On March 27, 2020 Monopar Therapeutics Inc. (Monopar or the Company) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported fourth quarter and full-year 2019 financial results and business updates (Press release, Monopar Therapeutics, MAR 27, 2020, View Source [SID1234555944]).

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Fourth Quarter and Recent Highlights

Initial Public Offering

In December 2019, the Company completed its initial public offering of 1,277,778 shares of common stock, including the underwriters’ exercise of their over-allotment option, at a public offering price of $8.00 per share before underwriting discounts and commissions.

The shares began trading on the Nasdaq Capital Market on December 19, 2019 under the symbol "MNPR."
Orphan Drug Designation

On February 18, 2020, the Company announced that the European Commission has granted Orphan Drug Designation for the Company’s Phase 2 clinical-stage drug candidate, camsirubicin, for the treatment of soft tissue sarcomas. Camsirubicin is being developed under a clinical trial partnership with Grupo Español de Investigación en Sarcomas (GEIS), an internationally renowned non-profit organization focused on the research and development of drugs for sarcoma cancers. The approximately 170-patient GEIS-sponsored camsirubicin Phase 2 clinical trial for the treatment of advanced soft tissue sarcoma is anticipated to begin in the second half of 2020.

European Orphan Drug Designation benefits include protocol assistance, reduced EU regulatory filing fees and 10 years of market exclusivity. Designated orphan medicines are also eligible for conditional marketing authorization. Camsirubicin has already received Orphan Drug Designation in the U.S. by the Food and Drug Administration (FDA), which provides for similar benefits such as fee reductions and 7 years of market exclusivity.
Fourth Quarter and Full Year Summary Financial Results

Results for the Quarter and Year Ended December 31, 2019 Compared to the Quarter and Year Ended December 31, 2018

Cash and cash equivalents as of December 31, 2019 were approximately $13.2 million, compared to approximately $6.9 million as of December 31, 2018. The increase in cash and cash equivalents was driven primarily by the approximately $9.4 million net cash proceeds from the Company’s initial public offering offset by approximately $3.0 million of cash used in operating activities.

Research and Development (R&D) Expenses

R&D expenses for the quarter ended December 31, 2019 were approximately $0.6 million, compared to approximately $0.5 million for the quarter ended December 31, 2018, an increase of approximately $0.1 million.

R&D expenses for the year ended December 31, 2019 were approximately $2.0 million, compared to approximately $1.8 million for the year ended December 31, 2018, an increase of approximately $0.2 million.

General and Administrative (G&A) Expenses

G&A expenses for the quarter ended December 31, 2019 were approximately $0.6 million, compared to approximately $0.4 million for the quarter ended December 31, 2018, an increase of approximately $0.2 million.

G&A expenses for the year ended December 31, 2019 were approximately $2.4 million, compared to approximately $1.6 million for the year ended December 31, 2018, an increase of approximately $0.8 million.

Net loss was $0.13 per share for the fourth quarter of 2019, compared to $0.10 per share in the comparable period in 2018. For the year ended December 31, 2019, net loss was $0.45 per share compared to $0.35 per share in the same period in 2018.

On March 27, 2020 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized CAR T platform technology engineered to target patient- and tumor-specific neoantigens, reported its financial results for the year ended December 31, 2019 and provided a corporate update (Press release, Xenetic Biosciences, MAR 27, 2020, View Source [SID1234555943]).

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Jeffrey Eisenberg, Chief Executive Officer of Xenetic, stated, "2019 proved to be a pivotal year for Xenetic with our completion of the acquisition of XCART, a differentiated CAR T platform technology that we believe has the potential to address a significant unmet need in B-cell non-Hodgkin lymphoma. Moving forward we believe 2020 will be an important year for the Company as we plan to continue driving the development of XCART. To that end, we have made encouraging progress with additions to our team and are advancing towards securing an academic collaboration. We believe this pathway will provide many advantages, including access to manufacturing suites, established CMC and regulatory infrastructure and cost and risk mitigation."

