer Education, where he also served for years as Editor-in-Chief and the Journal of Palliative Care, as well as being an editorial referee for six journals and grants referee for the National Cancer Institute. He has published more than 100 studies and four books related to oncology and medical education.

Dr. O’Donnell is the recipient of millions of dollars in grants from the National Institutes of Health, the American Cancer Society, and the Corporation for National and Community Service, among others. He has served on countless committees, and is a respected lecturer and a member of many professional societies, including the American Association of Cancer Education, the European Association for Cancer Education, and the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper). He graduated with an A.B. in biology summa cum laude from Harvard College and an MD from Harvard Medical School.

"The unique potential of PT-112 has intrigued me from my early involvement with Phosplatin. I am eager to draw on my years of experience in the oncology field to lead the clinical development team to an FDA approval to make this potentially highly impactful therapy available to cancer patients," said Dr. O’Donnell.

Mr. Summa most recently served as Oncology Director and Clinical Project Scientist with the Janssen Pharmaceutical Division of Johnson & Johnson where he was responsible for the clinical program for developing niraparib, a PARP-inhibitor under development in metastatic castration-resistant prostate cancer patients with DNA repair defects, including successfully procuring Breakthrough Therapy Designation. Previously he spent nine years with BIND Therapeutics in a variety of clinical roles in the area of oncology, including oversight of the clinical department. He began his career with Alkermes and served in drug development roles with Corixa Corporation and Momenta Pharmaceuticals as well. Mr. Summa has authored more than 15 publications, developed protocols for eight clinical trials and holds a patent for inhalable epinephrine. He is a member of six professional oncology-related organizations.

"I am looking forward to following my passion and offering my experience in oncology clinical trial development to contribute to the commercial availability of this unique therapy, and to working alongside the talented Phosplatin team, with its deep expertise and strategic vision," said Mr. Summa.

The Company’s previous Chief Medical Officer, Jose Jimeno, MD, PhD, has recently taken on new responsibility as VP Global Head, Virology and Inflammation, in a newly created division of PharmaMar (Madrid, Spain) and has become a member of the Phosplatin Therapeutics Scientific Advisory Board in order to continue his involvement with the PT-112 development program.

About PT-112

PT-112 is the first small molecule conjugate of pyrophosphate developed in oncology therapeutics. PT-112 promotes immunogenic cell death (ICD), or the release of damage associated molecular patterns (DAMPs) that lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents a potential best-in-class small molecule inducer of this immunological form of cancer cell death, and is under Phase 2 development. The first-in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients, and won "Best Poster" at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress within the Developmental Therapeutics category. The novelty of its pyrophosphate moiety also results in osteotropism, or the propensity of the drug to reach the mineralized bone. This property is of interest in cancer types that originate in bone, or frequently lead to metastatic bone involvement, such as metastatic castrate-resistant prostate cancer (mCRPC). The first human clinical results in mCRPC were presented at the 2020 Genitourinary Cancers Symposium on February 13, 2020.

On September 9, 2020 Alkermes plc (Nasdaq: ALKS) reported that that its Chief Executive Officer, Richard Pops, will participate in a fireside chat at the Cantor Virtual Global Healthcare Conference on Wednesday, Sept. 16, 2020 at 10:40 a.m. ET (3:40 p.m. BST) (Press release, Alkermes, SEP 9, 2020, View Source [SID1234564903]). The webcast may be accessed under the Investors tab on www.alkermes.com and will be archived for 14 days.

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