"In the fourth quarter of last year, we received our first royalty payment under our agreement with Takeda involving our proprietary PolyXen technology in the field of coagulation disorders. While the recognized payment was modest, it is what we expected given the early stages of this product launch. Importantly, the commencement of royalties reaffirms the value of our PolyXen intellectual property and the opportunity to leverage it to prolong a drug’s circulating half-life and potentially improve other pharmacological properties. Further, in these unprecedented times, we are reviewing our portfolio of intellectual property assets to see if we can contribute in any way to the global effort to address the current health threat of COVID-19," continued Mr. Eisenberg. "As we advance into the rest of 2020, despite the current climate of financial uncertainty, we are moving our operation and development efforts forward with a strong cash balance which we believe provides us with a cash runway through mid-2021."

XCART Platform Technology Overview: Significantly differentiated, proprietary CAR T therapy designed to develop cell-based therapeutics for the treatment of multiple tumor types of B-cell Non-Hodgkin lymphomas, an area of significant unmet need, with the potential to address an initial global market opportunity of over $5 billion annually.[1] Xenetic believes XCART has the potential to transform CAR T therapy.

The Company has recently bolstered its cell therapy manufacturing and CMC expertise and capabilities with the appointments of Greg MacMichael, Ph.D. and Maksim Mamonkin, Ph.D., to its Scientific Advisory Board. Both Dr. MacMichael and Dr. Mamonkin are actively engaged with the Company to advance the development of the XCART technology platform.

Expected 2020 Milestones

INTERACT meeting with the United States Food and Drug Administration ("FDA")
Enter into an academic site collaboration
Advance IND-enabling studies
Explore opportunities for Orphan Drug designation
PolyXen Platform Technology: Patent-protected platform technology designed for protein or peptide therapeutics, enabling next-generation biological drugs by prolonging a drug’s circulating half-life and potentially improving other pharmacological properties.

Program Highlights:

Exclusive License Agreement with Takeda Pharmaceuticals Co. Ltd. ("Takeda") in the field of coagulation disorders. Takeda currently has one active development program underway utilizing the PolyXen platform technology.
The Company received $17,000 in royalty revenue during the fourth quarter of 2019 representing its first payment under the Company’s license agreement with Takeda. This payment and expected future payments relate to a sublicense of Xenetic’s PolyXen intellectual property, entered into by Takeda with a third party in 2017. The Company expects this quarterly royalty payment to increase as the relevant product launch continues to be rolled out by the sublicensee.
Mr. Eisenberg concluded, "We are closely monitoring the evolving COVID-19 situation. While the situation is very fluid, to date we have not experienced any significant impact or delays on our projected timelines. The safety and wellness of our employees, partners and the community are of the upmost importance to us and we have implemented risk mitigation strategies to ensure the least amount of disruption to our operations as possible."

Summary of Financial Results for Fiscal Year 2019

Net cash used in operating activities for the year was $6.4 million. During 2019, our working capital increased by $10.1 million primarily due to our March 2019 registered direct offering and our July 2019 public offering that resulted in $16.1 million in combined net proceeds. This increase in working capital was partially offset by the Company’s reported net loss for the year ended December 31, 2019. The Company ended the year with approximately $10.4 million of cash.

On March 27, 2020 Sanofi reported that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Sarclisa (isatuximab) (Press release, Sanofi, MAR 27, 2020, View Source [SID1234555925]). The CHMP recommends Sarclisa in combination with pomalidomide and dexamethasone (pom-dex) for the treatment of adult patients with relapsed and refractory multiple myeloma (MM) who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy.

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The European Commission (EC) will review the CHMP recommendation and a final decision on the Marketing Authorisation Application for Sarclisa in the E.U. is expected in the coming months. Sarclisa has not been approved for commercial use in the E.U. Sarclisa was approved in the US on March 2 in combination with pomalidomide and dexamethasone (pom-dex) for the treatment of adults with RRMM who have received at least two prior therapies including lenalidomide and a proteasome inhibitor.

"Relapsed and refractory multiple myeloma is a complicated disease that continuously develops resistance to treatment, creating a significant need for continued innovation," said John Reed, M.D., Ph.D., Sanofi’s Global Head of Research & Development. "This positive CHMP opinion for Sarclisa brings us closer to our ambition to deliver a new treatment option for patients in Europe with relapsed and refractory multiple myeloma."

Sarclisa Phase 3 Study Results in Patients with RRMM

The CHMP positive opinion is based on data from ICARIA-MM, the first randomized Phase 3 trial to evaluate an anti-CD38 monoclonal antibody (mAB) in combination with pom-dex. In the ICARIA-MM study, Sarclisa added to pom-dex (Sarclisa combination therapy; n=154) demonstrated a statistically significant improvement of progression free survival (PFS) with a median PFS of 11.53 months compared to 6.47 months with pom-dex alone (n=153; HR 0.596, 95% CI: 0.44-0.81, p=0.0010). Sarclisa combination therapy also demonstrated a significantly greater overall response rate compared to pom-dex alone (60.4% vs. 35.3%, p<0.0001). In additional analyses, Sarclisa combination therapy compared to pom-dex alone showed a treatment benefit consistent across select subgroups reflective of real-world practice, including patients with high risk cytogenetics, those aged 75 years and older, patients with renal insufficiency, and patients who were refractory to lenalidomide.

The most common adverse reactions (all grades occurring in 20% or more of patients) in patients who received Sarclisa combination therapy were neutropenia (96%), infusion-related reactions (39%), pneumonia (31%), upper respiratory tract infection (57%) and diarrhea (26%). Serious adverse reactions that occurred in more than 5% of patients who received Sarclisa combination therapy included pneumonia (25.3%) and febrile neutropenia (12.3%). Permanent discontinuation of Sarclisa combination therapy due to an adverse reaction (Grades 3-4) occurred in 7% of patients, and 3% of patients discontinued due to an infusion-related reaction.

Multiple Myeloma: A Significant Burden to Patients

Multiple myeloma is the second most common hematologic malignancy1, with more than 138,000 new diagnoses of multiple myeloma worldwide yearly.2 In Europe, approximately 39,000 patients are diagnosed with multiple myeloma each year.3 Despite available treatments, multiple myeloma remains an incurable malignancy, and is associated with significant patient burden. Since multiple myeloma does not have a cure, most patients will relapse. Relapsed multiple myeloma is the term for when the cancer returns after treatment or a period of remission. Refractory multiple myeloma refers to when the cancer does not respond or no longer responds to therapy.

On March 26, 2020 Apexigen, Inc., a clinical-stage biopharmaceutical company, reported that Xiaodong Yang, M.D., Ph.D., President and Chief Executive Officer, plans to present an overview of the company at the upcoming Solebury Trout Virtual Investor Conference (Press release, Apexigen, MAR 26, 2020, View Source [SID1234590994]). The 25-minute presentation includes an interactive Q&A with participants prompted to submit questions electronically. Details are as follows:

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Date/time: Thursday, April 2, 2020 at 3:00pm EDT

Access: https://78449.themediaframe.com/dataconf/productusers/solebury/mediaframe/36519/indexl.htm

On March 26, 2020 Oncoceutics, Inc. reported that the Chinese National Intellectual Property Administration (CNIPA) has issued a Notice of Allowance for a Composition of Matter patent for ONC201, the company’s lead development candidate, as well as for several other compounds of the imipridone family (Press release, Oncoceutics, MAR 26, 2020, View Source [SID1234558314]). This new patent, along with a Composition of Matter patent for ONC201 issued in the United States in March 2019 and an intention to grant from the European Patent Office issued in August 2019, gives Oncoceutics extensive coverage for ONC201 around the world.

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Oncoceutics licensed this group of patents from The Scripps Research Institute in August 2019 to expand its already broad patent coverage of ONC201 and related compounds. With these new patents, Oncoceutics has more than 20 patents for ONC201 issued globally with 10 patents for ONC201 issued by the United States Patent Office.

"We are delighted that CNIPA has recognized ONC201 and our portfolio of novel compounds, called imipridones, and is reviewing the Composition of Matter patent in China," said Martin Stogniew, Ph.D., Chief Development Officer at Oncoceutics